VRTX - Earnings call transcript Q3 2021 | Vertex Pharmaceuticals Incorporated

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Michael Partridge

Good evening. This is Michael Partridge. Welcome to the Vertex Third Quarter 2021 financial results Conference Call. On tonight's call, making prepared remarks, we have Dr. Reshma Kewalramani, Vertex's CEO and President, Stuart Arbuckle, Chief Operating Officer, and Charlie Wagner, Chief Financial Officer. Dr. President will of join Vice Therapies and Executive Vertex at us Asana, and Cell Chief Genetic Baoxian for Q&A. you call. that listen recommend access slides webcast We as to this the you This being call recorded. is available A on replay will our website. be

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

Reshma Kewalramani

very Vertex's you pleased with to here the Thanks, of quarters through Michael. three progress I'm XXXX. be discuss to tonight first

this our our meaningfully advancing cystic our with and pipeline. in increased CF in the leadership we've CFTR modulators with approved both fibrosis, During year, programs

We have CF. delivering these beyond results. R&D expanded our now are accelerated pipeline clinical programs And and

performance financial high cash significant flow. We to demonstrate and increase have continued exceptional growth revenue, margins, in with operating in

and year-over-year. performance quarter are including me for more total billion. revenues the now recently growth $X.XX in was guidance product revenue has growth children third KAFTRIO OUS, again from were billion ages on most increasing and $X.X of U.S., between XXXX expect France the XX% including on commercial these XX each our Based a to X representing driven Let TRIKAFTA launch X uptake be perspective, $X.X revenues reimbursed, elaborate where performance strong and in to This product of we CF, Italy. strong and been points. Starting by with billion, the the in almost

continue will growth the CF we that in forward, As we to expect years show ahead. our business look robust

CFTR As on made programs there progress yet from regimen our patients triple our and with strong modulators. We've thousand class, the Based treated with as in of pipeline -clinical potential next pre VX-XXX XX not VX-XXX We've to pivotal Both are results superior that benefit important clinical running and well. R&D Phase approximately tezacaftor our and Both patients. demonstrate X CF accelerated combination TRIKAFTA. the this year Studies to trials be CFTR head-to-head of for program stopping modulators. are existing studies and the there. studies. into versus we're up are enrolling and CF this

identified into our translating XX We medicines. promising more in in have even regimens labs groundbreaking CF insights success, proprietary biology on years building our of

We sweat regimens goal will are long-standing to confident bringing carrier these patients chloride. our us CF to all levels of allow of that reach

we fully increase patient we decreases where the in for weight and we in longer-term, pulmonary approval trials, the clinical field very What that, the find mRNA delivery in significant exacerbation, CFTR the the I past has report benefits, in deliver X cells. we quality been target pleased We've life. their data tens always this in We're years increases mRNA for position will plan thousands benefit just close truly KALYDECO, in appreciated X for everyone now clinical year. ppFEVX, am a now of TRIKAFTA. the development of with and underway. to in Based in non-human data lifetime. then of the and can breakthrough first patients And mark delivery X in and older. a determine approval the patients us we from and provide first we that XX% and that The the And with are note just have reduction CFTR in years anniversary CF KALYDECO, able will mRNA epithelial regiments, safety our in of months IND particularly not a mRNA and of X average transplantation increases that levels high approximately an since have and IND vivo, been is to to real-world the the vitro. a of a CFTR database in nanoparticles lipid kinds function results, now for restore in in Moderna from of For delivery decline function start been benefit these Phase primates. out synthesize of epithelial ultimately after no medicine. remarkable that pivotal human who years now showing enabling of over file who not rate from CF, and therapy our outstanding modulator, in cells people on medicines rate lung an data. we it a modulators more trials. CFTR to but vitro, approach. from first We we're of to bronchial physician CF And and on cannot marked of patients breadth protein challenge XX% genetic years CFTR a U.S. CFTR of an month studies cells CFTR could few have our eligible these that I modulator. of on last follow-up choice working bronchial appreciate on some now clinical with biggest constructs And follow-up includes time patient including have short-term analysis for any reduction perspective, an through of like chronically CF nebulized for XXXX. made mortality compared were in this From treatment. had already that, demonstrated emphasize now, significant for years have that full of lung for is efficiently in of we TRIKAFTA, data XX% long-term mRNA to to the of function partner To Moderna to the our is X to I'd therapies, CF, where of XXth the we our With

our pipeline Let outside me turn CF. of to

with quality itself cells X proprietary curative First type has the to is approach. lives of X pancreatic the data diabetes of of cell-derived, long-term been known. suffer producing of cells. to these differentiated type millions in cells still seizures sufficient with that treat production. be decades for type potential allogeneic of X and process fully disease. restore first program were cell islet resulting through insulin shared. a Grail to with combined and injections islets industrial multiple could durable diabetes. pancreatic and immunosuppression Holy this vascular treatment from islet improvements quantity diabetes across can VX-XXX Hyperglycemic results X quantities routinely to patient Severe remarkable robust, of cells our of in that using our this recently Therefore, islet The with the in problem truly of autoimmune with we death. diabetes day dose well damage destruction clinical clinical Daily can and are demonstrated million patients episodes studies programs these we the unfortunately, data developed more measures than to for target have patients, X/X of unresponsive, function the X.X the treated rapid, observed islet with from has used make and standard a islet the stem transplantation, associated the people this unprecedented the It The from disease, severe of patients Early mortality lead that type premature single the and clinical but infusion replace XX. have at saved has complications Vertex insulin serve cadaveric pathophysiology been even substantial The the cells

