Acceleron Pharma (XLRN) 27 Feb 192018 Q4 Earnings call transcript

Company Profile

Participants

Todd James	VP, IR & Corporate Communications
Habib Dable	CEO, President & Director
Robert Zeldin	Chief Medical Officer
Kevin McLaughlin	CFO, Senior VP & Treasurer
Sujay Kango	SVP & Chief Commercial Officer
John Quisel	Chief Business Officer
Carter Gould	UBS Investment Bank
Yaron Werber	Cowen
Greg Harrison	Barclays
Eric Joseph	JPMorgan Chase & Co.
Gerard Smith	SVB Leerink
Jeffrey Hung	Morgan Stanley
Kennen MacKay	RBC Capital Markets
Holly Barra	Goldman Sachs Group

Call transcript

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Operator

Good afternoon, ladies and gentlemen, and welcome to the Acceleron Fourth Quarter and Full Year 2018 Earnings Conference Call. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to hand the call over to Mr. Todd James, Vice President, Investor Relations and Corporate Communications at Acceleron. Please go ahead.

Todd James

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

the Officer; for Habib are Chief Sujay Officer; John Robert Quisel, today Executive Kevin McLaughlin, Kango, Dable, Medical and Commercial our our Chief call Officer; Chief Officer. our our Officer; me Joining our Zeldin, Chief Chief Financial Business

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

Habib Dable

Thank years most pipeline. focused family after we entire Acceleron and of XXXX year disease founding, make areas. we've the three on XX joining to our important pipelines in one Pulmonary. and advanced for have was extremely super an and beta us of everyone industry, you, continued thank TGF- good I of the today. am across our proud progress Todd, and based you the Hematology, afternoon, Neuromuscular

and our milestones product Europe. States includes key approval programs, XXXX for and both commercial the XXXX several in the candidate and lead of ahead of all potential Looking including luspatercept launch United our

disease. in each from Neuromuscular X We and also important Phase of our expect ongoing trials pulmonary results

have and and trials diseases Celgene. luspatercept along collaboration and weakness, partner X FSHD multiple focal in MDS myelofibrosis. associated with or Neuromuscular, with myelodysplastic of in CMT. side Turning patients two beta-thalassemia ACE-XXXin to have Phase with We pipeline associated muscle Hematology anemia ongoing In we our syndromes, trials our currently two

our PULSAR Sotatercept both active pulmonary we'll in be trials patients are and and Lastly SPECTRA in PAH. evaluating with

front the The Congress year and announce and Hematology our middle attended BELIEVE and the very did Acceleron planning showing trials, BELIEVE thalassemia executing with luspatercept Focusing first key teams XX,XXX the at MDS to MEDALIST last at luspatercept, the results for in top-line phenomenal reviewed at from of set than risk lower a This stage discussed were beta- both from people. we strong excited and center. in more on job respectively. of results meetings were pivotal of List a MEDALIST ASH and over Phase ASH and key Center with at along XXXX The Capellini] X with [Anika two thousands at from trials hematology. presentations The Celgene ancillary the ASH, Among honors by Best along highlights and this a were attendee meeting the BELIEVE meeting to from Alan with packed center the other overflow. room presented from biggest a by attendees. multiple Milan full press conference only opening presented room them the first Moffitt among of of by trial presentations rooms Dr. trial, main MEDALIST clinical plenary trials breakthroughs And the of both scientific six University Cancer some for conference trials. distinguishing of as in Dr. to selected positive XX,XXX

Celgene the starting of results regulatory EU half -thalassemia, with in submit track we both and on VLA in to marketing in US and XXXX, important remain Finally, MDS of the the submission applications and the in to with April. the first FDA these beta expected

annual in overall non reached expand beta- additional its trial first-line trials, and lower X thalassemia includes trials we in trial which the Phase underway. the and XX myelofibrosis plan in options in agents. new continued this Phase current beta blood MDS of we of of unapproved have billion MDS, in Celgene X -transfusion MDS, significant COMMANDS medical to enrollment larger the luspatercept's need, need the This trial and recently Because patients. unmet limited already to are the anemia addition MEDALIST indications three with In luspatercept studied development therapies, BELIEVE to $X Phase with dependent patient with X associated population target for beta-thalassemia dire peak transfusions risk the alone. BEYOND myelofibrosis, the and lower to Patients sales risk of estimate red and global exceed cell and treatment -thalassemia with

