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.