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What changed in X4 Pharmaceuticals, Inc's 10-K — 2024 vs 2025

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Paragraph-level year-over-year comparison of X4 Pharmaceuticals, Inc's 2024 and 2025 10-K annual filings, covering the Business, Risk Factors, Legal Proceedings, Cybersecurity, MD&A and Market Risk sections. Every new, removed and edited paragraph is highlighted side-by-side so you can see exactly what management changed in the 2025 report.

+743 added−678 removedSource: 10-K (2026-03-17) vs 10-K (2025-03-26)

Top changes in X4 Pharmaceuticals, Inc's 2025 10-K

743 paragraphs added · 678 removed · 308 edited across 6 sections

Item 1. Business

Business — how the company describes what it does

110 edited+340 added−228 removed7 unchanged
Biggest changeDuring the seven-year exclusivity period, the FDA may not approve any other applications to market the same drug for the same disease, except in limited circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity.
Biggest changeIf a product that has orphan drug designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to orphan drug exclusive approval (or exclusivity), which means that the FDA may not approve any other applications, including a full NDA, to market the same product for the same approved use or indication for seven years, except in limited circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity by means of greater effectiveness, greater safety or providing a major contribution to patient care or if the holder of the orphan drug exclusivity cannot assure the availability of sufficient quantities of the orphan drug to meet the needs of patients with the same use or indication for which the already-approved or licensed product was approved or licensed.
Either we or Genzyme may terminate the Genzyme Agreement in the event of the bankruptcy or uncured material breach by the other party. Genzyme may terminate the Genzyme Agreement if we or our affiliates initiate a patent challenge of the patents licensed under the agreement.
Either we or Genzyme may terminate the Genzyme Agreement in the event of a bankruptcy or uncured material breach by the other party. Genzyme may terminate the Genzyme Agreement if we or our affiliates initiate a patent challenge of the patents licensed under the agreement.
Royalty rates are subject to reduction under the agreement in specified circumstances, including in any country if we are required to obtain a license from any third party to the extent our patent rights might infringe the third party’s patent rights, if a licensed product is not covered by a valid claim in that country, or if sales of generic products reach certain thresholds in that country.
Royalty rates are subject to reduction under the Genzyme Agreement in specified circumstances, including in any country if we are required to obtain a license from any third party to the extent our patent rights might infringe the third party’s patent rights, if a licensed product is not covered by a valid claim in that country, or if sales of generic products reach certain thresholds in that country.
Subject to customary rights of each party to earlier terminate the agreement, the term of the agreement continues, on a country-by-country basis, until the later of: (i) the tenth (10th) anniversary of the first commercial sale of mavorixafor, (ii) expiration of regulatory market exclusivity of mavorixafor or (iii) expiration of the last-to-expire licensed patent in such country.
Subject to customary rights of each party to earlier terminate the Norgine Agreement, the term of the agreement continues, on a country-by-country basis, until the later of: (i) the tenth (10th) anniversary of the first commercial sale of mavorixafor, (ii) expiration of regulatory market exclusivity of mavorixafor or (iii) expiration of the last-to-expire licensed patent in such country.
Our obligation to pay royalties for each licensed product expires on a country-by-country basis on the latest of (i) the expiration of licensed patent rights that cover that licensed product in that country, (ii) the expiration of regulatory exclusivity in that country and (iii) ten years after the first commercial sale of such licensed product in that country.
Our obligation to pay royalties for each licensed product expires on a 9 country-by-country basis on the latest of (i) the expiration of licensed patent rights that cover that licensed product in that country, (ii) the expiration of regulatory exclusivity in that country and (iii) ten years after the first commercial sale of such licensed product in that country.
Orphan drug exclusivity does not prevent the FDA from approving a different drug for the same disease or condition, or the same drug for a different disease or condition. Among the other benefits of orphan drug designation are tax credits for certain research and a waiver of the NDA application fee.
Orphan drug exclusivity does not prevent the FDA from approving a different drug or biologic for the same disease or condition, or the same drug or biologic for a different disease or condition. Among the other benefits of orphan drug designation are tax credits for certain research and a waiver of the NDA application fee.
Genzyme has retained the non-exclusive right to conduct preclinical research involving 11 compounds in any field, including any fields licensed to us, but has not retained rights to conduct any clinical development or commercialization of those compounds identified in the agreement in any of the fields licensed to us.
Genzyme has retained the non-exclusive right to conduct preclinical research involving compounds in any field, including any fields licensed to us, but has not retained rights to conduct any clinical development or commercialization of those compounds identified in the agreement in any of the fields licensed to us.
License Agreements License Agreement with Genzyme In July 2014, we entered into a license agreement with Genzyme Corporation (“Genzyme”), a wholly owned subsidiary of Sanofi, pursuant to which we were granted an exclusive license to certain patent applications and other intellectual property owned or controlled by Genzyme related to the CXCR4 receptor to develop and commercialize products containing licensed compounds, including but not limited to, mavorixafor.
License Agreements License Agreement with Genzyme In July 2014, we entered into a license agreement (the “Genzyme Agreement”) with Genzyme Corporation (“Genzyme”), a wholly owned subsidiary of Sanofi, pursuant to which we were granted an exclusive license to certain patent applications and other intellectual property owned or controlled by Genzyme related to the CXCR4 receptor to develop and commercialize products containing licensed compounds, including but not limited to, mavorixafor.
Pursuant to the terms of the Norgine Agreement, we shall receive the following payments from Norgine: (i) an upfront payment in the amount of €28.5 million (such payment was received in January 2025), (ii) up to €226.0 million upon the achievement of certain regulatory, commercial and sales milestones, and (iii) escalating double-digit royalties of up to mid-twenties on any future net sales in the Territory.
Pursuant to the terms of the Norgine Agreement, we are entitled to receive the following payments from Norgine: (i) an upfront payment in the amount of €28.5 million (such payment was received in January 2025), (ii) up to €226.0 million upon the achievement of certain regulatory, commercial and sales milestones, and (iii) escalating double-digit royalties of up to mid-twenties on any future net sales in the Territory.
If the applicant has provided a Paragraph IV certification to the FDA, the applicant must also send notice of the Paragraph IV certification to the NDA and patent holders once the application has been accepted for filing by the FDA.
If the ANDA applicant has provided a Paragraph IV certification to the FDA, the applicant must also send notice of the Paragraph IV certification to the NDA and patent holders once the ANDA has been accepted for filing by the FDA.
As of December 31, 2024, we are obligated to pay Genzyme future milestone payments in the aggregate amount of up to $13.0 million, contingent upon our achievement of certain late-stage regulatory and sales milestones with respect to licensed products, and tiered royalties based on net sales of licensed products that we commercialize under the Genzyme agreement.
As of December 31, 2025, we are obligated to pay Genzyme future milestone payments in the aggregate amount of up to $5.0 million, contingent upon our achievement of certain late-stage regulatory milestones with respect to licensed products, and tiered royalties based on net sales of licensed products that we commercialize under the Genzyme Agreement.
The term of the Genzyme Agreement will continue until the later of the expiration of the last-to-expire valid claim of the patents licensed under the agreement that cover any licensed product, the expiration of regulatory exclusivity applicable to any licensed product, and 10 years from the date of first commercial sale of any licensed product.
The term of the Genzyme Agreement will continue until the later of (i) the expiration of the last-to-expire valid claim of the patents licensed under the agreement that cover any licensed product, (ii) the expiration of regulatory exclusivity applicable to any licensed product and (iii) 10 years from the date of first commercial sale of any licensed product.
Because antagonism of the CXCR4 receptor has been shown to increase the trafficking of white blood cells, we believe that therapeutic inhibition of the CXCR4/CXCL12 axis holds the potential to benefit people with a wide variety of diseases where there remain significant unmet needs, including chronic neutropenic disorders and certain types of cancer.
Because antagonism of the 6 CXCR4 receptor has been shown to increase the trafficking of white blood cells, we believe that therapeutic inhibition of the CXCR4/CXCL12 axis holds the potential to benefit people with a wide variety of diseases where there remain significant unmet needs, including CN disorders and certain types of cancer.
Of those nine, eight had G-CSF reduced at the earliest timepoint permitted and three were taken completely off of G-CSF prior to their Month 6 visit. • Mean reductions in G-CSF were 52% at Month 3 (n=8) and 70% at Month 6 (n=9), while mean ANC levels remained in the normal range. • The three participants receiving mavorixafor who remained on stable doses of G-CSF maintained mean ANC levels in the normal range at all timepoints. • Safety summary : Mavorixafor was generally well tolerated as a monotherapy and in combination with G-CSF, with no drug-related serious adverse events reported, consistent with previous clinical studies.
Of those nine, eight had G-CSF reduced at the earliest time-point permitted and three were taken completely off of G-CSF prior to their Month 6 visit. • Mean reductions in G-CSF were 52% at Month 3 (n=8) and 70% at Month 6 (n=9), while mean ANC levels remained in the normal range. • The three patients receiving mavorixafor who remained on stable doses of G-CSF maintained mean ANC levels in the normal range at all time-points. • Safety summary : Mavorixafor was generally well tolerated as a monotherapy and in combination with G-CSF, with no drug-related serious adverse events reported, consistent with previous clinical studies.
Due to its ability to increase the mobilization of mature, functional white blood cells from the bone marrow into the bloodstream, we believe that mavorixafor has the potential to benefit people with a range of immunodeficiencies – a therapeutic market that is principally served by injectable therapies frequently associated with treatment-limiting adverse events. In April 2024, the U.S.
Due to its ability to increase the mobilization of mature, functional white blood cells from the bone marrow into the bloodstream, we believe that mavorixafor has the potential to benefit people with a range of immunodeficiencies – a therapeutic market that is principally served by injectable therapies frequently associated with treatment-limiting adverse events.
Upon closing of the agreement, Norgine paid us a one-time, nonrefundable fee of €28.5 million for the transfer of such IP. Under our Genzyme agreement we owe a 15% royalty on this upfront payment and certain other regulatory and sales-based milestones that we earn under the Norgine license and supply agreement.
Upon closing of the agreement, Norgine paid us a one-time, nonrefundable fee of €28.5 million for the transfer of such IP. Under the Genzyme Agreement we paid a 15% royalty to Genzyme on this upfront payment, and we may owe a 15% royalty on certain other regulatory and sales-based milestones that we earn under the Norgine Agreement.
In addition, during the Phase 2 clinical trial, we conducted a sub-study comparing the mean percentage of functional neutrophils in samples from healthy donors (n=5) to participants in the Phase 2 study (n=9) using two common study methods.
In addition, during the Phase 2 clinical trial, a sub-study was conducted comparing the mean percentage of functional neutrophils in samples from healthy donors (n=5) to patients in the Phase 2 study (n=9) using two common study methods.
Under the centralized procedure, the EMA’s CHMP is responsible for conducting the initial assessment of a product and for several post-authorization and maintenance activities, such as the assessment of modifications or extensions to an existing MA.
Under the centralized procedure, the CHMP is responsible for conducting the initial assessment of a drug. The CHMP is also responsible for several post-authorization and maintenance activities, such as the assessment of modifications or extensions to an existing MA.
The tiered royalty payments are subject to royalty stacking, and to a material reduction on a country-by-country basis if a generic version of mavorixafor becomes available in the applicable country. We and Norgine will collaborate closely on regulatory filings, while we continue to be responsible for the ongoing global, pivotal Phase 3 4WARD clinical trial evaluating mavorixafor in chronic neuropathy.
The tiered royalty payments are subject to royalty stacking, and to a material reduction on a country-by-country basis if a generic version of mavorixafor becomes available in the applicable country. We and Norgine will collaborate closely on regulatory filings, while we continue to be responsible for the ongoing 4WARD clinical trial evaluating mavorixafor in CN.
Orphan drug designation must be requested before submitting an NDA. After the FDA grants orphan drug designation, the generic identity of the drug and its designated orphan use are disclosed publicly by the FDA. Orphan drug designation does not convey any advantage in, or shorten the duration of, the regulatory review and approval process.
Orphan drug designation must be requested before submitting an NDA. After the FDA grants orphan drug designation, the identity of the therapeutic agent and its potential orphan use are disclosed publicly by the 15 FDA. The orphan drug designation does not convey any advantage in, or shorten the duration of, the regulatory review or approval process.
A “similar medicinal product” is defined as a medicinal product containing a similar active substance or substances as contained in an authorized orphan medicinal product, and which is intended for the same therapeutic indication.
A similar medicinal product is defined as a medicinal product containing a similar active substance or substances as contained in a currently authorized orphan medicinal product, and which is intended for the same therapeutic indication.
Upon the first potential sale of our drug candidate in the U.S., we have incurred a royalty on annual net sales at a rate of 6% up to $150 million, and would incur 10% on the portion of annual net sales between $150 million and $300 million, and 12% thereafter.
Royalties: Upon the first sale of our drug candidate in the U.S., we incur a royalty on annual net sales at a rate of 6% up to $150 million, and would incur 10% on the portion of annual net sales between $150 million and $300 million, and 12% thereafter on annual net sales of over $300.0 million.
Chronic neutropenia is defined as periods lasting more than three months persistently or intermittently where there are abnormally low levels of neutrophils circulating in the blood, and may be idiopathic (of unknown origin), cyclic (episodes typically occurring every three weeks), or congenital (of genetic causation).
Chronic Neutropenia Market Overview and Opportunity CN is a primary, rare blood condition defined as periods lasting more than three months persistently or intermittently where there are abnormally low levels of neutrophils circulating in the blood, and may be idiopathic (of unknown origin), cyclic (episodes typically occurring every three weeks), or congenital (of genetic causation).
The centralized procedure is mandatory for certain types of products, including products produced by biotechnological processes, products designated as orphan medicinal products, advanced-therapy medicinal products (gene-therapy, somatic cell-therapy, or tissue-engineered medicines) and medicinal products containing a new active substance indicated for the treatment of HIV, AIDS, cancer, neurodegenerative disorders, diabetes, auto-immune and other immune dysfunctions or viral diseases.
The centralized procedure is compulsory for specific medicinal products, including for medicines developed by means of certain biotechnological processes, products designated as orphan medicinal products, advanced therapy medicinal products (gene therapy, somatic cell therapy, or tissue engineered medicines) and medicinal products with a new active substance indicated for the treatment of certain diseases (HIV/AIDS, cancer, neurodegenerative disorders, diabetes, auto-immune diseases and other immune dysfunctions, and viral diseases).
Orphan Drug and Rare Pediatric Disease Designation Under the Orphan Drug Act, the FDA may grant orphan drug designation to drugs intended to treat a rare disease or condition—generally a disease or condition that affects fewer than 200,000 individuals in the United States (or affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales of such drug in the United States).
Orphan Drug Designation and Exclusivity Under the Orphan Drug Act of 1983, the FDA may grant orphan drug designation to a product candidate intended to treat a rare disease or condition, which is generally a disease or condition that affects fewer than 200,000 individuals in the United States, or 200,000 or more individuals in the United States for which there is no reasonable expectation that the cost of developing and making available in the United States a drug or biologic for this type of disease or condition will be recovered from sales in the United States for that product candidate.
Specifically, new drugs are eligible for Fast Track designation if they are intended to treat a serious or life-threatening condition and preclinical or clinical data demonstrate the potential to address unmet medical needs for the condition. Fast Track designation applies to both the product and the specific indication for which it is being studied.
Specifically, new products are eligible for fast track designation if they are intended to treat a serious or life-threatening disease or condition and data demonstrate the potential to address unmet medical needs for the disease or condition. Fast track designation applies to the combination of the product and the specific indication for which it is being studied.
In September 2022, we announced positive results from this Phase 1b clinical trial, which enrolled a total of 25 participants: • 100% of study participants responded to treatment with a single dose of 400 mg of mavorixafor, alone or dosed concurrently with G-CSF: ▪ Participants achieved a mean ANC increase at peak of >3,000 cells per microliter. ▪ Consistent responses were seen across all of the chronic neutropenic disorders studied – idiopathic, cyclic, and congenital neutropenia. • All neutropenic participants (n=14) reached normalized ANC levels (>1,500 cells per microliter): ▪ When assessed as a monotherapy in participants with severe chronic neutropenia who were not being treated with G-CSF (n=6), a single dose of mavorixafor led to normalized ANC levels in all participants within 2 hours, with a mean ANC increase at peak of ~2,500 cells per microliter. ▪ When assessed in participants with moderate or severe neutropenia despite being treated with G-CSF (n=8), 100% reached normalized ANC levels, suggesting the potential of mavorixafor to both normalize the neutrophil counts in patients with partial response to G-CSF and also to potentially enable the reduction or elimination of G-CSF dosing. • When assessed in participants with chronic neutropenia with normalized ANC counts on chronic G-CSF (n=11), all participants experienced a consistent and sustained increase in ANC, suggesting mavorixafor’s potential to reduce or possibly eliminate G-CSF treatment in these patients. • Mavorixafor was well tolerated in the study; all treatment-related adverse events were deemed to be low grade, 9 consistent with previous clinical studies in WHIM syndrome, and no treatment-related serious adverse events were reported.
In September 2022, we announced positive results from this Phase 1b clinical trial, which enrolled a total of 25 patients: • 100% of patients responded to treatment with a single dose of 400 mg of mavorixafor, alone or dosed concurrently with G-CSF: ▪ Patients achieved a mean absolute neutrophil count (“ANC”) increase at peak of >3,000 cells per microliter. ▪ Consistent responses were seen across all of the chronic neutropenic disorders studied – idiopathic, cyclic, and congenital neutropenia. • All neutropenic patients (n=14) reached normalized ANC levels (>1,500 cells per microliter): ▪ When assessed as a monotherapy in patients with severe CN who were not being treated with G-CSF (n=6), a single dose of mavorixafor led to normalized ANC levels in all patients within 2 hours, with a mean ANC increase at peak of ~2,500 cells per microliter. ▪ When assessed in patients with moderate or severe neutropenia, a single dose of mavorixafor in combination with G-CSF (n=8), all patients reached normalized ANC levels. • When assessed in patients with CN with normalized ANC counts on chronic G-CSF (n=11), all patients experienced a consistent and sustained increase in ANC. • Mavorixafor was well tolerated in the study; all treatment-related adverse events were deemed to be low grade, consistent with previous clinical studies in WHIM syndrome, and no treatment-related serious adverse events were reported.
CXCR4 receptor stimulation by its cognate ligand, CXCL12, has been shown to play a key role in the maturation and mobilization of white blood cells such as neutrophils, lymphocytes (including both B cells and T cells), and monocytes, into the bloodstream.
CXCR4 is a cell receptor that helps regulate the movement of immune cells within the body. CXCR4 receptor stimulation by its cognate ligand, CXCL12, has been shown to play a key role in the maturation and mobilization of white blood cells, such as neutrophils, lymphocytes (including both B cells and T cells), and monocytes, into the bloodstream.
Our annual reports on Form 10-K, quarterly reports on Form 10-Q, proxy statements, current reports on Form 8-K and amendments to those reports filed or furnished pursuant to Section 13(a) or 15(d) of the Exchange Act are available free of charge on our website as soon as reasonably practicable after electronically filing such reports with the SEC.
We make available free of charge on our website our Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, and amendments to those reports, and other filings pursuant to Section 13(a) or 15(d) of the Exchange Act, as soon as reasonably practicable after each is electronically filed with, or furnished to, the SEC.
The Company retains all rights to mavorixafor outside the Territory and specific reserved rights within the Territory. Norgine may grant sublicenses to its affiliates and certain third parties subject to the terms of the agreement, except that it may not sublicense the commercial rights granted under the agreement for certain countries without our explicit consent.
Norgine may grant sublicenses to its affiliates and certain third parties subject to the terms of the agreement, except that it may not sublicense the commercial rights granted under the agreement for certain countries without our explicit consent.
We also incur royalties for certain sublicense fees that we earn from sublicensees of intellectual property (“IP”) that we license from Genzyme. For example, in January 2025 we entered into an exclusive licensing and supply agreement with Norgine whereby we sublicensed intellectual property, including IP that we license from Genzyme.
Sublicense Fees: We also incur fees for certain sublicense revenue that we earn from sublicensees of intellectual property (“IP”) that we license from Genzyme. For example, in January 2025 we entered into the Norgine Agreement whereby we sublicensed IP, including IP that we license from Genzyme.
The Phase 3 trial is a global, randomized, double-blinded, placebo-controlled trial assessing the safety and efficacy of once-daily oral mavorixafor, with or without stable doses of G-CSF, in people with idiopathic or congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections.
The 4WARD trial is a global, randomized, double-blinded, placebo-controlled trial assessing the safety and efficacy of once-daily oral mavorixafor, as a monotherapy or in combination with stable doses of G-CSF, in patients with idiopathic or congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections.
We may terminate the Genzyme Agreement immediately upon notice to Genzyme if we reasonably believe that the development or commercialization of a licensed compound or product under the Genzyme agreement would result in a material safety issue for patients.
We may terminate the Genzyme Agreement immediately upon notice to Genzyme if we reasonably believe that the development or commercialization of a licensed compound or product under the Genzyme Agreement would result in a material safety issue for patients. Norgine Agreement On January 13, 2025, we entered into a license and supply agreement with Norgine.
Expedited Development and Review Programs The FDA has a Fast Track program that is intended to expedite or facilitate the process for development and review of new drugs that meet certain criteria.
Expedited Development and Review Programs The FDA offers a number of expedited development and review programs for qualifying product candidates. The fast track program is intended to expedite or facilitate the process for reviewing new products that meet certain criteria.
The Phase 2 study results below are from the two study treatment groups: mavorixafor monotherapy (n = 10 at baseline) and mavorixafor in combination with injectable G-CSF (n=13 at baseline). • Mavorixafor monotherapy : Consistent with previously presented analyses, results from participants receiving mavorixafor monotherapy showed that mavorixafor durably increased mean ANC from baseline, with mean ANC reaching normal levels at Month 3 (n=9) and Month 6 (n=8). • Further analysis showed that those with severe CN achieved clinically meaningful increases in mean ANC levels out to six months (n=4), reaching levels typically targeted by physicians for patients with severe CN. • Mavorixafor in combination with injectable G-CSF : In the study, physicians chose to reduce G-CSF dosing in nine of 12 (75%) eligible participants.
