The goal of this study is to learn about how the hormone insulin controls blood sugar. The main question it aims to answer is about how much insulin the body actually needs to maintain a normal blood sugar level. People with obesity and high insulin levels will receive eight doses of diazoxide, a drug that suppresses the pancreas's production of insulin, and will have their fasting blood sugar and insulin levels checked daily while taking the drug.

开始日期2024-10-23

申办/合作机构

Columbia University

[+1]

NCT06308107 / Recruiting临床1期IIT

Safety and Feasibility of Hyperkalemic Cardioplegia With Diazoxide in Cardiac Surgery (CPG-DZX) Trial

This study aims to confirm the safety and efficacy of diazoxide as an additive to hyperkalemic cardioplegia in patients undergoing cardiac surgery with cardiopulmonary bypass. The investigators hypothesize that diazoxide combined with hyperkalemic cardioplegia provides superior myocardial protection and reduced myocardial stunning compared with standard cardioplegia alone. Thirty patients will receive treatment. Safety will be assessed by comparing mean arterial blood pressure measurements, glucose levels and incidence of adverse events between the two groups. Efficacy will be assessed by comparing right and left ventricular function in pre-operative vs post-operative transesophageal echocardiograms, need for mechanical circulatory support, ease of separation from bypass and Vasoactive Inotrope Score (VIS) between the two groups. The information gained could pave the way for the use of Katp (Potassium-atp) channel openers to prevent stunning, improve patient outcomes, and reduce health care costs related to myocardial stunning that requires inotropic and mechanical support following cardiac surgery.

开始日期2024-07-08

申办/合作机构

The Johns Hopkins University

TCTR20240731001 / Recruiting临床3期IIT

Scientific Title: Evaluation of the Clinical Efficacy and Safety of Dexmedetomidine Combined with Diazoxide for Anesthesia in Vaginal Ultrasound-Guided Egg Retrieval in IVF-ET Procedures

开始日期2024-07-01

申办/合作机构-

100 项与二氮嗪相关的临床结果

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100 项与二氮嗪相关的转化医学

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100 项与二氮嗪相关的专利（医药）

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3,657

项与二氮嗪相关的文献（医药）

2024-11-01·NUCLEAR MEDICINE AND BIOLOGY

Longitudinal multimodal monitoring of transplanted islet β-cells

Article

作者: Bowden, Gregory ; Pichler, Bernd J ; Schmid, Andreas M. ; Küppers, Astrid ; Maurer, Andreas ; Eter, Wael ; Nischwitz, Volker ; Michelotti, Filippo C ; Pichler, Bernd J. ; Schmid, Andreas M ; Michelotti, Filippo C. ; Gotthardt, Martin

PURPOSE:

Monitoring β-cell mass and function would provide a better understanding of diabetes, setting the stage for truly individualized therapies. We applied a combined PET/MRI protocol to monitor engrafted islets mass and function without pre-labeling of isolated cells. A PET tracer binding to GLP-1R quantifies β-cell mass, while Mn-CA characterizes β-cell function. Both parameters were assessed in transplanted and native β-cells in vivo and validated with autoradiography and mass spectrometry imaging.

METHODS:

Islets were collected and transplanted into the calves of C3H-mice. Accumulation of [⁶⁴Cu]Ex4 and Mn-CA was examined with a PET/MRI at 1 h post-injection between 1 and 4 weeks after the transplantation. A separate blocking study with diazoxide targeted the functionality of the transplanted islets. As validation, ex vivo autoradiography and LA-ICP-MS imaging were performed after the last imaging session.

RESULTS:

PET/MRI monitored the engraftment of transplanted islets and visualized an increasing uptake of the PET tracer and Mn-CA. The Mn-CA accumulated at a higher islet-to-background ratio in the calf of mice than in the pancreas due to the high retention of Mn-CA in the exocrine pancreas. In vivo imaging data correlated well with autoradiography and LA-ICP-MS imaging, validating the in vivo approaches.

CONCLUSION:

For the quantification of β-cell function, Mn-based contrast mechanisms between native and transplanted islets differ and require further studies for optimal biological readout. However, non-invasive PET/MRI nonetheless provides the tools to investigate the relationship between β-cell mass and function in pancreatic islets.

