Milestone Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Regulatory and Corporate Update

FDA PDUFA review goal date of March 27, 2025 for CARDAMYST$(TM)$ (etripamil) nasal spray for Paroxysmal Supraventricular Tachycardia (PSVT)

Assuming FDA approval, proposed launch in PSVT targeted for mid-2025

Milestone well-capitalized to commercialize upon approval with royalty financing

MONTREAL and CHARLOTTE, N.C., March 13, 2025 (GLOBE NEWSWIRE) -- Milestone$(R)$ Pharmaceuticals Inc. (Nasdaq: MIST) today reported financial results for the fourth quarter and full year ended December 31, 2024 and provided a regulatory and corporate update.

"We are focused on the potential FDA approval for CARDAMYST and the opportunity to help the millions of patients suffering from PSVT," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "Our launch preparations are well underway. We have resources secured to make CARDAMYST available to patients and healthcare providers mid-year, assuming FDA approval."

Fourth Quarter 2024 and Recent Program Updates

CARDAMYST for patients with PSVT

   -- New Drug Application (NDA) for CARDAMYST for PSVT under review at U.S. 
      FDA Milestone is currently working to obtain U.S. Food and Drug 
      Administration (FDA) marketing approval of CARDAMYST for the treatment of 
      PSVT. The FDA accepted the NDA for CARDAMYST in May 2024 and has set a 
      Prescription Drug User Fee Act (PDUFA) review goal date for March 27, 
      2025. 
 
   -- Milestone hosted a Commercial Launch Plan investor event The event, which 
      took place in New York City on February 25, 2025, provided an in-depth 
      overview of the company's commercial strategy for CARDAMYST, if approved. 
      A replay of the event, as well as a copy of the slides, can be found on 
      the corporate website here. 
 
   -- Notice of Allowance received from the U.S. Patent and Trademark Office 
      (USPTO) on a new Method of Use patent for etripamil nasal spray The new 
      patent covers the repeat dose regimen used in the RAPID Phase 3 study 
      that evaluated CARDAMYST in PSVT and proposed for the package insert as 
      part of the CARDAMYST NDA. The issuance of the Notice of Allowance 
      potentially extends the intellectual property protection for CARDAMYST 
      in the United States until July 2042, which is an additional 6 years of 
      potential protection for the company's intellectual property portfolio. 
 
   -- Poster on Etripamil to be presented at American College of Cardiology 
      Annual Meeting (ACC.25) The poster "Consistency and Predictiveness of 
      Conversion Among Multiple Episodes of Paroxysmal Supraventricular 
      Tachycardia (PSVT) treated with Etripamil: Outcomes from the NODE-303 
      trial" is scheduled to be presented on March 30, 2025 by James Ip, M.D., 
      Division of Cardiology, Weill Cornell Medicine, New York Presbyterian 
      Hospital. 
 
   -- Milestone hosted a key opinion leader webinar entitled "Learnings from 
      the Field: Managing PSVT and Studying AFib-RVR in the Community Setting" 
      The webinar, which took place October 8, 2024, was the second of a series 
      of planned webinars learning from community-based health care providers, 
      featured Aamer H. Jamali, MD, FACC and Farhad Rafii, MD, FACC, both 
      from Interventional Cardiology Medical Group in West Hills, CA. A replay 
      of the webinar is available on the Milestone corporate website here. 

Etripamil for patients with AFib-RVR

   -- Milestone is on track to initiate a Phase 3 trial evaluating etripamil in 
      AFib-RVR in 2025. The protocol of a Phase 3, potentially registrational 
      study has been finalized and study start-up has commenced. The study will 
      use the patient self-administration 70-mg repeat-dose regimen, of 
      etripamil nasal spray, outside of the medically supervised setting, 
      similar to that used in the PSVT development program. 

Fourth Quarter and Full Year 2024 Financial Results

   -- As of December 31, 2024, Milestone had cash, cash equivalents, and 
      short-term investments of $69.7 million, compared to $66.0 
      million on December 31, 2023. 
 
   -- There was no revenue recorded for the fourth quarter or full year 
      ended December 31, 2024, compared to $1.0 million in the year 
      ended December 31, 2023. Revenue in 2023 was related to a milestone 
      payment received from Corxel Pharmaceuticals, formerly known as Ji Xing 
      Pharmaceuticals, under the Company's License and Collaboration Agreement. 
 
   -- Research and development expense for the fourth quarter of 2024 was $3.9 
      million, compared with $5.5 million for the prior year period. For the 
      full year ended December 31, 2024, research and development expense 
      was $14.4 million, compared with $31.1 million for the prior year. The 
      decrease year over year was primarily due to lower clinical expenses as a 
      result of the completion of phase 3 studies. 
 
   -- General and administrative expense for the fourth quarter of 2024 
      was $4.0 million, compared with $3.4 million for the prior year period. 
      For the full year ended December 31, 2024, general and administrative 
      expense was $16.7 million, compared with $15.9 million for the prior 
      year. The increase was driven primarily by an increase in outside service 
      costs, partially offset by a decrease in personnel costs. 
 
