Opus Genetics Acquires Two Gene Therapy Candidates for Treating Blindness
Opus Genetics, a clinical-stage gene therapy company in Research Triangle Park devoted to treating retinal diseases, has acquired the rights to two pre-clinical product candidates from Iveric Bio, a New Jersey biopharmaceutical company also focused on treating retinal diseases.
Opus will develop the novel gene therapy candidates to address bestrophin-1 (BEST1)-related inherited retinal diseases and rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP).
“The addition of these innovative BEST1 and RHO-adRP programs significantly increases the patient population that could benefit from Opus therapies and complements our existing pipeline of gene therapies for inherited retinal diseases,” said Ben Yerxa, Ph.D., chief executive officer of Opus and a board member of the North Carolina Biotechnology Center.
“Opus is building an engine for addressing multiple inherited retinal diseases, and deals like this one help us achieve our desire for sustainable growth of our infrastructure, operations, scientific expertise and ultimately our patient impact, with the goal of at least one IND (investigational new drug application) per year,” Yerxa said.
Opus anticipates filing an IND application for BEST1 in the second half of 2023.
The BEST1 gene therapy is designed to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells so the cells can produce bestrophin-1 protein, critical to vision.
BEST1-related diseases are estimated to affect about one in 69,000 people, or nearly 5,000 people, in the United States.
RHO-adRP is one of the most common inherited retinal diseases, estimated to affect about one in 51,000 people, or more than 6,000 people, in the United States.
Terms of the deal
As part of the deal with Iveric Bio, Opus will assume responsibility for the global research, development and commercialization of the BEST1 and RHO-adRP programs. In exchange, Iveric received an upfront payment of $500,000 and a “high single-digit percentage” ownership stake in Opus.
Iveric is also eligible to receive development and regulatory milestone payments, sales milestone payments, and a low single-digit earn out on net sales of the products. Iveric retains certain rights with respect to the potential future commercialization of gene therapy products for BEST1 and/or RHO-adRP under certain circumstances.
“Affecting over 28,000 people across the U.S., EU and UK, BEST1 and RHO-adRP represent a significant portion of all inherited retinal diseases and an urgent unmet need for effective treatment,” said Bart P Leroy, M.D., Ph.D., head of the Department of Ophthalmology, professor of ophthalmology and member of the Center for Medical Genetics at Ghent University and Ghent University Hospital in Belgium. He is also an attending physician in the Division of Ophthalmology and The Raymond G. Perelman Center for Cellular and Molecular Therapeutics at the Children’s Hospital of Philadelphia.
Inherited retinal diseases “are ideal targets for genetic therapies to stop the retinal degeneration and improve the lives of patients living with severe vision loss or blindness,” Leroy said.
Such diseases account for up to 20% of all blindness in individuals aged 16-64. They affect as many as 430,000 patients in the United States and an estimated 5.5 million patients worldwide, according to Opus.
Raising Series A funding
Opus was founded in January 2021 and has 19 employees. It is backed by the Retinal Degeneration Fund, the venture capital arm of Foundation Fighting Blindness, a non-profit foundation set up in 1971 to find treatments and cures for inherited retinal diseases. The foundation is the world’s leading private funder of retinal disease research.
Opus is operating with $19 million in seed financing led by the Retinal Disease Fund with participation from the Manning Family Foundation and BIOS Partners. It is now working to raise $35 million in Series A funding.
Opus’ gene therapy approach uses adeno-associated viruses (AAV) to ferry functional genes into cells that have genetic mutations responsible for inherited retinal diseases. More than 280 genes to date have been associated with these diseases.
The AAV technology is based on the work of Opus’ scientific founders, Jean Bennett, M.D., Ph.D., of the University of Pennsylvania, and Eric Pierce, M.D., Ph,D., of Harvard Medical School and Massachusetts Eye and Ear. The technology addresses inherited retinal diseases that have no approved treatment.
First program to enter clinic soon
In December the U.S. Food and Drug Administration cleared Opus’ Investigational New Drug application for a Phase 1/2, first-in-human clinical trial of OPGx-001 in patients with Leber congenital amaurosis resulting from mutations in the LCA5 gene.
OPGx-001 is an AAV vector designed to deliver a functional LCA5 gene to retinal photoreceptors.
“We founded Opus a little more than a year ago to quickly move promising potential treatments into the clinic for patients in need,” Yerxa said. “This FDA clearance of our IND application for OPGx-001 for LCA5 marks a significant milestone for Opus, as our first program to enter the clinic.”
Preclinical studies in models of LCA5 have provided support for the safety and efficacy of OPGx-001. Opus will begin its first-in-human trial of OPGx-001 in early 2023.
The Phase 1/2, open-label, dose-escalation trial will evaluate the subretinal delivery of OPGx-001 in nine adult patients with LCA5. The objective is to evaluate safety and potential benefit.
Once safety in adults has been cleared, Opus plans to add a pediatric cohort.
Manufacturing partner selected
In April, Opus entered into a strategic manufacturing services agreement with National Resilience, a technology-focused biomanufacturing company dedicated to broadening access to complex medicines, to support the development and manufacturing of Opus’ gene therapies.
Resilience will provide process and analytical development, quality control testing, and good manufacturing practices services for IND-enabling toxicology and first-in-human material for Opus’ AAV vector-based gene therapies for inherited retinal diseases for use in both preclinical studies, and future clinical trials in the U.S. The work will be conducted at Resilience’s facilities in Waltham, Mass., and Research Triangle Park.
“Resilience embraces our innovative model to create a clinical manufacturing infrastructure that’s scaled to address rare inherited retinal diseases, said Yerxa. “Leveraging Resilience’s expertise puts Opus in the best position to efficiently advance our AAV-based gene therapies into the clinic and toward the patients who need them.”