2014-9-25国内、国际新药信息汇总

　　

                      9月15日-24日CDE重要新药受理一览

                               
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                                        发布日期：2014-09-25  来源：大智慧阿思达克通讯社

下图为大智慧医药组根据国家食药监总局公开信息整理的9月15日-24日重要新药受理信息，按注册类型排序，供参考。

　　

                               
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礼来与阿斯利康合作开发阿尔茨海默病治疗药AZD3293

                               
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发布日期：2014-09-25  来源：新药汇  
礼来制药（NYSELY）与阿斯利康9月24日宣布达成协议，携手参与 AZD3293的研发和商品化工作，AZD3293是一种口服β分泌酶（BACE）抑制剂，目前正被作为潜在的阿尔茨海默病治疗药物进行研发。

                               
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阿尔茨海默病的进展以脑部淀粉样蛋白斑块的蓄积为特征。BACE 是与β淀粉样蛋白的形成有关的一种酶。抑制 BACE 预计可预防淀粉样蛋白斑块的形成，并最终延缓疾病的进展。

AZD3293是一种口服强效小分子 BACE 抑制剂，并已在I期临床试验中显示出可降低阿尔茨海默病患者和健康志愿者脑脊液中的β淀粉样蛋白水平。阿斯利康在2014年早些时候宣布了推动 AZD3293进入注册临床试验阶段的计划。

在此协议框架之下，礼来制药需支付阿斯利康最高达5亿美元的特定研发和注册里程碑的费用。两家公司将平摊今后所有的 AZD3293研发和商品化费用，同时在全球范围内分享利润、共担损失。

阿斯利康和礼来制药旨在迅速推动 AZD3293进入针对早期阿尔茨海默病患者开展的II/III期临床试验。礼来制药将主导临床研发工作，并与来自阿斯利康神经科学创新药物研究中心的研究人员合作，而阿斯利康则将负责药品的生产。两家公司将共同负责 AZD3293的商品化工作。

“礼来制药致力于阿尔茨海默病领域的科研历史已长达25年以上，我们致力于开发出能够改变和延缓这种危害严重疾病病程的新型治疗药物，”礼来制药高级副总裁、礼来生物制药业务部总裁 David Ricks 表示：“礼来制药的后续产品线包括潜在治疗药物、以及针对已知疾病标记物所开发的靶向性诊断试剂，通过与阿斯利康建立合作伙伴关系，这一后续产品线的发展将获得坚实的支持，阿斯利康是我们强有力的战略合作伙伴，并在为罹患这一危害严重的疾病的患者提供新型治疗药物方面与我们拥有同样的热情。上述合作联盟将使我们朝向既定的目标继续迈进，我们的目标是希望到2025年，阿尔茨海默病能成为一种可以被预防的疾病。”

“阿尔茨海默病是目前医学界所面临的最大挑战之一，BACE 抑制剂能靶向性针对该疾病进展的其中一项关键因素。我们期待能与礼来制药携手合作，作为我们的合作伙伴，礼来制药在阿尔茨海默病治疗领域作出了长期承诺并积累了丰富经验，”阿斯利康创新药物和早期研发部执行副总裁 Mene Pangalos 表示：“我们相信，通过将我们两家公司的科学实力相整合，并共同分摊晚期研发阶段的风险和支出，我们将能加速推动 AZD3293的研发进展，并凭借这一颇具前景的全新举措，加速为全球阿尔茨海默病患者提供治疗支持的进程。”

“此外，这一合作联盟还将使得阿斯利康能在未来将战略重点精准聚焦于核心治疗领域，同时充分利用外部合作，将我们日益增长的产品线领域的潜力发挥到最大。”Pangalos 补充道。

礼来制药将会支付5千万美元（税前）的起始里程碑偿付，或大约相当于每股0.03美元（税后），这些偿付将从2014年第三季度营收中予以扣除。

关于阿尔茨海默病

阿尔茨海默病是一种致命性疾病，也是最常见的一种痴呆类型，占所有痴呆病例的60%至80%。阿尔茨海默病将继续成为美国公众所面临的最重要的健康挑战，估计有5百万名65岁以上的美国人罹患阿尔茨海默病。如果在治疗方面缺少长足进步，阿尔茨海默病将不仅仅会影响到的处于老龄化过程中的战后婴儿潮这一代人的家庭，而且还会危及许多发达国家的医疗保健系统。在美国，每年与阿尔茨海默病直接相关的支出就达到2030亿美元，如果在影响疾病进展、延缓痴呆相关性失能方面再不有所作为，到2050年，这一数字有望增加到1万亿美元。

关于礼来制药

礼来制药是一家全球领先的、以创新改善人类健康水平的医药公司。礼来制药诞生于一个多世纪之前，公司创始人致力于生产高质量的药品、从而满足切实存在的医疗需求。今天，我们仍然执着于这一使命，并基于此开展工作。在全球范围内，礼来制药的员工们一直在努力开发能够改变人类生活的药品，并将其提供给到那些存在医疗需求的患者。礼来制药还致力于改善公众对于疾病的理解、并更好地开展疾病管理，同时通过投身于慈善事业和志愿者活动回馈社会。如果需要了解更多关于礼来制药的信息，请登录www.lilly.com 和 http://newsroom.lilly.com/social-channels。

关于阿斯利康

阿斯利康是一家以创新为驱动的全球性生物制药企业，专注于研发、生产和销售处方类药品，主要涉及心血管、代谢、呼吸、炎症、自身免疫、肿瘤、感染和神经科学领域。阿斯利康业务遍及一百多个国家和地区，其提创新药物已惠及全球数百万名患者。相关信息，请登录以下网址：www.astrazeneca.com