   -- Commercial expense for the fourth quarter of 2024 was $4.4 million, 
      compared with $5.0 million for the prior year period. For the full year 
      ended December 31, 2024, commercial expense was $11.0 million, compared 
      with $15.1 million for the prior year. While successfully resolving the 
      Refusal to File letter issued by the FDA in December 2023, we implemented 
      a reduction in personnel costs, professional costs and other operational 
      expenses related to commercialization. 
 
   -- For the fourth quarter of 2024, net loss was $12.4 million or $0.19 per 
      share, compared to a net loss of $13.6 million or $0.32 per share for the 
      prior year period. For the full year ended December 31, 2024, Milestone's 
      net loss was $41.5 million or $0.67 per share, compared to a net loss of 
      $59.7 million or $1.39 per share for the prior year. 

For further details on the Company's financials, refer to the Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 13, 2025.

About Etripamil

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(TM), 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.

About 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.

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", "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 timing and outcomes of future interactions with U.S. and foreign regulatory bodies, including the FDA; the PDUFA review goal date and the potential approval by the FDA of CARDAMYST for PSVT; commercialization plans for CARDAMYST, if approved; Milestone's plans to commence and timing of a Phase 3 study in AFib-RVR; the potential extension of protection under Milestone's intellectual property portfolio; the potential of etripamil to provide health care providers with a new treatment option to enable on-demand care and patient self-management and provide patients with active management and a greater sense of control over their condition; and other statements not related to historical fact. 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; whether the clinical trials will validate the safety and efficacy of etripamil for PSVT or other indications; 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, including in its annual report on Form 10-K for the year ended December 31, 2024, 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.

Contact:

Kim Fox, Vice President, Communications, kfox@milestonepharma.com

Investor Relations

Kevin Gardner, kgardner@lifesciadvisors.com

 
 
                     Milestone Pharmaceuticals Inc. 
                      Consolidated Balance Sheets 
            (in thousands of US dollars, except share data) 
 
                               December 31, 2024     December 31, 2023 
                              -------------------  --------------------- 
Assets 
 
Current assets 
 
 
 
 
 
 
 
   Cash and cash equivalents   $          25,314    $          13,760 
 
 
 
 
 
 
 
   Short-term investments                 44,381               52,243 
 
 
 
 
 
 
 
   Research and development 
    tax credits receivable                   901                  643 
 
 
 
 
 
 
 
   Prepaid expenses                        1,840                3,178 
 
 
 
 
 
 
 
   Other receivables                       1,490                3,208 
                                  --------------       -------------- 
Total current assets                      73,926               73,032 
Operating lease right-of-use 
 assets                                    1,376                1,917 
Property and equipment                       197                  277 
                                  --------------       -------------- 
Total assets                   $          75,499    $          75,226 
                                  ==============       ============== 
 
Liabilities, and 
Shareholders' Equity 
 
Current liabilities 
 
 
 
 
 
 
 
   Accounts payable and 
    accrued liabilities        $           7,555    $           6,680 
 
 
 
 
 
 
 
   Operating lease 
    liabilities                              571                  546 
                                  --------------       -------------- 
Total current liabilities                  8,126                7,226 
Operating lease liabilities, 
 net of current portion                      874                1,457 
Senior secured convertible 
 notes                                    53,352               49,772 
                                  --------------       -------------- 
Total liabilities                         62,352               58,455 
                                  --------------       -------------- 
 
 
Shareholders' Equity 
Common shares, no par value, 
 unlimited shares authorized 
 53,353,984 shares issued 
 and outstanding as of 
 December 31, 2024, 
 33,483,111 shares issued 
 and outstanding as of 
 December 31, 2023                       288,048              260,504 
Pre-funded warrants - 
 12,910,590 issued and 
 outstanding as of December 
 31, 2024 and 9,577,257 as 
 of December 31, 2023                     53,076               48,459 
Additional paid-in capital                39,568               33,834 
Accumulated deficit                     (367,545)            (326,026) 
                                  --------------       -------------- 
 
Total shareholders' equity                13,147               16,771 
                                  --------------       -------------- 
 
Total liabilities and 
 shareholders' equity          $          75,499    $          75,226 
                                  ==============       ============== 
 
 
                   Milestone Pharmaceuticals Inc. 
                  Consolidated Statements of Loss 
         (in thousands of US dollars, except share and per 
                             share data) 
 
                                                Years Ended 
                                                December 31, 
                                        ---------------------------- 
                                            2024           2023 
                                        ------------  -------------- 
 
Revenue                                 $        --   $     1,000 
 
Operating expenses 
Research and development, net of tax 
 credits                                $    14,357   $    31,052 
General and administrative                   16,742        15,932 
Commercial                                   11,003        15,114 
                                         ----------    ---------- 
 
Loss from operations                        (42,102)      (61,098) 
 
Interest income                               4,164         3,967 
Interest expense                             (3,581)       (2,554) 
                                         ----------    ---------- 
 
Net loss and comprehensive loss         $   (41,519)  $   (59,685) 
                                         ==========    ========== 
 
Weighted average number of shares and 
 pre-funded warrants outstanding, 
 basic and diluted                       62,210,702    42,955,779 
                                         ==========    ========== 
 
Net loss per share, basic and diluted   $     (0.67)  $     (1.39) 
                                         ==========    ========== 
 

(END) Dow Jones Newswires

March 13, 2025 07:08 ET (11:08 GMT)