Mezagitamab in systemic lupus erythematosus: clinical and mechanistic findings of CD38 inhibition in an autoimmune disease.

Objective: To evaluate safety and mechanism of action of mezagitamab (TAK-079), an anti-CD38 monoclonal antibody, in patients with moderate to severe systemic lupus erythematosus (SLE).

Methods: A phase 1b double-blind, placebo-controlled, multicentre study was conducted in patients with SLE receiving standard background therapy. Eligible patients were adults who met the 2012 SLICC or ACR criteria for diagnosis, had a baseline SLE Disease Activity Index 2000 (SLEDAI-2K) score of ≥6 and were positive for anti-double-stranded DNA antibodies and/or anti-extractable nuclear antigens antibodies.

Use of Clinical Trial Simulations to Compare the Performance of Different Approaches for Population Analyses of Pediatric Pharmacokinetic Data.

The adequate characterization of the pharmacokinetics of a drug used in pediatrics is a mainstay of pediatric development programs and is critical for accurate dose selection in pediatrics. Analysis approaches can impact the estimation and characterization of pediatric pharmacokinetic parameters. Simulations were conducted to compare the performance of different approaches for analyzing pediatric pharmacokinetic data in the presence of extensive data from adult studies.

Upadacitinib pharmacokinetics and exposure-response analyses of efficacy and safety in psoriatic arthritis patients - Analyses of phase III clinical trials.

Upadacitinib is an oral Janus kinase inhibitor approved for the treatment of rheumatoid arthritis (RA) and recently approved by the European Medicines Agency for the treatment of psoriatic arthritis (PsA). The efficacy and safety profile of upadacitinib in PsA have been established in the SELECT-PsA program in two global phase III studies, which evaluated upadacitinib 15 and 30 mg q.d.

Breakthrough Therapy, PRIME and Sakigake: A Comparison Between Neuroscience and Oncology in Obtaining Preferred Regulatory Status.

Background: Because of the increasing demand for drugs addressing life-threatening and rare diseases, regulatory agencies have developed a variety of accelerated regulatory pathways. These programs are aimed at prioritizing the most promising drug candidates for diseases lacking satisfactory treatments. The most prominent accelerated programs introduced have been Breakthrough-Therapy Designation (BTD) in the United States, Priority Medicine (PRIME) in the European Union and Sakigake in Japan.

Enhanced Response to Drug-Induced QT Interval Lengthening in Patients with Heart Failure with Preserved Ejection Fraction.

Background: Patients with heart failure (HF) with reduced ejection fraction demonstrate enhanced response to drug-induced QT interval lengthening and are at increased risk for torsades de pointes. The influence of HF with preserved ejection fraction (HFpEF) on response to drug-induced QT lengthening is unknown.

Methods And Results: We administered intravenous ibutilide 0.

Breakthrough Therapy, PRIME and Sakigake: A Comparison Between Neuroscience and Oncology in Obtaining Preferred Regulatory Status.

Background: Because of the increasing demand for drugs addressing life-threatening and rare diseases, regulatory agencies have developed a variety of accelerated regulatory pathways. These programs are aimed at prioritizing the most promising drug candidates for diseases lacking satisfactory treatments. The most prominent accelerated programs introduced have been Breakthrough-Therapy Designation (BTD) in the United States, Priority Medicine (PRIME) in the European Union and Sakigake in Japan.