This study will investigate if the drug sodium phenylbutyrate (NaPB) impacts blood pressure and vascular function in healthy volunteers and in patients with diabetes.

开始日期2025-05-01

申办/合作机构

University of Pennsylvania

[+1]

NCT06773026

/ Recruiting临床2期IIT

A Phase 2, Open-label, Fixed-dose Study to Assess the Efficacy of Sodium Phenylbutyrate (ACER-001) in Treating Pediatric and Adult Patients with Medium Chain Acyl-CoA Dehydrogenase (MCAD) Deficiency Resulting from the Prevalent ACADM C.985 A>G (K304E) Mutation

This is a medical research study to test a medication in patients 4 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A>G (K304E) mutation. The medication is sodium phenylbutyrate (ACER-001), which is currently FDA approved for the treatment of Urea Cyle Disorders. Previous research suggests that sodium phenylbutyrate may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of sodium phenylbutyrate in patients with MCADD.

开始日期2025-04-01

申办/合作机构

University of Pittsburgh

[+1]

ChiCTR2400086139

/ Suspended早期临床1期IIT

A clinical effectiveness study of Sodium phenylbutyrate in the treatment of polycystic ovary syndrome

开始日期2024-07-01

申办/合作机构

复旦大学附属中山医院

100 项与苯基丁酸钠相关的临床结果

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100 项与苯基丁酸钠相关的转化医学

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100 项与苯基丁酸钠相关的专利（医药）

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3,736

项与苯基丁酸钠相关的文献（医药）

2025-04-01·Neural Regeneration Research

Treating amyotrophic lateral sclerosis with allogeneic Schwann cell–derived exosomal vesicles: a case report

Article

作者: Pearse, Damien D. ; Dietrich, W. Dalton ; Levi, Allan D. ; Guest, James D. ; Silvera, Risset ; Goldschmidt, Alexander J.P. ; Brooks, Adriana ; Graham, Patricia ; Goldschmidt-Clermont, Pascal J. ; Jimsheleishvili, George ; Khan, Aisha

Schwann cells are essential for the maintenance and function of motor neurons, axonal networks, and the neuromuscular junction. In amyotrophic lateral sclerosis, where motor neuron function is progressively lost, Schwann cell function may also be impaired. Recently, important signaling and potential trophic activities of Schwann cell-derived exosomal vesicles have been reported. This case report describes the treatment of a patient with advanced amyotrophic lateral sclerosis using serial intravenous infusions of allogeneic Schwann cell-derived exosomal vesicles, marking, to our knowledge, the first instance of such treatment. An 81-year-old male patient presented with a 1.5-year history of rapidly progressive amyotrophic lateral sclerosis. After initial diagnosis, the patient underwent a combination of generic riluzole, sodium phenylbutyrate for the treatment of amyotrophic lateral sclerosis, and taurursodiol. The patient volunteered to participate in an FDA-approved single-patient expanded access treatment and received weekly intravenous infusions of allogeneic Schwann cell-derived exosomal vesicles to potentially restore impaired Schwann cell and motor neuron function. We confirmed that cultured Schwann cells obtained from the amyotrophic lateral sclerosis patient via sural nerve biopsy appeared impaired (senescent) and that exposure of the patient’s Schwann cells to allogeneic Schwann cell-derived exosomal vesicles, cultured expanded from a cadaver donor improved their growth capacity in vitro. After a period of observation lasting 10 weeks, during which amyotrophic lateral sclerosis Functional Rating Scale-Revised and pulmonary function were regularly monitored, the patient received weekly consecutive infusions of 1.54 × 1012 (×2), and then consecutive infusions of 7.5 × 1012 (×6) allogeneic Schwann cell-derived exosomal vesicles diluted in 40 mL of Dulbecco’s phosphate-buffered saline. None of the infusions were associated with adverse events such as infusion reactions (allergic or otherwise) or changes in vital signs. Clinical lab serum neurofilament and cytokine levels measured prior to each infusion varied somewhat without a clear trend. A more sensitive in-house assay suggested possible inflammasome activation during the disease course. A trend for clinical stabilization was observed during the infusion period. Our study provides a novel approach to address impaired Schwann cells and possibly motor neuron function in patients with amyotrophic lateral sclerosis using allogeneic Schwann cell-derived exosomal vesicles. Initial findings suggest that this approach is safe.

