MONOGENIC PHARMACEUTICALS R&D
and Competitive Advantages
•Orally available, highly potent, non-toxic and hepatocyte-specific small molecule LDLR enhancers.
•New class of drugs & strong IP strategy.
•Low production cost with high market penetration potential.
•Proof-of-concept of our lead candidate MONO4-025 in hypercholesterolemic animal models (comparable to PCSK9 inhibitors; up to -75% in LDL), well-tolerated with no apparent adverse events and hepatotoxicity.
Unmet Clinical Needs for New LDLR Enhancers
and Market Opportunity
•High blood LDL-Cholesterol is a major contributor to heart attacks and Stroke (№ 1 cause of death globally ~17M/year).
•Statins reduce LDL-Cholesterol and prevent heart attacks by increasing LDL Receptor (LDLR) gene expression.
•~45M high risk patients (HeFH or ASCVD in US+EU) have uncontrolled cholesterol despite changing their diets, exercising and on maximally tolerated statin therapy.
•Injectable PCSK9 mAbs (FDA-approved 2015), only high LDLR enhancer alternatives, have shown that further LDL-lowering is safe and more cardioprotective; however not cost-effective.
•Urgent clinical need for new, oral and affordable LDL-lowering drugs.