XLRN - Earnings call transcript Q1 2021

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Operator

Good afternoon ladies and gentlemen and welcome to the Acceleron First Quarter 2021 Earnings Conference Call. [Operator Instructions] As a reminder this conference call is being recorded. I would now like to hand the call over to Ms. Jamie Bernard, Associate Director of Investor Relations at Acceleron. Please go ahead.

Jamie Bernard

to quarter everyone earnings welcome and our XXXX first call. Thanks & results release are to Investors our reporting press www.acceleronpharma.com. in website available page at corporate our the webcast The today's financial addition of for the on Media presentation

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

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

to adjusted now Appendix today's several to non-GAAP to addition In GAAP the turn measures the on Habib of in press presentation. our most at over exclude that measures financial With release items. to call which and website these measures today's CEO. comparable today's discussion would Reconciliations financial to non-GAAP available certain are of include I will references the are like Dable, our

Habib Dable

elaborate Despite, with to challenges the and we progress plans the planned shortly. off pulmonary all expansion future us Phase meaningful our hypertension along the brought Phase a continued with Phase today. joining in ongoing trials are positioning solidified at arterial and PULSAR for by thank pulmonary great I patients XXXX, for trial our last many REBLOZYL. execute growth. I'll Jamie II pandemic, to or clinical within you clinical from program year you afternoon high III on for initiated pleased say good III registrational PAH, commercial level to reporting our STELLAR of the trial multiple for Thank in that and positive and start program other on sotatercept continues sotatercept results the in am Since trials and achieving well a which us in team we

pulmonary to disease rare beyond commitment extends sotatercept. Our

developing interstitial SSc-ILD rare connective sclerosis-associated lung leading ACE-XXXX patients scleroderma. in the are systemic disorder of We and systemic or death a disease tissue cause with among

study to more upcoming hear expect in this months planning ACE-XXXX currently year later initiate can the patient are this You II as in expected about a Phase we to Ib/Phase population.

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from discovered results clinical published highlights Journal PULSAR we of the significance PAH. in England New published raising an are awareness the of in results the as of Xrd XX Acceleron III marks past sotatercept in for milestone with while Medicine. therapy time trial advancing the such Medicine been of sotatercept our results have Phase in program. patients Journal journal that This our England these also PULSAR of prestigious II months New the the appear month, a Phase last data, Just trial Having

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Our III registrational patients. known open STELLAR Phase enrolling trial and as is

randomized a is from versus the comparing efficacy patients sotatercept endpoint or placebo-controlled and in change XXX double-blind of safety As Class approximately WHO with in distance X-minute Functional walk PAH. weeks. placebo a XX STELLAR primary The study is baseline II reminder III at

we IV intermediate two also sotatercept ZENITH add III in become in stages that from all to with or mechanism unique with trials high-risk of for program. WHO to will are PAH. of HYPERION PAH additional Results in who our will newly therapy our hematology believe patients populations with disease. functional are Phase turn patients our potential diagnosed patients backbone disease understanding a studies and across like I'd We expanded of class And preparing its sotatercept's patient validate for to with III to and Phase

year our commercial new XXXX. reported remain in the beginning strong with of REBLOZYL and during had we starts pleased uptake first call the launch the a and earnings As sales patient in year-end of we

with million in of sales in first quarter resulting By million for fourth royalties million For net $XX $XX.X in $XXX in the the $XXX million. were BMS Acceleron. of comparison quarter approximately sales approximately reported royalties approximately REBLOZYL net approximately

