Vertex Pharmaceuticals (VRTX) 30 Oct 192019 Q3 Earnings call transcript

Company Profile

Participants

Michael Partridge	SVP, IR
Jeff Leiden	Chairman, CEO & President
Stuart Arbuckle	EVP & CCO
Reshma Kewalramani	EVP, Global Medicines Development & Medical Affairs and CMO
Charles Wagner	EVP & CFO
Salveen Richter	Goldman Sachs
Michael Yee	Jefferies
Alethia Young	Cantor Fitzgerald
Cory Kasimov	JPMorgan
Paul Matteis	Stifel
Phil Nadeau	Cowen & Company
Matthew Harrison	Morgan Stanley
Brian Abrahams	RBC Capital Markets
Liisa Bayko	JMP Securities
Geoff Porges	SVB Leerink
Whitney Ijem	Guggenheim

Call transcript

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Michael Partridge

Good evening, this is Michael Partridge, Senior Vice President of Investor Relations. Tonight, we will review with you Vertex's business progress and provide our Third Quarter Financial Results. Making prepared remarks on the call tonight, we have Dr. Jeff Leiden, Chairman and CEO; Stuart Arbuckle, Chief Commercial Officer; Dr. Reshma Kewalramani, Chief Medical 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

subject on We limitation forward-looking today's those including and press filings discussed this Exchange to Vertex's our in regimens the and other regarding continuing the development and and call of for in will detail Vertex's risks statements cystic without that with performance make release CF Commission. combination fibrosis, outcomes uncertainties and and financial management's statements, medicines, are the future materially. Securities events marketed on could current programs, actual our Vertex's commercialization based differ triple are assumptions, These

I to now will the call turn over Leiden. Dr. Jeff

Jeff Leiden

everyone. progress scientific has of create strategy of XXXX year transformational execute continue parts all differentiated by evening, for serial across business, our investing a significant innovation. as our we to medicines on to been Michael. Thanks, and Vertex clear Good in

revenues in in medicines. And people both progress innovation other transformative to future create growth advance continued our with to in diseases. internal are multiple both and additional significant making in pipeline enabled has from medicines across we rapid investment Our CF efforts globally and external CF more our serious potentially treating

Our in you with some strategy company XXXX the come. is growth pleased working accomplishments. to review of and for and to continued has positioned recent I'm for years our

the eight for right future with than week's of that ago, disease. toward approval every CF to journey the say First, Trikafta, I'm as approximately than joined in significant Vertex this last FDA first date medicines treat XX-year of small to our patients patients more of CF for to person goal XX CEO, of four in I around more proud XX,XXX approval for CF. years our Today, worldwide curing and before in step patients most for the our we marks thousands world. we're a cause the eligible are one countries to US. our for received group of CF KALYDECO treating

possible again would been as CF well as patients, patients to like value appropriately our CF Scotland. medicines Trikafta to benefit with cause our caregivers, and over collaboratively like the approval of The this Most of new significant and were would to who've that that the the decades. journey pleased recently have physicians reach to eligible us support treat families, two notably, I thank first the countries for were we've governments able of now the the the the XXXX medicines. reimbursement their not Foundation and agreements entire on past in have been CF time medicine and a the community. for I'm of announced agreements without in unwavering scientific work also would progress made reimbursement throughout in ORKAMBI we Australia, I Spain, securing SYMKEVI that we innovation outside highlight these and US. England, underlying the with clinical will advocates to

development clinical we diseases. Now significant different a of five programs. spanning potentially And for and generation period progression in our development transformative pipeline. Today, entering multiple medicines we're data to these have specialty clinical

XXXX of of data the studies, risk-lowering readouts expect proof-of-concept for key represent concept pipeline. proof we fact, and our through important In multiple initiation will events that

focused clinic. risk on success highly lower to early models, in predictive have in causal investments and biomarkers biology, followed increase our building developing preclinical several a for pipeline the Our probability years strategy disciplined and of development

on transformative serious only diseases shareholders. we significant sustain and that work society, medicines and our for create value patients, And for

therapeutic the and strategy multiple modalities. with Our external investments are aligned encompass

breakthrough that line and in medicines will building in of new cash a to such tool by programs and development our external our technologies kit Type hemoglobinopathies, develop dystrophy, X flow increased diabetes, muscular growing Duchenne investment diseases have new others. We with enable as acquiring us

a potential both acquisition of recently X with goal a cure therapy cellular diabetes. device Therapeutics that the to has is in Semma promising alone we R&D our aligned Type with and acquired This provide our bring development and Most developing an in implantable perfect CF. our beyond significant for internal efforts of programs example to complement opportunities growth a combination further strategy with are that efforts

expand medicines our to innovation and near-term new medicines, access on and The in has strategies gain forward to updating as worldwide well for we'll of and profile further approvals the growth continue are look Stuart reinvestment call both to now talk and over commercial in and to revenue months to corporate I about differentiated research we for earnings execution we over long-term detail highly our produced growth, continued the a for of the for to and create launch performance coming future you our more medicines. Trikafta. and progress our positioned turn

Stuart Arbuckle

launch third ongoing Thanks, the Trikafta the commercial our discuss expectations quarter Jeff. for and Tonight, our US. briefly the I'll performance in of for review

third increase a medicines. XXXX. and and increase notably globally as older. quarter quarter with from Most Uptake multiple multiple across result revenues products XX million, populations XX% the over past in year. This is the of product treating growth Our of were $XXX third ages Symkevi our patients Symdeko drove to revenue total and a patients in compared more

XX the of We expansion revenue to including result into patients Symdeko. Orkambi X for to a ages in children younger also X ages US, years years for as and growth saw X

