MyoKardia (MYOK) 9 May 192019 Q1 Earnings call transcript

Good day, ladies and gentleman, and welcome to the MyoKardia First Quarter 2019 Financial Results Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions further participate will follow at that time. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to introduce your host for today's conference, Ms. Corral. Michelle begin. may you Ma'am,

will you, Gianakakos. II and SVP Jimmy. on joining MyoKardia's today's you for Tassos of who SVP our to Today, Leading non-obstructive earlier Dr. and Harris, call Financial of A results in quarter afternoon Taylor Jay and enrollment we're mavacamten quarter accomplishment Good for CEO, today's including Tassos in our afternoon going is Marc we and joined Relations. is us Investor website. Edelberg issued Officer. Corral, recent is Communications this and HCM. financial thank joined reviewing call. first from Corporate financial our MyoKardia's of by Sanofi Clinical corporate all discuss and Thank Phase Semigran, be clinical I'm last press MyoKardia results Science; Head completion was available MAVERICK Michelle Chief as Medical our Development; release the

information those hand as to Tassos? CEO. or Please factors like for from over information filings by the undertake implied no call to that results I'd view revise May except information We XXXX. reminder, or well refer represent reflect will concerning X, call by statements. now to as to could required press of expressed future a update Company's statements to which statements, with to today's release discussed as any materially obligation differ or the As risk law. new our forward-looking today, include this forward-looking these events, as during the SEC the cause our actual

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EXPLORER of mavacamten Total R&D of Thanks, reflecting expense. Marc. a increase by from million and million, R&D and advancement late-stage MAVA-LTE. approximately highlights of quarter million This few including MAVERICK, with including approximately I'll into G&A the start were studies, of $XX.X XXXX driven results. expenses our a $XX first $XX $XX.X reflects year-over-year million, operating spend,

receive net And collaboration And our mavacamten million QX agreement first will of a approximately expense $XX the final Our be reimbursement Sanofi. end of from R&D the at quarter concluded was reminder, the quarter. our reimbursement related formally to as with of program. to Sanofi QX

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six-month milestone of the one on hit we're in completion MAVERICK far release with enrollment second with data the the of our milestone close major imminent XXXX, study. in PIONEER-OLE our we've So and key

we'll a will in we'll additional And and be readout study. year, our from PIONEER-OLE. Phase the Our Failure. well the MAVERICK reporting pivotal our data against In this clinical goals Phase into from as will II midyear. longer-term half from from ongoing of ESC provide advancing studies be topline Ia MYK-XXX productivity Phase our lastly, study EXPLORER our execution program, will enrollment in presenting this wrapping data month continue. third later be up I year, the at Heart second And MYK-XXX study, we Later the as data candidate

open questions. So the with now I'd to call up to like that,

Edelberg Semigran. with and Jay Tassos Dr. Marc me are here reminder, Dr. and a As

Our question of first Tazeen Thank comes Instructions] from Ahmad Bank with America. you. [Operator

now is open. Your line

Thanks Hi, between good you you can of afternoon. taking us how to market give I obstructive differ think obstructive is directionally non-obstructive the size? comparison questions the but HCM sense a get market? just my know a guys. for that the opportunities I larger, about wanted and of

joining U.S. at that Yes. minute, call. for around and – If one some the our certain can look leads regions world a good we I globally in carriers for to plus the Hi mutation minus people about or are that Tazeen. think got we in Thanks evidence XXX we've of a pathogenic HCM.

in XXX,XXX through that one XXX,XXX them a or them to and XXX,XXX the about So folks those But will so and medical so people, obstructed population of insurance of track XX% non-obstructed. climes two in only, seeking we attention U.S., translates the around system. be are thirds of or can that third today,

be patients we think that we the launch the in a the going into still a situation for benefit. context non-obstructed patients to U.S. that think about state minute as if obstructed at that's which as and was twice HCM relatively of the about product current for and XXX,XXX-or-so for assume So where many could potentially least, the we about obstructed both diagnosis is low rate addressing

diagnosis rate as the like between I registry on if increase and things other are well. think and in the approved, of we'll you're an increase We and those and that hopeful seeing our working mavacamten, efforts we're XXandMe with hard to share introduction see awareness,

once are become needed sales And the be commercial? Thanks you would of how Okay. force the that size you about then thinking Tassos.