2025-04-01·FOOD CHEMISTRY

New insights into the tenderization pattern of yak meat by ROS-ERS: Promotion of ERS-associated apoptosis through feedback regulation of PERK/IRE1/ATF6 and caspase-12 activity

Article

作者: Shi, Hongmei ; Duan, Yufeng ; Yang, Xue ; Song, Rende ; Zhang, Li ; Li, Yiheng ; Kong, Xiangying ; Hua, Yongli ; Zhang, Xinjun ; Shen, Ruheng

This study aimed to investigate the molecular mechanisms of reactive oxygen species (ROS)-induced endoplasmic reticulum stress (ERS) in apoptosis and meat tenderization during postmortem aging. Yak longissimus dorsi muscle was incubated with N-acetylcysteine (NAC), 4-phenylbutyric acid (4-PBA) and NAC + 4-PBA, respectively, and stored at 4 °C for 0 h, 12 h, 24 h, 72 h, 120 h and 168 h. The results showed that NAC and 4-PBA treatments significantly reduced ROS content and endoplasmic reticulum stress levels. Meanwhile, the specific inhibitory effect of 4-PBA affected Ca²⁺ content, caspase-12 activity, endoplasmic reticulum apoptotic cascade reaction and meat tenderization by preventing myogenic fiber degradation. Additionally, the combined treatment of NAC + 4-PBA had a more significant effect than the other groups, confirming the necessity of targeted regulation of the ROS-ERS axis. Overall, our findings provide new insights into the critical role of ROS-mediated ERS in caspase-12-dependent apoptosis and yak meat tenderization during yak meat postmortem.

2025-03-01·Molecular Genetics and Metabolism Reports

Single centre retrospective review of plasma branched-chain amino acid levels in children with urea cycle disorders: Impact of treatment modalities and disease severity

Article

作者: Dixon, Marjorie ; Yeo, Mildrid ; Rehsi, Preeya ; Chakrapani, Anupam ; Yeo, Jie Ming

Branched-chain amino acids (BCAAs) are important for normal growth, development, and function. In urea cycle disorders (UCDs), plasma BCAA levels can be relatively low; this has been attributed variously to low protein intake, hyperammonaemia, and nitrogen scavenger treatment. We undertook a retrospective review of plasma BCAA levels in individuals with UCDs comprising ornithine carbamoyltransferase deficiency (OTCD n = 22), arginosuccinate lyase deficiency (ASLD n = 12), and argininosuccinate synthase deficiency (ASSD n = 6). Scavenger treatment groups comprised sodium benzoate (NaBz, n = 20), sodium phenylbutyrate (NaPBA, n = 5), NaBz+NaPBA (n = 14), and a control group receiving neither NaBz nor NaPBA (n = 14). In these treatment groups, respectively, median (IQR) plasma levels of leucine were 54 (32), 55 (25), 58 (19), and 91 (70) μmol/L (leucine was lower in the NaBz group than the control, p = 0.0282) and numbers of individuals (%) with leucine below normal were 10/20 (50 %), 1/4 (25 %), 10/14 (71 %), and 2/9 (20 %). The pattern was similar for isoleucine and valine. In the NaBz group, plasma BCAA levels were inversely correlated with protein intake (p ≤ 0.01 to p ≤ 0.001), plasma ammonia level (p ≤ 0.01 to p ≤ 0.0001), and scavenger dose (p ≤ 0.0001). We speculate that individuals with greater disease severity may be prone to BCAA deficiency, caused by BCAA consumption when alternative urea disposal pathways are used. Practical reflections on our audit were that to increase the proportion of plasma BCAA levels in the normal range, we needed to alter the biological value of protein intake, prescribe higher doses of scavenger to facilitate safe levels of protein intake, and give EAA supplements if indicated.

