Pharming Group Q1 2023 Earnings Call Transcript

Quartr
Provided by Quartr
May 11, 2023

There are 8 speakers on the call.

Operator

Good morning or good afternoon, ladies and gentlemen. Welcome to our Q1 2023 results call. And before I go into the call, I would like to have the next slide and show you the forward looking statement slides as we will be making some forward looking statements that are based upon our future expectations and our current expectations and assumptions. And as you know, they may change in the future. And without having said that, next slide, please.

Operator

I'm here with my 3 colleagues, Anurag Reylan, Chief Medical Officer, Stephen Thor, our Chief Commercial Officer and Yaron Wackerman, our Chief Financial Officer. And they will be speaking after me, and I will start with a brief introduction. So next slide, please, and then the next one as well. So basically, what farming is all about is that we are building a sustainable rare business. And that rare business will be able to be funded from the positive cash flows that we continue to generate from Ruknest.

Operator

And you have seen over the last quarters that these cash flows from Ruknus have helped us to prepare and fund and the launch of Joenja in the United States and the further pipeline development that we have been doing. So we're in a very favorable position. And today marks the Q1 in which we have been able to actually get Joanna approved and where and the last quarter where we will be reporting on sales from our single product because going forward, as you have seen from the press release, we will start recording sales for Joenja in the United States as well. It's an important demarcation, I would say, in the history of our company that we are now going to work to get revenues from 2 products, albeit for the time being on only one major geography, the United States of America. But that will soon change as well.

Operator

So therefore, you can see what we're up to. We're up to successful commercialization of Giovinja for APDS and Anurag Rellon will talk about that later and additional rare disease indications where we cannot give you any specifics yet, but we will do that in the second half of the year. And of course, we still are looking for additional projects that are in mid to late stage development in rare diseases to actually further fill our pipeline and use and leverage further our commercialization infrastructures that we have in the U. S. And in Europe and that we are building up in additional markets as well.

Operator

If you look at the next slide at the pipeline, you see that it is and we have now specified it in terms of the various LENIOLE SIP activities that are taking place outside the United States, you see that we have a lot of stuff on our plate and a lot of things to look forward to. And 1st and foremost, of course, the radialisib approval in the European Union and the UK, but also the pediatric projects, the Japan projects and Canadian and Australian projects and last but not least, the additional indications for lenoisib. But it means that we can actually further down the line have space in the capacity and our commercialization capacity to launch additional products. Hence, why we are very continue to be very active to look for additional in licensing opportunities in other rare diseases and or merger and acquisition opportunities in the market. Let me just go back to for a moment, go back to Rokonetsen, And that is why we are so very proud, next slide please, of the product like Eurkinesq because it helps us getting a $200,000,000 business, if you look back last 12 months, with an outlook for single digit revenue growth.

Operator

And it continues to take a unique place in the market because it's the only recombinant treatment that feeds the root cause of hereditary angioedema by replacing that missing protein, that dysfunctional or missing C1F esterase inhibitor. And it has proven over the years to be well tolerated and effective. And it is the 2nd most prescribed product detailed for the Q2 tax. And you can't come much closer to 100% efficacy and reliability. And that's important because patients are relying on rucanest where they have either a very severe form of the disease and have very high frequency attacks and cannot get by with the significantly improved prophylactic therapies or which increasingly is the case, they actually rely on RUKUNAZ for being able to treat their breakthrough attacks, which almost half of the patients still suffer from in very varying frequencies, they rely on Rukunest as their breakthrough medication.

Operator

That's the increasing there we see an increasing use of Rukunest. Under that, Thank God for the patients who strongly improved prophylactic therapies. And we can do all these successes here because we have, as I was already alluding to, very strong commercialization infrastructure. Next slide, please. That is consisting of all these functions that you need.

Operator

And you see on the slide here, all these functions that you need to be successful in commercializing rare disease assets. And that is where today We look forward with very much confidence to further success of growing the company. And with the next slide, please with the approval of leniolusib recently. We now are embarking on a growth trajectory going forward because, as I said earlier, we will now be able to report on 2 products that are generating revenue for our company. Our strong ROOCADAS franchise that continues to show single digit revenue as we expect for this year.

