VRTX - Earnings call transcript Q4 2024 | Vertex Pharmaceuticals Incorporated

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Operator

Good day, and welcome to the Vertex Pharmaceuticals Fourth Quarter 2024 Earnings 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

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

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

to the turn I'll now Reshma. call over

Reshma Kewalramani

you thank call joining Thanks, evening all, Susie. Good and us the on for today.

QX quick in full our release. Before update diving press planned into like mid-XXXX, a I'd noted as and on year changes to provide earnings, for some leadership

innovation. transitioned this almost has Chief the itself then CF. be year, CBIC and Vertex's Commercial as Stuart led company career commercial And an with Stuart and from Commercial first, July since and years biopharma C of hepatitis organization [ establishing he COO. reimagined and in Officer, skill Officer after ] stellar Chief Arbuckle as will Chief great as then, dozen-plus and Vertex, at retiring X Operating to as on in the in XX-year Vertex a Officer

CFTR and of the helped and new modulators, CASGEVY all and and develop of the a of the at lead beginning has to into commercialization with way also through our KALYDECO organization been the has hound diversification through launches this all commercial the launches Stuart with He JOURNAVX. of era ALYFTREK.

work to and who a I privilege as first. consummate always developer to patients professional, had be the knows leader and excellent Stuart Stuart an have, puts with leads of poised, is He Anyone integrity with incredible has an uncommonly talent.

As seamless part for until Stuart planned carefully of our allow Vertex fully succession, which transition. on a at will is board July,

to this While my many Stuart take we still have he convey partnership for and opportunity deep for gratitude to months for has I want his that retires, before Vertex. done all

have thoughtful baton we plan pass significant in that a are very and confident enable been in planning, succession, to do Stuart very the from Thanks ahead. for are leaders on other and with to us stronger a mind. to senior the will execute position this we deliberate seamlessly careful experienced we long to how senior the time we always in opportunities and horizon We leaders so never transition

CFO very will COO am the additional on to Charlie whom take I as you of announce regard, of of April July In well, pleased and very that who all that on role know joined X. in Wagner, XXXX

Officer, disease commercial both and industry in range geographies promoted experience ago. years' I Duncan over Novartis X. in prior operational strategic, GSK at XX Chief multiple am and the areas American years to North equally joining roles XX of a wide across Vertex Duncan team Commercial of pleased and commercial and and to also the to longtime Head July EVP announce on McKechnie, be our that SVP will had

as well of side-by-side working with launches in all role time, CASGEVY CF architecting JOURNAVX the he which during played been an as the instrumental medicines and successful Stuart has Duncan the XXXX, of launches since U.S. our

has led health as Duncan the addition since America role the entire XXXX, of to the global his functions and head Vertex economics the commercial portfolio. global as across value North also access In well as

of at and forward Vertex, have as I'm continues bench steps the he I'm directly role outstanding responsibilities of CCO talent Duncan the working COO in as deep executive to joins proud the we looking into committee with and as July. Charlie and CFO and adds

execution set performance us our XXth earnings. us double-digit helped wrapped revenue year drive Fourth diversification. growth year continued of the up course the commercial quarter consecutive on and launch another revenue of to Moving strong CASGEVY in as outstanding for CF the

delivered We continue XXXX. patients XX% in quarter, growth representing fourth reach the more QX revenue and to $X.XX in billion versus

a milestone For to versus new product plus reached just year XX% the guidance year XXXX, billion, $XX of our over XXXX full billion of $XX.X and revenue revenue exceeded billion. $XX.X full

in ongoing for launch strong XXXX, we NDA to in recent December approved U.S. acute pain and base are on a moderate with our JOURNAVX and fifth we serve the next-generation this January approvals CASGEVY of on CF From driving approved the severe patients ALYFTREK, significant XX focused medicine X on expansion XX.

We of represents epidemic. an and millions efforts The of to public option oral signal see new non-opioid it first landmark, and Vertex, inhibitor the support is as the this important significant medicine health over for patients, approval JOURNAVX class in pain for for first to XX pain curb the as approval as society years. opioid

clinical many approval Our also privilege and is and made to program who R&D their patients for providers acknowledge have this and indeed and teams efforts in health it care thank possible. my the to this on years, worked the trials participated

global for for are we and focus commercialization and CASGEVY in X hand on continuing JOURNAVX ALYFTREK approvals working our waiting. these have and launch, broad the secure are teams keen patients access a and who recent to With reimbursement

years. have with to CF, over I approximately cell XXXX, in in-line the we potential we as coming In the XXX,XXX grow transfusion-dependent As transformative patients number of pipeline disease, patients the thalassemia. January, expanding therapies sickle advances, anticipate and medicines detailed of serve beta late-stage and approved the our significantly number serve the

acute approval serve thanks X more pipeline, renal JOURNAVX. And can of seek pain to with beyond patients add another just we X we patients late-stage and and peripheral including the that Now the million neuropathic moderate-to-severe pain, and XX on to million-plus then focusing mid- type acute million to certain with in patients U.S. XX more diseases those diabetes alone. than pain reach, the

development of X our we you on in track key milestones To studies momentum, for this pivotal clinical year. give a sense are

with First, insulin independence. us regulatory has for to completing year dosing I/II/III would Phase position and diabetes approval Zimislecel X which of the enrollment file this in cohort follow-up X in type study once

reaches the us Second, weeks povetacicept in to analysis of for accelerated once which interim potential IgAN, completing cohort in that treatment. enrollment cohort would approval file of XX position U.S.

cohort And accelerated the for in that approval file completing once of to potential cohort XX third, treatment. analysis for interim inaxaplin enrollment us U.S. position weeks AMKD, for would reaches which

in diabetic also severe both continue strong addition, neuropathy. neuropathy. In to VX-XXX, making progress we pain next-generation We're of the NaVX.X with execute patients diabetic peripheral the to moderate study and with in III acute peripheral suzetrigine Phase inhibitor

for indications into dominant trials X, like beyond povetacicept have of type VX-XXX II autosomal disease also VX-XXX this in a polycystic to dystrophy myotonic move ongoing study basket IgAN in medicines kidney in and Phase expect transformative potentially We summer.

