XLRN - Earnings call transcript Q4 2019

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Operator

Good afternoon, ladies and gentlemen and welcome to the Acceleron Fourth Quarter and Full Year 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. President, and Vice Acceleron. Relations Senior James, Corporate Investor affairs at Todd go Please ahead.

Todd James

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

of McLaughlin, Jay the Dable, Kango, Commercial our Officer; Head Kevin Habib Backstrom, Sujay this Dr. Chief Officer; are our & afternoon call Financial our Joining Officer. on Chief Development; Research our Executive Chief

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

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

Habib Dable

and US adult for Acceleron partner, call, that cell who blood of Thank time for It a of transfusions. at Shortly with announced joining the beginning the require collaboration after been and beta-thalassemia, certainly Squibb FDA treatment Bristol-Myers patients of our our has Todd. in us busy REBLOZYL thank you the approval Good Great. Acceleron anemia quarter earnings today. since November. third call regular afternoon, at everyone you, red

is the agent as Acceleron luspatercept maturation to the also REBLOZYL approval. erythroid medicine FDA known first discovered in receive first

Sujay commercial and Bristol’s Kango our deployed leadership Commercial XX oncology Officer, of hematology person have the complement under a We commercial footprint. team to Chief

top focus thalassemia urgency top excellence the target with the ensure patient remain awareness of to centers and and brand a our which throughout sense access, continue healthcare providers priorities. US teams in of to Our

last the selling a short start. the tail is great with the year, off launch a end at approval cycle, beta-thalassemia coming of to Despite

for and new April the shipped Myelodysplastic the receive which are opportunity If Xth, grow the FDA’s larger reach decision repeat week-over-week second we’ll we a we number growth of market MDS to are action of approval in seeing a in or date the prescribing awaiting We indication vials considerably. target our accounts. to PDUFA Syndromes by

in with larger we fact, red are estimated risk population with cell transfusions. there the that who X,XXX estimate sideroblasts require cell XX,XXX beta-thalassemia, the have more red require than US, transfusions. In patients lower blood who than ring much A blood X,XXX the MDS and US in to patients

Annual in well anemia. as most MDS the Meeting the new opportunity of trials patients had in X MEDALIST from long-term to as from X pivotal the initial myelofibrosis-associated share the the Turning Phase Phase recent X and in updates. the ASH and beta-thalassemia, results trial BELIEVE in with December, At to we analyses clinical a total abstracts respectively,

pleased to publication the of years. most the occurrence achieved also out highlights a more treated were from with Medicine, two line in Journal patients we the fatigue, the marking long-term to trial from results the median results point common peer the of this England first with reviewed in I’m luspatercept this of MEDALIST clinical decreasing events published over of close Among trial, duration that adverse the were and prestigious that headache New time. of journal. our the than asthenia, showed top Notably, recently XX% benefit

receiving XX profound treated with of clinical receive treated demonstrated the of adverse or cell combination more for a clinical X Long-term experienced clinical in luspatercept. effects strong events trial occurrence trial over of months patients red patients most with patients In XX% time. duration the activity, with than whether almost benefit benefit patients median also the Phase myelofibrosis with most and BELIEVE not, blood results decreasing common bone pain, ruxolitinib. in from arthralgia the and experienced with treatment transfusions Overall, of were they luspatercept seen dizziness the

evaluating results anemia X patients are positive the red in INDEPENDENCE Phase these treatment for a a JAK additional initiation this to cell are myelofibrosis, inhibitor transfusions. about Following to with and who luspatercept and design we new the with We now year. myelofibrosis preparing require of the details on blood study called closer trial you for providing forward look the of later trials pivotal

three further the late-stage erythroid for REBLOZYL’s toward by anemia REBLOZYL, a novel from of future validates mechanism for now as distinct look thousands consistent across of maturation a hematologic we disorders results the positive suffering platform patients As caused variety treatment seen of blood possible diseases.

