Pharming Group Q2 2025 Earnings Call Transcript

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Provided by Quartr
July 31, 2025

Key Takeaways

  • Positive Sentiment: Farming reported Q2 revenues up 26% year-on-year and an operating profit of $12.9 million compared to a loss in Q2 2024.
  • Positive Sentiment: RUCONEST sales jumped 28% in Q2 with an average of 21 new prescribers per quarter, underscoring its strong position in acute HAE.
  • Positive Sentiment: JOENGA patient count grew by 18 in H1 2025—exceeding all of 2024—and the product has now launched in the UK, with reclassification of VUS cases and pediatric expansion set to drive further uptake.
  • Positive Sentiment: A Cell-published study identified over 100 new PI3Kδ-activating variants, suggesting ~20% of 1,400 US VUS patients may be reclassified as APDS and revealing a potential prevalence up to 100× greater.
  • Positive Sentiment: Management raised 2025 revenue guidance to $335–350 million, is advancing phase II programs in PIDs and the KL1333 registrational trial, and holds $130.8 million in cash to fund its pipeline.
AI Generated. May Contain Errors.
Earnings Conference Call
Pharming Group Q2 2025
00:00 / 00:00

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Operator

Good day, and thank you for standing by. Welcome to the Farming Q2 and First Half twenty twenty five Financial 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. Please be advised that today's conference is being recorded.

Operator

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

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

Thank you, operator, and good morning or good afternoon, everyone, and and welcome to our q two twenty twenty five earning call. So I'm Fabrice Shiraki, the CEO of Pharming, and I'll be joined on this call today by Steve Thor, our chief commercial officer, and Anurag Grellin, our chief medical officer. Next slide. So on this call, we will be making forward looking statements that are based upon our current insights and plan. As you know, these may differ from future results.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

Next slide. Next slide. So as you saw in our press release earlier today, farming delivered a very strong quarter. Total revenues grew by 26% in the 2025 versus the same quarter last year, and we delivered meaningful operating profit of $12,900,000 compared to a loss in the previous year. This number excludes about $2,000,000 in non recurring Abliva acquisition related expenses.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

Our strong top line growth was fueled by the continued significant growth of Reconnect, 28% year on year, and the further acceleration of patient uptake on Joentra, with the increase of patients on drug in the 2025 already surpassing the total increase for all of 2024. This strong momentum for our two commercial assets support an upgrade to our full year 2025 revenue guidance, for which I'll provide more details later in the call. Next slide. Before we go into detail on our financial results and obviously on our recent regulatory and clinical development progress, I'd like to say that our results in the 2025 are a good illustration of the solid growth foundation that we've built. Over the past two years, farming has evolved from being a one asset company to having two fast growing commercial products and a high value late stage pipeline with two assets with over $1,000,000,000 potential each.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

ReConnect continues to grow double digits after ten years on the market. With its unique value proposition for HAE patients who experience more frequent and stronger attacks, and its very specialized manufacturing process, Ruconext is well positioned to remain a foundational drug to finance the growth of our portfolio and pipeline. Joynja is only at the very beginning of its life cycle. It is the only DMT for APDS, and in fact, it is the only therapy specifically approved for APDS. Joynja has several key short term growth driver in this indication with the reclassification of The US patients, the pediatric expansion, and the launch in key market.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

And the possible much higher prevalence of APDS, as suggested by the June publication in cell, provide, in my opinion, a significant upside. The ongoing development of jointia in two larger indications for genetically defined PIDs with immune dysregulation as well as for CVID has the opportunity to propel the brand to a whole new level. And finally, KL one triple three from the acquisition of Abliva, is another high value late stage development asset which has already successfully passed an interim analysis in a registrational trial. So this unique combination of commercial and pipeline assets is very much the the key reason why I joined farming six months ago as I see the opportunity for significant value creation in the near term as well as in the long term. Next slide.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

