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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。
LY）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。
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
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（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.
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.
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。
(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 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 DiseasesRESEARCH 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.
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.
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。
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。
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:
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."
此次灵北制药一共提交7项II和III期临床研究，其中3项为精神分裂，4项为重度抑郁症的辅助治疗，共有超过6000名患者参与。
精神分裂适应症的2个III期临床研究共有超过1200名患者参与。患者在接受治疗6周之后的阳性和阴性症状量表（PANSS）分数有大幅度的提升。PANSS为精神疾病病的评定工具。
在重度抑郁症的辅助治疗方面，brexpiprazole在II项临床III期试验中表现也要优胜于安慰剂。
灵北制药和大冢制药共同开发的brexpiprazole是一款实验性血清素-多巴胺活动调节剂(SDAM) ，可作用于多巴胺D2和5-HT2A受体。
我原来以为我是第一个这样认为的，但是，在写作本文的过程中，才发现根本不是。百济神州的两位创办人都通过媒体明确表达了要做中国的基因泰克的决心。百济神州的创办人只有两位，除了大名鼎鼎的王晓东外，另外一位是John Oyler，中文名为欧雷强（曾用中文名何台庆）。汤森路透旗下杂志Bioworld Today在去年曾经对John Oyler有一个深度访谈报道，该报道的标题就是“John Oyler Aims To Build China’s Genentech via Beigene”（John Oyler旨在通过BeiGene打造中国的Genentech）【1】。几乎在同时，王晓东也对中国的《医药经济报》记者说“以百济神州为代表的本土生物制药公司，必须进一步发展，当其经济实力能支持不以短期回报为目的的原创科研，就能成为中国的基因泰克”【2】。那么，百济神州的两位创办人有何底气来向世界宣称要做中国的基因泰克？百济神州能否做到？本文就试图谈谈自己的一家之言，也想藉此引起大家对该公司的关注，毕竟像百济神州这样的致力于研发创新原研药的中国本土公司并不多，事实上，不是“不多”，是很少。
毫无疑问，百济神州和基因泰克有很多不同之处，但是两家公司至少有两个共同之处。第一：两家公司的英文名都含有“Gene”（基因）, 百济神州的中文名尽管和基因不沾边，中文名却是Beigene(=Beijing+gene?)，事实上，就全球而言，含有(或部分含有)“Gene”的著名生物制药公司，除了Genentech(基因泰克)外，还至少有Amgen（安进）和Genzyme(健赞)等（更多公司信息，可参见我的另一博文：全球10大拥有好“基因(gene)”的生物制药公司）。其实，尽管有人说百济神州的中文名起的的不好，应该叫北京基因等。但是，我却觉得百济神州的名字取得非常好，显然也是王晓东当初深思熟虑过的，对百济神州的中文名字，我是这样理解的：百=很多，想方设法，济=悬壶济世，神州=神州大地=中国，连在一起就是：竭尽全力为中国造好药，造福、救治中国病人。我不知道当初王晓东是否对公司名也有此意，但是我的上述解释似乎不无道理。；2）百济神州和基因泰克都专注于抗癌药的研发（下文还会详及）。
当然，上述两点简单的共同之处并不能保证百济神州成为中国的基因泰克，那么是什么原因或者因素才会使其有望成此大愿呢？我个人认为，是如下的“三高”。下面谈谈是哪“三高”。
