Ocular Therapeutix's Axpaxli Outperforms Eylea in Wet AMD Trial

Ocular Therapeutix said its experimental eye disease drug was more effective in helping patients maintain their vision compared to Regeneron's approved treatment Eylea in a late-stage trial. The Massachusetts-based company said that for the first time, an investigational drug for wet age-related macular degeneration has shown better results than an approved treatment in a trial meeting strict FDA standards.

The trial enrolled 344 patients with newly diagnosed wet AMD and demonstrated that Ocular's drug, Axpaxli, outperformed Eylea, one of the most widely used treatments for the disease. About 74% of patients who received a single 0.45 mg dose of Axpaxli maintained their vision at 36 weeks, compared with nearly 56% of those receiving a single 2 mg dose of Eylea. After one year, nearly 66% of Axpaxli patients maintained vision, while less than half of the comparison group did.

The drug also demonstrated better fluid control in the eye, a key measure of disease management. Ocular said many patients on Axpaxli did not need additional "rescue" injections for almost a year, suggesting the treatment may reduce the number of clinic visits required.

The company said it plans to discuss the data with the U.S. Food and Drug Administration and expects to submit a marketing application based on the results. The chronic eye disorder, a leading cause of blindness among the elderly, causes blurred vision or a blind spot in the patient's visual field. The disease affects about 1.7 million Americans, according to the company.

Separately, full 12-month data from Ocugen's phase 2 eye disease gene therapy show the therapy significantly reduces the size of lesions, though the treatment's smaller effect compared to an earlier preliminary analysis sent the biotech's stock down. One year after an injection of OCU410, patients with geographic atrophy given the now-planned phase 3 dose had a 31% average reduction in their lesion size. While that 31% drop was enough to meet the trial's primary endpoint—and surpassed the lesion-reduction ability of two approved therapies—it wasn't as high as the 54% seen in a January preliminary analysis.

Ocugen reported no serious adverse events and no adverse events of special interest in the phase 2 trial. The Pennsylvania-based biotech now plans to launch a phase 3 trial of the candidate later this year. The phase 3 trial is planned to enroll up to 300 patients and have an adaptive design powered at over 95%. OCU410's phase 2 trial enrolled 51 patients.

OCU410 is an AAV-based therapy that delivers the gene for retinoid-related orphan receptor alpha, a key stress-regulating receptor. Geographic atrophy is a serious condition that occurs in patients with dry age-related macular degeneration, causing damage to the retina over time that steadily leads to vision loss. Apellis Pharmaceuticals was the first to snag FDA approval for a geographic atrophy drug in 2023 with C3 inhibitor Syfovre, followed closely by Astellas' Izervay. These two meds are given as frequent injections, in contrast to OCU410's planned one-time administration.

Ocugen is hardly the only company pursuing gene therapies for AMD. Other candidates include AbbVie and Regenxbio's sura-vec, now in two pivotal trials, and Eli Lilly's phase 3 ixo-vec, picked up from the acquisition of Adverum Biotechnologies last year.