program. Turning now to pulmonary our

in on PULSAR during Phase As results, PAH. line or I’d the Pulmonary were in sotatercept last to from to month Arterial we X, top X quickly reviewed an thrilled Hypertension results PAH. shown renowned those with positive also world experts which Slide of with like investor patients two recap call we trial announce in-depth

important blind, As of over of trials’ is powered study in X.X endpoint. on milligrams kilogram period. The measure or was double a randomized sotatercept subcutaneously stable to therapies PULSAR or PAH XX-week study X.X placebo, and sotatercept Resistance in XX% trial show the patients PVR, Vascular adjusted a days every Pulmonary per hemodynamic placebo-controlled you milligrams treatment reduction XXX primary the per an kilogram placebo XX recall, a receive background were may specific which to

We to secondary show The significant improvements the key was both of and a primary secondary distance. key walk powered in endpoint placebo also adjusted XX-meter improvement achieved the statistically endpoints. X-minute trial in

compared a of improvement to also change walk We clinically achieved least a absolute at XX-meter meaningful in baseline. X-minute distance

from well, as of functional class. endpoints health WHO an biomarker in secondary change baseline in other achieved including and cardiovascular NT-proBNP, important change trial The

generally previously of consistent with published sotatercept adverse safety data other In well terms its in diseases. events with tolerated was profile,

of efficacy further in XX the trial, long-term over the into enrolled which will period safety. Importantly, rolled provide the patients PULSAR into XX-month insight and extension trial

shift clinical years on to outcome. potential treatment of in be extremely of proud currently to significant efforts I’m sotatercept’s PULSAR not of all few our last the therapies. of PAH could the paradigm achieve believe team over available to We this the with to top continue of the benefit and we the in happier outcome trial, provide

We hope you of at Philadelphia, International as line trial of presentation American known XXXX PULSAR the May detailed Society XXth Pennsylvania. review for Conference the the also the on join can the of in APS results a from Thoracic top us XXth to more

forward future also look provide and regulatory to feedback for to Global with sotatercept development We for upcoming Health important Authorities with interactions PAH. the us plan

positive the the pipeline, overall disease. cement commitment the further at to from PULSAR trial our results pulmonary Looking

our modulate patients now volunteer research we We enroll molecules Therapeutics help to to at open with close PAH, continue Phase in with trial of and with associated XXXX, pulmonary us a healthy label target discovery study pathways collaboration the in that ACE-XXXX sotatercept is space. the specific and announced a X the indication small can underway structured Fulcrum identify of

With that, hematology our turn I’d program. like to to

partner, early regulatory clinical and completed lower Phase by treatment this our highlighted non-transfusion I of with in ‘XX. dependent We patients results X the Squibb of collaboration Bristol-Myers the trial trial and in to that in XXXX enrollment naive expect the top BEYOND line beta-thalassemia or with earlier, line activity enroll addition, In recently anticipate The late top X this trial luspatercept anemia end year. continues results associated from COMMANDS with MDS. Phase patients risk pivotal to

randomized Disease Moving the recap, ACE-XXX receive of from the Phase CMT Charcot-Marie-Tooth the program, to part moderate X in dorsiflexion two weakness CMT with ankle were top either neuromuscular with trial mild of next XX expect part with study this or to results our placebo. the month. we patients of approximately in ACE-XXX share or line to to To patients

muscle all month over designed and and randomized trial X-minute treatment six and The walk fraction, to time reported tolerability and is XX-meter period. assess in contract distance, patient volume disease burden change walk run, a fat total safety

see, been us a period productive can has you As it and busy Acceleron. at for

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

Kevin McLaughlin

refer our earlier the Habib. year afternoon, investments. of fourth for opportunity press everyone. $XXX.X you equivalents issued to Thanks, million I’d items. and cash and XXXX a to quarter quarter results release fourth our ended take year full summary and full the a approximately cash, to ended this review like few for and briefly We financial in with today Good

and acceptance approval for XXXX. $XXX.X the Habib? MAA the Collaboration million. to in totaling with support incurred gross the year was XXst, of milestone ended the related of is the of expenses the company company’s Total earned in Bristol-Myers for Squibb validation as REBLOZYL, as and year payments loss revenue November of expenses was REBLOZYL BLA $XX primarily company’s revenue the from upon derived net EMEA the million. million. XXXX was The partnership were by The all one-time $XX for and million and costs of the June December in well REBLOZYL $XXX.X year FDA FDA

