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Milestone Pharmaceuticals Reports Third Quarter 2023 Financial Results and Provides Clinical and Corporate Updates - New Drug Application submitted to the U.S. Food and Drug Administration for CARDAMYST , the conditional

Key Takeaway: Milestone Pharmaceuticals Inc. has reported its financial results for Q3 2023 and announced the submission of its first New Drug Application for CARDAMYST, aimed at treating Paroxysmal Supraventricular Tachycardia (PSVT). The company highlighted positive results from a Phase 2 study of etripamil for patients with Atrial Fibrillation with Rapid Ventricular Rate (AFib-RVR). Moving forward, Milestone is confident about its lead asset, etripamil, and its clinical development strategy as they approach 2024.

Market Sentiment Analysis

POSITIVE FACTORS

  • Milestone submitted its first New Drug Application for CARDAMYST.
  • Positive results from the Phase 2 study of etripamil for AFib-RVR.
  • The company has a well-defined strategy and strong confidence in etripamil's potential.

CONCERNS & RISKS

  • Risks associated with the regulatory approval process for etripamil.
  • Financial market uncertainties that could affect capital-raising efforts.

Full Press Release Details

Milestone Pharmaceuticals
Reports Third Quarter 2023 Financial Results and Provides Clinical and Corporate Updates
Montreal and Charlotte,
N.C., November 13, 2023 -- Milestone Pharmaceuticals Inc. (Nasdaq: MIST) today reported financial results for
the third quarter ended September 30, 2023, and provided a clinical and corporate update.
was transformational for our company. We believe the combination of the submission of our first New Drug Application for CARDAMYST in
treating PSVT and the release of positive results from our Phase 2 study evaluating etripamil to treat patients with AFib-RVR, demonstrates
that we are on track to advance our clinical development program," said Joseph Oliveto, President, and Chief Executive Officer of
Milestone Pharmaceuticals. "We are approaching 2024 with a well-defined strategy and even greater confidence around the potential
of our lead asset, etripamil, to help people living with these serious heart arrythmias."
Recent Program Updates
Etripamil for Patients with AFib-RVR
Investor and Analyst Conference Call and Webcast
The Company will host an investor and analyst
conference call and webcast on Monday, November 13, 2023, at 8:00 a.m. Eastern Time. The event will feature a review of the ReVeRA data,
an overview of AFib-RVR and current treatment landscape, characteristics of etripamil, and commentary on next steps for Milestone's clinical
development program for etripamil. To join the live call by phone, dial (877) 870-4263 (domestic) or (412) 317-0790 (international) and
ask to be connected to the Milestone Pharmaceuticals call. To access the live or recorded webcast and accompanying slides, please visit
the News & Events section of Milestone's investor relations website at investors.milestonepharma.com.
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 health care
providers 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 Atrial Fibrillation
with Rapid Ventricular Rate (AFib-RVR)
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 2030.1,2,3 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 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 elevated and often highly
symptomatic heart-rate attacks associated with PSVT and 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 on demand care and patient self-management. If approved, the 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 proof-of-concept trial for the treatment
of patients with AFib-RVR.
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 a Phase 2 proof-of-concept trial for the treatment of patients with AFib-RVR. The AFib-RVR study, ReVeRA, was recently presented
as a Featured Science presentation at the American Heart Association (AHA) Scientific Sessions 2023 and simultaneously published in Circulation:
Arrhythmia and Electrophysiology. Milestone Pharmaceuticals operates in Canada and the United States. Find
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 etripamil's potential to help patients living with these serious heart arrythmias;
etripamil's continued ability to achieve statistically superior ventricular rate reduction and improved symptom-relief when compared
to placebo; the timing and outcome of the FDA determination of whether the NDA is complete and accepted for review; the timing of the
FDA's potential review of the NDA; the timing of the launch of etripamil; the clinical benefit of etripamil for self-treating recurrent
episodes of PSVT without medical supervision; the timing of a Phase 3 registrational program for etripamil; and the anticipated growth
in incidences of AFib by 2025 and 2030 and the target addressable market of patients with AFib-RVR by 2030. 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, 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, 2022, 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.
Milestone Pharmaceuticals Inc.
Condensed Consolidated Statements of Loss (Unaudited)
(in thousands of US dollars, except share
Three months ended September 30, Nine months ended September 30,
2023 2022 2023 2022
Revenue $ - $ 1,500 $ 1,000 $ 1,500
Operating expenses
Research and development, net of tax credits 6,721 9,826 25,600 29,251
General and administrative 4,227 4,034 12,561 11,595
Commercial 4,412 2,670 10,137 6,537
Loss from operations (15,360 ) (15,030 ) (47,298 ) (45,883 )
Interest income 1,120 474 2,921 672
Interest expense (841 ) - (1,697 ) -
Net loss and comprehensive loss $ (15,081 ) $ (14,556 ) $ (46,074 ) $ (45,211 )
Weighted average number of shares and pre-funded warrants outstanding, basic and diluted 42,973,160 42,491,787 42,920,620 42,339,123
Net loss per share, basic and diluted $ (0.35 ) $ (0.34 ) $ (1.07 ) $ (1.07 )
Milestone Pharmaceuticals Inc.
Condensed Consolidated Balance Sheets (Unaudited)
(in thousands of US dollars, except share
September 30, 2023 December 31, 2022
Assets
Current assets
Cash and cash equivalents $ 9,879 $ 7,636
Short-term investments 65,867 56,949
Research and development tax credits receivable 569 331
Prepaid expenses 6,961 6,005
Other receivables 1,521 882
Total current assets 84,797 71,803
Operating lease assets 2,047 2,423
Property and equipment 272 257
Total assets $ 87,116 $ 74,483
Liabilities, and Shareholders' Equity
Current liabilities
Accounts payable and accrued liabilities $ 7,584 $ 5,644
Operating lease liabilities 530 495
Total current liabilities 8,114 6,139
Operating lease liabilities, net of current portion 1,583 1,996
Senior secured convertible notes 48,915 -
Total liabilities 58,612 8,135
Shareholders' Equity
Common shares, no par value, unlimited shares authorized 33,481,787 shares issued and outstanding as of September 30, 2023, 34,286,002 shares issued and outstanding as of December 31, 2022 260,502 273,900
Pre-funded warrants - 9,577,257 issued and outstanding as of September 30, 2023 and 8,518,257 as of December 31, 2022 48,459 34,352
Additional paid-in capital 31,958 24,437
Accumulated deficit (312,415 ) (266,341 )
Total shareholders' equity 28,504 66,348
Total liabilities and shareholders' equity $ 87,116 $ 74,483
Kim Fox, Vice President, Communications

Frequently Asked Questions

What are Milestone Pharmaceuticals' recent updates?

Milestone has submitted its first New Drug Application for CARDAMYST and reported positive Phase 2 results for etripamil in AFib-RVR.

What conditions does etripamil target?

Etripamil is under development for Paroxysmal Supraventricular Tachycardia (PSVT) and Atrial Fibrillation with Rapid Ventricular Rate (AFib-RVR).

How will etripamil improve patient care?

If approved, etripamil will enable patients to manage their symptoms independently with a self-administered nasal spray.

When is the Milestone conference call scheduled?

The investor conference call is set for November 13, 2023, at 8:00 a.m. Eastern Time.

What is the prevalence of AFib in the U.S.?

Approximately five million Americans are affected by Atrial Fibrillation, with numbers expected to rise significantly by 2030.

Last updated: Nov 13, 2023