Roche Announces Interim Analysis Results of Phase 3 HAVEN 6 Study of Eimexizumab

On December 13, Roche announced the results of the interim analysis of the Phase 3 HAVEN 6 study.
The data showed that the new hemophilia A drug Hemliba (emizumab) has no moderate or mild factor VIII inhibitors in the body.
Patients with high-degree hemophilia show good safety and effective bleeding control
.
At present, mature treatment guidelines have been established for severe hemophilia A, but there is less information and treatment guidelines on moderate and mild hemophilia A, which may lead to delayed diagnosis or missed diagnosis of bleeding events
.
Considering that this population may not use preventive treatments, they may experience a more severe clinical burden, with less than 30% of patients with moderate or mild hemophilia A living a life without bleeding events
.
HAVEN 6 is a phase 3 study conducted in patients with moderate or mild hemophilia A without factor VIII inhibitors in the body to evaluate the safety, effectiveness, pharmacokinetics and pharmacodynamics of Hemlibra
.
The interim analysis released this time includes data on 71 patients (69 males and 2 females)
.
Among them, 20 patients were patients with mild hemophilia A without factor VIII inhibitors, and 51 patients were patients with moderate hemophilia A without factor VIII inhibitors
.
Thirty-seven patients received factor VIII prophylactic treatment at baseline
.
The interim analysis was performed after 50 patients with moderate hemophilia A completed the study for at least 24 weeks of treatment or withdrew from the study
.
The data deadline is April 16, 2021
.
These data show that in the HAVEN 6 study, Hemlibra showed good safety and effective bleeding control.
80.
3% of patients had no bleeding events that required treatment, and 90.
1% of patients had no joint bleeding that required treatment
.
The annualized bleeding rate (ABR) is still low, consistent with the previously reported HAVEN 1-4 results
.
In addition, of the 50 patients aged 12 years or older who responded to the EmiPref questionnaire, 48 (96.
0%) preferred Hemlibra over the previous treatment
.
In the HAVEN 6 study, the most common adverse events (AE) that occurred in 10% or more of the patients were headache (14.
1%) and local injection site reaction (ISR) (12.
7%)
.
Eleven patients (15.
5%) reported Hemlibra-related AEs, of which ISR was the most common (12.
7%)
.
As of the data cut-off date, there were no deaths, thrombotic microangiopathies (TMA) or serious thrombotic events (TE) cases in the study, which reinforces the good safety of Hemlibra
.
A separate analysis of TE and TMA events of the Hemlibra treated population, including real-world data, was also displayed as a poster at the ASH annual meeting
.
These results indicate that as exposure increases, the assessment of reported unaccompanied aPCC (activated prothrombin complex concentrate) events is similar to the previous analysis, and the benefit/risk profile of Hemlibra remains unchanged
.
These data further confirm the good safety of Hemlibra, which is consistent with the results of the previous HAVEN and STASEY studies
.
Hemlibra is a humanized bispecific monoclonal antibody, which has a bispecific structure like two arms, which can combine the proteins required to activate the natural coagulation cascade-activated coagulation factor IX and coagulation factor X together, to restore the coagulation process in patients with hemophilia a, hemophilia a treatment goal to make zero bleeding patient becomes possible
.
In clinical studies, Hemlibra has been shown to significantly reduce bleeding events and improve body function
.
For patients with factor VIII inhibitors (anti-FVIII antibodies) in the body, Hemlibra can still perform well
.
In addition, because Hemlibra has no structural or sequence homology with factor VIII, it will not induce or enhance the production of direct inhibitors of factor VIII
.
Up to now, Hemlibra has been approved for the treatment of hemophilia A patients with factor VIII inhibitors in more than 100 countries around the world, and it has been approved for the treatment of blood type A without factor VIII inhibitors in more than 90 countries around the world For patients with friendly diseases, prevent or reduce the frequency of bleeding events
.
Hemlibra has been researched in one of the largest clinical trial projects, which includes 8 phase 3 studies.
The enrolled patients include patients with hemophilia A with or without factor VIII inhibitors in their bodies
.
In China, Hemlibra was approved in December 2018: for adults and children with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors in the body Conventional preventive treatment to prevent bleeding or reduce the frequency of bleeding
.
In May 2021, Hemlibra was approved in China for a new indication: it is used as a routine for adults and children with severe hemophilia A (congenital factor VIII deficiency, FVIII<1%) without factor VIII inhibitors in the body Preventive treatment
.
Reference source: Interim data from phase III HAVEN 6 study demonstrate favourable safety and efficacy profile of Roche's Hemlibra in people with moderate or mild haemophilia A