AP-101 is a recombinant human antibody designed to selectively deplete toxic, misfolded forms of SOD1 while preserving the protective function of physiological SOD1. 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. The company successfully completed a Phase 2 proof-of-concept study and prepares for Phase 3 clinical development.
SOD1 Pathology in ALS
Amyotrophic lateral sclerosis (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. 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.
AP-101 to Clear Misfolded SOD1
AP-101 is an investigational human-derived antibody directed against misfolded superoxide dismutase 1 (SOD1) designed to inhibit the spread of SOD1 pathology in the CNS of ALS patients. AP-101 received orphan drug designations from FDA, EMA and Swissmedic. The global phase 2 study with 73 participants met its primary endpoint related to safety and tolerability. The 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 6 months followed by 6 months of treatment with AP-101. These results were consistent with the hypothesis that targeting misfolded SOD1 is a disease-modifying approach in ALS. AL-S Pharma is currently preparing for a confirmatory Phase 3 clinical study.
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ALS is one of the most devastating human diseases and new therapies are urgently needed that can affect its underlying biology.
Michael Salzmann, CEO of AL-S Pharma