The purpose of this is study is to evaluate the long-term safety of DCCR (diazoxide choline) extended-release tablets) in patients with Prader-Willi syndrome.

开始日期2023-01-31

申办/合作机构

Soleno Therapeutics, Inc.

NCT05532020

/ Unknown status临床2期

An Open-Label Study to Assess the Preliminary Efficacy and Safety of DCCR (Diazoxide Choline) Extended-Release Tablets in Patients With Genetic Obesities

This is a phase 2 open-label, one-arm, clinical study to evaluate the efficacy and safety of DCCR (diazoxide choline) extended-release tablets over 1 year of treatment, in patients with genetic obesities.

开始日期2023-01-01

申办/合作机构

Soleno Therapeutics, Inc.

NCT04086810

/ Withdrawn临床3期

An Open-Label Study of Diazoxide Choline Controlled-Release Tablet in Patients With Prader-Willi Syndrome

The primary objective of this study is to monitor the long-term safety of DCCR in PWS patients.

开始日期2019-10-01

申办/合作机构

Soleno Therapeutics, Inc.

100 项与二氮嗪胆碱相关的临床结果

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100 项与二氮嗪胆碱相关的转化医学

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100 项与二氮嗪胆碱相关的专利（医药）

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1,362

项与二氮嗪胆碱相关的文献（医药）

2025-05-01·Cell Reports

Beta cells intrinsically sense and limit their secretory activity via mTORC1-RhoA signaling

Article

作者: Walker, Michael D ; Gover, Ofer ; Hamburg, Amit ; Krell, Saar ; Sharabi, Kfir ; Leibowitz, Gil ; Helman, Aharon ; Molakandov, Kfir

Precise regulation of insulin secretion by pancreatic β cells is essential to prevent excessive insulin release. Here, we show that the nutrient sensor mechanistic Target of Rapamycin Complex 1 (mTORC1) is rapidly activated by glucose in β cells via the insulin secretion machinery, positioning mTORC1 as a sensor of β cell activity. Acute pharmacological inhibition of mTORC1 during glucose stimulation enhances insulin release, suggesting that mTORC1 acts as an intrinsic feedback regulator that restrains insulin secretion. Phosphoproteomic profiling reveals that mTORC1 modulates the phosphorylation of proteins involved in actin remodeling and vesicle trafficking, with a prominent role in the RhoA-GTPase pathway. Mechanistically, mTORC1 promotes RhoA activation and F-actin polymerization, limiting vesicle movement and dampening the second phase of insulin secretion. These findings identify a glucose-mTORC1-RhoA signaling axis that forms an autonomous feedback loop to constrain insulin exocytosis, providing insight into how β cells prevent excessive insulin release and maintain metabolic balance.

2025-01-01·Journal of Medical Toxicology

Diazoxide-Associated Hyperglycemia: A Critical Case Precipitating Hyperosmolar Hyperglycemic State in a Child

Article

作者: Schultz, Brian ; Lawson, Jennifer A

INTRODUCTION:

Diazoxide is the first-line treatment for children with hyperinsulinemic hypoglycemia (HI). In these cases, diazoxide raises blood glucose levels by suppressing insulin release, preventing hypoglycemia, and potentially devastating end-organ sequelae. Hyperosmolar hyperglycemic state (HHS) is an exceedingly rare side effect of diazoxide. This complication has been described in neonates and in adults, but few children.

CASE REPORT:

An 8-year-old female with genetic duplication of glucokinase, and consequent hyperinsulinemia, presented to the emergency department with evidence of hypovolemic shock secondary to severe dehydration with signs of encephalopathy. Point-of-care glucose was > 600 mg/dL. Additional labs were consistent with HHS complicated by acute kidney injury, sodium 106 mEq/L, potassium 2.5 mEq/L, chloride < 60 mEq/L, carbon dioxide 20 mEq/L, glucose 2105 mg/dL, BUN 107 mg/dL, and creatinine 3.99 mg/dL. The patient received aggressive fluid resuscitation and vasopressor support, and was admitted to the pediatric intensive care unit. A diazoxide level was obtained during admission revealing serum concentration previously shown to be associated with hyperglycemia.

DISCUSSION:

We posit the patient was predisposed to hyperglycemia based on elevated diazoxide serum concentration. We hypothesize severe dehydration led to renal impairment, which decreased diazoxide clearance, causing worsening hyperglycemia and ultimately, HHS. The differential diagnosis also included diabetic ketoacidosis, surreptitious administration of diazoxide, spontaneous resolution of genetic condition, and malabsorption or excretory crisis but none of these adequately explained the patient's presentation. Regardless, this case highlights the potentially lethal complication of HHS as a side effect of diazoxide therapy.

