Earnings call transcript Q2 2023

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Operator

Good afternoon and welcome to the Vertex Pharmaceuticals Second Quarter 2023 Earnings Conference Call. [Operator Instructions] Please note, this event is being recorded.

I would now like to turn the conference over to Susie Lisa, Senior Vice President, Investor Relations. Please go ahead.

Susie Lisa

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

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

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

to turn now over Reshma. call the I'll

Reshma Kewalramani

Good After Susie. to strong all Thanks, quarter second year, joining you aspects into the across the momentum saw the a we continued start call the all and us on for of evening today. thank company.

billion to revenue And $X.X range drove we XXXX CF period. global year year XX% safe Our a first reaching guidance to more growth we expanded reach the $X.X raising second to revised this continues and prior are CF patients business full the half are product grow this product billion. than ever. of our quarter, In with versus performance, revenue

also of sickle provides the acute potential we expect deliver In in near-term for multiple excellence, another future severe our will opportunity CF. first in our which next transfusion-dependent to our extend commercial for combination cystic Vanzacaftor opportunity continue which therapy investing we triple for both thalassemia further the multibillion-dollar be of in VX-XXX in leadership and launches. exa-cel CF, disease beta In commercial fibrosis cell wave pain, we're in launches. and As to ahead

Phase our years diabetes the portion VX-XXX In addition to X program, of the clinic achievements the on type end progress to these year. are pipeline naked goal. in the include: in our continues IIB towards disease rapidly Inaxaplin marked patients. X cells track X programs Recent our the VX-XXX, X pivotal launches this the trial the develop and complete or VX-XXX, mid-stage by both to and remains study and plus clinical dosing In device now cells of areas,

Additionally, we for announced accelerated a we agreement expect strategic study therapy in programs. VX-XXX And an Phase line with the complete study to of end type the Lonza pain, by our cell diabetes XXXX. the now II finally, long-term peripheral neuropathic manufacturing for where X time

advancing and development, total, of disease past Slide In X X. on we're as stage in programs which late-stage X areas detailed the through now mid- proof-of-concept are

bring to R&D multiple our franchise, patients transformative in new areas. Beyond programs use and make the and exa-cel. the Duchenne's innovation. potentially quarter, business, This best-in-class medicines the NaVX.X for research with muscular first-in-class around pain portfolio from pipeline, program sourced also our gentler next programs of across has we're Vertex myotonic more both conditioning advancements agents internal X, wave in the additional CF reflecting to includes to this stage In clinical type the continued external and dystrophy CFTR bring dystrophy, and or to meaningful advancing for assets, disease globe

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

XX share therapy. we being better, the the complete studies XXX Phase these patients that III committed do end XXXX the goal SKYLINE X our combination vanzacaftor If and 'XX. RIDGELINE study all to to years ages in to in possible patients. the do am we're early it's While next-in-class above ages from and is release expect for XXX pleased so X X triple TRIKAFTA results delivers patients tremendous who benefit in to ones by and for to XX studies, and of I and

studies patients. high the in carries have The of triple to lead triple Phase the bronchial has patients the than a vanzacaftor vanzacaftor program II our of convenience in benefit, clinical And lower dosing. Vanza lower provide epithelial studies believe previous in and the improvements transport substantially greater with with chloride with We additionally, TRIKAFTA. burden. to with higher levels the chloride In than therefore, vanzacaftor triple expectations the to correspondingly clinical potential vitro direct in CFTR the enhanced transport of evidence is Triple on vanzacaftor chloride the human generated patients triple showed the once-daily chloride And readout function to of function TRIKAFTA. based sweat in CFTR assays from We, sweat restoration royalty cell date. further of totality in vitro, most showed

with CF in in cannot Moderna Another our mRNA CFTR modulators. from benefit our patients our is VX-XXX, who important program for more CF the at development partners CFTR portfolio therapy than X,XXX

third, small successful leading in both of We transport; cells. cells into have in and desired of of animals of reasons, X this enthusiasm what large second, for nebulize expression at protein we've and mRNA achieved delivery to protein HBE delivery date: CFTR key CFTR efficiency mRNA expression to approach of chloride the first, vitro; in on resulting based for high the levels an high

we of the of We and multiple ascending expect VX-XXX. CF SAD ascending to study this dose the enroll single complete initiate the continue to portion portion patients the dose in and dose SAD year. And or study

and patients gene patient diseases. transfusion-dependent our cure Exa-cel program for sickle XX,XXX. functional targets most Turning beta-thalassemia estimated disease approximately promise CRISPR/CasX-based cell now editing be of to population holds a an exa-cel, to for severe the which the onetime and these

the sickle FDA review will unmet front, potential high review cell advisory held regulatory patients. disease XXXX holds be a indicated PDUFA from beta-thalassemia priority with The XX, share accepted the date. in exa-cel March has to exa-cel date. transformative with our for has results the standard forward PDUFA and opportunity need, in we X the an look granted studies On the filings discuss to committee discuss the and December Along with the FDA the and

the U.S., in underway. the reviews Outside well are EU U.K., our exa-cel and filings the also for

