VRTX - Earnings call transcript Q1 2022 | Vertex Pharmaceuticals Incorporated

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Michael Partridge

Good evening. This is Michael Partridge. Welcome to Vertex's First Quarter 2022 Financial Results Conference Call. On tonight's call, making prepared remarks, we have Dr. Reshma Kewalramani, Vertex's CEO and President; Stuart Arbuckle, Chief Operating Officer; and Charlie Wagner, Chief Financial Officer; Dr. Vertex Genetic of and Chief Sanna, Cell Therapies and Dr. Bastiano join Chief Scientific Altshuler, for Officer, David will Q&A. that slides to as recommend you We you listen this access call. the webcast on a This will call recorded, and is be being available our website. replay

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

Reshma Kewalramani

Michael. Thanks,

be Before call will we his last I'd Vertex. Michael and recognize moment outstanding begin, us, to for service quarterly contributions with as take to to a this Michael Partridge's like

years, we Vertex part the the of and shown mid- with patients serve. late-stage and countless has he evolutions the true of has analysts team, Michael his of launch our company. broad, our led of recently, an of the our approach, Vertex team and been investors for cystic a many fibrosis medicines and integral emergence For XX with the and calm Vertex IR Michael and marketed our pipeline, Michael helping the the world. through been has milestones steady has and through face passion story with More of all leadership four share

development. sheet balance itself of has and This to molecules The enables billion and cash XX number genetic X serial strategy beyond the R&D a $X.X innovation small strategy. and of with disease have CF is development CF proving in our of potential discovery strong it and and therapies investments. been our made exemplar across targets company. months odds clear, our of transform, cell last the diseases R&D patients and areas. increase cure, the of discovery in to way biomarkers validated human causal drive more drug help so through bedside doing the and and long-term in translate number billion, the continued to quarterly not of we goal for progress with to is grounded want and CF the that all growth external across Vertex business, And advancement thank board grateful the in post start our quarter of remained significant the with rapid product and review. dedication, the for a the $X.X a now leading is excellence. rapid a the globally. strong year-on-year clinical Vertex. for pace research And growth to revenues CF QX across and grew are the with finished stage. operational performance CF all personally On maintained to off excellent margins pipeline non-GAAP I reflecting and XX% internal industry investment a We operating to his in of at an We treated innovation, he's done for strategy if pivotal bench success biology, half to that patients from development the Michael the dozen XX%. the multiple stage continued and continued pipeline POC in despite in Fundamentally, programs and in

disease, in clinical the X dollar disease is diabetes, and CF, potential IND more track type next year program growth in diabetes, pain have now in rapid we've The with APOLX-mediated cell data multibillion new editing in mid- in in the the in the self is kidney programs group our correctors the pipeline our opportunities broad of X of type initiated proof positive generated number wave representing thereafter. in and vivo of with next next later terms sickle in of and development, in company programs from in IND-enabling of late-stage and mRNA clinic. wave molecule With our gene diverse advanced. beginning with are therapies broader or in more the small studies continued and advancement areas now clinical CF, with this programs of trials on program been program that these X stage and inflection A has clinical in DMD. our for disease In of AATV plus succession number CF, a of concept the at never of beta-thalassemia, approaching filings modalities this device point the pipeline terms

model us I'll R&D globe. as positions medicines sustained business for highlights for context, combined growth strategy more transformative with the review R&D and innovation patients bring now continued we additional the well With as quarter. work our to to this the around Our

continue term. strengthen for leadership we to our the to in Looking our CF, future long

possible continues to And Stuart do discuss, in raises any who we're bar -- is TRIKAFTA said, with experience accumulate. TRIKAFTA, outperform if so. to real-world determined Our ones as to the That it the regimen for will development. be

and Our tezacaftor of Skyline Phase than enrolling program. VX-XXX, patients pivotal combination More XXX triple XXX clinical sites is through trial next-in-class are III in progressing rapidly development. open our

early to expect by late enrollment or XXXX XXXX. complete We

benefit once-daily than reminder, convenient VX-XXX/tezacaftor/XXX more clinical greater carries is that TRIKAFTA royalty for treatment a and a Vertex. the for obligation As a has potential lower

starting for modulator, completed, cannot do therefore, to on make an developing development clinical remain have in IND CFTR For partner, benefit, track together second protein patients our we who of with therapy XXXX program not with an thereafter. the mRNA IND-enabling submit than the a any more and CFTR half from X,XXX we been are and studies this Moderna.

