Pharming Group Q4 2024 Earnings Report

Pharming Group EPS Results

• Actual EPS: $0.05
• Consensus EPS: $0.07
• Beat/Miss: Missed by -$0.02
• One Year Ago EPS: N/A

Pharming Group Revenue Results

• Actual Revenue: $92.70 million
• Expected Revenue: $76.67 million
• Beat/Miss: Beat by +$16.03 million
• YoY Revenue Growth: N/A

Pharming Group Announcement Details

• Quarter: Q4 2024
• Date: 3/13/2025
• Time: Before Market Opens
• Conference Call Date: Thursday, March 13, 2025
• Conference Call Time: 8:30AM ET

Pharming Group (NASDAQ:PHAR) N.V., a biopharmaceutical company, develops and commercializes protein replacement therapies and precision medicines for the treatment of rare diseases in the United States, Europe, and internationally. The company offers RUCONEST, a recombinant C1 esterase inhibitor for the treatment of acute attacks in adult and adolescent patients with acute hereditary angioedema (HAE); and Joenja (leniolisib), an oral small molecule PI3K? inhibitor for the treatment of activated phosphoinositide 3-kinase delta syndrome. It also develops OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of HAE. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105. Pharming Group N.V. was incorporated in 1988 and is headquartered in Leiden, the Netherlands.

Pharming Group Q4 2024 Earnings Call Transcript

Key Takeaways

• 2024 revenue grew 21% to $297 M, driven by strong Rukonest and Joenja sales, with operating profit and positive operating cash flow in Q3 and Q4.
• Rukonest sales rose 11% to $252 M, backed by an 11% increase in prescribers and 24% growth in new patient enrollments, securing its spot as the #2 acute HAE treatment in the U.S.
• Joenja revenue surged 147% to $45 M, with 96 U.S. patients on paid therapy and 188 in global access or trials, ahead of launches in the U.K., Japan and other major markets.
• Pipeline expansion includes VUS reclassification to identify new APDS patients by mid-year, a pediatric APDS label in 2026, and Phase II trials in CVID and LPID to broaden patient reach.
• The $66 M acquisition of Abliva brings KL1333 for primary mitochondrial disease into the portfolio, with a pivotal study underway and a positive interim futility readout boost­ing long-term growth prospects.

Presentation

[Operator]

Good day and thank you for standing by. Welcome to The Farming Group Fourth Quarter Full Year twenty twenty four Results Conference Call and Webcast. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer session. To ask a question during the session, you will need to press star one and one on your telephone.

[Operator]

I would now like to hand the conference over to your first speaker today, Fabrice Shouaki, CEO. Please go ahead, sir.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

Thank you very much. Hello, everyone, and welcome to the Farming's full year and Q4 twenty twenty four financial results call. I'm Fabrice Shoaki, CEO of Farming, and I'll be joined on this call today by Steven Thor, our Chief Commercial Officer Anurag Rellan, our Chief Medical Officer and Jeroen Wachaman, our Chief Financial Officer. We will be making forward looking statements in this call that are based upon our current insights and plan. As you know, this may differ from future results.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

First of all, let me say that I'm really excited to be joining FARMING, and it's an honor to succeed Simon DeVries. I'm passionate about progressing medical sciences and bringing innovation to patients, and naturally, I feel deeply connected with FARMING's mission to serve the unserved rare disease patients. Over the past twenty five years, I've developed an 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 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 farming over the past fifteen years and its significant growth prospects.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

Rookanest has become one of the cornerstone on demand treatments for HAE. Joenja is already approved and launched in The U. S. For the treatment of APDS with a second number of patients already on treatment and it is due to be 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

We have a clear vision for pharma, 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 $297,000,000 above our guidance range, including a strong fourth quarter and with operating profit and positive operating cash flow in the last two quarters of the year. Rouconest grew 11% to two fifty two million dollars and 9% in the last quarter of the year, driven by a continued increase of new patient enrollment and the sustained expansion of our credit card base. I believe that given its unique profile and positive experience in difficult to treat patients, RECONEST will continue to grow and could even benefit from the potential increase of the on demand segment driven by the entry of new entrants.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

Joenja revenue increased by 147% to $45,000,000 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 U. S, 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 Steve Thor, our Chief Commercial Officer, who will give you a more granular perspective on the strong dynamic of RUKONET and Joenja.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Thank you, Fabrice. Good morning, everybody. As Fabrice just alluded to, we've delivered another strong performance in 2024. On RUKENESS, 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 $80,000,000 for the quarter.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

We ended 2024 with sales of $252,000,000 11 percent up on 2023. In In the next two slides, I'll review why RUKENESS continues to show such strength in growth and while we're confident it will continue to grow in the years to come, even as the market becomes more competitive. On JOENGIA, we continue to build our patient pipeline and transition eligible patients to paid therapy. And as you would expect, our team delivered significant growth over the first year of launch. Ending Q4, '60 '5 percent up on prior year of $13,100,000 but for 2024, plus 147% of $45,000,000 Of note, in addition to 96 patients plus five pending on paid therapy in The U.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

