- NDA submission for etripamil in patients with PSVT expected
in October 2023
- Data featured during oral session at Heart Rhythm 2023
Annual Meeting support the potential of etripamil in patients with
AFib-RVR
- Enrollment complete in Phase 2 ReVeRA study of etripamil in
patients with AFib-RVR; topline data expected in fourth quarter of
2023
MONTREAL and CHARLOTTE,
N.C., Aug. 10, 2023 /CNW/ -- Milestone
Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company
focused on the development and commercialization of innovative
cardiovascular medicines, today reported financial results for the
second quarter ended June 30, 2023,
and provided a clinical and corporate update.
"The remainder of 2023 represents a transformative time for the
Company as we approach our first New Drug Application (NDA)
submission for etripamil and continue to lay the groundwork
required to successfully launch what could serve as the first and
only U.S. Food and Drug Administration (FDA) approved treatment for
patients to self-treat their paroxysmal supraventricular
tachycardia (PSVT)," said Joseph
Oliveto, President, and Chief Executive Officer of Milestone
Pharmaceuticals. "Expanding assessment of the potential
clinical utility of etripamil, we have completed enrollment in
ReVeRA, our Phase 2 study of etripamil in patients with atrial
fibrillation with rapid ventricular rate (AFib-RVR), and we look
forward to sharing topline data from this study in the fourth
quarter of this year."
Recent Program Updates
Etripamil for PSVT
- NDA Submission for Etripamil Nasal Spray in Patients with
PSVT Expected in October. Milestone expects to submit its first
NDA for etripamil, the Company's investigational calcium channel
blocker that is administered by patients outside of the healthcare
setting, in patients with PSVT in October. Based on feedback from
the FDA, data from the Company's previously completed global Phase
3 clinical program, including the RAPID and RAPID-extension
studies, NODE-303 and NODE-301, are expected to fulfill the safety
and efficacy requirements for the planned NDA submission.
- Results from Phase 3 RAPID Clinical Trial of Etripamil Nasal
Spray in Patients with PSVT Published in The Lancet.
Results from the Company's Phase 3 RAPID clinical study were
recently published in The Lancet. The publication,
titled "Self-administered Intranasal Etripamil Using a
Symptom-Prompted, Repeat-Dose Regimen for Atrioventricular-Nodal
Dependent Supraventricular Tachycardia: The Randomised, Controlled
RAPID Trial," can be accessed via the following link:
https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(23)00776-6/fulltext.
Etripamil for AFib-RVR
- Data Featured During Oral Session at Heart Rhythm 2023
Annual Meeting Support Potential of Etripamil in Patients with
AFib-RVR. Data from an analysis of a subset of patients with
AFib-RVR in the NODE-303 study, which evaluated etripamil in
patients with PSVT, were featured during an oral session at the
Heart Rhythm 2023 Annual Meeting in May. Data demonstrated that
self-administration of etripamil in patients experiencing AFib-RVR
episodes resulted in a substantial reduction in ventricular rate
which was sustained over 60 minutes. The safety and tolerability
data from the analysis of these AFib-RVR episodes were consistent
with those observed across prior studies in PSVT.
- Enrollment Complete in the ReVeRA Phase 2 Proof-of-Concept
Trial in Patients with AFib-RVR, With Topline Data Expected in the
Fourth Quarter of 2023. Enrollment is complete in ReVeRA,
Milestone's Phase 2 double-blind, placebo-controlled,
proof-of-concept trial of etripamil nasal spray in emergency
department patients experiencing AFib-RVR. The trial, in which
patients were 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 elevated ventricular rates in
patients with symptomatic AFib-RVR. 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. Milestone expects to report topline data
from this trial in the fourth quarter of 2023.
Second Quarter 2023 Financial Results
- As of June 30, 2023, Milestone
had cash, cash equivalents, and short-term investments of
$87.6 million and 33.4 million common
shares issued and outstanding, with an additional 9.6 million
common shares issuable upon exercise of pre-funded warrants. Cash
resources as of June 30, 2023 are
expected to fund operations into mid-2025.
- Research and development expense for the second quarter of 2023
was $8.6 million, compared with
$10.7 million for the prior year
period. For the six months ended June 30,
2023, research and development expense was $18.9 million compared with $19.4 million for the prior year period. The
decreases in research and development expenses were related to
decreases in clinical developmental costs and clinical
personnel-related costs as a result of the Company's Phase 3
studies reaching completion.
