MyoKardia (MYOK) 5 Aug 202020 Q2 Earnings call transcript

Good afternoon, everyone. I'm Michelle Corral, MyoKardia's Head of Corporate Communications and Investor Relations. Welcome to today's call to review second quarter results and recent business updates. A press release summarizing Q2 results has been posted to the Investors section of the MyoKardia website. Leading today's call is MyoKardia's CEO, Tassos Gianakakos. by today joined is Dr. Tassos Chief Jay Chief Bill our Officer; Officer; Edelberg, and Officer. our Commercial Financial Taylor Fairey, Chief Medical Harris, our

Q&A, Chief Business remarks, the be prepared will Officer. by where for joined our their also we Bauer, Following Jake will we line open

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

really unpredictable Good and be MyoKardia, unique afternoon, these everyone. This especially during team been an of the prouder quarter. at couldn't times. I incredible has

The with second any remarkable be these. more even quarter would circumstances, accomplishments so under

in resoundingly positive leader less extend obvious was data staff are some obvious the as ever path readout Thank who to mission to families, of EXPLORER continue for now. us which HCM, like drugs. working world's the company, clear support cardiovascular I'd through is for obstructive our to EXPLORER lives disease is thank their designation, being first and around therapy position rare study cardiovascular the aspirations our you underscored have to bold you. today to reinforce targeted people science the These on and science. a their The HCM massive and its leading and than our millions from put breakthrough of with whose employees by in and the So their innovative as mavacamten ways. the to investigators some our MyoKardia to beyond suffering inflection harder recent HCM a of medicines who perhaps and loved registering in and a and the results people change studies the start and urgent in treatment for who cardiovascular them world participate the and ones the The right

we foremost, on course, the regulatory first Of for mavacamten HCM. laser-focused, and launch and approval obstructive U.S. remain of

to physicians we're community start have relationships enable to and from the the HCM anxious us with Our commitment caring been to deepen, to people who for with to and the prescribe continue HCM mavacamten. able be

our top So questions inventory out coming are concentrating let's right the of of with some on EXPLORER, now. we that confidence high

at? of will ways launch. globally. assured we're HCM the Today's towards time? likelihood What U.S., the could are over think doing addressing we the a key What COVID-impacted will between mavacamten's of the be value availability we launch What a over as potential to plans what's mavacamten that do we think questions the curve might the regulatory of outside company's what focus successfully the finally, be? how our do what therapy do How call the approaching we population now availability like therapy and we the and think What Rest ready well Asia factors influence be about mavacamten's of an And and and are patient What in addressable adoption this our are Europe, look expanding and priced on diagnosis look mavacamten? adoption of U.S. entire obstructive low environment? when? How do think time? could U.S. First, like? the improve as drug might label approval we'll approved we access And first approval to thinking looking on beyond? are perhaps be march list in as in we rate?

not beyond. introduction time focus August our EXPLORER While we mavacamten the our call I around of. U.S. of take emphasize XX. mavacamten the today, multiple of the extend though, of want around growing a hard in to leadership ESC moment, across during disease presentation plan be details and data our a to the preparations for potential on be on do discussing maybe to dimensions therapy you that HCM should a our belief to has area backbone aware working even that We're

First, within future obstructive valuable clinical continued HCM, generation trials. and of evidence ongoing from clinically

exploration continued have HCM, we mavacamten X where the we being HFpEF believe profiles HCM Next, discussed. and outside of targeted additional may disease obstructive non-obstructive benefit, have

deliberate aimed U.S. of including MYK-XXX. beginning. is reach Introducing the the of number with of the just mavacamten HCM HCM and globally. increasing finally, value people our the therapies, who the geographic at for can expansion the obstructive thoughtful And Third, treatment next-generation clinical of in access the with expansion mavacamten

is, company, think one as you As mavacamten as are we we know. a compelling therapeutic not

This atrium clinical currently Our important shown want that platform won't significant directly do fibrillation highlight as potentially a heart along in a unique the enthusiasm danicamtiv ventricle. to reduce atrial generate the generated with to at with today a R&D This is challenging differentiated are the X cardiovascular left rate but the We by burden novel spend therapies, effects of across lot in of a condition, I and study. the the globe. including time growing is has which continues failure. people activating very to finding potential on left approach danicamtiv, has

additional rigorous successfully patient of yielding through also thoughtfully Financial about. Chief molecules Jay, you. is XQ danicamtiv into regulatory today's expanding discussion and get financials that, Commercial and subgroups update in Officer. on U.S. Our into Medical learning a Fairey, first important my novel well-defined our Taylor about approach and patient colleagues, Bill populations advancing the timing thinking around we'd R&D be global let's Chief milestones danicamtiv. in walk Dr. scientifically Edelberg, MyoKardia's which and over mavacamten and way, like program and example you now Chief and we've valuable with update With results, mavacamten's to with an efficient great Harris, provide high-level another from of several then will our to our strategy done Jay Officer; Officer,

share meaningful I'm Thank to mavacamten. progress excited the for you, recent Tassos.

