Vertex Pharmaceuticals (VRTX) 31 Jan 202019 Q4 Earnings call transcript

Company Profile

Participants

Michael Partridge	Senior Vice President, Investor Relations
Jeffrey Leiden	Chairman, President and Chief Executive Officer
Reshma Kewalramani	Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer
Stuart Arbuckle	Executive Vice President and Chief Commercial Officer
Charles Wagner	Executive Vice President and Chief Financial Officer
Phil Nadeau	Cowen & Co.
Michael Yee	Jefferies
Salveen Richter	Goldman Sachs
Paul Matteis	Stifel Nicolaus
Alethia Young	Cantor Fitzgerald
Whitney Ijem	Guggenheim Securities
Robyn Karnauskas	SunTrust Robinson Humphrey Inc
Cory Kasimov	JPMorgan
Kostas Biliouris	Morgan Stanley
Liisa Bayko	JMP Securities
Brian Abrahams	RBC Capital Markets

Call transcript

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Michael Partridge

Good evening. Welcome to the Vertex Full-Year and Fourth Quarter 2019 Financial Results Conference Call. This is Michael Partridge, Senior Vice President of Investor Relations for Vertex.

Making prepared remarks on the call tonight, we have Dr. Jeff Leiden, Chairman and CEO; Dr. Officer; Financial Chief Officer. Medical Charlie Officer; Chief Chief Wagner, Kewalramani, Stuart Commercial Arbuckle, and Reshma call. We slides as this on that our recommend you website you access listen the webcast to a This replay website. conference being call recorded, will available on is be our and

statements our make call this marketed Securities These future could Exchange filings risks with forward-looking to in today’s statements, that in Commission. limitation Vertex’s uncertainties the assumptions. CF discussed based We pipeline detail subject those Actual including press the and on events performance, will financial and are and on our release regarding without differ current and management’s medicines, outcomes materially. and Vertex’s are

now to call over turn Dr. will the Leiden. I Jeff

Jeffrey Leiden

analysts JPMorgan Michael. weeks ago. two at the Thanks, everyone. investors evening, Good Conference We saw and many

moments just the for highlighting XXXX spend Vertex believe achievements future. we I’ll a and sets our what So apart few

truly a many And very pipeline exceeded bring in was and of or All Vertex. met CF well-positioned start XXXX the business our advance additional the to XXXX. we people in year our to diseases we medicines are remarkable at our a result, of goals parts the more to for broad set as year.

TRIKAFTA XX In was CF with of medicines approval TRIKAFTA cystic of efforts and a all new CF. to bring to XX% milestone all medicine our months is to treat most of remarkable the fibrosis, potential people to five to older with significant our for the October, the patients in that date, disease. this up U.S. ahead PDUFA holds date people in

is of patients early start. positive. clear XX and all across of launch a interest doctors As ages in feedback highly patients older patients very the TRIKAFTA you’ll to hear U.S. Stuart, both and the eligible in from is and from This off strong TRIKAFTA groups

modulators our U.S. to Australia others our reached England, France, thousands treatment new with in medicines Spain, key in XXXX. for patients of XXXX, a and begin allow CF agreements the will countries, we CFTR including of reimbursement number that many many Outside throughout

excellent broadening progress making We CF. advancing are pipeline beyond our and also

XXXX, enter outside of clinic in the multiple in we As now new we five medicines are diseases CF. with

continue the for of strategy areas. portfolio clinical of We each our advancing of medicines disease development into implement to a

alpha-X Phase the AAT underlying program, where lung. the liver include clinic, the deficiency, molecule VX-XXX, multiple at of the antitrypsin X correctors programs and development. small Key disease which have clinical our aimed both in we These recently addressing cause entered has include in

vivo CTXXXX, where and sickle CRISPR/CasX one-time announced these clinical ex which to suggests we that may functionally we a patients data Beta gene-editing diseases. cell cure treated thalassemia disease, therapy, able with for two be

islet our transform disease. cells autologous aimed of islet the cells where and first therapy correct small an disease, Phase where potentially progression this function will development we and in second, diabetes, device a alone, of are a of move cell halting developing XXXX. at a and And FSGS, into X treatment type to molecule the with the with X transplantation combination

but also Importantly, therapies. including multiple as approaches in Vertex genetic past, new where excelled modalities, pipeline molecules, these has span the such now and cell small programs

our we’ve modalities, for leading the with expertise and deliver manufacture unique For these acquired new that teams transformational best or companies and who diseases have to fit partnered therapies strategy.

