Hello, everyone, and welcome to the Q1 2025 Call. I'm Fabrice Chouraqui, CEO of Pharming, and I'll be joined on this call today by Stephen Toor, our Chief Commercial Officer, Anurag Relan, our Chief Medical Officer, and Jeroen Wakkerman, our Chief Financial Officer. We'll be making forward-looking statements in this call that are based upon our current insights and plans. As you very well know, this may differ from future results. First of all, let me say that I had a great first three months at Pharming. I mean, the passion and the commitment of all employees to serving rare disease patients is really palpable throughout the company, whether you are at the production facilities, at the headquarter or with the field. And this mindset of going this extra mile is clearly part of the DNA of the company and not just a few words on a culture slide as I've seen it actually too often. So everyone is clearly determined to realize the vision that we've set for the company, which is to make Pharming a leading global rare disease company with a diverse portfolio and presence in large markets that leverages a proven and efficient clinical development, supply chain and commercial infrastructure. Our results in the first quarter of 2025 are a good illustration of the solid growth foundation that we have built to realize this vision. We've had a strong start of the year with our total revenues increasing by 42% in the first quarter and a significant improvement of our bottom line, which supported an upgrade of our full year guidance. So going into a little more detail, RUCONEST grew 49% to $68.6 million driven by a continued increase of new patient enrollment and the sustained expansion of our prescriber base as well as a lower…

Thank you, Fabrice. Good morning, everybody. If you could go to the next two slides, the RUCONEST performance. So as Fabrice said, we delivered a very strong performance in Q1. RUCONEST is up 49% versus prior year. This reflects the strong trend that started in early 2023 as we emerged from the disruption caused by the pandemic. Since then, our sales teams have added new prescribers and new patients on a quarterly basis, which has translated to consistent quarter-on-quarter growth of RUCONEST sales. In addition to the underlying strength of the business, we also saw significantly less inventory buildup, as Fabrice mentioned, at the specialty pharmacies in Q4, so less inventory to wash out in Q1. Sales therefore largely reflect the demand created within the quarter. And I'm also pleased to share that our market access team also improved the number and speed of patient prior authorizations in Q1 enabling our patients to reorder RUCONEST earlier than in previous years. So these two factors combined also strengthened the RUCONEST performance in Q1. The key though to the past and future performance is of course RUCONEST. It always starts with a good product. And in RUCONEST, we have an excellent product serving all patient types, those being Type I, Type II and the normal C1 patient population. All three groups as it relates to RUCONEST have one thing in common. They all suffer from moderate to severe debilitating HAE attacks and they have them frequently. They also typically failed other targeted acute therapies such as actapam or having to re-dose to resolve their HAE attack. The RUCONEST delivers attack resolution for 97% of patients in a single dose is therefore a major factor in patients continuing to choose it to treat their acute attacks. Now as some of you know, I've…

Thanks, Steve. Let's turn now to those growth drivers you were talking about, including beginning with those near-term opportunities that we can see on the next slide. So as you heard from Steve, Joenja can have a real impact on patients' lives. But one of the problems in diagnosing APDS happens when the result of the genetic test shows what's called a variant of uncertain significance or a VUS. In fact, we found more than thirteen hundred patients in the U.S. alone who have received such a result. This happens because the variant is new and there isn't enough information about that variant to know if that variant is actually [Technical Difficulty] variants and screen those variants for PI3K hyperactivity. The results which will be published soon will show that many of these variants actually do lead to hyperactivity of the pathway and can eventually cause these variants to be reclassified as causing APDS. The next step in this process will be progenetic testing labs to review the data from this academic lab and the publication, reclassify these patients and then inform the doctors of any changes to the previous reports that showed these patients had of the VUS. We expect that together all of this will lead to the identification of many new APDS patients later this year as you heard from Steve. On the next slide, we can see some of the other opportunities that we have in terms of bringing leniolisib to more APDS patients in several key markets across the world and to expand the addressable population. To support geographic expansion, we have a number of regulatory applications underway in several key markets across the world on the left. With the EMA, European Medical Agency, we have a single outstanding CMC request and we remain on…

