First Solar Q3 2023 Earnings Call Transcript

Quartr
Provided by Quartr
October 26, 2023

There are 11 speakers on the call.

Operator

Good day and thank you for standing by. Welcome to the Farming Group N. V. Third Quarter 2023 Results Conference Call and Webcast. At this time, all participants are in a listen only mode.

Operator

After the speakers' presentation, there will be a question and answer session. As a reminder, the company will only take questions from dial in participants. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Simon De Vries, CEO of Farming Group. Please go ahead, sir.

Speaker 1

Thank you very much, and good morning or good afternoon, ladies and gentlemen. I'm here with my three colleagues, Stephen Thor, our Chief Commercial Officer and Erik Leland, our Chief Medical Officer and Jiru Lachamond, our Chief Financial Officer. And we are delighted to take you through the Q3 results of this year. Before I do that, however, I would like to point you to the forward looking statement slides because we may contain this presentation may contain or will probably contain Forward looking statements that, as you know, are statements of future expectations that are based on our current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in these statements. And the rest I leave to you to read.

Speaker 1

So let's just move on to the next slide and then, of course, to the next slide about building a sustainable business in rare diseases. And that's what it we are about. And this is, of course, a very Interesting moment in time, quarter results of 2023. And you see that on the left hand side, How we are going to build start to build that rare disease, the Steminal Rare Disease business. We have significant positive cash For more than DKK200 1,000,000 of moving annual total sales of RUKENEST that confront The Joenja launches and pipeline development to start with.

Speaker 1

And we are very pleased, of course, with the results and the strong revenue growth. RUKENAS, 18% up on the 2nd quarter and 11% up on last year's Q3. And also if you look back 9 months, So year to date, 2% up on last year. That means that we are on track to deliver Our low single digit revenue growth for RUKENESS for 2023. And then of course, you move to the middle pillar And you see there, of course, the global approvals and commercialization of GEOANJA that can be funded from those cash flows from Rucanex.

Speaker 1

And of course, we were Very pleased to get that very fast approval from the FDA back in March, brought the product to the market and got reimbursable reimbursed patients Almost immediately when we're on the market. Such that we can already record revenues in the Q2 of this year, which The Q1 Joenja was on the market. And now of course we're very proud of the continued growth of Joenja where in this quarter we booked CHF6.5 million of revenues and year to date CHF10.3 million. In addition to that, of course, regulatory reviews are ongoing for Joenja in Europe, Canada, Australia, Israel. And we have a pediatric clinical trial program ongoing as our label is currently 12 years and upwards.

Speaker 1

And of course, on the right hand side, you see further growth accelerators beyond Joenja in APDS, 1st and foremost. And that is where we will come back to you before year end to update you on that. We are in dialogue with the FDA about the 2nd Lelion indication and we'll provide you with more details towards the end of the year. And colleague Anurag, Ronan will talk about a lot more. And last but not least, as we have a very strong commercialization Infrastructure in both the U.

Speaker 1

S. And Europe. We're hunting as we speak for new in licensing opportunities or acquisitions for additional products in rare diseases that we can actually continue to develop, clinical development and bring to the market and successfully commercialize. So we're looking for products that have clinical proof of concept. And if you see at the next slide, you see that there is actually space to have such products in our pipeline.

Speaker 1

You see here the extensive work we do by enlarging our footprint by means of bringing lenulisib to markets A field like as far a field as Japan, but also Canada, Australia and Israel and of course the other indications. So you see there is space in this pipeline to further accelerates the growth of the company going forward. And with that said, I would like to now hand over to my colleague, Stephen Thor, who will give you some more insights in the commercialization operations on Ruukonext and Joanna. Stephen, over to you.

Speaker 2

Thank you, Simon. Good morning, everybody. As Simon said, I'll give you a brief overview of the Rukanes performance and also some insights around the JoEngine launch And update you on that progress today. Next slide please. So as communicated at the end of Q1, and as you're all aware, the market We're undergoing a significant event which affected all products.

Speaker 2

The event was short lived and as we said we would, we bounced back strongly in Q2 and Q3. As you can see in the first bullet, we posted strong growth in Q3. And even with the softness for Q1, as Simon showed, we have grown versus prior year. So we're really pleased with that performance. And this has been driven by strong performances across all of our leading metrics, But I especially want to flag new patient enrollments, which have exceeded 70 over each of the past three quarters, which is stronger than we've seen in the past.

