Atamyo Therapeutics SAS

EVRY, France--( BUSINESS WIRE )-- Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and cardiomyopathies, today announced that the U.S. Food & Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for ATA-200 to proceed in a Phase 1b/2b clinical trial. ATA-200 is a one-time gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5), a serious and debilitating condition that affects children and leads to loss of ambulation before adulthood. The multicenter Phase1b open-label dose escalation study ( NCT05973630 ) will evaluate safety, pharmacodynamics, efficacy, and immunogenicity in children receiving intravenous ATA-200, a single-dose Adeno-Associated Virus (AAV) vector carrying the human γ-sarcoglycan transgene. The deployment in the US of Atamyo’s clinical trial of ATA-200 is funded by The Dion Foundation for Children with Rare Diseases . This study has already received regulatory clearance in France and Italy. “This IND clearance in an important step to bring ATA-200 to US children suffering from this highly debilitating LGMD-2C/R5 disease,” said Sophie Olivier, MD, Chief Medical Officer of Atamyo. “We are proud to be the first treatment for LGMD-2C/R5 to enter into clinical development in the US and we are committed to opening the first US center before year-end,” added Stéphane Degove, Atamyo’s Chief Executive Officer. In addition, Atamyo announced the US FDA has awarded Orphan Drug Designation for ATA-200. Orphan Drug Designations by the FDA grants seven years of market exclusivity in the US. This Orphan Drug Designation for ATA-200 was granted a few weeks after ATA-200 received Rare Pediatric Disease Designation by the FDA. LGMD-2C/R5 is a rare genetic disease caused by mutations in the gene that produces γ-sarcoglycan, a transmembrane protein that is involved in the connection between muscle fibers and their environment. It affects an estimated 2,000 people in Europe and in the US. In the classical form, symptoms appear in early childhood and patients suffer from progressive muscular weakness leading to loss of ambulation before adulthood. Cardiac involvement, typically presenting as dilated cardiomyopathy, is reported in approximately half of patients and will eventually impact life expectancy. There is no curative treatment. The management of LGMD-2C/R5 is solely supportive. ATA-200, the gene therapy for LGMD-2C/R5, delivers a normal copy of the gene for production of γ-sarcoglycan. In preclinical mice models, ATA-200 demonstrated its tolerability and capability to correct symptoms and biomarkers of the pathology. Atamyo plans to initiate enrolling patients for this clinical trial in the 4 th quarter of 2024. The therapy is based on the research of Atamyo Chief Scientific Officer Isabelle Richard, Ph.D., Research Director at CNRS who heads the Progressive Muscular Dystrophies Laboratory at Genethon. About Atamyo Therapeutics Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD), with two clinical-stage programs targeting respectively LGMD-R9 and LGMD-R5. The name of the company is derived from two words: Celtic Atao which means “Always” or “Forever” and Myo which is the Greek root for muscle. Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments. For more information visit www.atamyo.com

