Latest news with #Elevidys

Sarepta downgraded to Market Perform from Outperform at BMO Capital

Business Insider

31 minutes ago

Business
Business Insider

Sarepta downgraded to Market Perform from Outperform at BMO Capital

BMO Capital analyst Kostas Biliouris downgraded Sarepta (SRPT) to Market Perform from Outperform with a price target of $70, down from $120. Elevidys treatment led to a second fatal case of acute liver failure in non-ambulatory patients, triggering management to look for potential changes to immunosuppressive regimen, the analyst tells investors in a research note. The firm believes the second death is part of Adeno-associated virus risk and Elevidys' benefit/risk remains favorable in ambulatory patients. However, BMO expects the news news to add pressure on Sarepta moving forward due to uncertainty around a potential withdrawal of Elevidys non-ambulatory approval. The company could also miss its Elevidys 2025 guidance or announce additional patient deaths, adds BMO.

Sarepta suspends non-ambulatory Elevidys shipments after second death

Business Insider

10 hours ago

Business
Business Insider

Sarepta suspends non-ambulatory Elevidys shipments after second death

Sarepta (SRPT) Therapeutics provided a safety update regarding Elevidys, an FDA approved gene therapy for patients with Duchenne muscular dystrophy. The company announced a second reported case of acute liver failure resulting in death. The cases of ALF to date have both occurred in non-ambulatory individuals with Duchenne. Sarepta is temporarily suspending shipments of Elevidys for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place. For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after Elevidys infusion, along with post-treatment monitoring, remains the same. Sarepta has also voluntarily paused dosing in the ENVISION clinical study. FDA concurs with this action, the company said. 'The pause will allow for the evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for the non-ambulatory patient cohort and incorporate any additional feedback from the FDA. Regulatory alignment is needed before screening and dosing in ENVISION may resume,' Sarepta added. The company is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for Elevidys.

Sarepta reports outcomes from trial of Duchenne muscular dystrophy therapy

Yahoo

15 hours ago

Health
Yahoo

Sarepta reports outcomes from trial of Duchenne muscular dystrophy therapy

Sarepta Therapeutics has announced new outcomes from its open-label Phase Ib Study 9001-103, also referred to as the ENDEAVOR trial, of adeno-associated virus (AAV)-based gene transfer therapy Elevidys (delandistrogene moxeparvovec-rokl) to treat Duchenne muscular dystrophy (DMD). The multi-cohort ENDEAVOR study is aimed at evaluating the therapy's safety and protein expression in male subjects with DMD. Across seven cohorts, the trial enrolled 55 subjects and dosed participants who were aged between four and seven years old at the time of treatment, with varying degrees of mobility, and those younger than four years old. The primary endpoint of the trial is the change from baseline in Elevidys micro-dystrophin protein expression quantity at 12 weeks, as per western blot measurement, with secondary outcomes measuring dystrophin-positive fibres. In addition, the trial's exploratory endpoints evaluate the change in vector genome copies per nucleus, the North Star Ambulatory Assessment (NSAA), and timed functional tests. Inclusive of the first 12-week duration, subjects will undergo a follow-up period of a total of five years. In cohort 6, subjects aged two years old at the time of treatment showed a mean expression of 93.87% of normal dystrophin levels when treated with the therapy, with 79.9% dystrophin-positive fibres. These outcomes were observed in biopsies 12 weeks post-treatment. The safety seen in this cohort was found to be in line with the clinical and real-world applications of the therapy. Sarepta noted that the company has shared safety and expression from the trial's cohort 4, in which subjects were aged three at the time of treatment and showed a mean protein level of 99.64% in biopsies taken at the same post-treatment interval. More than 25 subjects below four years of age were treated in Sarepta's clinical trials. Sarepta Therapeutics research and development head and chief scientific officer Louise Rodino-Klapac said: 'The strength of the biomarker results that we are seeing in younger patients is extremely encouraging, and we have a meeting with the US Food and Drug Administration next month to discuss expanding the Elevidys label to include younger patients.' Last December, Sarepta completed enrolment and dosing for the Phase III EMERGENE trial of SRP-9003 (bidridistrogene xeboparvovec), targeting limb-girdle muscular dystrophy Type 2E/R4. "Sarepta reports outcomes from trial of Duchenne muscular dystrophy therapy" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data

Sarepta Reports Second Death of Patient Using Its Gene Therapy

Bloomberg

16 hours ago

Health
Bloomberg

Sarepta Reports Second Death of Patient Using Its Gene Therapy

Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for a rare muscle disorder. The death comes three months after a teenage boy died after getting the one-time treatment, Elevidys. Both cases occurred in patients with Duchenne muscular dystrophy who are unable to walk, the company said.

[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

a day ago

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