Milestone Pharmaceuticals Reports First Quarter 2020 Financial Results and Provides Clinical and Corporate Update

MONTREAL and CHARLOTTE, N.C., May 14, 2020 /PRNewswire/ -- Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, today reported financial results for the first quarter ended March 31, 2020 and provided a clinical and corporate update.

"While the NODE-301 trial of etripamil for patients with paroxysmal supraventricular tachycardia (PSVT) did not meet its primary endpoint over the five hour observation period, we are encouraged by the topline data from the trial. With a favorable safety and tolerability profile as well as efficacy signals observed across earlier time points, topline results from NODE-301 reinforce our belief that etripamil has the potential to serve as the first self-administered therapy for the rapid termination of supraventricular tachycardia (SVT) episodes in the at-home setting," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "We look forward to working with regulators to determine next steps, with the goal of ensuring that etripamil is able to realize its full potential in patients with PSVT. In parallel, we continue to execute on the balance of the NODE program, including NODE-301B as well as open-label safety studies NODE-302 and NODE-303."

Recent Updates

    --  Reported Topline Results from Phase 3 NODE-301 Trial, Anticipates
        Regulatory Update in Early 3Q 2020. In March 2020, Milestone reported
        topline results from its Phase 3, multicenter, randomized, double-blind,
        placebo-controlled NODE-301 trial of its investigational new drug,
        etripamil nasal spray, the Company's novel short-acting calcium channel
        blocker, in patients with PSVT. Despite early activity, including the
        conversion of 61% of etripamil patients vs. 45% of placebo patients by
        45 minutes (p=0.02), a time period consistent with etripamil's known
        pharmacological activity, the study did not achieve its primary endpoint
        of time to conversion of SVT to sinus rhythm compared to placebo over
        the five hour period following study drug administration (p=0.12). The
        small number of placebo patients and prolonged efficacy measurement
        period was found to have confounded the results. The study did
        demonstrate statistically significant improvements in favor of etripamil
        over placebo in the important secondary endpoint of patient reported
        treatment satisfaction. Milestone believes the safety and tolerability
        data from the NODE-301 study will be supportive of at-home use of
        etripamil, with adverse events consistent with those observed in prior
        trials. The Company is determining next steps with regulators and
        expects to provide an update early in the third quarter of 2020.  The
        Company's full PSVT clinical program, including NODE-301B, NODE-302 and
        NODE-303, remains ongoing. NODE-301B, which was designed to collect
        double-blind data from randomized patients who had not yet experienced
        an event after the NODE-301 trial reached its target number of
        adjudicated SVT events, is expected to be analyzed separately as a
        second safety and efficacy data set.
    --  Reduction in Operating Expenses. Milestone expects to reduce planned
        operating expenses by 20-25% in order to focus its efforts on an
        optimized clinical development pathway for etripamil that will be
        determined following regulatory feedback. The cuts will primarily affect
        pre-commercialization activities. The goal of the operating cuts is to
        facilitate an additional efficacy study for etripamil in PSVT and to
        extend the Company's cash runway. The Company will update its cash
        runway guidance after meeting with regulators.
    --  Jeff Nelson Promoted to Chief Operating Officer. In March 2020,
        Milestone announced the promotion of Jeff Nelson to Chief Operating
        Officer. Mr. Nelson, who joined the Company in 2018 as Vice President of
        Program Management, brings to this new role over 15 years of experience
        in the pharmaceutical and biotech field, working primarily in project
        management, clinical operations, regulatory affairs, drug supply and
        distribution and public finance.

First Quarter 2020 Financial Results

    --  As of March 31, 2020, Milestone had cash and cash equivalents of $102
        million and 24.6 million shares outstanding.
    --  Research and development expense for the first quarter of 2020 was $11.9
        million compared with $7.8 million for the prior year period. The
        increase in expense was primarily driven by increased clinical
        development costs and manufacturing and formulation activities
        supporting its Phase 3 clinical trials.
    --  General and administrative expenses for the first quarter of 2020 were
        $2.7 million compared with $1.0 million for the prior year period. The
        increase was driven by additional headcount, professional fees and
        increased insurance costs.
    --  Commercial expense for the first quarter of 2020 was $2.2 million and
        remained consistant with the prior year period.
    --  For the first quarter of 2020, operating loss was $16.8 million compared
        to $10.9 million for the prior year period.

