VRTX - Earnings call transcript Q4 2020 | Vertex Pharmaceuticals Incorporated

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Michael Partridge

[Call Starts Abruptly] …Relations for Vertex. Making prepared remarks on the call tonight, we have Dr. Reshma Kewalramani, Vertex's CEO and President; Stuart Arbuckle, Chief Commercial Officer; and Charlie Wagner, Chief Financial Officer. Dr. David Altshuler, Chief Scientific Officer, and Bastiano Sanna, Chief of Cell and Genetic Therapies will join the Q&A portion of the call following the prepared remarks. 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

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

Dr. Reshma Kewalramani

a to around programs, an then for this including an begin performance, XXXX businesses unprecedented I'll and of of call our evening's the people XXXX and was pipeline, on governments, upcoming milestones. comments number our turn year with overview and R&D the all globe. generating backdrop product against to XXXX. representing also It the for commercial of faced extraordinary Vertex more pandemic, delivered the XX% $X.X the challenges billion remarkable growth compared Vertex. performance, year Despite was in than we revenues, a

treatment for known position. number advanced patients We in And increased year the In the our significantly fibrosis, with discovered cystic has up Vertex cystic financial after KAFTRIO, with marketing XX In By the developed and XXXX, the pipeline full CF Germany, as and eligible KAFTRIO end older UK, to of by we TRIKAFTA agreements received portfolio the first access also the U.S., majority substantially. the that EU, at Ireland EU thousands XX% latest years Denmark, approval in of TRIKAFTA. has treated on the of of fibrosis. of in treat our the year, in early the in direct or the TRIKAFTA U.S., QX, it’s the EU secured approval KAFTRIO. with England. and deal transformed to potential patients, the the reimbursement of the the authorization in also year, meaningfully strengthened to were of access of medicines vast patients reimbursement the across gained patients in and with the and medicines have end and in and these,

combination expand terms CF, sight line the to steps triple approvals of patients as well in in continued the to EU access in have we to of as growth to geographies, next extend more we In significant as new and and in olds rare the treatment our approaches regimens further to additional the X define younger small with other advancing molecule patients to starting combination U.S. CF. will mutations that with leadership long-term XX in also We're populations, year cure

the into are programs, to clinical our disease to also in move several therapies X seventh areas spanning goal We transformative first now therapy. our XXXX to area with And program year proof-of-concept and disease all Our people VX-XXX clinical disease. bring and for pipeline. our clinic multiple poised an remains important we delivered two advanced cell-based for in this modalities. type the was diabetes, development

which position, to continues Turning our financial strengthen. to

in we revenues the finished we and cash. As profitability growing, patients, with treat our more are billion year and $X.X

and respect way What innovation, strategy add financial to our invest our CF, is assets assets to firepower. this long-term create growth that and reinvest drive to We to identify external to same, the both growing value our fit that toolkit. internal our in our strategy, in complement internal that changed and and pipeline, in external. seek has CF continued position our R&D the tools With our are in is innovation, best remains steadfast capital belief

sheet, Given TRIKAFTA now XX%, and and treatment and our KAFTRIO, where of for to and in strategy in balance regimens line plus a our fit R&D stage. assets assets novel given the last we are are to on mid consider we focus late that with and sight position

the we're cell and pleased functional the also were is clinical We're share Annual targeted I'll sickle session the for groundbreaking recently development with patient molecules, data living to study Meeting. onetime milestones data our our years and most cell an on developing Medicine a I'll two published holding therapy, in on R&D cure detail, and thalassemia additional in programs. and potential follow-up disease multiple thalassemia of start disease with disease. potential In these we're with CTXXXX derisked course of a number American the CRISPR Moving transfusion-dependent presented of New small and update to of seven showed partner areas our active gene total, in plenary and follow-up the the clinically be CF and the with beta England outside program including focus in long-term pipeline, on provide of that proof-of-concept first the has upcoming Therapeutics. CF, modalities, people editing program December, These Journal a to spanning transform Society Hematology portfolio which on the In treated at including clinical the each CTXXXX, of outside of completed demonstrate enrolled in that to diseases. study. in

possible. to able patients advancing clinical this urgency, our to bring is be therapy development as are to soon through We with as goal and

patients duration the define Over enrollment of three small a additional for this of disease, molecule discussions underlying thereby that development manifestations or about address our our correctors milestones CTXXXX: of AATD follow-up for this the focused the therapy; regulators cause to lung on a antitrypsin of of efficacy completion Z-AAT ongoing package further with defect, and third, two of treat progression of the alpha-X course liver our more data presentation mark data folding longer the studies and of key In that both, for with are there approval the for we're progress the and of and durability CTXXXX. will the our CTXXXX; the of of will of needed disease. and filing XXXX, the clinical First, deficiency program, protein

