Acceleron Pharma (XLRN) 5 Aug 192019 Q2 Earnings call transcript

Good afternoon, ladies and gentlemen, and welcome to the Acceleron Second Quarter 2019 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will be given at that time.

As a reminder, this conference call is being recorded.

I would now like to hand the call over to Mr. of at Director Vice Joyce, Investor Acceleron. Relations Ed go ahead. Please

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

our John and Chief Chief of and McLaughlin, our Kevin me the today call Officer; Todd Officer; and for Officer; Investor Business are Financial Commercial Executive Quisel, Officer; James Joining Sujay President Chief Kango, Habib Corporate Dable, our Vice our Chief our Communications. Relations

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

I the Officer. would call our now Executive Dable, Habib like Chief over to to turn

you, medicine. based thank superfamily most advanced joining discovered disease is under with we areas, and This one have today. most but not and spanning you of the history, industry's Good TGF-beta Ed. of EU for the we're pipelines approval approaching Acceleron first of XX-year us multiple luspatercept, U.S. review times filings regulatory the potential one afternoon exciting built for only in Thank Acceleron's everyone

clinical priority. disease. neuromuscular Patient we in work pulmonary maturation for thinking erythroid Our In for commercial this team alongside approval. top led and to partner Acceleron are remained hard global our access parallel, a two hard agent preparing is programs cooperation luspatercept potential Celgene at first-in-class

with Phase to next X proof-of-concept sotatercept with results line ongoing trials, expected one help patient three completed top in X ACE-XXX three nine with Our from establish months. enrollment and trials will the Phase and placebo-controlled each all has two

and XXXX programs clinical achieve positioned meaningful we this medium to progress see, objectives. in can for our is we near regulatory are As term all and well and

that U.S. huge the BLA myelodysplastic to and teams and a important announced step therapy Agency and we for anemia. June, and achievement in the This both luspatercept, associated beta-thalassemia and European Acceleron Medicine for syndrome delivering this is Celgene FDA to the in respectively luspatercept this patients. MAA represents on filings another Turning accepted novel

FDA data With the European April the action review for marketing the the for application filing Likewise, for been X, target indication set indication. authorization beta-thalassemia target with data we and with and anemia beta-thalassemia granted acceptance, adult or December for validated X, MDS XXXX pleased are Agency. luspatercept action XXXX has by patient the priority a for associated Medicine MDS

in by desperately was believe red to or we and need conditions eliminating in therapy the the this EU closely potential decision patients and second half transfusion option burden. potentially to of blood filing these Patients the that U.S. improvement decreasing of on With forward with a look bring luspatercept approval. toward with treatment significant working the we move a to viable agencies are XXXX, expected could

luspatercept. potentially clinical to MDS committed remain with we development lower expanding continued treatment beta-thalasemmia, with in could unbilling patients, clinical to treatment line from clinical risk our luspatercept benefit end, patient also associated Further myelofibrosis three development first that To that and anemia populations plan anemia. non-transfusion-dependent with trials, additional

programs our with beginning I disease. in now like neuromuscular to Acceleron move led ACE-XXX would to

of or completed Phase volume in trials CMT. two novel X evaluating specific demonstrated recently ongoing target acting enrollment ACE-XXX Part full Charcot-Marie-Tooth currently We X function of with the therapy patients potential We’re trial as in FSHD locally of increases with Part facioscapulohumeral muscles in X substantial and muscles. muscle FSHD dystrophy from Part in trials full improve and to trial. muscular Results the or target previously CMT in enrollment the a of of X announced disease

of evaluating with is evaluating randomized Part placebo. X CMT ACE-XXX anterior or or the summarize beginning to XX to FSHD, quickly tibialis to in X bicep one-to-one weakness, ACE-XXX XX receive the placebo. with to randomized patients To to either or Part weakness, trial mild moderate either moderate trial tibialis of anterior with to is patients mild receive

percent motor in six evaluate to is outcome functions. months this as post Specifically randomized designed fraction include as muscle outlined treatment and measures intramuscular These fat the volume well top percent changes change slide, the line in trial change similar in period. in the the

