Milestone Pharmaceuticals (MIST) Investor update summary

Regulatory and Launch Timeline

• Cardamyst (etripamil nasal spray) is under FDA review with a PDUFA date of March 27, 2025, aiming for a mid-2025 commercial launch if approved.

• $75 million royalty financing is expected upon approval, providing runway into mid-2026 post-approval.

• Patent protection extends to 2042, supporting long-term commercial prospects.

• Launch priorities include sales force hiring, payer profiling, and patient support services.

• Fully integrated leadership team in place to execute a phased commercial launch.

Market Opportunity and Patient Impact

• Over 2 million Americans have PSVT, with 0.5–1 million actively engaged in the healthcare system annually, costing more than $5 billion.

• PSVT is highly symptomatic, unpredictable, and disruptive, with significant emotional and lifestyle impacts, including high rates of anxiety.

• Current treatments are often insufficient, inconvenient, or invasive, leaving a clear unmet need for an on-demand, effective solution.

• Cardamyst aims to empower patients with fast, reliable, self-administered relief, reducing reliance on emergency care.

• Cardamyst targets patients with moderate to severe, not infrequent episodes, representing the majority of the addressable market.

Product Profile and Clinical Data

• Cardamyst is a novel, fast-acting, on-demand L-type calcium channel blocker nasal spray, designed for self-administration during PSVT episodes.

• Demonstrated efficacy: over twice as many patients convert at 30 minutes vs. placebo, with a median time to conversion of 17 minutes and ~40% reduction in emergency department use.

• Safety profile is favorable, with mostly mild, transient nasal side effects and no serious adverse or cardiac events in pivotal trials.