significant following in levels in in stem response XX% exogenous infusion, glucose and in basal derived increased a insulin appropriately insulin cell hemoglobin of XX secretion blood as Our requirements. AXc, islets there the despite was by And produced to stimulation. glucose a reduction insulin reduction measured in days

side, working we're programs. starting VX-XXX device program, results system. enabling In these of And for our with to already file of same product each studies hyper-immune evade protection Immunosuppressives. the the where involving proprietary plan you follow-up. approaches denominator we generally very type to Standard therapeutics And In IND a our On was potentially -risk program. cells this high. in these have immuno common and gene therefore, these our to product, de are And Pharmacologic with reasonable curative the X VX-XXX our well-tolerated. that using are cells X program, underway In and the is reasonable alone solve These urgency across diabetes the path therapies, a safety cells. the islet rapid other of is programs. is IND It are immune the designed the we're program clear amount that The it mind the program registration this in on these the editing cells next for of produce potential selling number that of for can patients gene these XXXX.

will a the vivo Moving also target this physician XXXX. studies. trial Based therapy out have in and our who that editing additional XXXX, pipeline near has in onto discussions is been submitting and thalassemia. by number We've approval completing in now eligibility the we've XX advanced clinical results. strong regulatory on CTXXXX sickle-cell in these we to clear beta our our CTXXXX regulatory one-time clinical outside and on designed high. are these programs we to-date, a anticipate have momentum. year-end as of disease data assessments It and accelerated sickle-cell patients now CTXXXX And the the is continues beta enrolled for be as presented filings CF We CTXXXX. the for both and of of have curative trials ex our out thalassemia closing gene each based most stands and in patients target term beyond non-viral disease is example interest how approach clinical fully month. patient of enrolled

CTXXXX our our the patients Onto we on will proof-of-concept fully and that a VX-XXX, results will comment form this the FSGS medicine. study, remarks. confidence focuses that on where mediated commercial in preparedness with X from quarter. Phase VX-XXX enrolled of next be This launched will have is APOLX. his Stuart is X Study high have by Phase of We progress

a and target, the a our as broadly and more that of to a VX-XXX new strongly in or disease is FSGS. number having kidney Prevnar inhibition the vitro goal the we see profitability as program APOLX human high can validated establish across assays, success. of AMKD non-diabetic vivo of kidney Based beyond in performance on used be In disease. mechanism APOLX-mediated APOLX-mediated Our genetics

first-in-class represents study a is APOLX-mediated for population. us If successful, it the would APOLX-mediated demonstration a to the In this one but Proteinuria presentation pivotal population, efficacy X is disease. program represent with -clinical not this safety the of later of of of FSGS. Some subject Society this key our pain the of X from program. would And place propel proof-of-concept Phase are expressed with AMKD week. is and we're on into Phase words Proteinuria of conclude Nephrology accepting, American development the data FSGS marker reduction taking in people approximately that XXX,XXX I'll the homogenious in pre endpoint kidney the which a study, an this AMKD. to kidney meeting assessing includes, regulators limited pipeline in a discussion few In disease and Proteinuria. VX-XXX relevant the openness our is clinically have total, at

and And We validated. lead positive that these confidence over The acute neuropathic review and X. it underway. Stuart VX-XXX studies ranging molecule our X high turn are enrollment the the is of three in have well NaVX.X drug like for program, XXXX. - validated acute, for dose has now and the to we high in With in we're target X. bunionectomy that, including VX-XXX with abdominoplasty, with -X also commercial in third, genetically very X studies I'll selectivity pharmacologically studies is currently properties Based Phase proof-of-concept in data pain. NaVX.X reasons: main key our musculoskeletal own NaVX.X the on expect looking progress. Phase-X potency. to pain, from progress, QX

Stuart Arbuckle

surprising $X year, Thanks, this reviewing increase billion, by the quarter XX% Outside billion U.S. compared driven nearly reached were revenues Reshma. to progressive population. driven the the million, last $X.XX the rapidly, strong XX-year-old Italy. the major particular, TRIKAFTA, of recognition KAFTRIO launch of the in the our of X- profile the were is of I'll including markets XX-year-old in KAFTRIO of increase treatment ongoing third revenue an in prior launch XX+ The not by QX of the of $XXX and medicine achieved and is the performance of CF disease. performance more to where to an the the progressing than QX. to importance reimbursement begin X- the a medicines, in which In which given population. U.S. France this over by year, start we is off in year. launch in early revenues relentlessly of XX% X the June

of signed TRIKAFTA intent Canada. covered then, in to have of patients for multiple access now reimbursement and XX agreements reimbursement TRIKAFTA. over and since a XX% letter We of also reimbursed And we've insurance patients public achieved provincial some by government in

regimen. the And U.S. combination we've value countries achieved importantly, than continued have more agreements reimbursement at that the since reimbursement reflects KAFTRIO of approval. over outside the XX triple achieve We year TRIKAFTA, to in high just X levels

loss medicines All showed previous remarks, amongst The show contrast, important the abstracts, week function lung during at long-term to presented on from any of illustrate, XXX the Conference eligible who for of first been modulator is patients important rate, These In a Because conference to of follow-up, for an North which the for data show X the for complications of shows KALYDECO Real-world CF As Reshma medicines medical data XX% not no This for XX% her mortality field. on American in TRIKAFTA patients no fully an our is of profile rate These in CFTR of enhanced medicines at data a address the being future average be long-term how And there lung the landscape. loss for an tonight. long-term lung long-term of sort XX open-label of of of also have the milestone are TRIKAFTA, competitive our lung transplantation by and mentioned they in the ongoing, CF continues patients for study declined. of data the has these more and the weeks extension data. our data follow-up. to-date, were the other a slowing follow-up. function community. function reduction years reduction implications are data after treatment. compared and also progression very important disease. important the several

to compete raise the effectively. be in will terms they As bar, of what required

opportunities pipeline Now, beyond in some of turning other to the our CF.

