Name:
ECTRIMS 2022 – An interview with Professor Friedemann Paul & Horizon Therapeutics’ Kristina Patterson
Description:
ECTRIMS 2022 – An interview with Professor Friedemann Paul & Horizon Therapeutics’ Kristina Patterson
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Duration:
T00H05M54S
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Upload Date:
2022-11-21T00:00:00.0000000
Transcript:
Language: EN.
Segment:0 .
[MUSIC PLAYING]
FRIEDEMANN PAUL: Until very recently, we used to treat NMOSD which is a severe devastating autoimmune disease of the central nervous system with empirical drugs such as broad immunosuppressants or anti-CD20 and therapies that were not tested in randomized controlled trials. So the advent of these trials, especially the product by Horizon as well as the two other drugs that are now on the market are truly a game changer for treating NMOSD.
FRIEDEMANN PAUL:
KRISTINA PATTERSON: Neoromyelitis optica spectrum disorder is a rare but devastating disease in which even just one relapse can completely alter a patient's life. And since there were no approved therapies at the time, we knew that there was a chance to make a difference in patients' lives by bringing forth a therapy that could reduce the chances of this happening to them. That combined with the strong scientific rationale that CD19 positive B cells are key drivers of the cause of the pathogenesis of this disease, were basically the two reasons why we decided to go after this indication.
KRISTINA PATTERSON:
FRIEDEMANN PAUL: So this data clearly shows-- and this is data that stems from an RCT in contrast to as I said the previously used drugs on an empirical basis. This data clearly shows that this is a drug that is highly efficacious in reducing attack rates and risk of attacks in patients with NMOSD. And because in this condition the neurological disability emerges from attacks from incomplete recovery from attacks, prevention of attacks is the key therapeutic goal in NMOSD.
FRIEDEMANN PAUL: And this is achievable now with Uplizna. This was the largest RCT performed in this condition to date with more than 200 patients enrolled, most of them Echo 0.4 antibody positive. And there was a highly significant reduction in risk of attacks, more than 70% up to 77% in patients with the Echo 0.4 antibody compared to placebo.
FRIEDEMANN PAUL: Also, the open label extension of this study showed a clear and beneficial effect over longer periods of time. So, again, this is a highly significant and very impressive result that clearly shows that this is a drug that is very efficacious in preventing attacks in NMOSD.
KRISTINA PATTERSON: Well, it's actually really hard to choose because I'm really excited about all of our poster sessions. But the one that, I guess, that I would have to pick is the one where we show that the mechanism of action of an apolizumab really distinctly gets at some of the key causes of NMOSD. So we know that CD19 positive plasmablast and plasma cells produce aquaporin-4 antibodies. And these antibodies cause widespread destruction of the central nervous system.
KRISTINA PATTERSON: So in our poster we show that CD19 positive plasmablast and plasma cells are not only elevated during NMOSD attacks, but an apolizumab actually decreases, effectively depletes these plasmablast and plasma cells but also reduces aquaporin-4 antibody titers.
FRIEDEMANN PAUL: First of all, to see these new drugs that are now fortunately available finally after many years of clinical trials that they will find their way to patients and treating physicians and will improve lives of patients with NMOSD. The second point is I hope to see advancements in the field of symptomatic therapy. This is still a very strong unmet medical need, for example, pain in [? most-- ?] the vast majority of patients suffer from pain that is not treatable with the drugs we have.
FRIEDEMANN PAUL: So this is another issue that also should be addressed by research. Another point is cognitive dysfunction very frequent in NMOSD. So I hope to see more advancements there. And as a very, yeah, far-fetched perhaps long term goal would be neuro restoration or tolerizing therapies for NMOSD.
KRISTINA PATTERSON: Well, there's actually a lot. So I don't know that I have time to go into all of them. But I will focus specifically on an apolizumab and what our future plans are there. We currently have two phase three clinical trials, one in myasthenia gravis, where we look at both AChR-positive and MuSK-positive subsets separately and also IgG4-related disease for which there is no approved medication at this time. We also recently announced a phase two trial in pediatric NMO.
KRISTINA PATTERSON: [MUSIC PLAYING]