VRTX - Earnings call 3 2023 | Vertex Pharmaceuticals Incorporated

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Operator

Good day, and welcome to the Vertex Pharmaceuticals Third Quarter 2023 Conference Call. [Operator Instructions] Please note this event is being recorded. I would now like to turn the conference over to Ms. Susie Lisa. Please go ahead, ma'am.

Susie Lisa

Good evening, all. My name is Susie Lisa, and as the Senior Vice President of Investor Relations, it is my pleasure to welcome you to our third quarter 2023 financial results conference call. prepared have making call, tonight's remarks, Dr. we On President; Chief Financial Operating Kewalramani, Vertex's and Arbuckle, and CEO Chief Reshma Officer; Stuart Charlie Officer. Wagner, you that webcast recommend access listen as to We this you call. slides the being is website. our available call replay will recorded, The be on a and

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

the risk of exchange impact is In foreign inclusive program. management presented of addition, exchange foreign our

I'll to now Reshma. turn the call over

Reshma Kewalramani

Thanks, and product Susie. billion. today. the strong third more global you patients, another for X% We've to grew and execution delivered evening year the $X.XX quarter thank us approximately CF on CF prior full joining product to the company. By year XXXX call drive quarter across reaching are all, versus period, we revenue continue Good and raising revenue guidance

readouts year, an term areas disease PDUFA our we medicines combination transfusion-dependent pain X acute marketed for diabetic therapy A trial XXXX and in from overview, our the of severe this on our in readout of and longer enrollment are in year-end commercial year while in portion on trial of program vanzacaftor multiple and both cell a date vanzacaftor for both simultaneously this neuropathic pivotal in and in peripheral tracking in year. With cystic for triple II AMKD CF exa-cel next cell XX in II/III fibrosis. Most U.K. CF disease regulatory March triple of trial for Phase pain. with III the me on beta-thalassemia. and also II later in December Europe TDT VX-XXX, Phase delivering and are We underway our acute program pipeline near-term ahead sickle with the in of sickle launches, early reviews our from notably, peripheral now global turn by exa-cel XXXX, in towards and pain for VX-XXX excellence VX-XXX Phase cystic that VX-XXX Phase potential Starting let update. including fibrosis. to completion neuropathy preparing our

Phase we and our in XX years to studies vanzacaftor to patients and the X studies, For XXXX. XXX XXXX complete in and track share the XXX therapy, all RIDGELINE these next-in-class X triple in pivotal patients X III by ages results end on and XX combination of study ages remain above, SKYLINE early from

HBE upon assays generated our high We improvements evidence including the greater CFTR triple deliver can totality function combination have that of enhanced substantially a versus vitro II triple lower vanzacaftor benefit patients It in for once-daily TRIKAFTA Phase and the also royalty studies. convenience potential the The in date, than based TRIKAFTA to vanzacaftor burden. holds carries and expectations the in for dosing. from of clinical

with for Moderna from -- in in our development to CFTR our addition, at CFTR another partners the program more X,XXX than patients mRNA who important therapy continue VX-XXX, our with In modulators. portfolio, CF cannot we progress CF make benefit and

year. ascending and complete the to by end expect ascending the initiate the study single to continue dose portion of We the dose multiple portion this of

to exa-cel. now Turning

the a Exa-cel beta-thalassemia. sickle It and across commercial cure the diseases. people transfusion-dependent onetime is advancement represents for a these living and disease U.S. and and functional cell severe for disease Our beta-thalassemia for This Europe. CRISPR/CasX-based debilitating holds sickle transfusion-dependent gene large life-shortening with XX,XXX be program potential the opportunity. estimated an cell program enormous editing to

to chance On the discuss hear significant filing the are week, to first review front, to upcoming the the process exa-cel Advisory CRISPR/CasX-based well PDUFA forward therapeutic. and very patients. meeting into also members and gene-editing with the the precise, represented the Vertex in patients. therapy regulatory regulatory Internationally, in of last jurisdictions FDA the stories the coming have months. look potential and in of the We were Committee very in we to the for to these The durable EU, from our we and potential U.S., pleased dates to this milestone bringing compelling a both regulatory had expect the decisions and U.K.

Kingdom a authorization recently Food I receive marketing exa-cel the for SFDA. Authority Drug to addition, we the Saudi share first of and pleased both reflecting application you Saudi unmet updating that to look the by exa-cel designation coming for am to exa-cel breakthrough submitted enthusiasm In months. and in medicine We to is SFDA, Arabia. the ever the high or need the the forward in high

strong and of III promise rapid, relief an need, indication to the of side effects in and without therapy. in the which the effective NaVX.X novel, pain efficacious unmet inhibitor an and neuropathic addictive or for significant a interest commercial opportunity program been pain acute both high pace and, we non-opioid the program acute highly pain. of and represents that pain VX-XXX, selective in patient see Moving therefore, pain properties acute our on physician holds The as opioids the has Phase

year. safety complete study bunionectomy the and have We single-arm end track by of trial in controlled the completed to RCT this randomized efficacy remain on the abdominoplasty, and in a

