Pharming Group N.V.

good day and thank you for standing by welcome to the farming group fourth quarter full year 2024 results 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 to ask a question during the session you will need to press star one and one 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, Fabrice Chiraki, CEO. Please go ahead, sir.

Thank you very much. Hello, everyone, and welcome to the Farming Full Year and Q4 2024 Financial Results Call. I'm Fabrice Chiraki, CEO of Farming, and I'll be joined on this call today by Stephen Torr, our Chief Commercial Officer, Anurag Relen, our Chief Medical Officer, and Jeroen Wackermann, our chief financial officer. We will be making forward-looking statements in this call that are based upon our current insights and plans. As you know, this may differ from future results. First of all, let me say that I'm really excited to be joining farming, and it's an honor to succeed Simon De Vries. I'm passionate about progressing medical sciences and bringing innovation to patients. And naturally, I feel deeply connected with farming's mission to serve the unserved rare disease patients. Over the past 25 years, I've developed an experience across the business spectrum, from clinical research and clinical development to commercial leadership, business development, and capital formation. I've been able to experience the best of the two worlds. the rigor and sophistication of big pharma, and the value creation mindset and agility of venture capital and biotech. In light of this experience, I'm impressed with the development of farming over the past 15 years and its significant growth prospects. Rookernest has become one of the cornerstone on-demand treatments for HAE. Johanja is already approved and launched in the US for the treatment of APDS, with a significant number of patients already on treatment, and it is due to be launched in other key markets around the world. And last but not least, the recent completion of the acquisition of Abliva is another stepping stone in the development of the company. We have a clear vision for farming, which is to develop a leading global rare disease company with a diverse portfolio and presence in large markets, leveraging a proven and efficient clinical development, supply chain, and commercial infrastructure. Our results in 2024 are a good illustration of the solid foundation that we have built to realize this vision. Full year 2024 revenues increased by 21% to $297 million above our guidance range, including a strong fourth quarter and with operating profits and positive operating cash flow in the last two quarters of the year. Ruconest grew 11% to $252 million and 9% in the last quarter of the year. driven by a continued increase of new patient enrollment and the sustained expansion of our prescriber base. I believe that given its unique profile and positive experience in difficult-to-treat patients, Reconest will continue to grow and could even benefit from the potential increase of the on-demand segment driven by the entry of new entrants. JoinJar revenue increased by 147% to $45 million in 2024. The drug is only in its very early stage of its life cycle, with continued growth to be seen with the enrollment of new APDS patients in the U.S., the launch in key markets, including the U.K. in the coming months, and several well-defined opportunities to expand the addressable patient population, including the pediatric label extension and the development for much larger primary immunodeficiency indications. Let me now hand over to Steve Torr, our chief commercial officer, who will give you a more granular perspective on the strong dynamic of RECONNECT and JOINJUST.

Thank you, Fabrice. Good morning, everybody. As Fabrice just alluded to, we've delivered another strong performance in 2024. On RUCNES, we increased the prescriber base by 11% and new patient enrollments by 24%. This translated to a strong Q4 growing 9% over prior year and hitting almost 80 million for the quarter. We ended 2024 with sales of 252 million, 11% up on 2023. In the next two slides, I'll review why Rukiness continues to show such strength and growth and why we're confident it will continue to grow in the years to come, even as the market becomes more competitive. On Joengia, we continue to build our patient pipeline and transition eligible patients to paid therapy. And as you would expect, our team delivered significant growth over the first year of launch, ending Q4 65% up on prior year at $13.1 million, and for 2024, plus 147% of $45 million. Of note, in addition to 96 patients plus five pending on paid therapy in the U.S., we have an additional 188 patients on therapy globally under various access programs and in clinical trials that can all move to commercial therapy when the necessary registrations are received. In the forthcoming slides, I'll also outline the opportunities we see in the coming months and years that will both build the Joe Inger business for APDS and with new potential indications for the molecule, create a strong, high-growth franchise. Looking first at ruchinus, as I just stated, ruchinus is and will continue to be a growth driver for farming and an important treatment option for US patients and their doctors, which is why it's already the second most prescribed acute product in the US. And one of the key reasons for this is its mode of action. As you can see in the graphic, there are three inflammatory cascades involved in the development of an HAE attack. C1 esterase inhibition, represented in the graphic by the red C1INH markers, blocks numerous enzymes across all three pathways. So, while many patients are effectively treated by blocking a single point in these cascades, patients who don't respond to the target therapies available may benefit from Rucanus, since it works comprehensively across all these systems. C1 esterase inhibition ultimately stops bradykinin production via multiple points in the contact cascade, as well as other systems that may lead to attacks, which in turn, and this is important, leads to the 97% attack resolution in a single dose and a sustained response with 93% of patients' attacks stopped for at least three days. So let's look more specifically at Rukinus patients and what this means for them. The first thing to note is that Rucanus serves all patient types, those being type 1, type 2, and normal C1 patients. All three of these Rucanus patient groups have one thing in common, though. They all suffer from moderate to severe debilitating HA attacks, and they have them frequently. They've also typically failed other targeted acute therapies, such as a catapult or having to redose to stop their HA attacks. In the photos on the slide, you can see an actual ruchinous patient at the start of an attack, and then her recovery as it resolves at the four-hour mark and the 24-hour mark. For patients like this one suffering with a more severe course of disease, attacking frequently, and happen to redose on other therapies, knowing, as I just stated, that 97% of patients will stop their attack with a single dose, and almost all of them will be attack-free for at least three days is a very big deal. Rukiness efficacy and reliability allows our patients to better control and plan their lives, and that's why Rukiness will continue to have a strong position in the U.S. acute market and remain an important product for our company in the years to come. I'll transition now to Joinger, which, as you're aware, was launched in the U.S. in March 2023 and is available outside the U.S. through various access programs. We see a number of opportunities for Joinger, which I'll walk you through now. that farming's patient funding efforts are continuing as we build our patient pipeline in the U.S. and globally. In fact, we've already identified over 240 patients in the U.S. of whom 40% are already on paid therapy, and we've identified hundreds more in other key markets. So while we work hard to continue to pull through those identified patients and put them on therapy, we also have some important opportunities to drive growth in the near term and the medium term including efforts to expand the addressable population. So what are they? Looking at the second block on the slide, the first is the outputs from the VUS resolution program, which Anurag will discuss. That will deliver another bonus of APDS patients available for treatment this year and beyond. The second will be the pediatric indication launched in the U.S., which is expected in 2026. We currently have over 60 patients in our US pipeline and growing. And they will begin transitioning to Joengia as soon as the indication is approved. And the third is our geographic expansion program, which is to key markets around the world. And this begins this year with the UK launch. In fact, just today, NICE have published draft guidance in which they recommend the use of Joengia for NHS England and Wales. And then we have further anticipated launches in other important markets, including Japan, Germany, France, Italy, Spain, Canada, and Australia. That means Joe Inge will soon be available in most of the industry's top 10 markets. In addition to that, you can see in the final two blocks on this slide, Leniola-Cypher APDS is only part of the story. As you know, phase two trials have been initiated for two bigger indications, LPID and CVID. In fact, CVID, while still rare with a prevalence of 40 patients per million, transitions Leniolisib from a small, ultra-rare disease molecule to one with blockbuster sales potential, thereby creating a Leniolisib franchise delivering a significantly greater value for all stakeholders in the coming years. With that said, I'd now like to hand over to our Chief Medical Officer, Anna Agrelin, whose team are, of course, critical to driving these programs forward and realizing these opportunities, to provide us with a research and development update.