VRTX - Earnings call transcript Q2 2022 | Vertex Pharmaceuticals Incorporated

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Operator

Good day, and welcome to the Vertex Pharmaceuticals Second Quarter 2022 Earnings Call. [Operator Instructions]. I would now like to turn the conference over to Charlie Wagner, Chief Financial Officer. Please go ahead.

Charles Wagner

Good evening. Vertex' Officer. Wagner, Charlie is Chief Financial This Welcome Call. second quarter results conference XXXX financial to our call, prepared tonight's On making Dr. remarks, we have Reshma Stuart President; myself. CEO Operating Vertex's and Arbuckle, Officer; Kewalramani, Chief and webcast We that you this you the recommend slides access listen as to call. recorded, being available a replay is on call will website. our the and Also, be

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

Reshma Kewalramani

and wave our Thanks, the in progressing we getting clinical revenue based advancing for progress $X.X billion now from and with as reaching on guidance eligible in patients ready good make clinic. through or secured, updating as in the in portfolio region, uptake as enter international $X.X And $X.X all are CF rapid QX U.S. reimbursements innovation grew our next revenues billion. XX% $X.X CF the development, of disease to $X.X programs to recently We're goal year-on-year billion, strong R&D late-stage areas well continues the to towards as continued to TRIKAFTA our the to also entering Based the of new of Charlie, our product X billion. progress billion well on medicines. making all. Vertex evening to uptake this

designed last the to coupled quarter. drug advanced new inflection broad, leadership deep brings highlighted Vertex of research point differentiated expanding This odds point success in R&D increase is this development. stage is rooted clinical with which pipeline the in our strategy in and to working. This is and CF, inflection Our and discovery strategy,

and are programs patients in not programs areas, cell multibillion-dollar for individually these First acute past APOLX-mediated market diabetes, disease recently, kidney they X And seen including transformative, therapies disease, curative together, CF more pain stage. it of we and that if Vertex. taken all Each for represents multiple beta proof-of-concept a now potentially sickle enormous opportunity. deliver in represent thalassemia, the have potential and disease, type

continue external X balance or deals Verve we've acquisition, efforts the in with to our use efforts. we to strong invest With as to growth Viacyte business our for development of early diabetes in rapidly to announced today internal and assets increase to The internal programs. our has guidance from strong do addition, type sheet we the Catalyst accelerate sandbox $X.X stage pursue potential continuing development you complement external multiple quarter. our $X and and internal In complements see diseases. the us revenue are OpEx accelerates billion, advancing billion and innovation in innovation, example, collaboration this to the The that our pipeline,

of our to progress. CF. me review Let now pipeline the and with turn some Starting recent R&D

if TRIKAFTA who effective ones the For determined to set medicines, high it more can possible are has a modulators, so. we CFTR from develop very patients But to benefit is who do bar. be

and early patients Our next. end we XX on combination, III track of Phase complete older, years development or this triple enrolling VX-XXX/tezacaftor/VX-XXX, in to is the next-in-class enrollment and remain now by year

trials, initiated patients X old. in we've and to years SKYLINE the pivotal With development the SKYLINE XX in VX-XXX/tez/VX-XXX X X of recently also progress

CFTR a any make not benefit partners from an mRNA we're at modulator, Moderna. Lastly, do for with patients who protein developing CFTR our therapy cannot and

IND We half track the program in file the this of mRNA second the for are to year. on

CTXXXX. to Moving

advanced data most patients pipeline and treated we CF severe is thalassemia. presented exa-cel, In of data beta outside exa-cel Annual in of for Hematology cell longer Our June, gene Association. at our with and program sickle disease or Meeting with editing the program European the CTXXXX data, cure more These that follow-up be the onetime continue follow-up months with autologous with for myeloablative conditioning consistent patients the included to and which these durable, bone XX to marrow XX up patients demonstrate a functional continue potential holds of be and to exa-cel safety transplant. to

the the we filing next filing year. in on recently thalassemia package in beta remain both submit EU disease and package. MAAs QX the have our the U.K. have and terms reached discussions in to EMA and sickle and on this We In the with steps, MHRA concluded cell of on agreement of track

in to forward regarding the we with We and duration coming FDA weeks, of wrap particular, the expect number look to filing after updating that. our the patients and up conversations package, follow-up, the of you in

the now that underlying II of AMKD. the the to molecule unprecedented proof-of-concept which reduction results, cause been primary and VX-XXX showed is kidney EU. reduction II/III endpoint have or U.S. in or trial Therapy single Breakthrough approximately years. designation the the The in who of APOLX the targets of in placebo-controlled a inhibitor Priority or AMKD. treated Phase received a Turning of trial, and in APOLX-mediated small randomized, for pivotal Phase PRIME is patients proteinuria, is inaxaplin inaxaplin, a X disease, on kidney rate XX.X% function Inaxaplin Based in Medicines, decline the adaptive in designation

to U.S. late serve preplanned a trial basis is interim XX us enrollment all for We approval accelerated treatment. ongoing. is in the will designed study weeks seek patient as Importantly, at screening are the the positive, inaxaplin activation, the analysis have the March, of pivotal If it and site interim initiated analysis to in