III disease cure more clinical and Turning launch. to Phase and the data have for and both our cell look sickle to this we beta-thalassemia. medical and patients for sickle U.S. dosed plan complete, for the beta-thalassemia approvals we EU Starting at on pipeline sharing editing and to to year. cell beyond CTXXXX, end across We Enrollment provide studies by than programs. in to longer-term gene with CTXXXX, next more forms including of follow-up for our We now XXXX, both designed forward functional CF. more a is potential CTXXXX and regulatory approach disease XX commercial expect patients this be submit

results. VX-XXX people a kidney proteinuria Moving studies VX-XXX placebo reported included Phase II/III in following reference endpoint of accelerated agreement note, clinically announced APOLX-mediated XX.X, XX.X a XXX,XXX following the of arm II all SPID-XX, on time-weighted relief FDA studies, of FSGS, well VX-XXX mutations, March, the statistically SAEs to it's intensity made and adaptive we tolerated. VX-XXX of tested APOLX-mediated one to in decreased significant VX-XXX, statistically provide assessing that ability population tolerability XX.X difference in sum to to and events SPID-XX difference mild to adverse a following pivotal a respectively. baseline. of in with this the no and study decrease were therapy a our mild time adverse in AMKD molecule the could serious scores placebo significant in expectations. Transitioning patients U.S. opioid generally inhibitor X for of first initiated a program, Phase development. From acute we our X In standard which pain perspective, and with to approximately of which development well high and in pathway one a the related the NaVX.X on abdominoplasty interim abdominoplasty, pivotal and VX-XXX, primary In first-in-class, evaluation pain proteinuria the VX-XXX all VX-XXX, disease, with dose the in bunionectomy, XX.X design Europe now led disease indicate XX meaningful progress AMKD, and conduct bunionectomy. to In analysis, representing was study, in of showed non-opioid pain broad one, a AEs a at In placebo to novel at the March, particular highest superior were II disease; VX-XXX single were late to kidney the our people moderate two, majority SPIDXX, positive, X important kind a has to was an VX-XXX renal meeting the reduction inhibitor In of first-in-class relief. XX.X% and which into mean program. was Phase pain, the rapid, with consistent In APOLX late and and design people small or showed achieved treatment mean higher score, SPIDXX with were of of in December, approval if U.S. care three, There events from compared and or from no of tolerated compared with intensity proof-of-concept hours. severity. of dose function; In the to unprecedented doses. with sustained to the VX-XXX related to safety we and There greater pain rapid moderate.

pain the type type pivotal XXXX, to the for goal diabetes addictive affects people working type discussions VX-XXX, pending that regulators. in forward treatment potential unmet offer side and high diabetes. X stem conclude And Europe of our living potential fully the provide with VX-XXX, X into to of the more medicine, an X.X X opioids. a for diabetes million well-tolerated the program and advance with than could cell-derived In cell the cure class of acute alone, plan adverse effects differentiated pain with second functional people. our with need we urgency efficacious Given pain without pain VX-XXX are non-opioid with relief to a half and novel effective we therapy bring is advance replacement islet or for U.S. to development I'll

patients efficacy this has hold we're shared XXX, U.S. the with insulin that to and urgency As data level VX-XXX day At treated FDA earlier the X time, hemoglobin of first date. also week, patient dose was understand the working To X.X%. we on independence first program safety cells and placed we has with half by a of been in AXc announced from more. the at target achieved clinical with treated the to the the recap,

while in robust The measures pancreatic The patient lipase second decrease cell day and control exogenous experiencing insulin positive patient improved glucose a also a half of XX% had increases results dose use. achieved in through function simultaneously islet XXX.

patients dose, results together, and of at X, half both demonstrate patients Taken concept for proof VX-XXX. from the treated X

is day to dose the receive third a first of VX-XXX has the The full reached XX milestone. patient who

showed As XX, improving patient efficacy, control. of of the indications and levels with glycemic day increasing C-peptide encouraging early

occurs function patients X the SAEs XX dosed day first detailed study the overall study with related and The of no and profile assessment to patients perioperative VX-XXX. The used date, for the regimen program, visit. moderate, is to immunosuppressive pancreatic glycemic events in period. are in in the control the are consistent safety and the at islet date. of the majority Across data adverse to there the are to mild the These

on to out U.S. in device that close summary, protect are type diabetes, of word pivotal Of will understand mid- to with the track to continue working entering instead programs and course, that this later plus their continued all Vertex serve this cell in our X device the starting self be to with resume we To program, expeditiously In program, in we on progress as an using in protocol. patients areas possible per wave X followed for with immune late-stage deliver We programs this remain on rapid this FDA CF, and immunosuppression filing designed study that disease trial We year. device growth development, or and year. already constructively of later on make look the from progress significant we're our the sales to approach. can continues soon enter making including so and forward the programs X the In the function. to development, with system, address questions in a for to track as to be another program immunoprotective clinic IND X is quick

continued profile over it Stuart. turn and serial financial that, investment I'll that strong balance We innovation. With sheet have a to enables drive to

