2014-8-13 国际国内信息总汇

FDA授予单抗药OS2966及表观遗传学药物mocetinostat孤儿药地位【新药汇www.xinyaohui.com讯】 FDA授予单抗药OS2966和表观遗传学药物mocetinostat孤儿药地位，前者是一种抗CD29单抗，后者为选择性组蛋白去乙酰化酶（HDAC）抑制剂。

FDA授予OncoSynergy公司实验性单抗药物OS2966治疗胶质母细胞瘤（glioblastoma）的孤儿药地位。胶质母细胞瘤是最常见和最致命类型的成人脑瘤。OS2966是一种首创疗法，目前正在多个恶性和耐药性实体瘤模型进行评估。该药是一种中和性抗CD29单克隆抗体，能够选择性调节CD29（整合素b1亚基），这是肿瘤生长和恶化（包括增殖、侵袭、血管生成和耐药性）多个机制中的一种关键性通路驱动因子。临床前数据表明，OS2966可有效对抗多种实体瘤，包括复发性和治耐药性胶质母细胞瘤。

此外，FDA也已授予Mirati治疗公司实验性药物mocetinostat治疗弥漫性大B细胞淋巴癌（DLBCL）的孤儿药地位。今年6月，FDA也已授予mocetinostat治疗骨髓增生异常综合症（MDS）的孤儿药地位。mocetinostat是一种口服生物可利用、同型（isotype）选择性强效组蛋白去乙酰化酶（histone deacetylase，HDAC）抑制剂，该药是一种表观遗传学（epidemic）药物，正开发作为一种单药疗法用于弥漫性大B细胞淋巴癌（DLBCL）以及组蛋白乙酰基转移酶（HATs）存在特定基因突变的前列腺癌（bladder cancer）的治疗。

英文原文：FDA Grants Orphan Drug Designation for OncoSynergy's Investigational Monoclonal Antibody OS2966 in the Treatment of Glioblastoma

SAN FRANCISCO, Aug. 12, 2014 /PRNewswire-iReach/ -- oncoSynergy announced today that the FDA Office of Orphan Products Development (OOPD) has granted orphan drug designation for the investigational drug candidate OS2966, a neutralizing anti-CD29 monoclonal antibody, for the treatment of glioblastoma, the most common and deadliest primary adult brain tumor.

OS2966 is a first in class therapeutic being investigated in multiple models of highly aggressive and resistant solid cancers. OS2966 selecively modulates CD29 (integrin b1 subunit), a critical path driver of multiple mechanisms of tumor growth and progression including proliferation, invasion, angiogenesis, and therapy resistance. Pre-clinical data suggest OS2966 may be active against numerous solid cancers including recurrent and therapy resistant glioblastoma.

"The FDA's decision to grant orphan drug designation highlights the promise of our program and the dire unmet need in glioblastoma wher median survival is a mere 15 months despite maximal current therapy," commented Dr. W. Shawn Carbonell, MD, PhD, Founder and CEO of OncoSynergy.

The mission of the FDA OOPD is to advance the development of products for the diagnosis and/or treatment of rare diseases.  By providing incentives to sponsors the program has successfully enabled development of greater than 400 drugs and biologics for rare diseases since 1983.

"We are pleased to achieve this important regulatory milestone and to begin a collaborative relationship with the Agency and the OOPD as we advance OS2966 towards clinical trials," said Dr. Anne-Marie Carbonell, MD, Vice President of Clinical Development for OncoSynergy. "Orphan designation is a major step towards expediting this promising therapy to a patient population with few treatment options."

about oncoSynergy ¨C oncoSynergy is a biopharmaceutical company based in San Francisco, California focused on addressing unmet needs in oncology, particularly orphan cancers. Our vision is to radically improve outcomes for cancer patients though our unique "S.M.A.R.T." targeted approach allowing for broad inhibition of multiple fundamental cancer growth and progression mechanisms with a single drug. OncoSynergy's lead drug candidate, OS2966, blocks a critical path target driving multiple malignant cancer phenotypes and has demonstrated preclinical efficacy in several solid cancer models including therapy resistant glioblastoma.

Mirati Therapeutics Receives Orphan Designation from U.S. Food & Drug Administration for Mocetinostat in Diffuse Large B-Cell Lymphoma

SAN DIEGO, Aug. 11, 2014 /PRNewswire/ -- Mirati Therapeutics, Inc. (MRTX) today announced that the U.S. FDA has granted Orphan Drug Designation to mocetinostat, a spectrum selecive HDAC inhibitor, for diffuse large B-cell lymphoma (DLBCL). In June, mocetinostat was granted Orphan Drug Designation as a treatment for myelodysplastic syndrome (MDS).  Orphan drug designation is also being sought for bladder cancer patients with specific genetic alterations.

Mocetinostat is being developed as a single agent treatment in patients with diffuse large B-cell lymphoma (DLBCL) and bladder cancer with specific genetic mutations in Histone Acetyl Transferases (HATs) that we believe to be critically involved in the pathogenesis and progression of these tumor types.  Mocetinostat reverses aberrant acetylation resulting from HAT mutations and is predicted to halt tumor progression and reduce tumor burden in patients. Mocetinostat is also in Phase 2 clinical studies in combination with Vidaza as a treatment for intermediate and high-risk MDS.

"We have identified genetic alterations in histone acetylation pathways (CREBBP and EP300) in approximately one third of DLBCL and bladder tumors. Nonclinical tumor models exhibiting these mutations are particularly responsive to mocetinostat," said Dr. Charles Baum, M.D. PhD, president and CEO of Mirati. "Among other benefits, orphan designation provides seven years of market exclusivity to target these genetically defined patients with unmet medical need in the event we achieve regulatory approval."

The FDA's Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations or rare disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

about Mocetinostat

Mocetinostat is an orally-bioavailable, spectrum-selecive HDAC inhibitor. Mocetinostat is enrolling patients in a Phase 2 dose confirmation study in combination with Vidaza as treatment for intermediate and high-risk MDS. Mirati also plans to initiate Phase 2 studies of mocetinostat as a single agent in patients with mutations in histone acetyl transferases in bladder cancer and DLBCL. Initial data from the Phase 2 studies is expected by the end of 2014. In addition to the ongoing Phase 2 clinical trials, mocetinostat has completed 13 clinical trials in more than 400 patients with a variety of hematologic malignancies and solid tumors.

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