RedHill Biopharma的Opaganib新冠肺炎研究通过独立委员会第二次审查

第二次计划内独立安全监测委员会（SMC）审查一致建议继续按原方案进行opaganib治疗新冠肺炎的美国2期研究
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美国2期研究已招募到75%的患者，数据
预计将在本季度末公布
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与此同时，opaganib治疗新冠肺炎的全球2/3期研究的招募工作也进展迅速
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全球紧急使用授权申请已在计划中，将等待积极临床结果

以色列特拉维夫和北卡罗来纳州罗利市, Oct. 09, 2020 (GLOBE NEWSWIRE) -- 特种生物制药公司RedHill Biopharma Ltd.（纳斯达克：RDHL ）（以下简称“ RedHill”或“该公司”）今天宣布，该公司以opaganib（Yeliva®，ABC294640）¹治疗重症新冠肺炎住院患者的美国2期研究通过了独立安全监测委员会（SMC）的第二次计划内安全审查。委员会一致建议遵循原方案继续进行研究。SMC的建议基于对研究中接受至少7天治疗的前24名患者安全数据的非盲态分析。研究已招募到75%的患者。

opaganib的美国2期研究（NCT04414618）正在八个临床试验中心进行，计划在本月完成招募，预计数据将在今年年底前公布。2期研究不是为了验证疗效的效力，而是侧重于安全评估和识别疗效信号。

与此同时，opaganib针对重症新冠肺炎患者的全球2/3期研究（NCT04467840）正在15个研究中心迅速展开招募，即将完成270例患者的招募目标。这项研究以疗效评估为重点和目的。该项研究已在英国、意大利、俄罗斯、墨西哥、巴西和以色列获得批准，目前正在进一步扩展。

“此次计划内第二次独立安全审查涉及研究中60%患者的相关数据，审查的通过是当前进行的opaganib作为重症新冠肺炎患者潜在治疗药物开发过程中的一个重要里程碑，”
RedHill医疗总监Mark L. Levitt（医学博士，哲学博士）说。
“我们正在快速建立有关opaganib的更多数据和经验，opaganib研究的安全性数据库现已拥有近200例患者的数据。这给正在进行的全球2/3期研究提供了进一步支持。该研究的重点是确定疗效，并有望在年底前招募270名患者。”

RedHill正在与美国政府机构就可能提供的资金进行讨论，以支持opaganib为申请紧急用药批准以及扩大生产规模而取得的迅速进展。

关于Opaganib（ABC294640，Yeliva^®）
Opaganib作为一种新型化学实体，是专有、首创口服鞘氨醇激酶-2 (SK2) 选择性抑制剂，显示抗炎和抗病毒双重活性，靶向宿主细胞成分，有可能最大限度地降低病毒耐药的可能性。Opaganib还显示出抗癌活性，并有可能针对多种肿瘤、病毒、炎症和肠道适应症。

opaganib获得了美国FDA针对治疗胆管癌的罕见病用药批准，目前正在进行晚期胆管癌2a期研究评估和前列腺癌2期研究评估。此外还有一项全球2/3期研究和一项美国2期研究正在对opaganib治疗重症新冠肺炎的效果进行评估。

临床前数据显示，opaganib同时具有抗炎和抗病毒活性，具有减少炎性肺部疾病（如肺炎）和减轻肺纤维化损害的潜力。Opaganib表现出对导致新冠病毒的有效抗病毒活性，在人体肺支气管组织的体外模型中完全抑制病毒复制。此外，临床前体内研究²表明，通过降低支气管肺泡灌洗液中IL-6和TNF-α的水平，opaganib可降低流感病毒感染的病死率，并改善铜绿假单胞菌诱发的肺损伤。

opaganib最初由美国Apogee Biotechnology Corp开发，并在多项研究中获得成功，包括肿瘤、炎症、胃肠道和放射防护模型的多项临床前研究、针对晚期实体肿瘤患者的一项1期临床研究和一项额外的多发性骨髓瘤1期研究。

根据一项同情用药计划，以色列一家领先医院的新冠肺炎患者（按世界卫生组织的等级分类）接受了opaganib治疗。这些首批使用opaganib的新冠肺炎重症患者的治疗数据已经公布²。治疗结果分析表明，与同一医院回顾性配对病例对照组的患者相比，接受opaganib同情用药治疗的患者在临床结果和炎症标志物方面均有显著获益。opaganib治疗组的所有患者都在呼吸室内空气且无需使用插管和呼吸机的情况下出院，而对应的病例对照组中有33%的患者需要插管和使用呼吸机。opaganib治疗组的鼻高流量氧疗脱离的中位时间减少到10天，而匹配病例对照组则为15天。

Opaganib的开发因美国联邦和州政府机构给予Apogee Biotechnology Corp.的拨款和合同而得到支持，相关机构包括NCI、BARDA、美国国防部和FDA罕见病药物开发办公室。

正在进行的各项opaganib研究已在www.ClinicalTrials.gov上注册，该网站是美国国家卫生研究院提供的一项网上服务，为公众提供了访问公共和私人支持的临床研究信息的途径。  
       
