Sphingolipidoses

First quarter 2025 BRIUMVI U.S. net revenue of $119.7 million Raises full year 2025 global net revenue target to approximately $575 million and raises full year BRIUMVI U.S. net revenue target to approximately $560 million Conference call to be held today, Monday, May 5, 2025, at 8:30 AM ET NEW YORK, May 05, 2025 (GLOBE NEWSWIRE) -- TG Therapeutics, Inc. (NASDAQ: TGTX) (the Company or TG Therapeutics) today announced its financial results for the first quarter of 2025, along with recent company developments and provided an update on 2025 revenue guidance. Michael S. Weiss, the Company's Chairman and Chief Executive Officer, stated, "2025 is off to a strong start, and I’m incredibly proud of the progress made thus far. Our performance in the first quarter, including BRIUMVI’s $119.7 million in U.S. net sales, demonstrates the growing confidence in our treatment and the increasing adoption by healthcare providers. This momentum, combined with the positive feedback we’re hearing from both patients and clinicians, reinforces our belief that we can achieve our long-term goal of BRIUMVI becoming the number one prescribed anti-CD20 treatment based on dynamic market share.” Mr. Weiss continued, “In addition to our ongoing BRIUMVI commercialization efforts, we are continuing to invest in innovation, including simplifying the BRIUMVI treatment regimen, advancing subcutaneous BRIUMVI, and developing azer-cel for progressive MS. All with a focus on developing treatment options designed to improve the lives of individuals living with MS.” Recent Highlights & Developments BRIUMVI® (ublituximab-xiiy) Commercialization BRIUMVI U.S. net product revenue of $119.7 million for the first quarter of 2025, representing approximately 137% growth over the same period last year Expansion of commercialization outside of the U.S. with our partner, Neuraxpharm, with BRIUMVI now commercially available in additional countries in the European Union, United Kingdom and Switzerland BRIUMVI Data Presented five-year data from the open-label extension study of the ULTIMATE I & II Phase 3 clinical trials evaluating BRIUMVI in adult patients with relapsing forms of multiple sclerosis (RMS) which demonstrated that 92% of patients were free from disability progression after five years of treatment, an annualized relapse rate of 0.02 during year 5 of treatment (equivalent to one relapse occurring every fifty years of patient treatment), and an overall safety profile that remained consistent, with no new safety signals emerging over 5 years of continuous treatment and no observed cases of progressive multifocal leukoencephalopathy (PML) to date. Presented data from the ENHANCE Phase 3b clinical trial evaluating alternative dosing strategies for BRIUMVI in patients with RMS which demonstrated that: A single 600 mg BRIUMVI infusion on Day 1 was well tolerated in individuals with RMS regardless of prior treatment or B-cell depletion status Rapid 30-minute BRIUMVI infusions were well tolerated in patients with RMS Published two articles in medical journals: “Switching to Ublituximab from Prior anti-CD20 Monoclonal Antibody Therapy: A Case Report Series”, published in Frontiers in Immunology, demonstrating a retrospective case series of seven individuals with multiple sclerosis (MS) treated in private practice or at an MS clinic who switched to ublituximab from a different anti-CD20 monoclonal antibody therapy due to efficacy or tolerability concerns, published in Frontiers in Immunology “The Evolution of Anti-CD20 Treatment in Multiple Sclerosis”, published in CNS DRUGS, demonstrating the differentiating characteristics within the anti-CD20 monoclonal antibody class used to treat MS, published in CNS DRUGS. A single 600 mg BRIUMVI infusion on Day 1 was well tolerated in individuals with RMS regardless of prior treatment or B-cell depletion status Rapid 30-minute BRIUMVI infusions were well tolerated in patients with RMS “Switching to Ublituximab from Prior anti-CD20 Monoclonal Antibody Therapy: A Case Report Series”, published in Frontiers in Immunology, demonstrating a retrospective case series of seven individuals with multiple sclerosis (MS) treated in private practice or at an MS clinic who switched to ublituximab from a different anti-CD20 monoclonal antibody therapy due to efficacy or tolerability concerns, published in Frontiers in Immunology “The Evolution of Anti-CD20 Treatment in Multiple Sclerosis”, published in CNS DRUGS, demonstrating the differentiating characteristics within the anti-CD20 monoclonal antibody class used to treat MS, published in CNS DRUGS. Pipeline Status Phase 1 clinical trial evaluating subcutaneous BRIUMVI in patients with RMS remains ongoing Phase 1 clinical trial evaluating BRIUMVI in patients with Myasthenia Gravis (MG) remains ongoing Phase 1 clinical trial evaluating azercabtagene zapreleucel (azer-cel) in patients with primary progressive multiple sclerosis is now open for enrollment 2025 Financial Guidance Raises BRIUMVI U.S. net product revenue target to $560 million for the full year 2025 (prior guidance of $525 million for full year 2025) Raises total global revenue target to $575 million for the full year 2025 (prior guidance of $540 million for full year 2025) Second quarter 2025 BRIUMVI U.S. net product revenue target of $135 million Full year 2025 target operating expense of approximately $300 million (excluding non-cash compensation and cost of goods sold) 2025 Development Pipeline Anticipated Milestones Commence pivotal program of subcutaneous BRIUMVI Commence a pivotal program based on the improved dosing regimens evaluated in the ENHANCE trial, with the goal of enhancing the patient experience on intravenous BRIUMVI Continue enrollment of participants into the ongoing clinical trial evaluating BRIUMVI in autoimmune diseases outside of MS