advanced we our this of X of is to advance study. VX-XXX, is most progressing our an expect across study the say AAT in additional a protein. is to half to year. also on track in of Phase study functional for development Consistent advancing this through clinical the levels the to goal which areas, and molecule The strategy first is target safety, proof-of-concept corrector small and patients ranging into early through of approach, results Our to continues dose portfolio dose simultaneously molecule have assess our we The enroll multiple and each molecules that AAT XXXX. with clinic are PK disease

evaluating Our being lead proof-of-concept over currently studied Phase molecule study is in also a the APOLX-mediated program, safety, and the in proteinuria reduction X FSGS of PK the VX-XXX weeks. XX

this We study have XXXX. in expect to from results

and the one inhibitor a for Phase In is that X, at continues disease we to have have now enter clinic Phase our that additional progress that molecule also pain kidney X. completing APOLX-mediated to NaV through have We second X.X we year. least we a this expect program, molecule

very therapy program type IND. we our This treatment cleared first X type X Lastly, pleased destroyed. diabetes, to the VX-XXX, producing cell end cell transplant last potential has cells year. the diabetes. the recently islet transform towards IND of In insulin X that of allogeneic for we for are the submitted FDA the the type are And diabetes,

which islet to cell-derived cells to replace is fully significant fully these approach of clinic, pancreatic is very it proven have Advancement precedent well-established a the stem development. therapy cell benefits. is with and program the Our transplants, of cells. islet in achievement. islet the differentiated cell-based cell enter differentiated only It clinical first pancreatic approach follows is This Vertex's VX-XXX cadaveric

is clinical we a look safety difficult-to-control the off more the initiate with year. of measures updating control. running. overview, months. the of up in arm make on getting The we over progress Stuart. assess glycemic this We're X expect trial to focused of single With And to people it X/X hand course now quickly study you forward type clinical of I'll as We to that severe is and Phase coming and this study to the which diabetes, goal trial,

Stuart Arbuckle

I'm with you performance. review commercial continued to you, pleased strong our Thank Reshma.

global growth over $X.X significant This revenues billion, EU as XXXX respectively. were of $X.X QX with reflects and launched Our billion. full KAFTRIO TRIKAFTA year U.S. we revenues and the in

now our from outside As expected, persistence XX compliance patients, will revenues. eligible now the to XXXX, our And of over we tremendous months. U.S. in to and in have turns these In progress as commercial KAFTRIO. launch date, in a our a the following over that increase the since we the all anticipate and we bringing addition TRIKAFTA. in U.S. normalize the reflected to TRIKAFTA older, high considerable revenues approval very saw in the made focus rates well U.S. in is It year fourth rates demand continued significant quarter, seen launch and the of to of we for Starting QX have nearly medicines, coming maintaining that and

to recently approved the and sNDA X rare from mutations for recently TRIKAFTA olds XX for and approvals of in age with expect mutations, was year the rare and accepted U.S. in it for the XX majority review. priority and TRIKAFTA come near-term FDA younger We growth granted groups. older patients the to

Europe, In midyear. and in community the is enthusiasm KAFTRIO around CF approval interest an expect high. We amongst

majority all been have larger where Germany Our the notably fourth uptake of quarter patients KAFTRIO initiated. have of and in where already revenues access, patients markets countries eligible of most reflect substantial the England, across

as countries, similar Denmark. reimbursement in Ireland, in focused eligible seeing are uptake and provide Scotland, reimbursement we well across where EU these agreements, the with continuing all other to remain new patients launch on as the Wales access our We medicines. secured countries to agreements including We completing have

KAFTRIO. that in as the majority case We U.S., in EU was confidence patients the have be will of with vast high the CF ultimately, treated the

destination while get different. However, the journey will the to there be same, is the

to rate and uncertainties COVID-XX time also presents medicines. secure pandemic the of for the uptake significant agreements, required to addition In our reimbursement the new work around

expanded our country fully and in attend from dynamic this the not medicines closely. and patients half is virtual consultations patients pandemic treating registries agreements other in mutations, EU we we amidst in-person patients. physicians, We opportunity, U.S., the evolving launch, EU, growth. remaining continued the and around Canada around these age recently conducting XX the toward the uptake approvals XX,XXX the through now to have what's regions franchise as XX,XXX continues, patients older, ability one-third I are to making than Vertex's expansions. countries, groups with could and these other and in Australia. Europe, monitoring accumulation of of are now additional COVID reach The are a how two-thirds living these in with Today, level and Approximately lockdowns. Notably, such market the and in of we and and and you patients are estimate of or Canada. are reimbursement benefit with as outline CF are KAFTRIO patients progress new to these today. of data will through updated other next Australia who based are launches We and future expect reaching leaves The lower treated through who of and more are CF we in on reimbursement their an shared label an of world. about We there view think CF that sources patients from

we a quarter medicines transformative take in the work guidance. also like would Finally, together their community recognize Charlie at the year. to And to around now I and global to for partnership the full past the the Vertex, I'd results challenges to CF world. to bring and thank our dedication, unprecedented like and both despite fourth internationally, will unwavering year as for U.S. of CF review moment patients teams financial their the

