Company Overview - Milestone Pharmaceuticals Inc. (MIST) has received approval for its first commercial product, CARDAMYST nasal spray, aimed at converting acute symptomatic episodes of paroxysmal supraventricular tachycardia (PSVT) to sinus rhythm in adults [1][3]. Product Details - CARDAMYST nasal spray is a novel and rapid-acting calcium channel blocker designed for self-administration during unpredictable episodes of PSVT, marking the first FDA-approved treatment for this condition in over 30 years [3]. - The product is expected to be available in retail pharmacies by the first quarter of 2026 [3]. Market Context - PSVT affects more than two million Americans, with episodes characterized by rapid heart rates ranging from 160 to over 250 beats per minute, often occurring suddenly [2]. - Symptoms associated with PSVT include palpitations, sweating, chest pressure or pain, shortness of breath, sudden fatigue, fainting, and anxiety [2]. Stock Performance - On January 29, 2025, MIST was trading around $1.98, and it reached a 52-week high of $3.06 during intraday trading on December 12, 2025, before closing at $2.41, reflecting an 18.31% decline [4].

Core Insights - Milestone Pharmaceuticals has received FDA approval for CARDAMYST™ (etripamil) nasal spray, marking the first new treatment for paroxysmal supraventricular tachycardia (PSVT) in over 30 years, providing a self-administered option for over two million Americans with this condition [1][4][12] Company Overview - Milestone Pharmaceuticals is a biopharmaceutical company focused on developing innovative cardiovascular medicines, with CARDAMYST being its lead product aimed at treating PSVT [27] Product Details - CARDAMYST is a rapid-acting calcium channel blocker designed for the conversion of acute symptomatic episodes of PSVT to sinus rhythm in adults, allowing patients to manage episodes independently [3][14] - The product is expected to be available in retail pharmacies in the first quarter of 2026 [1] Clinical Data - The FDA approval is supported by a clinical trial program involving over 1,800 participants and more than 2,000 PSVT episodes, demonstrating that CARDAMYST users were twice as likely to convert to sinus rhythm compared to placebo [6][7] - In the Phase 3 RAPID trial, 64% of participants using CARDAMYST converted to sinus rhythm within 30 minutes, compared to 31% for placebo, with a median conversion time of 17 minutes for CARDAMYST versus 54 minutes for placebo [6][7] Market Potential - The approval of CARDAMYST opens the door for further development of etripamil for atrial fibrillation with rapid ventricular rate (AFib-RVR) under a supplemental New Drug Application (sNDA) pathway, targeting an estimated market of three to four million patients by 2030 [4][10][13] Financial Position - As of September 30, 2025, Milestone had cash and equivalents of $82.6 million, positioning the company well for the launch and commercialization of CARDAMYST [22] - The company has entered into a Royalty Purchase Agreement to secure additional funding contingent upon the FDA approval of etripamil [22][23]

Core Insights - Milestone Pharmaceuticals (NASDAQ:MIST) is currently viewed as a promising investment opportunity, with analysts at Jefferies raising the price target from $1 to $2, maintaining a Hold rating [1][2]. Company Overview - Milestone Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing cardiovascular medicines for self-administration by patients with episodic heart conditions [3]. Product Development and Sales Prospects - The price target increase is attributed to the company's confidence in its upcoming PDUFA, with a product launch scheduled for early next year [2]. - The company anticipates that the first quarter of next year will mark the beginning of significant sales, supported by the deployment of 60 representatives targeting up to 15,000 high-volume prescribers [2]. - Milestone is developing Etripamil, a nasal-administered, short-acting calcium channel blocker aimed at treating paroxysmal supraventricular tachycardia (PSVT) [2].

