IONS - Earnings call transcript Q4 2019 | Ionis Pharmaceuticals, Inc.

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Operator

Good day, and welcome to Ionis Pharmaceuticals Q4 and Full Year 2019 Financial Results Conference Call. All participants will be in listen-only mode. [Operator Instructions] After today’s presentation, there will be an opportunity to ask questions. [Operator Instructions] Please note this event is being recorded.

I would now like to turn the conference over to Wade Walke, Vice President, Investor Relations. ahead. go Please

Wade Walke

Sarah. Thank you,

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

website Officer; our our me and on accompany Chief slides our are Geary, With posted also Beth Chief have Development today. to on Richard and Brett Senior discussion We Hougen, Corporate Officer; Officer; Commercial Monia, Onaiza Vice call Financial Development. today's President Chief Executive Cadoret, of

our us Vice will Senior Research of for Swayze, join President Additionally, Q&A. Eric

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

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

Brett Monia

building XX with and we're needs. success In to help Thanks, you CEO. commitments today to speaking unmet for the upon by Wade, new call. joining as that delivering good greatest medical excited morning Ionis’ to be thanks has I'm our in and Ionis everybody us very patients success. achieved Today, on on today's role years, my enormous

the among our most that and achieving innovative, the make people in believe to and I resources fact, companies today productive highest we industry. have In Ionis

revenue, launches year. SPINRAZA's of year Biogen commercial financial progressed WAYLIVRA this was to expects growth strong. the over million from So soon. billion to outperforming revenue place with financial XXXX R&D billion in again now year as we delivered an discuss new XXXX ended exceptionally strong will XXXX income recap improved $XXX achievements. perspective, $X.X especially our with in And performance a markets cash, $X of and shortly, over continued plans blockbuster Beth even last exceptional expand guidance. in TEGSEDI net was the and Together year's to with and and

Importantly, we momentum this of we already while areas believe in for the the potential achieved exceptional upon even these building greatest financial results to we're business success. that all creation. have this value more achieve investing And year,

in and across discuss, in strategic medicines our franchises. advancing X our broadly Phase four for our We primary including medicines. five XXXX achieved results As we successes achieved positive goal, Richard clinical into proof-of-concept We pipeline, neurological will cardiometabolic studies. disease achieved

medicines advanced new into broaden scope advanced and into six five last Ionis-owned. X continued which of Phase medicines we XX are technology. We development, advance Also, and the our we of year, new to

further new supporting that populations beyond to our we to diseases. LICAs In growing LICA new studies, to indications broad of X technology medicines the demonstrated LICA Phase our two into potential medicines overall. developing, clinical programs pipeline addition two rare for rare and both proof-of-concept XX four for represent our moving advanced into LICA we expand patient And

substantially. from And Today, expanding initial could by greatly technology positive is proof-of-concept oral clinical with studies discovery Additionally, later platform results delivery ever. than we expected aerosol with our scope these of results of initiated to our our course Ionis studies, stronger optimizing in year. evaluate and drug progress the this benefit

and short-term value prioritizing this potential and the pipeline programs believe create the patients over growing year, to have shareholders. inflection And Our greatest is multiple XX are broad, we we for business key of medicines with of value mature and areas deep, that long nearing term points.

we're also and X we more results year, as For plan our later enhance to medicines new several our this have exciting with reach to drug ten development, LICAs, Ionis-owned or pipeline including with medicines By Initiate least stage new the platform. already by programs technology profile medicines. replenishing year. studies report overall new at five innovative Phase more And underway, commercialization. this medicines Phase of the to our six example, X further of novel advancing additional end antisense for of six optimizing we're proof-of-concept administration drug clinical identifying and into new identifying two achieved and or studies targets advance routes continue

new our to goal reach these more through achievements us positioned XXXX. or applications ten of drug Together well

Additionally, our we of maximize this each ensure and of further the year, strategies to while pipeline our our we're prioritizing advancement of Ionis-owned capabilities medicines. the developing value growth commercial and

