PTC299 是一种口服活性的 VEGFA mRNA 翻译抑制剂，可选择性地抑制转录后水平的 VEGF 蛋白合成。它还是二氢乳酸脱氢酶 (DHODH) 的有效抑制剂，可研究血液系统恶性肿瘤。

PTC299 is an orally active inhibitor of VEGFA mRNA translation and selectively inhibits VEGF protein synthesis at the post-transcriptional level. PTC299 is also a potent inhibitor of dihydrolactate dehydrogenase (DHODH).

PTC299 as an inhibitor of VEGFA mRNA translation in a phenotypic screen and evaluated in the clinic for treatment of solid tumors.?To guide precision cancer treatment, we performed extensive biological characterization of the activity of PTC299 and demonstrated that inhibition of VEGF production and cell proliferation by PTC299 is linked to a decrease in uridine nucleotides by targeting dihydroorotate dehydrogenase (DHODH), a rate-limiting enzyme for de novo pyrimidine nucleotide synthesis.

1256565-36-2

C25H20Cl2N2O3

467.34

(4-chlorophenyl) (1S)-6-chloro-1-(4-methoxyphenyl)-1,3,4,9-tetrahydropyrido[3,4-b]indole-2-carboxylate;Emvododstat

DMSO:50 mg/mL (106.99 mM),Need ultrasonic
Powder: -20°C for 3 years
In solvent: -80°C for 2 years