- NDA submission for etripamil in
patients with PSVT currently on track for 3Q23
- Company to host virtual KOL event
focused on etripamil for the potential treatment of AFib-RVR on
Monday, May 22, 2023
- Topline data from Phase 2 ReVeRA study
evaluating etripamil in patients with AFib-RVR expected in 2H,
2023
- Cash resources as of March 31, 2023, together with strategic
March 2023 financing, expected to
fund operations into mid-2025
MONTREAL and CHARLOTTE,
N.C., May 11, 2023 /PRNewswire/ -- Milestone
Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company
focused on the development and commercialization of innovative
cardiovascular medicines, today reported financial results for the
first quarter ended March 31, 2023,
and provided a clinical and corporate update.
"With a New Drug Application (NDA) submission currently on track
for the third quarter of this year and the expansion of our market
preparation activities, we believe we are well positioned to
execute on our mission of establishing etripamil as the first
fast-acting, patient-administered treatment for paroxysmal
supraventricular tachycardia (PSVT)," said Joseph Oliveto, President and Chief Executive
Officer of Milestone Pharmaceuticals. "In parallel, we increased
our efforts toward assessing the potential application of etripamil
in patients experiencing atrial fibrillation with rapid ventricular
rate (AFib-RVR) and we are making significant progress in our Phase
2 ReVeRA program which we expect to deliver topline data from later
this year. We look forward to hosting a virtual KOL event later
this month highlighting this important therapeutic area with data
from a subset of patients with AFib-RVR in the NODE-303 study."
Recent Program Updates
Etripamil for PSVT
- NDA Submission for Etripamil Nasal Spray in Patients with
PSVT Currently on Track for the Third Quarter of 2023. In
March 2023, Milestone announced
completion and database closures of the NODE-303 open-label safety
and RAPID extension studies of etripamil, the Company's
investigational calcium channel blocker that is administered by
patients outside of the healthcare setting, in patients with PSVT.
Data from these studies are being included in the PSVT NDA for
etripamil which is currently expected to be submitted to the FDA in
the third quarter of 2023. The Company believes feedback received
from the FDA supports the proposed approach for the NDA.
- Data Highlighting Decreased Emergency Department (ED)
Utilization in Patients with PSVT Treated with Etripamil Featured
at the Professional Society for Health Economics and Outcomes
Research (ISPOR) Annual Meeting. In May
2023, data highlighting decreased ED utilization in patients
treated with etripamil in the Phase 3 NODE-301 Part 1 and RAPID
studies were featured during a poster session at the ISPOR Annual
Meeting. The previously presented data showed that relative to
placebo, etripamil was associated with a significant reduction in
use of the ED for medical intervention (14% of etripamil patients
vs. 22% of placebo patients in the prespecified RAPID and NODE-301
Part 1 pooled analysis), reflecting a 39% relative risk
reduction.
Etripamil for AFib-RVR
- Data from a Subset of Patients with AFib-RVR to be Presented
at Heart Rhythm 2023 Annual Meeting. Data from a subset of
patients with AFib-RVR in the NODE-303 study, which evaluated
etripamil in patients with PSVT, will be featured during an oral
session at the Heart Rhythm 2023 Annual Meeting: "Effect of
Etripamil Nasal Spray on Ventricular Rate in Patients Experiencing
Symptomatic Atrial Fibrillation," on Friday, May 19, 2023 at 3:00 p.m. CT.
- Company to Host Virtual Key Opinion Leader Event on
Etripamil for the Treatment of AFib-RVR. Milestone will host a
virtual KOL event on Monday, May 22,
2023 at 8:00 a.m. ET to
discuss etripamil for the potential treatment of patients with
AFib-RVR. The event will feature a review of data being featured at
the upcoming Heart Rhythm 2023 Annual Meeting, an overview of
AFib-RVR, including the current treatment landscape and etripamil
characteristics, and commentary on next steps for the etripamil
development program in this potential indication. For details on
how to access the live event or replay, please visit
https://investors.milestonepharma.com/events-and-presentations.
- Topline data from ReVeRA Phase 2 Proof-of-Concept Trial in
Patients with AFib-RVR Expected in the Second Half of 2023.
Enrollment continues 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 are 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 second half of 2023.
Recent Corporate Updates
- Announced $125 Million
Strategic Financing with RTW Investments to Support the Advancement
of Etripamil for PSVT. In March
2023, Milestone announced that it entered into a
$125 million strategic financing with
existing shareholder, RTW Investments, LP and certain of its
affiliates ("RTW"). RTW has extended its investment relationship
with Milestone by purchasing $50
million of senior secured convertible promissory notes in a
private placement transaction. In addition, under the terms of a
purchase and sale agreement, following FDA approval of etripamil
and subject to certain conditions, RTW provided a $75 million non-dilutive synthetic royalty
financing commitment to Milestone in return for tiered rate, cash
royalty payments based on aggregate net sales of etripamil within
the United States.
- Seth H.Z. Fischer Appointed to Board of Directors. In
March 2023, Milestone appointed
Seth H.Z. Fischer to the Company's
Board of Directors. Mr. Fischer brings deep operational and
commercial expertise, gained through over 40 years in the
pharmaceutical and medical device industry. He previously served as
the chief executive officer and as a director of Vivus, Inc. Prior
to Vivus, Mr. Fischer served in senior positions of increasing
responsibility at Johnson & Johnson, most recently as company
group chairman, Johnson & Johnson and worldwide franchise
chairman of Cordis Corporation. Mr. Fischer also currently serves
on the boards of Agile Therapeutics, Inc., Marinus Pharmaceuticals,
Inc., Spectrum Pharmaceuticals, Inc., and Esperion
Therapeutics.