Stuart Arbuckle

the who uptake outside rapid in Thanks, business countries uptake consistent first reimbursed; to performance one, CF strong in commercial grew KAFTRIO where Revenue internationally Reshma. U.S., reimbursed first patients increased two, the and America, billion review younger TRIKAFTA, the the therapy. quarter and the following quarter at over who be are areas. into expansions. a TRIKAFTA the our than year-over-year on in X% and outside label children on progress XX% are product reimbursement benefit by robust CF, We therefore, $X.X three, expansion XXXX driven most billion North were are patients in our initiated patients CFTR Vertex's will of reimbursement $X.XX by I'm ongoing million, primarily over X These by rapid performance our categories: additional Australia, pleased countries approval. XX, fell who clear in more yet of more have plans Europe to ages following groups driven patients U.S. disease treatment recently in XX,XXX KAFTRIO of yet U.S. treatment, to the driven patients notably could CF where to mid-XXXX U.S. but revenue TRIKAFTA, geographies for revenues the reached the of in not to year. pace, past significant we the therapy. additional through age $XXX in with early path grew of grow future modulator of growth continues tonight not yet have agreements. QX come as year from with and curve; launch largely into continued continued towards we we starting a XX% patients TRIKAFTA, and of X not addressed X started where in XXXX

where above. of the example reimbursed in to agreements KAFTRIO now ages confident in vast available is these continued Australia, we reached to eligible the being new majority countries, have than and our XX over TRIKAFTA reimbursed and patients reach TRIKAFTA are notable time. We more are with XX for reach reimbursement most ability patients now

to In to children XX. We to for make Europe younger patients. in also extending in the patients. ages we for And for to approval granted secured continue progress Canada KAFTRIO treatment January, April, authorization sNDA Health U.K. the than recently U.S., submitted for for we ORKAMBI the X X and X an children of marketing years. less for In TRIKAFTA approval same ages

in TRIKAFTA age U.S. the group this We for X also the ages enrollment of children study to X completed XXXX. in for submitting of Phase and anticipate before III approval end

Reshma As further combination. proposition this powerful TRIKAFTA with strengthens clinical and real-world long-term value of mentioned, the experience

over for KALYDECO, decline, lung with We that medicines, ORKAMBI progression We on function function lung that that controls. TRIKAFTA average, over have evidence every show untreated lose any And CF shown of SYMDEKO period. X-year to treatment follow-up to the data lose with of CF previously treated a matched now patients. disease these do for function not patients our on Generally, compares function X% lung and one slows hallmarks data TRIKAFTA lung time patients of X% patients have year.

presenting forum. We will an important data new these medical at be upcoming

been provide that before As for last well programs with disease Starting programs which to would remarkable multiple opportunities. as our regulatory commercial our reached file and Reshma period beta-thalassemia Vertex on a hemoglobinopathies, cell thoughts next underway. XX the few approval this months to to be development. like among have year. noted, for Commercial plan and X a launch I editing activities gene development late-stage late-stage for end CTXXXX, sickle the in are X preparation therapy we CRISPR/CasX-based for of could

of facilitate Over the majority launch and Europe positions small the treatment excellence our in of are treat later of have key the centers multiple place With the sickle and treatment severe cell patient we year, A medical, sickle that a planned rapidly. including physicians U.S. Key and past for year, this and progressing the manufacturing. TDT are them functions, thalassemia the our including will refer vast deep patients. centers concentrated, diseases preparation across where commercial and who of leadership submissions understanding teams in patients and are will developed would with for activities markets, the number these journey. cell

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

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

U.S. are support and commercial we and treatment service in to with developing payers the and programs journey. patients robust throughout We engaging are public the patient already EU,

this CTXXXX We and of continue therapy living and to look cell potential to with sickle to to beta possibility bringing see tremendous forward for thalassemia. we groundbreaking the disease patients, help those

pain with the Reshma completion results now plan POC acute of II positive the the X acute in VX-XXX advance year. development noted, we Phase in and of in recent as both pain Finally, studies into studies, in to the half second pivotal this

with prescribed does the were XXXX, for fact some highly recently in the there than effective to provisional of would XX-month of have opioid National the from on and not Center novel, tremendous ending give these are U.S. a of to side There moderate Let the the with market VX-XXX, for initial effects days me severe for market risk you and some a Health opioids of improved To pain, see It for prior some opioids. profile, deaths every potential. increase overdose class a U.S. for estimated avoiding from including the the XX% with review that an treatment the is period. addictive that briefly U.S., in April we treatment including safety addiction which large the opportunity period the is A vast unmet XX-month concerns in you these, for care medicines problems pain patients billion market to pain. the compared more especially an qualities and our there XX,XXX have need of perspective of therefore latest medicines in benefit X.X target result of more proportion these that CDC's in scripts for an year, opioid. acute in standard acute pain unfortunate contribute is than data Statistics epidemic

on prescribed and last a as NAV to imagine instance, paradigm a resort, therapy a X.X opioid. in a in only one NSAIDs, patient escalated can started which step is For inhibitor,

medicines We of and our over indication continued look pipeline. we moderate about The model calls. this But VX-XXX Charlie. severe pain. pursuing demonstrated a will in are well. pain, patients I showed the market excited these as the fits and Vertex that globally musculoskeletal the the future forward acute we very positive the acute, neuropathic which about VX-XXX to more applicable plans discussing to other promise of our CF late-stage I by progress bringing am our call see first as of market in commercialization is pain, indications and to on turn across to now to types is segment POC for specialty mechanism

