- Submission seeks approval for treatment of an abnormal heart
rhythm, Paroxysmal Supraventricular Tachycardia or PSVT
- Comprehensive data package includes positive results from
pivotal Phase 3 RAPID trial which Company believes demonstrates new
calcium channel blocker, etripamil, is twice as
effective and three times as fast as a placebo in restoring normal
heart rhythm for patients suffering from PSVT
- CARDAMYST™, the conditionally approved brand name
for etripamil nasal spray, if approved, will be the first rapid,
reliable and at-the-ready option in the treatment of serious heart
arrhythmias
MONTREAL and CHARLOTTE,
N.C., Oct. 24, 2023 /PRNewswire/ --
Milestone® Pharmaceuticals Inc. (Nasdaq: MIST)
today announced the submission of a New Drug Application (NDA) to
the U.S. Food and Drug Administration (FDA) for etripamil for the
treatment of paroxysmal supraventricular tachycardia (PSVT). PVST
is a condition characterized by an abnormality in the electrical
system of the heart causing patients to have unexpected, often
severely symptomatic episodes of rapid heart rate.
Representing the largest data package ever studied in PSVT, the
Company believes the clinical trial program demonstrates that
etripamil provided superior time to conversion to normal heart
rhythm compared to placebo. The primary analysis and supportive
assessments from the pivotal RAPID Phase 3 clinical trial were
recently published in The Lancet.
"With the achievement of our first NDA submission, we want to
thank the investigators, clinical trial teams, our colleagues and,
most importantly, patients whose contributions were critical to
completing our registration trials," said Joseph Oliveto, President, and Chief Executive
Officer of Milestone Pharmaceuticals. "With this submission, we are
one step closer to getting etripamil into the hands of patients who
are seeking a new treatment option that will allow them to take an
active role in managing their PSVT."
The FDA has a 60-day filing review period to determine whether
the NDA is complete and accepted for review. If accepted, Milestone
Pharmaceuticals expects a standard review of 10 months of the
company's NDA. 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 Pivotal RAPID Phase 3 Trial
Recently published in The Lancet, RAPID is a global,
randomized, double-blind phase 3 clinical trial of etripamil versus
placebo in patients with PSVT. The trial was designed to evaluate
the safety and efficacy of self-administered etripamil for treating
PSVT. The RAPID trial achieved its primary endpoint with 64% of
patients who self-administered etripamil converting from
supraventricular tachycardia (SVT) to sinus rhythm within 30
minutes compared to 31% on placebo (HR = 2.62, p<0.001). At one
hour, the benefit was demonstrated in 73% of patients. In addition,
significant reductions in time to conversion in patients who
took etripamil were evident early and durable, with a
median time to conversion of 17 minutes (95% CI: 13.4, 26.5)
for patients treated with etripamil versus 54 minutes
(95% CI: 38.7, 87.3) for patients treated with placebo. Data
demonstrated statistically significant improvement in multiple
defined symptoms of PSVT in patients receiving
etripamil compared to placebo, using a patient-reported
outcome (PRO) questionnaire. The safety and tolerability profile of
etripamil is supportive of the NDA submission.
About Paroxysmal Supraventricular Tachycardia (PSVT)
An estimated 2 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 rapid heartbeats often exceeding 150 to 200 beats
per minute. Key features of PSVT include the sudden occurrence of
episodes and very rapid heart rate. The heart rate can spike
unpredictably and rapidly during an episode. The rapid heart rate
often causes 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 doctors are
dissatisfied with the lack of effective treatment options in
addition to a prolonged, burdensome, and costly trip to the
emergency department or, for some patients, an invasive ablation
procedure.
About Etripamil
Etripamil is Milestone's lead investigational product. It is a
novel calcium channel blocker nasal spray being developed for
elevated and often highly symptomatic heart-rate attacks associated
with PSVT and atrial fibrillation with a rapid ventricular rate
(AFib-RVR). It is designed to be a rapid-response therapy that is
self-administered by the patient, without the need for direct
medical oversight. If approved, etripamil is intended to provide
health care providers with a new treatment option to enable virtual
care and patient self-management. If approved, the portable
treatment, studied as self-administered, may provide patients with
active management and a greater sense of control over their
condition. Etripamil, proposed brand name CARDAMYST™, is well
studied with a robust clinical trial program that includes a
completed Phase 3 clinical-stage program for the treatment of PSVT
and soon-to-be-reported Phase 2 proof-of-concept trial for the
treatment of patients with AFib-RVR.
About Milestone Pharmaceuticals
Milestone Pharmaceuticals Inc. (Nasdaq: MIST) is a
biopharmaceutical company focused on the development and
commercialization of innovative cardiovascular medicines.
Milestone's lead product candidate etripamil recently completed its
Phase 3 clinical-stage program for the treatment of PSVT and is in
a Phase 2 proof-of-concept trial for the treatment of patients with
AFib-RVR. The AFib-RVR study, ReVeRA, was selected for a
Featured Science presentation at the American Heart Association
(AHA) Scientific Sessions 2023. Milestone
Pharmaceuticals operates in Canada and the
United States. Find out more
at www.milestonepharma.com.
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" 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 potential of etripamil nasal spray to be the first
rapid, reliable, safe, and at-the-ready option in the treatment of
serious heart arrhythmias; the continued ability of etripamil
provided superior time to conversion to normal heart rhythm
compared to placebo; the timing of the anticipated launch of
etripamil; the success of the NDA submission for etripamil nasal
spray and the timing of the FDA's approval of the NDA; the FDA's
final approval of etripamil's proposed trade name; and the status
and timing of the Phase 2 proof-of-concept trial of etripamil for
the treatment of patients with AFib-RVR. Important factors that
could cause actual results to differ materially from those in the
forward-looking statements include, but are not limited to, 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 and
Russian hostilities in Ukraine 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,
2022, under the caption "Risk Factors," as such discussion
may be updated from time to time by subsequent filings, we 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.
Contact:
Kim Fox, Vice President,
Communications
kfox@milestonepharma.com
704-803-9295
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SOURCE Milestone Pharmaceuticals, Inc.