Milestone Pharmaceuticals Inc. is a biopharmaceutical company dedicated to the development and commercialization of innovative cardiovascular medicines. Founded in 2003 and headquartered in Montreal, Québec, Milestone has established itself as a prominent player in the industry, leveraging its expertise to address unmet medical needs and improve the lives of patients living with complex heart conditions.
Financial Overview
Milestone's financial performance has showcased the company's commitment to strategic growth and prudent resource allocation. As of the latest reporting period, the company's cash, cash equivalents, and short-term investments totaled $76.40 million, providing a strong foundation to support ongoing research and development efforts. The company's net loss for the nine months ended September 30, 2024, stood at $29.16 million, a significant improvement from the $46.07 million net loss reported in the corresponding period of the previous year. Despite these recent improvements, Milestone has faced financial challenges since its inception, recording net losses of $59.7 million in 2023 and $58.4 million in 2022.
To strengthen its financial position, Milestone has taken significant steps. In 2019, the company's common shares began trading on The Nasdaq Global Select Market under the symbol "MIST", providing access to public markets to help fund ongoing research and development activities. Additionally, in 2023, Milestone closed a $50 million convertible debt financing with RTW Investments to further support its operations and development activities.
Regulatory Milestones and Product Pipeline
The core of Milestone's business revolves around the development of its lead product candidate, etripamil, a novel and potent calcium channel blocker designed as a rapid-onset nasal spray for the treatment of paroxysmal supraventricular tachycardia (PSVT) and atrial fibrillation with rapid ventricular rate (AFib-RVR). In the early years following its founding, Milestone devoted substantial resources to the preclinical and clinical development of etripamil, including conducting phase 1 and phase 2 clinical trials to evaluate its safety and efficacy for both PSVT and AFib-RVR.
In May 2024, Milestone announced that the U.S. Food and Drug Administration (FDA) had accepted the company's New Drug Application (NDA) for etripamil in the treatment of PSVT. This milestone comes after Milestone resubmitted the NDA in March 2024, following the FDA's initial Refusal to File letter in December 2023, which requested clarification about the data recorded for the timing of adverse events in the Phase 3 clinical trials. The FDA has set a Prescription Drug User Fee Act (PDUFA) date of March 27, 2025, for the potential approval of etripamil for PSVT.
Alongside the PSVT program, Milestone is also advancing the development of etripamil for the treatment of AFib-RVR. In 2024, the company held a Type C meeting with the FDA, which provided guidance on the sNDA pathway for the potential marketing approval of etripamil for the AFib-RVR indication, provided that etripamil is already approved for PSVT. Milestone is currently finalizing the Phase 3 study protocol for the AFib-RVR program, with plans to initiate enrollment in the first half of 2025.
Global Expansion and Partnerships
Milestone's commitment to expanding its reach extends beyond North America. In September 2024, the company's licensing partner, Corxel (formerly known as Ji Xing Pharmaceuticals), announced positive topline data from a Phase 3 trial of etripamil in PSVT in China. The trial met its primary endpoint, demonstrating a statistically significant and clinically meaningful difference in time to PSVT conversion compared to placebo, with a safety profile consistent with previous etripamil studies.
This global collaboration underscores Milestone's strategic approach to leveraging partnerships to advance the development and commercialization of etripamil in various regions. The company continues to explore potential opportunities to expand its reach and maximize the impact of its innovative solutions. In 2021, Milestone entered into a license and collaboration agreement with Ji Xing Pharmaceuticals (now Corxel) to develop and commercialize etripamil in China, Hong Kong, Macau, and Taiwan, further demonstrating its commitment to global expansion.
Operational Efficiency and Cost Management
Milestone's management team has demonstrated a keen focus on operational efficiency and cost management, which has been particularly evident during the company's recent cash conservation measures. In response to the revised timeline for the NDA submission, Milestone implemented measures to reduce spending through program deferrals and team restructuring. These efforts have helped the company maintain a strong financial position, with $76.40 million in cash, cash equivalents, and short-term investments as of September 30, 2024.
Furthermore, Milestone's ability to navigate the regulatory landscape and secure key milestones, such as the FDA's acceptance of the etripamil NDA, showcases the company's exceptional execution capabilities and commitment to advancing its pipeline.
