Damite Therapeutics Announces FDA Approval of SMT-M01 for the Treatment of Duchenne Muscular Dystrophy as an Orphan Drug and Rare Pediatric Disease

[[To comply with academic institution guidelines, the founders’ academic affiliations and roles are listed only at the end of the statement.]]

BOSTON, 16 September 2024 /PRNewswire/ — somite therapeuticsa fully integrated TechBio company leveraging big data and AI to deliver novel cell replacement therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to the Company’s lead program, SMT-M01, for the treatment of Duchenne muscular dystrophy (DMD).

“The orphan drug and rare pediatric disease designation of SMT-M01 is a significant milestone for somite Therapeutics and, more importantly, for patients suffering from Duchenne muscular dystrophy,” said Dr. Micha Breakstone, Founder and CEO of somite Therapeutics. “These approvals underscore the urgent unmet need in DMD and the potential of our AI-based approach to developing innovative cell therapies. We are committed to advancing SMT-M01 through clinical development as quickly as possible to make a meaningful difference for DMD patients and their families.”

The FDA’s Orphan Drug Designation program grants orphan drug status to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases affecting fewer than 200,000 people in the United States. The Rare Pediatric Disease designation and Priority Review Voucher Program are granted for serious or life-threatening diseases that primarily affect individuals ages birth to 18 years and affect fewer than 200,000 people in the United States. Once approved, the Rare Pediatric Disease designation also entitles the product to a Priority Review Voucher. This voucher can be used for a subsequent regulatory submission for another product or sold to another sponsor for expedited review of their regulatory submission.

“These FDA approvals confirm the innovative nature of our SMT-M01 program and its potential to address the significant unmet medical need in the treatment of Duchenne muscular dystrophy,” commented Dr. Kristin BrownSVP Translational Development at somite Therapeutics. “The designations will provide us with important benefits as we advance SMT-M01 through clinical development, including tax credits for qualified clinical trials, royalty exemptions, and eligibility for seven years of market exclusivity following regulatory approval.”

Damite Therapeutics' SMT-M01 program leverages the company's proprietary AlphaStem AI platform to develop a novel cell replacement therapy for DMD. The company plans to initiate clinical trials for SMT-M01 in the next 18 months with the goal of providing a mutation-agnostic therapeutic to patients with this devastating disease.

About somite

somite.ai is a venture-backed company with the goal of OpenAI of stem cell biologyDevelopment of AI foundational models for engineering human tissue for large-scale cell therapies for diseases such as diabetes, obesity and muscular dystrophies. somites AI platform, AlphaStemdrives a virtuous cycle: it enables new cell therapies and generates massive amounts of data that further improve the platform and enable even faster development of therapies with broader applications. The company's pipeline includes the promising SMT-M01 program for Duchenne muscular dystrophy (DMD) and the SMT-B01 program for metabolic disorders. Somite.ai was founded in October 2023 and has raised over $10 million to date.

Damite Management Team:

Micha Breakstone, PhD: CEO and Co-Founder – Repeat AI Entrepreneur (Chorus.ai acquired for 575 million US dollars)
Jonathan RosenfeldPhD: CTO and Co-Founder – Head of the Fundamental AI group at MIT FutureTech
Carl MorrisPhD: Scientific Director
Kristin BrownPhD: SVP Translational Development
Shai Kivity, Vice President of Strategy and Operations

Scientific co-founders:

Olivier Pourquie, PhD: Professor of Genetics and Pathology, Harvard University Medical School and Brigham and Women's Hospital
Allon KleinPhD: Professor of Systems Biology at Harvard University Medical School
Cliff TabinPhD: Head of the Department of Genetics at Harvard University Medical School
Jay ShendurePhD: HHMI Professor of Genome Sciences at the University of Washington

Media contact: [email protected]I
Website: www.somite.ai

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