FDA授予阿斯利康肺炎单抗药物MEDI3902快速通道地位

                               
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发布日期：2014-09-25  来源：生物谷

MEDI3902是一种单抗药物，可有效预防和治疗绿脓杆菌感染，预防性用药有望成为医院获得性肺炎的新治疗方法。

                               
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阿斯利康（AZN）旗下全球生物制品研发部门MedImmune近日宣布，FDA已授予实验性单克隆抗体药物MEDI3902快车道地位（Fast Track designation），用于预防由绿脓杆菌（P.aeruginosa）导致的医院获得性肺炎（nosocomial pneumonia，NP）。绿脓杆菌是一种高度耐药性细菌病原体，能在住院患者中导致严重的疾病。FDA的快车道计划，旨在促进针对严重疾病的药物开发和快速审查，以解决严重未获满足的医疗需求。

MEDI3902是一种新颖的单克隆抗体，结合了3种独特的作用机制来杀灭绿脓杆菌。临床前试验中，在多种动物模型中，MEDI3902在预防和治疗绿脓杆菌感染方面均表现出强大的功效。MEDI3902的预防性用药，有望成为控制住院患者获得性肺炎的一种潜在的新治疗途径。

目前，阿斯利康正在探索利用生物制剂帮助预防和治疗极具挑战性的感染性疾病，包括绿脓杆菌。近期开展的临床前研究表明，针对耐药菌株，工程化多机制单抗药物不仅能够保护免受感染并可与轻微活性的抗生素产生协同作用，还具有预防多种细菌导致的共感染（co-infection）。

此外，阿斯利康也正在II期临床中调查另一种单抗药物MEDI4893，该药靶向金黄色葡萄球菌（S.aureus）α-毒素，金黄色葡萄球菌在住院患者中可导致严重的葡萄球菌感染。

绿脓杆菌（P.aeruginosa）是一种高度耐药细菌病原体，在住院患者及免疫功能受损的群体中可引发严重感染。绿脓杆菌是从住院时间超过一周的患者中分离到的最常见的病原体，同时也是导致医院内感染的最常见原因。据估计，在美国，每年发生5.1万例绿脓杆菌感染，在人工气道机械通气的患者中，绿脓杆菌的检出率高达24%，耐药菌株中所观察到的致死率高达43%。

英文原文：MedImmune Receives Fast Track Designation from FDA for Development of MEDI3902 for Prevention of Nosocomial Pneumonia

AstraZeneca announced today that its global biologics research and development arm, MedImmune, has received fast track designation from the US Food and Drug Administration (FDA) for its investigational monoclonal antibody (mAb) MEDI3902 for the prevention of nosocomial pneumonia caused by Pseudomonas aeruginosa (P. aeruginosa), a highly drug-resistant bacterial pathogen that causes serious disease in hospitalised patients. The FDA’s Fast Track programme is a process designed to expedite the development and review of drugs to treat serious conditions and fill an unmet medical need.

MEDI3902, currently entering Phase I clinical trials, is a novel mAb engineered to combine three distinct mechanisms of action for disarming and clearing P. aeruginosa. In pre-clinical trials, MEDI3902 was found to produce enhanced effects for both prevention and treatment of the problematic bacterial infection in multiple animal models. Prophylactic use of MEDI3902 will be investigated as a potentially new therapeutic approach for controlling pneumonia in hospitalised patients.

“We are pleased that the FDA has granted Fast Track designation for MEDI3902, recognising the unique science behind this investigational monoclonal antibody and the importance of accelerating development of new medicines that may help prevent serious bacterial infections, such as nosocomial pneumonia, rather than solely relying on antibiotics to treat them,” said Steve Projan, PhD, FAAM, Senior Vice President, R&D and Infectious Diseases & Vaccines iMED Head, MedImmune. “At a time when antimicrobial resistance poses an imminent and urgent global public health threat, it’s more important than ever to develop new therapies that both prevent and treat hospital acquired infections. The Fast Track designation will streamline communications with the FDA throughout the development process on what is a very different approach to the bacterial resistance problem. If successful, we hope to bring this important new medicine to patients as quickly as possible.”

MedImmune is exploring ways of using biologics to help prevent and treat challenging infectious diseases, including P. aeruginosa. Recent preclinical studies with P. aeruginosa have demonstrated that engineered multi-mechanistic mAbs may not only protect against infection and work synergistically with marginally active antibiotics against drug-resistant strains, they may also have potential to prevent co-infections with multiple bacteria. MedImmune is also studying MEDI4893, a Phase II clinical candidate mAb targeting Staphylococcus aureus alpha toxin, a bacterial pathogen that can lead to serious staph infections in hospitalised patients.

about MEDI3902

MEDI3902 is an investigational novel bispecific monoclonal antibody engineered to combine three distinct mechanisms of action. This novel antibody design was found to produce enhanced effects for both prevention and treatment of P. aeruginosa infection in multiple animal models.

about P. aeruginosa

P. aeruginosa is a highly drug-resistant bacterial pathogen that causes serious infections in hospitalized patients and/or with people with weakened immune systems. It is the most common pathogen isolated from patients who have been hospitalized longer than one week, and it is a frequent cause of nosocomial infections. An estimated 51,000 healthcare-associated P. aeruginosa infections occur in the United States each year. Pseudomonal infections are complicated and can be life-threatening. P. aeruginosa has been identified as the causative organism in up to 24% of pneumonias in mechanically-ventilated patients, with fatality rates as high as 43% observed in drug-resistant strains.

about MedImmune

MedImmune is the worldwide biologics research and development arm of AstraZeneca, a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialization of small molecule and biologic prescription medicines. MedImmune is pioneering innovative research and exploring novel pathways across key therapeutic areas, including respiratory, inflammation and autoimmunity; cardiovascular and metabolic disease; oncology; neuroscience; and infection and vaccines. The MedImmune headquarters is located in Gaithersburg, Md., one of AstraZeneca’s three global R&D centers. For more information, please visit www.medimmune.com.

about AstraZeneca

AstraZeneca is a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of cardiovascular, metabolic, respiratory, inflammation, autoimmune, oncology, infection and neuroscience diseases. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit: www.astrazeneca.com.

