XLRN - Earnings call transcript Q3 2017

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Operator

Good day, ladies and gentlemen, and welcome to the Acceleron Third Quarter 2017 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 is being recorded. I’d now like to turn the call over to Todd James, Vice President, Investor Relations and Corporate Communication at Acceleron. ahead. go Please

Todd James

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

Chris Joining Corporate John Medical our our McLaughlin, Officer; Rovaldi, Executive me our today Development; Quisel, Matthew and Officer; Chief Dable, the Habib call Officer; our Kevin of Head Sherman, and for Financial Operations Management. of are Chief our Head Chief Program

of goal quarter, outline is Our After to afternoon to this key questions. upcoming updated provide that, and an results your overview look forward answering the financial we priorities. review our

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

Habib Dable

therapeutics areas, took strategy build Todd leadership focus about the talked and us led potential transform disease in and lives. good you our high review our pulmonary there disease. in At of progress an the lot best-in-class us in-depth core We with you, to many our first quarter and September need, place. hematology, And strategy multiple medical and we completion of which our within to and significant develop our area areas we vision for neuromuscular science treat patients’ to discussed everyone. therapeutic the disease a to unmet were today. corporate thank Thank and morning joining made past R&D of that of events This the long-term Day, key advancement

new package and focus our we our preclinical highlighted arterial data to with develop sotatercept in hypertension. commercialize robust pulmonary that announced rights Specifically and in we pulmonary, gained

We execute trials programs goal by upon move efficiently three also programs X of the Phase years the work corporate to we end to or made a clinic have in commitment within as three to our through XXXX.

of stock our a the our strategy were in million heels out the of long-term complete am option of for offering vision, proceeds, including able common in raised we important we On exercise team’s These on laying and which of successfully events public in follow future full net were to to very over-allotment the proud execute. ability I underwriters’ and company of all the October. our $XXX.X

to X in XXXX. as analysis the commercialization results driving the in on published mid-XXXX. individual And highlighted were the R&D you and will these Day, key in half The Results now luspatercept. prepare Phase sideroblast In MDS achievements to commence in patients initiatives are we well focused potential regardless providing trial expected from initiation additional Phase advance enrolled continue include line to Celgene MDS Oncology. of planned is program team fully hematology, and as in Myelodysplastic and for Lancet indications details status. as line we first in Beta-Thalassemia top lead in trial advance XXXX. and it now ring look At before supporting solving Syndromes our to Turning the forward quarter. X first with BELIEVE for trials We or to lower-risk our our to program trial with as as MDS MEDALIST X well continue Phase and we program of

is neuromuscular in in us in further pulmonary one beyond well in those year. our we Phase as believe are Phase the drive our X non-transfusion-dependent to trial Luspatercept expect also first allow the innovation will beta-thalassemia priority patients the success and later program’s as We X initiate franchises. to number and trial patients this myelofibrosis

to We strength muscle mass muscle build acting continue suffering and diseases in selectively to our patients local from neuromuscular designed muscle ACE-XXX, with our with maximize mass. franchise locally agent

treat We continue and X in to our FSHD enroll ongoing and CMT. patients in trials Phase two-part

of levels a Part is the Part identify muscle and forward As to reminder, key to into safety and objective evaluate X. X take in volume those changes to

muscle Part be strength evaluate look in January sotatercept. to or PAH focus safety recently focused the in cohort disease, our pulmonary changes on dose and in we XXXX. volume but X, to objectives will not be forward we providing and in preliminary key With For the only muscle announced arterial with pulmonary FSHD function one nationally results also will hypertension

