Full Year 2020 Orphazyme A/S Earnings Call
[music].
Good day, ladies and gentlemen, and thank you for standing by and welcome to the orphan <unk> and will report Twenty-twenty and.
At this time all participants are in a listen only mode.
So I just figured per state here and there will be a question and answer session.
Gotcha and during the session you will need to practice star one on your telephone and.
And that's why I had that this call is being recorded the second of March 'twenty, 'twenty, one and although I'd like to hand, the conference on the to your first speaker today Molly porch had the global communications. Thank you. Please go ahead.
Thank you Bernard and good day, everyone and thank you for joining us with me today from or for time or under thresholds interim Chief Executive Officer, and Chief Financial Officer, Thomas Lately, Chief Medical Officer, and Molly painter President of offers on the U S.
The slides for this call are available for download on the investors section of our website. Please note that the Q&A will take place at the end of the presentation via conference call.
Before we begin I would like to remind you that during today's call, we will be making certain forward looking statements.
Various remarks that we make during this call about the company's future expectations plans and prospects constitute forward looking statements.
The forward looking statements are subject to a number of risks uncertainties and assumptions new risk factors and uncertainties may emerge from time to time and it is not possible to predict all risk factors and uncertainties, nor can we assess the impact of all factors on our business or the extent to which any factors for a combination of factors may cause actual results to.
Differ materially from those contained in or implied by any forward looking statement.
And might have these risks uncertainties and assumptions the forward looking events and circumstances may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward looking statements.
In addition, any forward looking statements represent our views as of today and should not be relied upon as representing our views as of any subsequent date, while we may elect to update these forward looking statements and the future. We specifically disclaim any obligation to do so even if our views change now I'll turn the call over to Anders.
Thank you Mollie and welcome everyone and thank you for joining us.
On today's call I'll be making my introductory remarks and multi painter.
And you all commercialization strategy and then I'll review, our financials and then we'll move into Q&A with almost net losses will be available for questions.
Okay.
So.
On the slides.
Yesterday, we announced very exciting useful guidance, we appointed kicked off but that is on us.
Do you think take deposits there.
And basically beginning.
On April and.
And gave him and his family enough time to relocate to Copenhagen for me by States.
Chris is a global recognized leader with 25 years of experience and building for fiscal debt there.
Gross functional teams and free constant Christmas.
Christmas is the ideal yield for us.
Yes.
And the Leerink therapies to patients and he is.
Significant experience in commercializing rare disease drugs.
No wonder woman.
From a doctor and career at Amgen.
But he most recently served as senior Vice President and general manager for the year from coffee business.
And the Nexium.
And of ultra orphan diseases.
For 40.
And the.
Country.
And I must say personally, but also we see and.
And look forward and these interviews.
Increased up.
Upon his arrival next month and April.
And then on them.
Next slide.
2000 and building momentum slide.
And why this summer of 2020, we've worked and important momentum building vehicles on recovered as many goals again and extremely challenging backdrop for you to the ongoing global and then it.
During 2020, we significantly advanced our pipeline and air products by Mark Moore at first box and to approach and amplifiers I believe is a potential game changer, and certain music and deep red pieces.
Is that as our commercial sales and New York.
Kind of ignore and we spend our commercial books and he and Europe.
We reported per tend to draw from.
Cause it killed the achievement of these aggressive goals during 2020, we chose to St and our balance sheet globally.
It's Kevin spoke and Europe, and the U S and and and the U S. You also did a U S public offering.
And Q3 and.
And in total we raised more than one from 2 billion Danish gross.
Okay.
Overall, we're very pleased with the progress from 2020 and.
Horton and.
Is it based on who we are ready for it.
And since the launch of a muscle for them to see and.
And the United States.
Very simple.
Since before this adjacent products and five therapeutic benefit.
And the hail and five P M.
Okay.
And then the next slide which is the pipeline and the product.
So we are developing on multiples so rare diseases.
Diseases, we're limited on no suitable therapeutic options.
Beef and can create high valued and thanks for those patient population and the salaries.
And he's overnight.
On the title for fees.
And we are considering bringing and hepatitis diseases, which include empathy and wuxi and leases as debt.
And most of them all and.
And I just don't work.