Habib Dable

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

Operator

from Our Instructions] [Operator Werber Please ahead. Yaron from comes sir. Cowen. you, go Thank question first

line. If your line your is muted, please unmute

Yaron Werber

Thanks the question. quarter. Werber. our congrats Hey, Yaron for on [indiscernible] on for This taking is

So in the also us in the can you. you questions with have terms your dynamics adoption of Phase field, us that? the And X Thank share in REBLOZYL, share please? a can I please myelofibrosis, regarding progress just more few some on early on with you early

Habib Dable

question. is for the heard Yeah. thanks I to just correctly. your This sure question I that Habib, want Hi. make

of we And the was, I was, path more Is the the add Phase think indication. the share can question forward with first color launch REBLOZYL details question myelofibrosis? in in first on in any we can terms that any the X correct? the second

Yaron Werber

Yes. Thank you.

Habib Dable

Okay.

great. Okay,

Bristol-Myers, that Phase which we the together Phase collaborators as looking details with at be you announce the ruxolitinib study the shared combined the basically And we’ll we which in X So in moving luspatercept X was at announced of myelofibrosis at we’ve thrilled forward study, transfusion-dependent. were to with were with with patients obviously INDEPENDENCE our myelofibrosis. ASH, data

And to really so else study there’s to and point, that on XXXX. we continue track, go initiate that we in really Things add. at would nothing this had said

So we’re is of restore of where there validating the in to to ability a in rare disorders lot really forward hallmark moving these again, luspatercept the healthy for really another blood anemia blood space, about red formation cell able be disorders. excited

X,XXX what second initial roughly or transfusion-dependent with a the are of that patients small adult With about indication of respect to you continue in we estimated patients. who terms launch REBLOZYL, States, X,XXX be indication is very of question tell beta-thalassemia United very your the to pleased first can is the in Obviously, I we very, the launch.

a indication, one. So it’s unmet happy these an obviously with cater opportunity important still to patients to And to have need need. the an to be we’re able small unmet very significant is a

– think States. we in of actual about don’t sales, calling a the you’ll that any specifics when indication out it’s But United the we because the we – have notice didn’t very have itself, again, in launch terms small

that into it context the if got the November, with you end we in at about tail line you approval very – Secondly, with the therefore, holidays, couple not approval X or point. the major and X Congress and informative couple year think was put the revenues around this the even a and cycle low, of with material were at or a and of the that’s selling weeks

date date terms think how say, in the all which all But indication patients continuing action big pleased all of very we’re has with be lower the orders, anticipating So very the when which grow, moving a happy very how would we’re that MDS in and for who I the thus metrics, we’re regularly is about the hurdles, second PDUFA year, And the obviously, of very, of Xth, all, think are were risk said, we reimbursement when for would about week-over-week new hoping pleased transfused. sales, we April that how today. repeat the about into when of for indication the things on even to are continued launch progress. we we’re access think to with

Yaron Werber

plan? Thank it’s X sort I a the oral of sort Phase data PDXs move of afterwards, around about of sort X a about they’re an should is including switch rather have strategy in studies, the up? X development we quick you. just of think or study to doing with what is a And would you follow-on, and just think or ATS terms [indiscernible]

Habib Dable

of with all, respect you dentist talking presentation, ATS the Yeah, about I and I’m inhibitors. PDX – so said the in you’re Yaron, - studies, assuming to the first with and

this point to So fact we’re will can that we that at that we the confirm data and data plan be ATS. any color at the not than other present adding

any until Beyond sharing opportunity with got that, be details the regulators. will we meet after to not the we

Yaron Werber

Great, thank you.

Operator

Thank you.

Brill from Danielle Our comes next ahead. from go question Sandler. Please Piper

Danielle Brill

congrats Thanks guys. again to Hi, can and I questions have a prior for question there. guess on the all the start couple I if progress. I the follow-ups

expect you are XQ? we on any sales comment commentary out that with over things then launch? cadence for in the And you how launch REBLOZYL path can also on the First, given how of expecting And Habib might sotatercept? the tracking with will Do then be for the to year? just forward when about you be regulators the remainder interact think should called on

Habib Dable

Yeah.