Charlie Wagner

revenues product $X.X to an revenues increase In than sales $X.X Stuart. XXXX, the And XX% full our total to XXXX, fourth billion, combination, year the compared of a significant of of triple continued revenues commercial XX% more $X for first bringing compared Thanks accomplishment XXXX our strong with performance. were we for of more exceptionally the note, quarter quarter team. billion. Fourth to our of billion $X was than increase billion, quarter financial a QX

included $X.XX the in U.S. revenues U.S. $XXX in outside the fourth quarter revenues million and Our billion

KAFTRIO Our and grew XX% year, by the medicines quarter following several the reimbursement of the prior over ex-U.S. last agreements the other revenues over year. completion of uptake for driven our the

expenses quarter $XXX fourth the in $X.XX fourth were XXXX. of and full $X.XX million, XXXX billion XXXX. SG&A were Our quarter And our combined expenses $XXX year compared to R&D billion, million for to compared

support CF priority of Our in investment global rapid as to expansion reflected areas our new expanding as and our full into expenses clinical targeted disease business pipeline progressing well programs. high year cost the our increased

the our for strategy, we in and growth capital to spending, of cash. $X.XX R&D XXXX. of XXXX remarkable resulted XX% compared aligned corporate R&D billion, billion, year reinvestment Our our development, $X.XX profitability, internally with innovation, both disciplined XX% revenues strategy. top billion Consistent finished and margin to With operating our operating and in a Net priority for compared of combined with substantial an growth with $X.XX was billion deployment income continued revenue business programs our is in in in externally income with through XXXX. increase $X.X

Moderna. and in example, with important executed Skyhawk Therapeutics agreements XXXX, For we collaboration Affinia,

through of The and it to with shares share nearly over $XXX proved effective, million also X.X this our shares very purpose we dilution Additionally, primary ongoing program of Vertex cash is to program. acquire repurchase offset used repurchased. million

guidance. to Now

uptake Our full reflected U.S., access. key countries well considerable nearly KAFTRIO achieved as of XXXX EU of patients we performance in patients as in uptake have penetration eligible TRIKAFTA as where strong the

TRIKAFTA we In variables population X our in XX are significant $X.X the of we in we where our will revenues approved products This to midpoint. are reflecting there product growth fewer revenue at year-old XX% $X.X project that billion, guidance, regions reimbursed, in XXXX, the compared to for the total an approval XXXX plus reflects currently billion For guidance achieve expectation for XXXX. U.S. mid-XXXX. to expectations of

and U.S. related continuing is ongoing important factor the saw into patient This uncertainty dynamic rates with of of the maintained compliance. XXXX. in to persistence throughout is these to expect and guidance persistence We normalize high and as rates XXXX, factored compliance an TRIKAFTA However, pandemic. throughout is our we levels

other waves we reimbursement geographies. expect international and moving see agreements As of in as as Stuart ex-U.S. contribution forward, subsequent the further EU revenues launches to mentioned, to with well

is agreements However, they the therefore are of timing guidance. not our and these in included not predictable XXXX

billion to non-GAAP OpEx, to $X.X of guiding are we $X.XX For a range billion.

further generate continue to in key by largely to in driven allocating with order proof-of-concept our pipeline advance expect data programs programs We into to XX% greater OpEx XXXX. year-over-year investment the in and of important R&D growth, than clinic

closing, to continuing for Vertex business, non-GAAP tax our significant we of rate and a this look year. XXXX XXXX successful Our XX%, was we guiding XXXX. for range XX% are growth tremendously our XX% and was to to profitability and year In a forward and in

Now, for back to Reshma a few closing remarks.

Dr. Reshma Kewalramani

And was with medicines proven performance reflected in Throughout progress. breakthrough resilience and the past year, creating business yet an these lives. has and transforming Vertex the world. face grueling the these our and Thanks, science not out Charlie. pandemic unprecedented have was XXXX year way this and for look lead optimism. the has the of I challenges, in our of have entire that and we forward demonstrated pipeline will record subsided, challenges a while confidence of Vertex track

small to now ambition cell therapies an to call to Thanks. are diseases, that. do open genetic transform many of and and our pipeline And more poised the molecules, we'll have just We questions.

Operator

question Instructions] the [Operator first of Kasimov line you. Morgan. from Cory Thank with J.P. Our comes

Cory Kasimov

Hey. Good taking you. my guys. them Thank afternoon, questions. of you Two for for

provide conviction of enrollment? relative And on into to what good give -- of On still news database? you. next front, something terms serum looks a is levels development? here situation that would premature news you're the at the safety this you Thank taking on safety or characterize would stage on confidence AAT And program, going you regular the then, are also in where in is the no and has the added IDMC program, this the that as been XXX functional what's where AAT, you of

Dr. Reshma Kewalramani

is Cory, Hey this Reshma.

all of me do. program honestly, you. perspective, followed those is part Let our take is what safety both of that very questions indeed do and we carefully, for The AATD programs, from we as a just of

and regard Phase this maybe ongoing. looking really With we VX-XXX speaking, is study, are levels three X proof-of-concept from what for So, that to really broadly things.