remains you serious PAH, potential significantly standards-of-care now McLaughlin, be sotatercept will that, the to This through is illness agreement of and are milestone sotatercept quarter. seven financials modifying believe responsible Acceleron option the eligible of And to there for at know patients. be in and therapies. thrilled drugs robust even median not and a We life-threatening meaningful are People be available fund, modifying develop to is PAH range. are in receive rights to for we that years, approach to will to based were survival many which any sales gained with payments amended a net the first-in-class disease call disease the treatment years. Celgene. no rare I to Upon has PAH. PAH, Officer this the the our on a patients. to outcome payments the last in for agreement over PAH. XX% which therapeutic with achieve low for now Financial sotatercept generally in XX believe over royalties for and able multiple have five We PAH treatments to protein With disease to As has upfront young approved outperformed a diagnosed to turn TGF-beta per threatening the sotatercept a could the run Chief a life and under that Celgene preclinical impact superfamily package from we Kevin sotatercept in and were No with required global sotatercept commercialize Celgene, exceptional

Kevin McLaughlin

Thanks Habib and morning everyone. good

includes This quarter expenses comments. of presentation million $X XXXX expenses related September quarter revenue turn The of existing that Collaboration the and into net $XX.X million. revenue Celgene primarily and of cash, net were expenses the will million in fund resulted We XX, million. proceeds back from including the be $XX.X of million. net the company due $XX.X for the our of proceeds exercised investments, support offering final compared option an is was to the October third cash now and R&D equivalents additional equivalents September expenses for will our The partnership G&A million. the I was the XXXX. $XX.X the in XXXX. company for offering entirely of Habib operating as of from $X.X revenue entire third XX, to net requirements by million. sufficient $XXX.X in Our This a and believe projected million XX, partnered [second] our incurred cash, includes The of quarter $XXX.X cost-sharing very successful ended as for to underwriters cash to our million loss our posted investments programs. were and proceeds costs from over to September. the Total XXXX $X.X December in follow-on overallotment which

Habib Dable

priorities summarize like of remainder Thanks beyond. to Kevin. year quickly I’d the and now our for this

the in earlier luspatercept and X the initiate first top-line XXXX. trial BEYOND initiate Phase track full by myelofibrosis end with enroll trial plan of BELIEVE regarding in report and So and trials lower-risk we half the XXXX. to in are we MEDALIST trial Phase to X achieved also in Phase X Phase on MDS both to first-line, for non-transfusion-dependent continued results with enrollment X in this the plan And the patient advancement, Along trials first in mid-XXXX. our COMMANDS beta-thalassemia of year now Celgene, the we

Looking dose initial XXXX. will X trial dose-escalation X from this in the ACE-XXX, FSHD results Part we And of from at cohort January Phase X. anticipate include data update the arm the

in cohorts We XXXX. expect all data from dose

Phase with to of our results neuromuscular by initiate program advance trial year. to this X end X the CMT by continue Part XXXX. year-end with ACE-XXXX, also the plan systemic a trial We X And we expected dose-escalation Phase

in Also Phase discuss the we Anaheim. Finally, present results an presentation sotatercept to of and with in American oral half before first forward preclinical the the at planned Heart in week its an we PAH new we plan look of XXXX. educational to will the trial initiation X next Conference where hosting Annual Association webinar design

we work which We are these committed advance three within establishing three deliver development stage important each having programs drive upon and our we into of we them will late and goal X programs years robust as Phase to milestones. value clinical believe significant continue in toward of moving to to

areas goals evaluate disease stage focus and with Our has distinct in want to to business how we combined grow our our three time. late evolve challenged over to these us development

call term long vision plan help we a with luspatercept this We dollar as the deliver patients need. we turn the operator. have right strategy. where questions, to leverage for in accelerate and and a this be will to to to with opportunity franchises leadership drive transformative us hematology team believe build discovery to begins Our pulmonary open options robust multibillion pipeline In every and our therapeutic I and that with will unmet neuromuscular we the work luspatercept chronic management innovative anemia. development leader including area therapeutic life day fully And our drug in the cycle highest

