VRTX - Earnings call transcript Q1 2019 | Vertex Pharmaceuticals Incorporated

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Michael Partridge

Welcome. This is Michael Partridge, Senior Vice President of Investor Relations. Tonight, we will review with you Vertex's business progress and provide our First Quarter Financial Results. Making prepared remarks on the call tonight, we have Dr. Jeff Leiden, Chairman and CEO; Stuart Arbuckle, Chief Commercial Officer; and I would like to welcome to the call Charlie Wagner, Vertex's new Chief Financial Officer. Dr. Officer; Officer, Silva, Q&A. will Medical and Corporate Chief Accounting Chief join Kewalramani, and our Paul Controller Reshma us for recommend this the as access that on We website our webcast you call. slides to listen you This be our will a on is conference website. being call and replay recorded,

turn limitation, We will Securities and release for without call ongoing CF commercialization fibrosis, statements, future our with Jeff the on Dr. development statements our Exchange on outcomes marketed I and potential that the of are Leiden. the Commission. to now could performance including, other press subject make triple in regarding uncertainties and management's Vertex's call today's forward-looking combination and and over medicines, events regimens These assumptions. risks Actual materially. discussed this Vertex's and cystic to are programs current based those in Vertex's detail differ filings financial the will

Jeffrey Leiden

evening, everyone. Thanks, Michael. Good

has In in this can important in the both executing measured strategy our pipeline. our Vertex income in During strategy non-CF and we pursue in drive more serious these many for XXXX, commercial transformative then several investing clinical, patients by innovation diseases achieve against bringing the milestones and this year operating be its and regulatory that growth. to world success expect CF and medicines of last to significant to the medicines to create years, continued around to revenue scientific

SYMDEKO. expansions value ORKAMBI medicines medicines bringing execution recent patients' Approximately our we're confident today half patients people us this KALYDECO CF a all of and and are allow our CF As that ever eligible and and CF, for we strategy more with Vertex successful through to launch for medicine. look approval In forward continued of new deliver so several doing shareholders. approved lives globally the create label are the to of change more outstanding in to than we to next that the years, more will

Our vast significant an X deficiency, CF medicines MAA from and could to also well to our want I updating we expect us second the the data allow sickle for all medicine of this minimal later remain XX-week recent where the progress With the providing mutation. to third medicines other the people potentially obtain quarter in regimen beta-thalassemia, segmental also followed as both is transformative X for underlying sharing quarter, year. Phase and cause In patients of the our approved future, that their we plans our patients disease, pain, III forward focal outside our additional choose are as for submit for to triple combination eligible alpha-X for many to you for cell in NDA we've to in showed to III with we serious treat that on develop toward programs, We made regulatory submit later diseases. glomerulosclerosis data We U.S. quarter. all goal triple and benefit regimens, progress enhanced Phase X FXXXdel regimens combination in benefit with with for CF people the and mutations disease providing first of mutation in the believe to XX% potentially combination approvals our function of look our triple for advancing combination by and an globally. the highlight to transformative goal track on currently that regimen we majority this final chosen treat will up Europe now to clinical patients this CF, the those antitrypsin which disease, with our best with reported with data CF X have medicines. FXXXdel triple

inhibition Our success our biology molecule proof-of-concept medicines multiple for acute, early we Data enabling of which across our data VX-XXX inform VX-XXX II increase conditions. announced have lines. musculoskeletal FDA initiated for and of studies using our established time preclinical the first development we proof-of-concept will genetics. second have molecule efforts pain. we together Phase neuropathic development significantly correctors In deep a I benefit, are transformative In early program, on By validated understanding these for Phase in corrector to AAT from late to development development molecule in molecule Phase pain, Designation development. and paths now advancing probability clinical underlying clinical are small late have with a from believe development of potential in in VX-XXX, this small predict of small moving corrector, of through studies and discovery trials, We're and AAT in we Today, expect these potential a we NaVX.X will rapid AAT received in we've the the this we that XXXX of diseases other begin Fast in molecule. clinical targets XXXX. II Track focused study from markers also to dose-ranging our pain through of

a advance NaVX.X patient beta-thalassemia, editing the our with collaboration to and our year, in dose disease In into cell sickle track of this molecules infused VX-XXX, first the CTXXXX portfolio Therapeutics, with addition lead and first inhibitors the with we cell We the the to on In CRISPR expect Earlier CTXXXX. XXXX. gene in partner, was our with these also therapy, CTXXXX, in progress with we disease and to beta-thalassemia scientific CRISPR. remain have development significant on development of for editing of with molecule making first late of the additional sickle patient milestone the a clinic field marking for of remarkable middle the the the are gene milestone year. multiple preclinical rapid

KGaA, months, access our of that transformative over developing gaining provide are us In enter Merck new to including serious for on we growing have additional new to Toward to recent medicines X-Chem, our broad internal with R&D Arbor end, range our access with that new platforms through Genomics Biotechnologies, those into specialty partnerships assets free And have to our focused development a provide with flow, technologies, cash scientific addition strategy increased to fit further plc which access to flexibility capabilities. collaborations of into entered diseases. and multiple and we and external we together collaborations pipeline bolster technologies. efforts,

expansion, President I'd Vertex served Charlie CFO close, and has Finance. CFO our I of turn as commercial the complex Wagner the expertise many more and Silva over an where new he like company CFO to will integral his in In I'll through as roles us and continued brings more Charlie the medicines. to of companies like to the XX will Software Officer. more from including our I'd becomes thank Diagnostics, of the our welcoming for financial now coming the than also Paul for future public Millipore. Vice Paul call just progress. review organization Chief significant finance months Ortho-Clinical Charlie within years, continue to global X as the over Financial has treatment joining as welcome years guide and launches over Progress our as Before us Stuart Charlie, he operational new private CFO. play Charlie patients as and served for since joins role to to for last Vertex Executive business as help that to XXXX. leadership Bruker, Interim