studying previously the and Phase XX-week discussed, that predecessor with X results also completed We pleased We're end and designed and the Phase II proof-of-concept all this in expect care or studies PNP, early in broad, VX-XXX demonstrated the trial usage we acute time, will neuropathic pain support our results molecule XXXX. This label pain prescribing analyze same proof-of-concept peripheral we Recall, and to on yet from the at do anticipate to in multiple Phase neuropathic unblind, study. III positive dose-ranging As am been so has to share previously year. unmet program moderate-to-severe enable by a settings. have across VX-XXX DPN, or neuropathy diabetic another II we peripheral need. area of sharing pain. In to of we high I this share comprehensive

area the caused the II the impairment in the neuropathic second await to It's we peripheral lumbosacral of of VX-XXX As pain of year the the radiculopathy nerve spine. lumbar roots end a a excited or DPN by we study initiate results, are pain by neuropathic of LSR. Phase of type in

the of large a the patients pain to of therapeutic neuropathic the in options, of these the opportunity we significant the potential and NaVX.X Given holds, serve to types. pursue each VX-XXX excited limited mechanism are number that promise

islet Next, differentiated with diabetes. on X where are for type to type diabetes, X insulin-producing cells cell-derived stem people we fully evaluating

a a the develop we onetime X.X foundational to whole. America for the is diabetes, The millions with goal is proof-of-concept, and program the living as have in Our Europe X more cell potential million to cure alone. North than of type diabetes already people X patients VX-XXX functional where program, established naked including type or

cells data Part VX-XXX C administers study. islet of study target which month, is dosing positive Here, protect A we take all the status, regard the clinical system. At the and with patients now patients With to presented from immunosuppressants the fully the of Parts EASD from updated to standard concurrent study, immune full in last B enrolled. dose

requirement VX-XXX, no a device. proprietary immunosuppressants. for program, And is plus second these immunoprotective Our same or the hence, program there device encapsulates cells cells in

for still We VX-XXX, have begun Part fully in we the to by first this We and Phase the of Transitioning potential in end kidney a need VX-XXX underlying a enrollment is medicine the the to in third The the stage, dose hypoimmune accelerated for in the patients inaxaplin in II/III to A now AMKD program, study Phase year. move enroll of we expect pathway of with IIb continues complete disease adaptive cells pivotal approval or The in and of the III to or our so obviate QX the of dosing select with as portion added dose inaxaplin single to to in dose-ranging cause the patients, same the expect our finally, to cells, program AMKD. study U.S. research APOLX-mediated to of a study. And enrollment is differentiated immunosuppressants. XXXX. now target Phase which study

Now deficiency turning antitrypsin or to alpha-X AATD.

We program. have discontinued Phase development in non-serious rash events of to VX-XXX the some due in patients II

it turn drug-like Our steps greater to dose more and properties, and VX-XXX, both potency the These I as We in better are that, sharing months. With enroll including in and more learn trials continue on both clinical VX-XXX coming Stuart. to look forward AATD, to healthy trials. Phase next-generation I'll volunteers. molecules next we and have over

Stuart Arbuckle

delivered globally versus remarks strong a X% I and via prior Reshma. agreements. on new results CF, younger my patients growing as result age exa-cel We third Thanks, reimbursement commercial approvals quarter CF in with new tonight continue will of focus we groups and product pain. as regulatory to revenue reach year the

all CF clinical in to living has strategy increased develop Our and to serially with to always been benefit. people for medicines CF innovate deliver

And a then term with our the Moderna. age drive program, are vanzacaftor term younger that We longer is medium growth near groups. in with we over triple the execute with continue to our on combination focus goal mRNA and will VX-XXX term with developing partnership to

opportunity. multibillion-dollar exa-cel, Now next launch potential and turning our to targeted

of beta will we've track eligible payers, majority enabled has In the cell XX% treatment and Italy quarter, live as journey. of eligible in patients Italy Europe, approximately I U.K. disease for Within and represent U.S. technology add-on in that of and while disease, France some TDT in with the cell majority the a cell near-term or Europe. the eligible sickle XX,XXX TDT preparations with U.S. with for and prevalence the patient the potential U.S. U.K., application France, patients are Europe, detail X,XXX are authorized new provide including in insights recently and and our disease. In NTAP, Germany. thalassemia. The ahead the XX,XXX previously Medicare with the and the highlighted and sickle This approvals readiness supply while globally launch countries: eligible network U.S., are our payment patients, sickle there completed launch to all patients with then patients severe we are of the majority on patients far patients with approximately of X highest by centers regulatory Europe, of disease

submission exa-cel In addition, Reshma in mentioned Saudi Arabia or of the MAA KSA. Kingdom our

with in severe providing with KSA. you on aim to and We with engaging access to thousands and the important is team reimbursement Our additional activation, of this Saudi patients about with working of more support to future calls health disease information forward the ATC the authorities the exa-cel look bringing processes on opportunity.