Charles Wagner

Thanks, off as R&D pipeline is Stuart. XXXX again financial strong milestones, continued start excellent Vertex we and to an in in first deliver our quarter. to the delivered significant performance

total product revenues compared $X.X quarter XX% increase of to of were quarter billion, first XXXX. the an First

as Our growth following to U.S. patients primarily reimbursement again year, including robust of coming access the well as expanded was new a KAFTRIO driven over continued past geographies the continued therapy year compared in TRIKAFTA of uptake of the internationally, on growth prior by in number

XXXX non-GAAP and million quarter development. QX increase compared and advancing The year-over-year in XXXX. with expenses first mid- in in combined driven Our investments SG&A costs expenses $XXX pipeline to research increased by in $XXX R&D our was programs late-stage million now were and multiple

this asset going investments in first continue forward, reporting from the contingent we with in the anticipation or non-GAAP that filing licensing make practices end payments the connection of by XXXX acquisitions of development our regulatory activities of with of IP. or been in year. and quarter from to exclude, have recently by consistent Additionally, companies, charges and longer pre-commercial third-party Starting CTXXXX no research milestone peer results, upfront we collaborations, adopted in

figures on of new to This in QX were non-GAAP 'XX our and numbers only QX year also reported results basis have the non-GAAP be and updated for impacts prior change not affects We the 'XX presentation. material. with reporting consistent reported

our continued model non-GAAP operating efficient non-GAAP year-over-year. resulted of operating revenue $X.XX Our combined strong XX% growth, of operating billion, in increase QX margin of with and income XX% an

maintain the in short-term and Our profile. with was first of cash balance to for $X.X billion quarter strong a the We non-GAAP sheet rate effective we ended quarter investments XX%. XXXX continue very as tax

guidance. to Now

revenue product We rate. guidance year are non-GAAP XXXX CF full maintaining effective our previously and tax for issued

R&D non-GAAP ago. I We are upfronts adjusting milestones expenses reflect to and change the reporting in our and guidance SG&A for combined moments of described as

range XX% our potential billion. $X.XX are billion, Specifically, guidance $X.X at projected payments now total midpoint, to $X.X billion ongoing growth. a approximately collaborations. the including is remains At for of CF this billion or and $X from $X.XX of year-over-year upfront revenue in increase to expenses Non-GAAP billion milestone existing operating product or a

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

CF to our in we our XXXX, invested inflection number of look XX continued and diversifying demonstrate that the business webcast. through well the progress, CF in medicines the an pipeline for the time, long term. milestones is are point a we important maintain the Slide strong, have for are same into as new consecutive way XXXX. of double-digit Vertex broad going As At will CF and shown pipeline into year to In track company the of potentially these leadership on are we we and out on transformative The our disease remainder advances. have year we summary, now revenue areas beyond to on of growth as of Xth a

us further further while a significant exceptionally forward value time. our We As we reinvestment enables high for in through well leaves external innovation look to which progress and unique profitability you year. creation updating positioned strategy, significant the sustaining as internal result, business over

call Let's open questions. to now the

Operator

with Instructions] And question first Cory the [Operator from ahead. Please Kasimov JPMorgan. will go come

Cory Kasimov

first over appreciated. all all, It's Michael, of very to much thanks say to want obviously years. Just the been help

the nature question is slope your at the proteinuria predictive impacting materially trial time? reduction AMKD. eGFR of for kind in and in confidence based accelerated potential XX-week And of time curves an the for curious the point filing my So about III at I'm Phase of on on that VX-XXX your

Reshma Kewalramani

be of will third the enroll XXX about. asked you XXX,XXX II/III the X the the the broad a design for eligible could Phase is the singular so of patients program, study that drug. And key the that are with it's study feature population, Yes. Cory, the full that study this features to study. a It regard is

have if EGFR, that very at is relationship the tight, which high in We pathway what accelerated a built point. to studied time approval. analysis interim correlation Phase in prespecified And we provides that XX-week between the is is analysis II proteinuria, have the analysis a the of high in is unprecedented interim form reduction II measure confidence very I in AMK renal very reason because in and that of such function. specific an And FSGS. we called proteinuria in saw positive, is that's The XX.X% this with a aggressive the Phase

mentioned that you come XX.X% with proteinuria you reduction translations with terms in important of the was That that end was to II What So features in the we GFR. expect went and agency were them the level why Phase agreed would it do I at is confidence analysis. what the high of that out when these for I meeting. X to with upon interim a so

Operator

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

Jason Zemansky

started if looking see fairly upcoming may. read-throughs with of operating the asset strategically abundance do sound does with have Geoff. quarter. it guess, our then again, rare this you And the This want again, maybe CTXXXX an bit caution I a basically, focus within pain have Is given infrastructure? commercial you the seemingly on potentially to necessary then potential clinical is cell comfortable therapies. Michael. to hold I the in the efficacy Or And with? submission? few thinking to extend of quick for surprise. on congratulations you at negative on XX for are as a kind is any and it safety regulators was fit commercial programs the partnering the diseases, regulators like an establishing package advances? But Congratulations is. Could portfolio, about you're And and seem gene A Or of on the Jason how discuss

