•RedHill将受益于各项激励措施，大力开发胆管癌药物YELIVA®(ABC294640);同时如果该药物获得批准上市，还将获得七年时间的排他性销售权利；
  •晚期、不可切除、肝内和肝外胆管癌患者中的Ⅱa期YELIVA®临床研究计划将于2017年第三季度启动；
  •胆管癌是一种高度致命的恶性肿瘤，患者迫切需要更有效的全身治疗手段和药物;根据肿瘤类型和诊断时间的不同，胆管癌患者的5年相对存活率在2%至30%之间；
  •在晚期实体瘤患者中进行的Ⅰ期研究，YELIVA®成功达到了其主要和次要试验终点;一期研究对三名患者进行了试验，其中一例患者症状得到持续的部分缓解，另外两例患者病情则保持了稳定；
  •RedHill正在对YELIVA®进行几项Ⅰ/Ⅱ期临床研究，试验主要针对多种肿瘤学和炎症指征。其中的几项试验获得了国家癌症研究所(NCI)、Apogee生物技术公司和美国大学等的资助；
  •YELIVA®是一种专有的、首创的口服鞘氨醇激酶-2(SK2)选择性抑制剂，具有抗癌和抗炎活性的特点。

   RedHill Biopharma有限公司是一家专注于晚期临床阶段、专有口服胃肠炎等炎性疾病和癌症小分子药物的开发和销售的公司。该公司于2017年4月4日宣布，美国食品和药物管理局(FDA)已经批准了胆管癌药物 YELIVA®(ABC294640)的罕见病药物申请。
  获批罕见病药物后，RedHill将获得各种发展激励措施的帮助，包括合格临床测试的税收抵免、减免处方药用户费用(PDUFA 费用)等，同时如果该药物获得批准上市，还将获得七年时间的排他性销售权利。
  YELIVA®是一种专有的、首创的口服鞘氨醇激酶-2(SK2)选择性抑制剂，具有抗肿瘤和抗炎活性，可以靶向作用于多种肿瘤、炎症和胃肠道等适应症。通过抑制SK2酶，YELIVA®可以阻止丝氨酸1-磷酸(S1P)的合成，这样一来，丝氨酸1-磷酸促进癌症生长和病理性炎症的作用就被阻止。
  RedHill肿瘤医学主任Mark L. Levitt博士说：“胆管癌是一种预后极度不良的癌症。目前可供这类患者选择的治疗方案很少，而且疗效也十分有限。基于有希望的临床前资料，以及三位之前接受过治疗的肝外胆管癌患者，YELIVA®进行了Ⅰ期研究，我们希望YELIVA®能够为患者提供急需的新治疗方案。我们非常高兴该药物的罕见病药物申请获得了批准，目前我们正积极地Ⅱa期研究的准备工作，目的是评估YELIVA®在不可切除、肝内和肝外胆管癌患者的安全性和有效性，试验计划在今年第三季度正式启动。”
  胆管癌(胆管癌)是一种高度致命的恶性肿瘤，患者迫切地需要更有效的全身治疗手段。美国每年大约有8000人被诊断患有肝内或肝外胆管癌。最近的研究显示，胆管癌的发病率还在进一步增加，这主要是由于近期该疾病诊断技术的进展使得原本不能被诊断出来的的患者筛查了出来。目前，完全切除手术仍然是胆管癌的唯一治疗方法，但只有少数患者的肿瘤可以用此方法治疗。其他治疗方案包括放射治疗和化学治疗;然而，这些治疗方式的疗效也是十分有限的。尽管诊断和治疗胆管癌患者的能力总体上有所改善，但初次化疗失败的复发患者预后仍然很差，总体中位生存期约为1年。根据肿瘤类型和诊断时间的不同，胆管癌患者的5年相对存活率在2%至30%之间。
  YELIVA®在南卡罗来纳医科大学(MUSC)Hollings癌症中心对晚期实体瘤患者进行了Ⅰ期研究，该研究的最终结果满足了其主要和次要终点，证明了该药物耐受性良好，并且可以达到治疗效果的剂量安全地施用于癌症患者。
  在Ⅰ期研究中的3例胆管癌患者在之前均接受过了治疗，使用了YELIVA®后1例受试者获得了持续的部分缓解(总体生存=20.3个月)，其余2例患者延长了稳定期(总体生存分别为17.6个月和16.3个月)。
  RedHill计划在2017年第三季度启动Ⅱ期单臂临床研究，该研究将在晚期、不可切除的肝内、肝外胆管癌患者中进行。该研究将使用YELIVA®作为治疗胆管癌患者的单一药物，以确定胆管癌对这种治疗的疗效。
  YELIVA®用于治疗晚期肝细胞癌(HCC)的Ⅱ期研究正在南卡罗来纳医科大学Hollings癌症中心进行。该研究获得了NCI的180万美元资助，用于研究鞘脂代谢治疗各种实体性肿瘤的可行性，此外还得到了RedHill更多的支持。
  杜克大学医学中心正在进行一项关于YELIVA®治疗难治性或复发性多发性骨髓瘤的Ⅰb/Ⅱ期研究。该研究Apogee生物技术公司(Apogee)与Duke大学合作进行，获得了由NEC小企业创新研究计划(SBIR)200万美元的资助，此外还得到了RedHill的额外支持
  路易斯安那州立大学健康科学中心正在进行一项 YELIVA®的Ⅰ/Ⅱ期临床研究，以评估该药物对难治性/复发性弥漫性大B细胞淋巴瘤以及卡波西肉瘤患者的疗效。这项研究同样得到了RedHill的额外支持和NCI的资助。
  公司计划在2017年第三季度启动一项头颈癌患者黏膜炎的Ⅰb期研究，以评估YELIVA®作为放疗患者预防粘膜炎的放射防护剂的效果;2017年下半年将启动中度至重度溃疡性结肠炎患者Ⅱ期研究。

YELIVA®(Oncology)
YELIVA® (ABC294640) is a first-in-class, proprietary sphingosine kinase-2 (SK2) selective inhibitor, administered orally, with anti-cancer and anti-inflammatory activities, targeting a number of potential oncology, inflammatory and gastrointestinal indications.