AATD. This is that time to is patients the VX-XXX first is going with The safety. first

is exposure. key increases could to always to say, the functional third the levels. the also the mechanism easier see PK looking AAT a The effect be is target that lung divide relationship. is And here, remember, second and functional going the what And on we tells if parts. that is this chemistry. I us say, loop that easy, is be The our as I'm liver Vertex of effect, the That's the Pouring the cracking to this both and see there easier. AAT is close mechanism got dose increase biology, we in if but we cracking the time study, on to levels, first is levels chemistry two biology. to we in two not dose It a drug that raise that of parts, we AAT the it. pouring development at into it's on projected is the see

safety, for what assessment elevations functional that's PK we're and we're looking of so, really looking levels. of And is for AAT

Operator

you. Thank

Our line Sachs. Goldman with next the question of from Richter Salveen comes

Salveen Richter

size I you of it's can there to is areas focus, technologies kind an your of look there? the you and you fill much of R&D, deals, strategy, have So, or toolbox to accounted then, the pandemic? CF related for? expected one not what the question Could and guidance. are this for about year uncertainty you made And secondly, be a on recognize clarity you're while with but that to on just aligned any do here comment could interest accounting us regard with give how you comment time, approvals as agreements at reimbursement in And your same how BD of

Dr. Reshma Kewalramani

start little comment part then bit I'm and with launch the U.S. of Sure. going question, don't in a Salveen, and bit EU Stuart more to to off little us the I the a ask on second more just dynamics your this the is Reshma. about Why

cover will question that think your I CF. on

talking to interest BD, is here for So, Salveen, strategy really many as years, is we're the now. an that probably same eight with the regard last years the we've laid about what exactly out

complement diligent. efforts very, are our know, assets our strategy in internal fit interested We in strategy. our assets you interested that very We're R&D is R&D And in CF. that

toolkit. these markets. a over diseases continue causal having well. Stuart, market. us and to to I a kidney We and about Skyhawk tools human disease. and understanding funnel, doing talk Affinia human kind see certain of goes Disease, don't that a fall and causal diseases expect you the are falls recent great out okay, turn a be APOLX-mediated like Semma in example. to things that then, but about you J.P. little through fall We've at of I'm would that if today those there of one fit back of R&D fall polycystic fit showed all specialty clearance biomarkers our of criteria, good like with transformative transacted the that. us biology those thinking falls in and our examples also and fit disease, kidney criteria, should that looking we biology fit that. or the in Ribometrix CF potential, can acquisitions our like CF very serious tell think you potential. slide like markets fit criteria we're and on you're for And -- and you going given it they that diseases We've will, and lot have of if other IND, or biomarkers to It our to the has what transacted be to transactions that our about areas. Morgan tools, A And strategy Huntington's would what to translate out, specialty think the AATD Semma transformative It's is a glove bit in. about, like And Clearly because are that

Stuart Arbuckle

Reshma. Thanks, Yes.

and guidance what's into into about break I'm So, going U.S. Salveen, growth what are. and just talk ex-U.S. to down the of our drivers incorporated

TRIKAFTA XXXX. you U.S., as been have eligible know, vast U.S. the all groups the in initiated across during of on the in of patient the course patients majority So,

to incorporated so, degree been annualized of by we offset during the which in of the normalize the effect is kind having course our And compliance, those patients expect guidance to some initiated, persistence of XXXX. and

terms the the In midyear. we growth X for most happen TRIKAFTA U.S., XX is expected which probably notable approval the for to the of expect year in drivers to of olds,

ex-U.S. to Moving

strongly. have We triple like where access, more initiated do KAFTRIO those we have and have launch guidance, markets, do in only UK our But, where reimbursement can And combination those included obviously already where launched early anticipate, markets like actually the get have obviously, in in have the very the initiating and patients Ireland been of We reimbursement we patients countries gone majority and or the Germany. in markets those agreements, Denmark has countries. agreements. already we pricing you

for reimbursement be Australia to Canada. as continuing your have those, we also and and the Spain and is EU, last the well. EU, France, seeking both those countries approval we the are which is Italy. agreements. it's are remarks, part reimbursement, our pricing I impossible the to are been seeking reach are reason, in major one sort the exactly include filed of we kind of And to don't timing if for Outside parallel where those not the In obviously, seek his us else guidance. long Again, just guidance. of regulatory approval within longer included journey to Charlie of that on. can when XXXX then prepared So, because then, our where we've reimbursement a question, year term of of obviously, the in we in turn in for that countries them said would were as Obviously, will major in like we predict And and

than be our going if to goal, time. we we be the feel long modulators, CFTR up ever our confident triple with get been there and confident, That to to over XX% to treat for of time. continue that that very we're to has over not believe combination, more like able time We still and patients goal will a continues

