Q3 2020 Acceleron Pharma Inc Earnings Call

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Good afternoon, ladies and gentlemen, and welcome to the Acceleron third quarter 2020, <unk> earnings Conference call. At this time all participants are in listen only mode. Later, we will conduct a question and answer session and instructions will be gone.

Even at that time.

Serving patients.

Beginning with our hematology program, along with our collaboration partner Bristol Myers Squibb, we continue to expand the availability of rebels, though with multiple new regional approvals across the globe.

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 rental yield sales of approximately $96 million.

This compares with approximately $11.1 million in royalty revenues from approximately $55 million of net rebel sales in the second quarter.

Any benefit from rebels, those and all the mechanism of action.

As you'll recall there still is currently conducting two additional studies with with patterns stuff after paying for third called independence for the treatment of anemia in patients with myelofibrosis Ah cancer like blood disorder.

We are looking forward to productive years ahead as we continue to build the list patterns that franchise with plans to report topline results from the beyond study and non transfusion dependent data thalassemia at year end or in early 2021 and from the command study and first line lower risk M. D. S in 2022 or later.

Beyond these three trials or partner, there's still Myers Squibb recently amended it stays free trial and myelofibrosis within Rebuck to include a safety cohort in combination with this powder stuff.

Adding to this and your investigators sponsored trial was recently initiated evaluate with patterns of treatment in combination with lenalidomide and patience with lower risk M. D. S.

Probably before the end of this year.

In addition to stellar we expect to initiate two additional faith to be trials Hyperion and xena in the middle of next year with the long term vision and establishing status up the polka ability and early and late stage therapeutic strategies respectively.

We believe the overall development plan will generate a breath of data to support patterns, that's potential as a future backbone therapy M. P. A H.

Royalty revenue from net rebels sales of approximately $96 million all revenue was derived from the company's partnership with Bristol Myers Squibb.

I've commented in the past we've continued to see both.

The accounts that have prescribed in the past continue to prescribe meeting that we are getting repeat orders from them and then we are continuing to add new accounts on a weekly basis as well now the challenge is for me to give you a specific as to break.

Thomas on for Marty Thanks for taking the question.

Yes.

75%.

Potentially decrease in office visits and treatment compliance.

So that's kind of one of the other concentrations that we are seeing as as it's constantly looked at this part of the equation. Okay hope that helps thank you.

Got it yeah. That's helpful. Thank you.

Great. Thanks for calling thank you.

Our next question comes from Carnival with Barclays. You May I proceed with your question.

Just began to close off on the follow on question with respect to.

The commentary yeah, we haven't really shared any details or with respect to you know what the percentages of the of the more prevalent population versus the those the earlier in there and as you know in their treatment journey.

That said I you know I think it's very clear that you know through Q2 and Q3.

We had a you know a very large portion of those patients that due to that pent up demand and over time, you know that those those patients will not be making up the majority of the patient group and what we're seeing now its TJ leading to these new accounts and these new patient starts earlier in their treatment journey I really.

I'm, starting now to to pick up and that are driving the the uptake in terms of new treatment and new scripts.

Now with respect to the covert state you know you're absolutely right I mean, it it actually does play a role in terms of you know a bit of an opportunity that we can perhaps you know play a role in terms of the you know mitigating that the huge need for trying.

Fusion burdens. During these these this pandemic and you're right and you know, but that said during Q2 and Q3.

Thanks Carter.

Thank you. Our next question comes from Geoffrey Porges with STB Leerink you May proceed with your question.

Hi, This is Charles on for Jeff.

Two questions if I may 1st on proposal just one too.

Sure thinking about the changing competitive dynamics in the I guess Mds space, particularly for online with the heavy inhibitors and the seed 47. So how do you position your product versus potential competition and for Sotatercept I just wanted to I guess, a little bit detailed question.

For the stellar trials seem to include more newly diagnosed <unk> ph patients.

In cutting down the portion of the patients on troubles Triple therapy. So how should we think about the data potential data comparing two pollo, so well that they that come out to be stronger or weaker depending on the difference in the patient base line. Thank you.

Yeah. Thanks. Thanks for your question, So why don't I hand, it over to Jay to address the question right now in seller and then maybe Jerry I cannot see the ended over to Sue for the rubble question. Yeah. Sure. So just as a reminder, stellar really was modeled after Paul sorry. So just to start there and is designed to look to saying we have not really.

Corrected 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 Paul Sorry, we had modeled double and or triple therapy patients in their overall, we're very pleased with the impact that we had on six minute walk distance when we looked at the totality of the data so kind of going into this.

Have a very good position in the market for serving the patient population that'd be a serving right now.

Thank you.

Thank you. Our next question comes from Chris Raymond Piper Salmon you May proceed with your question.

Hi, This is Nicole Nebraska on for Chris Uhm, Congrats on the corner and and thanks for taking the question I guess just on does does 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 b D deal in the near future.

Yeah, Hey, Nicole Thanks, Thanks for your question so.

I think what I would say is right now.

<unk>, let me look at you heard from Kevin in terms of our financial position you're hearing a lot from C. J in terms of the lunch with <unk> and then obviously all of the efforts with respect to so tired or something really building.

That portfolio of phase three studies to establish the tighter set the other backbone therapy M. P. H I feel that we have a tremendous opportunity to execute and to generate value for shareholders. If we really focus and.

Really you know focus on executing one okay, they're very admirable position in terms of having to 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 questions establishing a leadership position for <unk>. We're also exploring other areas.

Within pulmonary hypertension, which hopefully you'll hear a little bit more about next year.

Second thing I'd say is organically, we talked briefly last call lost earnings call about eighth at 234, which is is in a healthy volunteers right now, but that we hope to be able to go into patient 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, and where they're really focusing on a small molecule approaching target identification with respect to modulating gene expression <unk>.

Pathways and and and here, we're specifically you know locked up in agreement with them areas within very pulmonary diseases as well.

So we're gonna, we're obviously looking to continue to invest both organically and Inorganically and it's indeed, there is an opportunity that would make sense that would complement our strategy right, now and and and and establishing ourselves as a leader and very pulmonary diseases, we'd be always very open to it but we absolutely do not see.

The necessity have to execute anything in the near term.

Great. Thanks for taking my question.

Yeah, well thank you for the question.

Thank you. Our next question comes from Eric Josef J P. Morgan you May proceed with your question.

Oh thanks.

Just looking to beyond readout later this year early next year I guess, how should we be thinking about the the non transfusion dependent population as a proportion of a bit about overall.

Does beyond I guess, who is.

Does it help you access those patients.

[noise] itself, if debating are positive what would you be looking forward to it.

If needed.

Move forward with a a label expansion study for non transfusion dependent.

T D population.

Yeah. Thanks for your question, Eric So in terms of the the market opportunity to perhaps I'll take that and then I'll hand over to T. J 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 and this was on.

Planning to over two almost two years ago and Doctor couple. The name is speaking Preston or in D day.

And she's pretty adamant that the non transfusions dependent that'd be the thalassemia community.

You know has an unmet need for innovation such as such as rebels, though just as much of the transfusion burden patient do just because of the fact that there you know just because they're not tense using does not mean that they're not symptomatic and suffering from many many aspects of of the.

Disease. 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 U S and Europe that the non transfusion dependent population would get with double the existing target groups that we have with the transfusion dependent indication.

With Citigroup you May proceed with your question.

Great. Thank you very much for taking the questions I just wanted to drill down a bit on pulse or a little bit more as you mentioned you're going to have the 24 week Echo data at A.J. coming up if you could just help us put that put that data and in the clinical significance.

And put it into perspective with respect to the other endpoints that you've obviously ready already reported DVR and six minute walk distance.

More specifically, how impactful a drug can or rather what what.

How impactful could can separator set be with just the echo data.

In addition to indifferent to the PVR and six minute walk data that you've already reported.

Yes, so hey changed around the corner. So the details of the Echo data will move share when we get because it's publicly disclosed through A.J., but.

But just to kind of put into context, you know as we've reported out in the Nonclinical work things that we were anticipating or hope to see in the clinic would.

Thank you.

Thank you. Our next question comes from Kennen Mackay with RBC capital markets. You May proceed with your question.

Hey, guys. This is vikram on for Kevin. Thank you so much for taking precautions and congrats on the quarter.

Just following up on.

Read off beyond trial topline results I'm a good cushion is there any internal bar that you guys are looking at for response rates in this population and how long.

That might be different from the other bit of how can you population.

Great. Thanks for the question because.

Jamie if you want to take that please.

Yes, so I think for this trial because these patients are not regularly transfused in there the non transfusion dependent the primary endpoint 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.

Find results could 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 endpoint that will be the threshold and then following that the earlier questions is seeing consistency within the secondary endpoints, which include the patient reported outcomes. So we're getting close.

That trial has been running for a while and as you heard from them be looking forward to those results potentially by the end of year, if not early part of next year.

Got it. Thank you so much and I just have a follow up maybe taking a step back and thinking about other indications for revenue will build and how you guys are thinking about beyond.

Sorry, not be on trial, but the.

Currently approved indications and Mds or myelofibrosis.

Where are you guys focusing on your internal R&D efforts.

So.

So yeah. 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 her deep early on I think things and 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 we're combining with lenalidomide or Rob.

Limits. So I would say look forward to seeing more of this combination type trials throughout the spectrum of Mds.

Myelofibrosis study is starting and again you heard that BMS, who is actually now added this pattern to their own.

Jaktwo inhibitor for draft in it so we're going to start to see again build out around I think in myelofibrosis space and then there are other areas of indication you can anticipate because of our effectiveness in beta.

Beta Thal is seen as we've seen and believe potentially looking at for example, health metallocene yet. So there is a full lifecycle plan that's underway.

And again as we established a presence in the commercial space. It gets easier as the combination studies of approved products and you're going to see those come forward. So it 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 my questions.

Thank you.

Thank you we have now reached the end of the Q and a session I would now like to turn the call back over to Debbie for any closing remarks.

Great. Thank you operator, and good before we conclude I think the opportunity to thank everyone for joining us.

I hope that everyone continues to stay safe and well during this current cobot environment and I look forward to connecting.

With many of you at some of the upcoming virtual conferences. I mean, you guys 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.

So.

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