thanks for Danielle. questions, your So

So maybe first. I’ll one answer the last

we previously of obviously roughly what that that as soon feedback and regulators year know, we’d appropriate share said we this the plan to we’ve that, interacting and then So with as be you mid before the time. year, said at the possible with

be With launch to we’re the growth of repeat, with what we week of access, with pleased that respect in progress week-over-week, to qualitatively sharing reimbursement we respect seeing and plan the we’re to what REBLOZYL. continue just be repeat Again, every terms to to making. customers

indication can Xth. MDS is remember PDUFA you what expect, the for until date Now, again, in the not terms April the of

probably quarter beta-thalassemia just you sales of the indication of can relatively, very simply because X,XXX or about X,XXX non-material a we you another is the low United we to So fact patients. that again, know, estimate you know, small low volume expect States or in where

believe Obviously, having we sales be much some I likely approved, MDS, QX, QX and in would be more is seen informative. at once you significant when MDS beyond, if more and with know, looking which would

the in terms could So potentially what growth I of cadence you of expect. that terms in at would look the than

Danielle Brill

evaluate one you Thanks. there may. that to might of Got it, for more, fibrotic any interest sotatercept us? for if And that’s that other it then plan be helpful. I diseases may just Are

Habib Dable

early about talk Yeah, that. so to too

that We’re with dilatory X and there. XX need to preliminary this significant to to the be obviously and have the out obviously study drug a potentially PAH first introduced we – wonderful in population very the drugs cater thrilled data outside Phase our to in opportunity the dilatory a of patient unmet are with

imagine, a can there’s work a of you still do lot and to opportunity sotatercept as there. So move tremendous to forward

look beyond the continue will with come the indications for of data, committee the will interrogate the time. will benefit we’re for, we that we Obviously, steering opportunities to looking but our Council and as from identify other opportunities regulators preliminary to that

assets we Now, be other JP in yet pulmonary more we at which ACE-XXXX, at other talked lung we areas disease, healthy what targeting volunteer tell is you know, with can believe as such at looking study, obviously areas be think which could about scleroderma, determine know And looking potentially did idiopathic earlier at Morgan, though, cetera, are such as, fibrotic-driven I now we a least fibrosis, diseases as mentioned well. we could we we you that indication. I have I et within to pulmonary interstitial disease, systemic where but

Todd James

Todd. it’s And hey, Danielle,

allows As Celgene/Bristol goes, far agreement sotatercept in indications. for as PAH our with the development amended

sotatercept case sense groups but current limited goes, to of which able would is ability, based one, we and so being as science And two of PAH, biology potential that develop PAH be far terms outside other the off of business it agreement. have if amended makes group the the PAH the to as for a from or group we’d

Danielle Brill

very the for it, and congrats Thanks Got helpful. questions again again.

Habib Dable

Danielle. Thanks, Yeah.

Operator

you. Thank

from from next SVB go comes Our question Geoffrey Leerink. Porges Please ahead.

Geoffrey Porges

at in on side, bracketing a going since of but particularly, to it would but you’re be congratulations, very helpful accustomed about trajectory and is haven’t guidance, it’s going are some us your number on suppose I financial to you know much expenses the for conversely, least to seem the Thank SG&A. any not the progress. get be, necessarily given I to A all R&D expense financial indication question, and Habib are you XXXX. studies which give team your investing one what of ending you

least expect might at whether overall you you even sense a right step level could or us some operating expenses give significant flux? this a there year in the about of whether up is So be though this

you back then a agency to a agency plan. we going development assume to the things then plan are going sotatercept, to know should and about with And to I with be the you’ve to just or but coming reluctant with interaction, back results that go been you regulatory your discuss will the

setting know we steps a So the the I of guess hear with could and on perhaps how, whether the that’s next whether in summertime, call you timing? right QX sometime the sotatercept expectation

Habib Dable

your for questions, Geoff. thanks Okay,

regarding guidance Jay CFO ask the ask Head our for So with our McLaughlin, Kevin of our questions bracketing, R&D, and will address to I’m and for first regarding that. to the I to financial the then And part, second regulators, Backstrom new discussions going answer that one. the sotatercept plan