base, broad geographic of the at our pipeline terms era Vertex of It's truly diversification our and presence. exciting revenue an in

in update detailed clinical we January, tonight the stage clinical the focus CF renal on Given I'll and update provided R&D pipeline. our

secure in Starting regimen, we modulator with approval are CF. working also underway while The international fifth ALYFTREK, to launch the is U.S. Vertex CFTR regions.

levels clinic As ourselves we ever we ALYFTREK patients and get globe, modulator CF the for as our CFTR in whole of With approval, to our CFTR modulator the bar work raised field the regimen we a the around are to regimen. function. with with CFTR patients already bring to mission that all each to higher continue as gains next-generation we normal

We shown improvement dependent be outcomes. in transport predictive CFTR modulator has other next-generation NG shown or tezacaftor. even CFTR and triple human than NG corrector; chloride efficacy. been ALYFTREK ALYFTREK even CFTR regimen of VX-XXX, X.X. bronchial This cell X.X. is clinical NG X.X a of greater combo our a potentiator; NG in have The to term In words, consists CF which epithelial the CFTR X.X VX-XXX, assays, regimen TRIKAFTA/KAFTRIO, highly greater

We clinic study this the a of regimen to in be in year. CF expect patients this

are dose enroll of cannot benefit MAD the study I/II the expected VX-XXX this for portion modulators. the later portion also MAD year. the and of to patients X,XXX-plus continue Phase Results CFTR from study from We of who the our

renaissance on see I now as in renal drug to Moving a what development. real therapeutic

renal We treat in the are potential X to hold that the advancing on cause disease of underlying severe therapeutics conditions. focused different promise

APOLX-mediated for AMKD. inaxiplin or kidney disease First,

is this patients X That completing enrollment of pivotal working As primary analysis study a our of AMKD. renal of study with alleles the mentioned, goal additional AMPLITUDE the say to AMPLITUDE cohort we're no comorbidities. towards of and interim year. APOLX is

completing conduct After reaches able will file to for XX in If weeks interim the of this accelerated we'll when potential be the treatment, U.S. cohort enrollment, positive, we analysis. approval

am study with X last month, in I AMPLIFIED to initiated addition, of we which pleased In announce inaxiplin type II a that diabetes. AMKD Phase including other patients comorbidities, proof-of-concept and a is new study

inhibition and action, frequency dosing volume preclinical antagonist and diseases. BAFF key APRIL route the high give patients cell-mediated of confidence small the autoimmune medicine promise IgAN diseases. with data pathogenesis and a of play povetacicept, of once-monthly and dual us in for roles Second, which as cytokines, pove's subcutaneous of plus date B-cell-mediated transformative in tool a clinical B the to administration This its other mechanism

now that Asia, expect the than to this we well this in IgAN pove of of trial in January, complete in underway. than have countries, report analysis dosing I XX including mentioned enrollment sites the clinical are more pleased more To we end, am in As U.S., and study Europe and cohort to the XXX enrollment year. in opened interim

And XX the this follow-up, we'll analysis. filing has Once conduct in weeks of approval accelerated the if for interim it positive, would potential U.S. support a cohort

stage there completion disease, Europe polycystic monogenic of or globally, the an people the are I that of amongst significant most yet and most in that ADPKD And are estimated common in living autosomal underlying third, disease the the renal no with common renal ADPKD, an Caucasians address in U.S. disease. represents program cause end-stage cause VX-XXX an treatments trial. the and additional disease Phase inherited is and ADPKD, nearing the U.S. are of XXX,XXX earlier we severe dominant where opportunity. kidney

to can It by majority characterized the of is kidney to a age lead function by variants ADPKD gene, with patients and proliferation life-shortening that loss the polycystin cysts to PKDX experienced encodes of the failure epithelial kidney the cysts. formation leads ADPKD which fluid or of by variants PCX PCX the that kidney of the kidney fluid-filled lead cases and and function The half ultimately are protein. of cells, failure. inherited the XX. X numerous increased Around in impair disease secretion These is of expansion that growth of caused

VX-XXX increase This function. potential progressive protein small The to formation decline and is cyst is correcting the growth. size arrest character folding kidney an of a function. in that by designed first-in-class causes ADPKD restore to target underlying assist has in the kidney cause PCX folding to molecule

patients of By XX% of to overall action, in protein-folding population. the of of mutations XX,XXX up about has its estimated potential mechanism at PKDX patients way of a VX-XXX ADPKD with subset or

full in to XXX,XXX CF, we the reach doing and serially over ADPKD. seek we're As patients with innovate time,

our complete to expect this the supportive study Phase are And results II we later VX-XXX, to I year. if Phase advance soon. For

that commercial over it to for update. a review, turn I'll With Stuart

Stuart Arbuckle

very Reshma. you much, Thank

know right very retiring that emotions at While having XX at and July, amazing journey the I last professional over the It's years. is personal from mixed I Vertex standpoint. from timing an to admit Vertex both in been

transformative to commercialize we As commercially on disease, and late-stage for today. sickle autoimmune never renal broad with clinical CF, a The we will more position medicines in transformative in cell patients differentiated successfully unique such diabetes, and from and very beta many business thalassemia we've our I'm strategy our diseases executed and pain, develop confident medicines, has than and better pipeline. develop are been and scientifically as

CCO. significant skills, opportunities. I'm highly these help heights. confident to as the He brings will maximize thrilled and outstanding new that decades have strategic every focused of I've Duncan also Vertex. developed our known rare to lead company transition and excellence leadership greater him combination results confidence people recruited the to right management Duncan for of thinking, operational we the have commercial a to I I'm and even organization outstanding talent to he

you of for helping engagement coming to and look I opportunity analysts support know months. to our and your many you the over forward now Although all July, this I will between to see years, and take and Duncan the I'd shareholders like get thank over to

comments early on that, including in the U.S. tonight X ALYFTREK, CASGEVY XXXX building continuing With the launch launches, the I the will in focus our foundation of of and of my pain. initial established launch global acute we on recent JOURNAVX launch

Once XXXX, full further and the to rapid grew the the We ALYFTREK patients, geographies. our to cause is now CF. in CF. we new younger rare CFTR patients results our as treat underlying in patients us taking for mutations quarter with expand again, modulators. enabling and approved to Starting progress of modulator fifth with patients of we strong delivered the reimbursement made year number eligible CFTR regulatory and CF

clinical early CF. to clinical current trials function lung further sweat CFTR for eligible has the standard care We were the of with which of noninferiority demonstrated with improvements patients ALYFTREK TRIKAFTA, as also chloride. on pleased in in trials by approval measured function demonstrated ALYFTREK

profile, approved not for with label. the become potentially new convenience of standard additional ALYFTREK the XX are believe for also sets the along in stage TRIKAFTA care an We once-daily CF. mutations ALYFTREK is the dosing of that covered by this

extends a into protection ALYFTREK Vertex XXXX our royalty for and for carries ALYFTREK. of finally, patent meaningfully matter, And from for burden lower XXXX TRIKAFTA composition

received We and ALYFTREK. for physicians from positive have feedback patients

eligibility simplify patients other convenience Early are our X to for including that who of modulators. and modulator, that treated with insights the not to today are for dosing enthusiasm being many rare those CFTR option once-daily expanded a a CFTR treatment XX show who new not offers have responsive of mutations the

switch these conversations and launch, frame with data the themselves to early In liver naive, of with days potential centers initiation discontinued monitoring the patients. requirements the are about familiarizing and

I'm have first all in these to already pleased interest that with seen treatment from and We report ALYFTREK groups the of have ALYFTREK. of initiated eligible patients, patients

patients As patients, we CF we think ALYFTREK. best believe the of will ALYFTREK, that uptake And majority the seek that for them. of for the that about confident is we are medicine

will expect to ALYFTREK the a be monitoring the for liver CFTR that of requirements in the improved dosing. short-term potential initiate We lifetime function and benefits considered context of the once-daily of

look the this the New and are reviews Union, United for best-in-class medicine European underway these Switzerland, forward including Canada, regulatory XXXX. to U.S., of markets potential in ALYFTREK, the Kingdom, in Australia Outside Zealand. approvals We in

for patients, It collections. a launch is regions. payers progress turning sickle thalassemia. and Now to with cell beta important enthusiastically patients all ATC CASGEVY, CASGEVY, our historic momentum and just and over transformative been has the the are launch by our and year onetime of which is treatment since cell Two disease received patient across policymakers gathering markers of activations physicians, approval

approximately goal XXXX. cells with As Middle we treatment collections exited to way the more first patients in infusions our of our activate And to globally. Approximately authorized have their XX already XXXX across than cell on regions well total in and XX East in all patient we both the have CASGEVY-edited centers, U.S. occurred January, reported XX ATCs

in continue XXXX, make Middle regulatory the approval secured XXXX, and East. and November XX, of Emirates. progress we exciting We on the Arab And approvals January respectively. to in in December KSA, regulatory secured of Bahrain In XXXX we United

region and We Qatar this are with working in Kuwait anticipated expand year. later further the to filings in

sickle in Emirates. now landscape, reimbursement payer hospital-based towards the are we with in Continuing recently United in in that reached Arabia NHS of coverage cell this sickle working have for In now disease. region, England we Bahrain, Arab patients Saudi and beta-thalassemia a with means patients to disease national coverage and eligible the access This the have England. reimbursement with CASGEVY cell treatment agreement

to in of Medicaid patients expand have are opt CMS segment, patients single may subsequently private the In million CMMI we the begin eligible States cell access believe either the in States lives the as month, as model, then last insurance, where to access U.S. Both further therapy or and over SCD TDT case agreements. to and And XXX which in negotiated through agreements. now participate for covered may gene access will through apply

on work are for build momentum XXXX in to in we beyond more XXXX patients We therapy opportunity. transformative to the we to get CASGEVY focused leveraging and and world. this for as foundation the around established multibillion-dollar this more

pain XX medicine to selective the first non-opioid days of profile. for effective post milestone because MoA, approval Shifting acute addiction now does first new are safety over use it It years. not on JOURNAVX to this currently pain as acute JOURNAVX is based combines potential. It severe pain so we in approval of signal pain. its with pain favorable inhibitor. types class oral represents of all across relief XX in moderate a is The And have indicated critical the

example, bones, seek million and pain in the care For therapy broken new injuries has the prescription post the to for potential to who moderate a of severe year a standard treat to patients U.S. surgery, each XX acute establish sports

of significant those concerns prescribed XX and each for potential. although acute and are or have Americans out opioids, year seeking Half pain addictive their tolerability million safety which effective, approximately

estimated will multibillion-dollar opioid U.S. pain year acute transform to another have the within franchise develop we year the for Vertex. disorder for the We people In believe each an XX,XXX opioid treated how use to of and first tragically acute fact, being is opportunity an now pain. build in prescribed

begun have of We JOURNAVX. were launch-ready for the and PDUFA commercialization date now

that access commercial is formulary and for deliver with establish to franchise. payers will pain to care patient makers rapid decision conditions our Our professionals, XXXX for success for health focus the engage long-term

broad we coverage a societal treatment JOURNAVX the one need patients and days the approval since into few a new importance of launch, is measure providing of positive for the both of incredibly with has that non-opioid unmet an the that media To received high the just believe while acute and indication pain. end, still physicians of option

for already to look forward from and being both tremendous we We've and interest them. seen to doctors information patients, and requests serve able

with Our moderate of acute safety in on types XXX-person physicians severe the JOURNAVX and sales its force and compelling is data providers care of pain. all health to role actively engaging efficacy and

with systems. institutional the engaging XXX related are setting, and we X,XXX hospitals In high-volume approximately roughly health

to sight in processes use of We support have setting. this of P&T to committee the typical many the line accelerate networks JOURNAVX in

national to our on are to group with accelerate access and our and regional discussions inappropriate We formula advancing payers JOURNAVX, limit reviews to purchasing controls. building and utilization management organizations work provide preapproval

with patients retail pharmacies, agreements country. are across to for lastly, availability stocking secure and at broad of we pharmacies working retail JOURNAVX regional national chains to And ensure the

to have our severe to WebMD acute broad now like Medscape physicians websites point-of-care relevant signal to patients with for for pain awareness begun promotional along in such embedded content non-opioid consumers, physicians as marketing. We nonpersonal for also first pain, initiatives moderate the inhibitor and oral promote and of

momentum access center both or equal to and interest Medicare the non-opioids a surgery we non-opioid to the add-on in the see used launched providing for continue state level payment on pain policymakers X Furthermore, January and on in ambulatory settings. and NOPAIN relief. outpatient The federal by hospital options for Act provides

be cosponsors alternatives expect for parties payment. last growing Congress term XX reintroduced the to had with new to this similar act added and to We of to is be from both expected JOURNAVX The in list add-on year medicines the support. and near pain approved

initiatives Additional progress. also to state federal and continue

support for to legislation the XXXX. XX weeks states in and states legislation introduced adding example, enacted plans to X use state-regulated X Medicaid already into just options. the For non-opioid of have XXXX, that

movement. We states this expect to more join

may per and access price. pain. or have our including the recognizes priced at patients JOURNAVX the On list pill between per also benefits a the to with in we front, XX-plus broad believe strikes it provide. $XX.XX It We day investment year pricing offsets cost at balance this price that $XX ensuring JOURNAVX brings society,

for in we feel to be can this invest with to to pricing come. we continue And significantly, the acute decades us leader and serving enables that chronic patients pain that so

in of to initiatives, is given investing patients later. with the access months by programs we early As the lastly, for And prescribed a to condition. end of expect previously strategically start month which assistance genomics acute are the enable that discussed, critical smooth, shipping launch, the including rapid wholesalers financial patients availability for in days we retail of few nature the JOURNAVX this eligible

XXXX conclude, on U.S. and ahead the ALYFTREK with acute era are a new we and very executing To exciting the and CF are CASGEVY, commercial launch XXXX. ongoing opportunities JOURNAVX launches the of in launch multiple had of we a to strong global in us execution moderate throughout of pain already the in growth in severe and diversification commercial of in

I'll to financials. now the turn to over Charlie review the call

Charles Wagner

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

Fourth XX% the quarter demand billion, in $X.XX the U.S. of January and newer strong XXXX in as recently XXXX many as where growth agreements. patient total increase in U.S. from product revenue long-term a U.S. growth resulted established including increased Ex favorable to CF net XX% exceptional the XX% as have in reimbursement of markets we U.S. effect well well and strong as revenue year-over-year to CF growth continued The markets quarter outside by revenue to dynamic. performance revenue was transitioned gross driven price the the

XXXX, from consecutive tenth billion the versus benefited in our Revenue XX% double-digit of Full of also items. growth. represents nonrecurring $XX.XX growth certain quarter year revenue year

sickle included of the to Fourth thalassemia. and for IPR&D commercial QX beta and AMKD pipeline well X million billion develop IgAN, diabetes, acquired launches. for vivo quarter of expanding $X.X as to XXXX in next-generation therapeutics Orna R&D, build-out disease investment quarter collaboration in type expenses, of for versus as billion are the fourth an included and and Also acquired results for SG&A of pivotal in capabilities including increase the DPN our cell of both IPR&D non-GAAP XXXX the ongoing $XX prior combined XX% increased studies XXXX. $X most compared expenses significant The areas year were

This year compares XXXX non-GAAP compared and of to SG&A were in quarter $XX XXXX. the expenses Included of $X.XX combined such charges in Full IPR&D XXXX. IPR&D acquired million to in fourth for $X.XX the billion Acquired R&D, IPR&D resulting acquired results of from Immune are Alpine vast were charges full charges XXXX year $X.XX million year the XXXX. majority $XXX acquisition. full with the billion Sciences billion

The costs driven in the were investment year-over-year JOURNAVX including pivotal R&D our was studies. SG&A X driven to of increase by year-over-year organization increase CASGEVY. by expenses and primarily in support in continued The pipeline, ongoing investments in commercial our launches

quarter million non-GAAP to was billion of XXXX billion year XXXX non-GAAP in Fourth operating income $X.X charge fourth income for non-GAAP to Recall in XXXX. non-GAAP in the XXXX $XXX year Full a of acquired operating IPR&D from billion billion that XXXX. operating income compared income $X.XX reflects $X.XX quarter compared $X.X full the was operating acquisition Alpine.

R&D expectations, in while Fourth rate benefited credits. 'XX with our tax line was higher tax of XXXX U.S. XX.X% effective our XX.X% non-GAAP in QX rate tax from quarter

year XXXX nondeductibility tax effective full Our charge. XX% the the Alpine of IPR&D reflects rate acquired of

XXXX XX.X%. Our non-GAAP rate in tax effective was

were XXXX to as non-GAAP a share non-GAAP of share $X.XX per interest the previously fourth compared XXXX, XXXX to in XXXX. quarter of lower noted. $XX.XX year $X.XX in income Full per tax result earnings Fourth compared earnings rate differential largely $X.XX quarter were and the

the equates reference, charge share Alpine a to $XX AIPR&D For basis. on per roughly

cash in billion investments. $XX.X with quarter the and ended We

development. unchanged business innovation, cash in by priorities innovation investment remain Our including to external prioritize as continue deployment we for

shares to our course also repurchase over program deployed $X.X share quarter. the shares XXX,XXX continued the X.X million and repurchase in including billion of XXXX, approximately We fourth

Now switching to guidance.

the growth billion, in $XX with $XX.XX total to For at of range growth. driver XXXX, U.S. exchange expect rates current of main we revenue a the as at approximately of the X% billion representing revenue midpoint total

in rights XXXX including will our XXXX. growth in the of outside This QX intellectual that where country of reflects reduction from ALYFTREK. impact one not expectation will as growth markets are ex that repeat channel in will of certain in be international violated. property be of CF launch pronounced revenue in occurred by continued CF medicines, for being our Note the U.S. most core portfolio a our increases impacted revenue inventory QX outlook This QX U.S.

a approved Guidance revenue also includes patients we in more as geographies. treat ramp-up CASGEVY continued in

recent in of year. very contribution JOURNAVX, from approval the Given the of half this the launch primarily in also guidance a back reflects U.S., the

up ahead assistance ramp provide of eligible of to immediate volumes sustainable JOURNAVX to a access revenue result that patients as broad while programs payer designed financial are expect coverage. work We to with secure we

margin from in long-term scale product will rate royalty new offset on goods on cost the Also that To we total progress give you higher be course the launches other context ALYFTREK prescription on as and of largely the on provide lower data lives expected of of the our XXXX covered by metrics over JOURNAVX, to will launch XXXX. they note towards the their benefit margins. gross

combined IPR&D IPR&D guidance for of $X.X and to charges. billion expenses project $X R&D, full anticipated the approximately year billion currently we a in includes $XXX million This SG&A, non-GAAP acquired For range XXXX.

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

cost the future in studies commercial of in will IIs trend The III a diversified driver X for studies 'XX, as over continue were commercial therapies the QX Phase opportunities X multiple ongoing of course increased investment for as multibillion continuing Phase and increase for well. this a our and and spending The supports on zimislecel, to full increasingly support renal potential commercial in JOURNAVX of of capabilities the dollar targeted as in Phase well portfolio XXXX and with launch III example. investments costs reflects XXXX year

investments and make while business targeted can on differentiated our model and these cash markets, Given attractive flow. focus we specialty profitability maintaining

effective the Our rate is range be expected tax to full of non-GAAP XX.X% to XX.X%. year XXXX in

growth, our commercial the Vertex regulatory important make revenue excellent again closing, portfolio. significant us for advancing CASGEVY delivered yet strong XXXX, In while progress launch that position gaining to across and approvals achieving pipeline results all in launches, continuing

with milestones studies continued global driving of are other to disease in the reach in multiple new enrollment X products enrolling significant slide We and milestones X and on the launching detailed U.S., year. These III progress CASGEVY Phase anticipated are this of XX. launch expected now them areas X

forward progress calls, you our on and ask to Q&A updating Susie future look the on We to period. I'll begin now

Operator

will from our And Goldman Instructions] with [Operator Richter come first Salveen question Sachs.

Salveen Richter

question program JOURNAVX, going we prelaunch the fibrosis, to here. these cystic further elaborate aspects? you and really how here. the miss processes on how going And P&T welcome and my success maybe can can Stuart, you just Maybe of JOURNAVX, back to accelerate work actually, then frame On retail think you and for your in pharmacies are you and committee efforts Duncan on mRNA to in us review on secondly,

Reshma Kewalramani

let to turn first, question. and it over mRNA the come Stuart? Salveen, back Stuart I'll Yes. to me then

Stuart Arbuckle

thanks it's wait. patient turns because retail very obviously, the Yes, they to of work across up for retail, been to with kind with get our have broad words. the terms their pain prescription JOURNAVX the there important, to we've with that ensure the chains largest because we when be working chains also U.S. Salveen, national so in Yes. availability obviously a product acute And can't broad availability in of and we regional want

U.S. shortly with are product the that availability retail in we wholesalers prepared broad end the I this in have And entire as to expecting I month the we expecting across And and said remarks, of by will my have thereafter. am

their and institutions pre-approval. with approval work their on our also on of terms payers both In processes

such working profile on Obviously, the clinical that compliantly now. with medicine. the them to of them as full are We need, they information provide

risk and move months. people which is important few our we'll we the given over weeks to to We also well. are to that access on for quickly have JOURNAVX with benefit price, them an as And indications updated progress provide do keep next the the you profile, have that component established looking and

Reshma Kewalramani

And Salveen, mRNA. question on regard with your to

So these last or they X,XXX are the no patients, available them. options have to so

in movement about And VX-XXX ALYFTREK is improvement XX% the one X% inhaled side a approved ppFEVX. so about And hand, other TRIKAFTA improvement on about an sweat we're the on therapeutic, medicines if and expecting because the think I on ppFEVX ORKAMBI mRNA, not the and any on a then with chloride. with

readout ppFEVX. a on be would So this

XX% it's acceptable important. could based than And at X% from what between be well. X% would you just XX% that the be look that, any number of I would if to more range medicines, as on be course, fine. approved a would But and as think And

ORKAMBI let's Remember exacerbations. translate long-term you XX-plus that what outcomes. a reduction improvement that's ppFEVX X%, the If to pulmonary in had in X% in say, acute into improvement or the percent translated

Operator

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

Evan Seigerman

your on a Also an you strong to It's to Congrats great retirement. this Stuart, watch company. honor congrats and 'XX XXXX. transform really a on been start

JOURNAVX. to on it a bit talk progressing? touch discussions have you So I want payers a the when to lives change retail me how therapy year? your to expect going that to little been is Can you many have, early course the is channel? access how about over with do to covered And How of

Stuart Arbuckle

launch on the and Again, thanks just me for as everybody, let well. July XXX% to and CASGEVY here driving JOURNAVX kind reassure going I'm I the And words. focused ALYFTREK X and be until it. appreciate

approval, In were prior discussions, payer the gone terms continued post obviously, They've to really payer we to And Evan. of approval. have well. them we've having our discussions

about first think no and the Everybody for I it's surprise. excited options everybody need non-opioid aware is of treatment unmet acutely this is that better the and opioids is the have fact decades. to

But I expecting payers expecting specific grow through point formulary. put get to time. how obviously, over that on time, expect it we choose or are expectations what many covered lives year. it JOURNAVX as We're the expect to broad grow throughout exactly will over I not on their and more am guidance at we we the to giving year access

Operator

Your will next come Jessica with Fye from question JPMorgan.

Jessica Fye

should CF. driving you guidance, by in how seems versus And about rates talk XXXX was wondering could of about CF contribution respective growth U.S. growth like it the about that's the U.S. U.S. line I driven kind those if the we largely outside rate top think that U.S. business? in ex to Thinking the what's the and

Susie Lisa

Charlie?

Charles Wagner

Jess, Yes, the for thanks question.

prepared billion, $XX in to at call of U.S. between in of highlighted prepared U.S. remarks, We midpoint. Our billion the we the differential and out the the remarks X% represents as guidance range the ex sort $XX.XX did

obviously, world. around growth the CF you see what's continue I on, the about strong in we think If very business think XXXX, to about going

ramping ALYFTREK as in continue ongoing that to launch the and expect of the CF. addition benefit to the We JOURNAVX well growth The U.S. XXXX. CASGEVY the into has as in launch

think strong If we'll in you see about growth international, CF.

the is call well. launch we out our would markets, so obviously, country most IP ramp one will rate pronounced out thought did to call we as impact is where there continues we has ex the for an In growth and quarter, most U.S. that respected. be That U.S. first not then And you. visible being that the on CASGEVY in ex that

the there little healthy So us. between growth with bit U.S. in the difference But is those line on overall, rate a a ex XXXX. very and top for U.S. of a dynamics,

Jessica Fye

I can just follow-up. throw And on a Great.

I also mentioned nonrecurring at that revenue to the benefited think some items. you quantify from XQ Possible all?

Charles Wagner

by that get in I'll quarter the strong. as qualitatively, was benefit not increase year. you, into price I'm that. was fourth going I'll the took very CF in the volume Yes. the well we strong to tell Growth driven just from earlier the in U.S. as

we a U.S. gross very net the addition, to In in dynamic favorable saw

Outside reoccur benefit settlements VAT those quarter. Gross like Typically, growth markets, in CF. onetime we a the the things to a in of other in But fourth the did first from some in worked quarter. We negative some and net we strong honestly quarter. out also meaningful rebates necessarily every don't different have quarter-to-quarter. the items, volume benefit that U.S., see fluctuates, little it's again, did more

Operator

with Geoff The Citibank. Meacham will next question come from

Geoffrey Meacham

offer all the I to just and also Duncan to up today. want announcements Stuart on as congrats well as Charlie

a any feedback. dive know guys and Phase some KOL taken you're JOURNAVX and on there for have gotten thinking III I maybe Phase Are into design? LSR. II just considerations more about deep you you've So the

still regulatory or qualitative patients you second care perhaps unmet you're populations. quantitative need TRIKAFTA is not just feedback coming anecdotes want And question early either ALYFTREK. on awaiting new know on I maybe feedback, any get then want I on get are to guys on from into but your perspective I to that? or on those that

Reshma Kewalramani

question, take Sure thing. to ask Geoff I'll ALYFTREK. LSR I'll and then on comment Stuart the

that into regulators. is, Where actually waiting are their meeting we're II. have everything we thing of the The it Phase our from we teams with right. is with our So can our glean analyses Phase possibly II the you end for big are deep analyses very of exactly

We are in out involved meetings. talking very to not only studies, key ourselves our we opinion those and are LSR doing preparing leaders, are but PIs regulatory for and who

of present have I'd update. this all our to at collect more don't and And what like we've obviously, it you understand package but with is the the FDA summer, this we you really need seek. an regulatory rather we all to I'll PNP interactions. in have after share are through to what this summer I to the packages, in once the the big looking would see for thing that Stuart? broad indication that include gone are what to we

Stuart Arbuckle

Yes. know, ALYFTREK On you approved, who ALYFTREK, and on modulator, above interest of never been who seen has mutations for naive CFTR particular, Geoff, patients which, in are eligible is we've are have even those as from over kind modulator now either CFTR strong a TRIKAFTA. additional a and

strong seen you might expect. So interest as we've there,

but discontinued. those who patients, on like But we've TRIKAFTA, are previously in who also something either those transition also interest seen

were to their X we patients we've their and ALYFTREK, interest packs prescriptions see. that that of I'm already say already which have to of So is across first the first first expecting. the population great And seen received pleased all received

Operator

Michael Jefferies. Yee Your next question will with come from

Michael Yee

launch. the not thoughts to working reimbursement, about the bad know it where not is about going paying Stuart, hopefully, think and the would something can as side thinking nervous your the coverage pain you in an the for not understanding you're payer which on that good you JOURNAVX, headline that. know with that just Presumably, important of thing. is a as into opioid, Medicaid comment But commercial thing the relates Medicare, coverage administration making public probably that's acute that, around as I you're well

should And something issue so on given things, you X the launch not of that are an reimbursement JOURNAVX. fairly that confident is be those

Stuart Arbuckle

is be let this Mike, company. It firing first all have your been more Vertex. I've first at Well, and to me never excited of I about both comment. cylinders, been address Yes. commercially. confident on scientifically future more the never

you have or And doubts to other about any things right any now. have launch either ongoing so in launch confidence that the I the we of of want JOURNAVX my wouldn't

to decision life. the that the on is next chapter move a time purely is It of right personal to my for me

for broad segments. get I time over In terms of across coverage payer coverage, JOURNAVX am us expecting to all

has you. pain opioids. perfectly events, to with for been For non-opioid addictive be adverse the potential effective that reasons described, reliever aware that a Everybody including Mike, of not enormous unmet is did the Everybody waiting need. the honest you of truly have

And expecting so broad over us access get I'm time. to

policy in of discussions been of about In a sort addition to think to having, can flight individual moves, JOURNAVX. already the both talked the of which many I to we've availability way, number are this I we've put if it federal state times and create,

feedback So huge obviously has but I'm launch, going to We're a the be into been success. I'm only days, excited. JOURNAVX X absolutely days the incredibly is business sure X tremendous and

Operator

William Pickering will with question Bernstein. from next come Your

William Pickering

placebo all about that your placebo designing on had you And X to for points assume I the retirement. effect in reasonable a compare X.X does effect how and we III on Stuart, the LSR? question the meaningful that be in progress, Phase what points Congrats Is And effect saw suzetrigine? did congrats a clinically ballpark? what would versus DPN study? placebo placebo delta

Reshma Kewalramani

is I first, set full has the appropriately Phase many has our taken a that to we Phase have gabapentin group. group, but are trial Phase tell been I not approved. that procedure. the the III, studies share and Phase will I you while the Phase primary many On and from Yes. you JOURNAVX you, III DPN area, And advantage that will DPN have week the III research, are Will, were note But with surgical note III will your going read I studies addition and appreciate specifics you placebo earlier through your medicines to trial, Will. that there did doing data II in there that size what also in own group in several rich a only and of

And think outcomes as there. we've that the all account about taken we into

advantage So tells study literature what suggested the of And care while takes I have are full sample you please all the you. general placebo effect, available ranges including you level of you won't well have that share yes, the size, there. note out indeed the we the that of that that a specifics, with designed, that's we've taken bet.

Operator

question with Next Liisa Bayko come will Evercore ISI. from

Liisa Bayko

involved through just offer get and It's transition fun. my line be in and great you. vicariously actually. everybody to -- living We'll to both have I'll a congratulations

to I So ask wanted about program. the pain

Just to available couple seems channels. in different stocking? the of like sort stocks of like any a maybe you trying describe get It are questions. you Can

So some just there. commentary wanted

of pain is big how right are longer. about We where setting action ambulatory earlier a the kind care onset was of also, and is you call patient? did who line thinking of falls And the if in a like action bit and the a it this week little topic in onset

And line? right in you terms So of where how for the suzetrigine. second it are first, positioned is thinking or patient is -- of third what

Reshma Kewalramani

JOURNAVX line our Liisa, pain first for moderate Stuart? going prescribed upfront, be to to I give the here I'm to want say is pain. but you to some acute over to medicine it color, for severe just goal to turn Stuart

Stuart Arbuckle

Yes.

the to So your retail product Liisa, across country in pharmacies stocking. hospital we are Yes, addition in as on to question looking just to broadly first address well. in the get stocked

striving this would are thereafter. that end do we feel shortly the happen very to and of as confident will that, said, product wholesalers in by I I that, be yes, month So then the channel our and with the

it feels to product for broad those using spectrum a label populations. said, the of prescribe physicians procedures opioids I that's most do often prescribing afterwards. in think about they some they're the treatment a of think are about from acute how be talking the many, they week many setting thing weeks heard is there. different or across different ones procedure in concerned might we like somebody got going And pain. moderate situations need Reshma they a to severe after people And the JOURNAVX As early of to be in a the great where discharge about where for we've X

relatively So But Reshma be on. seeing that how I early goal is it pain are acute be to think that certainly people I severe about patients regardless moderate our JOURNAVX an etiology. for will is area will where as of treatment treated think and said, the there

And so it's across I the spectrum. going to used broadly expect be

Reshma Kewalramani

have one just Charlie comment your we from Liisa, more on question.

Charles Wagner

The stocking while to What reason increasing year, that I of assistance we'll why in volumes for absolutely half would programs comment, of our ahead commented we Stuart's of revenues. the the see work patient to the first secure broad seek add is you'll which that access. impact we is

the second And half the the more year. loaded revenue so while there of half of will be in will be stocking contribution first year, in really the to the channel the

Operator

Next question will Cowen. come TD from Phil Nadeau with

Philip Nadeau

on your transitions. Stuart, announcements and Charlie today's Duncan, [indiscernible] and congratulations

On on you script. accurate, are expectations prescription of our ALYFTREK. questions, us JOURNAVX on Vertex's some sense that be third-party from give the will data couple In terms for can duration and of on sources what of the average a and one we a JOURNAVX, see

the ALYFTREK, in Royalty there as hear to you are could on And owed. dispute. of and is a exactly to we resolved? Pharma milestones what something? Can give between dispute sense us that some is When on Now that procedures getting Royalty going you what the

Reshma Kewalramani

is this matter then second about I'll really and So it opinion. turn to let JOURNAVX. a first, talk me your ALYFTREK, to over question take Stuart of On not

the are. We what responsibilities contract spells have out our contractual Foundation very and CF clearly with a agreement the

I questions that. And any don't interpretation JOURNAVX, few opinion room on a Stuart, or on on for see so that.

Stuart Arbuckle

be On JOURNAVX. is is a days time It severe that does considered first I than thing although pain. for pain so the say days. of is limitation of or limitation therapy, the would moderate lens acute less for label acute Phil, to a pain the XX not within therapy contained label, to have Yes. lasting

of for -- or in length obviously, non-steroidals, around setting inpatient the very the see of In discharge the about days. terms setting, in it's you XX of therapy length at there outpatient therapy in short --

going the leads we to have it it's of sort which because less as I the somewhere possibly little For the would is JOURNAVX the more people X- can. what have, that reasons all to the baggage out to opioids doesn't that days nonsteroidals in to expect opioids, they markedly is like as probably X-day give expect range for of want therapy be

Reshma Kewalramani

a about Stuart, And data in quick maybe inpatient. split be there's retail in happens and accurate, the there them will the question what into prescription out the

Stuart Arbuckle

Yes.

a So buy, Phil. little data on depends you prescription what bit it

setting. comment in it is very on that's in bit to volume-related setting It's will hard what the overall how prescription So it's available. data be. more exactly robust a But retail hospital data little the good

the be of XXXX. well prepared as a X number the of as providing success Charlie markers said our providing, of prescriptions information We'll early on updates his calls will covered -- lives in as of on of be our for in markers remarks, ongoing most also as important launch We the one success.

Operator

Eli come The next with from question Merle UBS. will

Eliana Merle

rationale basically, step give this segments. recapture previously who been guess, modulators, your with I You on had ALYFTREK? color are their a you been had you've X,XXX that mentioned on any back no new ALYFTREK, seen And segment back, were CFTR starts can what to all longer the from starts different patients from patients confidence patient taking other the modulator Can coming coming you the for but that a CFTR what but globally. and of drives discontinuing

Stuart Arbuckle

Yes.

have has that good and is first ALYFTREK patients well. terrific a it's the mean, getting shot confidence a those I itself. back day we Obviously, our the profile as drives it efficacy So at once clinical of that thing

on CFTR all is not CFTR that to reasons. we range on be whole unfortunately best stay they of CFTR these want patients for really the patients to fact a modulator. certainly know They on can. CF that a a if it have the modulator And and the profile to able So be been modulator want

any were the they they those gives that's to came on those back us a thinking and in back It's on patients ALYFTREK. to exactly you things, be I So at give people are what stage. discontinued small initiated relatively the of the date. granularity have lot come going the that confidence can't of why a patients when granularity and grand on know individual or what to this ALYFTREK to scheme I even of patients don't number

Operator

question of with Ahmad will from Bank Tazeen come America. Next

Tazeen Ahmad

CF. One on

pipeline, So we be if ALYFTREK? year, of for are TRIKAFTA to how just thinking And of from your about then how rate a sense opportunity of from you And to get to expecting about so, thinking you're type data that should be the cannibalization I to this that the set show diabetes expectations X there. for clarify, and just wanted should we switching? see

Reshma Kewalramani

All right.

Let me to Stuart. turn it over

Stuart Arbuckle

Yes.

and TRIKAFTA yes, older benefit So day. of one transition ALYFTREK to may and being modulators we our even to of are on the given has it patients benefit/risk it patients profile ALYFTREK, be the has once expecting CFTR on from who a

We switching on set be. we think rate guidance given will of or expectations haven't any what the

Reshma Kewalramani

the That's II Phase talked be has that that being we On insulin let's have the naked VX-XXX is is question, portion maybe patients trial follow-up in year the cell free. the the enrollment complete TXD to the way position to this, summarize about and we'll going this the of a when of of it is that's to it file best program in the program, call year, group X program, development. now lead

are maybe are something a and with maybe because We their of transplanted immunosuppressants. maybe have who or in XX,XXX, are of X so been very diabetes like who reason kidney on brittle highs And that estimating or already their is that patients and sugars terms something that XX,XXX therefore low, already transplant high, type say diabetics, we lows and it's that opportunity, the people like XX,XXX so. people

with diabetic then to aim patients. program, hyporemute and encapsulated to edits, device there. the And gene all required that's of we course, which in immunosuppression also that working on immunosuppressions. program, certain alternative that have And I/II, X of research cell the with to same the we're Phase we next make the And same aim type no programs is VX-XXX get cells in the with

to all those latter get our to of them patients. be goal programs, All would

Operator

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

David Risinger

my Duncan questions. as have congrats So I and Charlie to well. And add Stuart, X wanted to I

can that plans I'm that So first, secure with access, respect in the broad discuss you bit to formulary little hoping a detail. more to

other drug physicians. challenges. So are but It the just Surgeons all. to and be on won't don't edits there time prescribed is if a hassles for go have step formulary, through

able access So to to the unfettered to to get be do necessary pay you that expect are rebates suzetrigine? broad

the second, the to company's with violating IP, And countries other that doing single is as from Vertex's then what take country to same respect to country? the action prevent that are plans

Reshma Kewalramani

Stuart go David, and first comment. to let then Charlie me ask will

Stuart Arbuckle

Yes, Dave, okay.

if prescribers to access you So medicine, management lots have not really you're way putting broad getting is utilization you're broad of agree significant in right, use the access of controls with your getting actually if you. XXX% hurdles

preapproval own And on been the with its management so with agree is absolutely them but just because of access, is access discussions government I broad and since access with the certainly goal broad our but insufficient. controls, not nature channels, commercial we've that it's both in minimal been and utilization and the broad having that's approval your

are commercial government to part key in talking a indeed what the I we is that and say, the area. payers, so And of both as

the some policy initiative well. you introduced what of And in been as it's that's actually see

alternatives to the for actually week. So Congress reintroduced instance, which just last was to pain act,

things for the things. branded to no non-opioid that the management with utilization that X of point Part Medicare are generic includes equal patients, a One, is co-pays no there also D One making. it controls and opioids you for it's

Reshma Kewalramani

a word. Charlie, quick

Charles Wagner

incredibly question CF, that know portfolio enforcing decision late very a successful an country and in made they IP in goes been market. IP defending Russia, robust it is disregard IP XXXXs. have copy. our we Dave, around an to every strong in and We've one you and and the allow have to out unauthorized where The Yes, the

our But that enforce every is a for that let's a sort I that unique are to issue measure market rights. this We reason. taking isolated. is very possible feel say, of,

Operator

Brayer from Olivia Next Fitzgerald. with question will Cantor come

Olivia Brayer

getting feedback quantity parameters do whether you place conversations? have so P&T Do yet formulary coverage? way, JOURNAVX? an another other just in management And just you're be Or around consider any for yet be asked long you sense actually before what from recommend would limits far know you process? there utilization maybe to any or how expedited payer will officially a Maybe put will take it committees those

Stuart Arbuckle

little certainly and to a and second to the of not wanting to commercial I certainly step getting kind our management ago. I'll physicians prescribe considering medicine controls Olivia, with as medically new of a I get edits think have can opioids, said minimizing ethically and very things on payers, access I utilization inappropriate. on Dave, in discussions patients the said, the in utilization like bit side, of government Yes, which answer discussions We management would are repeat fact controls my just the a aware way what of through involved and certainly generic and those about be that

discussion we've been a is to have. we So continuing having prior launch, to that that and are

the we as the run-up of formulary in things doing of approval it them how prior to a compliantly the to quickly about providing we with that think the we one understood to sure were approval JOURNAVX terms committees, was to way were institutions processes, making In institutional could the compliant P&T approved. particularly in again, they allow review that we those in to the when the wanted setting, everything

obviously reengaged some believe cases. accelerate their post XX than that are will and accelerate everywhere faster do products, certainly significantly XX months to can. a of We've approval. we typically up take with some to to looking processes institutions we can in those new They I number that for they them do.

Susie Lisa

please? you up That'll Seth, wrap do this if could it

Operator

available XXX-XXXX replay shortly event (XXX) today's for of ma'am. access attending or be well replay our dialing today. XXX-XXXX participation code Thank will by for conclude today's X This you the as presentation. your Thank (XXX) call you question-and-answer will session XXXXXXXX. using concludes conference as call A after Yes, today. X our for

You may now disconnect.

Vertex Pharmaceuticals (VRTX) 10 Feb 252024 Q4 Earnings call transcript