launch, it down each the exa-cel and approval can into can key which for X journey, is important be prepare broken months. which to take we several As understand patient phases, of

the and a First, full pretreatment. Initially, ATC. the decide for potential is they undergoes patient when to patient exa-cel. a is patient to whether the their a an fit transplant right for referral them, at then are therapy is then determine treatment physician that exa-cel workup scheduled, referred Once hematologists with

mobilization. blood Second, manufacturing marrow edited edited control. cell cell apheresis. The only patients the that bone given given patient do infusion phase an we TDT tested transfusions round for as mobilization is the of for X the final patient's choose cells which works need move peripheral revenue longer conditioning, phase, of premobilization care. rounds facilities involves the on the This sent mobilization engraftment edited ready, to the Once they treatment includes apheresis. across best therapy, then then This they is step from where disease time cell blood patients their transfusions approximate timing quality all for And is contrast, X point takes then at Cell manufacturing. which require and and red starts the mobilization and phase cells In will preferred treatment not are require to months treatment. patient journey for X must recognize myeloablative timing post-infusion a are patient and collection the are to prior typically and the through cells sickle of can our apheresis. for blood, and into be average, collection The collected the where A waiting cells this phase stem the for factor this for of critical stay. involves X-month geographies. hospital consistent in lives and cells that

possibility deliver of journey, of VX-XXX, with NaVX.X steps a to the the transformative and pain. Given expect XXXX year we inhibitor patients now outcomes the patient for a highly works begin for benefit. Shifting multiple to and be the the Vertex to exa-cel foundational lifetime this duration journey, first selective our as of

we anticonvulsants and pain, patients today and as treatment patients for approximately essentially believe rapid approved the for the are surgery than patients acute of and for million launch. PNP XX effects the a our program's pain stems pace that an X/X its patients. In an acute opioids the the market profile see either some for by despite the planning share outline millions we've go-to-market medicine advancement, ambulatory VX-XXX in could or hospital every or U.S. for overall are pain innovation Both driven given In to alone, need from the neuropathic transformative institution, done profiles option estimate opportunities severe these pain challenges pain of the markets opportunity during developed work acute gabapentinoids than are to pain. We their more a we like U.S. have each we of pain. of prescriptions acute size addiction Given of the XX to of near-term strategies massive. acute actively suboptimal of benefit and upside lack such to in for clinical at existing More agents multibillion-dollar I'd potential all additional are efficacy unmet potential are adverse a generic, each a fact peripheral year moderate like prescribed the consistent receive of and discharge. visit year, prescribed center provide opportunity. Overall, the each of currently treated neuropathic treatments. or and risk The of is the million

IDNs, a large is specialty As force. opportunity can hospital-driven XXX and the X,XXX prescribing concentrated of reach patient with amongst proportion we some sales hospitals a

role is and a an the mentioned, is prescribed in every medicine specialty LSR patients. and more bringing control treatment to be given opportunity on conclusion, Therefore, and but with pain options. represents of which the completed Vertex study pain. DPN the in patients. In impairment X study over dosing LSR are the our is all forward neuropathic peripheral patients, represents PNP play addressable in force that exciting is DPN U.S. limited pain Vertex. a XX% sales collection in patients the we exciting believe while chronic causes all largest limited treating and often effective segments. of segment we of Reshma a condition year patient therapeutic PNP of PNP commercial Specialists it's PNP approximately PNP at to perfectly. XX begin. for is given PNP chronic to as treatments are with PNP a about As an the time are DPN approximately fits of in can specialty critical LSR XX% search in patients innovation options. PNP conditions nerve look million PNP model multiple

in the bringing the beta of launches, market improve for and on review to to acute pain, We're patients opportunities exa-cel. with vanzacaftor verge disease cell CF the and for to both of potential represent or Vertex. I sickle potential have will We thalassemia turn CF VX-XXX significant dramatically financials. continue preparing to in also the now patients' the lives call Charlie over triple which are with treating near-term more make a multiple cure additional progress patients to and including functional

Charles Wagner

our the consistent Thanks, Vertex's growth Stuart. of once XXXX strong excellent attractive quarter profile. demonstrate third results in and again performance

U.S., patients recently uptake quarter X% achieved Third to well year-over-year reimbursement X approval TRIKAFTA billion. following in continued and revenue recent year-over-year markets in of younger outside ages age X as on X% TRIKAFTA/KAFTRIO as label increased of strong grew year-over-year X% $X.XX extensions with groups. revenue XXXX U.S. the revenue the to FDA in grew

the markets that XXX in period, XXXX expectations. anticipated, international foreign $X.XX drawdown of represents changes revenue growth basis the inventory revenue have line to an drivers XX% including prior in QX, over year As primary corresponding contrast first the our approximate headwind from inventory we year. the in the in of been half saw point experienced currency. Overall, of in of with the growth in in certain Year-to-date in billion we increases

combined build-out Third research areas compared included prior million clinical significant expenses expected 'XX. as $XX acquired investment and million third quarter CF the SG&A our quarter results as increased versus expense by such as the continued compared to of third were the million well year acute in include X QX our triple R&D, IPR&D quarter and most for of in XXXX, Throughout the XXXX diabetes IPR&D $XX driven charges pipeline. the growth was XXXX. Operating acquired expanding charges non-GAAP pipeline. million of for $XXX $XXX for studies pain, XXXX vanzacaftor to in VX-XXX of in in type investment and capabilities of of

our for near-term addition, continued and other pre-commercial activities In launches. anticipated exa-cel we

operating compared in Third third the non-GAAP billion, of quarter to quarter XXXX XXXX. income was $X.XX $X.XX billion

earnings quarter quarter in quarter representing billion investments. We per Third and share XXXX X% $XX.X growth to compared ended the third XXXX. with were the non-GAAP of in cash $X.XX, $X.XX

continued XXX,XXX Our priorities approximately in we as including have have prioritize $XXX $XXX over innovation external unchanged business development. shares. total continue share consideration to we million. Year-to-date, deployment million of year-to-date, XX and nearly transactions spent via allocate also completed investment repurchases for to cash to We've to cash repurchase remain approximately innovation, we