Australia. Spain, underscores our continue progress as the progress make Scotland, can be our governments work positive outcomes toward regarding the to When CF announcements patients. access by Outside providing evidenced flexibly Orkambi we for in collaboratively and that medicines and these achieved. in our recent and Symkevi, we and countries for reimbursement Together, achieving US, in reimbursement England,

fourth cause and was cause a Representing CF. with underlying those treatment FXXXdel their cause most have approval CF of age are Trikafta in for and at XX,XXX the of largest report that for those patients of broad treats population are XX,XXX of the time older in approval this medicines the Trikafta, already and the not patients patients remaining the approximately medicine years of eligible data, XX are to eligible or Symdeko. XXXX have patients a CF with at one medicine in the is least eligible approximately but community's the not the patients or one Trikafta last patients of the Trikafta Orkambi, far of Trikafta least that disease. one The Vertex's treat mutation medicine. shared that to one the The years minimal FDA treated first by our patients to of these reflection the currently there one Now the been one underscoring to provide have mutation. already and with physicians, patients treat I the strong urgency of and by We with the people CF with by at are who for the medicines for estimate mutation, approximately speed of the is US, interest the launch. pleased CF now function is ages underlying underlying Of mutation week till only The launch with our XX,XXX prescribed And of approved strength who fit of their FDA US. XX for am Kalydeco, the the any their also to criteria. Trikafta label who in FXXXdel eligible. older, is were of being FXXXdel CF US

X,XXX XXXX the expect will new minimal new Revenue a of will treated treatment minimal growth patients by and overtime in primarily vast be with be majority we driven of function Trikafta. patients function

of the constraints of proportion or patients eligible Orkambi, that with such actually why CF Symdeko peak centers Trikafta factors Trikafta We take launches. reach The important over initiate and its currently coupled a to significant longer in number also patients for of volume schedule time. of compared uptake on launch switch expect large prior it of to may to the will a level Kalydeco, to large patients capacity are

launch additional is of Reimbursement in an the Trikafta. factor

US disease already both with of CF the begun in $X.X obtain to Based provide. the dynamics and our and CF time total on reflected payers benefits These positive. of following to been guidance discussions government from the the experience are payers expect approval feedback broad in updated We the commercial prior has payers understanding clinical the Trikafta We've reimbursement initial billion XXXX medicines. with the to medicines approval at similar modifying of week. the billion we $X.XX and data that revenue our our provided last of that strength

as as uptake early will these account early into XXXX set trends factors, next We guidance we year. for as take well also

Reshma to that now review that, continued I'm our pleased are patients with turn revenue the we more the and With In many around medicines will to pipeline recent I bringing call trajectory the of over our globe growth. progress. summary, to

Reshma Kewalramani

significance community, it's Jeff's for synthesized would of to became amazing CF part the I a like first drug that Trikafta. for years, a to story able early Vertex first elexacaftor, our both Stuart. of approval, key the corrector echo Thanks, XXXX about next it the and the Trikafta that scientists that for comments generation was think an to truly that us to And US. thank milestone. in a now getting entire I'd to support from development lab CF It's was the the of the this in development community bring little patients four remarkable under Vertex molecule to in like their and through and

globally working also and bring approvals US. Trikafta gain this Trikafta approvals patients. celebrate on the for outside more milestone, we diligently for We're to seek As track to patients are younger to additional regulatory to we

countries First in Europe, then in globally. additional

a we significant are children development and of And of enrolling Outside we also to clinical X are in progress. period Trikafta Phase years. entering XX study CF, data now generation a X ages of

we into We data X-X patients XXXX. and and this readouts studies disease efficacy of with gene editing measurements the provide partner therapy Phase beginning sickle sickle The of the beta and cell clinical year with expect first our cell studies thalassemia expect Therapeutics. treated Phase In the from the disease X beta year data progression Crispr novel this multiple X data disclose throughout and for later and safety thalassemia, multiple clinical pipeline later expect to include will we molecules to important from CTXXXX. our CTXXXX with Phase of

functional two of endpoints The approximately the Z program, oral XX deficiency, and finalized AAT expected AAT the tolerability. level for will of begin to expect study now have change the VX-XXX as doses XX our is and for small corrector have safety blood, X a mutations enroll the VX-XXX we in will proof-of-concept molecule in to study our be evaluate well placebo the and compared who to with protein first days. patients The as of quarter. AAT In primary design multiple study this Phase

study expect We in from to obtain XXXX. data the

mediated function are selective our reduce multiple developing APOLX would stage and NaVX.X our evaluate healthy a Phase inhibitors we're is in corrector of study urine. stage development. we which second in in a Phase VX-XXX, VX-XXX. later development, where to to plan complete of oral pain, VX-XXX for Phase we to also X in are molecule X XXXX, to inhibitor additional biological X AAT VX-XXX, represent our the X late for if is on initiate similar to in currently APOLX VX-XXX protein track this successful levels clinical Phase early small addition first important VX-XXX of in This advancing research. X, proof-of-concept are of programs, volunteers ability ongoing late this in our year. In including program. and through In kidney study we're the pipeline advancing research we other would proof-of-concept And multiple And an FSGS, is molecule diseases study we Phase

With year of positioned pipeline medicines Vertex. of transformative to is potentially other pipeline five launch our of a progression advancement of the the across Trikafta serious significant and and growth diseases, be XXXX multiple for

over now the I'll call to turn Charlie.

Charles Wagner

Reshma. Thanks,

our to comments quarter CF a review products performance financial on make and addition financial I'll of third results, guidance the our XXXX on few trajectory In XXXX. comments for our tonight, Stuart's financial our

guidance All will of I the results discuss and tonight non-GAAP. are

and Symdeko mentioned, quarter product compared largely in on of XXXX uptake in the we total revenues third XXXX saw to Stuart growth continued Symkevi. the double-digit As of based

R&D $XXX to SG&A $XXX XXXX. resulted million, million third of revenues the for and and third of third compared third significant income spending of A quarter quarter XXXX quarter expenses operating disciplined XX% in in Our increase $XXX to in XXXX. a the growth million quarter compared combined the were

million Net million income of the $XXX in third XXXX compared was the of XXXX. to for quarter quarter $XXX third

strong of income spending, revenue financial year-to-date similar growth. operating trends Our results disciplined show resulting exceptional and growth in

$X.XX Our through total same third quarter CF increase XXXX XXXX. revenues over XX% the period of in billion, were the a

resulting XXXX were $X.X and year-to-date billion year-to-date $X.XX in billion to $X.X Our $XXX to for XXXX, SG&A billion million expenses R&D compared XXXX. for operating combined for of income compared

ended a as globally, To collaborations. a flow create through invested in and innovation As we external and patients innovation reinvest treating billion intention future internal have more in CF to we strategy cash in the million profitability outflow that end with to external to and third of We note, in new approximately have $X.X result marketable of and Semma both XXXX, reflected cash our increase, medicines. we date and $X quarter in acquisitions clear in completed our of acquisition at billion compared our would $X.X the so balance. early the in billion however, quarter approximately quarter, yet cash $XXX cash XXXX. I Therapeutics that fourth was not securities

long-term more our flexibility and growth. XXXX beyond, additional we ahead increases cash expect to provide flow deals in continued and we fuel to for As QX look to

guidance our XXXX. trajectory Now and on financial thoughts for XXXX to high-level

revenue As the approval total revenue Trikafta new revenue existing our growth revenues XXXX the growth R&D schedule large of our and total trajectory Trikafta revenues, we reflects volume week in seek launch guidance CF know, rate. centers of anticipated tonight effective revised and XXXX treatment product we the guidance the rate US. significant reviewed continue large earlier, Trikafta. Stuart to initiate we are our exact of last SG&A tax by our launch growth dynamics to the are with Trikafta The well part primarily to specifically of capacity XXXX, XX% upward expenses, positioned likely strong in XXXX CF and over of our patients the The and guidance midpoint on in depend CF combined that of will and driven with you of reiterating for and of

acquisitions growth our significant our also will particularly With Semma. and revenue of investments in in recent Exonics expected XXXX, innovation, increase following we

the range XX% will operating current in programs preclinical and of growth higher diseases. year diabetes, is XX% in expenses be somewhat X we expectation other have the to selling rate Vertex DMD, in for the While and invest typically per therapies support research over type manufacturing that last in our of years, grown genetic few as our XXXX of in

issue which significantly growth guidance we've will increases the and revenue our expect operating XXXX continued we other few in and outpace Currently, Trikafta to launch. QX to earnings Importantly, a increases drive revenue plan in of any financial at expansion for income of seen expenses, operating margins. once operating months we our call,

the I'm coming Jeff. forward with and performance of the pleased that, continued business our will With months. look to back to updating hand over I call you

Jeff Leiden

Thanks, Charlie.

is and of the near to exceed each we the year. or and to success of often the is the part strategy our of we for our the start of we meet you As goals important outlined key place of overlooked XXXX, Vertex in the of strength at business. drive An end success the the on have on execute track team

with strategy that team is are and globally, a people from we more to of been innovation, diverse of never treating a CF have launch strong we rapidly committed and expanding a in progressing position revenues today. and stronger than our increasing the With serial Trikafta, pipeline

With that, will line questions. I the to open

Operator

our Instructions] Richter, comes Goldman first [Operator question Salveen And Sachs. from

line now is Your open.

Salveen Richter

the for uptake Thanks. we Scotland. Spain these on trajectory just the with recently any decisions quarter? and in How the that Australia regions Maybe view have starting played and and and question England should reimbursement out in implications a fourth

Stuart Arbuckle

question. Yes. thanks the is Salveen, Stuart. for This

we from been getting in to So great, complete which access these we're discovering for the of patients. reimbursement terms obviously is very productive It's thrilled. a journey new agreements, quarter as medicines

the England, firstly, each uptake, really up is I would place in they put days. to get sold the overall, I way In consideration in administrations to And as the it and that the various of would about same think in the have terms the is, they markets to say, an prescribe of the to within quoted medicines. these process allow were that physicians hoping example XX

a So there can staff we patients. initiating even is lag a bit before obviously, of

after number those that have And in be would we then of of markets. I about really, that, the there each thinking patients

markets the much rates that's on in see we markets, CFTR the the across geographies, across reimbursed, been in initiation those in will the majority over like initiate the those products, expect general, tend world modulators of the that world We vast patients time other XX% of around we XX% to across to one range.

so in As as rates we rates be you stay ever do can need when products industry, seen and XX% compliance we things in the are two the a tend the XX% so time range know, we not they in to as unfortunately compliance the XX% of everybody markets, a across in to my are medicines on somewhere state, medicines, persistence products, it's range. eventually across to rate, the function good those across reach steady see I've in are then factor XX% to

factors So, of into those would I kinds be are the taking account.

patients. tells you to is is really we're little kind really population, uptake is us a large England, of of So that patient to That's where it state, is size and X,XXX then steady if take levels to get a of bit the a by like long it's peak of number. of couple to going get things, likely how question eligible a kind somewhere about that given to take you very the one

the in centers treated XX England. are patients those of All CF in

the of probably of there actually so, those in to like And being all kind capacity biggest patients to have the eligible constraint is and just able that that's here a limiter. the centers see US, rate

patients the get time. certainly of majority is over up vast expect just ramp to likely to those So we to

late those to because work In through obviously, I've reimbursement haven't before the told 'XX, we in about you way 'XX have terms we just things start administrative our agreements our can just adjustment and actually need so initiating patients. the come year guidance of in any we included

Salveen Richter

Great, looking the dose And and about are could transition you're just what program, just could question many you then this at comment into a doses registrational thanks. how AAT trial. second a the cohorts, on and

Reshma Kewalramani

Hi, Salveen, Reshma. it's

I getting to think reasonable study the much dose-ranging cohorts or going Phase select a II XX prepared a the going study going that in think as it's study specifics this is few around people my I remarks, right and going our timeframe. said do be And and about to studies, VX-XXX you're indeed So very to like that CF see dose this in so, look very for doses the in that's from because I'll away help and our this such. is important to Phase stay be I X dose. the to feel us

respect, that in going I think important. very it's to be So

Salveen Richter

Yes.

Operator

our you. Thank Yee question from And is of your open. line next Michael comes now Jefferies,

Michael Yee

all expect thanks. of Hi on the whether the would you first, prioritize could progress Trikafta questions. Congrats the the you think patients Maybe the het/min frame expectations just centers approval. around you that of guys, Two be would Trikafta all some logical. uptake to on early and and would

So the we of to AAT functional quite a just be would for relates And seem data then on they're so How data, for much. confident sufficient to endpoint priority you that if so be even how in Reshma, that question get other they but are primary a Thanks approval. a that AAT to AAT swapping the also positive of maybe follow-up het/min's on workshop, less the are though is eight-hour from much think others. first? we I interested

Stuart Arbuckle

will and Hey, I'll talk to Reshma AAT. then take Mike, Trikafta question it's the Stuart here.

mutation, broad one approximately the in patients of label those that's to quickly, in is you thrilled know XX,XXX with US. here the so Trikafta, approval get XX,XXX, terms obviously FXXXdel as Of we the So the anybody allele know, who and are underlying have don't have their on are today. currently about you cause the a who treat a function patients, minimal patients other disease those mutation to as medicine well of X,XXX the were

one for any again, far uptake, this number largest things, CF of of by capacity had away absolute by be is is centers. and governed going terms to we've In launch. is the couple a the new This eligible ever patients at product of

in back more we big instance, as XXXX. introduced Orkambi had twice we when July than For as

a So challenge. capacity clearly, centers to of is the be going

of terms one think from center, you everything prioritizing about heard every we've just I patients, In response. center and Mike, talk to if you'll different get

most that in. treat prioritize what they that some talk you another, were get het/min's, have going severely are will another, irrespective they to will of others you'll said their as said some they the treat have to they have impacted coming mutation patients said If patients is,

over to I'd tell I are and to So function treat indeed love to AAT. centers. majority that But patients to those are going limited that over I expect time think center; process going their they Symdeko individual treated just real to to how the but you tell can to you that's to individual patients minimal as Reshma rate it's of of to they the Orkambi vast the What be tell who a expect all time. patients answer the limited and ability is one through of currently are. with though being transition centers treated, you as will those there there's those also majority of vast number be

Reshma Kewalramani

approvals on liver of the determine real their see point individual to With our with recognition work their and be X both to was of based regard Phase also sponsor, a the the the levels I and the nice to to a open-minded gravity encouraged like. that's to approach I very gotten the the they could disease, -- that acknowledged molecule that's and to the there. disease the by of are that And was interesting what one small that comments workshop AAT date I the companies trials holds It was that to and were took couple on ago, that based that unmet there. invited just regard each approach the the only data have exactly pleased to there also thought the corrector remains look going and disease. we potential with where months with With treat augmentation be them the was found the their agency need. is, lung to to place, to agency. community It high the be really Right. be AAT

Michael Yee

Okay. Thank you.

Operator

& Thank from Nadeau you. Company. And Cowen Phil our next of question comes

Your now is line open.

Stuart Arbuckle

Phil, there. you're

Operator

Phil, And Cantor of your please comes our And phone Alethia on Fitzgerald. question Young if from unmute. is mute next

now Your line open. is

Alethia Young

you I my guys, kind fits on bigger Hey thanks addressable question you medicines, taking bit like angle and and there your like And talk of kind know are guys to of, the are United Semma wanted still said some sorts of Kingdom, assume all patients you guess from to how the made. you where a I warehouse for degree. do going. world it's there, there I two, be but separately, that innovate kind a a in much of in all people, as a congrats I controls is far where you've about the progress deal as it would X,XXX, will the of think little some market and different scenario

things. should think models, any that we there there might management in to about that our is happen So kind

Jeff Leiden

has strategy with the then Yes. Thanks, a X in the diabetes endocrinologists, Stuart, Semma, relatively and type US a number out hypoglycemic hemoglobin Jeff. Alethia. our well is for that suffer of long-term just high now, off can and take rates you reminding good it's XXX by right, I'd disease, in them AXc start reach a that they, therapy turns returning specialty patients a to I'll a has multiple of for fact will saved their is very fits England around to is really normal insulin This Semma X.XX not disease are that that from the take so them insulin question regard as very the that glycemia disease million of With and Type treated levels serious you certainly it small centers cardiovascular or you about perfectly so and question. years these force X interestingly, been way know lives patients as sales normal almost from episodes. despite as

a it with strategy. patient there So large and fits unmet our need, this subset is obviously

cadaveric a that studies into can you than essentially There patients if long-term small you patients, decade. of transplanted maybe up more were with and only cures, numbers under the immunosuppression those they can some of for years successfully patients, knew patients a also of islet more cures, were who were those We cure transplant XX were not disease. islets were than these of number

diseases say were is really been as X well our I'd one he of diabetologist this interested geneticists, interest obviously we've keenly in for the with issues two some has there So as David time. Altshuler, it a Type been and watching as of diabetes, so disease.

