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What changed in ARS Pharmaceuticals, Inc.'s 10-K — 2024 vs 2025

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Paragraph-level year-over-year comparison of ARS Pharmaceuticals, Inc.'s 2024 and 2025 10-K annual filings, covering the Business, Risk Factors, Legal Proceedings, Cybersecurity, MD&A and Market Risk sections. Every new, removed and edited paragraph is highlighted side-by-side so you can see exactly what management changed in the 2025 report.

+727 added−736 removedSource: 10-K (2026-03-09) vs 10-K (2025-03-20)

Top changes in ARS Pharmaceuticals, Inc.'s 2025 10-K

727 paragraphs added · 736 removed · 469 edited across 6 sections

Item 1. Business

Business — how the company describes what it does

201 edited+80 added−140 removed273 unchanged
Biggest changeWe believe neffy’s and our intranasal epinephrine technology product candidates’ design, particularly the compact size and “no needle, no injection” delivery, eliminates needle-related apprehension and pain, improves portability and ease of use, is highly reliable, and will increase prescriptions for epinephrine, making it more likely that patients and caregivers will administer epinephrine sooner, achieve more rapid symptom relief, and prevent the allergic reaction from progressing to a level of severity that could lead to hospitalization or even death.
Biggest changeWe believe neffy’s and our intranasal epinephrine technology product candidates’ design, particularly the compact size and “no needle, no injection” delivery, eliminates needle-related apprehension and pain, improves portability and ease of use, is highly reliable, and will increase prescriptions for epinephrine, making it more likely that patients and caregivers will administer epinephrine sooner, achieve more rapid symptom relief, and prevent the allergic reaction from progressing to a level of severity that could lead to hospitalization or even death. 9 Table of Contents Our Approach neffy 2 mg is approved in the U.S, E.U., U.K., Japan, Australia, and China for the emergency treatment of Type I allergic reactions, including anaphylaxis, for adults and children who weigh 30 kg or greater; neffy 1 mg is approved in the U.S., Australia, and China for the emergency treatment of Type I allergic reactions, including anaphylaxis, for patients who are four years of age and older and weigh 15 kg to less than 30 kg. neffy and our intranasal epinephrine technology product candidates are designed to address the shortcomings of intra-muscular injectable devices.
Pursuant to the agreement, Aegis granted us an exclusive, worldwide, sublicensable license under patents and know-how relating to the INTRAVAIL drug delivery technology to research, develop, make (subject to Aegis supplying the INTRAVAIL drug delivery technology to us under a supply agreement), use, sell, offer for sale, import, and otherwise commercialize products incorporating epinephrine compounds (“Aegis Licensed Compounds”), including the neffy nasal spray.
Pursuant to the Aegis Agreement, Aegis granted us an exclusive, worldwide, sublicensable license under patents and know-how relating to the Intravail drug delivery technology to research, develop, make (subject to Aegis supplying the Intravail drug delivery technology to us under a supply agreement), use, sell, offer for sale, import, and otherwise commercialize products incorporating epinephrine compounds (“Aegis Licensed Compounds”), including the neffy nasal spray.
The agreement will continue until the expiration of the last-to-expire Aegis Royalty Term, unless sooner terminated. We have the right to terminate the agreement at any time after a specified notice period to Aegis.
The Aegis Agreement will continue until the expiration of the last-to-expire Aegis Royalty Term, unless sooner terminated. We have the right to terminate the Aegis Agreement at any time after a specified notice period to Aegis.
Pursuant to the agreement, we granted Alfresa (i) an exclusive, sublicensable license under our patents relating to neffy to develop, use and import epinephrine compositions (“Alfresa Licensed Compositions”) and related products (“Alfresa Licensed Products”) in Japan (the “Alfresa Territory”) and to promote, distribute, offer for sale and sell Alfresa Licensed Products in the Alfresa Territory, and (ii) a non-exclusive, sublicensable license to manufacture and commercialize Alfresa Licensed Products under the license described in clause (i), under our technology to make and have made Alfresa Licensed Compositions and Alfresa Licensed Products in and outside the Alfresa Territory solely for the purpose of exercising the license described in clause (i) in the Alfresa Territory.
Pursuant to the Alfresa Agreement, we granted Alfresa (i) an exclusive, sublicensable license under our patents relating to neffy to develop, use and import epinephrine compositions (“Alfresa Licensed Compositions”) and related products (“Alfresa Licensed Products”) in Japan (the “Alfresa Territory”) and to promote, distribute, offer for sale and sell Alfresa Licensed Products in the Alfresa Territory, and (ii) a non-exclusive, sublicensable license to manufacture and commercialize Alfresa Licensed Products under the license described in clause (i), under our technology to make and have made Alfresa Licensed Compositions and Alfresa Licensed Products in and outside the Alfresa Territory solely for the purpose of exercising the license described in clause (i) in the Alfresa Territory.
During the term of the agreement, (1) we and Alfresa are obligated to use commercially reasonable efforts to develop a Alfresa Licensed Product throughout the Alfresa Territory, and (2) Alfresa is obligated to use commercially reasonable efforts to (A) seek pricing and reimbursement approval, (B) seek and maintain regulatory approval for the Alfresa Licensed Products through the Alfresa Territory, and (C) market, promote and otherwise commercialize Alfresa Licensed Products in the field throughout the Alfresa Territory.
During the term of the Alfresa Agreement, (1) we and Alfresa are obligated to use commercially reasonable efforts to develop a Alfresa Licensed Product throughout the Alfresa Territory, and (2) Alfresa is obligated to use commercially reasonable efforts to (A) seek pricing and reimbursement approval, (B) seek and maintain regulatory approval for the Alfresa Licensed Products through the Alfresa Territory, and (C) market, promote and otherwise commercialize Alfresa Licensed Products in the field throughout the Alfresa Territory.
Alfresa has the right to terminate the agreement (1) at any time after a specified notice period to us, or (2) upon notice to us if a binding decision is rendered invalidating any of our patents.
Alfresa has the right to terminate the Alfresa Agreement (1) at any time after a specified notice period to us, or (2) upon notice to us if a binding decision is rendered invalidating any of our patents.
Pursuant to the agreement, we granted Pediatrix (i) an exclusive, royalty-bearing, sublicensable license under our patents relating to neffy to develop, use, register and import epinephrine compositions (“Pediatrix Licensed Compositions”) and related products (“Pediatrix Licensed Products”) in China, Macau, Hong Kong and Taiwan (the “Pediatrix Territory”) and to promote, offer for sale and sell Pediatrix Licensed Products in the Pediatrix Territory; and (ii) an exclusive, royalty-bearing, sublicensable license to manufacture Pediatrix Licensed Compositions and Pediatrix Licensed Products solely for the purpose of exercising the license described in clause (i) in the Pediatrix Territory.
Pursuant to the Pediatrix Agreement, we granted Pediatrix (i) an exclusive, royalty-bearing, sublicensable license under our patents relating to neffy to develop, use, register and import epinephrine compositions (“Pediatrix Licensed Compositions”) and related products (“Pediatrix Licensed Products”) in China, Macau, Hong Kong and Taiwan (the “Pediatrix Territory”) and to promote, offer for sale and sell Pediatrix Licensed Products in the Pediatrix Territory; and (ii) an exclusive, royalty-bearing, sublicensable license to manufacture Pediatrix Licensed Compositions and Pediatrix Licensed Products solely for the purpose of exercising the license described in clause (i) in the Pediatrix Territory.
The agreement will continue until the expiration of the last-to-expire Pediatrix Royalty Term. Pediatrix has the right to terminate the agreement at any time after a specified notice period to us.
The Pediatrix Agreement will continue until the expiration of the last-to-expire Pediatrix Royalty Term. Pediatrix has the right to terminate the Pediatrix Agreement at any time after a specified notice period to us.
Pursuant to the ALK Agreement, we granted to ALK a worldwide (other than the United States, Japan, mainland China, Hong Kong, Taiwan, Macau, Australia and New Zealand) (“ALK Territory”), exclusive license under certain of our patents and know-how to develop, manufacture and commercialize products containing epinephrine administered intranasally, including EURneffy (the tradename for neffy 2 mg in the European Union) (epinephrine nasal spray) (“Products”), for all human uses, including the immediate or emergency treatment of allergic reactions (including Type I) and anaphylaxis and urticaria, and other future indications as agreed by the parties.
Pursuant to the ALK Collaboration Agreement, we granted to ALK a worldwide (other than the United States, Japan, mainland China, Hong Kong, Taiwan, Macau, Australia and New Zealand) (“ALK Territory”), exclusive license under certain of our patents and know-how to develop, manufacture and commercialize products containing epinephrine administered intranasally, including EURneffy (the tradename for neffy 2 mg in the European Union) (epinephrine nasal spray) (“Products”), for all human uses, including the immediate or emergency treatment of allergic reactions (including Type I) and anaphylaxis and urticaria, and other future indications as agreed by the parties.
Prior to a change of control and outside of a set period of time after which we commence change of control negotiations, we may terminate the ALK Agreement with respect to all countries in the European Economic Area (“EEA”) upon prior written notice to ALK and payment of a termination fee that is the higher of an agreed mid-nine digit amount and the fair market value of the Products business in the EEA at the time of such termination.
Prior to a change of control and outside of a set period of time after which we commence change of control negotiations, we may terminate the ALK Collaboration Agreement with respect to all countries in the European Economic Area (“EEA”) upon prior written notice to ALK and payment of a termination fee that is the higher of an agreed mid-nine digit amount and the fair market value of the Products business in the EEA at the time of such termination.
Dodecyl maltoside or Intravail is purchased through our license agreement with Aegis Therapeutics, LLC from two manufacturers, Dr. Reddy Laboratories and Inalco, which are based in India and Italy, respectively. The unit dose sprayer device used to delivery drug product in neffy is produced by Aptar Pharma (“Aptar”) and Silgan Dispensing Systems (“Silgan”).
Dodecyl maltoside or Intravail is purchased through our license agreement with Aegis Therapeutics, LLC (“Aegis”) from two manufacturers, Dr. Reddy Laboratories and Inalco, which are based in India and Italy, respectively. The unit dose sprayer device used to delivery drug product in neffy is produced by Aptar Pharma (“Aptar”) and Silgan Dispensing Systems (“Silgan”).
Epinephrine injectable products have a reported shelf-life range from the date of product manufacture of 18 to 24 months, with a volume-weighted average shelf-life of approximately 22 to 23 months. Our FDA and EC label indicates that neffy 2.0 mg is stable at room temperature for 30 months at 25 o C.
Epinephrine injectable products have a reported shelf-life range from the date of product manufacture of 18 to 24 months, with a volume-weighted average shelf-life of approximately 22 to 23 months. Our FDA and EC label indicates that neffy 2 mg is stable at room temperature for 30 months at 25 o C.
Based on the current list price of neffy and our target total gross-to-net yield, we estimate that the initial addressable market opportunity of 6.5 million patients who have been prescribed epinephrine during 2020-2022 is approximately $3 billion in annual net sales for neffy .
Based on the current list price of neffy and our target total gross-to-net yield, we estimate that the initial addressable potential market opportunity of 6.5 million patients who have been prescribed epinephrine during 2020-2022 is approximately $3.5 billion in annual net sales for neffy .
If we develop any new intranasally administered product that contains epinephrine and files a new drug application in the United States for such product (“New Product”), upon ALK’s request such New Product will be included as a Product under the ALK Agreement, subject to ALK bearing the costs of development of such New Product for its licensed territory.
If we develop any new intranasally administered product that contains epinephrine and files a new drug application in the United States for such product (“New Product”), upon ALK’s request such New Product will be included as a Product under the ALK Collaboration Agreement, subject to ALK bearing the costs of development of such New Product for its licensed territory.
An MA for an orphan medicinal product may only include indications designated as orphan. For non-orphan indications treated with the same active pharmaceutical ingredient, a separate marketing authorization has to be sought. 47 Orphan medicinal product designation entitles an applicant to incentives such fee reductions or fee waivers, protocol assistance, and access to the centralized marketing authorization procedure.
An MA for an orphan medicinal product may only include indications designated as orphan. For non-orphan indications treated with the same active pharmaceutical ingredient, a separate marketing authorization has to be sought. Orphan medicinal product designation entitles an applicant to incentives such fee reductions or fee waivers, protocol assistance, and access to the centralized marketing authorization procedure.
In October 2023, the MHRA announced a new Notification Scheme for clinical trials which enables a more streamlined and risk-proportionate approach to initial clinical trial applications for Phase 4 and low-risk Phase 3 clinical trial applications. 49 Marketing authorizations in the UK are governed by the Human Medicines Regulations (SI 2012/1916), as amended.
In October 2023, the MHRA announced a new Notification Scheme for clinical trials which enables a more streamlined and risk-proportionate approach to initial clinical trial applications for Phase 4 and low-risk Phase 3 clinical trial applications. Marketing authorizations in the UK are governed by the Human Medicines Regulations (SI 2012/1916), as amended.
Over time, epinephrine is oxidized and loses potency resulting in a finite shelf-life, and the neffy solution inside the unit dose sprayer changes to an amber to brown color. Epinephrine is the API used in neffy . We intend to use Cambrex Profarmco (“Cambrex”) as one of our commercial sources for epinephrine API.
Over time, epinephrine is oxidized and loses potency resulting in a finite shelf-life, and the neffy solution inside the unit dose sprayer changes to an amber to brown color. Epinephrine is the API used in neffy . We use Cambrex Profarmco (“Cambrex”) as one of our commercial sources for epinephrine API.
Other types of changes to the approved product, such as adding new indications and additional labeling claims, are also subject to further FDA review and approval. 37 The FDA may withdraw approval if compliance with regulatory requirements and standards is not maintained or if problems occur after the product reaches the market.
Other types of changes to the approved product, such as adding new indications and additional labeling claims, are also subject to further FDA review and approval. The FDA may withdraw approval if compliance with regulatory requirements and standards is not maintained or if problems occur after the product reaches the market.
In the United States, dosing recommendations for epinephrine use by intra-muscular injection are from 0.1 mg to 0.5 mg depending on weight with repeat dosing administered as needed to control a severe allergic reaction. 0.1 mg, 0.15 mg and 0.3 mg are the approved doses for the epinephrine auto-injectors.
In the United States, dosing recommendations for epinephrine use by intra-muscular injection are from 0.1 mg to 0.5 mg depending on the patient’s weight with repeat dosing administered as needed to control a severe allergic reaction. 0.1 mg, 0.15 mg and 0.3 mg are the approved doses for the epinephrine auto-injectors.
However, companies may share truthful and not misleading information that is otherwise consistent with a product’s FDA-approved labelling. 38 Hatch-Waxman Act Section 505 of the FDCA describes three types of marketing applications that may be submitted to the FDA to request marketing authorization for a new drug.
However, companies may share truthful and not misleading information that is otherwise consistent with a product’s FDA-approved labelling. Hatch-Waxman Act Section 505 of the FDCA describes three types of marketing applications that may be submitted to the FDA to request marketing authorization for a new drug.
In a meta-analysis of 32 studies evaluating epinephrine injectable administration techniques, 23% to 35% of participants failed to achieve the correct administration technique following training. Further, there is variability in respect to whether auto-injector devices are able to reliably deliver a sufficient dose of epinephrine.
In a meta-analysis of 32 studies evaluating epinephrine injectable administration techniques, 23% to 35% of participants failed to achieve the correct administration technique following training. Further, there is variability with respect to whether auto-injector devices are able to reliably deliver a sufficient dose of epinephrine.
Manufacturing Agreement with Renaissance In September 2020, we entered into a manufacturing agreement with Renaissance Lakewood, LLC (“Renaissance”), which was subsequently amended in July 2023 and September 2024 (the “Renaissance Agreement”). Pursuant to the agreement, Renaissance agreed to manufacture for, and provide to us, neffy nasal unit dose sprays (“Renaissance Products”).
Manufacturing Agreement with Renaissance In September 2020, we entered into a manufacturing agreement with Renaissance Lakewood, LLC (“Renaissance”), which was subsequently amended in July 2023, September 2024 and July 2025 (the “Renaissance Agreement”). Pursuant to the agreement, Renaissance agreed to manufacture for, and provide to us, neffy nasal unit dose sprays (“Renaissance Products”).
The MHRA can provide guidance to a company that is unsure which set of regulatory rules to follow. 50 Pricing, Coverage and Reimbursement Reimbursement authorities in Europe may be more restrictive than payors in the United States. In Europe, pricing and reimbursement schemes vary widely from country to country.
The MHRA can provide guidance to a company that is unsure which set of regulatory rules to follow. Pricing, Coverage and Reimbursement Reimbursement authorities in Europe may be more restrictive than payors in the United States. In Europe, pricing and reimbursement schemes vary widely from country to country.
Mr. Lowenthal, our Co-Founder, President, Chief Executive Officer, and one of our directors, has more than 25 years of biotechnology and pharmaceutical development experience including leading the regulatory approvals of VALTOCO (diazepam nasal spray) and NARCAN (naloxone nasal spray). Dr.
Lowenthal, our Co-Founder, President, Chief Executive Officer, and one of our directors, has more than 25 years of biotechnology and pharmaceutical development experience including leading the regulatory approvals of VALTOCO (diazepam nasal spray) and NARCAN (naloxone nasal spray). Dr.
Under the agreement, Aegis received an upfront license fee of $50,000 and is entitled to receive development milestone payments of up to $3.95 million in aggregate and commercialization milestone payments up to $16.0 million in the aggregate for each Aegis Licensed Product.
Under the Aegis Agreement, Aegis received an upfront license fee of $50,000 in 2018 and is entitled to receive development milestone payments of up to $3.95 million in the aggregate and commercialization milestone payments up to $16.0 million in the aggregate for each Aegis Licensed Product.
Our registration stability studies demonstrate that neffy is stable at room temperature for up to 30 months, based on stability data meeting specifications with the 2.0 mg dose of neffy for 30 months and the 1.0 mg dose of neffy for 24 months.
Our registration stability studies demonstrate that neffy is stable at room temperature for up to 30 months, based on stability data meeting specifications with the 2 mg dose of neffy for 30 months and the 1 mg dose of neffy for 24 months.
Clinical Trials in the EU Similarly to the United States, the various phases of non-clinical and clinical research in the EU are subject to significant regulatory controls. 44 Non-clinical studies are performed to demonstrate the health or environmental safety of new chemical or biological substances.
Clinical Trials in the EU Similarly to the United States, the various phases of non-clinical and clinical research in the EU are subject to significant regulatory controls. Non-clinical studies are performed to demonstrate the health or environmental safety of new chemical or biological substances.
Either we or ALK may terminate the ALK Agreement in the case of the other party’s insolvency or in the event of an uncured material breach of the other party, except that we may not terminate the ALK Agreement for ALK’s material breach of its commercial diligence obligations.
Either we or ALK may terminate the ALK Collaboration Agreement in the case of the other party’s insolvency or in the event of an uncured material breach of the other party, except that we may not terminate the ALK Collaboration Agreement for ALK’s material breach of its commercial diligence obligations.
Additionally, appropriate packaging must be selected and tested and stability studies must be conducted to demonstrate that the product candidate does not undergo unacceptable deterioration over its shelf life. 35 U.S.
Additionally, appropriate packaging must be selected and tested and stability studies must be conducted to demonstrate that the product candidate does not undergo unacceptable deterioration over its shelf life. U.S.
Either party may terminate the agreement for uncured material breach of the other party, or upon notice for insolvency-related events of the other party that are not discharged within a defined time period.
Either party may terminate the Alfresa Agreement for uncured material breach of the other party, or upon notice for insolvency-related events of the other party that are not discharged within a defined time period.
To achieve this goal, we have assembled a management team with extensive experience in the development and commercialization of drugs, such as recently approved nasal sprays NARCAN (naloxone nasal spray) and VALTOCO (diazepam nasal spray). Our company was founded by Richard Lowenthal, M.S., MSEL, Robert Bell, Ph.D. and Sarina Tanimoto, M.D., MBA. Pratik Shah, Ph.D. was our first external investor.
To achieve this goal, we have assembled a management team with extensive experience in the development and commercialization of drugs, such as approved nasal sprays NARCAN (naloxone nasal spray) and VALTOCO (diazepam nasal spray). Our company was founded by Richard Lowenthal, M.S., MSEL, Robert Bell, Ph.D. and Sarina Tanimoto, M.D., MBA. Pratik Shah, Ph.D. was our first external investor. Mr.
ALK may terminate the ALK Agreement for convenience upon prior written notice or for a safety or regulatory concern. We may terminate the ALK Agreement in the event ALK makes certain challenges to certain of our patents.
ALK may terminate the ALK Collaboration Agreement for convenience upon prior written notice or for a safety or regulatory concern. We may terminate the ALK Collaboration Agreement in the event ALK makes certain challenges to certain of our patents.
There remains a significant unmet need for treatments for allergies that can produce Type I reactions. We are conducting clinical studies to support the expansion of labeling for neffy and our intranasal epinephrine technology product candidates to outpatient epinephrine use in other Type I allergy conditions such as urticaria for which epinephrine intra-muscular injectables are not approved.
