- Praxis Precision Medicines Inc (NASDAQ:PRAX) announced topline results from Part B of its Phase 2a study evaluating the safety and efficacy of PRAX-944 for essential tremor (ET).
- Treatment with PRAX-944 resulted in clinically meaningful improvements in function, supported by improvements in tremor amplitude.
- In the open-label period through Day 42, patients treated with PRAX-944 demonstrated mean improvement from a baseline of 42% in the daily living score.
- Following randomization, the difference between patients who remained on treatment (N=6) through Day 56 and those randomized to placebo (N=5) was clinically and statistically significant.
- PRAX-944 was generally well tolerated in Part B of the Phase 2a study, with no new safety findings. In the study, eight of eleven participants completed the open-label period at the highest dose of 120 mg.
- Three evaluable participants discontinued during the open-label period due to Adverse Events (AEs).
- Treatment-Emergent Adverse Events (TEAEs) were all mild to moderate, except for one severe AE of essential tremor that occurred in a placebo arm patient following the withdrawal of PRAX-944.
- As of March 31, Praxis held $222.5 million in cash, cash equivalents, and marketable securities, expected to fund operations into Q3 of 2023.
- Price Action: PRAX shares closed 8.54% lower at $7.39 on Monday.