VX-XXX, NaVX.X inhibitor. first-in-class pain. to And Moving a novel

and ratio in acute, pain. II. VX-XXX high have very benefit/risk across both NaVX.X is demonstrated this from And validated neuropathic We musculoskeletal expectations pharmacologically Phase and profile a genetically target because program a desirable

program significant relief meaningful a commercial VX-XXX of near-term year, of opportunity. which X results pain this recap, in shared To studies clinically the proof-of-concept we positive and represents Additionally, Phase demonstrated II from acute VX-XXX, earlier efficacious these need pain, post-abdominoplasty on results, one and Therapy received the has and significant highly Breakthrough post-bunionectomy for setting the well-tolerated non-opioid highlighting the VX-XXX in the one medicines. setting. in designation, Based statistically pain

trials X III treatment very will shortened are in II. and acute completed II days evaluate abdominoplasty. post-bunionectomy our postsurgical we These end in approximately program X exact VX-XXX We of to explored pain. trials same VX-XXX FDA Phase randomized The are track include program the settings acute of the VX-XXX pleased pain placebo-controlled by The A with will that have other studies XX duration, to remain of Phase for and evaluate pivotal agreement Both pivotal meeting on reach followed up acute severe single-arm initiate across Phase an pain. include Phase to days treatment year. and We arm. the of follow-up. safety III on types the moderate development effectiveness of this will multiple the program dosing study opioid by to third XX days also with end of safety these will of

towards support proof-of-concept our the study In have dose-ranging initiating studies pain VX-XXX year. II Phase to neuropathic in end of preclinical addition, of this completed a we

insulin. the and an the of data hemoglobin exogenous insulin. of glucose improvements on remained glucose-responsive innovative exogenous B. at that Time To showed These more baseline the who with next first time screen of the X alone complications. and the June, XX.X% word and in proof-of-concept and featured baseline concurrent Patient in and previously clinic. because timing glucose open insulin-independent. dose. patients reductions in therapies. In results to at X hemoglobin at case preclinical half gives last AXC measurements. versus a Turning micro a secretion, the day a shared diabetes. XXX patients than patient, macrovascular in with timing were achieved enroll developing AXC continues in XX.X% clinical with range VX-XXX at programs and Part We've has granular were XXX in with the and type United first that in achieved reduction in Both enrollment our Patient data is at States range in X versus from at of XX.X% XX.X% of with of risk The XX% we remained included important X range elimination A presentation glucose data the Scientific are it The day close, range the development achieved rapidly that comprehensive we is presented a oral now Canada late Part in trial, approaching wave which as targeted month, or, the treated resumed new to much are correlated and Sessions. insulin ADA ADA

X our type CFTR device file mRNA our AATD to diabetes and this INDs program, in expect molecules, the program cells year. next-generation For we and

DMD for in and is program for hand IND-enabling we IND plan in it With that, this editing asset I'll overview. gene a XXXX. in to Stuart over studies, to commercial our file Lastly, the

Stuart Arbuckle

pleased review most and with our the the towards in and some advanced start commercial Reshma. opportunity for CF. performance growth Thanks, areas future to CF, the our continued tonight I'm strong in pipeline. of path plans disease I'll

the in growth both them of younger new and impressive In in XX remain TRIKAFTA to with X continued of ages launch and U.S., continue the rapid performance seen group, Outside persistence XX. to Italy. focus to U.S., patients, we we CF being the its Our multiple the KAFTRIO France, quarter, rapid with European this compliance U.S. agreements, patients in we and most business uptake X the in pace add age Spain have to now high and internationally. across notably recently all very children patient across reimbursement reached turned countries our groups. KAFTRIO of Europe of where We've

therefore, start. yet age curve. the were we patients One, We patients younger who and CFTR to will These modulator yet through TRIKAFTA benefit we are therapy. Australia the where X North than therapy, into and of XX,XXX not launches yet patients America, in from fell initiated both are ongoing Europe X off Canada where year a Australia and categories. three, who but the in expansions. recently of addressed in reimbursed and a in launch had Additionally, groups be in reimbursed not largely primarily geographies began who with patients more Two, on are could early not strong label countries

majority time. confident over We ability reach of patients vast in are these the our to

reimbursements new in securing good We half launching headway first and have our XXXX. the of made medicines

progress we patients. expanding Additionally, important make access continue younger to in to

on in X with to For no of results new completed signals. sweat lung positive efficacy old and safety X clearance instance, study years we patients chloride the the and endpoints of TRIKAFTA index

present are year. submit meeting later and filings We to these data on regulatory of a this year global track the end to by expect at medical the