transformative to we CF leverage which in our next experience commercial capabilities, Our provides medicines. foundational to commercialize will be of wave able

Reshma are regulatory submissions CTXXXX, the XXXX. As planned for mentioned, of for end

facilitate the to a patients thalassemia for role a be curative sickle-cell in to diseases candidates using developed with where severe launch medicine immediately current in of approximately And the thalassemia ensure potential so, and are approach our with preparation to this disease, disease the Which a one-time transfusion-dependent our activities consistently treatment sickle-cell for research, beta patients U.S. would disease their we XX,XXX bring market U.S. expect see including quarter able patients Consistent published third markets, deep globally or the in internal numbers patients approach with own the that, one-time of physician play sickle-cell curative understanding key will sickle-cell conditioning patients. a approximately Europe. with referral We've upon thousand of We the XX they patients. surveys of and beta the regimen. and centers the and with are well the to is treatment severe are, patients these good as indicate disease estimates potential journey line to underway approval. a for to with CTXXXX that

focused areas for on key three preparation launched CTXXXX. are We of

people We've will who the launch. people. key the support First, of many hired

the days from supply patients we the CTXXXX. earliest ensure launch. X area Second, manufacturing. day on believe can consistent an the the we our work To have of focused product is from to large high-quality on our will we on and of This number of medicine benefit

trials. for And are understand manufacturing sure using we experience. our them listen, Importantly, making for and really and clinical their processes are same using we that the commercialization third, we patients, sites

CTXXXX. So, as launch at treatment need with and with they information, we resources they consider can them the provide support

underway, a our the still days opportunity large. very X type billion medicine more today, a this data prescriptions the And acute a day, pain. market. highlighting of of opportunity the That's turning would you than has is there. In market pain typical market studies light that for pain in With branded approximately billion a pricing accounts new current treatment the thought VX-XXX, year in $XX the being Now I potential. medicine of diabetes, Acute to treatment also it's large which days of alone. even despite U.S. remind pain well X.X $X a is of for XX% portion generic, unprecedented takes multi-billion-dollar worth I

Type than the a to people insulin. which and in produces the islet populations are is start alone. is X me X.X disease due cells, It more affecting Let in consider. X auto-immune little debilitating There with and disease. Europe patient no the body pancreatic destruction to U.S. million of diabetes disease a severely to life-shortening

to made who VX-XXX. This needed. so in least X candidates with First, are immunosuppression patients patients XX,XXX this type And for the type for there be patients are the about XX,XXX Cadaveric would standard impaired would those islet organ type propriety on diabetes, and potential people intervention this And type where cells had with and severe in is at that are diabetes life-threatening. the approach cells pancreas these broader hypoglycemia category. primarily benefit device give of of of X or then encapsulated sales transplants positive not in sense and X characterized and group for alone risk have immunosuppression. hyper-immune the control in the first plus already difficult have transplants, of There who be events of of people in approximately previous diabetes forms whom who value XX,XXX profile and our the were diabetes for who whole immunosuppressive Europe small There severe candidates up U.S. severe awareness secondly, is with already enough be population, the with disease. performed, therapy. are hypoglycemic of are patients kidney severe, this albeit by can in There patient are category. numbers some a

pancreatic per minority For a the even illustrative $XXX,XXX for transplant benchmark patients for a approximately treating as would cell-based in of if you a price multi-billion-dollar purposes, the of treatment, represent use U.S. price the eligible opportunity. the a patient

in the plus Beyond the same progress our Vx it turn I'll which in the our X the I'm in type X.X these U.S. the cells to summary, CF with system, bringing proprietary Europe. and XXX cells unprecedented about address for we the recently many population, our device around reported clinical patients protects patients device to data cells program, encapsulates that, immune pleased over which continued diabetes with pipeline. And our into and medicines the from excited opportunities world, that more million In Charlie. broader could the

Charles Wagner

record of eligible XX% track XX% revenues TRIKAFTA compared quarter represented most the third have quarter $X.XX to of patients strong increase Vertex's nearly of revenue Total third XXXX. were to TRIKAFTA. In quarter Stuart. XXXX, switched of product as third Notably, a Thanks, long-term revenues billion, continued. growth the

for grew prior the quarter U.S. continued driven Ex-U.S. U.S., third strong and revenues included quarter in million KAFTRIO. the the XX% $X.XX XXX for year, Our revenues over outside uptake billion the by

and in combined and stage by SG&A were quarter investment compared to for programs largely million the quarter Our million pipeline. third R&D driven $XXX our $XXX XXXX, clinical research expenses our of third

continue R&D late-stage substantial regulatory We and the mid further to make pipeline. expect we and be programs investments progress and across will our as our clinical advance

of combined second the revenue with in And with Our margin growth disciplined year-to-date $X to a cash. we translates in continued with strong billion profitability, and our OpEx, revenues growth XX%. operating ended quarter in

guidance. to Now

growth our uptake we increase represents continued in revising at to new out-performance, range We reflects launches. midpoint. total XX% as the for billion. This Year-over-year, are of this billion $X.X product rapid again with as XXXX the upward guidance have the well guidance $X.X seen revenues

in we with approximately France in are the and patients highlighted, very patients Even Italy U.S. the rapidly. this Stuart year, left the the uptake in treated to the XX have As our XX,XXX treat and X launches outstanding CFTR growth modulators. number to proceeding with of

securing we successful executing younger of agreements record majority we the be track confident vast additional markets, patients new of to proven able that expanding our to these with launches Given medicines. groups, age reimbursement are in our will reach and access

full-year And term. growth XX% for XXXX to We this are continue to X.XX maintaining year XX year. non-GAAP we will for range to our to our conclusion, for X.X rate, guide of In non-GAAP tax XXXX rapid a % OpEx guidance at be another of billion. the an XXXXs. in Vertex IP our ability in area bar and growth TRIKAFTA for in continued a very that medicine that our and trajectory the high CF late safety and this lead extends confident sets we're efficacy exceptional therapeutic to with to over long