As strategy. thrilled commercial and recently executing leadership will XXXX business We're Officer. close forward. corporate part advances In team appointed catalysts, Onaiza building on our latest to experience leadership guidance Richard on Ionis Corporate have of and call questions. the out recap moment, and Cadoret to and results XXXX Beth will review Development our initiative, Her the we Chief will pipeline extensive be as a and our our the team as and achievements I'll invaluable with moderate and XXXX pipeline some in and financial planned Commercialization this Onaiza

insight to some into additional So turn at new Onaiza call who now, Ionis. Cadoret, role over will I'll provide her the

Onaiza Cadoret

at morning good and Brett, transformative everyone. the I'm for you, quite company. excited to Thank this time join Ionis

we said, unique position are a of in Brett strength. As

advancing of deep expansive, number a and with an have We growing medicines. late-stage pipeline

with delivering transformational team, a broadly are to us who strong technology severe needs. unmet And very we medicines to committed operate Our applicable, is mindset novel medicines patients. and bring to and to with enabling patients innovative most scientific rigor experienced importantly, have

the corporate of to am and commercial I assume company's excited leadership efforts. development

value in pipeline Our goal maximize the to of medicine our remains unchanged. each

to to pipeline plan to develop plan our We preserving organization capabilities, ahead, commercial our our our each of optimal growing commercial scientific pursue us and medicines Ionis-owned identifying this Looking bring of patients. strategy, medicines, pathway goal culture from by allowing the for we innovation. while established to

most patient to Pfizer will we such populations parallel, develop to with for and In the LICA. Akcea, diseases commercialize strategic as medicines partnerships, those X angiopoietin-like collaboration continue with recent largest with a our potential address establish

forward to We Ionis goals as they you updating our year. also strategy look Akcea addition year. new strategic potential work commercial the our medicine support pursue throughout of forward their a through evolves and continuing to pipeline to Akcea to look the the later I this on as

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

Beth Hougen

XXXX double Onaiza. R&D and commercial compared exceptional $XXX million including in revenue billion, by and financial licensing $XXX XXXX. was financially. over including nearly to revenue, exceeded our than million $XXX We Akcea-APO(a)-LRx driven year, Thank in $X.X you, of over for We more guidance significantly XXXX an nearly revenue earned Akcea-ANGPTLX-LRx. million

over income net respectively. Our revenues non-GAAP substantial $XXX in million $XXX income over and operating million resulted and of

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

$X.X our significant debt effective reduce for achieved while annual cash balance rate, our XXXX, sheet. percent most strengthening the us our our maturity our to XX% which to a which refinanced future by significantly price, interest eight one interest We increased achieved a dilution. results increased these We may and portion of which extend and balance exceptional cash billion approximately conversion to we of further expense reduced convertible our debt, enabled

execute The stock to returned confidence repurchase and to we debt through the late by our we repurchase to these months, last repurchasing our and near able value we we aggressively year. significant of underscores And transactions future commitment shares financial share the several and our have shareholders. in continuing refinancing resources, of implemented invest were common for company million X term shareholders program while to of our business. creating our longer value of also program last across because our the a Over stock the

At patients growth sales in XXXX million $X resulting patients launched where Ionis, and SPINRAZA in in a the U.S. year, in U.S. worldwide. SPINRAZA for Biogen we from there were nearly both more generated presence, growth. opportunity end has this markets significant the last more the than XX,XXX With of SMA revenue an important approximately Biogen Now growth XX% Biogen from milestone treatment net in achieved increase see commercial and addressable turning existing XX,XXX in patients and to worldwide our adult outside royalty to markets newly direct than through revenue. compared on of to over billion $XXX XXXX.

sales is commercially TEGSEDI prescribing in steady achieved throughout growth physicians countries. resulting increase and quarter, achieved growth quarter-over-quarter Today, And fourth a the XX% for XX particular In we physicians. starts. patient new note, in than XXXX product TEGSEDI WAYLIVRA from more of we in We available year. of $XX in prescribing in the and U.S., saw the new million

academic see both cardiologists, and to and continued neurologists of a community in We hematologists the settings. mix

countries also but focused coverage the only continued longer-term. Additionally, is working to Akcea Akcea’s is this PTC U.S. in to and U.S., in year. for additional Latin to access the to countries. market launching prepare for they TEGSEDI patients on translate Latin TEGSEDI not as American today, provide access have broad America, In expand Outside Brazil TEGSEDI reimbursement efforts additional is into into in Therapeutics

a for In an in devastating access from with disease. benefiting enrolling early of FCS treatment on Germany ATU, program. WAYLIVRA now medicine the only in patients market reimbursed the is the is approved patients France, this Akcea