peak low of of to Celgene XXX% opportunity translate potential the for royalty much anemia. peak these as XX% mid myelofibrosis estimates global cost, for estimate Acceleron. as $X also the tiered associated another covering approximately the in have to program potential We sales royalty and significant revenue to sales our billion With rate

all Chief unmet would execution an of at highly on continued in you As with opportunity expect with our disease expansion. upcoming the Medical will Celgene along and activities launch and over the pulmonary clinical now to Officer, Robert remain programs. Zeldin neuromuscular regulatory call need an our readiness focused I this and Acceleron trial to and magnitude, teams hand review

Robert Zeldin

I'd Habib. disease. to in with Thanks like neuromuscular begin ACE-XXX

safety charcot-marie-tooth part primary in profile on moved muscle At of Meeting protein and results, over part or ACE-XXX, experienced to XX%, by typically muscle the safety a expression a Based is positive mean We results continue a favorable increases along reduction FSHD assess to change in agent followed two. or the which for XX-week selection patients muscle motor a neuropathy from locally is baseline FSHD FSHD open-label CMT timed FSHD. efficacy by of and to endpoint tolerability the the volume over period. magnetic enable in safety greater Annual mass two CMT while such percent treatment FSHD trials and an Muscle with ACE-XXX The six-month muscle tissue, presented that we dormant treatment in atrophy. World disease and an muscle and limb via positive from advance nerve volume resonance dose fraction walking function into period. which six-month including fat leads in evaluated tests endpoints CMT, dystrophy we as Phase changes the period both tests acting designed of the toxic a placebo the to in part facioscapulohumeral versus disease secondary trials tests ACE-XXX total of muscle as absolute of Society in with total as with October, is with treated caused CMT. imaging our a well part than myopathy in is one X and in muscular upper these the Patients with trials, open-label to

to the the be announce the disease will the the reported full track patient also recruitment second half ongoing. disease of FSHD trial am top-line program. of to I year. We evaluating in enrolment trials. Patient like recently results XX in both completed With is and our CMT in that specific patients I'd to we on outcomes trial remain pulmonary that to present turn happy

therapies PAH of PAH, hemodynamics median improve reverse the of only blood developing new we X over in lungs. American pulmonary Deep XX characterized vascular our ability post Women's drug Pulmonary very high we Brigham PAH. Heart approvals for As preclinical November, Sotatercept's and last Also right years; in XX the be for Association collaborators these There function Sotatercept urgent at model research disorder the patients despite we years our and to survival Annual the Hospital hypertension arteries the pressure in Scientific to PAH remodeling at is is diagnosis. still need an or pulmonary an described and to November, Sessions the excited hypertension in Dive X which are in presented by rare progressive a on and demonstrated event in at ventricular Sotatercept structure so Arterial or animal and

is multiple Our patients Sotatercept's Phase enroll with with primary resistance PAH. This months. one placebo-controlled a to in functional period endpoints. World double-blind Organization will PULSAR randomized The is vascular The PAH in trial treatment evaluate pulmonary XX is endpoint activity change that secondary patients is group ongoing. six XXX X class primary trial Health with

recently addition more cardiac pet our study the in initiated to will SPECTRA. With Kevin endpoints This cardiopulmonary I the of at endpoints trial. financials. the invasive called traditional also in McLaughlin, assessed PAH trial CFO We and PULSAR trial exercise will call turn review innovative IC that or being an over MRI to to exploratory look tests the

Kevin McLaughlin

Thanks Robert.

were of Our XXXX million cash, cash December XXXX. equivalents and investments $XXX.X as of $XXX.X million to December XXst, XXst, compared as

including remarks. is a the expenses shareholders. luspatercept offering incurred will was is receive net current with costs recently follow-on $XX successfully royalty expenses plan the ended stock Total underwriters to common and the from that This a year Based requirements posted revenue full $XXX.X Acceleron of time million. all over million now our option million for our Additionally, by Habib XXXX of the operating the expenses existing the until sales. from our to for December over we million. were loss sufficient year projections, net new will exercise for $XXX.X from includes operating R&D The the the back equivalents of of significant for partnership the from related XXst, Celgene and of and together approximately allotment believe support Collaboration proceeds of $XX.X I be execute to for able G&A luspatercept. final the institutional million. of offering on cash, to fund were primarily $XXX.X we revenue company as year investments net company proceeds cash expect to company's The and turn and expenses the projected presentation such we to revenue million. $XXX.X and in

