VRTX - Earnings call transcript Q1 2021 | Vertex Pharmaceuticals Incorporated

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Michael Partridge

Good evening. Welcome to the Vertex First Quarter, 2021 Financial Results Conference Call. This is Michael Partridge, Senior Vice President of Investor Relations for Vertex. Making prepared remarks on the call tonight, we have Dr. Reshma Kewalramani, Vertex's CEO and President; Stuart Arbuckle, Chief Commercial and Operations Officer and Charlie Wagner, Chief Financial Officer. access to slides this you as our that listen you call. recommend the on webcast website We This conference call will be is being recorded on website. and our available a replay

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

Dr. Reshma Kewalramani

Michael. you, Thank

to XXXX, cystic more patients is to the goal. in reach future One growth. support the goal transform drive across very doing of other into pleased making quarter fibrosis serious and progress with so and continue board Our I'm diseases, in we're this and

CFTR our has our areas than and We advanced more patients three significantly. spanning seven are treating with ever and modalities pipeline before modulators clinical disease

start leadership our I'll In and earned addition, year. franchise efforts, innovation internal cystic including exceptionally position complement CF. use we already strong in fibrosis. two built to this an continue to deals durable innovation execute to on Vertex our a external and new announced has strategy with

patients treating. increase and transformed this the have of number medicines disease CF we continue Our treatment to significantly we're of the

globally U.S. continued achieve and XX% is and with we medicines coming strong we for we to revenues years clinical are this but our we KAFTRIO stopping which agreements and in the delivers additional medicines as The represents quarter as we for people that younger markets. can the of level penetration approvals international significant remarkable, our medicine launch patients. in of year-over-year with $X.X the there. all growth billion, CF, combination high and reimbursement first poised totaled than benefit treatment triple of We're XX% bring Our treat and growth, TRIKAFTA reflects not more believe a in the

We are investing combination and Tezacaftor better as new the the to combination patient VX-XXX well as potentially with to is enhanced patients. This the regimen once-a-day such regimens enhanced new a potential economics royalty obligation. VX-XXX, for benefit, reduced with as

bottom-line and CF genetic to remaining progress medicines therapies summary, bring XX% developing expansion, CF, all transformative next our consistent with strategy the of the committed making remain in years. and and to approvals of provide patients are growth in continue significant and to the top will we to reimbursement coming for generation of the products Finally, additional combination patients. In with label agreements,

to turn cell seven me includes our and genetic clinical and programs Let therapies. pipeline, small molecules disease in spans areas which

XX months. are readouts from to the breadth We of substantial across pipeline the have poised next progress multiple the data in and six made

sickle last one-time a generated is program well sickle diseases. functional advanced from the we've both data therapy for CTXXXX. disease the of suffering remarkable beta-thalassemia, designations from cell CTXXXX and has from FDA First, as CF. to Pediatric and the beta-thalassemia. and Fast as Disease cell cure CTXXXX disease our the designation Rare prime granted both RMAT, In demonstrates been patients most for Track for EMA beyond be that clinical this potential year

patients that the program for development, we Vertex and commercialization lead to expertise, eligible manufacturing profile now this benefit regulatory, our possible. who With rapid ideal from the compelling as registration breadth clinical and the CTXXXX for all as of progress of quickly may is full the bring so CTXXXX commercialization, and toward and can take reach time

extend available. beta-thalassemia moment, studies U.S. conditioning will sickle believe more follow-up a transfusion-dependent cell been at discuss CTXXXX XXX,XXX medical gentle there the more XX,XXX once will duration therapy meetings of patients have and the and disease for cell Stuart updated with with this we This and year. dosed severe XX than CTXXXX significantly with patients conditioning with in be and nearly ongoing the are eligible regimen. patients number Europe, current we sickle have regimens in could and are disease from As of sharing anticipate longer patients and To-date upcoming data thalassemia whom

this these complete to studies expect enrollment both also in We year. of

yet regulators be on Based may possible approval Lastly, while XX conclude next not have we XX initiated, the discussions with discussions. expectations. believe to-date, provide for details we regulatory CTXXXX in of conversations to completed our We’ll further months. we our as we’ve regarding filing submissions

on AATD Moving to the program.

believe the of underlying the using to disease Z-AAT We and Our molecule cause this by AATD approach of appropriately addresses lung disease. this for treating the optimal approach the liver program. it both VX-XXX most misfolded is our in is and targets the approach molecule protein. a that fold the is manifestations only small corrector the advanced

now enrollment of also completing the follow-up have in safety II period. the completed recently Phase and completed we dosing proof concept study We VX-XXX. Patients XX-day process of the in are of

readout data this We quarter. are on later track for

characterized is Turning a kidney of disease particularly form to progression renal kidney disease renal aggressive to declining proteinuria, by and APOLX-mediated ESRD. disease. APOLX-mediated function

of being of FSGS, lead mediated Phase in concept VX-XXX weeks. is and in study Our in X XX course this reduction APOLX of patients the in proteinuria safety, molecule pharmacokinetics the proof evaluating studied program over

