IONS - Earnings call transcript Q4 2018 | Ionis Pharmaceuticals, Inc.

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Operator

Good morning, everyone and welcome to Ionis Pharmaceuticals 2018 Financial Results Conference Call.

As a reminder, this call is being recorded. At this time, I would like to turn the call over to Wade Walke, Vice President, Investor Relations to lead off the call. Please begin.

Wade Walke

Thank you, William.

related note pro are financial forma exclude compensation please in using results begin, non-cash of awards. place when expense we that now we non-GAAP that Before equity to discussing our is and a results a our the not in description change the This of merely adjusted calculation. in change

that including to non-GAAP to find and release and the Investors I will business. a of our of to section we tables, GAAP reconciliation always, measures We we how the As financial go the today. encourage financial press better related everyone manage results business the economics believe Ionis to discuss financial website our of non-GAAP represent

Damien contains Officer; like I Business Chief Stan Executive website Chief which have accompany on X, today. your our Crooke, and who posted Officer; Monia, Financial draw the statement. Officer; Q&A. discussion are to me Chairman McDevitt, Brett and for join Board would Beth will on We our of also Hougen, language Chief Chief our forward-looking that slide With call us slides to today's Operating Officer, attention

call We expectations will statements additional to beliefs. statements With begin actual our subject which to for detail. this differ uncertainties the I over results the consult are Factors Stan. that, and certain and and to risks These our Risk filings our SEC on may making are based you I'll in call, materially. forward-looking current on encourage turn discussed

Stan Crooke

exceed of we And us an today's enabling was sustainable profitability. XXXX, consecutive in of strongest With our our XXXX income non-GAAP Wade, income, every growth to-date. and everyone. our enabling are our we achieved Thanks, operating call. XXXX in financial Today for of our revenue business, morning, operating high goal you us realize element on third year good all-time to Thank to long-term position us joining substantially in in guidance.

our performance our lives performance, pipeline is our activities our compared with in to a these patients invest medicine, advance continuing well as continuing from the changing in as continued with results to major to and royalties while driver of to of achieved strong SPINRAZA's than be first-in-class investing SMA. XXXX SPINRAZA strong we in blockbuster doubling commercial fundamentally medicines, Importantly, XXXX. of SPINRAZA a more financial transformational revenue technology.

PTC and We're patients' in to Akcea the positive the are both with accomplishing priority hATTR globally. marketing last through review. for PTC partnership year, America. in TEGSEDI filed to and the these the early, of U.S. and live available We continue for TEGSEDI We're physicians SMA SPINRAZA. treated to is with care efforts to they the thanks patients stronger launch launch, a affiliate quarter of do SPINRAZA experience, with of over of see years longer thousands to off that better grow patients generated the of from the and longer, the Akcea. treatment first start, standard with people And global on EU. Brazil, in excited pleased Therapeutics and coming part SMA. with make both TEGSEDI are real-world is for who on are in sales partial so our with TEGSEDI patients revenue landed million strong which TEGSEDI's the the Not in impact Latin of $X our in In feedback from that about We aiming U.S. Late and life. quality patients authorization EU,

potential the is with hATTR TEGSEDI. largest in populations patients, for approval important an region a of one of As Brazil milestone

by in will numerous XX beyond. of drive we potentially that have end or underway of pipeline than medicines XXXX of for pivotal these trials four at XXXX. And expect pivotal into we growth advance we with programs more have to Novartis of million least disease to believe targeting medicine APO(a)-LRx, potential transformative XX, our the of for Monday, is ALS, more medicines have all We -- these when APO(a), licensed end the they and Today, to and TTR, across On which These before we have all include neurodegenerative earned decision advance from Huntington's this medicines already targeting additional platform. value-driving the this and catalyst medicines the LICA have of our Akcea year. year. achieved Novartis this And an APO(a)-LRx. validation LICA we $XXX medicines important our significant

the populations our for such demonstrates people are moving LP(a)-driven we this Phase After initiate Huntington's patients the Akcea in game-changing with X of the initiate in LRx in Phase from SODX of patient milestone half on with TTR data mutations meeting based millions year. with potential this the of payment rapidly large very to advanced the treat medicine, first from earned of the LICA to with the cardiovascular Biogen IONIS-SODX as ALS and FDA, As to million $XX Phase APO(a)-LRx disease. study most technology enrolled disease. study second and Roche gene. first the study Akcea We IONIS-HTTRx patients positive in the the program X/X we as patient a in they pivotal which the in licensed X plan

therapeutic in global progressing technology of with are pipeline expect we data from roots of numerous diverse invented. have business large therapeutics of fronts. and Phase areas and many a and successful patient we be applicability efficiency and our to by model, very our coupled And our targeted Phase broad X Ionis medicines earnings start the the versatility could we on across addressed while this studies innovation broadly representative our year. X exciting are at of next continuing technology grow which addition, administration our of number pipeline In year, because revenue executing In many and of to populations multiple the leader of that RNA an

