Q3 2023 Pharming Group NV Earnings Call
Speaker 1: transcript
Speaker 1: Oh.
Okay.
Speaker 2: transcript
Speaker 2: Good day and thank you for standing by. Welcome to the farming group and the third quarter, 2023 Results Conference call and webcast. At this time, all participants are in a listen only mode. After the speakers presentation, there'll be a question and answer session. As a reminder, the company will only take questions from dial-in participants.
Good day, and thank you for standing by and welcome to the farming Group N V. Third quarter 2023 was always conference call and webcast. At this time all participants are in a listen only mode. After the speaker's presentation there'll be a question and answer session. As a reminder, the company will only take questions from Darling.
Speaker 2: transcript
Speaker 2: To ask a question during the session, you will need to press Star 1-1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press Star 1 and 1 again. Please be advised that today's conference has been recorded. I would now like to hand the conference over to your first speaker today. Simon DeFrees, CEO of Farming Group. Please go ahead, sir.
Participants to ask a question during the session you will need to press star one on your telephone you will then have an automated message advisor knowhow in this race to withdraw your question. Please press star one on one again. 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.
C O a forming group. Please go ahead sir.
Speaker 3: transcript
Speaker 3: Thank you very much and good morning all good afternoon ladies and gentlemen. I'm here with my three colleagues, Steven Thor, Chief Commercial Officer, Andrew Allen, Chief Medical Officer, and Jeroon Wacomon, Chief Financial Officer.
Thank you very much and good morning, or good afternoon, ladies and gentlemen.
David My three colleagues are Stephen <unk>, our Chief commercial Officer, Underactive Allen, our Chief Medical Officer, Andrew <unk>, Our Chief Financial Officer, and we are delighted to take you through the third quarter a result of.
Speaker 3: transcript
Speaker 3: and we are delighted to take you through the third quarter 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, statements of future expectations that are based on our current expectation assumptions.
This year before I do that however, I would like to point you to the forward looking statements flights because we may contain this presentation may contain real probably contain forward looking statements as you know our statements of future expectations that are based on our current expectations and assumptions and involve known and unknown risks and uncertainties.
Speaker 3: transcript
Speaker 3: and evolved known and all our risks and uncertainties that could cause actual results, performance or events to different material from those expressed or implied to be statements. And the rest I leave to you to read.
Could cause actual results performance or events to differ materially from those expressed or implied in these segments and the rest I leave to you to read them.
Speaker 3: transcript
Speaker 3: So let's just move on to the next slide and of course to the next slide about building a sustainable business in rare diseases.
So, let's just move on to the next slide and then of course to the next slide about building a sustainable business.
Speaker 3: transcript
Speaker 3: And that's what we are about. And this is of course, the very interesting moment in time, water results of 2023. And you see that on the left hand side, how we are going to build, start to build that rare disease, sustainable rare disease business.
In rare diseases, and that's what we are about this.
This is of course.
Barry.
Interesting moment in time.
The results of 2023, and you see that on the left hand side, how we are going to build that start to build that.
Rare disease sustainable rare disease business.
Speaker 3: transcript
Speaker 3: We have significant positive guidelines for more than 200 million of moving annual total sales of Rookanest that confront the geo-enja launches and pipeline development to start with. And we are a great least of course with results and a strong revenue growth of Rookanest, 18% up on the second quarter and 11% up on last year's third quarter.
I have significant positive cash logs for more than 200 millions.
All right.
Alright, moving annual total sales of Brokenness that confronts a joanne Jacques and launches and pipeline development to start with and we are a great piece of course, which results in a strong revenue growth <unk>.
18% up on the second quarter, and 11% up on last year's third quarter.
Speaker 3: transcript
Speaker 3: And also, if you look back nine months, so here to date, through percent up on last year, that means that we are on track to deliver our low single digits revenue growth for Rukinesse for 2023.
And also if you look back nine months year to date, 3% up on that last year that means that we are on track to deliver our.
Low single digit revenue growth for <unk> for 2023.
Speaker 3: transcript
Speaker 3: And then of course, you move to the middle pillar, and you see there of course the global approval and commercialization of Joe NJAP that can be funded from those cash flows, from the recognized. And of course, we were pleased, very pleased to get that very fast approval from the FDA back in March, brought the product to the market and got reimbursable, reimburs patients almost immediately when we were on the market.
And then of course, you move to the Middle pillar and you see there of course, the global approvals and commercialization of Joanne jobs that can be funded from dose gaslog recognize.
Of course, we are pleased very pleased to get that very fast approval from the FDA back in March brought to the products through the markets.
Reimbursable reimburse patients almost immediately when we are on the market.
Speaker 3: transcript
Speaker 3: First, we go already to record revenues in the second quarter of this year, which was the first quarter of June . It was on the market. And now, of course, we're very proud of the continued growth of June . We're in this quarter, we've booked 6.5 million of revenues and year-to-date 10.3 million.
So should we go already record revenues in the second quarter of this year, which was the first quarter, Georgia, what's on the market and now of course, we're very proud of the continued growth of your edge outs.
This quarter, we booked $6 million of revenues and year to date $10 3 million.
Speaker 3: transcript
Speaker 3: In addition to that, of course, the regulatory reviews are ongoing for Joenja in Europe , Canada, Australia, Israel, and we have a PDIT critical trial program ongoing as our label is currently 12 years and upwards.
In addition to that of course, our regulatory reviews are ongoing for Japan, and Europe, Canada, Australia Israel.
And we have a pediatric clinical trial program ongoing as our label is currently 12 years and upwards.
Speaker 3: transcript
Speaker 3: And of course, on the right hand side, you see further growth accelerators beyond Joenja in APDS, first 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 CFEA about the second Alliance indication and will provide you with more details to our end of the year and colleague Anna Rack, rather than the fourth by the more. And last but not least, as we have a very strong commercialization,
And of course on the right hand side, you see further growth accelerates beyond Joanne giant <unk> first and foremost and that is why we will come back to you.
Before year end to update you on box, we are in dialogue with the FDA.
The second.
<unk> indication and we will provide you with more details towards the end of the year in Iraq right out of the book.
But more and loss of Adobe's as.
As we have a very.
Strong commercialization.
Speaker 3: transcript
Speaker 3: structure in both the US and in Europe . We're hunting as we speak for new analyzing opportunities or acquisitions for additional products in rare diseases that we can actually continue to develop, build development and bring to the market and successfully commercialize. So we're looking for products that have clinical growth process.
Infrastructure in both the U S and Europe were hunting as we speak for new in licensing opportunities or acquisitions.
Additional products in rare diseases that we could actually continue to develop critical development and bringing to the market.
We commercialized so will it be for products that have clinical proof of concept.
Speaker 3: transcript
Speaker 3: And if you see at the next slide, you see that there is actually space to have such products in our pipeline. You see here the extensive work we do by enlarging our footprint by means of bringing many other zip to markets and field like, as far as fields as Japan, but also Canada, Australia and Israel, and of course the other indication. So you see there is space in this pipeline to further accelerate the growth of the company going forward.
As you'll see at the next slide.
There is actually space.
To have such products in our pipeline, we see here. The extensive work we do by enlarging our footprint by means of the ownership to markets.
Feels like as far afield, as Japan, but also Canada, Australia, and Israel and of course, the other indication. So you see their space in this pipeline to further accelerate the growth of the company going forward.
Speaker 3: transcript
Speaker 3: I would add that I would like to now hand over to my colleagues Steven Tor, who will give you some more insights in the commercialization operations on the Rukinesh and Duanej. Steven, over to you.
With that said I would like to now hand over to my colleague Stephen tore who will give you some more insights into commercialization operations.
And Georgia, Steven over to you.
Speaker 4: transcript
Speaker 4: Thank you Simon, good morning everybody. As Simon said, I'll give you a brief overview of a Rookinesse performance and also some insights around the Joinger launch and update you on that progress today.
Thank you Simon good morning, everybody.
And Simon said I'll give you a brief overview of our refinish performance and also some insights around the Joe and <unk>.
Are you on the progress to date next slide please.
Speaker 4: transcript
Speaker 4: So I communicated at the end of Q1 and as you're all aware, the H8 market under a significant event which affected all products. The event was short-lived and as we said, we were, we bounced back strongly in Q2 and Q3.
So as communicated at the end of Q1 and as you are aware the HLA market.
Underwent a significant event, which affected all products. The event was short lived and as we said we would we bounced back strongly in Q2 and Q3.
Speaker 4: transcript
Speaker 4: As you can see in the first bullet, we've posted strong growth in Q3, and even with the soft NIFQ1, as the Simon showed, we have grown versus prior year, so we're really pleased with that performance.
As you can see the first bullet we posted strong growth in Q3, and even with the softness of Q1 as Simon showed we have grown.
Versus prior year, So we're really pleased with that performance.
Speaker 4: transcript
Speaker 4: 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...
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 <unk>.
Stronger than we've seen in.
Speaker 4: transcript
Speaker 4: And so hence Simon said we continue to go to go to go to go to the low single digit growth for Ruchen as this year.
In the past.
And so hence as Simon said, we continue to.
Got it for low single digit growth for recognize this year.
Slide please.
Speaker 4: transcript
Speaker 4: So as many of you know, Rukinespa's launch in 2015. We've actually been active in the HA 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.
So as many of you know <unk> was launched in 2015, we've actually been active in the HLA community since around 2000.
And over those 23 years, we collaborated with key stakeholders, including the clinic ones and patient efficacy groups such as the Hai.
Speaker 4: transcript
Speaker 4: And that has been the remaided driver for the consistent success of Ruchenest over the nine years post launch. And it's why the prescriber base continues to grow with 700 in the US today. And also why we've retreated over 2000 patients and that metric continues to grow as well. So I think what that clearly underlines is the importance and the ongoing need for a recombinant IV, C1S2 raise inhibitor. And that's despite the fact over 70% of patients are now on prophylactic treatment today.
And that's been the major driver for the consistent success of <unk> over the nine years post launch and it's why the prescriber base continues to grow with 700 in the U S to date and also why we've treated over 2000 patients in that metric continues to grow as well so.
So I think that clearly on the lines is the importance and the ongoing need for recombinant <unk> inhibitor and that's despite the fact over 70% of patients on oral prophylactic treatment today.
So next slide please.
Speaker 4: transcript
Speaker 4: So moving to Jo Enger, as Simon showed earlier in the presentation, we're off to a very strong start in the US with our launch.
So moving to Joe and Joe Simon showed earlier in the presentation, we're off to a very strong start in the U S with our launch.
Speaker 4: transcript
Speaker 4: And as indicated in previous calls, and I think there's everybody on this call nose 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're part of with an organization called Panther Rx that specializes in ultra rare and rare diseases.
And as indicated in previous calls and I think as everybody on this call knows in relation to the environment more broadly.
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 Panthera about Rx that specializes in ultra rare and rare diseases.
Speaker 4: transcript
Speaker 4: to build a program that we thought would enable us to quickly provide patients with access to to
Build a program that we thought would enable us to quickly provide patients with access to Joe and Joe.
Speaker 4: transcript
Speaker 4: once they're covered for chronic use. And I'm pleased to report that in only six months' post-launch, Farman's access to medical teams in partnership with key opinion leaders across the country has secured APDS coverage policies in over 90% of our target plans across...
Once that covered for chronic use and I'm pleased to report that in only six months post launch farm ins access of medical teams in partnership with key opinion leaders across the country.
<unk> <unk> coverage policies and over 90% of our target plans across.
Speaker 4: transcript
Speaker 4: that's across commercial and government payers. And the result is a 93% approval rate with zero denial.
That's across commercial and government payers and the result is a 93% approval rate with zero denials.
Speaker 4: transcript
Speaker 4: And so I just want to repeat those two. 93% approval rate was zero denials, despite the rarity of this condition and the heavy lifting education. So that's really a very strong book.
And so I just want to repeat those 293% approval rate was zero denials. Despite the rarity of this condition and the heavy lift and education. So that's really a very strong performance.
Speaker 4: transcript
Speaker 4: Also, you know, from enrollment to ship into patients, the gold standard in rare diseases 30 days. I'm pleased to report to you that we're averaging 26 days typically and sometimes are getting from enrollment to put in product and patients' hand in less than 20.