Semma In X years, with for prior in for and gene the our leading DMD and Exonics, we in leader the business including development, approach four therapy transactions in more editing type DMX. than diabetes cell XXXX completed the a of acquisitions

biomarkers by improve clinical of targets almost serious focused of In that our success. and of validated focusing in the a of will and decade was the execution combination discovering predictive strategy diseases against for areas developing transformative XXXX summary, medicines specialty probability on

The growth. us and position as playing in positioned out and is been success company beyond. or has stronger we for CF never had planned will strategy and continued exactly for long-term Our better short-term future

in will about me the to who over year more detail Reshma, ahead. call turn now the Let talk

Reshma Kewalramani

Thanks, Jeff.

to Our XXXX and progress has positioned us years for growth in for come. continued XXXX many

bringing advancing on to medicines our innovation. CF focused building are in financial and strength more and people, to pipeline investment internal our support We external continued

the In for six expect XX children for the to XXXX, submit and in years triple Europe approval ages gain older combination we XX. to approval in patients TRIKAFTA to and for in U.S.

and are are of Beyond proof-of-concept and our advances pipeline clinical a preclinical period clinical early through molecules multiple in CF, multiple now data potentially medicines. advancing we readouts late and entering with transformative development

study the recently a Z in data X VX-XXX initiated and in patients corrector, molecule mutation copies With Phase with study two the expect of from we our small XXXX. AAT the proof-of-concept program, of

completed we in protein open levels X in late in In the expect an to XXXX X VX-XXX in proof-of-concept of evaluate and Phase a study with to FSGS, APOLX-mediated Phase label study the XXXX VX-XXX. initiate reduction urine

Phase multiple is In our mid optimal or pain, proof-of-concept focus to from having the inhibition find to late-stage X with molecules advance NaVX.X into established now and VX-XXX molecule studies, in studies. data

into We development be of and into the are a in advance additional of advancing development first-half portfolio XXXX. an continuing molecule clinical to medicines will X Phase

Phase PK We have display VX-XXX, not development X because discontinued optimal and tolerability did profile. of it

business of our progress and Beyond in we’ve through molecule research development our in with strategy, therapies, line activities. small our and primarily genetic progressing portfolio programs, cell building significant made a

We data our and Both for this enroll data are provide disease. to by continue for studies cell our additional clinical CTXXXX for treatment CRISPR/CasX ex vivo and expect XXXX. we thalassemia to recent program in encouraged sickle highly beta gene-editing

us program October type team of on function. of fully mature Semma preserving novel XXXX. Therapeutics X protect The cell to I’d in This comes immune scale a approach health scientists the biology islet have of cracked also at system, Semma like production highlight up of device from therapy and our acquisition cell to from and both and developed cells our these the has cells the for to implantable while diabetes.

program clinical this into early progress to development in goal or XXXX ambitious an set have late We XXXX.

XXXX. and in forward updating CF diseases outstanding the on look to we’ve coming years. progress And progress and summary, In you months our I over made multiple other in

over turn Stuart. now to I’ll the call

Stuart Arbuckle

with commercial to you evening this XXXX. performance pleased review Thanks, Reshma. strong our for I’m

Our were revenues of from This Germany, launch full-year driven XX%. billion, $X early globally U.S. in the of expansions medicines of billion approval SYMDEKO represents launch in label revenues which primarily full-year growth growth by the year-over-year up the for and impact was the XXXX, XXXX total $X and TRIKAFTA the CF in our U.S. and in CF

to strong of launch off is The a start. TRIKAFTA very

making fourth $X.XX billion, revenues already our approximately including $XXX revenues CF Our medicine. total product of quarter million, TRIKAFTA top-selling were TRIKAFTA

medicines expected, that $XXX are eligible approximately which fourth for note would largest the launch. of launch-related include, of million. our population and patient XX,XXX TRIKAFTA patients of Approximately our I quarter one stocking approval the time eligible U.S., in as the at CF for revenues represents

For of X,XXX the these their treat CF. to underlying people, of is a had the this cause medicine time first they have

interest from groups seeing patients, including strong from new as are other well We CFTR all eligible modulators. initiations, as transitioning of our patients

we and job The multidisciplinary market support, an approval. were teams centers to government the a field have a we to teams the supply, ready remarkable Together, responding CF And for CF start the in produce have launch, doing for centers the job teams these in to patient launch. commercial track factors since high-patient and on still medicines. the to other early for marketing phenomenal demand. and broad with seen are U.S., TRIKAFTA and Our combined obtain access, have their these reimbursement payers. commercial while what our early have done strong And similar October, been to

certain in key in we XXXX U.S., agreements patients multiple medicines Vertex of first thousands many countries, treatment the reimbursement with reached time. the initiate for Outside enable will which to

While initiating international based the treatment main driver expect with U.S. TRIKAFTA more will patients Vertex’s be XXXX, revenue also growth our medicines the we increase an on of in revenues in outside

In globe. pleased patients many bringing that medicines our summary, we I’m the to more around are

over And to turn I’ll Charlie. with now that, call the

Charles Wagner

this Stuart. financial evening I additional remarks also discuss guidance. our financial will XXXX Thanks, I will regarding our and XXXX results provide