For other conditions that can produce Type I reactions, there remains a significant unmet need. We are conducting clinical studies to support the expansion of labeling for neffy and our intranasal epinephrine technology product candidates to outpatient epinephrine use in other Type I allergy conditions such as urticaria for which epinephrine intra-muscular injectables are not approved.
However, this system can lead to overstated immune and inflammatory responses that results in adverse outcomes known as hypersensitivity reactions. Type I allergic reactions are potentially life-threatening hypersensitivity reactions that can occur within minutes following exposure to an allergen and need to be treated immediately to relieve troublesome symptoms, mitigate severity and avoid a potentially fatal event.
However, this system can lead to overstated immune and inflammatory responses that result in adverse outcomes known as hypersensitivity reactions. Type I allergic reactions are potentially life-threatening hypersensitivity reactions that can occur within minutes following exposure to an allergen and need to be treated immediately to relieve troublesome symptoms, mitigate severity and avoid a potentially fatal event.
ALK is obligated to use commercially reasonable efforts to obtain and maintain regulatory approval for Products through the European Commission and within specified countries within ALK’s territory.
ALK is obligated to use commercially reasonable efforts to obtain and maintain regulatory approval for Products through the European Commission and within specified countries within the ALK Territory.
For this and more comprehensive risks related to our intellectual property, please see “ Risk Factors—Risks Related to Our Intellectual Property. ” We also seek to protect our intellectual property in part by entering into confidentiality agreements with companies with whom we share proprietary and confidential information in the course of business discussions, and by having confidentiality terms in our agreements with our employees, consultants, scientific advisors, clinical investigators, and other collaborators and contractors and also by requiring our employees, commercial contractors, and certain consultants and investigators, to enter into invention assignment agreements that grant us ownership of any discoveries or inventions made by them while in our employ.
For this and more comprehensive risks related to our intellectual property, please see “ Risk Factors—Risks Related to Our Intellectual Property. ” 26 Table of Contents We also seek to protect our intellectual property in part by entering into confidentiality agreements with companies with whom we share proprietary and confidential information in the course of business discussions, and by having confidentiality terms in our agreements with our employees, consultants, scientific advisors, clinical investigators, and other collaborators and contractors and also by requiring our employees, commercial contractors, and certain consultants and investigators, to enter into invention assignment agreements that grant us ownership of any discoveries or inventions made by them while in our employ.
ALK Supply Agreement On November 9, 2024, in connection with the ALK Collaboration Agreement, ARS and ALK also entered into a commercial supply agreement (the “Supply Agreement”), under which ARS will supply ALK’s requirements (and ALK will purchase from ARS its requirements) of Products for five years for a specified supply price, after which ALK may elect to transition to itself or its contract manufacturer the manufacture and supply of Products.
ALK Supply Agreement On November 9, 2024, in connection with the ALK Collaboration Agreement, ARS and ALK also entered into a commercial supply agreement (the “ALK Supply Agreement”), under which ARS will supply ALK’s requirements (and ALK will purchase from ARS its requirements) of Products for five years for a specified supply price, after which ALK may elect to transition to itself or its contract manufacturer the manufacture and supply of Products.
On August 9, 2024, the FDA approved neffy 2 mg for the emergency treatment of Type I allergic reactions, including anaphylaxis, in adults and children who weigh 30 kg or greater. As a result, we initiated the commercial launch of neffy 2 mg in the United States, with product becoming available for shipment on September 23, 2024.
On August 9, 2024, the FDA approved neffy 2 mg for the emergency treatment of Type I allergic reactions, including anaphylaxis, in adults and children who weigh 30 kg or greater. As a result, we initiated the commercial launch of neffy 2 mg in the United States, with product becoming available in September 2024.
Robust PDs within a range comparable to injection products with no risk of accidental blood vessel injections PD responses including systolic blood pressure and heart rate were within normal physiologic changes and comparable to auto-injector products, with maximum changes less than EpiPen. neffy has no potential for the accidental blood vessel injections observed with injection products such as EpiPen, which can lead to rapid and high epinephrine exposures that cause rapid increases in systolic blood pressure and can lead to cerebral hemorrhage or other cardiovascular side effects.
Robust pharmacodynamics (“PD”) within a range comparable to injection products with no risk of accidental blood vessel injections PD responses including systolic blood pressure and heart rate were within normal physiologic changes and comparable to auto-injector products, with maximum changes less than EpiPen. neffy has no potential for the accidental blood vessel injections observed with injection products such as EpiPen, which can lead to rapid and high epinephrine exposures that cause rapid increases in systolic blood pressure and can lead to cerebral hemorrhage or other cardiovascular side effects.
We may also terminate the ALK Agreement if ALK commercializes a non-injectable epinephrine product or manufactures such a product in the United States.
We may also terminate the ALK Collaboration Agreement if ALK commercializes a non-injectable epinephrine product or manufactures such a product in the United States.
These limitations discourage patients and caregivers from carrying these devices and administering epinephrine in a timely manner. Both patient adoption and use of intra-muscular injection devices has been limited among eligible patients with severe Type I allergic reactions at risk of anaphylaxis.
These limitations discourage patients and caregivers from carrying these devices and administering epinephrine in a timely manner. Both patient adoption and use of intra-muscular injection devices have been limited among eligible patients with severe Type I allergic reactions at risk of anaphylaxis.
We are eligible to receive regulatory and development milestones of up to $20.0 million and commercial sales-based milestones of up to $300.0 million, provided that $55.0 million of such sales-based milestones are contingent upon us obtaining regulatory approval for the Product in Canada by a specified time.
We are eligible to receive regulatory and development milestones of up to $15.0 million and commercial sales-based milestones of up to $300.0 million, provided that $55.0 million of such sales-based milestones are contingent upon us obtaining regulatory approval for the Product in Canada by a specified time.
If third parties prepare and file patent applications in the United States that also claim technology to which we have rights, we may have to participate in interference or derivation proceedings in the U.S. Patent and Trademark Office (the “USPTO”) to determine priority of invention.
If third parties prepare and file patent applications in the U.S. that also claim technology to which we have rights, we may have to participate in interference or derivation proceedings in the U.S. Patent and Trademark Office (the “USPTO”) to determine priority of invention.
Under the ALK Agreement, we are obligated to transfer to ALK the existing marketing authorizations for the Products in ALK’s territory. We are also required to conduct certain development and regulatory activities for Products in support of obtaining further regulatory approval of Products in ALK’s territory, and will transfer such regulatory approvals to ALK.
Under the ALK Collaboration Agreement, we are obligated to transfer to ALK the existing marketing authorizations for the Products in the ALK Territory. We are also required to conduct certain development and regulatory activities for Products in support of obtaining further regulatory approval of Products in the ALK Territory, and will transfer such regulatory approvals to ALK.
Our corporate headquarters are located at 11682 El Camino Real, Suite 120, San Diego, California 92130, and our telephone number is (858) 771-9307. Our corporate website address is www.ars-pharma.com. Information contained on, or accessible through, our website shall not be deemed incorporated into and is not a part of this Annual Report on Form 10-K.
Our corporate headquarters are located at 11682 El Camino Real, Suite 300, San Diego, California 92130, and our telephone number is (858) 771-9307. Our corporate website address is ars-pharma.com . Information contained on, or accessible through, our website shall not be deemed incorporated into and is not a part of this Annual Report on Form 10-K.
Using direct-to-consumer omnichannel strategies to drive awareness and patients asking for neffy , we believe we can quickly and efficiently reach a majority of the approximately 3.2 million patients in the United States who filled a prescription for an epinephrine intra-muscular injectable device in 2023, as well as the 3.3 million patients who have received a prescription, but either refused or discontinued treatment.
Using direct-to-consumer omnichannel strategies to drive awareness and patients asking for neffy , we believe we can reach a majority of the approximately 3.2 million patients in the United States who filled a prescription for an epinephrine intra-muscular injectable device in 2023, as well as the 3.3 million patients who have received a prescription, but refused or discontinued treatment.
We believe payers recognize the value and innovation of neffy for Type I allergic patients, including potential cost-savings to the healthcare system due to greater carriage and early use of epinephrine devices.
We believe payors recognize the value and innovation of neffy for Type I allergic patients, including potential cost-savings to the healthcare system due to greater carriage and early use of epinephrine devices.
Specifically, the Regulation, which is directly applicable in all EU Member States, introduces a streamlined application procedure through a single-entry point, the "EU portal", the Clinical Trials Information System (“CTIS”); a single set of documents to be prepared and submitted for the application; as well as simplified reporting procedures for clinical trial sponsors.
Specifically, the Regulation, which is directly applicable in all EU Member States, introduces a streamlined application procedure through a single-entry point, the “EU portal”, the Clinical Trials Information System (“CTIS”); a single set of documents to be prepared and submitted for the application; as well as simplified reporting procedures for clinical trial sponsors.
From 2010 to 2023, the number of epinephrine intra-muscular injectable devices sold in the United States has increased by approximately 6.5% annually based on IQVIA unit sales data, primarily due to the increasing size of the overall population affected by severe Type I allergies, led by food-based allergies. • Potential promotional lift due to new marketing and education efforts by a branded product such as neffy .
From 2010 to 2023, the number of epinephrine intra-muscular injectable devices sold in the U.S. has increased by approximately 6.5% annually based on IQVIA unit sales data, primarily due to the increasing size of the overall population affected by severe Type I allergies, led by food-based allergies. • Potential promotional lift due to new marketing and education efforts by a branded product such as neffy .
Marketing authorization holders and/or manufacturing and import authorization, or MA holders and/or distribution authorization holders may be subject to civil, criminal or administrative sanctions, including suspension of manufacturing authorization, in case of non-compliance with the EU or EU Member States’ requirements applicable to the manufacturing of medicinal products. Data and Market Exclusivity The EU also provides opportunities for market exclusivity.
Marketing authorization holders and/or manufacturing and import authorization, or MA holders and/or distribution authorization holders may be subject to civil, criminal or administrative sanctions, including suspension of manufacturing authorization, in case of non-compliance with the EU or EU Member States’ requirements applicable to the manufacturing of medicinal products. 44 Table of Contents Data and Market Exclusivity The EU also provides opportunities for market exclusivity.
An MA may be granted only to an applicant established in the EU. The centralized procedure provides for the grant of a single MA by the European Commission that is valid throughout the EEA (which is comprised of the 27 EU Member States plus Iceland, Liechtenstein and Norway).
An MA may be granted only to an applicant established in the EU. 42 Table of Contents The centralized procedure provides for the grant of a single MA by the European Commission that is valid throughout the EEA (which is comprised of the 27 EU Member States plus Iceland, Liechtenstein and Norway).
These obligations may include limiting personal data processing to only what is necessary for specified, explicit, and legitimate purposes; requiring a legal basis for personal data processing; requiring the appointment of a data protection officer in certain circumstances; increasing transparency obligations to data subjects; requiring data protection impact assessments in certain circumstances; limiting the collection and retention of personal data; increasing rights for data subjects; formalizing a heightened and codified standard of data subject consents; requiring the implementation and maintenance of technical and organizational safeguards for personal data; mandating notice of certain personal data breaches to the relevant supervisory authority(ies) and affected individuals; and mandating the appointment of representatives in the UK and/or the EU in certain circumstances.
These obligations may include limiting personal data processing to only what is necessary for specified, explicit, and legitimate purposes; requiring a legal basis for personal data processing; requiring the appointment of a data protection officer in certain circumstances; increasing transparency obligations to data subjects; requiring data protection impact assessments in certain circumstances; limiting the collection and retention of personal data; increasing rights for data subjects; formalizing a heightened and codified standard of data subject consents; requiring the implementation and maintenance of technical and organizational safeguards for personal data; mandating notice of certain personal data breaches to the relevant supervisory authority(ies) and affected individuals; and mandating the appointment of representatives in the UK and/or the EU in certain circumstances. 41 Table of Contents The U.S.
Newly discovered or developed safety or effectiveness data may require changes to a product’s approved labeling, including the addition of new warnings and contraindications, and also may require the implementation of other risk management measures. The FDA closely regulates the marketing, labeling, advertising and promotion of drug products.
Newly discovered or developed safety or effectiveness data may require changes to a product’s approved labeling, including the addition of new warnings and contraindications, and also may require the implementation of other risk management measures. 35 Table of Contents The FDA closely regulates the marketing, labeling, advertising and promotion of drug products.
Similar requirements may apply where the products are not co-packaged but the medicinal product information makes an explicit reference to a specific medical device. Medicinal Products in the United Kingdom The United Kingdom’s (“UK”), withdrawal from the EU on January 31, 2020, commonly referred to as Brexit, has changed the regulatory relationship between the UK and the EU.
Similar requirements may apply where the products are not co-packaged but the medicinal product information makes an explicit reference to a specific medical device. 46 Table of Contents Medicinal Products in the United Kingdom The United Kingdom’s (“UK”), withdrawal from the EU on January 31, 2020, commonly referred to as Brexit, has changed the regulatory relationship between the UK and the EU.
Needle containing intra-muscular injection products are known to be painful and cause reluctance to dose. 18 Easy to use No critical dosing errors during self-administration with 2.0 mg neffy by Type I allergy adult subjects (EPI-17).
Needle containing intra-muscular injection products are known to be painful and cause reluctance to dose. Easy to use No critical dosing errors during self-administration with 2.0 mg neffy by Type I allergy adult subjects.
Such patients experience multiple episodes each year, and we believe they would likely use multiple doses of our intranasal epinephrine technology product candidate each year to resolve their symptoms. Therefore, the market opportunity for treating flares in chronic spontaneous urticaria patients may be as meaningful as the Type I allergy including anaphylaxis indication.
Such patients experience multiple episodes each year, and we believe they would likely use multiple doses of our intranasal epinephrine technology product candidate each year to resolve their symptoms. Therefore, the market opportunity for treating flares in chronic spontaneous urticaria patients could be as large as the Type I allergy including anaphylaxis indication.
We made a $0.5 million milestone payment to Aegis upon the achievement of a regulatory milestone during 2019, a $1.0 million payment to Aegis upon the FDA’s acceptance of our U.S.
We made a $0.5 million milestone payment to Aegis upon the achievement of a regulatory milestone in 2019, a $1.0 million payment to Aegis upon the FDA’s acceptance of our U.S.
Generally, our activities in other countries will be subject to regulation that is similar in nature and scope as that imposed in the United States, although there can be important differences. Additionally, some significant aspects of regulation in Europe are addressed in a centralized way, but country-specific regulation remains essential in many respects.
Generally, our activities in other countries will be subject to regulation that is similar in nature and scope as that imposed in the U.S., although there can be important differences. Additionally, some significant aspects of regulation in Europe are addressed in a centralized way, but country-specific regulation remains essential in many respects.
Accidental injections to the hands or fingers of a caregiver or a child occur more than 3,500 times a year in the United States with epinephrine injection devices. • Less hesitation to dose epinephrine. Early administration of epinephrine can reduce the severity, risk of hospitalization and mortality associated with severe Type I allergic reactions.
Accidental injections to the hands or fingers of a caregiver or a child occur more than 3,500 times a year in the U.S. with epinephrine injection devices. • Less hesitation to dose epinephrine. Early administration of epinephrine can reduce the severity, risk of hospitalization and mortality associated with severe Type I allergic reactions.
There are approximately 1 million patients in the United States diagnosed with chronic spontaneous urticaria and treated with antihistamines or biologic treatments, such as Xolair, that still experience frequent flares, which is an indication for which epinephrine has never been formally developed as a prescription product, despite being used in-hospital to resolve such acute symptoms.
Development in Chronic Spontaneous Urticaria There are approximately 1 million patients in the U.S. diagnosed with chronic spontaneous urticaria and treated with antihistamines or biologic treatments, such as Xolair, that still experience frequent flares, which is an indication for which epinephrine has never been formally developed as a prescription product, despite being used in-hospital to resolve such acute symptoms.
Item 1. Business. As used in this Annual Report, unless the context indicates or otherwise requires, “ARS,” “ARS Pharma,” the “company,” “we,” “us,” “our,” and other similar terms refer to ARS Pharmaceuticals, Inc., a Delaware corporation and its consolidated subsidiaries. neffy is a trademark of ours that we use in this Annual Report.
Item 1. Business. As used in this Annual Report, unless the context indicates or otherwise requires, “ARS,” “ARS Pharma,” the “company,” “we,” “us,” “our,” and other similar terms refer to ARS Pharmaceuticals, Inc., a Delaware corporation and its consolidated subsidiaries. neffy and EURneffy are trademarks of ours that we use in this Annual Report.
We believe neffy could address the needs of the approximately 3.2 million patients in the United States who currently fill intra-muscular injectable prescriptions, the approximately 3.3 million former injectable patients in the United States that either refused to fill, or did not renew an intra-muscular injectable device prescription, and the more than 13.5 million eligible Type I allergy patients in the United States who are at risk of severe allergic reactions that are not prescribed an epinephrine product.
Market Opportunity We believe neffy could address the needs of the approximately 3.2 million patients in the U.S. who currently fill intra-muscular injectable prescriptions, the approximately 3.3 million people in the U.S. that either refused to fill, or did not renew an intra-muscular injectable device prescription, and the more than 13.5 million eligible Type I allergy patients in the United States who are at risk of severe allergic reactions that are not prescribed an epinephrine product.
We have entered into a commercial supply agreement with Renaissance Pharma, and believe they have sufficient capacity to satisfy our long-term requirements, although we are evaluating alternating sourcing options.
We have entered into a commercial supply agreement with Renaissance, and believe they have sufficient capacity to satisfy our long-term requirements, although we are evaluating alternative sourcing options.
An integrated data analysis graph summarizing the key outcomes for registration for both single and repeat doses of neffy is shown below. 16 Single doses of 2.0 mg neffy compared to single doses of approved 0.3 mg injection products Repeat doses of 2.0 mg neffy compared to repeat doses of approved 0.3 mg injection products 17 The hemodynamic response, measured by systolic blood pressure and heart rate, was observed even 1 minute after administration of neffy , and was comparable to some injection products including EpiPen, and was greater than 0.3 mg intra-muscular needle-with-syringe.
An integrated data analysis graph summarizing the key outcomes for registration for both single and repeat doses of neffy is shown below. 17 Table of Contents Single doses of 2.0 mg neffy compared to single doses of approved 0.3 mg injection products Repeat doses of 2.0 mg neffy compared to repeat doses of approved 0.3 mg injection products 18 Table of Contents The hemodynamic response, measured by systolic blood pressure and heart rate, was observed as soon as 1 minute after administration of neffy , and was comparable to some injection products including EpiPen, and was greater than 0.3 mg intra-muscular needle-with-syringe.
A low dose of epinephrine is important for safety as overexposure to epinephrine can lead to adverse events. 14 Limitations of Existing Injectable Epinephrine Products Epinephrine intra-muscular injectables have been proven to be highly effective if they are administered timely and effectively, and work as intended, but the limitations of these products include painful application, inconvenient size and a complicated mechanism of administration.
A low dose of epinephrine is important for safety as overexposure to epinephrine can lead to adverse events. 15 Table of Contents Limitations of Existing Injectable Epinephrine Products Epinephrine intra-muscular injectables have been proven to be highly effective if they are administered timely and effectively, but the limitations of these products include painful application, inconvenient size and a complicated mechanism of administration.
Either party may terminate the agreement (1) for uncured material breach of the other party, (2) upon notice for insolvency-related events of the other party that are not discharged within a defined time period, (3) on a product-by-product basis if the manufacture, distribution or sale would materially contravene any applicable law, (4) by providing the requisite notice if (a) the authorization and approval to distribute or sell Renaissance Product in the U.S. is not granted on or before a specified date, (b) the authorization and approval representing more than a certain number of units of Renaissance Product sold in the U.S. during the last calendar year is withdrawn by the FDA, or (c) we decided in our sole discretion to cease commercializing the Renaissance Product in the U.S., (5) in the case of a force majeure event that continues for six months or more, or (6) a violation by the other party of trade control or anti-corruption laws. 31 Supply Agreement with Ompi In October 2024, we entered into a supply agreement (the “Ompi Agreement”) with Nuova Ompi S.r.l.
Either party may terminate the agreement (1) for uncured material breach of the other party, (2) upon notice for insolvency-related events of the other party that are not discharged within a defined time period, (3) on a product-by-product basis if the manufacture, distribution or sale would materially contravene any applicable law, (4) by providing the requisite notice if (a) the authorization and approval to distribute or sell Renaissance Product in the U.S. is not granted on or before a specified date, (b) the authorization and approval representing more than a certain number of units of Renaissance Product sold in the U.S. during the last calendar year is withdrawn by the FDA, or (c) we decided in our sole discretion to cease commercializing the Renaissance Product in the U.S., (5) in the case of a force majeure event that continues for six months or more, or (6) a violation by the other party of trade control or anti-corruption laws.
