RG2822

RG2822 is a potent, brain-penetrant small molecule histone deacetylase (HDAC) inhibitor currently in preclinical development for the treatment of rare and aggressive pediatric brain cancers, such as atypical teratoid/rhabdoid tumors (AT/RT) and medulloblastoma. Developed by Regent Biosciences, the compound is specifically designed to cross the blood-brain barrier, a significant challenge in treating central nervous system malignancies. Preclinical research conducted at Johns Hopkins University has demonstrated that RG2822 exhibits strong synergistic activity when used in combination with paxalisib (a PI3K/mTOR inhibitor), leading to significantly extended survival in animal models compared to single-agent treatments. By inhibiting HDAC enzymes, RG2822 modulates the epigenetic landscape of tumor cells, promoting cell cycle arrest and apoptosis.