Q4 2024 Pharming Group NV Earnings Call

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Joseph Pantginis,

Operator: Good day, thank you for standing by. Welcome to the Pharming Group Q4, 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. 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 Chouraqui, CEO. Please go ahead, sir.

Fabrice Chouraqui: Thank you very much. Hello, everyone, welcome to the Pharming 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 Stephen 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.

I'm passionate about progressing medical sciences, and bringing innovation to patients and naturally I feel deeply connected with farming mission.

The unserved rare disease patients.

Fabrice Chouraqui: Over the past 25 years, I've developed an experience across the business spectrum, from preclinical research and clinical development to commercial leadership, business development, and capital formation. Having 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 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 a significant number of patients already on treatment, and it is due to be launched in other key markets around the world. Last but not least, the recent completion of the acquisition of Abliva is another stepping stone in the development of the company.

Over the past 25 years, I've developed and experience across the business spectrum.

From preclinical research and clinical development to commercial leadership business development and capital formation.

I've been able to experience the best of the two wells, the rigor and sophistication of Big pharma.

And the value creation mindset and agility of venture capital Inditex.

In light of this experience.

I'm impressed with the development of farming over the past 15 years and it has significant growth prospects.

Ruger nice.

Because one of the cornerstone ondemand treatment for H E.

Joined Jounce is already approved and launched in the U S for the treatment of H D. D S.

Seek a number of patients already on treatment.

And it is due to be launched in all our key markets around the world.

And last but not least the recent completion of the acquisition of the Bolivar is another stepping stone in the development of the company.

Yeah.

Fabrice Chouraqui: 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 have built to realize this vision. Full year 2024 revenues increased by 21% to EUR 297 million above our guidance range, including a strong Q4 and with operating profit and positive operating cash flow in the last two quarters of the year. RUCONEST grew 11% to EUR 252 million and 9% in the last quarter of the year, driven by a continued increase of new patient enrollments and the sustained expansion of our prescriber base.

We have a clear vision for Tommy.

Which is to develop a leading global rare disease company.

With a diverse portfolio and presence in large markets.

Leveraging our proven and efficient clinical development supply chain and commercial infrastructure.

Our results in 2024 are a good illustration of the solid foundation that we built to realize this vision.

Full year 2024 revenues increased by 21%.

$297 million above our guidance range.

<unk> strong fourth quarter, and with operating profit and positive operating cash flow in the last two quarters of the year.

We'll go next.

11% to $252 million or 9% in the last quarter of the year.

Driven by a continued increase of new patient enrollment and the sustained expansion of our credits fiber base.

Fabrice Chouraqui: 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 expansion and the development for much larger primary immunodeficiency indications. Let me now hand over to Stephen Toor, our Chief Commercial Officer, who will give you a more granular perspective on the strong dynamic of RUCONEST and JOENJA.

I believe that given its unique profile and positive experience in difficult to treat patients with.

<unk> will continue to grow and could even benefit from the potential increase of the on demand segment driven by the entry of new entrants.

Yeah.

Joined Jive revenue.

Increased by 147% to $45 million in 2024.

The drug is only in its very early stage of its lifecycle.

With continued growth to be seen with the.

Enrollment of new Apd as 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 expansion.

And the development.

For much larger primary immunodeficiency indications.

Let me now and over to Steve's door, our Chief commercial officer, who will give you a more granular perspective on the strong dynamic of reconnect and join us.

Stephen Toor: 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 EUR 80 million for the quarter. We ended 2024 with sales of EUR 252 million, 11% up on 2023. In the next two slides, I'll review why RUCONEST continues to show such strength in 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.

Steve: Thank you for Bruce Good morning, everybody.

As Chris just alluded to we have delivered another strong performance in 2024.

<unk>, we increased the prescriber base by 11% in new patient enrollments by 24%.

Steve: 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.

Steve: And the next two slides I'll review wide weakness continues to show strength and growth and while we are confident they will continue to grow in the years to come even as the market becomes more competitive.

Steve: On Georgia, we continue to build out patient pipe to uninsured just transition eligible patients to pain therapy.

Stephen Toor: As you would expect, our team delivered significant growth over the first year of launch, ending Q4 65% up on prior year at EUR 13.1 million, but for 2024, +147% of EUR 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 business for APDS, and with new potential indications for the molecule, create a strong high-growth franchise. Looking first at RUCONEST.

Steve: You would expect our team delivered significant growth over the first year of launch.

Steve: In Q4, 65% up on prior year at $13 $1 million of the 2024, plus 147% or 45 minutes.

Steve: Of note. In addition to <unk> 96 patients plus five pending on pay therapy in the U S. We have an additional 188 patients on therapy globally under various access programs and clinical trials that can all move to commercial therapy, when the necessary registrations received.

Steve: In the forthcoming slides also outlined the opportunity.

Steve: And as we say in the coming months and years that will both build the Georgia business Aps.

Steve: And with new potential indications for the molecule create a strong high growth franchise.

Steve: Looking first of all up request as I. Just stated <unk> is and will continue to be a growth driver for solving and an important treatment option for U S patients and their doctors.

Stephen Toor: 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. 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. While many patients are effectively treated by blocking a single point in these cascades, patients who don't respond to the targeted therapies available may benefit from RUCONEST, since it works comprehensively across all these systems.

Steve: Which is why it's already the second most prescribed to key product in the U S and one of the key reasons for this is its mode of action.

Steve: As you can see in the graphic there are three inflammatory cascades involved in the development of NHI attack.

Steve: So you want us to raise inhibition represented in the graphic by the rate C. One O A&H markers.

Steve: <unk> numerous enzymes across all three pathways.

Steve: So while many patients who are effectively treated by blocking a single point of these cascades patient.

Steve: Patients, who don't respond to the target therapies therapies available may benefit from Brokenness since it was come to comprehensively across all these systems.

