Delandistrogene moxeparvovec

iStock, Sylverarts WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II FORWARD-53 trial. Wave plans to accelerated approval of the candidate in 2026. Wave Life Sciences’ Duchenne muscular dystrophy exon-skipper elicited what the company’s CEO called “unprecedented” results, significantly improving functional benefit and reversing the hallmark muscle damage of the disease in 11 boys in a mid-stage trial. “Bottom line, [the] data is impressive,” Truist Securities wrote in an investor note Wednesday, citing an improvement in time-to-rise (TTR), reversal of muscle damage and dystrophin expression. Wave’s shares rose 7% as the markets opened to $10.10, from $9.44 at close on Tuesday. Wave announced 48-week data from the Phase II FORWARD-53 trial of its exon-skipper WVE-N531 on Wednesday. The drug is an oligonucleotide being studied in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. The data, from 11 boys ages 5-11 with DMD—10 ambulatory and one non-ambulatory—showed a 3.8 second difference on the time-to-rise scale versus natural history controls, “the largest effect observed relative to any approved dystrophin restoration therapy at 48 weeks,” according to Wave. Treatment with WVE-N531 also led to a 1.2-point improvement on the North Star Ambulatory Assessment (NSAA), which is a scale used to measure functional motor abilities in ambulant children with Duchenne. Mizuho analysts said this result was not statistically significant, “but a notable trend,” according to a Wednesday note. WVE-N531 also led to a 28.6% reduction in fibrosis, driven by decreases in inflammation and necrosis and “coupled with transition from regenerative to mature muscle,” according to Wave. On an investor call Wednesday, CEO Paul Bolno called the overall data “unprecedented” and emphasized this result, saying, “This is the first time that a significant reversal in muscle fibrosis has been observed,” with an exon-skipping therapy. Dystrophin expression—a key biomarker in DMD, which is caused by a mutation in the gene for the dystrophin protein—stabilized between 24 and 48 weeks, the biotech reported, averaging 7.8%, with 88% of boys achieving greater than 5% average dystrophin. WVE-N531 was safe and well-tolerated, with no serious adverse events, according to Wave. These results come amid a busy March for the DMD space. Last week, several companies—including Dyne Therapeutics, REGENXBIO and Genethon—presented new data for their candidates at the 2025 Clinical & Scientific Conference of the Muscular Dystrophy Association in Dallas, with both Dyne and REGENXBIO on track for regulatory submissions next year. Wave, too, intends to accelerated approval of WVE-N531 in 2026, following FDA feedback, according to Wednesday’s press release. Wave’s results may also be a balm for the DMD community after the revelation last week that a teenage patient taking Sarepta’s approved gene therapy Elevidys had died. The death was linked to acute liver failure, according to Sarepta, which added that a cytomegalovirus (CMV) infection was “a possible contributing factor” to the death. The DMD space has seen significant progress in the past decade, capped by the 2023 approval of Elevidys as the first gene therapy for the disease. However, “Despite progress in Duchenne, there remains a significant unmet need for therapeutics that meaningfully impact disease progression,” Pat Furlong, founder and president of Parent Project Muscular Dystrophy , said in a statement Wednesday. “Paired with monthly administration and a continued favorable safety profile, WVE-N531 represents a significant step forward – not just for individuals amenable to exon 53 skipping, but also for the broader exon skipping field.”

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