PARP-1-IN-1 is a highly selective and orally active inhibitor of the enzyme PARP-1, with an IC50 value of 0.96 nM. It exhibits excellent tolerance and demonstrates significant activity in a single dose in the MDA-MB-436 xenotransplantation model [1].

PARP-1-IN-1 (compound Y49) (48 hours) has effective inhibition on different cancer cells ( IC 50 s of 9.64, 123.5 106.3 μM for MX-1, MCF7 and A548 cells, respectively) [1]. PARP-1-IN-1 (2.5-10 μM, 48 hours) successfully inhibits the activity of PARP-1 and reduces the production of PAR in A549 cells, which is dose-dependent to some extent [1]. Western Blot Analysis [1] Cell Line: A549 cells Concentration: 2.5 μM, 5 μM and 10 μM Incubation Time: 48h Result: Inhibited the activity of PARP-1 successfully and reduced the production of PAR in cancer cells. PARP-1-IN-1 was dose-dependent to some extent.

PARP-1-IN-1 (compound Y49) (50 mg/kg/day; p.o.; daily for 18 days) inhibits the growth of MDA-MB-436 tumor in BALB/c nude mice [1]. Animal Model: Female athymic BALB/c nude mice (inoculated MDA-MB-436 cells) [1] Dosage: 50 mg/kg Administration: p.o., daily for 18 days Result: Inhibited the growth of MDA-MB-436 tumor significantly, and no significant change in the body weight of PARP-1-IN-1 treated mice.