Full Press Release Details
Milestone Pharmaceuticals
Announces FDA Acceptance of New Drug Application for CARDAMYST
Montreal and Charlotte, N.C., May, 29,
2024 -- Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical
company focused on the development and commercialization of innovative cardiovascular medicines, today announced that the United
States Food and Drug Administration (FDA) accepted the Company's New Drug Application (NDA) on May 26, 2024 for CARDAMYST (etripamil)
nasal spray, its lead investigational product for the management of paroxysmal supraventricular tachycardia (PSVT). The FDA Prescription
Drug User Fee Act (PDUFA) target date is 10 months from the acceptance date of May 26, 2024.
"The FDA's acceptance of our NDA for
CARDAMYST brings Milestone one step closer in our mission in providing a new, convenient and effective treatment option for patients with
PSVT," said Joseph Oliveto, President, and Chief Executive Officer of Milestone Pharmaceuticals. "We understand the frequent
impact that PSVT has on patients, as well as the underappreciated burden it places on their families and caregivers. Our progress toward
providing a sense of security for patients is a result of the dedicated Milestone team, patients, and investigators to whom we are thankful."
The CARDAMYST clinical trial program represents
the largest data package ever studied for an acute drug treatment intended for patient self-management of PSVT events. Milestone continues
to advance commercial preparations to support the anticipated launch of etripamil with the proposed trade name, CARDAMYST. The brand name
is conditionally approved by the FDA.
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.
(etripamil) nasal spray
CARDAMYST 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. The clinical trial program for CARDAMYST 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.
About Milestone Pharmaceuticals
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 CARDAMYST (etripamil) nasal spray, a novel calcium channel blocker that is being studied for patients
to self-administer without medical supervision to treat symptomatic episodic attacks associated with PSVT and AFib-RVR.
Forward-Looking Statements
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
our ability to bring a new PSVT therapeutic option to market; the timing and outcomes of
future interactions with U.S. and foreign regulatory bodies, including the
FDA; the timing of the launch of etripamil; and our ability to advance commercial preparations to support the anticipated launch
of etripamil. 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; 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,
Communications, kfox@milestonepharma.com
Kevin Gardner, kgardner@lifesciadvisors.com