Pharming Group N.V. (PHAR) September 2026 Conference Transcript

Good morning, everyone, and thank you for joining me at the H.C. Wainwright 28th Annual Global Investment Conference. My name is Joshua Corson, and I'm a Senior Equity Research Associate. Today, I would like to introduce our presenters, Kenneth Leinart, CFO, and Michael Levitan, VP of Investor Relations of Farming.

The floor is yours. Good morning, everyone, and welcome to our short presentation here at the conference. Unfortunately, due to personal reasons, our CEO, Fabrice Shoaki, is not able to attend today and Michael and myself will share the presentation and the updates that we have prepared for you. So welcome and thanks for being interested in hearing our update related to farming. We will today be doing a couple of forward-looking statements. I'm just drawing your attention. I'm sure you're already experts in the details written on the slide. Farming is a unique company that has a strong commercial foundation and a high value pipeline. As an overview before we get into the details, we have Rookanest which is approved for acute HAE. So it's a cornerstone on-demand therapy for high-burden HAE patients and for us a very durable cash engine. It's a drug that has been on the market for more than 10 years. We'll come back and talk a bit more about that. We also have Joenja which is approved for APDS with the opportunities to expand geographically but also into pediatric and also from a prevalence perspective. We'll talk more about that as some of you might be aware of the approval we received on Friday and which was communicated in a press release. We also have two very significant readouts of phase two studies that is planned for the fourth quarter of this year and altogether this area represents a very significant part of our pipeline with more than 1 billion of revenue potential in peak sales. The last and newest kit on the block is Nepassimone that also earlier was known as KL1333. We are expecting that the pivotal falcon study will read out in 2027 as we are progressing with the enrollments this year this is another asset that will give you more detailed insights on that represent more than 1 billion of potential peak sales as part of our current pipeline this year farming is expecting about 375 to 395 of revenues as per the guidance that we also provided during our latest earnings call. The vision for the company is unchanged. We want to develop a leading global rare disease company with a diverse portfolio and presence in large markets, leveraging the proven and efficient clinical development, supply chain, and commercial infrastructure that we already have in place. And as you already have seen from the previous page, we have two commercialized products in the US and an already established platform for further commercialization there. So let's take a quick look into Rookanest. So Rookanest has a durable market position in high burden HAE. It's a differentiated value proposition. It targets the root cause of HAE across all pathways, and it has the efficacy and reliability of an IV self-administration. It's mostly used by patients experiencing more severe and frequent attacks, and it has a highly specified manufacturing process that ultimately starts with the milk from transgenic milk from rabbits and we are seeing that you know one year after introduction of an oral competitor product that the overwhelming majority of the ruchanest patients remain on therapy so i'd like to share a little bit more details on that with you so first of all in the second quarter of this year we generated 72.3 million of sales with ruchanest it's about 10 down year and year because of some of the variability we're seeing with the competitor entering the market. But it's also 24% up versus the first quarter of this year. So we now see a stabilization and a return to revenue growth expected by the end of this year. Some of the improvement in the underlying demand data that we are seeing is that in the second quarter, we had 84 new patients enrollment, and we have 17 new prescribers. And that, as I mentioned already earlier, is more than 10 years after the product launched. So a very significant performance. We also see a resilient patient base where the active number of patients by the end of Q2, in the way we're measuring, is 93% of that a year ago. So a very stable and significant number. With that, let me turn over to Michael, who will now talk us a little bit through the Lineolisip and Joenja franchises.

