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Praxis Precision Medicines Announces Positive Data from Randomized Withdrawal Sub-Study and Long-Term Extension of Essential1 Study for Ulixacaltamide
Patients dosed with ulixacaltamide up to 14 weeks showed maintained or improved efficacy results, as measured by mean changes in the modified Activities of
About this update from Praxis Precision Medicines, Inc.
[{"type":"text","content":"Patients dosed with ulixacaltamide up to 14 weeks showed maintained or improved efficacy results, as measured by mean changes in the modified Activities of Daily Living 11 (mADL111) Patients withdrawing from ulixacaltamide to placebo experienced worsening in mADL11 Sub-study execution confirmed design features for the upcoming Phase 3 program expected to initiate in Q4 2023 BOSTON, Aug. 08, 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 further data from two additional analyses of the Essential1 study for 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 and being developed for Essential Tremor. “We were extremely happy to see the OLE and randomized withdrawal results providing further support of the potential durable benefit and safety profile of ulixacaltamide,” said Marcio Souza, president and chief executive officer of Praxis. We believe the randomized withdrawal sub-study confirmed the robustness of the Phase 3 design and, together with the 14-week data from the OLE of Essential1, reinforce the assumptions used for our upcoming Phase 3 program. We look forward to sharing more about these data and our Phase 3 program details at our upcoming R&D portfolio day in early October.” Open-Label Extension Following completion of the initial 8-week double-blind treatment phase in Essential1, eligible patients had the option to continue their access to ulixacaltamide in an open-label extension (OLE) phase. Participants who continued to the OLE phase remained blinded for a 6-week lead-in period. There was no change to the overall safety results through 14 weeks of treatment.65 patients who completed the double-blinded portion of Essential1 were eligible to participate in the OLE2 and completed the week 14 assessment.Patients who were eligible and continued on ulixacaltamide (n= 39) experienced an additional mean improvement in mADL111 of 1.7 points from 3.09 at Week 8 (95% CI: 0.98, 5.2) to...