Pharming Group H1 2023 Earnings Call Transcript

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August 3, 2023

Key Takeaways

  • Rukenest revenues rebounded strongly with 20% growth in Q2 over Q1, underpinning the company’s confidence in achieving its low-single-digit growth target for the full year.
  • The FDA approved Joenja just before its PDUFA date, enabling a rapid U.S. launch that enrolled 60 APDS patients and put 43 on paid therapy by June 30, with no reimbursement denials to date.
  • Foresight into regulatory milestones includes submissions in Canada, Australia and Israel, an open pediatric trial for 4–11-year-olds (and another planned for ages 1–6), and ongoing FDA discussions for a second leniolisib indication.
  • Leadership was strengthened with the appointment of Dr. Richard Peters as chairman-elect and Alexander Breitenbach as chief business officer, enhancing the company’s capacity for rare-disease in-licensing and growth.
AI Generated. May Contain Errors.
Earnings Conference Call
Pharming Group H1 2023
00:00 / 00:00

There are 10 speakers on the call.

Operator

Good morning or good afternoon all and welcome to Farming's First Half twenty twenty three Results Call. My name is Adam and I'll be your operator for today. I will now hand the floor over to Simon DeVries to begin. So Simon, please go ahead when you are ready.

Speaker 1

Thank you very much, Adam. Good morning or good afternoon, ladies and gentlemen. Welcome to our Q2 and first half twenty twenty three financial results call. Next slide, please. I'm here with my 3 colleagues, Doctor.

Speaker 1

Anna Magallalan, our Chief Medical Officer Stephen Thor, Our Chief Commercial Officer and Jeroen Wackerman, our Chief Financial Officer, to take you through the highlights of these results and of course, to answer all your questions that you may have afterwards. But before I do that, of course, I would like to point you to the next slide that says something about forward looking statements because we will be making some forward looking statements, of course, today that are based upon our current plans, belief, market circumstances, etcetera, that may, of course, change towards the future. And then without further ado, I would like to move on to 2 slides ahead and basically share with you some of the excitement Over the last 6 months that we had, of course, and we had a very busy 6 months and indeed made a lot of progress. And but first and foremost, We're, of course, very, very pleased to see that Ruckenest did it again, so to say, 20% growth Over the Q2 results versus the Q1 is, of course, a very spectacular recovery from what was a one off weakness in the Q1. And that means that if you look at it, we're very confident that we can actually continue to be on track for the low Single digit growth for the entire year.

Speaker 1

And we say that based upon the leading indicators that all point in the right direction. Of course, Stephen We'll talk a little bit more about that later in his part of the presentation. Now that Significant cash flows, of course, that Ruknest has been generating over the years not only helped us to back in the days of 2019 In license Joenja from Novartis, but also helped us, of course, to finance the development of Novartis and of course of Joenja And of course, to actually prepare for the successful U. S. Launch.

Speaker 1

And that's exactly what happened. The FDA approved the product A couple of days before the PDUFA date, and we were very quickly off to a strong start in the market. And you've seen that. And I dare to say that in the Ultra Rare business, having already 43 patients on paid Therapy in the Q1 that you report results out of 60 that are already in the pipeline as of 30th June is no mean feat and expectation since so far that I've seen analyst reports predicting 1st quarter sales for Joenja. We've also made a lot of progress, Of course, in the regulatory reviews, we've submitted the file to a number of other territories, Canada, Australia and Israel.

Speaker 1

And of course, you made strides forward with the pediatric program, of course. And that's important because the current although the current license For let Joanneja already incorporates the 12 years and upwards. So already partly the pediatrics, there is a big unmet medical needs to be fulfilled in those children younger than 12 years. And we're working very hard to actually get that pediatric program done so We can actually submit the file for the extension with the towards the younger children. And then of course, point that we believe that we can achieve with leniolusib, that is the second indication.

Speaker 1

And we, as you may have read already, have been already submitting our plans and have initiated discussions with the FDA on how on Our proposed development program for the second indication, and Anurag will come back to that a little bit later. And of course, last but not least, That can actually further leverage our commercialization structure that we have in place. And next slide, please. There you see a visual of the pipeline that has now changed, of course, but still, of course, under review with the in the European Union and the U. Okay.

Speaker 1

The pediatric trial is, of course, has started. LENIOZ of Japan, we're on the brink of starting that trial that we agreed with the Japanese authorities to do, Anurag will come a little bit later on, on Japan. And of course, you see here that we made progress, as I just alluded to, By submitting the files towards the Canadian, Australian and Israeli authorities, So just to increase the footprint for LENIO or the SIP or Joenja, it's called later in the geographical footprint. And then of course, we also made great strides forward, next slide please, with our leadership, strengthening of our leadership. We're very pleased To have found Doctor.

Speaker 1

Richard Peters as a new Chairman-elect to succeed Paul Zachary, who came to his maximum term as allowed under Dutch law as Chairman of our company. As you can see From the outline on Doctor. Peters, he's got an impressive track record, a lot of experience In the healthcare industry, but also in academia, but especially in the rare diseases business with very successful companies such as Genzyme or Sanofi Genzyme as it's called nowadays, he's been CEO of 2 NASDAQ listed by the companies. As you can see, He held positions in earlier successful companies such as Amgen and Sanofi. And of course, he is a medical Doctor by Education has actually also served as an editor for very prestigious journals.