吉利德在日本提交丙肝鸡尾酒疗法LDV/SOF新药申请

                               
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发布日期：2014-09-25  来源：新药汇  

Sovaldi是丙肝明星药物，上半年销售突破58亿美元。近日，吉利德向日本提交丙肝鸡尾酒疗法LDV/SOF上市申请，临床中，该鸡尾酒疗法治愈率达100%。

                               
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吉利德（Gilead）近日宣布，已向日本药品与医疗器械管理局 (PMDA）提交丙肝鸡尾酒疗法LDV/SOF新药申请（NDA），寻求批准用于基因型1慢性丙型肝炎（HCV）成人患者的治疗，在临床试验中，LDV/SOF治愈率达到了100%。

鸡尾酒疗法LDV/SOF（ledipasvir/sofosbuvir，90mg/400mg）为每日一次的固定剂量复方片，其中ledipasvir（LDV）为NS5A抑制剂，sofosbuvir（SOF）为核苷酸类似物聚合酶抑制剂。如果获批，LDV/SOF将简化基因型1丙肝患者的临床治疗，仅需每日口服给药，而无需注射干扰素及联合利巴韦林（RBV）。

丙肝鸡尾酒疗法LDV/SOF新药申请（NDA）的数据包，包括一项在日本丙肝患者中开展的III期研究（GS-US-337-0113）的顶线数据。该项研究中，经LDV/SOF（无RBV）治疗12周后，初治治疗组（n=83/83，100%）和经治治疗组（n=88/88，100%）均实现了100%治愈率（SVR12），伴有肝硬化的丙肝群体（n=75/76）治愈率达99%。

在工业化国家中，日本的肝癌发病率最高，这主要是由于丙型肝炎病毒（HCV）感染所致。在日本，有超过100万丙肝患者，基因型1 HCV是最常见的毒株，约占70%-80%，该国当前基因型1 HCV感染的标准治疗方案，涉及长达48周的聚乙二醇化干扰素注射、口服RBV片剂其他药物，对于某些特定患者可能不适合。

LDV/SOF NDA的数据包，还包括另外3项III期研究（ION-1，ION-2，ION-3）的数据，分别评估了8周、12周、24周LDV/SOF方案治疗基因型1丙肝患者的疗效和安全性。数据表明，3项研究中，LDV/SOF（无利巴韦林）治疗组治愈率达到了94%-99%。

目前，丙肝鸡尾酒疗法LDV/SOF正在接受FDA和EMA的监管审查。今年6月，吉利德向日本PMDA提交了Sovaldi（sofosbuvir，SOF）的新药申请，寻求批准Sovaldi联合利巴韦林（RBV）用于基因型2 HCV的治疗。

英文原文：Gilead Submits New Drug Application to Japan’s Pharmaceutical and Medical Devices Agency for Fixed-Dose Combination of Ledipasvir/Sofosbuvir for Chronic Hepatitis C Genotype 1 Infection

-- 12-Week Course of Ledipasvir/Sofosbuvir Achieved 100 Percent Sustained Virologic Response (SVR12) in Japanese Phase 3 Study --

-- Ledipasvir/Sofosbuvir Simplifies Hepatitis C Treatment to a Single, Once-Daily Tablet --

FOSTER CITY, Calif.--(BUSINESS WIRE)--Sep. 24, 2014-- Gilead Sciences, Inc. (Nasdaq:GILD) today announced that the company has submitted a New Drug Application (NDA) to Japan’s Pharmaceutical and Medical Devices Agency (PMDA) for approval of an investigational once-daily fixed-dose combination of the NS5A inhibitor ledipasvir (LDV) 90 mg and the nucleotide analog polymerase inhibitor sofosbuvir (SOF) 400 mg for the treatment of chronic genotype 1 hepatitis C virus (HCV) infection in adults. The data submitted in the NDA, which include a Japanese Phase 3 study showing 100 percent SVR12 rates, support the use of LDV/SOF for 12 weeks in treatment-naïve and treatment-experienced patients with chronic genotype 1 HCV infection, including those with cirrhosis. Patients who achieve SVR12 are cured of HCV infection. If approved, LDV/SOF would simplify HCV treatment for genotype 1 patients in Japan to one daily tablet, eliminating the need for interferon and ribavirin (RBV).

Primarily due to HCV, Japan has one of the highest rates of liver cancer of any industrialized country. Of the more than one million people in Japan chronically infected with HCV, 70-80 percent are infected with the genotype 1 strain of the virus.

The NDA is based on data from a Phase 3 clinical trial conducted in Japan (GS-US-337-0113) among 341 treatment-naïve and treatment-experienced genotype 1 patients. In the study, 100 percent (n=83/83) of treatment-naïve and 100 percent (n=88/88) of treatment-experienced patients receiving 12 weeks of LDV/SOF without RBV achieved SVR12. Adverse events observed with LDV/SOF without RBV were generally mild and included nasopharyngitis (28 percent), headache (6 percent) and malaise (5 percent).