Beth I'll our provide to Now, the to update. financial turn over call

Beth Hougen

everyone. Thank you, our in we by Throughout demonstrate million morning advantages to of model successful continued XXXX, $XXX the Stan. Good business generating revenue.

than profitability. operating deliver XXXX us which The was due income revenue doubled to XXXX from increased enabled more substantially large in significant revenues commercial XXXX. of to compared revenue growth compared operating in $XX to of increase exceeding part SPINRAZA to our non-GAAP million, XX% Our royalties guidance

the expenses finishing operating range non-GAAP non-GAAP Importantly, because of novel cash results activities pipeline by substantial and our million would in of achieved and our technology. all year I'm million of model. low $XXX met operating million. the year our in just $XXX we business operational of and continued execution end XXXX advancing be pleased these Last while say We guidance commercial our expenses. and the efficient guidance to balance, to investing technology increase range that we to provided with our the below $XXX

compared to with investment we a our TEGSEDI $XXX commercializing due globally. XXXX, net Of particular expenses to of in primarily GAAP on note million increased income Our ended XXXX basis.

the tax non-cash believe that tax is to a we recorded income. our offset assets. be due large these primarily future it was income net with to reversed benefit portion likely of our we the We when use associated substantial deferred one-time we to assets able valuation Our taxable will allowance tax

and net confident royalty regarding we XXXX, our the important strong earned financial right driven that U.S. earned take the nearly million million double step outlook this approvals of adult few revenue growth is the billion business. SPINRAZA the and continued outside this We time more growth $XXX the Worldwide markets SPINRAZA, in results which our by In of and in segment due than years XXXX. in past estimate the adolescent in in we new the XXXX, to sales are of of sales over grew was patient U.S. $XXX from from $X.X to to our

commercial fourth the last clinical trials. of globe patients including were X,XXX quarter from treatments As SPINRAZA patients as over year as expanded the program SPINRAZA patients on well around access the there and

was over these countries reimbursement Additionally countries. in place three nearly of all with as approved of in year, January this of XX in SPINRAZA

first TEGSEDI we profile Stan patients treatment. as treatment. U.S. encouraged hATTR East, absence associated Looking the Biogen and our and patient's, sales Outside Akcea cost additional with the Biogen are markets, TEGSEDI begin SPINRAZA running. patients and marketing are the PTC XXXX, adolescent mentioned, and payers expects being Middle XX% now in Latin only the up be these And billings make to patience to China and treated all EU Brazil. growth. revenue worldwide well largest as source responding continues the up other successful approximately continued patient treatment. but treatment. in are Older growth America U.S. on sustainable patients more patients in expects with now segment, with of opportunities Asian adult treatment the significant to Latin self-administration drug represent as patients Akcea The world. of aspects manage access and revenue is Germany TEGSEDI and program new the with on to helping In are expanding Akcea from the priority to are expanding We And across physicians the and that under to $X around Akcea's start launch are collaborations the subcu in and quarter million R&D growth. strong their in first In we physicians Canada all of for a partnership to us. review and more Connect TEGSEDI's sales of than for Connect application as in is TEGSEDI America, a of markets committed U.S. significant is off market. favorably Europe its In

a Biogen Last R&D IONIS-FB-LRx. $XXX including large progress XXXX they which our managing Last component portion of approximately per our approximately do we have we earned Roche expenses collaboration to to In funds, fees $XX our directly upfront to fall prudently numerous represents million our from XXXX, as recent R&D million our of to million expenses. quarter. strategic we our amortization R&D line element $XXX year project leveraging this of work $XX with revenue. R&D earned profit year revenue in from we bottom earned with for million revenue. and by the $XXX when non-GAAP collaboration licensed Importantly partners our year our our our partners pipeline, million revenue from Last and of from payments medicines. significant License

AstraZeneca this clinical from in fourth to $XX our for revenue R&D earned payments And from partners, them. we our our million medicines In quarter, than is we is they licensed payments $XX revenue, significant commercial from the Biogen we R&D Milestone For XXXX, and component from milestone earned to earned for payments. last IONIS-SODXRx, earned In of treat with people revenue. more we another reflecting component we advance Last as our fourth advance. medicine million earned the of we from we manufacturing from the partners our million $XX ALS. when of our services create example, for in $XX component value medicines. nearly million the first generated primarily supplies novel two milestone perform quarter, year,

growth together by in revenues. Now turning commercial to ranging increase XXXX, operating the and SPINRAZA our this sales for and in year. our We will R&D both guidance that to driven with Biogen's SPINRAZA product high TEGSEDI revenues an this teens, product for anticipate with mid sales. revenues revenue grow expect growing profit Consistent in from we year,

anticipate growth also We R&D in revenues.