- General and administrative expense for the second quarter of
2023 was $4.4 million, compared with
$3.9 million for the prior year
period. For the six months ended June 30,
2023, general and administrative expense was $8.3 million compared with $7.6 million for the prior year period. The
increases were related to an increase in personnel-related costs
and consulting fees for general and administrative expenses.
- Commercial expense for the second quarter of 2023 was
$3.4 million, compared with
$2.2 million for the prior year
period. For the six months ended June 30,
2023, commercial expense was $5.7
million compared with $3.9
million for the prior year period. The increases were
related to additional personnel and professional costs required to
expand capabilities and operations in anticipation of potential
commercialization.
- For the second quarter of 2023, operating loss was $16.4 million, compared to $16.8 million for the prior year period. For the
six months ended June 30, 2023,
Milestone's operating loss was $31.9
million, compared to $30.9
million in the prior year period.
About Paroxysmal Supraventricular Tachycardia
Paroxysmal Supraventricular Tachycardia (PSVT) is a highly
symptomatic and impactful heart arrhythmia characterized by
unpredictable attacks of a racing heart that afflicts approximately
two million Americans. Symptoms of PSVT, including palpitations,
chest pressure, and shortness of breath are often debilitating,
causing the patient to stop their current activities or avoid
pursuits altogether. 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. The uncertainty of when an episode of SVT
will strike or how long it will persist can provoke anxiety in
patients, which can have a negative impact on their day-to-day
life. Many doctors are unsatisfied by the lack of effective
treatment options besides a prolonged, unpleasant, and costly trip
to the emergency department or, for some patients, an invasive
ablation procedure.
About Atrial Fibrillation with Rapid Ventricular Rate
Atrial fibrillation (AFib) is a common arrhythmia marked by an
irregular and often rapid heartbeat. AFib is estimated to affect
five million patients in the United
States, a prevalence projected by the Centers for Disease
Control to increase to twelve million patients by 2030. Atrial
fibrillation with rapid ventricular rate (AFib-RVR) is a condition
that many patients with AFib experience and includes episodes of
abnormally high heart rate, often with symptoms of palpitations,
shortness of breath, dizziness, and weakness. Oral calcium channel
blockers and/or beta blockers are used to reduce the heart rate in
this condition. When AFib-RVR occurs, symptoms are often burdensome
enough to cause patients to seek acute care in the emergency
department, where standard-of-care procedures include intravenous
administration of calcium channel blockers or beta blockers, or
electrical cardioversion, under medical supervision. Milestone's
initial market research indicates that 30-40% 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.
About Etripamil
Etripamil, Milestone's lead investigational product, is a novel
calcium channel blocker nasal spray. It is designed to be a
rapid-response therapy that is self-administered by the patient,
without the need for direct medical oversight, and is being
developed for elevated and often highly symptomatic heart rate
attacks associated with PSVT and AFib-RVR. If approved, etripamil
is intended to provide health care providers with a new tool to
enable virtual care and patient self-management, and to impart upon
the patient a greater sense of control over their condition.
Milestone is conducting a comprehensive development program for
etripamil, with Phase 3 trials completed and an NDA submission
expected in October 2023 in
paroxysmal supraventricular tachycardia (PSVT). Milestone also has
a Phase 2 proof-of-concept trial that has completed enrollment and
will report topline data in patients experiencing atrial
fibrillation with rapid ventricular rate (AFib-RVR) in the fourth
quarter of 2023.
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 paroxysmal
supraventricular tachycardia (PSVT) and is in a Phase 2
proof-of-concept trial for the treatment of patients with atrial
fibrillation with rapid ventricular rate (AFib-RVR). Milestone
Pharmaceuticals operates
in Canada and the United States. For more
information, visit www.milestonepharma.com and follow
Milestone on Twitter at @MilestonePharma.
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 to serve as a promising
therapy for PSVT patients; the timing of the NDA submission for
etripamil nasal spray; the potential for etripamil to serve as the
first and only FDA-approved treatment for patients to self-treat
their PSVT; the potential for clinical trial data from the Phase 2
ReVeRA program later this year; the ability for the data from the
Company's previously completed global Phase 3 clinical program,
including the RAPID and RAPID-extension studies, NODE-303, and
NODE-301, to fulfill the safety and efficacy requirements for the
planned NDA submission; and our ability to fund operations into the
middle of 2025. 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.