Our oral months. MHRA and with the new patients secondary presentation, provided meeting follow-up met the most in and key the trials EMA some the June, basis filings. primary endpoints than that recent of Both EHA XX in were regulatory more of data

conditioning demonstrate XX% bone and of durable crises patients. by consistent safety The in transfusion XX% severe for SCD vaso-occlusive continue and and of from profile The to benefit independence as measured transplantation. results with patients exa-cel transformative, consistent EHA generally for marrow freedom TDT was busulfan patients

opportunity are younger patients for is pediatric underway. significant disease exa-cel and trials beta the thalassemia sickle Another in in both and cell

than intervene We and occur. the before in prevent focus This target of number and earlier important organ pediatric have patients area dosed half more damage have opportunity of patients. multiple enrolled ever both they the and complications given potentially to studies is an other

disease beta-thalassemia. thousands to U.S. transfusion-dependent we this As U.K. potentially the therapy bringing cell of and in to with patients forward and look sickle conclusion reviews the in and onetime severe dates to PDUFA EU, curative come approach the

the Turning effects of acute without late effective am or or all the that with and highly to and promise In XXXX trials randomized III our share pain of pleased results this pain pain, opioids. side program to VX-XXX, novel, studies, addictive the complete properties our selective in efficacy X next and that safety early Phase will relief available holds I a X NaVX.X year, inhibitor by controlled study of single-arm 'XX. end

has an pain pace this The efficacious, high non-opioid acute strong we rapid therapy. Phase of indicative in been the see III of which and interest need unmet program as

high with studies these Phase the for studies confidence methodology, genetic of itself; multiple in the predecessor and compared with Phase the and proof-of-concept and VX-XXX target; have of We to III validation the Phase II molecule given: the III three, design trials and program. one, similar the two, endpoints our pharmacologic outlook

model-to-severe the and recall would the pain support on that mule surgical Closing enable setting acute post-discharge phase been or ambulatory including across and which in to level prescribing designed Hospital home. acute has pain, a usage program III center in broad the

studying VX-XXX that represents type and another of peripheral yet significant unmet pain opportunity. area A or DPN. neuropathic peripheral need also of diabetic are We neuropathy another in multibillion-dollar market

in recently has year XXXX in this of to I pain the We DPN complete neuropathic study or current positive with we study VX-XXX this data we the proof-of-concept by with time to accelerated peripheral the dose-ranging a and molecule completed VX-XXX. late enrollment. results proof-of-concept share will share The have study XX-week Phase is previously expect have am line This 'XX. and pleased delivered early predecessor end in DPN II study. for

type evaluating we're where for diabetes, X differentiated type islet with cell-derived, to diabetes. cells people insulin-producing now Moving stem fully X

functional including a of cure develop North the people diabetes, living than America million patients the to more in is X.X for with alone. and millions Europe goal type Our X

at engrafted and or the improved endogenous all program and VX-XXX the X is control in reducing had data at the on the islet immunosuppressants updated concept. standard presented of trial presentation VX-XXX or treated produced system. while for Diabetes with recent program, demonstrated showed therapy have study patients we which naked foundational exogenous to program cells, the A Meeting. In our The ADA insulin islets take We VX-XXX the The B clinical patients TXD cell American that use. proof eliminating immune Parts insulin Association glycemic of already protect from cell the

at of independent. long-term events, as on we've a Importantly, Based insulin trial least of AXCs dose. VX-XXX X clinical our therapy on are trajectory in a the were in saw course already Canada. follow-up similar concurrently the and global Part hemoglobin elimination X as patients who on Europe has full at patients U.S. with maintained addition severe patients sites results, And the to and complete X where advanced patients opened were treated study hypoglycemic the earlier open now below were target follow-up. these now plan, X% their Further, in those to with part trial the C, of year

these Our for has share no a which study device immunoprotective VX-XXX am enrollment proprietary concept have in device. that second initiated patient. program, of pleased in the to demonstrated program there already and VX-XXX, plus requirement dosed have the I is encapsulates And the cells already we cells proof first hence, same immunosuppressants.