Operator

Thank Instructions]. you [Operator

the Our first of from UBS. Gould comes question Carter of line

open. now is line Your

Carter Gould

Matt, the you of morning they been this is end, in updated design ASH as the on that you've longer program corrections good by specifically seeing can are MDS and luspatercept [indiscernible] and for to at Great, commentary front MS any hemoglobin know an help context, treatment you around the in in MS first luspatercept get think thank the still there year it reason through thanks questions. and to then expect I lasting on like we guys, be thoughts some Have the criteria. COMMAND you. put Two some seen will a details were holidays can of then do the study you in I'd taking yet for just your working line to IST rebrew it finalized the

Matt Sherman

trial was that's normally so in luspatercept three now lead scheduling sensor this anemab population, trials the again Hi so to an that and would , low for you in is hemoglobin have fibrosis those mild in patient our this as and study the we still days, we're very patient responses know in to Phase expect to questions, data is initiation extension the single any have the mentioned initiated our with Carter in very clear a we now year. regard while the numbers the luspatercept Matt first so of the about difference thanks still patient early study robust still with X in the we

population the to second Moving frontline MDA luspatercept terms on question lower in commenced study. you of risk

We tuned on for are initiating actual the when to an study study update guess that that I trial stay and give specifics we so planning of everyone get to closer design that. the

Operator

you. Thank

question next Karnauskas Our Robyn the comes line Citigroup. of from of

Your now is open. line

Unidentified Analyst

this on Hey cannot she Kripa sick, guys call. is she she's be apologizes Robyn, a bit for on the

when should question that you expect market ACE-XXX also dependent talk release beta XXXX, about the the meeting wondering to population. just study another stands anytime at a data Thank we a the for can the you. would little data this how initiating, be press non-transfusion in opportunity include bit if you you or early was medical thal you presenting you're So and

Todd James

first Hey communication in January, in release I'll goes, given there talk as and to pass then one take population, planned conferences the Kripa that the are muscle the to far no to we’d I'll MTD Matt data it’s ACE-XXX over Todd, about January. now

but be you of additional results the an conference press half will – pulmonary in muscle release. could press and data be year, the that So the to upcoming released first given at in then the

Unidentified Analyst

Great, thanks.

Todd James

patient do And to about want talk Matt, with the the population you NTD group?

Matt Sherman

Sure.

of the United for groups presented into it’s populations, and more the two the those transfusion-dependent patients population. group think severely be tend So, before you are transfusions in again, less of devided regions. being transfusion-dependent. The they there’s because maybe States Beta-Thalassemia of many non-transfusion-dependent actually more By patients, major more who affected, the each and those but of the being non-transfusion-dependent it the question. patients they Europe of actually certainly complications when we come as XX-XX% those of from the outside In about affected suffer to disease. misnomer patients non-transfusion-dependent And avoiding severely but the thanks population of

XX:XX. the of secondary lower associated have some certainly bone they have with So disease complications hemoglobin, endocrinopathies, of they the the

to they So be of are tend new sicker really in actually need therapies. population and

for the also the TD will patient and opportunity for So both NTD population. market there be significant

Operator

Thank you.

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

Your open. line is now

Geoffrey Porges

the and disclosed you slipped a ACE-XXX timing and what result Can FSHD us the in cohort? XXXX. know On remind in for January find what you? patients like can to that indication, we describe will participating again, and look the in Thanks. it slightly study? patients Is in I January demand good that will you the be they find small you’re in now And go from to or the have situation

Habib Dable

I’ll to more is you’re to about the first enrollment. But, tell it with part what hand take over the seeing and yes, Matt and he I’ll bit a the little right. respect to patients

the at us cetera, which percent January. from going And end look didn't identifying small at the of looking is the that the on goal to tail patient sense the that disclose function. in have being first the study, guided end year, is really dosing by get drawn at to to We enrollment numbers do data December, be continue now in at we dose clocked, on et in in previously cohort will the obviously, when of the really track, it really looking again, to mass, pleased And cleaning was data but and right on make to were and of we’ll We announced read continue we any function that plan now MRI looking conclusions a we what right to patient part study. you much at focus strengthened but of be the is the the with enrollment we’re muscle the and won’t look are strengthened we Everything’s January. that a the safety. disclose going first take to

terms Matt, what that, a exactly do to we, bit of be in enrollment? more with little in looking so at want will And add you color the

