VRTX - Earnings call transcript Q4 2018 | Vertex Pharmaceuticals Incorporated

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Michael Partridge

Welcome to the Vertex Full Year and Fourth Quarter 2018 Financial Results Conference Call. This is Michael Partridge, Senior Vice President of Investor Relations for Vertex. Tonight, we will review our continued efforts to develop new medicines for all people with cystic fibrosis, recent advances in our research and development pipeline, and our 2018 financial results and 2019 guidance. 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 Paul Silva, Vertex’s Interim Chief Financial Officer. Corporate Chief Vertex’s XXXX. Officer been has Controller Accounting and since Paul he worked that Group have his time. with And team Relations closely and the Investor since

website. Following website We call and recommend Dr. you access The and Officer prepared conference slides is join listen that Jeff, a on to webcast be Chief call. our will Q&A. recorded Kewalramani, on you replay Medical Paul, for us the remarks from as being our Reshma will Stewart this

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

Jeff Leiden

Thanks, evening, Michael. everyone. Good

the Over the you transformative strategy by progress made to drivers that pleased with this for and the have past expect you I discuss evening strategy multiple executing record diseases we to our in In VX-XXX create and significant new combination continuing clinical we regimen innovation. research several drug for a commercial different serious a well clinical, combination Phase to leverage midyear. long many will I am shared and milestones track than submit medicines to CF and CF, data remain triple no include the the obtain expect XXXX to across important track Vertex’s serial years, as to of data which review the growth from XXXX, on for quarter later and we application X our triple this on important development pipeline. in as reimbursement with

also our initiating more to treatment XXXX. expect see with We patients medicines throughout

And label study which revenue and potential pipeline, XXXX key In X inform of from will development are global ranging of have in of a development initiated the XXXX, a I correctors, our dose Phase including studies expected expansions from our Phase to drive X each first and that briefly plans treatment our sickle agreements AAT enrollment we further thalassemia, beta for of in in expected we reaching VX-XXX and you pain. half reimbursement with year. review disease second these and molecule this As evening. first CTXXXX. in XXXX our editing enter programs of data both now advancing of result is Phase growth of the this molecule non-CF of pipeline will year. portfolio Xb In our our entered Phase of program, X/X gene small will cell this development late clinical a

advance a of in all up And our of to that disease. combination fibrosis, on treat the to CF of dramatic the yet with of we disease the half First today verge stand all the to potential triple Vertex CF approximately with are for XX% eligible people medicine. another hold people treatment this with cystic regimens

XXXX in progress was two Our in CF by important achievements. marked

benefit regimens around reimbursement combination for successful U.S., or corrector VX-XXX. X this showed the This treated studies next-generation regimens for studies. development. first through patients or plans regimen a one the Phase two Phase regimen VX-XXX the initiate that either of we the our offer and and we being SYMDEKO year, will a in saw across in triple launch the clear development well this include ORKAMBI, those ago benefit Phase triple key and ORKAMBI. announced multiple which It treated with in KALYDECO In of provide number obtained already world, the two may completion a mutation medicines. increase and year Stewart minimal significantly that combination X FXXXdel agreements as moment. who with of was evidence progress approximately on completed X as the just X are our the X second, and combination function was in the And data expansions X may with VX-XXX of dramatic patients X,XXX X for advancing one label FXXXdel SYMDEKO our result for the These mutation we being the that review we to a made enrollment First, patients call excellent enhanced data with mutations compelling Phase that of significant two triple different approved

approval. data new We a submit VX-XXX will the which for remain a later to us year, quarter obtain track will XXXX. triple for no We combination regulatory enable for regimen on drug the submit combination choose mid in this triple of the best to first regimen application regimen than to

the even profiles. for high the including once future, that triple are CF but also daily triple better other next generation set correctors we may regimens combination have committed regimens contain medicines bar with data, have that combination We a enhanced and to creating

future and in CF. have development for We treatment preclinical molecules of that improvements clinical provide may the multiple

portfolio are lessons sweat and believe the that We our with to believe and in the with de-risk our learned people just us in CF alpha-X to with clinical medicines of development discovery of CF of outside a the AAT. many chloride potential recently disease be similarities and serious apply start our program rapidly us diseases large initiated will with protein programs of our advancing efforts progress CF cause we of where measurements molecule of will development development able functional small important for and we medicines to we preclinical and will for treat I range and provide of first between recent and this our clinical marker activities deficiency a AAT The did biomarker striking of CF. where AAT the an need. circulating our AAT that antitrypsin across early medical the we potential new targeting of our late are early to as unmet Now accelerate through corrector.

different NaVX.X program neuropathy. in proof-of-concept of pain, study further enrolled reduction surgery half to in and announced in following Phase VX-XXX in development from we study bunionectomy conditions our this recently validate expect VX-XXX acute marks people significant from data research X inhibition study pain the in in of development a fiber potential of positive VX-XXX Turning third pain and results the dose-ranging with this we Positive for small study could showing first A Xb this for Phase role year. data three pain in positive of now of fully pain. of the the treatment pivotal VX-XXX and pain. data support is These

in development a NaVX.X additional preclinical in expect inhibitors advance of XXXX. portfolio and We multiple to of have into first late the development molecules clinical these

are pain We discovery significant of new potential targeting invest continuing medicines. need for the other molecules additional pain new mechanisms to in also given the

first to these our these programs technology gene-editing trials Moving we disease with CTXXXX beta both two studies serious the important of of These partner diseases. thalassemia initiated evaluate represent and clinical Therapeutics, in using on a treatment clinical the studies diseases. cell now to the CRISPR for sickle have where CRISPR/CasX

provide to are we technologies, development or evaluating these constantly external access new that advanced we opportunities R&D new As platforms efforts could assets.