Also as you know for.
From enrollment to ship into patients the gold standard in rare diseases, 30 days and I am pleased to report to you that we are averaging 26 days typically and sometimes you're getting from enrollment to putting product in patients and in less than 20 days.
Speaker 4: transcript
Speaker 4: And really this is based on exceptional customer focus and execution, which further instills, I believe, in our confidence in our stakeholders, but most importantly, the patients and treating positions.
Really this is based on exceptional customer focus and execution, which further it still was I believe.
Our confidence in our stakeholders, but most importantly the.
Ah patients and treating physicians.
So next slide please.
Speaker 4: transcript
Speaker 4: So I've mentioned, you know, farming strong customer and patient focus, and I think combined with the exceptional execution, I've already mentioned a US team of delivered strong results in that first six months since law.
So I've mentioned.
Farming strong customer and patient focus and I think combined with the exceptional execution I have already mentioned our U S. Team has delivered strong results in that first six months since launch.
Speaker 4: transcript
Speaker 4: We have as a slide show 70s intelligible patients.
We have as <unk>.
Slide show 76 eligible patients 63 shipping and that represents well over half of the eligible patients we have on therapy.
Speaker 4: transcript
Speaker 4: 63 shipping and that represents well over half of the eligible patients we have on therapy.
Speaker 4: transcript
Speaker 4: This is led to the revenues of 10.3 million the Simon also showed. And as I already mentioned, pay discussions haven't continued to go very well, which creates importantly an excellent environment for us to pool those patients through once they're diagnosed and enrolled.
This has led to the revenues of $10 3 million of the southern also showed.
As already mentioned payer discussions have continued to go very well, which creates importantly, an excellent environment for us to pull those patients through once they're diagnosed in the road.
Speaker 4: transcript
Speaker 4: 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 on resources now, family testing. Most of the patients we have are patient zero, so to speak. So we believe there's a lot of opportunities there to help those families by better educate them and testing all of them to see whether there are others in need of therapy.
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 family testing most of the patients we have a patient zero. So to speak. So we believe there is a lot of opportunity there to help those families, but better educating them and test and all of them to see.
Whether there are others in need of therapy.
Speaker 4: transcript
Speaker 4: And with that, I'd like to hand over now to our CMO doctor Dr. Anna Regrim.
And with that I'd like to hand over now to our CMO Dr talks around our growth.
Thanks, Steve.
Speaker 5: transcript
Speaker 5: 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, or 1500 patients with APDS. We have already found more than 640 of those patients.
I'll begin on the next slide with a little background information about Aps. So Aps was first described in 2013 and based on our estimates and literature review, we believe that there are more than <unk> hundred patients worldwide diagnosed with atvs.
<unk> hundred patients with Aes, we have already found more than 640 of those patients.
Speaker 5: transcript
Speaker 5: These patients who have APDS have really had limited treatment options until recently to all of the symptoms of the disease. The disease manifests itself in childhood and worsens over time, without anything specifically indicated for treatment. Positions and patients were quite limited in their treatment options.
These patients who have <unk> have really have limited treatment options until recently to only treat the symptoms of the disease the disease manifests itself in childhood.
Worsens over time without anything specifically indicated for treatment.
<unk> and patients we're quite limited in their treatment options.
Speaker 5: transcript
Speaker 5: And as with most rare diseases, the signs of symptoms vary across patients. This makes the challenge of diagnosis even more difficult beyond just a rare disease.
And as with most rare diseases, the signs and symptoms vary across patients. This makes the challenge of diagnosis, even more difficult.
But just a rare disease.
Speaker 5: transcript
Speaker 5: Fortunately, there is a genetic test that can provide a definitive diagnosis for A.K.D.S. and it will be spending more time in the coming slides talking about our plans and efforts to help find more patients with A.K.D.S.
Fortunately there is a genetic test that can provide a definitive diagnosis rate definitely spending more time in the coming slides talking about our plans and efforts to help find more patients with atvs.
Speaker 5: transcript
Speaker 5: On the next slide, we can see what Joenja now brings to patients in the U.S. as a potential treatment option for them or their condition. It is approved by FDA for the treatment of APBS 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 meetings to girls that significance other clinical, politically relevant endpoints.
On the next slide we can see what <unk> brings to patients in the U S.
Potential treatment option for them or their condition. It is approved by FDA for the treatment of Atvs in adults and pediatric patients from ages 12 years old and older.
We have randomized clinical trial data showing that Joanne <unk> met both primary endpoints as well as meeting a.
A significant other clinical clinically relevant endpoints.
Speaker 5: transcript
Speaker 5: In addition, we've seen a well tolerated and generally safe adverse event profile that were no drug-related serious adverse events in the study or withdrawals due to the drug in the study.
In addition, we've seen a well tolerated and generally safe adverse event profile there were no drug related serious adverse events in the cellular withdrawals.
Due to the drugs in the study.
Speaker 5: transcript
Speaker 5: And more importantly, we have long-term data and I'll be sharing some of that with you that we've been 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, some in some cases, to discontinue the use of immune globulin replacement therapy, reduction in infection rates, and persistence of the benefits that we see from the randomized clinical trial.
And more importantly, we have long term data and I'll be sharing some of that with you that we have been publishing and presenting at conferences recently.
About the long term benefits of using Joanne.
Over several years in many cases and this includes patients some in some cases to discontinue the use of immune globulin replacement therapy reduction in infection rates and persistence of the benefits that we see from the randomized clinical trial.
Speaker 5: transcript
Speaker 5: And we can see that both in their in in key measures of what's called lymphopuloperation. So their lymph nodes continue to stay not enlarged. And we see benefits also in their immune cell function.
And we can see that both in there.
<unk> measures of what's called Lymphoproliferative, so their lymph nodes.
Can you to say.
Not in large and we see benefits also in their immune cell function.
Speaker 5: transcript
Speaker 5: And as Steve mentioned, this is 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 conditions.
And as Steve mentioned this has led to a strong start for joining John I think this speaks both to the unmet need that exists in this Aps population, but also speaks to the seriousness of the condition.
Speaker 5: transcript
Speaker 5: 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. Now, as we discussed in August , we received the day 180 list of outstanding issues from the European Medicine Agency, and we can confirm now that in October we have submitted our response.
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 two months later if.
Speaker 5: transcript
Speaker 5: We remain on track to expect an opinion in this quarter and with potential approval two months later.
Speaker 5: transcript
Speaker 5: If we received 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 two months later. And as we previously announced, we've started a program in Japan to enable registration there eventually. And we've also now filed in Australia, Canada, and Israel, and those applications are proceeding along their review plan.
If we receive a positive opinion.
The <unk> in this quarter. We can then go ahead and file with the U K NHRA agency.
With potential approval also two months later and as we previously announced we have started a program in Japan to enable registration there eventually and we've also now filed in Australia, Canada, and Israel and those applications are proceeding along their review plants. We've also started a named patient program.
Speaker 5: transcript
Speaker 5: 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 one to six year old study has now started recruiting, so we expect that first patient also to be treated very soon.
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 is already complete in the four to 11 study and the 1% to six year old study has now started recruiting so we expect that first patient also too.
We treated very soon.
Speaker 5: transcript
Speaker 5: 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.
And as Simon mentioned, we have been engaged with FDA about second indication and we expect to be able to provide further details on the second indication later this quarter.
Speaker 5: transcript
Speaker 5: 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 Leneolos that we can share. These data highlight the seriousness of APDS and I think it also highlights the experience that we have with Leneolos have been treating many of these patients.
On the next slide I want to review with you some of the patient finding efforts that we initiated and are ongoing at this time.
First of course is <unk> is a rare disease and it's critical to raise awareness about Aps and now we have a plethora of data also on lineal so that we can share.
These data highlight the seriousness of eight Etfs and I think it also highlights I think the experience that we have with Lenny also been treating many of these patients.
Speaker 5: transcript
Speaker 5: On top of that, we have our ongoing Navigate APDS program that offers no cost testing available to patients in the U.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.
On top of that we have our ongoing navigate Aps program that offers no cost.
<unk> available to patients in the U 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.
Speaker 5: transcript
Speaker 5: 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 US, especially, we find that most of those patients actually don't have family members that have even been tested.
And then a big effort that we've really pushing on right now is that when we look at the diagnosed patients that was heavily U S, especially we find that most of those patients actually don't have family members that haven't even been tested and we know that Aps is an inherited disease, but there is this gap in terms of testing.
Speaker 5: transcript
Speaker 5: And, you know, 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 family members themselves, to be able to reduce the barriers to allow further testing amongst family members, which we think will be important to help uncover and find more patients.
So we have initiated several efforts here, both with physicians and with family members themselves to be able to reduce the barriers to allow further testing amongst family members, which we think will be important to help uncover and find more patients.
Speaker 5: transcript
Speaker 5: On the next slide, I want to review with you a little bit of information on something called Variance of Uncertainty Signific.
On the next slide I want to review with you a little bit of information on something called variants of uncertain significance and what these are genetic test results that are basically unclear or.
Speaker 5: transcript
Speaker 5: And what these are, are genetic test results that are basically unclear or I would better say unclassified.
I'd better say unclassified.
Speaker 5: transcript
Speaker 5: And with the growth in genetic testing, we get more of these inconclusive results. These are basically variants that have not been previously seen.
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.
Speaker 5: transcript
Speaker 5: And this is a, this is really frustrating for patients and doctors because they have patients who have clinical symptoms of APD as often, but the genetic test result is inconclusive.
This is <unk>.
Really frustrating for patients and doctors because they have patients who have clinical symptoms of Ats often with the genetic test result is inconclusive and so we have several efforts ongoing in I'm not going to review all of them with you in detail here, but these efforts involve trying to review the existing data.
Speaker 5: transcript
Speaker 5: We have several efforts ongoing, and I'm not going to review all of them with you in details here, but these efforts involve trying to review the existing data and try to co-late all of that information and publish that. We just started a partnership, for example, with Genomenon to develop these genomic landscapes.
We can try to coordinate all of that information and Publix published that we've just started a partnership for example, with genome anon to develop these genetic genomic landscapes, which will be available to all clinicians to be able to easily access variant information. We have a number of efforts ongoing to increase the availability of functional testing.
Speaker 5: transcript
Speaker 5: which will be available to all clinicians to be able to easily access variance information.
Speaker 5: transcript
And then lastly, I think I'm really excited about this possibility.
This new effort that.
Speaker 5: transcript
Speaker 5: 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.
We haven't that we started.
Looking at a way to in a single experiment tests, all possible variance and quickly determine whether a result is pathogenic or disease, causing or not.
Speaker 5: transcript
Speaker 5: And I think the nice thing here in a sense is that this is a problem, it's a new problem for APDS, 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.
I think the nice thing here in a sense is that this is a problem. It's a new problem for Aps, 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 patients.
Patients who.
They still have this unclear diagnosis.
Speaker 5: transcript
Speaker 5: On the next slide, you can see so 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 conditions, or 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.
<unk>.
On the next slide you can see some of the conferences, we've been presenting at some of these abstracts. We presented these abstracts very both in terms of what we're talking about in terms of the serious seriousness of the condition. So the mortality for example associated with Aps, but also the health care costs associated with Atvs and especially untreated.
Operator: Good day and thank you for standing by.
Operator: Welcome to the Farming Group and the 3rd quarter 2023 Results Conference call and webcast. At this time, all participants are in a listen only mode.
At Ats.
Operator: After the speaker's presentation, there'll be a question and answer session. As a reminder, the company will only take questions from dial-in participants. To ask a question during the session, you will need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 1 and 1 again. Please be advised that today's conference has been recorded.
These data I think highlight very nicely.
The serious burden.
These aps patients face on top of that we continue to get more data out of our clinical trial program. So we have a second interim analysis that will be published at the <unk> Conference next month.
Speaker 5: transcript
Speaker 5: On top of that, we continue to get more data out of our clinical trial program.
Speaker 5: transcript
Speaker 5: So we have a second intermanals that will be published at the IPEC conference next month. And we also have a number of case series as well as
We also have a number of case series as well as abstracts on single patients. These are from many times from our expanded.
Speaker 5: transcript
Speaker 5: on single patients. These are from many times from our expanded program or compassionate use program.
Operator: I would now like to hand the conference over to your first speaker today.
Simon DeFrees: Simon DeFrees, CEO of Farming Group, please go ahead sir. Thank you very much and good morning all good afternoon ladies and gentlemen. I'm here with my three colleagues, Stephen Toor, Chief Commercial Officer, Andrew Allen, Chief Medical Officer, Andrew Luckamon, Chief Financial Officer and we are delighted to take you through the 3rd quarter Results of this year.
Speaker 5: transcript
Speaker 5: where, for example, the second bullet under the eye pit heading is a patient who was previously transplanted, unsuppressedfully unfortunately, but then was treated with Leniolusib under this compassionate use program and the...
Program or a compassionate use program where for example, the second bullet under the.
Heading as a patient who was previously transplanted unsuccessfully unfortunately, but then was treated with any oldest of under this compassionate use program.
Speaker 5: transcript
Speaker 5: Data at the abstract will show the benefits of this patient was able to experience.
The data at the abstract will show the benefits of this patient was able to experience.
Speaker 5: transcript
Speaker 5: On top of that, on the next slide, you can see some of the publications. So the first publication is the first interim analysis, and that's available now in a full paper. And then the next publication is also a key publication describing the mechanism of action of lenulose in APBS. And I think it describes clearly how APBS leads to this primary immune deficiency with this immune dysregulatory phenotype and how lenulose benefits in these patients.
Simon DeFrees: 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, our statements of future expectations that are based on our current expectation assumptions and have all known and all our risks and uncertainties that could cause actual results, performance or events to different material from those expressed or implied to the statements and the rest I leave to you to read. So let's just move on to the next slide and of course to the next slide about building a sustainable business in rare diseases and that's what we are about and this is of course the very interesting moment in time, more to results of 2023 and you see that on the left hand side, how we are going to build that start to build that rare disease, sustainable rare disease business.
On top of that on the next slide you can see some of the publications. So the first publication is the the first interim analysis and that's available now.
The whole paper and then the next publication is also a key publication describing the mechanism of action of <unk> and Aps and I think it describes clearly how Aps leads to this primary immune deficiency with this immune dysregulation phenotype and how linear also benefits in these patients.
Speaker 5: transcript
Speaker 5: And with that, I'll turn it over to my colleagues, Yerun Walkerman or Chief Amantso Walsh.
And with that I'll turn it over to my colleague <unk>, our Chief Financial Officer.
Speaker 6: transcript
Speaker 6: Thank you very much, Anirak. Focusing first on the financial highlights of the third quarter, 2023, total revenues increased to 66.7 million, so an increase of 12.5 million or 23%.
Thank you very much on Iraq.
Focusing first on the financial highlights of the third quarter 2023.
Total revenues increased to $66 7 million, an increase of $12 5 million or 23%.
Speaker 6: transcript
Speaker 6: Gross profit increased to 58.4 million and increase of 6.5 million Operating cast increased from 44.7 to 56.8 and the increase of 12.1 million is mainly because of R&D in this additional investment.
Simon DeFrees: We have significant positive guidelines for more than 200 millions of moving annual total sales of rukenests that confront the geo-enja launches and pipeline development to start with and we are very pleased of course with results and a strong revenue growth of rukenests, you know 18% up on the second quarter and 11% up on last year's third quarter and also if you look back nine months so here to date through percent up on last year that means that we are on track to deliver our little single digits revenue for rukenests for 2023 and then of course you move to the middle pillar and you see there of course the global approval and commercialization of geo-enja that can be funded from those cash flows from rukenests and of course we were pleased very pleased to get that very fast approval from the FDA back in March brought the products to the market and got reimbursable reimbursement almost immediately when we are on the markets. Just as we go already you record revenues in the second quarter of this year which was the first quarter of geo-enja was on the market and now of course we are very proud of the continued growth of geo-enja where in this quarter we book six and a half million of revenues and year-to-date 10.3 millions.
Gross profit increased $2 58.
$4 million, an increase of $6 5 million.
Operating cost increase.
Increased from $44 seven to $56 eight.
The increase of $12 1 million is mainly because of the R&D and additional.
Speaker 6: transcript
Speaker 6: of 8 million and marketing and sales of 5 million. And that is all directed towards the launch, or most of it is directed towards launch of Geoenja and the third development.
Additional investments.
$8 million.
Marketing and sales of $5 million and that is all.
Directed towards the launch of most of it is directed towards the launch of <unk> and the further developments of the market.
Speaker 6: transcript
Speaker 6: The operating laws was, profit in this case is 1.9 million, and the net profit was 3.5 million in the quarter. And that was on the back of positive financing income and a tax credit, which is a timing effect.
The operating loss was.
Profit in this case is one nine.
Net profit was $3 5 million in the quarter.
And that was on the back of the positive financing income.
Tax credit, which you said.
Timing effects.
Speaker 6: transcript
Speaker 6: Simon mentioned it already, but the cash equivalence increased to $199 million at the end of the quarter.
Simon mentioned, it already but the cash and cash equivalents increased to $199 million at the end of the quarter.
Okay.
Yes.
Speaker 6: transcript
Speaker 6: Looking at the figures year to date, nine months year to date, the revenue increased by 9% to 164.1 million. Grouch profit increased to 146.
Looking at the figures year to date nine months year to date.
Revenue increased by 9% to 164 1 million.
Simon DeFrees: In addition to that of course the regulatory reviews are ongoing for geo-enja in Europe, Canada, Australia, Israel, you know and we have a pediatric clinical trial program ongoing as our label is currently 12 years and upwards. And of course, on the right hand side, you see further growth accelerators beyond Jeroen Giant APDS, first and foremost, and that is where we will come back to you before year end to update you on that.
Gross profit increased to 146 and <unk>.
Speaker 6: transcript
Speaker 6: And as was guided earlier this year, we further increased our OPEX as I said, into mainly marketing and sales and R&D, and the operating cost were 175.3 million IE in increase of 48.4 million versus the same period last year.
As well as <unk>.
Earlier this year, we saw the increase.
Opex.
As ive sets into mainly marketing and sales and R&D.
The operating costs were.
$175 3 million I E. An increase of $48 4 million versus the same period last year.
Simon DeFrees: We are in dialogue with the FDA about the second Lillian's indication and will provide you with more details to where she ends of the year and colleague Anurag Relan, the fourth by the more. And last but not least, as we have a very strong commercialization infrastructure in both the U.S, and in Europe, we are hunting as we speak for new inalyzing opportunities or acquisitions for additional products in rare diseases that we can actually continue to develop medical development and bring to the market and successfully commercialize.
Speaker 6: transcript
Speaker 6: Consequently, the operating loss was 6.5 million for the three quarter periods. And that loss was 7.4 million, which is an improvement from Q2 this year.
Consequentially the operating.
Loss was $6 5 million.
Forward.
Three quarter period, and then Thats loss was $7 4 million, which is an improvement from Q2 this year.
Speaker 6: transcript
Speaker 6: We got to the next slide.
Okay.
Yes.
So we got to the next slides.
Speaker 6: transcript
Speaker 6: We see the growth in revenue over the quarters. The third quarter revenue was 66.7 million, and that's say 23% increase from last year.
We see the growth in revenue over the over the quarters.
Simon DeFrees: So we are looking for products that have clinical group of concept. And if you see at the next slide, you see that there is actually space to have such products in our pipeline. You see here the extensive work we do by enlarging our footprint by means of bringing laniolisip to markets as far as Japan, but also Canada, Australia and Israel, and of course the other indication. So you see there is space in this pipeline to further accelerate the growth of the company going forward.
The third quarter revenue was $66.7 million, and let's say, 23% increase from last year.
Speaker 6: transcript
Speaker 6: driven by both Rukunest and obviously Joenji, as you can see on the picture, and also accelerated growth is seen in Rukunest. So you will remember that in Q1, there was a temporary reimbursement issue, it was a market circumstance.
Driven by both <unk>, and obviously, Joe and John as you can see on the picture.
And also accelerated growth is seen in <unk>. So you will remember that in Q1.
It was a temporary reimbursement issue was a market circumstance. So we had a dip in sales in <unk>, but we are very well recovered from that and we are now.
Speaker 7: transcript
Speaker 7: So we had a dip in seals in Rukunas, but 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.
Stephen Toor: I would like to now hand over to my colleagues Steven Tor, who will give you some more insights in the commercialization operations on the Rukinesse and do engines. Steven, over to you. Thank you so much, morning everybody. As Simon said, I will give you a brief overview of a Rukinesse performance and also some insights around the Jo and Jo watch and update you on that progress today. Next slide please. So I was communicated at the end of Q1 and as you are aware, the H8 market underwent a significant event which affected all products.
On a positive.
A positive note.
And that is in line with the guidance that we gave earlier this year.
Looking at the.
Speaker 7: transcript
Speaker 7: Cost development, as I said, investment in the Geoengeo launch and further development of of the learning ownership continues. And we have a quarterly
Yes.
Cost developments as I said investments in the <unk> launch and further development of linear ownership continues.
And we have a.
Speaker 7: transcript
Speaker 7: OPEX of the almost 57 million, 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 Annatmin cost relatively stable.
Quarterly.
Opex almost 57%.
$1 million and you'll see also that the increase is mainly as you would expect both in.
Stephen Toor: The event was short-lived and as we said we were, we bounced back strongly in Q2 and Q3. As you can see in first bullet we have posted strong growth in Q3 and even with the software for Q1, as Simon showed, we have grown versus prior year. So we are pleased with that performance. 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 have seen in the past. So Simon said we continue to guide for low single-digit growth for Rukinesse this year. Next slide please.
Research and development and <unk>.
Marketing exit in our marketing and sales and the general on that meant cost relatively stable.
Speaker 7: transcript
Speaker 7: The increase in R&D or the stable for now was maybe guided towards clinical operational and medical affairs. And if you look at the development over the quarters as we guided earlier here, is fairly stable, especially if you exclude the one-off milestone payment in the second quarter.
The increase in R&D or the stable for now.
Mainly.
<unk> guided towards clinical operational and medical medical SaaS and if you look at the development over the quarters as we guided earlier.
It's fairly stable, especially if you exclude the one off milestone payments in the second quarter.
Speaker 7: transcript
Speaker 7: I'm going to the outlook for this year. We remain on track for single-digit growth in Rikunas revenues.
And then going to the outlook for this year.
Stephen Toor: So as many of you know, Rukinesse was launched in 2015. We've actually been active in the H8 community since around 2000 and over those 23 years we've collaborated with all key stakeholders including the clinic ones and patient advocacy groups such as the H8E8 and that has been the major driver for the consistent success of Rukinesse over the nine years post launch and it's why the prescriber base continues to grow with 700 in the US today and also why we've retreated over 2000 patients and that metric continues to grow as well. So I think what that clearly underlines is the importance and the on-going need for a recombinant IV C1 esterase inhibitor and that's despite the fact over 70% of patients are now on prophylactic today.
We remain on track for single digit growth in recognized revenues.
Speaker 7: transcript
Speaker 7: And that's only been conferred by the numbers that we've shown in Q3. Joenja was improved in the first quarter and we've been commercializing in the US since early April as my colleague Steve, he alluded to.
And that's only been confirmed by the numbers that we showed in Q3 joined here was improved in the first quarter and we've been commercializing in the U S. Since early April as my colleague Steve.
Two.
Speaker 7: transcript
Speaker 7: The CHMP opinion is expected in the fourth quarter of this year and the marketing authorization subject to the positive outcome of the review is expected in Europe two months later. We will file a linear analysis with UK's MHRA following the ECPRP route and we will continue to invest in accelerated growth for the future in operating costs. The CHMP opinion is expected in the fourth quarter of this year and the marketing authorization subject to the positive outcome of the review.
The <unk> opinion is expected in the fourth quarter of this year and marketing authorization subject subject to positive outcome of the review.
<unk> is expected in Europe. Two months later, we will file and then the other ship Uk's.
Uk's MH NHRA following <unk> routes.
Stephen Toor: So next slide please. So moving to Jeroenja, as Simon showed earlier in the presentation, we're off to a very strong start in the US 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 RRX that specialises in ultra rare rare diseases to build a program that we thought would enable us to quickly provide patients with access to Jeroenja once they're covered for chronic use.
We will continue to invest in accelerated growth for the future.
Operating costs.
In the remainder of the year, we will.
Speaker 7: transcript
Speaker 7: detail further up plans to develop a linearism in additional indications. And as Simon mentioned, we keep looking for investments on in licensing and also acquisitions of assets to in-readies for future growth.
Detailed further our plans to develop <unk> in additional indications.
As Simon mentioned, we keep looking for investments.
In licensing and acquisitions.
<unk>.
Assets to <unk> for future growth.
Speaker 7: transcript
Speaker 7: With that, I want to start off kick off the Q&A. So any questions are welcome.
With that I want to.
Start I'll kick off the Q&A. So any questions are welcome. Thank you.
Stephen Toor: And I'm pleased to report that in only six months post-launch, Barman's access to medical teams in partnership with key opinion leaders across the country has secured APDS coverage policies in over 90% of our target plans across that's across commercial and government payers. And the result is a 93% approval rate with zero denials. And so I just want to repeat those two, 93% approval rate with zero denials despite the rarity of this condition and the heavy lift and education.
Speaker 2: transcript
Speaker 2: Thank you. To ask a question, you will need to press star one and one on your telephone and wait for your name to be announced. To withdraw your question, please press star one and one again.
Thank you to ask a question press star one on your telephone for your name to be announced soon.
Your question. Please press star one on one again.
We will now go to your first question.
Speaker 2: transcript
Speaker 2: And your first question comes to line of Christian Glenny from Stevele, please go ahead.
And your first question comes from the line of Christian Glennie from Stifel. Please go ahead.
Speaker 7: transcript
Speaker 7: Yep, good morning, good afternoon guys. Thanks for taking the questions. Three, please. I'll tell them in order.
Stephen Toor: So that's really a very strong performance. Also as you know, from enrollment to shipping to patients, the gold standard in rare diseases 30 days. I'm pleased to report to you that we're average in 26 days typically and sometimes are getting from enrollment to put in product in patients hand in less than 20 days. And really this is based on exceptional customer focus and execution which further instills I believe in our confidence in our stakeholders but most importantly the patients and treating positions.
Yes. Good morning, good afternoon, guys and thanks for taking the questions.
Speaker 7: transcript
Speaker 7: Let's start with Rookiness and a strong courtly spell print.
Three please I'll take them in order, let's start with <unk>.
Strong quarterly sales print.
Speaker 7: transcript
Speaker 7: Just to be clear around whether there's anything to be aware of in the Q3 numbers, maybe there's stocking or some impact that means it's...
To be clear around whether there's anything to be aware of.
Q3.
Numbers.
They are stocking or some impact that means it's it should be a clean quarter and then any implications full as we think about the fourth quarter as well typically your strongest quarter, particularly in the U S for reconnect any reasons why Q4 would.
Speaker 7: transcript
Speaker 7: It should be a clean quarter and then in implications for, as we think about the fourth quarter as well, typically your strongest quarter, particularly in the US, for a reconnest. Any reasons why Q4 wouldn't fill B your strongest quarter for the year.
Stephen Toor: So next slide please. So I've mentioned, you know, farming strong customer and patient focus and I think combined with the exceptional execution I've already mentioned, a US team of delivered strong results in that first six months since launch. We have as the slideshow 76 eligible patients 63 shipping and that represents well over half of the eligible patients we have on therapy. This is led to the revenues at 10.3 million that Simon also showed and as I already mentioned, pay discussions haven't continued to go very well which creates importantly an excellent environment for us to pool those patients through once they're diagnosed and enrolled.
Still be your strongest quarter for the year.
Speaker 3: transcript
Speaker 3: Thanks, Christiana, for the interesting question. Now, would you like to comment on that, Steve?
Thanks, Chris and it's an interesting question now would you like to comment on that Steve.
Speaker 4: transcript
Speaker 4: Yes, sure. Thanks, Christian. So you're right. Q3 is strong and we occasionally see stocking, but it's sporadic. There's no real pattern to it. So I was still expected this stage, Q4 to be our strongest quarter and for us to see out the year strong.
Yeah sure. Thanks Christian.
So you're right Q3 is strong and we occasionally see stocking, but it is sporadic there's no real pattern to it.
So I would still expect at this stage Q4 to be our strongest quarter in first to see how the year strong.
Speaker 7: transcript
Speaker 7: Thanks, and then maybe a quick follow up there. I mean, anything, I mean, you've called up the new patient's start. I mean, it's just curious why you, you know, you're still getting the...
Thanks, and then maybe a quick follow up but I mean anything.
Caught up the new patient starts I mean, it's just curious why you.
You're still getting this furniture.
Speaker 4: transcript
Speaker 4: strong patient starts and position uptake and everything else to comment on that. Yes, I think it's interesting we actually restructured our team a month before COVID hit and then we had to
Strong patient saw some physician uptake and then anything else to comment on that.
Stephen Toor: 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 on resources now family testing. Most of the patients we have our patient zero so to speak. So we believe there's a lot of opportunities there to help those families by better educating them and testing all of them to see whether there are others in need of therapy.
Yes.
He is interesting we actually restructured our team a month before Covid hit and then we have to most of them. So I think youll see a combination of different things happening, but certainly the restructure that team is now paying off and Thats why youll see actually an increase in enrollments and expect consistent increase in enrollments across three quarters. So I think that makes a big difference in using I would say is we have a very well tenured team.
Speaker 4: transcript
Speaker 4: So I think you'll see an accommodation of different things happen. But certainly the restructure of that team is now paying off. And that's why you're seeing actually an increase in enrollments and effective system increase in enrollments across three quarters. So I think that makes a big difference. And the other thing I'm saying is we have a very well-tenured team.
Speaker 4: transcript
Speaker 4: So deep relationships within our positions offices and with their staff.
Anurag Relan: And with that I'd like to hand over now to our CMO doctor Dr. Anaregrim. 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 or 1500 patients 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.
So deep relationships within our physicians offices and with the staff and I think that's really helps in identifying new patients I mean, as you would've heard us allude to we have a much broader mix of patients now that we have historically, whereas if it launches you would expect it was refractory patients who.
Speaker 4: transcript
Speaker 4: I think that's really helped in identifying new patients. I mean, as you would have heard us a little too, we have a much broader mix of patients now than we have historically. Whereas if it launches, you would expect, it was refractory patients who are...
Speaker 4: transcript
Speaker 4: predominantly using routes and SNL, it's across Marble, Modern, and SIVE and it's pretty evenly spread. So I think there's a combination of time in market, trust, execution, and as I mentioned earlier, continued need for IVC, when S2RAs inhibitors, despite the quite disruptive changes to the market.
Predominantly using <unk> now it's across mild moderate and severe it is pretty evenly spread. So I think it's a combination of time and market Trust execution.
And as I mentioned earlier continued need for IV see when esterase inhibitors, despite quite disruptive changes to the market.
Speaker 7: transcript
Speaker 7: Okay, thank you. And then turning to your ender then, if we can, just firstly on the European approval process, the advisory group committing a group meeting, as I understand it's still to be held, is there a timing on that or any outcome that comes from that advisory group?
Anurag Relan: 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. 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.
Okay. Thank you and then turning tissue and you then if we can.
Just firstly on the.
The European approval process, the advisor group commissioning.
Meeting is still to be how does is there a timing on that or any outcome that comes from that advisory group meeting.
Speaker 5: transcript
Speaker 5: Hi Christian, et cetera. So 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. Others end to say that we continue to expect the CHMP opinion in this quarter.
Hi, Christian etcetera.
We.
Anurag Relan: Fortunately, there is a genetic test that can provide a definitive diagnosis for APDS, and we spend 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, 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.
Meeting has been scheduled it is a closed meeting.
We're not going to provide any further guidance on the ongoing regulatory interactions other than to say that we continue to expect the CHF <unk> opinion in this quarter.
Speaker 7: transcript
Speaker 7: Okay, thank you. And then, as we think about, obviously the Q3 numbers around patient.
Okay.
And then.
And then as we think about obviously the Q3 numbers around patient.
Speaker 7: transcript
Speaker 7: Patients on pay therapy and things, I mean anything to flag in terms of expectations for Q4 versus the current run rate of enrolled patients and patients on therapy. And then particularly maybe there's any commentary you can insight and give on.
Patients will pay therapy, and things I mean anything to flag in terms of expectations for Q4.
Anurag Relan: We have randomized clinical trial data showing that Joenja met both primary endpoints, as well as meetings, several significant other clinical, clinically relevant endpoints. In addition, we have 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. And more importantly, we have long-term data, and I'll be sharing some of that with you that we've been publishing and presenting a conference recently about the long-term benefits of using Joenja over several years in many cases.
Is this sort of current run rate of enrolled patients and.
And patients on therapy, and then particularly may be this any any commentary insight you can give on.
Speaker 7: transcript
Speaker 7: that the numbers of patients obviously presumably large-pork proportional starters, but how many bridge packs are people having to give you simply implying you're getting patients on to pay therapy pretty quickly, but other bridges still something that's a reasonable factor in the matter.
The numbers of patients obviously.
Large pub proportional starters, but how many bridge.
So people having to Cape you seem to imply youre getting patients onto paid therapy pretty quickly.
The bridge is still something thats reasonable factor in the mix.
Speaker 4: transcript
Speaker 4: Have you got a comment on that? Certainly. So again, we're at the exact question. We continue to aggressively pursue our patient fighting efforts. And actually, we're ramping that up now through that early launch and conversions. In terms of starter and bridge, yeah, I mean, we've actually caused most of those patients have been on starter, but we're...
Maybe Steve you want to comment on that certainly so.
Again, it's not like say Christian we continue to aggressively pursue our patient finding efforts nicely. We're ramping that up now we're through that early launch and conversion strengths.
Anurag Relan: And this includes, for patients, in some cases, to discontinue the use of immune globular 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 called lymphopuloperation. So their lymph nodes continue to stay not enlarged, and we see benefits also in their immune cell function. And as Steve mentioned, this is 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.
To start on a bridge I mean, we've actually across most of those patients have been on starts.
Speaker 4: transcript
Speaker 4: We're having to bridge very fewer. When we do, we bridge for a pretty short period of time, relative to what we may have seen in with Rucheness back in the past. And that's because of this.
We happened to bridge very fuel when we do we prescribe.
Pretty short period of time relative to what we might have soon with <unk> back in the past and Thats because of this.
Speaker 8: transcript
Speaker 8: pretty fast approval rate, now ability to get commercial product in patients hands quickly. So yeah, it's going very well in that regard and we're not having to give away too much restock. Okay, great, thanks. I'll turn back in the queue.
Pretty fast approval rate and our ability to get commercial product in patient's hands quickly. So.
Yes.
It's going very well in that regard and we're not going to give away too much restocked.
Okay, great. Thanks, I'll jump back in the queue.
Thank you.
We will now go to our next question.
Speaker 2: transcript
Speaker 2: And the next question comes from the line of Alice Campbell from Royal Bank of Canada. Please go ahead.
Yes.
And the next question comes from the line of Alistair Campbell from Royal Bank of Canada. Please go ahead.
Anurag Relan: On the next slide, you can see some of our things that we're doing beyond the work that we've done in the US. As we discussed in August, we received the day 180 list of outstanding issues from the European Medicine 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 two 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 two months later.
Speaker 9: transcript
Speaker 9: Thanks so much, hopefully you can hear me. A couple of questions please. Another follow on Rukuness, which is a following on from Christians' questions to an extent. But you're obviously pointing towards adding prescribing physicians, and that's great.
Hi, Thanks, very much hope you can hear me.
Couple of questions. Please the follow on <unk>, which is a following on from Christian's question to an extent, but you're obviously pointing towards adding prescribing physicians and that's growing at around give or take 10% per annum, but also the products growing at low single digits. How should I think about that disconnect is that a price a factor or is that lower utilization per patient or is it just being.