consecutive $XXX IONIS-HTTRx. million eight are we're growth. of advanced start, We making they XXXX a year revenue III Novartis the more off AKCEA-APO(a)-LRx the Roche our we of $XX from million, to when strong revenue program projecting earned with the $XXX we this earned than for when already licensed Phase million and And

and invest are $XXX million our We the non-GAAP in million, in plan $XXX to $XXX million our range TEGSEDI to expenses to the continue of range R&D We a SG&A projecting $XXX technology. commercializing basis. in pipeline on million and to both expenses advancing of and

this In consecutive significant growth than non-GAAP are revenue with income strong fourth expenses, projecting and income. $XXX end our potential of modest increase we year based operating year on more in year of making operating non-GAAP this addition, for million, our to

profitability. we last step. several Over the operating the sustained have And next achieved years, taking are we important today,

while line this our us We and from described, expertise enabling invest leveraging on technology power performance, business The and on year our and future. results developing aggressively the together coupled basis I projecting to be into resources. are our bottom of have model, with and just the commercializing the medicines, strong non-GAAP partners' financial profitable to efficient a our

also cardiovascular AKCEA-APO(a)-LRx than consider that commercial patients more represents. and A outcome cardiovascular are global example, is study Lp(a) substantial with that conduct million disease. There Accessing large organization fund. worldwide market will opportunity driven this will Novartis a the For required. X require

AKCEA-APO(a)-LRx medicine to AKCEA-APO(a)-LRx of reasons, good provides patients of success for most in is to participation these the Further, choose For commercial Ionis' partnering the route need. for type which partner. a efficient the our example medicines in represents we optimizing

tiered our to the to earn $XXX AKCEA-APO(a)-LRx, $X end cash mid-teens XXXX our on In will low royalties the $XXX payments $X invest we from billion addition positioned paid the doing we to pipeline. license can range. with Novartis With million up milestone XX% project to us are the so, well and end million and in of in than In ranging that in of commercial XXXX, fee for we we cash. more at approximately billion technology sales

We create patients are to potential substantial confident our value investments near shareholders have in and the our term. and longer the for

clearly exceeding TEGSEDI, peers. we us to while financial our the to beginning our achieved advancing ended $XXX take this And and aggressively technology, date financial summary, achievement from investing pipeline significant continue to our that demonstrated strongest substantially Akcea apart portion Novartis momentum. in guidance this by choice also plan In our and We fee of our our Akcea XXXX with in launching in year position million our sets we XX% strength investing license an from stock. XXXX in as

the to with strength to I'll the maximizing continue deliver and our patients update over on provide both and we financial call Brett turn the pipeline. have to an that shareholders. our flexibility value We And to

Brett Monia

Beth. Thanks,

achievements, eventful you this successes see our can As year was throughout XXXX slide business. numerous with the an key showing recent from

added performance our Beth we growth drive of strong. sales revenue financial to continued its discussed, was quarter SPINRAZA As launch. from from just first TEGSEDI and

successes and us across our regulatory meaningful in year beyond. Now our XXXX next and this clinical the year, turning positioned achieved we for catalysts pipeline, to pipeline numerous

AKCEA-APO(a)-LRx, of IONIS-HTTRx pivotal and discussed, have year four including expect in end we the to least before As AKCEA-TTR-LRx, this have IONIS-SODXRx. we at medicines programs

let me each of potential with you has to Now address of AKCEA-APO(a)-LRx the the elevated Lp(a) caused disease patients brief a on update these. millions give cardiovascular by levels.

study hereditary of polyneuropathy hereditary study by amyloidosis, initiate outcomes the Huntington's natural in preparations already to with X half a And Last well Novarti's planned targets underway. also with Phase wild-type first year. began so TTR patient open this label for patients to second and moving patients also Phase pivotal month, Roche The medicine patients study Roche the data from for that We study is initiate TTR this of the complement X to while directly study underway AKCEA-TTR-LRx program medicine plan study and the the in to to in that world's will cause a study closely cardiovascular X for enrolling to continuing medicine X next-generation X/X to the first followed study we measure rapidly IONIS-HTTRx. study Phase set year. of disease. ongoing are the outcomes Phase our patients cardiomyopathy second we are to pleased with for in The patients conducting a this robust as Phase serve extension is Phase a possible. initiate which history amyloidosis X a are study get in as generate rapidly a TTR

plans from year. Roche extension Additionally, this report to study the data open-label

with the well demonstrated pipeline study Biogen to ALS. this directly encouraged patients diverse exciting lowering to Phase to study. the functional plans have with by for IONIS-HTTRx trends disease, cause this treatment the In as AstraZeneca rapid have and revised. the fluid these head the patients and X/X of decline in positive queue by Phase which towards of in we slowing in data ratings patients to that clinical as months is disease a levels first exception. X significant reductions associated ALS. meaningful program start people demonstrates with large complete Huntington slowly SODX-related three ALS progression. the December, study ongoing licensed of targeting protein enter a understood scale we statistically spinal ours, disease we study medicine neck the Huntington's only on substantial a recall with additional cohort and lives results, after an data SODXRx potential impact advance danvatirsen This registration of this year is with anti-PD-LX to the in With SODX on the ALS X Biogen X/X potential track adding measured clinical of was to SODXRx from as on in benefits with no see combination associated IONIS-SODXRx. cerebral of always in is based and Phase a months, expect stream in and slow fourth steady the our halt Phase anticipated to a the new and with supporting potential to antibody or And In cancer, with this danvatirsen the potentially putting durvalumab study may perhaps You coming progressing events X year. XXXX on of Phase in Based were patients

as we addition, mid-year that study FactorXIRx from late agent completed year with the sharing of Phase this forward IONIS-FactorXIRx looking selectively disease study intrinsic in an data also increased end-stage data this a the In from to volunteer novel year. a X to separating first we're to looking renal value patients follow-on enrollment and risk of around in last of potential is the targets anti-thrombotic from the demonstrate coagulation LICA We're IONIS-FactorXILRx study the also X activity forward pathway antithrombotic bleeding. of Phase the strategy the midyear. healthy

one with In Bayer forward. both review the has is opportunity Following in the forward. these regulatory continuing or currently these the path under FDA EU. to we're confirm U.S., programs WAYLIVRA of to to work a studies, advance the regulatory

from WAYLIVRA X Head the later events In And pipeline. summer, translating second Stan beta company. into the now our thalassemia host coming I'll host focused on expanded quarter, to a opportunities. investor well. we're year, which in And year. in the the Frank we months, data Phase the FPL believe details lead we programs we a to how therapeutic of turn will call important We to of Research around in data this Watch will will cystic advances close recent into middle for call. both technology report our later as the medicines Additionally, And or from back neurology technology And the familial better-performing of portion insights webcasts. of lipodystrophy our in soon. with continued to Bennett, fibrosis future this patients plan into this to advances study this report acromegaly Stan be announced partial of to the our webcast plan and deep-dive provide we the two participate you over to are in and demonstrating invite planning

Stan Crooke

Brett. Thanks

XX been innovator For therapeutics strong confident now be leader the foreseeable we Ionis and that foundation has Based that years I'm innovation, extremely the efficient a RNA-targeted technology, leading to yes. business on culture the in have of we'll a and for with our novel continue built future. model,

We are value profitable our history, sustainably now maintained a Throughout innovation. patients delivering to to and shareholders. company our we've multiproduct commitment

ever. In TEGSEDI greater at advance in drivers more as advancing can demonstrated our We've technology, have opportunities. value. commercial SPINRAZA is resulting better and fact technology, in transformative apply those of our we rapidly near-term than medicines Because rapid that two the represent today, of we technology to and pipeline advances today that pace we a advances performance our our commercial

but to not pivotal Most than confidence at earnings of medicines advancing XX XX our development, our just due patients and today in for our the end and position our and our least in to four we're term more And the commercialize we to importantly, position we not wherewithal in also the be successes our in shareholders increase value trials have deliver with to successfully programs for just near mid We pivotal in -- expected With the excellent to cash continue revenue future. develop to advance financial to an year. this medicines late-stage by business and the can and that financial we the gives have longer have technology. to strength, a foreseeable us term. and continue medicine healthy

We to set are up our of transformative developing recognition tomorrow. advocates open who Rare we hope for treatments you'll want families team In we I serious to William, to to rare diseases. patients, disease thank in mention rare turn wanted call celebrating for and with please? awareness Disease for rare Q&A, And join partners that, event, call Day if the have this Q&A. been the Before I important the community celebrating I'll that with efforts tomorrow the our you diseases. our in Q&A tireless over us up for these can raise and and for devastating --

Operator

sir. today be Thank And questioner will go & Instructions] you, first with Needham [Operator Chad Please Company. the Messer ahead.

Chad Messer

and SODX question more XXXX. or Is My patients? that congrats a should Are scientific from Great. we might formal about it expect? this additionally great Thanks for expect -- taking elsewhere what at just a What to question's follow-up? and my there more hear we year? on

Stan Crooke

should course as as information thanks, target benefit reductions, and What that think option I this you suggests and information toward Biogen of Chad. Well license rapidly expect led to course preliminary that to SODX possible. is registration move safety on exercising of

Chad Messer

Okay. Thanks, Stan.

Stan Crooke

You bet.

Operator

And our next questioner David Morgan Lebowitz today Stanley. will ahead. go Please with be

David Lebowitz

question. on you an the much us them right I we taking discussions can guess, expect when now? you my you -- for to very Thank And are they make status Hi. where update? on the Could WAYLIVRA, update of

Stan Crooke

conversations They are able that's really to very both in say fully The all engaged. are are sides today. progress and active I'm and

David Lebowitz

us for that. the over outcomes you for trial? you And design to through run jumping APO(a). Could potential Thank a Sure. just

Stan Crooke

Brett, do you want to?