pipeline Genomics We and into and technologies. KGaA, entered announced aligned Plc our Arbor our increased to including Merck in to X-Chem. that entered explore with with Biotechnologies, as us flexibility broaden enable recent multiple well continue to deals do with with serious years modalities recently diseases have further XXXX more our CRISPR multiple with are would and agreements We collaborations strategy, that Therapeutics collaboration as

our look medicines. years, significant for approved mid treating growth Vertex potential more based on we earnings patients future As toward and XXXXs CF future and solely revenue through the the has with

I Importantly, I scientific advancing to now that to the and at CF a diseases progress. portfolio call long-term that innovation drive innovation. Vertex, our of of review created Stuart efforts transformative Vertex to rapidly pleased believe over with shareholders is potentially forefront future other medicines growth. the and external enhance we internal to real for serious we am patients will value for to the have R&D through treatment If commercial our turn ability and diseases. And serious of transforming be continues other also

Stuart Arbuckle

SYMDEKO continued quarter. to with this of XXXX. revenue growth included in globally. SYMDEKO for being which pleased we review throughout million guidance strong and primary our for CF evening, XXXX The and you are in in XXXX, medicines am driver revenue $XXX I in performance Approximately, the quarter in shows Jeff. with approved U.S., by revenues CF as treat million patients with growth the the from our product also U.S., fourth revenues our significant fourth was commercial in medicines patients XX,XXX our Thanks, launch resulted driven more and which quarter currently of in CF this revenues the $XXX the of treated the the

number SYMDEKO those initiated with and on compared come who patients Our a or remain eligible result of from full of billion previously for direct never U.S., patients including XX% the groups XXXX. from to ORKAMBI SYMDEKO discontinued all ORKAMBI CF and increase in initiate were or is for Demand large those to treatment billion, switched $X.XX more which a saw has XXXX who a of KALYDECO SYMDEKO. many patients $X.XX year the XXXX, we revenues globally. treating In

out profile Given And this than pleased play previously SYMDEKO, the we might compliance of to with be rates greater those we persistence real are expected seen that and world. ORKAMBI. see the in had

$X.XX of and XX reimbursement received KALYDECO increase multiple countries. to reached other XXXX the now in XX% Ireland growth XXXX. the in in of to expansions during potential children well allowed I and reflected patients Germany, the reached XXXX, provided beyond. which direct in launch a label is CF time younger are also will agreements an we for medicines more into and have Sweden will SYMDEKO, Australia, and and Outside we initiate X agreements as result is access important Denmark contribute treat U.S. cause These that as in growth These older including well KALYDECO treating additional XXXX compared guidance we a to to Italy, discuss on While our treatment Netherlands, based reimbursement ages the and to adding patients’ the revenue first in we for for contribution the were medicines anticipate XXXX. new revenues, The XXXX previously agreements in revenue in will approval the ages increasing disease a throughout U.S., agreements following to billion, revenues XXXX XXXX. do underlying of patients in XX, anticipate years for moment. to patients of their patients

our pathway and approved remained U.S. EU recent triple but obtaining progress immediate access SYMDEKO ongoing rapid SYMKEVI the only Given approval the the reimbursement future known that of not CF to medicines, focused our also agreements our portfolio a to efforts regimens, in in as reimbursement access rapid for on developing in provide combination medicines. long-term

win-wins We and as to countries they governments have they reached and view patients in for future have foreseeable agreements for certainty in agreements of to patients Vertex these to and future. that and types current from governments will immediate the budget multiple certainty these access innovations provide CF as

has agreements Our for launching the growth SYMDEKO for important XXXX. revenue continued ORKAMBI reimbursement U.S. outside us the XXXX our labels in medicines for and progress in KALYDECO expanding in achieving and positioned

growth the for is midpoint CF product at XXXX. which guidance over to $X.XX $X.XX XX% revenues XXXX Our reflects approximately billion billion,

channel provide time. the I when our this medicines first would in guidance agreements revenues would a the Achieving of revenues that not and of in our thus occurred achieve at only $XX additional As our as at we build adjustments, experienced the impact we which million from we our that to reimbursement from reflects of update first in reimbursement regions expect typically approximately XXXX net and end inventory future revenue appropriate gift, currently from timing that higher upside XXXX in negative gross our anticipated the quarter is quarter. are see note year to reimbursed. could entirely where agreements guidance to XXXX of we

financial the may for our these am full coming CF pleased made year. growth medicines of growth call our other revenue further than We continued expectation which to our you the with for globally, than the lower resulted I continued to strong dynamics more and quarter review many guidance. we revenue that have creation revenues turn of as guidance. look more talking bringing throughout in to And further our year fourth to noted expect has now be in support first investment revenue patients diseases. quarter to I for new the growth despite with new the Paul we results our CF that from patients CF forward in in the progress over offset medicines XXXX product and and could will sequentially

Paul Silva

financial Thanks, cash which XX% everyone. drove evening good disciplined flow. with operating marked operating coupled in by into performance Stuart and in was revenues investment Vertex’s growth our and increases CF significant XXXX income our business, in margins,

In to revenues and guidance SG&A for our on rate. XXXX our evening guidance, tax XXXX will addition and financial additional XXXX effective also I Stuart’s revenue results. I expenses will comments this XXXX remarks and financial discuss regarding provide for R&D our

are All of will I the results non-GAAP. discuss and guidance that

in that patients and the also where by KALYDECO revenue since in saw lives contributed XXXX, important medicine of we its for time launch As patients. around the driven Stuart and in in the KALYDECO label billion younger both the approval $X for in U.S. expansions revenues role the demand revenues the to its first KALYDECO the underscores primarily of product the label thousands as the increase continued SYMDEKO globe noted, reached strong XXXX. to growth we new a significant plays expand of CF This

quarter $XXX of XXXX and quarter R&D combined SG&A were in fourth to $XXX the compared expenses fourth Our million XXXX. million

