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What changed in Monopar Therapeutics's 10-K — 2023 vs 2024

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Paragraph-level year-over-year comparison of Monopar Therapeutics's 2023 and 2024 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 2024 report.

+398 added−370 removedSource: 10-K (2025-03-31) vs 10-K (2024-03-28)

Top changes in Monopar Therapeutics's 2024 10-K

398 paragraphs added · 370 removed · 249 edited across 5 sections

Item 1. Business

Business — how the company describes what it does

75 edited+52 added−57 removed49 unchanged
Biggest changeFurther, the FDA may require that certain contraindications, warnings or precautions be included in the product labeling. In addition, the FDA may require Phase 4 testing which involves clinical studies designed to further assess pharmaceutical product safety and effectiveness and may require testing and surveillance programs to monitor the safety of approved products that have been commercialized.
Biggest changeIn addition, the FDA may require Phase 4 testing which involves clinical studies designed to further assess pharmaceutical product safety and effectiveness and may require testing and surveillance programs to monitor the safety of the approved product that has been commercialized. 15 Table of Contents Regulatory Framework in Australia The Therapeutic Goods Administration (“TGA”), through the Therapeutic Goods Act 1989 (the “Act”) and the Therapeutic Goods Regulations, is responsible for the efficacy, quality, safety and timely availability of drugs and medical devices in Australia.
The TGA is required by statute to complete its evaluation for approval of a medicine in the standard pathway within 255 working days. The priority review pathway has a target timeframe of 150 working days and allows for faster assessment of vital and life-saving prescription medicines.
The TGA is required by statute to complete its evaluation for approval of a medicine in the standard pathway within 255 working days. The priority review pathway has a target timeframe of 150 working days and allows for a faster assessment of vital and life-saving prescription medicines.
Clinical studies are conducted under protocols detailing, among other things, the objectives of the clinical study, dosing procedures, subject selection and exclusion criteria, how the results will be analyzed and presented and the parameters to be used to monitor subject safety. Each protocol must be submitted to the FDA as part of the IND.
Clinical studies are conducted under protocols detailing, among other things, the clinical study objectives, dosing procedures, subject selection and exclusion criteria, how the results will be analyzed and presented and the parameters to be used to monitor subject safety. Each protocol must be submitted to the FDA as part of the IND.
One or two Phase 3 studies are required by the FDA for an NDA or BLA approval, depending on the disease severity and other available treatment options. ● Post-approval studies, or Phase 4 clinical studies, may be conducted after initial marketing approval.
One or two Phase 3 studies are required by the FDA for an NDA or BLA approval, depending on the disease severity and other available treatment options. ● Phase 4. Post-approval studies, or Phase 4 clinical studies, may be conducted after initial marketing approval.
The FDA may refer applications for novel pharmaceutical products or pharmaceutical products which present difficult questions of safety or efficacy to an advisory committee, typically a panel that includes clinicians and other experts, for review, evaluation and a recommendation as to whether the application should be approved and under what conditions.
The FDA may refer applications for novel pharmaceutical products or pharmaceutical products which present difficult questions of safety or efficacy to an advisory committee, typically a panel that includes clinicians and other experts, for review, evaluation and recommendation as to whether the application should be approved and under what conditions.
Sponsors of promising new prescription medicines (with only preliminary clinical data available) can seek fast-tracked registration through the provisional approval pathway. All pathways require evidence that medicines are made according to Good Manufacturing Practice ("GMP"). GMP describes principles and procedures to ensure therapeutic goods are of high quality.
Sponsors of promising new prescription medicines (with only preliminary clinical data available) can seek fast-tracked registration through the provisional approval pathway. All pathways require evidence that the medicines are made according to Good Manufacturing Practice (“GMP”). GMP describes principles and procedures to ensure therapeutic goods are of high quality.
The FDA’s goal is to evaluate the protocol within 45 days of the request to assess whether the proposed trial is adequate, and that evaluation may result in discussions and a request for additional information. A SPA request must be made before the proposed trial begins, and all open issues must be resolved before the trial begins.
The FDA’s goal is to evaluate the protocol within 45 days of the request to assess whether the proposed trial is adequate, and that evaluation may result in discussions and a request for additional information. An SPA request must be made before the proposed trial begins, and all open issues must be resolved before the trial begins.
The trial sponsor must also comply with the requirements of any other Commonwealth and/or state and territory legislation in relation to clinical trials and the supply of therapeutic goods. A company or organization wishing to supply a therapeutic good in Australia must apply for market authorization from the TGA.
The trial sponsor must also comply with the requirements of any other relevant Commonwealth and/or state and territory legislation in relation to clinical trials and the supply of therapeutic goods. A company or organization wishing to supply a therapeutic good in Australia must apply for market authorization from the TGA.
This application covers novel compositions and uses of cytotoxic radioisotopes attached to antibodies that bind to uPAR, thereby creating precision targeted radiotherapeutics, also known as uPRITs, for the treatment of severe COVID-19 and other respiratory diseases.
This application covers novel compositions and uses of imaging and cytotoxic radioisotopes attached to antibodies that bind to uPAR, thereby creating precision targeted radiotherapeutics, also known as uPRITs, for the treatment of severe COVID-19 and other respiratory diseases.
The FDA reviews all NDAs and BLAs submitted before it accepts them for filing and may request additional information rather than accepting an NDA or BLA for filing. Once the submission is accepted for filing, the FDA begins an in-depth review of the NDA or BLA.
The FDA reviews all NDAs and BLAs submitted before it accepts them for filing and may request additional information rather than accepting an NDA or BLA for filing. Once a submission is accepted for filing, the FDA begins an in-depth review of the NDA or BLA.
If the FDA determines the application, manufacturing process or manufacturing facilities are not acceptable, it will outline the deficiencies in the submission and often will request additional testing or information. In addition, the FDA will require the review and approval of product labeling.
If the FDA determines that the application, manufacturing process or manufacturing facilities are not acceptable, it will outline the deficiencies in the submission and often will request additional testing or information. In addition, the FDA will require the review and approval of product labeling.
We face competition from pharmaceutical and biotechnology companies, many of which are larger and better financed than us. We also face competition in our efforts to develop and commercialize new oncology therapeutics from academic and government laboratories.
We face competition from both pharmaceutical and biotechnology companies, many of which are larger and better financed than us. We also face competition from academic and government laboratories in our efforts to develop and commercialize new oncology therapeutics.
According to FDA guidance for industry on the SPA process, a sponsor which meets the prerequisites may make a specific request for a SPA and provide information regarding the design and size of the proposed clinical trial.
According to FDA guidance for industry on the SPA process, a sponsor which meets the prerequisites may make a specific request for an SPA and provide information regarding the design and size of the proposed clinical trial.
The submission of an NDA or BLA is subject to the payment of substantial user fees; a waiver of such fees may be obtained under certain limited circumstances. 19 Table of Contents In addition, under the Pediatric Research Equity Act (“PREA”), an NDA, BLA or a supplement thereof must contain data to assess the safety and effectiveness of the pharmaceutical product for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective.
The submission of an NDA or BLA is subject to the payment of substantial user fees; a waiver of such fees may be obtained under certain limited circumstances. 14 Table of Contents In addition, under the Pediatric Research Equity Act (“PREA”), an NDA, BLA or a supplement thereof must contain data to assess the safety and effectiveness of the pharmaceutical product for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective.
There are several companies that are developing radiopharmaceuticals for cancers including, but not limited to: Bayer AG, Bristol Myers Squibb, Eli Lilly and Company, Novartis AG, Actinium Pharmaceuticals, Inc., Johnson & Johnson, Telix Pharmaceuticals Limited, Lantheus Holdings, Inc. and Genentech, as well as several early-stage companies that are developing a wide range of targeted radiopharmaceuticals for advanced cancers.
There are several companies that are developing radiopharmaceuticals for cancers including, but not limited to: Novartis AG, Bayer AG, Bristol Myers Squibb, Eli Lilly and Company, Actinium Pharmaceuticals, Inc., Johnson & Johnson, Telix Pharmaceuticals Limited, Lantheus Holdings, Inc., AstraZeneca, and Genentech, as well as numerous early-stage companies that are developing a wide range of targeted radiopharmaceuticals for advanced cancers.
We intend to satisfy any disclosure requirement under Item 5.05 of Form 8-K regarding an amendment to, or waiver from, a provision of our Code of Business Conduct and Ethics by posting such information on our website as specified above. 22 Table of Contents
We intend to satisfy any disclosure requirement under Item 5.05 of Form 8-K regarding an amendment to, or waiver from, a provision of our Code of Business Conduct and Ethics by posting such information on our website as specified above. 19 Table of Contents
The TGA may decide to seek advice from an expert advisory committee, such as the Advisory Committee for Complementary Medicines (ACCM); ● The decision: When making the decision under section 26AE of the Therapeutics Goods Act (1989) on whether to list the medicine in the ARTG, the decision maker (the delegate of the Secretary of the Department of Health) will review all documentation associated with the application, including the dossier, evaluation reports, responses to requests for information, and advice from expert advisory committees; ● Finalization: Sponsors need to provide a patent certificate under subsection 26B(1) of the act, or notification that this is not required before the medicine can be listed in the ARTG; ● Conduct post-marketing requirements, if any: a drug may be selected for a post-market compliance review at any time.
The TGA may decide to seek advice from an expert advisory committee, such as the Advisory Committee for Complementary Medicines (“ACCM”); ● The decision: When making the decision under section 26AE of the Act on whether to list the medicine in the ARTG, the decision maker (the delegate of the Secretary of the Department of Health) will review all documentation associated with the application, including the dossier, evaluation reports, responses to requests for information, and advice from expert advisory committees; ● Finalization: Sponsors need to provide a patent certificate under subsection 26B(1) of the Act, or notification that this is not required before the medicine can be listed in the ARTG; ● Conduct post-marketing requirements, if any: a drug may be selected for a post-market compliance review at any time.
The TGA assesses the application, and if market authorization is granted, the therapeutic good is entered on the Australian Register of Therapeutic Goods ("ARTG"). The TGA uses three pathways to evaluate a prescription medicine: the standard pathway, the priority review pathway and the provisional approval pathway.
The TGA assesses the application, and if market authorization is granted, the therapeutic good is entered on the Australian Register of Therapeutic Goods (“ARTG”). The TGA uses three pathways to evaluate a prescription medicine: the standard pathway, the priority review pathway and the provisional approval pathway.
Additionally, before approving an NDA or BLA, the FDA will typically inspect one or more clinical sites as well as the site where the pharmaceutical product is manufactured to assure compliance with GCP and cGMP.
Additionally, before approving an NDA or BLA, the FDA will typically inspect one or more clinical sites as well as the site(s) where the pharmaceutical product is manufactured to assure compliance with GCP and cGMP, respectively.
The EMA also has designations for Orphan Drugs, which, if applicable, can provide for faster review, lower fees and more access to advice during drug development. While the marketing authorization in the European Union is centralized, the system for clinical studies (application, review and requirements) is handled by each individual country.
The EMA also has designations for Orphan Drugs, which, if applicable, can provide for faster review, lower fees and more access to advice during drug development. While the marketing authorization in the EU is centralized, the system for clinical studies (application, review and requirements) is handled by each individual country.
Our product candidates will also have to compete with alternate treatment modalities, such as improvements in radiation treatments, which are also subject to continual innovation and improvement.
Our product candidates will also have to compete with alternate treatment modalities, such as improvements in radiation treatments, which are also subject to continuous innovation and improvement.
However, there are a number of ways that patients can gain access to products that have not been approved for use in Australia: ● The Special Access Scheme ("SAS") allows a health practitioner to access an unapproved therapeutic good for an individual patient on a case-by-case basis; ● Medical professionals can apply to the TGA to become an ‘Authorised Prescriber’ of a specific unapproved good to specific patients with a particular medical condition.
However, there are several ways that patients can gain access to products that have not been approved for use in Australia: ● The Special Access Scheme (“SAS”) allows a health practitioner to access an unapproved therapeutic good for an individual patient on a case-by-case basis; ● Medical professionals can apply to the TGA to become an ‘Authorised Prescriber’ of a specific unapproved good to specific patients with a particular medical condition.
Accordingly, it is not certain that submission of an IND will result in the FDA allowing clinical studies to begin, or that, once begun, issues will not arise that suspend or terminate such clinical studies. During the development of a new drug, sponsors are given opportunities to meet with the FDA at certain points.
Accordingly, it is not certain that submission of an IND will result in the FDA allowing clinical studies to begin, or that, once begun, issues will not arise that may suspend or terminate such clinical studies. 13 Table of Contents During the development of a new drug, sponsors are given opportunities to meet with the FDA at certain points.
These studies are used to gain additional experience from the treatment of patients in the intended therapeutic indication. ● Progress reports detailing the results of the clinical studies must be submitted at least annually to the FDA and written IND safety reports must be submitted to the FDA and the investigators for serious and unexpected adverse events or any finding from tests in laboratory animals that suggests a significant risk for human subjects.
These studies are used to gain additional insight from the treatment of patients in the intended therapeutic indication. ● P rogress reports detailing the results of the clinical studies must be submitted at least annually to the FDA and written IND safety reports must be submitted to the FDA and the investigators for serious and unexpected adverse events or any finding from tests in laboratory animals that suggests a significant risk for human subjects.
The FDA will not approve the product unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to assure consistent production of the product within required specifications.
The FDA will not approve the product unless it determines that the manufacturing processes and facilities are compliant with cGMP requirements and adequate to assure consistent production of the product within required specifications.
In some instances, doctors also need to have their application approved by a human research ethics committee or endorsed by a specialist college; ● Depending on the level of risk involved, a sponsor of a clinical trial can make either a notification or application to the TGA to use an unapproved good in the trial; ● Under the Personal Importation Scheme, individuals can legally import a three-month supply of some unapproved therapeutic goods for personal use, without TGA approval.
In some instances, doctors also need to have their applications approved by a human research ethics committee or endorsed by a specialist college; ● Depending on the level of risk involved, the sponsor of a clinical trial can either notify or apply to the TGA to use an unapproved good in the trial; ● Under the Personal Importation Scheme, individuals can legally import a three-month supply of some unapproved therapeutic goods for personal use without TGA approval.
The mission statement of the TGA is “To ensure the safety, quality and efficacy of therapeutic goods available in Australia at a standard equal to that of comparable countries, and that premarket assessment of therapeutic goods is conducted within a reasonable time." The TGA administers two pathways for clinical trials, the Clinical Trials Notification ("CTN") and Clinical Trials Approval ("CTA") schemes.
The mission statement of the TGA is “To ensure the safety, quality and efficacy of therapeutic goods available in Australia at a standard equal to that of comparable countries, and that premarket assessment of therapeutic goods is conducted within a reasonable time.” The TGA administers two pathways for clinical trials, the Clinical Trials Notification (“CTN”) and Clinical Trials Approval (“CTA”) schemes.
Clinical studies must be conducted in accordance with Good Clinical Practice (“GCP”) guidelines. Further, each clinical study must be reviewed and approved by an independent institutional review board (“IRB”), at, or servicing, each institution at which the clinical study will be conducted.
Clinical studies must be conducted in accordance with GCP guidelines. Further, each clinical study must be reviewed and approved by an independent institutional review board (“IRB”), at, or servicing, each institution at which the clinical study will be conducted.
The process required by the FDA before a non-biological pharmaceutical product may be marketed in the U.S. generally involves the following: ● Completion of preclinical laboratory tests, animal studies and formulation studies according to Good Laboratory Practices (“GLP”), and other applicable regulations; ● Submission to the FDA of an Investigational New Drug application (“IND”), which must become effective before human clinical studies may begin; ● Performance of adequate and well-controlled human clinical studies according to the FDA’s current Good Clinical Practices (“GCP”), to establish the safety, efficacy and optimum dose of the proposed pharmaceutical product for its intended use; ● Submission to the FDA of a New Drug Application (“NDA”) or Biologics License Application (“BLA”), for a new pharmaceutical product; ● Satisfactory completion of an FDA inspection of the manufacturing facility or facilities where the pharmaceutical product is produced to assess compliance with the FDA’s current Good Manufacturing Practice standards (“cGMP”), to assure that the facilities, methods and controls are adequate to preserve the pharmaceutical product’s identity, strength, quality and purity; ● FDA audits of the preclinical and clinical study sites that generated the data in support of the NDA or BLA; ● FDA review and approval of the NDA; and ● Fulfillment of FDA post-marketing requirements, if any. 17 Table of Contents The lengthy process of seeking required approvals and the continuing need for compliance with applicable statutes and regulations require the expenditure of substantial resources, and approvals are inherently uncertain.
The process required by the FDA before a non-biological pharmaceutical product may be marketed in the U.S. generally involves the following: ● Completion of preclinical laboratory tests, animal studies and formulation studies according to Good Laboratory Practices (“GLP”), and other applicable regulations; ● Submission to the FDA of an Investigational New Drug application (“IND”), which must become effective before human clinical studies may begin; ● Performance of adequate and well-controlled human clinical studies according to the FDA’s current Good Clinical Practices (“GCP”), to establish the safety, efficacy and optimum dose of the proposed pharmaceutical product for its intended use; ● Submission to the FDA of an NDA or Biologics License Application (“BLA”), for a new pharmaceutical product; ● Satisfactory completion of an FDA inspection of the manufacturing facility or facilities where the pharmaceutical product is produced to assess compliance with the FDA’s current Good Manufacturing Practice standards (“cGMP”), to assure that the facilities, methods and controls are adequate to preserve the pharmaceutical product’s identity, strength, quality and purity; ● FDA audits of the preclinical and clinical study sites that generated the data in support of the NDA or BLA; ● FDA review and approval of the NDA; and ● Fulfillment of FDA post-marketing requirements, if any.
Even if such data and information are submitted, the FDA may ultimately decide that the NDA or BLA does not satisfy the criteria for approval. Data obtained from clinical studies are not always conclusive and the FDA may interpret data differently than the sponsor interprets the same data.
Even if such data and information are submitted, the FDA may ultimately decide that the NDA or BLA does not satisfy the criteria for approval. Data obtained from clinical studies are not always conclusive and the FDA may interpret data differently than the sponsor's interpretations of the same data.
Also in May 2021, we and NorthStar filed a provisional composition of matter patent application titled “Urokinase Plasminogen Activator Receptor-Targeted Radiopharmaceutical” covering a radiotherapeutic consisting of our proprietary antibody MNPR-101 bound to Ac-225 via the isotope binding agent PCTA.
Also in May 2021, we and NorthStar filed a provisional composition of matter patent application titled “Urokinase Plasminogen Activator Receptor-Targeted Radiopharmaceutical,” which covers a radiotherapeutic consisting of our proprietary antibody MNPR-101 bound to Ac-225 via the isotope binding agent PCTA.
The IRB also approves the informed consent form that must be provided to each clinical study subject or his or her legal representative and must monitor the clinical study until completed. 18 Table of Contents Human clinical studies are typically conducted in three sequential phases that may overlap or be combined: ● Phase 1.
The IRB also approves the informed consent form that must be provided to each clinical study subject or his/her legal representative and monitors the clinical study until completed. Human clinical studies are typically conducted in three sequential phases that may overlap or be combined: ● Phase 1.
As of March 8, 2024, we had not reached any milestones and had not been required to pay XOMA Ltd. any funds under this license agreement. The first milestone payment is payable upon first dosing of a human patient in a Phase 2 clinical trial. Onxeo S.A.
As of March 14, 2025, we had not reached any milestones and had not been required to pay XOMA Ltd. any funds under this license agreement. The first milestone payment is payable upon first dosing of a human patient in a Phase 2 clinical trial.
The study will be conducted at Melbourne Theranostic Innovation Centre ("MTIC")headed by Professor Rodney Hicks, MBBS(Hons), MD, FRACP, FICIS, FAAHMS, and will use one of the world's most sensitive clinical total-body PET/CT scanners, the Siemens Biograph Vision Quadra, to image the targeting ability of MNPR-101-Zr in cancer patients.
The study is being conducted at Melbourne Theranostic Innovation Centre (“MTIC”), headed by Professor Rodney Hicks, MBBS(Hons), MD, FRACP, FICIS, FAAHMS, and uses one of the world's most sensitive clinical total-body PET/CT scanners, the Siemens Biograph Vision Quadra, to image the targeting ability of MNPR-101-Zr in cancer patients.
In addition to regulations in Europe, Canada, Japan, Australia and the U.S., there are a variety of foreign regulations governing clinical studies, commercial distribution and reimbursement of future product candidates which we may be subject to as we pursue regulatory approval of the MNPR-101 radiopharmaceutical program, camsirubicin, or any future product candidates internationally. 21 Table of Contents Compliance with Environmental Laws Since we do not have our own laboratory or manufacturing facilities, we do not estimate any annual costs of compliance with environmental laws.
In addition to regulations in Europe, Canada, Japan, Australia and the U.S., there are a variety of foreign regulations governing clinical studies, commercial distribution and reimbursement of future product candidates which we may be subject to as we pursue regulatory approval of ALXN1840, the MNPR-101 radiopharmaceutical program, or any future product candidates internationally. 18 Table of Contents Compliance with Environmental Laws Historically, we have not operated our own laboratory or manufacturing facilities.
