Regeneron announced top-line results of a phase 3 study showing that evinacumab, its investigational angiopoietin-like 3 antibody, reduced LDL by 49% in patients with homozygous familial hypercholesterolemia who were taking other medications.
The cohort included patients with familial hypercholesterolemia (HoFH), who are at very high risk for CVD events as early as adolescence, and who had mean LDL of 255 mg/dL despite receiving treatments such as maximally tolerated statins, PCSK9 inhibitors, ezetimibe, LDL apheresis and lomitapide (Juxtapid, Aegerion Pharmaceuticals), according to a press release issued by the company.
“People born with homozygous familial hypercholesterolemia — the rare and most severe form of FH — are in urgent need of additional therapies to lower life-threatening cholesterol levels. HoFH causes aggressive heart disease even in childhood, and today’s treatments often are not enough for these individuals,” Katherine Wilemon, founder and CEO of the FH Foundation, said in the release. “These evinacumab phase 3 results bring hope to those who need it most. The FH Foundation is grateful to the researchers who advanced this science and the individuals who participated in this clinical trial.”
According to the release, evinacumab met the primary endpoint of LDL reduction at 24 weeks compared with placebo (evinacumab, 47% reduction; placebo, 2% increase; P < .0001).
In addition, the company stated in the release, evinacumab was associated with an absolute change in LDL from baseline of 132 mg/dL (evinacumab, reduction of 135 mg/dL; placebo, increase of 3 mg/dL; P < .0001), whereas 47% of the evinacumab group achieved LDL of less than 100 mg/dL compared with 23% of the placebo group (nominal P = .0203).
The results were consistent in “null/null” patients, considered to be the most difficult to treat, according to the release.
In secondary endpoints, evinacumab was associated with reductions in apolipoprotein B, non-HDL and total cholesterol compared with placebo, according to the release.
The full data are expected to be presented at a medical meeting in 2020, a company spokesperson told Healio.
Evinacumab received a breakthrough therapy designation from the FDA in 2017, which enables development and review to be expedited, but is not yet approved for commercial use in the United States.
Disclosure: The study was funded by Regeneron. Wilemon is an employee of the FH Foundation.