As to evidence moving following of submission start-up quarter in Tassos as We're for for mentioned, first or we're positive prepare of a FDA drug new our EXPLORER, a of VALOR-HCM, with mavacamten application, HCM. the results quickly the forward XXXX. the build studies intended several therapy the to of also quickly backbone from planned planned first NDA, with moving

regulatory our with start update. Let's

of out to data reached to arrange the Based readout, a dossier that ask filing. that sufficient receipt are The is recent feed EXPLORER right data provides that it's to for clear the working Therapy the we planned the their we further is comments and expectations, and track. review in regarding agency toward meeting our pre-NDA on on materials, our steps. we validation the the Designation questions next with to alignment the Breakthrough Following data agency are

breakthrough need reminder, gravity also improvement of was review HCM expedites substantial a evidence of This for a for recognizes on therapies. the benefit recently candidate and hypertrophic clinical the of As and the of cardiomyopathy subject multi-hour development Digital therapeutic designation existing with their new the designation from options. participated. and HCM, impact individuals offered health. session an living, externally daily of their and family in life June, disease burden living led of its comments Day which about talking Development Drug testimonials the on This long-term and the FDA

events all on encouraging important to visit FDA this We the to therapeutics. are event to for speak website commitment with proud in patients, event. replay novel ongoing encourage HCM the of to These be of this division able to view the a to development the partner their cardiovascular the of

in XXXX. do since our been increasingly the the therapy such they and as common form for rare cardiovascular been areas, disease in necessary didn't the only become premeeting of the there's certain that to received oncology, we was nephrology designation, feedback remain approved and granted on it breakthrough of one data, the designation breakthrough designation ramping was feel exceedingly division, where the breakthrough meeting. Based introduced has While

ahead clear ongoing and for to agency feedback submission a throughout breakthrough the access us, have We advice process. and of path ensures the

lives and design Phase III After with procedure. organization a that an to the submission Cleveland as patient with III in a once-daily obviate preparation, the the first Clinics our goal we the We study difference dosing to It's in Last evaluating begun that use delayed produce submitting a disease to to believe of VALOR excited The the trial. our NDA the we're September, to show our in for anticipate being FDA of area The in step is an will can conduct of HCM our therapy. continue oral surgical NDA. of mavacamten we study invasive VALOR patients. next medication to counterparts evidence XXXX. exciting reduction unique dose ability a the pandemic, We to is academic of leadership. work will study the CRX need research to have closely make septal due alternative quarter dramatic the just filing VALOR-HCM and announced commitment with partnership Phase mavacamten's clinical our for our in one

needs further initial continue build the could strategy studies [indiscernible] HCM is beyond and look submission part this needed community. of that for we conduct the of package of program, meet As to our value mavacamten the those medical supplemental to ultimately one to

committed possible will prospect to payers may point beyond a label provide we to well start as heart application alternative once-daily of to be are patients. are regulatory we these NDA need Association of to with dosing an obstructive the objectives, it that will mechanism of its oral in so our IV clinically-based HCM for that looking forward enables Clinic to VALOR on largest-volume many us Indeed, oHCM the are such U.S. to we we our doctors, to likely real-world important and of mavacamten as mavacamten first VALOR preparing. HCM the being provide beyond an program, for supplemental compelling among mavacamten's EXPLORER, benefit. procedure ability as patients patients. that Starting closing, based with VALOR elective work whom including look to submission or open with approval criteria. extremely study with test currently expands as to initial a we anticipated as for are move label. of Now of help the sites have Class dozen several the that obstructive could patients, to serve with the and the including avoid an accomplishment Heart Mayo approach New data to surgery, expand ultimately York The participating our allows the enthusiastically the mavacamten test to centers, indication outcome that the sets supplement solely for NDA and forward and our and Cleveland as future distinct HCM medication able Clinic, as to In to the results new important positioned us sites

about ahead mavacamten as beyond in component, As bringing another the we move we're the U.S., to U.S. thinking people

update me let to an turn global Bill. over Bill? on For now our the strategy, call

Designation, with treatment Jay. coupled data, strength a belief of potential our in be the advancement EXPLORER of Breakthrough obstructive mavacamten The HCM. to Thanks, the has the Therapy our that significant reinforces

build and earlier. of majority We activities, look and that will partners details we to some education bit include in part for creative be world are strategy of this own build territories bring of footprint work keenly to focused which commercial to of the on work. these more a establish that and to the our patients call Tassos engage for to value a on and infrastructure discuss planned I'm of will to U.S. our -- around forward the our territories sharing able ESC the excited we precommercial as medical and Today, alluded we our mavacamten footprint, which around

as the Our investigating leader to efficiently by that the that such launch we at To Europe. the as least provide in in partners ensuring population, least world's mavacamten parts order with treatment we in regions to are at to the objective of with is we'll HCM, XX% the accomplish be opportunity in patients to mavacamten as to currently quickly certain access possible, of And with XXXX. ultimate and know of of working HCM be the access global some ourselves have goal, world. aim this

a well approach Here sometimes we'll is as our are of thinking how core the on how tenets decision country-by-country the evaluating as we global and basis. be region-by-region about

optimize launching and is region. mavacamten of country a local a priority the requires be This that the First, the focused should HCM brand. in given to disease organization the of clear partner to as with as effort uptake the community both brand well or foster

global company's the brand core to image a of control we establish will everywhere. brand reflective and and our we of Second, characteristics that distinct our intend product's values,

the by global territory. we perspective pricing take pricing lead on and Next, will a strategy

you As countries know, model negotiate reference-based a to use price. many

be dynamics. will these to managing Our launch sequencing crucial

We worldwide the that X not of We and will finally, at XX disease. can where local initial lead into mavacamten know It's strategically important have discriminate. optimize in all estimated the million. in HCM will a presence and decisions on of those we opportunity regarding XXX is significant. will And obstructive value as a indication. does establish significant its the we will as long-term HCM to are the shareholders. territories people for we prevalence MyoKardia believe number form translates worldwide This of every estimated HCM, go our beyond a development well that develop

