Momenta制药近日宣布，FDA已授予其新型抗肿瘤候选药物necuparanib（M402）治疗胰腺癌的孤儿药地位。

necuparanib是一种硫酸乙酰肝素类似物，这是一种新颖的肿瘤候选药物，被设计为对肿瘤细胞具有广泛的作用。目前已有大量研究表明，在癌症患者中，使用肝素治疗静脉血栓可产生抗肿瘤活性，但这些肝素产品的使用剂量受抗凝血活性限制。necuparanib由普通肝素衍生而来，已被设计为显著降低抗凝血活性，同时保留相关的抗肿瘤特性。

在接下来几个月里，Momenta预计将完成I/II期临床试验的Part A剂量递增部分研究，该试验中，将necuparani联合Abraxane（nab-紫杉醇）和吉西他滨用于晚期胰腺癌的治疗，研究结果预计将于2014下半年获得。此外，Momenta计划于2014年底启动Part B部分，该部分研究将调查necuparanib+Abraxane+吉西他滨组合疗法相对于Abraxane+吉西他滨组合疗法的抗肿瘤活性。

英文原文：Momenta Pharmaceuticals Receives Orphan Drug Designation for Necuparanib (Formerly M402) in Pancreatic Cancer

CAMBRIDGE, Mass., June 5, 2014 (GLOBE NEWSWIRE) -- Momenta Pharmaceuticals, Inc. (Nasdaq:MNTA) today announced that its novel oncology candidate, necuparanib (formerly M402), has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of pancreatic cancer.

"We are pleased to receive Orphan Drug Designation for necuparanib, which highlights the great need for new medications for patients suffering from pancreatic cancer," said Jim Roach MD, Chief Medical Officer of Momenta Pharmaceuticals. "We are encouraged by the progress of the program to date, and in the next several months, we anticipate completing Part A of our ongoing Phase 1/2 study of necuparanib in combination with Abraxane® and gemcitabine. We look forward to sharing the results from Part A and advancing the product into the Phase 2 part of the study in the second half of 2014."

The FDA's Orphan Drug Designation program provides orphan status to drugs and biologics intended to treat, diagnose or prevent rare diseases/disorders, defined as affecting fewer than 200,000 people in the U.S. This designation provides certain incentives, including federal grants, tax credits, waiver of PDUFA filing fees and a seven-year marketing exclusivity period against competition once the product is approved.

About Necuparanib

The United States Adopted Names (USAN) Council has recently adopted "necuparanib" as the unique non-proprietary name for M402. Necuparanib, a heparan sulfate mimetic, is a novel oncology drug candidate engineered to have a broad range of effects on tumor cells. The use of heparins to treat venous thrombosis in cancer patients has generated numerous reports of antitumor activity; however, the dose of these products has been limited by their anticoagulant activity. Necuparanib, which is derived from unfractionated heparin, has been engineered to have significantly reduced anticoagulant activity while preserving the relevant antitumor properties of heparin. In the next several months, Momenta expects to complete the Part A dose escalation component of the Phase 1/2 trial evaluating necuparanib in combination with Abraxane® (nab-paclitaxel) and gemcitabine in patients with advanced metastatic pancreatic cancer, and to report clinical data from Part A in the second half of 2014. The company plans to initiate Part B of the study by the end of 2014. Part B will be a randomized, controlled, proof of concept study to evaluate the antitumor activity of necuparanib in combination with Abraxane plus gemcitabine, versus Abraxane plus gemcitabine alone.

About Momenta

Momenta Pharmaceuticals is a biotechnology company specializing in the detailed structural analysis of complex mixture drugs and is headquartered in Cambridge, MA. Momenta is applying its technology to the development of generic versions of complex drugs, biosimilar and potentially interchangeable biologics, and to the discovery and development of novel products.