On June 24, 2024, at the 28th Japanese Cancer Molecular Targeted Therapy Association held from June 19 to 21, NANO MRNA <4571> announced the report on the clinical results of a physician-led trial of PRDM14 (siRNA) in the NANO MRNA pipeline by Dr. Shunji Takahashi, Head of the Comprehensive Oncology Department at the National Cancer Center Hospital.
PRDM14 siRNA / YBC polymer complex was administered to 10 patients with HER2-negative metastatic breast cancer, and there were no serious side effects and safety was not a significant issue.
There were 6 PD out of 10 cases with anti-tumor effect, and 4 cases with SD. Long-term SD was observed in one case with the highest dose, and the progression-free survival period was 25-180 days (median 45 days).
Pharmacokinetics showed an increase in PRDM14 siRNA exposure with increasing dose of PRDM14 siRNA / YBC polymer complex administration, and effective plasma concentrations extrapolated from non-clinical results were achieved at the highest dose, demonstrating the usefulness of YBC polymer as a nucleic acid drug delivery system in clinical practice.
The "YBC polymer" used in the same trial is also used in the physician-led trial "TUG1 ASO", which targets highly malignant glioblastomas among brain tumors. Based on the results of this trial, it is expected that sufficient drug exposure can be expected for TUG1 ASO as well, aiming to provide new mechanisms of action drugs for diseases without effective treatments.