Discovery of STC15, the First Molecule Specifically Targeting an RNA Methyltransferase Enzyme to Enter Clinical Development

RNA epigenetics is an unexplored and exciting field for drug discovery. While post-transcriptional RNA modifications have been known for over 30 years, their mechanism and functional consequences have only recently begun to emerge. STORM Therapeutics is the first company devoted to pursuing RNA-modifying enzymes as drug targets. Tackling new classes of enzymes such as RNA methyltransferases generates challenges in assay development, screening, and downstream hit to lead progression. This talk will describe some of the challenges we have faced in prosecuting our RNAMT programmes and the techniques we have developed to meet them. We will also describe the discovery of our first-in-class oral METTL3 inhibitor STC-15, the first inhibitor of an RNA methyltransferase to enter clinical development and share emerging data from the phase one clinical trial.