So, please? to take part, the Kevin, do first you want

Kevin McLaughlin

doing? how’re Sure, you thanks Geoff, Habib. Listen. Hi,

you give guidance You’re know, historically expense longer we or as given correct, that the and have we guidance. not cash no

safe a additional the all our us. expenses obviously is this. trials The step though, think preparing sotatercept be you with for continues as up year know, on, have say, I will will to – are by what’s and we we in those funded

no funded So they’ll there’s all sharing by cost us. there be so

Now, of type step the not or determined we the communicated obviously, that run. of And have that will by trials be up has yet been to determined. size

So, we the company. of a us commercial bit in footprint fully know, support, to I you you to order moving are think the a growing that, company know,

want be to And think you will so have well but are things, effective We to I the in well control on keep enough spend the know manner think in which we that do things you there us that know, we board. I we necessary. as know we make very and in growth, cash sure we

Habib Dable

the question, answer could Jay? please Thank you. second Jay, you

Jay Backstrom

Hi. Yes, certainly.

is Jay. this So,

are know, interrogating So data. Geoff in answering the you question, actively both, we

we’re imagine, as and get process like we authorities review developing in some FDA with meet from we’d input that also plan, them well. to data other can to you is but the So, health when intention of as socialize and our them, with the

our authorities, by right engaging Habib move been refine on meet a forward active with midyear. as be committee like we’d the we’ve meetings to steering of to be said just health that But you plan, to know, until that. very able it’s that of those we actually as further and kind earlier, aggressive now, kind with to able So

So pretty that’s a reasonable point. time

Geoffrey Porges

Habib, which and you of of they’re about, Advisors to could Could far? responding some you’ve share Expert follow-up they’re product? in able what, with been PAH the thinking the Right. just is, feedback know, you question, your sense can a I so from us with how you them give other how share about

Habib Dable

I sure the opportunity. I’m Geoff, we you, I actually, the that had McLaughlin] Yeah, Yeah, not you on of time if two date. no, we’re are call kind I that is thought us opinion getting action can is the very board, [Val what joined leaders, respect of who leaders after call with [indiscernible], and you respond and to first And the feedback the the tell think the did, had with leaders a in very, for tell [ph] what PAH. about across the mechanism excited you right the can that is new having

innovation. that, fact to important disease is in survival the seven over the really, That’s you last approvals to significant got area a have that, this in XXs because think, rates that you know, three through, The improvement XX comprehend progress I years. six, know, in XX you years know, to years now tremendous you’ve from, that, early you know, improved of five maybe

age XX XX, the seven are when being years old. patients when five affected you But median think years know, there’s about the that survival are, by of to still you this horrible rates,

We need. will opportunity patients, lives, but impact to to cater able modifying, a potentially to hope patients’ to able that, and have sotatercept to able significant and these be indeed, very, have to if the And we to be do be to on remodeling very that disease. not unmet are we the significant I only here an disease introduce potentially

tell And excited I very community you that about is can that. the

of feedback a And I’m is a the one also of still that driving even optimism. there the be but more to concordance It’s to think cross feedback lot data. But is the That one optimistic, I today. excited, that that’s receive, endpoints on still positive driving of of the endpoint everyone say getting they’re that reaction fact same to is that the have consistency end seems the to would time, Geoff, do. that I work to the significant potential, said, the pleased data specific but the that’s a with we not the lot and across at we – at there just multiple finish do work according based really day, line. of

Geoffrey Porges

Great. Thanks very much for that color.

Habib Dable

Yeah, thanks.

Operator

you. Thank

from comes Carter go Please Gould Our next Barclays. from question ahead.

Carter Gould

for material have X you color congrats progress. guys. regulators on sotatercept yet? the to Bristol the in – appreciate the up guess of taking terms and two to Habib, I terms leave questions Xs you on the side, And any guess it with on – color believe there specifically side, meeting provided call and end manufacturing be Hey, on then with all a of there. in potential the requested to spot, position just I I going timing, had great on on yeah, Thanks Phase the with that there the manufacturing all maybe with scaling all put with of interactions on that the you FDA own Phase but your you are

Habib Dable

interactions typically with and we’ve are with information forward, not with a Okay, haven’t respect Carter, Once from request you. practice we the respect the the regulators cetera, any that feedback third-party the of to we – do. materials, material. shared we CMO to With that’s will on leveraging to got to definitely so regulators any a path and the the FDA, respect with we sotatercept share appropriate that et

Operator

Thank you.

