BioPharma

With small RetroSense deal, Allergan hoping for big win

Allergan paid $60 million to buy gene therapy firm RetroSense, which treats retinitis pigmentosa, but given the company’s treatment has orphan drug designation, the payout could be big.

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Allergan’s recent deal for gene therapy company RetroSense Therapeutics, based in Ann Arbor, Michigan, could be a nice win for the big biotech.

For $60 million, and potential milestone payments down the line, Allergan picks up the developmental retinitis pigmentosa (RP) therapy RST-001, which has Orphan Drug Designation and is currently in a phase I/IIa trial.

RP is a group of congenital retinal diseases driven by mutations in the rhodopsin gene. The disease initially impacts retinal cells called rods, which provide peripheral and night vision. As the rods degenerate, neighboring cones, which allow us to see color and detail, also start dying. Many patients are legally blind by the time they are 40, and there are virtually no effective treatments. Roughly 100,000 Americans suffer from RP.

To treat RP, RST-001 takes an optogenetic approach, bringing the channelrhodopsin-2 (ChR2) gene to retinal ganglion to give these cells light-sensing abilities. This approach may also benefit patients with dry macular degeneration.

“From a gene therapy standpoint, it makes a lot of sense,” said Amit Dhawan, medical director at Kantar Health. “You have a specific issue, you lose light sensitivity in the retina, you have a specific type of cells, rods and cones, and you have a specific gene.

The deal shines a spotlight on gene therapy, which is experiencing a renaissance after some early, high-profile failures. Glybera made by uniQure, Amsterdam, Netherlands, which treats lipoprotein lipase deficiency or the inability to break down fatty acids, has been approved in Europe. Others are in the pipeline.

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“Gene therapy continues to come of age,” Dhawan said. “You’re going to see it in these rare diseases that are well-defined and targeted to a specific area.”

The deal augments Allergan’s significant eye care business and is the most recent entry in an ongoing buying and selling spree. In August, they acquired another ophthalmic biotech, ForSight VISION5. They also completed the sale of their generics business to TEVA Pharmaceutical.

RST-001 is still in early stage trials, and there’s no data yet on safety or efficacy. The drug will probably take around five years to get to market, which was likely priced into the deal. In addition, Dhawan notes that the treatment could end up being quite expensive and payers may need to develop new reimbursement models. However, given the unmet medical need and the potential reward, this may be a good risk for the company.

“This is a very good fit for Allergan,” Dhawan declared. “They’re paying a little bit of money for a potentially big win.”

Photo: appleuzr, Getty Images

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