REDWOOD CITY, Calif., June 28, 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 the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia..

加利福尼亚州红木市，2024年6月28日（环球通讯社）--Soleno Therapeutics，Inc.（“Soleno”）（纳斯达克：SLNO），一家临床阶段的生物制药公司，开发用于治疗罕见疾病的新型疗法，今天宣布向美国食品和药物管理局（FDA）提交新药申请（NDA），以批准DCCR（二氮嗪胆碱）缓释片，用于治疗四岁及以上患有食欲亢进症的个体的Prader-Willi综合征（PWS）。。

“Submission of the DCCR NDA to the FDA marks a significant milestone not only for Soleno, but for people living with PWS,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “This regulatory submission advances us one important step closer to our goal of bringing to market a new therapeutic for individuals with PWS that addresses the life-threatening hyperphagia and other key aspects of this serious and rare condition.

Soleno Therapeutics首席执行官医学博士Anish Bhatnagar说：“向FDA提交DCCR NDA不仅对Soleno，而且对PWS患者来说都是一个重要的里程碑。“这份监管文件使我们离我们的目标更近了一步，即为PWS患者推出一种新的治疗方法，以解决威胁生命的食欲亢进和这种严重而罕见疾病的其他关键方面。

We look forward to working with the FDA throughout the review process. We extend sincere gratitude to the team at Soleno, investigators, study site teams, advocacy organizations and most importantly, the individuals with PWS and their families who were instrumental in completing our DCCR development program.”.

我们期待在整个审查过程中与FDA合作。我们衷心感谢Soleno的团队、调查人员、研究现场团队、倡导组织，最重要的是，感谢PWS的个人及其家人，他们为完成我们的DCCR发展计划做出了贡献。”。

DCCR has Breakthrough and Fast Track Designations in the U.S., as well as Orphan Drug Designation for PWS in the U.S. and E.U. The FDA has 60 days to determine whether the NDA is accepted for review. Soleno has requested Priority Review of the NDA, which would provide a target review period of six months by the FDA after the NDA has been accepted..

DCCR在美国有突破性的快速通道指定，在美国和欧盟也有PWS的孤儿药指定。FDA有60天的时间来确定NDA是否被接受审查。索莱诺已要求优先审查保密协议，在保密协议被接受后，FDA将提供六个月的目标审查期。。

About PWS

关于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 individuals with PWS and their families.

美国Prader-Willi综合征协会估计，每15000例活产中就有一例发生PWS。这种疾病的标志性症状是食欲亢进，这是一种慢性且危及生命的感觉，包括强烈持续的饥饿感，食物占领前，寻求食物和食用食物的极端冲动，严重降低了PWS患者及其家人的生活质量。

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.

PWS的其他特征包括行为问题，认知障碍，肌张力低下，身材矮小（未接受生长激素治疗），体内脂肪过多，发育迟缓和性发育不完全。。

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..

在Prader Willi Research基金会进行的一项全球调查中，96.5%的受访者（父母和照顾者）认为食欲亢进，92.9%的人认为身体成分是新药可以缓解的最重要或非常重要的症状。目前还没有批准的疗法来治疗这种疾病的食欲亢进/食欲，代谢，认知功能或行为方面。。

About DCCR (Diazoxide Choline) Extended-Release Tablets

关于DCCR（二氮嗪胆碱）缓释片

DCCR 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.

DCCR是一种新型的专有缓释剂型，含有二氮嗪胆碱，二氮嗪的结晶盐，每天给药一次。母体分子二氮嗪已在数千个人中用于新生儿，婴儿，儿童和成人的几种罕见疾病中，但尚未批准用于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.

Soleno构思并建立了广泛的专利保护，用于治疗PWS患者的二氮嗪，二氮嗪胆碱和DCCR。DCCR开发计划得到了来自健康志愿者的五项已完成的1期临床研究和三项已完成的2期临床研究的数据的支持，其中一项是针对PWS患者的。

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.

在PWS 3期临床开发计划中，DCCR有望解决PWS的标志性症状食欲亢进，以及其他一些症状，例如攻击性/破坏性行为，脂肪量和其他代谢参数。二氮嗪胆碱已被指定为美国治疗PWS的孤儿药。

and E.U., and Fast Track and Breakthrough Designations in the U.S..

。。

About Soleno Therapeutics, Inc.

关于Soleno Therapeutics，Inc。

Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company recently submitted an NDA to the FDA, supported by its Phase 3 development program, for its lead candidate, DCCR (diazoxide choline) extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi syndrome (PWS).

Soleno专注于治疗罕见疾病的新型疗法的开发和商业化。该公司最近在其第三阶段开发计划的支持下，向FDA提交了一份NDA，用于其主要候选药物DCCR（二氮嗪胆碱）缓释片，这是一种每日一次的口服片剂，用于治疗Prader-Willi综合征（PWS）。