Beyond traditional PCSK9 inhibition
A next-generation approach to PCSK9 inhibition
Supported by a robust patent portfolio that includes over 50 peptides derived from the PCSK9’s catalytic domain, our lead candidate, AQR-008, is designed to deliver a more precise and effective approach to PCSK9 modulation.
Unlike traditional PCSK9 inhibitors, AQR-008 is designed to:
TARGET ONLY LDL RECEPTORS, WITHOUT INTERFERING WITH PCSK9 SYNTHESIS OR FUNCTIONS.
EFFECTIVELY LOWER LDL-C LEVELS IN A BROADER PATIENT POPULATION.
PROVIDE A POWERFUL SOLUTION FOR HYPERCHOLESTEROLEMIA, INCLUDING SEVERE OR COMPLEX CONDITIONS SUCH AS FAMILIAL HYPERCHOLESTEROLEMIA AND EXCEPTIONALLY HIGH LDL-C LEVELS.
AQR-008’s unique MoA
AQR-008 follows a distinct approach, with streamlined development based on a validated clinical pathway:
Targets the EGF-A domain on the LDL receptor (LDL-R)
It does not bind to or interfere with PCSK9 synthesis
Does not interfere with other PCSK9 functions
Why this matters
By avoiding full PCSK9 suppression, AQR-008 enhances LDL receptor activity, enabling:
More effective LDL cholesterol (LDL-C) reduction.
Enhanced LDL-R recycling leading to reduced LDL-C levels.
A new treatment for hypercholesterolemia, high-risk and statin-intolerant patients.
AQR-008: the future of LDL-C lowering · This orthogonal mechanism sets AQR-008 apart from existing PCSK9 inhibitors, aiming to offer a highly effective cholesterol management therapy that can be used alone or in combination with other treatments.
Innovation with the patient in mind
ORAL DELIVERY
In contrast to injectable alternatives, our medication is meticulously designed with the anticipation of oral administration. We anticipate that this approach will enhance patient adherence and accessibility.
COST-EFFECTIVE PRODUCTION
Our streamlined development process facilitates cost-effective manufacturing, ensuring affordability.
CONVENIENT STORAGE
The drug can be stored without refrigeration, making it suitable for patients across diverse geographic and economic conditions.
POTENTIAL ADDITIONAL FUNCTIONS
Should research confirm that PCSK9 has additional functions within the body, Aqur’s precision-focused approach could provide a distinct and substantial advantage over existing therapies.
STRATEGIC DEVELOPMENT PLAN
Currently in the early preclinical development phase, Aqur is implementing a comprehensive strategic plan. Our development roadmap encompasses the following phases:
Collaborating with AI
Formulation development
Bioanalytical method development
In vitro and in vivo peptide profiling
Preclinical (PoM) studies
Preclinical toxicology studies & IND submission
Merck’s
MK-0616
Merck & Co., Inc. is currently advancing its oral cyclic peptide PCSK9 inhibitor, MK-0616, through Phase 3 clinical trials.
AstraZeneca’s AZD0780
AstraZeneca is developing AZD0780, an oral small-molecule PCSK9 inhibitor currently in early-stage trials.
Both drugs in development 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.
Aqur’s AQR-008 stands out as a unique approach
Unlike existing treatments that either reduce PCSK9 production or inhibit its functions, AQR-008 employs a more novel approach:
Targets only the LDL-R and does not bind with nor interfere with PCSK9.
Preserves LDL-R function, allowing it to clear LDL from the bloodstream.
Prevents LDL-R degradation, allowing them to be recycled and continue to clear LDL.
Maintains stable blood levels, avoiding the fluctuations seen with other PCSK9i for more consistent results.
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