Neurophth Completes Patient Enrollment for Phase III clinical trial for the Gene Therapy treatment of LHON

Neurophth Therapeutics announced today that the last patient has been enrolled in the Phase III gene therapy clinical trial for the treatment of Leber hereditary optic neuropathy (LHON) in China. It marks the completion of patient enrollment of the Phase I/II/III, multi-center, two-parts study aimed at evaluating the safety, tolerability, and efficacy of NR082 in LHON patients with ND4 mutations.

“We are extremely excited to announce the completion of the last patient visit in this Phase III study, which marks a significant advancement for NR082 as well as a minor step toward the commercialization of China’s self-developed ocular gene therapy. It often takes a decade to develop an innovative drug, and Chinese ophthalmic gene therapy drug costs much more than that,” said Professor Bin Li, Founder, Chairman and CEO of Neurophth. “We would like to express our gratitude to all clinical investigators, trial participants and their families for their time and commitment, and also the excellent teamwork of the entire company to complete the Phase III clinical trial within 5 months. We are confident that the company will live up to our promise to commercialize Chinese in vivo gene therapy soon and make it available to patients.”

“The design of the NR082 clinical trial and the selection of the optimal dose were based on multiple successful previous studies and in accordance with the CDE. Neurophth and CRO collaborated closely to overcome the challenges of COVID-19 and complete the enrollment of all patients successfully,” said Dr. Xiaoning Guo, Chief Medical Officer of Neurophth. “We are optimistic about the outcome of this clinical trial. If the study meets the pre-defined endpoints, we will submit a New Drug Application as soon as possible for the early launch of NR082 in China and in the meantime, advance the progress of clinical trial in the U.S. to offer a direly needed treatment for LHON patients.”