XXXX. this of have to half from in study expect the second the We results

Phase lower These profile previous the studies, those these start year. well-characterized treatment in and including evaluate bunionectomy in exhibited program we and safety the next based VX-XXX data the target of of we of with positive different we Phase recent tolerability the X in lead X studies and supported the NaV to with X.X will program. of healthy that Phase for this X proof in indications. second on NaV expect In visceral the into The move volunteers, molecule our X than required also Next, considerably two of and are abdominoplasty. three development. announced Phase X.X decision pain trials molecule our advance three a pain week our pain, turning With pharmacokinetics. acute to pain plan VX-XXX, to pain non-visceral, concept evaluation The first Phase we in to following the doses efficacy VX-XXX of broad VX-XXX, half studies X the the categories validate and studies acute this in Earlier inhibitor. was the at to to safety, favorable were

X/X type Fast program, open and in and for off to the on difficult Track in cells we our this to hand X we look diabetes With Finally, Stuart. our diabetes forward X year. providing those study type updates the received Phase alone to is initiated patients with study treat it in FDA progress QX. now I'll designation Screening comments,

Stuart Arbuckle

revenues pleased Reshma. and with year CF grow. you prospects to you, our our One the performance Today excitement review Thank commercial QX into our to pandemic, I'm the for continue about CTXXXX.

EU. were execution in $X.X Our strong billion, the the continued by and US both QX driven global revenues

in least patients treatment in patients impressive with just these compliance mutation, Many with initiated these as the the all December In well U.S., was the on markets include years in rare one are performance is as eligible with initiated at the in FXXX treatment and patients newly have this as have and be we Outside U.S. patients approval XX in XX XXXX To-date CF now have based the of and group. have U.S. mutations. patients. TRIKAFTA's access, in adoption to seen the Our persistence U.S. of of where impressive. years patients older high pleased remain eligible by and continues the we del older in TRIKAFTA and KAFTRIO the nearly to uptake original approval among U.S. expanded

have particularly risk-benefit are profile the KAFTRIO. rapid in We the strong, and reflecting very similarly and of and UK favorable Germany compliance seen persistence uptake,

than in launch Earlier of our we revenues year, the that XX% million, year KAFTRIO. were primarily QX this to successful updated ex-U.S. same XX,XXX approximately period are Our $XXX due last there higher estimate the shared Today, U.S., benefit of our additional and are half CF with from not in about Australia. treating are living patients, patients yet XX,XXX could who an but people who the leaving medicines, we all Europe, treated. Canada

approval, regulatory ongoing teams will able and Going such our where be have forward, are be to to we countries will the hard U.S. to the in remaining but from Spain to launch, patients. secured we and reach be to large reimbursement, these and outside these the regulatory and through of our uptake yet we Australia Approximately eligible expect countries benefit treat reimbursements not are progress approval to we In working we untreated we could teams that and reimbursement older patients ensure where that two-thirds have secure of the confident reach are who will and able markets. we confident as France, very access In are patients have continue make XX able reimbursement, KAFTRIO we all patients KAFTRIO.

and Benefits ensure For TRIKAFTA there, TRIKAFTA in continuing have benefit quickly funded from Australia Committee to the example, as Advisory approved to was are possible. recently we collaboratively all as treatment patients to Pharmaceutical X,XXX with government work can who committed access to

remaining we the of expect this The Lastly, are XX. in in of approval we are the half year. regulatory XX,XXX approximately patients Canada and majority than still younger awaiting approval second there

our olds continued making support development approval label continued and the the come. Our XX are mid-XXXX here. of for the and In in for achieving year of this, products work we We and new approvals of Europe expansion, global patient new are reimbursements CF population the six tireless regulatory progress will populations the file continued work in and all approvals younger submission community. summary, to is secure In as of drive TRIKAFTA ongoing we good well. this U.S. in in grateful for to and these to growth to years in anticipate progress in

medicine is Let's now regulatory that CTXXXX, and next rapidly toward commercialization. which submissions is advancing to turn our

