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Praxis Precision Medicines Announces Plans to Begin Ulixacaltamide Phase 3 in Essential Tremor by Year End After Completing End of Phase 2 Meeting with FDA
Phase 3 program to include one parallel group study and one randomized withdrawal study mADL11 as primary endpoint Single 60 mg/day dose to be tested BOSTON,
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"Phase 3 program to include one parallel group study and one randomized withdrawal study mADL11 as primary endpoint Single 60 mg/day dose to be tested BOSTON, June 09, 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 the outcomes from a recent end-of-Phase 2 meeting with the U. S. Food and Drug Administration (FDA) regarding plans to advance ulixacaltamide into Phase 3 for essential tremor (ET). “Our constructive discussion with the FDA established the path forward towards registration in the U.S. for ulixacaltamide. This program brings a new mechanism to a large and underserved population. We are very glad to have the support for so many aspects of the program, including two different Phase 3 trial designs, which provide multiple ways to demonstrate efficacy while also being efficient with resources,” said Marcio Souza, president and chief executive officer of Praxis. Key elements of the registration plan: Use of the modified Activities of Daily Living 11 (mADL111) as the primary endpoint is acceptable. In the Phase 2 Essential1 study, mADL11 was nominally significant (p=0.042)Agreement to use a single dose of 60 mg for the Phase 3 trialsBase case assumption confirmed for two Phase 3 trials, one of which will be a 12-week, parallel design study and one of which will be a 12-week randomized withdrawal study for stable respondersSafety database required for a New Drug Application (NDA) at the minimum required by ICH guidelines: 300 patients with six-months of exposure and 100 patients with one-year of exposureAgreement that the completed and planned clinical pharmacology and toxicology studies would be sufficient for submission of an NDA The protocols for the Phase 3 trials are being finalized and Praxis intends to submit to the current Investigational New Drug Application shortly. About Ulixacaltamide Ulixacaltamide is a differentiated and highly selective small molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity. Ulixacaltamide, the most advanced program within Pr...