二氮嗪胆碱(Diazoxide Choline) - 药物靶点：Potassium channel_在研适应症：多食症，普拉德-威利综合症_专利_临床

Prospective observational study developed to generate more evidence of the safety profile, clinical characteristics and outcomes of patients with PWS treated with VYKAT XR.

开始日期2025-11-26

申办/合作机构

Soleno Therapeutics, Inc.

NCT05701774

/ Active, not recruiting临床3期

An Open-Label Study of DCCR (Diazoxide Choline) Extended-Release Tablets in Patients With Prader-Willi Syndrome

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.

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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1,298

项与二氮嗪胆碱相关的文献（医药）

2025-05-01·Cell reports methods

Profiling human iPSC-derived sensory neurons for analgesic drug screening using a multi-electrode array

Article

作者: Dussor, Gregory ; Biswas, Swati ; Truong, Vincent ; Walsh, Patrick ; Kolber, Benedict ; Price, Theodore ; Pancrazio, Joseph ; Fofie, Christian Kuete ; Granja-Vazquez, Rafael

Chronic pain is a global health issue, yet effective treatments remain limited due to poor preclinical-to-human translation. To address this, we developed a high-content screening (HCS) platform using hiPSC-derived nociceptors to identify analgesics targeting the peripheral nervous system. These cells, cultured on multi-well microelectrode arrays, achieved nearly 100% active electrodes by week 2, maintaining stable activity for at least 2 weeks. After 28 days, we assessed drug effects on neuronal activity, achieving strong assay performance (robust Z' > 0.5). Pharmacological tests confirmed responses to key analgesic targets, including ion channels (Nav, Cav, Kv, and TRPV1), neurotransmitter receptors (AMPAR and GABA-R), and kinase inhibitors (tyrosine and JAK1/2). Transcriptomic analysis validated target expression, though levels differed from primary human DRG cells. The platform was used to screen over 700 natural compounds, demonstrating its potential for analgesic discovery. This HCS platform facilitates the rapid discovery of uncharacterized analgesics, reducing preclinical-to-human translation failure.

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.

2025-05-01·JOURNAL OF BIOLOGICAL CHEMISTRY

Conversion of glutamate into proline by the leucine analog BCH enhances biphasic insulin secretion in pancreatic β-cells

Article

作者: Gheibi, Sevda ; Spégel, Peter ; Cataldo, Luis Rodrigo ; Straub, Susanne G ; Fex, Malin ; Ardalani, Hamidreza ; Sharp, Geoffrey W G ; Mulder, Hindrik ; Nocquet, Lisa

Biphasic insulin secretion, which fails in type 2 diabetes, can be provoked by various nutrient stimuli, glucose being the superior physiological one. To identify pathways that may play a role in β-cell stimulus-secretion coupling, we compared β-cell and islet functional, secretory, and metabolic responses to glucose and 2-aminobicyclo-(2,2,1)-heptane-2-carboxylic acid (BCH), a leucine analog, that acts as an allosteric activator of glutamate dehydrogenase. We employed a range of techniques, including insulin secretion assays, mitochondrial activity measurements, ATP/ADP ratio assessments, and cytosolic Ca²⁺ level quantifications. Metabolomics was used to analyze cellular metabolite profiles in response to glucose and BCH. Additionally, we investigated the role of proline synthesis by silencing ALDH18A1, encoding proline 5-carboxylate synthase, in both clonal β-cells and human islets. BCH and glucose similarly induced a biphasic insulin response in INS-1 832/13 cells, paralleled by increased mitochondrial activity and raised ATP/ADP ratios, plasma membrane depolarization, and elevated cytosolic Ca²⁺ levels. Metabolomics revealed that proline levels increased significantly only in BCH-stimulated β-cells. Silencing ALDH18A1 disrupted insulin secretion in response to both glucose and BCH, accompanied by reduced cytosolic Ca²⁺ levels, ATP/ADP ratios, and mitochondrial activity. Our findings demonstrated that BCH-induced activation of glutamate dehydrogenase leads to the conversion of glutamate into proline, which apparently enhances β-cell stimulus-secretion coupling. This work identifies a previously unrecognized role of proline metabolism in β-cell function and provides novel insights into the complex regulation of insulin secretion.

