The China’s Center for Drug Evaluation (CDE) website has indicated that BioNova Pharmaceuticals (Shanghai) Ltd.’s novel small molecule, BN104, is on track to obtain Breakthrough Therapy Designation (BTD). This development marks a significant milestone for the treatment of relapsed/refractory acute leukemia patients carrying KMT2A rearrangement and/or NPM1 mutation in China.
BN104: A Selective Inhibitor of Menin for Acute Leukemia Treatment
BN104 is a groundbreaking, highly selective small molecule inhibitor of menin, a scaffold protein that is pivotal in various biological pathways, including cell cycle regulation and genome stability. The drug is specifically under development to address acute leukemia with rearranged mixed lineage leukemia (MLLr) genes or acute myeloid leukemia with NPM1 mutations.
Pre-clinical Success and Regulatory Clearances
Pre-clinical studies have demonstrated BN104’s favorable safety and toxicity profile, along with preliminary efficacy in a mouse model. This has set the stage for BN104 to receive an Orphan Drug Designation (ODD) from the US FDA in April 2023. Building on this momentum, BN104 was cleared for trials in China in August 2023 and in the US in September 2023, highlighting its potential as a treatment for leukemia patients worldwide.-Fineline Info & Tech
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