Cool.
And purple and we have some new muscle diseases with coach and aggregation plays a central role protein and activation rate okay.
And so I wouldn't be.
And there's a whole market and many news and it switches diseases.
And patients.
And our survey.
And he disorder that is it.
Limits and conditions with high unmet medical need and we have reported the pivotal data and completed our NDA submission and the U S and received a priority review.
June 17, this year for two five the biting approaches and the prepaid and in Europe and November 2020.
English sheep disease, our phase two data showed clinical meaningful effect as early as six months regarding differently.
And we are excited about these data and look forward to provide and update on Apple and.
And the coming months.
And we must and we are looking forward to top line data from other registrational trials and stuff.
I B M and are less and the first half of this year as a reminder.
We have received both orphan and fast track this and Nathan for those two indications.
And with that overview of our cargo and significant milestones with the.
Keith this year.
And also on Tuesday this year.
I will then turn the call with them on.
Who will review all commercialization strategy for on multiple Molly.
Yeah. Thank you Anders and I'm really excited to talk today about the enormous potential and we have with Aramark for Mam.
And and and the next slide and it'll go over our commercial overview and as you know we're driving the program.
And towards the market and both U S.
And Europe and for for rare Neurodegenerative diseases on dirt highlighted in his opening remarks.
So our lead indication niemann pick disease type thing or otherwise known as <unk> and P. C.
Make it there about 2000 patients and the U S and Europe.
And bind to the diagnosis rate around 40 to 70 per cent.
N P C typically start for many patients and their childhood with around half of the patients a day under 15 years old from.
From children have very aggressive phenotype.
And as you see very very quickly that they look fine.
Fine motor and <unk>.
Cognitive skills and have difficulty swallowing.
And they have a very aggressive phenotype.
And are unlikely to survive beyond their teenage years.
Very devastating for families.
There are currently no approved drugs for the treatment of MPC and the U S.
And beyond and P. D. We are investigating are mark I'm all for a O N.
IBM and gaucher disease.
We believe the AOS and IBM has the potential to be blockbuster blockbuster.
The market opportunity.
With about.
50000 patients for AOS, and 40000 and stay tuned for IBM.
These are two indications, which patient population.
That are looking for answers and new treatment option.
Currently no approved therapies exist for IBM and the U S.
And the next slide.
We talk about our established platform. We've worked very hard in 2000, Twenty's and felt our organization for the lines and as it stands today, we're ready for the potential launch of Aramark Mall and M. P. C. This year.
We now have that fit for purpose commercial model that is scalable for NBC and additional indications.
We have established our U S headquarters and Chicago, Chicago, where I left located and we're putting together a strong multidisciplinary team and the U S with experience at more than 15 ultra rare for a rare disease launch it.
Our key account managers and national accounts reactors are in place and ready.
Part of this work, we made sure to keep our manufacturing and supply on track to be sure. We meet anticipated demand given our narrow window of approval.
Our commercial organization is very strong across all these disciplines and we're poised and ready to go upon approval.
And until such time for you.
And we'll continue to enroll patients into our early access program now expanded to 12 sites and the U S.
We're actively engaging with U S payers to support patient access.
And have completed and scheduled presentations, representing 77 per cent of managed lives nationally and 55 per cent of the total and P. C community.
This is all happening despite COVID-19.
And so debt is work that is happening virtually outside the U S. We've established Yankees and France and Germany.
And we're building on your European commercial infrastructure with the hiring of general managers and France.
Doc region, Spain, and the UK.
For the next slide we'll start talk and you can talk about how we have talked about our commercial readiness.
So, let's talk about our global strategy and.
And how will work to drive adoption upon approval. There's a three three pronged approach for the U S Europe and the rest of the world.
And you asked do we expect for air market will be the first to.
And market for M C.
Our research shows that payers will for well prioritize approved treatments as first line therapy, which is very positive for us.
And Europe, we expect air Mark them, all to become and add on therapies and they go for that which is approved and in EU for N P C and and.
Yes.
Off label and the U S.
And about 40 to 50 per cent of patients.
For the rest of the World you plan to position Air Mark Mall as a first line therapy, and all territories, including U S and Europe, hopefully transition patients who are currently on off label therapies to Arab ethanol.