CF XXXX million of to compared income to in and was XX% fourth $X.XX The primarily increase billion $XXX quarter XXXX XX% XXXX due as $X.XX XXXX. medicines revenues in triple support full XX% the globally. of to our R&D significant well compared our Net to for of for in combination as expenses year year portfolio of growth million a investment the for income of the in of fully advancement compared margins operating XXXX SG&A The $X.XX XXXX. were and compared in use operating to resulted expenses regimens Our billion, to $XXX increase these XXXX. was billion

The growth compared in Our in product full $XXX net to net strong XXXX. full a significant in of our year XXXX quarterly million result income $X.XX year was and was billion income the CF increase revenues.

also compared $X.XX We marketable securities the billion of in at the billion approximately XXXX. with $X.XX to and end year ended cash

SG&A tax rate. cash anticipated guidance, for XXXX well as and and for our to XXXX generate combined R&D Now, expect continue financial in to are We product we significant Stuart revenues CF guidance as beyond, today, to providing our as and expenses discussed effective

XXXX billion We support expect our combined efforts, R&D increase The of to combination reflects incremental to investments triple and support investment regimen SG&A a expenses primarily $X.X CF development the pipeline expansion billion. of to compared and diseases. the $X.XX into to potential launch of additional

losses. based guidance, the basis, a of a the benefit recorded in to quarter we of net related valuation fourth to Now allowance tax billion $X.X the GAAP our XXXX on on operating non-cash reversal

The we non-GAAP Following GAAP The and to until rate occurring fully will the and the release with year XXXX valuation year provision this of we tax our expense in allowance, quarter-to-quarter operating a also vast our be XX%. to highest recording this provision the of tax losses. net XX% begin will be our tax fluctuate non-cash tax will majority rate use full fourth in rates expect quarter.

Jeff. that, has forward I Vertex earnings that you a noted, our look and continued flow. in resulting on the Jeff unique in and based to progress. growth expanding With growth is long-term As back on to I in turn CF operating revenue increases call margins updating cash outlook will

Jeff Leiden

closing, and Thanks, across business. XXXX year Paul. of In successful commercial highly execution our clinical was a

CF XXXX other for operating our recent across income and both and Our R&D in diseases. near-term positioned important beyond portfolio for growth in and in continued over have us revenue and achievements months in milestones

well is business. seen creating exceptionally that, working medicines the progress by our evidenced I to I you With as across line the rapid Our year. serial through to questions. innovation updating And forward coming transformative look open over of strategy the will

Operator

[Operator of you. question Instructions] Thank from Our comes Yee Michael first Jefferies.

is line Your open.

Michael Yee

that could out maybe myself? equation? a I and the will the address. getting Hey, you is you combinable, ahead Two those been of is UK better itself and about recently one thought say what’s help speaking nicely And specific very safe region say whether thanks second pharmacoeconomic topics in more question be are that coming about a value will giving AAT did Are Street talk just broadly that pipeline you some confidence obviously I news the congrats to the question so second for connections solve having on work a quarter. in. that, they great similar, talk maybe and to there comfort question One is potent, Thanks one or than the drug to much. how am more better two

Stuart Arbuckle

Mike. with into Hey, reimbursement we like agreement want and year the today, and Israel important UK, results I secured reference in do agreements to the reflected and we XXXX kids have Last on a and in I go you in new UK. progress made continued were XX Australia will on Denmark. share the great view ORKAMBI momentum the first from where places for Luxembourg question we year I and XXXX before that Sweden agreements of and places like in pricing It’s X for mean, old point take have Stuart. Germany. we to in I to able to that’s the

world. continue methodologies would we we using to patients medicines pursue energy continuing value and because real can with bit get our every assess eligible we is the and to are getting that have make Where successful to we of to to transformative we appropriate UK. of goal the these progress So, for is unique due in access access large to what great countries in those patients consider the been for around be part

have and the is a a had the process. own so each important UK known probably country healthcare think developed up UK Mike split to managed from country, as you I it’s its perspective,

Scotland, in have progress. made we So great

with We on submitting we verge an government we methodology, going going to we not in and have submitted they In of certainly about assess England agreement give optimistic and appropriate and an unfortunately, hopefully reach reached we on consider the there agreement that. we how the get them to XXXX. ORKAMBI but to will would yet medicines are with are Scottish up access them an be to have we what are our to are SYMKEVI on

in good valuing methodology, the for say we fight will of don’t study November. look in you that those I are we anyway have England going close patients the that out we into the triple in around And nice even We we continue if to for the in combination if to as Phase when nice last model which access the saw come world. gets of the the will to put thing The know as are released transformative a with put think the to even with to as get results XXX X existing is results we results regimen through rest these medicines. what the like we valuation patients even nature

even triple, we to need for England to work to medicines. continue to going change So the the with for are all assessment methodology

Michael Yee

Okay.

