About Matthew Kapusta
Matthew Kapusta, CEO of uniQure, has discussed the company's gene therapy Hemgenix, which he described as a one-time treatment for Hemophilia B that provides constant Factor 9 expression. Kapusta stated that Hemgenix is now offered to 10,000 to 15,000 patients across Europe and the United States, and he noted that clinical data from three studies involving about 70 patients showed a greater than 90% reduction in infusions, with nearly all patients remaining off prophylactic replacement therapy. He characterized the therapy as potentially more cost-effective than existing treatments, citing annual replacement therapy costs of $500,000 to $600,000 for severe Hemophilia B.
Kapusta has also highlighted uniQure's history as a gene therapy pioneer, noting the company was founded in 1998 and achieved two approved gene therapies. He emphasized the company's investment in manufacturing capabilities, stating that uniQure controls its manufacturing process and built facilities from the ground up. In earlier remarks, Kapusta discussed the economic value of gene therapies, describing the potential for a one-time administration to replace ongoing high-cost treatments, and noted that pricing would require discussions with payers.
Source: AI-verified profile updated from Matthew Kapusta's recent appearances.
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Transcript (9 segments)
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Matthew Kapusta0:00
The purpose of gene therapy is for one-time administration that can have very durable effects. Obviously proving that it's curative requires time, but the first gene therapies in hemophilia have now followed patients for more than eight years and showed durable effects, so it's looking very good.
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Interviewer0:16
The more pivotal trial will be the Phase 3, and I understand that you are currently enrolling patients and are finding great interest in that. When can we expect the first readout from the Phase 3, and also when could we expect possibly for this candidate to hit the market?
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Matthew Kapusta0:36
Yeah, so certainly those are the important questions. We haven't provided public guidance on that, but we did announce yesterday some very positive results for our Phase 2b study. We are currently enrolling patients in the pivotal study. We will be providing guidance on the process of enrollment early next year. Our first goal is to complete the enrollment of the study. We'll be looking at Factor 9 activity, which is the biomarker for efficacy, about six months after we treat the last patient, and then bleeding data after one year of enrolling the last patient as well.
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Interviewer1:07
How big is the total addressable market for this drug?
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Matthew Kapusta1:11
Well, there's probably around 400,000 people in the world that have hemophilia. Approximately 70,000 of those have hemophilia B. Today there's probably more than $10 billion of revenue predominantly going after factor replacement for those patients that require infusions. So the notion of that much revenue a year in factor replacement being replaced by a one-time administration is pretty attractive for the patients and certainly the healthcare economic community.
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Interviewer1:42
400,000 worldwide. How many of them are in the United States?
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Matthew Kapusta1:47
Well, there's probably around five to seven thousand patients with hemophilia B in the United States, and probably four times that many that have hemophilia A.
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Interviewer1:59
And in terms of the price tag, the piece that we played a profile of BioMarin treatment and Meg had said that that would be seven figures likely. Is that what we're looking at in terms of what the cost to the patient would be?
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Matthew Kapusta2:12
Well, what I'll tell you is this: the cost of care for a severe hemophilia patient, for those infusions that they take, can be somewhere around six to seven hundred thousand dollars a year, and that's for the rest of that patient's life. So if you think about the cost of care for a severe hemophilia patient for their entire life, it's very significant. And so if you have a gene therapy that can potentially provide curative benefit, there's no doubt significant healthcare economic value. And obviously the exact price of that will have to require discussions with the payer community as well.