I also see great opportunity to build a leading global rare disease company by leveraging the strong rare disease capability platform that we have built over the years, and that has yielded to this strong performance. And I must say that I'm extremely pleased to see the excitement and the commitment of our employees to realize this vision. So let me now turn to Steve Thor, who will provide you with more detailed information on the continued growth of RUCONESS and the further acceleration of joint GI patient uptake in APDS. Over to you, Steve.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Thank you, Fabrice. Morning, everybody. Next slide, please. So as Fabrice has said, RUCONESS delivered a strong performance with double digit revenue growth of 28%, taking us to a revenue of $80,400,000 in Q2 of this year. And as you can see on this slide, this is being driven by the ongoing growth in prescribers.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Over the years since launch back in 2015, we have consistently added new RUCONESS prescribers as they recognize the value RUCONESS brings to patients suffering with moderate to severe hereditary angioedema. In fact, we've added an average of 21 new prescribers in each of the past six quarters, and this leads directly to the continuing increase in new patient enrollments, which obviously translates to regular total volume increase over prior year, which is twenty seven percent in The US. In part, the reason for this is RUKINESS has a unique profile in the acute on demand HAE market, which makes it an important treatment option for moderate to severe patients who experience more frequent attacks. It is this differentiation in the acute segment of the market that explains the strong momentum that RootConnect continues to have and the growth prospects we have for RootConnect over the long term. Next slide, please.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Now as I've said in, you know, just now and in other calls, in Ruchiness, we have a highly effective product that serves all patients within the HAE spectrum. So that's type one, type two, and normal C1. And what all three groups have in common is they all suffer from moderate to severe debilitating attacks and they have them frequently. They also have typically failed other single pathway specific targeted acute therapies such as acataban, which have either not been sufficiently effective or often the case, also at least a re dosing. In the photos on this slide, you can see an actual RUKINS patient at the start of the attack and then a recovery as it resolves at the four hour mark and then the twenty four hour mark.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

And this is exactly the type of patient I mean with a more severe course of disease attacking frequently and having to redose on other therapies. If you're an patient with this disease profile, having RUCONEST on hand, delivering complete resolution in a single dose for ninety seven percent of attacks, with half getting complete attack resolution within four and a half hours and the vast majority within twenty four hours, RUCONEST is both critical and reassuring. In fact, I recently attended along with other staff members farming the HAEA Summit in Baltimore, which over 1,400 patients attended. And this is regards to RUCONEST as a sentiment I repeatedly heard. And it's for these reasons that RUCONEST will continue to have a strong position in The U.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

S. Acute market and will remain an important product for our company in the years to come. Next slide, please. So switching gears now to Joenga and the Joenga launch, backing from 2023. As with Ruchaness, we've delivered a strong double digit revenue growth quarter on quarter of 15% with 12.8% in net revenue sorry, 12,800,000.0 in net revenue.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

We also achieved a further acceleration in patients on paid therapy in Q2 with a 12 patient increase doubling the six patients we increased in Q1. And importantly, as you can see in this graph, that increase of 18 patients in the first half of this year exceeded the total increase for all of 2024. And these results reflect the excellent patient finding and conversion capabilities of our commercial team. Importantly, we've also launched JOENGRA in The UK in April, and I'm pleased to report that the first patients are now on commercial therapy. Now this is an important step as we execute our focused geographic expansion plans, having already identified over 900 patients of whom one hundred and eighty five are being treated through various access programs.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Next slide, please. So importantly, we expect to sustain that acceleration with a number of growth catalysts to come. We have the conversion of patients already in the funnel, already found and identified. As Fabrice mentioned, we have the reclassification of VUS patients in The US starting in the second half of this year. Now, we already know fourteen hundred such cases right now, and we ultimately expect twenty percent of those patients at least to be diagnosed with APDS.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

We also have the pediatric label expansion, and we expect U. S. Approval in the second half of next year. And we already have fifty patients identified between the ages of four and 11 who will be eligible for treatment, and many of those are already on therapy in various types of access programs. And of course, we will continue to identify more and more pediatric patients in the coming months and years.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

