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Edited Transcript of XLRN earnings conference call or presentation 11-May-20 9:00pm GMT

Q1 2020 Acceleron Pharma Inc Earnings Call

Cambridge May 12, 2020 (Thomson StreetEvents) -- Edited Transcript of Acceleron Pharma Inc earnings conference call or presentation Monday, May 11, 2020 at 9:00:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Habib J. Dable

Acceleron Pharma Inc. - CEO, President & Director

* Jay T. Backstrom

Acceleron Pharma Inc. - Executive VP and Head of Research & Development

* Kevin F. McLaughlin

Acceleron Pharma Inc. - Senior VP, CFO & Treasurer

* Sujay R. Kango

Acceleron Pharma Inc. - Executive VP & Chief Commercial Officer

* Todd James

Acceleron Pharma Inc. - Senior VP of Corporate Affairs & IR

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Conference Call Participants

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* Carter Lewis Gould

Barclays Bank PLC, Research Division - Senior Analyst

* Eric William Joseph

JP Morgan Chase & Co, Research Division - VP & Senior Analyst

* Geoffrey Craig Porges

SVB Leerink LLC, Research Division - Director of Therapeutics Research & Diversified Biopharma and Senior Research Analyst

* Jeff Hung

Morgan Stanley, Research Division - Equity Analyst

* Kennen B. MacKay

RBC Capital Markets, Research Division - MD & Co-Head of US Biotechnology Research

* Kyuwon Choi

Goldman Sachs Group Inc., Research Division - Equity Analyst

* Leland James Gershell

Oppenheimer & Co. Inc., Research Division - MD & Senior Analyst

* Mark William Connolly

Crédit Suisse AG, Research Division - Research Analyst

* Samantha Lynn Semenkow

Citigroup Inc, Research Division - Senior Associate

* Yaron Benjamin Werber

Cowen and Company, LLC, Research Division - MD & Senior Biotechnology Analyst

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Presentation

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Operator [1]

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Good afternoon, ladies and gentlemen, and welcome to the Acceleron First Quarter 2020 Earnings Conference Call. (Operator Instructions) As a reminder, this conference call is being recorded.

I would now like to hand the call over to Mr. Todd James, Senior Vice President, Corporate Affairs and Investor Relations at Acceleron. Please go ahead.

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Todd James, Acceleron Pharma Inc. - Senior VP of Corporate Affairs & IR [2]

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Thanks, and welcome, everyone, to our first 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 & 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; and Sujay Kango, our Chief Commercial Officer.

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.

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [3]

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Thank you, Todd. Good afternoon, everyone, and thank you for joining us today.

Before we get started, I wanted to take a moment to address the COVID-19 pandemic that is affecting all of us today. First and foremost, our hearts go to everyone who has been directly impacted. And to the health care and other workers who are on the front line, we thank you for your selflessness to help keep us all at pace.

As the situation continues to evolve, we have been moving quickly to protect and support our employees and the patients that we serve. In early March, we implemented a work-from-home policy and successfully transitioned to virtual external stakeholder engagement. We continue to advance business critical, laboratory, manufacturing and related support activities as a responsible essential business, while taking the appropriate measures to minimize risks and ensure the safety of the limited number of employees who are still coming into our facilities. Although it's too early to tell how impactful this pandemic will ultimately be, at present, we have been fortunate to experience minimal effects on our business and operations. This is in a large part due to the foresight commitment and flexibility of the entire Acceleron team. Over the coming weeks and months, we will continue to monitor the situation carefully and follow guidance from local and federal health authorities.

Despite some adjustments to our day-to-day operations necessitated by the current environment, we continue to make strong progress on the clinical and regulatory front. About one month ago, the FDA approved Reblozyl, also known as luspatercept, for its second indication. The treatment of anemia failing erythropoiesis-stimulating agent, requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndrome with ring sideroblast or myelodysplastic myeloproliferative neoplasm with ring sideroblast and thrombocytosis.

Reblozyl was first approved in November for the treatment of anemia in adult patients with beta-thalassemia, who require regular red blood cell transfusions, becoming the first and only approved erythroid maturation agent.