S, 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 Joininger business for APDS and with new potential indications for the molecule create a strong high growth franchise. Looking first at RUKENESS, as I just stated RUKENESS is and will continue to be a growth driver for farming and an important treatment option for U. S. Patients and their doctors, which is why it's already the second most prescribed acute product in The U.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

S. 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 Rugenest since it works comprehensively across all these systems.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

C1S2A's 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 ninety seven percent attack resolution in a single dose and a sustained response with ninety three percent of patients attacks stopped for at least three days. So let's look more specifically at RUKENESS patients and what this means for them. The first thing to note is that RUKENESS serves all patient types, those being type one, type two and normal C1 patients. All three of these RUKENESS 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 acatopan or are having to redose to stop their HAE attack.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

In the photos on the slide, you could see an actual RUKENESS patient at the start of an attack and then her recovery as it resolves at the four hour mark and the twenty four hour mark. For patients like this one suffering with a more severe course of disease attacking frequently and happen to redose on other therapies, knowing as I just stated that ninety seven percent of patients will stop their attack with a single dose and almost all of them will be attack free for at least three days is a very big deal. RUKENESS efficacy reliability allows our patients to better control and plan their lives and that's why RUKENESS will continue to have a strong position in The U. S. Acute market and remain an important product for our company in the years to come.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

I'll transition now to Joininger, which as you're aware was launched in The U. S. In March 2023 and is available outside The U. S. Through various access programs.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

We see a number of opportunities for Joininger, which I'll walk you through now. Our pharming's patient finding efforts are continuing as we build our patient pipeline in The U. S. And globally. In fact, we've already identified over two forty patients in The U.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

S. Of whom forty percent are already on pay 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 addressable population. So what are they? Looking at the second block on the slide, the first is the outputs from the BUS resolution program, which Anurag will discuss.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

That will deliver another bonus of APDS patients available for treatment this year and beyond. The second will be the pediatric indication launched in The U. S. Which is expected in 2026. We currently have over 60 patients in our U.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

S. Pipeline and growing and they will begin transitioning to Joanna as soon as the indication is approved. And 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.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

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, lineolacifor APDS is only part of the story. As you know, Phase two trials have been initiated for two bigger indications, LPID and CVID. In fact, CBID was still rare with a prevalence of forty patients per million transitions lenovoCID from a small ultra rare disease molecule to one with blockbuster sales potential, thereby creating the lenovoCID franchise delivering a significantly greater value for all stakeholders in the coming years.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

With that said, I'd now like to hand over to our Chief Medical Officer, Anurag Grellan, whose team are of course critical to driving these programs forward and realizing these opportunities to provide us with a research and development update.

[Anurag Relan, Chief Medical Officer at Pharming Group]

Thanks, Steve.

[Anurag Relan, Chief Medical Officer at Pharming Group]

And here we can see our growing pipeline. For someone who's been at farming for many years, it is incredibly impressive to see how this has expanded from only rupanast for HAE to now include multiple products and indication, which can support the vision you heard Fabrice layout that we have for Martin. 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 two studies underway, including a new program in CDID 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 Olima. Before turning to development, as you know, the VUS project has been a focus of our patient finding work.

[Anurag Relan, Chief Medical Officer at Pharming Group]

A significant challenge in diagnosing APDS patients occurs when a patient's genetic test result shows a BUS or a variant of unknown significance. This happens because the variant is novel and there isn't enough information to determine if the variant is disease causing. In fact, there are over twelve hundred patients in The U. S. Alone who have received the VUS result in the genes associated with APDS.

[Anurag Relan, Chief Medical Officer at Pharming Group]

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 midyear 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.

[Anurag Relan, Chief Medical Officer at Pharming Group]

APDS symptoms as you know begin at a young age and more than twenty five percent 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 four to 11. The study demonstrated that lenulocyte 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 filings starting with FDA to expand the label to able to treat younger patients with APDS.

[Anurag Relan, Chief Medical Officer at Pharming Group]

In addition to our work in APDS, we are developing Lenoelisib 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 FDA recently granted Fast Track designation for the program. We are announcing today the second program in CDID, 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.

[Anurag Relan, Chief Medical Officer at Pharming Group]

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 prevalent estimates how the new study we announced today in CVID significantly increases the patient population that could potentially benefit from lineolysis. Across APDS and these new programs, the central role of PI3K delta in lymphocytes is clear in driving the immune dysregulation and supports the investigation of LENOLLISID. 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 Leniolus into more APDS patients in several key markets across the world and expand the addressable population.

[Anurag Relan, Chief Medical Officer at Pharming Group]

As you heard from Steve, bringing Leniolus to additional APDS patients represents a significant near term opportunity. And in Europe, we have already concluded the clinical benefit and safety of Lenoelisib or 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 twenty twenty six. 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 PMDA in the middle of this year. And you heard me already talk about the pediatric study in the four to eleven age group, but we also have an additional trial in children as young as one year of age, which is nearing completion of enrollment.

