XLRN - Earnings call transcript Q1 2018

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Operator

Good afternoon, ladies and gentlemen, and welcome to the Acceleron First Quarter 2018 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 Brian. our available corporate press at Call. financial to welcome, on release www.acceleronpharma.com. addition Quarter our Thanks, page everyone, website Conference Investors First & And of webcast, for presentation today's XXXX Media Earnings reporting in are to the The

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

and afternoon progress, for the updated our is this we an provide first recent quarter goal after along overview forward questions. our operational reviewing your that to taking of priorities financial Our with look results to corporate upcoming

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

Habib Dable

and for us joining you afternoon thank you, Todd and everyone good Thank today.

luspatercept with off focus long serious for The by discovered area an pulmonary Celgene medicines internally our high first-in-class strategic the anemia chronic and business So we this rare term leadership patients advancing our best-in-class harnessing development or develop to period kicks building opportunities. about this vision unmet of disease. power first to for leadership our therapeutic medical with had for therapeutic continue and and important and collaboration XXXX medicines productive as as starts foundation patients. diseases. need in vision plan partners a TGF-beta in of to to new areas we a Acceleron biology and build We expand quarter neuromuscular think and hematological, of We our our

across We and additional multiple clinical are lifecycle five targeting prospects. management evaluating currently are indications trials

and agent, first with utilize Our wholly-owned both with our Phase In local trial Myostatin+ franchise that uniquely systemic inhibit pulmonary multiple proteins. or muscle is approach PAH. positioned X a to pulmonary close are arterial hypertension we a our initiating to neuromuscular sotatercept in TGF-beta very disease,

to prepare to our and development. luspatercept for fully committed late clinical stage continue remain We key activities moving in to upcoming programs

be execution our is we team to operational on programs XXXX by and three of late toward entire focused goals. XXXX. push integrated end as a meticulous developments X company by have the Phase Our To a pharmaceutical in fully by

on of we X dependent lower plan the release robust quarter. results top this non-transfusion-dependent versus no to chronic Myelofibrosis risk risk the of in well and to multi-billion year, with expect luspatercept. annual have to MDS, Turning This BELIEVE X BEYOND lower the the who Beta-Thalassemia beta-thalassemia with are in Syndromes line The beta-thalassemia MDS track luspatercept trial trial evaluates trial treatment in in as transfusion the or approved opportunity with diagnosed patients in anemia, Phase in seven across treatment first patients line positive Hematology, clinical or and luspatercept Enrollment trials MDS, middle dollar successful trials superiority with epoetin head-to-head ongoing MEDALIST Phase peak medicines present The have rings Phase currently to indications. as Phase if treat trial v and and Phase X Phase X ongoing trial remained Myelodysplastic Beginning Myelofibrosis. at the initiate to a X are it COMMANDS currently X cash alfa. year. patients with ongoing we sales

website provide Phase an in X to During abstracts quarter, Hematology released Association expect ASCO be the XXth. progress in Meeting ASCO Meeting a Annual May EHA on At Phase luspatercept to design be and the ASCO, also presentations on the and trial meeting and ASCO update are to we The the expected X the available trial or been will the outlining on titled poster upcoming plan European the ongoing and there trial in have to second MDS June. we present update Myelofibrosis. at of Annual presentation

conference For outline release conference, X at press XXth. structure to on presentations the EHA, May a we plan the roadmap available to our once distribute Phase out

with and a therapy. now muscle Turning beginning franchise to ACE-XXX, Myostatin+ locally-acting our neuromuscular