The Phase 2 study results below are from the two study treatment groups: mavorixafor monotherapy (n = 10 at baseline) and mavorixafor in combination with injectable G-CSF (n=13 at baseline). • Mavorixafor monotherapy : Consistent with previously presented analyses, results from patients receiving mavorixafor monotherapy showed that mavorixafor durably increased mean ANC from baseline, with mean ANC reaching normal levels at Month 3 (n=9) and maintained to the end of study (Month 6 (n=8)). • Mavorixafor in combination with injectable G-CSF : In the study, physicians chose to reduce G-CSF dosing in nine of 12 (75%) eligible patients.
Following these positive results, an amendment to the Phase 1b clinical trial was initiated aiming to evaluate the use of daily oral mavorixafor with or without injectable G-CSF for up to 6 months in participants with chronic neutropenic disorders as a Phase 2 clinical trial.
Phase 2. An amendment to the Phase 1b clinical trial was initiated as a Phase 2 clinical trial to evaluate the use of daily oral mavorixafor with or without injectable G-CSF for up to six months in patients with CN disorders.
(“Catalent”), which is our sole manufacturer for the final capsule drug product formulation of mavorixafor. The term of the master services agreement with Catalent expires on December 31, 2028 and may be terminated by (1) us upon 30 days-notice to Catalent or (2) by either party following a material breach by the other party that remains uncured for 30 days.
The term of the master services agreement with Catalent expires on December 31, 2028 and may be terminated by (i) us upon 30 days-notice to Catalent or (ii) by either party following a material breach by the other party that remains uncured for 30 days.
A certification that an ANDA or 505(b)(2) NDA will not infringe the already approved product’s listed patents, or that such patents are invalid, is called a Paragraph IV certification.
A certification that the new product will not infringe the already approved product’s listed patents or that such patents are invalid or unenforceable is called a Paragraph IV certification.
The term of the agreement shall be automatically renewed for additional three year terms unless either party provides the other party written notice of its intent not to renew the Agreement at least one year prior to the applicable termination date of the agreement. In the event of automatic renewal, the royalty payment rate drops to a single digit royalty.
The term of the Norgine Agreement will be automatically renewed for additional three-year terms unless either party provides the other party with written notice of its intent not to renew the agreement at least one year prior to the applicable termination date of the agreement.
Clinical Trial Results to Date for Mavorixafor in Chronic Neutropenic Disorders In 2022, we conducted a proof-of-concept Phase 1b open-label, multicenter study designed to assess the safety and tolerability of oral mavorixafor, with or without injectable G-CSF, in participants with chronic neutropenic disorders, including idiopathic, cyclic, and congenital neutropenia.
In 2022, we conducted a proof-of-concept Phase 1b open-label, multicenter study designed to assess the safety and tolerability of oral mavorixafor, with or without injectable G-CSF, in patients with CN disorders, including idiopathic, cyclic, and congenital neutropenia. Patients received a single dose of 400 mg oral mavorixafor to assess the magnitude of treatment response.
In addition, under the Pediatric Research Equity Act (“PREA”), certain NDAs or NDA supplements must contain data to assess the safety and effectiveness of the drug for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the drug is safe and effective, unless the sponsor has received a deferral or waiver from the FDA.
In addition, under the Pediatric Research Equity Act (“PREA”), an NDA or supplement to an NDA must contain data to assess the safety and effectiveness of the biological product candidate 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.
Third-party payors are increasingly challenging the price and examining the medical necessity and cost-effectiveness of medical products and services, in addition to their safety and efficacy.
Third-party payors are increasingly challenging the prices charged for medical products and services, examining the medical necessity and reviewing the cost effectiveness of pharmaceutical or biological products, medical devices and medical services, in addition to questioning safety and efficacy.
The 52-week trial is expected to enroll 150 participants aged 12 years and older with both an ANC less than 1,500 cells per microliter and 2 or more infections requiring intervention during the 12 months preceding the trial.
The 52-week trial is expected to enroll 176 patients aged 12 years and older. Key study inclusion criteria are an ANC at baseline of less than 1,000 cells per microliter and 2 or more infections requiring intervention during the 12 months preceding the trial.
Accelerated assessment might be granted by the CHMP in exceptional cases, when a medicinal product is expected to be of major public health interest, particularly from the point of view of therapeutic innovation.
Accelerated evaluation might be granted by the CHMP in exceptional cases, when a medicinal product is expected to be of a major public health interest, particularly from the point of view of therapeutic innovation. Upon request, the CHMP can reduce the time frame to 150 days if the applicant provides sufficient justification for an accelerated assessment.
We must also make one-time sales milestone payments of $0.5 million, $1.5 million and $3.0 million upon achieving cumulative net sales of $50 million, $150 million and $300 million, respectively.
The remaining regulatory milestone of $5.0 million is triggered upon the notification by the EMA of regulatory approval of our first drug application. Sales Milestones: We must also make one-time sales milestone payments of $0.5 million, $1.5 million and $3.0 million upon achieving cumulative net sales of $50 million, $150 million and $300 million, respectively.
In April 2024, we received FDA approval of mavorixafor, which is being marketed in the U.S. under the trade name XOLREMDI, for use as an oral, once-daily therapy in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes.
XOLREMDI® (mavorixafor) for WHIM syndrome We have one commercially approved product, XOLREMDI® (mavorixafor), which has received accelerated approval in the United States from the FDA for use as an oral, once-daily therapy in patients 12 years of age and older with WHIM syndrome, to increase the number of circulating mature neutrophils and lymphocytes.
We currently have a master services agreement, as amended from time to time, and a commercial supply agreement with Evotec A.G. (“Evotec,” previously known as Aptuit, Oxford), pursuant to which Evotec manufactures the active pharmaceutical ingredient (“API”) mavorixafor for use in our clinical and commercial supply. We also have a master services agreement in place with Catalent Inc.
(“Evotec,” previously known as Aptuit, Oxford), pursuant to which Evotec manufactures the API mavorixafor for use in our clinical and commercial supply. We also have a master services agreement in place with Catalent Inc. (“Catalent”), which is our sole manufacturer for the final capsule drug product formulation of mavorixafor.
These results demonstrated that the mean percentage of functional circulating neutrophils in CN participants in this sub-study was comparable to that of healthy donors after six months of mavorixafor dosing. This was the first clinical demonstration that the neutrophils mobilized by mavorixafor are fully functional.
These results demonstrated that the mean percentage of functional circulating neutrophils in CN patients in this sub-study was comparable to that of healthy donors after six months of mavorixafor dosing. Phase 3 (4WARD). The 4WARD study is a Phase 3 clinical trial of mavorixafor in patients with certain CN disorders.
To obtain reimbursement or pricing approval, some of these countries may require the completion of clinical trials that compare the cost-effectiveness of a particular product candidate to currently available therapies. Other member states allow companies to fix their own prices for medicines but monitor and control company profits.
To obtain reimbursement or pricing approval, some of these countries may require the completion of clinical trials that compare the cost effectiveness of a particular product candidate to currently available therapies.
As a result, following successful completion of a Phase 2 study evaluating mavorixafor in people with certain chronic neutropenic disorders, we have initiated a pivotal Phase 3 clinical trial of mavorixafor (the “4WARD” study) to evaluate the efficacy, safety, and tolerability of oral, once-daily mavorixafor with or without human granulocyte colony-stimulating factor (“G-CSF”) in people with congenital or acquired primary autoimmune and idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections.
We are focused on the continued progress of our global, pivotal Phase 3 clinical trial (the “4WARD” trial) to evaluate the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without stable doses of granulocyte colony-stimulating factor (“G-CSF”)) in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections.
Orphan designation must be requested before submitting an application for an MA. Orphan designation does not convey any advantage in, or shorten the duration of, the regulatory review and approval process.
Orphan drug designation does not convey any advantage in, or shorten the duration of, the regulatory review and approval process. Designated orphan medicines are eligible for conditional MA.
An ANDA applicant, or a 505(b)(2) NDA applicant that is relying on FDA’s finding of safety and effectiveness for a previously approved drug, is required to certify to the FDA concerning any patents listed for the approved product in the FDA’s Orange Book.
To the extent that a Section 505(b)(2) applicant is relying on studies conducted for an already approved product, the applicant is required to certify to the FDA concerning any patents listed for the approved product in the Orange Book to the same extent that an ANDA applicant would.
The primary endpoint of the trial is a two-component endpoint that includes the annualized infection rate and ANC response in the mavorixafor-treated group versus the placebo group. Key secondary endpoints are expected to include analysis of the severity and duration of infections, antibiotic use, fatigue, and quality of life parameters.
The trial is designed to test for the co-primary endpoints of reduction in annualized infection rate and ANC response, defined as an increase in >500 cells per microliter in the mavorixafor-treated group versus the placebo group. Key secondary endpoints include analysis of the severity and duration of infections, antibiotic use, fatigue, and quality of life parameters.
The filing of a patent infringement lawsuit within 45 days of the receipt of a Paragraph IV notice automatically prevents the FDA from approving the ANDA or 505(b)(2) NDA until the earlier of 30 months, expiration of the patent, settlement of the lawsuit, or a decision in the infringement case that is favorable to the applicant.
The filing of a patent infringement lawsuit within 45 days after the receipt of a Paragraph IV certification automatically prevents the FDA from approving the ANDA until the earlier of 30 months, expiration of the patent, settlement of the lawsuit or a decision in the infringement case that is favorable to the ANDA applicant. 505(b)(2) New Drug Applications As an alternative path to FDA approval for modifications to formulations or uses of products previously approved by the FDA pursuant to an NDA, an applicant may submit an NDA under Section 505(b)(2) of the FDCA.
In January 2025, we achieved a regulatory milestone of $3.0 million, which was triggered upon notification of the acceptance by the EMA of our first drug application. Such regulatory milestone was paid in March 2025. The remaining regulatory milestone of $5.0 million is triggered upon the notification by the EMA of regulatory approval of our first drug application.
Regulatory Milestones : The Genzyme Agreement includes certain regulatory milestones that require us to make payments upon achievement. In January 2025, we achieved a regulatory milestone upon notification of the acceptance by the EMA of our first drug application, which triggered a payment of $3.0 million. Such payment was made in March 2025.
Such differences in national pricing regimes may create price differentials between E.U. member states. There can be no assurance that any country that has price controls or reimbursement limitations for pharmaceutical products will allow favorable reimbursement and pricing arrangements for any of our products. Historically, products launched in the European Union do not follow price structures of the United States.
There can be no assurance that any country that has price controls or reimbursement limitations for pharmaceutical products will allow favorable reimbursement and pricing arrangements for any of our product candidates. Historically, products launched in the EU do not follow price structures of the U.S. and generally prices tend to be significantly lower.
Food and Drug Administration (“FDA”) approved mavorixafor, which is being marketed in the U.S. under the trade name XOLREMDI®, for use as an oral, once-daily therapy in patients 12 years of age and older with WHIM syndrome (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes.
We have one commercially approved product, XOLREMDI® (mavorixafor), which has received accelerated approval in the United States from the FDA for use as an oral, once-daily therapy in patients 12 years of age and older with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, to increase the number of circulating mature neutrophils and lymphocytes.
Moreover, agreements with physicians often must be the subject of prior notification and approval by the physician’s employer, his or her competent professional organization as well as the regulatory authorities of the individual EU Member States. These requirements are provided in the national laws, industry codes or professional codes of conduct, applicable in the EU Member States.
Violation of these laws could result in substantial fines and imprisonment. Payments made to physicians in certain EU Member States also must be publicly disclosed. Moreover, agreements with physicians must often be the subject of prior notification and approval by the physician’s employer, his/her regulatory professional organization, and/or the competent authorities of the individual EU Member States.
Norgine will be responsible for all market access and commercialization activities and will eventually hold all marketing authorizations in the licensed territories. We will manufacture and supply mavorixafor to Norgine. Norgine shall be required to pay a supply price for the licensed product derived from the contract manufacturing organization costs plus a low double-teen digit of the contract manufacturing costs.
Norgine will be required to pay a supply price for the Licensed Product derived from the contract manufacturing organization costs plus a low double-teen digit percentage of the contract manufacturing costs.
The extension period can be shortened if the FDA determines that the applicant did not pursue approval with due diligence. The total patent term after the extension may not exceed 14 years. For patents that might expire during the application phase, the patent owner may request an interim patent extension.
The time can be shortened if the FDA determines that the applicant did not pursue licensure with due diligence. The total patent term after the extension may not exceed 14 years from the date of product approval.
In February 2025, we announced that we had entered into an agreement with Taiba Middle East Fz LLC (“taiba rare”) to distribute and commercialize XOLREMDI for the treatment of WHIM syndrome in select Middle East and North African countries including Saudi Arabia, United Arab Emirates, Qatar, Oman, Kuwait, Bahrain, and Egypt following any regulatory approvals in those countries.
In February 2025, we announced that we had entered into an agreement with Taiba Middle East Fz LLC (“taiba rare”) to distribute and commercialize XOLREMDI for the treatment of WHIM syndrome in select Middle East and North African (“MENA”) countries. We also have a license agreement with Abbisko Therapeutics Co. Ltd.
There is no guarantee that a product will be considered by the EMA to be an innovative medicinal product, and products may not qualify for data exclusivity.
Products may not be granted data exclusivity since there is no guarantee that a product will be considered by the EU’s regulatory authorities to include a NCE.
Norgine Agreement On January 13, 2025, we entered into a license and supply agreement with Norgine. Under the terms of the agreement, we granted Norgine the exclusive right to develop, manufacture and commercialize mavorixafor in Europe, Australia, and New Zealand.
Under the terms of the Norgine Agreement, we granted Norgine the exclusive right to conduct regulatory activities on, manufacture and commercialize mavorixafor (the “Licensed Product”) in Europe, Australia, and New Zealand (collectively, the “Territory”).
Even if a product is considered to be an innovative medicinal product so that the innovator gains the prescribed period of data exclusivity, another company could nevertheless also market another version of the product if such company obtained an MA based on an MA application with a complete and independent data package of pharmaceutical tests, preclinical tests and clinical trials.
Even if a compound is considered to be a NCE and the MA applicant is able to gain the prescribed period of data exclusivity, another company nevertheless could also market another version of the medicinal product if such company can complete a full MAA with their own complete database of pharmaceutical tests, preclinical studies and clinical trials and obtain MA of its product.
Failure to comply with these requirements could result in reputational risk, public reprimands, administrative penalties, fines or imprisonment.
These requirements are provided in the national laws, industry codes, or professional codes of conduct, applicable in the individual EU Member States. Failure to comply with these requirements could result in reputational risk, public reprimands, administrative penalties, fines or imprisonment.
We supplement base cash employee compensation with awards of stock options and/or restricted stock units under our equity incentive plans. We review employee performance annually and our Compensation Committee approves associated merit increases and annual incentive bonus payments during the first quarter of the year annually.
We review employee performance annually and our Compensation Committee approves associated merit increases and annual incentive bonus payments during the first quarter of the year annually. When needed, we augment our employee base with outside consultants who specialize in various fields.
Such scrutiny has resulted in several Presidential executive orders, Congressional hearings and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for products. Congress has also passed additional reform measures.
Moreover, there has recently been heightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which has resulted in several Congressional inquiries and proposed and enacted federal and state measures designed to, among other things, reduce the cost of prescription drugs, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drug products.
The completed Phase 2 study of mavorixafor was a six-month, open-label clinical trial that enrolled a total of 23 participants diagnosed with idiopathic, congenital, or cyclic chronic neutropenia.
For patients being treated with mavorixafor in combination with G-CSF, physicians had the option to reduce the dose of G-CSF starting at Month 3. The completed Phase 2 study of mavorixafor was a six-month, open-label clinical trial that enrolled a total of 23 patients diagnosed 7 with idiopathic, congenital, or cyclic CN.
In addition, in some foreign countries, the proposed pricing for a drug must be approved before it may be lawfully marketed. The requirements governing drug pricing vary widely from country to country. For example, some E.U. jurisdictions operate positive and negative list systems under which products may only be marketed once a reimbursement price has been agreed.
Finally, in some foreign countries, the proposed pricing for a drug must be approved before it may be lawfully marketed. The requirements governing drug pricing vary widely from country to country.
Pharmaceutical Coverage, Pricing and Reimbursement Sales of pharmaceutical products in the United States will depend, in part, on the extent to which the costs of the products will be covered by third-party payors, such as government health programs, and commercial insurance and managed health care organizations. These third-party payors are increasingly challenging the prices charged for medical products and services.
Sales of any product, if approved, depend, in part, on the extent to which such product will be covered by third-party payors, such as federal, state, and foreign government healthcare programs, commercial insurance and managed healthcare organizations, and the level of reimbursement, if any, for such product by third-party payors.
The allowable patent term extension generally is calculated based on half of the drug’s testing phase—the time between IND clearance and NDA submission—and all of the review phase—the time between NDA submission and approval up to a maximum of five years.
The allowable patent term extension is typically calculated as one-half the time between, the latter of the effective date of an IND and issue date of the patent for which extension is sought, and the submission date of an NDA, plus the time between NDA submission date and the NDA approval date up to a maximum of five years.
Patent Term Extension After NDA approval, owners of relevant drug patents may apply for up to a five-year patent extension.
Patent Term Extension In the United States, after an NDA is approved, owners of relevant drug patents may apply for up to a five-year patent extension, which permits patent term restoration as compensation for the patent term lost during the FDA regulatory process.
If our operations are found to be in violation of any of these laws or any other governmental regulations that may apply to it, we may be subject to significant civil, criminal and administrative penalties, damages, fines, imprisonment, exclusion from government funded healthcare programs, such as Medicare and Medicaid, and the curtailment or restructuring of our operations.
If our operations are found to be in violation of any of such laws or any other governmental regulations that apply, we may be subject to penalties, including, without limitation, civil, criminal and administrative penalties, damages, fines, exclusion from government-funded healthcare programs, such as Medicare and Medicaid or similar programs in other countries or jurisdictions, integrity oversight and reporting obligations to resolve allegations of non-compliance, disgorgement, individual imprisonment, contractual damages, reputational harm, diminished profits and the curtailment or restructuring of our operations. 19 Data Privacy and Security Numerous state, federal, and foreign laws govern the collection, dissemination, use, access to, confidentiality, and security of personal information, including health-related information.
The agreement grants Norgine an exclusive license to (i) distribute, market and sell the our product mavorixafor for all indications in the European Economic Area, Switzerland, the United Kingdom, Australia and New Zealand (collectively, the “Territory”), following regulatory approval. Additionally, Norgine was granted a co-exclusive license to manufacture mavorixafor for the Territory within the field.
On January 13, 2025, we announced a license and supply agreement (the “Norgine Agreement”) with Norgine, pursuant to which Norgine was granted an exclusive license to distribute, market and sell our drug product for all indications, including WHIM and CN, in the European Economic Area, Switzerland, the United Kingdom, Australia, and New Zealand.
The centralized procedure is optional for products containing a new active substance not yet authorized in the EU, or for products that constitute a significant therapeutic, scientific, or technical innovation or which are in the interest of public health in the EU.
For medicinal products containing a new active substance not yet authorized in the EEA before May 20, 2004 and indicated for the treatment of other diseases, medicinal products that constitute significant therapeutic, scientific or technical innovations or for which the grant of a MA through the centralized procedure would be in the interest of public health at EU level, an applicant may voluntarily submit an application for a MA through the centralized procedure.
The FDA is not bound by the recommendation of an advisory committee, but it generally follows such recommendations. Before approving an NDA, the FDA will typically inspect one or more clinical sites to assure compliance with GCP. Additionally, the FDA will inspect the facility or the facilities at which the drug is manufactured to assess compliance with cGMP.
The FDA is not bound by the recommendations of an advisory committee, but it considers such recommendations carefully when making decisions. Before approving an NDA, the FDA will typically inspect the facility or facilities where the product is manufactured.
The overall ten-year period will be extended to a maximum of eleven years if, during the first eight years of those ten years, the MA holder obtains an MA for one or more new therapeutic indications which, during the scientific evaluation prior to their authorization, are determined to bring a significant clinical benefit in comparison with currently approved therapies.
An additional non-cumulative one-year period of marketing exclusivity is possible if during the data exclusivity period (the first eight years of the 10-year marketing exclusivity period), the MA holder obtains an authorization for one or more new therapeutic indications that are deemed to bring a significant clinical benefit compared to existing therapies.
The IRA also eliminated the “donut hole” under the Medicare Part D program (effective January 1, 2025) by capping the beneficiary maximum out-of-pocket cost at $2,000 per year and creating a new manufacturer discount program. The ACA continues to be subject to judicial or challenges and may be subject to legislative healthcare reform measures in the future.
The IRA also eliminates the “donut hole” under the Medicare Part D program beginning in 2025 by significantly lowering the beneficiary maximum out-of-pocket cost and creating a new manufacturer discount program.
A product can be designated as a Breakthrough Therapy by FDA if it is intended to treat a serious or life-threatening condition and preliminary clinical evidence indicates that it may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.
A product can receive breakthrough therapy designation if preliminary clinical evidence indicates that the product, alone or in combination with one or more other drugs or biologics, may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
Failure to comply with applicable requirements may subject a company to a variety of administrative or judicial sanctions, such as imposition of clinical holds, refusal by the FDA to approve pending NDAs, warning letters, product recalls, product seizures, total or partial suspension of production or distribution, injunctions, fines, refusals of government contracts, restitution, disgorgement, civil penalties and criminal prosecution.