Stuart Arbuckle

question, the I to On over AAT Jeff. hand will I think

Jeff Leiden

cracked saying then of from Jeff. biology important question Thanks our program pour on next-gen AAT. a very of the it’s and had I right have learned you the biology the of can think Vertex many Mike, ways, on this chemistry. we AAT. Hi, In We think we believe sort the crack we that lesson CFTR for as the

We correctors functional and from form. cells protein in a secreted that be causes have can liver reform to multiple the

right a better experience tolerable. than it’s ago Vertex’s to portfolio the molecules have just our And can a clinic multiple on if one is get risk and you next-gen they and that be take molecule with because chemistry [indiscernible] and that following started create that action CFTR half reasoning we the so more as right to molecules. about into And the a like predominantly and mitigation efficacious, the drug strategy and will. as the are protein we And they these better and year obviously the just have

the is with most the going strategy in multiple our cells clinic and chemistry is to molecules. the a programs, them we’ve in forward AAT Bring the idea on get of with CF and molecules. early so, which be of frankly small the going And best and quick are pour those pretty forward compare create

one think going sort And think and so, don’t better shouldn’t of properties. different because way compare them, well we that we putting get to molecules be we them and to I we’re as continue of just and these surprised of see by as third forward as XXX better really molecules to said, us added the we a

Michael Yee

you Thank it. so much. Got

Operator

you. Thank

Porges Our next question Leerink. of comes from Geoffrey

lines open. Your are

Geoffrey Porges

Thank question and you very great much on the for taking results. also my congratulations

ORKAMBI from And how patients, you our XX,XXX First, And a a we ideally, should for of many Europe? and just by KALYDECO, the the of main question us for that the beyond could comfortable to rate models XX% going are you could you us many breakdown sense you geographies. U.S. from give tax give the forward SYMDEKO secondly and then Paul, come how XX% that in model XXXX. come

suggest. think you that. be to what that’s about want I clear I

Stuart Arbuckle

Yes, now questions one-third in first are ex-U.S. I couple of the there being it’s take and two-thirds the about it’s patients U.S. XX,XXX will about Stuart. the treated who actually of

$XX the for the QX revenues $XXX.X products of was breakout geographies was for terms U.S. of the a million, in KALYDECO our of ex-US QX the terms In the million. of

the U.S. U.S., just in that was reflecting the and And ex-U.S. million. execute million $XX $XXX we it was ORKAMBI, launch was and began Germany. $XXX was largely For to $X million in SYMDEKO ex-U.S. million

after breakout So that that’s quarter, you to to have with up call. the be catch annual want happy I’d you ones, the for the if

Geoffrey Porges

very Thanks much.

Paul Silva

detailed your know question, you to more Hi, for about to let a just kind little And in specific of how we’re Paul. Jeff, question. it’s thinking Thanks I’m going give the bit taxes.

not our in rate be due in income revenues a million you is for future majority to it’s what expect business XXXX, visibility the of cash by expect to full on XX% So, of globally. years, primarily the is effective XX% we everyone Europe. give that as actually year. effective driven and tax I’m the the to advances continue our or to we the provision. U.S. rate going able tax could lower And to in actually grow non-cash that mix And geographic of be expect go federal non-cash point to to make want rate get we’re then other reimbursement guidance And and the XXXX. rate is $XX taxes pay that of I the pay until compared just of we kind the $XX million XXXX tax in to we approximately going only in to that for

Geoffrey Porges

Great. Thanks very Paul. much,

Operator

you. Thank

from next question Phil from Our comes Nadeau Company. Cowen and

now Your line is open.

Phil Nadeau

thanks taking my questions. afternoon and Good for

portfolio triples that Just labels expect guidance, an in a you you the Can agreements for is coming on the of about couple agreements portfolio to get drivers, you second you can much geographies in question negotiating referenced. us versus and from Do is revenue wait from give it before portfolio revenue territories? which existing have you that agreements you growth approved? XXXX? the on so remind the is how example then proactively do Thanks. much for you label approval coming existing growth are XXXX additional sense country currently could in us go how of and And or to something expansions how have is with some

Stuart Arbuckle

take I’ll so both Yes, of those.

don’t to ages be within are the the sort for three would we that vast notable terms U.S. the also countries six the things. today. so those achieve So a in probably it is is saw a label able in XXXX reflects XXXX, SYMDEKO be of analyzing agreements as large to to guidance of contribution were and really we not launches contribution expansion from are One the of there. are in guidance get SYMDEKO assuming patients that and we many whom that effect KALYDECO reimbursement the we new new where that and expecting both in most have ORKAMBI coming to But XXXX. in launch is, of In in of into annualized our a from we expansions of patients XX the XXXX, label majority new from XXXX ’XX the label because we

to seen update to like we Until, you’ve upside today. if it in Phil. you able revenue material, portfolio were and one get also if portfolio would the significant reimbursement have only long rich reimbursement companies geographies to assumes have have portfolio we at well, an then revenues XXXX new we our where with agreements see Just in reiterate they our agreements that were as guidance agreement potentially time. agreements, If agreement,

So, it would be it’s more, where for there them getting SYMDEKO have that slightly all are describe is places difficult and different. agreement agreements an they we we in a have Australia the different a instance Denmark, in Netherlands the reimbursed, Ireland, approved number so have to path but when we like to by TGA. portfolio of the

which are a of physicians about but there in these well looking for as early could level been advance our and those of them a agreements of win varies that’s do But actually those is is, from country. to There countries how the in In wins Indeed, terms is aware combination like remember as of been approvals on, choose governments talking win expressed country we And governments for markets regulatory at soon regulatory quickly that advancing the look and as us, so results patients regimens be how us for that during treating need that agreed. would kick they get win, patients to win, regulatory agreements those because approval able approved who that, has that to again portfolio a patients. for and to many the triple key have CF are impressive and have and the to government. we to we the is approval. in one of get by best in our there interest is reimbursement portfolio has portfolio they win even be for give sooner Vertex medicine one budgets interest England are significant agreements. to agreements countries, of an portfolio The

Phil Nadeau

portfolio the agreements, you That’s helpful. and Australia, follow-up one Netherlands. mentioned Denmark on Maybe just Ireland,

sales, well revenue territories as from So, be as those four would model XXXX it to expect we Germany? as reasonable SYMKEVI

Stuart Arbuckle

EU So, approval agreements large we be ORKAMBI where indicated and in remind in that parts, the in is quickly. would for the regulatory in those for XX Ireland portfolio countries place, you instance. are where have will places I for a we plus population in SYMKEVI get as to same like access relatively able

On seen the uptake we of rate. very high ORKAMBI persistence Netherlands, and have impressive very

trading choice medicine. up, patients a And so whilst will and they be the will best they is essentially have physicians which think

Phil Nadeau

you. helpful. That’s Thank

Operator

you. Thank

Alethia question comes of Cantor Our from Young next Fitzgerald.

now is open. line Your

Stuart Arbuckle

are Alethia, mute? on Hi, you

Alethia Young

you hear Can me?