2024-12-24·PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Growth hormone receptor in VGLUT2 or Sim1 cells regulates glycemia and insulin sensitivity

Article

作者: Frazao, Renata ; Tavares, Mariana R. ; Alves, Guilherme A. ; List, Edward O. ; Cruz, Alessandra G. ; Donato, Jose ; Amaral, Andressa G. ; dos Santos, Jessica D. M. ; Kopchick, John J. ; dos Santos, Willian O. ; Camporez, João Paulo

Growth hormone (GH) has several metabolic effects, including a profound impact on glucose homeostasis. For example, GH oversecretion induces insulin resistance and increases the risk of developing diabetes mellitus. Here, we show that GH receptor (GHR) ablation in vesicular glutamate transporter 2 (VGLUT2)-expressing cells, which comprise a subgroup of glutamatergic neurons, led to a slight decrease in lean body mass without inducing changes in body adiposity. VGLUT2 ∆GHR mice exhibited reduced glycemia and improved glucose tolerance and insulin sensitivity. Among different glutamatergic neuronal populations, we found that GHR inactivation in Sim1-expressing cells recapitulated the phenotype observed in VGLUT2 ∆GHR mice. Furthermore, Sim1 ∆GHR mice exhibited reduced endogenous glucose production and improved hepatic insulin sensitivity without alterations in whole-body or muscle glucose uptake. Sim1 ∆GHR mice were protected against acute but not chronic diabetogenic effects of exogenous GH administration. Pharmacological activation of ATP-sensitive potassium channels in the brain normalized blood glucose levels in Sim1 ∆GHR mice. In conclusion, the absence of GHR signaling in VGLUT2/Sim1-expressing cells causes a persistent reduction in glycemia and improves hepatic insulin sensitivity. Central glucose-sensing mechanisms are likely involved in the reduced glycemia exhibited by Sim1 ∆GHR mice. The current findings uncover a mechanism involved in the effects of GHR signaling in regulating glucose homeostasis.

114

项与二氮嗪胆碱相关的新闻（医药）

2025-11-11

·GlobeNewswire-Health Care

Soleno Therapeutics Announces Entry Into $100 Million Accelerated Share Repurchase Agreement

REDWOOD CITY, Calif., Nov. 11, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, announced today that its Board of Directors has authorized a share repurchase and that it has entered into an Accelerated Share Repurchase Agreement (“ASR”) with Jefferies LLC (“Jefferies”) to repurchase $100 million of Soleno’s common stock. “These actions demonstrate our confidence in our commercial launch and the compelling opportunity for VYKAT XR to become a foundational therapy for patients with hyperphagia associated with Prader Willi syndrome,” said Dr. Anish Bhatnagar, Chief Executive Officer and Chairman of the Board of Soleno Therapeutics. “We achieved profitability in the third quarter of 2025 and we believe the expected future cash generation profile of our business is significantly underappreciated by the capital markets and that repurchasing shares of Soleno is a compelling and attractive opportunity.” Under the ASR agreement, the company will make an initial payment of $100 million with an initial delivery of approximately 1,511,553 shares based on Soleno’s closing price on November 10, 2025. The final number of shares repurchased under the ASR will be based on the average of the volume-weighted price of Soleno’s common stock during the term of the transaction and will be subject to adjustments related to the terms and conditions of the ASR agreement. The ASR is expected to be completed in the first quarter of 2026. Cautionary Note Regarding Forward-Looking Statements This 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. 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 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. About Soleno Therapeutics, Inc. Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The Company’s first commercial product, VYKATTM XR (diazoxide choline) extended-release tablets, formerly known as DCCR, is a once-daily oral treatment for hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome. For more information, please visit www.soleno.life. Corporate Contact:Brian RitchieLifeSci Advisors, LLC212-915-2578

2025-11-05

·EndPoints

Soleno's stock drops despite rise in Prader-Willi drug sales

Soleno Therapeutics said sales of its Prader-Willi syndrome therapy have doubled since its launch, but investors are concerned that fewer new patients are starting the drug. For the third quarter of 2025, Vykat XR generated $66 million in revenue, beating analysts’ consensus of $47 million and more than doubling its Q2 earnings, which pulled in between $31 million and $33 million . Vykat XR was approved in March . As of the end of September, around 764 patients were actively using Vykat XR, according to a Tuesday release . In the third quarter, 199 new doctors started prescribing Vykat XR, bringing total patient start forms to 1,043 since the drug’s launch. However, the company’s stock $SLNO dropped by as much as 29% at market open Wednesday. The stock performance was attributed to concerns over a decline in new patient start forms between the second and third quarters, according to a Stifel analyst note on Tuesday. For the third quarter, the company reported 397 patient start forms, down from 646 forms in the second quarter. Further, more patients are also choosing to discontinue Vykat XR, bringing into question its long-term trajectory. Yet Soleno said that some of those patients have restarted the drug, according to the analyst note. Nonetheless, Stifel analysts think the company’s stock drop is “way overdone” and they remain “optimistic longer-term.” Vykat XR is projected to have peak sales of over $2 billion. Since Vykat XR’s early commercial success, other therapies in development for Prader-Willi syndrome have gained attention from investors . Aardvark Therapeutics has a Phase 3 asset in its pipeline, and Rhythm Pharmaceuticals is investigating a candidate in a Phase 2 study. But Acadia Pharmaceuticals recently discarded a Phase 3 Prader-Willi syndrome candidate after it failed in a late-stage study in September. Editor’s note: This story was updated to reflect the company’s stock move after market open.

