Pharming Group Q1 2025 Earnings Call Transcript

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Provided by Quartr
May 8, 2025
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Operator

Hello, and welcome to the Farming First Quarter twenty twenty five 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. I would now like to hand over to Fabrice who's CEO.

Operator

Please go ahead.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Thank you. Hello everyone and welcome to this Q1 twenty twenty five call. I'm Fabrice Chouacchi, I'm the CEO of Pharming and I will be joined on this call today by Stephen Tor, our Chief Commercial Officer, Anurag Rellen, our Chief Medical Officer and Yaron Wachemann, our Chief Financial Officer. Next slide. In this call, we'll be making forward looking statements that are based upon our current insights and plans.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

As you very well know, this may differ from future results. Next slide. First of all, let me say that I had a great first three months at Pharmi. The passion and the commitment of all employees to serving rare disease patients is really palpable throughout the company, whether you are at the production facilities, at the headquarter or with the field. And this mindset of going this extra mile is clearly part of the DNA of the company and not just a few words on a culture slide as I've seen it actually two of them.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Next slide. So everyone is clearly determined to realize the vision that we've set for the company, which is to make farming a leading global rare disease company with a diverse portfolio and presence in large markets that leverages a proven and efficient clinical development, supply chain and commercial infrastructure. Next slide. Our results in the first quarter of twenty twenty five are a good illustration of the solid growth foundation that we have built to realize this vision. We've had a strong start of the year, with our total revenues increasing by 42% in the first quarter and a significant improvement of our bottom line, which supported an upgrade of our full year guidance.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Going into a little more detail, RUCONEST grew 49% to $68,600,000 driven by a continued increase of new patient enrollment and the sustained expansion of our prescriber base, as well as a lower inventory destocking versus the previous quarter. RUCONET has a unique profile on the on demand HAE market, which makes it an extremely valuable treatment for moderate to severe patients who experience more frequent and stronger attacks. And this differentiation really explains the strong momentum and growth prospects that we have for Reconnect in the long term. When it comes to joint job, revenue increased by 9% driven by robust new patient enrollment, offset by the impact of stocking in the last quarter of twenty twenty four and higher but expected gross to net adjustment versus a year ago. The efforts to identify new patients after the initial bolus of launch is really starting to pay off with six new patients in The US this quarter, the largest quarterly increase since Q2 twenty twenty four.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

We expect to see a sustainable acceleration of the number of new patients on Joynja starting in the second half of the year with three clear growth catalysts to come: first, the reclassification of VUS patients in The US Second, the pediatric label expansion And third, the launch in key markets outside The US, which started actually with The UK just a month ago. In the first quarter of twenty twenty five, our operating loss narrowed very significantly year over year and we even generated a profit for the third quarter in a row if we exclude the non recurring Abliva acquisition related expenses. So given our strong growth outlook, I believe that our ability to be disciplined financially will be as important as generating strong top line growth to unlock significant value creation in the near and long term. And as a first step, we've made a decision to cut G and A expenses by 15% or $10,000,000 on an annual basis to optimize capital allocation to grow our business. Next slide.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Our pipeline continues to progress well during this quarter. Both the Genetic PID and CBID Phase two studies are now initiated and enrolling patients. These two indications in patients with immune dysregulation who may benefit from a modulation of their PIK3 delta signaling pathway have the potential to propel Joynja to a whole new level given the much higher patient prevalences. The clinical development team also worked very hard to resume the enrollment of the phase two registrational trial for KL-one hundred thirty three in MTD just a few weeks after the completion of the Ablivia acquisition. Before I let Anurag tell you more about these $2,000,000,000 plus pipeline opportunities, let me first hand over to Steve Thore, our Chief Commissioner Officer, who will give you a more granular perspective on the strong dynamics of Ruconext and Joynja.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Steve?

