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Item 2 — Management's Discussion and Analysis
Catalyst Pharmaceuticals, Inc. · 10-Q · Q1 FY2026 · Period ended Mar 31, 2026
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Introduction
Management’s Discussion and Analysis of Financial Condition and Results of Operations (MD&A) is intended to provide an understanding of our financial condition, changes in financial condition and results of operations. The discussion and analysis is organized as follows:
•Overview. This section provides a general description of our business and information about our business that we believe is important in understanding our financial condition and results of operations.
•Basis of Presentation. This section provides information about key accounting estimates and policies that we followed in preparing our condensed consolidated financial statements for the first quarter of fiscal 2026.
•Critical Accounting Policies and Estimates. This section discusses those accounting policies that are both considered important to our financial condition and results of operations and require significant judgment and estimates on the part of management in their application. All of our significant accounting policies, including the critical accounting policies, are also summarized in the notes to our interim condensed consolidated financial statements that are included in this report.
•Results of Operations. This section provides an analysis of our results of operations for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025.
•Liquidity and Capital Resources. This section provides an analysis of our cash flows, capital resources, off-balance sheet arrangements, and outstanding commitments.
•Caution Concerning Forward-Looking Statements. This section discusses how certain forward-looking statements made throughout this MD&A and in other sections of this report are based on management’s present expectations about future events and are inherently susceptible to uncertainty and changes in circumstance.
OVERVIEW
We are a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult to treat diseases. We currently sell three commercial stage drug products, FIRDAPSE® (amifampridine), AGAMREE® (vamorolone), and FYCOMPA® (perampanel) in the United States. We are also currently seeking to further expand our product portfolio, with a focus on acquiring the rights to immediately and near-term accretive assets to treat rare (orphan) diseases across therapeutic areas, including clinical-stage opportunities with established proof of concept and a clear regulatory pathway to approval. With an unwavering patient focus embedded in everything we do, we are committed to providing innovative, best-in-class medications with the hope of making a meaningful positive impact on those affected by these conditions.
Currently, we have a total of 74 field personnel supporting FIRDAPSE® and AGAMREE® which includes Regional Account Managers, Area Business Directors, National Account Directors, Thought Leader Liaisons and Area Marketing Directors. This also includes 17 Patient Access Liaisons and insurance navigation support personnel who support both FIRDAPSE® and AGAMREE® and 15 Medical Science Liaisons who help educate the medical community about scientific literature concerning our drug products and the diseases they treat.
When we launched AGAMREE® in March 2024, we utilized the FIRDAPSE® commercial and medical field-based forces to market AGAMREE®. Effective April 1, 2025, we separated these field-based forces into two distinct units, one for each function expressly focused on supporting FIRDAPSE® and one for each function expressly focused on supporting AGAMREE®, respectively. This strategic change was made in an effort to allow us to better focus the support for each product. Further, we have two National Account Directors on our commercial team who exclusively focus on the oncology market for Lambert-Eaton Myasthenic Syndrome (LEMS) patients who also have cancer.
Recent Developments
On May 6, 2026, we entered into an Agreement and Plan of Merger (the Merger Agreement) with Angelini Pharma S.p.A., an Italian Società per azioni (Angelini Pharma or Parent) and Angelini Cielo Inc., a Delaware corporation and wholly-owned subsidiary of Parent (Merger Sub), providing for the merger of Merger Sub with and into the Company (the Merger), with the Company surviving the Merger as a wholly-owned subsidiary of Parent.
For further information about the Merger Agreement and the Merger, see the Current Report on Form 8-K that we filed with the SEC on May 7, 2026 reporting with more particularity on the terms of the Merger Agreement and the proposed Merger.
FIRDAPSE®
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On November 28, 2018, we received approval from the U.S. Food & Drug Administration (FDA) for our new drug application (NDA) for FIRDAPSE® Tablets 10 mg for the treatment of adult patients (ages 17 and above) with LEMS, and in January 2019, we launched FIRDAPSE® in the U.S. Further, on September 29, 2022, the FDA approved our supplemental NDA (sNDA) to expand the indicated age range for FIRDAPSE® Tablets 10 mg for the treatment of LEMS to include pediatric patients six years of age and older. Additionally, on May 30, 2024, the FDA approved our sNDA increasing the indicated maximum daily dosage of FIRDAPSE® tablets for the treatment of patients with LEMS from 80 mg to 100 mg. We are also planning to evaluate FIRDAPSE® in the future for the treatment of other diseases.
We sell FIRDAPSE® in the U.S. through a field-based force experienced in neurologic, central nervous system or rare disease products consisting at this time of approximately 26 field personnel, including sales (Regional Account Managers and Area Business Directors), National Account Directors and Thought Leader Liaisons. This includes two National Account Directors and three Thought Leader Liaisons who exclusively focus on the oncology market for LEMS patients with cancer. We also use non-personal promotion to reach the 20,000 neurologists who are potential LEMS treaters and the 16,000 oncologists who might be treating a LEMS patient who also has cancer (principally small cell lung cancer). Finally, we make available for online ordering a no-cost LEMS voltage gated calcium channel (VGCC) antibody diagnostic testing program for use by physicians who suspect that one of their patients may have LEMS and wish to reach a definitive diagnosis.
Further, we are continuing to expand our digital and social media activities to introduce our products and services to potential patients and their healthcare providers. We also work with several rare disease advocacy organizations (including the Myasthenia Gravis Foundation of America, the National Organization for Rare Disorders, and the LEMS Family Association) to help increase awareness and level of support for patients living with LEMS and to provide education for the physicians who treat this rare disease and the patients they treat.
On August 6, 2025, we reported that the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Small Cell Lung Cancer (SCLC) now include additions involving LEMS, amifampridine, and the tests for PQ- and N-type VGCC antibodies. The updated NCCN Guidelines® for SCLC relating to LEMS now include symptom specificity—characterized by proximal muscle weakness and autonomic dysfunction. Under "Signs and Symptoms of Small Cell Lung Cancer” (SCL-A 2 of 2), the guidelines recommend diagnosis through a neurological evaluation, ideally in consultation with a neurologist, which may include testing for PQ- and N-type VGCC antibodies. Additionally, under “Principles of Supportive Care” (SCL-D), the guidelines recommend that amifampridine should be considered as a treatment in consultation with neurology. We are currently working to increase awareness of these oncology clinical practice guidelines among oncology practices that treat patients with SCLC.
We are supporting the distribution in the U.S. of FIRDAPSE® through Catalyst Pathways®, our personalized treatment support program for patients who enroll in it. Catalyst Pathways® is a single source for personalized treatment support, education and guidance through the challenging dosing and titration regimen required to reach an effective therapeutic dose. The program also includes distributing the drug through a very small group of exclusive specialty pharmacies (primarily AnovoRx), which is consistent with the way that most drug products for ultra-orphan diseases are distributed and dispensed to patients. We believe that by using specialty pharmacies in this way, the task, which can be difficult, of navigating the health care system is far better for the patient needing treatment for their rare disease and the health care community in general.
In order to help patients with LEMS afford their medication, we, like other pharmaceutical companies that market drug products for orphan and ultra-orphan, rare diseases, have developed an array of financial assistance programs intended to reduce out-of-pocket costs in order to make FIRDAPSE® accessible and affordable. A co-pay assistance program has been designed to reduce commercial patients’ out of pocket costs to as little as $0 whenever possible (currently an average of less than $2 per month). Our co-pay assistance programs, including the one for FIRDAPSE®, are not available to patients enrolled in state or federal healthcare programs, including Medicare, Medicaid, Department of Veterans Affairs (VA), Department of Defense (DoD), or TRICARE. However, we have, at times, donated funds to one or more qualified, independent charitable foundations dedicated to providing assistance to any U.S. LEMS patients in financial need who meet those independent organizations' guidelines. In addition, we have a program in place to help patients who are uninsured and underinsured. Subject to compliance with applicable regulatory requirements, our goal is that no LEMS patient is ever denied access to their medication for financial reasons.