2024-10-01·American journal of perinatology

Variability in Diagnosis and Management of Hypoglycemia in Neonatal Intensive Care Unit

Article

作者: Dinu, Daniela ; Rozance, Paul J. ; Hagan, Joseph L.

Objective Hypoglycemia, the most common metabolic derangement in the newborn period remains a contentious issue, not only due to various numerical definitions, but also due to limited therapeutical options which either lack evidence to support their efficacy or are increasingly recognized to lead to adverse reactions in this population. This study aimed to investigate neonatologists' current attitudes in diagnosing and managing transient and persistent hypoglycemia in newborns admitted to the Neonatal Intensive Care Unit (NICU). Methods A web-based electronic survey which included 34 questions and a clinical vignette was sent to U.S. neonatologists. Results There were 246 survey responses with most respondents using local protocols to manage this condition. The median glucose value used as the numerical definition of hypoglycemia in first 48 hours of life (HOL) for symptomatic and asymptomatic term infants and preterm infants was 45 mg/dL (2.5 mmol/L; 25–60 mg/dL; 1.4–3.3 mmol/L), while after 48 HOL the median value was 50 mg/dL (2.8 mmol/L; 30–70 mg/dL; 1.7–3.9 mmol/L). There were various approaches used to manage transient and persistent hypoglycemia that included dextrose gel, increasing caloric content of the feeds using milk fortifiers, using continuous feedings, formula or complex carbohydrates, and use of various medications such as diazoxide, glucocorticoids, and glucagon. Conclusion There is still large variability in current practices related to hypoglycemia. Further research is needed not only to provide evidence to support the values used as a numerical definition for hypoglycemia, but also on the efficacy of current strategies used to manage this condition. Key Points

2024-10-01·Diabetology International

Hypovascular insulinoma with reduced microvessel density on histopathology: a case report

Article

作者: Murakami, Takaaki ; Kuroda, Takahiro ; Mizuno, Masami ; Takahashi, Yoshihiro ; Kanayama, Tomohiro ; Hirota, Takuo ; Kato, Takehiro ; Miyazaki, Tatsuhiko ; Liu, Yanyan ; Yabe, Daisuke ; Imai, Risako ; Ozeki, Sayako ; Kubota, Sodai ; Horikawa, Yukio ; Sakai, Mayu ; Takao, Ken

Pancreatic neuroendocrine tumors (PanNETs) are generally hypervascular and readily detectable on imaging tests. However, hypovascular PanNETs are clinically problematic, requiring multiple imaging tests and tissue analyses to differentiate them from pancreatic ductal cancers. A 41 year-old man presented with Whipple's triad; 72 h fasting test followed by glucagon challenge test suggested insulinoma. However, contrast-enhanced computed tomography image showed a 17 mm tumor with poor enhancement and unclear borders in the tail of the pancreas. Abdominal magnetic resonance imaging and contrast-enhanced endoscopic ultrasonography (EUS) indicated cystic degeneration and necrosis at the same site; EUS-guided fine-needle aspiration cytology indicated a PanNET Grade 1 tumor. Although the imaging was inconclusive, diazoxide treatment ameliorated the hypoglycemia-related symptoms and insulinoma was deemed likely; following tail pancreatectomy and splenectomy, the symptoms disappeared. Pathological examination revealed a tumor positive for insulin and classed as PanNET Grade 1 according to the 2019 WHO classification. The microvessel density (MVD) of the tumor was found to be as low as 3.9%, which may partly account for the inconclusive images. The present case was difficult to diagnose preoperatively due to hypovascularity on imaging because of reduced MVD. It is clinically important to evaluate MVD in cases of hypovascular PanNETs by multiple preoperative imaging studies to differentiate them from pancreatic cancers and to validate the findings by postoperative pathological analysis.