enough the other the And the to patients the limiting was were that way can cadaveric a and problem for islets problems there islets you need so and number you of need different naked was two immunosuppression weren't potentially to reach.

those that two, addressing the watching solved companies two last And we were problems. problems years. of So were to has actually the for those convinced who both three Semma are over last six eight we months,

and levels out first -- can when shown ES islets cells even fashion, diabetes models cure those they've animal are cells figured actually they're So able industrial islets they of on iPSC they successfully differentiate them so human to and or front, and into cells that in human to how human an have a industrial the take into make transplanted.

islet device them problem and the response. of immune number also So that from which able you the then that put was to that's solution they've invented the the can is in a able, protect

does products, they that like immunosuppression. call has what transplanted really of islets is which into could second the transplanted can be device, naked a we two in up which without opens that be patients, transplant require the just liver that product, liver into And are islets can ultimately treated immunosuppression have so, cadaveric believe they have the substantially. they And we Semma the obviously the number and islets that

in a a given recent fits disease to new therapy, able pretty unmet our have these that the we we and which saw in specialty approach disease, it's need, it's disease it's that it's and breakthroughs a quickly. it's transformative of So disease perfectly, a high fit towards strategy a summary, move scientific were when perfect so a

Stuart Arbuckle

of what kind England, uptake I in Alethia, markets. expectations really all And to for said back on earlier about I go

said, for the agreement various the there's eligible genotypes the NHS. X,XXX As included across that struck you and with ranges medicines we in age various England in the and patients about are

many every persistence the the the terms If I those can uptake it's patients said markets rates and around rates medicine, the XX% at world you products ranges, the range have range. and of how around been world. XX% in not initiated somewhere again the of XX% the on are to similar look Again, in for genotypes age those across in in to stay as XX% patients patient

So if and modulator, then factor that markets X,XXX persistent mid-range be steady patients where kind be pick assumptions we just you we all if somewhere around around the state, other think things get those and on those to could of of kind you world. a be I could of about those the CFTR take of two might when

that discussed In something demand terms the Obviously for there has pent-up UK. heard of in been we've in of warehousing products. lot a that these

their people they're anticipation we've heard of wanting warehouse likely that that's the that so And could in clearly initiated. heard I about something to have patients not of to be people triple, happen. saying

heard something not UK. I've it's in say, I talked the As about that

Operator

Kasimov Cory from is question next our with JPMorgan. And you. Thank

Your line is open. now

Cory Kasimov

in questions. guys. of I'll agreement afternoon my for the France. don't have a good about one -- Thanks formal with countries, ask few Hey, taking you one yet

that should Thanks. comes the how in on through revenue are and pipeline numbers So, to regarding deal you beta patients a have recognition would type side, follow-up, able can approved patient your on you And or sickle for remind see then us of terms CF at ultimately all cell. at of many drugs you over thal that how in to could we Are data, about this the we later of point? year duration country. once on there think one CTXXXX expect of elaborate and already

Stuart Arbuckle

Cory, it's Stuart. France. I'll start on

is now, reimbursement right. don't You're of in markets a France one few the where have we place. agreement

bring we'll We conclusion, and Scotland with certainly able recently and the and are discussions Australia. hopeful have a to and Spain we as French in discussions I'm very active those authorities successful England be to in that

within and access are are approximately the You're patients who currently there's Orkambi program patients Orkambi. French XXXX are taking an Orkambi was XX patients for number in of receiving correct, plus through system, there who a available early or so France.

turn and recognition In might change, Charlie. to over that revenue terms of I'll how that

Charles Wagner

Yes. Charlie. is Cory, this

at repeat. the forward, the prior book into we through prior at contracted period price price, going non-GAAP and low a it will as which revenue, revenue will that a recording obviously value as comes agreed-upon recognition, revenue the book been for booking we intention currently, that when the revenue, we be of to catch-up equal on between we'll difference it price. revenue would And sales won't the For our relatively which we've at have France an period price been land revenue out, then revenue negotiated

Reshma Kewalramani

are. regard Cory, with cell CTXXXX and to kind thalassemia sickle and we programs, where of here's beta

half a sickle for beta are studies dozen We open sites for the open cell, have about enrolling. sites about thal, dozen a

must I heard fact to have a data think you in we this position Crispr our comment be share the at on quarter. partners will that

this levels, Phase first things. foremost efficacy think hemoglobin and other the I levels will unsurprisingly amongst interesting is be what F And and in to very on see X-X study. should safety hemoglobin side, certainly tolerability expect you

Cory Kasimov

very you much. Thank Great. Okay.

Operator

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

Your now open. line is

Paul Matteis

Great. EMA Thanks wondering as to the the with the and one Stuart, with in could validating triple discussions the the I the output AAT, it AAT or produced mechanics I important On And assay conversations you or a was top a as or of you and for one from to so AAT if and Jeff then functionality about couple questions assay, wondering bit is the your the was there much on I triple UK. agreement and using congrats if how levels the give of on talk corroborate not readout regarding by Thanks guess on efficacy secreted, reimbursement gated is the the progress. any, much. of I VX-XXX that where little whether drug. have on and you're latest the an far are the could just by approval. us so line of

Reshma Kewalramani

Reshma. is This sure. Sure,

question. assay AAT be tackle me Let

with our Slide VX-XXX treatment in the Y-axis left AAT with So levels. what a actually you've functional you'll and panel VX-XXX the animal there and seen, data shows models, the remember you to is the happens as

terms a the assay easy, a measuring the the same of animal it's reasonably in but it's do we've human it's what will studies we and like never reasonably you've assay in studies what I clinical in studies. measuring functional to animal easy simple So and seen a say in been trial, us that is be a the

Stuart Arbuckle

Orkambi it to the approved does Paul, agreement just include the on Kalydeco Yes. the UK, just and the announced agreement, the not Symkevi. its and current one triple week, indications be crystal includes current clear, combination, that last commercial we

at is but first So included. combination, medicine the the very Obviously, CF is approval ability triple to in there instance. the get of of high triple to to in UK awareness our the and governed both by community NHSE, EMA be the a in access level not going is

Operator

question from Cowen Company. next our Phil and of comes Nadeau And you. Thank

line Your open. now is

Phil Nadeau

for Good afternoon. my taking question. Thanks

Sorry on about before, think mute. was I it

think have be First centers, mentioned many on month, on patients limiting see capacity at of getting of every every patients the that quarter, a year how exactly Do sense US, times XXXX? a CF their I to during capacity Trikafta new launch you've the will every you is physician couple in the what could therapy.

