Pharming Group N.V. Q4 FY2025 earnings call

• CEO Fabrice Chiraki introduced the call, noting 2025 ended strong operationally and financially with revenue growth, operating profit, and increased cash position. • Discussed growth of commercial assets Ruconest and Joenja, Ruconest's position in HAE treatment and Joenja's growth in U.S. and international markets. • Outlined 2026 revenue outlook of $405 - $425 million, expected operating expense growth, and pipeline milestones including trial enrollments and data readouts. • CFO Kenneth Leonard discussed 2025 financial results, Q4 2025 revenue drivers, full - year results, and 2026 outlook including revenue expectations, operating expense guidance, and G&A cost reduction impact. • CMO Dr. Anna Raverellen talked about pipeline progress, including phase two trials for PIDs, APDS regulatory milestones, and work on VUS and napasomone development.

Total revenues grew by 15% in the fourth quarter of 2025 and by 27% for the full year. Ruconest grew 26% year - on - year and by 9% in the fourth quarter, contributing to revenue. Joenja grew 29% year - on - year and by 53% in the fourth quarter. Ruconest's global revenue grew 26% for the full year with 20% volume growth in the U.S., and Joenja had full - year revenue of $58 million with 29% growth, with U.S. patient growth and international market demand contributing. In Q4 2025, Ruconest had 9% global revenue growth and 10% volume growth in the core U.S. market, while Joenja had 53% revenue growth reaching $19.8 million globally.

• Expect 2026 revenue between $405 and $425 million, representing 8% to 13% growth. • For Ruconest, Q1 2026 inventory drawdowns expected to impact U.S. revenue growth by 7% - 9% year - on - year. • Joenja growth expected to accelerate with annual growth approximately 10 percentage points higher than 2025. • Operating expenses expected to range from $330 to $335 million including over $60 million incremental R&D investments, with $9 million favorable impact from 20% G&A headcount reduction. • U.S. pediatric APDS indication approval timeline awaited after FDA Type A meeting, with U.S. pediatric revenues excluded from 2026 guidance.

Q: Juvenger growth this year, how much of that are you expecting to come from the U.S. market versus international markets? And then secondly, looking at the U.S. market, of those 61 eligible patients that are not on treatment in the U.S., How many of these do you think could be converted to paid therapy versus how many have been considered and ruled out? And then finally, in terms of root and S dynamics, you mentioned that you have heard of patients coming back to treatment having tried the orals. Do you have any, I guess it would be anecdotal, commentary on how quickly the patients return having tried the orals?

A: Very good. Thank you so much, Lucy, for those questions. I'll turn to Lieven in a minute, actually. Let me start with the last one on Reconest, and then we can take your two questions on Joe and Ja. It is true that we've seen some patients trying and coming back. As you know, and as Lieven reinforced, HA is a very significant severe disease, and patients got to be controlled, and especially patients who were used to highly reliable treatments. And so when those patients try another treatment and see that they're their crisis is not properly controlled, they tend to come back to their previous medication fairly quickly, because not controlling a crisis could be life - threatening. Now, when it comes to joint gas growth, as we said, we see the growth this year, in 2026, being fueled both by the continued growth in the U.S., as well as growth increasing in international markets. Obviously, the growth in international markets will come from the U.K., where the drug has been launched, new launch countries, and this will happen in a staged fashion, since once we receive marketing approval, will have to negotiate a price and reimbursement. So ultimately, a bigger portion of the growth will come from international markets, but that's going to be gradual. When it comes to more specificities on Joe and Jas' patient funnel, I'll ask Livan to comment. Thank you. Thank you, Lucie, for your questions. how we would think about the patients considered for Joindra or eligible for Joindra in the U.S. and the pull - through to patients on therapy, that there would be a lag there as patients are going through the patient journey, a fairly complicated patient journey, seeing multiple physicians, the enrollment and then the reimbursement process before we get to patients on paid therapy. And so how I would think about the delta there and the opportunity, there's still a substantial proportion of patients both eligible and potentially reimbursable that would drive growth for us in the U.S. in 2026. Maybe Lucy, this is Kenneth speaking. Maybe I can just add, out of the expected 200 growth in 2026, it will probably be around about 70%, 75% that will come from the U.S. So 75% from the U.S.? Of the growth in 2026 for Drenja, yes.