The NDA is also supported by SVR12 results from three Phase 3 studies (ION-1, ION-2 and ION-3) evaluating eight, 12 or 24 weeks of LDV/SOF among genotype 1 HCV patients. Trial participants included patients from the United States, Europe and Puerto Rico who were treatment-naïve or who had failed previous treatment, including protease inhibitor-based regimens, and also included patients with compensated cirrhosis. Trial participants in the ribavirin-free arms (n=863) achieved SVR12 rates of 94 to 99 percent.

LDV/SOF is currently under regulatory review in the United States and European unio.

On June 27, 2014 Gilead submitted an NDA to Japan’s PMDA for SOF as a single agent in combination with RBV for the treatment of genotype 2 HCV infection. SOF as a single agent has been approved by regulatory authorities in the United States, European unio, Australia and Canada under the tradename Sovaldi®.

LDV/SOF and SOF are investigational products in Japan and their safety and efficacy have not yet been established.

about Gilead Sciences

Gilead Sciences is a biopharmaceutical company that discovers, develops and commercializes innovative therapeutics in areas of unmet medical need. The company’s mission is to advance the care of patients suffering from life-threatening diseases worldwide. Headquartered in Foster City, California, Gilead has operations in North and South America, Europe and Asia Pacific.

FDA批准Celgene口服PDE4抑制剂Otezla治疗斑块型银屑病

                               
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发布日期：2014-09-25  来源：生物谷

乔布斯之死令抗癌药Abraxane名声大噪，今年，重磅药物Otezla连添2适应症，年销售峰值将突破20亿美元。

                               
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生物技术巨头新基（Celgene）在2013年凭借其明星药物Abraxane（注射用紫杉醇[白蛋白结合型]）着实风光了一把，该药是一种化疗药物，因乔布斯之死和癌中之王——胰腺癌适应症而名声大噪。今年，Celgene又收获了一枚重磅药物Otezla，该药6个月内接连收获2个适应症，与市售药物相比，Otezla具有无需监测及口服的巨大优势，业界预测，Otezla的年销售峰值将突破20亿美元。

新基（Celgene）近日宣布，口服药物Otezla（apremilast）获FDA批准，用于适合光疗和系统疗法的中度至重度斑块型银屑病（Plaque Psoriasis）成人患者的治疗。Otezla是一种口服、选择性磷酸二酯酶4（PDE4）抑制剂，该药是FDA批准的首个也是唯一一个用于斑块型银屑病治疗的PDE4抑制剂。

Otezla将为广泛的斑块型银屑病患者群体提供一种有价值的治疗选择，包括以前使用过生物制剂或常规系统性药物治疗的患者群体。银屑病（psoriasis）是一种由不受控免疫反应导致的皮肤慢性炎症性疾病，全球患者总数超过1.25亿人。

Otezla的获批，是基于2项研究（ESTEEM-1和ESTEEM-2）的主要疗效和安全性数据。这2项研究均为多中心、随机、双盲、安慰剂对照研究，在中度至重度斑块型银屑病（Plaque Psoriasis）成人患者中开展。研究中，Otezla使患者斑块型银屑病病情取得了显著且具有临床意义的改善。此前，FDA已于今年3月批准Otezla用于活动性银屑病性关节炎（PsA）成人患者的治疗。

Otezla销售峰值超20亿美元

业界认为，尽管面临着注射型药物肿瘤坏死因子（TNF）抑制剂的竞争，尤其是艾伯维的Humira（阿达木单抗）和辉瑞/安进的Entrel（etanercept），但Otezla用药不需要常规的实验室监测，且是一种口服药物，相比市售药物，Otezla具有巨大优势，将为患者和医生提供一种重要的治疗选择。

evaluatePharma此前预测，Otezla在2018年的销售额将达到12.19亿美元，而此次FDA批准Otezla新适应症，业界预期，Otezla的销售峰值有望突破20亿美元。

关于Otezla（apremilast）：

Otezla（apremilast）是一种口服小分子磷酸二酯酶(PDE4)抑制剂，在细胞内调控促炎症和抗炎介质的网络。PDE4是一种环磷酸腺苷(cAMP)特异性PDE，是炎性细胞中主要的PDE。PDE4抑制可提升细胞内cAMP水平，通过调控TNF-α、IL-23和其他炎性细胞因子的表达相应下调炎性反应。cAMP升高也会增加抗炎细胞因子，例如IL-10。

英文原文：Oral OTEZLA? (apremilast) Approved by the U.S. Food and Drug Administration for the Treatment of Patients with Moderate to Severe Plaque Psoriasis

In phase III studies, OTEZLA resulted in significant and clinically meaningful improvements in plaque psoriasis

OTEZLA demonstrated a consistent safety and tolerability profile across clinical trials

SUMMIT, N.J.--(BUSINESS WIRE)-- Celgene Corporation (NASDAQ: CELG) today announced that the U.S. Food and Drug Administration (FDA) has approved OTEZLA? (apremilast), the Company's oral, selecive inhibitor of phosphodiesterase 4 (PDE4), for the treatment of patients with moderate to severe plaque psoriasis for whom phototherapy or systemic therapy is appropriate. OTEZLA is the first and only PDE4 inhibitor approved for the treatment of plaque psoriasis. Psoriasis, a chronic inflammatory disease of the skin resulting from an uncontrolled immune response, affects more than 125 million people worldwide.