And finally, as I mentioned, we have launches in other major markets where we will achieve appropriate pricing and reimbursement, with forthcoming approvals expected in Japan, the European Union and Canada over the next twelve months. And this, of course, reflects the significant progress for Joengia in the APDS indication that we've made in the past two years. Next slide, please. Now all that said, Joengia is still in the early stage of its life cycle, which can be seen on this slide. In APDS today, we're focused on approximately five hundred patients in The U.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

S. Alone. With the VUS reclassifications I mentioned earlier, that represents an important opportunity to expand that patient pool of 500. In fact, in the paper that Fabrice mentioned published himself that Anurag will expand upon, it suggests a much higher prevalence. So all of this could totally change how we think about APDS.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

And then as you look at this slide, in addition to APDS, we have phase two clinical programs ongoing for new primary immune deficiencies indications. The first PID with immune dysregulation into PI3K delta signaling where the patient pool is at least 2,500, so five times that of APDS. And a CVID with immune dysregulation with a patient pool of at least 13,000. So these US patient numbers shown here highlight the significant global opportunity we actually have in our hands today to expand the Joenga franchise in APDS and with these new indications. And this will enable farming to deliver on its mission to serve unserved rare disease patients and to build on our existing commercial success potentially delivering a $1,000,000,000 Joenga franchise.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

So I'd like now to hand over to Anurag Grellin, our Chief Medical Officer, to discuss these opportunities and our pipeline in more detail.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

Thanks, Steve. Next slide, please. As you've been hearing, we have supported work to help patients who have received a VUS or variant uncertain significance lab test result. Again, these are patients who have had symptoms of a primary immune deficiency, but there was not enough information available to determine if their genetic variant in their genes related to APDS was disease causing. Here, I'm very excited to recap the details from a new study published last month that expands this understanding of APDS.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

This work published in Cell found more than 100 new variants that lead to PI3K delta hyperactivity. This has two important implications. First, as shown on the left, the data from the study will be used by genetic testing labs to reevaluate variants that were previously thought to be a VUS. But now with this new information, patients could be reclassified as having APDS. In addition, we are planning to expand these studies further to generate and evaluate even more variants to help more patients in the future.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

With these data, we expect that twenty percent of the more than fourteen hundred patients in The US who have received a VUS test result could ultimately be reclassified as APDS. On the right, you see the other key finding from the study, that the new variants found suggest a much higher prevalence of APDS, perhaps even 100 times greater. The early work from the study also implies a broader clinical phenotype or clinical symptoms for patients with such hyperactive variants. We are now planning additional work here to understand these clinical aspects, but also to get a better sense of what this greater prevalence could be. So much more to come on this, including in the near term.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

Next slide, please. In addition to this exciting new science leading to growth opportunities, we continue to make progress on our pipeline, having achieved a number of key milestones during the quarter. First, to expand the addressable population for APDS, I'm pleased to report that in June, we filed our regulatory application for Joenja in Japan for APDS patients ages four to four years old and older. In addition, in fact, today, we intend to file our application with FDA for label expansion for pediatrics for children ages four to 11. For the two phase two proof of concept studies ongoing in primary immune deficiencies with immune dysregulation, these studies remain on track to read out in the second half of next year.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

Again, there's strong scientific rationale here, and we have even seen some early encouraging signs with the compassionate use in this group. These populations represent more than 20 times larger group of patients, as you heard from Steve, that we have currently in APDS. So the unmet need here is significant. Lastly, we have made significant progress with restarting the registrational study for KL-one triple three, our new asset for primary mitochondrial disease. As I mentioned in the past, this pivotal study has already undergone a positive futility analysis, and we have reactivated now previous sites and opened new ones as well.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

With several patients now having been randomized and dosed. We continue to expect a 2027 readout for this important program. Next slide. Let's zoom out now and look at our pipeline. Having been at Farming for quite a number of years, it is truly impressive for me to see this dramatic transformation for the company from even just a couple of years ago.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

