Good afternoon, ladies and gentlemen and welcome to the Acceleron Third Quarter 2020 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. As a reminder, this conference call is being recorded. I would now like to hand the conference call to Ms. Jamie Bernard, Associate Director of Investor Relations at Acceleron. Please go ahead.

Thanks, and welcome everyone to our third quarter 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 recent Form 10-Q and 10-K on file with the SEC. With that, I would now like to turn the call over to Habib Dable, our CEO.

Thank you, Jamie, and good afternoon, and thank you all for joining us today. I hope that everyone remains safe and well during this challenging time. Acceleron continues to operate and execute toward our corporate objectives, while taking the utmost caution to limit risk for all of our stakeholders, including patients and our employees. I could not be more proud of our team's resilience, productivity and collaboration during this period. We have continued to execute commercially and operationally, with tremendous progress on the clinical and regulatory fronts, even with the majority of the team working remotely. Above all, we remain committed to our collective mission of serving patients. Beginning with our hematology program, along with our collaboration partner Bristol Myers Squibb, we continue to expand the availability of REBLOZYL, with multiple new regional approvals across the globe. Most recently, Health Canada approved REBLOZYL for the treatment of adult patients with red blood cell transfusion-dependent anemia associated with beta-thalassemia. With this approval, REBLOZYL, also known as luspatercept, became the first and only medicine of its class approved for patients in Canada. Beyond Canada, REBLOZYL achieved several regulatory milestones in the second quarter with the FDA and European Commission and is now approved for both beta-thalassemia and MDS indications here in the United States and in Europe. Outside of the North America co-promote territory, Bristol Myers is responsible for the commercialization of REBLOZYL in the rest of the world. They recently launched REBLOZYL in Austria and Germany and we anticipate other European countries to follow suit over the next six to 12 months, as the product gains expanded reimbursement and access on a country-by-country basis. Turning over to the commercial launch. I want to recognize the joint commercial team's incredible resourcefulness in the face of current challenges caused by the pandemic. Their implementation of a virtual sales promotion and education model has driven early sales success. We are encouraged by the early launch trajectory of REBLOZYL and its adoption for the treatment of anemia in adult patients with beta-thalassemia or lower-risk MDS, with ring sideroblasts who require regular red blood cell transfusions. As you can see on this slide, the net REBLOZYL sales reported by our partner Bristol Myers and Acceleron's associated royalty revenues, continue to grow quarter-over-quarter through the early stages of the commercial launch. For the third quarter, Acceleron recognized approximately $19.3 million in royalty revenue from net REBLOZYL sales of approximately $96 million. This compares with approximately $11.1 million in royalty revenues from approximately $55 million of net REBLOZYL sales in the second quarter. Prior to REBLOZYL's approval by the FDA in MDS, the physician and patient community has been anxiously awaiting a new treatment option for almost 10 years. Naturally, this dynamic resulted in a significant proportion of early uptake, being driven by pent-up demand from a group of prevalent patients, who had either previously failed or had inadequate response to ESAs and had higher transfusion burden. Currently and moving forward, we expect REBLOZYL demand to be driven primarily by patients earlier in their MDS journey, who are failing or having inadequate response to an ESA within two to three months and experiencing an increase in transfusion burden. We believe this will moderate relative growth for the fourth quarter, as we close out a successful first year for REBLOZYL and will provide steady growth from new patient starts and increased duration of treatment in 2021 and beyond. We also continue to monitor for any potential effects that COVID-19 may have on sales or promotional activities, especially in regions that may see a rise in cases and hospitalization. In parallel with these important regulatory and commercial milestones, we continue to expand our clinical development activities of REBLOZYL to patients with anemia-associated with other blood disorders, who may benefit from REBLOZYL's novel mechanism of action. As you'll recall, Bristol is currently conducting two additional studies with luspatercept and preparing for a third called INDEPENDENCE for the treatment of anemia in patients with myelofibrosis, a cancer-like blood disorder. We are looking forward to productive years ahead, as we continue to build the luspatercept franchise, with plans to report top line results in the BEYOND study in non-transfusion-dependent beta-thalassemia at year-end or in early 2021 and from the COMMANDS study in first-line lower-risk MDS in 2022 or later. Beyond these three trials, our partner, Bristol Myers Squibb recently amended its Phase III trial in myelofibrosis with INREBIC to include a safety cohort in combination with luspatercept. Adding to this, a new investigator-sponsored trial was recently initiated to evaluate luspatercept treatment in combination with lenalidomide in patients with lower-risk MDS. The outcomes of these trials will provide greater insight into luspatercept's potential safety and efficacy in combination settings and potentially broaden our understanding of its applicability for additional patients suffering from chronic anemia associated with myelofibrosis or MDS. To this point, the joint teams are actively evaluating additional disease areas, in which patients with anemia could potentially benefit from our erythroid maturation agent. If we are successful across all current and future diseases associated with anemia, our long-term estimate for peak annual sales of REBLOZYL continues to be more than $3 billion. In addition to our many recent achievements in the REBLOZYL program, 2020 has been an important year for Acceleron in pulmonary arterial hypertension, or PAH, where we have continued to make considerable progress, following the positive top line results from the PULSAR Phase II trial of sotatercept. Last month, many of you were able to join us for our webcast, in which we highlighted the trial design for our upcoming registrational Phase III sotatercept trial called STELLAR. To briefly review, STELLAR is a Phase III randomized double-blind placebo-controlled study to compare the efficacy and safety of sotatercept versus placebo. Similar to our PULSAR proof-of-concept study, eligible participants will be adult patients with WHO classified Group 1 PAH who are functional Class II or III; and unstable PAH background therapy, including mono, double or triple combination therapies. We remain on track to initiate this trial before the end of this year. In addition to STELLAR, we expect to initiate two additional Phase three trials, HYPERION and ZENITH in the middle of next year, with the long-term vision of establishing sotatercept's applicability in early and late-stage therapeutic strategies respectively. We believe the overall development plan will generate a breadth of data to sotatercept's potential as a future backbone therapy in PAH. While we are excited about our Phase three program for sotatercept, our ongoing PULSAR Phase two trial, continues to generate additional important data for analysis. At the upcoming virtual 2020 American Heart Association Scientific Sessions taking place from November 13th to the 17th, we will be presenting 24-week echocardiographic data showing the potential impact of sotatercept on right ventricular function. Also at AHA, the principal investigator of the SPECTRA Phase two trial will present preliminary interim results from the first set of trial participants. We plan to host an investor and analyst call during the conference to review these presentations and look forward to sharing details from the call soon. Finally, we expect to announce results from the open-label extension portion of the PULSAR trial, as well as additional results from SPECTRA during the first half of 2021. 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?

