MyoKardia (MYOK) 8 Aug 182018 Q2 Earnings call transcript

Good day, ladies and gentlemen, and welcome to the MyoKardia Second Quarter 2018 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 will be given at that time. [Operator instructions] As a reminder, this call is being recorded. I would now like to introduce your host for today’s conference, Michelle Corral, Head of Corporate Communications and Investor Relations. ma’am. ahead, go Please

Gianakakos. thank Harris, Corral, joining Communications MyoKardia’s today’s will Medical recent CEO, Relations. and Dr. reviewing today call. Semigran, be Financial Marc and for Officer. I’m Leading Chief Tassos MyoKardia’s is Michelle Head and call is you afternoon, joined us Chief and Business June Dr. are updates by news and Taylor for release the Jake Officer; today’s our Investor Media of on Tassos Chief Operating us Good Bauer, Development financial of they regarding Investors found Corporate our portion call. section with second here the Lee, Officer; Q&A of Officer Chief also results. today’s the website. A MyoKardia be and quarter for can today’s Together, press

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

catalyst progress creation. will clinical making and two for sometime everyone, with joining Good afternoon, upcoming encouraging and where on thanks for we’re value highlighting programs spend call, us reviewing the today’s we our

a where is into that, midway I through with big perspective picture on jump MyoKardia Before XXXX. wanted to we start

you’ll Phase trial included parallel, new of the any study, our Xb for In start in for company. Phase which X the capped pivotal which more we’re dosing patient clinical about The completion hear driving today. by trials in a is milestone DCM, three our quarter huge second commencement of mavacamten of towards

in committed to view exceptional we’re efforts a hard simultaneously stage power people and pipeline and in speaks gaining patients our dedicated the pivotal community. exceptionally which day. clinicians this working to without MyoKardia’s of MyoKardia the while to of more our confidence of every in Moving would None approach, the the to building quality the and of novel agents support possible team be more the of and HCM and

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the we’re top we exemplifies We’re will that endpoints data optimize EXPLORER on EXPLORER plan where assess expect drug The putting first. study and in the And dose. functional physicians approach an symptoms to and their that patients The advantage taking of define in for trial, in and trial and our expected also each used motivate XXXX. priorities while changes with during many the for real how PIONEER pleased driving adjustment of world, capacity. we the the line patient’s at successful our all with Phase allowing be is dosing order X the of both principles second-half to really in in the evidenced-based, of mirrors design learned organization.

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ability clinical optimize of patients us relative obstructive real to dose dose likely mavacamten helping Earlier is six Phase in to this addition way clinical to that non-obstructive of this MAVERICK HCM the initiated world. MyoKardia is planned in initiating our year, studies year, their year. EXPLORER is the will treat PIONEER, indication. one patients are those Since underway. the to of In we and use the how X four trial mavacamten HCM reflective second of personalize now beginning studies this

as This long-term EXPLORER of in trial. overall extension patients enrolling with begin MAVERICK study is mavacamten part our FDA. study we’ll year-end, Before and known program the reviewed long-term registration extension Mava-LTE the

XX extension with patients lined a participate enrolling PIONEER We’re more the in the months. coming open-label few goal up of in where our enrollment to hit we patients study,

first year. We to MYK-XXX in dilated And our advanced finally, of with a expect be this to into cardiomyopathy next year. shared this from we’ve candidate Phase share the quarter study data later initial data for trial Xb

to for to our provide of plan ascending ongoing trial multiple patients is Phase clinical advance over trial this MYK-XXX. before who an the X now, turn a XXX Phase it will update end I’ll into Marc on Our dose year. X Marc? DCM in

everyone. Thank you, and Tassos, good afternoon,

excited to and talk But why on lot about of activator on spent you time like We have to about a I’d today, this I’m compound mavacamten. sometime spend MYK-XXX, appropriately program. lead our

