Core Insights - Praxis Precision Medicines (PRAX.US) shares surged 240% to a three-year high of $194.90 following the announcement of positive results for their essential tremor drug Ulixacaltamide in two pivotal Phase III studies [1] Group 1: Study Results - Study 1 demonstrated a 4.3-point improvement in the Modified ADL-11 score at week 8 with a p-value of <0.0001, and all key secondary endpoints showed statistical significance [1] - Study 2 indicated significant maintenance of effect with a p-value of 0.0369, and the first key secondary endpoint also showed statistical significance with a p-value of 0.0042 [1] Group 2: Safety and Regulatory Progress - Ulixacaltamide exhibited good safety data, with no drug-related serious adverse events reported in both studies, and tolerability consistent with previous trials [1] - The company has submitted a pre-NDA meeting request to the FDA to advance the drug approval process [1]

周四，Praxis Precision Medicines(PRAX.US)早盘飙升240%，创三年新高，报194.90美元。消息面上，该 公司宣布特发性震颤药物Ulixacaltamide在两项关键III期研究中达到主要终点：Study1显示第8周 Modified ADL-11评分改善4.3分p<0.0001，所有关键次要终点均具统计学显著性;Study2显示维持效果显 著p=0.0369，且第一个关键次要终点具统计学显著性p=0.0042。Ulixacaltamide安全性数据良好，两项研 究中未出现药物相关严重不良事件，耐受性与既往试验一致。公司向FDA提交了Ulixacaltamide的新药 预申请pre-NDA会议请求，推进药物审批进程。 ...

Patients treated with ulixacaltamide in the parallel-group study (Study 1) showed a mean improvement from baseline in the Modified Activities of Daily Living 11 at Week 8, the pre-specified primary endpoint, of 4.3 points (p<0.0001) All key secondary endpoints in Study 1 - rate of disease improvement over 12 weeks, PGI-C and CGI-S - were also statistically significant (p<0.001) Study 2 met its pre-specified primary endpoint, with patients showing superior maintenance of effect while on ulixacaltamide versu ...