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uniQure (QURE) Gene Therapy HEMGENIX Cuts Bleeds 90% in 5-Year HOPE-B Trial Data
The HOPE-B trial showed a 90% reduction in annualized bleeding rate and 94% of patients avoided continuous prophylaxis five years after one HEMGENIX infusion.
- CSL announced five-year HOPE-B results confirming durable safety and efficacy of a one-time HEMGENIX infusion, with data published in NEJM and presented at ASH.
- In the pivotal Phase 3 HOPE-B trial, fifty-four adult male participants completed a six-month lead-in, received a single intravenous HEMGENIX dose, and included patients with or without AAV5 neutralizing antibodies.
- Clinical results indicate the mean adjusted annualized bleeding rate fell by approximately 90%, joint bleeds decreased by 93%, and spontaneous bleeds dropped by 94% from lead-in baseline to year five.
- Adverse-Event monitoring found no serious treatment-related adverse events, ALT elevations required corticosteroids for nine participants , and two deaths during the study were assessed as non-treatment-related.
- More than 75 individuals across eight countries have received HEMGENIX in real-world settings, it holds approvals in multiple jurisdictions, and participants will be followed in the IX-TEND 222-3003 extended follow-up study for up to 15 years.
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Fox 4 Kansas City+32 Reposted by 32 other sources
Data Published in the New England Journal of Medicine Confirm the Long-term Durability and Safety of HEMGENIX® (etranacogene dezaparvovec-drlb) Over Five Years
·Kansas City, United States
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Leaning Left2Leaning Right4Center12Last UpdatedBias Distribution67% Center
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11%
C 67%
R 22%
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