VRTX - Earnings call transcript Q3 2020 | Vertex Pharmaceuticals Incorporated

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Michael Partridge

Good evening. Welcome to the Vertex Third 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; Dr. Officer, the the prepared Scientific the portion Chief following call remarks. Altshuler, will Q&A join of David We recommend that our website you access the you to webcast listen this call. as slides on replay is recorded, website. call available conference This and a on will being our be

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

Reshma Kewalramani

Thanks, then general Michael. turning to I'll begin review the Stuart programs performance. for details of it our provide business before over a few R&D across comments commercial on the tonight with a and

First, with business. for CF our CF. remarkable to XXXX growing a starting be year continues

launch we guidance to perspective be to as we driven third to on year-to-date the CF, the performance known are Europe. billion. both. billion provide I in as is revenue strong in characterize launch Our are are upward to a $X aspects performance, and and Based product revising sure that a two our outlook our again XXXX through our by KAFTRIO, the There $X.X on U.S., important want building TRIKAFTA we on quarter progress was in that triple combination

And see than from development and factors: poised as exceptional. Driven mutations, of top is expansion X to which as XXXX. lower geographically, There's beyond and that, are groups about our by are significant have label, XXXX, ways XX% at we and medicines for through more the the Near expansions. will expansion growth, medicines, CF of and growth. who for patients XX% deliver geographic Stuart more eligible many The been treatment. currently the CF XXXX for line bottom drivers triple to beyond eligible in moment. yet a two revenue our last XX,XXX there a will CFTR growth and reimbursement over are more growth into cannot growth population first reimbursed patients we who least modulators. there go this age additional is has of on become begin term, combination to benefit think these the treatment well in who to details of anticipate even who

business our I of aspect is want leadership. to other The address

will we regimen. this last when the have fibrosis, patent We very position the at cystic as to time high in strong and by believe coverage into set leadership a and the TRIKAFTA significant base patients benefit least case considering the of bar XXXXs for long this late a a

the pipeline. Turning to

a working for inherently that the disease it exactly and can of increase a candidates pursuit so portfolio externally expected portfolio of to to success deficiency this bench and CF. is program. outside a And validation and X be proof-of-concept and out diabetes target Our projected on human opportunity. a first-in-class we for we in biomarkers strategy is programs diseases. diseases we development they are potential an patients. different disease the endeavor, the cell And year. The internally market. very represent represents this our and CF. therapy. programs potentially to next has as by Individually, in our are program each causal of in our large the disease, alpha-X serious develop serious to applying high areas deep understanding sickle and based FSGS a disease, innovation, a the X approach the exactly advanced high-risk relentless between A selected each Because for first-in-class And the fidelity with of to The strategy, a best 'XX. bedside. already working beta-thalassemia, these we broad we Vertex biology, pipeline we the programs pipeline chances are With reason advancing strategy best-in-class is with in deliberate collectively, serial read success same treatment or now on every of investing of reflects begin is strive Once in number in of transformative end from very of type a in both important treatments in This patients pursue clinic to have bring for drug disease. data one drug transformative antitrypsin are to is

fulfill early their Some compounds fail, not and will potential. some will inevitably programs

molecules firmly all of the the investment create R&D drive patients succeed. of corporate for or them portfolio, if to, is programs will the believe innovations returns they all That and terms the of and succeed, not on the outcomes return exceptional Vertex we and for In of have will, not our our X even And X for exceptional that will strategy. that shareholders. emerge

multiple the for research through to mid-stage This in advancement is That early molecule, a of have is and portfolio potential program. of approach to our Lastly, of anticipated early our words recent or our work. into in This clinical extraordinary is disappointing. the development. unusual parallel, is is a and neither a discontinuation clinical program Hence, it in program The overall is strategy. AATD nor development. approach. discontinued But an molecules any VX-XXX say, few example at about portfolio it

or Our enthusiasm for how follow-on is the does molecules. AATD we it not diminished. our And change VX-XXX program think about not

some Let me R&D some of events. reviewing CF. more in and highlight pipeline now specific programs our with detail turn to upcoming Starting

forward a lastly, with treatment of and the track XX and of front, underway. CFTR to protein III a study who bring approval of we on Phase the to cannot this pleased progress serve CFTR is patients TRIKAFTA CF And CF in our not second TRIKAFTA potential preclinical olds in efforts served patients modulators. X-year The XX% continue hence, X in age any On with XXXX. quarter make completed to with nucleic the our years using and acid I'm by to in last be are the have for also X- We last this filing study XX% of now to therapies mRNA Moderna, patients. collaboration do for

safety study This is treatment people study up. in to XX The a Moving days, II ongoing and Phase to proof-of-concept continues follow XX study is AATD. dose-ranging is XX The Phase II VX-XXX days duration of with enroll approximately and by dose AATD. patients. followed of of

We this in preclinical goal development first X with least expect new at through year. program a XXXX. from results to of also We're entering the correctors next molecule the half of advancing clinic corrector small see additional

Next, the CTXXXX program.

