Vertex Pharmaceuticals (VRTX) 25 Oct 172017 Q3 Earnings call transcript

Company Profile

Participants

Michael Partridge	Vertex Pharmaceuticals, Inc.
Jeffrey M. Leiden, M.D., Ph.D.	Vertex Pharmaceuticals, Inc.
Jeffrey A. Chodakewitz	Vertex Pharmaceuticals, Inc.
Ian F. Smith	Vertex Pharmaceuticals, Inc.
Stuart A. Arbuckle	Vertex Pharmaceuticals, Inc.
Michael J. Yee	Jefferies LLC
Matthew K. Harrison	Morgan Stanley & Co. LLC
Geoffrey C. Porges	Leerink Partners LLC
Geoffrey Meacham	Barclays Capital, Inc.
Alethia Young	Credit Suisse Securities (NYSE:USA) LLC
Brian Abrahams	RBC Capital Markets LLC
Shawn Fu	JPMorgan Securities LLC
Phil Nadeau	Cowen & Co. LLC
Robyn Karnauskas	Citigroup Global Markets, Inc.
Lilian Wan	Bank of America Merrill Lynch
Adam Walsh	Stifel, Nicolaus & Co., Inc.
Tony Butler	Guggenheim Securities LLC

Call transcript

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Michael Partridge

Good evening. This is Michael Partridge, Vice President of Investor Relations for Vertex. Welcome to our third quarter 2017 financial results conference call. Participants are now in a listen-only mode. And later, we will open the lines for question. recorded. is call This be tonight will our available replay on later website. A Dr. Chairman and Leiden, CEO; Jeff Dr. Medical Ian Officer; Officer, Chief Chodakewitz, Jeff and prepared this will Smith, remarks evening. provide Operating Chief Q&A. Commercial join Officer Stuart Arbuckle, us Chief for will

and are the We will make release, on of management's detail cystic I'll the Commission. Dr. statements slides, to commercialization which Securities be and on our on found GAAP in materially. of statements and ongoing webcast XX-K and differ discussed those current non-GAAP today's results posted filings our These in are website. subject in financial with Vertex's tonight's the and the ORKAMBI, Jeff over non-GAAP These Leiden. could can financial uncertainties the based turn and this the risks in Vertex's to assumptions. press programs GAAP call. outcomes fibrosis forward-looking development events measures future statements release regarding have press and regarding performance, including financial KALYDECO of other use to and Exchange Information other been call our and now now Actual reconciliation and

Jeffrey M. Leiden, M.D., Ph.D.

across Good business our We've been for has Thanks, made of everyone. treating of vision and long-term CF. parts a year significant with tremendous evening, Michael. moved XXXX closer realizing progress all our Vertex. to people all

patients. data recently, that showed of of able for demonstrate CF being and be treat we our multiple regimens eligible medicines. the people to During to the we've More number XX% may increased year, treated with triple all we approved up that from combination

this our And have our for approved CF full-year increased product tonight, people we result a quarter-to-quarter CF medicines, with increasing As product treating guidance revenues. total are more revenues year. throughout of

continue strength has significant forward creating going for growth revenue we financial earnings shareholders. this substantially value Our and expect and increased,

progress last months. Let review briefly me three the now over our

medicine transformative to is CF treat the older XXX globally. two with X,XXX gene. approximately KALYDECO. KALYDECO First, use CF we of the with continue than for number people and of labeled FDA in who of treatment the to function five and now August increase KALYDECO residual have more a people to mutations X, continues On with one patients initiating ages KALYDECO. be approved CFTR And

two, Phase Second, on the Medicines older of copies CF CF-patient announced population. the data with and combination we function tezacaftor/ivacaftor, date action based NDA in applications Review Europe have mutation ages have And the for who Priority half XX FDA two responsive the one positive in of this XXXX. the FDA U.S., in granted currently XX, treatment of the February X two FXXXdel of accepted expect people the people one year, use and earlier our is approval Agency have European has We residual and second of set to the studied mutation. and In FXXXdel XXXX. tezacaftor/ivacaftor mutation the tezacaftor/ivacaftor. One, who that the different an

to who in the many awaiting its are currently U.S. to approval. eagerly commercial the medicine team this bring Our patients preparing is

results far, in clinical increases it for has saw het/min multiple increases which evaluated in different patients CFQ-R. clinical FXXXdel VX-XXX. next-generation the in and regimens measures, significant VX-XXX, in and increases both and in homozygous in activity and study triple our correctors, three two across FEVX, chloride VX-XXX In one been sweat we announced thus these studies, which combination we in regimens. four-week regimens, Third, July, included Across

good saw also We tolerability.

with triple and regulatory We these VX-XXX next-generation agencies. correctors to expect in studies VX-XXX, collect and VX-XXX early data and continue X next year into from regimens Phase pending combination from to data discussions

of to pivotal the first development two XXXX triple in half begin regimens. of We combination up expect to

daily. of that of to cause each next-generation the a CF triple their These different medicine may one treated a regimens the the regimens be of XX% opportunity of dosed and population corrector, once for have that may addresses the with underlying provide be these triple expect regimens We may disease.

earnings people the significant with to of progress consistently and believe you transformative treated significant We and advance patients, XXXX. to shareholders. And growth invest turn this seeing investigational out our eligible our into medicines and we progress has medicines new development and Jeff the the call for employees Chodakewitz meet delivering make medicines clinical creates We proud continue are we on our and forward company potential these being specific over in now I'll well the to for of some look comments forward internally updating programs. expect to revenue shareholders. as select to bring we positioned Dr. goals medicine. this around good of sustained very with in and as creating you most of to the collaborations, development life-changing world. our the value for medicines. for to number We're We increasing areas on patients scientific in made and how helping well progress We're innovation both and our understand

