I'm from the European Biopharma team here at Jefferies. It's a pleasure today to be joined by Fabrice, CEO of Pharming Group, for this fireside chat. Fabrice, let's just dive right into it. I think if I hand over to you for a few introductory remarks, then we'll kick off with a bit of Q&A.
Absolutely. Thank you so much, Ben. Thank you for being with us today. I'm the new CEO of Pharming, joined about three months ago, spent the past five years at Flagship Pioneering in Boston. Prior to this, 10 years at Novartis and 10 years at BMS. I was the President of U.S. at Novartis. I mean, the fact that I had the opportunity to enjoy, I would say, the rigor and sophistication of big pharma and the nimbleness and value creation focus of biotech, and specifically a biotech fund like Flagship, I mean, led me to Pharming, as I see a lot of value to unlock. I mean, this is a company that developed extremely well over the past decade or so. I'm very fortunate to inherit a very strong growth platform with a steady revenue stream, very strong growth.
I mean, you've seen the result that we posted in Q1, + 39% versus last year. Also, and potentially most importantly, a very, very strong late-stage pipeline, not early-stage obscure asset, but really either a new indication or proven, I would say, a drug like Joenja already on the market. Also a very, very exciting opportunity with Abliva, which is in a registrational trial, having gone through already a positive interim study. Very excited by the opportunity and obviously looking to interact with you, Ben, today.
Brilliant. Look, let's start on that then. Obviously, since joining as CEO earlier this year, what has been most striking to you about the company? If we do the flip side as well, what do you think is what you would like to deliver now that you are more familiar with the setup of the firm?
Yeah. As I said, I mean, I think we are fairly uniquely positioned in this environment, being a biotech with a steady and predictable revenue stream, strong growth in the short and long term, but also a very strong pipeline. For me, it's a little bit the best of the two worlds in terms of the predictability of what you can call a small pharma, but really the value creation potential of a biotech. I really see Pharming as a biotech. Perhaps to answer your questions, Ben, for me, it's very much about developing Pharming as a large small company as opposed to a small large company.
If there are any adjustments to make, it's to make sure that as we transition to the next level, we remain really very close to our entrepreneurial roots and develop as a large small company and remain, I mean, maintain this biotech mindset that made Pharming a successful company so far.
Brilliant. Before we jump into the drugs, I want to touch on your recent first quarter results. You did raise the revenue guide. Could you tell us a little bit about what's contributing towards that momentum that you're seeing?
Yeah. I mean, it's very healthy growth from the two assets we have on the market, Ruconest and Joenja. The underlying demand is very strong. I mean, Ruconest has been on the market for 10 years and has a very, very sinking prospect in the future. That's also something that I was able to see in my first few months is that Ruconest is far from being at the end of its life cycle. It's a drug that may well be actually, and I mean, may remain a cornerstone of the HAE on demand treatment for the next few decades. When you come to Joenja, I mean, it's a drug which is at the very beginning of its life cycle. I'm sure we'll have the opportunity to talk about this. Stronger underlying demand on both generates actually strong growth and therefore a strong quarter.
Makes sense. Let's start on Ruconest since we're there anyway. I guess you've been quite clear about your expectations for this year, especially when we're considering facing the potential approval of an oral version around middle of the year. So what impins that confidence that Ruconest can continue growing at the high single-digit % rate in the face of that competitor launch?
I think what makes Ruconest unique, it's value proposition to patients, which is linked to its unique mode of action and its efficacy profile. It's a drug which over the years has carved out a subsegment of the HAE on demand market and being used mostly by moderate to severe patients who have more severe, more frequent attacks. These patients often have gone through other treatments. They failed other treatments. Really, the high efficacy of a drug like Ruconest allows them to get their life back. That's why you see a lot of stickiness when it comes to the prescription. You can see actually that even with the prospect of new drugs being launched, quarter after quarter, there's still more doctors prescribing the drug. There's still more patients using the drug, given its highly differentiated profile in this segment.
What about beyond this year? Are all of those reasons relevant for 2026 that you believe that you can continue to grow strongly in similar fashion?
Absolutely. Obviously, I mean, when you have new drugs being launched, you always see some short-term disruptions. I don't have a crystal ball. I can't say how it is going to pan out. There are always, especially in the U.S., a lot of free drugs floating around. Although I doubt that actually many patients who had actually a long journey to end up with a drug that can control them will switch. In the long run, I actually even think that this new treatment could actually be an opportunity for Ruconest to grow even further. Why is that? Because when you think about the on-demand market, you have a very established brand. It's not a very dynamic market. Actually, very few doctors know if they're, I mean, are engaging with their patient to know whether they are controlled.
With new drug, you see actually a much more dynamic market, more switchers, doctors actually spending more time with their patients, understanding whether they're controlled or not. Ruconest is being seen as the high-efficacy treatment. I'm really looking forward to this market becoming a bit more dynamic. I see that as an opportunity for Ruconest in the long run.
I guess, are there any other growth levers to round it off that could be available for Ruconest in the near future?
Already, I would say, if we can see actually more suboptimally controlled patients being treated with Ruconest, I think that could be a great, yes, great for the drug.
Yep, that makes sense. Look, let's move on to Joenja then. Do you want to just say a couple of words just to set the scene for people who may be a little bit newer?
Yeah. Joenja is a drug that treats APDS. It's a rare disease, an ultra-rare disease affecting about one patient per million. I mean, what's very exciting with Joenja is the long-term potential of the drug. Today, we have a label which is limited to the adult APDS population. We saw a good uptake in the first year of the launch. As usual for an ultra-rare disease drug, after an initial bolus of patients, you are in the trenches. You see, you always have a small portion of the opportunity. Those patients are well identified that actually switch to your drug quickly. Afterwards, you have to work to identify those patients and get them on drug. That's what we're doing. We're very happy to see that actually the work is bearing fruits.
In Q1, we saw a real acceleration of new patients on the drug. There are actually new opportunities coming, new growth catalysts. We will see the reclassification of VUS patients coming in the second part of the year. These are patients who got a genetic test, but for whom this test was not conclusive. There will be new data being published very soon that shows that actually a portion of these patients, about 20%, are actually APDS patients. That is going to be another opportunity to expand the addressable population. It will not happen overnight. The same way, we will see a bolus of patients. Again, that is going to fuel the, it is going to be an additional growth lever that is going to fuel the growth for some years. In the early part of next year, we will also expand our label.
We expect to expand our label to the pediatric population. That is going to be also the source of another lever. In the short term, a drug that will have actually a number of growth catalysts and that should result in the acceleration of the uptake. In my opinion, that is traditional with what you see with an ultra-rare disease drug as opposed to, I would say, a classic specialty or primary care drug. I mean, primary care drug, often you wait for access, so it is flat for a long time, and then you get the uptake or the specialty care where actually it is more linear. I think it is a drug which meets perfectly actually the uptake trajectory of what I believe is a strong, I mean, a strong value creation story for an ultra-rare disease drug.
That makes sense. Look, I think we've previously spoken about that there's about over 1,300 patients in the U.S. with one of these VUS test results. Perhaps you've just said 20%, which potentially could be reclassified depending on the publication. Could you outline what the next steps are in a tangible manner for this VUS in terms of the process required to begin converting the patients from the second half of the year?
Absolutely. As I said, again, these are patients who had a test. They have been identified. The doctors thought actually they may have had APDS, but the test came unconclusive. Now the new data that are going to be published in a top peer-reviewed journal anytime soon says that actually 20% or so of those patients are actually APDS patients. It is going to work. I think the genetic testing labs are going to incorporate those results into their model. They are going to look at their patient database, reclassify those patients that have to be reclassified, reach out to their doctors, and the doctors will have actually to reach out to their patients. That is why some of the process will go fast for some of the patients.
It may take more time with others, but ultimately, it's a sinking pool of patients that will be added onto the drug.
That makes sense. Makes a lot of sense. I guess the last time we caught up in London, we were talking a little bit about the U.K. potential launch of Joenja. Could you talk about what the initial feedback has been there from the patients, the physicians that you've been seeing in the region?
Absolutely. I think I told you about some growth catalysts inherent to the drug per se. There is another one as well in the near term, which is actually the geographic expansion. I mean, today, although we are a Dutch company, the bulk of our revenues are coming from the U.S. I mean, we are using Joenja to expand geographically. We're doing that in a financially disciplined way. We've targeted actually eight countries in which we believe it makes actually sense to build operations. The first one is the U.K. I mean, we've launched a drug a month ago. I was there. I was very impressed with the engagement of the doctors. I mean, the first feedback I'm getting is very, very encouraging.
Again, we are expecting a substantial amount of patients to come from countries outside of the U.S., these key eight markets, but also outside those eight markets. We won't have presence, but obviously through access programs and potential local partnership, we expect to make the drug available in a financially sensible way.