Brett Monia

Sure, Stan. happy to

the So now. putting that obviously design David, touches of the final is study Novartis on

be to the details type is of study that will Novartis Vascepa. that look outcome cardiovascular exemplary more be One previously, that and we study in before, that authorities -- some presented But details. will that as so this this had for It's you FDA study study As pursue. is the provide be later But an and REDUCE-IT a mentioned the -- near study might the very size meetings in we've and with exact be -- may describing EU, place. outcome study. regulatory out well example they will is both the good we've future on year and may study Obviously, in coming for the later.

Stan Crooke

is had lipid elevated prior a patients other do have who their who control. Yeah, in study and cardiovascular that risk under event LP(a), this have factors remember

the studied in XXX-patient such this the study So, precisely we results. our patients that impressive resulted in are in II study patients Phase

So prevention population outcome an represents study some number are Beth differences study. a mentioned that and has primary X study, prevention patient a those obviously as the -- that then million and secondary from

look Novartis the with going study some that a patient it’s in have They've context. outcome ready to first quite in is Clearly, at as that enrolled. get for number for large of place been obviously a get a go. So take it team. time preparing good to an study, this And months to actually and

So Novartis beginning ahead. that we're looking exciting forward to as moves

David Lebowitz

Thank very much. you

Operator

next the go be And ahead. questioner Van will today Tyler Jaffray. Piper with Buren Please

Tyler Van Buren

Thanks morning, questions. for the taking good Hey, guys.

respect pivotal us bit we to The with hands, the program. SODX registrational guidance your first any it's of sort could of one out I little Phase can rough of potential but a was and understand receive Huntington's data? III give that you program the when

Stan Crooke

they that. think Huntington serve as that is registration III I much XXX I basis said -- substantial runs data. for and history have more a they registration. I -- as meantime, study think, which a very in might they the Recall extension over parallel I study Phase long-haul and patients Roche meantime the next correctly, if continuing a hope have indicated think think that Well, year, in believe for plan the with, the are Biogen natural large registration. effort will provide we that performing mounting study open-label very and both that And both I as early recall And obviously, Roche early

Tyler Van Buren

then franchise, And like see have respect forward That's And first what with to to Great. can us and the Phase in pivotal? And TEGSEDI year a helpful. and need in could I U.S.? Brazil in see relative to approval? the Europe that you the the move we opportunity broader later -- tell to to TTR-LRx could look TTR to when program what we secondarily, that

Stan Crooke

with a any this to I we will answer second certain I -- is There that Phase effective But based source think, of you drug as opportunity, X. the And say, absolutely Well, Portuguese certain are quite is and that. nearby to that information significant, size regard that refer would primary very the I Latin on migration. potent move of isn't course, the question, into the Brazil of information because question. America I Akcea in to have, countries population about the the of

in I a mind. is timing and our just proceeding leave would And And registration the so, opportunity it's there. market quite the significant

more I you you Akcea. our then detail want talk If suggest to colleagues at

Tyler Van Buren

Great. taking Thanks for questions. the

Stan Crooke

bet. You

Operator

our questioner Baral with be Cowen. will today ahead. And go next Ritu Please

Ritu Baral

taking potential on Huntington's Hi, question file up accelerated original the I'm comment and Roche's on to going guys. follow Thanks for and Tyler's like. which accelerating a un-Roche question. very -- filing is

extension I think just strategies. two functional could would history ongoing trial data that One, history the open-label the there the natural be that open-label end additional the extension data And also biomarker there, is the from speculation be X potentially, make that to comparison robust be concomitant started, of the you over but more on Phase filing Stan, data? data XXXX. as clinical mentioned from natural from year? Could by data

Stan Crooke

Well, two, pursued. being correct. the is XXX% conjecture There as are as

study history spinal mentioned, significant seen that contemporaneous we've encouraging serve reduction about of clear Roche HTT, is, to is data. in the for we in quasi-competitor look at the fairly been the encouraging And think behavior with way open-label I consistent have extension And as study. has the that. that. -- that and of I'd Roche think with I And fluid trends the natural is benefits. plan That's as a in

the comments could then of you and your question. answer part take I probably think So those latter

Ritu Baral

the for paradigm, wild-type helpful. cardiac X after go population. the the right U.S. hereditary the the where it. And now? potential just treatment are design that change And That's TTR and that your approval mentioned, you then, how Phase that might both for How tafamidis Got the thinking a a in the follow-up question paradigm? like you you would timing, and on the design about given

Stan Crooke

I'm going It's space. to a that not competitive question. answer

plans appropriate competitive time a this We manage about highly those are to we and will space. at excited that's discuss our

that's know we We have -- so obviously coming. planned for

Ritu Subhalaksmi

then. Fair enough. going stick at it. substitute to Got right. All a I'm

If you could--

Stan Crooke

Give I can me answer. one

Ritu Baral

the through emerge year you that CF of us bit nature maturity the should the programs? those a promised we program later of of the beta-cell you -- Can take that said from little the and data, time -- on acromegaly this those data data some could get what's

Stan Crooke

and evidence of benefit. reduction Target

Ritu Baral

three? it. Got all Across

Stan Crooke

Target benefit. yes, -- is I with question that's that reduction a and answer. some can't Yes

Ritu Baral

the taking for Thanks Great. questions.