these the have in and at myosin frustrated for in target of necessary a Sarcomeric In in not supply adverse to dilated failure In have many Mass from aspiration heart that is Xb healthy blood compound cycle actin to XX as design for see of normal enrolled. preclinical XXX the The to patients essentially And contractility are aim of enables intended. to DCM or an normalized a to MYK-XXX that can an in from care the is Insufficient seen doctor blood. absence years We quite crucial occurs each chamber, contraction. approach an we is also in part detracting phase blinded increase fluid without left increasing MYK-XXX blood relaxation an of HCM, the of to the and mavacamten, contractility left adaptive gather contractility. available. function hopeful study relaxation. our hindrance contractility and this that time This actin diastolic in compliance, but randomized impact very the all of and stable ventricle presented As ventricular selected are myosin contrast resulting heart without the failure. by of actin of is our disengage that able and cardiac that the in maximize that patients have must is studies, the the trial cardiac was heart this directly the heart General In increases might a with to into remodeling represents dose our ventricle allow If impact years, in towards XXX in treating the oxygenated underlying diminished evidence heart as see of limited by function. that contraction, information succeeded. single increase decreasing retention group it’s for on muscle. refreshes over function under more was contract patients DCM we detracting the ascending currently with such pumped developed cause without ventricle on The which is not enlarges. mavacamten such DCM new positive Relaxation the Myosin binding provided normal patients, the congestion. changes for see address DCM healthy will different we will from relax clinical and compliance. it therapies pumping the and will works ago. improvement reversing nourishment acts had muscle thereby research to heart [ph] left prognosis. to force the in we cardiomyopathy, placebo-controlled my similar restoring we active to I as a is improving heads left the has when to patients designed was Phase a we with MYK-XXX filling it activity engage to to development disease. only is of contraction next fill from patients from patient and one heart

have eight of and up cohort the doses receives will to XXX or patients each placebo. patient in first We sequential

able placebo. washout and then single receive trial not the aim are twofold. the really of is the dose safety the in placebo and there is effect overnight period dose or we compare of see patient. a Following Xb and either dose, same given the and others the next monitoring, a to that we of way, against dose The activity In this patients Phase only

We want is well may ensure we safety. be for First believe and looking to that a foremost, doses that we therapeutic are in tolerated at XXX range.

Next, thus from we’re Thus, we Xa share function. of first diastolic as so with from discern, proof Xb top while in plan trial contractility contractility looking details XXX Phase improve further XXX that along we’ve for improve preserving mechanism. to healthy volunteers. data this this initial Later we performed line year, and trial, Phase far can our XXX

in contractility there targeting be will starting effects are patients, manifestations neurohormonal year-end to use a recent such multiple the trial directly and and disease. our the devices activator primary I’m cardiac that mechanical diminish are address progress the outcomes intent by DCM heart well problem, patients very circulatory failure these DCM the support also rather positively the with docs Xa in heart activation, secondary Implanting impact treatments by in their approaches and the we advances transplantation medical target biomechanical that address We of Phase or programs the no establishing therapies. of contractility. therapy, potential excited Despite directly proof-of-concept. to drivers cardiac about ascending DCM side as of of know the we patient diminished of drastic dose complications

this our subgroups the in might clinic of our Taylor This to learn review over As activator Day. inform actively more I attributes, benefit second the at programs. will most XXX’s we about from development contemplating call more that With potential turn upcoming We R&D will quarter. turn plan agents. results for share our in thinking to we the that, the financial are to

you, Marc. Thank

for in successful quarterly the second and which I’d quarter, our support offering Before net the shareholders follow-on discussing million thank of proceeds like $XXX our raised financial participation results, our during for company. to their

earlier-stage and believe of recognition of and from the we as that the with the these MyoKardia’s sufficient with we But pleased to value-creating to progress which only operating significant our investments at and with this you balance will mavacamten, made We’re into our plans financing to collaboration and with expect XXXX, current ahead. wisely XXX date fund but have that with have funds not our opportunities also as Sanofi, have progress we With fund commitment use anticipated cash our desire point, with also to to efficiently. well programs. payments be

numbers. the to turning Now

quarter release press quarter Our with the quarter are operating and second second of results in XX-Q XXXX. million, SEC. Revenue the compared in the with $X.X million was afternoon’s filed detailed this in $X

XXXX, first the For in we in $X.X compared million partner months six the XXXX. same $XX to of recorded during revenues, million period

inclusive sharing $X.X revenue credits, and an our We payment, overall research in related of million R&D million as the quarter the and preclinical expenditures Sanofi with in an which $XX.X recorded the to and from first-half, related second-half expenses. progress have associated XXXX by second MYK-XXX, were continuation the the mavacamten been advancement program. both costs million of for $X.X increase the $XX.X will as this for as for well cost the with were year. first-half $XX.X over pipeline deferred reimbursement revenue million, year G&A $XX.X to of and Sanofi million million made expenses have remaining driven R&D respectively, mavacamten and in and and The over the the quarter million the for G&A expenses headcount. $XX.X be increases prior in increase

programs, million, first-half. the expenses in level, by XXXX and our for across of per expect MAVERICK Net $XX.X activity million, operating for was clinical studies. ramp the $X.XX loss share from share the in quarter with primarily our or We of $X.XX EXPLORER second-half particular, will first-half per increase driven and the $XX.X the second or

start June we call Tassos compared XXXX, we now the investments, ended to Q&A. before the with year. I’d of open like of lines few the turn million As the back at $XXX.X Tassos? this cash $XXX.X quarter remarks in for and a to XX, for summary to million