151

项与二氮嗪胆碱相关的新闻（医药）

2026-04-22

·PRNewswire

SLNO Investor Alert: Soleno Therapeutics, Inc. Securities Fraud Lawsuit - Investors With Losses May Seek to Lead the Class Action After Allegedly Hiding Post-Launch Safety Crisis: Levi & Korsinsky

From Optimism to Disillusionment: How Investor Sentiment Shifted NEW YORK, April 22, 2026 /PRNewswire/ -- Levi & Korsinsky, LLP provides context on the dramatic shift in investor sentiment surrounding Soleno Therapeutics, Inc. (NASDAQ: SLNO) and its only commercial product, VYKAT XR (diazoxide choline extended-release tablets, or DCCR), approved to treat hyperphagia in individuals with Prader-Willi syndrome ("PWS"). A securities class action has been filed on behalf of shareholders who purchased SLNO stock between March 26, 2025 and November 4, 2025. Find out if you are eligible to recover your SLNO investment losses or contact Joseph E. Levi, Esq. at [email protected] or (212) 363-7500. Soleno's stock commanded significant investor enthusiasm following the March 26, 2025 FDA approval of VYKAT XR as the first-ever approved therapy for PWS hyperphagia. The Company touted nearly 650 patient start forms, 295 unique prescribers, and $32.7 million in quarterly net revenue, fueling confidence that VYKAT XR would become the standard of care for a rare disease community with no prior approved options. The Early Optimism The FDA's approval of VYKAT XR without a boxed warning, REMS requirement, or contraindication for diabetes generated strong initial enthusiasm among shareholders and the PWS community alike. Management characterized the launch as "a very strong start" with "exceptionally encouraging" reception from families and providers. Investors reportedly embraced the narrative that VYKAT XR had a "well-established safety profile" supported by over four years of clinical data. The Growing Concerns Beneath the optimistic surface, the lawsuit contends troubling signals were already emerging in online communities where PWS families gather. According to the complaint, the largest Facebook group dedicated to VYKAT XR discussion saw its tone shift from ambivalent to sharply negative during the summer of 2025. Parents began reporting: Severe fluid retention causing inability to walk or wear shoes Blood sugar levels spiking to pre-diabetic and diabetic ranges Hospitalizations for symptoms consistent with potential heart failure Decisions to discontinue the drug or active consideration of stopping treatment The action alleges that the four most-engaged posts in the group during this period were sharply negative, and the ratio of negative and ambivalent commentary to positive reactions suggested the initial wave of adoptions by desperate families was already exhausting itself. The Breaking Point On August 15, 2025, Scorpion Capital published a 415-page investigative report alleging that Soleno's clinical trials were fundamentally flawed and that DCCR posed far greater safety risks than the Company had disclosed. The report included interviews with eight of the drug's own trial investigators, the filing states, who broadly characterized VYKAT XR as a failure and expressed reluctance to prescribe it. The report further alleged that DCCR carried high risks of pre-diabetes, diabetes, pulmonary edema, and congestive heart failure, and that Soleno had obscured alarming fluid retention data by conflating it with increases in lean body mass. Sentiment Arc and Investor Harm The complaint asserts that the contrast between management's public assurances and the reality experienced by patients and documented by independent researchers represents a material gap that harmed investors. Just nine days before the Scorpion Capital report, management had stated there were "no new safety signals" in the post-marketing setting and that discontinuation rates were "substantially lower than what we saw even in clinical trials." The lead plaintiff deadline is May 5, 2026. "Investor confidence depends on receiving truthful information from the companies they invest in. When optimism is built on incomplete safety disclosures, the correction can be swift and severe." -- Joseph E. Levi, Esq. Speak with a securities attorney about recovering your Soleno losses or call (212) 363-7500. Levi & Korsinsky, LLP is a nationally recognized shareholder rights firm. Over the past 20 years, the firm has secured hundreds of millions of dollars for aggrieved shareholders. Ranked in ISS Top 50 for seven consecutive years. CONTACT: Levi & Korsinsky, LLP Joseph E. Levi, Esq. Ed Korsinsky, Esq. 33 Whitehall Street, 27th Floor New York, NY 10004 [email protected] Tel: (212) 363-7500 Fax: (212) 363-7171 SOURCE Levi & Korsinsky, LLP 21% more press release views with Request a Demo

上市批准专利侵权

2026-04-21

·PRNewswire

SLNO Shareholder Alert: Soleno Therapeutics, Inc. Securities Class Action Lawsuit Investors With Losses May Join -- The Gross Law Firm