Speaker 2

And so hence, as Simon said, we continue to guide the low single digit growth for Rutonesque this year. Next slide, please. So as many of you know, RUKEKINES was launched in 2015. We've actually though been active in the community since around 2000. And over those 23 years, we collaborated with All key stakeholders including the clinical ones and patient advocacy groups such as the HAEA and that has been the major driver for the consistent success of Roux enest Over the 9 years post launch and it's why the prescriber base continues to grow with 700 in the U.

Speaker 2

S. To date and Also why we've treated over 2,000 patients and that metric continues to grow as well. So I think what that clearly underlines is the importance and the ongoing need for recombinant IV C1 esterase inhibitor and that's despite the fact over 70% of patients are now on prophylactic treatment today. So next slide, please. So moving to Joengia, as Simon showed earlier in the presentation, we're off to a very strong start in the U.

Speaker 2

S. With our launch. And as indicated in previous calls, and I think as everybody on this call knows in relation to the environment more broadly, Access once a patient is diagnosed is one of our key pillars for success and the ongoing success of this launch. And we partnered with an organization called Panther Rx that specializes in ultra rare or rare diseases to build a program that we thought would enable us to quickly provide patients with access to Joengia once they're covered for chronic use. And I'm pleased to report that in only 6 months post launch, Pharman's access and medical teams in partnership with key opinion leaders across the country A secured APDS coverage policy is in over 90% of our target plans across that's across commercial and government payers.

Speaker 2

And the result is a 93% approval rate with 0 denials. And so I just want to repeat those 2, 93% approval rate with 0 denials Despite the rarity of this condition and the heavy lift on education, so that's really a very strong performance. Also, as you know, From enrollment to shipping to patients, the gold standard in rare disease is 30 days. And I'm pleased to report to you that we're averaging 26 days typically and sometimes you're getting from enrollment to putting product in patient's hand in less than 20 days. And really this is based on exceptional customer focused and execution, which Further instills, I believe, confidence in our stakeholders, but most importantly, the patients and treating physicians.

Speaker 2

So next slide, please. So I've mentioned farming strong customer and patient focus and I think combined with the exceptional I've already mentioned, our U. S. Team have delivered strong results in that 1st 6 months since launch. We have as the slide shows 76 eligible patients, 63 shipping and that represents well over half of the eligible patients we have on therapy.

Speaker 2

This has led to the revenues of $10,300,000 that Simon also showed. And as already mentioned, payer discussions have and continue to go very well, which Creates importantly an excellent environment for us to pull those patients through once they're diagnosed and enrolled. So finally, I just want to flag now that we're through the immediate launch phase. And with many of the previously identified patients on therapy, We'll be placing additional time and resources now on family testing. Most of the patients we have are patient 0, so to speak.

Speaker 2

So we believe there's a lot of opportunity there to And with that, I'd like to hand over now to our CMO, Doctor. Anar Abraham.

Speaker 3

Thanks, Steve. I'll begin on the next slide with a little background information about APDS. So APDS was first described in 2013 And based on our estimates and literature review, we believe that there are more than 1500 patients worldwide diagnosed with APDS We have already found more than 640 of those patients. These patients who have APDS have really had limited treatment options until recently to only treat the symptoms of the disease. The disease manifests itself in childhood and worsens over time without anything specifically indicated for treatment, Physicians and patients were quite limited in their treatment options.

Speaker 3

And as with most rare diseases, the signs and symptoms vary across patients. This makes the challenge of diagnosis even more difficult beyond just a rare disease. Fortunately, there is a genetic test that can provide a definitive diagnosis for APDS and I'll be spending more time in the coming slides talking about our plans and efforts to help find more patients with APDS. On the next slide, we can see what Joenja now brings to patients in the U. S.

Speaker 3

As a potential treatment option for them for their condition. It is approved by FDA for the treatment of APDS in adults And pediatric patients from ages 12 years of old and older. We have randomized clinical trial data showing that Joenja met both primary endpoints as well as meeting several significant other clinically relevant endpoints. In addition, We've seen a well tolerated and generally safe adverse event profile. There were no drug related serious adverse events in the study or withdrawals due to the drug in the study.

Speaker 3

And more importantly, we have long term data and I'll be sharing some of that with you that we've Publishing and presenting at conferences recently about the long term benefits of using Joenja over several years in many cases. And this includes for patients in some cases to discontinue the use of immunoglobulin replacement therapy, reduction in infection rates And persistence of the benefits that we see from the randomized clinical trial. And we can see that both in key measures of what's So their lymph nodes continue to stay not enlarged and we see benefits also in their immune cell function. And as Steve mentioned, this has led to a strong start for Joenja. I think this speaks both to the unmet need That exists in this APDS population, but also speaks to the seriousness of the condition.

