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]

[Executive Summary and Recent Highlights](index=1&type=section&id=Milestone%20Pharmaceuticals%20Reports%20Second%20Quarter%202025%20Financial%20Results%20and%20Provides%20Regulatory%20and%20Corporate%20Update) Milestone Pharmaceuticals reported Q2 2025 financial results, including FDA acceptance of its CARDAMYST™ CRL response and a new PDUFA date - Milestone Pharmaceuticals reported Q2 2025 financial results and provided updates, including the FDA's acceptance of its CRL response for CARDAMYST™ (etripamil) Nasal Spray, setting a new PDUFA target date of **December 13, 2025**[1](index=1&type=chunk) - The company strengthened its balance sheet through a public equity offering in July, raising up to **$170 million gross proceeds** if all warrants are exercised, and extended a **$75 million royalty purchase agreement payment** from RTW through the end of 2025[1](index=1&type=chunk)[2](index=2&type=chunk) - CEO Joe Oliveto emphasized the team's focus on the potential approval and rapid commercialization of CARDAMYST for PSVT patients[2](index=2&type=chunk) [Program and Regulatory Updates](index=1&type=section&id=Second%20Quarter%20and%20Recent%20Program%20Updates) [Etripamil for PSVT](index=1&type=section&id=Etripamil%20for%20patients%20with%20PSVT) The FDA accepted Milestone's response to the Complete Response Letter (CRL) for CARDAMYST™ (etripamil) Nasal Spray, assigning a new PDUFA target date of December 13, 2025, leading the company to restart targeted pre-launch activities - FDA accepted the Company's response to the **Complete Response Letter (CRL)** for CARDAMYST™ (etripamil) Nasal Spray[3](index=3&type=chunk) - A new Prescription Drug User Fee Act (PDUFA) target date of **December 13, 2025**, has been set[3](index=3&type=chunk) - Milestone has restarted targeted pre-launch activities for CARDAMYST, maintaining its launch infrastructure[3](index=3&type=chunk) [Etripamil for AFib-RVR](index=1&type=section&id=Etripamil%20for%20patients%20with%20atrial%20fibrillation%20with%20rapid%20ventricular%20rate%20(AFib-RVR)) Milestone has finalized the Phase 3 study protocol for etripamil in AFib-RVR with FDA concurrence but has paused enrollment to prioritize resources for the anticipated launch of etripamil in PSVT - Phase 3 study protocol of etripamil in AFib-RVR finalized following FDA review and concurrence[3](index=3&type=chunk) - Initiation of enrollment in the AFib-RVR study has been paused to prioritize resources for the expected launch of etripamil in PSVT[3](index=3&type=chunk) [Corporate and Financial Updates](index=2&type=section&id=Second%20Quarter%20and%20Recent%20Corporate%20Updates) [Corporate Developments](index=2&type=section&id=Corporate%20Developments) Milestone completed a public equity offering in July 2025, raising significant gross and net proceeds, and extended its royalty purchase agreement with RTW to December 31, 2025, to support the commercial launch of CARDAMYST Public Equity Offering Proceeds (July 2025) | Metric | Amount (Millions USD) | | :-------------------------------- | :-------------------- | | Total Gross Proceeds (if all warrants exercised) | Up to 170 | | Immediate Net Proceeds | Approximately 48.7 | - The proceeds from the public offering are intended to fund the continued development and commercial launch of CARDAMYST in PSVT, as well as for working capital and general corporate purposes[6](index=6&type=chunk) - Milestone extended the marketing approval deadline in its **$75 million Royalty Purchase Agreement** with RTW from September 30, 2025, to **December 31, 2025**[6](index=6&type=chunk) [Financial Performance Overview](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) Milestone reported no revenue for Q2 2025, an increase in R&D and commercial expenses due to launch preparations, a decrease in G&A expenses, and a higher net loss compared to the prior year [Cash Position and Liquidity](index=2&type=section&id=Cash%20Position%20and%20Liquidity) As of June 30, 2025, Milestone's cash, cash equivalents, and short-term investments totaled $43.4 million, a decrease from December 31, 2024, but significantly bolstered by $48.7 million in net proceeds from a subsequent public offering Cash, Cash Equivalents, and Short-term