Jeffrey A. Chodakewitz

patients good very been CF. to CF across goal make us the this Thanks, bringing multiple with We're with to evening. studies our pleased Jeff, which closer year, able and we've of treating clinical are progress all

in three combination ORKAMBI will clinical the ongoing I'll that will make different completed. from I programs. I triple CF with recently start First, our comments review results. on results Then studies

that are the result Phase have years two to with was endpoint five results tolerated. who and of age ORKAMBI We the the CF children mutation. to The of was pleased treatment X copies of tonight primary have study two FXXXdel the well announced in show of safety,

chloride also our the nutritional evaluate five of two. the medicines study, Based the that earlier benefit the safety young the including findings and ORKAMBI has XXXX. CF-related of These younger change children U.S. the younger disease are first progressive in and first intervening to devastating and been quarter CF sweat and in disease. of disease. our the submit with as from show may to medicines patients in to a expect by measures, regulatory the improvements approval the And establish In that status. goal ORKAMBI for is a to study showed in two as results and we in for Europe so regulatory we course authorities basis on addition,

ivacaftor line of meet addition to not who receiving taking primary from The those CF no placebo mutation Now did announced in tezacaftor/ivacaftor. was added compared evaluating to endpoint to X and tezacaftor a monotherapy. they Phase already were ivacaftor. with older the FEVX the in who We ivacaftor XX data a top copy ages people and study difference already addition study to receiving there addition mutation with as that of one those second-gating in tezacaftor for the were FXXXdel its in

tolerated, generally was with Importantly, safety the study data was consistent that and of the profile safety observed the combination. show from tezacaftor/ivacaftor in prior that combination well the studies

we around plan patients will to KALYDECO, majority that patients looked sweat CFQ-R. of gating to we sweat we no CFQ-R. of receiving seek FXXXdel The gating approval do for they a statistically difference an significant tezacaftor/ivacaftor results, all have a and expect do that chloride, saw secondary world mutation For are X the and also mutation. who approximately Based not millimol on continue to compared these in and so. with chloride in we at In endpoints, decrease ivacaftor vast monotherapy was also

primary ENaC Phase its study study mutation. XX-day X We've ages VX-XXX, results from to did produce in ENaC to study not tolerated, our patients homozygous are study The inhaled showed when who VX-XXX. a a saline. with ORKAMBI, VX-XXX announced ORKAMBI. older receiving CF of was well for or XXX FEVX from meet Safety XX inhibitor, monotherapy. the also inhibitor, with and the changes an patients efficacy safety to and VX-XXX dosed In VX-XXX of of these profile Now, added when that The who endpoint. was addition generally data administered not already of studies ORKAMBI consistent in that are prior the observed without did the hypertonic addition the FXXXdel

patients I advance. on some primary ciliary regimen continue to VX-XXX conclude a combination as in Phase they of will X We with conduct dyskinesia. to with continue monotherapy triple our remarks study

first role an different on combination once-daily plans. that included to good hand of CTP-XXX, remain call play in important updating combination treats VX-XXX formerly to of X in track into XXXX, experiment for forward over the VX-XXX to could you positioned of regimens Phase I VX-XXX. the Phase already Ian. up a the well our and of and regimen development I half CF. the of triple now will that to activity have studies. that, in on with four tolerability With We future the which underlying announced regimens pivotal three studies are We We and to two look in begin potentiator, cause VX-XXX have four-week triple demonstrated clinical we significant the recently correctors, X, two next-generation

Ian F. Smith

the deliver been evening growth to Thanks, year discuss and revenue This trajectory product $XXX the on demand reflects medicines. million revenues on million our Revenues financial And make a of poised beyond. represents XXXX CF has the and significant Jeff, strength in strong of the to full-year to and further financials, year. for quarter and based growth. our Total and into review comments continued business. will XXXX Tonight, I compared good XX% XXXX our of our increase performance third financial earnings a $XXX guidance third XXXX everyone. first. XXXX last quarter we're

treated throughout XXXX with increased the approved as medicines. have of revenues quarter Our the product number patients grown we've each

expect growth and launch the Also, we such We ORKAMBI of the potential this we continue demonstrated U.S., XXXX in as also as quarter. outside the and gain additional strong new medicines have countries in in to have reimbursements KALYDECO tezacaftor/ivacaftor. to

by U.S. has XXXX quarter this The increase medicine most For million, of globally, continued million of third of in quarter children driven the year the ages ORKAMBI, uptake compared $XXX we specifically growth $XXX been quarterly reported X XX XXXX. the in the third revenues to an of product to

the $XXX to following quarterly FDA this third quarter were been has strong quarter XXXX. the by patients for recent million the mutations with approvals. sales Third medicine growth by $XXX for residual The KALYDECO in function year driven the million U.S. uptake compared

Operating revenues. were Our acceleration an the of the compared and This regimens of third XXXX. third to increase investment margins for our margin. third XXXX quarter Non-GAAP our quarter XXXX. the million third profit our to strong $XXX quarter third of for in anticipated for profit CF quarter to continue the portfolio medicines million product quarter in CF SG&A future. non-GAAP due growth in XXXX was $XXX significant growth our million to XXXX our The with were R&D continued in was the we non-GAAP growth expect the non-GAAP advancement XXXX. to combination triple expenses profit was by net compared million Strong in combined of third the in total and net in the of driving XX% to product launch support primarily the growth, globally. net operating driven $XXX the for revenues $XX expand revenue quarter largely of XX% and is expansion to And compared margin of

We marketable Pharmaceuticals upon securities. have and we strong quarter, with our completing sheet XXXX. paid asset cash, purchase in of This approximately the now agreement billion during compares $X.X billion we as CTP-XXX, for $XXX equivalents, at the million a beginning the balance third And $X.X third Concert cash ended VX-XXX. quarter, to to

and total recent in the to of $X.XX revenue turning increasing $X.X was strong the medicines to to billion billion guidance of billion and our are increased product based to XXXX. expansions, previously year. full-year on we this $X.XX label This our in underlying financial August Now, guidance, billion CF $X.X demand CF

in now to year and throughout among the billion the $X.XX XXXX. based six is medicine of this ORKAMBI, strong which on would demand reimbursed. product and ages where revenues is that in ORKAMBI net guidance people older potential For does assume in reflects product in ORKAMBI any expect the with revenues, U.S., the the for note in currently billion not the $X.XX revenues countries we I underlying recognition CF France

