Zinger Key Points
- Hemgenix maintained factor IX levels at 37.4 IU/dL in year four, with a 90% reduction in annualized bleeding rates compared to baseline.
- At year four, 94% of patients remained off continuous prophylaxis, and no patients resumed it between year three and year four.
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CSL Limited CSLLY and uniQure Inc QURE announced the four-year results from the pivotal HOPE-B study confirming the long-term durability and safety of a one-time infusion of Hemgenix (etranacogene dezaparvovec-drlb) for adults hemophilia B patients.
The data showed that through four years, Hemgenix continues to deliver elevated and sustained factor IX activity levels, can offer long-term and greater bleed protection compared to prophylactic treatment, can eliminate the need for routine factor IX prophylaxis, and maintains a favorable safety profile.
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Approved in 2022 by the FDA, Hemgenix is the first gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes.
It is also the only approved gene therapy for hemophilia B to treat adult patients with and without AAV5-neutralizing antibodies.
In the Phase 3, open-label, single-dose, single-arm HOPE-B trial, 54 adult male participants with severe or moderately severe hemophilia B, with or without preexisting AAV5 neutralizing antibodies, were infused with a single dose of Hemgenix.
Of the 54 participants who received Hemgenix, 51 completed four years of follow-up. Hemgenix produced mean factor IX levels of 41.5 IU/dL (n=50) at year one, 36.7 IU/dL (n=50) at year two, 38.6 IU/dL (n=48) at year three and 37.4 IU/dL (n=47) at year four post-infusion.
In addition, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by approximately 90% from lead-in as compared to year four.
Furthermore, joint bleeds were reduced from a mean ABR of 2.34 at lead-in to 0.09 during year four. In year four, 94% of patients remained free of continuous prophylaxis treatment.
No patients returned to continuous prophylaxis between year three and year four.
There were no serious adverse events related to treatment.
Price Action: QURE stock is down 5.95% at $15.25 at last check Friday.
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