On this slide 10, which will transition to I want to emphasize our exciting and near term opportunity with air Mark them all to become standard of care for MPC.
There's been a lot and movement and the empty development space lately.
And the key programs from large pharma being closed which has caused tremendous disappointment is well informed patient community.
In addition, it's worth noting that the becka became generic and 2020, which we believe.
With these recent events and the MPC and development landscape, we are more confident.
And then ever and our ability to deliver the first novel disease modifying treatment to patients suffering with empathy and we believe the Aramark mall is well positioned to become the standard of care and that's indication.
And the next slide I'll talk about one other reason that we feel so strongly that earmark for mall and M. D C.
And is the extensive physician survey work that we conducted and the outcome and feedback that we've received the kols feedback suggest that patients would receive are mark them all as an important part of their treatment regimen, whether it's first line or add on.
This feedback is incredibly encouraging not only for <unk>, but for the mtc patients that we serve.
I'd like to point out a specific quote here from Dr. Mark Patterson, Who's a leading expert on and P. C from the Mayo clinic, and the U S and globally recognized no neurological expert.
And you reinforce it.
And yes, we see community that had been waiting for decades for an approved therapy for this disease and the ability and the availability excuse me of air marker mall.
Well offer and important treatment option for clinicians and great hope for affected individuals and their families.
We're very pleased to have the support of these key opinion leaders as we prepare for a potential launch.
Next slide please.
A key part of authorized approach since inception has been and emphasis on partnering and our strong relationships with scientific collaborators and patient advocacy groups.
We have established relationships with.
Specialist centers and are building, our steering committees and advisory Board.
For example, key opinion leader and treatment team.
To build out our medical education and Congress program.
This slide really shows our partnerships with all for rare disease indications, including the recently formed partnerships with check rare and.
Burning platform for health care professionals that is helping to educate on M. P C.
Our partners continue to inform our approach.
Elevate how we show up for our patient communities, we aim to serve and remain a key component to our strategy and all four indications moving forward.
And the next slide.
I'll talk about how I'll close my remarks here and proud to announce the brand name for Aramark and <unk>, which is my thoughts.
We've been working very hard on this campaign and we look forward to share any additional details and we are able.
So to reiterate we have and huge opportunity to make a real difference in patients' lives.
And this work is very important to me.
I've dedicated my careers for helping people and their families living with rare disease with rare diseases and I really am deeply moved by the patient community.
And their drive and their push for innovation.
For two minutes responsibility for these patients and I.
I hope that we're able to deliver on their expectations.
With that.
I'll turn the call back over to Anders to review financials for 2020 on.
The floor is yours.
Thanks, Molly during 2020, and we laid the foundation for our future with investments and our commercial infrastructure and preparation for the potential launch of a Muslim NNPC and.
As you said Marty described we have established a fit for purpose commercial business and when I won't persist and to bring the product for the patient is approved and fill and maximize the potential of thought and marketable should it be success.
Successful and other indications for live and you go from the figures.
Here you can see the thickness.
In summary for for 2020.
Net loss widened from minus 300 for Devon.
For the balance of 630 free for 2020 guidance as a result for the increased operating expense is associated with our prelaunch activities and to support our growth.
Operating expenses amounted to $609 billion.
Net loss for 2020 and I'll go through this more detailed on the next slide the additional $24 million and Lux came from this financial expenses, mainly related to the increase and net foreign currency losses of 13 million Danish kroner equivalents.
$2 1 billion as well as increase and interest expenses from data to the loan agreement and create capital for $6 7 billion Danish kroner or equivalent to one from $1 billion for U S dollars total.
Net loss per share was 22 three things for us.
And again for 2020 compared to 16 four nine.
And the previous year.
69, so we conclude the year.
727 million and tests.
Around $119 million.
Our cash position was significantly boosted.
Boosted with the prior year as a result of the two for certain financial ratios. During 2020, all complete and the U S listing.
Yeah.
And.
Look on the next slide the operating expenses of legacy to spend a bit more time on the operating expenses, which increased significantly during 2020 compared to prior year, particularly at <unk> and <unk>.
G&A costs were driven by prelaunch activities and to build a love for our commercial infrastructure and global organization.
