RESEARCH TRIANGLE PARK, N.C. — Opus Genetics, Inc. (Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced that its investigational LCA5 gene therapy program, OPGx-LCA5, has been accepted into the U.S. Food and Drug Administration’s (FDA) Rare Disease Evidence Principles (RDEP) program.
OPGx-LCA5 is a potential gene therapy for Leber congenital amaurosis type 5 (LCA5), a rare inherited retinal disease caused by mutations in the LCA5 gene. The condition leads to early-onset, progressive vision loss and often results in severe visual impairment or blindness in childhood. There are currently no approved therapies specifically targeting LCA5.
RDEP is a new FDA initiative intended to support the development of therapies for ultra-rare genetic diseases typically affecting fewer than 1,000 patients in the U.S. The program enables early and ongoing collaboration between the FDA and sponsors to align on regulatory strategy, clinical trial design, and innovative approaches to generating evidence needed to support potential approval.
“RDEP eligibility represents an important element of our regulatory strategy as we seek alignment with the FDA on our pivotal Phase 3 program for OPGx-LCA5,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “Given the rarity and severity of this disease, early engagement with the FDA alongside our RMAT designation will help inform a more efficient and streamlined development pathway. We look forward to collaborating with the FDA as we pursue a potential treatment option for patients affected by this devastating inherited retinal disease.”
As part of the RDEP program, the FDA will work closely with Opus Genetics to guide the ongoing development of OPGx-LCA5, including considerations for clinical trial design, approaches to generating efficacy in a small patient population, and strategies to support demonstration of clinical benefit. The program also provides a framework for evaluating substantial evidence of effectiveness, including the potential use of a single adequate and well-controlled study supported by confirmatory evidence.
About OPGx-LCA5
OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania. Data from pediatric participants demonstrated large gains in cone-mediated vision, and the therapy remains well tolerated with no ocular serious adverse events or dose-limiting toxicities. The adult cohort showed durable improvements in cone sensitivity and visual function out to 18 months. OPGx-LCA5 has also received Rare Pediatric Disease, Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA.
About Opus Genetics
Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs). The Company is developing durable, one-time treatments designed to address the underlying genetic causes of severe retinal disorders. The Company’s pipeline includes seven AAV-based programs, led by OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration, with additional candidates targeting RDH12, MERTK, RHO, CNGB1 and NMNAT1. Opus Genetics is also advancing a small-molecule therapy, Phentolamine Ophthalmic Solution 0.75%, beyond its approved use for pharmacologically induced mydriasis, with a supplemental new drug application under review for presbyopia and an ongoing Phase 3 pivotal trial for mesopic, low contrast conditions after keratorefractive surgery (dim light disturbances). The Company is based in Research Triangle Park, NC. For more information, visit www.opusgtx.com.
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