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

billion, total approximately product XX% mentioned, increase As revenues fourth compared CF had we quarter XXXX. of Stuart to $X.XX a

quarter expenses quarter spending $XXX fourth SG&A $XXX in including to increase income million, resulted XXXX $XXX in of XX% R&D a revenues quarter and the the of to the growth fourth million, compared XXXX. XXXX significant disciplined fourth compared operating were Semma, million operating of in of expenses Our the quarter combined in Exonics and and The fourth

quarter the XXXX $XXX for to for income the of fourth compared million of was quarter Net $XXX million, XXXX. fourth

results reflect a income spending, story financial full-year and Our resulting growth strong exceptional revenue in disciplined growth. similar operating of

for revenues were a increase XXXX. CF XXXX XX% Our over billion, $X total full-year

compared XXXX expenses R&D SG&A were to $X.XX for Our and $X.XX combined XXXX. billion, billion

Our billion of for full-year $X.XX more $X.XX XXXX, for compared was income a increase year-over-year XXXX, XX%. billion operating than to

have we result patients deliberately in to flow external cash and treating profitability CF future create our increase a internal both innovation globally, more medicines. of as reinvested As and

billion significant $X.X new in approximately and approximately innovation acquisitions the and we $X.X to the year BD invested end activity, with $X.X compared we marketable ended external billion collaborations. cash billion through in the securities, at of XXXX, XXXX. Even In with

provide fuel beyond, investments increases XXXX to for look and flexibility ahead we to continued flow cash we expect more As in to growth. our additional long-term

revenue total expenses and XXXX positioned of agreements strong product as providing the and have the reimbursement of growth revenues, XXXX. to financial non-GAAP The guidance. uptake SG&A U.S. guidance well tax recent for as for our in TRIKAFTA effective on strong continued for XXXX CF Today, and combined Now we’re Vertex R&D rate. anticipated completion outside

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

dynamics I are our would XXXX guidance. note a few reflected in that

TRIKAFTA, that do XXXX. $XXX approximately inventory of the to As strong in launch-related part that mentioned, build repeat an quarter launch expect the we million not expected saw of of in Stuart we fourth

dynamics XXXX with revenues. persistence our TRIKAFTA guidance. will factored other our CFTR experience Also, CF our medicines with and compliance as of move we is through modulators, affect all into And therefore, our as

we XX% Lastly, XX% net to XXXX. for gross of adjustments to expect

the than Focusing first higher in our of reflects XXXX, well to to net each be This fourth QX adjustments gross modestly generally XXXX higher the quarter. revenues in previous of year revenues. inventory on as to we the as impact the build, that quarter relative QX quarter expect are

expansion We SG&A into driven billion $X.XX R&D and additional billion. is and diseases. pipeline primarily expect R&D by the to The launch of $X to our of expenses compared XXXX XXXX TRIKAFTA of increase globally the combined

X diabetes therapies, and type DMD. includes R&D Our our and and expense increased investment genetic to growth programs including advance cell

to vast to rate with Now full-year use highest our quarter. losses. we tax where The XX% occurring our fully our fluctuate non-cash may expect XX%. non-GAAP rate will until to fourth in rate be tax of The we the operating guidance, expense tax net majority tax the quarter-to-quarter be provision

to continue that And margins, pipeline. with and expanding a continued continued revenue growth and long-term Vertex discipline, cash flow an earnings As is to expect CF based on potential Jeff spending increase. noted, operating has growth unique we in

concluding to few back Jeff Now for a comments.

Jeffrey Leiden

Thanks, Charlie.

As hope this for call you quarterly indulge minutes couple I CEO, last for me a comments. some is final as my of

for honor incredible First, teams. it has sport. have Vertex years. tremendous the several say been and happened development successes and always to a team is eight our I ultimate without the past drug the of pleasure would discovery None lead

thank senior all team whom in most in work. First, I of XX my team, journey. years want industry grateful the to for been seen me entire the Vertex leadership have I’m team, and for the stronger our I’ve so of proud never with and their entire including a

it’s our and The to for of position success strategy transformational patients grow innovation outstanding to the what recent medicines is achievements, future. to for and employees Vertex the long-term us serial leaders commitment the business deliver for us of driving and will senior differentiate to and our force also the execute

to losing and advice. constant CF first and CEO trying encouragement we when investors medicines. Board their a I Even to became I the still develop year, of several transformative and for our our Second, hundred thank dollars million support, want were Directors first we’re

community, who And Executive all want work transformative for toward amazing bring for their are patients all diseases waiting. cure patients, their you the encouragement importantly, with journey persistent I with and to thank and caregivers finally, Chairman enthusiastic as to participation more patients their courage, with this continuing a CF to to look of and devastating medicines entire this to most in families forward I disease. serious

