SIR2501

SIR2501 is a brain-penetrant, small molecule inhibitor of SARM1 (sterile alpha and TIR motif containing 1) being developed by Sironax. It acts as a non-covalent, allosteric inhibitor that prevents axonal degeneration, which is a common feature of various neurodegenerative and peripheral nervous system diseases. The drug is currently in Phase I development for indications including amyotrophic lateral sclerosis (ALS) and chemotherapy-induced peripheral neuropathy (CIPN), specifically to prevent or mitigate paclitaxel-induced neuropathy in patients with solid tumors. Preclinical data have demonstrated neuroprotective effects in models of ALS and CIPN, and a Phase I trial in healthy volunteers has been completed to assess safety, tolerability, and pharmacokinetics.