the have muscle X the all controlled part effect one milestones at ACE-XXX the Earlier were half from initiated results and issue. the of tibialis is X of multiple in the trial the double-blind during We we announced mean of six the Phase presented cohorts outcomes results evaluate We Meeting Angeles volume much of milligram in nerve which of preliminary XX% on Annual at plan muscle safety in disease healthy over favorable was a XXXX. cohorts one and selected part after tolerated profile months part XXth year, X final in in treatment. results We ACE-XXX X in as the quality Phase showed controlled dose Phase recently oral placebo We last this absolute part fat double-blind overall trial brachii ACE-XXX well in those one for published total journals presentation improvements from increase in demonstrated which in of Academy from of American achieved second and preliminary the trial in FSHD observed and functional two program anterior fraction. reductions placebo present in Phase biceps muscle of XXX to Results to muscle also year-to-date. a neuromuscular Notably, FSHD, a of two was session. with you month patients Neurology and best Los data volunteer a

results expect second of report two We XXXX. preliminary part to half during the

for Fast for that announced ACE-XXX, currently and that program medical patients the with FSHD. there granted recently no Track are milestone which is therapies. the also This designation high in FDA approved FSHD need the important FDA confirms an for unmet it We

We where are X we in X Phase trial through one we to the or by advancing full end one preliminary Phase year. part of disease, of developing in two and to currently this half second XXXX. from report plan are ACE-XXX We part initiate CMT expect in also the CMT trial the of Charcot-Marie-Tooth results the part of

ascending The evaluate pharmacodynamic safety, systemic will three our in subjects. evaluated being double-blind cohorts tolerability placebo ACE-XXXX, in volunteer X currently muscle enroll dose trial XX Myostatin+ to Moving and healthy is single and trial. multiple randomized controlled agent Phase three

walk vascular Key over of to are will three XXXX. worsening, is expect placebo during during plus change plus a the report clinical of assess in X control recently care first treatment six with life. double-blind we placebo-controlled treatments. arm in period. or secondary trial the functional and webinar across endpoint We standard PBR dose dose We Class the PAH in The A Phase half to results patients and standard educational an PAH PULSAR plan primary quality of of enroll on class minute Pulmonary, low distance, care. design resistance to summarize, a the design trial and X X highlighted Phase trial. March XXth. of include X pulmonary a from be briefly trial this X Functional To month endpoints sotatercept In high XX change

run the now trial We With turn the expect through this preliminary that, to report Chief initiate will the to XXXX. PULSAR half of in for I Kevin and our to quarter. results call the quarter McLaughlin, the Officer, financials first Financial over

Kevin McLaughlin

Thanks, everyone. Habib and good afternoon

cash, equivalents to and XX, XX, were $XXX.X investments cash cash, cash investments of as Our XXXX, of and $XXX.X equivalents this XXXX million, compares December March million.

R&D we projected Collaboration by be the in existing first to open and million. and now of that luspatercept. was reminder, call the and questions. cash related cash, expenses expenses believe for a the from is incurred is The Company XX, quarter Operator? Celgene XXXX. posted XXXX first expenses for the investments G&A loss equivalents will Total March million. net million. a of $XX.X $X.X million ended costs $XX.X operating expenses all As of million. Company the to to our This first support revenue requirements sufficient for primarily quarter partnership $X.X $XX.X fund I revenue includes The were of our our and will into quarter

Operator

you, first with Thank Our Gould question of [Operator Sir. Instructions] will from Carter come UBS. line

Your line is now open.

Carter Gould

tighten timeline from of about Two did the of that Good FSHD me, imply part for those the Thank just of any any changes the afternoon, sort period we data the February might a talk think FDA, the design which for at I the and believe around you guess taking that you, which the population first to guys. up back guess the added can two strength then also of my I quarter it's close also you. six assumptions in maybe part AAN rationale change I Thanks at you that guess to why And in in during the change period, the to make materialize. looking follow-up I heterogeneous change to the and a I two steps study when fairly strength about questions. guess month move FSHD in three for months treatment study? your got