NEW YORK, April 21, 2026 /PRNewswire/ -- The Gross Law Firm issues the following notice to shareholders of Soleno Therapeutics, Inc. (NASDAQ: SLNO ). Shareholders who purchased shares of SLNO during the class period listed are encouraged to contact the firm regarding possible lead plaintiff appointment. Appointment as lead plaintiff is not required to partake in any recovery. CONTACT US HERE: CLASS PERIOD: March 26, 2025 to November 4, 2025 ALLEGATIONS: The complaint alleges that during the class period, Defendants issued materially false and/or misleading statements and/or failed to disclose that: (a) the Soleno Phase 3 clinical trial program for product diazoxide choline extended-release tablets, DCCR, had systematically downplayed, misrepresented, and/or concealed significant evidence of safety concerns potentially related to the administration of DCCR, including issues related to excess fluid retention in clinical trial participants; (b) as a result of (a) above, the administration of DCCR to treat hyperphagia in individuals with PWS posed materially greater safety risks than disclosed by the Company or its executives; and (c) as a result of (a)-(b) above, DCCR had materially lower commercial viability and undisclosed risks related to the likelihood of significant and widespread adverse events after its commercial launch, including risks related to patient discontinuation rates, lower patient adoption, prescriber reluctance, adverse regulatory action, and potential reputational and legal fallout. DEADLINE: May 5, 2026 Shareholders should not delay in registering for this class action. Register your information here: NEXT STEPS FOR SHAREHOLDERS: Once you register as a shareholder who purchased shares of SLNO during the timeframe listed above, you will be enrolled in a portfolio monitoring software to provide you with status updates throughout the lifecycle of the case. The deadline to seek to be a lead plaintiff is May 5, 2026. There is no cost or obligation to you to participate in this case. WHY GROSS LAW FIRM? The Gross Law Firm is a nationally recognized class action law firm, and our mission is to protect the rights of all investors who have suffered as a result of deceit, fraud, and illegal business practices. The Gross Law Firm is committed to ensuring that companies adhere to responsible business practices and engage in good corporate citizenship. The firm seeks recovery on behalf of investors who incurred losses when false and/or misleading statements or the omission of material information by a company lead to artificial inflation of the company's stock. Attorney advertising. Prior results do not guarantee similar outcomes. CONTACT: The Gross Law Firm 15 West 38th Street, 12th floor New York, NY, 10018 Email: [email protected] Phone: (646) 453-8903 SOURCE The Gross Law Firm 21% more press release views with Request a Demo

临床3期

2026-04-21

·EndPoints

Soleno sold at a discount to Neurocrine due to dwindling European prospects

The bleak European approval potential for Soleno Therapeutics’ genetic obesity drug essentially forced the US drugmaker to sell itself for less than it was once worth. In a rare case, Soleno sold at a price lower than its initial buyout offer, according to new documents outlining its $2.9 billion deal with Neurocrine Biosciences announced earlier this month. Neurocrine’s offer was roughly a 38% drop in per-share value from another company’s earlier offer in May 2025. Soleno had secured an FDA nod for its Prader-Willi syndrome drug and reached profitability as a single-asset commercial biotech, but its longer-term prospects and faltering expansion potential in Europe led a few companies to back out of the process, according to the Monday SEC filing. The Soleno deal marks the second such case this year of a biotech selling at a discount to the initial offer. Terns Pharmaceuticals’ exit to Merck fell by $1 billion during due diligence. Soleno looked poised for a strong launch last March, when it secured the first FDA greenlight for a medicine for Prader-Willi syndrome, a rare condition that leads to insatiable hunger and obesity. Other drugmakers like Acadia, Rhythm and Aardvark were seeking to make a mark in the field , too. Weeks after the US nod for the drug, called Vykat XR, Soleno started the approval request process in the European Union. A few weeks later, on May 22, an unnamed biopharma company submitted an $86 per share cash offer to acquire Soleno, according to the regulatory documents released Monday. That company, referred to as “Party A,” quickly backed out a month later, citing “analysis regarding the longevity of Soleno’s intellectual property portfolio and concerns about the size of the market opportunity of Soleno’s business in Europe,” according to the SEC filing. Another unnamed biopharma, called “Party B,” also pulled out of discussions in early February because of “concerns about Soleno’s business prospects in Europe and a desire to continue to monitor Vykat XR’s commercial launch in the United States.” Later that month, on Feb. 27, the biotech received word from the European Medicines Agency that “caused Soleno concern that it may not receive approval from the EMA.” Despite those concerns, on March 18, Neurocrine CEO Kyle Gano delivered to Soleno CEO Anish Bhatnagar an initial $50 per share cash offer. Soleno’s stock had sunk to $32.80 per share at that time. Its 52-week peak had been about $90 per share. On March 30, a third unnamed company said it wouldn’t make an acquisition offer until after Soleno’s first quarter earnings results and an oral hearing at the EMA regarding its approval application. That same day, Neurocrine’s CEO upped his offer to $53 per share. On the day of the April 6 deal announcement, Gano told analysts that Neurocrine wouldn’t seek European approval. The next day, Soleno said it withdrew on its EMA approval application.