Stan Crooke

Okey-dokey.

Operator

And questioner today our with Paul will be Stifel. Matteis next

Paul Matteis

I know a potential talk then identification do with of more you the today from And have wondering rates? incidents you SODX? the about market about there. was path of are SODX separately On sense if any patient opportunity do just we where we what could regulatory And perspective

Stan Crooke

I can't regulatory go on than into we more Well path. have detail the

I with that think chat Patient pretty just study you're be easy. to enrolled and going be identification proven rapidly. to very is have The Biogen. so For far patient to

know treatment. seek these who they a are have So, to are patients motivated problem highly

mutants issue at and all And ready identifying has there the sure sorts -- Biogen it hasn't enrolled things of out in small and in identifying that. fraction, trial, but to-date, for so getting the been there don't patients. you of regard But represent are with participate And market would be very actually expect patients exact. where remarkably the I rapidly. including the to SODX going will an least been forward really those clinical they as remember Clearly, moves and a all

Brett Monia

patients. X,XXX

Stan Crooke

X,XXX About tell my patients. patients me. colleagues X,XXX

Paul Matteis

worldwide? Okay. Is that or U.S.

Stan Crooke

Worldwide.

Paul Matteis

cardiac credibly if forward other it. cardiac surprised I Alnylam is that study patisiran outcomes program in a data yet that tafamidis it's thing interesting only Or pursuing follow-up playing was you market LICA And At has for forward for and non-outcome the like of TTR where compete then one as really go credible -- a path But the tafamidis least this with not on an time indication. to answer to level stage the feel is or with us here? one strategy to the a understand you? a thanks. kind do to CVOT Okay, way to path at that field

Stan Crooke

latter. I'll answer the

marketplace. will important for very you approval you goal. priced a having Brett be I And do getting a is that demonstrate amplify? approval the value first think think I sold drug to step. the data want sufficient and get can is in to Getting

Brett Monia

also They've And Tafamidis what real has keep treat outcome to going to are to exactly with drug looking think a study in mind benefits for That's Pfizer right, that's be to we that show people set keep an -- TTR a Stan. cardiomyopathy. I conducted and for bar. have

the the study, taking very cardiologists from we're just this providing available We're of outcomes are there needs be very all to readouts very get information are but of of the to course Of will Stan study. part that our a important. Functional as part us. part They’ll -- believe of commercialization be also out taking also be not of important in We in. all mentioned just information approval to us, a important a standpoint. that's to all

So final design study our we're taking part it be are and all touches that together of the in. on And now. the will coming

Stan Crooke

well. go sometime the well That study will very continues study Dr. right data really One in cardiac have item have recall, with may think I ongoing that in this site. people is updated encouraging. an year be of forgotten to if Brett? Benson's disease we And patients that And as study do still manifestations were you the

Brett Monia

hereditary Absolutely. now We're as going the of data and wild patients to type years better. both beyond some folks are these getting treatment in three cardiomyopathy. present And go

Stan Crooke

how the And thinking that want outcome Phase informative also is the development. X in study and to our about we conduct

those tuned for we're presenting forward to looking data, stay them. So

Paul Matteis

Okay. Thanks very much.

Operator

be our go Markets. will questioner Kim And Capital next from today Do BMO ahead. Please

Unidentified Analyst

I taking you little is filling This danvatirsen. for go Thank refresher question. you give wondering could just the for guys Neil can a if if back in could how was opportunities to [ph] And may beyond Hi. Do. neck additional see head to you're cancer thinking and pursuing Thank Phase what those? there data? what the on be other you. and And about with expect X then we we

Stan Crooke

Brett, seen saying with will danvatirsen we've is both safety and definitive head I'll and but very benefit and begin give regard are by of very, just the encouraging neck. answer, what than broader it to

Brett Monia

last year, ESMO AstraZeneca and late Yes. patients at cancer. with And presented probably neck in head refractory Neil, you recall as

that very year that's patient the were head ramping up the on use make that the complete a population. durable cancer. in AstraZeneca or responses Some is Phase will see in that responses And data response to We study to go neck this data impressive expand had shown you'll and and to pivotal decision study's partial very And and responses that X rate. this essentially this year.

cancer this two with but announced these too, a indications. they're are lung they've remember to referred believe even I moving combination; addition, expanding In into combination they other is beyond that durvalumab. And now also with this their have PD-LX that cancers they also

And efficacy. cancer. going as in most a and safety, excellent reminder, for Generation turning or this indication. advanced a X.X cancer is our well program impressive just chemistry and into this for it's So broad very tolerability again any And is

Stan Crooke

it We're excited colleagues too. about I think are AZ about it at quite our excited and

Unidentified Analyst

I for the question. that. Thanks appreciate Great. answering

Stan Crooke

You bet. Thanks.

