Pharming Group NV (PHAR) 2024 Q4 法說會逐字稿

Thank you very much. Hello, everyone, and welcome to the Farming Full year and Q4 2024 financial results call. I'm Fabrice Chouraqui, CEO of Pharming , and I'll be joined on this call today by Steven Toor, our Chief Commercial Officer; Anurag Relan, our Chief Medical Officer; and Jeroen Wakkerman, 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 Pharming, and it's an honor to succeed Sijmen de Vries. I'm passionate about progressing medical sciences and bringing innovation to patients. And naturally, I feel deeply connected with Pharming's mission to serve the unserved rare disease patients. Over the past 25 years, I've developed an experience across the business spectrum from pre-clinical research and clinical development to commercial leadership, business development, and capital formation.

Have 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 [dietech]. In light of this experience, I'm impressed with the development of Pharming over the past 15 years and its significant growth prospects.

RUCONEST has become one of the cornerstone on-demand treatments for HAE. Joenja is already approved and launched in the US for the treatment of APDS with the second number of patients already on treatment. And it is due to the 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 Pharming, 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've 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, a 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, RUCONEST will continue to grow and could even benefit from the potential increase of the on-demand segment driven by the entry of new entrants.

Joenja 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 US; the launch in key markets, including the UK 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 Toor, our Chief Commercial Officer, who will give you a more granular perspective on the strong dynamic of RUCONEST and Joenja.

Thank you, Fabrice. Good morning everybody. As Fabrice just alluded to, we've delivered another strong performance in 2024. On RUCONEST, 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 RUCONEST 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 Joenja , we continue to build our patient pipeline and transition eligible patients to paid therapy. And as you would expect, our team delivers significant growth over the first year of launch, ending Q4 65% up on prior year at $13.1 million, but for 2024, 147%-plus of $45 million.

Of note, in addition to 96 patients plus 5 pending on paid therapy in the US, 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 Joenja of business for APDS and with new potential indications for the molecule, create a strong, high growth franchise.

Looking first at RUCONEST, as I just stated, RUCONEST is and will continue to be a growth driver for Pharming 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 C1-INH 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 RUCONEST, since it works comp 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 RUCONEST patients and what this means for them. The first thing to note is that RUCONEST serves all patient types, those being type 1, type 2, and normal C1 patients. All three of these ruinous 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 icatibant or are having to redose to stop their HAE attack.

In the photos on the slide, you can see an actual RUCONEST patient at the start of an attack, and then her recovery as it resolves at the 4-hour mark and the 24-hour mark. For patients like this one suffering with a more severe course of disease, attacking frequently and having 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 attacked free for at least three days is a very big deal.

RUCONEST efficacy and reliability allows our patients to better control and plan their lives, and that's why RUCONEST will continue to have a strong position in the US acute market and remain an important product for our company in the years to come.

I'll transition now to Joenja, which, as you're aware, was launched in the US in March 2023 and is available outside the US through various access programs. We see a number of opportunities for Joenja, which I'll walk you through now. Our Pharming's patient finding efforts are continuing as we build our patient pipeline in the US and globally. In fact, we've already identified over 240 patients in the US 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 address 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 would deliver another bonus of APDS patients available for treatment this year and beyond.

The second will be the pediatric indication launched in the US which is expected in 2026. We currently have over 60 patients in our US pipeline and growing, and they will begin transitioning to Joenja as soon as the indication is approved. And the third Is our geographic expansion program, which is the 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 Joenja 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 Joenja 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, leniolisib APDS pediatric is only part of the story.

As Phase 2 trials have been initiated for two bigger indications, open and CVID. In fact, CVID, while still rare, with the prevalence of 40 patients per million, transitions leniolisib from a small ultra rare disease molecule to one with blockbuster sales potential, thereby creating the 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, Anurag Relan, whose team are, of course, critical to driving these programs forward and realizing these opportunities to provide us with a research and development update.