Our gene disease cell editing beta-thalassemia program gaining sickle is transfusion-dependent and in momentum.

gene Results dosed engrafted. that for will the summer, enrolled both X for the X clinical the vivo noting in this EHA CTXXXX, disease. beta-thalassemia sickle patient you data ex additional then, a X had provided were on for and included patients progress, June patients conference total been at results in proof-of-concept studies. and also cell Since As had in CRISPR-CasX patients updated recall, the presented disease. with study, we editing we and enrollment of with in have dosing across successfully Also treated you all which

program, follow-up weeks. over the Given proof-of-concept including patients data of is our enrollment the proteinuria by and progress durations XX to longer look FSGS more study II across end reduction reporting the forward of safety, year. and trials, regard this to VX-XXX. we these Evaluating With in additional pharmacokinetics Phase APOLX-mediated ongoing clinical in the of

and to key to from the before our this the half in manufacturing we on submit the for IND And 'XX. required the initial track application FDA are completed and type this obtain IND-enabling expect X data work, We were which an year, year. the first we in have to study of priorities therapy of diabetes, cell end with studies

including the CF and in In genetic condition. islet therapies disease of which strong disease alone, QX, approach we've on this the summary, I'm growth over positioned portfolio to Stuart. holds the small important are truly We and living now to And making. many advancement the is our made optimistic across in who ways, and business and program of number patients this we progress transform continued expect continued to are about across different for cells This in potential areas. cell molecules, the so multiple progress the well study first and we're with focus of

Stuart Arbuckle

positive. a little patients. been The and that recognize enthusiastically tirelessly response evening, Thank U.S. I centers is the a eligible the review our first ago. It COVID-XX of provided CF for year approved immediate CF worked am the disruption just for treatment the product you commercial performance. to pandemic. patients approval has community to amazing with Payers even have welcomed to continued you, this and in the think pleased TRIKAFTA was Reshma. incredibly to value initiate The the during caused over the by medicine access almost strong

being patients As uptake U.S. the were the are $X.XX third for revenues impressive majority TRIKAFTA. in Total of a billion, the treated in eligible the vast product reflecting now CF U.S. this result, today, quarter with

the they the personal relatively pandemic. to that at continue of of are We patients built maintaining observe that beginning up high the levels inventory

receive we focused to maintaining in patient we important our on KAFTRIO are and performance in revenue late In compliance been factors delighted August, persistence Europe. to forward, Moving have approval contributing year-to-date. of rates that for high the were earlier-than-expected an the

to and to immediate combination medicines, England, continues enabled posed begun in where secured Ireland, fully execute provides first which by of which have to launch. the reimbursement KAFTRIO new ahead what other patients Germany, the our countries We surge we Scotland, for Denmark. triple has Europe, countries. access multiple Wales in team approval, COVID-XX, Despite access our and is including challenges to remote

agreements access working where all have are and for we also countries We secure patients don't yet reimbursement place. eligible to in in

patients the time, is majority high. And the U.S., expect in enthusiasm be CF treated. As community we vast the over in of will

significant impact launch fourth of to become a the in of driver growth expect quarter. the the We starting KAFTRIO

progress factors are we've revenue impact date. these pleased bringing year medicines Charlie by guidance, I potential on revised of am reflected our their quarter the the our and and in which review to of to will patients in XXXX, made detail. for more All results full in fourth

reach working with CF are after to possible see we are patients in to beyond teams And CF expect we hard approval. eligible XXXX. as regulatory growth quickly that able ensure Our to revenue as all continued

has all it CF always as patients been, the CFTR to might our who benefit remains, treat goal with XX% Our modulators. of

our is patients eligible start medicines have progress are they patients. there treating Today, this great done. from many working primarily and made have Europe, to we all are and While have toward are still reimbursement of in patients towards although far to and our These yet these goal, are our access treatment. job who, teams

confidence CF the heading approval future countries reimbursement The in for than XX; As near portfolio next, who for for Australia; age will and X more we and we regulatory about the this with our mutations at the U.S. label combination first, approvals of our and treat CF medicines. disease. CFTR to with able we shared CFTR results of of because be the patients tremendous in We our in have both look we expected in for beyond, and children reaching become there challenging this as years to in to that to you additional medicines regulatory our are delivering belief XX% as into teams secure remain continuing reimbursement grateful progress. in service our our third, am and all XXXX and shortening age we internationally, and Vertex in all our It during times, groups think and younger in of approvals CF include: patients EU; eligible our and to very additional to expansions update to steadfast for to the on And commitment XX,XXX that believe KAFTRIO of come. in all eligible CF reimbursement drivers additional long-term reimbursement achieve I dedication triple modulators triple the reimbursement our high business patients with will patients agreements. key combination who our forward growth our agreements continued reach have geographies, ability is life in such delivered I regulatory finally, for to in these additional living patients' modulators. CF

quarter thank like guidance. with once Finally, results I now community reach would again shared financial and for to our goals. the third their us CF to review collaborating Charlie to will our commitment

Charles Wagner

of quarter strong the the U.S. performance TRIKAFTA third we Stuart. with our by Thanks, the XXXX, In headlined continued exceptional in financial results

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

outside quarter and Revenues from Our driven completion And over year. grew U.S. included quarter in the the of year see U.S. beyond. revenues $X.XX launched million quarter in quarter prior our XX% U.S. the of revenues third following as $XXX the several revenues the in third by uptake the billion medicines agreements Stuart in fourth expect outside continued late in third to to international and KAFTRIO we contribution so last mentioned, reimbursement the late the increased

