On August 12, 2020 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, reported financial results for the second quarter ended June 30, 2020 and provided a clinical and corporate update (Press release, Milestone Pharmaceuticals, AUG 12, 2020, View Source [SID1234563544]).

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"With the benefit of having completed our first Phase 3 clinical trial and a clear and efficient registration path defined for etripamil in paroxysmal supraventricular tachycardia (PSVT), we remain fully committed to bringing this therapy to PSVT patients highly in need of better treatment options," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "We are on track to reopen the RAPID study later this year and believe the updated study design will allow us to best maximize the potential clinical utility of etripamil, including in patients with more persistent supraventricular tachycardia (SVT) events. With our recent $25 million private placement, we believe we are well positioned to execute on the remainder of the PSVT program and look forward to providing updates on our progress in the coming months."

Recent Updates

Announced Regulatory Guidance and Updated Clinical Development Plan for Etripamil in PSVT. In July 2020, Milestone announced a clinical and regulatory update for its pivotal program with etripamil in PSVT following interactions with the U.S. Food and Drug Administration (FDA).

The FDA indicated that two studies, the second part of NODE-301, which the Company has renamed the RAPID study, and the completed NODE-301 study could potentially fulfill the efficacy requirement for Milestone’s New Drug Application for etripamil in patients with PSVT. In addition, the FDA agreed to the following program changes:

An updated statistical analysis plan (SAP) for both the RAPID and NODE-301 studies under which the primary efficacy endpoint will be defined as time to conversion over the first 30 minutes, with a target p-value of less than 0.05 for each study. When employing this updated SAP, results from the NODE-301 study show that 54% of etripamil patients vs. 35% of placebo patients converted within 30 minutes (HR 1.87, p=0.02).
In an effort to maximize the potential treatment effect of etripamil, the RAPID study will allow for an optional repeat administration of study drug (either 70 mg of etripamil or placebo) in patients who have not experienced symptom relief within 10 minutes of the first study drug administration.
Milestone expects to reopen enrollment in the RAPID study later this year, with data anticipated in late 2021 or early 2022.

Raised $25 Million Through Private Placement. In July 2020, Milestone entered into a securities purchase agreement with affiliates of an existing shareholder, RTW Investments, LP, whereby Milestone sold pre-funded warrants to acquire an aggregate 6,655,131 common shares at a purchase price of $3.7465 per pre-funded warrant. Each pre-funded warrant is exercisable for one common share at an exercise price of $0.01 per share, has no expiration date, and is immediately exercisable, subject to certain beneficial ownership limitations. Aggregate proceeds received by the Company were $25 million.
Second Quarter 2020 Financial Results

As of June 30, 2020, Milestone had cash, cash equivalents, and short-term investments of $85.4 million and 24.7 million shares outstanding.
Research and development expenses for the second quarter of 2020 were $8.6 million compared with $10.5 million for the prior year period. For the six months ended June 30, 2020, research and development expenses were $20.5 million compared with $18.3 million for the prior year period. The increase in the first half of 2020 reflects the increased clinical development costs and manufacturing and formulation activities supporting Milestone’s Phase 3 clinical trial and the efforts in developing a clinical pathway for etripamil.
General and administrative expenses for the second quarter of 2020 were $3.0 million compared with $1.6 million for the prior year period. For the six months ended June 30, 2020, general and administrative expenses were $5.7 million compared with $2.6 million for the prior year period. The increase reflects additional increasing insurance costs, professional fees and administrative headcount in the first half of 2020 compared to the same period in 2019 primarily to support the compliance requirements of being a public company.
Commercial expenses for the second quarter of 2020 were $1.5 million compared with $2.2 million for the prior year period. For the six months ended June 30, 2020, commercial expense was $3.7 million compared with $4.4 million for the prior year period. The decrease in the three and six months ended June 30, 2020 reflects the efforts in reducing operating expenses affecting primarily pre-commercialization activities as Milestone focuses its efforts on an optimized clinical development pathway for etripamil.
For the second quarter of 2020, operating loss was $13.1 million compared to $14.3 million in 2019. For the six months ended June 30, 2020, Milestone’s operating loss was $29.9 million compared to $25.3 million in 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 patients with atrial fibrillation with a rapid ventricular rate, with subsequent studies expected in other conditions where calcium channel blockers are used.