Full Press Release Details
Milestone Pharmaceuticals
Announces Resubmission of New Drug Application for Etripamil for Treatment in Paroxysmal Supraventricular Tachycardia
Montreal and Charlotte, N.C., March 28,
2024 -- Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical
company focused on the development and commercialization of innovative cardiovascular medicines, today announced the resubmission
of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for etripamil, the Company's lead investigational
product for the management of paroxysmal supraventricular tachycardia (PSVT).
"We appreciate FDA's guidance through
the resubmission process. We believe we have addressed all the issues raised in the Refusal to File letter and look forward to working
with the Agency as it reviews our application," said Joseph Oliveto, President, and Chief
Executive Officer of Milestone Pharmaceuticals. "If approved, we believe that etripamil nasal spray will be a valuable treatment
option for patients suffering from PSVT."
The NDA for etripamil was resubmitted based on
guidance from the FDA obtained in a Type A meeting, which was conducted after receipt of a Refusal
to File (RTF) letter. The resubmission package included restructured data sets that captured timing of reported AEs and certain
data files reformatted to facilitate FDA's analyses. No additional efficacy or safety data were requested as part of the RTF.
About Paroxysmal Supraventricular Tachycardia
An estimated two million people in the United
States 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 procedures.
About Atrial Fibrillation with Rapid Ventricular
An estimated five million Americans suffer from
atrial fibrillation (AFib), a common arrhythmia marked by an irregular, disruptive and often rapid heartbeat. The incidence of AFib is
expected to grow to approximately 10 million by 2025 and up to about 12 million by 2030. A subset of patients with AFib experience episodes
of abnormally high heart rate most often accompanied by palpitations, shortness of breath, dizziness, and weakness. While these episodes,
known as atrial fibrillation with rapid ventricular rate (AFib-RVR), may be treated by oral calcium channel blockers and/or beta blockers,
patients frequently seek acute care in the emergency department to address symptoms. In 2016, nearly 800,000 patients were admitted to
the emergency department due to AFib symptoms where treatment includes medically supervised intravenous administration of calcium channel
blockers or beta blockers, or electrical cardioversion. With little available data for AFib-RVR, Milestone's initial market research indicates
that 30 to 40% of patients with AFib experience one or more symptomatic episodes of RVR per year that require treatment, suggesting a
target addressable market of approximately three to four million patients in 2030 for etripamil in patients with AFib-RVR.
Etripamil is Milestone's lead investigational
product. It is a novel calcium channel blocker nasal spray under clinical development for frequent and often highly symptomatic episodes
of PSVT and AFib-RVR. It is designed as a self-administered rapid response therapy for patients thereby bypassing the need for immediate
medical oversight. If approved, 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. CARDAMYST , the conditionally approved brand name for etripamil nasal spray, is well studied
with a robust clinical trial program that includes a completed Phase 3 clinical-stage program for the treatment of PSVT and Phase 2 trial
for the treatment of patients with AFib-RVR.
Milestone Pharmaceuticals Inc. (Nasdaq: MIST)
is a biopharmaceutical company developing and commercializing innovative cardiovascular solutions to improve the lives of people living
with complex and life-altering heart conditions. The Company's focus on understanding unmet patient needs and improving the patient
experience has led us to develop new treatment approaches that provide patients with an active role in self-managing their care. Milestone's
lead investigational product is etripamil, a novel calcium channel blocker nasal spray that is being studied for patients to self-administer
without medical supervision to treat highly symptomatic episodic attacks associated with PSVT and AFib-RVR.
Forward-Looking Statements
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
the timing and outcomes of future interactions with the FDA; expected growth of incidence of AFib by 2030; our future target addressable
market; the potential of etripamil to provide health care providers with a new treatment option to enable on-demand care and patient self-management
and provide patients with active management and a greater sense of control over their condition. Important factors that could cause actual
results to differ materially from those in the forward-looking statements include, but are not limited to, whether our future interactions
with the FDA will have satisfactory outcomes; whether and when, if at all, our NDA for etripamil will be approved by the FDA; whether
the FDA will require additional trials or data which may significantly delay and put at risk our efforts to obtain approval and may not
be successful, the risks inherent in biopharmaceutical product development and clinical trials, including the lengthy and uncertain regulatory
approval process; uncertainties related to the timing of initiation, enrollment, completion, evaluation and results of our 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, Russian hostilities in Ukraine and
ongoing disputes in Israel and Gaza 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, including in its annual report on Form 10-K for the year
ended December 31, 2023, under the caption "Risk Factors," as such
discussion may be updated from time to time by subsequent filings Milestone may make with the U.S. Securities &
Exchange Commission. 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.
Kim Fox, Vice President, Communicationskfox@milestonepharma.com
Chris Calabrese, ccalabrese@lifesciadvisors.com
Kevin Gardner,kgardner@lifesciadvisors.com