[新药申请]FDA批准BHT-3034治疗重症肌无力的新药申请

中文：http://www.drugfuture.com/Article/Print.asp?ArticleID=1379

Bayhill制药公司基于其专有的BHT-DNATM平台以开发针对自身免疫性疾病和免疫介导性疾病的新型治疗方法，近日宣布向美国FDA提交的BHT-3034新药申请成功获批，BHT-3034是一种用于免疫治疗自体免疫性神经肌肉接头疾病重症肌无力患者疾病改善的DNA质粒疫苗。该份IND的提交是基于有希望的临床前疗效研究结果，即疾病严重程度明显减轻和抗乙酰胆碱受体抗体减少，以及GLP毒理学研究没有证据表明其有毒性和免疫原性。该IND还包括一项I / II期剂量递增临床研究，其给予40例重症肌无力患者肌肉注射BHT-3034.

BHT-3034是利用Bayhill公司专有的BHT-DNATM平台进行研发进入临床试验的第3个化合物。

该公司创办人兼董事、斯坦福大学免疫学跨学科项目主任Lawrence Steinman医师表示，“我们为通过这个机会为重症肌无力患者带来一种新的缓解疾病方法，感到非常兴奋。BHT-3034有通过一种高度特异的方式显著降低自体免疫反应的潜能，从而证明其临床益处。

PS.感谢病友mgp的丈夫于2012年1月提供信息，我近日才看到邮件，迟发歉意！

英文如下：

http://www.dailystrength.org/c/Myasthenia_Gravis/forum/11617055-bht3034-mg

Bayhill Therapeutics Inc, a clinical-stage biopharmaceutical company utilizing its proprietary BHT-DNA™ platform to develop novel and targeted autoimmune and immune-mediated disease treatments, today announced the successful clearance of an IND with the US FDA for BHT-3034, a disease-modifying DNA plasmid vaccine immunotherapy for patients with the autoimmune neuromuscular junction disease myasthenia gravis. The IND was filed based on promising preclinical efficacy results showing significant disease severity attenuation and anti-acetylcholine receptor autoantibody reduction, as well as GLP toxicology studies showing no evidence of toxicity or immunogenicity. The IND also included a phase I/II dose escalation clinical study to treat up to 40 subjects with myasthenia gravis with 12 weekly doses of BHT-3034 intramuscularly.

“We are excited by this opportunity to bring forward a novel disease modifying therapy to patients with myasthenia gravis. BHT-3034 has the potential to significantly reduce the autoimmune response in a highly specific manner and thereby demonstrate a clinically meaningful benefit.”

Company founder and director Lawrence Steinman, MD, Chairman of the Interdepartmental Program in Immunology at Stanford University, stated, "We are excited by this opportunity to bring forward a novel disease modifying therapy to patients with myasthenia gravis. BHT-3034 has the potential to significantly reduce the autoimmune response in a highly specific manner and thereby demonstrate a clinically meaningful benefit."

This represents the third compound to enter clinical testing from Bayhill Therapeutics' proprietary BHT-DNA™ platform. The first compound, BHT-3009 for multiple sclerosis, has achieved success in a phase I/II trial where safety and antigen-specific immune tolerance was demonstrated, and in a phase II trial where a responder population of high serum anti-MBP (myelin basic protein) subjects demonstrated a > 70% reduction in brain lesions.

To move its programs forward in the most efficient manner, Bayhill Therapeutics also announced the retention of P² Partners, LLC to assist the Company in the pursuit of a strategic transaction.

SOURCE Bayhill Therapeutics

［此帖子已被 sairicai 在 2012-2-17 12:41:41 编辑过］