Thanks, Steve. And here we can see our growing pipeline. For someone who's been at Pharming for many years, it is incredibly impressive to see how this has expanded from only RUCONEST for HAE to now include multiple products and indication which can support the vision you heard Fabrice's layout that we have for Pharming.

Since APDS is a primary immune deficiency with immune dysregulation, and there are other more prevalent PIDs with similar features, we're especially excited for the work that we have started here with two Phase 2 studies underway, including a new program in CVID or common variable immune deficiency. And last but not least, we have on this slide KL1333 in a pivotal study with the recent acquisition of Abliva.

Before turning to development, as the VUS project has been a focus of our patient finding work. A significant challenge in diagnosing APDS patients occurs when a patient's genetic test result shows a VUS or a variant of unknown significance. This happens because a variant is novel and there isn't enough information to determine if the variant is disease-causing.

In fact, there are over 1,200 patients in the US alone who have received the VUS results in the genes associated with APDS. Over the past year, we've been supporting a project to gain additional insights into these VUS. The recently concluded study, which will be published soon, has shown there are many new variants that lead to hyperactivity in this pathway.

The next step in the process is for genetic testing labs to review these data and determine which variants can now be reclassified as causing APDS. We expect by mid-year these efforts will lead to the identification of many new APDS patients.

Now, let's turn to the work going on in pediatrics, where we have an active clinical program with recent data to support regulatory filings. APDS symptoms, as you know, begin at a young age, and more than 25% of the patients we've found are below the age of 12. Since the disease is progressive, it is important to be able to treat the condition earlier in its course.

In December, we were excited to report the topline result from the first clinical study in children with APDS ages 4 to 11. The study demonstrated that leniolisib was generally safe and well tolerated, and we saw benefits across the two co-primary endpoints, which were consistent with what we have observed in older APDS patients. These data will be presented at a conference in May, and in the second half of this year, we will begin regulatory filing starting with FDA to expand the label to be able to treat younger patients with APDS.

In addition to our work in APDS, we are developing leniolisib for primary immune deficiencies with immune dysregulation, which you see in the diagram are a subset of all PIDs. APDS, in fact, is one such example of a PID with immune dysregulation, and we have started two more programs in this area.

The first program is a genetically defined group of PIDs, which we started a study in October and we are continuing to enroll patients. And the FDA recently granted fast track designation for the program.

We are announcing today the second program in CVID which is a clinically defined group and represents an even larger group of PID patients. The study is now open for enrollment, and we expect the first patient to be dosed later this month.

The patients in both of these studies lack effective therapies to manage the immune dysregulation that leads to disease progression and early mortality. And you can see in the prevalence estimates how the new study we announced today in CVID significantly increases the patient population that could potentially benefit from leniolisib.

Across APDS and these new programs, the central role of PI3Kδ in lymphocytes is clear in driving the immune dysregulation, which supports the investigation of leniolisib. I look forward to updating you further as we progress with these exciting new programs.

To recap, we have a number of regulatory and clinical activities to bring leniolisib into more APDS patients in several key markets across the world and expand the addressable population.

As you heard from Steve, bringing leniolisib to additional APDS patients represents a significant near term opportunity. And in Europe, we have already concluded a the clinical benefit and safety of leniolisib, where we have a single CMC issue remaining as part of our review with EMA and we expect to be able to address this in January of 2026.

We also have marketing authorization in the UK and you heard from Steve already. NICE published their final draft guidance today, which will eventually enable reimbursement in the UK.

In Japan, we have completed an interim analysis of a small trial there, and this will now enable our filing with PNDA in the middle of this year. And you heard me already talk about the pediatric study in the 4 to 11 age group, but we also have an additional trial in children as young as one year of age, which is nearing completion of enrollment.

In all, there are quite a number of projects to be able to expand the addressable population, including these new TID indications, which support the long-term growth of leniolisib. And now turning to our third program in our portfolio via the just completed Abliva acquisition TL133. This is being developed for primary mitochondrial diseases, which are a group of rare disorders or mutations in mitochondrial DNA lead to impaired energy production.