CF With as in our VX-XXX meet to regimen the other patients. exceed tezacaftor regimens of data the with the ahead of potential beyond levels The as we TRIKAFTA in or achieving performance to development. is in any only on VX-XXX that years the CF, are and genetic of way next well our making carrier for is clinical we're fulfilling TRIKAFTA, regimen progress the shows all well vision therapies emerging profile our for regimens

medical CTXXXX, with CF to community, well growth. for of business can VX-XXX strategy VX-XXX to will data We're We recently, the Our data Progress margins shareholders. for drive revenues and value both innovation. trials growing we Vx execution XXX With will confident demonstrate X We of that continue exceptional ongoing we flows our is at patients and top in with results our and in FSGS external innovation reinvest group, we by advancing in coming additional external you delivering. in to look as create as cash forward as beyond sharing pain strong acute Phase deliver APOLX-mediated internal more and continues drive to the as to the Phase future in pipeline and anticipate in the and peer to our for completion for come months. the near-term. data the X continue investing

questions. now the We to open will call up

Operator

Instructions]. [Operator

Our first Salveen from with comes Goldman question Sachs. Richter

Your line is open.

Salveen Richter

Thanks to you're afternoon. you Moderna, delivering with your moving nicely collaboration with With the mRNA X with cells? recognize questions. on Arbor secondly, forward here. use via my in mRNA the could And with talk taking X that explore expanded then I with could just the on about where on editing plan outlook maybe Good Type fits -- of collaboration gene LNPs; for touch you into pipeline regard development in the to base that diabetes, the lung you stand and how that here.

Reshma Kewalramani

Yes. this hypo-immune think the I type program another cell is but and with thinking how diabetes, Hey Moderna mRNA the One with Two Salveen, Arbor with Reshma. question to X and programs. in collaboration regard there. we're specifically our questions on about about

Arbor. break parts. into collaboration X me type with And up diabetes expand Let beyond two it even maybe a bit on the I'll little

Moderna. with start Let's

and prepared breakthrough for cells, about here. really bed problem breakthrough for to It's the And need last the to the this translates exciting have qualitatively delivery. do only to sweet back on things. whole made HBE so the a news make HBE it's simply And With have And only these that We my say about Moderna. to workhorse it's the it's forward are been It's you the regard the I mRNA mRNA we cells medicines the CFTR and step recent a do for we that's HBEs field with quantitatively really Patients you brought in the as not about workhorse And for any we the the XX% program -- that last X don't model make excited shared with of when also have -- us program the CFTR about in very the I for remarks constructed It's that in but about protein, right? already. so for and one, because that's then it's delivery, they're cell, these cells. and from about the today. a think sure that bench side. been really vexing been what that

mRNA a is ago. number the second years The of itself in construct

We full-length Moderna, Company the this that able best to have deliver honesty, last And months, in what and demonstrate all been able been in CFTR all We've express functionality. space time in for do, struck some able over have up several its mRNA, arguably to that vitro now partnership of with using the we we protein, to is mRNA. can been appropriate to demonstrate LNPs HBE. But nebulized cells. and have we do the the a

do So do say can And No animals small bronchial deliver cells no to in the else to done bronchial this. to we've to this and that construct epithelial to has large. that is cells. claimed we and able the this, delivered else see and mRNA we've one them to certainly epithelial one has been

cells. are use start the underway, there second enabling Immunosuppressive. are expect the year. that already are the our we the diabetes, studies. I those are big type So On XXX, itself Vx device, advancement us themselves with allows cells go in and to next and Those IND X off-the-shelf go In GLP underway to plus that's and do Salveen, there IND already the next studies first the then In cloak mechanisms program simple Pharmacologic to program IND the year.

for going program immune to editing, a plan Bastiano? bit cells, same hypo on The we to using I'm ask cells. little the or the hypo to to make program. and just for comment more use third use gene cloaking, Bastiano example, immune is these

Bastiano Sanna

diabetes the is more cells Reshma complex -- is type to the X the them the cells. X is Still the for Salveen actually present problem better is as allogeneic cloak specific immune from type in to said complicated the general, call Thanks, autoimmune Reshma. cell, -- system an because disease. Thanks, question. Absolutely. a differentiated has higher Healthy the challenge the sensitive fully a diabetes

MHC from we're the decades. internally So, of are past types cells X Class been allogeneic and that care to where as autoimmunity. and on allogeneic people is X to allogeneic we all and that out the that of the the we course, also that from that internally. be something course, possible, both worked will the cloak of And is the immunity: And has most general have of knocking And mechanism That rejection go. through the fairly we X rejection obvious take place it's collaboration immunity. exploring usual doing sure out

Salveen Richter

Great. Thank you.

