Milestone Pharmaceuticals Inc . (NASDAQ:MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, today reported financial results for the fourth quarter and year ended December 31, 2021 and provided a clinical and corporate update.
"2021 was a year of focused execution across our ongoing clinical programs which are evaluating etripamil in patients with PSVT and AFib-RVR," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "We are well-positioned for a transformative 2022, with topline data from the pivotal Phase 3 RAPID trial on track for the middle of the second half of the year. We are committed to unlocking the full potential of etripamil, if approved, to serve as a meaningful therapeutic option for patients with episodic cardiovascular conditions. We look forward to providing updates on our progress throughout the coming quarters."
Recent Updates
- Company Expects to Report Topline Data in Mid-Second Half 2022. The RAPID trial, which is targeting a total of 180 confirmed PSVT events, is expected to randomize approximately 500 patients 1:1 to receive either etripamil or placebo. To maximize the potential treatment effect of etripamil, patients will be directed to administer a repeat dose of study drug if they do not experience symptom relief within 10 minutes of the first study drug administration. The primary efficacy analysis for both the RAPID trial and the completed NODE-301 trial will be time to conversion of supraventricular tachycardia (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) submission for etripamil in patients with PSVT.
- New Clinical Analysis Evaluating the Drug Characteristics and Safety of Etripamil to be Presented at the American College of Cardiology (ACC) 71st Annual Scientific Session and Expo. New analyses on the safety, tolerability, pharmacokinetics and pharmacodynamics of etripamil will be presented at the upcoming ACC 71st Annual Scientific Session and Expo taking place from April 2-4, 2022, in Washington D.C. The presentation, titled "Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intranasal Etripamil in Healthy Japanese and Non-Japanese Adults", will be featured during a poster session on April 4, 2022 at 12:15 p.m. ET.
- Appointment of David Bharucha, M.D., Ph.D., as Chief Medical Officer. In February 2022, Milestone announced the appointment of Dr. David Bharucha as Chief Medical Officer. Dr. Bharucha is a cardiac electrophysiologist who brings to Milestone over thirty years of global drug development and clinical experience across a range of therapeutic areas, with a focus on cardiovascular medicine.
- Heart Rate Data from NODE-301 Study Presented at the American Heart Association (AHA) Scientific Sessions 2021. In November 2021, new data from a post-hoc analysis of the Phase 3, randomized, double-blind, placebo-controlled NODE-301 trial were presented during an e-poster session at the AHA Scientific Sessions 2021 meeting. The data demonstrated that etripamil significantly decreased heart rate prior to conversion to sinus rhythm. A copy of the presentation, titled "Etripamil Nasal Spray Reduces Heart Rate in Patients with Paroxysmal Supraventricular Tachycardia Prior to Conversion to Sinus Rhythm", is available on the Publications page of the Milestone website.
- Recruitment Continues in the ReVeRA Phase 2 Proof-of-Concept Trial in Patients Experiencing Atrial Fibrillation with Rapid Ventricular Rate (AFib-RVR). Enrollment continues in ReVeRA, Milestone's Phase 2 double-blind, placebo-controlled, proof-of-concept in-patient study of etripamil nasal spray in patients experiencing AFib-RVR. The study, in which patients are randomized 1:1 to receive either 70 mg of etripamil or placebo, is designed to assess the safety and efficacy of etripamil nasal spray to reduce the ventricular rate in patients with AFib-RVR. The trial is being conducted in Canada in collaboration with the Montreal Heart Institute and other research centers. The primary endpoint will assess reduction in ventricular rate, with key secondary endpoints including the time to achieve the maximum reduction in rate and duration of the effect.
Fourth Quarter and Full Year 2021 Financial Results
- As of December 31, 2021, Milestone had cash, cash equivalents, and short-term investments of $114.1 million, compared to $142.3 million as of December 31, 2020, and 29.9 million common shares and 12.3 million common shares issuable upon exercise of pre-funded warrants outstanding.
- Research and development expense for the fourth quarter of 2021 was $10.9 million, compared with $5.8 million for the prior year period. For the full year ended December 31, 2021, research and development expense was $38.7 million, compared with $34.5 million for the prior year. The increase of research and development expense is due to personnel-related costs, higher clinical consulting fees and CRO costs due to advancing RAPID Phase 3 efficacy and NODE-303 safety trials in etripamil for the treatment of PSVT along with an increase in non-cash compensation costs related to share-based compensation expense.
- General and administrative expense for the fourth quarter of 2021 was $3.8 million, compared with $1.7 million for the prior year period. For the full year ended December 31, 2021, general and administrative expense was $12.4 million, compared with $10.3 million for the prior year. The increase of general and administrative expense is due to an increase in share-based compensation expense.
- Commercial expense for the fourth quarter of 2021 was $2.2 million, compared with $1.3 million for the prior year period. For the full year ended December 31, 2021, commercial expense was $7.0 million, compared with $5.9 million for the prior year. The increase of commercial expense is due increase in personnel related costs and marketing activities.
- For the fourth quarter of 2021, operating loss was $16.9 million, compared to $8.8 million for the prior year period. For the full year ended December 31, 2021, Milestone's operating loss was $42.9 million, compared to $50.0 million for the prior year.