Speaker 9: transcript
Speaker 9: products growing at low single digits. How should I think about that disconnect? Is that a price effect or is that lower utilization prepared?
<unk> prescribers, youre, writing or sort of less active so that's question one and then.
Speaker 9: transcript
Speaker 9: And then question two, if I could just sort of push my luck a bit on additional indications, looking at some of those publications you flagged on the presentation, I think you're one of the suggestions you could look at areas where MTOR and HIVAS are currently used, getting the same pathway, and that sort of brings to mind.
Question, two if I could just 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 that you could look at areas, where <unk> inhibitor, just trying to use the same pathway.
Anurag Relan: And as we previously announced, we've started a program in Japan to enable registration there eventually, and 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.
What it brings to mind areas like autoimmune with things like transplant rejection, but it's also oncology areas like neuro endocrine tumors and kind of.
You can see whether it's any of those areas are things youre thinking about right now thanks.
Speaker 4: transcript
Speaker 4: So maybe the first question you want to answer, Stephen? Certainly. So you're right to flag, I think the apparent or the perceived disconnect. For the most part, what I was
So maybe the first question you want to answer it Steven.
Certainly so.
You're right to flag I think the apparent.
<unk> disconnects.
For the most part.
Anurag Relan: And the 1 to 6 year old study has now started recruiting, so we expect that first patient also to treat it 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 4: transcript
Speaker 4: We went from 30% of patients on prophylactic therapy three, four years ago to 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. 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're seeing patients in that severe end or frequent attacks move into the moderate and from the moderate into the...
What I would say that is.
We went from 33% of patients on prophylactic therapy, three four years ago to over 70% now in the mid seventies.
Youll see us better control patients, having less attacks, therefore, utilizing less acute therapy, but we haven't actually lost that many patients. 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 type of attacks. So we've seen patients, whom that severe and frequent attacks and move into the moderate moderate.
Anurag Relan: 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 lanyoles that we can share. These data highlight the seriousness of APDS and I think it also highlights the experience that we have with lanyoles who have been treating many of these patients.
Speaker 4: transcript
Speaker 4: into the mild area. So it's more patients, more prescribing positions, but often a less severe course of disease, leading to slightly less acute utilization.
And to the into.
The moat area. So it's more patients move to prescribing physicians, but often less of a course of disease, leading to slightly less acute utilization.
Speaker 9: transcript
Speaker 9: Can I clicky-fall off on that? If that sort of makes change in the patient population.
Can I quickly follow up on that.
Is 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.
Speaker 9: transcript
Speaker 9: Let's say it's more or less played out. Does that mean that perhaps as more physicians are added, could that disconnect actually lessen over time? Logically, yeah.
Anurag Relan: On top of that, we have our ongoing Navigate APDS program that offers no cost testing available to patients in the US 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've really pushing on right now is that when we look at the diagnosed patients that we have in the US, 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.
Could that disconnect could actually lessen over time.
Logically, yes, yes, it could.
Okay. Thank you.
And then I think your question about our next indication.
Speaker 5: transcript
Speaker 5: I think you're thinking along the same lines that we are. For example, we know that in APBS, in the past especially, but even now in some areas where a joint is not available, M-Torn-Tibeters are used.
I think youre thinking along the same lines that we are for example, we know that.
In Aps.
In the past, especially but even now in some areas. We're joined just not available.
Speaker 5: transcript
<unk> inhibitors are used they have tolerability issues.
Not quite.
Perfect target.
The condition itself. So we are looking at other areas, where <unk> are used.
Anurag Relan: So we've initiated several efforts here, both with physicians and with family members themselves to be able to reduce the barriers to allow further testing amongst family members, which we think will be important to help uncover and find more patients.
Speaker 5: transcript
Speaker 5: proved to be a better suited for that disease.
Lineal Zip code.
To be a better.
Suited for that disease, we're not specifically I can already comment that we're not specifically looking within oncology.
Speaker 5: transcript
Speaker 5: We're not specifically, I can already comment, and we're not specifically looking within oncology. I think it's something that we may do in the future, but at the present time, we're really focused on other rare diseases where we think that this pathway is overactive.
I think it's something that we may do in the future, but at the present time, we're really focused on other rare diseases.
Anurag Relan: On the next slide, I want to review with you a little bit of information on something called variants of uncertain significance and what these are our genetic test results that are basically unclear or I would better say it's not the same. It's the 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 a, 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.
Where we think that this pathway is overactive.
Speaker 5: transcript
Speaker 5: that be some of the things that we see. Basically, we're trying to take the learnings from APDS.
Some of the things that we see basically trying to take the learnings from Etfs.
Speaker 5: transcript
Speaker 5: Some of the features of APBS that we see, where else do we see that type of...
Some of the features of Atvs that we see where do we where else do we see that type of.
Speaker 5: transcript
Speaker 5: problem in the immune system. 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. Great, thanks very much.
Problem in the immune system 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.
Great. Thanks very much.
Anurag Relan: And so we have several efforts ongoing and I'm not going to review all of them with you in details here but these efforts involve trying to review the existing data and try to collate all of that information and publish that. We just started a partnership, for example, with Genomenon to develop these genetic genomic landscapes, which will be available to all clinicians to be able to easily access variants 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 haven't, 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.
Thank you.
We will now go to the next question.
Speaker 2: transcript
Speaker 2: And your next question comes from the line of Cecilia Hernandez, Van Lanschock-Kempon, please go ahead.
And your next question comes from the line of Cielo Hernandez.
<unk> Kempen. Please go ahead.
Speaker 10: transcript
Speaker 10: And thank you for taking my question. Could you walk us through the development of your operating expenses? As you've mentioned, we've seen an increase in marketing and sales costs due to the launch. But will this increase further or is this level what we can expect for the common quarters? And also an R&D cost with the second indication for Leonid's Fisable to target profitability next year. Thank you.
Thank you for taking my question could you walk us through the development of your operating expenses that you mentioned have you seen an increase in marketing and sales costs due to the launch.
This increase.
This increase further our debt level.
Or is this level once against expect for the coming quarters and also in R&D costs with the second indication for Liana. This is it feasible to target profitability next year. Thank you.
Speaker 6: transcript
Speaker 6: Yeah, thank you very much for the question, Shushila. On OPEX for Duenja, we will support the European launch going forward. So we will shift probably some of the funds from the US to Europe .
Yes. Thank you very much for the question Sheila on Opex for.
For <unk>, we will see.
Anurag Relan: And I think the nice thing here in a sense is that this is a problem, it's a new problem for APDS 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.
Sure.
The European launch going forward. So we will we will shift probably some of the funds from the us to Europe.
Speaker 6: transcript
Speaker 6: on a net basis to say what the outcome of that shift will be.
On a net basis too early to say what the odds.
Come up that shift will be.
Speaker 1: transcript
Speaker 11: And for next year also on the new indication, yes, we will invest in R&D and in a trial. Again, there is to say what the exact cost is. But I don't expect necessarily a reduction in LPEX next year. Thank you.
And for <unk> for next year also on the on the new indication, Yes, we will we will invest in R&D.
Jeroen Wakkerman: on the next slide you can see so the 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 conditions for the mortality for example associated with APDS but also the health care costs associated with APDS and especially untreated APDS and these data I think highlight very nicely the the serious burden that these APDS patients face on top of that we continue to get more data out of our clinical trial program so we have a second interanalysis 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 program or compassionate use program where for example the second bullet under the IPIC heading is a patient who was previously trying to transplant it unsuppressively unfortunately but then was treated with Lenny Olisib under this compassionate use program and the data at the abstract will show the benefits of 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 the first interanalysis and that's available now in a full paper and then the next publication is also a key publication describing the mechanism of action of Lenny Olisib in APDS and I think it describes clearly how APDS leads to this primary immune deficiency with this immune dysregulatory phenotype and how Lenny Olisib benefits in these patients and with that I'll turn it over to my colleague Yerun Wacherman or Chief Financial Officer thank you very much Anirak focusing first on the financial highlights of the third quarter 2023 total revenues increased to 66.7 million so an increase of 12.5 million or 23 percent gross profit increased to 58.4 million an increase of 6.5 million operating cast increased from 44.7 to 56.8 and the increase of 12.1 million is mainly because of R&D initial additional investments of 8 million and marketing and sales of 5 million and that is all directed towards the launch of most of it is directed towards the launch of Geoengia and the third development of the market. The operating loss was a profit in this case is 1.9 million and the net profit was 3.5 million in the quarter and that was on the back of the positive financing income and a tax credit which is a timing effect.
And the trials.
Again, there is too early to say what the exact cost is but I don't expect necessarily a reduction in opex next year.
Okay. Thank you.
Thank you.
We will now go to the next question.
And your next question comes from the line of.
Speaker 2: transcript
Speaker 2: Joan Panitingenneth, from HC Way MI Please Please go ahead.
Panting goodness from H C. Wainwright. Please go ahead.
Speaker 11: transcript
Speaker 12: Hey guys, thanks for taking the question. Couple of you don't mind. So first, I wanted to focus on students comment on the HA market. Obviously there's a lot of...
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 HIV market. Obviously, there is a lot of disruptive changes to the market that are either ongoing or coming.
Speaker 11: transcript
Speaker 12: market that are either ongoing or coming. And I'll phrase my question this way. Obviously, we know where we stand and where farming stands with regard to the role of Rookinest. But I guess maybe more feedback from your new prescribers, for example, and even existing
And I'll phrase my question. This way, obviously, we know where we stand and where farming stands with regard to the role of <unk>, 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 <unk>.
Speaker 11: transcript
Speaker 12: threats that are coming even though they are focused on the prophylactic standpoint. So basically the external views of, you know,
You want to comment on that Stephen.
Speaker 4: transcript
Speaker 4: Sure, I mean, you know, Joe, we, uh, morning by the way, we, uh, we held out balls pretty regularly. We're all of us, uh, active in the, you know, myself and our regular regular in front of customers in the US.
Sure.
Joe Good morning by the way.
We hope that bodes pretty regularly where all of this activity in the U S. Open on a rag originally in front of the customers in the U S.
Speaker 4: transcript
Speaker 4: I think certainly some of these positions are quite excited by what may be coming in the future. I think what's interesting for me is...
And I think.
I think certainly some of these because some physicians are quite excited about what might be coming in the future I think what's interesting for me is.
Speaker 4: transcript
Speaker 4: Patients still need, despite all of these disruptions, that occasional bolus of...
Patients still need despite these disruptions.
Occasional bolus of.
Speaker 4: transcript
Speaker 4: therapy that you get from an IV product such as ruchines. So although we see disruption, and as I mentioned earlier, we see sometimes a decrease or in some places, even a normalcy, want an increase in utilization, we still see the need.
Therapy that you get from an IV products, such as root goodness, so, although we see disruption and as I mentioned earlier.
We see sometimes a decrease or in some places even a normalcy when it's an increase in utilization, we still see the need to so I certainly wouldn't want to be complacent and we look at the future as closely as any company would.
Speaker 4: transcript
Speaker 4: 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 disruption, certainly since I joined finally seven years ago.
But through all of the disruption certainly since I joined <unk> seven years ago.
Speaker 4: transcript
Speaker 4: We see that initial period of disruption and fluctuation in volume, and then we see things settled down, and we see a continued clinical need for, I know, VAC, myastrasin, give it a go.
The initial period of disruption and fluctuation in volume and then we see things settle down and we see a continued clinical need for <unk> inhibitor. So.
Speaker 11: transcript
Speaker 12: Does that also question Joe? No, no, it certainly does. I appreciate that. And I guess, you know, looking more towards just switching a little bit to...
Does that answer your question no. It certainly does I appreciate that and I guess looking more towards.
Just switching a little bit too Joe and Joe.
Speaker 11: transcript
Speaker 12: assuming a positive CHMP opinion later this quarter, maybe if you could provide a little more color with regard to your country by country strategy, I know.
Assuming a positive <unk> opinion later this quarter, maybe if you could provide a little more color with regard to your country by country strategy. I know previously you discussed targeting Germany first but how should we view beyond that.
Speaker 11: transcript
Speaker 12: August , you know, Marching, Germany first, but how should we view...