Acceleron discovered Reblozyl just over a decade ago. And as you can imagine, these approvals mark a proud moment for our team and our long-time partnership with Celgene and now Bristol-Myers Squibb. More important, there are huge victories for the thousands of patients with MDS and beta-thalassemia, who until now have been waiting for a new option to treat their anemia. Outside of the U.S., the committee for medicinal products for human use of the European Medicines Agency recently issued a positive opinion on Reblozyl, recommending its approval for the treatment of transfusion-dependent anemia in adult patients with lower-risk MDS and beta-thalassemia. We expect the EMA's decision on the marketing authorization application for Reblozyl in the second half of this year.

I'd like to recognize the joint development team at Acceleron and Bristol-Myers Squibb under continued achievements. In the first quarter, results from both the pivotal Phase III MEDALIST and BELIEVE trials were published in the New England Journal of Medicine. These publications were Acceleron's first in its prestigious peer-reviewed journal.

Moving to Reblozyl's commercial launch. Our field team is hard at work alongside our partner, Bristol-Myers Squibb, targeting the top health care providers and centers of excellence throughout the U.S. The first 5 months of commercial sales have been encouraging. We recognized approximately $1.5 million in royalty revenue on net Reblozyl sales of approximately $8 million for the first quarter of 2020. Importantly, we are experiencing new and repeat orders from medical centers across the country, which has continued with the recent launch in the MDS indication to date. The current public health environment does introduce certain challenges that we continue to monitor, including patients and the potential caregiver's ability to manage the regular physician appointments and our field teams transition to a virtual sales model as of mid-March.

As we look toward our long-term vision for Reblozyl, we believe its novel mechanism of late-stage erythroid maturation could enable it to become a possible platform treatment for thousands of patients suffering from anemia caused by a variety of hematologic diseases. To that end, in partnership with BMS, we are presently conducting 2 additional trials and preparing for a third called the independence trials. This pivotal Phase III trial will evaluate luspatercept for the treatment of anemia in patients with myelofibrosis who are on a JAK2 inhibitor and require red blood cell transfusion. Between now and the end of 2022, we anticipate a steady flow of top line results from the BEYOND study in non-transfusion-dependent beta-thalassemia and the COMMANDS study in the front line, lower-risk MDS. And we also continue to evaluate additional disease areas in support of our vision for luspatercept as a true platform treatment for anemia.

While Reblozyl remains a top priority, we are equally excited by the progress we are making in pulmonary disease. In January, we were absolutely thrilled to announce positive top line results from the PULSAR Phase II trial in pulmonary arterial hypertension, or PAH. We believe these data demonstrate sotatercept's ability to potentially reshape the treatment paradigm for patients with this devastating disease on top of currently available therapy. Most recently, sotatercept was granted breakthrough therapy designation by the FDA and priority medicines designation by the EMA, a first for an Acceleron-discovered medicine and for a therapeutic candidate in PAH. These designations allow for more regular interaction with the agencies and additional access to senior division members along with expedited application filings and application review period following pivotal Phase III trial results. We look forward to engaging in end of Phase II discussions with the global health authorities by the middle of this year as we work to finalize our Phase III development plan in the second half. We currently expect to initiate a pivotal Phase III trial globally at the end of this year or early 2021.

Following the cancellation of the in-person American Thoracic Society 2020 International Conference due to COVID-19 pandemic, ATS is now planning to host certain sessions virtually. The web-based session, including the detailed presentation of top line results from the PULSAR Phase II trial is now expected to take place before the end of June. We will provide more information on timing and how to access the presentation as soon as it becomes available. Acceleron will host a separate webcast presentation for investors and analysts following the session. We hope that many of you will be able to join the call.

And with that, Kevin McLaughlin, our CFO, will review the financials. Kevin?

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Kevin F. McLaughlin, Acceleron Pharma Inc. - Senior VP, CFO & Treasurer [4]

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Thanks, Habib. Good afternoon, everyone. I would like to refer you to our press release issued earlier today for a summary of our financial results for the first quarter 2020 and take this opportunity to briefly review a few items.

We ended the first quarter with approximately $415.6 million in cash, cash equivalents and investments. Revenue for the first quarter of 2020 was $4.3 million, which includes $2.8 million of cost-share revenue, and as Habib mentioned earlier, approximately $1.5 million of royalty revenue from net U.S. sales of Reblozyl of approximately $8 million. All revenue was derived from the company's partnerships with Bristol-Myers Squibb.