States. in the patient new the to underlying from was from quarter-over-quarter demand patient United transition launch the in the to the slight which to contributing continue factor We sales initial largest earlier group bolus in decline

patient been the penetration launches the decrease countries to the has with States. year through our launched volume hematology States in their pre-COVID Germany the Squibb We the the coverage. in across new compared to overall United existing there is significant multiple indications brand BMS expects underlying in Outside new last reimbursement opportunity patients Austria demand globe partner as following believe journey of well further this long-term Bristol-Myer in grow United there into going an earlier in treatment and MDS REBLOZYL Additionally, is third the where of year. launch level. of in quarter within

by and work of multiple REBLOZYL's may the the medicine we regions approval patients trials results in launch luspatercept MDS in REBLOZYL associated half with presenting the including June to this with in bring underway adult from that to of achievement to and in lead increase beta-thalassemia first-line by BMS anemia been in overall non-transfusion-dependent BMS, outside X and Phase are launches visits with of in the the progress COVID-XX patients return has and we forward of to the independent additional diseases MDS following important Additional Alongside and end myelofibrosis. global continue an markets look We evaluate BEYOND a to US vaccination to lower-risk myelofibrosis X novel from trial X action. will Phase trial two on benefit year. require summer patient Phase biggest of the blood for rates, second inhibitor initiated we top-line XXXX reopening the are who later. this in COMMANDS cell who in physician a mechanism the development in growth recently X and in months trial And REBLOZYL of or the Phase patients this to-date from expect our expect red activities, following JAKX transfusion. with results continued end populations

more anemia, peak to current potential in indications billion. sales REBLOZYL with continue annual $X associated future success of of Assuming we and than estimate

I looking Research pipeline June planned at rare virtually upcoming very forward Finally, to across place ongoing take Day our which pulmonary trials XX. updates our Development our & providing disease on and on am numerous will much

And that from leading with of with McLaughlin, Acceleron's our you Kevin look information detailed of the Please a more but robust are pulmonary to plan setting. and to program would you and on connecting the person, nevertheless a Kevin? physicians management many forward could in We senior to I contributions wish your soon. in that, it looking have like call review the for team. with to We events CFO financials several members take virtual over be be the questions. hand available we'll to then

Kevin McLaughlin

$XX.X opportunity to includes review Habib. $XX.X results like Bristol-Myers were from million expenses for for afternoon, royalty equivalents the our XXXX All I'd first GAAP basis, cash for and total loss cash, the With net a I'd On the was of to this our in million partnership On summary the company's to for take $X.X to quarter basis with Non-GAAP net $XXX.X sales and cost first press were approximately issued the Squibb. Thanks, of quarter everyone. REBLOZYL. first XXXX million. was revenue briefly investments. costs was for adjusted the $XX.X refer We Operator? with of financial share call of from was quarter million. and of today which items. that Good revenue the few to GAAP for million expenses earlier a you XXXX a million. $XX.X $XX.X and quarter of first quarter first a the like derived of company's Non-GAAP total costs the net $XX.X open ended quarter XXXX and Revenue release first revenue questions. million, loss million.

Operator

comes Yaron first of question the Your with Instructions] [Operator Cowen. from line Werber

is line Your open.

Yaron Werber

Maybe you're any online Germany summer and half. Thanks the fast of into my ones you. just which of that over, the how now question. drives come expect In or others? the couple are going curious then you for that come so in have visibility And that secondly you. faster in just the me, for sounds some Austria what cadence COVID excuse expecting you specifically -- -- Thank the than the much it of more other is when that's Europe, Hi. to a countries second of are the and online? a do second US Is taking thinking you Yes. half? And do questions rebound launched to Habib resolves

Habib Dable

Yaron. Great. Thanks your for question,

the those been And and driven partners respect our much very, uptake Austria Europe with the visibility ex-US two by the launch terms last and first as the in very question Germany. with markets. of in just two from heard the been initial conference you countries call week we've So pretty on to has pleased

start start think kick to seeing starting full to I in. that you this going countries you XX our second the start and Countries been can months that main BMS guidance expect the post-approval and to to Yaron of some seeing in staggered half in onboard you're Typically reimbursements really in year some of XX partners markets. basis Germany France, in of can reimbursement with that's as what a that have is is half consistent you to expect months on when Belgium, like the coming Austria countries. are of Nordic the Italy well. the year And at providing Netherlands, addition the second in Europe

