Q4 2020 Acceleron Pharma Inc Earnings Call
Good afternoon, ladies and gentlemen, and welcome to the Accelerant and fourth quarter and full year 'twenty and 'twenty earnings Conference call. At this time all participants are in a listen only mode. Later, we will conduct the question and answer session and instructions will be given at that time.
Mind you This conference call is being recorded.
Yeah, that's behind the call over to MS. Jamie Bernard Associate director of Investor Relations and Exceleron. Please go ahead.
Thanks, and welcome everyone to our fourth quarter and full year 2020 earnings call the press.
Release reporting our financial results and addition to the presentation for today's webcast are available on the investors and media page of our corporate website at Www Dot accelerant and pharma Dot com.
Joining me on the call. This afternoon are Habib Dudley, our Chief Executive Officer, Kevin Mclaughlin, Our Chief Financial Officer, Dr. Jay Backstrom, our head of research and development you Jay Kengo, our Chief commercial Officer, and Todd James Our senior Vice President of corporate Affairs, and Investor Relations and.
The reminder, you'll be making forward looking statements regarding our financial outlook and additions of regulatory product development and commercialization of him and research activities. These statements are subject to risks and uncertainties that may cause actual results to materially differ from those forecasted and description of these risks can be found and our most recent form 10-K on file.
And with the SEC with that I would now like to turn the call over from the beat Bobby I see yes.
Thank you Jamie and good afternoon, everyone and thank you all for joining us today.
2020 was of great year for Accelerant, and we made significant progress and reached multiple of crucial milestones across our pulmonary and hematology programs I could not be more grateful to the entire accelerant and team for their contributions to the many successes we were able to achieve.
Reflecting on the past year I'd be remiss, if I did not first acknowledge those directly affected by the pandemic the hell.
The care workers and many others, who continue to work tirelessly to help protect and support our communities.
Like many of our peers here and accelerant and we faced multiple operational challenges as we quickly pivoted to a remote work environment and March.
I'm incredibly proud of our team's resilience and resourcefulness and despite the necessary adjustments and we continue to be committed to our mission and operate with the sense of urgency for patients.
We remain well positioned for a productive year ahead and support of our long term vision to build the industry, leading pulmonary and hematology franchises.
To briefly highlight our accomplishments I will begin with Eric seller on led pulmonary program.
And early 2020, the announcement of positive topline results from the Pulsar Phase II trial established proof of concept first of kind of stepped in patients with pulmonary arterial hypertension or ph.
Following the results, we received breakthrough therapy and prime designation from the tighter set by the FDA and EMEA respectively.
We also presented detailed findings from the Pulsar trial at a number of major medical meetings, including the American Thoracic Society, and the American Heart Association annual Congresses.
Right before the end of the year, we were able to initiate a registrational phase III trial called stellar which I will touch on and more detailed during today's call.
In addition to Chicago.
We are advancing 813, 34, and accelerant of discovered asset, which recently received orphan drug designation from the FDA for the treatment of systemic sclerosis.
We plan to initially develop <unk> 34, and systemic sclerosis associated interstitial lung disease or that's the ILD.
It's rare pulmonary disorder affects more than 50000 patients and the United States and Europe and is the main contributor to mortality in patients with systemic sclerosis.
We are currently and the process of planning for a phase one b phase III study to evaluate the activity of <unk> 34 in patients with the SSC ILD and expect to start the trial and 2021.
Moving to our hematology program alongside our partner Bristol Myers Squibb, we achieved multiple regulatory milestones during red was the first full year of commercial availability and the United States.
Including approval and MBS and continued market expansion.
<unk> also known as the patents that the the first and only erythroid maturation agent approved and the United States Europe, and most recently in Canada for the treatment of anemia, and certain blood disorders.
Despite the external challenges and the face of the global pandemic, our joint commercial team was able to drive early sales success and I will.
Highlight the program and more detail shortly.
Diving little deeper into our pulmonary program. Following the top line results presentation from the Pulsar phase II trial at Ats and June we.
We had the opportunity to share updates from pulsar as well as the spectra trial are smaller open label Phase two trial at the American Heart Association scientific sessions in November.
The pulse of our presentation highlighted 24 week echocardiogram results, which was recognized by H eight and the cardiopulmonary best abstract.
The results showed that the tighter set improved right ventricular function the.