For more information, see “ Risk Factors—Risks Related to Our Intellectual Property. ” 29 Our Collaboration and Licensing Agreements License Agreement with Aegis In June 2018, we entered into a license agreement with Aegis Therapeutics, LLC (“Aegis”), which was amended in July 2020 and January 2021.
For more information regarding the risks related to our intellectual property, see “ Risk Factors—Risks Related to Our Intellectual Property. ” Our Collaboration and Licensing Agreements License Agreement with Aegis In June 2018, we entered into a license agreement (the “Aegis Agreement”) with Aegis Therapeutics, LLC (“Aegis”), which was amended in July 2020 and January 2021.
Government authorities in the United States (at the federal, state and local level) and in other countries extensively regulate, among other things, the research, development, testing, manufacturing, quality control, approval, labeling, packaging, storage, record-keeping, promotion, advertising, distribution, post-approval monitoring and reporting, marketing and export and import of drug products such as those we are developing.
Government authorities in the United States (at the federal, state and local level) and in other countries extensively regulate, among other things, the research, development, testing, manufacturing, quality control, approval, labeling, packaging, storage, record-keeping, promotion, advertising, distribution, post-approval monitoring and reporting, marketing and export and import of drug products.
Either party may terminate the agreement for uncured material breach of the other party, or upon notice for insolvency-related events of the other party that are not discharged within a defined time period. Collaboration and License Agreement with Alfresa In April 2020, we entered into a collaboration and license agreement with Alfresa Pharma Corporation (“Alfresa”).
Either party may terminate the Aegis Agreement for uncured material breach of the other party, or upon notice for insolvency-related events of the other party that are not discharged within a defined time period. 27 Table of Contents Collaboration and License Agreement with Alfresa In April 2020, we entered into a collaboration and license agreement (the “Alfresa Agreement”) with Alfresa Pharma Corporation (“Alfresa”).
In reviewing the NDA for such a product, however, FDA reviewers would consult with their counterparts in the device center to ensure that the device component of the combination product – the sprayer - met applicable requirements regarding safety, effectiveness, durability and performance.
In reviewing the New Drug Application (“NDA”) for such a product, however, FDA reviewers would consult with their counterparts in the device center to ensure that the device component of the combination product – the sprayer - met applicable requirements regarding safety, effectiveness, durability and performance.
Bell, our Co-Founder and Chief Scientific Officer, has more than 25 years of product development experience including leading R&D at Barr Laboratories, Somerset Pharmaceuticals and UDL Laboratories. Dr.
Bell, our Co-Founder and Chief Scientific Officer, has more than 25 years of product development experience including leading research and development at Barr Laboratories, Inc., Somerset Pharmaceuticals, Inc. and UDL Laboratories, Inc. Dr.
A new drug must be approved by the FDA through the NDA process before it may be legally marketed in the United States. Drugs are also subject to other federal, state and local statutes and regulations.
Drug Development Process In the United States, the FDA regulates drugs under the FDCA, and implementing regulations. A new drug must be approved by the FDA through the NDA process before it may be legally marketed in the United States. Drugs are also subject to other federal, state and local statutes and regulations.
Further, there may be additional health reform measures. 42 Moreover, there has been heightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which has resulted in several U.S.
Further, there may be additional health reform measures. 39 Table of Contents Moreover, there has been heightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which has resulted in several U.S.
Shah, our Chairman, has more than 30 years of experience founding and leading biopharmaceutical companies and healthcare investment decisions including his role as Chairman and Chief Executive Officer of Design Therapeutics, former Chairman of Synthorx (now part of Sanofi) and former Chief Executive Officer of Auspex Pharmaceuticals (now part of Teva Pharmaceuticals).
Shah, our Chairman, has more than 30 years of experience founding and leading biopharmaceutical companies and healthcare investment decisions including his role as Chairman and Chief Executive Officer of Design Therapeutics, Inc., former Chairman of Synthorx, Inc. (now part of Sanofi S.A.) and former Chief Executive Officer of Auspex Pharmaceuticals, Inc. (now part of Teva Pharmaceutical Industries Ltd.).
Product candidates that we develop must be approved by the FDA, before they may be legally marketed in the United States and by the appropriate foreign regulatory agency before they may be legally marketed in foreign countries.
Product candidates that we develop must be approved by the FDA, before they may be legally marketed in the U.S. and by the appropriate foreign regulatory agency before they may be legally marketed in foreign countries.
Presidential executive orders, congressional inquiries, and proposed and enacted legislation designed, among other things, to bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs and reform government program reimbursement methodologies for pharmaceutical products. For example, the IRA, among other things, (i) directs the U.S.
Presidential executive orders, congressional inquiries, and proposed and enacted legislation designed, among other things, to bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs and reform government program reimbursement methodologies for pharmaceutical products.
Intellectual Property We strive to protect our intranasal epinephrine product candidates by seeking, maintaining, and defending our patent rights in the United States and internationally.
Intellectual Property We strive to protect our intranasal epinephrine product candidates by seeking, maintaining, and defending our patent rights in the U.S. and internationally.
We anticipate that our promotional reach will eventually exceed greater than 80% of the current epinephrine prescriptions in the United States through a combination of sales force expansion by early 2026 and non-personal promotion that includes continuing medical education programs in collaboration with allergist societies, speaker bureaus, peer-to-peer programs and participation in regional and national medical conferences. • Continue to obtain payer coverage and grow sales of neffy.
We anticipate that our promotional reach will eventually exceed greater than 80% of the current epinephrine prescriptions in the U.S. through a combination of continued sales force expansion and non-personal promotion that includes continuing medical education programs in collaboration with allergist societies, speaker bureaus, peer-to-peer programs and participation in regional and national medical conferences. • Continue to obtain payor coverage and grow sales of neffy .
We expect this to be especially true for neffy , given that 99% of the physicians surveyed in our quantitative market research studies indicated that they would prescribe neffy if asked by a patient and approximately 70% of physicians would recommend neffy .
We expect this to be especially true for neffy , given that 99% of the physicians surveyed in our quantitative market research studies indicated that they would prescribe neffy if asked by a patient and approximately 70% of physicians would recommend neffy assuming equal market access conditions as epinephrine autoinjectors.
Delivery of higher exposures of epinephrine increases the risk of overexposure and potential adverse events including gastrointestinal (GI) symptoms due to swallowing of the excess epinephrine that is not absorbed. neffy and our intranasal epinephrine technology product candidates have high bioavailability matching the approved doses of injection at a low dose of 2.0 or 1.0 mg intranasally.
Higher exposures of epinephrine increase the risk of overexposure and potential adverse events including gastrointestinal (“GI”) symptoms due to swallowing excess epinephrine that is not absorbed, if delivered orally. neffy and our intranasal epinephrine technology product candidates have high bioavailability matching the approved doses of injection at a low dose of 2 mg or 1 mg intranasally.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeTo the extent neffy is not commercially successful, our business, financial condition and results of operations would be materially adversely affected, and the price of our common stock would likely decline. neffy is our only product that has been approved for sale and it has only been approved in the United States for the emergency treatment of Type I allergic reactions, including anaphylaxis, in patients who are four years of age and older and weigh 15 kg to less than 30 kg (in the case of neffy 1 mg), and in adults and children who weigh 30 kg or greater (in the case of neffy 2 mg), and in the EU (under the trade name EURneffy , and only in the 2 mg form) for the emergency treatment of allergic reactions (anaphylaxis) due to insect stings or bites, foods, medicinal products and other allergens as well as idiopathic or exercise induced anaphylaxis, for adults and children with a body weight 30 kg or greater.
Biggest changeTo the extent neffy and EURneffy are not commercially successful, our business, financial condition and results of operations would be materially adversely affected, and the price of our common stock would likely decline. neffy is our only product that has been approved for sale.
The future regulatory and commercial success of neffy and our current or future intranasal epinephrine technology product candidates for additional indications is subject to a number of risks, including the following: • successful completion of nonclinical studies and clinical trials; • successful patient enrollment in clinical trials; • successful data from our nonclinical studies and clinical trials that support an acceptable risk-benefit profile of our intranasal epinephrine technology in the intended populations and indications; • satisfaction of applicable regulatory requirements, including to satisfy applicable rules governing combination products; • potential unforeseen safety issues or adverse side effects; • receipt and maintenance of marketing approvals from applicable regulatory authorities; • remaining in compliance with post-marketing regulatory requirements; • obtaining and maintaining patent and trade secret protection and regulatory exclusivity for our intranasal epinephrine technology; • making arrangements or maintaining existing arrangements with third-party manufacturers, or establishing manufacturing capabilities, for both clinical and commercial supplies of our intranasal epinephrine technology; • entry into collaborations to further the development of neffy and our current and future intranasal epinephrine technology product candidates in other jurisdictions or for additional indications; • continuing to grow our sales, marketing and distribution capabilities and commercializing any approved products, whether alone or in collaboration with others; • successfully commercializing neffy and our current and future intranasal epinephrine technology product candidates; • acceptance by patients, the medical community and third-party payors of neffy and our current and future intranasal epinephrine technology product candidates; • obtaining and maintaining third-party coverage and adequate reimbursement; • products, following approval, maintaining a continued acceptable safety profile; • effectively competing with other therapies; • ensuring that we promote and distribute our products consistent with all applicable healthcare laws; and • enforcing and defending intellectual property rights and claims.
The future regulatory and commercial success of our current or future intranasal epinephrine technology product candidates and neffy for additional indications is subject to a number of risks, including the following: • successful completion of nonclinical studies and clinical trials; • successful patient enrollment in clinical trials; • successful data from our nonclinical studies and clinical trials that support an acceptable risk-benefit profile of our intranasal epinephrine technology in the intended populations and indications; • satisfaction of applicable regulatory requirements, including to satisfy applicable rules governing combination products; • potential unforeseen safety issues or adverse side effects; • receipt and maintenance of marketing approvals from applicable regulatory authorities; • remaining in compliance with post-marketing regulatory requirements; • obtaining and maintaining patent and trade secret protection and regulatory exclusivity for our intranasal epinephrine technology; • making arrangements or maintaining existing arrangements with third-party manufacturers, or establishing manufacturing capabilities, for both clinical and commercial supplies of our intranasal epinephrine technology; • entry into collaborations to further the development of neffy and our current and future intranasal epinephrine technology product candidates in other jurisdictions or for additional indications; • continuing to grow our sales, marketing and distribution capabilities and commercializing any approved products, whether alone or in collaboration with others; • successfully commercializing neffy and our current and future intranasal epinephrine technology product candidates; • acceptance by patients, the medical community and third-party payors of neffy and our current and future intranasal epinephrine technology product candidates; • obtaining and maintaining third-party coverage and adequate reimbursement; • products, following approval, maintaining a continued acceptable safety profile; • effectively competing with other therapies; • ensuring that we promote and distribute our products consistent with all applicable healthcare laws; and • enforcing and defending intellectual property rights and claims.
Negative or inconclusive impressions of the results from our earlier clinical trials of neffy for the emergency treatment of Type I allergic reactions or any other clinical trial or nonclinical studies in animals that we have conducted, could mandate repeated or additional nonclinical studies or clinical trials and could delay marketing approvals or result in changes to or delays in nonclinical studies or clinical trials of our current and future intranasal epinephrine technology product candidates, including urticaria.
Negative or inconclusive impressions of the results from our earlier clinical trials of neffy for the emergency treatment of Type I allergic reactions or any other clinical trial or nonclinical studies in animals that we have conducted, could mandate repeated or additional nonclinical studies or clinical trials and could delay marketing approvals or result in changes to or delays in nonclinical studies or clinical trials of our current and future intranasal epinephrine technology product candidates, including for urticaria.
Each EU Member State’s decision is communicated to the sponsor via the centralized EU portal. Once the clinical trial approved, clinical study development may proceed. The CTR foresaw a three-year transition period which ended on January 31, 2025.
Each EU Member State’s decision is communicated to the sponsor via the centralized EU portal. Once the clinical trial is approved, clinical study development may proceed. The CTR foresaw a three-year transition period which ended on January 31, 2025.
Although there are currently various mechanisms that may be used to transfer personal data from the EEA and UK to the United States in compliance with law, such as the EEA and UK’s standard contractual clauses, the UK’s International Data Transfer Agreement / Addendum, and the EU-U.S.
Although there are currently various mechanisms that may be used to transfer personal data from the EEA and UK to the United States in compliance with law, such as the EEA standard contractual clauses, the UK’s International Data Transfer Agreement / Addendum, and the EU-U.S.
Furthermore, while we intend to protect our intellectual property rights in our expected significant markets, we cannot ensure that we will be able to initiate or maintain similar efforts in all jurisdictions in which we may wish to market our product.
Furthermore, while we intend to protect our intellectual property rights in our expected significant markets, we cannot ensure that we will be able to initiate or maintain similar efforts in all jurisdictions in which we may wish to market our product.
Accordingly, our efforts to protect our intellectual property rights in such countries may be inadequate, which may have an adverse effect on our ability to successfully commercialize neffy and our current or future intranasal epinephrine technology product candidates in all of our expected significant foreign markets.
Accordingly, our efforts to protect our intellectual property rights in such countries may be inadequate, which may have an adverse effect on our ability to successfully commercialize neffy and our current or future intranasal epinephrine technology product candidates in all of our expected significant foreign markets.
We co-own or exclusively license patent applications in our portfolio relating to neffy and our current or future intranasal epinephrine technology product candidates that are pending at the patent offices in the United States, Europe, Japan, and other foreign jurisdictions, however, we cannot predict: • if and when patents may issue based on the patent applications we own, co-own or exclusively license; • the scope of protection of any patent issuing based on the patent applications we own, co-own or exclusively license; • whether the claims of any patent issuing based on the patent applications we own, co-own or exclusively license will provide protection against competitors, • whether or not third parties will find ways to invalidate or circumvent our patent rights; • whether or not others will obtain patents claiming aspects similar to those covered by the patent applications we own, co-own or exclusively license; • whether we will need to initiate litigation or administrative proceedings to enforce and/or defend our patent rights which will be costly whether we win or lose; • whether the patent applications that we own, co-own or exclusively license will result in issued patents with claims that cover neffy and our intranasal epinephrine technology product candidates or uses thereof; and/or • whether we may experience patent office interruption or delays to our ability to timely secure patent coverage to any potential additional indications or any future product candidates.
We own, co-own or exclusively license patent applications in our portfolio relating to neffy and our current or future intranasal epinephrine technology product candidates that are pending at the patent offices in the United States, Europe, Japan, and other foreign jurisdictions, however, we cannot predict: • if and when patents may issue based on the patent applications we own, co-own or exclusively license; • the scope of protection of any patent issuing based on the patent applications we own, co-own or exclusively license; • whether the claims of any patent issuing based on the patent applications we own, co-own or exclusively license will provide protection against competitors, • whether or not third parties will find ways to invalidate or circumvent our patent rights; • whether or not others will obtain patents claiming aspects similar to those covered by the patent applications we own, co-own or exclusively license; • whether we will need to initiate litigation or administrative proceedings to enforce and/or defend our patent rights which will be costly whether we win or lose; • whether the patent applications that we own, co-own or exclusively license will result in issued patents with claims that cover neffy and our intranasal epinephrine technology product candidates or uses thereof; and/or • whether we may experience patent office interruption or delays to our ability to timely secure patent coverage to any potential additional indications or any future product candidates.
We may also experience numerous unforeseen events during our clinical trials that could delay or prevent our ability to receive marketing approval or commercialize for our current and future intranasal epinephrine technology product candidate, including urticaria, including: • regulators, IRBs, ethics committees or other reviewing bodies may not authorize or issue positive opinions permitting us or our investigators to commence a clinical trial, or to conduct or continue a clinical trial at a prospective or specific trial site; • we may not reach an agreement on acceptable terms with prospective contract research organizations (“CROs”) and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; • a delay in receiving study or clinical trial material from outside the United States; • the number of subjects or patients required for clinical trials of our current and future intranasal epinephrine technology product candidates, including urticaria, may be larger than we anticipate, enrollment in these clinical trials may be insufficient or slower than we anticipate, and the number of clinical trials being conducted at any given time may be high and result in fewer available patients for any given clinical trial, or patients may drop out of these clinical trials at a higher rate than we anticipate; • our third-party contractors, including those manufacturing neffy or conducting clinical trials on our behalf, may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all; • we may have to amend clinical trial protocol(s) submitted to regulatory authorities or conduct additional studies to reflect changes in regulatory requirements or guidance, which we may be required to resubmit to an IRB or ethics committee and regulatory authorities for re-examination; • unforeseen safety events may occur during the course of a clinical trial and these events may result in the temporary suspension or termination of a clinical trial, or require urgent safety measures or restrictions to protect human subjects during the conduct of a clinical trial; • regulators, IRBs, ethics committees or other reviewing bodies may fail to approve or issue positive opinions or subsequently find fault with the manufacturing processes or facilities of third-party manufacturers with which we have entered and may enter into agreement for clinical and commercial supplies, or the supply or quality of our current and future intranasal epinephrine technology product candidates or other materials necessary to conduct clinical trials of our current and future intranasal epinephrine technology product candidates, including urticaria, may be insufficient, inadequate or not available at an acceptable cost, or we may experience interruptions in supply; and • the potential for policies or regulations of the FDA, the EMA, the EU or any other applicable foreign regulatory authorities to significantly change in a manner rendering our clinical data insufficient for approval.
We may also experience numerous unforeseen events during our clinical trials that could delay or prevent our ability to receive marketing approval or commercialize for our current and future intranasal epinephrine technology product candidates, including for urticaria, including: • regulators, IRBs, ethics committees or other reviewing bodies may not authorize or issue positive opinions permitting us or our investigators to commence a clinical trial, or to conduct or continue a clinical trial at a prospective or specific trial site; • we may not reach an agreement on acceptable terms with prospective contract research organizations (“CROs”) and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; • a delay in receiving study or clinical trial material from outside the United States; • the number of subjects or patients required for clinical trials of our current and future intranasal epinephrine technology product candidates, including for urticaria, may be larger than we anticipate, enrollment in these clinical trials may be insufficient or slower than we anticipate, and the number of clinical trials being conducted at any given time may be high and result in fewer available patients for any given clinical trial, or patients may drop out of these clinical trials at a higher rate than we anticipate; • our third-party contractors, including those manufacturing neffy or conducting clinical trials on our behalf, may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all; • we may have to amend clinical trial protocol(s) submitted to regulatory authorities or conduct additional studies to reflect changes in regulatory requirements or guidance, which we may be required to resubmit to an IRB or ethics committee and regulatory authorities for re-examination; • unforeseen safety events may occur during the course of a clinical trial and these events may result in the temporary suspension or termination of a clinical trial, or require urgent safety measures or restrictions to protect human subjects during the conduct of a clinical trial; • regulators, IRBs, ethics committees or other reviewing bodies may fail to approve or issue positive opinions or subsequently find fault with the manufacturing processes or facilities of third-party manufacturers with which we have entered and may enter into agreement for clinical and commercial supplies, or the supply or quality of our current and future intranasal epinephrine technology product candidates or other materials necessary to conduct clinical trials of our current and future intranasal epinephrine technology product candidates, including for urticaria, may be insufficient, inadequate or not available at an acceptable cost, or we may experience interruptions in supply; and • the potential for policies or regulations of the FDA, the EMA, the EU or any other applicable foreign regulatory authorities to significantly change in a manner rendering our clinical data insufficient for approval.
If we or the third parties with whom we work fail, or are perceived to have failed, to address or comply with applicable data privacy and security obligations, we could face significant consequences, including but not limited to: government enforcement actions (e.g., investigations, fines, penalties, audits, inspections, and similar); litigation (including class-action claims) and mass arbitration demands; additional reporting requirements and/or oversight; bans on processing personal data; orders to destroy or not use personal data; and imprisonment of company officials.
If we or the third parties with whom we work fail, or are perceived to have failed, to address or comply with applicable data privacy and security obligations, we could face significant consequences, including but not limited to: government enforcement actions (e.g., investigations, fines, penalties, audits, inspections, and similar); litigation (including class-action claims) and mass arbitration demands; additional reporting requirements and/or oversight; bans or restrictions on processing personal data; orders to destroy or not use personal data; and imprisonment of company officials.
Any subsequent applicant who files an ANDA seeking approval of a generic equivalent version of neffy or an NDA submitted under the 505(b)(2) regulatory pathway referencing neffy must make one of the following certifications to the FDA concerning the patents listed in the Orange Book for neffy : (a) the patents that are listed have expired; (b) the date on which such patents will expire; or (c) such patents are invalid or will not be infringed upon by the manufacture, use or sale of the generic equivalent version of neffy or the drug product submitted under the 505(b)(2) regulatory pathway referencing neffy .
Any applicant who files an ANDA seeking approval of a generic equivalent version of neffy or an NDA submitted under the 505(b)(2) regulatory pathway referencing neffy must make one of the following certifications to the FDA concerning the patents listed in the Orange Book for neffy : (a) the patents that are listed have expired; (b) the date on which such patents will expire; or (c) such patents are invalid or will not be infringed upon by the manufacture, use or sale of the generic equivalent version of neffy or the drug product submitted under the 505(b)(2) regulatory pathway referencing neffy .