Habib Dable

be I'll led as this that study as your going to this over again be to the endpoint. you Carter, the group kind we've we're are as going I to we Matt to going where six recruiting And maybe give for at biceps study of patients, us to considering a group that. Carter some the question. Hey, it's why so you to we've allude is you of it of would reasons and XX and we to in XX think and Habib, details XX mentioned two Yeah, the as be part started which well specifics mentioned the placebo-controlled be TI in initiated patients in to a looking some pass month thanks

Matthew Sherman

the as just a the of hi, to us is programs talks that about months six to us from endpoint initially, we models six design so give ability the extend of and more now trial. gives Yeah. had some months continuous as the chronic we government opportunity completed well the under the we part trial endpoint pretty, that allows more to months the the we Is clear? to Carter. to of why if not and for development to do first that us of original PAH the an initial the animal will two, in so patients it Habib that and Thanks, as endpoint three part redesign early at confidence during XX program design look compared studies we Yes terms dosing a data do

Carter Gould

population the moving one to of Yeah. And from heterogeneity and just far then as as the part two? part

Matthew Sherman

Yeah, also and for in weak right. be do be - for very we similar. great too design Really it'll it they of the normally be able we really strength too and so so require to study like think would we be strong populations year are heterogeneity thing patients cannot cannot they have for eligible the just be

confirmation and than so more In never disease look for addition, grab really we who wide to or genetic the homogeneous relative rather have a of patient disease population just at also patients. first be

Carter Gould

you. right. Thank All

Operator

come Thank Porges our will question with from you. And Geoffrey line Leerink. next of

line now open. Your is

Geoffrey Porges

about trial taking mentioned fundamentalists in questions you've in I of changed quick C CBB well might the the Phase - prior you have trial Pro different call. the from response. for the treatment, a X, apologies question as which we've much very but or the a that you indulgence the as if expectations duration slightly and level had treatment, late earlier few the things population, few and quite talking for this the Thank the

Thanks. So various or increase expect would response all neutral? basically changes be transfusion you those of the likely to it would rate

Habib Dable

for Habib. hey, question. Yeah, Geoff, Thanks your this is

consistently of we So there's possible done expect Phase terms we've in obviously terms as things, with around Phase factors of anchor of between much minimize we've to done and responses is in much number could X. pretty right. two it's what X to we what differences in try a On

previous entry where a XX, MEDALIST no the actually weeks study So at study saw perhaps which are at rebel with ago population and MEDALIST the previous and you over very And study of about four. was the a designed MDS-XXX a very MEDALIST one eight where minimum similar very years ASH, similar we criteria where to of an study three exception. the image one data the you when transfusion MDS-XXX another independent but back forward its transfusion presented at at way this transfusions to study that was criteria the if study go but sorry for eligibility they looking that two and the over similarly and had looking which for read look the you group was

that X.X%. in placebo the was Now, versus of that study you rate saw efficacy XX.X%

other the if saw looked marker, study Now, also we at of original say look our you Phase studies, an efficacy I would rate in X Phase XX%. the

a XX you that of a in week in study make weeks, at were the eight period looking XX rolling were the Now, terms great be looking actually study point period. TIs the you population, in MEDALIST at a MEDALIST week where in of

could we MEDALIST you in as arguably tend very So when see to wouldn't drift an into in why downwards, could say, to Phase go but X placebo of of controlled, of from you get larger higher blinded-studies, rate that well arguably XX% impact just more that and expect example one study general you development efficacy X And a the Phase think from fix in window you Phase when your to suggest Phase you when of move drug X. X might, you changes. from because to

where would add XX% indeed So much in I a but efficacy a the want the we what that? out know falling a of and That's don't if and be are changes to some successful win if from the to as things win you we example our see came between Matt one the if anything in as trials a consider perspective, we of that would somewhere win. would very see we XX.X%

