Companies in #Amyotrophic Lateral Sclerosis

Clinical-stage biotech developing allogeneic umbilical cord blood-derived T-regulatory cell therapies for autoimmune diseases and inflammatory disorders.

MediciNova is a biopharmaceutical company developing small-molecule therapeutics licensed from Japanese originators for serious diseases with unmet medical need, and is dual-listed on Nasdaq and the Tokyo Stock Exchange. Its lead compound MN-166 (ibudilast) is an oral anti-inflammatory and neuroprotective agent in clinical development for amyotrophic lateral sclerosis (the Phase 2b/3 COMBAT-ALS registration trial enrolled 234 patients, with top-line data expected by year-end 2026), progressive multiple sclerosis, chemotherapy-induced peripheral neuropathy, degenerative cervical myelopathy, glioblastoma and prevention of acute respiratory distress syndrome; an expanded-access program in ALS is supported by a $22 million NIH grant. The second compound, MN-001 (tipelukast), is an orally bioavailable small molecule in development for fibrotic and metabolic disorders including nonalcoholic fatty liver disease and hypertriglyceridemia. Management has framed 2026 as a data-driven year led by readouts from these two programs rather than by financing.