While data from our studies of our intranasal epinephrine technology product candidates demonstrated nasally delivered epinephrine reached blood levels comparable to those of already approved epinephrine injectable products, we do not know whether any future clinical trials or studies that we may conduct will demonstrate adequate efficacy and safety necessary to result in obtaining regulatory approval to market our current and future intranasal epinephrine technology product candidates, including urticaria.
While data from our studies of our intranasal epinephrine technology product candidates demonstrated nasally delivered epinephrine reached blood levels comparable to those of already approved epinephrine injectable products, we do not know whether any future clinical trials or studies that we may conduct will demonstrate adequate efficacy and safety necessary to result in obtaining regulatory approval to market our current and future intranasal epinephrine technology product candidates, including for urticaria.
Even if we ultimately prevail in such infringement claims, the monetary cost of such litigation and the diversion of the attention of our management and scientific personnel could outweigh any benefit we receive as a result of the proceedings. neffy or our current or future intranasal epinephrine technology product candidates may face competition from generic inhalable epinephrine products sooner than expected, and our patents may be challenged.
Even if we ultimately prevail in such infringement claims, the monetary cost of such litigation and the diversion of the attention of our management and scientific personnel could outweigh any benefit we receive as a result of the proceedings. neffy or our current or future intranasal epinephrine technology product candidates may face competition from generic intranasal epinephrine products sooner than expected, and our patents may be challenged.
Significant clinical trial delays also could shorten any periods during which we may have the exclusive right to commercialize our current and future intranasal epinephrine technology product candidates, including urticaria, or allow our competitors to bring products to market before we do and impair our ability to successfully commercialize such product candidates, which may harm our business and results of operations.
Significant clinical trial delays also could shorten any periods during which we may have the exclusive right to commercialize our current and future intranasal epinephrine technology product candidates, including for urticaria, or allow our competitors to bring products to market before we do and impair our ability to successfully commercialize such product candidates, which may harm our business and results of operations.
Any of these occurrences could have a material adverse effect on our business, financial condition, results of operations, cash flows and prospects. 92 Competitors may infringe our patents, trademarks, copyrights or other intellectual property that relate to our research programs and product candidates, their respective methods of use, manufacture and formulations thereof.
Any of these occurrences could have a material adverse effect on our business, financial condition, results of operations, cash flows and prospects. Competitors may infringe our patents, trademarks, copyrights or other intellectual property that relate to our research programs and product candidates, their respective methods of use, manufacture and formulations thereof.
We face competition with respect to our current indications for our intranasal epinephrine technology, including neffy , and will face competition with respect to any future indications of our intranasal epinephrine technology or other product candidates that we may seek to develop or commercialize in the future from large pharmaceutical and biotechnology companies, specialty pharmaceutical and generic drug companies, academic institutions, government agencies and research institutions.
We also face competition with respect to our current indications for our intranasal epinephrine technology, including neffy , and will face competition with respect to any future indications of our intranasal epinephrine technology or other product candidates that we may seek to develop or commercialize in the future from large pharmaceutical and biotechnology companies, specialty pharmaceutical and generic drug companies, academic institutions, government agencies and research institutions.
All of these changes could increase our costs and otherwise adversely affect our business. Any delay in obtaining, or an inability to obtain, any regulatory approvals, as a result of Brexit or otherwise, would prevent us from commercializing our product candidates in the UK and restrict our ability to generate revenue and achieve and sustain profitability.
All of these changes could increase our costs and otherwise adversely affect our business. Any delay in obtaining, or an inability to obtain, any regulatory approvals, as a result of Brexit or otherwise, would prevent us from commercializing our product candidates in the UK and restrict our ability to generate revenue and achieve and sustain profitability in the future.
Other states are considering and may adopt similar laws. 78 Outside the United States, an increasing number of laws, regulations, and industry standards govern data privacy and security. For example, the EU’s General Data Protection Regulation (“EU GDPR”), the United Kingdom’s GDPR (“UK GDPR”) (collectively, “GDPR”) and Australia’s Privacy Act, impose strict requirements for processing personal data.
Other states are considering and may adopt similar laws. Outside the United States, an increasing number of laws, regulations, and industry standards govern data privacy and security. For example, the EU’s General Data Protection Regulation (“EU GDPR”), the United Kingdom’s GDPR (“UK GDPR”) (collectively, “GDPR”) and Australia’s Privacy Act, impose strict requirements for processing personal data.
Any of the above events could significantly harm our business, prospects, financial condition, and results of operations. 70 Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidate. We expect our expenses to increase in connection with our planned operations.
Any of the above events could significantly harm our business, prospects, financial condition, and results of operations. Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidate. We expect our expenses to increase in connection with our planned operations.
Any such supply disruptions, including disruptions in procuring items that are essential for our development activities and securing manufacturing slots for the products needed for such activities, could adversely impact our ability to initiate and complete nonclinical studies or clinical trials and generate sales of and revenue from neffy or our current or future intranasal epinephrine product candidates, which could have a material adverse effect on our business, financial condition, results of operations and growth prospects. 66 COVID-19 affected and other public health crises may in the future affect employees of third-party CROs located in affected geographies that we rely upon to carry out our clinical trials.
Any such supply disruptions, including disruptions in procuring items that are essential for our development activities and securing manufacturing slots for the products needed for such activities, could adversely impact our ability to initiate and complete nonclinical studies or clinical trials and generate sales of and revenue from neffy or our current or future intranasal epinephrine product candidates, which could have a material adverse effect on our business, financial condition, results of operations and growth prospects. 66 Table of Contents COVID-19 affected and other public health crises may in the future affect employees of third-party CROs located in affected geographies that we rely upon to carry out our clinical trials.
We entered into exclusive licensing and collaboration agreements for the development and commercialization of neffy with Alfresa Pharma Corporation in Japan; Pediatrix Therapeutics, Inc. in China, Macau, Hong Kong and Taiwan; CSL Seqirus in Australia and New Zealand; and ALK in all other unpartnered geographies outside the United States.
We entered into exclusive licensing and collaboration agreements for the development and commercialization of neffy with Alfresa Pharma Corporation in Japan; Pediatrix Therapeutics, Inc. in China, Macau, Hong Kong and Taiwan; Seqirus in Australia and New Zealand; and ALK in all other unpartnered geographies outside the United States.
If any of these actions were to occur, it could result in significant expense and delay or limit our ability to generate sales revenues. 54 Competitive products may reduce or eliminate the commercial opportunity for neffy or our current and future intranasal epinephrine technology product candidates.
If any of these actions were to occur, it could result in significant expense and delay or limit our ability to generate sales revenues. Competitive products may reduce or eliminate the commercial opportunity for neffy or our current and future intranasal epinephrine technology product candidates.
In addition, we may not have sufficient resources to bring these actions to a successful conclusion. 91 If we are found to infringe a third party’s intellectual property rights, we could be forced, including by court order, to cease developing, manufacturing or commercializing the infringing product candidate or product.
In addition, we may not have sufficient resources to bring these actions to a successful conclusion. If we are found to infringe a third party’s intellectual property rights, we could be forced, including by court order, to cease developing, manufacturing or commercializing the infringing product candidate or product.
In addition, the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the federal False Claims Act; • federal civil monetary penalties laws impose civil fines for, among other things, the offering or transfer of remuneration to a Medicare or state healthcare program beneficiary if the person knows or should know it is likely to influence the beneficiary’s selection of a particular provider, practitioner, or supplier of services reimbursable by Medicare or a state healthcare program, unless an exception applies; • HIPAA which prohibits, among other things, knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations, or promises, of any of the money or property owned by, or under the custody or control of, any healthcare benefit program, regardless or the payor (e.g., public or private), willfully obstructing a criminal investigation of a healthcare offense, and knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent statement in connection with the delivery of or payment for healthcare benefits, items or services; like the federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation; • the federal Physician Payment Sunshine Act, which requires manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to the government information related to payments or other “transfers of value” made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners) and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members; • HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (“HITECH”), and their respective implementing regulations, which impose obligations on “covered entities,” including certain healthcare providers, health plans, and healthcare clearinghouses, as well as their respective “business associates” and their covered subcontractors that create, receive, maintain or transmit individually identifiable health information for or on behalf of a covered entity, with respect to safeguarding the privacy, security and transmission of individually identifiable health information; 77 • federal price reporting laws require manufactures to calculate and report complex pricing metrics to government programs, where such reported prices may be used in the calculation of reimbursement and/or discounts on approved products; • federal and state consumer protection and unfair competition laws broadly regulate marketplace activities and activities that potentially harm consumers; and • analogous state and foreign laws and regulations of each of the laws described above, such as anti-kickback and false claims laws, that may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers; laws that require biotechnology companies to comply with the industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the government and may require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures; laws that require biotechnology companies to report information on the pricing of certain drug products; and laws require the registration or pharmaceutical sales representatives.
In addition, the government may assert that a claim including items or services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the federal False Claims Act; • federal civil monetary penalties laws impose civil fines for, among other things, the offering or transfer of remuneration to a Medicare or state healthcare program beneficiary if the person knows or should know it is likely to influence the beneficiary’s selection of a particular provider, practitioner, or supplier of services reimbursable by Medicare or a state healthcare program, unless an exception applies; • HIPAA which prohibits, among other things, knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations, or promises, of any of the money or property owned by, or under the custody or control of, any healthcare benefit program, regardless or the payor (e.g., public or private), willfully obstructing a criminal investigation of a healthcare offense, and knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false, fictitious or fraudulent statement in connection with the delivery of or payment for healthcare benefits, items or services; like the federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation; • the federal Physician Payment Sunshine Act, which requires manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) to report annually to the government information related to payments or other “transfers of value” made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners) and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members; • the Health Insurance Portability and Accountability Act (“HIPAA”), as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (“HITECH”), and their respective implementing regulations, which impose obligations on “covered entities,” including certain healthcare providers, health plans, and healthcare clearinghouses, as well as their respective “business associates” and their covered subcontractors that create, receive, maintain or transmit individually identifiable health information for or on behalf of a covered entity, with respect to safeguarding the privacy, security and transmission of individually identifiable health information; 79 Table of Contents • federal price reporting laws require manufactures to calculate and report complex pricing metrics to government programs, where such reported prices may be used in the calculation of reimbursement and/or discounts on approved products; • federal and state consumer protection and unfair competition laws broadly regulate marketplace activities and activities that potentially harm consumers; and • analogous state and foreign laws and regulations of each of the laws described above, such as anti-kickback and false claims laws, that may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers; laws that require biotechnology companies to comply with the industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the government and may require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures; laws that require biotechnology companies to report information on the pricing of certain drug products; and laws require the registration or pharmaceutical sales representatives.
Although we expect to vigorously defend our patents from infringement by third parties, there can be no assurances that we will be successful with respect to such defense or any other legal proceedings which may arise in the ordinary course of our business.
In general, although we expect to vigorously defend our patents from infringement by third parties, there can be no assurances that we will be successful with respect to such defense or any other legal proceedings which may arise in the ordinary course of our business.
This means that medicinal products that are tested and released in the UK must be retested and re-released when entering the EU market for commercial use. 74 On February 27, 2023, the UK Government and the European Commission reached a political agreement on the so-called “Windsor Framework”.
This means that medicinal products that are tested and released in the UK must be retested and re-released when entering the EU market for commercial use. On February 27, 2023, the UK Government and the European Commission reached a political agreement on the so-called “Windsor Framework”.
Without patent protection, we may be open to competition from generic versions of neffy . 89 We cannot ensure that patent rights relating to inventions described and claimed in our pending patent applications will issue or that patents based on our patent applications will not be challenged and rendered invalid and/or unenforceable.
Without patent protection, we may be open to competition from generic versions of neffy . We cannot ensure that patent rights relating to inventions described and claimed in our pending patent applications will issue or that patents based on our patent applications will not be challenged and rendered invalid and/or unenforceable.
Any of these occurrences may harm our business, financial condition, and prospects significantly. 61 In addition, clinical trials of our intranasal epinephrine technology product candidates are and have been conducted in carefully defined sets of patients who have agreed to enter into clinical trials.
Any of these occurrences may harm our business, financial condition, and prospects significantly. In addition, clinical trials of our intranasal epinephrine technology product candidates are and have been conducted in carefully defined sets of patients who have agreed to enter into clinical trials.
These competitor products may compete with our product, and our patents or other intellectual property rights may not be effective or sufficient to prevent them from competing. 86 Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions.
These competitor products may compete with our product, and our patents or other intellectual property rights may not be effective or sufficient to prevent them from competing. Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions.
Any termination, breach or expiration of any of these licensing or collaboration agreements could have a material adverse effect on our financial position by reducing or eliminating the potential for us to receive license fees, milestones and royalties.
Any termination, breach or expiration of some of these licensing or collaboration agreements could have a material adverse effect on our financial position by reducing or eliminating the potential for us to receive license fees, milestones and royalties.
We believe that comparisons from period to period of our financial results are not necessarily meaningful and should not be relied upon as indications of our future performance. 68 We have incurred significant losses since our inception.
We believe that comparisons from period to period of our financial results are not necessarily meaningful and should not be relied upon as indications of our future performance. We have incurred significant losses since our inception.
Although we try to ensure that our employees, consultants and independent contractors do not use the intellectual property, proprietary information, know-how or trade secrets of others in their work for us, we may become subject to claims that we caused an individual to breach the terms of his or her non-competition or non-solicitation agreement, or that we or these individuals have, inadvertently or otherwise, used or disclosed the alleged intellectual property, proprietary information, know-how or trade secrets of a current or former employer or competitor.
Although we try to ensure that our employees, consultants and independent contractors do not use the intellectual property, proprietary information, know-how or trade secrets of others in their work for us, we are, and may in the future, become subject to claims that we caused an individual to breach the terms of his or her non-competition or non-solicitation agreement, or that we or these individuals have, inadvertently or otherwise, used or disclosed the alleged intellectual property, proprietary information, know-how or trade secrets of a current or former employer or competitor.
These laws may further limit or restrict the advertising and promotion of our products to the general public, and may also impose limitations on our promotional activities with health care professionals. 72 In addition, later discovery of previously unknown side effects, adverse events or other problems with our products or their manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may yield various results, including: • restrictions on the manufacturing of such products; • restrictions on the labeling or marketing of such products; • restrictions on product distribution or use; • requirements to conduct post-marketing studies or clinical trials; • warning letters or untitled letters; • withdrawal of the products from the market; • refusal to approve pending applications or supplements to approved applications that we submit; • recall of products; • restrictions on coverage by third-party payors; • fines, restitution or disgorgement of profits or revenues; • exclusion from federal health care programs such as Medicare and Medicaid or comparable foreign programs; • suspension, variation or withdrawal of regulatory approvals; • refusal to permit the import or export of products; • product seizure; or • injunctions or the imposition of civil or criminal penalties.
These laws may further limit or restrict the advertising and promotion of our products to the general public, and may also impose limitations on our promotional activities with health care professionals. 73 Table of Contents In addition, later discovery of previously unknown side effects, adverse events or other problems with our products or their manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may yield various results, including: • restrictions on the manufacturing of such products; • restrictions on the labeling or marketing of such products; • restrictions on product distribution or use; • requirements to conduct post-marketing studies or clinical trials; • warning letters or untitled letters; • withdrawal of the products from the market; • refusal to approve pending applications or supplements to approved applications that we submit; • recall of products; • restrictions on coverage by third-party payors; • fines, restitution or disgorgement of profits or revenues; • exclusion from federal health care programs such as Medicare and Medicaid or comparable foreign programs; • suspension, variation or withdrawal of regulatory approvals; • refusal to permit the import or export of products; • product seizure; or • injunctions or the imposition of civil or criminal penalties.
Consequently, even if we achieve profitability in the future, we may not be able to utilize a material portion of our NOL carryforwards and other tax attributes, which could adversely affect our business, cash flow, financial condition or results of operations. 71 Risks Related to our Legal and Regulatory Environment neffy and our current or future intranasal epinephrine technology product candidates are subject to ongoing obligations and continued regulatory review, which may result in significant additional expense. neffy and our current or future intranasal epinephrine technology product candidates could be subject to post-marketing restrictions or withdrawal from the market and we, or any current or future licensing and collaboration partners, may be subject to substantial penalties if we, or they, fail to comply with regulatory requirements or if we, or they, experience unanticipated problems with our products following approval. neffy and our current or future intranasal epinephrine technology product candidates, as well as, among other things, the manufacturing processes, post-approval studies, labeling, post-approval pharmacovigilance monitoring, advertising and promotional activities for neffy , is subject to ongoing requirements of and review by the FDA, the EMA and other applicable regulatory authorities.
Consequently, even if we achieve profitability in the future, we may not be able to utilize a material portion of our NOL carryforwards and other tax attributes, which could adversely affect our business, cash flow, financial condition or results of operations. 72 Table of Contents Risks Related to our Legal and Regulatory Environment neffy and our current or future intranasal epinephrine technology product candidates are subject to ongoing obligations and continued regulatory review, which may result in significant additional expense. neffy and our current or future intranasal epinephrine technology product candidates could be subject to post-marketing restrictions or withdrawal from the market and we, or any current or future licensing and collaboration partners, may be subject to substantial penalties if we, or they, fail to comply with regulatory requirements or if we, or they, experience unanticipated problems with our products following approval. neffy and our current or future intranasal epinephrine technology product candidates, as well as, among other things, the manufacturing processes, post-approval studies, labeling, post-approval pharmacovigilance monitoring, advertising and promotional activities for neffy , is subject to ongoing requirements of and review by the FDA, the EMA and other applicable regulatory authorities.
Based on the initially approved indication for neffy , we anticipate that neffy will compete primarily against epinephrine intra-muscular injectable products, for the emergency treatment of Type I allergic reactions including EpiPen and its generics, which is marketed by Viatris, Inc. and Teva Pharmaceuticals, Inc.; Adrenaclick, which is marketed by Amneal Pharmaceuticals, Inc.; Auvi-Q, which is marketed by Kaleo, Inc.; and Symjepi, which is marketed by Sandoz, Inc., a Novartis division.
Based on the initially approved indications for neffy , we anticipate that neffy will compete primarily against epinephrine intra-muscular injectable products, for the emergency treatment of Type I allergic reactions including EpiPen and its generics, which is marketed by Viatris, Inc. and Teva Pharmaceuticals, Inc.; Adrenaclick, which is marketed by Amneal Pharmaceuticals, Inc.; Auvi-Q, which is marketed by Kaleo, Inc.; and Symjepi, which is marketed by Sandoz, Inc., a Novartis division.
We publish privacy policies, marketing materials, whitepapers, and other statements, such as statements related to compliance with certain certifications or self-regulatory principles, concerning data privacy, and security.
We publish privacy policies, marketing materials, and other statements, such as statements related to compliance with certain certifications or self-regulatory principles, concerning data privacy, and security.
If we are unable to maintain sufficient insurance coverage at an acceptable cost or to otherwise protect against potential product liability claims, it could prevent or inhibit the development and commercial production and sale of neffy or our current or future intranasal epinephrine technology product candidates, which could harm our business, financial condition, results of operations and prospects. 63 If our information technology systems or data, or those of third parties with whom we work, are or were compromised, we could experience adverse consequences resulting from such compromise, including but not limited to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; and other adverse consequences.
If we are unable to maintain sufficient insurance coverage at an acceptable cost or to otherwise protect against potential product liability claims, it could prevent or inhibit the development and commercial production and sale of neffy or our current or future intranasal epinephrine technology product candidates, which could harm our business, financial condition, results of operations and prospects. 62 Table of Contents If our information technology systems or data, or those of third parties with whom we work, are or were compromised, we could experience adverse consequences resulting from such compromise, including but not limited to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; and other adverse consequences.
If our operations are found to be in violation of any of the laws described above or any other governmental regulations that apply to us, we may be subject to significant penalties, including administrative, civil and criminal penalties, damages, fines, disgorgement, exclusion from governmental health care programs, a corporate integrity agreement or other agreement to resolve allegations of non-compliance, imprisonment, and the curtailment or restructuring of our operations, any of which could adversely affect our ability to operate our business and our financial results.
If our operations, or those of our partners and collaborators, are found to be in violation of any of the laws described above or any other governmental regulations that apply to us, we may be subject to significant penalties, including administrative, civil and criminal penalties, damages, fines, disgorgement, exclusion from governmental health care programs, a corporate integrity agreement or other agreement to resolve allegations of non-compliance, imprisonment, and the curtailment or restructuring of our operations, any of which could adversely affect our ability to operate our business and our financial results.
Physicians may not prescribe or patients may not use neffy or our current or future intranasal epinephrine technology product candidates for its approved indication or in accordance with neffy ’s our current or future intranasal epinephrine technology product candidates’ instructions or any warnings that identify known potential adverse effects, side effects, and patients who should not use neffy or our current or future intranasal epinephrine technology product candidates.