FSHD run. the cohort walk climb, of six-minute test, the In four tibalis stare the trial, these include walk anterior XX-meter

these as determined therapy in to health-related the We health improved an overall life demonstrates help we’re in medical outcomes for evaluating change neuromuscular of as the unmet and index potential addition outcome specific with by the well ACE-XXX trial functional also and quality CMT measures, the FSHD disease safety In reported that index and the as needs. if patient tolerability new to become both trials. patients believe disease has important outcomes, in

believe trial in past results half enrollment PAH. CMT sharing results XX the We the first over of in patients where from X that FSHD therapeutic program XXXX We results for quarter pulmonary of of underscores to our pulmonary rapid in forward PAH. program with anticipate Phase excitement we like both recently in I'd move pulmonary and urgency our top line trials. look sotatercept the enrollment lead arterial new and to XXXX. We to candidate completed the options for for hypertension PULSAR's months, now the or PULSAR the the second

signaling In vessel PAH for muscularization treatments by Sadly pulmonary sotatercept the to therapies improved potentially We though, patients a or is combination models sotatercept the and reduce right or of resistance approved are BMPRX reversed used five heart alone engage vasodilation capacity vascular to exercise the the progression slow Currently, pathway for in vascular vessels promotes and pulmonary believe indicators PVR. pulmonary median ability rebalancing disease. target of each homeostasis. seven has and to the These in only PAH, that improve to the pathways that the of three years. failure. restoring fundamental survival disease main only preclinical

Phase patients. blind therapies. sotatercept As outline care placebo double on to the a in slide, safety sotatercept of Sotatercept A standard designed PAH placebo, evaluate or in the PULSAR total of controlled study is randomized X trial and patients low-dose high a this dose with combination efficacy receive randomized, of XXX were

trial Following continue trial the six-minute XX chain top line month period. month be primary reporting eligible primary and the to from We period, is the change from the The PVR in participants six from first the anticipate secondary PULSAR walk key and in of will distance. XXXX. points baseline end end in is point of the extension the results trial is baseline treatment quarter

Additionally, is PAH with currently team exploratory enrolling our the SPECTRA study. into clinical patients

CFO And McLaughlin, financials. with that, over review to I'll our to turn the Kevin the call

Habib. everyone. afternoon Thanks, Good

such we current sufficient receive luspatercept requirements as cash, projections, cash will million. $XXX.X investments of we and investments XX, of gross to on cash acceptance XXXX were equivalents and be and receipt BLA $XXX.X our expect balance until believe equivalents cash, royalty sales. equivalents investments of a cash to XXXX earned Our the $XX This as significant time milestone current XX, luspatercept operating upon million million. and cash, This projected revenue that the plan payments fund and June December Based compares operating cash filings. of from includes MAA

June $XX the second the either Collaboration The luspatercept of $XX.X partnership $XX.X largely of This revenue $XX.X million, all due with to first by million. million the of support million, for milestone The the EMA. $XX R&D potential the expenses FDA loss Celgene from upon and luspatercept for the XXXX related in for is quarter Total and to the $XX.X million. includes quarter expenses $XX approval Company and Company's G&A or payments. second of is was expenses XX, next quarter includes is second a from Company net incurred revenue The posted were milestone million. million. and expenses collaboration related ended partnership costs the gross

presentation Habib final will now the I turn remarks. over back for

Kevin. Thanks

luspatercept and summarize hematology. beyond, So beginning priorities with to of remainder this for the year in briefly

Our number the application FDA is priority EMA. one with marketing reviews the ongoing and

of expansion with populations, additional are anemia. associated the in diseases other focused ongoing patient with the clinical potential development along on We luspatercept execution also the of trials in

in XXXX from followed first of expect from trial results Phase we line PAH, ACE-XXX, XXXX half months, first both X of quarter in line in CMT X SPECTRA expect results the trial next in trial from starting second trial top the X both and results the we For XXXX. in PULSAR the Phase finally, the top FSHD preliminary in And with quarter.

