Drs. Susan Hayflick, Penny Hogarth and Ody Sibon told PKAN families recently that they are working with two companies to create a PKAN drug to ensure sufficient amounts for a clinical trial.
Speaking with PKAN families via a Facebook live stream video Nov. 6, the researchers said they are hopeful the compound that they are calling CoA-Z will correct a metabolic process involved in producing coenzyme A, called CoA. CoA is involved in metabolism and is thought to be low in individuals with Pantothenate Kinase-Associated Neurodegeneration, the most common form of NBIA. In the PKAN mouse, CoA-Z does everything Hayflick, Hogarth and Sibon would want to see before moving their studies into a human clinical trial.
Hayflick told families at the international family conference in June that CoA-Z was one of two compounds her team considered to be possible treatments for PKAN. The second compound is already approved by the Food and Drug Administration for other purposes, but tests since then have indicated it would not be effective for PKAN.
As a result, Hayflick and Hogarth and their team at the Oregon Health & Science University, along with Sibon’s team in The Netherlands, are now focused on getting CoA-Z to trial. The drug must be made to exacting standards to be sure the final product is pure. Making the compound to these standards and in sufficient quantities for a clinical trial could take six months or more.
Once it’s ready, the researchers plan to ask the FDA to approve a trial that would be open to all people with PKAN and be designed specifically to ease the burden of study participation for families. If the FDA approves of the trial’s design and sufficient funding is secured, the clinical trial would start in North America and then in Europe, Australia, India and many other countries as quickly as possible.
The CoA pathway is the focus of other PKAN-related compounds that either are being tested or are in development. A San Diego-based biopharmaceutical company, Retrophin, is conducting a clinical trial on its PKAN drug, RE-024, and information on enrolling can be found at www.pkanfortstudy.com. CoA-related drugs are being developed by a European company, Acies Bio, and by a team at St. Jude Children’s Research Hospital led by Dr. Suzanne Jackowski and a company in Palo Alto, CoA Therapeutics.