We have two marketed products, and we're bringing these to more patients with geographic expansion and pediatrics. On top of that, we have a growing pipeline with two proof of concept studies in the KL pivotal program, both serious diseases and significant patient populations. All in all, quite a lot to be excited about. I'll turn it back to Fabrice now to review our financials and outlook for the rest of the year.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

Thank you, Anurag. So as you've seen, we had another strong quarter with revenues up 26% versus last year. Gross profit increased by 27% to $84,200,000 mainly due to the increase in the revenues. The operating profit jumped to $12,900,000 compared to an operating loss of $3,100,000 in the second quarter of last year. This number excludes $2,100,000 of non recurring Abliva acquisition related expenses.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

Excluding those non recurring expenses only, our total operating expenses did not increase significantly. Cash and marketable securities increased from $108,900,000 at the end of the 2025 to $130,800,000 at the end of the 2020 And this meaningful increase was primarily driven by the net cash flows that we have generated from our operating activities. Next slide. When we look at the first six months of the year, clearly I believe that the financials show the consistent strong execution of our strategy. Total revenues increased by 33% and gross profit increased by 37% compared to the 2024.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

The increase in opex was mostly driven by $15,000,000 of ABLEVA related expenses, of which $9,900,000 are non recurring in nature. The operating profit, excluding non recurring Abiliva acquisition related expense, amounted to $13,700,000 compared to a loss of $1,200,000 in 2024. Cash and marketable securities decreased by $38,600,000 to $130,800,000 primarily driven by the $66,000,000 acquisition of Abliva shares and the $9,900,000 in non recurring Abliva acquisition related expenses. And this was partially compensated by the cash flows that we have generated in Q1 and Q2. Next slide.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

So on the back of the strong Q2 results and the outlook for the remainder of the year, we are raising our 2025 total revenue guidance to between $335,000,000 to $350,000,000 and this is up from prior guidance between $325,000,000 and $340,000,000 This new guidance implies full year revenue growth between 1318%. We expect total operating expenses for 2025 to be between $3.00 4,000,000 and $3.00 $8,000,000 which is slightly above the prior guidance for flat opex plus ABLIVA related expenses, which I've shared at the last quarter, which was around $3.00 $3,000,000 This small variances in our expense projection is largely due to the $5,300,000 negative impact of the higher euro dollar exchange rate. And obviously we are striving hard to save a portion of this increase, while not impacting actually the growth momentum. On that note, we're making very good progress in the development of the plan to ensure a sustainable 15% cut of G and A expenses that I announced last quarter to optimize capital allocation to grow our business. I'm also pleased to let you know that we are making good progress in the recruitment of our new CFO, and I hope to be in a position to make an announcement in the not too distant future.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

So finally, I think it's important to bear in mind that we continue to expect that our available cash and future cash flows will cover our current pipeline and pre launch costs. I believe actually that is actually putting farming in a very unique position in the biotech environment. Next slide. So in summary, I can only say that we have delivered a very strong quarter in the first half due to the strong performance of RUCONESS and JOINGR. With its unique profile, strong patient experience and specialized manufacturing process, I really believe that RUCONNECT is well positioned to remain for the foreseeable future a treatment of choice for HAE attacks.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

In APDS, we are accelerating the pace of enrollment of new patients ahead of the start of the reclassification of BUS patients in H2, ahead of the expected pediatric label expansion next year, and ahead of the geographic expansion in eight countries. As you've heard from Steve, we've already launched Joenga in The UK, where actually the first signal are very, very encouraging. Our pipeline is progressing at pace with the objective to generate two blockbuster assets. So as you can see, are building a solid platform for sustainable growth and value creation with a series of clear catalysts in the short and near term. With that, let me now open the line for questions.

Operator

Thank We will now take the first question from the line of Sushila Hernandez from Van Nanskot Kempen.