Reshma Kewalramani

a there's in questions different few there. Jason,

pleased out Let seen see the CTXXXX, momentum very we've we the pain study. that we've it into and to try with in me On parse CTXXXX. been how

than forward we've As remarks, are really every filing orphan for And We've secured RMAT this enrollment designation we more in available dosed my of tail I the here is said our prepared regulatory the year. and patients prime towards end complete. XX planning Europe. in

going pain pain musculoskeletal and the it give pain or to back Stuart On X more as don't feel me acute as neuropathic pain pain the the same way some see comments, make pain commercialization. pain turn you to or the fully color. I'm validation the something We will about do program, more Vertexian. over Stuart, I and let or being like categorize some this based That with knee pain a pharmacologic own mechanism said, to genetic on and little expect musculoskeletal. color that across such validation, effective to states. our VX-XXX X on I be the

Stuart Arbuckle

we I misconception a is there a we're And Jason, us not define Yes. an a think that as of a orphan rare how company as or ourselves. bit about company. And Reshma described that's

description where we we NAV as or genetically in that with where we whether think perfectly. strategy said, specific understand they're or the pain in fits dare biology, can validated them and focus now to targets, And markets the infrastructure. X.X on human pharmacologically Reshma diseases is research of either specialty access a Our case

accounts for is said, prescriptions commercial The than those in about Reshma that segment opportunity market segments a different the of the $X in enormous. more with is B the chronic generic, musculoskeletal. you XX% neuropathic treatment And acute, various the of As can over U.S. think Acute alone. being acute year pain days the pain paying there's at and $X.X valued billion. billion despite the market

similarly significant the through opportunity, asset forward dollar market in Reshma as liability said, bring very, We able could pain, are without if effect the pain, that medications. side pain is efficacy, And in very we pain opportunity better that's that opportunity. specialty or specialty we of to acute a also infrastructure. opioid has So multibillion other an multibillion-dollar think opioids acute a in and neuropathic service

Operator

from with Karnauskas come question Truist Robyn Next Securities. will

Robyn Karnauskas

just are there's seeking of So you a that characteristics as You've to list quick Phase inclusion maybe you question be? patients work of Could drug indicated CTX-X. what treatment? are? to enrollment a to X patients are likely How do how genotype? you X creating you do color buckets centers, patients AMKD. willing more be on Do of few And been many some and ones, your to have the therapy. would give some who to the start get the actually quick more you've ones. criteria genotypes are XXX,XXX second for more really think might the us or is trial their actually And initial a talking registry challenging might for patients most be III

Reshma Kewalramani

not kidney performed and into color proteinuria, AMKD bring enrollment seek that renal test on into then the test, the actual that function we've it CTXXXX our is disease and thalassemia to study not cell is on APOLX-mediated for I'm the start for is disease. II/III we have fairly our this the will is and The because some trial with genotyping. APOLX ensure genotyping to that a It's entry we beta reason program. features we but that, commonly simple Phase it's we forward can alleles Robyn, I'll you performed. ask reduced clinical patients who X speed. And Recognizing now, some sickle medicine that commonly criteria to provide really for kind the offer of to going key right are AMKD, both by anything blood the patients simple. added and before Stuart have didn't had

with sites study running many the in up and U.S. the it's EU. The first a in is and global

of is are patients care. standard all on second The

have gets study plus to patient the go to is is there the and arm where no This of And study. care. who standard placebo to if on part get arm and where to up concurrent they So of Phase be can, they enroll wish, have we is standard and the genotyping care of genotyped running a other VX-XXX a third, patients study can II/III gets then going X study. in

helpful CTXXXX we that on the over enrollment. it think to I'm see. for going be measures to progress to I those make are and patients turn very you Stuart, going as that you

Stuart Arbuckle

thanks for the Robyn, Yes, question.

the fusion EU. about dependent patients who the sickle in previously, or -- as cell and commented there's transfusion we've So thalassemia XXX,XXX U.S. have disease development,

you going CTXXXX conditioning that can imagine, we for the the to be don't that as population are there However, given regimen. entire is is eligible current think --

the based and We research likely physicians our of majority XX,XXX that's U.S. to be that to with XX,XXX they talking the in types of are other think those criteria which research, the but candidates. potential of the people's XXX,XXX. Of about sickle that our are study, around So here the patients on cell, think about inclusion are also XX,XXX, of

be So BSA-based we the conditioning that are likely potential is CTXXXX think candidates patients to existing that regimen. the with for

Operator

Sachs. will Please The come next Salveen ahead. Goldman Richter go with question from

Salveen Richter

has pleasure Michael, you. with a it been working

be You'll clinical missed on here. Two One questions. CTXXXX. very

just front, has EU? commercial you could On precedent with bluebird, the this by then construct? CRISPR. payer how on the the And is their secondly, much -- program, with difficulty been editing on globally on And details set provide particularly given some in Duchenne the front gene partnership the

