お知らせ • May 18
Milestone Pharmaceuticals Announces Late-Breaker Oral Presentation of NODE-301 Data at ACC.21
Milestone Pharmaceuticals Inc. 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. 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. The NODE-301 trial is a Phase 3, multicenter, randomized, double-blind, placebo-controlled trial of etripamil, the Company's lead investigational product, etripamil. The study is designed for a population of those PSVT patients who historically experience SVT episodes lasting 20 minutes or longer or episodes requiring termination in the emergency department. Following an in-office test dose of etripamil, 97.5% of patients were randomized (2:1) to receive either 70 mg of etripamil or placebo. Upon onset of PSVT symptoms, patients applied a wireless cardiac monitor to their chest to record heart rhythm, performed a vagal maneuver, and, if symptoms persisted, administered study drug. Of the 198 patient-reported events for which study drug was administered, a total of 156 were confirmed to be SVT events by a central independent adjudication committee and used to assess the study's efficacy endpoints. The primary endpoint of the NODE-301 study is time to conversion of an SVT episode to sinus rhythm after the administration of study drug, as confirmed by a central independent adjudication committee. The NODE-301 trial did not meet its primary endpoint. Secondary study endpoints include relief of symptoms commonly associated with an episode of SVT such as heart palpitations, chest pain, anxiety, shortness of breath, dizziness, or fainting, and rating of treatment satisfaction questionnaire for medication (TSQM).