Health AI Central
Peer-Reviewed Publication
Front Oncol2022;12832816.January 1, 2022Journal Article

Emvododstat, a Potent Dihydroorotate Dehydrogenase Inhibitor, Is Effective in Preclinical Models of Acute Myeloid Leukemia.

Arthur Branstrom1, Liangxian Cao1, Bansri Furia1, Christopher Trotta1, Marianne Santaguida2, Jason D Graci1, Joseph M Colacino1, Balmiki Ray1, Wencheng Li1, Josephine Sheedy1, Anna Mollin1, Shirley Yeh1, Ronald Kong1, Richard Sheridan3, John D Baird4, Kylie O'Keefe5, Robert Spiegel1, Elizabeth Goodwin6, Suzanne Keating6, Marla Weetall1
1Research, PTC Therapeutics, Inc., South Plainfield, NJ, United States.
2Notable Labs, Foster City, CA, United States.
3InSeption Group, Lansdale, PA, United States.
4Clinical, PTC Therapeutics, Inc., South Plainfield, NJ, United States.
5Commercial, PTC Therapeutics, Inc., South Plainfield, NJ, United States.
6Scientific Writing, PTC Therapeutics, Inc., South Plainfield, NJ, United States.
PubMed PMC DOI PDF

Abstract

Blocking the pyrimidine nucleotide de novo synthesis pathway by inhibiting dihydroorotate dehydrogenase (DHODH) results in the cell cycle arrest and/or differentiation of rapidly proliferating cells including activated lymphocytes, cancer cells, or virally infected cells. Emvododstat (PTC299) is an orally bioavailable small molecule that inhibits DHODH. We evaluated the potential for emvododstat t…

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