Thanks, Habib. Good afternoon everyone. I'd like to refer to our press release issued earlier today for a summary of our financial results for the third quarter of 2020, and take this opportunity to briefly review a few items. We ended the third quarter with approximately $887.4 million in cash, cash equivalents and investments. Revenue for the third quarter was $22.6 million, which included $3.3 million of cost share revenue and as Habib mentioned earlier, approximately $19.3 million of royalty revenue from net REBLOZYL sales of approximately $96 million. All revenue was derived from the company's partnership with Bristol Myers Squibb. The company's net loss for the third quarter was $39.2 million. With that, I'd like to open the call to questions. Operator?

Thank you. Our first question comes from Yaron Werber with Cowen. You may proceed with your question.

Hi, this is Gabe on for Yaron. Thanks for taking my questions and congratulations on another great quarter. First, I just wanted to note, whether you could share any insight into the U.S. prescription trends for REBLOZYL in terms of new and repeat orders by accounts, as you've kind of you shared in previous calls? And then, just a quick question to help kind of better understand the additional Phase three trials for sotatercept for label expansion. You mentioned early and late-stage interventions. I'm just curious, how those populations might be different than what's included in STELLAR. Because for example, I would think, add on to mono or double therapy could be fairly early in someone's diagnosis, if you're being additive let's say double therapy or being added to triple therapy could be somewhat late in someone diagnosis? So, how would those two studies really expand on what you might actually already be able to see from STELLAR? Thank you.