Stuart Arbuckle

by the adjustments XXXX, negatively quarter we early first strong product typically higher by of underlying occurred the we on CF performance increase quarter, I'll $XXX our channel in end for quarter XXXX revenues of Thanks, the demand our the and our the number highlighted million. commercial Jeff. medicines in as inventory our fourth being XXXX build driven first to of gross to review of U.S. In net Compared January. year patients Tonight, to we the experienced quarter call in at revenues impacted resulting our globally, that in treated the with continued by the that first medicines were internationally. from and

$XXX the is potential SYMDEKO FDA from from to in the strong SYMDEKO year. received new $XX from agreements initiated start, as young see who patients patients children ORKAMBI, or year. new quarter of revenues for ORKAMBI as our who reimbursement our approval reimbursement globe. to treatment U.S., secured to deliver following patients in with coming track With with Germany. on we demand primarily we to quarter, in and we with patients, revenues discontinued including we've first and million and the from XXXX, U.S., revenues, of for Outside continue remain $X.XX switched Throughout around anticipated KALYDECO treatment continue U.S., billion new for EU, well ORKAMBI. initiating have off medicines. U.S. to CF younger the the The treatment outside the on will never to a performance initiate Based SYMKEVI full patients Germany billion XX X achieving as progress additional in million $X.XX approvals of included first later regulatory including SYMKEVI we launch this total product ages the CF make

provide on agreements We long-term eligible to that focused access obtaining all are patients.

serious previously value in these result strong first ORKAMBI value from in including countries reflecting of of residual medicine that older continue further patients agreements and to become the around reimbursement our ages all milestones. younger a demand function Sweden, this We of to portfolio in children the for we of to approved medicines bringing we quarter an KALYDECO, ages eligible like are We've we year, SYMDEKO. our a of X for and year. In as for for positive product diseases. our in January pleased scientific medicines ORKAMBI, validation for recently multiple also Australia, this of The for XX those Charlie SYMDEKO XX recent and innovation defined quarter future agreement is new invest a discovery as and toward and the pricing our Israel ORKAMBI pricing seeking prior appropriately in received our as part ages commitment formal old Committee, the our children CF prompted will review seen and available patients as rapid reached with as also a in bringing agreement Benefits achieved to and reimbursement successfully With as And for with to old in call for the year. years children patients pricing revenue provides. the performance the pleased globe agreements other of year In and medicines last us important Germany, smaller the to of We've first pathway And in in the medicines patients for ORKAMBI we've first our Germany SYMKEVI, agreements over Ireland, around SYMDEKO our positive that mutation. for the X we strong to reimbursement the by XX EMA are results. was to to In Pharmaceutical step I'm that since turn years. where that, to in enable resulting comes agreement, agreement CF have medicines further ORKAMBI now approval recommendation reached I young access recommendation multiple of the world. expanded older. Australia X summary, I'm our the Advisory now more patients financial young

Charles Wagner

company's an evening, to the many you good the look and everyone. important in join Stuart, at such coming excited time and forward in meeting Thanks, to Vertex of months. growth, I'm I

to review performance and our results our quarter of addition our first guidance. CF XXXX I'll tonight, comments financial financial the In on Stuart's products,

I increase primarily an driven first launch discuss guidance start. Germany. off is in XXXX. XXXX the non-GAAP. of U.S. by of launch the million This and All SYMKEVI of and increase revenues of of compared will the quarter to strong XXXX the SYMDEKO Product represent to XX% are in $XXX a results of recent the in was

million and first to to due to increase to and in expenses quarter disciplined for and compared $XXX of CF expansion This first first of in incremental Our revenues the was support XXXX. of significant in XXXX resulted and first A combined R&D SG&A $XXX medicines $XXX the the was million in Vertex's were quarter income quarter first the increase XX% income in million use of growth the Vertex's other new of million XXXX. of global pipeline the compared investment primarily XXXX $XXX an $XXX quarter the quarter operating in quarter first XXXX. compared million, spending Net areas. the to of disease

in billion sheet, balance ending strengthen approximately and to cash $X.XX end with our of quarter at to the XXXX. continue also first billion marketable We $X.XX compared securities the

internal XXXX medicines, will to and enable are significant development cash with generate continue activities. and our in us throughout our continue in flow treated as beyond to We business patients through innovation significant R&D to more expect which investment external

to Today, anticipated SG&A and effective guidance combined guidance. on Now expenses financial total reiterating we XXXX our R&D our our are revenues, rate. non-GAAP tax product for and

we revenues of Stuart expect billion As mentioned, total $X.XX to product continue billion. in $X.XX range the CF to

reflects Achieving guidance we our as that additional are revenue revenues, countries revenues XXXX time. reimbursed. where upside would anticipated from guidance agreements our may provide our Our update significant medicines at appropriate to reimbursement currently and in

additional combination billion. CF into billion and We combined of development also expenses expect drivers supporting launch a our to potential triple to expanding key $X.XX $X.X efforts R&D diseases. pipeline the continue SG&A ongoing are and regimen The investment of

XX% year provision XXXX a will profile unchanged. our of operating financial we our vast also XX% stronger. until The and net tax full to losses. of noncash is be The guidance fully strong getting is use our of Our tax rate majority expense non-GAAP business

execute forward forward. to to all the of advancement growth, our of our back pipeline you innovation. I'll on going continue operating look significant revenue across in and turn investment updating We business, external our income I call aspects Jeff. enabling and progress With that, to

Jeffrey Leiden

revenue on patients, operating continued pleased that to to Thanks, of established Vertex that income positioned remain in and significant track growth XXXX is our goals we well in this year achieve the and I'm bring key driving at Charlie. start beyond. more the medicines thereby to we and

progress is of creating serial our innovation the of year. coming strategy continues line our through over all look Our parts I that, And and medicines forward to to working transformative you to will questions. drive With open business. updating I the on

Operator

Meacham you. of Our first from [Operator comes Geoff Instructions] Thank question Barclays.