第一：起点高。百济神州的起点高表现在许多方面，但是最主要的还在于其创办人的水平高。王晓东的学术影响力就无需多言了，简单说两个头衔: 美国科学院院士，北京生命科学研究所（NIBS）所长, 另外，他自己的研究领域也和癌症乃至抗癌药密切相关。相信大多数科学网网友不知道的是，王在创办百济神州之前，还曾在美国创办了Joyant Pharmaceuticals公司(一家肿瘤生物技术公司)。最近在新闻中看到，王晓东和施一公、饶毅被人并称为“三剑客”，但是，这三位牛人却没有人是真正的“剑”客，但至少有两人（尤其是饶）是“博”客。他们三人有许多共同之处，但是也各有特点：三人中，只有王没有在科学网开博客，只有饶没有开公司的经验，只有施两者都干过。闲话少叙，百济神州的另外一位创办人John Oyler能和王晓东联手自然也不是泛泛之辈，在创办百济神州前，这位地道的美国人就曾经创办了多家公司并任CEO，其中包括总部在北京的保诺科技公司(BioDuro)，后以7700万美元的价格出售给了著名的CRO公司PPD。John Oyler丰富的创办和管理公司的经验和王晓东的超强的学术水平和影响力可以说是珠联璧合，两人的合作是百济神州成功的重要保证，百济神州今天的成功可以说是赢在了起跑线上。
第二：研发策略高。百济神州尽管创办才仅仅4年，目前也只有约150人，但是，其研发管线已经有10种实验性药物（均为创新原研药），其中已经有3种已经推进到了临床试验阶段。新药研发是一个非常烧钱的行业，短短4年能做到如此佳绩，百济神州的研发策略当然有高明之处。根据我粗浅的了解，这个策略我简答的归纳为“买进卖出”。对于一个刚创办的公司而言，如果想靠从头寻找新的药物靶点，这种漫长而风险很高的生意，没有哪个投资人会钱多的没有地方化来让别人烧的。因此买进别的公司不被看好，或已经放弃的新药项目就是一个很重要的策略了，当然寻找合适的卖方和合适的项目就要考验公司决策人的水平了。比如在2011年百济神州就从杨森引进了两个临床阶段肿瘤化合物（一个单克隆抗体药和一个小分子化学药）【3】，这个买卖也引起了包括业内知名媒体如GEN的关注【4】。
当然，如果只“买进”，不“卖出”，短时间内又不能有新药上市销售，公司很快就会资金链断裂。百济神州自己没有上市新药，拿什么卖呢？其实这个“卖”也是国际制药界普遍采用的模式，实际是就是一种合作。真正使百济神州声名鹊起、变身业内高富帅的，还是2013年11月与默克雪兰诺(Merck Serono)做成的一桩大买卖，按照协议，默克雪兰诺将获得百济神州的在研药物BGB-290（PARP抑制剂）在中国以外市场的开发权，百济神州负责该药在中国的开发和商业化，并有望获得最高达1.7亿欧元（=2.32亿美元）的收益，这还不算如果该药上市后的销售提成【5】。这是中国大陆本土药企与跨国制药公司签订的最大协议，开创了跨国药企与中国本土企业在创新药研发的成功先例，这是双方继2013年5月合作开发抗癌新药BGB-283(BRAF)抑制剂之后的再度携手。BGB－283和BGB－290是由百济神州自主研发的靶向型小分子抗癌药，两者目前都已经进入临床试验阶段，这两个项目的首笔里程碑成果收入分别在今年的5月和9月已经到账，分别为500万和900万美元，也就是说短短几个月，百济神州已经有1400万美元的收入。如果不算香港的和记黄埔（Hutchison Medipharma), 这也是中国史上第二个类似合同，此前，第一个是中科院上海生科院在2010年转让给法国赛诺菲的一种蛋白抗肿瘤药物，合同金额是6000万美元【6】。按照国际惯例，上面几个合同都不是一次付清的，是根据试验进展分期付款的，中科院上海生科院最后拿到手多少钱，2010年后未见媒体公开报道。但是1400万美元，百济神州却是已经到手了。
百济神州研发策略高还表现在项目选的好，比如BGB-290项目，这种PARP抑制剂之所以能受百济神州和默克雪兰诺的青睐，还是有原因的，网上已经有人做了深度分析【7】，不在赘言。对于百济神州的三个在研单抗药，有一个药的靶点没有透露，另外两个的靶点分别为PD-1和PD-L1，这两个基于免疫检查点的新靶点现在是跨国制药公司的竞争热点，也是国际生物制药的前沿领域，有两个PD-1抗体已经分别在日本和美国获批上市，而PD-L1抗体目前全球尚没有已经上市的（参见我的另一博文：抗癌新药最火的分子靶点－兼议肿瘤免疫治疗）。今年9月3日，百济神州和德国的勃林格殷格翰(BI)宣布，签署战略合作协议，将由BI为百济神州自主研发的免疫肿瘤新药单抗PD-1临床试验提供符合cGMP标准的抗体【8】，估计明年百济神州的PD-1就可以开始临床I期试验。我国本土也有多家公司和科研单位研发PD-1和PD-L1抗体，但目前没有一种进入临床试验阶段，百济神州PD-1是目前研发进度最快的了。百济神州和之所以和德国的BI合作估计也是无奈，我国目前抗体生产代工水平（达到发达国家监管标准）还很低，目前中国本土企业，只有药明康德能干这活，今年5月，药明康德为中裕新药生产的艾滋病新药Ibalizumab获得美国FDA批准用于新药临床试验，这是目前已知国内生产的无菌生物产品首次获得美国FDA认可，用于经美国FDA批准的新药临床试验，具有里程碑意义【9】。