Financial Performance - Milestone Pharmaceuticals reported cash, cash equivalents, and short-term investments of $82.6 million as of September 30, 2025, up from $69.7 million at the end of 2024[6]. - The company reported a net loss of $11.9 million for the third quarter of 2025, compared to a net loss of $9.4 million for the same period in 2024[7]. - Total liabilities increased to $67.2 million as of September 30, 2025, compared to $62.4 million at the end of 2024[13]. - The company's accumulated deficit reached $413.2 million as of September 30, 2025, up from $367.5 million at the end of 2024[13]. Research and Development - Research and development expenses for Q3 2025 were $3.9 million, slightly down from $4.0 million in Q3 2024, while total R&D expenses for the nine months ended September 30, 2025, increased to $12.6 million from $10.4 million in the prior year[6]. - The Phase 3 protocol for etripamil in atrial fibrillation with rapid ventricular rate (AFib-RVR) has been finalized, with plans to leverage data from the PSVT NDA for regulatory approval[4]. - Etripamil has shown aligned efficacy and safety across multiple studies, with higher conversion rates for symptomatic PSVT episodes compared to placebo[3]. Commercial Activities - Commercial expenses surged to $4.6 million in Q3 2025, compared to $1.9 million in Q3 2024, reflecting increased preparations for the launch of CARDAMYST[6]. - The company completed a public equity offering that provided net proceeds of approximately $48.7 million, which, along with a $75 million royalty payment upon FDA approval, is expected to fund the launch of CARDAMYST[5]. - The Prescription Drug User Fee Act (PDUFA) target date for CARDAMYST (etripamil) is set for December 13, 2025, with promotional launch plans in place pending FDA approval[1].

Core Insights - Milestone Pharmaceuticals is preparing for the potential launch of CARDAMYST (etripamil) nasal spray for treating symptomatic paroxysmal supraventricular tachycardia (PSVT) with a PDUFA target date set for December 13, 2025 [1][6] - The company has strengthened its financial position through a successful equity offering and an amended Royalty Purchase Agreement, providing resources for a successful launch [2][5] - Etripamil is also being developed for atrial fibrillation with rapid ventricular rate (AFib-RVR), with plans for a pivotal Phase 3 program following positive Phase 2 trial results [4][9] Financial Overview - As of September 30, 2025, Milestone reported cash and cash equivalents of $82.6 million, an increase from $69.7 million at the end of 2024 [12] - The company reported a net loss of $11.9 million for the third quarter of 2025, compared to a net loss of $9.4 million in the same period of 2024 [12][18] - Research and development expenses for the third quarter of 2025 were $3.9 million, slightly down from $4.0 million in the prior year [12][18] Clinical Development - Etripamil has shown promising efficacy and safety in clinical trials, with data from over 600 patients indicating higher conversion rates for symptomatic PSVT compared to placebo [6] - The company plans to leverage data from the PSVT NDA for the supplemental NDA regulatory approval pathway for AFib-RVR [4][6] - Recent presentations at the American Heart Association (AHA) Scientific Sessions highlighted the potential of etripamil as a rapid, self-administered treatment option [6][9]