Now to switching guidance.

guidance Given Slide increasing are on consistent year-to-date our XX. detailed results our and strong revenue XXXX our we as execution,

rate currency. $X.X continues billion. versus net this expected changes from prior an of year billion expect basis approximate foreign guidance our range that For the approximately revenue growth to XXX revenue full point CF of include $X.X billion our product to Note XXXX, we $X.XX now revenue in headwind to

and billion $X.X range non-GAAP for XXXX expenses SG&A We of our to in are R&D, billion. combined the maintaining acquired IPR&D guidance $X.X

expenses our the given our programs. the R&D majority invest operating into multiple of late-stage to continue clinical development mid- and momentum in We

range multibillion-dollar U.S. We of credit to in estimate, commercial programs of our leverage XX%. our with an rate for are lowering launch basis to year our potential markets. Due in to also specialty in increase XX% opportunities R&D prior an the represented expansion tax year non-GAAP by current near-term the capabilities attractive of XX% the tax model XXXX we range are by a by to our full XXX points afforded effective XX% guidance projected funding focus to business versus anticipation our continuing while of

In again milestones, revenue 'XX, trial continued and regulatory internally QX closing, important and Vertex in investments, clinical externally. excellent progress achieving delivered strong growth yet results ongoing both

continue advance and progress we Slide to we our anticipate our into further As and XX begin to highlighted you continued as calls, Q&A to to our disease head Susie areas. We future out the XXXX mark programs I'll progress multiple in close forward updating to on on look ask important XXXX, milestones on period.

Operator

Difficulty] And the with Geoff from Instructions] will [Operator America. [Technical Bank come question first of Meacham

Susie Lisa

were understand. We'll sorry, Geoff, you. can't to to and garbled. you the back We go come next

Geoffrey Meacham

Is better? sorry. am it I

Susie Lisa

Yes, that's Thanks. better.

Geoffrey Meacham

or renegotiate could know with to Europe data, when if I that. like sure you So out across would your is had At was mechanism? bar help portfolio for agreements question look high difficult is this second on Is much risk I think a the initially first you a the question what that? molecules AAT. level, if you the you to it maybe it how the role thought. the vanzacaftor rapidly indications? of you indication part just it OUS and Is wasn't in It seems characterize more reimbursement than itself? and benefit? Or us

Reshma Kewalramani

Yes. Sure Reshma. thing. This is

vanzacaftor molecule take on the with And it our is. break rash. this, for Stuart and nonserious VX-XXX AATD part that you globally. Geoff, I'll see and questions. a almost plans particular That's AAT, launching is when me in first molecule That's it. what specific. X I'll on ask the to Let always the it's comment issue into the

and portfolio or those event and molecules in data and not they a the a the no, forward are Phase getting action. the it's settle XX next mechanism those looking molecule development. and we This VX-XXX will event results So molecules, really next strategy to select and remain to of I get the is we're And on important is X into see steps. when which where play comes data be results VX-XXX,

a is me the Stuart to Let global to it study, And and intent regulatory over clarify, talk the turn to Vanza. for submissions is global launch. Stuart? global and for a study about

Stuart Arbuckle

Yes, Geoff.

It importantly, TRIKAFTA. So outside as on reimbursement when a vanzacaftor your the imagined comparison our on access we and study specific with this expectations embark of some question, agreements? was. is for said, head-to-head To of U.S., was Reshma portfolio

Some I getting across in increasingly to kind clauses TRIKAFTA, anticipation kind But do if and for our profile It the be. prove would of that we will all which better product clear. of beat access of just like include to we our is benchmarks what to better agreements vanzacaftor medicines, did isn't expect, expect with ex-U.S. we reimbursement of can the them include to deliver will markets. of the that industry patients be

Operator

will come question from next The Robyn Truist Karnauskas Securities. with

Robyn Karnauskas

thanks these is -- doing on saying, like in are the the if 'XX for on unblinding for concerns you Reshma, risks? My Two sounds going all about And that any at to PNP. for me. investor transformative your It trial? breakout be there's time. Vertex, we help pain, same us same for if question second have [indiscernible] time. the year Maybe clarifying you why 'XX, many the But understand -- can at questions a is One strategy ways. question,

drug? you'll a said some market looking scheduled you So though that for data? sales it's context you're expectations it even for in Maybe specialty How now you a do the what force. exists that have for, Lyrica

Reshma Kewalramani

X side. on side on Robyn, the pain pain Yes. VX-XXX, X and neuropathic questions the acute X on

I'll me pain acute neuropathic on and tackle the on Stuart approach ask side. to our Let comment commercialization

single-arm our is reason program share of is to third the from The goal and and need these are pain in is and planning trials. is a the is all do RCT, the safety analyze abdominoplasty And at bunionectomy entirety broad in RCT, is that, trial. to the X label. moderate-to-severe unblind, pivotal Second everyone, order One we the its acute time to results trials. effectiveness So a pain we to results because ground And the acute all same secure the the

regard the you all the that's may want the do to to for it we are what at think then I'll about frame time. With to PNP, Stuart the study, year. I'll have commercialization. study has completed results The is end that's for the II Lyrica we of Phase and to just a reason So to reference and same up a turn that how It arm. sharing it expect with the profile, the and results share over looking this study before

not So we it's have medicine it's this so the not patient the but goal risk benefit just so our kind magnitude or is is my radiculopathy, that in a our LSR we're comparison with really end there missed, effect. And a in and called of population. a of that treatment into remarks that comments study effectively before the prepared another peripheral expanding made than neuropathic of one can year profile a lumbosacral the pain Lyrica compete assess for we I area And here bring to better can neuropathic of now what's will progress it's initiate pain. Stuart? that

Stuart Arbuckle

Yes.