of much that And point terms happening going start And the course. launch specifically to starting of we're things quite these to global of of come the are to opening markets some view that forward And again, year? this of that to more driving And in will And being the US, to on those face-to-face countries in an basis. to launch. I new that's couple it the believe success contributing And your to a inflection patients very of But the going contributing do physicians of see has driven more ability is obviously, second we're And sales with be the by we second their to are be of secondly, so see having representatives what's really countries access. a that come there's from with the the half start continued elements in nicely question a ex-US part on in segues as that what is as different that starting significantly. And contribute really lot into vaccination believe a starting success point obviously to lot the brand. in. up the is increase then of overall more rates. play in to of of frequent frankly, the looking to of having

pre-COVID visits heard new Yaron at patient XX% that seen You have may space last BMS are population, specifically, have to now. in decrease the actually in noted MDS week, partners to XX% from in levels the that MDS about our they they've a where

most patient growth, the subside really see new this virus a rates of half metrics what a transmission visit really we're and when is that mix. to to going And hoping vaccination as also normal MDS hopefully this space. to return and so second to And that patient we're as it in importantly, a going to comes continue significant in growth in but be then Yaron is driver finally the the return

QX, these in Medalist of our just heavily transfusion do not burden. approximately so dropped study frankly patients patients population were quite these treated Now high the heavily the the that did And bolus being we in that that transfused well patients treated the out. patients had patient XX% MDS something were of were in waiting for and were really even

the population. close were we almost XX%. half in QX, to definitely to In would about drop for on-target number that expect levels persistency XX% So the to what saw we nothing

we much the that bolus. over and quarter that basis majority REBLOZYL growth. so or pretty end group really tail the be not MDS going population surrogate are will at a on the target be be believe if for of the driving that on so the within And to long-term of the next we'll And that all

when drug remain being very signs to half progress the I obviously to as we drug our you very together to very, So we're $X management growth. see And indeed some the going have and that now this seem at said the of day prospects starting a opportunities end again, this with, some we significant of all successful. yes, long-term billion-plus is the put contribute believe of And -- life that to cycle on confident. second be bullish very still then we're

Yaron Werber

Thank you.

Habib Dable

Yaron. you, Thank

Operator

Your comes Wolfe question next Akash line the with from Research. of Tewari

is line open. Your

Akash Tewari

Thanks so much. guys. Hey,

interesting placebo not PULSAR? the Thank an crossover, That data. maybe range? to the six that and versus three concern treatment on months XX. why was improvement background kind to range do ex XX-meter really on placebo this versus the had, between said, think more you imbalance the average looking sign of a XX any additional So true in treatment and was arm the one walk I think, improved six-minute in to was congrats initial we of XX-meter of Is It a reduction marked updated PULSAR there week see at the distance phase? Additionally, is kind XX sotatercept treatment to the improvement background the patients in starting there within to treatment in you.

Habib Dable

our your Dr. for to going that of I'm Akash. Development, Yes. Thanks question to hand Backstrom. and over Jay Research Head

Jay Backstrom

you Yes. Thank for the question.

total -- just everybody again, respect take meters. walk change distance, what for So, to a came they the same occur they protocol, in didn't across so been in the the change respect on in no, about have expect would to it's to per the that medications, from on. that in look with with medicines With from trial if baseline XX six-minute you change

the you cohort in SPECTRA I talked we're that XX data to And to it's the going for updated opportunity do in we look we XX-meter before I think If that range. that at present some later have XX-meter about the to add range. think in where on in range. you the very change programs on in frankly plus no baselines been upfront have XX-meter the the of look are setting from conducted And the top therapy if that patients even of across

data the So background double/triple I are us for six-minute And walk really things STELLAR against study. really our therapy. of the nicely think up distance overall very sets strong

Operator

you. Thank

Carter comes question the with Gould from of Your Barclays. next line

is open. Your line

Carter Gould

I prior market I multi-component to background a PULSAR? just question. for following question. presentation all ATS color Are in guess, would And we And Good just metrics on additional the also Any therapy? of guess, for by expect I two front going Habib, me, clarifying appreciated. I questions get can results we in be guess, the And guess the color the out guys. broken additional on afternoon on Great. also what changes that then dynamics. improvements? maybe Habib, the thanks

XXXX. COMMANDS, terms believe of the we completion line In updated think June time the got for primary I that noticed

year-end you. Appreciate You're for readout wanted clarity XXXX? Just about talking still there. Thank it. in some the

Habib Dable

then I'll question continue. for of rest one the Thanks hand the and it COMMANDS I'll the Carter. to for tackle right Jay now

what respect tell the the or XXXX end can same of COMMANDS, So is guidance with to that remained I you has later.

