Juxtapid Efficacy

Juxtapid was studied in a Phase 3 multinational, single-arm, open-label, 78-week trial involving 29 adults (16 males,13 females) with HoFH, aged 18 to 55 years.

Primary Endpoint 

40% Reduction in LDL-C at Week 26 of Treatment with Juxtapid from a Mean Value of 336 mg/dL to 190 mg/dL (n=29)

  • Efficacy was assessed in the intent-to-treat (ITT) population utilizing a last observation carried forward (LOCF) analysis.
  • The mean and median percent changes in LDL-C from baseline were -40% (P< 0.001) and -50%, respectively.

Juxtapid clinical trial data demonstrated 40% reduction in LDL-C

Additional Efficacy Data

50% Reduction in LDL-C in patients who completed 26 weeks of treatment; n=23

Juxtapid clinical trial data in patients with homozygous FH

 

LDL-C Reductions Were Sustained After 26 Weeks
For the study population overall, average reductions in LDL-C, TC, apo B, and non-HDL-C were sustained after 26 weeks during chronic therapy, when adjustments to concomitant lipid-lowering treatments were allowed.

Adverse Events Contributed to Premature Study Discontinuation for Five Patients
The adverse reactions that contributed to treatment discontinuations included diarrhea (2 patients; 7%) and abdominal pain, nausea, gastroenteritis, weight loss, headache, and difficulty controlling INR on warfarin (1 patient each; 3%).