- Evinacumab added to maximum statins for patients with homozygous familial hypercholesterolemia (HoFH) yielded a 49 percentage point difference in low-density lipoprotein cholesterol (LDL-C) vs a slight increase in the placebo group.
- 24-week results from the randomized ELIPSE HoFH trial.
Why this matters
- Evinacumab treatment in a small open-label phase 2 study yielded a similar 49% reduction from baseline LDL-C levels.
- 43 in evinacumab group vs 22 receiving placebo.
- 94% were receiving statins (77% receiving high-intensity statins).
- Other therapies included PCSK9 inhibitor, ezetimibe, lomitapide.
- 63% were receiving at least 3 lipid-modifying drugs.
- At week 24, LDL-C:
- Had declined in the evinacumab group by 47.1% from baseline.
- Had increased in the placebo group by 1.9%.
- Between-group least squares mean difference was −49.0 (95% CI, −65.0 to −33.1) percentage points.
- This difference in terms of LDL-C level was −132 (95% CI, −175 to −89) mg/dL.
- Results were similar regardless of background therapy.
- The evinacumab group also had desirable declines in other lipids.
- Adverse events: 66% with evinacumab, 81% with placebo.
- Liver enzyme increase: 5% with evinacumab, 10% with placebo.
- Only a few patients had preexisting antibodies to evinacumab, which did not seem to affect pharmacokinetics.
- No cardiovascular events.
- Double-blind, placebo-controlled phase 3 trial.
- Evinacumab dose: 15 mg/kg body weight infusion every 4 weeks.
- Funding: Regeneron Pharmaceuticals.
- Relatively short, small number of patients.