Solid Biosciences, Inc.

The dynamics of the Friedreich's ataxia market are anticipated to change as companies across the globe are thoroughly working toward the development of new drug therapy options to treat this disease. Key products such as RT001 (Retrotope), Vatiquinone (PTC Therapeutics), and Leriglitazone (Minoryx Therapeutics) might influence the market size during the forecast period. LAS VEGAS, June 5, 2025 /PRNewswire/ -- DelveInsight's Friedreich's Ataxia Market Insights report includes a comprehensive understanding of current treatment practices, Friedreich's ataxia emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Friedreich's Ataxia Market Report According to DelveInsight's analysis, the market size for Friedreich's ataxia is anticipated to surge at a significant rate by 2034. The United States is expected to account for the highest market size of Friedreich's ataxia, of the total market size in 7MM in comparison to the other major markets, i.e., EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan. The prevalence of Friedreich's ataxia in the United States has been gradually increasing, reflecting greater awareness and improved diagnostic capabilities. Advances in genetic testing have allowed for earlier and more accurate identification of the condition, contributing to higher reported cases. Additionally, increased access to healthcare may facilitate more comprehensive assessments and documentation of rare diseases like Friedreich's ataxia. As research progresses, ongoing efforts to understand the genetic and environmental factors influencing this condition will be crucial for developing effective treatments and support systems for affected individuals. Based on a secondary research genetically confirmed Friedreich's ataxia patient has not been found in the Japanese population, and the majority of patients with Friedreich's ataxia-like phenotype may be confirmed as AOA1, AOA2, ataxia with vitamin E deficiency or autosomal-recessive spastic ataxia of Charlevoix-Saguenay if examined by genetic testing. Leading Friedreich's ataxia companies developing emerging therapies, such as Retrotope, PTC Therapeutics, Minoryx Therapeutics, Biogen, Lexeo Therapeutics, Larimar Therapeutics, Design Therapeutics, Solid Biosciences, and others, are developing novel Friedreich's ataxia drugs that can be available in the Friedreich's ataxia market in the coming years. The promising Friedreich's ataxia therapies in the pipeline include RT001, Vatiquinone, Leriglitazone, Omaveloxolone, LX2006, nomlabofusp, SGT-212, and others. Discover which therapies are expected to grab the Friedreich's ataxia therapeutics market share @ Friedreich's Ataxia Market Report Friedreich's Ataxia Market Dynamics The Friedreich's ataxia market dynamics are expected to change in the coming years. Advances in the understanding of Friedreich's ataxia have significantly clarified the role of frataxin and the disease's natural history, providing crucial insights that have guided the development of targeted therapeutic approaches aimed at addressing the underlying pathology. The approval of omaveloxolone, a NF-E2-related factor 2 activator, by the FDA in 2023 and the EU in 2024, marks a milestone in disease-specific treatment. Concurrently, the rising prevalence of Friedreich's ataxia, driven by population growth and improved diagnostic technologies, is expected to fuel market demand. Meanwhile, biomarker development is progressing, with promising tools such as frataxin quantification and imaging biomarkers enhancing clinical trial sensitivity and enabling more precise patient stratification. Furthermore, potential therapies are being investigated for the treatment of Friedreich's ataxia, and it is safe to predict that the treatment space will significantly impact the Friedreich's ataxia market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the Friedreich's ataxia market in the 7MM. However, several factors may impede the growth of the Friedreich's ataxia market. Progressive neurodegeneration in Friedreich's ataxia causes irreversible damage to the spinal cord, peripheral nerves, and cerebellum, leading to cumulative and disabling impairments; however, diagnostic delays due to reliance on clinical evaluation and genetic testing, especially in atypical or late-onset cases, combined with challenges in clinical trial design stemming from small patient populations, disease heterogeneity, and limited validated endpoints, as