Our U S and Switzerland based commercial team with <unk>.
90 employees and we increase the mill cross sales activities, particularly for MPC.
And case with the scientific community.
Based on these programs.
Net interest expense.
And it was also in 2020 compared to prior year with Mega juices to Eagle Ford cuts and destinations and other external assistance and all.
Also CF based compensation.
In addition, we had.
And for you.
And as and space and finance legal and Ctrip for the growing old and faces.
As we have become globally.
Okay.
R&D expenses for 2020 order gross compared to prior year as we advanced our trial and IP M&A. This and also create a C and supported our registration activities and and MPC and particular the increased cost for food.
And both of the free clinical pharmacology with freight and with trust income.
<unk> clinical safety and reporting activities and ongoing trials and the expansion of our R&D like Coke and for us.
And the next slide please.
Okay.
That's our financial outlook as we said before we expect for 2021 from the transformational year for folks on.
And with the potential approval of our first pass.
And the readout of two late stage for us.
For this year.
The outcome and the timing of these events are clear and key factors and driving how do we think about 2021 and our outlook. We expect our operating expense for principal and wanted to be and a range of 800 for 850 million being from us.
This is driven by continued investment on R&D to support the advancement and completion of our multiple clinical trials and the IP M&A list included data Readouts and preparation for wildly successful.
Anticipate continuing this and commercial infrastructure from the U S.
Through the for product launches on locomotives is approved.
And kind of take rate on regulatory outcome, but overtime you are preparing for success and are well positioned to don't on market.
And more within a narrow window of a potential approval.
Our revenue forecast.
Currently anticipate a gradual increase in sales from our multimode during the second half in the range of 62 to 120 day as soon as all.
Okay equivalent to around $10 million to $20 million.
Yeah and this forecast also includes small amount of potential income from named patient sales and other countries.
Importantly, our outlook for 'twenty and 'twenty, one assumes that we award for calls with you bought it from USDA loans.
And once approved.
And that doesn't come monetize it probably for the U box and the value of these are skewing towards each other.
Between 95 to 100 and Tim Murray.
So taking all this into account we expect our operating expense to total between 808 on 50 million Danish kroner.
And so and the year with an excess of 306 million, Danish kroner, and cash or recruitment to around $57 million.
We believe we have a strong financial position and balance sheet Vegas for commercialization and MPC and additional indications while also continuing to total and leading rare disease company.
Next slide please.
Before getting for Q&A, Let me review, what's coming up for the oldest on and this transformational year ahead of us.
And in Florida, and you're focusing on getting a multiple to the mtc patients with early access programs and.
And I won't go preparation for launch and commercial rollout both in U S and Europe will also explore possibilities for partnerships and the rest of the world.
We have completed and based on mix in the U S for a multiple and MPC and our <unk> date is June 17.
We also submitted the marketing authorization application in November last year, and we anticipate the potential approval.
For fourth quarter of this year.
And beyond for MPC, we expect top line data from <unk> and IBM product and the first half of this year.
And the decision on developing on multiple <unk>.
As you've heard today is very exciting time for <unk> with a lot happening for our company and importantly for the patients.
And the city.
And I would like to and second coal to the moderator for Q&A.
And <unk>.
Okay.
Thank you ladies and gentlemen, we'll now begin the question and answer session.
Or if you wish to ask a question. Please press the star and number one and your telephone keypad and wait for your name to be announced.
Yes.
Once again for a question. Please press star one on your telephone.
Your first question comes from the line of Thomas Powers from Danske Bank. Please ask your question.
Thank you very much a couple of for questions for from and Yeah. So so just if we just dive into the.
For your guidance sort of on guidance. So I'm just wondering how much impact should we expect from from the <unk>.
The commercial preparations in both the <unk> and IBM and trials if they of course read out positive and then in the Saudi and in addition to debt.
And I'm just wondering if you should have the capacity internally.
And to submit two NDA filings.
In parallel and.
And could you maybe give us some guidance on the expected filing timelines.
Mid <unk> after we have the top line readout here and in the second quarter and then I have a couple of pipeline questions for so so maybe we could just kick off with the financial stuff here.
Yeah.
Yeah. Thank you so much.