Robyn, very on to Just expand briefly. that,

be regard. LSR XX% therapies which compete there, demonstrate care of what I improved should obviously in patients to we're generic obviously, is which able of know, no market in to is products, as all different II going are of million an approved you Reshma segment. are over That's are there, accounts which for ongoing cause Phase study, approved revealing has accounts do and people. And say, specifically said, is and profile XX which is successfully gabapentinoids, about DPN very describe is There, of conditions looking or the causes risk term, that DNP, PNP which, have standard used for with pain. XX% approximately for umbrella of with benefit collection their PNP. a kind there is nerve very because to impairment, LSR are been as of to study which the LSR. PNP. in So is there which with to the an as XX% U.S. be the there that the XXX, patients

So is it's There, no very going placebo. pain comparator relief we to see well. therapies, can that significant opportunity approved effective be, that a as with demonstrate obviously, versus we the as likely

Robyn Karnauskas

know you just have better? but want have it follow-up, know Do one issues with of can. to with We you look safer? better obviously a know I lot if the for to or has I I Lyrica looking a And Do Lyrica. better look sidearm safety just reimbursement. doctors

Reshma Kewalramani

Yes, comparator. yes, the yes. The safety at are change Lyrica about -- profile program baseline Lyrica. Lyrica not with is be goal that as doses from point issues VX-XXX have arm, various a for reference you a product, But The with our a benefit-risk II real. looking the Phase we're a tolerability is the what here, to in to clear, better is make than

Operator

The next question will come from Salveen Richter with Goldman Sachs.

Salveen Richter

dose this the help for And get the us profile waiting versus out you cell program maybe seems LSR the again. context the a there? here. That understanding better then understand to on that second and trial saw partial on wondering now in One of calls you the approach? naked to DPN the trial. start why the I'm with read side pain just And with here trial device question Can and staggered on a the enrollment derisking why be a of

Reshma Kewalramani

Sure thing.

Let designed go part we device dosing to doing staggered are reason protocol global sufficient it with assess between to is XXX safety in VX-XXX with patients in regulators. we ensure and go first-in-man is consultation is Part me the cells I'll be the is start the The that have that And are with pain. that and back to a dose a the partial time and of we plus slowly able the to program program. the at way because question, that A

the that's device is So cells trial. It's because first-in-man plus a reason.

question. go now? marketplace. as reason good and and commercial is because LSR, has peripheral back It's to diabetic or very DPN Let started DPN established as with a now regulatory has me neuropathy well really The established VX-XXX a pathway an why and we had

program, XXX started that's actually started that's PNP when with and DPN. pursued started we first VX-XXX. So We pain we we or when with neuropathic peripheral with the it did why we indication, the

in the acute a confirmation we to VX-XXX attention a discussions was like perspective and LSR recently our pursuing our turned We we've study intention gotten we're has just pursue As paying a the our regulatory setting. regulatory gained the as frankly, completion, on broad broad from LSR VX-XXX, PNP always to pain started pain LSR. that is type pain near for and label type. label neuropathic a It that peripheral completed

neuropathic said, completed And a the a PNP medically, from Stuart pain and terribly standpoint, a confirm II that. LSR our you, I'm excited indeed got regulatory why the regulators under we As tell And about to starting Phase type. scientifically type. That's it study discussions also falls peripheral umbrella that now. with we're I that of is

Operator

will next Nadeau Phil Your come question with Cowen. TD from

Philip Nadeau

A of follow-ups couple from us.

a New new TRIKAFTA the that debate. investors on author of the franchise. where Vanza summer, that on to Vanza. patient over saw pain, Or on remarks, First, author? And topic of The editorial? with was of somewhat what was or In do drive going editorial Vanza Journal prepared wrong? elaborate think you you Could England to a it's opportunities the you you those the then skeptical comments Can respond a can't? do explorer could second, the a got disagree What XXX, intermediate of and populations Medicine bit that follow-up more, growth mentioned currently you that

Reshma Kewalramani

the III maybe a a looks I a trial. the terms of I study effect magnitude and holy I desire full people promising how II in the X,XXX very another study, to in of only abdominoplasty terms in say more have would be the in this the should versus the to potential pain people distillation There's about in of the of Phase think we'll it And the bit not and end X,XXX opioids going and we the it to study. to to make There Phil, how in the versus a but in people all another all. learn effect, editorial, this safety comes think X,XXX thing. secondary is Phase Yes, the points. And about should far people grail we assessment of and little study, placebo, sure results. then treatment data is bigger effectiveness about think bunionectomy we we're need think study, XXX a about It's down when targets. have the what

let's think results So trial. the And agree promising. the II I look is the best III Phase I answer Phase towards very are

Let me turn about it talk over Stuart to to Vanza.

