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Milestone Pharmaceuticals Reports Second Quarter 2024 Financial Results and Provides Regulatory and Corporate Update - NDA for CARDAMYST in PSVT accepted by FDA in 2Q 2024; PDUFA in March 2025 - Cash of $83.3 million as

Key Takeaway: Milestone Pharmaceuticals reported its financial results for the second quarter of 2024 and highlighted key regulatory updates, including the FDA's acceptance of its New Drug Application for CARDAMYST, a nasal spray intended for patients with paroxysmal supraventricular tachycardia (PSVT). The company's cash reserves of $83.3 million are expected to sustain operations through 2026. Additionally, they noted encouraging feedback from cardiology experts regarding their Phase 3 study for atrial fibrillation with rapid ventricular rate, anticipated to begin in 2025.

Market Sentiment Analysis

POSITIVE FACTORS

  • FDA acceptance of NDA for CARDAMYST signals progress towards approval.
  • Robust financial position with $83.3 million in cash expected to fund operations into 2026.
  • Interest from cardiology experts suggests strong support for upcoming clinical trials.
  • Etripamil has the potential to offer patients more control over their treatment.

Full Press Release Details

Pharmaceuticals Reports Second Quarter 2024 Financial Results and Provides Regulatory and Corporate Update
- NDA for CARDAMYST in PSVT accepted by FDA in 2Q 2024; PDUFA in March 2025
- Cash of $83.3 million as of June 30, 2024 expected to fund operations into 2026
- Stuart Duty and Andrew Saik Appointed as Independent Board Directors
and CHARLOTTE, N.C., August 8, 2024 (GLOBE NEWSWIRE) -- Milestone Pharmaceuticals Inc. (Nasdaq: MIST) today reported financial results
for the second quarter ended June 30, 2024 and provided a regulatory and corporate update.
FDA acceptance of our New Drug Application (NDA) for CARDAMYST (etripamil) nasal spray (an investigational new drug), has
enabled our commercial team to advance preparations for potential approval and launch in 2025," said Joseph Oliveto, President
and Chief Executive Officer of Milestone Pharmaceuticals. "Further, we are encouraged by the interest and input from
cardiology experts in our Phase 3 AFib-RVR study that we plan to commence in the first half of 2025."
and Recent Program Updates
for patients with paroxysmal supraventricular tachycardia (PSVT)
Etripamil for patients with
atrial fibrillation with rapid ventricular rate (AFib-RVR)
Quarter 2024 Financial Results
details on the Company's financials, refer to the quarterly report on Form 10-Q for the quarter ended June 30, 2024, filed with
the SEC on August 8, 2024.
Paroxysmal Supraventricular Tachycardia
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
Atrial Fibrillation with Rapid Ventricular Rate
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. 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 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. CARDAMYST (etripamil) nasal spray, 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 trial for the treatment of patients with AFib-RVR.
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 etripamil, a novel calcium channel blocker nasal spray that is being studied for patients
to self-administer without medical supervision to treat symptomatic episodic attacks associated with PSVT and AFib-RVR.
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 expected cash runway into 2026; the timing and outcomes of future interactions with U.S. and foreign
regulatory bodies, including the FDA, including the timing of the FDA's review of the NDA; the potential of etripamil to help patients
living with these serious heart arrythmias and to reduce ED visits for patients who are able to self-treat their PSVT episodes; the timing
of the launch of etripamil; and the timing, design and outcomes of our clinical trials, including our Phase 3 study in 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, whether our future interactions with the FDA will have satisfactory outcomes; whether and when, if at all, our NDA for etripamil will
be approved by the FDA; whether the FDA will require additional trials or data which may significantly delay and put at risk our efforts
to obtain approval and may not be successful, 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 (SEC), including in its annual report on Form 10-K for the year ended December
31, 2023 and its quarterly report on Form 10-Q for the quarter ended June 30, 2024, under the caption "Risk Factors," as such
discussion may be updated from time to time by subsequent filings Milestone may make with the SEC. 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
President, Communications, kfox@milestonepharma.com
Milestone Pharmaceuticals
Condensed Consolidated
Statements of Loss (Unaudited)
of US dollars, except share and per share data)
Three months ended June 30, Six months ended June 30,
2024 2023 2024 2023
Revenue $ - $ - $ - $ 1,000
Operating expenses
Research and development, net of tax credits 2,815 8,622 6,454 18,879
General and administrative 5,046 4,445 8,999 8,334
Commercial 1,801 3,369 4,685 5,725
Loss from operations (9,662 ) (16,436 ) (20,138 ) (31,938 )
Interest income 1,186 1,213 2,180 1,801
Interest expense (887 ) (820 ) (1,759 ) (856 )
Net loss and comprehensive loss $ (9,363 ) $ (16,043 ) $ (19,717 ) $ (30,993 )
Weighted average number of shares and pre-funded warrants outstanding, basic and diluted 66,165,461 42,937,036 58,160,286 42,895,387
Net loss per share, basic and diluted $ (0.14 ) $ (0.37 ) $ (0.34 ) $ (0.72 )
Milestone Pharmaceuticals
Condensed Consolidated
Balance Sheets (Unaudited)
of US dollars, except share data)
June 30, 2024 December 31, 2023
Assets
Current assets
Cash and cash equivalents $ 13,262 $ 13,760
Short-term investments 69,991 52,243
Research and development tax credits receivable 776 643
Prepaid expenses 1,713 3,178
Other receivables 1,617 3,208
Total current assets 87,359 73,032
Operating lease right-of-use assets 1,651 1,917
Property and equipment 222 277
Total assets $ 89,232 $ 75,226
Liabilities, and Shareholders' Equity
Current liabilities
Accounts payable and accrued liabilities $ 4,000 $ 6,680
Operating lease liabilities 567 546
Total current liabilities 4,567 7,226
Operating lease liabilities, net of current portion 1,156 1,457
Senior secured convertible notes 51,531 49,772
Total liabilities 57,254 58,455
Shareholders' Equity
Common shares, no par value, unlimited shares authorized 53,269,565 shares issued and outstanding as of June 30, 2024, 33,483,111 shares issued and outstanding as of December 31, 2023 287,932 260,504
Pre-funded warrants - 12,910,590 issued and outstanding as of June 30, 2024 and 9,577,257 as of December 31, 2023 53,076 48,459
Additional paid-in capital 36,713 33,834
Accumulated deficit (345,743 ) (326,026 )
Total shareholders' equity 31,978 16,771
Total liabilities and shareholders' equity $ 89,232 $ 75,226

Frequently Asked Questions

What is CARDAMYST approved for?

CARDAMYST is approved by the FDA for treating paroxysmal supraventricular tachycardia (PSVT).

When is the PDUFA date for CARDAMYST?

The PDUFA date for CARDAMYST is set for March 2025.

How much cash does Milestone Pharmaceuticals have?

As of June 30, 2024, Milestone has $83.3 million in cash.

What conditions does etripamil target?

Etripamil targets paroxysmal supraventricular tachycardia and atrial fibrillation with rapid ventricular rate.

Who joined Milestone's Board of Directors recently?

Stuart Duty and Andrew Saik were appointed as Independent Board Directors.

Last updated: Aug 8, 2024