Speaker 1

So we're very pleased That Richard was happy to join us as our Chairman Elect. And of course, we will organize An extraordinary general meeting of shareholders in the not too distant future to actually get him elected. And then We're very pleased as well to and that is especially in the context of our growth strategy and our ambition to actually in license or acquire additional late stage assets That's for rare diseases, but Alexander Breitenbach has agreed to join us as our Chief Business Officer, who will be tasked with Yes. The growth strategy the development and execution of our growth strategy and our future plans. And he's a very experienced individual as well, Several senior positions, most recently as Chief Businesses Officer and Chief Development Officer at ACM Biosciences, a Swiss Basel based company, but also a very distinguished and long career in Roche, in Basel, In the Business Development and the Business Development Group.

Speaker 1

So we're very pleased to have strengthened our team with these two Extremely experienced and talented individuals. Next slide, please. Let's just look at and then the next one, please, at what it actually means and what it brings and what we need and what is our bread and butter. That's our very strong rare disease commercial infrastructure. We have what it takes to actually be successful in this.

Speaker 1

We've already proven that, and we're about to prove that again With Joandra going forward. So we have these dedicated sales forces in the U. S, European Union and also we have people in the Middle East and North Africa Calling on these immunologists and academic hospitals. We have medical affairs team, and that is medical affairs team. So that's, of course, very important, Especially in rare diseases, you have a very strong medical affairs team that include medical directors, medical science liaisons, Molecular Genesys and Publication Specialists, very big medical teams that are Absolutely essential for your success in commercialization of rare diseases.

Speaker 1

And then, of course, as Market access becomes more and more important. We have built up our market access teams over the last few years on both sides of the oceans. They include national account directors in the U. S, health, economic research specialists and directors, both sides of the ocean, to actually work with the authorities to get the product to reimbursement. And you've seen how quickly we got the product reimbursed in the U.

Speaker 1

S. So I think we have an and team in place there. And then there's patient support teams at Hub Services in the U. S, reimbursement managers, Patient Care Managers, we have a 3rd party nurse force to take care of our patients and clinical educators. And then last but not least, we're extremely active With everybody, including our sales force teams, to be at conferences.

Speaker 1

And there's a lot of education disease education ongoing also directly to patients That we actually facilitate. We support the patient organizations and all. So basically, it's an extremely well oiled team And that has shown, for instance, with the launch of Bleniolyship, they know what they have to do given the fact that we got it immediately reimbursed and found and identified those patients in a new disease, right? A disease was not even discovered 10 years ago. And as you can see on the next slide, we're really Ready to leverage all of this for Joenja as and when Joenja gets approved, of course.

Speaker 1

And As you heard earlier, we've also now decided to expand to Australia, Japan, of course, and Canada that are not yet on this map because it's just the established business. Okay. And then one more slide from my end here about the durable commercial asset that Ruganes represents. It's quite rare, I would say, that a product that's already 9 years in the U. S.

Speaker 1

Market is still growing and is still We're getting more and more meaningful. Why is it getting more meaningful? And why is it growing? Because it has a very special place. It's the only recombinant protein replacement therapy that is available in the hereditary angioedema market.

Speaker 1

And that's very important because although there is a big trend has been going towards prophylactic treatments for hereditary angioedema, and those prophylactic treatments have become better. There's also The notion and the fact that almost half of these patients suffer from so called breakthrough attacks under their prophylactic therapy. That's when it becomes important to have your breakthrough medication at hand at all times. And this is actually where RECONEST is now beginning And beginning to make inroads and being used for breakthrough medication. And that is a very important aspect because it means that more and more patients are discovering That it is the right drug to have for your breakthrough as breakthrough medication and that more and more physicians are actually discovering that RUQUEENESS is the right choice And yes, RUQUEENEST is an IV drug that is self administered, but we also know that The absolute vast majority, almost all patients are very, very confident and very well trained to actually do these self injections.

Speaker 1

And they're perfectly happy with that because they can rely on the product. And you can see it on the slide here that has an incredible good track record with regards efficacy and that they can basically count on to stop that breakthrough attack As it becomes clear that they're getting a breakthrough attack. That's why we think that Rugnes has this very special we know that Rugnes has this very special place in the market and will continue to have this very special place in the markets going forward. And then I would like to switch over to APDS, and I'll switch over to now ask for Anurag presented the Dejio Andres story. Anurag, over to you.

Speaker 2

Thanks, Simon.

Speaker 3

And on the next slide,

Speaker 2

we can see a little bit of information about APDS, Which is a rare, serious and progressive primary immune deficiency and as you heard from Simon just first described about 10 years ago. We think that if APDS affects about 1.5000000 1.5 patients per 1,000,000 based on literature estimates and our own patient finding efforts. And I'll talk a little bit about those patient finding efforts in a moment. But with those efforts, we've identified now more than 640 patients in key global markets already. We think that that represents a portion of the total of 1500 patients or so that are out there with APDS.