359

项与苯基丁酸钠相关的新闻（医药）

2025-02-28

·喜鹊医药公司

北医三院樊东升：渐冻症药物临床试验最新进展汇总 | 前沿聚焦

本文内容来源：破冰心锲以上视频为渐愈互助之家邀请ALS权威专家、北医三院樊东升教授线上直播的内容。 1、AMX0035（苯丁酸钠和牛磺酸二醇组合） 2024年4月4日，Amylyx Pharmaceuticals宣布与美国食品和药物管理局（FDA）和加拿大卫生部达成协议，主动撤市治疗肌萎缩侧索硬化（ALS）患者的药物Relyvrio（AMX0035）。 2024年3月8日，Amylyx Pharmaceuticals公司宣布，经FDA批准的渐冻症（ALS）药物Relyvrio（AMX0035）在关键的III期PHOENIX研究中未达到主要终点。这对被认为是治疗这种致命的神经性疾病为数不多有效药物之一的Relyvrio是一个重大打击。PHOENIX 是一个全球性的对664名ALS患者进行的 48 周随机安慰剂对照的 III期临床试验。在公布的数据中，Amylyx的药物表现不佳，也未达到患者报告的生活质量结果评估、总生存期以及慢肺活量（SVC）衡量的呼吸功能等次要终点。 2022年9月29日，美国FDA宣布，批准Relyvrio（苯丁酸钠和牛磺酸二醇口服固定剂量配方）上市，用于治疗肌萎缩侧索硬化（ALS）。这也是116年以来FDA批准的第3款ALS治疗药物。 Relyvrio的售价约为15.8万美元，约合114.09万人民币/年。 2022年06月15报道，Amylyx制药公司宣布，加拿大卫生部（Health Canada）已附条件批准Albrioza（又名AMX0035，PB-TURSO）：用于治疗肌萎缩侧索硬化症（ALS，俗称“渐冻症”）。 2022年5月9日报道，Amylyx公布了AMX0035治疗肌萎缩侧索硬化症（ALS）2期CENTAUR试验校正安慰剂交叉后的长期生存分析结果。事后分析表明，经校正安慰剂交叉，AMX0035与安慰剂相比，表现出更大的生存获益，患者中位生存期延长10.6至18.8个月；而在最初的预先指定意向治疗（ITT）分析中，AMX0035与安慰剂相比，患者中位生存期仅延长了6.8个月。相关结果发表于同行评议医学期刊《Muscle & Nerve》。链接： 3期临床失败，Amylyx渐冻症新药确定主动撤市老药组方Relyvrio渐冻症III期失败，Amylyx自愿暂停产品销售渐冻症药物信息（十二）：Amylyx Pharmaceuticals公司的AMX0035 2、托夫生注射液 2024年10月8日，国家药监局（NMPA）官网显示，渤健（Biogen）的托夫生注射液（商品名：凯盛迪™）获批进口，用于治疗SOD1 基因突变的成人肌萎缩侧索硬化症 (ALS)。这是全球首个SOD1-ALS疾病修正治疗药物，在国内通过免临床途径获批上市。根据公示，渤健在2023年7月14日向中国国家药监局药品审评中心（CDE）递交了5.1类新药托夫生注射液的上市申请，并获得受理。 2024年5月，托夫生在欧盟获准用于医疗用途。 2024年3月21日，托夫生首先在我国瑞金海南医院应用于临床；不到 7 个月后，托夫生正式在我国获批。 2023年4月25日，美国食品和药物管理局（FDA）宣布，批准QALSODY（Tofersen/托夫生）100毫克/15毫升注射治疗患有超氧化物歧化酶1突变（SOD1）的成人肌萎缩侧索硬化症。该适应症是根据QALSODY治疗的患者中观察到的血浆神经丝轻链（NfL）减少的基础上加速批准的。正在进行的Tofersen在症状前患者中的3期ATLAS研究SOD一ALS将作为验证试验。神经丝是神经元受损时释放的蛋白质，是神经退化的标志。QALSODY是第一种针对ALS基因突变的治疗方法。链接：渐冻症药物来了！「托夫生注射液」国内获批上市渐冻症首个SOD1对因治疗药物在乐城完成国内首例应用其他在研的SOD1药物又有哪些？