How things should go.
Speaker 12: transcript
Speaker 13: Do you want me to take a song? No, no, that's being... Come on.
Do you want to take this one.
Speaker 4: transcript
Speaker 4: You know, so thanks, thanks, you know, 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 foreign Europe . Our ag mentioned the UK and the submission there. Now specifically there, now it's not part of the EU. And then we'll get through Spain, Italy, France as major nations, but we won't ignore the rest of Europe either. So we have a pretty lean operation in Europe and we cluster the other.
Thanks, Joe.
Youre 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 four in Europe I already mentioned the UK in a submission that specifically that's not part of the year.
Jeroen Wakkerman: Simon mentioned it already but the cash and cash equivalence increased to 199 million at the end of the quarter. Looking at the figures year-to-date, nine-month year-to-date, the revenue increased by 9% to 164.1 million, Gross Profit increased to 146, and as was guided earlier this year, we further increased our OPEX as I said, into mainly marketing and sales and R&D, and the operating cost were 175.3 million, IE, and increased of 48.4 million versus the same period last year. Consequently, the operating loss was 6.5 million for the three quarter periods, and then that loss was 7.4 million, which is an improvement from due to this year.
And then we'll go through Spain, Italy, France, as major nations, but we won't ignore the rest of Europe, either so we have a pretty lean operation in Europe, and we cluster.
Speaker 4: transcript
Speaker 4: 22 member states around those major markets and you'll see us through 24 and 25
22 member states around those major markets and you will see us through 'twenty four 'twenty five.
Speaker 4: transcript
Speaker 4: you know, in a state sequence, start to go to all those markets. And by the way, we've identified patients, I think in every single one of those markets.
In a steady sequences start to go through all of 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 a base of operations in APAC in the key markets that saw the Japan Youll go in trial in Japan, and a submission with health, Canada right now.
Speaker 4: transcript
Speaker 4: In addition to that, we have submissions in Australia, which will enable us to set up a base of operations in APAC in the key markets outside of Japan. Yongo in China and Japan, and a submission with Health Canada right now as well as that, I believe, each rank market globally. So I think we have a pretty careful, well-considered sequenced approach that gets us into markets at the right time. And you'll see that pointed out through 24 and 25.
As well was that I believe.
Lease rental market globally. So I think we have a pretty carefully well considered sequenced approach that gets us into markets at the right time, and youll see that putting up through 'twenty four 'twenty five.
Speaker 11: transcript
Speaker 12: 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 regular run-trior in the label?
Got it and then just lastly, I guess, it's a quick logistical question.
With regard to the U S. As an unclassified variance if you will is.
Is there anything that needs to be done as you generate data about these variants on the regulatory front or in the label.
Jeroen Wakkerman: If we go to the next slide, we see the growth in revenue over the quarters. The third quarter revenue was 66.7 million, and that's a 23% increase from last year, driven by both Rukunest and obviously Joenji, as you can see on the picture. And also, accelerated growth is seen in Rukunest, so you will remember that in Q1, there was a temporary reimbursement issue, was a market circumstance, so we had a dip in sales in Rukunest, but 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.
To identify these.
Speaker 5: transcript
Speaker 5: Hey Joe, good morning. So the answer there is no. Because these patients actually have APDS and the label is for APDS. Right now the question is variant which hasn't been previously described, the 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 US.
Hey, Joe Good morning, So the answer there is no because these patients actually have hdds in the label is.
That.
As for Aps right now the question is this variant which hasnt been previously described what prevents 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 S and many many results come back into this into the U S classification, but.
Speaker 5: transcript
Speaker 5: 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 is reclassified base, usually on functional testing data or even as I mentioned that using that multiplex approach. Once that's reclassified then that patient has atvs and would qualify for treatment for the label.
Speaker 5: transcript
Speaker 5: Once that's reclassified, then that patient has ADDS and would qualify for treatment for the label. Great, thanks.
Great. Thanks for the clarification and thanks for all the answers guys.
Speaker 2: transcript
Speaker 2: Thank you. We will now go to your next question.
Thanks.
Thank you.
We will now go to your next question.
Speaker 2: transcript
Speaker 2: And your next question comes from the line of
Jeroen Wakkerman: Looking at the cost development, as I said, investment in the Joenji launch, and further development of Lonealyship Confuse, and we have a quarterly OPEX of the almost 57 million, 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 enactment costs are relatively stable. The increase in R&D, or the stable for now, was maybe guided towards clinical operational and medical affairs, and if you look at the development over the quarters, as we guided earlier this is fairly stable, especially if you exclude the one-off milestone payment in the second quarter.
And your next question comes from the line of.
Speaker 2: transcript
Speaker 2: Heartage thing from Oppenheimer, please go ahead.
Hi, Todd.
<unk> from Oppenheimer. Please go ahead.
Speaker 13: transcript
Speaker 14: Great, thank you. Thanks for the, I got a couple of questions and really nice update everyone.
Great. Thank you. Thanks for the I got a couple of questions and really nice.
Update everyone.
Speaker 13: transcript
Speaker 14: The two questions I have just falling up to a previous question on the cadence of the launches XUS.
Two question, Todd just falling up to a previous question on the cadence of the launches.
Speaker 13: transcript
Speaker 14: I know, you know, Australia, Canada, Israel, and in 24 and 25, you know, Europe , later this year, and then discussions there.
I know.
Australia, Canada, Israel, and 24% and 25 Europe.
Later this year and then discussions there can you just give us an idea of the relative Tam that market.
Speaker 13: transcript
Speaker 14: Can you just give us an idea of, you know, the relative time of that market? And then, you know, 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, you know, ready to get on drug. And then, you know, how would pricing, you know, potentially look relative to the US market? So that's the first question.
And then could there be bolus is 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 hardwood pricing potentially look relative to the U S market. So that's the first question.
Speaker 13: transcript
Speaker 14: The second question is just on your family testing, you know, previous research we had done before a lenulist 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, your 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. You know, what would you expect to see as you wrap up this family test? Thanks for the question.
The second question is just on your family testing.
Jeroen Wakkerman: Going to the outlook for this year, we remain on track for single-digit growth in Rukunest revenues, and that's only being conferred by the numbers that we've shown in Q3. Jeroen Jeroen was improved in the first quarter and we've been commercialising in the US since early April as my colleague Steve alluded to. The CHMP opinion is expected in the fourth quarter of this year and the marketing authorization, subject to the positive outcome of the review, is expected in Europe two months later.
Research, we had done before <unk> was approved in.
Vacates there could be as little as one more family member and that might just three more extended family members.
That might have some.
Indication of Atvs and could potentially qualify for treatment I don't think its early days, but if you could just give us some some color around there what do you expect to see as you as you wrap up this topic.
Speaker 4: transcript
Speaker 4: Thanks, I'm Kai. Hey Steve, you want to comment on those special numbers at XCUS? And certainly, and I didn't quite catch the front into the questions at all. So if I miss anything then please just pick me up as we go through. So I think you're right to flag that.
Thanks for the question.
Thanks, Steve.
Steve you want to comment on that specialty numbers ex U.
Yes.
Certainly and I didn't quite catch the front end of the question to Tom.
If I Miss anything please just picking up as we go through so I think you're right to flag that.
Jeroen Wakkerman: We will file Lenny Alistair with UK's MHRA following the ECPRP route and we will continue to invest in accelerated growth for the future in operating costs. In the remainder of the year we will detail further our plans to develop Lenny Alistair in additional indications and, as Simon mentioned, we keep looking for investments on in licensing and also acquisitions of assets to in-rear disease for future growth.
Speaker 4: transcript
Speaker 4: You know, the medical affairs group across the world have been actively identifying patients with ear pinion leaders and those key centers in each country. So there will be a bolus of patients waiting therapy and many of them will already be in the early access program, for example.
Sure.
The the medical Affairs group across the World have been actively identifying patients with key opinion leaders in those key centers in each country. So there will be a bolus of patients waiting therapy and many of them would already be in the early access program. For example that will be a slight delay because from approval in many of those countries. You then need to negotiate.
Speaker 4: transcript
Speaker 4: 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 US will, well, as you know, for the most part, always be lower. It's highly unlikely that it will match that price.
Reimbursement, which means we we set the price slightly later than the than the clinical approval.
But in terms of your pricing question.
Price outside of the U S.
As you know for the most part always below it.
Speaker 4: transcript
Speaker 4: 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
You might be able to match that price.
And there'll be a variation in what that price it looks like country by country.
Operator: With that I want to start off pick off the Q&A so any questions are welcome. Thank you. To ask a question you will need to press star one and one on your telephone and wait for your name to be announced. To withdraw your question please press star one and one again.
I'd say, though without without preempting, what that price might look like.
Speaker 4: transcript
Speaker 4: 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.
As with all ultra rare diseases are 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 13: transcript
Speaker 14: 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.
Thanks, Steve 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.
Christian Glennie: We will now go to your first question and your first question comes the line of Christian Lenny from Steveal, please go ahead. Good morning. Good afternoon guys. Thanks for taking the questions. Three please, I'll tell you in the order.
Speaker 5: transcript
Speaker 5: Yes, our target is a great question. It's certainly something that we're trying to address right now, which is, you know, we know it's an autosomal-e-dollerant transmitted disease. So, you know, we expect that there should be other family members with the condition. And as we said, many times, it may not be immediate family member, even extended family member.
Yes.
Great question, and it's certainly something that we're trying to address right now which is.
We know, it's an autosomal dominant or 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.
Stephen Toor: Let's start with Rukines and a strong court please fail print. Just to be clear, whether there's anything to be aware of in the Q3 numbers, maybe there's stocking or some impact that means it should be a clean quarter and then in implications for as we think about the fourth quarter as well, typically your strongest quarter, particularly in the US for Rukines, any reasons why Q4 wouldn't, wouldn't still be your strongest quarter for the year.
Stephen Toor: Thanks Christian. That's an interesting question now. Would you like to comment on that, Steve? Yes, sure. Thanks Christian. So you're right. Q3 is strong and we occasionally see stocking but it's sporadic. There's no real pattern to it. So I was still expected this Q4 to be our strongest quarter and for us to see how the year's strong.
Speaker 5: transcript
Speaker 5: What we've been trying, what we've been a little surprised by, though, is, and then I guess it relates to the fragmented nature of our healthcare system, is that oftentimes these family numbers haven't been tested. Some of that is just due to lack of awareness, some 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.
What we've been trying to.
I'm a little surprised by though is and then I guess it relates to the fragmented nature of the of our health care system is there.
Oftentimes these family members haven't been tested.
Some of that is just due to lack of awareness amongst patients about the genetics of the disease and some of that is just due to the health care system idiosyncrasies, and how it's difficult to get genetic testing got so we're changing the <unk>.
Speaker 5: transcript
Speaker 5: So we were changing the way we approach this and really moving, putting the patient right at the center of this.
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 so if a patient.
Speaker 5: transcript
Speaker 5: so allowing patients and families to actually initiate tests.
Speaker 5: transcript
Speaker 5: So if a patient is diagnosed with APDS and a family member wants to get tested, we've initiated a program that will allow that to happen.
Is diagnosed with Atvs and a family member wants to get tested. We've initially we started a program that will allow that to happen by having the family never themselves initiate the process and it doesn't need to go through the for example, the patient specialists, who may not be immediately available to see the family member for example, or it may not even be a patient on the table.
Stephen Toor: Thanks and then maybe a quick follow up there. Anything 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 position uptake and things. Anything else to comment on that? Yes, I think it's interesting. We actually restructured our team a month before COVID hit and then we had to multiple them. So I think you'll see in 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 an effective system increase in enrollments across three quarters.
Speaker 5: transcript
Speaker 5: by having the family member themselves initiate the process and it doesn't need to go through the, 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.
Remember in most cases, but I think we are.
Speaker 5: transcript
Speaker 5: removing these barriers to genetic testing to allow appropriate testing in family. It is, I think, will likely be a significant.
Moving these barriers to genetic testing to allow appropriate testing in family is I think a will likely be a significant.
Speaker 5: transcript
Speaker 5: 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.
Source of newly diagnosed patients.