Speaker 2

The signs and symptoms of APDS vary widely. As with many rare diseases, there's also a significant delay to diagnosis Because of these varying symptoms and the rarity of the disease, these delays result in significant morbidity and mortality for these patients And it's something that we're really trying to address with our own patient finding efforts and making Jolanja available of course. The treatments for APDS up till now have been focused on symptom management. So really when we think about the problems that these patients face, so with infections, so giving them antibiotics or giving them immunoglobulin replacement therapy, really not addressing the root cause, however. Genetic test however is a very simple way to make the diagnosis of APDS and we'll talk a little bit about some of the things that we're doing on this front too in a moment.

Speaker 2

On the next slide, we can see that Joenja was now approved by FDA earlier this year. Joenja modulates this hyperactive pathway and as a result allows the immune system now to develop properly. This development of the immune system When it occurs improperly in these APDS patients leads to all of the problems that we see in these patients with autoimmune phenomena, As well as all of the infection related problems that these patients face. With Joenja, we can try to balance this pathway and to support this immune function in a proper manner. Next slide.

Speaker 2

And here's a highlight of some of the data on Joenja. You can see in the top left the indication statement that Joenja is approved in the U. S. To treat patients who are 12 years and older with APDS. And this was on the basis of a randomized placebo controlled study, which showed that Joanna met both co primary endpoints as well as meeting the several secondary endpoints and respiratory parameters as well.

Speaker 2

From a safety standpoint, there were no drug related serious adverse events or withdrawals due to Gilenya in the study. But more importantly, what we saw was that when patients took Joanna on a long term basis in the open label study, we saw many other downstream clinical benefits, including reductions and discontinuation in the use of IVIg or immunoglobulin replacement therapy as well as reduction in infection rates. And these results were consistent with what we saw in the randomized double blind placebo controlled part of the study. And we continue to collect further data And we'll report this data on lymphadenopathy as well as some of the biomarkers that were collected in the study, including markers of abnormal B cell development as well as the production of some of the antibodies that we see are elevated in untreated APDS patients. And as you've seen from our data so far, we're off to a good start with the Patients being able to start to launch a very quickly in the U.

Speaker 2

S. And Steve will report on this progress further shortly. Next slide please. And what are we doing now to look for APDS patients? So when we think about APDS, it's it's really part of a larger pool of patients with what's called inborn errors of immunity or sometimes also called primary immune deficiency.

Speaker 2

And through those efforts, we've, as I said, identified more than 6 40 patients worldwide with APDS, Among which there are 200 identified in the U. S. Those efforts you can see in the box on the left are really focused around education, Testing patients, family testing, multiple medical affairs activities including raising awareness about the disease, raising awareness about the condition itself and really working with patients and clinicians to help identify patients. We also know as with most rare diseases, a large pool of undiagnosed APDSS patients. So we're out there with our medical and commercial teams trying to identify these patients.

Speaker 2

We know that many of these patients are seen by immunologists, but we also know that they're also being seen By other providers where they don't even have a diagnosis yet of a primary immune deficiency or an inborn error of immunity. And we're making available in the U. S. A comprehensive genetic testing program to help get those undiagnosed patients the proper diagnosis. And as a result of this genetic testing and the wider spread use of genetic testing, we're also encountering many patients in the 3rd box here on the right.

Speaker 2

And these are patients who have a result that is inconclusive and this is called a VUS or a variant of uncertain significance. And what that means is that They have a variant in one of the two genes that leads to APDS, but that variant has not been described previously. There's not much information in the literature about it or there is no information in the literature about it. So we're doing a number of efforts now to help those patients, There are a significant portion of that have this inconclusive result that have a primary immune deficiency that have already had a genetic test, But have a result that doesn't give them a final diagnosis. And we're doing a number of things that you see there, including looking through the literature, Going to other clinical laboratories and seeing what testing may have been done, we're also embarking on functional testing, both in the U.

Speaker 2

S. As well as worldwide. And then importantly, family testing to try to see if we can figure out if this variant is present in other family members and if those family members also have the condition or not. And as we move forward, I expect that we're going to find a significant number of patients in this population of patients Who currently are sort of in limbo with this variant of uncertain significance diagnosis currently. Next slide.

Speaker 2

And then thinking ahead beyond the FDA, as you heard from Simon, We are continuing to work with the European regulators and we expect an opinion from the CHMP later this year with a potential approval subject to a positive opinion 2 months later, we and then if we think about the U. K, if the CHMP issues its positive opinion, we can file in the U. K. Very soon thereafter with a potential approval also 2 months later. The Japanese clinical study is open for enrollment and I Back to the first patient to be enrolled and treated there in this quarter.

Speaker 2

And as you heard from Simon, we've also made progress with filings In Canada, Australia and Israel, we've launched our named patient program to make Joenzi available in certain markets And we announced earlier this year that the pediatric patients are able to enroll in the 1st pediatric study, which is in the age group of 4 to 11 years old. And as Simon mentioned, we're also making significant progress in looking at other indications for lenulosin. And I expect to be able to talk about this in greater detail later this year toward the end of Q3, beginning of Q4 as we go through some of the regulatory discussions about our clinical development plans. Also later this year, we're going to be starting Our second pediatric study and so this is in the youngest age group. These are again patients who are age 1 to 6 years old And I expect to be able to start that study in the Q3 of this year.

