MONOGENIC PHARMACEUTICALS R&D ​​
​and Competitive Advantages​

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•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.​

MONO4-025

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Unmet Clinical Needs for New LDLR Enhancers

and Market Opportunity

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•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.


DMSO