General Updates | Phase III study of rurioctocog alfa pegol in PUPs with severe hemophilia A: safety & efficacy data

Yeah, so this year I presented a late-breaking abstract. It was a phase three prospective study, a multi-center study, and it was looking at the safety, immunogenicity, and hemostatic efficacy of rurioctocog alfa in patients less than six years of age. This was a two-part trial. The first part of the trial included patients under the age of six years with severe hemophilia A, and they were either started on prophylaxis or on demand with rurioctocog alfa, which is also called Adynovate. And the primary endpoint was incidence of inhibitor development. And in the analysis, there were 100 that were evaluated that had at least 100 exposure days. And of those 100 patients, only 11% developed an inhibitor, which is significantly lower than what was reported in other trials. Five of those inhibitors were low titer and six were high titer inhibitors. And the average time to development of the inhibitor is about 7.6 exposure days, and nobody developed an inhibitor after 20 days. The efficacy was good. The average on-demand annualized bleed rate was about 2.3. And on prophylaxis, it was between 0.96 and 1.7, so very low. The response to bleeds was greater than 95% reported as excellent or good for the number of infusions needed for a bleed was typically one to two. And for the second part of the study, they wanted to look at how patients responded to immune tolerance induction. And so there were 11 patients that developed an inhibitor. Eight of them enrolled. Ultimately, six of them underwent ITI or seven of them underwent ITI. And what they found was that five of the patients had success. And that’s with the dosing regimen of 50 units per kilo three times a week or 100 to 200 units per kilo a day. And one of the patients had partial success and one of them was a failure. So very high success rate and there were no hypersensitivity reactions related to that immune tolerance. Yeah. So this was exciting to show that there were a very low incidence of inhibitors. And so now people are wondering, what do you do if you have a new baby with severe hemophilia A? So, you know, now it’s 2025 when the study started, emicizumab was not FDA approved. And so it’s clearly changed. And so in the United States, you certainly will see patients continue to be started on emicizumab as a young child. I think what will happen is as though people may consider using this option as their on-demand therapy or their option for surgical treatment. And certainly if you’re a patient or a family that wants to not be on emicizumab, you could certainly start this in patients as well. You certainly could consider giving 20 exposure days and then decide to switch to emicizumab at that time. So I think the options are still there as before, but what we’ll likely see is more patients shift to Adynovate as their on-demand therapy.

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