Akcea to new plans this year. Additionally, in launch countries EU

Through the to Latin is FDA assuming Akcea’s this we effort, to Brazil can new re-filing foundation work the expansion U.S. have believe provide support approval. strong markets Therapeutics access this this in PTC working In of beginning goal commercial the year, for we year. continuing across that are products And with WAYLIVRA year. to America a we into both with

to $XXX value from amortization milestone and This pipeline revenue Our technology. fees, more the with payments, and in demonstrates than our XXXX significant upfront of license increases R&D doubled payment. million substantial growth

licensing million IONIS-HTTRx, payments, and $XXX disease consisted programs franchise, and AKCEA-ANGPTLX-LRx within AKCEA-APO(a)-LRx, revenue new advancing nearly primarily collaboration. milestone under $XXX $XX including our our million million R&D from revenue, neurological of including in including for fee licensee nearly and Biogen in numerous million $XXX several medicine IONIS-MAPTRx Our

from with nearly for also AstraZeneca. cardiometabolic XI recognize programs program our medicine including million NASH our and with $XX Factor Bayer advancing our franchise, several patients with We for

investments objective. technology XXXX $XXX pipeline non-GAAP increased our and XXXX operating in expenses Our with to driven stated consistent compared our million to by

made also antisense capabilities. investments technologies the we strategic reach in of in our expand can We and our developing believe commercial technology

The invest multiple ability an priorities. we meaningfully element of year financial to We enviable strength. key profitable in our continued be our a strength. This revenue position of in financial to sources expect to to from while earn is strategic continuing aggressively be

by revenue commercial countries. from as TEGSEDI WAYLIVRA We growing continued expect performance strong growth new into and we expand SPINRAZA and in revenues driven

We also medicines development. milestones we expect to as in achieve advance important our

revenue, in of revenue we sources this project than earning year. multiple more these $XXX With million

These $XXX to last investments compared AKCEA-APOCIII-LRx include are projecting expenses year. for in ongoing a attributable projected pipeline. making and $XXX expenses at program non-GAAP investments study our FCS We operating of million. AKCEA-TTR-LRx Phase are the year patients million XXXX The X we conducting to operating Ionis in this is initiating Phase our in increase owned to X

our pipeline. of have launches. those commercial XX to Because We not NDAs to We of support and need also into to of support outside countries the built U.S. do our we to that, necessary goal Together in ahead growth. advance TEGSEDI SG&A expenses expand these commercial team through investments means expect new the expansion to or U.S. we XXXX. in the TEGSEDI’s mid-stage more support necessary TEGSEDI’s This place the our infrastructure increase

in between TEGSEDI and anticipate launch we countries year. WAYLIVRA both Additionally, throughout synergies the medicines valuable we as realizing additional

WAYLIVRA our revenues. keep and we reasons, growing projecting these to while expenses SG&A comparable For XXXX are TEGSEDI to level

of to pipeline. and with update $X.X in provide turn on at our we strength to strategic call the priorities our have over I'll With an on end the to execute that billion the cash fully Richard XXXX, financial

Richard Geary

you, Thank Beth.

achievements. our with XXXX We mentioned, made important Disease significant Neurological Brett year Franchise. rapidly progress was As full a growing of

people example, with and and are studies tofersen progressing last with IONIS-HTTRx X For on entered for Huntington's people for disease schedule. Phase year SODX-ALS

from study data year, enrollment Alzheimer's than patients And last Biogen in completed by IONIS-MAPTRx an IONIS-MAPTRx ongoing fact, blinded supported Alzheimer's proof-of-concept ahead disease in expressed recently part X this for pipeline. their in enthusiasm early months with their We important disease of significantly late Biogen our already of earlier several has the encouraging licensed study. of anticipated, interim schedule. as robust Phase program

patients This study well mutations Additionally, with form most patients is these study represent disease. over Biogen which associated includes Parkinson's million as targeting sporadic patients recently of with medicine cause of Together familial initiated an Parkinson's common populations our both a as with disease. exciting LRRKX LRRKX the the genetic three of worldwide.