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Thank you, Fabrice. Good morning, everybody. If you could go to the next two slides, the RootConnect performance. So as Fabrice said, we've delivered a very strong performance in Q1. RootConnect is up 49% versus prior year.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

This reflects the strong trend that started in early twenty twenty three as we emerged from the disruption caused by the pandemic. Since then, our sales teams have added new prescribers and new patients on a quarterly basis, which has translated to consistent quarter on quarter growth of Bruker Nest sales. In addition to the underlying strength of the business we also saw significantly less inventory build up as Bruce mentioned especially pharmacies in Q4 less inventory to wash out in Q1. Sales therefore largely reflect the demand created within the quarter. I'm also pleased to share that our market access team also improved the number and speed of patient prior authorizations in Q1 enabling our patients to reorder RUKINESS earlier than in previous years.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

So these two factors combined also strengthen the RUKINESS performance in Q1. The key though to the past and future performance is of course RUKinesse. It always starts with a good product. So next slide please. And in RUKinesse we have an excellent product serving all patient types, those being type one, type two and the normal C1 patient population.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

All three groups as it relates to RUKINESS have one thing in common. 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 icatapam or are having to re dose to resolve their HAE attack. The RUKINESS delivers attack resolution for ninety seven percent of patients in a single dose is therefore a major factor in patients continuing to choose it to treat their acute attacks. Now as some of you know I've been with the company for almost nine years so I've witnessed firsthand the continued growth and also the enduring strength of the Root and S business.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

So I can testify that as well as being a very good product, it's also the result of very deliberate positioning and disciplined messaging to the needs of the more severely affected patients, primarily by our sales team, also supported though by the services we provide to ensure patients get covered, they get trained, and are able to benefit from RUKINESS confident to infuse themselves over the long term. Now on this slide you can see an actual patient and in the photographs you can see the patient at the start of an attack and then a recovery as it resolves at the four hour mark and the twenty four hour mark. So for patients like this, as I said, suffering with a severe course of disease, attacking frequently and having to re dose another therapies, knowing that ninety seven percent of patients will get their attacks stopped in a single dose and almost all of them will be attack free for at least three days is critical to their decision to use Ruchinest. So Ruchinest efficacy, its reliability allows our patients to better plan and control their lives. So the severe course of disease our patients have and our team's execution of excellence is why RUPES will continue to have a strong position in The US acute market and remain an important product for us for many years to come.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Next slide please. Transitioning now to Joenga. As Fabrice said, we increased sales by 9% to £10,500,000 in Q1. And importantly, we saw an acceleration in Q1 adding six more patients ending the quarter with one hundred and two patients on paid therapy in the identified over two forty. Additionally we have one hundred and eighty seven patients on treatment globally and we've now identified close to nine hundred patients worldwide.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

And as you'll see in subsequent slides Joendra is still in the very early stages of its life cycle with many well identified opportunities to accelerate growth in the near term including the VUS program rollout, a pediatric label extension and the launch of Joengia in key markets outside of The US which actually started just this call last quarter in The UK at a launch meeting in London which I had the privilege to attend. I can share that physicians and patients there are just as excited as those in The US by the prospect of now accessing Joenga for APDS and the potential it has to transform patients lives with APDS just as it has in The US. As with Ruchinist though it always starts with a good product and in Joenga we have the only product indicated to treat APDS. A serious debilitating and often fatal disease. Next slide please.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

I think as you all know APDS is a primary immune deficiency caused by pathogenic variants in either of the two genes that encode the PI3K delta enzyme. The result is the immune system doesn't develop properly having more immature cells and less functional cells. This leads to the symptoms APDS patients suffer with which are often serious, have a negative effect on health, quality of life and the patient's ability to live what we might consider a normal life. Unfortunately, APDS can and often does also lead to early mortality. So until the development and launch of Joenga, APDS patients had no targeted or indicated medications to treat the underlying cause of their disease.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Importantly, Joenga was specifically designed to correct the underlying immune defect selectively inhibiting PI3K delta and normalizing that hyperactive pathway thereby restoring balance to the immune system. And in doing so, joenga has that potential to transform patients' lives. Next slide please. So let's look briefly at a typical patient with APDS on joengia. In this case it's a 24 year old male that we followed up over six years.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

