Pharming Group N.V.

Good day and thank you for standing by. Welcome to the Farming Group NV fourth quarter and full year 2025 Financial Reserve 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, please 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 note that today's conference is being recorded. I would now like to hand the conference over to your first speaker, Mr. Fabrice Chiraki, CEO. Please go ahead, Sal.

Thank you, operator. Good morning and good afternoon, everyone, and welcome to our Q4 and full year 2025 earning call. I'll be joined on this call today by Liven March, our new chief commercial officer, Anurag Rallon, our chief medical officer, and Kenneth Leonard, our Chief Financial Officer. On this call, we will be making forward-looking statements that are based upon our current insights and plans. As you know, these may differ from future results. As you saw in our press release, farming ended 2025 on a strong note, operationally and financially. Total revenues grew by 15% in the fourth quarter of 2025 and by 27% for the full year. Thanks to disciplined cost management, we delivered $26 million of operating profits in 2025 compared to a loss in 2024. We also significantly increased our cash position. Operating cash flow came in at $55 million in 2025, putting our cash position at year-end above that of the end of 2024 level, before the acquisition of Abreva. 2025 was marked by significant growth of our two commercial assets, Ruconest and Joenja. Ruconest grew 26% year-on-year and by 9% in the fourth quarter, with its efficacy with With its reliability and its rapid onset of action, Reconest is poised to remain an established on-demand treatment option for difficult-to-treat patients in an evolving HA treatment landscape. Liebern will talk more about Reconest's performance and its unique value proposition. Joinger grew 29% year-on-year and by 53% in the fourth quarter. fueled by the acceleration of new patients on drug in the U.S., but also increased demand in international markets, including in the U.K., where the drug was launched last spring, and in other countries through purchases under government-supported access programs. These results underscore farming's transformation from a single-asset company into a highly profitable high-growth diet tech with two commercial products and a late-stage pipeline with two programs offering billion-dollar self-potential. We highlighted these programs at our Invest Today last month, offering investors unique insights to our high-value pipeline. RecurNest is the foundation of our portfolio and a reliable cash engine for the future. even in a more crowded HAE on-demand market, given its efficacy profile on the difficult-to-treat patient subpopulation. Coenzyme is just at the beginning of its cycle, with multiple growth catalysts in APDS, international geographic extension, and potential expansion into much larger PIDs. Lefazimone, which we used to call KL1333, for primary mitochondrial disease is another billion-dollar-plus opportunity, with the registrational study now well underway. This combination of durable revenue, first in disease innovation, and an advancing late-stage pipeline positions farming well for substantial near-term and long-term value creation. Let me now turn to our outlook for the remainder of 2026. As announced at our investor day in February, we expect 2026 revenue between $405 and $425 million this year, representing an 8% to 13% growth with operating expenses increasing at a slower overall pace, even with substantially higher R&D investment in our pipeline fuel future growth. We expect continued recognized growth for the reason mentioned earlier and accelerating joint chart growth, fueled by the uptake in APDS patients above 12 years in the U.S. and potential future regulatory approval internationally. Regarding the U.S. pediatric label extension, we've been working to address the FDA request and we expect to have greater clarity on the resubmission requirements and timeline following the FDA Type A meeting, which is now scheduled at the end of March. 2026 is an important year for our pipeline, with the materialization of potential value inflection points. We have now completed the enrollment of two lanylizib Phase II trials for larger prevalence PIDs, and we expect a top-line data readout in the second half of this year. We also expect to complete enrollment in the Napa Zimone Pivotal Study this year, to be in a position for a data readout at the end of 2027. We are clearly determined to maintain strong financial discipline to optimize capital allocations on our growth drivers. This is critical as we strive to build an efficient and scalable organization and make farming a leading rare, ultra-rare disease company and deliver sustainable value for our shareholders. Let me now turn to Liv and March for deeper insight on the performance of our commercial portfolio.

