Audent Therapeutics starts dosing patients for rare pediatric disease Crigler-Najjar Syndrome, as it tests recently fast tracked AT342

Audentes Therapeutics has started with dosing of patients in a Phase 1/2 clinical trial of its product candidate AT342 for the treatment of Crigler-Najjar Syndrome.

A biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, has named the study, VALENS. It is a multicenter, multinational, open-label, randomized, ascending dose study designed to evaluate the safety and efficacy of AT342 in approximately 12 Crigler-Najjar patients one year of age and older.

Audentes expects preliminary data from VALENS to be available in the second quarter of 2018.  In addition, FDA has granted Rare Pediatric Disease and Fast Track designations for AT342.

Suyash Prasad, Senior Vice President and Chief Medical Officer said that there are no approved products to treat Crigler-Najjar, a devastating rare disease known for severe jaundice, extremely high levels of unconjugated bilirubin in the blood, and the consequent risk of irreversible neurological damage and death. Prasad noted that the current standard of care requires most patients to spend more than 10 to 12 hours per day under phototherapy lights. Furthermore, Prasad said that natural history data from the company’s LUSTRO run-in study shows that even with this burdensome treatment, bilirubin may only be reduced to levels just below those considered to be neurotoxic.

Matthew R. Patterson, President and Chief Executive Officer added that the fst track and rare pediatric disease designations provide significant benefits, including opportunities to work with the FDA to expedite the development of AT342, and the potential to obtain a valuable Rare Pediatric Disease Priority Review Voucher upon approval. “We are eager to leverage these benefits and to work closely with the FDA and Crigler-Najjar patient community as we advance the development of AT342,” said Patterson.