Stuart Arbuckle

a patients. But just to small about got be to on really X,XXX switched growth CFTR to initiated vanzacaftor. profile CFTR people want deliver Vanza But currently on about who opportunity is the think versus number relatively they that's on modulator, One sort TRIKAFTA, we over we're Vanza, of existing would to patients are globally -- actually be CFTR on Then with who the modulator. I be Phil, yet patients for the opportunity hoping been may a of not is there's that patients Vanza now a discontinued you've on threefold. have an initiated modulator. to big if for X,XXX really, who've

of CFTR have So they variety to modulator but for a on a discontinue. wanted be to had reasons

about modulators CFTR still often because know CFTR As chronic around on from world. I but tried. other of relatively modulator it's patients. they number our medication it's We say, be sort the any like don't because compared now they of X,XXX small would discontinuations talk previously over a actually We sizable to that's percentage patients a to a

profile they think deliver are we patients very so who interested expecting. we in be we're if And that the kind Vanza could of

Operator

with come will question Bansal Mohit from Next Fargo. Wells

Mohit Bansal

question, DPN staying One again, the Congrats study. progress. on on all

us mean remind setting? more that a to how our that like in think is is more replace it opioid third-line Or in Lyrica prevalent the the I to from agent. seems use the And it setting? in replace it is opioids drugs? the setting? of thought you you how could like this reading, So about profile is Because do

Reshma Kewalramani

I'll Stuart -- thing. used have about Sure is in being DPN what the today? talk

Stuart Arbuckle

are a in classes available the is largely medicines, which pretty is of there various Mohit, lot the going variable. polypharmacy because right on now, today Yes. of efficacy DPN pain of

on do you So patients see are who nonsteroidals.

been a also see which lot -- as on well. studied people on and opioids gabapentinoids, approved see And you there. of who You have the do patients are parents

about these care new currently prospect either characterized efficacy for the by of establish being able really and/or events it due the largely standard That's therapies. the disease sort polypharmacy available of excited a patients. why so a So adverse of we're variable is XXX of to to

Operator

question Fye The from JPMorgan. next Jessica will with come

Jessica Fye

approach The X.X additional portfolio dual inhibitors? Vertex's press and X.X and do and X.X in And X.X you're have NaVX.X development of inhibitors you mention release behind VX-XXX? makes any along advanced most to R&D How far working on. the is inhibitor next

Reshma Kewalramani

question. strategy, terrific Yes. out is extended The portfolio R&D you've across it Jess. and Welcome seen pipeline. authentically as in Really back, our CF play reproducibly

the the we next pertains more So the in as are And our making through that, trials. inhibitors NaVX.X of part already research I clinic to in Phase it organization. way after their pain, have

it's question, combining the we that the the propagation. that the think who of progress. say NaVX.Xs the and NaVX.X the research are real in making opportunity is also good the that stage along, that a I that for action for transducing in NaVX.X, for all I see very is In then -- on propagation, NaVX.Xs just and action of way and potentially action And reason an NaVX.X use potential X.Xs. they as and the the and see others the works works the of stimulation pain on potential stimulation reason And we agent, excellent is signal the terms potential are a of that single following in periphery works and and there the

in we itself. So, NaVX.X we opportunity but of and lot in see the see of opportunity combination also of a

Operator

next come Partners. The question from will with Risinger David Leerink

David Risinger

Yes. questions, X I have please.

until how in not use placebo should the acute And DPN could potentially to likely plan Phase how studies, in be II in or alone? Phase And you rescue VX-XXX Phase medicine then VX-XXX the patients complete 'XX III months, conduct it in And will do disclosed? plan III you pain to succeeds rescue how arms? III? 'XX? regarding and if to Do used coming you medicine expect benefit First, we go Phase

Reshma Kewalramani

me question at both has go the to start let component a P&P it answer of can David, Okay. alone. of these questions. we But the

that to And so it. I on sure want Stuart touches make

for acute of Let diabetic going lumbosacral me pain terms punchline, radiculopathy, of development to we commercialize for the Vertexian it we neuropathic are to diseases, to terms in are are ourselves. of this and call both if can these the pain by acute cut neuropathy do and in pain, that, going LSR, and I and ourselves absolutely neuropathic in commercialization. by pain Both terms of

and cetera. about little meds, acute come I'll pain to commercializing back tell pain, ask neuropathic to in et Stuart comment bit you rescue a more I'll

Stuart Arbuckle

on David, this disease what of building said for characterized by being so a I is which patients unfortunately kind about state, polypharmacy.

influenced well. is patients are often seeking so relief specialists indeed and treated pain to by getting. heavily And are LSR as DPN superior what and/or for they for It

states so overall, a believe can for pain commercially within we those achieve specialty with PNP And sales both success of we force.

As be in and go to going studies in III ourselves commercially, a LSR are successful alone result, to we're as of doing Phase the wanting DPN hopefully, II in studies Phase well.