Investments | Period | Amount (Millions USD) | | :----------------------- | :-------------------- | | June 30, 2025 | $43.4 | | December 31, 2024 | $69.7 | | *Change* | *($26.3)* | - Subsequent to the quarter end, the company raised approximately **$48.7 million in net proceeds** from a public offering[6](index=6&type=chunk) [Operating Expenses](index=2&type=section&id=Operating%20Expenses) Research and development expenses increased due to higher consulting costs, while general and administrative expenses decreased primarily due to lower legal and personnel fees. Commercial expenses significantly rose in preparation for CARDAMYST's launch Research and Development Expense | Period | 2025 (Millions USD) | 2024 (Millions USD) | Change (YoY) | | :----------------------- | :------------------ | :------------------ | :----------- | | Q2 | $3.7 | $2.8 | +$0.9 | | Six Months Ended June 30 | $8.6 | $6.5 | +$2.1 | - The increase in R&D expense was primarily due to higher consulting and outside service costs, partially offset by lower personnel-related costs[6](index=6&type=chunk) General and Administrative Expense | Period | 2025 (Millions USD) | 2024 (Millions USD) | Change (YoY) | | :----------------------- | :------------------ | :------------------ | :----------- | | Q2 | $3.8 | $5.0 | -$1.2 | | Six Months Ended June 30 | $8.9 | $9.0 | -$0.1 | - The decrease in G&A expense was primarily due to a decrease in legal fees, professional fees, and personnel costs[6](index=6&type=chunk) Commercial Expense | Period | 2025 (Millions USD) | 2024 (Millions USD) | Change (YoY) | | :----------------------- | :------------------ | :------------------ | :----------- | | Q2 | $5.1 | $1.8 | +$3.3 | | Six Months Ended June 30 | $15.5 | $4.7 | +$10.8 | - Increases in commercial expense were a result of additional personnel costs, professional costs, and other operational expenses related to preparation for the launch of CARDAMYST[10](index=10&type=chunk) [Net Loss](index=3&type=section&id=Net%20Loss) Milestone's net loss increased for both the second quarter and the six months ended June 30, 2025, compared to the prior year periods Net Loss and Comprehensive Loss | Period | 2025 (Millions USD) | 2024 (Millions USD) | Change (YoY) | | :----------------------- | :------------------ | :------------------ | :----------- | | Q2 | ($13.0) | ($9.4) | ($3.6) | | Six Months Ended June 30 | ($33.7) | ($19.7) | ($14.0) | Net Loss Per Share (Basic and Diluted) | Period | 2025 (USD) | 2024 (USD) | Change (YoY) | | :----------------------- | :--------- | :--------- | :----------- | | Q2 | ($0.20) | ($0.14) | ($0.06) | | Six Months Ended June 30 | ($0.51) | ($0.34) | ($0.17) | [About Etripamil](index=3&type=section&id=About%20Etripamil) Etripamil is Milestone's lead investigational product, a novel calcium channel blocker nasal spray for PSVT and AFib-RVR, designed for self-administration - Etripamil is Milestone's lead investigational product, a novel calcium channel blocker nasal spray for PSVT and AFib-RVR[8](index=8&type=chunk) - It is designed as a self-administered rapid response therapy for patients, bypassing the need for immediate medical oversight[8](index=8&type=chunk) - CARDAMYST™ is the conditionally approved brand name for etripamil nasal spray, supported by a robust clinical trial program including a completed Phase 3 for PSVT and Phase 2 for AFib-RVR[8](index=8&type=chunk) [About Milestone Pharmaceuticals](index=3&type=section&id=About%20Milestone%20Pharmaceuticals) Milestone Pharmaceuticals is a biopharmaceutical company focused on developing innovative cardiovascular solutions, empowering patients with self-management - Milestone Pharmaceuticals Inc. is a biopharmaceutical company focused on developing and commercializing innovative cardiovascular solutions[9](index=9&type=chunk) - The company aims to provide new treatment approaches that empower patients with an active role in self-managing their care[9](index=9&type=chunk) - Etripamil, a novel calcium channel blocker nasal spray, is its lead investigational product for self-administration to treat symptomatic episodic attacks of PSVT and AFib-RVR[9](index=9&type=chunk) [Condensed Consolidated Financial Statements](index=5&type=section&id=Condensed%20Consolidated%20Financial%20Statements) [Condensed Consolidated Balance Sheets](index=5&type=section&id=Milestone%20Pharmaceuticals%20Inc.