IONS - Earnings call transcript Q3 2019 | Ionis Pharmaceuticals, Inc.

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Operator

Good morning and welcome to Ionis Pharmaceuticals Third Quarter 2019 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

Thanks John.

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

also that accompany today. website slides our our on posted We have discussion

Officer; today’s call Officer. Board are Stan Hougen, Chairman and our Officer; the Executive Operating on Crow, and of Brett Chief me Beth Chief Financial Chief With Monia,

like Slide consult We'll our for our actual I our risks the to which current which to details. draw differ forward-looking statements may in SEC making filings results beliefs. to your are statement. certain I X, are language and uncertainties on encourage to you our attention risk expectations and forward-looking to statements, additional materially. discussed based contains factors be These subject

And to with that, I'll over Stan. turn the call

Stan Crow

and good Thanks, for morning, us. you Wade, everyone. Thank joining

commitment in medicines, value to technology, robust of the in financial to increase performance. which our commercial pipeline and our led our innovation Our is today reflected has

guide improvement. we strong Because of – year, first our in are significant to XXXX significantly nine performance this of increasing months the of our

income. on operating net income. net of in This more approximately third year means operating to We’re track we our $XXX $X our million fourth in deliver that revenue, than year income, than $XXX to billion in million and deliver income, more of expect

to We’re element aggressively our as achieving as justified invest every continuing of while results across business. these strong

the are foundation treatment extremely pleased to they show broadly with throughout performance. of progress care product. continuing getting] SMA stronger business growing have data, with SPINRAZA of treated of of and ages patients all we’re getting blockbuster the success all supported body and achieving making [they We’re longer for SPINRAZA’s the continued worldwide by the is the

adding is role. reach commercial to AKCEA Damien its AKCEA necessary key leadership assumed and the his skills seeing team, organization Damien already as goals. positive momentum the at with new development We’re leadership member help to

to WAYLIVRA. first The promoted of and after AKCEA’s organization management commercial to TEGSEDI Officer Kyle U.S. senior leading Jenne, the was several the team recently Chief additions who Commercial is

Importantly, we’re WAYLIVRA. and Latin RNA-targeted with first for market AKCEA at across see expanding the approved With Brazil, patients momentum polyneuropathy now build the to in treatment beginning TEGSEDI TEGSEDI a with TTR opportunity is world. of approval in TEGSEDI is therapy America. the

TEGSEDI the Given activities with this launch form region strategic of for in the add plan we market place in development medical and key patients hereditary the therapeutic of America Latin AKCEA. start disease Brazil number to that is substantial Brazil, TEGSEDI and in for support

and market Germany reimbursement successfully EU TEGSEDI for and underway. Germany successfully the preparations EU. is successfully AKCEA by Akcea the following beginning in and is is the launches now to negotiations in are and country progress NICE on also completed TEGSEDI in where acceptance additional completed and market pricing completed UK Austria make negotiations now the Consortium. negotiation Sweden a Medicines Scottish in on pricing significant in

from medicine on Germany in only the WAYLIVRA benefiting disease. approved is now FCS the this patients of market with treatment for devastating the

the and data to and more Additionally, our find path efforts Latin long-term PTC experience ongoing new U.S. EU to market is with into preparing the for believe support year. in country we expand AKCEA next launches regulators will Canada, WAYLIVRA for EU is preparing WAYLIVRA. and America, our to in a the

in and AKCEA-ANGPTLX-LRx the This strengthening of in Pfizer have metabolic and Most for further economics rare substantial backend large evidence develop Damien we common. diseases to AKCEA technology our very its cardiovascular is much and are momentum and with for and to provide patients have populations also treatment both patient confidence benefits his that and certain advancing gained diseases and recently, to more ability with partners and pipeline. commercialized entered collaboration a targeted front that AKCEA team diseases. collaboration up the was and our

medicines and to have report development, early and soon AKCEA-TTR-LRx Phase will AKCEA in of Phase plans AKCEA-APO(a)-LRx, AKCEA-ANGPTLX-LRx. year, and two next X data studies X AKCEA Additionally, AKCEA-APOCIII-LRx from

In pipeline, addition across with achievements quarter. medicines broad value Ionis to pipeline driving the recent also in our multiple this achieved AKCEA the we successes

goal We’re with on underway. XXXX X track to ending of programs Phase achieve four our

for than patient TTR for shortly. We cardiovascular these are Pivotal SODX-ALS LPA Huntington's with driven large Phase substantial X diseases patients progressing addressing better studies and made to progress our disease population. with medicines beginning Amyloidosis are other and very disease and programs with

In collaboration to the B Factor buyer the thromboembolic our XI licensed treatment treatment of LICA virus of for GSK hepatitis advance and with to financed new patients program for ANGPTLX-LRx, addition with chronic disease for HBV the Pfizer infection. medicine

disease. its Our in patient as neurological disease, large disease Biogen advanced the X/X pipeline treatment the Parkinson's well. Phase first another of for patients with dosed study

partner added for Biogen and that’s two neurological our medicines with We new also pipeline, three wholly-owned. one

in continue advance increasing and and continues our drug these an we Importantly, commercial substantial our successes, while and businesses to continuing as technology. aggressively growing Today, including businesses, our position development to at the technology pace. our we leader RNA-targeted our and financial in advancing invest pipeline medicine across and in to strengthen achieve broadly discovery can investing

have we the this balance potential these create flow, also value, goals course, strong shareholder and in near-term. highest cash all investments these shareholders while patient and of can of and greatest value believe all to achieve returning priorities our we because our are, to and achieve our of because sheet These the

future authorized of Board to allocation potential repurchases strategy. of long-term we business value. creating with of additional in million that the to pleased to part This near-term our announce capital $XXX in commitment as up for overall consider share reflects an to us confidence initial So, our and of the program repurchase today, our action the has Directors our I'm have that and

is shares buying our a believe also really investment. that We back good stock-price, at today’s

to – update to by followed will And call who update financial it provide now, over I’ll progress. pipeline turn on turn our a Beth you Brett, over the

