Swedish Orphan Biovitrum AB (publ) KOL Event Call Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Christopher Winston Uhde - SEB, Research Division - Analyst : I have quite a few, so I'll start and get back in the queue. But I guess -- Professor Mascarenhas, to begin with. So I guess when we -- when one looks at the forecast that people have out there, it's clear that there are a lot of patients who -- the market, let's say, does not expect to get on pacritinib who are severely thrombocytopenic. So what proportion of these patients are on some kind of therapy? And what are the main obstacles to getting REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JULY 19, 2023 / 1:30PM, SOBIV.ST - Swedish Orphan Biovitrum AB (publ) KOL Event Call more of them on therapy and on pacritinib in particular. And I guess sort of related to that, I mean, yes, so what therapy do most severe thrombocytopenic myelofibrosis patients get today? And how much is off-label RUX? John Mascarenhas So I think historically, patients with extreme thrombocytopenia, we're typically getting nothing because nothing could be delivered effectively. And I hate to say that many of these patients ended up in hospice, and we're simply getting supportive care or low doses of ruxolitinib, which provides minimal benefit, but definitely not maximal or optimized benefit. So you would have patients sitting on 5 milligrams once a day, 5 milligrams twice a day of ruxolitinib with some degree of symptom benefit, very minimal spleen improvement. It was suboptimal to say the least. I think when pacritinib was approved, it was a wealth in addition because it was not simply another JAK inhibitor. This was a JAK inhibitor that met a need in the less than 50,000, which was excluding -- so to answer your question of what percentage of patients would be eligible for pacritinib? Probably 80% of patients with platelet counts less than 50,000. I couldn't tell you what percentage are actually getting pacritinib now. I can tell you that it's increasing. I do webinars with physicians across the country, in different geographic areas in the U.S. And what is remarkable and consistent across those webinars with small groups of 10 to 12 physicians is that there's already a recognition within the community for a drug that was only approved in March of 2022. There's already a recognition of pacritinib, the name Vonjo, the product label, where the drug would be used and even a willingness and interest in using it in those patients. So I'm not seeing any resistance or concern about using pacritinib in those patients. And the reality is because it really isn't a great drug to use. So this drug becomes a very obvious choice for those patients. I think that the -- to try to answer the rest of the question, I think that the -- where this moves from now is for those patients who are sitting on inadequate low doses of ruxolitinib is the comfort level of the practitioners to then switch them over to pacritinib. And of course, it's always hard, I think, when you have over a decade of experience with one JAK inhibitor, this is a ramp-up of providing another option and the confidence to move to that option. So what I typically see are patients -- physicians are prescribing pacritinib to the patients who couldn't get anything, they get that comfort level and then they start to look at the patients who are on suboptimal doses of ruxolitinib with an eye to start switching them to pacritinib to try to maximize benefit. So I think you'll see increasing utilization of patients switching from inadequate low doses of RUX to pacritinib. We also see a lot of bleed-over. I myself, for sure, do it. I see a lot of my colleagues doing it where even if the label is 50,000 or less, if you have someone who has a platelet count of 75,000, you know that if you try to deliver effective doses of RUX, you will simply compromise the platelet count and then get into the cycle where you end up dose reducing and then limiting the effect of ruxolitinib. So even in those patients, many physicians now are adopting an approach where they are looking at to obtain pacritinib. And I have to say, it's not an obstacle to obtain pacritinib. So I wouldn't say that insurance approval has been an obstacle and getting pacritinib for their patients, even slightly above that 50,000 range. And I think that, that will continue to expand as the comfort level with the drug increases.