Physicians may not prescribe or patients may not use neffy or our current or future intranasal epinephrine technology product candidates for its approved indications or in accordance with neffy ’s our current or future intranasal epinephrine technology product candidates’ instructions or any warnings that identify known potential adverse effects, side effects, and patients who should not use neffy or our current or future intranasal epinephrine technology product candidates.
Any decreased use of neffy or our current or future intranasal epinephrine technology product candidates or limitation on our ability to export or sell neffy or our current or future intranasal epinephrine technology product candidates would likely adversely affect our business. 76 Our relationships with customers, health care professionals and third-party payors may be subject to applicable healthcare laws, which could expose us to penalties, including administrative, civil or criminal penalties, damages, fines, imprisonment, exclusion from participation in federal healthcare programs such as Medicare and Medicaid, reputational harm, the curtailment or restructuring of our operations and diminished future profits and earnings.
Any decreased use of neffy or our current or future intranasal epinephrine technology product candidates or limitation on our ability to export or sell neffy or our current or future intranasal epinephrine technology product candidates would likely adversely affect our business. 78 Table of Contents Our relationships with customers, health care professionals and third-party payors may be subject to applicable healthcare laws, which could expose us to penalties, including administrative, civil or criminal penalties, damages, fines, imprisonment, exclusion from participation in federal healthcare programs such as Medicare and Medicaid, reputational harm, the curtailment or restructuring of our operations and diminished future profits and earnings.
In any patent infringement proceeding, there is a risk that a court will decide that a patent that we own or have licensed is invalid or unenforceable, in whole or in part, and that we do not have the right to stop the other party from using the invention at issue.
In any patent infringement proceeding, there is a risk that a court will decide that a patent that we own, co-own or have exclusively licensed is invalid or unenforceable, in whole or in part, and that we do not have the right to stop the other party from using the invention at issue.
Additionally, there may be negative findings regarding components of neffy or our current or future intranasal epinephrine technology product candidates by other parties. Any negative findings by third parties may impact neffy for its initially approved indication and labeling, or the future approvability or labeling of our current or future intranasal epinephrine technology product candidates, including urticaria.
Additionally, there may be negative findings regarding components of neffy or our current or future intranasal epinephrine technology product candidates by other parties. Any negative findings by third parties may impact neffy for its initially approved indications and labeling, or the future approvability or labeling of our current or future intranasal epinephrine technology product candidates, including for urticaria.
We have registered and pending trademarks in the United States, as well as in several foreign jurisdictions, including the United Kingdom, EU, and Japan. We may not be able to obtain applicable corresponding health regulatory approval to use these trademarks for our product.
We have registered and pending trademarks in the United States, as well as in several foreign jurisdictions, including but not limited to the United Kingdom, EU, and Japan. We may not be able to obtain applicable corresponding health regulatory approval to use these trademarks for our product.
Remote work has become more common and has increased risks to our information technology systems and data, as more of our employees utilize network connections, computers, and devices outside our premises or network, including working at home, while in transit and in public locations.
Remote work has increased risks to our information technology systems and data, as more of our employees utilize network connections, computers, and devices outside our premises or network, including working at home, while in transit and in public locations.
The amount of time required for the FDA or a comparable foreign regulatory authority to qualify a new supplier and confirm that our manufacturing processes meet the necessary standards could cause delays in the manufacturing and marketing of neffy and our current or future intranasal epinephrine technology product candidates and could, depending on the particular product, have a material adverse effect on our results of operations and financial condition. 81 We may be unable to maintain or establish required agreements with third-party manufacturers or to do so on acceptable terms.
The amount of time required for the FDA or a comparable foreign regulatory authority to qualify a new supplier and confirm that our manufacturing processes meet the necessary standards could cause delays in the manufacturing and marketing of neffy and our current or future intranasal epinephrine technology product candidates and could, depending on the particular product, have a material adverse effect on our results of operations and financial condition. 84 Table of Contents We may be unable to maintain or establish required agreements with third-party manufacturers or to do so on acceptable terms.
The existence of these provisions could negatively affect the price of our common stock and limit opportunities for our stockholders to realize value in a corporate transaction. 100 Our amended and restated certificate of incorporation designates the state courts of the State of Delaware or, if no state court located within the State of Delaware has jurisdiction, the federal court for the District of Delaware, and the federal district courts of the United States of America to be the exclusive forums for substantially all disputes between us and our stockholders, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors, officers and employees.
The existence of these provisions could negatively affect the price of our common stock and limit opportunities for our stockholders to realize value in a corporate transaction. 104 Table of Contents Our amended and restated certificate of incorporation designates the state courts of the State of Delaware or, if no state court located within the State of Delaware has jurisdiction, the federal court for the District of Delaware, and the federal district courts of the United States of America to be the exclusive forums for substantially all disputes between us and our stockholders, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors, officers and employees.
Any disclosure, either intentional or unintentional, by our employees, the employees of third parties with whom we share our facilities or third-party consultants and vendors that we engage to perform research, clinical trials or manufacturing activities, or misappropriation by third parties (such as through a cybersecurity breach) of our trade secrets or proprietary information could enable competitors to duplicate or surpass our technological achievements, thus eroding our competitive position in our market. 95 Trade secrets and unpatented know-how can be difficult to protect.
Any disclosure, either intentional or unintentional, by our employees, the employees of third parties with whom we share our facilities or third-party consultants and vendors that we engage to perform research, clinical trials or manufacturing activities, or misappropriation by third parties (such as through a cybersecurity breach) of our trade secrets or proprietary information could enable competitors to duplicate or surpass our technological achievements, thus eroding our competitive position in our market. 99 Table of Contents Trade secrets and unpatented know-how can be difficult to protect.
These changes include aggregate reductions to Medicare payments to providers of up to two percent per fiscal year pursuant to the Budget Control Act of 2011, which went into effect on April 1, 2013, and due to subsequent legislative amendments, will remain in effect until 2032, unless additional Congressional action is taken.
For example, additional changes include aggregate reductions to Medicare payments to providers of up to two percent per fiscal year pursuant to the Budget Control Act of 2011, which went into effect on April 1, 2013, and due to subsequent legislative amendments, will remain in effect until 2032, unless additional Congressional action is taken.
If any of these third parties fail to meet expected deadlines, adhere to our clinical protocols or meet regulatory requirements, or otherwise performs in a substandard manner, our clinical trials may be extended, delayed or terminated.
If any of these third parties fail to meet expected deadlines, adhere to our clinical protocols or meet regulatory requirements, or otherwise perform in a substandard manner, our clinical trials may be extended, delayed or terminated.
If we or our collaboration partners fail to comply with the regulatory requirements in international markets and/or obtain and maintain applicable marketing approvals, our target market will be reduced and our ability to realize the full market potential of neffy will be harmed. 56 If we are unable to successfully develop neffy or our current or future intranasal epinephrine technology product candidates for additional indications, or experience significant delays in doing so, the commercial potential of neffy or our current or future intranasal epinephrine technology product candidates will be more limited.
If we or our collaboration partners fail to comply with the regulatory requirements in international markets and/or obtain and maintain applicable marketing approvals, our target market will be reduced and our ability to realize the full market potential of neffy will be harmed. 55 Table of Contents If we are unable to successfully develop our current or future intranasal epinephrine technology product candidates, or neffy for additional indications, or experience significant delays in doing so, the commercial potential of our current or future intranasal epinephrine technology product candidates or neffy will be more limited.
We take steps designed to detect, mitigate and remediate vulnerabilities in our information systems (such as our hardware and/or software, including that of third parties with whom we work). We may not, however, detect and remediate all such vulnerabilities, including on a timely basis. Vulnerabilities could be exploited and result in a security incident.
We take steps designed to detect, mitigate and remediate vulnerabilities in our information systems (such as our hardware and/or software, including that of third parties with whom we work). We have not and may not in the future, however, detect and remediate all such vulnerabilities, including on a timely basis. Vulnerabilities could be exploited and result in a security incident.
Further, even if any product candidate we develop or acquire was to receive marketing approval, we would continue to bear the risks that the FDA or similar foreign regulatory authorities could revoke, vary or suspend approval of our product candidate or that safety, efficacy, manufacturing or supply issues could arise with such product candidate. 60 The results of early-stage clinical trials and preclinical studies may not be predictive of future results.
Further, even if any product candidate we develop or acquire was to receive marketing approval, we would continue to bear the risks that the FDA or similar foreign regulatory authorities could revoke, vary or suspend approval of our product candidate or that safety, efficacy, manufacturing or supply issues could arise with such product candidate. 59 Table of Contents The results of early-stage clinical trials and preclinical studies may not be predictive of future results.
While we may litigate to defend against these claims, even if we are successful, litigation could result in substantial costs and could be a distraction to management and other employees.
While we are, and may in the future, litigate to defend against these claims, even if we are successful, litigation could result in substantial costs and could be a distraction to management and other employees.
If later stage clinical trials do not produce favorable results that meet regulatory authority criteria, our ability to obtain regulatory approval for our current and future intranasal epinephrine technology product candidate, including urticaria, may be adversely impacted. 59 Our failure to successfully initiate and complete clinical trials of our current and future intranasal epinephrine technology product candidates, including urticaria, and to demonstrate the efficacy and safety necessary to obtain regulatory approval to market our current and future intranasal epinephrine technology product candidates, including urticaria, would significantly harm our business.
If later stage clinical trials do not produce favorable results that meet regulatory authority criteria, our ability to obtain regulatory approval for our current and future intranasal epinephrine technology product candidate, including for urticaria, may be adversely impacted. 58 Table of Contents Our failure to successfully initiate and complete clinical trials of our current and future intranasal epinephrine technology product candidates, including for urticaria, and to demonstrate the efficacy and safety necessary to obtain regulatory approval to market our current and future intranasal epinephrine technology product candidates, including for urticaria, would significantly harm our business.
A significant disruption at any of these facilities or otherwise within our supply chain, even on a short-term basis, could impair our ability to produce and ship products to the market on a timely basis or at all, which could have a material adverse effect on our business, financial position, and results of operations. 83 We rely on third parties to conduct our nonclinical studies and clinical trials.
A significant disruption at any of these facilities or otherwise within our supply chain, even on a short-term basis, could impair our ability to produce and ship products to the market on a timely basis or at all, which could have a material adverse effect on our business, financial position, and results of operations. 86 Table of Contents We rely on third parties to conduct our nonclinical studies and clinical trials.
Our current and anticipated future dependence upon others for the raw materials associated with, and the manufacture of neffy and our current or future intranasal epinephrine technology product candidates may adversely affect our profit margins and our ability to successfully commercialize neffy or our current or future intranasal epinephrine technology product candidates on a competitive basis. 82 We are dependent on international third-party licensees and assignees for the development and commercialization of neffy and our current and future intranasal epinephrine technology product candidates outside the United States.
Our current and anticipated future dependence upon others for the raw materials associated with, and the manufacture of neffy and our current or future intranasal epinephrine technology product candidates may adversely affect our profit margins and our ability to successfully commercialize neffy or our current or future intranasal epinephrine technology product candidates on a competitive basis. 85 Table of Contents We are dependent on international third-party licensees and assignees for the development and commercialization of neffy and our current and future intranasal epinephrine technology product candidates outside the United States.
As a result, our patent portfolio may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours. The patents we currently co-own or exclusively license for neffy and our intranasal epinephrine technology product candidates are expected to expire as early as 2038, absent any patent term adjustments.
As a result, our patent portfolio may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours. 92 Table of Contents The patents we currently own, co-own or exclusively license for neffy and our intranasal epinephrine technology product candidates are expected to expire as early as 2038, absent any patent term adjustments.
Receiving priority review from the FDA does not guarantee approval within the six-month review cycle or at all. 62 Product liability lawsuits against us or any of our current and future licensing and collaboration partners could divert our resources and attention, cause us to incur substantial liabilities and limit commercialization of neffy or our current or future intranasal epinephrine technology product candidates.
Receiving priority review from the FDA does not guarantee approval within the six-month review cycle or at all. 61 Table of Contents Product liability lawsuits against us or any of our current and future licensing and collaboration partners could divert our resources and attention, cause us to incur substantial liabilities and limit commercialization of neffy or our current or future intranasal epinephrine technology product candidates.
In such cases, we may decide that the more prudent course of action is to simply monitor the situation or initiate or seek some other non-litigious action or solution. Intellectual property litigation may lead to unfavorable publicity that harms our reputation.
In such cases, we may decide that the more prudent course of action is to simply monitor the situation or initiate or seek some other non-litigious action or solution. 97 Table of Contents Intellectual property litigation may lead to unfavorable publicity that harms our reputation.
Though we carefully manage our relationships with our CROs, investigators and other third parties, there can be no assurance that we will not encounter challenges or delays in the future or that these delays or challenges will not have a material adverse impact on our business, financial condition and prospects. 84 Our reliance on third parties requires us to share our trade secrets, know-how and other proprietary information, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed.
Though we carefully manage our relationships with our CROs, investigators and other third parties, there can be no assurance that we will not encounter challenges or delays in the future or that these delays or challenges will not have a material adverse impact on our business, financial condition and prospects. 87 Table of Contents Our reliance on third parties requires us to share our trade secrets, know-how and other proprietary information, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed.
If we are unable to effectively manage our recent and expected growth, our ability to generate revenues or achieve future profitability could be reduced and we may not be able to implement our business strategy, including the successful commercialization of neffy . 98 Risks Related to the Securities Markets and Ownership of Our Common Stock The market price of our common stock could be volatile.
If we are unable to effectively manage our recent and expected growth, our ability to generate revenues or achieve future profitability could be reduced and we may not be able to implement our business strategy, including the successful commercialization of neffy . 102 Table of Contents Risks Related to the Securities Markets and Ownership of Our Common Stock The market price of our common stock could be volatile.
These broad market fluctuations may also adversely affect the trading price of our common stock. In the past, following periods of volatility in the market price of a company’s securities, stockholders have often instituted class action securities litigation against those companies.
These broad market fluctuations may also adversely affect the trading price of our common stock. 103 Table of Contents In the past, following periods of volatility in the market price of a company’s securities, stockholders have often instituted class action securities litigation against those companies.
Further, we expect to incur additional costs associated with operating as a public company. We may require significant additional amounts of cash in order to commercialize neffy for its currently approved indication in the United States, or for our current or future intranasal epinephrine technology product candidates which receives regulatory approval.
Further, we expect to incur additional costs associated with operating as a public company. We may require significant additional amounts of cash in order to commercialize neffy for its currently approved indications in the United States, or for our current or future intranasal epinephrine technology product candidates which receive regulatory approval.
Though these license agreements may provide guidelines for how our trademarks and trade names may be used, a breach of these agreements or misuse of our trademarks and trade names by our licensees may jeopardize our rights in or diminish the goodwill associated with our trademarks and trade names. 94 Intellectual property rights do not necessarily address all potential threats to our competitive advantage.
Though these license agreements may provide guidelines for how our trademarks and trade names may be used, a breach of these agreements or misuse of our trademarks and trade names by our licensees may jeopardize our rights in or diminish the goodwill associated with our trademarks and trade names. 98 Table of Contents Intellectual property rights do not necessarily address all potential threats to our competitive advantage.
As a result, our headcount has increased from 23 full-time employees and 5 part-time employees as of July 31, 2024 to 155 full-time employees and 5 part-time employees as of December 31, 2024. We may need to further expand our headcount in the future to support our growth strategy.
As a result, our headcount has increased from 23 full-time employees and 5 part-time employees as of July 31, 2024 to 158 full-time employees and 5 part-time employees as of December 31, 2025. We may need to further expand our headcount in the future to support our growth strategy.
If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business and results of operations, including the imposition of civil, criminal and administrative penalties, damages, monetary fines, disgorgement, integrity oversight and reporting obligations, possible exclusion from participation in Medicare, Medicaid and other federal healthcare programs or comparable foreign programs, contractual damages, reputational harm, diminished profits and future earnings, and curtailment of our operations, any of which could have a material adverse effect on our ability to operate our business and our results of operations. 97 We may encounter difficulties in managing our growth, which could disrupt our operations.
If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business and results of operations, including the imposition of civil, criminal and administrative penalties, damages, monetary fines, disgorgement, integrity oversight and reporting obligations, possible exclusion from participation in Medicare, Medicaid and other federal healthcare programs or comparable foreign programs, contractual damages, reputational harm, diminished profits and future earnings, and curtailment of our operations, any of which could have a material adverse effect on our ability to operate our business and our results of operations.
We also rely on our licensing and collaboration partners, our CROs, third-party logistics providers, distributors and other contractors and consultants to utilize information technology systems and networks to process, transmit and store electronic information in connection with our business activities, including in connection with our clinical trials. 64 Our ability to monitor these third parties’ information security practices is limited, and these third parties may not have adequate information security measures in place.
We also rely on our licensing and collaboration partners, our CROs, third-party logistics providers, distributors and other contractors and consultants to utilize information technology systems and networks to process, transmit and store electronic information in connection with our business activities, including in connection with our clinical trials. 63 Table of Contents Our ability to monitor these third parties’ information security practices is limited, and these third parties may not have adequate information security measures in place.
In order to successfully commercialize neffy , we must continue to build our sales, marketing, distribution, managerial and other non-technical capabilities.
In order to successfully commercialize neffy , we must, among other things, continue to build our sales, marketing, distribution, managerial and other non-technical capabilities.
General Risk Factors If equity research analysts do not publish research or reports, or publish unfavorable research or reports, about us, our business or our market, our stock price and trading volume could decline. The trading market for our common stock will be influenced by the research and reports that equity research analysts publish about us and our business.
If equity research analysts do not publish research or reports, or publish unfavorable research or reports, about us, our business or our market, our stock price and trading volume could decline. The trading market for our common stock will be influenced by the research and reports that equity research analysts publish about us and our business.
Some of the factors that may cause the market price of our common stock to fluctuate include: • failure to meet or exceed financial and development projections we may provide to the public; • failure to meet or exceed the financial and development projections of the investment community; • our ability to maintain regulatory approval for neffy , or obtain regulatory approvals for additional indications; • failure of neffy or our current or future intranasal epinephrine technology product candidates, to achieve commercial success; • failure by us to maintain our existing third-party license and supply agreements; • failure by us or our licensors to prosecute, maintain, or enforce our intellectual property rights; • changes in laws or regulations applicable to neffy or our current or future intranasal epinephrine technology product candidates; • any inability to obtain adequate supply of neffy or our current or future intranasal epinephrine technology product candidates or any of its components, or the inability to do so at acceptable prices; • adverse regulatory authority decisions; • introduction of new products, services or technologies by our competitors; • the perception of the pharmaceutical industry by the public, legislatures, regulators and the investment community; • announcements of significant acquisitions, strategic collaborations, joint ventures or capital commitments by us or our competitors; • disputes or other developments relating to proprietary rights, including patents, litigation matters, and our ability to obtain patent protection for neffy or our current or future intranasal epinephrine technology product candidates; • additions or departures of key personnel; • significant lawsuits, including patent or stockholder litigation; • if securities or industry analysts do not publish research or reports about our business, or if they issue an adverse or misleading opinion regarding our business and stock; • changes in the market valuations of similar companies; • general market or macroeconomic conditions, including as a result of actual or threatened tariffs and potential for trade wars; • sales of our common stock by us or our stockholders in the future; • trading volume of our common stock; • announcements by commercial partners or competitors of new commercial products, clinical progress or the lack thereof, significant contracts, commercial relationships or capital commitments; • adverse publicity generally, including with respect to other products and potential products in such markets; • the introduction of technological innovations or new therapies that compete with potential products of ours; • changes in the structure of health care payment systems; and • period-to-period fluctuations in our financial results. 99 Moreover, the stock markets in general have experienced substantial volatility that has often been unrelated to the operating performance of individual companies.
Some of the factors that may cause the market price of our common stock to fluctuate include: • failure to meet or exceed financial and development projections we may provide to the public; • failure to meet or exceed the financial and development projections of the investment community; • our ability to maintain regulatory approval for neffy , or obtain regulatory approvals for additional indications; • failure of neffy or our current or future intranasal epinephrine technology product candidates, to achieve commercial success; • failure by us to maintain our existing third-party license and supply agreements; • failure by us or our licensors to prosecute, maintain, or enforce our intellectual property rights; • changes in laws or regulations applicable to neffy or our current or future intranasal epinephrine technology product candidates; • any inability to obtain adequate supply of neffy or our current or future intranasal epinephrine technology product candidates or any of its components, or the inability to do so at acceptable prices; • adverse regulatory authority decisions; • introduction of new products, services or technologies by our competitors; • the perception of the pharmaceutical industry by the public, legislatures, regulators and the investment community; • announcements of significant acquisitions, strategic collaborations, joint ventures or capital commitments by us or our competitors; • disputes or other developments relating to proprietary rights, including patents, litigation matters, and our ability to obtain patent protection for neffy or our current or future intranasal epinephrine technology product candidates; • additions or departures of key personnel; • significant lawsuits, including patent or stockholder litigation; • if securities or industry analysts do not publish research or reports about our business, or if they issue an adverse or misleading opinion regarding our business and stock; • changes in the market valuations of similar companies; • general market or macroeconomic conditions, including as a result of actual or threatened tariffs and trade wars; • sales of our common stock by us or our stockholders in the future; • trading volume of our common stock; • perceived and actual risks associated with our Credit Agreement; • announcements by commercial partners or competitors of new commercial products, clinical progress or the lack thereof, significant contracts, commercial relationships or capital commitments; • adverse publicity generally, including with respect to other products and potential products in such markets; • the introduction of technological innovations or new therapies that compete with potential products of ours; • changes in the structure of health care payment systems; and • period-to-period fluctuations in our financial results.