The pharmaceutical product candidates that we develop must be approved by the U.S. Food and Drug Administration ("FDA") and the Therapeutics Goods Administration ("TGA") before they may be legally marketed in the U.S. and Australia, respectively. See “Risk Factors – Risks Related to Clinical Development and Regulatory Approval”.
The pharmaceutical product candidates that we develop must be approved by the FDA and the Therapeutics Goods Administration (“TGA”) before they may be legally marketed in the U.S. and Australia, respectively. See “Risk Factors – Risks Related to Clinical Development and Regulatory Approval.” U.S.
We anticipate hiring additional employees in clinical operations, regulatory affairs and other departments, to help manage our clinical studies, regulatory submissions, and manufacturing to support the MNPR-101 program and camsirubicin development, business development and corporate strategy.
We anticipate hiring additional employees in regulatory affairs, clinical operations, and other departments, to help manage regulatory submissions, clinical studies, manufacturing, business development and corporate strategy.
Under E.U. regulatory systems, marketing applications for pharmaceutical products are typically submitted under a centralized procedure to the EMA. The centralized procedure provides for the granting of a single marketing authorization that is valid for all E.U. member states.
Under the European Union (“EU”) regulatory systems, marketing applications for pharmaceutical products are typically submitted under a centralized procedure to the European Medicines Agency (“EMA”). The centralized procedure provides for the granting of a single marketing authorization that is valid for all EU member states.
In December 2015, we converted to a Delaware C corporation. Our principal executive offices are located at 1000 Skokie Blvd, Suite 350, Wilmette, IL 60091. Our telephone number is (847) 388-0349. Our corporate website is located at www.monopartx.com .
Corporate Information We were formed as a Delaware limited liability company in December 2014, with the name Monopar Therapeutics, LLC. In December 2015, we converted to a Delaware C corporation. Our principal executive offices are located at 1000 Skokie Blvd, Suite 350, Wilmette, IL 60091. Our telephone number is (847) 388-0349. Our corporate website is located at www.monopartx.com .
Employees Our operations are currently managed by five individuals (including our executive chairman and our Acting Chief Medical Officer), of whom two have a PhD, two have an MD, two have an MBA, one has an MSc in health economics and policy, one has an MS from Stanford University, and one is a former CPA.
Employees Our operations are currently managed by six individuals (including our executive chairman and our Acting Chief Medical Officer), of whom two have a PhD, two have an MD, two have an MBA, one has an MS in health economics and policy, one has an MS in electrical engineering and one has an MS in legal studies.
Being a novel biologic, it is eligible for 12 years of exclusivity in the U.S. under the Biologics Price Competition and Innovation Act (“BPCI Act”), and it will benefit from varying durations of similar exclusivity in numerous other countries. The Radio-Immuno-Therapeutic derivative of MNPR-101 ("uPRIT") patent, if granted expires in 2041.
Being a novel biologic, MNPR-101 is eligible for 12 years of exclusivity in the U.S. under the Biologics Price Competition and Innovation Act (“BPCI Act”), and it will benefit from varying durations of similar exclusivity in numerous other count ries. The earliest to expire of the patent applications for radiopharmaceutical derivatives of MNPR-101, if granted, would expire in 2041.
Additional information can be found in the section entitled “Risk Factors – Risks Related to Our Business Operations and Industry.” 14 Table of Contents MNPR-101 Radiopharmaceutical Program Competition Our MNPR-101 radiopharmaceutical program, including MNPR-101-Zr and MNPR-101 conjugated to therapeutic radioisotopes, is susceptible to all of the competitive factors listed in the first paragraph of this section on Oncology Market Competition.
Additional information can be found in the section entitled “Risk Factors – Risks Related to Our Business Operations and Industry.” 12 Table of Contents MNPR-101 Radiopharmaceutical Program Competition Our MNPR-101 radiopharmaceutical program, including MNPR-101-Zr for imaging and MNPR-101-Lu and MNPR-101-Ac for therapy, is susceptible to all the competitive factors listed above under Oncology Market Competition.
They have worked at industry leading companies such as BioMarin Pharmaceutical Inc., Raptor Pharmaceuticals, and Onyx Pharmaceuticals. As of March 8, 2024, we had ten employees; nine of whom were full-time.
They have worked at industry leading companies such as BioMarin Pharmaceutical Inc., Raptor Pharmaceuticals, Abbott Laboratories, Onyx Pharmaceuticals, and Amgen. As of March 14, 2025 , we had sixteen employees; fourteen of whom were full-time.
In addition, to complement our internal expertise, we have contracts with medical and scientific consultants, manufacturers, laboratories, and contract research organizations that specialize in various aspects of drug development including clinical development, preclinical development, manufacturing, quality assurance, and regulatory affairs. Corporate Information We were formed as a Delaware limited liability company in December 2014, with the name Monopar Therapeutics, LLC.
In addition, to complement our internal expertise, we have contracts with medical and scientific consultants, manufacturers, laboratories, and contract research and development organizations that specialize in various aspects of drug development including clinical development, preclinical development, manufacturing, quality assurance, and regulatory affairs.
MNPR-101 for Radiopharmaceutical Use In collaboration with NorthStar, we filed a provisional patent application entitled “Precision Radioimmunotherapeutic Targeting of the Urokinase Plasminogen Activator Receptor (uPAR) for Treatment of Severe COVID-19 Disease” with the USPTO on June 15, 2020.
In collaboration with NorthStar, we filed a provisional patent application entitled “Precision Radioimmunotherapeutic Targeting of uPAR for Treatment of Severe COVID-19 Disease” with the U.S. Patent and Trademark Office (“USPTO”) on June 15, 2020.
For the uPAR-targeted radiopharmaceuticals, CuraSight, a Danish biotech company, is currently developing a non-antibody-based uPAR radiodiagnostic and radiotherapeutic pair which binds to a different epitope on uPAR as compared to MNPR-101. Camsirubicin Competition We believe our camsirubicin program, if approved, could replace doxorubicin as the first-line treatment for ASTS.
For the uPAR-targeted radiopharmaceuticals, CuraSight, a Danish biotech company, is currently developing a clinical-stage non-antibody-based uPAR radiodiagnostic and radiotherapeutic pair which binds to a different epitope on uPAR as compared to MNPR-101.
Item 1. Business You should read the following discussion in conjunction with our financial statements as of December 31, 2023, and the notes to such financial statements included elsewhere in this Annual Report on Form 10-K. Overview We are a clinical stage biopharmaceutical company focused on developing innovative treatments for cancer patients.
Item 1. Business You should read the following discussion in conjunction with our financial statements as of December 31, 2024, and the notes to such financial statements included elsewhere in this Annual Report on Form 10-K. Overview Monopar Therapeutics Inc.
Some of our patents are currently near expiration and we may pursue patent term extensions for these where appropriate or to let patents lapse. See “Risk Factors – Risks Related to our Intellectual Property”.
Some of our patents are currently near expiration and we may pursue patent term extensions for these where appropriate.
Under the terms of the non-exclusive license with XOMA Ltd., we are to pay only upon clinical, regulatory and sales milestones which could reach up to $14.925 million if we achieve all milestones. The agreement does not require the payment of sales royalties. There can be no assurance that we will reach any milestones.
Under the terms of the non-exclusive license with XOMA Ltd., we are to pay only upon the achievement of clinical, regulatory and sales milestones, potentially totaling $14.925 million. The agreement does not require the payment of sales royalties.
The patents covering the composition of matter of MNPR-101 will expire in 2025 and the patents covering the MNPR-101 epitope will expire in 2027.
The U.S. patent covering the composition of matter of MNPR-101 will expire in 2027 with the corresponding Japanese patent expiring in 2025, and the U.S. patent covering the MNPR-101 epitope will expire in 2029 with corresponding Japanese patent expiring in 2027.
Positron emission tomography (“PET”) imaging data of preclinical human tumor xenograft mouse models for triple-negative breast, colorectal, and pancreatic tumors expressing uPAR display high, selective and durable uptake of MNPR-101 conjugated to the imaging radioisotope zirconium-89 ("MNPR-101-Zr"). Additionally, preclinical triple-negative breast and pancreatic cancer mouse model studies with MNPR-101 conjugated to therapeutic radioisotopes ("MNPR-101-RIT") have shown promising anti-tumor activity.
We have demonstrated promising preclinical data to-date for our MNPR-101 radiopharmaceutical program. Positron emission tomography (“PET”) imaging data of preclinical human tumor xenograft mouse models for triple-negative breast, colorectal, and pancreatic tumors expressing uPAR display high, selective and durable uptake of MNPR-101-Zr, our imaging agent.
The FDA may also impose clinical holds on a pharmaceutical product candidate at any time before or during clinical studies due to safety concerns or non-compliance.
If resolution cannot be reached within the 30-day review period, either the FDA places the IND on clinical hold, or the sponsor withdraws the application. The FDA may also impose clinical holds on a pharmaceutical product candidate at any time before or during clinical studies due to safety concerns or non-compliance.
Additionally, the complete response letter may include recommended actions that the applicant might take to place the application in a condition for approval.
Additionally, the complete response letter may include recommended actions that the applicant might take to place the application in an updated condition for approval. If a complete response letter is issued, the applicant may either resubmit the NDA or BLA, addressing all the deficiencies identified in the letter, or withdraw the application.
Failure to comply with the applicable U.S. requirements at any time during the product development process, approval process or after approval, may subject an applicant to administrative or judicial enforcement.
The process of obtaining regulatory approvals and the subsequent compliance with appropriate federal, state, local and foreign statutes and regulations require the expenditure of substantial time and financial resources. Failure to comply with the applicable U.S. requirements at any time during the product development, approval or after approval process may subject an applicant to administrative or judicial enforcement.
Phase 4 may be conducted for post-marketing surveillance or resolution of treatment uncertainties. Clinical development pathways are becoming less rigid with respect to phase and seamless adaptive trial designs and other cross-phase studies exist.
Clinical development pathways are becoming less rigid with respect to phase, seamless adaptive trial designs and other cross-phase studies.
This RIT demonstrated 98% radiochemical purity and high stability and has the potential to be a highly selective, potent treatment for a variety of cancers, severe COVID-19, and other diseases characterized by aberrant uPAR expression. 13 Table of Contents Camsirubicin Camsirubicin (GPX-150) is covered by manufacturing process patents.
This radiopharmaceutical demonstrated 98% radiochemical purity and high stability and has the potential to be a highly selective, potent treatment for a variety of cancers, severe COVID-19, and other diseases characterized by aberrant uPAR expression. On June 11, 2024, Monopar and Northstar announced that the companies amended and extended their radiopharmaceutical collaboration.
Other International Regulation In addition to regulations in the U.S. and Australia, there are a variety of foreign regulations governing clinical studies and commercial sales and distribution of our future product candidates.
In some instances, pharmacists are allowed to substitute medicines, including different strengths or forms of a product, without a prescribing doctor’s approval where a medicine is unavailable. 17 Table of Contents Other International Regulations In addition to regulations in the U.S. and Australia, there are a variety of foreign regulations governing clinical studies and commercial sales and distribution of our future product candidates.
Moreover, several Phase 1 PET imaging studies in advanced cancer patients show that uPAR can only be detected in the tumor and not in normal tissues, making it a potentially attractive target for cancer therapies, including radiopharmaceuticals.
For example, it is estimated that the tumors and/or tumor associated cells in 97% of breast, 89% of bladder, 87% of pancreatic and 85% of colorectal cancer patients express uPAR. Moreover, several Phase 1 PET imaging studies in advanced cancer patients show that uPAR can be clearly detected in tumors, making it a potentially attractive target for radiopharmaceuticals.
Before testing any compounds with potential therapeutic value in humans, the pharmaceutical product candidate enters the preclinical testing stage. Preclinical tests include laboratory evaluations of product chemistry, toxicity and formulation, as well as in-vitro and animal studies to assess the potential safety and activity of the pharmaceutical product candidate.
Preclinical tests include laboratory evaluations of product chemistry, toxicity and formulation, as well as in-vitro and animal studies to assess the potential safety and activity of the pharmaceutical product candidate. These early proof-of-principle studies are done using sound scientific procedures and thorough documentation.
The sponsor must submit the results of the preclinical tests, together with manufacturing information, analytical data, any available clinical data or literature and a proposed clinical protocol, to the FDA as part of the IND. The IND automatically becomes effective 30 days after receipt by the FDA, unless the FDA has concerns and notifies the sponsor.
The conduct of single and repeat dose toxicology and toxicokinetic studies in animals must comply with federal regulations and requirements, including GLP. The sponsor must submit the results of the preclinical tests, together with manufacturing information, analytical data, any available clinical data or literature and a proposed clinical protocol, to the FDA as part of the IND.
If a complete response letter is issued, the applicant may either resubmit the NDA or BLA, addressing all of the deficiencies identified in the letter, or withdraw the application. 20 Table of Contents If a product receives regulatory approval, the approval may be significantly limited to specific diseases and dosages or the indications for use may otherwise be limited, which could restrict the commercial value of the product.
If a product receives regulatory approval, the approval may be significantly limited to specific diseases and dosages or the indications for use may otherwise be limited, which could restrict the commercial value of the product. Further, the FDA may require that certain contraindications, warnings or precautions be included in the product labeling.
MNPR-101 is designed to selectively bind to uPAR. uPAR is highly expressed in multiple types of tumors, including breast, pancreatic, and colorectal cancers but not on most normal cells. It is estimated that the tumors and/or tumor associated cells in 97% of breast, 89% of bladder, 87% of pancreatic and 85% of colorectal cancer patients express uPAR.
The program uses MNPR-101 to target uPAR as a means to accurately deliver radioisotope payloads to the tumors. uPAR is highly expressed in multiple types of tumors, including breast, pancreatic, and colorectal cancers but not on most normal cells.
MNPR-101 Our patent portfolio for our MNPR-101 antibody (huATN-658), as well as its epitope, consists of two issued U.S. composition of matter and their methods of use patents and corresponding (granted and pending) patents and patent applications in multiple foreign jurisdictions, including the European Union, Japan, and other Asian countries. These patents are owned by us.
The patents cover methods of treatment and dosing of ALXN1840 for Wilson disease, methods of manufacture for tetrathiomolybdate, and certain drug product formulations. MNPR-101 Our patent portfolio for our MNPR-101 antibody as well as its epitope consists of two issued U.S. composition of matter and their methods of use patents and corresponding issued patents in Japan.
We have not incurred any license or royalty obligations and the license has been terminated effective January 2024. 12 Table of Contents Intellectual Property Portfolio and Exclusivity An important part of our strategy is obtaining patent protection to help preserve the proprietary nature of our product candidates, and to prevent others from developing competitive agents that are similar.
We are currentl y conducting Phase 1 clinical trials and cannot reliably predict when we will be able to commence a Phase 2 clinical trial, if at all. 10 Table of Contents Intellectual Property Portfolio and Exclusivity An important part of our strategy is obtaining patent protection to help preserve the proprietary nature of our product candidates, and to prevent others from developing similar competitive agents.
We presently depend on third-party contract manufacturers for all our required raw materials, Active Pharmaceutical Ingredients (“API”), and finished drug products for our preclinical and clinical studies. We are having clinical batches of MNPR-101-Zr manufactured in preparation for treating patients in our Phase 1 radiopharma program in advanced cancers.
We expect to depend on third-party contract manufacturers for all our required raw materials, Active Pharmaceutical Ingredients (“API”), and finished drug products.
We are building a drug candidate pipeline through in-house development as well as the licensing and acquisition of therapeutics in late preclinical and in clinical development stages. We leverage our scientific and clinical experience to help reduce the risk and accelerate the clinical development of our drug product candidates.
We build our drug development pipeline through both in-house efforts and licensing of late preclinical and clinical-stage therapeutics, leveraging our scientific and clinical expertise to mitigate risk and to accelerate development.
Manufacturing We do not currently own or operate manufacturing facilities for the production or testing of MNPR-101 radiopharmaceutical program, camsirubicin, or MNPR-202, nor do we have plans to develop our own manufacturing operations in the foreseeable future.
In April 2024, Monopar announced the filing of a provisional patent protecting certain MNPR-101 radiopharmaceutical optimization inventions, and in October 2024, Monopar announced a further addition to its radiopharmaceutical intellectual property portfolio with a provisional patent filing on new radiopharmaceutical compounds and linkers. 11 Table of Contents Manufacturing ALXN1840 We do not currently own or operate manufacturing facilities for the production or testing of ALXN1840, nor do we have plans to develop our own manufacturing operations in the foreseeable future.
In such a case, the IND sponsor and the FDA must resolve any outstanding concerns before the clinical study can begin. If resolution cannot be reached within the 30-day review period, either the FDA places the IND on clinical hold, or the sponsor withdraws the application.
The IND automatically becomes effective 30 days after receipt by the FDA, unless the FDA has concerns and notifies the sponsor. In such a case, the IND sponsor and the FDA must resolve any outstanding concerns before the clinical study can begin.
In some instances, pharmacists are allowed to substitute medicines, including different strengths or forms of a product, without a prescribing doctor’s approval where a medicine is unavailable. U.S. Pharmaceutical Product Development Process In the U.S., the FDA regulates pharmaceutical products under the Federal Food, Drug and Cosmetic Act (“FDCA”) and implementing regulations.
Pharmaceutical Product Development Process In the U.S., the FDA regulates pharmaceutical products under the Federal Food, Drug and Cosmetic Act (“FDCA”) and implements regulations. Pharmaceutical products are also subject to other federal, state and local statutes and regulations.
The choice of which route to use (CTN or CTA) lies firstly with the Australian clinical trial sponsor and then with the Human Research Ethics Committee ("HREC") that approves the protocol. 15 Table of Contents Clinical trials of medicines and biologicals typically proceed through 'phases' of development, which generally follow: Phase 1 (human pharmacology), Phase 2 (therapeutic exploratory), and Phase 3 (therapeutic confirmatory).
These schemes provide avenues through which unapproved therapeutic goods may be lawfully supplied for use solely for experimental purposes in humans. The choice of which route to use (CTN or CTA) lies firstly with the Australian clinical trial sponsor and then with the Human Research Ethics Committee (“HREC”) that approves the protocol.
In May 2021, we and NorthStar filed a provisional patent application with the USPTO titled “Bio-Targeted Radiopharmaceutical Compositions Containing Ac-225 and Methods of Preparation.” Radiopharmaceutical therapy is a promising approach to treat cancer and other diseases using radioactive isotopes bound with proteins/antibodies to target and kill cells.
In May 2021, we and NorthStar filed a provisional patent application with the USPTO titled “Bio-Targeted Radiopharmaceutical Compositions Containing Ac-225 and Methods of Preparation.” A full international patent application (International Application Number PCT/US2022/0378956) titled, “Trivalent Radioisotope Bio-Targeted Radiopharmaceutical, Methods of Preparation and Use” that claims priority to the provisional filing date was filed under the PCT on May 20, 2022.
Overall, the preclinical imaging and therapeutic efficacy study results demonstrate the potential utility of MNPR-101 as a precision targeting radiopharmaceutical agent for both imaging and therapy in multiple cancer indications. We are actively working towards enrolling our Phase 1 dosimetry clinical trial in Australia for MNPR-101-Zr in patients with advanced cancer.
Additionally, preclinical triple-negative breast and pancreatic cancer mouse model studies with MNPR-101 conjugated to therapeutic radioisotopes lutetium-177 and actinium-225 have shown promising anti-tumor activity. Overall, the preclinical imaging and therapeutic efficacy study results demonstrate the potential utility of MNPR-101 as a precision targeting radiopharmaceutical agent for both imaging and therapy in multiple cancer types.
In February 2024, we received Human Research Ethics Committee (HREC) clearance in Australia to commence our Phase 1 dosimetry trial for MNPR-101-Zr in patients with advanced cancers. The trial will utilize total body PET/CT ("positron emission tomography–computed tomography") imaging to assess tumor uptake, normal organ biodistribution, and safety.
We are currently actively enrolling our Phase 1 imaging/dosimetry and our therapeutic clinical trials in Australia for MNPR-101-Zr and MNPR-101-Lu in patients with advanced cancer, respectively. 8 Table of Contents MNPR-101-Zr Phase 1 Imaging and Dosimetry Clinical Trial In February 2024, we received Human Research Ethics Committee (“HREC”) clearance in Australia to commence a first-in-human Phase 1 imaging and dosimetry clinical trial for MNPR-101-Zr in patients with advanced solid tumors.
Also, we have completed manufacturing of the clinical batches of drug product for camsirubicin which are being used in our ongoing Phase 1b dose-escalation camsirubicin clinical trial. Oncology Market Competition The pharmaceutical industry in general, and the oncology therapeutics sector in particular, are characterized by intense competition.
We are aware of two gene therapies that are in clinical development for the treatment of Wilson disease: UX701 (Ultragenyx Pharmaceutical Inc.) and VTX-801 (Vivet Therapeutics). Oncology Market Competition The pharmaceutical industry in general, and the oncology therapeutics sector in particular, are characterized by intense competition.
CSI Singapore has tested MNPR-202 in preclinical cancer models with promising results. License, Development and Collaboration Agreements XOMA Ltd. To humanize our MNPR-101 antibody, we have taken a non-exclusive license to XOMA (US) LLC’s humanization technology and know-how.
We will jointly share ownership of the filed patent application on the us e of PCTA as a linker with Ac-225, which has shown that MNPR-101 has superior binding and yield with Ac-225 over the current industry-leading linker, DOTA. XOMA Ltd. To humanize our MNPR-101 antibody, we have taken a non-exclusive license to XOMA (US) LLC’s humanization technology and know-how.
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We currently have several compounds in development: 1) MNPR-101-Zr, a clinical stage uPAR-targeted radiodiagnostic imaging agent; 2) MNPR-101-RIT, a late preclinical stage radiotherapeutic for advanced cancers; 3) camsirubicin (generic name for MNPR-201, GPX-150; 5-imino-13-deoxydoxorubicin), a Phase 1b clinical stage novel analog of doxorubicin engineered specifically to retain anticancer activity while minimizing toxic effects on the heart; and 4) an early stage camsirubicin analog, MNPR-202, for various cancers.
Added
(“Monopar,” the “Company,” “we,” “us,” and “our” and similar terms mean Monopar Therapeutics Inc. and its subsidiaries except where the context otherwise requires) is a clinical-stage biopharma company developing an innovative treatment for Wilson disease and novel radiopharmaceuticals for oncology. Our Wilson disease product candidate is ALXN1840, a late-stage, investigational once-daily, oral medicine.
