Phase III ELIPSE HoFH trial results of REGN 1500 met primary endpoint in homozygous familial hypercholesterolemia
The data showed the trial met its primary endpoint, with patients who added evinacumab to other lipid-lowering therapies reducing their LDL-C by 49% from baseline at 24 weeks compared to the placebo group, who received other lipid-lowering therapies alone, the primary endpoint of the trial (p<0.0001). Nearly all (95%) patients in the evinacumab arm entered the trial on statins and 79% were on pcsk9 (proprotein convertase subtilisin kexin type 9) inhibitors. Nearly half of evinacumab-treated patients reduced ldl-c to under 100 mg dl (nominal p="0.0203)," despite entering the trial with average ldl-c levels of 260 mg dl on other lipid-lowering therapies. In the trial, evinacumab was generally well-tolerated. During the double-blind treatment period, 66% of evinacumab patients and 81% of placebo patients experienced at least one adverse event (ae). During the double-blind treatment period, aes that occurred in at least 5% of patients, and more commonly with evinacumab, were influenza-like illness (11% evinacumab, 0% placebo) and rhinorrhea (7% evinacumab, 0% placebo). There were no deaths, major adverse cardiovascular events or discontinuations due to aes. detailed results from this trial will be used as the basis of regulatory submissions around the world, with the FDA submission expected to be completed by mid-2020. Evinacumab is an investigational fully-human monoclonal antibody that binds to and blocks the function of angiopoietin-like 3 (angptl3), in patients with homozygous familial hypercholesterolemia (HoFH). Regeneron previously announced topline positive results of this trial in august 2019. Data were presented as a late-breaking presentation at the american college of cardiology's annual scientific session together with world congress of cardiology (acc.20).></0.0001).>
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Regeneron Pharmaceuticals, Inc. announced positive results from a Phase III trial evaluating Evkeeza (evinacumab) in children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH).
The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation under exceptional circumstances for the medicinal product Evkeeza, from Regeneron, intended for the treatment of adult and adolescent patients aged 12 years and older with homozygous familial hypercholesterolaemia (HoFH).Evkeeza will be available as concentrate for solution for infusion (150 mg/ml).
The FDA has approved Praluent (alirocumab), from Regeneron, as an adjunct to other low-density lipoprotein cholesterol (LDL-C)-lowering therapies in adults with homozygous familial hypercholesterolemia (HoFH) to reduce LDL-C.