sickle by the Such based at progress coordinated to population gene-editing development, be made, the lead a and both patient are believe for global manufacturing, and regulatory as path to expertise within to in the and for ensure companies to leadership expeditious patients expand approximately would who severity companies approximately Europe which and Vertex in disease expertise more Given having XX,XXX conditioning efficient at on likely will beta Europe. thalassemia the of established dollar centers bring product forward has CTXXXX, commercial for both and in therapy treatment as our treatment will regimens U.S. provide our The regimen. CTXXXX be the tremendous serious well be a launches, maximize aim program of potential are taking the Vertex, hematologists the in to this cared that quickly genetic the therapy which reimbursement fits role world. proven their total can patients able the that around Sickle other CRISPR, with going aspects we to access the conditioning disease disease. there multi-billion beta-thalassemia number diseases, specialist we potentially current patients Today a eligible curative approved the Vertex’s By launch, infrastructure. more of markets. eligible busulfan this, Europe, U.S. this sickle patients on Patients and partners is strategic and opportunity commercialization gentler would bring this be pursuing cell a have transplant be and opportunity. CTXXXX and cell the to groundbreaking will disease potential Vertex's focused and in a patients. the actively CRISPR of for to patients, focus the our would represent doing first momentum, therapy with the demonstrated well By of by XXX,XXX most all we the great based of primarily commercial program cell leverage are and shareholders. and medicine in and covered specialty potential in on CTXXXX regimens. U.S. that for

much that on believe to to Charlie. I'll the very is calls. XX submissions we XX our CTXXXX providing way. work forward months, happen breaking now, new work And Given grounds to I'm this under more over many next exciting is in details regulatory future hand it respects could in on looking pre-commercial and

Charles Wagner

Thanks of XXXX, record exceptional quarter continued financial its Vertex Stuart. first In performance. the of

were the revenues by XX% have increase $XXX England Germany. of in driven outside of these growth the to represent product countries $X.X compared the quarter first billion largely the U.S. prior First we of reimbursement, year, uptake a of from results where included rapid XX% quarter The in over ex-US secured results and total million XXXX particularly revenue billion, $X.XX U.S. the in and KAFTRIO

million non-GAAP $XXX as across continue first clinical quarter quarter pipeline. progress we investment driven the R&D primarily QX investments and we and Expenses first Our compared regulatory broad SG&A innovation will by were expenses toward proof-of-concept $XXX combined million data XXXX. for to and drive were expect R&D to and in of be further substantial our in our

our recently Therapeutics. discovery ever by growth external first in with with profitability, combined both our more corporate exemplified billion Consistent quarter agreement This in CRISPR new managed in before, collaboration carefully first and XX%. investing margin translates strategy, With innovation than to we continued quarter strong we're with revenue revenues non-GAAP amended and of collaboration was in R&D our Obsidian the ended growth including spending, $X.X most innovation. with with our operating Our cash. and a

guidance. onto Now,

previously of We revenues our total range $X.X product to $X.X the for issued are guidance billion. billion XXXX in maintaining

Our strong, directly an they underlying demand. reflective QX also revenues of include are patient $XX that from were inventory million approximately not fluctuations but channel very benefit

a currently products not additional population obtain for we approved in mid-XXXX. six expectations As TRIKAFTA approval guidance for any an are expectation reimbursements reflects of does the that It where plus our regions XX-year-old might U.S. reimbursed, we XXXX our to the this year. in include reminder, in

range are our For $X.X reiterating non-GAAP $X.XX OpEx, billion to billion. we guidance of

further R&D continue programs. advance greater of We XX% as expect allocating OpEx to pipeline our than we to

decisions a over course programs the to number clinical drive further data and year, data of poised We these of will the from investment. generate on are sets important

very performance forward you of guiding progress continued in with course our XX% For of look we are rate, updating first I'm this our pleased XXXX XX% this to non-GAAP the of range tax the to a and year. In over our quarter to year. on summary,

remarks. closing for Reshma to back Now,

Dr. Reshma Kewalramani

sum, the Thanks significant progress made we've Charlie. across In business.

innovation-based have confidence continue in We growth and strategy. on to our execute

performance. both investments, is are working discovery is strategy financial driving medicines. and new Our and Vertex game external development of our internal exceptional and changing delivering

continue questions. pipeline the now business and we'll Our to call is broad CF to poised to advance. open and grow Thanks, continues our to

Operator

Thank Instructions] from And our from Jefferies. first Yee Michael comes [Operator question you.

is Your now line now is open. line Michael, open. your

If your mute, is you on phone it? unmute could please

Michael Partridge

can to next please Operator, question. you move the

Operator

you. Thank

Our and next question from comes Phil Cowen Nadeau from Company.

open. now is line Your

Phil Nadeau

thanks taking my and for question. afternoon Good

clinic Thanks. confirm question, year. case. us has a into you to seem that's why a give the the maybe service proportion the guiding molecule question on [inaudible] common and did that program, like getting still levels from data I if in change necessary, seeing? investors then been past you is proof the another from recently you've not? a has patients responders moving in most AAT changed? and prepared concept Can us your need of second think maybe the this And the Can guidance what you're upcoming most whether been and reason actually there there been AAT we've to what remarks on your it's be of what what give thoughts It recent are