JP question Eric go Morgan. Joseph from next Our Please ahead. comes from

Eric Joseph

unique there far just to whether sort wondering thinking the seeing for of REBLOZYL with is a – you’re that taking question fourth that items any ordering there’s in sort so any R&D Thanks run in think rate or quarter cadence reimbursement from where guys. patterns appropriate the know coming there still any or about XXXX? but – level the questions. whether ahead of prescription any whether second pushback and pull I’m any MDS, running we’re an I And the hurdles Hey, are through spend of in I of just spend OpEx, formal back in of Thanks. the the patients on into? that MDS

Kevin McLaughlin

Eric, Hey, $XX Fulcrum deal that there Kevin. quarter, went related this R&D million we was that the fourth Yeah. charge did. the is the through In to

a So that, and it’s pop it observation that. you’re because you know, good – of did up

Habib Dable

And regarding the ask our wants chime sales , Officer, Chief you in in if incremental really I’ll there anything Commercial to-date, say can terms Sujay of he if that to is color, Sujay.

Sujay Kango

Yeah, that sure. question. Eric for thanks

we MDS for into So that pointed and data dig as kind you that till out, very are approval MDS. to respect point, to know, awaiting you still the time us sets with difficult any of

access, coverage plan. I very we’re good tell they’re to going for any kind is know, most to know, insurance cetera, of that’s as to-date, regards companies seeing REBLOZYL However, according of have, you you of pushback, the et can with label. to and you not And covering

regards coming of in is beta-thalassemia we’re as where pockets it, know, seeing pleased ramp of and is spread uptake per and the utilization, where that’s to country. as for seeing now as the the se, well. how for across know, whereby So the, just broadly It’s seeing it we accounts from you’re up are we’re with the not influenza beta-thalassemia, you pockets pretty you across utilization the of

Okay.

helps. that hope So

Operator

Thank you.

Our next Stanley. Jeffrey ahead. comes Morgan from question Please go Hung from

Jeffrey Hung

are What the off Thanks of different Eric’s Maybe label for a you questions. anecdotally can about if hearing taking REBLOZYL? I use way. ask question

Sujay Kango

that, I maybe right. restate can So,

perspective, not tell the any is see off so is is date we our are there what we’re come label is and see data you we’re we’re medical coming analyzing Xth. approval saying least April set, information, queries PDUFA that a can you are around corner it. in there from at through probably people know So to interest of waiting the But about just what the

with people getting the the about are looking data and just set we’re probably the inquiries publication, medical into information. So

where kind right are we now. that’s of So

Jeffrey Hung

whether reorder Okay, limited I thanks. reorder? And patients proportion repeat and REBLOZYL reasons you cited patient doesn’t but about of a guess what with so don’t numbers, a orders if typical then talked understanding

Sujay Kango

come Early repeat days, through. as I now, the seen not of haven’t orders

So different going patterns, back orders, of know, accounts but between matchup one the for to we the that drug. has because very repeat gone far, point is you positive so we some are it not ordered we be account in and ordering you pretty getting consistency time it’s the one much know, reordered every different whereby, at has right, to

happy new very with prescriptions are repeat seeing between prescriptions. are the how lines we we So trend and

Jeffrey Hung

Okay. Great, thanks.

Sujay Kango

Jeff. Thanks,

Operator

Thank you.

Our next question comes Please from from ahead. Yigal Citi. go Nochomovitz

Unidentified Participant

seeing Hi, [indiscernible] our our Yigal. you an what Thanks is of authorization. and in for for on prior But of approved? if and couple percentage with taking noted payers be of a positive for amount And problems physician eligible curious, you And seeing? and some seem really spoken they REBLOZYL this know, prior good with discussion to for interest? off, sort into they’ve could expectations is seem view. you’re on I’m prescribing KOLs they’ve beta-thal majority run patients comment indication on their what to We’ve indicate that question. this of volume your their the you’re a get prior of auth

Sujay Kango

one right. question good a important very know and You

to a to and buy you’re well, product timeframe Part remind the of ask this triggers bill the a just are almost get insurance launched that it under till is in all that. very companies and now right. always we’ve audience going period is you medical benefits Part authorization, for common time listening when B are you have product going know a you right miscellaneous very REBLOZYL of which J-Code. permanent to And doing this where just J-Code because as code almost need as side J-Code, you’re we And during side, all So under a have prior this we of a the the B