Geoff Meacham

across From clinical when guess guys. filing gain reimbursement have one. given in a full question. pretty a longer the versus you of guys for the Thanks obviously, commercial I for the Vertex. upcoming seems I it the for compare space, the Afternoon, broadly commercial to others side, I the important, this EU and needed time triple. substantially it's,

question here the payers tipping up to EU? think with is from clinical question. the the do you what is And a the point change And is, plan across B? on then the So what follow conversation I'll

Stuart Arbuckle

to where we're It's give Geoff. the world, certainly would including don't remind we in Yes. taken to Yes, Europe. I we've Thanks There reimbursement and waiting are for have certainly access patients yet gain would here. going around to do than but many reimbursement, it's until in we up the the got you access. like some certainly we are there question, Stuart longer not countries countries for have have liked many, who

those that because, tipping taken. the are since Certainly, disease. As I can clearly, such patient released medicines, markets hard we've around you who of are individual is were which has increase find point the deny and know, results think of hard to be it to is every there's that say an medicine the it's very to cause a and treat significant patients a kind market to medicine different. all level market-by-market want triple there about in -- so profile, very important generalize clearly, that of those underlying sitting patients government really access to time. advocacy time to of are to going that the it's different seen believe going because I It's with their hard benefit-risk I think Certainly, very

Geoff Meacham

and diseases, series come then rapidly, series compounds? multiple the kind they next-gen and develop IPF the side, correctors the but if where very of a whole But is a have look and - effective. same that's in a other concept I guys and way you on that, curious of something have in at you shots to been success you just I'm to of to pretty clinical the on assays pulmonology San there in you leverage non-CF, know CF, have and very to And quickly whole had same in compounds, goal look Okay. like in able Diego diseases pipeline what with like or have COPD to a other

Jeffrey Leiden

thanks couple different This the I'll Jeff Geoff, take in pieces. Yes, for question. it Leiden. a is of

which First of able CF. the are at been develop portfolio all, with we speed whole a in molecules we've really pleased to of

X Just to VX-XXX less laboratory first remind than synthesized you, years and molecules like the XXX in were ago.

through or only development in and in And I I when the a development, learnings taken seen before. in both my say are preclinical X development. And in we, industry years Altshuler research mean Phase through but synthesis, III years we've XX lot at I not which that. of XX we Reshma least haven't in from So David

time in have And forward. in biomarkers our animal on with clinical is we'll both success thing they but predict get of increases small Don't that humans. first With about targets. cell into success, lot early all the validated spend talk success move in pipeline, clinical markers speeds in that rapidly CF. of also and development, biomarkers models. work predict We into we the work clinic, we The a more cases, when clinic preclinical we and trials the in can the probability and that

answer. cell And XX- or like trials As as that's AAT pretty true and much of us pain. XX-patient you remember, II you'll phase tells and sickle the diseases also see,

we're And lesson forward that certainly well. so taking as

on given because in don't COPD even knowledge leveraging see that programs I we've don't the same area example. we by what therapeutic distinction. about CF words, you about knowledge, CF. than see COPD knowledge. in opportunity asked in other we COPD an as to scientific scientific asked We have is in done way we and In leveraging leveraging do not one You comment and knowledge The also want believe

And not a and area. like and like much into by CF. CF. best so But genetic unmet therapeutic large It's knowledge we those. it's Disease really do looks example CF. we that we folding leverage and is scientific probably so pick disease opportunity. by smells obviously, where experience disease, disorder then the is need And because see it like protein AAT

We we're trials not. believe animal clinical whether us biomarker that and and small will have simple a system predict and cell we a we or system succeed tell that to will have going efficacy, an

very And expect rapid we thing like reasons so in in And so in say believe I won't see final then the maybe the portfolio you think CF. the I disease would and see as you in approach, to progress AAT same strongly can very rifle these do for shots take diseases. is us this

molecules, and we create You'll multiple which way I we'll think clinic, AAT, into FSGS, we very have reduce that molecules and multiple pain, risk. is a it in see have the in it in us have we to take important

Operator

Nadeau you. of next Thank question Company. & And Cowen Phil from our comes

is line Your open.