well as financial barriers such as high R&D costs and reimbursement hurdles, collectively complicate drug development and restrict patient access to emerging therapies. Moreover, Friedreich's ataxia treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the Friedreich's ataxia market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the Friedreich's ataxia market growth. Friedreich's Ataxia Treatment Market Current treatments for Friedreich's ataxia primarily focus on alleviating specific symptoms rather than addressing the root cause of the disease. Despite advancements, symptom management continues to be the standard approach to care. While there is no cure, several strategies can help improve the quality of life for those affected. The most serious complication, heart disease, can often be managed with medications commonly used in the general population, such as ACE inhibitors, beta-blockers, and diuretics, which help reduce cardiac strain. In cases of irregular heart rhythms, pacemakers or drugs may help stabilize heart function. Diabetes, another potential symptom, can be treated with insulin. Surgical interventions are occasionally required to address foot deformities or scoliosis; though significant, these procedures are generally effective. Physical therapy plays a crucial role in maintaining muscle flexibility and mobility, while speech therapy can assist in improving communication and swallowing abilities by retraining facial and tongue muscles. Emerging research targeting the root cause of Friedreich's ataxia focuses on frataxin, a protein involved in iron regulation and oxidative stress. Gene therapy approaches have shown potential, especially gene replacement strategies, although issues like targeted delivery, genotoxicity, and regulation of gene expression remain unresolved. Alternative approaches such as protein replacement using frataxin precursors or TAT-frataxin fusion proteins are also under investigation. SKYCLARYS (omaveloxolone) is approved for treating Friedreich's ataxia in patients aged 16 and older, though its safety and effectiveness in younger individuals are still unknown. Omaveloxolone (RTA-408) is a semisynthetic oleanane triterpenoid with anti-inflammatory and antioxidant effects. It activates the Nrf2 transcription factor, which is crucial for controlling oxidative stress, a function impaired in Friedreich's ataxia due to mitochondrial dysfunction. Enhancing Nrf2 activity through omaveloxolone may thus offer therapeutic benefits. In September 2023, Biogen finalized its acquisition of Reata Pharmaceuticals, the company behind omaveloxolone. To know more about Friedreich's ataxia treatment options, visit @ Friedreich's Ataxia Medication Friedreich's Ataxia Pipeline Therapies and Key Companies Some of the drugs in the pipeline include RT001 (Retrotope), Vatiquinone (PTC Therapeutics), Leriglitazone (Minoryx Therapeutics), Omaveloxolone (Biogen), LX2006 (Lexeo Therapeutics), CTI-1601 (Larimar Therapeutics, Inc.), SGT-212 (Solid Biosciences), and others. Vatiquinone, developed by PTC Therapeutics, is an experimental small molecule that targets 15-Lipoxygenase, an enzyme critically involved in regulating oxidative stress and inflammatory responses linked to neurological disorders. In March 2014, the U.S. FDA granted it Fast Track designation for treating Friedreich's ataxia, and it also holds Orphan Drug designation for the same condition. Currently, vatiquinone is in Phase III clinical trials for Friedreich's ataxia. In February 2025, the FDA accepted its New Drug Application (NDA) for the treatment of both pediatric and adult patients with Friedreich's ataxia, awarding it Priority Review with a PDUFA target action date of August 19, 2025. RT001, developed by Retrotope, is a deuterated ethyl linoleate that inhibits lipid peroxidation, aiming to mitigate cellular damage and improve mitochondrial function. It received Fast Track status from the FDA in February 2021, and had earlier been granted Orphan Drug status in 2016 for Friedreich's ataxia. A Phase II/III trial evaluating its safety and efficacy in Friedreich's ataxia was completed in August 2021. Leriglitazone, a product of Minoryx Therapeutics, is an orally active, selective PPARγ agonist with a promising profile for central nervous system (CNS) disorders. It was designated an Orphan Drug by both the FDA and EMA in 2019 for Friedreich's ataxia. Clinical evaluation in Phase II trials showed beneficial outcomes in