Stephen Toor: 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. 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 RUCONEST patient groups have one thing in common, though. They all suffer from moderate to severe debilitating HAE 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 attacks.

Steve: <unk> inhibition ultimately stops bradykinin production via multiple points in the contact Cascade as well as other systems that may lead to attacks, which.

Steve: Which in turn and this is important lease the 97% of the resolution in a single dose and.

Steve: And a sustained response with 93% of patients attached stops for at least three days.

Steve: So, let's look more specifically at <unk> patients and what this means for them.

Steve: The first thing to note is that <unk> serves all patient types those being taught one tied to a normalcy when patients who.

Steve: Three page all three of these <unk> patient groups have one thing in common mode.

Steve: We all suffer from moderate to severe debilitating HAE attacks and they happen frequently.

Steve: They are also typically failed other targeted acute therapies, such as a caterpillar or having to re dose to stop their HIV attacks.

Stephen Toor: 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 attack-free for at least 3 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.

Steve: In the photos on the slide you can see our actual <unk> patients at the start of an attack and then a recovery as a result of the four Aramark number 24 hour Mark.

Steve: For patients who had this one suffering with the most vehicles a disease of Tech you frequently are happy to re dose on other therapies, knowing as I, just stated that 97% of patients will stop the attack with a single dose.

Steve: And almost all of them will be a tax free for at least three days is a very big deal.

Steve: Rutinose efficacy reliability allows outpatients to better control and plan their lives.

Steve: And Thats why weakness will continue to have a strong position in the U S acute market and remain an important product for our company needs to come.

Stephen Toor: 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. Now, 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. We've identified hundreds more in other key markets. 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. What are they?

Speaker Change: Our transition now to Joe and Jeff, which as Youre aware was launched in the U S. In March 2023, and is available outside the U S through various access programs.

Speaker Change: We see a number of opportunities for Germany, which I'll walk you through now that farming patient finding efforts are continuing as we build our patient pipeline in the U S and globally.

Speaker Change: In fact, we've already identified over 240 patients in the U S. 40% in the already on pay therapy, and we've identified hundreds more in other key markets.

Speaker Change: So while we work hard.

Speaker Change: <unk> supports relies identified patients and put them on therapy.

Speaker Change: We also have some important opportunities to drive growth in the near term and the medium term, including efforts to expand the addressable Drexel population. So what are they looking at the second block on the slide. The first is the outputs from the U S resolution program, which Eric will discuss.

Stephen Toor: 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 launch 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. 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 JOENJA for NHS England and Wales. We have further anticipated launches in other important markets, including Japan, Germany, France, Italy, Spain, Canada, and Australia.

Speaker Change: That will deliver another bolus of patients available for treatment this year and beyond.

Speaker Change: The second will be the pediatric indication launch in the U S, which is expected in 2026.

Speaker Change: We currently have over 60 patients in our U S pipeline and growing and they will begin transitioning to Joanne just as soon as the indication is approved.

Speaker Change: And the third.

Speaker Change: Is it geographic expansion program.

Speaker Change: Which is to key markets around the world who this begins this year with the UK launch in fact, just today Neuss, a published draft guidance in which they recommend the use of joined yet for NHS, England and Wales.

Speaker Change: And then we have further anticipated launches in other important markets, including Japan, Germany, France, Italy, Spain, Canada and Australia.

Stephen Toor: That means JOENJA will soon be available in most of the industry's top 10 markets. You can see in the final 2 blocks on this slide, leniolisib for APDS is only part of the story. As you know, phase II trials have been initiated for 2 bigger indications, ALPS and CVID. 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, 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.

That means showing you will soon be available in most of the industry's top 10 markets.

Speaker Change: In addition to that you can see in the final two blocks on this slide Linea <unk> is only part of the story as.

Speaker Change: As you know phase two trials have been initiated the two bigger indications opened an EBIT effect.

Speaker Change: In fact, <unk> was still red with a prevalence of 40 patients per million transitions linea <unk> from a small ultra rare disease molecules. So when with blockbuster sales potential, thereby trading millennial received franchise delivering a significantly greater value for all stakeholders in the coming years.

Speaker Change: With that said.

Ghrelin: Now I'd like to hand over to our Chief Medical Officer, underwrite ghrelin, whose team or of course critical to driving these programs forward to realizing these opportunities to provide us with the research and development update.

Anurag Relan: Thanks, Steve. 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 indications which can support the vision you heard Fabrice lay out 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 2 phase II studies underway, including a new program in CVID, or common variable immune deficiency. 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 you know, the VUS project has been a focus of our patient-finding work.

Speaker Change: Thanks, Steve.

Speaker Change: And here, we can see our growing pipeline for someone who's been at farming for many years. It has incredibly impressive to see how this has expanded from only <unk> to now include multiple products and indications, which can support division you heard from Bruce lay out what we have performed.

Speaker Change: Since <unk> is the primary immune deficiency with immune Dysregulation and there are other more prevalent tid's with similar features we're especially excited for the work that we have started this year with two phase III studies underway, including a new program and CVD or common variable immune deficiency.

Speaker Change: And last but not least we have on this slide a L. One for ofer.

Speaker Change: <unk> study with the recent acquisition.

Speaker Change: Yes.

Speaker Change: Before turning to development as you know that the U S projects has been a focus of our patient finding work a significant challenge in diagnosing atvs patients occurs when a patient's genetic test results shows that the U S or a variance of unknown significance.

Anurag Relan: 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 a VUS result in the genes associated with APDS. Over the past year, we have been supporting a project to gain additional insights into these VUSs. 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.

Speaker Change: This happens because we're very as novel and there isn't enough information to determine if the variance as disease, causing in fact, there are over 200 patients in the U S alone who have received at the U S results in the genes associated with Aes.