will I questions. open now to the line

Operator

Instructions] you. Co. first & question comes Thank [Operator from Nadeau Our Phil with Cowen

open. now is line Your

Phil Nadeau

my team the for Jeff, let Thanks of It’s the be achieved question something of to to my question. evening. you in have quite CEO the the me first you that really your just congratulate in Then, Vertex all eight years Good been and numbers. tenure. and is watch. taking terms

they maybe option transitioning had among mentioned the new of holdouts markets from? $XXX in no patients, being of Stuart, First, – sense you prior of SYMDEKO, you in three therapy, who really new Where and do to buckets give dropouts in user added other TRIKAFTA in populations. three to from the demand? those and us million initiations end dynamics patients the Could some ORKAMBI those the come

on And two then million the is run current seems to like second, of weeks the of three it $XXX rate. inventory really just given inventory, inventory, just

to there that a to So build, that not you’re is it’s you average be inventory that on expected of kind clear during given of already your the channel? come rate inventory for comments, out just QX. the subsequent had It’s channel no run Thanks. in inventory

Jeffrey Leiden

TRIKAFTA I’ll can take talk uptake, inventory. Phil, Great. Charlie the and to Yes. then question the on the

number There you strong very as know, of patient So, are launch. a eligible we populations. had a

and seen patient and groups launch, a to XXXX. strong As from we you have all from tell those of level high continue interest into of we patient in expect that uptake groups had all might we’ve the

handle to Charlie. And that so throw I’ll to over question, the inventory

Charles Wagner

is a but to build bounces Yes. the to a it’s commented was inventory think inventory say, magnitude what And day – that’s Phil, that I’d less a on to level. the little – bit the Inventory mentioned. won’t you fair do – even day therefore, build nor two we drawn quarter I say first of the about is of significantly. repeat or all, than fourth probably a couple a that’s down the that, right But your quarter, – of expect any it question, to expected. around in build that, the given that we get

Phil Nadeau

helpful. for Thanks and my very congrats the performance. on questions That’s

Operator

you. Thank

Our comes with Jefferies. question Yee next Michael from

open. now is line Your

Michael Yee

what position. Hi, a now leaving just to just where there great characterize of just happen And, of guess, and XXXX, result. can congrats just swapping number thanks I the could you think wanted you that we comments so you parts swapping? do think, question maybe for and, again, percent about But was some And great I are swapping buckets on Stu, ask, you think with Jeff, how on could moving dynamic for the were. how and the obviously, a you them that? made about throughout

next. broad there’ll a mean, know just on about maybe question I the on I Reshma. for XXXX. AAT get much. for and a very that of a It’s we It I of a fast speed Can focus a you’ve that so seems you’re you data? there then study? talk how short lot Thank just be And What study. you data. like can guidance timeline know XXXX doing that broad given

Jeffrey Leiden

the on TRIKAFTA. of Yes. Mike, uptake

the the the TRIKAFTA. given are TRIKAFTA majority Obviously, vast process includes terms I going patients of But TRIKAFTA. across for to being said, where tell. TRIKAFTA profile, who to long are of to groups are that to the the overlapping eligible one how and patients modulators. are who destination, in transition labels, in As expect superiority we’ve hard with interest of going to CFTR launch. those our existing the majority the that take, we’re Over switch Exactly those of we it’s early all of will time, seen vast currently have of we patient treated

Reshma Kewalramani

With deficiency very regard Hi, XXXX. Phase program, one I is the that’s the the alpha-X furthest referring the toward think, ahead. that’s dose-ranging to one antitrypsin started tail-end to you’re It’s one proof-of-concept X VX-XXX, we of the Mike.

days. actually, it’s very really early So

XXXX, I’m study. to around that going it’s color just too expecting program way will results We’re with give have but getting that the you from early in that. more we just

Michael Yee

Thank you. Okay.

Operator

you. Thank

comes Our Richter Sachs. Salveen next Goldman question from with

now is open. Your line

Salveen Richter

that and, question at all congrats achieved for Jeff, Thanks you’ve taking my on Vertex.

will just cadence then XXXX? TRIKAFTA you on of the form XXXX during launch could how firstly, uptake So comment the

pipeline, diabetes, requirements for the look And thoughts not. for like biopsy the here? for at X regarding any pathway proof-of-concept look does antitrypsin the regulatory new we a then with around secondly, And then alpha-X the secondly, need here as liver what study or Type

Stuart Arbuckle

Yes. Salveen, take the on it’s I’ll Stuart. XXXX. and on impact question the XXXX

to off patients. said start. announced there results we’ve many the So obviously, are very, given very strong today, that, a Having

We get TRIKAFTA. to need to continue onto

of expecting to that’s billion terms billion. patients our XXXX are built $X.X guidance we $X.X and of continued into So growth in through adding

said, and we’ve or revenues about impact one of As into as with the shape is revenues. you impact think have of take impact It an and how compliance other does those seen factor Charlie cadence other our although will that persistence the to the modulators. account, CFTR as

I think Our expectation strong the will X, that on and particularly type and with will think is levels be compliance Reshma And then of AAT persistence I TRIKAFTA, take the high those. profile. clinical given the

Reshma Kewalramani

Sure.