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

more pipeline with $XXX income that as our both of quarter targeted first profitability, revenue we strong combined with in priority for clinical an cost into to quarters reinvest of to an among strategy, cash. in growth growth to for XXXX. and resulted in disease corporate significant our our was the year-to-date quarters large-cap our is million of net in of of moderate the XXX% continued diseases. X rapid with Year-to-date XXXX income of year-to-date of operating first areas several XXXX serious our in innovation, In CF engine top medicines global With development spending, and finished increase X expansion the summary, $X.X XXXX. strategy second with deployment in aligned well and business new the operating of performance combination our the expansion billion internal capital billion and $X.X $X.XX and revenues, XX%, financial billion, of in support growth, biotech. expenses of Vertex's Consistent as R&D innovation in progression investment The important scale in the programs. compared income the Our increased for R&D in exceptional compared is accelerate attractive margin to operating external to our an transformative unique represents profitability reflect

ongoing on to Now billion range to revenues over of and XXXX. view the of revised This guidance billion, again guidance. for which a are product upward the quarter current compliance in revenue. the impact persistence total the primarily of XX% impressive of KAFTRIO midpoint and once performance pleased our growth our we $X.X U.S., Today, revise reflects reflects and launch fourth the TRIKAFTA to trends at guidance financial $X.X XXXX to

of non with $X GAAP OpEx, in we year-over-year our For maintaining increase guidance $X.XX R&D. of are billion to billion, the the vast concentrated majority

progression technologies are further be and investments programs expansion critical pipeline investments our data XXXX, our as of the generate progress quarters. in With XXXX primary of in programs coming results as the The and driver in across to of will these and well poised capabilities. we in clinical important

ongoing investments patients we I/II we program enter which move therapies This as and diabetes expect example, clinical initiate in our a type remain For cell in our and advance our further genetic next X development. to programs expect include program, a Phase into as CTXXXX priority we and study Phase the year. Vertex efforts to I/II clinic

repeat, we so utilization to are Lastly, rate that tax remain subsequent non-GAAP full due years. in at our XXXX tax certain range that year tax expected I note rate not is to of of the to guidance XX% are to our expected to rate benefits our to the revising changes would XX%. tax a lower in XX% XX% non-GAAP assets, XXXX

for concluding few Reshma to back Now remarks. a

Reshma Kewalramani

and the head potential very of and into with breadth the I'm XXXX, pipeline. of the pleased CF achieved we As both, our franchise growth

the I pipeline CFTR labs science CRISPR patients development long-term in such investing working our modulators transformative partnerships medicines five strategy clinic built we strategy therapies in our and deliver progress CF, in of to we we best-in-class updating I on Thank and business are research months. in continue transformative external proof-of-concept with by readouts CF beyond. the and to investing receiving and success. track internal development. potentially as to programs more through to up coming is treatment, have broad our history, of of continued and point of innovation externally, and to the of multiple to our additional and first-in-class diseases eligible multiple internal the as acquisitions so extending forward in already open Semma our will a the as be for into as This With the portfolio our external with programs. this into development, is the outside pipeline innovation Exonics, diseases especially recognizing innovation now coming outside teams, advancing, make with business internally differentiated R&D investing the becoming current our CF call it confidence in Our you the of and in look providing R&D forward and a with Therapeutics the in and game-changing well want clinic and science, we'll And business Throughout growth. of programs move and progress R&D of XXXX multiple clinic proven to of questions. core you, across and year. in and now on

Operator

Instructions]. [Operator

Phil comes Nadeau question line the & first from Company. Cowen Our from of

Philip Nadeau

sounds have won't volunteer in studies anything suggest between can LFT molecules? were XXX preclinical in point you And with or figure bioavailability healthy do XXX? are about would same -- to the optimistic that investors based therefore, really differences to issues should the particular mechanism-based think last how specific. the on did? elevations And with week, out two that the Or our XXX there be that I'm over about trying that the you any It XXX like, particular, or issue from about So whether question? we how conversations molecule curious, is in studies the

Reshma Kewalramani

Reshma. is this Phil,

with to and that comment start David me I'm who's with Altshuler, us, to question, Let ask further. going

about You're on or be considered in from important there considered mechanism. the whether really specific asking And be findings should about XXX should different XXX molecule a is another XXX. question how question with there's

bit to you this that I that just specific and to let where mechanism. some of idiosyncratic tell I'll that on I and understanding, believe David give we XXX And give molecular molecules, a VX-XXX. you I'm have chemical going no on have David is reason are From our between are specifically then you So tell mechanism to believe to XXX the ask ask few little different differences different versus molecule and data And are, color me XXX and entities. a on you we to is clearly then this which more color. all the structures. these David?

