1、 2024 Syneos Health.All rights reserved.Navigating Tumor-Agnostic Drug DevelopmentValue,Challenges,and Strategic Insights for Sponsors2IntroductionTumor-agnostic drug development has been an important milestone for cancer treatment by targeting specific genetic traits present across various cancer t

2、ypes.Through innovative trial designs such as basket trials,these strategies uncover response biomarkers and resistance mechanisms,going beyond traditional tumor classifications.Their impact on clinical research signals a transformative shift toward personalized,genomically guided therapies in cance

3、r care.KEYTRUDA(pembrolizumab)MerckMSI-H/dMMRVITRAKVI(larotectinib)Loxo Oncology/LillyNTRK gene fusionROZLYTREK(entrectinib)Ignyta/RocheNTRK gene fusionKEYTRUDA(pembrolizumab)MerckTMB-HJEMPERLI(dostarlimab)GSKdMMRTAFINLAR+MEKINIST(dabrafenib+trametinib)NovartisBRAF V600ERETEVMO(selpercatinib)Loxo On

4、cology/LillyRET-fusionENHERTU(trastuzumab deruxtecan)Daiichi SankyoHER2May2017Nov.2018Aug.2019Jun.2020Aug.2021Jun.2022Sep.2022Apr.20243Tumor-Agnostic Approval HistoryThe landscape of tumor-agnostic approved drugs has witnessed an important leap forward in recent years,with eight assets earning accel

5、erated approval from the FDA(US)Figure 1,four approvals by the EMA(Europe),and five by the PMDA(Japan).These treatments cover various drug targets for different types of cancer,giving hope to patients,especially those with rare tumors and few treatment options.Pembrolizumab,a PD-1 inhibitor,made a n

6、otable impact in 2017 as the first tumor-agnostic drug to be approved for tumors with high DNA Mismatch Repair(dMMR-H)or high Microsatellite Instability(MSI-H).Following this,larotrectinib and entrectinib emerged as examples of precision medicine,targeting Neurotrophic Tyrosine Receptor Kinase(NTRK)