Speaker 2

Next slide. And just a quick update on where we are with EMA and the progress that we've made so far. So as you know, we submitted our responses to the CHMP Day 120 list of questions. And just a couple of weeks ago, we received further questions, what's called list of outstanding issues That's part of the day 180 procedure. We also understand now from CHMP and we're quite pleased with this is that The CHW will consult an ad hoc expert group and they've acknowledged that the condition is rare and there's an unmet need here And they'd like to seek further guidance from an ad hoc expert group of clinicians to talk a little bit about APDS and the line of sight trial results to put them in Thanks.

Speaker 2

This will be a closed meeting that will involve pharma representatives where we'll have some millennials to investigators as well as APES patients. And as I said earlier, we're expecting the CHMP opinion in the Q4 of this year and subject to Next slide. It's over to Steve.

Speaker 3

Thank you, Eric. Good morning and good afternoon everybody. I'm going to provide you with a short update on Rukenus progress So in Q2 in the first half and also did you end your launch in Q2 of this year. Next slide please. So as Simon touched upon already, the disruption that we saw in the Q1 were market wide and they also affected our competitors.

Speaker 3

And as indicated in the Q1 call, they were transitory. In the Q2, RICUN has performed well. The leading revenue indicators, including growth in unique prescribers, new patient enrollments And vials shipped to patients were all strong. In fact, new enrollments have hit 70 plus for both Q1 and Q2 despite the Q1 market issues. And this really clearly reflects the underlying demand in the market and for RUKENESS specifically.

Speaker 3

In the Q2, RUKENESS revenues globally increased by 20 compared to the Q1. Furthermore, we saw a 2% increase in revenues when comparing the 2nd quarters of 2023 And 2022. So given the strong bounce back in the second quarter, which we did signal in the Q1 call, we're maintaining our outlook to low single digit revenue growth for the rest of 2023. Next slide please. This slide shows the number of unique prescribers continues to grow in the U.

Speaker 3

S. 687 represents 62% of the Groton community and is still growing 9 years post launch as Simon mentioned earlier. And that's despite the significant changes to the market throughout those years. This clearly shows that despite the prophylactic launches and the genericization of Piraziv, there remains both the place And need in the treatment of Alimentarum for a C1S2ase inhibitor and an enduring need for rucanus. We expect to continue growing the unique prescriber base in the coming months years and with it of course the base of patients that benefits for rucanast.

Speaker 3

Next slide please. So let's turn now to the Joangel launch. As you would expect, pharma has bought its A game and all its rare disease commercialization experience to bear in the U. S, which As with all companies, it's a must win market. We have 54 salespeople and leaders comprised of the existing Bruker Nest team where approximately 30% of APDS patients reside.

Speaker 3

And then the Jo Inger institutional team, which joined us in August of last year, They focus on the centers of excellence to which the other 70% of APDS patients either already are being treated or will be referred to. These two teams along with other colleagues that Simon already outlined in an earlier slide, are tasked with both identifying patients and ensuring HCPs have all the information and education they need to confidently prescribe Joenja. We also have amongst others clinical educators to drive family mapping and test. I think Alaric outlined how important that is. And just to reiterate, APDS is not the same or dominant condition.

Speaker 3

So there's a fifty-fifty chance the siblings and other family members are also going to have APDS. And so family testing is critical for both Damon, their welfare and the long term growth of Johengia, which as we know is a progressive disease. Okay. Next slide, please. Just briefly Before I pass over to Yaron, a couple of things around the value proposition.

Speaker 3

As Zalarag already shared, and we should just remind ourselves, this is the only indicated Option for APDS patients is a precision medication that's important. When a physician test positive and a physician prescribes, both the physician and the payer know they're prescribing a treatment that will actually work on modifying the root cause of the disease. Hence at Pharmacy, we knew therefore that we were launching a product with physicians and their underserved patients would need access to and access to quickly. So let's briefly look at The impact on a patient. Next slide please.

Speaker 3

So this is John. He's a 20 something year old man He's been dealing with APDS since he was 11 years old. Like most patients, he's been in and out of hospital, was having difficulty developing social skills, keeping friends And we're basically willing to try anything to remove the stress and the burden of taking 11 pills in the morning and 9 pills at night to try and manage his symptoms. Joenger, of course, has removed that heavy pill burn and is treating the underlying cause of John's APDS. Since being prescribed Joenger, John's life has already changed significantly for the better.

Speaker 3

His lymph nodes have decreased in size. And I think you can see in the thought bubbles, his quote around that, his percentage are actually gone as opposed to just being reduced in size. And importantly, he's starting to think about his future and what he wants to do in life. And as you see here, that includes actually going to college, something he couldn't have envisaged That's basically the promise that Joenja can and will continue to deliver for our patients and it's what will continue to fuel This launch phase and also our future growth. Next slide please.

Speaker 3

So As we discussed in the last call, the preparation for this launch was very, very rigorous and thorough. And as expected, we're off to a very good start. Our first fully reimbursed commercial shipments of Joinger occurred within 2 weeks of FDA approval, which is very quick and Certainly unusual. And to date, we've enrolled 60 APDS patients and shipped to 43 of them on payer approved products, with the remaining 17 To the end of the first half in process. With respect to market access, the teams are doing outstanding work and we continue to make good progress Partnering with the national and regional payers, including state Medicaid programs to prepare for clinical review and coverage policy development, many of which are now rolling out.