$XXX we recent underlying product eligible KALYDECO, label patients. seeing to expansions expect based As to strong $XXX we and the million net are revenues on now demand million of by

we product guidance, to challenging approval CF mindful forecast but early of As make we it that individual XXXX, anticipate and we products. Therefore, the in product revenue expected total for tezacaftor/ivacaftor will no are individual ahead to think guidance, revenue potential revenue guidance. providing XXXX launch full-year

Now $X.XX XXXX. expenses combined operating expect to and billion we billion continue SG&A for R&D to to non-GAAP $X.XX of expenses,

expenses two supply potential a XXXX, drug will we be with a of time, driver in for a In investment plan and and supply expect triple any track studies on same for of we increase operating includes and sustainable operating triple initiation significantly a operating up As the well our XXXX, to financial growth we commercial the conclusion, to pivotal growth. regimen. non-GAAP regimens, do will see success key combination chain in combination for will Therefore, earnings At investment our revenue in margins that as are expenses. XXXX. the high margins to exceed expand profile growth operating clinical deliver

number increasing have the treat with our driving financial success in performance CF of the that Our patients medicines strong reported today. we is we

our with the ahead. with for positioned look and the questions. to patients with share the benefit development. goal assets in progress to We rapidly And of we I are you line we many and realize in the to more treat enhancing are our open CF well continuing months We those pipeline that advancing forward to that, treating weeks

Operator

sir. you, Thank

Our first the line from Michael of question Jefferies. of comes Yee

open. is Your line

Michael J. Yee

and in VX-XXX about next triple Two-part great was, the an in X you year. that way uptake expectation program the second accelerate you figure question. homozygous and look the of that? and then as like the overall of or both the there duration het/mins question think is the of should out much to is the forward, you terms Thanks ORKAMBI around do afternoon. clinical think And good Hey, pretty Phase One could study, going strategy a quarter.

that? are do early, cannibalizes to is it we Where to franchise? are market? Thanks. significantly that opportunities appreciate the I Do think doublet a grow opportunities grow While How there it bit? the think about you there overall

Jeffrey A. Chodakewitz

all expect for the the question. my Hey, regimen given to triple Michael. portfolio. the comments, Thanks on data Maybe, collect we

XXXX. Leiden really maybe a in tezacaftor/ivacaftor Stuart can point, how say that let's detailed have a we discussion. XXXX. into about of the expect timelines, strategy pivotal studies, can But early view Jeff more to to in the talk and that we approaching think good At We we're have launching then you how the about

Jeffrey M. Leiden, M.D., Ph.D.

Hi, Michael. It's Jeff.

high-level regulatory Let to view. now. give a you discussions in Again, me just start we're

couple able we obviously think, would be Jeff is, we and finish obviously, those. to worth data, be said to I So a won't based view the plan, our you current till One the said, on final making. But will I give be point of points, of them. on take in Chodakewitz as that that those have a be that different programs next-gen incorporate up programs once-a-day correctors two And would two based one might point. from hope, X, data is one And programs to regimen VX-XXX. would we'll the our those Phase forward. see again, of That's which

The the phrased we've trials it, your through question be. you learned in obviously CF. learned is of lot to we've way second directly to point a which the what those trials gone as length needs X multiple Phase We've

endpoints. For weeks the to they those about how instance, learned powered want. to up weeks data for We've collect to of to the data. And get XX for we've let's size about we to and acute say, long-term trials, those have lot collecting a effects be X learned XX how

results as the the that triple, to of on our So these is with And overall that we all medicines regulatory so seeing who strategy quickly can het/min we're right trials these based the that streamline to as possible come. use now, strength patients particularly to so knowledge. to the the possible of with as patients, agencies as more particularly have don't get much anything

those As you we the But our in is trials patients drugs possible. those who discussions, certainly these the think waiting about to them of finalize streamline get or quickly those to beginning as to half as intention able first we those to are be how year. for next we'll to explain and

Stuart A. Arbuckle

Michael, it's And, here. Stuart

CF more the the a opportunity modulator. think it going to. tremendous we tezacaftor/ivacaftor, benefit/risk grow profile whether be think, you provides of I'd allow CFTR to kind certainly the it that patients a probably the We the of we with There's given to is really franchise, treated it's has, do. to new On treatment couple of option groups different which patients got point

first population. in homozygous be The the would

events due given likely As think you profile there, tezacaftor/ivacaftor. number which to that's to I a risk know, ORKAMBI tezacaftor/ivacaftor, a adverse population with ORKAMBI patients often discontinued who demand high had for see we've the of is benefit of

a any initiated homozygous profile. never another I number benefit who've know that of ORKAMBI patients. could function that's mutations. those have the their been treat because views And cause also those risk of patients responsive further but outside all And those obviously in be the population, of there's option often of same in to disease. population the on We think yet excited treatment a for there, and third don't would of be in have one patients Not the down population KALYDECO, U.S., the ivacaftor naïve, again, patients that treatment then We the indication function homozygous by residual be their the good residual option. approved on could line, here for those tezacaftor/ivacaftor the new are for for outside are potentially of the who the And obviously underlying U.S. so the population. a the KALYDECO

a see the being important which So, very grow patients treated of can option, we number modulator. certainly the new CFTR tezacaftor/ivacaftor with treatment as

Michael J. Yee

is they follow-up months? magic reason is the picked? or just, that any numbers magical final ORKAMBI, anything six why three there there be Is couldn't for those months that My about ask were

Jeffrey A. Chodakewitz

different can different benefit said, clearer There know X we as a should use. like or have weeks. in to benefit, consideration been pulmonary to three XX within certainly hospitalization, I that. see the The Michael, we this. pretty things are that exacerbations, I little two antibiotic There's acute and about – long-term quickly

end with up then provide typically there's, measurements regulatory that If of we the wait necessarily don't of is take view, the that, regulatory and those said need Having all is need a to try don't going one. means the database to file going six to to way all an safety adequate that submissions, believe who to obviously, of you we're anything we that strategy to that's that which for of patients what the course, fits look particularly which to come months, we to have today. do at point which that

