MyoKardia (MYOK) 2 Nov 172017 Q3 Earnings call transcript

Good morning, and thank you for joining us today for MyoKardia Financial Results and Corporate Update Call for the Third Quarter of 2017. Leading today's call is MyoKardia's CEO, Tassos Gianakakos. Tassos is joined today by Dr. Marc Semigran, MyoKardia's Chief Medical Officer, and Jake Bauer, Senior Vice President of Finance and Corporate Development. A press release of the company's third quarter 2017 financial results and progress. X:XX at available became p.m. updates afternoon. Eastern Time this to This section media on press can be releases investors of and found the website. MyoKardia

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

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patients drive severe and of dedication from diseases. mission data suffering further towards the early, us of team's peek cardiovascular the mavacamten’s still gives and lives from changing is While a into strengthen our PIONEER potential our

and sooner Phase the extension Since originally updated planned. we've long-term the we we discuss program an Importantly, symptomatic, with opportunity the with had obstructive provided HCM PIONEER on to than and EXPLORER-HCM to focusing registration met trial move study. you mavacamten’s last into FDA results X us

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Turning to MyoKardia's other pipeline programs.

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to Thanks and good everyone. you, Tassos afternoon

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can class patients difference being work able of disabled mean For the even or improvement by their disease. to one between being NYHA

been Our of has move suggestions. FDA, study FDA a the with input implementing commitment to stage are from patients in look We dosing We their we development and advancing XXXX. never the dialogue as towards forward stronger. we in second this quarter expected mavacamten received EXPLORER continuing expeditiously the through Phase late now to X

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first quarter planning data to in of XXXX. cohort share are that We the from

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dose us will lower understanding to potential mavacamten beta assessing cohort of of an of any mavacamten impact addition a this give blockers activity. In second on the if

echocardiogram. tend need characteristic from at of estimated of the with the believe I awareness We this their exercise transplantation. for X hearts or as when reduce also from that fall left hypertrophy suffer more either all they chest obstructive of do diagnosis no- the with as obstructive or severe have patients are NHCN. with of increases. number life also of Phase counterparts cardiac differ and one can another they show Their patients is and on into ventricle, HCM with pain study LVOT progress HCM, this HCM like Patients may These thickening a and in capacity. not starting present They category. obstruction later in patients to It the to breath, HCM disease its rest HCM compared the approximately OHCM not exertion. diagnosed walls MAVERICK-HCM the grow trial clinical non-obstructive patients second indication, third OHCM XXX,XXX shortness

designed genetic common clinical and us defects obstructed with safety biomechanical between of non of HCM study population. to in an the assess will and impact first an investigating this to endpoints the of mavacamten XXXX. forward response in OHCM population. and Given look We and the obstructive certain commence provide understanding look will this quarter is HCM, efficacy for mavacamten the we of in safety, but at dosing dose anticipate also non X The Phase trial in

Association that XX data will mention the of at on Sessions Scientific to November occurring myocardial American upcoming want Finally, I XX. presentations several Heart have new researchers through

from I mavacamten burden is elucidate in flow will of activity to our to is turn now precision fitness another dividends assess to breaker are medicine Jake of mechanisms people in ever a the data device One It it further of to science in myocardial to than preclinical a to of trackers disease the late I targeting be similar oral how these basic use describe of that cardiomyopathy. cardiovascular that on millions our more novel of see With to the will excited non-invasively wearable In worn the will wrist the approach the the financials. presentation discussed. digital of by MyoKardia’s the that, the abnormalities studies over yielding am discuss presentations understanding blood session. HCM unique heart.

the attractable million cash equivalents received the quarter offering $XXX.X in Net million compared the successful as December $XX.X completed Thank million. XX, of $XXX.X the position stock third you, company's ended of investments The with totalled cash in million, August. proceeds increase XXXX. follow-on Marc. to X from approximately shares of MyoKardia is of to offering common cash, and

from to XXXX. was third million anticipated million, we [ph] $X.X XXXX. with Sanofi, Collaboration Taken together the able fund for revenue period our being license compared collaboration into receipts operations same least the and with quarter $X.X at in anticipate with in