EXPLORER symptoms are directly, around the rates pressing treatment addressing that in XX need population current European regions. Europe As representing world. the we the new treatment for treatment that a rather alleviating of the over across Consequently, we see countries. anticipate in widely seeking the is the and and than similar the were XX% the trial reminder, U.S. is And know toward limited diagnostic and disease Europe, first geared the from where as other centers here vary U.S. approval U.S., alternatives XX options outside region we

task HCM in continuing his directly groups van differences opportunity of an assess been in year, the In with engaged EU centers is these have to follow major U.K. Head so countries. as per care our learned was and is community HCM both In hundreds between far several team, and the and many Weperen our excellence. in develop countries we we've the center. hired there cases, for and more we've basis. Europe regular many, He in KOLs February So slight All this patient of thoroughly office of established Amsterdam. a the over years. often the centers centralized region What HCM large that with highly Willem some on The

will many treatment expect these treatment these in likely play, patients. community instances, drive to centers cardiologists role the We initiation, while a maintenance of in

their treatment colleagues. an desire in They HCM, underdiagnosed to a to are disease. that willing the particularly interested in potential of educate acknowledge local strongly HCM are colleagues, for a finally, patient better with need segmentation. in medical to this distribution the We're leaders and treatment now education. horizon. reimbursement. on XXXX. journey assessing that routes the to U.S., attention filing eager market prepare engaging opinion that to they in in with with HCM interactions EXPLORER the U.K. community pan-European patterns, as investigators, country-specific to key working subgroup optimal agree eligible the to European of authorities a determine The new group, therapy a And EU market As KOLs and chain also expertise access the there's unmet treat also and understand and anticipation experts and Europe, potential and preparing is guidelines with patient express in strategy. there's the prevalence, we're mavacamten increasing regulatory regard among HCM pool, national Today underlying We're to we patient supply opportunities We're cardiology engaging their are

commercial our Next assessment year, regulatory fourth steps requesting the the this Europe for launch sequencing completing our with authorities strategy our of assessment, -- meetings in in go-to-market include quarter and EU for the region. us including

intractable XX,XXX. plan partnership system anticipate for that could next mavacamten's and promotes for health to disease We a and classification research anticipate highly Now research that that HCM means local genetic behind today rare for We've clinical pharma obstructive developed study with study a with Japan the for III encompasses model aligned obstructive in the has of HCM financially patients in Europe Phase a the beginning is care Medical following market discussions We designated both U.S. first and with registration HCM. population pathway Japan's enable and year. we Japan. third-largest organization and diseases. diagnosed Devices conduct is similar these a believe as government half Agency a with which patients. a rare that Pharmaceuticals the the supports disease, in in and Japan, an been had on disease have Japan care development

the of will III Over assess value in Japan that or whether the course Phase partnership. further from opportunity we a independent and program, ascertain commercialization best derived efforts is in

but There, maintain would HCM I'd criteria that X to partner inclined there brand, regional have for is pharmaceutical priority. some few must we're feel willing over affect to to turn in estimated I to and/or partner in expect of are a mavacamten the for. million Now it to we while We a be the #X are And development programs, briefly with that with control very numerous pricing and also strongly the China, locally-based people. successful, where novel that mentioned clear make a therapeutics the earlier. like area entity that we meets looking to over to the be companies experience region,

launch. being Taylor? in we're the for an forward to on I model. our the we worldwide With ensure keeping quarter. I'll we're as here the China, it efforts undertaking as that, do for we thoughtful mavacamten's and stage you the patients activities results financial to go for so that mavacamten as to bring forward set all update And over well anticipated company's to correct global hand to the on so updated U.S. Taylor second right look quite partner the with to

compared quarter start to results, positive ended the net successful and totaling in everyone. of strong cash $XXX We million in and with position a million approximately in raised the financing million during balance Bill, financial second came $XXX the the of afternoon, which the announcement quarter proceeds. at XXXX good $XXX Thanks, increase investments our from EXPLORER following we of second our The year. of cash

appreciation there all opportunities MyoKardia So conditions. thrilled mavacamten our in you ahead very for the new to let team, we participation deep and particular, precision our excitement long-standing support our apply of our many the to to behalf patients our of have mission of investors and in offering. pause and seriously, of heart we and and the cardiomyopathy our lie me our failure in the On in as need bring of so responsibilities to on additional so share we're deliver to approach for express shareholders medicine many take that

While the focus mavacamten year-end or with of as a the to many anticipated year. expect we We're today's support next early much Virtual in HCM. XX bring the Scientific Starting introduction. planning progress August in mavacamten's portfolio EXPLORER to upcoming the on we presentation obstructive data with between and late-breaker Dr. review efforts data at discuss and Dr. IR now also on underway of the mavacamten will our can, Iacopo presenting has been steady, Olivotto to several virtual patients to milestones currently HCM investigator with across EXPLORER, as activities be remains for principal precommercial to ESC those Olivotto, event Congress. a obstructive some

major of HFpEF a course, patients in mavacamten dysfunction, year. a will program year-end. diseases study of The Of be anticipate diastolic HCM our the II drug subset of before of in the first first obstructive application Phase in quarter new of our we our beginning driver submission next