【加速批准】FDA批准了Biogen（渤健）公司的渐冻症新药QALSODY（Tofersen/托佛森）-治疗SOD1基因突变 3、SAR443820 2024年2月16日，在一份监管文件中披露，Denali Therapeutics与赛诺菲合作开发治疗肌萎缩性侧索硬化症（ALS，渐冻症）的RIPK1抑制剂SAR443820/DNL788的2期Himalaya试验（共招募305名参与者）未能达到ALS功能评分量表（AL-SFRS-R）的主要终点。受该消息影响，周五Denali的股价在早盘交易中下跌了近8%。SAR443820/DNL788是一种有效的、选择性的、脑渗透的RIPK1小分子抑制剂，RIPK1是肿瘤坏死因子受体通路中的关键信号蛋白，是炎症和细胞死亡的调节因子。大脑中RIPK1活性的增加导致神经炎症和细胞坏死，并导致神经退行性变。在阿尔茨海默病、ALS等自身免疫性疾病和神经系统疾病的临床前模型中，RIPK1抑制已被证明具有有益的作用。根据中国药物临床试验登记与信息公示平台，赛诺菲本次登记的是一项随机、双盲、安慰剂对照的国际多中心2期临床研究，旨在ALS成人受试者中评估SAR443820的疗效和安全性。该研究计划在国际入组261人，中国境内入组44人。研究的主要目的是评估SAR443820与安慰剂相比在减缓ALS进展方面的作用。链接： Denali/赛诺菲的ALS药物2期试验未能达到主要终点 4、中国也会参与的诺华公司申报的临床诺华（Novartis）：VHB937注射用浓溶液作用机制：TREM2激活抗体适应症：肌萎缩侧索硬化 2024年12月，诺华申报的1类新药VHB937注射用浓溶液获批临床，拟用于肌萎缩侧索硬化（ALS）的治疗。根据诺华公开资料，VHB937是一种稳定和激活TREM2全人源抗体。TREM2是一种跨膜受体，其功能的降低可能会导致阿尔茨海默病和其他形式的痴呆症。VHB937可选择性激活人M2A巨噬细胞和人iPS小胶质细胞中具有亚纳摩亲和力的TREM2，并通过减少脱落增加TREM2细胞表面表达。诺华正在美国开展一项2期临床，旨在评估VHB937对早期ALS患者（ALS症状出现2年内）的疗效和安全性。 10月9日，诺华的VHB937注射用浓溶液的临床试验（IND）申请获中国CDE受理。链接：渐冻症药物信息（一二八）：诺华公司的VHB937注射用浓溶液 5、硝酮嗪 2023年10月15日，广州喜鹊医药有限公司(简称喜鹊医药)1类创新化学药物硝酮嗪(TBN)治疗肌萎缩侧索硬化(ALS)临床II期试验总结会暨III期试验方案讨论会在北京顺利召开。本次会议特邀硝酮嗪治疗ALS 临床II期研究的11家临床医院的研究者参会。喜鹊医药创始人王玉强教授向参会专家表示热烈的欢迎，并介绍了硝酮嗪的研发历程。喜鹊医药自主开发的“全球新”1类小分子药物硝酮嗪的骨架分子来源于传统中药川芎的活性成分，喜鹊团队通过对其分子结构进行修饰优化，使其药理活性和安全性大幅提升，并具有独特的作用机理。作为中药现代化的典型代表，硝酮嗪曾连续三次荣获国家“新药创制”重大专项资助，在缺血性脑卒中、糖尿病肾病等难治性疾病领域分别完成了中国临床II期试验，并获得美国I期临床试验批件。喜鹊医药临床医学负责人张在军博士系统地汇报了硝酮嗪治疗ALS II期临床的试验结果。临床II期试验结果显示，硝酮嗪具有良好的安全性和耐受性，在入组的155例患者中，尽管硝酮嗪未达到治疗第180天时ALSFRS-R评分相对基线差值组间差异的主要终点，但在核心次要终点指标中，与安慰剂组比较，硝酮嗪治疗显著延缓ALS患者握力下降(P=0.037)；在年龄小于65岁、进展较慢的患者(筛选期3个月内ALSFRS-R评分下降1-2分)中，硝酮嗪呈剂量依赖性地延缓多项核心指标下降幅度，硝酮嗪1200 mg治疗显著改善握力、延髓功能和呼吸功能评分，比安慰剂对照组改善分别达到50.14% (P=0.011)、64.71% (P=0.040)和85.92% (P=0.021)。链接：重磅JAMA论文！