1. Efficacy of alirocumab in 1191 patients with a wide spectrum of mutations in genes causative for familial hypercholesterolemia 
Joep C. Defesche, PhD, Claudia Stefanutti, MD, PhD, Gisle Langslet, MD, Paul N. Hopkins, MD, MSPH, Werner Seiz, MD, PhD, Marie T. Baccara-Dinet, MD, MSc, Sara C. Hamon, MA, PhD, Poulabi Banerjee, PhD, John J.P. Kastelein, MD, PhD, FESC 
Journal of Clinical Lipidology 
Volume 11, Issue 6, Pages e7 (November 2017)
DOI: /j.jacl
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2. Figure 1
Mean percentage reduction from baseline in LDL-C level according to mutation status at (A) Week 12 and (B) Week 24. LDL-C data analyzed for Phase 3 trials only. No patients from the Phase 2 trial were included because of the different doses/dosing schedules. Data were not available for all patients in the Phase 3 trials at all time points. Control was placebo in all trials except for the ALTERNATIVE trial (which had an ezetimibe control arm and an atorvastatin calibrator arm). *Alirocumab 75 mg Q2W with possible increase to 150 mg Q2W at Week 12. In panel A (Week 12), all patients in this group were receiving 75 mg. APOB, apolipoprotein B; GOF, gain-of-function; LDL-C, low-density lipoprotein cholesterol; LDLR, low-density lipoprotein receptor; PCSK9, proprotein convertase subtilisin/kexin type 9; SE, standard error; Q2W, every 2 weeks.
Journal of Clinical Lipidology  , e7DOI: ( /j.jacl )
Copyright © 2017 National Lipid Association Terms and Conditions

3. Figure 2
Percentage change in LDL-C at Week 24 for all patients receiving (A) alirocumab 75 mg Q2W (with possible dose increase to 150 mg) and (B) 150 mg Q2W according to mutation status. LDL-C data analyzed for Phase 3 studies only. No patients from the Phase 2 study were included because of the different doses/dosing schedules. APOB, apolipoprotein B; GOF, gain-of-function; LDL-C, low-density lipoprotein cholesterol; LDLR, low-density lipoprotein receptor; LDLRAP1, LDL receptor adaptor protein 1; PCSK9, proprotein convertase subtilisin/kexin type 9.
Journal of Clinical Lipidology  , e7DOI: ( /j.jacl )
Copyright © 2017 National Lipid Association Terms and Conditions

4. Supplementary Figure S1
Summary of studies included in the analysis. The figure indicates the number (n) of patients with diagnosis of FH from each trial. ∗Alirocumab 75 mg Q2W was increased to 150 mg Q2W at Week 12 depending on LDL-C at Week 8. †Some patients from ALTERNATIVE were included in the sequencing analysis although they did not have a recorded diagnosis of FH, as they had very high baseline LDL-C levels (>5.0 mmol/L, ∼193 mg/dL). See Table 1 in the main article. ‡Maximally tolerated statin was defined as atorvastatin 40 to 80 mg, rosuvastatin 20 to 40 mg, or simvastatin 80 mg; lower doses were allowed with an investigator-approved reason such as intolerance or local practices. FH, familial hypercholesterolemia; LDL-C, low-density lipoprotein cholesterol; LLT, lipid-lowering therapy; Q2W, every 2 weeks; Q4W, every 4 weeks.
Journal of Clinical Lipidology  , e7DOI: ( /j.jacl )
Copyright © 2017 National Lipid Association Terms and Conditions

5. Supplementary Figure S2
Percentage change in LDL-C at Week 12 for all subjects receiving (A) alirocumab 75 mg Q2W (with possible dose increase to 150 mg at Week 12) and (B) 150 mg Q2W according to mutation status. LDL-C data analyzed for Phase 3 studies only. No patients from the Phase 2 study were included because of the different doses/dosing schedules. In panel A (ie, alirocumab 75/150 mg regimen at Week 12), all patients were receiving 75 mg Q2W. APOB, apolipoprotein B; GOF, gain-of-function; LDL-C, low-density lipoprotein cholesterol; LDLR, low-density lipoprotein receptor; LDLRAP1, LDLR adaptor protein 1; PCSK9, proprotein convertase subtilisin/kexin type 9; Q2W, every 2 weeks.
Journal of Clinical Lipidology  , e7DOI: ( /j.jacl )
Copyright © 2017 National Lipid Association Terms and Conditions