Beth Hougen

Thank Stan. you,

income of year, this we first strong third million on delivered the million Our In months continued net financial operating basis. of results and $XXX quarter. in of non-GAAP income a both $XXX nine the

achieved also operating income on GAAP net substantial income a and basis. We

$XXX and contributed in both advance, These by were XX% more including last revenue, a the in SPINRAZA’s results increase driven revenues, million partnered nearly substantial performance commercial Growth our than strong compared as continued to increase. blockbuster programs R&D to revenues year.

generated maintained we net from strong receive cash Pfizer through to worldwide quarter, we $X.X sales in billion increase sheet with payments of $X.X when last compared cash, quarter, SPINRAZA end third this Bayer the the third nearly our balance the we In to year. and increase expect and billion our of of balance an over quarter. XX%

revenue our of SPINRAZA patients treatment increased by by performance, XX% million last $XXX joint than number compared worldwide. more quarter SPINRAZA’s more last than to from increased XX%, year. X,XXX The approximately Reflecting patients on royalty to of total

to New in both the patient starts the this around contributed growth. U.S. and world

believe represent for adult a quarter by in growth. Biogen SMA SMA of and U.S., the and X% largest a segment, growth were the over driver increasing third significant patient continued we adult In patients opportunity quarter patients that as last the

in place the SPINRAZA in approved with to on in increased XX outside number last approximately U.S. and of countries by compared Now patients over reimbursement countries, the XX%, XX quarter.

markets drivers Pacific the take in this as established and in Latin such rapid growth. primary Japan, were in up EU plus key and performance Strong America Asia of markets

than estimates a Additionally, SPINRAZA patients Biogen expects initial Biogen recently in has began is opportunity the that and larger XX,XXX these SPINRAZA presence. in first direct continue in with China markets much growth SMA for where believe treatment. key global to than patients more the markets

the the patients of believe type. will all treatment to of of SMA evidence as of SPINRAZA ages of remain body for and the robust global SPINRAZA supporting a Importantly, care foundation profile grow, continues we

academic months Turning particularly with now WAYLIVRA, of and and during disease of continues U.S., the maintaining centers. to is The the and $XX community relationships of the physicians AKCEA’s education, team In product AKCEA in first we to earned strong centers launch this make million on with year. TEGSEDI WAYLIVRA focus the TEGSEDI progress from TEGSEDI. while sales nine

growing Outside genetic-testing following the now we now TTR is approved targeted the the into TEGSEDI polyneuropathy are than RNA with America. of in first in translating people approval program Latin that X,XXX pleased more extremely use AKCEA’s Both program. with therapy of initiatives Additionally, are Brazil of U.S., more TEGSEDI. is prescribing of these for using treatment physicians physicians the

PTC their Therapeutics start launch plans to activities Brazil in immediately.

to expanding also more look potentially countries. broadly We American forward other Latin in

the for in now in all countries Additionally, country preparation additional underway. launches AKCEA’s Sweden, on are UK, four patients are and and Austria, in treatment EU

quite now all has AKCEA’s running and patient physicians feedback support up and positive. commercial from markets is been program across

EU EU Latin the also now PTC. are with WAYLIVRA year, forward we're approval additional next launch we TEGSEDI with pleased in see following potentially program to America AKCEA Patients So, expand in in the to into expand being treated to is preparing Germany this launch. through look countries and

Reflecting of the a Biogen, research advancing revenue revenue value payments from numerous of included XX% and in recently GSK, milestone targeting with for X/X Phase milestone and Novartis, substantial by million. of $XXX increasing our technology, $XXX fees upfront R&D [indiscernible], from and more included license increased revenue $XXX to than million million R&D amortization in treatment most people payments, payment initiation including programs disease. $XX million nearly Parkinson's study for in the and of [indiscernible] which from development $XX the million for

of $XXX million in upfront all we substantially fourth AKCEA-ANGPTLX-LRx for quarter recognize to license our expect the payment We results. Pfizer's of generated

of recent Our for can medicines increase in we and our the technology. demonstrates AKCEA-ANGPTLX-LRx license economics command

the to payment, we sales. $X.X tiered milestone to addition billion to up and $XXX upfront million receive on In payments eligible royalties net double-digit are in

conducting and adding development activities. by future is substantial for Pfizer activities and these Moreover, value regulatory paying

XI-LRx, in of recognize Bayer us. the Additionally, milestone represent fourth-quarter from Ionis full assume will payment substantial results. $XX million the our we Bayer will to Factor development in value which

operating of compared first year. increased million $XXX nine expenses non-GAAP to to months in this the last Our year,

technology. investing across TEGSEDI including in broadly investing are and our our business, advancing expanding and and pipeline the our WAYLIVRA We launching EU commercializing advancing in

performance strong improve Our financial us XXXX year to our guidance. has this led

in to now revenue. earn billion are presenting We $X

operating aggressively original We while expenses, are across maintaining continuing to guidance invest business. the our for

for the of range the do anticipate SG&A we at However, and being end expenses. R&D both lower

our income XX%. increasing nearly represents five-fold margin to are operating which more represents of We million, last than more guidance $XXX operating an over And year. than importantly, increase

net increasing our $XXX to to our million and $X.X guidance more billion. also We’re are we increasing guidance cash than income

pipeline, our broad medicines, commercial our and are investments technology. priority highest Our