"OTEZLA offers an important new treatment option for patients whose symptoms are not adequately improving with their current treatments. In clinical trials, OTEZLA reduced redness, thickness, and scaliness of plaques in patients with moderate or severe plaque psoriasis," said Dr. M. Shane Chapman, Section Chief of Dermatology at Dartmouth-Hitchcock Medical Center. "Because the product labeling does not require routine laboratory monitoring, oral OTEZLA may be a welcome new option for patients and physicians looking for a different treatment experience."

The approval of OTEZLA was based primarily on safety and efficacy results from two multi-center, randomized, double-blind, placebo-controlled studies - ESTEEM 1 and ESTEEM 2 - conducted in adult patients with moderate to severe plaque psoriasis: body surface area (BSA) involvement of ≥10%, static Physician Global Assessment (sPGA) of ≥3 (moderate or severe disease), Psoriasis Area and Severity Index (PASI) score ≥12, and candidates for phototherapy or systemic therapy.

"OTEZLA offers a valuable treatment option for a spectrum of plaque psoriasis patients - patients who are treatment-na?ve as well as patients who are treatment-experienced, including those previously treated with biologic agents or conventional systemic agents," said Scott Smith, President Inflammation & Immunology for Celgene Corporation. "The FDA approval of OTEZLA for plaque psoriasis, together with the previous approval for psoriatic arthritis, reflects Celgene's commitment to extending the reach of our research and science in an effort to improve the lives of people worldwide living with chronic inflammatory diseases."

In the ESTEEM studies, OTEZLA treatment resulted in significant and clinically meaningful improvements in plaque psoriasis as measured by PASI scores at week 16. Clinical improvement as measured by sPGA scores of clear to almost clear were also demonstrated in both studies.

The safety of OTEZLA was assessed in 1,426 patients from three clinical trials. Side effects of OTEZLA were diarrhea, nausea, upper respiratory tract infection, tension headache, and headache. Before starting OTEZLA, patients should inform their doctor if they have a history of depression or suicidal behavior and if these conditions or other mood changes develop or worsen while taking OTEZLA. Patients taking OTEZLA should have their weight checked regularly.

"Psoriasis is a serious autoimmune disorder commonly associated with comorbidities," said Randy Beranek, president and CEO, National Psoriasis Foundation. "Effectively treating psoriasis is an important part of managing a patient's overall health. Having a new treatment like OTEZLA is important so patients can have more options and can work closely with their providers to find what works best for them."

OTEZLA? is available in the U.S. and is dispensed through a comprehensive network of specialty pharmacies. For more information about OTEZLA distribution and the exclusive treatment support services (including reimbursement assistance and 24/7 nurse support), doctors and patients can contact Otezla SupportPlus? at 1-844-4OTEZLA (1-844-468-3952) or visit www.OTEZLA.com for more information.

OTEZLA was approved on March 21, 2014 by the U.S. Food and Drug Administration (FDA) for the treatment of adults with active psoriatic arthritis. A New Drug Submission (NDS) for psoriatic arthritis was submitted to health authorities in Canada in the second quarter of 2013. A NDS for psoriasis in Canada as well as a combined psoriatic arthritis/psoriasis Marketing Authorization Application (MAA) in Europe were all submitted to health authorities in the fourth quarter of 2013.

加拿大Tekmira药厂抗埃博拉药TKM-Ebola获FDA批准人体试验

                               
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发布日期：2014-09-25  来源：Reuters  浏览次数：0

9月22日，加拿大制药商Tekmira Pharmaceuticals(NASDAQ：TKMR)宣布，美国食品药品管理局(FDA)已批准其研制的一款实验性药物在紧急情况下供已经证实或怀疑感染埃博拉病毒的患者使用。

                               
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Tekmira Pharmaceuticals Corporation's head office is pictured in Burnaby, British Columbia August 5, 2014.

    早在今年7月30日向美国联邦食品药物管理局 (FDA)请愿，要求FDA尽快批准一种由加拿大Tekmira药厂研发的新药TKM-Ebola，这种药物被认为对于治疗埃博拉有特效。

其对杀死灵长类动物身上的埃博拉病毒相当有效，2014年初开始第一阶段人类临床试用。

但2014年7月，FDA 宣布禁止临床试用，理由是试用是以志愿者为对象的。而事实上，14位参与试用的对象都安全无恙。

世界卫生组织22日发布最新疫情通报称，在西非多国蔓延的埃博拉疫情已经造成2793人死亡，另有5762人被病毒感染。其中利比里亚的疫情仍十分严重，该国已有1578人死于此病，另有3022人感染。

U.S., Canada allow emergency use of Tekmira's Ebola treatment

(Reuters) - Canadian drugmaker Tekmira Pharmaceuticals Corp (TKM.TO)(TKMR.O) said on Monday that U.S. and Canadian regulators have authorized the use of its Ebola treatment in patients who have confirmed or suspected infections from the deadly virus.

The Vancouver-based company said its treatment, TKM-Ebola, has been administered to patients on an emergency basis and the repeat infusions have been well-tolerated.

The drug was administered to Rick Sacra, a doctor who contracted the virus in West Africa, and who has shown promising signs, the Nebraska Medical Center said in a statement.

TKM-Ebola, an RNAi therapeutic, works by preventing the virus from replicating.

Expanded access protocols, authorized by the U.S. Food and Drug Administration and Health Canada, allow drug developers to offer experimental therapies to patients with serious diseases who cannot participate in controlled clinical trials.