September costs XXth months revenue of X to million three the million, The the September three $XX in XXXX, period expansion the from in was X Phase XXXX. Phase the PIONEER-HCM ended XXXX. in for for months period ended the collaboration XXXX, R&D studies, $X.X $XX.X increased collaboration clinical revenue research for same of XXXX. million primarily biology study and $XX.X to including programs. first is recognition attributable for XXXX payment $XX.X for the first in quarters compared chemistry for MYK-XXX, and and three driven to X $X million the ended XX, by plus same Sanofi. and R&D the expenses XXXX our were million million was and expenses and increase expenses nine $XX related discovery expenses increase in period received the license study $X.X million to million of compared preclinical development for were same development for of up and General MyoKardia’s expenses Research continuation months the to contract compared the The ongoing the For XXth research, year clinical mavacamten XXXX. in in administrative million, $XX.X staff period for same the September months were from

per $XX.X $X.XX loss $X.XX share the the loss same was $XX.X $X.X Net ended recruiting to million loss in was for $X.XX the XX, attributable million of to For in as or stock was expenses. or September The to period XX, in quarter were million increase And million increase attributable primarily noted per compared Net expenses the operating for share Q&A. above. in nine headcount, the share for change share XXXX. expenses compared or G&A Operator? in compared months third per to marketing to period the and expenses ended in the to same XXXX, employee to or a with well September slight that, net expenses, open $XX.X The the XXXX. $X.XX third nine $XX.X increase per like XXXX, months an million quarter XXXX, was call $XX million, as XXXX. of I’d for G&A for compensation primarily

Instructions] question [Operator from you. Ian Thank is BMO Somaiya First of Capital.

Your line is open.

- of a maybe just the as all Phase just coming And and clinical you. couple X to to progress. me let trial. I was hoping on the regulatory congratulations points it clarify just say relate Thank

that Is mentioned the Marc, delta that. XX% you First a clinically. agrees to FDA XX% the to with? wanted [indiscernible] the Just also significant to – of definition clarify being that

as Sure. threshold. function. is The as feel a I of being well think FDA I by how agreed an represents The patients and it primary described And that generally with upon an they pVOX important how pVOX clinicians being agreed on significant magnitude acceptable endpoint. impact

beta in just X that blocker pertains as to blockers, the the know combination that it of And is the mentioned expect we that Okay. Phase plan I previously? and you've should was something beta studies.

of you year. this know, the not next into details are that X be call. We'll portion first our we probably at Well, the Phase - into study going design of doing depth in

do we into EXPLORER allow who going continue However, beta to plan are to patients blockers. on

a Okay. an question pVOX. to ability terms And more in some of beta blockers have last patients it just of beta as relates the to shown blockers, benefit selective

we guess, expect beta Just PIONEER study, the the know, blockers - if curious the and patients allows in on you trial? it the the cohort that are for Phase I X then of if type should next does what limit

the cohort beta a sorry, blocker. we did Ian limit the In blocker PIONEER B, the certain not I'm used beta clarify. to Right. in

So the both at a selective not beta that used the think that that time you probably and in are I this we in spectrum blockers limit there are EXPLORER and non-selective of beta would type used. blocker I know - think and that will the the community of

I'll right. guess, back Thank queue. All I get in you. Okay.

Suisse. is Vamil next Our Credit question from of Divan

is line Your open.