I call questions. will to open now Operator? the

Gould question [Operator Thank from comes first Instructions] UBS. you, the of sir. line of Carter Our

open. Your line now is

couple. Andrew is for Carter. taking question. our I a This guys. Thanks on Hi, had for

you in of just you on order is FSHD start that if mixed as but other both the the side? great. think follow-up way, given demonstrated about in Phase on the how de-risk had how profile efficacy efficacy Phase to X, if you one guess on I other first data So, I that movements study, potential there, demonstrated luspatercept, in the and you tibialis to across would cohort, the put critical be in biceps patients? more that X can that a will

Yes.

the be excellent important forward think for is able you Phase mentioned, are an is we to your most be in study at, year. looking This I question. as be thanks and there CMT move to which are as was us X, out It's quarter of So, that reading a number with in next Habib. question for will FSHD going as of the first study, the things both well the to

reliably our primary studies improvement driven obviously everybody, forward we're then muscle the both patients and the point remind to by looking really to that total the functional to treating. path ability is going Just volume, at end in of the increase is

alone data minimum ensure looking or able so, be will been its And to some at or achieve FSHD alone that we biceps, the that or thresholds. CMT whether we TAs to

about at And four-stare muscle, thresholds six-minute walk, have those are we talk of anterior we run, looking looking timed tibialis are improvement the at such XX-minute some functional previously in specific as that climb. walk

but looking looking in are also are from double-digit. ourselves we've the we we a again, as given that's at deviations And And placebo what some for really threshold. trends

and X. go you then forward of having alone So, data back terms and we're in if not or path FSHD with by to the conversations we get biceps today at totality looking after TA to will regulators, or Phase would with going that our you be we for after to alone decide or as alone, in the whether

In And And operating? it where on XX at I and in efforts you. -- can positioned Celgene is luspatercept, was co-promote believe as scheme where its looking do the team you here? seeing or your here? amplify on Thank to Acceleron Acceleron own you that the how Bristol XX, see U.S. layer

I’ll maybe Chief Commercial some elaborate on it of and question on pass Yes, to details. great Kango that’s to a the Officer, Sujay our,

Sure, right, us question, thanks force we co-promote articulated, for that before to and that as people And a first and team. we have allows articulated you have field there. about dedicated XX a is have the as foremost,

is the side solely is that focused approach we taking actually on So, surround Celgene to with sound. ensure we’re luspatercept and a there

operating we're opportunity, going to two ensure docile as we'll with to So the coordination, with centers that maximize as the top accounts and be there’s right. all well collaboration,

what So, an to for particular cohort to satisfy do and sort needs. and luspatercept drive cetera. amplify and proposition going value we're optimal is, to is limited not synergize What et a oppositions of customer coordinate we’re necessary

you. Thank

Werber comes Cowen. question of next from Our Yaron

Your line is open.

Great, for thanks question. my taking

first have one little ’XX in decision I now a second just or of EMA filed April. It of approval couple maybe early for was in but the mentioning housekeeping, half questions, I Habi maybe believe you’re the So, a luspatercept.

expecting QX? maybe XX-month second So, And is follow then it the are a then of I a goes EMA months and I'm about X just to clock, and have of understand, the to well. it thinking -- into up still as trying CHMP X sort you is half? sort

Yes.

decision. again, et of So That yes, into stops, you cetera. potential a all half. Yaron, you've the the as half us count for that expectation take second alluded would our were second for is clock when And to

And exactly does for it which you’re would but that, haven’t elaborated which right fall into, we quarter so beyond the clock that all account stop.

been any that EMA there request to has Okay we for recently about? need know

and is track No on everything expected. as going remains

X, X then it’s be patients XXX in And of sort or XX so PULSAR randomized for the XX, to and X patients, a full it’s Okay. overall, to study, so. arm going it's to a

has at really a you to I so, my statistically for question And guess different? do like think powered point? with, trend are this significant Or you be at is looking

show for for is reduction end respect powered approximately PVR. Yes point, so, a for it with end difference to the is point difference, and to PVR, again looking in XX% primary and primary we’re

one dose powered in the against them, both Okay. point end high the is it is that the of placebo? doses? Or And primary is really

Both.

terrific. you. both It’s Thank okay

Thank you.

of Our Piper comes next question Danielle Jaffray. Brill from

is line open. Your

for the me on the guess again as study. Thanks PULSAR for the questions. last up following question I A well, for couple

an go In addition be justify Or to into need Phase to to PVR improvement walk end ultimately PVR, on will distance the see enough to do you the point moving six-minute X? forward?