David Altshuler

thanks And Reshma. for question, you, Thank the Phil.

mechanism. regard we in not we and don't with although that findings of or whether that are to There's we reason really conclude mechanism for can't course, First, LFT X are the XXX, most on on these there of logic know think sure, to findings are we idiosyncratic lines no see or know, convincing.

it that mechanism are in based, And stopping a is very of programs chemical as typically not these common abnormalities based. first early is cause to LFT The have development.

those of regard carry see where that histology. histology human hepatotoxicity. multiple just ZAAT a studies liver evidence mechanism whatsoever The and second our done in hepatotoxicity, with gene, we happens. are And and seen small improvement the molecules, We not models, specific the actually see only with no polymers and liver we that do to injury we is treat in I'll have interaction models of mouse and that in note our

to XXX us it's that we And mechanism things data, forward conclude chemical abnormalities, and makes with specific the it's that certainly based the mouse data. LFT being most nature to so common premature combined look and of common think the

and the different potent we They to of We into even opportunities represent next-gen treat put molecules, is to XXX. in have experience, metabolism. asked different chemically correctors and different XXX close these to class physical you just disease. XXX same They're And X structures. just different Finally, molecules. different the properties. have more than quite the properties saw our in different CF does clinic. multifold, that XXX and multiple And close, about really

strategy. portfolio our underlies actually that so And

Operator

comes Our line the next Goldman Salveen question Richter of Sachs. from from

Salveen Richter

you speak can optimize them program, And third-generation compounds and how might Ford? a you able I your AAT to on to the On question. be second the have

Reshma Kewalramani

some the our once for case pursuing like a to generation coming think compounds my I AATD. early have I pipeline, that prepared the what's comments. disease absolutely through David development. our of idea sandbox, asking honestly, relentless transformative David, thing forward in want were to VX-XXX And is I'd this as happening Salveen, any in of said to have behind make And approach ask multiple therapy. exactly make our to molecules? And of about remarks, you by bringing parallel that's next-generation I'm across defined you The molecules in we the you up say we going are points about but one is, a in AATD, in AATD. do of entire about

David Altshuler

move some the time do, preclinical always that that differentiated we're that have look Just of properties late for a features. to for as improved are to we've in been get improvements and and excited we all would working development in to scaffolds molecules potency features in you have multiple forward and would

Salveen Richter

your Ford? as then in into secondly, and business stand speak you development if commenting setback And maybe strategy kind this the of as mRNA programs to outlook terms here clinic? on well and moving And changed the of has on CRISPR-CasX they just initial where this your can

Reshma Kewalramani

thing. sure that. me Salveen, Yes, let Reshma. This is take

had long business Now development. in we a interest have

Last year, We know we you have be this internal fundamental belief innovation, external. And it interest billion did worth activities. remains. business or investing development in $X.X of that over in

exactly remains XX%. Our same. in the technologies could business interested with of are in internal say, help very particularly CF, one have we of is R&D, same the X and And pillars in our terms to with approach interest, to us strategic development specifically, desire last that remains. That the be that that

in could are Affinia in in We tackle and continue And our Sandbox. are we deals we tools others. did interested that interested be with very very Moderna that and assets like diseases and to equally Arbor

use obviously, really growing we're I'll out. strategy. our to get strength. mRNA. focused I sheet to financial has capacity quickly have fit strategy Now this our within the the bigger that that to or very pretty are deals. And laid changed, And what we that certainly balance that do on deals we development perspective, from think, And business thing is our through more prepared disciplined for is I've opportunities one our

think XX% I Moderna with the CRISPR-CasX, way asking I'm you on be working must CRISPR program. about Moderna. last the teams our therapeutics. San with and partnership pleased And the with collaboration in Diego CF are with together that is in

As we the results you beta-thalassemia we've in dosed early and heard Those are duration functional being small and more in more summer. is year we're very the impressive prepared much the cell, patients summer those EHA, the two to enrolled and patients, very sure, cure sickle more with the for it the And a to we've since of remarks, that to since patients. patients. longer presented patients. X us, over really were results end looking treated before those that But of follow-up. of numbers for in forward intend And we share

Operator

Geoff from of next comes Our from line Bank the of question America. Meacham

Geoffrey Meacham

the behind portfolio setback reaction but there's the that lot was CF. COVID if any, couple. confidence Europe I are question thought countries tweaks plan? the in going a The is, after have And AAT a the full in then just implying it of to question, I Stuart, overdone, One, is stock Reshma, with not the launch quarantine. investor into there bit and what, second a accelerating

broad. to in Salveen's scale growth Is on the won't potentially catch a there an up So like starts for emphasis just through to it's when is larger question. or to moderate late-stage it mid- It early, CFs pipeline to put time wise? BD? The pipeline urgency move seems

Reshma Kewalramani

the for question. Thanks Yes.

to ask of Stuart then the go Let and first, part second back for come question. me I'll your

Stuart Arbuckle

Yes, Geoff, for the thanks question.

over So as to treat and from destination number the expect, exactly been that KAFTRIO in the obviously, there. pandemic. rapidly vast are to get the and going obviously, saw factors patients. is is, The we in we and time, that one community that has eligible there the quickly how Europe will a changing we But here real of are to clearly, are the question U.S., majority ongoing we reaction high fully of important

of hasn't for planning In had was really question, about that's terms changing virtual and Obviously, how uncertainty, easy patients launch that at be leads planning pandemic our to all. will some uncertainty think executing a the now. which right physicians to launch, interact. We changed fully what causes launch our it been since it we I your are planning, and March for

there. question short expect trends the how few we is launch, -- early we into trends So certainly quickly it's The weeks have encouraging. The to why get that's a we quickly peak how as say, And to encouraging. to the uncertain kind early get to the of to I real we of uptake. I as will get just we but very are will that vast Obviously, majority quarter, how this just them were over are, time. say, in

