Acceleron Pharma Inc. (XLRN) CEO Habib Dable on Q3 2019 Results - Earnings Call Transcript

Nov. 06, 2019 4:40 PM ETMerck & Co., Inc. (MRK)
SA Transcripts profile picture
SA Transcripts
132.5K Followers

Acceleron Pharma Inc. (XLRN) Q3 2019 Earnings Conference Call November 6, 2019 10:00 AM ET

Company Participants

Ed Joyce - Director of Investor Relations

Habib Dable - Chief Executive Officer

Kevin McLaughlin - Chief Financial Officer

Todd James - Vice President, Investor Relations and Corporate Communications

John Quisel - Chief Business Officer

Sujay Kango - Chief Commercial Officer

Conference Call Participants

Yaron Werber - Cowen

Danielle Brill - Piper Jaffray

Mark Connolly - Credit Suisse

Eric Joseph - JP Morgan

Geoffrey Porges - Leerink

Yigal Nochomovitz - Citigroup

Jeff Hung - Morgan Stanley

Ed White - HC Wainwright

Leland Gershell - Oppenheimer

Paul Choi - Goldman Sachs

Kennen MacKay - RBC Capital Markets

Operator

Good morning, ladies and gentlemen and welcome to the Acceleron Third Quarter 2019 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 call over to Mr. Ed Joyce, Director of Investor Relations at Acceleron. Please go ahead.

Ed Joyce

Thanks and welcome everyone to our third quarter 2019 earnings call. The press release reporting our financial results and has clinical abstracts 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 for the call today are Habib Dable, our CEO; Kevin McLaughlin, our Chief Financial Officer; John Quisel, our Chief Business Officer; Sujay Kango, our Chief Commercial Officer; and Todd James, Vice President, Investor Relations and Corporate Communications.

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-K on file with the SEC.

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

Habib Dable

Great, thank you, Ed. Good morning, everyone and thank you for joining us today. So as you can see Acceleron had deep expertise across the TGF-beta superfamily with significant development in the three disease areas. This is really an exciting time for us with a number of near-term clinical and regulatory updates across our entire pipeline in the coming weeks and months. I’m extremely proud of the tremendous progress of all of our disease area teams.

Beginning with luspatercept, the USFDA and the European Medicines Agency are currently reviewing the BLA and MAA filings, respectively, for the treatment of anemia in adult patients with beta-thalassemia who require regular red blood cell transfusions and for the treatment of anemia in adult patients with myelodysplastic syndromes who are RS positive and require red blood cell transfusions.

With FDA granting priority review for the beta-thalassemia indication, not far away from the potential of our first approval of an Acceleron discovered medicine with the PDUFA target action date of December 4th, 2019. For the MDS indication, the FDA has set a targeted action date of April 4th, 2020.

Our commercial organization has worked hard preparing for this milestone and I am pleased to report that will be ready upon FDA approval to begin making luspatercept available to patients. In addition, regulators in the EU are expected to deliver their decision on applications for both indications in the second half of 2020.

We’ll continue to work closely with both agencies to advance this therapy toward approval. We believe that luspatercept could bring significant benefit to patients with these blood disorders by potentially eliminating or decreasing the red blood cell transfusion burden. This is a huge achievement for Acceleron and our collaboration partner, Celgene, and another step forward in delivering this innovative therapy to patients.

We are looking forward to another important and productive ASH Annual Meeting, a total of six clinical abstracts on luspatercept have been accepted and are now publicly available on ASH’s website. Key presentations include new and updated analyses from the pivotal MEDALIST and BELIEVE Phase 3 trials in MDS and beta-thalassemia, respectively, as well as interim results from our ongoing Phase 2 myelofibrosis trial.

I’ll now summarize some of the important data points included in these abstracts. Beginning with MEDALIST, which enrolled patients with lower risk who are RS positive. The updated analyses with treatment through the January 2019 data cut show that 47.1% of patients treated with luspatercept achieve red blood cell transfusion independence relative to the 37.9% response rate at week 24 seen in the top line data cut in May 2018.

So this abstract based on the more recent data cut, we calculated median duration of clinical benefit, which is defined as red blood cell transfusion independence for at least eight weeks and/or modified HI-E response. For patients responding to luspatercept, the median duration of clinical benefit was 83.6 weeks or approximately 21 months.