Speaker 3

On the next slide, you can see some of our things that we're doing beyond the work that we've done in the U. S. As we discussed in August, We received the day 180 list of outstanding issues from the European Medicines Agency and we can confirm now that in October we have submitted our responses. We remain on track to expect an opinion in this quarter and with potential approval 2 months later. If we receive a positive opinion from The CHMP in this quarter, we can then go ahead and file with the UK MHRA Agency with potential approval also 2 months later.

Speaker 3

And as we previously announced, we've started a program in Japan to enable registration there eventually. We've also now filed in Australia, Canada and Israel and those applications are proceeding along their review plans. We've also started a named patient program to eventually be able to help patients obtain access in territories across the world. And our pediatric study is enrolling quite well with the majority of enrollment already complete in the 4 to 11 study and the 1 to 6 year old study Has now started recruiting, so we expect that first patient also to be treated very soon. And as Simon mentioned, We have been engaged with FDA about the second indication and we expect to be able to provide further details on the second indication later this quarter.

Speaker 3

On the next slide, I want to review with you some of the patient finding efforts that we've initiated and are ongoing at this time. The first of course is APDS is a rare disease and it's critical to raise awareness about APDS. And now we have A plethora of data also on lineolysis that we can share. These data highlight the seriousness of APDS And I think it also highlights, I think the experience that we have with Lineos have been treating many of these patients. On top of that, we have our Ongoing Navigate APDS program that offers no cost testing available to patients in the U.

Speaker 3

S. And Canada. And these patients, once they have this testing available, often have questions. So we have genetic counselors available to help them Consider the testing and then also review the results with them. And then a big effort that we're really pushing on right now is that when we look at the diagnosed patients That we have in the U.

Speaker 3

S. Especially, we find that most of those patients actually don't have family members that have even been tested. And we know that APDS is an inherited disease, but there's this gap in terms of testing. So we've initiated several efforts here both with physicians and with We will now begin the Q3 results conference call and webcast.

Speaker 1

We will now begin the Q3 results to be able to reduce the barriers

Speaker 3

to allow further testing amongst family members, which we think will be important On the next slide, I want to review with you a little bit of information on something called variance of uncertain And what these are, are genetic test results that are basically unclear or I would better say unclassified at this point. And with the growth in genetic testing, we get more of these inconclusive results. These are basically variants that have not been previously seen. And this is really frustrating for patients and doctors because they have patients who have clinical symptoms of APDS often, But the genetic test result is inconclusive. And so we have several efforts ongoing and I'm not going to review all of them with you in detail here.

Speaker 3

But these efforts Involve trying to review the existing data and try to collate all of that information and publish that. We've just started a partnership, for example, with Genomenon to develop these genomic landscapes, which will be available to all clinicians to be able to easily access Varian's information. We have a number of efforts ongoing to increase the availability of functional testing. And then lastly, I think I'm really excited about this possibility of this new effort that we started Looking at a way to in a single experiment test all possible variants and quickly determine whether a result is Pathogenic or disease causing or not. And I think the nice thing here in a sense is that this is a problem.

Speaker 3

It's a new problem for APPS, But we're really using a playbook that exists already for many other genetic diseases and we're following that playbook To be able to help these APDS patients who may still have this unclear diagnosis. On the next slide, you can see some of the conferences we've been presenting at and some of these abstracts we presented. These abstracts vary both in terms of what we're talking about in terms of the seriousness of the condition. So the mortality, for example, associated with APDS, But also the healthcare costs associated with APDS and especially untreated APDS and these data I think highlight very nicely The serious burden that these APDS patients face. On top of that, we continue to get more data out of our clinical trial program.

Speaker 3

So we have a second interim analysis that will be published at the IPIC conference next month. And we also have a number of case series as well as abstracts On single patients, these are from many times from our expanded access program or compassionate use program where, for example, the second bullet under the IPIC heading is a patient who was previously transplanted unsuccessfully unfortunately, But then was treated with lenulosib under this compassionate use program and the data at the abstract will show the benefits that this patient was able to experience. On top of that on the next slide, you can see some of the publications. So the first publication is the 1st interim analysis and that's available now In the whole paper. And then the next publication is also a key publication describing the mechanism of action of lenulosib in APDS.

Speaker 3

And I think it describes clearly how APDS leads to this primary immune And with that, I'll turn it over to my colleague, Arun Wacherman, our Chief Financial Officer.

Speaker 4

Thank you very much Anurag. Focusing first on the financial highlights of the Q3 2023. Total revenues increased to CHF66.7 million, so an increase of CHF12.5 million for 23%. Gross profit increased to €58,400,000 an increase of €6,500,000 Operating costs increased from €44,700,000 to €56,800,000 And the increase of DKK12.1 million is mainly because of R and D additional investments of €8,000,000 and marketing and sales of €5,000,000 And that is all directed towards the launch or most of it is directed towards The operating loss was profit in this case is €1,900,000 and the net profit was €3,500,000 in the quarter And that was on the back of positive financing income and a tax credit, which is a timing effect. Simon mentioned it already, but the cash and cash equivalents increased to €199,000,000 at the end of that quarter.

Speaker 4

Looking at the figures year to date, 9 months year to date, The revenue increased by 9% to €164,100,000 Gross profit increased to €146,000,000 And as was guided earlier this year, we further increased Our OpEx, as I said, into mainly marketing and sales and R and D. And the operating costs were €175,300,000 I. E. An increase of €48,400,000 versus the same period last year. Consequentially, the operating loss was €6,500,000 for the 3 quarter period and the net loss was €7,400,000 which is an improvement from Q2 this year.

Speaker 4

If we go to the next slide, We see the growth in revenue over the quarters. The 3rd quarter revenue was €66,700,000 and that's a 23 percent increase from last year, driven by both Rucanesh and obviously Joenzi, as you can see on the picture. And also accelerated growth is seen in Ruckernest. So you will remember that in Q1, There was a temporary reimbursement issue. It was a market circumstance.

Speaker 4

So we had a dip in sales in Rukunasp, We have very well recovered from that and we are now on a positive note and that is in line with the guidance that we gave earlier this year. Looking at the Cost development, as I said, investments in the Joenja launch and further development of Lerniala ship continues. And we have a quarterly OpEx of almost 57 €1,000,000 And you see also that the increase is mainly, as you would expect, both in Research and Development and marketing and sales and the general and admin costs are relatively stable. The increase in R and D or the stable for now was mainly guided towards clinical, operational And Medical Affairs. And if you look at the development over the quarters, as we guided earlier in the year, is fairly stable, especially if you Then going to the outlook for this year, we remain on track For single digit growth in RUKONASS revenues and that's only been confirmed by the numbers that shown in Q3.

Speaker 4

Joenzi was improved in the Q1 and we've been commercializing in the U. S. Since early April as my colleague Steve, you alluded to. The CHMP opinion is expected in the Q4 of This year and the marketing authorization subject to a positive outcome of the review is expected in Europe 2 months later. We will file Lenny Aldersip with UK's MHRA following the ECDRP route, And we will continue to invest in accelerated growth for the future in operating costs.

Speaker 4

In the remainder of the year, we will detail further our plans to develop linealoship And as Simon mentioned, we keep looking for investments in licensing and also With that, I want to start off kickoff the Q and A. So any questions are welcome. Thank you.

Operator

Thank you. We will now go to your first question. And your first question comes from the line of Christian Glenny from Stifel. Please go ahead.

Speaker 5

Yes. Good morning, good afternoon, guys. Thanks for taking the questions. 3, please. I'll take them in order.

Speaker 5

Let's start with Rucanesh and a strong quarterly sales print. Just to be clear around whether there's anything to be aware of In the Q3 numbers, maybe the stocking or some impact that means It should be a clean quarter. And then in implications for as we think about the Q4 as well, typically your strongest quarter, particularly in the U. S. For Ruknest.

Speaker 5

Any reasons why Q4 wouldn't still be your strongest quarter for the year?

Speaker 1

Thanks, Christian. That's an interesting question. Would you like to comment on that, Steve?

Speaker 2

Yes, sure. Thanks, Christian. So you're right. Q3 is strong and we occasionally see stock in, but it's sporadic. There's no real pattern to it.

Speaker 2

So I would still expect at this stage Q4 to be our strongest quarter and for us to

Speaker 5

Thanks. And then maybe a quick follow-up. I mean, anything I mean, you've called up the new patient starts. I mean, it's just Curious why you're still getting this very strong patient starts and physician uptake and anything else to comment on that

Speaker 2

Yes. I think it's interesting. We actually restructured our team a month before COVID hit and then we had to mothball them. So I think you're seeing a combination of different things happening, but certainly the restructure of that team is now paying off and that's why you're seeing actually an increase in enrollments and that System increasing enrollments across three quarters. So I think that makes a big difference.

