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BOSTON, Sept. 04, 2024 (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 that it will present preclinical and clinical data from three of its epilepsy programs at the International League Against Epilepsy (ILAE) 15th European Epilepsy Congress (EEC), being held from September 7 to 11, 2024 in Rome, Italy. “We are pleased to highlight the continued progress across our portfolio of epilepsy assets, building on the strong evidence we have already generated in the elsunersen EMBRAVE study and the relutrigine EMBOLD study,” said Steven Petrou, chief scientific officer and co-founder of Praxis. “We are currently enrolling registrational trials for elsunersen and relutrigine, and look forward to initiating our ENERGY program with PRAX-628 this year.” Praxis at EEC 2024 | Roma Convention Center La Nuvola Connect with members of our team at booth #441 or head to one of our presentations listed below where we will share updates across our epilepsy portfolio. Oral Presentation Tuesday, September 10, 01:02 p.m. – 01:10 p.m. CESTLocation: CaravaggioSession: Platform Session – Pediatric Epileptology Significant Seizure Reduction in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy following Treatment with Elsunersen, a Novel Antisense Oligonucleotide: Findings from the EMBRAVE Study Poster Presentations Sunday – Tuesday, September 8 – 10, 1:30 p.m. – 3:00 p.m. CESTLocation: Forum P083. Pharmacokinetics, Tolerability and Cardiac Safety for PRAX-628, a Precision Medicine Therapeutic Set to Initiate Registration Enabling Studies for Focal Onset Seizures P898. A Novel Antisense Oligonucleotide for the Treatment of Early Onset SCN2A Developmental and Epileptic Encephalopathy: A First-in-Patient Report in a Preterm Infant with Refractory Status Epilepticus P908. EMBOLD: A Clinical Trial of PRAX-562 in Subjects with Developmental and Epileptic Encephalopathies P074. A Novel Translational Concordance Framework Identifies Preclinical Seizure Models with Highest Predictive Validity in Focal Onset Seizures P339. A Novel Method to Define an EEG Composite for the Detection of Drug Effects of Next Generation Small Molecules for Epilepsy About Relutrigine (PRAX-562)Relutrigine is a first-in-class small molecule in development for the treatment of developmental and epileptic encephalopathies (DEEs) as a preferential inhibitor of persistent sodium current, shown to be a key driver of seizure symptoms in severe DEEs. Relutrigine’s mechanism of precision sodium channel (NaV) modulation is consistent with superior selectivity for disease-state NaV channel hyperexcitability. In vivo studies of relutrigine have demonstrated dose-dependent inhibition of seizures up to complete control of seizure activity in SCN2A, SCN8A and other DEE mouse models. Relutrigine has been generally well-tolerated in three Phase 1 studies and has demonstrated biomarker changes indicative of NaV channel modulation. Data from the Phase 2 EMBOLD study demonstrated in a heavily pre-treated population a well-tolerated, robust, short- and long-term improvement in motor seizures alongside maintained seizure freedom in some patients with SCN2A- and SCN8A-DEE. Relutrigine has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA, and ODD from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE. About PRAX-628PRAX-628 is a next-generation, functionally selective small molecule targeting the hyperexcitable state of NaV channels in the brain that is currently being developed as a once daily, oral treatment for adult focal onset seizures and generalized epilepsy. Preclinical data demonstrates PRAX-628 is differentiated from standard of care, with the potential to be best-in-class for focal onset seizures. In vitro, PRAX-628 has demonstrated superior selectivity for disease-state NaV channel hyperexcitability. In vivo studies of PRAX-628 have demonstrated unprecedented potency in the maximal electroshock seizure (MES) model, a highly predictive translational model for efficacy in focal epilepsy. Data from the PRAX-628-101 study demonstrated that PRAX-628 can be safely dosed in healthy subjects to greater than 15 times the predicted human equivalent of the rodent MES EC50, a translational indicator that suggests a therapeutic window with unprecedented magnitude relative to approved therapies. About Elsunersen (PRAX-222)Elsunersen is an antisense oligonucleotide (ASO) designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early- onset SCN2A-DEE to treat seizures and other symptoms in patients with gain-of-function SCN2A mutations. In vitro studies of elsunersen have demonstrated reduction in both SCN2A gene expression and protein levels. In vivo, elsunersen has demonstrated significant, dose-dependent reduction in seizures, improvement in behavioral and locomotor activity and increased survival in SCN2A mouse models. Data from the EMBRAVE study demonstrated well-tolerated, significant and sustained seizure reduction in patients with SCN2A-DEE. Elsunersen has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPD) from the FDA, and ODD and PRIME designations from the European Medicines Agency (EMA) for the treatment of SCN2A-DEE. The Elsunersen program is ongoing under a collaboration with Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), and RogCon, Inc. To learn more about the EMBRAVE study, please visit https://www.embravestudy.com/. About Praxis  Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis is applying genetic insights to the discovery and development of therapies for rare and more prevalent neurological disorders through our proprietary small molecule platform, Cerebrum™, and antisense oligonucleotide (ASO) platform, Solidus™, using our understanding of shared biological targets and circuits in the brain. Praxis has established a diversified, multimodal CNS portfolio including multiple programs across movement disorders and epilepsy, with four clinical-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on Facebook, Instagram, LinkedIn and Twitter/X.