Our $XXX means through value combined sheet shareholders program and about cash profitability an to begin flow we with our to repurchase initial commitment able are share strong also million. balance to returning continued

further shareholders programs if value We shareholders. of stock plan of this that market our to to even our consider open And this transaction. repurchases anticipate us to additional provide increasing conclusion ability we repurchase share The we the return strength, and nation value technology, will distinguishes from our program more under peers. to to make in program, value financial

with on to I’ll Brett turn over the that, an to And provide call pipeline. update our

Brett Monia

through Thanks, through care on XX foundation even we pipeline for all over report advancing have with to treatment. many of market treatment continues Biogen towards the all And clinical progressing medicines SPINRAZA three patient trials. and development the SPINRAZA global more Beth. demonstrating research is ages the data of Today, prolonged improved SMA of performance SMA in patients of of types. new, longer-term medicines

to A developing pre-symptomatic have And have from months, now some babies like of disease. of of now infants would began the SPINRAZA disease. SMA to on stabilization reached the in kids shine their study study, see rapidly in these treatment continues and without as new later study healthy patients whom normal approximately nor we continue counterparts. adulthood, improvement measures on their Remember, with with for data kids nurture [indiscernible] XX show their

walk also SPINRAZA X study on of dose treatment the a treated Today, including safety have some on shine the SPINRAZA ability supportive benefit with established with explore [indiscernible]. achieving is potential preparing in well to over expanded gained who of SPINRAZA have Biogen on greater some nearly patients higher is patients been years. X,XXX to profile the dosing. with is today patients SPINRAZA for of even

dose higher fewer study soon all will devote potential which patients ages, the safety SPINRAZA of X/X we loading begin the increased and to efficacy achieve with Phase to with evaluate SMA a with adults. in doses expect including Biogen's

we which Additionally, for follow-on Biogen treatment development medicine year. and enter of a dosing, SMA the next are less frequent focused could developing on

thorough X the Huntington's Roche HTTRx, including enable study. in patients Phase as the Ionis in each announced known This treatment every more allows enrolment Phase evaluation recently in HDX is study. this expansion also generation add four-month comprehensive X a with progressing to regimen. patients China disease. of expanded will a from also It dosing RGXXXX to Roche program group,

studies of of history also be medicine evaluating program the expansion in modifying hopes with open along Roche’s X thoroughly is rapidly SODX-ALS, study Phase responsible. bring Phase for to evaluate pivotal is potential label clinical Ionis treatment medicine for of all HTTRx patients disease the important first well. the tofersen are Huntington’s the [valor] comprehensive of natural of This this disease with and with X/X elements patients X% Biogen of The disease. progressing study to this the treatment devastating to

Phase Biogen will of genetic the enroll over patients cause our of work form X/X also This X initiated most represents together with worldwide. study disease, we targeting common which patients study sporadic recently a well patients to mutations as million as [mark-X] Parkinson's. of Parkinson’s with medicine

forms including disease disease. various have we both and development pipeline Biogen, Parkinson's now research also to expand that pipeline many wholly in eight medicine In of range addresses neurologic Alzheimer's top broad diseases, Under continued stage more. with together our and a generation and our [Indiscernible] medicine. collaboration substantial we advance in on a on ALS of in parallel,

In advances years their technology addition pipeline transformative and a many in of commercial for come. our continue potential across to to enable pipeline, making the advances enhance we’re potentially and medicines, our robust expand performance our of broad medicines the to

our value are the diseases our patients. our broad rare the substantial common millions treat value, we course, increasing in confident on potential, technology This of to partners based the reflected partners is our of commercial from recognizing which in is recognize Today and of most received effecting range a the collaborations. from

also are medicines in We enthusiasm this patient its potential treat strong to program large million For with available substantial have that populations. infection not repeatedly therapy. virus than chronic XXX year, our patients with and with adequately world treated expressed LICA recently investments currently platform Furthermore, has licensed the around the more developed hepatitis our made about partners B example, HBV several our address of GSK technology.

less profile attracted low-volume monthly enabling robust are the or medicines frequent on based to partners Our LICA even efficacy potency increased with LICA’s dose.

are study our X,XXX over one of of year in the of treated patients by partners importantly, total people to in up More safety treated LICA Phase profile including XXX favorable platform AKCEA-APO(a)-LRx. demonstrated LICA as the X for the medicine, confident our take nearly to a

being High partner confidence many. in in is our LICA technology demonstrated

This Pfizer XI cardiovascular. advancing AKCEA-APO(a)-LRx of example, to Ionis' Novartis For LRx the for to antigenic outcome AKCEA-ANGPTLX-LRx patients for the and of treat millions AKCEA-TTR-LRx outcome millions chose millions medicine. cardiomyopathy patients will study, TTR with study with diseases. patients be with certain advance thromboembolic licensed Factor for Bayer treat Lp(a)-driven first cardiovascular is And with metabolic together disease. the cardiovascular an studies to for

with target of chemistry, a and types antisense our incorporate of broad continue Furthermore, of benefit to Today, substantial paid we through and antisense investment in to drugs advance This antisense our cell has range to diseases medicinal validation dividends. across continues innovate routes pay many of and advantages we patients our medicines. technology. optimization these nearly and delivery, can advancements organs all and most

feasibility we antisense along diseases performance substantially to commercial chemistries, of aerosol lung, medicines have have the enhanced of of more broad reach for of our example, For a range for our technology. we expanding delivery further potent already but with with success shown the potential the

first delivering the medicine the Our sodium ENaC pulmonary gen epithelial channel X.X targets in or long.

