Aerie Pharmaceuticals Inc (NASDAQ:AERI) reported the successful 90-day topline efficacy results of its Rocket 4 Phase 3 clinical trial of product candidate RhopressaTM, 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 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 RhopressaTM for non-inferiority to twice-daily timolol. The range for the primary endpoint includes patients with baseline IOPs from above 20 mmHg (millimeters of mercury) to below 25 mmHg.

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

Separately, Aerie announced that it has withdrawn the RhopressaTM NDA (new drug application) that was submitted to the FDA in the third quarter of 2016. The filing was withdrawn as the result of a third party manufacturing facility in Tampa, Florida not being ready for pre-approval inspection by the FDA.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 RhopressaTM NDA filing at that time.

Management will host a conference call and provide accompanying slides to discuss the clinical results and the RhopressaTM NDA at 5:00 p.m. ET today.

RhopressaTM Phase 3 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.
  • RhopressaTM also demonstrated non-inferiority compared to timolol at the pre-specified secondary endpoint range of above 20 mmHg to below 27 mmHg, and also at a range of above 20 mmHg to below 28 mmHg.
  • The Rocket 4 efficacy results for RhopressaTM demonstrated a consistent level of IOP lowering across all baseline IOPs in the trial, and throughout the 90-day efficacy period.
  • The most common RhopressaTM adverse event was hyperemia, or eye redness, which was reported in approximately 40 percent of patients, 85 percent of which was scored as mild. Other adverse events, which have also been observed in previous RhopressaTM clinical trials, were reported in 5 percent to 12 percent of patients. There were no drug-related systemic or serious adverse events.

“These successful Rocket 4 topline efficacy results once again confirm that RhopressaTM is a highly efficacious and well-tolerated drug. In fact, RhopressaTM performed better at the higher baseline ranges in Rocket 4 than in both Rocket 1 and Rocket 2. This is in addition to RhopressaTM 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 RhopressaTM 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 RoclatanTM, patient enrollment in the second Phase 3 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 RhopressaTM 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 RhopressaTM.”

Richard A. Lewis, M.D., Aerie’s Chief Medical Officer, commented, “We have accumulated a wealth of information on how RhopressaTM performs, with nearly 2,000 patients dosed and approximately 150 investigator sites engaged in our Phase 3 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.”  (Original Source)

Shares of Aerie closed today at $33.28, down $0.84 or -2.46%. AERI has a 1-year high of $41.72 and a 1-year low of $10.82. The stock’s 50-day moving average is $34.63 and its 200-day moving average is $21.22.

On the ratings front, AERI has been the subject of a number of recent research reports. In a report issued on October 6, Cantor analyst Elemer Piros reiterated a Buy rating on AERI, with a price target of $50, which implies an upside of 45% from current levels. Separately, on the same day, Brean Capital’s Difei Yang reiterated a Buy rating on the stock and has a price target of $63.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Elemer Piros and Difei Yang have a yearly average return of 4.1% and a loss of 5.6% respectively. Piros has a success rate of 41% and is ranked #1030 out of 4188 analysts, while Yang has a success rate of 34% and is ranked #3927.

Overall, 8 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $51.00 which is 48.2% above where the stock opened today.

Aerie Pharmaceuticals, Inc. is a clinical stage pharmaceutical company, which is focused on the discovery, development and commercialization of therapies for the treatment of patients with glaucoma and other diseases of the eye. It product candidates are Rhopressa and Roclatan.