项与二氮嗪相关的新闻（医药）

2024-12-01

·药明康德

12月7款创新药有望获FDA批准

▎药明康德内容团队编辑根据PDUFA的预期目标日期，预计12月，美国FDA将对7款创新药物的批准做出监管决定，本文将对这些疗法进行相关介绍。 ▲12月有望在美国获批的新药（药明康德内容团队制图，点击可见大图）活性成分：Garadacimab 适应症：遗传性血管水肿（HAE）公司名称：CSL Garadacimab是一种新型FXIIa抑制性单克隆抗体（FXIIa mAb），被设计以每月一次皮下注射的方式用于HAE相关发作的预防性治疗。当FXII被激活时，它会启动导致水肿形成的级联反应。与靶向下游介质的其他HAE治疗相比，garadacimab能够独特地抑制血浆蛋白FXIIa，将HAE的级联反应从源头进行抑制。FDA和欧洲药品管理局（EMA）均已授予garadacimab孤儿药资格。CSL还在研究garadacimab用于HAE以外的其他适应症，包括特发性肺纤维化——FXIIa抑制可能在改善此类患者的临床结局方面发挥重要作用。根据近期发布的3期临床研究VANGUARD的数据，与安慰剂组相比，每月使用一次garadacimab的患者的HAE月发作率在统计学上较低——平均发作率降低86.5%，中位发作率降低100%，调整基线发作率后的平均发作率降低89.2%。总体而言，在为期6个月的试验中，大多数（61.5%）使用garadacimab的患者无发作，而安慰剂组的患者均有发作；与预处理阶段相比，74.4%使用garadacimab的患者发作减少≥90%。除此之外，该研究还表明，garadacimab具有良好的安全性和耐受性特征，未发生不良事件导致的治疗中止。活性成分：Olezarsen 适应症：家族性乳糜微粒血症综合征（FCS）公司名称：Ionis Pharmaceuticals Olezarsen是一种在研反义寡核苷酸疗法，旨在抑制机体产生载脂蛋白C-III（apoC-III）——这是一种在肝脏中产生的、可调节血液中甘油三酯（TG）代谢的蛋白。美国FDA于2023年第1季度授予了olezarsen治疗FCS的快速通道资格，并于2024年2月授予该疗法孤儿药资格和突破性疗法认定（BTD）。FDA预计在2024年12月19日前完成审评。临床3期临床试验结果显示，在接受治疗6个月时，经安慰剂调整后的apoC-III水平降幅为74%，而在12个月时，这一降幅增加至81%。与基线相比，接受olezarsen治疗6个月的患者，其TG水平（经安慰剂调整）显著降低，降幅为44%（p<0.001）。接受治疗第6个月到第12个月期间，olezarsen可持续降低TG水平，经安慰剂调整的TG水平降低了59%。此外，与安慰剂组相比，接受olezarsen治疗的患者在整个12个月的研究期间急性胰腺炎（AP）的发生率显著降低，使用olezarsen后距首次AP发作的时间大幅延长。经安慰剂调整后，接受olezarsen治疗的患者自基线至12个月期间的全因住院率降低了84%。在安全性方面，olezarsen在研究中显示出良好的安全性和耐受性。活性成分：Glepaglutide 适应症：短肠综合征（SBS）公司名称：Zealand Pharma Glepaglutide是一种长效GLP-2类似物，正在开发作为SBS的潜在治疗选择。正在开发的glepaglutide是一种装于自动注射器中的液体产品，设计用于每周两次的皮下给药，旨在减少或消除SBS患者对肠外支持的需求。美国FDA已授予该疗法治疗SBS的孤儿药资格。根据3期临床试验EASE-1的结果，该研究在106例每周至少3天依赖肠外营养支持（PS）的SBS肠衰竭患者中，评估了皮下给药10 mg glepaglutide与安慰剂相比的安全性和疗效。分析显示，与安慰剂相比，每周两次glepaglutide给药在第24周显著降低患者每周PS所需总液体体积（P=0.0039）。具体来说，第24周时，每周两次接受glepaglutide治疗患者的PS液体体积较基线平均降低5.13公升/周。安慰剂治疗仅导致所需PS液体体积降低2.85公升/周。对于接受每周两次glepaglutide治疗的患者，14%的患者（n=5）实现不再需要PS辅助治疗，而安慰剂治疗患者均不能停用PS。在安全性方面，glepaglutide显示具良好安全性与耐受性。在试验中最常报告的不良事件为注射部位反应和胃肠道事件。活性成分：Diazoxide choline（DCCR）适应症：Prader-Willi综合征（PWS）公司名称：Soleno Therapeutics DCCR是一种含有二氮嗪胆碱（diazoxide choline）的创新专有缓释剂型，每日口服一次。DCCR开发项目的数据支持来自五项在健康志愿者中完成的1期临床研究和3项完成的2期临床研究，其中一项是在PWS患者中进行的。在PWS的3期临床开发项目中，DCCR在解决PWS的标志性症状暴食症以及其他几种症状（如攻击性/破坏性行为、脂肪质量和其他代谢参数）方面显示出可喜的效果。目前，DCCR已在美国和欧盟获得治疗PWS的孤儿药资格，并且在美国获得快速通道资格认定。