The company's net loss for the first quarter ended March 31, 2020, was $50.9 million.

And with that, I'd like to open the call to questions. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from the line of Martin Auster from Crédit Suisse.

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Mark William Connolly, Crédit Suisse AG, Research Division - Research Analyst [2]

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This is Mark on for Marty. So my question was for SPECTRA, has been -- has there been any impact on data collection in patient dosing due to COVID-19? And if so, how much flexibility is there built into the protocol?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [3]

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Mark, thanks for your question. Maybe I'll pass that over to our Head of R&D, Jay Backstrom, to address.

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Jay T. Backstrom, Acceleron Pharma Inc. - Executive VP and Head of Research & Development [4]

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Yes. So we've been working very carefully with the sites to really allow them to continue to have access to patient dosing. Both FDA and EMA have issued guidance in the context of the pandemic for management under GCP. We're very much compliant and on top of that. Fortunate to say so far, patients have been able to still access medicines without having a risk of really being denied treatment. So far, the dosing has been fine.

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Operator [5]

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Our next question comes from the line of Yaron Werber from Cowen.

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Yaron Benjamin Werber, Cowen and Company, LLC, Research Division - MD & Senior Biotechnology Analyst [6]

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Great to see such a strong number on Reblozyl. So my questions have to do with -- do you have any sense how much of it is demand and was there any sort of traditional inventory kind of pull in such an early stage in the launch? And do you have any sense how much of sales were unlabeled versus potentially early adoption into MDS or MS, sort of for anemia more broadly?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [7]

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Yes. Thanks for your question, Yaron. And I can tell you on the Acceleron end of things, we are also very, very pleased with the first quarter sales. Just to remind everyone, that first quarter was represented sales for our first indication in adult beta-thalassemia patients requiring regular red blood cell transfusions in the U.S. alone. And so we're very much looking forward to building upon that now with our -- with the MDS being approved in the second quarter. And just -- again, I'll start it off and I'll pass it over to Sujay, our Chief Commercial Officer, to add some color. But just to remind everyone, the method of deliveries here is really a drop shipment. And so not a lot in terms of typical inventory buildup that you run in terms of the first part of your question. But I'll pass it on over to Sujay to add a little bit more color.

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Sujay R. Kango, Acceleron Pharma Inc. - Executive VP & Chief Commercial Officer [8]

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Thanks, Habib. And Yaron, thanks for that question. So as Habib said, right, so the way BMS and we've created the distribution model is the order is collected to the wholesalers and we directly ship it to the account. Plus if you recognize that this is more of a buy-and-build Medicare Part B product, so the likely situation in a traditional model where the wholesalers hold inventory and then they send it out doesn't occur in this. And within 24 hours, the product goes to the account. So we don't anticipate a whole amount of any buildup of inventory at a local physician's office or a hospital level. They usually order it as soon as they have a patient in mind, they send the order through and within 24 hours, the vials are shipped to the doctor's office or the hospital. So normally speaking, there wouldn't be a buildup of inventory. So most of this is almost directly related to a patient that they're starting on treatment within a day or so. Okay. Thanks a lot for that question, Yaron.

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Yaron Benjamin Werber, Cowen and Company, LLC, Research Division - MD & Senior Biotechnology Analyst [9]

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And just a quick follow-up on launching in the COVID-19 environment is a little bit of a double-led story. This is a certainly a tough environment to sort of -- to start detailing a new product, but this is a product that people have known about. And we've also been hearing that there are some supply issues in blood centers. So can you tell me what -- can you tell us what feedback you're hearing so far?

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Sujay R. Kango, Acceleron Pharma Inc. - Executive VP & Chief Commercial Officer [10]

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So Habib, you want me...

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [11]

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Yes. You can continue, Sujay.