▎药明康德内容团队编辑 潜在“first-in-class”癫痫疗法2期临床结果积极 Praxis Precision Medicines今天宣布，在研疗法relutrigine在2期临床概念验证研究中获得积极顶线结果，该研究评估了relutrigine在SCN2A和SCN8A发育性和癫痫性脑病（DEE）患者中的疗效。 名为EMBOLD的2期临床试验是一项随机双盲、安慰剂对照研究，招募了符合条件的2-18岁早发性SCN2A-DEE或SCN8A-DEE患者。试验结果显示，在EMBOLD研究中，relutrigine表现出良好的安全性和耐受性。最常见的不良事件（AE）包括感染、呕吐、发热、嗜睡和便秘，没有患者因不良事件而中途退出。 在疗效方面，接受relutrigine治疗的患者中，经安慰剂矫正的运动性癫痫发作次数与基线相比下降46%。值得一提的是，在接受relutrigine治疗的患者中，5名患者实现了28天无癫痫发作，而安慰剂组中无患者达成这一状态。 ▲Relutrigine显著降低癫痫发作次数（图片来源：Praxis公司官网） Relutrigine是一种潜在“first-in-class”的小分子药物，正在开发用于治疗发育性和癫痫性脑病。它是一款选择性钠离子通道调节剂，可抑制病态钠通道的过度兴奋。持久的钠离子通道过度兴奋是驱动SCN2A-DEE或SCN8A-DEE的关键因素。Relutrigine已获得美国FDA授予的孤儿药资格和罕见儿科疾病（RPD）资格。 31价肺炎球菌疫苗挺进3期临床试验 Vaxcyte今日宣布，其候选31价肺炎球菌结合疫苗VAX-31在一项针对1015名50岁及以上健康成人的1/2期临床试验中取得了积极的顶线结果。基于这一积极结果，该公司已决定将VAX-31推进至成人3期临床试验。 在这项1/2期研究中，VAX-31在所有研究剂量中均显示出良好的耐受性，并在整个六个月的评估期间展现了与获批20价肺炎球菌结合疫苗相似的安全性。VAX-31在所有研究剂量中对所有31种血清型均表现出强劲的调理吞噬作用（OPA）免疫反应。在中剂量和高剂量下，VAX-31与获批疫苗相比，针对两种疫苗共享的20种血清型，均达到或超过了OPA反应非劣效性标准。高剂量的VAX-31与获批20价疫苗相比，20种血清型中18种的平均OPA免疫反应更强，其中7种血清型的免疫反应显著高于活性对照。对于VAX-31独有的11种血清型，所有三种剂量与获批20价疫苗相比均达到了优效性标准。 两项3期临床试验正在进行中，莱姆病候选疫苗最新结果发布 Valneva和辉瑞（Pfizer）公司今天宣布，双方联合开发的莱姆病（Lyme disease）候选疫苗VLA15在2期临床试验中获得积极的免疫原性和安全性数据。数据显示，参与者在接种第二剂增强疫苗（booster）后一个月的免疫反应和安全性与首次接种增强疫苗后的报告相似。新闻稿表示，目前尚无获监管机构批准的人用莱姆病疫苗，而VLA15是临床开发进展最快的莱姆病候选疫苗，目前有两个3期临床试验正在进行中。 最新的2期临床试验VLA15-221的结果再次展示了该候选疫苗可激发针对覆盖的六种血清型的显著记忆性抗体反应，这些结果在儿童（5至11岁）、青少年（12至17岁）和成人（18至65岁）参与者中均保持一致。在第二次增强接种后，高比例的参与者发生了血清转换，所有年龄组中，针对外膜蛋白A（OspA）血清型的血清转换率（SCR）均超过90%，与首次增强接种后的SCR一致。接种首次和第二次增强疫苗一个月后（即第19个月和第31个月），参与者的抗体几何平均滴度相似且维持在较高水平。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] Praxis Precision Medicines announces positive topline results from the EMBOLD study in SCN2A and 8A developmental epilepsies, highlighting the disease-modifying potential of relutrigine. Retrieved September 3, 2024, from https://investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-announces-positive-topline-results-0  [2] Vaxcyte Reports Positive Topline Data from Phase 1/2 Study of VAX-31, its 31-Valent Pneumococcal Conjugate Vaccine Candidate, in Adults Aged 50 and Older. Retrieved September 3, 2024, from https://www.globenewswire.com/news-release/2024/09/03/2939548/0/en/Vaxcyte-Reports-Positive-Topline-Data-from-Phase-1-2-Study-of-VAX-31-its-31-Valent-Pneumococcal-Conjugate-Vaccine-Candidate-in-Adults-Aged-50-and-Older.html [3] Valneva and Pfizer Report Further Positive Phase 2 Booster Results for Lyme Disease Vaccine Candidate. Retrieved September 3, 2024, from https://www.globenewswire.com/news-release/2024/09/03/2939282/0/en/Valneva-and-Pfizer-Report-Further-Positive-Phase-2-Booster-Results-for-Lyme-Disease-Vaccine-Candidate.html 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