Habib Dable

expected year you, priorities remainder Kevin. submit Hematology, with applications marketing we for Celgene the Thank plan to briefly I'll lower-risk of Beginning -thalassemia US beta and the our And and EU both beyond. that to BLA of FDA XXXX, MDS with starting regulatory authorities the and summarize in luspatercept this Great. to and the of with and half in submission the April. the first health in

addition, results submit MEDALIST and X plan publication the Phase In to for XXXX. in BELIEVE we

in myelofibrosis, X reporting and results to top-line Phase X expect BEYOND trial for to of COMMANDS the X in For in phase the enroll continues preliminary initiated The results half trial provide anticipate recently XXXX second patients. phase XXXX. the top-line trials we

expect our also luspatercept clinical initial We for trial in discuss plan XXXX. expansion to

present by to Phase the CMT. from our for preliminary year neuromuscular trials second the plan Looking for we FSHD, part end programs, in of X XXXX of two with half results ACE-XXX XXXX at and

for for time. over in We has the we the and to believe results it commercial significant PULSAR lastly provide preliminary healthy through preliminary expect trial we volunteer to to and half In And of and created for PAH, XXXX. expect phase further indication continue results in first trials ASC-XXXX expansion year. this X [Technical provide to its closing, will date so SPECTRA luspatercept need the Difficulty] do the launch

also value proof-of-concept are We XX Phase next ACE-XXX important and data to over disease neuromuscular approaching with pulmonary both in X Sotatercept the inflection XX and months. to produce expected points

We focusing on advancing while have transformative the in year shareholders. confident to our busy and long- our term will for ahead and call remain these questions. both therapies now Operator? business ability potentially to creation I patients, open continue for a value

Operator

[Operator UBS. of Instructions] Our first question comes from Gould Carter

open. now is line Your

Carter Gould

that moving Great. I to how Good of that forward taking enrollment strength, of then thinking of was expect on enrolled, function perform hurdle question. one? and even what's benefit on detail into more any wanted in but potentially Thanks Part to first the color or a compares separation that about terms ACE-XXX, you're afternoon, guys. clinically the Thank need a the how in a study. you. the to kind kind the terms them meaningful clinical you clear on I of on phase arm for of color show And essentially little just on placebo since get guess studies B and to the demographics X Any there. maybe how Part

Habib Dable

Hey, off add kick Thanks just color. for This Habib. to of to Carter. question. any is and over kind Maybe additional Robert I'll then it your it hand

top-line the where we the respect plan of --in ACE-XXX studies as with to well charcot-marie-teeth both for disclose as in to So half X Part results of plan right FSHD charcot-marie-tooth we which second in this you're year. to

to. of study alluded part you you important knows The as about is this all function as

up safe tolerable a volume have been date growth in to generally muscle both able terms in total We double-digit demonstrate of and in to way. indications

a functional is what muscle and able translate Now do growth to to to able want to replicate to into we be that that be benefit.

Limb Now test PUL et biceps looking called in for et we're When by the when time muscles, Upper FSHD the Performance as pegs at to charcot-marie-tooth as both timing the and TA the engage distance cans them. like where run, at well measurement we're a biceps as where looking at the or muscle well we're in measuring, six-minute you're end walk you're using meter in FSHD, asked exercises both dosing functional stair looking patients the improvements walk for are looking at as points TA climb, as putting cetera. stacking cetera such holes, various and

assessments be to again life University at totality. We're in but improvement. end informed will power as know look a of working demonstrate powering at be like looking part data of anything we're day, studies muscle top at three So to double-digit looking we if hope what looking the spectrum like totality there's total we're to of Carter, the of end increases volume we very the again able again at of of studies on where able And collaborators I functional a a terms the through that to be we're well able of these be phase terms in said, the validated PRO Rochester. add? including all increases. two And the of looking to in that I the functional at Robert, be to endpoints the you'd function, we show quality with don't for a studies, to would go/no-go. of of us we across a And of in data

Robert Zeldin

comprehensive. Too

Todd James

that off they targeted add the to for Hey, baseline moderate scores trial just Todd. the patients mild would it's the based demographics. Carter, see I muscle about We patients. of

So in and the gone involved an improvement provide with ACE-XXX far a be line couldn't therapy the in an wouldn't too we with the patients that's like they that so trial. were patient's

Operator

Danielle from comes question of Piper next Jaffray. Our Brill

Your line is now open.