It is also important to note, that most patients in the trial achieving transfusion independence and/or erythroid response with luspatercept in the MEDALIST study had multiple periods of response with cumulative clinical benefit, durability superior to that of patients receiving placebo.

Moving to the key BELIEVE trial abstract. Median duration of clinical benefit defined as the time of first response of greater than and equal to 33% reduction in red blood cell transfusion units over any 24 weeks to discontinuation due to any cause in that episode, for luspatercept responders was 53.5 weeks as of the May 2018 data cut.

47 or 21% of patients receiving luspatercept had no loss of response within the entire study period. The average number of red blood cell units saved over any 24 weeks in all luspatercept responders was 6.55 units, and was 8.16 units for patients with a baseline transfusion burden of more than 15 units over the 24 weeks.

Lastly, the interim results included in the ASH abstract for our ongoing study in patients with anemia associated with myelofibrosis suggest clinically meaningful activity of luspatercept, including those patients receiving concomitant ruxolitinib.

Notably, in the cohorts including treatment with ruxolitinib in combination with luspatercept, cohort 3A 8 or 57% of the non-transfusion-dependent patients achieved the mean hemoglobin increase of at least 1.5 grams per deciliter.

In the transfusion-dependent group, cohort 3B, 6 patients or 32% achieved transfusion independence over any consecutive 12 weeks. Also, within this cohort, 10 or 53% of patients achieved a 50% or better reduction in red blood cell transfusion burden compared to baseline.

Moving to the cohorts for patients receiving treatment with luspatercept alone. In trial cohort 1, 3 or 15% of non-transfusion-dependent patients achieved the mean hemoglobin increase of at least 1.5 grams per deciliter.

In trial cohort 2, 2 or 10% of transfusion-dependent patients achieved transfusion independence over any consecutive 12 weeks. Looking at safety, results showed that a minority of AEs were grades 3 to 4 and severity, which is consistent with previous studies in MDs and beta-thalassemia.

With that, I’d like to move to our pulmonary program with focus on our PULSAR Phase 2 trial with sotatercept, which received orphan drug designation from the FDA and PAH in September. PULSAR is a randomized double-blind, placebo controlled study designed to evaluate the efficacy and safety of sotatercept in PAH patients.

A total of 106 patients have been randomized to receive placebo 0.3 milligrams per kilogram of sotatercept or 0.7 milligrams per kilogram of sotatercept in combination with standard of care therapies.

Following the 6 month primary treatment period, participants in the trial will be eligible to continue in the 18 month extension period. The primary endpoint of the trial is the change in baseline in Pulmonary Vascular Resistance, a key secondary endpoint is changed from baseline in 6-minute walk distance. We look forward to reporting top line results from the PULSAR trial in the first quarter of 2020 and preliminary results from our SPECTRA Phase 2 exploratory trial in 2020.

I’ll now turn to our neuromuscular program with updates from our ACE-083 trial in Charcot-Marie-Tooth Disease or CMT. Although where CMT is the most commonly inherited neurological disorder with the US prevalence exceeding 100,000 patients, there are no FDA treatments for CMT.

We’re currently conducting Part 2 of our Phase 2 CMT trial, which was designed to assess the efficacy and safety at ACE-083 versus placebo in a total of 40 patients, randomized one to one over 6 month treatment period, followed by a 6 month open label study.

The primary endpoint is the percent change in total muscle volume in fat fraction as measured via MRI, with secondary endpoints, including motor function tests such as time to walking test. We’ll also be evaluating disease-specific patient reported outcomes. We anticipate reporting top line results from Part 2 of this study in the first quarter of 2020.

And with that, I’d like to hand over the call to Kevin McLaughlin, our CFO to review the financials.

Kevin McLaughlin

Thanks, Habib. Good morning, everyone. Our cash, cash equivalents and investments as of September 30th, 2019 were $468.3 million compared to December 31st, 2018, cash, cash equivalents and investments of $291.3 million.

Based on our current operating plan and projections, we believe that current cash, cash equivalents and investments will be sufficient to fund projected operating requirements until such time as we expect to receive significant royalty revenue from luspatercept sales.

Collaboration revenue for the third quarter was $4.2 million. The revenue is all from the company’s partnership with Celgene and is related to expenses incurred by the company in support of luspatercept.