Great. taking up just some with of the on discussion FDA. Thanks my questions. so much Also the for following

the then term mentioned long phase. You EXPLORER-HCM also the extension and

of like? terms what Just numbers application, terms to understanding up of get just you would in need curious have safety you of clear patient the what when database sort filing look if mean the the of a follow to you exactly in and of do

Hey, Tassos here. Vamil. Its

planning placebo you do patients. So those we'll the But double extension on we've enrol on EXPLORER to We pieces details as in XXX study. you which get estimating about roll the is Those we like are agreement and endpoint, on blind to be more giving intend to registration primary to the we're tell well. to in trial been XXX addition long-term a the study major allow patients remaining elements as key we starting to guys EXPLORER is closer can a what controlled, into the program.

question, before, will low that bit think but looking going sort then the this a we you And Okay. second Is of my anything cohort talked cohort from about just on little dose before looks in you're starting? there first I It waiting now. design have for the around quarter you second in your about difference that cooperate one a or for and then from final starts? may the EXPLORER-HCM that like it one that

lot Yeah. We're whole necessarily not more going talking EXPLORER. the today Vamil to to be about design a

to fact incorporated here an us obviously the up feedback we're the we're and FDA going cohort think - B. I That from incorporating time to the that we gain giving opportunity that converting. ramp is have

always we'll of intended So incorporate thinking as into certainly it's we're all data it the subsequent generating study. our the

question my Thanks – quarter. All this and on progress taking congrats the Thanks. for all on right. Okay. also

you. Thank Thanks, Vamil.

with Birchenough Fargo Jim Securities. is from question Wells Next

Your line is open.

the and progress. congrats on for Hey, all guys. the Thanks details

Just EXPLORER-HCM. on

second is of then the to agreement over long- to that cumulate question on there that just that efficacy just is extension is FDA extension, data side or term from wants measuring your follow for Just XX pVOX timeframe the this might And you or something FDA week safety on time. that sense want to it study? specific wondering have the see longer the a do period on is

take that I'll one. Well,

than not for to But is of terms placebo. that EXPLORER, week campaign the compared of efficacy show terms In when a XX in we're more the intention final of going period pVOX duration think again, of to our I details. to into mavacamten go

again very what's I patients as fairly to the to extension function here, that structure mandates mavacamten. did and take cardiac any to - follow FDA to to ourselves long-term express as continue time know we're I we but And interested not are patients these going carefully. think happen over going

sort the hospitalizations be events no in outcomes just or clear cardiovascular longer in thing term up? to or But on is that of follow there terms focus of

fact pre-vascular the mortality. in did demonstrations EXPLORER the not insist FDA shorter is the or not in any morbidity in portion and The there even of term on No randomized trial.

Great. taking Thanks the for questions.

you. Ritu [Operator question of from Cowen. is Next Thank Baral Instructions]

line is open. Your

non-obstructive I may may for your tell timelines data for not question. guys. MAVERICK Phase taking how study? Hi, with when you data contrast guess or X Thanks any expect the trial. in to on us and Can might we completion the moving create from that obstructive enrolment

Surely.

certainly beta really studies opposed You myomectomy quite know, unique to with obstruction The next. lead an HCM planning do. on to on investigation patients going data can is is obstructive effects as we're as to be what an and we're such is possibly really on non-obstructive the in are mavacamten. might And in in think of agent mavacamten improvement septal and how of this no they HCM there ablation how myocardial blockers address there such what what an that non- because there and to the as I population. And tracking are feel they

you’d there's What directly, it be it think in MAVERICK? be of mean ppOX gradient, endpoint How endpoints it we be talking about. would - dyspnea. would should Will about that priority of the I sort be so no

fairly know, into Yeah. we're you that Well looking carefully.

we certainly as feel, know, as possible measure endpoints well well as go they think both how to objectively I You much how patients want can that do. that after

statistical what less we blockers, background these dose, data we well as to how about? beyond cohort A, EXPLORER. lower and in expect going look higher as maybe inform how should we efficacy, you know, in what to cohort, constitutes lower significance, the expect expect to should the contrast that the data you success, know should trying will as we given response beta variability then guess, I'm in about I And it. Got it from just forward the QX think

cohort EXPLORER really as lower we to its know dose was the to A help was those study. B here. exposure you the fill dose lower The Ritu, early cohort saw its very relationships designed given doses, us what activity at Tassos goal and in in

In medications at see addition, background to we're activity these influence going mavacamten’s if look beta blocker all. to

in have variable full expect ranging studies and So necessarily about fill goals. that about dose to sometimes can the the really but responses of say with that roll we'll us first would in mind, out and in we we of quarter. that dose more both helping lot surprising, the range the be get that understand those might data not achieve a it when set It's

for the you. Operator taking Thanks it. Thank Got questions.