patients with the condition. The anticipated launch of these emerging therapies are poised to transform the Friedreich's ataxia market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Friedreich's ataxia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about Friedreich's ataxia treatment pipeline @ Friedreich's Ataxia Clinical Trials Recent Developments in the Friedreich's Ataxia Market In April 2025, Lexeo Therapeutics, Inc. reported encouraging interim results from all dose groups of LX2006 in the treatment of Friedreich ataxia cardiomyopathy. Data from both the Lexeo-sponsored SUNRISE-FA Phase 1/2 trial (NCT05445323) and the investigator-initiated Phase 1A trial at Weill Cornell Medicine (NCT05302271) showed that LX2006 led to meaningful improvements in cardiac biomarkers and functional outcomes. Additionally, all participants who underwent cardiac biopsies demonstrated increased frataxin protein expression. In February 2025, the FDA accepted the New Drug Application (NDA) for vatiquinone, intended for the treatment of both children and adults with Friedreich's ataxia. The application has been granted Priority Review. In January 2025, Solid Biosciences Inc. announced that the FDA has granted Fast Track designation to SGT-212, its AAV-based gene therapy candidate for treating Friedreich's ataxia. Friedreich's Ataxia Overview Friedreich's ataxia is a rare genetic disorder that results in progressive damage to the nervous system and problems with movement. It typically starts during childhood and gradually leads to worsening muscle coordination. The condition is caused by a mutation in the FXN gene, which provides instructions for producing a protein called frataxin. People who inherit two faulty copies of this gene, one from each parent, develop the disease. In atypical cases, Friedreich's ataxia is classified into two subtypes: late-onset FRDA (LOFA), which begins between the ages of 26 and 39, and very late-onset FRDA (VLOFA), which starts after age 40. Symptoms generally appear between the ages of 5 and 15, with the earliest signs often including difficulty walking, poor balance, and slurred or slow speech. Diagnosing Friedreich's ataxia involves a detailed clinical evaluation, including a comprehensive physical examination to identify issues such as balance problems, loss of joint position sense, absent reflexes, and other neurological signs. In addition, genetic testing now offers a definitive method for confirming the diagnosis. Friedreich's Ataxia Epidemiology Segmentation The Friedreich's ataxia epidemiology section provides insights into the historical and current Friedreich's ataxia patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The Friedreich's ataxia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Diagnosed Prevalent Cases of Friedreich's Ataxia Onset Type Diagnosed Prevalent Cases of Friedreich's Ataxia Total Treated Cases of Friedreich's Ataxia Scope of the Friedreich's Ataxia Market Report Therapeutic Assessment: Friedreich's Ataxia current marketed and emerging therapies Friedreich's Ataxia Market Dynamics: Key Market Forecast Assumptions of Emerging Friedreich's Ataxia Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Friedreich's Ataxia Market Access and Reimbursement Download the report to understand which factors are driving Friedreich's ataxia market trends @ Friedreich's Ataxia Treatment Market Table of Contents Related Reports Friedreich's Ataxia Epidemiology Forecast Friedreich's Ataxia Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Friedreich's ataxia epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Friedreich's Ataxia Pipeline Friedreich's Ataxia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Friedreich's ataxia companies, including PTC Therapeutics, Retrotope, Reata Pharmaceuticals, Minoryx Therapeutics, Larimar Therapeutics, LEXEO Therapeutics, Exicure, StrideBio, Voyager Therapeutics, Lacerta Therapeutics, among others. Ataxia Market Ataxia Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key ataxia companies, including AstraZeneca, Merck Sharp & Dohme LLC, Bayer, Roche, among others. Ataxia Pipeline Ataxia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ataxia companies, including Retrotope, Reata Pharmaceuticals, PTC Therapeutics, Metro International Biotech, LLC, Design Therapeutics, Larimar Therapeutics, Minoryx Therapeutics, EryDel, Biogen, Matrix Biomed, IntraBio, Biohaven Pharmaceuticals, Inc., Stealth BioTherapeutics Inc., Acasti Pharma, Seelos Therapeutics, Kissei Pharmaceutical, Vico Therapeutics, Q-State Biosciences, Locanabio, Lexeo Therapeutics, Voyager Therapeutics, CRISPR Therapeutics, Capsida Biotherapeutics, AavantiBio, StrideBio, Wave Life Sciences, REPROCELL, SHIONOGI & Co., CORESTEM, Blade Therapeutics, Exicure, Lacerta Therapeutics, Healx Ltd., Uniqure, Ionis Pharmaceuticals, Jupiter Neurosciences, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

