Praxis Precision Medicines (PRAX) 2025 Conference June 04, 2025 08:10 AM ET Speaker0 Good morning everyone. Welcome to the Jefferies Global Healthcare Conference twenty twenty five. With me today I have the great pleasure of introducing Praxis Precision Medicine's CEO and dear friend Marcio Souza. Marcio, how are you doing this morning? Speaker1 Doing great. Living the dream. Speaker0 Amazing. Well, we'll just jump right into it because there's so much exciting work you guys are doing at Praxis. Top of mind ...

Praxis Precision Medicines (PRAX) FY 2025 Conference May 20, 2025 09:30 AM ET Speaker0 Okay. Welcome, everybody. I'm Doug Sal, senior analyst at H. C. Wainwright. We are thrilled to have with us Praxis Precision Medicines, represented by the company's CFO, Tim Kelly. And Marcio Sauso, the company's CEO, is supposedly en route, so he might join us midway through the presentation. So you don't have to worry if somebody barges in and sits down. Speaker1 So it's Marcio versus the trains this morning. So I I thi ...

BOSTON, May 20, 2025 (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 that it will be participating in three upcoming investor conferences: About Praxis The Company will be available for one-on-one meetings during all events. Interested investors should contact their ...

市场机会 - Vormatrigine针对3.5百万常见癫痫患者的市场机会超过25亿美元[20] - Relutrigine针对超过20万高癫痫负担的发育性癫痫患者的市场机会超过30亿美元[20] - Elsunersen针对约2000名SCN2A基因特征的发育性癫痫患者的市场机会超过5亿美元[20] 临床试验结果 - Vormatrigine在临床试验中实现了100%的患者反应率，且在治疗后达到3-13倍的MES EC50暴露[29] - Relutrigine在28天周期内显示出显著的癫痫发作减少和前所未有的无发作状态[41] - EMBOLD Cohort 1研究显示，使用Relutrigine治疗的患者在持续暴露下实现了高达90%的中位数癫痫发作减少[58] - 在EMBOLD Cohort 1中，70%的患者在基线时稳定使用钠通道阻滞剂，且不良事件大多为轻度至中度[59] - EMBOLD Cohort 1结果显示，患者的无癫痫发作天数在治疗期间显著增加，达到最高63天[61] - 在EMBOLD Cohort 1中，57%的患者在警觉性方面表现出改善，71%的患者在癫痫发作严重性和强度方面有所改善[63] - Elsunersen在SCN2A GoF患者中显示出39%至43%的癫痫发作减少，且无不良事件被认为与研究药物相关[82] 未来展望 - 预计POWER1研究的顶线结果将在2025年下半年公布[40] - EMBOLD Cohort 2的顶线结果预计在2026年上半年公布，2026年将提交新药申请(NDA)[17] - 预计EMERALD广泛发育性癫痫研究将在2025年中期启动[17] - 2025年下半年将开始POWER2的入组[17] - 预计2025年第三季度将公布Essential3研究的顶线结果[117] - 2026年将提交SCN2A GoF的NDA申请[117] - PRAX-100 SCN2A LoF的候选声明预计在2025年中发布[118] 现金流与市场动态 - 公司的现金流预计将持续到2028年[6] - 当前美国发展性和癫痫性脑病（DEE）市场患者超过200,000人，预计未来几年将随着护理和诊断的改善而增加[48] 新产品与技术研发 - Solidus的临床前产品组合预计将在2026年进入临床试验[100] - EMBRAVE Part A支持注册包，显示出Elsunersen的临床意义[86] - EMBOLD Cohort 2设计为关键研究，以确认Relutrigine的疗效[65] - EMBRAVE3注册试验将扩展治疗至出生时，涵盖系统性年龄基础的患者[93] 医生与患者管理 - 40%的患者在神经科医生处未接受治疗[108] - 85%的神经科医生就寻求ET治疗的患者进行就诊[108] - 近一半的神经科医生很少将ET患者转诊给专科管理[108] 评估与研究 - Essential3研究的主要终点与FDA一致，评估日常生活活动的改善[111] - TETRAS ADL量表的总分为33，评估患者日常活动能力[109] - Ulixacaltamide在Essential3研究中进行两项研究，分别为安慰剂对照平行组研究和随机撤药研究[113]

