Ophthalmology Therapeutix is filing with FDA after wet AMD drug outperforms Regeneron's Eylea in Phase 3 testing
Oural Therapeutix's drug, being developed for wet age-related macular degeneration, is better at helping patients preserve their vision than Regeneron Pharmaceuticals' blockbuster drug, the standard treatment for the vision-loss disorder, and Phase 3 results also suggest the therapy has the potential to provide patients with less frequent eye injections to treat the vision-loss disorder.
A second Phase 3 test of ophthalmic drug Axpaxli is underway. But based on the preliminary results reported from the first test, the company said it plans to meet with the FDA to discuss plans for regulatory submission.
In wet age-related macular degeneration (AMD), abnormal blood vessel growth beneath the retina leaks fluid, affecting the macula, the central part of the retina. The main drug ingredient in Axpaxli is axitinib, a small molecule tyrosine kinase inhibitor designed to stop blood vessel growth. The drug uses bioabsorbable hydrogel technology that can release the drug for anywhere from days to months. Oural targets dosing intervals of six months or longer.
The Axpaxli data reported Tuesday came from a Phase 3 trial that evaluated a single dose of Oural's drug compared with a single dose of Regeneron's Eylea. These doses followed the eight-week loading portion of the study, in which all 344 study participants with newly diagnosed wet AMD received two injections of Eylea.
The results showed that at week 36, 74.1% of patients in the Axpaxli group maintained vision, compared with 55.8% in the Eylea group, a statistically significant result that met the trial's primary endpoint. The study still hasn't evaluated the durability of treatment. At week 52, 65.9% of patients in the Axpaxli group maintained vision, compared with 44.2% of patients in the control group.
Oural said the investigational drug has been generally well tolerated so far, with no reports of ocular or systemic serious adverse events. At week 52, participants resumed the treatment they originally received and will receive treatment again at week 76. Masking will continue and the study will follow patients through week 104. The company plans to present more detailed results from the study so far next week at the Macular Society's annual meeting in San Diego.
Although the study hit its target, Ocular shares opened Tuesday at $6.41, down more than 27% from Friday's closing price. William Blair analyst Lachlan Hanbury-Brown said in a research note that the difference in treatment effectiveness between the two groups was smaller than investors expected. Even so, he said investment banks believe the results support FDA approval. He noted that the Axpaxli unit's results were in line with expectations. The smaller difference in treatment effect was due to better-than-expected performance in the control group rather than poor performance of Axpaxli.
The second Phase 3 test of Axpaxli is expected to produce preliminary data in the first quarter of next year. Hanbury-Brown said the data to date are positive for this study and a separate Phase 3 program evaluating the drug in non-proliferative diabetic retinopathy.
“While the additional data will help to fully characterize Axpaxli's profile, we believe these data can de-risk its wet AMD development program and may also de-risk its diabetic eye disease HELIOS program,” Hanbury-Brown said.
Reducing dosing frequency is important for Ocular as it aims to break into the wet AMD space dominated by Regeneron's Eylea (marketed by Bayer outside the U.S.) and Roche's Vabysmo. Eylea is administered as an eye injection every four weeks for the first three months, followed by maintenance doses every eight weeks. Vabysmo is administered every four weeks for four months, then every four to eight weeks, depending on the patient's response to treatment. While both drugs block the protein VEGF, which promotes blood vessel growth, Vabysmo combines this mechanism of action with inhibiting another protein called Ang-2.
Oural has competition in the application of tyrosine kinase inhibition to treat wet AMD. The main pharmaceutical ingredient in EyePoint Pharmaceuticals' drug candidate Duravyu is vorolanib, a tyrosine kinase inhibitor delivered using proprietary technology that enables sustained delivery of treatment to the eye. Two Phase 3 studies are ongoing, evaluating doses every six months. EyePoint said it expects preliminary data from the first study in mid-2026.
Photo: Karen Bleier/AFP via Getty Images
