Pharmacological induction of KLF2 and reversal of endothelial dysfunction for the treatment of hypertension

NIH RePORTER · NIH · R43 · $310,743 · view on reporter.nih.gov ↗

Abstract

PROJECT SUMMARY There is a clear unmet need for new differentiated anti-hypertensive therapies, as high blood pressure and its pathological sequelae remain significant burdens to human health despite several classes of approved drugs. The vascular endothelium is a dynamic interface that regulates vasotone, inflammation, hemostasis and vascular remodeling. Dysfunction of the vascular endothelium, including vasoconstriction, impaired vasoreactivity, inflammation, thrombosis and loss of vascular quiescence, is a key driver of many vascular diseases. Riparian Pharmaceuticals recently discovered novel small molecules that target endothelial dysfunction by activating the endothelial Krüppel-like factor 2 (KLF2) pathway, a key node of vasoprotection. The transcription factor KLF2 is an upstream regulator of critical vasodilatory, anti-inflammatory, anti-coagulatory and homeostatic genes. KLF2 promotes vasodilation and endothelial function by several mediators but a principal mechanism is the transactivation of the endothelial nitric oxide synthase (eNOS) gene. eNOS and its product NO are widely appreciated key components of vascular function having vasodilatory, anti-coagulatory and anti-inflammatory effects. We believe KLF2 induction is a promising new therapeutic approach to the widely studied but persistent challenge of reduced NO bioavailability in hypertension. We have extensively studied the pharmacology of our first-in-class KLF2-inducing therapeutic program. In this project, we plan to validate our therapeutic hypothesis in established hypertensive rat models. We hypothesize that KLF2 and eNOS induction by our lead candidate will promote a vasoprotective phenotype, improve endothelial function and lower blood pressure in normotensive and hypertensive animals. Success here will advance this program into IND-enabling studies and clinical evaluation of a new therapeutic approach to hypertension.

Key facts

NIH application ID
10484143
Project number
1R43HL164171-01
Recipient
RIPARIAN PHARMACEUTICALS, INC.
Principal Investigator
William James Adams
Activity code
R43
Funding institute
NIH
Fiscal year
2022
Award amount
$310,743
Award type
1
Project period
2022-05-01 → 2024-04-30