Steven W. Pipe, MD, on the Implications of the Final Data From Hemgenix’s HOPE-B Trial

This is the second part of an interview with Steven W. Pipe, MD. For the first part, click here.

“I think for patients who are interested in a genetic therapy like this, who want to be liberated from prophylaxis, this is really the only approach that really delivers that. It truly is a one-time infusion that can lead to a transformative impact for patients...”

The pivotal phase 3 HOPE-B clinical trial (NCT03569891), which provided data to help support the FDA’s 2022 approval of CSL Behring/uniQure’s etranacogene dezaparvovec (marketed as Hemgenix), a gene therapy product for the treatment of severe or moderately severe hemophilia B with or without preexisting AAV5 neutralizing antibodies (NAbs), continued to gather data on the treated participants for several years following the approval.¹ Notably, the end-of-study results from the trial were finally presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida by principal investigatory Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health.²

Following a discussion on the conference floor with Pipe about the key results he presented at the meeting, CGTLive® asked him about the big picture implications of the latest data. Pipe emphasized that for patients seeking freedom from ongoing prophylactic treatment, this approach stands apart: It is a true one-time infusion that can deliver sustained factor IX expression and effective bleed control, offering a transformative outcome. With 5 years of HOPE-B follow-up now available, along with data from other trials, he said clinicians can have growing confidence in the durability and long-term safety of the therapy.

He also highlighted a milestone for real-world treatment, noting that 50 patients have now been treated with Hemgenix in the commercial setting, and pointed out that real-world observations will provide additional data over time. Pipe also stated that unanswered questions remain, particularly around the potential of preventing liver enzyme elevations that may occur in some patients.

For more coverage of ASH 2025, click here.

REFERENCES
1. FDA approves first gene therapy to treat adults with hemophilia B. News release. FDA. November 22, 2022. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treat-adults-hemophilia-b
2. Pipe S, Miesbach W, Recht M, et al. End-of-study analysis of the HOPE-B trial confirms the durable efficacy and safety of etranacogene dezaparvovec hemophilia b gene therapy over 5 years. Presented at: ASH 2025 Annual Meeting. December 6-9, 2025; Orlando, FL. Abstract #538