DMD treatment SMT-M01 gets FDA orphan drug, rare disease tags
Cell replacement therapy designed for all patients, regardless of mutation
SMT-M01, a Duchenne muscular dystrophy (DMD) treatment, was granted orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA).
The cell replacement therapy from Somite Therapeutics uses artificial intelligence (AI) and large, complex data sets to improve production of multiple human cell types.
The company plans to begin clinical trials within 18 months, aiming to offer a cell replacement therapy for DMD patients regardless of genetic mutation. “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,” Micha Breakstone, PhD, founder and CEO of Somite, said in a company press release.
The FDA designations represent “a significant milestone for Somite Therapeutics and, more importantly, for patients suffering from Duchenne muscular dystrophy,” Breakstone said. “These designations underscore the critical unmet need in DMD and the potential of our AI-driven approach to develop innovative cell therapies.”
The two designations are designed to encourage the development of therapies for rare diseases. The orphan drug program is intended for potential treatments affecting fewer than 200,000 people in the U.S., and provides incentives such as tax credits and seven years of market exclusivity if a therapy is ultimately approved. Rare pediatric disease designation is granted to treatments for severe or life-threatening rare conditions primarily affecting children. The designation makes the company eligible for a voucher that it can use to expedite the approval process for another treatment. The voucher may also be sold to another company.
Cell therapy as DMD treatment
DMD is characterized by progressive muscle degeneration and weakness. The disease is caused by mutations in DMD, a gene that codes for the production of a protein called dystrophin that serves as a shock-absorber in muscle.
Cell replacement therapy is designed to replace missing, damaged, or diseased cells. Somite takes its name from temporary embryonic structures called somites, which are the origin of muscle and skeletal tissues.
In the context of DMD, the company intends to provide a source of somite-derived satellite cells, which are precursors to skeletal muscle cells and are responsible for skeletal muscle’s robust regenerative capacity. However, they are difficult to isolate from muscle biopsies, as they are relatively rare compared to other cell types. In addition, growing them in number in the lab makes satellite cells lose their regenerative potential.
Somite’s drug development process works by creating a computational twin of the embryo so that it mimics the embryo’s development and behavior to help with decision-making. According to the company, AI enables the rapid identification of innovative protocols to generate new cell types, discover regulators of cell differentiation, and optimize protocols. SMT-M01 uses the company’s Alphastem AI platform.
Kristy Brown, PhD, senior vice president of translational development at Somite, said the 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.”
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