Shanghai-based ABM Therapeutics Inc. has announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its small molecule BRAF inhibitor, ABM-1310. This designation is in relation to the molecule’s potential use in treating BRAF V600 mutant glioblastoma (GBM), a rare and aggressive form of brain cancer.
ABM-1310: A Promising BRAF Inhibitor for GBM
ABM-1310 is an oral BRAF inhibitor that has demonstrated high selectivity, high water solubility, and high permeability of the blood-brain barrier, which are crucial for drugs targeting brain tumors. The drug is currently undergoing a Phase I study in advanced solid tumors with BRAF V600 mutation in both China and the US. Early results from these studies have shown good anti-tumor efficacy, safety, and tolerability, indicating its potential as a treatment for GBM.
Phase I Study Progress in China
A Phase I study specifically for the drug in GBM is well underway in China. This study is important for advancing the understanding of ABM-1310’s efficacy and safety profile in patients with this specific type of brain cancer. The Orphan Drug Designation from the FDA not only highlights the unmet medical need for treatments like ABM-1310 but also provides incentives for ABM Therapeutics to continue its development efforts.-Fineline Info & Tech