These sanctions could include, among other actions, the FDA’s refusal to approve pending applications from the sponsor, withdrawal of an approval, a clinical hold, untitled or warning letters, product recalls or market withdrawals, product seizures, total or partial suspension of production or distribution, injunctions, fines, refusals of government contracts, restitution, disgorgement and civil or criminal penalties.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeOur business could be adversely affected by economic downturns, inflation, increases in interest rates, natural disasters, public health crises, political crises, geopolitical events, such as the war in Ukraine and in Gaza, or other macroeconomic conditions, which have in the past and may in the future negatively impact our business and financial performance. 58 The global economy, including credit and financial markets, has experienced extreme volatility and disruptions, including, among other things, severely diminished liquidity and credit availability, declines in consumer confidence, declines in economic growth, supply chain shortages, increases in inflation rates, higher interest rates and uncertainty about economic stability.
Biggest changeFurther, the global economy, including credit and financial markets, outside of recent tariffs, has experienced extreme volatility and disruptions, including, among other things, severely diminished liquidity and credit availability, declines in consumer 48 confidence, declines in economic growth, supply chain shortages, increases in inflation rates, higher interest rates and uncertainty about economic stability.
We are currently dependent on a single third-party manufacturer for the manufacture of mavorixafor, the active pharmaceutical ingredient (“API”) and a single manufacturer of mavorixafor finished drug product capsules.
We are currently dependent on a single third-party manufacturer for the manufacture of the active pharmaceutical ingredient (“API”) for mavorixafor, and a single manufacturer of mavorixafor finished drug product capsules.
Our future funding requirements, both near and long-term, will depend on many factors, including, but not limited to: • the outcome, timing and cost of regulatory reviews, approvals or other actions to meet regulatory requirements established by the FDA and comparable foreign regulatory authorities, including the potential for the FDA or comparable foreign regulatory authorities to require that we perform more studies for our product candidates than those that we currently expect; • our ability to obtain marketing approval for our product candidates, including for additional indications; • the success of our exclusive licensing and supply agreement which Norgine and any potential regulatory and commercial milestone payments that we may receive under that agreement; • the scope, number, initiation, progress, timing, costs, design, duration, any potential delays, and results of clinical trials and nonclinical studies for our current or future product candidates; • the number and characteristics of product candidates and programs that we develop or may in-license; • the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights covering our product candidates, including any such patent claims and intellectual property rights that we have licensed from Genzyme pursuant to the terms of our license agreement with Genzyme or from other third parties; • our ability to maintain, expand and defend the scope of our intellectual property portfolio, including the cost of defending intellectual property disputes, including patent infringement actions brought by third parties against us or our product candidates; • the cost and timing of completion of commercial-scale manufacturing activities with respect to our product candidates; • our ability to establish and maintain licensing, collaboration or similar arrangements on favorable terms and whether and to what extent we retain development or commercialization responsibilities under any new licensing, collaboration or similar arrangement; • the cost of establishing sales, marketing and distribution capabilities for any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products on our own; • the success of any other business, product or technology that we acquire or in which we invest; • the costs of acquiring, licensing or investing in businesses, product candidates and technologies; • our need and ability to hire additional management and scientific and medical personnel; • market acceptance of our product candidates, to the extent any are approved for commercial sale; • the effect of competing technological and market developments; • the costs to operate as a public company; and • business interruptions resulting from pandemics and public health emergencies, geopolitical actions, including war and terrorism or natural disasters including earthquakes, typhoons, floods and fires.
Our future funding requirements, both near and long-term, will depend on many factors, including, but not limited to: • the outcome, timing and cost of regulatory reviews, approvals or other actions to meet regulatory requirements established by the FDA and comparable foreign regulatory authorities, including the potential for the FDA or comparable foreign regulatory authorities to require that we perform more studies for our product candidates than those that we currently expect; • our ability to obtain marketing approval for our product candidates, including for additional indications; • the success of our exclusive licensing and supply agreement which Norgine and any potential regulatory and commercial milestone payments that we may receive under that agreement; • the scope, number, initiation, progress, timing, costs, design, duration, any potential delays, and results of clinical trials and nonclinical studies for our current or future product candidates; • the number and characteristics of product candidates and programs that we develop or may in-license; • the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights covering our product candidates, including any such patent claims and intellectual property rights that we have licensed from Genzyme pursuant to the terms of our license agreement with Genzyme or from other third parties; 37 • our ability to maintain, expand and defend the scope of our intellectual property portfolio, including the cost of defending intellectual property disputes, including patent infringement actions brought by third parties against us or our product candidates; • the cost and timing of completion of commercial-scale manufacturing activities with respect to our product candidates; • our ability to establish and maintain licensing, collaboration or similar arrangements on favorable terms and whether and to what extent we retain development or commercialization responsibilities under any new licensing, collaboration or similar arrangement; • the cost of establishing sales, marketing and distribution capabilities for any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products on our own; • the success of any other business, product or technology that we acquire or in which we invest; • the costs of acquiring, licensing or investing in businesses, product candidates and technologies; • our need and ability to hire additional management and scientific and medical personnel; • market acceptance of our product candidates, to the extent any are approved for commercial sale; • the effect of competing technological and market developments; • the costs to operate as a public company; and • business interruptions resulting from pandemics and public health emergencies, geopolitical actions, including war and terrorism or natural disasters including earthquakes, typhoons, floods and fires.
For XOLREMDI and any other product candidates that receive marketing approval in the future, if we or others identify undesirable side effects caused by such product candidates (or any other similar drugs) after such approval, a number of potentially significant negative consequences could result, including: • regulatory authorities may withdraw or limit their approval of such product candidates; • regulatory authorities may require the addition of labeling statements, such as a “boxed” warning or a contraindication; • we may be required to create a medication guide outlining the risks of such side effects for distribution to patients; • we may be required to change the way such product candidates are distributed or administered, conduct additional clinical trials or change the labeling of the product candidates; • regulatory authorities may require a Risk Evaluation and Mitigation Strategy to mitigate risks, which could include medication guides, physician communication plans, or elements to assure safe use, such as restricted distribution methods, patient registries and other risk minimization tools; • we may be subject to regulatory investigations and government enforcement actions; • we may decide to remove such product candidates from the marketplace after they are approved; • we could be sued and held liable for injury caused to individuals exposed to or taking our product candidates; and • our reputation may suffer.
For XOLREMDI and any other product candidates that receive marketing approval in the future, if we 43 or others identify undesirable side effects caused by such product candidates (or any other similar drugs) after such approval, a number of potentially significant negative consequences could result, including: • regulatory authorities may withdraw or limit their approval of such product candidates; • regulatory authorities may require the addition of labeling statements, such as a “boxed” warning or a contraindication; • we may be required to create a medication guide outlining the risks of such side effects for distribution to patients; • we may be required to change the way such product candidates are distributed or administered, conduct additional clinical trials or change the labeling of the product candidates; • regulatory authorities may require a Risk Evaluation and Mitigation Strategy to mitigate risks, which could include medication guides, physician communication plans, or elements to assure safe use, such as restricted distribution methods, patient registries and other risk minimization tools; • we may be subject to regulatory investigations and government enforcement actions; • we may decide to remove such product candidates from the marketplace after they are approved; • we could be sued and held liable for injury caused to individuals exposed to or taking our product candidates; and • our reputation may suffer.
Market acceptance of any of our approved product or product candidates for which we receive approval in the future depends on a number of factors, including: • the efficacy and safety of such product candidates as demonstrated in clinical trials; • the clinical indications for which the product candidate is approved; • acceptance by hospitals, physicians and patients of the product candidate as a safe and effective treatment, particularly the ability of mavorixafor and our other product candidates to establish themselves as a new standard of care for the indications that we are pursuing; • the potential and perceived advantages of our products and product candidates over alternative treatments as compared to their relative costs; • the prevalence and severity of any side effects with respect to our products or product candidates, including mavorixafor; • our ability to offer any approved products for sale at competitive prices; • the timing of market introduction of our products as well as competitive products; • our pricing, and the availability of coverage and adequate reimbursement by third party payors and government authorities; • relative convenience and ease of administration; and • the effectiveness of our sales and marketing efforts and those of our potential future collaborators.
Market acceptance of any of our approved product or product candidates for which we receive approval in the future, if any, depends on a number of factors, including: • the efficacy and safety of such product candidates as demonstrated in clinical trials; • the clinical indications for which the product candidate is approved; • acceptance by hospitals, physicians and patients of the product candidate as a safe and effective treatment, particularly the ability of mavorixafor and our other product candidates to establish themselves as a new standard of care for the indications that we are pursuing; • the potential and perceived advantages of our products and product candidates over alternative treatments as compared to their relative costs; • the prevalence and severity of any side effects with respect to our products or product candidates, including mavorixafor; • our ability to offer any approved products for sale at competitive prices; • the timing of market introduction of our products as well as competitive products; • our pricing, and the availability of coverage and adequate reimbursement by third party payors and government authorities; • relative convenience and ease of administration; and • the effectiveness of our sales and marketing efforts and those of our potential future collaborators.
If we, our product or product candidates or the manufacturing facilities for our product or product candidates fail to comply with cGMPs and other applicable regulatory requirements, the FDA may, among other things: • issue warning letters; • request modifications to promotional materials or require us to provide corrective information to healthcare practitioners; • require us to enter into a consent decree, which can include imposition of various fines, reimbursements for inspection costs, required due dates for specific actions and penalties for noncompliance; • seek an injunction or impose civil or criminal penalties or monetary fines; • suspend or withdraw regulatory approval; • suspend any ongoing clinical trials; • refuse to approve pending applications or supplements to applications filed by us; • suspend or impose restrictions on operations, including costly new manufacturing requirements; or • seize or detain products, refuse to permit the import or export of products, or require us to initiate a product recall.
If we, our product or product candidates or the manufacturing facilities for our product or product candidates fail to comply with cGMPs and other applicable regulatory requirements, the FDA may, among other things: • issue warning letters; • request modifications to promotional materials or require us to provide corrective information to healthcare practitioners; • require us to enter into a consent decree, which can include imposition of various fines, reimbursements for inspection costs, required due dates for specific actions and penalties for noncompliance; • seek an injunction or impose civil or criminal penalties or monetary fines; • suspend or withdraw regulatory approval; • suspend any ongoing clinical trials; 45 • refuse to approve pending applications or supplements to applications filed by us; • suspend or impose restrictions on operations, including costly new manufacturing requirements; or • seize or detain products, refuse to permit the import or export of products, or require us to initiate a product recall.
To the extent that any disruption or security breach were to result in a loss of, or damage to, our data or applications, or inappropriate disclosure of confidential or proprietary information our development and commercialization efforts could be delayed and we could be required to notify impacted stakeholders (including affected individuals, regulators and investors) what could lead to significant liability through litigation and regulatory investigations and enforcement actions, including under state (e.g., state breach notification and consumer protection laws), federal (e.g., HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (“HITECH”)), and international law (e.g., the GDPR).
To the extent that any disruption or security breach were to 62 result in a loss of, or damage to, our data or applications, or inappropriate disclosure of confidential or proprietary information our development and commercialization efforts could be delayed and we could be required to notify impacted stakeholders (including affected individuals, regulators and investors) what could lead to significant liability through litigation and regulatory investigations and enforcement actions, including under state (e.g., state breach notification and consumer protection laws), federal (e.g., HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009 (“HITECH”)), and international law (e.g., the GDPR).
Before obtaining regulatory approvals for the commercial sale of any product candidate, we must successfully meet a number of critical developmental milestones, including: 34 • developing dosages that will be well-tolerated, safe and effective; • completing the development and scale-up to permit manufacture of our product candidates in commercial quantities and at acceptable costs; • demonstrating through pivotal clinical trials that each product candidate is safe and effective in patients for the intended indication; • establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers; and • obtaining and maintaining patent and trade secret protection and non-patent exclusivity for our product candidates.
Before obtaining regulatory approvals for the commercial sale of any product candidate, we must successfully meet a number of critical developmental milestones, including: • developing dosages that will be well-tolerated, safe and effective; • completing the development and scale-up to permit manufacture of our product candidates in commercial quantities and at acceptable costs; • demonstrating through pivotal clinical trials that each product candidate is safe and effective in patients for the intended indication; • establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers; and • obtaining and maintaining patent and trade secret protection and non-patent exclusivity for our product candidates.
Moreover, disputes may arise regarding intellectual property subject to a licensing agreement, including: • the scope of rights granted under the license agreement and other interpretation-related issues; • the extent to which our product candidates, technology and processes infringe on intellectual property of the licensor that is not subject to the licensing agreement; • the sublicensing of patent and other rights under our collaborative development relationships; • our diligence obligations under the license agreement and what activities satisfy those diligence obligations; • the inventorship and ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners; and • the priority of invention of patented technology.
Moreover, disputes may arise regarding intellectual property subject to a licensing agreement, including: • the scope of rights granted under the license agreement and other interpretation-related issues; • the extent to which our product candidates, technology and processes infringe on intellectual property of the licensor that is not subject to the licensing agreement; • the sublicensing of patent and other rights under our collaborative development relationships; • our diligence obligations under the license agreement and what activities satisfy those diligence obligations; • the inventorship and ownership of inventions and know-how resulting from the joint creation or use of intellectual 58 property by our licensors and us and our partners; and • the priority of invention of patented technology.
Those factors may include the design or results of clinical trials, the likelihood of approval by the FDA or similar regulatory authorities outside the United States, the potential market for the subject product candidate, the costs and complexities of manufacturing and delivering such product candidate to patients, the potential of competing products, the existence of uncertainty with respect to our ownership of technology, which can exist if there is a challenge to such ownership without regard to the merits of the challenge and industry and market conditions generally.
Those factors may include the design or results of clinical trials, the likelihood of approval by the FDA or similar regulatory authorities outside the United States, the potential market for the subject product candidate, the costs and complexities of manufacturing and delivering such product candidate to patients, the potential of competing products, the existence of uncertainty with respect to our ownership of technology, which can exist if there is a challenge to such ownership without regard to the merits of the 54 challenge and industry and market conditions generally.
Our certificate of incorporation provides that the Court of Chancery of the State of Delaware is the exclusive forum for any derivative action or proceeding brought on the Company’s behalf, any action asserting a breach of fiduciary duty owed by our directors, officers, other employees or stockholders to the Company or our stockholders, any action asserting a claim against the Company arising pursuant to the Delaware General Corporation Law or as to which the Delaware General Corporation Law confers jurisdiction on the Court of Chancery of the State of Delaware, or any action asserting a claim arising pursuant to our certificate of incorporation or by-laws or governed by the internal affairs doctrine.
Our certificate of incorporation provides that the Court of Chancery of the State of Delaware is the exclusive forum for any derivative action or proceeding brought on the Company’s behalf, any action asserting a breach of fiduciary duty owed by our directors, officers, other employees or stockholders to the Company or our stockholders, any action asserting a claim against the 66 Company arising pursuant to the Delaware General Corporation Law or as to which the Delaware General Corporation Law confers jurisdiction on the Court of Chancery of the State of Delaware, or any action asserting a claim arising pursuant to our certificate of incorporation or by-laws or governed by the internal affairs doctrine.
If the FDA or comparable foreign regulatory authorities become aware of new safety information after approval of any of our product candidates, these regulatory authorities may require labeling changes or the FDA may require establishment of a Risk Evaluation Mitigation Strategy (“REMS”), impose significant restrictions on our product’s indicated uses or marketing, or impose ongoing requirements for potentially costly post-approval 40 studies or post-market surveillance.
If the FDA or comparable foreign regulatory authorities become aware of new safety information after approval of any of our product candidates, these regulatory authorities may require labeling changes or the FDA may require establishment of a Risk Evaluation Mitigation Strategy (“REMS”), impose significant restrictions on our product’s indicated uses or marketing, or impose ongoing requirements for potentially costly post-approval studies or post-market surveillance.
In addition to the possibility of litigation relating to infringement claims asserted against it, we may become a party to other patent litigation and other proceedings, including inter partes review proceedings, post-grant review proceedings, derivation proceedings declared by the USPTO and similar proceedings in foreign countries, regarding intellectual property rights with respect to our current or future technologies or product candidates or products.
In addition to the possibility of litigation relating to infringement claims asserted against it, we may become a party to other patent litigation and other proceedings, including inter partes review proceedings, post-grant review proceedings, derivation proceedings declared by the USPTO and similar proceedings in foreign countries, regarding intellectual property rights with 57 respect to our current or future technologies or product candidates or products.
Any such delays could negatively impact our business, financial condition, results of operations and prospects. Interim top-line and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data.
Any such delays could negatively impact our business, financial condition, results of operations and prospects. 44 Interim top-line and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data.
Even if patents covering our product candidates are obtained, once the patent life has expired, we may be open to competition from competitive products, including generics or biosimilars. Given the amount of time 52 required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized.
Even if patents covering our product candidates are obtained, once the patent life has expired, we may be open to competition from competitive products, including generics or biosimilars. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized.
Information about certain clinical trials, including results (positive or negative) will be made public according to each country’s clinical trial registration policies. Competitors may use this publicly available information to gain knowledge regarding the progress of development programs. 37 Product development involves a lengthy and expensive process, with uncertain outcomes.
Information about certain clinical trials, including results (positive or negative) will be made public according to each country’s clinical trial registration policies. Competitors may use this publicly available information to gain knowledge regarding the progress of development programs. Product development involves a lengthy and expensive process, with uncertain outcomes.
We have limited experience manufacturing pharmaceutical products on a commercial scale and some of these manufacturers will need to increase their scale of production to meet our projected needs for commercial manufacturing, the satisfaction of which may not be met on a timely basis. 48 We rely on third-party CROs to conduct our preclinical studies and clinical trials.
We have limited experience manufacturing pharmaceutical products on a commercial scale and some of these manufacturers will need to increase their scale of production to meet our projected needs for commercial manufacturing, the satisfaction of which may not be met on a timely basis. We rely on third-party CROs to conduct our preclinical studies and clinical trials.
Such litigation, if instituted, could result in substantial costs and diversion of management attention and resources, which could significantly harm our business, financial condition, results of operations and reputation. 60 “Penny stock” rules may make buying or selling our securities difficult which may make our stock less liquid and make it harder for investors to buy and sell our securities.
Such litigation, if instituted, could result in substantial costs and diversion of management attention and resources, which could significantly harm our business, financial condition, results of operations and reputation. “Penny stock” rules may make buying or selling our securities difficult which may make our stock less liquid and make it harder for investors to buy and sell our securities.
We also could be required to: • seek new or additional collaborators for one or more of our current or future product candidates at an earlier stage than otherwise would be desirable or on terms that are less favorable than might otherwise be available; or 31 • relinquish or license on unfavorable terms our rights to technologies or product candidates that we otherwise would seek to develop or commercialize ourselves.
We also could be required to: • seek new or additional collaborators for one or more of our current or future product candidates at an earlier stage than otherwise would be desirable or on terms that are less favorable than might otherwise be available; or • relinquish or license on unfavorable terms our rights to technologies or product candidates that we otherwise would seek to develop or commercialize ourselves.
Our inability to promptly obtain coverage and adequate reimbursement rates from both government-funded and private payors for XOLREMDI (mavorixafor) or for any future approved product candidates could have a material adverse effect on our operating results, our ability to raise capital needed to develop additional product candidates and commercialize products and our overall financial condition.