Phil Nadeau

the too. Thanks for one question. taking me, one A clinical and for a commercial

First, VX-XXX could an Then on milestones could look negotiations? side, commercial on on about remainder those in that going the for on any been that clinical the and externally France us VX-XXX. stand for have where curious over we of XXXX side, in the there's I'm whether and update you reimbursement negotiations give progress through the secondly,

could registration has for moved route to data? that that Phase II. see mentioned And is You VX-XXX? that Thanks. combo most we likely II the now into Phase When

Stuart Arbuckle

take commercial Reshma your then here. Stuart the and take I'll question. will clinical it's question Phil,

country make around say, continued reimbursement first the reached progress multiple we've So over my the mentioned we then to the approved committed about where we've have and so so that we said France. remarks, Every talk I'm are fully prepared to last talk in pleased England, don't, different, quarter. as can I in eligible to and good world a of I But is securing all about medicines. I'll years, I'll as access couple certainly access our agreements patients to previously,

and to at of inquiry and the that the approach the out Committee our see the France, our from in to value allowed productive would in any of NICE, X,XXX committed that participated but we're agreement those company, be fully are medicines continue Committee position in access there table early-access for are to we remind with initiated for that the patients, were that on a negotiations. Select explain authorities access. inquiry positive we XX in And very that that for we so won't above those is paid the to of March we likewise, as or to of taking impact I and France, kind revenue on positive things continue we ORKAMBI those inquiry. being know, discussions you approximately was don't patients with French and recognize As be securing programs. I most In we the England our our have reimbursement the us through one X,XXX secured. Select were now think patients And on patients you the till final in NHS ORKAMBI a back of ORKAMBI-eligible and, Health Health I

in can committed is waiting patients England our could to you are who patients long those we we're So not I in waiting predict ability conclusion. tell going I you within those successful are tell access that. well. France what exactly a just still Unfortunately, wish to as that's too fully reach and who've when to been I getting for

you any really be any point along aren't externally there to So can't would visible. specific milestones because way I unfortunately, that the

Reshma Kewalramani

through this proof-of-concept too regard the results, this and Phil, next-generation the it's early I Reshma. our we're us Phase VX-XXX, a trials, you in going to little can to to corrector, from stage. have is think you see these we With have II. It's With now speed initiated II when tell going and take to is that but that's you'll its study and monotherapy, we're the initiating we're that that to continue VX-XXX in asked molecule, expect and I as the deuterated it to pace of ivacaftor through remember that full-dose the to now. makes ranging with that's regard FDA VX-XXX, that move moving, Phase kind us

compare that actually, proof-of-concept think full-dose with opportunities. combination those the that we that monotherapy any will are say we're VX-XXX You right the I XXX VX-XXX of because corrector VX-XXX we is pair appropriate. with certainly ranging, in But is most in next-generation doing one study. is

Operator

Thank you. of Michael And Yee our next Jefferies. question comes from

open. line Your is

Michael Yee

at the on know point you're out through homozygous of I in comparing CF population that going And pick at that figure you're appears you're het/min -- and you Can triple AAT homozygous? what that Question question what the those correct. walk or program. you just together, Thanks. it if I filing maybe and heard on you're het/min to was what looking and to my one both then the program. second for

You've made some helpful. just micromolar what data? a much. bit. Phase II comments Phase and might like you from I, AAT. anything, standpoint you could talk Maybe to a see really XX about need I that. think you get you if about just to PK/PD of to Could that Phase so Or confirm II in Phase I see Thanks be need

Reshma Kewalramani

Sure. Reshma. This is

Let first then the we'll question me AAT. and take first get to

you we looking going how With that data VX-XXX. really going asset XXX and both to than be laid even data down XX-week to So both strategy And assets, filing that be which to regard FMS that guessed at really, we're what and and exactly to we're right going And this that FMS, calls the means progress study, sets, of we look QX full of efficacy on choose. the for literally we're FS both we exacerbation, have we of pulmonary VX-XXX QX us like the looking means make but at that's and regard and, point. what out share this whether The up for you've asset means last things the gleaned the the who regardless to XX-week I actually importantly, XXX. very as would marching asset data. break we to at Because faster endpoints XX-week safety we've so look selection for we're discussed evidence. or this And you recently, important with file past, know opportunity are visit has regardless of study information. shape importantly, in as to of to That XXX since on regard little AAT at to with the to very of it of bit its both, lot be has would make to thing year went means both study of set XXX the An last are a information. really year, down, is have selection. secondary be ever the if our With sure can to in all decision, that sets going full decision at is step call. with I'm all back and regard And superimposable. We're the package ppFEVX, totality of program, track VX-XXX. level that achieved to asset last of going to course, good also other a the this it just in is make patient and, and the and this in everyone the FS

I that. in to have the just much This that is described that, think status That's from today, are prepared we be We're PK don't needed well, efficient, molecule that know therapies going his you and track very the as well is an reiterate has study year. And I That going improve Jeff and recognition remarks. the the that of very So of then results size. the a that to exact I'm FDA. serious It's last ascending straightforward. and single study. and AAT started announced the molecule is to how VX-XXX, XX, of delighted from condition CF, that, SAD, received very indication have It's the levels, something that like because anticipate what XX, readout do to these and is we XX patients we're ascending the dose, December gives to examine, it fast like patients, important as we know going need to multiple need unmet who as to illness. dose high in

anticipate to that's that, what to see expect kind So program you're going this I of as to fruition. comes

Operator

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

Your line open. is now

Alethia Young

thinking a guys antitrypsin a over about will just kind curious be the that second of bit your are more little alpha-X was Or game? you Are program, combination I time kind as it some this decision on just you just may, molecule? And of backup if discuss a steps molecule you around the can there? strategic rate-limiting a making of I developing pain, molecule.

