Paper in CELL Further Validates PIKfyve as Promising Therapeutic Target

By Robert Scannevin, PhD, Chief Scientific Officer

The journal Cell recently published a paper, “PIKFYVE inhibition mitigates disease in models of diverse forms of ALS” (Hung et al, 2023, Cell 186, 1-17) that we believe further validates PIKfyve as a promising therapeutic target in ALS and sheds light on a novel mechanism for potentially clearing pathological protein aggregates. Using our CONVERGE™ platform, Verge identified PIKfyve as a target so it is encouraging to see robust, independent validation across a number of sporadic and familial in vitro and in vivo ALS models. These studies demonstrate that PIKfyve inhibition reduced motor impairment and extended survival in multiple animal models of ALS, as well as in diverse patient-derived motor neurons. 

PIKfyve inhibition targets a common dysregulated pathway in ALS and has potential broad clinical utility, unlike investigational therapies that focus on specific genetically defined populations that make up 10% or less of all ALS patients. We are looking forward to continuing to advance our clinical development program in ALS with VRG50635, our lead small molecule PIKFYVE inhibitor. If you would like to learn more about PIKfyve and its discovery as a possible therapeutic target for ALS, check out this paper in Nature.