Our inability to promptly obtain coverage and adequate reimbursement rates from both government-funded and private payors for XOLREMDI or for any future approved product candidates could have a material adverse effect on our operating results, our ability to raise capital needed to develop additional product candidates and commercialize products and our overall financial condition.
We are not aware of any threatened or pending claims related to these matters or concerning the agreements with our senior management, but in the future 55 litigation may be necessary to defend against such claims. If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights or personnel.
We are not aware of any threatened or pending claims related to these matters or concerning the agreements with our senior management, but in the future litigation may be necessary to defend against such claims. If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights or personnel.
If we, any of our CROs or our clinical trial sites fail to comply with applicable GCP requirements, the clinical data generated in our clinical trials or a specific site may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications.
If we, any of our CROs or our clinical trial sites fail to comply with applicable GCP requirements, the clinical data 53 generated in our clinical trials or a specific site may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications.
If we cannot successfully manage the promotion of our products, we could become subject to significant liability, which would materially adversely affect our business and financial condition. 41 Our commercial success depends upon attaining significant market acceptance of our approved product or product candidates, if approved, among hospitals, physicians, patients and healthcare payors.
If we cannot successfully manage the promotion of our products, we could become subject to significant liability, which would materially adversely affect our business and financial condition. Our commercial success depends upon attaining significant market acceptance of our approved product or product candidates, if approved, among hospitals, physicians, patients and healthcare payors.
These products may compete with our products in jurisdictions where we do not have any issued patents and our patent claims or other intellectual property rights may not be effective or sufficient to prevent them from so competing. Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions.
These products may compete with our products in jurisdictions where we do not have any issued patents and our patent claims or other intellectual property rights may not be effective or sufficient to prevent them from so competing. Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign 60 jurisdictions.
If these in-licenses are terminated, or if the underlying patents fail to provide the intended exclusivity, competitors or other third parties may gain the freedom to seek regulatory approval of, and to market, products identical to ours and we may be 53 required to cease our development and commercialization of our product candidates.
If these in-licenses are terminated, or if the underlying patents fail to provide the intended exclusivity, competitors or other third parties may gain the freedom to seek regulatory approval of, and to market, products identical to ours and we may be required to cease our development and commercialization of our product candidates.
To the extent that we raise additional capital through the sale of equity or convertible debt securities, your ownership interest will be diluted, and the terms of 32 these securities may include liquidation or other preferences that adversely affect your rights as a holder of our common stock.
To the extent that we raise additional capital through the sale of equity or convertible debt securities, your ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a holder of our common stock.
If we are unable to complete development for additional indications for mavorixafor or any other product candidates that we may develop, we will not be able to commercialize and earn revenue from them. We may develop mavorixafor, and potentially future product candidates, in combination with other therapies, which could expose us to additional risks.
If we are unable to complete development for additional indications for mavorixafor or any other product candidates that we may develop, we will not be able to commercialize and earn revenue from them. We may develop mavorixafor, and future product candidates, in combination with other therapies, which could expose us to additional risks.
If we do not 42 establish sales and marketing capabilities successfully, either on our own or in collaboration with third parties, we will not be successful in commercializing our product or product candidates. We face substantial competition that may result in others discovering, developing or commercializing products before or more successfully than we do.
If we do not establish sales and marketing capabilities successfully, either on our own or in collaboration with third parties, we will not be successful in commercializing our product or product candidates. We face substantial competition that may result in others discovering, developing or commercializing products before or more successfully than we do.
Any products that we commercialize may become subject to unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, which would harm our business. The laws and regulations that govern marketing approvals, pricing, coverage and reimbursement for new drug products vary widely from country to country.
Any products that we commercialize may become subject to unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, which would harm our business. The laws and regulations that govern marketing approvals, pricing, coverage and reimbursement for new drug products vary 49 widely from country to country.
With respect to patent rights, we do not know whether any of the pending patent applications for any of our products or product candidates will result in the issuance of patents that protect our technology or products, or which will effectively prevent others 51 from commercializing competitive technologies and products.
With respect to patent rights, we do not know whether any of the pending patent applications for any of our products or product candidates will result in the issuance of patents that protect our technology or products, or which will effectively prevent others from commercializing competitive technologies and products.
If we enter into arrangements with third parties to perform sales, marketing and distribution services, our product revenue or the profitability of these product revenue to us may be lower than if we were to market and sell any products that we develop ourselves.
If we enter into arrangements with third parties to perform sales, marketing and distribution services, our product revenue or the profitability of these product revenues to us may be lower than if we were to market and sell any products that we develop ourselves.
If we fail to comply with any of our obligations under the Genzyme license agreement, or we are subject to a bankruptcy, Genzyme may have the right to terminate the license agreement, in which event we would not be able to market any product candidates covered by the license.
If we fail to comply with any of our obligations under the Genzyme license agreement, or we are subject to bankruptcy, Genzyme may have the right to terminate the license agreement, in which event we would not be able to market any product candidates covered by the license.
There have been numerous changes over the past ten years to the patent laws and to the rules of the United States Patent and Trademark Office (“USPTO”), which may have a significant impact on our ability to protect our technology and enforce our intellectual property rights.
There have been numerous changes over the past ten years to the patent laws and to the rules of the United States Patent and Trademark Office (“USPTO”), which may have a significant impact on our ability to protect our technology and enforce our 55 intellectual property rights.
As a result, we might be subject to price regulations that delay our commercial launch of a product, possibly for lengthy time periods, and negatively impact the revenues we are able to generate from the sale of the product in that 44 country.
As a result, we might be subject to price regulations that delay our commercial launch of a product, possibly for lengthy time periods, and negatively impact the revenues we are able to generate from the sale of the product in that country.
If we experience problems with these third parties, the manufacturing of mavorixafor could be delayed, which could harm our results of operations. • We rely on third-party contract research organizations (“CROs”) to conduct our preclinical studies and clinical trials.
If we experience problems with these third parties, the manufacturing of mavorixafor could be delayed, which could harm our results of operations. • We rely on third-party clinical research organizations (“CROs”) to conduct our preclinical studies and clinical trials.
Additionally, our approved product and any future approved products could be subject to marketing restrictions or withdrawal from the market, and we may be subject to penalties if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products.
Additionally, our approved product, XOLREMDI, and future approved products, if any, could be subject to marketing restrictions or withdrawal from the market, and we may be subject to penalties if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products.
Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation.
Furthermore, because of the substantial 59 amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation.
Even though we have obtained approval for one of our product candidates from the FDA, we may never obtain approval for our product candidates outside of the United States, which would limit our market opportunities and could harm our business.
Even though we have obtained FDA approval for one of our product candidates, we may never obtain approval for our product candidates outside of the United States, which would limit our market opportunities and could harm our business.
Any such access, disclosure or other 57 loss of information could result in legal claims or proceedings, liability under laws that protect the privacy of personal information, government enforcement actions and regulatory penalties.
Any such access, disclosure or other loss of information could result in legal claims or proceedings, liability under laws that protect the privacy of personal information, government enforcement actions and regulatory penalties.
Any significant change in market conditions, including a sustained decline in our stock price, that indicate a reduction in carrying value may give rise to impairment in the period that the change becomes known.
Any significant change in market conditions, including a sustained decline in our stock price, that indicate a reduction in carrying value may give rise to a significant impairment charge in the period that the change becomes known.
Protecting against the unauthorized use of our patented technology, trademarks and other intellectual property rights is expensive, difficult and may, in some cases, not be possible.
Protecting against the unauthorized use of our patented technology, trademarks and other intellectual property rights is expensive, difficult and may, 56 in some cases, not be possible.
If reimbursement is not available or is available only to a limited degree, we may not be able to successfully commercialize XOLREMDI or any future product candidate for which we obtain marketing approval. Even if favorable coverage and reimbursement status is attained, less favorable coverage policies and reimbursement rates may be implemented in the future.
If reimbursement is not available or is available only to a limited degree, we may not be able to successfully commercialize mavorixafor or any future product candidate for which we obtain marketing approval. Even if favorable coverage and reimbursement status is attained, less favorable coverage policies and reimbursement rates may be implemented in the future.
Our ability to commercialize XOLREMDI or future product candidates successfully depends in part on the extent to which coverage and adequate reimbursement for these products and related treatments are available from government health administration authorities, private health insurers and other organizations.
Our ability to commercialize mavorixafor or future product candidates successfully depends in part on the extent to which coverage and adequate reimbursement for these products and related treatments are available from government health administration authorities, private health insurers and other organizations.
In particular, we are currently evaluating mavorixafor for the treatment of chronic neutropenic disorders, which are rare diseases with limited patient pools from which to draw for clinical trials. The eligibility criteria of our clinical trials will further limit the pool of available trial participants.
In particular, we are currently evaluating mavorixafor for the treatment of chronic neutropenic disorders, which are rare diseases with limited patient pools from which to draw for clinical trials. The eligibility criteria of our clinical trials will further limit the pool of available trial patients.
A decline in our value could also cause you to lose all or part of your investment. If the commercial opportunity for mavorixafor in WHIM syndrome and other chronic neutropenic disorders is smaller than we anticipate, our potential future revenue from mavorixafor for the treatment of any of these diseases may be adversely affected and our business may suffer.
A decline in our value could also cause you to lose all or part of your investment. If the commercial opportunity for mavorixafor in chronic neutropenic disorders is smaller than we anticipate, our potential future revenue from mavorixafor for the treatment of any of these diseases may be adversely affected and our business may suffer.
We cannot be sure that coverage and reimbursement will be available for XOLREMDI or any other product that we commercialize and, if coverage and reimbursement is available, the level of reimbursement. Reimbursement may impact the demand for, or the price of, any product candidate for which we obtain marketing approval.
We cannot be sure that coverage and reimbursement will be available for mavorixafor or any other product that we commercialize and, if coverage and reimbursement is available, the level of reimbursement. Reimbursement may impact the demand for, or the price of, any product candidate for which we obtain marketing approval.
Progress reports are required at quarterly intervals, every six months and at annual intervals depending upon the country, and more frequently if serious adverse events occur. Our approved product and our product candidates that receive marketing approval will be subject to continual requirements of and review by the FDA and other regulatory authorities.
Progress reports are required at quarterly intervals, every six months and at annual intervals depending upon the country, and more frequently if serious adverse events occur. Our approved product, XOLREMDI, and future product candidates that receive marketing approval, if any, will be subject to continual requirements of and review by the FDA and other regulatory authorities.
To date, we have funded our operations to date primarily with proceeds from sales of common stock, warrants, and prefunded warrants for the purchase of our preferred stock and our common stock, sales of preferred stock, proceeds from the issuance of convertible debt, and borrowings under loan and security agreements.
To date, we have funded our operations primarily with proceeds from sales of common stock, warrants, prefunded warrants, and preferred stock, proceeds from the issuance of convertible debt and borrowings under loan and security agreements.
If we raise additional funds through licensing, collaboration or similar arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research and development programs or product candidates or grant licenses on terms that are not favorable to us.
If we raise additional funds through licensing, collaboration or similar arrangements with third parties, we may be required to relinquish valuable rights to our technologies, future revenue streams, research and development programs or product candidates or grant licenses on terms that are not favorable to us.
Obtaining and maintaining adequate reimbursement for XOLREMDI may be particularly difficult because of the higher prices typically associated with drugs directed at smaller populations of patients.
Obtaining and maintaining adequate reimbursement for mavorixafor may be particularly difficult because of the higher prices typically associated with drugs directed at smaller populations of patients.
Our management, personnel and systems currently in place, after the 2025 Restructuring, may not be adequate to support future operations. The pharmaceutical industry is highly competitive and is subject to rapid and significant technological change, which could render our technologies and products obsolete or uncompetitive.
Our management, personnel and systems currently in place may not be adequate to support future operations. The pharmaceutical industry is highly competitive and is subject to rapid and significant technological change, which could render our technologies and products obsolete or uncompetitive.
See the section in this Annual Report on Form 10-K for the fiscal year ended December 31, 2024 entitled “Business – Government Regulation – Other Healthcare Laws and Compliance Requirements.” Efforts to ensure that our business arrangements with third parties comply with applicable healthcare laws and regulations involve substantial costs.
See the section in this Annual Report on Form 10-K for the fiscal year ended December 31, 2025 titled “Business – Government Regulation – Other Healthcare Laws and Compliance Requirements.” Efforts to ensure that our business arrangements with third parties comply with applicable healthcare laws and regulations involve substantial costs.
Adverse pricing limitations may hinder our ability to recoup our investment in XOLREMDI or future product candidates, even if those candidates obtain marketing approval.
Adverse pricing limitations may hinder our ability to recoup our investment in mavorixafor or future product candidates, even if those candidates obtain marketing approval.
Risks Related to the Marketing and Commercialization of Our Product Candidates Our approved product and any future approved products may still face future development and regulatory difficulties and will be subject to extensive post-approval regulatory requirements.
Risks Related to the Marketing and Commercialization of Our Product Candidates Our product candidates that have received regulatory approval may still face future development and regulatory difficulties and any approved products will be subject to extensive post-approval regulatory requirements.
We cannot be certain that we will be able to obtain regulatory approval for, or successfully commercialize, mavorixafor for chronic neutropenic disorders other than WHIM, or any other product candidate. Our business depends almost entirely on the successful clinical development, regulatory approval and commercialization of mavorixafor.
We cannot be certain that we will be able to obtain regulatory approval for, or successfully commercialize, mavorixafor for disorders other than WHIM, including CN, or any other product candidate. Our business depends almost entirely on the successful clinical development, regulatory approval and commercialization of mavorixafor.
Our license agreement with Genzyme imposes upon us various diligence, payment and other obligations, including the obligation to pay Genzyme (i) future milestone payments in the aggregate amount of up to $13.0 million as of December 31, 2024, contingent upon our achievement of certain late-stage regulatory and sales milestones with respect to licensed products, of which we achieved a regulatory milestone of $3.0 million in January 2025 and paid in March 2025, (ii) our obligation to pay Genzyme tiered royalties based on net sales of licensed products that we commercialize under the agreement; and (iii) our obligation to pay Genzyme a certain percentage of cash payments received by us or our affiliates in consideration for the grant of a sublicense under the license granted to us by Genzyme.
Our license agreement with Genzyme imposes upon us various diligence, payment and other obligations, including the obligation to pay Genzyme (i) future milestone payments in the aggregate amount of up to $10.0 million as of December 31, 2025, contingent upon our achievement of certain late-stage regulatory and sales milestones with respect to licensed products (ii) our obligation to pay Genzyme tiered royalties based on net sales of licensed products that we commercialize under the agreement; and (iii) our obligation to pay Genzyme a certain percentage of cash payments received by us or our affiliates in consideration for the grant of a sublicense under the license granted to us by Genzyme.
See the sections of this Annual Report on Form 10-K for the fiscal year ended December 31, 2024 entitled, “Business – Government Regulation – Pharmaceutical Coverage, Pricing and Reimbursement” and “Business – Government Regulation – Healthcare Reform.” We are subject to anti-corruption laws, as well as export control laws, customs laws, sanctions laws and other laws governing our operations.
See the sections of this Annual Report on Form 10-K for the fiscal year ended December 31, 2025 titled, “Business – Government Regulation – Coverage and Reimbursement” and “Business – Government Regulation – Healthcare Reform.” We are subject to anti-corruption laws, as well as export control laws, customs laws, sanctions laws and other laws governing our operations.
The strategic restructuring activities include (i) discontinuing of research efforts, (ii) closing the Company’s facility in Vienna, Austria, (iii) pausing pre-clinical drug candidate programs, (iv) scaling the U.S. commercial field team and supporting roles across the Company and (v) streamlining other spending to support the ongoing clinical development of mavorixafor for the larger population of those with chronic neutropenia.
The strategic restructuring activities included (i) discontinuing of certain research efforts, (ii) closing the Company’s facility in Vienna, Austria, (iii) pausing certain pre-clinical drug candidate programs, (iv) scaling back the U.S. commercial field team and supporting roles across the Company and (v) streamlining other spending to support the ongoing clinical development of mavorixafor for the larger population of those with CN.
Some of the factors that may cause the market price of our common stock to fluctuate include: • our ability or the ability of our collaborators to develop product candidates and conduct clinical trials that demonstrate such product candidates are safe and effective; • our ability or the ability of our collaborators to obtain regulatory approvals for product candidates, and delays or failures to obtain such approvals; • failure of any our product candidates to demonstrate safety and efficacy, receive regulatory approval and achieve commercial success; • failure to maintain our existing third-party license, manufacturing and supply agreements; • failure by us or our licensors to prosecute, maintain or enforce our intellectual property rights; • changes in laws or regulations applicable to our current or future product candidates; • any inability to obtain adequate supply of product candidates or the inability to do so at acceptable prices; • adverse decisions by regulatory authorities; • introduction of new or competing products by our competitors; • failure to meet or exceed financial and development projections that we may provide to the public; • the perception of the pharmaceutical industry by the public, legislatures, regulators and the investment community; • announcements of significant acquisitions, strategic collaborations, joint ventures or capital commitments by us or our competitors; • disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain intellectual property protection for our technologies; • additions or departures of key personnel; • significant lawsuits, including intellectual property or stockholder litigation; • announcements by us of material developments in our business, financial condition and/or operations; • if securities or industry analysts do not publish research or reports about us, or if they issue an adverse or misleading opinions regarding our business and stock; • changes in the market valuations of similar companies; • general macroeconomic, political and market conditions and overall fluctuations in the financial markets in the United States and abroad; • sales of our common stock or our stockholders in the future; • trading volume of our common stock; • adverse publicity relating to our markets generally, including with respect to other products and potential products in such markets; • changes in the structure of health care payment systems; • period-to-period fluctuations in our financial results; and • the other factors described in this “Risk Factors” section and elsewhere in this Annual Report In addition, companies trading in the stock market in general have experienced extreme price and volume fluctuations that have often been unrelated or disproportionate to the operating performance of these companies, which has resulted in decreased stock prices for many companies notwithstanding the lack of a fundamental change in their underlying business models or prospects.
Some of the factors that may cause the market price of our common stock to fluctuate include: • our ability or the ability of our collaborators to develop product candidates and conduct clinical trials that demonstrate such product candidates are safe and effective; • our ability or the ability of our collaborators to obtain regulatory approvals for product candidates, and delays or failures to obtain such approvals; • failure of any our product candidates to demonstrate safety and efficacy, receive regulatory approval and achieve commercial success; • failure to maintain our existing third-party license, manufacturing and supply agreements; • failure by us or our licensors to prosecute, maintain or enforce our intellectual property rights; • changes in laws or regulations applicable to our current or future product candidates; • any inability to obtain adequate supply of product candidates or the inability to do so at acceptable prices; • adverse decisions by regulatory authorities; • introduction of new or competing products by our competitors; • failure to meet or exceed financial and development projections that we may provide to the public; • the perception of the pharmaceutical industry by the public, legislatures, regulators and the investment community; • announcements of significant acquisitions, strategic collaborations, joint ventures or capital commitments by us or our competitors; • disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain intellectual property protection for our technologies; • additions or departures of key personnel; • significant lawsuits, including intellectual property or stockholder litigation; • announcements by us of material developments in our business, financial condition and/or operations; • if securities or industry analysts do not publish research or reports about us, or if they issue an adverse or misleading 64 opinions regarding our business and stock; • changes in the market valuations of similar companies; • general macroeconomic, political and market conditions and overall fluctuations in the financial markets in the United States and abroad; • sales of our common stock or our stockholders in the future; • trading volume of our common stock; • adverse publicity relating to our markets generally, including with respect to other products and potential products in such markets; • changes in the structure of health care payment systems; • period-to-period fluctuations in our financial results; and • the other factors described in this “Risk Factors” section and elsewhere in this Annual Report.
Successfully completing clinical trials and obtaining approval of an NDA is a complex, lengthy, expensive and uncertain process, and the FDA, or a comparable foreign regulatory authority, may delay, limit or deny approval of mavorixafor for the treatment of other indications for many reasons, including, among others: • disagreement with the design or implementation and sufficiency of our clinical trials; • failure to demonstrate the safety and efficacy of mavorixafor or any other product candidate for its proposed indications and that; 35 • any clinical and other benefits of mavorixafor or any other product candidate outweigh its safety risks; • deficiencies in the manufacturing or control processes or failure of third-party manufacturing facilities with which we contract for clinical and commercial supplies to comply with current cGMPs; • insufficient data collected from clinical trials of mavorixafor or any other product candidate, or changes in the approval requirements that render its nonclinical and clinical data insufficient to support the filing of an NDA or to obtain regulatory approval; or • changes in clinical practice in or approved products available for the treatment of the target patient population that could have an impact on the indications that we are pursuing for mavorixafor or our other product candidates.
Successfully completing clinical trials and obtaining approval of an NDA is a complex, lengthy, expensive and uncertain process, and the FDA, or a comparable foreign regulatory authority, may delay, limit or deny approval of mavorixafor for the treatment of other indications for many reasons, including, among others: • disagreement with the design or implementation and sufficiency of our clinical trials; • failure to demonstrate the safety and efficacy of mavorixafor or any other product candidate for its proposed indications and that; • any clinical and other benefits of mavorixafor or any other product candidate outweigh its safety risks; • deficiencies in the manufacturing or control processes or failure of third-party manufacturing facilities with which we contract for clinical and commercial supplies to comply with current cGMPs; • insufficient data collected from clinical trials of mavorixafor or any other product candidate, or changes in the approval requirements that render its nonclinical and clinical data insufficient to support the filing of an NDA or to obtain regulatory approval; or • changes in clinical practice in or approved products available for the treatment of the target patient population that could have an impact on the indications that we are pursuing for mavorixafor or our other product candidates. 41 The FDA or a comparable foreign regulatory authority may also require more information, including additional nonclinical or clinical data to support approval, which may delay or prevent approval of our commercialization plans, or cause us to abandon the development program.
Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through public or private equity or debt financings, third-party funding, marketing and distribution arrangements, as well as other collaborations, strategic alliances and licensing arrangements, or any combination of these approaches.
Until such time, if ever, as we can generate product and licensing revenues that are in excess of our operating expense, we expect to finance our cash needs through public or private equity or debt financings, third-party funding, marketing and distribution arrangements, as well as other collaborations, strategic alliances and licensing arrangements, or any combination of these approaches.
Our approved product and product candidates that receive regulatory approval will be subject to extensive ongoing requirements by the FDA and comparable foreign regulatory authorities governing the manufacture, quality control, further development, labeling, packaging, storage, distribution, safety surveillance, import, export, advertising, promotion, recordkeeping and reporting of safety and other post-market information.
Any product candidate that receives regulatory approval will be subject to extensive ongoing requirements by the FDA and comparable foreign regulatory authorities governing the manufacture, quality control, further development, labeling, packaging, storage, distribution, safety surveillance, import, export, advertising, promotion, recordkeeping and reporting of safety and other post-market information.
Our ability to generate profits from operations and thereafter to remain profitable depends heavily on: • our ability to generate revenue from XOLREMDI; • outcomes and timing of regulatory reviews, approvals and other actions; • our ability to manufacture any approved products on commercially reasonable terms; • our ability to maintain an effective sales and marketing organization or suitable third-party alternatives for any approved products; • the scope, number, progress, duration, endpoints, cost, results and timing of clinical trials and nonclinical studies of our current or potential future product candidates, including in particular the scope, progress, duration, endpoints, cost, results and timing for completion of our Phase 3 clinical trial of mavorixafor for the treatment of chronic neutropenic disorders; • our ability to raise sufficient funds to support the development and potential commercialization of our product candidates; • our ability to market our approved product and obtain marketing approval for our product candidates; • our ability to establish and maintain licensing, collaboration or similar arrangements on favorable terms and whether and to what extent we retain development or commercialization responsibilities under any new licensing, collaboration or similar arrangement; • the success of any other business, product or technology that we acquire or in which we invest; • our ability to maintain, expand and defend the scope of our intellectual property portfolio; • the number and characteristics of product candidates and programs that we pursue; • hire additional clinical, regulatory and scientific personnel; and • incur additional legal, accounting and other expenses associated with operating as a public company.
Our ability to generate profits from operations and thereafter to remain profitable depends heavily on: • our ability to generate revenue from mavorixafor; • outcomes and timing of regulatory reviews, approvals and other actions; • our ability to manufacture any approved products on commercially reasonable terms; • our ability to maintain an effective sales and marketing organization or suitable third-party alternatives for any approved products; 36 • the scope, number, progress, duration, endpoints, cost, results and timing of clinical trials and nonclinical studies of our current or potential future product candidates, including in particular the scope, progress, duration, endpoints, cost, results and timing for completion of our 4WARD Phase 3 clinical trial of mavorixafor for the treatment of chronic neutropenic disorders; • our ability to raise sufficient funds to support the development and potential commercialization of our product candidates; • our ability to market our approved product and obtain marketing approval for our product candidates; • our ability to manage our cash expenditures in line with our budget as approved by our Board of Directors, which reflects the cost savings we anticipate from our restructuring activities executed in 2025; • our ability to establish and maintain licensing, collaboration or similar arrangements on favorable terms and whether and to what extent we retain development or commercialization responsibilities under any new licensing, collaboration or similar arrangement; • the success of any other business, product or technology that we acquire or in which we invest; • our ability to maintain, expand and defend the scope of our intellectual property portfolio; • the number and characteristics of product candidates and programs that we pursue; • our ability to hire additional clinical, regulatory and scientific personnel; and • the extent of legal, accounting and other expenses that we incur to continue to operate as a public company.
Our net operating loss (“NOL”) carryforwards could expire unused and be unavailable to offset future tax liabilities because of their limited duration or because of restrictions under U.S. tax law. As of December 31, 2024, we had U.S. federal and state NOLs of $372.9 million and $374.2 million, respectively.
Our net operating loss (“NOL”) carryforwards could expire unused and be unavailable to offset future tax liabilities because of their limited duration or because of restrictions under U.S. tax law. As of December 31, 2025, we had U.S. federal and state NOLs of $432.0 million and $383.9 million , respectively.
Even if we are able to successfully achieve regulatory approval for these product candidates, we are unable to predict the extent of any future losses and do not know when any of these product candidates will generate revenue for us, if at all.
Our ability to generate revenue and become profitable depends upon our ability to successfully commercialize mavorixafor. Even if we are able to successfully achieve regulatory approval for our product candidates, we are unable to predict the extent of any future losses and do not know when any of these product candidates will generate revenue for us, if at all.
In some cases, the price that we intend to charge for any product candidates, is also subject to approval. Obtaining approval for any future product candidates in the European Union from the European Commission following the opinion of the 43 EMA would be a lengthy and expensive process.
In some cases, the price that we intend to charge for any product candidate is also subject to approval. Obtaining approval for any future product candidates in the European Union from the European Commission following the opinion of the European Medicines Agency (“EMA”) could be a lengthy and expensive process.
To date, we have not generated significant revenues from product sales and cannot predict whether or when we will be able to generate meaningful revenues from sales of XOLREMDI at levels or on timing necessary to support our investment and goals.
We may never be able to generate meaningful revenues from sales of our product candidates and approved commercial products at levels or on timing necessary to support our investment and goals. 38 To date, we have not generated significant revenues from product sales and cannot predict whether or when we will be able to generate meaningful revenues from sales of our product candidates and our approved commercial products at levels or on timing necessary to support our investment and goals.
Investment in biopharmaceutical product development is highly speculative because it entails substantial upfront capital expenditures and significant risk that any potential product candidate will fail to demonstrate adequate effect or an acceptable safety profile, gain regulatory approval, become commercially viable, or maintain commercial viability.
We are a commercial-stage biopharmaceutical company. Investment in biopharmaceutical product development is highly speculative because it entails substantial upfront capital expenditures and significant risk that any potential product candidate will fail to demonstrate adequate effect or an acceptable safety profile, gain regulatory approval, become commercially viable, or maintain commercial viability. Since inception, we have incurred significant operating losses.
Our ability to generate revenue from XOLREMDI, mavorixafor or any of our current or future product candidates also depends on a number of additional factors, including but not limited to our ability to: • successfully complete development activities, including all necessary nonclinical studies and clinical trials; • complete and submit New Drug Applications to the FDA and obtain regulatory approval for indications for which there is a commercial market; • complete and submit marketing applications to, and obtain regulatory approval from, foreign regulatory authorities; • set and obtain a commercially viable price for our products; • obtain commercial quantities of our products at acceptable cost levels; • further develop a commercial organization capable of sales, marketing and distribution for the products we intend to sell ourselves in the markets in which we have retained commercialization rights; • find suitable collaborators to help us market, sell and distribute our approved products in other markets; and • obtain coverage and adequate reimbursement from third-party, including government, payors. 33 In addition, because of the numerous risks and uncertainties associated with product development, including the possibility that our product candidates may not advance through development or demonstrate safety and efficacy for their intended uses, the FDA or any other regulatory agency may require additional clinical trials or nonclinical studies.
Our ability to generate revenue from mavorixafor or any of our future product candidates also depends on a number of additional factors, including but not limited to our ability to: • successfully complete development activities, including all necessary nonclinical studies and clinical trials; • complete and submit NDAs to the FDA and obtain regulatory approval for indications for which there is a commercial market; • complete and submit marketing applications to, and obtain regulatory approval from, foreign regulatory authorities; • set and obtain a commercially viable price for our products; • obtain commercial quantities of our products at acceptable cost levels; • further develop a commercial organization capable of sales, marketing and distribution for the products we intend to sell ourselves in the markets in which we have retained commercialization rights; • find suitable collaborators to help us market, sell and distribute our approved products in other markets; and • obtain coverage and adequate reimbursement from third-party, including government, payors.
(the “Hercules Loan Agreement”) and subject to certain operational covenants. Based on our current cash flow projections, excluding additional sources of external financing, we anticipate that we will not be able to maintain the minimum cash required to satisfy this covenant for at least the next 12 month period following the issuance of these consolidated financial statement.
Based on our current cash flow projections, excluding additional sources of external financing, we anticipate that we will be able to maintain the minimum cash required to satisfy this covenant for at least the next 12 month period following the issuance of these consolidated financial statements.
Additionally, the addressable patient population for our indications may be limited or may not be amenable to treatment with mavorixafor, and new patients may become increasingly difficult to identify or gain access to, which would adversely affect our results of operations and our business.
Additionally, the addressable patient population for our indications may be limited or may not be amenable to treatment with mavorixafor, and new patients may become increasingly difficult to identify or gain access to, which would adversely affect our results of operations and our business. 39 We depend almost entirely on the success of our future product candidate, mavorixafor.
Regardless of merit or eventual outcome, liability claims may result in: • reduced resources of our management to pursue our business strategy; • decreased demand for any products that we may develop; • injury to our reputation and significant negative media attention; 45 • withdrawal of clinical trial participants; • significant costs to defend any related litigation; • substantial monetary awards to trial participants or patients; • loss of revenue; • increased insurance costs; and • the inability to commercialize any products that we may develop.
If we cannot successfully defend ourselves against claims that our product candidates or products caused injuries, we could incur substantial liabilities. 50 Regardless of merit or eventual outcome, liability claims may result in: • reduced resources of our management to pursue our business strategy; • decreased demand for any products that we may develop; • injury to our reputation and significant negative media attention; • withdrawal of clinical trial patients; • significant costs to defend any related litigation; • substantial monetary awards to trial patients or patients; • loss of revenue; • increased insurance costs; and • the inability to commercialize any products that we may develop.
The current expectation is that we will retain our future earnings to fund the development and growth of our business. In addition, the terms of our debt agreements preclude us from paying dividends. As a result, capital appreciation, if any, of our common stock will be your sole source of gain, if any, for the foreseeable future.
The current expectation is that we will retain our future earnings to fund the development and growth of our business. As a result, capital appreciation, if any, of our common stock will be your sole source of gain, if any, for the foreseeable future.
Our drug development programs and the potential commercialization of our product candidates will require substantial additional cash to fund expenses. For some of our product candidates, we may decide to collaborate with pharmaceutical and biotechnology companies for the development and potential commercialization of those product candidates. We face significant competition in seeking appropriate collaborators.
For some of our product candidates, we may decide to collaborate with pharmaceutical and biotechnology companies for the development and potential commercialization of those product candidates. We face significant competition in seeking appropriate collaborators.
The ability of the FDA to review and approve new products can be affected by a variety of factors, including government budget and funding levels, the ability to hire and retain key personnel and accept the payment of user fees, and statutory, regulatory and policy changes. Average review times at the agency have fluctuated in recent years as a result.
The ability of the FDA to review and approve new products can be affected by a variety of factors, including government budget and funding levels, ability to hire and retain key personnel and accept the payment of user fees, and statutory, regulatory, and policy changes.
We depend on license agreements with Genzyme, Beth Israel Deaconess Medical Center, Georgetown University and Dana-Farber Cancer Institute to permit us to use patents and patent applications. Termination of these rights or the failure to comply with obligations under these agreements could materially harm our business and prevent us from developing or commercializing our product candidates.
We depend on license agreements with Genzyme and others to permit us to use patents and patent applications. Termination of these rights or the failure to comply with obligations under these agreements could materially harm our business and prevent us from developing or commercializing our product candidates.
For example, as a result of the COVID-19 pandemic, we previously have experienced a slower enrollment pace in some of our clinical trials. 39 If we cannot identify patients to participate in our clinical trials or if patients are unwilling to participate in our clinical trials for any reason, including if patients choose to enroll in competitive clinical trials for similar patient populations, the timeline for recruiting patients, conducting studies and obtaining regulatory approval of mavorixafor and any other current or future product candidates that we may develop may be delayed.
If we cannot identify patients to participate in our clinical trials or if patients are unwilling to participate in our clinical trials for any reason, including if patients choose to enroll in competitive clinical trials for similar patient populations, the timeline for recruiting patients, conducting studies and obtaining regulatory approval of mavorixafor and any other current or future product candidates that we may develop may be delayed.
We have completed a Section 382 study that has identified ownership changes that will limit the future use of our NOL carryforwards. See Note 16, Income Taxes , for a further discussion of these limitations.
We have completed a Section 382 study that has identified ownership changes that will limit the future use of our NOL carryforwards. See Note 17 of our Annual Report on Form 10-K for a further discussion of these limitations.
Additionally, any product candidate for which we obtain marketing approval could be subject to marketing restrictions or withdrawal from the market and we may be subject to penalties if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products. • The FDA and other domestic and foreign regulatory agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses.
Additionally, our approved product, XOLREMDI, and future approved products, if any, could be subject to marketing restrictions or withdrawal from the market, and we may be subject to penalties if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products. • The Food and Drug Administration (“FDA”) and other regulatory agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses.
For example, over the last several years the U.S. government has shut down several times and certain regulatory agencies, such as the FDA and the SEC, have had to furlough critical FDA, SEC and other government employees and stop critical activities.
For example, over the last several years the U.S. government has shut down several times, including as recently as October 2025, and certain regulatory agencies, such as the FDA and the SEC, have had to furlough critical government employees and stop critical activities. In 2025, the FDA has been subject to reductions in force and reorganization.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeThe Director of Infrastructure, Operations & Cybersecurity is responsible for the day-to-day monitoring and remediation of cybersecurity risks. The board is responsible for informed oversight of our risk management process. The board administers this oversight function through various board standing committees that address risks inherent in their respective areas of oversight.
Biggest changeThe Board administers this oversight function through various board standing committees that address risks inherent in their respective areas of oversight. The Board has delegated oversight for cybersecurity risk management to the Audit Committee. The Audit Committee reviews the Company’s policies and procedures with respect to cybersecurity risk management.
ITEM 1C. CYBERSECURITY Cybersecurity Risk Management and Strategy Our management recognizes the impact that cybersecurity threats could have on our business operations, our compliance with regulations, and our reputation. We have identified cybersecurity as a critical business risk as part of our overall risk management strategy, which our board of directors oversees.
Item 1C. Cybersecurity Cybersecurity Risk Management and Strategy Our management recognizes the impact that cybersecurity threats could have on our business operations, our compliance with regulations, and our reputation. We have identified cybersecurity as a critical business risk as part of our overall risk management strategy, which our Board of Directors (the “Board”) oversees.
Although risks from cybersecurity threats have to date not materially affected us, our business strategy, results of operations or financial condition, we have, from time to time, experienced threats to and breaches of our and our third-party vendors’ data and systems. For more information, see Item 1A. Risk Factors.
Although risks from cybersecurity threats have to date not materially affected us, our business strategy, results of operations or financial condition this fiscal year, we have, from time to time, experienced threats to and breaches of our and our third-party vendors’ data and systems. For more information, see Item 1A. Risk Factors. 67
Our vendor evaluation procedures include, as appropriate, the review of vendors’ SOC 2 Type 2 reports and a vendor security questionnaire. Governance Related to Cybersecurity Risks Our Director of Infrastructure, Operations and Cybersecurity, who has over ten years of experience managing organizational operations, security and infrastructure, manages the Company’s cybersecurity program.
Our vendor evaluation procedures include, as appropriate, the review of vendors’ SOC 2 Type 2 reports and a vendor security questionnaire. Governance Related to Cybersecurity Risks Our Director of Infrastructure, Operations and Cybersecurity (“IT Director”), who reports to our Chief Financial Officer, has over ten years of experience managing organizational operations, security and infrastructure.
We have implemented a cybersecurity program in accordance with our risk profile and business that includes, among other things, written policies, monitoring and filtering procedures, and employee training.
We have implemented a cybersecurity program in accordance with our risk profile and business that includes, among other things, written policies, monitoring and filtering procedures, and employee training. We have also developed an incident response policy and procedure designed to facilitate the timely reporting and assessment of cybersecurity incidents.
We have also developed an incident response policy and procedure designed to facilitate the timely reporting and assessment of cybersecurity incidents. 63 Our cybersecurity risk management program, which is part of our enterprise risk management program, aims to identify risks related to the Company, including risks from cybersecurity threats.
Our cybersecurity risk management program, which is part of our enterprise risk management program, aims to identify risks related to the Company, including risks from cybersecurity threats. We engage third party advisors, including assessors and cybersecurity consultants, to assess, validate and enhance our cybersecurity program.
Removed
We have a cyber subcommittee (the “Subcommittee”), which includes members of our finance, legal, and IT departments, that meets periodically to discuss the Company’s ongoing cybersecurity efforts. The Subcommittee reports to the Chief Operating Officer (“COO”) and Chief Financial Officer (“CFO”), who report outputs from the Subcommittee regarding potential cybersecurity risks to the Company’s executive team and the board.
Added
The IT Director manages the Company’s cybersecurity program and is responsible for the day-to-day monitoring and remediation of cybersecurity risks. The IT Director meets periodically with members of our finance and legal departments to discuss the Company’s ongoing cybersecurity efforts. The Board is responsible for informed oversight of our risk management process.
Removed
The board has delegated oversight for cybersecurity risk management to the Audit Committee. The Audit Committee reviews the Company’s policies and procedures with respect to cybersecurity risk management.
Added
Since the beginning of fiscal year 2025, we have not identified risks from known cybersecurity threats or incidents that have materially affected us or are reasonably likely to materially affect us.