with from other pulmonary X/X our advancing the results for people sharing we this look are ENaC our with programs in along treatment emerging cystic and forward to updates year medicine and study disease within fibrosis pulmonary other pipeline. next We of Phase

another oral on chemistry to our once of believe also in antisense commercially we’re represent Gen the viable deliver oral value LICA will of patients with We’ll provide studies an to clinical substantial yet our tablet the combining research. medicine. verge advance significant by these daily progress substantial a developing enabled, results our as our The antisense pioneering chemistry. next with sharing from dosing ability again, make to potential X.X medicine advancing year. We to forward by early antisense a We look

look to year eventful. around pipeline has this months come. numerous events forward look to quite productive in the driving of further months nine highly through year. end providing and first value the I to the technology highlights exciting, The forward and of next additional We year been and

the over And this back the portion I’ll turn to of now, call call. close to Stan

Stan Crow

Thanks, value our our a Brett. efficient strength of Ionis and it's technology today reflected technology We build on financial of and our the foundation is product directly model. in of business a our

Our of then technology. strength to financial of us flexibility gives our value the our medicines maximize and the each

the of are demonstrated economics technology as partners by of value recent our our Our recognizing transactions. the substantial

Ionis day. Our business and to shareholders are value strategy patients delivering is more sustainably succeeding, every and we profitable, today, and is

potential investing medicines, therapeutic aerosol medicines of routes medicines, our new faster over areas. ever while advancing administration, the value pipeline commercially business, enabling delivering attractive pipeline an for our new and in and growing delivering in our advancing of new wholly-owned future medicines, while technology XX we’re oral of pace, potentially Importantly, including broad and our of advancing

you of us discuss anyway. years On I we to of to December now deep working an not exciting to the advances work geeks, in real we this right believe we’re heart, it's many just the will the the be to technology. the This techno with in plan aside, forward for this but to a powerful opportunity be and more form for discuss hope recent ever webcast as is and that host to come. a positions we the in dive in our on science faint technology you’ll look XX, probably our join

and the patients The come. very to to confidence webcast, successes will for value us we shareholders in today we continue strength and more to watch announced information the built which years financial that give about So, deliver our business and will be shortly.

please. set Q&A. you turn the I'll for can up for over Sean, if us that, Q&A with And call

Operator

question-and-answer will Messer We today Our question from will Company. Please & begin session. first ahead. come Instructions] [Operator with the go now Needham Chad

Chad Messer

believe Great. feeling good – are thanks the and sort strong really congrats I the you're for share of on taking very results. question about buybacks my how of financial these indicator business.

Stan Crow

Appreciate Chad. it. Hi,

Chad Messer

you to recent I tafamidis launch, thoughts cardiomyopathy, indication? trial guys with TTR especially are a identified Pfizer about you comments given believe that patients Just X,XXX share in the some wondering that about if said from your since about could they they start over

Stan Crow

we’re this believe us very performances done. market Well, is well, for performance great LICA extremely other other the and they sizable that to disease and opportunity know and we going like think is the have the confident a I deliver there of that form there TEGSEDI medicines of space a LICA

Chad Messer

Alright, great. about of And know, concept oral lot either sense move as an using have antisense new existing forms then, not be hearing we’ll, more what makes sure as thoughts very newer been sort have? first drugs to, the future, injection amenable the basically excited know, a that a you about of might that in know, you subcu any on you you to but and tried-and-true of I’m know, indications know, you or, you that,

Stan Crow

lot will And webcast. Brett above. at about describes say our first you’ll it, December it answer technology on hear it XX, the upcoming be is, the all I'd hearing a of and more You your as question to

Chad Messer

Alright, was of kind expecting. what I

congrats forward again. that webcast to Looking and

Stan Crow

Okay, Chad. thanks so much

Operator

go Jim Fargo. ahead. Birchenough Our question with come will Please next from Wells

Jim Birchenough

Yes, hi, guys.

and see the Let great my that. me add and buyback share the confidence underlying to congratulations

could follow-up. and lumpy want over And outlook the maybe the some to got guess, sense profitability? then, be just financial or expecting in give side Do to given we’re thinking of think So, and sure things, revenues R&D one, both still little a we about a contribution of I’ve just of us revenues terms make and questions, the should of to how time smooth you about couple number then we sustainable out of guiding revenues earnings? I a a expect

Stan Crow

the Well, operating financially net are be of reporting year year our our confident we’re yes, income. successful first we next And income of business. another very optimistic year and and that now year third across and will

things on to going forward and We next are and excited better company look about year. the that year are in really another AKCEA the

Jim Birchenough

things the that is could just maybe just then And think, investment highlighted in capital of technologies space. medicine more recently the allocation, leadership I historically of in maintain one you've or in genetic terms even your

your allocating could And beyond this antisense of in maybe area or to how that? you're your you so, to talk medicine? capital go extend dominance to And efforts

