Companies in #Neurodegenerative Disease

Commercial-stage biopharmaceutical company developing small-molecule therapies for rare and neurodegenerative diseases; its lead drug AQNEURSA (levacetylleucine) is FDA-approved for Niemann-Pick disease type C and, as of September 2026, ataxia-telangiectasia — the first approved treatment for that disease anywhere.

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.

Neuphoria Therapeutics is a clinical-stage biopharmaceutical company developing CNS-focused therapeutics for neuropsychiatric and neurodegenerative conditions. The company's lead partnered program MK-1167, an adjunctive Alzheimer's disease therapy being co-developed with Merck under a 2022 partnership, was discontinued in mid-2026 after a planned interim Phase 2 efficacy review. This was the second Merck-Neuphoria compound to be discontinued (MK-4334 was previously shelved in 2022), effectively ending the partnership.

Passage Bio is a clinical-stage genetic medicines company developing gene therapies for central nervous system diseases, built around a research collaboration with the Gene Therapy Program at the University of Pennsylvania's Perelman School of Medicine. Its lead asset, PBFT02, is an AAV1 gene therapy delivering a functional granulin (GRN) gene for the treatment of frontotemporal dementia caused by progranulin deficiency, and is the sole program the company continues to advance after electing to wind down its earlier GM1 gangliosidosis, Krabbe disease and metachromatic leukodystrophy candidates. In June 2026 Passage Bio agreed an all-stock merger with Remix Therapeutics, a clinical-stage company developing small-molecule modulators of RNA processing; on completion the combined company will be renamed Remix Therapeutics, trade on Nasdaq as RMTX, and pursue Remix's lead mRNA degrader REM-422 in adenoid cystic carcinoma and AML/HR-MDS, with legacy Passage Bio holders retaining a minority of the combined entity. The company has flagged going-concern doubt on a declining cash balance and an accumulated deficit of roughly $720 million, and the transaction is subject to a stockholder vote including a proposed reverse stock split.