So first, let second. AATD and I’ll the me tackle take diabetes question

through diabetes that’s acquired program acquisition. So Semma we with to program, the cell the the therapy that regard

the mean, more program. sickle thal I akin proof-of-concept program I you beta and molecule about something to small our be think cell a So, to can imagine versus

mean a the you AXc. I by endpoints what will right into for good going And go a glucose the the is, that we’re the fairly And into hemoglobin side watching. clinic to hypoglycemic of healthy watching alone straightforward something patients. safety with device, are program be be be expect think kind with whether what we’re the step. are cell But for. you I I It’s going on or able to to not could that the go we we levels, of sense that ones, gives kind cell going think, episodes have to Clearly, volunteer

and again, be a anticipate The going and thing I other reasonable think I duration. that that cell, similar size are mention to the is, to to you reasonable thal studies very beta sickle can, proof-of-concept a

regulatory have With regard to not further had interactions. the AATD we program,

their with be work before, I this looking I’ve be. was with ongoing FDA levels. key was points the the for as AAT What on XXXX October that so, conference. from endpoint for the would we that program that’s what that would on regulatory functional indicated each commented And enabling anticipate sponsor impressed would is the approach And I depending they agency serum

regulatory to will that’s look think we point remind to data the receive their as that, get the need just just I you forward beyond interactions. approval that on through And companies based augmentation at. levels, AAT we the

say the And treat approach is lung approach those small opportunity and thing different the the that our is, holds molecule corrector manifestations. and I’ll both last obviously to the very there than liver out

And what what going would to endpoints through as so, manifestations be those and talking we’re those look like obviously, well. liver

Salveen Richter

Great. Thank you.

Operator

you. Thank

Matteis question comes Stifel. with next Our from Paul

open. now is line Your

Paul Matteis

so you for questions. Thank the Great. taking much

on question looks on take pretty you it you’re to quick full-year with one XQ, your number Just at outlined. like already just close guidance. we inventories considering when comments Even annualizing the

we so, And just being the go there that into to conservative, or you a Are wanted understanding could slow? of a is better reason that uptake get just your dynamics guidance.

success And the potential for then, quickly just program? a ways bar true just think the on in commercial we benefit? much. was insulin And program that I so clinical you second independence, for realistic do Thanks about therapy about are other there like should how is diabetes, think goal you or cell curious feel do a

Charles Wagner

is on question is characterize take conservative. the this And Paul, would Charlie. guidance. first I I’ll not guidance the Hey,

know it’s fourth agreements the well I launch TRIKAFTA the appropriate, given about quarter. what signed think the in we reimbursement so as far, we that as

that. the in guidance, we meaningful implies the over to inventory But QX build, obviously $XXX Again, you will and persistence the it’s revenues into again and on of the the touch remarks, come course run if the tempting growth – of to that. out just then inventory the million XXXX. you’re the back over impact at compliance want look touched billion is back on significant at topic, to And and for the billion take extrapolate $X.XX from of rate into $X.XX as

think number billion guidance So sets candidly, we the $X.X think the you factor us for once the XX% another year-over-year, very things those absolutely $X.X And about a billion is increase rate, at so when up year. growth you strong in, midpoint, appropriate. it that

Reshma Kewalramani

the and take question Paul, cell X diabetes. therapies I’ll program type on the

you that’s whether approach closed-loop systems that’s think there, this is realize episodes. think about insulin about if think you it AXc, good, particularly side, what X at or whether speak other about to have disease, glucose over you diabetes particularly type and about is to available neither million current – X people the these in the the glucose really or to hypoglycemic or just So think injection you and who the control look the you safe you nor anything is hemoglobin on

the people you that transplants, Then, deficiencies very cadaveric shows have terms hypoglycemia. cell transplants, glucose they look cadaveric if transplants, do well of and control actually that islet at have in with who don’t

there of are for course, immunosuppression. problem is issue the just islets there then cadavers and there Now, there, the transplant – not aren’t enough enough of is

way hypoglycemic islet really the and real cadaveric this why and in to cells. So have with a control the only is produce, transplant glucose approach holds Group the we’re but Melton so excited episodes. up like come potential beauty that without this scale Doug very for excellent Semma not And the the about these to

the real what goal that’s So here is.