Reshma Kewalramani

DMD going know, through program talk about over start me DMD with it I'm Bastiano. CTX. over turn asked in just to going second, Salveen, -- question, is turn I'm it The going to I Salveen, and to I'm to Stuart before, X you've the to one, then to

nice and for really the ask in made Bastiano, color with editing-based a on IND progress give IND-enabling you I this? can have year. bit that next are on program. plan little program, to a studies gene We more is This we

Bastiano Sanna

Sure.

little durable a it's transformational we you deliver near genetics human near dystrophin the line the therapy by on only approach lengths, go gene other or a to potential approach, and various is gene is a bit know, mild disease. are truncated a deliberately based version dystrophin have cure. have deliver micro has because The the editing functional protein, which Gene on they So to than other D&D different dystrophin example, for in that dystrophy, is for approaches with going approaches a like back of to near full-length contexts, used with very all exon-skipping but the believe because -- called which to for as actually what full our chosen a full-length the required therapy actually is patients approach reason. for that the dystrophin be is we

be has really, since this both to of and what for hard worked development us the our that like we close development science. the and opposed given in analytical other happened to on development You to field process the together through process attention technology-wise as and we that pay did things have acquisition Axonics. because we sure then, with technology best really remember that those have purity product came program And

and we So IND to the soon in pleased as file our be XXXX beginning to said, studies clinical thereafter. aim and approach very development we feel very in about optimistic and Vesa IND-enabling

Stuart Arbuckle

what paid with based think the we there's basis Salveen, securing on as license capabilities I U.S. are elsewhere. CF, the team world get the medicine and that given which payers, that kind payment for patients our or models, withdrawal therapies approval. the license the community that bring the the on of overseas. optimistic to onetime transformative EU transformative potential both set Clearly, that pending precedent part reimbursement I'm disappointment to functional CTXXXX demonstrated has in for of are prelaunch here to to our question. going think don't about engaging with and never we, I planning Bird in in the in happened However, around that Blue holds, transformative of a be cures And going able has the was

Operator

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

Michael Yee

Phase terms expect year say not patients and that? these identifying can first questions speed of portion X you confidence of a follow-up APOLX-mediated of the II end about or of data have the at We just And and the kidney around II disease. in people enrolling what and sort confidence whether going and on your the question back in was and patients not between the slope period diseases whether and any EGFR the in you good these the the the question idea over heterogeneity what or to getting you're on and is for or decline? portion, different III Phase have these there's time whether And assuming which in around within enrolled. not a a second relates of decline to So Phase you for that

Reshma Kewalramani

and Yes. enrollment December for enrollment, just that We started III in data to Mike, shared simply months presented in really X to March. too it after the Phase the with early on VX-XXX. dynamics Phase regard and just II is comment

around too genotyping trial opening treatment that the I sites little to arms that But fact call. think a will of both as early enrollment. the get this help commented, we number where the are So with and is globe study, I the a study

why I accelerated I On year. portion, have per rapid opportunity decline. decline, drug, that, ccs of kind APOLX north is GFR is potential that And important. X And those a X when impact mean very endpoint the of of the it say question the Phase the you with to alleles rapid your really which the in this group X-year this patients, -- have gives when III assess you of

That or very call very of it II we EGFR, in in FSGS regardless XX% APOLX call demonstrated what So the Phase with reduction, is the whether you reduction another approximately was that of is in you it high. kidney with disease, XX.X% and alleles, function X kidney measure correlated on the the proteinuria. tightly -- people function is measure of

running on we So and are and the we've about getting I good and both designed in endpoint clinical the way good up proteinuria eGFR. trial very terms of where the very feel about

Operator

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

Phil Nadeau

all add years. over of for thanks in your help me let Michael, the my adventure. Best luck your next

here. going from questions Two missed certainly to us. You're be

quarter, VX-XXX.On called the then you inventory last and commercial brief First, build. commercial out

inventory mentioned call. haven't the on yet You

VX-XXX, quarter. the is on inventory curious the FDA early it's the days was there's stands still since not and clinical any convinced. initiated. you that where hold have But So Then why second in appreciating do whether of sense destocking

that you U.S. some in the about? on And is benefit or Is -- more think mentioned site, thoughts need release, what to any then instituted. all to have you the positive. curious there X what of increase you risk you release second the risk think last, ex hear. be numbers preliminary desire worried trial U.S. have do And they to because need press any I they're patients you believe VX-XX, was didn't benefit they to continue would don't the enrolling hold course, is the hold on to if

Reshma Kewalramani

on quarter. the bit start your Charlie you how question Charlie, about to for ask inventory quarter do the see talk for the with a inventory? me and little the want you Let to revenues

Charles Wagner

Yes. Thanks the for question.

increase specifically. fluctuations quarter. the of out in in We for million previously, see patient all we've fourth We channel the not to at didn't on inventory quarter. order mentioned first $XX As out magnitude quarter-to-quarter to destocking us the it's inventory We inventory $XX just and some see uncommon it call of million. called did an