第三：国际化水平高。百济神州的国际化水平在中国本土的制药企业中恐怕是最高的，除了创办人和老总是美国人之外，该公司另外一位高管Steven Young也是一位美国人，他现任该公司药物化学代理首席总监，他此前默克研究实验室（West Point）副总裁及药物化学首席总监。我个人推测， Steven Young这个工作经历也许对于百济神州和默克的合作起到了重要作用。由于百济神州有三个药在临床试验阶段，有更多的药即将进入临床，所以亟需临床试验方面的管理人才，所以今年8月底两位在临床开发和药政事务方面分别很有经验的副总加盟了百济神州。两人都有较长的在跨国药企的工作经验，两人跳槽到百济神州不但说明该公司的吸引力在快速增强，也说明百济神州可以提供具有国际竞争力的薪水待遇。另外，更重要的是，从两人的简历来看，两个新高管和百济神州两位创办人没有多大的直接关系，而公司目前其他的总监以上的管理团队基本都是John Oyler和王晓东此前所创办的公司的旧部。所以，我个人认为，这两位高管的加盟对于百济神州的管理而言具有里程碑意义。百济神州管理团队的国际化也决定它其他方面的国际化，比如与跨国公司的合作，在澳大利亚进行的临床试验等。
没有人能随随便便成功，一个公司尤其是生物制药公司更是不可能随随便便成功，百济神州的成功法宝肯定还有许多，本文意在抛砖引玉。今年初，有人总结了生物制药公司成功创业所需要六大要素，其中也百济神州和基因泰克也都被做为典型公司进行了分析【10】。本人不才，只能简单了列了上述的“三高”，当然，百济神州最终的成功还要靠自己的药的疗效高，作为普通人，我们“不看广告，看疗效”。
主要参考资料：
John Oyler Aims To Build China’s Genentech via Beigene，http://www.beigene.com/zh-cn/pdf/BWT%20112213%20Biopeople.pdf
生物创新药跨国合作“瓶颈”待破，http://web.yyjjb.com:8080/html/2013-11/18/content_201603.htm
百济获得授权引进两个临床阶段肿瘤化合物，http://www.beigene.com/zh-cn/pdf/2011-10-14.pdf
BeiGene Inks Co-Development and Licensing Deals for Two of Janssen’s Anticancer Candidates,http://www.genengnews.com/gen-news-highlights/beigene-inks-co-development-and-licensing-deals-for-two-of-janssen/81245823/?kwrd=BeiGene
Merck Serono, BeiGene Pursue Second Cancer Compound in $232M+ Deals,http://www.genengnews.com/gen-ne ... -232m-deal/81249109
赛诺菲出手四亿：上海生科院卖掉“重磅炸弹”，http://edu.qq.com/a/20101202/000152.htm
百济神州PARP抑制剂进展顺利，默克雪兰诺支付首笔900万美元里程碑付款，http://www.yypharm.cn/news/zyqy/2014-09-16/821.html
勃林格中国首例CMO的示范意义，http://web.yyjjb.com:8080/html/2014-09/10/content_213947.htm
药明康德为中裕新药生产的艾滋病新药Ibalizumab获得美国FDA批准用于新药临床试验，http://www.wuxiapptec.com.cn/press/detail/209/18.html
创业启示：生物制药公司成功创业需要哪些要素？, http://www.yypharm.cn/news/jishuwenxian/2014-01-22/623.html
胃癌（gastric cancer）是全球癌症相关死亡的第3大常见病因，也是发病率排名第4的最常见恶性肿瘤。尽管罗氏赫赛汀（Herceptin）已获批用于HER2阳性胃癌的一线治疗，并为生物标志物（biomarker）驱动的个性化治疗铺平了道路，然而，礼来2014年4月获批的血管生成抑制剂Cyramza（ramucirumab）将满足临床治疗中对更加有效的二线疗法的巨大医疗需求。目前，胃癌后期研发管线依旧相当活跃，在预测期内，广泛药物类别中多种新颖疗法的预期上市，将使不可切除性或转移性胃癌的临床治疗方案进一步多元化。
根据全球领先的制药与医疗保健顾问公司Decision Resources日前发布的一份新报告，未来10年（2013-2023），不可切除性局部晚期和转移性胃癌临床治疗领域，5种靶向性治疗药物的上市，将帮助推动美国、日本、欧洲5大主要市场（法国、德国、意大利、西班牙、英国）胃癌市场高速发展，从2013年的11亿美元，增长至2023年的38亿美元，其中，日本的胃癌发病率最高，使该国成为最具商业吸引力和有利可图的市场。
礼来的血管生成抑制剂Cyramza（ramucirumab）于2014年4月获FDA批准，是FDA批准用于转移性胃癌二线治疗的首个靶向疗法，该药将部分满足晚期胃癌二线治疗中的巨大医疗需求。然而，胃癌市场中，更有效、更安全及耐受性更好的药物，仍存在相当多的市场机会。
