Dose/Exposure-Response Modeling to Support Dosing Recommendation for Phase III Development of Baricitinib in Patients with Rheumatoid Arthritis

CPT Pharmacometrics Syst Pharmacol. 2017 Dec;6(12):804-813. doi: 10.1002/psp4.12251. Epub 2017 Oct 17.

Abstract

Baricitinib is an oral inhibitor of Janus kinases (JAKs), selective for JAK1 and 2. It demonstrated dose-dependent efficacy in patients with moderate-to-severe rheumatoid arthritis (RA) in a phase IIb study up to 24 weeks. Population pharmacokinetic/pharmacodynamic (PopPK/PD) models were developed to characterize concentration-time profiles and dose/exposure-response (D/E-R) relationships for the key efficacy (proportion of patients achieving American College of Rheumatology 20%, 50%, or 70% response rate) and safety endpoints (incidence of anemia) for the phase IIb study. The modeling suggested that 4 mg q.d. was likely to offer the optimum risk/benefit balance, whereas 2 mg q.d. had the potential for adequate efficacy. In addition, at the same total daily dose, a twice-daily regimen is not expected to provide an advantage over q.d. dosing for the efficacy or safety endpoints. The model-based simulations formed the rationale for key aspects of dosing, such as dose levels and dosing frequency for phase III development.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Antirheumatic Agents / administration & dosage*
  • Antirheumatic Agents / adverse effects
  • Antirheumatic Agents / pharmacokinetics*
  • Arthritis, Rheumatoid / blood
  • Arthritis, Rheumatoid / drug therapy*
  • Arthritis, Rheumatoid / enzymology
  • Azetidines / administration & dosage*
  • Azetidines / adverse effects
  • Azetidines / pharmacokinetics*
  • Clinical Trials, Phase III as Topic
  • Computer Simulation
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Drug Administration Schedule
  • Humans
  • Janus Kinase 1 / antagonists & inhibitors
  • Janus Kinase 2 / antagonists & inhibitors
  • Models, Biological*
  • Predictive Value of Tests
  • Purines
  • Pyrazoles
  • Sulfonamides / administration & dosage*
  • Sulfonamides / adverse effects
  • Sulfonamides / pharmacokinetics*
  • Treatment Outcome

Substances

  • Antirheumatic Agents
  • Azetidines
  • Purines
  • Pyrazoles
  • Sulfonamides
  • JAK1 protein, human
  • JAK2 protein, human
  • Janus Kinase 1
  • Janus Kinase 2
  • baricitinib