Latest news with #Duchennemuscular

Business Wire

21-07-2025

Business
Business Wire

Rosen Law Firm Urges Capricor Therapeutics, Inc. (NASDAQ: CAPR) Stockholders with Large Losses to Contact the Firm for Information About Their Rights

NEW YORK--(BUSINESS WIRE)--Rosen Law Firm, a global investor rights law firm, announces that a shareholder filed a class action lawsuit on behalf of purchasers and acquirers of Capricor Therapeutics, Inc. (NASDAQ: CAPR) securities between October 9, 2024 and July 10, 2025, both dates inclusive (the 'Class Period'). Capricor is a clinical-stage biotechnology company. For more information, submit a form, email attorney Phillip Kim, or give us a call at 866-767-3653. The Allegations: Rosen Law Firm is Investigating the Allegations that Capricor Therapeutics, Inc. (NASDAQ: CAPR) Misled Investors Regarding its Business Operations. According to the lawsuit, throughout the Class Period, defendants provided investors with material information concerning deramiocel, Capricor's lead cell therapy candidate drug for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). Defendants' statements included, among other things, Capricor's ability to obtain a Biologics License Application (BLA) for deramiocel from the U.S. Food and Drug Administration (FDA). Further, defendants provided these overwhelmingly positive statements to investors while, at the same time, disseminating false and misleading statements and/or concealing material adverse facts concerning its four-year safety and efficacy data from its Phase 2 HOPE-2 trial study of deramiocel. The lawsuit alleges this caused shareholders to purchase Capricor's securities at artificially inflated prices. When the true details entered the market, the lawsuit claims that investors suffered damages. What Now: You may be eligible to participate in the class action against Capricor Therapeutics, Inc. Shareholders who want to serve as lead plaintiff for the class must file their motions with the court by September 15, 2025. A lead plaintiff is a representative party who acts on behalf of other class members in directing the litigation. You do not have to participate in the case to be eligible for a recovery. If you choose to take no action, you can remain an absent class member. For more information, click here. All representation is on a contingency fee basis. Shareholders pay no fees or expenses. About Rosen Law Firm: Some law firms issuing releases about this matter do not actually litigate securities class actions. Rosen Law Firm does. Rosen Law Firm is a recognized leader in shareholder rights litigation, dedicated to helping shareholders recover losses, improving corporate governance structures, and holding company executives accountable for their wrongdoing. Since its inception, Rosen Law Firm has obtained over $1 billion for shareholders. Attorney Advertising. Prior results do not guarantee a similar outcome.

Major biotech stock tumbles 35 percent after major rare disease setback

Miami Herald

18-07-2025

Business
Miami Herald

Major biotech stock tumbles 35 percent after major rare disease setback

Biotech stories typically tread a very fine line between promise and pressure. Sometimes a company that's been a pioneer in rare diseases suddenly finds itself at a crossroads. Don't miss the move: Subscribe to TheStreet's free daily newsletter With years of headline-making science behind it, one major biotech stock is now facing a moment that flips the script on how investors and regulators see its future. It's not about a singular decision, but a pattern coming into focus. What's surfacing now is a test of just how far early bets can stretch. Image source: Nagle/Bloomberg via Getty Images Sarepta Therapeutics (SRPT) has been at the core of the conversation around Duchenne muscular dystrophy (DMD) treatments, but it hasn't been without its controversies. Back in 2016, Sarepta won FDA approval for Exondys 51, the first drug to target a specific DMD mutation. It got the approval through the agency's accelerated pathway, which fast-tracks promising treatments to reach patients based on early data. However, that decision sparked debate. The clinical trial included just 12 boys, and while dystrophin, a key muscle protein, saw a boost, its skeptics questioned whether that translated into real-world benefits. Related: JPMorgan reveals 9 stocks with major problems Europe's regulators dismissed the drug in 2018, and U.S. insurers placed tight restrictions on coverage. Nevertheless, the therapy carved out a strong niche, serving a small group of patients with exon 51 mutations. Sarepta followed things up with Vyondys 53, another exon-skipping treatment, and saw a similar pattern emerge. It followed an initial pushback, eventual FDA approval, and ongoing requests for stronger data from insurers and clinicians. Even with these challenges, the DMD market remains strong. The disease affects close to 13,000 boys in the U.S., and treatments topped $2 billion in North American sales last year. Globally, that market could potentially reach $6.5 billion by 2034. More News: Moody's drops 2-word warning on housing marketRigetti shakes up quantum computing with bold advanceTikTok's next move has Google and Meta sweating bullets Sarepta's biggest bet came with Elevidys, a gene therapy priced at $3.2 million per dose. The treatment was estimated to help roughly 90% of U.S. DMD patients. That massive scale and price reignited debate around cost, access, and evidence. The company's path has been ambitious. However, with investor confidence shaken by safety questions and shifting strategy, the road ahead seems mostly rocky. Sarepta Therapeutics is feeling the heat after a third patient death linked to its gene therapy program came to light. Following the development, its stock tanked roughly 35% on July 18, having lost 88% year-to-date. The company confirmed that an early-stage trial patient being treated for limb-girdle muscular dystrophy died last month from acute liver failure. That news adds to the scrutiny over the safety of Sarepta's experimental treatments. This follows two earlier deaths tied to Elevidys, a gene therapy for DMD developed with Roche (RHHBY). Related: UnitedHealth's shocking move to please Wall Street Those patients were said to be in advanced stages of the disease and had lost their ability to walk. Sarepta says it acted quickly, notifying regulators and investigators "in an appropriate and timely manner." Nonetheless, the fallout is growing. The company has shut down a related clinical trial and paused the distribution of Elevidys for non-ambulatory patients. It's discussing safety protocols for high-risk groups with the FDA. Adding to the duress, Sarepta announced a massive corporate shake-up this week. It rolled out a sweeping restructuring plan, cutting 36% of its workforce (500 jobs). It's also looking to stop producing multiple drug programs to focus more tightly on siRNA and gene-silencing therapies, which potentially could be a lot more promising pipeline bets. Management expects these cost-cutting measures to save around $400 million annually starting in 2026. Given the combination of patient deaths, halted trials, and a shift away from some therapies, Sarepta may now be entering a major reset phase. Related: One Washington move could shake Big Pharma to its core The Arena Media Brands, LLC THESTREET is a registered trademark of TheStreet, Inc.

[Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients

Business Upturn

15-06-2025

Health
Business Upturn

[Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients

After a thorough clinical review, the benefit-risk for the use of Elevidys in non-ambulatory patients with Duchenne has been re-assessed, following two cases of fatal acute liver failure Effective immediately, dosing of non-ambulatory patients, irrespective of age, is paused in the clinical setting; dosing of non-ambulatory patients is discontinued in the commercial setting Roche is working closely with relevant health authorities, investigators and prescribing physicians to ensure they are informed and patient care is being appropriately modified The benefit-risk of Elevidys treatment in ambulatory Duchenne patients remains positive and treatment guidance is unchanged Basel, 15 June 2025 – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ (delandistrogene moxeparvovec), for non-ambulatory Duchenne muscular dystrophy (DMD) patients, irrespective of age, in both clinical and commercial settings. In the commercial setting, non-ambulatory patients should no longer receive Elevidys. In the clinical trial setting, enrolment and dosing of non-ambulatory patients will be immediately paused until additional risk mitigation measures (e.g. immune modulatory treatment) are implemented in the study protocol. Health authorities, investigators and physicians are being informed so that patient care can be quickly adjusted. This decision follows careful assessment of two cases in non-ambulatory patients of fatal acute liver failure (ALF), an identified risk of Elevidys and other AAV-mediated gene therapies, which led to a reassessment of the benefit-risk profile as unfavourable for patients with DMD who are non-ambulatory. The new dosing restrictions do not impact the treatment of ambulatory DMD patients of any age, and the benefit-risk ratio remains positive in the ambulatory patient population. 'We are deeply saddened by the loss of these two young men and are urgently working to mitigate any risks related to the use of Elevidys,' said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. 'Patient safety is always our highest priority. Therefore, we have recommended halting treatment with Elevidys in non-ambulatory patients with immediate effect.' DMD is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. The two fatal ALF cases occurred in non-ambulatory patients, out of a total of approximately 140 non-ambulatory patients treated with Elevidys globally to date. Following the first case of fatal ALF, European regulators requested that Roche and Sarepta put temporary clinical holds on Elevidys studies 104 (NCT06241950), 302 (ENVOL, NCT06128564) and 303 (ENVISION Study 303, NCT05310071). The temporary clinical holds are still in effect. Outside of Europe, dosing will be paused, effective immediately, for the ENVISION trial. The dosing restrictions will also go into effect for future dosing of commercial non-ambulatory patients. Elevidys has been approved by regulatory authorities in eight Roche territories for the treatment of DMD including Bahrain, Brazil, Israel, Japan, Kuwait, Oman, Qatar, and the UAE. In 2019, Roche entered into a global collaboration agreement with Sarepta Therapeutics, Inc. to commercialise Elevidys in territories outside the U.S. Roche and Sarepta jointly manage the clinical studies for Elevidys. Roche is the sponsor of the ENVOL study; Sarepta is the sponsor for all other studies. Overview of the Elevidys clinical development programme Studies in non-ambulatory people with DMD Ongoing ENVISION (Study 303, NCT05881408), a global Phase III study investigating the safety and efficacy of Elevidys in participants who are ambulatory (aged 8 to <18 years old) and non-ambulatory (no age limitation). This study is already on temporary clinical hold in Europe. Outside of Europe, recruitment will be paused. ENDEAVOR (Study 103, NCT04626674), a two-part, open-label, Phase Ib study assessing Elevidys micro-dystrophin protein expression and safety of Elevidys in seven cohorts of boys with Duchenne, across different ages, mutations and stages of disease progression. No longer recruiting; long term follow up ongoing. Studies in ambulatory people with DMD Study 101 (NCT03375164), a Phase I/II study evaluating the safety of Elevidys in four ambulatory participants aged 4 to <8 years old with Duchenne. The study is complete. Study 102 (NCT03769116), a Phase II clinical trial evaluating the safety and efficacy of Elevidys in patients with Duchenne aged 4 to <8 years. The study is complete. Study 104 (NCT06241950), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys in association with imlifidase in individuals aged 4 to 9 years with pre-existing antibodies to recombinant adeno-associated virus serotype, rAAVrh74. The study is on temporary clinical hold in Europe. HORIZON (Study 105, NCT06597656), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys following plasmapheresis in individuals aged 4 to 8 years with pre-existing antibodies to