News from CoA-Therapeutics: Phase 1 trial with new drug for PKAN has started

The latest news from CoA Therapeutics, Inc., a subsidiary of BridgeBio Pharma, Inc. is that the Californian company is working on a new potential therapy for PKAN patients. "CoA" stands for coenzyme A and the company's name already indicates that it specializes in developing drugs to treat diseases associated with coenzyme A deficiency, such as PKAN patients. Now CoA Therapeutics has informed us about the current status of its PKAN research program:

The necessary preclinical animal studies with the small molecule drug candidate BBP-671 have been completed and the US Food and Drug Administration (FDA) has approved CoA Therapeutics' application to use the investigational new drug (IND) in humans. In April 2021, a Phase 1 trial was therefore launched to test the safety of BBP-671 in healthy volunteers before it is tested in PKAN patients. The CoA Therapeutics team plans to begin clinical trials in PKAN patients in late 2022, once it has been established that the drug candidate is safe and a suitable dose for PKAN patients has been determined.

The company plans to test its drug on PKAN patients worldwide. In the US, BBP-671 has already received orphan drug designation for rare diseases and also for rare pediatric diseases. In the European Union, BBP-671 was recently granted orphan drug designation by the European Medicines Agency (EMA). The CoA Therapeutics team is committed to advancing this potential therapy for PKAN patients as quickly and safely as possible. To learn more about CoA Therapeutics, please visit the company's website: https://coatherapeutics.com/

Hoffnungsbaum e.V. will continue to keep PKAN families informed of any new developments regarding this planned study.