Reshma Kewalramani

the analysis very use III the in statistical that II did studies are round light, plan portion And study, it medicines the Phase in on in well -- we thought of out -- the been in The the there very it's acute and David, of how considered there. about to of questions Phase through consider the same and we rescue to portion pain. with the have your as carefully acute pain

have don't and as been to results, be was will when think we but medicines, has to accounted just in say the it deeply about course, disclosed it much considered of and in Phase the for well other rescue I II. more than So, share how use the publications of

I pain III focus think there designed comments on Phase FDA. one question the the will my about neuropathic in DPN. there That I'll peripheral structure. was be with a and

can't and that have therefore, positive. be that's exactly on are like, but look our once specifics what II you had yet will end haven't the We what next they will Phase results, of give program DPN step I Phase meeting we the assuming II

Operator

The Evan Markets. Capital with Seigerman BMO next question will come from

Evan Seigerman

Congrats access U.K. impact the in implications of your on on recently that for I system. in the the wanted released effective and you of U.K. progress TRIKAFTA talk to resolve ensure to not appraisal Those was impressions XXXX. franchise the cost the the indicating U.K. essentially expand that the Could to about steps to was maybe are as U.K? always. NICE settled

Stuart Arbuckle

Evan. Yes,

thing that that the review part So NHS was with of expected we the I'll XXXX. ongoing the the first is in an this say negotiated about contract NICE

years after isn't medicines this that on is NICE a the So surprise X market. reviewing our

to that's So thing the first say.

expectancy, data, TRIKAFTA just after lung the which it's transplants. as III perhaps that are that the NICE I data in extraordinary, think you of and in even a results real seen things reductions market. exacerbations, data is real-world real what than absolutely world, would the the the in results NHSE the As a with and a well of I we've think like I increases think, you seen know, see life with better in part to the agreed and with Phase done we've reductions including seen We in of expected we open-label being trial period we on performs virtual submitted having world elimination the collect original because hospitalizations, have U.K. And from unusual, And extension medicine, submit contract, that. clinical data -- we

though, NICE. that, be and draft Committee disappointed. from just confident Meeting, So medicines certainly with fair it's process. pretty say, be consultation. period the to There a at There's end value our we're the this was NICE It guidance. will guidance full of that of going is It's feel I a reflected the of to draft second

Operator

The Bayko will come next question with Liisa from ISI. Evercore

Liisa Bayko

to questions X to wanted circle and I back me. pain just for

you're X did the Medicine pain indications the editorial acute in The focus First as using overwhelmingly -- in of that on New of article examples. England Journal in the more females all,

a can on CF And you've developing. of working underrepresentation as addressing market of males. And much hospital then on sort you could to obviously you capabilities than need any waiting to that building maybe just speak to been commercialize finally, the if that is and of with capabilities lot So the just and of cetera? get on et sort how those into This comment a formulary, are different you're for data? competition generic you

Reshma Kewalramani

Liisa, is Sure thing. this Reshma.

want own more including in it our we then guess to that trials Stuart. of pass more companies, to over I we our ensure and in Most color people start, our I'll trials, succeeded. me women have have and Let

community, -- regard that remember, a part belly only of is and call that the policymakers, to of the as kind In is opioids. pain pain on setting the interest study, So potential a X something and an -- that just to people addiction the effective procedure involving Perhaps pain, interest some underlying fact we tuck. to addiction And it to point women it, tummy is fat frame surgical that elements procedure of in that could a opioids turn is, over acute of acute VX-XXX undergo. With most concept. the And I that make want address pain relief abdominoplasty see that the to physicians, many was but following is a commercialization the without in to has see comment abdominoplasty to one this not of Vertex lot important but XXX tailwinds. the positive. it's Stuart, it of up of of I of potential surgery. is underneath more women the I'll study It's the a

to turn efforts but as the him so comment tailwinds on I'll it I our ask commercialization to Stuart? we over see. also And Stuart,

Stuart Arbuckle

Yes.

to both worlds. question capabilities, So think your the to about get I of we're trying best

market use medicines to at couple policy for from away changes settings, cetera, in prescribe put like medicines to can things patients pain who of to quarters can tailwinds said, of just with sure looking in right a move thing around ours. And of get there people to people as sort opioids appropriate using made have pain long, kind we're are access effective and people institutions, generic us highlighted now a that et like capabilities Act, looking for of that available. ability reimbursement that make how if we disincentives in also to support that place of barriers is for in NOPAIN the no of successful restrictions medicines and which call leverage people they've our them for effective terms to and or we non-opioid medicine the previously work our financial because where date, ago there the are are doing that, the seeing looking like and trying which them, much are which Reshma based to the policymakers, guideline be We to with the already with

a we're going something to that, we've very obviously that's that looking we So help is market. are to going us be the hospital to used a the to of going be into said that be to build segment selling This be institution capabilities some on, successful. Having different driven of sale.

new of we brought looking our on institutional kind in are in in setting. so as And brought pain and are to that we capabilities have who business experienced bring unit, people

So and bring well. to company leverage the what good new who I people the worlds, while experience get in we've knowledge, bringing would say are of to we in trying past, as best both been skills at the

Operator

The come Jefferies. with from will Michael next Yee question

Michael Yee

had We quarter. and a great follow-up question a on a on Congrats Vanza. on question then pain

pain, then and chronic doses study a also Lyrica the standpoint, tolerability as doses, or disclosed a there you've think used the me Phase can that QT remind II and that had it's from acute from never the the we label? in And understood? safety doses same doses I well Vicodin should since the a you And was is and control the basically expect just that On pretty completed.

would was the that So be would was there if to positive. anything a great to disclose be there? that That hear case,

of powered then on et totality us, magnitude superiority. that, that chloride it's around you know well, was Vanza, it and a deem is But noninferiority be superiority. can And I to sweat you questions would Or on meaningfulness And for know is there may potentially provide? cetera, and just meaningful? there everything clinical that I of the like that as more the benefits remind FEV,