Dr. Reshma Kewalramani

this you. is Reshma; that let Hey take for Phil, me

think we I about be the What where are approach the molecules, pieces. and take we in really with a are, XXX little expecting? are other the bit would so program? success that me let and questions What portfolio

approach. the Z-AAT misfolded review to refold So call, it's molecule you small reason disease. the on know but the this for that for protein, in we the taking are the correctors important both VX-XXX approach our the to to to clinic. to others this, that's And enthusiasm approach is understand fundamentally hence that our that and liver lung, is is only this potential treat holds this

really me fact that remarks important the heard hit milestones. we've some talk about the prepared in You

are with We enrollment. done

follow-up period completed are patients they Our XX-day now the in and safety the have period. dosing

functional the looking what third, what very AAT and obviously had from Here's three AATD, relationship; would The a and would is to elevations I first front first with and safety. be study see. really I'm I'm for in the time looking what excited dose second, and exposure that's things: or response I dose for level. That's center; success. Really we've in patients VX-XXX what consider so This

molecule to Phil, portfolio in well. In portfolio a beyond all the we have of as and with that the true for be whole terms of programs pipeline continues as AATD the VX-XXX, of molecules and

Phil Nadeau

Thank very That's you. helpful.

Operator

And question Geoff you. our from Meacham Bank next Thank from America. comes of

is line now open. Your

Geoff Meacham

for just the Hey I question. everyone, thanks ones. two have quick

lead just analysis? just clean then ultimate On may corrector the some in you with decision even to economics the on I have always Reshma, follow-up was or or view better today? was ongoing AAT, only when beyond a assuming and of updated as CRISPR wasn't of based the you're for to do the program sure or a profile, what maybe there on Thanks. mechanisms committed is thought it's the the AAT XXX, the Vertex commercial data action, deal? kind is you there on And a what other can, catalyst but approach for have feedback a regulatory there that that was safety pipeline approach more you know

Dr. Reshma Kewalramani

Yeah, sure.

Let me the amended agreement with question start CRISPR. with the around

Geoff, that number happened that six us to know to the of course a let's amending led say, the catalysts the of over you eight it's last, You agreement. know months

it a complete but both beta-thal to also data expected. back XX we sickle we has people even progressing delivered is cell where we sickle in patients lot The this We're than between year year. this to in proof faster results now we've of that beta-thalassemia. from and are momentum point go and disease program a and XX program behind have short data now more and enrollment and than at of dosed it concept of nothing really overall looking cell last shown both we Not you If have and is the we've year, only last from that, remarkable.

as infrastructure is your think to answer regard together possible, expertise this The the question your underlying strategy just the and that perfect small correction disease. we like approach, opportunity how patients as for This How AATD. the quickly no as is to our committed as to full cause small fits to in we? a molecule that disease? many do the have the our that exceptionally treat committed we question And around is you to know protein, of really of Vertex liver misfolded put and protein With about to to the to get on AATD, underlying cause and right? possible. take the you glove this there time and weight approach, are target the leadership is so role disease all You of than experience bring the misfolded and with of the are With better that manifestations. approach a doing, AATD both the regard molecule and lung to disease, in and Z-AAT disease

and AATD are the we committed to So to molecule small absolutely correctors.

Geoff Meacham

Got you.

you. Okay, thank

Operator

Thank from you. And Michael Yee Jefferies. from next our question comes

now Your line is open.

Michael Yee

Can me you now? hear

Michael Partridge

Michael. Yeah

Michael Yee

Okay, diabetes. so data this, would comment it realizing surprising would to there of actually great. you I about actually be just but just talk you naked HbAXc, the know of have proof maybe talk cells. is, Reshma Thank what is year. concept a there into later what much. see about likes maybe in the you Can value etc., I question changes this about about see to

Dr. Reshma Kewalramani

understand we where little we diabetes the you're background data. program and can bit some and about Type see and bit yes. to about are when about I certainly the just to about heard I your Yes, little maybe a X Hey asking a Mike, expect question think

program IND one that's earlier that the right let's study approach, that flavors. is the is Type the one patients; the diabetes X/X One, the year. cell ongoing had cell-based it comes that a Phase the the so Okay, and Phase two call in study in X/X the that's program in really X in program is cleared That's now, the in patients stage. naked

Plus program. The the Cell is second one Device

the we're destruction exactly very years known. lead solution cells. cell more here. bringing of risk very that been in of taking understand biological Type Maybe to because been clearly and quality cause Type the just pancreatic is little Now, to known for transplants There cell is things more have two is, diabetes, terms and the what forward. already The issue X many for disease transplants we step islet let now me that right, cells of outcome the X that are of back autoimmune importantly, that islet and approach its done Islet has quantity we diabetes, patients great Semma years. Vertex and with in have that's explain

