Professor and head, coagulation disorders and comprehensive care center, University Hospital of Frankfurt, Germany

Wolfgang Miesbach, MD, PhD

“In the future, I think hemophilia centers should be able to offer gene therapy after it has been approved. And one possibility, for example, would be to initiate a hub and spoke model in which there would be a close cooperation between experienced and non-experienced centers to enable patients to be treated with gene therapy.”

The phase 3 HOPE-B study (NCT03569891) met its primary endpoint, with etranacogene dezaparvovec (EtranaDez, AMT-061; CSL Behring, UniQure) significantly reducing annualized bleeding rate (ABR) and demonstrating superiority to Factor IX (FIX) prophylactic therapy. The therapy demonstrated efficacy in patients both with and without neutralizing adeno-associated virus vector antibodies.

The final analysis of HOPE-B were presented by investigator Wolfgang Miesbach, MD, PhD, at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting, February 2-4, 2022. Miesbach serves as professor and head of coagulation disorders at the Comprehensive Care Centre at University Hospital of Frankfurt, Germany.

Miesbach and colleagues found that adjusted ABR was reduced by 64% (

= .0002) and FIX-treated bleeds reduced by 77% (

<.0001) after a 6-month lead-in period over months 7 to 18. Almost all participants (98%) treated with the full dose of EtranaDez were able to discontinue prophylaxis.

spoke with Miesbach to learn more about research that remains to be conducted with EtranaDez. He discussed potential ways the therapy could be integrated into treatment for patients with hemophilia B.

Final analysis of pivotal HOPE-B study demonstrates durable and sustained therapeutic effect of etranacogenedezaparvovec gene therapy in hemophilia B – Data presented at EAHAD 2022. News release. CSL Behring. February 4, 2022. https://www.cslbehring.com/newsroom/2022/hope-b-data-presented-at-eahad2022

Videos

The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed future integration of EtranaDez into hemophilia B treatment. 

Wolfgang Miesbach, MD, PhD, on Future Research with EtranaDez 

“We can conclude, and this was a really surprising finding of the study, that in addition to patients with prior hepatitis B, prior hepatitis C and HIV infection, patients with preexisting antibodies to AAV, up to a certain titer, can be include in the study.”

Etranacogene dezaparvovec (EtranaDez, AMT-061; CSL Behring, UniQure) reduced annualized bleeding rate (ABR) in patients with hemophilia B, both with and without neutralizing adeno-associated virus vector antibodies, compared to superior to Factor IX (FIX) prophylactic therapy.

These findings, from the final analysis of the phase 3 HOPE-B study (NCT03569891), were presented by investigator Wolfgang Miesbach, MD, PhD, at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting, February 2-4, 2022. Miesbach serves as professor and head of coagulation disorders at the Comprehensive Care Centre at University Hospital of Frankfurt, Germany.

Miesbach and colleagues found that, after a 6-month lead-in period, adjusted ABR was reduced by 64% (

<.0001) over months 7 to 18. Almost all participants (98%) treated with the full dose of EtranaDez were able to discontinue prophylaxis.

spoke with Miesbach to learn more about the demographics and findings from the HOPE-B study. He discussed the surprising finding that treatment was effective in participants with prior hepatitis B or C, HIV infection, or AAV antibodies.

The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed demographics of the HOPE-B study. 

Addressing AAV Antibodies in Hemophilia B Gene Therapy: Wolfgang Miesbach, MD, PhD 

“Significant improvements were seen in 4key domains, participants’ feelings, their work and school life, their treatments and in their future domains with reductions between 6and nearly 15 points.”

Etranacogene dezaparvovec (EtranaDez, AMT-061; CSL Behring, UniQure) reduced annualized bleeding rate (ABR) in patients with hemophilia B compared to superior to Factor IX (FIX) prophylactic therapy in findings from the final analysis of the phase 3 HOPE-B study (NCT03569891).

Investigator Wolfgang Miesbach, MD, PhD, presented the final analysis at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting, February 2-4, 2022. Miesbach is a professor and head of coagulation disorders at the Comprehensive Care Centre at University Hospital of Frankfurt, Germany. Miesbach and colleagues found that adjusted ABR was reduced by 64% (P = .0002) and FIX-treated bleeds reduced by 77% (

<.0001) over months 7 to 18 after the 6-month lead-in period. Most participants (98%) treated with the full dose of EtranaDez were able to discontinue prophylaxis.

spoke with Miesbach to learn more about the efficacy findings seen in the HOPE-B study. He stressed that significant improvements were seen in bleeding events as well as in quality-of-life scores.

Articles

The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed new efficacy findings from the HOPE-B study. 

EtranaDez’s Efficacy in Hemophilia B: Wolfgang Miesbach, MD, PhD

“The HOPE-B trial was the first phase 3 study in hemophilia B and has the largest cohort of gene therapy for hemophilia B to report to date. It is also the only trial to include participants with neutralizing antibodies to AAV and this is really unique.”

Etranacogene dezaparvovec (EtranaDez, AMT-061; CSL Behring, UniQure) is superior to Factor IX (FIX) prophylactic therapy in reducing annualized bleeding rate (ABR) in patients with hemophilia B, according to results from the final analysis of the phase 3 HOPE-B study (NCT03569891).

Data from the analysis were presented at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting, February 2-4, 2022, by investigator Wolfgang Miesbach, MD, PhD, professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany. Miesbach and colleagues found that, after the 6-month lead-in period, adjusted ABR was reduced by 64% (