Operator

next of comes Cowen. Nadeau Phil the from question Your with line

Phil Nadeau

-- AAT and the come noted further the past, an follow-up of with the a difference VX-XXX in a had the I molecule could effect and idiosyncratic could exposures effect was has In of was And VX-XXX. maybe also any of extension understanding curious to I that. on or the or of up tripped and to team issue whether class what be AATD. exposures a way, be patients that protein volunteers a any you if overcome? In that how liver? between had healthy because between lot aggregating there's so, sync

Dr. Reshma Kewalramani

Sure. This is Reshma.

those Let for questions me answer you.

way think, following. the the I the AATD best to is think program about

the We who the very about learn patients the not the and are it's were enrolled one trial. to And the There's was fully we was right enthusiastic holds we because because and simply all not both, potential more much the early, enroll didn't molecule. mechanism, only treat didn't XXX terminated that study complete it, lung. unsurprisingly, liver can there.

We looking is VX-XXX study forward those and to enrolling. The results. are

to need be but little bit patients, not that longer we patient, more now. it's dosing and a much are We

half this this results of half the have will -- first We XXXX.

Operator

Thank you.

Our next from the with line question of Yee Michael Jefferies. comes

Michael Yee

to of financial out. quarter great along Can maybe feel to forward And where then, can of which $X the about market you much size sizes guys. deal. advancing a whole balance comment think corrector Thank strategically really and and Hey, or second a but also billion, made on the congrats had and the comment where now, still that's two bigger remind are about those there. where things Reshma CF was what just the a different billion? potentiator. or late-stage Can billion, of just if super well? $XX resource nice there other was of gamut different team, once-daily year and a is those a on BD, capacity. doing comfortable side people. Vertex late-stage One had is different certainly, can great just moving the you Thanks, you. you're Maybe know levels a close and range span maybe that that, and questions. transport sizes be, I you mid cap and much and $XX us can how cycle I sheet you and high more chloride cash, to that doing question, mid about are, of whether and Vertex's mean a company there's

Dr. Reshma Kewalramani

Hi Michael, it's Reshma.

me a about question talk and I'll first, little Let more take then come back second the bit around and BD.

VX-XXX. you're And right. potentiator once-a-day the is So,

team We work. with at Altshuler, have Diego the busy been San the been work of have correctors. in David at development more busy

We have the combination also it, X that's VX-XXX, agency asked And let's potentiator; of way its development. next-gen once-a-day you'll corrector; Phase call the with through monotherapy us teza, do made the remember, a to that's and VX-XXX, a study VX-XXX.

give interactions. All the all we planning on that's regulatory as looking to now we'll you been that forward really And completed. program. And is update what doing soon of certainly our we're of an at all data, as get through of those,

development high no it be that's CF before completely going a is come level, different forward anymore. because than going drug placebo at is in remember, anything Just to there

steps. through program on But, with all And that's don't from and I'm hear next this so, the progressed. surprised to you conversations we're ready don't be pleased until are me how you very concrete if we give of

BD. let's Okay, tackle

try a in take it bit a and put the together. way this today a and walk bit to of and here's where little me then When allocation we're texture we are minutes to you color terms BD, capital and all couple really through Let I more of you of business going, in give just a behind where and think detail. about little

January years clinic Company so were the ago, the active in to We at look with back you If three patients. XXXX. programs in

pain if we pain. in That's and volunteers, Fast it. I'm That's diabetes CF just right? are and three which going patients, forward in type healthy patient-based years, six X the we count into at So, where areas the seven. studies, if count is now molecule counting quickly, looking going in molecules we're that's be I disease program, not healthy And in very patients right volunteers, to now I were. --

a are I think about in closed different. $X the closed little bit we bit revenues billion. $X we think it's billion. January, if XXXX very it's $X And we If something than again, about think had of out previous revenues, I Where So close of billion. to I north a XXXX cash of out cash, north $X billion, with little today, year, more

notions for sheet Phase want have place Phase in assets it's no allows be at that R&D assets. to place. way about really the indicating X assets might to preconceived about at, going timing to a -- tools the to to of fit are late-stage preconceived has be transaction. I I'm a It look looking I dollar about notions have for fit not at. no in and and assets, the and It amount mid just different X, of our of the the transformative. to that has those of going continue We're previously. are at the able transaction we And a toolkit. strategy We think example, our strategy. look clear R&D also we're I and a that outlined us look indeed the sheet. strategy, that's balance to are strength exactly balance our focus is I different that be And what to if I very in assets And that the that same now on

judgment we be when find to able and we're ready And, that, thoughtful very be evaluate. to going We asset, to to have have the and we the value. patience that to be add about

Operator

of Geoff Bank next Meacham comes question with from America. of Our the line

Geoff Meacham

Thank a but specialized fair future of go busy I or it drug been spirit lead similar, that of outlook? somewhat CF your when on know, have the the you've rare orphan that the have is to been could bigger by updated data essentially company, front, then, helped you. you of share indications pipeline BD at a the Reshma, view helped strategy, is Stuart, second two CF guys you guys ones. Can I updated what reflected assumptions market And path? your to obviously in maintained after be reluctant down recently And you this larger patients main look but yourself say and -- you whether that on the or or efficacy? you disease XXXX having question, BD same you could epidemiology. focus. an company, address just you as transformational label Charlie

Dr. Reshma Kewalramani

then to comment on your and ask epidemiology question. back going for to CF come second first, Stuart I'll I'm

Stuart Arbuckle

Geoff, for question. the Thank thanks

last noted CF we from XX,XXX epidemiology our in CF recently and that living Canada, regions And drove various XX,XXX. really years, that the regions. indeed the countries of the and reason on in And increased. ground epidemiology all improvements it our on the everybody, over the review you and or had the data in data. quality new registries those as We obvious patients registries these Europe in updated was So, just to latest of was us establishment to with capture change in to few review, Australia periodically view and that that U.S., some

ask, but in have increase asked any country, is, that phenomenon. really answer It question It we Just one one any concentrated you to one the region? board been didn't a wasn't. was that was consistently an really across

in And and you the often also really something was in as uncommon see you that factor here. we of rare number have increases is medicines, patients, that's and great diseases transformative a believe well not eligible orphan when

In already terms XXXX -- is number is. And using Canada, being the it that EU. the modulators That's those in treated with U.S., are are whether the guidance. our patients now. our epidemiology denominator of that new long-term our CFTR a in Absolutely, Australia included we and indeed, of

Dr. Reshma Kewalramani

really orphan drugs. and the Geoff, you question disease glad about Hey asked it's rare Reshma. I'm

is is the don't acquisition. are rare that our give markets. So, I company. And like the you I calling that Semma example we company disease about aren't of diabetes company can is us type know a disease What a and X rare we reason specialty because you a best

So, from So, away disease a the rare key the transformative. being the diabetes, type there that X right? to acquisition and therapy million And well far people, when that type it's from suffer X diabetes. think holds X you cell-based over be Semma the about potential is

that very diabetics group. you're treated the correct, specialty that for. looking endocrinologists, is So, a key are And we type are X a by

disease, rare best markets. X diabetes So but type the not really, And us for specialty is example. the key is

Operator

comes next Bansal with the Citigroup. Our of question from line Mohit

Mohit Bansal

in One for markets, of look help could markets. likes numbers Stuart, for But, if me CF you new bit Latin that a you beyond did America questions. Couple understand far these as even about you when these little of prevalence developed or talk going -- is prevalence is the it's think you understand population And, incidence the at basis, not about side. there lower Do outside this expansion I core markets as second still part zero. lower R&D is China, but and on point? of opportunities a these the on more

correctly, proteinuria you if or at Because patients, urine I are for these help thinking X understand creatinine they you sometimes? trial, reduction start protein of there? understand us how at As you mentioned, about to XX so can ratio in VX-XXX meaningful

Dr. Reshma Kewalramani

I'll Sure, do want And sure. start? XXX. you Stuart, take then, to

Stuart Arbuckle

of of Mohit, Yes. XX,XXX patients other an the are that we And we Indeed, affiliate the robust, have and reimbursement markets, epidemiology The and absolutely, our we Canada, other much it's Australia. and to countries patients right. disease less is for serve. there countries, much established we U.S. the the than the living process could other higher some of to in mature, that down like the specific much and medicines and in it continue securing we much we so the are terms these Brazil. more less that there. is EU, But, in CF of with established longstanding with in believe there in The some are issue really do registries. in less U.S., prevalence countries confidence have much where EU is in you're

those really, epidemiology. but core a see ones to the markets, good we where of handle have outside the opportunity patients serve really on So, we are to those continue

Dr. Reshma Kewalramani

question we proof-of-concept Phase segmental are glomerulosclerosis. And X your molecule the Mohit, small to FSGS, with for in about studying VX-XXX, focal that everyone, regard remind to that's

more disease And proteinuria, programs, double-digit all effect. a so of reduction. me, progress would is a It's range, we is to X And than unrelenting in transformative right. the or that protein the dialysis heavy that to they onto to usually for happens these unfortunately what You're We're as for are be grams. either patients here nephrotic looking transplantation.

something to regard, PK; with AATD patients; for are this into first looking if for As said, and what study, the regard you're safety, we proteinuria. from in that a everyone see question time I this from going X looking decrease the remind Phase study the would going we for. in to be that be expect to I'll results year. And double-digit that I'm great proteinuria do and

Operator

of Our Matteis Paul next Stifel. question comes line with from the

Paul Matteis

couple follow-ups. A of quick

the that know take Are of side, guys especially there in if there hand have BD you? your don't you of to potential has bunch editing, or mRNA, the around a gene I hands any indications been lot platform you interested step different And pretreated On you're have in. and much. Reshma. how quick tell feedback heavily and was severity patients then, of study? population part a follow-up, on you the in Thanks depends on been this standard anything one We you a interesting discussion tools. a in are therapy, disease your But, did of in can by the calls you modalities proteinuria areas care. you're so back, the that how APOLX, data interpret few about enrolling doc they've us the Is the there