Operator

And on top of that, we are expecting Joy India sales from Q2 onwards to be increasing going forward. And it's now time, I think, to hand over to my colleague, Doctor. Anurag Rallen, our Chief Medical Officer, to dive a little bit into the APDS and into JoAnja. Anurag, over to you.

Speaker 1

Thanks, Simon. Before we look at the launch of JoAnja, let's review a little bit about APDS. And we can see it on the next slide that APDS is a rare primary immune deficiency, also known as an inborn error of immunity That was only first characterized just a little over 10 years ago in 2013. We estimate that it affects approximately 1500 patients in some of the Countries that you see listed there and that's based on a prevalence of 1.5 per 1,000,000. To date, we have already identified more than 500 of patients across these areas.

Speaker 1

The treatment options until recently have been quite limited for APDS patients. We'll talk a little bit about that. But this is really the treatment has been limited because it's only been focused on the symptoms of the disease. These symptoms begin early in childhood, but do not address the root cause of APPS. And the signs and symptoms vary, and we'll talk a little bit about what those look like, but they vary even within a family.

Speaker 1

And this results in significant delays and especially due to the delayed onset of the diagnosis. The diagnosis itself is actually quite simple to make when the clinician thinks of it and that's really through this genetic test that can provide a definitive diagnosis. Next slide. As we see here, APDS really impacts patients in many ways. There's, of course, the physical manifestations and you see that really in the top left With the recurrent infections that these patients have, the enlarged lymph nodes and glands, they have numerous infections that lead to damage in their lungs and a whole slew of symptoms there that you can see due to this progressive serious disease that develops early in childhood.

Speaker 1

On top of the physical manifestations, there's a real Impact on these patients' quality of life. Of course, there's the social aspects where they can't work or can't go to school or do their normal daily activities. There's a mental aspect of being afflicted with a chronic disease with where The treatment up till now has really been focused on the symptomatic management. And then lastly, the treatment burden, Frequent hospitalizations, unnecessary surgeries many times, numerous doctor visits just to get a diagnosis and then really being limited in terms of what can be On the next slide, we can see what causes APDS. And this is due to a genetic defect at least to this hyperactivity of this pathway, the PI3K pathway.

Speaker 1

And that when that pathway is overactive, That results in this dysregulated environment for the BMT cell to develop properly. When these Cells of the immune system don't develop properly, you see a chain reaction set off. And on the right side, you can see all of the Symptoms that result as because of this hyperactive pathway leading to this immune imbalance. Of course, it's a primary immune deficiency, so you see recurrent infections. These can be in the lungs, in the upper respiratory tract, the lower respiratory tract.

Speaker 1

It can be also be commonly associated with herpes viruses, especially EBV and CMV viruses. One of the hallmarks of the disease is lympho proliferation. So what we see that as in these patients is swollen lymph nodes and enlarged spleen and also disruption of their lymphoid tissue across their body. They also can have gastrointestinal manifestations And commonly because the immune systems are immune system is not functioning properly, they have autoimmune issues including cytopenias and other autoimmune disorders. And as I mentioned earlier, bronchiectasis, which is a complication in their lung, where this is irreversible also commonly develops in these patients.

Speaker 1

The most severe manifestation and what often takes these patients' lives is the development of lymphoma due to this unchecked lymphoproliferative process and this Immune imbalance that occurs as a result of this disrupted PI3K activity. On the next slide, we can see sort of the treatment options that were available to manage these patients prior to the approval of GYNJOLIA in the U. S. On the one hand on the left, you can see it was limited to trying to address the immune deficiency. So using antibiotics to prevent infections, to treat infections, But also using immunoglobulin replacement therapy as a way to augment their own immune system.