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Our Product Pipeline 7 Table of Contents Our Product Candidates MNPR-101 for Radiopharmaceutical Use Our MNPR-101 radiopharmaceutical program was developed by us for advanced cancers expressing urokinase plasminogen activator receptor ("uPAR"). This proprietary, novel first-in-class radiopharmaceutical program aims to identify and selectively kill the tumors expressing uPAR, which include a majority of triple-negative breast, colorectal, and pancreatic cancers.
Added
Our radiopharmaceutical program consists of MNPR-101, a proprietary humanized monoclonal antibody that is being developed across multiple product candidates, conjugated with different radioisotopes, for the treatment of advanced solid tumors expressing urokinase plasminogen activator receptor (“uPAR”).
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The program uses MNPR-101, our proprietary humanized monoclonal antibody targeting uPAR, as a means to accurately deliver radioisotope payloads to the tumors. We have demonstrated promising preclinical data to-date for our MNPR-101 radiopharmaceutical program.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeCompetition in the biopharmaceutical industry for technically proficient marketing, sales, and distribution personnel is intense and attracting and retaining such personnel may significantly increase our costs. There can be no assurance that we will be able to establish internal marketing, sales, or distribution capabilities or that these capabilities will be sufficient to meet our needs.
Biggest changeTo market any products directly, we would have to establish a marketing, sales, and distribution force that has technical expertise and could support a distribution capability. Competition in the biopharmaceutical industry for technically proficient marketing, sales, and distribution personnel is intense and attracting and retaining such personnel will significantly increase our costs.
If we or our licensees, development collaborators, or suppliers are unable to manufacture our products in sufficient quantities or at defined quality specifications, or are unable to obtain regulatory approvals for the manufacturing facility, we may be unable to develop and/or meet demand for our products and lose time to market and potential revenues.
If we or our licensees, development collaborators, or suppliers are unable to manufacture our products in sufficient quantities or at defined quality specifications, or are unable to obtain regulatory approvals for the manufacturing facility, we may be unable to develop and to meet the demand for our products and lose time to market and potential revenues.
Accordingly, we have not yet begun to build out any sales or marketing or distribution capabilities.
Accordingly, we have not yet begun to build out any sales, marketing or distribution capabilities.
If we are unable to establish, or contract for, effective sales and marketing and distribution capabilities, or if we are unable to enter into agreements with third parties to commercialize our product candidates on favorable terms or on any reasonable terms at all, we may not be able to effectively generate product revenues once our product candidates are approved for marketing.
If we are unable to establish, or contract for, effective sales, marketing and distribution capabilities, or if we are unable to enter into agreements with third parties to commercialize our product candidates on favorable terms or on any reasonable terms at all, we may not be able to effectively generate product revenues once our product candidates are approved for marketing.
If we are unable to successfully settle future claims on terms acceptable to us, we may be required to engage in or to continue costly, unpredictable and time-consuming litigation and may be prevented from, or experience substantial delays in, marketing our drug candidates.
If we are unable to successfully settle future claims on terms acceptable to us, we may be required to engage in or to continue costly, unpredictable and time-consuming litigation and may be prevented from, or experience substantial delays in, marketing our drug candidates.
Our largest stockholders have rights, subject to some conditions, to require us to file registration statements covering their shares or to include their shares in registration statements that we may file for ourselves or our stockholders. We have also registered shares of common stock that we have issued and may issue under our employee equity incentive plans.
Some of our largest stockholders have rights, subject to some conditions, to require us to file registration statements covering their shares or to include their shares in registration statements that we may file for ourselves or our stockholders. We have also registered shares of common stock that we have issued and may issue under our employee equity incentive plans.
Pursuant to the Certificate of Incorporation and indemnification agreement, each non-employee director and officer who is made a party to a legal proceeding because he or she is or was a non-employee director or officer, is indemnified by us from and against any and all liability, except that we may not indemnify a non-employee director or officer: (a) for any liability incurred in a proceeding in which such person is adjudged liable to Monopar or is subjected to injunctive relief in favor of Monopar; (b) for acts or omissions that involve intentional misconduct or a knowing violation of law, fraud or gross negligence; (c) for unlawful distributions; (d) for any transaction for which such non-employee director or officer received a personal benefit or as otherwise prohibited by or as may be disallowed under Delaware law; or (e) with respect to any dispute or proceeding between us and such non-employee director or officer unless such indemnification has been approved by a disinterested majority of the Board or by a majority in interest of disinterested stockholders.
Pursuant to the Certificate of Incorporation and indemnification agreement, each non-employee director and officer who is made a party to a legal proceeding because he or she is or was a non-employee director or officer, is indemnified by us from and against any and all liability, except that we may not indemnify a non-employee director or officer: (a) for any liability incurred in a proceeding in which such person is adjudged liable to us or is subjected to injunctive relief in favor of us; (b) for acts or omissions that involve intentional misconduct or a knowing violation of law, fraud or gross negligence; (c) for unlawful distributions; (d) for any transaction for which such non-employee director or officer received a personal benefit or as otherwise prohibited by or as may be disallowed under Delaware law; or (e) with respect to any dispute or proceeding between us and such non-employee director or officer unless such indemnification has been approved by a disinterested majority of the Board or by a majority in interest of disinterested stockholders.
If there is a resurgence of COVID-19 or any future pandemics arise, we may experience disruptions that could severely impact our business, preclinical studies and clinical trials, including: ● Delays in receiving approval from the FDA, the TGA in Australia and other foreign regulatory authorities to initiate our planned clinical trials; ● Delays or difficulties in enrolling and monitoring patients in our clinical trials; ● Delays or difficulties in clinical site initiation, including difficulties in recruiting clinical site investigators and clinical site staff; ● Delays in trial drug shipments due to vaccine shipments tying up available pharmaceutical product shipping lanes and increasing their cost; ● Diversion of healthcare resources away from the conduct of clinical trials, including the diversion of hospitals serving as our clinical trial sites and hospital staff supporting the conduct of our clinical trials; ● Risk that participants enrolled in our clinical trials will acquire diseases while the clinical trial is ongoing, which could impact the results of the clinical trial, including by increasing the number of observed adverse events; 26 Table of Contents ● Interruption of key clinical trial activities, such as clinical trial site data monitoring, due to limitations on travel imposed or recommended by federal or state governments, employers and others or interruption of clinical trial subject visits and study procedures, which may impact the integrity of subject data and clinical study endpoints; ● Interruption or delays in the operations of the FDA, the TGA, and other foreign regulatory agencies, which may impact approval timelines; ● Interruption of, or delays in receiving, supplies of our product candidates from our contract manufacturing organizations due to staffing or supply shortages, production slowdowns, global shipping delays or stoppages and disruptions in delivery systems; ● Limitation on employee resources that would otherwise be focused on the conduct of our preclinical studies and clinical trials, including because of sickness of our employees or their families or the desire of employees to avoid contact with large groups of people. ● Refusal of the FDA, the TGA, and other foreign regulatory authorities to accept data from clinical trials in affected geographies; and ● Impacts from prolonged remote work arrangements, such as increased cybersecurity risks.
If there is a resurgence of COVID-19 or any future pandemics arise, we may experience disruptions that could severely impact our business, preclinical studies and clinical trials, including: ● Delays in receiving approval from the FDA, the TGA in Australia and other foreign regulatory authorities to initiate our planned clinical trials; ● Delays or difficulties in enrolling and monitoring patients in our clinical trials; ● Delays or difficulties in clinical site initiation, including difficulties in recruiting clinical site investigators and clinical site staff; ● Delays in trial drug shipments due to vaccine shipments tying up available pharmaceutical product shipping lanes and increasing their cost; ● Diversion of healthcare resources away from the conduct of clinical trials, including the diversion of hospitals serving as our clinical trial sites and hospital staff supporting the conduct of our clinical trials; ● Risk that participants enrolled in our clinical trials will acquire diseases while the clinical trial is ongoing, which could impact the results of the clinical trial, including by increasing the number of observed adverse events; 23 Table of Contents ● Interruption of key clinical trial activities, such as clinical trial site data monitoring, due to limitations on travel imposed or recommended by federal or state governments, employers and others or interruption of clinical trial subject visits and study procedures, which may impact the integrity of subject data and clinical study endpoints; ● Interruption or delays in the operations of the FDA, the TGA, and other foreign regulatory agencies, which may impact approval timelines; ● Interruption of, or delays in receiving, supplies of our product candidates from our contract manufacturing organizations due to staffing or supply shortages, production slowdowns, global shipping delays or stoppages and disruptions in delivery systems; ● Limitation on employee resources that would otherwise be focused on the conduct of our preclinical studies and clinical trials, including because of sickness of our employees or their families or the desire of employees to avoid contact with large groups of people. ● Refusal of the FDA, the TGA, and other foreign regulatory authorities to accept data from clinical trials in affected geographies; and ● Impacts from prolonged remote work arrangements, such as increased cybersecurity risks.
Patient enrollment may also be affected by other factors, including: ● delays in U.S., Australian or other foreign regulatory approvals to start the clinical trial; ● coordination with any clinical research organizations to enroll and administer the clinical trials; ● coordination and recruitment of collaborators and investigators at individual sites; ● size of the patient population and the effectiveness of the process for identifying patients; ● design of the clinical trial protocol; ● eligibility and exclusion criteria; ● perceived therapeutic risks and benefits of the product candidates being studied; ● availability of competing commercially available therapies and other competing products’ clinical trials; ● time of year in which the trials are initiated or conducted; ● severity and prognosis of the diseases under investigation; ● ability to obtain and maintain subject consents; ● ability to enroll and treat patients in a timely manner; ● risk that enrolled subjects will drop out before completion of the trials; ● proximity and availability of clinical trial sites for prospective patients; ● ability to monitor subjects adequately during and after treatment; ● logistical challenges posed by the time-limited shelf-life of our current or future drug candidates; ● patient referral practices of physicians; and ● potential long-term effects of COVID-19, any resurgences thereof or any future pandemics. 32 Table of Contents Our inability to enroll a sufficient number of patients for clinical trials would result in significant delays and could require us to abandon one or more clinical trials altogether.
Patient enrollment may also be affected by other factors, including: ● delays in U.S., Australian or other foreign regulatory approvals to start the clinical trial; ● coordination with any clinical research organizations to enroll and administer the clinical trials; ● coordination and recruitment of collaborators and investigators at individual sites; ● size of the patient population and the effectiveness of the process for identifying patients; ● design of the clinical trial protocol; ● eligibility and exclusion criteria; ● perceived therapeutic risks and benefits of the product candidates being studied; ● availability of competing commercially available therapies and other competing products’ clinical trials; ● time of year in which the trials are initiated or conducted; ● severity and prognosis of the diseases under investigation; ● ability to obtain and maintain subject consents; ● ability to enroll and treat patients in a timely manner; ● risk that enrolled subjects will drop out before completion of the trials; ● proximity and availability of clinical trial sites for prospective patients; ● ability to monitor subjects adequately during and after treatment; ● logistical challenges posed by the time-limited shelf-life of our current or future drug candidates; ● patient referral practices of physicians; and ● potential long-term effects of COVID-19, any resurgences thereof or any future pandemics. 29 Table of Contents Our inability to enroll a sufficient number of patients for clinical trials would result in significant delays and could require us to abandon one or more clinical trials altogether.
The market price of our common stock is likely to remain highly volatile and may fluctuate substantially due to many factors, including: ● announcements concerning the progress and success of our clinical trials, our ability to obtain regulatory approval for and commercialize our product candidates, including any requests we receive from the FDA or TGA for additional studies or data that result in delays in obtaining regulatory approval or launching our product candidates, if approved; ● unstable market conditions in the pharmaceutical and biotechnology sectors or the economy as a whole; ● price and volume fluctuations in the overall stock market; ● the failure of our product candidates, if approved, to achieve anticipated commercial success; in the time projected by securities analysts and others; ● announcements of disruptions in supply and manufacturing of radioisotopes or raw materials required to manufacture radioisotopes, and any events that may disrupt the timely supply of radiopharmaceuticals to clinical sites; ● announcements of the clinical success, NDA approval or introduction of new products by us or our direct competitors; ● announcements of developments concerning product development results or intellectual property rights of others; ● litigation or public concern about the safety and/or efficacy of our potential or approved products; 55 Table of Contents ● actual fluctuations in our quarterly or annual operating results, and concerns by investors that such fluctuations may occur in the future and are indicative of internal problems; ● deviations in our operating results from the estimates of securities analysts or other analyst comments; ● additions or departures of key personnel; ● healthcare reform legislation, including measures directed at controlling the pricing of pharmaceutical products, and third-party coverage and reimbursement policies; ● announcements or publicity concerning current or future strategic collaborations; ● discussion of our Company, our stock price or our potential future market value by the financial and scientific press and online investor communities; and ● market responses to the fluctuating conditions of COVID-19 or any future pandemics or to the Russia-Ukraine war or Israel-Hamas war.
The market price of our common stock is likely to remain highly volatile and may fluctuate substantially due to many factors, including: ● announcements concerning the progress and success of our clinical trials, our ability to obtain regulatory approval for and commercialize our product candidates, including any requests we receive from the FDA or TGA for additional studies or data that result in delays in obtaining regulatory approval or launching our product candidates, if approved; ● unstable market conditions in the pharmaceutical and biotechnology sectors or the economy as a whole; ● price and volume fluctuations in the overall stock market; ● the failure of our product candidates, if approved, to achieve anticipated commercial success; in the time projected by securities analysts and others; ● announcements of disruptions in supply and manufacturing of ALXN1840, radioisotopes or raw materials required to manufacture radioisotopes, and any events that may disrupt the timely supply of radiopharmaceuticals to clinical sites; ● announcements of the clinical success, NDA approval or introduction of new products by us or our direct competitors; ● announcements of developments concerning product development results or intellectual property rights of others; ● litigation or public concern about the safety and/or efficacy of our potential or approved products; 52 Table of Contents ● actual fluctuations in our quarterly or annual operating results, and concerns by investors that such fluctuations may occur in the future and are indicative of internal problems; ● deviations in our operating results from the estimates of securities analysts or other analyst comments; ● additions or departures of key personnel; ● healthcare reform legislation, including measures directed at controlling the pricing of pharmaceutical products, and third-party coverage and reimbursement policies; ● announcements or publicity concerning current or future strategic collaborations; ● discussion of our Company, our stock price or our potential future market value by the financial and scientific press and online investor communities; and ● market responses to the fluctuating conditions of COVID-19 or any future pandemics or to the Russia-Ukraine war or Israel-Hamas war.
In order for us to commercialize any treatment for cancer or any other disease indication, we must obtain regulatory approvals of such treatment for that indication. Satisfying regulatory requirements is an expensive process that takes many years and involves compliance with requirements covering research and development, testing, manufacturing, quality control, labeling and promotion of drugs for human use.
In order for us to commercialize any treatment for Wilson disease, cancer or any other disease indication, we must obtain regulatory approvals of such treatment for that indication. Satisfying regulatory requirements is an expensive process that takes many years and involves compliance with requirements covering research and development, testing, manufacturing, quality control, labeling and promotion of drugs for human use.
These cost increases and delays may affect our clinical material manufacturing which will likely have an adverse effect on our financial condition. In addition, the effects of responses to inflationary conditions, such as significantly increased interest rates, on the economy and market conditions are difficult to predict.
These cost increases, tariffs, and delays may affect our clinical material manufacturing which will likely have an adverse effect on our financial condition. In addition, the effects of responses to inflationary conditions, such as significantly increased interest rates, on the economy and market conditions are difficult to predict.
Doing business internationally involves a number of risks, including but not limited to: ● multiple, conflicting and changing laws and regulations such as privacy regulations, tax laws, export and import restrictions, employment laws, regulatory requirements, and other governmental approvals, permits and licenses which can vary jurisdictions to jurisdiction with different degrees of review and enforcement; ● failure by us to obtain and maintain regulatory approvals for the use of our products in various countries; ● rejection or qualification of foreign clinical trial data by the competent authorities of other countries; ● additional potentially relevant third-party patent and other intellectual property rights that may be necessary to develop and commercialize our products and drug candidates; ● complexities and difficulties in obtaining, maintaining, enforcing and defending our patent and other intellectual property rights; ● difficulties in staffing and managing foreign operations by a small-scale organization; ● complexities associated with managing multiple payor reimbursement regimes, government payors or patient self-pay systems; ● limits, as a U.S.-based company, in our ability to penetrate international markets; ● financial risks, such as longer payment cycles, difficulty collecting accounts receivable, the impact of local and regional financial crises on demand and payment for our products and exposure to foreign currency exchange rate fluctuations; ● natural disasters, political and economic instability, including wars, terrorism and political unrest, outbreak of disease, boycotts, curtailment of trade and other business restrictions, implementation of tariffs; ● certain expenses including, among others, expenses for travel, translation and insurance; and ● regulatory and compliance risks that relate to anti-corruption compliance and record-keeping that may fall within the purview of the U.S.
Doing business internationally involves a number of risks, including but not limited to: ● multiple, conflicting and changing laws and regulations such as privacy regulations, tax laws, export and import restrictions, employment laws, regulatory requirements, and other governmental approvals, permits and licenses which can vary jurisdictions to jurisdiction with different degrees of review and enforcement; ● failure by us to obtain and maintain regulatory approvals for the use of our products in various countries; ● rejection or qualification of foreign clinical trial data by the competent authorities of other countries; ● additional potentially relevant third-party patent and other intellectual property rights that may be necessary to develop and commercialize our products and drug candidates; ● complexities and difficulties in obtaining, maintaining, enforcing and defending our patent and other intellectual property rights; ● difficulties in staffing and managing foreign operations by a small-scale organization; ● complexities associated with managing multiple payer reimbursement regimes, government payers or patient self-pay systems; ● limits, as a U.S.-based company, in our ability to penetrate international markets; ● financial risks, such as longer payment cycles, difficulty collecting accounts receivable, the impact of local and regional financial crises on demand and payment for our products and exposure to foreign currency exchange rate fluctuations; ● natural disasters, political and economic instability, including wars, terrorism and political unrest, outbreak of disease, boycotts, curtailment of trade and other business restrictions, implementation of tariffs; ● certain expenses including, among others, expenses for travel, translation and insurance; and ● regulatory and compliance risks that relate to anti-corruption compliance and record-keeping that may fall within the purview of the U.S.
Our contracted MNPR-101-Zr manufacturing plant as well as our raw material supplier are currently located in the U.S., but the Russia-Ukraine war and Israel-Hamas war may adversely affect the sourcing of radioisotopes and timely supply of MNPR-101-Zr to clinical sites.
Our contracted MNPR-101-Zr and MNPR-101-Lu manufacturing plant as well as our raw material supplier are currently located in the U.S., but the Russia-Ukraine war and Israel-Hamas war may adversely affect the sourcing of radioisotopes and timely supply of MNPR-101-Zr and MNPR-101-Lu to clinical sites.
To the extent that any of our product candidates are ultimately sold in a foreign country, we may be subject to similar foreign laws and regulations, which may include, for instance, applicable post-marketing requirements, including safety surveillance, anti-fraud and abuse laws, and implementation of corporate compliance programs and reporting of payments or transfers of value to healthcare professionals. 38 Table of Contents Negotiated prices for our products covered by a Part D prescription drug plan and other government programs will be lower than the prices we might otherwise obtain.
To the extent that any of our product candidates are ultimately sold in a foreign country, we may be subject to similar foreign laws and regulations, which may include, for instance, applicable post-marketing requirements, including safety surveillance, anti-fraud and abuse laws, and implementation of corporate compliance programs and reporting of payments or transfers of value to healthcare professionals. 35 Table of Contents Negotiated prices for our products covered by a Part D prescription drug plan and other government programs will be lower than the prices we might otherwise obtain.