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Sujay R. Kango, Acceleron Pharma Inc. - Executive VP & Chief Commercial Officer [12]

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Okay. Perfect. So Yaron, you raised the question whereby there's an opportunity, as you're pointing out. So we've heard from the purposes of the blood supply is a challenge because many donors have a challenge going in and the donor supply has come down a bit. So with that, there is an opportunity with regards to Reblozyl, as you pointed out, because we do offer transfusion independence for the right patients with MDS. So that's an opportunity because it's squarely in line with the benefits that we do provide. On the flip side of the equation, we do also know based on our assessments that there are new patients or actual patients for hematology going into the centers, that has come down, right? So it's a balancing aspect of it. Whilst our early launch metrics are very good and we are very encouraged, we have to wait and see over a period of time what is the overall net-net effect of this, but we are very encouraged with the early start of the product through launch as well as virtual detailing because we are doing that including speaker programs. We do also have those outlined and we've already started to execute those. So more to come in the second quarter as we monitor this, okay?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [13]

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Yes, Yaron, this is Habib. And just to complement what Sujay is saying, you need to remember, this is a community who has been -- had the opportunity to benefit from the publication in the New England Journal of Medicine. As I mentioned earlier, we were simply thrilled to be able to be accepted in this publication. And so awareness levels as a result of that significantly increase. And again, there hasn't been anything new in this space in over a decade. And therefore, the opportunity for us to get our messages out there to a market that's been waiting for something new provides us with a wonderful opportunity to increase share of voice.

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Operator [14]

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Our next question comes from the line of Geoffrey Porges from SVB Leerink.

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Geoffrey Craig Porges, SVB Leerink LLC, Research Division - Director of Therapeutics Research & Diversified Biopharma and Senior Research Analyst [15]

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I just wanted follow-up questions about sotatercept. Can you just give us a sense of -- obviously, the ATS presentation is pushed back a little bit. What's the latest timing on your meeting with the FDA? And what's your latest thinking on the possibility of filing directly with this data? And lastly, you highlighted the cash balance, Kevin. Do you expect that your cash reserves are sufficient to fund pivotal sales, assuming they're required for sotatercept?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [16]

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Geoff, this is Habib. Thanks for your questions. Regarding the first question on ATS, you're right. The live Congress would have been happening in a few days. And unfortunately, like many and all other congresses actually that it has been canceled. And what the ATS has done is they've moved to a virtual format. We're currently working with them, and we feel very confident that we will be leveraging that Congress for our presentation before the end of June in a virtual format. So you can expect that from a data point of view.

With respect to your question regarding the opportunity in terms of filing directly and interactions that we've had with the FDA and other health authorities, a couple of things. One, what we said is that by the middle of this year, our goal is to have those interactions with the health authorities, the various health authorities. And to be able to get back with you shortly after that Q3 time frame, maybe Q4, but on the Q3 time frame and being able to share with you our thinking based on those conversations and the advice that we received on what a Phase III trial would look like. Now -- and I'll allow Jay to -- if you would like to add a little bit more color on that, but in terms of your question, just to finish up on the question regarding the ability to file directly. I'd like to remind everybody, the PULSAR study has been designed and powered as a proof-of-concept study. And we're simply thrilled with the fact that we were able to hit statistical significance on the primary endpoint as well as our secondaries. But at the same time, I do want to remind everyone that the primary endpoint was pulmonary vascular resistance. And we all know that, that is an important hemodynamic endpoint. But at the same time, it's equally important for us to be able to identify a key secondary endpoints such as 6-minute walk distance, which would be very important for us as we think about designing our registrational studies in terms of having some sort of a functional benefit, for example. So I've heard the conversations in the chatter regarding the ability to file on this data. I think if you look at past precedent, that would not be the case. I think we have the added benefit of having a prime designation as well as breakthrough therapy designation with the FDA. I think that gives us a tremendous opportunity to benefit from frequent senior-level counsel as we think about designing our pivotal studies for Phase III, and we're very much looking forward to engaging with the regulatory bodies in that regard. And if and when, we have that -- all of that lined up in the third quarter or perhaps early in the fourth quarter, we'll be coming to you with that registrational study design as soon as we can.

Regarding the dollar, in terms of the ability to fund, it's too -- it's presumptuous right now to think about what we would have in terms of needs and cash needs to run our pivotal study. I'll tell you right now though, our goal is to move forward with global registrational studies independently. And based on the conversations that we will have and the funding requirements for those studies, I can -- I feel very confident that we're going to raise -- need to raise more money to be able to fund those Phase III studies on our own. Kevin, Jay, would you like to add anything to that, if there's anything that I missed?

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Jay T. Backstrom, Acceleron Pharma Inc. - Executive VP and Head of Research & Development [17]

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This is Jay. I think you summed it up really well from the clinical regulatory front. We're certainly on track to stay on time with our interactions with the agency. And we said previously, we're really planning and prepared to execute a Phase III study. So we're in that planning process and more to come after we engage with the health authority.

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Kevin F. McLaughlin, Acceleron Pharma Inc. - Senior VP, CFO & Treasurer [18]

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Yes. And this is Kevin. And Geoff, thanks for the question. I think that you know we ended the first quarter with about $450 million in cash, as Habib said, we're pleased with that. But we are always looking forward to the future and what our cash needs will be. First, certainly, the focus for us will be continued work in the research department as well as our focus on the sotatercept program going forward from a study standpoint, from a manufacturing preparation standpoint, and obviously, from a market development standpoint.

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Operator [19]

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Our next question comes from the line of Carter Gould from Barclays.

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Carter Lewis Gould, Barclays Bank PLC, Research Division - Senior Analyst [20]

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I offer my congrats on the MDS approval. I guess maybe just a follow-on on the previous question. Just in terms of sort of the sensitivities around being able to start the PAH study later this year, can you maybe just give a little bit more detail? I mean, obviously, we've got to wait for the design to kind of be released. But when we think about other additional gating steps, can you maybe just go into a little bit more color there? I think I believe Kevin just mentioned manufacturing, some -- any additional color there would be helpful in terms of where you stand and other kind of gating steps? And then maybe, I guess, my reach question, you seem to be kind of characterizing the Phase III program as a single study. Should we think about when that gets disclosed around a single pivotal study? Or could we get greater detail on the broader kind of clinical program?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [21]

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Yes. Thanks for your questions, Carter. I'll start off with the last question. If it came across as a single study are proving presumptuous, that's absolutely not the case. We're not ready to say that at all until we've had those proper conversations with the health authorities. But in terms of our thinking and some of the gating strategies, et cetera, I'll hand that over to Jay to elaborate on how we're thinking about that at this point.

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Jay T. Backstrom, Acceleron Pharma Inc. - Executive VP and Head of Research & Development [22]

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Yes. I think the key thing is to actually to engage with authorities and get agreement on the trial designs. And I think what Habib said earlier, we're really fortunate with breaking therapy designation and the prime designation. It really affords us the opportunity to effectively engage, and that's our plan. I mentioned earlier, study start-up, there's a lot of steps behind that. Many of these we can take even in advance of the protocol. But essentially really getting that protocol up ready and agreed by health authorities or the protocols, (we're working on a full development plan).

As we get that forward, I think we're going to be geared to go. We've been really fortunate that we've run a multicenter trial with the top experts, all of whom are obviously enthusiastic about the program given their results. So we're leveraged significantly as we think about starting up a Phase III. On the timing that we had envisioned, I think, even at the beginning was to get something ready by the end of the year. So far, I mean, everything is really aligning against that. We'll have, obviously, much more detail when we've completed our health authority interactions. And frankly, all of us are looking forward to sharing those plans with you, but it would be premature prior to the health authority feedback.

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Operator [23]

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Our next question comes from the line of Eric Joseph from JPMorgan.

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Eric William Joseph, JP Morgan Chase & Co, Research Division - VP & Senior Analyst [24]

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Just a couple on Reblozyl and beta-thalassemia. The first is, if you could just remind us about the split and coverage in that -- split and reimbursement in that segment and whether you anticipate any change in that mix as a result of a weakening economy potentially here? And then secondly, I'm wondering if you could provide us with an update on -- an enrollment update in the pediatric Phase II trial. What's sort of positive data would look like in that study? And just generally, how should we think about the incremental commercial opportunity in the pediatric segment relative to the current adult transfusion-dependent label?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [25]

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Okay. Thanks for your questions, Eric. So for the first question regarding the access split, I'll ask Sujay to address and then maybe you can pass it on to Jay to talk a little bit about where we are with the pediatric indications.