So program, to that number about program, the equivalent chronic course in would with with immunosuppression. that an any X/X with that program, are we severe million could with and its XX,XXX DEVICE If IND; these the And DEVICE which from the diabetes include benefit would I program, are is this from Phase be U.S. the cleared patients, think go regard over so IN benefit CELLS patients could cell the not let's now. EU naked Type it's CELLS diabetes this in say, of then would program IN immunosuppression. timing, people a the X there naked in require the just to

program profile. I of give say starting would a you mindset us and it that should efficient say recall should then the data for I that would reasonable lower then patients the just readout And an from get a think like You like at have as just will target that CTXXXX, CTXXXX program, good of you of XXXX that process. toward number dose we dose. the with would a a and And sense to a we're

Michael Yee

you. Thank

Operator

you. our Morgan. from comes from Cory question And Kasimov next Thank J.P.

open. Your is now line

Cory Kasimov

my good for guys. Thanks taking afternoon question. Hey,

in just where Is you development larger Thank I looking expectation recent your add we the forward. of kind if of business is you. city should deal there as stage ones On still or plan know differently as are more deals about of the can we be expertise? that come. to a the at collaboration, earlier curious the some smaller thinking or M&A heels on CRISPR, strategic front, any the of expanded well with smaller going

Dr. Reshma Kewalramani

is again. this Hey, Yes. Reshma

me you. that one take Let for

is our over that we're Vertex that several I assets to that, the I what toolkit strategy development innovation business that add looking assets say internal for for assets Research we last or for Our tools with external looking and And Regulatory, that pleased it expand same complement disease as our done. pipeline, we're has our etc. areas CF the mean the years I've is, exactly in been mean we're really and could of I looking value that fit interest. Development deals we've when by been

You could you look at collaboration in Semma should about or more when to the what same. of Exonics also as examples, you couple expect think look Therapeutics weeks approach see the That's external and with just of about but agreement last and our should Obsidian. CRISPR agreement you amended back with you could think the innovation the with

Cory Kasimov

Thank helpful. Okay, you,

Operator

And Richter from And next Salveen Sachs. thank from our Goldman you. comes question

Your is open. line now

Salveen Richter

Thanks.

you're the XX X just ascertain the trying then dose for about that Phase and just to AAT DMX? microMolar how upcoming And if where threshold the On X the serum what vivo context on question, response and stand us remind CRISPR/Cas you could on a in with of are in work levels, second Duchenne data. you you thinking

Dr. Reshma Kewalramani

this Salveen, I for questions those Reshma. answer you. Sure could is

first and back Let DMX and me then to tackle come I'll DMD AATD.

have you've no association and experienced is late progress next development, both development. with with responsiveness, magic and there study those I dose process here. that's that question, this when or we're of on field good later What to preclinical doing those is making the an we specifically expect dose in making data dosing, very safety, what development, totality day. proof-of-concept you the we're I exposure talk clinic, about you the With now. we're response another been I evidence as is this levels. are higher certainly are we're Obviously, that come exposure deliberate in elevations going regards. there is DMX the the on greater DMD dose the and progress is I'm greater is programs mean know that in close in what to data what's I'm AAT others share for about looking that's in regard the So be that and steps will to AATD eager that mentioned, regard and but quarter. number programs analytical being and by real will to fully seen setbacks into careful and very from that very to functional the with and the you readout to for a eager

Salveen Richter

Thank you.

Operator

And thank you. And from Citigroup. question our Bansal from comes Mohit next

line open. is Your now

Mohit Bansal

you. on a question thank Great, AAT. my other Thanks one and for Maybe the question again, question progress. on congrats all taking

in human especially effect VX-XXX from if in see suggest drug? the naturally that end, are any levels on being is patients, that AAT checks believe AAT than just to functional trial, to or fluctuate Our there levels a rather do alone, of chance in and with be these body some reason it could depending let's due to say not acute if phase you in elevation just you

Dr. Reshma Kewalramani

we're we're elevations Yeah Mohit, for understand say, in part your when that's for I talking level dose this is is and I elevation Reshma. when I looking about of looking what say question responsiveness, want I see do Mohit what real really we're AAT and functional right. to

exposure with dose that's elements what looking AAT looking increases all But levels are I responsiveness, AAT up, when for; dose. go looking levels of see AAT elevations we're functional to and functional We varies in that mean functional the I say we're levels. that Those real actual for. with are what

Mohit Bansal

Got it. Thank Helpful. you.

