Characterizing Safety & Proof-Of-Concept for VRG50635, a Novel Small Molecule Inhibitor ALS Candidate Therapeutic Targeting PIKfyve

Time: 11:30 am
day: Day Two Track B AM


  • Rationale for testing PIKfyve inhibition in ALS
  • Learnings of safety, tolerability and PK/PD in healthy volunteers
  • Design of a novel proof-concept study for the initial investigation of VRG505635 in ALS