Michael J. Yee

Got much. thank you so it,

Operator

you. Thank

Our next Morgan from Harrison question comes Stanley. Matthew of

please. Your question,

Matthew K. Harrison

ask Perfect, parts. was and pipeline afternoon, question, hoping could thanks to I Good everybody. it a maybe of non-CF two very much. sort ask I in

course how So, pipeline specifically milestones year see the over injury into spinal help cord about maybe might you internally plan pipeline. And externally one, us asset? either to your think I the some of about or next then non-CF we the the Thanks. non-CF about for could and you that guess, just talk of invest

Jeffrey M. Leiden, M.D., Ph.D.

is I'll part the second an can the Leiden. I a sort Jeff to Ian well this as first Matt, it, maybe work take question. internal BD on it's and get and of part question as

about about the five. them before as or And talked you clinic, hear pain. them. sickle you'll enter five We're them. about investments quite we is First, many, I'd real we R&D they expect nicely. where we've includes talked pipeline, XXXX, that told Matt, innovation the about is we've enter is programs something which four we've internal about that said this many we in industry, BD. in value talking anyway, four AAT, Those with that We've and at in is our we With to investment Vertex, created to external clinic remind talked scientific or programs to and adrenoleukodystrophy, more internal our you of do times in investment both expect disease, it's that cell some those respect believe what actually and and in bring, are our advancing the

anticipate you've bring number will, technology platform, deals more you early-stage also about next both programs hearing you a of if strengthen investment, think our the I and external that products. us to year. do in With can respect seen those So,

BD job we our more of us won't more for hasn't of forward does for the changed You'll of financial change and have the which years those. maybe do But investments, firepower nice except strategy Ian that several sort last summarizing going see a fact now.

Ian F. Smith

sure. Yeah,

the So, the we to create years in it very I ago, invested couple we different where our by, today, prepared capital preservation of my any debt. approximately have position flow. $X.X sheet medicines; without company we billion a were today have would significant out whereas mode pointed balance And remarks obviously, as one do cash taking that in actually was we start than I'd is in cash in

access we I point, have so, capital do to create. to Jeff's to to is and have significant And we that invest a believe

have we externally, a where so of approach. And got three-points strategy it's

notably should orthogonal modulation, you First are CFTR to in that and is, try modifying the as have to opportunity improve done in couple we deals most approaches that the And out. And more look approach see to everything we've plays with approaches identify going look a to we one do in really done compared that study at And to regimen. where the was we everything our at we of may by there And QX in own we've We Concert potentially agents. but complementary the regimen CF. where a have CF. area. we'll how disease once-a-day be

already away a with going and our stage. but And to of which give on look notably, has we've tends we'd lot mentioned, in. Jeff the last looking into to we've to people later this the modalities be outside areas the earlier this consider getting little at been and to Vertex it's scientific at then areas done collaborations so. to large-scale footprint. Moderna. as well successful at been and opportunities of And a we of nicely; the areas kind – the disease deal or time is touch approach stage. as we're Secondly, capital. it on. whether third over disease looking think don't with be we still focus area that And that Vertex apply world to those probably we us call, But consistent that actions They on also is years very opportunistic point, and most are plug more there's Therapeutics, tend want I carried done with earlier kind at and disease who is diseases tremendously that XX CRISPR And of molecule for the different going Vertex interested to that. small on transactions

with match we've our consistent these in can how with CF. developing medicines scientific we creation things approach done to So,

our capital to it generate is So grow our that's and important that applying business. as we approach, for us

Matthew K. Harrison

could just then, cord And I data the on next ask will I we asset Thanks. Perfect. specifically, mean, see Okay. spinal year?

Jeffrey A. Chodakewitz

don't patients acutely Really, I Jeff enrolled, even the remind then it It's and follow then treated to that It's Chodakewitz. Matt. enrollment, for time. once studies would data. on get get that. quite rate Hey, all expect a of and the that are patient we is the you we to these them just going depend and of long-term bit takes some time

tell, it's wouldn't So any just year. really early expect too I but to next

Matthew K. Harrison

Okay, perfect. Thanks very much.

Operator

Thank you.

of from question next comes Our Geoffrey Porges Leerink.

open. Your line is

Geoffrey C. Porges

understand increase observed failure or triples? And through presumably, the does secondly, basis and chloride France And numbers. Thanks go France does sense congratulations tez/iva still a much, revenue? mentioned you which is when and and of reported what cumulative And your very that from results gating you fit financial how and the help now converted with that non-GAAP into from Thanks. the the mutations, us progress. activity sweat question, is, with the in the quick on us on preclinical Can your guidance. the assay? decrease over your maybe, a updated assessing and excluded you give in change the confidence will great in the that for be of then revenue the actually that that and Ian, cash might could Jeff is think that the

Ian F. Smith

the thanks Geoff. So, question, for

together we as – get the just answer complex well. can it's call me Let after a and

hat put just CPFA my on. So, I'll

revenue. first France, So, of do record all, we not

you balance We France, and in medicines give price don't sheet agree be to we to the out. for are some than France because amount to sheet we they're an got approved the that sheet. we cash that's top million price we paying to the But on that have that that because may ATU $XXX the we actually for France revenue revenue $XXX – definitive bolus. forward. If list allow getting that we recorded of in keep agreement our have And lower reimbursement currently access in recorded in about as be on it's recognition be would the accumulate and a of in us it balance if we paid program, going the the record have back because would in a reimbursement on would through is price the XXXX, were the million, receive would that quarter balance

earnings. cash revenue if have But the to the of to it in sheet at. X, go estimate of at is recognition reimbursed or an That you're is through in just of passes sits that It be Now, through now that are we're X make the XX there revenues as now all companies, not will Vertex, January will no actually and January changing. because value XXXX. sits aware bolus in line and retained rules December on we does probably this of applies complexity the of this not is bolus of all what bolus we that the now balance actually revenue And goes XXXX already.

the France. is, start how for that's in we recording backlog X, unfortunately. try don't through do go to Hopefully, There getting as have you're happens medicines get you So, just forward, if proceed. that paid be you in XXXX, what recall it simple, cash. What at – your earnings, January but in going keep to revenues of a will to XXXX. bolus not retained recognition It'll

Jeffrey M. Leiden, M.D., Ph.D.