And it's.
Good question. So the cost so what we have included and the outlook is.
The filing cost for.
For this might be and if there.
Yes.
And then also as you address you know for sure.
And capacity and so on and so it's something that we're working on financing.
Solution on but I'll, let you.
Yeah.
And so food and <unk>.
Defining process, but on the cost side.
And it's very much depending on data, what's the extra costs, we can see and.
Coming in and in 2021.
Because it is very strong data and a less that's one situation and then if it's from basin IBM is another one because of difference between the indications and both combined is perfect and then.
And of course, we will have two two.
To engage with patient organizations.
Weighted Medicare with their size and and so on so we will have the ramp up and cost on certain areas.
If I put a number now before we have the data.
Comfortable with Cowen.
But.
Platelet would you would your guidance.
Our ability to to file two products simultaneously and maybe also if you have it.
And any let's say.
Decided to talk about timing.
So that's on.
Yes, thank you and as well Thomas obviously.
Filing and NDA and MAA does take a lot of resource and so you rightfully asked that question.
We have to.
It seems one working on the scale and one on the IBM <unk>.
And looking to take input from debt.
The resources needed.
Obviously.
And in a scenario, where both trials lent themselves for for submission of a file we will have to look at the border.
Simple indications.
And would have to take priority that will be based on both on the assessment of the likelihood of success, but also on the unmet need.
And we will we will have we will include additional resources to advance filings as quickly as possible.
You should anticipate debt, it's not been exactly.
Parallel filing so it will be important and sequence in the in the filing with the credit quality and.
And it's still and this year.
Okay. Thank you and then and if I could just ask on on the air This trial.
Just.
And.
Perhaps to the open label trial, you managed to recruit 120 patients.
Or you can say about half of what you had and the phase III and and combined with the historical data with an average survival for 18 months. So I'm. Just wondering if you have now we have some whether it's been a few months here. So so anything that you view that makes you and maybe a little bit more upbeat or downbeat on based on on the number of patients.
And based on all the affects us like debt drop out rates or whatever you see.
And amongst these patients that could sort of.
Reflect the non.
Both patients who are able to recruit and the into open label trial and then I have a last question also just on the EAP.
And I think Bob you said, you had and they will have for a 12 centers and in your prepared remarks. So I'm just wondering how many and proceed patients is actually by now on the early access program and then do you have any sort of predictions on unexpected number that you aim for him and now with other in connection with let's say midyear launch maybe and now.
We at least have to with the two 270 program for Matt and Cros and now being terminated so I'm just wondering whether youre seeing a big uptake of patients coming through this early access with was that program being shut down and thank you.
Okay.
Yes.
And like you used to.
To talk about the drawdown on the list and then already paying debt. If you could comment on where we are on the <unk>.
And the U S and India.
Expectations for for further improvement.
So so first as a reminder, we are still blinded.
Two treatment allocations and so I will refrain from from.
Speculating on the outcome of the trial.
Having 120 patients rolling over from the.
From the randomized file into into the open label.
It's absolutely within within the frame that day.
That I expected.
And mind, you have had Ah and Nash.
Number of patients have good traction on for patients who have met.
Their survival endpoint.
And so have died and we have a number of cases grew as expected have discontinued from the trial on.
All the numbers we have to date.
On a blinded level are well within our expectation.
Yes.
Okay.
Thank you and then on VIP.
Yeah on the Atwood team.
I'll take that question.
It's okay.
And we have I believe now to your point, we've had on increased interest on.
Obviously with some of the market events taking place.
But.
Just around 35 page.
Patients enrolled.
And with a split towards and children and adults skewing to decide and.
And more adult patients.
But a fair amount of children have enrolled as of late so.
And we anticipate on that to continue.
Gross.
<unk>.
There is a significant need for these folks.
Alright, great. Thank you very much.
Mhm.
Yeah.
Yes.
Your next question comes from the lineup and Theres from Red Eye. When you ask your question.
Okay.
Good afternoon can you guys Sammy.
Yes.
So and or something.
And to get my head around the outlook as well.
And do.
Do it and my Chris, but there is a description and said there between what you guide for operating loss and the cash position and it's there for instance, something that lies behind that you see a buildup of cash.