——生物标志物助推个性化治疗：处于早期和晚期临床阶段的许多药物，均针对携带特定生物标志物的亚组患者群体，尤其是HER2和c-Met。罗氏HER2靶向性药物Perjeta和Kadcyla、安进处于III期临床的c-Met抑制剂rilotumumab的出现，将助推胃癌治疗超更加个性化的方向前进。
——罗氏将统治HER2阳性胃癌市场：Kadcyla和Perjeta将争夺HER2阳性胃癌市场份额，该市场将由罗氏的HER2专营权统治。
——HER2阴性胃癌存巨大医疗需求：接受采访的专家强调，胃癌临床治疗中，迫切需要能够延长患者生命的更有效的治疗方案，尤其是HER2阴性患者群体，该群体不适合罗氏赫赛汀（Herceptin）的治疗。
——Cyramza和Kadcyla竞争二线治疗：接受采访的专家预计，礼来Cyramza将迅速成为胃癌二线治疗的首选药物。然而，Decision Resources则认为，罗氏Kadcyla将成为胃癌二线治疗的重大突破，该药将成为经罗氏赫赛汀（Herceptin）治疗后病情复发的HER2阳性胃癌患者群体的标准治疗药物。
——c-Met临床前景存疑：罗氏单抗药物MetMab的失败，使业界对于c-Met抑制剂是否能在胃癌领域获得成功产生了相当大的疑问。然而，接受采访的专家指出，在这些临床试验中，c-Met的积极性（positivity）需要被更好的界定，这类药物也许能够使比原先预期更小的患者群体受益。
——胃癌治疗将多元化：未来几年内，随着更多药物的上市，胃癌临床治疗将多元化，尽管这些药物多数为化疗辅助药物。总体而言，在未来，胃癌临床治疗将逐渐摆脱当前单纯化疗为主的治疗方案，将朝更加个性化的治疗方法迈进。
英文原文：The Gastric Cancer Market is Expected to More Than Triple Over the Next Ten Years, Capturing Nearly $3.8 million by 2023
Several Emerging Therapies Will Expand Treatment Armamentarium for Unresectable Locally Advanced or metastatic Gastric Cancer, According to Findings from Decision Resources Group
September 18, 2014 - Burlington, Mass. – Decision Resources Group finds that the gastric cancer market will grow exponentially from over $1.1 billion in 2013 to nearly $3.8 billion by 2023. Fuelling this growth will be the launch of five targeted therapies for unresectable locally advanced and metastatic gastric cancer. Eli Lilly’s Cyramza, which was approved by the U.S. Food and Drug Administration in 2014, is the first targeted therapy to enter the second-line metastatic setting and thereby partially fulfills an unmet need. Nevertheless, considerable market opportunity remains for more effective, safe and tolerable therapies.
——Other key findings from the Pharmacor report entitled Gastric Cancer:
Use of predictive biomarkers: A number of drugs in early- and late-phase clinical development are targeting patient subpopulations that carry a specific biomarker – notably HER2 and c-Met. The emergence of Roche/Genentech/Chugai’s HER2-targeted agents, Perjeta and Kadcyla, and Amgen’s c-Met inhibitor rilotumumab will propel gastric cancer treatment toward more-personalized therapy.
——New market entrants for HER2-positive gastric cancer: Kadcyla and Perjeta will compete for a share of this small patient segment, making it a dynamic space dominated by the Roche HER2 franchise.
