Atamyo Therapeutics

Atamyo Therapeutics announces 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 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 Stephane 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. 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. For more details: read the complete press release: https://lnkd.in/gdCYY-Aw #genetherapy #LGMD #Musculardystrophy #raredisease #orphandrug

Day 3 at ESGCT 2024 in Roma, sharing with the community Atamyo Therapeutics progress on our LGMD-R9 program. 2 posters were presented on October 23 highlighting the results obtained in collaboration with Genethon: ·      “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., ·      “Natural history of limb girdle muscular dystrophy R9: two-year follow-up of a European cohort.” S Olivier (Atamyo) et al.,

Sophie Olivier, MD, Chief Medical Officer of Atamyo Therapeutics, presented preliminary results from a Phase 1-2 gene therapy study of ATA-100, AAV9 vector encoding FKRP in patients with Limb Girdle Muscular Dystrophy R9/2i at the recent World Muscle Society Congress 2024 in Prague. Highlights of the results include: - Global functional improvement in first cohort patients as compared to natural evolution - ATA-100 biodistribution in first 9E12 vg/kg cohort in line with biodistribution associated with efficacy in preclinical models - Improvement of αDG glycosylation observed in patients’ biopsies in first 9E12 vg/kg cohort - Confirmed satisfactory safety profile Atamyo is the first spin-off of Genethon in Gene Therapy. A pioneer in the development of gene therapies, Genethon has been working to find treatments for LGMDs for over 25 years. Researchers of the Progressive Muscular Dystrophies Laboratory at Genethon, under the leadership of Pr. Isabelle Richard, identified both the genes and the mechanisms responsible for many of these diseases and are using their skills and expertise in gene therapy and LGMDs to develop a new generation of effective and safe gene therapies for neuromuscular diseases. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD). To learn more about Atamyo visit our website: atamyo.com #lgmd #musculardystrophy #genetherapy Stephane Degove

In recognition of LGMD Awareness Day today, Atamyo Therapeutics announces the enrollment of the last patient in the dose-escalation phase of its Phase 1b clinical trial of ATA-100, a gene therapy for patients with limb-girdle muscular dystrophy Type 2I/R9 (LGMD-2I/R9, protocol code ATA-001-FKRP).   “The first patients dosed with ATA-100 have experienced promising functional results and the gene therapy has been overall well tolerated in all treated patients with no unexpected safety signal,” said Atamyo CEO and Co-Founder Stephane Degove. “Updated results of the clinical trial will be featured in an oral presentation at the 29th International Annual Congress of the World Muscle Society (WMS) in Prague, October 8 - 12, 2024.” Atamyo is also announcing the filing of an Investigational New Drug (IND) application with the US FDA for a Phase 1b/2b clinical trial of its gene therapy, ATA-200, for patients with LGMD-2C/R5. The deployment in the US of Atamyo’s clinical trial of ATA-200 (NCT05973630) is funded by The Dion Foundation for Children with Rare Diseases. In addition, Atamyo announced the US FDA has awarded Rare Pediatric Disease Designation for ATA-200. The designation was requested based on the potential for ATA-200 to address an unmet medical need for LGMD-2C/R5, a serious and debilitating condition that affects children and leads to loss of ambulation before adulthood. “We are thrilled to have completed the recruitment of the dose escalation phase of our Phase 1b study of ATA-100. Data of the dose escalation will serve at selecting the dose to carry over in the pivotal phase of the study,” said Sophie Olivierr, MD, Chief Medical Officer of Atamyo. “We look forward to presenting the preliminary results of the first dose cohort at the forthcoming WMS Congress and at American Society of Gene & Cell Therapy’s Breakthroughs in Muscular Dystrophy Conference Nov. 19 – 20, 2024 in Chicago.” For more details read the complete press release: https://lnkd.in/gyWeQYCz #LGMD #MUSCULARDYSTROPHY #GENETHERAPY

Atamyo Therapeutics plans to participate in 6 upcoming conferences in the US and Europe to highlight recent developments in our programs targeting limb-girdle muscular dystrophies (LGMDs). Highlights include: - CEO Stephane Degove will provide corporate overviews at Sachs’ 20th Annual Biotech in Europe Forum in Basel, Chardan’s 8th Annual Genetic Medicines Conference in New York City, and the Cell & Gene Meeting on the Mesa in Phoenix. - The company also will make 6 presentations on its limb-girdle muscular dystrophy (LGMD) clinical development and research programs at 3 scientific conferences in the US and Europe. - A publication in Nature Communications describes the work of CSO Isabelle Richard 's team on the use of artificial intelligence (AI) to design a new generation of capsids for more effective gene therapy vectors For details of dates and times of presentations, see the complete Press Release: https://lnkd.in/g3uyg728 Sophie Olivier #meetingonthemesa #lgmd #musculardystrophy European Society of Gene and Cell Therapy. World Muscle Society American Society of Gene & Cell Therapy #genetherapy Genethon

Professor Isabelle Richard, Chief Scientific Officer for Atamyo Therapeutics has received the Fifth Annual LGMD2i Pioneer Award from CURE LGMD2I FOUNDATION and the Speak Foundation for her scientific leadership in LGMD 2i/R9.  The award was announced today in The Speak Foundation’s LGMD News Magazine. Shown here, left, is Professor Richard with Kathryn Bryant Knudson, Founder and President of the Speak Foundation. This recognition is for Isabelle Richard’s groundbreaking research on LGMD and comes after an earlier award by the Jain Foundation Inc for Leadership in Collaboration presented at the 2024 Dysferlin Conference. Isabelle Richard, Ph.D., is Research Director at National Center of Scientific Research (CNRS) in France and head of the Progressive Muscular Dystrophies Laboratory at Genethon. We are proud to have Professor Richard as part of our team at Atamyo.   Atamyo Therapeutics is grateful to the Speak Foundation and LGMD News Magazine for this wonderful article detailing the recent visit made by Kathryn Bryant Knudson, founder of the Speak Foundation to visit the labs and offices of Genethon and Atamyo in Evry, France. We express special thanks to Melanie Bordes, a patient advocate who leads AFMs LGMD Patient Interest Group. Read the article here: https://lnkd.in/gR8RNVEf #lgmd #musculardystrophy #genetherapy Kelly Brazzo

