Soleno Therapeutics Announces Peer-Reviewed Publication Of Data Comparing DCCR Treatment To The Natural History Of Prader-Willi Syndrome
Soleno Therapeutics Announces Peer-Reviewed Publication Of Data Comparing DCCR Treatment To The Natural History Of Prader-Willi Syndrome
Soleno Therapeutics, Inc. (Soleno) (NASDAQ:SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced the publication of the comparison of results from the Company's Phase 3 placebo-controlled study (C601) and open-label extension study (C602) evaluating investigational, once-daily DCCR (Diazoxide Choline) Extended-Release tablets in patients with Prader-Willi syndrome (PWS), to data from the PATH for PWS (PATH) natural history study, in the Journal of Neurodevelopmental Disorders. The article, entitled, "Behavioral Changes in Patients with Prader-Willi Syndrome Receiving Diazoxide Choline Extended-Release Tablets Compared to the PATH for PWS Natural History Study," can be found HERE.
Soleno Therapeutics, Inc.（Soleno）（纳斯达克股票代码：SLNO）是一家开发治疗罕见疾病的新疗法的临床阶段生物制药公司，今天宣布公布了该公司的3期安慰剂对照研究（C601）和开放标签延期研究（C602）结果的比较结果，该研究评估了每天一次的研究性DCCR（二氮氧化胆碱）延期释放从PATH for PWS（PATH）自然史研究的数据来看，普拉德-威利综合征（PWS）患者的片剂 神经发育障碍杂志。这篇题为 “与PWS自然史研究的PATH相比，接受二氮氧化物胆碱缓释片剂的普拉德-威利综合征患者的行为变化” 的文章可以在这里找到。
Data from DCCR-treated participants in the C602/C602 cohort were compared to results from a cohort of comparable participants from PATH using the same caregiver-completed questionnaires to measure hyperphagia (Hyperphagia Questionnaire for Clinical Trials [HQ-CT]) and PWS-related behaviors (the Prader-Willi Syndrome Profile [PWSP]) in six domains: aggressive behaviors, anxiety, compulsivity, depression, disordered thinking, and rigidity-irritability.
将来自C602/C602队列中接受DCCR治疗的参与者的数据与来自PATH的一组可比参与者的结果进行了比较，使用护理人员填写的相同问卷来测量食欲过度（临床试验超吞问卷 [HQ-CT]）和PWS相关行为（普拉德-威利综合征概况 [PWSP]）六个领域：攻击行为、焦虑、强迫性、抑郁、思维障碍，以及刚性-易怒性。
Hyperphagia: Participants treated with DCCR showed highly statistically significant and clinically meaningful improvement with DCCR administration relative to participants in the PATH study at 6 and 12 months (p<0.001 for both comparisons).
食欲亢进：与PATH研究的参与者相比，接受DCCR治疗的参与者在6个月和12个月时使用DCCR时表现出极高的统计学意义和临床意义的改善（两项比较均为p
PWS-related behaviors: As with hyperphagia, highly statistically significant and clinically meaningful improvements were seen in DCCR treated participants compared to those in the PATH study in all behavioral domains of the PWSP at 6 and 12 months (p≤0.003 for all domains).
PWS相关行为：与食欲过度一样，与PATH研究的参与者相比，接受DCCR治疗的参与者在PWSP的所有行为领域在6个月和12个月时均出现了具有高度统计学意义且具有临床意义的改善（所有领域的p≤0.003）。
"The PATH for PWS study is an important evaluation of the natural history of individuals with PWS," said Dr. Theresa Strong, Director of Research Programs for FPWR. "These highly significant improvements with long-term DCCR treatment compared to the PATH data suggest that DCCR has the potential to provide much needed and substantial improvement in the lives of those living with PWS and their families. We are excited to see DCCR advance through the regulatory process and look forward to continuing to support Soleno in its efforts."
FPWR研究项目主任特蕾莎·斯特朗博士说：“PATH for PWS研究是对PWS患者自然史的重要评估。”“与PATH数据相比，长期DCCR治疗的这些非常显著的改善表明，DCCR有可能为PWS患者及其家人的生活提供急需的实质性改善。我们很高兴看到DCCR在监管程序中取得进展，并期待继续支持索理诺的努力。”
"DCCR has the potential to substantially improve the quality of life for individuals with PWS and their families," said PATH Study Principal Investigators, Jennifer Miller, M.D. and Shawn McCandless, M.D. "These data clearly demonstrate that long-term treatment with DCCR resulted in changes in hyperphagia and other behavioral complications of PWS that are meaningfully improved compared to the natural history of the disease. We believe these results support the significant potential of DCCR in PWS and are eager to offer a much-needed treatment option to patients in need, if approved."
PATH研究首席研究员、医学博士詹妮弗·米勒和医学博士肖恩·麦坎德利斯说：“DCCR有可能显著改善PWS患者及其家人的生活质量。这些数据清楚地表明，长期使用DCCR治疗会导致PWS的食欲过度和其他行为并发症发生变化，与该疾病的自然史相比，这些并发症得到了显著改善。我们认为，这些结果支持了DCCR在PWS中的巨大潜力，如果获得批准，我们渴望为有需要的患者提供急需的治疗选择。”