Vertex Pharmaceuticals (VRTX) 6 May 242024 Q1 Earnings call transcript

Good day, and welcome to the Vertex Pharmaceuticals First Quarter 2024 Earnings Conference Call. [Operator Instructions] I would now like to turn the conference over to Ms. Susie Lisa. Please go ahead.

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

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

foreign management exchange foreign presented addition, impact to the call In of Reshma. program. I turn over inclusive exchange of the is now our risk will

versus of billion period. continuing growth representing quarter Thanks, patients, delivering Susie. another CF momentum strong our in from continued in the today, performance us Good prior 'XX to revenue QX, XXXX, board. call with Vertex evening all, kicked more the and joining thank across you $X.X year XX% on reach we've off the for excellent

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less for normal levels the diagnosis years cystic of half to of sweat chloride underscore than as XX to of and the to this study and and millimoles chloride of Included eligible Results towards patients achieving superior the with the program results diagnostic age below or vanza vanzacaftor in patients of patients carrier to met program millimoles patients our RIDGELINE randomized pivotal chloride the impact in milestone levels We TRIKAFTA this advance To vanzacaftor XX% CF. early We level sweat fibrosis, could chloride. and vanza normal the burden. III liter from round XX continue to to in level per liter. carrier as goal carrier demonstrated ultimate levels the X patients new sweat XX that the the an sweat announced substantially of pivotal lower bringing offers considered vanza was below important triple, Vanda out our of it's range consider the aspiration. than pleased levels age very of we royalty the sweat XX the study the of Phase these by of to all X non-inferior potential also or are XX studies in more on of our in a to threshold and and the Results February of in sweat the important indicate of high on to below was progress convenience proportion treatment the X TRIKAFTA profile we expectations, levels results believe we're therapy triple standard towards once-daily note the that chloride. lung measured including, of set sweat from per of chloride reached dosing a level function chloride above be achieved in in

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finally, The quality the if superiority. order will for study. XX-week and And pain first key into to of key evaluate the secondary of XX-week secondary will the a test life have completing score, open-label to pregabalin is of suzetrigine a same placebo. In of on opportunity successful, will the second we safety roll subset to NPRS the test non-inferiority and versus for patients suzetrigine, measures extension study long-term effectiveness endpoint

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pain, both of suffering unmet need forward transform Americans of as and and these potential tens we have Just to acute address neuropathic millions conditions. treatment CF, look the the the of of with helping we to the the transform of believe treatment we

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half as in about benefit, drug-like models the preclinical against including clinically and B broadest affinity in the plan second RUBYXX in renal of overview. its that, basket turn with profile, as best-in-class IP robust clinical proteinuria excited potential with cell patient well diseases, preclinically X-week a year potency and study both every across portfolio. Important in initiation I'll through potentially dosing, the RUBY-X remission, patient the and that cell-driven IgAN once direct APRIL including of in BAFF properties of efficacy animal dimensions, hematuria, Phase for injection are subcutaneously a good assays pathways better in with the respectively. With ongoing Phase diseases tolerability include look and and readouts to IgAN, Stuart II data safety a this pove We III multiple GFR low upcoming over volume, milestones and the it with and povetacicept of field development and from high in studies and autoimmune high cytopenias, commercial in in and

of And a diversification Reshma. I'll outlook discuss some era provides the highlights as commercial suzetrigine pain. for launch entering of acute Thanks, and ongoing then in CASGEVI first CF. the we're new

we eligible delivered Reshma modulators. we our to results again continue As patients the once noted, as CFTR receiving grow CF strong in number of

driven First was we approval growth strong in quarter U.S., also in year. this ages year-over-year April TRIKAFTA, quarter, continued of the rollout years in of X, Outside in access to course countries X driven including in X year. patients following growth of performance and of population U.S. the last this saw in EU uptake by the drive X patient by patients in EU more KAFTRIO old this to the the November we age approval and the to in following will group ages continue XXXX, over

medium term. short, and CF outlook Our the in in long is bright

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access signal acute peripheral cells We option treatment neuropathic having for potential suzetrigine. NaVX.X professionals we to expand the provide continue the the In with care increase believe this patients number the work suffering pain. to from and millions inhibitor of in to pain ATCs East, now and collected ATCs transformative local Middle patient of health to has from activated addition and patients to region. Shifting our the in a selective highly first

opportunity, going and my of its effective to and both surgical with trials quarter, combination safety potential of acute range comments suzetrigine I'm unmet address across of both Throughout relief a pain and be severe used ideally will moderate nonsurgical limit commercial date, has need in clinical This the we efficacy clear to X/X acute this each moderate-to-severe a the for webcast, strong concentration of safety setting. pain. profile approximately patients with a a physicians, favorable to to conditions, that profile. compelling On is within and opportunity commercial in to among including infrastructure. the are patients acute magnitude, across This XXX range patients a concentration pain be settings. the high shown details that roll on pain IDNs. approximately with with a X,XXX being and treated There approximately million institutional investor around medicine the up U.S. in prescribed institutions prior tolerability to can setting Accordingly, they further provided XX specialty year the and served in

have in We X discharge in XX% XX% for institutional treatment, the prescribed offices. days setting. XX% an are and also prescribed physicians' their dispensed billion of detailed settings of over prescribed pain are are mix and calendar acute

with This some on you landscape. update quarter, insights payer legislative I'll and on provide our and the go-to-market strategy an

or key will physicians fit, to procedures, anesthesiologists Extensive can and physician set are pain include institutional and surgical we X,XXX helped focused professionals The likely of pain pain are conditions seek pay acute has general champion discharge adopt also specialties department with institutions high-volume and the to emergency high XX% the conditions significantly plastic pain suzetrigine. orthopedic, that acute medicine be or prescriptions. an such reduce account care for approximately identify setting, typically utilization. market require medicines initial And targeting us at we on prescription types as specialists. clinical surgeons, health procedure opioid replace of and specific where that We related these research given

dynamics the commonly to year. in of we in discharge opioids calendar effect hospital X profile, the discharge. Treatment billion to state and for is where days occur prescription in XX% in approximately the prescription due pain Given for includes of U.S. this and at limits is roughly segment length days the the use will side medicines this suzetrigine Recall, new length earliest each X setting level. shorter concerns this X.X expect to addiction treatment be approved X The acute of institutions, represents and uptake setting prescribing average approximately IDN weeks. at

key PBMs, including We pharmacists, with are GPOs. payers, across and engaging IDNs landscape, already decision-makers formulary and the access

and to great launch half the of throughout these in of We our stakeholders first this engage support also the coverage expect the make ahead second plan formulary progress of the year, launch We've adoption. to decisions discussions accelerated contracting commercial build-out for year and potential in the team. to formulary thus made in of

now engaged fully the new Our and III are force in team finalizing board customer-facing field leadership and data hiring after hiring of the the the colleagues. field trained we now have on until of Phase XXX are

Pain significance important the policy like to alternatives track XXXX and Pain on treatment world know like we bills Act in the already pain legislation of Finally, with Act No the in recently of introduced. the implementation for

encourage consideration remove decision Our choosing population long-standing to: digitally policy help makers. focus barriers approach Overall, two, plan on for one, branded efforts science, to strong initiatives and shape non-opioid alternatives; of and high a commercialization with non-opioid. state use health enabled continue financial a and we federal a to

supplement commercial time an exciting our and advocacy policy conclusion, complement Vertex. and patient efforts to it's both activities. In public addition, In at be

CF to combination. in more well vanzacafta treat planning for of and around are triple world continue patients advanced the launch the We the

over Europe We acute Middle Charlie the well underway financials. a suzetrigine preparations to And CASGEVY era of of as review U.S., are launch in the pain pain growth. I'll the new of we launch the now to redefine and treatment turn the further entering our with in diversification to diversified the and seek drive revenue call East. fundamentally commercial for are

start performance the profile. again our growth demonstrates excellent Stuart. Vertex's year once strong consistent attractive to Thanks, and

channel start solid were to some the XX% increased expectations line and X% phasing inventory to outside markets. XX% our XXXX outperformance quarter this strong select international of the in First U.S. with $X.X year-over-year in with due growth in with billion U.S. of The revenue drivers

vanzacaftor to continued last following quarters, was the clinical as of growth April non-GAAP offset TRIKAFTA, suzetrigine certain partially costs QX of ages year increase non-GAAP subsequent similar billion and the QX XXXX costs including in strong to $X X investment XXXX XXXX. for flat upcoming first pattern benefit the channel IP SG&A of dynamics quarter. in in into of $X.X expenses activities inaxaplin growth billion launch over portfolio compared XXXX. this and COGS organization phasing $XX in reverse inventory. We in from performance recent for AKD R&D to associated anticipate expected the such initiate first seasonally our the inventory painful by to the million saw CASGEVY launches, in Non-GAAP the increase broad quarter advancement multiple in patient to quarter of by higher and non-GAAP transition gross First of in Outside from commercial X to 'XX reduced including SG&A versus trials million U.S. preparation commercial in patients earlier-stage and combined expenses suzetrigine net and acute in neuropathy the was our of also growing of CASGEVY, acquired R&D the advance launch driven approval The as acquired by by quarter costs we to 'XX the new and successful to first and Included from in in peripheral We 'XX the well of in R&D includes R&D SG&A population R&D as is we R&D in the build-out further potential were of QX will driven invest IP quarterly typical 'XX diabetic the and Phase year-over-year remainder completion $XXX First charges suzetrigine results, a continue team. of of III were first the the late-stage KAFTRIO in development half relatively the U.S., pain. investment in and offset reflect compared of XXXX. program III charges Phase our R&D for quarter expenses

non-GAAP First in compares was income to quarter first income benefit XX.X% and Vertex's XXXX. operating includes to quarter XX.X% tax XX% from quarter million a in adjustment billion, non-GAAP discrete operating XXXX increase the rate 'XX tax $X.X a income First of of XXXX QX to in a compared $XXX reserves. We non-GAAP effective

First expense lower rate as first the per and were quarter and with in billion share phasing the investments. to quarter ended well tax quarter XXXX $X.XX $X.XX of including compared benefits non-GAAP earnings in as and XXXX. We from $XX.X revenue a cash

We and our priority III-ready for for will transformative Alpine is cash this potential given innovation, business fund in in need. close povetacicept capital Alpine a best-in-class of deployment, hand prime potential area acquisition billion see including We external via unmet the innovation development. use to which invest portion customary IgAN, multibillion-dollar Phase to disease is with high certain to subject of on this expected quarter, conditions. Sciences, a the to Immune of $X.X a example

exploring other diseases. forward We also full to in pove's serious look potential

of XXX,XXX deployed in million shares. we quarter the $XXX Additionally, cash repurchase first over to

exchange to midpoint at range revenue revenue guidance. $XX.XX X% switching total growth XXXX guidance There change Now is rates. current our of at of product billion to to no the billion, $XX.XX representing

high have revenue this outlook. into We visibility

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XX% expense to continue For billion of IP and operating include range This total non-GAAP of projected within commercial approvals to rate Vertex again our of to range IP continues Upon of at charges. of acquisition, note including accounting, in anticipated charges closing, revenue currently potential Alpine's delivered we R&D remainder expect year operating will growth, we close the full absorbed to effective this be excellent results Alpine acquired regulatory in million off non-GAAP the $X.X change acquired closing. also non-GAAP transaction to posted launches. billion guidance expenses, yet $XXX guidance There's but to Vertex we R&D. no the SG&A, start R&D be and XX%. In approximately tax for the potential XXXX to full year combined impacts operating IPR&D time XXXX XXXX strong year as the range determined $X.X project any expenses the

and capabilities We and of our our also mid- disease progressed preparation clinic near-term pipeline in strengthened ADPKD. earlier-stage launches, for area in additional our entered XXth the

Alpine announced strategy. leverage fit Importantly, aim to Post we pove. regulatory with Vertex's commercialization development we acquisition anticipated capabilities clinical, Vertex's of Sciences, and and the to compelling accelerate Immune a close, of commercial also

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further we As begin important this to pipeline Alpine you progress and XXXX, from Susie to mark anticipate I'll disease on future multiple ask Immune transaction we not on that milestones Slide areas. updating the until note our close. We forward slide will progress as look through XX move on programs continued Sciences now calls, in to reflect Please Q&A our detailed post period.

ahead, Susie. Go Instructions] Ms. [Operator

great. That's No.

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

filings. on few had I a the

have chloride to label. the that's So question III is question. a curious from data you the can you'll think I'm the Obviously, sweat get Phase of benefit? claim seems first It like do kind just for on the Do you elevate benefit? perspective for So the a sweat regulatory first chloride the guys Vans. you will what

wondered pain. The to guys or if question drive as you option can second an in make think I claim regulatory lyric, that are to that. for -- the opioids same acute of the for can push a to XXX one, to kind climate you Do -- going

is this Jeff, Reshma. Yes.

those Let take me questions.

marker. labels, CFTR have reflected you back look or pharmacodynamic triple, indeed at are because modulated all sweat is and that vanzacaftor always PD go a labels, and the if you'll in the of chloride the it we see they On

that set the just at triple of filing. the studies label chloride in the Obviously, the vanza So will data are the be we I reflected expect point submitted having fully from

So diabetic was the the of not will your submitted I at submission. I On we because Jeff, negotiations already that have we're the expect broaden filing about prepared the to as point let VX-XXX, the and XXX label as few question initiated this my neuropathy, pain about be But filing history modules, acute if I yet. but of and DPN, remarks, a me label. quarter. complete that the rolling peripheral sweat chloride question serves in already in think the that's guide, study, said absolutely expect, We've we a the

acute end the about will to exactly discussion there, are true to placebo same be have it generated versus but that We insofar that be it. want it diabetic data. there that And regulators submitting the point, when Phase and opioid the a the I peripheral with pain how acute of in the are comes primary neuropathy will we the display all arm expect the as they results data in data as are III pain

from the us prescribers. we that I reason is arm will able to for III be the pre-gabalin say have Phase reason studies, a in We for acute the are not at III. and point the it away far bit for study with studies DPN are to label Phase starting negotiations exactly DPN have But pain a the quite to data that the just share which for

Reshma, just to a quick follow-up that.

to I Medicare kind to of you'll do opioids. Just alternative know label, comes you when think need help the mean, don't that with reimbursement? it yet but to you the obviously, on

Yes.

and ensuring on no I are the there And So that for and the barriers going Stuart? pain data, primary side, that Jeff, we endpoint that are regard that very important minute. the important policy. as reimbursement think to a to comment Stuart in non-opioid, place a prescribing on to with to be see data I'll acute ask most securing

Reshma. Yes. Thanks,

broad seeking we is are patient product decides there. do So niches of to severe looking to pain, to and product acute first type are agents have they acute so used remember, the that physician to the label preposition us other if thing be fit. that want the the for any wants I level possible would say, moderate could broadest use looking pain really Jeff, for not to we're or relative see that's a out the so We to patients and ability the in physicians a

tolerability efficacy strong potential. the moderate-to-severe as which said, in in its support addictive mechanism, safety we really pain have is to it important the see that is we VX-XXX have combination to given very acute data all the clearly primary additional the Reshma fact As doesn't with that and endpoint, talks

to think for. which prescribe going going the range at is to thing and full the product safety, that's decide looking efficacy really most of important be allow we're to want So the they to I physicians who

come from question will JPMorgan. next with Fye Jessica Your

advancing musculoskeletal a various perhaps X.X detail. I'm you NaVX.X extent for molecule curious for consider would engaging pain your it's to the and Vertexian programs, maybe another commercial partner sales not a

kind value so you like to potential thoughts any leave on if table. the about curious Just that, of not have

for question, Jeff. Yes. that Thanks

stage, X fully add, the the because, want NAVX.X that the set combinations, pain, pain, a in serve the it's already the molecule potential. right, I serve here. everything of osteoarthritis and NAV VX-XXX, time see NAVX.X else, pain. as X.X I kind the And And fully our or then with just you everything to X.X, else. go to intend areas expect when know, do patients pain, So time And musculoskeletal in that all X predecessor our distinct musculoskeletal We demonstrated neuropathic of But to assets we pain. kind that pain pain we VX-XXX acute at to patients. say will and is I step would

So we X primary acute or those will that patients. patients, the that's a commercialization step that, Vertexian. the first, there But pain, for NAVX.X see neuropathic need will but and musculoskeletal research of it at that in we value VX-XXX, cell medicine alone see combination whether or musculoskeletal be is to a absolutely do not for we those going development to after And commercializing do the completely time. care because a or the again, them to VX-XXX then we're ourselves pain, help we ones and come pain, next-in-class get see any and or with we either is and NAVX.X

we First, and do and will pain, neuropathic ourselves. acute that

from Richter Salveen Next come will question with Sachs. Goldman

in hospital Two part in the are importance cited makers, to help the engaging treaters versus how here key can legislative decision the taking With regard the pain you tailwinds second outcomes who be here of lower of to reduction whether And work make together or recovery required, to decision? argument room opioids? need to here is usage make in the it reported? they physician And might it what you really of on account administrators is data into using rates being program. make with an us time my addictions opioids the question for and understand or acute or to that specific verticals hospitals a

Sure, comment. ask Salveen, Stuart to me let

Yes.

So institutional medicine are setting. stakeholders Salveen, the you making new all going to be of important use of in the in on the described decisions a that

advocates obviously, their But are It's as are certainly the process as stakeholders, discharge setting, I be of not physician it earliest advocate a that particularly all to setting are bit is pharmacy going standardized going a This the the team, collectively. setting to typically suzetrigine. the administrators put suzetrigine. like the where decision that little my within be likely for of medicine whether on to is the decide efficacy based that various the suggested to physicians, important. who to hospitals the use, the And newly is of institutional it created why standard They're interested be going where be something through a administrators our is go to of the And some be remarks process prepared which making using and that sort going straightforward. So and we see uptake safety there the is going they're through in go more on in medicine. that's typically process. process a it relatively to all going be P&T formulary committee

In you essentially that for referring with terms that sparing patient opioid the other choice to outcomes the is way clearest I of describing when Suzetrigine would every think been have of were of treated that is data the some providing is patient.

kind I data we've and impactful sort be the and efficacy just to is in described. of various So think safety that to already available that from shared. And compelling all already going readily the that got stakeholders of data, data we've other data addition in we've many to is ways, pretty the

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

to on about U.S. forward? the how and growth know this Love the any you United year. cells trying if many I'm cell should what of going of the this in additional how just States and next like maybe to patients provide trajectory gotten collected, we the could to think understand year have collections in their be color

number bit I on color on Yes. of Evan, not they are seeing. where region. cell commentary number Stuart's to we're are globe I think collection But process exactly going give the the if have stealing really and pleased you January, of the about and set if very very just of the to ask December in you Stuart what CASGEVY thunder, when who little comment patients to And will patients activated that so expectations, each which specifically was is I approved, that and in already started am collection. we're ATCs was a cell around

So to is add? anything want there you

are so got the have seeing collections, on we're collections expecting we and build we're feedback delighted that in all that Only activations the trends we've and based said, already. that had as to cell of momentum Reshma to the

As during we've operating, ramp the and she United trends course are a said to of those foundational also be expect mentioned continue are including CASGEVY. going up which always we every to that we the was for obviously, represents patients year which to States, region in in XXXX,

question will The UBS. Colin with Bristow next come from

us pipeline. which won't or in of? we differentiated? you this see aware the Phase expect mean to Maybe XXX that be should you're does I how completed first I. recently the if And quarter think color And I to for, quick moves then sort any on could taking be be may, also one, XXX housekeeping a Phase on advancing you I inventory of X, any detail this give advancement pain just you

and into which I up Colin, to comment on let will XXX XXX. and me ask break questions. One first, I'll X on on back that come inventory, then Charlie

in I first phasing from prepared remarks, in some my channel Yes. the benefit international Colin, that we quarter mentioned of saw inventory.

expect I on quarter, was about. million begin $XX in That and benefit quarter. million to of that's talking you're $XXX the to in the to second reverse that assume the what I order So

to than to little ready medicines manufacturing What bit XXX we package, do things innovation. one out a into Phase On go. just to of waterfront and just because than take to is are little for There's elements coming terms reasons, of oral looking VX-XXX our why on specifically, looking is serial here can we this further preclinical mouth. go only overarching our XXX all need other may strategy. be X surgery not major who of management and bit the IV, that be XXX the all get ready very is in goal innovation That's those that a both the pain terms our molecules of Colin, by really II. in we XXX, to other XXX, patients to of is and further oral is one, able are But part for differentiation? is the behind. ahead serial including for for

here The right the properties progress goal in have to be that the drug-like with big ensure therefore, combined good medicines with that is second making is preclinical we development. NAVX.X, can also which

And little there. we're a looking And be ahead. XXX because on soon why already for. as next, really going So of that's further as data to we're it's the what all it's just XXX, bit working

Terence from Morgan come Stanley. will Flynn with question Next

was me wondering just in for III give directional East. insight I for you insight on presentation pricing I the a was data. well. on CASGEVY. as venue any if Great. reimbursement the any potential And Phase relates and can us just question XXX Maybe the then a X-part wondering Middle second to

Yes, we Congress those say, in you more data not on shall with meetings, publications. but expect XXX in on Phase should VX-XXX form certainly fall results. III only

a And So CASGEVY East. really comment opportunity Stuart in meetings. It fall ask us. I'd on say let the is to exciting for Middle me

reflect individual that's the provide in everywhere why the and say, Terrance, world specifically your philosophy commercializing patients to around price the from Yes, level, we data bit product where is to us, the the suffice have pricing the it, signed of we've there transformative of it. CASGEVY. more color And receiving going kind reimbursement benefits little country value on don't answer opportunity a an to at question, the which for that to lifetime but can same we're provide going accrue existing agreements super are we're the that exciting be we we

question will The TD come Cowen. next Nadu Phil from with

from Two us.

made Sustagen something a formulary or discussions. comment programs I reimbursement of government an you the could something And big suggesting your that are the sense particular, Brazil in help reimbursed and opioids legislative of us Can pain discussions initiatives, that Could opioids of about So access think absence and formulary that in then incentivize after be in you that some first, be the will will the that access CF small speak setting. acute in it's likely pain acute Stuart, how commercial you XXX franchise. could to you be thought more enabling And ahead give second, interesting bit setting on the effect. question. that? for

Phil. Yes, Brazil. comment I will in Stuart CF to both on ask

separate. there approval that from with on common sure on have formulary just is and of as the policymakers, awareness Stuart. and using are to make that and stakeholders same separate to discussions formulary have are to aware stakeholders They of Suzetrigine, of Over of we frankly, are just But the high both non-opioids. the we discussions but the theme our enthusiasm You well all you, as are opioid discussions suzetrigine, those discussions there very reimbursement page. those is regulatory know crisis. The

Yes.

opioids. looking really reduce generic say, that, seen we've at just currently Reshma, Phil, indeed disincentives in would institutions that which dominated brand So that to market for in so the a to a I to is by for are is what to obviously add non-opioid policy initiatives selecting and patients financial far trying

generic using Pain alternatives that's things a the looking then the to on is disadvantages to market surgical the important is a a at an in in that providing opioids. setting pain, which payment are D, so co-pay above the ensuring already patient are no like there additional and And Act outpatient for Medicare in center is no non-opioid ambulatory Part there side, DRG where patient there. And why branded

are patients right using in of kind are different dispensed medicine more institutions in that stakeholders the the the landscape. that's prescribed we're the the whether the this were clinically being institutional and having kind policy be slightly on the setting. Those is say discussions who I'd medicines the with much So based of and to there, which

describe that's they some So between I from would but the in difference they focus institutional -- policy side a obviously are They're the different the how the conversations. side. ways, have to linked

follow-up. generic that survey, expectation for have question Maybe opioid. Vertex would our as we a of and through to well? step a that XX% The thought just is Is physicians a basis patients recently did

perspective instance, happening. hope have happen be speculate reasonable I can't my step to expect addictive side and available, has on a a what's don't X,XXX an very therapy excellent profile, lack I patient from and for not to think But there would exactly including which very to addictive product when which has significant liability, with, safety potential. including effect it's of good control that's has what's a that, efficacy through really institutions. is pain to tolerability a going potential

estimate TRIKAFTA. So second launch a patients now I reimbursement answer it the national particularly part to in in as medicine number of be there, don't through already the something think government patients Brazil. of over would are to think I TRIKAFTA and access be now. we wouldn't prepared going did and advocating eligible agreement is that available sales, for your would those there's X us make would a That's patient for mentioned which X,XXX don't patients have of reasonable. those question, who remarks, allow the around we and named my around with have all to didn't Brazil, but be that We medically I which certainly I was

question with Fitzgerald. next will Cantor The Brayer come from Olivia

of And what's that but the know that priority quick pain? What's about build-out, clarification a revenue start CASGEVY. talked base when level acute for actually just to see you'll recognition commercial your on confidence case you'll I Stuart, and get program, your from review and you've

Just wanted finished the to patients cell having collection clarify collection versus that process. I already have initiated just heard X

Reshma. is this Olivia,

on to review I'll been since to most with thing collection, are the comment again, in months, CASGEVY. Let X received in On commercialization. ask we and whether X the X going just where patient on number question us just submission VX-XXX me or we'll of exactly take to the then about XX time we be. the X, their to pain, and is take complete On Stuart then X, some and what XX where set priority FDA we're expectations days but and for which with on the the final cell in last few we and after thrilled things say. has on further any journey. number not will exactly commented so review know the or We're lines is the approval, important ATCs, each we've takes not tell Maybe it acute to paying

indicators all leading priority the not review favorable. However, whether are will or get quite we of

fast have We status. track

pain when the medicine have have they launch FDA going have about has designation to where the to breakthrough me a and there. efficacy not high shown Stuart addictive acute that potential. I'll and the question We enthusiasm our that are it with we does over be high conversations we're for have and turn for out with

Yes.