Matthew Sherman

Geoffrey. Hi,

of our experience and X and the X variation well outside So study, a two of yeah study X population very your robust from data some from similar confirmatory studying one confident the very trial Phase X we're leading these have patient you're sides X experience while great deal Phase Phase X endpoint Phase Phase struggles Phase in as trial similar of go if clearly was of a design in back randomized, new site that decrease through Phase your transition regulatory we from the the and study. X designed to of trying the [indiscernible] pennants taking many the which for lot studies no curse chance to as road of was really that to with Phase from placebo-controlled X were lot well

Geoffrey Porges

on that lower you the Phase be So Matt probably a and beta fact disease that population could excluding to expect intelligence I level little the just a the got follow-up than in would earlier in C list course lenalidomide? the Pro of given X you've you're study

Matthew Sherman

EFAs if we consider negative patients overall population. received - those yeah it's I prior prior - And face prior statement range say baseline have positive the X then wider patient of levels. population earlier level it's subset earlier little therapy? of them a from of or in for both those disease as the the more just range head who know EPO don't the of year In that to they're know with could Phase that as you a

Geoffrey Porges

and Yeah, results. your thanks, guys. look very That's forward helpful to

Habib Dable

Great. Thanks, Geoff.

Operator

Karnauskas come And of you. line our Citi. from Thank next with will question Robyn

line is Your open. now

Kripa Krishnamoorthy

And an guys. on that just could be or Robyn. wondering you've the issue is follow-up X seen larger Hey, you MBS either about the trials. any Kripa the in for studies I the in X a FSHD? was This Phase Thal safety think question the Phase if concern way I in had

Matthew Sherman

much drugs it feel converses Matt, some in been Phase as about of very It's stable Hi the quite last several just both and December each Kripa, the good events able MDS including and indications. so of that just of safety as that what both year number X know and look and remain best describe profile to so thalassemia we've described non-serious the patients thalassemia different at from cross you that? the this is stable now so of deviates the state month patients excess actually Phase for or the experience with the the me that you last adverse MDS a on XXXX so. events, series the if announce, updates highlight year we X either the over in of series we been sort number patients population very those two a month database so patients number nothing on in the of were the to-date adverse that been of total That's for safety a populations we've and

Kripa Krishnamoorthy

Okay, just be this great. trial is doing there trial? regulatory can that have And approval is you clarity of currently a any Phase pivotal to approval the follow-up to for how do should you. there, thinking a accelerated provide you're do to on FSHDs process expect quick Thank any about you or likelihood X FSHD, we

Todd James

Todd. it's Kripa, Hey,

our comes out always the agencies move would have So we for X agencies. a of different to X and but without with a meeting if the amazingly have Phase X a can endpoints case ahead positive, two a and plan X chance is there's discuss study, plan with completion on a were regulatory have to interaction relevant with forward basic allow be registrational successful larger that part be the Phase that the with to Phase the the that would go post expedited to FDA the we is base approval Phase

Kripa Krishnamoorthy

Great, thank you so much.

Todd James

Thanks, Kripa.

Operator

Joseph question next our come with from Morgan. And JP you. Eric will Thank

now Your line open. is

Eric Joseph

Hey, guys. my for taking Thanks questions.

second the if be and you Just one Phase trial move to you those one of forward of one some we it compare two number to medical there part different half you're a to look get is and year the a guidance congress X, should was data thinking little what about doing that at cohorts, any can Thanks focused with the relative we think here of on there the when provide here quick to and this how of a part one you're can FSHD? two part a sort FSHD with on what that particularly part on reason

Habib Dable

rate this we're at the half of have far part to results as as decide as wait congress what at cohort the into a for part neuropathy know being each FSHD as so point. be to year, does the have so see results point it's is of second different a just you presentation Yeah, dose the this a dose versus a to could expect have medical wait efficacy see two, at if to the a in myopathy, at optimal to there this potential we moving or to