FIRDAPSE® is currently marketed for the treatment of LEMS in Canada through our exclusive sublicensee, KYE Pharmaceuticals, Inc. (KYE). We supply product to KYE at agreed upon prices and we are also eligible to earn sales milestones and sales royalties based on net revenues from sales of the product in Canada. Further, FIRDAPSE® is commercially available (since January 21, 2025) for the treatment of LEMS in Japan through our sublicensee for Japan, DyDo Pharma, Inc. (DyDo). We generate revenue from DyDo through the receipt of additional milestone payments, as such milestones are achieved, and a transfer price on the product supplied by us to DyDo (in lieu of royalties).
We control six U.S. patents for FIRDAPSE® that are listed in the FDA's Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book), the earliest of which expires in 2032 and the latest of which expires in 2037. Generic drug manufacturers were permitted to submit applications for the product challenging our patents starting in 2023 and the FDA is now permitted to approve such ANDA products following the expiration of our orphan drug exclusivity (ODE) on November 26, 2025, subject to any pending
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30-month stays of approval as required under the Drug Price Competition and Patent Term Restoration Act of 1984 (Hatch-Waxman Amendments).
With respect to ANDA filers, in January 2023, we received Paragraph IV Certification Notice Letters from three generic drug manufacturers (Teva Pharmaceuticals USA, Inc. and Teva Pharmaceuticals, Inc. (collectively Teva), Hetero USA, Inc. (Hetero), and Lupin Pharmaceuticals, Inc. (Lupin)) advising that they each had submitted an ANDA to the FDA seeking authorization from the FDA to manufacture, use or sell a generic version of FIRDAPSE® in the U.S. The notice letters each alleged that the six patents protecting FIRDAPSE® that are listed in the Orange Book in connection with FIRDAPSE® are not valid, not enforceable, and/or will not be infringed by the commercial manufacture, use or sale of the proposed product described in these ANDA submissions. Under the Federal Food, Drug, and Cosmetic Act (FDCA), as amended by the Drug Price Competition and Patent Term Restoration Act of 1984, we had 45 days from receipt of the notice letters to determine if there were grounds to bring a lawsuit and, if so, to commence patent infringement lawsuits against these generic drug manufacturers in a federal district court, which would trigger a statutory stay precluding the FDA from final approval of the subject ANDA until May 26, 2026 or entry of judgment holding the patents invalid, unenforceable, or not infringed, whichever occurs first in all cases. In that regard, after conducting the necessary due diligence, we filed lawsuits on March 1, 2023 in the U.S. District Court for the District of New Jersey against each of the three generic drug manufacturers who notified us of their ANDA submissions, thus triggering the stay.
In June 2024, Lupin converted five of its Paragraph IV Certifications in its ANDA to Paragraph III certifications acknowledging the validity and their ANDA’s infringement of five of those patents, the latest ending in 2034. We subsequently dismissed all of our claims against Lupin related to those five patents but maintained our claim against Lupin for the remaining Paragraph IV certification for U.S. Patent No. 10,626,088, which is the patent expiring in 2037.
On January 8, 2025, we reached a settlement with Teva in which Teva agreed not to market a generic version of FIRDAPSE® in the U.S. any earlier than February 25, 2035, if approved by the FDA, unless certain limited circumstances customarily included in these types of agreements occur. In accordance with the settlement agreement, the parties terminated all ongoing patent litigation between us and Teva regarding FIRDAPSE® patents pending in the U.S. District Court for the District of New Jersey. Later, on August 26, 2025, we reached a settlement with Lupin on substantially the same terms for market entry.
The pending FIRDAPSE® patent litigation against the remaining defendant, Hetero (relating to the FIRDAPSE® Orange Book-listed patents expiring in 2032, 2034 and 2037) was settled on May 6, 2026. This Settlement Agreement resolves the patent litigation brought by SERB and us in response to Hetero’s ANDA seeking approval to market a generic version of FIRDAPSE® prior to expiration of the applicable patents. Pursuant to the terms of the Settlement Agreement, Hetero will not market its generic version of FIRDAPSE® in the United States any earlier than January 2035, if approved by the FDA, unless certain limited circumstances customarily included in these types of agreements occur. In accordance with the Settlement Agreement, the parties will terminate all ongoing patent litigation between us and Hetero regarding FIRDAPSE® patents pending in the U.S. District Court for the District of New Jersey. We also agreed to pay a litigation avoidance fee in the amount of $11.0 million to Hetero as part of the settlement. As required by law, Hetero and we will submit the Settlement Agreement to the U.S. Federal Trade Commission and the U.S. Department of Justice for review.
AGAMREE®
On June 19, 2023, we entered into a License and Collaboration Agreement (AGAMREE® License Agreement) and an Investment Agreement (Investment Agreement) with Santhera Pharmaceuticals Holding AG (collectively, Santhera). Under the AGAMREE® License Agreement, we contracted to obtain an exclusive North America license, manufacturing and supply agreement for product candidate, AGAMREE®, a novel corticosteroid for the treatment of Duchenne muscular dystrophy (DMD). We also obtained the North American rights to any future approved indications for AGAMREE®. Under the Investment Agreement, we agreed to make a strategic investment into Santhera. Both transactions closed on July 18, 2023.
Finally, under our AGAMREE® License Agreement with Santhera, we agreed to purchase commercial supply of AGAMREE® from Santhera at agreed upon prices until we completed our process to transition to our own direct supplier. In that regard, we are in the process of transitioning final goods manufacturing to a U.S. location (estimated to be completed by the end of 2026).
Concurrent with the closing of the AGAMREE® License Agreement, we made a strategic investment into Santhera in which we acquired 1,414,688 of Santhera’s ordinary shares (representing approximately 11.26% of Santhera’s outstanding ordinary shares immediately following the transaction) at an investment price of CHF 9.477 per share, with the approximately $15.7 million USD in equity investment proceeds to be used by Santhera for Phase IV studies of AGAMREE® in DMD and future development of additional indications for AGAMREE®. On April 29, 2026, we filed a disclosure of significant shareholdings under the Swiss Financial Markets Infrastructure Act (FMIA) to disclose that our 1,414,688 shares of Santhera's ordinary shares represented 9.39% of Santhera's outstanding ordinary shares as of that date. On May 7, 2026, the closing price of Santhera’s common shares on the SIX Swiss Exchange was CHF 17.56 per share (approximately $22.55 USD based on then-current exchange rates).
When we launched AGAMREE® in March 2024, we utilized our FIRDAPSE® commercial and medical field-based forces to market AGAMREE®. Effective April 1, 2025, we separated these field-based forces into two units, one for each function expressly focused on
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supporting FIRDAPSE® and one for each function expressly focused on supporting AGAMREE®, respectively. This strategic change was made in an effort to allow us to better focus the support for each product.
