AP-101 progressed through a Phase 1 study initiated in 2019, establishing safety and tolerability in ALS patients using a 3+3 design. The subsequent Phase 2 multicenter, randomized, double-blind, placebo-controlled trial launched in 2021 across Europe, South Korea, Canada, and the U.S., enrolling 73 patients – 52 with sporadic ALS and 21 with SOD1-ALS. The study included a 24-week treatment period followed by a 24-week open-label extension. Primary endpoints were safety and tolerability, with secondary and exploratory endpoints assessing pharmacokinetics, pharmacodynamics, biomarkers – including neurofilament levels in cerebrospinal fluid and serum, as well as various clinical endpoints. The rationale for testing in both populations is based on evidence that misfolded SOD1 contributes to both sporadic ALS and SOD1-ALS. The company presented positive phase 2 results at the 36th International Symposium on ALS/MND.