Any unremediated critical or high risk vulnerabilities could pose material risks to our business. Further, we may experience delays in developing and deploying remedial measures designed to address any such identified vulnerabilities.
Any unremediated critical or high risk vulnerabilities could pose material risks to our business. Further, we have and may in the future experience delays in developing and deploying remedial measures designed to address any such identified vulnerabilities.
Unless and until we can secure alternative sources for microvials, drug product manufacturing and final packaging, our dependence on Renaissance and Ompi will subject us to the possible risks of shortages, interruptions, and price fluctuations.
Unless and until we can secure alternative sources, drug product manufacturing and final packaging, our dependence on Renaissance will subject us to the possible risks of shortages, interruptions, and price fluctuations.
In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of patents, once obtained. Depending on future actions by the U.S.
In addition to increasing uncertainty with regard to our ability to obtain patents in the future, this combination of events has created uncertainty with respect to the value of patents, once obtained. 91 Table of Contents Depending on future actions by the U.S.
If the equity and credit markets deteriorate, it may make any necessary debt or equity financing more difficult, more costly or more dilutive. We believe that our existing cash and cash equivalents will be sufficient to fund our planned operations for at least three years.
If the equity and credit markets do not improve or further deteriorate, it may make any necessary debt or equity financing more difficult, more costly or more dilutive. We believe that our existing cash and cash equivalents will be sufficient to fund our planned operations for at least three years.
We have not received U.S. non-patent marketing exclusivity for neffy , which was approved by the FDA under the 505(b)(2) regulatory pathway. Without non-patent marketing exclusivity for neffy , we may face competition by third parties seeking to market generic versions of neffy as early as our approval by the FDA.
We have not received U.S. non-patent marketing exclusivity for neffy , which was approved by the FDA under the 505(b)(2) regulatory pathway. Without non-patent marketing exclusivity for neffy , we may face competition by third parties seeking to market generic versions of neffy .
The Regulation foresees a three-year transitional period and permits EU Member States to use common HTA tools, methodologies, and procedures across the EU, working together in four main areas, including joint clinical assessment of the innovative health technologies with the most potential impact for patients, joint scientific consultations whereby developers can seek advice from HTA authorities, identification of emerging health technologies to identify promising technologies early, and continuing voluntary cooperation in other areas.
The Regulation permits EU Member States to use common HTA tools, methodologies, and procedures across the EU, working together in four main areas, including joint clinical assessment of the innovative health technologies with the most potential impact for patients, joint scientific consultations whereby developers can seek advice from HTA authorities, identification of emerging health technologies to identify promising technologies early, and continuing voluntary cooperation in other areas.
If we or a collaboration partner are unable to develop, receive regulatory approval for our intranasal epinephrine technology for the additional indications we are developing it for, including urticaria, or if we experience delays as a result of any of these risks or otherwise, our ability to grow our business will be limited. 57 If the FDA does not conclude that our intranasal epinephrine technology product candidates for future indications satisfies the requirements for the Section 505(b)(2) regulatory approval pathway, or if the requirements for any such future indications under Section 505(b)(2) are not as we expect, the approval pathway for additional indications will likely take significantly longer, cost significantly more and entail significantly greater complications and risks than anticipated, and in either case may not be successful.
If we or a collaboration partner are unable to develop, receive regulatory approval for our intranasal epinephrine technology for the additional indications we are developing it for, including urticaria, or if we experience delays as a result of any of these risks or otherwise, our ability to grow our business will be limited. 56 Table of Contents If the FDA does not conclude that our current or future intranasal epinephrine technology product candidates, or neffy for additional indications, satisfy the requirements for the Section 505(b)(2) regulatory approval pathway, or if the requirements for such product candidates or additional indications under Section 505(b)(2) are not as we expect, the approval pathway for those product candidates or additional indications will likely take significantly longer, cost significantly more and entail significantly greater complications and risks than anticipated, and in either case may not be successful.
We anticipate that our expenses will increase substantially if and as we: • maintain and expand our sales, marketing, distribution, manufacturing, supply chain and other commercial infrastructure to support the commercialization of neffy and any other indications for which we may obtain regulatory approval; • continue to develop and conduct nonclinical studies and clinical trials for neffy on a post-approval basis and our current or future intranasal epinephrine technology product candidates; • seek regulatory approvals in the United States and in the EU for our current or future intranasal epinephrine technology product candidates, and in other geographic regions for our current or future intranasal epinephrine technology product candidates; • seek to identify future product candidates; • experience any delays or encounter any issues with any of the above, including but not limited to failed studies, negative or mixed clinical trial results, safety issues or other regulatory challenges, the risk of which in each case may be exacerbated by a health epidemic or pandemic; • add clinical, scientific, operational, sales, financial and management information systems and personnel, including personnel to support our product candidate development and commercialization efforts and help us comply with our obligations as a public company; • maintain, expand and protect our intellectual property portfolio; and • acquire or in-license other product candidates and technologies.
We anticipate that our expenses will increase substantially if and as we: • maintain and expand our sales, marketing, distribution, manufacturing, supply chain and other commercial infrastructure to support the commercialization of neffy and any other product candidates for which we may obtain regulatory approval; • continue to develop and conduct nonclinical studies and clinical trials for our current or future intranasal epinephrine technology product candidates and for neffy on a post-approval basis; • seek regulatory approvals in the United States and in other geographic regions for our current or future intranasal epinephrine technology product candidates; • seek to identify future product candidates; • experience any delays or encounter any issues with any of the above, including but not limited to failed studies, negative or mixed clinical trial results, safety issues or other regulatory challenges, the risk of which in each case may be exacerbated by tariffs, trade wars, geopolitical conflicts and a health epidemic or pandemic; • add clinical, scientific, operational, sales, financial and management information systems and personnel, including personnel to support our product candidate development and commercialization efforts and help us comply with our obligations as a public company; • incur and pay the interest expense under the Credit Agreement; • maintain, expand and protect our intellectual property portfolio; and • acquire or in-license other product candidates and technologies.
For example, the California Consumer Privacy Act of 2018 (the “CCPA”), applies to personal information of consumers, business representatives, and employees, and requires businesses to provide specific disclosures in privacy notices and honor requests of California residents to exercise certain privacy rights.
For example, the California Consumer Privacy Act of 2018 (“CCPA”) applies to personal information of consumers, business representatives, and employees who are California residents, and requires businesses to provide specific disclosures in privacy notices and honor requests of such individuals to exercise certain privacy rights.
Our ability to commercially supply neffy and our current or future intranasal epinephrine technology product candidates depends, in part, on the ability of third-party manufacturers to supply, manufacture and warehouse the raw materials, active pharmaceutical ingredient (“API”) and other important components related to the manufacture of our intranasal epinephrine technology product candidates, including Intravail and our nasal sprayer apparatus.
Our ability to commercially supply neffy and our current or future intranasal epinephrine technology product candidates depends, in part, on the ability of third-party manufacturers to supply, manufacture and warehouse the raw materials, API and other important components related to the manufacture of our intranasal epinephrine technology product candidates, including Intravail and our nasal sprayer apparatus.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeRisk management and strategy We have implemented and maintain various information security processes designed to identify, assess and manage material risks from cybersecurity threats to our critical computer networks, third-party hosted services, communications systems, hardware and software, and our critical data, including intellectual property, and confidential information that is proprietary, strategic or competitive in nature, including clinical trial data (“Information Systems and Data”).
Biggest changeRisk management and strategy We have implemented and maintain various information security processes designed to identify, assess and manage material risks from cybersecurity threats to our critical computer networks, third-party hosted services, communications systems, hardware and software, and our critical data, including intellectual property, and confidential information that is proprietary, strategic or competitive in nature, including clinical trial data (“Information Systems and Data”). 106 Table of Contents Our Chief Executive Officer supervises our IT department (the “IT Department”), which coordinates with our cybersecurity incident management team , which consists of, among others, our Chief Financial Officer, Chief Legal Officer, Head of IT, and a third-party IT and cybersecurity consultant (“CSI Management Team”) to identify, assess and manage our cybersecurity threats and risks.
The board of directors’ audit committee is responsible for overseeing our cybersecurity risk management processes, including oversight of risks from cybersecurity threats. 103 Our cybersecurity risk assessment and management processes are implemented and maintained by certain members of our management, including the CSI Management Team.
The board of directors’ audit committee is responsible for overseeing our cybersecurity risk management processes, including oversight of risks from cybersecurity threats. Our cybersecurity risk assessment and management processes are implemented and maintained by certain members of our management, including the CSI Management Team.
Our Chief Executive Officer, Chief Financial Officer and Chief Legal Officer are responsible for hiring appropriate personnel, helping to integrate cybersecurity risk considerations into our overall risk management strategy, and communicating key priorities to relevant personnel. Additionally, they are responsible for approving budgets, helping prepare for cybersecurity incidents, approving cybersecurity processes, and reviewing security assessments and other security-related reports.
Our Chief Executive Officer, Chief Financial Officer and Chief Legal Officer are responsible for hiring appropriate personnel, helping to integrate cybersecurity risk considerations into our overall risk management strategy, and communicating key priorities to relevant personnel.
Our cybersecurity incident response policy is designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including our Chief Executive Officer, Chief Financial Officer, and Chief Legal Officer. These members of management work with our CSI Management Team to help us mitigate and remediate cybersecurity incidents of which they are notified.
These members of management work with our CSI Management Team to help us mitigate and remediate cybersecurity incidents of which they are notified. In addition, our cybersecurity incident response policy includes reporting to the audit committee of our board of directors for certain cybersecurity incidents.
Removed
Our Chief Executive Officer supervises our IT department (the “IT Department”), which coordinates with our cybersecurity incident management team , which consists of, among others, our Chief Financial Officer, Chief Legal Officer, Head of IT, and a third-party IT and cybersecurity consultant (“CSI Management Team”) to identify, assess and manage our cybersecurity threats and risks.
Added
Additionally, they are responsible for approving budgets, helping prepare for cybersecurity incidents, approving cybersecurity processes, and reviewing security assessments and other security-related reports. 107 Table of Contents Our cybersecurity incident response policy is designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including our Chief Executive Officer, Chief Financial Officer, and Chief Legal Officer.
Removed
In addition, our cybersecurity incident response policy includes reporting to the audit committee of our board of directors for certain cybersecurity incidents.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeItem 3. Legal Proceedings. From time to time, we may be involved in various claims and legal proceedings relating to claims arising out of our operations. See Note 10 - Commitments and Contingencies of this Annual Report, which is incorporated by reference in this Item 3, for any required disclosure. Item 4. Mine Safety Disclosures. Not applicable. 104 PART II
Biggest changeItem 3. Legal Proceedings. From time to time, we may be involved in various claims and legal proceedings relating to claims arising out of our operations. See Note 10 - Commitments and Contingencies of this Annual Report, which is incorporated by reference in this Item 3, for any required disclosure. Item 4. Mine Safety Disclosures.
Added
Not applicable. 108 Table of Contents PART II

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeHolders of Common Stock As of March 17, 2025, there were 16 holders of record of our common stock. Because most of our common stock is held by brokers, nominees, and other institutions on behalf of stockholders, we are unable to estimate the total number of stockholders represented by these record holders.
Biggest changeHolders of Common Stock As of March 4, 2026, there were 13 holders of record of our common stock. Because most of our common stock is held by brokers, nominees, and other institutions on behalf of stockholders, we are unable to estimate the total number of stockholders represented by these record holders.
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Use of Proceeds On December 3, 2020, Silverback Therapeutics, Inc. (“Silverback”) commenced its initial public offering (“IPO”) pursuant to a registration statement on Form S-1 (File No. 333-250009) that was declared effective by the SEC on December 3, 2020, for 11,500,000 shares of its common stock for sale to the public at a price of $21.00 per share.
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In addition, in December 2020, the underwriters exercised their over-allotment option to purchase 1,725,000 additional shares of Silverback common stock in the initial public offering at the public offering price of $21.00 per share, such that the aggregate offering price of the IPO was $277.7 million.
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The net offering proceeds to Silverback, after deducting underwriting discounts and commissions and offering costs, were $255.3 million. No offering expenses were paid directly or indirectly to any of the Silverback directors or officers (or their associates) or persons owning 10% or more of any class of Silverback’s equity securities or to any other affiliates.
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The underwriters for the Silverback initial public offering were Goldman Sachs & Co. LLC, SVB Leerink LLC, Stifel, Nicolaus & Company, Incorporated, and H.C. Wainwright & Co., LLC. On November 8, 2022, Silverback completed its reverse merger with privately-held ARS Pharmaceuticals, Inc. On November 9, 2022, the combined company changed its name to ARS Pharmaceuticals, Inc.
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The net proceeds from the IPO are held in cash and cash equivalents, primarily in treasury money market accounts, and investments, primarily in U.S. Treasury securities.
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Through December 31, 2024, approximately $243.0 million of the net proceeds from the IPO have been used, of which, (i) an estimated $51.7 million was used toward development of Silverback’s product candidates, (ii) $0.8 million was used to repay outstanding indebtedness, (iii) $16.0 million was used for transaction costs related to the Merger, including $7.0 million in severance and change in control benefit payments made to Silverback’s former officers, (iv) an estimated $65.4 million was used for development and pre-commercial launch activities related to neffy , (v) an estimated $78.1 million was used for working capital and general corporate purposes, and (vi) an estimated $31.0 million was used for commercial-related activities. 105 There have been no updates to the planned use of proceeds information from the IPO as described in our final prospectus filed with the SEC pursuant to Rule 424(b)(4) on December 4, 2020, except as otherwise disclosed in our Annual Report on Form 10-K, filed with the SEC on March 31, 2022, and our Quarterly Report on Form 10-Q, filed with the SEC on August 11, 2022.
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We intend to use the remaining net proceeds from the IPO, together with our existing cash and cash equivalents, to fund the manufacture and commercialization of neffy for the emergency treatment of Type I allergic reactions and other indications, if approved, as well as for working capital and other general corporate purposes.
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We may also use a portion of the net proceeds from the IPO to license, acquire or invest in complementary businesses, technologies, products or assets. However, we have no current commitments or obligations to do so. Item 6. [Reserved]

Item 7. Management's Discussion & Analysis

Management's Discussion & Analysis (MD&A) — revenue / margin commentary

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Biggest changeCommercial Launch Initiated in September 2024 Our launch strategy for neffy in the United States involves an initial direct sales force outreach to high-volume prescribers of epinephrine accounting for 40% to 45% of prescriptions in the last year through an efficient sales force comprised of 118 individuals serving as sales reps, virtual reps and areas sales managers that began field operations in early October 2024; active participation since November 2024 of approximately 2,500 healthcare professionals in our neffy experience program that allows healthcare professionals to use neffy firsthand as rescue therapy for anaphylaxis during in-clinic allergen challenge; extensive non-personal promotion including continuing medical education programs in collaboration with allergist societies, speaker bureaus, peer-to-peer programs and participation in regional and national medical conferences; engagement and contracting with payers to obtain timely coverage with favorable gross-to-net discounting; our neffyconnect program that provides support to physicians and patients including our $25 co-pay savings card, $199 cash price and patient assistance programs; a telemedicine service to conveniently obtain a prescription online; partnerships with patient advocacy organizations including disease awareness campaigns in 2025; and branded direct to consumer advertising including a celebrity that is expected to commence in the second quarter of 2025.
Biggest changeFor more information regarding our partners and collaboration agreements, see “ Business—Our Collaboration and Licensing Agreements. ” Our launch strategy is also supported by: active participation since November 2024 of approximately 2,800 healthcare professionals in our neffy experience program that allows healthcare professionals to use neffy firsthand as rescue therapy for anaphylaxis during in-clinic allergen challenge as well as for the ongoing collection of real-world evidence that supports neffy’s clinical equivalence to injection; extensive non-personal promotion including medical education programs in collaboration with allergist societies, speaker bureaus, peer-to-peer programs and participation in regional and national medical conferences; engagement and contracting with payors to obtain timely coverage with favorable gross-to-net discounting; our neffyconnect program that provides support to physicians and patients including our $25 co-pay savings card, $199 cash price and patient assistance programs; our neffy inSchools programs, where more than 9,000 schools to date have opted into receiving two cartons of neffy at no cost with accompanying school nurse education about neffy ; partnerships with patient advocacy organizations including disease awareness campaigns; and multi-channel branded direct to consumer advertising including connected television, point of care, endemic and programmatic display, social media, and paid search that initiated in May 2025, as well as linear television advertising that started in June 2025.
Until we consistently generate positive net income, if ever, our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials, our expenditures on other development activities, the cost for regulatory filings, expenses for commercial activities to establish, maintain and enhance sales, marketing and distribution capabilities for neffy , the timing and volume of our product sales, and our ability to earn potential regulatory and commercial milestones under our license and collaboration arrangements.
Until we consistently generate positive net income, if ever, our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials, our expenditures on other development activities, the cost for regulatory filings, expenses for commercial activities to establish, maintain and enhance sales, marketing and distribution capabilities for neffy , the timing and volume of our product sales, and our ability to earn potential royalties and regulatory and commercial milestones under our license and collaboration arrangements.
The revenues under collaboration agreements consists of $73.1 million under the ALK agreement for the delivery of a license to develop, manufacture and commercialize products containing epinephrine administered intranasally in the ALK territory excluding the EEA, $0.4 million under the ALK agreement for revenue recognized under the regulatory services performance obligations, $6.0 million from a regulatory milestone under the Alfresa agreement, $1.5 million for the first event milestone under the Seqirus Agreement, and $0.5 million for the delivery of the license for neffy in the Seqirus Territory in combination with the transfer of know-how under the Seqirus Agreement.
Revenue under collaboration agreements consists of $73.1 million under the ALK Collaboration Agreement for the delivery of a license to develop, manufacture and commercialize products containing epinephrine administered intranasally in the ALK Territory excluding the EEA, $0.4 million under the ALK Collaboration Agreement for revenue recognized under the regulatory services performance obligations, $6.0 million from a regulatory milestone under the Alfresa Agreement, $1.5 million for the first event milestone under the Seqirus Agreement, and $0.5 million for the delivery of the license for neffy in the Seqirus Territory in combination with the transfer of know-how under the Seqirus Agreement.
The increase in our operating liabilities was due to an increase in accounts payable and accrued liabilities of $16.4 million and an increase in contract liability of $2.1 million. The non-cash charges consisted primarily of non-cash stock-based compensation of $14.5 million, partially offset by $7.3 million in net amortization of discounts on short-term investments.
The increase in our operating liabilities was due to an increase in accounts payable and accrued liabilities of $16.4 million and an increase in contract liability of $2.1 million. The non-cash charges consisted primarily of non-cash stock-based compensation of $14.5 million, partially offset by net accretion of discounts on short-term investments of $7.3 million.
For a complete discussion of forward-looking statements, see the section above entitled “Forward Looking Statements.” Our actual results could differ materially from those expressed or implied in any forward-looking statements as a result of various factors, including those set forth under the caption “Item 1A.
For a complete discussion of forward-looking statements, see the section above entitled “Forward Looking Statements.” Our actual results could differ materially from those expressed or implied in any forward-looking statements as a result of various factors, including those set forth under the caption “ Item 1A.
Research and development expenses include: • salaries, payroll taxes, benefits and stock-based compensation charges for personnel engaged in research and development efforts; • external research and development expenses incurred under agreements with contract research organizations, or CROs, investigative sites and consultants and other third-party organizations to conduct our clinical studies and development activities; • costs related to manufacturing neffy and our intranasal epinephrine technology product candidates for clinical trials and process validation studies, including fees paid to third-party manufacturers; • costs related to compliance with regulatory requirements and regulatory filings; and • indirect expenses including insurance and facility-related expenses.
Research and development expenses include: • external research and development expenses incurred under agreements with contract research organizations (“CROs”), investigative sites and consultants and other third-party organizations to conduct our clinical studies and development activities; • costs related to manufacturing neffy and our intranasal epinephrine technology product candidates for clinical trials and process validation studies, including fees paid to contract manufacturing organizations (“CMOs”) and other third-party manufacturers; • costs related to compliance with regulatory requirements and regulatory filings; • indirect expenses including insurance and facility-related expenses; and • salaries, payroll taxes, benefits and stock-based compensation charges for personnel engaged in research and development efforts.
The terms of these agreements may include payment to us of one or more of the following: non-refundable, up-front license fees; clinical, regulatory, and/or commercial milestone payments; clinical development fees; and royalties or a transfer price on net sales of licensed products if neffy receives marketing approval in these regions.