Total costs and expenses for the third quarter were $53.1 million. This includes R&D expenses of $37.6 million and G&A expenses of $15.5 million. The company posted a net loss for the third quarter ended September 30, 2019 of $45.4 million.

I will now turn the presentation back over to Habib for final remarks.

Habib Dable

Thank you, Kevin. This is a very exciting time at Acceleron, with multiple near-term clinical updates in all three of our disease areas. With respect to luspatercept, we and our partner, Celgene are preparing for a potential first approval and commercial launch in the US. Further, we look forward to presenting six clinical abstracts related to luspatercept at the ASH Meeting in December.

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

Question-and-Answer Session

Operator

[Operator Instructions] Our first question comes from Yaron Werber with Cowen. Your line is open.

Yaron Werber

Great, thanks for taking the question. And I was going to maybe start by focusing on the Phase 2 myelofibrosis data, Habib and give us a little bit of a sense. So if we look at the combination with rux sort of as expected look pretty good, and you’re saying between 32 and 57%.

In cohort A, you have a mean duration of response of 12 weeks and cohort B – in 3B you’re looking at 32 weeks. So maybe you can give us a little bit of an explanation, maybe if you can, what confers the difference in the durability of response between the two of them?

And then the second question, it looks like luspatercept mono did not do quite as well as combo, our patients on the mono arm were they naturally more advanced? Would they have failed rux in the past? I’m trying to understand why that data doesn’t look as good in the context of the prior sotatercept data? Thank you.

Habib Dable

Yeah, thanks for your question, Yaron. So as you can imagine, we’re pretty excited about this data. And you’re right in the important rux cohorts, we did see efficacy rates of 32% and 53%. That said, I do very much look forward to sharing with you more of the details in terms of the why and what some of our suspicions are at ASH.

But I’m sure you can appreciate that beyond this right now, beyond the – what’s in the press release, we really can’t share a lot more details and we do very much look forward to doing that in a few weeks at the ASH presentation, but I can tell you from this data based on some of the, I guess, the hurdle rates we had provided ourselves in the past we and our collaboration partners are very excited about this and very much looking forward to sharing with you our plans moving forward in a few weeks.

Yaron Werber

Okay. And can you maybe give us a little bit of a sense, is the data at ASH going to be more mature? Because you’re continuing to enroll patients or is it going to be based on this initial 74 patients?

Todd James

Hey, Yaron it’s Todd. Yeah so most likely the same similar number of patients, so not much more mature. But as you can imagine, across an oral presentation versus a very defined amount of orders that you can put in an abstract, there’ll be additional details in the presentation on Monday at ASH.

Yaron Werber

Okay. And maybe final question with respect to a headcount for luspatercept, for beta-thalassemia. Any new thoughts as to whether you’ve heard from FDA relating to a panel and whether you still potentially expecting one?

Todd James

Yeah. Hey, Yaron it’s Todd again. Yeah so what we’ve said historically is that given the new mechanism of action with luspatercept as erythroid maturation agent that always a high potential for an outcome here. We haven’t gotten into specific details on whether it would be one or both indications, so the teams continue to plan as if there could be one. But until the FDA would put something on the federal registry as being 100% happening, there’s – the company can comment further.

Yaron Werber

Okay, got it. Thank you.

Habib Dable

Thanks, Yaron.

Operator

Our next question comes from Danielle Brill with Piper Jaffray. Your line is open.

DanielleBrill

Hey, guys. Thanks for the question. So I’m not sure if I’ll get much of an answer here. But Habib, I’m just curious you mentioned the 25% to 30% response rate across cohorts to just by moving forward. How should we think about the opportunity now, should we only be modeling the 60% of the patients that are on background reps? Any color you can provide would be helpful.

Habib Dable

Yeah, so I understand your question, Danielle and thanks for that. I think right now and I guess you did preface your question why it’s really not the same much more than we are today. I will just simply repeat in this cohort with the rux combination, which we’ve always deemed to be the most important cohort for multiple factors. We are very, very pleased with these results.

We will continue to share with you more information on how these results will translate and a little bit more color behind these results in a few weeks, and very much looking forward with sharing you our plans in terms of moving forward in myelofibrosis with luspatercept.

