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Milestone Pharmaceuticals Reports Second Quarter 2025 Financial Results and Provides Regulatory and Corporate Update FDA Accepted the Company's Response to the CRL for CARDAMYST (etripamil) Nasal Spray; New PDUFA Target

Key Takeaway: Milestone Pharmaceuticals has reported its second-quarter financial results for 2025 alongside important regulatory updates. The FDA has accepted their response to the Complete Response Letter (CRL) for the CARDAMYST (etripamil) nasal spray, which targets PSVT, with a new PDUFA date set for December 13, 2025. Milestone has also secured significant funding through public offerings and royalty agreements, enhancing its financial stability and operational capacity. However, the company still faces ongoing net losses and uncertainty regarding future regulatory approvals.

Market Sentiment Analysis

POSITIVE FACTORS

  • FDA accepted the response to CRL for CARDAMYST, indicating progress towards approval.
  • Public offering raised gross proceeds of up to $170 million, strengthening the company's balance.
  • CARDAMYST is designed for self-administration, promising improved patient care for PSVT and AFib-RVR.

CONCERNS & RISKS

  • The company continues to report net losses, indicating financial challenges.
  • Regulatory approval timeline remains uncertain, with PDUFA target set for December 2025.

Full Press Release Details

Milestone Pharmaceuticals
Reports Second Quarter 2025 Financial Results and Provides Regulatory and Corporate Update
FDA Accepted the Company's Response to
the CRL for CARDAMYST (etripamil) Nasal Spray; New PDUFA Target Date of December 13, 2025
Milestone Strengthens Balance Sheet to Fully
Commercialize CARDAMYST if Approved
Public Offering Raised Total Gross Proceeds
of up to $170 Million if all Warrants are Exercised
$75 Million Royalty Purchase Agreement Payment
from RTW Extended Through End of 2025
MONTREAL and CHARLOTTE, N.C., August 12,
2025 (GLOBE NEWSWIRE) -- Milestone Pharmaceuticals Inc. (Nasdaq: MIST) today reported financial results for the second quarter
ended June 30, 2025 and provided corporate and regulatory updates.
"With the FDA's recent acceptance
of our response to the CRL, our team is energized as we work toward the potential approval of CARDAMYST in its first indication, PSVT,"
said Joe Oliveto, President and Chief Executive Officer of Milestone. "In parallel to our regulatory progress, we completed
an equity financing in July with high-quality investors which strengthened our balance sheet and extended our operating runway. Our
goal is to make CARDAMYST quickly available to PSVT patients, should the FDA grant approval this year."
Second Quarter and Recent Program Updates
for patients with PSVT
for patients with atrial fibrillation with rapid ventricular rate (AFib-RVR)
Second Quarter and Recent Corporate Updates
on the Company's financials, refer to the Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, filed with
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 approval of the NDA for CARDAMYST for PSVT; Milestone's
ability to receive additional cash proceeds from the warrants issued in the Offering; Milestone's ability to receive the $75.0 million
royalty payment under the Royalty Purchase Agreement on the timeline provided, or at all; Milestone's expected operating runway;
CARDAMYST's potential as a novel treatment option to help patients with PSVT; Milestone's ability to make CARDAMYST quickly
available to PSVT patients following FDA approval, if received; the success of Milestone's launch infrastructure; 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 June 30, 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.
Milestone Pharmaceuticals Inc.
Condensed Consolidated Balance Sheets (Unaudited)
(in thousands of US dollars, except share
June 30, 2025 December 31, 2024
Assets
Current assets
Cash and cash equivalents $ 42,499 $ 25,314
Short-term investments 918 44,381
Research and development tax credits receivable 1,079 901
Prepaid expenses 748 1,840
Other receivables 924 1,490
Total current assets 46,168 73,926
Operating lease right-of-use assets 1,090 1,376
Property and equipment 159 197
Total assets $ 47,417 $ 75,499
Liabilities, and Shareholders' (Deficit) Equity
Current liabilities
Accounts payable and accrued liabilities $ 8,768 $ 7,555
Operating lease liabilities 515 571
Total current liabilities 9,283 8,126
Operating lease liabilities, net of current portion 641 874
Senior secured convertible notes 55,238 53,352
Total liabilities 65,162 62,352
Shareholders' (Deficit) Equity
Common shares, no par value, unlimited shares authorized, 53,494,261 shares issued and outstanding as of June 30, 2025, 53,353,984 shares issued and outstanding as of December 31, 2024 288,263 288,048
Pre-funded warrants - 12,910,590 issued and outstanding as of June 30, 2025 and 12,910,590 as of December 31, 2024 53,076 53,076
Additional paid-in capital 42,188 39,568
Accumulated deficit (401,272 ) (367,545 )
Total shareholders' (deficit) equity (17,745 ) 13,147
Total liabilities and shareholders' equity $ 47,417 $ 75,499
Milestone Pharmaceuticals Inc.
Condensed Consolidated Statements of Loss (Unaudited)
(in thousands of US dollars, except share
Three months ended June 30, Six months ended June 30,
2025 2024 2025 2024
Revenue $ - $ - $ - $ -
Operating expenses
Research and development, net of tax credits 3,669 2,815 8,647 6,454
General and administrative 3,759 5,046 8,926 8,999
Commercial 5,103 1,801 15,481 4,685
Loss from operations (12,531 ) (9,662 ) (33,054 ) (20,138 )
Interest income 516 1,186 1,213 2,180
Interest expense (951 ) (887 ) (1,886 ) (1,759 )
Net loss and comprehensive loss $ (12,966 ) $ (9,363 ) $ (33,727 ) $ (19,717 )
Weighted average number of shares and pre-funded warrants outstanding, basic and diluted 66,380,118 66,165,461 66,333,024 58,160,286
Net loss per share, basic and diluted $ (0.20 ) $ (0.14 ) $ (0.51 ) $ (0.34 )

Frequently Asked Questions

What is CARDAMYST?

CARDAMYST is a nasal spray containing etripamil, designed for self-administration to treat PSVT symptoms.

What is the new PDUFA target date for CARDAMYST?

The new PDUFA target date for CARDAMYST is December 13, 2025.

How much did Milestone raise in its public offering?

Milestone raised total gross proceeds of up to $170 million, if all warrants are exercised.

What conditions does etripamil aim to treat?

Etripamil targets symptomatic episodes of PSVT and atrial fibrillation with rapid ventricular rate.

What did Milestone report for the second quarter of 2025?

Milestone reported a net loss of $12.966 million for the second quarter of 2025.

Last updated: Aug 12, 2025