XLRN - Earnings call transcript Q4 2017

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Operator

Good afternoon, ladies and gentlemen, and welcome to the Acceleron fourth-quarter and full-year 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 call is being recorded. I would now like to hand the call over to Mr. Investor Corporate President, at Vice James, Relations Communications and Acceleron. Todd ahead. go Please

Todd James

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

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

goal look updated an overview financial forward questions. to Our priorities. and of review the to for upcoming progress, XXXX answering full-year your operational afternoon this results outline is we our that, provide our recent After

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

Habib Dable

our and quickly three extremely and business. Thank – and during time area enrollment us to proud in we outline we pipeline partner few neuromuscular I'm syndromes leveraged currently thank a pulmonary. areas year and of completed planning diseases. XXXX you, vision neuromuscular the first our made and lead strategy We today. September R&D about advance hematology, from trials And strong our with in luspatercept day Phase our candidate, to And of progress our and full is Celgene, and thinking to year the beta-thalassemia corporate Acceleron spent therapeutic myelodysplastic Phase disease II half Todd. in entire a continue rights joining for to neuromuscular III the to leadership which Phase of program trials to MEDALIST Good you build the lot one, XXXX recap maintain Celgene. finally, the events, and in in ACE-XXX, for of hypertension two us the BELIEVE of in retained we half begin for afternoon, XXXX. balance sheet. pulmonary everyone, within Three, a or II our arterial future. important first was an we We new PAH across of allowing sotatercept a Two, we

offering XXXX. net With of successfully the follow-on cash completion a raising $XXX.X in runway our extending into in the fall, proceeds, million

from drive the With important With clearly trials, and defined therapeutic XXXX review growth partner strategy for BELIEVE to for and in each clinical events report with long-term these and on topline Phase we we our MEDALIST on hematology luspatercept. beginning the strategic operational with ongoing to and treatment are track year, highlights results Celgene both the in our execution focused product vision enrollment full I'd looking most are candidate completed lead and III value and and and beyond, to to trials area, creation. focus like last mid-XXXX. our forward now recent

we officer, launch from joins in red chief late transfusions frequent potential blood on us dependent who Based hired erythroid that will are red cell Sujay multiple luspatercept's blood an designed is in multiple results differentiation I important II our medicine late-stage luspatercept TGF-beta cell inhibit anticipation we to a minutes, it know, of anemia who patients could chronic recently due blood commercial disorders. of across you on proteins regulate be a As discuss few AbbVie. maturation. that XXXX, rare maturation thousands for agent an In Phase believe Kango, to and commercial

luspatercept for trial a of first preparing We and to regulatory multiple filings Sujay product lower initiate our And of luspatercept the to risk doing alongside commercial Phase partner the team positions trial us called this Celgene. first-line accelerate franchise work prepare luspatercept readiness COMMANDS been as product we We're lead support launches with are to ESA-naïve and across in for expect fully his we we've also the III in companies trial half MDS. experience join thrilled the our have our new XXXX. the to candidate and

results planned and of the We patients. patients several over opportunity of The II in confidence trials II to Hematology, updates risk fourth response luspatercept preliminary non-transfusion Society planned our of we in extension from with II The enroll dependent and Regular ongoing the clinically-meaningful base II luspatercept ongoing clinical trial demonstrated Phase continue making December, patients ASH at XXX our straight presented depth XX to where to benefit transfusion combination long-term monotherapy build enroll drive ongoing. with XX% ASH, in XX% the luspatercept's We red the trials to in of reported Phase updated American upon new in MDS. myelofibrosis is erythroid independence patients. trial BEYOND the and achieving the over cell presented therapy beta-thalassemia Phase patients. trials Phase lower is of blood or and the in update results patients annual luspatercept