Atamyo Therapeutics and The Dion Foundation for Children with Rare Diseases today announced a key partnership to expand into the US a first-in-human clinical trial of ATA-200, Atamyo’s gene therapy for the treatment of the g-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5).  Highlights: - The Dion Foundation for Children with Rare Diseases will finance expansion in the US of Atamyo’s first-in-human trial of ATA-200. - The Phase 1b, dose-escalation study to evaluate the safety and efficacy of ATA-200 in children has already received regulatory clearance in Europe. - ATA-200 is a single-injection gene therapy aimed to treat limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5) caused by mutations in the g-sarcoglycan gene France-based Atamyo is a clinical stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and cardiomyopathies. The US-based Dion Foundation is a non-profit organization devoted to increasing awareness and allocating funds towards research and development of potential treatments of rare genetic diseases, such as limb-girdle muscular dystrophy. The Dion Foundation will finance deployment in the US of Atamyo’s clinical trial of ATA-200 (NCT05973630). The study, sponsored by Atamyo, has already received regulatory clearance in France and Italy. This multicenter, Phase 1b, open-label, dose escalation study will evaluate safety, pharmacodynamics, efficacy, and immunogenicity in children of intravenous ATA-200, a single-dose Adeno-Associated Virus (AAV) vector carrying the human γ-sarcoglycan transgene. “We are so grateful for the opportunity to establish a partnership with Atamyo to help facilitate bringing their groundbreaking research to a clinical site in the US for the very first clinical trial for children with LGMD2C/R5. This is a monumental step for the entire LGMD community,” said Courtney Dion, Co-Founder and President of the Dion Foundation. “We are thrilled by this key partnership and grateful to the Dion Foundation for their financial support which aims to Stephane Degove, CEO of Atamyo. “We are already engaged in the preparation of an IND filing in the US for ATA-200.” Atamyo is a spin-off of Genethon, a non-profit research organization that is a pioneer in developing gene therapies for rare diseases. ATA-200 is based on the research of Atamyo CSO Isabelle Richard, Ph.D., Research Director at CNRS in France and head of the Progressive Muscular Dystrophies Laboratory at Genethon. Genethon CEO Frederic Revah observed, “This partnership between the Dion Foundation and Atamyo represents another major milestone in our efforts over the past 30 years to develop gene therapies for rare diseases and bring them to patients worldwide.” For more about Atamyo Therapeutics: Atamyo.com For more about The Dion Foundation: TheDionFund.Org #MUSCULARDYSTROPHY #LGMD #GENETHERAPY

(another) great initiative by FDA to improve the development of treatments for rare diseases

Atamyo Therapeutics is thrilled to announce the financing of its ATA-100 gene therapy by the European Innovation Council (EIC). The EIC Accelerator blended grant+equity financing will support ATA-100 clinical development up to registration in Europe and US. We are also proud to be part of the <7% innovations selected by the EIC, this represents another recognition of the life-changing potential of ATA-100 for LGMD-R9 patients.

Atamyo Therapeutics has announced the US Food and Drug Administration (FDA) has awarded Fast Track Designation for ATA-100, an AAV-based gene therapy for the treatment LGMDR9 in Phase 1/2 clinical trials in the US and Europe. In the clinical trial of ATA-100 for the LGMDR9, the company said enrollment progress is on track and the first 2 patients in the second high-dose cohort have been treated. Updated results of the clinical trials will be presented at the 29th International Annual Congress of the World Muscle Society (WMS) in Prague (8-12 October 2024). The designation was requested based on the potential for ATA-100 to address an unmet medical need for LGMDR9, a serious and debilitating condition that affects young adults and leads to loss of ambulation within 10 to 15 years. Preliminary data from the ongoing European Phase 1b/2b study were submitted in support of the application. The clinical trials (EudraCT 2021-004276-33, NCT105224505) are multicenter Phase 1/2 studies evaluating safety, pharmacodynamics, efficacy, and immunogenicity of intravenous ATA-100, a single-dose Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene. The studies will consist of two phases: an open-label dose escalation phase (Stage 1) and a double-blind placebo controlled, randomized phase (Stage 2). Enrollment of the first low-dose cohort of the study in Europe (protocol code ATA-001-FKRP) is completed with promising initial functional results and two patients have already been treated in the high-dose cohort. ATA-100 has been overall well tolerated to date in all treated patients with no unexpected safety signal. Updated results of the clinical trials will be presented through an oral presentation at the 29th International Annual Congress of the World Muscle Society (WMS) in Prague, October 8 to 12, 2024. “This new Fast Track Designation in the US and the good clinical progress of our LGMD-R9 program confirms its life-changing potential for patients affected by LGMD-R9,” said Stephane Degove, Chief Executive Officer and Co-Founder of Atamyo Therapeutics. “We look forward to sharing ATA-100 updated clinical trial results with the community in October,” he added. To read the complete press release: https://lnkd.in/guNrXECN For more information about Atamyo visit our website: atamyo.com #genetherapy #LGMD #musculardystrophy