These data are very encouraging that's right ventricular failure is the hallmark of P. H and the key determinant of the patient survival.
The presentation from the ongoing spectra trial showed treatment with the kind of ex that resulted in substantial improvement and multiple hemodynamic measures along with exercise tolerance and capacity at week 24.
Importantly, the results are consistent with pulsar and further support our belief that the tighter set of unique mechanism of action has the potential to reverse pulmonary vascular remodeling.
Turning now to our phase III clinical development plan and vision Crystal Patterson.
At the end of the year, we initiated our phase III Registrational trials stellar and as a reminder, stellar as the phase III randomized double blind placebo controlled study comparing the efficacy and safety of the tightest debt versus placebo and approximately 284 patients diagnosed with P. H with functional class II or III.
The primary endpoint is the change and six minute walk distance of 24 weeks compared to the baseline.
We're also preparing to conduct two additional phase III trials Hyperion to explore the tightest that and the early stage intervention, setting and zenith and patients with who functional class for ph.
Results from our phase III studies will add to our understanding of the tighter step of unique mechanism and support its potential to be of backbone therapy for patients with all stages of the ph.
Moving to hematology 'twenty and 'twenty was the successful first year for Red blood cell our partner BMS reported ex approximately 274 million and of $115 million and net sales of red blood cell for the full year and fourth quarter respectively.
While it is still early in the commercial launch of rebel itself. We are pleased with the market penetration that our sales team along with our global collaboration partners at BMS of the cheap.
Specific to the Mds patient population, where there had not been a new drug approval and over a decade.
<unk> portion of early sales were driven by pent up demand from patients who are experiencing inadequate responses to standard of care or who are suffering from high transfusion burden.
And on our third quarter earnings call. We described the start of the shifting demand to patients earlier and their M. D. S journey.
We are continuing to observe this ongoing shift and the type of patients treated with red blood cell, which we expect to contribute to potential short term moderation of growth or flattening of sales and the first quarter.
The commercial teams are focused on achieving further penetration into the longer term underlying demand and patients earlier and their MBS journey, along with optimal dose of <unk>.
Brazil to maximize the patient benefit and increase duration of treatment and this population to drive growth and 2021 and beyond.
Physician feedback remains positive with significant awareness of the brand as we continue to add prescribing accounts in the U S.
In addition, we are still in the very early stages of the launch of breathless, though outside of the U S and the.
The second half of 'twenty, and 'twenty, BMS launched replicable, and Austria, and Germany, and we anticipate additional launches and various markets globally over the course of 'twenty and 'twenty one.
The reimbursement.
We also continue to monitor for any potential effects of COVID-19 may have on sales of promotional activities and all region.
And we think about the long term vision for the brand and our partner BMS continues to advance the development of rub yourself of the treatment of additional patient population struggling and union associated blood disorders.
There are currently three trials underway to further investigate and this pattern. That's the effect on patients with first line lower risk Mds non transfusion dependent beta thalassemia and myelofibrosis.
BMS is and the process of initiating the independents trial and patients with myelofibrosis, who are on the JAK two inhibitor and require red blood cell transfusions and.
And we look forward to the presentation of results from the beyond trial and beta thalassemia and the first half of 'twenty and 'twenty, one and the commands trial and MBS and 2022 or later on.
All in if we continue on the successful trajectory with these current and potential future applications. We estimate annual peak sales of bread, Brazil of more than $4 billion.
And with that I would like to hand, the call over to Kevin Mclaughlin, our CFO to review the financials and then we'll be available to take your questions Kevin.
Thanks, Habib and good afternoon and everyone.
I'd like to refer to our press release issued earlier today for a summary of our financial results for the fourth quarter and full year 2020, and take this opportunity to briefly review a few items.
We ended the fourth quarter and full year with approximately $857.5 million and cash cash equivalents and investments.
Revenue for the fourth quarter of 2020 was $25 $9 million, which includes $2 $9 million of cost share of revenue and $23 million of royalty revenue from net sales of rebel of sale.
Revenue for the full year was $92 $5 million, which includes $12 $7 million of cost share of revenue 54 of them point $8 million of royalty revenue from net sales of red blood cell and the recognition of the $25 million regulatory milestone.
From the approval of rubble zone and Europe, all revenue was derived from the company's partnership with Bristol Myers Squibb.