Item 2. Properties

Properties — owned and leased real estate

1 edited+2 added−3 removed1 unchanged
Biggest changeThe base monthly payment on the lease is approximat ely $91 thousand as of December 31, 2024, s ubject to specified annual increases of approximately 3% during the term of the lease and not including operating expenses, certain utilities, taxes and insurance for which we are responsible.
Biggest changeThe base monthly payment on the lease is approximat ely $96 thousand as of December 31, 2025, not including operating expenses, certain utilities, taxes and insurance for which we are responsible. We have the right to sublease the premises, subject to landlord consent. Item 3.
Removed
We have the right to sublease the premises, subject to landlord consent and we have the right to renew the lease for an additional five years at the then-prevailing effective market rental rate.
Added
Legal Proceedings From time to time, we may become involved in legal proceedings or be subject to claims arising in the ordinary course of our business.
Removed
We lease approximately 1,200 square meters of laboratory and office space in Vienna, Austria under a lease that will expire in March 2028, with a monthly payment of approximately $24 thousand. ITEM 3.
Added
We are not currently a party to any material legal proceedings and we are not aware of any pending or threatened legal proceedings against us that we believe could have a material adverse effect on our business, operating results or financial condition.
Removed
LEGAL PROCEEDINGS From time to time, we may be involved in lawsuits, claims, investigations and proceedings, consisting of intellectual property, commercial, employment and other matters which arise in the ordinary course of business. While the outcome of any such proceedings cannot be predicted with certainty, as of December 31, 2024, we were not party to any legal proceedings.

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock commenced trading on the Nasdaq Global Market under the symbol “ASNS” on November 16, 2017. Prior to that date, there was no public trading market for our common stock.
Biggest changeItem 5. Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Market Information Our common stock is traded on the Nasdaq Capital Market under the symbol “XFOR”.
Equity Compensation Plan The information required by Item 5 of Form 10-K regarding equity compensation plans is incorporated herein by reference to Item 12 of Part III of this Annual Report on Form 10-K. Recent Sales of Unregistered Securities None. Purchase of Equity Securities by the Issuer and Affiliated Purchasers None.
Equity Compensation Plan The information required by Item 5 of Form 10-K regarding equity compensation plans is incorporated herein by reference to Item 12 of Part III of this Annual Report on Form 10-K. Recent Sales of Unregistered Securities None. Purchase of Equity Securities by the Issuer and Affiliated Purchasers None. Item 6. [Reserved] 69
All of the shares of common stock held by brokerage firms, banks and other financial institutions as nominees for beneficial owners are deposited into participant accounts at DTC, and are considered to be held of record by Cede & Co. as one stockholder. Dividend Policy We have never declared or paid cash dividends on our common stock.
All of the shares of common stock held by brokerage firms, banks and other financial institutions as nominees for beneficial owners are deposited into participant accounts at DTC, and are considered to be held of record by Cede & Co. as one stockholder.
We currently intend to retain all available funds and any future earnings to fund the development and expansion of our business and we do not anticipate paying any cash dividends in the foreseeable future.
Dividend Policy We have never declared or paid cash dividends on our common stock. We currently intend to retain all available funds and any future earnings to fund the development and expansion of our business, and we do not anticipate paying any cash dividends in the foreseeable future.
Holders of Our Common Stock As of March 13, 2025, there were 48 holders of record of our common stock, one of which is Cede & Co., a nominee for Depository Trust Company (“DTC”).
Holders of Our Common Stock As of March 12, 2026, based on information from our transfer agent, there were 38 holders of record of our common stock, one of which is Cede & Co., a nominee for Depository Trust Company (“DTC”).
Removed
On March 13, 2019, we completed a business combination in accordance with the terms of the Merger Agreement, by and among us, X4 Therapeutics, Inc.
Added
The actual number of stockholders is greater than this number of record holders, and includes stockholders who are beneficial owners, but whose shares are held in street name by brokers and other nominees. This number of holders of record also does not include stockholders whose shares may be held in trust by other entities.
Removed
(formerly X4 Pharmaceuticals, Inc.) and the Merger Sub, pursuant to which, among other matters, Merger Sub merged with and into X4 Therapeutics, Inc., with X4 Therapeutics, Inc. continuing as our wholly-owned subsidiary and the surviving corporation of the merger.
Removed
Following the Merger, on March 14, 2019, we effected a 1-for-6 reverse stock split of our common stock and changed our name to “X4 Pharmaceuticals, Inc.” On March 13, 2019, following the completion of the Merger, our common stock began trading on the Nasdaq Capital Market under the symbol “XFOR”.