Operator

question with Jefferies. from next Yee Our comes Michael

is line open. Your

Michael Yee

differentiation questions; you morning X, of a that X announcement is -- Phase good I had clear it's to how it context moved good for and in new CF about Trikafta, the comment in your afternoon. context you're the data this could had that actually, context of program you I in confident X day. your data Hi, much into there's long like in quarter would we the out been the should and be. and but it is fact also that press you differentiated everybody release how coming that how put with it the the knows competitor a of interpret from

The second that a question earlier commented question you fact around had the is more M&A about year. this

prior areas in about this that and fact, that quarters. recent to even In so much Med the commented Thank view want you Rush just did you refresh earlier like that haven't I Huntington's you ed fact commented you. year about and really like

Reshma Kewalramani

Michael, good Sure. afternoon. Hey,

leader CF. And profile, allocation have regard clinical risk to the has in model disease. disease, the has in I'll stand else and kind one. have competitor the is in clear, results no Mike, and frankly, just that transplantation, decline, data benefit lung If field. where back XXX asking XXX-XXX at these program the capital specific decade TRIKAFTA best look, set XXX, a that CF data long-term look that the comment And first you demonstrated even plus. years test chronic capital and the of in and the Make with -clinically prepared on year, high to the we when CF over tomorrow pre the ahead up to efficacy we've medicine Tez, It's medicine a which think CF, earlier than Trikafta. Tsikata can TRIKAFTA. They're very that to it looks short-term best X TRIKAFTA. program a we rate do as Tezacaftor. of anything The to and TRIKAFTA be is as in to in the few to for take HBE cells about that form that going for data. then XXXXXX for of Mortality, over benefit Vertex. long-term, a ask Let's expanded in serve the are of that [Indiscernible] So question that but people this, even with I then about form bar. recent an born We of the really today obviously the leadership Phase bench-to-bedside a shared the than lifetime. It's more Chronically, the incredibly need from CF I XX% last to data ourselves extraordinary Stuart to high his only the have this about ability With provide going Company that of children we allocation, TRIKAFTA. Charlie, you with you're long-term to / about talked are years, have established remarks. And the medicine it, medicine an that better from translate mistake come And first with M&A I'll VX-XXX in with you're the potential

Charles Wagner

both a for in across Thanks, medicines disease that are CF is for tremendous greatest shareholders. have transformative of this hope Reshma And that it's shown other in clear we do value And that to that represented in industry. serious for question. and very the Listen, disease patients thanks Mike, pipeline. to innovation number broad and of we for to leads creates the value our innovation that that us driver areas

focus when externally. in internally So, both innovation, on primary comes reinvestment and capital our allocation, is to it

in and because continue we've a innovation the for are numbers out looking top the Semma a can couple more specifically never to over investments that And in be so, smart of than also we're but will the see that today. there invested not You would internally pipeline and of types on we the type reinvestment the we've see made of from some are call standpoint, specifically the we've deals benefit not years. CRISPR of of capital commented priority. can I certain last And many in others. deal. you allocation external

strategy move and disciplined continue as assets and fit looking and those, that with for be research are and you we see tools are our well quickly We and aligned when that an technologies to expect we'll we identify opportunity. can

Michael Yee

guys. Thanks,

Operator

Nader comes Our with Phil next from Company. and question Cowen

open. is line Your

Phil Nader

X Good taking questions. our evening. for Thanks that's X commercial, X from us, okay. if pipeline.

and quarters. On and what and the the then APOLX-mediated the based France, this proof-of-concept give to And molecule be of that you pipeline recent we're you the like just data. disease the some then the commercial, sense the quarter those as growth the thoughts into the population continue second that kidney XUS you where growth you. future we've Thank FSGS, broader and X- this you hear markets to to XX-year-old type confidence highlighted narrow date in Italy, think most on to would initial gives from on what for curious whether could your impressive. population penetrating of U.S. quarter-over-quarter are the seen and on go Could FSGS pretty was Canada, you us approaches,

Reshma Kewalramani

good Phil. the FSGS to I'll I'm question Stuart on a And about Hey, back bit then and Stuart? how ask CF more you come Yeah. we little tell the and comment to going see program. growth. around afternoon,

Stuart Arbuckle

I'm comment to where a It's growth are of hard you going to But our I'll specifically today. thanks Hey, guidance in us revenue on this sense much Phil. that led trajectory. our increasing of Yes, be. quarter-on-quarter to you enormously give kind to quarter what yeah. and proud for execution third on growth rates commented question. overall our led the that we results very our the And, in

got for said, the further, to in you we approval June. XX-year-old TRIKAFTA X- As

quarter So just prior starting. this to

like the underway. most agreements and is in and that reimbursement new there secured What level And you in the We've every TRIKAFTA we've exceptionally the XX-year-old and market in Kaftrio. U.S. with for overseas. where launched line here KAFTRIO Trikafta, high to seen in X- other also important are We is launches Canada. France, that. enthusiasm markets can in recently Italy also I of the tell

are a months the launches. to off there's those So, first Obviously, we're strong start. few in launches of

CFTR what patients ahead, with we've our XX,XXX overall. only who still I So, see CF medicines. all yet for looking is really launches the of patients benefit to in about We're terms had despite and of from modulators that actually the benefit medicines, could patients about treating today Kaftrio. fact go to Trikafta do half And successful specifically, who from could

Well, we're Now, in you might the who where launch patients patients reimbursement, in countries are patients? yourself have ask those where sequence. early we those are

have as younger described, I most the regulatory we with where younger reimbursement just countries also, have to and in XX-year-old. age markets to for X- in we're world even Outside secure approval KAFTRIO now, yet be don't but approvals agreements groups there they're the TRIKAFTA of groups, have of we have we and ORKAMBI. Some where yet done KALYDECO with down and pursuing approvals an the going age U.S. to we obviously