——Unmet needs: Interviewed experts stress that an urgent need remains for more-effective treatment options that can extend survival, especially for patients who are HER2-negative and thus not eligible for Herceptin.
——Second-line dynamics of Cyramza and Kadcyla: Interviewed experts expect that Cyramza will quickly become the treatment of choice in the second-line metastatic setting. However, Decision Resources Group expects that the launch of Kadcyla in this setting will be a major breakthrough in treatment, and that it will emerge as the second-line standard of care for HER2-positive gastric cancer patients who are refractory to Roche/Genentech/Chugai’s Herceptin.
Comments from Decision Resources Group Analyst Sehrish Rafique, M.Sc., Ph.D.:
——“The failure of Roche/Genentech’s MetMab has cast considerable doubt over whether or not c-Met inhibitors will be successful in gastric cancer. However, interviewed experts point out that c-Met positivity needs to be better defined in these trials and that perhaps these drugs benefit a smaller population of patients than originally anticipated.”
——“The gastric cancer treatment algorithm is going to diversify over the coming years with more options becoming available for patients with unresectable or metastatic disease. Whilst many of these agents are adjuncts to chemotherapy, overall we will see a move away from chemotherapy alone being the mainstay treatment for gastric cancer to a more individualized approach.”
上周，诺和诺德的减肥药利拉鲁肽注射液 (liraglutide、Saxenda)获得了FDA一个顾问委员会的支持，正准备用于造福肥胖领域“最杰出的人”，该公司首席科学官Mads Krogsgaard Thomsen对彭博社如是说。除了增加数百名的研发人员之外，诺和诺德拟寻求与大学和生物技术公司开展合作，来为这只产品出谋划策。
“我们正在采取一种整体组合的治疗方式，就像我们已经在糖尿病领域所做的一样，从而针对不同的患者实施个性化用药。” Thomsen说到，并被新闻媒体所引用。
这对于提振减肥药市场的信心来说，不啻是一剂强心针，将给其他被减肥药市场所吸引的制药公司带来一个好兆头。比如说Orexigen 公司刚获得批准的减肥药：盐酸纳曲酮和盐酸安非他酮缓释片剂 (Contrave)。该药销售合作伙伴是武田制药，后者称将为这一减肥药投入900人的销售队伍。
诺和诺德已经为Saxenda(利拉鲁肽注射液)准备了强有力的销售力量。如果诺和诺德和武田一切顺利的话，他们的减肥新药将可能触发长久以来分析师们所期待的数十亿美元的减肥药市场。据美国疾病预防控制中心，超过三分之一的美国成年人是肥胖患者，因此获得治疗资格的患者比比皆是。
而目前仅有200-1000万患者正在接受减肥药的治疗，Orexigen公司首席商务官Mark Booth在周二的演讲时表示。“减肥药市场是一个巨大的、快速增长和拥有巨大潜力的市场。”
但是到目前为止，创造新一代的减肥药的制药公司，已经在获得市场份额方面遇到了麻烦。他们正在竭力说服医生支持减肥药，患者使用减肥药和纳税人支付减肥药费用。
位于加利福尼亚的Vivus 2012年9月获得FDA批准的第二款新型减肥药Qsymia(苯丁胺和托吡酯的缓释制剂)陷入了销售困境，而其竞争对手来自艾瑞纳制药的一款新型减肥药氯卡色林 (lorcaserin、Belviq)表现也不尽如人意，尽管该产品的销售合作伙伴还是日本卫材。2013年，Qsymia的销售额仅为2370万美元，Belviq的净销售额刚刚超过2500万美元。