MDS looking As a luspatercept. dose patients results at all with reminder, therapeutic at levels treated are these

also baseline status. benefit provide as to to presented years subpopulation was have RS experience continues significant patients, response We EPO also multiple treatment Duration treatment. rates now three and of long-term luspatercept response levels, specific on nearing ESA on based

initiate patients or ASH, II than in further support These X,XXX a sotatercept. slide. MDS. to results a on have we for now on decision an II II lower-risk experience therapy update company-sponsored at luspatercept's monotherapy be years anemia of results XXX Phase the Phase inform investigator have to transformative with with XX% treatment potential I previous provided patient option patients in that Anderson trial combination With with Phase the both of The mentioned and these at more trial an MDS. Also myelofibrosis months M.D. Over patient the data, achieved response. lower-risk luspatercept collected the

muscle to reported initial continue one and demonstrated biceps preliminary with ACE-XXX, trial we tolerability agent ACE-XXX the and treatment in franchise, our demonstrated escalation Phase recently results FSHD. the locally-acting neuromuscular fat our cohorts dose cohorts in Both mean also of advance muscle part reductions of patients of we to The fraction. the XX% safety of out over and two cohorts. data from II where and increases one achieved volume tibialis total anterior in muscle brachii Turning

identify into part part to level in dose forward two. changes safety As evaluate one to a muscle take key reminder, objective and is of and the to volume a

and key strength For on initiate muscle the and are in We're the reported well of objectives encouraged as part changes two part volume the to to of two, in date as data function. trial XXXX. the quarter track are muscle second by

or advance in of to part Charcot-Marie-Tooth trial the II two II second trial initiate and the disease CMT to ACE-XXX II preliminary continue Phase report Phase XXXX of results and from to XXXX. of half by the in in end trial part our also We plan one of Phase CMT the

safety, I and three recently systemic The agent, our will trial evaluate tolerability single we multiple with pharmacodynamics. cohorts trial and to ACE-XXXX. For initiated three healthy a Phase volunteer dose enroll ascending neuromuscular

We XX this enroll total a of expect healthy for volunteers trial. to

at Additionally, encouraging the sessions our its with and sotatercept PAH, beforehand hypertension animal in results remarkable XXXX of Hospital PAH for we Based American highlighted to supporting plan from collaborator, on a host positive sotatercept Brigham further vasculature disease potential educational patients initiate approach discuss pulmonary mechanistic and with for preclinical modifying Women's two plan our Heart half on and focus Data first-in-class to to during first across biological November. an Yu II a with pulmonary data, models Phase preclinical in we pulmonary, Association therapy Paul showed activity with trial in the arterial the in trial our scientific design. well-established webinar to PAH. be of sotatercept the

So, quarter. through lookout to Chief now the soon. out a McLaughlin, call run be turn Officer, coming on Kevin that, to please our over will financials the Financial for our for the I With save-the-date

Kevin McLaughlin

afternoon, Good everyone. Habib. Thanks,

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

Habib Dable

Thanks, Kevin.

half topline ahead year initiate in quarter we by to year our one Phase plan half neuromuscular, for As I In the hematology, part ACE-XXX second the of touch III part midyear from to dose and and you productive XXXX. will priorities year-end. the CMT have of trials provide MEDALIST our busy all of the and of second plan in expect in by FSHD we us. FSHD of begin a XXXX XXXX CMT to Phase results and briefly heard, COMMANDS and part the have cohorts first In we on in and the and present trial remainder to from BELIEVE III beyond. results plan two two

XXXX. have year-end II closing, present the we or half discussing our sotatercept In our first goal ACE-XXXX, years late in PAH stage forward the III to half the finally, during and XXXX. initiating of of first and clinical during results XXXX Phase programs in moving we committed in to I a the first of within XXXX. looking to in pulmonary, And is development design three with into programs look by three Phase remain trial to we to For we're forward Phase plan trial half

call drive I our the Our questions. neuromuscular therapeutic this long-term in hematology open as area We to turn to leadership build disease. Operator? leverage luspatercept to multibillion-dollar in will one remain now with number priority. our ability our vision pulmonary in ability with which will and luspatercept, confident in opportunity begins

Operator

question UBS. [Operator Instructions]. from Gould line with of the comes first our Carter And

Your is open. line

Carter Gould

opportunity a ASH and III as of activity study EHA Hey, to development in recent on anyway Phase how the guys activity. since coming you of at past space start up? Thank you here Has readout guys. commercial There's Do you. the taking MDS the data plans? or for looking are years business Thanks still Phase share intend question. potential at MS the given in II the the the year, afternoon. changed lot Good that the in been development