Yes, so, Danielle. question it’s great a

the believe of has point be distance. will primary is going PVR on in we've be this enough been obviously, historical The gold end approvals seen six-minute we and not to to continue space. that So walk standard based

functional we've of the walk So, XX we're six-minute also improvement there given including some distance, and secondary looking for at meters. ourselves that thresholds

signaling. disease mechanism this the one could, BMP Now a area we the as disease we such, of approved modifying do perspective are action of first very from these approved approaching that unique drugs in rebalancing to patients. for that And we the of if said, believe potentially

study And have we're opportunity some to there, the we forward as as so, unique cardiac MRI, some be invasive such, looking we SPECTRA testing. at with looking and cardiopulmonary move an such at unique And points, points end Phase we about could think exercise X. as end

start want so, X in And as Phase we some X, on out be of could indeed, as an creative once as a think flip addition but to they we six-minute as to points we card at do some look looking about we get in PVR, also about think walk, Phase coming improvement yes, patients SPECTRA. Phase X, at see better we we the end

Okay.

So enough there? be would a trend

hearing that said, I couple threshold what we're stakeholders, to think have from increase again number in we've think better of to on care. I so Yes, standard we'd say. the that XX-meter what been like top That of look it's Whence too I we as achieve well, our read. at a think early is seeing we a with and a of SPECTRA, data, we

And Okay. remind Can update and on you might mentioned you next guys interest that? get when we us, then of might luspatercept, indications. other one indications to related for plans what some be an just

be formation in where blood distinct as way. show so announced able that, healthy safe positive only red Yes, beta-thalassemia last that it X coming in out MDS, we of lower but so we as summer, the thrilled did were also and not to Phase well results diseases, risk for was can very restored it a luspatercept cell two tolerable

ongoing to ongoing beta-thalassemia. is We've Now, BEYOND which just remind everyone, we is do settings today. ESA. three COMMANDS studies at non-transfusion-dependent We've frontline got at the myelofibrosis against We've the head-to-head have got which also the looking looking study, got the study, study.

is front-line in that you successful, could all had some add opportunity. myelofibrosis next myelofibrosis settings and of incremental at and we billion to recently if billion. the non-transfusion I over the opportunity So, peak believe independence, an we we beta-thalassemia where JPMorgan, X addition guess sales indeed indications including an an syndromes announced the that, and the If $X indications saw at myelodysplastic looked is of that at of

to that could Now ask an the net. beyond BEYOND teams, is Celgene those And prioritizing of our be at. teams there that, we teams are cast and else looking working we they even and an feel some and myelofibrosis the what also opportunity wider

need but benefits have unmet is could these where the believe mechanism also of to the luspatercept we where indications at meaningful looking are We very patients. high, a

And we alpha-thalassemia, to some high unmet some areas anemia the forward, potentially where indications come. of path need is a we as could believe and some those be more where so examples of have chemo-induced could

Thank you.

question comes from Morgan. JP Our from Joseph Eric next

Your line is open. now

first that I ACE-XXX. to shoulders, us, it with from the guess in talking biceps guess from that FSHD on of presumably questions of seems that the couple feedbacks TA is other muscles, important while some is docs I hip, a and we're doc, from function Just getting groin.

How, where potentially get ACE-XXX back might into might I'm guess muscles the muscle I -- fast other X other whether whether sort and of of of groups forward use what ACE-XXX kind the wondering, trial? to speak the groups anticipate if to, approved. Phase you So, if desirable how more

Okay.

there patient I that other your that Eric, on, based drives disability opportunity is important in muscles and but the Is that that. bit, and obviously out you've are early did some the but been disease is So feedbacks management. further you're of biceps progress The TA muscle what for the cut that think you a some receiving studies warrant from lifecycle asking an perhaps on are around question? may perhaps KOLs

Let's go.

okay. Yes,

going may I'm have but I it's Eric, question. think we the to that, assume lost,

were when that very guess these muscle the reasons. actual for TA it, profound and early to of for think have at we actually we the these we patients I a muscle of group about as the be dominant being affected a biceps to daily about looking been way. identify deliberate muscle One, So, able in the number activity livings

also we it's specifically tibialis muscle for are example, progression. the one in you anterior disease CMT that know think Furthermore, of with the muscle about if that earlier affected

if so, with or or both. FSHD successful indeed And are we CMT

will other obviously these an warrant and effects intramuscular we look would can the muscles to management into indeed, will a transformative then of patients' that yes, feel potential take have We lifecycle injection we at where lives, if dominant we move consider opportunities, it.