Paul Matteis

Great. Thanks for the it. appreciate color. I

Operator

Thank you.

Our Fitzgerald. Young question with from next Cantor comes Alethia

is line now open. Your

Alethia Young

in taking you Hey, Thanks for be guys. will incredible CEO the Jeff, on question. And missed quarter. my run. Congrats the seat, certainly,

might I CEO call that think we drop.

questions my much field, in just be guys talked seen it I when or guess, better? kind you’re the have bottlenecks about case two been are, have to the has biggest that been demand, what potentially about you and thinking you so little know the maybe a of So surprises? I

little and my more do XXXX? takes second puts in be what that are that is, think are you kind think and dynamic as early, KALYDECO, a trending a then a might of like question but the compliance the persistence about you And it Thanks. or

Jeffrey Leiden

very thanks Alethia, much the question. for Yes.

raised with described going potential for eligible Prior to they there you’ve a CF couple constraints. patients centers, felt given have them capacity XX,XXX about as bottlenecks about. right. us One the They of concerns to were that we might You’re launch, the TRIKAFTA. that be concerned it was was were

the on just patients working been have of some the jumped, an at amazing they centers, job job responding get to the say, a general, patient teams to spectacular have done high-level medicine. demand. initiated in there And to bottlenecks have have some multidisciplinary I certainly in whilst

product other a payers. get teams to very both our launch with new we as with clearly, significant have access. commercial been are from whether a the able always quarter working deliver the again, potential we payers. in results is job we And The have going and support great And bottleneck without government to fourth have wouldn’t done

of – bottlenecks we’ve turned actually those that have how pleasantly XXXX. both expect in those with out been and surprised So well I have continue QX to in

really, is In to it’s range expect really be medicines. is in terms given it similar going say do But too be for of we’d like early exactly tell and and it TRIKAFTA for what this the has high to in of that too which the profile, of to the compliance We world. for those to been to aspects with real in world going expect just to very persistence, other all certainly the real expect what it’s be to exactly the early it CFTR both medicine do look be of like. it you We our high. know modulators,

Alethia Young

Great. Thanks.

Operator

you. Thank

with Whitney from comes Guggenheim. question next Our Ijem

Your now open. is line

Whitney Ijem

guys. Hey, Thanks very question. on X I for the follow-up wanted to diabetes. type much

this wasn’t clear you on point? And at devices can clinic you’d color wondering forward cells us or exact what or I’m any or the moving like technology be it So not sounded the encapsulation. give whether into with encapsulation naked if the the more it

Reshma Kewalramani

reasons. few right. It’s regard to Sure. much Thanks talk question, late one for of you’re a specific my But your to question. preclinical favorite with development for so really about progress the

shots on speak. so here, We to goal two have

And, couple cells the different pathway potential example, is involved of One, a for alone. attractive areas. that development in

would diabetes. an they patients renal One transplant are of who are example, anyway be renal type have their on because transplant and of recipients as immunosuppressive them X who therapy

alone So pathway. nice cell cell approach naked or be the approach could a there the

and encapsulation the work is scale they’ve and a of really a up, but has only maturation others device. solve. device. cells be problem The to to Group nice particular, device the this in tried with done job it’s amazing and industrial the and done The have has the the not past The a Semma tough regard with development to

It it oxygenation for material. the state. to needs The and And is also seen the flow, to free large has cells to in device the think the succeed animals tells reasons. device to we and It with opportunity is a – that different geometry. keep have different and data I here insulin their that in And really with has fibrosis. but It in do glucose to to few to sufficient that do the that allow date, to the the with in there cells has that’s do including ensuring us get is has so.

Whitney Ijem

Got it. a quick just follow-up. And

first, program So sort be abandoned into encapsulation and the the nearest, cell it forward we first? moving which or will is you the clinic still of naked the approach, go will Thanks.

Reshma Kewalramani

Yes.

can of on a it two terms think think two as it’s in it you shots can which of programs. goal having You But and just matter of one of first. goes

Operator

you. Thank

Michael Partridge

please? question, next Operator,

Operator

Robyn from SunTrust Robinson Humphrey. Karnauskas comes question next with Our

now open. is line Your

Robyn Karnauskas

Jeff and for question great. in, put the Thank been you Hi. hard for you taking my work thanks all it’s

two for questions. One Charlie. So

least you’re not studies for years. First, looks like that the It you at be accumulating. may have and maybe about tight they’re as few the expensive, cash very next small do that’s they to

you continued then, So And the running VX-XXX? Reshma, cash lazy pain, about are how for without risk the thus for one thinking are balance maximizing you versus of looking original sheet? program. you Thanks. having just What