Five patients receiving relutrigine went for 28 days without a seizure, compared to none in the placebo cohort, Praxis Precision Medicines noted. Praxis Precision Medicines has scored another midphase win in epilepsy this year, with its sodium channel inhibitor shown to reduce seizures in children with two specific types of the neurological disorder.The EMBOLD study enrolled 16 individuals aged between 2 and 18 years who had been diagnosed with early-onset SCN2A-DEE or SCN8A-DEE—forms of epilepsy for which there are no approved treatments. These patients either received placebo or relutrigine, which inhibits persistent sodium current, a key driver of seizure symptoms in SCN2A-DEE and SCN8A-DEE.Participants who received relutrigine saw an average 46% reduction in their seizures during the double-blind part of the study, Praxis said in a Sept. 3 release. Disrupted movement improved by 23% based on a clinician’s assessment at Week 16, while communication improved by 31% and seizure severity and intensity by 62%.Five patients receiving relutrigine went for 28 days without a seizure, compared to none in the placebo cohort, the biotech noted. The primary endpoint of the trial was the drug’s safety, and Praxis reported that no patients discontinued their treatment due to an adverse event. Relutrigine was “generally safe and well tolerated,” the company said, with seven patients increasing their daily dose from 0.5 mg/kg to 1 mg/kg during the trial.The most common adverse events were infections, vomiting, pyrexia, somnolence and constipation, the biotech said.“When comparing to the baseline rates, patients in EMBOLD had over 2,000 fewer seizures since the beginning of the study,” Praxis CEO Marcio Souza said in the release.“Seizure freedom is the ultimate goal for patients, and we were humbled by the progress made with relutrigine during the EMBOLD study with over 30% of patients achieving this life-altering milestone,” Souza added.Praxis scored another midphase epilepsy win back in March when a high dose of its next-generation NaV blocker PRAX-628 was linked to a 100% complete response rate in epilepsy patients with photoparoxysmal response, a form of photosensitivity.