European date PDUFA for the XX filings this pleased September regulatory a with of XX% The risk in death in present and specifically were submitted patients data and TRIKAFTA, filing to months an with is for we less Cystic lung TRIKAFTA lower outcomes reduced long-term XX% U.S. lung patients risk a conference We at underscore Additionally, of of XX months on the data XX% These to Fibrosis and Europe June. improved for lower pulmonary X. the function, ORKAMBI than compelling exacerbations, age. transplant risk Society's of in CF. real-world

CFTR high launch Finally, I'd Outside for of of the and first-in-class these Each clinical activities. Today, market exa-cel. from a modulator. we patients need make or have therapy a our is on the estimate thus opportunity. developing pipeline each in programs, over best-in-class made CF, late-stage benefit and details our a stage do On like a strides and to as unmet cannot not approach, pipeline preparation our who treat these some Australia. few of CFTR Europe X,XXX and there advanced Reshma our to any XX programs provided North VX-XXX, inaxaplin patients quarterly X we our mentioned, exa-cel, past are Beginning protein our activities. of program, highlight serves with last impressive potential multibillion-dollar with and approximately represents very months. we America, are CFTR most clinical calls, a CF, We mRNA pre-commercial of in and

thinking and severe patients cell are our we who thalassemia how the market approach. recap There and EU. have about briefly the U.S. in XX,XXX are transfusion-dependent or approximately size I'll So beta disease, sickle

treat XX,XXX patients patients Our of patients. initial them with vast small the XX,XXX majority excellence of in whom disease severe of the U.S. of number cell sickle and are living the focus on majority in these will disease, U.S. A launch the centers severe with of vast with Europe will these

about that XX% than and reside suggests research in XX% patients XX countries. U.S. of Our patients of in X in more reside Europe states,

countries. and their potential have of all We in treatment referral centers the networks identified these

ready be we'll that to confident launch are exa-cel We following approval.

developing active We liaisons, and support they the service already science teams, we reimbursement are in teams, patients launch And treatment patient have and including the finally, are field. the throughout affairs and medical journey. hired to medical access programs robust

inaxaplin, be U.S. approximately U.S. pivotal talked and We've about VX-XXX, on patients living AMKD. of a Moving over patients, which previously development or AMKD to estimate to of which number in for the XXX,XXX with is Europe, in in the XX% we with them

on diagnosis campaigns are patients and and without increase awareness and existing AMKD. options, awareness is with targeted with disease defined kidney Awareness, is communities, support touch work health newly to of workshops to this medical testing with of To and genotyping within minority the organizations genetic raise seminars. of we scientific I'll the community therapies. patient Here, disease all of education AMKD working are we a AMKD given sponsored the which and are low and not surprising, undertaking

the work and materials Sponsoring Summits, APOLX campaign, for at will examples engagement this American about NephCure's year educating Health and people Health are Initiative. Black through digital Equity Fairs educational and Some this Health patients And education launch Kidney AMKD we to also Funds Matters provide tools of and include: which providers.

awareness In policy education we diagnosis. addition these and efforts, to AMKD also important on support are others and disease to working initiatives

of introduced New advocating are advocating the kidney National Act the entitled color. Foundation Kidney disease role Era NIH, Kidney we would of disease outcomes federal at kidney genetic investigate other rare research Preventing center the stakeholders also of establish in well as we're kidney the Specifically, a disease legislation address for End-Stage Disease that And as health screening screening. in communities alongside improving increase to spring in disparities kidney and

the huge news availability difference force patients active that AMKD access preventive screening to seen recommended, You screening at under list have which services screening to would added of cost, the chronic access kidney in services recently task U.S. If improving may would a in to for the consideration. have no have they disease has make patients. and

testing Additionally, testing patients. genetic more accessible with diagnostic we exploring are pathways make to to companies

Finally, VX-XXX in pain.

Today, though pain a acute of of will us visits the Consistent includes years. a or in Given acute, I'd to treatment this different by days In to our given on $X is over are going Today, pain. and safe per of including inhibitors a acute U.S., a pain, market addictive generic. of pain market is effective, even focus branded opportunity. a development Considering need for we on a types treatment partial a and the price NaVX.X are of pain prescribing. billion driven of as there multibillion-dollar potential safe share pain, a give by utility you medicines FDA captures a that We see XX roughly even roughly related year, management in treatment introduced organization with there program large no business the treatment the for approximately recently This pain concentration allow acute of pain well-tolerated announced hospital opportunity. outpatient with approximately neuropathic and oral market, prescriptions the musculoskeletal. billion to proportion past prescribing. like pivotal medicine strategy, medication of summary those that X/X following of new each and XX% have are the our pain been and patient's specialty commercial day, reach X I'm novel driven medicines effective hospital There for X.X agreement real inpatient billion $XX typical for market, pain without acute represents discharge. is for the small pain and

initiation commercializing globe, go-to-market relatively the severity in magnitude and pain nonopioid in excited bringing VX-XXX plans crisis we later to around to of of with patients. out therapies closing, opioid patients urgency Phase working for be even this U.S., and our the of bring In CF the the and the I'm studies the duration medicine more also given Importantly, novel year, this transformative outside teams future. about of to the short potentially TRIKAFTA III multiple the the ongoing and will build trials, to

over it turn to Charlie. I'll Now,

Charles Wagner

second performance. XXXX, Thanks, Stuart. In strong to the Vertex deliver financial continued quarter of

