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Acceleron Pharma Inc (NASDAQ:XLRN)
Q4 2020 Earnings Call
Feb 25, 2021, 5:00 p.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good afternoon, ladies and gentlemen, and welcome to the Acceleron Fourth Quarter and Full Year 2020 Earnings Conference Call. [Operator Instructions] Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions]

I would now like to hand the call over to Ms. Jamie Bernard, Associate Director of Investor Relations at Acceleron. Please go ahead.

Jamie Bernard -- Associate Director, Investor Relations

Thanks, and welcome, everyone to our fourth quarter and full year 2020 earnings call. The press release reporting our financial results, in addition to the presentation for today's webcast are available on the Investors and media page of our corporate website at www.acceleronpharma.com.

Joining me on the call this afternoon are Habib Dable, our Chief Executive Officer; Kevin McLaughlin, our Chief Financial Officer; Dr. Jay Backstrom, our Head of Research and Development; Sujay Kango, our Chief Commercial Officer; and Todd James, our Senior Vice President of Corporate Affairs and Investor Relations.

As a reminder, we will be making Forward-Looking Statements regarding our financial outlook in addition to regulatory, product development and commercialization plans and research activities. These statements are subject to risks and uncertainties that may cause actual results to materially differ from those forecasted. A description of these risks can be found in our most reform 10-K on file with the SEC.

With that, I would now like to turn the call over to Habib Dable, our CEO.

Habib Dable -- President and Chief Executive Officer

Thank you, Jamie and good afternoon, everyone, and thank you all for joining us today. 2020 was a great year for Acceleron. We made significant progress and reached multiple crucial milestones across our pulmonary and hematology programs. I could not be more grateful to the entire Acceleron team for their contributions to the many successes we were able to achieve.

Reflecting on the past year, I would be remiss if I did not first acknowledge those directly affected by the pandemic, the healthcare workers and many others who continue to work tirelessly to help protect and support our communities. Like many of our peers here at Acceleron, we face multiple operational challenges as we quickly pivoted to a remote work environment in March.

I'm incredibly proud of our team's resilience and resourcefulness despite the necessary adjustments and we continue to be committed to our mission and operate with a sense of urgency for patients. We remain well positioned for a productive year ahead in support of our long-term vision to build industry-leading pulmonary and hematology franchises.

To briefly highlight our accomplishments, I will begin with our Acceleron LED pulmonary program. In early 2020, the announcement of positive top line results from the PULSAR Phase II trial established proof-of-concept for sotatercept in patients with pulmonary arterial hypertension or PAH. Following the results, we received breakthrough therapy and prime designations for sotatercept by the FDA and EMA, 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 in more detail during today's call. In addition to sotatercept, we are advancing ACE-1334, an Acceleron discovered asset, which recently received orphan drug designation from the FDA for the treatment of systemic sclerosis.

We plan to initially develop ACE-1334 in systemic sclerosis associated interstitial lung disease, or SSc-ILD. This rare pulmonary disorder affects more than 50,000 patients in the United States and Europe and is the main contributor to mortality in patients with systemic sclerosis. We are currently in the process of planning for a Phase Ib Phase II study to evaluate the activity of ACE-1334 in patients with SSC ILD and expect to start the trial in 2021.

Moving to our hematology program, alongside our partner, Bristol-Myers Squibb, we achieved multiple regulatory milestones during Reblozyl first full year of commercial availability in the United States. Including approval in MDS and continued market expansion. Reblozyl, also known as luspatercept, is the first and only erythroid maturation agent approved in the United States, Europe and most recently in Canada, for the treatment of anemia in certain blood disorders. Despite external challenges in the face of the global pandemic, our joint commercial team was able to drive early sales success, and I will highlight the program in more detail shortly.

Diving a little deeper into our pulmonary program. Following the top line results presentation from the PULSAR Phase II trial at ATS in June, we had the opportunity to share updates from PULSAR as well as the SPECTRA trial our smaller open-label Phase II trial at the American Heart Association scientific sessions in November. The PULSAR presentation highlighted 24-week echocardiogram results, which was recognized by AHA as the cardiopulmonary best abstract.

The results showed that sotatercept improved right ventricular function, these data are very encouraging as right ventricular failure is a hallmark of PAH and a key determinant of a patient survival. The presentation from the ongoing SPECTRA trial showed treatment with sotatercept resulted in substantial improvements in 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 sotatercept's unique mechanism of action has the potential to reverse pulmonary vascular remodeling.