Good day, everybody. Let me start with Rucanest, where the U.S. business delivered another year of strong and resilient performance. In 2025, despite the first new wave of treatment options in the HAE market in almost five years, Rucanest continued to grow as an essential therapy for patients living with severe high-frequency HAE attacks. Our strategy remains consistent. focus on high-attack patients who require fast, reliable, on-demand treatment, and ensure that we continue to execute against that differentiated value proposition even as new agents enter the market. For the full year, Rucanest delivered 26% global revenue growth and a volume growth in the U.S. of 20%, a clear indicator of the strong and durable demand for Rucanest in the acute segment. In quarter four, we delivered 9% global revenue growth versus the prior year quarter, and a 10% volume growth in the core U.S. market, continuing the upward trajectory of Rukinest. As expected, through Q4, we began to observe impact of newly launched therapies for HAE in the U.S. market, with some patients trialing and some already returning to Rukinest. Despite these competitive dynamics, we welcomed over 60 new enrollments in the U.S. in the fourth quarter, slightly above Q3. Rukinus added both new patients and new prescribers in the quarter, underscoring the resilience of our position in the difficult-to-treat patient segments and the clinical trust placed in Rukinus in real-world settings. That said... The high burden of HAE matters for patients who have high-frequency attacks. Attacks are often unpredictable and potentially life-threatening, and symptom severity often escalates within hours. Importantly for patients experiencing multiple attacks per month or patients who experience suboptimal responses to other options, dependable rapid relief is not optional. It is essential. And this high-frequency attack segment is precisely where we have seen consistent, durable use of Rukaness over time and where we expect Rukaness to remain highly relevant even as new treatments enter the market. To that end, a meaningful proportion of new patient enrollments in the U.S. are switches to Rukaness from other on-demand therapies, further emphasizing the continued need that high-attack patients have for an effective, reliable, one and done therapy like Ruchinest. And so when you put this together, the unpredictability of the disease, the high burden carried by certain patient segments, the limitations of some other acute therapies, and consistently strong clinical performance associated with Ruchinest, the unique value proposition for Ruchinest remains clear. Looking ahead, we foresee some pressure on our growth early in the year, but with no change in the need for an effective, rapid onset, reliable one-dose treatment like Ruchinest. With that in mind, let me turn to Joenja. For Joenja, we delivered a strong fourth quarter, building on the momentum we established throughout the year. Revenue grew 53% compared to the fourth quarter in 2024, reaching 19.8 million globally. for the full year to end regenerative $58 million, representing 29% growth year on year, and demonstrating both sustained utilization from patients and the expanding clinical recognition and treatment of APDS. In the United States, patient growth remained a central driver of performance. By the end of 2025, we had 120 patients on paid therapy representing a 25% increase over year-end 2024. This steady expansion of our treated population reflects strong physician confidence, consistent engagement with patient communities, and a team that executes with discipline and with urgency. Equally important, we made significant progress in broadening the pool of identified APDS patients, one of the most critical leading indicators in an ultra-rare disease. In 2025, the number of U.S. patients we identified diagnosed with APDS increased by 40, more than double the increase of 18 we saw in 2024. This growth in identified patients with APDS shows that our educational efforts, our diagnostic partnerships, and our medical engagement in the U.S. are working. Outside the U.S., We saw strengthening demand across international markets, including a solid first-year uptake in the United Kingdom, following the launch in April 2025. We also benefited from government-supported access programs, which allowed us to reach more patients who currently have limited therapeutic options. Taken together, these results give us a strong platform for Joenja in the years ahead. Finally, we expect geographic expansion and the anticipated four to 11-year approval in the United States to be meaningful contributors to growth. These two catalysts remain materially important to increase the number of patients who can benefit from Joendra and expand the global footprint of the APDS business. Internationally, we've already demonstrated our ability to execute. In the U.K., where Joanger launched in April 2025, we have seen solid early uptake and engagement of community. That success reinforces that our teams have the capability, the infrastructure, and the strategic focus needed to deliver in new markets. In the United States, our commercial teams are fully prepared to launch Joanger for children ages 4 to 11, pending FDA approval, And importantly, we already have 52 eligible patients identified, one-third of them currently on therapy through our Early Access Program, ready to transition at approval. This will give us a running start and positions us for early momentum once the label is approved. Beyond the U.S., our international organization is deeply engaged in progressing regulatory submissions and ensuring that reimbursement discussions can start when approvals are granted across Europe, Japan, and Canada. Across these three regions, we have over 80 patients already receiving Joenza through early access mechanisms, awaiting full regulatory approval and commercial availability. This represents a significant bulk in foundation for launch acceleration once those approvals are secured. And so as we're stepping into 2026, we do so with confidence. For Joenja, we have the right growth catalysts in front of us, and we have an organization that has demonstrated that it can execute launches with precision and continuity. This gives us confidence not just in the next quarter, but in the sustained global expansion of Joenja over the coming years. And with that, I'll now hand over to Dr. Anna Raverellen, our Chief Medical Officer, who will walk you through our progress across the pipeline and the upcoming development and regulatory milestones.