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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)
Safety and tolerability of SAT-3247 as compared to placebo
| Arm | Type | Description |
|---|---|---|
| SAT-3247 60 mg | ACTIVE_COMPARATOR | SAT-3247 60 mg oral tablets administered daily for 12 weeks |
| SAT-3247 120 mg | ACTIVE_COMPARATOR | SAT-3247 120 mg oral tablets administered daily for 12 weeks; note the 120 mg dose will not be studied in the US and Canada |
| placebo | PLACEBO_COMPARATOR | 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, with studies enrolling adult and pediatric male patients. The drug has received FDA Fast Track, Orphan Drug, and Rare Pediatric Disease designations.
SAT-3247 is a small molecule therapeutic designed to address the underlying pathology of Duchenne Muscular Dystrophy. While the specific molecular target has not been disclosed, the drug is being studied for its potential to modify disease progression in DMD. Its mechanism is being evaluated in ongoing clinical trials.
SAT-3247 is being developed by Satellos Bioscience Inc., a biopharmaceutical company traded under the ticker MSLE. The company is conducting clinical trials of SAT-3247 in multiple countries, including the United States, Australia, and several European nations, for the treatment of Duchenne Muscular Dystrophy.
SAT-3247 is currently in Phase 2 clinical development for Duchenne Muscular Dystrophy. It has completed an early Phase 1 study and is now being evaluated in Phase 2 trials, including a pediatric ambulatory patient study and a long-term follow-up study. The drug is investigational and not yet approved.
SAT-3247 has been studied in three clinical trials. NCT06565208 was a completed first-in-human SAD/MAD study in adults. NCT06867107 is an open-label long-term follow-up study in adults. NCT07287189 is a Phase 2 study in pediatric ambulatory patients, currently recruiting in multiple countries.
SAT-3247 is the sole identifier for this investigational compound; no alternative names have been reported. It is a distinct small molecule being developed specifically for Duchenne Muscular Dystrophy and is not known to be marketed under any other brand name.