Earnings call transcript Q1 2023

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Operator

Good day, and welcome to the Vertex Pharmaceuticals First Quarter 2023 Conference Call. All participants will be in a listen-only mode. [Operator Instructions] After today's presentation, there will be an opportunity to ask questions. Please note, this event is being recorded.

I would now like to turn the conference over to Ms. Lisa. Susie ahead. go Please

Susie Lisa

Financial My President Susie is of you quarter to all. the my and Senior welcome XXXX first our Vice Investor to Conference name evening, Call. Lisa, is as pleasure Results Good Relations, it

remarks, call Operating Reshma Chief Kewalramani, have tonight's Charlie and Wagner, and On Officer. we Stuart CEO President; Dr. prepared making Arbuckle, Officer; Vertex's Chief Financial

be call. and the We slides as will this you a to recorded being access webcast on The that website. is recommend listen call our available replay you

We forward-looking to release Commission. the make in discussed statements will with that on Exchange press are the in this subject and today's uncertainties filings and call in our risks and Securities detail

future those Vertex's on basis. medicines, the without based results cystic call this note outcomes including management's will non-GAAP current limitation, are statements, that assumptions. on financial select on These also events and I regarding would performance guidance a materially. financial we presented could Actual that differ are and marketed Vertex's and fibrosis our evening pipeline review

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

I over turn the Reshma. now to will call

Reshma Kewalramani

with XXXX, today. product and Susie. on strong call Thanks, for have XX% versus We're grew a revenues of as to opened joining pleased quarter all, you start first XXXX. the quarter to CF us Good thank evening, global first the

Exa-cel to BLA secured for submissions rolling sickle five the age. and two U.S. for U.S. approval and we beta TRIKAFTA disease years completed thalassemia In in addition, cell patients of years

exa-cel in launch total, sickle stage five five. in VX-XXX past and for transfusion-dependent multiple proof-of-concept in mid the are eight we triple five late-stage as at cell because areas, we disease CF, In beta our slide depicted vanzacaftor the six development, which programs have or in of disease in now exciting and programs potential launches pain. acute and and on five see time especially years, goal, an including Vertex is with near-term thalassemia, Now five within

and of investing pipeline of capabilities. buildout the to With this trial drive we're compelling continued opportunities, commercialization breadth accordingly progress, success, clinical

eight clinic, type and strong a balance commercial franchise, addition, use strong advancing patients programs and and years through the sheet, in a NAVX.X talented well-positioned CF for deliver the uniquely muscular for and Myotonic agents and gentler as dystrophy preclinical exceptionally an durable for a Vertex next multiple beyond of In is team, opportunities, near-term and pipeline, conditioning disease pain, with exa-cel. wave already advancing development, dystrophy, committed never shareholders come. including X, in broad innovation areas Duchenne's the With been to to has rapidly for

I'll that progress details turn With CF. R&D with of overview, starting recent the to

SKYLINE clinical vanzacaftor and in-class as next the above, years SKYLINE enrollment has in XXX six is RIDGELINE Enrollment patients two progressing XX Phase trials and known patients completed as in is Our in rapidly. ages known well. also XX advancing its triple combination and X ages XXX study to

at study We we RIDGELINE and continue SKYLINE the end the to of the studies share, year, the time completion SKYLINE of this same as project completion approximately of the anticipate I'm by studies. now to the pleased the

recently chloride triple vanzacaftor bar high to-date, have into the we to restoration the on of of robust the clinic totality based transport set Respiratory very the Recognizing as the have with than triple HBE with evidence TRIKAFTA. vanzacaftor showed by high which greater translation the consistently Medicine. our from proven program Preclinically, have expectations and assays bench for the was in TRIKAFTA, generated Lancet reported

seen with vanzacaftor TRIKAFTA. clinical greater X Phase has the correspondingly and function program, triple sweat CFTR chloride that In drove lower of the levels been

for along dosing. once-daily enhanced has vanzacaftor the with As the such, the believe of triple potential convenience benefit clinical we

to expect lower vanza we the carry substantially burden. royalty addition, triple In

developing VX-XXX, Moderna in to pertains X,XXX CFTR portfolio CF for more our CF patients that the in from cannot we're study partnership modulators. who CFTR with benefit therapy important Another our than mRNA

VX-XXX of enrolling study ascending study portion the initiating dose SAD or study are this and the the ascending actively portion the SAD the have and dose single and We year. of dosing We multiple initiated of anticipate CF completing patients.

our Turning the exa-cel and both promise of now guidance, to CF as sickle quarter. one-time editing diseases. editing prior functional program treatment these to first be This to Per our for end BLA cell the be most as the commercial for TDT approved, submissions completed be disease and U.S. advanced is the for the cell exa-cel, gene expect thalassemia. next beta cure a at be sickle in to our exa-cel to and dependent outside severe last our program gene transfusion holds we we well launch. disease potential our of CRISPR-based

await now filings acceptance PDUFA and We assignment of of the our date.

in EMA requests and Review, FDA result are and by few We under MHRA a opportunity the review for an on include validated a filings comment for Our Stuart if and the from filings both granted have opportunity Priority announced, MAA launch exa-cel of will those months review. which minutes. eight submission. Internationally, previously our our submissions, preparations just market exa-cel. see further in as will significant time the