- Duchenne (SGT-003): Participant dosing ongoing in the Phase 1/2 INSPIRE DUCHENNE trial; Solid on track to discuss accelerated pathways with U.S. FDA later in 2025 - - FA (SGT-212): Dosing of first participant anticipated in the second half of 2025 - - CPVT (SGT-501): FDA IND on track for submission first half of 2025 - - Cash: Company ended first quarter 2025 with $306.9 million in cash, cash equivalents, investments and available-for-sale securities; Solid has anticipated cash runway into the first half of 2027 - CHARLESTOWN, Mass., May 15, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision, next generation, genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the first quarter ended March 31, 2025, and provided a business update. Bo Cumbo, President and CEO of Solid, commented: “The positive initial three patient, 90-day data in the INSPIRE DUCHENNE trial of SGT-003, our next-generation, investigational gene therapy to treat Duchenne muscular dystrophy, were a milestone in the development of a meaningful treatment candidate for this terrible disease. With the initial data demonstrating robust microdystrophin expression and improvements across biomarkers of muscle integrity and, excitingly, encouraging biomarkers of cardiac and liver health, we continue to dose participants as we prepare to engage with the FDA later this year on the potential for accelerated pathways. “To our patient communities, we know that safety is a critical factor in choosing any medical therapy. SGT-003, utilizing our proprietary, rationally designed capsid, AAV-SLB101, contains the lowest dose of any Duchenne gene therapy currently marketed or in clinical development, and to date, has demonstrated an encouraging safety and tolerability profile. We look forward to the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) where, on Saturday, we will provide an interim safety update from the INSPIRE DUCHENNE trial during our late-breaking oral presentation. “As an organization, we are intensely focused on driving innovation and advancing the C> field with the aspiration to make gene therapies safer, more effective, and truly accessible. These efforts originate with our differentiated and thoughtfully designed neuromuscular and cardiac therapeutic pipeline and expand to the development and broad out-licensing of next-generation delivery technologies, beginning with AAV-SLB101. We look forward to executing across these objectives over the coming quarters to drive meaningful value for all of our stakeholders,” Mr. Cumbo concluded. Company Updates Neuromuscular Pipeline SGT-003 for Duchenne Muscular Dystrophy (Duchenne) As announced on February 18, 2025, the Company reported positive initial clinical data from the first three participants dosed in the Phase 1/2 INSPIRE DUCHENNE trial. SGT-003 has been well tolerated in all participants dosed to date, with no treatment emergent serious adverse events (SAEs) or AEs of acute liver injury observed. The INSPIRE DUCHENNE trial continues dosing participants across multiple cohorts, and the Company expects to dose approximately 20 total participants by year-end. The INSPIRE DUCHENNE trial now has eight active sites in the U.S., Canada and Italy, with additional sites expected to be activated by year-end. The Company plans to meet with the FDA later this year to share safety and efficacy results from additional treated participants in the INSPIRE DUCHENNE study and to discuss accelerated regulatory pathways. The Company expects the meeting to occur in the fourth quarter of 2025. SGT-003 has been well tolerated in all participants dosed to date, with no treatment emergent serious adverse events (SAEs) or AEs of acute liver injury observed. SGT-212 for Friedreich’s Ataxia (FA) As announced on January 7, 2025, the FDA has cleared the IND for SGT-212 for the treatment of FA. SGT-212 is the first gene therapy candidate for FA to utilize a dual route of administration that was designed to promote restoration of therapeutic levels of the