Stanley Crooke

Yes.

even that, anything progressing is fronts. one, about is And invented greater we've technology today. technology. next today. expand Jim, use we're of our antisense might are the we're access it Number the extent looking of and to genomic two, and what enhance than to number broad or is is the We excited an Number on it progressing, two three, that then information doing performance any for three to

technologies you you antisense every of if the and couple would components like investment day. the Yes, three third, investments components don't in the long expect first strategy, because think those about of come before along enabling

that the So, to the we've made you'll those investments coming in expect I see that progress in on some do months begin areas based far. so

Jim Birchenough

your And what pipeline, for excited final programs, so? wholly-owned say wholly-owned partners products most the products speak just for question key and you the you're not and a in Brett, the you on partner visibility maybe get or over those next remarks, Maybe year will can that prepared question. to then know about? maybe said said your on maybe there's beyond but have I what much what are

Stanley Crooke

Brett?

Brett Monia

for The thanks question. the – Yes. Sure, Jim,

for Our therapeutic medicine how have depends on or the advanced the how I many transformative that program plus, the be wholly-owned pipeline potential what to areas. is is therapeutic a for market. growing coupled rapidly suppose a the answer impact areas, is, – to big – potential across with the on all medicine

year and very for to of a growth to hormone Phase readout exciting to move is acromegaly Phase have potentially X on X. receptor it's highlight is program that I'd couple a due in next and three

that's wholly-owned acromegaly year. could Phase patients with X that a in be in So, next program

MDS. a very alpha to is beta multiple intermedia, program thalassemia potential myelodysplastic program syndromes, populations, that disease TMPRSSX including It's has treat Our as the as thalassemias, exciting. well

and and opportunity it's of that It's for X, very drug. X, Phase in rapidly. moving lots Phase in well is performed very So,

pipeline, prion nicely. Lafora neuro disease, on very moving disease, forward also have validated with our nerve it’s disease platform. all commenting are we And our diseases worth degenerative Alexander's then obviously, And

last a pipeline. So, And on a run thing has watch lung platform our that. would is, the I say that very pulmonary then out disease high for upside potential or

mentioned behind ENaC more to our and in the come showing I is that. in webcast, As I, promise Phase program is great

Stanley Crooke

economics would Jim, about value. Alzheimer's number our of really of have, APOCIII, TTR-LRx It's study. you that that's AKCEA. savvy extraordinary of the the look is of to XI, commercialized We're if how incredible form of XXX with add to the large tend and one we asset in medicines I And and Factor be medicines wholly-owned. that to think APO(a), the have of potential ANGPTLX, excited Factor deliver patients And I going knee were lack the replacement people through tech at Parkinson's data XI forgotten the

deal. the are mistake it's, economic because extent, we licensed to come large that terms a – under the to wholly-owned from work. of it's and products solely are focus have, think I manifestation to dollars those it's on model a that some great going pipeline business our And of the again, will matter

subscribed said, add I I but would just fully comments I So, Brett obviously made, did what it. I that since to the

Jim Birchenough

for questions. the taking thanks Well, Correct.

Stanley Crooke

thanks. Okay,

Operator

from Van Buren Jaffray. Our with Tyler next ahead. question Piper Please will come go

Tyler Van Buren

of give data, Huntington's upcoming under you you to on then want events, in a composite to active what results. the Hi, of would the didn't course. once I the just guys, these release endpoint timeline, key the on I course. timing But data congrats get press your we updated therapy? the with the a thoughts on show get would open patients And be expect label, outcome and of successful with what know

Stanley Crooke

should a I Brett like answer should avoid. that sounds and question It

Brett Monia

we're of we're Tyler. Roche study. focused Well, stood, all randomized that's is the data XXXX randomized Stan, not if and study, Thanks, I course, or on mean, laser Phase filing which sooner, is on, thanks, that as what and that focused the X said, range, pivotal

of Phase study Roche data, include year, said has both label the history data study X as well that was XXX share Phase natural regimen study patients, of will which then from that that a about label open the data. the the the dosing we open Phase the X study, lot conducted which start X the they the initial Regarding will versus – changed plus results there which of evolved data next as

for, compared what obviously, in the as been As a improvement significant published on. look for to Huntington's far rating looking as has obvious to well That's scale for the documented the in in placebo study, endpoints randomized and you're clinical group.

Sort of the label X I to It's say. data, going relatively for strong Phase relative open small, it's looking randomized size a the would sample study. be small to trends,

to or cognition that study, sorts differentiate are looking be clinical it's to of Roche but it's history endpoints work X for those X to maybe And I as natural many be to strong data, be that trends for from going to be they're benefits of or subcomponents part such going natural they're for, ongoing things. are the history they some motor that, clinical Phase going function endpoints of But are be to just compared going part doing. going the looking and of – looking assume, same is that the in Phase the that –

Stanley Crooke

we're we open felt ongoing that We've as dosing. a to label every Just encouraged, for based that And that We on schedule always dose or study. the about the observing, was what were the focus remain to Roche two. this Roche decision four-month best comment the on medicine. made add very I does think pleased

open study. performance all-in-all, the seeing And that by and the just label the Roche in encouraged we're we're like decisions so, made that we

Tyler Van Buren

TEGSEDI, launch that. for then we're the on slower thanks seeing And relative trajectory a Okay, in to Onpattro.

not territories guys For as different in I going uptake U.S. different list obviously, Portugal soon you other and come you guys noticed So – are slide launched on in us Europe, maybe the understand territory. just than actually then the could example, later broadly, help a receiving is maybe did territories? and online U.S. what’s you in quarter in also should a different