regulatory later in meet pathway review HCM. to we with to year non-obstructive this plan and discuss agency Additionally, results the mavacamten's MAVERICK

cardiomyopathies and Lastly, to following II action. of studies gearing we're to a with quarter, second for to our in Phase starting plans for the half up fibrillation during new is and first genetic coming of at dysfunction A the research, the in in the for systolic unique presented mavacamten results at rolling weeks. mechanism order of year. pointed in with a this preparations which danicamtiv these danicamtiv atrial is clinical enrollment preclinical across launch expected patients to for to ongoing second the development a half quarter. study begin In and X provide forward details planning to of danicamtiv, commercial HCM, in look events respect clinical the encouraging of in XXXX. study We webinars obstructive and for deeper a our looking of expected efforts fourth out mavacamten danicamtiv dilated And we're begin series look

up there? you Q&A call me Let your Jeff, are open the questions. to now We're to period. the Operator? ready start

[Operator Instructions].

first the of from Just Rama. Anupam question line

Great.

Any ones the question top trial other at filing quick EXPLORER two from new data? we lines for in Just for ESC the that the be just EXPLORER get And the time looking analyses we is the XQ. what lot we a beyond from that me. should on know line

things are much potential gained given -- gating are preclinical expedite And can that be So the you could the expedite PIONEER factors in to have that what that results that any and getting or efficiencies already hand? done? so data there to filing

Anupam, Taylor. Sure. it's

These for we ESC, there additional complete that will data -- some results presented. So are the be will be from EXPLORER.

I line had of Now and we extensive at sure, it's most for was not set a the material -- there's information. release so top -- say will that, fairly

additional So out we'll and enrollment to you ESC. that be we've provided, over to analysis in we long-term of accrue some in. keep to additional that should there's But patient EXPLORER mind, so and be comes a that It's component at going resumed have time. expect extension continuing years, then have analysis

So data study. with generating timing, On submission of that the we good ESC isn't quarter the set of the the next about final journey year. here we're feel in timing stop the first this on

expectation have. right to people for I the think that's

collaborative Jay You agency, is we assured certainly be can should and over to as to received. by I expectation. hard the about the with as the -- talk the pushing that breakthrough designation Jay? but right certainly has been turn just QX, I'll rest aided And can. we're which super we process think, it

Yes.

all through agency. interactions there them. We already And opportunity, filing. we track to have we the be with submission have breakthrough, actually multiple we able to with this know with course, on with this and now the So integrated we're the eventual to to of our through now the preparing actually that the file we're the them, the interaction for what with CSRs, cetera, showed preparation, have have right opportunity documents the be et and able

question Next the line Tazeen from is Ahmad.

I to be little launches HCM your needed least that a on how paradigm at get the about thinking in wanted shift initially thoughts obstructive past We've U.S. Tassos, patients, the you're the of might bit about in the not mava example, it. eliminate detail need ablation, but for completely talked delay potential of in in surgical to maybe even some only when the for

the about to you have ramp think study, doctors, educated follow-up that you time? how based be understand think it? for over expectations, then do particular VALOR market aspect increase As I at that design, in how of least opportunity potentially need mava its And on initially, will the better to much a is, the can order

good from Tazeen, you. to hear

big dynamics reasonable giving mind, terms view Keep Bill some then the EXPLORER. have educational through in question working time. prescribers who add with launch, mavacamten for me efforts familiarity now start have let maybe specifics a your diagnosed on picture in been some a got the number of the of at around team So population of and that we've has in

So great. is this

a designed to they see -- the little matter sort on was standard the to relatively study -- context. to stepping the news care measure they're education we the building really that So for things is of and a that us generally old benefits. speaking, is there going into the The good we're educated, of and bit we're physicians, and be

elements physicians have So pretty mavacamten was Improving are interested to the all in we improving use safety essentially feel great. of symptoms, for and that about is the that hitting good of function, demonstrated it. profile us, their willingness to so want we

opportunity question on little the to And Bill elaborate an back adoption. piece a to after your VALOR educational and I'll come maybe so giving here

Tassos. we rate XX% as Thanks, we a that launch. estimate approach and and rate the range, period into a XX% in double to diagnostic fully We certain get Yes. somewhere of over diagnostic expect today to time

point couple to I mean, couple data this the recognize a mean things to clearly, form, more -- of So I talk around identified doctors barriers drug they drive some in right? I limiting today, the how rate looking one kind barriers exist more as we've doctors that treating some than echocardiography echos, as we to that of are be -- what and of of But step. they're to of interpretability are a quality is for to things. those think this good happens disease. already -- the start and an When to accurate going obstructive of and and a the centers diagnosis,

