Milestone Pharmaceuticals (MIST) investor relations material

Product overview and market need

• CARDAMYST is the first FDA-approved treatment for PSVT in over 30 years, offering a novel, fast-acting, portable nasal spray option with robust clinical data and patent protection until 2042.

• PSVT affects over 2 million people in the US, with significant healthcare costs exceeding $5 billion annually and current treatments considered inconvenient or ineffective.

• CARDAMYST aims to empower patients with self-administration, reduce emergency department visits, and provide a cost-effective solution for payers.

Commercial strategy and market opportunity

• Commercial launch began in February, focusing on new patient starts, prescription growth, and broad commercial coverage.

• No anticipated branded competition, low barrier to prescribing, and a sizable addressable market of 650,000–1 million patients treated annually.

• Targeting half of annually treated patients by reaching ~10,000 prescribers with a 60-rep sales force.

• Market access strategy includes payer engagement, retail distribution, copay savings, and reimbursement support to ensure affordability and ease of prescribing.

Clinical data and pipeline expansion

• CARDAMYST demonstrated a ~40% reduction in emergency department use for PSVT, with fast onset and well-tolerated safety profile.

• Phase 3 pivotal trial initiated for AFib-RVR, targeting a large addressable market with efficient regulatory pathway.

• Phase 2 ReVeRA trial in AFib-RVR showed substantial, rapid, and sustained reduction in ventricular rate, improved symptom relief, and lower use of rescue medication.

• Phase 3 trial (ReVeRA-301) will evaluate repeat-dose regimen, with endpoints focused on ventricular rate reduction and patient-reported symptom relief.