Stephen Toor: So I think that makes a big difference and the other thing I would say is we have a very well tenured team. So deep relationships within our positions offices and with their staff and I think that's really helped in identifying new patients. I mean as you would have heard us 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 are predominantly using root and S. Now it's across Marble, 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, a continued need for IVC want us to raise inhibitors despite the quite disruptive changes to them after. Thank you.
We're starting that program.
We've started a little bit of that already but really putting that in full force now.
Operator: Okay, thank you.
Speaker 13: transcript
Speaker 14: Great honor, that is that that that helps. Thank you everyone for the questions. Thank you.
Great that is.
That helps thank you everyone for the questions.
Thank you.
I will now go to the next question.
Speaker 2: transcript
And your next question comes from the line of Simon <unk> from Berlin. Please go ahead.
Speaker 7: transcript
Speaker 7: Yes, hello, thanks for taking my question. So you've already identified 158 PDS patients over the age of 12 in the US.
Yes, Hello, Thanks for taking my question.
So you've already identified 150, <unk> patient safety the age of 12 in the U S.
Speaker 14: transcript
Speaker 15: I was just wondering how many of those patients do ultimately expect to be able to enroll?
I was just wondering how many of those.
Patients do you ultimately expect to be able to enroll.
Christian Glennie: And then turning to Joensha then, if we can, just firstly on the European approval process, the advisory group committee group meeting, as I understand still to be held, is there a timing on that or any outcome that comes from that advisory group meeting? Hi, Christian, et cetera. So we, this meeting has been scheduled. It is a close meeting and we're not going to provide any further guidance on the ongoing regulatory interaction others than to say that we continue to expect the CHMP opinion in this quarter.
Speaker 3: transcript
I would say.
The vast majority of those assignment with almost all of them.
Speaker 5: transcript
Speaker 5: Okay, so almost all. And that's just the beginning, right? Because you heard. Yes, I know there are other patients, but I was just interested in patients who already identified. So we should assume over 90% or 95%. I think there will be very high percentage of patients that indeed will be interested to get into the Dementia treatment, correct? That's our experience so far.
Okay, so almost all of it.
And that's just the beginning right because you heard yes, I know there are other patients, but im I was just interested in.
In patients who are already identified so we should see may even 90% or 95% I think there will be a very high percentage of patients that indeed will be interested to get into that.
Regulatory Thats correct Thats our experience so far.
Speaker 14: transcript
Speaker 15: In particular in the US, you don't expect to encounter a problem with the last 10 or 20% because of lack of insurance coverage. How does that work? There will be some patients without insurance coverage. Can you get those as paid therapy as well?
Okay, and particularly 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.
Stephen Toor: Okay, and then, and then as we think about, obviously the Q3 numbers around patient, patients on pay therapy and things, I mean anything to flag in terms of expectations for Q4 versus the sort of current run rate of, of enrolled patients and patients on therapy. And then particularly, maybe there's, you know, any commentary you can insight and give on the numbers of patients, obviously, presumably a large portion on starters, but how many bridge packs are people having to give you simply, implying you're getting patients on to pay therapy pretty quickly, but other bridges still, something that's a reasonable factor in the mix.
I mean, how does that work I mean, presumably it will be some patients without insurance coverage.
Can you get that was just paid therapy on paid therapy as well.
Speaker 3: transcript
Speaker 3: Although those questions are difficult to answer, but generally speaking, if we look at our experiences, we don't see any issues with rates of dots coming up. And of course, we can also ask patients that have limited or non-insurance coverage, as always, in ways of means. Maybe you want to comment on that, Steven.
So those pressures are difficult to answer but generally speaking if we look at our experiences with <unk>.
We don't see any issues with their agency that's.
Coming up and of course <unk> also has patients that.
Limiters or no insurance coverage is always a base. It means maybe you want to comment on that Stephen.
Speaker 4: transcript
Speaker 4: Yeah, I think there's, I mean, there's all kinds of different.
I think there is.
I mean, there's all kinds of different.
Speaker 4: transcript
Speaker 4: types of support available for patients, even and also public.
Tied to support available for patients and also public.
Speaker 4: transcript
Speaker 4: programs that try to make sure that patients have access to something. So, if you notice, I'm like, I haven't looked at this for a while, but I believe we have very, very few ruchinist patients, for example, in our patient assistance program, which will be essentially free supply, on an ongoing basis.
Programs.
I want to make sure that patients have access to something so.
Stephen Toor: Have you, you want to comment on that? Certainly. So, as I said, Christian, we continue to, to aggressively pursue our patient finding efforts. And actually, we're ramping that up now, we're through that early launch and conversions. In terms of starter and bridge, yeah, I mean, we've actually, of course, most of those patients have been on starter, but we're, we're having to bridge very fewer. When we do, we bridge for a, for a pretty short period of time, relative to what we may have seen, and we've reconnest back in the past.
So youre always sunlight.
Due to this for a while but I believe we have very very few requests patients for example.
Patient assistance program, which would be essentially free supply on an ongoing basis.
Speaker 4: transcript
Speaker 4: And even when they are in there every year, we're working with them and with their physicians 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 Rookanest experience and my experience in red disease is outside of farming, I think the vast majority of patients in the end will be on paid therapy. It can sometimes just be a heavy lift in those last few percent of it together. Okay.
Every year, we are working with them and with their physicians to find that insurance plan for them, we'll find an auction that will be to pay therapy. So I apologize I can't give you a specific answer but if I look at the weakness experienced in my experience in rare diseases outside of farming I think the vast majority of patients in the yen.
Stephen Toor: And that's because of this pretty fast approval rate, and our ability to get commercial product in patients hands quickly. So, yeah, it's going very well in that regard, and we're not having to give away too much restock. Okay. Great. Thanks. I'll turn back in the queue. Thank you.
We will get will be on paid therapy. It can sometimes be a heavy lift in those last few percent together.
Okay. Thanks, very much that's very helpful.
Speaker 2: transcript
Speaker 2: Thank you. As a reminder, if you would like to ask a question, please press star one and one on your telephone and wait for your name to be announced. Oh no.
Thank you.
As a reminder, if you would like to ask a question. Please press star one on your telephone.
Alisa Campbell: We'll now go to our next question. And the next question comes from the line of Alisa Campbell from Royal Bank of Canada. Please go ahead. Thanks very much. Hopefully you can hear me. A couple of questions, please. Another follow-on on Ruchen S, which is a following-on from Christian's question to an extent, but you know, you're obviously pointing towards adding prescribing physicians, and that's growing at a balance. You've give a take 10% per annum, but also the product's growing at low single digits.
Your name to be announced.
Okay.
We'll now go to the next question.
Speaker 2: transcript
Speaker 2: And your next question comes from the line of Christian Glenny from Stevele Please Go ahead.
And your next question comes from the line of Christian Glennie from Stifel. Please go ahead.
Speaker 7: transcript
Speaker 7: I thought just quick follow up if I can on Joenger and the Pediatrics study in the 4 to 11 year olds looking at the raw material almost complete there. So just a reminder in terms of the endpoint for that trial, the timing of that endpoint and therefore when we might see data. And then the expected sort of rough mix of
Hi, guys just a quick follow up if I can on Gilenya and the pediatric study in the four 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 nine point and therefore, when we might see data and then do you expect to sort of rough mix of.
Alisa Campbell: How should I think about that disconnect? Is that a price effect, or is that lower utilization for patients, or is it just the incremental sort of prescribers you're adding, or sort of less active? So, that's question one. And then question two, if I could just sort of push my luck a bit on additional indications, looking at some of those publications you've flagged on the presentation, I think you're one of the suggestions you use is you could look at areas where M-Taurin is currently used, getting the same pathway.
Speaker 7: transcript
Speaker 7: The under 12s has it relates to this fall to 11 year olds. I mean, you know, if you think about say the 50 remaining patients identified in the US that we know how many of those would be...
The under 12, so as it relates to this full to 11 year olds.
If you think about say the 50 remaining patients identified in the U S that we know how.
How many of those would be four to 11.
Speaker 5: transcript
Speaker 5: So let me answer that question, the second question first, Christian. So we again, we based on our current experience in APDS about a quarter of patients overall are below the age of 12.
So let me answer that question. The second question first Christian.
Alisa Campbell: And that sort of brings to mind areas like all to the moon with things like transplant rejection, but it's also on collage areas like new or endocrine tumors. Can I press you to see whether any of those areas of things you're thinking about right now? Thanks.
C.
Again based on our current experience in Atvs about a quarter of patients overall are below the age of 12 among.
Stephen Toor: So, maybe the first question you want to answer, Stephen? 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 percent of patients on prophylactic therapy three, four years ago to over 70 percent 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 5: transcript
Speaker 5: Amongst that quarter, three quarters are in the age range.
Amongst that quarter three quarters are in the age range at least three quarters are in the age range of the.
Speaker 5: transcript
Speaker 5: At least three quarters are in the age range of the first study or four to 11 year old study.
The first study or four to 11 year old studies so.
Speaker 5: transcript
Speaker 5: 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 the older age groups, especially in that above four age groups.
We know these patients actually have the disease at birth and many patients do begin to manifest symptoms early on.
Oftentimes they are not diagnosed at that very early age and that results in many patients in this in the older age groups, especially in that above four age group.
Stephen Toor: 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 of attacks. So, we've seen patients in that severe end or frequent attacks move into the moderate and from the moderate into the mild area. So, it's more patients, more prescribing positions, but often a less severe course of disease leading to slightly less acute Can I quickly follow up on that?
Speaker 5: transcript
Speaker 5: So that's a little bit on the breakdown of the age distributions across HPS. In terms of the pediatric study, end point that we've had in the adolescents and adult study. So I think what we'll be able to do is once with the study is fully enrolled and we've got these last four patients enrolled, we'll give you some more guidance on the timing of
That's a little bit on the breakdown of the age distributions.
Across eight yes.
In terms of the pediatric study endpoint.
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.
Stephen Toor: If that sort of makes change in the patient population, let's say it's more or less played out, does that mean that perhaps as more physicians are added, could that disconnect could actually lessen over time? Logically, yes, it could. 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 joins is not available, mTOR inhibitors are used, they have tolerability issues, and they're not quite the perfect target for the condition it tells.
These last four patients enrolled will give you some more guidance on the timing of.
Speaker 5: transcript
Speaker 5: data releases as well as some of the regulatory work that we anticipate being able to do as the results are possible.
The data releases as well as some of the regulatory work that we anticipate being able to do if the results are positive.
Okay. Thanks Thats helpful.
Thank you.
Speaker 2: transcript
Speaker 2: There are currently no further questions. I will hand the call back.
There are currently no further questions I will hand, the call back.
Speaker 3: transcript
Speaker 3: All right, thank you very much. A few closing remarks. Thanks for attending. You can see now that as...
Alright, Thank you very much.
No closing remarks, thanks for attending you can see now that.
Speaker 3: transcript
Speaker 3: stated last quarter and the company is now starting a long growth trajectory supported by the foundation through CNES provides
As stated.
Last quarter.
Companies now starting a long growth trajectory.
Supported by the foundation of <unk> provides.
Stephen Toor: So we are looking at other areas where mTOR inhibitors are used, and where lenialists could prove to be a better suited for that disease. 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, we're really focused on other rare diseases where we think that this path way is overactive. That the, you know, 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 immune system.
Speaker 3: transcript
Speaker 3: And of course, driven by the future expansion of the Dio Enja, outside of the United States, but also inside of the United States.
And of course, driven by the future expansion of.
Joanne <unk> outside of the United States, but also inside of the United States.
Speaker 3: transcript
Speaker 3: supported by lots of efforts that we are undertaking and initiating to actually broaden the patient's base. And of course, as always, with new genetic diseases, the definition of the disease will broaden.
Supported by lots of efforts.
Our undertaking and initiating to actually broaden the patient base.
Of course, as always with new immune just with new genetic diseases.
The definition of the disease.
Speaker 3: transcript
Speaker 3: 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, putting us again as hopefully...
<unk> broader so therefore, we look forward to the future.
Optimism and.
With a company that will significantly grow and change over there over the coming years, putting us again.