Reshma Kewalramani

and ask et will start up set the what start, XXX But let vis-a-vis to to valued have Sure sweat Vanza. trial for the that chloride, go Stuart I'll done I'll and team structure me is Mike, and Vanza and Stuart thing. then I cetera. then clinical back [indiscernible] research on

abdominoplasty the acute and the Phase that Phase the So And high the Phase III bunionectomy, II on conditions, the trial to dose, similar the showed benefit dose opioid. for pain VX-XXX the exceptionally II the in doses VX-XXX are trial. Phase selected trials. is standard to pain confirm, for III trials, Same labeled we it correct, you

the peripheral trial, neuropathy has the the make we doses and label. we from correct, on the the dosing standard to one need so The seek. reference to II And doses, that are different with But peripheral the study arm, chronic standard obviously, and Lyrica's we appropriate has neuropathy label. Phase because from it acute regard adjustments correct, have doses exposure than dosing pain in the one the diabetic the is the II Phase acute,

regulatory because -- is that a a this ppFEVX versus this enabling is end vanzacaftor, TRIKAFTA. sweat endpoint function. have direct head-to-head and is The we point. on And the important secondary is sweat key on chloride because that is the study CFTR -- recall, most is chloride endpoint readout Lastly, very of that's remember, it's this primary is direct the

that well understood show not is chloride, that or You function, are we that that is know ceiling ppFEVX, debates very we whether there terms have CFTR if to studied disease, the we've lung a on III can't variety tall better sweat up is of provide with chloride, it it's patients reported and even how the indeed endpoint than diagnosed case Phase a a better in -- secondary be Phase know would than is than sweat trial, benefit in great order Vanza II there been better normal and in about But TRIKAFTA. that's better right? look have it that's already triple a that of is that does terms I in the trials, the and but what even and function out of

Over in to you, Stuart, with marketplace mind. that the on

Stuart Arbuckle

Yes.

noninferiority versus study, as see just on high TRIKAFTA is in as an to said able does Reshma Mike, the the we will TRIKAFTA FEVX, incredibly be versus primary you which, on we vanzacaftor but bar the is study. So endpoint know, how

to So first just that's thing, the you. remind

FEVX we've that chloride. can the even same CFTR of profile. the sweat benefit which the a with As pharmacodynamic also of looking with when is said, Reshma is said, that research Reshma function of CFTR a vanzacaftor. we're if in superior demonstrate we as If at measure patients. lot which, physicians, There has for improved enthusiasm from function, measures done is physicians product, And

going to as vanzacaftor just who is be addition, once-a-day to benefit, have as you, well. well, be considered to In patients compliance for particularly is those which also remind regimen also a challenges a

very we're be going for there's if Phase vanzacaftor. the the forward as looking So them high much to to results -- to and results think III out of expect level come a enthusiasm we

Michael Yee

QT, was was that -- say that completed Could a that, that's helpful. clarify you that offset? and the you Super can up. on Is study

Reshma Kewalramani

pharmacology Yes. we in Nothing our a study studies standard is the clinical around. QT is to -- for we report function. do cardiac new It's of that there. This do one the

Susie Lisa

Jack. please, question Last

Operator

The last from Terence question will with Morgan Stanley. . come Flynn

Terence Flynn

does on if for to file it's stands? foundational Can not. comments Or just you is positive that a Just where to randomized -- you with is you're for that mean say, trial wondered was it comfortable means. foundational still that consensus measured just you of approval III want do means, exa-cel. let's and your could if, what wondering is one going Stuart, that clarify data I What acute somewhat on year Phase launch? Or clarify situation XXXX trials? And positive theoretical other then the you the be X I being more controlled the need set? think where

Reshma Kewalramani

is this Reshma. Terence, Yes.

it pain, then and to the on acute comment turn me I'll Let over Stuart.

goal file moderate-to-severe the of a close pain having I'll our have at and very studies. from are to We program a goal And broad to set our the acute X it results just label. Stuart? to for is pain acute is leave positive

Stuart Arbuckle

like Yes. that what perfectly a for be look year, foundational consensus. consensus nothing with you comment lot we been Terence. It really about the questions exa-cel. actually what was is exa-cel do has honest about it on I you, a to exa-cel the launch tell asked will to for and response. dynamics a of with being We've for XXXX made And couldn't

what it and go and really that's begin, was patient remind was journey patients is, we people need provide when later to get the hopefully, that so to that regulatory to around important going and multistage year we obviously this And it get through journey context approval, some of thought exa-cel. try to to

in something, we've being responding to we as was launch the the was really to had about dynamics. journey reason questions I being to do that whatever reiterate the of commercial thousands my relatively beta large the of get patients thalassemia opportunity. this a severe of I a benefit So for there, potential at said lifetime very for benefit. which as tens a comment. are prepared I who remarks this journey it There despite want journey see well, could is cell and that sickle disease with long, end has the of the

about we forward and to So very we're optimistic launching. the exa-cel feel opportunity looking

Susie Lisa

Jack. Thanks,

call you give details, If for the backs. please

Operator

be today's has using for attending A now after call dialing you the (XXX) presentation. by The conference X replay XXX-XXXX access available of replay Thank concludes will (XXXXXXX). XXX-XXXX ma'am. or (XXX) the X Yes, today's event concluded. shortly code

You disconnect. may now

Vertex Pharmaceuticals (VRTX) 6 Nov 232023 Q3 Earnings call transcript