diabetes which need the to immunosuppressants. collaboration cells we program make our for is them This progress stage this system. these X edit program to in obviating to research same third into continue we immune another we program. cloak path differentiated CRISPR use fully from CRISPR/CasX represents Therapeutics and hyperimmune make our the The the with to in March, In type expanded to edits

in patients target first genetically Europe patients portion to XXX,XXX AMKD to defined kidney underlying Phase a the with now U.S. the and the II/III cause AMKD, pivotal pathway that alone. on single and Phase enroll in or IIB to remains year. potential this dose to program for Transitioning of U.S. a complete is of a the Inaxaplin Inaxaplin Recall VX-XXX, APOLX-mediated study track medicine dose-ranging affects disease or the and disease continues The accelerate adaptive approval to the with study approximately

to genetic leader testing and AMKD and through testing continue to awareness services. including also disease pathology a partnerships enhance with Arkana, diagnosis, a genetic renal work in We support in Natera, to availability leader

our affects To close targets and in people disease deficiency or program, Europe. liver that Alpha-X this XXX,XXX both on manifestations small America and of estimated the an lung antitrypsin molecule update North approach our an AATD

molecule AATD on its with second, remains levels exploring of properties year. for polymer forward functional patients a study clearance X next-in-class molecule II enrollment that the Overall, program liver complete track hypotheses: we is a in duration better is to Our VX-XXX; and and with the and longer year. drug-like AAT Phase this more I The by and plasma later program greater this projected the look results and anticipated potent with complete both to assesses to sharing VX-XXX, is treatment with XX-week trial VX-XXX, XXXX. potency ongoing Z AATD multifold in VX-XXX. is first, end program of Phase

exa-cel. on our With a turn that, details preparations to for update, to over launch it commercial provide Stuart I'll including

Stuart Arbuckle

adherence Thanks, we results for of second continue we as our treated quarter our From a on for strong Reshma. all patients CFTR levels focus with commercial patients and our modulators reaching maintaining had medicines. eligible perspective, high

to sickle we are in time, for addition, pipeline as our the At the support same pain and of AMKD to triple cell CF. and combination including to In X awareness new launches, other disease prepare we vanzacaftor such capabilities type assets. investing developing for manufacturing commercialization diabetes. potential multiple in capabilities moderate disease near-term in and VX-XXX beta-thalassemia severe continue exa-cel acute the transfusion-dependent severe for in

Given the our granted in U.S. and completed also our hemoglobinopathies the where PDUFA dates opportunity nearest term is U.S. exa-cel and filings in being we in have Europe regulatory

my our of exa-cel. being But with there pipeline I will more comments benefit North on At for people Europe modulator. but a CF. activities the beginning XX,XXX quarter, yet who briefly focus could on This Australia, about prelaunch than America, CF with year, in CFTR treated first were this were not and

We new this and a medicines approvals reimbursements continue uptake additional business. these patients will for to reaching near-term our on with patients continue driver of a growth and be focus bring to through to younger following our

was XXXX and groups. our quarter was by with consistent medicines Second outlook in XX% driven younger expanded age revenue use CF growth this of primarily of

written first and late across Following April, strong in X U.S. interest hours X we've after to the approval patients. prescription TRIKAFTA the from seen approval all children CF eligible uptake in the with just community ages

Outside and geographies. following approval, successful launches continued in and the has to growth ages older, KAFTRIO multiple in be strong U.S., reimbursement XX patient

In to this the end for ages addition, of for X in expect we ORKAMBI received in KAFTRIO the year. in by July EU X we children EU ages continue early to approval X and children in approval X to

study actively X. ages are to in X our also enrolling We KAFTRIO children

see reimbursement in continued modulators, medicines our for patients. CFTR growth uptake our approvals, we of by portfolio of Overall, driven younger and

our benefit future offer and provide of growth. a next-generation approach patients for discontinued longer will could our And those drive a Notably, than mRNA prior for approvals who for therapy therapy. treatment triple term, have option cannot the from X,XXX addition, to CF seeks In modulator improved VX-XXX, new who more also CFTR medicines efficacy CFTR modulators. patients vanzacaftor

eligible and and insights XX centers both medical in physician the earnings details and can holds previous provided be geographic diseases cell to Europe. patient estimated and with now patients exa-cel U.S. field and thalassemia proposed for I our patient severe concentration have of chronic patients those population, high On disease disabling the training on the care. quarterly market teams extremely and which Shifting curative of our of in The for potential burden and hiring transfusion-dependent sickle life-shortening network that an of calls, ATC education from research. and XX exa-cel beta and

perspective This on with and quarter, our I'd like to reimbursement access globally our and and payers provide policymakers. discussions

U.S. different therapies and dozen cell to channels experienced Our progress make payers With in teams and than across continue payers with Europe. government excellent commercial in are market, more discussions the pre-approval a transformative with the increasingly gene types on both and these of therapies.