Competitive Landscape and Market Opportunity
The PSVT market represents a significant opportunity for Milestone, as the company estimates that the condition affects approximately 2 million Americans and results in over 150,000 emergency department visits and hospital admissions, as well as up to 80,000 ablations per year. From this diagnosed population, Milestone has identified a target addressable market of 40-60% of patients who experience frequent and longer, moderate to severe episodes each year.
In the AFib-RVR space, Milestone is addressing a sizable market, with an estimated 5 million Americans suffering from atrial fibrillation. The company's novel approach to acute rate control presents a compelling opportunity to improve patient outcomes and reduce the burden on the healthcare system.
Risks and Challenges
While Milestone's progress has been notable, the company faces several risks and challenges inherent to the biopharmaceutical industry. The successful development and commercialization of etripamil remain subject to regulatory approval, clinical trial outcomes, and the ability to navigate the competitive landscape. Additionally, Milestone's reliance on partnerships and collaborations to expand its global footprint introduces potential risks related to the execution and success of these strategic relationships.
Financials
Milestone's financial performance reflects its ongoing investments in research and development, as well as its efforts to manage costs effectively. The company's total revenue for the nine months ended September 30, 2024, was $1.5 million, primarily derived from the license and collaboration agreement with Corxel. Research and development expenses for the same period were $17.9 million, while general and administrative expenses totaled $10.7 million.
For the most recent fiscal year (2023), Milestone reported revenue of $1.00 million. The company's net loss for 2023 was $59.69 million. Operating cash flow (OCF) for 2023 was negative $46.42 million, while free cash flow (FCF) was negative $46.54 million.
In the most recent quarter (Q3 2024), Milestone reported no revenue. The net loss for the quarter was $9.44 million. OCF and FCF for Q3 2024 were both negative $7.12 million. The company did not generate revenue in the most recent quarter, resulting in no year-over-year growth.
Research and development expenses decreased by 41% and 59% for the three and nine month periods ended September 30, 2024, respectively, compared to the same periods in the previous year. This decrease was primarily due to lower clinical trial costs as the company completed its Phase 3 PSVT studies.
Liquidity
As of September 30, 2024, Milestone maintained a strong liquidity position with $76.40 million in cash, cash equivalents, and short-term investments. This financial foundation provides the company with the resources to continue advancing its clinical programs and support potential commercialization efforts. The company's cash runway is expected to extend into mid-2026, based on current operating plans and expectations.
Additional liquidity metrics as of Q3 2024 include:
- Debt/Equity ratio: 2.25 - Cash: $12.80 million - Available credit line: Unlimited - Current ratio: 15.40 - Quick ratio: 15.40
These metrics indicate a strong liquidity position, with a high current ratio and quick ratio suggesting the company has ample resources to meet its short-term obligations.
Recent Developments
In April 2024, Milestone announced new clinical data demonstrating the real-world application of etripamil for conversion of recurrent PSVT. This data further supports the potential efficacy and safety of etripamil in treating PSVT episodes.
The company's Phase 3 RAPID clinical trial, which evaluated etripamil in patients with PSVT, achieved its primary endpoint in October 2022. The trial demonstrated a highly statistically significant and clinically meaningful difference in time to PSVT conversion compared to placebo.
Conclusion
Milestone Pharmaceuticals' unwavering commitment to innovation and patient-centric solutions has positioned the company as a rising force in the cardiovascular treatment landscape. With the potential approval of etripamil for PSVT on the horizon and a robust pipeline targeting additional indications, Milestone is poised to make a significant impact on the lives of patients living with complex heart conditions. As the company navigates the regulatory and competitive landscape, its focus on operational efficiency, strategic partnerships, and disciplined capital allocation will be crucial in driving long-term success and shareholder value.
The company's strong liquidity position, coupled with its promising clinical data and strategic partnerships, provides a solid foundation for future growth. However, as with any biopharmaceutical company, Milestone faces risks related to regulatory approvals, clinical trial outcomes, and market competition. The company's ability to successfully navigate these challenges and capitalize on its innovative pipeline will be key factors in determining its long-term success in the cardiovascular medicine space.