Full Press Release Details
Milestone Pharmaceuticals Announces Acceptance
of Marketing Authorization Application for Etripamil Nasal Spray in PSVT by the European Medicines Agency
Montreal and Charlotte, N.C., January 6, 2026 - Milestone
Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development
and commercialization of innovative cardiovascular medicines, today announced the acceptance of a Marketing Authorization Application
(MAA) by the European Medicines Agency (EMA) seeking the approval of etripamil nasal spray, developed to be the first rapid, reliable
option in the treatment of paroxysmal supraventricular tachycardia (PSVT) outside of the healthcare setting. Etripamil, which has the
conditionally approved brand name TACHYMIST in Europe, is an investigational, novel calcium channel blocker delivered via a nasal
spray by the patient for the potential treatment of PSVT and other cardiac arrhythmias. A decision on approval is expected by the first
"Following guidance from the EMA, this MAA incorporates the global
clinical data package that supported the U.S. Food and Drug Administration approval of etripamil for the treatment of PSVT," said Joseph
Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "TACHYMIST could become the first approved patient
self-administered therapy representing a meaningful step forward in patient care for the approximately two million people suffering from
About the Pivotal RAPID Phase 3 Trial
The MAA submission is supported by efficacy, safety, and tolerability
results from a robust clinical trial program based on data from more than 1,800 participants and more than 2,000 episodes of PSVT. This
includes the successful Phase 3 RAPID trial, a global, randomized, double-blind comparison of etripamil vs. placebo, published in The
Lancet in 2023. In clinical studies, participants using etripamil were two times more likely to convert symptomatic PSVT
to sinus rhythm and did so more than three times faster compared with placebo. The RAPID trial achieved its primary endpoint with 64%
of those who self-administered etripamil (N=99) converting from PSVT to sinus rhythm within 30 minutes compared to 31% on placebo (N=85)
(HR=2.62; p<0.001). At one hour, the benefit was demonstrated in 73% of participants. In addition, significant reductions in time
to conversion in those who took etripamil were evident early and were durable, with a median time to conversion of 17 minutes (95% CI:
13.4, 26.5) for those treated with etripamil vs. 54 minutes (95% CI: 38.7, 87.3) for those treated with placebo.
A consistent safety profile and treatment effects were observed
across all subgroups, including participants concurrently on beta blockers or calcium channel blockers. The most frequent adverse
events occurring in 5% of participants in randomized clinical trials were mild-to-moderate and transient in nature, including
local-site nasal discomfort, nasal congestion, rhinorrhea, throat irritation, and epistaxis. Less than 2% of trial participants
discontinued therapy due to adverse events.
About Paroxysmal Supraventricular Tachycardia
An estimated two million people in the United States, and an additional
two million people in Europe, are currently diagnosed with PSVT, which is a type of arrhythmia or abnormal heart rhythm. PSVT is characterized
by episodes of sudden onset rapid heartbeats often exceeding 150 to 200 beats per minute. The heart rate spike is unpredictable and may
last several hours. The rapid heart rate often causes disabling severe palpitations, shortness of breath, chest discomfort, dizziness
or lightheadedness, and distress, forcing patients to limit their daily activities. The uncertainty of when an episode of PSVT will strike
or how long it will persist can provoke anxiety in patients and negatively impact their day-to-day life between episodes. The impact and
morbidity from an attack can be especially detrimental in patients with underlying cardiovascular or medical conditions, such as heart
failure, obstructive coronary disease, or dehydration. Many health care providers are dissatisfied with the lack of effective treatment
options with patients often requiring prolonged, burdensome, and costly trips to the emergency department or even invasive cardiac ablation
Etripamil is approved by the U.S. Food and Drug
Administration (FDA) for the conversion of acute symptomatic episodes of paroxysmal supraventricular tachycardia (PSVT) to sinus rhythm
in adults under the brand name CARDAMYST . Etripamil is a novel calcium channel blocker nasal spray designed as a self-administered
rapid response therapy for patients, thereby bypassing the need for direct medical oversight. Etripamil is intended to provide health
care providers with a new treatment option to enable on-demand care and patient self-management. This portable, self-administered treatment
may provide patients with active management and a greater sense of control over their condition. TACHYMIST , the conditionally
approved brand name for etripamil nasal spray in Europe, is well studied with a robust clinical trial program that includes a completed
Phase 3 clinical-stage program for the treatment of PSVT. Currently, etripamil is in Phase 2 development for treatment of PSVT in pediatric
patients and Phase 3 development for acute treatment of atrial fibrillation with rapid ventricular rate (AFib-RVR) in adults.
About Milestone Pharmaceuticals
Milestone Pharmaceuticals Inc. (Nasdaq: MIST) is a biopharmaceutical
company developing and commercializing innovative cardiovascular medicines to benefit people living with certain heart conditions. Milestone's
lead product is CARDAMYST (etripamil) nasal spray, a novel calcium channel blocker, which is FDA approved for the conversion of
acute symptomatic episodes of paroxysmal supraventricular tachycardia (PSVT) to sinus rhythm in adults. Etripamil is also in development
for the treatment of symptomatic episodic attacks associated with AFib-RVR.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning
of the Private Securities Litigation Reform Act of 1995. Words such as "believe," "continue," "could,"
"demonstrate," "designed," "develop," "estimate," "expect," "may,"
"pending," "plan," "potential," "progress," "will", "intend" and
similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify
forward-looking statements. These forward-looking statements are based on Milestone's expectations and assumptions as of the date
of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially
from these forward-looking statements. Forward-looking statements contained in this press release include statements regarding: timing
and expectations regarding the EMA's review of the MAA, including the timing around a decision on approval; Milestone's expectations
on the market for PSVT in Europe; expectations in regards to etripamil's efficacy; etripamil's potential as a novel treatment
option to help patients with PSVT; and other statements not related to historical facts. Important factors that could cause actual results
to differ materially from those in the forward-looking statements include, but are not limited to, whether Milestone's future interactions
with the EMA will have satisfactory outcomes; whether and when, if at all, Milestone's MMA for etripamil will be approved by the
EMA; uncertainties related to the timing of initiation, enrollment, completion, evaluation and results of Milestone's clinical trials;
risks and uncertainty related to the complexity inherent in cleaning, verifying and analyzing trial data; and whether the clinical trials
will validate the safety and efficacy of etripamil for PSVT or other indications, among others, general economic, political, and market
conditions, including deteriorating market conditions due to investor concerns regarding inflation, international tariffs, Russian hostilities
in Ukraine and ongoing disputes in the Middle East and overall fluctuations in the financial markets in the United
States and abroad, risks related to pandemics and public health emergencies, and risks related the sufficiency of Milestone's
capital resources and its ability to raise additional capital in the current economic climate. These and other risks are set forth in
Milestone's filings with the U.S. Securities and Exchange Commission (SEC), including in its annual report on Form 10-K
for the year ended December 31, 2025 and its quarterly report on Form 10-Q for the quarter ended September 30, 2025, in each
case under the caption "Risk Factors," as such discussions may be updated from time to time by subsequent filings Milestone
may make with the SEC. Except as required by law, Milestone assumes no obligation to update any forward-looking statements contained
herein to reflect any change in expectations, even as new information becomes available.
Kevin Gardner, kgardner@lifesciadvisors.com
Rebecca Novak, rnovak@milestonepharma.com