Accordingly, although there can be no assurance that we will undertake or successfully complete any transactions of the nature described above, any transactions that we do complete may be subject to the foregoing or other risks, and could have a material adverse effect on our business, results of operations, financial condition and prospects. 47 Table of Contents Our business and operations are vulnerable to computer system failures, cyber-attacks or deficiencies in our cybersecurity, which could increase our expenses, divert the attention of our management and key personnel away from our business operations and adversely affect our results of operations.
Accordingly, although there can be no assurance that we will undertake or successfully complete any transactions of the nature described above, any transactions that we do complete may be subject to the foregoing or other risks, and could have a material adverse effect on our business, results of operations, financial condition and prospects. 44 Table of Contents Our business and operations are vulnerable to computer system failures, cyber-attacks or deficiencies in our cybersecurity, which could increase our expenses, divert the attention of our management and key personnel away from our business operations and adversely affect our results of operations.
Future legislation or executive or private sector actions related to healthcare reform could materially and adversely affect our business by reducing our ability to generate revenue at prices sufficient to reward for the risks and costs of pharmaceutical development, to raise capital, and to market our products. 50 Table of Contents There is no assurance that federal or state healthcare reform will not adversely affect our future business and financial results, and we cannot predict how future federal or state legislative, judicial or administrative changes relating to healthcare reform and third-party payers will affect the pharmaceutical industry in general and our business in particular.
Future legislation or executive or private sector actions related to healthcare reform could materially and adversely affect our business by reducing our ability to generate revenue at prices sufficient to reward for the risks and costs of pharmaceutical development, to raise capital, and to market our products. 47 Table of Contents There is no assurance that federal or state healthcare reform will not adversely affect our future business and financial results, and we cannot predict how future federal or state legislative, judicial or administrative changes relating to healthcare reform and third-party payers will affect the pharmaceutical industry in general and our business in particular.
The risk of a security breach or disruption, particularly through cyber-attacks or cyber intrusion, including by computer hackers, foreign governments, and cyber terrorists, has generally increased as the number, intensity and sophistication of attempted attacks and intrusions from around the world have increased.
The risk of a security breach or disruption, particularly through cyber-attacks or cyber-intrusions, including by computer hackers, foreign governments, and cyber terrorists, has generally increased as the number, intensity and sophistication of attempted attacks and intrusions from around the world have increased.
We have projected cash requirements for the near term based on a variety of assumptions, but some or all of such assumptions are likely to be incorrect and/or incomplete, possibly materially in an adverse direction.
We have projected cash requirements for the near term based on a variety of assumptions, but some or all such assumptions are likely to be incorrect and/or incomplete, possibly in a materially adverse direction.
Congress, the federal courts and the USPTO, as well as other jurisdictions around the world, the laws and regulations governing patents could change in unpredictable ways that would weaken our ability to obtain new patents or to enforce our existing patents and patents that we might obtain in the future. 41 Table of Contents Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
Congress, the federal courts and the USPTO, as well as other jurisdictions around the world, the laws and regulations governing patents could change in unpredictable ways that would weaken our ability to obtain new patents or to enforce our existing patents and patents that we might obtain in the future. 38 Table of Contents Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements.
The biotechnology and pharmaceutical industries are characterized by extensive and complex litigation regarding patents and other intellectual property rights. 42 Table of Contents We or any of our future licensors or strategic partners, may be party to, or be threatened with, adversarial proceedings or litigation regarding intellectual property rights with respect to our current or any potential future drug candidates and technologies, including derivation, reexamination, inter partes review, post-grant review or interference proceedings before the USPTO and similar proceedings in jurisdictions outside of the U.S. such as opposition proceedings.
The biotechnology and pharmaceutical industries are characterized by extensive and complex litigation regarding patents and other intellectual property rights. 39 Table of Contents We or any of our future licensors or strategic partners, may be party to, or be threatened with, adversarial proceedings or litigation regarding intellectual property rights with respect to our current or any potential future drug candidates and technologies, including derivation, reexamination, inter partes review, post-grant review or interference proceedings before the USPTO and similar proceedings in jurisdictions outside of the U.S. such as opposition proceedings.
The occurrence of any of the following risks could have a material adverse effect on our business, financial condition, results of operations and future prospects and prospective investors could lose all or part of their investment.
The occurrence of any of the following risks could have a material adverse effect on our business, financial condition, results of operations and future growth prospects and prospective investors could lose all or part of their investment.
As a result, if we earn net taxable income, our ability to use our pre-change net operating loss carry-forwards to offset U.S. federal taxable income will be subject to limitations, which could result in increased future tax liability to us had we not been subject to such limitations. 56 Table of Contents If securities or industry analysts do not publish research or publish inaccurate or unfavorable research about our business, our stock price and trading volume could decline.
As a result, if we earn net taxable income, our ability to use our pre-change net operating loss carry-forwards to offset U.S. federal taxable income will be subject to limitations, which could result in increased future tax liability to us had we not been subject to such limitations. 53 Table of Contents If securities or industry analysts do not publish research or publish inaccurate or unfavorable research about our business, our stock price and trading volume could decline.
Further, if this occurs, our competitors may take advantage of our investment in development and trials by referencing our clinical and preclinical data and launch their product earlier than might otherwise be the case, and our competitive position, business, financial condition, results of operations, and prospects would be materially harmed. 40 Table of Contents Intellectual property disputes could require us to spend time and money to address such disputes and could limit our intellectual property rights.
Further, if this occurs, our competitors may take advantage of our investment in development and trials by referencing our clinical and preclinical data and launch their product earlier than might otherwise be the case, and our competitive position, business, financial condition, results of operations, and prospects would be materially harmed. 37 Table of Contents Intellectual property disputes could require us to spend time and money to address such disputes and could limit our intellectual property rights.
Patent and other intellectual property rights also will not protect our drug candidates and technologies if competitors or third parties design around such drug candidates and technologies without legally infringing, misappropriating or violating our patent or other intellectual property rights. 43 Table of Contents The cost to us in defending or initiating any litigation or other proceedings relating to our patent or other intellectual property rights, even if resolved in our favor, could be substantial, and any litigation or other proceedings would divert our management’s attention and distract our personnel from their normal responsibilities.
Patent and other intellectual property rights also will not protect our drug candidates and technologies if competitors or third parties design around such drug candidates and technologies without legally infringing, misappropriating or violating our patent or other intellectual property rights. 40 Table of Contents The cost to us in defending or initiating any litigation or other proceedings relating to our patent or other intellectual property rights, even if resolved in our favor, could be substantial, and any litigation or other proceedings would divert our management’s attention and distract our personnel from their normal responsibilities.
Many of these companies, firms, and other institutions have greater financial resources than us and may be better able to withstand and respond to adverse market conditions within the biopharmaceutical industry, including without limitation the lengthy product development and regulatory approval processes for product candidates. 46 Table of Contents We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively.
Many of these companies, firms, and other institutions have greater financial resources than us and may be better able to withstand and respond to adverse market conditions within the biopharmaceutical industry, including without limitation the lengthy product development and regulatory approval processes for product candidates. 43 Table of Contents We face significant competition from other biotechnology and pharmaceutical companies, and our operating results will suffer if we fail to compete effectively.
The timing of our clinical trials depends on our ability to recruit patients to participate as well as to subsequently dose these patients and complete required follow-up periods. 31 Table of Contents In addition, we may experience enrollment delays related to increased or unforeseen regulatory, legal and logistical requirements and COVID-19-related or other future pandemic-related as well as issues related to currently ongoing or any future geopolitical risks at certain clinical trial sites.
The timing of our clinical trials depends on our ability to recruit patients to participate as well as to subsequently dose these patients and complete required follow-up periods. 28 Table of Contents In addition, we may experience enrollment delays related to increased or unforeseen regulatory, legal and logistical requirements and COVID-19-related or other future pandemic-related as well as issues related to currently ongoing or any future geopolitical risks at certain clinical trial sites.
These laws also specifically address data protection measures such data residency requirements, requirements for handling personal health records and data subject rights that detail specific rights Australian citizens have on the collection and use of their personal data. 48 Table of Contents In addition, California enacted the California Consumer Privacy Act (“CCPA”), which creates new individual privacy rights for California consumers (as defined in the law) and places increased privacy and security obligations on entities handling personal data of consumers or households.
These laws also specifically address data protection measures such data residency requirements, requirements for handling personal health records and data subject rights that detail specific rights Australian citizens have on the collection and use of their personal data. 45 Table of Contents In addition, California enacted the California Consumer Privacy Act (“CCPA”), which creates new individual privacy rights for California consumers (as defined in the law) and places increased privacy and security obligations on entities handling personal data of consumers or households.
Any success we may achieve at a given stage of our clinical trials does not guarantee that we will achieve success at any subsequent stage, including without limitation final FDA or other regulatory organizations’ approval. 29 Table of Contents We may encounter delays or rejections in the regulatory approval process because of additional government regulation resulting from future legislation or administrative action, or from changes in the policies of the FDA or other regulatory bodies during the period of product development, clinical trials, or regulatory review.
Any success we may achieve at a given stage of our clinical trials does not guarantee that we will achieve success at any subsequent stage, including without limitation final FDA or other regulatory organizations’ approval. 26 Table of Contents We may encounter delays or rejections in the regulatory approval process because of additional government regulation resulting from future legislation or administrative action, or from changes in the policies of the FDA or other regulatory bodies during the period of product development, clinical trials, or regulatory review.
If we fail to comply with these requirements, we would be subject to possible regulatory action and may be limited in the jurisdictions in which we are permitted to sell our products and will lose time to market and potential revenues. 33 Table of Contents It is uncertain whether product liability insurance will be adequate to address product liability claims, or that insurance against such claims will be affordable or available on acceptable terms in the future.
If we fail to comply with these requirements, we would be subject to possible regulatory action and may be limited in the jurisdictions in which we are permitted to sell our products and will lose time to market and potential revenues. 30 Table of Contents It is uncertain whether product liability insurance will be adequate to address product liability claims, or that insurance against such claims will be affordable or available on acceptable terms in the future.
To the extent that we enter into any marketing, sales, or distribution arrangements with third parties, our product revenues per unit sold are expected to be lower than if we marketed, sold, and distributed our products directly, and any revenues we receive will depend upon the efforts of such third parties. 36 Table of Contents If we are unable to establish such third-party marketing and sales relationships, or choose not to do so, we would have to establish in-house marketing and sales capabilities.
To the extent that we enter into any marketing, sales, or distribution arrangements with third parties, our product revenues per unit sold are expected to be lower than if we marketed, sold, and distributed our products directly, and any revenues we receive will depend upon the efforts of such third parties. 33 Table of Contents If we are unable to establish such third-party marketing and sales relationships, or choose not to do so, we would have to establish in-house marketing and sales capabilities.
In addition, the success of the Company's current and future radiopharmaceutical programs will depend on several factors, including the following: ● sourcing clinical and, if approved for commercialization, commercial supplies for the materials, such as radioisotopes, used to manufacture our product candidates; ● sourcing or establishing manufacturing capabilities to produce adequate amounts of our product candidates; ● securing reliable supply chain for our product candidates given that isotope half-life times are limited; ● utilizing imaging agents to visualize tumor uptake in advance of administering our therapeutic candidates, which may increase the risk of adverse side effects; 27 Table of Contents ● facilitating patient access to the limited number of facilities able to administer our product candidates; ● using medicines to manage adverse side effects of our drug candidates that may not adequately control the side effects or that may have detrimental impacts on the efficacy of the treatment; and ● establishing sales and marketing capabilities upon obtaining any regulatory approval to gain market acceptance of the Company's novel radiopharmaceutical imaging and therapeutics.
In addition, the success of our current and future radiopharmaceutical programs will depend on several factors, including the following: ● sourcing clinical and, if approved for commercialization, commercial supplies for the materials, such as radioisotopes, used to manufacture our product candidates; ● sourcing or establishing manufacturing capabilities to produce adequate amounts of our product candidates; ● securing a reliable supply chain for our product candidates given that isotope half-life times are limited; ● utilizing imaging agents to visualize tumor uptake in advance of administering our therapeutic candidates, which may increase the risk of adverse side effects; 24 Table of Contents ● facilitating patient access to the limited number of facilities able to administer our product candidates; ● using medicines to manage adverse side effects of our drug candidates that may not adequately control the side effects or that may have detrimental impacts on the efficacy of the treatment; and ● establishing sales and marketing capabilities upon obtaining any regulatory approval to gain market acceptance of our novel radiopharmaceutical imaging and therapeutics.
This could result in continuing uncertainty regarding compliance matters and higher costs necessitated by ongoing revisions to disclosure and governance practices. 45 Table of Contents Despite ongoing compliance training and periodic education, our employees and consultants may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements, which could result in delays or terminations of our development programs and adversely affect our business.
This could result in continuing uncertainty regarding compliance matters and higher costs necessitated by ongoing revisions to disclosure and governance practices. 42 Table of Contents Despite ongoing compliance training and periodic education, our employees and consultants may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements, which could result in delays or terminations of our development programs and adversely affect our business.
If we are not able to raise needed funding under acceptable terms or at all, then we will have to reduce expenses, including the possible options of curtailing operations, abandoning opportunities, licensing or selling off assets, reducing costs to a point where clinical development or other progress is impaired, or ceasing operations entirely. 25 Table of Contents Market variables, such as inflation of product costs, labor rates and fuel, freight and energy costs, as well as geopolitical events could likely cause us to suffer significant increases in our operating and administrative expenses.
If we are not able to raise needed funding under acceptable terms or at all, then we will have to reduce expenses, including the possible options of curtailing operations, abandoning opportunities, licensing or selling off assets, reducing costs to a point where clinical development or other progress is impaired, or ceasing operations entirely. 22 Table of Contents Market variables, such as inflation of product costs, labor rates and fuel, freight and energy costs, tariffs, as well as geopolitical events could likely cause us to suffer significant increases in our operating and administrative expenses.
A termination of the license agreement might force us to cease developing and/or selling MNPR-101-Zr or MNPR-101 RIT, if either gets to market. Data provided by collaborators and other parties upon which we rely have not been independently verified and could turn out to be inaccurate, misleading, or incomplete.
A termination of the license agreement might force us to cease developing and/or selling MNPR-101-Zr or MNPR-101-Lu, if either gets to market. Data provided by collaborators and other parties upon which we rely have not been independently verified and could turn out to be inaccurate, misleading, or incomplete.
Patients who are prescribed medications for the treatment of their conditions generally rely on third-party payors to reimburse all or part of the costs associated with their prescription drugs. Coverage and adequate reimbursement from government healthcare programs, such as Medicare and Medicaid, and private healthcare insurers are critical to new product acceptance.
Patients who are prescribed medications for the treatment of their conditions generally rely on third-party payers to reimburse all or part of the costs associated with their prescription drugs. Coverage and adequate reimbursement from government healthcare programs, such as Medicare and Medicaid, and private healthcare insurers are critical to new product acceptance.
Our lack of experience in these critical areas makes it difficult for a prospective investor to evaluate our abilities and increases the risk that we will fail to successfully execute our strategies. 44 Table of Contents Furthermore, if our business grows rapidly, our operational, managerial, legal, and financial resources will be strained.
Our lack of experience in these critical areas makes it difficult for a prospective investor to evaluate our abilities and increases the risk that we will fail to successfully execute our strategies. 41 Table of Contents Furthermore, if our business grows rapidly, our operational, managerial, legal, and financial resources will be strained.
As a result, government authorities and other third-party payors have attempted to control costs by limiting coverage and the amount of reimbursement for particular medications. Increasingly, third-party payors are requiring that drug companies provide them with predetermined discounts from list prices and are challenging the prices charged for medical products.
As a result, government authorities and other third-party payers have attempted to control costs by limiting coverage and the amount of reimbursement for particular medications. Increasingly, third-party payers are requiring that drug companies provide them with predetermined discounts from list prices and are challenging the prices charged for medical products.
In addition, there may be long-term effects from radiopharmaceutical treatment, including late radiation toxicity, with any of the Company's current or future product radiopharmaceutical candidates that it cannot predict at this time. It is difficult for us to predict the time and cost of the development of our product candidates.
In addition, there may be long-term effects from radiopharmaceutical treatment, including late radiation toxicity, with any of our current or future product radiopharmaceutical candidates that we cannot predict at this time. It is difficult for us to predict the time and cost of the development of our product candidates.
Additional federal, state and local laws and regulations affecting our operations may be adopted in the future. We may incur substantial costs to comply with, and substantial fines or penalties if we violate, any of these laws or regulations. 49 Table of Contents We have limited the liability of and indemnified our directors and officers.
Additional federal, state and local laws and regulations affecting our operations may be adopted in the future. We may incur substantial costs to comply with, and substantial fines or penalties if we violate, any of these laws or regulations. 46 Table of Contents We have limited the liability of and indemnified our directors and officers.
Perceptions of these challenges and risks in the market may adversely impact our stock price and our ability to successfully raise funding as we focus our preclinical and clinical efforts on our radiopharmaceutical program. 28 Table of Contents We do not have and may never have any approved products on the market.
Perceptions of these challenges and risks in the market may adversely impact our stock price and our ability to successfully raise funding as we focus our preclinical and clinical efforts on our radiopharmaceutical program. 25 Table of Contents We do not have and may never have any approved products on the market.
Our ability to commercialize any products successfully will depend, in part, on the extent to which coverage and adequate reimbursement for these products and related treatments will be available from third-party payors, such as government authorities, private healthcare insurers and health maintenance organizations.
Our ability to commercialize any products successfully will depend, in part, on the extent to which coverage and adequate reimbursement for these products and related treatments will be available from third-party payers, such as government authorities, private healthcare insurers and health maintenance organizations.
See “Cautionary Statement Concerning Forward-Looking Statements.” 52 Table of Contents Our present and potential future international operations may expose us to business, political, operational, and financial risks associated with doing business outside of the U.S. Our business is subject to risks associated with conducting business internationally.
See “Cautionary Statement Concerning Forward-Looking Statements.” 49 Table of Contents Our present and potential future international operations may expose us to business, political, operational, and financial risks associated with doing business outside of the U.S. Our business is subject to risks associated with conducting business internationally.
Also, future success of our drug candidates, if approved will depend on gaining and maintaining acceptance by physicians, patients, third-party payors and other members of the medical community as their being efficacious and cost-effective alternatives to competing products and treatments.
Also, future success of our drug candidates, if approved will depend on gaining and maintaining acceptance by physicians, patients, third-party payers and other members of the medical community as their being efficacious and cost-effective alternatives to competing products and treatments.
If we are able to raise financing, it may be on terms that are unfavorable to us and if we are unable to raise sufficient funds or find a suitable pharmaceutical partner, we may have to discontinue or delay clinical development of our current or future product candidates.