Matt Sherman

very actually very are we said, pleased with And well. Habib the enrollment. as So, it’s going

successive As two the we being muscle muscle a patients in muscle part is of XX:XX for groups dose study, in the patients cohorts and about and both first bicep talked in this part the study six enrolling the testing per cohort great group. asking muscles level. the next been the into We’ve patients with X:XX year also of great that by the site the There so number has has at the and very proceeding nicely. this been uptick had begin patient enthusiasm escalation

Society, lot MDA of -- ASH at that patients manages clinic a the as patients as and with both series. has national So around registries have society contact since seen well these the the Association, and country the MDA it’s many of

the are program [indiscernible] is participate and a wheelchair enrollment in shortage Unfortunately so the for disease because disease patients solution their also very that within trial. well severe out disease. usually really coming has genetic respiratory no very only now to our members tendency but quickly support drop for pharmacologic has dependency So aggressive sometimes right led this to even genetic more patients their family and the as pricing to particular to multi-family the spread patient in so word no of

Todd James

little And add to bit just that, to a that.

or we as increase are MRI what fraction. least in that and last on think as needed far in changes cycles will we show muscle also function muscle are within data for if up plan be We you the to three able will we X% take fat three are As [indiscernible] seeing at in we increases at and/or think after volume, weeks interested the overall providing on in to reduce fat looking very And in January. as we treat that and that dose intramuscular can for in to five strength looking the is we an the the as for then dose we an are and are compartment take months muscle patients muscle. lead fibrosis far what

Matt Sherman

one there’s muscle just comment just yes important. comment tissue is get very I muscles, not as the Todd’s are Matt more And to conversion one of this and we again, tissues the mean

Todd function talking the of different functional when between better terms traction in are So two that indicated we’re as muscle. versus you these talking leads muscle to the the muscle components, of about balance about contract

replaced muscle atrophies the as has so it’s that important able locked obviously So [indiscernible] themselves future is nothing and talent discuss be you at functional muscle, are that by muscle an and with with to analysis part that that do volume that we be will will to time.

Operator

Thank you.

Barclays. question comes next Our Choi of of the line Paul from

now is open. line Your

Paul Choi

for there at we regard the for My in for that us American first to to are want year? particular out indicator with at? question abstract Heart Could starting sotatercept you in are think anything any you data preclinical of you Association. next to that -- a what for look that properties is and you And data leading trial on us look be the good the the should terms may the call frame

Habib Dable

I I date to think before we data to of excited opportunity that Matt us be the more preclinical I -- Heart thought a you would researchers a to there is R&D have Phase Matt had little from we see bit the both you are to in of are the So [indiscernible] pretty that that. and from of share accepted guess pretty thing little the and first guiding some also to are well well, of R&D do and is date it from I say the a hand see as on again you you in-house American terms you'll [indiscernible] but shed share over more informing terms from the for with that from to bit some to that opportunity what that going once of forward in what from fact what’s little experts the you that and [indiscernible] as in-home excited again questions some we opportunity about from X and to on to but want at more and a moving presentation color and the us with model presenting have able obviously with answer us be first the preclinical

Matt Sherman

we pre-clinical Association. deeper have that at American Yes, so John presenting into dive going the here we're he's abstract Heart that Quisle to actually

John Quisle

the data at think is which Sure [indiscernible] in [indiscernible] at decision I of very types looking and across from the a data in great those both a failure, of but an had ways things measure collaboration. this of excellent pre-clinical look showed greater similar of Paul full comes models collaboration greater models vasculature than as pulmonary Paul in in a I pretty and you models as through of about at R&D seeing the to Matt, we're and loud Hugh where we as gold mentioned all suites Habib whole excited Paul vasculature, is and standing the viewed pulmonary with well then remodeling there totaling, in detail terms two see of mono so at right measure field the heart diversity standard with as the the we And day. generally as endpoints clear disease you it the at think animals HA the data we're

