Pharmaceutical Business review

NeuroVive grants LHON therapy license to BridgeBio Pharma

BridgeBio also announced that it has launched a subsidiary company Fortify Therapeutics to further develop this chemistry for local treatment of Leber's Hereditary Optic Neuropathy (LHON), with an initial financial commitment of $20 million USD.

NeuroVive's NVP015 program for other mitochondrial disorders will continue without any changes in focus or timelines.

LHON is caused by mitochondrial DNA mutations in subunits of NADH dehydrogenase (complex I), leading to reduced oxidative phosphorylation and energy production in retinal cells.

The disease predominantly affects young adults, and results in sudden onset of progressive and severe vision loss.

The licensed succinate prodrugs have the potential to overcome the disease by bypassing the dysfunctional metabolic pathway, providing an alternate source of energy to the retinal cells.

BridgeBio CEO Neil Kumar said: "As a targeted treatment for a genetic disease, the LHON program is a clear fit with the BridgeBio model.

"We have been impressed with the ability of these compounds to rescue specific genetic mitochondrial deficiencies, and we have assembled a team of international experts to further develop a subset of the NVP015 chemistry to address this devastating disease."

Fortify Therapeutics will develop selected lead compounds derived from NeuroVive's novel NVP015 succinate prodrug program into drug candidates for the localized treatment of LHON.

These compounds have been selected because they have properties that make them suitable for delivery to the eye.

The licensing agreement for this particular subset of the NVP015 program has a total deal value of approximately $60 million USD, which includes limited initial funding for research, and later milestone payments and a single digit royalty stream, that are dependent on successful development and market approval.

NeuroVive CEO Erik Kinnman said: "The agreement with BridgeBio is important to both NeuroVive and our innovative NVP015 program, as it validates the quality of the program, our business development model and potential in a variety of mitochondrial disorders.

"We will work closely with BridgeBio to further develop this chemistry subset and make the LHON program successful. It is important to note that our intentions for the NVP015 program are unchanged, and we are progressing towards experimental proof-of-principle during 2018."

Source: Company Press Release.