Stuart Arbuckle

and step be Yes. Phil, the XXX talking Orkambi CF they in not all sadly visits, who the seeing, launches exacerbations of it's approach clear, people the for whilst centers who rate they've patients the CF got them we the Essentially of and Symkevi there capacity heard of in Yes, centers CF of are is centers, their Stuart. how huge to Trikafta, run-up to heard yet hear we just Trikafta, amount obviously the when coming patients about they've it's planning it who and time, is enthusiasm it to things got regular about are are with like the they've we've admitted patients eligible got that. also their all same there, is being Trikafta. and to limiting from were of other launch for a

very as on while that well. things So are to going lot important they a very, other them, of have is Trikafta

total, in there said, Trikafta I so be will at As CF States, is who for XXX in XX,XXX United eligible treated or patients centers. the who the are

As for patients I said, XX,XXX were Orkambi, for Symkevi. about who was about it eligible X,XXXX

why we it bit uptake overall of very takes high be we're think, that's longer to time, we Orkambi get levels Symkevi. those little it going to over whilst a kind than levels peak to uptake of us of that so it and think a get to And may with did

Phil Nadeau

it. Got

And process just that triple reimbursed current to UK. you that the a reports the had can you had in follow-up prior different you. evaluation be talk floor from have a thank in UK, on by the starting the to the the through triple need question to order what kind Paul's mentioned subject -- it'll NICE for medicines little is you've bit more then you're to Okay, about The press in secure reimbursement is agreement the a NICE, question, second about a have the ground be process, that whole and to go than or go that past. of process through to before that an

Stuart Arbuckle

great Phil. question, Yes,

made fold, we through data, of demonstrating couple and that currently is will in in with that the public seen know that data We've of that and we've to the January and we get, the seen that of we and important types we've that we CFTR additional will agreed how US yes, will long-term open-label one us as are our a the timing with submitting the now The will get have. more NHS see long-term for benefits that and around recommendation. Orkambi, triple NICE more submit planning you submit do we and these modulators, be Symkevi. positive that are that when Kalydeco, with The we long-term reason extension is benefit terms EMA collect So that to we studies we said pending to data that CHMP discuss the agents in allow XXXX. the to NICE and of it we've combination

The be appropriate think a NICE submitting its is second undergoing that reason is the review why we of methodology. is an timing, time to currently

that made know, like evaluating As have takes to that and methodology, through that we're ours of types NICE have truly there which approach benefits we appropriately to to in and that kinds hopeful that value. you may ability some that review we've benefits to as medicines methods allow may value, bring. have long-term the the our that quite ours The their evaluation terms concerns about of better medicines some be medicines the of changes public, them

explained XXXX January that the why to we've that we So reasons said will we've agreed we're that's submitting committed when hopefully do the is triple that and timeline. on

Phil Nadeau

taking questions. Thanks for my very helpful. That's Perfect.

Operator

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

Your open. line is now

Matthew Harrison

a expect maybe you and available my minute you Can would taking in renal at what expect just study? Great. question. for on to hoping Good was given what parameters, you spend exactly could terms XXX. evening, year I assume next of on data, the next about I have thanks data that to some we talk look

Reshma Kewalramani

Sure.

transplant protein outcome say a very significant to renal So targeted a the there is called there and only death, disease. just and stage segmental the type on really The progression to speed a to disease renal glomerulosclerosis, the in manifests unfortunately urine. known is that molecule really is VX-XXX. one catch everybody has -- relentless end focal APOLX that's at and to axis that This FSGS, else up either is of is disease disease that itself or is is proteinuria, and it it's way kidney

right VX-XXX we're So Phase going we be study that We X next now program X, ready go are is where the with XXXX. in to dose-ranging SAD/MAD. to Phase are the to the we during year anticipate

disease given the small patient disease. has to when while of nature a And exactly it the going very to have to it's grievous very study size modest too early the that we're be and this expect population call I results,

significance get that the very is because of in in that and proteinuria, anticipate is I it dose-ranging of which going is So off and the convenient that running activity. there endpoint study endpoint will XXXX, to be proof the is biological

Matthew Harrison

to the you into other the Reshma, molecule about -- talk want

Reshma Kewalramani

Yes. Sure, sure.

late cystic portfolio know, is just diseases, and for for there, we making molecules in mediated behind through fibrosis the lead that there whole have and APOLX of us preclinical those a in you've kidney like so do portfolio seen through molecules, be a of this while their what and VX-XXX as will the one well the You is also way its that's clinic. going SAD/MAD, the in

Operator

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

is now line Your open.

Brian Abrahams

and of to maintain how questions to this for Hi, on guess, the than just on the first of the taking marketing need it early around more was up, how post preclinical typical Trikafta. guys plan think do educational all from to Two any we congratulations risk, clinical given relative nuances ending anything say effect Thanks. curious maybe you questions and side given and market the cocktails to off, tox that compliance thanks sort Symdeko. progress. so much you benefit some overall about do Symdeko, then should or me that persistence parallel work awareness on Orkambi approved, in my I I'm versus

Reshma Kewalramani

Sure.

Let on this then I'll comment then compliance education me start ask persistence. to Stuart on off and

to regard nice So different. in it the of with obviously, reflects were Trikafta I expect do we very nothing think the post-marketing tolerability quick benefit to thrilled setting, this approval of and the really medicines. and the With or unusual what

You we know gating an already that FRF in study had patients have initiated. that a F we

no, unusual. but that's different else nothing Stuart? So or those are continuing,

Stuart Arbuckle

in in positive, Trikafta benefit-risk see molecules as Brian, end my know, it you events would we in be range our terms that I with and world, very a be very the would XX% as would Symdeko, expectation. would tight of not other to its be from if it range the we've be as in is just terms mentioned a that towards that to that in can the the both benefits Kalydeco, CFTR I as discussions our the right terms seen range expect benefit-risk on the high, of expect with we real given that range. result across we'll of of that them they're compliance the sort with the to in earlier, we've Orkambi, fulsome and seen in being of adverse out what in will with we XX% them you Obviously, profile, rates, benefit-risk profile as Yes, Trikafta, been in have but how as as terms of modulators, we see of that of expect, I profile and studies, expect very medicine, they top plays physicians compliance the

Brian Abrahams

Thanks much. so

Operator

of And you. [ph] our comes Bank Geoff next question America. Thank of from

open. now is line Your

Unidentified Analyst

the for thanks on congrats and approval big question the guys, of fast Trikafta. Hey

of So what a focus back are more secured getting out, learned roll for really going question for I on reimbursement access Kalydeco, And need insurance doing see guys can assistance barrier? So for would lessons that what the percent you reduce I some you you and the say it's have with guess Symdeko ago co-pay the assume question that? a have sort is that's one. with what Stuart recently, roughly guys And years how second are you or follow-up.

Stuart Arbuckle

Yes.

would So US, Geoff, seen and quickly. access to Orkambi in in terms terms in pretty going through been broad in getting Symdeko, we've the all very here reimbursement Kalydeco way of place of back put what say has learned the actually that reimbursement I we and have

with Now, in up a rapid than and move of quickly across move and ended move kind some payers government some pretty but them all of others, broad obviously and faster we've slower the seen it's reimbursement. commercial than range and general, so we've others,

how very years me in our now reaction the with very, been say, we've that benefits and that And with as positive. now good to has gives reaction hope in medicines is payers. for team have will out we've very the there been severe of a of there I terms date talking out seven obviously the is, today, think disease that had, the these the US, the thing I that sense payers happen this is they have

team secure access. feel our good very prepared that I broad So is very to well

out will barriers. to be as justice them have insurance trained insurance terms of and to we navigate to is great which team there they here barriers, support In yes, patients do always some, a help the provide

left you to pay. we standard going that behind are we have ability sure going suite and make the commitment In no supporting those of offerings we that is do may their needs, certainly financial to in is what who patient can of are terms would to that expect patients our have to of we do everything because

Unidentified Analyst

program, are When but a hard, of on though, to then, want the maybe of and even, then is most I on much of think you, how everyone's early later pain so capacity pretty to opportunities stage Okay. what have follow-up like is the a you lot and bringing even assets. you ask been priority Jeff. outside them seems for diversify And or CF, about it or just Reshma working guys a

launch. in to the the Trikafta the As from you. leverage see you Thank begin P&L

Jeff Leiden

one. question a maybe It's great I'll take that Geoff,

have been did done we've I today deals. getting was -- Board think hard, in than the entire in on we've I than showing done We these working more that both diversified we continue and XXXX built and team our a toolbox deals you our see us XXXX, also and XXXX Obviously, good pipeline and considerably to we've expect should combined that's have do that. we our to thing. XXXX

our XXXXs. I essentially see consistent to buy late revenue think we've us pretty revenue provide you to what expect shouldn't very or all assets into been growth. along CF will the assets on-market buy well that We need franchise in do growth; saying to probably don't stage is really

build more the own can our both very And add stage and where much able group, way, we of internal can in much, that a much we're so so, from being for position patients development to value by also value nice assets, for better shareholders. in get we we better think invest value regulatory much and earlier and

last I products stage, the continue you'll products. So certainly more particularly technology not early bolt considerably but add stage have editing gene us on late assets, strategy, which to very do our do market the and deals, you we very to don't deals you to see expect think over year should know late-stage value, broadened that of to those we is see can where on or us as

Reshma Kewalramani

wanted add just to I to that.

You pipeline actually talked development. interesting know where many already and mid-stage are, molecules about into have we how you pain. to mentioned It's already the we

year. expect So to in Phase X, X this CTXXXX next is AAT already X, Phase and FSGS, in expect XXXX I in we Phase quarter start be X, to be it'll

Unidentified Analyst

Great.

you. thank Okay,

Operator

comes of our JMP Bayko And Liisa next from you. Securities. Thank question

Your open. line is now

Liisa Bayko

questions just thanks help ask question. you for the about Hi, to wanted with my to pipeline. I taking

is of first group what you're Z to better that AAT program, understand down mutations. for its I The break you molecule kind just on corrector, types the maybe the AAT Can mutation one. of patients the noticed with into population focusing with two you're small addressing kind first

Reshma Kewalramani

right deficiency, So there The alpha have the different who you majority are but majority of patients of subgroups who the patients of that ill are X-antitrypsin have mutations. patients the Z mutation. are have Z

the XX% so we're that on. So plus have the Z mutation, focused that's surprise group no that

Liisa Bayko

then And of qualifying you is as about FSGS by of similar sort mediated is that All cases, these -- about kind again bit curious that FSGS, know right, a that APOLX of little majority the one. that I makes sense. question. just

