CSL Behring Announces Positive 3-Year Study Results for Etranacogene Dezaparvovec in Hemophilia B

King of Prussia, Pennsylvania – CSL Behring has released promising 3-year results from the pivotal HOPE-B study, confirming the sustained long-term durability and safety of etranacogene dezaparvovec-drlb (Hemgenix®) in individuals with hemophilia B.

The data, demonstrating prolonged elevated factor IX activity levels following a single infusion, were recently presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition.

“The long-term follow-up data from the HOPE-B study reinforces that a one-time treatment with Hemgenix can produce elevated and sustained factor IX levels and reduce the rate of annual bleeds for years in people living with hemophilia B,” said Steven Pipe, MD, professor of pediatrics and pathology at the University of Michigan and principal investigator of the HOPE-B pivotal trial. “Most importantly, the data show that nearly all the Phase III trial participants three years post-treatment with Hemgenix have remained free from the need for regular prophylactic infusions, which is groundbreaking for the hemophilia B community.”

Results from the phase 3, open-label, single-dose, single-arm HOPE-B trial revealed mean factor IX levels of 41.5 IU/dL at year 1, 36.7 IU/dL at year 2, and 38.6 IU/dL at year 3 post-treatment. Notably, 94% of patients remained free of continuous prophylactic therapy.

Furthermore, the 3-year data highlighted a 64% reduction in the mean annualized bleeding rate for all bleeds during months 7 to 36 of the study, sustaining the same bleed reduction observed in the study’s primary endpoint. Median bleeds per participant decreased from 2.0 during the lead-in period to 0.0 during years 1 to 3.

No serious adverse events linked to etranacogene dezaparvovec-drlb treatment were reported. Etranacogene dezaparvovec-drlb demonstrated excellent tolerability, with most treatment-emergent adverse events considered mild and occurring mostly within the first 6 months post-treatment. The most common adverse event was an increase in alanine transaminase, with 16.7% of participants receiving supportive care.

“Gene therapy is a novel treatment that addresses unmet needs in the hemophilia B community and the data presented at ASH continues to provide confidence in the clinical benefits of Hemgenix,” said Steven Pascoe, MD, chief medical officer of CSL. “As part of our commitment to the hemophilia B community, we will continue to follow the participants from the HOPE-B study as well as those who have been treated with Hemgenix post-FDA approval to generate additional evidence supporting the long-term safety, efficacy and durability of this one-time treatment. We encourage healthcare professionals to continue to have open conversations with their patients about individual treatment goals and whether Hemgenix may be an appropriate treatment option.”

Of the 54 male participants with severe or moderately severe hemophilia B administered etranacogene dezaparvovec-drlb during HOPE-B, 52 successfully completed the 3-year follow-up.

In addition to the HOPE-B trial results, CSL presented 2 posters on etranacogene dezaparvovec-drlb, evaluating its efficacy in patients with comorbidities.



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