Turning and of novel NaVX.X properties relief addictive the or pain next opioids. side to of inhibitor selective pain promise that program effective VX-XXX, holds effects our highly our without the

a pain, with we the for itself Two, and pain. in in target. musculoskeletal and NaVX.X one, this VX-XXX NaVX.X positive acute acute, pharmacologically neuropathic, multiple predecessor given, across our VX-XXX is have and inhibitor validated proof-of-concept outlook confidence with genetically have program, We results

Phase trials granted for last year designations been And program VX-XXX already has and progress the initiated We have II substantially development and pivotal three, three VX-XXX Phase across enrollment studies is concluded. the continue nicely. Track Breakthrough U.S. X dosing pain in pain Therapy Fast Phase our in acute with X and to we the positive acute to similar X

home. including goal at of broad settings, moderate-to-severe have discharge the care the enable studies care support usage acute our to across that designed pain would a site post These multiple been of and in and prescribing label

acute near-term end X towards next continue Phase program the of the pivotal anticipate this opportunity. pain potentially We of or commercial year completing significant beginning another creating to and

a to peripheral dose and I we this twelve-week X a continue of in study of completing to proof-of-concept patients study enroll or year in of we also am dose-ranging pain. anticipate neuropathic neuropathy, pleased X Phase addition, In peripheral towards beginning form share, diabetic this of VX-XXX end Phase the next.

were first of England disease Medicine. publication Transitioning to for the with kidney or results now of pleased March, VX-XXX, of the the very inaxaplin APOLX-mediated Phase the underlying AMKD. target Journal the medicine cause potential we New or In X in inaxaplin to

the coverage in the the see editorial England data this by was underscoring study. We the the Journal accompanied feature Importantly, paper behind of an on Medicine importance medicine's New as and inaxaplin of potential. and the a science the

the potential to the XX-weeks a if in XXX,XXX multibillion-dollar ranging basis portion U.S. and treatment seek the remains track serve approximately in of global a patients the unlock to pivotal to see Phase which, with X, dose Europe approval to accelerated market preplanned we on as The AMKD interim bring X this U.S. treatment, year. inaxaplin, complete first-in-class opportunity. the With the analysis and a study has Phase Xb positive, study at Recall, of this could

America X.X million now one we committed and are more for medicine delivering North this are and a not, transformative, curative with disease. people Europe diabetes Type in to to Moving There alone, than if one Type diabetes.

same differentiated already one type producing which cells, proof-of-concept. portfolio, programs diabetes of fully all use three have which in insulin We islet demonstrated our the have

the that of Our portions or are target patients of part now In pleased receiving with receiving first study, study cells A and am the fully immunosuppressive B standard and the naked program I islet dose. both cell Part with A both the half program system. protect patients Part a dosed. share to staggered from VX-XXX, immune enrolled part dose patients the use full of and B to dosing the

This which with duration be year, in is step Part more faster follow-up the target timelines. sharing medical of longer patients in forward data look will ADA June. concurrently facilitate Sessions to C congresses in with program the next dose. Scientific full The at including from We patients the and VX-XXX should this treated

study look the forward plus we the have Canada program Site and for in in VX-XXX the proprietary study Both U.S. in and system, underway the in cleared. and dosing encapsulates program, same enrolling second requirement shield hence immune these cells immunosuppressants. are the is from and activities body's in that device or Canada, and CTA to initiation IND activation cells there to patients in Our a the U.S. I, is no designed device and near-term. the II cells Phase the both this

enable expanded cells Third, to we system. obviating will type our which one to collaboration another immunosuppressives. our stage In this CRISPR-CasX make This added cells. we to would and the March, for need us edit cloak from program hypoimmune the use continues path new agreement with the represent to Therapeutics research diabetes to immune into in the This program progress. licensing same CRISPR them

Lastly, in track. TXD of one the ViaCyte Phase program finished Phase remains VCTX-XXX program portfolio, the X study. hypoimmune on using in This Group ViaCyte line and a enrollment the cell dosing also is X,

with healthy Antitrypsin week VX-XXX is the Phase program, AATD that complete to VX-XXX. AATD VX-XXX is assess Let AAT this both and enrollment study in serum eight with Alpha-X liver continues in Phase which The for The levels. is year. X X me for our and will and program a properties X VX-XXX, molecule drug-like the projected Phase clearance functional for potency year. volunteer study study multi-fold better greater conclude or X to patients Deficiency complete of study both this to enroll XX of with the polymer next the class study Phase later This projected

With turn Stuart. over to that, the I'll now call

Stuart Arbuckle

to acute of focus patients CFTR and Reshma. in modulators combination also among our our term therapy, From maintain CF. continues eligible perspective, in pain, and the while a vanzacaftor on high preparing commercial all for reach near potential triple levels for launches exa-cel, be VX-XXX patients for already to adherence Thanks,

week, as in XX U.S. had new the two Canada, opportunity for the CF bringing commercial pus our with first for CF to comment our ages and the expected, FDA treatment of we TRIKAFTA the the on and a their we including given which first adds XXXX. medicines launch our approval preparations, have outlook I for quarter that term filings to our in to children we the TRIKAFTA cause results then driven exa-cel outlook underlying Europe first years Last address who six have then quarter to ages completed our a will will portfolio. regulatory now younger the years, confident I'll in remain children predominantly in the review in approximately first U.K. received U.S., for by and growth five growth Europe, strong for XXX patients, U.S., and and time patients longer disease. the We

