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Soleno Therapeutics Announces Oral Presentation Featuring DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome at ENDO 2024

Soleno Therapeutics Announces Oral Presentation Featuring DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome at ENDO 2024

Soleno Therapeutics 宣佈在 2024 年 ENDO 上以用於普拉德-威利綜合徵的 DCR(二氮氧化膽鹼)緩釋片劑爲特色的口頭報告
GlobeNewswire ·  05/23 04:05

REDWOOD CITY, Calif., May  22, 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 data from the randomized withdrawal period of Study C602 of DCCR (diazoxide choline) extended-release tablets in Prader-Willi Syndrome (PWS) will be featured in an oral presentation at the Annual Meeting of the Endocrine Society (ENDO 2024), being held June 1-4, 2024 in Boston, Massachusetts.

加利福尼亞州雷德伍德城,2024年5月22日(GLOBE NEWSWIRE)——開發治療罕見疾病的新療法的臨床階段生物製藥公司索理諾療法公司(“Soleno”)(納斯達克股票代碼:SLNO)今天宣佈,普拉德威勒中DCCR(二氮氧化膽鹼)緩釋片劑C602研究的隨機撤回期數據內分泌學會年會(ENDO 2024)將在2024年6月1日至4日在馬薩諸塞州波士頓舉行的內分泌學會年會(ENDO 2024)上以口頭方式介紹綜合症(PWS)。

Details of the presentation are as follows:

演示的詳細信息如下:

ENDO 2024

遠藤2024

Title:

Withdrawal of DCCR (Diazoxide Choline) Extended-Release Tablets Worsens Hyperphagia and Increases Weight and BMI in a 16-week Double-blind, Placebo-controlled, Randomized Withdrawal Period in Patients with Prader Willi Syndrome

Format:

Oral Presentation

Session:

OR31. Adipose Tissue, Appetite, and Obesity: Updates on Central Regulation of Appetite and Feeding

Date/Time:

Sunday, June 2, 2024 at 2:45 – 3:00 pm ET

Presenter:

Evelien Gevers, M.D., Ph.D. (Barts Health NHS Trust and Queen Mary University London)

標題:

在普拉德威利綜合徵患者的16周雙盲、安慰劑對照的隨機戒斷期內,停用DCCR(二氮氧化膽鹼)緩釋片劑會加劇吞嚥過度並增加體重和體重指數

格式:

口頭演講

會話:

或者31。脂肪組織、食慾和肥胖:食慾和進食中央調節的最新情況

日期/時間:

美國東部時間 2024 年 6 月 2 日星期日下午 2:45 — 3:00

演示者:

Evelien Gevers,醫學博士、博士(Barts Health NHS 信託基金和倫敦瑪麗皇后大學)

About PWS
The 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 feeling of intense, persistent hunger, food pre-occupation, extreme drive to food seek and consume food that severely diminish the quality of life for patients 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 morbidities (e.g., obesity, diabetes, cardiovascular disease) and mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior). In a global survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents (parent 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.

關於 PWS
美國普拉德-威利綜合症協會估計,每15,000例活產中就有一例發生PWS。這種疾病的標誌性症狀是食慾過多,這是一種長期的、危及生命的感覺,即劇烈持續的飢餓、食物全神貫注、極度渴望尋找和食用食物,嚴重降低了PWS患者及其家人的生活質量。PWS的其他特徵包括行爲問題、認知障礙、肌張力低下、身材矮小(未使用生長激素治療時)、體內多餘脂肪的積累、發育遲緩和性發育不完全。食慾過多可導致嚴重的發病率(例如肥胖、糖尿病、心血管疾病)和死亡(例如胃破裂、窒息、因尋食行爲導致的意外死亡)。在Prader-Willi Research基金會進行的一項全球調查中,96.5%的受訪者(父母和看護者)將食慾過多評爲92.9%的受訪者,將身體成分視爲新藥可以緩解的最重要或非常重要的症狀。目前尚無經批准的治療該疾病的食慾過度/食慾、代謝、認知功能或行爲方面的療法。

About DCCR (Diazoxide Choline) Extended-Release Tablets
DCCR is a novel, proprietary extended-release dosage form containing the crystalline salt of diazoxide and is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of patients in a few rare diseases in neonates, infants, children and adults, but has not been approved for use in PWS. Soleno conceived of and established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline and DCCR in patients 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 patients 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. Diazoxide choline has received Orphan Drug Designation for the treatment of PWS in the U.S. and E.U., and Fast Track and Breakthrough Designations in the U.S.

關於 DCCR(二氮卓膽鹼)緩釋片
DCCR 是一種新型、專有的緩釋劑型,含有二氮氧化物的結晶鹽,每天給藥一次。幾十年來,成千上萬的新生兒、嬰兒、兒童和成人的罕見疾病患者都使用了二氮卓類的母體分子,但尚未獲準用於PWS。Soleno構思並建立了廣泛的專利保護,用於治療PWS患者的二氮氧化物、二氮氧化物膽鹼和DCCR。DCCR開發計劃得到了五項已完成的健康志願者的1期臨床研究和三項已完成的2期臨床研究(其中一項針對PWS患者)的數據支持。在PWS的3期臨床開發項目中,DCCR顯示出有望解決食慾過多(PWS的標誌性症狀)以及其他幾種症狀,例如攻擊性/破壞性行爲、脂肪量和其他代謝參數。二氮氧化膽鹼已在美國和歐盟獲得用於治療PWS的孤兒藥稱號,在美國被認定爲快速通道和突破性藥物。

譯文內容由第三人軟體翻譯。


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