Look, that's the two of the growth levers that we've got for Joenja . We've got the first one, obviously, VUS, the second one, geographic. Arguably, there's a third one in there as well, which is potential expansion to very similar diseases. Could you talk a little bit about what these diseases are and the relative prevalence of them and also what data is supporting that transition into these alternative areas?
Absolutely. I think this is actually the big opportunity. I think, I mean, I've just described actually short-term growth catalysts that will fuel the uptake of the growth. What Ben, you're describing, this potential two new indications could propel the brand to a whole new level. Here we're talking about primary immunodeficiencies with immune dysregulation and CVID, common variable immunodeficiencies with also immune regulation. These are patients who have very similar phenotypes to APDS patients. Their phenotype is actually the clinical symptoms is linked to a dysfunctional PI3K- delta pathway as well. We've seen actually, we've had, we have actually a handful of these patients in our access program. We've heard actually very encouraging feedback from doctors. Obviously, you cannot conclude on just a few patients. That's why we are carrying out these two phase two proof of concept trials.
We have very high expectations for these two studies that will read out next year.
Very exciting. Look, we've spoken a lot about volumes and areas for volume growth, but we also have to obviously touch on pricing as well. I think in your first quarter release, you did mention that the gross net adjustments for Joenja were a bit of a drag on the growth. How is this net price of Joenja expected to evolve throughout 2025 and beyond?
There was a bit of impact, and it was linked actually to Medicare. Joenja, it's about 45% commercial, 45% Medicaid, and 10% Medicare. Even though Medicare is a small portion, as you know, there's been actually some change in the Medicare program. That has a small impact actually on the gross to net, 2%, an additional 2%. That was actually a one-off, very, very clearly with a clear rationale. I don't anticipate any changes, any additional changes during the year. This happened the first quarter, and that's it. Overall, I mean, what I can say again that, I mean, we have a label that will allow us to actually fuel the growth of the brand before those potential new indications. With those two new indications, we have a drug which potentially could well become actually the first billion-dollar drug for Pharming.
Good, good. Look, we'll move on and switch gears a little bit to Abliva, I think, that you recently acquired. The primary asset in there is KL1333, assuming that's how you guys refer to it as well.
I promise you we will try to find a better name.
That's for the primary mitochondrial disease. What do you, when you were assessing that asset, what did you find particularly attractive about it and what led to that decision?
I mean, primary, so mitochondrial disease, MCD, are clearly a group of diseases which are extremely debilitating. That doesn't allow patients to live a normal life. Actually, there is a significant prevalence. I mean, just U.S. top five EU market, we're talking about 30,000 patients. These are patients which are treated in, who are well identified, treated in center of excellence, including in the U.S., who often actually are part of advocacy group. What really excited us is obviously first and foremost the unmet need. I think that's why we, I mean, what Pharming, what we live and breathe at Pharming is being a rare disease company is being able to provide really transformative treatment to these patients. That fits completely with our mission. That fits completely with our capabilities as well.
I think we have developed amazing capabilities when it comes to clinical development, access, supply chain in this rare disease, which is not necessarily straightforward. The potential is there. I mean, this is a drug for which you have a very consistent set of data, preclinical data, animal, so in vitro, in vivo, phase one. This is a company that we've been following for years. We've been following that program. We could see that they had this registrational phase two ongoing. After they did this interim analysis and showed that this interim analysis was positive on the two endpoints, two endpoints which were actually agreed with the regulators. No biomarkers, real endpoints. We felt actually that we had a real opportunity to supplement our pipeline with a very high potential asset. That triggered actually the acquisition.
That makes sense. I guess, as you say, you've had the interim analysis. We're still waiting for the results, the full results from that trial. Could you talk to us a little bit about how the design of the trial actually positions the asset versus competitors in that landscape? Secondly, as a second part of that, what would a successful trial look like, I guess?
Yeah. So again, it's a trial which will enroll about 180 patients. There's been an interim analysis being done on 40 patients again. It's really not negligible. There are no actually approved treatments for mitochondrial diseases at the present time. The unmet need is very, very significant. As I said, the endpoint have been pre-agreed with the regulators, which is important. There are no misunderstanding downstream. We expect to complete the trial in 2027 and expect to launch probably early 2029.
Makes sense. I guess to what extent does Pharming's existing commercial setup support or provide synergies for what may be required if that trial is successful? How much additional investment could be required to set that up through to then?