So, maybe you Onpattro is in U.S., larger guys percentage of slower uptick a which are sales? seeing

Stanley Crooke

Yes.

seeing on we're not what important but we we're a happy – in is an last shifts see And us. U.S. oversight AKCEA, coming we're the quarter, we certainly What very optimistic happening really it's at Brazil I sorry. are is the the we bit new so U.S., significant now in do the leadership if certainly growth very a quarters. with at right slide, happened So, market. will accelerate not are important opportunity the it obviously much for think looking the was in – considered And that momentum. what's

UK We in the with are pleased Sweden, and Austria. very what’s happened

us and important So, these markets too. of important opportunity are Portugal new course is for an

of new little what think this we will quarter seeing out we them, is are team we improvement. progress see performance, So, that a I and the based next even of will and in are optimistic see we hopefully hoped, the we an year on an TEGSEDI next behind that but better

Tyler Van Buren

Okay. much. Thank you so

Operator

next ahead. come go Morgan from will with Our Lebowitz Stanley. David question Please

David Lebowitz

and much pushed to able the the as Thank be a is you forward run recent you give very form? drug talk of opposed for my taking through form being question. non-LICA to guess data I us of LICA HPV Would the that the about reason

Stan Crow

receptors. to an Yes. infection HPV on do that question. we have effect We answer don't the What has asialoglycoprotein a that full believe is

but out we see of performance great we parent. that the level increased So, potency we didn't saw the expected, also of

we’re so, a drug. that the And made, little what along. we’re with is the decision and with PSK also further has agree we about excited seeing It

in drug]. kinds it parent seen viral haven’t quite performance the while. the of overall, So, I of a exciting, was [the looks reductions very think like those And

David Lebowitz

my taking for question. Thanks

Stan Crow

to add that? want Brett, you do to anything

Brett Monia

I No, you don't. Stan, think it. I got

nicely. You covered it

Operator

come Vincent will question with next ahead. Bernstein. from go Please Chen Our

Vincent Chen

the progress. Congratulations on

just one. the and in about more color, that is endosome? to the won’t to you this question that but to a of it portfolio your potency less cell have programs something dose, apply what the for is about some approach drug to Broadly, work. dosed how increasing or increasing the implications matter alluded what's might probably have as that drug? Is our Speaking the such SMA that half-life extent of escapes Huntington's increasing other windows and fraction You increasing this? a could what and drugs? therapeutic active the it the geeky the readily I to or say, program these technology else the frequently might enter this you is, you drug in provide bit Is to of a it of

Stan Crow

I solid we we then than really on XX, now. and tell December enjoy I'm what and can going think will will are what These you you more into detail much get questions tell you more to

chemistry We remember continue to that those of relatively compared of obviously to and medicinal invest in newer program other all programs. neurological is areas,

broadly approaches is can central exploring there. were so, some And nervous we value fact We distribute that the these chemistry very the long you define know drugs novel understand And still that medicinal we there they in system. add that think how last.

we’ve it’s over the have, taking that years XX of and all knowledge plus all So, learned that it. we just applying

and delivery points I We improving will hope. technical Did as this That for cell, – the to very as for call, well release. and the another that’s very, of active into want be but approving gets delivery talking progress doing complex us, as area get I focal antisense on cell we're I about this don't endosomal try to think it, explain or in to you? help very that it the reserve. core what and within of

Vincent Chen

Yes. Thank third. forward much. I the look very to you

Stan Crow

Yes.

talking length be at will We about that. all some

Operator

will Cowen. Our go next Please come Ritu Baral with ahead. question

Ritu Baral

more did Hi of four were new – for the the you you wholly-owned, the and where disease of that data that Stan taking sort guys, in perfectly. thanks sequencing new follow-up mentioned two question could from believe can the pipeline questions programs one on for want clinical with seeing I CNS mentioned. be they I for Biogen we then to thanks prion you programs that and the there love and ones of what Would mentioned, programs? additional know what’s CNS of timelines little a to Bret etcetera? elaborate some the my

Stan Crow

are that. really about that, exciting relatively So, medicine. opportunity. we excited we people into of that And But of are know, Biogen before all wanted remind near-term that with XX% And We importantly an is, drug. to is. I is we observations on how and that based will you get take the

And Brett. over will question I so, the turn with that addition, to

Brett Monia

Thanks, Thanks. Sure. Ritu.

but our talked is [indiscernible] will pipeline Lafora program The entered our So, we’ve were disease pipeline, new storage recent continuing about at neuro Lafora development disease point that into undisclosed two drug, before to we’re programs a and owned CNS. our targets to the with and programs add continue the that this are which exciting glycogen the Biogen. wholly- we is of

that expecting The populations be disease. about not shortly. – we're by you program. Ionis, disease and an excited to So, wholly- that a very significant really mentioned it That’s is not from with know has development reach owned a treated. we a a, And prion to very drug that rather, that’s a before you are Biogen is program ultra-rare drug,

for more disclose We are So, to well. coming. up Biogen pipeline but don’t with our more wholly-owned expand front, and neuro continues right drugs as them

Stan Crow

Ritu to general is give it a you, a data. just to even early going And be sense when clinical of there little is

next I when think become we clear. that much it until will hold year will off on

Ritu Baral

programs December involving encapsulation you that those it programs these And formulations or of little pure are it. is going Got XX, for pulmonary I a bit like agents something talk a guess, or potency program on sort enhancement to are more new on oral delivery? pure, like then play about your based

