VRTX - Earnings call transcript Q2 2020 | Vertex Pharmaceuticals Incorporated

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Michael Partridge

Good evening. Welcome to the Vertex Second Quarter 2020 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. Reshma Kewalramani, Vertex's CEO and President, Stuart Arbuckle, Chief Commercial Officer and Charlie Wagner, Chief Financial Officer. access to slides this you as our that listen you call. recommend the on webcast website We This conference call will be is being recorded, on website. and our available a replay

based financial subject on We current limitation, would forward-looking today's to those including, events note CF financial and management's are press call could are filings I differ we that marketed discussed this the this and Exchange performance to our in Actual the medicines, and regarding outcomes this non-GAAP. and the that call will on in assumptions. of will review Vertex's Reshma guidance detail risks statements materially. also results Vertex's over now Dr. our without Kewalramani. that on with evening make call release Commission. uncertainties and will turn statements, I and future are pipeline all the Securities These

Reshma Kewalramani

triple potential of Vertex to of to especially in able on notably, patients. proof-of-concept acknowledge I'm team want the by employees XXXX all recent of progress our launch our that a continues opinion strong performance goals therapy our the year COVID-XX the the Thanks, our for be by continued data this remarkable And measured stage to triple most The the combination been U.S. regimen continue the programs, landmark of as challenges reimbursement aspects in curative and beta-thalassemia CTXXXX deliver of in accomplish in patients has CHMP all pipeline the late the of agreement preclinical to for our cell and the access to of this completion resilience and to the advancement commitment and of Michael. of despite across to positive for all TRIKAFTA England. for both expand Vertex presented Europe, pandemic that for proud the disease. business, a sickle underscores combination clinical

our opinion would of therapy patients positions come. CF be if regimen, reflecting to provides uptake medicines. coming in to profile KAFTRIO. have majority our Europe cause Outside treat earlier-than-expected us vast the cause we medicine many benefit eligible XX,XXX with first June, TRIKAFTA, which, addressing which to combination speak significant by triple Turning believe of known disease. foundation that medicine the of TRIKAFTA fundamental for U.S. the underlying to treatment CF Europe will the first the in in for the and approved, the the CHMP approval the the reimbursement months, Today, we begun rate underlying this as at in appreciation to years obtained will have for This for The medicine, which received new broad therapeutic in of disease. speed of a we of U.S., this with the the their an which positive provide patients of up be the and

and potential add a significant that KAFTRIO on this Following III these positive year include month showed patients mutation or label in based FXXX X of mutation patients. reported Europe, approval with plan to expansion earlier this the residual a later to seek the KAFTRIO anticipated additional the del data we function gating benefit in Phase

recently year-to-date and landmark as England fibrosis. once treating uncommon were of expand KAFTRIO, the regulatory our the allowing to in This value of belief of these in of our include those and other Charlie Denmark driven detail and medicine we in our agreements will TRIKAFTA, reflects a this in on discuss European in begin agreement XXXX to NHS that Stuart pleased with in moment. are which more our and a achievement cystic occurs. as by performance, our include in reimbursement and England Ireland also benefit such are revising again KAFTRIO We shared advance is medicines. builds revenue able thousands approval to medicines innovative we truly receiving approval primarily Commission on agreements, that reimbursement guidance, Reaching our other patients patients upward Based is well underlying a cause as for

Beyond of additional retain worldwide, to to goal regulatory treat CF line approvals the including of clear approvals, all of XX% we triple younger our and reaching agreements sight patients XXXX, obtain bringing we reimbursement to patients long-standing combination globally. as

differentiated able we've also been we studies now in the broad initiate genetic clinical Despite where of have and of able CF, new pipeline to dosing challenges all beyond and we during molecule, for serious of pandemic, programs period. our are our COVID-XX therapies a this a cell diseases. small been and to to reinitiate enrollment of and Turning advancing the trials, clinical range

have have resumed diseases, sickle A with response additional which cell clinical of and meeting. VX-XXX means treatment. study engrafted, an me potential meeting even II as of treat curative in but in ability all Let CTXXXX all in not are have and momentum sites these we additional highlighted have enroll across yet gain last our important resume to weeks. we editing been that their the This CTXXXX, is enrollment we continues dosed both as in and the of been reinitiated sites sign detail had program in trial conditioning editing month of that to now and at have reopened AHA CTXXXX more at more patients, XXXX. AAT, X clinical report and of enrolling and to Phase few in studies some, Therapeutics, study. had patients and a that to patients the And data indicated treated CRISPR-CasX recent later trials. of the total dosing into In expect successfully we early new This of announced our patients of serious beta-thalassemia go screening program data In clinical VX-XXX the on the AHA note, and with all program the programs. gene now gene CRISPR potential disease. Of X these partner, we patients

the VX-XXX. We for have study will and also the now AAT from from data data next of months, expect Phase toward of to We when readouts or second data each end study timing Ultimately, corrector, pace picture the our similar the enrollment initiated we have for dynamics VX-XXX. VX-XXX a the In II several dictate studies much now with a clearer will these and VX-XXX XXXX. XXXX QX enrollment for of expect molecule.