On the left hand side you can clearly see the severity of the symptoms that this patient endured and the burden and impact it had over a prolonged period of this 24 year old man's life. After starting Joengia, our patient was able to stop infusions of immune globulin, had no hospitalizations, his blood platelets increased and critically the organ damage, in this instance the lungs, hasn't worsened. The clinical impact means this young man has been able to walk and drive without difficulty, graduate high school and university and has now secured a full time job. Results like this is what I meant when I stated on the previous slide JoAnja's potential to transform the lives of APDS patients. It's also why myself personally, my team and in fact all of Farming's employees are excited for our global launches for APDS and what the molecule will deliver for patients and farming over the long term.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Next slide please. Finally I want to review those near term opportunities I mentioned for Joengia in both APDS and the longer term beyond. Right now our teams have in their hands the patients we've already found and those remaining patients that bridge the gap from what we have today to the full prevalent populations in major markets around the world. We can also look forward to some milestones that are important additional opportunity to expand that addressable patient population. And if you look at the second block on this slide, the first is our targeted geographic expansion program to key markets.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

As I mentioned this has already begun with The UK launch and the teams are preparing for further launches on approval in Germany, France, Italy, Spain, Japan, Canada and Australia which means JoAnja will soon be available in most of the top 10 pharma markets around the world. The second is the outputs from the VUS resolution program, which Anurag will discuss later. That will deliver another bonus of APDS patients available for treatment this year and beyond. And the third will be the pediatric indication launch in The US, which is expected in 2026. We currently have over 60 pediatric patients in our US patient pipeline and that is growing.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

And they will be transitioning to Juenga as soon as that indication has improved. Those are all significant near term opportunities. In addition to that, as you see in the final two blocks of this slide, leniolusib for APDS is only part of our story. Trials have also been initiated using leniolusib to treat PIDs with immune dysregulation including CVID, which while still a rare disease with a prevalence of forty patients per million transitions leniocid from a small ultra rare disease molecule to one with blockbuster sales potential, thereby creating a leniocid franchise delivering significantly greater value for all stakeholders in the coming years. So we here and our teams have a lot to be excited about.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

With all that said, I'll hand over now to our Chief Medical Officer Anurag Grellan whose teams are critical to driving these programs forward to provide us with an R and D update.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

Thanks Steve. Let's turn now to those growth drivers you were talking about including beginning with those near term opportunities that we can see on the next slide. So as you heard from Steve, geolingia can have a real impact on patients' lives, but one of the problems in diagnosing APDS happens when the result of the genetic test shows what's called a variant of uncertain significance or a VUS. In fact, we found more than thirteen hundred patients in The US alone who have received such a result. This happens because the variant is new and there isn't enough information about that variant to know if that variant is actually variants and screen those variants for PI3K hyperactivity.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

The results, which will be published soon, will show that many of these variants actually do lead to hyperactivity of the pathway and can eventually cause these variants to be reclassified as causing APDS. The next step in this process will be for genetic testing labs to review the data from this, academic lab and the publication, reclassify these patients and then inform the doctors of any changes to the previous reports that showed these patients had a VUS. We expect that together all of this will lead to the identification of many new APDS patients later this year as you heard from Steve. On the next slide, we can see some of the other opportunities that we have in terms of bringing leniolusib to more APDS patients in several key markets across the world and to expand the addressable population. To support geographic expansion, we have a number of regulatory applications underway in several key markets across the world on the left.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

With the EMA, European Medical Agency, we have a single outstanding CMC request, and we remain on track to respond to this in January 2026, which could then allow for approval soon afterwards. You heard from Steve, we already have MHRA approval, and we launched the drug there. We also have approval in Australia and an application under review in Canada. In Japan, we expect to file mid this year. And then I think one of the other important things you heard from Steve is the pediatric need in APDS.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

This is a genetic disease that where symptoms begin early in childhood and given the progressive nature, treatment is critical. To address this, we have a program with two studies that have actually completed enrollment now, and we intend based on the results of the first study to be able to file with FDA in the third quarter of this year with an expected six month review of this efficacy supplement. Once we have the data from the second study, we'll follow a similar path to be able to bring cholangia to even younger patients. And then in the next couple of slides, I'll be talking about the two phase two studies we have underway to expand the use potentially of leniolusib even further to additional primary immune deficiencies. And you see that here now on this slide where we're looking at using leniolusib for primary immune deficiencies with immune dysregulation.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

Can you advance to the next slide? There we go. This group of patients with primary immune deficiencies with immune dysregulation is a group that's similar to APDS with their clinical features, the unmet need, and early mortality. It's also a group that's significantly larger than APDS. But the rationale for setting leniulose here is very clear.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

The PI3K pathway in lymphocytes plays a clear role in the dysregulation seen in these patients, which drives the lymphoproliferation and the autoimmunity. It's actually the same approach that we have that we've seen be successful in treating APDS patients where modulating that enzyme can address the immune dysregulation. We also have a positive experience in a handful of patients who've been treated with leniolusib now for more than six months as part of a compassionate use request that we've received from physicians across the world. So now we have two phase two dose range finding studies underway. One in a genetically defined group of primary immune deficiencies and the other in what's called CDID or common variable immune deficiency, both with these hallmarks of immune dysregulation, and we expect the first results from these studies to be available in mid twenty twenty six.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

And on the next slide, you can see the third molecule that we have in our portfolio, which is KL-one thousand thirty three for primary mitochondrial disease. This is a disease of impaired energy production due to mitochondrial DNA mutations which cause fatigue and muscle weakness in these patients, which you can see described in the quote from the patient on the right. This is being developed now for primary mitochondrial diseases with these, which is a group of rare disorders where these patients have these mitochondrial DNA mutations. There's a large number of these patients already diagnosed in The US and large countries, and they're usually treated at centers of excellence and part of a large advocacy group. KL-one thousand thirty three addresses the underlying problem by normalizing the NAD plus to NADH ratio, which is abnormal in these patients.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

And we have a registration enabling study underway with endpoints that have been supported by regulators, including FDA. We've already completed a blinded interim analysis in which both endpoints pass utility. And we've also, as you heard from Fabrice, restarted recruitment in the second wave of this study and expect readout in 2027 with potential approval later in 'twenty eight. And now on the last slide here, you can see our expanded pipeline, which will provide significant growth prospects beyond rucinectin HAE and Ziowenja in APDS. Across this portfolio, we can use our rare disease expertise and development infrastructure to bring products to patients where there's significant unmet need.

Anurag Relan
Anurag Relan
Chief Medical Officer at Pharming Group

I'll turn it over to Yiru now to review the financial performance.

Jeroen Wakkerman
Jeroen Wakkerman
Chief Financial Officer at Pharming Group

Thank you very much Anurag and good afternoon, good morning everybody. As you've seen in the previous part of the presentation in the next slide please, Q1 was a very strong quarter for farming. The revenues grew by 42% driven by Ruconext growth of 49% and Joengia by 9% versus Q1 last year. And the key growth drivers were additional demand for both Ruconext and Juengia from increased numbers of patients and the sustained expansion of our prescriber base. The gross profit is up 50%, which is the result of the additional revenues and a gross margin improvement of 4% to 89%.

Jeroen Wakkerman
Jeroen Wakkerman
Chief Financial Officer at Pharming Group

Operating expenses increased because of the non recurring Abliva acquisition related expenses of $7,800,000 Excluding those one off costs, OpEx would be $70,400,000 which is well below the Q4 twenty twenty four OpEx level and in line with our guidance. Again, adjusting for the ABLEVA non recurring costs, the operating profit was $800,000 The net loss was 14,900,000.0 and that increased loss was primarily due to the non recurring I believe acquisition related expenses, most of which were non tax deductible. And the net results were also impacted by foreign exchange exposure and increased tax costs. The cash and marketable securities decreased since the end of the 2024 by 60,500,000.0 to 108,900,000.0 and that was primarily driven by the purchase of the Abliva shares totaling $66,100,000 We had a positive operating cash flow in the quarter for the third quarter in a row, even with the non recurring Abliva cost included. On the next slide, see the financial impact of the Abliva acquisition.

Jeroen Wakkerman
Jeroen Wakkerman
Chief Financial Officer at Pharming Group

So we completed the acquisition of Abliva via a public cash offer to the shareholders to acquire all outstanding shares for approximately $66,100,000 in March 2025. You see the immediate financial consequences on this slide. As mentioned before, the non recurring acquisition related expenses were 7,800,000.0 and that is the bridge between the GAAP operating loss and the adjusted non GAAP operating profit on this overview. The acquisition was accounted for as a business combination with substantially all of the value of the acquisition concentrated in one single assets KL1333. The acquisition price is allocated on our balance sheet to the fair value of the acquired identifiable assets and liabilities and the excess is recorded as goodwill and you see the numbers on the slide.