So date to terms on kind and treatment recruitment be going going once our middle our going that, get our we'll the of well. of CASGEVY, revenue when recognition. of in normal infusion. we're revenue to regulators here. PDUFA is and is way. in where very process would question could has a we Obviously, rolling an are of extended small completed we the at recognition Obviously, selling the we molecule, This This is teams say, the be from unlike in expect distributing which an is are submission to therefore, it be I indication launch around and ready the we've

to I kind suggest that people recognition And aware lag, so revenue isn't that of are going with there CASHGEVY. really around of be would

Chattopadhyay come with Securities. Debjit will question from Next Guggenheim

got a couple. I

agency this is strength measurements. going is do First navigating ask or the Otsuka have approvable to data? on Or Phase GFR launch underway, the on ready an How is IgAN. for the cleared correction you to Vertex DMX, When about endpoint? you and likely in myotonia think commercially? placing are it's force then again, with And with IND will thinking I/II

dystrophy actually Let But both those. or talk had extensively. I/II about program On of to this in it me a haven't in DMX X. patients. myotonic Phase a chance is take that We is type

to known serious disease, in nephropathy seen defect, and lot so for most as the is the With nephrologist disease, to what as gotten going I to only know to in The IgA think in because about of this the chronic but to serious to And So disease we that thing efficacy the the not is yet is IgA leads we renal in real answer might I've haven't assess approval, agency diseases. -- have are or to openness want assess rare opportunity regard and to endpoint doesn't in GFR the phase know disease, efficacy opportunity On kinds over don't therefore, seen is disease well. the is translate that GFR we've point that for see and proteinuria rare at that function. Myotonia would therapies because is these study decline and target safety, for serious to looking as there. most end-stage disease But have and that of to I works time accelerated renal I a important a cause genetic that that it's is insofar this is the underlying trial. your nephropathy, a this very that specifically a transplantation. is on possible the any around endpoints clinical a be of important a one a death disease or the thing the decline

we've proteinuria. medicine So Phase remarks, through if preclinically that in many efficacy. clinically best-in-class everything is Poly II prepared have seen from has that dimensions, And certainly reductions I my and as high we but in across including a said

choose. will I drug physicians think the that's that

question, One Chuck. please, quick last

ma'am. Yes, Ms. ISI. will from come with That Bayko Evercore Lissa

So just X me. from

yet some you and there. the addition going different could just potential slightly may just had about that then there you the I nephropathy. of any for I APRIL, program. some down be on? benefits that's Was list. for kind points really for increase gross how don't follow-up about whatnot, price how you some this you sales in bliss of of key know if could kind the noticed I thinking a thought having differentiate might CF color quarter-over-quarter. point. highlight you U.S., you there's wondering or to changes, the just to of net think TRIKAFTA highlight because describe kind IgA Just how on you're for And differentiator more in Have what's biopsies that Can looked one great And your inventory of quarter, higher on

we and IgAN, we can this Let also It's first, can is with the fullness show that me of and see it's that preclinically, I'll APRIL. then how and the attractive share an certainly features have question all and we correct comment of to we this ask is that dual APRIL. on Charlie of You measurement On with BAFF you'll preclinically, that of And of BAFF IgAN one and in out most inhibition. well of Yes, take can as information, pointing this the do The certainly of the inhibitor. a time. as inhibitor CF. are dual povetacicept inhibition

a of a know, composite And It the data you along has because, very results. the hallmark those data from is is that's dual hematuria has particularly is available, with clinical data, encourage results this but WCN of the and hematuria it it look as and which with meeting has I this think APRIL, find BAFF showed, It poster disease interesting, results, you to that hematuria interesting on the has I that more However, already proteinuria. clinically inhibitor very, at GFR results. proteinuria, the proteinuria I'd remission. Alpine is

on Charlie to it turn me and over about question Let U.S. the CF

read in on into quarter the volume saw U.S. much Lisa, fluctuations. too strong growth sequential the year-over-year. I quarter, Yes, We wouldn't

normally that see net As the we isn't benefit increase reflected fully quarter seasonal the quarter we some in really of price balance comes do, year. gross the to the the throughout and first of the in

very the those the U.S. affect Chuck. strong the year-over-year U.S. very, So comparison. But all and factors in overall, growth outside of Thanks,

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

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