Speaker 1

On the right side, you see the issues that they were faced with these patients in terms of immune dysregulation. So trying to control the immune system with steroids or other immune suppressments, including drugs like mTOR inhibitors. None of these therapies, however, were approved for APDS treatment. And in the rare cases, some of these patients were given a stem cell transplant, although transplantation itself is a high risk procedure in these patients. On the next slide, we can see what JOLANJA now offers.

Speaker 1

And it's an immune modulator that addresses the root cause of this Hyperactive pathway, excuse me, in these patients and is designed to treat that cause by normalizing this pathway, this PI3K delta pathway. As a result, what we see is a normal balance of the development of the immune system cells. And we can see that when we measure the immature cells and the functional cells, Now they progress normally through their development path. And on the next slide, we can see The summary of what this Joenja approval now offers to patients. It is indicated for patients who are 12 years of age and older who have APDS.

Speaker 1

I'll be reviewing with you some of the randomized data, but it met the randomized study met both primary endpoints. And we're also going to review some of the secondary endpoints and exploratory measures that we're seeing in this study. The drug was generally well tolerated and there weren't steady withdrawals due to drug related adverse events. And on the right side, you can see some of the other data that we generated with Joanna, specifically that would have long term data showing reductions including discontinuations in the use of immunoglobulin replacement therapy and as well as reductions in infection rates. These study results were consistent with what was observed in the double blind placebo controlled study, including long term data on lymphadenopathy as well as some of the immune phenotype.

Speaker 1

And as Steve will report in a few minutes, We are well positioned to hit the ground running with JOLANJA. Next slide. Here is a depiction of the label as well as the packaging. And on the next slide, we can see some of the data from the randomized control study. As I mentioned, Joenja met both co primary endpoints, which saw a reduction in the lymph node size on the left as well as an improvement in the naive B cell count compared to placebo.

Speaker 1

This strongly indicated a correction of the underlying immune defect. And you can see that when you see the size of the lymph nodes decrease relative to placebo as well as on the right side, you can see how the naive B cell proportion increased in these patients, again, relative to placebo. Both measures were statistically significant and these were both the these were the 2 co primary endpoints in the study. Next slide. And when we look at the open label data, what we saw is over time a reduction in the number of days that these patients had infections over the course of the year, and we saw that reduce the longer that they were on JOINJA.

Speaker 1

At the same time, what was observed and this was again Spontaneous really in the study where these where physicians and patients were able to stop using in many cases IRT therapy and many of them also reduced the use of IRT therapy as the study progressed. Next slide. Now looking ahead, we have a number of other milestones later this year. Steve will report on the launch that's been started just last month. And as Simon mentioned, We are under review under Aetna Europe and we're continuing to expect a CHMP opinion later this year with an approval 2 months later.

Speaker 1

We also expect to file in the UK later this year with an approval soon thereafter. We're also expecting to start the Japanese clinical study, which is a small study in up to 5 patients to support a regulatory submission there, and we'll be doing that in the first half of this year still. We, of course, had started earlier this Here a pediatric study in children ages 4 to 11 and that is going on. And we expect to start our 2nd pediatric study in the Q3 of this year in even younger children. Next slide.

Speaker 1

Turning now toward another program that we have that's an earlier stage program and this is a partnership with Orchard Therapeutics to develop an ex vivo autologous Stem cell gene therapy for HAE. We'll continue to make progress on developing the vector here to enhance expression levels And that vector is now being tested in a number of HAE disease models in animals. And we anticipate being able to provide further updates as we move toward preparing an IND filing later this year. Next slide. And I'll turn it over here to Steve to give you a commercial update.

Speaker 2

Thank you, Anurag. Good morning, everybody. Over the next four slides, I'm going to provide you with an overview of the Q1 RUKENAS performance and some early insights as to the progress of the Joangel launch just 6 weeks after approval. As you're aware, There were market wide issues that impacted some government insured patients, resulting in a delayed product shipments. Our internal data and external audit data show significant declines for all acute prophylactic products and sorry, all acute and prophylactic products and that impacted all companies serving patients.

