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

MGB Biophamra建议进行一项IIa期研究，以确认MGB-BP-3在艰难梭菌相关腹泻（CDAD）患者中的安全性，耐受性和疗效。美国CDC已将C.这是一个紧迫的威胁，因为它每年仅在美国就影响近50万人，年死亡率约为30，000。欧盟的CDAD发病率估计与美国相似，但没有集体数据。目前CDAD治疗的主要未满足的医疗需求是初始治疗后非常高的复发率和由高毒力核糖型BI/NAP 1/027引起的低治愈率。MGB-BP-3对所有念珠菌的杀菌活性均超过万古霉素，具有压倒性的上级优势。在所有临床前试验中研究的艰难梭菌菌株，包括这种高毒力核糖型。GlobalData报告称，仅在美国，CDAD治疗的市场规模估计就将超过10亿美元，使MGB-BP-3成为一种潜在的数十亿美元药物。在我们与FDA的PIND会议上，我们提出了一个综合的，快速的MGB-BP-3临床开发计划。FDA对此表示支持，并接受了我们的II期研究设计。拟定的IIa期研究将选择耐受性和疗效最佳的剂量/方案，用于在BI/NAP 1/027核糖体型CDAD患者中进行的万古霉素随机化、比较性IIb期研究。FDA已经同意，我们只需要进行一项关键的III期研究以提交NDA。MGB-BP-3在成功完成临床开发后，有可能成为CDAD的新标准治疗。

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.