Habib Dable

less options, is have for XX [indiscernible] has them these upset able drugs know And disease potential approved five that of therapeutic years. the are sadly five earlier seven treatment in the dilated that all patients what all issues add to what as the year so be the clinic that to we somewhere none of indicated the in then of we within really that between the to macro have we of is pulmonary is for and as to survival expect to them US we've [indiscernible] be modifying, to a disease drug show

last drugs really on major first disease the on years. we hasn't the needle background So be the being XX this therapy potential having sadly for you can that know disease XX actually modifying or to know in it's have you added with decline the impact the that moved

Paul Choi

can us on therapies be they maybe [indiscernible] that [indiscernible] for currently remind therapy patients have as well. or and can the do itself thanks and to have enrolled Phase those Great, on the with X being you failed

Habib Dable

go the in more the webinar have therapies the that of Well details with we, a to into say of treatment a half provide of can design certainly little generally couldn't at pushed we well. and we'll extent of get bit current actual as the this and study to the of imagine year. and a first decisions care and next background inefficient [indiscernible] but of we'll we to will the would the trial you year end therapy, tricyline inhibitor those more the next just, plus of once detail standard be includes give quest discuss to to care just care PCE-X And [indiscernible] therapy have the the

Operator

Thank you.

of Nomura. next Christopher comes the line from question Our Marai of

Your now line is open.

Christopher Marai

the see sort quarters I First XXX really of up and if that's to benefits muscle maybe a realistic to update that if expect possible. you on was next see then wondering mass given follow know you under might functional is I've time and your which increases something in

Habib Dable

ACE-XXX study into mass. Chris X at Habib, everyone again, to your by parts. to is divided looking looking at the the it’s And Part primarily remind again question and FSHD CMT is finding. dose going do study so in muscle we’re for two that both really just of thanks

the is conclusions draw any function. study look strength at and designed but on again take and function, in to will not strength We a X Part

us second where be that what patients will will next – is XX half you terms that fashion be a our So randomized be goal controlled forward. terms we will FSHD functional specifically is will that placebo X in looking be of in study, in in initiate we at goal endpoints example, guiding at able of looking and study we to now in be to, timing in the for year. recruiting our the of And of study, to particular Part

Christopher Marai

potentially benefits that you do course to mass that can benefits, you measure an about in just muscle takes Obviously, functional thinking to think into increase trial? see that you I’m in time then right. those how functional And how relatively But long translate that potentially see rapidly.

Habib Dable

you guided in that to don’t know we guided if of thoughts terms you that. want to speculated really date, to haven’t we I that Matt, any much or but any actually yet? have haven’t share, So to making

MattSherman

yes, pretty to this function. see and increases might sniffing good within to that for six one effects you increasing the an of So, studies thinking And about actually very there’s twelve in weeks considering exercise muscle exercise start after strength proxy be this can beginning program. some

we one with increase although three been and of and this is in that might believe that specific strength we could time So that we imagine patients. range only specific haven’t part terms timing start in an benefit in to function see

Christopher Marai

just respect more non-transfusion-dependent of diseased sort thought with with in of given sort given or bone what’s they respect luspatercept the patients then And to what’s outcome that XX:XX. to improves luspatercept your of to are that the Thanks. patient��s sort likelihood

Matt Sherman

terming endpoints ongoing we’re monitoring that studies. in of Yes, our the one we’re

present So results those bone studies data we we disease? once able will in to be from have of terms

Christopher Marai

than But the by I have more patients luspatercept the bone patients? disease fact transfusion-dependent mean, impaired these that will

Matt Sherman

your I question. Maybe missed

luspatercept of have because activity I disease the with bone So … impaired more

Christopher Marai

luspatercept the impairing normal process ability on Correct. that Maybe to given…