即便如此，分析师们仍然坚持减肥药市场的巨变即将到来。美国最好的银行，富国银行的分析师Matthew Andrews表示，Contrave到2020年的销售额将达到6.34亿美元，如果Orexigen能使该药物获准用于治疗糖尿病，其销售额将达到12亿美元。
他同时还预测，到2020年，Belviq的销售额将达到4.81亿美元，Qsymia的销售额将达到3.96亿美元，与此同时彭博社的分析师们预测Saxenda的2017年的销售额将达到4.29亿美元。
此外，艾瑞纳制药也正在制定自己的发展计划。该公司希望将Belviq与减肥药芬芬(Fen-phen)中的芬特明联合使用，从而规避芬-芬的心血管风险来刺激销售。
“具体的占比不能透露，但可以肯定的是，糖尿病领域是赛诺菲全球以及中国的主要增长引擎之一。”
中国市场的容量的确能够支撑起赛诺菲对糖尿病业务的战略定位。中国已成为全球糖尿病患者最多的国家。最新数据显示，中国糖尿病患者人数已达1.14亿，约占全球糖尿病患者总数的三分之一；其中2型糖尿病患者，也是赛诺菲糖尿病产品面对的主要患者群体，人数占全部糖尿病患者的93%~95% 。
也不全部都是好消息。在中国，尽管糖尿病市场的潜力巨大，但一直以来被参与者视为挑战的情况在于，糖尿病患者的诊断率处于低位，“中国糖尿病诊断率低于欧美一半以上，治疗效果达标率也不足40%，这是我国最大的问题。”
培训医生提高诊断率
为提高诊断率，赛诺菲给出的解决方案是增加医生的培训，林锦荣介绍了赛诺菲目前正在进行的一项长达五年的医生培训项目CIDE，“希望通过CIDE等项目，培训更多基层医生以提高诊断率。”
CIDE 项目是由赛诺菲公司提供支持，卫生计生委疾病预防控制局领导，中国疾病预防控制中心慢病中心和中华医学会糖尿病学分会(CDS)组织实施，世界卫生组织合作中心国际糖尿病中心(IDC)参与合作的人才培养项目。
此项目的最终诉求点在于通过培训专家，然后再由这些专家深入基层，对县级医院的医生进行相关信息的传递。而之于赛诺菲，这一项目的最大收益则来自，“希望通过医生教育，增进他们对基础胰岛素的了解，使大家真正了解使用基础胰岛素的好处。”
这里的基础胰岛素，通常被认为是来得时——赛诺菲糖药病用药重磅产品的代名词。作为赛诺菲的“重磅炸弹”，来得时全球年销售额超过70亿美元，且在中国长效胰岛素中占据65%的市场份额。
当然，赛诺菲对医生培训项目的借力还不止如此。作为跨国药企拓展基层市场硕果仅存的成功案例，“3年前，赛诺菲中国建立了专注于县级市场的独立团队，从最初100人左右的规模，发展到今天1000人的团队，覆盖到全国25个省、超过1000个县。”
赛诺菲的成绩，部分源自于其CIDE项目的顺利拓展，解决了基层医生对糖尿病诊疗知识的缺乏问题，也增加了赛诺菲与此市场医生的接触。林锦荣对赛诺菲基层市场所取得的成绩颇为满意，“目前，我们已培训超过两万名县级医生。”
而为了提高糖尿病的治疗达标率，赛诺菲延续了之前与政府部门、学会合作开展项目的思路，于今年同中国医师协会联手开展了“基础胰岛素临床规范使用优化管理项目”（以下简称“血糖管理三人行”）。
所谓“血糖管理三人行”，是将医生、护士及患者组成有机整体，采用计算机辅助系统，利用 PAD、短信平台等现代通信手段，对门诊胰岛素治疗患者展开系统化管理的项目。
这一项目最大的亮点在于摈弃了以往单一、单向的医患沟通，不仅能帮助患者及时的血糖监测和胰岛素剂量调整，也能提升患者对治疗方案的依从性,以便捷、高效的方式促进患者的血糖监测和治疗方案落实，促进空腹血糖达标。
在项目的具体实施过程中，医生在对门诊初始胰岛素使用患者做出诊断后，将联合护士对患者进行为期 3 个月的教育、指导和管理，帮助初次使用胰岛素的患者掌握胰岛素规范使用技能及自我管理的知识。
按照赛诺菲的规划，2014 年，“血糖管理三人行”项目计划在全国约 400 家医院陆续开展，覆盖全国 30 个省份共 101 个城市的 1500 多名糖尿病专职医护人员和14万名糖尿病患者。
新产品发力市场
毋容置疑，赛诺菲的学术推广活动为其产品的市场认可搭建了一条可行的通路，而同时进行，且与学术推广相匹配的就是产品的市场发力。
在第三届赛诺菲口服降糖药东西方论坛上，其糖尿病事业部医学总监骆天红为赛诺菲之后的上市产品列出了长长的计划表。预计未来赛诺菲将在中国上市7个产品，其中既包括现有口服降糖药亚莫利与二甲双胍的复方制剂，也有二甲双胍与阿格列汀的复方制剂，还包括糖尿病最新治疗激励的SGLT2、GLP-1和两个血糖检测仪等。
“关于上市时间，它取决于国家食品药品监督管理局审批流程和结果。其中，Lyxumia（GLP-1药物）、血糖仪（BGSTAR）以及第二代来得时笔，已提交上市申请，目前正在走审批流程。在中国的上市时间仍未定，但我们希望能加快上市进程。”林锦荣表示。
事实上，赛诺菲从进入中国糖尿病市场开始，一直在试图打造自己的糖尿病用药从诊断到胰岛素治疗的全链条产品，成为糖尿病治疗系统解决方案的提供者。其后续产品及血糖仪上市后，有助于其目标的实现，而其中的挑战则来自审批速度和产品更新换代后的竞争力。
按照现有糖尿病用药的研究方向，两方面的产品在未来市场上的发力明显，一方面是最新作用机理的糖药病产品，例如GLP-1、SGLT2等，而另一方面则是更为便捷用药方式的产品，例如口服胰岛素等。也许在现阶段的中国市场，这些对赛诺菲而言，并非挑战，因为来得时的“一家独大”，足以抵御来自其他竞争对手。