Speaker Change: Over the past year, we have been supporting our project to gain additional insights into the U. S is the recently concluded studies, which will be published soon as shown there are many new variance that lead to hyper activity in this pathway.

Speaker Change: The next step in the process is for genetic testing labs to review these data and determine which variance to now be reclassified as causing Ada es.

Speaker Change: We expect by midyear. These efforts will lead to the reputation of many new Aps patients.

Anurag Relan: 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 have 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 top-line results 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 observed in older APDS patients.

Speaker Change: Now, let's turn it over to work going on in pediatrics or we have an active clinical program with recent data to support regulatory filings.

Speaker Change: <unk> symptoms as you know began at a young age and more than 25% of the patients with five or below the age of 12.

Speaker Change: Since the disease is progressive it is important to be able to treat the condition earlier and scores and.

Speaker Change: In December we were excited to report the top line results from the first clinical study in children with Atvs ages four to 11.

Speaker Change: <unk> demonstrated that <unk> was generally safe and well tolerated and we saw benefit benefits across the two co primary endpoints, which were consistent with what we have observed an older ABS basis.

Anurag Relan: These data will be presented at a conference in May, and in H2 of this year, we will begin regulatory filings, 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 immunodeficiencies 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.

Speaker Change: These data will be presented at a conference in May.

Speaker Change: In the second half of this year, we will begin regulatory filings starting with FDA to expand the label to be able to treat younger patients with Acs.

Speaker Change: In addition to our work in Hdds, we are developing ready also for primary immune deficiencies with immune Dysregulation, which you see in the diagram or a subset of <unk> <unk>. In fact is one such example of a PIV with immune Dysregulation and we have started two more programs in this area.

First program is a genetically defined group of <unk>, which we started a study in October and we are continuing to enroll patients in the FDA recently granted fast track designation for the program.

Anurag Relan: 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. 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.

Speaker Change: We are announcing today the second program in CDI, which is a clinically defined group and represents an even larger group of the IV patients.

Speaker Change: Study is now open for enrollment and we expect the first patient to be dosed later this month.

Speaker Change: The patients in both of these studies lack of effective therapies to manage the immune dysregulation that leads to disease progression and early mortality.

Speaker Change: And you can see in the prevalence estimates how the new study, we announced today <unk> significantly increases the patient populations that could potentially benefit for millennials.

Across Aps and these new programs the central role of <unk> Delta and lymphocytes is clear and driving the immune dysregulation, which supports the investigation of laterals.

Anurag Relan: 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 to 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. In Europe, we have already concluded 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 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.

Speaker Change: I look forward to updating you further as we progress with these exciting new programs.

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

Speaker Change: As you heard from Steve, bringing millennials additional Aps patients represents a significant near term opportunity.

Speaker Change: In Europe, we have already concluded a clinical benefit and safety of our Nielsen or without 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.

Speaker Change: 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.

Anurag Relan: In Japan, we have completed an interim analysis of a small trial there, and this will now enable our filing with PMDA in the middle of this year. 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 1 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 PID indications, which support the long-term growth of leniolisib. Now turning to our third program in our portfolio via the just completed Abliva acquisition, KL1333. This is being developed for primary mitochondrial diseases, which are a group of rare disorders where mutations in mitochondrial DNA lead to impaired energy production.

In Japan, we have completed an interim analysis of a small trial there and this will now enable our filing with <unk> in the middle of this year.

Speaker Change: And you heard me already talk about the pediatric study in the in the four to 11 age groups, but we also have an additional trial in children as young as one Europe age, which is nearing completion of enrollment.

Speaker Change: In all there are quite a number of projects to be able to expand the addressable population, including these new indications will support the long term growth millennials.

Speaker Change: And now turning to our third program in our portfolio via the just completed I believe the acquisition tailwind Triple tree.

Speaker Change: This is being developed for primary mitochondrial diseases, which are a group of rare disorders, where mutations in mitochondrial DNA lead to impaired energy production.

Anurag Relan: These disorders can have array of diverse clinical features. 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. KL1333 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. There was also a blinded interim analysis in which both endpoints passed utility. Having just completed the acquisition, we plan to begin enrollment in the 2nd wave of the study as soon as possible with sites that are already open.

Speaker Change: These disorders that have a ray of diverse clinical features but a common element is a severe fatigue and muscle weakness seen in these patients which of course leads to a poor quality of life given the degree of symptoms.

Speaker Change: There are a large number of these patients already diagnosed across the U S and large European countries, where they're treated at centers of excellence as part of a strong advocacy group.

Speaker Change: Hale one triple three addresses the underlying disorder by normalizing the N plus two <unk> ratio, which is abnormally low in these patients. There is a pivotal study underway with endpoints agreed upon with FDA and there was also a blinded interim analysis in which both endpoints passed futility.

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

Anurag Relan: 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 need. Now I'll turn it over to Jeroen to review our financial performance and outlook.

Speaker Change: As with the rest of our portfolio. We see this program is one where we can use our rare disease expertise and infrastructure to bring much needed products to patients where there is significant unmet medical need now.

Speaker Change: Now I'll turn it over to your room to review, our financial performance and outlook.

Jeroen Wakkerman: Thank you very much, Anurag. Q4 was a very robust quarter for Pharming. Revenues grew by 14%, one four, versus a very strong Q4 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 a lease contract and just over EUR 1 million of Abliva acquisition cost. Operating profit increased by EUR 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.

Speaker Change: Thank you very much on Iraq.

Speaker Change: Q4 was a very robust quarter for farming revenues grew by 14% one four versus a very strong fourth quarter in 2023.

<unk> growth was 9% and <unk>, 66%.

Speaker Change: Gross profit grew by 9% and that is lower than the revenue growth that was driven by a one off inventory and Pam and following a reduction.

