Opus Genetics has received the rights to two pre-clinical gene therapy candidates from Iveric Bio.
I need some background on Opus Genetics.
Opus Genetics is a clinical-stage gene therapy company funded by the Retinal Degeneration Fund, Foundation Fighting Blindness’s venture arm. Focused on addressing the unmet needs found in the inherited retinal diseases (IRDs) space, Opus features an adeno-associated virus (AAV)-based gene therapy portfolio that is positioned to leverage novel orphan manufacturing scale and efficiencies.
The FDA cleared its investigational new drug (IND) application in December for a phase 1/2 first-in-human trial of OPGx-001 for Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene in early 2023.
So what are the candidates?
Opus will be developing novel gene therapy candidates to treat bestrophin-1 (BEST1)-related IRDs and rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP).
Tell me about BEST1 and RHO-adRP.
BEST1-related genes affect approximately 5,000 in the U.S. The BEST1 gene therapy delivers a functional copy of the BEST1 gene to retinal pigment epithelial (RPE) cells to create BEST1 protein and stabilize homeostasis between RPE cells and photoreceptors.
One of the most common IRDs, RHO-adRP affects over 6,000 people in the U.S. (via)
What are Opus’s plans for them?
Opus expects to complete further IND-enabling studies as well as filing for an investigational new drug (IND) application for BEST1 by the second half of 2023.
What else does this deal include?
Opus will be taking over global research, development, and commercialization of the BEST1 and RHO-adRP programs. Iveric Bio received $500,000 upfront, a “high single-digit percentage” ownership stake in Opus, and is eligible to receive development and regulatory milestone payments, sales milestone payments, as well as a low single-digit earn out on net sales of the products.
Further, the company is retaining certain rights to potential future commercialization of BEST1 and/or RHO-adRP gene therapy products in certain circumstances. (via)
Why is it significant?
Opus’s CEO, Ben Yerxa, PhD, stated that the company has a goal of submitting at least one IND application per year. By expanding on Opus’s existing gene therapy pipeline to potentially treat multiple IRDs, this deal greatly increases its target patient population and the potential to create sustainable long-term growth from an infrastructure, scientific, and operational perspective.