We've said that we would completely self-fund this acquisition and the development as well as the pre-launch of the drug. I think that's the, as I alluded to at the very beginning, I think that's the, I would say the great thing about Pharming is that, I mean, we can self-fund our development. We have a very robust cash flow coming from Ruconest. In my opinion, that makes the company stand out in the current environment. Given our capabilities, as I said earlier, there's a real fit. When you think about those diseases often, people often ask me, oh, is it going to be a different Salesforce or is it going to be the same Salesforce. When you think about your commercial resources, your commercial investments, nowadays, actually the Salesforce aspect is just actually a small portion.
What makes or breaks, I would say, the success of a drug launch is very much your medical affair capabilities and your access capabilities. Two areas in which actually we've actually, we have demonstrated, I mean, great, we have a great track record. Okay, Ruconest, Joenja. I would say those resources, we will not need more resources. Those people are very eager actually to start working on KL1333. Again, one of the priorities is to find a name. It should be, it's clearly a product that will be value-accretive very quickly for the company.
That's good. I think your management's building a bit of a track record about their BD and M&A in a smart way and opportunistic way that they've gone about that. How do you view BD going forward? Is it something that remains a priority or something that's more opportunistic? That's the first part. I also would be interested to see what you're hearing or seeing about the actual BD and M&A environment as it stands given the ongoing U.S. politics, which I promise you we'll touch on in a moment.
Listen, BD is very much at the core of our development. The company made a conscious decision a few years ago to dismantle its research operation, feeling that we didn't have the critical mass. I mean, coming from being a scientist by training, having done actually five years of very intense preclinical research and taking off a great company in this field, I completely concur with the decision of the previous management. Now, as BD is at the center of our strategy, it's not, I mean, it's not that we're going to be working hastily and we need actually to do BD deals every year or we set targets. I think today, as I've explained, we have actually a lot to do. We have very, very clear and significant growth catalyst in the near term.
We have a pipeline with a very significant value creation and some value inflection point in the near and midterm. There is no urgency to act. Yet it is at the center. We continue to identify opportunities. We want to be disciplined. It has to be value-accretive. The company, as you've said, Ben, has redeveloped a very significant know-how in this field. I know well, having done a lot of BD in my life, that these are real capabilities that you develop. I mean, it's easy to do deals. It's not easy to do good deals. I think companies are always very happy to be on the front page of the newspaper for their deals. Then afterwards, you know, you need to see whether those deals have been value-accretive and very few are.
I think we have at Pharming developed a track record to do value-accretive deals. I intend to pursue this mindset. We will continue to do this. As I said, not a priority. If we believe that we have to act on some opportunity that we follow, we will do so. That is not the priority at the present time.
That makes sense. Look, the business is largely focused at the moment in the U.S. on the sales basis. I think your manufacturing is based in Europe. That begs the question, how would you fare or would you look to approach potential pharma tariffs? I know we do not have a crystal ball to see whether it is going to happen, but what are your thoughts around that?
Sure. I mean, my thought is that obviously you have to be prepared. I don't have a crystal ball indeed. I don't know whether there'll be tariff, you know, on drugs, whether those tariffs will impact rare disease drugs, when this may happen. Yet I want to make sure that we are at the forefront of this and can mitigate the impact. When you look at our portfolio, I mean, when it comes to KL1333 and Joenja, we're talking about small molecules. Easy, it's here, it's fairly easy actually to make adjustments in your supply chain, in your manufacturing process. These are low-volume, small molecules. This is fairly easy for Ruconest. I mean, the fact that Ruconest, as in my opinion, many decades ahead is the very, very specific manufacturing process, proprietary, very complex.
For those of you who don't know, I mean, this is a drug which is a recombinant protein made out of the milk of transgenic rabbits. As you can imagine, that cannot be transferred overnight. Now, it doesn't mean that there are aspects of your supply chain, of your manufacturing process that you cannot transfer. That means that your entire supply chain cannot be transferred. There are still actually a lot of things that you can do to mitigate the impact of tariffs. We are actually actively looking at it. If tariffs were to kick in at some point, we'll be ready to mitigate their impact. I want to manage expectations here. I don't want to trade the long term for the short term.
I think we have created an amazing transgenic platform in the Netherlands that actually will be the source of very significant cash flow in the long run. That will remain. Now, again, there may be, there will probably be some adjustment to the supply chain that can allow us to mitigate tariffs on Ruconest.
Very exciting. Thank you. That takes us to the top of the time. Fabrice, thank you so much for joining me. It's been a pleasure as always. Thank you all for listening in. If you have any questions, please reach out to the team or.
Thank you.