have XX% In thalassemia, are with or XX% remaining private sickle patients of of the U.S., patients by programs approximately cell disease with through government via the covered coverage majority insurance. the and beta Medicaid

availability an cell already a SCD. from XX have for states encouraged administrators the coverage including of enthusiasm distinct states the of by of in of cost marrow of the procedure. administrators prepare with policies payment Medicaid accounting disease, exa-cel the XX for for teams the separate for of are prevalence well state like therapy, the with the the on therapies with Our focus all cost enablement steps they patients of sickle from engaged bone as highest Medicaid estimated Medicaid the XX% to proactive taking transplant as particular the exa-cel We're

the model Medicaid-focused towards cell its The federal and government's treat Medicaid disease and potential and state CMS model to to of pathways interest recognition gene finding anticipated XXXX. therapy exa-cel of payment cell of value The innovative in continues solutions evidence launch clear like programs. for the gene their progress therapies make in is also sickle access transformative

payers. commercial to Shifting

timely X coverage And We of is upon with decisions approximately lives. commercial approval. including have for our commercial facilitate payers high potential goal account of engagement to that exa-cel a top XX% payers, had levels the

disease are discussions estimates, epidemiology models. been encouraging our data potential clinical on pre-approval have Our and focused and burden, payment

working MAA well in underway to targeted for the also patients thus, reviews secure Europe, are paving markets. way reimbursed and In are we our access European the on

on systems burden the We care have to them been and health society. systems significant on educate engaging with health disease patients,

with cure. in functional different potential their models In authorities of the meeting health value addition, and onetime have holistic to understand to European been interest a we communicate payment

unmet of interest particularly there new for and Given urgent beta from patients in disease with for high the eligible patient the physicians, sickle and thalassemia, significant concentrations population. is cell need geographies treatments

time at be To remarkable a conclude, Vertex. it's to

more patients exa-cel make approval. to multibillion-dollar of promise to continue transformative our the We grow approach for for progress and CF while we treating as the resulting opportunity continues excitement potential patients market

acute We and vanzacaftor CF have of the lives. VX-XXX both near-term in which pain, are for multiple including the improve also to in launches, potential preparing dramatically additional patients' triple

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

Charlie Wagner

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

expansion revenue with was XXXX year-over-year U.S. XX% year-over-year led by well markets drive helped the growth patients X% younger as quarter uptake TRIKAFTA-KAFTRIO extensions continued the Growth reimbursement of recent in to increased increase TRIKAFTA in label Similarly, X in billion. ages as groups X. to approval in achieved U.S. strong age XX% recently outside Second $X.XX of on following a FDA groups. revenue younger age

Second revenues certain half. channel in draw increases quarter inventory second half first international in also in which are down markets expected the from benefited and to

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

type XXXX to CRISPR our Second reflects for a expense program IP in hypoimmune milestone made million X for quarter Therapeutics R&D progress $XX diabetes.

investment pipeline advancing which and resulting and IP expected charges, acquired and in in for for vanzacaftor the VX-XXX continued significant Aside type driven diabetes. The as external R&D operating and pain disease includes the prior was versus growth for by assets clinical X in different expense our the year late-stage clinical areas Triple-NCF investments most acute higher from included increased our research across of innovation investment areas. X studies mid-

pre-commercial addition, launches, and to near-term anticipated will continue we programs, exa-cel continued activities multibillion-dollar given other market late-stage for opportunities the and In patients appropriately. and potentially mid- our for our invest we benefits to transformative

income XXXX was quarter operating billion non-GAAP second $X.XX Second quarter to compared billion of XXXX. $X.XX in the

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

guidance. switching to Now

revenue to billion XXXX in strong the XXX now compared our on our $X.XX including billion, to product billion of the Given we of our percentage year XX, increase to detailed half guidance our as net with $X.X to an expected an $X.X guidance revenue are $X.X Note TRIKAFTA that launch our rate, U.S., Slide $XXX approximate X first number we increasing $XXX of to and headwind execution, results in consistent prior ages the patients successful to XXXX, full prior X billion. of expect for revenue growth our consistent includes revenue range point this million million expectations. CF

cell to cystic our X revenue fibrosis exa-cel product given products from PDUFA reflect for revenue XXXX addition, U.S. date guidance December only. In and continues disease, sickle

XXXX expenses development, reflects SG&A of million IP acquired IP raising to CRISPR from a $XXX in This from R&D, in R&D Entrada guidance. $X.X higher R&D also and with are X billion, $X.X diabetes. We and with collaborations guidance non-GAAP to increase DMX expenses increase for type combined business our range including new billion an prior of

the and year. of now guidance the $XXX milestones the start non-GAAP projected million of expense upfronts million to approximately includes $XXX Our XXXX compared operating at

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

the markets. to programs afforded continuing in of funding commercial launch focus model specialty an the represented our dollar while business also near-term expansion attractive of leverage by are capabilities We with by multibillion our in anticipation opportunities our potential

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

revenue closing, strong and trial and Vertex for excellent XXXX. quarter second the In of internally externally. growth, important milestones, delivered completed invested results regulatory clinical We on significant delivered updated programs

milestones in in multiple continued highlighted on Slide progress As anticipate XXXX, as further we important advance programs mark disease XX we continue to to our areas. our

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

Susie Lisa

the Charlie. question? first Thanks, you Gary, view can please

Operator

[Operator first Cowen with Instructions] Nadeau & from Phil question Our is Company.

Phil Nadeau

prepared likely, be least Just Vertex your mentioned second, in committee on the on thanks prep. a have is you to for at what the that exa-cel. Stuart, couple advisory And or all the for In discussed or remarks at does is an likely of comments then release, advisory the debated you any mentioned sense committee? commercial press

some X We have for post speed patients from and do Vertex get therapy starts gene that? to having learn after at What does approval. seen you the could exa-cel off a are relatively to slow not so increase approval? cell with launches months actually or late What of adopting patients which dosed

Reshma Kewalramani

Reshma. is this Phil,

With then me there take of in past, to mechanism launch question the the And forthcoming. Let either. more This that an discussed comment have Stuart be AdCom I the new informed committee. as given us date FDA we've your the part which expect further exa-cel, have will and the is the regard first has the on advisory of not don't I'll we to of ask readiness. unexpected today be don't will approach Those we as details. We we'll action. commercial know

advisory take the committees to conventionally, date. X about the X months before place PDUFA However, usually

the looking general that's that at. we're So framework

voices data, talk at to risk and to to to potential We opportunity the patient's the committee. share transformative our have and have to talk benefit are the about the very advisory excited the heard about

Let on Stuart comment me readiness. to it turn launch to over

Stuart Arbuckle

our on. are close that's with as to doing and approval entirely try possibly the both the opportunity really and get as so launch most in conditions know, U.S. without we internationally we provide and regulatory a payers, to and and is for thanks you secure Obviously, first as be And we're step access reimbursement, Phil, that's within access my no Obviously, working there can. that Yes, for to important for to going what why the everything on to get I focus because comments question. patients treated. and are not we can but the access successful control reimbursement reimbursement that's

process on the of from remind the future, In curve is I that's But high. depend obviously, interest very that patients. almost as you We has that the previous medicines know, know CF terms a patient now, do actually is vertical. multi-month go that's expect to uptake to exa-cel. on slower, that, largely to see to be have to returned before I've obviously, treated said And Obviously, coming with where patient in drug through the and have to they're get exa-cel a launches they with this the we cells edited, the to a their be want that to go kind with to you dosed to than as through going bone the for our we in with product. that, cells physicians site with of exa-cel physician uptake calls, because, would have are the then have equivalent The the decide then marrow they and of their transplant gene has collected. schedule therapy. essentially They a

to a it for start process multi-month from is any So finish patient. individual

launch, a said multibillion-dollar do believe It's there's in exa-cel see always big expect so in than market but cystic uptake to in to as lot continue we've And we that's opportunity a and we opportunity a why the slower fibrosis this we future. rate slightly of interest. be

Phil Nadeau

if may. Great. I One follow-up,

launch. place Is recent be the would warranty On agreements something reimbursement, gene one considering think or seen in by you therapy that helpful? you're put we've

Stuart Arbuckle

again? Sorry, question. Phil, you catch it I didn't say quite the Can

Phil Nadeau

Yes.

reimbursement think agreement. a Vertex of as of something launch helpful for reimbursement gene a structure therapy launch like one be would the that Is recent the in part is reimbursement of considering agreements, included exa-cel? terms or warranty would In

Stuart Arbuckle

Yes.

of looking X you they're get range are Phil. what of reason ask options, you different We for a is considering for, if The answer. payer, X kind that

you pay, another ask you answer. another If get

done functional as be interested onetime that. outcomes-based price think and in we've at who There Others agreements cures. upfront things a we're more just benefits for like looking just on paying are to straight flexible. going are with look the are to So cystic be to fibrosis, I much going some of

word of flexibility. designing of models defining in the now, and payment right listening I be. we're what But and kind probably nature key mode the so And will think our is

Operator

next Bayko The Liisa from with Evercore question ISI. is

Liisa Bayko

good on quarter. the Congratulations

on give Just for a X pouch? plus the data wondering sort next your view if could the cells the type program, cells readout us both and you of diabetes

Reshma Kewalramani

VX-XXX thing. the the on Liisa, naked cells program. TXD regard let's to Sure side, the program, call that's, with it

an should You the have to conference presentation. diabetes be there fall readout oral at expect will where a data

program, the regard program, you dosed, plus you the from initiated program device patient. immunosuppressants, On expect We've milestone just heard a of that's we as which reached not when be device my to enrollment. sharing readout hear first remarks, We should either we've cells with XXX patient. require we cells us in prepared to just And from a results results terms communicate. data that plus won't to does or by in have decision patient the

Liisa Bayko

focusing a I market about you are could curious and commercial of How -- thinking obviously where? market broad on have could I'm path be Okay, opportunity. a rollout you then on estimates fair very how depending it. you that very centers? of and range certain mean be kind on are enough. the think wide about the to be -- of types for pain, kind going And as you your think about this

Reshma Kewalramani

right. Liisa, an We Sure. you're enormous as see opportunity. this

opportunity. to you Stuart tell ask approach how me plan we Let Stuart? to that

Stuart Arbuckle

Yes.

management our at So acute acute they prescribed be the And in prescriptions medicine of of just XXX very and pain, same acute covered sales large for IDNs. a accounts pain true going obviously wide the so which that are can pain of pain prescribed being which on the by facility. activities. dispensed primary they're given fits cover then is sites with the are shy XXX studies concentrated of but the when in leave of that, also universe of by prescription it's X I'd subject concentrated to ongoing or percentage specialty for they're pain. Liisa. force institutions, somewhere the prescribers. large be percentage to -- and on That true centers, a time, prescribed about range prescriptions with is our prescriptions around focus one the facility. a focus is settings commercialization their our positive whilst approximately are in say true indeed that we to is a a acute is launch discharge That in in therapies can centers indication range XXX And going like the or the We things where institution ambulatory markets. surgical that think be equally first their nicely patients Those either are

Operator

Richter Salveen [Operator Instructions] is next The with Sachs. from question Goldman

Salveen Richter

Just a follow-up on the acute pain program.

understanding apart a help target you upon just to aspect, data given done the mentioned, to marketing outcome out whether of -- this regard here law can a positive to there's falls played kind pain into what that prescribers any understand, of -- to successful to how the with launch and from contracts plays logistically out idea be kind Just no the well. us this? any of and regard with ensure the Just ensure be how needs you needs you can

Reshma Kewalramani

Sure. I'll Stuart to Salveen, ask comment.

Stuart Arbuckle

thanks Yes, question. for Salveen, the

the safe consider we VX-XXX existing updates likely be supportive their and other a there's of to acute likely of things pain treatment non-opioid see are number pain. One a guidelines, effective think is, couple medicine. are in once think to there the is I availability that of of guidelines I a

I can for change understandable reasons in years and few restricting prescriptions, In of for length what think and have them, over restricting policies going put have about been hospitals increasingly focus. types of one who the you prescribe policies largely that time. which states have see are in for all place addition mentioned patient we last been The to their

a that getting being we VX-XXX. policy subsequent And the potentially I are to very supportive will of beginning the systemic welcome that's to of focus initiatives uptake think which medicines the change to those I think VX-XXX of see pain non-opioid changed support like approved. it

in efforts, think to own at So there's our of addition commercialization I launch. the a number supportive

Operator

Question is Meacham America. Bank from Geoff of with

Geoff Meacham

lines, wanted had time what on improved for that commercial kind And guys success the expect know the kind you when -- from think, of think follow-up see I the like, today are optimization on to you But focused a I what What as some that? should know what well. I of guess, Just that I and we conditioning for? exa-cel. look of about regulatory are regimen, data we'll that? does are

Reshma Kewalramani

about regimen conditioning, programs people U.S. disease the profile for academia internally, with, what full opportunity see with that in active and partners are we which benefit thalassemia like problem, problem. our And see approximately a and this forms people we sickle serve to on XXX,XXX cell thalassemia. disease Geoff, most gentler set sickle as with or and launch and will conditioning at based Europe this think on we and program cell severe this the the the the looks other working as positive of CRISPR are have an working improved the is Sure. the of XX,XXX we with busulfan risk exa-cel beta having What beta biotechs

And happening do problem this months solved. that so a coming years. this see the tomorrow I see a as I will It's in solution not be but and

and sparing have the very not you targets those have effects cells specifically the doing, to side What the other busulfan. the so in we very cells. busulfan, conditioning of including are see of compartment cytopenias significant a And limited the the cells that with that opportunity regimen all hematopoietic stem see that is to

will do and in coming I see of you'll are that the I is we think this we think see and working broad mean I decades. months others a progress So on for an our it area don't and and do the in because solution years. that because application

Operator

with Our from Robyn next Securities. question is Truist Karnauskas

Robyn Karnauskas

departing. and we're noise. Sorry plane for the a I'm board on

of bar what the step Lyrica. your previous to that for So drug could the question similar neurotrophic with you I for us kind know study is, Maybe previous is looks your chemo-related in in whether We've diligence might huge it thought wanted is you well. some a know neuropathy need. unmet second. peripheral I to in work this the done a due as mean? population

Reshma Kewalramani

Yes.

So overall profile benefit Robyn, for pain therapies. together the better is bar to risk taken than have existing neuropathic a

we has limitations that for because reason use central limitations you system the the frankly, is what best recycled neuropathic pain there terms that the the know, depression reusing also nervous side. As are And we is fundamentally have. from in efficacy existing a that's comes we but of for medicine for on therapy are neuropathic that safety/AE pain

Lyrica we're diabetic for II and neuropathic Phase context. a arm for what from is to baseline has study going pain change peripheral improvement looking also proof-of-concept our So dose-ranging scores, in be

Lyrica context. So we'll well a arm the to the effect see as able for be treatment magnitude as of

Operator

David Partners. The next is question from with Leerink Risinger

David Risinger

Yes. AAT please, the to to efficacy results frame for timeline watch two your to I you wanted change gears, Could next candidates. potential XXXX? And in those readouts year?

