CSM-101 / Casma Therap - LARVOL DELTA

Csm-101, a small molecule agonist of trpml1 for parkinson's-related disorders (Neuroscience 2025) - "CONCLUSIONS CSM-101 is a promising drug candidate associated with significant efficacy in preclinical models of GD and PD. The findings support the concept that targeting lysosomal homeostasis via activation of TRPML1 is an attractive therapeutic approach."

CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

CSM-101 is a Small Molecule Agonist of TRPML1 for Parkinson's-Related Disorders (MDS Congress 2025) - "CSM-101 is a brain-penetrant TRPML1 agonists that demonstrates significant efficacy in preclinical models of GD and PD. Given the overall profile of CSM-101, this drug candidate is being advanced to evaluate the concept of targeting lysosomal homeostasis in neuronopathic forms of GD and GBA-PD."

CNS Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

Casma Therapeutics Nominates CSM-101 as a Development Candidate for the Treatment of Rare and Common Forms of Parkinson’s Disease (Businesswire) - "Casma Therapeutics, Inc....today announced the nomination of its first development candidate, CSM-101, a first-in-class TRPML1 agonist....The company will share preclinical data supporting the progression of CSM-101 into IND-enabling studies in an oral presentation at the GBA1 Meeting 2025, taking place June 5-7 in Montreal, Canada.... In preclinical studies, CSM-101 demonstrated high CNS exposure, potent activity across clinically relevant models, significant reductions in toxic lipid levels, decreased neuroinflammation, and improved survival in multiple Gaucher’s disease models. In GBA-associated Parkinson’s disease and idiopathic Parkinson’s disease models, CSM-101 reversed a pathological phenotype, lowered toxic alpha-synuclein levels and preserved dopaminergic neurons, supporting its potential as a disease-modifying therapy."

Commercial • Preclinical • Gaucher Disease • Parkinson's Disease