Matt Sherman

the So the They range function developed necessarily, bone marrow similar. affect so more of have related bone accelerated The bone is they the the is in osteoporosis. disease marrow, bone remains disease the necessarily X:XX not disease. to bone not

ago they So the transfusion-dependent that do those that level bone that of number to in in same, infectious mice in models drug to a they we were But in years in remains the showed those bone biological patients. treated activity the the loss some the fact itself was by with that was preclinical of that able more marrow luspatercept. supported improve have data also ability believes

Todd James

Chris, then And Todd, I would add. it’s just

those half increase a majority a just had EHA patients hemoglobin. we in Our NTD XX most recent at update and to a over patients gram and of gram included

So that really confirm in in seeing Phase luspatercept this that X patient study. population robust activity larger with we hope

Operator

you. Thank

Our MacKay comes next Kennen the RBC. from of line of question

Your line open. is now

Kennen MacKay

wondered about more you different are how about if sort patients covering front-line number of risk lower and reports just dose. anything and for can of specifically ultimately ESA of in trial COMMANDS A that of here. trial of and you intermediate thinking efficacy MDF and there I is design responsiveness the given of sort the on talking comment

for thinking your get ESA to should luspatercept? wanted well as for comparative be we Just about the what here perspective as on

Habib Dable

Hey your Habib. Kennen, question. for Thanks this is

COMMANDS start details. yet So mentioned obviously disclosing with details on and design a the little to will closer Matt has specific that given more as we the we as study respect bit haven’t to we get of

the the we well going to are million $XXX $XXX probably the including are putting it’s of ESAs you one are be and that I be us extremely that all make to said at we be think we the Now now it’s are right we because United looking that things forward reimbursed. estimate at one being by for that the going in model is front-line benefit ESAs right sure about And important we that sure as that to now will that million that have to of forward know of embraced moving and will them a make that fact States important is stakeholders the payers. this used off-label setting I looking when really in that value say we in way

be it used this succeed that thinking right for be at type will to well, that our we to an so able we believe being now at is looking to be an is any therapy And of in-trench we will space. a do avoid step able off-label. from superior on-label initial us which that and do we if that And asset here, in we

with we be we getting in a that able doing that. pretty are line And traction designing setting able in by and barriers be first to will this into so potentially superiority confident enter to remove we ESAs will so study any against

Todd James

Phase patients back the or in had either respond eight only even add, results really as J&J Hellström-Lindberg response Hey database Kennen, it’s that and it to rates at has XX% also even off to their in predicts the of Epo score similar ESAs. transfusion per look units been study at as responded if scoring scale from any further that or Epo larger presented you Amgen and patient’s Todd. just XXXX burdens impacts X I that had or that the based to either two ability well they would Phase lowest ability if to X weeks the patients XX% levels on-board toe strong

before out So that we are being the the of the by and we details the in of to the look those all year. trial half able accelerate some trial forward still finalize are the to first initiates right working as things

Habib Dable

this will I that we announced will that study. be positive to may add RF negative and of be part be is RF that recruiting RF thing we both would of COMMAND close Yes, Kennen independent on R&D patients study patients that only the so September for the status, in

Matt Sherman

response covered a again was so One separate and studies we those better population. and line rate to cohorts in XXX patients point in large less is the rate patients by EVELYN a very highly the response the only got controlled study all here you so patients need a called Matt [indiscernible] [indiscernible] that very in important a very XXX parallel XXX was this receiving low all on of done because in a a than sub two referred patients Terence, baseline XXX puts patients by both have so of their search know trial, study responded, rate Amgen and very for favorable randomized study a arm favorable still dose XX% you active more but you I a with know first he response from this group to patients, that XX% of RK bring Todd a very responsive the have an know here placebo fine studies alpha group using the Jansen and

Kennen MacKay

a baseline up on frontline, the depo with just could obviously be at baseline. stratify of patients this maybe, the you're that maybe a I which could higher given study you couple responses some follow need that thinking depo then in through sort unmet about, mechanism in guess quick the that of enrollment in of And