Item 7. Management's Discussion & Analysis

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

51 edited+32 added−58 removed32 unchanged
Biggest changeIf we are unable to obtain funding, we could be forced to delay, reduce, or eliminate some or all of our research and development programs, product portfolio expansion or commercialization efforts, which would adversely affect our business prospects, or we may be unable to continue operations. 71 Cash Flows The following table summarizes our cash flow activities for each of the periods presented: Year Ended December 31, 2024 2023 (in thousands) Net loss $ (37,450) $ (101,167) Adjustments to reconcile net loss to net cash used in operating activities (96,126) 4,311 Changes in operating assets and liabilities 2,675 344 Net cash used in operating activities (130,901) (96,512) Net cash provided by (used in) investing activities 66,990 (14,883) Net cash provided by financing activities 20,294 88,516 Impact of foreign exchange on cash and restricted cash (156) 99 Net decrease in cash, cash equivalents and restricted cash (43,773) (22,780) Cash, cash equivalents and restricted cash, beginning of period 100,248 123,028 Cash, cash equivalents and restricted cash, end of period $ 56,475 $ 100,248 Operating Activities: During the year ended December 31, 2024, net cash used in operating activities was $130.9 million, primarily resulting from our operating losses and other (expense) income, net, of $142.5 million adjusted for noncash expenses of $8.9 million and changes in our operating assets and liabilities of $2.7 million.
Biggest changeHistorical Cash Flows The following table summarizes our cash flow activities for each of the periods presented: Year Ended December 31, 2025 2024 (in millions) Net loss $ (79.2) $ (37.5) Adjustments to reconcile net loss to net cash used in operating activities (3.8) (96.1) Changes in operating assets and liabilities (2.6) 2.7 Net cash used in operating activities (85.6) (130.9) Net cash provided by investing activities 8.2 67.0 Net cash provided by financing activities 238.6 20.3 Effect of exchange rate changes on cash, cash equivalents and restricted cash 0.2 (0.1) Net increase (decrease) in cash, cash equivalents and restricted cash 161.4 (43.7) Cash, cash equivalents and restricted cash, beginning of period 56.5 100.2 Cash, cash equivalents and restricted cash, end of period $ 217.9 $ 56.5 Operating Activities During the year ended December 31, 2025, net cash used in operating activities was $85.6 million, primarily resulting from net losses of $79.2 million adjusted for net non-cash income of $3.8 million, primarily related to gains on changes to the fair value of our Class C warrants that are measured quarterly at fair value, and $2.6 million of changes to operating assets and liabilities primarily related to a reduction in accounts payable and accrued expenses.
As our common stock does not have a sufficient history of trading, we estimate our volatility based on the historical volatility of publicly traded peer companies. We estimate the expected term of our stock awards by utilizing the “simplified” method, which calculates the expected term based on weighted average midpoint of the award’s vesting and expiration dates.
As our common stock does not have a sufficient history of trading, we estimate our volatility based on the historical volatility of publicly traded peer companies. We estimate the expected term of our stock awards by utilizing the “simplified” method, which calculates the expected term based on the weighted average midpoint of the award’s vesting and expiration dates.
The variable consideration includes estimates for discounts, product returns, rebates that will be due to U.S. federal and state payors, such as Medicaid, based on agreements that we have with these payors who provide medical insurance to the end patient, and 75 estimated co-pay assistance payments for patients who enroll in our patient assistance program.
The variable consideration includes estimates for discounts, product returns, and rebates that will be due to U.S. federal and state payors, such as Medicaid, based on agreements that we have with these payors who provide medical insurance to the end patient, and estimated co-pay assistance payments for patients who enroll in our patient assistance program.
Definite-lived intangible assets related to capitalized milestones under license agreements are amortized on a straight-line basis, which aligns with the pattern over which the economic benefit of the intangible assets are consumed, over their remaining useful lives, which are estimated to be the remaining patent life.
Definite-lived intangible assets related to capitalized milestones under license agreements are amortized on a straight-line basis, which aligns with the pattern over which the economic benefit of the intangible assets is consumed, over their remaining useful lives, which are estimated to be the remaining patent life.
When we conclude that the achievement of such milestones is probable, we include the value of the milestone in the transaction price using the most likely amount method. For product sales, we determine the amount of variable consideration to include in the transaction price by using the expected value method.
When we conclude that the achievement of such milestones is probable, we include the value of the milestone in the transaction price using the most likely amount method. For product sales, we determine the 77 amount of variable consideration to include in the transaction price by using the expected value method.
Net revenue recognized for each period is the amount for which, based on our estimate, it is probable that a significant reversal of cumulative revenue recognized will not occur. At the end of each subsequent reporting period, we re-evaluate these estimates based on new information and actual operational trends and if necessary, adjusts these variable consideration estimates.
Net revenue recognized for each period is the amount for which, based on our estimate, it is probable that a significant reversal of cumulative revenue recognized will not occur. At the end of each subsequent reporting period, we re-evaluate these estimates based on new information and actual operational trends and, if necessary, adjust these variable consideration estimates.
We may take advantage of certain of the scaled disclosures available to smaller reporting companies and will be able to take advantage of these scaled disclosures for so long as (i) our voting and non-voting common stock held by non-affiliates is less than $250.0 million measured on the last business day of our second fiscal quarter or (ii) our annual revenue is less than $100.0 million during the most recently completed fiscal year and our voting and non-voting common stock held by non-affiliates is less than $700.0 million measured on the last business day of our second fiscal quarter.
We may take advantage of certain of the scaled disclosures available to smaller reporting companies for so long as (i) our voting and non-voting common stock held by non-affiliates is less than $250.0 million measured on the last business day of our second fiscal quarter or (ii) our annual revenue is less than $100.0 million during the most recently completed fiscal year and our voting and non-voting common stock held by non-affiliates is less than $700.0 million measured on the last business day of our second fiscal quarter.
We performs the following five steps to determine the amount of revenue to recognize: (1) identify the customer and contract with the customer; (2) identify the performance obligations in the contract; (3) determine the transaction price, adjusted for variable consideration resulting from potential returns, rebates, discounts, and down-stream charges; (4) allocate the transaction price to the performance obligations in the contract; and (5) recognize revenue when we satisfies the performance obligations, which is upon shipment of our drug product to the customer or delivery of licensed rights to the customer.
We perform the following five steps to determine the amount of revenue to recognize: (1) identify the customer and contract with the customer; (2) identify the performance obligations in the contract; (3) determine the transaction price, adjusted for variable consideration resulting from potential returns, rebates, discounts, and down-stream charges; (4) allocate the transaction price to the performance obligations in the contract; and (5) recognize revenue when we satisfy the performance obligations, which is upon shipment of our drug product to the customer or delivery of licensed rights to the customer.
The majority of our service providers invoice us in arrears for services performed, on a pre-determined schedule or when contractual milestones are met; however, some require advance payments. We make estimates of our accrued expenses as of each balance sheet date in the consolidated financial statements based on facts and circumstances known to them at that time.
The majority of our service providers invoice us in arrears for services performed, on a predetermined schedule or when contractual milestones are met; however, some require advance payments. We make estimates of our accrued expenses as of each balance sheet date in the consolidated financial statements based on facts and circumstances known to us at that time.
However, because of the numerous risks and uncertainties associated with the future sale of our approved drug product and the research, development, and commercialization of future product candidates, we are unable to estimate the exact amount of our funding requirements.
Due to the numerous risks and uncertainties associated with the future sale of our approved drug product and the research, development, and commercialization of future product candidates, we are unable to estimate the exact amount of our funding requirements.
Prior to the closing of the Merger and the listing of our common stock on the Nasdaq Capital Market, our board of directors historically determined, as of the date of each option grant and with input from our management, the assistance of a third-party valuation specialist the estimated fair value of our common stock on the date of grant based on a number of objectives and subjective factors.
Prior to the closing of the merger with Arsanis Inc in March 2019 and the listing of our common stock on the Nasdaq Capital Market, our board of directors historically determined, as of the date of each option grant and with input 76 from our management, and the assistance of a third-party valuation specialist, the estimated fair value of our common stock on the date of grant based on a number of objectives and subjective factors.
Liquidity and Capital Resources Sources of Liquidity To date, we have funded our operations primarily with proceeds from sales of common stock, warrants and prefunded warrants for the purchase of our preferred stock and our common stock, sales of preferred stock, proceeds from the issuance of convertible debt and borrowings under loan and security agreements.
Liquidity and Capital Resources Sources of Liquidity To date, we have funded our operations primarily with proceeds from sales of common stock, warrants, prefunded warrants and preferred stock, proceeds from the issuance of convertible debt and borrowings under loan and security agreements. Public and Private Equity Offerings.
The estimate of costs that we expect to incur related to the 2025 Restructuring as well as the decrease in annual spending, and the timing thereof are subject to a number of assumptions and actual results may differ.
The estimate of costs that we expect to incur related to these workforce reduction as well as the decrease in spending, and the timing thereof are subject to a number of assumptions and actual results may differ.
As discussed further below, o n January 13, 2025, we entered into a License and Supply Agreement (the “Norgine Agreement”) with Norgine Pharma UK Limited (“Norgine”), pursuant to which Norgine is granted an exclusive license to distribute, market and sell our drug product for all indications in the European Economic Area, Switzerland, the United Kingdom, Australia and New Zealand.
O n January 13, 2025, we announced a License and Supply Agreement (the “Norgine Agreement”) with Norgine Pharma UK (“Norgine”), pursuant to which Norgine was granted an exclusive license to distribute, market and sell our drug product for all indications in the European Economic Area, Switzerland, the United Kingdom, Australia, and New Zealand.
See Note 5 for more information on our goodwill impairment test as of December 31, 2024. Intangible Assets, Net.
See Note 6 for more information on our goodwill impairment test as of December 31, 2025. Intangible Assets , Net .
Phase 3 Clinical Trial in Chronic Neutropenia We continue to progress our global, pivotal Phase 3 clinical trial, (the “4WARD” study) to evaluate the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without stable doses of G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections.
We continue to progress our global, pivotal Phase 3 clinical trial, (the “4WARD” trial) to evaluate the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without stable doses of granulocyte colony-stimulating factor (“G-CSF”) in people with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia (“CN”) who are experiencing recurrent and/or serious infections.
Our short-term and long-term funding requirements will depend on and could increase significantly as a result of many factors, including: • the scope, number, initiation, progress, timing, costs, design, duration, any potential delays, and results of clinical trials and nonclinical studies for our current or future product candidates, particularly our Phase 3 clinical trial of mavorixafor for the treatment of individuals with chronic neutropenic disorders; • the outcome, timing and cost of regulatory reviews, approvals or other actions to meet regulatory requirements established by the FDA and comparable foreign regulatory authorities, including the potential for the FDA or comparable foreign regulatory authorities to require that we perform more studies for our product candidates than those that we currently expect; • our ability to obtain marketing approval for our product candidates; • the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights covering our product and product candidates, including any such patent claims and intellectual property rights that we have licensed from Genzyme pursuant to the terms of our license agreement with Genzyme; • our ability to maintain, expand and defend the scope of our intellectual property portfolio, including the cost of defending intellectual property disputes, including patent infringement actions brought by third parties against us or our product or product candidates; • our ability to establish and maintain licensing, collaboration or similar arrangements on favorable terms and whether and to what extent we retain development or commercialization responsibilities under any new licensing, collaboration or similar arrangement; • the success of any other business, product or technology that we acquire or in which we invest; • the costs of acquiring, licensing or investing in businesses, product candidates and technologies; • the effect of competing technological and market developments; and • the costs to operate as a public company Until such time, if ever, as we can generate substantial product revenue, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, and marketing, distribution or licensing arrangements with third parties.
Our short-term and long-term funding requirements will depend on and could increase significantly as a result of many factors, including: • the scope, number, initiation, progress, timing, costs, design, duration, any potential delays, and results of clinical trials and nonclinical studies for our current or future product candidates, particularly our Phase 3 clinical trial of mavorixafor for the treatment of individuals with chronic neutropenic disorders; • the outcome, timing and cost of regulatory reviews, approvals or other actions to meet regulatory requirements established by the FDA and comparable foreign regulatory authorities, including the potential for the FDA or comparable foreign regulatory authorities to require that we perform more studies for our product candidates than those that we currently expect; • our ability to obtain marketing approval for our product candidates; • the cost of filing, prosecuting, defending and enforcing our patent claims and other intellectual property rights covering our product and product candidates, including any such patent claims and intellectual property rights that we have licensed from Genzyme pursuant to the terms of our license agreement with Genzyme; • our ability to maintain, expand and defend the scope of our intellectual property portfolio, including the cost of defending intellectual property disputes, including patent infringement actions brought by third parties against us or our product or product candidates; • our ability to establish and maintain licensing, collaboration or similar arrangements on favorable terms and whether and to what extent we retain development or commercialization responsibilities under any new licensing, collaboration or similar arrangement; • the success of any other business, product or technology that we acquire or in which we invest; • the costs of acquiring, licensing or investing in businesses, product candidates and technologies; • the effect of competing technological and market developments; and • the costs to continue operating as a public company. 75 Critical Accounting Policies and Significant Judgments and Estimates Our consolidated financial statements are prepared in accordance with generally accepted accounting principles in the United States.
Selling, general and administrative expenses also include direct and allocated facility-related costs, as well as professional fees for legal, patent, consulting, investor and public relations, accounting, and audit services. Selling, general and administrative expenses increased by $26.0 million to $61.5 million for the year ended December 31, 2024 as compared to $35.5 million in the prior year.
General and administrative expenses also include direct and allocated facility-related costs as well as professional fees for legal, patent, consulting, investor and public relations, accounting, and audit services. General and administrative expenses decreased by approximately $18.1 million in the year ended December 31, 2025 as compared to the prior year.
We update our estimates related to the probability and timing of achievement of the operational milestones each period until the award either vests or is forfeited . 74 The fair value of stock option grants is estimated on the date of grant using the Black-Scholes option-pricing model, which uses as inputs the fair value of our common stock and assumptions we make for the volatility of our common stock, the expected term of the stock options, the risk-free interest rate for a period that approximates the expected term of our stock options and an expected dividend yield.
The fair value of stock option grants is estimated on the date of grant using the Black-Scholes option-pricing model, which uses as inputs the fair value of our common stock and assumptions we make for the volatility of our common stock, the expected term of the stock options, the risk-free interest rate for a period that approximates the expected term of our stock options and an expected dividend yield.
Unless and until we reach profitability in the future, we will require additional capital to fund our operations, which could be raised through a combination of equity offerings, debt financings, other third-party funding, marketing and distribution arrangements, or other collaborations and strategic alliances.
Until we reach profitability, we will need to raise additional capital, which cannot be assured, to fund our operations and meet our financial obligations beyond this period. Such additional capital could be raised through a combination of equity offerings, debt financings, other third-party funding, marketing and distribution arrangements, or other collaborations and strategic alliances.
In January 2022, we raised $3.0 million from the sale of shares of our common stock through the LPC Agreement. 70 Public and Private Equity Offerings — Over the past several years we have funded our operations primarily from sales of common stock, warrants and prefunded warrants through both public offerings and private placements.
Over the past several years we have funded our operations primarily from sales of common stock, warrants and prefunded warrants through both public offerings and private placements.
Any such adjustments are recorded on a cumulative catch-up basis in the period of the adjustment. Smaller Reporting Company Status We are a smaller reporting company as defined in the Exchange Act.
Any such adjustments are recorded on a cumulative catch-up basis in the period of the adjustment. Smaller Reporting Company Status We are a smaller reporting company (“SRC”) as defined by Rule 12b-2 of the Exchange Act and Item 10(f)(1) of Regulation S-K.
FDA approved our NDA for mavorixafor, which is being marketed in the U.S. under the trade name XOLREMDI, for use as an oral, once-daily therapy in patients aged 12 years of age and older with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, to increase the number of circulating mature neutrophils and lymphocytes.
We have one commercially approved product, XOLREMDI ® (mavorixafor), which has received accelerated approval in the United States from the FDA for use as an oral, once-daily therapy in patients 12 years of age and older with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, to increase the number of circulating mature neutrophils and lymphocytes.
For the year ended December 31, 2024, our net losses were $37.5 million and net cash used in operating activities of $130.9 million.
Net cash used in operating activities for the year ended December 31, 2024 was $130.9 million, primarily resulting from our operating losses of $142.5 million adjusted for noncash expenses of $8.9 million and changes in our operating assets and liabilities of $2.7 million.
Non-cancellable lease obligations are $1.4 million in 2025, $1.3 million in 2026, and $0.3 million thereafter. 72 Funding Requirements Based on our cash, cash equivalents and marketable securities on hand as of December 31, 2024 and our current operating plan, we believe that our cash, cash equivalents and marketable securities will not allow us to fund operations for at least the next 12 months.
Capital Resources Based on our cash, cash equivalents and marketable securities on hand as of March 17, 2026, and our current operating plan, we believe that our cash, cash equivalents and marketable securities will allow us to fund operations for at least the next 12 months.
We may also incur additional costs not currently contemplated due to events that may occur as a result of, or that are associated with, the actions described for the 2025 Restructuring above.
We may also incur additional costs not currently contemplated due to events that may occur as a result of, or that are associated with, the actions described above. Regulatory Update and Out-License Agreements In early 2025, we submitted a Marketing Authorization Application (“MAA”) to the EMA seeking regulatory approval to commercialize mavorixafor for WHIM syndrome in the European Union.
Hercules Loan Agreement — We entered into a Loan and Security Agreement, as most recently amended in August 2023 with Hercules Capital, Inc, (the “Hercules Loan Agreement”). The Hercules Loan Agreement provides for an aggregate term loan facility of up to $107.5 million, less $75.0 million borrowed to date.
We are a party to a loan and security agreement (the “Hercules Loan Agreement”), which provides for a term loan facility of up to $107.5 million, under which we have borrowed an aggregate of $75.0 million of term loans to date, representing the maximum borrowings as of December 31, 2025.
The strategic restructuring activities include (i) discontinuing of research efforts, (ii) closing of our facility in Vienna, Austria, (iii) pausing pre-clinical drug candidate programs, (iv) scaling the U.S. commercial field team and supporting roles across our business and (v) streamlining other spending to support the ongoing clinical development of mavorixafor for the larger population of those with chronic neutropenia.
As part of this restructuring, we (i) implemented a net reduction of our employee headcount by 43 employees, representing approximately 30% of our total workforce, including our U.S. commercial field team, (ii) commenced the closure of our research and development facility in Vienna, Austria, (iii) paused our pre-clinical drug candidate programs and (iv) streamlined other spending to support the ongoing clinical development of mavorixafor for the larger population of those with chronic neutropenia.