Tekmira Chief Executive Officer Mark Murray said the company's supplies of the treatment are limited.

The company is developing TKM-Ebola under a contract with the U.S. Department of Defense.

Tekmira's shares closed 15 percent up at C$26.01 in Toronto and up 17 percent at $23.61 on the Nasdaq.

BioCryst获NIAID额外资金支持研发治疗出血性病毒感染药BCX4430

                               
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发布日期：2014-09-25  来源：globenewswire  

BioCryst Pharmaceuticals已获得美国国立变态反应与感染性疾病研究所200万美元的资金支持，用以继续研究治疗出血性病毒感染疾病的药物--BCX4430。

BioCryst Pharmaceuticals已获得美国国立变态反应与感染性疾病研究所（National Institute of Allergy and Infectious Diseases，NIAID）200万美元的资金支持，用以继续研究治疗出血性病毒感染疾病的药物--BCX4430。

根据与BioCryst达成的相关协议，NIAID已针对BioCryst的两项产品--GMP级BCX4430原料药及制剂产品--行使了相关责任和权利。目前，BioCryst已收到用于研发BCX4430的200万美金支持，BCX4430用于治疗包括埃博拉病毒和马尔堡病毒病在内的出血热病毒感染。如果所有的支持资金到位，整个协议的资金规模预计达2630万美元。

该项目的全部或部分经费是由来自美国NIAID、NIH（National Institutes of Health）、DHHS（Department of Health and Human Services）的联邦资金所提供的。

BioCryst的BSAV研究计划的目的是开发用于治疗对人民健康和国家安全构成威胁的病毒的、广谱的、注射和口服药物。

BCX4430是由BioCryst与美国政府机构在动物使用规则监管办法（Animal Rule regulatory pathway）下合作开发的。

BCX4430是BSAV化合物中较为重要的一个，是一种RNA依赖型RNA聚合酶的抑制剂，对9个家族超过20种的RNA病毒显示了广谱的抗病毒活性，包括脊髓灰质炎病毒、披膜病毒、布尼亚病毒(bunya viruses)、沙粒病毒、副粘病毒、冠状病毒、黄病毒。

BioCryst Receives Additional NIAID Funding for Manufacture and Development of BCX4430 to Treat Hemorrhagic Virus Diseases

RESEARCH TRIANGLE PARK, N.C., Sept. 18, 2014 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced that the National Institute of Allergy and Infectious Diseases (NIAID) has exercised two additional options under its contract, which provides for GMP drug substance and drug product manufacture of BCX4430. Exercise of these options includes up to a $2.0 million increase to the existing development contract.

The contract modification and the two additional options exercised represent up to $4.0 million to BioCryst in order to accelerate the development of BCX4430 as a treatment for hemorrhagic fever viruses, including Ebola virus and Marburg virus disease.  NIAID, part of the National Institutes of Health, granted this five year term contract to BioCryst in September 2013.   Approximately $19.9 million of funding has been awarded to date under the contract. With this additional award, the BCX4430 development contract has been increased in value to $26.3 million, if all options are exercised.

This project is funded in whole or in part with Federal funds from the National Institute of Allergy and Infectious Diseases, National Institutes of Health, Department of Health and Human Services, under Contract No. HHSN272201300017C.

about the BSAV Program & BCX4430

The objective of BioCryst's BSAV research program is to develop broad-spectrum parenteral and oral therapeutics for viruses that pose a threat to health and national security. The lead BSAV compound is BCX4430, an RNA dependent-RNA polymerase inhibitor that has demonstrated broad-spectrum activity against more than 20 RNA viruses in nine different families, including filoviruses, togaviruses, bunyaviruses, arenaviruses, paramyxoviruses, coronaviruses and flaviviruses. BioCryst is developing BCX4430 in collaboration with U.S. Government Agencies following the Animal Rule regulatory pathway.

about BioCryst Pharmaceuticals

BioCryst Pharmaceuticals designs, optimizes and develops novel small molecule drugs that block key enzymes involved in rare diseases. BioCryst currently has several ongoing development programs: oral inhibitors of plasma kallikrein for hereditary angioedema, including BCX4161 and several second generation compounds; peramivir, a viral neuraminidase inhibitor for the treatment of influenza, and BCX4430, a broad spectrum viral RNA polymerase inhibitor. For more information, please visit the Company's website at www.BioCryst.com.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst's actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: that BioCryst or its licensees may not be able to enroll the required number of subjects in planned clinical trials of its product candidates and that such clinical trials, may not be successfully completed; that the Company or its licensees may not commence as expected additional pre-clinical studies or human clinical trials may not be commenced as expected or such studies may not be successfully completed; that the FDA may require additional studies beyond those planned for BCX4430, or may not provide regulatory clearances which may result in delay of planned clinical trials, or may impose a clinical hold BCX4430, or withhold market approval for BCX4430; that the Company may not be able to obtain additional funding for BCX4430; that government funding or other contracts for BCX4430 may have certain terms and conditions, including termination provisions, that subject the Company to additional risks; that the Company may lose current funding for the program; that the Company may never file an IND for BCX4430; that its actual financial results may not be consistent with its expectations, including that 2014 operating expenses and cash usage may not be within management's expected ranges. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst's most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and current reports on Form 8-K, all of which identify important factors that could cause the actual results to differ materially from those contained in BioCryst's projections and forward-looking statements.

