Highlighting Early-Stage Promising Data of AAK1 Inhibitor SAT-3247 in Duchenne Muscular Dystrophy: Phil Lambert, PhD

WATCH TIME: 5 minutes

"This is an orally available small molecule, which really matters for patients with DMD—making treatment simpler and more accessible is a huge advantage."

Treating Duchenne muscular dystrophy (DMD) has been a challenge because of its progressive nature and the limited score of current therapies. While some treatments are effective in slowing progression, they don’t fully address muscle degeneration and regeneration. Emerging research on adaptor-associated kinase 1 (AAK1) inhibitors offers a new approach by targeting cellular pathways like endocytosis and protein trafficking, with the potential to improve muscle function and reduce inflammation.

The 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19, in Dallas, Texas, featured the latest updates for several agents in the neuromuscular pipeline, including SAT-3247, an AAK1-targeting small molecule. Designed by Satellos Bioscience, the therapy is currently being assessed in a phase 1, first-in-human study of healthy volunteers and patients with DMD, focusing on safety and pharmacokinetic (PK) data. Overall, results demonstrated excellent tolerability and predictable, dose-proportional PK with no concerning accumulation.

In an interview with NeurologyLive^®, Phil Lambert, PhD, discussed the early-stage data in detail, summarizing the key findings and how it supports translatability from preclinical models. Lambert, chief scientific officer at Satellos, discussed how the study confirmed that the oral formulation and tablet strengths aligned well with projected efficacious exposures, eliminating the need for reformulation ahead of phase 2. Though early patient data from those with DMD is still being collected, Lambert noted that these results mark a meaningful step in moving this once-daily, patient-friendly agent forward.

Click here for more MDA 2025 coverage.

REFERENCES
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.