[Anurag Relan, Chief Medical Officer at Pharming Group]

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 linealism. And now turning to our third program in our portfolio via the just completed IBLIVA acquisition, TL1333. 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. These disorders can have array of diverse clinical features, but common elements 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 U.

[Anurag Relan, Chief Medical Officer at Pharming Group]

S. 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 plus 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 a blinded interim analysis in which both endpoints passed futility. 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, Chief Medical Officer at Pharming Group]

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.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

Thank you very much Anurag. Q4 was a very robust quarter for Pharma. Revenues grew by 141.4% versus a very strong fourth quarter in 2023. Recurrence growth was 9% and Joenzi at 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, a merchandise production issue at one of our CNOs.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

The OpEx was fairly stable and largely as expected in the quarter. The small increase was caused by one off costs including a full impairment of a lease contract and just over $1,000,000 of ABLIVA acquisition costs. Operating profit increased by $5,600,000 driven by higher gross profit as a consequence of strong sales and active OpEx management. This was a second quarter in a row that we generated operating profit. And the net results went from a loss in Q4 twenty twenty three to a net profit in Q4 twenty twenty four.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

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 Rubenest with a strong growth of 11% and LENIALOSIP that grew by 147%. OpEx increased by 10% due to investments in Joenja which is at a rate well below the revenue increase.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

Operating profit on a like for like basis improves and that's when taking out the big one offs that we have 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 Ibelieve acquisition process and timeline. The 66,100,000.0 acquisition of Ibelieve 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.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

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 million and $335,000,000 which means a growth rate of 6% to 13%. And the assumption underlying the guidance midpoint is a high single digit LucaNest growth and continued strong growth of Duenja with an acceleration in the second half of the year from the positive impact from BU assets as Anurag mentioned before. For Duenja, we expect continued growth of patients on paid therapy and The U.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

S. Pricing at an annual WACC of USD594000. 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. And our preliminary estimate for I believe related OpEx is $30,000,000 for 2025 including $17,000,000 R and D costs and the remainder consists of non recurring transaction and integration costs.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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 GOINGER 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

First, the potential new indication of PID with immune dysregulation is a great opportunity to continue to expand Joininger's sales potential. Second, the acquisition of IBLIVA pivotal stage program in mitochondrial disease brings another asset with significant revenue potential. Our 2025 revenue guidance of $315,000,000 to $335,000,000 illustrates our momentum and obviously we look forward to updating you on our progress. Let me now open the line for questions.

[Operator]

Thank you.

[Jeff Jones, Managing Director and Senior Analyst at Oppenheimer & Co. Inc.]

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 KL-one hundred and thirty three 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 a clarification for our second question, For the one hundred and eighty eight patients you mentioned that are on the expanded access program, which includes clinical trials, are any of those patients paid?

[Jeff Jones, Managing Director and Senior Analyst at Oppenheimer & Co. Inc.]

And perhaps could you provide some breakdown in terms of the territories where those patients are?

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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.

[Anurag Relan, Chief Medical Officer at Pharming Group]

Hi, Geoff. So with respect to primary mitochondrial diseases, when we talk about the 30,000 number of patients that are in The U. S. And the large European markets, this actually is 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.

[Anurag Relan, Chief Medical Officer at Pharming Group]

So that's how we came up with this estimate of thirty thousand. So in essence, all of those thirty thousand are the addressable population.

[Jeff Jones, Managing Director and Senior Analyst at Oppenheimer & Co. Inc.]

I appreciate that. Thank you.

[Anurag Relan, Chief Medical Officer at Pharming Group]

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

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Hi, Jeff. Good morning. So of the one hundred and eighty eight, 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 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 a revenue line that we would discuss publicly.

[Jeff Jones, Managing Director and Senior Analyst at Oppenheimer & Co. Inc.]

Appreciate that guys. Thank you.

[Operator]

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

[Benjamin Jackson, Senior Equity Research Associate at Jefferies]

Great. Thanks for the question. Just two for me. If we first start on one that's not too exciting. So the $30,000,000 anticipated additional OpEx from the Blivaraf acquisition, are you able just to touch on how much of that is recurring?

[Benjamin Jackson, Senior Equity Research Associate at Jefferies]

Apologies if you've noted that already in the call. And then the second one, just interestingly, do you see any theoretical exposure to potential U. S. Tariffs to apply to drugs? Again, at this point that it's very unclear what's going to happen and we don't necessarily know where it's going.

[Benjamin Jackson, Senior Equity Research Associate at Jefferies]

And then I guess as a result of that, have 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

Thank you, Ben. So let me take your first question about the $30,000,000 of OpEx spend on Abliva in 2025 as announced by Jeroen. About SEK 17,000,000 will be R and D and the rest will be non recurring transaction and integration costs. Now 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 U. S.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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 are monitoring very, very closely.

