Opus Genetics NASDAQ: IRD is advancing a portfolio of gene therapies for inherited retinal diseases, with two programs already in the clinic and three more expected to enter clinical testing within the next 12 months, President Dr. Ben Yerxa said during a fireside chat at RBC Capital Markets’ 2026 Global Healthcare Conference.
Yerxa said the company was co-founded by Dr. Jean Bennett, who also co-founded Spark Therapeutics and was an inventor of LUXTURNA. He said Opus has built a seven-asset portfolio focused on inherited retinal diseases, using known capsids and promoters rather than “science projects.”
“We generally try to avoid risk on risk,” Yerxa said, citing the use of AAV2, AAV5, AAV8 and AAV9 capsids. “The agency’s comfortable with them. No science projects. It’s all just like development work.”
Regulatory environment and platform strategy
Asked by RBC biotech analyst Lisa Walter about regulatory volatility at the FDA, Yerxa said Opus has not seen a major change at the project-team level. He said the absence of a permanent head of CBER was “probably a little bit more disruptive” for the company than changes at the top of the FDA, given CBER’s relevance to gene therapy programs.
“For rare disease and gene therapy in our space, it’s been pretty steady and pretty favorable for us,” Yerxa said.
Yerxa said inherited retinal diseases are attractive for gene therapy development because treatment can be delivered directly into the eye through subretinal injection, using small doses with limited systemic exposure. He also said the eye allows clinicians to observe the treated tissue directly after administration.
“It really limits potential adverse events on the safety side,” Yerxa said. “On the CMC side, which is half of the drill in gene therapy, we need very small batches.”
BEST1 program expected to generate September data
Opus’ BEST1 program is being developed for Best disease, which Yerxa said affects an estimated 8,400 patients in the United States. He said the recessive form is rare, with roughly 300 to 350 U.S. patients, while the dominant form accounts for the vast majority of cases.
Yerxa described the dominant form as an insidious disease in which patients may initially have relatively good vision but ultimately lose central vision over time. He said there is currently no treatment and that Opus is the only company running a trial in the condition.
The company’s OPGx-BEST1 therapy uses an AAV2 capsid, the native VMD2 promoter and subretinal administration. Yerxa said the dose being tested is around 1.5E9, or about 100 times lower than LUXTURNA, because the target protein is not highly expressed.
The Phase 1/2 BEST1 study is designed as a basket trial enrolling both recessive and dominant Best disease patients. Yerxa said the study contemplates two dose levels, with five patients per cohort. A sentinel patient is treated first, followed by a 30-day safety review and a data safety monitoring board meeting before additional patients are enrolled.
Yerxa said Opus has guided for three-month data from the first five patients in September. Based on those data, the company will decide whether to dose escalate or move into the pivotal portion of the trial.
The sentinel patient was 63 years old, had been legally blind since her early 30s and entered the study with counting-fingers vision, Yerxa said. After treatment, the patient showed a 12-letter best-corrected visual acuity gain versus baseline, which Yerxa described as unexpected but consistent with a reduction in central retinal fluid.
For the September update, Yerxa said Opus expects to report a broad set of functional and structural measures, including best-corrected visual acuity, low-luminance visual acuity, microperimetry, contrast sensitivity and several optical coherence tomography analyses. He said potential pivotal endpoints could include BCVA, low-luminance visual acuity, microperimetry or OCT-based structural measures.
LCA5 pivotal study uses patients as their own controls
Yerxa also discussed Opus’ LCA5 program, which targets a form of Leber congenital amaurosis. He said affected children are “pretty much blind at birth” or within the first one to two years of life, but retinal structure can remain relatively preserved into the 20s and 30s.
Yerxa said the company’s sentinel LCA5 patient was 26 years old with hand-motion vision at baseline and could read the large “E” on an eye chart 30 days after treatment. He said data from adult and adolescent patients supported discussions with the FDA that allowed Opus to adapt the study from Phase 1/2 into a continuous Phase 1/2/3 trial.
The program has RMAT status and RDEP status, Yerxa said. He added that LCA5 has an estimated population of about 170 patients, fitting within the RDEP program’s threshold of fewer than 1,000 patients.
The Phase 3 study is enrolling a six-month natural history run-in period, with each patient serving as their own control. Yerxa said Opus is waiting for final FDA feedback on the pivotal endpoint and statistical analysis while preparing to dose patients in the second half of the year. The study will include both pediatric and adult patients, and Yerxa said the company believes the program could be eligible for a priority review voucher.
Three additional retinal disease programs nearing the clinic
Opus also plans to advance RDH12, MERTK and RHO programs into the clinic. Yerxa said RDH12, which came from Bennett’s lab, will be the first of the three to begin dosing later this year. The program uses an AAV8 capsid and targets photoreceptors.
MERTK uses an AAV2 capsid and targets retinal pigment epithelium. Yerxa said the trial will be conducted in Abu Dhabi, where MERTK is the third-leading cause of genetic blindness in the Middle East, and that Opus has discussed grant funding with the Department of Health there.
The RHO program targets retinitis pigmentosa associated with the RHO gene and is designed as a knockdown-and-replace approach for a dominant disease. Yerxa said it could begin dosing in the first half of next year. RDH12 and MERTK are “clearly pediatric,” while RHO is mostly adult-onset, he said.
Yerxa added that at least five of Opus’ seven programs could be eligible for pediatric priority review vouchers.
Presbyopia partnership and cash runway
Opus also has a partnered presbyopia program with Viatris, with a PDUFA date in October, according to Yerxa. He described the program as a financial asset for Opus, with a regulatory milestone on approval, a double-digit royalty that tiers into the 20% range and additional regulatory and sales milestones.
Yerxa said Opus has $90 million in cash and a runway into 2029. He said that runway could carry the company to its first potential priority review voucher auction.
About Opus Genetics NASDAQ: IRD
Opus Genetics Inc is a clinical-stage ophthalmic biopharmaceutical company, focuses on developing and commercializing therapies for the treatment of unmet needs of patients with refractive and retinal eye disorders. Opus Genetics Inc, formerly known as Ocuphire Pharma Inc, is based in Farmington Hills, Michigan.
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