Milestone Pharmaceuticals Reports First Quarter 2021 Financial Results and Provides Clinical, Regulatory, and Corporate Update
MONTREAL and CHARLOTTE, N.C.,
"We continue to work closely with our existing clinical trial sites, as well as add new sites, to address the impact that the COVID-19 pandemic has had on patient enrollment in the pivotal Phase 3 RAPID trial of etripamil in patients with PSVT. While we are encouraged by recent improvements in enrollment rates, we now expect to report topline data from the RAPID trial in the second half of 2022," said
Recent Updates
- Announced Exclusive License Agreement with
Ji Xing Pharmaceuticals to Develop and Commercialize Etripamil for PSVT inGreater China . Milestone today announced that it has entered an exclusive license and collaboration agreement withJi Xing Pharmaceuticals (Ji Xing), a biotechnology company headquartered inShanghai and backed byRTW Investments, LP (RTW) focused on advancing innovative medicines inChina , to develop and, if approved, commercialize the investigational drug etripamil in patients with paroxysmal supraventricular tachycardia (PSVT) and additional cardiovascular conditions inGreater China . Milestone will receive an upfront cash payment consisting of$15 million and a$5 million equity investment by RTW Investments. In addition, Milestone is eligible to receive up to$107.5 million in milestone payments and royalties on future sales of etripamil inGreater China . Milestone will supply etripamil and delivery devices to Ji Xing. Ji Xing will be responsible for development and commercialization costs inGreater China . - Company Expects to Report Topline Data from Pivotal Phase 3 RAPID Trial in 2H22. Enrollment remains ongoing in the pivotal Phase 3 RAPID trial of etripamil nasal spray in patients with PSVT. The ongoing COVID-19 pandemic has restricted access to many medical centers, reduced visits to physician offices, and caused patients to delay medical care. The Company continues to work diligently with investigators to identify potential site-specific solutions to mitigate these COVID-related delays, and has also increased the number of participating centers. Milestone now expects to report topline data in the second half of 2022. The Company believes its current cash resources, including the upfront payment from Ji Xing and proceeds from the equity investment from RTW, will be sufficient to support operations beyond the data readout into mid-2023.
The RAPID trial, which is targeting a total of 180 adjudicated paroxysmal supraventricular tachycardia (PSVT) events, is expected to randomize approximately 500 patients 1:1 to receive either etripamil nasal spray or placebo. As previously announced, to maximize the potential treatment effect of etripamil, patients will be directed to administer a second dose of study drug if they do not experience symptom relief within 10 minutes of the first study drug administration. The primary efficacy analysis for both the RAPID trial and the completed NODE-301 trial will be time to conversion of SVT over the first 30 minutes following initial study drug administration, with a target p-value of less than 0.05 for each trial. The RAPID and NODE-301 trials could potentially serve to fulfill the efficacy requirement for a future New Drug Application (NDA) for etripamil in patients with PSVT. - NODE-303 Open-Label Safety Study Amended to Include Repeat Dose of Etripamil. Following an interim safety data analysis of the RAPID study, the Company requested and the
U.S. Food and Drug Administration (FDA) agreed to amend the ongoing NODE-303 study to allow a repeat dose of etripamil if symptoms persist for 10 minutes after the first dose. NODE-303 is a global open-label study which primarily evaluates the safety of etripamil when self-administered without medical supervision during single or multiple SVT episodes. Important secondary measures include efficacy, patient quality of life, and pharmacoeconomic assessments. - Data from NODE-301 Trial Featured During Oral Presentation at the
American College of Cardiology 70th AnnualScientific Session and Expo (ACC.21). An oral presentation titled, "Etripamil Nasal Spray Relieves Symptoms And Reduces Emergency Room Interventions In Patients With Paroxysmal Supraventricular Tachycardia (PSVT): Analysis Of Clinical Outcomes In The NODE-301 Trial" (#403-13), was featured at the recent ACC.21 virtual meeting. The presentation was delivered byBruce Stambler , MD, FHRS,Piedmont Heart Institute ,Atlanta, GA , and investigator in the NODE-301 trial. - Enrollment Continues in ReVeRA Phase 2 Proof-of-Concept Trial Evaluating Etripamil in Patients with Atrial Fibrillation and Rapid Ventricular Rate (AFib-RVR). In
March 2021 , Milestone announced that the first patient was enrolled in ReVeRA, its Phase 2 proof-of-concept study of etripamil nasal spray in patients experiencing AFib-RVR. Patient enrollment is ongoing, with the trial expected to enroll approximately 50 patients randomized 1:1 to receive either 70 mg of etripamil nasal spray or placebo. The Phase 2 double blind, placebo controlled, proof-of-concept study is designed to assess the safety and efficacy of etripamil nasal spray to reduce ventricular rate in patients with AFib-RVR experiencing an episode of elevated heart rate requiring treatment. The trial will be conducted inCanada in collaboration with theMontreal Heart Institute and other research centers. The primary endpoint will assess reduction in ventricular rate, with key secondary endpoints including the time to achieve the maximum reduction in rate and duration of the effect.
