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

Regulatory strategy and FDA interactions

• NDA for Ulyxa (ulixacaltamide) for essential tremor submitted with standard review, not priority, to optimize economic impact and maintain strong FDA relations.

• Decision driven by IRA negotiation timelines and desire to maximize revenue at peak drug adoption.

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

• Positive and timely interactions with FDA, with leadership overlap between neurology divisions aiding communication.

Clinical and labeling considerations

• NDA submission included a proposal for clinicians to maintain a 20mg dose for an extra week to address tolerability, aiming to increase patient retention.

• Labeling discussions ongoing; proposal supported by phase II data and included in submission.

• Daytime dosing was protocol-driven, but real-world flexibility for nighttime dosing exists and is supported by trial data.

• Tolerability issues are distinct from safety concerns and will be addressed through physician education at launch.

Launch preparation and commercialization

• Pre-launch activities include disease awareness campaigns, prescriber identification, and sales force build-up.

• Targeted advertising and disease state education (DSC) to ramp up after AAN conference.

• Specialty pharmacy distribution and patient support hub planned to streamline access and manage prior authorizations.

• Sales force sizing expected to exceed 300 to ensure broad coverage and avoid launch constraints.

• Sequential hiring approach with full deployment a few months before launch, balancing investment with certainty of approval.