Neurophth Secures $95 Million for Gene Therapy Clinical Trials

Rare Daily Staff

Neurophth Therapeutics, a Chinese gene therapy company focused on genetic ophthalmic diseases, raised nearly $95 million in a series C+ financing to advance clinical trials and expand its R&D capabilities and pipeline.

Yangtze River-CMB International Industry Fund, Wuhan Optical Valley Financial Holding Group, Wuhan Hi-Tech Holding Group, Hubei KTLC, and Guangzhou Jinkong Fund led the financing with participation by iCMG-SDIC Capital, Silicon Paradise Asset Management, and Yangtze River Industry Fund.

“Since the beginning of this year, we’ve made substantial progress in the clinical trials of our products,” said Bin Li, founder, chairman, and CEO of Neurophth. “We plan to keep focusing on developing gene therapies for ophthalmic patients worldwide. With the support of our investors and our technological acumen, we’re positioned to accelerate our clinical trials and R&D of new drugs, delivering gene therapies to patients more quickly.”

In early 2023, Neurophth’s leading candidate NR082 (rAAV2-ND4), in development for the treatment of ND4-mediated Leber hereditary optic neuropathy, marked a historic milestone as the first gene therapy drug to complete patient enrollment for a phase 3 clinical trial in China. Additionally, the company concluded the enrollment of the first patient for phase 1/2 clinical trials in the United States. NFS-02 is Neurophth’s second candidate and the only gene therapy targeting ND1-LHON under development that has received IND approval from both the China National Medical Products Administration (NMPA) and the U.S. Food and Drug Administration (FDA). Currently, Neurophth is conducting a global multi-center clinical trial of the drug candidate.

LHON is caused by defects in mitochondrial genes encoding for proteins called NADH dehydrogenase. About 95 percent of LHON cases are due to mutations in the mitochondrial genes ND1, ND4 and ND6, which encode proteins of the respiratory chain Complex I, an important component of energy production within the mitochondrion and therefore the cell. The ND4 mutation is responsible for the majority of LHON cases, representing around 70 percent in Europe and North America and 80 to 85 percent in Asia. Onset of vision loss due to LHON typically occurs between 15 and 35 years of age. Vision loss often occurs in a sequential fashion where one eye is affected first, followed by the second eye on average two months later.

Neurophth’s most advanced investigational gene therapy drug candidate, NR082, for the treatment of Leber’s hereditary optic neuropathy (LHON) associated with mtND4 mutation (ND4-LHON), has been granted orphan drug designation by the U.S. FDA and the European Medicines Agency (EMA).

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