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After spending most of their lives nearly totally blind, a few patients can see objects for the first time after receiving a gene therapy being developed by a biotech in Research Triangle Park.
Opus Genetics is a small firm trying to address causes of blindness through its development of gene therapy treatments that target inherited retinal diseases. The company is raising capital to support these efforts after seeing positive early results in its first clinical study.
Opus in September began dosing the first cohort of patients in a Phase 1/2 trial evaluating the company's lead program, OPGx-LCA5. These were three patients in their 20s with Leber congenital amaurosis, a rare inherited eye disorder that causes severe vision loss within the first few months of a patient's life.
Testing in this first cohort was mostly around the goal of evaluating the safety of the gene therapy. And while the company saw positive safety signals, what was more surprising is what Opus learned about the therapy's efficacy.
The first subject in this cohort had what Opus CEO Ben Yerxa described as hand motion vision. Essentially, this subject could see the motion of a person's hands waving in front of them, but could not, for instance, make out the number of fingers on each hand. But 30 days after receiving the gene therapy, the subject's vision improved and, for the first time, they could read the largest letters on an eye exam chart.
Opus continued to enroll the next two subjects and similarly no safety issues and positive responses with the subjects improving to have some form of vision. Yerxa said one person went from seeing only large blurry objects to seeing mountains for the first time in their life.
The results were a surprise to clinicians running the trial and to Opus. Yerxa said the company wasn't expecting these sorts of efficacy results at this level of treatment.
"When these gene therapies work, the results can be really remarkable," Yerxa said.
With these early positive results, Opus is planning to advance the program with another cohort of patients at the next highest dose. Yerxa said dosing for this cohort could begin in mid-summer.
Opus also plans to talk with the U.S. Food and Drug Administration to expand the study population to include subjects 13 years or older. Currently, the trial is only for adults. Reaching patients earlier in their lives could lead to even better results, Yerxa said.
But moving this program forward, plus advancing the rest of the company's pipeline, requires more capital, Yerxa said. Opus is speaking with investors with the goal of raising financing that would support the company through 2025.
Yerxa did not cite a specific dollar amount for the round, but said that timeline would get the company's lead program, OPGx-LCA5, through the remaining Phase 1/2 study and ready for a pivotal trial that could lead to a regulatory filing with the FDA.
The funding would also support early-stage clinical trials for the company's second program — which is nearly ready to move into the clinic. And the funding would enable Opus to prepare a third program for clinical studies. Both of these programs, like the rest of the company's pipeline, focus on inherited retinal diseases.
Opus raised about $4.5 million last year. This funding round followed Opus raising $19 million in seed funding prior to its launch in 2021. The Retinal Degeneration Fund, the venture arm of the Foundation Fighting Blindness, led the initial round with a $10 million investment.
The past two years have marked a difficult time for financing for small biotech and pharmaceutical companies. But there are signs the environment is beginning to improve with an increase in deal activities, including mergers and acquisitions and IPOs. Yerxa described the situation as a "very slow thaw" that is incrementally improving every month. Opus is also applying for grants and sources of non-dilutive funding.
These early positive positive clinical trial results should help the company in its conversations with investors as it tries to tackle causes of blindness through its development of gene therapy treatments that target inherited retinal diseases.
"It gives us confidence that this first program has been significantly de-risked in terms of moving it forward all the way," Yerxa said.