上市批准临床3期临床2期

2025-10-29

·望远见微

【外资招商】第227期-生物医药企业专刊

编者按欢迎阅读“境外招商之企业巡礼”系列文章！在这个系列中，我们旨在为各地政府提供外资招商引资的宝贵资源，通过每期深入介绍2-3家从属于战略性新兴产业领域、具有一定规模和成长性、并且尚未在中国实际布局的企业，为各地政府部门围绕产业链建设进行精准招商提供参考和借鉴，共同探索如何通过科技创新促进地方经济发展的新路径。本期我们专注于生物领域。放眼当下，在全球健康需求多元化、合成生物学技术突破及可持续发展理念深化的推动下，生物领域内的创新药研发、生物育种、生物制剂三大核心方向正经历深度变革。接下来，我们将带领读者了解一家深耕于罕见病研究的公司。 . 聚焦于罕见病治疗的公司：Soleno Therapeutics 在全球罕见病药物领域，Soleno凭借VYKATXR的突破性地位确立了细分赛道领先优势：PWS作为影响每15000名活产婴儿的罕见病，此前无针对性暴食症状治疗药物，VYKATXR的获批填补了全球治疗空白，成为当前该适应症的唯一标准疗法。行业趋势契合度上，全球罕见病药物市场需求旺盛但供给稀缺，VYKATXR凭借“首创性、适用人群广、安全性良好”三大优势，被分析师预测全球销售峰值可达11亿至20亿美元，公司也有望依托该产品从细分赛道向多罕见病适应症拓展，巩固行业地位。基本信息 1.1 企业简介 Soleno Therapeutics是一家生物研究企业，其总部位于美国加利福尼亚州，成立于1999年，其员工总数为92人。联系方式为企业邮箱：info@sileno.life 1.2 主要产品/服务公司当前核心产品与技术管线高度聚焦小分子化药领域，以钾离子通道为核心靶点，形成 “上市产品+潜力管线””的布局： VYKATXR（原DCCR）：作为全球首个获批用于治疗PWS患者暴食症状的药物，于2025年3月26日获FDA批准，适用于4岁及以上患者。其通过独特机制调节食欲相关通路，临床试验显示，重启治疗的患者在16周后多食症问卷（HQ-CT）总分下降6.3分，且在六个行为领域均有改善，常见副作用包括多毛症、水肿等，未被要求加注“黑框警告”，安全性获监管认可。临床前管线储备：围绕钾离子通道靶点布局其他罕见病适应症研发，但目前公开信息中尚未披露具体在研药物名称及阶段，管线重心暂集中于VYKATXR的商业化与适应症拓展。患者支持服务：配套推出“SolenoOne™”项目，为患者提供保险协助、用药指导等全流程支持，提升药物可及性与治疗依从性，强化商业化落地效果。资本市场 2.1 融资情况该公司并未公开融资情况。 2.2 IPO及股价 2.3 财务表现公司财务表现随核心产品获批进入转型期，从研发驱动向“研发+商业化”双轮驱动过渡：2025年第二季度实现总营收0.33亿美元，这是VYKATXR上市后的首个完整季度收入，远超市场预期，主要来自美国市场的药品销售；净利润方面，2025年第二季度归母净利润为-0.05亿美元，同比大幅收窄78.46%，亏损收窄得益于营收增长与规模效应初显，但尚未实现盈利。现金流方面，2025年第一季度末现金储备为2.9亿美元，7月通过公开募股再筹集2亿美元，为商业化推广与后续研发提供充足资金，分析师预计2025年有望实现盈利，2026年销售收入或达2.68亿美元。此外，公司市值已达37.7亿美元，过去一年股票回报率达60.77%，资本市场对其商业化前景认可度较高。区域布局公司业务以“美国为核心，辐射全球”为策略，聚焦罕见病药物的研发、审批与商业化：在研发端，依托美国本土科研资源推进管线进展，核心产品VYKAT XR的临床试验数据已在儿科内分泌学会（PES）等权威平台发布，夯实临床证据基础；在商业化端，2025年4月在美国启动VYKAT XR销售，快速打开本土市场；在全球化布局上，2025年内已向欧洲药品管理局（EMA）递交VYKAT XR上市申请，预计获批后进入欧洲市场，后续计划逐步拓展至其他有罕见病用药需求的地区，形成“美国+欧洲”双核心市场格局。同时，通过与分析师机构及患者协会合作开展市场教育，提升PWS诊断率与药物认知度，为长期增长铺垫。当前，该公司并未在中国进行业务布局。小结 Soleno Therapeutics作为罕见病药物研发领域的后起之秀，通过聚焦PWS这一未被满足的临床需求，以VYKAT XR的获批实现了从研发型企业向商业化企业的关键跨越。公司总部位于美国科技核心区，依托精准的管线布局与高效的临床推进，填补了全球PWS暴食症状治疗空白，首波销售数据远超预期彰显市场需求潜力。对于有罕见病药物研究需求的地区，这是一家值得关注的公司。 END