喜鹊医药创新药物TBN治疗ALS临床2期试验研究成果登JAMA Network Open | 喜鹊动态 6、藿苓生肌颗粒 2024年12月，上海医药全资子公司上海上药睿尔药品有限公司自主研发的SRD4610用于肌萎缩侧索硬化症（ALS）适应症获得美国食品药品监督管理局孤儿药资格认定（Orphan-drug Designation）。SRD4610是一种复方中药创新药，目前该项目已在国内完成针对肌萎缩侧索硬化症的Ⅱ期临床试验。该项目由上海医药自主研发，截至目前，没有同靶点的产品获得FDA批准上市以治疗ALS。藿苓生肌颗粒处方来源于上海中医药大学附属医院的临床经验方，主治精气损伤，气血亏虚，脏腑不足，经脉肌肉失养所致肌肉萎缩，肢体无力，肌肉震颤，肌萎缩侧索硬化症见上述证候者。该研究由北京大学第三医院担任组长单位，由河南省人民医院、中山大学附属第一医院、广东省中医院、浙江大学医学院附属第二医院、四川大学华西医院、西安交通大学第一附属医院、福建医科大学附属协和医院、哈尔滨医科大学附属第一医院等15家研究中心共同开展。本研究为Ⅱ期、多中心、随机、双盲、与利鲁唑平行对照、优效设计研究，以验证藿苓生肌颗粒和利鲁唑对中医诊断为脾气不足、肾阳亏虚证的ALS患者48周的疗效和安全性。这是首次使用标准循证方法评估中药治疗ALS的临床研究方案。临床前研究显示，中等剂量藿苓生肌中药配方（简称：藿苓生肌方）能够显著延长SOD1G93A小鼠的平均生存期和病程。此外，藿苓生肌方能防止腰椎脊髓运动神经元丢失，纠正细胞凋亡相关蛋白的异常表达；缓解腓肠肌萎缩和神经肌肉接头的丧失，抑制神经胶质细胞活化并抑制炎症反应。上述结果表明藿苓生肌方是治疗ALS的潜在新药。链接：上海医药SRD4610获得美国FDA孤儿药资格认定 7、MTS004口崩片 2022年8月2日，杭州剂泰医药(METiS Pharmaceuticals)宣布企业自主研发的MTS004口崩片于2022年6月获国家药监局药品审评中心（CDE）批准药物临床试验（IND）。MTS004口崩片于同年3月向CDE提出临床试验申请，是一款2.2类改良型新药，适应症为假性延髓情绪（PBA），属于神经系统疾病。假性延髓情绪（Pseudobulbar Affect，PBA），是一种继发于多种神经系统疾病或损伤的情绪表达障碍。通常表现为频繁、不自主、无法控制的与患者情绪状态不协调或不相称的大笑和/或大哭，俗称“强哭强笑综合征”。III期临床试验是一项在继发于神经系统疾病的假性延髓情绪患者中评估MTS004的疗效和安全性的双盲、随机、安慰剂对照、多中心研究。该临床试验，在樊东升教授的带领下，于2023年4月8日正式启动，2023年6月27日首例受试者入组（FPI），2024年4月8日第264例受试者完成入组，入组历时仅9.5个月，效率高、质量好。试验受到北京市卫健委高度重视，成功经验已整理成文并发送各区卫健委、北京经济技术开发区社会事业局、各医疗机构学习。链接：剂泰医药MTS004口崩片IND获批，用于治疗假性延髓情绪（PBA）「新锐公司」剂泰医药：人工智能技术平台AiTEM，加速实现药物开发“量子”飞跃 8、依达拉奉舌下片依达拉奉舌下片是南京百鑫愉医药有限公司和北京天坛医院共同开发、具有独立自主知识产权的2.2类改良型新药，用于抑制肌萎缩侧索硬化所致功能障碍的进展，是全球首个渐冻症舌下给药上市药物。 2021年5月，南京百鑫愉医药提交依达拉奉舌下片的上市申请，2021年7月该上市申请获CDE受理，同时被纳入优先审评，适应症为抑制肌萎缩侧索硬化（ALS）所致功能障碍的进展。2022年9月14日获得新药注册（国药准字H20220019 ）。 2023年2月18日，由南京百鑫愉医药主办的依达拉奉舌下片真实世界研究启动会暨奉易达上市会，在北京成功召开。目前北京大学第三医院进行依达拉奉舌下片治疗肌萎缩侧索硬化症 (ALS) 的多中心、前瞻性、非干预登记研究 (真实世界研究)。链接：渐冻症药物信息（七二）：南京百鑫愉医药的依达拉奉舌下片 —— end —— （特别声明：以上内容来源于破冰心锲、蔡磊渐愈互助之家、樊东升教授、蔡总-渐愈互助-1群、网络，版权归原作者所有，本文不构成任何投资建议，且非治疗方案推荐，如有侵权，请联系删除，谢谢）