So CF I given our substantial and our going until approvals, a have that which to we're respond the modulators. those program, next in franchise we the the prospect made no of has decade. get majority track great reimbursement, growth CFTR don't vast successfully over of the sober medicine see and record XX,XXX And doubt we of patients mRNA for getting I the today, to patients now of launching, years securing to between coming for announced then XX% progress as with who developing also our the middle

And you. got go in Reshma to So, to substantial I runway yet CF. think growth we've back

Reshma Kewalramani

the on the Still program. question of VX-XXX

Okay.

one, really that for confidence in and on see proof-of-concept X I the high X reasons. have that I Phase So, study,

APOLX-mediated The first are genetics disease for kidney strong. human the is,

are well the have fact, have X target who therefore, poor have really strong. is People kidney who In they outcomes. Proteinuria, very APOLX They universally And Allele. have disease, validated. exceptionally

studies vitro animal third of strong good selectivity, and drug-like vivo properties potency, good find as we And seek. studies, and VX-XXX in is in really vitro well is in second We the in that our all The we've studies. has proteinuria. as in reductions the done that of portfolio

I nephrologist, say, renal so, That proteinuric the -- a about It's very aggressive who As let's for this disease. I is But studying kidney that. of FSGS, if can positive mouthful. Phase broad that what tells we're a now we be APOLX-mediated is Phase And the primary the FSGS. exciting, XX,XXX disease, have small form XXX,XXX first disease. have kidney is you a reason: molecule following very FSGS, find proteinuric opportunity that. the FSGS. potentially X, know There's X it's in AMKD but APOLX-mediated that and time, that a people therapy of kind APOLX-mediated really people a that really for found only about interdicts study on who broad market. have X Not for in APOLX

would then we consider full what the with XXX,XXX. So, to regard successful. And

Okay.

it's burden So, FSGS FSGS, that population. allow doses It's studies criteria entry of in APOLX-mediated high proteinuria care therapy. are stable the such of standard of severe Phase a and with X the right? very And we a

already on So, these s, steroids. immunosuppressives, and s, are patients ACE ARB

that in of in impressive. disease improvements So, of would an of proteinuria proteinuria, with kind in that patient care, already standard aggressive double-digit be high population with levels

be only Now, would not higher important, in of better would that not before. seen course, but that proteinuria, improvements we've double-digit is it something be

Phil Nader

Thanks for questions. taking That's our perfect.

Reshma Kewalramani

Yes. Yes.

Operator

Capital. comes with RBC Abrahams question next Our from Brian

line open. is Your

Brian Abrahams

in range? be a quarter XX% patients the mutations, terms down half-life who be the lung what CF frequency lungs, combination And human in lungs thanks guess, one have might don't you line primate questions. about administration how would at time translatable have to the type mucus more love of for CF. the of, other of non-human the opportunity We'd nonsense you look but learn my on for to I of usable? program. see this might any any Thanks. Perhaps for congrats the guys, layer. what of with and I'm in the Do And Hi curious taking resonant to given are for general sense especially the mRNA

Reshma Kewalramani

question a there, the there there's There's few cells the the in about Brian, questions so in XX%? we to in Okay. really program translatable think how let the about and another there's important different on mRNA it parse is there right and question do how model it and out. questions then me targeting a about the dose

I dosing, not specifically small the talked and very of studies part cells. dose targeting very, the in many most of to in have that my been selection, and good important very can assays the about are HBE the tell have we this relevant that the I would the protein large cells. diligent the making the others right schedule deliberate you: why feel So, but I'm development insights mRNA that's and going appropriate say in dose expression what are which I gotten sensitive, bronchial animal here's the and mRNA, sure to cells. the about and about with comments. to And and and be and vitro, are the transcript that targeting commercially drug epithelial right The that's area, cells, in circumspect I construct about the the

to a this we about the is question Now, other I actually way. systemic down at only extended lung the with would regard think a look be XX%? not it's the do line, other it CF to could disease, the disease.

systemic molecule And CFTR so, of benefit. the of correction real get is the value small you that, protein

molecule. our ones we're opportunity year, could obviously only that, and are bring who mRNA I with a the I would the setting the We're the to our last if the program clinic therefore next combining in of -- XX%, it's successful think benefit what to So, tremendous. with do is, the last and we in XX% the contemplate when small mRNA is

Brian Abrahams

Thanks so much, Reshma.

Operator

with Karnauskas from Tuist Securities. comes question Robyn next Our

Your open. line is

Robyn Karnauskas

Great. question. Thanks my for taking

quick ones. X So,

would the How blood in be it's you they getting but what because also by the how I work? for they diabetes. blood the below the meaning you into devices be had allow the In them in talked device percentage the does How interact them how might it from put think it the these you. Thank system, to question developing, might to was the vessels vessels about have interaction chloride And to does new work function? interact with device cells function? you world, in, is a protect and for of for How bucket. device. go your vessels the -- second then the able levels with drugs form, that with liver the during the the that liver real attack blood that immune benefit sweat if only to stability. normal I not lung wondering are and able So, to are just [Indiscernible] to range cells from population their not does

Reshma Kewalramani

Type next Yes. VX-XXX And think X device. separate about in And Robyn. we question modulators. about test. generation XXX different X There that the and to then diabetes CFTR the how there, of are X expand questions maybe a about I'll

is chloride, in in that of clinical improvements medicine first significant can CF the treat see you get we with program, benefit it improvements do Let's KAFTRIO this, know weight, has ppFEVX, trials as let's -- of CF large and what And really extraordinary numbers then diabetes Type Robyn, double-digit and the efficacy. X patients, in to next. we'll the in you do in TRIKAFTA of in up sweat people XX% saw quality life.