Sushila Hernandez
Equity Research Analyst at Van Lanschot Kempen Investment Banking

Two from my side on Joenda. So on the VUS patient reclassification, how do you expect to see this translate on new patients on paid therapy? So how fast will twenty percent of these fourteen hundred patients get on paid therapy, and what are the bottlenecks? Thank you.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

I'll Sushiya, thank you so much for your question. I'll start answering your question, and I'll hand over to Anurag. So clearly, this reclassification of BUS patients into APDS will happen over time. As we've said, we expect about twenty percent of all The US patients identified to ultimately be reclassified as APDS. So it's a it's a significant opportunity, which will clearly expand significantly the the total addressable patient populations.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

What's clear is that this reclassification will stop in the second half now that diagnostic labs, which have already tested those patients, will be able to use the data in the self publication and see which of those The US patients should now be reclassified as a PDF. They'll then contact the doctors to inform of the change act of this change. So we expect to see a number of patients in the second half of the year to be reclassified as APDS. But this opportunity, obviously, will take some time to be fully captured. And so we should see VUS, the VUS reclassification, as an additional opportunity on top of adult APDS, and tomorrow on top of the potential label expansion to pediatric to fuel the growth of joint venture.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

Anurag? I think there's a couple other points to Number one, the growth in the VUS patients, So this is an ongoing problem. You've seen that number grow consistently as more patients get tested. The pool of VUS patients continue to increase. So I think that recognizes the scope and scale of the problem.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

The second is we have some experience already here, with VUS patients being reclassified. So, of course, we're doing it now, and through this work supported at Columbia, this has been done at a large scale. But we have some experience doing this one by one where patients were tested, individually, and they had their, functional tests showing hyperactivity, and that was used then to reclassify them and then eventually get them on to JoAnja. So we know how that process works, and we know that once they're reclassified as APDS, they can be quickly reimbursed, to get on to Joenga therapy, if the doctor desires. So we we do know where how this process works, and we have a good understanding, as Faris outlined, of what the next steps are, in terms of the reclassification, but then also what the steps are in eventually to get these patients on therapy.

Sushila Hernandez
Equity Research Analyst at Van Lanschot Kempen Investment Banking

K. Thank you. And one more question, if I may. So of these 185 APDF stations globally, could you break this down for the ones that are in The UK and Japan?

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

We we don't give actually the breakdown in the in the various countries at this stage, Sushila.

Sushila Hernandez
Equity Research Analyst at Van Lanschot Kempen Investment Banking

Okay. That's clear. Thank you.

Operator

Thank you. We will now take the next question from the line of Jeff Jones from Oppenheimer. Please go ahead.

Jeff Jones
Managing Director - Senior Analyst at Oppenheimer & Co. Inc.

Thank you, guys, and congrats on a really strong quarter. Two questions from us, one on Juwensha, one on RUKEMEST, if we could. On Juwensha, you highlighted the growth in patients on therapy in the first half of this year and really nice growth. But if we look at revenue for Joenshin, the first half of this year versus the last half, the last half of last year, revenues are actually down just slightly. So can you comment on the conversion of patients on therapy to revenue, and that lag that we see here between the last '24 and the '25?

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

Absolutely. I mean, this is an excellent question. There is there's not been any any change in the very high conversion conversion rates. Alright? Actually, what you've highlighted is due to an increase in in stock inventory in q two last year, which actually is making this this growth lower. So it's just inventory management. That's it.

Jeff Jones
Managing Director - Senior Analyst at Oppenheimer & Co. Inc.

Okay. That's helpful. And on Ruchinest, we've obviously just seen the approval of cepotralstat, and it's getting ready to launch. So we know these are targeting somewhat different populations, and you've highlighted the strong efficacy of rucinest as well as its use in patients who failed other therapies. Are there segments of the rucinest patient population that you feel are at risk from the sevitralstat launch? And how should we be thinking about that impact?