I So, Jeff Leiden. glad got the Geoff. This I'm the question. easier of part is

study looked in believe why clinical carefully. asked at reasons there together, We we've a very benefit. two that of about the Obviously, the show this didn't really data sort questions two that explain that are You as tez/iva results. fit there you'll see,

entered who important is, might patients these The the extremely expected. treated were reason as KALYDECO you reason, first and maybe most study the in have well

that You characteristics. can in their baseline see

quite chloride has three sweat on carrier actually is more they of So, for XX, And years. levels. close drug the a is to average patient normal, a had the of They had essentially. been which than BMI which XX.X,

when they well. that the of it to made this And along having which incremental study CFTR ended and corrector they very to part benefits, those equation so in acute in were because millimol. the high other identify X KALYDECO that from chloride X obviously in incrementally has goes were the set at first patients tez, thing that a doing study, improves improvements with which only difficult and is, that The simply so, function with sweat bar single study, we're gen this about see can that FEVX clearly you

combination sweat a high the and difference about that but bar fact It detect adding started millimol. of FEVX. acute in did, X only still chloride. single was study the the made to so were And just the It incremental first-gen only we difficult it improved which that when by corrector,

Geoffrey C. Porges

Okay. Thanks, Jeff.

Jeffrey M. Leiden, M.D., Ph.D.

Sure.

Operator

you. Thank

Our next Barclays. from Geoff Meacham question comes of

line is open. Your

Geoffrey Meacham

possible heterozygous Thanks, CF based data, at the just your you've homozygous specific question. I or for see very mutations. When guess is it's of look triple the at triple look the obviously medicines entirely for you could delta-F, for when developed patient Great. regardless on And that program, very guys, a with that specific development I we be if treated a today. what

you then on delta-F? little from than follow-up. have the out would strategy just the place had to data homozygous homozygous het/mins Do going you allele already Or do agree with would that going? triples agree what A), delta-FXXX and for ahead the then, with I with what And single And first different obviously you would get go medicine? in one delta-F, of you and go a that? – bit in wait more one-size-fits-all you So, data be a B),

Jeffrey M. Leiden, M.D., Ph.D.

think months is and A Hey, pointing how Jeff concept important This about we're we've because question, last, the changed I that internally, whole as originally CF of triple Leiden. out we've a and so, is this over each which might disease three really strategy buckets that require here or lot great XX talked our gotten patients, particularly thought something more or approaching you're say, data. Geoff. as We of has around question different four whom medicines. of

therapy you for our suggests. you patient, the have results you're monotherapy said, certainly believe, you're treated dramatically whether triple another. that the allele triple that FXXX you KALYDECO Phase data alleles, be or we're have do what That's another, say, with two just FXXX one, with will doublet changed best one So you a triples. and X that's as And from for maybe and let's homozygous regimen, seeing for we het/min, a whether

KALYDECO on a remain gating have mutation on all allele. or all of on who what There take responsive second-gen up monotherapy XX% if single a to will you of a not corrector patients, might, instance, it those believe, So are allele patients on be together, be triple. not FXXXdel, for means that who but the mutation small will one likely set really because a that other we the is patients

make the We're And very hundreds therapy obviously you're have don't who largely may codons. XX% we not left treated a or those gene strategies. patients with stop bucket. small but CFTR those We're farther so patients going because patients fully KALYDECO. they the editing. of with on from to a those away. number, corrector whether those And patients that of the They're will therapy, be then are patients. working final gene believe obviously And genetic that's need those, any believe we any talking in protein, of a are few And benefit

to a are a So I now. from we That's see there years just remain the and triple and think to way therapies, up towards going effect, the waiting believe you effects. single to a it that few few, is be Ten getting moving as about for will be percent carrier genetic patients will rapidly on on KALYDECO enrollment, the we world that be which think number maximum monotherapy. XX% We're of how small regimen hopefully post a said.

Geoffrey Meacham

you population, is of feel like the good they today full need priority enough and are than nonsense mutation that large a technologies optimization? on And less for helpful. the the But delivery you XX% population comfortable draft. gene of segment the that that's fairly is a with editing you and address systems today? mentioned, that are do gene to segment? still you And do the you the and or patients, Jeff, for Okay, further then, And

Jeffrey M. Leiden, M.D., Ph.D.

that first all with part consistent So treating the first we're we have committed for to patients and of all, of CF. been question, your insistent very

So our not the one higher than that's and priority, is population other.

gene delivery I out, going difficulty move to what's CF organs. because actually that the to have that but those, the remember going the be think point area. of as and the reach most of by therapy a make if not that's simply a delivery, of that close we're or that interestingly, we're ailed course, that's gene gene deliveries of therapy a treating that mucus to to to get as of or editing won't systemic you're occluded because approaches the lung full our other patients different, surface to just sure quite correct is nucleases, editing away. organ. you through, think disease, they're And we're an further large of XX% have triples And already we we is I gene are Because of then technologies, to particularly all

who impression We're patient it. So editing have We're longer-term coming to We're hard is give because of gene that a population committed doing stop approaches, gene types problem. those absolutely is that's patients working want don't codons, the you quickly problem on that to doing I a therapy. treat. difficult that's it an easy but to and

asked, is to with done going also wait after think we homozygous we to and the question, do triples, to patients of whether het/min part were answer You first have no. your that to the I the

simultaneously move with day patients intention those timeline, from forward is Whether benefit Our month the to triple. week going we of are or all exactly timeline same it's move plan all the eligible we of forward patients. on that the to think to because or

Geoffrey Meacham

Got you.

Okay, That's helpful. thanks.

Operator

Thank you.