CF in XXXX. growth Turning to continued beyond longer outlook. the term We see

Reshma at and in As XX,XXX our this more not people there yet who CF than benefit, were beginning treated North of mentioned, with were approved with Europe, medicines. the America, year, been but could Zealand Australia, New

of driver progress growth Australia portfolio CF key multiple also reaching and younger and for agreements the in This also patients Europe, new patients make our Beyond reimbursements. securing most additional younger Zealand. to in recently approvals, through spanning includes continue New we

studies, growth population versus the care. Model the of second survival in due A CF XX.X compelling year the therapy recently Fibrosis validate overall term projected positive is median projected which include increase survival. model health including for in published improved Journal growth outcomes of CF a related with of driver of and in and the portfolio survival outcome product long to were long outlook TRIKAFTA data real data Cystic to standard term the world

improved The to which our patients, opportunity therapy including an who option a medicines. generation discontinued portfolio treatment with triple, CF those new bring driver will to vanzacaftor next our our third for potentially provides growth and other be

from helped are who one fourth patients mRNA We growth is our modulators. who a in And extended therapy CFTR approximately cannot which VX-XXX, than for X,XXX discontinued our to that modulators. more CFTR estimate longer benefit that designed X,XXX drive of patients is existing term, have CF provide portfolio there the factor

transfusion-dependent beta Shifting with exa-cel, now and for cell curative holds thalassemia. which sickle patients potential to disease

our U.K., regulatory the our for of teams commercial exa-cel this the are approval dollar potential completion recent submissions multi-billion launch With and opportunity. the and U.S., preparing Europe, of in

neither Sickle silent cell beta disease are thalassemia diseases. undiagnosed and nor transfusion-dependent

have and launch XX,XXX a that severe carries initial of often chronic high disease sick the U.S. will focus disabling with been These patients the on entire Our their patients approximately burden care. a lives in Europe. most

opportunity the cure. have They could soon potential lifetime for a

uptake market and like are through to launch of therapy Our generation, including patients, insight their product exa-cel. a providers, engagement the perceptions preparations potential informed direct by research and and with of extensive gene understand payers

phases. shown enthusiasm patients. provide and Starting someone interest with for can therapy to be key The strong all begin therapy the benefit in for treatment and period by by them, are collection where patients exa-cel summarized and journey right by genetic could pre-treatment stakeholders encouraged that are referred to hematologists We genetic receive patients. three for a for a One, is decide their transformative process. the authorized cell the center an

experience treating of than their want Two, options exa-cel learn cells, and genetic myeloablative cells choice three, therapy the is is a their patient and the a followed patient vast indicate conditioning, research the manufacturing then and believe is physicians. about where where the patients lived balance, for treatment edited their treatment through them. the more a sickle successful through future Survey engraftment. majority are quarter receives period and And right that from patients product. edited drug exa-cel strongly become for cell own Of data the the beta and to more thalassemia period

providers, With approaches approaches differences a gene preference recognition learned lentivirus. various clear edited using over genetic there between for a exa-cel and we've is strong the gene therapies insertion of like therapy

over a market of gene therapy editing approach that providers research Our mechanisms. other approximately suggests gene XX% prefer

focused treatment initiate systems and to ensuring upon experienced. Our centers the best both and for strong patients for authorized toward support administratively patients providing the exa-cel key we on are ensure and made providers teams journey to progress are logistically Accordingly, access possible treatment prepared approval. have to treatment

to highest Payers engaged of located those key important of process. in patients. Approximately in process become prevalence U.S. in the targeted focus. Similarly. centers European XX XX are another all are four sites markets, with in ATC ATC areas all an actively the area are

in and and government We commercial Europe. are and the payers actively policymakers engaging key with U.S.

First, payers with the regard to commercial in U.S.

focused in including in patient Our dramatic and and transfusions are patients. sickle need reductions for beta cell profile patients the of and clinical hospitalizations for conversations on disease VOCs exa-cel thalassemia

are cost patient where exceed populations in that $X We exa-cel's lifetime confident value payers million. can will care current recognize of

We XX% patients to transfusion-dependent and working insured. cell for approximately who insurers with disease are broad ensure the access sickle of are commercially thalassemia

Now procedure to access to also already payment Medicaid is and in disease to payment for thus, ensuring place Medicaid addition exa-cel therapy are by in ensure with the reimbursement launch. that the government therapy, cell many a therapies with sickle Encouragingly, to patients of agencies with payment to models states the access the working gene continue cell XX% Approximately of have and we patients exa-cel. U.S. providing been the are transplant insured policies for and state payers. agencies already we've separate the adoption for in costs. a engage state We severe recognize around broad procedure ahead element to separate for and the critical