IPO

100 项与二氮嗪胆碱相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	二氮嗪胆碱	-	-

研发状态

批准上市

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适应症	国家/地区	公司	日期
多食症	美国	Soleno Therapeutics, Inc.	2025-03-26
普拉德-威利综合症	美国	Soleno Therapeutics, Inc.	2025-03-26

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适应症	最高研发状态	国家/地区	公司	日期
高甘油三酯血症	临床3期	-	Essentialis, Inc.	2011-03-01

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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


NCT03714373 (Study 2-RWP, FDA_CDER) 人工标引	临床3期	多食症 \| 普拉德-威利综合症	77	VYKAT XR	鏇壓網鏇糧齋積齋繭淵(選鑰憲艱醖顧鹽繭簾廠) = 觸獵憲蓋憲夢遞艱廠鬱遞構蓋顧艱艱選衊衊願 (顧夢鑰網蓋鬱憲廠鏇獵, 1.1)	积极	2025-03-27
				Placebo	鏇壓網鏇糧齋積齋繭淵(選鑰憲艱醖顧鹽繭簾廠) = 艱窪製鬱遞遞醖築窪簾遞構蓋顧艱艱選衊衊願 (顧夢鑰網蓋鬱憲廠鏇獵, 1.1)
C602丨DESTINY PWS extension study，DESTINY PWS (C601)丨DESTINY PWS/C601，C601，DESTINY PWS (Pubmed)	-	普拉德-威利综合症	343	二氮嗪胆碱	鏇憲鏇艱齋觸膚願壓製(餘壓襯夢壓蓋齋膚憲鬱) = 觸顧製選壓鬱鏇鑰願顧製憲範獵壓齋鏇範鹽簾 (壓繭鏇蓋範簾鹹淵膚製 ) 更多	积极	2024-04-26
				natural history	鏇憲鏇艱齋觸膚願壓製(餘壓襯夢壓蓋齋膚憲鬱) = 觸觸簾獵觸襯壓鏇夢顧製憲範獵壓齋鏇範鹽簾 (壓繭鏇蓋範簾鹹淵膚製 ) 更多
NCT03714373 (C602, GlobeNewswire) 人工标引	临床3期	普拉德-威利综合症	77	DCCR	願繭鏇蓋網鏇積夢顧蓋(獵襯製鏇衊艱鬱淵鏇艱): P-Value = 0.0022 更多	积极	2023-09-26
				Placebo
NCT03440814 (CTgov)	临床3期	普拉德-威利综合症	127	DCCR	廠廠觸淵糧製積壓範簾(壓廠壓願齋範積築鑰鏇) = 網窪衊壓遞襯積齋艱構願製壓夢鬱網淵夢鑰製 (製遞鏇積膚鬱艱遞夢築, 0.879) 更多	-	2023-09-21
NCT03714373 (C602 \| DESTINY PWS extension study, GlobeNewswire) 人工标引	临床3期	普拉德-威利综合症	115	Diazoxide Choline	鏇網鑰觸簾衊鬱壓齋膚(願廠鹹餘繭願壓構膚窪) = 衊鹹醖鹽鏇鑰選衊衊製簾窪鏇壓積鏇積遞製齋 (齋淵衊構獵壓選壓構餘 )	积极	2021-09-08
				Placebo	-
C601 (NAASO2020)	临床3期	普拉德-威利综合症	127	DCCR	餘觸選獵壓憲襯廠糧網(網窪遞鏇壓糧築蓋醖廠) = 獵齋願網襯襯齋鑰積構網醖夢鹽鬱鹽窪艱築築 (製鹽艱夢襯艱積衊廠鏇 ) 更多	-	2020-11-04
				Placebo	餘觸選獵壓憲襯廠糧網(網窪遞鏇壓糧築蓋醖廠) = 鏇遞製衊繭醖醖鹹繭範網醖夢鹽鬱鹽窪艱築築 (製鹽艱夢襯艱積衊廠鏇 ) 更多