DanielleBrill

Okay, thanks. And then I guess, you indicated that treatment durations would likely be shorter in patients, not on background rux as well due to the worse prognosis, you know, you're not providing detailed and data but head you're thinking on this involved at all.

Todd James

Hey, Danielle it’s Todd. Yeah, so there’s two patient types that would be eligible for monotherapy, luspatercept one would be a treatment of HI-E patients or patients that wouldn’t receive rux.

So this is most likely a more newly diagnosed patient, that patient would, as you could imagine, have a fairly good prognosis. And then there is the patients that you were more referencing, it’s the post-rux patients, those patients have a very poor prognosis, kind of on the lines of 18-month survival.

And as you could imagine, that first patient that I was talking about pre-rux, a good kind of 60%-plus of those patients will end up on rux eventually, and that’s on top of the rux prevalent patients today.

Those also patients that will be receiving rux in the future, that’s what makes the rux combination the more – the larger addressable patient population and the unmet medical need in the myelofibrosis kind of patient groups.

DanielleBrill

Got it. And will get the breakdown of the pre-rux for resistant rux patients sum at ASH, correct?

Habib Dable

There will absolutely be additional details within the presentation, but as you can imagine, the presentation itself hasn’t been fully finalized so it’s hard for us to commit to every single detail that can potentially be in the presentation.

DanielleBrill

I see, got it. Thanks for the question.

Todd James

Thanks.

Habib Dable

Thanks, Danielle.

Operator

Our next question comes from Martin Auster with Credit Suisse. Your line is open.

Mark Connolly

Hi, everyone. This is Mark on for Marty. Thanks for taking my question. I guess I was hoping to drill more into the response rate we saw and you know, thinking about a potential Phase 3 study, I was curious do you have a sense for how we should think about how a placebo patient would perform on the transfusion-dependent primary endpoint or the hemoglobin primary endpoint? Thanks for taking my question.

Todd James

Hey, Mark, it’s Todd. Yeah so there’s only one historic reference that you can really look at here. And again, always impossible to make cross study references as we can tell with the references that we’re looking at ahead of the MEDALIST data, always hard to say how it will play out. But within the Celgene pomalidomide Phase 3 study, the placebo RBC-TI in that study from a few years ago was approximately 16%.

And so that’s the best reference point. But again, not necessarily what would occur in an additional Phase 3 [technical difficulty] study. And there’s no reference or there’s no reference for hemoglobin response in study as one hasn’t been executed, but you can imagine it’s very challenging for a patient on placebo to get a random hemoglobin increase, it’s a very defined straightforward lab endpoint.

Mark Connolly

Got it, thank you.

Todd James

Thanks.

Operator

Our next question comes from Eric Joseph with JP Morgan. Your line is open.

Eric Joseph

Hey, guys. Thanks for taking the questions. Just a couple on myelofibrosis. I guess can you just remind us how you’re thinking about the relative sizing of myelofibrosis anemia is by severity, non-transfusion-dependent versus transfusion-dependent? And I guess in terms of looking to the next steps, do you have a sense of what a registration endpoint might look like in the transfusion-dependent population with highlighting mean hemoglobin rise here as one of the endpoints in the Phase 2 study. I guess it’s now sort of I guess what additional feedback you look to get from either regulators or a position to kind of build consensus on what a registration endpoint might be in transfusion-dependent patients? Thanks.

Todd James

Hey, Eric it’s Todd. Thanks for your questions. Yeah so the way we generally view the patient breakdown based off of our research is within the prevalent pool about 60% of patients are receiving ruxolitinib and whether they have a baseline transfusion burden or not at that time, they will eventually, a majority of them will eventually have a transfusion burden, and given the mechanism, it ends up a large majority of them have it a transfusion burden, and a minority of them are anemia-only.

And that’s likewise with the non-rux patients at diagnosis you’ll see maybe a third of patients have a transfusion burden and just a year after diagnosis, you’ll see that getting closer to 50% and given that how the disease progresses, you’ll see that number increase pretty steadily over the patient’s disease which with a general median survival of kind of five plus years in this patient population.

As far as potential Phase 3 endpoints, if we would go ahead with a transfusion-dependent patient population, they’d be fairly similar to what you saw here in the Phase 2 trial, most likely RBC transfusion-independence over any consecutive 12 week period over the primary treatment period.