[Benjamin Jackson, Senior Equity Research Associate at Jefferies]

Perfect. Thank you.

[Anurag Relan, Chief Medical Officer at Pharming Group]

I think there was a question on whether there's any stocking or inventory buildup as in anticipation of the tariffs.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

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

[Benjamin Jackson, Senior Equity Research Associate at Jefferies]

Very clear. Thank you very much.

[Operator]

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

[Alastair Campbell, Institutional Equity Sales at RBC Capital Markets]

Hi there. Thanks very much for taking the questions today. And as I say, let me start with RUKENEF. 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?

[Alastair Campbell, Institutional Equity Sales at RBC Capital Markets]

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? Then finally, maybe turning to CVID, just a sense of the timeframe for those trials. Should Phase II trials be sufficient for approval?

[Alastair Campbell, Institutional Equity Sales at RBC Capital Markets]

And then how you think those trials likely to be scoped in terms of size and duration? Thank you.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

So I'll thank you so much for your question, Alastair. I'll start answering some of them and hand over to Steve and Aaron Reich for the last one on Tivid. I want to reinforce that actually there's not been much talking actually in Q4 and the result the very, very, very strong result that you saw actually on Reconnect were enlarged by enlarged actually driven by strong demand. So that should be very clear. When it comes to the positioning of RECONAF, 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 years after years since the launch of the drug more than ten years ago, we feel confident that RUCONNECT will remain a treatment of choice for HAE attacks despite new over entrants.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Certainly. The only thing I would add to that Alastair 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 inclusion 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 Rufinus has a place both in the short term and the long term and will continue to be a growth driver for farming.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

Thank you.

[Anurag Relan, Chief Medical Officer at Pharming Group]

And then Alastair, on the question about the CVID studies, we'll have some more details after we dosed the first patient, but this will be a Phase two 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.

[Operator]

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

[Joe Pantginis, Managing Director at H.C. Wainwright & Co.]

Hey, guys. Joe Pantginis from H. C. Wainwright. Thanks for taking the questions.

[Joe Pantginis, Managing Director at H.C. Wainwright & Co.]

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 RECONEST and building the new prescriber base in The U. S, 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

All right. Thank you, Joe, for your kind words. When it comes to ROCONEST, I've actually over the past few days, actually met a few RECONEST prescribers and so got a first hand experience of what's happening in Delhi medical practice. I think we see a growing an increased number of prescribers with RECONEST as they can see that the drug has a unique profile. And as a consequence, it has a unique value proposition for a specific segment of the patient.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

Those difficult to treat patients with who are experiencing actually a number of breakthrough attacks. And those doctors, I think, are reinforced by their experience 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Sure. Thank you, Fabrice. Hey Joe. I think to answer your question of the yes versus no dynamic, we're ten 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 RUKENESS because they've simply not had a patient or they've had their first one.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

So what we find especially with those physicians is they're very open to the concept of RUKENESS because they're now having to treat these severe frequently attacking patients 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 and we are very confident that more and more patients sorry, physicians will need to prescribe and will use and be open to it.

[Joe Pantginis, Managing Director at H.C. Wainwright & Co.]

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 rightsizing of the sales force, the impact of new assets or even further in licensing of assets to look forward to?

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

It's 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've heard today about the momentum that 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 Simon in the past. I think our success will come from great ambition, a relentless focus on execution, rigorous P and 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 accretive deals that could actually drive shareholder value in the mid and lower end.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

End. 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 interest of the organization.

[Joe Pantginis, Managing Director at H.C. Wainwright & Co.]

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

[Operator]

Thank you. We will now go to the next question. And the question comes from the line of Simon Skols from First Berlin. Please go ahead.

[Simon Scholes, Senior Analyst – Biotech and Resources at First Berlin]

Yes, good afternoon. Thanks for taking my questions. I've got two. Just to follow-up on the I believe costs, I was wondering if you could give us an indication of how those costs might evolve during 2026 and 2027. And then on CVIT, my understanding is that on PI3K delta you will require a Phase three.

[Simon Scholes, Senior Analyst – Biotech and Resources at First Berlin]

I was just wondering if you could give us an indication of why you might not require a Phase three on CVIT?

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

All right. So let me quickly answer your question about the spend on the Abliva program. So as I said, and you heard it from Jeroen, about $30,000,000 this year, $17,000,000 R and 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 $120,000,000 to $125,000,000 okay? Okay.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

So we do the deduction with the $30,000,000 that we're still in that boat, 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 it comes to the CVV, I'll let Anurag actually clarify

[Anurag Relan, Chief Medical Officer at Pharming Group]

perhaps some misunderstanding. Yes. Hi, Simon. So 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 three study as we've done with APDS. We're not anticipating these are rare diseases, so these are still relatively small programs.

[Anurag Relan, Chief Medical Officer at Pharming Group]

The Phase II program in the first PID with immune dysregulation is 12 patients. The CDID indication Phase II program will be slightly larger, but we still anticipate a Phase III requirement to enable registration. Of course, as the Phase two readout, we'll be able to tell you more what those Phase three look like, but that's our current plan.