Reshma Kewalramani

decade. about question years, the think about for and the think And development, I talking reflect you're if that, your business back in next see the I how pipeline, I I growth just coming do stand including really

parse Let X out that parts. me into

don't CF. to have about sure about making forget we very careful be that We

our means So where about, in to of But we going get some have we up is XX% approval we you XX,XXX-plus to regulatory and patients. of And CF are then, we the there's yet then reimbursements. those patients. with to talk are It not are place XX% that course, heard geographies there's Stuart at but as in which where And you some are TRIKAFTA, to that the all today. and we of patients approvals patients. there where gotten last can future CF, haven't reimbursement, that a KAFTRIO, there serve patients is

specifically is very more more for The minute a patients more want pipeline and this. and months by the not sure The that to Phase go year is to to a now, the Then the patients, And not today. I multiple take really from X company. getting let's go more today, is II. not So from I is CF to years future, few pipeline. more growth through make this non-CF now, in non-CF it's and of this even in treating already pipeline

So I just a results me take disease the let we it's clinical over in shared We this currently beta-thalassemia, CTXXXX of in results treatment. anticipate important. end minute two. underline proof-of-concept through who patients areas. this proof-of-concept sickle one stage in we from going really shared time and and few another their just X program, make points. cell And proof-of-concept In beta-thalassemia sure the CRISPR important summer data year That's is the are the AATD, for by disease frame. when the

that apart. XXX were, and that say, those certainly was clinic in apart, like disappointing, succeed. of XXX already molecules while not And and let's contemplates results apart, in It's expect programs why fully XXXX. Phase half that. years do remember II, not we already the X months and XXX strategy in that's And the our You'll months first something may

on patients in this that to year, going end a that's to volunteers, that into patients, means FSGS, in in the is which go X go then little not Phase IND bit now, program, the also healthy in means Phase to diabetes II in going that's to Moving And type just be II. a in the then in by XXXX. more pipeline, but of patients in

hope pipeline I through X the helps. that And up more areas with this out to few the diabetes. we're and the X in when really in application are in for the to year. coming and and last really forward CRISPR at think already think IND we in pipeline you that calendar stand going the back and of I type the "Where what X disease look I I carefully months coming in XX place, So by CF, a mean good that clinic to is the much really?" even data in, say, look you

Operator

Our of Matteis the question comes from next from line Paul Stifel.

Paul Matteis

fact that in if One implying about would healthy have AAT data release, if that and the Could that? on going long-term you details. they triple price. these that And with seen the the were might have anticipating? that were may -- kind volunteers? compare not lower make disclose to with anything of you to And know what so, mix as of indicated the do I mentioned country in than of you're mind. AAT you patients just AAT, one about operate question were On ex-U.S. think don't how the to outside the But low. of year exposure levels differently? commercial, in U.S. XXX from Did you the study that one then the you analysis you're granular X, PK livers press price that kind or price in of that the you're initially, TRIKAFTA, launching commercially,

Reshma Kewalramani

comment me pricing. on first and I'll question your part to Well, let the of ex-U.S. Stuart then ask take first,

And study to full we're we regard that out groups, an with that completing were have the are the the we that we range have closing in. occurred. across the means of the What and did of the don't From is the treatment of all to see that looking XXX, is that AAT obviously patients exposure exposure the period, is we study. it, levels have let's that all which So and looking lower target incomplete elevations target call access for. study process bringing exposure dose of range we is all in to, in that the data what data therapeutic at we that expect, for than that is dose the

And standard nonetheless, differences one's cetera. that gosh, volunteers differences. thinks but There's about. we Differences of that think But healthy why in one than about medicines, there actually when obvious, in -- fact a things other there are formulations, diet, healthy disease. are has right, the age, you're low number of concomitant disease yes, et Now and in different the gender, is well, PK? list patients one a fairly

So bring certainly data, all the those all we're evaluate as going things Absolutely, we that. of kinds we in about. to think the are of

though, I medicine XXX think and is pathway XXX the II, view full the that to study that's point, important a we're forward and this looking of mechanism. this give is the us to Phase in

who You livers. asked turn There's have no that a have question I in differently the people AATD U.S. about over and there, we medicines. react evidence in it before Stuart, they to oral to XXX,XXX about

evidence any to over I that. you the about Europe. So pricing have of for Stuart, don't in question

Stuart Arbuckle

Yes.

think that immediately. able So to get X types countries to think Paul, the about we to it's the medicine I of are important

where next the contrast, as the U.K., negotiated were. Ireland, the you list market, are Whereas Germany, like is price German immediate access in at So the Denmark, XX the you structure countries approval, because over to and the which of price, final provide immediate of negotiate whilst like months. there, we prior the price, negotiated access there, we KAFTRIO it the countries at for one first essentially price of get our for selling patients

to where more negotiated have going we're forward, markets and prices. more entering moving be we So

negotiated as we pretty in how negotiations. are was, provide they're finalized be price the our because to our Europe a other patients in. prices hard negotiated value really that to we no across But range narrow the same matter expecting will benefit say the country know to products question those I'm compare, and general, because in what for medicines, haven't Your they

Operator

question from Cantor. from line Our of Young comes next the Alethia

Alethia Young

And to kind one another around I wanted ask of AAT. just

of Just that of kind formulating these thoughtful you've clear about when needed you amount AAT very be large of been can you know that. the of kind the to molecules, some efficiency work created? of the talk scientific about done to address you've kind that's to I were going

just more some wanted get on detail to that. So

Reshma Kewalramani

to molecule. David some Thanks. to I'm going of this Sure. give Yes. think we you color how ask about

you insights. I we're And the color. you you're will a that to David had, that answer of nonobvious some Obviously, but you give a getting some ask is the insight scientists really is it's I'll going our David protein. protein think give Alethia, AAT question because what give -- to this as an abundant not to, our to

