Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease marked by the degeneration of motor neurons in the spinal cord and brain resulting in progressing paralysis and death. In absence of effective treatment, patients survive on average two to five years following their diagnosis. The worldwide population of ALS patients is estimated 450,000.

For the potential future treatment of ALS, Neurimmune developed antibodies against misfolded forms of superoxide dismutase 1 (SOD1) and C9orf72 related peptides. C9orf72 is the main genetic causes of ALS and frontotemporal dementia. Neurimmune's anti-SOD1 antibody AP-101 is currently in clinical Phase 2 development in ALS patients.

Misfolded SOD1 accumulates in spinal cord and brain of patients with ALS, suggesting a role as a drug target. Neurimmune’s SOD1 lead antibody is highly effective at therapeutic doses in transgenic models of ALS: Chronic antibody treatment significantly reduces SOD1 pathology and rescues spinal cord motor neurons, resulting in less muscle atrophy, improved motor functions and increased survival times.

Neurimmune co-founded and co-financed the subsidiary AL-S Pharma AG to develop the SOD1 antibody AP-101 (NI005) to clinical proof-of-concept in collaboration with TVM Capital Life Science and Lilly's Chorus unit.