Aerie Pharmaceuticals, a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of first-in-class therapies for the treatment of glaucoma and other diseases of the eye, has reported the successful 90-day topline efficacy results of its Rocket 4 phase III clinical trial of product candidate Rhopressa, a novel once-daily eye drop being tested for its ability to lower intraocular pressure (IOP) in patients with glaucoma or ocular hypertension. The Rocket 4 trial is designed to provide adequate six-month safety data for European regulatory filing purposes. Rocket 4 is not necessary for new drug application (NDA) filing purposes.

Rocket 4 enrolled a total of approximately 700 patients and is a two-arm six-month trial, which includes a 90-day efficacy readout evaluating once-daily Rhopressa for non-inferiority to twice-daily timolol. The range for the primary endpoint includes patients with baseline IOPs from above 20mmHg (millimeters of mercury) to below 25mmHg.

The study achieved its primary efficacy endpoint demonstrating non-inferiority of once-daily Rhopressa compared to twice-daily timolol.

Separately, Aerie announced that it has withdrawn the Rhopressa NDA that was submitted to the FDA in the third quarter of 2016. The filing was withdrawn as the result of a third party manufacturing facility in Tampa, Florida not being ready for pre-approval inspection by the FDA. The drug product contract manufacturer has advised Aerie and the FDA that it expects to be prepared for FDA inspection in January 2017, and Aerie expects to resubmit the Rhopressa NDA filing at that time.

RhopressaTM phase III Highlights for Rocket 4:

• RhopressaTM dosed once-daily achieved its primary efficacy endpoint demonstrating non-inferiority compared to twice-daily timolol for patients with baseline IOPs ranging from above 20 to below 25 mmHg.
• Rhopressa also demonstrated non-inferiority compared to timolol at the pre-specified secondary endpoint range of above 20mmHg to below 27 mmHg, and also at a range of above 20mmHg to below 28mmHg.
• The Rocket 4 efficacy results for Rhopressa demonstrated a consistent level of IOP lowering across all baseline IOPs in the trial, and throughout the 90-day efficacy period.
• The most common Rhopressa adverse event was hyperemia, or eye redness, which was reported in approximately 40% of patients, 85% of which was scored as mild. Other adverse events, which have also been observed in previous Rhopressa clinical trials, were reported in 5% to 12% of patients. There were no drug-related systemic or serious adverse events.

“These successful Rocket 4 topline efficacy results once again confirm that Rhopressa is a highly efficacious and well-tolerated drug. In fact, Rhopressa performed better at the higher baseline ranges in Rocket 4 than in both Rocket 1 and Rocket 2. This is in addition to Rhopressa having demonstrated non-inferiority to latanoprost for baseline IOP ranges of above 20 mmHg to below 25 mmHg in the recent readout from the Mercury 1 clinical trial,” said Vicente Anido, Jr., Ph.D., chairman and chief executive officer at Aerie.

Dr. Anido continued, “Rocket 4 is designed to provide adequate six-month Rhopressa safety data for our filing with the European regulatory authorities. We expect this trial to be completed in the second quarter of 2017. Updating on Roclatan, patient enrollment in the second phase III registration trial, Mercury 2, is well underway and we remain on target for the 90-day topline efficacy readout in the second quarter of 2017.”

Dr. Anido added, “Regarding the withdrawal of the Rhopressa NDA, we plan on refiling the NDA in January of 2017, which is when we and the FDA have been advised by our contract manufacturer that they expect to be prepared for FDA inspection. As part of a 60-day review by the FDA, we also received a few minor routine inquiries, none of which were related to safety or efficacy of Rhopressa.”

Richard A. Lewis, M.D., Aerie’s chief medical officer, commented, “We have accumulated a wealth of information on how Rhopressa performs, with nearly 2,000 patients dosed and approximately 150 investigator sites engaged in our phase III clinical trials. It is very clear to me that, if approved, this novel IOP-lowering product that targets the diseased tissue has the potential to be a meaningful new therapy for patients with glaucoma or ocular hypertension.”