Since April 1, 2025, we have sold AGAMREE® in the U.S. through a dedicated field-based force of approximately 16, including sales (12 Regional Account Managers and two Area Business Directors) and two Area Marketing Directors. We are further supporting the distribution of AGAMREE® through our Catalyst Pathways® patient services program to ensure that patients have access to a dedicated, personalized support team that assists families through the AGAMREE® patient journey, from answering questions to coordinating financial assistance programs for eligible patients. Additionally, we have also donated to qualified, independent charitable foundations dedicated to providing assistance to DMD patients in financial need who meet the independent organization's guidelines. Subject to applicable regulatory requirements, our goal is to ensure that no DMD patient is ever denied access to their medication for financial reasons.
DMD, the most common form of muscular dystrophy, is a rare and life-threatening neuromuscular disorder characterized by progressive muscle dysfunction, ultimately leading to loss of ambulation, respiratory failure, and fatality. Current standard treatment for DMD involves corticosteroids, which often come with significant side effects. It is estimated that between 11,000 and 13,000 people in the U.S. are affected by DMD, with approximately 70% of patients currently receiving a corticosteroid treatment. We believe that steroids are and will continue to remain the foundational therapy for DMD patients and dosed concomitantly with other therapies.
AGAMREE®’s unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity. As such, it is considered a novel corticosteroid that we hope has the potential to demonstrate a better tolerated side effect profile relative to traditional corticosteroids, while demonstrating comparable efficacy. This mechanism of action may allow vamorolone to emerge as an effective alternative to traditional corticosteroids in children, adolescents, and adult patients with DMD. In that regard, we are currently enrolling up to 250 patients in our SUMMIT registry study to evaluate long-term patient safety and quality of life data in patients treated with vamorolone, with the hope of offering a deeper understanding of the product's potential long-term benefits for patients, such as in the areas of growth parameters, bone health status (including the occurrence of fractures), BMI, cardiovascular status, muscular, hormonal and ophthalmologic status, QoL, and adverse events.
On October 13, 2023, Santhera announced that the European Union’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive position in favor of AGAMREE® for the treatment of DMD patients aged four and older. In its recommendation for approval, CHMP acknowledged that there was a positive benefit-risk profile of AGAMREE® in such patient population, including certain safety benefits of AGAMREE® compared to traditional corticosteroids in the treatment of DMD. Further, on December 18, 2023, the European Commission (EC) granted to Santhera marketing authorization for AGAMREE® for the treatment of DMD in patients ages four years and older and on January 12, 2024 Santhera announced that AGAMREE® had received approval by the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom. Further, on January 15, 2024, Santhera announced that AGAMREE® was commercially launched in Germany. Additionally, on January 16, 2025, the National Institute for Health and Care Excellence (NICE) issued positive Final Guidance that recommends AGAMREE® for use in the National Health Service (NHS) in England, Wales and Northern Ireland for the treatment of DMD in patients four years of age and older and on February 13, 2025, Santhera announced an agreement with the German National Association of Statutory Health Insurance Funds (GKV-SV) on the reimbursement for AGAMREE® for the treatment of DMD. This agreement makes AGAMREE® the first product to receive an agreed federal price in Germany for the treatment of DMD in patients four years of age and older, independent of the underlying genetic mutation. Finally, on January 15, 2026, Santhera announced that the Swiss Agency for Therapeutic Products (Swissmedic) approved AGAMREE® for the treatment of DMD in patients four years of age and older.
Further, Santhera has recently reported on the results of two studies, the Guardian Trial and the Lionheart trial, which provide useful information about some of the issues that are intended to be covered in the SUMMIT study. In addition, at the recent Muscular Dystrophy Conference held in March 2026, we sponsored an industry forum in collaboration with Santhera on the topic of real-world experience in using vamorolone for the treatment of patients with DMD.
We are also evaluating life cycle management of AGAMREE® with the intention to explore the possibility of additional indications beyond DMD. In furtherance of that objective, we are currently conducting a Phase 1 study in healthy adults comparing a single dose of vamorolone, prednisone, and deflazacort, and studying the immunosuppressive effect of multiple ascending doses of AGAMREE®. This study will evaluate whether there is an immunosuppressive dose of vamorolone that might be considered for future indications and for the use of our product in conjunction with gene and cell therapies that are approved to treat DMD and require a concurrent immunosuppressive regimen of a corticosteroid when administered. We expect to have the results of both parts of this study during the second quarter of 2026. Further, we are hopeful that the recent addition of DMD to the Recommended Uniform Screening Panel by the U.S. Department of Health and Human Services will help support earlier detection of the disease and more timely access to treatment options.
Further, the joint steering committee that we established with Santhera at the time we obtained a license for AGAMREE® is actively collaborating on the lifecycle management and development of the product. There can be no assurance that we can develop and commercialize our product for the treatment of diseases other than DMD.
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In the U.S., AGAMREE® has New Chemical Entity exclusivity that expires in October 2028. AGAMREE® also has Orphan Drug Exclusivity expiring in October 2030. AGAMREE® is further protected by seven Orange Book listed patents expiring as early as May 28, 2029 and as late as July 16, 2040. The Company has also requested Patent Term Extension (PTE) and will update the relevant expiration date in the Orange Book upon a final determination by the U.S. Patent and Trademark Office (USPTO). On June 25, 2025, the FDA published a notice (Determination of Regulatory Review Period for Purposes of Patent Extension; AGAMREE®) in the Federal Register regarding the requested extension of patent numbers 8,334,279, 10,857,161, and 11,833,159, all of which currently expire on May 28, 2029, as listed in the FDA's Orange Book. With the publication of this notice, there is a 180-day period for third parties to submit comments and/or due diligence petitions to the FDA. If no comments or petitions are submitted within this period, the FDA will notify the USPTO so that the USPTO can then determine the length of the PTE for each patent for which an extension was requested. Upon completion of the PTE determination, USPTO will mail a Notice of Final Determination of PTE. We will then have one month from the mailing of the Notice of Final Determination to elect one of the three patents for an extension patent term.
The earliest a generic manufacturer could submit an ANDA for vamorolone is October 26, 2027. If we were to pursue a patent infringement action of any such ANDA challenges of any of AGAMREE®’s Orange Book patents, then the automatic statutory 30-month stay would prevent FDA approval of such ANDA until April 26, 2031.
On July 23, 2024 we entered into a license, supply and commercialization agreement with KYE, which is already our sublicensee for FIRDAPSE® in Canada, granting KYE the exclusive Canadian commercial rights to market AGAMREE® in Canada for DMD and other indications. Under the agreement, KYE was responsible for obtaining regulatory approval of the product from Health Canada and on October 2, 2025, KYE announced that Health Canada has approved AGAMREE® for the treatment of DMD in boys four years of age and older. Under our sublicense agreement with KYE for this product, we will supply product to KYE and also receive sales milestones and sales royalties based on net revenues from sales of the product in Canada. We currently expect that KYE will launch the product in Canada sometime in 2026.
FYCOMPA®
On December 17, 2022, we entered into an agreement with Eisai Co., Ltd. (Eisai) for the acquisition of the U.S. rights to FYCOMPA® CIII. FYCOMPA® is a selective non-competitive antagonist of AMPA receptors, the major subtype of ionotropic glutamate receptors. It was the first, and still is the only, drug of its class to be approved for epilepsy. Studies suggest that AMPA receptor antagonism can lead to reduced overstimulation and anticonvulsant effects, as well as inhibiting seizure generation and spread. FYCOMPA® is a controlled substance and is approved with a boxed warning in its labeling. FYCOMPA® is used to treat certain types of focal onset seizures (seizures that involve only one part of the brain) in adults and children four years of age and older. It is also used in combination with other medications to treat certain types of primary generalized tonic-clonic seizures (also known as a “grand mal” seizure, a seizure that involves the entire body) in adults and children 12 years of age or older. Perampanel is in a class of medications called anticonvulsants. It works by decreasing abnormal electrical activity in the brain.