Stuart Arbuckle

We hear now. you

Alethia Young

Yes.

Okay, Maybe cool. wrong my with headset. that’s

a I I internally, on I I talk talk coming thoughts you have just anything the a apologize. kind about is or mean supplemental you a that wondered it’s Thanks. bit M&A but delayed is of might position that? until little obviously your about what doing together if and external you CFO Sorry around wanted like interesting kind little out kind bit And also to pipeline of well? of think M&A you figure just then could as

Jeff Leiden

Yes, I Alethia, is will Jeff. those. take this

around M&A it’s with what couple of talking As just I years, innovation call been etcetera. collaboration about external really you focused licensing know, for we’ve the strategy, three last areas. not a consistent,

current particular simply now yes, anything where less to high our a us. is obviously less with bucket to the seeing interest data. of be complementary bar regimens would set we and or we’ve be combination in because that we’re additive feel would first triple such The Honestly, that great

we’re the in tool pipeline. what very seen those different all are The tools deals, you and us the to that it’s and build second interested of think allow Genomics box I to diseases diseases do and Plc. had effort of Arbor, us deals And some let so our and acquiring serious X-Chem, concerted that number will genetic diseases call technology a we we’re is kind a of Those CRISPR, in. the would that technologies address and think interested of that optimally should address extent to a complete area you’ve us tools

consistent those think pipeline doing late-stage clinical pipeline would value be commercial need transformative should acid is do be products You the X our of as where should next with on-market us but gene years nucleic them provide interest, CF specialty regulatory strategy to to that to third by What won’t through assets And that. we continue the some therapies development can expect expect diseases overall in for find obviously development growth growth, of etcetera. we And going to would medicines see in you you those we And our the see assets that essentially do markets. and areas therapies, area years then us for us are and see stage add of serious that to led simply X because revenue don’t over general, groups. revenue or we deals earlier is assets.

around us that to would do obviously building focus group do continue up those firepower expect that. deals more to you are we to say can so substantial we’ve And ramped financial innovation external see allow those do of I areas. us three our deals to to and

to expect advancing fair internal think for with rapidly it’s us pipeline some our see as do very to innovation And complement you well. external so I

Alethia Young

a Very Thanks lot. thorough.

Operator

And Barclays. you. comes next Geoff Meacham question Thank from of our

now is open. line Your

Geoff Meacham

just is In I at is Thanks like to words, that year half. the follow-up. the up all. between about it the anything for push NDA guys. still And end the other is or know and coming or plan pulmonary obviously your metrics for of ask points or I select want that or perhaps have of when if XQ non-clinical to the your of the And like? maybe two guidance? be getting FEVX about could still less be second in question. the first could the you done I of the yet differentiation something Hey, to exacerbations triple part at factor versus there then terms look the has package, have the the you to half in of one or also the changed that

Jeff Leiden

compare drivers top-line very is of line sight and to to see have that the With This a which the quick fully can when I quarter. to – going you to the again, assets, not take but in I’ll respect Thanks, Geoff. good the data first is just the first take the we maybe part XXX review, there, enrolled, we the of know we expect timing you two this, Jeff. a how just second Reshma we’re part.

very to reported we’re we’ve of that so confident the XXX. that And that we have already data for can kind we’ll data compare that

assets efficacy I also if PK, course we’re It will at like of like things these As all well the to PD, but the as chloride. that both way drug-drug two said interactions, you as of going FEVX, you profile, work. preclinical before, measurements, goes complete look talked the that with things is sweat about and includes back programs the tolerability, to way

review assets. So, a both very this complete will of be

once We’re having forward. able the on move very do to first assets to a nice we data the we’re of I track in both on again, file asset in pick much then think we’ll a the and be get And best to XXX quarter. position that nice complete

will address and first to decision would able let NDA with quarter, the I’ll that schedule We’re to confident the we of mid-year which there. package respect that the be make the what’s on filing. Reshma allow question that in part But second

Reshma Kewalramani

Sure. Thanks, Jeff.

exact So, confidence in happen. something to of of we is to going kind or NDA same get already VX-XXX VX-XXX, year, mid very VX-XXX the the very going complete and I is for we’re have we’re that’s same we the going know to we amount data that QX, happen on to with of the making are by regard that’s we regardless data CTD the package, high for need all shortly the that year. last that of for this we’re data whether to XXX. And track shared packaging, data have late going have for that of the file, same That sure you for and in the the selection much

Geoff Meacham

has know reimbursement, some have maybe maybe community Okay. of put differently a payers oh that in a the do just little had But ask and on methodology And to pressure defined France. past, was the the for you follow-up, been looking or escalate allowing can OUS, a the to kind things this triple, something then the could guys sort about a process? of reimbursement patient case asked the I a and to in maybe you bit of help strategy pretty is just the wanted I in do know to asked, you you this different accelerate and speed help just there up I UK differently obviously

