Wildon Farwell, MD, Chief Medical Officer at Satellos, discusses the safety and efficacy of SAT-3247 to treat adults with Duchenne muscular dystrophy (DMD).
DMD is a genetic neuromuscular disorder characterized by progressive muscle wasting. DMD occurs primarily in males, though in rare cases may affect females. The symptoms of DMD include progressive weakness and atrophy of both skeletal and heart muscle. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. DMD is caused by mutations in the DMD gene that codes for dystrophin.
Early Clinical Trial Data
Recent data presented at the 30th Annual Congress of the World Muscle Society shows promising clinical data demonstrating initial efficacy of once-daily oral SAT-3247 in adults with DMD. SAT-3247 is an oral, small molecule drug being developed to regenerate skeletal muscle lost due to DMD, independent of dystrophin and regardless of exon mutation status. A phase 1a/b study looked at effects of SAT-3247 in five patients ages 20 to 27 years of age over a 28-day period.
As Dr. Farwell explains, the data presented demonstrated that SAT-3247 was safe and well-tolerated across the study with a desirable pharmacokinetic profile. Individuals treated with SAT-3247 also demonstrated a 118.6% mean improvement in maximum grip strength in the dominant hand and a 97.9% mean improvement in the non-dominant hand. This represents an approximate doubling of grip strength from around 2 kg to around 4 kg, far greater than seen in published natural history data for this age group.
Additionally, patients exhibited a 5.8% mean improvement of predicted forced vital capacity, also inconsistent with natural history. All other measures remained stable over the study period and no drug-related adverse events of moderate severity or higher were observed and no dose-limiting toxicities occurred.
Next Steps
The five patients who participated in the phase 1b study were invited to enroll in an 11-month open-label follow-up study which is also looking to enroll additional male patients ages 16-25 years. The primary endpoints of this study are to evaluate long-term safety and tolerability, as well as the effect of SAT-3247 on fat fraction in biceps brachii muscle. Secondary endpoints include the effect of SAT-3247 on fat fraction and impact on muscle force and function.
Based on this initial data, there are also plans for a phase 2 randomized, double-blind, placebo-controlled, global, proof-of-concept study of SAT-3247 in ambulatory children with DMD. Primary endpoints will evaluate safety and tolerability of SAT-3247 and effect on muscle force. Secondary endpoints will evaluate SAT-3247’s impact on muscle quality, function, and regeneration.
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To learn more about DMD and other rare musculoskeletal conditions, visit https://checkrare.com/diseases/musculoskeletal-diseases/