Reshma Kewalramani

about molecules, study. this and a a which we to program. which everyone ground one, I in have is making asking David, is through Phase X that program Phase where is VX-XXX think our way VX-XXX And II I study its you're just AATD on Sure. the

fits Our and fact it disease comes the a program that the excitement for strategy glove. Vertex like particular this from

and are the target that this disease. order you believe We molecule medicine. the the And transformative manifestations manifestations. have one do a is small holds only liver to both treat I both and our of liver lung in seeking to approach to treat potential need both lung to And

long-term Phase are long-term II of It's And at clearance there, in polymer. levels liver and the Our of impact blood AAT dosing study. on study. VX-XXX is in study a which is the we a both looking XX-week functional

polymer might our analysis in ago, studies. the a levels in-human in a our reduction through years serum a You data in Z II the interested on why recall liver few XX-plus post-hoc from study, And we're polymer. percent going we which saw that VX-XXX XXX of is so first we Phase are

look but under sometime of that Exact timing, more at the we'll by by dynamics at these expect our we'll be I we're so have share enrollment do to trials belt be and time. XXXX going get next both able need to expect I from year, So sharing few year. results results the results the a months we'll to I that next all sometime to that expect

a it's 'XX milestone. So

Operator

Flynn with Morgan The next Terence from is question Stanley.

Terence Flynn

Stuart, you at that be mentioned the there could year not were on start eligible. potentially drug XX,XXX about of patients the

new Just assuming where end, XXXX achieve that figure you your guidance? wondering will

Stuart Arbuckle

Yes, Terence.

years the accounting all forensic detail gone of giving last to the of kind few kind the of of we've away So over different patient numbers.

We to And in this we've as including treating progress updated in of change, kind in we're going reimbursements beginning or talk give and for -- to that, including patients, said in remarks so years this following the may year so his not we time. where we've and last about our I'm if of detail the in groups other guidance age more of the time. more may there's But we've mine, update estimate substantial a in secured into when I countries other at next make good year. you couple done an at continued But not set than at we and younger those as numbers going launches. Charlie

Operator

question Wells Next is with from Mohit Bansal Fargo.

Mohit Bansal

of between chloride on talked how some the -- color chloride more vanzacaftor sweat this correlation about you get Is improvement combo, not to trial. amount chloride [ph] I'm the there the SCD versus mistaken, reduced you with XX% the do wanted cast about dry sweat the was Just -- a than improvement. improved improvement the combo. sweat If

trying just understand think this SEB bar for combined? to we new So how the should that about

Reshma Kewalramani

lung the both the and with in regard for and next-in-class triple, this age ORKAMBI With that pivotal a TRIKAFTA. way early you And of year. group Mohit, our plus from year all our the association is through we program all CF. X sweat can very on SIM I to in question about that's KALYDECO chloride and age CFTR think across between vanzacaftor program yes. chloride in the XX-plus-year-old and to regimen There that see is Phase from group your results complete studies previous improvements This strong III next year the modulators, you're ppFEVX, and function. talking sweat expect improvements that's in

So is strong. that relationship

qualitatively have the from what have very been in hard believe you the even our important triple, reason in not experiments, this triple, expect preclinical the is is so, order HBE vanzacaftor cells. than of the vanzacaftor let me Or a the assays better another it for but we but HPE predictive I in tall such the vanzacaftor terms triple. to the In which only way, vanzacaftor should quantitatively including preclinically know triple put TRIKAFTA high enthusiasm

call look triple on -- the variable sizes in make and a more we a when chloride II than TRIKAFTA. ppFEVX better endpoint. the Phase Phase It's have been studies done, because obviously call vanzacaftor across hard are to is ppFEVX have the And II that smaller sample the even studies, sweat

sweat that collected, I to vanza data is the chloride. So at right look of all sweat of TRIKAFTA chloride. the is even measurement from And on than measure think we've indeed better

Operator

Yee The next question is from Michael with Jefferies.

Michael Yee

neuropenic study, was enrollment. pain II Phase chronic You announced that complete pain the

guess, placebos, early of is 'XX, exciting side, get I on because So chronic was there's chronic guess confident biology of that's I end data the as the you early you as super and some also, 'XX. Can strong, quite even you the opioids, little talk bit pain and also and with a last vicoden But challenging studies also gen. can I one can about, data data, guess, historically, the mixed feel acute well results. be with

consideration to II? Phase a ask the this probability success give take should study confidence just acute we your into study how from about So and it just versus this an around I wanted expectation standpoint,