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100 项与二氮嗪胆碱相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
-	二氮嗪胆碱	-	-

研发状态

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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


Literature 人工标引	临床3期	普拉德-威利综合症	77	diazoxide choline extended-release	遞顧範襯範衊鬱積鹽壓(願範製淵鏇鬱簾構窪鹽) = 築願廠醖遞繭願鹽齋廠餘鏇範襯鏇願淵鬱齋觸 (選膚壓範鑰簾鹹齋築選, 1.12) 更多	积极	2026-01-02
				placebo	遞顧範襯範衊鬱積鹽壓(願範製淵鏇鬱簾構窪鹽) = 廠齋鹹鬱網膚願蓋壓選餘鏇範襯鏇願淵鬱齋觸 (選膚壓範鑰簾鹹齋築選, 1.09) 更多
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	餘艱壓淵衊構願窪醖膚(糧蓋襯齋鹽衊廠鬱艱獵) = 鏇鹽鬱築鬱繭遞觸壓獵衊築積憲鬱簾糧築願顧 (鹽積膚獵壓遞築製鑰觸, 1.1)	积极	2025-03-27
				Placebo	餘艱壓淵衊構願窪醖膚(糧蓋襯齋鹽衊廠鬱艱獵) = 顧鹹遞鑰膚觸選艱獵鹽衊築積憲鬱簾糧築願顧 (鹽積膚獵壓遞築製鑰觸, 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	簾憲遞衊糧夢夢艱膚觸(夢願餘壓餘構範選選網) = 廠製鑰鏇範獵觸鑰顧網獵襯顧繭獵襯獵廠築衊 (襯蓋觸齋膚窪餘艱鏇憲 ) 更多	积极	2024-04-26
				natural history	簾憲遞衊糧夢夢艱膚觸(夢願餘壓餘構範選選網) = 顧淵壓艱廠糧觸鹹簾鬱獵襯顧繭獵襯獵廠築衊 (襯蓋觸齋膚窪餘艱鏇憲 ) 更多
NCT03714373 (C602, GlobeNewswire) 人工标引	临床3期	普拉德-威利综合症	77	DCCR	窪夢餘醖憲蓋壓襯積鏇(壓選糧艱築糧廠繭範餘): P-Value = 0.0022 更多	积极	2023-09-26
				Placebo
NCT03440814 (DESTINY PWS, CTgov)	临床3期	普拉德-威利综合症	127	DCCR	窪醖壓製窪齋醖醖鬱淵(顧築鹹齋壓簾糧範獵窪) = 鑰遞壓鬱廠艱窪鏇齋遞鑰簾願蓋餘積齋積蓋襯 (衊憲淵鹽衊膚觸獵餘鏇, 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	淵簾鏇衊網築範簾鬱鑰(遞構繭範鑰顧糧憲淵選) = 淵鏇窪製蓋範襯製廠鏇積襯獵膚膚蓋夢繭憲積 (範衊獵獵廠網築窪憲積 )	积极	2021-09-08
				Placebo	-
C601 (NAASO2020)	临床3期	普拉德-威利综合症	127	DCCR	觸醖鏇願鹽餘餘窪鏇醖(鏇積繭淵蓋醖鏇壓繭糧) = 顧簾廠艱製選淵願繭糧艱簾簾淵鏇鏇壓觸獵夢 (襯願餘築願餘築膚獵窪 ) 更多	-	2020-11-04
				Placebo	觸醖鏇願鹽餘餘窪鏇醖(鏇積繭淵蓋醖鏇壓繭糧) = 廠醖膚築齋壓繭製窪衊艱簾簾淵鏇鏇壓觸獵夢 (襯願餘築願餘築膚獵窪 ) 更多