Continue enrollment of participants into the Phase 1 clinical trial evaluating azer-cel for the treatment of autoimmune diseases, beginning with progressive forms of MS Present updated data at major medical conferences throughout the year Financial Results for First Quarter 2025 Product Revenue, Net: Product revenue, net was approximately $119.7 million for the three months ended March 31, 2025, compared to $50.5 million for the three months ended March 31, 2024. Product revenue, net for both the three months ended March 31, 2025 and March 31, 2024, consisted of net product sales of BRIUMVI in the United States. License, Milestone, Royalty and Other Revenue: License, milestone, royalty and other revenue was approximately $1.2 million for the three months ended March 31, 2025, compared to $13.0 million for the three months ended March 31, 2024. License, milestone, royalty and other revenue for the three months ended March 31, 2024 is predominantly comprised of a $12.5 milestone payment under the commercialization agreement with Neuraxpharm for the first key market commercial launch of BRIUMVI in the European Union. R&D Expenses: Total research and development (R&D) expense was $46.4 million for the three months ended March 31, 2025, compared to $32.7 million for the three months ended March 31, 2024. The increase in R&D expense for the three months ended March 31, 2025, was primarily attributable to manufacturing expenses incurred in connection with our ublituximab subcutaneous development work during the period. SG&A Expenses: Total selling, general and administrative (SG&A) expense was $50.3 million for the three months ended March 31, 2025, compared to $34.6 million for the three months ended March 31, 2024. The increase was primarily due to other selling, general and administrative costs, including marketing, personnel and consultants, associated with the commercialization of BRIUMVI for the three months ended March 31, 2025. Net Income (Loss): Net income was $5.1 million for the three months ended March 31, 2025, compared to a net loss of $10.7 million for the three months ended March 31, 2024. Cash Position and Financial Guidance: Cash, cash equivalents and investment securities were $276.2 million as of March 31, 2025. We anticipate that our cash, cash equivalents and investment securities as of March 31, 2025, combined with the projected revenues from BRIUMVI, will be sufficient to fund our business based on our current operating plan. CONFERENCE CALL INFORMATIONThe Company will host a conference call today, May 5, 2025, at 8:30 AM ET, to discuss the Company’s financial results from first quarter 2025. To participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics. A live audio webcast will be available on the Events page, located within the Investors & Media section, of the Company's website at http://ir.tgtherapeutics.com/events. An audio recording of the conference call will also be available for a period of 30 days after the call. ABOUT BRIUMVI® (ublituximab-xiiy) 150 mg/6 mL Injection for IVBRIUMVI is a novel monoclonal antibody that targets a unique epitope on CD20-expressing B-cells. Targeting CD20 using monoclonal antibodies has proven to be an important therapeutic approach for the management of autoimmune disorders, such as RMS. BRIUMVI is uniquely designed to lack certain sugar molecules normally expressed on the antibody. Removal of these sugar molecules, a process called glycoengineering, allows for efficient B-cell depletion at low doses. BRIUMVI is indicated in the U.S. for the treatment of adults with RMS, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease and in the EU and UK for the treatment of adult patients with RMS with active disease defined by clinical or imaging features. A list of authorized specialty distributors can be found at www.briumvi.com. IMPORTANT SAFETY INFORMATIONContraindications: BRIUMVI is contraindicated in patients with: Active Hepatitis B Virus infection A history of life-threatening infusion reaction to BRIUMVI WARNINGS AND PRECAUTIONS Infusion Reactions: BRIUMVI can cause infusion reactions, which can include pyrexia, chills, headache, influenza-like illness, tachycardia, nausea, throat irritation, erythema, and an anaphylactic reaction. In MS clinical trials, the incidence of infusion reactions in BRIUMVI-treated patients who received infusion reaction-limiting premedication prior to each infusion was 48%, with the highest incidence within 24 hours of the first infusion. 0.6% of BRIUMVI-treated patients experienced infusion reactions that were serious, some requiring hospitalization. Observe treated patients for infusion reactions during the infusion and for at least one hour after the completion of the first two infusions unless infusion reaction and/or hypersensitivity has been observed in association with the current or any prior infusion. Inform patients that infusion reactions can occur up to 24 hours after the infusion. Administer the recommended pre-medication to reduce the frequency and severity of infusion reactions. If life-threatening, stop the infusion immediately, permanently discontinue BRIUMVI, and administer appropriate supportive treatment. Less severe infusion reactions may involve temporarily stopping the infusion, reducing the infusion rate, and/or administering symptomatic treatment. Infections: Serious, life-threatening or fatal, bacterial and viral infections have been reported in BRIUMVI-treated patients. In MS clinical trials, the overall rate of infections in BRIUMVI-treated patients was 56% compared to 54% in teriflunomide-treated patients. The rate of serious infections was 5% compared to 3% respectively. There were 3 infection-related deaths in BRIUMVI-treated patients. The most common infections in BRIUMVI-treated patients included upper respiratory tract infection (45%) and urinary tract infection (10%). Delay BRIUMVI administration in patients with an active infection until the infection is resolved. Consider the potential for increased immunosuppressive effects when initiating BRIUMVI after immunosuppressive therapy or initiating an immunosuppressive therapy after BRIUMVI. Hepatitis B Virus (HBV) Reactivation: HBV reactivation occurred in an MS patient treated with BRIUMVI in clinical trials. Fulminant hepatitis, hepatic failure, and death caused by HBV reactivation have occurred in patients treated with anti-CD20 antibodies. Perform HBV screening in all patients before initiation of treatment with BRIUMVI. Do not start treatment with BRIUMVI in patients with active HBV confirmed by positive results for HB surface antigen (HBsAg) and anti-HB tests. For patients who are negative for HBsAg and positive for HB core antibody [HBcAb+] or are carriers of HBV [HBsAg+], consult a liver disease expert before starting and during treatment. Progressive Multifocal Leukoencephalopathy (PML): Although no cases of PML have occurred in BRIUMVI-treated MS patients, JC virus infection resulting in PML has been observed in patients treated with other anti-CD20 antibodies and other MS therapies. If PML is suspected, withhold BRIUMVI and perform an appropriate diagnostic evaluation. Typical symptoms associated with PML are diverse, progress over days to weeks, and include progressive weakness on one side of the body or clumsiness of limbs, disturbance of vision, and changes in thinking, memory, and orientation leading to confusion and personality changes. MRI findings may be apparent before clinical signs or symptoms; monitoring for signs consistent with PML may be useful. Further investigate suspicious findings to allow for an early diagnosis of PML, if present. Following discontinuation of another MS medication associated with PML, lower PML-related mortality and morbidity have been reported in patients who were initially asymptomatic at diagnosis compared to patients who had characteristic clinical signs and symptoms at diagnosis. If PML is confirmed, treatment with BRIUMVI should be discontinued. Vaccinations: Administer all immunizations according to immunization guidelines: for live or live-attenuated vaccines, at least 4 weeks and, whenever possible, at least 2 weeks prior to initiation of BRIUMVI for non-live vaccines. BRIUMVI may interfere with the effectiveness of non-live vaccines. The safety of immunization with live or live-attenuated vaccines during or following administration of BRIUMVI has not been studied. Vaccination with live virus vaccines is not recommended during treatment and until B-cell repletion. Vaccination of Infants Born to Mothers Treated with BRIUMVI During Pregnancy: In infants of mothers exposed to BRIUMVI during pregnancy, assess B-cell counts prior to administration of live or live-attenuated vaccines as measured by CD19+ B-cells. Depletion of B-cells in these infants may increase the risks from live or live-attenuated vaccines. Inactivated or non-live vaccines may be administered prior to B-cell recovery. Assessment of vaccine immune responses, including consultation with a qualified specialist, should be considered to determine whether a protective immune response was mounted. Fetal Risk: Based on data from animal studies, BRIUMVI may cause fetal harm when administered to a pregnant woman. Transient peripheral B-cell depletion and lymphocytopenia have been reported in infants born to mothers exposed to other anti-CD20 B-cell depleting antibodies during pregnancy. A pregnancy test is recommended in females of reproductive potential prior to each infusion. Advise females of reproductive potential to use effective contraception during BRIUMVI treatment and for 6 months after the last dose. Reduction in Immunoglobulins: As expected with any B-cell depleting therapy, decreased immunoglobulin levels were observed. Decrease in immunoglobulin M (IgM) was reported in 0.6% of BRIUMVI-treated patients compared to none of the patients treated with teriflunomide in RMS clinical trials. Monitor the levels of quantitative serum immunoglobulins during treatment, especially in patients with opportunistic or recurrent infections, and after discontinuation of therapy, until B-cell repletion. Consider discontinuing BRIUMVI therapy if a patient with low immunoglobulins develops a serious opportunistic infection or recurrent infections, or if prolonged hypogammaglobulinemia requires treatment with intravenous immunoglobulins. Most Common Adverse Reactions: The most common adverse reactions in RMS trials (incidence of at least 10%) were infusion reactions and upper respiratory tract infections. Physicians, pharmacists, or other healthcare professionals with questions about BRIUMVI should visit www.briumvi.com. The full Summary of Product Characteristics approved in the European Union (EU) for BRIUMVI can be found here Briumvi | European Medicines Agency (europa.eu). ABOUT BRIUMVI PATIENT SUPPORT in the U.S. BRIUMVI Patient Support is a flexible program designed by TG Therapeutics to support U.S. patients through their treatment journey in a way that works best for them. More information about the BRIUMVI Patient Support program can be accessed at www.briumvipatientsupport.com.ABOUT MULTIPLE SCLEROSIS Relapsing multiple sclerosis (RMS) is a chronic demyelinating disease of the central nervous system (CNS) and includes people with relapsing-remitting multiple sclerosis (RRMS) and people with secondary progressive multiple sclerosis (SPMS) who continue to experience relapses. RRMS is the most common form of multiple sclerosis (MS) and is characterized by episodes of new or worsening signs or symptoms (relapses) followed by periods of recovery. It is estimated that nearly 1 million people are living with MS in the United States and