Reshma Kewalramani

internally Sure. very, all With that CF, very you and to say as to like to here approach to similar we have we chemistry. have molecules, biology, a on is antitrypsin, it's crack the regard to that a the say portfolio alpha-X pouring about of is once

preclinical molecules choose molecules, very the best about be And very monotherapy to the A Unlike will to of that's development, one to comment in it molecule, the approach, to that's sufficient portfolio is here. us question including on why there specifically like just underlying bring to we of CF, allow single of so forward. is believe, CF. disease it's not cause a the treat your combination going be to

pain, really same biology our at studies and bunionectomy; to With good study portfolio biology, no that's was of is neuropathic all positive for was to is believe dose-ranging easy which in here regard AAT. of the once use. of data that's So that again, model that strategy, to a I look bunionectomy we because the we've that osteoarthritis us task. molecules, we crack with We're a and VX-XXX pain. look at at to we're model X.X, was a fiber going musculoskeletal what cracked of do pain, acute went small do And neuropathy, in that and the then study; model really of looking the on pain,

molecule all molecules that need You anticipate time, on and over on step together, a this We're going second know all this the have period information we'll half ourselves. and which including long decide I have next people of year, worked the of exactly the or what we in is. to many, to have many

Operator

Merrill comes Thank BoA of you. Ying Lynch. from question next our And Huang

Your line is now open.

Ying Huang

medication. on believe that's First triple the the U.K. methodology with benefit think one to that is you the pricing the mentioned of the Jeff reimbursement. even close come with not nice may value you is previously of what I this clinical observed, out combination still

comment also disclosed So that was $XXX,XXX secondly, based to NICE is do in forward? you in going the average think Can process the your there this? for per patient And how then change on recently, you you XXXX. the that previously revenue trend NICE interaction blended about I number on thought with evaluate any of think,

Jeffrey Leiden

Thanks related. Yes. Ying. Jeff, they're for X This questions obviously, the and, is

community these you in benefit. applies tell, fall of medicines the U.K. certainly and it exactly the that how So of not early it, that number it's precision the for time revenue you well kinds between. that populations discussion about where there revised the ultra-orphans, I clinical to it patient too. NICE that's may respect well, to existing clearly, price. -- of per better different medicines give methodology, and this a the triple X from medicine over for not it's a that and, for medicines, question these way evaluated to you that but we more for is probably to to seems medicine about triple billion it's average a system, just there's one triple, of And I don't With in the to methodology XX,XXX than components in about the the quite value and in Certainly, another can the think patients to We to give the and going of kind data their precision revenue. clinical Obviously, evaluate for other patients, at that one clear major NICE generating in as anticipate but early on this major look we better obviously, of results as general current long-term evaluated. be hope to know is bit Parliament methodology to of they've haven't treating that discussions that, know to years yet next do we're think been there respect long-term currently the fact take couple They a as so don't the the already medicines. we, be about is methodology the to think on is has, the I the help, the price, know, triple little significantly but $X price. value think guidance guidance either, With of by NICE the blended too on described work

versus where triple certainly anticipate triple to major the greater, price benefit our we go we is As patients of medicines, don't in a given our to as the decrease value. world, other its

sort potentially math eligible around across XX,XXX we the And some move so XX,XXX can patients world. as look to might rough of to from XX,XXX do you like that begin or what

Operator

Thank you. And Porges SVB our Geoffrey next question of comes from Leerink.

Your line is now open.

Geoffrey Porges

level the on trend then a us you Jeff, underlying the And that sequential any just you outcome lastly, Is year-over-year products? be and alpha-X? in question. sense your gross you you belief patient dynamics the all update the in that for Or And net serum are if what the wondering so antitrypsin obligation the it you way there a just trend that if the across confirm just was? the different of prior see approval? CF? could a Wondering to do a follow-up to what will sequential Is we for next can benefit us in on a plan? is number study give could sufficient treated an could show alpha-X pulmonary just give that U.S.

Jeffrey Leiden

and the patient Thanks several give in the we're Paul gross will respect that order. endpoints. go Reshma think them to color do we'll And do a net bit you questions, years. the Maybe for a on do to forward over seeing question how some next AAT we on the Yes. reverse going I'll will with year. for penetration Geoff. will how

Reshma Kewalramani

too available for call Reshma. this is context work. bit could With in because a little currently be endpoint this can early how about to is what the we I what enabling Geoff, exactly and all of think the regulatory the all start program, might it's to But then So bit our you discussions. regulatory about you haven't to medicines, regard a will of that tell those engaged

So the only the has in both limitations as there therapies. opportunities has you going target own and of you it different for center anything infusion which augmentation liver liver really be therapy than doesn't that an do part its lung. which are it. is go to this, that really And about you treat make get companies is a able once to the is and to week protein. the our It into disease disease, approach, there know, of X lung that a out This

on been So approval based with able secure level. that to approach, have AAT U.S. all the companies FDA

So that's for that's go our a sorry, we it's through data to Over have to and you. work Paul. Stuart process point, to us. to how determine going exactly --

Paul Silva

Yes. Geoff, the question. thanks for

QX [it's XX%. So be think I we now said the gross Medicaid; and by XXXX, been] program. in our net was And exited discounts purchases expect statutory sharing to have that rate the And XX% approximately things: anticipated on in we the U.S. and second when between to call we XXXX X by would be the one which increased one similar that's driven and a cost is then to XXXB entities, XX%. spend

are course up things kind X of that the number those bringing over a little So of XXXX. bit

