AstraZeneca's Oral Small Molecule PCSK9 Inhibitor AZD0780 Granted Clinical Trial Approval in China for Hyperlipidemia

Sep 11, 2024 14:30 CST Updated 14:30

AstraZeneca

Biopharmaceutical Manufacturer

▎WuXi AppTec Content Team Report

Today (September 11), the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China) Official website announcement,AstraZeneca's AZD0780 Film-Coated Tablets Receive Clinical Trial Implied Permission, Intended for Use inOn the basis of standard treatmentLow-Density Lipoprotein Cholesterol (LDL-C)Treatment of Dyslipidemia in Patients Not Meeting Targets。

Public information shows that AZD0780 is aOral PCSK9 Small Molecule Inhibitor。According to the Phase 1 clinical research data published by AstraZeneca in May this year, when AZD0780 is used in combination with the statin rosuvastatin,Can reduce patients' LDL-C levels by nearly 80%。The product is currently in Phase 2 clinical trials internationally, and this marks its first approval for clinical trials in China.

Screenshot source: CDE official website

Elevated LDL-C levels in plasma are a major risk factor for cardiovascular diseases, estimated to cause approximately 2.6 million deaths globally each year. Despite the availability of various approved therapies, the global burden of dyslipidemia continues to rise. Over 70% of patients with atherosclerotic cardiovascular disease (ASCVD) still do not achieve their LDL-C target values, indicating an unmet need for more diverse and effective treatment options for high-risk patients.

In May this year, AstraZeneca announced positive results from the Phase 1 clinical trial of AZD0780 at the European Atherosclerosis Society (EAS). The study evaluated the pharmacodynamics, pharmacokinetics, safety, and tolerability of AZD0780 as a monotherapy and in combination with rosuvastatin on plasma LDL-C levels in untreated patients with hypercholesterolemia.

Analysis shows that in untreated patients with hypercholesterolemia, AZD0780 demonstrated a significant 52% reduction in LDL-C levels on the basis of rosuvastatin treatment.The overall reduction in patients' LDL-C compared to baseline reached 78%.In addition, preliminary data comparing fed and fasting conditions suggest that AZD0780 has flexibility with regard to food intake. AZD0780 also demonstrated good tolerability.

PCSK9It is an emerging target in the field of lipid-lowering drug development. Inhibiting PCSK9 can reduce LDL-C levels in the body, thereby achieving the goal of treating diseases. Globally, monoclonal antibody therapies and siRNA therapies targeting PCSK9 have previously been approved for marketing.As progress advances, scientists have begun to explore the development of more drug types targeting PCSK9, includingSmall interfering RNA (siRNA), Antisense oligonucleotides (ASO), Gene editing therapy, Vaccines,Small molecules,Antibody-like protein drugsEtc.。

Among them, small-molecule drugs are highly anticipated due to their convenient administration and ease of combination with other medications. The small-molecule PCSK9 inhibitor developed by Merck (MSD) was previously...Selected as the DrugHunter.com 2023 AnnualTop Ten "Star" Small MoleculesOne of.

AstraZeneca's small molecule PCSK9 inhibitor has been approved for clinical trials in China, marking a new advancement in the PCSK9 target field.

References:

[1] Official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China. Retrieved Sep 11, 2024, from https://www.cde.org.cn/main/xxgk/listpage/9f9c74c73e0f8f56a8bfbc646055026d

[2] AZD0780, an oral PCSK9 inhibitor demonstrated significant LDL-C reduction on top of statin in Phase I trial. Retrieved May 29, 2024 from https://www.astrazeneca.com/media-centre/medical-releases/azd0780-an-oral-pcsk9-inhibitor-demonstrated-significant-ldl-c-reduction-on-top-of-statin-in-phase-i-trial.html

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