we on or to a ensure really studies Phase is consider not. these that into these X moving Part X for of focus benefit functional as the have now, we for reliable Phase But two and X patients

portion? here. won't Can guess comment may with approved. open of and the PULSAR label just you other follow-up if line I hopefully, trial on concern, fully Great one hopefully I over my disconnect rate

Todd. it's Eric, Hey

really out. breaking you're Sorry,

well, was hard we've but really the hear. to that rest So, recruited heard then and PULSAR

continuing either to the converted current the dose and the the into PVR frequency rates extension being on up dose in label I'm the portion for their follow open back or rollover lower here? of patients high interested whether

Yes, just in and example, Yes, to this talk hit we’re when a into not it's for Todd, enrollment spot about finished that’s the me patients get question. the for more about Eric. all six months. we the would Thanks be point, the percent announcement rollover through able talk have as something line but we top to that could at

get And rollover that be. better would point be to a so, rate that to what data would into

Thank you.

Our Suisse. next Martin Auster from question Credit of comes

line Your is open.

and Habib for taking Hi questions, my thanks company.

of a trial from one kind standard endpoint PVR or trial some the to First just The primary earlier guess powered of the I and luspatercept is question. SPECTRA follow-up an things clarify reduction. is for

flows you you of if bring current pipeline so the what sort kind magnitude in is looking Wanted assets Just want at mid-stage external much Thanks. thinking the coming secondary Conversely, had to powered minute seek BD a think pipeline? kind obviously you be six good on you’re can quickly partnerships cash to about been to you’ve to of clarify to of for both around for it’s question not of What you've And forward, do how kind pretty endpoint think cash then discipline the which success bit XXX% with really stage from about potentially going my have global and in there's you you registration those? luspatercept. do little geographic a potentially you walk? generating got would a position adequate or programs cash about that resources as pivotal and significant of manage flow is assets in. talk second

Yes, so great, Marty.

yes, around really So, PVR. it's high the for powering endpoint part question and for the powering, primary of the first was

intent strategy is forward, and So, current moving second of around it’s question and well the the as from really part, our organic both our as our cash the resources inorganic. meat on

of providing each when for guidance we was every in be would the to I one things had So as time raised time that. and remind last time done the that cash prior rate we again capital a articulated first very run I just January not have to

our therefore the have believe that is that burn that overlapping one suggesting we point raise. our if we’re took a based we year Marty various never of as assuming rate you saw and you're another typically will is reason the will trigger of luspatercept trials us Phase and that received the planned where really for would royalties current conclude on And milestones visibility to exactly need that as that able all X to which and for we cash

any inorganic and to Now indeed all said, on would Phase heels that that but a build a potentially X forward us then obviously if from the indeed our that be strength, card position the of and the change in again of opportunity feel X forward, moving as to we we of posture, or to of out strategy that commitment to our help an flip opportunity Phase we we’ve areas the therapeutic positive data. outlined our have move if programs leadership that Phase would X on there’s one

looking at partner whether and generating off that indeed And alone. it felt and Phase be to we that’s if would or value ideal X not then it seeking better or investments go an be we'd

of best we we is X X, to and so, conclude look sit excellent the able Phase will assess we're position to then able where forward. in now a those take affecting path be where to good our end at an think we at we're them Phase as but I studies. And right all what sit, And the of

forward? you sense position. terms profitability to an those in in maintaining building Or investors profitability poly on of to is metric enviable pipeline? kind a that's in agree frame start accruing dependent opportunities of you're as Do front and or retaining you that of in you terms that important in as going royalties the any have the going be be up can or I selling going for how the of

is do we best. to is goal that I Yes, the And to Marty, patients focus And do bringing so the serving. to medicines to innovate. what on that I end transformative the again, we're day of our at think

successful. into in and we position think Phase our we're moving we're forward of cash from future point enviable if of in view terms an ability move to would a X our I how dictate

be we're think therapeutic where position areas results I that between positive three going on Phase pretty enviable are Xs We've in X from are, that two we've we're got we three pulmonary also hematology. got But or Phase in a a situation and what strategy not it assess able those wonderful and take of best to investment all the to neuromuscular. their conclusion, And to studies whether here we're will to be. in then

will focus, that disciplined, do we we continuing is our making said, and we're be value primary so our as way sure to always That that innovate also to a to shareholders. return in

you. Thank

Our Porges SVB next Geoffrey question comes Leerink. of from

This Hi, is filling how I in and for the and how Neil how revenues and explain you be luspatercept between Geoff. the expenses you. responsibilities EU? doing? And just Celgene-Bristol? U.S. upcoming you will have and one the shared in regarding question report you Can intend you do Thank to launch.