pouring In the evaluate data APOLX-mediated us strategy each evaluating with into advance molecule of sites discover allow Our molecule the to to chemistry II study further simultaneously our multiple pick and is the through then of as in reduction molecules of development, goal biology weeks. the we open for II multiple can advance proof-of-concept Phase proteinuria best to consistent now is development. and study are for cracking did This trial to Phase and best VX-XXX, to clinical from that over late-stage on we FSGS, then XX enrollment the screening our pick just in CF. and

with the Stuart. an to type obtain diabetes. data from The isle X in IND-enabling this in X our for patients first advancement to alone evaluate of to expect late therapy study our XXXX, of are We study key tracking support type of cells initiation in for we year priorities with to submission XXXX. support diabetes diabetes, let the IND IND have were and And package cell the in that turn on the summary it type the activities in the business X data preclinical me key portfolio, COVID-XX the over half the to through generation remained of and and review pandemic. first of of With of track R&D

Stuart Arbuckle

evening our the all TRIKAFTA Thanks, $XXX performance. patients. Reshma. I revenues eligible this majority of very continued you uptake am across strong U.S. review rapid the quarter the million, in vast second pleased for to reflecting commercial were

We the both public feedback from private rapid from centers medicine. positive, and strong benefit for patients consistent profile this been received have CF risk payers, broad and with and has highly and reimbursement

TRIKAFTA working our worked immediate in for for a with to the reimbursement for that since of anticipated levels year. of have ability compliance to agreement XX patients receive performance so We execute agreement its their England unique the to with and that with time. as on many in come future increased next the which the an accelerating this in up now TRIKAFTA patients on to patients the underlying approval, older Further Europe patient be am disease U.S. of all last as October is for result include to expansion phenomenal as to which XXXX the Our community approval for a the in new to this potential few allow of is This to have have medicine strong and even to own KALYDECO, I expectations. in has KAFTRIO. the in persistence of well our pandemic. am amidst CF a minimal reimbursement from to with teams This mutation expansions the our to function will EC of us patient cause approval recent help trends bring SYMKEVI to KAFTRIO the they with launch Europe. benefited exceeded commitment a England first months, medicine refills very medicines. patients to of get provides I early label upon CF for among the build approval, and And community, thankful the treated also ORKAMBI, ages CF tirelessly inventory will our from with certainty first be opportunity earlier-than-expected launch, and proud revenues strong COVID-XX such date XX,XXX

pleased to and with value We very importance would us CF the of to possible. medicines. towards are the working NHS we nations and collaboratively England agreement thank a commitment of which their as developed soon agreements agreement, reflection with cause recent working for We as this like And of U.K. with the community the are our other to expanded is treating months. the CF finalize with equivalent in over patient underlying this

our I anticipated view of raising strong launch the in a revenue launch we our will And XXXX, year. in following U.S. how the perspective start regarding EU. for result the and KAFTRIO the As offer are guidance the dynamics to again we

are vast we X months have the the majority the treatment. patients eligible U.S., First, begun in and launch now all into of TRIKAFTA

treatment pandemic. second of to for see expect conduct the may patients' rapidly clinical and to third, half significantly rates initiation work the to launch virtually. that time to KAFTRIO in seen we with have have persistence an ability their launching This are to It be Europe support KAFTRIO. execute and global evolving and and normalize be will levels also compliance year. physicians inventory the patient teams had amidst we we date of initiation visits Second, first to and our launch impact laboratory a important And high, the will ongoing in and them the

we vast U.S., the ultimately patients in Europe. expect in the to of As treat eligible majority

trajectory However, the launch of Europe uncertain. in the exact is

All are which of these revenue factors and quarters Charlie their momentarily. detail review reflected in more potential will guidance, future on in revised impact our

revenue growth We by and TRIKAFTA beyond and reimbursement expect patients. additional KAFTRIO by and XXXX. will to younger This outside CF the primarily be growth the labels U.S. treat approvals agreements expanding for KAFTRIO driven

access as this to As and not EU Switzerland. And eligible progress countries of discussions with triple submissions patients are for with possible. providing yet more reach this where the markers our complete we year, KAFTRIO medicine to our as combination following patients to medicines, potential the of regulatory Australia later in plan for we begin of are goal approval continued rapidly our reimbursed in

would application beginning submit to for X,XXX new quarter age for their a the XX If approved, track some a The supplemental the X mutation. treat on del XXXX. remain TRIKAFTA patients cause children to drug ages in be XXXX. in in fourth with the new of years XFXXX also of eligible CF U.S. U.S. to medicine the in We of

we've U.S. submission by Vertex on Phase to and trajectory followed even similar progress in additional approval in over KAFTRIO months support remains the expansion long-term in We label the be efforts bring younger expect globally Charlie. turn with call and to children. significant XXXX. years. pleased III revenue and of this coming I TRIKAFTA studies growth a more the would by to patients outside summary, seen rapidly In near-term we the now am And and we'll to significant date commercial as

