1. HEMGENIX shows 94% patients free from prophylaxis after five years. 2. Mean factor IX activity levels sustained at 36.1% after five years. 3. Favorable safety profile maintained; treatment-related adverse events were minimal. 4. More than 75 patients globally received HEMGENIX in real-world applications. 5. HEMGENIX remains the only gene therapy available for hemophilia B patients.
FAQ
Why Bullish?
The sustained efficacy and safety profile of HEMGENIX supports its market position; historically, positive trial results have led to stock price increases in biotech companies.
How important is it?
The five-year results highlight HEMGENIX's effectiveness, likely boosting CSL's credibility and sales in the hemophilia market.
Why Long Term?
Long-term benefits from HEMGENIX may establish CSL's dominance in hemophilia B treatment, driving future growth.
Related Companies
HEMGENIX® Shows Long-term Efficacy and Safety in Five-Year Study
CSL Limited (ASX: CSL; USOTC: CSLLY) has released promising results from the five-year (60-month) follow-up of its pivotal Phase 3 HOPE-B study, confirming the long-term durability and safety of a one-time infusion of HEMGENIX® (etranacogene dezaparvovec-drlb) for adults with hemophilia B. This significant data was published in the New England Journal of Medicine (NEJM) and presented at the American Society of Hematology (ASH) Annual Meeting, reinforcing HEMGENIX's role as a transforming treatment for hemophilia B.
Key Findings from the HOPE-B Study
- Durable Factor IX Activity: Mean factor IX activity levels were sustained at greater than 36% over five years post-infusion, with specific results of 41.5 IU/dL (year one), 36.7 IU/dL (year two), 38.6 IU/dL (year three), 37.4 IU/dL (year four), and 36.1 IU/dL (year five).
- Sustained Bleed Protection: There was a 90% reduction in the mean adjusted annualized bleeding rate (ABR) from the lead-in period (4.16) compared to year five (0.40). Joint bleeds and spontaneous bleeds were reduced by 93% and 94%, respectively.
- Freedom from Prophylaxis: 94% of patients were free from continuous prophylaxis treatment five years following the infusion.
- Favorable Safety Profile: No serious adverse events were attributed to HEMGENIX treatment, with only 100 treatment-related adverse events recorded, mostly in the initial four months post-infusion.
Expert Insights on HEMGENIX
Dr. Steven Pipe, a respected leader in hemophilia research at the University of Michigan, stated, "The five-year HOPE-B results mark a pivotal milestone for gene therapy, providing clear, long-term data of the ability of HEMGENIX to potentially transform care for adults with hemophilia B." He emphasized that for patients dependent on frequent prophylactic treatments, the success of a single infusion offers an opportunity for a life less burdened by ongoing therapy requirements.
Deborah Long, MD, FCCP, Senior Vice President of Medical Affairs at CSL, expressed pride in the study's outcomes, stating, "These results highlight the meaningful difference HEMGENIX can make—helping people experience fewer bleeds compared to prophylaxis treatment and freeing them from the burden of regular ongoing treatment."
Long-Term Monitoring and Global Adoption
The five-year data represent the final analysis for the HOPE-B study, although participants will continue to be monitored under the IX-TEND 222-3003 extended follow-up study, tracking outcomes for up to 15 years post-treatment. Currently, more than 75 individuals in eight countries have received HEMGENIX, reflecting a growing global acceptance of this therapy, which has regulatory approval in numerous regions, including the United States, Canada, and Europe.
About Hemophilia B and HEMGENIX
Hemophilia B is a life-threatening genetic disorder caused by insufficient clotting factor IX, making patients susceptible to severe bleeding episodes. HEMGENIX is currently the only commercially available gene therapy option for adults suffering from this condition, demonstrating significant therapeutic benefits and a favorable safety profile.