On January 24, 2023, we closed our acquisition of the U.S. rights to FYCOMPA®. In connection with the acquisition, we purchased Eisai’s regulatory approvals and documentation, product records, intellectual property, inventory, and other matters relating to the U.S. rights for FYCOMPA®, in exchange for an upfront payment of $160 million in cash. We also agreed to pay Eisai royalty payments on net sales after all FYCOMPA® patents had expired, with such royalty payments reduced after generic equivalents enter the market. In conjunction with the closing of the asset purchase, we entered into a Supply Agreement under which Eisai agreed to manufacture FYCOMPA® for us for at least seven years at prices listed in the Supply Agreement (to be updated on a yearly basis).
Patent protection for FYCOMPA® tablets and oral solution expired in 2025, and, to our knowledge based on publicly available information, three ANDA filers for the tablet formulations have, to date, obtained approval for and are marketing a generic version of FYCOMPA® tablets and one ANDA filer has, to date, obtained approval for and is marketing a generic version of FYCOMPA® oral suspension. We continue to sell FYCOMPA® tablets and oral suspension despite the loss of exclusivity, although we ceased all marketing efforts for the product at the end of 2025.
Business Development
We continue to advance our strategic initiatives and portfolio expansion efforts, focusing on broadening and diversifying our rare (orphan) neurology product portfolio with innovative therapies that address critical unmet medical needs. In that regard, we are currently exploring immediate or near-term accretive, clinically differentiated and adequately de-risked opportunities, with a keen focus on rare (orphan) disease products across therapeutic areas and treatment modalities. These prospects include evaluating products and companies with existing commercial drug products or drugs in development, including clinical stage opportunities with established proof of concept and a clear regulatory pathway for approval, for potential licensing or acquisition. We maintain a well-established U.S. presence, which remains the cornerstone of our commercial strategy, while continuously evaluating strategic opportunities to expand our global footprint.
We employ a disciplined, comprehensive, and exhaustive approach to identifying and evaluating opportunities that we believe will add significant value to our company over the near, mid, and long-term. However, no definitive agreements have been entered into to date to acquire the rights to any additional products, and there can be no assurance that any of the Company's business development initiatives will be successful.
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Capital Resources
At March 31, 2026, we had cash and cash equivalents of approximately $755.9 million. Based on our current financial condition, including our profitability, cash flows generated from operations and forecasts of available cash, absent the use of cash to acquire potential business development opportunities, we believe that we have sufficient funds to support our operations for at least the next 12 months. There can be no assurance that we will continue to be successful in commercializing FIRDAPSE® and AGAMREE®, that our forecasts of revenues from sales of FYCOMPA® will be accurate now that the product has lost patent exclusivity, or that we will continue to be profitable and cash flow positive. See “Management’s Discussion and Analysis of Financial Condition and Results of Operations - Liquidity and Capital Resources” below for further information on our liquidity and cash flow.
Share Repurchase Program
On October 1, 2025, we announced that our Board of Directors has authorized a new share repurchase program to repurchase up to $200 million of shares of our common stock between October 1, 2025 and December 31, 2026. Repurchases under the new share repurchase program may be made through a variety of methods, including open market or privately negotiated purchases. The timing and amount of shares repurchased will depend on the stock price, business and market conditions, corporate and regulatory requirements, alternative investment opportunities, acquisition opportunities and other factors. We are not obligated to repurchase any specific amount of shares of common stock, and the new share repurchase program may be suspended or terminated at any time.
We are using existing cash on hand to fund our share repurchase program. We also believe that we can execute our share repurchase program without impairing the advancement of our business development strategy. In that regard, as of May 7, 2026, we had repurchased 1,740,713 shares of our outstanding common stock for an aggregate purchase price of approximately $39.9 million ($22.91 average price per share).
Basis of Presentation
Revenues.
During the three months ended March 31, 2026, we generated revenues from product sales of FIRDAPSE®, AGAMREE®, and FYCOMPA®. We expect these revenues to fluctuate in future periods based on our sales during such periods of our products.
We received approval from Health Canada on July 31, 2020, for FIRDAPSE® for the symptomatic treatment of LEMS and as of December 31, 2020, our sub-licensee KYE launched FIRDAPSE® in Canada. During the three months ended March 31, 2026, revenues generated under our collaboration agreement with KYE were immaterial. In July 2024, we announced that we had entered into a collaboration agreement with KYE for the commercialization of AGAMREE® in Canada. On October 2, 2025, KYE issued a press release reporting that its NDS to commercialize AGAMREE® in Canada had been approved by Health Canada.
On September 24, 2024, we were informed by DyDo that it had received approval of its New Drug Application for the sale of FIRDAPSE® in Japan. Further, DyDo advised us that they launched FIRDAPSE® in Japan in January 2025.
We expect revenues from both the KYE and DyDo agreements to be immaterial in 2026, as distribution ramps up in each jurisdiction and KYE begins to market AGAMREE® in Canada.
Cost of Sales.
Cost of sales consists of third-party manufacturing costs, freight, royalties, amortization related to milestone payments, and indirect overhead costs associated with sales of our products. Cost of sales may also include period costs related to certain inventory manufacturing services, inventory adjustments charges, unabsorbed manufacturing and overhead costs and manufacturing variances.
Research and Development Expenses.
Our research and development expenses consist of costs incurred for company-sponsored research and development activities, as well as support for selected investigator-sponsored research. The major components of research and development costs include acquired IPR&D, preclinical study costs, clinical manufacturing costs, clinical study and trial expenses, insurance coverage for clinical trials, consulting, and other third-party costs, salaries and employee benefits, stock-based compensation expense, supplies and materials, and allocations of various overhead costs related to our product development efforts.
Prior to January 2023, all of our research and development resources had been devoted to the development of FIRDAPSE®, and until such time as we acquire or license new products we currently expect that our future development costs will be attributable principally to the continued development of FIRDAPSE®, and AGAMREE®.
Our cost accruals for clinical studies and trials are based on estimates of the services received and efforts expended pursuant to contracts with numerous clinical study and trial sites and clinical research organizations (CROs). In the normal course of our business we contract with third parties to perform various clinical study and trial activities in the on-going development of potential products. The financial terms of these agreements are subject to negotiation and vary from contract to contract and may result in uneven payment flows. Payments under the contracts depend on factors such as the achievement of certain events or milestones, the successful enrollment of patients, the allocation of responsibilities among the parties to the agreement, and the completion of portions of the clinical study or trial
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or similar conditions. The objective of our accrual policy is to match the recording of expenses in our condensed consolidated financial statements to the actual services received and efforts expended. As such, expense accruals related to preclinical and clinical studies or trials are recognized based on our estimate of the degree of completion of the event or events specified in the specific study or trial contract. We monitor service provider activities to the extent possible; however, if we underestimate activity levels associated with various studies or trials at a given point in time, we could be required to record significant additional research and development expenses in future periods. Preclinical and clinical study and trial activities require significant up-front expenditures. We anticipate paying significant portions of a study or trial’s cost before they begin and incurring additional expenditures as the study or trial progresses and reaches certain milestones.
Selling, General and Administrative Expenses.