Carter. our definitely So be able the that is guidance there it on our goal in late but half to that none to changed XXXX -- first get XXXX, -- that of has

So of Jay with rest I'll respect to over the again the hand question, to please. it you

Jay Backstrom

Yes.

up week the so to Carter, is So going. here the still XX trial is update

around you that effectively That by as data, and more NT-proBNP improvement. be the see have at we broken can Class on granularity so see well will the Functional safety the you'll the out presentation walk data just focus additional update of WHO because And safety six-minute as dose.

be an ongoing an will that not we that the So this it's results, a but event at still opportunity be will then future present focus here. complete to ATS. trial Again, and have

Operator

you. Thank

of Brill Your the question next comes Danielle Raymond James. from line with

Your line open. is

Danielle Brill

Thanks maybe conversations the guys. XX in months? follow-up, And of the months, just maybe seems six-minute ZENITH in trial much at comment how investors either extending updated over then walk primary recently or with the seeing HYPERION assessment question. on in XX-week any and selection your why improve you. you magnitude primarily the context selecting have had design on to Habib I've some primary a and months? to Hi, talking Thank benefit you as endpoint do endpoint you're confident the six thoughts out the at primary time. so data endpoint Can about of and for six

Habib Dable

with off how continue well are also but XX, WHO in see your that week we to only way Functional I'll Danielle, as for walk outlining by as in NT-proBNP by pleased start we improvement and the not at Class. six-minute question Thanks Yes. maybe

looking approximately who XX%. kind at of at months to had when had patients everyone, we XX that at primary Just a number of XX% now improvement the class is remind six at versus were we weeks endpoint where functional

improvement to in And very we you're disease continue right, very, see across so pleased. to of a progressive endpoints concordance a are

see confidence I'll is and something of as And to we behind note those thinking some a timing very over of so and the Jay improvement, going terms why to watch obviously very, you rightfully continue disease about. our endpoints them. excited in to continue But chose in we're to the this it to hand of we're that progressive on

Jay Backstrom

Yes. start And were would -- what mean, Maybe see to I'll I just as we talked there. would we for. I the think we looking be before expect open-label extension, about as what we defined abstract presentation stable, come And the and what look listen the said by and the at you Habib ATS that's or progression see. if to you'll improve. and disease maintain frankly, in you

into ran Now the with and X about And randomized with a XX-week to and trial, respect we're you study, look when XXX-patient we the we're Phase we're approach question endpoint. your titration to both in a combined six-minute three-arm going doses at endpoint, study significance a confidence treating. a small doses very we on doing Both had combined -- a doing not distance. walk

expect before, over XX-week try get me get confidence what endpoint you've I and a a the program significant. efficient, seeing, heard study, program and of that a with and As clearly than six-minute with with efficient registration the XX the word quicker, to our very is walk the size endpoint think to way that weeks is, obviously, of consistency a to frankly, we're a I XX-week, say improvement

protocols, Europe. with I We've endpoint think both solid. the FDA socialized in and is the

regulatory this biased with authorities we've got on So the approach.

if recognize, three we're it's for also secondary at place. But multi-component and so the study very have we endpoints STELLAR, look think good a you STELLAR, And endpoint. programs, in in one included of a again, I

although which walk have then this It's look endpoint It's It's time of which when you an expect will endpoint. the that, a cohort added variable of such upfront HYPERION, time a included probability and we not an event-driven in if built the measured looking, trial we is distance. And endpoint, cohort, these talked functional on lot turn see are ZENITH patients sotatercept similarly, that cetera, we not will events take of and as We able think R&D some reduce a so the design at high-risk the It studies. at the et primary anticipate across so I'll the but thoughtful there that to morbidity in exactly to in and with events and about, mortality we those we'll is again, risk the then share a at at to week may like clinicaltrials.gov, program. demonstrate Day. study, a not and and of really be It's XX. intermediate at we'll we a more clinical the potential be six-minute haven't of that now posted worsening, here be that, having other for events. we number also morbidity to improvements, for would mortality

positive the STELLAR. very technical Phase you've Phase positive So done given and we've I have way that except of have that's II to strengthened II solid it from what a I very when success program, to the and together, potentially with to have done high feel data. we We strengthen is probability a and a little again, study all STELLAR, Phase III there, vary best do put what

Danielle Brill

much. Thank it. Got so you

Habib Dable

Thank Danielle you,

Operator

Your question of the comes next from with Eric JPMorgan. Joseph line

is open. line Your

Eric Joseph

Hi, Thanks taking the good for evening. question.

we expectations in on of Just which is a pandemic time STELLAR, experience from with particular one sort Is should the such not lines fair frame -- to of quick a PULSAR it recruitment us representing graph that larger, the thanks. is how really, if challenge. it terms from

Habib Dable

Again, over Jay? you, to

Jay Backstrom

Yeah.

the We're as end the be, trial hoped of the year. opened up would have us of we couple on I as a to things. So, what I'll say, track, at

working which clearly, far, running. so in there's interest the program, hard study is up the Eric, in and got the to investigators the We've centers to so clearly and So, good, good. get

where things so far, good. at place a I like So feel we're so

we it's the of think very and beginning again, the in with recruitment early Of But the study. fortunate I in early PULSAR. phase were course,

I get continue is we to before right? got and They're enrollment on to middle hard calls on, think able we'll going better, I've us as how got we said of our centers we get the as to things be get Habib then interested. open pandemic so But data back similar on good, getting to open. place. the that were presented. the we on influence so to hopefully, trial. and I we're ability like with business. that out to had so accrual, on we're centers routine We'll the want we pandemic, we're the The hence, to patients to patients really working completed commented the They previous just and but far, are And a the open-label keep into feel follow, won't even see ask track. and go, in if

Eric Joseph

the Do has you think pandemic effected prevalence?

Jay Backstrom

affected it the prevalence? Has

Eric Joseph

prevalence mortality have among increased might impact an infection and there to susceptibility just that I as the patients Has PAH guess, mortality really had – – on been

Jay Backstrom

manage it's across, vulnerable that's tell programs patients our group, to our from have our over haven't still patients and through experience without are both came generalizing seen it. the access and medicine Obviously, into COVID. we you so Yeah. a on – open-label, can with open-label, What I from pandemic, to who trial, able they're our own not We on and for to sure. fine XX%-plus, succumbing

trust but that But with minimize seeing question, would pulmonary vaccination they I strategies we're not good question, interesting that effect that. hopefully, in management, can So our and interesting studies.

Eric Joseph

question. the taking for Thanks Great.

Habib Dable

you, Eric. Thank

Operator

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

Your line open. is

Geoffrey Porges

Appreciate Thank Jay. taking question, the you very much.

So MDS I'm that the wanted you in the HYPERION newly That's lastly, frontline patients in you just what won't you Thanks. US first need Europe? And or MDS patient superiority? study any know PULSAR EPO that you very XXX And I regulatory in relatively all study, trial? pointed and I'll on MDS. keep approval the theme then, the on Jay, of experience happening about the STELLAR. because a Apologies on could the saw for diagnosed arm confident is effect out And clear to but follow-up absolutely study, arm? it in care? have the small active is banging question. recruitment control that questions. luspatercept you're the medical patients the going observations then Could STELLAR is the wondering of you about not from how on comment of experience – talk or then proportion the to patients going to are for show what seeking So are be in you what's to just the that to achieve transfusion-free I

Jay Backstrom

let's Well, I? six-minute shall with start walk, Okay.

Habib Dable

please. Yes,

Jay Backstrom

Yeah.

distance. the start with So six let's minute walk

a we regulatory distance, the program I took not benefit, of being we've and discussed As and aligned have an is measure mentioned, six-minute Geoff, full them breakthrough walk authorities advantage the functional we health granted acceptable with only is but which so in of endpoint. PRIME therapy designation with STELLAR, the entire that primary endpoint,

alignment. So that we've got

all included In endpoints addition, other in the secondary that. we discussed

think, that, So good. I is to the

now have, you SPECTRA. the to together have we patients, also that with crossover when you PULSAR look put the recognize think totality I information we of additional the at

clinical in when body bring you time, over addition then parameters other So within of the have we'll the evidence all on and to that. programs, STELLAR we're a course, adding of of

they've regulatory and like reopened. of partner completion period So pandemic a far. BMS it's some but straight the certainly adding endpoint, to COMMANDS, of close effect they're answer, centers. yes, With the programs, our I running get anything, so study working to those acceptable we socialized. the decision the program back time, took It since did on in They're additional a the for BMS March. have think

is of that expect. Recall in. narrow patient challenge last population are about require a that So principally the on and with It's then accrual. to get actually they're getting fairly has And patients lot still to so in been to trial. interest pushing patients clear your the patients respect transfusions. a Nonetheless, what that question there these

to begin are by and get But not at once And get circumstances so on honestly, start XX% levels in just above Fenaux, Platzbecker best XX% best the in EPO does an -- for out It the two where range. levels. it to not work XXX, start transfusions quite well. -- stratifying XXX, don't get of begin to transfusion you for in look We they once of to something and were ESAs they to particularly At over or in see, work independence last levels the ESA. well. settings EPO if doesn't years EPO from get the that you even the publications you patients

have you luspatercept, we take that that So that patients at those population. the the quite a the had have a independence, that durable. really which looking data achieving study, even the were at we MEDALIST really beginning. with trial patients transfusion And of if where then in at range you look We look if data XX% over two-unit the take is is in at

I those some without give you to should into of on So more success on probability the of detail Geoff, the bookends sense what that expect going kind stats, for think two study. of

Geoffrey Porges

you, Thank Great. Jay.

Jay Backstrom

Thank you, Geoff.

Operator

Your of Nochomovitz Citigroup. next from with line question comes Yigal the

line open. Your is

Yigal Nochomovitz

product program? will questions. SSc-ILD see meet very ACE-XXXX. one Thanks. the what describe much patients Phase the in you're that you Ib/II I in to Hi. this believe just on taking you had for Thanks the for target with profile Could for the objectives hoping data

Habib Dable

as Thanks going well. Jay to question, to that over Yigal. your for hand I'm

Jay Backstrom

Yes.

gone So the details. we really Ib of designed as a it haven't we lot and a into

with be kind to opportunity do the If that we I quick But And clin try to really I of for as where preceded into important it them to with we X.X as I that's be Phase feel predicted portion. is that will to to frame guidance was it, to in really -- it good that engagement. in for the anticipate Day what to of of get to go But into to and did impressed. PK/PD back PK/PD a mg that. a with extrapolate active to good understanding be clinical identify as to team some And to is were clinical give they Ib it to my R&D going mg I generate there's trying there their endpoints spot just It company. a this to what teaser go-forward biomarker so data rich pharm get they the what what's and that's setting need be number about would look as in target dose in have opening sotatercept a give us some we to They be us parameters, able define same ways pharmacology, place the X.X think doses and II. in we manner we So that come model further. going to me we do will into and possible think both saw to coming and dose. the they very and their on doses. that

and based in where the for how to say latitude step moving Ib, that and the And the any look as then go is Ib going to critical most some that to we're forward schedule So right. into established we've that run. we on to program getting always I have that once dose development then and

position nicely us And will for II. Phase that the so

we study us and So it that. will we're the study as share the critical. centers Ib the things can prior a we it and now getting in affords we're But cohort-type to be see starting right opened phase. that opportunity It's we absolutely

study no data We're yet. to share getting ready. So the

Yigal Nochomovitz

you Got much, it. Thank very Jay.