诺和诺德糖尿病复方新药Xultophy获欧盟批准

                               
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发布日期：2014-09-25  来源：pipelinereview

诺和诺德（Novo Nordisk）糖尿病复方新药Xultophy近日获得了欧盟委员会的批准，公司准备在2015年上半年使Xultophy在欧盟上市。

                               
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Xultophy（原名IDegLira）是每日只需注射一次的糖尿病复方制剂，其中包含了丹麦制药公司——Novo Nordisk的Tresiba（insulin degludec，德谷胰岛素）和市场上销售领先的GLP-1类似物Victoza（liraglutide，利拉鲁肽）。

新产品Xultophy主要与口服降血糖药物联合用于成人2型糖尿病患者的治疗，以帮助患者在单独或联合使用基础胰岛素不能产生足够药效时更好地控制血糖。

Novo Nordisk首席科学家MadsKrogsgaard Thomsen讲到，“我们相信，Xultophy代表着一种改变2型糖尿病治疗模式的趋势，同时我们希望能为欧洲的2型糖尿病患者提供该药”。

欧盟对Xultophy的批准是基于其一项后期研究结果，该研究显示Xultophy可在避免胰岛素治疗的常见副作用的同时，降低血糖水平。

日本早在2012年就已批准了Xultophy，而瑞士也在本月早些时候批准了Xultophy。

Xultophy此次在欧盟的获批与其在美国的命运形成鲜明的对比，目前FDA还未批准Xultophy中的活性成分——Tresiba，这不仅推迟了Novo Nordisk的单方制剂Tresiba和复方制剂Xultophy在美国的上市，也推迟了另一种含有Tresiba的复方制剂Ryzodeg在美国的上市时间。

Novo Nordisk A/S: Xultophy® (IDegLira) approved in Europe

Novo Nordisk today announced that the European Commission has granted marketing authorisation for Xultophy® for the treatment of type 2 diabetes mellitus in adults. The authorisation covers all 27 European unio member states.

Xultophy® is the brand name for IDegLira, the first once-daily single injection combination of insulin degludec (Tresiba®) and liraglutide (Victoza®). Xultophy® is indicated for the treatment of adults with type 2 diabetes mellitus to improve glycaemic control in combination with oral glucose-lowering medicinal products when these alone or combined with basal insulin do not provide adequate glycaemic control.

“We believe that Xultophy® represents a new paradigm with the potential to transform how type 2 diabetes is treated. We look forward to making the product available to people with type 2 diabetes in Europe,” said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk.

Xultophy® was approved in Switzerland on 12 September 2014. Novo Nordisk expects to launch Xultophy® in the first European countries in the first half of 2015.

about Xultophy®

Xultophy® is a once-daily, single injection combination product consisting of insulin degludec (Tresiba®), a once-daily basal insulin analogue with an ultra-long duration of action, and liraglutide (Victoza®), the once-daily human GLP-1 analogue.Xultophy® has shown consistent results in improving glycaemic control in insulin-naïve people with type 2 diabetes as well as those uncontrolled on basal insulin.

For people uncontrolled on basal insulin therapy, Xultophy® has demonstrated a significant reduction in HbA1C of 1.9% with a mean weight loss of 2.7 kg and a low rate of hypoglycaemia comparable to that of insulin degludec.

Novo Nordisk submitted the application for marketing authorisation for Xultophy® in the EU on 31 May 2013.

澳大利亚Yaz公司开发可让女性1年只有3次月经的新药Yaz Flex

                               
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发布日期：2014-09-25  来源：澳大利亚《每日电讯报》  

据报道，澳大利亚科研人员研发了一种能让女性一年只来三次月经的药，这种药不会损害女性的生育能力，长期服用兼具避孕功效，当女性想要宝宝时只要停药即可。从9月24日起，这种药开始在澳大利亚各大药店出售，在澳大利亚女性中引起轰动，不少女性表示愿意尝试。

                               
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这种药的学名是Yaz Flex，前身是一种叫做Yaz的避孕药。和大多数避孕药一样，Yaz不能长期连续服用，药里含有的高剂量雌激素会让女性失去受孕能力。后来，澳大利亚一家药品研发公司对Yaz进行了研发提升，把它开发成一种能抑制月经的新药。德国拜尔承担了这种药的生产任务。

Yaz Flex由一个巴掌大小的药盒装着，这个看上去不起眼的盒子具有计数和控制药片分发的能力，每盒有120片，每天只吐出3片药，服药者不能多吃，同样，少吃一片也不行，药盒的自动报警功能会滴滴响，直到使用者乖乖吃药。

澳大利亚新州家庭规划部门的医药主管狄波拉·贝特森博士称赞这种药“帮女性减少了很多顾虑”。他说，“自己决定月经的次数让女性少了很多烦恼，我们做过的万人问卷调查显示，超过70%的女性愿意服用这种药。”狄波拉说，减少月经次数对女性没有害处，还能保护女性的生育能力。

“女性一生平均有几百次月经，每次月经过后都会让子宫壁变薄，直到月经消失，当然，月经消失后女性也就基本失去了生育能力。Yaz Flex把女性每年的月经次数减少，在一定程度上拉长了女性一生的月经周期，延长了女性的生育年龄。对那些一心扑在事业上没空要孩子的中年女性来说，Yaz Flex的问世是个好消息。”

新州一家公立医院的妇产科教授罗德·巴博尔也认为Yaz Flex是种不错的药。“很多国家的医学研究证实，减少女性的月经次数是安全的，但一直没有一种受认可的办法帮女性减少月经，Yaz Flex提供了不错的参考。”

