Companies in #Amyotrophic Lateral Sclerosis

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

LeonaBio, formerly Athira Pharma, is a clinical-stage biopharmaceutical company developing small-molecule therapeutics for diseases with high unmet need in oncology and neurology, with operations based in Bothell, Washington. Its lead program is lasofoxifene, a nonsteroidal selective estrogen receptor modulator (SERM) licensed exclusively from Sermonix Pharmaceuticals for most global territories and developed as a targeted therapy for estrogen receptor-positive, HER2-negative, ESR1-mutated metastatic breast cancer; the ongoing Phase 3 ELAINE-3 trial is evaluating lasofoxifene in combination with the CDK4/6 inhibitor abemaciclib after progression on aromatase inhibitors and CDK4/6 inhibitors. The company's second clinical program, brelgometon (ATH-1105), is an orally available, brain-penetrant small molecule that positively modulates the neurotrophic HGF system and is being developed for amyotrophic lateral sclerosis following a completed first-in-human Phase 1 study in 80 healthy volunteers. LeonaBio reported a $19.0 million net loss for the second quarter of 2026 with $51.1 million in cash and investments, and had enrolled 495 of approximately 600 planned patients in ELAINE-3 as of that update. The company trades on the Nasdaq Capital Market under the symbol LONA.

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.

Pasithea Therapeutics is a clinical-stage biotechnology company developing small-molecule and antibody therapies for RASopathies, MAPK pathway-driven tumours and central nervous system disorders. Its lead candidate, PAS-004, is a next-generation macrocyclic MEK inhibitor designed for chronic dosing with a long half-life and partial pathway inhibition intended to widen the therapeutic window relative to earlier MEK inhibitors; it is in a Phase 1b dose-escalation study in adults with neurofibromatosis type 1, with cutaneous neurofibromas as the primary development focus and additional potential in BRAF-driven tumours and ETS2-driven inflammatory disease. The company is also developing PAS-003 for amyotrophic lateral sclerosis and PAS-001 for schizophrenia. Pasithea was founded in 2020 by Dr. Lawrence Steinman and listed on Nasdaq in September 2021; it is headquartered in Miami Beach, Florida and operates with a small team of five employees.