Product Development and Regulatory Status - The company is focused on developing and commercializing CARDAMYST (etripamil) as a portable nasal spray treatment for supraventricular tachycardia (SVT) and atrial fibrillation with rapid ventricular rate (AFib-RVR) [82]. - In March 2025, the company received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for CARDAMYST, with a new PDUFA target date set for December 13, 2025 [83][97]. - The company plans to leverage data from the PSVT program to support a supplemental New Drug Application (sNDA) for etripamil in AFib-RVR [85]. - The FDA highlighted two key Chemistry, Manufacturing and Controls (CMC) issues in the CRL, including concerns about nitrosamine impurities and the need for facility inspection [96]. Clinical Trials and Efficacy - The company completed a successful Phase 2 study for etripamil in AFib-RVR, showing rapid and statistically superior ventricular rate reduction compared to placebo, with a median time-to-conversion of 17 minutes in the NODE-303 study [84][98]. - The Phase 3 RAPID trial of etripamil demonstrated a significant difference in time to SVT conversion, with 64.3% of patients converting within 30 minutes compared to 31.2% for placebo (HR 2.62; p<0.001) [99]. - In the NODE-301 studies, patients self-administering etripamil sought additional medical interventions 43% less frequently (15% vs. 25%; p=0.013) and had 39% fewer emergency department visits (14% vs. 22%; p=0.035) compared to placebo [100]. - The Phase 3 trial (JX02002) in China showed that 40.5% of patients self-administering etripamil converted from PSVT to sinus rhythm within 30 minutes, compared to 15.9% for placebo (HR 3.00; p<0.001) [103]. - The ReVeRA study reported a significant reduction in ventricular rate of 29.91 bpm (p<0.0001) for patients treated with etripamil compared to placebo [105]. - The Phase 3 AFib-RVR study will enroll patients with a history of symptomatic AFib episodes, using a self-administered regimen of 70 mg per dose [109]. Market Potential and Financial Overview - The estimated addressable market for CARDAMYST in PSVT is approximately 500,000 to 800,000 patients, with a peak demand potential of 2.5 million to 4 million episodes treated per year [89]. - The prevalence of AFib is expected to grow from 10 million to over 12 million Americans by 2030, with a target addressable market of up to 4 to 5 million patients for etripamil in AFib-RVR [93]. - Total healthcare expenditures for PSVT patients range from $20,000 to $30,000 per patient annually, with at least $5 billion spent annually in the U.S. on PSVT management [90]. - The American Heart Association reported that atrial fibrillation resulted in $25 billion in direct medical costs in 2016, with projected costs reaching $46 billion in direct costs by 2030 [95]. Financial Performance and Expenses - As of September 30, 2025, the company reported an accumulated deficit of $413.2 million and net losses of $11.9 million for Q3 2025 [111]. - The company expects to continue incurring significant losses as it focuses on obtaining regulatory approvals and preparing for commercialization of etripamil [112]. - For the three months ended September 30, 2025, commercial expenses increased by $2.7 million, or 142.1%, compared to the same period in 2024, primarily due to additional personnel and operational expenses related to the launch of CARDAMYST [135]. - For the nine months ended September 30, 2025, commercial expenses rose by $13.5 million, or 204.8%, compared to the same period in 2024, driven by increased personnel and operational costs for CARDAMYST [136]. - Research and development expenses, net of tax credits, increased by $2.2 million, or 20.8%, for the nine months ended September 30, 2025, compared to the same period in 2024, mainly due to higher consulting and outside service costs [132]. - Total operating expenses for the three months ended September 30, 2025, were $11.8 million, a 23.0% increase from $9.6 million in the same period in 2024 [128]. - The net loss for the three months ended September 30, 2025, was $11.9 million, representing a 26.3% increase from a net loss of $9.4 million in the same period in 2024 [128]. - The company reported a net loss of $45.6 million for the nine months ended September 30, 2025, compared to a net loss of $29.2 million for the same period in 2024 [157][158]. Cash Flow and Funding - As of September 30, 2025, the company had cash, cash equivalents, and short-term investments totaling $82.6 million, with an accumulated deficit of $413.2 million [142]. - The company expects to fund operations through equity and/or debt financing until substantial revenue from product sales is generated [155]. - The company incurred non-cash charges of $3.9 million related to share-based compensation during the nine months ended September 30, 2025 [157]. - Cash provided by financing activities increased to $49.1 million in the nine months ended September 30, 2025, up 53.0% from $32.1 million in 2024 [161][162]. - The net proceeds from the 2025 Offering were $48.6 million after deducting underwriting commissions and estimated offering expenses [144]. - The company anticipates an increase in commercial expenses if the FDA approves the NDA, as it will invest in infrastructure and personnel for the product launch in the United States [138]. - Net cash used in operating activities for the nine months ended September 30, 2025, was $36.3 million, a 64.8% increase from $22.0 million in the same period of 2024 [156]. Operational Model and Risks - The company operates with a significant outsourcing model, relying on a smaller core team to manage a larger number of outsourced vendors and consultants [110]. - The company is subject to foreign exchange rate risk due to transactions in Canadian dollars, with a net monetary exposure of $2.7 million as of September 30, 2025 [173]. - The company anticipates that any inability to secure adequate funding may lead to reductions in spending or suspension of planned programs, adversely affecting its business [155]. - The company has not entered into off-balance sheet arrangements as of the reporting date [163].

Core Insights - Milestone Pharmaceuticals announced consistent efficacy and safety findings for etripamil nasal spray in managing paroxysmal supraventricular tachycardia (PSVT) across multiple clinical trials [1][3][7] - The data will be presented at the American Heart Association Scientific Sessions 2025, highlighting the potential for self-administration of etripamil to improve patient management of PSVT [1][3] Study Findings - Etripamil demonstrated a median time to conversion of symptomatic PSVT episodes of 18.5 minutes, with a conversion rate of 59.6% by 30 minutes and 63.2% to 75.1% by 60 minutes post-administration [7] - Safety data indicated a low rate of test-dose failures at 1.4% and predominantly mild, transient adverse events localized to the nasal site [7] Product Information - Etripamil is a novel calcium channel blocker nasal spray designed for self-administration, aiming to provide rapid response therapy for PSVT and atrial fibrillation with rapid ventricular response (AFib-RVR) [6][9] - The New Drug Application (NDA) for etripamil, branded as CARDAMYST, is currently under evaluation by the U.S. FDA, with a target decision date set for December 13, 2025 [5][9] Company Overview - Milestone Pharmaceuticals focuses on developing innovative cardiovascular medicines, with etripamil being its lead investigational product aimed at improving the management of heart conditions like PSVT [1][9]