临床3期上市批准申请上市临床2期

2025-02-27

·FiercePharma

Zevra to net $150M from priority review voucher sale tied to Miplyffa approval

The $150 million price tag for Zevra’s PRV is slightly above the $100 million mark that many vouchers have traded at in the past, though it’s in line with several other recent transactions. For Zevra Therapeutics, last year’s approval of the rare lysosomal storage disorder drug Miplyffa appears to be the gift that keeps on giving.After receiving a rare pediatric disease priority review voucher (PRV) in tandem with Miplyffa’s FDA green light in September, Zevra has locked in a deal to sell the PRV for $150 million.Zevra did not disclose the identity of the purchaser. The transaction itself is expected to occur within 30 to 45 days, the company said Thursday.The cash from the sale will be used to fuel R&D, plus the commercial rollouts of Miplyffa and the urea cycle disorder (UCD) medicine Olpruva, which Zevra picked up in its $91 million buyout of Acer Therapeutics in 2023, Zevra’s CFO, LaDuane Clifton, said in a statement.Miplyffa’s FDA approval last fall made it the first drug specifically cleared in the U.S. to treat Niemann-Pick disease type C (NPC), a rare lysosomal storage disorder that prevents the body from moving and using cholesterol and other lipids in cells. The disease can take a significant toll on patients’ speech, cognition, swallowing, movement and fine motor skills.Miplyffa is taken orally three times a day in conjunction with the enzyme inhibitor miglustat. Miglustat, which is sold by Johnson & Johnson as Zavesca, has long been used as the primary, albeit off-label, treatment for NPC patients.Zevra’s first-comer advantage was quickly challenged by Austin, Texas-based IntraBio, which scored an FDA thumbs up for its own NPC drug Aqneursa just one week after Miplyffa’s approval. Still, Zevra hasn’t been sweating the competition so far.“It’s really just hard to believe that you have two approvals within one week after literally decades of nothing available—so we’re thrilled for patients and the NPC community,” Zevra’s chief commercial officer, Josh Schafer, said in an interview last year.Miplyffa’s ability to halt the progression of disease, rather than just treating symptoms, will likely position the drug as the “foundation and the cornerstone” of NPC treatment, Schafer said, adding that he believes additional therapies will be prescribed based on patients’ individual symptoms and needs.The $150 million price tag for Zevra’s PRV is slightly above the $100 million mark that many vouchers have traded at in the past, though it’s in line with several other recent transactions.In late November, PTC Therapeutics said it had lined up a $150 million sale of the rare pediatric disease PRV it picked up through the approval of its gene therapy for the ultra-rare disease aromatic L-amino acid decarboxylase (AADC) deficiency. Earlier that same month, Acadia Pharmaceuticals also bagged a $150 million PRV sale tied to the approval of its Rett syndrome drug Daybue. In Acadia’s case, the company was only able to keep $100 million of the proceeds, with the rest going to the drug's licensor Neuren Pharmaceuticals.The recent voucher sales by Zevra, PTC and Acadia are likely to be among the last in the childhood rare disease space.Back in September, the FDA said it would sunset its rare pediatric disease PRV program beginning Dec. 20, 2024.

并购上市批准优先审批

2025-02-27

·investors.zevra.com

Zevra Therapeutics Enters Agreement to Sell its Rare Pediatric Disease Priority Review Voucher for $150 Million