Charles Wagner

Stuart. Thanks,

launch performance following product financial continued highlighted quarter CF the second the was TRIKAFTA. significant in Our exceptional, for revenues growth of by

increase quarter Second XX% bringing CF over a year-to-date compared billion, just $X.XX product our to billion. XXXX, to total revenues were revenues $X

driven agreements included in uptake revenues grew $X.XX in the reimbursement are countries by the treatment quarter multiple revenues our majority of medicines million U.S. year, revenues U.S. outside have our now reimbursed, completion late initiated U.S. patient of strong the the Our the vast over the following second patients the billion $XXX outside Revenues U.S. in XX% the in U.S. XXXX. SYMKEVI, medicines. and prior and in of ORKAMBI eligible from second and where the in with quarter Both in outside

SG&A XXXX to $XXX second $XXX R&D quarter XXXX, of compared quarter to our expenses combined Our were million second bringing and million year-to-date the million. $XXX expenses for

half $X.XX of operating income increase Year-to-date As the significant the $X.XX in to of pipeline previously, half of CF our to greater compared of XXXX of expansion operating for revenues, into we've reflect new XXXX first in growth medicines income an the margin of investment operating XXXX. billion diseases. support globally XXX% spending said XX% income moderate million more with our expenses our use in billion, The of to combined and XXXX. resulted year-to-date $XXX growth for first year-to-date net and was compared the in

Now financial CF which midpoint, guidance to revenues billion range at our product revising total the growth to for guidance. XX% to Today, of XXXX $X.X upward over a we XXXX. $X.X reflects are billion,

globally the reflects the guidance reiterate the in Europe remarks. in of Germany. anticipated moderating the England, to uptake TRIKAFTA approval Denmark of launch CF our that the his with KAFTRIO medicines the Stuart it's and few mentioned factors midyear and specifically Our Ireland, strong exceptional performance in results, in U.S., Even a to date of important other and of

this we've of date compliance the in for our fact, among of First, stage are the the and persistence strongest medicines and any at for best strong. launch. they're TRIKAFTA trends And to seen

we are the a expect state less such, these However, metrics launch, as we to of the second the steady a than into year and reach still during half year.

TRIKAFTA the quarters. their a early benefit Second, resulted saw as as of hand end and patients TRIKAFTA from in on second higher we first refilling amount both a we previously COVID-XX having at the noted, patients the This pandemic prescriptions emerged. of

second I our conducting XXXX we given we is revised Our guidance rate assumes of in incorporated Europe note revenue see revenues half And we the the inventory while patient this and XXXX. that that ongoing increased pandemic into finally, are throughout also have guidance, a normalizes European incremental the KAFTRIO fact that of actual that the some would unpredictable, in virtual and uptake XXXX will level launch. for

for the that we rebalanced pandemic, COVID-XX on we With in I as our own have which quarter technology cash. OpEx the and disease Our revenue global infrastructure our top as spending tax reinvest note $X.X making of future invest accelerate in example, savings for to for are guidance our operation finished strong the in for is to medicines allocation a in to in to priority however, recognized certain second and of creation engine years, areas have workforce. actively work-from-home And profitability, incremental capabilities we interested. by billion unchanged. our result our remains capital the internal clinical and would expenses the trials innovation some support of with R&D certain external in also travel recent remote in both costs, investments programs and and we

Now concluding a back for to remarks. Reshma few

Reshma Kewalramani

Thanks, Charlie.

people, was as The the period trying first impact of half across XXXX widespread the societies and we grappled world of a with the businesses COVID-XX pandemic. for

business, our global and we significant business a Vertex occasion rise challenges, with how yet to thrive, and and work, interact shared in continue to the in Vertex operate thousands employees, the people As changes courage our of despite determination. these to with continues

year new you, the to X financial the and XX to X program the and future in remains commercial upcoming growth. with multiple increasing development progresses. as And I year, of the this support develop diabetes questions. to continued second our as clinic medicines half long-term discover the the momentum Vertex As months, open type with next true year and now for to look than business today this earlier and to internal call we remains innovation patients support Thank approaching our our brighter to data across execution, we'll of strength forward you pipeline readouts said both strong I and the the updating and enter ever.

Operator

[Operator Instructions].

from question the first of Morgan. Our comes Cory with Kasimov JP line

Cory Kasimov

I AAT about program. wanted ask to the

II Phase XXX initiated. it the XXX there is this any XXX the study between study versus looks patients for that like has and XXX. Are CT.gov, that's ongoing Now differences XX on key XX patients

specific behind there's differences if wondering So any rationale other and that trials? potential any the X between

Reshma Kewalramani

doses a placebo VX-XXX Cory, follow-up. it's same days X another the safety XXX not XX exactly few days follow-up, a of the month Yes. with different and treatment In of study a of X-month different our Reshma. group, few study, essence, similar, group, study. as doses the is about which of placebo Again, is in a safety XX if

very can think You similar. of them as

Operator

the comes Salveen And next with Sachs. Goldman our line question from of Richter

Salveen Richter

decision are board the half? for countries to with and you And CF guidance in factoring the then in where progress you editing accounting with positive pipeline, second the to do stand at DMX on franchise, with how regard England On for regard CHMP program? coming DMD, you how the are the gene

Reshma Kewalramani

Salveen, is Reshma. this

Let me come a ask take of first and how the then is and about thinking guidance, CHMP. step European we're course, back Charlie? tackle question approval, and to there commission DMX. still and DMD about I'll of Charlie And