Dr. Reshma Kewalramani

some some be But say, is Yes. background treatments And With including patients often are these when that is to effect, are you not can durability steroids. There treatment there. sometimes the particularly effective. with there as we're placed the even is see patients you patients these can proteinuria. that on, -- unfortunately, not enrolling, regard heavy

are studying tool heavy when question to transforming about committed and not key to for the are what diseases. about what be proteinurics, and that's let looking limiting. diseases, you our regard do is we to And your So, transforming toolkit tools we're the With at. really we're have? We

a that molecules, of example. can protium know impacted by small minority for be You small the

mRNA to get so, are we're to going David and give make disease our we you a if done ask a we've on, the target collaborations And molecules, we there of and want And the can't with you tools. we're of comments? are one going some small to you we on gene couple of by modalities. going I'm certainly words to to therapies give a editing where get of that working there other with Altshuler do David, of sense couple a just

Dr. David Altshuler

Sure. Thanks, Reshma.

opens diseases. And to potential, those a aiming through the where therapies biology therapy. collaboration a we a role we for those either that say, target But, Skyhawk get as select strategy transformational of example, partnerships invent needed. you CFTR from time to continue As or or target tools as the with and we that our partnership, molecule disease. in should technology the degradation the we see and find including with continue is or said, you on. benefit think collaborated of case Moderna, right last XX% In who I causal a as mRNA there's with with approach. In the us companies, technology, goes as other and people protein drugging cases, to approach deals are small a to the our collaborations a adding, we'll won't and modulators, multiple protein which our then, you don't mentioned, through will of and the playing disease our And make identified we CF, at Kymera we've validated underlying do starts Ribometrix for do for think technologies we'll such that way particular RNA, up has as with

Operator

you. Thank

Cantor. Our line of next question the from with comes Alethia Young

Alethia Young

And a about in in clinical talk kind of on transplant guess, to designs, your on Hey my plans just Thanks. taking for bit progress. bit you COVID little IND. that of VX-XXX talk I plan guys. the population? kind this, questions Thanks navigating or you Can want a of all trial and Congrats light more how I little about

Dr. Reshma Kewalramani

a patients, clinical I out It's about where the therapy patients, is, product sickle to pretty AXc, are our the would in remarkable Sure. The CTXXXX I C-peptide are be that Hey in think for cell-based no measures It's therapy -- being into performance I'm able the see about very the This being and about very can our and a much and Alethia, what goes program we are so looking of brink approach step. seriously outcomes cell trial. therapy glucose, that the trial healthy volunteer very VX-XXX approach single-arm like of X reasonable results. diabetes. and straightforward, beta-thal And to the of cell type now hemoglobin a for is is to tell actually would at, we thanks really level. an This going point at CRISPR that number we question. the able enthusiastic a the directly on therapy say we're is program. that a going to this think disease patients. There disease. to

program And so, and the I progress. of special, this this I'm very think very something see brink to eager is

Alethia Young

that? from impacts or be is to to navigate straightforward pretty you Any do COVID, think, going it

Dr. Reshma Kewalramani

that in is of sure. and is But, I for is is in COVID top with lot said, approach done of something of specialized Yes. this centers, challenge us see it, don't and a way, vaccines and are if having our pandemic that coming, all trials that continues being an are mind for clinical work, how so to this pretty hospitals to which the COVID able good do to clinical trial looks part have say people continue is manage, to learned that to me.

Operator

of line Our with Wang Gena next from Barclays. the question comes

Gena Wang

follow regarding also Thank you question. for XXX. will taking my I question Alethia's

step next the particular question completion trial for for glucose CTXXXX us second And lead currently anticipating trial the that's Reshma, enrollment the for this to will -- you with my benefit -- the can that patients complete benefit XXXX, is or beta-thalassemia share achieve sickle wondering, step? program? each goal be the Just trial any that to with cell, say you --the anything want XX regarding for could next registration

Dr. Reshma Kewalramani

two are CTXXXX about type about X one one diabetes. There and questions, Okay.

the first, X type come back take CTXXXX we'll Let me question and diabetes. then around to

CTXXXX, remarkable, but you with So, correct. are really

to looking constitute in that will cell are I looking and that trials, trials disease, be the anticipate patients the that studying we're to in complete do both patients ongoing the that package. are these sickle We And enrollment beta-thalassemia do and we're XXXX. in regulatory yes, that

before, about yet us heard have not talk you've we've we initiated conversations, them. those but As completed

be do our of composition these So, so year. expect to forward looking doing the patients we're package. to yes, I filing But this

things program going are producing cell On is So islet patients. differentiated, these and insulin cells derived, to stem This first the VX-XXX looking first are we what time that fully for? first. the

C-peptide, C-peptide excited It's indication trials bringing brink clinical able too to there safety. the really what glucose, So, exactly terms in an urgency. hemoglobin for down the is And get of looking results looking I that what outcome blood early straightforward, obviously we're to with the for call get and that impact And see and We're is drive and to an insulin. a enrolling fairly cells up the sugar evaluating are these looking to getting am be and on our very is trial we're levels we're see here measures to what sites AXc. We're really will being little But, running. to such. of patients dosed.