关于RedHill Biopharma           
RedHill Biopharma Ltd.（Nasdaq：RDHL) 是一家主要关注肠胃疾病治疗的特种生物制药企业。RedHill目前推广的胃肠药物包括：Movantik^®——用于治疗阿片类药物引起的成人便秘³、Talicia^®——用于治疗成人幽门螺杆菌（H. pylori）⁴感染和Aemcolo^®——用于治疗成人旅行者腹泻⁵。RedHill的主要临床后期开发项目包括：(1) RHB-204，计划针对肺部非结核分枝杆菌 (NTM) 感染进行关键3期研究；(2) opaganib (Yeliva^®)，首创SK2选择性抑制剂，针对多种适应症，正在进行新冠肺炎治疗的2/3期研究，以及针对前列腺癌和胆管癌进行的2期研究；(3) RHB-104，针对克罗恩氏病进行的3期首次研究取得了积极成果；(4) RHB-102 (Bekinda^®)，针对急性胃肠炎和胃炎的3期研究取得了积极成果，针对IBS-D的2期研究也取得了积极成果；(5) RHB-107，一种处于2期研究的首创丝氨酸蛋白酶抑制剂，针对癌症和炎性胃肠疾病，并且正在针对新冠肺炎进行评估；以及 (6) RHB-106，一种肠道准备胶囊。有关该公司的更多信息，请访问：www.redhillbio.com。

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements may be preceded by the words “intends,” “may,” “will,” “plans,” “expects,” “anticipates,” “projects,” “predicts,” “estimates,” “aims,” “believes,” “hopes,” “potential” or similar words. Forward-looking statements are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control and cannot be predicted or quantified, and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, the risk that the Company’s Phase 2/3 study evaluating opaganib will not be successful; the risk that the antiviral activity of opaganib shown in the preclinical human lung cell model will not be demonstrated in clinical trials; the risk of a delay in receiving data to support emergency use applications; the risk that other COVID-19 patients treated with opaganib will not show any clinical improvement; the risk that clinical trials with opaganib in Brazil, Israel, the U.S., Italy, Russia, the UK, Mexico or elsewhere for the treatment of COVID-19, if conducted at all, will not show any improvement in patients; the risk of a delay in applying for emergency use authorizations, if at all; the development risks of early-stage discovery efforts for a disease that is still little understood, including difficulty in assessing the efficacy of opaganib for the treatment of COVID-19, if at all; intense competition from other companies developing potential treatments and vaccines for COVID-19; the effect of a potential occurrence of patients suffering serious adverse events using opaganib under compassionate use programs, as well as risks and uncertainties associated with (i) the initiation, timing, progress and results of the Company’s research, manufacturing, preclinical studies, clinical trials, and other therapeutic candidate development efforts, and the timing of the commercial launch of its commercial products and ones it may acquire or develop in the future; (ii) the Company’s ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials (iii) the extent and number and type of additional studies that the Company may be required to conduct and the Company’s receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings, approvals and feedback; (iv) the manufacturing, clinical development, commercialization, and market acceptance of the Company’s therapeutic candidates and Talicia^®; (v) the Company’s ability to successfully commercialize and promote Movantik^®, Talicia^® and Aemcolo^®; (vi) the Company’s ability to establish and maintain corporate collaborations; (vii) the Company's ability to acquire products approved for marketing in the U.S. that achieve commercial success and build and sustain its own marketing and commercialization capabilities; (viii) the interpretation of the properties and characteristics of the Company’s therapeutic candidates and the results obtained with its therapeutic candidates in research, preclinical studies or clinical trials; (ix) the implementation of the Company’s business model, strategic plans for its business and therapeutic candidates; (x) the scope of protection the Company is able to establish and maintain for intellectual property rights covering its therapeutic candidates and commercial products and its ability to operate its business without infringing the intellectual property rights of others; (xi) parties from whom the Company licenses its intellectual property defaulting in their obligations to the Company; (xii) estimates of the Company’s expenses, future revenues, capital requirements and needs for additional financing; (xiii) the effect of patients suffering adverse events using investigative drugs under the Company's Expanded Access Program; and (xiv) competition from other companies and technologies within the Company’s industry. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the Securities and Exchange Commission (SEC), including the Company's Annual Report on Form 20-F filed with the SEC on March 4, 2020. All forward-looking statements included in this press release are made only as of the date of this press release. The Company assumes no obligation to update any written or oral forward-looking statement, whether as a result of new information, future events or otherwise unless required by law.

注意：本新闻稿是公司以英文发布的官方新闻稿的译文，为方便查阅之目的而提供。

¹Opaganib是一种试验性新药，尚未批准商业销售。
²Xia C.等。 Transient inhibition of sphingosine kinases confers protection to influenza A virus infected mice（神经氨酸激酶的短暂抑制对感染甲型流感病毒小鼠的保护作用） Antiviral Res. 2018年10月; 158:171-177. Ebenezer DL等. Pseudomonas aeruginosa stimulates nuclear sphingosine-1-phosphate generation and epigenetic regulation of lung inflammatory injury（铜绿假单胞菌可刺激细胞核鞘糖苷-1-磷酸的生成和肺炎症损伤的表观遗传调控） Thorax。2019年6月；74(6):579-591。
³ 3  Movantik^®（naloxegol）的完整处方信息请见：www.Movantik.com。
⁴ Talicia^®（奥美拉唑镁、阿莫西林和利福布汀）的完整处方信息请见：www.Talicia.com。   
⁵ Aemcolo^®（利福霉素）的完整处方信息请见：www.Aemcolo.com。