- Thursday, September 4, 2025 @ 1:00 pm
In 2016, Neurimmune and TVM Capital Life Science co-created AL-S Pharma to develop AP-101. AL-S Pharma has executed its innovative clinical plan for AP-101 in collaboration with an international network of ALS experts.
- Phase 2 of AP-101 in sporadic ALS and mutant SOD1-ALS met its primary safety and tolerability endpoint
- Clinically meaningful changes in outcome measures and stabilization of biomarkers were observed
- Data will be presented at upcoming scientific conferences and AL-S Pharma plans to engage with regulatory authorities later this year
- AP-101 is a first-in-class investigational human antibody targeting misfolded SOD1, a pathological hallmark of ALS
AL-S Pharma today announced positive topline results from the Phase 2 clinical study evaluating AP-101, a potential first-in-class, disease-modifying treatment for amyotrophic lateral sclerosis (ALS). AP-101 is a monoclonal antibody targeting misfolded SOD1, a key pathology in both sporadic ALS and mutant SOD1-ALS.1 AP-101, in addition to standard of care, met its primary endpoint related to safety and tolerability. Clinically meaningful changes in exploratory clinical outcome measures related to survival and non-invasive ventilation, as well as stabilization of clinical disease-staging and neurofilament biomarkers, were observed following 12 months of treatment. Results will be shared with regulatory authorities, presented at upcoming ALS conferences, and submitted for publication in a peer-reviewed scientific journal.
“We are excited by the topline Phase 2 results of AP-101 for ALS, a devastating degenerative disease for which new treatments are urgently needed,” said Michael Salzmann, PhD, Chief Executive Officer, AL-S Pharma. “We are grateful to the participants, their families and the international network of ALS experts who made this trial possible, and we look forward to sharing results from the study at the International Symposium on ALS/MND.”
“This is the first Phase 2 study to assess a SOD1-targeted therapeutic in both sporadic ALS and in ALS patients with mutations in the SOD1 gene. The study allowed us to rigorously assess patient safety, pharmacokinetics, and early signals of biological activity,” said Peter Andersen, Professor and Senior Consultant Neurologist, Umea University, Sweden, and Principal Investigator for the AP-101 Phase 2 study. “The completion of the trial marks an important step in evaluating the therapeutic potential of AP-101 for broad use in patients with ALS. The results support the hypothesis that misfolded SOD1 protein plays a more general role in ALS”.
ALS is a fatal neurodegenerative disease characterized by loss of both upper and lower motor neurons. Symptoms typically begin focally with spread in a contiguous anatomical pattern, leading to muscle weakness, respiratory issues, swallowing and speaking difficulties, and ultimately to death. Very limited therapeutic options are available.
SOD1 pathology has been proposed to be a disease driver in both genetically determined SOD1-ALS as well as in sporadic ALS.2,3 Pathological, genetic and preclinical evidence suggest that SOD1 misfolding and the formation of neurotoxic SOD1 species drive cell death in the motor system. AP-101 is a first-in-class antibody therapeutic targeting misfolded SOD1.
AL-S Pharma’s Phase 2 study is a multicenter, randomized, double-blind, placebo-controlled study evaluating safety, tolerability, pharmacodynamic markers, and pharmacokinetics of AP-101 in both patients with sporadic ALS (N=52) and patients with mutant SOD1-ALS (N=21) over 24 weeks followed by a 24-week open-label extension and a safety follow-up period. Further information on the clinical study of AP-101 for ALS can be accessed on ClinicalTrials.gov (study number NCT05039099).