Eric Joseph

thanks. it, Got

Operator

Thank our question from next of you. Nomura. come line Marai And will Christopher with

line Your is open. now

Christopher Marai

the of quick us of should we taking a how with your for for first about preparations organizational through walk the with I my question, Thanks wondering Habib and the there potential ability marketing the I'm could and you have just respect status your of you. just maybe one for of guys. think just maybe Thank some wholly-owned? is pipeline luspatercept if greater rest launch to clinical for

Habib Dable

Thanks would I preparations establishment the for the so. hey, launch for of question the is been say Chris. thing that now ongoing joint for guess have first had your and committee. we Yeah, the I commercial a with while

of have specifics with terms of of partners and continue in the regarding the closely been prepare as that a like those luspatercept patients number other our working that to we activities have commercial every involved project some we of ongoing we part, been and to there's Just terms try we're the have groups been Celgene part as landscape payer patterns and at going been with the bring intimately the into payers work on the to ongoing there an about we focusing market engagement enter orphan of mindset space our luspatercept research that diseases with that with amount on drug. it the of understanding with tremendous and patient with also the advocacy that's with the more in is learn very

we understanding also necessary are like going We spending exactly time to footprint lot the launch. when a in of is look what

one what without take that look and has co-promote is that Celgene it get opportunity. is the necessary footprint Sujay to perhaps now opportunity What partners other of on to terms there terms do looking with to on bringing sure Acceleron the going of to leader recruiting intend for you is the very a Kango you they preparation with recent interactions of the maximize opportunity and your and the we committees. maximize can I then working America to to the right it in it goal a like is color one in based pass what to top That closely of on at the very, and commercial know, parts things over one drug important in make As we're things we some North versus we're that of number that board was and commercial two with our said, you few of if

Sujay Kango

thanks Thank the Sure. you, and Habib for questions.

is as towards Habib like both simultaneously. at we're anticipating least are joint and preparation, foremost launching we have at that the committees First working to goal indications point, said our time this

preparations the of access traders that going to priority. as a of across sort well launch groups seamless a we thinking number our as cohort the ensure one that sort and have ensuring that And patient the maximal how rapid it do uptake So the approval every about sort traders it. to both product really MDS do of both around are quick got launch that around of get aspect we and to operational once angle joint ensuring in a looking a the a relation is of regulatory we've at ensure this teams second beta-thalassemia commercial we in

marketplace. are those well patient are for journey the undertaking the around of currently are the this support the the optimal relation in patient community that leverage plans building all to what points are into elements So those as we delivery the needs right as physician programs relation and understanding patients efforts of community where the product special in to identifying patient to

Christopher Marai

the Okay. Sujay. color Thanks for

how company drug obviously rare informing more not wondering how look In versus from detail a that a addition disease disease clarify your do you. folks processes MDS - you to look rare our one is could and you at then betal-thal this has? the franchises beta-thal I was Thank perhaps that is you is specialist

Sujay Kango

That's the great questions.

apply the on how that's disease durability how of the the as the the understanding beta-thalassemia important sort an head. the some Some able right to all about relation of in the could of for rare nail between of and of we applicability factor definitely you our future from their question MDS patient to ensuring patient that and patient approach in care relation our in you to approach centric be models It's we and so learning hit maybe franchises, sort are segments we support and right them provide of or Ultra-red to beta-thalassemia to with needs patients. that then ability and resources that take absolutely we are ensuring the

was question as to right. the audience other Your to specialty relating

is as course MDS. we've mapped presence segment is population a be there MDS given well that segment out of oncology for they're between certainly is of different and But largely those on the our of we've and and groups will to two beta-thalassemia, treating, be identified between a MDS treaters we'll are audiences in that's and overlap hematology the So that has physician tremendous sort target MDS sort physician predominantly common tweeters covering those be sort segment Celgene small some and insight the and their which of otherwise, the going fields. intersection but that beta-thalassemia both

and it a national we both build to on footprint relay to have lot have expertise a our a So audiences. existing across on so speak

Christopher Marai

you. thank Great,

Sujay Kango

Thanks, Marai.