The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants. We believe we are on track to complete enrollment in the 4WARD study in the third or fourth quarter of 2025.
The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll up to 176 patients, with full enrollment expected in the third quarter of 2026. The U.S.
In May 2023, we sold shares of common stock and, in lieu of common stock, pre-funded warrants to purchase shares of common stock in a private placement for gross proceeds of $65.0 million, before offering expenses.
Most recently in August 2025, we sold shares of common stock and, in lieu of common stock to certain investors, pre-funded warrants to purchase shares of common stock in a private placement offering (the “Q3 2025 PIPE”) resulting in net proceeds of $81.0 million, after deducting placement agent fees and offering expenses.
Investing Activities: During the year ended December 31, 2024, net cash provided by investing activities was $67.0 million, primarily due $105.0 million of cash from the sale of a PRV, partially offset by net investments in short-term marketable securities.
Investing Activities During the year ended December 31, 2025, cash provided by investing activities of $8.2 million primarily includes net sales of short-term marketable securities.
During the current year, our U.S. entity generated taxable income due to the sale of a PRV voucher, which generated $105.0 million of taxable income, and product revenue, partially offset by available deductions, net operating loss carryforwards and available research and development credits.
Our income tax provision for the year ended December 31, 2024 of $0.3 million reflected U.S. federal and state taxable income that included the sale of a priority review voucher, generating $105.0 million of taxable income, partially offset by available deductions, net operating loss carryforwards, which were limited under IRC 382 due to several qualifying ownership changes, and available research and development credits.
Research and development expenses related to our X4P-002 and X4P-003 programs was not significant in 2024 or 2023 relative to our overall research and development expenses. Selling, General and Administrative Expenses Selling, general and administrative expenses consist primarily of salaries and related costs, including stock-based compensation for personnel in sales and marketing, executive, finance and administrative functions.
The overall decrease in research and development expenses in the current year was partially offset by higher clinical costs associated with our 4WARD trial. 72 General and Administrative Expenses General and administrative expenses consist primarily of salaries and related costs, including stock-based compensation, for personnel in executive, finance, and administrative functions.
Recently Issued Accounting Pronouncements See Note 2 to the consolidated financial statements. ITEM 7A. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK As a smaller reporting company, we are not required to provide disclosure for this Item.
Item 7A. Quantitative and Qualitative Disclosures About Market Risk As an SRC, we are not required to provide the information requested by this Item.
There was no cost of revenue in prior periods. Cost of revenue primarily consists of $0.5 million of amortization of an intangible asset related to accrued and paid milestone payments associated with our Genzyme license agreement, $0.2 million of sales-based royalty payments accrued and paid under our Genzyme license agreement, and $0.1 million of drug product costs.
Operating Cost and Expenses: Cost of Revenue Cost of revenue primarily consists of amortization of an intangible asset related to accrued and paid milestone payments associated with our license agreement (the “Genzyme Agreement”) with Genzyme Corporation (“Genzyme”), a wholly owned subsidiary of Sanofi, and sales and sublicense-based royalty payments due thereunder.
In December 2022, we sold shares of common stock and, in lieu of common stock, pre-funded warrants to purchase shares of common stock in a public offering for gross proceeds of $65.1 million, before offering expenses.
Capital Requirements During the year ended December 31, 2025, we sold shares of common stock and, in lieu of common stock to certain investors, pre-funded warrants to purchase shares of common stock in a private placement offering and an underwritten public offering for aggregate net proceeds of $226.6 million, after placement agent and underwriting fees and offering expenses.
To date, we have sold approximately $14.3 million of our common stock, net of offering costs, under the ATM Sales Agreement.
To date, for the year ended December 31, 2025 and for the three months ended December 31, 2025, we have sold $24.2 million, $9.7 million, and $0.7 million, respectively, of our common stock, net of offering costs, under the ATM.
Research and Development Expenses Research and development expenses consist primarily of costs incurred in connection with the development of our product candidates, including employee salaries and related expenses for personnel in our clinical operations, biostatistics, medical affairs, manufacturing and technical, quality and regulatory affairs departments; external expenses incurred in connection with the clinical development of our product candidates, including under agreements with third parties, such as consultants and contract research organizations (“CROs”); the cost of manufacturing drug products for use in our clinical trials, including under agreements with third parties, such as consultants and contract manufacturing organizations (“CMOs”); facilities, depreciation and other expenses, which include direct or allocated expenses for rent and maintenance of facilities and insurance; and costs related to compliance with regulatory requirements.
Research and Development Expenses Research and development expenses consist primarily of costs incurred in connection with the development of our product candidates, including employee salaries and related expenses, clinical development expenses, internal and third-party costs of manufacturing our drug products for use in our clinical trials.
For example, In March 2022, we sold shares of common stock and, in lieu of common stock, pre-funded warrants to purchase shares of common stock in a private placement for gross proceeds of $3.0 million, before offering expenses.
Private Placement Financing and Management Changes During the third quarter of 2025, we sold shares of common stock and pre-funded warrants to purchase shares of common stock in a private placement that resulted in net proceeds of approximately $81.0 million, after deducting placement agent fees and other expenses.
Overview We are a biopharmaceutical company discovering, developing, and commercializing novel therapeutics for the treatment of rare diseases and those with limited treatment options, with a focus on conditions resulting from dysfunction of the immune system. On April 29, 2024, we announced that the U.S.
Risk Factors. Overview We are a biopharmaceutical company developing, and commercializing novel therapeutics for the treatment of rare hematology diseases.
We have an ATM Sales Agreement with the Sales Agents, pursuant to which we have offered to sell and continue to offer to sell, at our sole discretion through one or more of the Sales Agents, shares of our common stock.
ATM Sales Agreement. We are party to a Controlled Equity Offering SM Sales Agreement (“ATM”), dated as of August 7, 2020, pursuant to which we may offer and sell shares of our common stock through one or more investment banks.
ITEM 7. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS You should read the following discussion and analysis of our financial condition and results of operations together with our consolidated financial statements and the related notes and the other financial information included elsewhere in this Annual Report.
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS The following discussion and analysis is intended to provide material information around events and uncertainties known to management that are relevant to an assessment of the financial condition and results of operations of X4 Pharmaceuticals and should therefore be read in conjunction with our audited Consolidated Financial Statements and the related notes thereto and other disclosures included as part of this Annual Report on Form 10-K, including the disclosures under Part I, Item 1A.
Our tax provision in the year ended December 31, 2023 was related to our U.S. security corporation, which holds our investment portfolio, and our Austrian subsidiary.
Provision for Income Taxes Our income tax provision of $41.0 thousand for the year ended December 31, 2025, which was primarily related to our Austrian subsidiary, was lower than our income tax provision in the prior year.
Cash used in investing activities for the year ended December 31, 2023 was primarily related to net investments in short-term marketable securities. Financing Activities: During the year ended December 31, 2024, net cash provided by financing activities was $20.3 million, consisting primarily of proceeds borrowed on our Hercules Loan Agreement.
Cash provided by financing activities for the year ended December 31, 2024 included $20.0 million of new borrowings on our loan facility.
We estimate that the workforce reduction will be substantially completed in the first quarter of 2025. We estimate that it will incur charges of approximately $3.0 million for severance and other employee termination-related costs, primarily in the first quarter of 2025.
As part of this initiative, we further reduced our workforce by approximately 50%. We incurred expenses of approximately $4.9 million during the third quarter for severance and other employee termination-related costs related to this strategic restructuring. This workforce reduction was substantially completed in the third quarter of 2025.
We will manufacture and supply drug product to Norgine for a supply price derived from our product manufacturing cost plus a low double-teen digit margin. 67 Results of Operations Comparison of the Years Ended December 31, 2024 and 2023 The following table summarizes the results of our operations for the periods indicated: Year Ended December 31, 2024 2023 Change (in thousands) Product revenue, net $ 2,557 $ — $ 2,557 Costs and operating expenses: Cost of revenue 797 — 797 Research and development 81,643 72,017 9,626 Selling, general and administrative 61,518 35,505 26,013 Gain on sale of non-financial asset (105,000) — (105,000) Total operating expenses 38,958 107,522 (68,564) Loss from operations (36,401) (107,522) 71,121 Total other (expense) income, net (739) 6,433 (7,172) Loss before provision for income taxes (37,140) (101,089) 63,949 Provision for income taxes 310 78 232 Net loss $ (37,450) $ (101,167) $ 63,717 Product Revenue, Net Net revenue from the sale of our drug product was $2.6 million for the year ended December 31, 2024.
Results of Operations The following table summarizes the results of our operations for the years ended December 31, 2025 and 2024: Year Ended December 31, (in millions) 2025 2024 Change Revenue $ 35.1 $ 2.6 $ 32.5 Cost and operating expenses: Cost of revenue 5.8 0.8 5.0 Research and development 72.7 81.6 (8.9) General and administrative 43.5 61.6 (18.1) Gain on sale of non-financial asset — (105.0) 105.0 Total operating expenses 122.0 39.0 83.0 Loss from operations (86.9) (36.4) (50.5) Total other income (expense), net 7.7 (0.7) 8.4 Loss before income taxes (79.2) (37.1) (42.1) Provision for income taxes — (0.4) 0.4 Net loss $ (79.2) $ (37.5) $ (41.7) Revenue License and Other In January 2025, we granted an exclusive license to Norgine to distribute, market and sell our product for all indications in the European Economic Area, Switzerland, the United Kingdom, Australia and Ne w Zealand following regulatory approval.
We capitalized as intangible assets certain milestone payments due under our key in-license agreement. 68 Year Ended December 31, 2024 2023 Change (in thousands) Direct research and development expenses by product candidate: Mavorixafor (X4P-001) $ 41,483 $ 41,163 $ 320 X4P-002 159 (34) 193 X4P-003 150 75 75 Unallocated expense 39,851 30,813 9,038 Total research and development expenses $ 81,643 $ 72,017 $ 9,626 Research and development expenses increased by $9.6 million for the year ended December 31, 2024, as compared to the prior year primarily due to an increase in unallocated expense .
Year Ended December 31, (in millions) 2025 2024 Change Direct research and development expenses by product candidate: Mavorixafor $ 41.4 $ 41.5 $ (0.1) X4P-002 — 0.2 (0.2) X4P-003 — 0.2 (0.2) Unallocated expense 31.3 39.7 (8.4) Total research and development expenses $ 72.7 $ 81.6 $ (8.9) Research and development expenses decreased by $8.9 million in the year ended December 31, 2025 as compared to the prior year primarily due to decreases in spending associated with our 2025 strategic restructurings, including lower spending on non-clinical programs, lower consulting fees, lower drug substance manufacturing costs, and lower regulatory costs.
These increases were partially offset by higher interest income earned in the current year on our marketable security portfolio as compared to the prior year. Income Taxes For the year ended December 31, 2024, we recorded an income tax provision of $0.3 million related to our U.S. operations and related to our Austrian subsidiary.
Other Income (Expense), Net Year Ended December 31, 2025 2024 change (in millions) Interest income $ 4.6 $ 5.8 $ (1.2) Interest expense (8.9) (8.8) (0.1) Change in fair value of Class C warrant liability 12.8 1.9 10.9 Other (expense) income, net (0.8) 0.4 (1.2) Total other income (expense), net $ 7.7 $ (0.7) $ 8.4 Other income (expense), net, increased approximately $8.4 million in the year ended December 31, 2025 as compared to the prior year primarily due to higher gains in the current year on fair value adjustments related to our Class C warrants, partially offset by lower interest income earned on our marketable security investment portfolio .
In June 2022, we sold shares of common stock and, in lieu of common stock, pre-funded warrants to purchase shares of common stock in a private placement for gross proceeds of $55.7 million, before offering expenses.
Q4 2025 Equity Financing In October 2025, we closed an underwritten public offering of our common stock and, in lieu of common stock to certain investors, pre-funded warrants to purchase shares of common stock, raising net proceeds of $145.6 million, net of underwriting discounts and estimated offering expenses.
During the year ended December 31, 2023, net cash provided by financing activities was $88.5 million, primarily due to $60.0 million of proceeds from the sale of our common stock and pre-funded warrants, borrowing net of repayments and issuance costs of $19.8 million on our Hercules Loan Agreement, and $8.7 million of proceeds from warrant exercises.
During the year ended December 31, 2024, cash provided by investing activities was $67.0 million, primarily due to the receipt of $105.0 million of cash from the sale of a PRV, partially offset by net investments in short-term marketable securities. 74 Financing Activities During the year ended December 31, 2025, cash provided by financing activities of $238.6 million was primarily due to net proceeds of $226.6 million from the sale our common stock and pre-funded warrants in private placement and public offerings and $11.9 million from sales of our common stock through our ATM program and purchase agreement with Lincoln Park Capital Fund LLC.
The increase in selling, general and administrative expense was primarily due to an increase of $15.2 million in selling and marketing expenses, including increases in sales and marketing personnel and external marketing costs to support the ongoing launch activities of our approved product, XOLREMDI in the U.S.
The decrease to general and administrative expenses was primarily due to an $8.0 million decrease in compensation expense due to lower head count in our sales, general and administrative functions, and a decrease of $9.0 million in sales and marketing expenses in the current year as compared to the prior year during which we incurred commercialization sales and marketing launch costs related to XOLREMDI.
Removed
Some of the information contained in this discussion and analysis or set forth elsewhere in this Annual Report, including information with respect to our plans and strategy for our business and related financing, includes forward-looking statements that involve risks and uncertainties.
Added
Food and Drug Administration (“FDA”) has granted Fast Track designation to mavorixafor for the treatment of CN, which is defined as periods lasting more than three months persistently or intermittently where there are abnormally low levels of neutrophils circulating in the blood, and may be idiopathic (of unknown origin), cyclic (episodes typically occurring every three weeks), or congenital (of genetic causation).
Removed
As a result of many factors, including those factors set forth in the “Risk Factors” section of this Annual Report, our actual results could differ materially from the results described in or implied by these forward-looking statements.
Added
CN disorders are rare blood conditions similarly characterized by increased risks of infections and cancer due to abnormally low levels of neutrophils in the body. In all cases, the CXCL12/CXCR4 pathway is the key regulator of neutrophil release from the bone marrow.
Removed
For the discussion of the financial condition and results of operations for the year ended December 31, 2023 compared to the year ended December 31, 2022, refer to “Management’s Discussion and Analysis of Financial Condition and Results of Operations” to our Annual Report on Form 10-K filed with the SEC on March 21, 2024.
Added
WHIM syndrome is a rare combined primary immunodeficiency and CN disorder. In connection with our long term strategy to successfully complete the 4WARD Phase 3 trial in patients with moderate and severe CN, we are no longer prioritizing investment in the WHIM indication.
Removed
WHIM syndrome (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) is a rare combined primary immunodeficiency and chronic neutropenic disorder. Concurrent with the U.S. approval of XOLREMDI and pursuant to its Rare Pediatric Disease designation, the FDA granted us a PRV that we sold to another drug sponsor shortly thereafter.
Added
Pursuant to registration rights agreements, we registered the shares of common stock issued and issuable under pre-funded warrants under a registration statement on Form S-3 that was declared effective by the SEC on September 17, 2025.
Removed
XOLREMDI Commercial Launch We are currently engaged in our U.S. launch of XOLREMDI in WHIM syndrome, continuing our engagements with physicians and rare disease patient advocacy organizations and our disease-awareness campaign to further the understanding of WHIM syndrome and educate patients and physicians on the importance and benefits of early diagnosis.
Added
Concurrent with the financing and effective August 12, 2025, our former President and Chief Executive Officer, Paula Ragan, PhD, and Chief Financial Officer, Adam Mostafa, stepped down from their respective roles and their employment was terminated. Dr. Ragan also resigned from the Company’s Board of Directors (the “Board”), and Michael Wyzga transitioned from Board Chair to Lead Independent Director.
Removed
We have entered into agreements with a third-party logistics organization and a specialty pharmacy to support the distribution of XOLREMDI in the U.S., to mitigate barriers to product access, and to provide a suite of patient support services to help patients through their treatment journey.
Added
The Board appointed Adam R. Craig, M.D., Ph.D, MBA as Executive Chairman, John Volpone as President and subsequently as Chief Operating Officer, and David Kirske as Chief Financial Officer.
Removed
We submitted a Marketing Authorisation Application (“MAA”) to the European Medicines Agency (“EMA”) in early 2025 seeking regulatory approval to commercialize mavorixafor for WHIM syndrome outside of the U.S. Such MAA was accepted for processing by the EMA in January 2025.
Added
Strategic Restructurings • In the first quarter of 2025, we implemented a strategic restructuring of our business operations, workforce and capital spending to focus efforts on advancing mavorixafor to treat patients with CN.
Removed
In February 2025, we announced that we had entered into an agreement with taiba rare to distribute and commercialize XOLREMDI for the treatment of WHIM syndrome in select Middle East and North African countries. We continue to explore additional potential commercial and distribution opportunities in geographies where we may be able to efficiently leverage any of our regulatory approvals.
Added
We incurred charges of approximately $2.1 million for severance and other employee termination-related costs related to this strategic restructuring. 70 • In the third quarter 2025, we announced an additional strategic restructuring designed to further sharpen operational focus and align resources with our long‑term strategy to successfully complete the 4WARD Phase 3 trial in patients with moderate and severe CN.
Removed
The U.S. approval of XOLREMDI in the WHIM syndrome indication is the first for mavorixafor, which is an orally active, selective antagonist of chemokine receptor CXCR4, a key regulator of the movement of immune cells throughout the body.
Added
These strategic restructuring actions have allowed us to decrease our operating expenses, including research and development and general and administrative expenses, from $143.2 million in 2024 to $116.2 million in 2025.
Removed
Due to its ability to increase the mobilization of white blood cells from the bone marrow into the bloodstream, we believe that mavorixafor has the potential to provide therapeutic benefit across a variety of immune system disorders in addition to WHIM syndrome.
Added
Such MAA was validated for processing by the EMA in January 2025. In February 2026, the EMA’s Committee for Medicinal Products for Human Use adopted a positive opinion recommending the grant of marketing authorization, under exceptional circumstances, for mavorixafor for the treatment of WHIM syndrome in the European Union.
Removed
Phase 2 Clinical Study in Chronic Neutropenia Following positive results from a Phase 1b clinical study of a single dose of mavorixafor in people with idiopathic, cyclic, and congenital chronic neutropenia (“CN”), we recently completed and announced positive results from a Phase 2 clinical study evaluating the durability of effect, safety, and tolerability of chronic dosing of once-daily oral mavorixafor with or without concurrent treatment with injectable granulocyte colony-stimulating factor (“G-CSF”) in the same patient population. 66 The results from the completed six-month study showed that once-daily oral mavorixafor was generally well tolerated and durably increased participants’ absolute neutrophil counts (“ANC”) both as a monotherapy and in combination with G-CSF, the only therapy approved in the U.S. for severe chronic neutropenia.
Added
The positive opinion has been submitted to the European Commission for review, and we expect the European Commission to issue a final approval decision in the second quarter of 2026.
Removed
In addition, when tested in combination with G-CSF, mavorixafor treatment enabled physicians to substantially reduce G-CSF dosing while maintaining normal mean ANC levels.
Added
For the year ended December 31, 2025, we recognized $27.6 million for the delivery of the license and $1.0 million for the provision of research and development services to Norgine.
Removed
We believe that successfully developing and commercializing mavorixafor to provide a new therapeutic option to individuals diagnosed with certain immunodeficiencies has the potential to revolutionize the current treatment landscape, which is principally served by injectable and infused therapies. 2025 Restructuring In February 2025, we implemented a strategic restructuring of our business operations, workforce and capital spending to focus efforts on advancing mavorixafor to treat those with chronic neutropenia, while also optimizing its U.S. promotion of XOLREMDI (the “2025 Restructuring”).
Added
We had no license or other revenue during the year ended December 31, 2024. 71 Product Revenue, Net We began recognizing product sales in June 2024 following FDA approval of XOLREMDI on April 29, 2024 and its subsequent commercial launch in the United States.
Removed
As part of the 2025 Restructuring, we implemented a net reduction of our employee headcount by 43 employees, or approximately 30% of our total workforce.
Added
Net product sales were as follows for years ended December 31, 2025 and 2024, and for each of the quarterly periods therein.
Removed
We expect the 2025 Restructuring will decrease annual spending by $30 to 35 million and believe it will have sufficient funds to support operations into the first half of 2026.

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