Habib Dable

is This Carter. question. your Hey, Habib. Thanks for

have with anemia. JAK specifically, patients in particular And spleen, So, – space. the to inhibitors deal severe by – drug-induced specifically, myelofibrosis, to Impact But that you as of luspatercept. not or acquisition JAK are with also inhibitor, myelofibrosis these with suffering in the activity many move there And, a also myelofibrosis also is studying volume an agent the recent are And given enlarged need. their and with us patients that a in One, space that partners, are lack quite this patients to cases speaks encouraging to in moderate finding respect are space but patients those looking give opportunity suffering for opportunity in are opportunity it's from studying treat And disease-induced very you With a on respect for the in anemia. terms the only themselves suffering committed commitment right. the you more at cases, for But for of at on I'll Celgene because, it something the we to an looking idea EHA. more for recent needing to pass potentially in having who patients with patients to from it many from of expect on forward in they fronts. really anemia, And the know, comforting unmet we number know, anemia. also data, with the to they're of a which that available turn what in very frankly, of as their given encouraging hopefully rociletinib. is more we And, we're you're over as one will is Todd space, having potential to to it potentially them Celgene's an pharmacology alternative see an can the space, luspatercept. to to luspatercept. work fact so gives open solutions are luspatercept of available to

Todd James

Hey, Carter.

development same. progress. data remain off first with trial continues it initiated see MF to just could so about year, Celgene Phase expect that you the clinicaltrials.gov, talk last So, myelofibrosis. And to II in and We midyear trial the based to to mid-to-late XXXX the plan, continues about of

we've As far frequently, updates. quite the medical as been II at our presenting data this congresses, upcoming plans every Phase six-month

And we to as submit to the accepted continue us congresses be for continue the whether or so, will as will for IIs far abstracts not. upcoming Phase to say. Hard

so, we'll And anticipate ASH. it this presented EHA when at color able least would be to that the or But at IIs be live. would one go the provide be Phase year, abstracts time we whether at

Operator

the Porges line Thank of Leerink. Geoffrey you. question And comes with our next from

line Your open. is

Bradley Canino

ones our end. for Thanks clinical Hey, Brad on this couple questions. Had is my Geoff. on a of for coming taking

based First patients think proportion do knowledge be right on for the RS-negative. study, to the population, COMMAND of are your you RS-positive now luspatercept, of what in versus likely front-line

Todd James

Todd. Sure. Hey, Brad. It's

So, been ESA the a population. refractory as lot you patient about second population the MEDALIST know, is given prevent line, in talking indication we've the

disease. the median. XX% RS-positive incidence so, lower live are talking in population. we And RS-negative in to their about patients And patients to versus about recently five years, most believe prevalent eight due have the there's the slower five-year we more, two-thirds pool, been to of the on the progression These

like they'll three So, five. progress more to

RS-negative so, pool due to population. more patients, difference that the the towards the actually time progression in patient the incidence weighted And was for

prevalent we or RS-negative in like you incidence then, see that setting. two-thirds that is first-line the you the refractory to think about conversion setting, believe or And the due pool. more when within probably So, rate that progression the

Bradley Canino

that and moving Okay, forward thank how to taking you. ACE-XXX, FSHD would into muscle us tell many for can two you for be part on you dose give? then, required dose injections have the you're yet And to disclosed

Matthew Sherman

Hi. This is Matt, Brad.

at data treated, of also So, were the of as in terms increases anterior one volume and Morgan showing measure fat study, muscle we the the in and and in J.P. a of function. that muscle the folks updated the FSHD very part important biceps muscle, from fraction, quality in tibialis the decreases muscles

that We we'll have forward yet not disclosed levels part the be taking trial. of dose two into

have year. We initiated will quarter part in of this in that two indicated be the second

give tuned information stay about and you we'll to be So, further able that.

Bradley Canino

wanted timeline. just I then, a And to clarify

– stated previously had You

Matthew Sherman

I'm sorry.

drugs You did ask about and injections. We do for injections. administer the

into we the Phase increases muscle the of healthy the four we significant injections the TA I as in both total the as take also that So, is volume we across muscle. rectus length trial time divided and in very dose what This the showed in piloted muscle at where well femoris in muscle. volunteer

Bradley Canino

preliminary, one your will from data but And Okay, the then, part XXXX. full you previously be preliminary to for in by results now trial CMT cohorts? still release you. release that's committed escalation you dose year-end Are changing Thank or a that press with clear. your stating guidance all

Todd James

Todd. It's Brad. Hey,

still of last weeks we preliminary about So, second part we results follow-up cohorts because some half, dose. the – the dose able will it one after from period, will be the yeah, past – be three in have

that the And that year half the of to so, CMT, both three in we won't be be be we all second until weeks provide follow-up, final. through of But further update. dose after to the FSHD say get we'll able cohorts and able through last we'll for and

So, specific really, little but year-end, no change been in guidance before the we've half. bit second with still a more

Bradley Canino

your for be will the you've between could And for what healthy muscle at Okay, expectations XXXX, therapy, you of size differ this measuring compare on And, volunteers will got what how increase in the I looked two systemic and guess, molecules? XXX? it. you then, the from

Matthew Sherman

Brad. Hi,

here. Matt again, So,

So, ACE-XXXX systemically agent. administered is a Myostatin+

focused did So, your in [indiscernible] I'm as for question? in be of scan and changes have we on extremity we increases trial, muscle part those MRI MRI by the sorry, we'll looking the second scans at lower generally patients.

Bradley Canino

expectations systemic your on more what acting versus for know from muscle to volunteers increase agent. saw your in size healthy you wanted guess, I agent of a local

Matthew Sherman

volume. So, increases we mechanism, to again, expect muscle based on in substantial see the

in ligands. to acting versus of comparison the the administered agent, given locally of target We same do range terms haven't information agent but head a of systemically they really the head

So, increases see muscle expect we to significant volume. in

Habib Dable

increase far able our strength similar function guiding patients. to as and is Brad, a success, really hopefully how expectations need like we in subsequently with for feel as X% a we're agent for But systemic be – or to acting ACE-XXX, what you either then see locally to

everyone So, hoping that's And give more in see in into a I II. on at we the go healthy bit volunteers, Phase to to – something the MRI then, and everyone hopefully, also subsequently we're have the color earlier patients volunteers Phase ACE-XX images both the healthy myostatin that little to with Phase baseline really diseased initially I, at to in thigh I in when selective agents Phase just muscle relative in trial patients. in in looked our look change

Matthew Sherman

initiated will And half of have as in in and, data trial XXXX. we that timing, of trial indicated, first the the terms from we've we

Operator

all from Thank you. me. that's Okay,

Habib Dable

Brad. Thanks,

Todd James

you. Thank

Operator

Thank you. next Karnauskas And Citi. line Robyn comes with our question of the from

open. is line Your

Greg Harrison

the Robyn. Greg for for Hi. This is Harrison taking on Thanks question.

mechanism the guys you one you would how from decide that which is target XXXX. anti-myostatin, does action indications on Another to want help of with Since it? typical different

Matthew Sherman

the development, this indication some over detail there, And not DMD, talking in really indication detail, for the muscular and ALS we we've that although into R&D presentation mechanism were went Phase the spinal II novel back And an FSHD, about We yet for Greg. include Thanks, we went September. some at in ACE-XXXX. selected atrophy.

preclinical muscle increases data of showed those preclinical showing that in range, a several of in amount in very fair indications. So, we meaningful

move as to – be with the be would program. able we So, provide to updates, we that along would you

Greg Harrison

you. thank Great,

Operator

from Joseph question Morgan. our comes next you. line Thank of the with And Eric JP

is line open. Your

Eric Joseph

guys. for the Hey, questions. Thanks taking

response Just on docs as of one sort sense trial months. color COMMANDS going that for date it of Is design therapy, Phase the and sort of reasonable against be you're superiority on we differentiation trial. sort [indiscernible] But data want seen expectation some will on response up or know gets that to we've as historical the Thanks. focus more I the what of longer-term erythroid to that on a the measures, me sustained in would get prior based erythroid considering related here. additional the duration coming to from or view thing? on I get a that would just underway avoidance, transfusion meaningful ESAs, of II