These disorders can have a array of diverse clinical features, but a common element is the severe fatigue and muscle weakness seen in these patients, which of course leads to a poor quality of life, given the degree of symptoms. There are a large number of these patients already diagnosed across the US and large European countries where they're treated at centers of excellence and part of a strong advocacy group.

KL133 addresses the underlying disorder by normalizing the NAD+ to NADH ratio, which is abnormally low in these patients. There is a pivotal study underway with endpoints agreed upon with FDA and there was also blinded interim analysis in which both endpoints passed futility.

Having just completed the acquisition, we found to begin enrollment in the second wave of the study as soon as possible with sites that are already open.

As with the rest of our portfolio, we see this program as one where we can use our rare disease expertise and infrastructure to bring much-needed products to patients where there is significant unmet medical needs. Now turn it over to Jeroen to review our financial performance and outlook.

Thank you very much, Anurag. Q4 was a very robust quarter for Pharming. Revenues grew by 14%, 1-4% versus a very strong fourth quarter in 2023.

RUCONEST growth was 9% and Joenja 66%. Gross profit grew by 9% and that is lower than the revenue growth and that was driven by a one-off inventory impairment following a reduction -- RUCONEST production issue at one of our CMOs.

The OpEx was fairly stable and largely as expected in the quarter. The small increase was caused by a one-off cost, including a full impairment of the lease contract and just over $1 million of Abliva acquisition costs.

Operating profit increased by $5.6 million driven by higher gross profit as a consequence of strong sales and active OpEx management. This was the second quarter in a row that we generated operating profit. And the net results went from a loss in Q4 '23 to a net profit in Q4 2024.

We had a positive operating cash flow in the quarter and in fact for the second quarter in a row. The cash and marketable securities reduced slightly due to interest costs and currency effects.

And looking at the full year results. Full-year results in 2024 were good. Revenue was at 21%, which was driven by both RUCONEST with a strong growth of 11% and leniolisib that grew by 147%. OpEx increased by 10% due to investments in Joenja, which is at a rate well below the revenue increase.

Operating profit on a like-for-like basis improved and that's when taking out the big one-offs that we had in 2023. Our cash position reduced and that was driven by the refinancing of our convertible bonds earlier in 2024 for a lower amount than the previous bonds.

A quick update on the Abliva acquisition process and timeline. The $66.1 million acquisition of Abliva is now completed and Abliva shares approved for delisting next week. We initiated the compulsory acquisition procedure for the remaining Abliva shares, and the acquisition was -- the acquisition of the shares was funded with available cash. And the transaction really illustrates our strategy of developing a high-value pipeline.

Moving to the financial guidance for this year 2025. We expect the total revenues to land between USD315 and USD$335 million, which means a growth rate of 6% to 13%. And the assumption underlying the guide as mid points is a high single digit RUCONEST growth.

And continued strong growth of Joenja with an acceleration in the second half of the year from the positive impact from VUS, as Anurag mentioned before. For Joenja, we expect continued growth of patients on paid therapy and the US pricing at an annual whack of USD594,000.

Regarding operating expenses, we expect them to be flat on the previous year on 2024, prior to the impact of Abliva. We have not yet integrated I believe, and that process should start next week, and our preliminary estimate for Abliva related OpEx is $30 million for 2025, including $17 million R&D costs and the remainder consists of non-recurring transaction and integration costs.

And we will provide an update of these costs in our Q1 call in May. With that, I would like to hand over back to Fabrice.

Thank you, Jeroen. As you've heard, our inline portfolio has generated strong growth in 2024, and we are exiting Q4 with a solid momentum. With its unique profile and strong patient experience, RUCONEST is well positioned to remain one of the treatment of choice for HAE attacks.

In APDS after an initial strong Joenja uptake, we are now working to identify and unroll new patients before capturing two well-defined opportunities with the VUS and the expected pediatric indication. We continue to invest in our long-term growth with the objective to generate two blockbuster assets.

