Pioneering a new era in cholesterol management
~1 billion
people affected by high cholesterol worldwide
~100 million
adults in the United States are affected by high cholesterol
~18.6 million
lives lost annually due to Atherosclerotic Cardiovascular Disease (ASCVD)
#1
Cardiovascular Disease is the number 1 cause of death worldwide, responsible for ~31% of all annual global deaths
Hypercholesterolemia (high cholesterol), particularly elevated Low-Density Lipoprotein Cholesterol (LDL-C), is a major causal risk factor for atherosclerotic cardiovascular disease (ASCVD), a leading form of cardiovascular disease that includes coronary artery disease, heart attack, and ischemic stroke.
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The liver’s dual role in cholesterol management
The liver plays a central role in cholesterol regulation. It produces cholesterol, an essential component used in the synthesis of hormones, vitamin D, and bile acids, while also removing excess LDL cholesterol from the bloodstream. Maintaining the balance between cholesterol production and cholesterol clearance is critical for cardiovascular health.
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LDL-R & PCSK9
Low-Density Lipoprotein Receptors (LDL-R), located on the surface of liver cells, play a critical role in cholesterol regulation. These receptors bind circulating LDL cholesterol (“bad” cholesterol) and remove it from the bloodstream. Under normal conditions, LDL-R can be recycled back to the cell surface and reused multiple times.
Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9), a protein produced primarily by the liver, regulates this process. When PCSK9 binds to LDL-R, it directs the receptor toward degradation rather than recycling, reducing the liver’s ability to remove LDL cholesterol from circulation.
Higher PCSK9 activity results in fewer LDL receptors being recycled back to the liver cell surface. As receptor availability decreases, LDL clearance declines and blood LDL cholesterol levels rise, contributing to the development of atherosclerosis.
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How PCSK9 inhibitors (PCSK9i) are transforming cholesterol treatment
PCSK9-targeting therapies help preserve LDL receptor recycling by preventing PCSK9 from promoting LDL-R degradation. As more receptors remain available on the liver cell surface, the liver can remove LDL cholesterol more efficiently from the bloodstream.
The clinical success of PCSK9-targeting therapies has further validated the importance of the PCSK9–LDL-R pathway in cholesterol management and cardiovascular risk reduction.
These advances have created growing interest in new approaches to modulating the PCSK9–LDL-R pathway, including investigational therapies designed to act through differentiated mechanisms.
PRALUENT
Regeneron & Sanofi
REPATHA
Amgen
LEQVIO
Novartis
LIPFENDRA
Merck & Co.
LEROCHOL
Lib Therapeutics
Together, these therapies generate over $4 billion annually and have become a preferred treatment option for cholesterol management in high-risk patients, thanks to their superior efficacy, fewer side effects and contraindications compared to statins.
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Advancing cholesterol management with AQR-008
At Aqur, we are developing AQR-008, an investigational peptide designed to modulate the PCSK9–LDL-R pathway, with the goal of lowering LDL cholesterol and ultimately reducing the risk of atherosclerotic cardiovascular disease.
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A differentiated approach to the PCSK9–LDL-R pathway
Unlike existing approaches that directly target PCSK9 or reduce its production, AQR-008 is designed to act from the LDL-R side of the PCSK9–LDL-R interaction.
AQR-008 is designed to target the EGF-A domain of the LDL receptor (LDL-R), potentially interfering with PCSK9 binding while preserving LDL-R recycling.
By supporting LDL-R recycling, AQR-008 is designed to increase functional receptor availability and potentially enhance LDL uptake and clearance from the bloodstream.
AQR-008 is not designed to suppress PCSK9 production or directly target circulating PCSK9, potentially allowing other biological functions of PCSK9 to remain unaffected.
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Ongoing Preclinical Development
AQR-008 is in early preclinical development, and its proposed mechanism of action is subject to further preclinical and clinical evaluation.
As development progresses, we continue to evaluate and optimize AQR-008 and related peptide variants. Accordingly, the peptide sequence ultimately selected for further development under the AQR-008 designation may differ from the sequence evaluated in studies to date.
Through this differentiated mechanism of action, AQR-008 has the potential to introduce a new approach to LDL-C lowering and expand future treatment options for patients with hypercholesterolemia and those at risk of ASCVD.
Merck’s
LIPFENDRA
LIPFENDRA, recently approved by the FDA, is an oral cyclic peptide PCSK9 inhibitor.
AstraZeneca’s AZD0780
AstraZeneca is developing AZD0780, an oral small-molecule PCSK9 inhibitor currently in early-stage trials.
Both LIPFENDRA and AZD0780 share the same mechanism of action (MoA), binding to PCSK9—similar to the approved and commercialized injectable inhibitors Praluent (Sanofi/Regeneron) and Repatha (Amgen), which are already on the market.
At Aqur, we’re not just lowering cholesterol ...
... we’re saving lives, one breakthrough at a time!
AQR-008 marks the commencement of Aqur’s journey
We are committed to expanding our drug pipeline with high-impact therapies targeting cardiovascular disease and other critical health challenges. Our goal is to build a comprehensive portfolio that addresses unmet medical needs and enhances patient care.
Beyond cardiovascular health - exploring new frontiers
Published research suggests broader biological roles for the PCSK9 pathway. Aqur may evaluate additional indications after establishing the pharmacology and development path of AQR-008 in hypercholesterolemia.
Aqur: Leading the way in the future of cholesterol management
Guided by scientific research and focused on patient care, we are not merely following innovation—we are creating it.