Speaker 3

And to date, no patient has been denied access to JoAnja. So as you can imagine, we're very pleased with the Q1 of post launch progress And expect to build on our early success in the coming months to both get eligible patients on therapy and grow the patient funnel for future patients. And that will continue the brand growth and of course lay the foundations for Farming's continued success into 2024. So So now I'd like to hand over to Jeroen, who will cover the financials.

Speaker 4

Yes. Thank you very much, Steve. Good morning, good afternoon, everybody. Moving to the next slides on the financial highlights for the Q2 of 2022. Our revenues grew to €54,900,000 which is a growth of 9% versus last year.

Speaker 4

And this €54,900,000 consists of 3 point €8,000,000 of Joenja sales for the Q1 that we obviously report that Joenja sales, a €51,100,000 from Rugenas. And that is a growth of 2% from last year's Q2, 20% even from Q1. So a very strong recovery, as Simon and Steve mentioned before as well from Q1. Gross profit grew to €49,200,000 which is plus 7%, which is roughly in line with the growth in revenues. Operating costs increased by CHF 23,400,000 and that's on a number of items, Mostly marketing and sales, but I'll give you some more detail later.

Speaker 4

Also important to understand for the Q2 is that we had and in what we call other income, so that's between gross profit and operating costs in the P and L. We had an income of $21,100,000 For the sale of the priority review voucher to Novartis, which was as per the agreed contract in 2019. And also good maybe to tell you that in the last year's Q2, we also had some big other income that was connected to the Bayer Connections transaction, as you may remember, which brought us in €12,800,000 To cut a long story short, the increase in other income from this year to last year was €8,000,000 Then on the operating profit, euros 5,300,000, that's a decline and that's roughly in line with the increase in the operating costs. And the net profits went from €15,700,000 to €1,300,000 so a decline of €14,400,000 And that is, by the way, an improvement from Q1 where we had a loss in a net loss in the quarter. And if we look at the first half results on the next slide, total revenue grew by 1% to 97 point 4.

Speaker 4

The gross profit, €87,600,000 with a gross margin of 90% was a good gross profit income. Operating costs, CHF 118,500,000 from CHF 82,200,000 last year. So that's an increase of CHF 36 €200,000 more detail about that later. The operating profit was €8,400,000 negatives on operating loss, so to say, And the net loss was has been €10,900,000 so far in the year. If we then look at the revenue breakdown for the last few quarters on the next slide, You see indeed the enormous growth from Q1 and well, the drop obviously was from the reimbursement issues that were apparent in the whole HAE market, but we shut a strong recovery of 20% in the second quarter to €51,100,000 and you see the €3,800,000 sales from Joenzi for the first time in our history.

Speaker 4

Moving to the next slide on the costs, because they went up significantly, just a bit of color on where we spend the money. So in the Q2, we have the €10,000,000 of milestone payment to be paid To Novartis related to Joenzi. And we had a basically over time, you see a Substantial increase in marketing and sales to support the launch of Joenja. So Steve just mentioned that the launch preparation was rigorous and thorough and as well as also what you see in the money that we spend on geoengine. You see that a fairly stable development of G and A cost and an increase in R and D in the Q2 of this year of 5,300,000, which is largely related to the work we're doing on the approvals in several international markets like Europe, Canada, Australia and Israel and a buildup of our medical departments, both in U.

Speaker 4

S. And in Europe. So apart from the cost categories, another way of looking at the increase In cost in the first half of this year, that was plus €36,000,000 is that so we spent €10,500,000 in total on the milestones. We spent €7,000,000 of the €36,000,000 increase on LENIODAZEF out of pocket expenses and think about R and D costs, but also marketing, market access and amortization costs of the license. And we added €60,000,000 of The payroll and general expenses, most of it is in payroll, because we increased the number of people since last year from 319 at the end of the second half last at the end of the first half last year to 383 now.

Speaker 4

So an increase of 64 FTEs and that is across the board, most of them in marketing and sales, but across the board with growing the company as an investment in future growth. A short overview on the next slide of the cash flow over the last quarter. It increased by the cash And cash equivalents increased by SEK 8,000,000 to SEK 192,400,000 and that was mostly related to The sale of the Priority Review Voucher, which is considered a cash flow from investing activities. So that was the overview of the financial highlights. Then going to the outlook for this year on the next slide.

Speaker 4

Driven by the strong recovery, we continue to guide on low single digit growth for recognized revenues for the full year. Joenja was approved at the end of March by the FDA, and we've been commercializing the product since April this year. We expect a CHMP opinion for the EU in the Q4 of this year and the marketing authorization subject to that positive outcome 2 months later for Europe. In the UK, we will be filing leniadisib with the MHRA following the European Commission Decision Reliance procedure. And again, that is subject to the positive outcome of the EMA review.

Speaker 4

We continue to invest in operating costs to accelerate the growth And further details on the plans to develop Additional indications for LENIELOSYP will be provided later this year. And last but not least, we continue to look at investments With that, I want to move to the next slide and get back to the operator to operate the Q and A, and please feel free to ask any question. Thank you.

Operator

Thank you. And our first question comes from Christian Glenny from Stifel. Christian, your line is open. Please go ahead.