%20Condensed%20Consolidated%20Balance%20Sheets%20(Unaudited)) The unaudited condensed consolidated balance sheets present the company's financial position as of June 30, 2025, compared to December 31, 2024, showing changes in assets, liabilities, and shareholders' equity Condensed Consolidated Balance Sheets (Unaudited) | | June 30, 2025 (in thousands of US dollars) | December 31, 2024 (in thousands of US dollars) | | :------------------------------------------ | :------------------------------------------ | :------------------------------------------ | | **Assets** | | | | Cash and cash equivalents | $42,499 | $25,314 | | Short-term investments | $918 | $44,381 | | Total current assets | $46,168 | $73,926 | | Total assets | $47,417 | $75,499 | | **Liabilities** | | | | Total current liabilities | $9,283 | $8,126 | | Senior secured convertible notes | $55,238 | $53,352 | | Total liabilities | $65,162 | $62,352 | | **Shareholders' (Deficit) Equity** | | | | Accumulated deficit | ($401,272) | ($367,545) | | Total shareholders' (deficit) equity | ($17,745) | $13,147 | [Condensed Consolidated Statements of Loss](index=6&type=section&id=Milestone%20Pharmaceuticals%20Inc.%20Condensed%20Consolidated%20Statements%20of%20Loss%20(Unaudited)) The unaudited condensed consolidated statements of loss detail the company's financial performance for the three and six months ended June 30, 2025, and 2024, highlighting operating expenses, net loss, and net loss per share Condensed Consolidated Statements of Loss (Unaudited) | | Three months ended June 30, 2025 (in thousands of US dollars) | Three months ended June 30, 2024 (in thousands of US dollars) | Six months ended June 30, 2025 (in thousands of US dollars) | Six months ended June 30, 2024 (in thousands of US dollars) | | :------------------------------------------ | :------------------------------------------ | :------------------------------------------ | :------------------------------------------ | :------------------------------------------ | | Revenue | $— | $— | $— | $— | | Research and development, net of tax credits | $3,669 | $2,815 | $8,647 | $6,454 | | General and administrative | $3,759 | $5,046 | $8,926 | $8,999 | | Commercial | $5,103 | $1,801 | $15,481 | $4,685 | | Loss from operations | ($12,531) | ($9,662) | ($33,054) | ($20,138) | | Net loss and comprehensive loss | ($12,966) | ($9,363) | ($33,727) | ($19,717) | | Net loss per share, basic and diluted | ($0.20) | ($0.14) | ($0.51) | ($0.34) | [Forward-Looking Statements](index=4&type=section&id=Forward-Looking%20Statements) This section outlines forward-looking statements based on expectations and assumptions, subject to risks that may cause actual results to differ materially - This press release contains forward-looking statements regarding future events, conditions, or circumstances, identified by words such as 'potential,' 'expect,' and 'will'[11](index=11&type=chunk) - These statements are based on Milestone's expectations and assumptions and involve risks and uncertainties, meaning actual results may differ materially[11](index=11&type=chunk) - Important factors that could cause differences include FDA approval outcomes, timing of clinical trials, market conditions, and the sufficiency of capital resources, as detailed in SEC filings[11](index=11&type=chunk) [Contact Information](index=4&type=section&id=Contact%3A) Provides contact details for investor relations inquiries - For investor relations inquiries, contact **Kevin Gardner** at kgardner@lifesciadvisors.com[12](index=12&type=chunk)