Q: Hey, everybody. Good morning. Thanks for taking the questions. I know might not be able to give color here ahead of the type A, but I'm going to ask anyway. Obviously, the reasons for your discussions, as Anurag said, were the clinical pharmacology and the analytical batches. Is there anything that you would consider sort of the lead rate - limiting step here? That's question number one. Question number two, regarding Rucanest, you touched upon this a little bit, but I guess You know, the Rukaness case can be split into two components, in my belief. So first, you have the medical component, which continues to make the case. And I'd like to touch upon the investor component and specifically your comments that, you know, patients are still seeing switches from the new therapies to Rukaness. So I was hoping you can provide some additional color as to why those switches are taking place.

A: Thank you, Joe, for these questions. So on the Taipei meeting, it's very difficult to speculate. I think Anurag has been clear on the question that were raised by FDA, and we are really looking forward to engage with the agency later this month to address their feedback and discuss a path forward. too soon to tell. Clearly, given what they've raised, we feel actually that these questions are addressable. And once again, we look forward to engaging with them. When it comes to RUCONEST, I'll let Leverne answer your question. So thanks for your question, an important one. So as we've seen new agents entering the HAE market in the U.S. between June and August last year, as expected, we saw some trialing of both acute agents and new prophylactic agents in the U.S. market. What we're observing in our data currently, and it's still early, is within about three to six months, some of these patterns may start to shift. And we've seen some return of Rukines patients that have originally adopted or trialed a different product coming back to Rukines. But again, early days, and we're monitoring this closely and we continue to execute competitively based on Rukines' very different value proposition for patients specifically in the high attack segment where they are increasingly concerned with reliability and a fast on - demand treatment like Ruconest.

Q: Good morning, guys, and congrats on the great year 2025. A couple of questions from us. You spoke a little bit to the cadence of moving patients from early access onto paid therapy. Any notable variations between Europe, Japan, Canada as you look to that? And then, as you look at the other primary immune deficiencies for Joengia and the Phase II readouts that you're anticipating, can you speak a little bit about next steps, what types of additional trials might be needed, and how to think about the path forward there?

A: Thank you, Jeff, for these questions. access to to pet therapies for for for joan ja in international markets most of these markets are access centrally driven access so the dynamic is different compared to the u.s. where you send each patient you know needs to deal with different players so So in a centralized access system, things are slower at the beginning because you need to negotiate, obviously, with the authorities for reimbursement. When you get reimbursement, then afterwards the reimbursement process is extremely efficient. So we don't expect to see the same type of dynamic internationally. When it comes to higher prevalent PMDs, I'll ask Anne - Marie to elaborate. Hi, Jeff. So on the question about what happens next, so as I mentioned, the studies have completed enrollment. We expect results later this year. The results that we're looking for, again, the endpoints that we're evaluating are clinically relevant endpoints similar to the ones that we looked at in APDS, as well as other clinically relevant endpoints. As we look at those endpoints, we'll plan to have a discussion with FDA and other regulators about the path forward. I think our base case here is that we would expect to do a phase three randomized type of study. However, I think you've also seen from FDA some openness and willingness to look at alternative mechanisms and pathways for patients with rare diseases, especially those where there's a plausible mechanism and there's a mechanism that's understood. So, those are the types of discussions that we would have with FDA, again, once we have the data to be able to plan the path forward.

Q: Yes. Thank you for taking my questions. On Juvengia, you mentioned a different launch dynamic for international markets. So what kind of timelines are you working with for getting these patients in Japan and Canada on paid therapy? And in the UK, will you also start reporting the numbers of patients on paid therapy? So currently, how many patients in the UK are on paid therapy and how many have you identified?

A: So when it comes to timelines, so we've elaborated a bit on our expectations when it comes to regulatory timelines in the very near future, when it comes to CHMP opinion from Europe and PMDA approval in Japan. In Japan, specifically, since you asked the questions, we would be submitting a price very shortly for reimbursement, and it takes about three months, actually, for the price to be granted. So today, we have launch timelines planned for the summer. We will be reporting more information on the international market on Joe and Ja in a pool fashion as soon as we have launches in more than one country. So this should happen very soon. It's absolutely essential, and you can count on transparency here. For the second part of your question, I'll let Lieveon elaborate. Certainly. As Reese has said prior, as we look at different approvals coming online at different times in this year, every country fundamentally will be a country - by - country process as the approval and reimbursement processes are quite unique for the countries that we discussed. So how we think about conversion is conversion will depend on physician experience, diagnostic confirmation and testing, and access. And we are building those enablers systematically with the international teams to make sure that we are able to execute upon approval.