If we are able to raise financing when needed, it may be on terms that are unfavorable to us and if we are unable to raise sufficient funds or find a suitable pharmaceutical partner, we may have to discontinue or delay clinical development of our current or future product candidates.
These risks and uncertainties include without limitation the following: ● Patents that may be issued or licensed may be challenged, invalidated, or circumvented; or may not provide any competitive advantage for other reasons. ● Our licensors may terminate or breach our existing or future license agreements, thereby reducing or preventing our ability to exclude competition; termination of such license agreements may also subject us to risk of patent infringement of patents to which we no longer have a license. ● Our competitors, many of which have substantially greater resources than us and have made significant investments in competing technologies, may seek, or may already have obtained, patents that will limit, interfere with, or eliminate our ability to make, use, and sell our potential products either in the U.S. or in international markets. ● As a matter of public policy regarding worldwide health concerns, there may be significant pressure on the U.S. government and other international governmental bodies to limit the scope of domestic and international patent protection for cancer treatments that prove successful. ● Countries other than the U.S. may have less restrictive patent laws than those upheld by the U.S. courts; therefore, non-U.S. competitors could exploit these laws to create, develop, and market competing products.
See “Business - Intellectual Property Portfolio and Exclusivity.” These risks and uncertainties include without limitation the following: ● Patents that may be issued or licensed may be challenged, invalidated, or circumvented; or may not provide any competitive advantage for other reasons. ● Our licensors may terminate or breach our existing or future license agreements, thereby reducing or preventing our ability to exclude competition; termination of such license agreements may also subject us to risk of patent infringement of patents to which we no longer have a license. ● Our competitors, many of which have substantially greater resources than us and have made significant investments in competing technologies, may seek, or may already have obtained, patents that will limit, interfere with, or eliminate our ability to make, use, and sell our potential products either in the U.S. or in international markets. ● As a matter of public policy regarding worldwide health concerns, there may be significant pressure on the U.S. government and other international governmental bodies to limit the scope of domestic and international patent protection for cancer treatments that prove successful. ● Countries other than the U.S. may have less restrictive patent laws than those upheld by the U.S. courts; therefore, non-U.S. competitors could exploit these laws to create, develop, and market competing products.
If we, our contract research organizations ( “ CROs ” ) or our IT vendors experience security or data privacy breaches or other unauthorized or improper access to, use of, or destruction of personal data, we may face costs, significant liabilities, harm to our brand and business disruption.
If we, our contract research organizations ( “ CROs ” ) or our information technology ( “ IT ” ) vendors experience security or data privacy breaches or other unauthorized or improper access to, use of, or destruction of personal data, we may face costs, significant liabilities, harm to our brand and business disruption.
If any of our radiopharmaceutical program product candidates are approved, their commercial success will depend upon competitive products, public perception of radioisotopes and the degree of their market acceptance by physicians, patients, healthcare payors and others in the medical community.
If any of our radiopharmaceutical program product candidates are approved, their commercial success will depend upon competitive products, public perception of radioisotopes and the degree of their market acceptance by physicians, patients, healthcare payers and others in the medical community.
We could be forced to expend significant resources in response to a cyber security breach, including repairing system damage, increasing cyber security protection costs by deploying additional personnel and protection technologies, paying regulatory fines and resolving legal claims and regulatory actions, all of which would increase our expenses, divert the attention of our management and key personnel away from our business operations and adversely affect our results of operations.
We could be forced to expend significant resources in response to a cybersecurity breach, including repairing system damage, increasing cybersecurity protection costs by deploying additional personnel and protection technologies, paying regulatory fines and resolving legal claims and regulatory actions, all of which would increase our expenses, divert the attention of our management and key personnel away from our business operations and adversely affect our results of operations.
Third-party payors also may request additional clinical evidence beyond the data required to obtain marketing approval, requiring a company to conduct expensive pharmacoeconomic studies in order to demonstrate the medical necessity and cost-effectiveness of its products.
Third-party payers also may request additional clinical evidence beyond the data required to obtain marketing approval, requiring a company to conduct expensive pharmacoeconomic studies in order to demonstrate the medical necessity and cost-effectiveness of its products.
As a company we have limited experience in negotiating, establishing, and maintaining strategic relationships, conducting clinical trials, and managing the regulatory approval process, all of which will be necessary if we are to be successful.
As a company we have limited experience in negotiating, establishing, and maintaining strategic relationships, completing successful clinical trials, and managing the regulatory approval process, all of which will be necessary if we are to be successful.
Moreover, the targetable population for our MNPR-101 radiopharmaceutical program and camsirubicin may further be reduced if our estimates or addressable populations are erroneous or sub-populations of patients within the addressable populations do not benefit from our MNPR-101 radiopharmaceutical program or camsirubicin.
Moreover, the targetable population for our ALXN1840 and MNPR-101 radiopharmaceutical program may further be reduced if our estimates or addressable populations are erroneous or sub-populations of patients within the addressable populations do not benefit from our ALXN1840 and/or MNPR-101 radiopharmaceutical program.
There can be no assurances that we will be able to obtain such designations. 30 Table of Contents Even if we obtain orphan drug designation for a product candidate, we may not be able to maintain orphan drug exclusivity for that drug.
There can be no assurances that we will be able to obtain such designations. 27 Table of Contents Even if we obtain orphan drug designation for a product candidate, we may not be able to maintain orphan drug exclusivity for that drug.
Also, eligible or amenable patients may become increasingly difficult to identify and access due to many different factors, such as increasing competition in the radiopharmaceutical space.
Also, eligible or amenable patients may become increasingly difficult to identify and access due to many different factors, such as increasing competition in the Wilson disease and/or radiopharmaceutical space.
Our ability to generate revenue and achieve profitability will depend on, among other things, successful completion of the development of our product candidates; obtaining necessary regulatory approvals from the FDA and international regulatory agencies; establishing manufacturing/quality, sales, and marketing and distribution arrangements with third parties; obtaining adequate reimbursement by third-party payers; and raising sufficient funds to finance our activities.
Our ability to generate revenue and achieve profitability will depend on, among other things, successfully completing the development of our product candidates; obtaining necessary regulatory approvals from the FDA and international regulatory agencies; establishing manufacturing/quality, sales, and marketing and distribution arrangements with third parties; obtaining adequate reimbursement by third-party payers; and raising sufficient funds to finance our activities.
Our prospects must be considered in light of the uncertainties, risks, expenses, and difficulties frequently encountered by companies in their early clinical stages of operations.
Our prospects must be considered in light of the uncertainties, risks, expenses, and difficulties frequently encountered by biopharma companies in their early and late clinical stages of operations.
In addition, because we are incorporated in Delaware, we are governed by the provisions of Section 203 of the Delaware General Corporation Law, which generally prohibits a Delaware corporation from engaging in any of a broad range of business combinations with any “interested” stockholder for a period of three years following the date on which the stockholder became an “interested” stockholder. 57 Table of Contents
In addition, because we are incorporated in Delaware, we are governed by the provisions of Section 203 of the Delaware General Corporation Law, which generally prohibits a Delaware corporation from engaging in any of a broad range of business combinations with any “interested” stockholder for a period of three years following the date on which the stockholder became an “interested” stockholder. 54 Table of Contents Item 1C.
Risks Associated with our Common Stock Existing and new investors will experience dilution as a result of future sales or issuances of our common stock and future option exercises under our 2016 Stock Incentive Plan and any amendments to the plan.
Risks Associated with our Common Stock Existing and new investors will experience dilution as a result of future sales or issuances of our common stock, future exercises of the pre-funded warrants, and future option exercises under our 2016 Stock Incentive Plan and any amendments to the plan.
In the wake of the COVID-19 pandemic, the Russia-Ukraine war, the Israel-Hamas war and other geopolitical factors, economic conditions have become strained, with inflation and supply chain challenges impacting businesses worldwide. These conditions affect fuel costs and shipping, resulting in higher costs and delays for various types of supplies.
In the wake of the COVID-19 pandemic, the Russia-Ukraine war, the Israel-Hamas war and other geopolitical factors, economic conditions have become strained, with inflation and supply chain challenges impacting businesses worldwide along with potential new tariffs. These conditions affect fuel costs and shipping, resulting in higher costs and delays for various types of supplies.
Any of these factors may prevent the Company from completing our preclinical and clinical trials that we may initiate, or from commercializing any product candidates we may develop on a timely or profitable basis, if at all.
Any of these factors may prevent us from completing our preclinical and clinical trials that we may initiate, or from commercializing any product candidates we may develop on a timely or profitable basis, if at all.
Before we can begin to commercially manufacture camsirubicin, MNPR-101-Zr, MNPR-101 RIT, MNPR-202 or any other product candidate, we must obtain regulatory approval of the manufacturing facility and process. Manufacturing of drugs for clinical and commercial purposes must comply with current Good Manufacturing Practices requirements, commonly known as “cGMP.” The cGMP requirements govern quality control and documentation policies and procedures.
Before we can begin to commercially manufacture ALXN1840, MNPR-101-Zr, MNPR-101-Lu, or any other product candidate, we must obtain regulatory approval of the manufacturing facility and process. Manufacturing of drugs for clinical and commercial purposes must comply with current Good Manufacturing Practices requirements, commonly known as “cGMP.” The cGMP requirements govern quality control and documentation policies and procedures.
Commercial third-party payors often rely upon Medicare coverage policy and payment limitations in setting their reimbursement rates, but also have their own methods and approval process apart from Medicare determinations. Therefore, coverage and reimbursement for pharmaceutical products in the U.S. can differ significantly from payor to payor.
Commercial third-party payers often rely upon Medicare coverage policy and payment limitations in setting their reimbursement rates, but also have their own methods and approval process apart from Medicare determinations. Therefore, coverage and reimbursement for pharmaceutical products in the U.S. can differ significantly from payer to payer.
Actual events or results may differ materially from those discussed in the forward-looking statements as a result of various factors, including the risk factors set forth under this “Risk Factors” section in this Annual Report on Form 10-K. The financial and operational projections that we may make from time to time are subject to inherent risks.
Actual events or results may differ materially from those discussed in the forward-looking statements as a result of various factors, including the risk factors set forth under this “ Item 1A -Risk Factors ” section in this Annual Report on Form 10-K. The financial and operational projections that we may make from time to time are subject to inherent risks.
We will need to raise additional capital in the future; the amount of additional capital needed will vary as a result of a number of factors, including without limitation the following: ● receiving less funding than we require; ● higher than expected costs to manufacture and ship our active pharmaceutical ingredient, radioisotopes, and our product candidates; ● higher than expected costs for preclinical testing; ● the cost and availability of radioisotopes such as Ac-225 or Zr-89, or any other medical isotope we may incorporate into our product candidates; ● an increase in the number, size, duration, and/or complexity of our clinical trials; ● slower than expected progress in developing our MNPR-101 radiopharmaceutical program, camsirubicin, and MNPR-202 or other product candidates, including without limitation, additional costs caused by program delays; ● higher than expected costs associated with attempting to obtain regulatory approvals, including without limitation additional costs caused by additional regulatory requirements or larger clinical trial requirements; ● higher than expected personnel, consulting or other costs, such as adding personnel or industry expert consultants or pursuing the licensing/acquisition of additional assets; and ● higher than expected costs to protect our intellectual property portfolio or otherwise pursue our intellectual property strategy.
We will need to raise additional capital in the future; the amount of additional capital needed will vary based on a number of factors, including without limitation the following: ● receiving less funding than we require; ● higher than expected costs to manufacture and ship our active pharmaceutical ingredient, radioisotopes, and our product candidates; ● higher than expected costs for preclinical testing; ● the cost and availability of radioisotopes such as Ac-225, Lutetium-177 or Zr-89, or any other medical isotope we may incorporate into our product candidates; ● an increase in the number, size, duration, and/or complexity of our clinical trials; ● slower than expected progress in developing our MNPR-101 radiopharmaceutical program, other product candidates, including without limitation, additional costs caused by program delays; ● higher than expected costs associated with attempting to obtain regulatory approvals, especially as they relate to ALXN1840, including without limitation additional costs caused by additional regulatory requirements or larger clinical trial requirements; ● higher than expected personnel, consulting or other costs, such as adding personnel or industry expert consultants or pursuing the licensing/acquisition of additional assets; and ● higher than expected costs to protect our intellectual property portfolio or otherwise pursue our intellectual property strategy.
Although our directors and officers are accountable to us and must exercise good faith, good business judgement, and integrity in handling our affairs, our Second Amended and Restated Certificate of Incorporation (the “Certificate of Incorporation”) and indemnification agreements executed by all of our non-employee directors and officers provides that our non-employee directors and officers will be indemnified to the fullest extent permitted under Delaware law.
Although our directors and officers are accountable to us and must exercise good faith, good business judgement, and integrity in handling our affairs, our directors and officers liability insurance policy, our Second Amended and Restated Certificate of Incorporation (the “Certificate of Incorporation”) and indemnification agreements executed by all of our non-employee directors and officers provide that our non-employee directors and officers will be indemnified to the fullest extent permitted under Delaware law.
Completion of our clinical trials and commercialization of our product candidates require access to, or development of, facilities to manufacture a sufficient supply of our product candidates. We will utilize third parties to manufacture our MNPR-101 radiopharma program and camsirubicin. We currently have manufacturing arrangements for MNPR-101-Zr and camsirubicin for clinical use.
Completion of our clinical trials and commercialization of our product candidates require access to, or development of, facilities to manufacture a sufficient supply of our product candidates. We will utilize third parties to manufacture our MNPR-101 radiopharmaceutical program. We currently have manufacturing arrangements for MNPR-101-Zr and MNPR-101-Lu for clinical use.
While we believe we have significant competitive advantages with our expertise in small molecules and biologics, and rare disease clinical development, along with a strong intellectual property portfolio, we currently face and will continue to face competition for our drug development programs from companies that are developing doxorubicin analogs/replacements, or are targeting uPAR.
While we believe we have significant competitive advantages with our expertise in small molecules and biologics, and rare disease clinical development, along with a strong intellectual property portfolio, we currently face and will continue to face competition for our drug development programs from companies that are developing Wilson disease drugs or are targeting uPAR.
In some countries, the legal compliance with pharmaceutical patents, patent applications and other intellectual property regulations is very weak or actively evaded in some cases with government aid. In addition, the U.S.
In some countries, the legal compliance with pharmaceutical patents, patent applications and other intellectual property regulations is very weak or actively evaded in some cases with government aid.
To date, we have funded our operations through net proceeds from the initial public offering of our common stock, net proceeds from sales of our common stock through at-the-market sales programs, private placements of our preferred and common stock, and the net receipt of funds related to our acquisition of camsirubicin and related assets.
To date, we have funded our operations through net proceeds from the initial public offering of our common stock, net proceeds from sales of our common stock through at-the-market sales programs, public offerings, private placements of our preferred and common stock, private placement of pre-funded warrants, and the net receipt of funds related to our acquisition of camsirubicin and related assets.
As such, it is difficult to accurately predict the developmental challenges that the Company may incur for its product candidates as they proceed through product discovery or identification, preclinical studies and clinical trials, and, if approved, commercialization.
As such, it is difficult to accurately predict the developmental challenges that we may incur for our product candidates as they proceed through product discovery or identification, preclinical studies and clinical trials, and, if approved, commercialization.
Patent and Trademark Office (“USPTO”) and patent offices in other jurisdictions have often required that patent applications concerning pharmaceutical and/or biotechnology-related inventions be limited or narrowed substantially to cover only the specific innovations exemplified in the patent application, thereby limiting their scope of protection against competitive challenges.
In addition, the USPTO and patent offices in other jurisdictions have often required that patent applications concerning pharmaceutical and/or biotechnology-related inventions be limited or narrowed substantially to cover only the specific innovations exemplified in the patent application, thereby limiting their scope of protection against competitive challenges.
Any future decision by the FDA and TGA will be driven largely by the data generated from our currently ongoing or any future planned trials.
Any future decision by the FDA and TGA will be driven largely by the data generated from prior preclinical and clinical trials, our currently ongoing, or any future planned trials.
Starr, our Executive Chairman of the Board of Directors (referred to as the “Board”), and Chandler D. Robinson, our President and CEO, could materially disrupt our business and materially delay or prevent the successful product development and commercialization of our product candidates. We have an employment agreement with Dr.
Starr, our Executive Chairman of the Board of Directors (referred to as the “Board”), and Chandler D. Robinson, our Chief Executive Officer and Board member, could materially disrupt our business and materially delay or prevent the successful product development and commercialization of our product candidates. We have an employment agreement with Dr.
Such delays will adversely impact our business and financial condition. 51 Table of Contents Effective collaboration with the FDA for the approval of drug candidates is a highly demanding process which can result in increased time and expense to gain approvals. Our Company has in-house expertise and experience in the management of drug approvals.
Such delays will adversely impact our business and financial condition. 48 Table of Contents Effective collaboration with the FDA for the approval of drug candidates is a highly demanding process which can result in increased time and expense to gain approvals. We have in-house expertise and experience in the management of drug approvals.
Qualified consultants and drug research organizations are also available to aid in our drug approval process; however, there is a meaningful risk that discussions and interactions inherent in the drug approval process and future developments or new improvements will result in delays, added expenses and new scientific/medical requirements which will cause adverse financial results and will likely impact the price of the Company’s stock.
Qualified consultants and drug research organizations are also available to aid in our drug approval process; however, there is a meaningful risk that discussions and interactions inherent in the drug approval process and future developments or new improvements will result in delays, added expenses and new scientific/medical requirements which will cause adverse financial results and will likely impact the price of our common stock which could impact our ability to raise capital when needed.
These estimates or reports may prove to be incorrect, and new studies may demonstrate or suggest a lower estimated incidence or prevalence of patients who might be eligible or amenable to MNPR-101-Zr, MNPR-101-RIT, and camsirubicin.
These estimates or reports may prove to be incorrect, and new studies may demonstrate or suggest a lower estimated incidence or prevalence of patients who might be eligible or amenable to ALXN1840 or to MNPR-101-Zr, MNPR-101-Lu, or MNPR-101-Ac.
Even if we complete the clinical trials we discussed with the FD A or TGA, there i s no guarantee that at the time of submission the FD A or TGA will accept our new drug application ( “ NDA ” ) or biologics license application ("BLA") based on the trials discussed.
Even if we complete the clinical trials we discussed with the FD A or TGA, there i s no guarantee that at the time of submission the FD A or TGA will accept our NDA or BLA based on the trials discussed.
We, or any future collaborators, may not be able to obtain and maintain orphan drug exclusivity for our product candidates in the U.S. and Europe. Camsirubicin has been granted orphan drug designation for the treatment of soft tissue sarcoma in the U.S. and in the EU.
We, or any future collaborators, may not be able to obtain and maintain orphan drug exclusivity for our product candidates in the U.S. and Europe. ALXN1840 has been granted orphan drug designation for the treatment of Wilson disease in the U.S. and in the EU.
The amount of future losses and when, if ever, we will become profitable are uncertain. We do not have any products that have generated revenues from commercial sales, and do not expect to generate revenues from the commercial sale of products in the near future, if ever.
The amount of future losses and when, if ever, we will become profitable are uncertain. To date, we do not have any products that have generated revenues from commercial sales.
The biopharmaceutical industry is subject to rapid technological change. We have many potential competitors, including major drug and chemical companies, specialized biopharmaceutical firms, universities and other research institutions. These companies, firms, and other institutions may develop products that are more effective than our product candidates or that would make our product candidates less competitive or obsolete.
We have many potential competitors, including major drug and chemical companies, specialized biopharmaceutical firms, universities and other research institutions. These companies, firms, and other institutions may develop products that are more effective than our product candidates or that would make our product candidates less competitive or obsolete.

