Alcon’s $770 million takeover of Aerie Pharmaceuticals has delivered an early win. The lead candidate in the acquired pipeline met the primary endpoint in a pair of phase 3 dry eye trials, positioning Alcon to file for FDA approval in the middle of the year.
The studies randomized around 930 people with dry eye to receive AR-15512, a topical transient TRPM8 agonist, or vehicle control. After 14 days, a higher proportion of patients on AR-15512 had a 10 mm or greater increase in tear production, causing the program to meet its primary endpoint with a p value of less than 0.0001.
Alcon saw improvements in tear production as early as day one. The effects persisted through to day 90, although the former Novartis unit is yet to share data from the trial. Alcon is also yet to comment on the phase 3 trial’s other secondary endpoints such as ocular discomfort, which was once a focus of AR-15512 development.
AR-15512 failed to improve tear production or ocular discomfort on day 28 in an earlier study, causing that trial to miss its co-primary endpoints. Aerie looked past the setback, focused on successes against secondary goals—including tear production at day 14—designed a phase 3 program around the lessons of the earlier trial and was rewarded with a buyout bid from Alcon, plus positive top-line phase 3 data.
Alcon plans to use the data, which is free from serious ocular adverse events, to file for FDA approval in the middle of 2024. Talking to investors on a quarterly results conference call in November, Alcon CEO David Endicott said it would “probably take four months, five months to get [the data] into a submission format.” The eye care specialist is also working to wrap up a long-term safety study by April.
Approval will position Alcon to start selling a first-in-class treatment for dry eye disease, a condition that the company says affects an estimated 38 million people in the U.S. Around 20 million of those people are undiagnosed, according to Alcon, and fewer than 10% of people with a diagnosis take a prescription drug.
Most patients initially try artificial tears, and most pharmacologic topical therapies, including Bausch + Lomb’s Xiidra, target inflammation. AR-15512 has a different mechanism, as researchers have tied TRPM8 to the detection of ocular surface dryness.