EVRY, France--( BUSINESS WIRE )-- Atamyo Therapeutics , a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular disease, today announced its participation in six upcoming conferences in the US and Europe to highlight recent developments in its programs targeting limb-girdle muscular dystrophies (LGMDs). Atamyo CEO Stephan Degove will provide a corporate overview at Sach’s 20 th Annual Biotech in Europe Forum, Chardan's 8th Annual Genetic Medicines Conference and the Cell & Gene Meeting on the Mesa 2024. • Sach’s 20th Annual Biotech in Europe Forum, held in Basel, Switzerland, on September 25-26, 2024: Presentation on September 25, at 11:40 am (CEST), in the Ruby Room. • Chardan's 8th Annual Genetic Medicines Conference, held in New York City, on September 30 and October 1, 2024: Presentation on September 30 at 11:00 am (US EDT), in the Embassy Room. • Cell & Gene Meeting on the Mesa, held in Phoenix, AZ, on October 7-9, 2024: Presentation on October 8, at 5:45 pm (US MST), at the FLW Ballroom F. Two communications at the 29th Annual Congress of the World Muscle Society, October 8-12, 2024, in the Prague Congress Centre, Prague, Czech Republic, will describe Atamyo’s LGMD programs. • “Preliminary results from a Phase 1-2 gene therapy study of ATA-100, AAV9 vector encoding FKRP, in patients with Limb Girdle Muscular Dystrophy R9.” Oral presentation by S. Olivier (Atamyo), at Congress Hall on Saturday, October 12, 2024, 8 am - 8:15 am (CEST) • “MRI characterization of the cardiac involvement in LGMD2i/R9.” JY Hogrel (Institute of Myology, Paris) et al., Poster Board Number 87P at Forum Hall, on Wednesday, October 9, 2024, 5:15 pm - 6:15 pm (CEST). Two communications at the European Society of Gene & Cell Therapy 31 st Annual Congress, October 22-25, 2024 in La Nuvola, Rome, Italy, will describe Atamyo’s LGMD programs. Both posters will be presented during Poster Session II on Wednesday 23 October, 1:30 pm to 3 pm (CEST). • “Development of a quantitative alpha-dystroglycan glycosylation test in patients with Limb Girdle Muscular Dystrophy R9 treated in ATA-001-FKRP open-label multicenter AAV trial.” E Thevenot (Genethon) et al., Poster number P0088 • “Natural history of limb girdle muscular dystrophy R9: two-year follow-up of a European cohort.” S Olivier (Atamyo) et al., Poster number P0264 Two communications on Atamyo’s LGMD-R9 program will be presented at the ASGCT’s Breakthroughs in Muscular Dystrophy Conference, November 19-20, 2024, in Chicago, IL. • “Development of Gene Therapy for LGMD-R9.” Oral presentation by E. Gicquel (Genethon), on November 19, 2024, 5:15 p.m. - 5:30 p.m. (US CDT), Great Lakes East Ballroom at the Westin Michigan Avenue Hotel. • “Overcoming Phenotypic Variability in Dystrophic Models: A Machine Learning Method for Refined Gene Therapy Evaluation.” E. Brureau (Genethon) et al. Poster P27. Session on November 16, 2024, 8 a.m. - 7 p.m. (US CDT), Great Lakes East Ballroom at the Westin Michigan Avenue Hotel Publication in Nature Communications of a Next-Generation Gene Therapy Vector for Muscle Diseases, Using AI Predictive Methodology to Improve Efficacy and Safety. • “An engineered AAV targeting integrin alphaV beta 6 presents improved myotropism across species,” by Ai Vu Hong (Genethon), et al., was published in Nature Communications ( https://doi.org/10.1038/s41467-024-52002-4 ). The paper describes development of the next-generation LICA-1 capsid by Isabelle Richard, Ph.D., Atamyo’s Chief Scientific Officer, and her Progressive Muscular Dystrophies Team at Genethon. Atamyo has rights to the LICA-1 capsid for cardiomyopathies indications. About Atamyo Therapeutics Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD), with two clinical-stage programs targeting respectively LGMD2I/R9 and LGMD2C/R5. The name of the company is derived from two words: Celtic Atao which means “Always” or “Forever” and Myo which is the Greek root for muscle. Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments. For more information visit www.atamyo.com

PARIS--(BUSINESS WIRE)-- Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), provided updates in its latest Newsletter on results of its gene therapy clinical trials for Duchenne Muscular Dystrophy and advances in gene therapies for limb-girdle muscular dystrophies by its spinoff Atamyo Therapeutics. Also in an interview, Roger Hajjar, M.D., Director of the Gene and Cell Therapy Institute of Mass General Brigham in Boston, renowned gene therapy researcher and entrepreneur, offered insights on what attracted him to join Atamyo Therapeutics as the newest board member of the Genethon spinout company. “What resonated with me was the fact that Atamyo is a research endeavor by investigators within Genethon whose work is being translated into the clinic,” Dr. Hajjar said. “It is a very nice demonstration of a bench to bedside approach. It’s definitely a new model.” Genethon CEO Frederic Revah, Ph.D., commenting on gene therapy trials for Duchenne Muscular Dystrophy, observed, “The effective dose in this first phase of the trial is lower than that used in other gene therapy programs using microdystrophin. Based on the recommendation of the independent expert committee (Data Monitoring Committee), we are now preparing the pivotal part of our clinical trial in the European Union.” Dr. Revah’s commentary and Dr. Hajjar’s interview are part of Genethon’s current Newsletter highlighting the organization’s product development, research collaborations and scientific journal publications. Read the full Newsletter. About Genethon As a pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a non-profit laboratory that was established by AFM-Telethon. A first gene therapy for spinal muscular atrophy to which Genethon contributed has obtained a product license. With more than 200 scientists and professional staff, Genethon is pursuing its aim to develop therapies which change the lives of patients suffering from rare genetic diseases. Thirteen products stemming from Genethon’s R&D or from collaborations are in clinical trial for diseases of the liver, blood, immune system, muscles and eyes. Seven other products could enter clinical trials over the next five years. More information at . View source version on businesswire.com: Contacts Stéphanie Bardon 06.45.15.95.87 communication@genethon.fr Source: Genethon View this news release online at: