Results supported by key neuroaxonal injury biomarkers pNfH and NfL after six months of treatment
AL-S Pharma’s AP-101 is an investigational human-derived antibody therapeutic that selectively targets the misfolded, toxic form of SOD1 to disrupt progressive spread of ALS
Data featured in oral presentation at the AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Diseases
Preparations underway to advance AP-101 into confirmatory Phase 3 clinical trial with initiation aimed for late 2026
AL-S Pharma AG, a clinical-stage biotechnology company discovering and developing human antibodies that target misfolded proteins implicated in amyotrophic lateral sclerosis (ALS), today announced the presentation of new clinical data from the global Phase 2 clinical trial (AP-101-02) evaluating the company’s lead program, AP-101, in patients with ALS. AP-101 is an investigational human-derived antibody directed against misfolded superoxide dismutase 1 (SOD1). AL-S Pharma’s AP-101 is designed to inhibit the spread of SOD1 pathology in the central nervous system of people living with ALS, helping the body’s immune system clear these harmful proteins.1
The global Phase 2 clinical trial met its primary endpoint related to safety and tolerability. New results provide additional evidence of clinically meaningful disease modification and prolonged survival supported by reductions in key neuroaxonal injury biomarkers, serum neurofilament light chain (NfL) and cerebrospinal fluid phosphorylated neurofilament heavy chain (pNfH) after six months of treatment. Adverse events were comparable to placebo, and no AP-101-induced antibody responses were reported, reinforcing AL-S Pharma’s clinical development approach.
The AP-101-02 clinical trial results will be featured in an oral presentation at the AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders. The presentation will be delivered by Angela Genge, M.D., chief medical officer of AL-S Pharma, today at 8:40AM CET in Hall A2 (Session 5210).
“These data show something we rarely see in ALS – objective evidence of clinically meaningful disease modification that tracks directly with prolonged survival,” said Dr. Genge. “The Phase 2 study results with AP-101 are consistent with the hypothesis that targeting misfolded SOD1 is a disease-modifying approach in ALS.2 At AL-S Pharma, we believe AP-101 could fundamentally transform the treatment of ALS. We look forward to continue advancing the AP-101 clinical program so that we can bring a much-needed new treatment option to people living with ALS rapidly.”
The prespecified analysis of an exploratory composite endpoint revealed that early treatment with AP-101 prolonged survival and delayed ventilatory support in comparison to study participants receiving placebo for six months followed by six months of treatment with AP-101. Positive treatment effects were observed in both the sporadic ALS cohort (p = 0.013) and the SOD1 mutation carrier cohort (p = 0.036). Effects on survival were accompanied by disease stabilization as measured by King’s staging. Functional decline measured by ALSFRS-R was reduced in study participants with elevated misfolded SOD1 at baseline and in SOD1 mutation carriers.
AL-S Pharma is currently preparing for a confirmatory Phase 3 clinical trial of AP-101 in ALS aimed to initiate end of 2026. AP-101 received Orphan Drug designations from the U.S. Food and Drug Administration, the European Medicines Agency, and Swissmedic.
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