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

So the as as, Steve said, and I'll let him speak in a in a minute, the vast majority of ReConest patients are patients who have failed other treatments. There are more severe patients, who have more frequent crisis, more severe crisis, and they've not been able to be controlled appropriately with with other treatments. So that's actually why they need actually an IV formulation with the characteristic that you know and the efficacy level that Steve reinforced to be controlled. So there is really hardly any specific patients that are more prone to switch. And those patients know, mean, based on what and again, we've met many of them in in Baltimore, as Steve as Steve said, actually, many many of them actually knows how long it took them actually to find the right the right drug.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

There may be a few mutations that that are treated by RUCONNEST. This one actually may may switch. I would be surprised to prepare because in some instances, let's not forget that Rucinex is an IV drug. There's been for years, subcu treatments, and so I'm I'm sure that, my patient would have preferred to be treated with, subcu treatment and also generic treatment. Keith?

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Thank you, Fabrice. Hi, Jeff. I'll build very briefly on on what Fabrice said. I think you hit the key points. But the important thing here is that the majority of our patients have experienced pretty an unpleasant course of disease that's often led to them being hospitalized, etcetera, etcetera.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

And they're they're all the cataban failures for the most part. Now it's important to note, Jeff, that the cataban failures were actually excluded from the sibotrelstat pivotal trial. So we really are targeting quite different patient populations. And probably the key the key numbers to remember here are, you know, with rucinase, ninety seven percent of attacks are resolved in a single dose with half of them within four and a half hours. So if you're this type of patient that's experienced this course of disease, those are really key numbers.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

So I would never sit here and suggest there won't be disruption in the market. There always is some with where launches are concerned. But we're confident that our patient population is well served by Rukones and continue to be because, as I said, these are not the same types of patients that these two drugs will be treating.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

And what's interesting to note as well is that despite actually the upcoming launch of selvotristat, we're seeing more prescribers willing to use RECONEST, more EureCONEST patients on the drug, and that's not the typical pattern, you know. If doctors felt that a new drug will be better for the patient, they would actually warehouse those those patients. So clearly, we are very encouraged by by seeing the trend, and that's reinforced the distinctive value proposition of Rucinect.

Jeff Jones
Managing Director - Senior Analyst at Oppenheimer & Co. Inc.

Great. Appreciate it very much, guys. Thank you.

Operator

Thank you. We will now take the next question from the line of Benjamin Jackson from Jefferies. Please go ahead.

Ben Jackson
Ben Jackson
Equity Research Senior Associate at Jefferies

Brilliant. Thanks for the question. Ben Jackson, Jefferies here. Just two on JoAnja from my side, please. The first thing, can I just push you further on this VUS uptake in the second half of the year and follow-up with what the first question on the Q and A was?

Ben Jackson
Ben Jackson
Equity Research Senior Associate at Jefferies

And specifically, what is the bottleneck here? Because I appreciate that it's kind of a little bit out of your hands with regards to reclassification, but you're talking about the fact that you've got experience with reclassification. If the doctors approve it, it's quickly reimbursed, you very rarely see rejections of reimbursement. So what is it that's keeping you on the more cautious side of the rate of up uptake? Is it is it down to The US labs taking time to reclassify and that data being sifted through?

Ben Jackson
Ben Jackson
Equity Research Senior Associate at Jefferies

Like, what specifically is the bottleneck here that should keep us cautious? And then secondly, on the geoengine side again, can you can we just touch on a little bit more about this additional potential clinical phenotype that you're finding, for the APDS patients? And what is the path there to a potential expansion of label, for example, that would that would reclassify a, obviously, essentially a large number of patients? Is it as simple as the genetic work gets done, the, kind of the clinical phenotype is better understood, and then the breadth of patients that are available to start treatment are then suddenly expanded? Or to do this, is it gonna require potentially a slightly different diagnosis that's gonna require clinical studies and take a little bit longer.