approximately 85% are initially diagnosed with RRMS.1,2 The majority of people who are diagnosed with RRMS will eventually transition to SPMS, in which they experience steadily worsening disability over time. Worldwide, more than 2.3 million people have a diagnosis of MS.1 ABOUT TG THERAPEUTICSTG Therapeutics is a fully integrated, commercial stage, biopharmaceutical company focused on the acquisition, development and commercialization of novel treatments for B-cell diseases. In addition to a research pipeline including several investigational medicines, TG Therapeutics has received approval from the U.S. Food and Drug Administration (FDA) for BRIUMVI® (ublituximab-xiiy) for the treatment of adult patients with relapsing forms of multiple sclerosis, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, as well as approval by the European Commission (EC) in Europe, the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom, and Swissmedic in Switzerland, for BRIUMVI to treat adult patients with RMS who have active disease defined by clinical or imaging features. For more information, visit www.tgtherapeutics.com, and follow us on X (formerly Twitter) @TGTherapeutics and on LinkedIn. BRIUMVI® is a registered trademark of TG Therapeutics, Inc.Cautionary StatementThis press release contains forward-looking statements that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release. In addition to the risk factors identified from time to time in our reports filed with the U.S. Securities and Exchange Commission (SEC), factors that could cause our actual results to differ materially include the below. Such forward looking statements include but are not limited to statements regarding expectations for the timing and success of the commercialization and availability of BRIUMVI® (ublituximab-xiiy) for RMS in the United States, the European Union, the United Kingdom, Switzerland or other jurisdictions outside of the United States; anticipated healthcare professional (HCP) and patient acceptance and use of BRIUMVI for the approved indications; expectations of future revenue for BRIUMVI, expenses or profits; expectations for our pipeline products; and statements regarding the results of the ENHANCE or ULTIMATE I & II Phase 3 studies. Additional factors that could cause our actual results to differ materially include the following: the Company’s ability to maintain and continue to maintain a commercial infrastructure for BRIUMVI, and to successfully or in the timeframe projected, market and sell BRIUMVI; the risk that trends in prescriptions are not maintained or that prescriptions are not filled; the failure to obtain and maintain payor coverage; the risk that HCP interest in BRIUMVI will not be sustained; the risk that momentum in sales for BRIUMVI will not be sustained during the course of the year; the risk that the commercialization of BRIUMVI does not continue to exceed expectations; the risk that our BRIUMVI revenue targets will not be achieved; the failure to obtain and maintain requisite regulatory approvals, including the risk that the Company fails to satisfy post-approval regulatory requirements, the potential for variations from the Company’s projections and estimates about the potential market for BRIUMVI due to a number of factors, including, further limitations that regulators may impose on the required labeling for BRIUMVI (such as modifications, resulting from safety signals that arise in the post-marketing setting or in the long-term extension study from the ULTIMATE I and II clinical trials); the Company’s ability to meet post-approval compliance obligations (on topics including but not limited to product quality, product distribution and supply chain, pharmacovigilance, and sales and marketing); the Company’s reliance on third parties for manufacturing, distribution and supply, and other support functions for our clinical and commercial products, including BRIUMVI, and the ability of the Company and its manufacturers and suppliers to produce and deliver BRIUMVI to meet the market demand for BRIUMVI; potential regulatory challenges to the Company’s plans to seek marketing approval for the product in jurisdictions outside of the U.S.; the uncertainties inherent in research and development; the risk that any individual patient’s clinical experience in the post-marketing setting, or the aggregate patient experience in the post-marketing setting, may differ from that demonstrated in controlled clinical trials such as ULTIMATE I and II; the risk that the Company does not achieve its 2025 development pipeline anticipated milestones in the timeframe projected or at all, including commencing a pivotal program for subcutaneous ublituximab, commencing a pivotal program based on data from the ENHANCE trial, enrolling patients into a trial evaluating BRIUMVI in an autoimmune disease outside of MS, or enrolling patients into a trial evaluating azer-cel; regulatory developments, legislative actions, executive orders, including the imposition of tariffs and policy changes in the U.S. and other jurisdictions; and general political, economic and business conditions. Further discussion about these and other risks and uncertainties can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our other filings with the SEC. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. This press release and prior releases are available at www.tgtherapeutics.com. The information found on our website is not incorporated by reference into this press release and is included for reference purposes only.CONTACT: Investor Relations Email: ir@tgtxinc.comTelephone: 1.877.575.TGTX (8489), Option 4 Media Relations: Email: media@tgtxinc.com Telephone: 1.877.575.TGTX (8489), Option 6 1. MS Prevalence. National Multiple Sclerosis Society website. https://www.nationalmssociety.org/About-the-Society/MS-Prevalence. Accessed October 26, 2020. 2. Multiple Sclerosis International Federation, 2013 via Datamonitor p. 236. Condensed Balance Sheet Information (in thousands): * Condensed from audited financial statements