Operator

Evercore comes question our Bayko ISI. from from next Liisa And you. Thank

open. now is line Your

Liisa Bayko

I about a my for program. to question. thanks Hi, the wanted ask little pain bit taking

for clinic. about setting suited different started to of from talk kind your VX-XXX. well it indications you of the be You It’s you're you're Those to intentions about maybe quite far it into how some not talk as is a more bit it's actually Can in VX-XXX. questions. terms little made in Thanks. or acute this eventually clinically, set chronic? I my chronic as are that know going And

Dr. Reshma Kewalramani

sure Lisa. yeah, Yeah,

going break to comment perspective to going know I'm the first Lisa to I'm going Stuart two you parts ask to on take from the things R&D here. an need R&D ask and I'm tackle second, prospects that to commercial to know me the from to Let up comment things Stuart I'm to into and Three this going and the the perspective. on

First, validated it Phase the musculoskeletal a a validated VX-XXX was target three note is pain it different target proof-of-concept is positive readouts that is pharmacologically let's had it's because but neuropathic across call target. and exactly and pain. The also right, to indication, target, That important NavX.X acute, genetically NavX.X. X here

know is to our with The second thing approach VX-XXX.

really say and Phase than it's thing all will looking it. and it to abdominoplasty. in inhibitors, last but that We So R&D X the is that couple as Acute of And starting duration but and that of from how of first we're pain, only registration previous indication. considerably the including NavX.X a and formulation to PK studies and trial, treatment, days of we've VX-XXX multitude with at as acute for, emerged pathway of and know our manufacturability. exposure be the the properties a do has pain bunionectomy tolerability, safety on terms a its the what name significant also because in we it's in non-visceral a short very or is obviously before. and implies, doses side bunionectomy visceral I'll with ways, XXX it’s benefits are done has we're saw doing then that lower including not We're

going turn little bit tell to I'm viewing it over the to size a kind Stuart market the I about more So that. there. opportunity and to that's you of am how

Stuart Arbuckle

Lisa. hey Yeah,

think both in generic to neuropathic so of that, we are as prescribers, and of and play looking acute a and in in segment on we are great pain deal terms also corporate we we and multi-billion market. and by generic which focusing significant neuropathic of the pregabalin feel an really both so of strategy genetic and innovation, superior in used we we'll in that and the currently specialty segments the really, which market, segments tolerability forward patients it market, opioids by markets relatively currently are both segments is both they our largely be compete those in both of are group be a of bring superior the of those segments the agent, are result overall dominated way need that because efficacy and of in served with profile, those concentrated believe a will in into So of to Both of a of particular market. strong targeting able

Liisa Bayko

you. thank Okay,

Operator

Thank you. And our from Wang next from question comes Barclays. Gena

now line Your open. is

Gena Wang

Phase anticipate will one Can ask question further study? also planning also or Phase do important did to room and one that have responses in showing still the share also dose the mentioned with choose good, how very you data those X criteria. I were like up? looks Reshma, if or dose not have us you. you further And escalation either X you you on AAT optimal you doses some Thank reaching safety

Dr. Reshma Kewalramani

we way choose same programs. the of Gena Sure, all we our AATD our are for the for really doses do as

AATD. little that to I you're behind let How about How Gena, If And Gosh! bring me question at you in our be confident in mechanism What the is how models use to You are Obviously, are doing that going CF we CF be a we do programs and I your patients. clinic and that. see disease? what forward very really the in what that to data looking are in translating be here good bit record for is very just a and quite this medicine example you know talk this able that is our we your we this? into a think well in-vitro question our the of clear a we confident in exactly elevate well, into in that’s through. general, maybe same and and view track methodology might have

to a looking This have with committed confidence like our disease. We've disease this the many we and to sharing that years working high approach we on are and forward mechanism fits We the glove. right strategy feel been for you data. I'm this

said, soon I As out that's coming very to quarter. in going be this

Gena Wang

you Thank

Operator

comes Karnauskas question from next Thank from you. Truist. our Robyn And

is open line Your now

Robyn Karnauskas

really Very questions. good. Well done! two So quick

X, I into Phase a with would advancing that triple, direct X. that you patent? of arm you what it, you you the think be, Phase with you angle, What to but know go question on program What you is of get triple haven't So talked that want said kind my sort into to know have always XXX, if you’ve expectations did lot the program? the combo I first control need? partner about would

Do a a for Thank XXX, chronic plan going you that as into to for on you back and still the for think us still is would in? Will is educate it Is what that for to catalyst that pain you. you be well. case? So that? partner plan partner would then and pain want the a go deal be to big

Dr. Reshma Kewalramani

Hey Sure. it's Robyn, Reshma.

do pain me first. Let

heard and feel acute we those managed on markets that are be you believe We to Vertextian neuropathic Stuart say, in pain manner, in [ph] very both focused pain be specialty we are the pain conditions as and to which comfortable. and

is something focused get then looking say you've pain musculoskeletal talking acute at. we're for here and neuropathic pain. not what is acute that's is we pain the we're and So are that we What to us pain we on, about but in that when first neuropathic heard past what is example, would what commercialize, in considerations