[Simon Scholes, Senior Analyst – Biotech and Resources at First Berlin]

Okay. And just one last one. I mean, do you also expect CVID to get a FDA Fast Track designation at some stage?

[Anurag Relan, Chief Medical Officer at Pharming Group]

We will certainly look at all of those types of options. These are severe diseases. They have a similar course as APDS. There's early mortality associated with them. There's these are sick patients who have these conditions.

[Anurag Relan, Chief Medical Officer at Pharming Group]

So we do anticipate being able to work with the regulators to try to expedite the development.

[Simon Scholes, Senior Analyst – Biotech and Resources at First Berlin]

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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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 Farming. I believe that we are exiting 2024 with a very solid momentum, but they are very clearly identified growth opportunities ahead of us. And as per my answer to the question, our success will come from our ability to realize them, manage our P and L rigorously and maintain a very high level of ambition given clearly the growth prospects that we can have with our in line brands with Abliva.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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.

[Operator]

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

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[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

Thank you very much. Hello, everyone, and welcome to the Farming Full Year and Q4 twenty twenty four Financial Results Call. I'm Fabrice Shoaki, CEO of Farming, and I'll be joined on this call today by Steven Thor, our Chief Commercial Officer Anurag Rellan, our Chief Medical Officer and Jeroen Wachaman, our Chief Financial Officer. We will be making forward looking statements in this call that are based upon our current insights and plan. As you know, this may differ from future results.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

First of all, let me say that I'm really excited to be joining Pharmee, and it's an honor to succeed Simon de Vries. I'm passionate about progressing medical sciences and bringing innovation to patients. And naturally, I feel deeply connected with FARMING's mission to serve the unserved rare disease patients. Over the past twenty five years, I've developed an 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 worlds, the rigor and sophistication of Big Pharma and the value creation mindset and agility of venture capital and biotech.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

In light of this experience, I'm impressed with the development of farming over the past fifteen years and its significant growth prospects. Rugenest has become one of the cornerstone on demand treatments for HAE. Joenja is already approved and launched in The U. S. For the treatment of APDS with a second number of patients already on treatment and it is due to be launched in other key markets around the world.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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 pharma, 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 twenty twenty four revenues increased by 21% to $297,000,000 above our guidance range, including a strong fourth quarter and with operating profit and positive operating cash flow in the last two quarters of the year. Rouconest grew 11% to $252,000,000 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

I believe that given its unique profile and positive experience in difficult to treat patients, RECONEST 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 $45,000,000 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 U. S, 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 indication. Let me now hand over to Steve Dorr, our Chief Commercial Officer, who will give you a more granular perspective on the strong dynamic of RUKONET and Joininger.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Thank you, Fabrice. Good morning, everybody. As Fabrice just alluded to, we've delivered another strong performance in 2024. On RUCNEST, 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 eighty million dollars for the quarter.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

We ended 2024 with sales of $252,000,000 11 percent up on 2023. In the next two slides, I'll review why RUQUEENESS continues to show such strength in growth and while we're confident it will continue to grow in the years to come, even as the market becomes more competitive. On JOYENGIA, we continue to build our patient pipeline and transition eligible patients to paid therapy. And as you would expect, our team delivered significant growth over the first year of launch, ending Q4 '60 '5 percent up on prior year at $13,100,000 but for 2024 plus 147% of $45,000,000 Of note, in addition to 96 patients plus five pending on paid therapy in The U. S, 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.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

In the forthcoming slides, I'll also outline the opportunities we see in the coming months and years that will both build the Joininger business for APDS and with new potential indications for the molecule create a strong high growth franchise. Looking first at RUCNEST, as I just stated RUCNEST is and will continue to be a growth driver for farming and an important treatment option for U. S. Patients and their doctors, which is why it's already the second most prescribed acute product in The U. S.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

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. C1s arrays inhibition represented in the graphic by the red C1INH 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 RUKENESS since it works comprehensively across all these systems. C1S2A's 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 ninety seven percent attack resolution in a single dose and a sustained response with ninety three percent of patients attacks stopped for at least three days.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

So let's look more specifically at RUKENESS patients and what this means for them. The first thing to note is that RUKENESS serves all patient types, those being Type one, Type two and normal C1 patients. All three of these RUKENESS 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 acalepan or are having to redose to stop their HAE attack. In the photos on the slide, you could see an actual RUKENESS patient at the start of an attack and then her recovery as it resolves at the four hour mark and the twenty four hour mark.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

For patients like this one suffering with a more severe course of disease attacking frequently and happen to redose another therapies, knowing as I just stated that ninety seven percent of patients will stop their attack with a single dose and almost all of them will be attack free for at least three days is a very big deal. RUKENESS efficacy reliability allows our patients to better control and plan their lives and that's why RUKENESS will continue to have a strong position in The U. S. Acute market and remain an important product for our company in the years to come. I'll transition now to Joininger, which as you're aware was launched in The U.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