1Q 2025 Total Revenue of $125.2M, a 15% Increase Year-over-Year at CER Expanding Portfolio through In-Licensing of DMX-200 Phase 3 Program for Rare Kidney Disease with Significant Market Potential in the U.S. Maintaining 2025 Guidance for Galafold, Reflecting Strong Underlying Demand Updating 2025 Pombiliti + Opfolda Guidance with New Patient Starts Accelerating in 2H Adjusting 2025 Total Revenue Growth Guidance to 15-22% at CER Reiterating GAAP Profitability During H2 2025 Conference Call and Webcast Today at 8:30 a.m. ET PRINCETON, N.J., May 01, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced financial results for the first quarter ended March 31, 2025. “Amicus delivered another consecutive quarter of significant double-digit revenue growth. Looking forward, the underlying patient demand we observed in Q1 will drive robust growth for Galafold, and we continue to expect accelerating Pombiliti + Opfolda sales as the year progresses, driven by patient starts from new launch markets as well as anticipated acceleration in U.S. switches. While some unexpected factors impacted revenue in the quarter, the key performance indicators for both products are very strong and we remain on-track to achieve GAAP profitability during the second half of 2025 and to deliver double-digit revenue growth this year and beyond,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. Mr. Campbell continued “We are also thrilled to have announced the in-licensing of the U.S. commercial rights to Dimerix’ DMX-200, a first-in-class treatment in Phase 3 development for people living with FSGS, a rare and potentially fatal kidney disease. This aligns perfectly with our strategy to leverage our rare disease commercial infrastructure and brings a third program with blockbuster market potential to our portfolio. Amicus is well positioned to create substantial value for shareholders and to deliver on our mission for patients, and we very much look forward to working with Dimerix to bring this much needed therapy to people living with FSGS in the United States.” First Quarter 2025 Financial Highlights: Total revenues for the first quarter 2025 were $125.2 million, reflecting operational growth measured at constant exchange rates (CER)1 of 15% and a currency headwind of $1.4 million or 1%. (in thousands)Three Months Ended March 31, Year over Year % Growth 2025 2024 Reported at CER1 Galafold® 104,244 99,359 5% 6% Pombiliti® + Opfolda® 21,005 11,044 90% 92% Net Product Revenues$125,249 $110,403 13% 15% Galafold® (migalastat) net product sales for the first quarter 2025 were $104.2 million, representing a year-over-year increase of 5%, or 6% at CER1. Strong patient demand of 14% in the quarter was partially offset by order timing and the ongoing impact of the higher than anticipated VPAG (Voluntary Scheme for Branded Medicines Pricing and Access) rebate in the U.K. Given the significant underlying demand, the Company anticipates Galafold revenue to accelerate in the second quarter and is reiterating its full year 2025 revenue growth guidance of +10-15% at CER.Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) net product sales for the first quarter 2025 were $21.0 million, representing a year-over-year increase of 90%, or 92% at CER1. First quarter sales reflected the timing of patient starts in new launch countries and the ongoing impact of the higher than anticipated VPAG rebate in the U.K. The Company now expects the benefit of patient starts in new launch markets to be more weighted towards the second half of the year. Taken together with an anticipated acceleration in U.S. switches, the Company is therefore adjusting its 2025 revenue growth guidance range for Pombiliti + Opfolda to +50-65% at CER.Total GAAP operating expenses of $121.5 million for the first quarter 2025 decreased by 2.5% as compared to $124.6 million for the first quarter 2024. Total non-GAAP operating expenses2 were up 10.4% to $94.5 million for the first quarter 2025 as compared to $85.6 million for the first quarter 2024.GAAP net loss was $21.7 million, or $0.07 loss per share basic and diluted, for the first quarter 2025, compared to a net loss of $48.4 million, or $0.16 per share basic and diluted, for the first quarter 2024. Non-GAAP net income2,3 was $9.0 million, or $0.03 per share basic and diluted, for the first quarter 2025, compared to a non-GAAP net loss of $4.6 million, or $0.02 per share basic and diluted, for the first quarter 2024.Cash, cash equivalents, and marketable securities totaled $250.6 million at March 31, 2025, representing a slight increase as compared to $249.9 million at December 31, 2024. Corporate Updates: Pombiliti + Opfolda selected as preferred treatment for adults living with late-onset Pompe disease in the Netherlands. The five-year agreement will enable broad and sustained access to Pombiliti + Opfolda for adults living with late-onset Pompe disease in the Netherlands currently receiving enzyme replacement therapy or naïve to treatment. First commercial patients are expected to begin treatment in 2Q 2025. The Netherlands has the highest prevalence of Pompe disease in Europe with over 150 individuals living with the condition. Pombiliti + Opfolda regulatory approval granted in Canada and Australia for adult LOPD patients. The Company continues to anticipate a regulatory decision in Japan this year as well as additional reimbursement agreements throughout the year. The Company also remains on track for up to 10 new launch countries in 2025, which include more than 650 individuals living with LOPD.Commercial manufacturing and supply services agreement reached with Sharp Sterile to manufacture Pombiliti drug product in the U.S. This agreement is another important step in further diversifying the supply chain for Pombiliti.Entered into exclusive U.S. licensing agreement with Dimerix. As announced separately, Amicus has licensed exclusive rights for the U.S. commercialization of Dimerix’ Phase 3 program, DMX-200, a first in class treatment for FSGS, a rare and fatal kidney disease with no approved therapies and significant market potential.Amicus is focused on delivering significant long-term revenue growth and anticipates surpassing $1 billion in total sales in 2028. The Company anticipates continuing to grow its current commercial business with Galafold and Pombiliti + Opfolda resulting in strong total revenue growth. 