澳大利亚珀斯生物制药公司NeuroScientific Biopharmaceuticals已与美国FDA确定于8月31日召开Pre-IND会议[1]。该会议将围绕该公司的专利干细胞技术平台StemSmart在治疗难治性克罗恩病中的应用展开，是向FDA正式提交IND申请的关键监管步骤[1]。公司计划在2024年年底前向FDA提交完整的IND申请[1]。
早期特殊通道试验结果为该平台的临床推进奠定了基础[1]。在该试验中，StemSmart技术在5名患者中均显示疗效，且未出现严重不良事件[1]。公司首席执行官Nathan Smith表示："与FDA接口对于确保计划中的临床试验设计达到支持产品临床转化的标准至关重要"[1]。
克罗恩病全球市场规模超过100亿美元[1]，体现了该治疗方案的市场潜力。NeuroScientific计划在美国和澳大利亚推进该技术的发展[1]。
NeuroScientific Biopharmaceuticals, a Perth-based biopharmaceutical company, has scheduled a Pre-IND meeting with the U.S. FDA for August 31 to advance clinical development of its patented StemSmart stem cell technology platform for treating refractory Crohn's disease [1]. The Pre-IND meeting represents a critical regulatory milestone prior to submitting a formal Investigational New Drug application, with the company targeting a complete IND submission by the end of 2024 [1].
Early-stage special access program trial results demonstrated efficacy across all five participating patients with no serious adverse events reported [1]. The global market for Crohn's disease treatment exceeds USD 10 billion [1]. CEO Nathan Smith stated that "interfacing with the FDA is crucial to ensure that the design of our planned clinical trials meets the standards required to support product clinical translation" [1]. The company is targeting both the U.S. and Australian markets for the StemSmart platform [1].