The terms of these agreements may include payment to us of one or more of the following: non-refundable, upfront license fees; clinical, regulatory, and/or commercial milestone payments; clinical development fees; and royalties or a transfer price on net sales of licensed products if neffy receives marketing approval in these regions.
At the current list price for a two-pack of neffy and our target total gross-to-net yield, the estimated 6.5 million patients currently prescribed an epinephrine autoinjector in the United States represents an initial addressable market opportunity of approximately $3 billion in annual net sales, while the remaining 13.5 million diagnosed patients that have not been prescribed an epinephrine product represent an additional addressable market opportunity of approximately $7 billion in annual net sales.
At the current list price for neffy and our target total gross-to-net yield, the estimated 6.5 million patients currently prescribed an epinephrine autoinjector in the United States represents an initial addressable market opportunity of approximately $3.5 billion in annual net sales, while the remaining 13.5 million diagnosed patients that have not been prescribed an epinephrine product represent an additional addressable market opportunity of approximately $7.0 billion in annual net sales.
We believe the market opportunity for neffy in the United States alone is significant.
We believe the market opportunity for neffy in the United States is significant.
Selling, General and Administrative Expenses Selling, general and administrative expenses consist primarily of salaries, benefits, equity-based compensation for personnel in executive, finance, business development, sales and marketing and other corporate administrative functions.
Selling, General and Administrative Expenses Selling, general and administrative expenses consist primarily of salaries, benefits, stock-based compensation for personnel in executive, finance, business development, sales and marketing and other corporate administrative functions.
Our remaining payment obligations to OrbiMed under the Aegis Agreement are contingent upon our achievement of certain commercial milestones and have been reduced to $11.0 million as of December 31, 2024. Under the Aegis Agreement, we are also required to make royalty payments to OrbiMed based on a mid-single-digit percentage of net product sales.
Under the Aegis Agreement, remaining payment obligations to OrbiMed are contingent upon our achievement of certain commercial milestones and have been reduced to $9.0 million as of December 31, 2025. We are also required to make royalty payments to OrbiMed based on a mid-single-digit percentage of net product sales.
Our future funding requirements will depend on many factors, including: • the scope, progress, results and costs of researching and developing our intranasal epinephrine technology for additional indications; • the scope and costs of clinical and commercial manufacturing of neffy and our intranasal epinephrine technology product candidates; • the timing of, and the costs involved in, obtaining marketing approvals for our intranasal epinephrine technology for additional indications; • the number of additional indications for our intranasal epinephrine technology that we may pursue and their development requirements; • the costs of commercialization activities for neffy and our intranasal epinephrine technology product candidates, to the extent such costs are not the responsibility of any collaborators, including the costs and timing of building and maintaining product sales, marketing, distribution and manufacturing capabilities; • revenue received from commercial sales of neffy ; • the timing and amount of any milestone and royalty payments under the ALK Agreement, Pediatrix Agreement, Aegis Agreement, Alfresa Agreement, Recordati Termination Agreement, and the Seqirus Agreement; • the extent to which we in-license or acquire rights to other products, product candidates or technologies; • our headcount growth and associated costs as we expand our employee headcount and building and maintaining a commercial infrastructure; • the costs of preparing, filing and prosecuting patent applications, maintaining and protecting our intellectual property rights, including enforcing and defending intellectual property related claims; and • the costs of operating as a public company. 115 Until such time, if ever, as we can generate substantial product revenues to support our cost structure, we expect to finance our cash needs through a combination of our existing cash, cash equivalents, short-term investments, equity offerings, debt financings and other capital sources which may include collaborations, strategic alliances, marketing, distribution or licensing arrangements or other arrangements with third parties.
Our future funding requirements will depend on many factors, including: • revenue received from commercial sales of neffy ; • the timing and amount of any milestone and royalty payments under the ALK Collaboration Agreement, ALK Co-Promotion Agreement, Pediatrix Agreement, Aegis Agreement, Alfresa Agreement, Recordati Termination Agreement, and the Seqirus Agreement; • the scope, progress, results and costs of researching and developing our intranasal epinephrine technology for additional indications; • the scope and costs of clinical and commercial manufacturing of neffy and our intranasal epinephrine technology product candidates; • the timing of, and the costs involved in, obtaining marketing approvals for our intranasal epinephrine technology for additional indications; • the number of additional indications for our intranasal epinephrine technology that we may pursue and their development requirements; • the costs of commercialization activities for neffy and our intranasal epinephrine technology product candidates, to the extent such costs are not the responsibility of any collaborators, including the costs and timing of building and maintaining product sales, marketing, distribution and manufacturing capabilities; • the extent to which we in-license or acquire rights to other products, product candidates, or technologies; • our headcount growth and associated costs as we expand our employee headcount and building and maintaining a commercial infrastructure; • our ability to service our current credit facility under the Credit Agreement and access, if and when needed, additional amounts of principal provided for under the Credit Agreement; • the costs of preparing, filing, and prosecuting patent applications, maintaining and protecting our intellectual property rights, including enforcing and defending intellectual property related claims; and • the costs of operating as a public company. 118 Table of Contents Until such time, if ever, as we can generate substantial product revenues to support our cost structure, we expect to finance our cash needs through a combination of our existing cash, cash equivalents, short-term investments, equity offerings, debt financings and other capital sources which may include collaborations, strategic alliances, marketing, distribution or licensing arrangements or other arrangements with third parties.
In addition, we cannot forecast to what degree our licensing, supply and distribution arrangements would affect our development plans and capital requirements. 110 The duration, costs and timing of clinical trials and development of neffy and our intranasal epinephrine technology product candidates for the treatment of additional indications will depend on a variety of factors that include: • per patient trial costs; • the number of patients that participate in the trials; • the number of sites included in the trials; • the countries in which the trials are conducted; • the length of time required to enroll eligible patients; • the number of doses that patients receive; • the drop-out or discontinuation rates of patients; • potential additional safety monitoring or other studies requested by regulatory agencies; • the efficacy and safety profile of neffy and our current and future intranasal epinephrine technology product candidates; • the cost to seek regulatory approvals for our intranasal epinephrine technology product candidates in additional indications and any product candidates that successfully complete clinical trials; • the timing, receipt, and terms of any approvals from applicable regulatory authorities including the FDA and non-U.S. regulators; • maintaining a continued acceptable safety profile of neffy and our intranasal epinephrine technology product candidates; • establishing or maintaining commercial manufacturing capabilities or making arrangements with third-party manufacturers in order to ensure that we or our third-party manufacturers are able to make product successfully; • significant and changing government regulation and regulatory guidance; • the impact of any business interruptions to our operations or to those of the third parties with whom we work; and • the extent to which we establish additional strategic collaborations or other arrangements.
The duration, costs and timing of clinical trials and development of neffy and our intranasal epinephrine technology product candidates for the treatment of additional indications will depend on a variety of factors that include: • per patient trial costs; • the number of patients that participate in the trials; • the number of sites included in the trials; • the countries in which the trials are conducted; • tariffs and international trade relations; • the length of time required to enroll eligible patients; • the number of doses that patients receive; • the drop-out or discontinuation rates of patients; • potential additional safety monitoring or other studies requested by regulatory agencies; • the efficacy and safety profile of neffy and our current and future intranasal epinephrine technology product candidates; • the cost to seek regulatory approvals for our intranasal epinephrine technology product candidates in additional indications and any product candidates that successfully complete clinical trials; • the timing, receipt, and terms of any approvals from applicable regulatory authorities including the FDA and non-U.S. regulators; • maintaining a continued acceptable safety profile of neffy and our intranasal epinephrine technology product candidates; • establishing or maintaining commercial manufacturing capabilities or making arrangements with third-party manufacturers in order to ensure that we or our third-party manufacturers are able to make product successfully; • significant and changing government regulation and regulatory guidance; • the impact of any business interruptions to our operations or to those of the third parties with whom we work; and • the extent to which we establish additional strategic collaborations or other arrangements.
Item 7. Management’s Discussion and Analysis o f Financial Condition and Results of Operations. You should read the following discussion and analysis together with our financial statements and related notes included in “Item 8. Financial Statements and Supplementary Data” in this Annual Report. The following discussion contains forward-looking statements that involve risks and uncertainties.
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations. You should read the following discussion and analysis together with our financial statements and related notes included in “ Item 8. Financial Statements and Supplementary Data ” in this Annual Report. The following discussion contains forward-looking statements that involve risks and uncertainties.
Other Income, net Other income, net consists primarily of interest income from our cash, cash equivalents, and short-term investments, and net amortization and accretion associated with our short-term investments.
Other Income, net Other income, net consists primarily of interest income from our cash, cash equivalents, and short-term investments, interest expense on our outstanding debt, and net amortization and accretion associated with our short-term investments.
This consisted primarily of purchases of short-term investments of $356.0 million, maturities of short-term investments of $258.0 million, payments of milestone obligations under license agreements of $7.5 million, and purchases of property and equipment of $0.6 million. During the year ended December 31, 2023, the cash and cash equivalents used in investing activities was $87.2 million.
During the year ended December 31, 2024, the cash and cash equivalents used in investing activities was $106.1 million. This consisted primarily of purchases of short-term investments of $356.0 million, payments of milestone obligations under license agreements of $7.5 million, and purchases of property and equipment of $0.6 million, partially offset by maturities of short-term investments of $258.0 million.
We do not own or operate manufacturing facilities. We currently rely on third-party manufacturers and suppliers for neffy and our intranasal epinephrine technology product candidates, and we expect to continue to do so to meet our nonclinical, clinical and commercial activities. Our third-party manufacturers are required to manufacture our product under cGMP requirements and other applicable laws and regulations.
We do not own or operate manufacturing facilities. We currently rely on third-party manufacturers and suppliers for neffy and our intranasal epinephrine technology product candidates, and we expect to continue to do so to meet our nonclinical, clinical and commercial activities.
This consisted primarily of net income of $8.0 million, an increase in our operating liabilities of $18.5 million, an increase in our operating assets of $20.3 million, and non-cash charges of $7.4 million.
This consisted primarily of a net loss of $171.3 million, an increase in our operating assets and operating liabilities of $38.5 million and $18.5 million, respectively, and non-cash charges of $20.4 million.
Material Cash Requirements The total amount of unconditional purchase obligations related to the supply of raw materials is $64.9 million as of December 31, 2024. Payment obligations by year are as follows: 2025 ($8.2 million), 2026 ($10.5 million), 2027 ($11.8 million), 2028 ($13.8 million), and $2.9 million per year thereafter through 2035.
F uture Contractual Cash Obligations The total remaining unconditional purchase obligations related to the supply of raw materials is $55.3 million as of December 31, 2025. Our remaining payment obligations by year are as follows: 2026 ($9.1 million), 2027 ($11.8 million), 2028 ($13.8 million), and $2.9 million per year thereafter through 2035.
Prior to the FDA approval of neffy in August 2024, costs incurred for the manufacture of neffy were recorded as research and development expenses, which resulted in zero-cost inventory.
Prior to the FDA approval of neffy in August 2024, certain inventory components were purchased to manufacture neffy and recorded as research and development expenses, resulting in zero-cost inventory components.
We believe neffy ’s “no needle, no injection” approach addresses a significant unmet need in the use of epinephrine, which, except for neffy , is currently approved only in injectable formulations for the emergency treatment of Type I allergic reactions. There are approximately 40 million people in the United States who experience Type I allergic reactions.
We believe neffy ’s “no needle, no injection” approach addresses a significant unmet need in the use of epinephrine. There are approximately 40 million people in the U.S. who experience Type I allergic reactions.
As of December 31, 2024, no zero-cost inventory was determined to be obsolete. Research and Development Expenses To date, our research and development expenses have been related primarily to clinical development, process development and manufacturing costs of neffy and our intranasal epinephrine technology product candidates.
Research and Development Expenses To date, our research and development expenses have been related primarily to clinical development, process development, and manufacturing costs of neffy and our intranasal epinephrine technology product candidates.
Of this group, approximately 20 million people have been diagnosed and experienced severe Type I allergic reactions that may lead to anaphylaxis, and approximately 6.5 million were prescribed an epinephrine autoinjector.
Of this group, approximately 20 million people are reported to have been diagnosed and experienced severe Type I allergic reactions that may lead to anaphylaxis, and approximately 6.5 million of those were prescribed an epinephrine autoinjector. However, in recent years, only an estimated one-half of those consistently carry their prescribed autoinjector with them.
We expect revenues under collaboration agreements to fluctuate in future periods based on our ability to meet various regulatory milestones, and contingent on successfully obtaining regulatory approval for neffy in the licensed regions, commercial milestones, royalties or transfer price earned from our partner’s net sales and the supply of commercial product as set forth in the agreements described earlier. 109 Cost of Goods Sold Cost of goods sold consists primarily of direct and indirect costs related to the manufacture of neffy for commercial sale, including third-party manufacturing costs, raw material and component costs, packaging services, freight, storage costs, distribution fees, amortization of capitalized in-licensed costs, and royalties on product sales.
We expect revenues under collaboration agreements to fluctuate in future periods based on our ability to meet various regulatory milestones, and contingent on successfully obtaining regulatory approval for neffy in the licensed regions, commercial milestones, royalties or transfer price earned from our partner’s net sales and the supply of commercial product as set forth in the agreements described earlier.
This consisted of $69.4 million from the upfront payment from ALK that was allocated to the EEA License and $3.0 million from stock option exercises and the employee stock purchase plan.
During the year ended December 31, 2024, the cash and cash equivalents provided by financing activities was $72.4 million. This consisted of $69.4 million from the upfront payment from ALK that was allocated to the EEA License and $3.0 million from stock option exercises and issuance of common stock under the employee stock purchase plan.
We have funded our operations primarily with proceeds from the Merger (see Note 1 - Nature of Business to the notes to the consolidated financial statements included in this report), private placement of convertible preferred stock, licensing, supply and distribution arrangements with our commercialization partners, bank debt, and limited net product sales.
We have funded our operations to date primarily with proceeds from the merger with Silverback, private placement of convertible preferred stock, issuance of common stock, licensing, supply and distribution arrangements with our commercialization partners, debt, and net product sales.
As of December 31, 2024, we had cash, cash equivalents, and short-term investments of $314.0 million. We have incurred net losses from operations in most years since our inception. Our net income was $8.0 million for the year ended December 31, 2024 and our net loss was $54.4 million for the year ended December 31, 2023.
We have incurred net losses in most years since our inception. Net loss for the year ended December 31, 2025 was $171.3 million, and net income for the year ended December 31, 2024 was $8.0 million. As of December 31, 2025, we had an accumulated deficit of $294.6 million.
During the year ended December 31, 2023, net cash used in operating activities was $59.3 million. This consisted primarily of a net loss of $54.4 million, a decrease in our operating liabilities of $5.9 million, an increase in our operating assets of $1.4 million, and non-cash charges of $2.4 million.
During the year ended December 31, 2024, net cash provided by operating activities was $13.5 million. This consisted primarily of net income of $8.0 million, an increase in our operating assets of $20.3 million, an increase in our operating liabilities of $18.5 million, and non-cash charges of $7.4 million.
Our ability to raise additional funds may be adversely impacted by potential worsening global economic conditions and disruptions to and volatility in the credit and financial markets in the United States, including due to bank failures, and worldwide resulting from macroeconomic factors.
Our ability to raise additional funds may be adversely impacted by macroeconomic factors that may result in worsening global economic conditions and disruptions to and volatility in the global credit and financial markets, including due to tariffs, trade wars, inflation, high interest rates, recessionary concerns, recessions, bank failures, geopolitical conflicts, and general economic uncertainty.
As of December 31, 2024, we had no accrued interest or penalties related to uncertain tax positions. 116 Critical Accounting Policies and Significant Judgments and Estimates Our management’s discussion and analysis of our financial condition and results of operations are based on our financial statements, which have been prepared in accordance with U.S. generally accepted accounting principles (GAAP).
Critical Accounting Estimates Our management’s discussion and analysis of our financial condition and results of operations are based on our financial statements, which have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”).
The decrease in our operating liabilities was primarily due to a decrease in contract liability of $3.1 million and a decrease in accounts payable and accrued liabilities of $2.8 million. The increase in our operating assets was primarily due to an increase in prepaid and other assets of $1.4 million.
The increase in our operating liabilities was primarily attributable to an increase in accounts payable and accrued expenses of $18.9 million, partially offset by a decrease in contract liability of $0.4 million.
The increase of $24.4 million was primarily due to a $11.8 million increase in marketing-related expenses, a $10.6 million increase in payroll-related expenses, a $4.6 million increase in stock-based compensation, a $2.3 million increase in outside services, a $1.6 million increase in legal fees, a $1.4 million increase in meals and travel-related expenses predominantly incurred by the sales team, a $0.4 million increase in professional fees for accounting, auditing and tax, and a $0.3 million increase in conference and seminar expenses.
The increase of $158.4 million was primarily due to increases in marketing-related expenses of $111.4 million, personnel-related expenses of $24.2 million, stock-based compensation expense of $7.9 million, outside services of $4.2 million, travel and meals expense of $3.6 million incurred mainly by our sales personnel, conference and seminar expense of $2.5 million, audit, tax and valuation fees of $1.5 million, legal fees of $0.6 million, and other general operating costs of $2.5 million.
Revenues were $89.1 million and less than $0.1 million for the years ended December 31, 2024 and 2023. The revenues for the year ended December 31, 2024 includes $81.5 million in revenues under collaboration agreements, $7.3 million in net product revenues for sales of neffy, and $0.4 million in revenue under supply agreements .
Revenue for the year ended December 31, 2025 includes $72.2 million in net product revenues for sales of neffy in the United States, $9.7 million in revenue under collaboration agreements, and $2.4 million in revenue under supply agreements.
Cash flows The following table summarizes our cash flows for the years ended December 31, 2024 and 2023 (in thousands): Year Ended December 31, 2024 2023 Net cash and cash equivalents provided by (used in) operating activities $ 13,548 $ (59,266 ) Net cash and cash equivalents used in investing activities (106,101 ) (87,180 ) Net cash and cash equivalents provided by financing activities 72,399 6,899 Net decrease in cash and cash equivalents $ (20,154 ) $ (139,547 ) Operating Activities During the year ended December 31, 2024, net cash provided by operating activities was $13.5 million.
As of December 31, 2025, we had cash, cash equivalents, and short-term investments of $245.0 million. 116 Table of Contents Cash flows The following table summarizes our cash flows for the years ended December 31, 2025 and 2024 (in thousands): Years Ended December 31, 2025 2024 Net cash and cash equivalents (used in) provided by operating activities $ (170,866 ) $ 13,548 Net cash and cash equivalents provided by (used in) investing activities 56,768 (106,101 ) Net cash and cash equivalents provided by financing activities 104,598 72,399 Net decrease in cash and cash equivalents $ (9,500 ) $ (20,154 ) Operating Activities During the year ended December 31, 2025, net cash used in operating activities was $170.9 million.
For arrangements that include sales-based royalties or transfer price, including milestone payments based on the level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied).
Sales-based royalties or commercial milestones based on product sales where a license is the predominant item are recorded at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied).
Under the Recordati Termination Agreement, we are also required to make royalty payments to Recordati of up to €5.0 million (approximately $5.2 million in U.S. dollars) in the aggregate from sales of Recordati Licensed Product(s) in the Recordati Territory. Future royalty payment amounts are indeterminate since they depend on future revenues, which are uncertain.
Under the Recordati Termination Agreement, we are required to make royalty payments to Recordati of up to €5.0 million in the aggregate from sales of neffy in the Recordati Territory, of which up to €4.6 million (approximately $5.4 million in U.S. dollars) remain as of December 31, 2025.
Since prior to August 2024, costs incurred for the manufacture of neffy were recorded as research and development expenses, the cost of goods sold for the year ended December 31, 2024 utilized zero-cost inventory and therefore consisted primarily of distribution fees, royalties, and intangible assets amortization. Research and Development Expenses .
Cost of Goods Sold . Cost of goods sold for the year ended December 31, 2025 was $20.4 million, as compared to $1.0 million for the year ended December 31, 2024. Prior to August 2024, costs incurred to manufacture neffy were recorded as research and development expenses, and product sales subsequent to August 2024 partially utilized zero-cost inventory components.
Our clinical, regulatory, manufacturing, and non-clinical development costs for the periods presented below reflect an allocation of expenses associated with personnel costs, equity-based compensation expense, and indirect costs incurred in support of overall research and development, such as facilities-related costs.
Our clinical, regulatory, manufacturing, and non-clinical development costs for the periods presented below reflect an allocation of expenses associated with personnel costs, stock-based compensation expense, and indirect costs incurred in support of overall research and development, such as facilities-related costs. 113 Table of Contents We cannot determine with certainty the timing of initiation, the duration or the completion costs of current or future clinical trials and the manufacturing costs of neffy and our intranasal epinephrine technology product candidates due to the inherently unpredictable nature of clinical development and manufacturing activities.