Total costs and expenses for the year with $259 $8 million the company's net loss for the year ended December 31, 2020 was $166 million.
Before we turn to the questions I'd like to take a moment to echo the sentiments and.
Say, how proud I am of our team's ability to execute throughout 2020.
With that I'd like to open up the call to questions.
Operator.
At this time I would like to inform everyone to ask a question and you will need to press star one on your telephone.
The draw your question press, the pound or hashed E. Please standby, while we compile the Q&A roster.
Your first question comes from Yaron Werber from Cowen. Please go ahead.
Yeah, Hi team and the congrats and thanks for taking the question really great to see of good launch in this environment.
So a couple of questions I have on revenues and maybe the touch first Habib you mentioned, you're expecting maybe a flattening of the sales in Q1 is that and partially due to the donut hole or is there anything specific from Q4. The Q1 or is that just ongoing kind of the journey shift and then what drives the reacceleration in the U S.
So that's the first of the first question and then second what's the cadence of let's say new countries coming on board and second half of I'm, sorry, This year and Europe. Thank you.
Yes. Thank you for your question and your own.
So with respect to the commentary in terms of moderation of sales growth or even perhaps flattening, it's really not much different than what we had said on the third quarter earnings call, where we are at that time of your own. We described a start and the shifts and demand of patients to earlier and their Mds journey.
And we're continuing to observe this ongoing shift and the type of patient that's being treated with the rep Lasalle and we expect the shift.
And to contribute to a potential short term moderation or even flattening in the first quarter and the meantime, the commercial teams continue to be focused on achieving further penetration going.
Managing the long term underlying demand.
And capturing.
And the patients that are earlier in the Mdx journey and and also focusing on on the optimal dose of rebels, though and all and all to maximize the potential benefit and the persistency I E. The increase duration of treatment with this population, which we believe is going to drive significant growth.
Once we burn through that the pent up demand.
And when you think about.
And what the investments that we that BMS is making and rubbed Brazil in terms of lifecycle management.
And the continued high awareness and.
And the early feedback that we're getting in terms of.
How doctors and patients are.
Experiencing early days with from Brazil, we have high confidence and the success of for future growth.
And potentially further indications through the lifecycle management and as a result of that together DNS and accelerant and just recently had increased guidance for peak sales potential of over $4 billion. If indeed, we are successful and with those lifecycle.
Programs.
With respect to the cadence.
Haven't really given specific details obviously dms is responsible for the commercialization outside of the U S. We've already talked about the approval and in Austria and in Germany, which has already contributed to the sales in 2020, but what you can expect it's probably a significant uptick and those.
The countries that are getting reimbursement and in the second half of 2021.
And just the final question the beyond study and we're expecting data initially sort of late last year and then early this year on the it looks like its first half and non transfusion dependent and just give us a sense of the.
It seems to be a slight shift what what's driving the thank you.
Yes, So I think last year, we said the is going to be the first half of this year, we haven't changed the guidance, we're keeping that to the you know you can expect to see that data.
And the first half of 'twenty and 'twenty, one and.
And you can probably estimate.
The respect of Congresses that would be the most suitable for that and so and we're sticking to that and your own.
Alright, thank you.
Yes, thanks for your questions.
Your next question comes from Carter Gould from Barclays. Please go ahead.
Great. Good afternoon, guys. Thanks for taking the question I guess first I believe now that we're coming up sort of approaching a year.
Since the Mds launch.
Maybe just your initial of kind of sense on how duration and persistence of sort of playing out and then maybe just.
Quick follow up as we think about some of those O U S launches why Wouldnt, we expect the same sort of bolus effect that we saw in the U S to play out on the country by country basis. Thank you.
Yes, Thanks, Carter really insightful questions and specifically the second one and when we talk about the.
The commentary regarding a potential flattening or moderation of sales that is and the markets that we've already launched and such as the U S and so the pattern that we're referring to you're right. It's country specific and so if you were to look.
Look at the country by country Theres, absolutely no reason to believe the different markets would have a different pattern. So that's that's a very fair comment.
With respect to the your question on persistency.
And it actually is probably the most underlying factor behind the commentary and the trajectory, which is very very much expected and the market, where there's been very little innovation over the last decade, or so and where the pent up demand is very very high Ed.
