Delandistrogene moxeparvovec

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of individuals with Types 2 or 3 spinal muscular atrophy (SMA) Late-breaking oral on Elevidys’ Embark two-year data and pooled analysis of Study 101, 102 and Endeavor, demonstrated clinically meaningful and statistically significant improvements across key measures of motor function in boys with Duchenne muscular dystrophy (DMD) Muscle pathology as measured by MRI continued to generally favour Elevidys across muscle groups in boys with Duchenne up to two years 17 March 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that it will present new data at the Muscular Dystrophy Association (MDA) conference, 16-19 March, 2025, in Dallas, Texas, from its neuromuscular portfolio, including 12 oral and poster presentations. These data demonstrate the breadth of Roche’s neuromuscular portfolio across spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). “These longer-term results are encouraging for people living with SMA and Duchenne, as they demonstrate sustained improvements or stabilisation in mobility and independence for those receiving our disease-modifying treatments,” said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development. “The positive SUNFISH five-year results encompass one of the broadest SMA populations to be studied to date. We are also very encouraged by the data being shared across our Elevidys studies, affirming the potential of the first gene therapy for the treatment of Duchenne to stabilise or slow disease progression.” Five-year exploratory data from the pivotal SUNFISH study of Evrysdi in people with Types 2 or 3 SMA After up to five years of treatment in the SUNFISH trial (NCT02908685, n=231), SMA patients on Evrysdi demonstrated overall long-term stabilisation of motor function improvements from baseline that were observed during the first year, as measured by Motor Function Measure 32 (MFM-32). This marks the final readout from SUNFISH, which has reinforced the efficacy, safety and longer-term impact of Evrysdi in a broad population of people with Types 2 and 3 SMA, aged 2-25 years at enrollment. Untreated natural history data presented together with the SUNFISH five-year data show that without treatment, patients from a similar population can experience a significant decline in motor function over the same five-year period. Patients (>12 years of age) and caregivers both reported continuous improvement or stabilization in levels of independence for performing daily activities, such as dressing, picking up objects and washing, as measured by the SMA Independence Scale (SMAIS-ULM). Additionally, mean treatment adherence with Evrysdi over the multi-year period was over 99%. The overall rates of adverse events (AEs) and serious adverse events (SAEs) were reflective of the underlying disease and were consistent with previous data. No treatment-related AEs led to withdrawal from the study. Muscle health and longer-term functional outcomes from individuals with Duchenne’s disease after treatment with Elevidys, vs. well-matched external control groups Elevidys showed statistically significant and clinically meaningful improvements across key measures of motor function two years after treatment in EMBARK (Phase III, NCT05096221) and three years after treatment in a pooled analysis of Study 101 ( Phase I/II, NCT03375164, n=4), Study 102 (Phase II, NCT03769116, n=26), and ENDEAVOR Cohort 1 (Phase Ib, NCT04626674, n=20), compared to well-matched external control groups. Collectively, these data demonstrate that treatment with Elevidys results in long-term stabilisation or slowing of disease progression in individuals aged four through eight years of age at the time of treatment. Topline data from year two of the EMBARK trial were announced in January 2025. No new safety signals were observed in the EMBARK study over the two-year duration. To further evaluate the effect of Elevidys on disease progression, muscle health and changes in muscle pathology were assessed by magnetic resonance imaging (MRI) in a subset of individuals in EMBARK part one. At week 52, results showed stabilisation or slowing of disease progression in Elevidys-treated patients compared to placebo-treated patients. At week 104, MRI changes from baseline continued to generally favour Elevidys versus placebo at week 52 across muscles and muscle groups. Duchenne is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Approximately one in 5,000 boys worldwide are born with Duchenne, while Duchenne in girls is very rare. Everyone who has Duchenne will lose the ability to walk, upper limb, lung and cardiac function and mean life expectancy is 28 years. A diagnosis of Duchenne will require full-time caregiving which is most often provided by parents, the majority of whom will find it difficult to carry out usual work or household activities and suffer from depression and physical pain. Duchenne is caused by mutations of the