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Item 2. Properties

Properties — owned and leased real estate

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Biggest changeItem 2. Properties While we are currently on a month-to-month lease for our executive headquarters at 1000 Skokie Blvd in the Village of Wilmette, Illinois for our corporate offices, we may enter into a longer-term lease in the next twelve months.
Biggest changeItem 2. Properties We recently entered into a 36-month lease beginning April 1, 2025 for our executive headquarters at 1000 Skokie Blvd in the Village of Wilmette, Illinois at a monthly rate of $3,580/month. The Company is also currently leasing additional office space in the same building on a month-to-month basis for $2,379 per month.
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We recently entered into a lease for a small wet laboratory space and certain equipment at the Helix 51 Bioscience Incubator at The Rosalind Franklin University of Medicine and Science in North Chicago, Illinois at a rate of $1,000/month, which is cancellable after 6 months with 30 days advance written notice.

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeRegistration Rights We are subject to an agreement with TacticGem, LLC (“TacticGem”), our largest stockholder, which obligates us to file a Form S-3 or other appropriate form of registration statement covering the resale of any of our common stock by TacticGem, or its members Gem Pharmaceuticals, LLC, or Tactic Pharma, LLC, upon direction by TacticGem.
Biggest changeRegistration Rights Following the dissolution of TacticGem, each of Tactic Pharma and Gem retained the right to obligate us to file a Form S-3 or other appropriate form of registration statement covering the resale of any of our common stock by either of them.
Item 5. Market for Registrant ’ s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Market Information Our common stock is listed under the symbol “MNPR” on the Nasdaq Capital Market. Holders As of March 8, 2024, there were 17,454,925 shares of our common stock outstanding held by 30 holders of record and approximately 3,200 beneficial stockholders.
Item 5. Market for Registrant ’ s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Market Information Our common stock is listed under the symbol “MNPR” on the Nasdaq Capital Market.
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Through the date hereof, TacticGem has not required us to file such a resale registration statement, although there can be no assurance we will not be required to do so in the future. Recent Sales of Unregistered Securities. There were no securities issuances that were not registered under the Securities Act during the reporting period.
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Holders As of March 14, 2 025, there were 6,112,593 s hares of our common stock outstanding held by 31 holders of record and approximately 3,300 beneficial stockholders.
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However, in connection with the dissolution of TacticGem, Tactic Pharma and Gem agreed with each other that neither could exercise such rights without the consent of the other prior to December 31, 2025. Recent Sales of Unregistered Securities.
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As previously disclosed, during the fourth quarter of 2024, we issued 387,329 initial shares to Alexion in connection with the acquisition of the ALXN1840 license. Pursuant to related anti-dilution rights, we subsequently issued an additional 157,188 shares to Alexion.
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Also as previously disclosed, during the further quarter of 2024, we issued pre-funded warrants to purchase 882,761 shares at a purchase price of $23.789 per pre-funded warrant to an institutional investor in a private placement.
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The foregoing securities were sold without registration under Securities in reliance on the exemptions provided by Section 4(a)(2) of the Securities Act as transactions not involving public offerings. There were no other securities issuances that were not registered under the Securities Act during the reporting period.