Milestone
Pharmaceuticals Inc.
|
Condensed
Consolidated Statements of
Loss (Unaudited)
|
(in thousands of
US dollars, except share and per share data)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three months ended
June 30,
|
|
Six months ended
June 30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue
|
|
$
|
—
|
|
$
|
—
|
|
$
|
1,000
|
|
$
|
—
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating
expenses
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development, net of tax credits
|
|
|
8,622
|
|
|
10,657
|
|
|
18,879
|
|
|
19,425
|
General and
administrative
|
|
|
4,445
|
|
|
3,918
|
|
|
8,334
|
|
|
7,561
|
Commercial
|
|
|
3,369
|
|
|
2,231
|
|
|
5,725
|
|
|
3,867
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss from
operations
|
|
|
(16,436)
|
|
|
(16,806)
|
|
|
(31,938)
|
|
|
(30,853)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest
income
|
|
|
1,213
|
|
|
158
|
|
|
1,801
|
|
|
198
|
Interest
expense
|
|
|
(820)
|
|
|
—
|
|
|
(856)
|
|
|
—
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss and
comprehensive loss
|
|
$
|
(16,043)
|
|
$
|
(16,648)
|
|
$
|
(30,993)
|
|
$
|
(30,655)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted average
number of shares and pre-funded warrants
outstanding, basic and diluted
|
|
|
42,937,036
|
|
|
42,278,563
|
|
|
42,895,387
|
|
|
42,260,682
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.37)
|
|
$
|
(0.39)
|
|
$
|
(0.72)
|
|
$
|
(0.73)
|
Milestone
Pharmaceuticals Inc.
|
Condensed
Consolidated Balance Sheets (Unaudited)
|
(in thousands of
US dollars, except share data)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
June 30, 2023
|
|
December 31, 2022
|
Assets
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
32,591
|
|
$
|
7,636
|
Short-term
investments
|
|
|
55,000
|
|
|
56,949
|
Research and
development tax credits receivable
|
|
|
483
|
|
|
331
|
Prepaid
expenses
|
|
|
5,400
|
|
|
6,005
|
Other
receivables
|
|
|
1,092
|
|
|
882
|
Total current
assets
|
|
|
94,566
|
|
|
71,803
|
Operating lease
assets
|
|
|
2,175
|
|
|
2,423
|
Property and
equipment
|
|
|
278
|
|
|
257
|
Total
assets
|
|
$
|
97,019
|
|
$
|
74,483
|
|
|
|
|
|
|
|
Liabilities, and
Shareholders' Equity
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
Accounts payable and
accrued liabilities
|
|
$
|
6,287
|
|
$
|
5,644
|
Operating lease
liabilities
|
|
|
522
|
|
|
495
|
Total current
liabilities
|
|
|
6,809
|
|
|
6,139
|
Operating lease
liabilities, net of current portion
|
|
|
1,729
|
|
|
1,996
|
Senior secured
convertible notes
|
|
|
48,073
|
|
|
—
|
Total
liabilities
|
|
|
56,611
|
|
|
8,135
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Shareholders'
Equity
|
|
|
|
|
|
|
Common shares, no par
value, unlimited shares authorized 33,363,971 shares issued and
outstanding as of June 30, 2023, 34,286,002 shares issued and
outstanding as of
December 31, 2022
|
|
|
260,169
|
|
|
273,900
|
Pre-funded warrants -
9,577,257 issued and outstanding as of June 30, 2023 and
8,518,257
as of December 31, 2022
|
|
|
48,459
|
|
|
34,352
|
Additional paid-in
capital
|
|
|
29,114
|
|
|
24,437
|
Accumulated
deficit
|
|
|
(297,334)
|
|
|
(266,341)
|
|
|
|
|
|
|
|
Total shareholders'
equity
|
|
|
40,408
|
|
|
66,348
|
|
|
|
|
|
|
|
Total liabilities
and shareholders' equity
|
|
$
|
97,019
|
|
$
|
74,483
|
Contact:
David
Pitts
Argot Partners
212-600-1902
milestone@argotpartners.com
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SOURCE Milestone Pharmaceuticals, Inc.