We're We've -- in Society working advocacy on geared cetera. that with we're and So of campaign, interpretation point in content launched that. and We with HCPs. with working campaign, improve the disease help the toward state we're state process disease we're continually groups, quite bit, major launched the ACC Echocardiography, the to et you've year, of ways AHA, we quality medical this adding is American is last us of November about, a and of to a We're campaign. echos. continually working adding. also This doing at as heard the exposed talk associations, which HCM

with are well. RMLs working as Our field the

the different and done, that lot a done, is a the didn't VALOR VALOR, would expect do you give A lot kind we mavacamten. we for we to of a share expect physicians. get calls, -- functional addition which impact diagnostic is addresses impact we And right, here Class EXPLORER alone, think doctors behind a rates lot these improve be throw that be we us to how TPPs expect important EXPLORER, And VALOR there's evidence I asked running I the So thoroughly with in of out launch we it's But research, to lift. based think going functional the uptake. population. you the but to fully I the on TPP how sicker believe were surgery. we've on market the is patients, there's point asked we for clear to the key need VALOR III, the chance and expect it dramatically does subgroup the that an I that IV to to that late reduce And VALOR explore over may, of to Class drug. a If when we think

we in a much intends that the company utility doing the believe to work it's think, the for in and of right mavacamten group, So small will this help out, round It do we very that thing a there to is population. lead space. that

for I an evidence add this Tazeen, overarching is study -- talking bigger the is and here, first clinically just to believe valuable. we strategy would generate VALOR us a we're that about at level, continue to is just

in with adding patient patients, And of Clinic not sites. terms of sites Mayo some volumes they are or the in part and are a addition other so to engaging HCM they EXPLORER. Cleveland a Mayo of that see, more and sicker So dozen the along were here, Cleveland #X and we

to label the there aspects the beyond influence uptake. impact, study that So to are various say, might the let's

So like patients clinical we to with EXPLORER beyond, getting we'll the benefit as more label the on efforts standard and that we're increase to hopefully that care of our from on to let's going, work. more based look evidence thrilled of elaborate more study look initial continue continue for from be that us say, for to

line of the Next question Ritu from Baral.

thinking as from in there's And follow-up. on MRI I've got the data, on that to capture what a EXPLORER. comparison if you to how up timing? COVID guys comment especially going, I timing from data cohort as now VALOR And are far is confounding? cohort you Can especially any of follow wanted that any

as MRIs, was the data within there a MR. COVID EXPLORER, to but all data that in the know timing out but we actually the got a continue We'll wasn't you later which reporting substudy actually presentation on. the data, to cardiac have be to in obviously long-term future analyzed, So that. that get little of bit extension the patients opportunity extend updates impact a these on is on We'll follow patients the and negative

time XXXX, is than rough than out NDA are guess wondering, Got later talking further it. that I'm I And from that? Or frame, filing? XXXX that? or later Later the like like we even

needs specific to gotten congress be EXPLORER will present part will that defined. filing totality of of data data we've so the The that from the be the the

Understood.

want interactions. follow-up about Okay. then your your quick FDA And I a comments on

with that didn't -- a need instead, guess a have and the on in just gave there BDT don't -- with you precise Could mean, investors a that You the to right just over got the meeting, comments status. to that the you confidence have moving forward comment CV on had road especially office pursue NDA dossier indicated head what new the were on you such I reorg you And meeting. now. the rough you you really I

have... you the how with you confident reorg what and are personnel So the and new

with we've relationship it division a before development of the Well, this agency into mavacamten great the had in got throughout clinic. even the

throughout was in process the line with that feedback good with were with to we we be the additional So breakthrough -- do what come that that and designation the dialogues we the and expectations preparing was breakthrough, preread got our us extraordinarily opportunity we would submitted materials there. our them And the back with and have for have meeting, submission. that what were It pre-NDA we for the clear. told

So so we the actually clear. meeting need was -- because the because result feedback didn't the

of the Birchenough. from line question Next Jim

progress on all the breakthrough Congrats the designation. on and

part a So was one what EXPLORER of Heart maybe of just starting valued focus with one designation? was sense there granting And breakthrough the classification, peak York breakthrough that designation. Or aspect the New point, was end VOX, in FDA it by Association of the the was of that, the data composite?

the meaningful the totality think, incredible fact unmet was of The key of end physicians secondary that statistical Well, in of done, to I the I well significance, and how Obviously, need FDA is [indiscernible] point there elements there. on it the top medical think important evidence. the HCM. of all level clinically are valued primary the and highest patients

strong case all told have a that So breakthrough, to very, were for builds it. received and pleased we very

And then following just VALOR. on up

goal understand septal you for obviating septal and the is the of what trying And or need With level surgery, reduction the the what ablation to indication the what's need criteria do surgery? surgery to for actually obviate what improvements LVOT need surgery. would need? ablation for to of for obviate Just the other

considering to So medical reduction mercury. patients XX therapy highly be a they gradient of millimeters symptomatic have recommend therapy over and of septal guidelines when despite for continue

need ventricle go opened coronary or them option part get then therapy is alternatively, think, improvement, for replace getting alcohol septal well improvement mavacamten, improvement criteria for artery. the onto actually reduce the With as to patients, symptomatic once-daily actually below of these an functional relief, obstruction need -- is guidelines functional remove medicine, symptomatic lab patients and these to majority inject and the the a procedure, For and open gradients to them who symptomatic based only and where go give the we and heart reduction septum X for readily. able of in what the as of can cath into you be quite and part the we which on the these take will EXPLORER, give to the patients guideline

mercury. of the over half diagnosis get you patients below gradients get below of of criteria As millimeters EXPLORER to X/X the able able And know, in to obstruction, patients the millimeters XX trial, were mercury. XX over of the of a below their were getting for

very this So we're confident going forward. in trial

final preparation. terms just seems the just of one question in general, very prelaunch And guideline-driven. Cardiology, in

how Committees? so now you decide getting for is important groups And there those in front the HCM? get guidelines do What important can guidelines? that of are to anything are Guideline obstructive to And on