Speaker Change: <unk> production issue at one of our ships.

Speaker Change: Opex was fairly stable are largely as expected in the quarter.

Speaker Change: The small increase was caused by one off costs, including a full impairment of our lease contracts.

Speaker Change: Just over $1 million of Abreva acquisition costs.

Speaker Change: Operating profit increased by $5 6 million driven by higher gross profit as a consequence of strong sales and active Opex management.

Speaker Change: This was the second quarter in a row that we generated operating profit.

Jeroen Wakkerman: The net results went from a loss in Q4 2023 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. Now looking at the full year results. Full year results in 2024 were good. Revenue was up 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.

Speaker Change: And the net results went from a loss in Q4 23 to a net profit in Q4 2024.

Speaker Change: We had a positive operating cash flow in the quarter and in fact for the second quarter in a row.

Speaker Change: The cash and marketable securities reduced slightly due to interest costs and currency effects.

Speaker Change: Now looking at the full year results.

Speaker Change: Full year results in 2024 records.

Speaker Change: Revenue was up 21%, which was driven by both refinanced with a strong growth of 11% and Lenny Allison that grew by 147%.

Speaker Change: Opex increased by 10% due to investments in Georgia, which is at a rate well below the revenue increase.

Speaker Change: Operating profit on a like for like basis improves and Thats when taking out the big one offs that we had in 2023.

Jeroen Wakkerman: Our cash position reduced, 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 EUR 66.1 million acquisition of Abliva is now completed, Abliva shares approved for delisting next week. We initiated the compulsory acquisition procedure for the remaining Abliva shares, the acquisition of the shares was funded with available cash. The transaction really illustrates our strategy of developing a high-value pipeline. Moving to the financial guidance for this year, 2025.

Speaker Change: Our cash position reduced and that was driven by the refinancing of our convertible bonds earlier in 2024 for a lower amounts in the previous box.

Speaker Change: A quick update on the I believe our acquisition process and timeline.

Speaker Change: The $66 1 million acquisition of Liza is not completed and I believe a shares approved for.

Speaker Change: Delisting next week.

Speaker Change: We initiated the compulsory acquisition procedure for the remaining I believe of shares.

Speaker Change: And the acquisition was.

Speaker Change: A deposition of the shares was funded with available cash.

Speaker Change: And then transaction really illustrates our strategy of developing a high value pipeline.

Speaker Change: Moving to the financial guidance for this year 2025.

Jeroen Wakkerman: We expect the total revenues to land between EUR 315 and 335 million, which means a growth rate of 6% to 13%. The assumption underlying the guidance midpoint is a high single-digit RUCONEST growth and continued strong growth of JOENJA, with an acceleration in the H2 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 annual WAC of $594,000. Regarding operating expenses, we expect them to be flat on the previous years, on 2024, prior to the impact of Abliva. We have not yet integrated Abliva, and that process should start next week. Our preliminary estimate for Abliva-related OPEX is EUR 30 million for 2025, including EUR 17 million R&D costs, and the remainder consists of non-recurring transaction and integration costs.

Speaker Change: We expect total revenues to land between 315, and $335 million, which means a growth rate of six to 13.

Speaker Change: And the assumption underlying the guidance midpoint is a high single digits <unk> growth.

Speaker Change: And continued strong growth of <unk> with an acceleration in the second half of the year from the positive impact from the U S. As <unk> mentioned before.

Speaker Change: For <unk>, we expect continued growth of.

Speaker Change: Ah patients on <unk> therapy, and the U S pricing at a annual west of 594000 U S dollars.

Speaker Change: Regarding operating expenses, we expect them to be flat on the previous year's on 2024 prior to the impacts of a breather.

Speaker Change: We have not yet integrated I believe and that process should start next week.

Speaker Change: And our preliminary estimate for I believe related to Opex is $30 million for 2025.

Speaker Change: Including $17 million R&D costs, and the remainder consists of nonrecurring transaction and integration costs.

Jeroen Wakkerman: 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.

Speaker Change: And we will provide an update of these costs in our Q1 call in May.

Speaker Change: With that I would like to hand over back to <unk>.

Fabrice Chouraqui: Thank you, Jeroen. As you've heard, our in-line 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 enroll 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 for PID with immune dysregulation is a great opportunity to continue to expand JOENJA sales potential. Second, the acquisition of Abliva's pivotal stage program in mitochondrial disease brings another asset with significant revenue potential.

Speaker Change: You run as you've heard our in line portfolio generated strong growth in 2024.

Speaker Change: And we are exiting Q4 with solid momentum.

Speaker Change: With its unique profile and strong patient experience <unk> is well positioned to remain one of the treatment of choice for FX.

Speaker Change: In Mds after an initial strong OIBDA update we are now working to identify and enroll new patients.

Speaker Change: For capturing two well defined opportunities with them.

Speaker Change: The U S.

Speaker Change: And the expected pediatric indication.

Speaker Change: We continue to invest in our long term growth.

Speaker Change: With the objective to generate two blockbuster asset.

Speaker Change: First the potential new indication for PID with immune Dysregulation.

Speaker Change: He is a great opportunity to continue to expand joined just sales potential.

Speaker Change: Second the acquisition of I believe as pivotal stage program in Mexico in renal disease brings.

Speaker Change: <unk> another asset with significant revenue potential.

Fabrice Chouraqui: Our 2025 revenue guidance of EUR 315 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.

Speaker Change: Our 2025 revenue guidance of $3 $15 million to $335 million.

Speaker Change: It is great our momentum.

Speaker Change: And obviously, we look forward to updating you on our progress.

Speaker Change: Let me now open the line for questions.

Operator: Thank you. Your first question comes from the line of Jeff Jones from Oppenheimer. Please go ahead.

Speaker Change: Thank you Carlo asked a question you will need to press star one on one on your telephone and wait for you.