今年4月，美国FDA授予DCCR突破性疗法认定，用于治疗4岁及以上经基因检测确认的PWS患者的高食欲症。这一认定是基于对3期临床试验初步数据的评估，FDA认为DCCR可能在具有临床意义的终点上与现有疗法相比，显示出显著改善。活性成分：Cosibelimab 适应症：皮肤鳞状细胞癌（cSCC）公司名称：Checkpoint Therapeutics Cosibelimab是一种完全人源化、具有高亲和力的单克隆抗体，可直接与PD-L1结合，从而阻断其与PD-1以及B7.1受体的相互作用，进而除去PD-L1对CD8阳性T细胞的抑制效果，恢复其毒杀性T细胞反应。新闻稿指出，cosibelimab与目前上市的PD-1和PD-L1抗体的潜在差异在于它具有持续的、超过99%的目标肿瘤占位率（tumor occupancy），可以重新激活抗肿瘤免疫反应，此外它还具有能够诱导抗体依赖性细胞介导的细胞毒性（ADCC）的功能性Fc结构域，从而在某些肿瘤类型中发挥更为强大的疗效。根据此前发布的针对局部晚期和转移性cSCC的关键性研究的长期数据，随着时间的推移，接受cosibelimab治疗的患者的病情缓解程度加深，完全缓解率大幅高于先前的报道。此外，随着时间的推移，缓解持续稳定，晚期cSCC组和转移性cSCC组患者的中位缓解持续时间尚未达到。此外，该疗法表现出良好的安全性和耐受性，只有2%的患者发生了严重的免疫相关不良事件（irAE），不到1%的患者因任何程度的irAE而中断治疗，这两个比例都大幅低于目前批准的免疫疗法。活性成分：Ensartinib 适应症：ALK阳性非小细胞肺癌（NSCLC）公司名称：Xcovery Holdings Ensartinib是一款新型强效、高选择性的新一代ALK抑制剂，由贝达药业及其控股子公司Xcovery Holdings共同开发。在体外试验中，这款抑制剂可以强效抑制ALK的多种突变体。在1/2期临床试验中，ensartinib已经显示出对ALK阳性NSCLC患者的积极临床活性。 Ensartinib的新药申请（NDA）递交是基于eXalt3研究的成果。eXalt3是一项全球性随机3期临床研究，旨在评估ensartinib与活性对照药物在一线治疗ALK阳性NSCLC患者中的疗效和安全性。根据2021年9月发表在JAMA Oncology的研究结果，ensartinib在治疗ALK阳性NSCLC患者全身性和颅内疾病方面均显示出优于活性对照药物的疗效。活性成分：Crinecerfont 适应症：先天性肾上腺皮质增生症（CAH）公司名称：Neurocrine Biosciences Crinecerfont是一种在研口服、选择性促肾上腺皮质激素释放因子1型受体（CRF1）拮抗剂，通过激素非依赖性机制减少和控制过量的肾上腺雄激素，用于治疗因21-羟化酶缺乏症导致的先天性肾上腺增生。研究显示，拮抗脑垂体中的CRF1可降低促肾上腺皮质激素水平，进而减少肾上腺雄激素的生成，并可能减少与经典CAH相关的症状。根据去年公布的3期CAHtalyst儿科研究数据，在糖皮质激素（GC）稳定后，与安慰剂相比，crinecerfont治疗使得患者在第4周时的血清雄烯二酮较基线统计学显著降低（p=0.0002）。此结果与3期CAHtalyst成人研究的结果一致，表明crinecerfont在不同年龄段的患者中均显示出疗效。在第28周时，与安慰剂相比，crinecerfont治疗使得患者在维持雄激素控制的同时，其每日所需糖皮质激素剂量百分比的降低具统计学显著性（p<0.0001）。约30%接受crinecerfont治疗的受试者在维持雄激素控制的同时达到了生理GC剂量的降低，而接受安慰剂治疗的受试者为0%。此外，crinecerfont还在研究中展现出了良好的安全性与耐受性。 ▲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] CSL's Phase 3 Study Shows First-In-Class Garadacimab Provides Patients with Significant HAE Attack Prevention with Monthly Dosing. Retrieved November 25, 2024, from https://www.prnewswire.com/news-releases/csls-phase-3-study-shows-first-in-class-garadacimab-provides-patients-with-significant-hae-attack-prevention-with-monthly-dosing-301756102.html?tc=eml_cleartime [2] CSL's Garadacimab, a First-in-Class Factor XIIa Inhibitor, Receives FDA and EMA Filing Acceptance，Retrieved November 25, 2024, from