Yes. Thanks for your question, Gabe. So, with respect to your first question, in terms of net prescription trends and the persistency and repeat orders, perhaps Sujay Kango, our Chief Commercial Officer can touch that and then maybe, perhaps hand it over to Jay for the second question, Gabe.

Thanks, Habib. And with regards to the color with REBLOZYL prescriptions right, as we have sort of have commented in the past, we continue to see both the accounts that have prescribed in the past continue to prescribe, meaning that we're getting repeat orders from them. And then, we are continuing to add new accounts on a weekly basis as well. Now, the challenges for me to give you a specific as to breaking down one versus the other, we just kind of have a large proportion of repeat accounts that come in. And every week, we are adding in new accounts as well. So, the trend is on a positive direction and we've penetrated, as we've identified fast starts or high-potential accounts pretty much majority of the COEs or centers of excellence for MDS are prescribing -- rather I should say or ordering the medicine, as well as some of the faster accounts that we have high potential. They are -- majority of them are ordering the medicine. So, we feel pretty confident that we are getting a good chunk of our prescriber base already adopting the medicine for REBLOZYL prescriptions okay?

This is Jay. Just to go to your question on the clinical trials, we went through all of the STELLAR details on our last month. Deliberately didn't go into the details of the other two studies, the HYPERION and ZENITH. Although just to say that, they will be complementary. I think your point about being careful not to have overlapping populations is fair. But as we have these designed the trials, they will be complementary. We intend to describe those later next year.

Thank you. Our next question comes from Martin Auster with Credit Suisse. You may proceed with your question.

This is Thomas on for Marty. Thanks for taking the question. I guess a few on REBLOZYL. Curious, if you can provide any more color around this bolus effect that you're seeing. What gives you confidence that growth will kind of moderate going into Q4? And then, any color you can provide around there or on the kind of rate of moderation or how we should think about growth going into next quarter? And then, curious, if you can comment, a follow on the impact of EU sales this quarter and how we should think about that going forward over the next few quarters as well? Thanks.

Yes. So, Thomas, it's a really good question. This is Habib. Maybe, I'll take that first question with respect to the commentary on moderation. And then maybe, Sujay if you want to add anything on some of the very, very early metrics in Europe then, I'll hand it over to you. But I guess just to start again the REBLOZYL launch in our opinion is off to a very, very strong start. As you've heard from Sujay, customer feedback has been positive. Awareness levels have been high. You heard from our partners at Bristol Myers this morning, in terms of the robustness of the uptake. So, we're very, very pleased with the uptake so far. And in the long term, we continue to believe that we are on track to build a multi-billion dollar brand for the treatment of anemia. Now in the short term though, and very specific to your question on the fourth quarter, there are a few factors that I think everyone should appreciate, when they're thinking about estimates and expectations, especially coming off a quarter-over-quarter growth of approximately 75%. The first is really what I described earlier in the call and it's really the transition in new patient starts based on MDS patient profile. Following the approval in April, we were experiencing pretty significant growth from a pent-up demand from a group of prevalent lower-risk MDS patients. And I think we're all very, very pleased with that update. But today and going forward we believe that the true growth is going to be driven primarily by patients earlier in their MDS journey. And this will obviously lead to improvement in the duration of clinical benefit and treatment as we continue to penetrate the market of patients who are failing or having inadequate responses to ESAs within two or three months and that we're experiencing an increase in transfusion burden. Now also specific to the fourth quarter and this probably comes as no surprise to anybody but November and December include several holidays that will obviously reduce the total number of selling days relative to the third quarter. And lastly, we're experiencing here in the U.S. and throughout many parts of the world, a surge in COVID cases and hospitalizations that we expect would potentially decrease in-office visits and treatment compliance until cases start to reduce significantly. And as such you put all this together, we believe that this can contribute to moderation or a flattening in revenue for the fourth quarter versus Q3. But again we continue to drive new patient starts earlier in the MDS journey, and we feel that that will be the foundation of our long-term growth trajectory in forming an asset, which I -- we still believe will be over $3 billion once -- if all of the indication and life cycle management programs are successful.

Sujay, Europe I mean it's really early days I guess with respect to some of the numbers, but is there anything that you can share? I know you can -- I think the Bristol actually parsed out the $96 million I believe it was $92 million and $4 million for ex-U.S. sales but perhaps you can add some incremental color?

Yes. No thank you, Habib, and thanks Thomas. So the one -- only one additional thing I can add to is, which I was alluding to earlier with regards to the continued moderation of the growth, right? It's the fact that we are seeing new accounts come on board. And the new accounts coming on board, we continue to basically look at the fact that these are newer patient starts and that's what gives us confidence that there is underlying demand, which is continuing to be adopted as we add new accounts into the prescribing metrics. So that's one of the other considerations that we are seeing as we've constantly looked at this part of the equation. Hope that helps. Thank you.

Yeah. That’s helpful. Thank you.

Great. Thanks, Tom.

Thank you.

Our next question comes from Carter Gould with Barclays. You may proceed with your question.

Great, good afternoon. Congrats on the results, and thanks for taking the question. I'm going to follow up on the -- Marty's previous question there. I guess just -- can you maybe just give a little bit more color on the breakdown? If you had to, kind of, like think about that mix in 2Q versus 3Q, did that -- I mean are you starting to see that move ready, or was that relatively stable quarter-on-quarter as you think about that prevalent pool versus those patients? I think you kind of called it earlier in their treatment course that prevalent pool versus those patients. I think you kind of called it earlier in their treatment course. That would be helpful. And I guess Habib on that point then as you think about 4Q, you talked about COVID impacts but just last quarter COVID was seen as a potential -- driving a potential tailwind for you guys in terms of keeping patients out of the centers and getting transfusions. So is there any sort of -- is there a change in your thinking there, or maybe you could just elaborate there? And then separately on sotatercept, in the past we've talked about preclinical work that needs to be done in order to justify moving into broader indications in PH -- beyond PAH. Can you give us an update on those efforts and when we might see some sort of clinical development strategy manifest? Thank you.

Yeah, Carter, thanks. There's a lot packed in there. And so regarding the sotatercept question, I'll hand that over to Jay. But just again to close off on the follow-on question with respect to the commentary. Yeah, we haven't really shared any details with respect to what the percentage is of the more prevalent population versus those that are earlier in their treatment journey. That said, I think it's very clear that through Q2 and Q3 we had a very -- a large portion of those patients that -- due to that pent-up demand. And over time that -- those patients will not be making up the majority of the patient group. And what we're seeing now as Sujay is alluding to these new accounts and these new patient starts earlier in their treatment journey are really starting now to pick up and that are driving the uptake in terms of new treatment and new scripts. Now with respect to the COVID statement and you're absolutely right. I mean it actually does play a role in terms of a bit of an opportunity that we can perhaps play a role in terms of mitigating the huge need for transfusion burdens during this pandemic. And you -- but that said, during Q2 and Q3, we obviously were seeing some of that with some of the patients. But most importantly now as we look forward and really it's an unknown, what we're seeing now is record numbers that are spreading across the globe. And what our concern is that perhaps office visits. So again nothing that takes away from the opportunity to potentially reduce the need for transfusion burdens in this very difficult environment. But what we are seeing now with these record numbers around the world is that we're concerned that there may be -- again I don't have a crystal ball but there may be a decrease in in-office visits and treatment compliance as things start to shutdown in some areas of the world. And so that was really what that commentary was about Carter. So no change in thinking in terms of the opportunity there with respect to red blood cell transfusions. With respect to sotatercept, can I hand that over to you Jay please?

Yeah, sure. Good question. So we have not published nor disclosed the nonclinical work that we've had underway. But I can tell you that that is nearing completion as we think about moving into for example Group II or III or beyond. So we haven't disclosed that yet either. But as we do that Carter, we're also preparing to establish a proof-of-concept study in one of those areas. So it will be something that I'm looking forward to sharing more detail on, similar to what we talked about with the other two programs for the Phase III for sotatercept and Group I PAH. Look forward to being able to talk more about that in detail as we get into next year.