It's question. Bill. Great

those their eye HCM partnership so the them And presents, on guidelines a EXPLORER opportunity data represented So guidelines. ACC. we're an well well when we're AHA, that as by kind with and long-standing driven keeping in working which we flow, and to with in have positioned of get timing, relationship the mavacamten their are guideline as

that and any as of don't sure, or as run, term, -- already don't there we going guidelines critical as say, we're I as important, the a be see -- barrier have to going we there. many being are far the to included short importance, patients In was might far yes. that doubt mava. to guidelines that lack critical long so for be in As we're in whether able to organizations out of if barrier of to some the publication going being the opportunity part guidelines supplemental are for I there's that There a those being as this trying drive. are guideline with right? are a referred drug out enough being to pent-up that find to at not to background been kind at work still we're going is be launch, or there's that evaluate, trying symptomatic We're used be that to think have something an X we're that as of in surgery or launch still therapy demand is guideline I to whether on there not. launch

is question line Next of Young. the Alethia from

At some of the questions. progress. I guess two

a or From your is team. market a treatment on early. know picture, just of launching kind the to for one, and Are bigger the you claims? the the it's around about around set? like comments differ guess, frame relation your in you launching Just kind for Tassos of partnering big I then curious I'm and are And kind opportunity us of basis, globe. geographic kind question maybe how for doctors XXX,XXX I a landscape? how help kind interest aspirations of of And think dynamics second Can you like like

again. Bill it's Alethia,

I and close Let of me can start, maybe points. your missed off some if Tassos

nuances learning market are various and I think the the dynamics. was about your geographies we the what around question first

-- we the the addresses kind a there's can is everywhere learning also the we've as need unmet of regions these an that Europe, for that about we're getting in disease help something desire some into well, there. of from where and actual care. explored despite deep underlying unmet be already what physicians There's an that we've done symptomatic but other are clearly look. is there and We're hard-core patients continue clearly need that just of fairly of So and to research standard particularly Europe,

are example, the whereas U.K. patients, to only will the initiate not number patients like the is they are treat Some countries Germany, -- manage an for have to are a therapy. where countries of excellence, cardiologists example where as will centralized. they of there community-based the more centers, centralized a that. but and Whereas centers maybe it's way There referred of clearly also patients, approach

we're dynamics. through So working those

more things opportunity. are pent-up dissimilarities terms in upshot is seems of So I pricing these there vary. and Obviously, countries to of of of kind similarities types it than and think demand and regions going that the be across to those

So I hope I answered...

Alethia, it's Tassos here.

then true was it. that That or on, Yes. for are XXX,XXX really the you And launching bigger my diagnosis question of smaller, a the of just for claims, kind exists. was second philosophically, exactly kind

time, Alethia, we I Yes, Well, you're super thanks. the you repeat know but kind to it -- question. this did have us.

the looking want haul, is -- cardiovascular world's mean, we the And and we're in our -- long medicines I are this starts be to to that for and leading We HCM. build strategy company. with

central really relationships physicians, the is about with globe this around are leadership, build to area the So us. our health insurers who "customers," disease authorities, patients, the the the are

improve you need So And to going to -- patients them really the to, think those how access to that that. build our we're operationally the then let's from tenets ability might -- are to we kind therapy directly. customize. and relationships of some optimize say, get And get I Bill on with heard region-by-region consistent basis, a

a to play we to bring are think diseases, prepared that I way. to This disease. -- is global that it's got take you a you've be company. And therapies want transformative global much So when very on

of the line Marty is from Next question Auster.

market likely in they're supporting kind the of curious kind then pricing down road, just that different decisions either I second wanted of back of pictures if and your like I'm my of just who of planning introducing things HCM post-approval of asset sort to were curious if terms would that. the I with you it things this around in resonance if And XXX to work kind other be same like studies of and I'm any curious made. you've target about of at update talking going timing profiles readout are something question how if strategic before complementary forward go around have or think much thoughts different that ICER with kind there's groups that. But kind post point. around product to VALOR opportunities pricing pricing confirming the of Bill those that feedback you're payers and was -- or -- clinicians analysis share getting to cost-effective studies, done with either or was like potential influence about when kind and

building Jay Taylor U.S. value XXX. of it's a out to we've our and ultimately. And taking kind on payers this work modeling. abroad, health got burden group. lot kicked or model, forward maybe into or then be dossier Marty, effectiveness economics of and -- prop We're a overall on. work for off the it we're We've the We're going of preliminary to there and the in over and outcomes building the first rolls Bill. illness that take information to one, all our that cost I'll we're midst with we're some doing kick of our in value

to testing as doing different are some a initial were is started we significant about encouraging. drug intervening a there, going way. of where And -- payers aren't did price we're the so What restrictions in work be framework, So as price with lot comfortable quite pricing the the a in let around the market to and think engage X learned at me our we ago. points U.S. pricing of -- work access and about we've back years payers we're We specifically to be comfortable research that going we're up. signs

the payers with drug So a And MCOs the learning our more. the data they're returning and there, the and in begun not PBMs payers, the and we're calls We've on to they're in lot the overly interested based to getting patients they're share EXPLORER of being interested concerned step key about out price. very quite rate-limiting it.

get And those build launch. so we'll we as relationships to to continue closer

or ultra-orphan prepared you disease-type getting pricing So the disease-type Not been pricing, ready kind pricing. kind the based look, have on to of going others what more around been. orphan about aligning we're work we or described. I not look guide of We've to than yet feel we've like the talking to close like is

per in still $XX,XXX we're So to this range. $XXX,XXX annual

I -- of that are think today like. help. we're will modeling the cost will look surgeries VALOR and these I expensive, what

just don't way only us to XXX? see of see any all only really upside. the I gives I VALOR on at out. think I it VALOR So regardless Taylor reads downside upside. or Tassos