David Altshuler

the you Thank for question. Sure.

only asking in the molecule drug more excited say, the promising appropriately this approach a to disease why X this that lung reasons disease because question refold excited address liver should the we've than made, is solution the or because has thing One fold, although been is the AAT key there remain and and condition. I lot is of to of the both and can potential so is the X the of program it's you're about the molecule And very important of AAT.

done XXX, not and obviously we've about to do. not our in has cells based also concern X about molecules it's we molecule mechanism on because can And commitment this animals experiments or really and just that molecules all and other the underscores it excited being beyond problem. very So have XXX whether a so much

Operator

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

Michael Yee

Great. it of it's liver. is might hypothesis guess and aggregates actually drug, either because be that that to Two you're some liver-targeted in AAT. part you a there specifically I question can see maybe questioning the of mechanism-based a from around trying in the buy some

actually did related? something it's other So guess guess, better? fast question come there liver-targeted is, drug, XXX? model us mechanism confidence than some a the it's do since in have I one, a my purchase two here, just be study make not feel that's the is, I you related to are how degree preclinical might of little confident it the XXX, for how on of that And at early least And you as bit and

Reshma Kewalramani

is question of mechanism. important you question going bottom is the finding think think why we've PK A Altshuler is bottom not there think XXX. your studies, because I or way ask we detail our But sense this help And recurrent David II through seen that. finding can that mouse about an specifically I'll I'll promise walk David reason the I Obviously, on a Mike, actually has that, going the no David going safety him the There to finding, line liver a understand promise not why this models to primary multiple studied in because that go great no and in time. more I'm exposure to just And way you say any have to we're Phase to reasoning. this we that and or done have to the think just comment in is molecule David? finding. that of for is point. lines I'll mechanism a ask -- be in animal the one pipeline Yes. in believe Mike, liver. absolute our do this VX-XXX promise to then, I I that our David the this don't on models you line, evaluating is don't with is end we course, this that is but safety, we is that ask What to can to on assess that, us through

David Altshuler

that protein just not liver. of And made a I'll that's liver-targeted the a body. is I acts the actually thing think want in the actually, systemic. things through is the actually, about And cells on drug you. clear. AAT thanks before one this be excited the we made of say This this expressed one in we're in have in the that drug is liver, evidence I is a other only for actually Thank to a approach I it's no that is, drug. I go lot is question. mean, And

this on act the does it three say are: think to disease. because premature, come And it the and or that an think you the go I us liver. incorrect the be, drug this said, chemical as on I Most of is back think, mechanism. know X nature, then And question, mechanism though, lines just the we would certainly to conclude so though a even are again, LFT not targeted I'll arguments And liver, to to Even for make that Reshma can't it's is logic the abnormalities of in on sure. through

is is liver then minds multiple chronic aware AATD. studies, AAT we're this for we've And just by have with having people disease, toxicity, a ZAAT but human The studied improvement. mouse somehow XXX,XXX any the mechanism liver this of second models, different we see as compounds, I evidence of being know, multiple with associated corrected different sort we can't that, tolerate toxicity in of no is, people's the and there's perhaps and with think in that third patient And specifically the and liver medicines. not protein

as more we the data for So telling I you just and molecule, forward collect to conclusion. nonetheless, -- right certainly it's we'll we it. think in don't the XXX look are and But next excited

Michael Yee

comment And gone you've and a and versus just feel far XXX better how to the XXX? follow-up, enough on

Reshma Kewalramani

Yes. Yes.

days. early Sorry that, very about Mike. It's

in are early the of We just study. that parts

to details our have a first of don't going complete, is given more to study expect the that some It's 'XX. results you. I we take for that So time obviously in half

Operator

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

Turner Kufe

This is Cory. Turner for on

in TRIKAFTA? that context XX% I And that trying CF modulators? opportunity aren't treatment for just just have do TRIKAFTA, And on the patients get quantify understand amenable of patients one So to then for where just beyond bring difficult to in how the XX,XXX the for the that more ultimate follow-up. of quick or do eligible pockets you CFTR to label are you see estimated

Reshma Kewalramani

want I'm to ask Yes. make use Reshma. a nucleic that to going sure set we want correctly. XX,XXX to over that make acid but patients. we potential. just last is the I The a Stuart represents total sure get I'm those This clear. turn we The XX% going number offer to comment, whom to I is Stuart. that approach. XX% the that for to XX,XXX who frame need The opportunity, medicine one TRIKAFTA like could just being And