Operator

questioner Fye JPMorgan. today our Please ahead. with next go Jessica be will And

Jessica Fye

you TEGSEDI? Thanks online for you just my wondering sort Whether question more there if there's ramp-up SMA, color to guys. was of expectations taking could risdiplam? that in question. relates of competition for patients to could from you come it then I more a are your And the shape versus bolus latest on any additional the kind your potential for Hey can online as going? steady second talk thinking about coming switching of provide

Stan Crooke

they position provide and cautiously and but more to market -- managing a going -- as than years the early I getting the running team -- her practitioners very manage to optimistic feel organization after yesterday. the be command. work what's and who that I ramp up it's fraction Sarah and physicians colleagues such both information to been the And don't system the our standing I am about drug I'm regard high-quality a provided have the it. and And and as get to and and did likely very patients start of Jessica, with that and should've TEGSEDI to impressed by encouraged process the access position Boyce through question, I'm look at Akcea I least was days, With and at TEGSEDI's its at it's of can on

encouraged, what question we're So I me but you it's And the early can all can days. remind second was? there. really And do that's

Jessica Fye

-- thinking emerging how potential SMA? kind competition from and your Just of on latest risdiplam

Stan Crooke

them, of Well we standard but SPINRAZA of care. watching is course all are of

data The treated I total it's you infants vast think are experience the if majority data symptoms, normal of hard think and the these to We -- like in to an children. developing healthy treatment the achievement. standard the healthy. NURTURE at very, prior beat look vast quite get information treat of of And the we of very, makes amount to that going difficult be the to better care the SPINRAZA we displace. that's patients the information, longer we very fact have, the these think strength that's

very it's we're molecules about of and But watching We're course we'll the again that optimistic think blazed So are I that advising for SPINRAZA like additional colleagues go. proud fact and small good the think proceed. our how we follow us they people ground. drugs see all, where too. of all also I the future we're that patients coming are in and news we We these the

Jessica Fye

Great. Thank you.

Operator

will Mani SVB. And our ahead. Please next questioner today be with go Foroohar

Rick Bienkowski

This filling Mani. Thanks for our questions. Rick is in HI. for taking

of Huntington's tempo thinking How a thinking bit HTTRx? approval broad about currently launch opportunity after geographies? are one. the the is what you question a potential across are of you of the commercial about first in The And different

Stan Crooke

about question, I'm TEGSEDI? sorry, the was first it

Rick Bienkowski

I'm HTTRx? sorry. program and The Huntington's

Damien McDevitt

The commercial opportunity?

Stan Crooke

we've to the that? -- when what anything to when exercised you said they I add Roche Damien much add and want Well, we do can't option. has all said to

Damien McDevitt

opportunity a Roche's XX,XXX there worldwide, commercial it's we that's so very and No, view. consistent patients with is know significant

Rick Bienkowski

Great. geographies approval gating could question should in right. geographies? as about second the we of other approval think the a and My in is, approval approval That's in EU, helpful. U.S. of EU? follow the potentially outside U.S. WAYLIVRA thinking approval Or when for All factor the in about regulatory

Stan Crooke

anymore. for a any not and of U.S. much gating drug really is it's not gating

markets. give approved most a -- access and We're FDA United I to will EU believe happen. intend strongly of States for confirm today that that. other think Clearly, working in WAYLIVRA to the should the the approval think for we path fully we that to an be us with

So it up the the EU obviously, us is for market additional an markets. opens important itself consideration and

Rick Bienkowski

all. Great. taking questions. our for That’s Thanks

Stan Crooke

Thanks.

Operator

Birchenough And next Wells ahead. Fargo will Please be Jim Securities. the today with questioner go

Yanan Zhu

taking This Jim. is for Thanks Hi. the dialing questions. in Yanan for

if may. questions, three we have We

capital to like get cash. consider we'd complementary or with first, editing? Would thoughts Thanks. therapeutics you on allocation RNA gene therapy your acquiring billion or So in $X gene strategies assets in

Stan Crooke

that we planning quite our that's It's for dollars. think calls highest commercializing technology. commercialized we've little We a a a been this a is that active be mentioned our process. investment advancing primary our while, allocation have think, commercial medicines on through very Well affiliates our And will in pipeline, our couple underway of -- I capital for while. in for leverage and the

could enhance delivery hand, will that are development of facilitate even other there the On of potentially drugs our that identification out antisense like certain ways there that -- other opportunities tissues better muscle. to the could of

bring next where And long so, of is we're exploring. people intention those areas are platform. over And the big to who haul, actively be all the the course, our

looking all so we're things as well. at those And

is So if and -- we'd luxurious at we're sensible expand doing pretty we of as still grow the look foolish And aggressively opportunities profits. as those also in be we position being flat didn't in we business able absolutely invest think to are and to it. investments our of every area

