As a biopharmaceutical company focused on developing transformative therapies for retinal diseases, Iveric Bio aims to lead with the strength of its science. The company’s lead pipeline asset, avacincaptad pegol, is a pegylated RNA aptamer that specifically inhibits complement factor 5 (C5)1, and is currently in development for the potential treatment of geographic atrophy (GA). Inhibiting C5 preserves crucial physiologic function initiated at earlier steps in the complement pathway, while still shutting down activation of inflammatory processes and cell-killing membrane attack complex.2,3
There are no currently approved treatments for GA, a debilitating disease that severely impacts patients, limiting their ability to drive, read, and see the faces of friends and family. In September 2022, Iveric announced that GATHER2, its second phase 3 clinical trial for avacincaptad pegol, had achieved the pre-specified 12-month primary endpoint of mean rate of growth in GA with a high degree of statistical significance. With these findings and the positive results from GATHER1, the company’s first phase 3 trial of avacincaptad pegol in GA, Iveric achieved something that has never been done before in GA — deliver two phase 3 studies that met their pre-specified primary endpoints at 12 months of slowing GA progression.
"In September 2022, Iveric announced that GATHER2, its second phase 3 clinical trial for avacincaptad pegol, had achieved the pre-specified 12-month primary endpoint of mean rate of growth in GA with a high degree of statistical significance. With these findings and the positive results from GATHER1, the company’s first phase 3 trial of avacincaptad pegol in GA, Iveric achieved something that has never been done before in GA — deliver two phase 3 studies that met their prespecified primary endpoints at 12 months of slowing GA progression."
In addition, there were no events of endophthalmitis, no intraocular inflammation events, and no ischemic optic neuropathy events through month 12 in GATHER2. The most frequently reported ocular adverse events were related to the injection procedure. The incidence of choroidal neovascularization (CNV) in the study eye through month 12 was 6.7% in the avacincaptad pegol group and 4.1% in the sham control group.
With these results, Iveric is moving forward with its plans to submit a New Drug Application (NDA) to the FDA by the end of the first quarter of 2023.
A Corporate Philosophy in Action
The release of the positive 12-month data from GATHER1 was a bellwether moment for Iveric, and the company started preparing for GATHER2 in earnest. Following a brief pause due to COVID-19, the GATHER2 momentum continued. The data released from GATHER1 also affirmed the philosophy that Chief Executive Officer, Glenn Sblendorio has continually sought to instill in the company’s culture—if Iveric can establish a strong scientific rationale for its pipeline candidates, interest will follow. That idea was further solidified when Pravin U. Dugel, MD, came on board as Chief Business and Strategy Officer in March 2020.
Over the course of his decades-long career as a private-practice retina physician and practice owner, Dr. Dugel had been tracking Iveric and its pipeline. “Iveric is going where nobody’s gone before, and that to me was extraordinarily attractive,” Dr. Dugel said. “This was an opportunity to drive decisions based on science that will serve physicians and their patients. We are a science-driven company focused on patients and retinal diseases for which there is no treatment.”
Dr. Dugel became President of Iveric Bio in May 2021. The company also hired Dhaval Desai, PharmD, as Chief Development Officer and Chris Simms as Chief Commercial Officer. Iveric also has a deeply experienced board of directors, led by current Chairman Adrienne L. Graves, Ph.D.
Looking to the Future
Along with the promise of avacincaptad pegol, Iveric is building a deep portfolio of therapeutic and gene therapy product candidates that broaden the company’s potential to provide treatment options for other devastating retinal conditions with unmet needs. Avacincaptad pegol and IC-500 target two different pathways associated with the development and progression of AMD, complement and HtrA1, respectively. Both therapeutic approaches are backed by genetic association studies, preclinical data, and scientific literature that together provide a potentially comprehensive strategy for the various stages of AMD. The company believes that both assets will pair well together, one being gene agnostic and the other gene specific, and each having a different and distinct mechanism of action. These two assets will form the building blocks of an AMD franchise that Iveric Bio is working to establish.
Beyond that are a suite of adeno-associated virus (AAV)-delivered gene therapy research and development programs. The company is pursuing multiple mini-gene research and development programs (spliced portions of larger genes packaged to fit into standard AAV vectors) for Leber congenital amaurosis type 10, autosomal recessive Stargardt disease, and USH2A-related inherited retinal diseases, such as the retinal degeneration associated with Usher syndrome type 2A.
According to Mr. Sblendorio, having potentially game-changing science is one reason Iveric continues to attract interest in its pipeline. By ensuring that science continues to guide Iveric’s strategy, and the company’s strong leadership steers it forward with integrity, Iveric is well positioned to provide potential therapies and hope for millions of patients with retinal diseases.
Read the related press release on Eyewire+.
1. Jaffe GJ, Westby K, Csaky KG, et al. C5 inhibitor avacincaptad pegol for geographic atrophy due to age-related macular degeneration: a randomized pivotal phase 2/3 trial. Ophthalmology. 2021;128(4):576-586.
2. Kassa E, Ciulla TA, Hussain RM, Dugel PU. Complement inhibition as a therapeutic strategy in retinal disor¬ders. Exp Opin Biol Ther. 2019;19(4):335-342.
3. Xu H, Chen M. Targeting the complement system for the management of retinal inflammatory and degenerative diseases. Eur J Pharmacol. 2016;787:94-104.