Jennerex授予FDA孤儿药指定为Pexa-VEC在肝细胞癌（HCC）

StephenW

Jennerex Granted FDA Orphan Drug Designation for Pexa-Vec in Hepatocellular Carcinoma (HCC)

Top-Line Data From TRAVERSE, Phase 2b Pexa-Vec Trial in Hepatocellular Carcinoma, Expected at Year End

SAN FRANCISCO, CA--(Marketwired - May 8, 2013) - Jennerex, Biotherapeutics, Inc., a private, clinical-stage biotechnology company focused on the development and commercialization of best-in-class targeted oncolytic immunotherapies for solid tumors, today announced that the United States Food and Drug Administration (FDA) granted orphan drug designation for Pexa-Vec (JX-594, pexastimogene devacirepvec) for the treatment of hepatocellular carcinoma (HCC, commonly referred to as liver cancer). Pexa-Vec, Jennerex's lead product candidate, is an oncolytic immunotherapy designed to 1) rapidly de-bulk tumors via tumor cell lysis, 2) induce a systemic anti-tumor immune response, and 3) selectively target tumor vasculature resulting in a rapid reduction in tumor blood flow. The Pexa-Vec clinical program in liver cancer includes TRAVERSE, a global Phase 2b trial in advanced HCC patients who have failed sorafenib therapy.

"We are pleased to receive FDA orphan drug designation for Pexa-Vec. This designation is an important step in the development of Pexa-Vec as a potential treatment for this lethal disease," said Laurent Fischer, M.D., president and chief executive officer of Jennerex. "We look forward to announcing top-line data from our TRAVERSE trial by the end of this year and to bring Pexa-Vec to the market as soon as possible."

The FDA provides orphan drug designation to drugs that seek to treat rare diseases or conditions for which there may be few adequate therapies. Orphan drug designation is intended to encourage companies to develop therapies for the treatment of diseases that affect fewer than 200,000 individuals in the United States. The designation will provide Jennerex with the opportunity of seven years of marketing exclusivity, grant funding to defray costs of clinical trial expenses, tax credits for clinical research expenses and potential waiver of the FDA's application user fee. Jennerex received orphan drug designation on Pexa-Vec for the treatment of HCC from the European Medicines Agency (EMA) in 2009.

Hepatocellular carcinoma is the fifth most common cancer worldwide and the third leading cause of cancer death, with over 600,000 new cases diagnosed annually resulting in more than 90 percent mortality(1) . The annual incidence rate in the U.S. is estimated to be 20,000 cases per year(2) . Currently, there are few approved treatment options for advanced HCC patients.

About Pexa-Vec

Pexa-Vec (JX-594, pexastimogene devacirepvec) is an investigational oncolytic immunotherapy designed to 1) rapidly de-bulk tumors via direct killing of tumor cells 2) induce a systemic anti-tumor immune response and, 3) selectively target tumor vasculature resulting in a rapid reduction in tumor blood flow. Pexa-Vec was engineered from vaccinia vaccine, which has been used for decades as a vaccine in healthy individuals. Pexa-Vec was also engineered to express GM-CSF, a white blood cell growth factor, which activates a systemic immune response to kill tumor cells throughout the body. Pexa-Vec exploits the unique characteristics of vaccinia, including its stealth extracellular envelope form, which allows the virus to survive in the bloodstream in the presence of neutralizing antibodies, leading to its ability to be administered both intravenously (IV) and intratumorally (IT). Unlike many targeted therapies that rely on a single target, Pexa-Vec is applicable to multiple solid tumor targets.

Pexa-Vec is currently being evaluated in an international, randomized Phase 2b clinical trial (TRAVERSE) in patients with advanced HCC who have failed sorafenib therapy. It is also being tested as monotherapy in sorafenib-naïve HCC patients and in combination with sorafenib. In addition, Pexa-Vec is being evaluated in a Phase 1-2 clinical trial in patients with treatment-refractory colorectal cancer as monotherapy and in combination with irinotecan, and in a Phase 2a clinical trial in treatment-refractory kidney cancer patients.

Phase 1 and Phase 2 clinical trials in multiple cancer types to date have shown that Pexa-Vec, delivered either directly into tumors or intravenously, induces tumor shrinkage and/or necrosis and is well-tolerated (over 250 patients treated to date). Objective tumor responses have been demonstrated in a variety of cancers including liver, colon, kidney, lung cancer and melanoma. Pexa-Vec has had a predictable and manageable safety profile to date which includes flu-like symptoms that typically resolve in 24 hours.

Pexa-Vec is the lead product candidate from Jennerex' SOLVE platform, a groundbreaking approach offering new therapeutic options for patients with life-threatening cancers that can be injected directly into tumor tissue or administered systemically by infusion.

Pexa-Vec is partnered in Europe with Transgene, a member of the Institute Merieux group, In South Korea with Green Cross and in China with Lee's Pharmaceuticals.

About Jennerex

Jennerex Biotherapeutics, Inc. is a clinical-stage biotechnology company focused on the development and commercialization of first-in-class, breakthrough targeted oncolytic immunotherapy products for solid tumors. The Company is focused on two main programs, lead product candidate, Pexa-Vec (JX-594, pexastimogene devacirepvec), which is in mid-stage clinical development for the treatment of advanced primary liver cancer and colorectal cancer and JX-929 which is under investigation for a variety of other solid tumors. Jennerex is headquartered in San Francisco and has related research and development operations in Ottawa, Canada and Busan, South Korea. For more information about Jennerex, please visit www.jennerex.com.

