Roche (and Roche group member Chugai) recently presented data from a phase I/II clinical study of their investigational bispecific antibody therapy NXT007, in people with hemophilia A who do not have factor VIII inhibitors. NXT007 is being positioned as a next-generation subcutaneous prophylactic treatment that builds on the technology used to develop emicizumab (Hemlibra®).
Emicizumab, manufactured by Genentech (also a member of the Roche Group) is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children of all ages, newborn and older, with hemophilia A with and without FVIII inhibitors. Like emicizumab, NXT007 is a humanized antibody that brings together two factor proteins (factor IXa and factor X), to mimic activated FVIII in its role of allowing the continuation of the coagulation cascade. The ongoing NXTAGE trial program is evaluating the safety, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of NXT007.
The latest NXT007 data was presented in an oral presentation shared at the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington D.C., which concluded on June 25th. According to press releases announcing the companies’ highlights presented at ISTH, part B of the trial enrolled 30 “Hemlibra-naïve” individuals aged 12 to 65 years with severe hemophilia A and no factor VIII inhibitors. Participants were divided into four cohorts and received maintenance doses every four weeks following an initial (higher dose) loading period of four to six weeks. The primary analysis occurred after at least six participants in each cohort completed 16 weeks of treatment.
Patient participants in cohorts B-3 and B-4, who received the highest doses of NXT007, experienced zero treated bleeds during the maintenance dosing period, indicating complete prevention of bleeding events. Cohort B-1, which had the highest baseline annualized bleed rate (ABR) of 12.83, saw a reduction to an ABR of 1.20. Cohort B-2, starting with a lower baseline ABR of 2.17, also showed a decrease, with an ABR of 0.28 during treatment. In addition, no serious adverse events or thromboembolic events were reported across all dose levels.
“These NXT007 data are promising for people with hemophilia A and underscore our ongoing commitment to advancing care and addressing the real-world challenges faced by this community,” said Levi Garraway, MD, PhD, Roche’s Chief Medical Officer and Head of Global Product Development. “Hemlibra established a new standard of care, and our focus is to continue to deliver breakthrough innovation that might ultimately help people with hemophilia to live their lives in a manner unaffected by this condition.”
Phase III clinical studies of NXT007 are slated to begin in 2026.
Source: PharmaExec June 24th, Roche press release dated June 22nd