frataxin protein to address neurologic, cardiac and systemic clinical manifestations of FA. The Company expects to initiate a first-in-human, open-label, Phase 1b clinical trial of SGT-212 in the second half of 2025. The trial is expected to enroll non-ambulatory and ambulatory adult participants living with FA across up to three cohorts and is designed to evaluate the safety and tolerability of systemic and bilateral IDN administration of SGT-212. Cardiac Pipeline SGT-501 for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) IND-enabling Good Laboratory Practice (GLP) toxicology studies of SGT-501 in non-human primates were completed in the first quarter of 2025. The Company anticipates submitting an IND for SGT-501 for the treatment of CPVT in 1H 2025. Platform Technologies – Capsids & Other Capsids & Promoters The Company is building multiple cardiac and neuromuscular next-generation capsid and promoter libraries with final capsid selection from the first cardiac capsid library anticipated in the fourth quarter of 2025. AAV-SLB101 is the Company’s proprietary capsid used in SGT-003, which in the initial three participants dosed in the INSPIRE DUCHENNE study demonstrated rapid and robust levels of vector transduction, microdystrophin expression, and reduced liver targeting. Solid currently has partnership agreements with 19 academic labs, institutions and corporations for the use of AAV-SLB101, with additional partnerships expected to be executed over the coming quarters. First Quarter 2025 Financial Highlights Cash Position: Solid had $306.9 million in cash, cash equivalents, and available-for-sale securities as of March 31, 2025, compared to $148.9 million as of December 31, 2024. The Company expects that its cash, cash equivalents, and available-for-sale securities as of March 31, 2025, will enable it to fund its operational runway into the first half of 2027. Research and Development (R&D) Expenses: R&D expenses for the first quarter of 2025 were $30.9 million, compared to $18.9 million for the first quarter of 2024. The increase of $12.0 million was primarily due to a $7.4 million increase in costs for SGT-003 primarily related to manufacturing, regulatory, and clinical costs, a $1.9 million increase in personnel-related expenses, a $1.3 million increase in other development program expenses primarily due to manufacturing costs, a $0.8 million increase in costs for SGT-212 primarily related to regulatory and clinical costs, and a $0.7 million increase in costs for SGT-501 primarily related to research, regulatory, and clinical costs. General and Administrative (G&A) Expenses: G&A expenses for the first quarter of 2025 were $9.1 million, compared to $8.0 million for the first quarter of 2024. The increase of $1.1 million was primarily due to a $1.7 million increase in personnel-related costs, partially offset by a $0.4 million decrease in other G&A expenses. Net Loss: Net loss for the first quarter of 2025 was $39.3 million, compared to $24.3 million for the first quarter of 2024. About Solid Biosciences Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including Duchenne muscular dystrophy (Duchenne), Friedreich’s ataxia (FA), catecholaminergic polymorphic ventricular tachycardia (CPVT), TNNT2-mediated dilated cardiomyopathy, BAG3-mediated dilated cardiomyopathy, and additional fatal, genetic cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid’s mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit www.solidbio.com. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the company; the ability to successfully achieve and execute on the company’s goals, priorities and key clinical milestones; the company’s SGT-003 and SGT-212 programs, including expectations for additional CTA filings, site activations, planned enrollment, planned regulatory interactions and the potential accelerated approval pathway for SGT-003; the company’s expectations for submission of INDs; the cash runway of the company and the sufficiency of the Company’s cash, cash equivalents, and available-for-sale securities to fund its operations; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the company’s ability to advance SGT-003, SGT-212, SGT-501, SGT-601 and other preclinical programs, capsid libraries and other enabling technologies on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of the company’s product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; replicate preliminary or interim data from