Pharming Group N.V.

Good day and thank you for standing by. Welcome to the Farming Group NVQ1 2024 results conference call and webcast. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 1 1 again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Simon De Vries. Please go ahead.

Thank you very much, Sandra, and welcome, ladies and gentlemen, to this first quarter results conference. I'm here with my next slide, please. I'm here with my three colleagues in order of speaking. Stephen Tor, our chief commercial officer, who's joining us from our New Jersey office. Dr. Anurag Rallan, our chief medical officer, joining us from our U.S. office as well. And Jeroen Wakkerman, our chief financial officer, who's based with me here in Leiden. And before I do that, I would like to point out to the next slide, please, number three. That is the forward-looking statement slides. We will be making forward-looking statements today in our presentation, and those are based upon our current plans and, you know, and insights and situations of the current market circumstance. And of course, actual results may differ from these forward-looking statements, so you cannot necessarily rely on those. So having said that, I would like to go to slide number four, and maybe even onwards to slide number five, because you've seen my picture already. yes and here we are we're building this global rare leading global rare disease biopharma company and we have we do that uh based on three pillars and this is a very familiar slice for you who have been here before on the left hand side the foundation of our company the product ruconest that comes from our own research and uh it has already been on the us market for almost 10 years it was approved in july july 2014 recombinant protein replacement therapy for hereditary angioedema attacks. And as you can see, we are still growing this product. And we had an 8% growth versus last year, this quarter. Good growth last year as well. and we see increasing numbers of prescribers and patients. And there's many options for these patients available, but Ruconest continues to play an important role, and we expect it to be continuing for the foreseeable future. And Steven will talk a lot more about that in his part of the presentation. Next, you see there Joenja, the product we in-licensed from Novartis for the treatment of APS, an ultra-rare immune disorder, a new disease that was only discovered 10 years ago, and we already have the first disease-modifying drug on the market that we launched now a year ago. And you see we had a very successful introduction of the product in the U.S. market. Stephen will allude to that as well. We're very proud that we can actually record 9.6 million sales in this quarter, good growth versus 4Q of last year. And you see here, if you add both numbers, we realized 28 million dollars of sales for this product in the first 12 months on the market. It's new to describe an ultra rare disease and there's a very strong focus therefore on patient finding. Both Steven and Anurag will talk about that, how we go about that to find those patients. We're also very pleased to see that very recently we got the second approval in Israel at the end of April 24. We have a lot of regulatory reviews ongoing and a lot of trials ongoing and Anurag will talk about that a lot more. And on the right hand side, the the possibility to leverage our commercialization infrastructure both in the us and outside of the us by not only uh you know finding new uh compounds for hunting for uh for clinical stage or later stage compounds uh or to in license or acquire to actually leverage but First and foremost, we have a very interesting opportunity in laniolisib to develop it further for a second indication for primary immune deficiencies with immune dysregulation beyond APDS, where we are preparing in final stages for preparing a phase two study. And in addition, we're working on a third primary immune deficiency indication in early stage at this point in time. And Anurag will talk about that a lot more. And on the bottom of the slide, you see that we have a a total revenue guidance out between $280 and $295 million for this year, you know, driven, of course, by Juangia, but, you know, the foundation is, of course, Rufinest underneath that. And then you see the next slide, a little bit more in detail, because we're going to talk a lot about the potential for Juangia today. Juangia for APDS is the first stage where we are currently now in the market with a 12-plus indication in the U.S., where we already found a significant portion of the identified patients on paid therapy. We have a lot of ongoing search for patients and so-called variants of uncertain significant mutations that's ongoing. The next step, of course, where you already see, and that's, of course, an interesting observation, almost $1.1 million of sales in this quarter already outside of the U.S. So, in other words, We're starting to work on the global expansion of the product and the pediatric studies will further boost that to get a full label and full geographic coverage for geoengine APDS. And then, as I said already earlier, the bigger indication that we are starting a concept trial in the not-too-distant future for the bigger indication of PIDs with immune dysregulation with similar symptomatology to APDS. So having given you this introduction, I would like to now point out, I would like to now hand over to my next speaker, to Steven Tor, our Chief Commercial Officer. Steve, over to you, please.

Thank you, Simon. Thank you, everybody.