Jeroen Wakkerman
Jeroen Wakkerman
Chief Financial Officer at Pharming Group

Moving on to the next slide, on the financial guidance, on the back of the strong Q1 results and the outlook on the remainder of the year, we raised the 2025 total revenue guidance to between $325,000,000 and $340,000,000 up from the prior guidance between $315,000,000 and $335,000,000 And this new guidance implies a growth between 914%. We expect operating expenses to be flat versus last year prior to the impact of ABLEVA and we expect $30,000,000 in ABLEVA related operating expenses and that includes research and development costs and non recurring transaction and integration expenses. Available cash and future cash flows is expected to cover the current pipeline investments and pre launch costs and that hasn't changed since the last guidance. With that, I would like to hand back to Fabrice to close the presentation.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Thank you, Jeroen. So as you've heard, I mean we are off to a strong start of the year, which is fueled by the continued strong growth of ReConest and acceleration of new patients on Joyncia, the largest increase since Q2 twenty twenty four. As a consequence, we are raising our full year guidance and we are also setting the foundation for strong financial discipline with as a first step, the decision to decrease annual G and S spend by $10,000,000 to optimize capital allocation and drive sustainable growth. We also continue to invest in our pipeline with the objective to generate two blockbuster assets. The potential new indications for PIDs with immune dysregulation are an opportunity to significantly expand joint JAD addressable patient population beyond APDS.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

And KL1333 in mitochondrial diseases is another asset with a billion dollar plus revenue potential. So as you can see, we are building a solid platform for sustainable growth and value creation with a series of key catalysts in the short and near term. I personally believe that today more than ever, our goal to develop a leading rare disease company is really enriched. Let me finish this call by thanking sincerely Yaron Wakerman, our Chief Financial Officer for his contribution to the growth of farming over the past four years. With this, I'll open the line for questions.

Operator

Thank

Operator

And our first question comes from the line of Jeff Jones from Oppenheimer. Your line is open. Please go ahead.

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

Good afternoon, Thank you for taking the question and congrats on a great quarter. Jerome, one quick clarification for you on the financials. Can you clarify on the flat OpEx for 2025 pre I believe while reducing G and A by 10,000,000 Should we be then thinking about the $10,000,000 adjusting into say R and D? Or how should we think about that?

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

So as I said, actually, this is really an adjustment that we're making on G and A. So general and administrative expenses to really optimize the capital location to drive the growth of our business further. So clearly the goal is not to touch R and D, not to touch our commercial expenses, which clearly are the engine of the short and long term growth.

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

Got it. I appreciate that. And then one thing that is obviously a topic of a lot of discussion right now is the potential for tariffs with The U. S. And given your European manufacturing and most of your sales in The U.

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

S, can you comment on how you're thinking about this at this point and opportunities for mitigation?

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Obviously, I mean, we don't have the details on the size and on the scope of those potential import tariffs. So it's very difficult to to comment without these specifics. What I can say is that we've been working to evaluate a range of scenarios to see really how we can mitigate the risk and the impact of potential tariffs. This could include adjustments in our supply chain, in our manufacturing, adjustment in our custom price. I think it's too soon actually to go into more details, but our goal is to be proactive so we have a set of options if this actually becomes a reality.

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

I appreciate that. I'll jump back into the queue.

Operator

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

Operator

Your line is open.

Joe Pantginis
Managing Director at H.C. Wainwright & Co.

Hey guys. Good morning and good afternoon. Very nice to see the continued growth in the assets and looking forward to the expansion of the pipeline. So, two part question if you don't mind. So first, I wanted to get a sense and this is one of the obvious questions with regard to product growth, potential impact on tariffs right now and on your supply chain.

Joe Pantginis
Managing Director at H.C. Wainwright & Co.

And, as part of that, you saw nice Rukinesq growth in the first quarter. Just curious how, first quarter insurance and Medicare resets might have impacted that? And then the second question is maybe for Steven. You know, as you're looking to continue to drive new doctor, signing ons for RUCONNEST, what are some of the key education or they need to wait to see more factors before they actually sign on for RUCONEST? Thanks guys.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Steve, would you like to comment actually on the Medicare reset and the growth?

