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Neurophth Therapeutics completes patient enrollment in phase I/II clinical trial of esonadogene mvoparvovec


The trial of esonadogene mvoparvovec (Opvika) will take place in the US for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON).

(Image Credit: AdobeStock/natali_mis)

(Image Credit: AdobeStock/natali_mis)

Neurophth Therapeutics announced the final patient has been enrolled in their phase I/II clinical trial of esonadogene mvoparvovec (NR082;Opvika) for the treatment of Leber hereditary optic neuropathy caused by ND4 mutation (ND4-LHON).

According to Neurophth,1 the trial will be a Phase I/II, single-arm, multi-center study evaluating the safety and efficacy of esonadogene mvoparvovec in LHON patients with ND4 mutations and be conducted in the US.

This comes after the FDA approved the Investigational New Drug (IND) application of esonadogene mvoparvovec in January 2022.

"We are extremely grateful for the trust and support of patients and their families, as well as the dedication of all researchers. They have played a crucial role in the successful completion of patient enrollment for the Phase I/II clinical trial," said Bin Li, MD, PhD, founder, chairman, and CEO of Neurophth. "Neurophth has taken a solid step forward in going global. As our motto is "In China, for global", we will strive to bring China's medical development and more innovative gene therapies to patients worldwide."

LHON is a maternally inherited mitochondrial disease that mostly affects young men between the ages of 14 and 21 with binocular vision impairment and blindness. According to Neurophth, the therapy works by “using recombinant adeno-associated virus as the vector to deliver the correct genes to the patients' damaged optic ganglion cells through introvitinreous injection, thus repair the mitochondrial biological respiratory chain, and restore the vitality and visual function of optic ganglion cells.”2

Aside from the phase I/II trial in the US, Neurophth is in a phase 3 trial of the therapy in China.2

  1. Neurophth Announces Completion of Patient Enrollment for Opvika® Phase I/II Clinical Trial in the U.S. Press release; February 19, 2024. Accessed February 20, 2024. https://www.prnewswire.com/news-releases/neurophth-announces-completion-of-patient-enrollment-for-opvika-phase-iii-clinical-trial-in-the-us-302064859.html
  2. Neurophth Pipeline. Accessed February 20, 2024. https://www.neurophth.com/en/ProjectD-65.html
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