Pharming Group N.V.

Good day and thank you for standing by. Welcome to the Farming Group NV Fall Year 2023 Resorts Conference Call and Webcast. At this time, all participants are in a listen-only mode. After the speaker's presentation, there'll be a question and answer session. As a reminder, the company will only take questions from dial-in participants. 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 one and one again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Simon de Vries, CEO of Farming Group. Please go ahead, sir.

Thank you very much, Breda. Welcome, ladies and gentlemen. Good morning, good afternoon, wherever you are to our conference. Please next slide. And we're very happy to take you to the full year results. And you can give me the next slides because before I do that, I would like to point out to that forward-looking statements slides that you now see where we will be making forward-looking statements that are based upon our current beliefs and expectations, which may, of course, differ from what we expect. So having said that, I would like to now go to the next slide where you can see my face and then move on immediately to the next slide. Thank you very much. That's the one. Yes, we are indeed looking back to 2023 as a very successful year where by delivering strong growth, we built the foundation for that global rare disease company that we are setting out to build. And that's, we can build that, we can all, you know, finance that by, of course, the cash flows that come from the marketing of Rookernest, which delivers considerable positive cash flows that can help us to build that foundation further. So we delivered, we're very pleased that we delivered to more than $227 million of revenue, which is a 10% growth versus last year, which exceeded significantly the expected single-digit growth for Rookernest. and we saw that because we had some very good parameters forward-looking parameters that that were really working the number of patients increased numbers of prescribers increased and you know basically it means that patients continue to be reliant on rookiness despite increased therapy options that are available in the market which of course are good for patients but you can see that rookiness continues to be that reliable cornerstone and My colleague, Tor, will elaborate a little bit further on that, obviously. Now, the next exciting bit, of course, that we could, therefore, start to execute on was the launch in the United States for Joangia Leniolisip, which is a product we in-licensed from Novartis in 2019. And we were very proud to actually, you know, almost immediately after FDA approval in April, bring that product to the market and immediately get commercial coverage for that product and bring in more than 18 millions of sales during those first nine months in the market. In addition to that, Gioenja has been filed with a number of regulatory authorities in Europe, Canada, Australia, Israel, and as of two days ago in the United Kingdom, And we are awaiting, of course, and we're working with all these regulatory authorities, and we are awaiting, you know, the next steps and the necessary approvals in the not too distant future. Then, of course, very important, the label of Joenja is for 12 years and older. We have a number of pediatric trials that are ongoing, which are, of course, important. And last but not least, we also have a completed small Japanese clinical trial. So we are preparing for submission to Japanese authorities by the end of this year. And my colleague Anurag Rallon, our chief medical officer, will talk more about that. And as it is an ultra rare disease and a very new disease that we only described 10 years ago, we have a very strong focus on patient finding. And again, Dr. Anurag Rallon will talk about that later. And then I move to the right hand side, which, of course, is even more exciting going forward towards the future. because we strongly believe that laniolisib has vast additional potential for further development, and hence we have basically set out to start a Phase IIb study in the second quarter of this year for the subsequent indication, which is significantly larger than APDS in patient numbers, PID with immune dysregulation, And again, Anurag Rehland will talk about that in more detail a little bit later in this presentation. And last but not least, we are very, very active. We continue to be very active to in-license or acquire clinical stage programs in rare diseases in preferably immunology, hematology, respiratory, and gastroenterology to further leverage our commercialization structure that we have been building up and are building up as we speak. And then on the bottom of the slide, you see that for the first time in our history, we gave total revenue guidance, and that will be between 280 and 295 million dollars, which is driven, of course, by Juenja, but strongly supported by Rukunesta during 2024. And then I would like to go to the next slide, please, where you see then a depiction of the pipeline that we have, RUCONEST obviously in the market, JOENJA in the U.S. market, and you see that subsequently on the regulatory review by many of the regulatory authorities, and the pediatric program that's ongoing, and the Japan program that's ongoing. And then, of course, the very exciting fact that we are going to do this, the Phase IIa study for lanylysis for the subsequent indication. And last but not least, our very early stage program, the HAE gene therapy, that is in early clinical, preclinical stage. With that said, I would like to turn over to my colleague, Stephen Tora, Chief Commercial Officer, to take you through the commercial update. Thank you.

Stephen Tora Thank you, Simon. Good morning, good afternoon, everybody. So, this is a slide I think you'll be largely familiar with, and I think the key thing to take away from this is that the key features of Rucanus, so namely The only recombinant C1, treating the root cause of the disease, and 97% efficacy in one dose is what continues to fuel the Ruconest growth and our success over the last nine years. These product features are allied to the excellent work our commercial, medical, and patient services teams deliver for customers and patients and their carers is also why Ruconest remains a highly relevant part of the conversation in the USHA community and globally. That's remained the case despite three prophylactic launches over the last few years, leading to generally better controlled patients and the genericization of a catapult. And I fully expect it to remain the case even in the face of acute competition in the future. And that's because HA patients using Rucanus tend to have generally a more severe course of disease. And in that instance, the need for virtually guaranteed and fast efficacy that stops the attack in its tracks is critical. And it's typically the patient's only real objective. In most cases, that overriding need for efficacy won't be replaced by a convenience play. So looking at 2023, RUCAN-S performance was characterized by the continued growth in both prescribers and new patient enrollments. And it was also successful despite that market-wide event we saw in Q1 last year related to government-reimbursed patients. Now, we have seen some disruption from that this year, but it's been muted by the almost halving of those patients' out-of-pocket costs in the U.S. And moving into 24, we've also seen that the strength of our leading indicators has continued into this year, namely new prescribers and continued support from existing prescribers and new patient enrollments. So despite some continued disruption in that government segment, we're still on track for where we expect to be in Q1. Next slide, please. So this is, again, something you're very familiar with. Farmers have been in this space and committed to this community for over 20 years now. And it's really that commitment combined with the product itself, Ruconest, our people, and the excellence they deliver, which is why you see on the right-hand side there, our prescriber base continues to grow, and as a result, our patient base continues to grow. And it's why, as I said, we remain enthused and confident in the continued growth of Ruconest both this year and in the years ahead. Next slide, please. So switching gears now to Joengia. So switching gears to Joengia, we had, as you know, a strong first year. And as we close out that first year at the end of this month, I'd just like to reinforce what a great launch the team here at Farming delivered for us. So the launch preparation, as you've heard me say in the past, was first class, and I think I wouldn't underplay what Simon said earlier, which is that within one week of approval, we had commercially covered patients with product in hand, which is outstanding. The patient base steadily grew throughout the year, and we exited, as you know, at the end of the year with 81 patients on paid Joe Inger therapy. In that time, importantly, we've seen very few discontinuations, and we have adherence rates close to 90%. So as we convert that caseload that we'd already identified at launch, our focus unsurprisingly moving forward remains finding those new patients for this rare disease, and then given APDS is an autosomal-dominant condition, testing their families to uncover additional patients so that they too can benefit from the management of treatment with GeoEngine, which, as you know, is the only indicated product with which you can treat APDS. And then the other important factor to talk about here in addition to the U.S. is of course alongside that launch, we continue to build out our ex-U.S. capabilities in preparation for launches in the European Union, the United Kingdom, Japan, Australia, and other countries in the Asia-Pacific region, and also in the Middle East and here in North America and Canada. So with that, I'd like to pass over now to Dr. Allen, who's our Chief Medical Officer for a medical update.