Habib Dable

Hey, is your Habib. This Eric. question. for Thanks

than subsets II you XXX where the trials than the rates XX% endogenous to with trials important. we in with our So, unmet company-sponsored doctors looking probably the And respect had there within of where ASH to had to you seen what that HI-E so to at guide can have I'd becomes that And were trials see presented EPO in response, if you two less the you where or of far, were improvement erythroid, HI-E also the need XXXX. above of was Phase those with the where rates you really some compares quality XX%. specific a response levels, response to When that a cases, took those at response, two that XX%. look at respect of extremely want. respect and you HI-E HI-E rates data And XXX amplified, to less hematologic

response. and rates of continue when our patients luspatercept, you So, one response we're from very therapy response take quality gain last presented quality, one the do in patients there, We the moved rate were is also the of XXXX. follow-up when of we data XX.X II of ways response gaining where was studies, patients back to know, duration. And months. able the on think beneficial of only to April the look that months. not EHA duration Phase well continuing of ASH you year, important for information of profiles tolerability encouragement at as were [indiscernible] And you data as we at as here responders to of at because cut with touched And very that update to that who safety TI XX you quality updated biggest to the point overall the to benefits the the we of again to of September on a

II So, these transfusion really for again, of important of patients independence going reminder, MEDALIST And quality the the to within response us of much of that responders, when terms again, were study follow in to rates XX%. be very informed as Phase or the study you the think continue seeing patients. us it's a to the

of and is study in COMMANDS number going head-to-head of those markers, of from from to design So, a go but study, that a also from when not think only based on into the with of J&J. company-sponsored ESAs we superior right fashion against you certain all a degree data the studies points, confidence our thing do Amgen absolutely

Eric Joseph

maybe as of kind in to how And baseline of guides patient those scope it. and see with in you're that characteristics, are what Thanks. levels Got just and those reference of terms EPO useful studies, J&J of baseline targeting terms might COMMANDS? size following the and we in trials sort Amgen up of

Matthew Sherman

Matt. Yeah. is Hi, Eric. This

There that expectations open-label so, I to were go in they rates. two studies think, two done of ESA falsely actually response terms that ESAs. elevated were a randomized of studies into only Just with of lot studies, they were were done the that, so – those

Jameson double-blind was study was randomized J&J they well [ph] two patients. patients. these Amgen So, XXX were study The The studies, XXX controlled studies.

XXX again, the robust HI-E in So, sets. for study. Jameson data less And the response the was study XX% the the IWG patients levels response study. than in and less XXX than Amgen J&J rate the only only responding Again, population in in in with XX% population ESA study, fairly the showed Amgen

had high of the patient – –as the able we degree excess lower population of Habib and XX% similar a patients in data that that risk, reported that shows first-line is responses population the So, for be we'll prevalent one fact, that, a population. MDS superiority compete show with we've feel to in and

Todd James

Eric, it's Todd.

about far to and we're trial, head-to-head more go that's as then you think into as far for be and versus we'll with endpoints, these should size that as can what III superiority, and that to as the potential closer J&J our far to some BELIEVE of MEDALIST As of get a and you line levels given show something going expectations few in the and Phase the Amgen trials as in the here. trials thinking baseline be just from months about how initiation trial EPO want

Eric Joseph

Got it. got it, Very helpful. Thanks, guys.

Todd James

Thanks, Eric.

Operator

Thank you. And the of our Marai from next question Christopher comes Instinet. line Nomura with

line open. Your is

Christopher Marai

of I see for and the questions we you with might XXX in Thanks outcome progress. able FSHD, I year, Hi. Thank in, wondering really us second the your you. congrats a maybe further later that registrational what you would measures can trial respect to on might half perhaps of would for taking be on what remind plans this we should on the data looking assume, all some XXXX, there. be functional that would to terms be early kickoff here was if re-examine that comment of maybe

Todd James

Chris. Hey, question. Todd. the Thanks It's for

move clear provide So, year. part able registrational, to into half that then and then, that one dose a need And parts That's and one be and one we believe what in the we then data in as either that we part cohorts II. health to two III Phase on we're – registrational the just Phase don't believe earlier we to – we'll after be a cohort two three and part as the two authority expect are you far And from take part we'll of year. updated get in second the feedback. could the want

patients ends and and placebo. per to expectations, as be continue there's far no function group As small, the patient six strength

nice So, quarter as this expectations which really on two latter the could we'll I'd as far and off a then half in a to XXXX. you in and two function, read of year will randomized, where you of the of get be strength able point placebo-controlled and the results strength expect And function really trial will for larger be that second kick from to trial, part part endpoints.