Unidentified Analyst

just quick to This everyone. [Indiscernible] wanted Hey, a questions couple that Danielle Brill. I on. is on had for hit I

give the preliminary up we trial, coming second us the to half? on data the in to color looking that's you going would some First was myelofibrosis be be able should what for in out be

Habib Dable

Yes. I know thanks for question. your

they just would study in at X our with with we these the looking XX of patients And from of So then without enlarged --XX we're out half year who be running at phase that and of these are that don't spleen. luspatercept a dependent dependent as luspatercept because everyone we remind non XX an looking guided XX patients also that patients, to have the have XX is on patients. we treated recruited of ruxolitinib. patients Again, presenting that And combination patients. being asset we looked the as at looking ruxolitinib at fact we well this we're transfusion that myelofibrosis patients, transfusion second the are this way combination where in was data again

the are patients cases bone fibrotic it's end of unmet And day, that important to fact so anemia I the the the to due think --in they with at need these the highlight many some just suffering of because in from marrow.

the X announce to as you in drug-induced disease recently is led suffering patient, we're suffer inhibition. remember our is as with the combination because also marrow, luspatercept Celgene from be study and part at anemia transient, use JAK please ruxolitinib Phase inhibitor as because but we'll is think of of general, of anemia many of well which something together myelofibrosis therefore us they're is the in mechanism cases fibrotic This collaborators our as of looking JAK but also bone where well where the Fedratinib study. the when with looking patients X/ And it JAK it also at there these mono an where action on of impact inhibition so about induce in --

Todd James

it's Hey, Todd.

X the the as transfusions increase X X.X endpoints to for or XX we for week the XX that hemoglobin the depending whether primary time, in patients the four-week period no transfusion consecutive have cohort looking within that on Phase there week consecutive only we're the over XX far primary unit period. As over patient's There which of for burden six-month And it's anemia a independence are or weeks greater of looking gram treatment transfusion cohort. within a period first for treatment the the primary weeks a XX X period. or

So results year. the you endpoints see expect the when do of second that's main that we to present could half the in

Habib Dable

Great, thanks, Todd.

Unidentified Analyst

on you it was for so coming expand the Could you it, That how doing the right much. and with was you're about? little is that thank Got you readiness getting I a helpful. guys really activities had guess And question now bit? I luspatercept. What are you second mentioned ready launch

Habib Dable

with with be under in that a to your So we and standard could recently made we April BLA now April we a from mode. later thanks year announcement for could launch review anticipate Obviously, process expected be in the filed that question.

of we to well doing that, in were fact also that our review be Celgene said, with launch if together and earlier collaboration that as a Now a we we with activities are number that as priority would we partner able surprised indeed were preparation the prepared. for

So terms going in I and it's from on. pricing a manufacturing that's tell our positioning Whether of work in tremendous you amount terms in a activities of of can in strategy, that research, of there's number readiness. supply. terms launch of preparations our

we're a of the respect maybe terms Sujay, of specifics commercial in little you of bit perhaps, launch in of Now to things terms partners. with to now with on right you color add working want that some our

Sujay Kango

Habib. Thanks, Sure.

necessary commercialization the that And approval. mentioned, get on clearly and launch for that already right as together the and have what things are of place. when as be Habib forefront understanding putting of we needs teams couple that joint patient we we in So do would part as programs

a around patients planning perspective necessary, all that what we the engagement engagement early instance peer preparing are appropriate, to think within is that the materials is trying that to are the where from US. in access understanding component ensure that peer So the have

the appropriately to time at members the right team we right So are able deploy point.

a we'll to So go planning at launch patient's all with phase to for mind. the that day this pre-launch are is on in our one have prepare doing ensuring what forefront ready of work be the needs we and

we working on. what that's So are

Operator

Cowen Yaron question next and of from comes Werber Our Company.

now is line open. Your

Yaron Werber

endpoint. consecutive and a best mentioned -- Thank week one specific you Is clarify consecutive much. questions is be a needs point? XX sort just the of that have the that so any data that within consecutive first to record of you Todd endpoint. you couple the XX-week to or Great. I maybe XX-week it XX-week within and the time it

Todd James

And Yes. it's the first.

if So that within week to instances considered X responder XX X those XX XX all XX it's within XX or XX week that week to to be would period, or a period.