Reshma Kewalramani

sure. Sure,

one that bit So little a is different.

group APOLX. that after in that of So finding that, actually a etiologies is is right by FSGS mediated a there within it's heterogeneous group homogeneous pathology. is is results named FSGS on But that

it's it's a group, mediated FSGS, mediated APOLX of APOLX subgroup that pursuing the and one also disease is homogeneous actually there of a mediated that groups is other are we FSGS, all pursuing on but focused not are it we turns APOLX, renal are we're FSGS. all FSGS So by out

Jeff Leiden

cause strong reason there because is genetics that the of is human very Liisa And evidence is disease. that APOLX the

Liisa Bayko

guess Okay, of group? great. FSGS how prevalent do know what FSGS how patients, And this that related is -- and then you -- guess to is that I the I particular how

Reshma Kewalramani

vast, but short you are who two, important studies the there's important really nicely that's topic, have here's that I versus so today's three is call this is like percent plus looked happen that there African-American I'll a FSGS I'll amongst I'll who majority, have at more FSGS progressive. patients XX more the mentioned patients, serious what that for nephrologist, so done to it are maybe other those disease know only So studies, APOLX and to really I have and not, patients keep APOLX, make related vast be FSGS, point say have points mediated to

why we pathway. this it the interested and Jeff here factor are that's disease so this As in and in said, is causal

Jeff Leiden

And in by been sequencing. This [indiscernible] you just know way is is described the literature. that's

Liisa Bayko

Okay, great. Thank you.

Operator

And our Leerink. Geoff SVB comes question Porges you. next Thank from of

now open. Your line is

Geoff Porges

Kalydeco, on much. Thank Charlie, give you you. the was operating CF little more a and change a clarification Thanks. cash margins. Symdeko, could you us the just free of the on the we level Thank to tell very models. just then, the one of you of tell to and what then little quarter and the bit. US patients set And in your number medicines us our breakdown flow could bit Stuart, of kind And total direction so Orkambi, Kalydeco

Charles Wagner

Geoff. Sure,

the in up you can the On afterwards follow we cash on with free flow quarter, that.

highlighted, the so strong. very the started we the I end ended which after development that is include with to flow more generation the you the with that the As cash during not for on the $X.X year, year quarter track that's And be spending though our on we can did with after, nearly in happened -- cash operating billion billion than to see this what Semma disbursement even year. year Semma, leverage business and we continues shortly performance cash $X the are about

to in the seen of you've year. a operating expect, first quarter, saw of fourth Second you additional low was the ramp the ramp your in run the up expenses in third the around of margin, mid-XXs bit a half part in do quarter. in we've kind an little up question -- of We

end that continued the to around so XX% operating continue should and Again, well. drive could of expect on line which up business that to year, XXXX spending, into low and growth a we disciplined year was we with So margins significant the the you top range. as last over mid-XXs in the and increase

Stuart Arbuckle

in was the wanted I US, of you breakdown just it of terms And think ex-US. Geoff, Kalydeco

Geoff Porges

Well, the markets. three

Stuart Arbuckle

three. All Okay. all got right. I

quarter, the and ex-US Symdeko $XXX was US $XX US, $XX ex-US million, So ex-US was was Kalydeco in Orkambi the in the quarter. quarter $XXX million Symkevi US million was $XXX the was the in million $XX and million in million

Charles Wagner

Geoff, is looking details you've would in pretty this. for Charlie. on been the add, I this consistent

that some from even and patients the treat to within this significant going move us majority we're detail. a medicines with approval, and a of level label forward, the Going allow away triple the there the vast with of see expect are going got would switching more portfolio medicines, to to Obviously, a of overlap triple. of we've we level now of switching is we significant

As breakdown a very, the detailed result, over very product by less becomes meaningful time.

out So moving break total CF report by would product. be to revenues our and forward, intention that

away ex-US by revenues, US very break providing our also but from we will and out geography. detail will product We move

Geoff Porges

Perfect. I you. what will know to Thank expect.

Michael Partridge

time have we more one question. Operator, for

Operator

you. our Thank And last comes Ijem Whitney question of Guggenheim. from

open. is line now Your

Whitney Ijem

Hey, guys of the quick couple thanks A taking questions. for follow-ups.

sort mentioned, plans for had are curious, on naked First program, believe, guided on the is first FSGS, They half Semma. track? a that XX a previously for to still renal to driven are And you by kind of do for start cell the term? basket then of FSGS I specifically you any diseases study of or focusing near there APOLX as on just

Jeff Leiden

take Yes, FSGS question. Reshma the question take Jeff. Semma the and Whitney, this will is I'll

give naked it's in pleased I trials. they to a we be terms start to with that, precise our Semma, to can I but tuned actually anticipate plus certainty. they're with or little do date -- to both as finish in when more the But with future, programs, we five cells little this you clinical progress regulators, anything discussions it it's cells, the the program, near and very they device In sign like the meaning three are years we a regulators of in isn't stay out a as think a discussions early will and

Reshma Kewalramani

also is the protein very to much high are has more different high, enough kidney it's other. basket example the there think so faster the the progression our that the approach. levels not to for FSGS compared we are it that that that and and But, have disease to no, non-diabetic With there component at first real get APOLX trials. actually we'll that's patients of get good we unmet need The there and go FSGS a component. sick, then regard question is to They're mediated about an question, FSGS a

a is going here do that we're focus FSGS, APOLX as standalone to mediated our trial. and So

Michael Partridge

Thank you, everyone, their in for questions. for tuning and analysts

additional If you tonight. will Have have Investor be questions, good team office the Relations the available evening. a in

Operator

conclude does conference this Ladies our call. gentlemen, and

disconnect. You may now

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