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Praxis Precision Medicines Announces Topline Results from the Essential1 Study of Ulixacaltamide for the Treatment of Essential Tremor and Continued Advancement of Program to Phase 3

Based upon observed efficacy and safety profile, Praxis intends to engage with the FDA in an end of Phase 2 meeting and initiate a ulixacaltamide Phase 3 study for the treatment of essential tremor in 2H23

Ulixacaltamide demonstrated improvement in modified Activities of Daily Living (mADL) primary efficacy endpoint relative to placebo that did not reach statistical significance (p=0.126), and achieved nominal statistical significance in TETRAS-ADL secondary endpoint (p=0.026)

Nominal statistically significant improvements observed in Clinical Global Impression-Severity (CGI-S) and in Patient Global Impression-Change (PGI-C)

Ulixacaltamide was well tolerated in the Essential1 study, with no new safety findings

Excerpt from the Press Release:

BOSTON, March 03, 2023 (GLOBE NEWSWIRE) — Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced topline results from the Essential1 study evaluating the efficacy, safety and tolerability of ulixacaltamide (PRAX-944) for the treatment of 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, that did not reach statistical significance. Nominal statistically significant improvement was observed in the TETRAS-ADL score secondary endpoint. Additional secondary endpoints were supportive of the ulixacaltamide efficacy profile, including nominal statistically significant improvements in the Clinical Global Impression-Severity (CGI-S) and Patient Global Impression-Change (PGI-C) scores. Praxis intends to engage with the FDA in an end of Phase 2 meeting and initiate a Phase 3 study for the treatment of essential tremor in the second half of 2023 based upon the observed efficacy and safety profile.

“The impact of essential tremor transcends the action tremor that is the hallmark of the disease. Essential tremor can be debilitating, causing significant disruption of everyday activities. There is significant unmet need for an effective and tolerable precision medicine that can improve patient functioning, and to date there exists no medicine specifically developed for tremor,” said William G. Ondo, M.D., Professor of Neurology at the Houston Methodist Hospital and Weill Cornell Medical College. “The results from Essential1 give hope to the essential tremor community that a targeted therapy is within reach, and we eagerly anticipate additional data from the ulixacaltamide program.”

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