Yaron Werber

the And done same that way obviously just the -- yes how study single with in rate? XX% response XX% remind you to defined? endpoint that us that that Sotatercept was it center defined Was was can

Todd James

the right whether we're IST looking that's the we've done that's how MDS was endpoints it treatment and Yes, or over for eight-week, period. there a Sotatercept the consecutive trials

Yaron Werber

Okay, you're both of to both going you're whether Priority reviewing - I you're application? Can division if is hearing the correct remind Is FDA going asking times filings for comfortable to indications? like just the sharing division Review. same I'm reviewing the for way this know great. actually like which it be it And don't you sounds then us, you're maybe so

Habib Dable

Yes.

that? to So answer would like Robert you

Robert Zeldin

What?

Habib Dable

Division of Hematology.

Robert Zeldin

applications. yes, it's So will our that hematology, division review both expectation is

Yaron Werber

single will be same review review there them at of the priority a they'll and so Okay, time.

Habib Dable

Yes.

the that's -- case single. be necessarily not it that would it's So

for assumptions but process. case just looking you standard clear, review is would practice Yaron, remind base standard that, I'm obviously, a So that to again be just so at asking and we our

Operator

of Our next Robyn Citi. comes question Karnauskas from

line open. now Your is

Unidentified Analyst

questions. for Thanks on Robyn. taking the Nicole is this for Hi,

a be --do potential of the Just communications going process your --there you the regulatory between expect any and And application? they're would you be hurdles back you authorities sense between that to that the process acceptance get concerns to the the EU? the similar from US same could about regulatory or regulatory or did

Habib Dable

Robert would like -- to you

Robert Zeldin

was Yes. that pre very meeting positive I open a think I discussion. that think I mean the BLA at

first receiving April. authorization subsequently think we now application. the with concern I the folks I not could as FDA in -submit intend any identify areas the the specific was And the application with marketing about And agency half. excited in aware the to of clearly are file

Unidentified Analyst

any Okay, Where then with beta given cell competitive been for between you great. luspatercept take being there and multiple do up fall? evolving And see dependencification? transfusion investigated. the transfusion in different non follow And you greater a be a difference quick the just modalities dependent do expect and potential landscape to

Habib Dable

Yes.

part two transfusion in your study -thalassemia, the let first. question just me with everyone was so respect it to patients. answer So of The second to studied dependent BELIEVE beta remind

approval initial be that. for that the exactly we looking is So will

Now through there probably also to that equivalent I think study. exploring though that dependent we're transfusion it's population the a note BEYOND important population the to is is significant

because and And stage high so not just so in being are cited that need but a by population are XXX recruiting there again to kind it's patients cases of is very and obviously is Capellini is fact with transfused very that actually this many symptomatic. we're Dr. this there unmet population what they many, the repeat a on of a

much to very looking that we're of results study. the So forward

Now - thalassemia competition --what in potential would be. of beta for in luspatercept of question of part the and and first your how terms the terms

beta- unmet respect that the other management a to want to other which With that to be that proceed beta- healthy restore of potentially has will two approved, anemia. need luspatercept things thalassemia red cell the if we that demonstrate are myelofibrosis thalassemia, be risk diseases to. catering diseases. date are we and a to confidence to of feel MDS there tremendous we of as as we formations lifecycle One to is such with will chronic has activities well been us proud blood distinct given as very extremely in Lower ability able

if that option community many, that safe therapy thrilled lot Now, to is is and obviously, Do to as an to is another therapy. have with patient this feel be on for right unmet such a of I proven now find who feel a a many a to population for I and that it opportunity the or cater feel option to efficacious, know be needs> for for tremendous gene not and space very there significant And going be patients will may there's feel No, that if the I gene don't. a I indeed that qualified attention option. going luspatercept are space I patients, I'm therapies

Operator

next question Geoff from Our comes Barclays. of Meacham

is Your line now open.