Reshma Kewalramani

days earlier us VX-XXX, we this And information what agency know. shared with their questions week request. Phil, on of regard XX the is your to we questions what regulation, is list to their provide or By

to We and are and highly resume constructively type And agency the deeply with looking working confident to in we're diabetes. in forward this the X U.S. committed as trial program expeditiously to the and running up point soon right remains in as in out as The in up The is now in running, U.S. the trial regions Canada. you trial rightfully X Canada. and and possible.

regard to have in plan. that Canada hold not nothing time. been to with in additional in questions do from that's It And always any U.S., concerns have intend up or on opening they Health we with coming to for and the sites We any received have. word the sites, the more has

Operator

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

Colin Bristow

on quarter, thank all the future. the Congrats you help for This in Michael, and the is Colin. the best

AbbVie the So assets some Can about us you would gave threat. that [Indiscernible] NextGen you domain. intelligence recently previously we Cone thoughts be AbbVie spoken had early advance competitive into targeting and clinic. you NBDX into or correct? had the you have on in point. it's was the this also give I confidence before if XXX have And to curious I'm triplet a the you about work amount then was there insight ultimately and plan AbbVie which of not done, side, talked that Reshma, press they think the any readout, competition Therapeutics, the on and

get to your on I'd also. So this be curious take approach

Reshma Kewalramani

CF talk in directly about I Yes. But Colin, let's I'll competitor. any my general. practice. to add David going little then That's a and speak start I'm won't not comment. to ask to Colin,

on patients out Over bringing ourselves and only medicines we've if our innovating than today. the goal the in absolutely, And sustained in is nucleic a it. of but I'm have to forward ask tezacaftor. the few And the molecules just HBE do. the last translates even remains closest not position assays, And unchanged. a possible, been a comments why from them, really which know workhorse much we've comments going so we in something established our what and therapy bringing own years, X,XXX the David more have we to confidence CF, it care give for we and standard the TRIKAFTA we the and the we've acid into element TRIKAFTA. translational case the has that competitor you leadership today expanded remains why when of on few forward confidence it, our and of lab, efficacious David, are VX-XXXXXX, our a TRIKAFTA you of clinic. to in and

David Altshuler

Rashmi. Absolutely,

and proven triple be HBE to we with that has chloride improved where years, itself also And or relevant transport the the VX-XXX, over true As Gladeco, predict Vertex through deployment been again can the Symdeko, VX-XXX, be in over assay. and HBE saw of translationally you've that's seen the outcomes. over assay clinical and TRIKAFTA

the FEVX as also, as did change. chloride Phase study showed that II other you improved such very a We remember, sweat attributes and promising

in to we the our So believe bring not XXX continue possibly we that everything out. believe follow and that the we we VX-XXX TRIKAFTA, do might promising own but it which proven could Phase it. work III. things molecules that the triple and and assay, goes just on those beyond is XXX most to that on, molecules We do we've really in And outdo small challenge

Operator

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

Mohit Bansal

well. thank and congratulations, my as very again, you for Michael And much

might XXX. do these C-peptide? patients peptide eventually? you level couple expect last of do when there for after is independence on an Was XXX. increase a first you for maybe a comment a diabetes questions lead And flattering Could day see to to effect you the And that program. you on for levels target that the insulin do patient C-peptide plateau at X have So -- related the

Reshma Kewalramani

sure Yes. they insulin don't exogenous I comment, patient than level a you we're to is independent food, criteria. say glucose patient of that's Well, question take less going ask insulin declare less criteria. the know strict fasting I'm a actually that really well. Bastiano very that questions. And by as Their to insulin. number because good but after a level strict than reframe XXX bit and is insulin all is to way I glucose any reason And longer, explain postprandial, to to want is is and little able The criteria make this it's those they their XXX. one the meet independence is

in you about good what why experience cells question a asked about the and quantity Bastiano Bastiano? to terms effect. and to I'm Now transplants, over do durable we our quality approach. expect a cadaveric talk approach long-term with durable turn it is of we that of to think of durability And our the going

Bastiano Sanna

sales allows which to talk is occurring what fully our important sales problem, And of actually our me quality differentiated, it and the beta undistinguishable really cells. in from naturally is the about are this nature

they biology. So a common share

That cells is beta is it these where years and of comes transplant the long therefore what consistent actually biology learned with that their is time. set the for has first, are means with at patients is that successful, over the differentiation, a which immunosuppressive can That last itself. when pretty cells much lifetime. therapy last islets, when with been their compliant very, about the a is know very what about we they what all to Now and they numbers known cadaveric

of believe beta as context there to linear a of approach that the long a is and believe biology which So cells that as cells same comes natural ourselves, the the. the transplant, to the in we When we being is lifetime. last similar being for potential do, it therapeutic the

