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 commercial launch and a supplemental NDA for AFib-RVR, with Phase III for AFib starting in Q2 this year and launch expected mid-2025.

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

• $75 million royalty financing agreement in place, with payment expected upon approval and cash runway projected into mid-2026.

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

Clinical Data and Patient Impact

• CARDAMYST is a nasal spray calcium channel blocker designed for rapid, self-administered relief of PSVT and AFib-RVR, showing over twice the conversion rate and three times faster action than placebo in pivotal trials.

• Safety profile is favorable, with mostly mild, local adverse events and no serious cardiac concerns.

• RAPID trial data show a 40% reduction in emergency department visits versus placebo, supporting patient quality of life and payer value.

• Cardiologists expect to prescribe CARDAMYST to the majority of unablated PSVT patients.

Commercial Strategy and Market Opportunity

• Targeting over 2 million PSVT patients in the US, with an initial focus on the 650,000–1 million already engaged in the healthcare system; aim for 50% market penetration.

• Pricing strategy guided by payers to remain below specialty drug thresholds (under $900 per episode), with broad retail distribution and no special handling required.

• No anticipated branded competition and a low barrier to prescribing support early demand generation.

• Launch preparations include hiring sales leadership, planning a 50–60 person sales force to cover 10,000–12,000 prescribers, and phased expansion over three years.

• Patient support, copay mitigation, and reimbursement programs are prioritized for launch.