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CSL Behring's Gene Therapy HEMGENIX® (etranacogene dezaparvovec-drlb) Four Years Post-Infusion Data Continue to Show Sustained Efficacy and Safety in Adults with Hemophilia B

1. HEMGENIX shows 94% of patients free from prophylaxis after four years. 2. Mean factor IX levels sustained at 37%, confirming HEMGENIX's efficacy.

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FAQ

Why Very Bullish?

The strong efficacy data may enhance CSL's market position, similar to past successful treatments boosting stock prices.

How important is it?

The results strengthen CSL's portfolio in hemophilia treatments, directly impacting investor sentiment.

Why Long Term?

Long-term data suggests sustained demand and potential for future growth, akin to previous biotech breakthroughs.

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CSLHEMGENIX
94 percent of patients eliminated factor IX prophylaxis and remained free of continuous prophylaxis through four years post-treatment Mean factor IX activity levels were sustained at near normal levels of 37% through four years post-treatment, reinforcing the efficacy of HEMGENIX in the treatment of hemophilia B Phase 3 HOPE-B data showed that a one-time treatment with HEMGENIX provided long-term bleed protection as mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by approximately 90% from lead-in as compared to year four KING OF PRUSSIA, Pa. , Feb. 7, 2025 /PRNewswire/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today 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 living with hemophilia B.

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