Stan Crow

a pure potency play. It’s

clinical and of trial, that delivery just recall showed very the it given enough are to really very formulated all medicines the the in at the but of various really drugs key Brett – in drugs remarkably organ to the blazed so distribute that are cells be and not these on, and demonstrated will a with you drug will we and sailing every and December in And work know you agents XX you we administered we IL-X potency these asthma then a aerosol. that. is didn’t for devices And, that feel that know forward, of the we that took some by seeing we nice available, like pharmacology. broad quite good that any that lung. about that could share really now before in simply call. are how chain meaningful so but all developing they are the increase quite understanding the by and organ continue just know these move you the invest the organ ago, the organ end and to years in into in the and other performance We was distribution, we trials we many You so We exciting

Ritu Baral

that Thanks on at for Great. bar. at that open Congratulations questions. was Everybody the oral the to the taking poster, exclusion of poster DIA.

Stan Crow

feasible about What hearing remind just you it a KYNAMRO. just we lot needed a will we that Do the in And and we more coming excited. from are that with up be already was we go XX us. not months, We on the it. will step showed potency was think you in remember, big we you

Ritu Baral

Thanks.

Stan Crow

Okay.

question. Next

Operator

will with Please Jessica from go question Fye next ahead. Our Morgan. come J.P.

Daniel Wolle

Hi. Jessica Fye. Thanks to taking decline well? of LICA worry questions. a This light products product LICA the can the widening observed for platelet for for make case Daniel versus is as you other product, the with the issue the our about In expect reason HBV

Stan Crow

issue, not minor add at XXX way of what drugs. even, show. you in an a it There I do to the it doses mg and no not data really What actually clinically not represented. significant platelets No, lot want those a that? is confusion remotely with I LICA data significant won’t about Brett, are anything platelet that. was is just That’s at is was the actually that There even tell the issue very declines all. month you to do happened will that. of

Brett Monia

as to said doses meaningful and add the drops those very high to were in were platelets you anyway. at in that were not Stan, Just those clinically

Lp(a)-LRx on confidence it [APO(a)-LRx] Novartis just Bayer confidence really illustrates also and LRx platform. the And Pfizer in our XI but has the demonstrated So, our just of no LICA been by has LICA on by efficacy, not Factor at and from drugs like licensing and all (sic) bearing again, safety and tolerability. platform the and ANGPTL#-LRx

Stan Crow

study. month mg in have normal recall who a single below at we Even patient XXX have I a that level not if correctly did gotten

Brett Monia

That’s correct, Stan.

Stan Crow

It was presented was it those simply that are confusion. regret that the the caused that, way but facts. We a

Daniel Wolle

Thank you.

Operator

with Our question Leerink. Please from ahead. Mani Foroohar go next come SVB will

Mani Foroohar

question Hi, thanks for and results. on my the taking congratulations

to capital for the First the you my than which of thank all, normal in behalf investors returning rather your coverage. to on shareholders seems of reverse, be

Stan Crow

way the not By normal. we are

Mani Foroohar

I couple of have a questions. quick

then you add-on, First, have how one failures ENaC of of question I failure number as therapies, developed regarding in the an more that would and that after. be the cystic oral given fibrosis do guys would the effective role patients, about think that developed opportunity, in be an

Stan Crow

is the of this reduces. that at modifier, not One performance is it of the sodium the things that the most it cause about disease. and not a of channel It’s it a modulator, fact directly interesting improves in

very cystic I is on think to primary add technologies a least of animal And at early approach based other lung is to I their from returns fibrosis, And that disorders. significant the but you so other just winner is want clinic, really and it because mechanistic we it a of to what do antisense and disease do with anything? specificity. to lack there the difficult Brett managing have like accessible data about not for

Brett Monia

Yes.

be for or should on patients do for said. It what treatable. small-molecule Mani, all effective this, of as – as not ENaC well you mutations activators. useful therapy that progress that should add-on and add be the have inhibitors that Just those should are It as CFTR

So, the cystic fibrosis mentioned, well diseases. different ENaC pulmonary with XX% broader for treatable that just small are going of types with with XX%, CFTR treatable we of a on this is patients one potential And combo is the as treat think much its think to of we indication molecules the to drug that has population of patients mechanism not because Stan are be as action.

Stan Crow

We, as have I spent Yes. have, lead, one [indiscernible] rule, general comment. as add we want and a to we on. Factor would more so Xi

which we important investment. pulmonary something do the but we that And important, than so in and number much investment in provide diseases can important a believe broader a it us is of had had to mechanism simply patients a that is So, substantial this benefit fibrosis, that very is more is for cystic something use we other fibrosis, to and can an that. really could believe cystic pursue opportunity that we these for us convinced be very

Mani Foroohar

expect sounds presumably active drug for drop for essentially the in And maintain if to in an think you unlimited So, ins overall you when arms? will follow-up, so you wrong, the powering allowing tafamidis cardiomyopathy the have impact worse? will in maintaining do look at of with as like the who whole? How in patients I placebo my balance, tafamidis study placebo do efficacy on about Tafamidis a arm, study, may do am assumptions think more correct it scale I have you TTR-LRX balance Okay. drop-in and is how the are about benefit. the guys you both me an obviously survival but use

Stan Crow

Brett?