Christopher Marai

Right.

And you. we to should on Thank you're so, that of the design expect an on looking update when there? kind execute

Todd James

Sure.

a the that and kicked you in XXXX gave at we on updated trial preliminary since design So, the which we've an off update when out late for can look design clinicaltrials.gov study we look shortly.

treatment. other three – updated six-month six of limb in like were run to walk time initially point two be for We've things test, we're looking on on and dose the on going two that, cohorts. that walk across six-minute will FSHD randomized And at an focused there's the expanding at upper XX-meter XX be of also placebo-controlled, patients of the limb, and upper ACE-XXX extending health in we we size inventory functional endpoints one-to-one. to And walking. We'll XX – up cohort looking the the we'll and performance score at that to PRO in interested looking treatment also patients randomized, the months so, each patients, be XX,

Christopher Marai

functional with patients affected one endpoint or suppose, of potentially is you then, to And Thank could is outcome series patient would you. be to might FSHD a intramuscular muscle basically on worthy outcome, to there the registrational move target able just Okay. that respect able be, some in a benefits that I clinical by That you with modification an any population, groups. form of the of endpoint? to lastly, injection. similarly resolve, be respect Would group a specific that could you

Todd James

your question. to So, understand sorry, want just Chris,

of anterior to at bicep, in the different look we You're the outside tibialis potentially, asking patient try muscle affected a or could quicker? if to move

Christopher Marai

Yes.

in by identify whether a impacted those would sub-patient with population design you two lack could most be groups muscle meaningfully trial? to FSHD So, with muscle groups, registration particular to those a respect even of that function

Matthew Sherman

Chris. It's Matt. Hi,

eligible patients it's mild that weakness. mild right I the for designed folks moderate not taking it's normal are subpopulation have the to have are FSHD who have in We're who muscle patients patients weakness. severe But study designed who function muscle think weakness muscle the the it's to moderate have study. now, enrolling that as nor of we

whether their this And weakness So, foot then, function or in have the one in not drop biceps they into of subsets extremity course, population. them anterior a those in muscle it's cohorts of of upper the tibialis in and puts the in a or the two patients. or walking

the now, III. this, is we right way So, fastest to Phase feel

be off this in of And the we of that we second [indiscernible] Todd study indicated, will the part of the half part in from data kicking as of the latter the study just two two indicated, the quarter As XXXX. year.

Christopher Marai

Okay, thank you.

Operator

of Thank you. And line the Barclays. the with Choi from Paul next question comes

open. is line Your

Paul Choi

Hi. And you Good some the moment all thinking think – about about regard studies approach questions. evolve thinking in here I'm to a thanks that your myostatin-positive maybe any the more are Can might NA how XXX your back still about, our is of competing just market your maybe there the with stage. landscape I at to afternoon. recognizing approaches. the just assets versus here. mind. mind, could competitive versus active evidence some the If a for supporting for of early And in competing [ph] And taking XXX go

Todd James

Paul. Todd. question. your for Thanks It's Hey, Yeah.

antibodies combination molecules or acting the is point So, NA even And agent. make a disease. different these myostatin active that all Regeneron's of to recent I selective of myostatin selective think different are there's here most plus ACE-XXX [ph] locally selective an very important is very for is

for diseases that CMT the where other diseases these like muscle ACE-XXX from there's local so, even local so other afflicted that are is ACE-XXXX sweet have systemic muscle and in really with and muscle our spot systemic and with molecules approaches patients weakness. weakness weakness, is patients FSHD, And

franchise with a systemic as with synergistic portfolio, a certain be both of might ACE-XXX and to But so, locally And lot compounds our diseases. in have any our agent. And sense there view a being two even systemic we with in acting wouldn't agent. the makes approach competitive

