By Patricia Wood
San Francisco-based CoA Therapeutics reports that it has successfully completed the Phase I study of healthy volunteers for a potential drug compound for Pantothenate Kinase-Associated Neurodegeneration (PKAN).
The Phase 1 trial of BBP 671 examined the safety, tolerability and effects of the drug on healthy volunteers, such as absorption rate, effect on metabolism, how it is excreted and how it is distributed throughout the body. The goal was to not only assess the drug’s safety but to determine a suitable dose and identify any potential problems before advancing to testing in PKAN individuals.
The company will present its findings at the 4th Pan American Parkinson’s Disease and Movement Disorders Congress, May 26 to 28, in Miami, Florida. CoA Therapeutics also will share study results later this year with our community, in addition to informing other scientists, clinicians and companies working on PKAN therapies.
The NBIA Disorders Association and several other NBIA patient organizations helped CoA Therapeutics develop an anonymous survey of PKAN individuals for assessing the compound’s effects when it moves to testing in affected individuals. Survey participants gave feedback on cognitive abilities, physical limitations, performance of daily activities and impact of travel. In all, 183 surveys from 23 countries were completed. Based on that feedback, the company decided to use the drug in small tablet form for the PKAN trial. (You can read a summary of the survey results here).
CoA Therapeutics says the company is focused on two activities this year: improving the formulation of compound BBP-671 and getting feedback on the clinical trial design from regulators in the European Union (European Medicines Agency) and the United States (Food and Drug Administration). Company officials will meet with regulatory authorities later this year and hope to have clinical sites open in the US and Europe by mid-2023. Anna Wade, vice-president of operations said, “We appreciate that time is of the essence for individuals with PKAN and their families, and we are moving as fast as possible.”