Thanks, is and portfolio incredibly Taylor. advance first study. clinical as time drive completion and towards exciting an we our This our for our expand of pivotal organization

stream pipeline to out four XX a portfolio. Looking next you across expect across can steady of to along with months, momentum increase XX the milestones the our

initial expect year data readout we There patients extension begin from and a long-term support periodic mavacamten, regulatory filing. that the mavacamten’s open-label related long-term extension will For continued from trial to planned be into enrolling EXPLORER thereafter will MAVERICK an to of our next use. with data PIONEER reporting early

benefits We early EXPLORER where readouts timing from insights will non-obstructive at options are MAVERICK, characterized in update in of as potential inform any, to non-obstructive data the if have give provide XXXX. expected diastolic Those mavacamten’s into and HCM, data well an for as are registration few, second-half path us additional overall data the in patients. in as time update both for We’ll next and dysfunction, diseases by there as HCM for that year MAVERICK studies. on enrollment an well for

data longitudinal start discovery In recently data a ascending looking we’ll a end later the SHaRe clinical Xb busy from we XX trial accepted before X third multiple within months. multiple trial has homegrown publication including publications edition seminal circulation. into or second-half, pipeline, forward a Phase And year, Registry finally, at in Phase earlier our an X Phase a to next presentations share that look this study current and For paper our for candidate so our expect advancing the where to of upcoming and been the for from we’re the of MYK-XXX, year. have looking dose

been other that accepted from Annual the Association team describing HCM disease Heart SHaRe burden presentation research for Chicago American of also November. at received just Meeting abstracts aspects this word multiple Our in have a

models it describing addition, data cardiac you also of presentation HCM the We forward In HCM several function discussing for abstracts to with AHA. available. patients novel in genetic at this and effect mavacamten on have publicly been preclinical as in accepted becomes look of

and upcoming ongoing and our discuss up efforts New advancing initiatives XXth to research new I’d and pipeline we’ll now currently and reflect here as our – details October in clinical just our for that We’re with York, to broad June commitment share where planning our programs emerging And candidates the open questions. additional Day on treatment of Marc are and our a Marc to and the colleagues understanding me call reminder, Lee cardiomyopathies. like and with the me Jake Bauer. Taylor, R&D

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

is open. line Your

to I Can my ago. at obstructs a taking Thanks one Maybe non-obstructive for you’ve the of on for almost obstructive proof-of-concept study, afternoon. HCM. good readout. the from But out you from you that are talk you year for a HCM level the ways still learned non-obstructive based Hi, us Based confidence? questions. your got chances success on on thinking results of about know, study? for what how it’s about the

Tazeen. Hi,

not about in and two excited heart the the have different registration of door MAVERICK a then impacts how potential diseases also reason will – of by are it a for diseases in number we’re feel and are help underlying The what is etiology is or particular which these twofold. and the give folks diastolic But same. a non-obstructive forward for confidence mind, We’re it in clarify of But that we that for dysfunction. our this HCM primarily to in opens driven the kind path potential relate good patient muscle us segment the keep improves to for disease heart up then mavacamten us potentially and study genetics only reasons, the compliance, diastolic indication. will

contraction motors in confidence heart force beat. know be mice force to increases seen important PIONEER, the the of an squeeze we’ve the reduce contraction, we the force to ability several gaining all And of and contraction in mutations that the the has reduction in led of which particular, clinical each benefit. at that excess now more has it’s obstruction So about with to during causing in engaged overall mavacamten

So Also program case correlations there want is to study to which really as order in that proof that that biomarkers there’s the mechanism, understand those we to etiology need biomarker the the other to and of of and since nice remind that, that, will we’re to similar do, goal non-obstructive for how be saw to contractility. certainly we have patients, we study Things our use to we really given measurements patients. believe identify But in but in we don’t obstruction HCM have clinical studied PK/PD in to non-obstructive X patients, dose reduction properly folks are reason need the a there identify there. appropriate benefit. those to and looking ideas Phase

explanation, obstructive for there independent? much pretty that the get they obstructive you to And that anything for like, an Thanks or comfortable Tassos. was just see to – they or always more in with were results study needed

I intention study always disease but really think, non-obstructive are benefit affects the the clinical the of saw was patients, in and are kind patients PIONEER. these the same. our provided how in differences the to There we evaluate underlying drivers

for And really this in at so patient mavacamten the population really like have many treatment obstructive studying don’t options group us worthwhile it’s who all.

much. very Thanks Okay.