Greg Harrison

this Harrison Geoff. Thanks taking question. the Greg for Hi, on for

that potential the luspatercept factors So with the up if as do launch horizon, is and and on populations? now same you could what development so, penetration possibly MEDALIST think the BELIEVE the indications outcome? end expanded enable And could that garnering

Habib Dable

I make your sure I question. to understood So just want

the MEDALIST in will correct? indications asking You're in to or not robust need, we as terms of the is for be follow-on they the unmet of terms equally in feel whether BELIEVE that opportunity, -- the and beyond that

Greg Harrison

exactly. Yes,

Habib Dable

Okay, great.

be BELIEVE, we on So same a MEDALIST that to feel page. again very When sure population. look make going BELIEVE we're to and let's and at and that start with the catering we we're MEDALIST significant

that we information when have most Europe. So the at feel look example for and we we datasets the at got that we've robust that US look around, we and

patients serve. able an feel For to the be the between population, there's opportunity approximately that Europe that and to about US MEDALIST we we'll XX,XXX have

approximately it's population, patient we XX,XXX. that the feel With BELIEVE

expand Now opportunity admittedly, studies. by going to be subsequent to success driven in for the is our that

feel is patient risk believe treatment COMMAND year, indeed we line that XX,XXX for that right we successful, which double. successful, beta-cell So alone has a that's of is patient is MDS that first our an population study study if the study rate naive that in is incidence approximately the of Todd? population could lower BEYOND If indeed

Todd James

XX,XXX to XX,XXX Yes.

Habib Dable

about severe if across myelofibrosis moderate allows suffer XX,XXX from group and XX% -XX,XXX provide a them at to well. get and year. this XX,XXX to much US look to to the disease space as of the myelofibrosis, thing patient to us that Europe able in to approximately is luspatercept XX,XXX setting be at you it probably and into patient When is nice And look that you population with XX% anemia. patients between earlier

looking these XX,XXX not patients the as at anemia before, this suffering drug-induced patients So or are fibrotic due or ruxolitinib to chronic as said well as marrow, independent not now you're to again anemia. XX,XXX on bone another they're whether I to as and from

the opportunity a be role this as can an here luspatercept So for a profound erythroid play to pretty agent in group. maturation

groups we're population. a looking working that the to And lifecycle of have to are beyond at all underserved at looking indications. identified We're They Celgene. we and a potential forward to high very with talked strategy. an have very our very have expanding need unmet disease about at partners these already closely then that management beyond moving So continue obviously motivated the

prioritizing of you've and but chronic come to talk that to more that think exploring many a exploring, us as we're heard continues anemia just one I one we're analysis as bit about and it of that hopefully one induced is little induced many chemo areas finalize. anemia. And that's

Greg Harrison

you. Thank Great.

Habib Dable

and our plus of that study billion the in and Celgene $X and myelofibrosis opportunity if forward we now to we as said that have a where our more successful indication, that look always incremental approved the that way in by next move alone. given opportunity at drug And of we just $X $Xbillion you is bit on is indication terms a granularity an just risk little the with opportunity, partners at and kind that. of indications that we've it's traditionally the off recently opportunity indeed these in MDS this indication just we myelofibrosis, billion the commercial year represented lower if close beta-thalassemia believe in

Operator

Eric Our Joseph of next JPMorgan. question comes from

open. now is line Your

Eric Joseph

data myelofibrosis, path whether non transmuting sort the couple to you most luspatercept on and A versus combination you factors questions. about there's how And development? of in the where of the pursuing a populations? and Thanks later trials us. transfusion for a I the be guys. talk from Fedratinib taking the dependent Whether forward? thinking little individuals pursue to stage can first good studies registration --individualized need Hey, and way wholesale we are that the dependent maybe about would indication bit guess assuming safety should

Habib Dable

Yes.

answer question too So, Eric, that first is to think right I early pretty easy now. the in

last see what and of guided obviously wait the Let's on development phase think and then all and and And what signal in that Fedratinib, we'll feedback this we'll think at with the top path I luspatercept I by disclosed point agencies. that then are of the their myelofibrosis how can let's reads this on that of X. been phase see already also X a strong we'll then I With commitment based sends to the anchor. it's that data to terms forward in ½ of out, regulatory you terms respect month comes their out tell phase studies. with be have appropriate that you very disclosed to the share for important note interactions with Celgene The have as

