BridgeBio Pharma has entered into an exclusive licensing agreement with NeuroVive Pharmaceutical AB for the development of a subset of chemical compounds under NeuroVive’s NVP015 program. The goal is to further develop therapeutic solutions for mitochondrial diseases.
BridgeBio also launched Fortify Therapeutics, a subsidiary that will work on the development of candidate therapies based on NV015 technology for localized treatment of Leber’s hereditary optic neuropathy (LHON). BridgeBio has committed $20 million to the new effort.
“As a targeted treatment for a genetic disease, the LHON program is a clear fit with the BridgeBio model,” Neil Kumar, PhD, CEO of BridgeBio, said in a news release. “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.”
LHON is caused by mitochondrial complex I dysfunction. This describes a situation in which energy conversion in the first of five protein complexes in the mitochondria fails to function normally in retinal cells. These light-sensitive cells comprise the layer at the back of the eye and send signals to the brain that forms a visual image of what a person sees.
Because of this dysfunction, retinal cells do not produce enough cell energy, resulting in a sudden onset of progressive and severe vision loss.
Research has shown that delivering succinate — a chemical compound that activates complex II within the mitochondria energy production chain — in cells with mitochondrial complex I deficiency rescues the cell’s ability to produce energy.
The NVP015 program enables succinate delivery in the cell via a prodrug technology. A prodrug is an inactive drug that is first activated when it enters the body by transforming its chemical structure.
Fortify Therapeutics will use the NVP015 succinate prodrug to develop previously selected lead compounds into therapy candidates for targeted treatment of LHON. The selected compounds are suitable for delivery to the eye.
The agreement outlines limited initial funding for research, and milestone payments later, plus a single-digit royalty stream dependent on successful therapy development and market approval. The contract’s value is about $60 million.
“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,” NeuroVive CEO Erik Kinnman said.
“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,” he added.
NeuroVive’s NVP015 program will proceed without changes in focus for other mitochondrial diseases.