Charles Wagner

Thanks Robyn. question. for Sure, the

well we flexibility. us running cash, business very out, now gives are and right As is you point model which generating the

with innovation also in use to access XXXX some both enabling internal perfect saw a our innovation. to terms programs We business, continue in technology year with you got us active feel some $X.X great in have fit to the billion a of that and very that is on reinvest of number external deals and that are Again, research a the cash strategy. us our of best

disciplined, have continue flow we’ll forward, that to the priority certain by active the be that not will you but we cash in will I’m be. to strategy of business where in research additional To deals you will see and Everything stay the needs volume to number that’s in of a corporate to active be us going going XXXX. cash or the to So governed XXXX, be certain committing continue development. – flow. a say extent we’re continue strategy

Reshma Kewalramani

Reshma. Robyn, the take question this I’ll pain. about is

think the chemistry. just program confident pain we’ve three biology. first cracked like in that the because is neuropathic pain X feel would CF that studies, the and about acute Vertex in with all VX-XXX programs. I Robyn, So, approach we is, I on the The pain And where in Phase of we right biology, saw pour and are pain the here results of and And crack frankly, speak then our in osteoporosis. saying

So we’re what efficacy, pour X, call course, And safety But molecule about talking once and disease that for or doing we’re of pain about a molecule, state, particularly the is which chemistry. perfect something now be can this this is ideal me part is day, looking the is let twice finding, on we’re table in PK, the condition is with what in that stakes. instance. this really dosed a given really

ensure can without We medicine need with taken food. that be food or to this

pain, is immediately to about able be going to post-surgery really food with being take If it talking you’re acute our important.

about and thinking We’re DDIs COGS. also

or there. search conditions, chemistry we’re pouring as I this and as the guess, I to it three in about should for the stage this of at groups our would condition, for on ideal as actually you describe distinct is molecule pain really, so the think And it describe we I

Robyn Karnauskas

All right. you. Thank

Operator

Thank you.

with from JPMorgan. question comes next Our Cory Kasimov

is Your now line open.

Cory Kasimov

a question. made guess, Congrats the weeks little great of regarding AAT. have could is that taking Only this for year. on I Conference a have you a more exciting this for couple question, Thanks guys. afternoon, you first ago, quarter. good pre-announced Hey, you I Investor

and you diagnosis clinical development? of you while there efforts do to broader-based the ramps, to As kind facilitate the with process what education in rate can are help work any seeing

Reshma Kewalramani

this let’s take Yes. Cory, in parts. two

mind, then such. Reshma. few opportunity and would little market you over If a to make and I’ll this turn is us about a more comments bit going it Stuart to tell the to I’m

there and where with Cory, isn’t community, diagnosis. newborn absolutely and is to and Unlike we clinical start XXX% our the to started what there do, true. isn’t what this really CF, to as which alluding start you’re trials is a already screening realize engage you disease we’ve

is is the for And frequently. XXX done not while antigenic cleared there level, diagnosis marked a that CE assay

with working are the We community.

Alpha-X Foundation. amount engaged of an are see education more. group I that We and providing see opportunity with good do And I to real do the a even

point. comment vantage Let Stuart to his me ask from

Stuart Arbuckle

with Yes. about estimated an of today, but what U.S. to Cory, this underestimate, the that Almost your with the current a as ZZ Only point. so be know standard a in that patients and that’s of fraction just take the XXX,XXX starting market point, definitely, are currently about treated those actually of fraction the which terms diagnosed, therapy. is people we the are care, those augmentation diagnosed a are IV EU. in actively the let’s to only and of genotype

the bring are replace which able IV some the clearly on impact disease, multiple we is we to lung cause potentially product, of liver the augmentation has a treats is to market both of small molecule, the is underlying there if therapies. here. to the So and which One the think an and opportunities oral

to the diagnosis rate. to a think increase Another be patients also diagnosed. there are is significant increase we treatment those opportunity the already who do But in would rate opportunity

is not you the solution, for so It’s should don’t treatment diagnosed that patients for I currently think, have diagnosis go But problem. the as be where guidelines blood difficult within simple with people The done COPD. do. a looking often that, don’t to case, It’s test. a included

anticipate so able bring And better solution be an do that we to if diagnosis increase in those to market. are we could a rates, the

Cory Kasimov

the taking questions. appreciate I guys. Thanks Great.

Operator

you. Thank

from question Morgan Stanley. Matthew next with Harrison Our comes

Your line is now open.