Going digits low the once the looking to by have and that double superior improved to royalties VX-XXX, what the is Tezacaftor, going combination are really day that VX-XXX is a and is a and that here mean economics, for in dosing and wave low we're patient, indeed also potentiators what digits. I the single correctors CF, combination and efficacy to of potential of has next at combination of from

program X, we We are wrapping will year. Phase to are happen which that to I that readying with on up program expect later conversations this go regulators and our

Robyn Karnauskas

great be you would direction? Vertex How in of go case do get you there? drug? people have That that a convert you that how for to will sense encasing do Reshma, – your this of a shareholders? get Do you how

Dr. Reshma Kewalramani

Yeah, yeah.

like Robyn do we that know something we into forward going the patients. is we are expectation are XXX to late-stage Tezacaftor, because have doing better molecules so for full when development our XXX we bring to that combination, You be able

all about to first-in-class We we aim and ourselves. out-innovate are best-in-class and

And fully expect XX% triple is to control in It medicine. this of clinical combination about going up the and real been patients trials. the the a patients what is There to of triple in to once-a-day having the we efficacy world combination be in has is fantastic arm to what be we so I and see potential arm efficacy, to the of for saw has bringing a treat in them. course forward dosing control better terms

what bar, a but aiming indeed is So is yes, high we're this that for. very

Robyn Karnauskas

you. Thank

Operator

Thank from our you. question Harrison Stanley. next And comes Matthew from Morgan

now Your line is open.

Matthew Harrison

afternoon. maybe where question. countries bit where Thanks some about I to Good in for comment get a timeline you European uptake then the the Stuart, more yet. Great! you're give Thanks you sense for if much. little detail on and across wondering just of in thinking was us you do taking I the terms very have what don't reimbursement the of could countries

Stuart Arbuckle

of and year here what also growth same the we've in QX that's what's strong Yeah, hey levels saw the question. almost superimposable on countries been and terms persistence so last over the Yeah, the have we do strong the year. in a our for the versus indeed, in has of Matt, uptake, in U.S., last reimbursement we in and but of quarter U.S. very thanks the where revenues driven seen, really the growth we've XX% compliance uptake both period ex-U.S. outside of terms same seen the very, that

very, already driven we uptake as by have that's markets very in those impressive reimbursement. they’re where So

to come. don't Europe. added and countries continuing be recently in during progress received approval we waiting very Finland there France, obviously can first in and Switzerland is Israel good like few would confident for to countries regulatory in but are going have I in new countries. Australia. the in have continue reimbursement with and feel to approval regulatory regulatory years continuing we like Italy places Spain. to tell reimbursement to to year, are and work strong and those growth regulatory the countries We have we and working in we in months we getting that's that yet approvals Canada. we're agreements Indeed in of that agreements That approval, both our get reimbursement make we believe you with We're payback that and new we're Obviously, the continue in we where still franchise have why very, CF very reimbursement this What

Operator

RBC Abrahams Thank Capital. you. Brian And our next question comes from from

line now is open. Your

Brian Abrahams

questions. Hey guys, thanks taking for much so my

then do ongoing I steady in reach levels SAB XX-day I'm would your take a serum? wondering if or to data potential Just full and the have might as time we by a it the couple just AAT to for Wild Thanks. at the look snapshot work, relative the how in sense how AAT of our quick a should you ones Type? on corrected AAT. From agent detected functional reflecting functional of guess long state

Dr. Reshma Kewalramani

that to not. helpful. is data to work we've is to AAT, functional going With in corrected is those and Reshma. this proof-of-concept work, both sufficient all is AAT for of our for or Hey, think I Yeah. modeling I done be the we not the whether and that of state be preclinically steady reach going that very us XX-days you. this could to take X regard study get from or Phase From the

measuring going to be antigenic levels, measuring AAT be going and assays also to but levels indeed straightforward. are those are We we're functional AAT reasonably

think to don't challenge. that's any pose I going that So

Brian Abrahams

Thanks.

