New Data from Phase IIIb STASEY Study Reinforces Safety and Efficacy of Roche’s Hemlibra in People with Hemophilia A and Factor VIII Inhibitors

July 13, 2020 /Bio ValleyBIOON/ -- Roche recently announced the results of the second interim analysis of the Phase III STASEY study at the 2020 International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress, further reinforcing the safety profile of Hemlibra (emicizumab) established in the Phase III HAVEN clinical program. In the STASEY study, Hemlibra demonstrated efficacy in adult and adolescent patients with hemophilia A who have factor VIII inhibitors, with no new safety signals identified, consistent with previous safety observations. Further new analytical data indicate that patients treated with Hemlibra can undergo certain minor surgical procedures without the need for additional prophylactic clotting factors.

Levi Garraway, M.D., Chief Medical Officer and Global Head of Product Development at Roche, stated, “These important safety data continue to add to the substantial body of clinical evidence, reinforcing the potential of Hemlibra to redefine the standard of care for patients with hemophilia A. The STASEY study reflects our ongoing commitment to providing valuable insights to meet the needs of the hemophilia community and to enhance our understanding of Hemlibra in clinical practice.”

STASEY is a single-arm, multicenter, open-label Phase IIIb clinical study in which patients received Hemlibra for a mean duration of 50.9 weeks. The annualized bleeding rates (ABRs) for all bleeding events (including treated bleeds, treated spontaneous bleeds, treated joint bleeds, and treated target joint bleeds) were low, with 167 patients (85.6%) experiencing zero treated bleeds. In the STASEY study, two thrombotic events (TEs) unrelated to Hemlibra were reported: one was ST-segment elevationMyocardial Infarction, the treating physician assessed it as unrelated to Hemlibra; the second case was a hypertrophic clot at the tooth extraction site, which is a known complication of the procedure.

The second interim analysis of the STASEY study included data from 193 patients with hemophilia A who had factor VIII inhibitors and received once-weekly prophylactic treatment with Hemlibra. No thrombotic microangiopathy or serious thrombotic events (TEs) related to Hemlibra were reported, and no new safety signals were observed. Adverse events (AEs) related to Hemlibra were reported in 33 patients (17.1%). In the STASEY study, the most common adverse events occurring in ≥10% of patientsAdverse ReactionsThe common cold symptoms were nasopharyngitis (12.4%), headache (11.9%), and injection site reactions (ISRs, 11.4%). The reported ISRs were mild or moderate in severity, and no patients discontinued treatment due to ISRs. The annualized bleeding rate (ABR) was also consistent with the observations from the previously reported Phase III HAVEN studies.

A separate analysis described the procedures and outcomes of minor surgeries and unplanned major surgeries in patients receiving Hemlibra treatment, although this was not a formal surgical endpoint in the STASEY study. The results indicated that hemophilia A patients with factor VIII inhibitors who underwent certain minor surgeries while on Hemlibra might not require additional prophylactic clotting factors. Most minor surgeries (n=20/31) were performed without prophylactic clotting factors (64.5%), and 85% of these cases (n=17/20) did not require postoperative bleeding management. In unplanned major surgeries (n=9), eight cases were managed with prophylactic clotting factors, four of which successfully resolved bleeding using recombinant factor VIIa. These findings are consistent with the surgical analysis previously observed in the pivotal HAVEN studies.。

Hemlibra is a bispecific monoclonal antibody that brings together two proteins essential for activating the natural coagulation cascade and restoring the natural coagulation process—coagulation factors IXa and X—thereby restoring hemostasis in patients with hemophilia A. Clinical studies have demonstrated that Hemlibra significantly reduces bleeding episodes and improves physical function. Hemlibra remains highly effective in patients with factor VIII inhibitors (anti-FVIII antibodies).

Hemlibra was developed by Chugai Pharmaceutical and is currently being co-developed by Chugai Pharmaceutical, Roche, and its subsidiary Genentech. The development of this drug aims to help overcome the current clinical challenges faced by patients with hemophilia A: short duration of action of existing drugs, the development of factor VIII inhibitors, and the need for frequent intravenous infusions.

Hemlibra was first approved for marketing in October 2018. To date, it has been approved in more than 90 countries for the treatment of patients with hemophilia A who have factor VIII inhibitors (based on the HAVEN 1 and HAVEN 2 studies), and in more than 70 countries worldwide for the treatment of patients with hemophilia A who do not have factor VIII inhibitors (based on the HAVEN 3 and HAVEN 4 studies), to prevent or reduce the frequency of bleeding episodes. The development of this drug aims to help overcome the current clinical challenges faced by the hemophilia A population: short duration of action of existing drugs, the development of factor VIII inhibitors, and the need for frequent intravenous infusions.

It is worth noting that Hemlibra is the first novel class of therapy approved in nearly 20 years for patients with severe hemophilia A without factor VIII inhibitors. It is also the only prophylactic treatment suitable for self-administered subcutaneous injection in patients with hemophilia A, regardless of the presence or absence of factor VIII inhibitors, offering multiple dosing regimens (once weekly, once every two weeks, or once every four weeks via subcutaneous injection).

According to the performance report released by Roche, Hemlibra achieved sales of CHF 1.38 billion in 2019. With the expansion of the indicated population and market growth, commercial sales of Hemlibra are expected to increase significantly. In 2018, Clarivate Analytics predicted that Hemlibra’s sales would reach $4 billion in 2022. (Bioon.com)

Original Source:New data from phase IIIb study reinforces safety profile of Roche’s Hemlibra in people with haemophilia A