Turning now to our Phase III clinical development plan and vision for sotatercept. At the end of the year, we initiated our Phase III registrational trial STELLAR. And as a reminder, STELLAR is a Phase III randomized, double-blind, placebo-controlled study comparing the efficacy and safety of sotatercept versus placebo in approximately 284 patients diagnosed with PAH with Functional Class II or III. The primary endpoint is the change in 6-minute walk distance at 24 weeks compared to baseline.

We are also preparing to conduct two additional Phase III trials, HYPERION to explore sotatercept in the early stage intervention setting and ZENITH in patients with who functional class IV PAH. Results from our Phase III studies will add to our understanding of sotatercept's unique mechanism and support its potential to be a backbone therapy for patients with all stages of PAH.

Moving to hematology. 2020 was a successful first year for REBLOZYL. Our partner, BMS, reported approximately $274 million and $115 million in net sales of REBLOZYL for the full year and fourth quarter, respectively. While it is still early in the commercial launch of REBLOZYL, we are pleased with the market penetration at our sales team, along with our global collaboration partners at BMS have achieved.

Specific to the MDS patient population, where there had not been a new drug approval in over a decade, a large portion of early sales were driven by pent-up demand from patients who are experiencing inadequate responses to standard of care, or who were suffering from high transfusion burden.

And on our third quarter earnings call, we described the start of a shift in demand to patients earlier in their MDS journey. We are continuing to observe this ongoing shift in the type of patients treated with REBLOZYL, which we expect to contribute to potential short-term moderation of growth or flattening of sales in the first quarter. The commercial teams are focused on achieving further penetration into the longer-term underlying demand of patients earlier in their MDS journey, along with optimal dose of REBLOZYL to maximize patient benefit and increased duration of treatment in this population to drive growth in 2021 and beyond.

Physicians' feedback remains positive with significant awareness of the brand as we continue to add prescribing accounts in the US. In addition, we are still in the very early stages of the launch of REBLOZYL outside of the US. In the second half of 2020, and BMS launched REBLOZYL in Austria and Germany, and we anticipate additional launches in various markets globally over the course of 2021 as they receive reimbursement. We also continue to monitor for any potential effects that COVID-19 have on sales or promotional activities in all regions.

As we think about the long-term vision for the brand, our partner BMS continues to advance the development of REBLOZYL for the treatment of additional patient populations, struggling with anemia associated blood disorders. There are currently three trials under way to further investigate luspatercept effect on patients with first-line lower-risk MDS non-transfusion-dependent beta-thalassemia and myelofibrosis. BMS is in the process of initiating the independence trial in patients with myelofibrosis who are on a JAK-2 inhibitor and require red blood cell transfusion.

And we look forward to the presentation of results from the BEYOND trial in beta-thalassemia in the first half of 2021 and the COMMANDS trial in MBS in 2022 or later. All in, if we continue on this successful trajectory with these current and potential future applications, we estimate annual peak sales of REBLOZYL 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 will be available to take your questions. Kevin?

Kevin McLaughlin -- Chief Financial Officer

Thanks, Habib. Good afternoon, everyone. I would 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 in cash, cash equivalents and investments.

Revenue for the fourth quarter of 2020 was $25.9 million, which includes $2.9 million of cost share revenue and $23 million of royalty revenue from net sales of REBLOZYL. Revenue for the full year was $92.5 million, which includes $12.7 million of cost share revenue, $54.8 million of royalty revenue from net sales of REBLOZYL and the recognition of a $25 million regulatory milestone from the approval of REBLOZYL in Europe. All revenue was derived from the company's partnership with Bristol-Myers Squibb.

Total costs and expenses for the year were $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 would like to take a moment to echo Habib's sentiments and say how proud I am of our team's ability to execute throughout 2020.

With that, I would like to open up the call to questions. Operator?

Questions and Answers:

Operator

[Operator Instructions] Your first question comes from Yaron Werber from Cowen. Please go ahead.

Yaron Werber -- Cowen -- Analyst

Yeah. Hi, team and congrats. And thanks for taking the question. Really great to see a good launch in this environment. So a couple of questions I have on REBLOZYL, maybe to touch first, Habib, you mentioned you are expecting maybe a flattening of sales in Q1. Is that partially due to the donut hole or is there anything specific from Q4 to Q1 or is that just ongoing kind of the journey shift? And then what drives the reacceleration in the US? That is first question. And then second, what is the cadence of, let's say, new countries coming on board in second half -- I'm sorry, this year in Europe? Thank you.