S. In March 2023 and is available outside The U. S. Through various access programs. We see a number of opportunities for Joininger, which I'll walk you through now.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Our pharming's patient finding efforts are continuing as we build our patient pipeline in The U. S. And globally. In fact, we've already identified over two forty patients in The U. S.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Of whom forty percent are already on pay 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 addressable population. So what are they? Looking at the second block on the slide, the first is the outputs from the BUS resolution program, which Anurag will discuss. That will deliver another bonus of APDS patients available for treatment this year and beyond.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

The second will be the pediatric indication launched in The U. S. Which is expected in 2026. We currently have over 60 patients in our U. S.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Pipeline and growing and they begin transitioning to Joenga as soon as the indication is approved. And 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. And then we have further anticipated launches in other important markets including Japan, Germany, France, Italy, Spain, Canada and Australia.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

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, lineolacifor APDS is only part of the story. As you know, Phase two trials have been initiated for two bigger indications, LPID and CVID. In fact, CVID was still rare with a prevalence of forty patients per million transitions lenovoCID from a small ultra rare disease molecule to one with blockbuster sales potential, thereby creating the lenovoCID 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 Gwelyn, 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.

[Anurag Relan, Chief Medical Officer at Pharming Group]

And here we can see our growing pipeline. For someone who's been at pharma for many years, it is incredibly impressive to see how this has expanded from only RUCINUS for HAE to now include multiple products and indication, which can support the vision you heard Fabrice layout that we have for Martin. 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 two studies underway, including a new program in CDID 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 Ovelina. Before turning to development, as you know, The U.

[Anurag Relan, Chief Medical Officer at Pharming Group]

S. 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 the variant is novel and there isn't enough information to determine if the variant is disease causing. In fact, there are over twelve hundred patients in The U.

[Anurag Relan, Chief Medical Officer at Pharming Group]

S. Alone who have received the VUS result in the genes associated with APES. 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.

[Anurag Relan, Chief Medical Officer at Pharming Group]

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 twenty five percent 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 four to 11.

[Anurag Relan, Chief Medical Officer at Pharming Group]

The study demonstrated that lenulocyte 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 lineolysis 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.

[Anurag Relan, Chief Medical Officer at Pharming Group]

We are announcing today the second program in CDID, 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 prevalent estimates how the new study we announced today in CVID significantly increases the patient population that could potentially benefit from linealcin. Across APDS and these new programs, the central role of PI3K delta in lymphocytes is clear in driving the immune dysregulation and supports the investigation of linealcin.

[Anurag Relan, Chief Medical Officer at Pharming Group]

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 LENULISID to more APDS patients in several key markets across the world and expand the addressable population. As you heard from Steve, bringing Lenolusiv to additional APDS patients represents a significant near term opportunity. And in Europe, we have already included the clinical benefit and safety of Lenolusiv, 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 twenty twenty six. 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, Chief Medical Officer at Pharming Group]

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. And you heard me already talk about the pediatric study in the four to eleven 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 PID indications, which support the long term growth of linealism. And now turning to our third program in our portfolio via the just completed I believe that acquisition TALE1333. 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, Chief Medical Officer at Pharming Group]

These disorders can have array of diverse clinical features, but common elements 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 U. S. 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 plus to NADH ratio, which is abnormally low in these patients.

[Anurag Relan, Chief Medical Officer at Pharming Group]

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. 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. 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.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

Thank you very much Anurag. Q4 was a very robust quarter for pharma. Revenues grew by 14% versus a very strong fourth quarter in 2023. Rupenesh growth was 9% and Joenzi at 66%. Gross profit grew by nine percent and that is lower than the revenue growth and that was driven by a one off inventory impairment following a reduction, a merchandise production issue at one of our CNOs.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

The OpEx was fairly stable and largely as expected in the quarter. The small increase was caused by one off costs including a full impairment of a lease contract and just over NOK1 million of ABLIVA acquisition costs. Operating profit increased by $5,600,000 driven by higher gross profit as a consequence of strong sales and active OpEx management. This was a second quarter in a row that we generated operating profit. And the net results went from a loss in Q4 twenty twenty three to a net profit in Q4 twenty twenty four.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

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 Rubenest with a strong growth of 11% and LENIALOSIP that grew by 147%.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

OpEx increased by 10% due to investments in Duengya which is at a rate well below the revenue increase. Operating profit on a like for like basis improves and that's when taking out the big one offs that we have 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 Ibeliva acquisition process and timeline. The 66,100,000.0 acquisition of Ibeliva is now completed and Abliva shares approved for delisting next week.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

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 million and $335,000,000 which means a growth rate of 6% to 13%. And the assumption underlying the guidance midpoint is a high single digit LucaNest growth and continued strong growth of Duenja with an acceleration in the second half of the year from the positive impact from BU assets as Anurag mentioned before.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

For Duenja, we expect continued growth of patients on paid therapy and The U. S. Pricing at an annual WACC of USD594000. 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.