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Absolutely. Thank you, Fabrice. Morning, Joe. Thank you for

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

the two questions. So just on Medicare, clearly, as you're aware, the IRA and out of pocket expense impacts bottomed out this year which is good for patients. I would say that it certainly didn't hurt us but it wasn't a major factor in our growth Joe. The growth as I said just came from the underlying strength of the brand but the trend but also the focus that we have on those severely affected patients. So yeah it had some impact but it wasn't major for us and certainly not for Rukinesst.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

In terms of what new doctors like to see, I would say in those either centers of excellence or those locations where physicians have a bigger patient base, then we're already very well positioned for patients that are attacking frequently and for whom other medications just aren't working or they happen to re dose a lot. In some of the other practices where we see growth in prescribers it's often where they only have one or two patients. So it's really the point at which they get reviewed. So it's not the positioning or the profile of the product, it's more the opportunity for that physician to actually use. Which I think is one of the factors why aside from the execution stuff I mentioned you see that consistent growth pre pandemic and then you see it return again when the offices reopen and we're able to get back out there and remind physicians of exactly where Root Connect sits within the market.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

So I think does that answer the question for you, Joe?

Joe Pantginis
Managing Director at H.C. Wainwright & Co.

Yeah, it certainly does, Steve.

Joe Pantginis
Managing Director at H.C. Wainwright & Co.

Okay.

Operator

Thank you. We will take our next question. Our next question comes from the line of Alastair Campbell from RBC. Please go ahead. Your line is open.

Alistair Campbell
Alistair Campbell
Equity Analyst at RBC Capital Markets

Thanks very much, everyone. Thanks for taking my questions today. Just a couple, please. First of all, on Juenga. So a few things happening in Q1.

Alistair Campbell
Alistair Campbell
Equity Analyst at RBC Capital Markets

But maybe if you could just help me understand, as we think about price evolution from 2024 into 2025, what do you think the true underlying net price evolution will be in The U. S? Secondly, on the VUS opportunity, maybe you could sort of talk us through a bit more detail about the process of getting those patients transferred onto paid therapy and how quick you think that can Or is that going to be a bit of a slow build? So maybe just touch on that.

Alistair Campbell
Alistair Campbell
Equity Analyst at RBC Capital Markets

And then finally, Jerome, good luck in the future, Jerome. Maybe one last question before we head off. Given the dynamics on tax, obviously, loss making Q1, but a tax expense in Q1. You help us understand what we should be expecting for the full year? Thank you.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Thank you, Alistair for this question. So I'll let first Jurun answer the tax question and then Steve can provide actually a color on your two questions on Jo and John.

Jeroen Wakkerman
Jeroen Wakkerman
Chief Financial Officer at Pharming Group

Yeah, thanks Alastair for the question. In terms of the tax line in Q1, it was really affected by three key things. One was the I believe related costs that they were non tax deductible. Then we had some share based payment costs that are also non deductible. So that increases obviously the effective tax rate.

Jeroen Wakkerman
Jeroen Wakkerman
Chief Financial Officer at Pharming Group

And we had a release of a deferred tax assets from the past. Now, those topics are all one offs apart from share based compensation that will increase a little bit, but most of it is one off. The high tax cost that we had in Q1 is a one off and we don't expect that to continue in the remainder of the year.

Alistair Campbell
Alistair Campbell
Equity Analyst at RBC Capital Markets

Thank you.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

Thank you, Yuriy. Yeah, Alastair. So just in terms of you asked about net pricing evolution, we expect roughly 75%, seventy six % of our pricing action to flow through to the net line this year. And then in terms of approval evolution, I think as you've seen both within The U. S.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

But also globally in key markets, we've continued to build the funnel. And we've seen an acceleration as both Faris and I mentioned earlier in the call in patients coming on to paid therapy in Q1, which was really nice to see. And that reflects everything that we've put in place in the support services and the reimbursement services, etcetera, etcetera, to convert patients as quickly as possible. The good news is that the vast majority of patients are being converted relatively quickly once they're enrolled and with very little market access barriers being put in place. One would imagine you know the severity of the disease, the lack of treatment options and the fact this is the only indicated product are big factors there.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

But as I said in previous calls it's not linear. You you have patients these are very complicated patients with all kinds of comorbidities. Know we also as I mentioned have a lot of pediatric patients who simply don't qualify right now. So it's not linear. There has been consistency though and Q1 was great to see and you know we expect to see more and more patients converting through the year.