Jay Backstrom

Yigal. Thank you,

Operator

the Your Corinne next Sachs. Jenkins question Goldman comes of from line with

open. Your line is

Corinne Jenkins

that worsening, wondering into enrolling time of and endpoint the was the history clinical natural be regard HYPERION study? with going what's the to to you're I for population of to that Hi. patients

Habib Dable

been to which that. come at between you look the them possible, fact, an cohort. really a patients a looked out XX% -- if really And it XX% take population. getting depends at like control can risk under functional and in goal studies newly is achieve high It high-risk events upfront in Class there patients diagnosed year. We've well. for We selected The through recent if population. from such and this is risk question. higher on I this way the they're maybe have so that several intermediate is good defined some are at Yes, There's

clinical worsening If endpoint. of composite there at is a the you the time look data principally out, kind to

does mortality. that So all-cause include

bring it. typically that quite the get look for drive out event particularly events. have this really But where some the patients, There's recent with patients control. is lines criteria and because deaths not that to should that publications it's good showing therapies to we hungry on if such upfront to other reduction, be desire patients of ones hospitalization the patients to do enriched it. most of be under is PVR PULSAR just of treatment honestly, selected top add because would to data Fortunately these of that's field at for a controlled. can common you the there's advantage worsening that We've number of tend And the strong I believe early a that and out, fortunate. deliberately are the population goal that

we're it but So working expect trial to that's open, so what need rescue. to they get like hard the

Corinne Jenkins

clarification. Maybe That's point helpful. just Yes. one more of

now. Reblozyl, You with prevalent that a were therapy? new of is that Is they're coming on patients respect mentioned or patient earlier -- you in to about XX% population, were on that QX XX% total patients

Jay Backstrom

with the that's portion Reblozyl the States total to that's in because the a forget MDS Yes. we No, as population population on beta-thalassemia don't with have well. still of on United respect

Corinne Jenkins

Great.

Okay. Thank you.

Operator

next Your of with Gershell Leland comes line from Oppenheimer. the question

line Your is open.

Leland Gershell

potential New to population in We Good in you. for might we taking hemoglobin in question for the see Thank and about requirements thanks that ask for wanted monitoring for context afternoon. the color. patient the predictability license a in in of thanks any for safety profile, maybe go hemoglobin? for in questions to the of on appreciate that Want my of Jay, terms Any And the inconsistency we concerns possibility patients. the sotatercept the the Journal. on and PULSAR data England increases ask come patients publication I did Hi. PAH? should with PAH how think about who just the

Jay Backstrom

Yes.

let So start. just me

was I PULSAR think to quite instructive us.

was worked at it I XXXX on in sotatercept when MDS. actually I Celgene know, you was -- for As

effect that deciliter. at data per look that grams we the When an take hemoglobin a to of of on the expected range part it profile. is its as So X.X has we presented X.X in it's

with the the associated dramatic. So that is it. not actually change have ERAs in the of number a anemia fact And magnitude of therapies

What think benefit. that probably anything not high up upper and end The Touching frankly, with. going we concerned mg I know little monitoring those potential dose at to those at is a most the second some do it Actually, dose of will is require X.X already up third the then something potentially is with further higher by So be that looked our and steadily of see And isn't would something have after you patients. be that patients in will it certainly that about that potential any attenuate don't but with we are increases. be think normal running to this bit don't strategy mg to we continues getting usually to patients a where that rise effect the anything X.X hemoglobin. dose. that are or really

therapies. are just So, look of this take -- potentially being it's easy the to to is already other for because anemia some present -- at. Patients managed considered of

in is into of -- can we're it's be patients I neatly of woven routine study. So, kind going replicating our what kind for think which care

Leland Gershell

lot and indications pathway forth. this potential with obviously just question indication so and fibrotic you of mechanism then with And do further pipeline your and a Thanks. in in could a terms

the pulmonary the about Just be hear any other plans or may looking to that what further as side? And wondering to forward you have? allude we'll go in to development to do outside Thanks. R&D at maybe if you have maybe Habib you than R&D could may you wait until

