VRTX - Earnings call transcript Q4 2017 | Vertex Pharmaceuticals Incorporated

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Michael Partridge

Good evening. This is Michael Partridge, Vice President of Investor Relations for Vertex. We're pleased to be able to talk with you tonight about our Fourth Quarter and Full Year Financial Results for 2017 and about our continued progress with the long-term leadership in the treatment of cystic fibrosis. Dr. Jeff Leiden, Chairman and CEO; Dr. Officer; remarks Smith, this Medical will Operating Chief prepared provide evening. Chodakewitz, and Chief Ian Officer, Jeff Stuart Arbuckle, Chief Q&A. Commercial Officer will join us for recommend today's the to access that as We supplement a webcast the release. press slide from information you These Relations on slides our Investor Page are download the for available on website. be This is will and website. our a recorded call on conference replay being available I call. this will statements forward-looking on remind you make that we will with release the uncertainties are and discussed risks in and subject our today's in statements filings These Commission. and press Securities Exchange to detail the These without financial management's cystic on financial including fibrosis, management limitation statements future ongoing those based other development commercialization of current regarding assumptions. and fibrosis Vertex's combination Vertex's are the programs, and of any performance, cystic Actual outcomes events differ and could materially. I will now turn the Dr. to call over Leiden. Jeff

Jeff Leiden

to who everyone. CF that in everyone KALYDECO. Today was day anniversary significant advance journey CF helped In expand X we've developed patients people the evening progress beyond. years, worldwide effective like one a the in for studies today's combination in to the function especially FXXXdel a as mutation begin decease. to patience, well the the half to for the both containing the triple have regarding new CF of of cause regimens us Phase the combination everyone have medicines mutation different of both medicine. of year. scientific number of special for a this ultimate of and our unwavering were of treated employees closer to treat develop in with believe made late-stage advance to XXXX, regimens. regimen that would data at have eligible Phase than regimens, number medicine their are one only as than When demonstrate Phase part track continue of these a we the for and VX-XXX for people in and we CF, profiles significant as approval which towards and first and thank to focused look FDA Good achieving for it to XXXX, and with productive development the correctors like two bring for XX community, regimens is once-daily our remarkable one underlying all CF next-generation as and we that commitment evaluate possible. with their and on medicines the past today the XXX we by goal VX-XXX and remain triple VX-XXX KALYDECO at on X, eligible when highly VX-XXX. begin patients Vertex Today, best moved X in achieving having bringing our We design of Michael. the Therefore remain marks decided will with into I'd triple have we sixth triple triple two to as approved acknowledging to program. forward programs more that four a grown highly with we from decease. to people first I to selected and CF has this and at combination important in for year has XX,XXX regimen. plan XXXX, caregivers, our the taken goal. eligible worldwide the of We potential Vertex Thanks, milestone has development. more few families, first into to medicine especially regimens, both today X,XXX data FDA discussions In programs collected announcement to We're a combination for CF. step finalizing first Phase X the for people compelling to this for minimal either clinical benefits support, different the patients announced remarkable consistent to forward The as the quickly regimen triple or Based upon combination combination totality regimen We their us of

As positioned I progress over we company parts year, across the look success. and of business the had all further back past made tremendous our for have

which of is increase patients treated revenue. to approved our continue eligible the for number driving We and with significant medicine

for development revenue our growth CF clinical growth. the cause ivacaftor those this beginning to we expect for significant contributor combination FDA significant and treatment Phase move in preparing The revenue to continue with two hope also this and into which awaiting two are earnings CRISPR beyond to regimen. and new disease, to devastating for still Thalassemia which medicine, further as their drive this selection day tezacaftor are begin we provided will awaiting new Therapeutics. in of will with option of of year. be of part Phase reported the disease cell growth data approval a partner We We combination triple X turn Beta positive important the correctors next-generation And CF of CTXXXX development Sickle X a

potential Dr. on in updating more detail. We I over look the development you progress move to coming today's continued call turn and expect into announcement diseases our also from Jeff to more year. to programs Chodakewitz other in one over year will review the our medicines clinical research this now to forward or internal

Jeff Chodakewitz

reported evening. X the VX-XXX four in co-formulated advance ivacaftor Phase and of very efficacy own our initial patients. our safety correctors extraordinary and on provide plans on share significant to review development. into than PK regimen to these studies out today combination CF benefit are ability Thanks, an and but patients different both minimal pleased vitro clinical like Jeff, were and CF others. of to next-generation Phase and initial provide profile, All drug more XXX X efficacy The including ongoing of and evidence into from their results may Phase regimens the good Collectively, the triple combination tezacaftor of only from X I'm VX-XXX in X to Phase perspective. labs our triple two combinations potential, our data advanced profiles, triple with also the compelling the interaction that property, their not based drug be studies development

All generated X criteria to-date selection data use. of validated the Phase the rigorous has we

absolute tolerability CFQR function percent study. each a We well mutation are chloride, change top-line as data minimal and efficacy patients as and as reporting safety mean predicted group with by FEVX, measured from and today sweat the information for within data

with There favorable. adverse following of triple or patient patient This combination with without the or adverse three tezacaftor rash the weeks. decreases XX.X, due VX-XXX considered were XX. which who to of sweat the in The lung VX-XXX week data moderate. received are combination and the treatment. were these dosing Across and VX-XXX sustained regimen. dose percentage of of due in significant safety majority sweat triple to patients the our the was was treatment were by study and improvements regimens this overall no one events. study chloride and Slide of period These XX.X evaluated XX.X, placebo for four-week First function interruption One on to resolved millimol dosing XX.X, ivacaftor a through we XX.X, evident for any the of groups. the saw Slide groups combination received Phase in across combination the The ivacaftor. the X events mild combination in completed In four data for shown generally patients to-date, were tezacaftor we decreases discontinuations combination three well doses shown combination profile triple restarted significant XX largest tolerated interrupted triple the dosing rash. per triple These the second period. With chloride, points and that of for and liter of further observed on combination, XX. observed dose any are XX.X triple data triple and

study and groups discontinuations received considered the The events moderate. due was profile one of Across the in well points Slide XX to none tezacaftor score, were combination triple placebo significant There milligram the are three these mild placebo study, XX. and XX.X, and patients now weeks. of safety and the ivacaftor. adverse tolerated occurred combination in ivacaftor for the tezacaftor treatment in events will We doses triple adverse I in those respiratory with group. data generally dose group. discontinuations VX-XXX reported was on VX-XXX with the the as majority combination X symptoms VX-XXX treatment of CFQR combination regimen. to four shown on for or XX.X, evaluated observed Phase also data This of were patient overall VX-XXX turn in for from the domain XXX respiratory the or the favorable. improvements and XX.X The reported regimen in triple two