Second of to XXXX. billion, compared increase revenues the an of were $X.X quarter quarter product XX% second

driven currency Our hedging execution several compared strong number to TRIKAFTA/KAFTRIO growth, continued prior by over exchange year, the again reimbursements from the approvals had patients resulting therapy an growth offset program, was impact last impact in well as of movements. year a on with market foreign launches. increased for and our from of Movements helps small active reported only because primarily on which as

offset CF, billion expenses of $XXX for in SG&A, pipeline, compared the higher Therapeutics. primarily $XXX combined exa-cel. X non-GAAP XXXX with and from advancing collaboration expenses for was launches the pain $X.X million pre-commercial in year-over-year our related activities CF were of acquired million IPR&D as CRISPR quarter quarter decline diabetes resulting type The for R&D was partially amended the the Our second second This to quarter effect second to XXXX. agreement expenses in well and as by especially and payment

revenue a strong to billion $X.XX payment model our This million QX operating was to non-GAAP combined operating in XX% resulted comparison strong XXXX, of continued growth the the efficient XXXX. operating second $XX in second the Our of the margin driven revenue and quarter quarter and compared growth, primarily to non-GAAP income included which year-over-year of CRISPR. product $XXX by million of with increase

and balance effective was remains XX%. $X.X Our the ended strong. second for in profile tax with billion XXXX very quarter of investments, our cash sheet non-GAAP We rate the quarter and

in our million subject and bringing the announced X will development certain curative with cash innovation tools, the the with Hart-Scott-Rodino on including including deals multiple of $XXX millions business waiting for transaction the we've of conditions, people goal accelerate therapy recently highlighted, that Reshma diabetes. to technologies Viacyte front, As acquisition external will bring of This to us assets of expiration period. type closing

to Now guidance.

well guidance $X.X a uptake of where TRIKAFTA/KAFTRIO with approximately as midpoint. rates. growth geographies U.S. new seen have billion revenue to reimbursement current continued $X.X XXXX in represents billion guidance increase we reflects product launches The at recently the on our XX% in are updated We exchange as raising rapid we range the of for secured performance our product to revenue based the Year-over-year,

Now OpEx. to

multiple we deliver financial and both out could well mid- profile, strategy With growth. programs as designed disproportionate advancing that investing rapidly expect strategy our with innovation to which in and strong development, we significant in our are to was fits innovation R&D with pipeline strategy each continue future as with now success Our of seeing R&D that drive and internal play existing our portfolio. our complements external late-stage

resulting expenses particularly billion. is the result, for upfront increase billion, a as now our trials, $X $X.X due a incremental The to AMKD pipeline of billion primarily of previous billion pain in payments. from As to advancement of are programs increase from into projected milestone $X.XX additional to to year the and non-GAAP range expenses clinical an late-stage operating well be our in guidance and $X.XX as

non-GAAP we tax rate to the XX%. project effective continue in Finally, a XX% range of to

As half this look are of we the on progress, we which number slide. of milestones second forward into XXXX XXXX, our and to important to outlined continued a mark consider early of several

delivering built of earlier discussed durable at development business disease in years We're to with set multiple and we've strong the to the double-digit clinic. and the We're Reshma growth, X on eighth come. also inflection CF long-term Vertex well in and to for diversifying adding our As growth leadership a potential consecutive way remarkably in medicines year a late-stage cash flow long-term to now our with new new call, enter in areas our is business revenue point.

also the programs of these cash a with our high-value opportunity. and CF, each of profitability developing represents multibillion-dollar continue which medicines, invest flow. transformative, are creating exceptional while in Fueled to can pipeline success our in by We intent we advancing delivering

throughout you of balance the our updating look the further progress As always, year. we on to forward

Let's now to call open the questions.

Operator

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

Salveen Richter

here. quarter Nice

sickle to what question duration Just what curious what number a with X bluebird? your does of the looking cell want And to out proportion cell, pipeline to there. at for they FDA and we with patients? here, where saw program regard you to years Just stand beta a with regard similar are

Reshma Kewalramani

these exa-cel the the Yes. Salveen, With it us regard conclusion looking and in FDA duration number of of for, reach patients for on down is the areas X what to to follow-up. this file is program and Reshma. is the to the

productive conversations the coming we X the and and these package, of -- for the terms have over are preclinical And we in It and in about of our are last with is many other the weeks. are very CMC conversations We wrap the areas. we been to months, done conversations these having all them in up -- manufacturing conversations, modules. expecting

number MHRA So and We'll to just you -- of we and have they'd like really on, update the that. don't that reiterate those are the able come talking speculate. will be we it patients in that these on But follow-up. to just we've have conclusion is down of EMA points; after been see the to and file similar X we've that. to duration topics to I to

Operator

will The next Jefferies. come Michael question Yee with from

Michael Yee

I on, guess, question inaxaplin. A

is You terms Phase to II Phase and you the year? I are on to going challenging end it in think talk a the how how III you've rolls about good is because and you it the whether portion, a comment of population Phase think insight a the III. portion II would then would of enrollment, Could necessarily I know enrolling that Phase into into data enroll. at give the of pace about

long how dynamic? that just with out about So help push guess, little Does And a that year? follow-up by out things a bit on does I exa-cel. then, the timing Salveen's out? a a X-year to that. it talk question that If is us push maybe requirement, Or

Reshma Kewalramani

about in Yes. exa-cel. Michael, and about there. AMKD questions were one two One there

Let me just out close on exa-cel first.

weeks. up the going coming are meetings these to the FDA discussions have we discussing, to were just wrap we our As in with

to the on And specifics speculate. the the is enrollment going a you timeline to need in point. on to patient that regard near population the AMKD, able We'll make about based not you and With give point you really we're the good a be future. so update difficulty to

the about disease. and to protein, CFTR disease, many with similarities by treating AMKD is our underlying XXX,XXX the the While population driven of It's CF, same thing the AMKD. awareness We're Phase is protein. difference with we've APOLX recognizing inserted this initiatives targeting program. the big disease genotyping. X the and cause approach diagnosis, in shares patients, CF Where into II/III genetically this, targeting But in comes

The that first them. XXX globally to is sites over are, patients to many we're opening simply sites the so where close we have and of

sites say enrolled everything the II/III raise Second, as groups we're then is part be is phase the will discuss, study the we a who and now we as That's APOLX Michael, the do all working with of III groups with of controlled right study. sites, upfront. the eligible the you the awareness. in physician are Phase observational ongoing. an be genotyping trial trial to I Stuart they're alleles as being trial. lastly, will And have up randomized a heard opened II/III patient That beauty and the could that point People X Phase to study that community genotype that and study. -- out, in whole Phase

Michael Yee

you the move II? say to giving data Phase Will anything, forward? So just on you're or committed

Reshma Kewalramani

Yes.

selected having had Phase on you expect the to us. from Sorry about a hear II -- of our we on data. the point dose question should the are part, you When at

Operator

Phil with from Cowen. will come next Nadeau The question

Unidentified Analyst

the how This now Maybe population. patients the enrolled is could Phil. [indiscernible] II/III just on Potentially, potential Phase thinking targeting you baseline on in give that progress. on XXX. on you're update for us an the AMKD for characteristics study Congrats the it's broader about

about thinking you're clinical sector the in how the potential differences and APOLX across [indiscernible]. looking eGFR curious Just proteinuria of

Reshma Kewalramani

Yes.

really this and how the of I in think about think the who the entry are their terms characteristics patients key II/III the we thinking enroll in criteria. we AMKD population? would baseline the unifying what reground would for study do question criteria To Phase are threefold is be

The first is X alleles. APOLX

than a is second that's the renal X.X The reduced And proteinuria greater third grams. function. is

study will this those in patients key all criteria. So have

out, of population renal to But well Now and you this that as a the range the point is reduced important X be we have a going rather to function. terms APOLX expect diversity of of as of fully patients kidney really course, spectrum proteinuria homogeneous as and the function. of there's alleles, disease, point proteinuria is in

Unidentified Analyst

And release very That's data helpful. enrolling? the there the Just you'll observational is et any I study that mentioned that to may, data, if Great. cetera? get chance from you you're that genotyping

Reshma Kewalramani

it's very observational because it Yes, specifics the already that I'm share IR the hundreds sure are study team and patients we is That teams enrolled. have great a sure screened that of planning the well to data. into a I'm simple question. genotyping can study is people is that give you offline.

Operator

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

Liisa Bayko

you're in give that. on follow you're looking for? What be you XXX benchmarks specifically, to the and was could able the us any what XXX looking I that SKYLINE of meaningfulness and for sense wondering clinical to are study if

Reshma Kewalramani

Liisa. thing, Sure Yes.

now XXX/XXX which XX-year-olds, and X- SKYLINE study of also the -- So is is in to study, ongoing. studies the the X which tezacaftor the SKYLINE RIDGELINE

Here's but you based which clinic. data know piece our not for what we're is of we that on in quantitatively looking what HBE every we the have, see assays, predictive only including qualitatively of

modulators readout, And Phase end been at on HBEs, see PD looking the and, we II here ppFEVX, preclinically Phase We the of where of real is to the are direct what day, of expect tez II even safety in best Phase That's the sweat the if really CFTR we that greater we studies. the about And sweat readout, potential in potential studies TRIKAFTA. And be greater what sweat at even of when has see and most TRIKAFTA. clinically chloride course, than the Phase those you I will. at is XXX/XXX our for be the that, the chloride expect we that levels talk TRIKAFTA. I has XXX/XXX function profile. than From ppFEVX, sweat expect II the looked we I and to studies readout remember, to Based looked chloride. that chloride to tez has and we consistently superior described, superior on