During 2019, we began to commit funds to developing our commercialization program for FIRDAPSE® and we have continued to incur substantial commercialization expenses, including sales, marketing, patient services, patient advocacy and other commercialization related expenses as we have continued our sales and marketing program for FIRDAPSE®. We are also now incurring substantial commercialization expenses for AGAMREE®, as we continue commercialization of this product. We expect that such expenses for FYCOMPA® will substantially decline as a result of declining sales resulting from the loss of exclusivity of the product with the entry of generic competitors for this product.
Our general and administrative expenses consist primarily of salaries and personnel expenses for accounting, corporate, compliance, and administrative functions. Other costs include administrative facility costs, regulatory fees, insurance, and professional fees for legal (including litigation) cost, IT, accounting, and consulting services.
Amortization of Intangible Assets.
Amortization of intangible assets consists of the amortization of the FYCOMPA® product rights, which are amortized using the straight-line method over its estimated useful life of 5 years, the RUZURGI® product rights, which are amortized using the straight-line method over its estimated useful life of 14.5 years, and the AGAMREE® product rights, which are amortized using the straight-line method over its estimated useful life of 10.5 years.
Stock-Based Compensation.
We recognize expense for the fair value of all stock-based awards to employees, directors, and consultants in accordance with accounting principles generally accepted in the U.S. (U.S. GAAP). For stock options, we use the Black-Scholes option valuation model in calculating the fair value of the awards.
Income Taxes.
Our effective income tax rate is the ratio of income tax expense over our net income before income taxes.
Recently Issued Accounting Standards.
For discussion of recently issued accounting standards, please see Note 2, “Basis of Presentation and Significant Accounting Policies,” in the condensed consolidated financial statements included in this report.
Critical Accounting Policies and Estimates
Our discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with U.S. GAAP. The preparation of these condensed consolidated financial statements requires us to make judgments, estimates, and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements, as well as the reported revenues and expenses during the reporting periods. For a full discussion of our accounting policies, please refer to Note 2 on the Financial Statements included in our 2025 Annual Report on Form 10-K that we filed with the SEC on February 25, 2026. Our most critical accounting policies and estimates include accounting for revenue recognition (including adjustments for government rebates) and valuation of intangible assets. We continually evaluate our judgments, estimates and assumptions. We base our estimates on the terms of underlying agreements, our expected course of development, historical experience and other factors that we believe are reasonable based on the circumstances, the results of which form our management’s basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates. There have been no material changes to our critical accounting policies and estimates from the information provided in Part II, Item 7, Management’s Discussion and Analysis of Financial Condition and Results of Operations included in our 2025 Annual Report on Form 10-K.
Results of Operations
Revenues.
For the three months ended March 31, 2026, we recognized total revenues of approximately $149.4 million, which included approximately $149.3 million in net product revenue (primarily in the U.S.), compared to total revenues of approximately $141.4 million,
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which included approximately $141.4 million in net product revenue (primarily in the U.S.), for the three months ended March 31, 2025. Total revenues for the three months ended March 31, 2026 and 2025 were as follows (in thousands):
For the Three Months Ended March 31, Change
2026 2025 $ %
FIRDAPSE® $ 98,859 $ 83,731 15,128 18.1
AGAMREE® 36,713 22,042 14,671 66.6
FYCOMPA® 13,771 35,627 (21,856 ) (61.3 )
Product revenue, net 149,343 141,400 7,943 5.6
License and other revenue 47 21 26 123.8
Total revenues $ 149,390 $ 141,421 7,969 5.6
The increase of approximately $7.9 million in net product revenue when comparing the three months ended March 31, 2026 and 2025 was primarily driven by increases in sales volumes for both FIRDAPSE® and AGAMREE®.
Further, FYCOMPA® net product revenue decreased by approximately $21.9 million or 61.3% from the three months ended March 31, 2025 compared to the three months ended March 31, 2026, due to the generic entry following the loss of exclusivity in 2025. We expect that net product revenue for FYCOMPA® will likely continue to decrease in the future since generic competitors entered the market following the expiration of the '571 patent on May 23, 2025.
For the three months ended March 31, 2026 and 2025, we recognized $47 thousand and $21 thousand, respectively, in license and other revenue, which consisted of royalties.
Cost of Sales.
Cost of sales was approximately $14.5 million for the three months ended March 31, 2026, compared to approximately $17.9 million for the three months ended March 31, 2025. The decrease when comparing the three months ended March 31, 2026 to the three months ended March 31, 2025 was primarily due to a decrease in royalty obligations related to FIRDAPSE®. Cost of sales in all periods consisted principally of royalty payments, which are based on net revenue as defined in the applicable license agreements. For FIRDAPSE®, royalties are payable on the terms set forth below in Liquidity and Capital Resources—Contractual Obligations and Arrangements, and increase by 3% when net sales (as defined in the applicable license agreement) exceed $100 million in any calendar year. Cost of sales for FYCOMPA® for the three months ended March 31, 2026 consisted of product costs and excludes the amortization of the FYCOMPA® intangible assets. Cost of sales for AGAMREE® for the three months ended March 31, 2026 consisted of royalties payable on the terms set forth below in Liquidity and Capital Resources—Contractual Obligations and Arrangements, product costs and excludes the amortization of the AGAMREE® intangible asset. Royalties on sales of AGAMREE® in future years may increase as a percentage of net sales exceed certain amounts of net revenues over $100 million. See Note 13 of the "Notes to Condensed Consolidated Financial Statements" included elsewhere in this report.
Research and Development Expenses.
Research and development expenses for the three months ended March 31, 2026 and 2025 were approximately $2.7 million and $3.9 million, respectively, and represented approximately 3% and 5% of total operating costs and expenses, respectively. Research and development expenses for the three months ended March 31, 2026 and 2025 were as follows (in thousands):
For the Three Months Ended March 31, Change
2026 2025 $ %
Salary and benefit expense $ 1,018 $ 1,112 (94 ) (8.5 )
Employee stock-based compensation expense 462 425 37 8.7
Research and clinical trial expense 1,141 2,090 (949 ) (45.4 )
Additional research and development expense 40 260 (220 ) (84.6 )
Total research and development expenses $ 2,661 $ 3,887 (1,226 ) (31.5 )
Research and development expenses remained relatively consistent during the three months ended March 31, 2026 when compared to the same period in 2025. During the three months ended March 31, 2026, research and development expenses consisted of costs for company-sponsored research and development activities, support for selected investigator-sponsored research, and costs for development activities supporting our commercial products.
We expect that research and development activities may become more significant in the future if we seek to execute on the development of additional indications for FIRDAPSE® and AGAMREE® and on our portfolio expansion efforts.
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Selling, General and Administrative Expenses.
Selling, general and administrative expenses for the three months ended March 31, 2026 and 2025 were approximately $49.3 million and $46.9 million, respectively, and represented approximately 65% and 60% of total operating costs and expenses, respectively. Selling, general and administrative expenses for the three months ended March 31, 2026 and 2025 were as follows (in thousands):
For the Three Months Ended March 31, Change
2026 2025 $ %
Selling $ 29,661 $ 29,901 (240 ) (0.8 )
General and administrative 13,860 11,585 2,275 19.6
Employee stock-based compensation 5,798 5,425 373 6.9
Total selling, general and administrative expenses $ 49,319 $ 46,911 2,408 5.1
For the three months ended March 31, 2026, selling, general and administrative expenses remained relatively consistent when compared to the same period in 2025. The increase in general and administrative expenses was primarily due to consulting fees related to multiple business initiatives, including business development activities.