Yes, there's for maybe questions pass anything little even and that on a I'll again, perhaps for on the we can commercial Kevin question that to reporting. Sujay, provide if detail great. Thanks the

Sure. Thanks for that, Habib.

collaborating And with closely Celgene. this juncture, of are so guide, data we sort very at

that day the our undergoing through we launch joint operating relation frequently our of trainings is map is launch they're in promotional to them a team plan, of are joint sort complete worked needs now so monthly through approach hired we one a or to the as strategy as together basis, almost well training. field aspect team a plan meets collaboration a committee that with developing discussions XX to out on approach our So to really have

with urgency the really a that place of will physicians. the of one, taking be jointly prepared and and patients And sort to we the sense those there's co-ordinations support of needs Celgene, are day of so, ensuring all

to approval, as focus. physician primary the population, our that's call physician So both equally have the have right. MDS once on a as we beta-thalassemia We well the target population

elements there's that on, and training that's So at at going no more going juncture. this this on preparation juncture, promotional there's are

up So and divvy we'll sort call. of

we really our level so now that's to are the Celgene with at compared actually Celgene. that of right basis coordinate territories, our larger decide local to we doing collaborators as territory planning execute a optimal planning part And Now, for account and what's territories their well. the with are to us

Neil. Hi, Kevin. is This

As us of far goes, portion for will direct large, appetite reimbursing reminder, cetera. Celgene force sales large reporting BMS as as very or commercial the our a a et be

P&L in a the sales but the revenue will you in down marketing expenses line see the corresponding lines. reimbursement So, in and up collaboration

reported profits obviously product reported revenue based the In will reported be achievement. be quarterly in basis addition, on related revenue and be will on will a revenue place the standalone related and so us, have for royalty royalty we'll that

you. Thank

Next comes question of the line Nochomovitz Yigal from of Citigroup.

is line Your open.

and clinical lead to more CMT possibly Habib, a on FSHD to just in to specifically, to question. the more Thanks. and likely differential in of benefit dig muscle just little or similar diseases bit either is that there reason growth? yield any believe Hi, growth. of And taking the context FSHD is some a for muscle volume those of I particular could thanks versus in the differences biology wanted in CMT the more a two that

question. Thanks for your

I'll Officer bit. elaborate a Business pass our that Quisel, on I think John Chief to to

treatment two up patients hypotheses Habib. trials, about for the we Sure. were that most Thanks these thought and Thanks one Certainly, a these provides different FSHD as benefit. where lot disease we view as testing setting about we your question. essentially,

ACE-XXX the of restore regard and leading to to the muscle if the function factor, can and don't pathology you those can it will FSHD, of which have bet of to muscle disease we in I a we on DUCs-X is one the take with rebuild fibers transcription is think So, work. a the over-expression premise more muscle With really patients. where likely in is the degradation strengthen starting that

CMT then of the give and that's and of test. flip-side, there consequently muscle we prescription a disorders of variety trial experienced innervation the in the fiber of where are neurons the patient On death intended that to setting loss and a

a there fibers. patients partial and that of innervate the slow So, are the are having neurons who muscle retraction

particular muscles manifesting foot fraction lose the the in And patient. still fibers is is affected limb only innervated have drop at bottom for what leg as strength, the where muscle the muscle a non- the you a have so, result, are TA of innervated you as the at and of fibers the

loss and using function. partial to then restore a have muscle like due of ACE-XXX, you if should if you muscle the can that innervation, is mass restore premise strength loss something the to of be able strength there the in And you

work. differential So two a that trial with premises tested CMT summarize, disease one again, that likely more in being and And like disease muscle is different which that trial. with origin FSHD being neurological to origin we to wouldn't tested handicap like in

