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™ (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
"We are focused on the potential FDA approval for CARDAMYST and the opportunity to help the millions of patients suffering from PSVT,” said
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 obtainU.S. Food and Drug Administration (FDA) marketing approval of CARDAMYST for the treatment of PSVT. The FDA accepted the NDA for CARDAMYST inMay 2024 and has set a Prescription Drug User Fee Act (PDUFA) review goal date forMarch 27, 2025 . - Milestone hosted a Commercial Launch Plan investor event The event, which took place in
New York City onFebruary 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 onMarch 30, 2025 byJames 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
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™, 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
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| Consolidated Balance Sheets | ||||||||
| (in thousands of US dollars, except share data) | ||||||||
| 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 |
288,048 | 260,504 | ||||||
| Pre-funded warrants - 12,910,590 issued and outstanding as of |
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 | ||||
| Consolidated Statements of Loss | ||||||||
| (in thousands of US dollars, except share and per share data) | ||||||||
| Years Ended | ||||||||
| 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 | ) | ||
Source: Milestone Pharmaceuticals Inc.