Praxis Precision Medicines, Inc. Q1 FY2022 earnings call

• Lessons from PRAX 944 data impacting the Phase 2b study, with differences in patient populations between PRAX 944 and Essential1. • Enrollment progress in the Essential1 Phase 2b study. • Changes in endpoints for Essential1 with agency support, including modifying ADLs as a key endpoint. • Conduct and data retention in the Aria study, with the last patient last visit on the 5th. • Cash runway moving from 2Q '23 to 3Q '23 due to portfolio prioritization and study delays.

No detailed product segment financial performance discussed in the transcript.

• Cash runway for Praxis Precision Medicines has been revised from 2Q '23 to 3Q '23 due to certain study delays and portfolio prioritization.

• During the call, it was mentioned to consult the SEC filings for additional detail on forward-looking statements and risks associated with the business.

Q: What are some of the key lessons from PRAX 944 data impacting the Phase 2b study and enrollment in Essential1?

A: Marcio Souza noted differences in patient populations between PRAX 944 and Essential1, and key components like a certain level of 3M at baseline. Bernard Ravina mentioned lessons on getting most people to high end of dose range and understanding endpoints like modified ADLs.

Q: Reasons for three discontinuations in the PRAX -114 study?

A: Bernard Ravina said one was unrelated medical condition, one was dizziness, and one was fatigue, all mild-to-moderate with only one severe AE of worsening tremor on drug withdrawal.

Q: Could Essential1 serve as a registrational study?

A: Marcio Souza said it's a matter of discussion with the FDA after finalizing the design, with driving issue being clinically and statistically significant change.

Q: Last patient last visit in Aria study and powering for 3 point difference?

A: Last patient last visit was on the 5th, and confidence in placebo behavior and drug exposure predictability, with focus on conduct and data cleanup.

Q: Increasing enrollment in Essential1 and stats plan?

A: Marcio Souza said it's in flux, exploring ways to prioritize doses and justify protocol amendments for agency discussion.

Q: Cash runway change?

A: Marcio Souza said portfolio prioritization and study delays moved cash runway to Q3 '23.

Q: Tremor rebound after drug removal?

A: Marcio Souza said it's tremor rebound, not physiologic dependence, seen with other medications.

Q: Why some patients had dose reduced in open-label?

A: Bernard Ravina said AEs like dizziness, difficulty paying attention, occurred early, so starting dose lowered for smoother ramp.

Q: Proportion of patients with clinically meaningful responses?

A: Marcio Souza said vast majority of patients responded on ADL, with removal of drug leading to loss of response.

Q: Screen failure rates in Essential1 study?

A: Marcio Souza said patients excluded for not being stable or severe enough, with central reviewer of severity and exclusion of non-severe patients.