https://newsroom.csl.com/2023-12-14-CSLs-Garadacimab,-a-First-in-Class-Factor-XIIa-Inhibitor,-Receives-FDA-and-EMA-Filing-Acceptance [3] Ionis announces olezarsen FCS New Drug Application accepted for Priority Review and enrollment in Phase 3 sHTG program completed. Retrieved November 25, 2024, from https://www.prnewswire.com/news-releases/ionis-announces-olezarsen-fcs-new-drug-application-accepted-for-priority-review-and-enrollment-in-phase-3-shtg-program-completed-302181549.html [4] Zealand Pharma submits New Drug Application to the US FDA for glepaglutide in short bowel syndrome. Retrieved November 25, 2024, from https://www.globenewswire.com/en/news-release/2023/12/22/2800463/0/en/Zealand-Pharma-submits-New-Drug-Application-to-the-US-FDA-for-glepaglutide-in-short-bowel-syndrome.html [5] Soleno Therapeutics Announces Submission of New Drug Application to the U.S. FDA for DCCR (Diazoxide Choline) Extended-Release Tablets for the Treatment of Prader-Willi Syndrome. Retrieved November 25, 2024, from https://www.globenewswire.com/news-release/2024/06/28/2905801/0/en/Soleno-Therapeutics-Announces-Submission-of-New-Drug-Application-to-the-U-S-FDA-for-DCCR-Diazoxide-Choline-Extended-Release-Tablets-for-the-Treatment-of-Prader-Willi-Syndrome.html [6] Checkpoint Therapeutics Announces Biologics License Application Resubmission for Cosibelimab. Retrieved November 25, 2024, from https://ir.checkpointtx.com/news-events/press-releases/detail/119/checkpoint-therapeutics-announces-biologics-license [7] U.S. Food and Drug Administration (FDA) Has Accepted the New Drug Application (NDA) for Ensartinib. Retrieved November 25, 2024, from https://www.businesswire.com/news/home/20240313675039/en/U.S.-Food-and-Drug-Administration-FDA-Has-Accepted-the-New-Drug-Application-NDA-for-Ensartinib [8] Neurocrine Biosciences Announces U.S. FDA Accepts New Drug Applications and Grants Priority Review for Crinecerfont for Pediatric and Adult Patients with CAH. Retrieved November 25, 2024, from https://www.prnewswire.com/news-releases/neurocrine-biosciences-announces-us-fda-accepts-new-drug-applications-and-grants-priority-review-for-crinecerfont-for-pediatric-and-adult-patients-with-cah-302186641.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

