CoA Therapeutics discontinues clinical trial for PKAN

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 had been developing as a potential treatment for pantothenate kinase-associated neurodegeneration (PKAN). This decision was made after studies showed that it was not possible to find a dose for the clinical trial that provided an appropriate balance between 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 important dosing and safety information. A major challenge identified during these additional studies was the wide variability in blood levels of BBP-671 observed in humans. Individuals taking the same dose of BBP-671 had very 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 PKAN animal models helped to establish a specific safety limit or target blood level for BBP-671 that cannot be exceeded due to safety and FDA regulations. It has been shown that the target blood level required for efficacy is dangerously close to the safety limit.

BBP-671 was originally discovered through research at St. Jude Children's Research Hospital in Memphis, Tennessee. CoA Therapeutics will now return ownership of BBP-671 and related compounds to St. Jude. All research data will also be shared to support any future research.

We are eternally grateful for our long-standing partnership with CoA Therapeutics. Their commitment to the NBIA community will not be forgotten. We appreciate their passion and desire to find a treatment for our PKAN families and the level of empathy and determination they have shown year after year on behalf of our families.

CoA Therapeutics has released a detailed official announcement for you to read along with a heartfelt message to the community:

CoA-Therapeutics-PKAN-Patient-Community-Update.pdf

Translation of an article by Amber Denton, President of the NBIA Disorders Association, from the newsletter of the NBIA Disorders Association
https://nbiadisorders.org/news-events/nbia-newsletters/