MedicalResearch.com Interview with:

Steven Pipe, MDProfessor of Pediatrics and PathologyLaurence A. Boxer Research Professor of Pediatrics and Communicable DiseasesPediatric Medical Director, Hemophilia and Coagulation Disorders ProgramDirector, Special Coagulation LaboratoryUniversity of Michigan

MedicalResearch.com: What is the background for this study?

Response: Hemophilia B is an inherited bleeding disorder where patients are missing clotting factor IX (9), a critical blood clotting protein.Patients with a severe deficiency are at risk for traumatic and spontaneous bleeds primarily into joints.Repeated bleeding into joints causes more than acute pain and swelling but also leads to inflammatory and degenerative changes in joints that eventually leads to severe debilitating arthritis that can be crippling.To try to prevent this, patients as young as infants are placed on regular infusions of clotting factor IX concentrates.The relatively short half-life of factor IX means patients must infuse on average once to twice a week.These can only be delivered intravenously parents and then patients themselves have to learn this.Prophylaxis must be continued lifelong to try to prevent bleeding events and protect joint health over the lifespan.This is a tremendous burden on the patient and their caregivers.

Even with regular prophylaxis, joint bleeds may still occur and arthropathy may still ensue.This is because the blood levels often reach critically low levels prior to the next infusion.Gene therapy aims to deliver a functional copy of the factor IX gene such that the patients own liver will make a continuous supply of factor IX that is delivered to the bloodstream.At steady state with levels close to or within the normal range, patients would no longer be subject to bleeding events and would not require prophylaxis any longer.We hope that such a one-time treatment would produced durable, functionally curative levels of factor IX.

MedicalResearch.com: What are the main findings?

Response: The 3 key takeaways are:

MedicalResearch.com: What should readers take away from your report?

Response: I think it is best to characterize this as a treatment that liberates patients with hemophilia from the burden of repeated prophylactic infusions of clotting factor to protect them from recurrent bleeding events.Gene therapy offers a chance to have steady state levels of factor in the blood that would eliminate risk for spontaneous and even traumatic bleeding events.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: These are very encouraging results maximizes eligibility for patients by being able to treat in the presence of pre-existing neutralizing antibodies.Results are in a range that allow for cessation of prophylaxis and continued bleed protection.Results appear to be durable through the follow up period to date.The 26 week endpoint was a co-primary endpoint.The other endpoints are factor IX activity at 52 weeks and annualized bleeding rate over the 52 weeks post-dosing. Those endpoints should be analyzed in 2021 and decisions could then be made on submission for regulatory review.

MedicalResearch.com: Is there anything else you would like to add? Any disclosures?

Response: This could be potentially clinical practice changing, if approved. This looks to be a transformative therapy for patients and could be an option for patients across the adult lifespan we treated patients from age 19 through 75. Our patient population have been looking forward for a long time for a one-time therapy like this that would provide lasting protection from bleeds.

I have served as a paid consultant to uniQure and chair the Steering Committee for the global clinical trial program

Citation: ASH2020 Oral Abstract

First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII

Steven W. Pipe, MD1, Francesca Ferrante, MD2*, Muriel Reis3*, Sara Wiegmann4*, Claudia Lange5*, Manuela Braun5*and Lisa A Michaels6*https://ash.confex.com/ash/2020/webprogram/Paper139803.html

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Gene Therapy Liberates Hemophilia B Patients from Requiring Regular Infusions of Clotting Factor - MedicalResearch.com

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