Q: Hi there, thanks for taking my questions. My first one is a follow - up on Treventa and the patients on paid therapy in the US. You added 18 in H1 2025 and 6 in H2. Appreciate that this takes time, but are you expecting for this rate to pick up into 2026? Then the second question is just on costs. You're guiding 6 - 8% growth in OPEX in 2026. And you mentioned some phasing considerations on the revenue side. Are there any particular phasing considerations for the cost through the year, particularly around the higher R&D costs and G&A cost savings? And then just finally on M&A, in the release, you mentioned continued focus on potential acquisitions and in licensing opportunities. So any additional colour on what your thoughts are there would be helpful.

A: Very good. Thank you so much, Natalia, for your question. So I'll start with the last one when it comes to M&A. We have clearly a number of growth catalysts, both in our commercial portfolio and in our pipeline in the years to come. So obviously there is no urgency to do any transaction hastily. to compensate for any sort of weakness. Yet, we clearly aspire to leveraging proven capabilities and a great growth platform to take that to a whole new level and make farming a leading rare disease, ultra - rare disease player. And so we are constantly looking out for opportunities to expand our pipelines, Now it is absolutely essential that these opportunities, if they were to materialize, would be value - attractive. And that's really our commitment to our shareholders. So it's not about actually leveraging any external growth opportunities, but making sure that anything that we would consider would be complementary, would fulfill our mission, and will be quickly accretive from a value perspective. From a cost perspective, I'll let Kenneth elaborate. Yeah, thanks for the question. I think, you know, the way to think about it is in the 2025 baseline, we obviously have also some one - off costs that were related to the transaction off - year pleader, so non - recurring transaction costs of about $10 million. And we also communicated earlier that we had about $4 million in costs related to the G&A reduction program. But then when you're looking into the more than $60 million incremental investments in 2026, we of course see the $9 million of savings in G&A come fully through, And, you know, then we have seen the impact in our planning of the strengthened capital allocation, which has allowed us to keep also marketing and sales costs flat. So there are different dynamics that are playing in, but I think, you know, the future speaks for itself that we're fueling where we're seeing the opportunities to advance, in this case, 2026, the pipeline. and are very diligent around spent discipline across all other areas. And so lastly, coming to your question on joint jar, which is actually a very important question, because as I said, I really see joint jar taking a larger part of our revenues as the drug continues to grow significantly and realize its full potential. You heard that last year we really accelerated the uptake of the drug. We had more new patients on therapy in the U.S. that we had in 24. Also, we've identified more APDS patients in the U.S. in 25 than we did in 24. And so this is really fascinating. So, this year, we expect to continue to accelerate patient enrollment on joint jobs and, as a consequence, accelerate revenue growth.

Q: Yes, hello. Thanks for taking my questions. I've just got two. So I was wondering if you could give us a timeline on the performance and collation of the results from these additional experiments you need to perform with regard to the variance of uncertain significance. And also, are you still confident that 20% of these VUSs will turn out to be APDs? And then just on Rukanest, you were talking about a possible infantry - related decline in or infantry - related effect on sales in Q1. Is this an unusually large infantry drawdown that you're expecting to see in Q1? I mean, that was my impression. And if so, why do you think you saw such a large infantry buildup towards the tail end of last year?

A: Simon, we are now planning these new experiments. These experiments, again, we're working with Columbia. They're actually going to be using new technology. new base editing technology to be able to generate different types of variants, generate more controls as they go through this process. It's too soon to tell in terms of the timelines as well as the actual number of patients that will actually be reclassified. But as this work gets underway over the coming months, we should be able to provide more details around it. Okay, thanks. And, Simon, on the inventory part, the way we're thinking about it is that there's always this quarterly fluctuation of the business given the patient ordering patterns and the, you know, general movements. And we have also historically, if you go back in the previous years, seen that, you know, similar dynamics in the early part of the year. Now, it's a little bit, let's say, higher in terms of inventory drawdown and dynamics this year, and we kind of attribute that simply to some of those market dynamics are kind of settling now, and that inventory levels are just kind of returning to a little bit more of the normalized level. So impact - wise, it's a little bit higher, but the mechanics of how the quarters are fluctuating and the fact that their inventory impact in the first quarter are not new to us.