First Quarter 2021 Financial Results
- As of March 31, 2021, Milestone had cash, cash equivalents, and short-term investments of $129.9 million and 29.8 million common shares and 11.4 million common shares issuable upon exercise of pre-funded warrants outstanding.
- Research and development expense for the first quarter of 2021 was $8.6 million compared with $11.9 million for the prior year period. The COVID-19 pandemic contributed to delays in new clinical site initiation and patient enrollment, which translated into lower than expected research and development spending in the quarter ended March 31, 2021.
- General and administrative expense for the first quarter of 2021 was
$2.6 million compared with$2.7 million for the prior year period. - Commercial expense for the first quarter of 2021 was $1.4 million compared with
$2.2 million for the prior year period. The decrease of commercial expense in the quarter ended March 31, 2021 reflects efforts in reducing operating expenses affecting primarily pre-commercialization activities as Milestone focused its efforts on an optimized clinical development pathway for etripamil. - For the first quarter of 2021, operating loss was $12.6 million compared to $16.8 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 which affects approximately two million Americans. 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, calcium channel blockers approved for the termination of SVT episodes must be administered intravenously under medical supervision, usually in an emergency department or other acute care setting.
About Atrial Fibrillation and Rapid Ventricular Rate
Atrial fibrillation (AFib) is a common arrhythmia marked by an irregular and often rapid heartbeat. AFib is estimated to affect five million patients in
About Etripamil
Etripamil, Milestone's lead investigational product, is a novel calcium channel blocker designed to be a rapid-response therapy for episodic cardiovascular conditions. As a nasal spray that is self-administered by the patient, etripamil has the potential to shift the current treatment experience for many patients from the emergency department to the at-home setting. Milestone is conducting a comprehensive development program for etripamil, with Phase 3 trials ongoing in paroxysmal supraventricular tachycardia (PSVT) and now a Phase 2 proof-of-concept trial underway in patients with atrial fibrillation and rapid ventricular rate (AFib-RVR).
About
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," "potential," "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 potential of etripamil as a promising therapy for PSVT patients, the design, progress, timing, scope and results of the RAPID and ReVeRA trials, Milestone's ability to execute on the remainder of the PSVT program, Milestone's plans to study etripamil in atrial fibrillation patients, the sufficiency of Milestone's current cash resources to support its operations, Milestone's and Ji Xing's intention and ability to develop and commercialize etripamil in
INTERIM CONDENSED CONSOLIDATED STATEMENTS OF LOSS AND COMPRENHENSIVE LOSS |
||
(Unaudited, in thousands of US dollars, except share and per share data) |
||
Three months ended |
||
2021 |
2020 |
|
$ |
$ |
|
Operating expenses |
||
Research and development, net of tax credits |
8,595 |
11,872 |
General and administrative |
2,633 |
2,703 |
Commercial |
1,366 |
2,183 |
Loss from operations |
(12,594) |
(16,758) |
Interest income, net of bank charges |
80 |
415 |
Net loss and comprehensive loss for the period |
(12,514) |
(16,343) |
Weighted average number of shares and |
||
pre-funded warrants outstanding, basic and diluted |
41,256,248 |
24,548,777 |
Net loss per share, basic and diluted |
(0.30) |
(0.67) |
INTERIM CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited, in thousands of US dollars, except share data) |
||
|
|
|
2021 |
2020 |
|
$ |
$ |
|
ASSETS |
||
Current Assets |
||
Cash, cash equivalents and short-term investments |
129,859 |
142,310 |
Prepaid expenses and other current assets |
7,385 |
6,376 |
Total current assets |
137,244 |
148,686 |
Operating lease right-of-use asset |
914 |
980 |
Property and equipment |
285 |
308 |
Total assets |
138,443 |
149,974 |
LIABILITIES |
||
Current liabilities |
||
Accounts payable and accrued liabilities |
5,564 |
5,914 |
Current portion of operating lease liabilities |
252 |
245 |
Total current liabilities |
5,816 |
6,159 |
Operating lease liabilities |
635 |
696 |
Total liabilities |
6,451 |
6,855 |
Shareholders' Equity |
||
Share capital |
||
Common shares, no par value, unlimited shares authorized 29,846,000 |
251,716 |
251,682 |
Pre-funded warrants - 11,417,034 issued and outstanding as of |
48,007 |
48,007 |
Additional paid in capital |
9,883 |
8,530 |
Cumulative translation adjustment |
(1,634) |
(1,634) |
Accumulated deficit |
(175,980) |
(163,466) |
Total shareholders' equity |
131,992 |
143,119 |
Total liabilities and shareholders' equity |
138,443 |
149,974 |
Contact:
212-600-1902
david@argotpartners.com
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