uniQure/ CSL Behring's Hemophilia Gene Therapy Shows Sustained Efficacy and Safety At Four Years

CSL Limited (OTC:CSLLY) and uniQure Inc (NASDAQ: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.

Also Read: Why Is uniQure Stock Skyrocketing On Tuesday?

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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