And as you would remember from the the clinical trial, what we saw was a patient seeing a durable clinical.
Benefit, which was presented at ash, a few years ago approaching two years.
So what's happening is that and on top of that you saw much better response rates and patients who had a red blood cell transfusion burden of six units or less every eight weeks versus those that were greater.
And so what happens when you get a disproportionate amount of the patients.
That are early and the launch that are actually how the higher transfusion burden you would expect that the persistent and C to be lower than what you would've seen and the clinical trial. When the average was the was six.
And so that is the direct result of this pent up demand and as we continue to trends to transition into the patients that are earlier in their patient journey, we expect to continue to see persistence the increase and as well as we continue to educate the patient the treater group on the appropriate.
Dosing schedule, which will also enhance the efficacy and persistency.
Thank you.
Your next question comes from Chris Raymond from Piper Sandler. Please go ahead.
Hi, This is nicole good breadth of <unk> on for Chris Thanks for taking the question.
Maybe just one on on the pipeline for 13, and 34 and SSC ILD.
I know you guys have noted that there are about 50000 patients from the U S.
And as we think about the market opportunity are there certain subset of these patients that might respond better to therapy like 30 34.
Yes.
Early versus late onset of ILD or auto antibody status and how does that green how youre thinking about the design of the theme on Btu.
And any color here would be great.
Yeah. Thanks. Thanks for your question, Nicole and I think what I'll do the patch that went on to Jay Backstrom, our head of research and development Jay.
Yes, Hi, this is Jay so far one be too.
And we're keen to involve patients that have both evidence of skin disease as well as the potential to have the pulmonary involvement as most patients two and <unk>.
Really we're looking for the opportunity to very quickly within the context of that trial come to a conclusion of dose and our target engagement et cetera that would allow US then to move into phase two and we have some ideas around what could patients would look like the on that.
Opening study and if theres a lot of and.
Objectives that we're trying to achieve and will as we go and learn more about it as Habib had mentioned previously we do plan to do on R&D day, and I would invite.
We invite you to join us when we get that scheduled because it will take a much deeper dive into both the protocol and our thinking around the biomarker strategies the selections.
Great. Thank you.
Your next.
Question comes from Geoffrey Porges from SBB Leerink.
Hey, guys. This is mark Wang on for.
Sure Jeff.
So.
Two questions from me on the first would be what is the latest information about enrollment into the frontline Mds trial with reckless at all and how confident are you of that that goes on all of these superior to Esa and Esa naive population and the fall.
Flow up to that is what is the margin of benefit and required for the.
The goal on commercial success, given the large price difference between USA and.
Robert So.
Thanks.
Yeah. Thanks, Thanks for your questions.
So just to remind you.
The commands trial is 100% of operationalized by our partners at Dms and.
And what I can tell you is that although we do not give any specifics in terms of enrollment and the pace of enrollment. What we have said is that you can expect to see top line data results.
Sometime in 2022 or later.
The first thing with respect to our confidence in terms of what has informed us.
To move forward into the command study, that's really multifactorial based on what we have seen.
From some of it.
Some of the company sponsored phase III results with the.
Yes.
As well as what we've seen with our own phase II data with the cans like patients and I will pass that on over to Jay Backstrom to shed a little bit of light in terms of why we are so confident.
Two of them made the move forward into another Registrational study like commands and.
And perhaps talk a little bit about what.
What that margin of benefit that we saw at least and phase II looks like.
Yeah. So I think you summed it up well from the data that we have for patients that are requiring transfusions of at the level that just just to remind that in his patients.
Still require some transfusions to get in and we're looking at are those that potentially of an equal level above 200 that group doesn't do well and and Esa and frankly, it's a superiority study that we really have great confidence and so looking forward to getting that trial completed as of the you've mentioned the BMS team is really putting a lot of effort behind it.
I think from what I see today, the timelines and we proposed.
Should the should stay on track and we will see as we continue to go through the year.
Yes, and at the end of the day all we have is our own data that we've looked at with her on phase two and looking at other company sponsored datasets and obviously, we cannot make cross study comparisons and that's why we've designed the superiority of design study.
And that if indeed, we are successful we will able to demonstrate and show that benefit.
Yes.
Thank you.
Your next thanks for your questions.
Your next question comes from Eric Joseph from Jpmorgan. Please go ahead.
