Patients
Satellos is developing forazapadin (SAT-3247), a first-in-class oral treatment designed to restore muscle regeneration for people living with Duchenne muscular dystrophy. Our clinical program is advancing with urgency, care, and an unwavering commitment to safety.
At Satellos, our mission is to develop new medicines that restore muscle regeneration for people living with muscle diseases. Our potential treatment, forazapadin (SAT-3247), is focused on Duchenne muscular dystrophy — a condition where the body’s natural ability to repair muscle is severely impaired. We’re advancing our science with urgency and care, always focused on delivering meaningful results for the individuals and families affected by Duchenne.
Satellos is currently conducting two Phase 2 clinical trials evaluating forazapadin in people living with Duchenne muscular dystrophy.
Forazapadin (SAT-3247) is an oral treatment we’re developing to help improve how muscle repairs itself in people with Duchenne. The loss of dystrophin disrupts a key signal that guides muscle stem cells to respond to damage. Forazapadin is designed to restore that signal by targeting a protein called AAK1. By doing so, it is designed to help muscle stem cells divide and function more effectively — supporting the body’s natural ability to repair and rebuild muscle. This approach has the potential to benefit many people living with Duchenne, no matter their specific genetic mutation or severity of disease.
SAT-3247-CL-201: Phase 2 BASECAMP Study
Summary
This is a global Phase 2a trial of forazapadin (SAT-3247) in ambulatory DMD patients aged ≥ 7 and < 10 years. The trial will study two doses of forazapadin in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy.
Participants
51 ambulatory DMD participants aged ≥ 7 and < 10 years of age
Locations
U.S., UK, EU (Belgium, Spain, Poland), Serbia, Canada, and Australia.
For more information, visit: clinicaltrials.gov/study/NCT07287189
SAT-3247-LT-001: Phase 2 TRAILHEAD Study
Summary
This is an open-label long-term safety and efficacy study of orally administered forazapadin (SAT-3247) in patients with DMD that previously participated in SAT-3247-CL-101. The study will assess the long-term safety, tolerability and potential efficacy of a 60 mg of orally administered forazapadin.
Participants
Individuals who completed the initial Phase 1b trial
Location
Melbourne, Australia
For more information on this study, visit: clinicaltrials.gov/study/NCT06867107
Charlie’s Story
A family’s long-awaited joy turned to heartbreak when their young son was diagnosed with Duchenne muscular dystrophy. Through determination, hope, and the promise of science, they’ve found a path forward — but questions remain about what the future will hold. Read their story of love, resilience, and daring to dream of a different outcome.
Forazapadin (SAT-3247) is investigational, has not been approved by the FDA or any regulatory authority, and its safety and effectiveness have not been established.
We recognize the urgent need for new treatment options for families living with Duchenne. While we are working quickly to develop forazapadin, it is currently only available through clinical trials.
Where We Are Now
- We require more safety data for children and longer-term use before broader access can be safely considered.
What This Means
- Safety is our top priority. At this stage, we have determined that more safety experience is needed before we would provide forazapadin outside a controlled clinical trial.
- We understand this is frustrating, and we are committed to advancing as quickly as possible, while prioritizing safety.
- We will keep you informed and continue exploring ways to make this treatment available to more people living with Duchenne.
Satellos is committed to reaching people of all ages with forazapadin as quickly as possible, guided by the data and with safety always in mind.
Satellos regularly reviews its policies to ensure they align with applicable laws and regulations. We may update this policy at any time as needed.