Great. Thanks a lot.

Thanks, Carter.

Thank you. Our next question comes from Geoffrey Porges with SVB Leerink. You may proceed with your question.

Hi, this is Charles Song for Geoff. Two questions if I may. First on REBLOZYL. Just want to get your thinking about the changing competitive dynamics in the -- I guess MDS space particularly frontline with the HIF inhibitors and the CD47. So how do you position your products versus potential competition? And for sotatercept, I just want to, I guess, a little bit detailed question. For the STELLAR trials seem to include a more newly diagnosed PAH patients, in cutting down the portion of the patients on triple therapy. So how should we think about the data -- potential data campaign to PULSAR? Will the data come out to be stronger or weaker depending on this difference in the patient baseline? Thank you.

Yeah. No. Thanks for your question. So why don't I hand it over to Jay to address the question right now in STELLAR. And then maybe Jay hand it over to Sujay for the REBLOZYL question on the …

Yeah. Sure. So, just as a reminder, STELLAR really was modeled after PULSAR, so just to start there. It was designed to look the same. We have not restricted in any way the triple therapy group. So we anticipate that it will have triple therapy in it as well. Recall, as we presented the data from PULSAR, we had mono double and/or triple therapy patients in there overall. We were very pleased with the impact we had on six-minute walk distance when we looked at the totality of the data. So kind of going into this, frankly we have the trial designed in such a way that we expect similar success with it as we did with PULSAR. And maybe, I can hand it over to Sujay, for the REBLOZYL question.

Yeah. No. Thank you, Jay. With regards to competition, as we've sort of communicated, and you've seen from the uptake of REBLOZYL, because we are sort of a very novel therapeutic in the area of erythroid maturation, we provide a unique sort of value proposition, right? And that's sort of demonstrated by how quick there was an adoption of REBLOZYL by many of the accounts. Now having said that, we have sort of positioned ourselves in a way, where at least based on our indications right now, it is in the low-risk patient population where anemia is predominantly a problem. And that's kind of where we have been utilized in the RS-positive patient population. So that's where -- and now there are other sort of molecules that are being developed and some of them are more in the HMA space, so we don't see them directly as competing from sort of the low-risk patient population for MDS. And then, when you think about beta-thalassemia, again if you think about the other available options for gene therapy et cetera they tend to have a select group of patients that are going to be applicable because they need complete myeloablation et cetera, in relation to that as well as a different patient group, right? So we feel that, having more options for both these indications, whether it's MDS or b-thal, it's always good for the patients. But we feel that we have a very good position in the market for serving the patient population that we are serving right now.

Thank you.

Thank you. Our next question comes from Chris Raymond with Piper Sandler. You may proceed with your question.

Hi. This is Nicole Gabreski on for Chris. Congrats on the quarter. And thanks for taking the questions. I guess, just on biz dev, I guess what's your appetite for external innovation at this point, or I guess maybe just generally, do you see a need to augment your pipeline with a BD deal in the near future?

Yeah. Hey, Nicole, thanks for your question. So I think what I would say is, right now, the -- when we look at -- and you heard from Kevin in terms of our financial position, you're hearing a lot from Sujay, in terms of the launch with REBLOZYL. And then obviously all of the efforts with respect to sotatercept and really building out that portfolio of Phase III studies to establish sotatercept as a backbone therapy in PAH. I feel that, we have a tremendous opportunity to execute and to generate value for shareholders, if we really focus and really focus on executing what I would say is a very admirable position, in terms of having two potential blockbusters in our hands. Now that said, we are also very, very focused on continuing to innovate and identifying opportunities for innovation, whether that's organically or inorganically. Organically as you know, in terms of our quest of establishing a leadership position for sotatercept, we're also exploring other areas within pulmonary hypertension, which hopefully you'll hear a little bit more about next year. The second thing I'd say is organically, we talked briefly last call -- last earnings call about ACE-1334, which is in healthy volunteers right now. But that we hope to be able to go into patients next year, again in the pulmonary setting. With respect to business development, again at the end of last year, you may recall, we announced the deal with Fulcrum Therapeutics where we were able to partner with a neighbor in Cambridge, where they're really focusing on a small molecule approach and target identification with respect to modulating gene expression pathways. And here we're specifically locked up an agreement with them, areas within rare pulmonary diseases as well. So, we're obviously looking to continue to invest both, organically and inorganically. And if indeed, there is an opportunity that would make sense that would complement our strategy right now in establishing ourselves as a leader in rare pulmonary diseases we'd be always very open to it. But we absolutely do not see the necessity, to have to execute anything in the near-term.