Hey, guys. Thanks for taking the question. Good afternoon, I guess just on strength, so far as the <unk>.
Zoe.
Can you just comment on demand you're seeing and.
Org uptake that youre seeing and Rs negative patients.
Whether it's sort of.
And how efficacy has.
Emerged and that patient population and windows.
And then the patient sorry, payer pushback and I guess, just picking up of their clients and even the lifecycle management opportunities with new and.
CMS or thinking about can you just elaborate there on.
And some additional indications that you're pursuing is it.
Net of going vertical within Mds and on fibrosis or.
And the horizontal and other.
And the related indications of Youre thinking about thanks.
Yes, thanks for your questions, Eric and so I'll, let Jay answer the questions regarding lifecycle management in terms of what's ongoing and and how we're thinking about things with our partners, but with respect to your question on on Rs negative patients.
Really our insights there are from our own phase II study and.
In terms of in the market we have.
No reason to believe that there's any substantial use and the Rs negative population obviously the label.
As.
Specific to the Rs positive population and so any insights we have are really driven from our own clinical data, but I'll hand, it over to Jay to elaborate.
Yeah, So with respect to the lifecycle plan you can anticipate seeing kind of additional investigation within the MBS spaces and already.
And discussion in and out in the clinical trials Gov for combination strategies within the Mds space. So I think you'll continue to see more of that.
And there's obviously clearly and interest and the Rs negative patients is of course of the you mentioned, we had nice activity and our phase II data that is included and commands. So we can anticipate to see that.
So I think combination will probably be the the way to go and addition to us advancing and the earlier lines of therapy, and then build Airan beta thalassemia, because it really has turned out to be a really important therapy for patients and so if you think about Dallas EMEA and you can anticipate potentially alpha thalassemia around it so there will be <unk>.
And expansion around these areas.
But there's a lot of interest and the and the product and some more to come on that.
Hey, great. Thanks for taking the questions.
John Thank you Eric income and your next question comes from Ed White from H C. Wainwright.
Good afternoon, and thanks for taking my questions. So just a true.
Perhaps got a little bit more information on the AC 13, 34, I know you just said that you're going to give an update on the R&D day.
But.
Is it possible to narrow when the study the phase one b phase III study is going to begin you had said 2021 is there an issue with the study starting the study due to the pandemic or any other issues there.
With the start of it and just if you can give us an idea of perhaps the size of that study.
Okay.
Yeah, well, so where we are in the process of starting that study now alright. So we've had and interest in doing this over the past year working on the protocol now it's a matter of getting through the regulatory documents site selection site set up a lot of interest from investigators and that takes time independent of the pandemic just to get through all of the regulatory of her.
So we didn't put a specific we will start at the given day, but just recognize we're in process now.
From with respect to the trial design, it's the one be slashed too and that one be really as cohort driven and it really depends on how quickly we can identify the dose and theres opening cohorts and then we have a plan for a phase two expansion.
And then really the size of that will really be a function of the treatment effect and we see early on and so really I don't want to get too far ahead, because it's rich with the strategy around getting quickly to dose on PK PD and very.
Very much interested and like our strategy. So we'll tell you more about that when we get into the R&D day, but just recognize from in the process of setting that study of pounds.
Okay, great. Thank you and.
<unk> a question for you on the slide regarding the.
4 billion plus.
Annual peak sales potential and I'm just wondering if you can give us your thoughts on.
And what your assumptions are in that.
As far as the five different indications that you have listed there.
Yes. Thanks, Thanks for your question Ed.
No.
What we've previously said is that if you look at all of the indications within Mds and beta thalassemia I E. If you look at the medalist study the command study the beyond study the believe study and.
And if they are all successful and if the.
And up getting improved and on label, we believe that that has the opportunity to contribute to about half of that $4 billion plus opportunity.
Okay beyond that.
If youre looking at myelofibrosis and other potential indications that we will be exploring with our partners at BMS. We believe that the opportunity for rebel of Zelle has the has the chance to be able to make it above $4 billion plus and again, that's all predicated on the success of these lifecycle management programs and the continued successful lung.
<unk>.
Great. Thanks Habib.
Thank you Ed.
Your next question comes from Alex <unk> from Raymond James James Raymond James. Please go ahead.
