MONTREAL and CHARLOTTE, N.C., Oct. 09, 2025 (GLOBE NEWSWIRE) -- Milestone® Pharmaceuticals Inc. (Nasdaq: MIST) a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, today announced that it will present a poster presentation at the American Heart Association Scientific Sessions 2025, to be held November 7-10th in New Orleans, Louisiana. Data to be presented describe the efficacy, safety, and tolerability of self-administered etripamil by patients ...

Core Points - The FDA has accepted Milestone Pharmaceuticals' response to the Complete Response Letter (CRL) for CARDAMYST™ (etripamil) nasal spray, with a new PDUFA target action date set for December 13, 2025 [1][5] - Milestone has extended its $75 million Royalty Purchase Agreement with RTW Investments until December 31, 2025, to support the planned launch of CARDAMYST following FDA approval [2][4] FDA Review and Response - The FDA's acceptance of the response to the CRL is a significant milestone for CARDAMYST, and the company is optimistic about a potential approval decision later this year [3] - Milestone's response to the CRL included results from additional in-vitro studies and a plan to transfer manufacturing duties to vendors with recent FDA inspection history [5][6] Royalty Purchase Agreement - The Royalty Purchase Agreement allows RTW to purchase tiered royalty payments on etripamil's annual net product sales in exchange for a $75 million purchase price, contingent on FDA approval by December 31, 2025 [4] - The amendment to the agreement reflects RTW's ongoing commitment to Milestone and provides a future funding source for the company [2][3] Product Overview - Etripamil is a novel calcium channel blocker nasal spray designed for self-administration to treat paroxysmal supraventricular tachycardia (PSVT) and atrial fibrillation with rapid ventricular response (AFib-RVR) [7][9] - If approved, CARDAMYST™ aims to offer a new treatment option for patients, allowing for on-demand care and self-management [7][9] Clinical Development - Etripamil has completed a Phase 3 clinical program for PSVT and a Phase 2 trial for AFib-RVR, indicating a robust clinical development pathway [8]

Core Viewpoint - Milestone Pharmaceuticals is actively addressing the Chemistry, Manufacturing, and Controls (CMC) issues raised by the FDA regarding its lead product CARDAMYST for the treatment of paroxysmal supraventricular tachycardia (PSVT) and is preparing for a Type A meeting with the FDA to resolve these issues [1][2][6]. Financial Results - As of March 31, 2025, the company reported cash, cash equivalents, and short-term investments totaling $56.0 million, a decrease from $69.7 million as of December 31, 2024 [11]. - The company reported no revenue for the first quarter of 2025, consistent with the same period in 2024 [11]. - Research and development expenses increased to $5.0 million in Q1 2025 from $3.6 million in Q1 2024, primarily due to higher consulting costs [11]. - General and administrative expenses rose to $5.2 million in Q1 2025 from $4.0 million in the prior year, driven by increased outside service costs [11]. - Commercial expenses surged to $10.4 million in Q1 2025 from $2.9 million in Q1 2024, reflecting preparations for the launch of CARDAMYST [11]. - The net loss for the first quarter of 2025 was $20.8 million, compared to a net loss of $10.4 million in the same period last year [11]. Product Development and Regulatory Updates - The FDA issued a Complete Response Letter (CRL) for CARDAMYST in March 2025, citing two key CMC issues: the need for additional information on nitrosamine impurities and a new facility inspection to ensure compliance with Good Manufacturing Practices (GMP) [6]. - The Phase 3 study protocol for etripamil in patients with atrial fibrillation with rapid ventricular rate (AFib-RVR) has been finalized, but enrollment is paused to focus on resolving the CRL for PSVT [4]. - A new method of use patent for etripamil nasal spray was issued, potentially extending intellectual property protection until July 2042 [6]. - CARDAMYST was recognized in an independent survey as a new drug expected to significantly impact patient health, with 40% of respondents selecting it [6]. Company Overview - Milestone Pharmaceuticals is focused on developing innovative cardiovascular solutions, with etripamil as its lead investigational product designed for self-administration in treating symptomatic episodes of PSVT and AFib-RVR [9][8].