MyoKardia (MYOK) 8 May 182018 Q1 Earnings call transcript

Good day, ladies and gentlemen and welcome to the MyoKardia First Quarter 2018 Financial Results Conference Call. At this time, all participants are in a listen-only mode. Later we'll conduct a question-and-answer session and instructions will follow at that time. [Operator instructions] As a reminder, this conference call is being recorded. I'd now like to introduce your host for today's conference Ms. Corral. Michelle Ms. begin may conference. your you Corral,

and and [ph]. Thank introduce also Tassos for MyoKardia's Officer; website. focus portfolio. Marc Gianakakos. Q&A and on today's Taylor and June Communications and quarter Dr. you Development for On will this Semigran, pleased news Section our afternoon us you, Operating Officer. Head will is for Officer; to CEO, across A Corporate the Corral, Bauer, Chief Harris, call financial Medical Didi today's Investors regarding on who joining Michelle Media of can today's Business thank am Chief Chief be Leading Officer; we're our Chief release Good joined recent call. activities updates MyoKardia's Jake found Relations. Lee, Dr. be the Investor call, by first MyoKardia press of results Today's and call I MyoKardia's Financial on

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

Xb of or productive across start the advanced year, with in we've joining candidate our clinical into Myokardia now and this beginning the the trials for pipeline cardiomyopathy DCM. year Cardiology our of in indication. X Phase six at We therapy a trial our cohort Since MYK-XXX in PIONEER second X HCM company our initiated our and HCM protocol, Scientific from patients data everyone, mavacamten working the a study making and been we've obstructive trial Good Phase mavacamten mavacamten finalizing is alone. X us to MAVERICK dilated for second today. which clinical progress thanks EXPLORER-HCM in our obstructed with It’s American poised clinical are research Session. exciting months In Annual afternoon, pivotal few Phase the been Phase of hard College non-obstructive from of We parallel, on HCM. presented study

agency's once the these productive. been received have dialogue the next FDA writing we meantime, which few plan we in expect we along has lines feedback very an providing We In Our remain in update with on the weeks. underway patient are visits our second on held the well was track or taking quarter. recently in IRB dose and board processes to Participating in are place participants. meeting first investigator the approximately with clinical XXX review institutional EXPLORER site the

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MAVERICK-HCM Phase to the recent highlight two the begin on for as MYK-XXX study start expect the year this MAVERICK. in patients to We milestones EXPLORER call complete and Xb X I DCM. dosing they today. of The and of wanted initiation study Phase the

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talented our clinical organization. leadership continue we as and research the to So advance across we're pipeline, adding

to first GC candidates two as of update and companies and bringing them the to additions to and as with Cynthia experience CFO. of team closer recently Ladd Both now, make we with our commercial and Taylor companies, the to as organizations other evolution quarter he General made key with growth contributing great comes hiring substantial quarter functions Counsel provide their at as to navigating most Cynthia This us experience additions where entities. to Taylor on Harris as handed as XXXX. roles. results I transitioning commercial her an for as serving biotech Most approved over Taylor we MyoKardia’s recently in business to device will regimen. at stage of finance well to bringing leadership targeted Taylor? both two CytomX, Morgan significant before in cardiovascular public patients. leadership started financial medical public his with career products oversaw J.P. Cynthia CFO who our Taylor's move finance

the am excited jump let I impressed innovation precision you, me Thank to have of passion say here just the and Tassos. into science how cardiovascular team And disease. and been in extremely medicine MyoKardia I've to the before numbers, joined MyoKardia. we bringing by

and we of our late few clinical our in stage programs As engine forward And continues the drug as to discovery new mentioned, significant there drive produce XXXX leads. been has development. activity research as first months insights into

first Our in net of quarter organization during the to first of the is infrastructure research support approximately pipeline. and Hiring to XX internal focused continued our building since the capabilities our with the hires on year. maturation new clinical grow

