Praxis Precision Medicines (PRAX) TD Cowen 46th Annual Health Care Conference summary

Regulatory strategy and FDA interactions

• NDA submissions for Ulyxa (essential tremor) and relumitragene were filed, with Ulyxa proceeding under standard review to optimize economic impact and maintain strong FDA relations.

• No significant staff changes in the FDA review team for Ulyxa since June 2023, ensuring continuity and efficient communication.

• The agency has met or exceeded major request timelines, with only minor, non-consequential delays.

Clinical development and product labeling

• Ulyxa's NDA includes a proposal for clinicians to maintain a 20mg dose for an extra week to address tolerability, aiming to increase patient eligibility.

• Labeling discussions are ongoing, with clarity expected by the mid-cycle review meeting.

• Nighttime dosing was allowed in trials for shift workers, and real-world physician flexibility is anticipated.

Launch preparation and commercialization

• Pre-launch activities include disease awareness campaigns, prescriber identification, and sales force build-up, with a focus on both patient and physician education.

• Targeted advertising and disease state education will intensify after the AAN meeting, aiming to multiply patient awareness.

• Specialty pharmacy distribution and robust patient support hubs are planned to streamline access and ensure positive patient experiences.

• Sales force sizing is projected at around 300 to ensure broad coverage, with hiring ramping up as approval timing becomes clearer.