Latest news with #Duchennemuscular
Yahoo
14-05-2025
- Business
- Yahoo
Capricor Therapeutics Inc (CAPR) Q1 2025 Earnings Call Highlights: Navigating Challenges with ...
Cash Position: Approximately $144.8 million as of March 31, 2025. Revenue: $0 for Q1 2025, compared to approximately $4.9 million for Q1 2024. Research and Development Expenses: Approximately $16.2 million for Q1 2025, compared to approximately $10.1 million in Q1 2024. General and Administrative Expenses: Approximately $3.1 million for Q1 2025, compared to approximately $1.8 million in Q1 2024. Net Loss: Approximately $24.4 million for Q1 2025, compared to a net loss of approximately $9.8 million for Q1 2024. Warning! GuruFocus has detected 4 Warning Signs with CAPR. Release Date: May 13, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Capricor Therapeutics Inc (NASDAQ:CAPR) is on track with its BLA seeking full approval for its product candidate, damy cell, for treating Duchenne muscular dystrophy (DMD) cardiomyopathy. The company has a strong safety record for damy cell, demonstrated in over 700 infusions treating more than 250 patients. Capricor Therapeutics Inc (NASDAQ:CAPR) is transitioning from a translational medicine company to a commercial stage entity, actively working with its commercial partner NS Pharma on launch readiness in the United States. The company has a robust cash position with approximately $145 million, providing a financial runway into 2027 without additional cash infusions. Capricor Therapeutics Inc (NASDAQ:CAPR) is actively expanding its manufacturing capabilities, with plans to operationalize additional clean rooms by mid to late 2026 to meet potential demand. Revenues for the first quarter of 2025 were zero, compared to approximately $4.9 million in the first quarter of 2024. Operating expenses have increased, with research and development expenses rising to approximately $16.2 million in Q1 2025 from $10.1 million in Q1 2024. The net loss for the first quarter of 2025 was approximately $24.4 million, compared to a net loss of $9.8 million in the first quarter of 2024. There is uncertainty regarding the outcome of the FDA advisory committee meeting, which could impact the approval process for damy cell. Negotiations for European distribution with Nippon Shinyaku have been extended, indicating potential delays or challenges in finalizing agreements for the European market. Q: Have you had your site inspection in San Diego, and what are the key features of your preparation for the FDA advisory committee meeting? A: We haven't had our pre-licensing inspection yet, but it's scheduled for this quarter. Our facility was built for commercial manufacturing, so we feel prepared. Regarding the advisory committee meeting, we've had two mock sessions and passed with flying colors. The FDA is actively reviewing our file, and we are confident in our data and preparation. Q: Can you provide an update on the negotiations with Nippon Shinyaku for the European market, and how is NS Pharma preparing for the U.S. launch? A: We are in active negotiations with Nippon Shinyaku for Europe and are also exploring launching on our own. In the U.S., NS Pharma has built a strong sales and marketing team for Viltepso, and they are preparing to launch Daryel with 125 FTEs focusing on it. We are enhancing our management team to support this effort. Q: What are the key drivers that physicians see as proof of the efficacy of your therapy, and will you have four-year data to share at the advisory committee meeting? A: The key driver is the statistical significance of cardiac MRI data, which objectively measures cardiac function. We plan to present four-year data at the PPMG meeting in June, showing long-term stabilization in cardiac function, which is promising. Q: If the FDA issues a CRL for efficacy, what is your plan? Would you submit HOPE 3 data for DMD skeletal muscle function? A: Yes, if a CRL is issued, we would submit the HOPE 3 data for skeletal muscle dysfunction, as it is fully enrolled and expected to be positive. We would apply for both cardiac and skeletal indications based on the data. Q: How does the FDA view LVF as a surrogate endpoint, and how are you supporting this with your data? A: The FDA does not view ejection fraction as a surrogate endpoint in this rare disease context. Instead, they consider it an outcome measure. We have used real-world evidence and natural history data to support our efficacy claims, showing significant improvement in treated patients. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
BBC News
04-04-2025
- Health
- BBC News
Henley MP demands apology from Oxford's hospital trust
An MP has urged a hospital trust to apologise for "misleading" the family of a boy with a degenerative illness. The Liberal Democrat MP for Henley and Thame, Freddie van Mierlo, said Oxford University Hospitals NHS Foundation Trust (OUH) assured him it was ready to roll out a new medication for people with Duchenne muscular dystrophy (DND). He told the family of 11-year-old Ben, from Henley, who has the condition, but the family was subsequently told by the trust it could not commit to a timescale. OUH said it was exploring how to deliver the drug, but funding challenges meant the process was "not straightforward". Mr van Mierlo said the trust told him it was ready to launch an Early Access Programme to allow patients to access givinostat during a meeting in drug, which has shown positive results in trials, was given conditional approval in the UK in MP said: "I took the trust at its word and shared the information with my constituent. "To later find out the trust was not ready and could not provide Ben with the treatment he needs was devastating for the family." Ben was diagnosed with DND in 2017. The condition progressively weakens muscles and can limit life expectancy. Alex Clarke, Ben's dad, said: "This drug could slow down the progression of Ben's condition, but we need action now. "Every day that passes without access to givinostat feels like we are running out of time. "I urge OUH to follow through on their earlier assurances and get Ben the treatment he urgently needs." Professor Andrew Brent, chief medical officer at OUH, said: "Unfortunately, although the drug is being offered for free by the company, there are many other costs to delivery of the treatment as the medication needs close monitoring which will require additional staffing resource and expertise. "We are currently investigating how we might do this, but it is not straightforward without NHS commissioning funding to support the service and at a time when we, like all of the NHS, are being asked reduce our costs."We want to do all we can support patients with Duchenne and their families, and are therefore exploring whether there is any way we can deliver givinostat without stopping other essential care." You can follow BBC Oxfordshire on Facebook, X (Twitter) or Instagram.
