AL-S Pharma’s amyotrophic lateral sclerosis (ALS) monoclonal antibody (mAb) has met its co-primary endpoints in a Phase II trial.
In the randomised, double-blind, placebo-controlled trial (NCT05039099), AP-101 combined with standard of care (SoC) met the safety and tolerability endpoints in patients with sporadic ALS and mutant SOD1-ALS; however, AL-S Pharma did not share data at this point.
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In the study, the AP-101 cohort also reported clinically meaningful changes in non-invasive ventilation, stabilisation of clinical disease-staging and neurofilament biomarkers after 12 months of treatment – it did not clarify if these were also statistically significant.
The Swiss biotech said that data will be shared with regulatory authorities, presented at upcoming ALS conferences, and submitted for publication in a peer-reviewed scientific journal. The trial enrolled 52 patients with sporadic ALS and 21 patients with mutant SOD1-ALS.
Principal investigator and Umea University’s senior consultant neurologist Peter Andersen said: “The study allowed us to rigorously assess patient safety, pharmacokinetics, and early signals of biological activity. The results support the hypothesis that misfolded SOD1 protein plays a more general role in ALS.”
AP-101 is a mAb that targets misfolded SOD1, a key pathology in both sporadic ALS and mutant SOD1-ALS. Evidence suggests that SOD1 misfolding and the formation of neurotoxic SOD1 species drive cell death in the motor system.
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By GlobalDataIn 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.
While ALS therapies remain limited, there is a significant push to try to provide more alternative therapies for patients. The HEALEY platform trial is a signal of this effort, with the study running simultaneous regimens, investigating a variety of ALS candidates. To date, very few drugs in the trial have shown efficacy, with four regimens of the trial simultaneously failing in February 2025.
GlobalData reports the ALS market across the eight major markets (8MM: US, France, Germany, Italy, Spain, UK, Japan, and Canada) was valued at $318m in 2019. It is projected to grow at a compound annual growth rate (CAGR) of 15%, reaching $1.28bn by 2029. A key driver for this will be the launch of new pipeline therapies.
