Satellos gets IND clearance for phase 2 SAT-3247 study

Executive Summary

• Satellos Bioscience Inc. has received Investigational New Drug (IND) clearance from the U.S. FDA and approvals from global regulatory agencies (UK, Australia, Serbia) to initiate the Phase 2 pediatric study of SAT-3247 for Duchenne Muscular Dystrophy (DMD).
• The Phase 2 study, named "Basecamp," is a three-month, randomized, double-blind, placebo-controlled, proof-of-concept trial involving 51 ambulatory children with DMD.
• Initial enrollment is expected by the end of 2025, with interim data anticipated in Q2 2026 and adult DMD data (LT-001) in Q1 2026.

Key Details

• Regulatory Approvals:
  • USA: IND clearance by the FDA.
  • United Kingdom: Authorization of Clinical Trial Application (CTA) by the MHRA.
  • Australia: Acceptance of Therapeutic Goods Administration (TGA) clinical trial notification (CTN) scheme.
  • Serbia: Approval of CTA by the Medicines and Medical Devices Agency (ALIMS).
  • Pending: CTA under review in the European Union and Canada.
• Study Design (Basecamp):
  • Phase: Phase 2.
  • Duration: Three months.
  • Population: 51 ambulatory children with Duchenne Muscular Dystrophy (DMD).
  • Design: Randomized, double-blind, placebo-controlled, proof-of-concept.
  • Primary Endpoints: Safety, tolerability, and effect on muscle force.
  • Secondary Endpoints: Impact on muscle quality, function, and regeneration.
• Timeline:
  • First participant enrollment: End of 2025.
  • Initial interim data from Basecamp: Q2 2026.
  • Data from LT-001 study (adults with DMD): Q1 2026.
• Drug Profile (SAT-3247):
  • Mechanism: Oral small-molecule therapy targeting AAK1 to restore muscle repair and regeneration signals normally provided by dystrophin.
  • Previous Data (Phase 1a/b in Adults):
    • Safe and well-tolerated with desirable pharmacokinetic profile.
    • Part B results (28-day treatment) showed a 118.6% mean improvement in maximum grip strength in the dominant hand.
    • Part B results showed a 97.9% mean improvement in the non-dominant hand.
    • Grip strength increased from approximately 2 kg to approximately 4 kg (approximate doubling).

Notable Quotes

• "We are delighted to achieve U.S. and global clearance to start our phase 2 pediatric study of SAT-3247, which we have named Basecamp. This important milestone positions Satellos to demonstrate the potential for SAT-3247 to safely restore the body's ability to repair and regenerate muscle in children living with Duchenne, and to alter disease progression," said Frank Gleeson, co-founder and chief executive officer of Satellos.
• "With encouraging functional data from our phase 1b study in adults, we have confidence in the potential for SAT-3247 to make an impact for children with Duchenne, and we are excited to begin enrolment in the trial imminently."