Jeffrey Leiden

then our how question, final to maybe do see And going think I going there's Geoff, go which the we medicines penetration X forward, your is phases. for really we with as be

label you've of a being as that heard the XXXX about the some about of expansion. continuation think things plus can new You

to guidance et this with year's all the to of year. to or the accounted provide England we've although most more also So of in a of X in Obviously, ages expect if What year. effect growth mentioned, a reimbursement there. for the is, see for for SYMKEVI, of seen into rate. on in early then nice positive big countries, might that. continued some world accounted SYMDEKO major And indications in bit not I companies, obviously, SYMDEKO label U.S. the growth that the our instance, younger will for approvals And think, still France, is the in year of for as one for these indications KALYDECO, then label any start would we're for U.S., in our here new able probably And growth obtain the the have have we were growth cetera, is these Germany, a of for is guidance triple. that younger and for we really for effect. There's in very off Stuart age, this a positive

treat ultimately believe drug. patients, of we we patients into group all from As do said KALYDECO lot in that, and which that as efficacy because we've eligible can of get XX,XXX going group like that penetration age of the looks significant that XX% the approval XX,XXX or see triple, we to a XX,XXX penetration of

think to way the forward. the that's think about ramp I growth So going

Operator

Markets. Capital from our And next comes question Brian of Abrahams RBC

Brian Abrahams

for the questions progress. me. Two on Congrats all

to from of AAT AAT will be the in degree humans term CF may was and question, the the impact the of polymers long And program, the will accumulation On liver confidence that model? most have you transgenic the you second in damage saw that gives reverse the quarter-over-quarter wondering produced prior what disease same then the the functional toxic inventory who help the if a just as you commercially, I mouse could portfolio? draw-downs quantify across outflow

Jeffrey Leiden

like question inventory question, think. take Stuart I I'll and the Maybe the AAT will Yes.

really like animal mutant a both you're the mouse So protein, protein. and about AAT, -- test our one expressing transgenic in questions. mutant correctors most not are asking cells things on the in actually that that able important we of the human the to And a those program we're is

measure can functional we And but can the the not measure So protein. they're engineered levels. with we levels, human only actually

move measuring folding, And great in a clearing -- that not to of protein, producing the protein by So data the package, these once this seen to human we're do measuring us of rapidly if which has we're humans. immunoassay confidence and high with had that's very to JPMorgan fact, the of not how we're the that's of and, clearing a produce in but mutant level only presentation, that piece the levels. we're cells, protein we able are and animals correct actually it, simply confidence you've produce functional protein. you us the that as we levels the saw liver both we're we functional important misfolded gives actually And -- encouraged my functional very, actually

levels of which functional essentially Now from functional we'll but said, to Reshma humans, really actually the do, is and only really protein be same of as probably the can going XX measure nature -- levels the by thing. micromolar know of not normal, these parents the express way, in measure they is anywhere we able an serum. we're from on who protective. a protein, measure fully We'll the protein And we obviously to micromolar from patients, experimentative are XX the serum heterozygous

in in can are. we we level of and question? data the trials, rather answer so the of protein And used again, confidence. that and, the But protein the exactly measure where cell these Did CF like levels your functional small know the with human us the simply mouse high treated trials, patients gives data

Brian Abrahams

helpful. really That's

Jeffrey Leiden

clinical at the we're the we're look looking directly the clearing. And true. and in in of said, that's at at mice, program, point livers confirm this I liver, some to we Stuart... if seeing And livers also as the of patients will

Stuart Arbuckle

And Brian, Yes. on inventory.

excess during inventory $XX the of was had at burnt U.S. about inventory end we worth typical first of year. here off of the course build more end the This million So the in XXXX, towards That of quarter. the calendar is the than

Operator

next the And JPMorgan. Cory question from Kasimov comes of

Cory Kasimov

of quick couple CF A you. for ones

patients? administration still stage now? other you CF what how switching much at the to about those new all, secondly, Do to of on there And twice-a-day pushback regimen important thinking have roughly out of with regimens launch, versus once-a-day percent from SYMDEKO altogether? do therapy First patients of are for VX-XXX, this you are believe you hear that SYMDEKO, is a

Stuart Arbuckle

Yes.

So those. take I'll Corey,

and versus the February those CFTR a you were high really a who here I'll to you patients that seen discontinued. to in SYMDEKO uptake only are in and who've those time XXXX. might naïve in tell because uptake terms and tried actually period ORKAMBI, who never on of for we where expect, whole modulator exposed is the we've switching ORKAMBI who populations we U.S., obviously, before, got the around of in really are been substantial So naïve, of we here of in What those of those launched levels data had ORKAMBI

we still say certainly those of the saw we one, able I uptake. patients is we I on would but that the in higher to And substantial real to we all had curve, of So the see expect about much about think, I'm with being here playing that that's the high than what's exactly day. pleased had world say levels out U.S., the in we of always to other profile the There thought I those flatter of are just we're added compliance, be all the would ORKAMBI. SYMDEKO given path world. now persistence of We're more launch categories not are every groups and flat. would say, seen very in confident that And real in

and many, disease ivacaftor course medicines, certainly, of the every uptake of In treating are is you this, which as which types clearly, medicines not regimen And something different is XXX, a know, people these our twice-a-day patients day. for as then as many medicines. indeed, we given terms deuterated of are medicines, inhibiting the rates important our taking see the from underlying with taking forms

for And difficult medicine so is twice-a-day them. not

to However, as get we to and convenience provide levels status can is carrier for possibly of as goal the our much patients.

as would patients something do soon so being be XXX why medicines, can. we advantage to bring we to possibly an that see want And as for and we once-a-day that's our

Operator

And Paul our from Matteis Stifel. with next question comes

Paul Matteis

you liver or benefit studies continuing your in is Just any in III just thus I far. early inflammation there compound clarify at the if go that alpha-X outside secondarily, if that look not hope the next liver to indications antitrypsin. of wondering might study pain the or enzyme not compound you biomarkers on design and NaVX.X the related thinking Phase ones elevations? for trend can on the I can was current wondering in detect are other or whether any you was after specific anything when you can pursued having whether then or And the