Okay, believe there is got you those end likely? the run of respect can make to more climb, end walk X-minute before confidence it. any in that the more one points? And a XX-minute muscle walk, four then there? to the those you higher reason Or of different points, the seeing one any to with comments have stair just matter is Or data is it do or of

is of design at establishing be data-driven, know, endpoints companies we're, be run in trials the different data. few to totality with trials we these It really the so space the the related the measure the clinical appropriate and in and readout of that the really as to suite one we looking going of we’ll patients and endpoints are to a

as patients it’s Yes, Rochester with also and validating health data University Yigal, the well with validating index only a activities thing at where going look affect looking as we’re and the be at and the living, be, that which would questions, to the in number be we I index as that. will daily Phase the that, regulators X a working looking of of working of questionnaire PRO, we we’re assessing to would as FSHD well health add to closely CMT very

priority Was one say thoughts Okay or wasn’t you so, that maybe in review I'm have question received that you received just you not Celgene If makes sense. MDS standard if beta-thela's get this just like your that expected? Thanks. okay, to expected what what asked curious you review. you and there? And then yet, to

our Yes, our actually, that We efforts indications, said review, and indications, priority execute able it’s of that all been we or pleasantly one but I’ll not we are for regulatory to footprint commercial repeat from expected. prepared what supply from one we we footprint, execute. a got actually a review be for surprised anticipating both standard whether priority always got whether we expecting would we our have to of both that footprint, a prepared review or to that and on were

Thank you.

of question line of next comes Oppenheimer. from the Our Gershell Leland

Your is open. line

Thanks the great and progress. taking for questions

you sotatercept of came PULSAR came trial the therapies on ask into this in severity the patients PULSAR? other if the on terms essentially were in time the they of can trial on question nature First of who that the PAH they enrolled, and their on to into fully with of level comment want the

class of X normal X Yes, criteria Todd. and were so, got really patients trial Hey, from what included down split Phase expect X standard looking, the as based and as this inclusion Leland, off in size. it’s that far which you’d of and functional to the or enrolled countries then far as that that single, triplet, comes care a what for we patients a the is doublet And we’re the got so a country.

country. And so, we got of trial enrolled the per appropriate how that off based mix

questions on of then and great, just couple luspatercept. quick Okay

MEDALIST to I think for fully BELIEVE. the the publish you’ve you data planning mentioned and test

Just you in coming strategy, peer in can up that on future? those the we wonder be publication might if near seeing review comment if

Yes, studies so both the of still submit the goal we’re XXXX. to on track. And is in

comment enrollment? can I or can may, then any provide Great you if just on on and the color community last provide the you trial

obviously study. absolute full to expect for Leland bit you closer in today. provide new No, further that get top we’ll share And enrollment, color to can once continue timelines terms of to we with enrollment you a able to little exact more updates, nothing but release, we be be or the line pleased

you. Thank

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muscle, Hi up potential for moment. X the change duration what of the to, think the muscle longer Phase underway role maybe with getting Phase you sustainability X taking and here, And trials the secondly, of XXX on, what What duration with back is trial questions, a towards and and agencies to the good the you thanks our And wrapping for extension going here term? everyone. regard pivoting stage? afternoon as thinking maybe what you over comment you're change there the proceed And forward? the can the, of

hey it's Yes, again. Habib Paul,

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two, patients were so, three frequency every the weeks. study just we there in a the the of primary eight back at When to reason roll was eight to and part potentially study, two indeed, And they'll view. over a that again, either guess extension at look sustained look dosing or for functional took four weeks I randomized dosing, had point end point we'll so if the one in into that's And from to we'll able extended back a our see materialize for was as and we we weeks, to maintain and interval going efficacy six get everyone, you up to the data, read when know, we dosing part better effect. every part dosing remind on believe to be at but more appropriate be every the patients specifically, months. week And of intervals,

time, but very we're and will in that's same how long we out the fronts. again, a to to be things so With patients at one question to more of looking study that both extension respect drug the the on at the as going come, important last, follow

do can the I as you maybe and great. think as about you you're here And Phase where Okay, the up that, and you expansion? see, not-too-distant guess for opportunity development it a for future, label just data to potential luspatercept maybe non-transfusion-dependent what bigger think where or the optimistic? about more on is potentially quicker and being transfusion-dependent population? in area Thanks the Is in myelofibrosis. coming X

another good so Paul. question, Yes,

dependent But and because those patients looked on ruxolitinib who again, the we when study, for at enlarged ruxolitinib. of at X spleen the we non-transfusion-dependent. transfusion and also we on are looked Phase recruited So who not are

