Phase IIa Clinical Trial for a Novel Treatment of Clostridium Difficile-Associated Diarrhoea (CDAD)

MGB biophammra提议开展一项IIa期研究，以确认MGB- bp -3对艰难梭菌相关性腹泻（CDAD）患者的安全性、耐受性和有效性。美国疾病控制与预防中心已将艰难梭菌列为紧急威胁，因为仅在美国，它每年就影响近50万人，年死亡率约为3万人。据估计，欧盟的CDAD发病率与美国相似，尽管没有集体数据。目前CDAD治疗的主要未满足的医疗需求是初始治疗后复发率很高，高毒力核型BI/NAP1/027导致治愈率低。在所有进行的临床前试验中，MGB-BP-3对所有艰难梭菌菌株（包括这种高毒力核糖型）显示出压倒性的优于万古霉素的杀菌活性。GlobalData报告称，仅在美国，CDAD治疗的市场规模估计就将增长到10亿美元以上，这使得MGB-BP-3成为一种潜在的数十亿美元的药物。在与FDA的PIND会议上，我们提出了MGB-BP-3的综合快速临床开发计划。FDA支持并接受了我们的II期研究设计。拟议的IIa期研究将为BI/NAP1/027核型CDAD患者的万古霉素随机对照IIb期研究选择具有最佳耐受性和疗效的剂量/方案。FDA已经同意我们只需要进行一项关键的III期研究来提交NDA申请。如果成功完成临床开发，MGB-BP-3有可能成为CDAD的新治疗标准。

MGB Biophamra propose conducting a Phase IIa study to confirm the safety, tolerability and efficacy of MGB-BP-3 in patients with Clostridium difficile-associated diarrhoea (CDAD). The US CDC has designated C. difficile as an urgent threat, as it affects almost half a million people annually in the US alone, with a high annual mortality rate of around 30,000. The incidence of CDAD in the EU is estimated to be similar to the US, although there are no collective data. The major unmet medical need of current CDAD therapy is the very high recurrence rate after initial treatment and the low cure rate caused by the hypervirulent ribotype BI/NAP1/027. MGB-BP-3 has shown overwhelmingly superior bactericidal activity over vancomycin against all C. difficile strains investigated in all preclinical tests performed, including this hypervirulent ribotype. GlobalData reports that the market size for the treatment of CDAD is estimated to grow to over $1 bn in the US alone, making MGB-BP-3 a potentially multibillion dollar drug. At our PIND meeting with the FDA we proposed an integrated, fast track clinical development plan for MGB-BP-3. The FDA supported this and accepted our Phase II study design. The proposed Phase IIa study will select the dose/schedule with the best tolerability and efficacy for the randomised, comparative Phase IIb study against vancomycin in CDAD patients with the BI/NAP1/027 ribotype. The FDA has agreed that we only need to conduct one pivotal Phase III study for NDA filing. MGB-BP-3 has the potential of being the new standard of care for CDAD, on successful completion of clinical development.