Speaker Change: Name to be announced to ensure your question. Please press star one on one again.

Speaker Change: We will now go to your first question.

Speaker Change: One moment please.

Speaker Change: And your first question comes from the line of Jeff Jones from Oppenheimer. Please go ahead.

Jeff Jones: Good morning, guys, or afternoon, I guess. Fabrice, welcome to the team and congratulations on a fantastic first earnings update here. I guess 2 questions for us. First, with respect to KL1333 in the targeted 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. Then, just a clarification for our 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? Perhaps could you provide some breakdown in terms of the territories where those patients are? Thanks.

Hi, good morning, guys or afternoon that gas.

Speaker Change: And so Bruce Wildcards.

Speaker Change: So the team and congratulations on a fantastic first.

Speaker Change: Earnings update here.

Speaker Change: I guess two questions for us.

Speaker Change: First with respect to <unk> $1 33.

Speaker Change:

Speaker Change: And the target of primary mitochondrial disease could you speak a little bit to how this patient population breaks down.

Speaker Change: And what portion of that population you think would be treatable and are eligible for treatment based on the likely label there are a lot of.

Speaker Change: Mutations within that group and then.

Speaker Change: Just to clarify.

Speaker Change: Clarification for a second question.

Speaker Change: For the 188 patients you mentioned that R&D expanded access program.

Speaker Change: And which includes clinical trials are any of those patients paid and perhaps could you.

Speaker Change: Provide some breakdown in terms of the territories, where those patients are.

Speaker Change: Thanks.

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

Jeff Jones: Thank you Jeff.

Speaker Change: Let us elaborate actually on your question, so I'll I'll handover to underwrite first.

Speaker Change: To tell you more about that.

Speaker Change: The addressable population with the.

Speaker Change: The ongoing trial on <unk>.

Speaker Change: 1033.

Anurag Relan: Hi, Jeff. 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, we've already limited it to the group of patients that have the mutations that are going to be enrolled in this study. 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. That's how we came up with this estimate of 30,000. In essence, all of those 30,000 are the addressable population.

Speaker Change: Hi, Jeff So with respect to primary mitochondrial diseases. When we talk about the 30000 number of patients that are in the U S. In the large European markets is 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.

Speaker Change: The bite of Chondral, DNA mutation, which already represent 80% of all <unk> and then we broke that down further and looked at the mutations that are specifically being enrolled in this study. So thats. How we came up with this estimate of 30000. So in essence all of those 30000 are the addressable population.

Jeff Jones: I appreciate that. Thank you.

Speaker Change: Okay I appreciate that thank you.

Anurag Relan: I'll turn it to Steve now to answer your question about the access program.

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

Stephen Toor: Hi, Jeff. Morning. 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 the early access program or compassionate use and also in clinical trials. We do have a number on paid therapy through named patient programs. As you can appreciate, that's not a specific number or a revenue line that we would discuss publicly.

Steve: Hi, Jeff Good morning.

Steve: Of the 188, we actually have those patients in multiple countries around the world including.

Steve: Many of the key markets that I listed earlier.

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

Jeff Jones: Appreciate that, guys. Thank you.

Steve: I appreciate that guys. Thank you.

Operator: Thank you. Your next question comes from the line of Ben Jackson from Jefferies. Please go ahead.

Steve: Thank you.

Steve: Your next question.

Speaker Change: Comes from the line of Ben Jackson from Jefferies. Please go ahead.

Ben Jackson: Great. Thank you for the question. Just two from me. If we firstly start on one that's not too exciting. The EUR 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 noted that already in the call. The second one, just interestingly, do you see any theoretical exposure to potential US tariffs if they were to apply for 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. I guess as a result of that, have you noticed any kind of level of change in 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.

Alright. Thank you for the question just two from me.

Speaker Change: So let me start on one theres not too exciting of circa 30 million anticipated additional effects from the acquisition are you able just to touch on how much of that is recurring.

Speaker Change: Apologies, if you guys had already Nicole.

Speaker Change: The second one just interestingly do you see any theoretical exposure to potential U S tariff Eric to apply for trucks again at this point that it's very unclear whats going to happen and we we don't necessarily know where it's going and then I guess as a result of that have you noticed any kind of level of changing inventories of stocking or.

Speaker Change: Interest in the magic measure to hedge against the potential shortly of trade war or anything like that.

Speaker Change: You thought some color around that would be great. Thank you.

Fabrice Chouraqui: Thank you, Ben. Let me take your first question about the EUR 30 million of OPEX spend on Abliva in 2025, as announced by Eero. About EUR 17 million will be R&D, and the rest will be non-recurring transaction and integration costs. When it comes to the tariff, obviously we are monitoring the situation, and we're doing some homework in the background looking how we can minimize the impact of potential tariff 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. We can tell you more in due time. This is obviously something on which we are proactive and are monitoring very closely.

Speaker Change: Thank you Ben So let me take your first question about the $30 million.

Speaker Change: Opex.

Speaker Change: Spend on I believe in 2025 as announced by Euro and about $17 million will be R&D and the rest would be non recurring transaction and integration costs.

Speaker Change: Now when it comes to the tariff obviously, we are monitoring the situation and we're doing some work in the background looking how we can.

Speaker Change: Minimized.

Speaker Change: The impact of potential tariff if there were decided by the U S administration.

Speaker Change: Looking at some adaptation that we could make to our supply chain. This is obviously ongoing I cannot share any details.

Speaker Change: We.

Speaker Change: We can tell you more in due time, but this is obviously something on which we are proactive.

Speaker Change: And.

Speaker Change: And monitoring very very closely.

Ben Jackson: Perfect. Thank you.

Speaker Change: Perfect. Thank you.

Fabrice Chouraqui: Steve?

Speaker Change: Hey, Matt.