Core Insights - The FDA has accepted the company's response to the Complete Response Letter (CRL) for CARDAMYST™ (etripamil) Nasal Spray, with a new Prescription Drug User Fee Act (PDUFA) target date set for December 13, 2025 [3][4] - The company completed a public equity offering in July 2025, raising total gross proceeds of up to $170 million, which will strengthen its balance sheet for the commercialization of CARDAMYST if approved [5][6] - A $75 million Royalty Purchase Agreement with RTW Investments has been extended through the end of 2025, which is expected to fund the continued development and commercial launch of CARDAMYST [6] Regulatory Updates - The FDA's acceptance of the response to the CRL is a significant milestone for the company, as it aims for the approval of CARDAMYST for the treatment of paroxysmal supraventricular tachycardia (PSVT) [2][3] - The company has maintained its launch infrastructure and restarted targeted pre-launch activities in anticipation of the new potential approval date [3] Financial Updates - The public equity offering raised approximately $48.7 million in net proceeds after deducting underwriting commissions and estimated offering expenses, which will be used for the development and commercial launch of CARDAMYST [5][11] - As of June 30, 2025, the company reported a net loss of $13.0 million, compared to a net loss of $9.4 million for the same period in the previous year [11][17] - Research and development expenses for the second quarter of 2025 were $3.7 million, an increase from $2.8 million in the prior year period, primarily due to higher consulting and outside service costs [11][17] Product Development - The Phase 3 study protocol for etripamil in patients with atrial fibrillation with rapid ventricular rate (AFib-RVR) has been finalized, although enrollment has been paused to prioritize resources for the expected launch of etripamil in PSVT [4] - Etripamil is designed as a self-administered rapid response therapy for patients, potentially providing a new treatment option for on-demand care and patient self-management [9][10]

[SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS](index=4&type=section&id=SPECIAL%20NOTE%20REGARDING%20FORWARD-LOOKING%20STATEMENTS) The report contains forward-looking statements regarding the company's strategy, financial condition, operations, and market growth, subject to substantial risks and uncertainties - Forward-looking statements are based on current expectations and projections about future events and financial trends, but are subject to known and unknown risks, uncertainties, and assumptions[9](index=9&type=chunk)[10](index=10&type=chunk) - Key risks include clinical trial outcomes for etripamil (PSVT, AFib-RVR), commercialization ability, financing needs, regulatory approvals, market acceptance, intellectual property, government regulations, and competitive environment[10](index=10&type=chunk)[13](index=13&type=chunk) - The company operates in a competitive and rapidly changing environment, with new risk factors emerging, and cannot guarantee future results[11](index=11&type=chunk)[12](index=12&type=chunk) [PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This part presents unaudited interim condensed consolidated financial statements, management's discussion and analysis, market risk disclosures, and controls and procedures [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section provides unaudited condensed consolidated financial statements, including Balance Sheets, Statements of Loss, Shareholders' (Deficit) Equity, and Cash Flows, with detailed notes [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The balance sheets show a decrease in total assets and a shift to a shareholders' deficit as of June 30, 2025, driven by reduced short-term investments and accumulated deficit Condensed Consolidated Balance Sheets | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :-------------------------------- | :----------------------------- | :------------------------------- | | Cash and cash equivalents | $42,499 | $25,314 | | Short-term investments | $918 | $44,381 | | Total current assets | $46,168 | $73,926 | | Total assets | $47,417 | $75,499 | | Total current liabilities | $9,283 | $8,126 | | Senior secured convertible notes | $55,238 | $53,352 | | Total liabilities | $65,162 | $62,352 | | Total shareholders' (deficit) equity | $(17,745) | $13,147 | [Condensed Consolidated Statements of Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Loss) Milestone Pharmaceuticals Inc. reported increased net losses for the three and six months ended June 30, 2025, due to higher operating expenses and decreased interest income Condensed Consolidated Statements of Loss | Metric (in thousands) | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :-------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Revenue | $— | $— | $— | $— | | R&D, net of tax