One bilirubin to during of concomitant which of of levels treatment. patients' was transaminase bilirubin the was observed returned the The day treatment on follow-up the with safety dosing. final after baseline discontinuation increased to discontinuations period due elevation

the of and millimol data saw function XX.X and In the four-week on triple discontinuation period XX.X of observed and shown the XX. XX.X, are combination those dose triple lung second X.X, treatment treatment percentage we for resolved. Slide Slide rash per due chloride, through VX-XXX significant who was the XX. the sustained dosing are by The groups, of XX.X, in rash shown these to sweat second period, data of evident that discontinuation decreases following these were improvements points liter and received With combination the we week significant XX.X, on regimen

XX.X XX. score, also reported studies. also the XX.X, CFQR post on spirometry combination respiratory Slide dose evaluations the We the reported data for that I of shown are these both of symptoms XX.X, these triple regimens points improvements in and regimens patients domain observed would significant combination on in note for in as respiratory conducted triple we those

correctors daily from with received Bronco Phase triple regimens cause the evidence And ongoing X studies development. currently are are two the portfolio in regimen VX-XXX VX-XXX the half and function that the and mutation. and of expected part of is are the one of and to-date the Phase of twice parts our potentiator that mutation potential data X Phase regimens in of triple across so once next-generation treating study with no patients additional minimal from combination one patients the remaining advancement rapid place in X studies mutations. show clear, Data VX-XXX the of XXXX. of with triple into combination the of CF the and construction. daily each each regimens enrolled contains are in with support first benefits VX-XXX evaluating An These a FXXXdel fully potential studies VX-XXX of FXXXdel combination of daily ivacaftor The once

to generate triple opportunity quickly both from Our the to picked possible. advancing one dosed including daily maybe bring VX-XXX two to regimens, strategy gives of that different patients data once regimen into Phase VX-XXX X as best and us and as the combination

for Our combination triple VX-XXX available combination shared the Phase regarding triple with have our been data VX-XXX already for discussions FDA we X the have with productive and program FDA the regimen. and the regimens

now of one mutation, XXXX, these conduct for upon are X each the We Phase FXXXdel those we of remain to minimal finalizing in regimen, who first triple people function of one study have regimen and mutation, plan the a focused on program two completion the mutation. separate the two and a FXXXdel with to of programs We combination X studies half study Phase on in discussions. with each initiate design track CF and in first

of and of plan studies Phase first regimen the we with half VX-XXX the regimen triple triple in of the to the X to studies daily X VX-XXX and VX-XXX this once potential We with of plan toxicology combination evaluate for the in XXXX, the initiation triple combination also for VX-XXX X middle studies combination for completion initiate pending in the year. the VX-XXX. long-term Following Phase a data the non-clinical a regimen, daily the once potentiater regimen, the tezacaftor as Phase

you These begin second designs our to with mutation. have who to and planned also studies evaluate FDA triple in the combination discussions function plan are FXXXdel regarding the studies of complete, in of details updating a the just discussed evaluating are the we more study. I we to each each addition specific XXXX. regimen residual triple one regimen combination of or half to look mutation second gating In Once patients in these forward

helping I us everyone thank who for treatment these CF. the took to call their in part Before to I'd close, the to Ian. now turn commitment like studies advance I'll

Ian Smith

fourth financial quarter and XXXX to Jeff, guidance was compared non-GAAP XXXX. quarter to Thanks, XXXX in for XXXX represented of fourth for increase evening R&D revenues tonight year an financials, good first. Vertex in the our to and SG&A Total everyone. we combined CF XXXX fourth outstanding full-year our recorded and I'm $XXX XX% review million trends. and million product Revenues pleased the $XXX quarter

quarter therapy the in Our number XX,XXX approved Today, product as revenues have medicine. initiated for of grew patients treated we throughout with medicine. the we each increased we over eligible our patients XXXX, XX,XXX our of estimate

grow driven continue We in children fourth X expect eligibility particularly by we XXXX. patients medicine million, and of the compared significant rapid $XXX following ORKAMBI $XXX we throughout of product to The million, increase XXXX the expansion of reported to U.S. revenues quarter of with continued XX uptake compared label patients in was in growth in these treat number XXXX a the the driven KALYDECO revenues quarter by the by growth in This to to XX% the in function fourth to the the U.S. ages XXXX Fourth was of the fourth medicine XXXX. XX% residual increase XXXX. of mutation globally, uptake were patients mid the XXXX. XXXX quarter quarter

Our $XXX compared triple XXXX. Non-GAAP CF for were profit non-GAAP quarter total support in expenses combination million of acceleration the compared to quarter strong CF XXXX quarter was increase of our primarily to net treatment and of of increase product and portfolio the continued XXXX. This net the due combined million quarter was $XXX $XXX by fourth XXXX in profit $XX in of to the profit our patients million advancement investments with was regimens largely the The medicines SG&A fourth net revenue. and the fourth non-GAAP with R&D driven for non-GAAP million the growth globally. to fourth

the operating the financial patients XX% Our we in as increase compared XXXX medicines to for year XXXX. And of expect we full-year operating treat our to XX% has number performance expand in in to continue of resulted full margins our that a we future. continue non-GAAP margins with of the to

we cash approximately billion balance revolving paid under We cash credit beginning year. position ended quarter as XXXX of XXXX $X.X our strengthened marketable also sheet to that was compared This and securities year, we the equivalents, $XXX was $X.X down cash, first facility. at our increased the the during billion the million of in with outstanding after

and combined guidance. expenses. We SG&A financial to Now guidance provided turning today for non-GAAP R&D

provide when for for provide XX, the with As we have approval guidance we We XXXX, product in by revenue guidance The FDA XXXX combination. CF U.S. U.S received will guidance for stated and action countries previously, older the expect be who will revenue the include more outside do not FDA we is growth significant expect tezacaftor/ivacaftor revenue product tezacaftor/ivacaftor revenues to for XX date February and launch total we XXXX. and it patients in CF treat individual in products. of patients ORKAMBI driven eligible the When

impacted by channel each revenues quarter that experienced in think and will the XXXX. that of the inventory be we quarter first in higher first occurred anticipate fourth year net revenue XXXX, adjustments we quarter by gross about we As build the to

combination combined regimen, Now success expenses. to chain to launch billion. to In the of and expenses The the combination operating for the investment key triple of triple support $X.XX execution R&D SG&A expect of investment drivers $X.X for the supply commercial pivotal XXXX, planned regimens, of two non-GAAP studies potential are investment we tezacaftor/ivacaftor. incremental and billion

in increase Vertex our a significantly to to revenue XXXX. the continued that, open year we we operating business to continue and the revenue has line treat XXXX earnings our will in we margins anticipate patients do questions. of sustainable was as we now deliver medicine. number expenses, transformative and expect execution and of operating will As significantly positioned our growth, with I parts for exceed all With our the increase us growth earnings expand across for the

Operator

Instructions]. [Operator

comes of from Our from Leerink. first question Porges line the Geoffrey

Your question please.

Geoffrey Porges

comment little the the sweat doing Perhaps you you particularly which results XXX I that appears be And on XXX is was a and spectacular by then, chloride why planning with Thanks. was could why potent? little expected number XXX guys chloride since you the and to you on much Thank what more and XXX could it. be a Appreciate XX% suggesting active though little plan and want one XX% also difference comment sweat that bit bit a might a as combination? on between looks remarkable. a and X to data. you bit which no is congratulations it active, wouldn't bit a response Phase be cleaner, Jeff struck XXX more about to quite very little XX% response was

Jeff Leiden

this Thanks for Leiden. Geoff, Jeff is both Yes, questions.

need toxicity And to them look Let that next-gen in same as a way two is one feel is really is one then XXXX X. compound. with XXX strategic the mitigate Iva to our quickly the is portfolio the Phase forward because take that and But but. these make you our the -- take to due one I reason obviously first say the we supported one both a Phase of we of rare question the that remind into that here by I we with taking still into all X VI question. best corrector to we're that's pointed target regimens is way both it's because And showed that's we're a two answer with certainly actually data them as if why sure get of Iva, and forward of reason risk. risk to because confirm although second and and regimens modifying with that me very potentially taking of forward, regimens of you it's two and one we by were in approach some take with chemical potentially forward our risk, the to quite good, forward as off And And that possible. it’s are out one VI of right. one these your our just mitigating plan VI, Iva, four risk enough really obviously different results regimen similar taking VX-XXX significant that's fact results to to patients

We have or XXX patients. only seen it tens than in less

my safety eggs to back. that very find tolerability And both all Iva is with the issue so I or would and basket set would hate in that out rare some put there programs

So mitigate that about as better that that end the commercialize. the feel take and one at the and risk the day, let's decide we that's best the way is VX-XXX and of to assume we're going we regimen forward Iva

We have of bioequivalent once over data. regimen some VX-XXXX believe day simply with quickly bridge to the to a we opportunity type into a

And we eventually I hedge of use just a think once both a so at of winner be to and a regimen day day. or those have we're the approach trying the XXX, to the that out portfolio strategy turns it's whether it risks to to end for getting XXX

Geoffrey Porges

the And Great, between in FEVX? the sweat chloride and difference thanks the anything Jeff.

Jeff Leiden

so Yes, thanks for pointing sweat chloride.

know, nor and you sweat As FEVX sometimes chloride. of always a talk about we sort

are One millimol this good. that sweat so this suggests north of of And really which differences good you respect XX is regimens, is of honestly and which the are it's XX what very to XXX both allele truly sweat we patients' all chloride And think in underlying would any is is the that the to we're is to we reassuring FEVX at general XX the next-gen just really about they're put not XX only it in of if it's very one the the The the sweat drops they drops these I consistency an really with of effectively of is the response, really getting most difficult augmentation cause to news I treat data. we number picking impressed are not me data I things millimol that's think and totality profiles. don't remind not on Remember disease just XX between patients it's ahead one look with this value, seeing. chloride the and of it's profound. all seeing very, chloride, about there just remarkable.

the will we take So and these profile, based take totality both tolerability. bit sirloin are the but steak a and of to in mignon here it filet efficacy the picking end the safety, between winner upon

Operator

Thank you.

comes Yee Jefferies. question next Michael from from the of Our line

Your question please.

Michael Yee

part for question. Thanks the all announcement the of on data and congrats two question today, it's

was also about comment the is First what's maybe sort much you it to the the these safety rather duration of precedent part dose the the and response response win these a profile Bilirubin for there's case of Phase tolerability maybe much. safety as maybe duration on types it things on efficacy design not just or us second comment And filing relates how of is Phase of X really dose ask programs, X would then types the seems meaningful? two of that file maybe clinically remind so just than the but on of like of and to just then of things? Thanks and need the

Jeff Leiden

there. answer talk come I'll and the tolerability safety part response, question to will Mike. on the and can and of dose I Bilirubin, maybe about first talk timing the back Jeff Thanks

regimens, are back look they of XX all one sweat is again XX, multiple we you're at one look First I that XX, clear looking to you're chloride, case at about talking pretty response, but well is looking things considering we doses, respect a response to look mean at whether of at study. that whether hey patient to across come every to dose FEVX. did XX, will me dose totality four the remarkably at very and with four we impressive that's regimens we those

XXX, the sweat jump you again. the the are milligram similar milligrams And FEVX So outlier way. you to to which out and XX when XXX of pretty The XXX I which dose behaving is see then the you're in pointing go one quite up well that response compounds dose. response the chloride think the look

interested to dose the interested and in just of are response. course understand in not exposure the it response, trying really we we the is So what right, end that were

-- looked quite you we so And the at are XXX look is see in exposures went in what and one XX the of and but overlapping and the that XXX, the back studies clear is chloride all exposures interesting at particularly if in up that XXX milligram chloride, look FEVX and doses if we you and in clear and sweat a intervals. move in a jump a sweat those quite overlapping, FEVX and confidence you you you see to see jump at differentiation When exposure sweat you chloride.

long tolerability actually to turns out respect looking talk to Jeff so as in And you I response. be relevant to about well will which XXX is a second. let too And behaved exposure with Bilirubin as are thing versus

One best about I feel which which window, easier index doses, look across what that doing. the us these or excellent the we compounds to a here things dose for at one these that and in of to with the makes it There very and is each problem the good pick or for is all going really seeing no us of here any is is think will make compound evidence therapeutic tolerability reassuring process of pattern we of are doses tolerability. the or it's exposure between doses go

on the tolerability? Now maybe Jeff

Jeff Chodakewitz

really that point the key this as other elevations about dose on telling. an of patient the is of range specific that you liver no think here do think actually that across the further There question consistency and Mike finding. with elevation. terms your really very, any in isolated resolved very Bilirubin, or and on Sure. is evidence any was I I rapidly findings without were continued heard the one restarted interruption actually transaminate

pattern a that So that's concerning. clinically really isn't

an it don't as issue. So we see

Jeff Leiden

And don’t sessions then of I your Regulator having you data the our of day. data we've you European duration been are decision of on day our but or we’re know, we to FDA end at can year question, of a that's the as most finally, point just these these with studies, Safety speculate really and historical FDA. where the in of between months six some safety an

point this I things a is to of children think medicine, for take their important asking medicines lives. be are rest these would the the is

we of do efficacy regulators comfortable a And the decision the dataset we're end so make the efficacy, that day that will are and of will course and comfortable have making. to with. complete ratio be safety with want that at And of it be we that safety the sure

Operator

you. Thank

Our the from Barclays. next Meacham question of from Geoff comes line

Your question please.

Geoff Meacham

Hi guys, my also for the thanks question. congrats. I offer wanted to

Just ask maximize also different to the get as little I is ultimately two it's a as risk possible. benefit know positive way, bit obviously a quickly but questions goal to FEVX, to here

development? competitively So how do or of you road trying those could down guys available think Thanks. what you when just FEVX be. end balance think the an have two in perhaps upper and do the to you I'm new therapy other combination

Jeff Leiden

a they Let are related, bit Geoff. me answer

trying lung ORKAMBI by it of what that can those all really exacerbation, antibiotics would to indicator our the long-term pulmonary is which disease patient slope be lung going a FEVX likely acute by of to FEVX as a who curve, amount IV the not and you actually and will in move we of you that's reverse the chronic do both response clearly there disease course that to very different decline very trying different function of younger and with or XX create that that. the a that important and are true an XX-year-old decreases of day and the but to a FEVX an Remember our young ORKAMBI. disease hospitalization, able the all be favorably you includes is in be and what seeing are of we age the both It's starts are story there, back the includes be XXX particularly by KALYDECO starting are prevent And KALYDECO with in to acute efficacy end clear one-year-old XX success at just CF. things at modify combination that or the of is impacted because you is and than structural we on goal. benefit, very FEVX bigger FEVX this something And they So of an have goal in been all while to patients. who course results have you not it's may only first acute with measure ultimate as and get efficacy of two-year-old the ceiling really at to disease of the or fair a

But do long-term little will to be want than bit one is measurement, on clear you the we to FEVX acute be, as do. yet the all what disease that I we measured told the ceiling depends just the the I that is a to of goal things things the don't really explored fully acute much it have So it's one want ceiling, as long-term, treating course think who more by about. that I you modifying of FEVX, really are

modulating I want are a to that a be very One obviously at of useful with look going of last those anyone reasons longer-term for time can the is CFTR that and think the measure. to endpoints therapy do very us important triple interesting to trial and and we the placebo-controlled maybe this very,

Geoff Meacham

COPD but gone I guys have not a you on getting yet? path have data mean is as down looking into these as that get once the some quite interest CF something the proof-of-concept with Xs just IVF pulmonary like the Jeff or things technology follow-up Phase and not cellular And you assays, pulmonary what at or assays that and focus level guys or great or clinic what's to you

Jeff Leiden

better on you're a work are still Yes. you're correctors. to I thought actually me going me thought different going I to ask ask going getting to you question.

you let an right normal experiment means know but is So about know carrier we and And the and you allele, that normal of mutation, transport of we you have one because answer nature yourselves you of answer the yes. if from the XX% have me around get And don't in there. is question disease. a sort chloride reason are of that which somewhere a

is we now, close that into everybody believe turn carriers' a carriers early regimen ultimate with with of and we And getting we drive these some of and to so which course can regimens are treat data patients if the we goal. pretty can actually triple

these are we on we are correctors that and couple to in we’re because we who the medicines and focused to will to going these and better journey better journey when We now. finishing to up So we want that still efficacy. CF. other to right we alleles the continue totally pretty XXX CF for. be consider With and the IVF can we on on work finish get two But seeing XXX might of that's right here of going focused of has we COPD, next-gen work one over next years things are now everybody respect use that to course plenty triples or

Operator

Thank you.

Our next from Nadeau Phil of Company. Cowen from question comes and the line

please. question Your

Phil Nadeau

good would I Maybe then add afternoon. first congratulations Good data quarter, my on in one scientific a one. the financial

talk those what the like events. characteristic or a that's cleared were you lungs that clear adverse with and kind been sputum scientific but side characterization? rise side side the where Can tolerated were bit that pulmonary of have pulmonary gives where little effects, effects and mentioned you have in note the different it’s the coughing compounds they you are to well of KALYDECO the On are those type about some seen the did you

Jeff Chodakewitz

exactly in actually and cough the underlying adverse effects like that Chodakewitz. thanks, think fact that pharmacology of things you events think No of as it potential them So it's to almost right we're trying Jeff sputum reported they was marker as I really clear but have we a that get. it

would we as look studies the beginning from any to -- we in be say really kind all mentioned of is We've right very and in there. post-dosing prepared that I one nothing that So specifically in for to the data there look comment other these the just that's decreases remarks FEVX. way. got

the is it’s pattern what exactly describing. you're So

Phil Nadeau

could you second the have confounding one or side patients nothing that that to rise the not anything on is there Was Then Okay. there those in effects that effects trigger-related? really giving mentioned was concerning Bilirubin those about side rash.

Jeff Leiden

No.

thing, with about. of adverse of very people a You events small which any we're is to have remember, good these numbers talking

we really generally they moderate, the were to you is think told say tell. early confounders I thing it’s the too again important profile I think there safety as they that and mild But were favorable. quickly, resolve to other to any very

Phil Nadeau

on reasonable; finances. run guidance last growth what you same about approved know based be us year-over-year X% QX's kind is are some maybe I question not seem discuss the Great, you versus you XXXX? to rate. of you XXXX how hopefully general the you you the for on will Could for that Is Ian consensus in to can drivers of give going until feel about franchise for thanks. estimates And give month. the assume my growth one tez/iva math, next sense current about

Ian Smith

Sure. consensus call When guidance that won't look give we Thanks any thought the at question coming we up. for the come even you may to though question, Phil.

firstly the how on we which growth so we we does say growth Obviously consistent about that I the XXXX. approval XXXX would see will for number revenue of the the be of think factor are XXXX that the And waiting consensus is with in line. that reflect major tez/iva

And so like are get we’re in on would tez/iva. this once guidance that is, at be about is remind clarity CF will I our it consistent greater it it's the where call, total guidance. but revenue XXXX consensus how opportunity we those it, with to number it a revenue you to would give take who give as approval the we look we thinking we intent that

should from ORKAMBI should see tez/iva going to of we tez/iva. from we approval the to KALYDECO With tez/iva, see switches

be models just prepared we will the driven the would I of the tez/iva. work made after about then inventory QX affected anticipate approval remarks, the total with the have think ask the being of year. I So a on Group people that of also in to area remarks, by CF call to growth occurred we're our QX would IR by guidance a revenues. do committed with build through to XXXX, still my the for us And some consistent although

Phil Nadeau

sense the size give of Could inventory that of you helpful. us That's build?

Ian Smith

Yes, but nature it of wasn't very big, but I'll give you the comment it. on

normally So in not million. there buying that The size forward in QX in into $XX U.S. forward some Year felt actually Europe would it, some was overseas QX. and stocking pulled it's have given around the was of how $XX was that there occurred around U.S. New million the the to inventory

QX, So compliance on number if is we still has have great how impact helping it's going more a QX. patients the more, are think terms into good. gets pulled is QX so you drug QX, of demand strong about and in double that but in Obviously a offsetting

Operator

Thank you.

of line Credit Young from question Suisse. comes the next Our from Alethia

please. question Your

Alethia Young

Hey telling guys. Congrats we'll taking on very maybe my Thanks triple. for question. start very, the data a with

long-term question will the filing think talk with kind cell is step in when around CRISPR Just then kind or you're combinations things? And the you combination give in generating what the drive things? is the an IND taken may reimbursement data any and second sickle for updated European U.S. do you can is and as the program about overtime world just most as experience far be design thoughts that the deals, the of of all real portfolio you this of us

Stuart Arbuckle

then Stuart it's Leiden I'll so. here. Jeff on question Alethia question take Yes, cell first the sickle and the will take

behind developing in course that governments is one progress disease but we interested factors treatments making in of can the think that rapid of the these arrangements between patients. in XX% being in see up are of the underlying they I portfolio the treat driving

because most And in making around they've in patients. we're governments rate of so to the seeing that been these can yes how worldwide results that's been just they interested we're are good see and the the one of progress compelling things the

a that's to portfolio wanting about a very to strong So been talk certainly type arrangements. drive them

Jeff Chodakewitz

just the sickle Chodakewitz And it’s follow-up on Jeff cell. quick

We're know to and in very together IND IND expect during CRISPR to putting beta that of with in both the filed then looking when end happen give Beta along this a the and commented that be to initiate people CTA -- we'll the will we and you we sense. have those be cell thalassemia, will file As sickle studies year and that with process Exactly at we thalassemia. better the you CRISPR, for of then further to with XXXX. and going able in to be timing that we're process

Alethia Young

kind bringing of Europe? mean there I any these that on different programs around between conversation is U.S. follow-up and the into Just clients

Jeff Leiden

Beta thalassemia done be program sickle will in U.S. will and program Europe The the the primarily will be done cell

Operator

you. Thank

next Lynch. Ying comes from of Huang from the Our Bank question America of Merrill line

question Your please.

Ying Huang

And data on the duration do given seen you dose as X Phase efficacy guys in on in little you X same the trials. for taking safety data have those or will well rash you for the to Congrats on ask the far well on the Hi, same questions. require bilirubin, FDA FDA X. bit quarter. today's including in as differently the thanks think I then Maybe of X as did designation, two the amount analysis Thank Phase and Phase so the can of happen also higher requirement as lower you. Phase that efficacy dose the

Jeff Leiden

as first learned through years seven the we Leiden of lot a your medicine. I I’ll FDAs six answer together Yes, think number it’s different CF as trials question. these or during about And we a Jeff the worked over last

example to within the an effects be suggest well. we and full that XX going that true weeks as is tend we're know, see you as As everything FEVX to here seeing

points words, think be the months other can time that in six don't I shorter need you efficacy So your the general on data. side, in on

On likely so you important be or and the don’t a weeks soon that’s we other be in read well how long the hand that of as it eight one longer we which in know should database, very that is as big how here what safety be you get of weeks, we’re to particular data safety and is endpoints discussing what and length safety will should is exactly know. obviously now it on four going let and it’s each as those

Jeff Chodakewitz

-- about exposure so in terms dose was question linkage. an doses actually no there mixed across and it’s Jeff and your of Chodakewitz And with real

Operator

Thank you.

of from Flynn Our Terence Sachs. Goldman comes the question line from next

Your please. questions

Terence Flynn

see discussion this the that will the are arm program and include we at inputs. a will all what combo it taking two you And if potential for again pricing, I triple the as And pricing? was level to homozygous today some guys data how at thanks X and high wondering arm think patients does think for on is definitely tell at you. Thank me key the give question. of be will just about us can teza/iva for control some be, then what control change you Hi, framework as can just then that you the point? a Phase about us May

Jeff Leiden

deliver. the terms that KALYDECO the of really at able tez/iva from believe the the get point account are we've pricing comment going the into is the consistently we and strong regulators. we're benefit we're profile to we’ve to you able we it, do patient But that same we'll that to on not clinical for in as going magnitude of on that the got of size account to know, the consideration then is considerations the And and into obviously ORKAMBI we they as are Terence, very and specifically a taken benefit. taking approval population with tezacaftor/ivacaftor

when making account pricing tez/iva we be considerations pending on regulatory taking will decision the We those to same into approval. come

Jeff Chodakewitz

point still remember a patients will these ivacaftor With respect so at most control to on or ORKAMBI on. homozygous likely and be there And yes. homozygous almost control certainly will either the on not tezacaftor/ discussing of that we're be it but on will a be trial that placebo

Operator

Thank you.

Abrahams Brian the Our of next Capital question from line comes Markets. RBC from

Your please. question

Brian Abrahams

the thanks for as questions data and taking the and guys my on well. quarter congratulations my Hey

triple homozygous these potentially the to expect what I I’m unmet and het/mins the wondering think X backups? could broadly wondering are the you dosing about XXX as through still drove still or those Phase is group primarily and your did we het/mins On different to of what should selection sort plans first possibly as see follow this XXX right additional upcoming guess need then the to if just and discussion of for next-generation these with regulatory service speaking to but just concurrently. way I given for potential, Thanks. XXX through under the on of know barb And and the over XXX and correctors XXX

Jeff Leiden

we acute between so really different the is respect FEVX efficacy again and and could Ledien the this factors the of totality choosing profiles all side, with but one again. that the on -- the the Jeff additional co-formidability to well chloride four pill to the decision. means regimens and some those manufacturability Jeff other give certainly in dose tolerability that get -- into side, as respect and Yes, need data to those the With on went we sweat mean safety that I

any XXX our you preclinical box that XXX. bit were and in XXX have, in a XXX said just of As they in example some quite all I that good an check hard as taken you problem had XXX check Phase as data, should differences. mind day good. the give is advantage I'll so it We on were but and a box one to can XXX decision a to them XXX were put that’s was in an once of little there XXX could day it's Well of obviously potentially to have a clearly have a X into and put once going side know, be and a and didn't, you XXX that twice day. whereas a some a

as again want make triple we one two the a any the plan that that to pursue and went your and believe XXX remind notion other race, that standpoint. list get who question het/mins they these data two safety bit safety, efficacy down horse lanes, that we I whole one that And supports about just as have to of then was best it of and clinical was we efficacy, profile in homozygous. out quickly XXX homozygous a out, but overall strongly used alleles went be step should we het/mins an co-formidability little feel in our standpoint or versus and the you the has and these that they As went done benefit and back XX from possible. tolerability or by to has formulation we course sure manufacturability have

we need an effect any also was patients today when on very, homozygous modulation. would right But that you in because that the biggest add corrector FEVX You're I unmet looked a significant tezacaftor/ivacaftor. that the the the remind has CFTR het/mins triples when absolutely you they don't next-gen incremental to have very at

we And regulators so that great with bit leave do we’re slowdown these about every one feel most so benefit need most may and patients not those patients to effectively as as while any in acute and want we or don’t of be how great quickly. that done be we just get may talking in the the populations behind

Operator

you. Thank

Harrison Our Stanley. Morgan line Matthew from next question comes from the

question Your please.

Matthew Harrison

us I up with very the GI didn’t a pre-clinically I you and to of see of it all? I’m so initial I ORKAMBI the rash saw both guess clinical one before to some bit chest follow-up these Thanks match versus about side around trying tolerability you’ve taking I the was studies of little what you guess with here question. I much wanted wondering you in understand just studies, before question a you I comfort about some at unclear just of issues? some some components safety across just at they and you’ve talk Great. preclinical the then Thanks. these what do your And basis on high tacts patients guess is can was for seen you frequency effects seen some ask the commented is for the

Jeff Chodakewitz

saw think picture, no we all that talked it of look whether evidence you adverse the your that's that sense go about it’s matter give particularly actually to that want I about in it's was you we try did it. a with in I a highlighted question is regimen you actually Jeff the that specifically at studies moving a that for couple we comment that's There be comments. the a to look things Phase Bronchoconstriction to in the We do we why really it comes from of clinically Hey how look and them and and don’t see these though combining great through excited that X was Maybe profile standout about of at I we very big is labs that have profile nothing ORKAMBI very efficacy our One and we both no at there information. I tezacaftor/ivacaftor, even SAE’s Phase clinically, no tonight to those favorable it X about hope something had wanted it, of really think and we’re that. events expectations we Chodakewitz. forward. sure

good. very feel we So

Operator

Thank you.

next Cory J.P. Our the from Kasimov comes of Morgan. from question line

question Your please.

Cory Kasimov

would if could be guys ask X guess Hey and taking I the question the data, Tez good to afternoon another my potential to of I duration relative it’s thanks if safety possible standpoint already the durations tez/iva the XXX. questions. amassed want different there for you've from studies I'm way, regarding of Phase curious

how the missed reform, So and I we question standpoint? but might if tax tax the And apologize in one on from call given the already shorter then thanks. today’s be thinking first that I beyond company’s my about there second XXXX should be rate

Jeff Leiden

influence and does of And length new profile. out the what that fact a triple how tez/iva key is drives terms The good here. that very tez/iva profile No any we by combination the what safety size that tolerability certainly duration major that are finding the tez/iva of the deriskers with of first and your respect add agent in agent, you Cory, to have of about a in seen safety safety as favorable the the and new to be won't of the when -- clean the that question database. but with turns be very affected

of we with and have a database discussion and basically XXX two the safety to them combination at new appropriate new XXX the So a is about timing duration agent combination. really completely look that new what's

Tom Graney

kind taxes this. the on just you Cory And of through walk

First so within U.S. the now we right those that we you operating profits. profits as have operating all those I offset create remind losses do of losses

are liability, we have recording liability. we liability point tax either. not cash at minimal an effective And tax So minimal tax this

tax once operations As pay our structure the that through a stop within pay. matches in will we and accounting in taxes. get XXs be company would we and our having tax and the and those the in low results And actually we work that up start reporting we NOLs that had allows set tax global to we rate for,

we so as smaller and other rate have the but And profits been companies lowering of tax because the we like with a have is that of tax outside presence U.S. well. that many U.S. accumulate benefited the reform the also domestic benefit

Operator

Thank you.

Robert the line Our of Brian next Skorney question from comes from Baird.

Your question please.

Brian Skorney

question. Hey for Thanks good taking afternoon my guys.

to understand at Just based now this there start XXX chronic talks XXX to speak on that and you have and is? commentary and seen and don’t there completed plans I if your should take point comment we talks on what you and end what, anything can for around of programs organ species is pivotal there any, duration have you

Jeff Leiden

you could Brian asked clarify you question, the we about just -- and XXX, could XXX

Brian Skorney

Sorry, sorry.

Jeff Leiden

could So the reask just you again? question

Brian Skorney

studies end was seeing those your now organ sorry, in for long outlets? you two terms chronic is two the what and pre-clinic in new what of wondering yes are it on there gone ones, you’ve out have so Yes, anything is I how seen complete, parts talks you

Jeff Leiden

yet nothing The forward shortly. XXX not that for complete plants there it is Phase and very will is it affected was talk chronic to be complete talks our X. chronic for but in the So XXX take anyway

Brian Skorney

acuity And X? in then This no top as you at concern is it right. tez/iva of on ORKAMBI. the just for strategy risk looking drug is on about combining go-forward also There or of a XXX XXX want with Phase to strategy acuity confirm mitigation

Jeff Leiden

Yes correct.

this So a XXX all have profile. a about is clear both KALYDECO and twice XXX PK day once day being

Operator

you. Thank

Our the next Robyn from question of from Citigroup. comes Karnauskas line

Your question please.

Robyn Karnauskas

children and for candy have don’t know to home great call congratulations this we I'm question why data, time. take my with last on my I whenever consider Hi and from unlike with taking and guys, thanks working

So -- to want I ask just

Jeff Leiden

no So problem? questions, to do Robyn happy any those you are take have we two

Robyn Karnauskas

really expanding lost deposits. now, right to are They they're

Jeff Leiden

You shouldn't call. have said a that on public

Robyn Karnauskas

have said shouldn't on a call. I public that

to picture ask question I -- you. big So for want a

knowing you differently that like gave guidance planning $X so your to run is does M&A for exciting. and change have long out fast the -- So going this, this and of you secondly up Do is it to picture results? done? -- eventually, just are what time X you how big million make start goals. to a on that deal? do you the And sure How now go this clearly everyone sort for you knowing size your state, When you once conversion converting sales about the now this I'm remarkable, two data, think waiting Phase company you likely company the replicate even the for do could go

Jeff Leiden

questions good point now sort that quite out. questions, relevant ask and I great management Yes, my of impression agrees. as give data, about a I little ourselves we it working we are team rather on become will certainly for hope I you than see and while I we think you will as they my this how

we that all is to a for this we take of that's need largely CF an CF execution this fully and as seriously move enrolled, as patients. as XX% there, journey this possible journey to finish And of responsibility; very the running in task point finish data where very for we and get these we when quickly First you up out need Phase and to community. out get X

they with As connectors, to on effects we modulatory decision don’t be are on that and any we they take generic is even about some and point CFTR that I continue amendable going CF we feel to said work XX% as further well out. patients have those clinics because a approach have at we considerably therapies although those comedones are won’t need that protein of before everybody. soft when to do who as still need make minimal patients carrier And there working we whether better to it's good next-gen a into like said, a for is

mission XX% CF, see we of of therapies some patients the in can first really generic for just the if the this and this journey was change finished disease. prevent disease get So or course

some And you beyond about with my it’s to CF The this the times. of of journey of Can the HIV question I we another this the answer company so may at why said after do why can sort release disease is fluid done in what's sort and with invest start well JPMorgan, think you're CF. this second do get and in again? of I part you now news as many we oncology, today, do the diseases CF is the we HCV, times you already other in have multiple seen starting perform in an company, or fluid compound obviously sometimes

been diseases talking, on although So starting been a kinds have special innovation to four can are others [indiscernible] and this more including has we’ve company cells drugs of talk we very you about, working breakthrough five is can ones, create as some that nicely. haven’t and it that moving very talked and in about engine these sickle create those know or that AAT a -- and on we or

the financial to to in flavors. through kinds want programs BD internal to firepower some we have this external innovation search our And a then we our revenue of reinvest we because here want supplement of many lot use of programs. to of as we have also will out So use you plan

us do Moderna. do seen you've seen us You've CRISPR;

specialty see therapeutic gene more strategy, capabilities but and see diseases deals early around things supplementing and other expanding making pipeline. markets, and still stage gene bigger of transformative same of and therapy kinds and those our like could ones for editing to will modality drugs serious you You the our of some focused modalities

has to the are gotten we and in plan with why the execution, ways be really executes future about team confident more the gotten really simpler, the company. is many that's of we the way so And the so pleased strategy it's feel

Operator

you. Thank

question line comes from Adam Walsh of next Stifel. from Our the

please. question Your

Neil Carnahan

is on Hey guys, Neil this for Adam.

about you curve what far successful and to wondering as can a you if out kind for tez/iva launch expect preparations to as are uptake? minute doing Just and of guys to expect play you of just launch a how what then guys ensure about talk you

Stuart Arbuckle

launching would Yes here Yes thanks pretty Neil. got the commercial at good team I say products. has

products, the productive new has that is time engine described here, have benefit and time We CF new incredibly which had of, and age Jeff just generated development again. new research in as groups, indications,

much one to we patients well-oiled is management the launches comes has ensured team in things effectively account are are up executing scaled a our seeing. important U.S. additional here go, I have the trained group we they're case the group to patients onboarded the that teams most do expanded because who been ready are So machine of so these anticipating and to say in can trained, for when in the it pretty would

are in near can the we we’re as future. eagerly and we So anticipating ready the be as approval

being in we for difficult which secure -- how we to out, I’m In done exactly depend of to the confident the it's approval, timing trajectory the access say U.S. on reimbursement to do that’s terms to of it's us on the will going depend and able ORKAMBI play for are systems great KALYDECO world will to KALYDECO obviously terms launch those really going going we expect to out here have and then that's about certainly and dynamics and to compliance some uptick, in see compliance how and we ORKAMBI real in see everything of great play and of should persistence would have great anticipating. launch us what and the tell the we

Neil Carnahan

how All about right. they and for one that? EU Tez/Iva how agreements going countries, one had is I other we think with work already have And the ORKAMBI, should agreement then you on the those out just to think do

Stuart Arbuckle

as those we that really we markets time half parts process we the into that think the and products now the you further preparing regulatory Europe what agreement for and is tez/iva which the countries these product in with the with we're question in for stand regulatory EU. are agreements. in either will getting of physicians think around combination a kind an And think an anticipating one answer it's to of get get answer goes agreement pricing we do line reimbursement have these that different the data at obviously portfolio by else’s There is of the is great have patients in year agreements. planning apply as tez/iva. Ireland countries, the agreement And data to to tez/iva we only two an where we I patients this kind portfolio I where is line me really of for our or earlier that in to approval of I Well to some the a for in benefits hard where second of mentioned the the interest I kind portfolio triple give sequential to are whole for approval instance individual for that kind that In somebody you going one approach to of like there different going be ORKAMBI. and for access

Operator

Thank you.

Our next question Carter comes Gould of from UBS. the line from

Your question please.

Carter Gould

afternoon Congrats Thanks Good question. guys. on data. the taking for the

course can you about U.S. studies just and the pivotal see? of then to there And clinical pipeline on we aligned internal triple need pain, internal that your be should maybe of data, and comfort XXXX. the the on will the Thank level VXXXX the non-CF just expect acute talk data beyond on over out First any duration e-regulators combo you. are you safety come

Jeff Leiden

Jeff is this Carter, Leiden.

that regulators discussed all issues to we with studies and year this with both same in patients. with expect thal Phase exactly that we’ve let European pipeline Sickle agreement much call. both you start talking we’re get know, to And is non-CF the said the look. we about the as cell they'll So of goal course when in X our as between those possible and those And of U.S. studies know respect how regulators program regimen to beta we them aligns you those

expect Internal predict clinic will early clinical exactly first see to when also year. this start or the little It's to other more We Group one from to our a enter Research programs data. the we

Carter Gould

Thank you.

Jeff Leiden

one Operator, more we've got question. time for

Operator

the comes of Deutsche Jacob final line Bank. our then from from And Navin question Certainly.

please. question Your

Navin Jacob

Jacob, if taking May thanks questions Deutsche may. here quick question. Hi, the be for Bank. Navin two I

does which XXX is Phase and are first have taking X follow-up then I and of you XXX a commercial question. The into

Jeff Leiden

the with we that’s Yes, haven’t regulators. of doses we're yet finalized discussing that the one the things

it of and that the dosing dose. very response with we the a actually way One feel us we're of very compounds make did do for we easier did have these to best occurs that for pick both pleased we that studies reasons the results. the the that will We exposure

Navin Jacob

capital expand think you times that And for apologies brand severe of my to do investing think going then very quickly here has in same at you how be may, to but cut generating you’re time you in the often people new disease orphan these but yet value that about the for if amount commercial spent I price on drugs patients, diseases. Vertex populations as have significant het/min

should thinking is the or you we And value there see to actual efficacy KALYDECO the that especially given case Should be do our patients? the you’re question versus value providing you’re showing so pricing how be economic that discount here assumption flat I about you’re guess providing is that or that a actually here. even the base

Stuart Arbuckle

forward Well, the combinations these that until instance for not about the have into based Phase we Obviously that the on development, value tremendous having we're about certainly that the we we've X moving specifically announced safety data release today data triple and first patients clinical efficacy released start pricing. in speculating the we're profile demonstrate opportunity shown. just to provide think

today the as most us is CFTR important that's and take economic The going able two forward. the will the a into are the have with I've are providing of of considered consideration modulators we that clearly we those account with throughout continue in we the we’ll to is that released data path and XX% number primary benefit which we’re goal. that’s line down of the be them, terms considerations something always taken same that we benefit to the to for begin clear In that’s way considerations patients our and magnitude we provide thing value to gives said move the of it the that patients

Operator

I’d Thank the of remarks. program for does you. to today’s Michael conclude like this program. the to Partridge further hand back any session question-and-answer And

Michael Partridge

it turn Jeff actually Leiden. I'll to Thanks. over back

Jeff Leiden

see special you people, been remark the towards investment I think day the foundation then And the Nick have you model don't we breakthrough employees back well different one revenues years who just remark bodes in us Meaning very and out I we with do diseases before. and just we journey. had that want breakthrough and get today. products better And future finishing because CF now. to do this into strategy here. caregivers from the and that to it’s seven working Michael. disease. years said that’s reinvest may of a an with with I an with be me thanks which and scientific other nicely an stuck but leading done absolutely families I in dream think thank just with I a to think that that and and innovation thing, better when be to very may the echo strategy. a again pleased disease. because understanding community one Yes, internal internal where certainly being step parents of is I one and forward just standpoint the important It's start as incredible caregivers want all external more are I'll and to in that to been started that more are and that you see then leading in one. further very the to what pleased It's and that we this six, products Vertex we’re progress years I'm journey fact all get to those disease, for special committed to the often medicines and in for we we a two and remind we and able make patients it almost external can us XX the for really was are seeing ago one XX and play come an remarks

Michael Partridge

good is thank Thank for call. everybody a office that to for us Relations you, tonight’s The tonight additional the Jeff. This to Have joining evening. Investor your question. I'd and like team concludes you in questions answering the have.

Operator

conference. participation in you, ladies today’s does for Thank gentlemen and conclude your This the program.

day. You disconnect. may now Good

Vertex Pharmaceuticals (VRTX) 1 Feb 182017 Q4 Earnings call transcript