Operator

MacKay Instructions] RBC And come Kennen question next Thank from with Capital will Markets. our [Operator you.

Your line now open.

Unidentified Analyst

you question. make wondering Flanax part second half then and progress looking I [ph] Hi, would in on look afternoon. for question of as comfortable good what follow-up Phases have of potential on question toward advancing of the on readout you Thank Congrats is because if we the that on FSHD Kennen. registrational improvement, color sort start, A the taking thanks and that for well? This you. I was terms the are function forward of the

Habib Dable

the you FSHD, clarified, second XXXX is we I make Hey, the mention I just to sure right? Yeah. wanted think be that readout second to Flanax. half This half Habib. clear, that of

Unidentified Analyst

Yeah. That's right.

Habib Dable

Okay.

need being of a meaningful mass one point get So part really safety, we of mass to to we're which see the the time, equally the as fact that study a the muscle doses is functional drug higher granted muscle that and is this see you be be thrilled that we've that benefit. it readout half the to will to into part in same in were designed dose continued able to of be testaments the two that respect Track which we with to mass at to the a again to muscles we at of a muscle study in to in second favorable tolerability were and was the look We the resigned XXXX able increase great able in see if disease part response FDA with study With safety, translate thrilled a the increase of this the to us authority had the obviously Fast two the help to were XX% study. unmet that initiated tolerability the trying address and to know ACE-XXX.

XX% that benefit functional for be to in a and minute show will in walk around cetera. et a that? benefit a look mass you in could if translate that part studies other looking if we meaningful of X% to back Matt, a that have we benefit patients the benefit looked That of or could somewhere we've so anything actually to function, be into study meter studies that some and in functional two at increase could strength X% a distant, XX think the the walk where with And some at in seen in more add of muscle to

Matthew Sherman

No, no.

look I part on we're of and proportional so study. two one, think test the those well each as certainly of tests be being the but actually different into evaluating for currently to as in those covers as part muscles as testing and part the well, [indiscernible] for we'll we'll will be body them tested, point the

Unidentified Analyst

program. PAH to I thanks, And color. for that question Great, the Matt could and if sotatercept have –for ask Habib on

about be to any light of of of we of the will treatment PBR evolution you're options triple-therapy terms kind bar terms think starting double-therapy what tattered it standard you sort the of As it on of say whether that would the is the paradigm tested stuff of or or there the in triple-therapy the of in care and be in trial top past? that looking specifications towards

Habib Dable

going question Quisel. I'm to to John over Yes, Flanax, pass the

John Quisel

for Yeah, how Thanks the you? hey, are question.

intend design patients to do us a of care really the therapy looking thing be not at of standard of the your the probably the back and we're with the is trial look would therapy terms truly approach patients the for be visit shifting end aggressive medicine the of the capture standard and of top it what's top in the of with looking we're so trial, I we to are both reach will these So for and therapy we of what evolving allowing treatment a and double considered the increase of XX% the triple as that increasingly we each dosing point their story in quite question to allowing later on case care think so one we're their into and meaningful on stable background.

Unidentified Analyst

that XX% the be clear over in standard? improvement PBR to Just

John Quisel

apologies, right yeah improvement, of course. Yes,

Unidentified Analyst

color. the Thanks great. for Okay,

John Quisel

Great, thank you, Flanax.

Operator

time. Thanks you. I'm at further showing this no And questions

for So now, Habib comments I'd closing over some and Chief Executive back to remarks. like Mr. Dable, hand the conference Officer

Habib Dable

Thank at that Relations, in interest feel free and if seeing everybody look evening. enjoy and the James your any for rest President continued our of Todd please our thanks through us the further you those reach to company time much. Investor will of so of Vice and forward very EHA there's be questions to Great, and you for your

Operator

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

Acceleron Pharma (XLRN) 14 May 182018 Q1 Earnings call transcript