Stuart Arbuckle

back very assessment well Yes, said Geoff, people kind it’s aware And technology be the quite no officials that the they the well very Stuart to assessment lives, methodologies medicines. benefits, for very used purpose it’s long-term. get levels here. patients hoping very, chloride like strategy these to of making, types refer clinical community on going know, development I them get normal what assess we’re are of their I and of there’re fit view of early of the secret put the be so to these in as that in needs the transport restore that over is and to cystic frankly, are those point wouldn’t aware the to really progress products for earlier, access you I to medicines. fibrosis, our to to of appropriate here to not people’s are benefits really a of are plans deliver to these health is which types anybody key from medicines, rightly to methodology think our they very delivering look pressure I calling elected say on

component that many is to been is technology what on then other is, where where point been this and successful. I So, And around is in thing are view, assessment many, that we’ve we’ve from which the world, work the important countries done health think a successful. an we’ve of continuing of that we

in ministers and of their developing of and populations. able our see government to this issue bring it’s the have portfolio how being kind think I it’s the because it a substantial senior fast portion could time, taken benefits to interest an

me of are else the the success so And to the and most those everywhere factors world. in around two important UK

Operator

Thank Stifel. comes Paul you. from And our Matteis of question next

line Your now open. is

Paul Matteis

taking of can VX-XXX. work my so stringent Thanks much question here. Great. the on I talk more you guys about for all, doing logic Two-part guess of question. first

move an Phase need big unmet you we Phase I for have were positive AE why And into that wondering not and faster X, Given why a X? low then if it’s options table much. out-licensing that least secondarily, a you wondering as all alternative, there’s still yourself few a the have targeted opioid a just on an still or rate, Thanks strategic far as and confirmed to at VX-XXX a you’ve picked with was possibility? so subset –

Reshma Kewalramani

Sure, Reshma. thanks. This is

your pass and your I’ll question. Let question me take half to the it of Jeff the second on for of half then first

is dose-ranging you So, small in points. find bunionectomy. us pointed in our very in very with regulatory. regulators. inhibitor as a so out which lowest to VX-XXX and around do successful Regulators as out to is practical simple dose one two looking point are really expect dose-ranging we now reason One studies had that well is neuropathy three that to osteoarthritis they’re regard doing The we’ve efficacious NaVX.X three for us really just is the what to is and do the fiber

of drug a high a just read-out get we did chose in proof dose. is we a around second to development. What order mechanism The point is

need important we the only and has the is what and not to exposure of what implications informs response that which COGS course, relationship, be do for what dose out picking size efficacious other figure the also but that will such dose, Now is informs lowest to pill things.

the I that really you let is that’s that Xb doing. is that fully what on want So enrolled. we’re now the one to Phase do know update

a So, to we’re future. going in all to those of the be good spot near understand information of in pieces

Jeff Leiden

is take question I’ll commercial strategy really second a think. The maybe I question, part. the And

to as outpatient commercial it market. think of example As which acute that commercialization I a neuropathy, would parts a in pursue a an said and market both itself as is hospitals, market. that data address post-surgical, develop typically in assuming ourselves. office that and single that’s commercialize be market pain new a a don’t LNG and you’d that dentist is again we of fiber have like that supports form in a of as or ourselves, we like specialists market can of centers with it one an plan development market appropriate we to It’s We dollar feel procedural opportunity, of specialty specialty NaV set much that essentially a market very in small before, market large as could similarly mostly of can and multiple surgery pain markets, because and have data, offices. multi-billion our it’s oral ourselves the That’s think we we typically it addressed pain, be pain, post a

et that’s commercial our very you You pain build it’s market might market. know different not and where see a back to us perspective, cetera, a go And chronic would very, presence pain we from with predominantly that one inflammatory the that’s low care OA, a and develop that’s want then from the primary partner.

lot had a We about the of a experience won our you partners first, question community pain who due as amount class we fair imagine markets. analgesics these in Does of of have to, have interest commercial might strategy? that your given answer from

Paul Matteis

very much. you Yes. It Thank does.

Jeff Leiden

Sure.

Operator

you. Thank

question next Terence from Goldman comes Our of Sachs. Flynn

open. now is line Your

Terence Flynn

how if maybe recognizing now all data question. the now, KALYDECO years us more relative taking ORKAMBI. a I even you compares have Thank for the you at persistence to lot wondering, KALYDECO can improved to think plus? Thanks you. X help just on SYMDEKO And Hi. this was I on impact of quantify

Stuart Arbuckle

than I’ve it’s many certainly the things somewhere SYMDEKO launch, ORKAMBI fluctuate Yes, obviously years is is X more these persistence we and we quite the it’s over saw the personally certainly, on KALYDECO, ORKAMBI. seen in because Terence, years. months not saw this, we KALYDECO highest lot like KALYDECO, which any were ORKAMBI higher is were doing into anticipated without into with exact mentioned the kind the but getting SYMDEKO time XX level the as ORKAMBI, I’ve been is so a above that the between with of for ever seen numbers specifics longer, and But at exact we’ve and SYMDEKO

Terence Flynn

for you on more triple of launch? us in if seen Thanks. details warehousing give U.S. guys any And potential and can ahead any patient then the Okay. mix combo you SYMDEKO the the

Jeff Leiden

taken broader the up all ORKAMBI, ORKAMBI and seen the rapidly ORKAMBI, also seen patient who’ve switch of we’ve to It’s a either SYMDEKO been patients in terms you patients then in that from never actually of of on expect. KALYDECO. from were uptake So, across discontinued fair who we’ve groups number been uptake, for would those naive patients,

So, we’ve of broadly eligible really patient populations. the across seen uptake all

Terence Flynn

lot. a Thanks Okay.

Operator

Thank you.

next from Citi. comes Robyn of Karnauskas question Our

Your now line is open.