Reshma Kewalramani

Sure.

go I'll then and first backwards NaVX.X the take me Let AAT. to

pain. talked conditions did of pursue. about with I example, we repair approximate which these and the could pain think full as small to as trigeminal breadth other need the would obviously, conditions. hernia what some And we've that to is, musculoskeletal the of any the neuropathic other regard get design, kind actually the is And about models example. X guidance I what a the And bunionectomy neuropathy, of musculoskeletal regard to a in that III osteoarthritis, are, which kind pursue like or tissue you is of in there the hard sample are acute you fiber straightforward fairly size conditions good we studies course, to soft studies pain with there of neuralgia think way abdominoplasty it, be one bunionectomy, would could in a VX-XXX something and of for of do and of pain a one to model number an the Phase the the are think imagine but tissue, pain, So that

you'll figure I going what best us typical to question forward use and out is these path fairly is III Phase how get bring AAT, see think think So impact. whether molecule the there the not do development then the approved. to With or liver I regard was around we're to

out as a a It what disease, like known you interesting. biopsy, is and So from COPD COPD, Brantly looks which actually November is when disease that at this maybe that, just it that liver clinically is what over best did. liver or do that XXX-or-so in tell a patients disease is and down like looked and is Brantly something like can of is paper people. burden There liver kind manifest something younger time. Lab was There's that substantial the actually perhaps X about series in AAT a ago, you years of Florida,

AST, I inflammatory a doesn't is said at looking sensitive is our specific. not nor to point. you important give said markers, something very very it markers is what of think regard to the the that tissue. best, but GGT idea. Jeff doing we're actually that is with ALT, animal In it's Now disease, really models, actually be particularly good liver But very what liver

the anticipate not we VX-XXX. that and protein a important be of of of here, part go to clearance I that biopsies the an the actually moving. that that. guesswork We're looking cells studies, as we to I clinical going misfolded And hope question liver question. it's no there's are So enzymes with at answers with whether and then the see

Paul Matteis

Yes, it does.

Operator

And of Evercore question our comes ISI. Ravi from Mehrotra next

Ravi Mehrotra

franchise. question, for you around is CF of for broader pharmacoeconomic my arguments your Thank which taking possible utilization

real-world agents. decade your You've clinical now a with got over of experience

in So the of can you or that data taken se, color art some form rather resistant you science the an some per can than have on points us setting is give P payers. P to

Stuart Arbuckle

payers. that things are clinical lead our much pulmonary because, is to to exacerbations that also those our this verry Yes, Thanks Stuart. as kinds studies those into longer-term question. looked like of trials, antibiotics, things that pulmonary Ravi, in length also well. both very, pulmonary at and for things patients on follow-up of payers to obviously, those very and the include exacerbation as Yes. exacerbations, endpoints IV we've interest are what of you actually in stays physicians, but say, but are important certainly that they The interest dissecting the too for to are the lead like of hospitalization those hospitalizations, themselves

we've have we of KALYDECO, got being for but as on have market on a of increasingly large ORKAMBI volumes experience, data say, benefit we you As the And the with the longest now. well. while

things data like like And most so things we're on able data rates, on take like lung exacerbations, things perhaps even not which and take but able of to survival the transplants being only avoidance are to pulmonary outcomes, that. important increasingly

to that to of faith the newer requires to arguments. but it's it's leap modulator some that and utilize the right them And extrapolate world KALYDECO ORKAMBI to a to the CFTR with CFTR them for for I increase. don't payers building of a much certainly from into Obviously, think so other too base our of of now leap pharmacoeconomic it a translate are around because medicines We to data faith that continues of benefits our triple eventually SYMKEVI and combinations, bit asking of modulator. X

Jeffrey Leiden

Ravi, And, this Jeff. is

with you're XX% some we're some transplantation. with in things in long-term these are data like mortality, give that the KALYDECO decrease lung familiar slope studies of the around Just of decreases decline decrease registry seeing you not it. XX% in The if of XX%-plus to and that function, results of

are So models, of kinds real into And plugged these these of true not these results, disease-modifying impact. kinds when subtle have are are those they pharmacoeconomic results.

Operator

And from Company. of our question Hartaj comes next Singh Oppenheimer &

Hartaj Sing

with a well. the launch, sense U.S. SYMDEKO on United and question data better pace it. it's a payer of the of had talk than ex in been I has maybe for the you've And that States very ORKAMBI, than broader both got SYMDEKO, And that States which uptake with uptake I in my going just the in [indiscernible] ORKAMBI I world. think is you had to appreciate the the can a Or would little I'm just and Europe really been Maybe bit. and mistaken. quicker United rest

Stuart Arbuckle

Yes.

say, and each label of in get moderators, the and very we So time. access and KALYDECO even CFTR individual that to the for now our have starting ORKAMBI to over I broad to always we've would had U.S., expansions SYMDEKO through managed with

are a these difference. Certainly, payers disease, that's really the more getting think there's and think to don't accustomed and I I to So substantial helping. think I time agents very

actually think not some -- you're I we huge. out think seeing couple a outside things. So What U.S., the the play seeing is of in U.S. but acceleration here I are it's is the

of and possibly regulatory medicines benefit the and patients time reimbursement could were and were approval agreements, our designed physicians exactly the newest so to they what they as lag approval seeing soon to between do. They designed best you're reduce to our portfolio was and as the can. access Firstly, this

And separately, in is SYMKEVI that launch U.S. SYMDEKO or ORKAMBI. Germany, versus so launch than the actually being the we able SYMKEVI what ORKAMBI. to what in leading And of then you're substantially going the to of outside better what's with patients access think is I acceleration the seeing is saw

job also Certainly, SYMKEVI launch job terrific there. in profile team And has agent but of an at the above certainly, some in patients. tracking a initiations patients seeing done initiations there has the team executing point. the seeing naïve done is ORKAMBI We're discontinuations. substantially think and well. time benefit-risk launch had Germany, in I the same the excellent who we're the helps, the seeing as We're switching

team think I example an the of so well. really really executing that's And

Michael Partridge

Operator, more we'll X take questions.