Charles Wagner

Sure. Thanks, Salveen.

the from we do the and we revised Europe, on factored as approval assumption have in our into mentioned points we incremental not Reshma call, As yet. guidance but revenues approval of have out,

exact And clear. that so is timing the of not perfectly

guidance, in gave rate pandemics potential we a number said, impact factored that and That form that will including we very of the highlighted today. an ongoing the into for the be feel on good it virtual uptake. potent We it everybody of have the and also launch in the is about

Reshma Kewalramani

and program to then With the year, Exonics remember, in the to type got from time, we few diabetes programs, we DMX where regard that, acquired acquired late on we're you'll the that's where a we DMD and months and with. programs And of DMD, where course, DMD around with after and DMX the with that's we're we Semma, X summer and regard preclinical last working development. are

we We IND in pharmacology, our to have for and -- like. very look FDA. had good what conversations safety understand are the of clinical a We preclinical the looking and needs terms preclinical of with package they sense what have

give just to We and like are we're And in analytical the working mean we're hard very that through you know and let I'll going months, also I thinking to all very, things just development. that. anticipate of further I able updates. be coming on working you process diligently development

You've seen around make there that is right. sure in getting real the landscape dose that the you very the clear is to analytical importance are a what development about around dose and

good And time investing to all and clinic. so the amount get a as sure towards of effort we're that right make we that of progress we make our

Operator

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

Philip Nadeau

quarter. the on Congratulations

those Just on that a TRIKAFTA approved. a level will sense being couple be by brief us follow-up on label can have inventory us when currently? how patients a in in who at AAT. many patients you XX,XXX that second, one commercial reimbursement of of Could for the of Europe, And give of you of there'll will to Stuart, Commercial it's ones U.S., the give as and mentioned be you then the patients is sense time approval? held

Stuart Arbuckle

for Thanks question. Phil. the Sure,

for roughly will that's be eligible modulator FMF patients label existing the are across up about not for CFTR the -- per patient Europe include XX,XXX to the a time. who split, but with as exactly, patients, the patients mentioned, you populations. as split yes, So first XX,XXX to roughly Those patients

the largest in that. the Germany, obviously, cetera, to because the Spain, England countries Germany, is Other patients And follow we subsequently biggest U.K. the so Spain that's vast excellent other the in have patient U.K. the post nations obviously, Italy, in country like or so like agreement of begin is you and That's countries et single the will there. a are where developed population. where France, the is immediate approval. Then access reimbursement negotiations get and are Italy, places the majority France, EC

that that's patient kind So splits of out population how overall.

the we the that the a patients from made quarter. really in in have inventory, didn't It inventory any the see terms first the in second that In towards of sort patients incremental inventory hit of have not when not drawdown increase and inventory, quarter the patient patient first build second pandemic inventory an in build end in carryover did we of here we was that the seen the see quarter so quarter. U.S., of first and a on

Philip Nadeau

question from it out study of flesh next Could very the brief a there data the And into first likely What's that you be get be or an trial you AAT. once candidates? interim their the to Cory's to helpful. proof-of-concept steps two That's thinking way pivotal your would more on profiles? follow-up further move right will a on

Reshma Kewalramani

have do bit I and the a I in time go VX-XXX for for what after with that anticipate and had that? So a I safety yes, we you, be step have dose, hearing into a into proof-of-concept pivotal think sense look data at we it's moving antitrypsin but a next little about is alpha-X proof-of-concept development deficiency, the of patients once we we will get development. hard we pivotal once can have we data, and the

of in to on POC, yes, would programs what have cetera, I have exactly but X of having VX-XXX pivotal anticipate we a XXX point some at III we development. into depending to decisions, VX-XXX. and each Phase going we're the right is are those move the do Of programs make And course, are, lead. we et of timing after

Operator

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

Michael Yee

questions, Congrats Two everyone's on focused great quarter. AAT. Obviously, pipeline AAT. one on functional on a

cetera, your in to any biomarkers going look endpoints et is that, lungs, we that. there support trafficking help There around the you other think totality I on? questions or what's to at overall forward would know look of we outstanding Is to cetera. secondary the anything all et and

on actually any biomarkers XXX my So going I think to were help that's question then there is for what's if an second on? thing And interesting FSGS. support

great in disease. comments, renal are expert I some Reshma. you know an made You

strong of do of So amount that are what you a confident my a on show in amount happen question based Or a I that guess can need Phase in that to is, short you time? proteinuria mechanism reduction of II? time

Reshma Kewalramani

thing. Yes. Sure Yes.