Robyn Karnauskas

Hi. you taking guys Thank for questions. my

for are So to that the SYMDEKO, all, actually, for or you pipeline SYMDEKO given a that JPMorgan, learning KALYDECO asset, develop that seems like the look market, JPMorgan on first it you what discussed of switching like patients the you platform for about switching is what – at so patients part are ORKAMBI you there or from Thanks. longer these with a you a make cheaper, about thinking are are might in follow-up if triple have get just forward, a looking and [ph] switching externally a are do [indiscernible] looking question and mind more to something you just seeing remember point? patients, you and change going drug. the things is to SYMDEKO how to captive changed in differently? science Are you your it’ll the Second question. seeing might think It help be

Stuart Arbuckle

can then and Robyn, question here. I’ll question. the the pipeline Jeff Stuart your to Hey, take next first it’s innovation take

seeing we’re mean, of to patients to So, who those generally terms ORKAMBI or experience. SYMDEKO we’re KALYDECO what patients in SYMDEKO, have seeing with I switch from a great

original go of patients of many or back either seeing kind aren’t We medication. their those discontinue to

saw of I’ll our in way that that benefit innovation, direct back of people know benefit attractive comparative I to that top that SYMDEKO as terms profile don’t As who in the substantial capital, from so population, clinical on are SYMDEKO then think have people are I it’s and expectations. finding with going we you either majority what’s and much in Obviously, risk RF transition. Jeff. the driving transition overall data, making data or ORKAMBI, the of the is think of hand with and of add the that’s think is [indiscernible] I we in And the but line external

Jeff Leiden

But more just to going the other relevant of lots about just who Hey on to more is the do drugs. obviously and better strategy and think well this data medicines on and on Stuart to to triples, the other Robyn, the provide question one Maybe I better not label is your it’s and point because patients and as depend continue to are for the Jeff. the including things. here patients, switching,

And XXX, even that majority the switch do the significantly to data better we we’re these over assuming will which with the patients that long-term over of than valuation, SYMDEKO triple. so in vast you as is performance seeing know now expect

sort I all pipeline our you a miscommunicated of the that’s BD for obviously I sideline if that the and future. the JPMorgan to the strategy same apologize respect did With along. just is to So strategy,

those buckets and meaning as they’re tools them. done to out potentially we’ve do deals early assets and So, CF interested certainly, technology we’ve we’ll stage been the point transformative a you of three pipeline strategy continue diseases. always lot But of drugs that meet and serious in our for

find. not stage diseases in we licensing and As assets. you look in should know, they’re expect continue us on do to certainly register that then those for you But to we early them them to if easy deals see acquisition or to find

Robyn Karnauskas

you. Thank

Operator

you. Thank

with question Markets. Abrahams next from comes RBC Capital Our Brian

open. Your is line

Brian Abraham

background curious shorter other contribution you do maybe course the what front? and I on of bridge to more you’ve seen Hi. you’ll the over X to revenues on the whether KALYDECO any or been of to dosed Phase wondering Thanks type my to us was feedback to X-year-olds questions could triple, quarter. translational term give from ORKAMBI you hand, if to need preliminary term discussions that to X you’ve the that SYMDEKO. educational uptake work get And bit with switching background like for had functional and pay on little the the separately or the on very dynamic have long maybe congrats of data color adult am that a as in years I or much benefits relative in of then taking Thanks. treatments more outcomes

Stuart Arbuckle

the that’s each we’ve to patient how gone populations old to with uptake and to two as on patient five-year you So, Brian the to lead in and that populations ORKAMBI of strong strong take to being specific for triple many same it helps triple I now. and of hard of why really quarter groups gone ORKAMBI we because to so the it’s patients you same might exactly very will each a Reshma. guidance And data are ORKAMBI to from compensate in in the all kind have for ORKAMBI, a be CF that’s people we’ve that going know patient approved getting revenues of on switching imagine over who medicine dynamics as then about because first SYMDEKO fact one population medicines these will product that so to seen to and triple those now and and every the of are terms why CF question are with a kind I hand patient if you like there of and be number these

Reshma Kewalramani

Sure.

as four combination, studies study, have endpoint both in the FMS to and at primary regard know or ppFEVX. X with XXX triple So and XXX interim analysis the ppFEVX FX you in the weeks the programs the weeks it’s for

KALYDECO you decided through in because, ppFEVX ppFEVX of a that forward in very with data at the to go it’s interesting reason weeks our we treasure a trove is imagine, looking Now four time we over as is have can ORKAMBI, what very XX. you at example and four SYMDEKO, is for that what you is relationship week consistent with XX later there see you see modulators see week what a at or and and CFTR on with week experience,

actually amongst is physicians that well understood think as I well so patients. relationship And reasonably as

some to course XXX, ppFEVX, of rate the XXX to so we XX-week of decline we thing SMS and note pulmonary interesting such. to going can analysis going both have have other studies start also and The BMI, we’re to of is for the And in exacerbations, do are CFTR.

bit of the community, very I’ve quite not actually been the been that’s data coming physicians with a the studies well as how but have well. just understood patients and it’s impressed the by So,

Brian Abraham

much. Thanks very

Operator

Thank you.

from Matthew comes question Harrison Morgan Stanley. Our of next

now is Your line open.

