Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07287189 | Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients | PHASE2 | RECRUITING | 51 | — | — | Dec 8, 2025 | Jun 30, 2027 | Jun 5, 2026 | 21 | United States, Australia +6 |
| NCT06565208 | First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort | EARLY_PHASE1 | COMPLETED | 77 | — | — | Aug 21, 2024 | Apr 28, 2025 | May 15, 2025 | 2 | Australia |
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
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 |
| 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...