Additional Advances in Hemophilia Research and Clinical Trials With Guy Young, MD

Dr. Guy Young, Director of the Hemostasis and Thrombosis Program, Attending Physician in Hematology-Oncology, and Professor of Pediatrics at Keck School of Medicine at the University of Southern California (USC), is currently serving as co-chair of i3 Health’s CME/NCPD activity, Practice-Changing Advances in the Management of Hemophilia. With new developments in the field occurring over recent months, Dr. Young sat down with us to share recent updates in the treatment and management of hemophilia.

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Online Accredited CME/NCPD Activity

Key Developments in Hemophilia Treatment

1. Monoclonal Antibodies for Rebalancing Hemostasis

Two new monoclonal antibodies, marstacimab and concizumab, have been approved, marking significant steps forward:

• Marstacimab: Approved for hemophilia A and B (without inhibitors) in patients older than 12
• Concizumab: Approved for hemophilia A and B (with inhibitors) for the same age group

“These drugs offer new options for patients, and while their indications currently do not overlap, future studies aim to expand their applications to include pediatric populations,” Dr. Young explained.

1. Gene Therapy Advances

Recent data on gene therapy, such as giroctocogene fitelparvovec for hemophilia A, highlight the following:

• Higher factor levels and reduced steroid requirements compared to earlier therapies like valoctocogene roxaparvovec
• Expected licensing of this therapy later in 2025

Dr. Young remarked, “The new data from giroctocogene fitelparvovec suggest it may address some of the limitations seen in earlier gene therapies, such as the need for steroids due to elevated liver enzymes.”

Additionally, novel gene therapy strategies, including B-cell–based therapies and gene editing trials for hemophilia B, are set to address limitations of current adeno-associated virus (AAV)–mediated gene therapies. “These approaches aim to overcome some of the challenges we’ve seen and potentially offer more durable solutions,” he noted.

1. Innovative Agents for Bleed Management

• Fitusiran: Data show improved bleed management with fewer doses required during bleeding episodes. This agent, administered every other month, exemplifies the push for more convenient treatment regimens

“Bleeds are infrequent with fitusiran, but when they do occur, management is straightforward and requires fewer interventions,” Dr. Young highlighted.

1. Oral Therapeutics

An oral bispecific antibody, under preclinical evaluation, is expected to begin clinical trials. If successful, it will be the first oral agent specifically targeting hemophilia management.

“This would be a truly unique advance because every treatment we currently use, whether for prevention or to treat bleeds, is either subcutaneous or intravenous,” Dr. Young shared.

Educational Imperatives for Practitioners

Dr. Young emphasized the importance of continuous education to keep pace with the rapidly evolving treatment landscape. Since 2013, hemophilia care has seen revolutionary changes, including:

• Extended half-life factor therapies
• Subcutaneous prophylaxis drugs like emicizumab
• The emergence of gene therapies and rebalancing agents

“If you don’t know about the new treatments, how are you going to offer them to your patients?” Dr. Young asked. “Hemophilia care has gotten much more complex because we now have more choices, which is great, but it also requires ongoing education.”

Future Directions

Ongoing trials and innovative approaches promise significant advancements:

• Clinical trials for oral agents and gene editing techniques
• Research on expanding indications for recently approved therapies

“Later this year, we’re likely to see the first clinical trial for gene editing in hemophilia B,” Dr. Young revealed. “It’s exciting to consider how this might transform the future of care.”

Each therapy presents unique nuances in administration, dosing, and monitoring, requiring tailored patient management.

Key Takeaways

• New Approvals: Marstacimab and concizumab expand treatment options for hemophilia A and B
• Gene Therapy: Promising results from giroctocogene fitelparvovec and novel approaches like B-cell-based therapies
• Education: Staying updated is critical as treatments grow more complex
• Emerging Therapies: Oral agents and gene editing trials herald a new era in hemophilia care

Conclusion

The treatment of hemophilia is at an exciting juncture, with multiple new therapies reshaping care standards. Dr. Young advises healthcare providers to prioritize education and engagement with emerging research to ensure optimal patient outcomes. With innovation accelerating, the future of hemophilia management is brighter than ever.

About Dr. Young

Guy Young, MD, is Director of the Hemostasis and Thrombosis Program, Attending Physician in Hematology-Oncology, and Professor of Pediatrics at Keck School of Medicine at the University of Southern California (USC). Dr. Young has worked extensively on advancements in his field having published journal articles and abstracts and has delivered lectures, workshops, and presentations at national and international meetings.