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Sujay R. Kango, Acceleron Pharma Inc. - Executive VP & Chief Commercial Officer [26]

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Thanks, Eric, for the question. Thanks, Habib. So in the beta-thalassemia segment, as you asked, largely given the age group of these young adults, most of these patients are going to be on the commercial pay side of the equation. So that's going to be on the commercial. About 70% or so, we believe are going to be sitting over there. We've always anticipated about 30% or so of patients would be covered under government pay, and that could be a combination of Medicare, Medicaid, et cetera. So that's how we see the business for the beta-thalassemia side of the equation. Largely speaking, if you put it into context, Eric, right? If I could just put one more color over there. The MDS opportunity is a lot larger as you know 1,000 to 1,500 patients for beta-thalassemia versus approximately 30-odd-thousand in relation to MDS. It's a magnitude difference. And over there, a larger proportion, just the opposite are going to be on the Medicare side of the equation. About 70% of MDS patients are going to be mostly covered under Medicare side of the business versus 30% on the commercial pay. So gives you a context that net-net as the MDS side of the business comes into line, the ratios are going to significantly switch over to the Medicare pay versus the commercial pay. Yes, hopefully, that answers your question.

And Jay, if you want to cover the pediatric side of the question?

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Jay T. Backstrom, Acceleron Pharma Inc. - Executive VP and Head of Research & Development [27]

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Yes. Certainly. And for the pediatric, we're running a pediatric plan. It's an approved PIP as it's referred to in Europe. And those -- as is typical, those designs, those are often cohort designs. So you pick an age group and then you go down to earlier and earlier age groups. So it's early days for the pediatric plan to be thinking about the -- where we are with that program. Very early days.

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [28]

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Yes. And in terms of the opportunity, Eric, we estimate that it's about 3 or 4:1 adults to pediatrics in terms of the beta-thalassemia population. So the majority is in the adult. At least in the developed countries that -- where we've got the good data. Yes.

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Operator [29]

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Our next question comes from the line of Yigal Nochomovitz from Citi.

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Samantha Lynn Semenkow, Citigroup Inc, Research Division - Senior Associate [30]

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This is Samantha on for Yigal. Just on the ATS presentation, how much lead time are we going to get to know when the presentation is coming? Is it going to be in a press release that morning? Or are we going to get 1 to 2 weeks advance prior to -- as an ATS is going to announce that the presentation that's happening within that time frame.

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [31]

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Yes. Thanks for your question, Samantha. I'm going to pass it on over to Todd, our Head of Corporate Affairs, to address that. If you would, Todd?

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Todd James, Acceleron Pharma Inc. - Senior VP of Corporate Affairs & IR [32]

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Samantha, yes, thanks for your question. Yes. So a little bit out of our control and in the hands of ATS when they're going to outline the schedule for the session. But we would expect it to at least be a few weeks in advance of the actual session to give some people some lead time to open up their schedules. As you could imagine, with the canceling of the in-person event, the Congress would like to give their stakeholders enough time to be able to clear up their schedules to then be able to participate in this virtual event. So you could expect a decent amount of time in advance.

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Samantha Lynn Semenkow, Citigroup Inc, Research Division - Senior Associate [33]

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Okay. Great. And then can we expect the presentation will have a breakout of the data by the number of background therapies, 2 to 3 background therapies position the patients from?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [34]

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So with respect to the presentation, we'll have to wait for that. But what we've said already is that this is a very heavily pretreated population, where over 50% of the patients that were recruited into the PULSAR study were actually on triplet vasodilatory therapy, including prostacyclin.

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Samantha Lynn Semenkow, Citigroup Inc, Research Division - Senior Associate [35]

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Okay. Got it. And then I guess just one more on sotatercept. Are there any additional indications where you are expecting to start development, especially now that you ceased development of ACE-083, and your next stage pipeline appears pretty open for maybe another indication that you could start developing. Just curious on any thoughts you have there?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [36]

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Yes. So that's a really good question. So obviously, right now, all focus is on the PAH and Group 1 pulmonary hypertension. Obviously, as we continue to evolve the sotatercept story, there'll be more to come on where else we can take it. I would like to remind you, though, that we do have another asset in the pulmonary space that we're investigating. It's in a healthy volunteer study, ACE-1334, we announced that at the JPMorgan conference early this year. The -- more to come on that as that program evolves. In 2021, you can expect to hear more about that program and where we're taking it. So we're feeling really good about our development pipeline in pulmonary. And again, just finally, we engaged at the end of last year and collaboration with the company in Cambridge, Fulcrum Therapeutics, with -- and leveraging their small molecule approach to modulating gene expression and we have opportunities that we've locked up with them in the pulmonary space. So we're feeling actually very good about our prospects in pulmonary in the pipeline development.