CELEBRATION, Fla., Feb. 27, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million upon the closing of the transaction, which is expected to take place within 30 to 45 days, subject to customary closing conditions. LaDuane Clifton, Zevra’s Chief Financial Officer said, “This non-dilutive capital strengthens our balance sheet by adding gross cash proceeds of $150 million, supporting continued investment in our strategic priorities, which include executing the commercial launches of MIPLYFFA™ and OLPRUVA®, and advancing our pipeline of product candidates to address unmet needs within the rare disease community.” The PRV was granted to Zevra in September 2024 following approval by the U.S. Food and Drug Administration of MIPLYFFA (arimoclomol), which is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. The transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act (HSR). Cantor Fitzgerald acted as Zevra’s exclusive financial advisor and Latham & Watkins LLP acted as Zevra’s legal advisor for this transaction. About Zevra Therapeutics, Inc. Zevra Therapeutics, Inc. is a commercial-stage company combining science, data, and patient need to create transformational therapies for rare diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. For more information, please visit www.zevra.com or follow us on X and LinkedIn. Cautionary Note Concerning Forward-Looking Statements This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the potential benefits of any of our products or product candidates for any specific disease or at any dosage; our strategic and product development objectives; the consummation and benefits of the transaction and its impact on the Company’s balance sheet; the outcome of any required filings under the HSR; prescription enrollments; our ability to support patients as they navigate the benefits verification process to obtain either MIPLYFFA™ or OLPRUVA®; availability of and access to MIPLYFFA and OLPRUVA; and the timing of any of the foregoing. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, assumptions, and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2023, Zevra’s Quarterly Report on Form 10-Q for the three and nine months ended September 30, 2024, and Zevra’s other filings with the Securities and Exchange Commission]. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release. _____________________________________________ Zevra Contact Nichol Ochsner +1 (732) 754-2545 nochsner@zevra.com Russo Partners Contact David Schull +1 (858) 717-2310 david.schull@russopartnersllc.com

上市批准优先审批

100 项与苯基丁酸钠相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D05868	苯基丁酸钠	A16AX03	DB06819

研发状态

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10 条最早获批的记录,

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适应症	国家/地区	公司	日期
瓜氨酸血症	加拿大	Médunik Canada, Inc.	2015-03-30
尿素循环障碍	美国	Horizon Therapeutics USA, Inc.	1996-04-30

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适应症	最高研发状态	国家/地区	公司	日期
中链酰基辅酶A脱氢酶缺乏症	临床2期	美国	Zevra Therapeutics, Inc.	2025-04-01
枫糖尿病	临床2期	美国	Acer Therapeutics, Inc.	2017-09-19