Thank you, Kenneth. I'll touch on Joenja. For Joenja in APDS, we're seeing very strong momentum in revenue growth and the underlying commercial drivers. And this is true both in the U.S. and internationally. Revenue in the second quarter increased 40% to close to $18 million. Included in that was pretty robust international growth, where revenues increased to $2.5 million from $1 million a year ago prior. Patient growth continues to be quite strong in the U.S. We are now up to 132 patients on paid therapy in the U.S., and that's a 16% increase compared to the prior year. Importantly, we are continuing to expand the diagnosed APDS population. This is an ultra-rare disease, so finding additional patients is very important to make sure that therapy gets to patients who need it. And we're doing very strong progress there. On that note, in pediatrics, there are now 60 eligible patients who've been diagnosed with APDS in the pediatric age group between 4 and 11 that we're aware of. Last Friday, we just received approval for our first approval for pediatrics between 4 and 11. This is in the group of patients who weigh at least 27 kilograms. There was a PDUFA date of October 24th, but we came in ahead of that. And this is an approval for 40 milligram and 50 milligram twice daily dosing. And the commercial launch or commercial availability to patients will be in October next month. Separately, we have a separate SNDA filing for lighter patients in this age group. That was filed on July 30th. And we're awaiting feedback from the FDA who's reviewing the filing. And that will cover the lower doses. But, you know, very significant commercial opportunity. I think it's important to note the list price for Joenja and APDS is in the vicinity of $600,000 per year per patient. So this does represent a meaningful commercial opportunity for us. Looking forward to some of the key growth drivers for Joenja, a lot of detail on the slide that you can reference later. But the sources of growth in APDS include continued patient identification, as I mentioned, ongoing genetic testing, efforts to reclassify patients who have uncertain genetic testing results, and also broader prevalence expansion where there's a significant opportunity. But I think near term, the pediatric expansion represents an important growth driver through through 2027. And also we've now received regulatory approval in all of our key target markets, including the UK were launched, Europe where the first launch has taken place already back in July in Germany, Japan where we're now launched and several other markets. So I think we're really pleased with that progress and there will be important commercial expansion sort of on the back of those approvals. And that is driving the strong revenue growth we're seeing internationally, which should accelerate going forward, given the regulatory progress we're seeing. Importantly, we are running two phase two studies in significantly larger indications for genetically driven PIDs, primary immunodeficiencies, as well as common variable immune deficiency with immune dysregulation. And together, these indications represent prevalence of around 26 times APDS, so a much larger commercial opportunity. So I'll spend a little bit of time on that, but before I do, taking a step back to the biology, PI3K delta, and Joenja is a PI3K delta inhibitor, is really a master regulator of the immune system. And it's imbalance in this pathway that leads to immune dysregulation, which is seen in lympho perforation, either spleen or lymph node enlargement, autoimmunity, GI, or lung disease. And I think it's that dysregulation that's driving the benefit of the, driving the disease manifestations and the benefit of the drug. And it's really the scientific rationale for joenja. And this PI3K delta inhibition is really validated in APDS. In APDS, we've seen consistent and durable reductions in lymph node and spleen volume, and really sustained benefits for up to seven years for patients on therapy, well-established safety profile, and really life-changing benefits for patients who have no other approved treatment options. And this gives us confidence in the ability for leniolisib or joenja to have benefits in some of these larger indications. And looking at that, you can look here on this slide at the expansion opportunities. And these genetic PIDs are a group of other primary immunodeficiencies which have immune dysregulation connected to PI3K delta pathway, as well as CVID or common variable immunodeficiency with immune dysregulation where the patients are diagnosed clinically without a specific underlying genetic mutation. Again, the prevalence is 26 times APDS in totality. You can see the clinical manifestations are shared with what we've seen in APDS, specifically lymphoperforation, autoimmunity, and the end organ disease. And we're looking to the results of these two phase two studies to indicate the benefit that we can see in a much larger indication. Looking at the phase two trials that are ongoing, these are fully enrolled. We're expecting near-term readouts in the fourth quarter. Two separate trials and those two separate indications I indicated. There are 15 patients and 20 patients. And what we're looking for in these trials is both to measure and to evaluate the benefits in lymph node and spleen size reduction, as well as the other, you know, blood cell counts and end organ manifestations, safety, et cetera. And it's really the improvement across these measures that should guide the next stage of development. Our plans, our expectation is that we would initiate on the back of these phase two studies, a phase three trial in the broader CVID indication that encompasses the patient populations from both of these studies. So that's our hope, and we're looking forward to the clinical readouts to support that. Turning now to nepazamone, formerly KL1333, for primary mitochondrial disease. This is a drug that's being developed for patients who have mutations in mitochondrial DNA that lead to significant fatigue and muscle weakness. The endpoints in the trial are really tied to those manifestations. The mechanism of the drug, the drug normalizes the NAD plus NADH ratio, which is abnormal in these patients. And there's over 30,000 diagnosed patients between major European countries and the US that we're aware of that have been diagnosed. And currently we're in a registrational enabling study that's ongoing. The endpoints have been agreed to by the FDA. Importantly, and this was something we saw before we acquired a company called Obliva that owned this drug, is there was a blinded interim analysis in which both of these endpoints passed futility. So that gave us confidence in the product, led us to acquire the drug back in early 2023, sorry, 2025. and then we re-initiated the second wave of the study. That second wave of the study is currently enrolling at over 45 sites globally. We expect to complete enrollment by the end of this year and for the trial to read out by the end of 2027. You can see here a little more detail on the interim analysis. I think importantly, what the interim analysis concluded was that there was acceptable safety and tolerability. No change to the study design was required. 180 patients in the trial was confirmed as the total. Important to note that the 45 patients, the 40 patients in the first wave of the study are part of the overall patient enrollment, that 180. So that data that we saw, the data that was seen in the futility, the interim analysis will be part of the overall readout. And expectations are that if similar efficacy and safety benefits are seen in the overall study, as what was seen at the interim, this should result in success at trial completion.

Let me turn it back now to Kenneth. Thank you so much, Michael. So for the full year of 2026, as I already mentioned, we expect revenues to be around 375 to 395 at the midpoint so the 385 we will see continued very strong joint growth with about 38 year and year and as ruconest is stabilizing and returning to growth towards the end of this year we expect that at around a few you know minus three percent at guidance midpoint roughly we continue to have strong profitability our gross profit is around 89 and we have a strong discipline around managing expenses that this year i expected in the range of 315 to 320 million that's growing about one to three percent but does include that we are investing around about 40 million more into advancing our pipeline and you know at the same time we see marketing and sales cost being relatively stable and a significant reduction in the gna including a gna structural head count reduction program that we announced last year in October. So our focus on optimizing capital allocation to drive both short and long-term value creation is really strong and significant. We also, given the cash generation from Rukonest that I mentioned earlier, in the position that together with our available cash, which was about 160 million at mid of this year, and future cash flows generated that we can fully finance our current pipeline, so the clinical development cost and also pre-launch cost associated with that. Now, in summary, so farming is advancing as a leading global rare disease company. We have strong commercial foundation, as we talked about with Ruconest really being resilient and a durable cash engine, and Joenja having a very strong sales momentum we have significant growth opportunities michael talked about it in terms of expansion of joanja for apds but also with the upcoming two phase two readouts that we're expecting to see in the fourth quarter of this year and the napasimone falcon study that is planned to read out in 2027. so you know we have more disciplined operating model we have a scalable organization in place, we have a strong value creation focus, and we are aligning our shareholder base with the overall vision of the company. So with that, let me thank you again for having attended the short presentation and update here. We will have a few minutes for any questions you may have. So over to any of you.

Yeah, if anyone has a question, please feel free to come up to the mic.