Expert: Fully human IgG4 monoclonal antibody targeting angiopoietin-like protein 3 (ANGPTL3). Inhibits LPL and endothelial lipase, reducing LDL-C, triglycerides, and HDL-C. Works independently of LDLR — critical for HoFH patients with no functional LDL receptors.

Everyday: Blocks ANGPTL3, a protein that prevents your body from clearing fats from the blood. With ANGPTL3 blocked, your body can clear LDL, triglycerides, and other lipids more efficiently — works even when LDL receptors are completely absent (HoFH).

Targets: ["ANGPTL3"]

Primary EP: [{"id":"elipse-ldlc","name":"Percent change in LDL-C at Week 24","type":"PRIMARY","unit":"%","results":[{"notes":"-47% LDL-C reduction from baseline","value":-47,"arm_id":"evinacumab-elipse","arm_name

Efficacy: ELIPSE met primary endpoint: -47% LDL-C reduction with evinacumab vs +2% placebo (difference -49%, p<0.0001). Also reduced ApoB -37%, TG -50%, non-HDL-C -52%. First therapy to work independently of LDL receptors — critical for HoFH patients with no functional LDLR.

Safety: ELIPSE (n=65, 24 weeks): Nasopharyngitis 16%, influenza-like 7%, infusion reactions 7%, anaphylaxis 1% (1 patient). Discontinuation 2%. Overall well-tolerated for an ultra-rare disease population.

First-in-class ANGPTL3 inhibitor for HoFH. Works independently of LDL receptors — critical for patients with no functional LDLR (HoFH). +29% YoY. Small ultra-rare disease market (~1,300 US patients) but important for complete REGN lipid franchise story.

{"keyRisks":[{"category":"Anaphylaxis","description":"Anaphylaxis reported in 1 patient (1%) in ELIPSE. Observe during infusion.","incidenceRate":"1%"},{"category":"Infusion-related reactions","description":"Occurred in 7% of patients. Symptoms include pyrexia, chills, nausea, flushing, headache.","incidenceRate":"7%"},{"category":"Nasopharyngitis","description":"Most common AE reported at higher rate than placebo.","incidenceRate":"16%"},{"category":"Embryo-fetal toxicity","description":"Animal data suggests risk. Verify pregnancy status before initiating. Use contraception.","incidenceRate":"Animal data — contraindicated in pregnancy"}],"monitoring":["Observe during infusion for hypersensitivity","Lipid panel to assess response","Pregnancy testing"],"classWarnings":["ANGPTL3 inhibitor: novel mechanism — limited long-term safety data","Pregnancy contraindicated"],"hasBoxedWarning":false}