Speaker 2

And the other thing I would say is we have a very well tenured team. So deep relationships within our physicians' offices and with their staff. And I think that's really helped in identifying new patients. I mean, as you would have I heard us allude to. We have a much broader mix of patients now than we have historically, whereas if at launch, as you would expect, it was refractory patients who were Predominantly using Bruton S.

Speaker 2

Now it's across mild, moderate and severe and it's pretty evenly spread. So I think it's a combination of time in market, Trust, execution and as I mentioned earlier, continued need for IV C1 esterase inhibitors despite the Quite disruptive changes to the market.

Speaker 5

Okay. Thank you. And then turning to Joenja then, If we can, just firstly on the European approval process, the advisory group committing Group meeting, as I understand, is still to be held. Is there a timing on that or any outcome that comes from that advisory group meeting?

Speaker 3

Hi, Christian. It's Anurag. So we this meeting has been scheduled. It is a closed meeting and we're not going to provide any further guidance on the ongoing regulatory interaction other than to say that we continue to expect The CHMP opinion in this quarter.

Speaker 5

Okay. Thank you. And then And then as we think about obviously the Q3 numbers around Patients on paid therapy and things, I mean, anything to flag in terms of expectations for Q4 versus the sort of current run rate of enrolled patients and patients on therapy. And then particularly maybe there's Any commentary insight you can give on the numbers of patients obviously presumably a large proportion on starters, but how many bridge Packs that people having to give you seem to imply you're getting patients on to paid therapy pretty quickly. But I don't know if Bridge is still something that's a reasonable factor in the mix.

Speaker 1

Maybe Steve, do you want to comment on that?

Speaker 2

Certainly. So again, as Alex said, Christian, we continue to aggressively pursue our patient finding efforts and actually we're ramping that We're through that early launch and conversion stage. In terms of starter and bridge, yes, I mean, we've actually of course, most of those patients have been on starter, but We're having to bridge very few. When we do, we bridge for a pretty short period of time relative to what we may have seen in with Rukonus back in the past and that's because of this Pretty fast approval rate and our ability to get commercial product in patients' hands quickly. So yes, It's going very well in that regard and we're not having to give away too much free stock.

Speaker 6

Okay, great. Thanks. I'll jump back in the queue.

Operator

Thank you. We will now go to our next question. And the next question comes from the line of Alastair Campbell from Royal Bank of Canada. Please go ahead.

Speaker 6

Thanks very much. Hopefully, you can hear me. Couple of questions, please. Another follow on on RUKENESS, which is a following on from Christian's question to an extent. But you're obviously pointing towards adding And that's growing at about give or take 10% per annum, but also the products growing at low single digits.

Speaker 6

So how should I think about that disconnect? Is that Price effect or is that lower utilization per patient or is it just the incremental prescribers you're adding are sort of less active? So that's question 1. And then question 2, if I could just sort of push my luck a bit on additional indications, looking at some of those publications you flagged in the presentation, I think One of the suggestions is you could look at areas where mTOR inhibitors are currently used given the same pathway. And that sort of brings to mind areas like autoimmune with things like transplant rejection, but there's also oncology areas like neuroendocrine tumors.

Speaker 6

Can I press you to see whether any of those areas are things you're thinking about right now? Thanks.

Speaker 1

So maybe the first question you want to answer, Stephen?

Speaker 2

Certainly. So you're right to flag, I think, the apparent or the perceived disconnect. For the most part, what I would say that is, is we went from 30% of patients on prophylactic therapy 3, 4 years ago Over 70% now in the mid-70s. So what you're seeing is better controlled patients having less attacks and therefore utilizing less acute therapy. But we haven't actually lost that many patients.

Speaker 2

What we've seen is and we think of it as cohorts of mild, moderate and severe. So not the disease itself, but the number of attacks. So we've seen patients in that severe end or frequent attacks moving to the moderate and from the moderate into the mild area. So it's more patients, more prescribing physicians, but often less severe course of disease leading to slightly less acute utilization.

Speaker 1

Can I

Speaker 6

quickly follow-up on that? If that sort of mix change in the patient population is, let's say, it's more or less played out, does that mean that perhaps as more physicians are added. So could that disconnect could actually lessen over time?

Speaker 2

Logically, yes, it could.

Speaker 6

Okay. Thank you.

Speaker 3

And then I think your question about our next indication, I think you're thinking along the same lines that we are. For example, we know that in APDS, in the past especially, but even now in some areas where adjuvant is not available, mTOR inhibitors are used. They have tolerability issues and they're not quite the perfect target for the condition itself. So we are looking at other areas where mTOR inhibitors are used and where lineolosib could We're not specifically, I can already comment that we're not specifically looking within oncology. I think it's something that we may do in the future, but at the present time really focused on other Rare diseases, where we think that this pathway is overactive That the some of the things that we see, basically trying to take the learnings from APDS, some of the features of APDS that we see, where else do we See that type of problem in the immune system.

Speaker 3

And I think that's what we're in active discussions with FDA on trying to finalize the clinical trial plan. And I expect to be able to give an update on that later this quarter.

Speaker 6

Great. Thanks very much.

Operator

Thank you. We will now go to the next question. And your next question comes from the line of Cecilia Hernandez by Landshut Kempen. Please go ahead.

Speaker 7

Yes. Thank you for taking my question. Could you walk us through the development of your operating Expenses, as you mentioned, we've seen an increase in marketing and sales cost due to the launch. But will this increase further or level Or is this level what we can expect for the coming quarters? And also on R and D costs with the second indication for leonadustat, Is it feasible to target profitability next year?

Speaker 7

Thank you.

Speaker 4

Yes. Thank you very much for the question, On OpEx for GEO and GEO, we will support the European launch Going forward, so we will shift probably some of the funds from the U. S. To Europe. On a net basis, it's too early to say What the outcome of that shift will be.

Speaker 4

And for yes, for next year also on the new indication, We will invest in R and D and in a trial. Again, there is too early to say what the exact cost is. I don't expect necessarily a reduction in OpEx next year.

Speaker 7

Okay. Thank you.

Operator

Thank you. We will now go to the next question. And your next question comes from the line of Jay Pantginis from H. C. Wainwright.

Operator

Please go ahead.

Speaker 8

Hey, guys. Thanks for taking the question. Couple, if you don't mind. So first, I wanted to focus on Stephen's comment on the HAE market. Obviously, there's a lot of disruptive changes to the market that are either ongoing or coming.

Speaker 8

And I'll phrase my question this way. Obviously, we know where we stand and where farming stands with regard to the role of Rukinest. But I guess Maybe more feedback from your new prescribers, for example, and even existing prescribers about the Potential threats that are coming even though they are focused on the prophylactic standpoint. So basically, the External views of needing a rescue therapy such as Rugenest.

Speaker 1

You want to comment on that, Stephen?

Speaker 2

Sure. We I mean, as you know, Joe, we morning by the way, we hold our boards pretty regularly. We're all of Active in the year myself and Anurag are regularly in front of customers in the U. S. And I think certainly Some physicians are quite excited by what may be coming in the future.

Speaker 2

I think what's interesting for me is Patients still need despite all of these disruptions that occasional bolus of The therapy that you get from an IV product such as RUKENESS, so although we see disruption and as I mentioned earlier, we see sometimes Decrease or in some places even a normalcy one, an increase in utilization. We still see the need there. So I certainly wouldn't want to be complacent and we look at the future as closely As any company would, but through all of the disruptions, certainly since I joined FarmLink 7 years ago, we see that initial period of And then we see things settle down and we see a continued clinical need for an IVAC myosorase inhibitor. So Does that answer your question, Joe?

Speaker 8

No, no, it certainly does. I appreciate that. And I guess looking more towards Just switching a little bit to Joenja. Assuming a positive I know previously you discussed targeting Germany first, but how should we view beyond that, the how things should go?

Speaker 2

Do you want me to take that on?

Speaker 1

No, no. That's him. Go ahead.

Speaker 2

Yes. Thanks, Joe. The you're right. I mean, Germany would be the typical market To go to right out of the gate and we'll certainly do that. We also will then target the other big 4 in Europe.

Speaker 2

Arag mentioned the U. K. And a submission there. Now it's Specifically, there, no, it's not part of the EU. And then we'll go through Spain, Italy, France as major nations, but we won't ignore the rest of Europe either.

Speaker 2

So we have a pretty lean operation in Europe and we cluster the other 22 member states around those major markets. And you'll see us through 2024 2025 in a steady sequence start to go through all those markets. And by the way, we've identified patients, I think in every single one of those markets. And in addition to that, we have submissions in Australia, which will enable us to set up The base of operations in APAC in the key markets outside of Japan, the ongoing trial in Japan and a submission with Health Canada right now as well as that, I believe, Thanks for that market globally. So I think we have a pretty careful, well considered sequenced approach that gets us into markets at the right time.

