The IND is now active and Audentes plans to initiate VALENS, the multicenter, multinational, open-label, ascending dose phase 1 / 2 clinical study of AT342. Preliminary data from VALENS is expected to be available by the end of 2017.
Audentes Therapeutics president and CEO Matthew R. Patterson said: "We are pleased to achieve this important milestone and look forward to working with regulatory authorities and the medical and patient communities to evaluate AT342 for the treatment of this serious, life-threatening disease.
"Based on our thorough preclinical investigation, we believe that AT342 has the potential to provide a significant and durable reduction in serum bilirubin levels, to reduce or eliminate the need for daily phototherapy treatment, to decrease the risk of devastating neurological injury, and to significantly improve the quality of life for Crigler-Najjar patients and their families."
In addition to VALENS, Audentes plans to imminently begin LUSTRO, a clinical assessment and run-in study. LUSTRO is designed to enroll 16 to 18 Crigler-Najjar patients greater than one year of age and to characterize their disease course, natural history, bilirubin variability and phototherapy usage.
The study is intended to identify patients for potential enrollment in VALENS and to serve as a within-patient control for VALENS.
AT342 has been developed in collaboration with the University of Pennsylvania (Penn) under the leadership of James M. Wilson, M.D., Ph.D., a pioneer in human gene therapy and the director of the Gene Therapy Program and the Orphan Disease Center at the Perelman School of Medicine.
As part of the collaboration, Penn has licensed certain Penn-owned AAV technologies to Audentes, including rights related to AT342.