DMD gene, which affects the production of the muscle protein, dystrophin. Dystrophin is a critical component of a protein complex that strengthens muscle fibers and protects them from injury during muscle contraction. Due to a genetic mutation in the DMD gene, people with Duchenne do not make functional dystrophin; their muscle cells are more sensitive to injury and muscle tissue is progressively replaced with scar tissue and fat. As dystrophin is also deficient in vital organ systems such as the cardiovascular and respiratory systems, the effect is thus inevitably fatal, with an average survival limited to the third decade of life. SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation or deletion of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of functional SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Currently approved drugs, including Evrysdi, prevent degeneration or death of these cells, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost. Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form either by feeding tube or by mouth. In the United States a room-temperature stable Evrysdi 5 mg tablet formulation is also now available. Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the CNS and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core motor functions such as swallowing, speaking and breathing. Evrysdi is currently approved in more than 100 countries, with more than 18,000 people with SMA treated globally. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Elevidys is the first approved disease-modifying gene therapy for Duchenne and is designed to address the underlying cause of Duchenne through targeted skeletal, respiratory and cardiac muscle expression of shortened dystrophin produced by Elevidys. Elevidys is a one-time treatment administered through a single intravenous dose. Elevidys is contraindicated in individuals with any deletion in exons 8 and/or 9 in the DMD gene. Elevidys is approved for people living with Duchenne aged four years old and over regardless of their ambulatory status in the US, United Arab Emirates (UAE), Qatar, Kuwait, Bahrain and Oman. Elevidys is also approved for the treatment of ambulatory individuals aged four through seven years in Brazil and Israel. Filings have also been submitted to the European Medicines Agency (EMA) and regulatory authorities in Japan, Switzerland, Singapore, Hong Kong and Saudi Arabia. To date, more than 600 patients have been treated with Elevidys. 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, 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. 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. The content above comes from the network. if any infringement, please contact us to modify.

LUND, Sweden, March 21, 2025 /PRNewswire/ -- Hansa Biopharma AB, "Hansa" or the "Company" (NASDAQ Stockholm: HNSA), today published its Annual and Sustainability Reports for 2024. Peter Nicklin, Chair of the Board, Hansa Biopharma, said: "Hansa delivered several important milestones in 2024 and remains on track to advance innovative science and important new medicines. This is in large part due to the dedicated, results-driven employees, and the Company's commitment to delivering on its discovery, development and commercialization goals. In my third year as Chair of the Board of Directors, I remain inspired and excited for what is to come for Hansa." Søren Tulstrup, President and CEO, Hansa Biopharma, said, "2024 was a momentum-building year for Hansa. With year-on-year IDEFIRIX® sales growth of 83%, a successful financing round, and several critical pipeline catalysts for imlifidase and HNSA-5487, 2024 has positioned us well to advance our efforts in Autoimmune, Gene Therapy and Transplantation. 2025 promises to be an exciting and important year, marked by several data readouts and a planned BLA submission to the U.S. FDA in kidney transplantation under the accelerated approval pathway. Hansa's science-led, patients-first business model remains at the core of what we do, executed by a team of highly experienced and dedicated individuals. We remain focused on our mission to advance critical science that can address the unmet medical needs of people living with rare, immune-mediated diseases." 