The secondary endpoint that we used in the Phase 2, a proportion of patients that are seeing at least of 50% reduction in transfusions that would be a high probability of another secondary endpoint beyond that if we are able to commit to a Phase 3 in the future as of today, we continue to plan, we’ll be able to give more of those details once we finalize the protocol.

Eric Joseph

Got it. I did say transfusion-dependent I just meant to say non-transfusion dependent. If you were to move forward in the non-trans with a Phase 3 protocol and the non-transfusion kind of population, how should we think about registration endpoints there?

Todd James

Yeah, so we would need to talk to health authorities to finalize what that would be, given they’re not on transfusions obviously, some kind of transfusion endpoint would not be appropriate. So it probably be either potentially hemoglobin increase alone or hemoglobin plus patient reported outcome measure kind of a combination endpoint.

Eric Joseph

Great, thanks for taking the questions guys.

Todd James

Thanks, Eric.

Operator

Thank you. Our next question comes from Geoffrey Porges with Leerink. Your line is open.

Geoffrey Porges

Thank you very much and appreciate taking a couple of questions. First, could you confirm when the data cut off will be for the updated data? Because in MF, because I think you said May 10th was the data in the abstract.

And then could you just talk a little bit more about the population that the 40 patients, I think, here who were not on concomitant rux. I think you be unwilling to disclose the proportion that were pre-rux and post-rux, but presumably you might still have an opportunity in the pre-rux patients, but doesn’t look like in post-rux.

And then could you talk a little bit about the update from the MDS pivotal trial, it looks as though you said a median total duration of clinical benefit of 84 weeks, but that’s in the responders. So can you give us a sense of the total duration or the median total duration of a treatment across all the patients in that study, because clearly, it’s just the subpopulation who got to that 21 months? Thanks.

Todd James

Hey, Geoff it’s Todd. Yeah, so we’re not – given that the data cutoff date for what will be provided in the presentation isn’t included in the abstract. Just like everything else that will be presented, it remains under embargo. And so we’re not able to provide that detail. Likewise, with the breakdown of patient baseline characteristics within the monotherapy, that remains under embargo till the presentation.

As for duration of all patients receiving luspatercept in the Phase 3, I believe that’s also included in the abstract. And let me just get you up here for you. So median treatment duration for all patients was 50.9 months – weeks, sorry weeks. And then for the clinical benefit for responders, which when most people model, they mostly model the responders is the 83.6 weeks or the approximately 21 months. So that’s a nice update relative to what we presented at ASH last year.

Geoffrey Porges

Great. Okay, thanks. I’ll wait for the presentation.

Habib Dable

Thanks, Geoff.

Operator

Our next question comes from Yigal Nochomovitz with Citigroup. Your line is open.

Yigal Nochomovitz

Yeah. Hi, guys. Thank you very much for taking the questions. If we could move to and talk a little bit about sotatercept in the PULSAR Phase 2 trial. I had a question there in terms of your assumptions for treatment effect. Obviously, your study in sotatercept in combination with standard of care which usually includes the combination of background therapy, ERA/PDE-5 prostacyclin, usually two or three background therapies.

And you’ve shown some very nice preclinical work in the Celgene hypoxia model that sotatercept does a better job on pulmonary arterial pressure reduction as well as the reduction in right heart failure in comparison for the single agents, the ERA/PDE-5 prostacyclin.

But I’m wondering if you’ve done any additional work, I’m looking at how sotatercept would perform in addition to those mechanisms in a combination setting, because you’re just looking – you’re comparing two single agents, so that would maybe more reflective of the real world experience. So if you could just comment on that aspect and how you develop confidence in the design of the Phase 2 study? Thanks.

John Quisel

Yeah. Hey, this is John Quisel. So I think the second half of your question was relating to preclinical efforts to look at combination therapy if I heard that correctly.

Yigal Nochomovitz

Right, right.

John Quisel

And you’ve correctly said we presented data showing side by side and combination with sotatercept versus sildenafil or sotatercept on top of sildenafil. And as you summarize sotatercept reproductively outperform sildenafil very convincingly. And then we can see combination benefit on top of sildenafil. We have not presented data looking at double or triple combinations in the preclinical setting.

