医药信息速递：成都先导化学1类新药获批临床，新型HDAC I/IIb亚型选择性小分子抑制...

小刚刚

中国成都 2018年5月7日，成都先导今日宣布，公司已收到中国食品药品监督管理局（CFDA）核发的新型HDAC I/IIb亚型选择性小分子抑制剂HG146针对多发性骨髓瘤适应症的1类新药临床批件。

HG146是成都先导利用其核心技术平台DNA编码化合物库技术自主开发，拥有全新分子骨架结构的组蛋白去乙酰化酶（HDAC）I/IIb亚型选择性抑制剂。临床前研究结果证明其具有合理的药效和临床前安全性，与目前针对此类靶点的上市药物相比，展示出更好的有效性和安全性，并且具有肿瘤免疫治疗方面的潜力。

“我们将快速推进HG146的临床研究，希望该药物能够顺利早日使罹患疾病的病人受益。同时，成都先导将对核心技术平台DNA编码化合物库技术进行持续不断的创新，与合作伙伴一起利用这一技术产生更多的新药，满足更多的未满足临床需求。”成都先导董事长兼CEO李进博士如是说。

关于HG146

HG146主要通过上调细胞内组蛋白和微管蛋白乙酰化水平，调节抑癌基因的表达，并扰乱癌细胞内的蛋白稳态，诱发癌细胞凋亡。通过选择性地抑制HDAC I/IIb亚型，HG146能够抑制与多发性骨髓瘤的预后密切相关的信号通路，实现其抗肿瘤生物效应，同时降低了分子水平上可能存在的副作用。

关于多发性骨髓瘤

多发性骨髓瘤（Multiple myeloma, MM）是一种浆细胞（即效应B细胞）不正常增生，并侵犯骨髓的恶性肿瘤，至今仍是一种无法治愈的疾病。当浆细胞发生恶性癌变时，变为骨髓癌细胞（Myeloma cell），这些细胞倾向于聚集在骨髓内或骨外侧两端的坚硬部分，形成多个肿瘤病灶。多发性骨髓瘤会引发蚀骨细胞活化，导致骨骼外部的硬骨被破坏，引发骨骼疼痛、高钙血症、贫血、肾脏损害等症状。多发性骨髓瘤大多数患者的年龄在50岁以上，男女比例约为1.6：1。全球每年约有48.8万多发性骨髓瘤患者，并导致约10.1万人死亡。根据2017年中国国内的统计数据，多发性骨髓瘤在中国的发病率约为每10万人中2-3人，每年新增病例约2万人，总患者数估计超过10万人。根据2017年美国国内的统计数据，该疾病在美国的发病率约为每10万人中6.5人，占美国当年所有癌症病例的1.8%，约有30280例新确诊病例，以及约12590例患者死亡。

关于成都先导

成都先导是一家从事新药研发的生物技术公司。总部位于中国成都，在美国设有子公司。成都先导已建立了一个独特的早期新药发现平台，即DNA编码化合物库设计合成与筛选技术。目前，成都先导基于数百种不同的骨架结构，已经完成逾千亿结构全新、具有多样性和类药性DNA编码化合物的合成，并且已有多个案例证实了其针对已知靶点和新兴靶点筛选苗头化合物的能力。成都先导已与50余家制药、生物技术企业以及研究机构建立合作，致力于发现、开发新的治疗方案。

HitGen’sSmall Molecule IND Receives Approval by CFDA for Clinical Trials

Chengdu,China, May 7, 2018– HitGen Ltd. announced that its novel small molecule ClassI/IIb HDAC inhibitor HG146 (Category 1 of new chemical drug) received approvalby Chinese Food and Drug Administration (CFDA) to begin the clinical trials for Multiple Myeloma.

HG146 is aself-developed Class I/IIb HDAC inhibitor with novel chemical scaffold developedfrom HitGen’s proprietary DNA Encoded Library technology. Comparing with thecurrent listed HDAC inhibitors for multiple myeloma, its preclinical studiesshowed better efficacy and safety profile with potential application inimmuno-oncology.

“With this CFDA approval, we aim to start theclinical trials of HG146 as soon as possible. Meanwhile, we will continue toinnovate and improve our core platform of DNA Encoded Libraries, in order tobring more new medicines to patients while collaborating with our partners andsatisfying unmet clinical needs.” said Dr. Jin Li, Chairman and Chief ExecutiveOfficer of HitGen.

About HG146

In preclinical studies, HG146 has been shown to upregulatethe acetylation level of histone and tubulin, modulating the expression oftumor-suppressing genes and disrupting the protein homeostasis in cancer cellsto induce apoptosis. By selectively inhibiting Class I/IIb HDAC, HG146 caninhibit the critical signaling pathways related to the prognosis of multiple myelomaand realize its antitumor biological effects while reducing potential sideeffects.

About MultipleMyeloma

Multiple Myeloma (MM) is a type of neoplasm formed bymalignant plasma cells, which still remains an unmet medical need. Through a complex, multi-step process, healthyplasma cells transform into malignant myeloma cells developing in the soft,spongy tissue within bone marrow or two sides of bones to form multiple tumor lesions.MM will cause osteocyte activation, damage os osseum to further triggerosteodynia, hypercalcemia, anaemia, kidney damage, etc. The average age ofpatients suffering from MM is above 50 with the ratio of male to female 1.6:1.There are about 488,000 patients annually worldwide and about 100,000 of themwill lose their lives. According to China’s 2017 statistics, the incidence ofMM in China is about 2-3 out of 100,000 people with 20,000 new cases annually.Meanwhile, the incidence of MM in US is about 6.5 out of 100,000 peopleaccounting for 1.8% of all cancer cases in 2017, with 30,280 new cases and12,590 death.

About HitGenLtd.

HitGen is a biotechcompany with headquarters and main research facilities based in Chengdu, Chinaand with a subsidiary in the USA. HitGen has established a platform for smallmolecular discovery centred around DNA encoded chemical libraries (DELs).HitGen’s DELs contain more than 100 billion novel, diverse, drug-like smallmolecule and macrocyclic compounds. These compounds are members of DELssynthesised from many hundreds of distinct chemical scaffolds, designed withtractable chemistry, and yielding proven results for the discovery of smallmolecule leads against precedented and unprecedented classes of biological targets.HitGen is collaborating with over 50 pharmaceutical, biotech, and chemicalcompanies and research institutes in Asia, North America and Europe to discoverand develop approaches for novel medicines.

syhorchid

祝贺成都先导

beiwei5du

1类临床一大把，以前看到科伦什么的4个原料药FDA登记通过都要宣传一把，嘻嘻！