early-stage clinicals trials in the final data of such trials; compete successfully with other companies that are seeking to develop Duchenne, FA, CPVT and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, SGT-212, SGT-501, SGT-601 and other candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof and should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so. Solid Biosciences Investor Contact: Nicole Anderson Director, Investor Relations and Corporate Communications Solid Biosciences Inc. investors@solidbio.com Media Contact: Glenn Silver FINN Partners glenn.silver@finnpartners.com This press release was published by a CLEAR® Verified individual. Source: Solid Biosciences Inc.

Clinical Trials of Budoprutug in Primary Membranous Nephropathy (pMN), Immune Thrombocytopenia (ITP), and Systemic Lupus Erythematosus (SLE) on Track to Initiate in 2025 CLYM116 Progressing Towards Anticipated IND or CTA Submission in Second Half 2025 Appointed Kim Cobleigh Drapkin, CPA, and Bo Cumbo as Independent Directors and Perrin Wilson, Ph.D., as Chief Business Officer Strong Financial Position, with Cash Runway Expected Through 2027 WELLESLEY HILLS, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Climb Bio, Inc. (Nasdaq: CLYM), a clinical stage biotechnology company developing therapeutics for patients with immune-mediated diseases, today reported financial results for the first quarter ended March 31, 2025, and provided business updates. “2025 is a critical year of execution for Climb Bio and we continue to make excellent progress developing a differentiated pipeline targeting immune-mediated diseases with expansive therapeutic and commercial potential,” said Aoife Brennan, President and CEO of Climb Bio. “Our most advanced program, budoprutug, a potential best-in-class anti-CD19 monoclonal antibody designed to treat B-cell mediated diseases, remains on track to initiate clinical studies in ITP and SLE in the coming weeks and in pMN in the second half of 2025. We plan to provide clarity on the anticipated timing of clinical readouts later this year when enrollment dynamics are clearer. In parallel, we are advancing the subcutaneous formulation of budoprutug and subject to regulatory clearance, anticipate initiating a Phase 1 clinical trial in healthy volunteers in the second half of the year.” Dr. Brennan continued, “We have also made progress with our CLYM116 program, a potential best-in-class anti-APRIL monoclonal antibody with potential to provide therapeutic benefit to patients living with IgA nephropathy and other B-cell mediated diseases. We look forward to sharing detailed preclinical data from the program, including pharmacokinetic and pharmacodynamic markers of biological activity, in the second half of 2025 and we anticipate submitting an investigational new drug (IND) or clinical trial application (CTA) for CLYM116 by year end.” First Quarter 2025 and Recent Highlights FDA clearance for budoprutug Phase 2 pMN clinical trial. In March 2025, the Company announced that it had received clearance from the FDA to initiate a Phase 2 clinical trial of budoprutug in patients with pMN. This open-label, dose-ranging trial is designed to further evaluate the efficacy and safety of budoprutug in pMN. FDA clearance of budoprutug IND in ITP. In March 2025, the Company announced that it had received clearance from the FDA of its IND to initiate a Phase 1b/2a clinical trial of budoprutug in patients with ITP. In parallel, the Company is also pursuing ex-U.S. regulatory clearance for this clinical trial. This open-label, dose escalation and expansion trial is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary clinical efficacy of budoprutug in ITP. Pursuing ex-U.S. regulatory clearance for budoprutug clinical trial in SLE. Following the Company’s receipt of clearance from the FDA of its IND for SLE, the Company is also pursuing regulatory clearance to initiate the Phase 1b clinical trial of budoprutug in SLE at sites outside the United States. The open-label, single ascending dose study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary signs of clinical efficacy of budoprutug in SLE. Completed studies supporting a cell line switch for budoprutug. In March 2025, the Company announced that it was advancing the productivity and scalability of the manufacturing process for budoprutug to support later-stage clinical development. Expanded pipeline to include CLYM116, an antibody targeting the APRIL pathway