BLU2864 (40 nM and 200 nM; 5 d) shows inhibition of forskolin -induced cystogenesis in vitro[1]. Cell Viability Assay [1] Cell Line: mIMCD3 cells Concentration: 40 nM and 200 nM Incubation Time: 5 days Result: Inhibited forskolin induced in vitro cystogenesis of mIMCD3 cells cultured in Matrigel by 72% and 100% at 40 and 200 nM concentrations, respectively, relative to control.

BLU2864 (oral gavage; 45 mg/kg; once daily; 5 d) shows inhibition of renal PKA activity in Pkd1 RC/RC mice [1]. BLU2864 (oral gavage; 30 mg/kg; once daily; 5 d) inhibits PKA activity and ameliorates PKD in Pkd1 RC/RC mice [1]. BLU2864 (oral gavage; 30 mg/kg and 75 mg/kg; once daily; 34 d) reduces FLC tumor growth in vivo [2]. Animal Model: Pkd1 RC/RC mice [1] Dosage: 45 mg/kg Administration: Oral gavage; 45 mg/kg; once daily; 5 days Result: Suppressed kidney basal and total PKA activities by 74% and 87% at 3 hours and by 46% and 56% at 15 hours, respectively, in the BLU2864-treated mice compared with controls. Animal Model: Pkd1 RC/RC mice [1] Dosage: 30 mg/kg Administration: Oral gavage; 30 mg/kg; once daily; 5 days Result: Showed higher urine outputs at 15 weeks in the BLU2864-treated mice than in the controls. Showed lower kidney weights, kidney volumes as percent of body weights, and cyst indices. Showed renal basal and total PKA activities by 69% and 84% lower in the BLU2864-treated mice compared with controls. Animal Model: Mice harboring FLC PDX tumors [2] Dosage: 30 mg/kg and 75 mg/kg Administration: Oral gavage; 30 mg/kg and 75 mg/kg; once daily; 34 days Result: Inhibited tumor growth by 48.5% (P=0.003) and by 45.3% (P=0.0005), respectively, at day 34.