Habib Dable -- President and Chief Executive Officer

Yeah. Thank you for your question, Yaron. So with respect to the commentary in terms of moderation of sales growth or even perhaps flattening, it is really not much different than what we had said on the third quarter earnings call, where at that time, Yaron, we described a start in this shift in demand of patients to earlier in their MDS journey. And we are continuing to observe this ongoing shift in that type of patient that's being treated with REBLOZYL. And we expect this shift to contribute to a potential short-term moderation or even flattening in the first quarter.

Now in the meantime, the commercial teams continue to be focused on achieving further penetration, going -- managing the long-term underlying demand that are in capturing these patients that are earlier in their MDS journey and then also focusing on the optimal dose of Reblozyl, and all in all, to maximize the potential benefit and the persistency, i.e., the increased duration of treatment with this population, which we believe is going to drive significant growth once we burn through this pent-up demand.

And when you think about the investments that BMS is making in REBLOZYL in terms of life cycle management, the continued high awareness and early feedback that we are getting in terms of how doctors and patients are experiencing early days with REBLOZYL, we have high confidence in the success for future growth and potentially further indications through life cycle management. And as a result of that, together, BMS and Acceleron just recently had increased guidance for peak sales potential of over $4 billion, if indeed we are successful with those life cycle programs.

With respect to the cadence, we haven't really given specific details. Obviously, BMS is responsible for the commercialization outside of the US. We have already talked about the approval in Austria and in Germany, which has already contributed to sales in 2020. But what you can expect is probably a significant uptick in those countries that are getting reimbursement in the second half of 2021.

Yaron Werber -- Cowen -- Analyst

And just a final question. The BEYOND study, we are expecting data initially sort of late last year and then early this year, and it looks like it is first half in non-transfusion dependent. Just give us a sense that there seems to be a slight shift, what was driving that? Thank you.

Habib Dable -- President and Chief Executive Officer

Yeah. So I think last year, we said that it is going to be the first half of this year. We haven't changed the guidance. We are keeping that to the -- you can expect to see that data in the first half of 2021, and you can probably estimate respect congresses that would be the most suitable for that. And so we are sticking to that Yaron.

Yaron Werber -- Cowen -- Analyst

Great. Thank you.

Habib Dable -- President and Chief Executive Officer

Yeah. Thanks for your questions.

Operator

Your next question comes from Carter Gould from Barclays. Please go ahead.

Carter Gould -- Barclays -- Analyst

Great. Good afternoon, guys. Thanks for taking the question. I guess, first, Habib, now that we're coming up sort of approaching a year since the MDS launch, maybe just initial kind of sense on how duration and persistence is sort of playing out? And then maybe just as a quick follow-up. As we think about some of those OUS launches, why wouldn't we expect the same sort of bolus effect that we saw in the US to play out on a country-by-country basis? Thank you.

Habib Dable -- President and Chief Executive Officer

Yeah. Thanks, Carter. Really insightful questions, specifically the second one. And when we talk about the commentary regarding a potential flattening or moderation of sales, that is in the markets that we've already launched such as the US. And so the pattern that we're referring to, you're right, is country specific. And so if you were to look at country-by-country, there's absolutely no reason to believe that different markets would have a different pattern. So that's a very fair comment.

With respect to the -- your question on persistency, it actually is probably the most underlying factor behind the commentary and this trajectory, which is very, very much expected in a market where there's been very little innovation over the last decade or so and where the pent-up demand is very, very high. And as you would remember, from the clinical trial, what we saw was patients seeing a 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 in patients who had a red blood cell transfusion burden of 6 units or less every eight weeks versus those that were greater. And so what happens when you get a disproportionate amount of patients that are early in the launch that are actually -- have a higher transfusion burden, you would expect that the persistency to be lower than what you would have seen in the clinical trial when the average was 6.

And so that is a direct result of this pent-up demand. And as we continue to transition into the patients that are earlier in their patient journey, we expect to continue to see persistency increase and as well as we continue to educate the patient -- the treater group on the appropriate dosing schedule, which will also enhance efficacy and persistency.

Carter Gould -- Barclays -- Analyst

Thank you.