First, the potential new indication of PID with immune deregulation. It's a great opportunity to continue to expand Joenja just sales potential. Second, the acquisition of Abliva's pivotal stage program in mitochondrial disease brings another asset with significant revenue potential.

Our 2025 revenue guidance of $315 million to $335 million illustrates our momentum, and obviously, we look forward to updating you on our progress. Let me now open the line for questions.

(Operator Instructions) Jeff Jones, Oppenheimer.

Good morning guys, or afternoon I guess. And Fabrice, welcome to the team, and congratulations on a fantastic first earnings update here. I guess two questions for us. First with respect to KL133. In the target of primary mitochondrial disease, could you speak a little bit to how this patient population breaks down and what portion of that population you think would be treatable and/or eligible for treatment based on the likely label? There are a lot of sort of mutations within that group.

And then, just to clarification for a second question. For the 188 patients you mentioned that are on the expanded access program, which includes clinical trials, are any of those patients paid? And perhaps could you, provide some breakdown in terms of the territories where those patients are? Thanks.

Thank you, Jeff. Let us elaborate actually on your question. So I'll hand over to Anurag first, to tell you more about, the addressable population with the ongoing trial on KL1333.

Hi, Jeff. So, with respect to primary mitochondrial diseases, when we talk about the 30,000 number of patients that are in the US and the large European markets, this actually is already -- we've already limited it to the group of patients that have the mutations that are going to be enrolled in this study.

So specifically the mitochondrial DNA mutations, which already represent 80% of all PMDs, and then we broke that down further and looked at the mutations that are specifically being enrolled in this study, so that's how we came up with this estimate of 30,000. So in essence, all of those 30,000 are the addressable population.

Appreciate that. Thank you.

And then I'll turn it to Steve now to answer your question about the access program.

Hi, Jeff. Good morning. So of the 188, we actually have those patients in multiple countries around the world, including, many of the key markets that I listed earlier.

Those patients are predominantly in either in the early access program or compassionate use, and also in clinical trials. And we do have a number on paid therapy through name patient programs. But as you can appreciate. That's not a specific number or revenue line that that we would discuss publicly.

Appreciate that, guys. Thank you.

Ben Jackson, Jeffreys.

Great, thank you for the question. Just two for me, if we just start on. One that's not too exciting. So the $30 million anticipated additional OpEx from the Abliva acquisition, are you able just to touch on how much of that is recurring? Apologies if you've noticed that already in the call.

And then the second one, just interestingly, do you see any theoretical exposure to potential US tariffs if they were to apply to drugs? I get at this point that it's very unclear what's going to happen. And we don't necessarily know where it's going.

And then I guess as a result of that, if you noticed any kind of level of changing inventories or stocking or patient interest in a measure to hedge against a potential short-lived trade war or anything like that, your thoughts or color around that would be great. Thank you.

Thank you, Ben. So, let me take your first question about the $30 million of [OpEx] spent on Aliva in 2025 as announced by Jeroen. About $17 million will be R&D, and the rest will be non-recurring transaction and integration costs.

Now when it comes to the tariffs, obviously we are monitoring the situation and we're doing some homework in the background, looking how we can minimize the impact of potential tariffs if they were decided by the US administration.

We're looking at some adaptation that we could make to our supply chain. This is obviously ongoing. I cannot share any details, and we can tell you more in due time. But this is obviously something on which we are proactive, and I am monitoring very closely.

Perfect. Thank you.

I didn't think there was a question on whether there's any stocking or inventory buildup as in anticipation of the tariffs.

No, we have enough stock already for our needs within the US. And there's no planned inventory buildups at this point in time until we have a clearer picture of the situation.

Very clear. Thank you very much.

Alistair Campbell, RBC.

