- In April 2023, Athira Pharma announced promising Phase 2 trial results for its lead small molecule candidate, fosgonimeton, targeting the HGF/MET neurotrophic pathway for Alzheimer’s disease. The results demonstrated improvements in cognitive function and synaptic health, positioning the therapy as a potential disease-modifying treatment. This development underscores Athira’s commitment to pioneering neuroprotective mechanisms that address core neurodegenerative processes and highlights growing momentum in small molecule innovation for chronic neurological conditions
- In March 2023, Annovis Bio expanded its clinical program for buntanetap, a novel oral small molecule designed to inhibit multiple neurotoxic proteins implicated in Alzheimer’s and Parkinson’s disease. The company announced the initiation of a new Phase 3 trial for Parkinson’s patients, aiming to validate early data showing cognitive and motor improvements. This move reflects the broader industry push toward multi-target small molecule approaches that address complex neurodegenerative pathologies
- In February 2023, Gain Therapeutics presented new preclinical data on its proprietary small molecules targeting misfolded protein stabilization in neurodegenerative diseases. The company’s lead candidate, GT-02287, demonstrated neuroprotective effects in Parkinson’s disease models by enhancing lysosomal function and reducing α-synuclein pathology. These findings further reinforce the potential of structurally targeted small molecules in addressing the root causes of neuronal degeneration
- In January 2023, the European Medicines Agency (EMA) granted Orphan Drug Designation to a novel small molecule developed by BioHaven Pharmaceuticals for the treatment of amyotrophic lateral sclerosis (ALS). The designation provides regulatory and commercial incentives, accelerating the path to market for promising neuroprotective therapies in rare neurodegenerative conditions. BioHaven’s advancement highlights the growing strategic focus on expanding small molecule pipelines into underserved neurological indications
- In January 2023, NMD Pharma, a Danish biotech company, raised additional Series B funding to advance its clinical development of ClC-1 chloride ion channel modulators—first-in-class small molecules aimed at enhancing neuromuscular function in conditions such as spinal muscular atrophy and myasthenia gravis. This funding milestone underscores investor confidence in targeted neuroprotective strategies and reflects the growing diversity of mechanisms being explored through small molecule approaches
