Somite Therapeutics Announces FDA Orphan Drug and Rare Pediatric Disease Designations for SMT-M01 in Duchenne Muscular Dystrophy

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

BOSTON, Sept. 16, 2024 /PRNewswire/ -- Somite Therapeutics, a fully integrated TechBio company leveraging big data and AI to introduce 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) for the company's lead program, SMT-M01, for the treatment of Duchenne muscular dystrophy (DMD).

"Receiving both Orphan Drug and Rare Pediatric Disease Designations for 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 designations underscore the critical unmet need in DMD and the potential of our AI-driven approach to develop innovative cell therapies. We are committed to advancing SMT-M01 through clinical development as rapidly as possible to make a meaningful difference for DMD patients and their families."

The FDA's Orphan Drug Designation program provides orphan status to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. Rare Pediatric Disease Designation and Priority Review Voucher Program are granted for serious or life-threatening diseases that primarily affect individuals aged from birth to 18 years and impact fewer than 200,000 people in the U.S. Once approved, the RPD designation also makes the product eligible for a priority review voucher. This voucher can be used for a subsequent marketing application for a different product or sold to another sponsor to expedite their marketing application review.

"These FDA designations validate the innovative nature of our SMT-M01 program and its potential to address the significant unmet medical need in Duchenne muscular dystrophy," commented Dr. Kristy Brown, SVP Translational Development at Somite Therapeutics. "The designations will provide important benefits as we advance SMT-M01 through clinical development, including tax credits for qualified clinical trials, exemption from user fees, and eligibility for seven years of market exclusivity upon regulatory approval."

Somite 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 for patients with this devastating disease.

About Somite

Somite.ai is a venture-backed company aiming to become the OpenAI of stem cell biology, developing AI foundation models to produce human tissue for cell therapies at scale for diseases such as diabetes, obesity, and muscular dystrophies. Somite's AI platform, AlphaStem, fuels a virtuous cycle: It enables new cell therapies, generating massive data that further improve the platform, empowering even faster therapy creation 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. Incorporated in Oct. 2023, Somite.ai has raised over $10m to date.

Somite Management Team:

• Micha Breakstone, PhD: CEO and Co-founder - Repeat AI entrepreneur (Chorus.ai acq. for $575m)
• Jonathan Rosenfeld, PhD: CTO and Co-founder - Head of the Fundamental AI group at MIT FutureTech
• Carl Morris, PhD: Chief Scientific Officer
• Kristy Brown, PhD: SVP Translational Development
• Shai Kivity, VP Strategy & Operations

Scientific Co-founders:

• Olivier Pourquie, PhD: Professor of Genetics and Pathology, Harvard Medical School and Brigham and Women's Hospital
• Allon Klein, PhD: Professor of Systems Biology at Harvard Medical School
• Cliff Tabin, PhD: Chair of the Department of Genetics at Harvard Medical School
• Jay Shendure, PhD: HHMI Professor of Genome Sciences at University of Washington

Media Contact: [email protected]i 
Website: www.somite.ai

SOURCE Somite Therapeutics