is Yes. That set in I U.S. today is tell treating who in are it generally of a very the cardiologists folks can and these focused you, Europe.

excellence, and HCM. focused are centers patients cardiomyopathies a outside for in tertiary specialty additional between XX or then XX thousand And are these so maybe U.S. So about who couple split are of those heritable some really and of they the where centers. cardiologists are who are or of on generally there roughly cared

very effort is So a much for focused us. that

kind would I commercial or order model. the, but so, think XXX orphan in say, minus, very of I in the plus much of reps or

last Okay. And me. maybe then the question from

subset? therapy? amenable which In of be you adjustable in a to it's terms patients population Would would say certain most would your entire or this the you population think

from Well symptomatic I patients which telling think suffer seeking for improving who them we're and generating function. are us to the that sort we're The symptoms they have attention, who these symptoms, of are so functional evidence most that people far extent or medical of limitation. some that is

had opportunity that a valuable think So extension bit to patients much on, downstream. their have that process, further atrial Marc, us the the and ones help open-label more on and evidence change term start an like interruption getting have Yes more from little the aspects that add. able that those fibrillation get just disease with the If out, disease we're really are more to the and please those earlier, of to of physicians which folks. for or and really that complicated that gives for seeing exciting therapy much suffered involve this longer makes we bears that in some disease benefits are particularly some the more I it

in obstructive just There talked disease mean some therapies work in blockers. mean do Beta beta you for were case patients. of hardly is don't to a the clinicians with answering, fact that lot they experience about and past I patients the blockers, that targeted Yes. physiologically work. the would non-obstructive mean why they can respond even I no make I we've a

that MAVA that go. So to non-obstructive I the for think going is way HCM be to symptomatic

you. Thank Okay.

Morgan Stanley. Thank you. with comes next Jeff Hung our from And

now Your line open. is

aspects did should that wondering We other any expect Jeff. six as of on Hannah be this this additional you on? year, for aspects we focusing do same think there the later months with data you open-label is data? to for PIONEER Thanks. the And Hi, the were you report the are data largely

this is Jay. So

volume the be to to I are on Heart to call size. down sustained York reduction those of Association continues to progress exciting the the the as And to very of as how want PIONEER-OLE attention reduction class, really course, you the sustained reduction wanting mentioned, in improvements in think in the that durable atrial and in see I focus gradient impact sustained left will NT-proBNP. New benefits and

And I then more, if one can. just

expect whether DCM do advance the what for And that? data deciding this later to On the what you do for the bar you year, not? see to or as report program

those Yes. the we're We've doses, looking That's Phase data shared studies and and in And single-dose we're seeing in far people some about. We're what foremost I who function. range so first what volunteers in excited of really and for a program healthy have well-tolerated in reduced we're is which systolic program. I. single-dose Phase

safety. the in we're out and which ejected squeeze is on volume, some with at systolic blood parameters volume of the also amount importantly a tolerability the hemodynamic of So eye heart. looking that's stroke seeing every of keep But close increasing improvements

in stroke seeing pretty are stroke improvements exciting. XX%. to for in be tolerated volume well a of dose multiple And XX% us improvement that's XX% study volume We in a this ascending doses will single

what results similar far as so really looking seen really doses what we've single is we're in So the for.

Okay. Thank you.

Hannah. Thanks

you. Thank

Our Baral question next comes from with Cowen. Ritu

line open. Your now is

taking guys. for question. Hey, Thanks the

fraction or MAVERICK dropouts like that overall has the compliance, fully DSMB trial you've trial conduct enrolled Can close they're safety looks et you tell gone is how if with on trial looking few at cetera? a us to. now and ejection many reduction and Just that how during conduct had

Hi, It's Tassos Ritu. here.

into at so well. enrollment far. pleased the are excited of asses get for are that; dimensions, but going can about what that patients the we're who coming you data tell a in, specifics collecting, enthusiastic point, just assess that asking that at the the things really the that continuously not as we're we we the rate, look of quality to couple we and We along of So is study not the very of some progress but you're this

So where at. we're really of various we're in the happy facets, terms with

Marc. so one that that And per clarification with of said what's there? XXX-nanogram you said the and protocol hear I Got like was – the doses. some per do mL the I of just wanted stabilized designed it. that's at below you case, achieved above it mL how targets. XXX-nanogram patients or this, and designed a I target And your but misheard cohorts, did you X MAVERICK comments, Maybe you right, was X the If

Yes.