We expect that selling, general, and administrative expenses will continue to be substantial in future periods as we continue to sell FIRDAPSE® and AGAMREE® and as we take other steps in an effort to continue to expand our business, offset in part by reduced selling, general, and administrative expenses as more generic FYCOMPA® becomes available in the marketplace. We further anticipate that general and administrative expenses will be impacted in the second quarter of 2026 by a litigation avoidance payment in connection with the settlement of our patent litigation with Hetero.
Amortization of Intangible Assets.
Amortization of intangible assets was approximately $9.7 million for the three months ended March 31, 2026 compared to $9.3 million for the three months ended March 31, 2025. Amortization of intangible assets consists of the amortization of the FYCOMPA® rights, which are amortized using the straight-line method over its estimated useful life of 5 years, the RUZURGI® rights, which are amortized using the straight-line method over its estimated useful life of 14.5 years and the AGAMREE® rights and a sales-based milestone, which are amortized using the straight-line method over its estimated useful life of 10.5 years.
Each fiscal quarter, we review the value of our intangible assets to determine if they are impaired. If we determine one or more of our intangible assets are impaired during a future period we would record a charge in the amount of that impairment.
Stock-Based Compensation.
Total stock-based compensation for the three months ended March 31, 2026 and 2025 was $6.3 million and $5.9 million, respectively. During the three months ended March 31, 2026 and 2025, grants were principally for stock options and restricted stock units related to year-end bonus awards and grants to new employees.
Other Income, Net.
We reported other income, net in all periods, primarily relating to interest on our investment of our cash and cash equivalents of approximately $11.0 million and $7.9 million for the three months ended March 31, 2026 and 2025, respectively. The increase in other income, net for the three months ended March 31, 2026 when compared to the same period in 2025 was primarily due to higher invested balances and an increase in the fair value of our investment in Santhera. Since Santhera’s shares are traded on the SIX Swiss Exchange, they have a readily determinable fair value, and as a result the investment is measured quarterly, at fair value, with changes reported in other income, net.
The components of other income, net were as follows (in thousands):
For the Three Months Ended March 31,
2026 2025
Interest income, net $ 6,247 $ 5,275
Net gains (losses) recognized during the period on equity securities 4,750 2,644
Total other income, net $ 10,997 $ 7,919
Income Taxes.
Our effective income tax rate was approximately 24.3% and 20.4% for the three months ended March 31, 2026 and 2025, respectively. Differences in our effective tax and the statutory federal income tax of 21% are driven by state income taxes and anticipated annual permanent differences offset by equity compensation deductions. Our effective tax rate is affected by many factors, including the number
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of stock options exercised in any period, and our effective tax rate is likely to fluctuate in future periods (and may be higher than it was for the three months ended March 31, 2026).
We had no material uncertain tax positions as of March 31, 2026 and December 31, 2025.
Net Income.
Our net income was approximately $63.7 million for the three months ended March 31, 2026 ($0.52 per basic and $0.50 per diluted share) as compared to approximately $56.7 million for the three months ended March 31, 2025 ($0.47 per basic and $0.45 per diluted share).
Liquidity and Capital Resources
Since our inception, we have financed our operations primarily through revenues from product sales and multiple offerings of our securities. At March 31, 2026 we had cash and cash equivalents aggregating $755.9 million and working capital of $808.0 million. At December 31, 2025 we had cash and cash equivalents aggregating $709.2 million and working capital of $746.9 million. At March 31, 2026, substantially all of our cash and cash equivalents were deposited with two financial institutions, and such balances were in excess of federally insured limits. Further, as of such date, substantially all such funds were invested in U.S. Treasuries and money market accounts.
On September 8, 2023, we filed a shelf registration statement with the SEC to sell up to $500 million of common stock, preferred stock, warrants to purchase common stock, debt securities and units consisting of one or more of such securities (the 2023 Shelf Registration Statement). The 2023 Shelf Registration Statement (file no. 333-274427) became effective upon filing. On January 9, 2024, we completed a public offering of 10 million shares of our common stock under the 2023 Shelf Registration Statement, raising net proceeds of approximately $140.7 million.
Based on our current financial condition, including our profitability, cash flows generated from operations and forecasts of available cash, absent the use of cash to acquire potential business development opportunities, we believe that we have sufficient funds to support our operations for at least the next 12 months from the date of this report. There can be no assurance that we will remain profitable or that we will be able to obtain any additional funding that we may require in the future.
In the future, we may require additional working capital to support our operations depending on our future success with FIRDAPSE®, AGAMREE®, and FYCOMPA® sales, or the products we may acquire and continue to develop and whether our results continue to be profitable and cash flow positive. We may also need to raise additional capital to fund product acquisitions that are valued at more than our available cash. There can be no assurance as to the amount of any such funding that will be required for these purposes or whether any such funding will be available to us if and when it is required.
In that regard, our future funding requirements will depend on many factors, including:
•the cost of diligence in seeking potential acquisitions and of the completion of such acquisitions, if any future acquisitions occur;
•future clinical trial results;
•the scope, rate of progress and cost of our clinical trials and other product development activities;
•the terms and timing of any collaborative, licensing and other arrangements that we may establish;
•the cost and timing of regulatory approvals;
•the cost and delays in product development as a result of any changes in regulatory oversight applicable to our products;
•the amount of net revenues that we report from sales of FIRDAPSE®, AGAMREE®, and FYCOMPA®;
•the effect of competition and market developments;
•the cost of filing and potentially prosecuting, defending and enforcing any patent claims and other intellectual property rights; and
•the extent to which we acquire or invest in other products and the size of those investments.
We may raise additional funds through public or private equity offerings, debt financings, corporate collaborations or other means. We also may seek governmental grants for a portion of the required funding for our clinical trials and preclinical trials. We may further seek to raise capital to fund additional product development efforts or product acquisitions, even if we have sufficient funds for our planned operations. Any sale by us of additional equity or convertible debt securities could result in dilution to our stockholders. There can be no assurance that any such required additional funding will be available to us at all or available on terms acceptable to us.
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Cash Flows.
Our cash flow activities are summarized as follows (in thousands):
For the Three Months Ended March 31,
2026 2025
Net cash provided by operating activities $ 59,587 $ 60,043
Net cash used in investing activities (27 ) —
Net cash provided by (used in) financing activities (12,872 ) 3,060
Net increase in cash and cash equivalents $ 46,688 $ 63,103
The decrease in net cash provided by operating activities for the three months ended March 31, 2026 compared to the three months ended March 31, 2025 was primarily due to a $12.5 million sales-based milestone payment due upon the achievement of AGAMREE® net revenues of $100 million in a fiscal year, which was achieved in the fourth quarter of 2025 and paid in the first quarter of 2026. This was offset primarily by an increase in net income.
The increase in net cash used in investing activities for the three months ended March 31, 2026 compared to the three months ended March 31, 2025 was due to purchases of property and equipment.
The increase in net cash used in financing activities for the three months ended March 31, 2026 compared to the three months ended March 31, 2025 was primarily due to repurchases of common stock of approximately $14.6 million.
Contractual Obligations and Arrangements.
We have entered into the following contractual arrangements with respect to sales of FIRDAPSE®:
•Payments due under our license agreement for FIRDAPSE®. The following details the royalties under our license agreement:
•Royalties to our licensor for seven years from the first commercial sale of FIRDAPSE® equal to 7% of net sales (as defined in the FIRDAPSE® License Agreement) in each country for any calendar year for sales up to $100 million, with the rate increasing to 10% of net sales for any total net sales in excess of $100 million in North America; and
•Royalties to the third-party licensor of the rights sublicensed to us for seven years from the approval of the U.S. NDA for FIRDAPSE® at 7% of U.S. net sales (as defined in the license agreement between BioMarin (since transferred to SERB S.A.) and the third-party licensor) in any calendar year and after that 7th anniversary of the U.S. approval, royalties at 3.5% of U.S. net sales in any calendar year until the earlier of the 12th anniversary of the U.S. approval or the entry of a U.S. generic competitor. All royalty obligations to the third-party licensor for non-U.S. sales have concluded.
