Ophthotech (now Iveric) hits eye drug goals as shares jump for tiny biotech

While pivoting to gene therapies, microcap Iveric bio (once Ophthotech) has posted positive data from its key eye drug Zimura (avacincaptad pegol).

The complement factor C5 inhibitor hit its primary endpoint in a phase 2b study by reducing the rate of geographic atrophy (GA) growth in patients with dry age-related macular degeneration (AMD).

The reduction in the mean rate of GA growth over a year was 27.38% (p-value=0.0072) for the Zimura 2 mg group, compared to those taking a dummy treatment.

For the higher dose 4 mg group, this number hit 27.81% (p-value=0.0051), again as compared to the sham control group, hitting, the company said, statistical significance.

The company noted that there were also no ocular serious adverse events and no cases of endophthalmitis, with most issues coming from the injection of the drug itself.

This comes nearly a year after the company decided to focus on GA and dry AMD and away from a Lucentis combo in wet AMD.

“This is a major milestone for Iveric bio and a potentially significant advancement for patients with GA secondary to dry AMD who currently have no treatment options,” stated Glenn Sblendorio, CEO and president of Iveric bio.

“Based on these data, we intend to explore all options for the future development of Zimura, including the possibility for collaboration opportunities, licensing and / or potentially further internal development. I especially want to thank patients who participated in the trial, as well as our team, our collaborators and our shareholders that have supported Iveric bio.”

This also comes after Ophthotech changed its name to Iveric bio as part of its transition into a gene therapy biotech (what everyone seems to be doing when something in the pipeline goes wrong) earlier this year. The rebranding followed a period in which the biotech responded to a phase 3 setback by striking deals for retinal disease gene therapies.

The New York-based company was laid low in late 2016 by the failure of two phase 3 clinical trials of anti-platelet-derived growth factor Fovista in wet AMD.

That led Ophthotech to weigh strategic alternatives before deciding to apply its ophthalmic drug development expertise to the advancement of a portfolio of in-licensed gene therapies.

Iveric’s gene therapy pipeline is led by two preclinical assets, IC-100 and IC-200, that are designed to treat rhodopsin-mediated autosomal dominant retinitis pigmentosa and Best disease. The biotech secured rights to the programs through deals with the University of Florida Research Foundation and the University of Pennsylvania and plans to enter the clinic in 2020 and 2021.

But, while Iveric sees gene therapies as its future, its clinical pipeline remains centered on Zimura.

Shares in the biotech, which has a market cap of less than $40 million, jumped around 100% in premarket trading on the news.