credits | $3,669 | $2,815 | $8,647 | $6,454 | | G&A | $3,759 | $5,046 | $8,926 | $8,999 | | Commercial | $5,103 | $1,801 | $15,481 | $4,685 | | Loss from operations | $(12,531) | $(9,662) | $(33,054) | $(20,138) | | Interest income | $516 | $1,186 | $1,213 | $2,180 | | Interest expense | $(951) | $(887) | $(1,886) | $(1,759) | | Net loss | $(12,966) | $(9,363) | $(33,727) | $(19,717) | | Net loss per share | $(0.20) | $(0.14) | $(0.51) | $(0.34) | [Condensed Consolidated Statements of Shareholders' (Deficit) Equity](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Shareholders'%20(Deficit)%20Equity) The statements show a significant increase in accumulated deficit, resulting in a total shareholders' deficit of **$17.7 million** as of June 30, 2025, primarily due to net losses Condensed Consolidated Statements of Shareholders' (Deficit) Equity | Metric (in thousands) | June 30, 2025 | December 31, 2024 | | :-------------------- | :------------ | :---------------- | | Common Shares | $288,263 | $288,048 | | Pre-funded warrants | $53,076 | $53,076 | | Additional paid-in capital | $42,188 | $39,568 | | Accumulated deficit | $(401,272) | $(367,545) | | Total shareholders' (deficit) equity | $(17,745) | $13,147 | - Net loss for the six months ended June 30, 2025, was **$(33.7 million)**, contributing to the accumulated deficit[20](index=20&type=chunk) - Share-based compensation expense for the six months ended June 30, 2025, was **$2.7 million**[20](index=20&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) For the six months ended June 30, 2025, cash and cash equivalents increased, primarily from investing activities (redemption of short-term investments), despite significant cash used in operations Condensed Consolidated Statements of Cash Flows | Metric (in thousands) | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :-------------------- | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(26,518) | $(14,987) | | Net cash provided by (used in) investing activities | $43,527 | $(17,532) | | Net cash provided by financing activities | $176 | $32,021 | | Net increase (decrease) in cash and cash equivalents | $17,185 | $(498) | | Cash and cash equivalents – End of period | $42,499 | $13,262 | - Operating activities used **$26.5 million** in cash for the six months ended June 30, 2025, primarily due to a net loss of **$33.7 million**[163](index=163&type=chunk) - Investing activities provided **$43.5 million** in cash, mainly from the redemption of **$44.5 million** in short-term investments[165](index=165&type=chunk) [Notes to Unaudited Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) These notes detail the company's organization, accounting policies, risks, liquidity, revenue, investments, debt, equity, share-based compensation, net loss per share, royalty agreements, and subsequent events - Milestone Pharmaceuticals Inc. is a biopharmaceutical company focused on developing and commercializing cardiovascular medicines, with etripamil as its lead product candidate for PSVT and AFib-RVR[24](index=24&type=chunk) - The company incurred operating losses and had an accumulated deficit of **$401.3 million** as of June 30, 2025, but expects to support operations for at least the next 12 months with existing cash and proceeds from a July 2025 offering[35](index=35&type=chunk)[37](index=37&type=chunk) - No revenue was recorded for the three and six months ended June 30, 2025 and 2024, as license revenue is recognized only when milestones are reached under the agreement with Corxel Pharmaceuticals[38](index=38&type=chunk) - The company issued **$50.0 million** principal amount of 6.0% Convertible Senior Notes due 2029, with a net carrying amount of **$55.2 million** as of June 30, 2025[41](index=41&type=chunk)[42](index=42&type=chunk) - Total share-based compensation expense for all plans was **$1.3 million** for the three months ended June 30, 2025, and **$2.7 million** for the six months ended June 30, 2025[67](index=67&type=chunk) - On July 11, 2025, the company completed an underwritten public offering, generating approximately **$48.7 million** in net proceeds, and amended its Royalty Purchase Agreement with RTW, extending the marketing approval deadline for etripamil to December 31, 2025, for a potential **$75.0 