Great. Thanks for taking my question.

Yeah. No. Thank you for the question.

Thank you. Our next question comes from Eric Joseph with JPMorgan. You may proceed with your question.

Thanks. Just looking to the BEYOND readout, later this year or early next year I guess how should we be thinking about the non-transfusion-dependent population as a proportion of beta-thal, overall? Does BEYOND -- I guess does it help you access those patients in and of itself? If the data are positive, what would you be looking for to if needed, move forward with a label expansion study for non-transfusion dependent -- the NTD population? Thanks.

Yeah. Thanks for your question, Eric. So in terms of the market opportunity, perhaps I'll take that. And then I'll hand over to Jay, in terms of the way we're thinking about BEYOND. So I'd say two things about the opportunity Eric, is one, in terms of the unmet need you may recall, this is on probably over two -- almost three years ago and Dr. Cappellini was speaking for us at an R&D Day. And she's pretty adamant that the non-transfusion-dependent beta-thalassemia community has an unmet need for innovation, such as REBLOZYL just as much as the transfusion burden patients do, just because of the fact that, they're -- just because they're not transfusing does not mean that they're not symptomatic and suffering from many, many aspects of the disease. So that's the first thing. The second thing I'd say, in terms of the actual numbers, we estimate that, when you look at the beta-thalassemia population in the U.S. and Europe that the non-transfusion-dependent population would double the existing target group that we have with the transfusion-dependent indication. Jay, maybe you can add a little bit of color in terms of the way we're thinking about BEYOND.

Yes, certainly. So BEYOND, as you know, Eric, it was actually a program that was started when I was still at Celgene. And so there was – has been ongoing dialogue with the agencies around that trial. So what I would anticipate now with BMS our partner kind of in the lead here that once we see those results then we follow up and engage and depending on the strength of that data have further discussion with the agencies as to what would be required as the next step. So we're looking forward to seeing those results. To Habib's point, this is really a difficult population. Those that get – not getting regularly transfused their quality of life is significantly challenged. And I do believe we have an opportunity to make a difference here. So we're looking forward to sharing those results.

I guess separate from improvements in hemoglobin, hemoglobin rise, are there other key end points that you're focused on to I guess speak to ameliorating the burden that non-transfusion-dependent patients suffer?

Yes very much so. In fact patient-reported outcomes in the context of that primary end point will be an important secondary end point, which would then again speak to my point about the benefit that they derive from that improvement and the impact that it has on their quality of life measures is included in that. So that has been built into the trial.

Great. Thanks for taking the question.

Thanks, Eric.

Our next question comes from Yigal Nochomovitz with Citigroup. You may proceed with your question.

Great. Thank you very much for taking the question. I just wanted to drill down a bit on PULSAR a little bit more. As you mentioned, you're going to have the 24-week echo data at AHA coming up. If you could just help us put that data in clinical significance and put it into perspective with respect to the other end points that you've obviously already reported PVR and 6-minute walk distance. More specifically how impactful a drug can rather what the – how impactful can sotatercept be with the echo data in addition to the PVR and 6-minute walk data that you've already reported?