Speaker 5

Yes. Good afternoon, guys. I guess we'll start with Joenja and just initially on just to explain and sort of clarify The 60 patients on enrollment versus the 43 on paid therapy, is it the case that some of those are They're on therapy. The other 17 are on therapy already, but still working through in terms of reimbursement? Or how should we think about those

Speaker 1

Hey, Christian. When patients actually are enrolled, They get a free starter pack, which is a 1 month supply of, of LUENJA. So yes, Once they are enrolled, they have access to the medication, so they have it. And then of course, the administrative The procedures to get them reimbursed at the start. And if that happens within the month, the second month Will be a commercial pack.

Speaker 1

And if that procedure is not yet finished, then there will be Another month's supply of info in the form of a bridging pack provided. So to answer your question, yes, they're all having access to therapy once they are enrolled.

Speaker 5

And then in terms of the patient identified patient numbers, You've added 140 in terms of 640 versus 500 previously reported. I noticed that the U. S. Has stayed the same. Just where does those extra 140 patients come from?

Speaker 5

Is that mostly the new international markets, Canada, Australia, Japan

Speaker 1

Could you comment on that Anurag? Is that possible?

Speaker 2

Sure. Hi, Christian. So we are finding patients we're continuing to find patients in the U. S, We're also finding patients in other markets. So that does include Japan, Australia and other markets where we intend to commercialize first.

Speaker 5

Okay. Thank you. And then in terms of the you've got 200 patients in the U. S. Identified.

Speaker 5

If Your €1,500,000 in the U. S, there's €500,000,000 or so out there. That's already a 40 Percent diagnosis rate, where do you think that could that diagnosis rate could go over the next

Speaker 1

Would you like to ask that as well, Anurag?

Speaker 2

Sure. So we definitely see this trending up. I think When we think about markets in Europe and some other countries across the world where there are more centralized healthcare systems, We can see that the prevalence there already exceeds $1,000,000 in the U. S. Of course, we have more distributed and Less centralized system.

Speaker 2

So the diagnosis of these patients is and the care for these patients is a little bit more fragmented. And so we expect that over time that diagnosis rate will go up as there's increasing awareness of the disease, But there is also an available therapy available for these patients. So I think that we expect that to increase and At the minimum approach, what we see in some other countries, but likely increase beyond that. And that's why we think we're Being reasonable about an estimate of $1,500,000 per million. As I mentioned earlier, there's a significant population of patients that we found Beyond the 200 in the U.

Speaker 2

S, for example, that have this that have these variants of uncertain significance or What are termed the VUSs. And I think that that will that's another pool of patients who have symptoms of a primary immune deficiency, have had a genetic test, Oftentimes that's well before we've been involved in the picture even, but they still don't have a clear diagnosis. And I think this will likely lead To an increase in the number of diagnosed patients over time.

Speaker 5

Sorry, as a quick follow-up to that then, is it Your understanding that those patients would also be appropriate for Joendra even if they haven't gotten sort of formally fined

Speaker 2

Yes. That's correct. They would be appropriate for Joenja because they would have APDS If that's confirmed, right now they have a oftentimes they have symptoms and clinical manifestations Of APDS, and they have variants or mutations in 1 of the 2 genes that leads to APDS, the question is, are those mutations, are those variants disease causing? And that's part of the effort that we're helping clinicians Gather information, get access to further testing and help them resolve that question for those patients because they're also, like I said They're early, they're sitting in limbo and don't have a clear diagnosis yet. If they get a clear diagnosis of APDS, then those patients would be Potentially eligible for the treatment with Joangel.

Speaker 5

One final one if I can, I'll get back in the queue. Is it possible to give any rough sort of guidance for Duenzi for the year, whether that's in terms of patient numbers or revenues? Obviously, you've got 60 patients in the program already $3,800,000 revenue in the second quarter. The 60 patients is already a 40% penetration of The 150 patients in the U. S.

Speaker 5

That are over 12, so any sense for penetration rates or

Speaker 1

Yes. I understand that, Christian. But it's early days, right? Yes, we're off to a good start, and we would like to see a bit more development of the numbers. Obviously, the numbers are ticking up all the time, right?

Speaker 1

And we'll keep you updated on a quarterly basis for now until such time that we get a little bit more feel for the market And the development going forward, and then we will start giving some different guidance. So for now, no, not yet. I have to be a little bit more patient for that. But we will eventually, of course, do that. But we'll keep you on a quarterly basis updated on the number of patients that are on therapy and are enrolled.

Speaker 1

Okay?

Operator

The next question comes from Joe Pantginis from H. C. Wainwright. Joe, your line is open. Please go ahead.

Speaker 6

Hey, guys. Thanks for all the details. I appreciate it. So a couple of questions on the initial dynamics of the Joensha launch. So I guess and of course, prefacing it by this is all early still.

Speaker 6

What is the general time it takes or approximate time it takes To get drugs to patients once they're identified. And then second, when you look at these numbers, the 60 The end of 43, how many patients have gotten the drug or on the drug? Of the revenue number that you posted today, which like you said beat overall expectation, how much of that, if any, is based on just getting drug into the inventory channel? Thanks.

Speaker 1

That's an excellent question probably for Steve to go into a little bit more detail. Steve, could you pick that one?