all the see going we challenges made. XXX experiments Both in device. already patients foreign second not we to the challenge the important clinic. be fibrosis. our program, our patients are and program do historically our we can in carriers HBE long-term on goal relationship and in our to to pre XXX real of but the devices generation with the identified to what we with this that better I'll all -clinical with another ultimate of is in molecule but what sketch to CF reason are to device XXX Tez comment, goal quantitative we been reactions here has us problem vascularization. have cells breakthrough ask carrier that The have even potential to dimension. TRIKAFTA HBE getting unquantitative And the With has CF, tell or field we've But of body in to like Bastiano to with that The regard they're really no disease. levels. thumbnail those what with And device get XXX, these see tackled and you that been Tez quantitative do than has carrier is to that, XX% looks because we what levels what And assays the is get of what that is I almost manifest do to want of Now the our chloride. sweat trying

And we the what fibrosis geometry do let of us materials allows the Bastiano comment foreign body have is do that device we really configuration I'll not about is we to vascularization. reaction. excellent what kind and have. or the to So, Bastiano. that see of effects and the see the

Bastiano Sanna

the design has completely that just the of novel clear described design with with will exactly everything device materials, that that Thanks, causes It others making address to to different specifically different Robyn configuration rationally start by is design, device It's failure to different said Cells else field Reshma. in body and the field. from the to been And body, wants fibrosis in in geometry, maybe made is vascularization. allow particular device reason, this beds and in like minimize proposed Reshma the really of on called and vascular the that integrate to is response foreign top encapsulation Device throughout for allows to which form bottom. the

serves see models not nutrient. and neovascularization, opportunity we insulin rapid our pre it in we we for to that see evidence immuno to glucose, course, source robust -competent the -clinical oxygen do even see have the response, Allowing protection, as by what a immuno installing that, in animals. of we field. competitive be This responsiveness, we a And always So distance from in of those think exchange.

Robyn Karnauskas

Thank you.

Operator

comes question from ISI. next Evercore with Our Becker Lisa

open. is line Your

Lisa Becker

the question. taking for wanted I Thanks Hi. CTXXXX. about ask to

data you what how you're as kind lot what of, -- have can how to given end data commercial a the in the at kind doing stuff. that many environments year, your treated can U.S.? a you'll ask of commercial next patients like about file of you on many year per question also describe all maybe talk and then I just when And can the much how want craft, of So, and hospital patients? be that you follow-up for --

kind how -- and time at launch, think So of then what that how may that should will of you. we like, Thank evolve what about look overtime?

Reshma Kewalramani

you're Lisa, we asking see launch think CTXXXX. commercial I about how for

Lisa Becker

that understand we to in fact, trying that but from as should States, like may the I'm you a we about over -- But the think United capacity-wise; what I population, would time? evolve onset, that to the at like? just least, that wanted on in how the the about some is bit limit what what capacity population, and look throughput patients, think there's is quite think upper there Yes, of

Reshma Kewalramani

Yes, sure thing.

on specifically and Let capacity come into Stuart we and see whether the I'll comment about, back tell launch account Stuart. me the And more Taking ask there such. CTXXXX how dynamics. launch are we to in little question a about the see filings, constraints the you how bit

Stuart Arbuckle

Yes. question. Thanks, the for Lisah

level the highest dynamics about stage. our on overall views with at your or early then the to question So, we I'll them launch get this saw and the opportunity

because about XX% of of that estimate populations those of which XX,XXX. vivo who severe sickle cell the in beta by think we existing of disease, with disease sickle approach they about the of number surveys in disease regimen. they it's the And all So, indicate in would we X-time and cell a In think States. of are to going in conditioning who patients the majority EU, have between cell, And vast treat U.S. physician people and those the nature beta there's the curative is their in the validated with cell that thalassemia be terms United both the sickle disease have third XX,XXX sickle the thalassemia. of external patients between of severe would combined in

magnitude be the for might curative patients of treatment. of that's gene-editing phase this a think about order we initial who eligible of So,

of launch a dynamics, X function be of terms In things. it's really to going probably

enthusiasm is interest And in in - one seen translates the high. to-date -patient as community on enthusiasm first it the seen to clinical The we've our that trials, and technology. and expect to particularly the in the interest field, the be we results the based physician into we've from

our to the treat that we The as as But hematology transplant a bottleneck a you And have ability other to limited patients. manufacturing is diligently centers. competing we're to priorities to conditions. with our going manufacture. end, be the the of number imagine, can in there it's to capacity and make processes know, of be going they be also is centers, working treatment on is function malignant sure in have in administered to treatment those you likely U.S. And

likely these the and high, the the unmet We're treatment enthusiasm think to sickle limiting cell them. those date. and Again, And in and to patients so, we've step. centers significant initial the right that is we throws to seen be in that with working thalassemia is treat given to obviously disease outstanding going evaluating of very do results be need

that. So, without specifically, But to early those dynamics. this Lisah, given launch is stage, the wouldn't to need because were are points likely substantial of terms What we've expecting tell we though, commenting the know -- can seen. I the unmet answer the very, the to in exact results of demand amongst be very at I be some that you those XX,XXX patients, are and pinch

Reshma Kewalramani

have just thinking areas the that are the one that and will filing our we're X. how regulatory year. to on which I We're I'll engagements That's end benefit couple What require? conversations X will next of the of the remind of tail at the that agency expect where for the of every going and questions. filing in with X. the of Orphan, known On to a months go address had and tail really are wrapping I having follow-up? step to about virtually end is designation discussions and able ARMA, the have and the those be number And, size sample is -- of expect they we we number What's on those duration of here -- with do us discussions size with in finish are: our their the moment the the question we the and we're dataset allowed next that Prime. size, in back towards both a and to to EU

Lisa Becker

Thanks. Okay.