Praxis Precision Medicines (PRAX) Update / Briefing May 02, 2025 10:00 AM ET Speaker0 Good morning, everyone, and thanks for being with us here today in the office in Boston. I really appreciate it. And everyone else in the webcast as well for our GE portfolio updates at Praxis. As you can see here at the beginning, the way we tend to think about everything at Praxis is daring for more. So we're there for more today as well. I think it's a great show here in a not so sunny day in Boston, and hopefully, gonn ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 ☐ For the transition period from to Commission File Number: 001-39620 PRAXIS PRECISION MEDICINES, INC. N/A (Exact Name of Registrant as Specified in its Charter) Delaware 47-5195942 (State or o ...

Praxis Precision Medicines Provides Corporate Update and Reports First Quarter 2025 Financial Results On track for six major study readouts across four programs over the next 12 months Ready to initiate pivotal studies in two developmental and epileptic encephalopathy (DEE) programs in mid- year 2025: EMERALD for broad DEEs with relutrigine and EMBRAVE3 for SCN2A GoF with elsunersen Vormatrigine continues to generate a best-in-class safety profile with new data demonstrating no food effect and higher dosing ...

On track for six major study readouts across four programs over the next 12 months Ready to initiate pivotal studies in two developmental and epileptic encephalopathy (DEE) programs in mid-year 2025: EMERALD for broad DEEs with relutrigine and EMBRAVE3 for SCN2A GoF with elsunersen Vormatrigine continues to generate a best-in-class safety profile with new data demonstrating no food effect and higher dosing tolerability Praxis to host a virtual investor event on Friday, May 2, 2025 to discuss its DEE portfol ...

文章核心观点 临床阶段生物制药公司Praxis Precision Medicines宣布将于2025年5月2日上午10点至11点30分举办虚拟投资者活动，聚焦公司在发育性和癫痫性脑病的临床项目 [1][2] 公司信息 - 公司是临床阶段生物制药公司，将遗传性癫痫见解转化为中枢神经系统疾病疗法开发，利用专有小分子平台Cerebrum™和反义寡核苷酸平台Solidus™开展疗法研发，拥有多元化多模式中枢神经系统产品组合，有四个临床阶段候选产品 [3] 活动信息 - 活动由公司管理团队主持，聚焦公司在发育性和癫痫性脑病的临床项目 [2] - 可点击链接报名参加直播活动，活动回放将在公司网站“投资者+媒体”板块的“活动与演示”页面提供90天 [2] 临床项目 - EMBOLD研究评估relutrigine用于SCN2A和SCN8A发育性和癫痫性脑病 [4] - EMERALD研究评估relutrigine用于更广泛的发育性和癫痫性脑病 [4] - EMBRAVE3研究评估elsunersen用于SCN2A功能获得性发育性和癫痫性脑病 [4]

文章核心观点 公司宣布4月将参加两场投资者会议，管理层将在会议上进行公司概况介绍，会议网络直播回放将在公司网站提供90天 [1][2][4] 公司参会信息 - 公司将参加4月的两场投资者会议，分别是4月7日的第24届年度Needham虚拟医疗保健会议和4月17日的Piper Sandler春季生物制药研讨会 [1][4] - 公司管理层将在4月7日的会议上进行公司概况介绍，会议将进行网络直播 [4] - 两场会议期间公司可安排一对一会议，感兴趣的投资者可联系代表申请 [1] 会议回放信息 - 网络直播活动的回放将在公司网站“投资者+媒体”板块的“活动与演示”页面提供90天 [2] 公司简介 - 公司是临床阶段生物制药公司，利用遗传见解开发治疗中枢神经系统疾病的疗法 [1][3] - 公司通过专有小分子平台Cerebrum™和反义寡核苷酸（ASO）平台Solidus™开发疗法 [3] - 公司建立了多元化、多模式的中枢神经系统产品组合，有四个临床阶段候选产品 [3] 联系方式 - 投资者联系邮箱为investors@praxismedicines.com，电话为857 - 702 - 9452 [4] - 媒体联系人为Dan Ferry，邮箱为Daniel@lifesciadvisors.com，电话为617 - 430 - 7576 [4]