Item 7. Management's Discussion & Analysis

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

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Biggest changeOur future capital requirements will depend on many factors, including: ● the progress of clinical development and regulatory interactions and approvals of our MNPR-101 radiopharma program; ● the progress of clinical development and regulatory interactions and approvals of camsirubicin; ● the costs, timing and outcomes of seeking, obtaining, and maintaining FDA, TGA and other international regulatory approvals; ● the progress of preclinical and potentially clinical development of MNPR-202 (and related analogs); ● the number and characteristics of other drug product candidates that we may license, acquire, invent or otherwise pursue; ● the scope, progress, timing, cost and results of research, preclinical development and clinical trials and regulatory requirements for future drug product candidates; 66 Table of Contents ● the costs associated with manufacturing/quality requirements, establishing a reliable supply chain, and establishing or contracting for sales, marketing and distribution capabilities; ● our ability to maintain, expand and defend the scope of our intellectual property portfolio, including the amount and timing of any payments we may be required to make in connection with the licensing, filing, defense and enforcement of any patents or other intellectual property rights; ● our need and ability to hire or contract for additional management, administrative, scientific, medical, sales and marketing, and manufacturing/quality and other specialized personnel or external expertise; ● the effect and timing of entry of competing products or new therapies that may limit market penetration or prevent the introduction of our drug product candidates or reduce the commercial potential of our product portfolio; ● our need to implement additional required internal management, operational, record keeping and other systems and infrastructure; and ● the economic and other terms, timing and success of our existing collaboration and licensing arrangements and any collaboration, licensing or other arrangements into which we may enter in the future, including the timing of receipt of or payment to or from others of any license, milestone or royalty payments under these arrangements.
Biggest changeOur future capital requirements will depend on many factors, including: ● the development program for ALXN1840 in Wilson disease; ● the clinical development progress of MNPR-101-Zr in imaging advanced cancers; ● the clinical development progress of MNPR-101-Lu as a therapeutic agent in advanced cancers; ● the progress of preclinical and clinical development of MNPR-101-Ac; ● the progress of preclinical activities towards identifying novel targets and candidates to complement our radiopharmaceutical and rare disease programs; ● the number and characteristics of other drug product candidates that we may invent, license, acquire, or otherwise pursue; ● the costs, timing and outcomes of seeking, obtaining, and maintaining FDA, TGA and other international regulatory approvals; ● the scope, progress, timing, cost and results of research, preclinical development and clinical trials and regulatory requirements for future drug product candidates; 62 Table of Contents ● the costs associated with establishing or contracting for manufacturing/quality requirements and establishing or contracting for sales, marketing and distribution capabilities; ● our ability and related costs to maintain, expand and defend the scope of our intellectual property portfolio, including the amount and timing of any payments we may be required to make in connection with the licensing, filing, defense and enforcement of any patents or other intellectual property rights; ● our need and ability to hire or contract additional management, administrative, scientific, regulatory, medical, sales and marketing, manufacturing/quality and other specialized personnel or external expertise; ● the effect and timing of entry of competing products and/or new therapies that may limit market penetration or prevent the introduction of our drug product candidates or reduce the commercial potential of our product portfolio; ● our need to implement additional required internal management, operational, record keeping and other systems and infrastructure; and ● the economic and other terms, timing and success of our existing collaboration and licensing arrangements and any collaboration, licensing or other arrangements into which we may enter in the future, including the timing of receipt of or payment to or from others of any license, milestone or royalty payments under these arrangements.
The fair value method requires us to estimate the fair value of stock-based payment awards on the date of grant using an option pricing model or the closing stock price on the date of grant in the case of RSUs.
The fair value method requires us to estimate the fair value of stock-based payment awards on the date of grant using an option pricing model or, in the case of RSUs, the closing stock price on the date of grant.
In aggregate, companies they co-founded have achieved four drug approvals and three diagnostic medical imaging device approvals in the U.S. and the EU, successfully sold an asset developed by management which subsequently had a positive Phase 3 clinical trial, sold two oncology-focused diagnostic imaging businesses to Fortune Global 1000 firms, and completed the clinical and commercial development and ultimately the sale of a commercial biopharmaceutical company for over $800 million in cash.
In aggregate, companies they co-founded have achieved four drug approvals and three diagnostic medical imaging device approvals in the U.S. and the EU, successfully sold an asset developed by management which subsequently had a positive Phase 3 clinical trial, sold two oncology-focused diagnostic imaging businesses to Fortune Global 1000 firms, and completed the clinical and commercial development and ultimately the sale of a commercial biopharmaceutical company for $800 million in cash.
If we obtain regulatory approval of any of our current or future drug product candidates, we will need substantial additional funding for commercialization requirements and our continuing drug product development operations. As a company, we have not completed development through marketing approvals of any therapeutic products.
If we obtain regulatory approval of any of our current or future drug product candidates, we will need substantial additional funding for precommercial and commercialization requirements and our continuing drug product development operations. As a company, we have not completed development through marketing approvals of any therapeutic products.
We intend to continue evaluating drug product candidates for the purpose of growing our pipeline. Identifying and securing high-quality compounds usually takes time and related expenses; however, our spending could be significantly accelerated in 2024 and onward if additional drug product candidates are acquired and enter clinical development.
We intend to continue evaluating drug product candidates for the purpose of growing our pipeline. Identifying and securing high-quality compounds usually takes time and related expenses. Our spending could be significantly accelerated in the future if additional drug product candidates are acquired and enter clinical development.
To date, we have engaged in acquiring or in-licensing drug product candidates, entering into collaboration agreements for testing and clinical development of our drug product candidates and providing the infrastructure to support the clinical development of our drug product candidates.
To date, we have engaged in acquiring or in-licensing drug product candidates, and in entering into collaboration agreements for the preclinical testing and clinical development of our drug product candidates along with providing the infrastructure to support the clinical development of our drug product candidates.
We do not anticipate commercial revenues from operations until we complete testing and development of one of our drug product candidates and obtain marketing approval or we sell, enter into a collaborative marketing arrangement, or out-license one of our drug product candidates to another party. See “Liquidity and Capital Resources”.
We do not anticipate revenues from operations until we complete testing and development of one of our drug product candidates and obtain marketing approval, or until we sell, enter into a collaborative marketing arrangement, or out-license one of our drug product candidates to another party.
Our primary funding source over the past three years was sales of shares of our common stock under at-the-market sales programs through Capital on Demand™ Sales Agreements with JonesTrading Institutional Services LLC (“Jones Trading”).
Prior to the fourth quarter of 2024, our primary funding source over the past three years was sales of shares of our common stock under at-the-market sales programs. At-the-market sales were through the Capital on Demand™ Sales Agreements with JonesTrading Institutional Services LLC (“Jones Trading”).
Cash Flow Provided by Financing Activities The increase in cash flow provided by financing activities during the year ended December 31, 2023 , compared to the year ended December 31, 2022 , of $1,994,000 was primarily due to higher net proceeds from sales of our common stock through an at-the-market sales program.
Cash Flow Provided by Financing Activities The increase in cash flow provided by financing activities during the year ended December 31, 2024 , compared to the year ended December 31, 2023 , of $57,265,000 was primarily due to higher net proceeds from sales of our common stock through an at-the-market sales program and the October 2024 and December 2024 financing rounds.
Recently Issued and Adopted Accounting Pronouncements During the year ended December 31, 2023, there were two recently issued accounting pronouncements that are described in more detail in Note 2 of our consolidated financial statements included elsewhere in this Annual Report on Form 10-K.
See “Liquidity and Capital Resources.” Recently Issued and Adopted Accounting Pronouncements During the year ended December 31, 2024, there were two recently issued accounting pronouncements applicable to us that are described in more detail in Note 2 of our consolidated financial statements included elsewhere in this Annual Report on Form 10-K.
Treasury Bills. 64 Table of Contents Cash Flows The following table provides information regarding our cash flows for the years ended December 31, 2023, and 2022.
Treasury securities. 60 Table of Contents Cash Flows The following table provides information regarding our cash flows for the years ended December 31, 2024 and 2023.
We anticipate that the funds available as of March 8, 2024, will fund our obligations at least through June 30, 2025. We have based this estimate on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently expect.
We anticipate that the f unds available as of March 14, 2025, will fund our obligations at least through December 31, 2026. We ha ve based this estimate on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently expect.
Our senior executive team has relevant experience in biopharmaceutical in-licensing and acquisitions as well as developing product candidates through approval and commercialization.
Our senior executive team has relevant experience in biopharmaceutical in-licensing and acquisitions as well as developing product candidates through approval and commercialization. In aggregate, our team has co-founded BioMarin Pharmaceutical (Nasdaq: BMRN), Sensant Corp.
We have not paid dividends and do not anticipate paying a cash dividend in future vesting periods and, accordingly, use an expected dividend yield of zero. The risk-free interest rate is based on the rate of U.S.
Forfeitures only include actual forfeitures to-date as the Company accounts for forfeitures as they occur due to a limited history of forfeitures. We have not paid dividends and do not anticipate paying a cash dividend in future vesting periods and, accordingly, use an expected dividend yield of zero. The risk-free interest rate is based on the rate of U.S.
You should read the “Risk Factors” section of this Annual Report on Form 10-K, Item 1A, for a discussion of important factors that could cause actual results to differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis. 59 Table of Contents Overview We are a clinical stage biopharmaceutical company focused on developing innovative treatments for cancer patients.
You should read the “Item 1A-Risk Factors” section of this Annual Report on Form 10-K for a discussion of important factors that could cause actual results to differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.
Cash Flow Used in Investing Activities The increase to cash flow provided by investing activities during the year ended December 31, 2023 , compared to cash used in investing activities during the year ended December 31, 2022 , of $9,847,000 was related to the fact that our investment in U.S.
Cash Flow (Used in) Provided By Investing Activities The increase to cash flow used in investing activities during the year ended December 31, 2024 , compared to cash provided by investing activities during the year ended December 31, 2023, of $19,266,000 was related to the purchase of investments in U.S.
We anticipate that our expenses will increase substantially as we: ● continue the clinical and preclinical activities of MNPR-101-Zr and MNPR-101-RIT to image and treat cancer; ● advance the clinical development for camsirubicin; ● continue the preclinical activities, and potentially later-on enter clinical development, of MNPR-202 (and related analogs) for various cancer indications; 65 Table of Contents ● acquire and/or license additional pipeline drug product candidates and pursue the future preclinical and/or clinical development of such drug product candidates; ● seek regulatory approvals for any of our current and future drug product candidates that successfully complete registration clinical trials; ● establish or purchase the services of a sales, marketing and distribution infrastructure to commercialize any products for which we obtain marketing approval; ● develop or contract for manufacturing/quality capabilities or establish a reliable, high quality supply chain sufficient to support our clinical requirements and to provide sufficient capacity to launch and supply the market for any product for which we obtain marketing approval; and ● add or contract for required operational, financial and management information systems and capabilities and other specialized expert personnel to support our drug product candidate development and planned commercialization efforts.
We anticipate that our expenses will increase substantially as we: ● develop our ALXN1840 investigational drug candidate as a treatment for Wilson disease; ● progress our MNPR-101-Zr imaging and dosimetry clinical trial in advanced cancer patients; ● progress our MNPR-101-Lu therapeutic clinical trial in advanced cancer patients; ● continue the preclinical activities and potentially advance MNPR-101-Ac into the clinic as a therapeutic in advanced cancer patients; ● support intellectual property initiatives for our Wilson disease and radiopharmaceutical programs; ● identify and potentially invent or license novel targets and drug candidates complementing our radiopharmaceutical and rare disease programs, and pursue the future preclinical and clinical development and regulatory requirements of such drug product candidates; 61 Table of Contents ● seek regulatory approvals for any of our current and future drug product candidates that successfully complete registration clinical trials; ● establish or purchase the services of a sales, marketing and distribution infrastructure to commercialize any products for which we obtain marketing approval; ● develop or contract for manufacturing/quality capabilities or establish a reliable, high quality supply chain sufficient to support our clinical requirements and to provide sufficient capacity to launch and supply the market for any product for which we obtain marketing approval; and ● add or contract for required operational, financial and management information systems and capabilities and other specialized expert personnel to support our drug product candidate development, precommercial and planned commercialization efforts.
Treasury securities with maturities consistent with the estimated expected term of the awards. 63 Table of Contents Results of Operations Comparison of the Years Ended December 31, 2023, and December 31, 2022 The following table summarizes the results of our operations for the years ended December 31, 2023, and 2022: Year Ended December 31, (in thousands) 2023 2022 Variance Research and development expenses $ 5,600 $ 7,592 $ (1,992 ) General and administrative expenses 3,231 2,945 286 Total operating expenses 8,831 10,537 (1,706 ) Operating loss (8,831 ) (10,537 ) 1,706 Interest income 429 21 408 Net loss $ (8,402 ) $ (10,516 ) $ 2,114 Research and Development ( “ R&D ” ) Expenses R&D expenses for the year ended December 31, 2023 were $5,600,000, compared to $7,592,000 for the year ended December 31, 2022 .
Treasury securities with maturities consistent with the estimated expected term of the awards. 59 Table of Contents Results of Operations Comparison of the Years Ended December 31, 2024, and December 31, 2023 The following table summarizes the results of our operations for the years ended December 31, 2024 and 2023: Year Ended December 31, (in thousands) 2024 2023 Variance Research and development expenses $ 13,006 $ 5,600 $ 7,406 General and administrative expenses 3,156 3,231 (75 ) Total operating expenses 16,162 8,831 7,331 Operating loss (16,162 ) (8,831 ) (7,331 ) Other income 171 — 171 Interest income 404 429 (25 ) Net loss $ (15,587 ) $ (8,402 ) $ (7,185 ) Research and Development ( “ R&D ” ) Expenses R&D expenses for the year ended December 31, 2024, were $13,006,000, compared to $ 5,600,0 00 for the year ended December 31, 2023 .
Additional long-term funding is needed to generally to support our current and future product candidates through clinical trials, approval processes and, if applicable, commercialization. 67 Table of Contents Until we can generate a sufficient amount of product revenue to finance our cash requirements, we expect to finance our future cash needs primarily through a combination of equity offerings, including at-the-market sales programs, debt financings, strategic collaborations and grant funding.
Beyond our current funds, substantial additional long-term funding is needed to further develop our radiopharmaceutical and rare disease programs. 63 Table of Contents Until we can generate a sufficient amount of product revenue to finance our cash requirements, we expect to finance our future cash needs primarily through a combination of equity offerings, debt financings, strategic collaborations and grant funding.
We plan to continue the expansion of our drug development pipeline through acquiring or in-licensing additional product candidates, particularly those that leverage existing scientific and clinical data that helps reduce the risks of the next steps in clinical development. ● Utilize the expertise and prior experience of our team in the areas of asset acquisition, drug development and commercialization to establish ourselves as a leading biopharmaceutical company.
We plan to continue the expansion of our drug development pipeline through internal research and development, as well as acquire or in-license additional product candidates, particularly those that leverage existing scientific and clinical data to help reduce the risks of the next steps in clinical development.
The increase is due to interest earned on treasury bills and money market accounts that yielded higher interest rates in 2023 . Liquidity and Capital Resources Sources of Liquidity We have incurred losses and cumulative negative cash flows from operations since we commenced operations resulting in an accumulated deficit of approximately $60.2 million as of December 31, 2023.
Liquidity and Capital Resources Sources of Liquidity We have incurred losses and cumulative negative cash flows from operations since we commenced operations, resulting in an accumulated deficit of approximately $75.8 million as of December 31, 2024. We anticipate that we will continue to incur losses for the foreseeable future.
To date, we have not paid any claims or been required to defend any action related to our indemnification obligations. However, we may record charges in the future as a result of these indemnification obligations.
However, we may record charges in the future as a result of these indemnification obligations.
Indemnification In the normal course of business, we enter into contracts and agreements that contain a variety of representations and warranties and provide for general indemnification. Our exposure under these agreements is unknown because it involves claims that may be made against us in the future, but that have not yet been made.
Our exposure under these agreements is unknown because it involves claims that may be made against us in the future, but that have not yet been made. To date, we have not paid any claims or been required to defend any action related to our indemnification obligations.
For the year ended December 31, 2023, we sold 1,793,441 shares of our common stock at an average gross price per share of $1.21 for net proceeds of $2,072,503 after fees, commissions and expenses of $98,230.
For the year ended December 31, 2024, we sold 557,761 shares of our common stock at an average gross price per share of $7.72 for net proceeds of approximately $4.2 million after deducting fees, commissions and expenses of $107,806. We may implement additional at-the-market offerings in the future.
Year Ended December 31, (in thousands) 2023 2022 Variance Net cash used in operating activities $ (7,858 ) $ (7,228 ) $ (630 ) Net cash provided by (used in) investing activities 4,928 (4,919 ) 9,847 Net cash provided by fi nancing activities 2,027 33 1,994 Effect of exchange rates (17 ) (4 ) (13 ) Net decrease in cash and cash equivalents $ (920 ) $ (12,118 ) $ 11,198 During the years ended December 31, 2023 and 2022 , we had net cash outflows of $920,000 and $12,118,000, respectively, a decrease of $11,198,000.
Year Ended December 31, (in thousands) 2024 2023 Variance Net cash used in operating activities $ (6,404 ) $ (7,858 ) $ 1,454 Net cash (used in) provided by investing activities (14,338 ) 4,928 (19,266 ) Net cash provided by financing activities 59,292 2,027 57,265 Effect of exchange rates — (17 ) 17 Net increase (decrease) in cash and cash equivalents $ 38,550 $ (920 ) $ 39,470 During the years ended December 31, 2024 and 2023 , we had net cash inflow of $38,550,000 and net cash outflow of $920,000, respectively, an increase of $39,470,000.
We have not incurred any license or royalty obligations and the license has been terminated effective January 2024. 68 Table of Contents Service Providers In the normal course of business, we contract with service providers to assist in the performance of R&D, including drug product manufacturing, process development, clinical and preclinical development, and G&A including financial strategy, audit, tax and legal support.