Further, we will pay royalties to our licensor on net sales in Japan equal to a similar percentage to the royalties that we are currently paying for non-U.S. sales under our original FIRDAPSE® License Agreement for North America.
For the three months ended March 31, 2026 and 2025, we recognized an aggregate of approximately $5.1 million and $11.6 million, respectively, of royalties payable under these license agreements, which is included in cost of sales in the accompanying condensed consolidated statements of operations and comprehensive income.
•Payments due to Jacobus. In connection with our July 2022 settlement with Jacobus, we agreed to pay the following consideration to Jacobus:
•$30 million of cash, of which $10 million was paid at the closing of the settlement on July 11, 2022, $10 million was paid on the first anniversary of closing and $10 million was paid on the second anniversary of closing; and
•An annual royalty on Catalyst’s net sales (as defined in the License and Asset Purchase Agreement between Catalyst and Jacobus) of amifampridine products in the U.S. equal to: (a) for calendar years 2022 through 2025, 1.5% (with a minimum annual royalty of $3.0 million per year), and (b) for calendar years 2026 through the expiration of the last to expire of Catalyst’s FIRDAPSE® patents in the U.S., 2.5% (with a minimum annual royalty of $5 million per year); provided, however, that the royalty rate may be reduced and the minimum annual royalty may be eliminated under certain circumstances.
•Summary of changes to royalty obligations related to FIRDAPSE®:
•On January 25, 2026, we completed seven years from the date of first commercial sale of FIRDAPSE® in the U.S. On that date, the royalty on net U.S. sales that we previously paid to our immediate licensor at a tiered rate of 7-10% of net U.S. sales of FIRDAPSE® expired. Also, on January 1, 2026, as part of our acquisition and license agreement regarding RUZURGI® with Jacobus, the royalty rate we pay Jacobus on net U.S. sales of FIRDAPSE®
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and RUZURGI® increased from 1.5% to 2.5%. In addition to these two changes, there was also another change in FIRDAPSE® royalties owed by us on net U.S. sales that occurred in November 2025. On November 28, 2025, due to seven years passing from the date of the FDA approval of FIRDAPSE®, the royalty on net U.S. sales of FIRDAPSE® that we owe to our immediate licensor to satisfy their royalty obligations to their third-party licensor decreased from 7% of net U.S. sales to 3.5%. As a result, the overall royalty rate beginning on January 26, 2026, that we will pay to our upstream licensors for net U.S. sales of FIRDAPSE® will be 6%, which is down from a previous maximum rate of 18.5%.
For the three months ended March 31, 2026 and 2025, we recognized an aggregate of approximately $2.4 million and $1.2 million, respectively, of royalties payable to Jacobus.
We have entered into the following contractual arrangements with respect to sales of FYCOMPA®:
•Payments due under our asset purchase agreement for FYCOMPA®. In connection with our asset purchase agreement with Eisai Co., Ltd. (Eisai), we agreed to pay the following consideration to Eisai:
•We paid at closing a $160 million upfront cash payment, plus $1.6 million for reimbursement of certain prepayments.
•Royalties commencing on the expiration of the last patent for the product for each calendar year during the royalty term equal to 12% on net sales greater than $10 million and less than $100 million, 17% on net sales of greater than $100 million and less than $125 million and 22% on net sales greater than $125 million prior to the date of generic entry. Upon the entry of generic competition, these royalties will be reduced to 6% on net sales greater than $10 million and less than $100 million, 8.5% on net sales of greater than $100 million and less than $125 million and 11% on net sales greater than $125 million.
•Concurrently with the acquisition, the parties entered into two related agreements: (i) a short-term TSA for commercial and manufacturing services (to which transition services ended on December 31, 2023) and (ii) a long-term Supply Agreement for the manufacturing of FYCOMPA®. Under the TSA, Eisai provided certain commercial and manufacturing services to the Company for a transition period following the closing of the acquisition. Further, under the Supply Agreement, Eisai will manufacture FYCOMPA® for the Company for a period of seven years (or such longer period as is set forth in the Supply Agreement) following the closing of the acquisition.
We have entered into the following contractual arrangements with respect to AGAMREE®:
•Payments due under our license agreement for AGAMREE®. In connection with our acquisition from Santhera:
•At closing we paid a $75 million initial cash payment.
•In the fourth quarter of 2023, following regulatory approval of Santhera’s NDA for AGAMREE® by the FDA, we paid a regulatory milestone payment of $36 million. We are also obligated to pay additional regulatory milestone payments upon regulatory approval by the FDA in the U.S. of an NDA for the product for the first, second, and third additional indications in the amounts of $50 million, $45 million, and $45 million, respectively.
•We may be obligated to pay Santhera sales-based milestones of up to $105 million, which includes a sales-based milestone payment of up to $12.5 million upon achievement of revenues in the calendar year in which revenues exceed $100 million (which was achieved in the fourth quarter of 2025), and pay royalties if the applicable amount of net sales of all products in the territory in a single calendar year fall within the range of one or more of the net sales threshold levels set forth in the AGAMREE® License Agreement.
•At signing, we were obligated to purchase all of our finished goods requirements for products solely from Santhera at a set supply price until January 1, 2026, but the parties agreed upon an amendment to the license agreement that allowed us to start the process for creating our own supply chain to manufacture AGAMREE® earlier and we expect to complete that process by the end of 2026.
•Simultaneously with entering into the license agreement, we made a strategic equity investment into Santhera by acquiring 1,414,688 of Santhera’s ordinary shares (representing approximately 11.26% of Santhera’s outstanding ordinary shares immediately following the transaction) at an investment price of CHF 9.477 per share (corresponding to a mutually agreed volume-weighted average price prior to signing), with the approximately $15.7 million USD in equity investment proceeds, inclusive of the approximately $13.5 million USD fair value of the investment in Santhera and approximately $2.2 million USD of transaction costs included in acquired in-process research and development, to be used by Santhera for Phase IV studies in DMD and further development of additional indications for AGAMREE®.
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For both the three months ended March 31, 2026 and 2025, we recognized an aggregate of approximately $2.5 million of royalties payable under this license agreement, which is included in cost of sales in the accompanying condensed consolidated statements of operations and comprehensive income.
We also have entered into the following contractual arrangements:
•Purchase commitments. We have entered into a purchase commitment for FIRDAPSE® with a contract manufacturing organization for approximately $0.5 million per year. The agreement expires in December 2026.
•Lease for office space. We operate our business in leased office space in Coral Gables, Florida. We lease approximately 10,700 square feet of office space and we pay annual rent of approximately $0.5 million.
Off-Balance Sheet Arrangements.
We do not have any off-balance sheet arrangements as such term is defined in rules promulgated by the SEC.
Caution Concerning Forward-Looking Statements
This report contains “forward-looking statements”, as that term is defined in the Private Securities Litigation Reform Act of 1995. These include statements regarding our expectations, beliefs, plans or objectives for future operations and anticipated results of operations. For this purpose, any statements contained herein that are not statements of historical fact may be deemed to be forward-looking statements. Without limiting the foregoing, “believes”, “anticipates”, “proposes”, “plans”, “expects”, “intends”, “may”, and other similar expressions are intended to identify forward-looking statements. Such statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or other achievements to be materially different from any future results, performances or achievements expressed or implied by such forward-looking statements. Factors that might cause such differences include, but are not limited to, those discussed in the section entitled “Item 1A – Risk Factors.”
