Approval Probability
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ML Risk
SAT-3247 · 3 trials · 6 indications
Occurrence of treatment emergent adverse events and relationship to investigational product
occurrence of clinically significant changes in physical exam, clinical laboratory measures, vital signs, and ECG
change from baseline in muscle force as determined by dynamometry
Incidence, temporal profile, and severity of treatment emergent adverse events (TEAEs)
Changes from baseline in intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in biceps brachii following treatment with SAT-3247.
Safety and tolerability of SAT-3247 as compared to placebo
| Arm | Type | Description |
|---|---|---|
| SAT-3247 60 mg | ACTIVE_COMPARATOR | Part 1: SAT-3247 60 mg oral tablets administered daily for 12 weeks. Part 2: SAT-3247 60 mg oral tablets administered daily for an additional 9 months after completing Part 1 |
| SAT-3247 120 mg | ACTIVE_COMPARATOR | Part 1: SAT-3247 120 mg oral tablets administered daily for 12 weeks. Part 2: SAT-3247 120 mg oral tablets administered daily for an additional 9 months after completing Part 1; note the 120 mg dose will not be studied in the US and Canada |
| placebo | PLACEBO_COMPARATOR | Part 1: placebo oral tablets administered daily for 12 weeks |
| Treatment Arm | EXPERIMENTAL | SAT-3247 60 mg administered orally in a 5-days on/2-days off (weekday) dosing regimen |
| SAT-3247 | EXPERIMENTAL | SAT-3247 is an oral tablet that is a potent, muscle penetrant, small molecule inhibitor of AAK1; inhibition of AAK1 rescues perturbed asymmetric division of satellite stem cells, resulting in increased muscle regeneration in animal models of DMD. In vitro and in vivo animal pharmacology studies have demonstrated the efficacy of SAT-3247 in improving muscle strength and the necessary target coverage to maximize functional muscle improvement. Part A: Participants will receive one oral dose of SAT-3247 in accord with cohort assignment (1) 10 mg, (2) 50 mg, (3) 150 mg, (4) 300 mg, (5) 400 mg Part B: Participants will receive one oral dose of SAT-3247 daily for seven days in accord with cohort assignment (1) 60 mg, (2) 120 mg, (3)180 mg, (4) 240 mg Part C: Participants will receive one oral dose of SAT-3247 150 mg, following completion of Part A at the same dose Part D: All participants will receive one SAT-3247 60 mg dose once daily for 5 consecutive days of each of 4 weeks |
| Name | Type | Description |
|---|---|---|
| SAT-3247 | DRUG | SAT-3247 is a selective AAK1 inhibitor for oral tablet administration which promotes functional rescue of asymmetric satellite cell division, resulting in the robust production of muscle progenitor cells, subsequent improvement in muscle regeneration, and enhanced muscle function. |
| Placebo | DRUG | matching placebo oral tablets |
| matched placebo | DRUG | matched placebo |
Key Inclusion Criteria: * Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene. * Male DMD patients who are ambulatory and aged ≥ 7 to \< 10 years at the time of screening. * Stable dose of systemic glucocorticoi...
SAT-3247 is an investigational small molecule being developed for the treatment of Duchenne Muscular Dystrophy (DMD), a genetic neuromuscular disease. It is currently in Phase 2 clinical development and has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA.
SAT-3247 targets AAK1, also known as AP2-associated protein kinase 1. AAK1 is a kinase involved in regulating cellular processes relevant to muscle repair and regeneration. By targeting AAK1, SAT-3247 is designed to address the underlying muscle degeneration associated with Duchenne Muscular Dystrophy.
SAT-3247 is being developed by Satellos Bioscience Inc., a biotechnology company focused on regenerative muscle therapeutics. Satellos Bioscience trades under the ticker MSLE.
SAT-3247 is in Phase 2 clinical development for Duchenne Muscular Dystrophy. It has not been approved by the FDA and remains an investigational therapy. The Phase 2 program includes a randomized, double-blind, placebo-controlled study in pediatric ambulatory patients and an open-label long-term follow-up study.
SAT-3247 is being evaluated in three clinical trials. The Phase 2 study NCT07287189 is recruiting pediatric ambulatory DMD patients across the United States, Australia, Belgium, Canada, Poland, Serbia, Spain, and the United Kingdom. NCT06867107 is an open-label long-term follow-up study enrolling by invitation. NCT06565208, a first-in-human Phase 1 study, has been completed.
SAT-3247 has received three FDA designations for Duchenne Muscular Dystrophy: Fast Track, Orphan Drug, and Rare Pediatric Disease. These designations are intended to support and expedite the development of therapies for serious rare conditions. SAT-3247 remains investigational and has not received FDA approval.