Hi there, thanks very much for taking the questions today. And as I say, let me start with RUCONEST. It's great to see a level of confidence that this product will continue to grow. And just very briefly in the Q4 number just for modeling purposes, were there any stocking effects in there or is that largely driven by underlying demand?

And then thinking about the outlook for 2025, I mean, clearly you feel very confident that you will continue to grow despite new competition. Is that sort of based on your internal thinking or just intrigued if you've done any sort of market research to underpin that in terms of trying to assess what physicians think of the new product as it comes to market?

And then finally, maybe turning to CVID just a sense of the time frame for those trials. Should Phase 2 trials be sufficient for approval? And how you think those trials will likely be scoped in terms of size and duration. Thank you.

So I'll -- thank you so much for your question. Alistair., I'll start answering some of them, and over to Steve and Anurag for the last one on CVID.

I want to, reinforce that actually there's not been, much talking actually in Q4 and the result, the very strong result that that you saw actually on RUCONEST where enlarged -- by large actually driven by strong demands. So that should be very clear.

When it comes to the positioning of RUCONEST, again, as we've said, I mean, because of the unique profile of the drug and the very strong patient experience that we've been able to generate year after year since the launch of the drug more than 10 years ago, we feel confident that RUCONEST will remain a treatment of choice for HAE attacks despite new oral entrance.

As we've seen in other categories, there will probably actually be -- the new orals will be help to develop the market. They'll be able to position themselves for specific categories of patients based on our experience with Recones and on the feed the feedback we are gathering from doctors and more general marketing sights. This is our strong belief. And I'll ask Steve, obviously, to comment given his experience with the drug.

Certainly, the only thing I would add to that, Alistair, and you asked about market research is we've obviously pressure tested that through a number of advisory boards and steering committees over the past year, and the resounding conclusion of those interactions is exactly what Fabrice said, which is, this is a relatively unique drug in terms of its mode of action.

The patients we serve are severe and they attack frequently. And they have a positive experience. And for those reasons and as I say, validated externally, we believe that RUCONEST has a place both in the short term and the long term and will continue to be a growth driver for Pharming.

Thank you

And then, Alistair, on the question about the CVID studies, we'll have some more details after we dose the first patient. But this will be a Phase 2 study, of course. And we'll talk a little bit more about what those results and the timing of those results would look like once we begin the program formally. And we'll also then be able to talk to you about what the development path might look like.

Joe Pantginis, HC Wainwright.

Hey guys, Joe Pantginis from HC Wainwright. Thanks for taking the questions. Fabrice, obviously good luck at the helm here. Very successful company, so I think you're taking over at a great time as well. So first question is with regard to RUCONEST and building the new prescriber base in the US, how would you sort of describe the yes versus no dynamic as you're trying to convince physicians to be new prescribers to support the core growth of the asset?

All right. Thank you, Joe for your kind words. When we come -- when it comes to to RUCONEST, I've actually, over the past few days, actually met a few RUCONEST prescribers. And so I got a firsthand experience of what's happening in Delhi medical practice.

I think we see a grow -- an increased number of prescribers, with RUCONEST as they can see that the drug has a unique profile. And as a consequence, it has a unique valuable position for a specific segment of the patient, those difficult to treat patients who are experiencing actually a number of breakthrough attacks.

And those doctors, I think, are reinforced by their experience of of the drug. I mean some of them have spoke to me about a drug which is transformative. I'm using again the word transformative for the life of their patients.

And so, I think that's very meaningful given my experience, when you hear a clinician who is actually treated patients with such a devastating disease. You've seen, I mean, the picture actually of a patient, suffering from an attack, that means a lot. Steve, you want to elaborate a bit?

Sure, thank you, Fabrice. Hey, Joe. I think to answer your question of the yes versus no dynamic, we're 10 years post launch and obviously this is a well developed market and even in year 10 we were able to grow the prescriber base by 11%.