Associated Press
06-03-2025
- Business
- Associated Press
Italfarmaco Provides Overview on Givinostat as Treatment for Duchenne Muscular Dystrophy: Progress in Global Access, Regulatory Milestones and Clinical Trials
Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the company's drug for the treatment of Duchenne muscular dystrophy (DMD). The update highlights key regulatory milestones and ongoing clinical trials. Givinostat has been granted regulatory approval in the US (March 2024) and the UK (December 2024), marking critical milestones in the global effort to make the therapy available to patients. In the UK, givinostat was granted full approval for ambulant patients six years and older, while conditional approval was given for non-ambulant patients. In the US, full approval was granted for patients six years and older regardless of their ambulatory status, further emphasizing the treatment's potential across the disease spectrum. The European Medicines Agency (EMA) is currently reviewing the Marketing Authorisation Application (MAA), with a Committee for Medicinal Products for Human Use (CHMP) opinion expected in the first half of 2025. Regulatory filings in additional countries are actively progressing as part of Italfarmaco's commitment to global patient access. Carlos Barallobre, CEO of Italfarmaco, said, 'At Italfarmaco, our unwavering commitment is to the Duchenne community. The givinostat approvals in the US and UK, along with the promising long-term data we are generating, bring us closer to our goal: ensuring that every patient, regardless of location, has access to a therapy that can make a difference in disease progression. We are diligently working with global regulatory bodies to expedite availability, because we understand that for patients and families affected by DMD, every moment counts.' Ongoing Clinical Trials in DMD Three clinical trials are currently underway in Europe and Canada, designed to expand the potential use of givinostat and to provide long-term follow up on treated patients: NCT05933057: Evaluating non-ambulant patients aged 9 years and older NCT06769633: Evaluating pharmacokinetics and safety in younger patients aged 2 to 5 years, with recruitment recently completed for the first cohort of 4- to 5-year-olds NCT03373968: Confirming long-term tolerability and efficacy of givinostat treated patients, with up to eleven years follow-up Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group, stated, 'Our continuing clinical trial evaluation seeks to further affirm givinostat's clinical value in Duchenne muscular dystrophy, where treatment options remain limited, and by precisely targeting the biological drivers of muscle degeneration. We look forward to our ongoing interactions with the regulatory bodies and thank the clinicians for their tireless efforts and the DMD community and families for their continuing trust.' About Duchenne Muscular Dystrophy DMD is a progressive neuromuscular disorder caused by a mutation in the DMD gene which affects the production of a protein called dystrophin. 3 Dystrophin is a critical component of the dystrophin-associated protein complex (DAPC) which supports the strength, stability, function and repair of muscle cells. In DMD, muscle fibres are highly susceptible to injury and this continuous muscle injury leads to chronic inflammation, impairment of muscle regeneration and muscle replacement by fibrotic and fat tissue. 4, 5-7 The disease primarily affects boys, with symptoms usually first seen between two and five years of age. Symptoms worsen over time affecting the ability to walk. Eventually, heart and respiratory muscles are affected, which are the two main causes of premature death. 8 DMD is one of the most severe and common forms of childhood muscular dystrophy, with a worldwide birth incidence of around 1 in 5,050 boys. 9 About Givinostat Givinostat was discovered through Italfarmaco's research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). Givinostat is an orally administered histone deacetylases (HDAC) inhibitor. HDAC is activity upregulated in DMD muscle and has the potential to modify the expression of certain genes and biological processes involved in muscle repair and inflammation. 10, 11 About ITALFARMACO Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development and approval of many pharmaceutical products around the world. The Italfarmaco group has operations in more than 60 countries through directly controlled or affiliated companies. The company is a leader in pharmaceutical research, product development, production and commercialisation with proven success in many therapeutic areas including immuno-oncology, gynaecology, neurology, cardiovascular disease and rare diseases. Italfarmaco's rare disease unit includes programmes in Duchenne muscular dystrophy, Becker muscular dystrophy, amyotrophic lateral sclerosis and polycythaemia vera. References: Mercuri, E, Vilchez, JJ, Boespflug-Tanguy, O, Zaidman, CM, Mah, JK, Goemans, N. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024;23:393-403. Mercuri, E, Vilchez, JJ, Boespflug-Tanguy, O, Zaidman, CM, Mah, JK, Goemans, N. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Supplementary appendix. Lancet Neurol. 2024;23. Ryder S, Leadley RM, Armstrong N, et al. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review. Orphanet J Rare Dis. 2017;12(1):79. doi:10.1186/s13023-017-0631-3 Sandonà M, Cavioli G, Renzini A, et al. Histone Deacetylases: Molecular Mechanisms and Therapeutic Implications for Muscular Dystrophies. Int J Mol Sci. 2023;24(5):4306. Consalvi S, Saccone V, Giordani L, Minetti G, Mozzetta C, Puri PL. Histone Deacetylase Inhibitors in the Treatment of Muscular Dystrophies: Epigenetic Drugs for Genetic Diseases. Mol Med. 2011;17(5):457–465. Bez Batti Angulski A, Hosny N, Cohen H, et al. Duchenne muscular dystrophy: disease mechanism and therapeutic strategies. Front Physiol. 2023;14:1183101. Giuliani G, Rosina M, Reggio A. Signaling pathways regulating the fate of fibro/adipogenic progenitors (FAPs) in skeletal muscle regeneration and disease. FEBS J. 2022;289(21):6484–6517. Walter MC, Reilich P. Recent developments in Duchenne muscular dystrophy: facts and numbers. J Cachexia Sarcopenia Muscle. 2017;8(5):681–685. Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifirò G. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J Rare Dis. 2020;15(1):141. Comi G, Bertini E, Vita G, et al. S22.008: Development of the histone deacetylases inhibitor Givinostat in Duchenne Muscular Dystrophy. Poster. Neurology. 2018;90(15 (Supplement)). Licandro SA, Crippa L, Pomarico R, et al. The pan HDAC inhibitor Givinostat improves muscle function and histological parameters in two Duchenne muscular dystrophy murine models expressing different haplotypes of the LTBP4 gene. Skelet Muscle. 2021;11(1):19. Other enquiries: Patient Advocacy and Communications Lead| [email protected] KEYWORD: ITALY EUROPE SOURCE: Italfarmaco S.p.A. Copyright Business Wire 2025. PUB: 03/06/2025 05:00 AM/DISC: 03/06/2025 05:03 AM
Yahoo
10-02-2025
- Business
- Yahoo
Capricor Therapeutics to Participate in Upcoming Investor Conferences
SAN DIEGO, Feb. 10, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the Company is scheduled to participate in the following upcoming investor conferences. Event: Oppenheimer 35thAnnual Healthcare Life Sciences Conference (Virtual) Date: February 12, 2025 from 12:00-12:30 p.m. ET Webcast Link Click here Event: Barclays 27thAnnual Global Healthcare Conference (Miami, FL) Date: March 11-13, 2025 Event: 37thAnnual ROTH Conference (Dana Point, CA) Date: March 16-18, 2025 Capricor management will present updates on its lead program of deramiocel for the treatment of Duchenne muscular dystrophy (DMD) as well as provide other general scientific and corporate updates. For further information on these presentations, please visit Capricor's website at About Capricor Therapeutics Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. Deramiocel is currently in late-stage development for the treatment of Duchenne muscular dystrophy. Capricor is also harnessing the power of its exosome technology, using its proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit and follow Capricor on Facebook, Instagram and Twitter. Cautionary Note Regarding Forward-Looking Statements Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; the ability to achieve product milestones and to receive milestone payments from commercial partners; plans regarding current and future collaborative activities and the ownership of commercial rights; potential future agreements; scope, duration, validity and enforceability of intellectual property rights; future revenue streams and projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words 'believes,' 'plans,' 'could,' 'anticipates,' 'expects,' 'estimates,' 'should,' 'target,' 'will,' 'would' and similar expressions) should also be considered to be forward-looking statements. There are a number of important factors that could cause actual results or events to differ materially from those indicated by such forward-looking statements. More information about these and other risks that may impact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission on March 11, 2024, and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as filed with the Securities and Exchange Commission on November 14, 2024. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements. Capricor has entered into an agreement for the exclusive commercialization and distribution of deramiocel (CAP-1002) for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. Deramiocel is an Investigational New Drug and is not approved for any indications. None of Capricor's exosome-based candidates have been approved for clinical investigation. For more information, please contact: Capricor Media Contact:Raquel ConaKCSA Strategic Communications rcona@ Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@