Speaker Change: Okay.

Anurag Relan: I think there was a question on whether there is any stocking or inventory buildup as in anticipation of the tariffs.

Speaker Change: But I think there was a question on whether there is any stocking or inventory buildup in anticipation of the tariffs.

Stephen Toor: 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.

Speaker Change: We have enough stock already for our needs within the U S and there is no planned inventory buildups at this point in time until we have a clearer picture of the situation.

Ben Jackson: Very clear. Thank you very much.

Speaker Change: Alright, great. Thank you very much.

Operator: Thank you. Your next question comes from the line of Alistair Campbell from RBC. Please go ahead.

Speaker Change: Thank you.

Speaker Change: Your next question comes from the line of Alistair Campbell from RBC. Please go ahead.

Alistair Campbell: Hi there. Thanks very much for taking the questions today. As I said, let me start with RUCONEST. It's great to see a level of confidence that this product will continue to grow. 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? Then thinking about the outlook for 2025, clearly you feel very confident that you will continue to grow despite new competition. Is that sort of based on your internal thinking or I'm 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. Finally, maybe turning to CVID, just a sense of the time frame for those trials. Should phase II trials be sufficient for approval?

Hi, there thanks very much for taking the questions today.

Speaker Change: Let me start with <unk> great.

Speaker Change: C level conference.

Speaker Change: We'll continue to grow I'm, just very briefly in the Q4 number.

Just for modeling purposes was there any stocking effects in there was that largely driven by.

Speaker Change: Underlying demand and then thinking about the outlook for 2025, I mean, clearly you feel very confident that you will continue to grow despite your competition.

Speaker Change: Sort of based on your internal thinking just tweaked 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.

Speaker Change: And finally, maybe turning to CVR D. Just a sense of the timeframe for those trials should phase II trials would be sufficient for approval.

Alistair Campbell: How you think those trials will likely be scoped in terms of size and duration. Thank you.

Speaker Change: How do you think those trials will likely be scoped in terms of size and duration. Thank you.

Fabrice Chouraqui: Thank you so much for your question, Alistair. I'll start answering some of them and hand over to Steve and Eron right for the last one on CVID. I want to reinforce that actually there's not been much stocking actually in Q4. The result, the very strong result that you saw actually on RUCONEST were by and large actually driven by strong demand. That should be very clear. When it comes to the positioning of RUCONEST, again, as we've said, because of the unique profile of the drug and the very strong patient experience that we've been able to generate years after years since the launch of the drug more than 10 years ago, we feel confident that RUCONEST will remain the treatment of choice for HAE attacks despite new over entrants.

Speaker Change: So thank you so much for your question Alastair I'll start answering.

Speaker Change: Some of them in Andover too.

Speaker Change: <unk> and <unk> for the for the last one on <unk>.

Speaker Change: Want to reinforce that actually there has not been.

Speaker Change: Much stocking actually in Q4 and.

Speaker Change: The result of very very very strong result, so actually.

Speaker Change: On reconnect.

Speaker Change: And last by enlarge actually driven by strong demand.

Speaker Change: It should be very clear.

Speaker Change: When it comes to.

Speaker Change: The positioning of reconnect.

Again.

Speaker Change: As we've said I mean because.

Speaker Change: The unique profile of the drug.

Speaker Change: And.

The very strong patient experience that we've been able to generate years after years since.

Speaker Change: Since the launch of the drug more than 10 years ago, we feel confident that we can act where remain a treatment of choice.

Speaker Change: For <unk>, despite new or interests.

Fabrice Chouraqui: As we've seen in other categories, there will probably actually be the new orals will be able to help to develop the market. They'll be able to position themselves for specific categories of patients. Based on our experience with RUCONEST and on the feedback we are gathering from doctors and more general market insights, this is our strong belief. I'll ask Steve, obviously, to comment given his experience with the drug.

Speaker Change: As we've seen in other categories.

That will probably actually be the new <unk> will be out to develop the markets.

Speaker Change: Would be able to position themselves for a specific categories of patients.

Speaker Change: On our expanse with Rick on AD spend on the feedback we are gathering from doctors and more general market insights. This is our strong belief in all our Alaska steel obviously.

Speaker Change: To comment given these expanse with the driver.

Stephen Toor: 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. 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.

Speaker Change: Certainly the only the only thing I would add to that Alistair and you asked about market researches.

Speaker Change: Obviously pressure tested that through a number of advisory boards and steering committees over the past year.

We resumed the conclusion of those interactions is exactly we'll probably set which is this is a relatively unique drug in terms of its mode of action.

Speaker Change: The patients we serve as severe in the tech frequently and they are a positive experience and for those reasons.

Speaker Change: Say validated externally.

Speaker Change: We believe that <unk> is a place both in the short term and the long term and will continue to be a growth driver for farming.

Alistair Campbell: Thank you.

Speaker Change: Thank you.

Anurag Relan: Alistair, on the question about the CVID studies, we'll have some more details after we dose the first patient. This will be a phase II 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. We'll also then be able to talk to you about what the development path might look like.

Speaker Change: And then Alistair on the question about the <unk> studies.

Speaker Change: We'll have some more details after we dosed the first patient.

Speaker Change: But this will be a phase II 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 began the program poorly and we will also then be able to talk to you about what the development path might look like.

Operator: Thank you. Your next question comes from the line of Joe Pantginis from H.C. Wainwright. Please go ahead.

Speaker Change: Thank you.

Speaker Change: Your next question comes from the line of Jay <unk>.

Speaker Change: <unk> from H C. Wainwright. Please go ahead.

Joe Pantginis: Hey, guys. Joe Pantginis from H.C. Wainwright. Thanks for taking the questions. Fabrice, obviously, good luck at the helm here. A very successful company, I think you're taking over at a great time as well. 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?