Speaker 3

Yes, sure. Thanks, Simon. Good morning, Joe. So the first thing is, as Simon alluded to, once the patient is enrolled, we get the starter pack out. And so they're on therapy pretty quickly.

Speaker 3

And then in terms of the time it takes to get drug sorry, to get approval for patients, It's typically we've seen so far between 4 6 weeks, which is pretty quick. We've seen 1 or 2 is a little quicker, 1 or 2 is a little more, but think of it in a 4 to 6 week period. So we're not having to bridge too many patients beyond the starter pack. And then in terms of stocking, it's almost 0. I mean, we put some in right at the very start.

Speaker 3

We were able to process patients very quickly after launch, which means we've burned through that very quickly. And the way in which our partner works Panther Rx means we're basically practicing just in time delivery. So for the most part, that revenue is generated by demand, by patient demand.

Speaker 6

Great to hear. Thanks. And then oh, yes, absolutely. So I feel I have to ask my next obligatory question that I usually go When you look at OTL-one hundred and five, so obviously, you're holding it close to your chest. But I'm curious, With the ongoing preclinical models that you're doing, will we be able to see or when will we be able to see any of the data coming out of these models?

Speaker 1

Would you like to comment on that Anurag?

Speaker 2

Sure. Good morning, Joe. So we're making progress together with Orchard in those models. I don't have a firm timeline that I can say that it's going to this is going to progress in the next couple of weeks or not. But I can say that I expect probably towards later this year, we should be able to provide some further updates on where we are With that program, the data that those preclinical models are generating and how we plan to move the program forward.

Operator

The next question is from Sushila Hernandez from VLK. Sushila, your line is open. Please go ahead.

Speaker 7

Yes. Thank you for taking my questions.

Speaker 8

Also on Joanna, so you mentioned it takes 4 to 6 weeks To get approval, but could you just elaborate a bit more on the time frame identification to enrollment? Could you share a bit more on that? Thank you.

Speaker 1

Steve, would you like to comment on that?

Speaker 3

Yes. Thanks, John. So, it's obviously variable, but relatively quick once a Physician and the patient have that discussion. It's literally governed by the time it takes to complete the form, send it into the hub and then generate the starter pack. So, I mean, it's a day to a week, but really dependent on the speed at which that process happens.

Speaker 3

But it can be very, very quick, easily a day or 2.

Speaker 8

Okay. That's clear. Thank you. And just on the RECONEST, You mentioned that over 70 quarterly new patients. Where are these patients coming from?

Speaker 8

Are these treatment naive patients or did they switch?

Speaker 3

For the most part, these will be switch patients. At this point in the evolution of the market, There are very few treatment naive patients. So these are patients who are either not satisfied on their current acute therapy or require as for the guidelines of 2nd acute therapy to make sure that they have what they need to do with any breakthrough attacks.

Speaker 8

That's clear. Thank you. And then the final question, could you share progress on your BD activities? How is it progressing?

Speaker 1

That's a good one, Cecilia. We have a very active team. I think if I sort of recognize remember some numbers, they turned over 150 Opportunities over the last 12 months. There were the vast majority, of course, was quickly disposed of. But then we have an internal community, including Anurag and Stephen, We'll look then and take a next look.

Speaker 1

And then as of that, a smaller a much smaller number, of course, comes to the 4, and we do some more analysis on that. And We've done a few due diligence over this last year. It does last year as well. So we're making I think I can say the efficiency of the BD process has significantly improved over the last year. And We came a couple of times very close, but we're very careful, of course, with the first one.

Speaker 1

And as you know, in business development, It's all or nothing, right? There's no deal or there's a deal. So but yes, we remain optimistic About being able to acquire or in license preferably in license, of course, that's the preferred mode of action And another asset that is in mid- to late stage development so that we can actually plan for another asset To be launched in the not too distant future. And that's all I can say at the moment, I'm afraid.

Speaker 8

Okay. Thank you. Looking forward to updates on that front as well.

Speaker 1

Thank you.

Operator

And the next question comes from Natalia Webster from RBC. Natalia, your line is open. Please go ahead.

Speaker 9

Hi there. Thank you for taking my questions. I just have 2, 1 on Rootken Est and 1 on linear license. So on Rootken Est, We saw an improvement in Q2 and you reiterated your guidance for the full year. But I was wondering if there's any risk of sort of the reimbursement issues that we saw in Q1 recurring at all.

Speaker 9

And then also you mentioned sort of strong market demand and continued growth in unique subscribers. But I was wondering if you're able to Provide some color specifically around what you're expecting for H2 and sort of in the near term into 2024. Jackne, you're on LENIO license.

Speaker 7

Do you want to go to

Speaker 9

that first so I can ask the next question?

Speaker 1

Yes, probably Let Stephen answer that question first. Is it okay?

Speaker 3

So firstly, the event that happened in Q1, we're not expecting a repeat of that. It was A very specific market wide event that was unrelated to farming. It affected everybody and it was rectified towards the end of Q1. My suspicion is that's very much a one time event. I don't expect that to occur again in the future.

Speaker 3

And then in terms of growth in prescribers, it's actually been very consistent for the last 7 or 8 years, certainly, I've been with farming and we've retook control of RUKENESS. There's still, as I mentioned, another 38% of specific prescribers in the U. S. Who've not yet tried Rufinest, and we continue to call on them as well as our core customers. So at this point, I see no reason given the last 7 or 8 years to not expect that same steady growth as we move forward.