So we well. dimension as to look need that also at

if only you most because important focus are the I these be all in think fibrotic suffering bone across that's everyone, would we need to on, Quite to patients that suffering unmet of that also marrow, areas disease mechanism they're not from our focus area again, an and of inhibition. with from remind but on that probably simple those ruxolitinib the would argue a action patients just anemia drug-induced to frankly, myelofibrosis due it, Jak are anemia about due the of highest

point unmet you the that, internal it who cater ruxolitinib, ruxolitinib to patients been on patients of and on, that severe would have moderate the of and to if over that are estimates opportunity here think have from those most arguably an largest in view, this population. for likely be anemia about So that are transfusion-dependent, our from to patients, on myelofibrosis half us suffer that need

by And question the it haven't maybe the I'll anyways, you give, way, asked but state

Yes.

been an What group we research to this of in ourselves the lot receiving. a that hurdle XX% based have we've internal given about and on rate efficacy of also XX% particular feedback

us forward. hope the to what confidence give achieve, we to wanting to would move So, that's to

you. Thank

Stanley. Our Hung question next from comes of Morgan Jeff

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the Thanks taking for questions.

earlier you are MEDALIST they'll the luspatercept. get said bigger the from that patient patients, changes that the can benefit better have For the population, you

kinds So the initiatives education or diagnosis patients earlier encourage from time to commercial of what versus month for standpoint, other since you median MEDALIST? treatment a XX are considering in

Yes.

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in one Luspatercept. that else is terms other were be to of patients to of for keep nothing of ESAs on able as mind fact things Now, in on is the lot because go that, to in a to alternative XX study an the such there really months

may drug that that to hoping for a we And if the to an will as we that another get indeed in that. patients study, got luspatercept if we're an ESA approved be patient so, able be it COMMAND because us But market exactly COMMAND is by doing for really successful to most them importantly the fact earlier, is get on be refractory mere to treatment. important option is the approved earlier indeed study

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prior quarters, then like from SG&A we for of your so SG&A the Look relative and about thanks to rest QX up a sales year? reps, own little housekeeping such QX should to how Great, ramped given question. from as bit commercial think the preparations

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revenue an and see with also programs and coming SG&A ACE-XXX. by will it's program, commercial line increase of support you in marketing our in efforts corresponding both G&A increase So, the normal the the in environment local growth that Outside line. into collaboration owned sotatercept the additional the that the internally luspatercept in along of but

and seeing XXX if PULSAR can that side the the trial led trial program studies now is R&D with had it's any expect the Jeff, can most those we’ve here Phase quarter four quarter where we on in you’re that’s patients CMT X expected trials. FSHD, extension internally of with ever the running time you the so to at you the imagine given include this as SPECTRA through Todd increase the Hey, end

you. Thank

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

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thinking on elaborate guys, please for rate is and and to study myelofibrosis hurdle follow we Kennon. that how about is had Hey on up both XX% the hemoglobin could if you Vikram on a independence this you that? improvement XX% shall mentioned just for I transfusion that or be on

really we’re Hey wide Habib bar proportion endpoints any enough Todd us population we’re a casting that to we going a for Phase with patient so of as after describing and would and pretty this yes net is just that that multiple any hits broad patients that in X. patient it meet if be move that it’s forward population trial was the here potentially

over progression the likewise dependent do or of dependent know become anemia that in be course time transfusion of over dependent. patients could so, we it combination dependent the or disease or only rux this monotherapy non-transfusion transfusion be transfusion luspatercept majority a the with And could though

this At Thank to over I’d Dable sir? to back time, call you. like turn remarks, the any Habib closing for

close the meantime continued we call thanking as I by to the thank you you story a interest the you just the in I close the over everybody of joining again. great Thanks I winter the meeting off and many course Acceleron’s fall all remainder of and and your of in look year want summer. wishing for to for the very us. forward much

ladies today’s This conclude for Thank and and day. a have gentlemen. does conference. you you participation wonderful Thank your you sir and thank

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