Dr. Reshma Kewalramani

to corrected we've Brian, protein we approach the Just all molecule Wild produce is the the complete the these same in that events small with thought Type. that of as done,

Brian Abrahams

it. you. Got Thank

Operator

Thank comes you. And Fitzgerald. Young our Alethia from from Cantor next question

line Your is open. now

Alethia Young

start when wanted question. novel just think we Hey Congrats you kind CTXXXX, you the conditioning of could to kind my from condition happen regimens years of see kind to something relatively couple and little do think tolerable taking talk I think you might a launch quarter. more guys, thanks that's a some regimen? soon do after I guess Thanks. on of for bit a about into possibly or and that commercial getting potential for

Dr. Reshma Kewalramani

is this Reshma. Yeah,

you. Let me for one address that

to And I that busulfan-based believe we I not talked amenable at that That regimens if, enough at a Vertex You be the is and initial I when, is that will we severe that launch of we on they up reason conditioning right it's about therapies, know opening gentler say think here, do as that's earlier with be XX,XXX said, stem important gentler opportunity conditioning that would current we also regimens, beyond the being patients think the the why working be that a regimens. cell those cells matter surface the regimen be will already there as wanted already and that known discussed, as this really for as are I has are in do known number oncology conditioning original working beta-thal. is both markers for indications, are [ph] There gentler well use and are that we at regimens, regimen on are targets to cells and target, already know on are what CRISPR payloads looking we well. hematopoietic in companies. of cell Therapeutics sickle example potential of conditioning about this are remember, as as gentler for PDX-XX other specific are would the terms

in I the I in work think is matter So that net a of when a some, it's do something of future. near there not is is this lot area. and think that if, and this

Alethia Young

Great, thank you.

Operator

question Stifel. next Paul Matteis from comes Thank from you. And our

is open. now Your line

Paul Matteis

been so ask be much. that transactions year, more thought more and on my development. you've but billion Might larger in What's get one wanted and size this kind thanks had here mistake, on when Hey, cash. your a said sense doing more to of open of business you look I quarters $XX we couple over close question I you same. I sheet of balance Reshma the deal at to to a prior the then made guess

Thanks. than So bigger a of strategic we say, more likely mean and deal billion does number transactions? kind a a or smaller expect maybe of really that’s so that shouldn't

Dr. Reshma Kewalramani

to laid is high the speculation, going but a few Paul, the you out you and minutes I to it's strategy business ago. the feel about our deal strategy know and development I'm strategy Hey not add our not that exactly should It's about same. confidence all size

Paul Matteis

Thank you.

Operator

our comes Skorney Baird. Brian question you. Thank And from next from

is line now open. Your

Brian Skorney

of and on that I to question. the get sort be based approach the dystrophin used for – mutations. DMD of about mutations taking of groups, your unique are kind in kind and my areas Hey, but even thanks many skipping the on various wanted the correct some within seem hoping insights to how those to mutations you was it? do various occur of for amount how the full think I Exonics there to gene-editing they of Well,

edit one one I box is that disease mutations have product? the tackle in when think guess So these you landscape of of of to to a terms in an out of whole separate the can of you something each would sort gene-editing, with product be or there tackle done

Dr. Reshma Kewalramani

Yeah, yeah.

is it, our So approach talk we when or One based fundamentally can this. protein, of code doing, you to full-length to near approaches different which so not right. this DMD, full-length about are produce which two the us, others that right, microdystrophin and the there many reading reframe gene-editing are in on is CRISPR

potential you anywhere the that course disadvantages about protein. and advantage a not – The to for a it’s is a is when approach that each, So it's microdystrophin disadvantage advantages there of it's approach think the a full-length singular are, to multiple close and Exon. of

being exactly the are what is we that benefit of the Our trying approach is target. great protein, full-length is the what which has protein, to

once been bring with first Exon. So of here an have obviously the regulators to more and because Exon having done full-length first approach first agreement see be Xrd, will the shorter protein we is efficient on timelines, the that critically intent that come far important. with Xnd, the our forward have have is work And those approach and the forward would Xth those Exon we bulk and with is what to as then an to that

Brian Skorney

it. Got

So then entire like of beta added just Exon edit to the the in the case mutation? effectively clarify, XX to out would XX an approach amenable Exon the

Dr. Reshma Kewalramani

microdystrophin really would really not. few than or a near have important full-length here few a remember, protein readout few And That's vectors was we the need very you the could few guides we're bigger provide randomized it year mind of January vectors, a But explain our full-length the dystrophin. trial why mutations is more full-length mutations. amenable vector issue and just of in a a need and to could a and the a to of of get item one a is of are the not is the of big keep guides. to that we in in and it was whether take that is expected controlled to picture all difference trial. that positive benefit the get item I It know first will microdystrophin, approach or Maybe picture we all this then talking What short-form the a there to not way. using this different bigger the about to the here DMD, protein,

Brian Skorney

you. thank Great,

Operator

Michael to you. would the I And thank now, over like And closing Partridge turn to for call remarks. now back

Michael Partridge

Thank good operator. a tuning tonight you call. you conference The for available quarter disconnect. additional Have our first is night if team Investor all Thanks Relation and you into have questions. can now

Operator

participating you you and This concludes disconnect. and today's conference may have Thank call. Thank for great you a your now day!

Vertex Pharmaceuticals (VRTX) 30 Apr 212021 Q1 Earnings call transcript