Matt Sherman

design. detail be that to present as about one details that's the talk of we are study able the So parameters more the we'll for to able in

Kennen MacKay

this thinking we're on obviously are about Got up maybe about dosed the out Habib pricing should you, setting a your super that a ease in [indiscernible] a here, proposition be see potentially price to has commercial and and opportunity we bit to given we maybe depo the users premium patients or resistant to and acknowledging discount then year. thinking above parity one thinking little this insurance depo depo or there as here of just to of comments about in on follow could value priority

Habib Dable

know would you pricing, we've change guidance initial superiority design on a thinking to that right prove study. again, and respect the given the able DSAs be the study to to COMMAND not that $XXX,XXX we would with to Kanan is $XX,XXX now against So

Matt Sherman

patient you know over the on dosed you in units if get week they to suggested for As that $XX,XXX year. provision patients and stays gets well these at XX,XXX XX,XXX per a

Operator

Thank you.

Goldman Our Terence of the line Sachs. Flynn next from question of comes

is line open. Your now

Terence Flynn

too help talk potential thanks. to frame just Maybe know just the PH, wondering us of you think about of much on setting modification disease that for how in don't wondering patercept want it, capturing you a what just kind some disease but you I modifying about maybe about data just and the again the to potential, are of to trial, endpoints referred think how

Habib Dable

obviously of can there's we endpoints some a number bit be things pretty class etc, therapy we're vascular at more that looking you we've perhaps a on of already that again at, first could little that perhaps number know modifying the we're disease action remodeling the in a looking So the about. and elaborate mechanism of in could things Matt of even talked that John maybe about, but thinking you based be a this potentially excited in of and terms

John Quisle

So isn’t saying. what Habib this was

looked pressures. Some artery endpoints only are were studies these at typically pulmonary directly of the in the measured

peripheral a measure in the catheterization pulmonary lung function changes its and artery lesion. the within a in one using pulmonary and as measure could as in Also, addition, well of analysis CT care. the I at echocardiogram the pretty the our of pressures be imaging heart functional high measure so resolution valvular walk valve tricuspid functional the there itself improvement. be a look plexiform the X-minute also for to as such resistance regurge heart direct can heart, mean, tricuspid TAPSE the standard will measure some leaving at called status there of is vascular of the that’s functional which looks So in something now vascular function so a

behaves cells the is way the muscle the unchecked it's leading like of that disease I pulmonary endothelial to know malignancy. by non-malignant and mean, So you a interesting, There's smooth vascular. proliferation this cells, fibrosis within

So us is inform ability modifying disease to could there this not. some get or a at also look not that or whether process

Terence Flynn

drug which being some differentiated? But versus differentiated would those data that think because, the I which the would current current for do most most one of those see be you again, know endpoints. you drug of And

John Quisle

to it's simple the to And as affect going the I answer be maybe we the is more of really don't as that that overtime. affect anyone it’s differentiated really help most persistence see well and that a think

with the affect these over So we with not do treatment losing which continued time, dilated.

have these underlying to particular of product though over some even improvement the you in the continue So can parameters lesions some time progress.

If so and modifying disease be associated it’s that could And the activity. with vast. maintain perhaps we is could affect like and time every also

Operator

Our comes Securities. [Operator with Instructions] next Michael JMP King question from

Michael King

A been was of questions. to answered. a know I what lot want to Most previous have follow-up

Can about Just you're instrument guys of of quality start saying careful to know that? SF-XX you’re patterns prior wonder any about but I commenced, part in study the initiation of you been anything sort study I say to life just what being contemplating have the kind if you at the questionnaire are top design. as