About Paroxysmal Supraventricular Tachycardia

Paroxysmal supraventricular tachycardia (PSVT) is a rapid heart rate condition characterized by intermittent episodes of supraventricular tachycardia (SVT) that start and stop suddenly and without warning. Episodes of SVT are often associated with symptoms including palpitations, sweating, chest pressure or pain, shortness of breath, sudden onset of fatigue, lightheadedness or dizziness, fainting, and anxiety. Certain calcium channel blockers have long been approved for the treatment of PSVT as well as other cardiac conditions; however, when calcium channel blockers are used for the termination of SVT episodes, they must be administered intravenously under medical supervision, usually in an emergency department or other acute care setting.

About Etripamil

Etripamil, the Company's lead investigational product, is designed to be a rapid response therapy for episodic cardiovascular conditions. The novel calcium channel blocker is self-administered via a nasal spray which may shift the current treatment paradigm for many patients with PSVT from the emergency department to the at-home setting. Milestone is conducting a comprehensive development program for etripamil, with Phase 3 trials underway in PSVT, and plans to commence a Phase 2 proof-of-concept trial in atrial fibrillation patients with rapid ventricular rate, with subsequent studies expected in other conditions where calcium channel blockers are used.

About Milestone Pharmaceuticals

Milestone Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines. Milestone Pharmaceuticals operates in Canada and the United States. For more information, visit and follow the Company on Twitter at @MilestonePharma.

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 "may," "will," "expect," "plan," "anticipate," "estimate," "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 (i) the design, progress, timing, scope and results of clinical trials, (ii) potential interactions with regulators, (iii) future operating expense reductions and (iv) the possibility that data will support future development. 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 and evaluation of clinical trials, and whether the clinical trials will validate the safety and efficacy of etripamil for PSVT or other indications, among others, as well as risks related to pandemics and public health emergencies, including those related to COVID-19, and risks related the sufficiency of our capital resources and our ability to raise additional capital. These and other risks are set forth in Milestone's filings with the U.S. Securities and Exchange Commission, including in its quarterly report on Form 10-Q for the quarter ended March 31, 2020, under the caption "Risk Factors." 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.




     [In US dollars]                                                                                                         Three months ended March 31,

                                                                                                                                 2020                 2019


              Operating expenses

     Research and development, net of tax credits                                                                             11,872                7,765

     General and administrative                                                                                                2,703                  979

     Commercial                                                                                                                2,183                2,186

              Loss from operations                                                                                        (16,758)            (10,930)

     Interest income, net of bank charges                                                                                        415                  500

              Loss and comprehensive loss before income taxes                                                             (16,343)            (10,430)

              Income tax expense                                                                                                                    22

              Net loss and comprehensive loss for the period                                                              (16,343)            (10,452)

              Weighted average number of shares outstanding,

                    basic and diluted                                                                                   24,548,777              603,040

              Net loss per share, basic and diluted                                                                        $(0.67)            $(17.32)



              [In US dollars]                                                                                                  '000

                                                                                                                             March 31, 
     December 31,

                                                                                                                                  2020                  2019




                Current Assets

              Cash and Cash Equivalents                                                                                       101,816               119,818

              Prepaid expenses and other current assets                                                                         2,508                 2,681

                Total current assets                                                                               104,324               122,499

              Operating lease right of use asset                                                                                  451                   524

              Property and equipment                                                                                              381                   405

                Total assets                                                                                       105,156               123,428


                Current liabilities

              Accounts payable and accrued liabilities                                                                          5,170                 7,997

              Operating lease liabilities                                                                                         196                   330

                Total current liabilities                                                                            5,366                 8,327

              Operating lease liabilities                                                                                         158                   184

                Total liabilities                                                                                    5,524                 8,511

                Shareholders' Equity

              Share capital

              Common shares, no par value, unlimited shares authorized,

              24,559,470 shares issued at March 31, 2020 and                                                                  226,378               226,245
    24,505,748 shares issued at December 31, 2019

              Additional paid in Capital                                                                                        4,730                 3,805

              Cumulative translation adjustment                                                                               (1,634)              (1,634)

              Accumulated Deficit                                                                                           (129,842)            (113,499)

                Total shareholders' equity                                                                          99,632               114,917

                Total liabilities and shareholders' deficit                                                        105,156               123,428


David Pitts
Argot Partners

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SOURCE Milestone Pharmaceuticals, Inc.