And that the process. of out so it’s just part happening. It’s the that’s of not ordinary

services extensive support well coding our the billing as help patient as and are that members they that field through we processes. have BMS appropriately followed So from to we that required anticipated to team reimbursement accounts colleagues, ensure able these

get to just product sometimes it So it any the it’s in for such but be could denied it ways is a being the beta-thalassemia. not – takes timeframe that as reimbursed,

We you say of the I it’s not it So that’s till to trend. get want a to out that regards just permanent ordinary and in the positive anticipated that J-Code.

Unidentified Participant

Thank you. a timeframe then is That’s J-Code that’s And helpful. permanent? getting to normal the what

Sujay Kango

file when it can and codes, for BMS approved actually varies, required to got the you J-Codes. from get all et already, I the for you that the the already you tell product coding cetera. It filed and has we’ve permanent depends

this months be year, should we that’s hope. between upon anywhere later sometime to actually is XX hopefully XX the getting months you get So when that approved. product takes usually depending our It

So should it we year, get later timeframe, well sometime cycle that this early in so time. next year or anticipate it’s to so

Unidentified Participant

MDS separate J-Codes other they and or the be And for beta-thal would same?

Sujay Kango

the J-Code codes though. be would different code diagnosis same. are the product The The

Unidentified Participant

so Thank the taking Okay. much you question. for

Operator

Thank you.

Capital Please Our comes RBC Kennen next from MacKay question ahead. from Markets. go

Vikram Kaushik

Hi question. this our in taking Vikram for March. Kennen. Maybe readout on for CMT This quick is – hi, is on one thanks

thinking Going into how talked the the be shall endpoints internal the around them? And you readout, so we some what about functional the are of maybe about? expectations

Habib Dable

this Vikram Habib. for question. hi is Thanks Yeah, your

hurdle And total line able everyone, remind into a but ourselves we’ve just volume importantly, end muscle our results in be double-digit that given see to is have in part again first what to before do to to this that we typically in rate benefit. increases see more functional quarter. reveal seen part everyone the translate And two, volume. just muscle to total plan is to remind we again, hope what just one, to the So again, the of that top

a of X-minute a when we able that, move able at corrected way think the forward distance, a about to be what think us be in functional for hope is give placebo you benefits, you as such to to pathway walk when of to least confidence XX% benefit to see for approval. development And the example, in

see is want If we that confidence. as much trends very little what into and multiple we the move to Phase and in with the across some X us X Phase seeing and how we’re that we can in remember, Phase And X. ambiguity will confirming to X give as continue learn endpoints, Phase see typically

move to we so forward next to only the if would we confidence so And that do step. had

Vikram Kaushik

Thank it. you. Got

Habib Dable

Yeah, your thanks for question.

Operator

Thank you.

go from Oppenheimer. from next Our question Leland ahead. Please comes Gershell

Leland Gershell

quarterly with my that Habib perhaps used Is frequently was couple, at the under your least of couple not been certain distinction questions. acceleration impression to case? team. to wasn’t has is for a products a it Or I in aware as taking respect cycles, for more and annually. is Have follow-ups? to a what the just I’ve Hey, J-Code. frequently, had And Thanks in be I the applied of? versus that one, that

Sujay Kango

said, They we – could accelerating of as normally as a that’s anticipate valid should take we year. it as coding some codes these have so It’s but long and are that XX, later I question correct. some the this

Leland Gershell

be informative with from the And should could images of maybe on was remodeling define description And you filling agent? of sort on any to sotatercept. out see provide the SPECTRA? obviously, us the of that terms then you if want reverse wondering would SPECTRA I some there disease trial thresholds Okay. very quantifiable color for regard PAH, what more around in as you’d to activity more modifying

Sujay Kango

start So to Jay I’ll of some then blanks. the it I fill Backstrom over maybe can to in hand and