[Jeroen Wakkerman, Chief Financial Officer at Pharming Group]

And our preliminary estimate for I believe related OpEx is $30,000,000 for 2025 including $17,000,000 R and 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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 GOINGER 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

First, the potential new indication of PID with immune dysregulation is a great opportunity to continue to expand Joininger's 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 $315,000,000 to $335,000,000 illustrates our momentum and obviously we look forward to updating you on our progress. Let me now open the line for questions.

[Operator]

Thank you.

[Jeff Jones, Managing Director and Senior Analyst at Oppenheimer & Co. Inc.]

And Fabrice, welcome to the team and congratulations and on a fantastic first earnings update here. I guess two questions for us. First with respect to KL-one 33 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 a clarification for our second question, for the one hundred and eighty eight patients you mentioned that are on the expanded access program, which includes clinical trials, are any of those patients paid?

[Jeff Jones, Managing Director and Senior Analyst at Oppenheimer & Co. Inc.]

And perhaps could you provide some breakdown in terms of the territories where those patients are? Thanks.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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.

[Anurag Relan, Chief Medical Officer at Pharming Group]

Hi, Jeff. So with respect to primary mitochondrial diseases, when we talk about the thirty thousand number of patients that are in The U. S. And the large European markets, this actually is 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 eighty percent of all PMDs and then we broke that down further and looked at the mutations that are specifically being enrolled in this study.

[Anurag Relan, Chief Medical Officer at Pharming Group]

So that's how we came up with this estimate of thirty thousand. So in essence, all of those thirty thousand are the addressable population.

[Jeff Jones, Managing Director and Senior Analyst at Oppenheimer & Co. Inc.]

Appreciate that. Thank you.

[Anurag Relan, Chief Medical Officer at Pharming Group]

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

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Hi, Jeff. Good morning. So of the one hundred and eighty eight, 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 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 a revenue line that we would discuss publicly.

[Jeff Jones, Managing Director and Senior Analyst at Oppenheimer & Co. Inc.]

Appreciate that guys. Thank you.

[Operator]

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

[Benjamin Jackson, Senior Equity Research Associate at Jefferies]

Great. Thanks for the question. Just two for me. If we first start on one that's not too exciting. So the $30,000,000 anticipated additional OpEx from the Blivaraf acquisition, are you able just to touch on how much of that is recurring?

[Benjamin Jackson, Senior Equity Research Associate at Jefferies]

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 U. S. Tariffs to apply to drugs? Again, at this point that it's very unclear what's going to happen and we don't necessarily know where it's going.

[Benjamin Jackson, Senior Equity Research Associate at Jefferies]

And then I guess as a result of that, have 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

Thank you, Ben. So let me take your first question about the $30,000,000 of OpEx spend on Abliva in 2025 as announced by Jeroen. About SEK 17,000,000 will be R and D and the rest will be nonrecurring transaction and integration costs. Now 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 U. S.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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 are monitoring very, very closely.

[Benjamin Jackson, Senior Equity Research Associate at Jefferies]

Perfect. Thank you.

[Anurag Relan, Chief Medical Officer at Pharming Group]

I think there was a question on whether there's any stocking or inventory buildup as in anticipation of the tariffs.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

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

[Benjamin Jackson, Senior Equity Research Associate at Jefferies]

Very clear. Thank you very much.

[Operator]

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

[Alastair Campbell, Institutional Equity Sales at RBC Capital Markets]

Hi there. Thanks very much for taking the questions today. And as I say, let me start with RUKENEF. 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?

[Alastair Campbell, Institutional Equity Sales at RBC Capital Markets]

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? Then finally, maybe turning to CVID, just a sense of the timeframe for those trials. Should Phase II trials be sufficient for approval?

[Alastair Campbell, Institutional Equity Sales at RBC Capital Markets]

And then how you think those trials likely to be scoped in terms of size and duration? Thank you.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

So I'll thank you so much for your question, Alastair. I'll start answering some of them and hand over to Steve and Aaron Reich for the last one on Tivid. I want to reinforce that actually there's not been much talking actually in Q4 and the result the very, very, very strong result that you saw actually on RECONEST were enlarged by enlarged actually driven by strong demand. So that should be very clear. When it comes to the positioning of Obrichonez, 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 years after years since the launch of the drug more than ten years ago, we feel confident that RUCONNECT will remain a treatment of choice for HAE attacks despite new over entrants.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Certainly. The only thing I would add to that Alastair 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 inclusion 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 Rufinus has a place both in the short term and the long term and will continue to be a growth driver for farming.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

Thank you.

[Anurag Relan, Chief Medical Officer at Pharming Group]

And then Alastair, on the question about the CVID studies, we'll have some more details after we dosed the first patient, but this will be a Phase two 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.

[Operator]

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

[Joe Pantginis, Managing Director at H.C. Wainwright & Co.]