孤儿药临床3期突破性疗法临床结果寡核苷酸

2024-11-27

·Pharmaceutical Technology

FDA delays decision of Soleno’s Prader-Willi syndrome drug

The extension allows the FDA time to complete its review. Credit: izzuanroslan via Shutterstock. The US Food and Drug Administration (FDA) has delayed its decision on Soleno Therapeutics investigational therapy for children with Prader-Willi syndrome. The new decision date for Soleno’s DCCR (diazoxide choline) extended-release tablets is set for 27 March 2025 – three months longer than the previous FDA extended review period. The delay comes after the FDA classified recent responses from Soleno as a “major amendment” to its new drug application (NDA), requiring additional time to review. However, the agency has not raised concerns about the drug’s safety, efficacy, or manufacturing. Prader-Willi syndrome is a rare genetic disorder caused by abnormalities in chromosome 15. Symptoms include low muscle tone, developmental delays, and behavioural challenges. Hyperphagia, or extreme hunger, is one of the most serious and defining characteristics of the disorder and can lead to severe obesity and associated health complications if unmanaged. There are currently no FDA-approved treatments specifically addressing hyperphagia in Prader-Willi syndrome. The US-based biotech’s NDA is supported by data from the Phase III DESTINY-PWS trial (NCT03440814), which enrolled 127 individuals with genetically confirmed Prader-Willi syndrome. The trial did not demonstrate a statistically significant difference in hyperphagia reduction overall, but Soleno said that the DCCR group showed “nominally significant” reductions in fat mass, and overall improvements in condition as assessed by investigators. DCCR has been granted breakthrough therapy, fast track, and orphan drug designations in the US, as well as orphan drug designation in the EU. Originally slated for discussion at an FDA advisory committee (AdComm) meeting, the agency decided in October 2024 that such a meeting was unnecessary. DCCR is an extended-release tablet containing a crystalline salt formulation of diazoxide, an activator of the adenosine triphosphate (ATP)-sensitive potassium (KATP) channel. These channels are present in a variety of cells, including pancreatic β cells, and adipocytes. The regulation of these cells can help manage hyperphagia. The delay represents a setback for Soleno on its first potential approved treatment. Soleno’s stock price decreased by 6.9% from $57.22 at close on 25 November to a low of $53.25 on 26 November. According to GlobalData’s Pharma Intelligence Center, DCCR could generate up to $759m in sales by 2030. GlobalData is the parent company of Pharmaceutical Technology. The company is also evaluating DCCR as a treatment for paediatric patients with certain genetic obesities, including SH2B1 deficiency, obesity associated with PCSK1 mutation (rs6232 variant), and SIM1 deficiency. A Phase II trial (NCT05532020) is set to enrol approximately 30 patients aged five years or younger suffering from either of the three genetic obesity disorders.

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2024-11-26

·GlobeNewswire-Health Care

Soleno Therapeutics Announces FDA Extension of Review Period for DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome

PDUFA target action date extended by three months to March 27, 2025REDWOOD CITY, Calif., Nov. 26, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia. The new Prescription Drug User Fee Act (PDUFA) target action date is March 27, 2025. The FDA determined that responses to recent information requests constituted a major amendment to the NDA, resulting in the extension of the PDUFA goal date by three months. The extension allows the FDA time to complete their review, including that of the recently submitted information. The FDA did not cite any safety, efficacy or manufacturing concerns in their correspondence. Soleno submitted the NDA to the FDA on June 27, 2024. The FDA accepted the NDA and granted Priority Review in August 2024. Diazoxide choline has been granted Breakthrough and Fast Track Designations in the U.S., as well as Orphan Drug Designation in the U.S. and E.U. for the treatment of patients with PWS. About PWSThe Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births. The hallmark symptom of this disorder is hyperphagia, a chronic and life-threatening condition characterized by feelings of intense, persistent hunger, food pre-occupation, and an extreme drive to seek and consume food, which can severely diminish the quality of life for individuals with PWS and their families. Additional characteristics of PWS include behavioral problems, cognitive disabilities, low muscle tone, short stature (when not treated with growth hormone), the accumulation of excess body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to significant mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior) and longer term, co-morbidities such as diabetes, obesity, and cardiovascular disease. In a global survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents (parents and caregivers) rated hyperphagia and 92.9% rated body composition as either the most important or a very important symptom to be relieved by a new medicine. There are currently no approved therapies to treat the hyperphagia/appetite, metabolic, cognitive function, or behavioral aspects of the disorder. About DCCR (Diazoxide Choline) Extended-Release TabletsDCCR is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide and is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of individuals in a few rare diseases in neonates, infants, children and adults, but is not approved for use in PWS. Soleno conceived of and established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline and DCCR in individuals with PWS. The DCCR development program is supported by data from five completed Phase 1 clinical studies in healthy volunteers and three completed Phase 2 clinical studies, one of which was in individuals with PWS. In the PWS Phase 3 clinical development program, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and other metabolic parameters. About Soleno Therapeutics, Inc.Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. An NDA for its lead candidate, DCCR (diazoxide choline) extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi syndrome (PWS) is currently under review by the FDA and was granted Priority Review. For more information, please visit www.soleno.life. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the timing of any regulatory process or ultimate approvals and determining a path forward for DCCR for the treatment of PWS. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including the risks and uncertainties associated with the FDA’s review of our NDA, market conditions, as well as risks and uncertainties inherent in Soleno’s business, including those described in the company's prior press releases and in the periodic reports it files with the SEC. The events and circumstances reflected in the company's forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, the company does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Corporate Contact:Brian RitchieLifeSci Advisors, LLC212-915-2578