FSGS questions and I'll Let the me get AATD, it and around then proteinuria. parse out into to

things, PK/PD for the So the about medicine first at it if antigenic functional ask the or disease. at be of the for? exactly other really that last we to this program, those we me, We is are looking X VX-XXX minimize you looking VX-XXX, point levels the relationship is glean X and serum put safety. on AATD is This that with into is And exposure And studies? of what achievement that been that doses we part I the AAT the it can between Besides will can't breadth you that. levels. seeking. the the for and time the relationship information the are patients and the AAT has What's from the proof-of-concept this

amount multiple elements community long think questions that of story II study you of about long, the et And to time of proteinuria a well the nephrologists, are complete. study. it's about the key duration that to think of like is reason duration, really cetera, of the those proteinuria, of that weeks make regulatory to regard glean et as of sort about long relationship so and in be outcomes all that I something to of will study the be short, the explains a take and workshops proteinuria, renal onset our There duration time. cetera. proteinuria must you With that b ESRD, as for of sort between XX to the the time I see to if have proof-of-concept to And have just the we talked reduction a going Phase regard start reduction. is been agencies, is when So to imagining able academia, see we FSGS, with

Operator

our line comes of And the with question next Alethia from Cantor. Young

Alethia Young

the on Congrats quarter.

CTXXXX. of there now about cell a and one-and-done a want talk are world? little and sickle about think And just you course, versus lot Blood the the I like bit medicines potential of of just a Agios of to how and Novartises Global versus So positioning in beta-thal

Reshma Kewalramani

the Yes. don't a the excited that hemoglobin This achieved we how tell We've Reshma. Why CRISPR with fact with it's CTXXXX is talked about program X This north the is XX conducting am these have program question? engrafted. that they the well I first Therapeutics. We've that I levels Alethia, take talked proof-of-concept in you per of has dosed patients. now about X in are in I conjunction beta-thalassemia, We've grams can't right? and that patients, deciliter.

XX.X. The first patient is at

at per second XX.X units deciliter at XX The from patient, patient is grams months. per first X of transfused the are going The patients blood year.

limited a XX duration. no is that number one out months. of and blood limited patient, now year patients the of units still to second XX observed. transfused a transfusions per The Patient is in follow-up we have And number it while

well out also that no the getting regard who's had we've cell patient and past data F really with had the to are XX% And has sickle X-year VOCs. first north of we We're shared point. So there. also hemoglobins

difference that a transforming one-time treating and that think therapy no a having VOCs, and between I between difference then having of to is transfusions and disease. a having big a restored. life There that in great is manage some And is amount big a importance there's a a there is disease I no think chronic So disease. person's

enormous see I this promise particular So program. in

Operator

America. next the our Meacham question Geoff line of of with Bank comes And from

Geoffrey Meacham

CF. good quarter another Congrats on in

specifics, patients? XX% are about Reshma, a to the other for didn't for of a CF is programs, stock, of I know want products. committed can maybe Stuart, just new but I commercial pipeline penetration lot sales, detail are question give remaining talk guys example, you've And and So versus patients. the status that quarter, et sort then you switching to don't us maybe on you the -- And with the of you a XX% have XXX%, to cetera? I the see one. for CF TRIKAFTA seen the premature the know plan what of remind the I patients for het/min but of just from how maybe close coming

Stuart Arbuckle

it's here. Stuart Geoff,

So the thanks for question.

very the majority source patients say majority the terms uptake between were were of that. really a approved we or the vast modulator it's of not of In patients to was before who the and and ORKAMBI who across of CFTR prescriptions. There it's those When TRIKAFTA segments, KALYDECO either is TRIKAFTA we on SYMDEKO. vast mean difference in just those all much or naive

the are launch alluded in is there the uptake the more about curve, part been the much curve but So at we we who are now, pipeline very hand why, pretty really I'll the but that compliance. talking and launch Reshma. much trends initiated, universal the level persistence part it has to very at for are being guidance, over flat still of and are flat of the question, patients about much to in of as is which And Charlie the

Reshma Kewalramani

Yes.

pathophysiology made, commitment And that helpful for You be stock what the in get problem just and for you patients CFTR to because make come patients. they no protein. are XX%, about we to I'll have the remind just very underlying all to is and everyone, is, last don't the of right no patients so the and cannot down, either any as far protein protein these make is those premature or modulators tell

Moderna this So partnership approach good acid one way our progress needed, working be XX%. mRNA a nucleic another, I and in And to last we have is see area. to with this on with going programs an or in therapy

to more So come.

a that surface there's lung course, made of the has and delivery but get that trying is of we're also worth surface has about inflamed and area. consideration point The into in to full an past area, very other we've important also very is, repeating a a lung large large is talking here. delivery And The mucus.

delivery with underestimated, made of progress problem get XX% So I the to at we patients. but cannot Moderna, be ability our have for good there partners and optimistic about our last am our

Operator

comes Crédit question line next of from Evan our Seigerman And Suisse. with the

Evan Seigerman

fantastic another on again, quarter. Congrats,

won't reason able there ins some is the to issue say, of pandemic. and be domestically? here moment, overcome the So understand the issues the traction for you the And is KAFTRIO example, Europe? Stuart, reimbursement these launch for in pretty kind think outs potential in cautious so are the in I arguably Germany? to to you how of of Europe in similar seem any the of so starting about at commercialization more of you But upcoming have agreements, that pandemic U.S. an And