Yes. Taylor. Marty, it's

that I, have to Phase due the resumed one for but on enrollment This is we XXX thanks COVID, have pause So on we program put to of study those that we're resumed. question Phase had be was I and a in our -- MYK-XXX. study. in

we make We haven't we before it's would we guidance I enrollment there because reset relatively fully data sure and just that on expect when have challenges But guidance. moving want and near firm Phase understand timing along, to are still due we'll that to give COVID we future. in rates the

way the we're very in and thinking we've what few strategically, over changed to the is it's last mavacamten data. about the similarly find XXX guess delivered described hard Now mavacamten, the data in on still it that months has we've past. I gaps really to

disease And the the on of people. we're and as we area excited super still of unmet need be company raise that, have meet HCM for to is a leader acknowledge our the we're about bar care to to full for goal this. in to set continue early these that individuals standard but So who

as the to we Similarly, or lose subsequent in programs that could data can to if out sets patients. round mavacamten. the And learn about continue hope do a will earlier we need deliver what other And We the to a absolutely that HCM company would to going XXX on want And I we'll fill. residual so Phase from forward, on pipeline we're We've with got we to one. we we're be care it go more for the see path is. we think as there uniquely doing that that. need is to have

So we're any thing super bar that's it. The only high comes changed thinking about setting that's a HCM is, for behind in therapy the the mavacamten way is space. obviously, that

from Darout. of question line Next the Etzer

progress. the on Congrats

press if the could those treatment of talk procedures just of I And of sort XX wondered reduction XX couple of in wondered, the any about planned maybe if interim of centers VALOR. Just U.S. HCM patients septal what release, highlighted you a questions then represent. for discuss the on assumptions for the VALOR. the secondly, powering could maybe just the percentage effect does just centers around you HCM in the

[indiscernible] EXPLORER tolerability for really powered and all XX. getting you've not-too-distant that do that effect not likely time what paper design is well, of power with the them seen seen. the off the very ability the We'll of we're in can that we XX in patients if you've getting the Obviously, below of below But study. in So future. a later. get assumptions We'll discuss timing the

the participating, that. about well alcohol what sites that's actually is trial. our participating assumptions think for do [indiscernible] in taken are are as have that of that these surgical all actually powering the details that the consideration in We'll sites we there. So we into states When as both get

a great engage additional that opportunity to So [indiscernible]. it's centers

hurricanes now this Jay. is being the myectomy saying that his what the ablations the is was where are done -- and reduction choppy. apologies he's he's saying both that's That the East battling septal he XX is for But so Coast He's majority is and septal in here. Tassos treatments, sites with the the of line and alcohol the What U.S. represent

Jeff Hung. Next of question the from is line

us some to begin the had on a is what I you on first of advancing in remind next year? earlier for track ACT-X it half clinic? into gating questions your still are factors And of programs. the Can one

the about ACT-X, that muscle we're of proteins tip research lot It's think remind the the terms very And that in in at haven't is a of we phase. about advancing Thanks danicamtiv, program guidance, both which -- is excited Taylor, the Yes. an a Jeff, is cardiac asking it's for well here. cells. it's still the do and here? our in your the Tassos to audience, forward. And moving you pipeline. of I also activator talked about have tongue

Yes.

a We here our starting the the webinar we haven't on programs while. updated of preclinical haven't guidance we're planning on fourth mentioned That we is events something Jeff, series quarter. call in -- in a

we'll our we will and one research more detail. that's efforts, be have so And covering in dedicated to that

ACT-X And can some appear how that learnings from be targeted there given to for danicamtiv? HFrEF Great. indications Okay. does in any patient populations? for to potential apply you danicamtiv overlap might differ those Are from the that there ACT-X

mind. here, Yes. you if don't Taylor, Tassos it's

powerful I efficient is Jeff, think whole so what platform MyoKardia. you're and central think getting is to really there, the at at our philosophy we and why

in of you like biomarkers, relative and has to heard have different imaging audience Because about systolic to dysfunction markers patient Venn that with diagram So of think I the to is describe generating systolic dysfunction, about rather than to evidence, of we're sort we think where -- different today the profiles thing and you think they people all history, likely country treatments, we million help systolic the really least that clinical X thoughtful map is this territory. need about that go. dysfunction, strategies we're doses. how us, do us we across the and think, approaches, this And new profiles to that echocardiography, if know, refine in our as of most symptom function. different going We've I the different at is the get way got very what molecules to patient

our then of you and -- if think got danicamtiv. Danicamtiv is genetic now we at got with have group second HFrEF So DCM programs, we've aiming a now we've of ACT-X, AFib.

and to lot the differences underlying about in their we're a these what So biology going patients, they learn need.

something brings definitely ACT-X to the so table. different And

that. So standby on

to are really X with but are see, not think disease measurement we're we collecting and us we're profiles that leg that handful XX of insights are longer HFrEF a And people I X disease talking know much no give years, I thoughtfully going proprietary in a have to robustly. drug up important before about one about to categorize to crude maybe we're talking different is Just framed that with million mechanism. of will unearthed hoping we really more and probably ejection that what mechanism real been happen, matching think in that fraction,

Mohit Next question from of line the Bansal.