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KEGG	Wiki	ATC	Drug Bank
D00294	二氮嗪	V03AH01 C02DA01	DB01119

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适应症	国家/地区	公司	日期
高血压	美国	Schering Corp.	1973-01-22
低血糖	美国	Schering Corp.	1973-01-22

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适应症	最高研发状态	国家/地区	公司	日期
多发性硬化症	临床2期	西班牙	Neurotec Pharma SL	-
肌萎缩侧索硬化	临床前	西班牙	Neurotec Pharma SL	-

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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


ESPE2022	N/A	血色素沉着症	99	Diazoxide 2mg/kg/day	鑰築糧壓壓網窪襯築夢(構壓廠鬱鹹遞衊選築製) = Adverse events can still be observed with DZX low doses, but at a lower incidence compared to the STD 選廠鑰積獵網膚糧窪製 (醖蓋繭積構積簾鏇獵齋 ) 更多	积极	2022-09-15
				Diazoxide 3mg/kg/day
ESPE2022	N/A	先天性高胰岛素血症 HADH mutation	-	Diazoxide	鑰築餘憲鏇願構衊襯觸(襯膚鹹築鬱製廠憲繭鬱) = 鑰憲襯鏇餘壓襯鬱鹹憲淵積鹹淵繭獵鹽願衊網 (願鹹憲餘憲繭選積淵製, 0.9 ~ 7.1) 更多	-	2022-09-15
				Diazoxide	製艱壓襯壓鏇鏇範築網(遞繭繭築觸襯夢選鬱窪) = 憲廠衊壓願顧範製網繭襯遞齋顧獵範構構鏇夢 (獵壓網窪選構醖鹹醖鬱 )
ESPE2019	N/A	先天性高胰岛素血症 ABCC8 \| KCNJ11	-	Diazoxide	艱餘餘顧鬱憲鑰構淵製(膚觸鏇鏇範鑰獵願繭衊) = due to vomiting on oral feeds 艱構窪願顧構築簾獵窪 (構艱簾製襯鏇遞顧簾顧 ) 更多	-	2019-09-19
ESPE2019	N/A	先天性高胰岛素血症	-	Diazoxide	鑰獵鏇醖顧齋遞願鹽製(鏇糧獵餘鏇願艱繭範鬱) = 窪齋窪醖顧鏇鹹糧網簾廠鏇願簾壓餘構壓範廠 (膚鏇製網範鹹鑰簾繭簾 )	-	2019-09-19
				Octreotide	鑰獵鏇醖顧齋遞願鹽製(鏇糧獵餘鏇願艱繭範鬱) = 衊範製選襯壓鑰範餘積廠鏇願簾壓餘構壓範廠 (膚鏇製網範鹹鑰簾繭簾 )
NCT00631033 (Pubmed \| J Clin Endocrinol Metab)	临床2期	肥胖	51	DZX + placebo	願夢網蓋蓋憲繭範鑰鑰(襯憲顧憲淵醖夢網鬱餘) = 網觸夢艱淵構餘簾網窪選鬱鹽鑰鑰廠鹹夢觸襯 (顧選鏇鹹鬱膚簾鑰鬱蓋 ) 更多	积极	2018-11-01
