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Milestone Pharmaceuticals Reports First Quarter 2025 Financial Results and Provides Regulatory and Corporate Update Resolution of CRL Manufacturing issues in progress - Type A meeting requested No clinical safety or effi

Key Takeaway: Milestone Pharmaceuticals has reported its financial results for the first quarter of 2025 and provided updates on its regulatory status. The company is currently addressing issues related to a Complete Response Letter (CRL) from the FDA for its product, CARDAMYST, used for treating Paroxysmal Supraventricular Tachycardia (PSVT). A Type A meeting with the FDA has been requested to help resolve these manufacturing concerns. The company's lead investigational product, Etripamil, is noted for its innovative self-administration potential, although the financial report reflects an increased net loss compared to the prior year.

Market Sentiment Analysis

POSITIVE FACTORS

  • Milestone is actively working with the FDA to resolve CRL issues.
  • The company is confident about the approval of CARDAMYST as it may become a unique treatment for PSVT.
  • No safety or efficacy concerns have been raised in clinical studies so far.

CONCERNS & RISKS

  • The company faced challenges with CMC-related issues that led to the CRL.
  • There are uncertainties regarding the outcomes of future interactions with the FDA.
  • Milestone's recent financials show an increased net loss compared to the previous year.

Full Press Release Details

Milestone Pharmaceuticals
Reports First Quarter 2025 Financial Results and Provides Regulatory and Corporate Update
Resolution of CRL Manufacturing issues in progress
- Type A meeting requested
No clinical safety or efficacy concerns
MONTREAL and CHARLOTTE, N.C., May
14, 2025 (GLOBE NEWSWIRE) -- Milestone Pharmaceuticals Inc. (Nasdaq: MIST) today reported financial results for the first quarter
ended March 31, 2025. The Company also announced the submission of a meeting request to the U.S. Food and Drug Administration (FDA)
as the next step in the resolution of CRL issues.
"Our immediate priority is to engage with
the U.S. FDA in order to address the CMC-related issues raised in the CRL received for CARDAMYST as a treatment for PSVT,"
said Joe Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "We are confident we can work with
the FDA to fully respond to the CRL and remain committed to the potential of CARDAMYST. If approved, it will be the first and only self-administered
therapy for the rapid termination of episodes of PSVT."
First Quarter and Recent Program Updates
for Patients with PSVT
for patients with atrial fibrillation with rapid ventricular rate (AFib-RVR)
on the Company's financials, refer to the Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, filed with the SEC.
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 , 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
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.
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: the outcomes of future interactions with the FDA, including the potential Type A meeting; Milestone's ability to address
the issues raised in the CRL on a timely basis, if at all; the outcome of the potential NDA resubmission; CARDAMYST's potential
as a novel treatment option to help patients with PSVT; potential protections afforded by U.S. patents; CARDAMYST's ability to make
the biggest difference in patient health, as compared to other available treatment options; the timing of patient enrollment in the Phase
3 study of etripamil for AFib-RVR; and other statements not related to historical facts. 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; 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, international tariffs, 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, 2025 and its quarterly report on Form 10-Q for the quarter ended March 31, 2025, in each case under the caption "Risk Factors,"
as such discussions 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 becomes available.
Kim Fox, Vice President, Communications, kfox@milestonepharma.com
Milestone Pharmaceuticals Inc.
Condensed Consolidated Balance Sheets (Unaudited)
(in thousands of US dollars, except share
March 31, 2025 December 31, 2024
Assets
Current assets
Cash and cash equivalents $ 45,085 $ 25,314
Short-term investments 10,873 44,381
Research and development tax credits receivable 994 901
Prepaid expenses 2,356 1,840
Other receivables 1,167 1,490
Total current assets 60,475 73,926
Operating lease right-of-use assets 1,234 1,376
Property and equipment 176 197
Total assets $ 61,885 $ 75,499
Liabilities, and Shareholders' (Deficit) Equity
Current liabilities
Accounts payable and accrued liabilities $ 12,421 $ 7,555
Operating lease liabilities 542 571
Total current liabilities 12,963 8,126
Operating lease liabilities, net of current portion 758 874
Senior secured convertible notes 54,287 53,352
Total liabilities 68,008 62,352
Shareholders' (Deficit) Equity
Common shares, no par value, unlimited shares authorized, 53,464,273 shares issued and outstanding as of March 31, 2025, 53,353,984 shares issued and outstanding as of December 31, 2024 288,188 288,048
Pre-funded warrants - 12,910,590 issued and outstanding as of March 31, 2025 and 12,910,590 as of December 31, 2024 53,076 53,076
Additional paid-in capital 40,919 39,568
Accumulated deficit (388,306 ) (367,545 )
Total shareholders' (deficit) equity (6,123 ) 13,147
Total liabilities and shareholders' equity $ 61,885 $ 75,499
Milestone Pharmaceuticals Inc.
Condensed Consolidated Statements of Loss (Unaudited)
(in thousands of US dollars, except share
Three months ended March 31,
2025 2024
Revenue $ - $ -
Operating expenses
Research and development, net of tax credits 4,978 3,639
General and administrative 5,167 3,953
Commercial 10,378 2,884
Loss from operations (20,523 ) (10,476 )
Interest income 697 994
Interest expense (935 ) (872 )
Net loss and comprehensive loss $ (20,761 ) $ (10,354 )
Weighted average number of shares and pre-funded warrants outstanding, basic and diluted 66,285,406 50,155,111
Net loss per share, basic and diluted $ (0.31 ) $ (0.21 )

Frequently Asked Questions

What is Milestone Pharmaceuticals' main investigational product?

Milestone Pharmaceuticals' lead investigational product is etripamil, a calcium channel blocker nasal spray.

What condition does CARDAMYST aim to treat?

CARDAMYST is designed to treat paroxysmal supraventricular tachycardia (PSVT).

What recent regulatory step has Milestone Pharmaceuticals taken?

Milestone submitted a meeting request to the FDA to address CRL manufacturing issues.

How does etripamil provide patient care?

Etripamil is designed for self-administration, allowing patients to manage their symptoms without medical supervision.

What are the financial results for Milestone in Q1 2025?

Milestone reported a net loss of $20.8 million for the first quarter of 2025.

Last updated: May 14, 2025