Operator

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

Geoffrey Meacham

good program, I your the start year? of the on a size thinking if could about consider scope kind quarter. meetings like pretty opportunity to looks III at wasn't wanted pivotal ask had be of second pre-Phase you'll Congrats FDA, a you've When but I to perspective on with what I And wanted the that what success get the then on the market and how of XXX. broad high level. just sure you're half and the you

Reshma Kewalramani

thing. sure Reshma. Geoff, is This

start, where program it is near in me focus it musculoskeletal terms The enthusiasm such the to it, is the opportunity. has other talked I -- across and the have really acute over in for call and we most pain, is talk for genetic And advanced high the I'll it, term. and although let's neuroplastic of potential have is turn programs market I've one for I for about the pharmacologic have high This of but expectations very validation, the we to about it. are that reason that is Let pain. already Stuart reason then is the the reason market this pain, excitement opportunity now for program this acute right and, pain last is,

and program. program. reached agreement the our this end Let pivotal II FDA, Phase And of completed have we meeting have the on with me we describe yes,

X,XXX-or-so It's all one a example, exactly the about well that trial, pain. in so as we beauty third a II the one in or tissue abdominoplasty, designed moderate-to-severe pain have is Phase program. trials procedure-related and for the sprain I'm in randomized that turn for a So patients of for here's that program. program potential. you injury. and takes so acute single-arm study indication controlled Stuart, a like types, fracture That's the bunionectomy X of the as, this treatment really or over pain going And market broad to to soft it look

Stuart Arbuckle

Yes, Geoff.

acute we because our broad excited be is why could agreement about the pain a studies if market positive, it of we're the which moderate-to-severe will is so the And to and large. confidence, indication they incredibly with are lead have high level the so FDA

where remarks, alone. So the billion as year of about treatment X.X with those I in billion or a after said treatment their hospital a inpatient year patients outpatient hospital in are discharged the visit days influenced B days result in as initiated were or a pain. treatment X/X it's X they by my either are And for over given being U.S. either prepared of

vast So in it's that currently of alone U.S. market X.X today. the billion treatment know, the days, genericized. majority a But so, billion is you even $X as market

at a pain know and And multibillion-dollar profile, a great around we very that day. get should to is a able market of significant market oral so a opportunity. tolerability share safety $XX also a if to that medicine bring we then decent be branded highly price we pain has expect that That effective med and of to a

both And so enthusiasm that's Reshma why this a and high of have level I program. for

Operator

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

Evan Seigerman

ahead therapy and color you need to XXX really both opioid that, I a take about breakthrough show with the to quarter. following you in. on would of step use Congrats therapy be the perspective I'd color within to opioid back this kind Maybe to how question using on acute goal would love XXX pain. paradigm? on us pain. option but there's envision the some fitting get a think pain of the assume Kind you safety kind management And have of fitting together? And Geoff's to as still provide of for how if up

Stuart Arbuckle

that Stuart it's one. Evan, take Yes. here. I'll

very, managed, currently were to of something is of go pain sort opioids obviously, be then kind meds, it maybe people if would very else. then and to then to acute pain nonsteroidal, of kind taking might transition go way through the people over-the-counter So mild,

they where we're opioids. here essentially sort with would with what And therapy is a realistic proposition fitting this step profile clinical believe that's the approach to of of that would imagining see ourselves VX-XXX date a physicians, of sort between kind discussions NSAs based and medicine on we those in. initial prescription that's where So seen our be we we've and inserting

based on So and of safety profile fitting we the efficacy the seen. that's would we've tolerability treatment how see and into it profile sort paradigm that

Reshma Kewalramani

And opioid Reshma. public to is crisis. is country. what said, health a Evan, Stuart there a in epidemic it this is this add To raging

which a the think So CDC is over last in recent this the report XX% indicated XX,XXX most year, I deaths year. increase past

are and issue. enormous Stuart crisis. is we've care and addictive is And there enthusiasm and here, potential has community there think then that So current that There officials this this receptors. think, VX-XXX, mechanism. approach potential. groups not periphery, like a to spoken I pain physicians for historic huge over-the-counter day from And It's works of is health with, there that this described which drug kind the a in is medicines from it no no I a where central step a only because

Operator

The Colin with next will UBS. question Bristow come from

Colin Bristow

quarter. the on Congrats

-- So program. there's just the us of walk overlap. Could through VX-XXX Viacyte program on obviously, the just acquisition, areas you

how the here? so and about When some maybe before program it it for And we it short little to about bit year-end the then on trials? out sounds a position -- out And Just a VX-XXX, those endpoint. of a size, should and like you think population flesh prioritization initiation trial you'd just overall? how think acknowledging augments [indiscernible] more primary for in large it's but do be you topline is that just

