Milestone Pharmaceuticals (MIST) 25th Annual Needham Virtual Healthcare Conference summary

Key product and market updates

• CARDAMYST (etripamil) received FDA approval in December as the first new PSVT treatment in over 30 years, with commercial launch initiated in February and product placed in channels by January.

• The product is a portable, on-demand nasal spray for acute PSVT episodes, designed for patient self-administration at home, offering rapid onset and robust efficacy and safety data.

• Clinical trials showed a median conversion time of 17 minutes and 64% conversion within 30 minutes, with a favorable tolerability profile and minimal serious side effects.

• The company is targeting 10,000 key healthcare providers, covering 500,000 patients in 2026, with 60 fully trained sales reps already in the field.

• Initial commercial coverage was secured with Express Scripts, enabling manageable out-of-pocket costs and streamlined access for patients and providers.

Strategic and financial outlook

• 2026 is expected to be transformative, with the company transitioning from development to commercial stage and initiating phase III trials for AFib with rapid ventricular rate (RVR), a market 3–4 times larger than PSVT.

• The company reported $200 million in pro forma cash as of March, providing runway into late 2027 and line of sight to break-even.

• The market access strategy emphasizes reducing emergency department visits and hospital admissions, offering significant cost offsets for payers.

• Commercial focus in 2026 is on the commercial payer segment, with Medicare coverage expected to impact 2027.

• New-to-brand prescription data and payer coverage metrics will be reported quarterly to track launch progress.

Clinical development and future plans

• Phase III trial for AFib-RVR will use a similar at-home, double-blind design as the PSVT program, leveraging the sNDA pathway for potential single-study approval.

• Phase II data in AFib-RVR showed a rapid, sustained reduction in ventricular rate, supporting advancement to phase III.

• The company is operationally ready to start the phase III AFib-RVR trial, with site engagement underway.

• CARDAMYST can be used alongside or in place of background therapies, offering flexibility for different patient needs.

• No expectation of branded competition in the near term, supporting a strong commercial opportunity.