CGT budget and of gene approaches and be model. approximately access year, their this for access CMS's and In accelerate demonstrated access the addition, or broad through which Biden delivery demonstration the of administration exa-cel ensuring innovation novel in CGT Therapy testing February the announcement cell sickle commitment patients. intended to billion and the to Cell will for called through Gene a total $XX model like is a therapies project methods CMS center, exploration to of cell Medicaid and administered of access disease enhance The

payment delegate to important coordinate pathway The a or including Vertex. CGT OBAs in therapy models like two facilitate cell state for gene facilitate and Medicaid access agreements authority overtime will by model to CMS agencies manufacturers with ways, creating and this outcomes-based to innovative

the of access therapies inpatient underserved outcomes to in need the therapies as hospital access the We based severity bundle. understanding populations. and diseases view requires the transformative of from important model confirmed arrangement potentially separate CGT patient the any these indicator provide addition, to And of historically an broad has payment for for inpatient CMS these

activities calls. secure people living exa-cel, with you extremely for to outside to are potential to look We CF. and launch progress discover, in on all of years commercial our further for develop. forward on transformative excited our looking about continued you preparation and updating many launch future on first to also medicines the forward updating I'm our CF access

now turn I Charlie review the financials. to call will the over to

Charlie Wagner

quarter of our growth profile results the of Vertex's demonstrate attractive strong first macroeconomic Stuart. Thanks, performance consistent regardless conditions. XXXX in and

First strong by was billion. increase to $X.XX revenue younger continued as quarter XX% label year-over-year in U.S. uptake as well Growth of outside with led XXXX markets year-over-year age XX% increased on a groups. the into TRIKAFTA/KAFTRIO reimbursement, achieved recently extensions

with year-over-year U.S. revenue consistent ongoing grew CF X% performance.

SG&A XXXX. non-GAAP $X.X results to charges, first XXXX. the include just IPR&D, of to acquired IPR&D quarter quarter XXXX acquired billion in first of $XXX compared expenses R&D, and $XXX were XXXX compared of $X in million combined million charges million QX, quarter the of such First

XXXX our Therapeutics collaborations with First of IPR&D and Entrada which collaborations, expenses detailed internal XX, and DMX resulted one new Type gentler CRISPR slide our Therapeutics quarter agents into ImmunoGen innovation efforts diabetes. from several for expansion on with including relationship augment as conditioning the

Aside growth and resulting includes external by advancing investment across and IPR&D which our as stage was the charges, assets acquired in higher continued operating expense investments late driven clinical mid research eight innovation disease areas. different from our in expected pipeline, in

investments example, acute potential for in are and triple build-out studies as vanzacaftor towards pre-commercial in directed other pain, one as and activities exa-cel CF, for diabetes, VX-XXX well these near-term clinical the launches. type For

patients to Given the for to multibillion-dollar invest appropriately. programs, our potentially continue we market and transformative benefit opportunities will

the income First in of quarter to was $XXX quarter in the XXXX quarter, first compared operating million non-GAAP billion XXXX. $X.XX

quarter as remain cash our per generation sheet $X.XX. strong. flow investments, share ended in were We First and cash adjusted very balance with earnings the and quarter $XX.X billion

to guidance. switching Now

our on XX. to changes financial there consistent XXXX quarter detailed our first no as Given slide results our and execution, are guidance

to guidance year-over-year. point that, continues to our X.X inclusive representing expected an revenue to We revenue billion program. approximate X% X% to Note headwind to expect our $X.X $X.XX this billion, foreign risk include guidance product exchange of management growth, continue of growth percentage

mentioned experience our in reimbursements the have products Note to in February, reflect is CF, revenue we patients XXXX revenue inclusive our to revenue from of too we As only. U.S. XXXX and guidance TRIKAFTA years of expected product to like U.S. outside fibrosis visibility cystic ages given approval new the approvals early five guidance new range, that in strong which two continues

to Exa-cel UK approval as are still potential in EU, in and launch guidance included dates is US not and determined. be the

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

now non-GAAP year. million upfronts and XXXX Our operating BD the or milestones expense guidance recently completed $XXX versus at approximately the includes million the was of beginning previously of from that transactions anticipated existing $XXX

Our more our allocated and are development multiple continued stage to expenses than late of progress significant operating programs. R&D XX% the clinical and continue be to funding mid

near of funding capabilities are expansion opportunities our we Additionally, term potential. with our programs market anticipation in represented the launch multibillion the dollar commercialization by of

Our to XXXX guidance XX% non-GAAP unchanged. for projected also XX% full effective of is rate year tax

in revenue externally, industry our In also invested of to programs term Vertex first proud our growth, delivered the culture closing, and performed XXXX. We're innovation long and drive pipeline. leading internally of strong exceptionally continue well believe success. quarter we that diverse across accelerated will We of

progress Corporate XX. disease are our highlights to mark progress to areas. A We continued in milestones Responsibility on multiple from in we slide our And XX future look-forward on calls. our anticipate continue XXXX, found our further important to we updating as on recently you advance few programs as highlighted on slide published Report

And I'll the begin to Susie Q&A period. ask

Operator

Goldman first We Richter session. with come And from Please Salveen will go the will [Operator question-and-answer now begin Instructions] ahead. the Sachs. question

Salveen Richter

for taking here. Thanks be you a to With administered patients just treatment need three launch, speak question. occur that to time for the will patient your steps the to regard to overall discussed the after for my be that exa-cel in Could required to approval mind you drug? Good afternoon. get actually

work. then here foundational but secondly, doing insertion some competitor the time, done work And taken has gene they've clearly it's payer been and

the And all done they've front, think the the we also R&D do how work on as in but wondering as context context line this launch SG&A well. the is that not SG&A through how read year, about what we well? the progress launch, from launch might as to next to inflect this of just Just you. pain the in Thank and of look your

Reshma Kewalramani

exa-cel ask much about I'm very launch. for thanks the Salveen, bit the a the going talk to question. little timelines Stuart to Stuart? and about

