FDA designations boost AI-driven cell therapy approach for Duchenne muscular dystrophy

Boston-based Somite.ai has recently announced that the FDA has granted Orphan Drug Designation and Rare Pediatric Disease Designation for its flagship program, SMT-M01, for treating Duchenne muscular dystrophy. These designations will significantly speed up the development of SMT-M01, a program that uses Somite’s AlphaStem AI platform to develop a cell replacement therapy for Duchenne muscular dystrophy.

"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 Micha Breakstone, Founder and CEO of Somite Therapeutics in a press statement.

"These designations underscore the critical unmet need in Duchenne muscular dystrophy 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 Duchenne muscular dystrophy patients and their families."

While Duchenne muscular dystrophy is the most common hereditary neuromuscular disease​, it is still rare. The FDA grants orphan status to medicines intended to treat, diagnose, or prevent rare diseases impacting fewer than 200.000 people in the U.S. The Rare Pediatric Disease Designation Program is provided for very serious or life-threatening diseases affecting fewer than 200.000 children (0-18 years of age) in the U.S.

Globally, one in 3,500 male children​ are born with Duchenne muscular dystrophy. People affected by the disease experience progressive wasting (atrophy) of the muscles. Most people lose their ability to walk by their teenage years, and very few live past their twenties because of life-threatening conditions like heart muscle disease and breathing difficulties. No current treatment can halt the disease and available treatments are palliative.

Finding a potent treatment for Duchenne muscular dystrophy has been extremely difficult. Only recently, pharma giant Pfizer abandoned its gene therapy​ for the disease after it failed in a Phase III clinical trial.

Somite’s SMT-M01 takes a promising new approach. It is a cell replacement therapy, which aims to replace somite-derived musculoskeletal cells in patients with Duchenne muscular dystrophy. Somites are transient embryonic units that result in the growth of repetitive structures in the body, such as bones, cartilage, brown adipose tissue, and skeletal muscles. Somite.ai says it is currently the only company “proficient in producing somite-derived cells efficiently.”

Speaking of the recently granted Orphan Drug and Rare Pediatric Disease designations, Kristy Brown, Senior VP of Translational Development at Somite, said in a press statement: "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. [They] 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."

The most recent announcement comes a few days after Somite entered into a collaboration​ with the U.S. CDMO OmniaBio to develop and manufacture the SMT-M01 cell therapy for Duchenne muscular dystrophy. This collaboration will aid Somite in preparing the clinical trials for SMT-M01. The company plans to enter Phase I/II in the next two years.