Concizumab in patients with hemophilia A or B without inhibitors: 56-week cutoff results of the phase 3 explorer8 study
Year: 2026
Type: article
Study design: Clinical Trial
Abstract: ABSTRACT: Concizumab is a novel nonfactor replacement therapy for once-daily subcutaneous prophylactic treatment of hemophilia A/B (HA/HB) with and without inhibitors. Concizumab was superior to on-demand treatment in patients with HA/HB without inhibitors in the prospective, multicenter, open-label phase 3 explorer8 study. Here, longer-term efficacy and safety results from the start of the study up to the 56-week cutoff are presented. Males aged ≥12 years with HA/HB were randomized 1:2 to no prophylaxis (group 1) or concizumab (group 2) or allocated to concizumab (groups 3 and 4). Assessments at the 56-week cutoff included efficacy, pharmacokinetics/pharmacodynamics, and safety. The 56-week cutoff was defined as when all patients in groups 2 to 4 had completed the visit at 56 weeks or permanently discontinued treatment. Of 148 patients in the full analysis set, 21 were randomized to no prophylaxis (group 1: HA, n = 9; HB, n = 12), 42 to concizumab (group 2: HA, n = 18; HB, n = 24), and 85 to the nonrandomized concizumab groups (groups 3 and 4: HA, n = 55; HB, n = 30). After ≥24 weeks of treatment, 17 patients in group 1 switched to concizumab. Low median annualized bleeding rates for treated spontaneous and traumatic bleeding episodes were maintained at the 56-week cutoff in patients receiving concizumab (HA, 1.7 [interquartile range (IQR), 0.0-4.5]; HB, 2.8 [IQR, 0.0-6.4]), consistent with 32-week cutoff results. Concizumab plasma concentration remained stable, with no new safety concerns. Concizumab showed longer-term efficacy in patients with HA/HB at the 56-week cutoff and was considered safe and well tolerated. This trial was registered at www.clinicaltrials.gov as #NCT04082429.
Source: Blood Advances
Institutions: University of Southern California, Children's Hospital Los Angeles, Mahidol University, Ramathibodi Hospital, Sahyadri Hospital, Novo Nordisk (Denmark), McMaster University, Royal Free London NHS Foundation Trust, Universitätsklinikum des Saarlandes, Saarland University, Yonsei University, Severance Hospital, Yonsei University Health System, Yonsei Cancer Hospital, Nagoya University Hospital, Vilnius University, Vilnius University Hospital Santariskiu Klinikos, The Alfred Hospital, Monash University, University Hospital Zurich, Instytut Hematologii i Transfuzjologi, Lund University, Skåne University Hospital
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $3,800
Funder: Novo Nordisk
OpenAlex ID: W7165734041