Our next question comes of Alethia from Suisse. Credit Young

(USA) guys. LLC taking Credit have Hey, Europe, Young just - is on Thanks question. Securities line Alethia for I my Your Suisse actually. one open.

through you're with discussion, events close drive in second somewhat both each being approval we all? of XXXX, these As to I the going relatively about or guess of want How is ORKAMBI for just any the Thanks. of correlation at to does and other. half think there I kind tez/iva the that potential that dynamic the process think about of

Stuart A. Arbuckle

question, Alethia. Private in the negotiations for be Thanks It's Stuart countries. or multiple are here. I'll one. that ongoing take can

we XXXX principle a which are there success another country in today pleased like As you important Netherlands, reached in well. for Ireland, an places say know, patients to and agreement as And is Denmark. the had with Italy, time waiting just us. Germany, long who've we've significant There been I'm through

in very importantly, country never an prices We're pleased they principle like Europe, the with is the different think for the they with we're as of It's progress they're we the Europe. making same fast a as And matter in. live So across achieving for obviously. reflect that CF pleased value We us community because which ORKAMBI consistent the are in also medicine. transformative we're patient that value ORKAMBI. important countries know this we we would of or the the is what no with

a continuing as with pricing possibly we're and data company, relently and that could conclude Vertex Obviously, to number it arrangements, tez/iva getting is we have because I one disease sicker to benefiting in to as And patients about could allows quickly price as strike best can I we countries access because physicians certainly a they we and CF progress want as inquire of think certainly to we I and be has any triple and sicker So that's if at led are we reimbursement whilst indeed that kinds they're awareness a like because those, for medicines portfolio-type win combination a patients high-level possibly there country available. know reasonable those that about the Ireland. and agreements struck believe those would it's the the a and these is soon for do from us ORKAMBI. arrangements as of filing a obviously of win progressive the the

as with any wants are speed don't agreements LLC much thank to continue we to Alethia with But government Suisse those but into to waiting we'll So to obviously we possibly you. want certainly to a well. (USA) slow are open down agreement Securities as ORKAMBI today. reimbursement more who Credit pursue as because portfolio can, enter type ORKAMBI that access Young patients Great, - pricing and

Operator

you. Thank

Markets. Our next Brian question of from RBC comes Capital Abrahams

open. line Your is

Brian Abrahams

taking quarter. Two and Thanks the congrats my for questions on questions.

combo to secondly, that met And KALYDECO in support the residual the approval triple of FDA made FRT I preclinical recognize but potential ongoing some just that vitro the then combo cells cocktail threshold the off, done you for for triple interactions in instance, for one of once-daily combos, around First can't function forward, analyses the if setting. you've comment path you move and triple same the kinds wondering on for on had comment forward.

do and forward, the take VX-XXX-based. by KALYDECO-based that we that be risk wondering should take VX-XXX you or wouldn't mitigate one just having with move likely mean and you'd cocktails that Just one if forward you both be Thanks. to if two VX-XXX, probably

Jeffrey A. Chodakewitz

just Yes, those good VX-XXX but for questions. you you're very Brian. KALYDECO take, be Yeah. twice-a-day the to second the would one there. Thanks, And be certainly and could Two on of money We is would forward, one of them said right exactly take once-a-day, or reasons the question mitigate instance easy. – risk. VX-XXX for almost would the

about in data your we of to are FRT question continuing are with discussions other So yes, part And our those regulator. have the cells other RFs and that's and and on certainly many accumulate the mutations, then FRT cells, of an with different easy triple answer. the

Brian Abrahams

Thanks.

Operator

you. Thank

of Cory Our next JPMorgan. comes question from Kasimov

is open. line Your

Shawn Fu

Hey, a Cory. guys. on been for the have Shawn quarter. This two, nice I for might. is I question, Thanks if and it's

So, so, understanding first, want I France. kind I'm clarification A to recognition make follow-on. of just a from just sure the of revenues regarding correctly,

year? or to the of of thinking the recent from Now, a quarter that? then does subject regards be to to trial not for the your necessity here intention in get change rules, if change how of event do the that in the And program, then by planned was and results we're gating include into backlog patients? in trial first that new the end XXXX not revenues mutation secondly, triple recorded reimbursement separate next XXXX, the kind would tez/iva online come around so, we And where Thanks.

Stuart A. Arbuckle

recognition. Shawn. question, the Leiden take the can again Obviously, for on I'll thanks and over. try take first So, Jeff the I'll one revenue

revenues. in as we will will in that anticipate prior be medicines still And for to would get from point, that revenues providing be the going have an of reimbursed That France through on January not retained XXXX. receive cash period earnings. bolus France, XXXX, received on sheet starting sheet. balance will in X, in revenues bolus of for the record It that we France, we sitting the we forward our So, go not if At will price point will that we and that through go balance go get reimbursement we revenues sits revenue the the approval on bolus we actually At that of recorded that in estimated still XXXX. line. we do

XXXX but the record to the continue go revenue price through our in without line reimbursement, GAAP we estimate recognition. revenues in on an bolus in change approved revenue function a of the with It's so, or top longer And will for our line.

Jeffrey M. Leiden, M.D., Ph.D.

Jeff. the important a Thanks about. on And, it's lot one. The for is this thought because an we've Shawn, gating, one question that

corrector to is that addition very that in As of I benefit. drive said, well-treated from single we trial enough this FEVX what patients these learned isn't first-generation acute a KALYDECO to

a we second-generation from single could FEVX we lot However, with potentially a which activity these that a patients. allele that first-generation hypothesis acute the more even corrector, increasing FXXX and test obviously drive want well-treated in definitely that drives to

So our to intention those X our is include Phase patients in program.

triple pulmonary to X to this look only to improvement. use really more can that the pulmonary Phase acute the exacerbation, And study include improvement. chronic the antibiotics, one these things the question that trial aspect we from talk see it would mortality, the improvement That our know, between just because you improvement that I of because at The Just disconnect acute curve, of we about. want a going cetera. we see. improvement. very on chronic improvement FEVX clear, the is to like program, the whole important be are really CFTR regimens, as corrector But decline said design you fundamental can't there's we there's acute of I want you equally to and question, the the different of But separate really important, the a comment function et obviously gating in that be even remind trial. things hospitalization, somewhat in don't learned a answer patients you of of transplantation, trials, we've absolutely, produce And which our the you slope is

for see in in KALYDECO, is of improvements And that's actually medicines double-digit X.X% similar just exacerbation, we of terms cetera. chronically, with two But we you So, acute slope the see long in know, see X% these quite ORKAMBI, we FEVX; a between improvement acutely. decreases in when to decline, important what about really, as pulmonary look really run. over et of we patients the

could adding levels. the FXXX So, patients last alleles important them a do to get better more what these chronically single in a test We those FXXX will lot to activity acutely we do triple can believe or and it trials. want we we have activity a is closer ask, of maybe this thing know want to these where correction do now clinical even placebo It's could time more compare drive to with lot carrier simpler of off prove kinds because patients but we we really drive or trials double essentially more we hypothesis, we of treatment. the the by to closer that, the these in we and as some acute them

So, we in those will program. Phase the want include them to X

Operator

you. Thank

Co. & Cowen of Nadeau Phil from comes question next Our

line Your is open.

