AI Therapeutics is now OrphAI Therapeutics
A new generation of medicines.
OrphAI Therapeutics is advancing three drugs into clinical trials/development: AIT-101 for ALS; LAM-001 for Pulmonary Hypertension (PH), Bronchiolitis Obliterans Syndrome (BOS) and Pulmonary Sarcoidosis; AIT-102 for oncology indications including Ewing Sarcoma (ES), Rhabdoid Tumor (RT), and SWI/SNF mutated/dysregulated cancer.
Amyotrophic Lateral Sclerosis
LAM-002 (apilimod) is a first-in-class, PIKfyve kinase inhibitor that activates transcription factor EB (TFEB), the master regulator of lysosomal biogenesis. TFEB activation clears toxic aggregates that drive neurodegenerative disorders. A wealth of new data points to the accumulation of toxic proteins as a common mechanism in neurodegenerative conditions disorders including ALS, FTD, Parkinson’s disease and Alzheimer’s disease. LAM-002 has completed a Phase 2 clinical trial for patients suffering from ALS with a mutation in the gene c9ORF72.