Thank you very much Anurag and good afternoon, good morning, everybody. As you've seen in the previous part of the presentation in the next slide please. Q1 was a very strong quarter for Pharming. The revenues grew by 42% driven by RUCONEST growth of 49% and Joenja by 9% versus Q1 last year. And the key growth drivers were additional demand for both RUCONEST and Joenja from increased numbers of patients and the sustained expansion of our prescriber base. The gross profit is up 50%, which is the result of the additional revenues and a gross margin improvement of 4% to 89%. Operating expenses increased because of the non-recurring Abliva acquisition related expenses of $7.8 million. Excluding those one-off costs, OpEx would be $70.4 million which is well below the Q4 2024 OpEx level and in line with our guidance. Again, adjusting for the Abliva our nonrecurring cost, the operating profit was $0.8 million. Net loss was $14.9 million and that increased loss was primarily due to the non-recurring Abliva acquisition related expenses, most of which were non-tax deductible. And then that results were also impacted by foreign exchange exposure and increased tax cost. The cash and marketable securities decreased since the end of the 2024 by $60.5 million to $108.9 million and that was primarily driven by the purchase of the Abliva shares totaling $66.1 million. We had a positive operating cash flow in the quarter for the third quarter in a row even with the non-recurring Abliva cost included. On the next slide, we see the financial impact of the Abliva acquisition. So we completed the acquisition of Abliva via a public cash offer to the shareholders to acquire all outstanding shares for approximately $66.1 million in March 2025. You see the immediate financial consequences on this slide.…

Thank you, Jeroen. So as you heard, I mean we are off to a strong start of the year, which is fueled by the continued strong growth of RUCONEST and acceleration of new patients on Joenja, the largest increase since Q2 2024. As a consequence, we are raising our full year guidance, and we are also setting the foundation for strong financial discipline with, as a first step, the decision to decrease annual G&A spend by $10 million to optimize tactical allocation and drive sustainable growth. We also continue to invest in our pipeline with the objective to generate two blockbuster assets: The potential new indications for PIDs with immune dysregulation are an opportunity to significantly expand Joenja's addressable patient population beyond APDS. And KL1333 in mitochondrial diseases is another asset with $1 billion plus revenue potential. So as you can see, we are building a solid platform for sustainable growth and value creation with a series of clear catalysts in the short and near term. I personally believe that today more than ever, our goal to develop a leading rare disease company is really enriched. Let me finish this call by thanking sincerely Jeroen Wakkerman, our Chief Financial Officer, for his contribution to the growth of Pharming over the past four years. With this, I'll open the line for questions.

Thank you. [Operator Instructions] Our first question comes from the line of Jeff Jones from Oppenheimer. Your line is open. Please go ahead.

Good afternoon, gentlemen. Thank you for taking the question and congrats on a great quarter. Jeroen, one quick clarification for you on the financials. Can you clarify on the flat OpEx for 2025 pre Abliva I believe that, while reducing G&A by $10 million dollars. Should we be then thinking about the $10 million adjusting into, say, R&D? Or how should we think about that?

So, yes, so as I said, actually, this is really an adjustment that we're making on G&A, general and administrative expenses, to really optimize the capital allocation to drive the growth of our business further. So clearly, the goal is not to touch R&D, not to touch our commercial expenses, which clearly are the engine of the short- and long-term growth.

Got it. Appreciate that. And then one thing that is obviously a topic of a lot of discussion right now is the potential for tariffs with the U.S. And given your European manufacturing and most of your sales in the U.S., can you comment on how you're thinking about this at this point and the opportunities for mitigation?

So obviously, I mean, we don't have the details on the size and on the scope of those potential import tariffs. So it's very difficult to comment without these specifics. What I can say is that we've been working to evaluate a range of scenarios to see really how we can mitigate the risk and the impact of potential tariffs. This could include, I mean, adjustments in our supply chain, in our manufacturing, adjustment in our custom price. I think it's too soon actually to go into more details, but our goal is to be proactive. So we have a set of options, if this actually come becomes a reality.

Appreciate that. I'll jump back into the queue.

Thank you. Our next question comes from the line of Joe Pantginis from H.C. Wainwright. Please go ahead. Your line is open.

Hey, guys. Good morning and good afternoon. Very nice to see the assets and looking forward to the expansion of the pipeline. So, two-part question, if you don't mind. So first, I wanted to get a sense and this is one of the obvious questions with regard to product growth, potential impact on tariffs right now and on your supply chain. And as part of that, you saw nice RUCONEST growth in the first quarter. Just curious how first quarter insurance and Medicare resets might have impacted that? And then the second question is maybe for Stephen. As you're looking to continue to drive new doctor signing-ons for RUCONEST, what are some of the key education, or they need to wait to see more factors before they actually sign on for RUCONEST? Thanks guys.

Steve, would you like to comment actually on the Medicare reset and the growth?

Absolutely. Thank you, Fabrice. Good morning, Joe. Thank you for the two questions. So just on Medicare, clearly, as you're aware, the IRA and out of pocket expense impacts bottomed out this year, which is good for patients. I would say that it certainly didn't hurt us, but it wasn't a major factor in our growth. The growth, as I said, just came from the underlying strength of the brand, but also the trend, but also the focus that we have on those severely affected patients. So, yes, it had some impact, but it wasn't major for us and certainly not for RUCONEST. In terms of what new doctors like to see, I would say in those either centers of excellence or those locations where physicians have a bigger patient base, then we're already very well positioned for patients that are attacking frequently and for whom other medications just aren't working or they happen to re-dose a lot. In some of the other practices where we see growth in prescribers, it's often where they only have one or two patients. So it's really the point at which they get reviewed. So it's not the positioning or the profile of the product. It's more the opportunity for that position to actually use, which I think is one of the factors why aside from the execution stuff I mentioned, you see that consistent growth pre-pandemic and then you see it return again when the office is reopened and we're able to get back out there and remind physicians of exactly where RUCONEST sits within the market. So I think does that answer the question for you, Joe?