Stuart Arbuckle

those is our in across U.S., and year, of modulator, very we across which discontinued just treated vast we those had to the all vast, majority seen have experience strong have here thanks those a over very, who with therapy, SYMKEVI, already the eligible groups, the CFTR now question. a naive for not ORKAMBI uptake. for all who eligible Turner, were even and of patients Yes, those types, on Based were who being

that really identify to TRIKAFTA. on patients found more so I be you can't we it a have initiated And to of is -- date difficult group for you that

hoping TRIKAFTA prepared with of terms with approval we're the done in to of EU. KAFTRIO, remarks, some the In now U.S. replicate KAFTRIO that obviously, of here we've growth, my in we address with the what future in

and secure patients. already patients. for access access be have the the where U.K. rest by able to We the clearly Ireland, Germany are to we trends reimbursement we Europe that important, those is encouraged to early similar And across need of in agreements and

to we in those to a first further reimbursement KAFTRIO groups for through then those expanding as to and to getting patients obviously already TRIKAFTA to to that expanding like we portfolio and then And going mutations to rare don't access to olds, additional get the is patients we that age also deliver, for other aggregate, can total, count been and with who have to XX,XXX Australia, said, and will younger So entire CF label patients able today. younger step there's we the KAFTRIO, patients other the more what you countries In we our to we've if benefit. XX-year such able as like mutations. X- patients addition be to in think over

work know way eligible then, And to to about so the CFTR the are make we but to we addition won't the done, going we for from have hard develop continuing for ability be XX% one. that new patients benefit to go that good new feel interventions we the as try where are said, continue of a to is medicines and to modulator. we're long job that, And CFTR to in not who XX% and to Reshma have sure modulators

Turner Kufe

alternatively, Or to get that plan then could at of XXX seeing trying XXX the as That's we the chance just If Is helpful. we in data. bringing XXX publishing potentially glimpse that XXX to there potential ourselves? a a data data? see? what for extent data preclinical you And on remaining so, prior just understanding benchmark you're to results we're the do

Reshma Kewalramani

is really from didn't and out. All going exposure, we Again, in achieve finding the have we bring the range. the all there we we're questions. data low the that great program target Yes. VX-XXX to Yes. close key as Obviously, that

don't to the going that have high data be I is expectations So informative.

available those I we 'XX. be able the forward is VX-XXX, of to in enrolled think and the trial results. look and that before, see that those to get thing I said expeditiously half as should results. working to first be to are key certainly And And to

Operator

Our Citigroup. of next the Mohit from line Bansal comes from question

Mohit Bansal

FSGS. to And little bit a maybe switching

AAT that the mutation follow-up kidney in not and see relatively this APOLX question given trial So a it short, before small it that because the diseases. many stop implicated did could that of is data trial even we And is COVID. is, is possible mean, data? I

you this broader first like be to go to any indications successful, if have and do those into So plans compound indications? nephropathy were other lupus

Reshma Kewalramani

the FSGS. Mohit, Yes. much so for about question thanks

a XX-week to It's patients. dozen you're longer regard with So trial a reasonably the right, efficient trial, duration trial, a trial. is program. it's or than It so the AATD a

program, it's They're does enrolling the So the both both dosing XXX little early program difference the will we're are bit when just be early that and the acceptable one, bit studies endpoint there exactly a and results there. to too active, seem agency. know, call just agencies. have point out I'll to but the FSGS community that little And that discussions VX-XXX call. available. be a is there's to patients, a been for very a And the and it's program, regulatory there between on you the multiple and endpoint to as proteinuria

eager particularly I'm tell to I'm going David more So to ask disease. kidney you a look APOLX-mediated about at bit those results. to little Altshuler

this people the am David's one I that program. favorite some is tease Although most nephrologist of here,

to APOLX-mediated talk you So a that? kidney want looking I'll more David, do at. that let him tell about couple just quickly we about are you disease

David Altshuler

with in with forms driver. of in strongly other kidney certain many are by APOLX severe there got go human And the us because the first strikes and target go is genetics idea for proteinuric nondiabetic kidney genotype. into very APOLX FSGS, driven targets using genetically populations But to to validated the which strategy of major a is strategy after Absolutely. a is you've disease, compelling as needs. a which over right addition, for APOLX really many, unmet patients disease

risk And out, to that unusual is if would we on so would indications. need. for really it genetic first variety need with the one great proteinuric disease other expectation unmet it's of succeeds able there our proof-of-concept the get and a a that to but it which for we major figure certainly, mechanism and be kidney factors compelling effect And a -- most into of go common be disease

Operator

Our of next the Barclays. Gena question from from comes line Wang

Gena Wang

question sorry, also AATD. have ask And more I I'm one to about

regarding what bit being One did three as help you us between beta-thalassemia also differences a of will that is Anything the mention molecule, more you but drug show XXX small understand cell a And make will small question there my in feel regarding you terms could healthy better volunteers? little possible exposure confident molecule, and sickle program. profile? reasons. the XXX second clinical are that David, you So dose see

we see And the what will that path break plan so would for the beyond at ASH? be abstract And data patients X expect the should we also, also, the announced? And here? lay registration from

Reshma Kewalramani

managed I X up coming the Gena, think you know to get on I in we're questions there. hour.