2025 Financial Guidance Amicus’ updated financial guidance for 2025, which includes the upfront license payment and all other anticipated operating expenses of the licensing of DMX-200 in the U.S., is as follows: Previous Updated Total Revenue Growth117% to 24%→15% to 22% Galafold Revenue Growth110% to 15%→10% to 15% Pombiliti + Opfolda Growth165% to 85%→50% to 65% Gross MarginMid 80%→Mid 80% Non-GAAP Operating Expenses4$350M to $370M→$380M to $400M(Incl. $30M Upfront License Payment)GAAP Net IncomePositive during 2H 2025→Positive during 2H 2025 1 In order to illustrate underlying performance, Amicus discusses its results in terms of constant exchange rate (CER) growth. This represents growth calculated as if the exchange rates had remained unchanged from those used in the comparative period. 2 Full reconciliation of GAAP results to the Company’s non-GAAP adjusted measures for all reporting periods appear in the tables to this press release.3 Amicus defines non-GAAP Net (Loss) Income as GAAP Net (Loss) Income excluding the impact of share-based compensation expense, changes in fair value of contingent consideration, loss on impairment of assets, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, restructuring charges and income taxes.4 A reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure is not available without unreasonable effort due to high variability, complexity, and low visibility as to the items that would be excluded from the GAAP measure. Conference Call and WebcastAmicus Therapeutics will host a conference call and audio webcast today, May 1, 2025, at 8:30 a.m. ET to discuss the first quarter 2025 financial results and corporate updates. Participants and investors interested in accessing the call by phone will need to register using the online registration form. After registering, all phone participants will receive a dial-in number along with a personal PIN number to access the event. A live audio webcast and related presentation materials can also be accessed via the Investors section of the Amicus Therapeutics corporate website at ir.amicusrx.com. Web participants are encouraged to register on the website 15 minutes prior to the start of the call. An archived webcast and accompanying slides will be available on the Company's website shortly after the conclusion of the live event. About Galafold Galafold® (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body’s own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of people living with Fabry disease may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan. U.S. INDICATIONS AND USAGEGalafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data. This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. U.S. IMPORTANT SAFETY INFORMATION ADVERSE REACTIONS: The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. USE IN SPECIFIC POPULATIONS: There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus. It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition. Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis. The safety and effectiveness of Galafold have not been established in pediatric patients. To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. About Pombiliti + OpfoldaPombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood. U.S. INDICATIONS AND USAGEPOMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). SAFETY INFORMATION HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. See PI for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions ≥ 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) LINK and full PRESCRIBING INFORMATION for OPFOLDA (miglustat) LINK. About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on X and LinkedIn. Non-GAAP Financial Measures In addition to financial information prepared in accordance with U.S. GAAP, this press release also contains adjusted financial measures that we believe provide investors and management with supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We typically exclude certain GAAP items that management does not believe affect our basic operations and that do not meet the GAAP definition of unusual or non-recurring items. Other companies may define these measures in different ways. When we provide our expectation for non-GAAP operating expenses and profitability on a forward-looking basis, a reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded items may have a significant, and potentially unpredictable, impact on our future GAAP results. Forward Looking StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to the prospects and timing of the potential regulatory and pricing approval of our products, commercialization plans, manufacturing and supply plans, financing plans, the collaboration with Dimerix, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities and pricing and reimbursement authorities, are based on current information. Actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that regulatory authorities may not grant or may delay approval for our product candidates; the potential that required regulatory inspections may