Milestone Pharmaceuticals (MIST) Company Presentation summary

Product overview and clinical development

• CARDAMYST (etripamil) is an investigational, short-acting calcium channel blocker nasal spray for on-demand treatment of PSVT and AFib-RVR, with patent protection until 2042.

• PDUFA target date for FDA approval is December 13, 2025, following a response to a Complete Response Letter focused on CMC issues, not clinical concerns.

• Robust clinical data in PSVT show rapid, reliable conversion to sinus rhythm and a favorable safety profile, with most adverse events being mild and related to nasal administration.

• Phase 2 data in AFib-RVR demonstrate substantial and rapid reduction in ventricular rate, high patient satisfaction, and lower use of rescue medication.

• Phase 3 AFib-RVR program is ready to start, pending regulatory pathway via sNDA after PSVT approval.

Market opportunity and patient need

• Over 2 million patients in the US are diagnosed with PSVT, costing the healthcare system at least $5 billion annually.

• Current PSVT treatments are inconvenient, intrusive, or ineffective, with no new drug therapies since 1989.

• CARDAMYST is positioned as the first and only FDA-approved on-demand therapy for PSVT, empowering patients to self-treat and potentially reducing ED visits and hospital admissions.

• Cardiologists and electrophysiologists expect to prescribe CARDAMYST to the majority of unablated PSVT patients, with projected use of 3-5 times per year per patient at steady state.

• No anticipated branded competition and a low barrier to prescribing support a compelling commercial opportunity.

Financials and commercialization strategy

• $82.6M in cash and short-term investments as of September 30, 2025, with up to $265M in total potential funding for commercialization, including $75M royalty payment upon FDA approval.

• Strategic financing includes $50M in convertible notes and a non-dilutive synthetic royalty structure, with royalties ranging from 7% to 1% based on annual net sales.

• Commercial launch plans include rapid sales rep hiring, payer engagement, reimbursement support, and patient affordability programs.

• Focus on demand generation, quality coverage, and patient support services to drive adoption.

• Exclusivity for CARDAMYST extends to 2042 via a recently issued US patent covering repeat dose regimen.