Stuart Arbuckle

my takes Yes, months that beginning remarks, relatively to prepared so from Salveen, this as is extended process a I outlined multiple end. in

stay would nature the patient the important the if therefore from multi because has so the go are most and an a to through are of very because deciding hospital, that's like ingrafted edited said, their is I whether the where once they then a from that's is has approach into significant of schedule infused step to the they one see have they're is genetic very, life, As they undertaking with conditioning patient the undertake they myeloablative they that obviously product loss obviously in and with which to followed for when before very that want released hospital physician last their the the importantly cells and manufacturing week their to a that with individual. the mobilization that

potential is of a the payoff cure multi-month to get course, they're lifetime process. But, that for looking there

been schedule exa-cel. into then a need of So last from if multi-month life. it a to that to step Hopefully, benefit with their is one, receive obviously they process lifetime

both course, the of interaction disease healthcare In sickle and has payers, broader with that. cost learning we're by patients, families, of what system the the very their marketplace from on thalassemia payers associated terms we've the cell the from it we had burden severity encouraged reaction and their and of of more transfusion-dependent with understanding significant incredibly and do the both that

post as that is product there that the possible and to to is us try exa-cel nearest Also, that to how be very and to exa-cel possibly their positive profile encouraged appear to with by working access they has close we're reaction approval. ensure

Medicare. continuum, the both So importantly there we're really very and from and encouraged population commercial very response across Medicaid for payers, this by the

a on SG&A question evolution. had I you think

Charlie. handled probably I think best that's by

to So that you Charlie, one? want do take

Charlie Wagner

thanks. Salveen, Yes,

advancements not to of say we've reiterated I'm in I XXXX the you that OpEx course, our going XXXX. comment to Even expect, on is the very driven late that that OpEx R&D. OpEx the with of our in can are XXXX, few to got is will programs that pipeline, including invested much mid Given the stage majority, close XX% have multiple about in in guidance, we've and in by just growth a commercialization. over

ahead, but would more Looking expect moving shift low. commercialization, significant over a of medicines could a burden that importantly, specialty us we SG&A time. spend, little the profitability our model towards populations, It have towards business to you with commercial as in always programs mix see bit the on of be focusing on drive transformative ability quite gives we

Salveen Richter

Thank you.

Operator

from with David The next go ahead. come will question Please SVX Securities. Risinger

David Risinger

thanks much, thank and you Yes, the for very updates.

talk you in for was about if chronic your I hoping vision pain. So VX-XXX could

profile pursue do it So Phase is, development, what might But obviously, in X Vertex product? target in see trial, neuropathic as development the And succeed studying well? how how X interested you're consider in DPN. I'm you pain pain Phase you you product you. specifically, musculoskeletal your for Thank assuming that would in the

Reshma Kewalramani

Reshma. Hi, Dave. This is Sure.

market We Let One of is me it drug and chronic two. as the now as put back one acute distinct instead market, for see like for to we And pain. aside actually it. I'll take that see that one seeing I'll come three VX-XXX. for you. a areas

by we are that X that in we pain. the completion interest update today's where is in this end In we area the two, year, study of subdivided Phase of now immediate projecting on are the and musculoskeletal The proof-of-concept that Phase next. X early second first call already let's of And it, market being which chronic neuropathic our call pain. into the being,

already market that fully into musculoskeletal and that fits pursuing one, effective pain market. because, VX-XXX, very the We molecules model, see need. and form positive was say, say a pain. to NaVX.X specialty it high are very first And I but are of that is will rather VX-XXX it taken inhibition there expect conjuncture on we've general because commercialization in a Two that unmet their product I that also be based musculoskeletal as substantial a we our that well. as not for and sets because

So of in portfolio pain is We're this acute market. the will X. That that as program of next beginning terms first pain already end first. towards way the completed I of the and progressing our nearest in opportunity pain I be see year, phase to see

program, and a in I I will the pain medicine Vertexian neuropathic primary be that get because certainly, do as but that to and way requires see expect patients pain. it care will, help, that XXX work we'll believe musculoskeletal commercialization if don't I the a find outreach it disease it can Second, enterprise. will Vertex but through a to a

David Risinger

you. Thank

Operator

Please next come will from Mohit Fargo. Bansal question The with ahead. Wells go

Mohit Bansal

question on thank Maybe taking a on more for exa-cel, question. you Great, one my question that.

it works. amazing. to a bit talking opportunity on to maybe about the about talk So bit a we initial safety patients at show-me worried there and a will doctors, patients and be early you they I pretty it's know in But XX,XXX little the talk as also about you're -- at story. community looking clearly U.S. the in investment mean, is and and drug little I I mean X,XXX are point, opportunity, excited the XX,XXX they're about this when

there. understand we to be to part happen you to would where do So prescribers, talk could think missing important you would need for of the help us terms what how an think launch and disconnect when do And is payers Obviously, what like disconnect is? disappear? you -- what steps are in you

Reshma Kewalramani

Reshma. Sure, Mohit. This is

and has there your confidence significant is fact disease this full a that stakeholders showed and that and the is see to that that from of payers, product. their patients, we've is potential, in unmet show debate potential we medicine, potential. comment about no curative data medicine can in really from really There on I this the I'm for understanding exa-cel regard With the heard general that that. to to need. the And as curative transformative, think what real enthusiasm very on has to about high of what question if do not This of all from no stop. therapy is we've our potential? date offer going ask Stuart have level I

ask and we What the see say there size. sickle to as are also U.S. people and I'll with And XXX,XXX beta-thalassemia. market is Europe, remind Stuart I'll in what the cell that

We based with Stuart? program, conditioning set this are full we're XX,XXX. not targeting busulfan targeting that exa-cel

Stuart Arbuckle

Yes.

the per the who and Reshma that who those for very patients of patients XX,XXX, enrolled of in end sickle multiple population year in spectrum, thalassemia those the crises population. are cell transfusion-dependent trials. for disease our clinical in essentially, year similar As sort said, These having were the our to patient are the transfusions patients are occlusive those multiple severe

So whose patients disease and are a ones, of impacted loved health very -- impacts to really ability a care their very the and work, life, disease, system. very, it have these who all impacts they impacts of and very their significantly their by significant also burden significant for aspects very, lives are their burden

very potential So looking we've onetime clearly is forward therapies and the unmet something well in that groups, curative very to. need the payers, that understood, both much discussions is for the had physician very, with are very, patients, people

around questions innovative new have any these safety there to and be transformative I new are that and as product, with will things think are some like with any going really always technology therapies. people

can seeing and the from this enthusiasm And a we're being of about optimistic course own extent, experience. multibillion-dollar very certainly, live that high, some very, of But very over for the with their stakeholders to some all something time we're is and opportunity. that's solved people, level get only

Mohit Bansal

Thank you.

Operator

Meacham Geoff Bank from come ahead. America. will question Please with go next of The

Geoff Meacham

Thanks Good the for question. afternoon, everyone.

vanzacaftor triple. Just have Stuart, a couple. the on

to as curious Just on updated obviously, view differentiation, beyond dosing. your

to have that argument discontinued have a switch patients well doing you Just on the responding and triple? would TRIKAFTA, the vanzacaftor I mentioned the in logic be prepared been or that what's remarks -- is the TRIKAFTA, then think assuming maybe new patient to

And then pretty billion and BD. or almost have good need for you secondly, urgent no Charlie, $XX real guys pipeline in cash, Reshma,

that of areas investments are cell a need So of I'm we curious probably think forward? build-out like about that therapy? should capital more Thanks. about in just or uses going cash gene even

Reshma Kewalramani

Sure, Geoff.

comment to then tackle on and Stuart? capital allocation. vanzacaftor to I'll me Charlie Let ask first ask Stuart

Stuart Arbuckle

Yes. Hey, Geoff.

just people is people carrier can. cystic everybody our many possible vanzacaftor, get of to on chloride, goal sweat be it the to believe prevent can we as Carryovers we to developing in do fibrosis transport in essentially as because we we CF we as levels So remind pardon as chloride can me, as able to today. if call, know on will we of that,

so our As data we've it's you combination, to from levels as we with bar. with even to a establish we can able vanzacaftor get very, higher in-vitro you triple than very even assays, also clinical believe know, but from know, high TRIKAFTA which, been our the

when vanzacaftor. And we plus data the so with is ongoing, looking do XX seeing TRIKAFTA to study obviously, XX also to that, aimed just to but have that both And to we out. read X those compare the studies forward in our is were

going to those TRIKAFTA, As I think you new for identified, sort I be option, now. who our CFTR one combination. there really think prepared what of patients modulators. a vanzacaftor going superior are my modulators discontinued we there's the people patients have existing the in remarks, If exciting currently to being CFTR efficacy of it's be treatment two one X,XXX either with those of of And opportunities are see to I really globally, treated triple our is treatment about or with for for said who

they Actually, efficacy. our is to responded respond if question to major the medicines. why they might is haven't other not lack Now discontinuation vanzacaftor for of reason your

for adverse like about Our tends products in efficacious patient. things are amazingly every be events. It just to

it's I CFTR who people treatment discontinued previous modulators. have for one going so may to think our to do an consider option of attractive And be

data and that to also studies enrolled seeing very option But are patients be so and we're fully forward seeing in even for all I the forward to and this. TRIKAFTA. be even obviously, to So the to that ongoing, better We're has looking influential do will vanza treatment potential of many think an looking the data.

Charlie Wagner

And or change then, priorities. Geoff, on no all capital at allocation, our in strategy

externally. and continues Our both investment priority be innovation, internally to in

active we quarter. with the that first BD saw in You were

we've if I that's While in done over XX or project think look have we're it back forward so, focused. to may be BD transactions of and months the a the cell gene only place number that you true don't you last that therapy,

deals sandbox areas, program got strategy. and sense our make know, do we of agnostic we've that disease and are we'll fit modality the our and As you for a areas

out, so. an capital buyback been remiss at our five years be strategy. I Lastly, We've for share program do be allocation have we to well. about or I to point program part guess would continues ongoing as that And of that a

Geoff Meacham

you, Great. guys. Thank

Operator

question Company. The come go from will next Please and with Nadeau ahead. Cowen Phil

Phil Nadeau

do to A want acute questions. procedures bit the Then maybe for how approximately with Can product about and What the group ideal for good energy And more a pain. you effects? per your profile? talk who a achieve our how follow-on as be in does target VX-XXX many thanks Thank you level you Hi, have? patient taking target non-opioid to many would year, patient side that could on of that, patient few afternoon, population, option? you. discuss

Reshma Kewalramani

Sure.