for IgAN. In January 2025, Climb Bio entered into a technology transfer and exclusive license agreement with Beijing Mabworks Biotech Co., Ltd. (Mabworks) for the rights to develop and commercialize CLYM116 in the territory outside of Greater China. CLYM116 is a highly potent, Fc-engineered antibody that has the potential to enable more rapid, deep, and durable inhibition of APRIL signaling through its novel, pH-dependent mechanism of action. CLYM116 is currently in IND-enabling studies and the Company anticipates submitting an IND or CTA by year end. Appointed Kim Cobleigh Drapkin, CPA, and Bo Cumbo as Independent Directors in April 2025. Kim Cobleigh Drapkin, CPA, is a seasoned financial leader with over 30 years of experience guiding private and publicly traded biotechnology and pharmaceutical companies through strategic growth, financial planning, capital raises, and transformative transactions. Ms. Drapkin most recently served as Chief Executive Officer of Graphite Bio. Bo Cumbo brings over 30 years of experience in the pharmaceutical and biotechnology industries, with a proven track record of leading successful commercial launches for 11 specialty and rare disease therapies. Mr. Cumbo currently serves as President, Chief Executive Officer, and Director of Solid Biosciences. Appointed Perrin Wilson, Ph.D., as Chief Business Officer in February 2025. Dr. Wilson has over 17 years of experience in the pharmaceutical and biotech industry and has deep expertise in business development and commercial strategy. During her career, she has led brand strategy and launch preparations and has overseen multiple successful acquisitions and integrations. Anticipated Milestones Budoprutug (anti-CD19 monoclonal antibody): ITP Phase 1b/2a study - first patient in (H1 2025) SLE Phase 1b study - first patient in (H1 2025) pMN Phase 2 study - first patient in (H2 2025) Subcutaneous formulation - obtain additional non-clinical data (H1 2025) and initiation of a Phase 1 clinical trial in healthy volunteers (H2 2025) CLYM116 (anti-APRIL monoclonal antibody): Reporting preclinical data (H2 2025) and submission of IND or CTA (H2 2025) ITP Phase 1b/2a study - first patient in (H1 2025) SLE Phase 1b study - first patient in (H1 2025) pMN Phase 2 study - first patient in (H2 2025) Subcutaneous formulation - obtain additional non-clinical data (H1 2025) and initiation of a Phase 1 clinical trial in healthy volunteers (H2 2025) Reporting preclinical data (H2 2025) and submission of IND or CTA (H2 2025) First Quarter 2025 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $197.8 million as of March 31, 2025. Cash, cash equivalents and marketable securities are expected to fund operations through 2027. Research and Development (R&D) expenses: R&D expenses were $17.3 million for the three months ended March 31, 2025, including the $9.0 million upfront payment made to Mabworks in accordance with the license agreement, compared to $1.1 million for the comparable period in 2024. General and Administrative (G&A) expenses: G&A expenses were $5.7 million for the three months ended March 31, 2025, compared to $1.9 million for the comparable period in 2024. Other income, net: Other income, net was $2.2 million for the three months ended March 31, 2025, compared to $1.3 for the comparable period in 2024. About Climb Bio, Inc. Climb Bio, Inc. is a clinical-stage biotechnology company developing therapeutics for patients with immune-mediated diseases. The Company’s pipeline includes, budoprutug, an anti-CD19 monoclonal antibody that has demonstrated B-cell depletion and has potential to treat a broad range of B-cell mediated diseases, and CLYM116, an anti-APRIL monoclonal antibody currently in IND-enabling studies for IgA nephropathy. For more information, please visit climbbio.com. About Budoprutug Budoprutug is a clinical-stage, anti-CD19 monoclonal antibody being developed by Climb Bio to address a broad range of B-cell mediated, immune-driven diseases. Designed with enhanced effector function and low picomolar affinity, budoprutug targets and depletes CD19-expressing B cells, including plasma blasts that are key sources of pathogenic autoantibodies. Climb Bio plans to evaluate budoprutug in multiple clinical trials across three lead indications—primary membranous nephropathy (pMN), immune thrombocytopenia (ITP), and systemic lupus erythematosus (SLE)—which represent distinct mechanistic subtypes of immune-mediated disease. Early