Hi, This is the Alex on for Danielle Brill, Thanks for taking the question.
So just asking about the.
The phase two long term follow up data from Pulsar, we're expecting and the next few months.
What should we expect and how meaningful is the update.
And you know what new learnings from May come out of these updated results and a specific follow up will we and if so when would we expect to see of <unk>.
MPR to expression data.
Yeah. Thanks, Thanks for your question, Alex I'll hand that over to Jay Backstrom.
Cash from the initial pulse our results. It was 24 week data so with that open label, we have an opportunity to continue fall of patients up over time.
What I can tell you of which we shared previously is that of those that were eligible to get into open label, we still have over 95% of those patients still on trial and so we're continuing to follow of both for safety as well as for efficacy continue to follow of four six minute walk distance and functional class improvement et cetera. So youll.
C and additional extension and evaluation of that and which will give us some insight into not only the persistence of effect with the potential to see some patients perhaps should continue to improve on treatment and so it should be a very nice uptake from the open label extension.
And then Alex it's Ed.
Todd here, we actually had a presentation at a small conference.
Posted by the <unk> yesterday.
That looked into some of the the MTR two expression and other biomarker data that you can find on the publications section of the web to the website the.
And basically shows that.
So Todd or Sep binds active and a likely to expect as far as the mechanism goes on.
And then <unk>.
MTR two expression, whether it is not only driven by the familiar of one of the disease, but sometimes in the idiopathic form and.
The patients respond to so todd or separate regardless of having the mutation or not.
Great. Thanks.
Your next question comes from Alexandra Heller from Oppenheimer.
Hi, guys. This is Alex on for Leland. Thanks for taking my question. Kevin can you provide additional color on opex expectations for 2021 of the start up of multiple trials throughout the year.
Sure. So we don't give hard guidance on the relative to our expected spend what we are seeing however is given the increase and the number of trials specifically related to sit out of subs.
Our increased spending will begin to increase as we go forward through this year and into 'twenty and 'twenty two.
And that holds true as well with the sales and marketing area and we will be preparing a battery of area for growth as well.
The specific numbers now.
Okay. Thank you.
Your next question comes from.
And the chunk of it's from Citigroup. Please go ahead.
I haven't even came thanking for taking the questions I had two on the <unk>.
First one regarding the phase two be on trial, just would love to get your sense for expectations going into that readout and more specifically what would you consider a possible data and.
And our phase two trial look like and this population and then separately could you just comment briefly on steps youre, taking to maintain and favorable enrollment trends and commands and also and independents, which I recognize it's just getting started and light of headwinds from Covid and thank you.
Yes. Thank you I'll hand, the all of those development questions over to Jay.
Yes, so beyond was really looking to see for those patients that were not transfusion dependent and whether or not the drug would have an effect on raising hemoglobin and.
As you know from what we presented previously and our phase two data. This is a sweet spot I believe for less Pat or Sept, and its ability to raise hemoglobin. So one of the trial was designed I think we it was a randomized controlled study so hitting that endpoint I think will be significant for this group of patients who really have no option so that.
Will be nice to see that data presented and as you've talked about that will be the first half of the year.
I think of similar endpoint of if there's another trial required would look something like that because of that gain would be the main clinical issue from those folks and the anemia symptoms associated with it with respect to commands and again. This is at the EMS Operationalized study, but I can tell you having the.
The celgene portion of the BMS prior to that they are all in on this they are pushing hard they've got a lot of energy behind it.
What we've seen over the year and this I think is true for most of us running studies.
Clearly the pandemic has had an influence that the pain physicians and investigators have found a way to manage through that.
We're kind of getting into a good place quite honestly and hope from this persist as we see the.
And the potential for this to come down with vaccines going up I think all of those conditions favor those physicians being able to put patients back on trials, so they're pushing hard on that respect the independents and the <unk>.
Chile has been on protocol development last year, and they talked about getting the study up and ready and it's posted on Central's Dot Gov on my sense of this theyre getting very close to half of that study open and enrolling so same kind of push behind that program as well.
And so appreciate it thank you.
Thank you. Our next question. Your next question comes from Kennan Mackay from RBC capital markets.
Hey, Doug.
Thanks for taking the question and congrats on wrapping up 2020 on the on a strong note here.
A question on 13, 34 and <unk>.
