Genentech’s Hemlibra Maintains Strong Bleeding Control Results in Phase 3 Trial, Interim Analysis Shows

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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FVIII inhibitors

Injection of Hemlibra (emicizumab-KXWH) every four weeks helps control bleeding in hemophilia A patients, an interim analysis of the Phase 3 HAVEN 4 study shows.

The results, announced by Genentech, are in agreement with several previous clinical trials – HAVEN 1 (NCT02622321), HAVEN 2 (NCT02795767) and HAVEN 3 (NCT02847637) – showing Hemlibra’s benefits regardless of patients’ inhibitor status.

The ongoing HAVEN 4 study (NCT03020160) is a multicenter, open-label study aiming to evaluate three parameters – safety, efficacy and pharmacokinetics (drug response in the body) – of Hemlibra in hemophilia A patients.

Recruited participants – adults and 12-year or older adolescents – receive Hemlibra as prophylaxis (preventive treatment) administered by injection under the skin.

In the study first part, patients are treated with a single dose of Hemlibra at 6 mg/kg followed by the same dose administered every four weeks for at least 24 weeks. At this stage, researchers will determine the therapy’s pharmacokinetics (PK) profile – how the therapy moves inside the body, from the time of absorption, distribution, and metabolism to excretion. The first stage of the study included seven patients.

Following the first stage – called PK run-in – the remaining patients (41 of them) are included in the second phase of the study. At this stage, they’re treated with Hemlibra prophylaxis delivered as 3 mg/kg/week for four weeks. After, they receive 6 mg/kg every four weeks for a minimum of 24 weeks.

The trial’s primary outcome is to determine the number of bleeding events over time in the expansion phase patients – from 24 weeks up to approximately 20 months.

This year, the U.S. Food and Drug Administration (FDA) approved Hemlibra as a prophylactic medicine to prevent or lessen bleeding episodes in children and adults with hemophilia A with factor VIII inhibitors.

The therapy – a specific antibody for factor IX and factor X – mimics the action of factor VIII, triggering a normal clotting process.

“Current treatment regimens for hemophilia A can require frequent intravenous infusions. We are encouraged that HEMLIBRA prophylaxis administered by injection under the skin once every four weeks showed clinically meaningful bleed control in people with hemophilia A,” Sandra Horning, M.D., chief medical officer and head of Global Product Development at Genetech, said in a press release.

“Together with the findings from other Phase III studies, these interim results support the potential for HEMLIBRA to be dosed at different schedules, allowing patients to choose the option that’s right for them,” she added.

The HAVEN 4 study results are due for presentation at future meetings. In the near future,  Genetech hopes to submit the data for drug approval to health authorities around the world.