CAMBRIDGE, Mass. — Casma Therapeutics Inc. has received approximately $7.6 million in funding from The Michael J. Fox Foundation for Parkinson’s Research to support development of its lead therapeutic candidate targeting lysosomal dysfunction in Parkinson’s disease.
The non-dilutive funding, awarded through two competitive grant programs, will support preclinical development of CSM-101, a first-in-class TRPML1 agonist designed to restore lysosomal function in genetically driven neurodegenerative diseases. The funding will help Casma generate biomarker data and conduct investigational new drug (IND)-enabling studies needed to advance the therapy into first-in-human clinical trials.
“These awards reflect strong external confidence in both the relevance of TRPML1 as a therapeutic target and our disciplined approach to advancing CSM-101 to the clinic,” said Frank Gentile, Ph.D., Chief Executive Officer of Casma Therapeutics. “By supporting translational biomarkers and IND-enabling studies in parallel, this funding allows us to advance CSM-101 toward the clinic with a high level of scientific and regulatory rigor.”
CSM-101 is an orally available, central nervous system-penetrant small molecule designed to activate TRPML1, a lysosomal ion channel involved in cellular waste processing. Researchers have increasingly focused on TRPML1 due to evidence linking lysosomal dysfunction to both rare genetic forms and more common cases of Parkinson’s disease.
The funding consists of two complementary grants from The Michael J. Fox Foundation. A $2.1 million Targets to Therapies grant will support validation of TRPML1 biology and development of translational biomarkers intended to measure lysosomal activation and guide clinical trial design, including dose selection. A second $5.5 million Therapeutics Pipeline Program grant will fund IND-enabling studies required to move CSM-101 into clinical testing.
“The Michael J. Fox Foundation supports research that advances understanding of the biological pathways underlying Parkinson’s disease and translates that knowledge into well-designed therapeutic programs,” said Shalini Padmanabhan, Ph.D., Senior Vice President of Discovery and Translational Research at The Michael J. Fox Foundation. “Through two complementary investments, one focused on strengthening confidence in target biology and biomarker development and another supporting IND-enabling studies, we aim to address key translational gaps and enable more informed evaluation of emerging therapeutic strategies for Parkinson’s disease.”
Casma said the combined funding will allow the company to link mechanistic research on TRPML1 with translational biomarker development and regulatory preparation needed for clinical evaluation.
“The support from The Michael J. Fox Foundation for these two programs is designed to work together, from deepening our mechanistic understanding of TRPML1 biology to preparing CSM-101 for clinical evaluation,” said Leon Murphy, Ph.D., Chief Scientific Officer of Casma Therapeutics and the Principal Investigator for both grants. “By linking mechanistic understanding of TRPML1 activation with biomarker development and IND-enabling studies, we are establishing the translational foundation needed to evaluate target engagement in patients and advance CSM-101 with confidence in the clinic.”