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detailed are release SEC. afternoon's in with XX-Q filed this Our press operating first and quarter results the

to by commitment investing of driven open and represent period had with of net our an over equivalents that we As quarter support of ended the $XX.X turn pipeline XXXX, product arrangement back as Revenue XXXX. loss $X.X cash first to with cash with for line investments. the $X.X million and inclusive payments continue summary million and same Tassos is was in we for while fund the I'd on team operating development, were to Tassos? net the the Expenses credit, expenses to the increase related mavacamten the our XX, to were anticipate year headcount and Based quarter that million Sanofi By late in the Q&A. the XXXX first March our we planned first in quarter $X.X expenses cash a million quarter company and cost maintaining last investments. XXXX. the our growing before our million million, few in compared million organization. operations investment million $XXX.X into our in the $XX.X or per share. anticipated was stage for plan, a R&D program. the quarter XXXX from G&A current research together cash, our will like of $XXX.X in $X.X we Sanofi quarter of of increased sharing with comparison, first to in remarks With a advancement call $X.XX for

exciting our in company's track a itself organization. really Thanks, year, that incredibly this Taylor. is of. six is initiate This on big study Myokardia’s time trials first clinical we're proud all our including and an of pivotal for milestone to which

you next can portfolio, a along across XX the XX Looking milestones steady momentum to across increase which stream out with of I'll to summarize months, here. our pipeline the expect

receive the expect update milestones the study writing, following the second study design mavacamten, and the For coming final EXPLORER-HCM the of quarter weeks. the four we we’ve an in feedback in initiation which once on in the again FDA’s pivotal

from use. extension give open periodic mavacamten’s Second, the readouts into label PIONEER insight to data long-term us

which non-obstructed they those where EXPLORER characterized Next more dysfunction, if second we MAVERICK. data based in diastolic extension, mavacamten’s expect are path will finally, or potential of registration about there data and from And learn initiation much to on in EXPLORER be the to to the HCM half patients. the long-term complete XXXX available non-obstructed choices to diseases of benefits patients the few either MAVERICK, for by finalize a to any HCM

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publishing compounds, and include and data from our homegrown clinical the our the research into and our further into in and insight pipeline Day sharing journals in the of advancing advances understanding on on XX basis, of disease reviewed efforts Finally, year. digital more XX in of mechanisms forward earlier within third a screening X peer regular application candidate study and key health improve and our cardiomyopathies later looking our information disease and months technologies clinical novel We're at next programs in to our major meetings R&D milestones to research providing to management. pipeline Phase discovery medical of the

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

and with, me here Jake colleagues our Lee Marc June reminder a Bauer. and Semigran, As Taylor

of Ritu [Operator from first Cowen. comes question Baral Instructions] Our

you the the non-obstructive Hi gradient MAVERICK gauge dose Tassos, titrating are life. everyone, that in that taking up patients and in are is you titrated thanks or this in if patients, there how no to for case doing question.

going it's Tassos. Ritu, tolet Hey June question. the answer I'm

really a That's good Yes. question.

As you have patients gradient. know don't these a non-obstructive

really we're the not are in way, to titrate obstructive. able to we in So do able

patients, serum placebo those in two – cohorts these a have and mavacamten three range. active each target are cohorts, certain We we of then cohorts

in So target no the study. targeted. range being of concept it's being really yes, involved just proof this particular There's that that is serum titaration

that Where from the range derived more the into are PK/PD serum data is there or went PIONEER data it.

Yes.

of parameters recent can can some that I and the and eliminate population other how some where – benefits think that also largely was see on start we that's gradient we see eco where PIONEER obstructive derived to data from so determined. in

in one the of our the Ritu add I'll key reduction pharmacodynamic June's is blood mavacamten. to in to effect – we're to respond getting really the responded contractility, thing correlate of – the gaining confidence form just ability levels what we're

X and preclinical transcends as as program we're what Phase seeing that our well Phase in So X studies.

informed by really linking activity in that's – course it's we measurements So as of important platform. started to pharmacodynamic And that you that clinical PIONEER, saw.

of that we all patients. us So for start non-obstructive helps where these want understand to

how from MAVERICK, other population we've just the we endpoint this thinking efficacy then the evolved previous And about there discussions Got differently pVXX should it. or all just be that data? your are about PIONEER thoughts measures efficacy have we at and of they think either in as same have of had

