Praxis Precision Medicines, Inc. Q3 FY2021 earnings call

• Praxis celebrated one-year anniversary as a public company on NASDAQ. • Progress towards bringing CNS drugs to patients with a foundation approach based on four pillars: validating targets through genetics, leveraging translational tools, rigorous development paths, and patient-guided development. • Pipeline progress including clinical programs like PRAX-114, PRAX-944, PRAX-562 with ongoing and upcoming trials. • Financials: Cash and investments at $314 million as of end of third quarter, expected to fund operations into Q2 2023; operating expenses $44.8 million in Q3, up from prior quarter due to R&D expenses for ongoing and upcoming trials.

No specific product segment financial performance details provided in the transcript.

• Expect top line results from PRAX-114 trials in depression in first half of 2022, start Phase 3 study in MDD next year. • PRAX-562 ASSR biomarker study top line results in first half of 2022. • Phase 2 study for PRAX-944 in Parkinson's disease and Phase 2 trial for PRAX-562 in DEEs in first half of 2022. • Phase 1/2 trial for PRAX-222 in first half of 2022. • Top line readouts from PRAX-114 in PTSD and ET in second half of 2022, initiate PRAX-944 Phase 3 trial in ET before end of 2022.

• Forward-looking statements subject to risks and uncertainties. • Actual results could differ materially from forward-looking statements due to business risks; refer to SEC filings and supplement slides for detailed risk factors.

Q: About endpoints in Aria and Acapella study, elevating Day 3, etc.

A: Marcio Souza states they don't have plans to change endpoints, based on preclinical/clinical data and FDA consultation.

Q: About secondary endpoints in Aria study, somnolence and sedation.

A: Bernard Ravina says 114 has a well-tolerated profile, with short-lived hypnotic effect around Cmax, no tolerization to treatment effects.

Q: About Acapella study powering assumptions.

A: Marcio Souza explains Acapella is not powered for accuracy, exploring lower doses with trends in dose-ranging.

Q: About 944 faster titration study, nausea rates.

A: Bernard Ravina says safety profile in fast titration study similar to original, allows flexibility in dosing.

Q: About PTSD and CAPS-5, anxiety components.

A: Bernard Ravina discusses rationale for 114 in PTSD, connection to anxiety, sleep, and CAPS-5 domains.

Q: About essential tremor study data release and bar for moving forward.

A: Marcio Souza talks about expected data in tranches, looking for tremor reduction trends and randomized withdrawal phase results.

Q: About Phase 2 PTSD study design, flexed dose.

A: Marcio Souza explains rationale for flexed dose to understand exposure response and safety in PTSD population