So I modifying need on what they see to go was when, a this what disease just the want Dr. said if to would and indeed, kind drug. call, back repeat of [indiscernible]

first definitive other we et of disease That the all, to Now, get patient without for remodeling really that know, said, metrics to biopsy. confidence which one give that biopsying of could anything vascular remodeling. obviously a lung it’s look to remodeling, cetera, do aren’t there you are at or hard

can MRIs, can look heart. the that gain And we with in there’ll what’s imagine, cardiac when of lot of information terms you as be we a at happening the

most remember of element the pay functional will [indiscernible] is said, have, which modification if you very important also class well. improvement, We to disease attention what close as

an But improvement patients max end PULSAR and vasodilation the and above to at in over the the half therapy coming kind of the showing the regarding from. question these triplet of we study the is enthusiasm of on Remember, back are patients. going where day, Jeffrey’s in were

it’s for improvement that benefit go an they’re vasodilation, extract to right are be effects interrogate to well. the heart as whether on And above the opportunity to continue max we the looking so will patient to ultimately, SPECTRA be as to able functional going their end and that’s data that function class

at looking all be to going they’re of think Leland. I So those,

Leland Gershell

then. Thanks Okay.

Jay Backstrom

yeah, This is - just to agree.

Now, that I was well think summarized.

Leland Gershell

question you they And lot frankly a is and you them not going I luspatercept wondering, hematologic intermediate if Is with may, issues there to those AML perhaps lower high that agent MDS? know, on MDS, appreciated. one studied on be, is could having much more just on? that it then a A know, going reason now, risk without in right been other know, or study to All other issues rationale risk have of that advanced to, of scientific or that category clinical you patients. patient in both

of why Just high be risk, wondering not Thanks. scope a interest? why

Jay Backstrom

that, lower is with life risk, expectancy take risk risk I’ll Jay. the are group. high mean, and than this progression I the maybe really so for Yeah, different

your still to but survival to more try progression course are of aggressive AML. component to risk usually the a control require really prolong try So treatment a be bit that. point, to primary the to can it and to therapy And disease, little reduce anemia the

Combinatorial strategies considered. be can certainly and some

think where in available to development great a where place for I it we plan target the It’s there MDS given with absence low went luspatercept area we really good very was to was space. other place of in felt just therapies. a very, the that risk be for really a the

Leland Gershell

for much makes sense. right, All very my questions. taking Thanks

Kevin McLaughlin

Leland. Thanks,

Operator

Paul comes question our Choi And Sachs. you. Please from ahead. go next Goldman from Thank

Liz Ewing

progress thanks Paul. for this taking our for on is the and Hi, Congrats all on Liz question. recent

ruxolitinib saw and recently the ASH for non- myelofibrosis, the presented where we So rate lower response at Phase you and had data group. X luspatercept the

or that we’re have so visibility any much. see and if guys the dealt So just data opportunity wondering analyses its or impacting on done impact in have how that additional in the more Thanks any on you responses, general? X you Phase

Habib Dable

question. thanks your Great, for

I address think question? please myelofibrosis’s can this you Jay,

Jay Backstrom

Yeah.

at way that be take why. the study, the how patients little continued interpret were least little data. bit to at numbers and the amplify know, rux because JP between has you a small Morgan, as without underscore we at we – assess bit There So to the with careful that about a to look differences a would talked we additional effort been ruxolitinib the tend really to be

what the we X With is the present shared with respect liked had of ruxolitinib even is a combinations, know, continue Phase and we that a to you that earlier, think problem is very patients though, on patients anemia’s again, anemia with I rux. commonly XX% there used,

we that combination you as referenced, looks know, at to will, cohort, you X. that good. those able you advance also And looked for when just as so be compelling, data quite to a looked quite And phase really if to us

Liz Ewing

much. so thanks Great,

Habib Dable

for thanks your question. Great,

Operator

remarks. no CEO Thank further over closing questions. call I’d like you. show this turn to Dable, go to ahead. time, At for the Habib I Please

Habib Dable

conferences. Yeah, Thanks and up a Obviously, pivotal you we’ve to looking the thank the everybody for lot the a joining again, upcoming in is that at achieved call. you. very tail looking to shaping be XXXX continuing progress year to for with forward of some much really Acceleron, meeting forward end

Operator

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

disconnect. now may You

Acceleron Pharma (XLRN) 27 Feb 202019 Q4 Earnings call transcript