Opus Genetics Eyes 2029 Runway as Presbyopia Drop Wins FDA Approval

Key Points

• FDA approved phentolamine for presbyopia ahead of schedule, triggering a $10 million milestone payment from Viatris and providing access to up to $130 million in total milestones plus royalties.
• Opus Genetics is advancing its retinal disease pipeline: the LCA5 gene therapy registrational study is dosing patients with results expected by the end of next year, while the BEST1 higher-dose cohort is fully enrolled and data are expected in the second quarter of next year.
• With $80 million in second-quarter capital, roughly $20 million from exercised warrants, the expected phentolamine milestone and potential credit-facility draws, Opus projects its cash runway will extend into 2029.

Opus Genetics NASDAQ: IRD outlined commercialization expectations for its newly approved presbyopia eye drop, progress across its inherited retinal disease gene therapy pipeline and its funding strategy during an investor panel featuring Chief Executive Officer Dr. George Magrath and Chief Financial Officer Rob Gagnon.

The company said the FDA approved phentolamine for presbyopia on Friday, ahead of its PDUFA date. The treatment, supported by the VEGA-2 and VEGA-3 Phase III trials, is partnered with Viatris for U.S. commercialization.

Phentolamine Deal Includes Milestone and Royalties

Gagnon said FDA approval triggers a $10 million milestone payment to Opus that the company expects to receive during the current quarter. The Viatris agreement includes up to $130 million in total potential milestones, as well as royalties that begin in the lower teens and “quickly” scale to a higher level, though the exact royalty rates were not disclosed.

Magrath said phentolamine may be differentiated from other presbyopia drops by its safety and tolerability profile. He said it is the first approved presbyopia drop without a warning regarding the potential for retinal detachment and does not carry the same night glare or nighttime dimming issues associated with certain strong miotics.

According to Magrath, the product does not engage the ciliary body, which he said contributes to its cleaner safety profile and could allow for increased use of the drop.

LCA5 Registrational Study Underway

Opus is dosing patients in its registrational trial of OPGx-LCA5, a gene therapy candidate for LCA5-associated retinal disease. The pivotal study includes eight patients, each serving as their own control following a six-month pretreatment observation period.

The primary endpoint is the proportion of patients achieving more than a 7-decibel improvement in microperimetry within the central 16 points of the retinal testing grid. Magrath said the company’s prior study produced an average improvement of 10.5 decibels, giving management confidence in the trial threshold.

“The patients have all completed that six-month period, and we’re currently dosing,” Magrath said, adding that dosing should conclude soon and that a readout is expected by the end of next year.

Magrath estimated the addressable LCA5 population at roughly 150 to 200 patients, with most patients seen at the University of Pennsylvania. He said the concentration of patients at a single center could limit the commercial infrastructure needed to reach the population. He also said the program may qualify for a priority review voucher.

BEST1 Program Advances to Higher-Dose Cohort

Opus also discussed OPGx-BEST1, its gene therapy candidate for BEST1-related retinal disease. The company previously reported low-dose Cohort 1 data in which all five participants experienced a clinically meaningful improvement on at least one visual-function measure, four of five showed structural improvement, and no serious adverse events were reported.

Magrath described the findings as among the most compelling he has seen in ophthalmology trials, citing the disappearance of vitelliform material in patients with Best vitelliform macular dystrophy and improved retinal function in the same locations.

The company has enrolled 11 patients in the higher-dose Cohort 2, exceeding the original five-patient enrollment plan. Magrath said Opus expects to report the Cohort 2 data in the second quarter of next year, alongside a potential update on transitioning to a pivotal study.

The planned pivotal study is expected to use a microperimetry endpoint, which Magrath said has already been discussed with the FDA. He said the ongoing study has an adaptive design that could be directly carried into the pivotal trial, potentially reducing execution time and costs.

Opus estimates there are approximately 23,600 symptomatic, treatable BEST1 patients in the U.S. Magrath said the estimate was supported by a quantitative analysis of 150 eye-care providers and aligned with views expressed by key opinion leaders. Earlier estimates based primarily on genetically tested patients had put the population at about 8,400 patients, he said.

Magrath pushed back on concerns that the company had shifted its development focus from subretinal fluid to vitelliform material. He said subretinal fluid had initially been viewed as a “lowest common denominator” measure but is not an FDA-approvable endpoint and is more relevant in later-stage disease. The observed reduction of vitelliform material, combined with improved microperimetry results, exceeded the company’s expectations, he said.

Runway Projected Into 2029

Gagnon said Opus reported $80 million of capital as of the second quarter. He said warrants associated with the BEST1 Cohort 1 data release have largely been exercised, bringing in roughly $20 million. Together with the expected $10 million phentolamine milestone and potential draws under an existing credit facility, the company projects that its cash runway extends into 2029.

That forecast does not assume proceeds from any priority review voucher, Gagnon said. He noted that vouchers have recently been auctioned for more than $200 million and said a potential voucher associated with LCA5 could represent a future fundraising opportunity.

Management said its current budget and runway support advancing five programs, including RDH12, MERTK and RHO alongside LCA5 and BEST1. Magrath said the company plans to prioritize programs based on clinical evidence, particularly the ability of treatments to demonstrate vision improvement within three to six months. He added that Opus currently sees particular promise in the BEST1 program.

About Opus Genetics (NASDAQ:IRD)

Opus Genetics, Inc is a clinical-stage biotechnology company focused on developing gene therapies for inherited retinal diseases (IRDs), a group of rare genetic disorders that can cause progressive vision loss and blindness. The company's research is aimed at addressing the underlying genetic causes of these conditions rather than treating only their symptoms.

Its pipeline includes investigational ophthalmic gene therapies designed for specific IRD-causing mutations, including programs targeting conditions associated with the NR2E3 and BEST1 genes.

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