Operator

Your next question comes from Chris Raymond from Piper Sandler. Please go ahead.

Nicole Gabreski -- Piper Sandler -- Analyst

Hi. This is Nicole Gabreski on for Chris. Thanks for taking the question. Maybe just one on the pipeline for 1334 and SSc-ILD. I know you guys have noted that there are about 50,000 patients in the US and EU. And as we think about the market opportunity, are there certain subsets of these patients that might respond better to therapy like 1334, I guess, be it early versus late onset ILD or autoantibody status? And how does that frame how you're thinking about the design of the Phase Ib/II? Any color here would be great.

Habib Dable -- President and Chief Executive Officer

Yeah. Thanks for your question, Nicole. I think what I'll do is I'll pass that one on to Jay Backstrom, our Head of Research and Development. Jay?

Jay T. Backstrom -- Executive Vice President, Head of Research and Development

Yeah. Hi. This is Jay. So for our Ib/II, 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 do. And 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, etc., that would allow us then to move into Phase II. And we have some ideas around what good patients would look like.

But in that opening study, there's a lot of objectives that we're trying to achieve, and we'll -- as we go and learn more about it. As Habib had mentioned previously, we do plan to do an R&D Day, and I would invite you to join us when we get that scheduled because we'll take a much deeper dive into both the protocol and our thinking around biomarker strategy selections.

Nicole Gabreski -- Piper Sandler -- Analyst

Thank you.

Operator

Your next question comes from Geoffrey Porges from SVB Leerink.

Na Sun -- SVB Leerink -- Analyst

Hey, guys. This is Na Sun on for Geoff. So two questions from me. The first would be what is the latest information about enrollment into the frontline MDS trial with REBLOZYL? And how confident are you that REBLOZYL will be superior to PSA in an ESA naive population. And the follow-up to that is, what is the margin of benefit required for statistical and commercial success, given the large price difference between ESA and REBLOZYL? Thanks.

Habib Dable -- President and Chief Executive Officer

Yeah. Thanks for your questions. So just to remind you, the COMMANDS trial is 100% operationalized by our partners at BMS. 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 some time in 2022 or later. So that's the first thing.

With respect to our confidence in terms of what has informed us to move forward into the COMMANDS study, that's really multifactorial based on what we have seen from some of the company sponsored Phase III results with ESA as well as what we've seen with our own Phase II data with the [Indecipherable] 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 to make the move forward into another registrational study like COMMANDS and perhaps talk a little bit about what that margin benefit that we saw at least in Phase II look like.

Jay T. Backstrom -- Executive Vice President, Head of Research and Development

Yeah. So I think, Habib, you summed it up well. From the data that we have for patients that are requiring transfusions at the level that just remind that it is patients that still require some transfusions to get in. We're looking at those that potentially have an EPO level above 200. That group doesn't do well in an ESA. And frankly, it's a superiority study that we really have great confidence in. So looking forward to getting that trial completed. As Habib mentioned, the BMS team is really putting a lot of effort behind it. So I think from what I see today, the time lines that we've proposed, I think, should stay on track, and we'll see as we continue to go through the year.

Habib Dable -- President and Chief Executive Officer

Yeah. And at the end of the day, all we have is our own data that we've looked at with our own Phase II and looking at other company sponsored data sets. Obviously, we cannot make cross study comparisons. And that's why we have designed a superiority design study. And that if indeed we are successful, we will be able to demonstrate and show that benefit.

Na Sun -- SVB Leerink -- Analyst

Thank you.

Habib Dable -- President and Chief Executive Officer

Thanks for your questions.

Operator

Your next question comes from Eric Joseph from JPMorgan. Please go ahead.

Eric Joseph -- JPMorgan -- Analyst

Hi, guys. Thanks for taking the questions. Good afternoon. I guess just on trend so far with REBLOZYL, can you just comment on demand that you're seeing in -- or uptake that you're seeing in RS-negative patients, whether sort of -- I guess, how efficacy has emerged in that patient population? And then is there any patient -- sorry, payor pushback?

And I guess just picking up on your comments, Habib, about life cycle management opportunities that you and BMS are thinking about, can you just elaborate there on some additional indications that you're pursuing? Is it kind of going vertical within MDS or myelofibrosis or horizontal and other anemia-related indications that you're thinking about? Thanks.