Phase indication really for us us data provide this to Phase a the at characterization Other X. are this as like a that that meaningful Phase we are X. go going pharmacodynamic quality primary proof to to study. going This of might Other going things endpoint We're life where be So to endpoint and some a X you us that include things be know other forward a and this concept there's PDN, to predict also tolerability. may see pVXX to is whether is just of things – like go going effect to looking benefit. safety for that really NT-proBNP. with be of have with indicate It’s PK LVEF and

taking question. Thanks Great. the for

Ritu. Thanks,

next question Credit comes Marty Auster of from Suisse. Our

Hey running guys, up counter of help for don't you this question. be of for EXPLORER. difficulty the final bracket know you've kind meeting inclusion thinking my potential and the – in and a sense of to And wondering But us of that Thanks. was are the length you'll of taking number you're activate. of criteria patients you're terms now. quite trial sites right kind terms thanks getting we in got if sites any parting for what could of or maybe looking you and what Obviously if expecting timeline total I plan,

Marty. problem, No

in our patients So we're about study same going We're we've that overly here essentially concerned entry that fit find studied the criteria. patients are to PIONEER. to that not the ability patients the

study the to in study – of give lay excited them. we XX get the in so the We we as of engage out more coming want the as in of – Excellence that So or weeks But will there's roughly of terms here every design. on the underway. information study number Center U.S., to so we're major sites

call feel EXPLORER. that between the in between pretty that So XXX and to patients XXX end, and going for is mentioned I be we confident

So the reasonable in confidence. up to a enrolled able that running all be We us get should study and timeframe. of gives in

Yes. a provide I more the around can site that on little engagements. color

meeting want we the patient this of great of of just just as had really the mentioned our there of of our in treat Centers further we in Excellence our So country, U.S. the about Tassos site investigator XX this just are of dosing study. to the say point majority beginning almost first call, I to this anticipation centers, those XX adults in engaging all where quarter. them,

of on share engaging that details also we'll well. in are as and a future more Europe We number in the sites

patients in what across If those HCM being are patient I could remind many could follow-up, us roughly, of one centers looks followed? XX that mode like you of kind how terms

X,XXX seeing of Most a are hundreds. in those There's seeing – are patients of patients a them over range. wide some some sites Where centers. those

Yes.

you. Thank Okay.

of Anupam Our JP next Morgan. Rama comes question from

is and evening. you for updates progress here course guys, the this Thank taking on our this on the [indiscernible] for question. the all Anupam of for and quarter, Hi

the us meaning first So Phase the that benchmarks curve this DCM in I tell patient second accepted guess you maybe from using trial one since the success year. Xb dose for February. that would warrant had enrollment patients worked in on of And moving could outstanding ascending and if design. have HCM the here, how what tuned half on fine the anymore Maybe dosed are data which pivotal so the one that up PIONEER of an – what’s to is needs color could trial guys. the next trial up Phase design, Thanks then forward. be now on on can the in you but I provide the much, And update more coming is X

the writing. where Sure. design we Hi, about appropriately on feedback just and to Tessa have until [ph]. FDA We're with that from be want pretty and we comfortable the cautious sharing are we

go ready, the investigator sites getting We are getting here all the all IRBs in the approved, meeting moving underway. ahead systems operationally getting

to this So the here quarter. in dose we're ready patient first

we're study, activation a sure. appropriate here and it's which to gating making of right, look Phase study to range second the X significant to half study, the the about which will that is systolic without for Phase XX% this is happy as us it your in X – say and anticipating understanding this We're we're at to starting allows relate year, from at with tolerability going the that all neighborhood Xb a study signal, dose us the key through in the PD on safety recognizing of diastole. impact relates items question, let's any MYK-XXX As enable safety get

recall, we study same want effect, we patient done similar kind And already had replicate signal can and of is what the make just demonstrated to that’s sure where you we we healthy study as in in the that about. PD

update Thanks the great. for Appreciate Okay, guys. it.

comes Jim Fargo Our Instructions] from Wells [Operator Birchenough Security. next of question