Phil Nadeau

my taking Good afternoon. Thanks questions. for

revenue housekeeping by region? First, just and down ORKAMBI geographic one you break a Ian, KALYDECO Ian. for the could for

Ian F. Smith

pass over to Stuart. I'll yeah, it Actually,

he at has his the think fingertips. I amount

Stuart A. Arbuckle

I do, Phil.

U.S., in ORKAMBI, and million we in So, it $XX QX. ex-U.S. million $XXX million U.S., $XXX for the $XX in had in quarter was for And the ex-U.S. million the KALYDECO,

Phil Nadeau

helpful. be first X in that had past to Concert you ready Then question looked move but you year, very second next be wouldn't that half be the Phase That's the at forward may the was subsequently. development, suggested on that something to molecules into the of triple in going

gives moving the of what the months X forward the in first few So, of half kind that in year curious programs. in Concert's molecule has the confidence changed right I'm over more Phase you last initial

Jeffrey A. Chodakewitz

accumulate need to Phase done in in announcing we're programs. comfortable it what and has we've basically programs. that we so. include to would working program We've been changed the X these have hard the feel including today that Nothing Phil. feel is on we in really it we update Yeah, except data And an

we of think in to include and it make or allow regimen. And which us whether so, to do the not year first next decision with of about half will kind that it

Jeffrey M. Leiden, M.D., Ph.D.

lot into opinion. the would the allowed prior to through just were get Phase closure. a and unsure X add we Hart-Scott-Rodino around communications to gather in would potential XXXX. and, and therefore of get did to us X of early it's do review with the that I X when decision Phil, transaction, VX-XXX And, the was that in with and our We inclusion speed We Phase positive going Phase to and the programs were the internally therefore, quickly close we'd make that, incorporate data when a move in that

Phil Nadeau

X on Great, and programs. then one last question the Phase

the to months patients to course the safety package way you significant minimum Do of used data therapy? of precedent a between differentiated amount are an to out need you're the acute maybe acute spur make chronic drugs safety try own. the effects. their on the is for the XX supplied of that's though some including going enough it treatments or You've the to the be I'm to on side, some to approval FDA could there case drug the And patients in chronic what's like potentially of safety and be of effects for going beneficial a that that are curious chronically? kind effects for a number seems to shorter that

Jeffrey A. Chodakewitz

with key identified So, all of discussing you've Phil, what exactly the FDA. that's questions, we're but the

you about. All that of those talked

think lot the we It's give questions. trials seen those have finalized, don't until learned those these from I early regulators you the because just of to who for particularly our me patients any just anything each we answers us trials. too the allow to for X. from But we've Phase yet today. we've on a data all have specific those agreement don't streamline It have we As said, from will discussions

Phil Nadeau

my taking for thanks Sure, questions.

Operator

Thank you.

from of comes Citi. Karnauskas question Robyn next Our

open. line Your is

Robyn Karnauskas

Two questions.

really First on, stupid. maybe

for to when your big-picture think that. you do know, Thanks. become them for moving your and But – a good their to So, question second launch anything more a is analysis, your why in forward, you taking question the is besides you other thinking that focus on products? your would all on while you And But a big company? any these and throughout do a start you've for things are there think on know and two else getting communicate take both sort look you two strategy diversifying combos doing to of focus both is you How products you'll to XXXX. mentioned products they final reasons triple the like you deals that up strategy risk-mitigating been pipeline, forward the talk about forward? pipeline. to then when of And about will I that? about these Street working Ian, big-picture I if all things us?

Jeffrey M. Leiden, M.D., Ph.D.

Jeff. it's Robyn, Yeah.

right. absolutely So, you're

it one number and the triples we're the for about for reasons, on move We're can. obvious that Our talking just mean reasons forward to patient we we are. as as focusing doesn't That pipeline, focus, as much. quickly is not non-CF not

is risk why take what a just Your question said. strategy. And forward? it two really portfolio you It's was, mitigation

hate know, to by the in We two year a about And back any are as you regimen multiple Phase one waiting get we for take of well as because these have kind efficacy, patients both is forward, would X As endpoints multiple we regimens. and for safety. set Phase or surprise X patients therapy.

I one mitigating you feel would – but the to We're patient everything are were programs our is today, And actually. of really that if risk think as the those nicely. and So, just the by With And, to when at some clinic to I to intention of on of is moving sites. forward it's driving major to patients. pipeline, different one patients depend there programs into because to year. very again, assuming them if regimen, in XX% expect forward treats show Altshuler said, David taking just regimen going we them that certainly, yes, confusing clinic, next begin launch multiple pleased. pick them best there's data, differences we and we'll day, bring respect we into be talk very two the I populations, we're launch forward, less regimen think the to data. ask end And more some of bring no about significantly, begin the the we you me at would for the

to of us hear you So, expect should more these from in about several XXXX.

Robyn Karnauskas

thank Great, you.

Operator

you. Thank

comes from of Ying question Merrill next Huang Bank America Our of Lynch.

Your please. question,

Lilian Wan

Ying. for taking Thank on very Wan much Lilian is This for the you questions. for Hi.

have I a So couple.