Habib Dable -- President and Chief Executive Officer

Yeah. Thanks for your questions, Eric. So I'll let Jay answer the questions regarding life cycle management in terms of what's ongoing and how we're thinking about things with our partners. But with respect to your question on RS negative patients, really, our insights there are from our own Phase II study. In terms of in the market, we have no reason to believe that there's any substantial use in the RS negative population. Obviously, the label is 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.

Jay T. Backstrom -- Executive Vice President, Head of Research and Development

Yeah. So with respect to life cycle plan, you can anticipate seeing kind of additional investigation within the MDS space. As you know already, there's been discussion and out in clinicaltrials.gov for combination strategies within the MDS space. So I think you'll continue to see more of that. There's obviously clearly an interest in the RS negative patients as of course, Habib mentioned, we had nice activity in our Phase II data that is included in COMMANDS. So we can anticipate to see that.

So I think combination will probably be the way to go in addition to us advancing in the earlier lines of therapy. And then build around beta-thalassemia because it really has turned out to be a really important therapy for patients. And so if you think about thalassemia, you can anticipate potentially alpha thalassemia around it. So there will be continued expansion around these areas. But there's a lot of interest in the product, and so more to come on that.

Eric Joseph -- JPMorgan -- Analyst

Okay. Great. Thanks for taking the questions.

Habib Dable -- President and Chief Executive Officer

Thank you, Eric.

Operator

Your next question comes from Ed White from H.C. Wainwright.

Eric Joseph -- JPMorgan -- Analyst

Good afternoon. Thanks for taking my questions. So just to perhaps get a little bit more information on ACE-1334. 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 Ib, Phase II study is going to begin? You've said 2021. Is there an issue with starting this 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?

Habib Dable -- President and Chief Executive Officer

Jay?

Jay T. Backstrom -- Executive Vice President, Head of Research and Development

Yeah. Well, so we're in the process of starting that study now, all right? So we've had an interest in doing this over the past year, working on the protocol. Now it's a matter of getting to the regulatory document, site selection, site set up. A lot of interest from our investigators. And that takes time independent of a pandemic just to get through all the regulatory hurdles. So we didn't put a specific -- we will start at a given date, but just recognize we're in process now.

From -- with respect to the trial design, it's a Ib/II and that Ib really is cohort driven, and it really depends on how quickly we can identify the dose in those opening cohorts, and then we have a plan for our Phase II expansion. Really the size of that will really be a function of the treatment effect that we see early on. So I really don't want to get too far ahead because it's rich with a strategy around getting quickly to dose on PK/PD, 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 we're in the process of setting that study up in-house.

Ed White -- H.C. Wainwright -- Analyst

Okay. Great. Thank you. And Habib, a question for you on the slide regarding the $4 billion plus annual peak sales potential. I'm just wondering if you can give us your thoughts on what your assumptions are in that as far as the five different indications that you have listed there?

Habib Dable -- President and Chief Executive Officer

Yeah. Thanks for your question, Ed. So 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 COMMANDS study, the BEYOND study, the BELIEVE study, and if they are all successful and if end up getting approved and on-label, we believe that, that has the opportunity to contribute to about half of that $4 billion plus opportunity, OK?

Beyond that, if you're looking at myelofibrosis and other potential indications that we will be exploring with our partners at BMS, we believe that the opportunity for REBLOZYL has a chance to be able to make it above $4 billion plus. And again, that's all predicated on the success of these life cycle management programs and the continued successful launch.

Ed White -- H.C. Wainwright -- Analyst

Great. Thanks, Habib.

Habib Dable -- President and Chief Executive Officer

Thank you, Ed.

Operator

Your next question comes from Alex Nackenoff from Raymond James. Please go ahead.

Alex Nackenoff -- Raymond James -- Analyst

Hi. This is Alex on for Danielle Brill. Thanks for taking our question. So just asking about the Phase II long-term follow-up data from PULSAR we're expecting in the next few months. What should we expect? And how meaningful is this update and what new learnings may come out of these updated results? And a specific follow-up, will we -- and if so, when would we expect to see BMPR2 expression data? Thanks.

Habib Dable -- President and Chief Executive Officer

Yeah. Thanks for your question, Alex. So I'll hand that over to Jay Backstrom.