adeno-associated virus serotype, AAVrh74. The study is recruiting ambulatory patients. EMBARK (Study 301, NCT05096221), a multinational, Phase III, randomised, double-blind, placebo-controlled study assessing the safety and efficacy of Elevidys in ambulatory boys aged 4 to 7 years. The study duration is two years. The study is complete. ENVOL (Study 302, NCT06128564), a Phase II study evaluating the safety of Elevidys and expression of Elevidys micro-dystrophin protein in young children, including babies and newborns. The study is on temporary clinical hold in Europe and the UK. EXPEDITION (Study 305, NCT05967351), a Phase III long-term five-year follow-up study evaluating the safety and efficacy of Elevidys in those who have received Elevidys in a previous clinical study. EXPEDITION is enrolling by invitation. About Elevidys™ (delandistrogene moxeparvovec) Elevidys is a one-time treatment administered through a single intravenous dose and the first and only approved gene therapy for Duchenne. It is designed to target the underlying cause of Duchenne by delivering new instructions to cells to produce Elevidys-dystrophin in skeletal, respiratory and cardiac muscles. Elevidys aims to slow the progression of Duchenne by delaying the need for a wheelchair, protecting the heart from damage and a person's ability to breathe without a respirator for as long as possible. Elevidys uses adeno-associated virus (AAV) vector technology and consists of three parts: a transgene, promoter and vector. Its unique construct optimises delivery to all muscle types, including those of interest for Duchenne treatment. A robust clinical trial programme to understand its potential in a broad range of people with Duchenne, of all ages, ambulatory status and a wide range of DMD gene mutations is ongoing. To date, more than 900 individuals with Duchenne have been treated with Elevidys through Roche's clinical development program and in real-world settings. Elevidys has already been approved for the treatment of DMD by 10 regulatory authorities around the world, including the US and Japan. Elevidys is being developed by Roche in collaboration with Sarepta Therapeutics. About Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Duchenne primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. Average life expectancy is only 28 years. The physical, emotional and financial impact of Duchenne on those affected, their families and caregivers, is profound. Duchenne is an X-linked, rare neuromuscular disease caused by pathogenic variants (mutations) in the DMD gene that disrupt the production of functional dystrophin protein, leading to progressive and irreversible muscle weakness, diminished quality of life and premature death. Dystrophin strengthens and protects muscles and without it, normal activity causes excessive damage to muscle cells as they are more sensitive to injury. Over time, muscle tissue is replaced with scar tissue and fat, causing muscles to weaken. Although Duchenne progresses differently in each individual, its devastating trajectory is well established. Those with Duchenne will eventually lose the ability to use and move their limbs, to breathe on their own and are susceptible to respiratory infections. Muscle damage to the heart causes cardiomyopathy, including rhythm abnormalities and heart failure. Early diagnosis is important for timely intervention to prolong muscle function and preserve quality of life. There is a critical need for disease-modifying treatments that address the underlying cause of DMD before irreversible muscle loss occurs. About Roche in Neuroscience Neuroscience is a major focus of research and development at Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases. Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer's disease, Huntington's disease, Parkinson's disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today. About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world's largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit All trademarks used or mentioned in this release are protected by law. Roche Global Media Relations Phone: +41 61 688 8888 / e-mail: [email protected] Hans Trees, PhD Phone: +41 79 407 72 58 Sileia Urech Phone: +41 79 935 81 48 Nathalie Altermatt Phone: +41 79 771 05 25 Lorena Corfas Phone: +41 79 568 24 95 Simon Goldsborough Phone: +44 797 32 72 915 Karsten Kleine Phone: +41 79 461 86 83 Nina Mählitz Phone: +41 79 327 54 74 Kirti Pandey Phone: +49 172 6367262 Yvette Petillon Phone: +41 79 961 92 50 Dr Rebekka Schnell Phone: +41 79 205 27 03 Roche Investor Relations Investor Relations North America Loren KalmPhone: +1 650 225 3217 e-mail: [email protected] Attachment Ad Hoc Media Investor Release Update on Elevidys English Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. Ahmedabad Plane Crash

Capricor Therapeutics Inc (CAPR) Q1 2025 Earnings Call Highlights: Navigating Challenges with ...

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

Henley MP demands apology from Oxford's hospital trust

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.