Aerie Pharmaceuticals, a biotechnology company focused on the discovery and development of novel treatments for glaucoma, today announced positive top-line results from its Phase 2a study evaluating AR-13324 for the treatment of glaucoma. AR-13324 is the first of a proprietary new drug class in glaucoma discovered at Aerie that offers a unique dual mechanism of action, lowering intraocular pressure (IOP) by enhancing fluid outflow through the trabecular pathway while simultaneously decreasing fluid inflow to the eye. Biochemical profiling of AR-13324 has revealed inhibitory activity against Rho kinase (ROCK) and the norepinephrine transporter.
The seven-day study evaluated the safety and efficacy of three dose levels of AR-13324, administered once-daily in the morning. All three doses demonstrated a statistically significant lowering of IOP compared to placebo. Further, the study confirmed AR-13324's long duration of effect with peak efficacy at least eight hours after dosing and significant IOP reductions maintained over 24 hours. AR-13324 was well-tolerated. Mild to moderate and transient hyperemia (eye redness) that was observed after the first dose appeared to diminish by the end of the treatment period. Aerie is continuing to advance its novel compound into Phase 2b.
Alan L. Robin, M.D., Clinical Professor, University of Maryland, Department of Ophthalmology and Associate Professor of Ophthalmology and International Health, Wilmer Institute and Bloomberg School of Public Health, Johns Hopkins University, commented, "There remain significant unmet needs in glaucoma treatment, primarily around efficacy and patient compliance. To achieve target IOP levels, patients are often required to combine medications that control fluid outflow in the eye with medications that control fluid inflow. A single product that addresses both inflow and outflow has the potential to offer an enhanced pressure lowering effect and improve patient compliance. Based on these results, I believe Aerie's unique product is very promising, particularly in regard to the potential for once-daily evening dosing to provide peak efficacy in the morning when IOP is the highest. We certainly need new treatment options in glaucoma and I look forward to seeing further results from this development program."
Thomas van Haarlem, M.D., President and Chief Executive Officer of Aerie Pharmaceuticals, commented, "The Phase 2a clinical data demonstrate a significant potential for AR-13324 to become a new first-line treatment option for patients with glaucoma. We believe that once-daily evening dosing will reveal the full potential of AR-13324's efficacy profile. We are very excited about the progress of our diversified pipeline of first-in-class glaucoma treatments. In addition to AR-13324, we recently announced positive Phase 2a results for our ROCK inhibitor, AR-12286, in fixed combination with travoprost."
The double-masked Phase 2a study enrolled 80 people in the U.S. with primary open angle glaucoma or ocular hypertension and randomized them to receive either one of three doses of AR-13324 (0.01%, 0.02% or 0.04%) or placebo. In this first-in-man study, patients were dosed once-daily in the morning for seven days and evaluated at four different time points during the day. The study was designed to evaluate the safety, tolerability and efficacy of AR-13324, and to determine the top of the dose response. Patients in the 0.02% cohort achieved the greatest response during the treatment period, exhibiting a 6.9 mm Hg reduction in mean IOP at peak efficacy (eight hours post dose) and a 5.9 mm Hg reduction at trough (24 hours post dose). After seven days of treatment, mean diurnal IOP for this cohort was lower than the pre-study IOP recorded while patients were on their prescribed glaucoma medication.
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