I'll ask and and really you differences you're that. XXX. question, David David a between -- give CTXXXX the quick tackle XXX I'll And quickly about ask me asking on comment to I Let think to

should expect to you see the from program. CTXXXX So more data

have David, more little do both follow-up data to should in You and you to you question data. have beta-thalassemia sickle commented venue see a of we'll haven't and patients those expect with regard the that duration And be XXX more on? just already in disease. quick cell longer exact of to a on patient bit XXX,

David Altshuler

kind being All were idea specific structurally is being XXX expect I the we not chemical we say than different problems again. same necessarily XXX, And would the saying to abnormalities LFT see about we would meant molecule. particular the specific, to of of

Operator

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

Srikripa Devarakonda

into you whereas have cover your looking device? that on lines those expect This about to time of work is in done opportunities? independent you types of does for them the file of sequence? of left before two moving the tell us programs? Robyn. as they terms you when much think talked you had and for patients can at IND both I program, is do opportunities. believe Can that you different you in about How are a to diabetes Kripa Or question you be Are where

Reshma Kewalramani

about question sure. more to X appreciate diabetes program. bit I the type a and talk Yes, Yes. the the really opportunity little

you've characterized I think it see goal. I X opportunity on shots this as correctly.

in of people will The I have call serve the the And cells of That's the IND that is, first kinds cells. are with in who SHEs episodes. The this patients naked example, program what it, and end be few the there XXXX. the the starting that's could are going clinic that's let's a as one or year. by thousand an anticipate in naked severe hypoglycemic called we're

you and with are Now kill takes the SHEs that you nephropathy, become while can severely hypoglycemic you that years retinopathy, certainly awareness. neuropathy to develop. morbidity has of diabetes when have hyperglycemia because These lack

of transplant type the And who example, one obviously, of people, for X could already patients are immunosuppressive who that's group post So are this serve. kidney are are medicine. group second who on patients, diabetics

program. in the it alone the that But the for the to we cell high naked out But cell one fruit program. IND forward cells we're could I look going the in. go the is population forward to the bring that for and serve the encapsulated program now biggest have clearly, And the population months as the So that's the that you device. by. is to more second look naked about is telling could

Michael Partridge

the take two questions. up quick coming on we're more right Operator, so we'll hour,

Operator

Porges, from Leerink. comes the question next of line our Then Geoffrey

Geoffrey Porges

cash the Reshma, so billion you on in balance $X.X have sheet.

end You're will seems, generating flow free about you or billion the year. cash by $X.XX be it to close of the in

you no development activity, say you plan the huge that about capital? deploy that's business cash had balance $XX middle let's and billion in how amount a your Could of an call that but could You've the on to have cash, have debt. year. on give next of us in hypothetically, you So questions you idea sheet

of you thinking are what So doing?

Reshma Kewalramani

you thanks that's broader about allocation. as question. for -- you're than development Jeff, And and business asking this point capital a Yes. out, the question is

statement, in. also and chime level high to a make then me Charlie going I'm Let ask to

pleased very with business is way We the are progressing. the

believe Charlie pleased at to And how the this favorable turn our specifically we our to is a of think Charlie? is that going strength we best sheet. in. it about the with very of our deployment in are core, balance that. a over really to innovation. I'm position give you And that We of be We sense capital to recognize

Charles Wagner

the profitability, cash Jeff, strength point the course, Thanks, you question. and business, growth out. generate the I the do a Really, of of Reshma. of Yes. appreciate we with lot flow, as the

we very capital are naturally, on allocation. so focused And

the that we as platform Reshma best you've in to Semma be as that As last of seen BD, and have gaining in the invest helped Exonics. to deepen highlights, us and assets is believe years with innovation. capital active pipeline, capabilities over use And importantly, clearly access very us technologies really two broaden the and important well

for The have, point flexibility team we that best And is stay term. importantly, us capacity close deals. do long we'll intend be because active. over bigger you research you specifically, BD to ahead, looking very out, to continue feel to very is for that active. our and to BD the we do opportunity and expect corporate can we the to as drive potentially sheet our So But that the do ROI value, more balance add and strategy and

Operator

comes And our Evan final line question Crédit from from the for today of Seigerman Suisse.

Evan Seigerman

I'll be quick.

have show follow-up, that pivotal Do or or Vertex feedback could sickle So as story? on us of color to what to might in positive very beta-thalassemia. and any to a of you curative the as give terms be approaches a in sense disease trial potentially KOL need at commercial to calls, efficacy I've part just when a cell gotten you registration-directed to

Reshma Kewalramani

exactly I cell the cure tail little essence, and in a cell, exactly CTXXXX patients, that the X the see And being in bit they clearly, the how exceptional. do said, number, -- the asking people and of catch approach. require. I shared disease the while really beta-thal, represent, are sickle will question, didn't the regulators in X registrational the a functional in to summer those about duration we've follow-up from call sickle very end of too small results what beta-thalassemia of patients. early for That It's but you're think and we in number the

We is, multiple also the have like have secured with conversations that regulatory and us Fast that opportunity to helpful. Prime of cetera, designations very RMAT, And et course, give Track, regulators.

the in think So that the I that patients. are doing ones, right studies exactly right we're the

able a come patients and side, let to beta-thalassemia, for regulatory for the beta-thalassemia X back nonbeta forward after and those zero interactions. cell cell patients really zero, through packages VOCs mean beta looking have sickle I'm what who very In those multiple what I for know over bit you specifically little work patients are And will And much the are that you with like the year So zero who side. we specifically be look beta more to sickle period. time, in is example, to both to with severe we opportunity to regulators. X

Michael Partridge

tonight. We everybody, appreciate team, you, tonight be for Please the you on questions, to on Investor us. The reach Thank available We'd have additional joining help. Okay. to is Relations and call the us out it. webcast. if happy

Operator

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

may disconnect. now Good You day.

Vertex Pharmaceuticals (VRTX) 29 Oct 202020 Q3 Earnings call transcript