Operator

next of Skorney Our Baird. Brian question comes from with the line

Brian Skorney

of diabetes of the comes understand type any process or transplantation. sticking was how transportation sort to understand of you -- wondering into program, derivation cell mitigate from I X walk through cell immunosuppressive stem engineering of the on kind maybe islets cells, kind try to Also can I plan help how your effects? Thanks. that historical differs transplanted if sort it that us and the you goes ultimately program from but the gets of us

Dr. Reshma Kewalramani

hearing enthusiasm over. brimming Yes. What I so my for be must just question. thanks you again, a asking it. great much And guess,

I separate think two So, diabetes way would the programs. I is program type them the would X about really think of as programs, two and

comfortable X disease had who maybe bring that there type reason with benefits would does XX,XXX I maybe renal that that That are would end-stage one just to XX,XXX, naked could expect who diabetes, immunosuppressive people for very anyway, the and are and cell the are immunosuppression. one XX,XXX XX,XXX way from one, then, naked because program. the would the who transplant diabetes for there require cell And them. people on which a the and The brittle XX,XXX the is program. have for first their I call type so people of diabetes get another IND program that or cleared, X have benefit let's with maybe have cell be therapy that renal immunosuppression

is the course, here of high plus Now, cell program. device the fruit

insulin prior of into The to to about so that the Vertex important the thing is are designed to oxygen flow and team program, avoid that reasons second is excited right were flow, team acquisition, we immune using Semma a the pitfalls in to For to allowing we one the glucose that there not they late know and coming of therapy. allowing of material, Semma fibrosis, preclinical and right the geometry the understanding really cells but development. attack clear the allowing to cell-based how the

particularly is exciting that not program require does immunosuppressives. that because So,

making you we we'll development certainly is progress updated, preclinical through keep As preclinical that now. way make program, its that development, right late but on

Operator

from of with last Evercore the Our Liisa question Bayko line comes ISI.

Liisa Bayko

inventory. transplantation just a on very of then, on I'm questions quite you recent is different and much. the pancreatic ask you VX-XXX? Can jump launch today? going note. understand with durable. And cell how be I like islet anything a TRIKAFTA or question Europe, to little any this might What of there. you maybe a say, to And Hi about a then, durability, sort bandwagon it's more think inventory Were on bit curable just speak to the Thank there final that build in let's note? given couple

Dr. Reshma Kewalramani

comment Well, with Charlie inventory. on I'll diabetes that Reshma. then to and on ask and is start type keep This there. I'll X theme, Hey

Okay.

islets right. our been pancreas what's So, or cell-based the think we with transplant. excellent All done is question it and that is asked you about therapy an one, how should vis-à-vis transplant cell

exactly a is causes type The know. what most to important, way, to X important We this. things key four or most the far, three maybe So, in autoimmune by by thing it know benefit. It's transplants, that when cell islet replace can cells, islets cells. you of you or whole get be diabetes. we pancreas the And, those destruction pancreatic know know transplant

industrialized a they the make cells made, exciting so that differentiated to and real the producing seminal that insulin quantities. that here to the those Semma two And the this the fully that work. quantity developed quality not and team the of of differentiated, way was know Well, cells That we itself. is know the will know essence problem, and we problem take work. is into discovery actually We will islet us So, problem? is that things. don't What's in it islet stem

transfuse, so, -- quality of patients we cells to quantity with type X transplant to have diabetes. is And to what and give into the now

So, to ask address. so to I'm going that's real to really program us. to inventory excitement what you has the that this why Charlie, discovery question and is exciting been from that here an

Charlie Wagner

Yes. opportunity organization. our actually to chain Thanks. supply And this colleagues a I'm going manufacturing plug to and the to give in take

about most the and pandemic I have have ensure that that in can to that noteworthy to worked in ready teams think, I that we is inventory Europe the make flawlessly for launches KAFTRIO throughout we launch inventory to the for those our be we'll for XXXX. future comment available ensure were ensure ready patients

I inventory I your of to is you in or on there was XXXX tell think in quarter about course the fourth our revenue. impact specifically related the could guidance that levels. question noteworthy nothing

Operator

closing Partridge Thank now you. the This to concludes question-and-answer I turn session. will today's call back Michael for remarks.

Michael Partridge

operator. Thanks,

hour So, one the mark. at we're

get the and who queue didn't And, in other your the very tonight. We but is question, take for questions. to Relations conclude a folks I will call the there connecting here. in are the Investor happy you tonight team office thank know much

Operator

for your gentlemen, and Ladies participation. Thank you call. concludes this conference today's

You may now disconnect.

Vertex Pharmaceuticals (VRTX) 2 Feb 212020 Q4 Earnings call transcript