Novartis-backed GenSight gets cash for gene therapy launch

GenSight Biologics has raised (PDF) €22.5 million ($25.2 million) to prepare to bring gene therapy GS010 to market in the U.S. and Europe. The financing gives the Novartis-backed biotech enough cash to deliver data from two phase 3 trials next year and gear up for anticipated approvals on both sides of the Atlantic.
Paris-based GenSight raised the cash from a mix of new and existing institutional investors, most of which are based in the U.S. Strong interest from these backers saw GenSight ease past its initial target of €20 million to pull in €22.5 million in the private placement. When added to the €48.8 million GenSight had in the bank at the end of March, management thinks the money moves its runway out to the first quarter of 2019. 
That runway covers a critical period for GenSight. Topline 48-week data from two phase 3 trials of GS010 in patients with Leber hereditary optic neuropathy (LHON) are due in the second and third quarters of next year.
GenSight is looking to the trials for evidence GS010 improves the clarity of the vision of patients with LHON, a hereditary form of vision loss caused by mitochondrial defects. GS010 is injected into the eye to deliver the human wild-type ND4 gene via an adeno-associated virus to deliver. This gene encodes for a protein typically produced by mitochondria. 
One trial is assessing GS010 in patients who started losing their vision in the six months prior to enrolling in the study. The other is recruiting patients whose vision started deteriorating between seven and 12 months ago. Both trials are injecting GS010 into one eye of each participant and pretending to inject it into the other eye.
Data from an earlier phase 1/2 trial suggest the gene therapy is most effective in patients whose vision started deteriorating less than two years ago. A recent 96-week update found the treated eyes of such patients had a mean gain of 29 ETDRS letters, as compared to an increase of 15 letters in untreated eyes. ETDRS is the test showing progressively smaller letters opticians use to gauge vision. 
The performance of GS010 to date has enabled GenSight to secure the support of some big-name backers. Following the latest financing, its biggest shareholders are Novartis, Versant, Abingworth and Fidelity.
GenSight tried to turn this transatlantic support into a Nasdaq listing in 2015, but it was forced to downsize and ultimately scrap the plan in the face of an unreceptive market. GenSight was part of a clutch of European biotechs that arrived on Wall Street as the IPO window was closing. And like others from that group, including Basilea Pharmaceutica and Bavarian Nordic, it has so far held off on trying again in favor of raising cash from other sources.