CoA Therapeutics Inc., in April began a phase 1 study of the safety of a potential drug for Pantothenate Kinase-Associated Neurodegeneration (PKAN) by testing it in healthy volunteers.
The CoA Therapeutics team plans to begin clinical trials in PKAN patients in late 2022, once it is determined that the drug candidate they are calling BBP-671 is safe and a suitable dose for PKAN patients has been determined.
PKAN is one of the most common NBIA disorders.
The necessary preclinical animal studies with BBP-671 have been completed, and the Food and Drug Administration has approved the company’s application for the use of the Investigational New Drug in humans. Orphan drug designation has also been granted in both the US and Europe.
The patented compound changes the action of pantothenate, a key enzyme involved in metabolism, as it’s converted into CoA. CoA is deficient in PKAN individuals. The drug is a unique synthetic molecule that can immediately relieve the movement disorder and extend the life span in a mouse model with brain CoA deficiency. It compensates for the missing CoA, and the company hopes it will have a similar affect in PKAN individuals.