SAT-3247 and Restoring Muscle Regeneration in Duchenne Muscular Dystrophy: Phil Lambert, PhD

WATCH TIME: 5 minutes

"We're not putting dystrophin itself back, but we're actually putting back the necessary signaling component for the cell... to respond, we believe, much like it used to when it did have dystrophin."

Duchenne muscular dystrophy (DMD) causes progressive muscle weakness, primarily in boys. Traditionally, corticosteroids like prednisone and deflazacort slow muscle degeneration by reducing inflammation. Along with physical therapy and assistive devices, these treatments manage symptoms and improve quality of life. Recently, genetic therapies such as exon skipping and gene therapy offer new hope for addressing the genetic causes of DMD.

At the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19, in Dallas, Texas, Satellos Bioscience presented new phase 1 first-in-human data on SAT-3247, an investigational adaptor associated kinase (AAK1) targeting treatment for DMD. The study, which involved escalating single and 7-day doses in healthy volunteers, confirmed the safety of SAT-3247, with predictable, dose-dependent drug handling and no accumulation after repeat dosing. In addition, the oral pill demonstrated a promising pharmacokinetic profile that will be further confirmed in a cohort of patients with DMD that is currently still ongoing.

Phil Lambert, PhD, chief scientific officer at Satellos, sat down with NeurologyLive^® at the meeting, discussing the mechanism of SAT-3247, which targets muscle regeneration rather than replacing dystrophin. Lambert explained that DMD may be considered a disease of failed muscle regeneration due to stem cell dysfunction. SAT-3247 works by restoring the signaling pathways in stem cells, enabling them to regenerate muscle tissue more effectively. The drug targets AAK1, and inhibiting it allows stem cells to produce both new stem cells and progenitor cells necessary for muscle repair. After promising preclinical results, the next step is testing SAT-3247 in clinical trials to assess its potential benefits for patients with DMD.

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REFERENCE
1. Dubow J. First-in-human Phase 1 study of orally administered SAT-3247 in healthy volunteers and adult participants with Duchenne Muscular Dystrophy (DMD). Presented at: 2025 MDA Clinical & Scientific conference. March 16-19. Dallas, TX. ABSTRACT O98.