GNE-049 在 BRET 细胞试验中表现良好，在正交的靶向测量中，其抑制 MYC 表达（MV4-11 细胞系），EC50 为 14 nM。[1]
CBPD-409 通过直流诱导 CBP/p300 的强降解500.2-0.4 nM，IC显示出很强的抗增殖作用50VCaP、LNCaP 和 22Rv1 AR+ 前列腺癌细胞系中的 1.2-2.0 nM。[2]

GNE-049 shows acceptable PK in mouse, rat, dog, and monkey. GNE-049 is further evaluated in a rat single dose (30-250 mg/kg QD) toxicokinetic study. GNE-049 is selective for CBP/P300 and, importantly, quite selective (3820-fold) over BRD4(1), which revealed from the Determination of potency versus a selection of bromodomains. Furthermore, at the 250 mg/kg dose level, the ratio of the unbound drug concentration in the brain to unbound drug concentration in plasma (Kp,uu) 3 h post-dose is determined to be 0.43, indicating that GNE-049 is penetrating into the CNS and potentially resulting in the observed toxicity.