ASTU-045

ASTU-045 is a selective small molecule inhibitor of Methyltransferase-like 3 (METTL3), the catalytic subunit of the m6A methyltransferase complex. Developed by NovaliX and Aqemia using structure-based drug design, ASTU-045 targets the deposition of N6-methyladenosine (m6A) on eukaryotic mRNA, a process that plays a critical role in gene regulation and is often hijacked in oncogenesis. In preclinical studies, ASTU-045 demonstrated cellular target engagement and enhanced PBMC-mediated killing of cancer cells. In vivo, oral administration of the compound resulted in tumor stasis in the MC38 colorectal syngeneic mouse model and showed significant synergy when combined with anti-PD1 therapy, highlighting its potential as an epigenetic modulator to stimulate anti-tumor immunity.