Capex and or our net working capital for the current year.
And very insightful question here, so yes, so when the product is.
Essentially approved.
And whatever you produce that product.
And you put on the balance sheet, so there might be some cash flow investments or some investments and products that is not on on the P&L.
I would say that's the most alright, that's the biggest change that would take place and when we get the approval potentially.
Okay.
So buildup of working capital basically yes.
Yes.
Okay, and then in terms of debt early on.
The pipeline can you.
Can we expect and anticipated milestones for the current year in terms of preclinical or and or our research activities.
If there will be anything there would be and the second half.
It's an early stage program.
For sure we haven't.
And I have defined a new milestone for for second half yet.
Is it possibility on it.
Okay. Thank.
Thank you.
Yeah.
And your next question is from the line of Devine and from Bank of America. Please ask your question.
Yes.
Hey, good morning, and can you hear me.
Yes.
Okay perfect.
And can I ask a question on your upcoming data for E. L F.
Specifically what type of information should we expect to see at the top line.
And also is it your belief that you would be able to apply for approval based on debt.
Pacific study if it is in fact deposits held.
And then the second question is on the competitive landscape and E. L F.
There are quite a few companies that are trying to explore this indication one day, that's specifically like your thoughts on is from a company called the analytics, which.
Last year I believe for recently could you.
Statistically significant positive data for their study.
I wanted to know how you think about how your program could differentiate potentially from day one and.
Noting that the potential for AOS is quite large and there could be room for many players just wanted to get your thoughts in general about where you think.
And your program and E L F could be differentiated for that thank you.
Thank you.
Later, we will just start.
And to answer.
And you can color here I think.
Very good.
List price.
Yes.
And I tried to Chuck it down and so I hope I remember everything otherwise you may have.
Sorry for the questions well first.
What do you think kind of what is the information you would we expect from the topline results.
We will have 18 months, which is the primary and.
At this time point up the primary assessment and have.
18 months data on the primary endpoint for cost which is the company.
Combined effects and a function and survival.
On both components, which are also on secondary endpoints, so AOS and for that are.
Our and survival. We will also have information on on on the pulmonary function and additional secondary endpoints and we do anticipate to have information on the Biomarkers here the greatest interest.
Of course with the newer filament light chain.
And CSF and in.
And.
And plasma.
Also of course, we will have to safety and Tolerability information.
You asked about analytics and the analytics are phase II trials.
Could you just maybe give a sense of the probability.
Two days and so on the OE.
Your assumption and that is also based on the type C meeting and the scientific with FDA and scientific advice with with <unk> is that this trial supports and approval if positive gross in the U S and EU.
And of course, we would also then have ambitions to go beyond those regions.
Okay.
And just filed was specifically designed also to address both needs from FDA and EMA.
<unk> expense.
With this regard and this actually does take me to the comparison to the analytics program.
And a six months.
Randomized placebo controlled trial, followed by an open label extension.
Okay.
The primary endpoint and the analyst try and work the LSA for us are at six months.
They have since reported a.
Also and advantage of longer survival.
And with patients who were initially randomized to two.
<unk>.
Yeah.
For instance versus placebo.
Oh.
If you now want to compare our phase III trial that from 18 months trial is.
And if positive will demonstrate and effect both on survival and functioning and.
And we will have 18 months of randomized blinded data, which of course gives you.
And a longer time spent to understand the effect of the drop growth both efficacy buys and also from a safety and Tolerability perspective.
In addition to that we also have an open label extension that you also heard just earlier, so we will and can add additional data in patients who start treatment late and will even further understand the long term benefit of treatment of Italian more come on.
A remarkable.
Amplifying the heat shock response and not through stress response.
And with that.
Our remote promote would lend itself to combination and with the range.
Other treatments, so we do not see.
<unk>.
And <unk>.
Other treatments in development or approved being approved as b and in competition with unremarkable in our view.
The treatment of patients with <unk> will be a combination treatment that combines.
I'd.
Different treatments and hopefully tailored for treatments to the patients all along the lines of personalized medicine.
And here again, the broad and mechanism of action of <unk> lends.
And it's itself to be used as a component as a pillar of such combination treatment.
Yes.
Thank you so much and thank you Sir.