Ben Jackson
Ben Jackson
Equity Research Senior Associate at Jefferies

Just try and set our expectations for the timeline that we could get updates from this initial work that has been completed. Thank you.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

Thank you, Ben, for these two important questions. So let me start with the reclassification. I'll hand over to Anurag about clinical phenotypes and related to this potentially much increased prevalence of APDL. So on the reclassification, as you said, things take time. So first of all, I mean, the genetic labs need actually to use the data in the self publication and identify those patients that ultimately will be reclassified.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

We don't know, first, the the number. The self publication has generated a bit more than 100 new variants that were found to activate PI3K delta. Okay? There are more variants as well. So with this first batch of new variants, actually, we will not exhaust that opportunity.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

So first is to understand with those 100 plus variants, you know, how many patients will be reclassified. We cannot know that. So we need to let the labs actually to figure this out, and then they'll inform doctors. It always takes time, some patients are being seen, you know, on a regular basis, some patients obviously take more time to be reconnect. So that's why it's something that's going to happen over time.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

On top of it, obviously, we are going to commission more studies to generate more variants, so ultimately all The US patients could have a chance actually to be considered for reclassification. And we expect that about twenty percent will have a variant that may indicate a reclassification into as APDS. I hope this clarifies. Anurag, would you like to take the clinical phenotype question, which is an important one?

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

Sure. So when we first think about this, we need to start with the fact that, you know, what is our current label? Our current label is for the treatment Joanne just indicated for the treatment of APDS. These new patients that have been found essentially have APDS. So these are patients with APDS because they have a genetic abnormality.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

They have increased, p I three k delta function or hyperactivity, and they have symptoms. And the symptoms that have been found so far, again, at a very superficial level at this first stage, are consistent with many of the types of things that we see with APDS. There what we don't know yet, and this is what we the work that needs to be done is what that feature that clinical feature set looks like in more detail. Are they are they more focused on one type of symptom or another type of symptom? And so this, again, is the work that we're going to be doing now.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

We do but because these patients have a genetic abnormality and demonstrated hyperactivity in this pathway, these are all patients with APDS and potentially within the scope of our existing label. And with that, we don't really anticipate doing new clinical trials to, demonstrate something in this population. There may be some work that could be done, but, again, the current label includes APDS. I hope that answers your question, Ben.

Ben Jackson
Ben Jackson
Equity Research Senior Associate at Jefferies

Yeah. Very useful. Thank you.

Operator

Thank you. We will now take the next question from the line of Simon Scholes from First Berlin. Please go ahead.

Simon Scholes
Senior Analyst – Biotech & Resources at First Berlin

Yes. Hello. Thanks for taking my question. I've just got one. I was just wondering if the OpEx that you're forecasting for this year includes any milestones on leniolumab.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

It does include a five million milestone.

Simon Scholes
Senior Analyst – Biotech & Resources at First Berlin

Okay. Thanks for clarifying that.

Operator

Thank you. There are no further questions at this time. I would like to turn the conference back to Fabrice Shiraki for closing remarks.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

Thank you so much, operator. Thank you all for joining our call this morning or this afternoon. As you can see, we have built over the years a very strong growth platform. I believe that we are in a unique situation with a drug like Ruconex which can provide be a source of sustainable cash flows in the years to come and allow to fund a very high value late stage pipeline. So I personally joined farming as I saw farming in a sense as the best of pharma with the sustainable source of cash flow and the best of biotech with this very very promising and high value pipeline and obviously we are committed to developing the company and making farming a leading rare disease company in the years to come.

Fabrice Chouraqui
Fabrice Chouraqui
CEO and Executive Director at Pharming Group

You can see that quarter on quarter we are executing this strategy. There is no long term without any short term, and we look forward to telling you more as we progress. Thank you so much for your attention today.

Operator

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

Executives
    • Fabrice Chouraqui
      Fabrice Chouraqui
      CEO and Executive Director
    • Stephen Toor
      Stephen Toor
      Chief Commercial Officer
    • Anurag Relan
      Anurag Relan
      Chief Medical Officer
Analysts
    • Sushila Hernandez
      Equity Research Analyst at Van Lanschot Kempen Investment Banking
    • Jeff Jones
      Managing Director - Senior Analyst at Oppenheimer & Co. Inc.
    • Ben Jackson
      Equity Research Senior Associate at Jefferies
    • Simon Scholes
      Senior Analyst – Biotech & Resources at First Berlin
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