Core Insights - Milestone Pharmaceuticals Inc. is set to present data on the efficacy, safety, and tolerability of self-administered etripamil for paroxysmal supraventricular tachycardia at the American Heart Association Scientific Sessions 2025 [1][2] - The presentation will be led by Dr. James Ip and is scheduled for November 10, 2025 [2] - The company has submitted a New Drug Application (NDA) to the FDA for etripamil targeting abnormal heart rhythms [3] Company Overview - Milestone Pharmaceuticals Inc. focuses on developing and commercializing innovative cardiovascular medicines [3] - The company aims to benefit individuals with specific heart conditions through its product offerings [3]

Milestone Pharmaceuticals FY Conference Summary Company Overview - **Company**: Milestone Pharmaceuticals - **Lead Asset**: Etripamil, trade name Cardamist, a nasal spray calcium channel blocker for arrhythmias, particularly Supraventricular Tachycardia (SVT) and Atrial Fibrillation [3][4] Key Points FDA Interaction and Approval Process - Milestone received a Complete Response Letter (CRL) from the FDA focused on manufacturing issues, particularly related to nitrosamines [6][7] - The company met with the FDA in June to understand new guidance and submitted a complete response package shortly after [8] Market Opportunity - The SVT market is estimated to have around 2 million patients, with about half being potential candidates for treatment [11] - The potential market value for Etripamil is projected to exceed half a billion dollars [11] Launch Strategy - The company plans to deploy 50-60 sales representatives initially, focusing on commercial coverage to facilitate quicker access to the drug [15] - There is a strong motivation among patients for new treatment options, as there has been no innovation in this space for nearly 30 years [14][16] Patient Access and Affordability - The strategy includes making the drug easily accessible through retail pharmacies and ensuring affordability through commercial insurance [18][19] - The company aims for a net sales price of $500 to $1,000 per prescription, with a target of $750 in the launch year [31] Clinical Data and Pricing Strategy - The drug's value proposition is based on its clinical benefits, particularly in reducing emergency department visits, which can cost between $3,000 to $4,000 on average [26][27] - The pricing strategy is designed to ensure broad access, with co-pays expected to be $30 to $50 for commercial patients and $50 to $80 for Medicare patients [30] Safety and Efficacy - Etripamil is positioned as a safe option with a favorable safety profile, primarily involving mild nasal irritation [36] - The company believes that positive patient experiences will lead to increased usage and refills over time [37] Demand Generation and Market Penetration - The launch strategy emphasizes generating demand through awareness among healthcare providers and ensuring that prescriptions are filled at pharmacies [38] - Investments will be made in tools for medical and formulary exceptions to facilitate access for patients [39] Additional Insights - The company recognizes the importance of patient education and trust in the product, given the non-life-threatening nature of the condition [18][33] - The focus on a retail distribution model distinguishes it from specialty drugs, allowing for a more straightforward access strategy [39] This summary encapsulates the critical aspects of Milestone Pharmaceuticals' conference, highlighting the company's strategic approach to launching Etripamil and addressing market needs.

Company Updates - Milestone Pharmaceuticals Inc. will have its President and CEO, Joe Oliveto, participate in one-on-one meetings at the Wells Fargo Healthcare Conference from September 3 to 5, 2025, in Boston [1] - The company will provide a corporate update at the H.C. Wainwright 27th Annual Global Investment Conference from September 8 to 10, 2025, in New York [1] Webcast Information - A webcast of the H.C. Wainwright presentation will be available on September 9 at 12:00 PM EDT, with a replay accessible for approximately 90 days following the presentation on Milestone's website [2] Company Overview - Milestone Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative cardiovascular medicines for individuals with specific heart conditions [3] - The company has recently submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for etripamil, aimed at treating paroxysmal supraventricular tachycardia (PSVT) [3]