(1) http://www.who.int/mediacentre/factsheets/fs297/en/

(2) Ferlay J, Shin HR, Bray F, Forman D, Mathers C and Parkin DM. GLOBOCAN 2008 v2.0, Cancer Incidence and Mortality Worldwide: IARC CancerBase No. 10 [Internet]. Lyon, France: International Agency for Research on Cancer; 2010. Available from: http://globocan.iarc.fr.

StephenW

SAN FRANCISCO，CA  - （Marketwired  -  2013年5月8日） -  Jennerex，生物疗法公司，一个私人的，临床阶段的生物技术公司，专注于开发和商品化的同类产品中最佳的靶向溶瘤免疫治疗固体肿瘤，今天宣布，美国食品和药物管理局（FDA）授予孤儿药的Pexa-VEC（JX-594，pexastimogene devacirepvec），用于治疗肝细胞癌（HCC，通常简称为肝癌）。 pexa-VEC，Jennerex的铅候选产品，是设计为1的溶瘤细胞的免疫治疗）迅速去批量肿瘤通过肿瘤细胞裂解，2）诱导一个全身性的抗肿瘤免疫的反应，和3）选择性地针对肿瘤血管，导致迅速减少在肿瘤的血流量。 VEC Pexa在肝癌的临床方案包括导线，一个全球性的2b期试验中索拉非尼治疗失败的晚期肝癌患者。

“我们很高兴获得FDA孤儿药为Pexa-VEC这个指定是作为一个潜在的治疗这种致命的疾病Pexa-VEC的发展中的重要一步，说：”洛朗菲舍尔，MD，总裁兼首席执行官Jennerex。 “我们期待着宣布从今年年底我们的导线试验带来Pexa-VEC尽快以市场为顶线数据。”

FDA的孤儿药到寻求治疗罕见疾病或条件，而有可能是很少有足够的疗法的药物。孤儿药是为了鼓励企业开发疗法治疗的疾病，在美国影响少于200,000个人。 Jennerex指定将提供7年的独家销售权，补助资金支付的临床试验费用，税收抵免临床研究费用和潜在的豁免FDA的应用程序的用户费成本的机会。 Jennerex获得孤儿药在Pexa-VEC在2009年从欧洲药品管理局（EMA）用于治疗肝癌。

肝癌是第五个全球最常见的癌症和癌症死亡的第三大原因，有超过60万新发病例确诊，每年导致超过90％的死亡率（1）。在美国每年发病率估计为20000例，每年（2）。目前，也有少数经批准的治疗方案对晚期肝癌患者。

关于Pexa-VEC

Pexa-VEC（JX-594，pexastimogene devacirepvec），旨在通过直接杀伤肿瘤细胞的2 1）迅速散肿瘤溶瘤免疫治疗是一种调查）诱发全身性的抗肿瘤免疫反应，3）选择性针对肿瘤血管肿瘤血流量迅速减少。 pexa-VEC工程从牛痘疫苗，作为疫苗在健康个体中已经使用了几十年。 pexa-VEC也被设计来表达GM-CSF，白血细胞生长因子，激活一个全身免疫反应，杀死肿瘤细胞在体内。 Pexa-VEC利用牛痘的独有特性，包括其隐身外信封形式，这使得病毒生存在血液中的中和抗体的存在，导致它能够被管理静脉注射（IV）和肿瘤（IT） 。不像许多有针对性的治疗依赖于一个单一的目标，Pexa-VEC是适用于多种实体瘤目标。

Pexa-VEC目前正在评估国际，随机2b期临床试验（横向）与索拉非尼治疗失败的晚期肝癌患者。它也被索拉非尼的天真肝癌患者的单药治疗和联合索拉非尼测试。 -VEC Pexa此外，在1-2期临床试验，作为单药治疗和联合伊立替康治疗难治性大肠癌患者进行评估，并在阶段2a难治性肾细胞癌患者的临床试验。

第1阶段和第2阶段的临床试验表明，在多个癌症类型Pexa-VEC，直接交付到肿瘤或静脉注射，诱导肿瘤收缩和/或坏死和良好的耐受性（超过250名患者治疗）。客观肿瘤反应已被证明在各种癌症，包括肝脏，结肠，肾，肺癌和黑色素瘤。 Pexa-VEC有一个可预见的，可管理的安全性资料的日期，其中包括类似流感的症状，通常在24小时内解决。

Pexa-VEC Jennerex的解决平台，一个突破性的方法，可以直接注射到肿瘤组织或全身给药输注危及生命的癌症患者提供新的治疗选择是领先的候选产品。

-VEC Pexa在欧洲合作与转基因，研究所梅里埃集团的成员，在韩国绿十字和李氏大药厂在中国。

关于Jennerex

Jennerex生物疗法公司是一家临床阶段的生物技术公司，专注于开发和商业化的先入级，突破性靶向溶瘤免疫治疗固体肿瘤。该公司专注于两个主要项目，先导候选产品Pexa-VEC（JX-594，pexastimogene devacirepvec），在中期临床开发阶段，这是用于治疗晚期原发性肝癌和大肠癌和JX-929是根据调查多种其他实体瘤。 Jennerex是总部设在旧金山，在渥太华，加拿大和韩国釜山，并有相关的研究和开发业务。 Jennerex的更多信息，请访问www.jennerex.com。

（1）http://www.who.int/mediacentre/factsheets/fs297/en/

（2），布雷新HR Ferlay J，F，福尔曼ð，马瑟斯C和DM帕金。 2008 V2.0，发病率和死亡率全球GLOBOCAN：IARC CancerBase的第10号[互联网]。法国里昂的国际癌症研究署，2010年。可从：http://globocan.iarc.fr。

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