Milestone Pharmaceuticals (MIST) TD Cowen 45th Annual Healthcare Conference summary

Pipeline and Regulatory Update

• Etripamil (CARDAMYST) nasal spray is under FDA review for PSVT with a PDUFA date of March 27, 2025; approval could enable a commercial launch and a transformative year ahead, with promotional launch expected mid-2025.

• Approval in PSVT will open a supplemental NDA pathway for AFib-RVR, leveraging existing safety data; a Phase III study for AFib-RVR is planned to start in Q2, targeting a larger patient population.

• Patent estate for CARDAMYST potentially extended to 2042, supporting long-term exclusivity.

• Royalty financing agreement in place, providing $75M upon approval, with expected cash runway into mid-2026.

• Forward-looking statements highlight commercial, clinical, and financial plans subject to regulatory and market risks.

Market Opportunity and Patient Impact

• PSVT affects over 2 million people in the U.S., with 650,000–1 million actively engaged in the healthcare system and annual costs exceeding $5 billion.

• CARDAMYST is a nasal spray designed for rapid, self-administered relief, empowering patients and reducing anxiety about unpredictable episodes.

• RAPID trial data show more than 2x conversion to sinus rhythm and about 40% reduction in emergency department visits versus placebo.

• Safety profile is favorable, with most adverse events mild and related to nasal administration.

• Cardiologists expect to prescribe CARDAMYST to the majority of unablated PSVT patients, targeting about 50% of PSVT patients with burdensome episodes.

Commercialization and Launch Strategy

• Sales leadership and management team hired; sales reps to be onboarded post-approval, aiming for 50–60 reps covering 10,000–12,000 prescribers.

• Three main launch goals: build awareness and demand, secure payer access, and develop strong patient support and engagement.

• Payer strategy focuses on rational pricing below specialty drug levels (~$900/episode), with normal retail distribution and support for copay assistance.

• Phased commercial launch planned, expanding sales force and payer engagement over three years, with no anticipated branded competition.

• Pro-forma cash and investments of $76.4M as of September 30, 2024, with launch funding secured for 4+ quarters and $75M in non-dilutive royalty funding upon FDA approval.