ZURICH, Switzerland, June 24, 2026
AL-S Pharma AG announced positive Phase 2 clinical results for its lead investigational antibody AP-101 at the European Network to Cure ALS (ENCALS) 2026 Congress. The findings further support misfolded superoxide dismutase 1 (SOD1) as a promising therapeutic target in both SOD1-associated and sporadic amyotrophic lateral sclerosis (ALS). AP-101 is a first-in-class, Phase 3-ready human-derived antibody designed to selectively target the toxic misfolded form of SOD1, aiming to slow disease progression by preventing the spread of misfolded proteins while promoting their natural clearance from the nervous system
Phase 2 Trial Demonstrates Survival and Biomarker Improvements
The global Phase 2 AP-101-02 study achieved its primary endpoint of safety and tolerability while demonstrating clinically meaningful evidence of disease modification. Patients treated with AP-101 experienced reductions in key neuroaxonal injury biomarkers, including serum neurofilament light chain (NfL) and cerebrospinal fluid phosphorylated neurofilament heavy chain (pNfH), after six months of treatment. Exploratory analyses also showed prolonged survival and delayed need for ventilatory support compared with patients who initially received placebo. Positive treatment effects were observed in both sporadic ALS patients and individuals carrying SOD1 mutations, while disease stabilization was reflected through King’s staging and slower decline in ALS Functional Rating Scale-Revised (ALSFRS-R) scores among selected patient groups.
Phase 3 Program Planned Following Encouraging Results
The Phase 2 study enrolled 73 patients across the United States, Canada, the United Kingdom, the European Union, and South Korea, with participants randomized to receive intravenous AP-101 or placebo every three weeks. AP-101 maintained a favorable safety profile, with adverse events comparable to placebo and no treatment-induced antibody responses reported. Based on these findings, AL-S Pharma plans to initiate a confirmatory Phase 3 clinical trial in the first quarter of 2027. The therapy has already received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and Swissmedic.
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AL-S Pharma Highlights Emerging ALS Biology at ENCALS 2026
Alongside the clinical presentation, AL-S Pharma hosted a scientific symposium featuring internationally recognized ALS researchers who discussed advances in genetics, neurofilament biomarkers, protein misfolding biology, patient stratification, and future clinical trial design. If successfully confirmed in Phase 3, AP-101 could become one of the first disease-modifying therapies targeting misfolded SOD1 across both genetic and sporadic forms of ALS, potentially expanding treatment options for patients living with this devastating neurodegenerative disease.
Source: AL-S Pharma press release