Kostas Biliouris

Hello, questions me. Congratulations this for Kostas from Two on the on Matthew. is quarter.

can the how to dynamics is, of one please? think in XXXX, sense give for about European us first revenues you some The

Jeffrey Leiden

were to agreements, major reimbursement European that. in we finalize as of work agreements take having reimbursement towards secured And number of have to a even of be XXXX. the back-end anticipated, patients XXXX, can the much I’ll able you We not because contribution before of countries agreements a administrative those through did in Yes. we those see initiated. process

billion grow as to modulators more are our to Charlie the billion revenues expecting the are CFTR call. access in our guidance talked that earlier patients European that’s to able and in incorporated XXXX $X.X on $X.X We to

Kostas Biliouris

And question disease is APOLX-mediated on kidney program. you. second Thank my

suffice? you would whether market. I You urea you the as endpoints planning clinical was a to have protein use or key mentioned these are wondering efficacy more need that

Reshma Kewalramani

This take Thanks, Sure. one. Kostas. that is I’ll Reshma.

and the going Phase APOLX-mediated this VX-XXX So This into for is program for those now one, – familiar as who is in may actually. is clinic this not patients this be the into with X FSGS.

measure So, kidney might to Kostas, the a endpoint in renal saying that’s basically the a simple for That’s be is as along homogeneous a discussed be enabling you that, there mouthful. know, community, and lot past many regulatory thought and the support what what disease, what the urine. here may pro-generic thing of would to with idea is agencies, protein regulatory But fairly for years appropriate the a I’m do. have is

disease that’s for in that this the in one homogeneous is to And right protein disease evaluate. a been protein when leaking a the has that discussed most believe have and people extensively urine measure the you community

and Phase So the to going that’s X we’re getting that’s evaluating under is what now this be study that in way. study

Kostas Biliouris

much very you and congratulations, again. Thank

Operator

you. Thank

comes question from next JMP with Liisa Our Securities. Bayko

line Your open. is now

Liisa Bayko

on Hi. to a congratulations some the ask on the think that look team had And to countries. Thanks. you available modulators of ramping also could roll this past? been therapy has more into get different they sort the like, in the timing transition. just meaning, new wish specifics given about CFTR of countries, little to what of haven’t out. Wanted a sort access European that’s the you Wanted during and Can on to

Jeffrey Leiden

Yes.

agreements Europe. are of reimbursement mentioned the aspects now And as timing I Spain, is two really France, question, medicines, there’s that, in of different countries, current UK, think, et existing that approved major that in in CFTR countries the cetera, to earlier, of I we some expect for clearly, the One we which to Liisa. begin have our uptake modulators.

In out that regulatory with how terms we submission have of for the combination, in might that clearly, the authorities. play triple

this an the expectation trigger to is of reimbursement beginning regulatory in of really for discussions. the you know, Our as is year approval the and approval QX

guidance our regimen within revenues so within And for XXXX, that guidance. there is combination triple minimal included

Operator

Okay.

Michael Partridge

more Operator, we one for time question. have

Operator

you. Thank

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

Your is now open. line

Brian Abrahams

Hi TRIKAFTA efficacy been quarter been safety? trial different all there? your aligning and What’s and experience there, that’s feedback or thanks Jeff, what on clinical and to your unexpected real-world on degree, my experiences, with anything with To congratulations taking congratulations, the accomplishments. for the on questions

Thanks. your how as timeline And market well collect then secondarily, full and in of things you remind plan be you longer-term exacerbations, to that as the penetration important European updating can for us will think for outcomes the TRIKAFTA data U.S., like access? with that

Jeffrey Leiden

experience, So X physicians to I say, inspiring. patients impact really the similar lives has is and of in real-world efficacy it’s would the And positive, had – terms the in been saw level The say having I of been has say experience positive. universally when and I what almost program, Phase Brian. we very the feedback from on their their we’ve of

very, safety over seen real-world demonstrated play know, those profile. X Obviously, that something, But you us very studies benefit the saw anything is which certainly, been we to what we out that in Phase surprised programs. as needs risk a haven’t different in has the time. And has strong from

going of Reshma. what terms collecting, In data outcomes I’ll hand data that to over to be we’re and

Reshma Kewalramani

Sure.

out FMS a in – patients, exacerbations we patients program was Phase on large the went with XXX that really to X weeks. the for and already of that pulmonary know XX reported you that about reduction program was it the Brian, So, We XX%.

to collect We are data. continuing

into FS that FMS study out XX both through over the open So study the an goes label in weeks. extension, and patients rolled

collecting additional And doing, globe the have we’ve here registries, from U.S., studies various as to in CFTR addition our not well. as we that other done only we’re the but in with data around modulators that,

but we’re to have will look be and around label not globe. collecting to forward the open program, we extension, that also trials data collecting from So the only more the clinical registry data

Brian Abrahams

much. very Thanks

Operator

you. Thank

Michael Partridge

Okay. of We for listening call. here, know that On all everybody kind call Thanks there are thanks the tonight. everyone for earnings tonight’s other to words. also behalf

if to So to additional the the to IR same time, in you we’ll get the questions. let team happy them. you office at and have talk And is you

Operator

Ladies you and concludes call. Thank gentlemen, for participating. conference this today’s

now disconnect. may You

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