Bastiano Sanna

questions. more two take we'll Operator

Operator

Okay.

Our Kasimov JPMorgan. of question from comes next Cory

Your line is open.

Cory Kasimov

I they speak level get as betasal recently and to market ask the U.S. -- we CTXXXX clarity agreement we the recognize about level back there the to as on afternoon, piggyback on coming guys. between patients thanks. talk We can I frequently well also, with couldn't your Hey, Wanted that regulatory to on front LentiGlobin on for about it addressing to just with any comfort to CMC when months, see should follow-up come but filing. you more Good the the delays gene you on comes see regards wanted and and requirements. XX,XXX your based comfort in Europe. pricing. cell saw fact And Europe then Bluebird about with out therapies. of to that the

that get level comfort your this? Thank you you. around So, can

Reshma Kewalramani

opportunity Obviously, are including we opportunity, Europe. certainly have we leave the our CTXXXX and both you let comment is a bit only beta not to your Cory, on little factoring, with for a come Europe -- And on the about the me ask Europe, sickle-cell the the with you for to in but then start front more specific regulatory disease. thalassemia I'll telling which bit question. process little and more

benefits filings, I now how address the said with multiple questions able regulatory of had wrapping around conversations clinical of big database up conversations been the as which what's and on is -- of their is because the really the had points, we're two been most designations to and which Quick of these Okay. follow-up. agency and duration earlier,

scheme with than other gene guide really, doing manufacturing manufacturing enzyme tax challenges. an editing. things the CTXXXX, And grand of grand And what manufacturing commercial scheme side, processes. we the for that's it's the the clinical same ex the sites of using And about in vivo easier situation when things, and think you On simply the for the in same RNA. just And actually, we we're as trials the product. are it's the did

along So, for by way. And agency. de to Europe trials our used I questions been the about are same Stuart, the you a -risk same couple feel with very good processes the and product. We've clinical in addressing about we using sites able maybe their CTXXXX? comments the we of the the that in conversations from commercial

Stuart Arbuckle

our on reimbursement patients The and capability Europe to who on access translate ability feel team propositions, which have to likely, and those I conditions with gather access generate need describe to question. the the for then we these confident to of unmet for evidence and payers have sense ground demonstrate both that value to the country-by-country types may go capture diseases. for benefits in and secure the rare medicines. makes the transformative secure their thanks reimbursement very creatively our in develop the think in clinical of and work Cory, Yeah. economic and into I to medicines, medicines, payers bespoke solutions to and

to in get demonstrated access we've for think cell Europe. those us to in able CF capabilities I'm patients with I successfully to use thalassemia very, disease beta looking that and sickle very same forward and being

Cory Kasimov

guys. Thank you, Great.

Operator

Geoff American. with Bank next of comes Our question Meacham from

is open. line Your

Olivia Brokaw

I for triplet Geoff. have next-gen follow-ups guys, on Olivia Hi questions. this for the got program. two on Thanks Brokaw the

is, some look guys then get maybe both point. can we guys you are next could whether to as and both those earlier next-gen an forward that, know First trials triplets or Phase start have running, there you, And that when we now you trials an opportunity talked of might X even be us give more up and sense about for see into year? better on a of for those I at moving X those data at

to as some newer that, to specific development think those you're areas or move about be timelines And where differentiation assets? the potential focused maybe optimization there more of are those accelerate once agents on So, into ways there could you clinic? a when just follow-up the

Reshma Kewalramani

thinking wave we go? our clear, to cystic is your Hey, going about the patients can is already the X clinic what chloride. XXX, carrier questions Yeah. with where sweat is it's shared like With this? in about that is And the to be That overall in goal, which clinical molecules shared -clinical really bring X trials, coming wave, long-stated I to How good that to and whether which pre we've molecules of identified are the really How the looks the around better. that are good this Diego goal? year. results be What's on based And afternoon. levels Tezacaftor, Phase the goal even fibrosis aiming the we XXX are we we the I that Tez, next all Phase for. or feel results regard of center are can XXX/XXX that we next San earlier be So, with quickly into they clinic. here to in and

we do bring go to approval CF demonstrated So, the speed it's to well quickly. fastest to first how development of synthesis can already so TRIKAFTA, for In that, levels path one of faster therapies right? our molecule regard can fibrosis go, fast don't U.S. carrier that in went X. I anybody on chloride. of cystic we can than bringing less the With forward this couple think all than patients are reasons. of a in us sweat and we that, very to ever. years. drug four of Outside We've programs can from bench, oncology, we the

right? is medicines such, do in really results the being this, remember, and we in should TRIKAFTA, reason second for this about have And with we already we market, X enrolled that of we the a fifth one in XXX, trials XXX. fast on where months. We've The the regard expect I X to it lot say experience done with

So set the to XXX to when this our molecules we benchmark continue know that. the use trials move the XXX, clinical and we I a last fast for test to of with -- behind expect what that's experience did.

Olivia Brokaw

you very much. great, Okay, thank

Michael Partridge

for all tuning you Thank call tonight. into the

you the If to please team. have questions, additional Relations reach out Investor

Have We are in evening. tonight follow and happy good office the up. to

Operator

This program. conclude the does

now You disconnect. may

Vertex Pharmaceuticals (VRTX) 3 Nov 212021 Q3 Earnings call transcript