Brett Monia

Yes, sure. Mani.

to referring are of imbalanced allowing of in of on study to the care you so are patients standard tafamidis, that we’re wish LICA. we'll on of top obviously TTR and to take. care And not we As or cardiomyopathy standard stratify they for control on patients any that the have patients group other appropriately forms

similar are the own population. study, in assumptions same – TEGSEDI of out patient about So, and there as our with that’s from control can study data wealth we for tafamidis on that phase X as well overpowering Pfizer in based

kinds in and years history. patients which we with for on strong just just compared natural we presented thing I are that just maybe clinical just cardiomyopathy attention, evidence would is, an could wild-type Dr. and on we University, which in shown with hereditary. of remind with patients he have investigator in TEGSEDI longer he looking wanted other update Mani you three to TEGSEDI drive to, an Merrill in and to both that has that I -- Indiana at Phase benefit study all your or treated study, patient That's Benson been have endpoints add The to X take progress

as So, a in on have of and TEGSEDI fall we also performance back cardiomyopathy. natural history with data the to that patients

cardiomyopathy our phase will we that confident are X confident we we and in in study of So, powering treatment group. well feel our balanced very versus control the be arm assumptions our

Stan Crow

And we will underway way about future. very in here, about from things so are that you get hearing all of should in be, a the getting ready be to us big near

Mani Foroohar

guys. questions for taking my Thanks Great.

Operator

from question next Matteis ahead. will come Our Paul go Please, with Stifel.

Nate Tower

narrow put Thanks point the would for guys. modulator? finer the is studies, or patients a down question. are about enrolling on Maybe the CFTR This ENaC, question patients for you to you Nate Hi, on who for it eligible taking aren’t on all think just you would to CS are the prior clinical currently Paul.

Stan Crow

can who what I agent agent don't used be Bret have single think and clinical a program can get think therapy. in want development used be into to we we it’s it beyond be believe an to we the think We said. as details in can patients of used current mutations combination that that that unresponsive and are

there. leave will we it think I

Nate Tower

Got That that Or crazy guys dosing mucus at burden? have I that sense. pulmonary it. a it, something concern? inhaled is I but so looked route Could makes discuss you far? just the it on given is you the mean, know translates

Stan Crow

the having It’s at very with meaningful something in carefully. looked based a all. we and on believe patients. humans the early are well. affect answer airways. concern it’s animals the a distribution and clinic, carefully significant to IL-X us crazy asthmatic No, had looked we the in And very, in smaller then the at And clinical does of experience barrier We mucus – bronchioles returns benefit the telling The the not asked, very as layer was no. to now we

that I we’ve think on were XX, that with something think of December Brett but resolved absolutely we is that we sharing Brett? So, concerned be right some that and you will about, it

Brett Monia

Yes, Stan. that’s correct

Nate Tower

XXX platelet you. understand message was help why place? understood maybe in per declines, loud us one you the GSK and On first the on And dosing at clear Got but week can then mgs more. HPV,

Stan Crow

because increase of just Yes, the infection, expected the happened. research XX, we we in didn't with XX that only LICA gotten beyond much we HPV XX the project the know didn’t that’s we have know And That liver liver But development. in to still you of XX fashion very get level potency what that drugs or other throughout that the we’ve LICA, where the is example seen reason, is see some a but LICAs. only it is time, the potency. in level tied

Nate Tower

Got Interesting, for the color. thanks you.

Operator

from will ahead. question Cantor Fitzgerald. Merle next with Eli go Our come Please

Eli Merle

for guys. me squeezing HI, much in. so Thanks

of D, you on of targets, Just on what the that maybe in and has geographic program, guess, guess programs B to partner sort your with B thoughts your of the can key D already mixed your clinical I versus I comment advantage results, versus I Thanks. and you could specifically target explain B little and differences rational, D atrophy perspective a at scientific geographic on Factor or terms the guess given in your the decision looking bit Factor Roche factor more Factor atrophy? studied between some

Stan Crow

everything. when do. like will factors one these for comment us so It’s were crowding and pathways Well, I typically, we of to in complement at easy and on, that look we

And safety, of benefit so, various the data all significant complement factors we compared we other offers and potentially all factors. had, – the believe we to look based be that at will advantage on that of the some a as both and and

based it’s want to you Brett, anything So, that? do on data.

Brett Monia

Yes.

which Factor course, was remember they for to interesting to really of came conclusion partnered a Factor macular D a this age-related better they and us that Roche target to different is that on don't Factor on little complement a the looking complement is bit, into developed antibody is pathway data Factor pathway also, want factors lies I models, the alternative – in get more certainly B the where it’s fine our Just annual relevant to And what degeneration. important why that at in with it based details, is program it the is but D. generated in but a and directly, we’ve target, B

what Roche So, pathway, is ones it's based in B, the on pathway. expect believe the does on In It our data, in confident based that Factor as not only we're said. would the too. Stan and we

Stan Crow

is And well. progressing the study

to to bring a think, many more to study Wade, but answers I some certainly close. going progressing. going we're it’s here, So, we questions? the is this have be how probably awhile, should

Wade Walke

That is the last one, Stan.

Stan Crow

year results you sharing look pixels the thanks and picture XX focus and of of the continuing big rest to on bolts, to making. That’s technology day. great. we’re this the everyone. to this forward and deliver year. December the attention and your look appreciate on great We Well next forward behind on as year busy advances this with some And And nuts we the that the we

Operator

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

Ionis Pharmaceuticals (IONS) 6 Nov 192019 Q3 Earnings call transcript