Habib Dable

do Matt, to comment? you want

Matt Sherman

because you site have on want actually the careful full update no And our as start just when studies really to design. done very we although have very we clear saying -- that with the we're -- are being be we

to X show different anemic quality their hemoglobin for who you I related can’t And As of patients scores. X life straightforward a is we’ve in data well some are senior earlier been done of we incorporate programs. our -- had the And with look in improvement our where skills in too of -- say it's that improvement at to as of Phase study to our able an correlation in of quality who their were life refer studies Phase as subscale. patients as well subscales able across very different we've variety

we line as instruments So well. monitor life be yes quality bottom will of able to is

Michael King

looking And blood you are looking of minor been or about a regard you with to to measures going you’ve question, you lot things at biomarkers any and then going FSHD, muscle at muscle of looking be sort health health? -- one of you XXX, blood could sort be talked to of at I well, other case know also but are

Matt Sherman

we’re the only pile in the endpoints as such as certainly muscle markers so thinking are about muscle as the or is Part measured. and as MRI as muscle X Yes, of key well within Blood fraction stat different routinely components volume some at at total by study looking composition contract muscle. looking CPK

look can basis we those, to certainly diseases trial. actually correlate very tend and on which some those activity we measure But in these a in at disease well. very So those is really with tend be non-specific to that routine

Michael King

sounds FSHD. the moving know say than a just second assumption? than correct more the X -- And in the for in you more But in a am with dose about like study? you can dose considering what I half of using one it but respect maximum And rather interpret like dose. are forward more part that I want then Phase second the I X the don’t biologically than that don’t contemplating sounds of of study -- defined over a optimal Part sort tolerated dosing, to to

Matt Sherman

increase the the the in we to really show muscle. a a first based rectus the muscle, on volume question, the just were our able it’s femoris typical in in the muscle not to TA as and X experience we’ve keep dose to Phase MTD substantial X talked XX% defined the dosing, X% treating relates so three yes, to Part toxicity, the we part levels Yes,

that So treating milligrams up extrapolating muscle we XXX data XXX the of the size that and to able to XXX in three X Phase wanted to milligrams, that are the we’re some identify study, of the levels we and milligrams. dose test,

optimal take of choosing changes dose more that composition. yes based and forward we So on biological in see the muscle to

Michael King

I thought on going I question are -- there another Okay, misread to that or do a did read I that? quick sotatercept. Phase X, was you

Matt Sherman

think don’t I that’s No correct Mike.

Michael King

of didn’t think drug sort to the allocation was with I necessary. it question respect of And then top supply.

you you kind commence outside sorry yourself luspatercept on MDS do As -- infrastructure? Thanks. your for any guys supply, for Phase are X contractors, if in I using require expand how you you intend into manufacturing and drug the of doing would guys are am and beta-thal, manufacture so investment investigation you

Habib Dable

Habib. this Yes, so is Mike

to the Celgene cetera. and commercial costs, Phase So remind and vendors the Chris the Management by and X and from I et with off commercialization manufacturing related product, am do the then going is all Operations. it hand all Program this Two, subsequently I everyone Celgene over who is of the luspatercept of to handled Celgene that is I the will it funds But being to then Rovaldi Head kick XXX% drug the all of supply, side. want and thing first including

from a specifics get of And to little point impact on so shed related the [indiscernible]. light Chris you perhaps the into but can partnership bit and of view the

Matt Sherman

material just the for what substance, management that with and so we've XXX% stage and supply have Cellgene's we product chain to Cellgene a substance mentioned long as we standing for reiterate Sure reimbursed, our leverages collaboration commercial it’s well with to responsibility drug had the drug both drug relationships production. product mentioned vendors as supply the as stage team luspatercept now drug clinical

Operator

I'm showing like hand over closing for this Thank to any you. to at remarks. further the call time. questions back no Habib I'd

Habib Dable

Yes.

that forward in so seeing everybody everyone are everybody look to ASH thank much us either thanks and further and very So December, if no joining to a there wishing AHA day. with for at I'd questions great like or

Operator

participating conference have that thank does gentlemen a great for and day. Everyone, Ladies conclude all today's today's you in program, disconnect.

Acceleron Pharma (XLRN) 7 Nov 172017 Q3 Earnings call transcript