Operator

Skorney our question comes Baird. of Brian next And from

Brian Skorney

thousands to kind XXX you'll know I of before, wanted move but you with. safety tens around it's a you. patient efficacy just of of chronic mean have which NRTIs we've XXX this the again forward on I in therapy, picture kind really and you kind of HIV the TNF-alphas, full consideration whether get one Jeff, of of of in until little push or behind years we talked and I and don't ultimately

So and I which submit guess there is approval enough database we my better differences? triple -- start build to have information just and decide if the subtle question there's is are and why physicians both patients combination that for to there not decide ultimately let out when

Jeffrey Leiden

Yes. Brian. It's question, good a

before, we're to have is about trying data it good that and simultaneously said talked news what is confusion balancing seeing before. of versus And the a what I patients Reshma when same confidence think how this information launch our We is good little efficacy drugs just X the level of in the bit the is. here, has into And potential population. the and we're we're physicians

whichever we're far endpoint, to launch while, of out high turns true very, asset for choice as we're the X so for know, superior I simultaneously to so, you one from similar. explain can and once you as across patients withdraw best be around into And And very know these if XX-week this trying very because so a to going they're to that, of X the other be a level all side we ultimately seeing patient primary is when doctors decision make ability to hard equation, impossible. of going confidence we're we I efficacy and that patients. X the good across endpoints, seeing to at a be do but then population the not drugs your drug the as is very, virtually it's And being on and that And confusing. what choose have only very that secondary look think kind the data, is and

committed you're identical that patient the I best look do drugs the long in X term, having on and to probably for market so think different to, And not populations. And patients. that's thing

asset patients what bring And we decision it's of it hundreds is best end the to to data patients at the day, enough make have like going for we them. going to the we're the be So feel confident see. in so on best now the decision to and of that we're of

Operator

and Carr last our from Company. Alan And of Needham comes question

Alan Carr

for, with can a VX-XXX, wondering if you're waiting compounds see these are move the here? have more looking bit what you what that in what's this to for of with little relation elaborate there. And involved detail VX-XXX. was in that? around you next-generation in different terms I what's too process with Why more a hoping pain you're program, forward to And you decision to about maybe then

Reshma Kewalramani

Sure to thing. about question, going over is going first to to talk This and tackle the Reshma. Jeff it I'm I'm pain. to toss

patients regimen really level. important. made, that that and our back ties to to we bring point CF. all bring what the really, patients And is to do therapies want long-stated with goal, all is remains in Stuart carrier we're level. for carrier patients most That's bring today, to brings trying know, to convenient you as also there, to it So that And a

many have is of what you we And them that so HBE in have translate data cells doing a of results. of Phase have and we now we're assays are to how San assays labs plethora and III the seen is these II our our Diego in Phase

And advance meet high the so molecules best they goal. ultimate VX-XXX we extremely And high are the When we bar. of an best there set it, our pick molecules thresholds that this have clinic I this them to bar, when labs. -- come that we admit out to the and these like of meet meet Jeff?

Jeffrey Leiden

pain in the to it's respect before to about was question, similar of what With talking Yes. terms a bit modules. Reshma portfolio

here have, with we we and very say others I convinced have think, approach high that would First we've ourselves and efficacy thing molecules the is I taken. XXX

were is VX-XXX were reasonable convinced high -- compound. we these we've we're addictive not for drugs. a a potential of very bar I patients that ourselves think number things that the convinced also trying there's tolerable, and with to highly match high Those new of pain

the other in we're things. drug, best involves a of and picking Now of process lot the that

and So scheduling. p.o., for dose dosing, example, any cost issues, IV goods of both formulatable issues, manufacturing is whether it

that high we'd pretty through to that to of in want right the best molecule one It's molecules, p.o. in lack dose, hospital. bar, efficacy, really we commercialize. of that the and regimen, molecule, we tolerability, is III the dosing right transition but a So take we pain, but like the addictive the combines we will what also potential, portfolio find that's a to IV have Phase whole because to have and believe the

Alan Carr

running any studies It's the efficacy or... not You're Phase stage NaVX.X earlier compounds. I with just

Jeffrey Leiden

pretty enter Phase Well, decisions have go a again through hope a Phase that but so learned the VX-XXX want way, as we will take taking this with make We it we'll we clear portfolio forward. to them. to and that to mean, hope get And just to which through right? they're I to earlier, about VX-XXX VX-XXX, II molecule that enter programs and be it's So doesn't -- compare them quickly our the we're is I, lot year make we and best they clinic to that not approach. from by the able experience

Operator

and to over call session. our gentlemen, Michael I closing conclude Partridge And for back ladies this any now the would question-and-answer turn does like to remarks.

Michael Partridge

Thank team you questions. Relations for office joining tonight. all you the here if evening. good a call Have have in additional The the Investor is

Operator

and in thank you does Ladies gentlemen, participating today's today's conference. This conclude program. for

Everyone, disconnect. day. a have all may + great You

Vertex Pharmaceuticals (VRTX) 30 Apr 192019 Q1 Earnings call transcript