Stephen Toor: 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. Great, thanks very much.
Combination of our commercialization capabilities clinical development and regulatory.
Skills, putting goes again as hopefully the.
Speaker 3: transcript
Speaker 3: the ideal partner or go to partner in the future for other rare disease assets that we can take on board and actually complete clinical development slash approvals and do the successful commercialization as we do with Rukunis and with Joanne.
Operator: Thank you.
The ideal partner to go to partner in the future for other rare disease assets that we can take on board.
Cecilia Hernandez: We will now go to the next question. And your next question comes from the line of Cecilia Hernandez. Von Lanshot campaign, please go ahead. Yes, thank you for taking my question. Could you walk us through the developments of your operating expenses, as you've mentioned, we've seen an increase in marketing and sales cost due to the lunch. But will this increase, will this increase further level, or is this level what we can expect for the common quarters, and also an R&D cost with the second indication for Leon.
And actually complete the clinical development slash approvals and do their successful commercialization as we do with <unk> and <unk>.
Speaker 3: transcript
Speaker 3: 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.
Georgia.
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 outside next year. Thank you very much goodbye.
Speaker 2: transcript
Speaker 2: Thank you, this concludes today's conference call. Thank you for participating. You may now disconnect.
Thank you. This concludes today's conference call. Thank you for participating you may now disconnect.
Cecilia Hernandez: Is it feasible to target profitability next year? Thank you. Yeah, thank you very much for the question. Thank you, Cecilia. On OPEX for due India, we will support the European launch going forward. So we will shift probably some of the funds from the US to two Europe on a net basis to say what the outcome of that shift will be. And for, yeah, for next year also on the new indication, yes, we will invest in R&D and in a trial. Again, there is truly to say what the exact cost is, but I don't expect necessarily a reduction in OPEX next year. Okay, thank you. Thank you.
Operator: We will now go to the next question.
Okay.
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Okay.
Okay.
Yes.
Okay.
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Yes.
Thank you.
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Sure.
[music].
Jay Pantginis: And your next question comes from the line of. Jay Pantginis from HAC Waylight. Please go ahead. Hey guys, thanks for taking the question. A couple of you don't mind. So first, I wanted to focus on Steven's comment on the HA market. Obviously, there's a lot of disruptive changes to the market that are either ongoing or coming. And I'll phrase my question this way. Obviously, we know where we stand and where farming stands with regard to the role of Rookanest.
Jay Pantginis: But I guess, you know, maybe more feedback from, you know, 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, you know, needing a rescue therapy such as Rookanest. You want to comment on that, Steven? Sure. I mean, as you know, Joe, we, uh, morning, by the way, we, we, uh, we held our balls pretty regularly.
So.
And.
Jay Pantginis: We're all of us active in the, you know, myself and Anna Raga regularly in front of customers in the US. And I think, um, I think certainly some of these physicians are, are quite excited by what may be coming in the future. I think what's interesting for me is patients still need, despite all these disruptions, uh, that occasional bolus of, uh, of, of therapy that you get from an IV product such as Rookanest.
Jay Pantginis: So although we see disruption, and as I mentioned earlier, uh, we see sometimes a decrease or, in some patients, even a normalcy, want an increase in, in utilization, we still see the need there. So I certainly wouldn't want to be complacent. And we'll look at the future as closely as, as any company would. Uh, but through all of the disruptions, certainly since, uh, I joined finally seven years ago, we see that initial period of disruption and, and fluctuation in volume.
Jay Pantginis: And then we see things settled down and we see a continued clinical need for, I know, VHC mice raising a bit of it. So, does that answer your question, Joe? No, no, it certainly does. I appreciate that.
Stephen Toor: And I, and I guess, you know, looking more towards, um, just, just switching a little bit to, uh, Joenja, um, assuming a positive CHMP opinion, uh, later this quarter, um, maybe if you could provide a little more color with regard to your country by country strategy, I know previously you discussed, um, you know, marketing Germany first, but how should we view, uh, beyond that, the, uh, you know, how things should go? Uh, do you want me to take that time?
Stephen Toor: No, no, that's good. Go ahead. You know, so thanks, Joe. The, um, you're, you're right. I mean, Germany would be the typical market to go to right out of the gate and, and we'll certainly do that. We also will then target the, the other big four in Europe. Our Ag mentioned the UK and the submission there. Now specifically there, now it's not part of the EU. Uh, and then we'll get to Spain, Italy, France as, as major nations, but we won't ignore the rest of Europe either.
Stephen Toor: So we have a pretty lean operation in Europe and, and we cluster the other 22 member states around those major markets and you'll see us through 24 and 25, you know, in a state sequence, start to, to go through all those markets. And by the way, we've identified patients, I think in every single one of those markets. In an addition to that, we have, um, submissions in Australia, which will enable us to set up a, a base of operations, an APAC in the key markets outside of Japan.
Stephen Toor: You all go and try it in Japan and a submission with health Canada right now as well as that, I believe, a, a front market globally. So I think we have a pretty careful, well considered sequenced approach that gets us into markets at the right time. And you'll see that pointed out through 24 and 25. Got it.
Anurag Relan: 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 or in the label to identify these? Hey Joe, good morning. So the answer there is no, because these patients actually have a DDS and the label is that is for APDS. Right now, the question is this variant, which hasn't been previously described, 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 VUSs.
Anurag Relan: 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 a DDS and would qualify for treatment for the label. Great. Thanks for the clarification and thanks for all the answers guys. Thank you.
Hartaj Singh: We will now get to your next question. And your next question comes from the line of hard task thing from Oppenheimer. Please go ahead. Great. Thank you. Thanks for the, I got a couple of questions and really nice update everyone. The two questions I have just falling up to a previous question on the cadence with the launches XUS. I know, you know, Australia Canada, Israel and in 24 and 25, you know, Europe.
Hartaj Singh: Later this year and then discussions there, can you just give us an idea of, you know, the relative time of that market and then, you know, 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, you know, ready to get on drug and then, you know, how would pricing, you know, potentially look relative to the US market.
Hartaj Singh: So that's the first question. The second question is just on your family testing, you know, previous research we had done before, when you listen 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. And your indication of APDS and could potentially qualify for treatment, I know these are early days, but if you could just give us some some color around there, you know, what would you expect to see as you, as you wrap up this.
Hartaj Singh: Thanks for the question. Thanks. Steve, you want to comment on those patient numbers at XNUS? Certainly, and I didn't quite catch the front end of the questions at all. So if I miss anything, then please just pick me up as we go through. So I think you're right to flag that, you know, the medical affairs group across the world have been actively identifying patients with here, opinion leaders in those key centers in each country.
Hartaj Singh: So there will be a bolus of patients waiting therapy and 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 we set the price slightly later than the clinical approval. But in terms of your pricing question, the price outside of the US, well, well, as you know, for the most part, always be lower.
Hartaj Singh: It's highly unlikely to 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 without preempting what that price might look like is. As with all ultra rare diseases and rare diseases, we can still make make a market and build a very healthy business in each of those countries and that's fully what we expect to do. 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.
Hartaj Singh: Thank you. Yeah, so Hartaj, it's a great question and it's certainly something that we're trying to address right now which is, you know, we know it's an autosomally dominant transmitted disease. So, you know, 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. What we've been trying, what we've, you know, been a little surprised by those and then I guess it relates to the, you know, the fragmented nature of our health care system is that oftentimes these family members haven't been tested.
Hartaj Singh: Some of that is just due to lack of awareness, some even amongst patients about the genetics of the disease. And some of that is just due to the health care system, you know, 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 patients right at the center of this. So, allowing patients and families to actually initiate testing. So, if a patient is diagnosed with APBS and a family member wants to get tested, we've initiated, we started a program that will allow that to happen by having the family member themselves initiate the process.
Hartaj Singh: And it doesn't need to go through the, 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. But I think, you know, removing these barriers to genetic testing for to allow appropriate testing in family is, I think a, will likely be a significant source of newly diagnosed patients. And we're, we're starting that program, we started a little bit of that already, but really putting that in full force now. Great. I like that is that that that helps. Thank you everyone for the questions. Thank you.
Operator: So, now I'll go to the next question.
Operator: And your next question comes from the line of five months old from first Berlin.
Operator: Please go ahead. Yes. Hello. Thanks for taking my question. So, you've already identified 150 APBS patients over the age of 12 in the US. I was just wondering how many of those patients do you ultimately expect to be able to enroll? I'd say the vast majority of those Simon, they're almost all of them. Okay, so almost all. And that's just the beginning, right? Because you heard. Yes, I know there are other patients, but I was just interested in, in patients, you've already identified, so we should have seen over 90% or 95%.
Operator: Oh, I think there will be very high percentage of patients that indeed will be interested to get into that. Do you want to treat them to correct? That's our experience so far at least. Okay, and in particular, particularly in the US, there's not, you don't expect to encounter a problem with the last 10 or 20% because of lack of insurance coverage. I mean, how does, I mean, how does that work? I mean, presumably there will be some patients without insurance coverage.
Operator: Can you get those as paid therapy on paid therapy as well? Although those are questions difficult to answer, but generally speaking, if we look at our experiences, we don't see any issues with raising to dots coming up. And of course, we can also ask patients that have limited or no insurance coverage, but there's always a ways of means. Maybe you want to comment on that, Stephen? Yeah, I think there's, I mean, there's all kinds of different types of support available for patients.
Operator: And also a public program. The, the, the, try and make sure that patients have access to something. So, you know, I'm like, I haven't looked at this for a while, but I believe we have very, very few reconnest patients, for example, in our patient assistance program, which would be essentially free supply on an ongoing basis. And then when they're in there every year, we're working with them and with their physicians to find an insurance plan for them will find an option that will be to pay therapy.
Operator: So, I apologize, I can't give you a specific answer, but if I look at the reconnest experience and my experience in red as easy as outside of farming, I think the vast majority of patients in the end, we'll get, we'll be on paid therapy. It can sometimes just be a heavy lift in those last few percent together. Okay, thanks very much. That's very helpful.
Operator: Thank you.
Operator: As a reminder, if you would like to ask a question, please press star one and one on your telephone and wait for your name to be announced.
Christian Glennie: We'll now go to the next question. And your next question comes from the line of Christian Glenny from Steve, please go ahead.
Anurag Relan: Hi guys, I thought it was just quick follow up if I can on Joenja and the pediatric study in the four to 11 year olds, looking at enrollment almost complete there. So just reminder in terms of the end point for that trial, the timing of that point and therefore when we might see data and then the expected sort of rough mix of the under 12 as it relates to this fall to 11 year olds. I mean, you know, if you think about say the 50 remaining patients identified in the US that we know how many of those would be fall to 11.
Anurag Relan: So let me answer that question, the second question first Christian. So we again, we based on our current experience in a PDF about a quarter of patients overall are below the age of 12. Amongst that quarter, three quarters are in the age range, at least three quarters are in the age range of the first study or four to 11 year old study. So, you know, we know these patients actually have a disease at birth and many patients do begin to manifest symptoms early on.
Anurag Relan: 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 four age groups. So that's a little bit on the the breakdown of the age distributions across a yes in terms of the pediatric study and the end points that we've had in the adolescent and adult study. So, you know, I think what we'll be able to do is once the study is fully enrolled and we've got 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 is the results are possible.
Operator: Okay, thanks Joseph. Thank you. There are currently no further questions.
Simon DeFrees: I will hand the call back. All right, thank you very much. If you close your remarks, thanks for attending. You can see now that, as stated last quarter, that the company is now starting a law growth trajectory supported by the foundation of Rukinesh provides and of course driven by the future expansion of Jeroenja, outside of the United States but also inside the United States, you know supported by lots of efforts that we are undertaking and initiating that she brought in the patient base and of course, as always with new immunists with new genetic diseases, the definition of the disease will broaden.
Simon DeFrees: 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, putting us 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 clinical development and do the successful commercialization as we do with Rukinesh and with Jeroenja.
Operator: So that said, thank you very much for attending and we look forward to updating you again on our four-year results call which will be in March of next year. Thank you very much. Goodbye. Thank you.
Operator: This concludes today's conference call. Thank you for participating. You may now disconnect.