Eric Joseph

that Got sizing you that it. based which detect ACE-XXX, you question a point and be earlier it benefit on made And on expects able shown? to improvement measure maybe just that functional a trial be and see specifically expectation a you'll -- the are clarification is static to benefit a

Robert Zeldin

Yes.

the to data. was we're So totality look that I think going at Habib's of point the

look growth is So That's to functional assessments know the in totality. number benefit clear. most of we a have we efficacy them going of primary overall we're and endpoint now at the

advance individual in confidence think give the would on say like program. pick to know don't above we'd a those see I'd of the children that one we you So general XX% that to to would other. But our improvement say measures I us the guiding we're

Eric Joseph

number part we'll it. got around it, just subjects out? read you how or frequently are And Got the see that of assessed the of sort can intervals X in comment

Habib Dable

Yes.

the the to haven't three patients as coming you again timelines, in gotten injections done every weeks injections. in for of just intramuscular but are so assessment So granularity know what these that we the their are OP. Their

Operator

Our Gerard Smith question comes SVB Leerink. from of

Your line is now open.

Gerard Smith

two what then taking This XXXX you in for will the couple based we you data Thank different to about Can making be try for on the thanks is exactly you. of the we'll the of function not inferences on or what on disclosure, terms on the expect And appropriate? how later question. that we you muscle talk get you is for to safety sort remind two us specificities ligand Hey, data, the cetera? say different the A year? should phase volume agents? could thinking about about and in could muscle et disclose And ACE-XXX of XXXX it type make Gerard inferences Geoff. also one how

Todd James

Hey, Gerard, it's Todd.

far or very as say release to would the there discloses hard results, have that at at press least plan communication go those not be for fall As exactly Congress but the results. a to the

just to regular in single across you of comment trial is a site injection which saw as we far two as are we're outlined. them. like could are doses of there we to and five, at think one, style about very safety muscle dose ascending part PD would six as example potential see, we imagine for reactions would the if could it's looking the trial. expect in as one, things far quad as I be increases absolutely that expect do a that. grade volume And you this if As in can different you If what Typically, serious anything categorize

you it to nature. ACE-XXX, mid far percent agents part it very most XXXX as that a would likely us, and the in there before to if we've the increases that therapeutic seen increases increases locally have agent As seen can inject to like doses, of you systemic difficult at I have [Nagarmab] can dose single at be right one be into come muscle results is high in the see digit Regeneron, of fairly from that whether results, whereas ACE-XXX anything distinct high molecule very XXXX of least plus think levels, XX high it read we target do the agent that, acting take to

And the other. think so much yes to read don't through from there's one

as far affinity. As binding the

I'd I into John Quisel those think details. let get

John Quisel

Yes.

probably form ACE-XXX administered are you and with their remember very ACE-XXX of negative that safely. locally they're as molecules to as apart with the local is about like as that best they stimulate can being by of able different reach approaches view important growth systemic being So both know thing many The Todd ligands two ACE-XXXX to XXXX ligand being regulators myostatin of plus agent. minus relevant how binding call what the from to we mentioned can't our muscle muscle concentrations to can you inhibiting the in improve get so a function plus, to and and the so are

spectrum though the So of kind two muscle of shown like where systemic they teens, the similar feedback we've lab the from a engaging increases agent in the the in agents ligands. are surprising XXXX would be effects even probably

XXXX receptors a Just the [fall on cell based off protein the are key speaking are scaffolds generally ACEXXX based of around ligand. track structure surface it's the while one in stand] binding other is involved built the ligand a to that briefly

modalities to ligand we different they're So we've spectra very different with different, very binding very on the different due muscle similar effects mass. expect and their proteins

Operator

from Hung Our next Stanley. question comes Jeffrey Morgan of

now open. line Your is

Jeffrey Hung

you payers taking that and pricing? any value-based for you've the about talk with anything or Thanks you conversations questions. surprised discussions Can had on

Habib Dable

there's comment, discussion, So do of you terms research we to pay ongoing? respect Sujay, that's can or anything with want we much that to in I think but payer don't can say about share

Sujay Kango

Sure.

you as conducting to right. is research, pricing we a there. And payer know very So value Celgene getting insights based approach we are over methodological are

getting can taking that juncture at want have right. those insight population account I possible And that's this are patient comment. we So that of for the ensure into we access sort broadest that we to the what

guidance We phase launch FDA us good in they're to And disclose to So all the we are close think do based price get as we can through position that feel related the I this proposition with We insight. to can in very be a to payer that the we'll made then that X strong. mind early engagement at also that juncture and on insight. able that well. value collecting