Stuart Arbuckle

question. Evan, thanks for the

I were the really, the launch breaking would when about kind And So trajectory issue of think an up of in was your question, the you are pandemic little I is Europe concerned X in U.S. a why we say more now? things. bit,

we for remember, in of post approval and of you'll a first months our big in very, here you'll one is The rapid of have the from TRIKAFTA year. And very in the Europe. October -- QX got calls, as been the had the uptake approval, as deal in really last remember seen had the when the as obviously in U.S. that really first already few we been pandemic not Asia it and QX

think, pandemic into is and that. very I a launching, the so issue, like and still we a re-lockdowns dynamic we're openings are seeing where very very, and different things And situation

are different the I we very that situation, the we when Commission. a is approval think, into launching by get it So European

majority the the that is our organization from thing home. second are from home. commercial is vast all of different like that, working makes employees businesses, most The working Certainly, it

launching last see get to pandemic as patients ability is a us which and so medicine each is I for their thing The this a And virtually, the result we company. to as a linked about first together physicians which and pandemic. say, will the is would other be of and

which dynamic a into play that is all out. going it's said in how And have prepared we that's to just as so remarks, we to consideration. our uncertain take And

the into the think situation Europe I launching U.S. we're do in is to different enough we very be the fortunate able here in situation, to launch into So were to

Operator

And our Robyn with from line of next the SunTrust. Karnauskas question comes

Robyn Karnauskas

learned some more the are around bit we it's in positive for this you be that just on launch uptake ask is do really think just ORKAMBI? I in lessons United when you what regarding launch the what all this with Congratulations KALYDECO think versus just approved? little environment. the because more the some KAFTRIO -- I that wanted see like in Europe, What to itself COVID? question to addressed about dynamics. positive just about just the European on And States launch could reads and and basically, were progress TRIKAFTA about compliance So a the more reads you might

Stuart Arbuckle

it's for Yes. Stuart Thanks here. Robyn, the question.

In terms of take of the been the we from them because success. launch has a such can positives U.S. obviously, experience, many the terrific there's

levels think regimen positive there's obviously, positives its triple the the of CF within benefit very some high community combination profile. be, risk and I would awareness the very of of so And

has very pivotal played benefit indeed, Secondly, really we it that is real-world our overall really, The very, studies. in in out risk the And exactly strong. out saw profile risk as play positive. positive benefit is earlier efficacy profile and

definitely resulted and those of So levels positive in persistence are high that's compliance.

XXXX, a back as different all than positive we are So those say, very uncertainty, big in were physicians October I which this very, in I launching of of to ability think world environment the are launch The and unproven. just very virtual to situation in launching is get a ability is into many read-throughs. patients together we ways and seems their a terms in lifetime and both away, our

it Europe reimbursement process And to in Obviously, in clearly in likely and longer place we don't in Europe different. a the in did going process. the number take got advance to U.S. private have that's to public U.S. very where that in positives of place, agreement begin countries the one here reimbursement in have we of So here there than of and positive. clearly that approval. are bit have thrilled is that agreements a in England a That's we're yet the though, rapid very And is to reimbursement. got We're

positives, things be are different, difficult the So majority the as over this of to are final the time, said vast, there's are definitely Having confident many number that, leave that going there that time. thought KAFTRIO vast initiated launch which with us call. we on to the done a just of but believe U.S., to I is will in exact trajectory we have leads over you of patients be will which

Operator

Citi. from next with the Mohit And of our question comes Bansal line

Mohit Bansal

end question AAT the and on functionality. is my the My from progress. as Congratulations well regarding

is have a patients mutation. these this that -- Given

the be out even So may Z-AAT, that you when suggesting AAT. and which Z-AAT the of gets vial-type correct there to not protein is functionality as there's misfolding, going be literature the deleted still as good

issue? end, do it So levels? could an you AAT that are you to functional And exactly be managing the how think

Reshma Kewalramani

Reshma. is this Mohit, Sure.

in The molecule AAT, corrected functional call have disease deficiency, assay, in that lung the The have do you think to pathophysiology you're as you When can the so, from correctors assays the a the have lung disease, the we're of misfolded its neutralizing liver and of with ELISA alpha-X misfolded people this this it in autodigestion, to So point you doing the the job, the now and you they our small so work assess good. that lung allows our competence properly molecule, and protein a go the congestion that the and can of we that protein. blood, protect neutrophil molecule grounded way all assessed genetic that leads addressed you're which have but right, protein. just that have the and you small SERPINAX can and that AAT our the liver about liver that's of antitrypsin approach here. corrected sure fold elastase in we raising be alleviate gene, And mutation is how the looks correct can So both competence to is is the this because models, a approach. to It's make assays, the that part have of how the in And preclinically the disease to when high. disease. is protein we on mutation. is mutation we the And it functionality an that right?

