Drug intelligence / Profile preview

DSM265

Development stage
Phase 2
Lead developer
Medicines for Malaria Venture
Modality
Small Molecules
Administration
Oral
01

Overview

DSM265 is an orally active investigational small molecule antimalarial drug that selectively inhibits the dihydroorotate dehydrogenase (DHODH) enzyme of *Plasmodium* species. DHODH is essential for pyrimidine biosynthesis in the malaria parasite and is absent from human salvage pathways. By targeting this enzyme with high selectivity for the parasite form over the human equivalent, DSM265 disrupts both liver and blood stages of *Plasmodium falciparum*—including strains resistant to current therapies—making it a promising candidate for both treatment and chemoprophylaxis of malaria. The compound has demonstrated favorable pharmacokinetics with a long elimination half-life suitable for once-weekly dosing. Clinical trials have shown good tolerability but limited pre-erythrocytic protection as monotherapy; further development may require combination therapy or alternative dosing strategies. The drug was discovered through collaborations involving academic institutions and Medicines for Malaria Venture (MMV), with Takeda Pharmaceutical Company also supporting its development[1][3][4][5][6][7].

Other names
2-(1,1-difluoroethyl)-5-methyl-N-[4-(pentafluoro-lambda6-sulfanyl)phenyl]-[1,2,4]triazolo[1,5-a]pyrimidin-7-amine2-(1,1-Difluoroethyl)-5-methyl-N-[4-(pentafluoro-λ6-sulfanyl)phenyl][1,2,4]triazolo[1,5-a]pyrimidin-7-amine
02

Targets

DHODH (Dihydroorotate dehydrogenase (quinone), mitochondrial)PfDHODH (Plasmodium falciparum dihydroorotate dehydrogenase)

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