And the reason for that is, if you get outside of the centers of excellence, where they have large numbers of patients and experience, many physicians haven't had to consider RUCONEST because they've simply not had a patient or they've had their first one. So what we find, especially with those physicians is they're very open to the concept of ruins because they're now having to treat these severe, frequently attacking patients that they haven't before.

So, as I said, we have a strong base of prescribers, but as the market expands over time and as we go deeper into them, we are very confident that more and more patients -- physicians will need to prescribe. And we'll use and be open to it.

That's really helpful, Steve. Thanks for that. And then I guess for brief -- I certainly acknowledge this is a very early time to ask this question. Anything you could share with regard to changes you might or might not envision for the company's growth, example, is there any further right sizing of the sales force, the impact of new assets or even further inlicensing of assets to look forward to?

It's very early actually to tell you about about what will really be my vision and the roadmap for the company. I believe again given what you've heard today about the momentum, that's clearly -- we will continue to move forward. I mean, I've tried to share a bit about my vision for the company, which is actually very similar to what you heard from Sijmen in the past.

I think our success will come from great ambition, a relentless focus on execution, rigorous P&L management, and OpEx management specifically. And then the continued expansion of our pipeline looking how we can expand our pipeline with the current assets. And you've seen that there are a number of great opportunities with our current assets, but also continue to look at value accurative deals that could actually drive shareholder value, in the mid and long run. I'd be very happy to tell you more in the coming weeks and months as I'm becoming more and more knowledgeable with the intricacies.

Of course. I had to ask. And thanks for that, and thanks for all the color guide.

(Operator Instructions) (inaudible) Simon Scholes, First Berlin.

Yes, good afternoon. Thanks for taking my questions. I've got two. Just to follow up on the Abliva costs, I was wondering if you could give us an indication of how those costs might evolve during '26 and '27.

And then on CVID. And my understanding is that on PI3Kδ, you will require a Phase 3. I was just wondering if you could give us an indication of why you might not require a Phase 3 on CVID.

All right. So let me, quickly answer your question about, the spend on the Abliva program. So as I said, as you heard it from Jeroen, about $30 million this year, 17 R&D, the rest non-recurring transaction and integration cost, when we announced the deal at the end of last year, we've estimated the total cost of the the program to be around $125 million. So you do the deduction, with the $30 million that we're still in that deal obviously, we'll refine this as we complete the integration, resume the trial, but today we don't have any data that tells us that actually this will be any different. Now when you come to the CVID, I'll let Anurag actually clarify, perhaps some misunderstanding.

Yeah. Hi, Simon. So we do anticipate, and again this is early days and not having even those the first patient yet, but we do anticipate that there would be a need for a Phase 3 study as we've done with APDS. We're not anticipating -- these are rare diseases, so these are still relatively small programs. The Phase 2 program. In the first PID with immune regulation is 12 patients. The CVID indication phase two program will be slightly larger, but, we still anticipate a Phase 3 requirement to enable registration. Of course, as the Phase 2 read out, we'll be able to tell you more what those Phase 3s look like. But that's our current plan.

Okay, and just one last one. I mean, do you also expect CVID to get a FDA fast track designation at some stage?

We will certainly look at all of those types of options. These are severe diseases. They're -- they have a similar course as APDS. There's early mortality associated with them. There's -- these are sick patients who have these conditions, so we do anticipate being able to work with the regulators to try to expedite the development.

Okay, thanks very much. That's very helpful.

Thank you. There are currently no further questions. I will hand the call back to Fabrice for closing remarks.

Thank you very much, operator. Thank you very much to those of you who attended the call and those of you who were on the webcast. As I said, I'm very excited to be joining Pharming. I believe that we are exiting 2024 with a very solid momentum, that they are very clearly identified, growth opportunities ahead of us.

And as for my answer to the question, our success will come from our ability to realize them, manage our P&L rigorously, and maintain a very high level of ambition, given clearly the growth prospect that we can have with our inline brands with Abliva. And also I think the capabilities and the unique infrastructure that we have created that can really position the company as a leading rare disease company in the future. Thank you very much.