And as you also correctly stated, those are patients in the trial, so the trial does allow enrollment of that patients who were on a single vasodilator or dual vasodilator or even triple, presuming that the prostacyclin therapy is stable at the time of enrollment.

To summarize the reason why we think sotatercept is a very distinct mechanism, looking at rebalancing the BMPR2 signaling pathway, which we believe has the potential to be disease-modifying that really changed the way the vascular remodeling happens in those patients.

And our sense is that we don’t anticipate tremendous difference between having multiple layers of vasodilator in combination with sotatercept, but we have not done that or presented that data in a conference at this point.

Yigal Nochomovitz

All right, thank you very much.

Operator

Our next question comes from Jeff Hung with Morgan Stanley. Your line is open.

Jeffrey Hung

Thanks for taking the question. You have a pediatric Phase 2 for luspatercept beta-thal patients that recently showed up on clinicaltrials.gov. Can you talk a little bit more about this study and then remind us of your strategy for moving into the pediatric population for different indications?

Todd James

Hey, Jeff, yeah, it’s Todd. Yeah as everybody knows, given that beta-thalassemia is an inherited disease. You know, these patients are becoming symptomatic very early in life. Some patients start a transfusion burden as early as two years of age.

And so what we’re doing with this Phase 2 trial along with our partner, Celgene is, this is our first study in the pediatrics setting and so we’re looking across multiple age groups and multiple doses across approximately 48 pediatric subjects and we’ll be able to generate some nice data there to hopefully be able to move forward in the future with additional studies in pediatric thalassemia.

Jeffrey Hung

Thanks.

Operator

Our next question comes from Ed White with HC Wainwright. Your line is open.

Ed White

Hi, guys. Thanks for taking my question. I just wanted to ask a quick one on ACE-083. In the CMT patient population, I’m just wondering after stopping the trial and the development in FSHD, if you can just briefly explain why you’re continuing on or have confidence in the CMT population?

And then also do you have any other TGF-beta protein superfamily drugs in the pipeline for muscular or neuromuscular disorders? I see you have one preclinical for the pulmonary disease, but just wondering if you have anything else in muscular or neuromuscular? Thanks.

Habib Dable

Yeah. Hey, Ed this is Habib. Thanks for your question. So obviously we were disappointed in September when we shared the FSHD results with ACE-083. Again, asking why would we be continuing with the CMT program or where is the confidence coming from.

So I would say a couple of things. First of all, we were very deliberate in terms of these two studies, one being myopathy, one being a neuropathy such as CMT. And I also would remind you that and even in the FSHD group, we actually did see double-digit increases in muscle mass. But unfortunately that did not translate into a functional benefit, which is why we decided not to move forward.

CMT trials has already been fully enrolled. And as we’ve discussed previously, it will be reading out in the first quarter of next year. So it’s really at the tail end of that study. And so we do look forward to unblinding that study in the first quarter of next year.

And CMT is very much a different disease, I think based on the disappointment with FSHD, whatever percentage of success you add on translating muscle mass increases into a functional benefit, probably have gone down now for CMT. So I would say we’re cautiously optimistic now.

But that said, CMT is a different disease and being a neuropathy, you have damaged nerve leading to the atrophy unlike a toxic protein, which is associated with the atrophy in FSHD. And it is also a much more focal and distal disease, which may lend itself to a drug which is locally injected into a dominant muscle. And so again, looking forward to seeing how that study reach out in the first quarter.

Regarding other products in our pipeline, no, we are not investing in any products at this point in our in-house pipeline in neuromuscular and we’ll very much be looking forward to the CMT data, which I’m sure will help define our path forward in neuromuscular disease. You mentioned ACE-1334. Just to be clear, again, that asset is target to TGF beta 1 and 3 and we are looking forward to talking a lot more about that next year as our next pulmonary asset coming – that will be coming to the clinic.

Ed White

Great. Thank you, Habib.

Habib Dable

Yeah. Thanks, Ed.

Operator

[Operator Instructions] Our next question comes from Leland Gershell with Oppenheimer. Your line is open.