Thank you.

from is Martin with Our Credit next question Auster Suisse.

open. is line Your

that Phase reporting retard my Thanks was consider Thanks. an metrics you of kind on clinically for eye keeping could what out which MYK-XXX, review should and we be if you for I question. taking Xb the you’ll us be Yes. when relevant for? wondering On would

question really that. appreciate Hi, XXX, on really first talking be all, to we’re of Marty. happy about your so Well, I so –

Keep really we area a drug We’re potential. new the in got the is targeting approach around of in XXX of we’ve a discovery we’re enthusiasm DCM with that therapies excited lot is taking of with mind, same mavacamten. cost the disease and by its approach that XXX. and terms took needs pathways

its that view. of we that confidence in XXX’s So broad we MyoKardia a the data we will that and Certainly, underscoring around start potential. addition the the really seeing similar DCM gives potential to around platform folks hope at sharing the second-half us in have approaches are that as therapy, here share year

with specific data questions up. I’ll more let about So June the answer maybe study that, in coming the

Sure, thanks.

during So tolerability. really for primary as earlier the his Marc establish is indicated and of to study goal portion safety the conversation,

PD that you’ll PK/PD So have to defectum better is as ventricular that. then certainly by secondary we about assessed understanding understand bunch of goal a function. be hear better, PK And at and

are that on. probably important out things be So most we’ll those reporting the

Is thresholds forward clinical there to to look forward decisions? highlight move any of in want you’d of relevancy terms

I considered speak number. A necessarily we Marc don’t we that – that PD are elicit clinically to to But and useful. sort have response And establish able of are number to looking perhaps that’s that. that think, we such can varies

Marty. hi, mean, I Sure.

contractility and We’re these going echocardiographic will increase of be to detrimental in optimally want And again, associated cardiac any output sensibility. with indices see at be patients, contractility effect that looking improved to without on diastolic we function. in the an

Okay, queue in hop for thanks. back I’ll further questions. Thanks.

you. Wells next Thank Fargo Our from Birchenough question Jim Securities. with Instructions] [Operator is

Your is open. line

Congrats guys. all Hi, Yes. on progress. the

see in myosin taper maybe engage heads, on much How have what miles positive contrast and to you Just MYK-XXX. for between bit patient but I a you question more I’m to of variability? second maybe And traditional imagining too relaxation. of the impair a MYK following not for or see bandwidth Could we’d you what on then, walk just interaction the drug it’s versus typically much with actin. guess, preclinically inotropes? up a

to maybe as Thanks. speak So could you well? that

Sure. Hi, Jim, good afternoon.

thinking about other things like inhibitors oxymoron dobutamine, traditional So inotropes. first, the and and example. I regarding positive question you’re presume milrinone your PDEX the about and

heart I’m of cardiomyocyte. triggers programmed So transcription also heart – It’ll rate. works. that death increases Cyclic number a lead These to remember, It’ll cell in does It improvements improvement upstream may also sorry, actually lead apoptosis. of leads to a an the eventually to XXX they work where in fairly in Amp agents in contractility. mechanism or increase far

not the see preclinical XXX. we about Now an specific we’ve rate studies in get our that, increase heart to data, do with preclinical what is in seen

clinical are that used see are ways care. that agents of with these dilating the of for don’t standard effects associated traditional We also

the these that heart getting going our question disruption, Jim, part we’ve your In we’ve you’ve seen be is of of there, to with out that contractility with program the – able important. I mavacamten, think – And point been always diseases muscle you second balance. for to find the as balance

we’ve we targeted good where to agents, here Phase they’re diastolic feeling and Phase we’ve they’re balance of in got a where is the point these you relaxation. have got X because much drag all X out a may finding in going doing. too provide about what do And that think, I and work engagement of myosin as

