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Ophthotech gives update on retinal disease pipeline

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With eight products in its pipeline, Ophthotech is aiming to lead the gene therapy front for retinal diseases, Glenn P. Sblendorio, MBA, the company’s CEO and president, said at the 37th annual J.P. Morgan Healthcare Conference in San Francisco.

“Our goal as a company is to become a leader in developing gene therapies and novel therapeutics for the treatment of retinal diseases,” he said.

Over the past year, Ophthotech has expended its pipeline from one product to eight and has added industry experts to its board, including former Santen CEO Adrienne L. Graves, PhD, former Voyager Therapeutics Chief Financial Officer Jane Pritchett Henderson and Bausch Health Eye Care Chief Medical Officer Calvin W. Roberts, MD.

Zimura (avacincaptad pegol), Ophthotech’s lead product candidate, is currently in phase 2b clinical trials for geographic atrophy secondary to dry age-related macular degeneration, with topline data expected in the fourth quarter of 2019. Another phase 2b trial of Zimura, for the treatment of Stargardt disease, is currently enrolling, with topline data expected in the second half of 2020, according to Sblendorio.

The company is working with three research universities for its gene therapy programs: the University of Pennsylvania, the University of Florida and the University of Massachusetts Medical School.

“As we made a decision to get into gene therapy, it was really important, since we don’t have research labs, to work with leading scientific coordinators,” Sblendorio said.

A phase 1/2 trial is planned for 2020 for a treatment for rhodopsin-mediated autosomal dominant retinitis pigmentosa, which knocks down and replaces mutated rhodopsin proteins with healthy rhodopsin proteins and has shown proof of concept in animal models.

Additionally, a treatment candidate for Best vitelliform macular dystrophy will see a phase 1/2 trial initiated in 2021, according to Sblendorio.

Minigene programs for Leber congenital amaurosis and autosomal recessive Stargardt disease are currently in the research stage of development.

“This 2019, with a setup from 2018, will be a year we focus on execution and value creation,” Sblendorio said. – by Rebecca L. Forand

Reference:

Sblendorio GP. Ophthotech. Presented at: 37th annual J.P. Morgan Healthcare Conference; Jan. 7-10, 2019; San Francisco.