Benzinga | May 17, 2021 07:14AM EDT

Milestone Pharmaceuticals Gives an Update on Seconday Endpoints in its Phase 3 NODE-301 Trial

- Statistically Significant Improvements in PSVT-Associated Symptoms and Less ER Visits Observed in Patients Treated with Etripamil Compared to Placebo in the NODE-301 Trial -

- Data Presented at the American College of Cardiology's 70th Annual Scientific Session -



Milestone Pharmaceuticals Inc. (NASDAQ:MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, today announced the presentation of data related to key secondary endpoints from its Phase 3, multicenter, randomized, double-blind, placebo-controlled NODE-301 trial of etripamil nasal spray, the Company's novel investigational, short-acting calcium channel blocker, in patients with paroxysmal supraventricular tachycardia (PSVT). The oral presentation, titled "Etripamil Nasal Spray Relieves Symptoms And Reduces Emergency Room Interventions In Patients With Paroxysmal Supraventricular Tachycardia (PSVT): Analysis Of Clinical Outcomes In The Node-301 Trial", was featured during a late-breaker session at the American College of Cardiology's 70th Annual Scientific Session (ACC.21) that was held in a virtual format. A copy of the presentation will be available in the Publications section of Milestone's website.

"With current standards of care for the acute termination of PSVT restricted to the burdensome and costly acute care setting, this condition represents an area of particularly high unmet need," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "Etripamil holds the potential to enable patients to treat their episodes in the at-home setting and ultimately take control of their condition. We remain focused on the execution of our ongoing Phase 3 program and our vision to help patients suffering from episodes of SVT."

"Data support the potential for etripamil to improve the patient's experience, both decreasing common PSVT symptoms and reducing the need for emergency room visits relative to placebo," said Bruce Stambler, MD, FHRS, Piedmont Heart Institute, Atlanta, GA, and lead author of the presentation. "Once approved by FDA, etripamil could potentially serve as an effective, meaningful addition to the currently limited PSVT treatment armamentarium."

The NODE-301 trial, which enrolled a total of 431 patients across 65 sites in the U.S. and Canada, was an event-driven Phase 3 efficacy trial of etripamil versus placebo for terminating supraventricular tachycardia (SVT) episodes in the at-home setting. Data presented at ACC.21 build on previously reported data from the NODE-301 trial that showed statistically significant improvements in favor of etripamil over placebo in the important secondary endpoint of patient reported treatment satisfaction, as measured by a treatment satisfaction questionnaire for medication (TSQM-9), including global satisfaction and effectiveness scores, with questions addressing the relief of symptoms commonly associated with an episode of SVT. Treatment effectiveness (p=0.001) and global satisfaction (p=0.007) scores were greater in patients treated with etripamil than with placebo. Etripamil also had higher scores than placebo related to relief of specific symptoms associated with PSVT, including rapid pulse (p=0.002), palpitations (p<0.001), shortness of breath (p=0.008), dizziness (p=0.012), and anxiety (p=0.006). In addition, a 51% reduction (p=0.051) in the need for an emergency room (ER) visit and a 47% prolongation (p<0.05) in time to ER intervention was observed in patients who received etripamil versus placebo.

Enrollment is now underway in the Company's pivotal Phase 3 RAPID trial of etripamil nasal spray in patients with PSVT. This trial, which is targeting a total of 180 adjudicated PSVT events, is expected to randomize approximately 500 patients 1:1 to receive either etripamil nasal spray or placebo. To help maximize the potential treatment effect of etripamil, patients will be directed to administer a second dose of study drug if they do not experience symptom relief within 10 minutes of administration of the first dose of study drug. Under an updated statistical analysis plan, the primary efficacy analysis for both the RAPID trial and the already-completed NODE-301 trial will be time to conversion of SVT over the first 30 minutes following initial study drug administration, with a target p-value of less than 0.05 for each trial. The RAPID and NODE-301 trials could potentially serve to fulfill the efficacy requirement for a future New Drug Application (NDA) for etripamil in patients with PSVT.