如同其在糖尿病市场上的竞争对手所言，赛诺菲现在的当务之急是以市场表现来证明，其一直所推崇的糖尿病全方位一体化解决方案的竞争力，因为在中国糖尿病市场上，这样的方式，有成功的先例，同样也有失败者，例如拜耳，它如今正在试图卖出其血糖仪业务。
信源地址：http://www.y-lp.com/pages/Article.aspx?id=5051869255751714216
Novo Nordisk还表示，该临床试验未能达到非劣效的首要终点。然而，试验结果表明， Ryzodeg组和基础治疗组中患者的HbA1c均有所降低，分别降低了7%和6.8%，并且两组之间没有显著差异。
Ryzodeg是两种胰岛素类似物——德谷胰岛素（70%）和门冬胰岛素（30%）——的复方制剂。
该临床试验中，曾接受过基础治疗的患者随机分为两组，一组每日注射两次Ryzodeg；另一组接受基础治疗，包括每日注射一次德谷胰岛素，再加每日注射2-4次门冬胰岛素。
该项研究的首席研究员和内分泌专家Helena Rodbard指出，目前接受基础治疗的患者每日可能需要多达四次的注射，对患者来说是极大的负担，同时也很不方便。与基础治疗相比，Ryzodeg是一种新的治疗选择，Ryzodeg能在有效控制血糖的同时，减少低血糖的发生率和注射次数。对于需要基础胰岛素治疗强化方案的患者来说，这种新的治疗方法是一种十分简便的选择。
(Reuters) - Novo Nordisk's type 2 diabetes treatment Ryzodeg has proved effective in providing good blood sugar control with fewer injections than a so-called basal-bolus treatment, according to new data from a late-stage study.
Ryzodeg combines Tresiba, Novo Nordisk's great hope for future growth, with insulin aspart, a man-made form of insulin, also known as NovoRapid, in a single pen injector.
Novo Nordisk, the world's biggest insulin maker, has launched Ryzodeg commercially in Mexico. Analysts have estimated it has blockbuster potential which could mean sales in the billions of dollars in years to come.
The study, unveiled at a meeting of the European Association for the Study of Diabetes (EASD) in Vienna on Thursday, compared Ryzodeg with basal-bolus treatment, which in this case was Novo's Tresiba and two to four injections of Novo's NovoRapid.
After 26 weeks, both patient groups achieved good blood sugar control but the group taking Ryzodeg needed fewer injections and had fewer incidents of dangerously low sugar levels.
The basal-bolus regimen for treating type 2 diabetes involves taking a longer-acting form of insulin to keep blood sugar levels stable through periods of fasting and separate injections of shorter-acting mealtime insulin to prevent rises in blood sugar levels.
"Patients currently using basal-bolus regimens may need to take up to four daily injections, which can be a great burden and very inconvenient," Dr Helena Rodbard, the study's lead investigator and endocrinologist in Rockville, MD, USA, said.
"Ryzodeg is a new treatment option which provides proven glycaemic (blood sugar) control, with fewer injections and reduced rates of hypoglycaemia (dangerously low sugar levels) compared to basal-bolus regimens," Rodbard said.
