By: Amber Denton
It is with a heavy heart that we inform the community that CoA Therapeutics has decided to discontinue the clinical trial of BBP-671, the compound the company was developing as a potential treatment for Pantothenate Kinase-Associated Neurodegeneration (PKAN). This decision was made after studies showed they were unable to identify a clinical trial dose that adequately balanced safety (toxicity) and potential clinical benefit.
In March, we shared that CoA Therapeutics announced a delay in their PKAN clinical trial due to the need for additional laboratory studies on BBP-671 to determine critical dosing and safety information. A significant challenge identified during these additional studies was the wide variability of BBP-671 blood levels observed in humans. Individuals taking the same dose of BBP-671 showed vastly different blood concentrations of the drug. This inconsistency posed a significant risk in determining an appropriate and safe dose for PKAN patients.
Toxicology studies in animal models of PKAN helped to establish a specific safety limit, or target blood level, for BBP-671 that cannot be exceeded due to safety and FDA regulations. The target blood level needed for efficacy was shown to be dangerously close to the safety limit.
Despite multiple studies and efforts to control these large differences in human blood levels, the exact cause of these variations remains undetermined, making it impossible to predict individual responses to the drug. Some individuals may have exceeded the safety limit, while others might have fallen below the target level, resulting in no clinical benefit.
Originally discovered through research led by St. Jude Children's Research Hospital in Memphis, Tennessee, CoA Therapeutics will return ownership of BBP-671 and related compounds to St. Jude. All their research data will also be shared to support any future research.
We are immensely grateful for the long-standing partnership with CoA Therapeutics. Their dedication to the NBIA community will not be forgotten. We appreciate their passion and desire to find a treatment for our PKAN families, and the amount of empathy and determination demonstrated year after year on behalf of our families.
CoA Therapeutics has released a detailed official announcement that you can read here along with their heartfelt message to the community
