Journal
Safety and efficacy findings in the first-in-human trial (FIH) of the oral splice modulator branaplam in type 1 spinal muscular atrophy (SMA): interim results
LMI070X2201 is an open-label, multi-part, FIH study of oral branaplam (formerly LMI070) in infants with Type 1 spinal muscular atrophy with 2 SMN copies. The purpose of Part 1 of this study was to evaluate the safety, tolerability, PK, PD and efficacy after 13 weeks of treatment and to estimate the Maximum Tolerated Dose and optimal dosing regimen of enterally administered branaplam in patients with Type 1 SMA. Treatment extensions are allowed after the initial 13-week treatment period. Safety, PK, PD and efficacy outcomes were collected for an additional 13 months following completion of the initial treatment period.