First Quarter 2023 Financial Results
- As of March 31, 2023, Milestone
had cash, cash equivalents, and short-term investments of
$101.0 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 March 31,
2023, together with strategic March
2023 financing, expected to fund operations into
mid-2025.
- Research and development expense for the first quarter of 2023
was $10.3 million, compared with
$8.8 million for the prior year
period. The increase was related to an increase in clinical
expenses.
- General and administrative expense for the first quarter of
2023 was $3.9 million, compared with
$3.6 million for the prior year
period. The increase was related to increases in personnel-related
costs and consulting fees for general and administrative
expenses.
- Commercial expense for the first quarter of 2023 was
$2.4 million, compared with
$1.6 million for the prior year
period. The increase was related to an increase in personnel as
well as professional costs required to expand operations in
anticipation of the potential market approval and
commercialization.
- For the first quarter of 2023, operating loss was $15.0 million, compared to $14.0 million for 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, 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 soon to be submitted in the third
quarter of 2023 in paroxysmal supraventricular tachycardia (PSVT).
Milestone also has a Phase 2 proof-of-concept trial that is ongoing
in patients with atrial fibrillation with rapid ventricular rate
(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 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 clinical trial data from
the Phase 2 ReVeRA program later this year; 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.
|
Consolidated
Statements of Loss
|
(in thousands of
US dollars, except share and per share data)
|
|
|
Three months ended
March 31,
|
|
|
|
2023
|
|
2022
|
|
|
|
|
|
|
|
|
|
Revenue
|
|
$
|
1,000
|
|
$
|
—
|
|
|
|
|
|
|
|
|
|
Operating
expenses
|
|
|
|
|
|
|
|
Research and
development, net of tax credits
|
|
|
10,257
|
|
|
8,768
|
|
General and
administrative
|
|
|
3,889
|
|
|
3,643
|
|
Commercial
|
|
|
2,356
|
|
|
1,636
|
|
|
|
|
|
|
|
|
|
Loss from
operations
|
|
|
(15,502)
|
|
|
(14,047)
|
|
|
|
|
|
|
|
|
|
Interest income,
net
|
|
|
552
|
|
|
40
|
|
|
|
|
|
|
|
|
|
Net loss and
comprehensive loss
|
|
$
|
(14,950)
|
|
$
|
(14,007)
|
|
|
|
|
|
|
|
|
|
Weighted average
number of shares and pre-funded warrants outstanding, basic and
diluted
|
|
|
42,853,275
|
|
|
42,243,021
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.35)
|
|
$
|
(0.33)
|
|
|
The accompanying notes
are an integral part of these interim condensed consolidated
financial statements.
|
Milestone
Pharmaceuticals Inc.
|
Consolidated Balance
Sheets
|
(in thousands of
US dollars, except share data)
|
|
|
|
March 31, 2023
|
|
December 31, 2022
|
Assets
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
73,953
|
|
$
|
7,636
|
Short-term
investments
|
|
|
27,000
|
|
|
56,949
|
License
receivable
|
|
|
1,000
|
|
|
—
|
Research and
development tax credits receivable
|
|
|
414
|
|
|
331
|
Prepaid
expenses
|
|
|
5,447
|
|
|
6,005
|
Other
receivables
|
|
|
694
|
|
|
882
|
Total current
assets
|
|
|
108,508
|
|
|
71,803
|
Operating lease
assets
|
|
|
2,300
|
|
|
2,423
|
Property and
equipment
|
|
|
272
|
|
|
257
|
Total
assets
|
|
$
|
111,080
|
|
$
|
74,483
|
|
|
|
|
|
|
|
Liabilities, and
Shareholders' Equity
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
Accounts payable and
accrued liabilities
|
|
$
|
7,423
|
|
$
|
5,644
|
Operating lease
liabilities
|
|
|
507
|
|
|
495
|
Interest
Payable
|
|
|
33
|
|
|
—
|
Total current
liabilities
|
|
|
7,963
|
|
|
6,139
|
Operating lease
liabilities, net of current portion
|
|
|
1,862
|
|
|
1,996
|
Senior secured
convertible notes
|
|
|
47,320
|
|
|
—
|
Total
liabilities
|
|
|
57,145
|
|
|
8,135
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Shareholders'
Equity
|
|
|
|
|
|
|
Common shares, no par
value, unlimited shares authorized 33,337,214 shares
issued and outstanding as of March 31, 2023, 34,286,002
shares issued and
outstanding as of December 31, 2022
|
|
|
260,126
|
|
|
273,900
|
Pre-funded warrants -
9,577,257 issued and outstanding as of March 31, 2023
and 8,518,257 as of December 31, 2022
|
|
|
48,459
|
|
|
34,352
|
Additional paid-in
capital
|
|
|
26,641
|
|
|
24,437
|
Accumulated
deficit
|
|
|
(281,291)
|
|
|
(266,341)
|
|
|
|
|
|
|
|
Total shareholders'
equity
|
|
|
53,935
|
|
|
66,348
|
|
|
|
|
|
|
|
Total liabilities
and shareholders' equity
|
|
$
|
111,080
|
|
$
|
74,483
|
|
The accompanying notes
are an integral part of these interim condensed consolidated
financial statements.
|
Contact:
David
Pitts
Argot Partners
212-600-1902
milestone@argotpartners.com
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SOURCE Milestone Pharmaceuticals, Inc.