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Also known as AAK1 inhibitor
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 not yet approved for any indication and remains in clinical development. Satellos Bioscience is evaluating it in pediatric ambulatory patients and in long-term follow-up studies.
SAT-3247 targets AAK1, also known as AP2-associated protein kinase 1. AAK1 is a kinase involved in cellular processes, and inhibiting it is the intended mechanism of action for this small molecule. The drug is designed to address the underlying muscle degeneration in Duchenne Muscular Dystrophy through this target.
SAT-3247 is being developed by Satellos Bioscience Inc., a biotechnology company. The company's ticker symbol is MSLE. Satellos is the sponsor of the clinical trials evaluating SAT-3247 in Duchenne Muscular Dystrophy.
SAT-3247 is in Phase 2 clinical development. It has not been approved by the FDA. The program includes a completed first-in-human Phase 1 study and ongoing Phase 2 trials in Duchenne Muscular Dystrophy. The FDA has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations for SAT-3247.
SAT-3247 is being studied in three clinical trials. The Phase 2 trial NCT07287189 is evaluating SAT-3247 in pediatric ambulatory patients and is active but not recruiting. The Phase 2 long-term follow-up study NCT06867107 is enrolling by invitation. The completed first-in-human Phase 1 study is NCT06565208.
No, SAT-3247 is not FDA approved. It is an investigational drug still in clinical development. The FDA has granted it Fast Track, Orphan Drug, and Rare Pediatric Disease designations, which are intended to support and expedite the development and review of drugs for serious conditions.