SAT-3247, an investigational oral therapy targeting muscle regeneration in Duchenne muscular dystrophy (DMD), has shown promising results in its first-in-human clinical trial, according to a recently published press release.
SAT-3247 is a small molecule which aims to reset the body’s impaired muscle regeneration process. The drug works by targeting AAK1, a protein crucial for muscle stem cell activation in the absence of dystrophin, the protein deficient in DMD.
The ongoing Phase 1a/b study assessed SAT-3247 over 28 days in five men with DMD age 20-27. Results showed that SAT-3247 was well tolerated, with no drug-related side effects of moderate severity or higher.
The authors noted individuals demonstrated a mean 118.6% improvement in dominant hand grip strength and a 97.9% increase in the nondominant hand — an approximate doubling in grip strength.
Additionally, participants experienced a significant increase in the predicted amount of air exhaled after a deep breath, a measure of respiratory function. In comparison, untreated individuals with DMD typically show an annual 5% decline.
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“These early signs of efficacy in adults with more advanced disease are incredibly encouraging and support expanding our clinical program to the broader Duchenne community. SAT-3247 has been designed to be a convenient, oral therapy with the potential to improve outcomes, regardless of an individual’s specific dystrophin mutation,” said Dr. Wildon Farwell, Satello’s chief medical officer.
The five participants have been invited to continue treatment in an 11-month open-label extension study that will also include additional males aged 16-25. This follow-up study aims to evaluate long-term safety and the effect of SAT-3247 on fat fraction in muscle, along with secondary outcomes such as muscle force and function.
Based on these preliminary results, Satellos plans to launch a Phase 2 global, placebo-controlled study in ambulatory children with DMD. The upcoming trial will focus on both safety and functional outcomes. If confirmed in larger studies, SAT-3247 could represent a significant advancement in DMD treatment, offering hope to patients regardless of their dystrophin mutation status.
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