Praxis Precision Medicines Inc (NASDAQ: PRAX) announced topline results from the Essential1 study evaluating the efficacy, safety, and tolerability of ulixacaltamide (PRAX-944) for essential tremor (ET).
In Essential1, ulixacaltamide-treated participants demonstrated improvement relative to placebo participants in the primary endpoint, change from baseline to Day 56 in the modified Activities of Daily Living (mADL1) score, which did not reach statistical significance.
ADL is used as an indicator of a person's functional status.
Nominal statistically significant improvement was observed in the TETRAS-ADL score secondary endpoint.
TETRAS is a validated clinical scale to assess ET severity.
Additional secondary endpoints supported the ulixacaltamide efficacy profile.
Praxis intends to engage with the FDA at the end of the Phase 2 meeting and initiate a Phase 3 study for essential tremors in the second half of 2023 based on the observed efficacy and safety profile.
Ulixacaltamide was well-tolerated. No dose-response relationship was observed between participants assigned to the 60 mg or the 100 mg dose regimens in effect or safety.
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