Regeneron's Evinacumab Fights Ultra-Rare Form of High Cholesterol in Kids
Regeneron's Evinacumab showed positive results against HoFH. (Courtesy of Lev Radin/Getty Images)
Regeneron Pharmaceuticals shared positive results from its Phase III trial of a candidate drug for children with homozygous familial hypercholesterolemia (HoFH), a rare, life-threatening condition characterized by elevated circulating levels of low-density lipoprotein cholesterol (LDL-C) and accelerated, premature atherosclerotic cardiovascular disease (ACVD.
The latest Phase III trial results evaluated Evkeeza to treat HoFH, an inherited disease that is also the most severe form of familial hypercholesterolemia. HoFH affects one in 160,000 to 300,000 people worldwide, and around 1,300 people in the United States. Those diagnosed with the disease, including patients as young as teenagers, are at high risk for premature atherosclerotic disease and life-threatening cardiac events.
Evekeeza (evinacumab) was developed using Regeneron's VelocImmune technology, which uses a genetically-engineered mouse platform with a humanized immune system to create fully human antibodies. It's a fully human monoclonal antibody that binds to and blocks ANGPTL3, inhibiting lipoprotein lipase and endothelial lipase and regulating LDL-C and other circulating lipids.
Its generic name for approved indications in the U.S. is evinacumab-dgnb, with dgnb given per the U.S. Food and Drug Administration-issued Nonproprietary Naming of Biological Products Guidance of Industry. Evekeeza's effectiveness and safety have yet to be established in patients with other hypercholesterolemia causes, including those with heterozygous familial hypercholesterolemia (HeFH).
In Regeneron's study, the drug was tested in kids ages 5 to 11 years old. It met the primary endpoint of reducing the low-density lipoprotein cholesterol by 48% on the 24th week. About 79% of participants saw their LDL-C drop by at least 50% at week 24, also logging an average reduction of 132 mg/dL in LDL-C from baseline. All levels of lipid endpoint parameters also saw declines within the first eight weeks.
Some adverse events, mostly minor, were observed, including throat pain, diarrhea, abdominal pain, nasopharyngitis and headaches. There were two serious adverse events observed, aortic stenosis and tonsillitis, but these were unrelated to the treatment. Overall, Evkeeza was well-tolerated by all participants.
"Children living with HoFH have an incredibly rare and severe disease that causes dangerously high LDL-C levels. On current treatment options alone, many patients don't reach their treatment goals, leaving them with an uncertain future. Evinacumab has already demonstrated significant LDL-C reductions in adolescents and adults with HoFH. This latest Phase 3 trial illustrates the potential of this medicine to be a breakthrough HoFH therapy for children as young as 5-years old, helping them control their LDL-C early in the course of their disease," M. Doortje Reijman, M.D., the trial's lead investigator, said in a statement. Dr. Reijman is also a research associate in pediatric metabolic diseases and nephrology at Amsterdam University Medical Center.
The clinical trial using Evkeeza (Evinacumab) for pediatric patients living with HoFH is ongoing. Regeneron is taking care of development and distribution in the U.S. and is collaborating with Ultragenyx for activities outside the country.