Yes. So AHA is around the corner. So the details of the echo data we'll share when we get those publicly disclosed through AHA. But just to kind of put into context, as we reported out in the non-clinical work, things that we were anticipating or hope to see in the clinic would be replicating what we saw in the non-clinical work. And what we saw is actually an improvement in right ventricular function, right ventricular hypertrophy. And as you know echocardiographic data will give us some insight how that looks clinically against the effect that we had on reduction in PVR. So it was a very important end point that we included in it and actually looking very forward to sharing those data soon.

Okay. Great. And then you're also going to be reporting the open-label extension data from PULSAR in the first half of next year. I'm just wondering if you could talk a bit about your hypothesis there scientifically in terms of what you expect to see on PVR and 6-minute walk distance. Do you believe that you've achieved the maximal effect for PVR and 6-minute walk distance and that you're just going to maintain the benefit, or based on the mechanism of action of sotatercept, do you believe that you could continue to see increased building of efficacy over time?

Good questions which is actually part of the questions that we're posing within the trial itself. I think just to take a step back, the open-label extension will provide a richness of opportunity for safety and tolerability, which is obviously very important. Maintenance of effect as you talked about and whether there's continued improvement both functionally and potentially in the right heart cath data. So again, we're – that's the trial we're working on looking forward to completing it and sharing those data next year.

Thank you.

Thanks, Yigal.

Thank you. Our next question comes from Kennen MacKay with RBC Capital Markets. You may proceed with your question.

Hey, guys. This is Bikram on for Kennen. Thank you so much for taking our questions and congrats on the quarter. Just following up on the readout of BEYOND trial top line results, I have a quick question. Is there any internal bar that you guys are looking at for response rates in this population and how that might be different from the other beta-thal population?

Great. Thanks for the question, Bikram. Jay, do you want to take that please?

Yes. Well, so I think for this trial because these patients are not regularly transfused and they're the non-transfusion-dependent, the primary end point in the study was change in hemoglobin, so an increase in the hemoglobin. So that will be the measure. And like in any randomized study, we've had assumptions under there and defined results that we expect to see, so the bar would actually be a successful study. As we designed and powered we believe that if we meet the primary end point that will be the threshold. And then following that the earlier questions, we're seeing consistency within the secondary end points, which include the patient-reported outcomes. So we're getting close. That trial has been running for a while. And as you heard from Habib, we're looking forward to those results potentially by the end of the year if not early part of next year.

Got it. Thank you so much. And I just have a follow-up. Maybe just taking a step back and thinking about other indications for REBLOZYL and how you guys are thinking about BEYOND – sorry not BEYOND trial but the currently approved indications and MDS or myelofibrosis, where are you guys focusing on your internal R&D efforts?

Yes. So maybe I'll just continue then. So as you know obviously our partner BMS is taking the lead on the development at this stage. What you heard from Habib early on I think things that you can expect because of our presence in MDS building around that and we're starting to see now combinatorial studies as you heard one with combining with lenalidomide or Revlimid. So I would say look forward to seeing more of those combination-type trials throughout the spectrum of MDS. The myelofibrosis study is starting. And again you heard that BMS is actually now added luspatercept to their own, JAK2 inhibitor fedratinib. So we're going to start to see again build out around I think the myelofibrosis space. And then there are other areas of indication we can anticipate because of our effectiveness in beta-thalassemia, as we've seen and believe potentially looking at for example alpha-thalassemia. So there's a full life cycle plan that's underway. And again, as we establish the presence in the commercial space, it gets easier to do combination studies of approved products and you're going to see those come forward. So let's just stay tuned but look to see a build-out around both MDS and myelofibrosis plus some of those newer indications.

Got it. Thank you so much for taking our questions.

Thank you.

Thank you. We have now reached the end of the Q&A session. I would now like to turn the call back over to Habib Dable for any closing remarks.

All right. Thank you, operator. And I guess before we conclude I want to take the opportunity to thank everyone for joining us. I hope that everyone continues to stay safe and well during this current COVID environment and I look forward to connecting with many of you at some of the upcoming virtual conferences. And in the meantime, if you have any questions, please feel free to reach out to Todd or Jamie. Have a great evening everyone.

Thank you. Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.