Habib Dable

Yeah. Paul, Habib. this is

Just to quite activity opportunity neuromuscular seeing beyond actually other trends important delighted Paul, add ACE-XXX in got for of diseases. Myostatin+ GDF-X been that a now agents both reasons, of But serious both by XXXX now, to seeing you're diseases. that have or ourselves admit that very a significant you're we're in the as I treating and of more positioning that in terms as that in confident just know, the fact systemic [ph] local events terms we've the selected fact right the as and role with believe, agents course, we from GDF-X Todd, you myostatin. the very with are, we've to as comments being of to a studying act we a and fact be some – treating agent potentially that will the neuromuscular various well inhibiting play these But do obviously,

Paul Choi

might that see would – top just Great. out something would do regard we be in is with a I at result that medical something the the half And you year, line would that Phase coming be to meeting? second just or initial that of the with a

Todd James

Hey, Paul. It's Todd again.

of the be a results would update yes, year. be clear, at I of something will be would the medical Phase that in to XXXX So, the first just that first congress half and the probably we half

Paul Choi

Okay.

Todd James

with in conjunction release That conference. include will the also a

Paul Choi

Great.

Habib Dable

Phase stands the endpoints or to and regard either that being else with focus of the out PAH on some PAH less incrementally endpoints is as would anything the data more want on likely of you with the mind regard one would that to presented, the anything significant you quick be to think regard your in sort forward? to one investors going probably from II. With that you classic just that initial there learnings then, And

Habib Dable

again. Hey, Paul. It's Habib

So, obviously, of I respect endpoints, key we as this. think are, number think we to with collaborating with stakeholders the our a through

to input. from external some shed we'll do regulatory And colleagues call, experts. with the this internal benefiting we actually both during plan our host obviously, as would number well a And where are, We as be lot the time, we well as to focusing we'll that initiating you the be trial more on, studies guide design, on duration, of cetera. light endpoints et specific the webinar patients,

more So, come. to

Paul Choi

Great, okay.

We will questions. the Thanks for stay tuned.

Habib Dable

Thank. Yeah.

Operator

you. And comes with the Thank JMP Securities. of question next King our line from [Operator Instructions]. Mike

Your line is open.

Michael King

therapies and/or some other have for development directives? rethink strategy what questions think of satisfy about on Thanks just that might risen on DMD guys. FDA you following XXXX. some in. for just recent couple DMD good, wonder to I of that – up agency decisions in your suitable the surrogates Hey, could caused about for guidance has you and timelines XXXX squeezing of wonder me if on the recent just of I'm sort a And

Matthew Sherman

FDA Hi, opportunities great of is came the new that particularly guidance actually companies out, their a Mike. to some that to are This diseases, these for aware obviously, Matt. with area. we That's the question of some spoke because, of quickly more move in agents this

of these composites being a novel opportunity gives of and fully trail quickly. move think development the to us come want I potentially endpoints on with endpoints. aware to edge and new cutting the with be agents really or blaze up we're of So, aggressive them. And We

right it's target specific the the be what the about early a be to be specific too [indiscernible] and might timeline now considered endpoints think I little diseases. to that in what could specific

Todd James

Mike, And, Todd. it's

to be ACE-XXXX. indication haven't we picked clear, a for Just lead

disease discussed have of DMD that We as interested one in. areas four we're

evaluate ALS. FSHD disease with therapy, to area that and continue we along systemic So, SMA

in. XXXX, next is lead tuned think area that stay as more the we'll months the we the So, to XX Phase comes early In XX over to indication. the discuss I able be disease

Michael King

Thanks, guys.

Habib Dable

Thanks, Mike.

Operator

Thank questions further this closing would turn to you. his now CEO, for to call I'm Acceleron's showing remarks. Mr. the back no Dable, I like at time. Habib And

Habib Dable

Yeah.

to company. want interest for question, great in upcoming Wish their just evening. a look all I congresses. everybody your continued So, you forward for you And thank seeing our at to

Operator

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

Acceleron Pharma (XLRN) 27 Feb 182017 Q4 Earnings call transcript