The element that I'm wondering if the.
If anything.
And that was specifically designed into.
That agent or.
And that made it.
Exceptionally.
Potent within lung tissue pulmonary tissue or.
And whether there is applicable and others, Colorado or fibrotic.
Pieces beyond that thank you.
Yes, Thanks for your question Kevin.
And over to you on 13th of the divorce.
Yeah. So we like the profile of $13 34, because its effect on fibrosis. So just the kind of start there.
And moved into SSC ILD for a number of reasons, one of which we're obviously on the pulmonary space and like to be and pulmonary and like the skin portion of the disease, because it will give us some insight on pretty quickly as to whether or not and we're getting to biologic active dose and that may translate that into better understanding and faster decisions are on dose selection. So.
Where we stand but I have to tell you I also we haven't really talked a lot about it but as we get through that first portion and understand dose I do think we have some optionality as to where we go beyond there because it should work and other fibrotic disease areas and so we'll see where we're just getting started we got to get through that first day.
And it'll of identifying dose and finding tolerable dose that has the activities we expect that.
And I'm looking forward of getting that study open.
Your next question comes from Paul Choi from Goldman Sachs.
Hi, This is cringing and John good Paul.
Can you just talked about and can you differences and the patient population with high Purion XE and as compared to the seller and how that how you consider that was on your thinking and on appropriate trial design regarding the primary endpoint and resize link.
And the <unk>.
Total arm.
Yes, Thanks for your question, Oh, and because that's the journey as well.
Yeah, So and you can start with the easy one zenith is and functional class four and if you take a look at pulsar Paul serviced principally functional class II. Three. So this is a group of patients that quite honestly are not often included and studies and there are symptomatic of breast typically on maximal therapy and potentially for that pace.
And cohort the next thing for them might be transplant.
So if you think about that as a group, that's really and need across the spectrum very much distinguished from the pulsar data and we have a lot of enthusiasm for our investigators around getting that study open.
About Hyperion and how I would view the difference between Hyperion and pulsar is pulse or is the prevalent patient population and their disease and on therapy, and not getting to golar and not doing well the purion and we're trying to get them at the beginning of the diagnosis there early on and certain patient groups that don't get.
Relatively good control for their disease quote unquote on existing therapies are at high risk for worsening disease, and really not very good outcomes over time and as I think about the mechanism of what we believe the house the Tad or Sep works getting early in the course should be very nice we should have an opportunity to show some significant.
And that group on things like clinical worsening or other events. So earlier trial population and that's kind of how we're thinking about it.
You've heard Habib say and you know trying to manage the vision with the trials that we're running I believe at the end of the day, we've got a really nice breadth of opportunity too.
Fulfill the promise of if you will of what's the Etanercept can do across the patients.
Okay, and then just a quick follow up will you be using primarily the same sites for enrollment and then these and other studies and you are in dollar.
Well you know I think we like to go back the centers that have really done well for US right. As we think about running trials. Some centers are geared probably better for one trial and then for another we're.
And we're not exclusively using the centers that we've used before of course. We include those that want to and have shown their interest and our program and being able to find the patient and put them on Hyperion and fits in early diagnosed group likely will need some some centers that didn't participate with us and pulsar. So we'll give you a lot more detail on the size scope of those programs when we get.
Through our R&D day.
But there will be certainty overlap from the pulsar sites, but also on new sites as well.
Okay, Great. That's helpful. Thank you.
And as a reminder to ask a question press star one on your telephone.
And at this time, we have no additional questions. We have reached the end of the Q&A session I would now.
Now, let me turn the call back to Accelerant and CEO of the Daphne for closing remarks.
Yes.
Okay. Thank you operator, and I guess before we conclude I want to take the opportunity to thank everyone for joining us and you can see 2020 and.
And another great year for our company and with our clinical regulatory and commercial successes positioning us really well for the year ahead, I'm really looking forward to providing updates on a number of ongoing and planned trials and the continued success of rumblings of this year and as well of connecting with many of you.
At some upcoming virtual conference and then and obviously at the research and development day that we're planning for the middle of the year in the meantime, if you have any questions feel free to reach out to tighter Jamie and wishing everybody a great evening. Thanks, everyone.
Yes.
Ladies and gentlemen, this concludes today's conference call. Thank you for participating you may now disconnect.
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The year.
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