Hey, that terms the question a measure for guys. would to what envision non-obstructive similar in clinical there for Do best of echo into you DCM, Ritu’s theory And a that measured best is is also you and with might related think I clinical the guess taking a progress. cardiomyopathy. VOX. clinical correlate question question tracked would And be you're in of translate benefit This will then looking might of question. that peak it ventricular on the that benefit. at relaxation particular have Congrats measure all Thanks the for benefit? terms be you

marker terrific all Yes. patients, looking changes Hi, at non-obstructive be going think exertion of a indeed be to related with it hypertrophy. how it Jim. When going really I the and to improve wall of BNP stress with MAVE that in LV to is certainly patients function. biomarker wall which with the do with to Well, hope diastolic in HCM – gets to terms and is these the stress exertion, we

we terms as uniformly pressure prime velocity angular LV this filling and of measure look capitally echocardiographic want at we early micro the recognized time. happy I E – in in micro call velocity detail at at In any to to over micro look and it fairly, more assessments, to discuss flow think E of certainly want – peak we the

of these some life. were And signals certainly well. start that we correlate parameters hope that hopefully, peak we quality measuring mean will around with and other And proBNP as including VOX I get to of the some echo things

add. Jim, thing I'll think, one I

looking that We accelerometers. VOX about, hearts also blood What's capacity it's peak about are ability restoring the measurement. you measuring at at think the peak exercise interesting a oxygenated really When about is, what sub is maximal to care is exertion, get patients useful important.

it's thing here. doing start submaximal we if exploratory we're So we some that understand exercise can, if an with these can we feeling wearable can of reliably devices. But

be for it'll think the I really useful ecosystem.

pre-clinical in of a being we're and have diastolic systolic question as than is MYK-XXX from superior what other diastolic other activators I say benefit filling are our ejection there feeling that with studied. going believe to the year get we of looking at less of conjugate To and And data molecule contractility affects currently it's course, of that our our your in the terms on impairment time. function, agents is diastolic terms of the duration

that then be or need do to And something initial year. envision extension, wait year think on this any the just we would for would maybe next getting you data PIONEER you

on day update to but that. We're we'll ready speak. any Jim, kind you kick of so Too here to a soon study now off to tell,

on, months number six this a initiation, update let's treatment have get decent we the analysis. we patients, get with handle of a what good So of when we'll might on say

us will actually patients know patients for in are provide moment actively don't it we just speak until by happen. have how went make many one we to screening, data to As updated meaningful started

might market HCM you then to one final that now? allow population patients. a that you in are try if And pretty be just doing maybe seems of What terms right of it me, identify going just there's underdiagnosed and patients undiagnosed development,

a and have about shown there mutation for seeking the are of the medical Americans, at research least XXX,XXX to What others say, diagnosed are pathogenic So in of are about HCM. carry some our of is HCM. sort who research so system, attention believed them XXX,XXX or let's that

to disease? we really So mavacamten a of know of community. of looking get where Because itself to And want be to they? going Jim know and well. likely them is really the in used. first a we is XXX,XXX, as that is Where at frankly What group that's group a not the the there's to that be patients other lot about are level their

at for with as gauging both in exciting wearable are applications least over about an not of and can genetic, measurements So screened example at up, screening this ecosystem been pick some that correlate obstruction. on Apple MyoKardia members broader certain echocardiographic here as who particularly last or terms efforts to underway, There’s two well think of AHA involving that increasing at have yet watch technologies we’ve diagnosed. that can degree year published for family a

look there's picking watch. population. up you've apple to an population, of which have of with do AFib, of get – have So into lot they're to pattern an can from much bigger have folks seen a here a general And like course we to their the what get it, to the alert then at alert the bigger watch where opportunity and physician swaths people Apple

there expect be. to yet, just on managed couple working are important blockers. driven but them. to many I patient very, very those get going to to standard those of being extent the So have of much? answer patients symptomatic will HCM though which with is beta things adoption emphasize the diagnosed by do wave that of want don't be We of we're are How XXX,XXX, is about, there likely which beyond in But undiagnosed patients it. that thinking we're to How the that care we that first essentially poorly a old population, the

Thanks for taking the questions.

the Gianakakos. call I would now turn like you. to back Tassos Thank to

today and continued thank to the time everyone year. pipeline keeping MyoKardia get underway look for mission. milestones study EXPLORER the many months forward across our the our we of your to you you of coming for and Well, you. support [ph]. progress your we We Thank the as achieve informed our throughout and important as Didi Thanks, in

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