Speaker Change: Hey, guys, Joe Pan Janus from H C. Wainwright, thanks for taking the questions for Bruce obviously, good luck at the helm here very successful companies. So I think you are taking over.

Speaker Change: I had a great time as well. So first question is with regard to weaken next and building the new prescriber base in the U S.

Speaker Change: How would you sort of describe the yes versus no dynamic as youre trying to convince physicians to be new prescribers to support the core core growth of the asset.

Fabrice Chouraqui: All right. Thank you, Joe, for your kind words. When it comes to RUCONEST, I've actually, over the past few days, actually met a few RUCONEST prescribers, got a first-hand experience of what's happening in daily medical practice. I think we see an increased number of prescribers with RUCONEST as they can see that the drug has a unique profile. As a consequence, it has a unique value proposition for a specific segment of the patient, those difficult-to-treat patients who are experiencing actually a number of breakthrough attacks. Those doctors, I think, are reinforced by their experience of the drug. Some of them spoke to me about a drug which is transformative. I'm using, again, the word transformative for the life of their patients. I think that's very meaningful.

Speaker Change: Alright, Thank you Joe for.

Speaker Change: You kind of words when it becomes when it comes to reconnect I've actually over the past.

Few days actually Max <unk>.

Speaker Change: Prescribers and so got it.

Speaker Change: Our first test and experienced.

Speaker Change: What's happening in Dirty medical practice.

I think we see a growth an increased number of prescribers.

Speaker Change: With recognized as they can see that the drug has a unique profile.

Speaker Change: And.

Speaker Change: As a consequence, it has a unique value proposition for our specialty segments of the patient.

Speaker Change: It was difficult to treat patient with who are experiencing actually a number of breakthrough attacks.

Speaker Change: And.

Speaker Change: Those doctors I think are reinforced by the experience of.

Speaker Change: Of the drug I mean some of them.

Speaker Change: Spoke to me about the drug which is transfer many I'm using again the word transformation for the life of their patients and so.

Speaker Change: I think thats very meaningful.

Fabrice Chouraqui: Given my experience, when you hear a clinician who has actually treated patients with such a debilitating disease, you've seen the picture actually of a patient suffering from an attack, that means a lot. Steve, you want to elaborate a bit?

Speaker Change: In my experience I mean, when you hear a clinician.

Speaker Change: Who is actually treated patients with such a debilitating disease.

Speaker Change: You have seen.

Speaker Change: The picture actually of the patients.

Speaker Change: <unk> from a netback.

Speaker Change: That means a lot Steve do you want to elaborate a bit sure. Thank you for Bruce Hey, Joe.

Stephen Toor: 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. Even in year 10, we were able to grow the prescriber base by 11%. 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. What we find, especially with those physicians, is they're very open to the concept of RUCONEST because they're now having to treat these severe, frequently attacking patients that they haven't before. As I said, we have a strong base of prescribers.

Speaker Change: I think to answer your question of the yes versus no dynamic where 10 years post launch and obviously this is a well developed market and even in years and we were able to grow the prescriber base by 11%.

Speaker Change: 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 have not to consider routine us because they are simply not had a patient they've had the first one so what we found especially with those those physicians is that very open to the concept of brokenness because that now happen to treat these severe frequent.

Speaker Change: And youre talking patients they haven't before so as I said, you know we have a strong base of prescribers, but as the market expands over time and as we go deeper into them.

Stephen Toor: As the market expands over time and as we go deeper into them, we are very confident that more and more physicians will need to prescribe and will use and be open to it.

Speaker Change: We are very confident that more and more patients sorry, physicians will need to prescribe and will use them to be open to it.

Joe Pantginis: That's really helpful, Steve. Thanks for that. I guess, Fabrice, 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 in-licensing of assets to look forward to?

Bruce: That's really helpful. Steve Thanks for that and then I guess for Bruce.

Bruce: Certainly acknowledge this is a very early time to ask this question.

Bruce: Anything you could share with regard to changes you might or might not ambition for the Companys growth example, you know is there any further right sizing of the sales force the impact of new assets or even further in licensing of assets to look forward to.

Fabrice Chouraqui: It is still very early, actually, to tell you about what will really be my vision and the roadmap for the company. I believe, again, given what you have heard today about the momentum, that clearly we will continue to move forward. I have 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. You have seen that there are a number of great opportunities with our current assets, but also continue to look at value-accretive deals that could actually drive shareholder value in the mid and long run.

It's in its very early actually to tell you.

Bruce: About.

Bruce: What will really be my vision and the roadmap for the company I believe again, given what you've had.

Today about the momentum that's.

Bruce: Clearly, we will continue to move forward.

Speaker Change: I try to share a bit about my vision for the company, which is actually very similar to what you had from some Simon in the past.

Bruce: I think.

Bruce: Our success will come from Greg ambition.

Bruce: <unk>.

Bruce: Our relentless focus on execution.

Bruce: Rigorous P&L management, and Opex management specifically.

Bruce: And then the continued expansion of our pipeline looking how we can expand our pipeline with the current assets and you've seen.

Bruce: And that there are a number of greater Boston he's without that.

Bruce: But also continue to look at value accurately.

Bruce: <unk>.

Bruce: That could actually drive shareholder value.

Bruce: In the meat in the world.

Joe Pantginis: Greatly appreciate that.

Fabrice Chouraqui: to tell you more in the coming weeks and months as I am becoming more and more knowledgeable with the intricacies of the company.

Bruce: I'd be very happy.

In the coming weeks and months.

Bruce: As I'm, becoming more and more knowledgeable with the intricacies of LNG.

Joe Pantginis: Of course. I had to ask. Thanks for that, and thanks for all the color, guys.

Bruce: Of course, I had to ask and thanks for that.

Bruce: Thanks for all the color guys.