Matthew Harrison

was talk a the I margin very I if could you Thanks little bit Great. guess operating much. wondering progression. Paul about

about XXXX we relative You’ve X growth in beyond How that you’re obviously points and the or this thanks up much? X very revenue think to year. should talking moved about

Jeff Leiden

XXXX. It’s will guidance more the a highlight. actually. little for Maybe Jeff given question I strategic I of since Yes, take we’ve a you

expect see line in but guidance operating of to of and as growing income. obviously launches top a as mentioned consistent So, you’ve over current faster even years increases look the and we we forward reflecting XXXX. we that which the results in only in we going as operating expansion after triples, pretty next forward look last year, seen accelerates couple than operating margin XX% revenue get as that same to and there’s because forward Paul operating faster grows our And operating here trends a in growth, as of pattern revenue XX% expenses. triple think I growth considerably exactly the to expenses, Obviously, margin revenues

We approach. have maintained a pretty disciplined

income long flows, So, on expansion you should forward. without sort you does that cash detail operating in this term about think Paul how enough mentioned, continued term, which I results increases, if we think operating of that long think you you margin guidance, about think and give that giving also the going

Matthew Harrison

address very licensing compulsory then one the see helpful. specifically just call. can the and you if around there? pop. some countries I what other the if that’s there ask I forward about some reimbursement of on the people likelihood have And news in wondering path you was mean of as I anything with happening ex-U.S. asked just Yes, active could

Stuart Arbuckle

most desire or very debate what here. Matthew. think issue. really agree sciences we’re it’s say not that they including There option I finding inconsistent Stuart patients recognized the license with concerned ORKAMBI entirely a as our It’s on in multiple as and certainly life I their probably that uses yesterday. issue to that we’re we with would a soon a medicines is compulsory compulsory vibrant focused soon Yes, can UK it’s in future recently was is it on provides for pipeline that government for complicated fair itself ecosystem. call a the this discussed to reasons not UK been in of that. We debate the I mean focused viable to licensing on crown approved. the their as It’s particularly as as say, perspective solution they’re a foster access is about short term the that frankly in

Matthew Harrison

you much. very Great. Thank

Operator

you. Thank

JPMorgan. Cory Our Kasimov comes question next from from

open. line now Your is

Cory Kasimov

and I Thanks wanted then this you about my if volunteers. guys. question. to and the taking is wondering what data ask I initial afternoon, cart from talk expect for the learn good potential to realize healthy of And in can about putting you Hi, clinical might AAT cadence a what driven curious like assuming before the this little work goes just trying stage you horse to might understand program potentially may biomarker well I’m this expect clinical would it encompass? look here, but outcome for a to indication early pathway Thanks. registrational the broadly be what

Reshma Kewalramani

This Sure. is Reshma.

future through our me similar to taking so that to that’s XX, extending it’s to that see that early were the going that With you we as elevation were whether the studies. of in registration us regard standard patients. program healthy get to we path ascending molecule soon I be I go are and can and a readout this in those to volunteer four an the so market on we levels. infusion efficient tell program multiple that available that be of it’s Let able which approved know trial, healthy as have news the a existing program quantify on are to volunteers approach good on to into what in question. expect about fairly in me that that, efficient were through protein. ready by it’s XX is approved and people that to this our of don’t here activity. soon is AAT of Those in we’re done powered at for you is therapies, parts as This [indiscernible] therapy we get the based the fairly to know going be is and if here, small them those which reasonably AAT the The intended regard the going With but dose in be near molecules so a effect, going hand have cadence as take we reasonably let those to level

start that have one point that’s data So to you with.

We Phase go points have that’s regulators to to look important the going to at. be point X with an But our through are I conversations to see what data registration do enabling end think here.

Cory Kasimov

That’s helpful. you. Thank

Operator

next our Hartaj Singh And Oppenheimer Company. you. Thank and question from of

now is open. line Your

Hartaj Singh

Great. Thank my you question. for

had that in you the XX-week triple safety us had gone up? and are you’d on a you data data just you. the readouts coming for Thank through. Just as efficacy give the the XXX how report question also I as two update that also that projects data X indicated to you XX-week Can know both November an

Reshma Kewalramani

If our to how data XXX be Sure. terms in coming late will hear efficacy. can both go quarter I for the good and XXX gives of year, template safety last you this which I back share our from think very for of release to that expect you a

trial. about to you’re you side, now weeks you tell the us ppFEVX one, with from And on So, going with both on four FMX give to we’re we’re do go overall hear very, thought line going efficacy with you FX top going XXX. study just But very tolerability. to this beyond not regard about safety, like the very be integrity and discontinuations to that, the at the far and thoughtful regard maintaining to we’re going to the to

Operator

Thank you.

Michael Partridge

we now Operator, X hour are at mark. the

will one have time question. more we So, for

Operator

last Liisa JMP Bayko of And from question our comes Securities.

open. Your line is now

Liisa Bayko

around of Thank Hi. Well about be to you patent triple, triple lot the but that’d sort been the life did asked want ask you. and a could helpful. question’s the my if of I describe

Stuart Arbuckle

our that, medicine granted as of the X,XXX which around of the Liisa, the And the for the patent into so in determined case you folks both expiry ingredients and much longest actually so single the pending the Europe. life life applications, is be VX-XXX U.S. in asking pattern these thank Since you and case by to that. single if ingredients. the pill in plan have would VX-XXX co-formulate which you a patent is get know, to patent

Liisa Bayko

extension would or be any a And that query of that it. that’s

Stuart Arbuckle

By XXXX think early be to way about going too is there, to I’m it.

any obviously, speculate extensions pleased we and we’re point. that ways at We’re pattern on with this can’t really

Liisa Bayko

Alright. Thanks a Congratulations quarter. on much. good very

Michael Partridge

Thank officer is in for follow-up do team joining and The any Lisa. the tonight. questions. you to Thanks, further they’re us Relations happy Investor

Operator

conference. and in Thank for you gentleman. you, ladies conclude This Thank program. today’s participating today’s

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

Vertex Pharmaceuticals (VRTX) 6 Feb 192018 Q4 Earnings call transcript