Reshma Kewalramani

will done it understood very the get duration. We've procedures, And to efficient studies and expect VX-XXX. that here the let's we to cells device in transform, exactly is The to The if for. is short VX-XXX type these progress next It's a naked me program, RCTs, are quite Yes. X edit and us those the programs goal, an diabetes then them program aiming have tackle in hypoimmune to programs. and Viacyte we first, diabetes and program. randomized X third them VX-XXX. These Viacyte clinical let program we And that them. that certainly regard real quickly. cells to type are internally program. With did with XXX just X are is the know And same is aimed programs the as cells. this cell programs do well acquisition. We really exactly the call I goal X trials, the call again same which we program. at XXX what VX-XXX, That abdominoplasty. the it with evade question pivotal the the in disease. Colin, how for the quite The cure, are I'll We development system. surgical immune bunionectomy not

to of at goal are and we're What dose get our which Viacyte with the you the dosed type on can X this there. first doing well We is ability accelerating way of patients acquisition achieving VX-XXX. X diabetes, the value half see transforming from our to

cells hypoimmune in with we and to ones and CF, late-stage And with choose our here this take to goal I'm Viacyte manufacturing talent Specifically, particular do multiple programs expect a what and best about Viacyte as move one is clinical-stage then us development to GMP to GMP double-click manufacturing, access and really in to the program parallel I collaboration. are accelerate cell our technologies, did tools via ability brings and what -- that, cure. the CRISPR in IP capabilities If get that's talking we on going to lines, or the commercialization.

Operator

next SVB The come from Securities. question with Risinger will David

David Risinger

your not real of case I in weeks. be XX well world. of arm's you have care you have of that standard think, period the patients, So That's short at of a of Could expectations care, eGFR the arm couple will control taken on the questions a time and the to. the interim always spoken rate discuss AMKD control about which decline for I the is

that what be data? of interim expecting positive then the early assuming would the that you be the decline. And an it suggested on control do understand succeed you're has would filing arm's interim, FDA just helpful in handing supportive for it So to on the

Reshma Kewalramani

we and that end II analysis. the were based an interim that supported is agency With very that one of This reason the Unambiguously, the on. we one yes. reached of regard thing. had Sure at Yes. agreement which points the of meeting, the our interim we of on the to approval is of The accelerated analysis, the around Phase have north ball those other words, declines is question APOLX-mediated kidney APOLX-mediated alleles ensure That for CCs annually conclusion treatments. no who a driving important to have no is about is a in specific than they following: AMKD, certainly non-APOLX-mediated are as Unfortunately, disease year. and of or In different APOLX disease. disease. rate is kidney whole very X kidney FSGS talking We're treatments X of function. there targeted for are aggressive significant of wax a very reduction per points

this So I about continuing. are medicines, that standard is of even care though patients decline often on talked

to playing the in renal is progression opportunity it those slower. is patients make with X be of That's interim are treatment, so longer disease. not after at fit will to standard these fast, rate proteinuria the kidney able and it VX-XXX the because usually decline takes And AMKD. weeks of So non-APOLX-mediated just year, always to have we offers where which the the so treated the as it as why of case is Very, this designed different between assessment in very well versus program, with because the this we regard analysis decline XX care assess our function. difference have in to

Operator

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

Mohit Bansal

Charlie. have question a and Congrats on a I clarification the quarter. for

in at million situation a one. including investing couple are you're IPR&D that into so are profile heavily which that margin R&D R&D. acquisition $XXX of of this sustainable So given you years, guidance? forward point this pivotal I like question how a mean SG&A phase. many for good And the looks having is in going are mid-XX% is programs operating is, of the clarification entering Keeping mind, next Number in in combined, the -- your for and for we it Viacyte

Charles Wagner

Sure. Good question.

pipeline success had that the really recently As mentioned, we is unprecedented. in the we've

what quarters recent that is in data multibillion-dollar opportunities, not the we And our these programs, and and OpEx investing with drives surprisingly, great guidance. are the several so really in of increase success behind and the

of Specifically pain as the and in XX-plus AMKD And the highlight the biggest in the guidance drivers the success the OpEx importantly, increase, of percent R&D. increase is of the in change guidance. I all in would

to very, certainly and it's very we'll invest. So perspective, continue from good news our

for medicines develop have transformative margins, unlocked the serious there's fantastic allows innovation. of to to there, we model. us tremendous diseases, a consistently terms you ability in which reinvest When value In sustain

we And margins the for can attractive that operating future. model, believe sustain we very, so foreseeable with this very

in Your not transaction can't in the around in not accurately That Hart-Scott-Rodino the yet the included the we update Viacyte. know and waiting the close are And is for transaction. as when period we'll a specific is we question That accordingly. date, guidance. guidance forecast date close

Operator

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

Olivia Brayer

to submission there up and on wanted conversations different patients know of agreed on follow-up in but be that the upon metrics with follow any are great packages Congrats quarter. CTXXXX. then what beyond versus to in have MHRA the might EMA the U.K. filings. you're And duration quick question the FDA, could I of clarification still press I between and number a call specifically mentioned by out in to U.S. doesn't but the the in year-end file on on you you're it track Europe you It just release, file looks U.S. the like and guys

there. assumptions to clarify to to So see any if there's I just your wanted change