Speaker 2

The issues were resolved late in the quarter and as affected patients started shipping, root canal sales accelerated and the product staged the highest positive return or bounce back of all the acute products in the market. With the Q1 market wide issues and disruption behind us, we saw good sales in March and also strong sales in April as the recovery continued. And we also, of course, as a slide alludes to see strength in the underlying business, more especially high volumes of new patient enrollments and growth in prescribing physicians. We therefore expect sales to strengthen through Q2, and we continue to forecast low single digit revenue growth for RUKE NEXT in 2023. So now let's turn to the Joendra launch.

Speaker 2

As can be seen in this slide, Pharman is bringing all of its rare disease commercialization Our first of many launches on a must win market. We have 54 salespeople and sales leaders, And that's comprised of the RUKENESS sales team, where we think 30% of patients are treated by customers already very well served by farming And the new Joinger institutional team. And this team focuses on central locations or centers of excellence, to which we expect the other 70% of APDS patients to either currently be treated or be referred to. And between these two teams, we have the vast majority of the APDS market covered in the U. S.

Speaker 2

And importantly for you to know, our sales colleagues are comprised of experienced rare disease specialty and hospital representatives And sales leaders with launch experience and importantly patient finding experience. And as with the Roux enest team that successfully turned around The brand on reacquisition from Valeant, we've stopped that team with award winning salespeople to drive a successful launch. So They're our feet on the street. They're out there identifying patients. Importantly, as you see here, we also have clinical educators to drive family mapping and family testing.

Speaker 2

This is critical because this is not a similar dominant disease. So other members of the family are highly likely to have APDS. And it's important for them that they get access to Joenja as quickly as they can. And of course, it's an important source of new patients for pharma. We also have a dedicated full service concierge patient services program that ensures once a patient is diagnosed, there are 0 distractions And challenges to addressing or to getting Joandrea into a patient's hands.

Speaker 2

And I think that's important in what is a complex market to navigate. The program covers all of the basics, so the filling of prescriptions, financial aid and ongoing to support to ensure adherence and continued access to medication. And in terms of staffing, this is where we believe we're really differentiated from many of our competitors in the rare disease space. We have care coordinators providing a single point of contact often the same person, delivering consistent service and care and providing reassurance to patients and their families. Mass versus the more traditional commoditized call center model.

Speaker 2

We have the clinical educators, as I've mentioned already, there to support and educate patients And importantly, we have clinical pharmacists that will be available 24 hours a day to process Joinsa prescriptions, answer any questions patients might have And speed up approval rates, which having these guys on team really allows to happen. Importantly, we've also farmed with Panther Rx, many of you are familiar with the U. S. Market will who you'll know. They're an excellent value partner specializing in rare and ultra rare conditions.

Speaker 2

And that gives them unique insights and really helps them to deliver for our patients in the way that they expect and pharma expects. So this dedicated program and staff should speed access to medication, minimize bureaucracy and mistakes and cater, as I said, to the very Specific needs of these patients. Next slide, please. Before I get to the early results, I just want to talk briefly about the Value proposition for Joenja, which Anurag articulated very clearly earlier. So we should remember that Joingy is the only indicated treatment for APDS.

Speaker 2

It's a precision medication. So when the patient tests positive, The HCP and the payer know they're prescribing and approving the right treatment option for the patient. And Johengis disease modifying, it's working on the root cause of APDS, as Anarik said, for both immune deficiency and dysregulation. So, pharma, therefore, is launching a part of the physicians and their underserved patients need. And we have, as I've outlined, hopefully, the right infrastructure and services to get So let's look quickly at the progress so far.

Speaker 2

Next slide, please. So I think as you all know, the launch of market preparation was rigorous and thorough. And as expected, we're off to a very good start. So our first fully reimbursed commercial shipments of Johengria occurred just 2 weeks after FDA approval. To date, we've shipped to 23 patients, all on payer approved products, about half are from the early access or open label extension programs, And we continue to make good progress transitioning these 25 patients to paid product.

Speaker 2

The other half of those SHIP patients are patients that are new to Joenja. So most of the EAP patients are enrolled on paid therapy and we're steadily working through the OLE patients and all this while simultaneously building a new patient caseload. Importantly for the U. S, in the area of market access or managed care, we continue to make good progress with national and regional payers, including state Medicaid programs to prepare for clinical review of coverage policy development, and we expect to see those developed in the next 90 to 100 days. In the meantime, patients have been approved pretty quickly through the medical exception process.

Speaker 2

Looking at Medicaid specifically, Our teams have done an excellent job getting Joenger covered for APDS patients with already 2 thirds of the states listing the product in just 6 weeks. So as you can see, we have prepared, we're off to a fast and impressive start, only 6 weeks since we launched Joengine. And I Greatly look forward to updating you on Joenger launch progress later in the year when we can share the Q2 results for both Rupenest and Joenger. And with that, I'd like now to hand over to Jeroen, who will cover the financials.

Speaker 3

Yes. Thank you very much, Steve, and good morning, good afternoon. As next slide, please. As Steve mentioned earlier, as you can see on this slide, the Q1 results were lower. And that was due to the HAE market factors, which impacted the entire industry.

Speaker 3

And those industry wide factors have since resolved and we can confirm that we have strongly recovered. And if we look at the quarter, January was in line with last year and February is where we faced headwinds. March had a strong recovery and so had April. We've almost made up all of the shortfall and expect to recover the remainder. We therefore continue to expect single digit growth in recognized revenues for 2023.

Speaker 3

And on the slide, you see that the revenues in Q1 were €42,500,000 that's 9% down on last year for the reasons I mentioned. Gross profit developed in line with that. It went down by 8% to €38,500,000 And the operating costs increased from €40,000,000 to almost €53,000,000 and that was on the back of Lend dealership, both in R and D Investments and in sales and marketing costs. Operating profit and net profit reduced and the operating loss was €13,700,000 and a net loss €12,200,000 So the short of this quarter is that the sales shifted from Q1 to Q2, and we've seen that in April. And with regards to costs, we're investing in LENIELLE SIP.

Speaker 3

And obviously, we haven't recorded any revenues in Q1 yet for Leneodice and Kyrenja, but that will change in Q2. If we then go to the next slide, please, on the cost development, you see that we are continuing to invest in the launch of Jovenja. If If

Speaker 1

you look

Speaker 3

at the longer term trends over the quarters that are shown here, starting with the R and D bracket At the bottom, we see a reduction in quarterly R and D costs in Q2 and that is because of reduced investment in the Transigenic platform. You see a uptick again in Q1 this year because of leniolisib. Looking at the G and A, general and admin cost developments, we've seen a slight growth per quarter over the last quarters, which basically means investment ahead of company growth. Q1 was higher than last year, Q1 2023, but lower than previous years. And the big number in Q4, by the way, the 17.6% that you see is because of an impairment cost of a building.

Speaker 3

So that's not a repetitive cost. The marketing and sales cost, the biggest We've seen a quarterly growth of marketing and sales costs in 2022 with more investments in the Joenja launch, especially obviously, in Q4 last year. And I should note that the marketing and sales expenses for the U. S. Launch are high in this period, also in Q3.

Speaker 3

And going forward, we will see an increase in the marketing and sales costs in other key markets, namely Europe and the UK, in the trailing quarters as we prepare for launch. To get an indication of OpEx levels for the remainder of the year and therefore for full year, the Q4 2022 and the Q1 2023 OpEx levels are good indicators, albeit it may increase moderately. If we then go to the next slide, it's about the cash flow. The cash went down from €207,000,000 to €185,000,000 at the end of Q1. And the key reason is the net cash flows used in operating activities.

Speaker 3

The cash Loss was €10,200,000 from operations. Working capital increased by €12,000,000 that was mainly because of an increase in ESG and debtors and the latter was because of phasing. The cash flows used in financing activities is due to regular interest and lease costs, and we have some positive foreign exchange effects, bringing the cash to $185,000,000 Then the outlook on the next slide, We continue to expect low single digit growth in recognized revenues for the full year. Joenja was approved in the Q1 on 24th March by the FDA and we have been commercializing in the U. S.

Speaker 3

Since early April 2023. We expect in Europe a positive CHMP opinion in the 2nd half of this year and the marketing authorization to follow 2 months later. Subject to the positive outcome of the CHMP review, we will file for UK approval with the MHRA. We will continue to invest in future growth and to accelerate it, and that will obviously be focused on the Joenja launch. And we will provide further details of our plans to develop lenulosib in additional indications in the second half of this year.

Speaker 3

And to finish off with, we will continue to look for late stage In rare diseases, be it in licensing or in potential acquisitions. So we're still open for investments very much in that area. With that, this concludes the presentation. And I would like to go to the next and open up for questions and answers to any of the people attending the call from the pharma side. Thank you very much.

Speaker 4

Thank

Speaker 1

Our

Speaker 5

first question comes from Alastair Campbell of RBC. Alastair, your line is now open. Please go ahead.

Speaker 4

Thanks very much. A couple of questions, please. First of all, on Ripco, just so I understand what's going on here. Is this a feature of basically sales, which would have happened to you or is there actually genuinely a shortfall in sales? Just trying to understand if that had if the disruption hadn't happened, would you be more likely to get maybe something like mid single Low single digits.

Speaker 4

Question 2 is, I know it's very, very early, but just to get a bit of insight into the patients You put on to Joandra. At this stage, do you have sort of a sense of severity of those patients? Are they sort of Across the spectrum of severity or do you tend to come from year end? And if I could push my luck, you've got 23 patients on therapy now.

Operator

You were breaking up. You had a third question, Alsern?

Speaker 4

Sorry, third question was, Given you've got 23 patients on Durenger now, what do you think would be a good exit number for the end of the year?

Operator

Thanks. Let me answer the last one, and I'll go back to Stephen on the first two questions. We don't give as you can appreciate, it's early days, and we don't give any sort of forward looking statements on what we think is a reasonable number. I think it's too early. But you obviously agree with us that already having 23 patients on paid therapy and many more in the enrollment process gives you a good indication that we prepare to launch very carefully.

Operator

You also heard Stephen talking about how well we are progressing with getting the reimbursement sorted for Joenja. So let's keep it at this. And obviously, over the coming quarters, we will continue to report on those patient numbers. And going forward, we will give some more indications as and when we see a clear trend arising. So that's the answer to question number 3.

Operator

I'm sorry, I can't go in any further detail. And I would like to go back to Stephen about your question with regards to Rukanesque sales in the Q1 and the if there's any sort of differentiation in the severity of your Joanna patients. Steve, over to you.

Speaker 2

Thank you, Simon. In terms of the root canal sales, I really think our guidance wouldn't have changed. There was disruption in the Q1, which pushed patients out a little in terms of Delivery of product. So at this point, we're playing catch up. So I think the guidance would remain the same as it was in mid to late March when we last gave it, which is Low single digit revenue growth.

Speaker 2

In terms of severity of patients, I don't have deep insights. What I would hypothesize and perhaps invite Anurag, if he's got anything to add, is that these are patients largely already identified. At least half of them were in EAP or in the open label extension. So they were identified, therefore they were exhibiting symptoms and were at least at the moderate end of the scale.

Speaker 1

Yes. I think you got it. Yes. That's

Speaker 4

correct.

Speaker 1

Sorry. I was just going to

Operator

Go ahead, Ana. I think

Speaker 1

the key point is that there was a mix of patients here. So we have patients who were in the study that were have now started Joenja commercial paid product. We have patients who were in the expanded access program and we also have naive patients. So these are patients who are not in the expanded access program or in the study, we're now receiving Joenja leniolusib for the first time.

Operator

Okay. Did I answer your question, Alastair?

Speaker 4

That's good. Thank you.

Operator

All right. Thank you.

Speaker 5

Our next question comes from Cecilia Hernandez from VLK. Cecilia, your line is now open. Please go ahead.