YELIVA® inhibits SK2, a lipid kinase that catalyzes formation of the lipid signaling molecule sphingosine 1-phosphate (S1P). S1P promotes cancer growth, and proliferation and pathological inflammation, including TNFα signaling and other inflammatory cytokine production. Specifically, by inhibiting the SK2 enzyme, YELIVA® blocks the synthesis of S1P which regulates fundamental biological processes such as cell proliferation, migration, immune cell trafficking and angiogenesis, and are also involved in immune-modulation and suppression of innate immune responses from T cells. Preliminary evidence suggests that because of its specificity for targeting SK2, rather than SK1, YELIVA® may have a better therapeutic ratio than nonspecific sphingosine kinase inhibitors or those targeting only SK1.
RedHill acquired YELIVA® from U.S.-based Apogee Biotechnology Corp. in March 2015. Apogee previously completed numerous successful pre-clinical studies with YELIVA® in oncology, GI-inflammation and radioprotection models, as well as a successful Phase I clinical study in cancer patients with advanced solid tumors.
The final results from the Phase I study with YELIVA® in patients with advanced solid tumors confirmed that the study successfully met its primary and secondary endpoints, demonstrating that the drug is well tolerated and can be safely administered to cancer patients at doses that provide circulating drug levels that are predicted to have therapeutic activity. The study included the first-ever longitudinal analyses of plasma sphingosine 1-phosphate (S1P) levels as a potential pharmacodynamic biomarker for activity of a sphingolipid-targeted drug; administration of YELIVA® resulted in a rapid and pronounced decrease in levels of S1P with several patients having prolonged stabilization of disease.
A Phase II clinical study of YELIVA® for the treatment of multiple myeloma was initiated at Duke University Medical Center. The study is supported by a $2 million grant from the NCI Small Business Innovation Research Program (SBIR) awarded to Apogee in conjunction with Duke University, with additional support from RedHill.
A Phase II study with YELIVA® for the treatment of advanced hepatocellular carcinoma was initiated at  the MUSC Hollings Cancer Center. The study is supported by a $1.8 million grant from the NCI awarded to MUSC, intended to support a broad range of studies on the feasibility of targeting sphingolipid metabolism for the treatment of a variety of solid tumor cancers, including the Phase II study with YELIVA®, and will be further supported by additional funding from RedHill.
A Phase I/II clinical study evaluating YELIVA® in patients with refractory/relapsed diffuse large B-cell lymphoma and Kaposi sarcoma was initiated at the Louisiana State University Health Sciences Center in New Orleans. The study is supported by a grant from the NCI awarded to Apogee, with additional support from RedHill.
A Phase Ib clinical study to evaluate YELIVA® as a radioprotectant to prevent mucositis in cancer patients undergoing therapeutic radiotherapy is planned to be initiated.
A Phase II study to evaluate the efficacy of YELIVA® in patients with moderate to severe ulcerative colitis is planned to be initiated.
A Phase IIa clinical study with YELIVA® in patients with advanced, unresectable, intrahepatic and extrahepatic cholangiocarcinoma is planned to be initiated. YELIVA® was granted FDA Orphan Drug designation for the treatment of cholangiocarcinoma. 
The Phase I/II clinical studies, as well as the completed Phase I clinical study in cancer patients with advanced solid tumors, are registered on www.ClinicalTrials.gov, a web-based service by the U.S. National Institute of Health, which provides public access to information on publicly and privately supported clinical studies.