Reshma Kewalramani

Yes. Sure thing.

us because with allow really them. have side and We and have other have been we're regulators frequent designation conversations conversations, every the having this on pond that we to by of the fortunate offered

manufacturing with these was and we duration so The the same evolved EMA months. MHRA, over filing having conversations we're EMA and we've settled number were concluding the have in towards CMC follow-up. the all and And for those and many The with topic been time, out around modules. with out the conversation because of point we've settled as that package. FDA. as on patients end of discussions, And preclinical of conversations FDA We've MHRA and of other the on

to We have come conclusion.

we the coming the year. I follow-up. of FDA, get we of filing And after look MHRA simply concluded the number forward that. you of and and regard that We say will yet with We I duration With and patients have haven't so hit weeks, expect fully updating EMA. that on do are we discussions agreement. milestone the this towards few on do I in we our to the to have we end our and that haven't can expect in simply reached to track, therefore,

Operator

Oppenheimer. Hartaj Singh from The will come with question next

Hartaj Singh

you're into years but A really looks a about molecules about potency molecules of at year nice couple from bit memory these higher talk you little to had quarter. get concentrations Not or Reshma, the two and going the interest. that of to you're them increase ago, this of do a tissue AATD previously, bringing And to mentioned be clinic year. more to to trying -- like a the

the you molecules So then that about assuming approach how the with readout are going see this you could thinking clinic for And clinic year? in these get them we forward? year, the in this when a

Reshma Kewalramani

I'm and a are about to AATD do high the X many molecules wave. expect of next the glove. than molecules, disease to this for in of enter there remains more I but clinic, AATD the IND the in this least year, program, Vertex wave interest, excited at go to strategy a X like one the regard With that and that are next fits

I We next have have explore dial been that expect that dose I fully The can we results And expect year. doses low. the that going able we'll are to about the we're by range. potency. to talk up

liver lung progressing and molecule the manifestations approach opportunity to others, So the programs That's to are and this disease. remind the here, Hartaj, of just is small is tackle both the why transformative. not don't mind, inviting in All simply so my out eager are approaches tackle so to it. And we're us, and both to there manifestations why the pursue other of disease. therefore,

all And for. looking we're and next year, evaluate. what can really we have we at that's look results should that So

Operator

and will for come time question, Abrahams RBC Capital that one with more Brian Markets. from have We

Brian Abrahams

respect approach curious you you a might I you supply? stand help that long-term where and manufacturing the to helpful filings, for I'm where you see with study. know do that posted CTXXXX and manufacturing how be to ultimately recently you bridging scale would I'm with relatedly, the that much field then how As expand up? regimens? opportunity? conditioning And potentially or And next-generation respect expanding curious

Reshma Kewalramani

you I'll to long busulfan Yes. come bit and comment ask Stuart and on with the about in opportunity, going opportunity back the a tell little near-term manufacturing. term, I'm the to

Stuart Arbuckle

Yes.

for thanks question. Brian, So the

there's cell who XXX,XXX patients U.S. sickle sickle or across and and disease thalassemia. the across beta So EU, the TDT, have approximately cell we think

treatment to and going that in to XX,XXX around the are consider more given is those are launch those population busulfan are probably have being the clinical through is patients the that that trial, disease patients similar those of sickle initial to about Our in severe those that cell going disease, regimen. of We included States. likely majority are XX,XXX current the the think patients, this focus conditioning United on

launch population. So that's the initial

procedure outpatient this regimen, the what opportunity look But technically. on terms if gentler of will kind procedure. XXX,XXX much turn expand there, of to in may opportunity, certainly get truly well think of of It the that not Maybe number but we way significantly significantly would it a population. the like a can would Reshma beyond towards an conditioning having. higher terms we could more that comment significantly. a would In it it XX,XXX which consider all become people the into expand

Reshma Kewalramani

And guide And I manufacturing manufacturing, And say CRISPR-CasX, that with regard challenge the gene we're things, diseases of an CasX. is with tackled in example. is to because the in and what a RNA being are that about talking this vivo essence, grand than editing. scheme easier this is for other ex

in in we commercial sites space. the fundamentally the it's trial is that exact well. the process as that CRISPR, actually the point And space, that I be will thought I here of that terms make we've process at commercial to mean in manufacturing the of about sites. the same It's of clinical are development. actual manufacturing partners mean second same terms get-go. from in The manufacturing our therapy and to the this -- also using we credit that is the what using

CTXXXX. grand it scheme things, feel commercial gene good it. RNA, So ex where and it's editing, of of is about and the the we really said, in as I guide easier that's because manufacturing and an vivo are it's challenge CasX with we And

Charles Wagner

I Yes, QX to you, call was into everyone, for thank to just tonight. tuning the say going

are Investor have questions, who reach Good please this you evening. additional available team If Relations out in office the to the night.

Operator

now today's The conference Thank presentation. has you concluded. attending for

disconnect. now may You

Vertex Pharmaceuticals (VRTX) 5 Aug 222022 Q2 Earnings call transcript