We are currently conducting Phase 1 clinical trials and cannot reliably predict when we will be able to commence a Phase 2 clinical trial, if at all. 64 Table of Contents Service Providers In the normal course of business, we contract with service providers to assist in the performance of R&D, including drug product manufacturing, process development, clinical and preclinical development, and G&A including financial strategy, audit, tax and legal support.
Cash Flow Used in Operating Activities The cash flow used in operating activities during the year ended December 31, 2023, compared to the year ended December 31, 2022 was essentially flat.
The increase in cash used in investing activities during the year ended December 31, 2024, compared to the year ended December 31, 2023, was due to the increase in funds available to invest from the October 2024 financing.
In February 2024, the Company announced promising preclinical data and regulatory clearance in Australia to commence a first-in-human Phase 1 clinical trial of MNPR-101-Zr for imaging tumors in patients with advanced cancers. Monopar is moving aggressively towards opening enrollment to patients in this Phase 1.
In February 2024, we received regulatory clearance in Australia to commence a first-in-human Phase 1 imaging and dosimetry clinical trial with our novel radiopharmaceutical imaging agent MNPR-101-Zr (MNPR-101 conjugated to zirconium-89) in patients with advanced cancers, and in April 2024, we launched the Phase 1 trial.
The Company's radiopharmaceutical development team has conjugated MNPR-101, its proprietary first-in-class humanized monoclonal antibody that is highly selective against uPAR, to imaging and therapeutic radioisotopes, to develop highly precise imaging and therapeutic radiopharmaceutical agents that have the potential to image and eradicate tumors expressing uPAR while sparing healthy tissues.
We have conjugated MNPR-101 to imaging and therapeutic radioisotopes for the purpose of creating highly precise radiopharmaceutical agents that have the potential to image and treat tumors expressing uPAR while reducing exposure to healthy tissues.
During 2023 , we had net cash provided by investing activities as some of our investments made in 2022 matured and were used in operating activities, as well as higher net cash provided by financing activities due to more funds raised from sales of our common stock under an at-the-market sales program compared to 2022 .
The increase in net cash provided by financing activities during the year ended December 31, 2024, compared to the year ended December 31, 2023, was a result of higher proceeds from sales of our common stock under an at-the-market sales program and the October 2024 and December 2024 financing rounds.
This represents a decrease of $1,992,000 primarily attributed to (1) a $1,375,000 decrease for Validive clinical trial and manufacturing costs as a result of the termination of that program in March 2023, (2) a $904,000 decrease for a reduction in camsirubicin clinical trial and manufacturing costs, (3) a $126,000 decrease in R&D salaries, partially offset by (1) a $408,000 increase related to MNPR-101 radiopharma activity and (2) a $5,000 net increase in other R&D expenses.
This represents an increase of $7,406,000 primarily attributed to (1) a $8,557,000 increase related to the in-licensing of ALXN1840, (2) a $335,000 increase in R&D personnel expenses and (3) a $130,000 net increase in other R&D expenses, partially offset by (4) a $1,616,000 decrease in Validive clinical trial-related expenses due to the closure of the trial in March 2023.
We anticipate that we will continue to incur losses for the foreseeable future. We expect that our R&D and G&A expenses will increase to enable the execution of our strategic plan. As a result, we anticipate that we will seek to raise additional capital within the next 12 months to fund our future operations.
We expect that our R&D and G&A expenses will increase to enable the execution of our strategic plan. We anticipate that the currently available funds as of March 14, 2025, will fund our planned operations at least throug h December 31, 2026 .
General and Administrative ( " G&A") Expenses G&A expenses for the year ended December 31, 2023 , were $3,231,000, compared to $2,945,000 for the year ended December 31, 2022 .
General and Administrative ( “ G&A”) Expenses G&A expenses for the year ended December 31, 2024 , were $3,156,000, compared to $3,231,000 for the year ended December 31, 2023 . This represents a decrease of $75,000 primarily attributed to a decrease in G&A personnel expenses due to the reduction in equity grants to our Chief Executive Officer in 2024.
The agreement does not require the payment of sales royalties. There can be no assurance that we will achieve any milestones. As of March 8, 2024, we had not reached any milestones and had not been required to pay XOMA Ltd. any funds under this license agreement.
As of March 14, 2025, we had not reached any milestones and had not been required to pay XOMA Ltd. any funds under this license agreement. The first milestone payment is payable upon first dosing of a human patient in a Phase 2 clinical trial.
As discussed further below and elsewhere in this Annual Report, we expect that our current funds will be sufficient at least through June 30, 2025 for us to: (1) conduct and conclude our first-in-human clinical trial with our MNPR-101-Zr radiopharmaceutical program; (2) continue our ongoing open-label Phase 1b camsirubicin clinical trial; and (3) continue to research MNPR-202.
As discussed further below and elsewhere in this Annual Report on Form 10-K, we expect that our current funds will be sufficient at least through Decem ber 31, 2026, in order for us to: (1) assemble a regulatory package and file an NDA for the in-licensed ALXN1840 investigational drug candidate for Wilson disease; (2) continue to conduct and conclude our first-in-human imaging and dosimetry clinical trial with MNPR-101-Zr; (3) continue to conduct our first-in-human therapeutic clinical trial of MNPR-101-Lu; (4) advance our preclinical MNPR-101-Ac program into the clinic; and (5) invest in internal R&D projects to expand our radiopharmaceutical pipeline.
We are building a drug development pipeline through the licensing and acquisition of therapeutics in late preclinical or in clinical development stages. We leverage our scientific and clinical experience to help reduce the risk of and accelerate the clinical development of our drug product candidates.
We leverage our scientific and clinical experience to help reduce the risk and accelerate the clinical development of our drug product candidates. Financial Status Our cash, cash equivalents and investments as of December 31, 2024, were $60.2 million.
Our strategic goal is to acquire, develop and commercialize promising oncology product candidates that address important unmet medical needs of cancer patients. Five key elements of our strategy to achieve this goal are to: ● Continue the development of MNPR-101 for radiopharmaceutical use as a therapeutic as well as a diagnostic imaging agent .
Our strategic goal is to acquire, develop, and commercialize innovative treatments for patients with unmet medical needs. Key elements of our strategy to achieve this goal are to: ● Assemble a regulatory package for ALXN1840 to file an NDA .
Office Lease We are currently leasing office space on a month-to-month basis for our executive headquarters at 1000 Skokie Blvd., in the Village of Wilmette, Illinois for $4,238 per month. Legal Contingencies We are currently not, and to date have never been, a party to any adverse material legal proceedings.
Office Lease We recently entered into a 36-month lease beginning April 1, 2025 for our executive headquarters at 1000 Skokie Blvd in the Village of Wilmette, Illinois at a monthly rate of $3,580/month, and we also have a month-to-month lease for additional space at the same location for $2,379 per month.
We will seek to obtain needed capital through a combination of equity offerings, including at-the-market sales programs, debt financings, strategic collaborations and grant funding.
We will seek to obtain needed capital through a variety of methods, including but not limited to the sale of our common stock, debt financings, strategic partnerships or other sources of capital at our disposal. We invest our cash equivalents in money market accounts and U.S.
Contractual Obligations and Commitments License, Development and Collaboration Agreements XOMA Ltd. Pursuant to a non-exclusive license agreement with XOMA Ltd. for the humanization technology used in the development of MNPR-101, we are obligated to pay XOMA Ltd. clinical, regulatory and sales milestones which could reach up to $14.925 million if we achieve all milestones for MNPR-101.
Under the terms of the non-exclusive license with XOMA Ltd., we are to pay only upon the achievement of clinical, regulatory and sales milestones, potentially totaling $14.925 million. The agreement does not require the payment of sales royalties.
The expected term for stock options granted during the years ended December 31, 2023 and 2022, was estimated using the simplified method. Forfeitures only include actual forfeitures to-date as the Company accounts for forfeitures as they occur due to a limited history of forfeitures.
For options granted in 2024, the expected volatility rates are estimated based on our actual historical volatility over the four-year period from our initial public offering on December 18, 2019, through December 31, 2023. The expected term for stock options granted during the years ended December 31, 2024 and 2023, was estimated using the simplified method.
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Financial Status Our cash, cash equivalents and investments as of December 31, 2023, were $7.3 million.
Added
Overview We are a clinical-stage biopharma company with late-stage ALXN1840 for Wilson disease, and radiopharmaceutical programs, including Phase 1-stage MNPR-101-Zr for imaging advanced cancers along with Phase 1a-stage MNPR-101-Lu and late preclinical-stage MNPR-101-Ac225 for the treatment of advanced cancers.
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We will require additional funding to further advance our clinical and preclinical programs and we anticipate that we will seek to raise additional capital within the next 12 months to fund our future operations.
Added
On October 30, 2024, pursuant to a placement agent agreement with Rodman & Renshaw LLC, we sold 1,181,540 shares of our common stock at $16.25 per share in a public offering, yielding net proceeds of approximately $17.8 million, after deducting placement agent fees and other offering expenses.
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From January 1 to March 8, 2024, we sold 2,545,305 shares of our common stock at an average gross price per share of $1.29 for net proceeds of $3,194,310, after fees and commissions of $81,932. MNPR-101 for Radiopharmaceutical Use, Development Update Our proprietary, novel MNPR-101 radiopharmaceutical program is a first-in-class urokinase plasminogen activator receptor ("uPAR") targeting radiopharmaceutical for advanced tumors.
Added
On December 23, 2024, pursuant to an underwriting agreement with Piper Sandler & Co., we sold 798,655 shares of our common stock at $23.79 per share in a public offering.
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Camsirubicin Clinical Update The Phase 1b open-label, dose-escalating clinical trial of camsirubicin in patients with advanced soft tissue sarcoma (“ASTS”) is currently ongoing and enrolling in the fifth dose-level cohort (650 mg/m 2 ), which is nearly 2.5x the highest dose evaluated in any prior camsirubicin clinical trial (265 mg/m 2 ).
Added
Concurrent with that offering, we completed a private placement of pre-funded warrants to purchase 882,761 shares of common stock at a purchase price of $23.789 per pre-funded warrant to an institutional investor. The net proceeds of the shares and the pre-funded warrants sold were approximately $37.4 million after fees, commissions and other offering expenses.
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MNPR-202 and Related Analogs Updates MNPR-202, an analog of camsirubicin designed to potentially treat doxorubicin- and camsirubicin-resistant cancers, is being tested in preclinical models by our collaborator, the Cancer Science Institute of Singapore at the National University of Singapore.
Added
ALXN1840 for Wilson Disease ALXN1840 (bis-choline tetrathiomolybdate) is an investigational once-daily, orally-administered drug candidate in development for the treatment of Wilson disease, a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised.
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Discontinuation of Validive Development On March 27, 2023, we announced that based on Validive not meeting the pre-specified efficacy threshold in our interim clinical trial data analysis, we terminated the study, discontinued the development of Validive and subsequently terminated the license with Onxeo, SA. 60 Table of Contents Our Strategy Our management team has extensive experience in developing therapeutics and medical technologies through global regulatory approval and commercialization.
Added
Over time this excess copper results in the build-up of toxic copper levels in the liver, brain, and other organs, leading to damage that greatly impacts a patient’s life. Patients can develop a wide range of symptoms, including liver disease and psychiatric or neurological manifestations, such as personality changes, tremors and difficulty walking, swallowing or talking.
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Based on promising preclinical results utilizing radiolabeled MNPR-101, we have been cleared to conduct a Phase 1 dosimetry clinical trial of MNPR-101-Zr in patients with advanced cancers in Australia. ● Advance the clinical development of camsirubicin, by pursuing indications where doxorubicin has demonstrated efficacy.
Added
In some cases, the damage and loss of function may be irreversible. ALXN1840 is a novel small molecule designed to selectively and tightly bind and remove copper from the body’s tissues and blood. ALXN1840 has been granted Orphan Drug Designation and Fast Track designation in the U.S. and orphan designation in the EU.
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ASTS will be the first indication, which is anticipated to allow camsirubicin to go head-to-head against doxorubicin, the current first-line treatment. In this indication, camsirubicin previously demonstrated clinical benefit (stable disease or partial response) in 52.6% of patients evaluable for tumor progression in a single-arm Phase 2 study.
Added
Wilson disease affects 1 in 30,000 live births in the U.S. There are an estimated 10,000 Wilson disease patients in the U.S., with an estimated 5,000 patients currently diagnosed and being treated with SoC.
Removed
Clinical benefit was proportional to dose and was consistently observed at higher cumulative doses of camsirubicin (>1000 mg/m 2 ). Camsirubicin was very well tolerated in this Phase 2 study and underscored the ability to potentially administer camsirubicin without restriction as to cumulative dose (doxorubicin is limited due to heart toxicity to 450 mg/m 2 cumulative dose).
Added
Alexion completed a pivotal Phase 3 clinical trial of Wilson disease patients on ALXN1840, which met its primary endpoint in assessing copper mobilization over 48 weeks, defined as daily mean Area Under the Effect Curve (“AUEC”) for directly measured non-ceruloplasmin-bound copper (“dNCC”).
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Our current ongoing Phase 1b clinical trial continues towards establishing a new, higher recommended dose for the next Phase 2 ASTS clinical trial. ● Continue the development of MNPR-202 and related analogs in multiple types of cancers.
Added
In the trial, 214 patients were enrolled, and the trial was randomized, rater-blinded, and multi-centered, designed to evaluate the efficacy and safety of ALXN1840 versus standard-of-care (“SoC”) in patients with Wilson disease aged 12 years and older. Patients taking ALXN1840 experienced rapid copper mobilization, with a response at 4 weeks and sustained through the 48 weeks.
Removed
The 2-pyrrilino camsirubicin analog (MNPR-202) and related analogs represent proprietary compositions of matter designed to retain the non-cardiotoxic backbone of camsirubicin yet exhibit novel features in terms of antitumor activity and mechanism that distinguish these analogs from camsirubicin as well as from doxorubicin, potentially addressing camsirubicin- and doxorubicin-resistant cancers. ● Expand our drug development pipeline through in-licensing and acquisition of product candidates.
Added
The primary endpoint demonstrated three-times greater copper mobilization with ALXN1840 compared to the SoC arm (Least Square Mean Difference (“LSM Diff”) 2.18 µmol/L; p Additionally, data from patients in the Phase 3 clinical trial who exhibited at the time of study entry an incomplete and/or intolerant response (“IIR”) to prior treatment on SoC showed that more patients on ALXN1840 as compared to SoC in the trial exhibited improved neurological symptoms (45% vs. 20%, respectively) and fewer exhibited worsened neurological symptoms (5% vs. 17%, respectively) when assessed on a reported Minimal Clinically Important Difference (“MCID”) scale.
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In aggregate, our team has co-founded BioMarin Pharmaceutical (Nasdaq: BMRN), Sensant Corp (acquired by Siemens), American BioOptics (assets acquired by Olympus), Raptor Pharmaceuticals ($800 million sale to Horizon Therapeutics), and Tactic Pharma, LLC (“Tactic Pharma”) (sale of lead asset, choline tetrathiomolybdate, was ultimately acquired by Alexion in June 2018 for $764 million; Alexion was subsequently acquired by AstraZeneca). 61 Table of Contents Implications of Being an Emerging Growth Company We qualify as an “emerging growth company” as defined in the Jumpstart Our Business Startups Act of 2012 (“JOBS Act”).
Added
These data suggest ALXN1840 may reduce the risk of neurological worsening when compared to SoC. Alexion terminated the ALXN1840 program in Wilson disease based on its review of results from Phase 2 mechanistic trials and discussions with regulatory authorities.
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An emerging growth company may take advantage of specified reduced reporting burdens that are otherwise applicable generally to public companies.
Added
Their analysis of the Phase 2 mechanistic trials was that they failed to demonstrate a net-negative copper balance in Wilson disease patients during short-term treatment with ALXN1840 and to reduce hepatic copper concentration after treatment with ALXN1840. The decision not to progress the ALXN1840 program in Wilson disease was not related to any safety signals.
Removed
These provisions include, but are not limited to: ● inclusion of only two years, as compared to three years, of audited financial statements in addition to any required unaudited interim financial statements with correspondingly reduced “Management’s discussion and analysis of financial condition and results of operations” disclosures; ● an exemption from the auditor attestation requirement in the assessment of our internal control over financial reporting pursuant to the Sarbanes-Oxley Act of 2002 (“Sarbanes-Oxley Act”); ● an exemption from compliance with any new requirements adopted by the Public Company Accounting Oversight Board (“PCAOB”) requiring mandatory audit firm rotation; ● reduced disclosure about executive compensation arrangements; and ● an exemption from the requirement to seek non-binding advisory votes on executive compensation or golden parachute arrangements.
Added
Following Alexion’s decision, in October 2024, we entered into an exclusive worldwide license for the program and assumed responsibility for all future global development and commercialization activities. In the near term, we will be focusing on assembling a regulatory package and submitting an NDA.
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We expect to take advantage of these provisions until we are no longer an emerging growth company.
Added
We expect to submit an NDA to the FDA in early 2026. 57 Table of Contents MNPR-101 for Radiopharmaceutical Use, Development Update We have a proprietary first-in-class humanized monoclonal antibody, MNPR-101, that targets the urokinase plasminogen activator receptor (“uPAR”). uPAR is expressed on several of the more aggressive, deadly cancers including pancreatic, breast, ovarian, colorectal, and bladder cancers.
Removed
We will remain an emerging growth company until the earliest of (1) the last day of the year (a) following the fifth anniversary of the completion of our initial public offering which is December 2024, (b) in which we have total annual gross revenue of at least $1.235 billion or (c) in which we are deemed to be a large accelerated filer, which means the market value of our common stock that is held by non-affiliates exceeds $700 million as of the prior June 30th, and (2) the date on which we have issued more than $1.0 billion in non-convertible debt during the prior three-year period.
Added
In July 2024, we announced the enrollment of our first patient and in September 2024, we announced positive early clinical data validating the tumor-targeting ability of MNPR-101-Zr.
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We expect to no longer qualify as an emerging growth company at the end of 2024. We have elected to take advantage of certain of the reduced disclosure obligations in this Annual Report on Form 10-K, and may elect to take advantage of other reduced reporting requirements in future filings.
Added
In August 2024, we received regulatory clearance in Australia to commence a first-in-human Phase 1a clinical trial of our novel uPAR-targeted radiopharmaceutical therapy MNPR-101-Lu (MNPR-101 conjugated to lutetium-177) in patients with advanced solid cancers. We launched the trial in October 2024, and it is now active and open for patient enrollment.
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As a result, the information that we provide to our stockholders may be reduced and/or less detailed than what you might find from other public reporting companies.
Added
We dosed our first with MNPR-101-Lu in early December 2024.
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In addition, we are also a “smaller reporting company” as defined in Rule 12b-2 of the Exchange Act and have elected to take advantage of certain of the scaled back disclosure requirements available to smaller reporting companies such as avoiding the extensive narrative disclosure required of other reporting companies, particularly in the description of executive compensation.
Added
In October 2024, we presented clinical data at the European Association of Nuclear Medicine Annual Congress 2024 showing significant uptake of MNPR-101-Zr in a patient with advanced ovarian cancer together with preclinical and clinical data showing favorable biodistribution, tumor uptake, and low off-target binding of our uPAR-targeted radiopharmaceuticals MNPR-101-Zr, MNPR-101-Lu, and MNPR-101-Ac (MNPR-101 conjugated to actinium-225).
Removed
Our status as a smaller reporting company will not be impacted by the loss of emerging growth company status at the end of 2024, so we may continue to take advantage of these scaled disclosure requirements so long as we qualify.

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