Jay T. Backstrom -- Executive Vice President, Head of Research and Development

Yeah. No. From the initial PULSAR results, it was 24-week data. So with that open-label, we have an opportunity to continue to follow patients up over time. What I can tell you, which we've 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 both for safety as well as for efficacy, continue to follow for 6-minute walk distance and functional class improvement, etc. So you'll see an additional extension and evaluation of that, which will give us some insight into not only the persistence of effect, but the potential to see some patients perhaps continue to improve on treatment. So it should be a very nice update from the open-label extension.

Todd James -- Senior Vice President, Corporate Affairs and Investor Relations

And then, Alex, it's Todd here. We actually had a presentation at a small conference hosted by the PVRI yesterday that looked into some BMPR2 expression and other biomarker data that you can find in the publication section of the website that basically shows that sotatercept binds activin A, like you to expect as far as the mechanism goes and then BMPR2 expression, whether it is not only driven by the familial form of the disease, but sometimes in the idiopathic form and patients respond to sotatercept regardless of having the mutation or not.

Alex Nackenoff -- Raymond James -- Analyst

Great. Thanks.

Operator

Your next question comes from Alexandra Heller from Oppenheimer.

Alexandra Heller -- Oppenheimer -- Analyst

Hi, guys. This is Alex on for Leland. Kevin, can you provide additional color on opex expectations for 2021 as the start of multiple trials throughout the year?

Kevin McLaughlin -- Chief Financial Officer

Sure. So we don't give hard guidance on -- relative to our expected spend. What we are seeing, however, is given the increase in the number of trials specifically related to sotatercept, we -- our increased spending will begin to increase as we go forward through this year and into 2022. That holds true as well with the sales and marketing area, and we will be preparing that area for growth as well. But specific numbers, no.

Alexandra Heller -- Oppenheimer -- Analyst

Thank you.

Operator

Your next question comes from Yigal Nochomovitz from Citigroup.

Yigal Nochomovitz -- Citigroup -- Analyst

Hi, Habib and team. Thank you taking the question. I had two. The first one, regarding the Phase II BEYOND trial, just would love to get your sense for expectations going into that readout? And more specifically, what would you consider positive data? And what would a Phase III trial look like in this population? And then separately, could you just comment briefly on steps you're taking to maintain favorable enrollment trends in COMMANDS and also in INDEPENDENCE, which I recognize is just getting started, in light of headwinds from COVID? Thank you.

Habib Dable -- President and Chief Executive Officer

Yeah. Thanks, Yigal. I'll hand all those development questions over to Jay.

Jay T. Backstrom -- Executive Vice President, Head of Research and Development

Yeah. So BEYOND was really looking to see for those patients that were not transfusion dependent, whether or not the drug would have an effect on raising hemoglobin. And as you know, from what we presented previously in our Phase II data, this is a sweet spot, I believe, for luspatercept and its ability to raise hemoglobin. So the way the trial was designed, I think 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. As you talk about, that will be the first half of the year. I think a similar endpoint if there's another trial required would look something like that because that, again, would be the main clinical issue for these folks is the anemia and the symptoms associated with it.

With respect to COMMANDS, again, this is a BMS operationalized study, but I can tell you, having been with the Celgene portion of the BMS prior to that, they're all in on this. They're pushing hard. They've got a lot of energy behind it. I think 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 physicians and investigators have found a way to manage through that.

I think we're kind of getting into a good place, quite honestly. And hopefully, this persists as we see 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 trial. So they're pushing hard on that.

With respect to INDEPENDENCE, the trial has been on protocol development last year, and they talked about getting the study up and ready. It's posted on clintrials.gov. From my sense of this, they're getting very close to have that study open and enrolling. So same kind of push behind that program as well.

Yigal Nochomovitz -- Citigroup -- Analyst

Thanks, Jay. Appreciate it. Thank you.

Habib Dable -- President and Chief Executive Officer

Thanks, Yigal.

Operator

Your next question comes from Kennen MacKay from RBC Capital Markets.

Kennen MacKay -- RBC Capital Markets -- Analyst

Thanks for taking the question and congrats on wrapping up 2020 on a strong note here. Maybe a question on 1334 and the development there. Wondering if there is anything that was specifically designed into that agent or molecule that made it exceptionally potent within lung tissue -- pulmonary tissue or whether there's applicability in other sclerotic or fibrotic diseases beyond that? Thank you.

Habib Dable -- President and Chief Executive Officer

Yeah. Thanks for your question, Kennen. Jay, over to you on 1334.