Operator: Thank you. As a reminder, if you would like to ask a question, please press star one and one on your telephone keypad. That is star one and one if you would like to ask a question. We will now go to the next question. The question comes from the line of Simon Scholes from First Berlin. Please go ahead.

Bruce: Thank you.

Speaker Change: As a reminder, if you would like to ask a question. Please press star one on your telephone keypad that is star one if you would like to ask a question.

We will now go to the next question.

Speaker Change: And the question comes from the line of filings calls from Berlin. Please go ahead.

Simon Scholes: 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 2026 and 2027. On CVID, my understanding is that on PI3Kδ, you will require a phase III. I was just wondering if you could give us an indication of why you might not require a phase III on CVID.

Speaker Change: Yes. Good afternoon, thanks for taking my questions.

Speaker Change: Two.

Speaker Change: Just to follow up on the I believe a cough.

Speaker Change: I was wondering if you could give us an indication of how those costs might evolve.

Speaker Change: 26.

Speaker Change: In 2007.

Speaker Change: And then on.

Steve: Steve It on my understanding is that on <unk> LTE will require.

Steve: Our phase III I was just wondering if you could give us an indication of what you might not require.

Steve: Phase III on ceded.

Fabrice Chouraqui: All right. Let me quickly answer your question about the spend on the Abliva program. As I said, and you heard it from Jeroen, about EUR 30 million this year, EUR 17 R&D, the rest non-recurring transaction and integration costs. When we announced the deal at the end of last year, we've estimated the total cost of the program to be around EUR 120 to 125 million. Okay?

Steve: Alright, So let me quickly answer your question about.

Steve: The spend on the I believe a program so as I said.

Steve: You heard it from you rune about $30 million this year 17, R&D the rest nonrecurring.

Steve: Transaction and integration costs.

Steve: We.

Steve: We announced the deal at the end of last year, we've estimated the total cost.

Steve: The program to be around $121 $25 million.

Simon Scholes: Okay.

Fabrice Chouraqui: You do the deduction, with the EUR 30 million, that we're still in that bulk. Obviously, we'll refine this as we complete the integration, resume the trial. Today, we don't have any data that tells us that actually this will be any different. When it comes to CVID, I'll let Anurag Relan actually clarify perhaps some misunderstanding.

Steve: Okay.

Steve: The deduction, you know with the $30 million that we are still in that belt. Obviously, we will refine this as we complete the integration.

Steve: Resume the trial, but today, we don't have any data on that.

Steve: But actually this would be any different.

Speaker Change: Now when it comes to the CBS I'll, let Andrew I actually quantify.

Steve: Perhaps.

Anurag Relan: Yeah. Hi, Simon. We do anticipate, and again, this is early days and not having even dosed the first patient yet, but we do anticipate that there would be a need for a phase III study, as we've done with APDS. We're not anticipating. These are rare diseases, so these are still relatively small programs. The phase II program in the first PID with immune dysregulation is 12 patients. The CVID indication phase II program will be slightly larger, but we still anticipate a phase III requirement to enable registration. Of course, as the phase II read out, we'll be able to tell you more what those phase IIIs look like. That's our current planning.

Steve: Some some misunderstanding.

Steve: Hi, Simon So we do anticipate and again this is early days and not having EBIT dose the first patient yet, but we do anticipate that there would be a need for a phase III study.

Steve: As we've done with Aps, we're not anticipating these are rare diseases. So these are still relatively small programs.

Steve: The phase II program in the in the first PIV with immune Dysregulation is 12 patients. The CVI indication phase III program will be slightly larger, but we still anticipate a phase III requirement to enable registration of course as the phase III readout will be able to tell you more.

Steve: Those phase III look like but that's our current level.

Simon Scholes: Okay. Just one last one. Do you also expect CVID to get a FDA fast track designation at some stage?

Steve: Okay, and just one last one.

Steve: We also expect <unk> to get.

Steve: <unk>.

Steve: Fast track designation at some stage.

Anurag Relan: We will certainly look at all of those types of options. These are severe diseases. They have a similar course as APDS. There is early mortality associated with them. 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.

Steve: We will certainly look at all of those types of options. These are these are severe diseases or they have a similar course as atvs theres early mortality associated with them.

Steve: These are patients who have these conditions. So we do anticipate being able to work with the regulators to try to expedite the development.

Simon Scholes: Okay, thanks very much. That's really helpful.

Speaker Change: Okay. Thanks, very much that's very helpful.

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

Speaker Change: Thank you.

Speaker Change: There are currently no further questions I will hand, the call back to <unk> for closing remarks.

Fabrice Chouraqui: 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 there are very clearly identified growth opportunities ahead of us. As per 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 in-line brands with Abliva. 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.

Thank you very much operator.

Speaker Change: Very much to those of you who attended the call.

Speaker Change: And those of you on the webcast.

Speaker Change: As I said I'm very excited to be joining farming.

Speaker Change: I believe that we are exiting 2024 with a very solid momentum that they are very clearly identified.

Speaker Change: Growth of Baltimore is ahead of us.

Speaker Change: And.

Speaker Change: As for my answer to the question our success will come from our ability to realize them.

Speaker Change: Sure.

Speaker Change: Manage our P&L rigorously and maintain a very high level of ambition given.

Speaker Change: Clearly the growth prospects that we can have with our in line brands with Opdivo and also I think the capabilities and the unique infrastructure that we have created that can really positioned the company.

Speaker Change: A leading rare disease company in the future.

Speaker Change: Thank you very much.

Operator: Thank you. This concludes today's conference call. Thank you for participating. You may now disconnect.

Speaker Change: Thank you. This concludes today's conference call. Thank you for participating you may now disconnect.

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Speaker Change: Okay.

Okay.

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Speaker Change: Yeah.

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Speaker Change: Yes.

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