Leland Gershell

Hey, good morning guys. Thanks for taking my question. Just back on the myelofibrosis, not sure if you can answer this one either, but patients on ruxolitinib certain facts then we’ll often start to lose efficacy or become effectively a failure population. If you could characterize for us the population in your Phase 2 that was combo with rux to what extent those patients on rux prior to also being put on this [indiscernible] perhaps a waning of activity or approaching failure versus those who had already been – who had remained stable and this was an add-on with potential increased efficacy? Thank you.

Todd James

Hey, Leland, it’s Todd. Thanks for your question. Yeah, unfortunately given that isn’t within the abstract, we aren’t able to get into that amount of detail. Now all that we can say it’s based off of the trial design that patients were on stable rux dose when entering our trial.

Leland Gershell

Okay, we’ll have to wait for the ASH presentation, then. Thank you.

Habib Dable

Great. Thanks, Leland.

Operator

Our next question comes from Paul Choi with Goldman Sachs. Your line is open.

Paul Choi

Hi, good morning, everyone and thanks for taking our questions. Maybe just staying on the topic of MF, as you think about a potential pivotal trial design, I was wondering if you can maybe provide some color on clinician feedback and buy in with regard to the 1.5 grams per deciliter change as a clinical benchmark relative to some of the guidance or guidelines in society documents like the International Working Group that have looked for two or higher and just that your thoughts there as a potential input for clinical trial design here going forward?

Todd James

Hey, Paul it’s Todd. Yeah so today unfortunately there’s nothing for these patients. And so an agent and given the unmet need with anemia and that almost all patients have some form of anemia throughout the course of the disease and over 50% of them have moderate to severe anemia and most likely leading to a transfusion burden.

Any agent that shows activity here would be very much welcomed by the community and patients when we talk to docs about the unapproved agents that they use in the space, they get in the range of – and they don’t exactly define what they consider a response, they get a teens percentage response with EPO in these patients with pomalidomide, the Celgene round study had a 16% response rate, and so these are response rates in the teens.

And so what we’re showing whether it’d be in a hemoglobin response and the anemia-only patients or in the transfusion independence response and those cohorts are fairly significant activity relative to what doctors are using today in the field.

Paul Choi

Okay, thanks for that. And maybe just on the commercial side since you guys are approaching a product launch here in the not too distant future. I was wondering if you can maybe provide a little more color on how you and your partner will be sort of targeting or copromoting at potential accounts? Who goes in first? How do you share and sort of divide these territories and accounts? Maybe a little more color that would be helpful, thank you.

Habib Dable

Yeah, sure maybe a Sujay Kango, our Chief Commercial Officer can add a little bit of color there for you, Paul.

Sujay Kango

Hi, Paul great question and thanks, Habib. So as you know, we are complementary and synergizing our efforts with the copromotion partner, Celgene who has a tremendous leadership in hematology.

So they have the breadth of covering the entire nation and as we have communicated in the past, we have a total of 20 field-based people about 16 reps and the leadership. So as part of that we are focused largely on optimizing the high target accounts within the beta-thalassemia community, which would be likely first approval.

So with that in mind, what we tend to do is, we’re focused on really sort of going into the higher sort of desired targets where there’s going to be a larger opportunity for the patient population, and we are coordinating all our efforts jointly alongside.

So our reps have completely coordinated in knowing the Celgene reps and we are planning account plans together and through this kind of collaboration at a grassroots level, we know we can actually do a better job together in addressing the customer needs in an efficient and a timely manner, so we are able to optimize their needs and no patient would be left behind.

So that’s the approach we’re taking. So we are sort of targeting on the larger accounts alongside them and then they have the breadth so they can cover the rest of the country as well. So we’ll be able to cover everyone that’s necessary. Thanks. Hope that answers the questions.

Paul Choi

Yes, got it. Thanks for taking our questions.

Habib Dable

Okay.

Operator

Our next question comes from Kennen MacKay with RBC Capital Markets. Your line is open.

Kennen MacKay

Hi, thanks for taking the question and squeezing me in here. Two questions. One on the myelofibrosis data and then a separate one on sotatercept in PAH – in the MF data. I was just wondering if you could discuss the intra-patient variability a little bit, it seems like that might play a part in the patients who were achieving greater than 1.5 grams per deciliter at any time point which is sort of more of a best response versus patients who are sustaining hemoglobin elevations?