So window studies got in been From able forward. we know to got comfortable a DCM We’ve standpoint, cardiac of improvements which to order we’re the we’ve the course, get we’ve human looking in the in at look patients effect get designed us preclinical to to study output. help in subjects a with But, there. same.

add to prolonging actin the and between just interaction not myosin. on necessarily we’re And to Remember, that.

contractility heads myosin increasing engage actin mechanism through more enabling with that just We’re and to

good Really point.

the Maybe open-label and just one follow-up quick on extension. PIONEER

ahead data how many update total over? the do look we from patients transition How expect extension. to PIONEER were And extension? from many transitioned studied you from the have As that to open-label XX to previously, that open-label

Yes.

have enrolled. far So XX patients

have a enrolling. few to expect We more

for And us, XX goal Jim, us. the for is

effects the a coming of potentially use able – of around conclusions start We’re to months to data chunk and so some to really mava. at long-term decent draw over six draw or look now some

taking for Thanks questions. Great.

bet. You

Thank you.

is question with Credit Martin from next Our Auster Suisse.

open. is line Your

miss guys, me. Hey,

Hey.

titration. and dose something to want just on EXPLORER I the clarify

the of in then working kind off rest of off choosing would kind above going was the that gradient? PIONEER dosing same that study, that it’s XX. conservative the in dose curious one be by rest mentioned wonder doing a method above I other collisions Thanks. as to I – or provoc gradient is I’m follow-up driven XX, which titration And measures the gradient variability curious about to Is you’re how provides profile? that two the of whether just and understand. of or the those off You be, in more I

Yes.

going I’m I answer and I’m the I them first to try the then sure you to may to part. all. part ask second not got repeat

Certainly.

rest thought and, for –yes, So SRT. are which determining at provocation meaningful in XX at utilized for fact, XX in eligible patients threshold obstruction the all so be or to are are

use. those care and sort And who would of for of your our correctly, numbers there They And HCM. stated really I the think In if both. so read taking they every a and part with very clinicians that question resonate as which study And about, question, resonate to very every using need well you then they go sort second assessment supposed to procedures is and it’s reason well patient that again? same do go. the through of go those threshold. no can site before to through they where the And of repeat was do set assessing of are is – I judgment

for… to a the the was – of the other in one provoke have measure – I’m the off rest asking or understand, of the you in measurement just, sense in rest gradient gradient reason of of it the Yes, measuring variability good working was measurement trying does gradient. And do versus just

Right.

titration, physiology from of other, that perspective? profile the or dosing to about more that a way conservative one curious does dose do …controlling need just

ask as about the be maneuvers Yes, calculation. bit Valsalva a more. I’ll Right. mean, assert that of the this vary little study and gradients as power variability I such. depending status. Marc in And about vary the on designing about I think, anticipate can But we some these They talk to variable, we they depending how progressively on can They about fluid know.

So yes, some that’s but exists, do variability well understood.

So – hi, Marty.

I Just say. cardiology flavor, a bit to of add shall little

instructions we’re really EXPLORER, maneuver of provocable gradient. the And Valsalva to the the in be – are used to the whom will the how know experts many assess our and Valsalva. So to details field investigators, do in as giving and

both and gradients. And gradient. means found is fairly actually, the do out Now accurate of done, in the degree fairly a PIONEER, of exercise Valsalva Again, Valsalva the high when provoke to bringing a we between in did the methods. order properly agreement two

want is the gradient didn’t look to addition, have rest patients are of Given through the going to during they this studies. to clinically, in the Valsalva perform And number exercise may in at this and we of to have two clinicians yet more drug. They’ll how exercise. EXPLORER, put through titrations use them

you Thank much. great. very Okay,

Thanks, Martin.

session that the Gianakakos like Thank to call you. further to turn over our conclude any for Q&A today. remarks. does Tassos would for I back And

today launched the in time where Well, your good we’ve are our mission. thanks, studies this everyone, for feel your really MyoKardia of for quarter. that about continued support and We and we

EXPLORER in building Our confidence is start. high really and a momentum is off the on design of great study hopefully, it here the to great and we conveyed we’re we’re

to be the course R&D of and MyoKardia’s keeping We’re Thanks, able a all also XXth therapy, of in forward to you its upcoming power to again provide but of at really of approach. many you throughout the hope color year, the share more We Investor not this to look DCM progress and highlights again, our breadth to around some as second-half and informed Day. potential year October XXX, really see of starting to our which Conferences only the thrilled everyone. at

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