Objective: To determine safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of orally administered SAT-3247 in healthy adult volunteers and adult participants with DMD.
Background: SAT-3247 targets adapter associated kinase 1 (AAK1), a regulator of muscle stem cell asymmetric division and regeneration. In animal models of DMD, SAT-3247 improved markers of muscle regeneration and increased muscle strength.
Study Design: This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy volunteers (HVs) and adult participants with DMD. The study will be conducted in 4 parts. Part A is a single ascending dose (SAD) part that will enroll approximately 40 HVs in up to 5 dose cohorts. Each participant will receive a single oral dose of SAT 3247 or matched placebo on Day 1; each cohort will receive a higher dose than the prior cohort. Part B is a multiple ascending dose (MAD) part that will enroll approximately 32 HVs in up to 4 sequential dose cohorts. Each participant will receive a daily oral dose of SAT-3247 or matched placebo on Day 1 to Day 7; each cohort will receive a higher dose than the prior cohort. Part C will assess the effect of food on the PK of SAT-3247 in a fixed sequence, crossover design. The dose to be tested will be determined by the Safety Review Committee (SRC) following review of safety, tolerability, and available PK and PD data from Part A. Approximately 8 healthy participants who completed Part A at the anticipated dose level (in a fasted state) will crossover into a subsequent fed cohort and receive a single dose of the same randomized investigational product at the same dose level that they received in Part A but, following a high fat meal. Part D is an open-label multiple dose cohort that will comprise 10 adult males with genetically confirmed DMD. Each participant will receive SAT-3247 once daily for 5 consecutive days during each of the 4 weeks.
Results: Summary safety and pharmacokinetics data from the healthy volunteer portion of the study will be presented as well as an update on the enrollment of adult participants in the DMD part of the study.