Combination therapies for type 2 diabetes are becoming increasingly common as patients continually require additional medicines as the disease progresses.
Complications from diabetes caused by failing to keep blood sugar under control can be serious and may include heart disease, stroke, blindness or kidney disease.
With diabetes affecting 382 million people worldwide and the number of cases expected to rise to 592 million by 2035, according to the International Diabetes Federation, the disease represents a huge market for pharmaceutical companies.
The Danish company, whose main rivals include Sanofi and Eli Lilly & Co, aims to increase the number of people using its drugs to 40 million in 2020 from 23 million in 2012.
这项名为DURATION-NEO-1的III期试验在血糖控制不力的成人2型糖尿病患者中展开，旨在比较在研的每周一次自动注射艾塞那肽混悬剂与每日注射两次的艾塞那肽（Byetta，百泌达）的相关性质。该随机、开放标签的试验在59个试验点共招募了约377名患者。
阿斯利康表示，该试验达到了非劣效的主要终点，表明与Byetta相比，28周内每周一次自动注射艾塞那肽混悬剂能使HbA1c（血糖水平）达到更低的平均值。
Rockwood诊所内分泌科医生兼该试验主要研究者Carol Wysham指出，与Byetta相比，每周一次自动注射艾塞那肽混悬剂除了在降低HbA1c（血糖水平）方面的卓越能力外，在降低体重和其他血糖指标方面也与Byetta相似。该试验结果证明了通过一次性自动注射器给药的在研每周一次艾塞那肽混悬剂的耐药性和有效性。
至于试验的次要终点，相关数据表明，28周内艾塞那肽自动注射组和Byetta组中都有相似数量的患者达到了HbA1c降低7%的目标水平，而艾塞那肽自动注射组中更多患者HbA1c水平则是降低了6.5%甚至更低。
阿斯利康全球药品开发心血管和代谢部门负责人Elisabeth Bj?rk讲到，阿斯利康致力于更好地了解糖尿病患者的包括改善给药方式在内的不同需求。DURATION-NEO-1试验结果也支持了我们继续对糖尿病患者的承诺，即开发新型每周一次自动注射艾塞那肽混悬剂并研发新型给药装置。
AstraZeneca Posts Positive Data on its Type II Diabetes Drug - Analyst BlogPipeline updates are highly awaited events in the pharma/biotech sector as they play an important role in deciding whether or not to invest in a particular company. These updates provide information on experimental drugs and at times give an insight into the commercial potential of the candidate once it is successfully developed and commercialized.
A couple of days back AstraZeneca ( AZN ) announced encouraging data from the phase III DURATION-NEO-1 study which compared the use of an investigational formulation of exenatide once-weekly suspension for autoinjection to Byetta, a twice-daily exenatide injection in adults suffering from type II diabetes who did not show sufficient glycemic control. The data from the study was presented at the annual meeting of the European Association for the Study of Diabetes.
Approximately 377 type II diabetes patients, who were not achieving sufficient reduction in HbA1c (an indicator of glucose level in the blood) with diet and exercise or other oral anti-diabetic medications, were enrolled in the 28-week, randomized, open-label study.
At 28 weeks it was found that exenatide once-weekly suspension for autoinjection provided greater mean reductions in HbA1c compared to Byetta. The study met its primary endpoint of non-inferiority.
Our Take
As per data provided by the International Diabetes Federation (IDF), more than 382 million people suffer from diabetes worldwide. This number is expected to increase to 592 million by 2035. Type II diabetes accounts for most (approximately 90-95%) of all diabetes cases, as per Centers for Disease Control and Prevention data. Thus, the type II diabetes market is very lucrative.
If approved, the new once-weekly formulation of exenatide suspension for autoinjection provides type II diabetes patients with a more convenient treatment option over Byetta's twice-daily dosing.
However, we are concerned about the increasing competition in the type II diabetes market. This year alone GlaxoSmithKline's ( GSK ) Tanzeum and Eli Lilly and Company's ( LLY ) Trulicity were approved for type II diabetes among others. Both Tanzeum and Trulicity are administered once a week.
AstraZeneca carries a Zacks Rank #3 (Hold). A better-ranked stock in the health care sector is Gilead Sciences Inc. ( GILD ) carrying a Zacks Rank #1 (Strong Buy).
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