AL-S Pharma’s ALS Antibody AP-101 Meets Phase II Endpoints

Swiss biotech AL-S Pharma announced positive topline results from its Phase II trial (NCT05039099) of AP-101, a monoclonal antibody targeting misfolded SOD1 in amyotrophic lateral sclerosis (ALS). The randomized, double-blind, placebo-controlled study enrolled 52 patients with sporadic ALS and 21 with mutant SOD1-ALS. AP-101, combined with standard of care, met its co-primary endpoints of safety and tolerability.

Clinically meaningful signals were also observed, including stabilization of clinical staging, improvements in non-invasive ventilation, and reductions in neurofilament biomarkers after 12 months of treatment, though statistical significance was not disclosed. AL-S Pharma said results support the role of misfolded SOD1 in ALS pathogenesis.

The company plans to share full data with regulators, present at upcoming ALS conferences, and submit findings for peer-reviewed publication. With ALS therapies still limited, AP-101 adds to ongoing global efforts, such as the HEALEY platform trial, to expand treatment options.

05-09-2025