We Jim have Wells a Fargo follow up of Securities. from Birchenough

Your line open. is

I want line guys. correlates Hey, the improvement for questions, non- of Are exercise or end-systolic there volumes? to or up things of like that same tolerance follow ppOX you know, on pressure end any end-diastolic obstructive you patients.

some You to one an end-diastolic of planning particularly not pressure, get have And would we're do invasive that. described, you study. this know, just the point at to measurements

at look other measures the well prime and we of [ph] will for diastolic measurements On echocardiogram like surrogates tissue example. from cardiac pressure, e-ovary the the of as hand as doppler volumes, - some or

then power benefit? And you what what it. just the level make can these in patients, any powering with have a of power assumptions, you of comments study do what just have XXX sort – impact to maybe Got XXX on you finally, four HCM

that we'll entire discuss when think a design I you really detail EXPLORER-HCM year. go we the next of know, into

in that the population. have I as our to ourselves, this target we're primary patient - a clinicians going power convinces the we think want of capability certainly well that indeed to endpoint our assess

magnitudes of we in alluding as gritty saw effect closer as that get into similar expecting. as we is Marc more range patient dosing. nitty we'll But to X to XXX PIONEER we're what to get in pVOX Jim, and the

to no time. the further taking remarks. to call for you. like over Thanks at closing Tassos the any follow Operator great. ups. turn are I’d Thank for questions Gianakakos this Okay, There

thank has for XX seen by a data study. Paul, indication years. all a encouraged not And PIONEER an that afternoon. any you. converting candidate is novel and joining to you over EXPLORER-HCM this Mavacamten us and from really thank innovation We're drug in the in therapeutic pivotal

need effective if for biomechanical approved therapeutic greater. The precision really therapy quarters by the options targeting has first will new us of been be innovation So HCM. as would few safe represented never next and The busy be for underpinnings it ones. the

programs. here look the research expect of stream across steady ahead summarize milestones. I'll several We we you. for clinical activity you following and If can expect a

Phase dilated And For Phase patients XXX, a the end dose of in study ascending XXXX this the of initiation reporting of by the year. study. cardiomyopathy first out Xb data first single volunteer X the the symptomatic of secondly, from in quarter with healthy

there are milestones the For coming mavacamten months. in several

Scientific of from well as presentations our First, the data breaker including later new action. month, Association two Digital mechanism this on at as Heart Sessions American non-clinical Health data late mavacamten’s Substudy,

second Second, lower reported PIONEER XXXX. will cohort first dose be data of HCM quarter the the from of in

towards study the Third, the took second trial rich expected in to of in is of serious mission mavacamten the big we world quarter a XXXX. disease. XXXX. this platform, this plan Phase Phase to a believe the achieving is for is quarter initiate advantage for obstructive indication, of suffering driven we us. our during And cardiovascular products, by EXPLORER-HCM key step of HCM, pipeline non- building MyoKardia's a competitive our research a change quarter, patients from mission to We first pivotal patients also X finally, second dosing X begin

our This so competitive learn our further all is this of we we by research with the efforts and more best-in-class back therapies As desperately into clinic, targeted strengthening advantage need delivering those the in them. patients goal learning’s feeding who platform. to

working cardiovascular and innovating to hard be new we're been conditions so forefront patients, of long the by that have therapeutic inspired are on to options. We for proud the lacking help treatments

answer to you the months. and our and coming of to of We continued all fronts are informed your always keeping note and support for the you team progress on that please thank forward I look all questions. the here weeks rest available So over

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

Have disconnect. a You may day. now wonderful