Yanan Zhu

pipeline. remaining on is helpful. the on two it. LICA. And One very then are Got That's questions TTR

pipeline TTR your AMD. have newer approval for Geographic timeline targeting and about wondering as there you earlier using complement thoughts prospects Do the on is lowering development? dry Just that for on program data? And endpoint could Atrophy interim? question And Thanks. other any a be the in

Stan Crooke

little with meet dose exceed that. whatever we Well was TEGSEDI. just target the think timing with answering get level about and with about going. TTR a little with to AMD that's that's we we just to until is by has about demonstrated like standard a that defer reduce bit And TTR will certainly We been that and enrollment of that or study that study LICA, the LICA good with how can size information underway of the want. I'd questions Remember set CFB I for so a reduction getting all more

Yanan Zhu

it. Got you. Thank

Stan Crooke

You bet.

Operator

our And go questioner will be Gena Please today with next Wang Barclays. ahead.

Gena Wang

my taking for you Thank questions.

very ones. Just two quick

us with quarter can Europe I share is regarding the inventories how breakdown revenue. U.S. you know don't initial and -- The also much in revenue regarding the TEGSEDI the first and first stocking? one

Stan Crooke

to handle want that? Beth, you

A –Beth Hougen

Hi, Gena, Sure. Beth. it's

the that that for both QX and channel we and came inventories revenue do the manager confident so visibility revenue we in and So feel have from U.S. TEGSEDI appropriately. into Europe can

Gena Wang

contribution inventory Can $X you how million? much us this stocking to with like share

A –Beth Hougen

given We we and detail haven't and very of managing given particular that but have carefully. that tell are monitoring the you I channel that we very disease can a medicine into good rare that that level visibility it's carefully

Gena Wang

Okay.

Stan Crooke

they're of kinds of these for medicines general sort In just-in-time ordering.

Gena Wang

Okay.

Stan Crooke

It's know differently say it's drug. quite population least not a I handled -- the large from at patient bit that little

Gena Wang

see. I

So pretty inventory stocking weeks right? like that's reasonable to two one

Stan Crooke

simply as it. aren't We've much that. as to answer we going are going to answer it answered We

can respond. you and ahead So go again and I'll ask it

Gena Wang

Okay.

Stan Crooke

I appreciate your persistence.

We are very very, familiar with perseverance.

Gena Wang

Beth question. Okay, one ask maybe more I

current NOL? your what So is cumulative

Beth Hougen

next and NOLs that the somewhere taxable the cumulative more as tax years. $XXX income into assets is in year for future beginning to we million for future got range. in Our than the this sort available have We've offset -- of enough several

we've like begun positioned at at net line, forward. able now and income giving operating a line both the feel So income from grow the to going on the earnings and favorably very net guidance are we we perspective be to line to continue tax that

Gena Wang

Great. Thank you.

Stan Crooke

you. Thank

all your more are appreciate do interest. to to We take an going to question, and then we'll bring We one have the call end.

Operator

And will ahead. that Company. from & Laidlaw Jen Yale Please go with question be

Yale Jen

year. and questions, the on taking for congrats Thanks the

Huntington The is first program. HTT question about

-- open for over study, in data approval year? open-label that timeframe? the as year. be is of here report be First file could the the before study, file -- for a gating maybe probably will the of believe potentially terms the early reported history sum As usual would of it's next they that second I that for for at question that natural factor of the all, data a well Is sort

Stan Crooke

There of the of knowledge natural course is great history about Huntington's.

cake. study history icing some in the added is on the So senses, natural

in And some this serve lot to setting the those a been basis in quite disease open-label would while contrast for which there that filing has with of little information. relatively running rare as Here study, is -- that the a for diseases extension patients.

Yale Jen

Maybe one, one. quick just great. Okay,

show that it the get in I did next SODX, for of positive -- clear outcome? terms that if the year, current study the In extended Biogen study maybe filing

Stan Crooke

Yes. as and We're that, Biogen. excited is about

Yale Jen

thought Grateful and on great. congrats this. Okay, again

Stan Crooke

very Thanks much.

Stan Crooke

everyone thanks for Well, interest. your

off you thrilled Ionis very forward and an with of successful important we're in sharing you. you look great the to lots efforts be We proceeds. at to can these events as that exciting year. start We're with to important and XXXX, are you. a sharing and days words last was think our year think that tell from I really we Thank the year

Operator

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

now may your lines. disconnect You

Ionis Pharmaceuticals (IONS) 27 Feb 192018 Q4 Earnings call transcript