clinical data suggest budoprutug may offer durable B-cell depletion, rapid reductions in autoantibodies, and clinical remission in pMN. A subcutaneous formulation is also in development to enable broader patient access and potential home-based dosing. Budoprutug has been granted orphan drug designation by the FDA for the treatment of pMN. About CLYM116 CLYM116 is a preclinical-stage monoclonal antibody targeting APRIL (A Proliferation-Inducing Ligand), a key driver of pathogenic B-cell activity in autoimmune diseases. CLYM116 employs a novel pH-dependent bind-and-release mechanism to potently block APRIL signaling, promote lysosomal degradation of APRIL, and recycle the antibody to extend its half-life. This differentiated design offers the potential for rapid, deep, and durable inhibition of APRIL with a favorable safety profile and less frequent dosing. CLYM116 is being advanced for the treatment of IgA nephropathy (IgAN), with plans to initiate a Phase 1 clinical trial following completion of IND-enabling studies and subject to regulatory clearance. The molecule may also have broader utility across other B-cell mediated diseases where APRIL plays a critical role. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including without limitation statements regarding: future expectations, plans and prospects for Climb Bio; expectations regarding the therapeutic benefits, clinical potential and clinical development of budoprutug and CLYM116; the trial design for the planned clinical trials of budoprutug; the anticipated timelines for initiating clinical trials of budoprutug for primary membranous nephropathy, immune thrombocytopenia and systemic lupus erythematosus; plans to optimize the administration of budoprutug; the anticipated benefits of Climb Bio’s license agreement with Mabworks; expectations regarding the timing of an investigational new drug application or clinical trial application submission for CLYM116; the sufficiency of Climb Bio’s cash resources for the period anticipated; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “will,” “working” and similar expressions. Forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. Climb Bio may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. These risks and uncertainties include, but are not limited to, important risks and uncertainties associated with: the ability of Climb Bio to timely and successfully achieve or recognize the anticipated benefits of its acquisition of Tenet Medicines, Inc. and its license agreement with Mabworks; changes in applicable laws or regulation; the possibility that Climb Bio may be adversely affected by other economic, business and/or competitive factors; Climb Bio’s ability to advance budoprutug and CLYM116 on the timelines expected or at all and to obtain and maintain necessary approvals from the U.S. Food and Drug Administration and other regulatory authorities; obtaining and maintaining the necessary approvals from investigational review boards at clinical trial sites and independent data safety monitoring boards; replicating in clinical trials positive results found in early-stage clinical trials; competing successfully with other companies that are seeking to develop treatments for primary membranous nephropathy, immune thrombocytopenia, systemic lupus erythematosus, IgA nephropathy and other immune-mediated diseases; maintaining or protecting intellectual property rights related to budoprutug, CLYM116 and/or its other product candidates; managing expenses; and raising the substantial additional capital needed, on the timeline necessary, to continue development of budoprutug, CLYM116 and any other product candidates Climb Bio may develop. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Climb Bio’s actual results to differ materially from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in Climb Bio’s most recent filings with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Climb Bio’s views as of the date hereof and should not be relied upon as representing Climb Bio’s views as of any date subsequent to the date hereof. Climb Bio anticipates that subsequent events and developments will cause Climb Bio’s views to change. However, while Climb Bio may elect to update these forward-looking statements at some point in the future, Climb Bio specifically disclaims any obligation to do so, except as required by law. Investors and Media Carlo Tanzi, Ph.D. Kendall Investor Relations ctanzi@kendallir.com