服用Yaz Flex也有副作用，不过都在可控范围内，比如会引起头痛、恶心、胸闷、抑郁、不规则出血或血凝块，但医学数据表明这些副作用发生的概率极低。

　A NEW contraceptive pill that lets women go four months without a period will go on sale this week。

　　Yaz Flex comes in packs of 120 pills and women who use it continuously will have three periods a year。

　　The pill comes with a palm-sized dispenser that records usage, notices when a tablet has been missed and provides an alert that alternative contraception must be used。

　　The Clyk digital tablet dispenser can be programmed to sound a daily alarm to remind women to take the pill。

　　More than 80 per cent of women admit to forgetting to take their contraceptive pill at some stage and 31 per cent forget it once a month, according to a survey funded by Bayer, the drug company that makes Yaz。

　　Alex Trevor, who has used the new pill for a week, loves the new digital dispenser which "keeps me on track". "There have been times when I've forgotten to take the pill in the past," she said。

　　Yaz Flex is a new version of the existing monthly contraceptive pill Yaz and both contain a combination of hormones that over time help reduce acne. Australia is the launch market for the pill, which is made in Germany。

　　Associate professor and gynaecologist Dr Rod Baber said many medical studies had found skipping periods was safe and it could be useful for women who have heavy periods, their periods are painful or they get migraine associated with menstruation。

　　Ms Trevor, whose mother Dr Christine Read works for Bayer, said she had skipped periods on her old pill but Yaz Flex with its digital dispenser made it easier to keep track of wher she was in her cycle。

　　Common side effects of Yaz Flex include nausea, depression, headache, breast pain and unscheduled bleeding。

　　Women using it also face twice the risk of a blood clot as women using older versions of oral contraceptives, but Dr Baber said the risk was still low - 2.7 in 100,000 women. In pregnancy the risk of a blood clot is six in 100,000.

美国百特斥巨资购买Merrimack胰腺癌新药MM-398

                               
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发布日期：2014-09-25  来源：seekingalpha

美国百特9月24日宣布，公司将斥资购买Merrimack Pharmaceutical旗下胰腺癌新药MM-398。MM-398是一种纳米脂质体密封的伊立替康。该药物被美国食品药品监督管理局（FDA）和欧洲药监局授予孤儿药称号。

　　

                               
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　　根据双方签署的协定，美国百特将会首批支付Merrimack公司1亿美元，后者将会在新药第一个和第二个适应症研究和批准达到预期目标之后额外获得总计4亿美元。

　　除此之外，Merrimack公司在额外拓展MM-398两个适应症之后获得2.2亿美元，并在产品上市之后获得2.5亿美元销售分成。

　　收购完成后，美国百特将拥有MM-398在除美国和台湾之外所有地区的使用权。Merrimack Pharmaceutical负责美国市场，而台湾地区则归PharmaEngine公司所有。

　　Merrimack公司计划在2014年向FDA提交MM-398的新药申请。百特将会在2015年启动其他地区新药申请。

Merrimack Licenses MM-398 - Value Potential Dramatically ImprovesSummary

• Merrimack announces a license agreement with Baxter for MM-398.
• I amend my bear thesis to "hold" given that the arrangement helps validate the legitimacy of MM-398 for pancreatic cancer.
• This partnership deal comes rather expected. In my initial report, I emphasized that MM-398 could become the standard of care for pancreatic cancer.
  

In early July, I wrote a bear article on Merrimack (NASDAQ:MACK), with a thesis stating that the company's prospects could be limited. In highlighting the implications of Sanofi's (NYSE:SNY) recent action to sever ties to MM-121, I argued that MM-121's poor clinical success rate could have prompted Sanofi to conclude the license agreement because it anticipates further clinical failure. Deeper under the hood, Merrimack's remaining assets are still in early stage trials, which, in fact, have an inherently low success rate. However, the company announced today a license agreement with Baxter (NYSE:BAX) for MM-398, sending shares as much as 27% higher in pre-market trading. As a result, I amend my bear thesis to "hold" since Merrimack is on course to continue developing and eventually commercialize MM-398 under a lucrative partnership arrangement.

Some key highlights of the license agreement with Baxter are as follows:

• Baxter agrees to develop and market MM-398 outside the United States.
• Merrimack will receive $100 million upfront from Baxter in the current quarter and it now has the opportunity to unlock $120 million in milestone payments.
• Merrimack could receive another $280 million in development and milestone payments from Baxter if MM-398 shows potential in a second pancreatic cancer indication.
• The approval process for MM-398 is currently underway. Baxter anticipates regulatory approval submissions outside the United States in 2015.
• Merrimack retains commercial rights to the U.S. and Taiwan.
  

I have amended my bear thesis on Merrimack to "hold" for reasons two-fold. First, I expect that Merrimack will benefit from enhanced earnings growth going forward into next year. For in addition to the $120 million in milestone payments for the initial indication of MM-398, the company established a lucrative $280 million auxiliary agreement if the compound shows promise in a second pancreatic cancer indication. Merrimack will also receive tiered royalties and $100 million upfront from Baxter, which should benefit earnings for Q3 2014. Second, I maintain that MM-398 could become the standard of care for pancreatic cancer, which resembles a $1.2 billion market opportunity going into 2015. I believe Merrimack's ability to license the drug to a proven winner in Baxter demonstrates the drug's viability. However, I continue to be skeptical about the viability of Merrimack's other cancer treatment in MM-121. As a reminder, Sanofi spent in upwards of $400 million developing MM-121 before walking away from it. Thus, until we learn more about the conditions of the termination, as well as Merrimack's plans to acquire another partner, I will maintain coverage at "hold."