The continued successful commercialization of FIRDAPSE® (amifampridine), AGAMREE® (vamorolone), and FYCOMPA® (perampanel) CIII is uncertain. Factors that will affect our success include the uncertainty of:
•Whether the merger transaction with Angelini Pharma will be successfully completed, and the timeline for such completion;
•Whether we will be able to continue to successfully market and sell FIRDAPSE® and AGAMREE®, and continue to sell FYCOMPA® while maintaining full compliance with applicable federal and state laws, rules and regulations;
•Whether we will be able to continue to attract and retain the qualified personnel necessary to run our business;
•Whether we can continue to market our drug products on a profitable and cash flow positive basis;
•Whether any revenue or earnings guidance that we provide to the investment community will turn out to be accurate;
•Whether our estimates of the size of the market for FIRDAPSE® for the treatment of LEMS will prove to be accurate, and whether we can continue to increase FIRDAPSE® net product revenues as we have done in past periods;
•Whether the daily dose of FIRDAPSE® taken by patients changes over time and how that affects our net product revenues for FIRDAPSE® in future periods;
•Whether we will continue to be able to locate LEMS patients who are undiagnosed or are misdiagnosed with another disease, including LEMS patients who also have small cell lung cancer;
•Whether the addition of FIRDAPSE® to the NCCN Clinical Practice Guidelines for small cell lung cancer results in an increase in patients being treated with FIRDAPSE®;
•Whether patients will discontinue from the use of our products at rates that are higher than historically experienced or higher than we forecast;
•Whether new patients for our existing and future drug products can be successfully titrated to stable therapy;
•Whether we will be able to demonstrate, to the satisfaction of the FDA and third-party payors, that AGAMREE® offers advantages compared to other corticosteroids or competitor’s products;
•Whether DMD patients transitioning to current or future approved gene therapy treatments will delay initiating use of AGAMREE® while waiting for access to such gene therapy, or stop their AGAMREE® therapy during the course of their gene therapy treatment;
•Whether steroids will continue to be the foundational standard of care for the treatment of DMD as new DMD therapies are approved for commercialization in the future;
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•Whether we will be able to continue to successfully sell FYCOMPA® now that generic competition for FYCOMPA® tablets and oral suspension have entered the market;
•Whether reduced revenue resulting from generic FYCOMPA® entering the market will require us to impair all or a portion of our intangible asset for FYCOMPA®;
•The extent to which payors will continue to provide coverage and reimburse for our products at the price that we charge for our products;
•The ability of our third-party suppliers and contract manufacturers to continue to supply sufficient product to meet our customers’ needs in a timely manner;
•The impact on our profits and cash flow of adverse changes in reimbursement and coverage policies or regulations from government and private payors such as Medicare, Medicaid, insurance companies, health maintenance organizations and other plan administrators, or the impact of pricing pressures enacted by industry organizations, the federal government or the government of any state, including as a result of increased scrutiny over pharmaceutical pricing or otherwise;
•Changes in the healthcare industry and the effect of political pressure from and actions by the current Administration, Congress and/or medical professionals seeking to reduce prescription drug costs, and changes to the healthcare industry occasioned by any future changes in laws relating to the pricing of drug products, including changes made in the Inflation Reduction Act of 2022, changes (if any) to be made by the current Administration (including the possibility of seeking to impose Most Favored Nation pricing on drug companies or to impose a 100% tariff on branded or patented pharmaceutical products unless a company is "building" a manufacturing plant in the United States) and/or the current Congressional administrations, changes to the review and approval process at the FDA, imposing tariffs on imported product, or changes in the healthcare industry generally;
•The potential impact of tariffs on our cost of sales for products that are manufactured, in whole or in part, outside of the U.S.;
•The potential impact on our business of “most favorite nation”, or Most Favored Nation pricing, such as proposed in the GUARD and GLOBE Medicare demonstration projects, on what we will realize from the sale of our drug products if Most Favored Nation pricing becomes applicable to even a portion of our drug products;
•The ability of our third-party suppliers and contract manufacturers to maintain compliance with current Good Manufacturing Practices;
•The ability of those third parties that distribute our products to maintain compliance with applicable law;
•Our ability to maintain compliance with applicable rules relating to our patient assistance programs for our products;
•The scope and strength of our intellectual property and the outcome of challenges to our intellectual property, and, conversely, whether any third-party intellectual property presents unanticipated obstacles for FIRDAPSE® or AGAMREE®;
•Whether there will be a post-closing review by antitrust regulators of our previous or future Paragraph IV patent settlements or our previous acquisition transactions, and the outcome of any such reviews if they were to occur;
•Whether we will be able to acquire additional drug products under development, complete development required to commercialize such products, and thereafter, if such products are approved for commercialization, successfully market such products;
•Whether the FDA will approve generic versions of FIRDAPSE® following the expiration of our orphan drug exclusivity on November 26, 2025, subject to any pending 30-month stays of approval as required under the Drug Price Competition and Patent Term Restoration Act of 1984;
•Whether our patents will be sufficient to prevent generic competition for AGAMREE® after our orphan drug exclusivity for this product expires;
•Whether our clinical studies of AGAMREE® and our SUMMIT registry study of DMD patients being treated with AGAMREE®, will be successful and the impact, if any, of the results of these studies on our business;
•Whether we are able to successfully develop additional indications for FIRDAPSE® and obtain the ability to commercialize FIRDAPSE® for these additional indications;
•Whether we and Santhera Pharmaceuticals Holding AG can successfully develop additional indications for AGAMREE®, and obtain the ability to commercialize AGAMREE® for these additional indications;
•The state of the economy generally;
•The impact on our business of a prolonged U.S. government shutdown;
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•The scope, rate of progress and expense of our clinical trials and studies, pre-clinical studies, proof-of-concept studies, and our other drug development activities, and whether any trials and studies we undertake will be successful;
•Our ability to complete any clinical trials and studies that we may undertake on a timely basis and within the budgets we establish for such trials and studies;
•Whether FIRDAPSE® and AGAMREE® will be successfully commercialized in Canada on a profitable basis through KYE Pharmaceuticals, Inc., our sublicensee for the products in Canada;
•Whether FIRDAPSE® will be successfully commercialized in Japan on a profitable basis by DyDo Pharma, Inc., our sublicensee for the product in Japan;
•The impact on sales of FIRDAPSE® in the U.S. if an amifampridine product is purchased in Canada for use in the U.S.;
•Whether any efforts we undertake to expand the reach of FIRDAPSE® into other global regions will be successful;
•System failures or security or data breaches due to cyber-attacks, or cyber intrusions, including ransomware, phishing attacks and other malicious intrusions whether it occurs directly to us or indirectly through third parties; and
•Our ability to enhance our systems, processes, and procedures to appropriately support the growing complexity and scale of our business.
Our current plans and objectives are based on assumptions relating to the continued sale of FIRDAPSE®, AGAMREE® and FYCOMPA® and on our plans to seek to acquire or in-license additional drug products. Although we believe that our assumptions are reasonable, any of our assumptions could prove inaccurate. Considering the significant uncertainties inherent in the forward-looking statements we have made herein, which reflect our views only as of the date of this report, you should not place undue reliance upon such statements. We undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.