Speaker 6

Yes. Thank you for taking my question. On Recones, could you expand on the reimbursement disruptions affecting the HEE market Since Takeda showed an increase in tax euros sales this quarter, what were the circumstances that led to a more pronounced disruption on your end? And a second question, you mentioned that you expect quarterly fluctuations for Reconyx still. So what are the drivers behind these fluctuations that you are anticipating?

Speaker 6

Thank you.

Operator

I hand this question back to Stephen.

Speaker 2

Certainly, I mean, the disruption was primarily in the government sector, and some of those Patients saw disruptions in the co pays and the cost of accessing medication, which is what resolved itself as we went through the quarter. I can't comment to the company to the sales, obviously, of other companies. What I will say is if you look at Symphony Data, for example, and specifically their Metis database, You see that every company saw significant disruption in Q1. So I can't comment to that, but I can say that The disruption was specifically within the government insured patient sector and it resolved satisfactorily and all those patients certainly on the Rukunus side and they're receiving their medications.

Operator

And the other question,

Speaker 6

Yes. So indeed, you mentioned that you expect quarterly fluctuations of frequent sales. So what are these drivers behind these fluctuations that

Speaker 2

Apologies, I missed that. So it's the it tends to be, obviously, more a combination of seasonal and just the length of So for example, we tend to see some dips during the significant U. S. Holidays, so Independence Day, Thanksgiving, around Christmas. So it's not and also during the holiday season as well, where patients will stock in ahead of going on vacation And then not order as much during the periods when they're away.

Speaker 2

So it's and I think it's the fact we're not driven by the law of large numbers, right? We have amount of patients and when the change in their order and patterns changes, then you see changes in our order rates and therefore quarterly fluctuations. So there's nothing of real significance beyond that.

Speaker 6

Okay. Thank you.

Speaker 4

Thank you.

Operator

Does this mean there's no more questions?

Speaker 5

Our next question comes from Simon Scholes of First Berlin. Simon, your line is now open. Please go ahead.

Speaker 7

Yes, hello. I see you've taken the decision to Discontinued pump. I remember a few years ago, you expected your product under development to have quite a benign side effect profile compared with the Current market leader. I was wondering if you could comment on your decision to discontinue Given your expectation of those of that positive Cytoplank profile or and also, whether you took the decision to discontinue Maybe because it would have taken too long to bring the product to market if that played a role?

Operator

Yes. It's a good question, Simon. I think there's still significant unmet medical needs in Pompe. So that's not necessarily the case. We did not simply see the differentiating features that we felt confident enough to go forward with investing in in the project.

Operator

So therefore, we basically decided to stop and abandon this project. And there's also, of course, new developments on the horizon where other than protein replacement therapy, for instance, the GYS1 receptor antagonists are being developed as we speak. So we thought it was appropriate to stop this bearing, but not seeing any of the differentiating features. That was the main reason to make the decision.

Speaker 7

Okay. Thanks very much.

Operator

Any more questions,

Speaker 5

There are no further questions. I'll hand back to the management team for any closing remarks.

Operator

Okay. Thank you very much. Ladies and gentlemen, thanks for attending this Q1 results conference. As I was saying in the beginning of the call. This Q1 marks a clear demarcation.

Operator

We got our 2nd product approved here. We have an established commercial station infrastructure in the U. S, and we're building that up in Europe. So we're preparing rigorously for the launch of Pliniosip outside the U. S.

Operator

I hope you agree that we are off to a very good launch with regards to Joenja in the United States because of the fact that we already have these 23 patients on pay therapy within 6 weeks after launch, which is not a given in rare diseases, and many more are already in the process. So we look forward to very much forward, I would say, to coming back to you next quarter and report on not one, but 2 products that will drive our revenue. And of course, last but not least, we remain confident. I would like to again say that we remain confident in the robustness of our Arukenas business going forward. So thank you very much for being here again, and we look forward to updating you again on the next quarter results in the beginning of August.

Operator

Thank you. Goodbye.

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