And also on that note, I was wondering if doses had to be held in that trial if hemoglobin did rise to high which was something we’d seen in the prior IST, but MD Anderson had done with sotatercept.

And then just a quick follow-up on sotatercept in PAH. I was just hoping you could discuss a little bit more the decision to start the neuro Phase 2 SPECTRA trial and some of the differences versus PULSAR in which you’re hoping to see. It seems like both are on top of the standard of care but SPECTRA has with intra-patient dose escalation from 0.3 to 0.7, and was just hoping you can remind us on rationale here and rationale for the different primaries of PBR versus the [indiscernible]? Thanks again and looking forward to seeing the data at ASH.

Todd James

Hey, Kennen it’s Todd. I’ll start with your myelofibrosis questions and then I’ll hand it over to John Quisel for your question on the difference between PULSAR and SPRECTRA trials. For the two ways of being able to assess hemoglobin in the Phase 2 trial, I can’t get into the details to intra-patient variability.

The two endpoints are very distinct, but those are deemed acceptable from – of a response rate perspective for you might be remembering beta-thalassemia and MDS which we had at the positive Phase 3 data in at ASH last year that we were using.

And in the Phase 2, we were using mean hemoglobin as the response criteria, whether it’d be grabbing a 0.5 gram or 1 gram per deciliter depending on the patient population. So this mean hemoglobin though not as stringent as every assessment, is still a huge benefit to patients.

And then what was the second part, excursions. So as per the protocol, if a patient has a hemoglobin excursion, there is either a dosing down or down hold. But we’re not able to get into what that data actually looks like or if it happened at all at this point, because that isn’t included in the abstract today. So if it’s included in the presentation at ASH we’ll absolutely be able to discuss that at that time.

Kennen MacKay

Got you. Thanks, Todd.

John Quisel

Yeah, hey thanks, Todd. Yeah so back to your question on PAH and the two trials we’re running. So there are two – there’s a PULSAR trial and the SPECTRA trial. The PULSAR trial is the placebo controlled, blinded study that was designed for 100 patients, we enrolled 106. And we’re testing placebo 0.3 mgs per kg dose and 0.7 mgs per kg dose. And the primary endpoint is pulmonary vascular resistance with an important secondary endpoint of 6-minute walk.

So PULSAR is designed to put this product on a map of familiar endpoints that have been used for all the drugs that have come before primarily vasodilator type agents and give us a strong basis for comparing how sotatercept is performing one place on top of that standard of care.

SPECTRA is a trial we designed to really try to elicit and explore new endpoints and new potential effects of sotatercept and the reason for that is because of the potential disease modifying mechanism, quite distinct from the vasodilators that came before. We think that there’s potential to see different type of activity in the patients. And so we wanted to have a chance to look at some additional types of effects the drug may have.

So there we’re doing iCPETs, looking at exercised capacity with that technology and we’re also looking at cardiac MRI to have really the best possible way of visualizing the effects of sotatercept may have on right heart function then morphology. If you recall in the some of the animal data, we presented we’ve seen reversal of right heart enlargement after 4 weeks of treatment in the animal model.

So SPECTRA in order to accomplish that exploratory goal, we designed to be open label. We’re enrolling roughly – that it’s designed to enroll roughly 20 to 25 patients. And without the placebo control aspect of it, we felt that it would be desirable to have every patient capable of getting up to the 0.7 mg per kg dose and we felt it would be useful to explore tiering up to that dose by starting at 0.3 and then moving to 0.7 till we get a very nice look at how each patient is responding in each dose level.

Kennen MacKay

Got you. That’s super helpful context. Really appreciate all the caller there and again, looking forward to seeing the team at ASH.

Habib Dable

Thanks, Kennen.

Operator

Thank you. This concludes the question-and-answer session. I would now like to turn the call back over to Habib Dable for closing remarks.

Habib Dable

Okay, great. So thanks everybody again for joining our call today. Wishing you all a great day and very much looking forward to seeing all of you at ASH where we’re looking forward to sharing much more details on our programs. So with that, have a great day.

Operator

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

Recommended For You

Comments

To ensure this doesn’t happen in the future, please enable Javascript and cookies in your browser.
Is this happening to you frequently? Please report it on our feedback forum.
If you have an ad-blocker enabled you may be blocked from proceeding. Please disable your ad-blocker and refresh.