澳大利亚生物技术公司Amplia Therapeutics公布了其主要候选药物narmafotinib的最新实验室研究数据[1]。该研究由悉尼Garvan医学研究所Paul Timpson教授团队完成，表明这一焦粘着斑激酶（FAK）抑制剂可以靶向肿瘤纤维化、化疗耐药性和转移扩散等胰腺癌的核心特征[1]。研究显示，narmafotinib与化疗联合使用时能够减少肿瘤纤维化、增强抗肿瘤活性，并下调与化疗耐药性相关的多个基因活性[1]。
在临床试验中，该药物组合方案的总体缓解率达到36%，疾病控制率为70%，高于化疗单独治疗的50%[1]。中位总生存期为11.1个月，相比单独化疗延长超过两个月，无进展生存期为7.7个月[1]。Amplia已启动第2b期临床试验，计划于2024年第四季度开始在澳大利亚3至4个医疗中心招募约12名患者[1]。
除胰腺癌外，公司还推进了针对卵巢癌的研究。PRROSE试验与澳大利亚新西兰妇科肿瘤学组合作，计划招募15至20名高级别浆液性卵巢癌患者[1]。Amplia首席执行官Chris Burns表示，"这些研究加强了narmafotinib临床开发的科学基础，进一步深化了对其多方面作用机制的认识"[1]。截至6月季度末，公司持有现金储备2400万澳元[1]。
Australian biotechnology company Amplia Therapeutics has released new laboratory data demonstrating the potential of its lead candidate narmafotinib, a focal adhesion kinase (FAK) inhibitor, against pancreatic and ovarian cancers.[1] The research, conducted by Professor Paul Timpson's team at the Garvan Institute of Medical Research in Sydney, shows that narmafotinib targets key characteristics of pancreatic cancer including tumor fibrosis, chemotherapy resistance, and metastatic spread, providing further evidence for the drug's clinical development.[1]
In clinical trial data, the combination of narmafotinib with chemotherapy achieved an overall response rate of 36 percent and disease control rate of 70 percent, compared to 50 percent disease control with chemotherapy alone.[1] Median overall survival reached 11.1 months, exceeding single-agent chemotherapy by more than two months, while median progression-free survival was 7.7 months.[1] The company has initiated a Phase 2b trial, with patient enrollment expected to begin in the fourth quarter of 2024 across three to four medical centers in Australia, recruiting approximately 12 patients.[1] Additionally, Amplia is planning to evaluate narmafotinib's efficacy in ovarian cancer through the PRROSE trial in collaboration with the Australia and New Zealand Gynaecological Oncology Group (ANZGOG), targeting recruitment of 15 to 20 patients with high-grade serous ovarian cancer.[1]
CEO Chris Burns stated that the research "strengthens the scientific foundation of narmafotinib's clinical development and deepens our understanding of its multifaceted mechanism of action."[1] At the end of the June quarter, Amplia held cash reserves of AUD 24 million.[1]