So mL others at There some three cohorts. mL, we're get or picogram we targeting different XXX-nanogram where patients are where XXX. around to per per looking have we're

we so the in range obviously overall there be final. going to And therefore, expect a

fuller regimen our range dosing the this So patients. registration gives around us design we can actually that so right program for actually a

tightly titrating going there's XXX variability be So XXX, or range? some in you’re to to that not either

It's program. actually that information full know design built get in best to make registration we sure that of so the we PK can that to because we need range

Okay.

And evenly other…? – there distributed I sort of evenly range? patients in will so are be guess, Or this

are cohorts The X:X:X.

obstructive how symmetric and then Got and the you okay. obviously is the basically hypertrophy, non-obstructive about hypertrophy main it, the the – between the differences centrally. versus difference And talked non-symmetric Is of between, any dyspnea any guess, measures? between Is and of in differences the severity the profile rate of symptom obstructive there any there I and or non-obstructive? difference functional dyspnea other the

cetera. of with fibrillation, atrial capacity patients limited. in Heart as obstructive non-obstructive class, in York cardiomyopathy exercise rates well reductions received So both et increased They symptom as New hypertrophic are Association reductions

that patients And those we're of thought the to actually on limited. focusing characteristics patients would we able we're patients. get who the of baseline we identify PIONEER have benefit to are use profiles our for maximum – where So who

But two in in Is severe? it. population is symptomology there these dyspnea, less it types patients? Got other sorts more of differences of between Are severe populations? is it the overall the

loss is we target limitations exercise strikingly two our capacity are endpoints groups terms consumption the York I in the ischemia Association, the obstructive as But those so so mean the assess syncope, consciousness more Heart more patients. between common is patients. of to agree I New patients. symptomatic there of Hi, common And related functional in obstructive chest I and of anginal oxygen are Jay, similar two. mean capacity peak in in with

the Got and questions it. for Marc. Jay Thanks taking

Thanks, too. we

you. Thank

Anupam Our next Rama JPMorgan. question with from comes

Your line is now open.

specifically second and Hey, on for for much. Taylor, you good some but pipeline, cash in is the other broad the until was congrats guys. plans taking assumptions Thanks assumed question, that the and progress. so what question maybe of for the XXXX quick guidance noted half Thanks development Maybe a MYK-XXX? made on for you the comments

got we’ve We've mentioned, also we're after the to we as registration for for MAVERICK in in program assumptions so going non-obstructive. to Yes, mavacamten built Sure. full move what obstructive. in

strategic really key of to a good you support think and comment goal probably the that I there got is is pretty which idea a to So Tassos is full made, mavacamten. the benefit mavacamten, here realize

really terms we're the programs some both later initiatives in some we HCM we us this value the to we're do lot to full doing have building exploiting make take to follow-on as get recent to other there's as full good So of other is value Development then Clinical that of where well year. community the sure in on options offering a delivering once these and milestones mavacamten. and of also allowed But what is our

also to populations. so we potential with what do really some option in that guidance the a proof-of-concept this to we specifically later And from the current targeted II, data, MYK-XXX, data move runway. have can do we into next the steps able into get to working show there. built we up where patient that point this we've we're So talk that's not is Phase cash now option year show the team have but our to cash only built But into studies from right when the about potential and

taking for question. Thanks our Awesome.