potential. comment numbers ask both of on comment profile a dollar Stuart and bit, the the me target product on in and size, Let to patient terms I'll then market

the So Phil, if that and a doing soft X, Phase substantially X. we in is to is a a we pain of form similar recapitulate which for would we're hard home abdominoplasty study form a that tissue what a say, Phase model bunionectomy be recall, saw you'll of to as model, in in us. tissue a as That results run

terms that peripheral profile is run. XXX to ask size quick I benefit I'm and in of patients. pain way risk is the Stuart nervous mechanism that's absolute is on is durable, say by an not on and comment to efficacy, mean is centrally that a works to that attractive the going What action, by quite market and home system, of relief potential. addictive Stuart? no That dollars

Stuart Arbuckle

Yes. Thanks, Reshma.

we've Reshma are of and study for just to types, have to in pain, In the secure is label postsurgical relatively not which related been pain. moderate acute the So other us to procedure we for. pain to acute that for designed, is a severe contrast approvals we program got which number label recent that's a commented on, designed linked certain seeking to

a we're seeking seeking. with our nature for We moderate the surgical are could post severe the large, to that's not pain of label that studies, but indeed acute and label is be

in surgery, are we the sizing that. controlled of acute not broad it those billion things very market utilized prescriptions number be or pain, can't despite When procedures of and based of number the billion US therefore, like with very, being you XX-plus U.S. look percent market at it are is cheap, So which opportunity alone, NSAIDs market $X a the on it's roughly treatment is of kind for today. a medicine treatment days of generic, and that the the the X.X of standard in

a see very that for profile very, if opportunity we commercial has it Reshma VX-XXX So the described. significant

Operator

Jefferies. come ahead. will The next Yee Michael question with from go Please

Andrew Tsai

Andrew Tsai for all Thanks for the Michael on updates. Yee.

acute wanted study. I up So to follow chronic pain and on the

ruling first question And it have be to when think and year. year this Thank later expect understand us do see data? acute that then scenario? for Moderna. program should up pain vice a versa? for later this could CF out longer-term the we curious out X So we pain this coming question just we We possible But with set is, read-through to your you. should can have that is chronic second or data you Is SAD is come that Phase

Reshma Kewalramani

Sure.

a modulators. active were the they and our portion the right that question on the is day to to tell us of Let VX-XXX we who With did the have that's with It It SAD it the going mRNA used I'll to simply CFTR program can't ongoing that patients and the therapy. KALYDECO with then knew they approximately benefit a Moderna, now. back SAD X,XXX last first, or MAD example, from or come is around take When for TRIKAFTA, we patients for on pain. program regard me first so program from program. placebo

So and will rule be whether lines to SAD the year, with we time know year. plan be done we can't well have we efficacy. I we for, terms this can But expect out and you MAD the this expect of in when what into to

X to I chronic read-through musculoskeletal. or do acute from X. those could the and expect lines time I see the say which With from studies neuropathic forward What in see VX-XXX, to read and class. So to already pain I we're also looking studies from you to neuropathic regard the versa. acute there, NaVX.X That showed to. pain see really are Phase is read-through through don't vice the Phase efficacy And is what

program pain VX-XXX pain and study neuropathic pain, Phase the where results, in So across I'm VX-XXX as that's VX-XXX itself types. and Phase the precedents, in program neuropathic I study look for to acute three results, VX-XXX forward X X dose-ranging range looking the to the the

Andrew Tsai

Thank helpful. Very Very you. clear.

Reshma Kewalramani

Sure.

Operator

The next go with Will Pickering question from Bernstein. will ahead. Please come

Will Pickering

question. thank Hi, taking you for my

pain. one Another on

be time For CGT with treatment does the time could about the reasons line very existing and for drug to optimistic experience compare on the the cell compared VX-XXX? based model, expected neuropathic pain study, that access will on how this then for sickle launch favorably quickly program Thank you line you. your that to talk options to And exa-cel?

Reshma Kewalramani

Sure.

neuropathic think what expectations. how are we about first question our do pain on in VX-XXX The and

here pain expectations extends results, in the that reasons. you acute how are pain a in neuropathic multifold, general, with we I NaVX.X and and go when has that are signals the And pain. target the through As called I on for shared One, earlier, because these waiting X three high VX-XXX Phase program were for pain NaVX.X are have reasons that's the the grail signals setting been but channel, as propagated. often holy the to

something XXX the called also had with That neuropathy, small diabetic study study concept the molecule already the central That in VX-XXX, perception neuropathic pain. we're with and and Two, now, had diabetic same it patients, is neuropathy. that's doing neuropathy, fiber they neuropathic. for had to proof So that the pain. predecessor are for of patients delivered positive study patients acute the of is

multifold, molecule three, on went the success is is why with VX-XXX properties, has more and which another we seek VX-XXX. despite molecule potent to the And drug-like better

of all that sense gives have a and we together sort put You high that this. for for our you expectations why

had you think a I on second question TDT.

