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Early results from the Phase I/II NXTAGE trial show that NXT007 may achieve hemostatic normalization in patients with hemophilia A without factor VIII inhibitors.
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NXT007 shows promise in hemophilia A treatment: Early Phase I/II data reveal zero treated bleeds in highest-dose cohorts, indicating potential for hemostatic normalization.
Favorable safety profile supports advancement: No serious adverse events or thromboembolic events reported across all dose levels.
Phase III trials on the horizon: Roche and Chugai plan to begin three Phase III studies in 2026, including a head-to-head trial against Hemlibra.
Early data from the Phase I/II NXTAGE trial indicate that Roche’s NXT007, an investigational next-generation bispecific antibody, may achieve hemostatic normalization in people with hemophilia A who do not have factor VIII inhibitors. The findings, presented by Chugai Pharmaceutical at the 2025 International Society on Thrombosis and Hemostasis (ISTH) Congress, support advancing NXT007 into Phase III development, according to the trial investigators. Designed to build on the success of Hemlibra, NXT007 aims to deliver sustained bleed protection, reduced treatment burden, and greater flexibility through subcutaneous dosing.1
“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, chief medical officer, head, global product development, Roche, in a press release. “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.”
The ongoing, multicenter NXTAGE trial is evaluating the safety, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of NXT007. 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 loading period of four to six weeks. The primary analysis occurred after at least six participants in each cohort completed 16 weeks of treatment.2,3
Patients 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 suppression 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.
NXT007 was well tolerated at all dose levels. There were no dose-dependent trends in adverse events (AEs), and no serious AEs were attributed to the therapy. Additionally, no thromboembolic events were reported, and no participants discontinued treatment due to drug-related AEs.2
According to the Centers for Disease Control and Prevention, approximately 33,000 males are currently living with hemophilia in the United States. Nearly 70% of cases are diagnosed in infants younger than one month, and about one in five individuals with hemophilia A will develop an inhibitor to treatment products.4
Additional Phase II data are expected later this year, and three Phase III trials are scheduled to begin in 2026, including a head-to-head study comparing NXT007 to Hemlibra.1
“We are very excited that this first data in people with hemophilia A, suggests that NXT007 has the potential to provide hemostatic normalization,” said Osamu Okuda, president, CEO, Chugai, in a press release. “Based on these data, we plan to initiate three Phase III studies of NXT007 next year, including a head-to-head study with Hemlibra. Treatment in hemophilia A has drastically evolved in recent years, and there are now expectations for treatments with even higher efficacy and even more convenient dosing form. Together with Roche, we will focus on the clinical development of NXT007, aiming to deliver it as a new treatment option as soon as possible to people who need it.”2
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