Speaker 2

And you'll see that pointed out through 2024 and 2025.

Speaker 8

Got it. And then just lastly, I guess it's a quick logistical question. With regard to the VUSs and unclassified variants, if you will, is there anything that needs to be done as you generate data about these variants on the regulatory front or in the label to identify these?

Speaker 3

Hey, Joe. Good morning. So the answer there is no, because these patients actually have APDS and the label is That is for APDS. Right now, the question is this variant, which hasn't been previously described or previously published, When the genetic testing company gets that result, they don't know what to do with it. So they throw it into this bucket of the U.

Speaker 3

S. And many, many results come back into this VUS classification. But once that is reclassified based usually on functional testing data Or even as I mentioned that using that multiplex approach, once that's reclassified, then that patient has APDS and would qualify for treatment For the label.

Speaker 8

Great. Thanks for the clarification and thanks for all the answers guys.

Operator

Thank you. We will now go to your next question. And your next question comes from the line of Hartaj Singh from Oppenheimer. Please go ahead.

Speaker 9

Great. Thank you. Thanks for the I got a couple of questions and really nice update everyone. The two questions I have, just following up to a previous question on the cadence of the launches ex U. S.

Speaker 9

I Australia, Canada, Israel in 2024 and 2025 Europe later this year and then discussions there. Can you just give us an idea of the relative TAM of that market? And then could there be boluses in Europe, the UK, Australia, Canada, because you have been working very hard to identify patients. I imagine there are patients in these various territories Ready to get on drug and then how would pricing potentially look relative to the U. S.

Speaker 9

Market? So that's the first question. The second question is just on your family testing. Previous research we have done before lenulosib was approved indicated there could be as little as one more family member and as much as three more Extended family members that might have some indication of APDS and could potentially qualify for treatment. I know these are early days, But if you could just give us some color around there, what do you expect to see as you wrap up this tablet test?

Speaker 1

Steve, do you want to comment on those patient numbers ex U. S?

Speaker 2

Certainly. And I didn't quite catch the front end of the question, Hitash. So if I miss anything, then please just pick me up as we go through. So I think you're right to flag that the medical affairs group across the world have been actively identifying patients We're key opinion leaders in those key centers in each country. So there will be a bolus of patients awaiting therapy.

Speaker 2

Many of them will already be in the early access program, for example. There will be a slight delay because from approval in many of those countries, you then need to negotiate reimbursement, Which means we set the price slightly later than the clinical approval. But in terms of your pricing question, The price outside of the U. S. Will, as you know, for the most part always be lower.

Speaker 2

It's highly unlikely it will match that price. And there'll be a variation in what that price looks like country by country. What I would say though is without preempting what that price might look like is As with all ultra rare diseases and rare diseases, we can still make a market and build a very healthy business in each of those countries. And that's fully what we expect to do.

Speaker 1

Thanks, Steve.

Speaker 9

That's great, Stephen. And then just on the question of the family testing and then how big could that patient population be, just Roughly speaking. Thank you.

Speaker 3

Yes. So Hartaj, it's a great question. It's certainly something that we're trying to address right now, Which is, we know it's an autosomal dominant transmitted disease. So we expect that there should be Other family members with the condition. And as we said many times, it may not be immediate family members, even extended family members.

Speaker 3

What we've been trying what we've been a little surprised by those and then I guess it relates to the fragmented nature of our healthcare system Is that oftentimes these family members haven't been tested. Some of that is just due to lack of awareness, even amongst patients about the genetics of the disease. And some of that is just due to the healthcare system idiosyncrasies and how it's difficult to get genetic testing done. So we were changing the way we approach this and really moving putting the patient right at the center of this. So allowing patients and families To actually initiate testing.

Speaker 3

So if a patient is diagnosed with APDS and a family member wants to get tested, We've started a program that will allow that to happen by having the family member themselves initiate the process and it doesn't need to go through For example, the patient specialist who may not be immediately available to see the family member, for example, or may not even be a patient of the family member in most cases. So I think Removing these barriers to genetic testing for to allow appropriate testing in families is I think a will likely be a significant Source of newly diagnosed patients. And we're starting that program. We started a little bit of that already, but really putting that in full force now.

Speaker 9

Great, Anurag. That helps. Thank you everyone for the questions.

Operator

Thank you. We will now go to the next question. And your next question comes from the line of Simon Scholes from First Berlin. Please go ahead.