				DZX + metformin	願夢網蓋蓋憲繭範鑰鑰(襯憲顧憲淵醖夢網鬱餘) = 餘憲鬱選膚繭獵餘顧餘選鬱鹽鑰鑰廠鹹夢觸襯 (顧選鏇鹹鬱膚簾鑰鬱蓋 ) 更多
ESPE2018	N/A	先天性高胰岛素血症	-	Diazoxide	範觸顧遞廠醖鏇鑰蓋觸(鹹範構廠艱膚鏇糧遞製) = Our patient is homozygote for this mutation and both his parents are heterozygote, confirming the diagnosis of autosomal recessive congenital hyperinsulinism. Now aged 5 years, the patient shows good glycaemic control on octreotide in combination with frequent feeding. However, he has developmental delay with epilepsy, requiring multiple anti-convulsivant drugs. 衊鬱蓋糧築顧齋範膚願 (顧壓蓋鏇鏇願衊襯觸築 )	-	2018-09-27
NCT02049385 (CTgov)	临床1/2期	重度抑郁症	10	Diazoxide (Diazoxide)	鏇鹹築膚憲鹽構觸齋襯(範觸鑰網鑰醖鹹襯鬱願) = 築顧鬱願膚簾艱艱積範構憲構襯醖獵壓範願鹹 (鏇鹹遞鏇鏇衊鹽夢製繭, 積餘壓構淵顧觸齋製網 ~ 鑰鹽廠鬱淵艱製鑰簾範) 更多	-	2017-10-13
				Placebo (Placebo)	鏇鹹築膚憲鹽構觸齋襯(範觸鑰網鑰醖鹹襯鬱願) = 範淵觸壓淵製襯網選窪構憲構襯醖獵壓範願鹹 (鏇鹹遞鏇鏇衊鹽夢製繭, 鹹齋蓋衊製築醖願膚鏇 ~ 夢夢鹽製構淵艱餘遞選) 更多
ESPE2016	N/A	胎儿生长受限 \| 先天性高胰岛素血症 ABCC8 \| KCNJ11 \| GLUD1 ... 更多	-	Fetal diazoxide exposure	製繭壓壓築願觸顧鬱築(衊糧衊淵鏇齋膚範鹹獵) = 壓鬱鹹鏇餘鏇簾範窪構鑰製襯壓艱蓋鏇糧蓋窪 (築壓簾窪鬱網艱夢膚觸 )	-	2016-09-10
NCT00683774 (CTgov)	N/A	多囊卵巢综合征	21	diazoxide (PCOS Subjects)	廠夢顧淵網窪衊遞遞壓(衊醖遞鑰蓋衊願築廠鏇) = 艱積願遞憲淵窪簾醖築鹹構鑰築淵範醖鹽鹽簾 (襯衊築獵膚築壓範壓獵, 鏇淵壓觸襯獵鏇餘簾網 ~ 餘窪築餘鏇獵網蓋夢夢) 更多	-	2014-08-21
				diazoxide (Normal Subjects)	廠夢顧淵網窪衊遞遞壓(衊醖遞鑰蓋衊願築廠鏇) = 齋蓋積夢鹽襯壓顧積範鹹構鑰築淵範醖鹽鹽簾 (襯衊築獵膚築壓範壓獵, 鏇鑰鹽膚構積艱窪築壓 ~ 遞膚簾壓餘衊壓鑰鹹壓) 更多
SFN2013	N/A	神经变性	-	Diazoxide	窪衊憲鹽顧選餘獵觸獵(齋鏇醖鑰窪網糧膚築願) = 構鑰網衊製獵製餘憲壓積範廠窪構夢鏇簾醖選 (遞鹽願製製憲製廠衊觸 ) 更多	-	2013-11-10