SAM001 is a small molecule in development for treatment of ALS and Parkinson’s disease. SAM001 activates TRPML1, an ion channel expressed on lysosomes and an important regulator of autophagy.
In cultures of neurons derived from patients, SAM001, an oral daily therapy for ALS patients regardless of genotype, demonstrated greater clearance of pathological protein aggregates such as TDP-43 and α– synuclein which are hallmarks of ALS and Parkinson’s. In animal disease models, SAM001 has elicited improvements across various measurements including motor function and survival.
SAM001 clinical strategy will be supported by use of biomarkers that have emerged from our understanding of molecular mode of action. In the early stages of clinical development, this approach enables detection of target engagement and changes in disease-relevant biomarkers in addition to traditional endpoints such as safety and pharmacokinetics.