So that's Phase proof-of-concept not II, levels, why in measuring that's the functional entered level. Phase only II reason also we but antigenic we've into development, and are

readout. we So direct don't have a

Operator

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

Geoffrey Porges

it that are just England. volume-dependent? you that the place Irish incremental XX,XXX patients want I give the us similar to -- the sense in England. it mentioned, you Or cap for government And purely spend numbers on NHS of with Could a have agreement the in agreement, the of in to is what potential for in proportion you follow-up Stuart, that the the on secondly, patient a total in England? agreement is first

Stuart Arbuckle

Yes, Geoff, thanks for the question.

see England, obviously, reached that would our patients agreement. the thrilled have to U.K. ratio same in mutations. in as is the yes, we're So that you the other The roughly of FMF agreement for the

think it's population. at the look others when of that can to the population so you you size versus broadly And CF U.K. proportional the

is to including the I the to others it exact terms medicines. once terms that agreement. KAFTRIO, talk terms the approved In about of of access exact for additional of agreement, those medications, indications can't does It the and our existing similar include approved

and age it U.K. a going give that England total in certainty on groups. down cap include into to the include And does expenditure specifically. government So would some the this to case, NHS

Operator

with line comes the of our next from Barclays. question Wang Gena And

Huidong Wang

congratulations I strong on a my add very also quarter. to would like

have X I So questions.

revenue regarding quarter The roughly first no to refill translate XX,XXX patients. -- assuming would is revenue, the that if one rate

refilled? percentage rate? share for was can regarding patient the So bluebird the also And your would second trial, plan beta-thalassemia specifically, And my What could from question is, of question discontinuation experience? that registration you sickle the be what is is the what learn you cell. a

Stuart Arbuckle

heard sure not I'm all Gena, Yes. I of question. your entirely

you the you percentage the on have right. think that stayed are medicine hearing were if the of I asking medicine, about on -- patients I'm

Huidong Wang

so persistence, rate? amount of rate, the refill the refill the like As

Stuart Arbuckle

Yes. Persistence rate.

TRIKAFTA patients very were of approval Of have XX,XXX its approximately those, for on there's the label. majority yes, the initiated in U.S. the vast with broad those So FNE who eligible the rates the persistence very, and persistence call we opposite stayed very on discontinued. who are number have The the high. i.e. rates, therapy the as those medicine, patients of it, of

medicines launch. any Some time highest in we've seen at the of of our for the this

you And the There way, other so given surprising saw the our over that's specifics there, medicine Reshma. they can the whilst question increases getting only this think very, risk very, that doesn't beta-thal very trial. I not profile in of and sickle that benefit And the And over rate to it's of on -- high. not what hand high. we're the we I'll but very cell, is, imagine into discontinuation time, Obviously, aren't it go right? exact are pivotal

Reshma Kewalramani

Gena, sickle the what terms trials, the we show trials to need the regard designed think we've III that with trial you right to job. pivotal beta-thal do that I Phase in for of the and are and see

clearly reduction beta-thalassemia well. the the we've designed transfusion think In I few cell, on caveats, key. terms has sickle about VOCs, it's in endpoints, those are that of endpoints the that ones, and of very I And obviously, the CTXXXX independence very think, all the metrics in is I both Early of right those watch. think are patients to studies all performed stages, which but

Operator

Matthew Morgan And our line with the next comes question Stanley. of Harrison from

Matthew Harrison

are drug have I the staying that for patients you can approved on for Stuart, that SYMDEKO any Is residual guess two you and comment not noticed ORKAMBI? III groups, I or hoping just if question I the you think on could is Or levels the on And surprise guess are strategy that mainly may TRIKAFTA carrier comment me. look to yet? regulatory replacement groups if One, you which on that then program that was age protein observe versus, the has? level you second say, of just Phase similar related different therapy AAT. to what the you.

Stuart Arbuckle

thanks for Matt, the Yes, question.

of TRIKAFTA. other majority are transitioned who vast SYMDEKO for or patients to TRIKAFTA KALYDECO, eligible who as the transitioning or just ORKAMBI, are patient the So are for on groups have

So see there. groups, where we is less the business those remaining XX, age the are our who in are are that the what either, expand patients younger on of the you continue to indications than who so performance that

been new have been seen SYMDEKO in referenced continue ORKAMBI. following SYMKEVI, we've we say, or U.S., launching in that the reimbursement countries the growth where the cases, some and I have put -- U.S. in his here as Charlie to the and outside patients we remarks, U.S. or and should so place, in And outside patients agreements initiate

will what's It's Reshma's. and age either think I business. that So outside ORKAMBI existing that's And driving U.S. SYMKEVI KALYDECO, of or -- the groups younger be the SYMDEKO, but AAT,

Reshma Kewalramani

Stuart. Thanks, All right.

link of step take and to On you give and the are Let thinking a about Phase terms strings of III in question me together. of that just we one in AAT III what II and we're back how Phase plans? Phase our just doing what sense

placebo. in to obviously antigenic do the and of We're II, AAT we're right and and exposure there looking number at dose studying is get the safety, we're what are going dose. looking we're of at functional different versus And estimation to Phase the levels. doses the right? best a select levels to relationship serum AAT So trying dose, evaluate We the

Assuming I've based evaluate the before, measures. with our the II III position we next a meeting is in which as move assuming are that a results, that, to these Phase few but on we we that yet. seen, III Phase going haven't in And FDA Phase What had is obviously different forward do haven't said to step. to we're

We haven't that Phase II end meeting. had of

looking a that is be an levels. be framework serum the through indeed So agreement those meetings levels. come antigenic agency, to be We with at but would and conceptual end at looking need obviously, would out with functional the we we go framework, the but would other this

to to have an lung. polymers. planning the are with of this both treat again, from liver clearance the molecule the opportunity We have of assessment these the small approach, and And we remember, liver

that be levels therapy. the a package. their X need number have need of confirm to approvals going them with have gotten do to as agency. to based said go the we of that, augmentation that in U.S. to I've About make AAT. on We plan the of would we are acceptable. So package the that, companies and that times And We discussions a through be previously,

by package the the the of and And to interested really the so test, be about. the levels might we AAT correctors we that. or molecule our like and impact small clearance those need a function antigenic continue all is But functional that in, liver. AAT think imaging very function others Separately, and I'm something to for measured interested levels, considerations talk framework sure are pulmonary and through polymers from understanding we go our lung are on in of

Michael Partridge

Operator, Michael. this is

We'll minutes few a X just more questions. take have We left.