Speaker 10

Yes. Hello. Thanks for taking my question. So you've already identified 150 APDS patients over the age of 12 in the U. S.

Speaker 10

I was just wondering how many of those patients do you ultimately expect to be able to enroll?

Speaker 1

I'd say The vast majority of those, Simon, well, almost all of them. Okay. So almost all. And that's just the beginning, right, because you heard.

Speaker 10

Yes, I know there are other patients, but I'm just interested in patients you've already identified, so we should assume over 90% or 95%.

Speaker 1

I think there will be a very high percentage of patients that indeed will be interested to get into dementia treatment, correct. That's our experience so far at least.

Speaker 10

Okay. And particularly in the U. S, you don't expect to encounter a problem with the last 10% or 20% because of lack of insurance coverage. I mean, how does that work? I mean, presumably, there will be some patients without insurance coverage.

Speaker 10

I mean, can you get those as paid therapy on paid therapy as well?

Speaker 1

Although those questions are difficult to answer, but generally speaking, if we look at our experiences with RECONEST, We don't see any issues related to that coming up. And of course, Luca Nest also has patients that have limited or no insurance coverage, but Maybe you want to comment on that, Stephen?

Speaker 2

Yes. I think there's I mean, there's all kinds of different types of support available for patients and also public programs that try and make sure that patients have access Something. So to be honest, Simon, I haven't looked at this for a while, but I believe we have very, very few rucanous patients, for example, in our Patient assistance program, which will be essentially free supply on an ongoing basis.

Speaker 1

And

Speaker 2

even when they are in there every year, we're working with them and with their physician To find an insurance plan for them, we'll find an option that we'll need to pay therapy. So I apologize, I can't give you a specific answer. But if I look at the Rucanest experience and I think the vast majority of patients in the end will get will be on paid therapy. It can sometimes just be a heavy lift in those last few percent together.

Speaker 10

Okay. Thanks very much. That's very helpful.

Operator

Thank you. We will now go to the next question. And your next question comes from the line of Christian Glenny from Stifel. Please go ahead.

Speaker 5

Hi, guys. I thought just quick follow-up if I can on Joanna and the pediatric study in the 4 to 11 year olds. Looking at enrollment almost complete there. So just a reminder in terms of the endpoint for that trial, the timing of the And therefore, when we might see data. And then the expected sort of rough mix of the under 12s as it relates There's 4 to 11 year olds.

Speaker 5

I mean, if you think about, say, the 50 remaining patients identified in the U. S. That we know, How many of those would be 4 to 11?

Speaker 3

So let me answer that question the second question first, Christian. So the again, we based on our current experience in APDS, about a quarter of patients overall are below the age of 12. Amongst that quarter, threefour are in the age range At least 3 quarters are in the age range of the first study or 4 to 11 year old study. We know these patients actually have the disease at birth and many patients do begin to manifest symptoms early on, But oftentimes, they're not diagnosed at that very early age. And that results in many patients in this in the older age groups, especially in that above for age group.

Speaker 3

So that's a little bit on the breakdown of the age distributions across APES. In terms of the pediatric study endpoints, the endpoints that we had in the adolescent and adult study. So I think what we'll be able to do is once the study is fully enrolled and we have these last four patients enrolled, we'll give you some more guidance on The timing of the data releases as well as some of the regulatory work that we anticipate being able to do if the results are positive.

Speaker 6

Okay. Thanks. That's helpful.

Operator

Thank you. There are currently no further questions. I will hand the call back.

Speaker 1

All right. Thank you very much. A few closing remarks. Thanks for attending. You can see now that as stated last quarter, the company is now starting a long Growth trajectory supported by the foundation that Rouxenext provides and of course driven by the future expansion of Joenja outside of the United States, but also inside of the United States, supported by lots of efforts that we are undertaking and initiating to actually broaden the patient base.

Speaker 1

And of course, As always, with new immune with new genetic diseases, the definition of the disease will broaden. So therefore, we look forward to the future with optimism and with a company that will significantly grow and change over the coming years, putting us again as a combination of our commercialization capabilities, clinical development and regulatory skills, Again, as hopefully the ideal partner or go to partner in the future for other rare disease assets that we can take on board and actually complete the clinical developmentapprovals and do the successful Commercialization as we do with Rukunest and with Joenja. So that said, thank you very much for attending, and we look forward to updating you again on our full year results call, which will be in March of next year. Thank you very much. Goodbye.

Operator

Thank you. This concludes today's conference call. Thank you for participating. You may now disconnect.

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Earnings Conference Call
First Solar Q3 2023
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