AKV9

AKV9 is a first-in-class small molecule protein aggregation inhibitor developed by Akava Therapeutics. It was discovered in the Silverman Laboratory at Northwestern University and is designed to improve the health of upper motor neurons that degenerate in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), and ALS/frontotemporal lobar degeneration (FTLD). Mechanistically, AKV9 inhibits misfolded protein aggregation—including amyloid beta oligomers and mutant SOD1-induced aggregates—restores cellular integrity by improving mitochondrial and endoplasmic reticulum function, and protects neuronal structure. Preclinical studies have shown efficacy in mouse models of ALS with both SOD1 toxicity and TDP‑43 pathology as well as Alzheimer’s disease models. The compound has received FDA clearance for Phase 1 clinical trials in ALS.

Other names

1,3-Cyclohexanedione, 5-[(1S)-1-[3,5-bis(trifluoromethyl)phenoxy]ethyl]-5-((S)-1-(3,5-bis(trifluoromethyl)phenoxy)ethyl)-3-hydroxycyclohex-2-en-1-one5-[(1S)-1-[3,5-Bis(trifluoromethyl)phenoxy]ethyl]-1,3-cyclohexanedione