Progression in Complement Inhibitors, Retina Gene Therapies

May 3, 2022
Kevin Kunzmann

Veeral S. Sheth, MD, MBA, discusses promising agents in development for geographic atrophy and other under-addressed ophthalmic diseases.

Recent news in retina pharmacotherapy have reflected an introduction of long-sought innovation to highly in-need patient populations.

In the last 6 months, the US Food and Drug Administration (FDA) has granted landmark approvals to agents including ranibizumab delivered via Port Delivery System (PDS)—a novel implantation device that simplifies the administration of leading-class anti-VEGF therapy for patients with age-related macular degeneration (AMD). They also approved faricimab, a first-of-its-kind intravitreal bispecific biologic therapy—for treating both AMD and diabetic macular edema (DME).

While these indications reflect the sought-after future of ophthalmic care, they do not embody the entirety of potential borne from current research.

In an interview with HCPLive during the Association for Research in Vision and Ophthalmology (ARVO) 2022 Meeting this year, Veeral S. Sheth, MD, MBA, Director of Clinical Research at the University of Retina and Macula Associates, discussed the state of landmark research into another pair of drug class prospects: complement factor inhibitors for geographic atrophy (GA), and gene therapies for a litany of ophthalmic conditions.

On the subject of the prospective first GA drug class, Sheth highlighted a couple of complement inhibitor agents—avacincaptad pegol and pegcetacoplan—that look viable for regulatory decision in the near future.

“We’re getting closer and closer to things like Apellis’ molecule and IVERIC bio’s molecule taking that next step further and hopefully getting FDA approval so that we’ve got access to these for our patients,” Sheth said. “I think there’s exciting things in the pipeline if you look at some of these gene therapies for GA—and I think a lot of these are related to complement factor inhibition, but there’s a lot of other avenues as well.”

Regarding gene therapies, Sheth discussed later-stage advances from REGENXBIO with agents that could potentially reverse the effect of wet AMD in key patients, as well as Gyroscope Therapeutics—recently acquired by Novartis—in GA reversal.

“I think for us, anything that’s a little further along is exciting,” Sheth explained.
“Because I think gene therapy is still in that period of strong development—every quarter, we’re getting a new milestone from one of these companies. You’re seeing a really good uptick in their studies, and hopefully that all pans out. Because in the GA study, for example, it’s an unmet need—but also an opportunity to reduce treatment burden right off the bat.”

Sheth also discussed come of the parameters that would go into implementing novel gene therapies into ophthalmic care, such as the level of regulation that would go into conducting procedures—or what to expect in follow-up care with long-term patients.

“From the front end: are surgeons going to be able to do these procedures from their surgical centers?,” Sheth suggested. “And on the back end: what happens year 3, year 4, year 5? I think we’re still so early in the process we don’t know exactly yet. We’re starting to get data that gives us a better idea, but that’s always going to be something (clinicians) think about.”


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