Allergan snaps up gene therapy startup to restore vision in rare disease patients

Alleran

Allergan ($AGN) has bought gene therapy startup RetroSense Therapeutics to gain an early clinical candidate to treat the rare vision disorder retinitis pigmentosa (RP). The aim is to use this optogenetic approach to actually reverse vision deterioration in these patients.

The biopharma paid $60 million up front, with additional, undisclosed regulatory and commercialization milestones tied to the lead program, RST-001 to treat RP. This is the biopharma’s second recent deal for a novel ophthalmic technology--and part of a larger effort to advance its R&D after last year announcing it would sell its generics business to Teva ($TEVA).

"The acquisition of RetroSense and its RST-001 program builds on Allergan's deep commitment to eye care, and our focus on investing in game-changing innovation for retinal conditions, including retinitis pigmentosa, where patients desperately need treatment options," Allergan President and CEO Brent Saunders said in a statement.

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Allergan has a huge presence in ophthalmic treatments. In fact, it’s by far the company’s largest business with $636 million in revenues during the second quarter, which was an increase of 10% over the same quarter a year earlier. But the bulk of its established business is in eye drops to relieve conditions such as dry eye and glaucoma.

“The areas that we're active in in ophthalmology, are dry eye, of course, glaucoma, and increasingly, we're looking at new approaches for the retina,” summed up Allergan EVP and Branded Pharma President William Meury at an investor R&D day late last year.

RST-001 is certainly one such new approach. It’s a first-in-class gene therapy approach that is expected to introduce sensitivity to light in cells that were previously insensitive to it. In retinas in which rod and cone photoreceptors have degenerated, the tech is expected to introduce light sensitivity. It’s based on a photosensitivity gene, channelrhodopsin-2, and is designed to create new photosensors in retinal ganglion cells in an effort to restore vision to RP patients.

The candidate has Orphan Drug Designation and is in a Phase I/IIa trial in which the low-dose of patients have been safely dosed, the company said. There are about 100,000 people in the U.S. with the disorder.

"The RST-001 program and its optogenetic gene therapy approach could be a real breakthrough in the treatment of unmet needs across a host of retinal conditions, including RP," said Allergan Chief R&D Officer David Nicholson.

Allergan is looking to expand its foothold in ophthalmology and has been doing a series of deals to ensure that it’s able to do so with much of its recent activity focused on ophthalmic devices in particular.

Last month, it bought ForSight Vision5 for $95 million up front and a launch milestone for its clinical-stage periocular ring that’s designed to continuously release the drug bimatoprost over several months to lower eye pressure in glaucoma and ocular hypertensive patients.

In 2015, Allergan bought glaucoma microshunt company AqueSys for $300 million up front plus milestones and dry eye device tartup Oculeve for $125 million plus milestones.

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