Reshma Kewalramani

was think [Indiscernible]. I that a

Operator

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

Evan Seigerman

for everything Michael, I'll add, you thank over the years. just

OUS for kind on not factor expanded guidance anything pointing I'm recently. Charlie, greatly. to in decision think into you of you you speak the I to FX missed could impact your reimbursement, despite that raise in mentioned love prepared of will You Australia announced be what if to Did remarks. you. your X don't sales. not I

Charles Wagner

Yes. just billion $X.X Evan, for above us billion thanks revenue, remind the and for sets That's the year. for another $X.X XXXX reiterated guidance up growth. billion strong folks, question. $X Again, to really to XX%

this reiterated considered the news we takes. year. number we've puts good we that is of of a As Australia, course, guidance, and had

million feel that fluctuations a inventory like high the end of things all very the is range captured we guidance. FX, end together, which And accurately consider and in modest between the $XXX of also We in low headwind. is taken

Evan Seigerman

or no FX to And of real you can more impact that? then on kind speak --

Charles Wagner

modest. U.S., our one-thirds in Yes. of U.S. the It's are revenues pretty Two-thirds ex

that program of very impact foreign have exchange a We the some and effective blunt active of hedging changes. very

for so and compared growth modest again, with the relative sort XX% -- to us, of baseline very And rate, impact with of our a is pretty small peers. FX of some

Reshma Kewalramani

more have we Operator, one question. time for

Operator

question next come Please with Bayko go will Your ISI. ahead. Liisa Evercore from

Liisa Bayko

touch we'll work. I thank hard keep with of for his to guess Michael be in all one And last hope us. you'll the we'll

Michael Partridge

You bet.

Liisa Bayko

it nice was detail that's I number HBAXc I X to the for don't see of a of X tracking. little diabetes for just And it to get some how just reduced the there metrics. have at example, Like type know be if on some see we guess, XX. endpoints want the reached would a patient the an more we either then And points. level hadn't for you good. saying simulator just of wasn't -- so peak yet. appropriate (inaudible) C-peptides, didn't -- time those are the news, that's I shown if of inflow some curious some because I'm exogenous you other mention those maybe

to holes curious of just So fill here. the some in

Reshma Kewalramani

we amount Liisa, the day first at is we've really patient patient a at the Sure to dose of XX good independent. patients And X data shared day milestone half on now we first XXX dosed on patients to dose shared full fair terms and of insulin patients in is the efficacy. date, all the who Yes. efficacy, thing. who see of across all number out one, X

terms details CGM, at of all the of upcoming safety haven't this the to that's at and share AXc, but time details across of of the patients. congresses all as stimulated you all of points we in continuous and, the glucose monitoring expect C-peptide as year. levels, the the should all right, data fasting shared well course, us You're hemoglobin

will the patients follow-up. see all you of on So with the the of all data

Liisa Bayko

one just I then And more question, may. if

have you CTXXXX, for could of finalized And the us but need give file an Sorry, have to that might next I to data, FDA? going indication you be with out this data what missed great. then where would if you there, earlier, the present you're

Reshma Kewalramani

last shared we on, last over We European CTXXXX, patients. patients have meeting. On data believe, dosed at now XX I year XX Hematology the Yes.

do, the So in patients number of follow-up see discussed, you upcoming to to this we to is filing. X areas for the complete should duration the we've what see congresses they are the package With at information, data would agency, And have more to them. year. need the outstanding that we and our expect substantially as discussions like with of discussion the of and that regard

the You know and we all designations and like RMAT such. orphan have of

had the discussion we've U.S. to wrapping us opportunity up bring both and now wrap that conversations numbers the the up point. So It's to with have this and the those EU on duration. of conversations to

Liisa Bayko

have treated current you've like should in markets concentrated. you seems capacity bunch Do basis? of any think of the it many it how of looks of be and like And and sort the could sort sense about the patients market a scenario done a how on on are work we relatively yearly

Reshma Kewalramani

would just see think do CTXXXX. and X than to numbers treated. out to how in in that come less we're for we the majority Liisa, And to for treated have about have way overwhelming a very I'll in you're procedure the are the are patients Yes. with patients able be capacity done majority Since asking we where U.S. are that be have I U.S. states, of be have overall expertise. out in concentrated, Stuart this time, see mapped that qualify concentrated centers have and countries. the bed patients EU, to in XX it and team EU the for the mapped and of this for they going in the take The patients we the right out. qualify of quickly. It we the of therapy therapy expect helming centers in who this is to yes, really who concentrated

Operator

conference Please I Michael any would our the concludes question-and-answer remarks. closing ahead. like to to back over This session. Mr. go turn for Partridge

Michael Partridge

it's very and for everybody, your a fun Thank has an Thanks, also operator. of all interact to to Thanks, Vertex, been appreciated. also joining tonight. call with privilege much honor all of you and words, always the been kind you. It represent for

team always, and As questions. the Take are you I good additional have office if and tonight care, a the evening. have in

Operator

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

now may You disconnect.

Vertex Pharmaceuticals (VRTX) 6 May 222022 Q1 Earnings call transcript