Speaker 9

Great. Thank you. And then just secondly on Lenulisib, regarding the timing for the You say that you expect this in Q4 this year, but I think you said H2 previously. So just wondering if there's been any delay there or just being a bit more specific?

Speaker 1

Do you want to be answering that, Anurag?

Speaker 2

Yes. I think we're just continuing to work Collaboratively with the CHMP and EMA and answering their questions. And I think we're just giving some more detail on as we have more detail around the timeline there. That's why we're seeing the Q4 now.

Operator

Next question comes from Hartaj Singh from Oppenheimer. I apologize your line is open. Please go ahead.

Speaker 7

Hi, this is Sanyin Chung on for Hartcharge. Congratulations on the good quarter for I have questions on the PDS patients. Can we think about the growth rate of APT application number quarter by quarter? Is seasonality a factor to consider? Thanks.

Speaker 1

So you asked if there's any seasonality in APDS, is that right?

Speaker 8

Yes. I mean, the growth like how

Speaker 7

can we think of the growth range of APDS patient numbers Quarter by quarter and what seasonality be a factor to consider down the road?

Speaker 1

Steve, you'd like to comment on that?

Speaker 3

Thank you, Simon. I mean, obviously, it's an ultra rare disease. We had a bolus of patients to start with, We're still through the launch. We're still in the very much in the launch phase. So I'd expect steady growth as we move forward and really No seasonality, but obviously in the outer years, you start to see the rate of growth slow as you would do with any launch.

Speaker 3

But nothing specific in terms of seasonality Where we would expect significant troughs or spikes. Does that answer the question?

Operator

We have a follow-up from Christian Glenny from Stifel. Christian, your line is open. Please go ahead.

Speaker 5

Hi, thanks. Just I thought it was worth following up on, obviously, you filed applications Canada, Australia, Israel, potential approvals Next year, what's the commercial strategy there? Is that you're going to is it better Find partners, is it worth doing yourself in some of these markets? What should we expect there? And obviously, Anne, you Need to run a trial a bit further down the track.

Speaker 5

What would be the strategy?

Speaker 1

Yes, yes. First of all, Christian, before I hand over to Stephen, There's no trial requirements, right, in Australia and in Canada. We have submitted the files. We have received a priority review as well From those authorities. So it's a matter of waiting for, 1st of all, the validation of the file of the files, regulatory files.

Speaker 1

And then of course, we'll go through that and then we'll await the opinion of those regulatory authorities somewhere as alluded to in And it's somewhat, but this is probably mid the first half of next year. Then I'll hand over to Stephen to explain how we are Approaching the commercialization in those markets.

Speaker 3

Thank you, Simon. Hi, Christian. So certainly, I think to answer what's at the heart of the question, We have no plans to out license in any of those markets and give away or frankly the value of the product to other companies. The structure though in the go to market with just your engine would be different from, say, the U. S.

Speaker 3

Or Europe, where we have a pretty large footprint. So it would be a large sorry, a light cost efficient footprint. And in somewhere, for example, like Australia, you would service that market directly with Probably a hybrid model, a combination of directly employed farming employees and then local distributors and partners, you can help us Service certain elements of the Australian infrastructure. But to keep things really efficient from that market, you would also So Hong Kong, South Korea, which is the 3rd biggest market in Asia Pac and other smaller markets. So the plan is very much for us to keep control of the product, keep control of Bringing patients on therapy and obviously reaping the benefits of that for ourselves and our stakeholders.

Speaker 3

Does that answer the question, Prashant?

Operator

As we have no further questions, I'll hand back to the management team for any concluding remarks.

Speaker 1

Thank you very much. Thank you, ladies and gentlemen, for attending our conference. And I would like to remind you of, film the elements of the outlook for the remainder of the year. You've heard all the positive Developments on the leading indicators, the forward looking indicators underpinning the sales of Ruknest, we remain confident To continue to guide on the low single digit growth for RUKENESS revenues for this year. You've heard about The steady flow of new patients coming in for Joenja and of course, the immediately available Patients that could be brought to Duo Enget Therapy over the coming quarters.

Speaker 1

You've heard about the regulatory interactions that we have with the Europeans, the plans to follow-up in the United Kingdom and the submissions, of course, to broaden our footprint with the submissions in Canada and Australia and, of course, that we are continuing to invest in The launch preparations in those markets and, of course, the clinical trial plans that we have for the second indications of lenulosib, Which, of course, we will update you on as and when we have received the feedback from the regulator on our plans that have been submitted to the regulators with regards to both the what the new indication is. And that indication, by the way, as you already have learned from us, It has a bigger patient potential than APDS, and we will refill that later on during the year. And last but not least, We hope to be able to come back to you June the remainder of the year, of course, with in licensing or acquisition news because we are very keen and have the capabilities, of course, to further leverage our commercialization infrastructure that we have and that we are basically expanding towards the coming years, including markets like Australia, Canada, as you heard from Stephen, but also Japan And to bring more products to that market to further significantly accelerate the growth of our company going forward.

Speaker 1

So thank you again for being at our conference,

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