Risk Factors.” Overview We are a biopharmaceutical company focused on the commercialization and development of neffy (previously referred to as ARS-1 and, in the case of the 2 mg form, currently identified in the EU by the tradename EURneffy ) for the needle-free intranasal delivery of epinephrine for the emergency treatment of Type I allergic reactions, including anaphylaxis. neffy is the first and only FDA and EC-approved needle-free epinephrine product, and the first new delivery method for epinephrine in more than 35 years. neffy is a proprietary composition of epinephrine with an innovative absorption enhancer called Intravail, which allows neffy to safely provide intranasal delivery of epinephrine at a low dose within the exposures of approved injectable products across a range of dosing conditions (including repeat dosing and allergen challenge).
It is the first new delivery method for epinephrine in more than 35 years. neffy is a proprietary composition of epinephrine with an innovative absorption enhancer called Intravail, which allows neffy to safely provide intranasal delivery of epinephrine at a low dose within the exposures of approved injectable products across a range of dosing conditions (including repeat dosing and allergen challenge).
Future royalty payment amounts are indeterminate since they depend on future revenues, which are uncertain. On February 22, 2023, we entered into a termination agreement (the “Recordati Termination Agreement”) with Recordati Ireland, Ltd.
Future royalty payment amounts are indeterminate since they depend on future revenues, which are uncertain. In February 2023, we entered into a termination agreement (the “Recordati Termination Agreement”) with Recordati Ireland, Ltd. (“Recordati”) to reacquire the rights to neffy in Europe and certain European Free Trade Association, Russia/the Commonwealth of Independent States, Middle East and African countries (the “Recordati Territory”).
As a result, the cost of goods sold related to neffy will initially reflect a lower average per unit cost of materials, as previously expensed zero-cost inventory is utilized for commercial production and sold to customers. We expect the cost of goods sold for neffy to increase in relation to product revenues as we deplete these inventories.
As a result, the cost of goods sold related to neffy will initially reflect a lower average per unit cost of materials, as previously expensed inventory components are consumed in commercial production and sold to customers. As of December 31, 2025, we had $6.7 million in zero-cost inventory components remaining, and no zero-cost inventory components were determined to be obsolete.
Selling, general and administrative expenses also include pre-commercial launch activities prior to product launch, the initiation of commercialization activities in September 2024, legal fees incurred relating to corporate and patent matters, professional fees incurred for accounting, auditing, tax and administrative consulting services, market research costs, and insurance costs. 111 We expect our selling, general and administrative expenses to increase substantially in 2025.
Selling, general and administrative expenses also include pre-commercial launch activities prior to product launch, the initiation of commercialization activities in September 2024, legal fees incurred relating to corporate and patent matters, professional fees incurred for accounting, auditing, tax and administrative consulting services, and insurance costs. 114 Table of Contents Selling, general and administrative expenses have increased since the third quarter of 2024 due to the establishment of our sales force, the development and commencement of our marketing campaigns and initiatives, the ALK Co-Promotion Agreement, the hiring of additional sales and marketing personnel to support full commercialization activities, and the addition of infrastructure and programs to support commercialization activities.
Selling, general and administrative expenses were $71.7 million and $47.3 million for the years ended December 31, 2024 and 2023, respectively.
Selling, general and administrative expenses for the year ended December 31, 2025 was $230.1 million, as compared to $71.7 million for the year ended December 31, 2024.
This consisted primarily of purchases of short-term investments of $272.0 million, maturities of short-term investments of $185.0 million, and purchases of property and equipment of $0.2 million. 114 Financing Activities During the year ended December 31, 2024, the cash and cash equivalents provided by financing activities was $72.4 million.
Investing Activities During the year ended December 31, 2025, the cash and cash equivalents provided by investing activities was $56.8 million. This consisted of maturities of short-term investments of $307.0 million, partially offset by purchases of short-term investments of $242.0 million, payments of milestone obligations under license agreements of $7.9 million, and purchases of property and equipment of $0.3 million.
During the year ended December 31, 2023, the cash and cash equivalents provided by financing activities was $6.9 million, which consisted of proceeds from stock option exercises and the employee stock purchase plan.
Financing Activities During the year ended December 31, 2025, the $104.6 million of cash and cash equivalents provided by financing activities was attributable to net proceeds from the term loan under the Credit Agreement of $96.3 million, proceeds from stock option exercises and issuance of common stock under the employee stock purchase plan of $5.7 million, and proceeds from milestone obligations met under license agreements of $2.7 million.
Results of Operations Comparison of the Years Ended December 31, 2024 and 2023: The following table summarizes our results of operations for years ended December 31, 2024 and 2023 (in thousands, except percentages): Year Ended December 31, Dollar % 2024 2023 Change Change Revenue: Product revenue, net $ 7,255 $ — $ 7,255 * % Revenue under collaboration agreements 81,529 30 81,499 * Revenue under supply agreements 365 — 365 * Total revenue 89,149 30 89,119 * Operating expenses: Cost of goods sold 977 — 977 * Research and development (1) 19,580 20,266 (686 ) (3 ) Selling, general and administrative (1) 71,675 47,284 24,391 52 Total operating expenses 92,232 67,550 24,682 37 Loss from operations (3,083 ) (67,520 ) 64,437 (95 ) Other income, net 11,369 13,155 (1,786 ) (14 ) Income (loss) before income taxes 8,286 (54,365 ) 62,651 (115 ) Income tax provision 288 — 288 * Net income (loss) $ 7,998 $ (54,365 ) $ 62,363 (115 ) Change in unrealized gains and losses on available-for-sale securities 171 (358 ) 529 (148 ) Comprehensive income (loss) $ 8,169 $ (54,723 ) $ 62,892 (115 ) % ______________ * Not meaningful (1) Includes stock-based compensation expense as follows (in thousands): Year Ended December 31, 2024 2023 Research and development $ 2,955 $ 2,274 Selling, general and administrative 11,579 6,961 Total $ 14,534 $ 9,235 112 Revenues .
Results of Operations Comparison of the Years Ended December 31, 2025 and 2024 The following table summarizes our results of operations for the years ended December 31, 2025 and 2024 (in thousands, except percentages): Years Ended December 31, Dollar % 2025 2024 Change Change Revenue: Product revenue, net $ 72,192 $ 7,255 $ 64,937 * Revenue under collaboration agreements 9,716 81,529 (71,813 ) (88 %) Revenue under supply agreements 2,370 365 2,005 * Total revenue 84,278 89,149 (4,871 ) (5 %) Operating expenses: Cost of goods sold 20,423 977 19,446 * Research and development (1) 13,181 19,580 (6,399 ) (33 %) Selling, general and administrative (1) 230,122 71,675 158,447 221 % Total operating expenses 263,726 92,232 171,494 186 % Loss from operations (179,448 ) (3,083 ) (176,365 ) * Other income (expense), net: Interest income 10,669 11,369 (700 ) (6 %) Interest expense (2,599 ) — (2,599 ) * Total other income, net 8,070 11,369 (3,299 ) (29 %) (Loss) income before income tax (benefit) expense (171,378 ) 8,286 (179,664 ) * Income tax (benefit) expense (80 ) 288 (368 ) (128 %) Net (loss) income $ (171,298 ) $ 7,998 $ (179,296 ) * _____________ * Not meaningful (1) Includes stock-based compensation expense as follows (in thousands): Years Ended December 31, 2025 2024 Research and development $ 2,660 $ 2,955 Selling, general and administrative 19,435 11,579 Total $ 22,095 $ 14,534 Revenues .
On August 22, 2024, the EC granted marketing authorization in the EU for EURneffy (the trade name for neffy 2 mg in the EU), for the emergency treatment of allergic reactions (anaphylaxis), in adults and children who weigh 30 kg or greater.
The EC has granted marketing authorization in the EU for EURneffy 2 mg (the trade name for neffy 2 mg in the EU and U.K.), for the emergency treatment of Type I allergic reactions, including anaphylaxis, in adults and children who weigh 30 kg or greater and on January 29, 2026, the Committee for Medicinal Products for Human Use of the EMA adopted a positive opinion, recommending marketing authorization in the EU for EURneffy 1 mg for children who are four years of age and older and weigh 15 kg to less than 30 kg.
These aggregated decreases were partially offset by a $0.9 million increase in net amortization and accretion associated with our short-term investments. 113 Liquidity and Capital Resources Sources of Liquidity and Capital Since our inception, we have incurred significant operating losses and negative cash flows from our operations.
Liquidity and Capital Resources Sources of Liquidity and Capital Since our inception, we have incurred significant operating losses and negative cash flows from our operations.
In August 2024, we entered into a corporate sponsorship agreement with Food Allergy Research and Education, Inc. pursuant to which we have payment obligations of $9.0 million over a 28-month period. Our remaining payment obligations as of December 31, 2024 are $7.0 million. Estimated payments by year are as follows: 2025 ($4.0 million), and 2026 ($3.0 million).
Future performance-based payment amounts are indeterminate since they depend on future revenues, which are uncertain. In August 2024, we entered into a corporate sponsorship agreement with Food Allergy Research and Education, Inc., which was subsequently amended in May 2025. Our remaining payment obligations as of December 31, 2025 are $6.0 million.
On March 5, 2025, the FDA approved neffy 1 mg for the emergency treatment of Type I allergic reactions, including anaphylaxis, in patients who are four years of age and older and weigh 15 kg to less than 30 kg. neffy U.S.
Those estimated 3.2 million patients who currently fill their active epinephrine autoinjector prescription would represent approximately $1.8 billion in annual U.S. net sales at neffy ’s target estimated gross-to-net yield based on epinephrine device unit volume in 2025. 110 Table of Contents In August 2024, the FDA approved neffy 2 mg for the emergency treatment of Type I allergic reactions, including anaphylaxis, in adults and children who weigh 30 kg or greater, with neffy 1 mg subsequently approved in March 2025 for patients who are four years of age and older and weigh 15 kg to less than 30 kg.
From inception to December 31, 2024, we have raised $262.3 million in cash, cash equivalents and short-term investments, net of transaction costs, from the Merger; net proceeds of $76.3 million from the issuance of convertible preferred and common stock; $181.0 million from our collaboration, licensing, supply and distribution arrangements; $10.0 million from bank debt, $7.3 million from net product sales and $0.4 million in revenue under supply agreements.
We have funded our operations primarily with proceeds from the merger with Silverback Therapeutics, Inc. (“Silverback”) in November 2022, private placement of convertible preferred stock, issuance of common stock, licensing, supply and distribution arrangements with our commercialization partners, debt, and net product sales. As of December 31, 2025, we had cash, cash equivalents, and short-term investments of $245.0 million.
We enter into contracts in the normal course of business with third-party contract organizations and vendors for clinical studies, manufacturing and other services and products. These contracts generally provide for termination after a notice period. As of December 31, 2024, we have not recognized any reserves related to uncertain tax positions.
These contracts generally provide for termination after a notice period. 119 Table of Contents As of December 31, 2025, we have not recognized any reserves related to uncertain tax positions and had no accrued interest or penalties related to uncertain tax positions.
Clinical development and manufacturing timelines, the probability of success and development costs can differ materially from expectations.
Clinical development and manufacturing timelines, the probability of success and development costs can differ materially from expectations. In addition, we cannot forecast to what degree our licensing, supply and distribution arrangements would affect our development plans and capital requirements.
The non-cash charges consisted of non-cash stock-based compensation of $9.2 million, partially offset by $6.9 million in net amortization of discounts on short-term investments. Investing Activities During the year ended December 31, 2024, the cash and cash equivalents used in investing activities was $106.1 million.
The non-cash charges consisted primarily of non-cash stock-based compensation of $22.1 million, establishment of an inventory reserve of $2.2 million, depreciation and amortization expense of $1.4 million, and other non-cash items of $0.3 million, partially offset by net accretion of discounts on short-term investments of $5.5 million.
The decrease of $0.7 million was primarily due to a $1.3 million decrease in product development expenses, a $1.2 million decrease in consulting fees, a $0.8 million decrease in clinical trial costs associated with neffy, a $0.4 million decrease in payroll-related expenses, and a $0.6 million decrease in other operating expenses.
The decrease of $6.4 million was primarily due to decreases in IPR&D expense of $2.1 million from the achievement of the EMA regulatory milestone under the Recordati Termination Agreement during the year ended December 31, 2024, product-development related expense of $1.8 million, clinical trial costs of $0.8 million, personnel-related expenses of $0.8 million, and other research and development expenses of $0.9 million.
The decrease of $1.8 million was primarily due to a $2.1 million decrease in interest income from our cash, cash equivalents, and short-term investments, a $0.3 million decrease from the sale of in-process research and development obtained in the Merger, which occurred in the year ended December 31, 2023, and a $0.3 million decrease in other items.
The decrease of $3.3 million was primarily due to interest expense related to the term loan of $2.6 million and a decrease in net accretion of discounts on short-term investments of $2.1 million, partially offset by an increase in interest income from our cash, cash equivalents, and short-term investments of $1.4 million.
These aggregated decreases were partially offset by a $2.1 million increase in expense related to an EMA regulatory milestone under the Recordati Termination Agreement, a $0.8 million increase in outside services, and a $0.7 million increase in stock-based compensation, The following table summarizes our research and development expenses for the years ended December 31, 2024 and 2023 (in thousands): Year Ended December 31, 2024 2023 Clinical and regulatory $ 8,033 $ 9,057 Manufacturing and non-clinical development 11,547 11,209 Total research and development expenses $ 19,580 $ 20,266 Selling, General and Administrative Expenses.
The following table summarizes our research and development expenses for the years ended December 31, 2025 and 2024 (in thousands): Years Ended December 31, 2025 2024 Clinical and regulatory $ 6,943 $ 8,033 Manufacturing and non-clinical development 6,238 11,547 Total $ 13,181 $ 19,580 Selling, General and Administrative Expenses.
Either the Company or ALK may terminate the Supply Agreement in the event of an uncured material breach of the other party. Financial Overview Revenues We have recognized limited net product sales in the United States since the commercial launch of neffy in September 2024.
Our third-party manufacturers are required to manufacture our product under cGMP requirements and other applicable laws and regulations. 112 Table of Contents Financial Overview Revenues We have recognized net product sales in the United States since the commercial launch of neffy in September 2024.
Regulatory decisions are anticipated by mid-2025 in the U.K., the second-half of 2025 in Japan, year-end 2025 in Canada, and in the first-half of 2026 in China and Australia. 107 We reported positive topline results demonstrating statistically significant and clinically meaningful improvements in treatment-refractory chronic urticaria patients at the American Academy of Allergy and Immunology medical conference in February 2024, and anticipate initiating a Phase 2b randomized, placebo controlled outpatient clinical trial in chronic spontaneous urticaria patients on a chronic antihistamine treatment regimen who still experience flares or exacerbations.
We reported positive topline results demonstrating statistically significant and clinically meaningful improvements in treatment-refractory chronic urticaria patients at the American Academy of Allergy and Immunology medical conference in February 2024.
The total amount of unconditional purchase obligations related to hosted software license subscription fees is $3.3 million as of December 31, 2024. Payment obligations by year are as follows: 2025 ($1.4 million), 2026 ($1.5 million), and 2027 ($0.4 million).
Our remaining payment obligations by year are as follows: 2026 ($5.0 million), and 2027 ($1.0 million). Under the Credit Agreement, the outstanding principal of $100.0 million as of December 31, 2025 is due upon maturity on September 29, 2030.
This Phase 2b study is anticipated to initiate in the second quarter of 2025, with topline data anticipated in early 2026, followed by the potential initiation of a single pivotal efficacy study in 2026.
In the second quarter of 2025, we initiated a Phase 2b randomized, placebo-controlled outpatient clinical trial involving chronic spontaneous urticaria patients, on chronic treatment regimens, who still experience flares or exacerbations. Interim data from this clinical trial is anticipated in the second half of 2026, followed by the potential initiation of a single pivotal efficacy study in mid-2027.
Product revenue is recorded with each sale at wholesale acquisition cost , net of reserves for variable components, including but not limited to distribution service fees, prompt pay discounts, product returns, chargebacks, rebates, and co-payment assistance, which are collectively referred to as “Gross-to-Net Adjustments.” In accordance with ASC 606, the Company must make significant judgments to determine the estimates for certain Gross-to-Net Adjustments.
Revenue Recognition Product revenue, net Product revenue is recorded net of estimates for variable consideration, including distribution service fees, prompt pay discounts, product returns, chargebacks, rebates, co-payment assistance, and other incentives for certain indirect customers.
If the license to the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, revenue is recognized from non-refundable, up-front payments allocated to the license when the license is transferred to the customer and the customer is able to use and benefit from the license.
Revenue under collaboration agreements Revenue from licenses to our intellectual property is recorded when the license is transferred and the licensee can use and benefit from it, provided the license is distinct from other obligations.
Research and development expenses were $19.6 million and $20.3 million for the years ended December 31, 2024 and 2023, respectively.
Cost of goods sold consisted primarily of product costs and royalties, and during the year ended December 31, 2025, the establishment of an inventory reserve. Research and Development Expenses . Research and development expenses for the year ended December 31, 2025 were $13.2 million, as compared to $19.6 million for the year ended December 31, 2024.
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However (in 2023, for example), only 3.2 million filled their active epinephrine autoinjector prescription, and of those, only half consistently carry their prescribed autoinjector with them due to the many drawbacks of these devices. In aggregate, we estimate that up to 90% of patients prescribed an epinephrine device are not achieving an optimal treatment outcome today.
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Risk Factors. ” Overview We are a biopharmaceutical company focused on the commercialization and development of neffy (currently identified in the European Union (“EU”) and United Kingdom (“U.K.”) by the trade name EURneffy and in China by the trade name 优敏速 ) for needle-free intranasal delivery of epinephrine for emergency treatment of Type I allergic reactions, including anaphylaxis. neffy is the first and only U.S.
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These drawbacks include the use of needles, which can result in patient and caregiver injury as well as hesitation and delays in administration due principally to apprehension and pain of needles, allowing the allergic reaction to progress in severity leading to symptoms that seriously impact patient quality of life, to potential need for emergency services and/or hospitalizations, and to life-threatening symptoms or events.
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Food and Drug Administration (“FDA”) and European Commission-approved needle-free epinephrine product, also has approvals in the U.K., Japan, Australia, and China.
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In particular, intra-muscular injections are subject to dosing errors and risk of accidental blood vessel injections, which can cause a significant spike in the intravascular delivery of epinephrine potentially leading to serious cardiovascular complications or events.
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We believe the market opportunity for neffy in the U.S. is significant.
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We believe neffy’s and our intranasal epinephrine technology product candidates’ design, particularly the compact size and “no needle, no injection” delivery, eliminates needle-related apprehension and pain, improves portability and ease of use, is highly reliable, and will increase prescriptions for epinephrine, making it more likely that patients and caregivers will administer epinephrine sooner, achieve more rapid symptom relief, and prevent the allergic reaction from progressing to a level of severity that could lead to hospitalization or even death.
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Our launch strategy for neffy in the United States involves direct outreach to high-volume prescribers of epinephrine accounting for approximately 55% of prescriptions in the last year through an efficient sales force.
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Data from our studies of neffy and our intranasal epinephrine technology product candidates demonstrated nasally delivered epinephrine reached blood levels comparable to those of already approved epinephrine injectable products across single dosing, repeat dosing, self-administration or allergen challenge conditions, and produced statistically significant responses compared to injection on pharmacodynamic surrogates for efficacy even one minute after dosing with neffy and our intranasal epinephrine technology product candidates. 106 On August 9, 2024, the FDA approved neffy 2 mg for the emergency treatment of Type I allergic reactions, including anaphylaxis, in adults and children who weigh 30 kg or greater.
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As of December 31, 2025, our sales force is comprised of approximately 106 ARS Pharma employees, who serve as sales reps, key account managers, area sales managers, and national sales directors, as well as 10 virtual sales reps, and approximately 70 sales reps via our co-promotion partner, ALK U.S., who began field operations in June 2025 and will target up to 9,000 specified pediatricians and other prescribers in the U.S.
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As a result, we initiated commercial launch of neffy 2 mg in the United States, with product becoming available for shipment on September 23, 2024.
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We plan to expand our internal sales force to approximately 150 individuals beginning in the second quarter of 2026.
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This commercialization effort currently includes a direct sales force of 118 individuals targeting high-volume epinephrine prescribers that is supported by branded direct-to-consumer marketing, disease awareness campaigns with advocacy groups and non-personal promotion such as non-personal promotion including continuing medical education programs in collaboration with allergist societies, speaker bureaus, peer-to-peer programs and participation in regional and national medical conferences.
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To reduce the time burden of an in-person healthcare provider visit, we also launched a new commercial initiative in November 2025 called “Get neffy on Us” that offers patients a free visit with a virtual prescriber, along with a $0 co-pay for eligible patients with commercial insurance.
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Through our collaboration with ALK (discussed below), we anticipate that EURneffy will be made available to patients in certain EU member states in 2025.
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We also initiated a U.S. post-marketing registry-based study for neffy for the treatment of anaphylaxis in oral food challenge or allergen immunotherapy clinics in the second quarter of 2025, which is ongoing.

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