Stuart ask Let to that. on me comment

Stuart Arbuckle

model been not certainly with of efforts our it's implemented. exa-cel. on has CGT ongoing take would possibly to secure kind can. model, be fleshed and a for both of exa-cel going to to relying this close while access launch place complementary as only as announced, commercial to government out just obviously to on that that so approval we Well, consider for The be Yes. the payers, We're I and so successful and a then for in access

them. post As approval the in for and the continue to working extensive last discussions exa-cel signal been I prepared it up and is having how just relying CDT payers we through we've model What getting payers think is of to with though, few consider so I for underserved my and are with government the We're said of commercial months, on remarks, patient therapies both important and these a launch. do we'll innovative like successful access a exa-cel not traditionally population.

the past. indicator it's to that to So been real people has that these treated get opinion a patients of a want to of us, transformative in groundswell poorly medicines

Will Pickering

you. Thank

Operator

go Please question next from come ahead. with will The Fye JPMorgan. Jessica

Jessica Fye

placebo, I opioid you against Thanks. my comparator a randomized do about have Hey for commercial think the there's pain if arm. primary worse there. the Good taking I also efficacy or question an Thanks looks How commercial implications than better a evening. that believe trials opioid analysis have the but VX-XXX. Phase X question. on arm?

Reshma Kewalramani

this Jess, is Reshma.

opioid the arm, comparator program, and a arm. is but a remember of study, And study in you'll the study design, efficient for in You're the an we comparison, placebo X there to Phase versus study just that X a context. with not Phase is It X Phase secondary it opioid right regard was the had for the but reasonably endpoint also endpoint has VX-XXX design. like primary about the study given

I'll give it compares to of how sense you of for to will there put favorably, a press the treatment that that want magnitude what not was comparison. And and just the release out get point I to course, for the was if say we But it it you compares. effect you

Let me the implications. to it on comment commercial over turn Stuart to

Stuart Arbuckle

Yes.

very, all opioid-like very So you then we potential, a something very medicine, would option the obviously is superior to for a baggage, additionally which attractive tell opioid, them addictive treatment that, offered beneficial to and including without about. be today, that of If were efficacy we you would Jessica, pleased had probably that people they if but would that be an be considering. associated

if option. away And the treatment you without is want than that I attractive a option. think I anything However, to very efficacy opioid-like commercially baggage, have successful very don't you other a take would that very, be

Jessica Fye

as just there eye out And a I for be Are an Thanks. effects if X follow-up. we could seek any side we mechanism with should this readout? the keeping that approach Phase

Reshma Kewalramani

Reshma. this Jess, is

tolerability, If data, risk and and very you look abdominoplasty look safety very at profile looks at bunionectomy, benefit it well to both you the compares X in good. the specifically Phase And when placebo. very,

So profile. a really effect good-looking side

Jessica Fye

you. Thank

Operator

question next will The Bristow come from Colin Please with UBS. go ahead.

Colin Bristow

in a VX-XXX. to on second What just that, of ADA then we progress pipeline data for progress. VX-XXX. expect with cohorts? give on numbers from the on any and filing? And And good Could and to therapy. on us expect regards in the see CRISPR-based IND quarter congrats color any the us Maybe first And is DMD reasonable Hey, and to the more you then of follow-on XXXX? at it patient terms should the clinical all afternoon and

Thank could of Now terms us or cleared, any you. the you materials the of device? in is the the IND give design more color

Reshma Kewalramani

couple Sure, were Colin. there. There a of questions in

three around then portfolio Vertex, With really to I'll and talk X we the to type regard DMD. have first, Let X type about questions programs. diabetes me diabetes come the at back

VX-XXX. has share and and results and at congresses multiple the where Part pleased program going very program we're to starting the the with I'm year, B, naked that the that that's to A completed The through program completed, first Part immunosuppressive. That's is uses ADA. That's share the cell off-the-shelf

and of and more and What Those than that A worth expect you the And because say and you Program cohort there the we've that, that's two for longer-term VX-XXX of a -- the the more which are the for patients data cells, as full hypoimmune is same self-plus those are to year. which to patients cells foundation important are same data, is shared out program, Part device Program program. that know, X, is is should X, some foundation including you there cells really this. previously the than parts Part expect are B should patients'

than cells share the We've we I'm the details is producing. to any fully having got and that. the system. not differentiated is the insulin past. with first the second And The a immune that going program, more immune have in to system device. we In evade how are XXX made

material, third proprietary say a and glucose of device of of insulin release sensing as particular it's that structure the that and cells and date that seen And what animal coming as is the has worry oxygenation fibrosis. and into no system. models transport without so and a I immune But geometry, edits well allow large cells with for edited we've the immune of evade in nutrient to the the the will cells the course, system from that small,

data and With haven't CTA the IND and to taken different trial regard the guided to clearance than that start future. approach many DMD pleased is our With dosing in the field. patients when from are to we we but the regard we program, very you'll can approach to see expect being by we VX-XXX, with the in the of recall the near that,

goal what if of our There the very mild near that a is have human of disease. full-length or you That have our you full in for we microdystrophin. approach its which say the excited length IND is see IND-enabling get program. to year. and track we validation to and approach complete genetics half those on dystrophin, grounded the is such is version studies, no filed of the are towards the in this is using It and We're about is an genetic human studies program, second

Susie Lisa

Thanks, Colin.

us brings to that Just time.

So can it up, please? you wrap

Stuart Arbuckle

our and will use X Yes, XXXXXXX. today's or A of can conclude as our have (XXX) X event code you call question-and-answer XXX-XXXX well great session dialing conference access replay by concludes call the ma'am. (XXX) will after day. be today. a for as shortly That the available XXX-XXXX replay Everyone,

Vertex Pharmaceuticals (VRTX) 1 May 232023 Q1 Earnings call transcript