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

And they're very confident in that. Does that help Alistair?

Alistair Campbell
Alistair Campbell
Equity Analyst at RBC Capital Markets

Yeah, certainly does. Maybe, Steven, can I quickly come back? You're talking about '75, '70 '6 flow through to net this year. I mean, how would that broadly compare to what you saw in 'twenty four?

Stephen Toor
Stephen Toor
Chief Commercial Officer at Pharming Group

So obviously gross to nets are quite complicated and are affected by different things. Think it's probably slightly better in the book of business lends itself this year in terms of utilization of government versus commercial patients. So I think you know without having the direct comparison for it it's probably slightly better than last year.

Alistair Campbell
Alistair Campbell
Equity Analyst at RBC Capital Markets

Okay great. Thank you.

Operator

Thank you. Our next question comes from the line of Simon Scholes from First Berlin. Please go ahead.

Simon Scholes
Senior Analyst – Biotech and Resources at First Berlin

Yes, good afternoon. Thanks for taking my question. I've just got one. Just on the gross sorry, on the G and A cost reduction. I mean, given that revenues are going to be presumably going be continuing to grow quite strongly, reducing G and A costs by CHF 10,000,000 is not going to be that easy.

Simon Scholes
Senior Analyst – Biotech and Resources at First Berlin

I was just wondering what elements of G and A you expect to be able to reduce, which elements you were targeting in particular?

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Thank you, Simon for your question. I mean, at this stage I cannot comment actually on the detail of the plan and as soon as I can, I'll share more. It is indeed actually an ambitious plan. I believe it is something that we hope to do. I mean our ability as I said to manage, to ensure that we get the best return on every euro, dollar we invest is absolutely essential to unlock second value creation.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

We have a great growth outlook and it's both about top line and bottom line if we want to optimize the value creation. So doing that exercise, we're finalizing that exercise and when I can share more, I'll share more. But this is a target that we've set $10,000,000 on an annual basis.

Simon Scholes
Senior Analyst – Biotech and Resources at First Berlin

Okay, thanks very much.

Operator

Unless there are any further questions, I'd like to hand back to the speakers now. Thank you.

Fabrice Chouraqui
Fabrice Chouraqui
Chief Executive Officer and Executive Director at Pharming Group

Listen, with this, I think we're going to close our call. Thank you so much for your interest in the company. Thank you for your questions. We look forward to updating you on our plans and probably I'll see many of you in person through one on one meeting or at investor conference in the coming months. Thank you very much.

Operator

Thank you. That does conclude today's conference. Thank you for participating. You may now disconnect. Speakers, please stand by.

Executives
    • Fabrice Chouraqui
      Fabrice Chouraqui
      Chief Executive Officer and Executive Director
    • Stephen Toor
      Stephen Toor
      Chief Commercial Officer
    • Anurag Relan
      Anurag Relan
      Chief Medical Officer
    • Jeroen Wakkerman
      Jeroen Wakkerman
      Chief Financial Officer
Analysts
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Key Takeaways

  • Pharming delivered 42% total revenue growth in Q1, with RUCONEST sales up 49% to $68.6 million driven by new patient enrollment and a broader prescriber base.
  • JOENGIA revenues rose 9% to £10.5 million in Q1, adding six new US patients—the largest quarterly increase since Q2 2024—with upcoming catalysts including US VUS reclassification, pediatric label expansion, and global launches.
  • The pipeline advanced as two Phase 2 studies for leniolusib in primary immune deficiencies with immune dysregulation began enrolling and enrollment resumed in the registrational trial for KL-1333 in mitochondrial disease.
  • Management narrowed operating losses for the third consecutive quarter (ex-non-recurring acquisition costs) and initiated a $10 million annual G&A cost reduction to enhance financial discipline.
  • Based on Q1 performance and margin improvement, full-year 2025 revenue guidance was raised to $325–$340 million from $315–$335 million.
AI Generated. May Contain Errors.
Earnings Conference Call
Pharming Group Q1 2025
00:00 / 00:00

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