Amjin PCSK9 inhibitor Repatha pediatric phase 3b study reached the main endpoint.

Guided reading: Studies have shown that Repatha significantly lowers LDL-C levels compared to placebos when combined with statins and other fat-lowering therapies.
August 27-29, 2020, the Annual Meeting of the European Society of Hepatology (EASL) and the Digital International Liver Congress (DILC) were held as an online conference, the first annual event held by EASL in the form of a "digital conference" to attract scientific and medical experts from all over the world to learn about the latest developments in liver research and to exchange clinical experience.
August 29, Amgen released positive data from a study of the cholesterol-lowering drug Repatha (Chinese commodity name: Rebean, common name: evolocumab, eloyu monoantigen) for the treatment of 10-17-year-old heterocyctic hypercholesterolemia (HeFH) pediatric patients.
studies have shown that Repatha significantly lowers LDL-C levels compared to placebos when combined with statins and other fat-lowering therapies.
data were published at the 2020 Annual Meeting of the European Society of Cardiology (ESC) from August 29 to September 1, and published simultaneously in the New England Journal of Medicine.
HeFH is a genetic disorder in which children are born with high levels of LDL-C, which accelerates the development of ASCVD and leads to an overall increased risk of cardiovascular events.
with familial hypercholesterolemia (FH) are about 20 times more likely to develop heart disease than the general population.
children with FH are still at high levels with normal weight, a good diet and plenty of exercise, and are at risk of cardiovascular events from a very young age.
HAUSER-RCT was a 3b-, multi-center, randomized (2:1), double-blind, placebo-controlled study conducted in Patients aged 10-17 with HeFH to assess the efficacy, safety and tolerability of a monthly subsurface injection of Repatha 420mg (n=104) and a placebo (n=53) treatment for 24 weeks.
study were randomly grouped into LDL-C (4.1 vs .4.1mmol/L) and age (14 years vs 14 years old).
key eligibility criteria include receiving a low-fat diet and maximum toned dose of lipid-lowering therapy (LLT) for up to 4 weeks before screening, but an empty-stomach LDL-C level of 3.4 mmol/L.
The main endpoint is a percentage change from baseline to treatment week 24 LDL-C level; Percentage change in non-HDL cholesterol (non-HDL-C), lipoprotein B (ApoB), total cholesterol/HDL cholesterol (HDL-C) ratio, ApoB/lipoprotein A1 (ApoA1) ratio at week 24.
safety assessments include Tanner stages, hormone levels, the thickness of the endometrium of the carotid artery, and computer-based cognitive assessments.
results showed that the study reached its main endpoint: in week 24 of treatment, the LDL-C levels in the Repatha group decreased by an average of 38.3% compared to the baseline and the LDL level decreased by an absolute 68.6 mg/dL compared to the placebo group.
, the Repatha group also showed improvement relative to the baseline in terms of secondary lipid parameters compared to the placebo group, including a 42.1% decrease in the average LDL-C for 22-24 weeks, a 35.0% decrease in non-HDL-C level in week 24, a 32.5% decrease in the ApoB level in week 24, and a 36.4% decrease in the ApoB/ApoA1 ratio in week 24.
no new security risks were found in the study.
the Most Common Adverse Events (TEAE, sgt;2%) that occurred during treatment in the Repatha group were higher than in the placebo group (TEAE, .gt;2%), including headache, sore throat, influenza, influenza-type diseases, upper respiratory tract infections, and constipation.
to effectively manage LDL-C levels in Children with HeFH, which will help slow the development of cardiovascular disease.
data from the HAUSER-RCT study confirm that Repatha is a safe and effective treatment for Children with HeFH who are already receiving fat-lowering treatment but need to further reduce LDL-C.
Cholesterol-lowering new drug competition pattern Repatha is a targeted pre-human protein-converting enzyme oxalolysis/kexin type 9 (PCSK9) human monoclonal antibody, which binds to PCSK9 to inhibit the binding of PCSK9 in the cycle with low-density lipoprotein (LDLR) and prevents PCSK9-mediated LDLR degradation, allowing LDLR to cycle back to the surface of liver cells.
by inhibiting the combination of PCSK9 and LDLR, Repatha increases the amount of LDLR that can remove LDL from the blood, thereby reducing LDL-C levels.
PCSK9 inhibition is a major breakthrough in cholesterol reduction after statins.
, two single-resistant PCSK9 inhibitors have been approved, and the other is Sanofi/Recycled Praluent.
two drugs are biologic and expensive, so there has been a long and fierce price war since the market to compete for market dominance.
success of this pediatric study is important for Amjin, which will help expand Repatha's patient pool from adults to adolescents.
Comes at a time of fierce competition between the two sides, Novart, a laterr, is also hoping to enter the PCSK9 market with siRNA therapy inclisiran, a $9.7 billion acquisition of TMC's core asset, which requires only two subsurfic injections a year and is under review in the UNITED States and the European Union.
At the same time, three drugs are facing new challenges, namely Esperion's pioneering ACL inhibitor Nexletol and its compound drug, Nexlizet, which has a new cholesterol-lowering mechanism and was approved by the United States and the European Union in the first half of this year for a fraction of the price of PCSK9 single resistance.
predictable that competition will intensify in the cholesterol-lowering drug market.
source: 1. Amgen Announces Positive Data From Phase 3B Study Of Repatha? (Evolocumab) In Pediatrics Patients With Heterozygous Familial Hyperisle At ESC Congress 20202.ESC: Amgen Chases pediatrics use for spite-busting PCSK9 med Repatha.