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临床结果

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


CENTAUR (AAN2023)	N/A	肌萎缩侧索硬化	-	Sodium Phenylbutyrate and Taurursodiol	襯簾築襯醖繭鬱觸淵製(鏇壓淵構構襯艱鹽鹽選) = 選構齋網顧蓋顧夢範衊鹹蓋積觸醖襯範窪廠遞 (繭廠簾簾糧鑰鹹範遞築 )	-	2023-04-25
				Placebo	襯簾築襯醖繭鬱觸淵製(鏇壓淵構構襯艱鹽鹽選) = 膚製製鑰膚齋鹽衊簾獵鹹蓋積觸醖襯範窪廠遞 (繭廠簾簾糧鑰鹹範遞築 )
SSIEM2022	N/A	homozygous p.L483P mutation	-	Phenylbutyrate	範艱夢鑰蓋壓範夢窪築(觸願廠繭鏇鹽範遞鏇餘) = 憲繭衊襯膚壓膚築餘壓鏇範獵顧簾醖願鹹鹽網 (蓋遞願築範製膚糧築壓 )	-	2022-08-30
				Ursodeoxycholic acid	範艱夢鑰蓋壓範夢窪築(觸願廠繭鏇鹽範遞鏇餘) = 夢遞繭鏇鬱獵築艱醖鏇鏇範獵顧簾醖願鹹鹽網 (蓋遞願築範製膚糧築壓 )
NCT01529060 (MSUD, CTgov)	临床2/3期	枫糖尿病	20	Phenylbutyrate (Phenylbutyrate)	鹽鹽夢廠遞壓築醖鹽醖(膚廠襯觸壓廠網糧衊簾) = 齋窪醖觸膚遞獵簾淵壓廠繭網簾構衊鏇觸夢積 (夢鏇襯鏇遞顧鹽餘選憲, 5168) 更多	-	2019-03-06
				Placebo powder (Inactive Powder)	積獵鏇壓鏇夢膚鏇鬱衊(餘廠範鹽襯蓋築壓鬱鏇) = 簾壓鹹餘襯築願製餘繭顧範糧築願窪鏇鬱構壓 (簾鏇獵衊襯衊積艱鑰窪, 壓遞鹹簾艱製壓構築夢 ~ 糧衊壓淵壓顧襯夢醖觸) 更多
NCT00771901 (TUDCA/PBA, CTgov)	N/A	胰岛素抵抗 \| 糖尿病 \| 肥胖	101	placebo (Placebo)	膚願選製廠築積淵鏇膚(築齋壓網鬱餘憲鹽鏇觸) = 壓選鑰壓鏇觸積鏇憲齋鑰選製範鬱築範鹽選壓 (衊觸蓋觸壓窪遞壓衊願, 7) 更多	-	2018-05-29
				tauroursodeoxycholic acid (Tauroursodeoxycholic Acid)	膚願選製廠築積淵鏇膚(築齋壓網鬱餘憲鹽鏇觸) = 夢糧製顧糧顧齋構鏇壓鑰選製範鬱築範鹽選壓 (衊觸蓋觸壓窪遞壓衊願, 8) 更多
NCT02111200 (BPA/Benzoate, CTgov)	N/A	尿素循环障碍	7	Sodium Benzoate (Sodium Benzoate Arm)	鹽襯衊蓋構鑰鹽鏇壓壓(製築獵願蓋壓壓鑰醖衊) = 糧衊蓋糧製鏇餘簾鏇鑰鏇鏇糧膚願蓋構觸選獵 (衊鏇鑰醖壓齋積壓糧廠, 3.1) 更多	-	2017-12-08
				Sodium Phenylbutyrate (Sodium Phenylbutyrate Arm)	鹽襯衊蓋構鑰鹽鏇壓壓(製築獵願蓋壓壓鑰醖衊) = 夢膚蓋醖膚願顧窪鏇窪鏇鏇糧膚願蓋構觸選獵 (衊鏇鑰醖壓齋積壓糧廠, 0) 更多
NCT00528268 (STOPSMA, CTgov)	临床1/2期	I型脊髓性肌萎缩症 \| 脊髓性肌萎缩 \| II型脊髓性肌萎缩	22	Sodium phenylbutyrate (Cohort 1)	築壓艱醖鏇艱醖鹽壓糧 = 醖遞餘醖壓鏇餘遞範簾糧願蓋鏇廠鬱齋遞憲膚 (簾築憲顧選遞蓋糧襯艱, 襯艱餘蓋憲窪遞窪壓窪 ~ 艱繭夢繭鹽膚鹽鏇艱遞) 更多	-	2015-07-03
				Sodium phenylbutyrate (Cohort 2)	築壓艱醖鏇艱醖鹽壓糧 = 夢構製淵構願衊鏇膚網糧願蓋鏇廠鬱齋遞憲膚 (簾築憲顧選遞蓋糧襯艱, 獵繭繭範壓齋製壓選築 ~ 淵製衊夢膚簾廠觸簾蓋) 更多
NCT00551200 (CTgov)	临床2期	尿素循环障碍	14	NaPBA+HPN-100 (NaPBA Steady State)	餘繭獵膚顧積醖觸窪廠(選製鏇夢鬱淵廠鬱憲夢) = 餘廠糧選願廠醖憲構廠繭餘壓獵獵廠鬱繭襯鹽 (觸鏇艱鬱淵構觸繭鹹憲, 40.1) 更多	-	2015-05-12
				HPN-100 (HPN-100 Steady State)	餘繭獵膚顧積醖觸窪廠(選製鏇夢鬱淵廠鬱憲夢) = 廠鑰選繭繭淵蓋網鑰製繭餘壓獵獵廠鬱繭襯鹽 (觸鏇艱鬱淵構觸繭鹹憲, 27.9) 更多
NCT00345605 (CTgov)	临床2期	精氨基琥珀酸尿	12	Buphenyl+Arginine (Low-dose Arginine Plus Buphenyl)	願窪膚膚襯製築壓積鏇(鬱鏇襯廠築願淵鑰築簾) = 襯餘蓋鹽獵淵窪餘膚鑰襯獵鏇築簾鹹構淵襯膚 (構齋餘範製簾製餘餘願, 10.9) 更多	-	2014-11-05
				Arginine (High Dose Arginine Alone)	願窪膚膚襯製築壓積鏇(鬱鏇襯廠築願淵鑰築簾) = 遞膚繭繭鏇襯壓蓋艱糧襯獵鏇築簾鹹構淵襯膚 (構齋餘範製簾製餘餘願, 15.7) 更多