Yes, it certainly does, Steve.

Okay.

Thank you. We will take our next question. Our next question comes from the line of Alistair Campbell from RBC Capital Markets. Please go ahead. Your line is open.

Thanks very much, everyone. Thanks for taking my questions today. Just a couple, please. First of all, on Joenja, so a few things happening in Q1. But maybe if you could just help me understand, as we think about price evolution from 2024 to 2025, what do you think the true underlying net price evolution will be in the U.S.? Secondly, on the VUS opportunity, maybe you could sort of talk through a bit more detail about the process of getting those patients transferred on to paid therapy and how quick you think that can come through? Or is that going to be a bit of a slow build? Maybe just touch on that. Then finally for Jeroen, good luck in the future, Jeroen. Maybe one last question before we head off. Off. Given the dynamics on tax, obviously, loss making Q1, but a tax expense in Q1, can you help us understand what we should be expecting for the full year? Thank you.

Thank you, Alister, for this question. So I'll let first Jeroen answer the tax question and then Steve can provide actually a color on your two questions on Joenja.

Yes. Thanks, Alistair for the question. In terms of the tax line in Q1, it was really affected by three key things. One was the, Abliva, related costs that they were non tax deductible. Then we had some share-based payment costs that are also nondeductible, so that increases obviously the effective tax rate. And we had a release of a deferred tax asset from the past. Now those topics are all one offs apart from share-based compensation that will increase a little bit, but most of it is one off. So the high tax cost that we had in Q1 is a one off and we don't expect that to continue in the remainder of the year.

Thank you.

Thank you, Jeroen. Yes, Alastair, so just in terms of you asked about net pricing evolution, we expect roughly 75%, 76% of our pricing actions flow through to the net line this year. And then in terms of approval evolution, I think as you've seen both within the U.S., but also globally in key markets, we continue to build the funnel. Now and we've seen an acceleration as both of the reason I mentioned earlier in the call in patients coming on to paid therapy in Q1 which was really nice to see. And that reflects everything that we've put in place in the support services and the reimbursement services etcetera, to convert patients as quickly as possible. The good news is that the vast majority of patients are being converted relatively quickly once they're enrolled and with very little market access barriers being put in place. One would imagine the severity of disease, the lack of treatment options and the fact this is the only indicated product are big factors there. But as I said in previous calls, it's not linear. You have patients these are very complicated patients with all kinds of comorbidities. We also, as I mentioned, have a lot of pediatric patients who simply don't qualify right now. So it's not linear. There has been consistency though and as Q1 was great to see and we expect to see more and more patients converting through the year and they're very confident in that. Does that help, Alastair?

Yes, certainly it does. Maybe, Stephen, can I quickly come back? You're talking about 75%, 76% flow through to net this year. I mean, how would that broadly compare to what you saw in 2024?

So obviously, gross to nets are quite complicated and they are affected by different things. I think it's probably slightly better in the book of business lens itself this year in terms of utilization of government versus commercial patients. So I think without having a direct comparison for it, it's probably slightly better than last year.

Okay, great. Thank you.

Thank you. Our next question comes from the line of Simon Scholes from First Berlin. Please go ahead.

Yes, good afternoon. Thanks for taking my question. I've just got one. Just on the G&A cost reduction, I mean, given that revenues are going to be presumably going to be continuing to grow quite strongly, Reducing G&A costs by $10 million is not going to be that easy. I was just wondering what elements of G&A you expect to be able to reduce, which elements you're targeting in particular?

Thank you, Simon, for your question. I mean, at this stage, I cannot comment actually on the detail of the plan. And as soon as I can, I'll share more. It is indeed actually an ambitious plan. I believe it is something that we hope to do. I mean, our ability, as I said, to ensure that we get the best return on every Euro, every Dollar we invest is absolutely essential to unlock a single value creation. We have a great growth outlook, and it's both about top line and bottom line if we want to optimize the value creation. So we are we're doing that exercise. We're finalizing that exercise. And when I can share more, I'll share more. But this is a target that we've set, dollars 10,0 million on an annual basis.

Okay. Thanks very much.

Unless there are any further questions I'd like to hand back to the speakers now. Thank you.

Listen, with this, I think we're going to close our call. Thank you so much for your interest in the company. Thank you for your questions. We look forward to updating you on our plans. And probably, I'll see many of you in person through one-on-one meeting or at investor conference in the coming months. Thank you very much.