Jay T. Backstrom -- Executive Vice President, Head of Research and Development

Yeah. So we like the profile of 1334 because it's effect on fibrosis. So just to kind of start there. And moved into SSc-ILD for a number of reasons, one of which we're obviously in the pulmonary space or like to be in pulmonary. I like the skin portion of the disease because it will give us some insight pretty quickly as to whether or not we're getting to biologic active dose, and that may translate then into better understanding and faster decisions around dose selection. So that's 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 in other fibrotic disease areas. So we'll see. We're just getting started. We got to get through that first hurdle of identifying dose and finding tolerable dose that has the activity that we expect. But I'm looking forward to getting that study open.

Operator

Your next question comes from Paul Choi from Goldman Sachs.

Unidentified Participant

This is [Indecipherable] on for Paul. Can you just talk about key differences in the patient populations with HYPERION and ZENITH compared to STELLAR? And how about -- how you consider those when you're thinking about appropriate trial designs with regard to primary endpoint study, length and the control arm?

Habib Dable -- President and Chief Executive Officer

Yeah. Thanks for your question. I'll give back to Jay as well.

Jay T. Backstrom -- Executive Vice President, Head of Research and Development

Yeah. So maybe I can start with the easy one. ZENITH is in functional class IV. And if you take a look at PULSAR, PULSAR was principally functional class II, III. So this is a group of patients that, quite honestly, are not often included in studies. There are symptomatic [Indecipherable] typically on maximal therapy. And potentially for that patient cohort, the next thing for them might be transplant. So if you think about that as a group that's really in 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.

When I think about HYPERION and how I would view the difference between HYPERION and PULSAR, PULSAR is a prevalent patient population, had their disease, been on therapy and not getting to goal or not doing well. HYPERION, we're trying to get them at the beginning of their diagnosis. They're early on in certain patient groups that don't get relatively good control for their disease "on existing therapies". They're 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, how sotatercept works, getting early in the course should be very nice. We should have an opportunity to show some significant improvement in that group on things like clinical worsening or other events. So earlier trial population, that's kind of how we're thinking about it. You've heard Habib say and we're 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 to fulfill the promise, if you will, of what sotatercept can do across the patients.

Unidentified Participant

All right. And then just a quick follow-up. Will you be using primarily the same sites for enrollment in these other studies as you are in STELLAR?

Jay T. Backstrom -- Executive Vice President, Head of Research and Development

Well, I think we like to go back to centers that have really done well for us, right, as we think about running trials. Some centers are geared probably better for one trial than for another. 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 in our program and being able to find the patients to put them on. HYPERION, if it's an early diagnosed group, likely will need some centers that didn't participate with us in PULSAR. So we'll give you a lot more detail on the size, scope of those programs when we get to our R&D day. But there will be certainly overlap from the PULSAR sites, but also new sites as well.

Unidentified Participant

Great. That's helpful. Thank you.

Operator

[Operator Instructions] And at this time, we have no additional questions. We have reached the end of the Q&A session. I would now like to turn the call back to Acceleron's CEO, Habib Dable, for closing remarks.

Habib Dable -- President and Chief Executive Officer

Okay. Thank you, operator. And I guess, before we conclude, I want to take the opportunity to thank everyone for joining us. You can see 2020 has been another great year for our company 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 REBLOZYL this year and as well as connecting with many of you at some upcoming virtual conference 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 Todd or Jamie, and wishing everybody a great evening. Thanks, everyone.

Operator

[Operator Closing Remarks]

Duration: 41 minutes

Call participants:

Jamie Bernard -- Associate Director, Investor Relations

Habib Dable -- President and Chief Executive Officer

Kevin McLaughlin -- Chief Financial Officer

Jay T. Backstrom -- Executive Vice President, Head of Research and Development

Todd James -- Senior Vice President, Corporate Affairs and Investor Relations

Yaron Werber -- Cowen -- Analyst

Carter Gould -- Barclays -- Analyst

Nicole Gabreski -- Piper Sandler -- Analyst

Na Sun -- SVB Leerink -- Analyst

Eric Joseph -- JPMorgan -- Analyst

Ed White -- H.C. Wainwright -- Analyst

Alex Nackenoff -- Raymond James -- Analyst

Alexandra Heller -- Oppenheimer -- Analyst

Yigal Nochomovitz -- Citigroup -- Analyst

Kennen MacKay -- RBC Capital Markets -- Analyst

Unidentified Participant

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