be delayed or not be successful and delay or prevent product approval; the potential that we may not be successful in negotiations with pricing and reimbursement authorities; the potential that we may not be successful in commercializing Galafold and/or Pombiliti and Opfolda in Europe, the UK, the US and other geographies; the potential that the Dimerix collaboration and license agreement for of DMX-200 may not be successful, including without limitation expectations of the timing of Phase 3 clinical trial evaluating DMX-200; the likelihood of success of such clinical trial; the prospects for FDA approval of DMX-200 for FSGS or other indications; the estimated prevalence of FSGS; the achievement of any milestone and timing of any payments associated with milestones and the success of any efforts to commercialize DMX-200, including any projections of future financial performance or payments; ; the potential that we may not be able to manufacture or supply sufficient commercial products; and the potential that we will need additional funding to complete the manufacturing and commercialization of our products. With respect to statements regarding corporate financial guidance and financial goals and the expected attainment of such goals and projections of the Company's revenue, non-GAAP profitability and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2024 and our Quarterly Report on Form 10-Q to be filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof. CONTACT: Investors: Amicus Therapeutics Andrew FaughnanVice President, Investor Relationsafaughnan@amicusrx.com(609) 662-3809 Media: Amicus Therapeutics Diana Moore Head of Global Corporate Affairs and Communicationsdmoore@amicusrx.com(609) 662-5079 FOLD-G TABLE 1 Amicus Therapeutics, Inc.Consolidated Statements of Operations(Unaudited)(in thousands, except share and per share amounts) Three Months Ended March 31, 2025 2024 Net product sales$125,249 $110,403 Cost of goods sold 11,698 13,567 Gross profit 113,551 96,836 Operating expenses: Research and development 27,839 28,329 Selling, general, and administrative 91,827 88,029 Restructuring charges — 6,045 Depreciation and amortization 1,837 2,154 Total operating expenses 121,503 124,557 Loss from operations (7,952) (27,721)Other expense: Interest income 812 1,540 Interest expense (11,455) (12,436)Other income (expense) 550 (4,966)Loss before income tax (18,045) (43,583)Income tax expense (3,641) (4,836)Net loss attributable to common stockholders$(21,686) $(48,419)Net loss attributable to common stockholders per common share — basic and diluted$(0.07) $(0.16)Weighted-average common shares outstanding — basic and diluted 307,689,207 302,903,009 TABLE 2 Amicus Therapeutics, Inc.Consolidated Balance Sheets(in thousands, except share and per share amounts) March 31, 2025 December 31, 2024 Assets Current assets: Cash and cash equivalents$181,657 $213,752 Investments in marketable securities 68,916 36,194 Accounts receivable 88,323 101,099 Inventories 132,412 118,782 Prepaid expenses and other current assets 39,491 34,909 Total current assets 510,799 504,736 Operating lease right-of-use assets, net 22,138 22,278 Property and equipment, less accumulated depreciation of $29,842 and $28,775 at March 31, 2025 and December 31, 2024, respectively 28,718 29,383 Intangible assets, less accumulated amortization of $6,611 and $5,802 at March 31, 2025 and December 31, 2024, respectively 16,389 17,198 Goodwill 197,797 197,797 Other non-current assets 13,998 13,641 Total Assets$789,839 $785,033 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable$14,451 $12,947 Accrued expenses and other current liabilities 129,877 127,300 Operating lease liabilities 8,562 8,455 Total current liabilities 152,890 148,702 Long-term debt 390,708 390,111 Operating lease liabilities 44,196 45,078 Other non-current liabilities 8,487 7,097 Total liabilities 596,281 590,988 Commitments and contingencies Stockholders’ equity: Common stock, $0.01 par value, 500,000,000 shares authorized, 307,923,069 and 299,041,653 shares issued and outstanding at March 31, 2025 and December 31, 2024, respectively 3,016 2,944 Common stock in treasury, at cost; 7,390 shares as of March 31, 2025 (71) — Additional paid-in capital 2,939,673 2,926,115 Accumulated other comprehensive income (loss): Foreign currency translation adjustment 12,941 5,302 Unrealized loss on available-for-sale securities (135) (207)Warrants — 71 Accumulated deficit (2,761,866) (2,740,180) Total stockholders’ equity 193,558 194,045 Total Liabilities and Stockholders’ Equity $789,839 $785,033 TABLE 3 Amicus Therapeutics, Inc.Reconciliation of Non-GAAP Financial Measures(in thousands)(Unaudited) Three Months Ended March 31, 2025 2024 Total GAAP operating expenses $ 121,503 $ 124,557 Research and development: Share-based compensation 4,004 4,871 Selling, general and administrative: Share-based compensation 21,168 25,932 Restructuring charge — 6,045 Depreciation and amortization 1,837 2,154 Total Non-GAAP operating expense adjustments 27,009 39,002 Total Non-GAAP operating expenses $94,494 $85,555 TABLE 4 Amicus Therapeutics, Inc.Reconciliation of Non-GAAP Financial Measures(in thousands, except share and per share amounts)(Unaudited) Three Months Ended March 31, 2025 2024 GAAP net loss $ (21,686) $(48,419) Share-based compensation 25,172 30,803 Depreciation and amortization 1,837 2,154 Restructuring charges — 6,045 Income tax expense 3,641 4,836 Non-GAAP net income (loss) $8,963 $(4,581) Non-GAAP net income (loss) attributable to common stockholders per common share — basic and diluted $0.03 $(0.02) Weighted-average common shares outstanding — basic 307,689,207 302,903,009 Weighted-average common shares outstanding — diluted 309,654,136 302,903,009

