A First in man study to Evaluate the Safety, Pharmacokinetics, and
Pharmacodynamics of RP7214, a Dihydroorotate Dehydrogenase (DHODH)
inhibitor in Healthy Subjects
Abstract
RP7214 is a potent and selective inhibitor of human mitochondrial enzyme
dihydroorotate dehydrogenase (DHODH). This paper describes the results
from a Phase 1 study that evaluated safety and pharmacokinetics of
single and multiple ascending doses (SAD and MAD) and the food effect of
RP7214 in healthy subjects. Target engagement of DHODH was also
evaluated. A randomized, double-blind, placebo-controlled trial of
single-dose (100, 200, and 400 mg QD) and multiple doses (200 and 400 mg
BID for 7 days) followed by food effect at a single dose of 200 mg was
conducted. A total of 18 healthy volunteers (HVs) (6 subjects in each of
three cohorts) in the SAD part, 12 (6 subjects each in two cohorts) in
the MAD part, and 12 in the food effect study were enrolled. RP7214 was
well tolerated at all dose levels. None of the subjects reported any
RP7214-related adverse events. RP7214 showed dose-proportional
pharmacokinetics after single and multiple dosing. Steady-state
concentrations were reached within about 3–6 days. The mean plasma
half-life of RP724 at steady-state was approximately 13h. RP7214 showed
accumulation on multiple dosing.. Food did not impact the absorption of
RP7214. RP7214 showed dose-dependent inhibition of DHODH as measured by
analyzing accumulating DHO levels, confirming target engagement. The
rapid absorption and high systemic exposure of RP724 with a favorable
safety profile shows the potential for the development of RP7214 in
SARS-CoV-2 infection and acute myeloid leukemia. (NCT04680429).
Keywords: RP7214, dihydroorotate dehydrogenase, SAD, MAD, HV