Hey, guys. Joe Pantginis from H. C. Wainwright. Thanks for taking the questions.

[Joe Pantginis, Managing Director at H.C. Wainwright & Co.]

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 RECONEST and building the new prescriber base in The U. S, 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

All right. Thank you, Joe, for your kind words. When it comes to RUKONEST, I've actually over the past few days, actually met a few RECONEST prescribers and so got a first hand experience of what's happening in Delhi medical practice. I think we see a growing an increased number of prescribers with RECONEST as they can see that the drug has a unique profile. And as a consequence, it has a unique value proposition for a specific segment of the patient.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

Those difficult to treat patients with who are experiencing actually a number of breakthrough attacks. And those doctors, I think, are reinforced by their experience 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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

[Stephen Toor, Chief Commercial Officer at Pharming Group]

Sure. Thank you, Fabrice. Hey Joe. I think to answer your question of the yes versus no dynamic, we're ten 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 RUKENESS because they've simply not had a patient or they've had their first one.

[Stephen Toor, Chief Commercial Officer at Pharming Group]

So what we find especially with those physicians is they're very open to the concept of RUKENESS because they're now having to treat these severe frequently attacking patients 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 and we are very confident that more and more patients sorry, physicians will need to prescribe and will use and be open to it.

[Joe Pantginis, Managing Director at H.C. Wainwright & Co.]

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 rightsizing of the sales force, the impact of new assets or even further in licensing of assets to look forward to?

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

It's 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 heard today about the momentum that 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 Simon in the past. I think our success will come from great ambition, a relentless focus on execution, rigorous P and 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 accretive deals that could actually drive shareholder value in the mid and lower.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

End. 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 interest of the audience.

[Joe Pantginis, Managing Director at H.C. Wainwright & Co.]

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

[Operator]

Thank you. We will now go to the next question. And the question comes from the line of Simon Skols from First Berlin. Please go ahead.

[Simon Scholes, Senior Analyst – Biotech and Resources at First Berlin]

Yes, good afternoon. Thanks for taking my questions. I've got two. Just to follow-up on the I believe costs, I was wondering if you could give us an indication of how those costs might evolve during 2026 and 2027. And then on CVIT, my understanding is that on PI3K delta you will require a Phase three.

[Simon Scholes, Senior Analyst – Biotech and Resources at First Berlin]

I was just wondering if you could give us an indication of why you might not require a Phase three on CVIT?

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

All right. So let me quickly answer your question about the spend on the Abliva program. So as I said, and you heard it from Jeroen, about $30,000,000 this year, $17,000,000 R and 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 $120,000,000 to $125,000,000 okay?

[Simon Scholes, Senior Analyst – Biotech and Resources at First Berlin]

Okay.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

So due to deduction with the $30,000,000 that we're still in that boat, 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 it comes to the CVV, I'll let Anurag actually clarify perhaps

[Anurag Relan, Chief Medical Officer at Pharming Group]

some misunderstanding. Yes. Hi, Simon. So 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 three study as we've done with APDS. We're not anticipating these are rare diseases, so these are still relatively small programs.

[Anurag Relan, Chief Medical Officer at Pharming Group]

The Phase two program in the first PID with immune dysregulation is 12 patients. The CDID indication Phase two program will be slightly larger, but we still anticipate a Phase three requirement to enable registration. Of course, as the Phase two readout, we'll be able to tell you more what those Phase three look like, but that's our current plan.

[Simon Scholes, Senior Analyst – Biotech and Resources at First Berlin]

Okay. And just one last one. I mean, do you also expect CVID to get a FDA Fast Track designation at some stage?

[Anurag Relan, Chief Medical Officer at Pharming Group]

We will certainly look at all of those types of options. These are severe diseases. They have a similar course as APDS. There's early mortality associated with them. There's these are sick patients who have these conditions.

[Anurag Relan, Chief Medical Officer at Pharming Group]

So we do anticipate being able to work with the regulators to try to expedite the development.

[Simon Scholes, Senior Analyst – Biotech and Resources at First Berlin]

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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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 Farming. I believe that we are exiting 2024 with a very solid momentum, but they are very clearly identified growth opportunities ahead of us. And as per my answer to the question, our success will come from our ability to realize them, manage our P and 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.

[Fabrice Chouraqui, Chief Executive Officer and Executive Director at Pharming Group]

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.

Participants

Executives

• Fabrice Chouraqui, Chief Executive Officer and Executive Director
• Stephen Toor, Chief Commercial Officer
• Anurag Relan, Chief Medical Officer
• Jeroen Wakkerman, Chief Financial Officer

Analysts

• Jeff Jones, Managing Director and Senior Analyst at Oppenheimer & Co. Inc.
• Benjamin Jackson, Senior Equity Research Associate at Jefferies
• Alastair Campbell, Institutional Equity Sales at RBC Capital Markets
• Joe Pantginis, Managing Director at H.C. Wainwright & Co.
• Simon Scholes, Senior Analyst – Biotech and Resources at First Berlin