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Operator [37]

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Our next question comes from the line of Leland Gershell from Oppenheimer.

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Leland James Gershell, Oppenheimer & Co. Inc., Research Division - MD & Senior Analyst [38]

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Actually, my questions have already been asked and answered. But thank you again and congrats on the great progress.

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Operator [39]

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Our next question comes from the line of Jeff Hung from Morgan Stanley.

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Jeff Hung, Morgan Stanley, Research Division - Equity Analyst [40]

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I guess now that you have a full quarter of sales and perhaps some weeks of sales since the approval in MDS. I guess any updates to your views on how the trajectory will progress over the coming quarters? And are you hearing any early feedback in MDS that modifies your views on adoption rates?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [41]

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Yes, Jeff, thanks for your question. So the first quarter sales of approximately $8 million is one that we're very, very happy about. Again, just to repeat that first quarter, it is in the U.S.-only and with the detail indication only in terms of adult beta-thalassemia patients requiring regular blood cell transfusion. And now with the second quarter having a full quarter with MDS approved in the United States. Looking forward now, as you saw also, we had a positive CHMP opinion, which gives us tremendous confidence for getting -- moving forward in an anticipated approval in the second half of this year for Europe. And our enthusiasm for the launch, and you've heard from Sujay previously as well, given the number of new accounts, the number of repeat accounts that are prescribing, that we continue to feel very optimistic about all of the launch metrics. We haven't given any official guidance. But what I can tell you is that as we continue to launch Reblozyl in this virtual world, we continue to gain more and more confidence on the traction that we're gaining.

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Operator [42]

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Our next question comes from the line of Paul Choi from Goldman Sachs.

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Kyuwon Choi, Goldman Sachs Group Inc., Research Division - Equity Analyst [43]

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My first question is commercial and specifically with regards to your interactions with doctors. I was just wondering if you can maybe comment on whether the number of interactions has increased since you have the MDS label now? And if in your prior discussions with potential prescribers was maybe some of the feedback along the lines of, I'm interested, but not so much for beta-thal, but come back to me when you have MDS. So I was just wondering if you can maybe just comment on the level of activity directionally or if you could quantify it that you and your partner have seen since the approval?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [44]

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Yes. Thanks for your question, Paul, and I will hand it over to Sujay. But I can tell you, absolutely, the number of interactions have increased because in many cases, in the majority of cases, this is a different prescriber base. So the overlap between beta-thalassemia prescribers and MDS prescribers is quite limited. But I will hand it over to Sujay to add some color to that.

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Sujay R. Kango, Acceleron Pharma Inc. - Executive VP & Chief Commercial Officer [45]

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Sure. Thanks, Habib. So clearly, as Habib pointed out, customer base is a lot larger in the -- for the MDS indication. So yes, we have seen more interactions. Now the caveat to that is these are all virtual interactions, right, because if we launched this drug -- the indication for MDS almost right on top of the COVID-19 pandemic starting to occur and shelter in place orders. So with that kind of a situation, almost all of our interactions are now virtual. So that comes with a little bit of challenge with regards to how -- difficult to contract this particular aspect with the prior sort of where you are in-person promotion. Having said that, right off the bat, we've seen uptake within large centers of MDS. So we know that there is adoption occurring, which is sort of at the end of the day, that's what you want to see, and we're starting to see a lot more of that. And also we've also seen interest in even virtual speaker programs. We've got also questions about how quickly we can get the product to them. All of these questions are coming about from these virtual engagements. I've been on several of these engagements, and you ask some sort of color around it. So they're asking us questions with respect to the patient population, the label, the indication that we have and the interest is very strong from these early interactions that I've had with physicians, which have been with my reps as well as BMS reps, are doing joint calls with our reps as well. So net-net, I see a very favorable engagement and interest with Reblozyl.

