Conclusions
LNA-i-miR-221, cancer
CANCER, SOLID TUMOR
The promising results reported here warrant additional clinical studies. The favorable PK profile, the high bio-modulatory activity on canonical targets, the safety profile at 5 mg/kg dose of LNA-i-mirR-221, together with the clinical anti-tumor activity, indicate that this dose is appropriate for use in future clinical trials. Immediate development plans for LNA-i-miR-221 include the design and conduct of a phase II basket study to investigate the efficacy of LNA-i-miR-221 for the treatment of multiple solid tumor types in parallel. On this basis, this study acts as the first-in-human proof-of-concept study for the use of miRNA-targeting strategies in cancer and provides a framework for the future development of this first-in-class LNA inhibitor of miR-221.