And next with Auster Martin you. from our Suisse. Credit Thank comes question

now Your open. is line

Marty. for think experts taking is This we VOX driven improvement. about when Hi. benefit gradient mentioned guess, in respective first, I of from Thanks the components? each on and for the should questions. PIONEER to these diastolic of function in How they the change talking both that maybe degree improvements by Mark

in improvement the to improvements components what significant as need is a at obstructive impacted think we're overall peak seeing reduction of you look in a And holistically. So well. These diastolic are VOX, the symptoms. in in reduction take gradients, I this hearts the the severely

one separate that is out the think simply this going we integrated to So fully of the actually isn't and because other interrelationship. from one

Mark, more. is have mean, and right. remarkable, compliance. feel very is What this. is That's in this And there filling diastolic persists, published likely, real people some we're to now animal that haven't both it done data been pressure. do this That models lowering I increasing drugs and That and help out filling of if is we've volumes. better I people there seeing there, would pigs in improvements like in just too. add

a So on we in impact this can potentially want improves the population don't and see underestimate have we future open-label and non-obstructive to of drug data and time the like value if the potential extension. that diastole functions points pan mavacamten the that symptoms out in

And expected the in or MAVERICK you. Got VOX baseline can in Thank compare versus VOX PIONEER? baseline MAVERICK peak it. then peak you

demographics. targeting similar baseline are we So very

Thanks Mark.

you. Thank

Thank comes you. Fitzgerald. next from our Young And Alethia question with Cantor

now is line Your open.

Thanks. that regulatory are you think think outcomes think a with meta-analysis what the Hey, to Do bit profile there's my like you evidence a and think do guys. any about straightforward? kind maybe event. obstructive taking little I is death risk kind of I what of when cardiac thinking longer-term pathway, talk sensitivity more the seems to long-term studies which is there know not there's question. Thanks some next? for little over where the pathway suggests more from want I just non-obstructive Or on is about that of – you much similar about

Yes.

the big we and exciting the and of similar certainly hurdles and efficacy. at going we're do for think looking regulatory symptoms the can Jay, I in, for evidence terms the a Right safety, evidence FDA the those beyond to registration, in to think now of likely NT-proBNP, support in part chime quality are story that us can hurdles Those in really but going are in effect Alethia improvements be cetera, now, be it's pathway of they're function reductions able demonstrate here, et a in for They're conversations volume, if LA but I impact for second disease extension, some life and is how going open-label those. are day-to-day of if are to to to of also facets, we important. improvements improvements. burden we in those going in exciting. at two can that But reductions registration, the things really seeing we impact very

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particular bias exists And Jay, my kind some think studies about? on there's like any to that of comment outcome

are Well, remember, the functional have looking to impacted. They – that symptoms. we're that we're studying have in that They overall. patients trying we're improve limitations and MAVERICK

that of to allow that out would here, were expecting registration right positive so us those to based able on symptomatic type much we're we And way functional be we'll endpoints, that PIONEER pursue identify to the program. able results the identify coming a of

Great. Thanks.

Alethia. Thanks

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Yanan dialing Hi. for is the Thanks for question. Jim. taking This in

looked question, ejection at you from something HCM the perspective So study. safety in first is a fraction obstructive

side, from importance, importance is of similar HCM ejection non-obstructive less wondering just could So importance or that? you the safety-wise comment or more fraction on

we're dosing take regimen program regimen in into EXPLORER forward testing we've our way registration the non-HCM ejection could studying fraction important identify PK obstructive patients, the in identified the different dosing much that can we so the Yes. non-obstructive both Monitoring trial. why that the right the patients that effects on that's we're the as as and of now are doses well there we in range the

you're so from thinking side. Great. And HCM then also obstructive the

using dosing. in guide as imaging a success had gradient of the You method to

validated your MAVERICK, help can with to that no outcome. think the you or non-obstructive, So use do know the from probably volume imaging way? LV identify some you parameters for dosing I that's compliance could there for to future study potentially you like guide imaging then in is in or pivotal But data diastolic

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is Phase program. right we that to know trends, identify a will us think registration this us identify a – it II that for I allow what's trial allows that basically So to these

which know question. – fashion in I then MAVERICK. last well as MAVA-LTE Got EXPLORER enrolls study, from And from it. are running open-label you patient the as

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Yes.

MAVERICK out. of of of looking looking the results The from topline next that have come the this totality the populating on extension to and then at EXPLORER of the And we're these later year. forward year, the obviously looking really forward results together. is if all results will We’re long-term to data patients

Okay. Thank you.

Thank the queue turn I'd to Gianakakos in for further Tassos like call this the you. time. at showing And questions no back remarks. I'm to closing

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