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Kyuwon Choi, Goldman Sachs Group Inc., Research Division - Equity Analyst [46]

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Okay. That's very helpful. My follow-up question, if I could squeeze one more in, please, just with regard to -- I know the PULSAR is -- data is still yet pending here in terms of the presentation. But can you maybe just comment on, do you plan any additional data or longer-term follow-up updates over the course of 2020? And then can you maybe just elaborate on how you're thinking about potentially incorporating the data down the road as part of your registrational strategy?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [47]

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Yes. Thanks, Paul. That's actually a really good question. And just to remind everyone, the PULSAR study had a 6-month primary endpoint, yet every patient had the opportunity to roll over into an open-label extension for an additional 18 months in the placebo arm of that study. We gave randomized 1:1 on low-dose and high-dose sotatercept. And just to remind everybody, once again, when we cut the data and share the top line results of PULSAR at the end of January, there was 97 out of 106 patients who were eligible to roll over into the open-label extension with the first patient being eligible around December of 2018. At the time of cutoff, every single patient that was eligible to roll over was still in the open-label extension. So we take a lot of comfort from that statistic alone for multiple reasons. And the answer to your question, Paul, yes, we do plan to continue to analyze this data set. And there will be more to come over the course of this year and next year in terms of sharing with you the updates, both from an efficacy and safety point of view. So thank you for your question.

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Operator [48]

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Our next question comes from the line of Kennen Mackay from RBC Capital Markets.

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Kennen B. MacKay, RBC Capital Markets, Research Division - MD & Co-Head of US Biotechnology Research [49]

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You've obviously made tremendous progress with sotatercept in PAH and really excited for the ATS data. Have you -- you mentioned 6-minute walk test, is that the secondary endpoint you see as the most meaningful? And secondary to that, obviously, PAH is tremendously exciting. Has this data reignited interest in targeting additional indications with Bristol outside of this pulmonary carve-out, something like idiopathic pulmonary hypertension?

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [50]

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Yes. So thanks for your question, Kennen. The reason 6-minute walk distance is highlighted is because it was our key secondary endpoint, Kennen. We, obviously, will have the conversations with the regulators in terms of what the appropriate end points would look like moving forward for our pivotal study. But what you're seeing is with 6-minute walk distance being highlighted is just merely the fact that it was our key secondary. Now with respect to sotatercept and the versatility of sotatercept, one thing to remind you is, again, is that our intention with sotatercept with respect to pulmonary indications, is to be able to develop and commercialize it on our own, that is our goal at this point within pulmonary and pulmonary hypertension. Anything beyond that, obviously, we would disclose if those conversations ever occurred at the appropriate time. But right now, the focus is really on Group 1 pulmonary hypertension, and perhaps opportunities to grow beyond that. But we're very, very fortunate that we were able to carve out the opportunity for sotatercept in pulmonary hypertension for Acceleron to develop and commercialize on our own.

Now with respect to idiopathic pulmonary fibrosis, this is actually a really good question because, obviously, you're asking because not only perhaps of the relationship of the TGF-beta superfamily in this particular disease state, but also because of the high unmet need. Now I did mention earlier to an earlier question from one of the analysts regarding ACE-1334. ACE-1334 targets specifically TGF-beta 1 and TGF-beta 3. Again, it has fast track designation. We have not talked about and identified that indication. But you can assume it's going to be an indication that has fibrosis as part of -- within rare pulmonary disease as being the real contributing factor to the patient's unmet need. And so within that group, obviously, is idiopathic pulmonary fibrosis, scleroderma, interstitial lung disease. So you make an excellent point, but there could be other solutions that could be targeting that area that we'll be looking at very, very closely. So thanks for your question, Kennen.

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Operator [51]

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At this time, I am showing no further questions. I would like to turn the call back over to Habib Dable for closing remarks.

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Habib J. Dable, Acceleron Pharma Inc. - CEO, President & Director [52]

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Yes. Thank you. So thanks, everybody, for joining the call today. As you've heard, we've had a very strong quarter. The fundamentals of our company have never been stronger. We're well positioned for a productive and successful year ahead. And as always, if you have any additional questions, please feel free to reach out to Todd, and I'm wishing you all a great and safe evening for everybody. Thank you.

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Operator [53]

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