The persistence compliance first more and for patients Thank given from and you. the to do the to number impact X you Phase is lastly, is grow? trial? X of do guys secondly, have any question for And you that of ORKAMBI? see you what pretty seasonality the a Phase enroll think the or Do is, is that strong, XX? regional the data minimum required triple any X into you (XX:XX), between the rate numbers ORKAMBI inventory have room And

Jeffrey A. Chodakewitz

come didn't could we got you the we through So sure so make questions. very clear, if just

to was, grow. have question first the does Maybe... So ORKAMBI room

Lilian Wan

room that patients till do just potentially wait they to for market? of age wondering to come have you patients, newer and more or for still I'm you just could from persistence to XX. grow, It's X therapies think compliance particular do the subgroup

Jeffrey A. Chodakewitz

Yeah.

that take one. Stu Okay. will

Stuart A. Arbuckle

the very in which patients tend patients, levels on respiratory And It these to compliance anticipated, the of that as we to that are higher older of largely the did group parents as younger X actually in adverse the this because studies low. based was in we that the very XX patient what And rates clinical population we've are we them in levels U.S., dropout their the age which quite seen, seen persistence anticipated. quite compliance we profile events in that high, seen we supervision higher. and than and they're group. in both here we've and high be robust uptake under So rates that know were patient where the was we've in saw

the see I XX anticipate In penetration X don't reached in further terms U.S. growth grow I ORKAMBI both to awaiting because the here to us in we population, we've we're an outside that of peak indication in yes, that that of that patient give opportunity think are further in that group. also approval growth, And to U.S., and then yet still obviously, of further population. will the going

or see terms summer impacts any for we experience seasonality. of didn't ordinary really inventory second Did see thing compliance months. of this I was was inventory ORKAMBI in anything seasonality out question QX? QX didn't during had We that think impacts last we dip a in year your year around the QX. we with in experienced the One

QX, of the in see number of place, I we such that and compliance learned we why think we ORKAMBI why and the strong happened. programs any of put was dip the one didn't team The lessons that quarter. reasons in saw a with demand during

Jeffrey A. Chodakewitz

your second that triple? question patients the are there your was, question? of trials I clinical Is enough for think

Lilian Wan

thinking I'm about Yes, RA-wise.

Jeffrey A. Chodakewitz

experience had with the now enrolling these triples, that get there the a is and only very, strength into Phase of seeing demand patients, to confident trials X very results enough het/min very, regimens given are of trials but patients, that these and the high. homozygous not where clinical in lot we've So we're our try the very we're

example, patients gating I it so you patients, is, other the recruit are difficult a because for treated. more population as patients The in as the has you to enough I think the would trials, those in note KALYDECO-treated are little been there well well. know, only to thing

slow not down to that that's when those to get goes But in to come going convince We'll certainly more convincing. how sort back little them And so regulatory takes see we filings. of to any patients. a

Lilian Wan

much. Okay, you very thank

Michael Partridge

time I So, questions. more we have quick think now two operator, for

Operator

Yes, sir.

from Walsh comes Adam Stifel. question of next Our

line Your open. is

Adam Walsh

so thanks for taking much question. C. for my Jeff Hey, This is

On ORKAMBI should if any the the that study. ORKAMBI just that's how ENaC we assays might and would think Thanks. ongoing, with wondering earlier work. therapy primary the not whether inhibitor, of have there's we read-through study I combo combination or think think performance assay the from dyskinesia there any read-through your ciliary about to the suggested I'm

Jeffrey M. Leiden, M.D., Ph.D.

Maybe Jeff I'll is take that. This Leiden.

can ciliary the with beat we ciliary way read chloride just layer the X is make which The ENaC the the a can through doesn't do question we're adding the chloride that trial, to to benefit sodium layer the do I'm again, through direct So think answer frequency, through And so, the on and is to any you like HB that ongoing. primary And HB which can't dyskinesia? transport. mechanism. first, of best assays primary well. when layer typically increasing assays, the as of we a that our we're question CF, was in pretty are reasons inhibitors those isn't the ENaC finish, kinds in have we is, doing do we that question the in of you And the but I we ORKAMBI. do ciliary is patients that does frequency in about in pulmonary theoretical it just why the in we'll think looked It the and is It function to remind disease sure of ahead height a role in a Phase really dyskinesia? answer does works enhanced definitive fluid work the that comparison. the not that the that us with the trial hydration taught reason CFTR activity addition How seeing improve is, clinical there transport assays ciliary to after did fluid there ORKAMBI I do of of the to and complementary only think therefore, inhibitor ENaC obviously population ORKAMBI, transport. to increase do And of modulators. trial beating. But went that

Adam Walsh

finish? that When will

Jeffrey M. Leiden, M.D., Ph.D.

enrolled. you enrolling little we'd It We'll enrollment on get there. we in It's how and bit like timeline. know when the the and that out let plays depends complete a see it's But now finish how to well. understand

Adam Walsh

much. so Thanks

Operator

final our And question of from Butler Securities. comes Guggenheim Tony

Your line is open.

Tony Butler

that triple Briefly, if and to total when for period But and did single be reveal would much. to file, the very move forward the some out just on some populations. to medicine may once data data in Jeff, least to sense selected makes yet the alluded some I Thanks medicines, there of bear you triples, triples the among you a think – better come back all and July FXXXdel notion longer data that term necessarily And longer than study. patients, look better for the term distinguishing on others not at be het/mins. in homozygous that early looked which characteristics some seem time did

are upon fact, more but decision very may curious Thanks more sense. that not there populations hierarchical just in I'm based is importantly, that make if So only if, correct much.

Jeffrey A. Chodakewitz

question. Great Yeah.

parts. me Let take two in it

all and what through my just pretty First respect in both give on would with the which between have part X to second all make and of decision a distinguish is, our don't will, XX% the And to to the be Phase population. Having you we'll idea. were if see view upon probably those that robust believe different hopefully or triple data. and to that all, we And is the give decisions close data, said will launch really absolutely the we or distinguish Phase you They distinguish X depend is given the that, which regimen, side. one us the We enough early of a to is that size hope, the FEVX you data Phase that good combinations X% to and can data and distinguishing not of it's actually the all and allow a efficacy those get of between clear trials, best not right the may like final may range, safety X of and in your double-digit the yet quite trials trial once regimen. way hope, the see. will ability a course, us is question, we'll

of the launch between always of in best would different reproducible regimen say possibility we and significant the populations, the you we As population. course, or differences it, into saw each that the regimens chance different

bit do. it see, crystal just of you depend what will we what but a I'm gazing little like So, on on ball give to we'd to trying

Tony Butler

Thanks very much.

Michael Partridge

and Okay. office Investor be additional this now may team to Thanks, is disconnect. the Relations the The This concludes you if for evening. us going joining questions. have call, in you everybody,

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