Reshma Kewalramani

the use. and And studies Phase confidence is Stuart available acute conducted various of terms the the genetic a that and you acute And is program safety My single-arm acute that acute clinical as those both the from program. the allow on VX-XXX VX-XXX diabetic third ourselves for trials early equal late our randomized the program what studies, RCTs. Those X expect it of the you're level validation moderate-to-severe with in II pain in pharmacologic the molecule, results in X X of that pain III to Sure. bunionectomy, And will pain X are year, be described in neuropathic the is a the next. that this neuropathy abdominoplasty. the validation which broad, and right, program for I pain is label confidence Phase double-click With from study target also target. regard could we comes in of predecessor same peripheral expect the to efficacy the settings

is, there we gosh, would run. goal see that acute II in a VX-XXX the And Phase we if home for pain, what saw be

context. arm where is for neuropathy program, also So multiple-dose-ranging study have we peripheral proof-of-concept gabapentin the a diabetic a this

and looking context. study results there year, in be the is the assessments be we their you'll of should the terms pain pain is versus enrolled baseline be should from point have fully XX-week in next. that's make score to for time you fourth this available That late and arm what in the to early improvement So gabapentin able endpoint when also

Operator

The with next Capital Evan question Markets. is BMO Seigerman from

Evan Seigerman

needs a of the Kind patients to to follow-up to be to question what on triple added benefit Triple, about Vanza vanza the versus Talk TRIKAFTA the switch. of Mohit's get

levels wild-type of levels level ever mentioned sweat chloride? getting patients get to also of sweat carrier a of you You to chloride. Could

Reshma Kewalramani

Yes.

opportunity the Let and to of Stuart it we're turn commercial the question, vanza seeing through take Evan to how me the triple. over then for I'll second talk your half

at manifestation those mutation, so look no parents, you you at carrier have of carriers of we're have carrier chloride, when those So for chloride. targeting levels don't are why That's of of CF the you sweat virtually sweat disease. who disease. When levels

that gets CF. continue But to with that a ultimate the highest are just carrier we've levels bar our and patients CF, to and -- achieve. will carrier sweat you get more that's there, already don't of you triple potentiators that's additional the The to And some the Stuart? us not and -- research why patients that's there. That's fundamentally are identified a chloride. bar will and triple TRIKAFTA goal continues like unaffected. I'm vanzacaftor chase. not if fundamentally I'm carrier a I get You're of we of why correctors me, patient you But

Stuart Arbuckle

Yes.

through through said, So outcomes be that, uptake in Reshma of and in will as of CFTR function work. described delivering it's that CF sort an from the what's -- increases link demonstrated in of earlier, levels attractive our profile, benefit the because of between chloride increased speaking sort attractive and the with function measured in CFTR the proposition improvements in where that as has own of of itself if clinicians vanzacaftor Reshma know profile has and of and terms been terms sweat we

years but we years. of population In consider welcome probably additional also an of a over who north think treatment -- who have discontinue there's being will been which be think a CFTR And about modulator I had to have group X,XXX you the talking should potentially discontinued another patients we there know transitioning. over have important It's the number their available. modulators. were somewhere an do addition, that's on to do patients want is who option I patients CFTR

Operator

question The from UBS. Bristow is with next Colin

Colin Bristow

reasonable just still question. Congrats plan, in on if assuming DMD have half? Are on to the then in file be triple I quarter. would you then Maybe one to CRISPR-based the IND XXXX? maybe goes program. the to some track the of second all the could it clinical vanzacaftor a And quick And expect to data follow-on

you need think a major switches you do as patient launch what component to see to a Just be just this new that launch? for acquisition is of versus a

Reshma Kewalramani

there. the one do see there then DMD. on questions need about what separate triple vanza I Colin, and X to and in are think we One

DMD, I'll to question, the tackle me come Let DMX back vanza.

out the as a whole I'll broaden about question, and to muscular dystrophies talk I'm and DMX. it On going DMD. DMD to

from second multiple have DMX And in them in-licensed those for We are in lead our half for the now of in program Entrada. in program both half results IND-enabling have of through second one and this to actually studies is well IND of DMX the should And file both 'XX. IND-enabling that have DMD the as timing We in remains DMD also year. the going program is and in as studies for the DMX. lead programs in DMX. programs that already we The that

level disease. with And chloride who have is, to gene, virtually by they see program a Stuart sweat assessed of way chloride. With with measured the And in the study designed vanzacaftor in CF sweat is TRIKAFTA that is regard carriers, looking chloride no -- for III Phase vanza, versus the we're primary description that. carrier and to again, is reason those levels. patients head-to-head the just the who with have covered X of sweat the manifestations I endpoint is What the is think that

we're what that's measuring. So

going We in have are, course, there. of PPFEVX to

have I'll than profile on be, has vanzacaftor combination. the improvement also levels, patients. it royalty real if add we said, TRIKAFTA triple the to that it as the And Stuart is, a chloride to to burden sweat lower as expect described I value

Susie Lisa

brings Reshma. time. us Thanks, Could Colin. please? Thanks, it That Gary. to close you out,

Operator

conference concluded. presentation. today's now the you and question-and-answer concludes This the also is attending session for Thank

may You disconnect. now

Vertex Pharmaceuticals (VRTX) 1 Aug 232023 Q2 Earnings call transcript