on quarter. Mohit. the Congratulations This on for is Keith

have Just regarding question HFpEF. one

and has ENTRESTO you think in since points? from where biomarkers at you're on opposed also of looking the indications for stands us the Novartis end all working trial. you analysis outcome understand as And the approving Could we're filed on these the to drugs this point of subgroup on waiting So PARAGON-HF do the view decision functional FDA FDA your help on also, HFpEF, on in significance then indication? of

something points And let's the FDA's based to. HFpEF definite to biomarkers. first approval there's So think, that potential start that we've as a seen, open I as using field on end openness part they're for the function

going. to I has that. something paper where their that openness think very So them with much FDA applaud the in for really signaled, we and with this clearly position year line this we're is their last That's

patients a terms with able we'll HFmEF complements of distinct see program being are we're with that In able really we population to the that benefit targeting end, be program our develop from where higher to that that's at we where be actually HFpEF, to the there. EFs really think quite able clearly

I failure able more think diastolic dysfunction of lumping as be specifically to that we targeted heart [indiscernible] stop the of need start just preserved into much drill half ejection to really to and fraction. down

define become different able this able disease be we you that benefit think really I from been will way. in then the who's and recategorize leaders to therapies, able the So to to to be space actually

here Keith, keep purposes. for in it's have nothing there, the in Tassos transcript the Jay HFpEF, just mind that line ENTRESTO HFpEF. was The just had That here. we on event on approved

what around of planet. it's than different world. XX is unmet on But more doing the over So medical ENTRESTO people does. around the needs million is we're what one affecting major Diastolic dysfunction the fundamentally

and drugs developing answer to designing think get goal mechanistically and previous to are have effect for disease to my is important hopefully, previous about that call our eventually, maybe us different and So to -- this back transformative the it's question, on to going modification. or

clinicians disease well stand of to as opportunity are the burden, to function what biology matching and improved driver, of for, greatest mechanism disease drug hospitalization underlying disrupt fundamentally looking Our as effect the as mortality. quality we're view patients and progression symptom of disease is and improved you totality life,

to be, we -- need But that's how is the could danicamtiv and are there that There done molecules with our more benefit, that have and work be has mavacamten be view and so are and that in benefit might My is believe when design is something targeted. in there in going potential. We we something. will HFpEF designed not because that mind. to And ENTRESTO be in mechanistically my this estimation, designed. they that absolutely molecules really be for welcome really HFpEF been

the Singh. of Gobind from question line Next

Congrats on is from This Gobind for BTD the George. the and late-breaker.

to not at wondering thoughts your wrong, but trying I'm out, about or all the -- representative septum. was III. for to that had Just actually obstructive on that would the that a ablation EXPLORER overall around? about for to there it that. might is the in How population would that what I'm I'm problem Maybe should questions. the wondering I like percentage And we HCM be remember surgical great other candidate I'm surgical of is figure is is correction be referred I if septal it correctly, Class XX% a population because is be of way and about not that some think think sort a of I hear amenable something If mavacamten? it then few this was surgical the gets correction? thinking population,

and So I our both think Class in patients II that results Class good III we our EXPLORER. in have

in we overall symptom of got reduction So and terms point. primary good our benefit end

I are think that II III being be We're think really trend an the increasing to Class Class the This mostly able this and and Class model But predictive but beneficial will results results. will very IV, to for translate for previously Class who that then had overall, the II. VALOR. EXPLORER be more has been been of able of into some we therapy. be well reduction seeing patients referred number septal

It's Yes. Bill.

highly Europe. representative We EXPLORER the and here. add U.S. the population one just is me here Let in think thing of

the we think functional about probably little representative. Functional breakout closer XX-XX. when III XX-XX, about Class II, between that, symptomatic highly It's the Class Given is population, a but to

Next Nierengarten. from question David

of on VALOR. couple A questions

then or purposes? missed I presumably, that a time bias And the but something, that, how works physician? maybe of do you randomization pharmaceutical? the off, avoid explain what's second, First given point And the could surgery for surgeon recall, with versus you the point for on might it over time seems, converse as end immediately, What's and it improvements where strengthen patients, for taper time? improvements is the to experience the timing where I surgery continue true then mavacamten, choice the how over

Jay. is So this

eligible to or open-label it everyone will -- they elect either at the then being of the to whether get Let either study, extension. will VALOR me clarify. SRT get SRT In SRT, or mavacamten guideline who they're for are for who the come duration. are And referred the of into randomized study. And trial, deemed end they the end to allowed then patients continue at that, be in receive placebo a an be X-week for

the during that how meet the really study. that extension mavacamten just there? ablation open-label for open of that guidelines is additional so is constructed? so them Or for To point time that extension, -- support effective, that's would follow point presumably how septal And an guess, findings Kind if the you for you label, long? is is guess, that more the they get they I I well, there

Yes.

to to to continuing of a not surgery that to actually we'll period It's avoid of period weeks, benefit the prolonged patients but not able for a for XX would be over for to and time. So SRT a be have only need the able them time. these durable need it's be study first prolonged

Please are There at no further time. this Okay. continue. questions

for and us Jeff, Thanks, Great. everybody thanks to joining today.

at of we and our presented end that event August, ESC an investor will have data we'll being reminder a the shortly Just be hosting at that. after

So forward joining stay tuned for more thanks look for again to speaking you We today. us then, and details. with

conference. for and you, today's everyone, that concludes participating. Thank Thank you,

may You all now disconnect.