Habib Dable

Leland. it's question a great Yes,

So, us would for all hypertension. to I to that with develop thing pulmonary commercialize everyone of is and I first I need say BMS remind the think agreement the sotatercept allows licensing areas guess, across you

where group hear of from two and functional of robustness have terms you're hypertension But within We're quite or going heart of hypertension classes development But with day basically in and we're pulmonary terms path confidence plan forward. that there preclinical clinical work terms of PAH there findings group be And not to to the our associated that talked we've share beyond? and So, in hypertension a at is disease. in our PAH. group These obviously, tremendous in exploring amount commitment. share pulmonary our of preclinically that to a heavily. different obviously going one -- opportunities including able left we make work within three bit to we're today about are R&D yet pulmonary that about are any coming

to you're about specifically sotatercept. two only And but to also lot going X group a and and so yes more Day try where hear please it make the R&D hypertension pulmonary not about group

Leland Gershell

team. tuned Stay Thanks that. for

Operator

MacKay from of comes Capital question Kennen Markets. next Your RBC line the with

open. is line Your

Michael Murray

Hi, for is Thanks this Kennen. question. for Murray Michael on taking my

MDS growth How fruit you I beta-thalassemia And of Thank markets? do the the breakdown luspatercept launch marketing near-term what do larger But you. perspective? the your from think is another current and out is opportunity? from commercial then one. ground On a sales and between are just where remaining hearing quick you the know where sales is boots And on hands. the low-hanging

Habib Dable

leadership field the by in to Michael commercial people that the of States, saying do let can now the the the And in under to color start with are opportunity not North predominantly commercial Officer But terms launch XX we he BMS It's Great. in you America, Kango, the by in US hands BMS' some off our asked. in Yes. over me co-promote. Well, worldwide. our United in Sujay and is out answering first of Chief you. questions have hands. you've some Commercial perhaps have give of funded that Sujay we of live the

Sujay Kango

Yes absolutely.

And regard the second question get your fruit. I with if low-hanging as was you're to the to to kind question, sense the of little split. a a So, understood trying bit

as US part combined we of significantly is tell patients, kind almost the transfusion-dependent is more of which about MDS imagine which the XX,XXX low-risk can somewhere second revenue the low-risk tune in is take patient MDS the much to speaking as population. And Europe can close assume population We to I RF-positive MDS between can sort well indications between can cohort largely today totality really I of two in patient beta-thalassemia maybe population of the have to low-risk US you to how don't patients. that is However, the and of XX,XXX as first. sales that you say patient the is the you is tune right? the of the breakdown beta-thalassemia Now, what coming

beta-thalassemia think the US X,XXX patients. about you is you the population transfusion-dependent patient in have If about particularly that

the now almost majority You is is come revenue States at the right stream of in that the going to revenue United from least of stream bulk assume MDS. which the can the

generic you to but a a gives of pretty expect. framework hopefully So, reasonable that how

in fruit are on segment BMS conversations. commercial growth yet Now, was the as kind Habib of do the still assume capitalized your that the I sort lot patients We the prior continuing a of patient where at question. and because the earlier all see in first of haven't the primary do low-hanging growth journey question quantified that's is peers see on we the we MDS have our in how

Phase our to we coming and earlier moving can we forward. we do lines believe get of larger study segment the So, similar half further of that should what still ESA-treated registrational year that drive and have sort of second patient population growth treated a of in the within III

sight gives well. as So, hopefully you of that a line

Michael Murray

Thank does. you. it Yes,

Habib Dable

Michael. you, Thank

Operator

end the like Acceleron's session. to Q&A CEO back Dable to reached of closing We turn Habib the remarks. have I call for the would

Habib Dable

free if I of as during Thank stay much the the evening. a operator. And June. us to or Great. you any to looking weeks wishing in have R&D everyone Thanks the meantime, you, you questions, in connecting feel healthy and Jamie. as and in reach Todd ATS many with you please well hope in for meantime, next out the to great few virtual continues everyone joining safe all today. Very And Day around to forward

Operator

you participating. conference today's Thank This call. concludes for

disconnect. now may You

Acceleron Pharma (XLRN) 9 May 212021 Q1 Earnings call transcript