Yes.
Your next question comes from the line of <unk> from <unk> from Guggenheim Partners. Please ask your question.
Hey, guys can you on yes.
Yes.
No.
So just a couple of questions for me first one is on the revenue guidance that you provided some tons and tons of and then can you maybe talk about some of the push and pulls how should we think about the price.
And what about the range of patients that you assumed for this level of guidance.
And so.
And then.
It's a very good question, you asked and I won't ask you to talk about price before we get the approval.
Prudently and silhouette.
We have to look at it.
And the assumptions on volume pain that described the number of EAP patients that are in but also could come in until we get the approval and then of course in naive patients.
In the U S debt are not currently on treatment. So so so it's a combination.
Yeah, but is it the pricing is too specific for me to go into detail.
Okay, and then just one more question on the EAP adopted on melon crops and drug which has a reason for the guide can you remind us how big volume.
And what your expectations are for transitioning those patients.
Maybe and CEO program and what was the big between U S and Europe for day program. If you have that information. Thank you.
Yes.
We don't we don't have.
Specific information and we know that the trial was done globally, and and and also on Europe and U S.
But Alex you don't have and in charge of course.
If I can patients asking us.
That program goes.
Stuck and.
Having patient being treated in October of this year, so there's still some.
I don't know other specifics.
Thank you so much and thats all of them.
Thank you. Thank you.
Your next question comes from the line are free to borrow from Cowen. Please ask your question.
Good morning, guys. Thanks for taking the question I wanted to ask about any anticipated.
And safety monitoring requirements that you guys might have in the label and how that might impact either how your patient hub.
We will run.
And what it will mean to keep track of and ultimately.
The launch and I have a follow up.
Okay. Thank.
Thank you redo lately would you start.
Your debt.
Yeah.
I must say, we have not had and.
Concrete indication yet from the regulators on on on on obligations for safety monitoring for flow.
And on approval.
And I would actually like to refrain from speculating here.
Fair enough.
And then a question for Molly.
You remind us again, how the patient hub is set up is that other contract services are those.
And in house people and in the event and the potential early approval are they are they ready to go right now.
Mhm, yeah, yeah, they're ready to go right now so.
We are working with and outside source to set up our.
Some would deem and this business call it a hub or a patient services model.
And for each prescription to come through.
And the and be handled on a patient by patient basis.
Great and then last question.
Andre as you think about.
<unk> data and the IBM data coming and and the differences and market size.
I guess, how are you thinking about.
Potentially changing the price I know and we're in.
The answer to John's question, and you don't want to give specific price for MCC.
But what are the tools that you might be able to use.
Two I guess.
And then the price too.
And population.
And he is.
Yes.
Yeah. It's a good question and also because he wants to get the day to the full get the approvals of course is commodity also influences.
On the positioning and also on the price because.
If we have clear strong base and that's one scenario.
And so.
There's a lot of ethics here for <unk>.
Typically on the price to be.
<unk> made some assumptions on of course MPC alone and then we are also doing some similar zone.
Discussions on what to do it.
Strong data comes in because you know as the market itself must speak on a number of patients and also.
New patients coming into the disease, there for you and use might be five star and so is it.
The very big market potential and Cisco.
But molly.
And this is more on for you.
Area.
Doing a lot of thinking and this maybe you can provide a bit of color.
Yeah, I think I mean, I think you said most of it is we're scenario planning for them.
Various different for mutations and doing it based on.
Analogs and where the market is.
Today, So we can do so and responsibly.
And responsibly and.
And do it the right way.
And we're thinking through.
Every different scenario.
Yeah.
Got it.
I assume from all 800 [laughter].
Yeah.
Okay.
Yeah.
Once again for any questions. Please press star one on your telephone keypad.
There are no further questions at this time please continue.
Thank you very much.
And I would just total by saying that I appreciate it very much and participation in today's call and.
And we look forward to continue to provide you updates on that.
Appropriate, especially.
Half of this year and you can see all of the year.
Activity is going on so we will continue to provide information on on those activities and also pertains to prove and the second half and the EU.
And so you hear a lot from us.
This year.
Thank you very much.
That does conclude our call for today. Thank you for participating you may all disconnect.
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