2024 highlights In 2024, Hansa advanced the commercialization of IDEFIRIX® (imlifidase) in Europe as a desensitization treatment for kidney transplant patients, achieving record quarterly and full year sales. The ongoing commercialization of IDEFIRIX in Europe is being driven by continued strong market access in a total of 18 markets, including the five key markets of France, Germany, Italy, Spain and the UK. A growing body of data, real-world evidence and clinical guidelines on the utilization of imlifidase as a desensitization strategy for highly sensitized kidney transplant patients has been published. The most recent guidance published in Transplant International provides specific guidelines on appropriate use of imlifidase in clinical practice to enable kidney transplants in highly sensitized patients. Preparation for commercialization in the US is progressing, with complete randomization of the pivotal Phase 3 trial - ConfIdeS - announced in May 2024. Data readout for the trial and submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) are planned in 2H 2025. In October 2024, Hansa announced full results of the NICE-01 first in human trial and 12-month follow up analysis of HNSA-5487, demonstrating that it can robustly and rapidly reduce IgG levels, has redosing potential, and a favorable safety and tolerability profile. Initial clinical development will be focused on neuro-autoimmune disease with a well characterized role of specific autoantibodies in disease pathology and recurring acute phases - specifically, myasthenia gravis (MG). The company plans to align with regulatory authorities in the first half of 2025 on a clinical development path for HNSA-5487 in MG. In Autoimmune, enrolment was completed for the GOOD-IDES-02 Phase 3 trial in anti-glomerular basement membrane (anti-GBM) disease in December, with data readout expected in the second half of 2025. In Guillain-Barré Syndrome (GBS), Hansa announced positive full results from the 15-HMedIdeS-09 single arm Phase 2 study and an indirect treatment comparison to the International Guillain-Barré Syndrome Outcome Study (IGOS) in December 2024. In Gene Therapy, Hansa and Genethon announced initiation of GNT-018-IDES, a Phase 2 trial sponsored by Genethon evaluating the efficacy and safety of a single intravenous administration of Genethon's gene therapy GNT-0003 following pre-treatment with imlifidase in patients with severe Crigler-Najjar syndrome and pre-formed antibodies to AAV8. Enrolment continues in the SRP-9001-104 Phase 1b trial evaluating the use of imlifidase as a pre-treatment to Sarepta Therapeutics Inc.'s (Sarepta) ELEVIDYS (delandistrogene moxeparvovec) gene therapy in Duchenne Muscular Dystrophy (DMD). Hansa's journey towards a more sustainable business continues Over the last three years, Hansa has continuously evolved its approach to Sustainability aligning to the changes happening within the Company and the external environment. This year's Sustainability Report outlines the continued efforts the Company has advanced and our focus for 2025. The report highlights our work to address high unmet medical need in our three core therapy areas - Autoimmune, Gene Therapy and Transplantation, as well as our continued focus on ensuring that we are operating as a transparent, ethical business. Finally, we continue to believe that a diverse, inclusive, and supportive culture is integral to the Company's success. 2024 also marked the first year Hansa conducted a Double Materiality Assessment, an opportunity to better understand the Company's impact on stakeholders and the environment as well as what opportunities influence its business. The output - a list of prioritized material topics - provided insight and perspective on the Company's sustainability strategy and areas of focus for effective risk management. Hansa uses an annual company-wide survey by Great Place to Work®, a global authority on workplace culture, to seek employee feedback with the purpose of constantly developing and refining a healthy work environment with highly engaged and motivated colleagues. In 2024, Hansa was certified as a Great Place to Work® for the fifth consecutive year, with a 95% survey participation, Read the Annual and Sustainability Reports here. This is information that Hansa Biopharma AB (publ) is obliged to make public pursuant to the Securities Markets Act. The information was submitted for publication at 15:00 CET on 21 March 2025. Contacts for more information: Evan Ballantyne, Chief Financial Officer [email protected] Stephanie Kenney, VP Global Corporate Affairs [email protected] About Hansa Biopharma Hansa Biopharma AB is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving and life-altering treatments for patients with rare immunological conditions. The company has a rich and expanding research and development program based on its proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in autoimmune diseases, gene therapy and transplantation. The company's portfolio includes imlifidase, a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients and HNSA-5487, a next-generation IgG cleaving molecule with redosing potential. Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the U.S. The company is listed on Nasdaq Stockholm under the ticker HNSA. Find out more at and follow us on LinkedIn. ©2025 Hansa Biopharma AB. Hansa Biopharma, the beacon logo, IDEFIRIX, and IDEFIRIX flower logo are trademarks of Hansa Biopharma AB, Lund, Sweden. All rights reserved. This information was brought to you by Cision The following files are available for download: SOURCE Hansa Biopharma AB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED