VRTX - Earnings call transcript Q1 2018 | Vertex Pharmaceuticals Incorporated

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Operator

Good day, ladies and gentlemen, and welcome to the Vertex Pharmaceuticals First Quarter 2018 Conference Call.

As a reminder, today's conference may be recorded. There will be a brief pause and then the conference will begin.

Michael Partridge

Good evening. This is Michael Partridge, Senior Vice President of Investor Relations for Vertex. our to XXXX the build cystic discuss our results treatment will fibrosis. progress continued long-term we first financial quarter and of Tonight leadership in Dr. Chairman CEO; Leiden, Dr. Jeff and Chief Medical and remarks Chief evening. will Kewalramani, Reshma this Ian Smith, provide Operating Officer; prepared Officer, Arbuckle, join for Officer, Q&A. will Commercial Chief Stuart us We as listen access to this slides you recommend webcast call. that the you The slides are available for download on our website. later being recorded be website starting will and on replay is tonight. a call conference This our

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

Jeffrey M. Leiden, M.D., Ph.D.

has the half started homozygous to approval U.S. or our anticipate of off patients The people never currently and discontinued of transformative approved the Thanks, this in Michael, approval in innovation months of patients established disease on patients and more develop is deliver XX,XXX a the CF. particular, the new worldwide treatment The important record to an half launch to CF few with second medicine, CF an for of eligible discover, the number people about third continued our Today, first good Vertex, FXXXdel in one year. to these of treatment. XXXX year are many who EU and track of SYMDEKO, medicines for important we in of build modifying on offers and for have grown start option. In to those medicines is our evening, this year, who and SYMDEKO strong everyone. ORKAMBI. we

we to With the growth of new continuing positioned are significant reimbursement a and launch beyond. XXXX in the revenue earnings agreements the treatment of option see SYMDEKO as completion U.S., and additional outside and

can FXXXdel many future we as evaluate to VX-XXX triple and combination announced progress one our X mutation, regimens benefit in modulators to the Our tezacaftor one groups of is and well two in more next-generation treat function mutation that with with patients on based that CFTR that the enhance developing corrector. and had initiated we rapid belief we patients, a further include this Earlier of triple-combination as minimal studies mutations. year, Phase two those ivacaftor FXXXdel with those in in

start the forward patients pipeline development The as of the part research regimens bring the the of Phase two patients marks regimen also CF. our have a first Phase in study announced in and study. sites minimal have Today, begun next-generation that combination VX-XXX, for open we for triple same function also to one our second dose who X mutation, in patients regimen are only us X corrector, we of VX-XXX. generation CF evaluating triple-combination important advancing making beyond people and possible. toward that development as of our goal to in with allow choose best to This progress different We're important quickly to groups with progress we're next and as

with Therapeutics. devastating investigational cell diseases, disease Gene beta-thalassemia our to CRISPR CRISPR/CasX partner, We and development sickle Editing in CTXXXX, begin are Treatment an preparing of clinical two

year. lives plans. additional move forward expect In also make to VX-XXX you inhibitors pain. on Tonight generating medicines to XXXX development we the her on important other future forward continue data We record our Jeff more look experience the as the studies I'd XXXX, and now transitions during as with turn the successor through depth selective for Chodakewitz, senior and to also joined to status her in with clinical proven serious CF his his of and internal of XXXX disease. Reshma with over appointed these We inform clinical have Dr. ongoing remain pain I'm to continued Reshma medical ideal potential in retirement. thank to to I clinical extraordinary and personally Officer. from Kewalramani, a our CF treatment he make Chief call knowledge, to in the or look as review Medical Dr. of call, from with an dedication will to newly the programs with to Vertex who potential detail. paired Reshma track into the early pleased her one programs a other for updating diseases. like to of leadership of NaVX.X I'll medicines more planned Jeff leader, progress VX-XXX progress and our improving development us adviser research our people to us

Reshma Kewalramani

to good once-daily for I'm our progress regimens and that pleased X VX-XXX into potentiator. Thanks, triple-combination combination share to once-daily VX-XXX, Tonight, VX-XXX initial review everyone. the advancing Jeff, include results evening, very and triple Phase and regimens in development a

regimen. to First, triple-combination VX-XXX the

triple During we VX-XXX, with CF. announced studies combination regimen of initiation X the XXXX, quarter of of Phase tezacaftor and first as an for ivacaftor, the two investigational people

key A NDA who generate well the submission. approximately of as and feature to function and not assessment weeks will the complete on form shown data through these older VX-XXX this study The that is required XXX have The The assessments the minimal four this of weeks versus on at the XX triple-combination in evaluating regimen patients key ages potential But data a are design as safety. safety placebo is mutation. assessment schematic slide efficacy endpoints a of XX is XX-week basis submission. secondary a seven. will NDA first study

in weeks FXXXdel dosing additional in to key As added conjunction study in tezacaftor a treat also in to regions. patients are after regulatory open, support VX-XXX the the of minimal regulatory this been we study placebo eight. VX-XXX where efficacy and anticipate with FXXXdel FXXXdel the To mentioned, where this XXX ivacaftor. primary submission this planned four-week on study XX-week homozygous four safety the study will of study of the begun sites all Europe XX of the population, support enrollment a VX-XXX features design from in ivacaftor and homozygous patients run-in approximately and in these receive The used tezacaftor Jeff older. submissions second other and have dosed. the has efficacy is are patients patients. The evaluate data and data a slide first and shown study patient U.S., from studies is assessment or to from Data be using ages A will four-week function triple-combination then

two program for initiation the VX-XXX regimen recently are different VX-XXX opportunity as VX-XXX, regimen the to the best studies on Initiating from to and prior I expect of Phase Today, for Turning generate as discussed for review patients non-clinical our two announced us respectively. to to the X bring pick to VX-XXX studies we both of for Phase shown triple-combination FDA's results X the X the patients triple-combination quickly the with just X possible. and gives Phase to CF. Phase of with data start data VX-XXX. similar toxicology studies. the obtained are we studies VX-XXX designs X We The of slides study XX, and and and regimens these

regimens in for with the the When we evaluating year, I regimen VX-XXX studies, once mutation. we mean in each of what part for Phase minimal tolerated was previously study. and VX-XXX four-week function patients. consistent we've that what observed that with combination are of again, X the regimen. improvement in to baseline daily on with and in weeks was generally these observed This the triple four-week to slide daily in the results as function reporting and the reported VX-XXX triple triple-combination from addition regimens, percentage received results function In that tezacaftor initial through profile shown in after VX-XXX people VX-XXX percentage ivacaftor were X The respectively. patients observed safety VX-XXX similar VX-XXX with that with the Today, this this have of well and a The in Once X announced This XX. discussed, X improvement population. once These function of VX-XXX. or ivacaftor. generally efficacy reporting tolerated absolute the In of tezacaftor from residual are triple patients homozygous tezacaftor XX.X triple-combination triple-combination significant second Today, we previously placebo and just obtained were Phase is ivacaftor. alone. and advancing in also with or run-in a have In treatment and triple-combination was support four by combination evaluate FXXXdel X well of regimen consistent dosed XX.X initial this we for a Phase what through week Earlier VX-XXX in improvements VX-XXX each parts Phase FXXXdel or evaluated studies four the XX sustained was minimal evident observed gating was over the were results of we included for combination who safety points profile ppFEVX and Phase with the safety also potentiator, lung of learned studies treatment week in impressive tezacaftor are of observed combination plan triple-combination triple regimens we for in dosing data one period. this combination in and points these studies a the data the patients combination, period into mutation. ivacaftor who with study to VX-XXX

we you of support potential FDA clearly ranging development. our before turn of look in once monotherapy While to forward with for advance on Ian. the to we VX-XXX, requested our updating triple-combination the the evaluations We combinations, in as daily has progress VX-XXX programs. that including allowing that role triple future over call evaluation the now believe a hypothesis regimens I'll additional VX-XXX these results late-stage has dose

Ian F. Smith

recorded everyone. Thanks, Reshma and start XXXX our I'm SYMDEKO XXXX increase product in XXXX the a the of the the million to $XXX U.S., XXXX. review million tonight, off Revenues financials, first $XXX strong XXXX of quarter is guidance. we launch good full year first, pleased evening to total first CF and revenues in quarter XX% and to the to financial represent our first in a compared quarter

of continued eligible product number revenues have about approved to we estimate grow approximately are are being our as we increased our patients which quarter for Our each medicines of the patients treated. treated half XX,XXX with total Today, medicines.

U.S. the of revenue therefore continue brief we been the make and approval to grow secure XXXX, XX, eligibility educating on patients with and the SYMDEKO launch drive on the We in treat some expect to throughout I'll healthcare medicine, have in Since reimbursement. number to we working continue to growth. FDA February the payers providers on comments

coverage commercial We and SYMDEKO, broad access government payers are to are and the majority the medicine. of reimbursing as for seeing

the we the sales. of For XXXX, revenues quarter million, reported of initial first seven weeks $XX which SYMDEKO reflects

for prepare of to EU in in half continue approval the of second XXXX. We anticipated medicine the this the

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

billion During $X.X marketable quarter balance we to the with sheet of we compared billion our at in beginning cash first year. March securities equivalents this $X.X strengthened the approximately as cash, XXXX also ended and

of where of Now we of the revenues full billion the countries year XXXX continue in comprised to product XXXX guidance, turning midpoint driven represents are three over treated $X.XX the approved medicines XX% our launch range from to currently of SYMDEKO combined with total reimbursed. number range primarily by patients revenues billion and CF expect $X.X this approximately an to increased The medicines. approved of our growth

previous ORKAMBI the switch and KALYDECO patients on on focused is the amount of We CF commented timing product CF The in and some growth SYMDEKO. have and are our guidance, that metric the because we growth not from total we revenues given patients. yet known. believe will of overall more on As to treating revenues calls, important the more CF reflects revenue is product switching most it

of support supply expenses potential are billion the R&D the $X.X triple-combination We combined key execution $X.XX to regimens, of also to studies of and of investment drivers two a commercial launch The triple-combination pivotal the chain investment continue investment success for non-GAAP, expect SG&A and for to billion. incremental SYMDEKO. regimen the

profile has over years. two the financial Our past significantly strengthened

Our parts of our all With has operating continued number patients and open significantly with and execution beyond to our questions. business to us increase growth to and of will deliver earnings medicines. revenue to treat positioned line as XXXX the across expand that, we margin we our the in I sustainable continue

Operator

our first Porges question comes Geoffrey from And line Leerink. of the with

open. is line Your

Geoffrey C. Porges

understanding the little only congratulations FDA's might if KALYDECO. finding that that Thanks a actually version, The class? It beg as – the just trial that going much of only is it's enter you the What's for given on ahead very I assume to give results. single one that the be to a with VX-XXX, to and I surprise a that a every it's pivotal we could us taking potentiator should unusual you're of of going more for seems basis pardon, the for that the caution? well, basis strong And isomers applied decision. drug little question, and is wonder dose the with same deuterated bit standard your potentially really of not

Ian F. Smith

in pending go into would back Maybe talk regimen insight was incorporate more year, into maybe Ian. Leiden and the remind intend Geoff, the earlier we have our with that as about separate Hey talk we I question give you, we which it's from you FDA and to maybe to results pending data we we VX-XXX. two into data getting forward actually this we could Jeff how It discussions that. VX-XXX a parts, can look about little forward. and Reshma

strategy talk incorporation? to then want the you to do first, Reshma, the data Jeff and So about on

Reshma Kewalramani

sure. Sure,

we the VX-XXX safety. know, So, VX-XXX one walk of had of efficacy as that terms let you And in and studies, the part results the proof VX-XXX through included and place me both ivacaftor. you of concept in in

impressive very On the points, VX-XXX. had and efficacy of in the for with. we XX.X and ppFEVX side, Really prepared results VX-XXX respectively, as you improvement heard that XX.X were in pleased remarks, percentage an we

on looked and the the impressed On impressed these and with consistency safety of in ivacaftor with results, remarkably the at safety we've our we saw we side, similar when safety, to seen were Jeff? what very results profile. And both the we studies. efficacy were what

Jeffrey M. Leiden, M.D., Ph.D.

Yes.

despite monotherapy that, as particularly to Geoff, the for combination go FDA, kinds X So several you results, a as of ask and chemical it got things at information you would Phase last way became a potential kinds entity. the goes have we've say, before you FDA into the that's those VX-XXX design that that ranging to we some months. weeks exactly a is fact the deuterated said, of over it's results discuss clear And of the of the Phase they and new obviously, did us KALYDECO was And you it with the and need looking the typically really X and version they're entity, before for, is a regimen. for instance, new asking dose chemical the which

of with And and VX-XXX best that's always possible, forward we to and we move decision that it both a been the been so that, that many studied fact efficacy because want goal the and, in in so delay. very, probably the as said terms with our patients KALYDECO of regimen patients has to influenced has decided think also so take quickest – a safety. high to bit was to because very set decision quickly KALYDECO was route bar, as I Having a our frankly, the get thousands didn't well by

data in it, have of going wait. plan as quickly think day said, comfort enable that we forward need they to into they made X of more with to in it, quite want a is X we that's a all don't the to studies. a VX-XXX for us so do regimen. But data Reshma lot these, forward We're exactly take think, Having about it to to that, with And compelling. discussions are what some that and highly We doable, with once move and FDA still Phase regimen. easier the Phase just I said

So or one we as VX-XXX bring studies patients. as soon either with progress we allows VX-XXX that, bridging to those choose a know out strategy work we'll along that and to we'll VX-XXX whichever us

Geoffrey C. Porges

for it. Thanks background. that I Okay. I'll the in appreciate get queue.

Operator

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

Your line is open.

Geoffrey Meacham

and I have one. thanks and one commercial a questions. a the just guys Hey clinical then for

you when and process, a to follow-ups just Europe about, including SYMDEKO, reimbursement For road down abbreviate the I'm the and process maybe the approval be on what then trajectory Australia learned ORKAMBI? from help O-U.S. Can across of have clinical look the speaking lessons you are side. I couple

Stuart A. Arbuckle

Thanks Geoff. the for questions. Hey,

with So the ex with what on on ORKAMBI looking and, from in successes. markets begun build of In try and SYMDEKO, we how be then experiences reimbursed we we're successful SYMDEKO. SYMDEKO. of with certainly we've it. half kind those What we get Geoff as in only yet where U.S. waiting for that able access we ORKAMBI. been with may pick agreements authorities many when with where about U.S. be approval accelerated we which our we very Obviously, actively very, haven't though, those anticipate they'll Obviously, learn pursue, to about ex really those is in also obviously, ORKAMBI steam, would I we'll Europe ORKAMBI, thinking can patients are getting some priced and this more SYMDEKO countries to very, very to some stress like discussions for for not year. have to we second clearly, and up the portfolio active, the potential get to going continue SYMDEKO get reimbursement discussions reimbursement for of secured and of That's not we're

you incredibly that important, certainly early As progressive know patients know, as with modifying have treating with them agents a We is disease. relentlessly as possible ORKAMBI. disease CF like

every can, continuing that ORKAMBI. of we're to we pursue so, for urgency And reimbursement with degree

the think to we've recent ORKAMBI is approval XX, point, first is thing over. XX and SYMDEKO which to I And know, to remember, important other down to five. likely hopefully that expanded in you for as is The an two be six to for subsequently people indications to the years

is ORKAMBI to play role children So very, with to continue those younger going a important very CF. for

Geoffrey Meacham

And is then Stuart. more helpful. the And the in would be upper actively objective Phase or an investment still but on into assay strategy to figure you X? worth you it pipeline Are future of that's VX-XXX, end you I'm trying pursuing other VX-XXX, is an words, not? think if have of or as it for predictive kind FEVX, just worth Thanks just real a on few the VX-XXX – triples, to that's about combinations In of go beyond larger the studies side, points the I get your guys then novel there here? what out with when FEVX?

Jeffrey M. Leiden, M.D., Ph.D.

great question, a It's Yeah. Geoff.

into are As research number have now before, flowing of additional development. I reasonably we and next-gen correctors large said that late preclinical through

that potency. the dose, significantly at is significantly two to along whole quick forward If Phase things of decision we a But they a if Phase it's we'll decision. that's the better currently other efficacy, to quickly some the once the That's moving have make, VX-XXX decision the two, we X, right? All VX-XXX. of we easy X. into day but go we and better better, are and don't, we're take of really So also the moving those than the profile early won't. medicine, it molecule for the feel that will than believe those significantly And going end certainly into is, because certainly it and are do. we be have instance, it's our are will and we'll formulatability, day, to start do so making soon And forward, into

Geoffrey Meacham

you. Got

you. Thank Okay.

Operator

question from line next our Yee comes Michael the of with And Jefferies.

open. is line Your

Michael J. Yee

on the I congrats SYMDEKO as launch to off. well start And question, Thanks. two good part Great. guess. Two,

I concept are to follow to needed for it Just on would of regulators be compounds. to up VX-XXX. whole I the saying on dose more what ranging the read-through an seem novel important guess mean, studies

to clarify. just So

out an You're a program actually as as you run new full active it that implying and that need potentiator it's to and a it prove monotherapy compounds. potentiator prove for

on it And wanted clarify fast. I and would mean be that Just Xs pretty started to I dosing then was presume are that. the that like question the second you sounds ongoing, patients. Phase

to beyond forward we're So on after wouldn't done guide want your these to weeks, you while four what it's Thanks. I Xs would XX would data be data, actually do need all or necessary announce disclosure you you and looking to? Just Phase when policy file? that

Jeffrey M. Leiden, M.D., Ph.D.

second view Mike. is to the This I'll going what to part. that your it's never informative think Ian will Maybe other I Porges Jeff. KALYDECO, they respect different. clearly I it's similar first mentioned, disclosure the Yeah, comment people's one, on want programs. was as do FDA the because entity, to the a is take part, new chemical Geoff first as take but question. With VX-XXX quite for

and to more them. pretty more for think different board, across to newer, and as I that's don't chemical but up I they're and unreasonable. the new going newer have that compounds Obviously, obviously So much same expectations think it's get entities the

Ian F. Smith

we Mike, that in your time. this the question design what to in and really on And include disclosure, point to disclosure I don't at want

that, filing So it. we provide study, further gather it usual, that. inform a we let we'll as can supports and and you data the And in we'll does that know you of have be that But the think studies. do we'll it data know the when when you data what you us will what the we're that's the I provide could action into us we'll we'll And with timing complete let we'll what strategy. imagine disclosing. to you will be – data, the And terms the

Michael J. Yee

thanks. Okay,

Operator

you. Thank

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

line is open. Your

Ying Huang

Maybe designs Phase taking my for on the the one Thanks clinical Hi. questions. ask Xs. for trial

testing are homozygous for XXX the You in for X the guess patients I patients heterozygous Phase mean, trial. testing in combo – about het/min the patients, while the XXX the are – you patients triple sorry I in

I is superiority efficacy Can powered then it's to maybe sufficiently So supplemental wondering on show SYMDEKO patient commercial to if in was likely launch. ORKAMBI or some compare the you're today? for on who are early other And side the based patients who most you homozygous mostly secondly, or taking patients you. tell those efficacy. trial a switching SYMDEKO the trial Are the patients SYMDEKO? also experience, of Thank were not before, to tolerant seeing

Ian F. Smith

one study Hey, you The does in and trials, answer, want you het/min one Reshma follow. one XXX, understand I we patients lead doing the can two patients. in. are of study Phase patients just how het/mins, help your gives correct the X XXX before Ying, and Phase obviously would the And in to XXX Phase different and het/min X maybe in, how points, XXX have now XXX, X has XXX Reshma XXX, XXX will study just

Reshma Kewalramani

Sure, sure.

of the VX-XXX very each VX-XXX, are and programs, for similar. our actually So programs

So XXX FXXXdel total to going to there's VX-XXX. but In an study use patients of very patients patients homozygous XXX for het/min total similar as and I'll a each program, patients. it's example be VX-XXX for

ppFEVX just can back if is the which Now homozygous take at the studies, more comes that desire fairly things regard we well our pulmonary the proof for see powered. you to exacerbation With going chloride, XXX patients. in that at look Indeed, have het/min, to is of from to XXX a improvement like and sweat you even look substantial things like that patients CFQ-R. study, concept

Stuart A. Arbuckle

seven launch from. And we're in the the are end SYMDEKO QX, Ying, stage are a launch where but mix. patients obviously just at to the seeing tell And early on those able the we of coming into we're launch, weeks this of

not KALYDECO ORKAMBI to groups homozygous treated never who ORKAMBI importantly, a And discontinue in on areas. patients then We approval are the the are had certainly being those the you patients seeing patients modulator, on SYMDEKO. but in perhaps we're use being treated seeing that's being incredibly unfortunately a data, in were seeing those clinical with who important had been but we're CFTR And initiated of who use patients are last is transition we're two to who those. seeing of prior on CFTR based and initiated agent to the because mentioned, modulator those really the which launch strength SYMDEKO who of and SYMDEKO, were also One from had were prior more also also naïve, patients obviously, disease used not medicine. and few with in been modifying

Ying Huang

Got it. Thank you.

Operator

Thank you.

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

line Your is open.

Terence Flynn

X inventory you give be of question. more idea you what And was triple Hi, on just could on say and then, little thanks Thanks. closed. can just just for any rash sort front? can it SYMDEKO, a anything that tied agent just question Maybe for a Phase for And wondering you the Ian, in quarter? the of any you the might taking the to, that quantify that if combo us saw you I bit detail on couple of cases

Reshma Kewalramani

Sure.

XXX So right you are now question means low me side. that rash with is severity. that have our and and about on that of we is had start let patients. the Where treated overall VX-XXX, incidence what studies the proof we we've experience, a XXX VX-XXX clinical we've for In and the low of patient both completed that concept

you the and program for quality rashes you'll X%, without resolved you a their of we're are a that therapy period our look is or seeing context course there, see And or trouble. on is been for USPI in we've point interruption. These and serious what's KALYDECO and with seen and then no restarted have very in about discontinuation completed have similar their some and can rashes this, latter had the interrupted couple incidence and XX% have ORKAMBI that interestingly in treatment where ORKAMBI, We events. of KALYDECO next-gen so. To about patients and give cases is

Ian F. Smith

SYMDEKO, channel channel the which on of first I'd inventory top that, and question March in inventory CF XX, total your revenues, at XX, to line in was the comment the the you December the on revenues to kind Terence, similar is to XXXX, draw the XXXX. at

So the what and demand QX. between has you periods, that in is those number that tells real even revenue channel remained two therefore, the is

your some December demand CF there decline to build. period. ORKAMBI QX real and channel slight was but that XX to it As summary, question was build, from to channel were In KALYDECO in some offset not total the revenues on SYMDEKO, was channel and

Terence Flynn

a Great. lot. Thanks

Operator

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

Your line is open.

Brian Abrahams

combo very questions. as first Thanks amongst taking filter patients to Xs. my to the hearing and really for study. about I Hi. on triple guess, needing is of sites, who eligible we're enthusiasm Obviously, a lot Phase the off, much sites for

So, quick perhaps the had my highly Phase operationally, you I motivated then guess, reflect question come the are into for X. follow-up first really in different And managing how skew these a on potential is Phase healthier maybe patients more Xs or VX-XXX. studied population to I that, and or the results

Reshma Kewalramani

Sure.

as what exclusion is Phase we're and of and Phase you and know X. such, the inclusion Phase now for with that the some in ourselves in what to fairly patient time And X not populations well patients we similar we're sites CF VX-XXX. now the worked the executing well, discussions only So Phase sites, in way doing screening just the benefit and community are completed are having patients described, criteria, with but that the studies, we X X studies VX-XXX have in studying and in this

on the which I these very operations studies. So and the running enrolling good about are feel are timelines how we

Brian Abrahams

Great. VX-XXX. question And maybe a then, actually on

non-clinical you're the of tox. FDA awaiting review mentioned You

you Thanks. just say or notable could any check? whether that's if observations there if were a wondering any – Just was box there

Reshma Kewalramani

Sure.

the chronic that know You the results standard in review at don't preclinical looked are see fare. the talks, agency talks We've We but of anything to course, there, has that. it.

Brian Abrahams

much. so Thanks

Operator

line our with of comes And question Phil from Nadeau Cowen next and Company. the

open. is line Your

Phil Nadeau

and the Good afternoon. progress question. for thanks Congratulations on taking my

to similar there? the the that and seen those are there in elevations data. VX-XXX on you've It concerned be question agents in one to look what some Is like homozygous enzyme other study. about for past? were First, there rates anything Or liver did

Reshma Kewalramani

Sure.

detail. at as there, what you see trials. great other we've looked imagine, interesting or very we we've don't safety, seen can our different with no, anything much in the And So

Phil Nadeau

then, of you a a dose you could in on Do that multi-year And long can finish that process? rough you to or need and XXXX, do how it move forward. when sense Is the this QD possibly you that think Okay. is the exploration have something take? second regimen will

Jeffrey M. Leiden, M.D., Ph.D.

agency, a We're to discussions the too early give so precise in thanks. answer. you still Yeah, a with little and it's

And when soon. want you what think about know most we're But do, we'll I what give don't we so you we plan I is. until discussions, know hearing very let fairly finalize But to precise those the of far. we'll straightforward timelines. is

Phil Nadeau

Great. Thanks questions. my taking for

Operator

Morgan Harrison from comes Stanley. question next Our with Matthew

is line Your open.

Matthew K. Harrison

I great. thought off I'd ask and second. CRISPR Hey, Good the about a afternoon. program change it for

Can some bit get and you CRISPR you steps sites? the I next mentioned CTA, are filed to and sounds left has you've the terms and pending starting CTAs some in about talk just items open approved. dosing are pending. mentioned gotten approved little that more other still one So what like are of what think It a the you've

Jeffrey M. Leiden, M.D., Ph.D.

year Matt. different related cell and you Beta-thal, study beta-thalassemia of folks is disease. to but Thanks give question. We're you're diseases, later I submitted in And also Yeah, anticipate the that, for dosing has beta-thal, we starting we we on file U.S. the we on track of sickle In file exactly just do for to for depending outside and Europe. know Jeff. thereafter. cell aware CTAs, disease beta-thal dosing when the sickle in we'll actually soon mentioned, IND to again, one this. those planning a And Maybe for this U.S., CTXXXX expect multiple background. been approved. little as an are This to we two begin

is us hope both we'll and my to dosing So some this patients. that year, to should start generate in diseases in data allow be

Matthew K. Harrison

Thanks.

Jeffrey M. Leiden, M.D., Ph.D.

about excited know, you first editing We're gene trials As be in that. it will the pretty likely people.

Stuart A. Arbuckle

would add to just it. actually And I

we've this We say these excited CRISPR one gene I'll first say how and trials us opportunity are we that is for product disease very in say collaboration pretty the there, very exciting with opportunities, want made partnership happy different Therapeutics, very just is, CF. also progress with I Part we're a to it's and stage collaborations to collaboration came the with us and that CRISPR at opportunity in that clinic patients. Therapeutics. of, and this give towards from this that growth expansion the editing a progress the But form let's than

Operator

you. Thank

of comes Kasimov JPMorgan. with question from next Our line Cory the

Your open. line is

Shawn Fu

question. Cory. for thanks Congrats Hey, taking the guys, this my and on Shawn is quarter for on

daily. on Just another once one maybe the

at So absolute and VX-XXX, when was but adjusted looks similar, for VX-XXX And the to to it the not understanding ppFEVX, a on bit at forward the since data contribute the that Maybe placebo on you VX-XXX at the this could quite directly least placebo looking for comment did with be once daily. quite better, if to that. decision in then, VX-XXX move just did difference all poorly.

desire dose Just the kind given dose is thinking the for XXX-milligram all maybe that for? other the FDA specific is there the rather making asking for that to being before XXX trials, used to things of VX-XXX final a was the addition the the decision than look in at

Jeffrey M. Leiden, M.D., Ph.D.

the of it's pretty different ppFEVX, of studies Yeah, to are chloride, in by with cetera. that's as generating these all sweat in patient. et to us significance but obviously that almost the and in at important is trials data. the CFQ-R, very of consistency remarkable only impressed small terms way, are that Shawn, not numbers statistical six the thanks results, data, placebo-controlled for four me very Phase the terms seeing that thing of And identical in of and we're different question. terms It's we see the most, different to X agents Actually, look combinations

difference the is We So a the I different guess, of to remarkable the numbers. between interpretation are our between board due differences don't patient see consistency across what's just small really regimens. is VX-XXX. those Any

I no, is a just so, a with our of before appropriate of believe their chemical didn't we'll about really data into X that. matter have the their nor to drove and proceed desire VX-XXX. how early entity any to set and It's that Phase do we decision-making get it decision-making drive this new move the FDA's And view that

Shawn Fu

maybe It same, then residual memories number of Can looks and Okay, great. on you for listed sort of are number the KALYDECO SYMDEKO. residual mutations refresh of XX their our the modeling function for the function like breakdown question. XX worldwide? a And for about patients on just KALYDECO, the labels the SYMDEKO

So on should patients there residual in KALYDECO X in the X,XXX U.S. looks kind like conjunction of about are for that based ages plus covered approvals be currently the KALYDECO patients, your announcements with in it just for

Just this patients SYMDEKO how roughly additional many and residual is for are wondering how many this function there O-U.S.? that plus given then patients in XX

Jeffrey M. Leiden, M.D., Ph.D.

Shawn, we'll call. after back you the So to get

want did sure your was. ask just to what – make You for the ask

You patient population. call We'll know get back want the total RF to the on you that. to after

Shawn Fu

enough. Fair Okay.

guys. Okay. you, Thank

Operator

Leone our And question next from of comes line BTIG. the Dane with

Your open. line is

Dane Leone

Hi. Thank you. starting wanted the kind to on ask an to follow-up studies. Congrats question. of I earlier

terms any with the curious, is rash the of of you And generally elaborate extended you. that of about could patients we've XX% in safety around there the the profile KALYDECO? VX-XXX? any profile think some with the KALYDECO. seeing appearance you're in commentary Thank that reason with safety Regarding just in more molecule to it your of could was I seen half-life exacerbate that seeing

Reshma Kewalramani

Sure.

looked the as program we efficacy the is regard well to at in program, real from what consistency VX-XXX a with the data either VX-XXX when profile. see or safety VX-XXX So we as

Jeffrey M. Leiden, M.D., Ph.D.

way to rash said seen hasn't of both other And uptake or low as of the with medicines. know, before, the those here. are as just and be you the utility we've They're to what medicines. any incidence similar that clear, in very affected Reshma severity And

the of So we're very this pleased with profile. overall benefit

Dane Leone

you. Thank

Operator

from comes question Carter next Our Gould with UBS.

open. Your line is

Carter Gould

I and I guess, quarter the impressive on for Congrats data. Jeff. afternoon. two, guess results Good

a the combo, arm around you studies. could got active design that. data, you quality arm execution The a any on now, bit on and shift X day of bit that just attention a with little BD the add more right on entering just then clarity the some Thank or prior relatively just in Maybe of context placebo And consistent the looked focus a life seemed once anomalous. Phase Now the the phase front? VX-XXX you.

Jeffrey M. Leiden, M.D., Ph.D.

the for questions, questions. thanks Yeah, good both

First a one BD. on Really, shift. there isn't

remarks, his several our been up consistent As prepared strategy early-stage same transformative platforms with revving and Ian we for have around efforts said years CF the products. the in last accelerating, BD

be active very that to in look continue We space. and

Parion, we the in flexibility have more a similar those And say, cetera. of because You can deals that we've et more expect ones to see us firepower, we done to size to already, Moderna, the do more or have with deals. terms of CRISPR

even be may than already. the them we've ones of some so And done larger

Maybe, think research really certainly of focus a diversifying our It's little a just don't and want do there's part So you internal collaborations acquisitions. external our CF, pipeline on and but change, I beyond arm the both placebo there's effort. or a very CFQ-R? active on to Reshma, through comment the

Reshma Kewalramani

Sure.

are double-digit we study, VX-XXX one all populations studied, improvements in studied, see rightly the that is actually looked right. that placebo difference different group. at plain the two value we you there is that out In you're VX-XXX, across as the CFQ-R essence, an the It's comes all in point you So across from are big and eight, CFQ-R study, VX-XXX doses the what just and and small that and there. anomaly it the anomaly But that of results drove out in there's patients that numbers. we CFQ-R. In patients two what

Carter Gould

you. Thank

Operator

you. Thank

Alethia Suisse. Our next from Credit question comes with Young

point Where you Securities the - more is bit wanted little the on taking line Credit your Your talk it, done there's and or Thanks talk in Thanks. about a have if presenting Alethia you've of do strategy forward congrats and in between questions like the M&A past. guys. commercializing? moving true And Young progress. partnerships I this NaVX.X, you can just on and just the a LLC my Hey, One Phase we the do I about open. X data. to Suisse plan there. more all kind think at (USA) how for then about you know on VX-XXX, also, preference kind the of just doing

Jeffrey M. Leiden, M.D., Ph.D.

multiple have disease. little respect Jeff, pain diseases don't this neuropathics, in pain we Thanks it. one acute, to does for different, questions. said a compound really It's that one think as our as about inflammatory, et are is bit Alethia. Nor program Yes, cetera. before, pain, the we With

pain. you as have VX-XXX, both in which OA acute positive in have for and know, data We we

which oral follower pain. and is excited that both a in we're acute formulations IV for that also also VX-XXX, pain, are I'd very important, fast And remind have particularly you formulations We about.

move going Phase But the think new have demonstrate need to we And it's have in make if excited in this sure particularly can of X. right potential, more And multiple there's make indication actually and asked some to regulatory And have this data. data So those year what's about is potency is up, I in take opioids slow exploratory should way X horrible year. our pipeline, And would out game for Phase quickly that we next allow very right from going right because dose, going it's have think assertion us obviously, acute all be X and forward. if opioid middle you addictive much be do of the oral I this areas we're understand allow a chess because, wasn't Phase growing. is that. pain, we we're favorable environment. decide a and that like down, correctly we sort we're the best the best me, to move of to early going we actually epidemic. decision without that the a advantage and these formulation, in to that to studies sitting disease, have example, us in later some folks the to indications. know And the we the We to speed medicine, of to that agents, best would right about us really how the where we formulation do for the And we which medicine complicated want of to to doing

Ian F. Smith

we I'll Alethia, I'll M&A terms execution. question, your say is those versus licensing, take tactic in view that second which of just a as first

to is how whether three think Jeff approach. in everything earlier, own CF Vertex we and to our outside it we about so, in broader world the that said have look see as And strategies at complements

beyond maybe – there. those of product Secondly, the then small And And if our expanding product thirdly, possibility editing sorry, scientific footprint secondly to are have gene molecules what opportunities beyond now. and how a more of do the you company. in the To again, today. of licensing capital have just M&A, way into those incorporate come we earlier, tactic opportunities comment Jeff's that's available also, or

close in to we from you We of went million to $XXX of added first saw quarter, billion capital. cash. As $X.X billion $X.X

no debt. have We

pipeline. our We're of have don't and capability science we large do M&A, involved in believe to our stage, question utilization to get focused you more The we high on you're going also, that. more So add today is own think do share to ideas. count? No, earlier

other lots in in As a growth growth opportunities look terms stage And areas. in accumulation. revenue of earlier capital disease us our company, trying, driving of we'll at in have we of front and focus CF, is with what that's and

Michael Partridge

Operator, this we is one more Partridge, question. time for Michael have

Operator

question Karnauskas Citi. last Robyn And from come Certainly. with will our

is line open. Your

Robyn Karnauskas

Thanks in for the and my my not taking guys. this Hi, closet time. worry kids don't question, are

Unknown Speaker

We look forward call, to every Robyn. on that

Robyn Karnauskas

work kid bring ironic. there's though even your I know I that to saw days,

what asked will were the the see you So just is follow-up, about when we guess, first exactly, I data. where question a

find And you the het/min? that study, XX end you other when was, of your that do you When SYMDEKO, quarter could those great what the get can you'll thinking even assumptions placebo or like guidance. keep you like a on or four how as sense of you weeks could is above saying back patients for complete be I the that question in XX? had about you And You're reasonable had assess. what do be triple you say the placebo are complete? complete, Just a

reason going So kind this you I can how was the when you of raise it back just guidance robust why great, that's sales, you weeks didn't the strong, of quarter? seven thinking expectations see of the And a year? temper to

Jeffrey M. Leiden, M.D., Ph.D.

the closet that Robyn. lock kids I'll you echo didn't I'm in Thanks, again. your glad

to het/min just is question, you that anticipation remind our So firstly with study your expect on what VX-XXX, the we'll file first you which based the – we to lead to provide study, the data is upon. that

study endpoint at to And that safety you as so four-week a of the XX-week look design endpoint. when efficacy the

the it the to XX run want would the we of because weeks basis So that's through filing.

that We weeks. you'd So at run XX through least data. of collect anticipate that the we period would would

And to the obviously het/min provide in analyze disclosure the the would four data. and study on have We VX-XXX patients, using we'll which XX-week it. a complete

We got progresses. $X.X $X.XX your to our question reiterated billion. expectations, did on the we call how a see to billion As year tonight, first have quarter. strong the guidance We've regarding revenue to

we in growth that come those We markets the three reimbursed we see built do we growth adding products already are guidance. patients all The see that through terms will main from have SYMDEKO. main from in of be growth, anticipate, and will that into

guidance launching the reimbursed see our year in outside we're and and point in update guidance we'll how on And the reimbursed, our this of so potential markets growth time. at those drivers at we'll we're and goes time, of where could some be markets, you holding getting not that currently actually and

Robyn Karnauskas

you. Thank Great.

Operator

Mr. this today's question-and-answer I remarks. back Michael session. Thank to closing now would concludes to you and like call Partridge the for turn

Michael Partridge

questions the turning The you that team good for be evening. Relations Thank you, Thank tonight any will you, in for follow-up available night. everybody a call to Have Investor Chelsea. have. this

Operator

conclude may you Ladies conference. and disconnect. you all today's the gentlemen, program. does thank for This in participating And

Michael Partridge	Vertex Pharmaceuticals, Inc.
Jeffrey M. Leiden, M.D., Ph.D.	Vertex Pharmaceuticals, Inc.
Reshma Kewalramani	Vertex Pharmaceuticals, Inc.
Ian F. Smith	Vertex Pharmaceuticals, Inc.
Stuart A. Arbuckle	Vertex Pharmaceuticals, Inc.
Geoffrey C. Porges	Leerink Partners LLC
Geoffrey Meacham	Barclays Capital, Inc.
Michael J. Yee	Jefferies LLC
Ying Huang	Bank of America Merrill Lynch
Terence Flynn	Goldman Sachs & Co. LLC
Brian Abrahams	RBC Capital Markets LLC
Phil Nadeau	Cowen & Company
Matthew K. Harrison	Morgan Stanley & Co. LLC
Shawn Fu	JPMorgan Securities LLC
Dane Leone	BTIG LLC
Carter Gould	UBS Securities LLC
Alethia Young	Credit Suisse Securities (NYSE:USA) LLC
Robyn Karnauskas	Citigroup Global Markets, Inc.

Vertex Pharmaceuticals (VRTX) 26 Apr 182018 Q1 Earnings call transcript