million** purchase price[79](index=79&type=chunk)[81](index=81&type=chunk)[82](index=82&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=29&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides management's perspective on the company's financial condition and operations, detailing etripamil's status, market overviews, regulatory updates, operating expenses, and liquidity - Milestone Pharmaceuticals is focused on developing and commercializing CARDAMYST (etripamil) nasal spray for PSVT and AFib-RVR[84](index=84&type=chunk) - The FDA issued a Complete Response Letter (CRL) for CARDAMYST's NDA for PSVT in March 2025, citing CMC issues; the company submitted its response on June 13, 2025, which was accepted, and a new PDUFA target date of December 13, 2025, has been assigned[85](index=85&type=chunk)[97](index=97&type=chunk)[115](index=115&type=chunk) - Etripamil demonstrated positive Phase 2 data for AFib-RVR, showing rapid and statistically superior ventricular rate reduction and improved symptom relief; a Phase 3 study protocol has been finalized, but enrollment is delayed due to the PSVT NDA CRL[86](index=86&type=chunk)[106](index=106&type=chunk)[107](index=107&type=chunk) - The PSVT market is estimated to affect over **two million** Americans, with a target addressable market for CARDAMYST of **500,000 to 800,000** patients, potentially treating **2.5 million to 4 million** episodes per year[89](index=89&type=chunk)[90](index=90&type=chunk) - The AFib-RVR market affects an estimated **10 million** Americans, with a target addressable market of up to **four to five million** patients by 2030 for etripamil[94](index=94&type=chunk) - The company incurred net losses of **$13.0 million** and **$33.7 million** for the three and six months ended June 30, 2025, respectively, and expects significant losses to continue as it pursues regulatory approvals and commercialization[112](index=112&type=chunk)[113](index=113&type=chunk) - Commercial expenses increased significantly by **183.3%** (three months) and **230.4%** (six months) in 2025 compared to 2024, due to preparations for CARDAMYST launch, though ramping up was temporarily paused after the CRL[143](index=143&type=chunk)[144](index=144&type=chunk)[145](index=145&type=chunk) - The company's cash, cash equivalents, and short-term investments totaled **$43.4 million** as of June 30, 2025, and it expects these resources, along with recent offering proceeds, to fund operations for at least the next 12 months[149](index=149&type=chunk)[153](index=153&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=56&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section discusses the company's exposure to market risks, primarily interest rate and foreign exchange rate risk, noting minimal impact due to short-term investments and low foreign currency transactions - The company's primary investment objective is to preserve principal and liquidity, with cash, cash equivalents, and short-term investments totaling **$43.4 million** as of June 30, 2025[176](index=176&type=chunk) - An immediate **10%** change in interest rates is not expected to materially affect the fair market value of the investment portfolio or operating results due to its short-term nature[176](index=176&type=chunk)[177](index=177&type=chunk) - The company is exposed to foreign exchange rate risk due to transactions in Canadian dollars, with a net monetary exposure of **$2.1 million** in Canadian dollars as of June 30, 2025[178](index=178&type=chunk) - The company does not use derivative instruments or a formal hedging program for foreign exchange risk due to the low volume of foreign currency transactions[178](index=178&type=chunk) [Item 4. Controls and Procedures](index=58&type=section&id=Item%204.