BETHESDA, Md., April 30, 2025 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced an oral poster presentation at the International Association of Parkinsonism and Related Disorders (IAPRD) 30th World Congress on Parkinson's Disease and Related Disorders, being held May 7th-10th in New York City, NY. Details of the oral and poster presentation are as follows: Presentation Title: GT-02287, A Clinical-stage Allosteric GCase Modulator For The Treatment Of Parkinson’s Disease, Protects Dopaminergic Neurons Against Mitochondrial Toxin MPP+ Presenter: Joanne Taylor, Ph.D., Senior Vice President of Research, Gain Therapeutics Session: Oral Poster Presentation – Guided Poster TourSession Date: Saturday, May 10, 2025Session Time: 8:00-9:00 a.m. EST About GT-02287Gain Therapeutics’ lead drug candidate, GT-02287, is in clinical development for the treatment of Parkinson’s disease (PD) with or without a GBA1 mutation. The orally administered, brain-penetrant small molecule is an allosteric enzyme modulator that restores the function of the lysosomal enzyme glucocerebrosidase (GCase) which becomes misfolded and impaired due to mutations in the GBA1 gene, the most common genetic abnormality associated with PD, or other age-related stress factors. In preclinical models of PD, GT-02287 restored GCase enzymatic function, reduced ER stress, lysosomal and mitochondrial pathology, aggregated α-synuclein, neuroinflammation and neuronal death, as well as plasma neurofilament light chain (NfL) levels, a biomarker of neurodegeneration. In rodent models of both GBA1-PD and idiopathic PD, GT-02287 was shown to rescue deficits in motor function and gait and prevent the development of deficits in complex behaviors such as nesting. Compelling preclinical data in models of both GBA1-PD and idiopathic PD, demonstrating a disease-modifying effect after administration of GT-02287, suggest that GT-02287 may have the potential to slow or stop the progression of Parkinson’s disease. Results from a Phase 1 study of GT-02287 in healthy volunteers demonstrated favorable safety and tolerability, plasma and CNS exposures in the projected therapeutic range, and target engagement with a >50% increase in glucocerebrosidase (GCase) activity among those receiving GT-02287 at clinically relevant doses. GT-02287 is currently being evaluated in a Phase 1b clinical trial for the treatment of Parkinson’s disease with or without a GBA1 mutation. The primary endpoint of the trial, which is currently enrolling participants across 7 sites in Australia, is to evaluate the safety and tolerability of GT-02287 after 3 months of dosing in people with Parkinson’s disease. Gain’s lead program in Parkinson’s disease has been awarded funding support early in its development from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse – Swiss Innovation Agency. About Gain Therapeutics, Inc.Gain Therapeutics, Inc. is a clinical-stage biotechnology company leading the discovery and development of next generation allosteric therapies. Gain’s lead drug candidate, GT-02287 is currently being evaluated for the treatment of Parkinson’s disease with or without a GBA1 mutation in a Phase 1b clinical trial. GT-02287 has further potential in Gaucher’s disease, dementia with Lewy bodies, and Alzheimer’s disease. Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors. Gain’s unique approach enables the discovery of novel, allosteric small molecule modulators that can restore or disrupt protein function. Deploying its highly advanced Magellan™ platform, Gain is accelerating drug discovery and unlocking novel disease-modifying treatments for untreatable or difficult-to-treat disorders including neurodegenerative diseases, rare genetic disorders and oncology. Forward-Looking StatementsThis release contains “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are typically preceded by words such as “believes,” “expects,” “anticipates,” “intends,” “will,” “may,” “should,” or similar expressions. These forward-looking statements reflect management’s current knowledge, assumptions, judgment and expectations regarding future performance or events. Although management believes that the expectations reflected in such statements are reasonable, they give no assurance that such expectations will prove to be correct or that those goals will be achieved, and you should be aware that actual results could differ materially from those contained in the forward-looking statements. Forward-looking statements are subject to a number of risks and uncertainties, including, but not limited to, statements regarding: the development of the Company’s current or future product candidates including GT-02287; expectations regarding the timing of results from a Phase 1b clinical study for GT-02287; expectations regarding the timing of patient enrollment for a Phase 1b clinical study for GT-02287; the timing of any submissions to the FDA or other regulatory bodies and agencies; and the potential therapeutic and clinical benefits of the Company’s product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the Company’s business in general, please refer to the Company’s Form 10-K for the year ended December 31, 2024. All forward-looking statements are expressly qualified in their entirety by this cautionary notice. You are cautioned not to place undue reliance on any forward-looking statements, which speak only as of the date of this release. We have no obligation, and expressly disclaim any obligation, to update, revise or correct any of the forward-looking statements, whether as a result of new information, future events or otherwise. Investors:Gain Therapeutics, Inc. Apaar Jammu Manager, Investor Relations and Public Relationsajammu@gaintherapeutics.com LifeSci Advisors LLCChuck PadalaManaging Directorchuck@lifesciadvisors.com Media:Russo Partners LLC Nic Johnson and Elio Ambrosionic.johnson@russopartnersllc.comelio.ambrosio@russopartnersllc.com(760) 846-9256