Operator

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

Brian Abrahams

with And corrector congratulations thinking couple could of that AAT confer Functional or Have AAT your like? work pivotal well either done out to term, that patient A heterozygous on also. importance development where on a as benefits you've the of the potentially from might longer fit optimize complementary peak And preclinical interest XXX curious, plan? and really level a population the lines? also AAT look to assess a then like into of could externally talk schematic the My an exploring quarter. mechanisms, potential combinations along or you for of to laid outcomes. about trough you versus high future in augmentation those smoother sort -- the ways levels Can exogenous this. the what I'm internally

Reshma Kewalramani

but let in our replacement to the molecule. or augmentation Okay. are keep There's about a protein but it's things terms maybe bit basically all lot mind, question, think couple important. to approach we in we these packed it's with small of basic, A of what versus I think often and is into hard of tackle me what try therapy where that a little are exogenous, how about

of say that liver, causes lung. I exogenous is it protein, doing is will, effect cirrhosis. So is fibrosis the that you which because augmentation in element exogenous could production if have is lead the is the therapy gets to for reason could and that what let me nothing point out there. with One start therapy and stuck misfolded the the critical to regard to only on And

one help approach. the lung, big only could about exogenous than molecule different the possible that small therapy is the concept it So organ that very

a the have The a get let's you you is, of that on the the augmentation to infusion a go you raise is Friday. PK of And the say works therapeutic. course second week, way The center during peak-to-trough. therapy this point once that and good to you a augmentation week, you and protein the around went you center an received,

period, you in you AAT. more to don't people you don't accordance in happens who This intervening me, and what And from have you that what in have received. is any alpha-X changes what So your body that production antitrypsin deficiency, important is in very have is needs. Friday, Friday AAT because what to

AAT produces order multifold So can the need in body to happen of to center times into have example, for once-a-week when more for job, therapy. AAC infection, your you of None autodigestion. is which go that its inflammation, this medicine in do stress, prevent

to So there today are change small the of the It for maybe body decay many you X depending as molecule other to body. own around is that its has therapy hope not fact how of the once is promoter. that and third the a works that in dose the helps. is it just week control being that has point it its And and under over AAT week, the I the lung ability about, allows as talk this think needs we but are treat of and could what is can differences that you the liver. approach on both augmentation

Operator

question line with comes of last the Paul And our from Matteis Stifel.

Paul Matteis

Great. clinically derisk ZZ second, should with then or of I'll order more the it I with be I questions. I you to some think you was data, product on and now to mutant relates comment data a range AAT to next X to curious, continue was and you'd differences XXX, that, do going history as you that pharmacokinetics wondering you're based genetic patient confident trend molecule materially natural potency study? that Phase if to a could increase AAT in One, And production that potent? the need is of just are guess, what the II

Reshma Kewalramani

a AAT had questions. lot of We've Okay.

about it's So fairly guess our AAT. be last I question for to

doing. for really is looking before. that around looking we're is we proof-of-concept II one terms XXX and tell done and have is So XXX, just No little I levels Remember what this we a that what are this does a looking is we're has question what me let behind studied bit study. of for not think you question that of in to target molecule, the the And what your a we compared at? in Phase are really how right?

We to translation through the we going this first along. ones time now with TRIKAFTA from to bench are When and is to this CF, learn KALYDECO KALYDECO. and the and the already came bedside trial. AATD are SYM by translation translation gone we about to And the were patients with we'd the there, our with to ORKAMBI AATD doing like obviously the

both the once pouring the and it's I'm on know easier. for study: get exposure and First, -- the relationship ensure and between the we're on that that So levels exposure to I'm we with chemistry, proof-of-concept, PK/PD what disease, looking right? third, we cracked here's that we and we have looking put patients levels, met to second, safety; easy, functional and Then movement because in dose a drug is will to; chemistry not that the and looking antigenic we're up. from and for exposure is that but this pouring cracking we've in saying know that to levels for proof-of-concept have between need for we is good movement tell looking biology upward safety We we're what AAT what and us AAT get we biology, see the into the

taking we that then proof-of-concept, so those about think once And we can have steps. next

what looking from Phase that's I'm for really study. II So our

Michael Partridge

joining a for all evening. safe, you team appreciate us. have The Stay Relations Investor and good you available have tonight if is Thank questions. additional We in. you Okay. tuning

Operator

you for today's gentlemen, and conference concludes and disconnect. call. you this Thank Ladies now may participating,

Vertex Pharmaceuticals (VRTX) 30 Jul 202020 Q2 Earnings call transcript