%20Controls%20and%20Procedures) Management evaluated the effectiveness of disclosure controls and procedures as of June 30, 2025, concluding they were effective, with no material changes in internal control over financial reporting during the quarter - Disclosure controls and procedures were evaluated as effective at a reasonable assurance level as of June 30, 2025[181](index=181&type=chunk) - No material changes in internal control over financial reporting occurred during the quarter ended June 30, 2025[182](index=182&type=chunk) - Management acknowledges that control systems provide only reasonable, not absolute, assurance and are subject to inherent limitations such as human error, circumvention, and management override[183](index=183&type=chunk) [PART II. OTHER INFORMATION](index=60&type=section&id=PART%20II.OTHER%20INFORMATION) This part covers other information not included in the financial statements, such as legal proceedings, updated risk factors, unregistered sales of equity securities, and exhibits [Item 1. Legal Proceedings](index=60&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings and is unaware of any pending or threatened legal actions that could adversely affect its business or financial condition - No material legal proceedings are currently ongoing or threatened against the company[185](index=185&type=chunk) [Item 1A. Risk Factors](index=60&type=section&id=Item%201A.%20Risk%20Factors) This section updates risk factors, focusing on new risks from international trade policies and reiterating substantial dependence on etripamil's successful clinical development and regulatory approval, especially after the FDA Complete Response Letter - International trade policies, including tariffs and trade barriers, could adversely affect the business by increasing R&D expenses, disrupting supply chains (e.g., APIs from China), and hindering commercialization[187](index=187&type=chunk)[188](index=188&type=chunk)[190](index=190&type=chunk) - The company's future success is substantially dependent on obtaining regulatory approval for etripamil, its only product candidate; the FDA issued a CRL for etripamil's NDA for PSVT, citing CMC issues, and there's no assurance of approval even after the response submission[194](index=194&type=chunk)[198](index=198&type=chunk) - Healthcare legislative reforms, such as the 'One Big Beautiful Bill Act' (OBBBA) and the Inflation Reduction Act (IRA), could negatively impact the business by reducing healthcare costs, limiting market access, and increasing governmental scrutiny over drug pricing[201](index=201&type=chunk)[203](index=203&type=chunk)[204](index=204&type=chunk) - Future changes to tax laws, including the OBBBA's impact on R&E expenditures, could increase tax liability and adversely affect profitability[207](index=207&type=chunk)[208](index=208&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=70&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This item is marked as 'Not applicable,' indicating no unregistered sales of equity securities or use of proceeds to report for the period - No unregistered sales of equity securities or use of proceeds to report[210](index=210&type=chunk) [Item 3. Defaults Upon Senior Securities](index=70&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This item is marked as 'Not applicable,' indicating no defaults upon senior securities to report for the period - No defaults upon senior securities to report[210](index=210&type=chunk) [Item 4. Mine Safety Disclosures](index=70&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is marked as 'Not applicable,' indicating no mine safety disclosures to report for the period - No mine safety disclosures to report[210](index=210&type=chunk) [Item 5. Other Information](index=71&type=section&id=Item%205.%20Other%20Information) None of the company's directors or executive officers adopted, modified, or terminated a Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement during the fiscal quarter ended June 30, 2025 - No Rule 10b5-1 or non-Rule 10b5-1 trading arrangements were adopted, modified, or terminated by directors or executive officers during the quarter ended June 30, 2025[211](index=211&type=chunk) [Item 6. Exhibits](index=71&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed as part of the Form 10-Q, including organizational documents, equity incentive plans, certifications from executive officers, and XBRL-related documents - The report includes certifications from the Principal Executive Officer and Principal Financial Officer (Exhibits 31.1, 31.2, 32.1)[212](index=212&type=chunk) - XBRL (eXtensible Business Reporting Language) documents are provided for structured data reporting (Exhibits 101.INS, 101.SCH, 101.CAL, 101.DEF, 101.LAB, 101.PRE, 104)[212](index=212&type=chunk)