Jeffrey Hung

going and then on strategy and of you Great about in your thoughts pulmonary franchises. for neuromuscular alone? terms partnering maybe in and talk -- versus your the Can

Habib Dable

good as secondary both areas appropriate to are just end therapeutic we of fully committed a We on successfully gives It's us our XXXX. execute day to at able the were obviously the in which to question, firepower day think Yes. I the Jeff, we disclosed R&D during for the And good look in the based take Acceleron. point. these two be we'll the and of will of a most decision value end we to terms for on phase programs a that drive that make at and strategically at conclusion. And what drive studies data, would

is we maximize I think these necessity to rather do them. to positioning otherwise than programs in potentially to be of all these a run to most partnering partner important that be afford able the nuance programs not we the to value would because ourselves position are of to to

I think it's a very important nuance.

Operator

comes Our Capital Kennen from Markets. RBC MacKay next question of

Your line is now open.

Kennen MacKay

peak to Thanks liked $X questions. billion Couple sales the at quite reminiscent centerfield of of quick I billion question. bleachers $X for move taking in Ruth is that the Babe that's pointing it. guidance Habib

about clearly luspatercept Then or I'm these in immunosuppressive impact On --obviously MDS, potentially low in was out either the and usage likely maybe there's of how with but we clinic? transformation some hemoglobin major I therapies MEDALIST antineoplastic in there it g-csf patients, requirements guess leukemic into could on compound some and transfusion patients? think or high also in correct luspatercept Thank how thought of be also data in is a there were anything as usage the wrong looks difference own me like will these factor should or but leukemic. us impact this in of Could platelets. impacting MEDALIST any for but you. for of remind What supportive on you intensity that there's impacted how sort when no if and label these care there

Habib Dable

comment. that how you to not cut the I'm sure want bit. much all you little to think out if I'm get ask So of I'm going Robert into depth Zeldin but I question a sure that. And caught of that, all not Dr. to and first part

Robert Zeldin

Yes.

you repeat -- If I could think

Habib Dable

repeat first first part. part --just The the of you

Kennen MacKay

On an if difference was for looked been forest I in some there trial. any g-csf points, like wondering effect that requirements on yes, was had of the those presented MEDALIST. was -- in plots there in MEDALIST

Robert Zeldin

population having No. thrombocytosis to in tell of the on treated I data patient that basis, the but review individual study. I comprehensively the regarding an speak have the can't in very specifics you we MEDALIST patients concerns can no safety

Kennen MacKay

you reduce the Oh, your of need could was from the no. last thinking -- I some more that for --therapies

Habib Dable

the question sorry, Kenn, of did we and the terms each over hearing of by answered the from tripped other it. was I in we got that --I you first think comment part So, in think Robert

answered. believe don't The second part was of your question though I

that to So you Kennen. repeat want again,

lost we we Okay, okay, at him, all. catching weren't

operator. the question, to go next to have we'll Okay,

Operator

And Terrence your from Goldman Sachs. of next comes Flynn question

now Your line is open.

Holly Barra

for on guys. Terrence. Hi, Holly is This

of Just data? the you. you one the COMMANDS that is potentially we and see enrollment can when status us Thank from trial discuss could

Todd James

year. It's late Holly. the off kicked Todd. our Hey, in partner COMMAND in trial the of Sure. with Yes, third we Celgene quarter last

that's up as sites as running to up the imagine enrollment about initial you X trial the get to and in sites So first you on XX pretty getting but patients trial, can into months quickly. starts pick the of more all and obviously

we're here. So in early the still days

we clinical data, when trials. As could trials.gov to typically of you on are haven't Q&A End initial forecast some expect as far listed guided for but that today, they

Operator

this Habib our turn back call Dable the over question-and-answer closing conclude And to now does any remarks. I'll session. for

Habib Dable

interest Thank continued have for that there participation. your everybody if night. thanks no in No, questions, conferences you to investor And forward at Yes. everybody look with you company. your meeting future for is good or other the --

Operator

Ladies you and your gentlemen, in This for program. today's thank today's conference. participation conclude

a You all great may Everyone have disconnect. day.

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