Also advanced for earlier development. and treatment Angelman syndrome into Biogen the year we of this program our

We progress excited are the our we're addressing diseases. Ionis-owned neurological with particularly pipeline with making of medicines

with patients Phase X underway enrollment AKCEA-TTR-LRx TTR hereditary of Patient study in NEURO-TTR our now transform amyloid is in polyneuropathy.

and for Our genetic Ionis-owned recognizing the recently need treatment EMA, was this devastating disease. therapeutic the Alexander of patients drug disease the with options significant for by orphan medicine for granted status fatal

including We and also Ionis-owned added medicines treatments development disease for Lafora neurological into year, several last additional disease. prion

recent the Phase cardiometabolic elevated in is and with patients X Lp(a). advancements franchise, outcome cardiovascular study to now Turning cardiovascular enrollment underway established our the in and disease AKCEA-APO(a)-LRx for Horizon

We're of our key importantly, CARDIO-T TTR us Phase hereditary AKCEA-APO(a)-LRx wild of in beginning corporate one pleased Novartis advance X patients AKCEA-TTR-LRx cardiomyopathy. to one advanced X with At last to transform achieve rapidly this our medicines of Phase of with initiated X type we outcome year, the our objectives. study forms and year, into and Phase of into how is medicine helping this development, four

demonstrating AKCEA-APOCIII-LRx and in lowering endpoint robust tolerability Both We key additional also line risk results safety substantial reductions factors achieved top with and primary Phase cardiovascular favorable profiles. from triglyceride for reported studies and their AKCEA-ANGPTLX-LRx. medicines proof-of-concept positive X,

worth by it's from patients think includes XX multiple we a treated LICA that have pipeline XX in with our outcome improved and dosing year. LICAs Phase noting characterized medicines X evidence treated substantial one substantial have that for game LICAs LICAs cardio-metabolic with X,XXX greatly and including development for a changer over now I we represents regimens, in people LICA Phase our number therapeutic and now our of With potency studies increasing today, to-date including data areas technology. real X technology

of IONIS-GHR-LRx for X several treatment and acromegaly, medicine the potentially the delivered into and IONIS-ENaC-X.XRx we Phase hereditary including of rare year, Finally, angioedema IONIS-PKK-LRx diseases. for medicines last treatment advanced our aerosol for pulmonary Ionis-owned cystic disease a fibrosis development additional

All on year. of to these track programs results this are report later

we numerous see, this can year. you throughout expect as catalysts So

have We in planned of in to FCS. studies study patients Phase six with X expect Phase AKCEA-APOCIII-LRx progress, X including a

in studies disease proof-of-concept of we clinical oral and report cardiovascular results Phase studies. to of expect medicines from delivery multiple our expect one We for antisense X

new stage with and and our together numerous XX advance said have Ionis-owned to position programs and into of many early goal achieve to plan mid marketing pipeline. our and well others new applications or into catalysts programs also We add to clinical broadening us development through partner more our technology advancing as These XXXX.

with call this close keep the to Brett this catalysts, I'll that continued you development. over the turn on to expect We many will continue updated to medicines as year the pipeline our we call. back advance of in to And portion

Brett Monia

Thanks Richard.

please today, end of families, patients, need. Saturday, the this lives transform like medicines partners the XX me Day to developing February I'd to Rare in our that we who Disease remind patients in everyone and is of Before and have recognition join this in been important advocates thanking in day,

living millions including about Ionis most broad more and worldwide treatment I'd diseases, on rare everyone awareness to invite like people options. of how Twitter to is Ionis without of follow to identified learn for the LinkedIn diseases, of adequate with raising range a thousands

the a in Ionis. Today history we're unique of at place

ever financial than key to patients inflection to greatest year we advancing value and that strength with plan Ionis-owned including our points. all This growing and aggressively more to our and numerous broader areas is the in potential value have mature create use for pipeline before shareholders, nearing pipeline. invest Our programs

and expand strategies by, antisense drug complementary now the that, platform. and our investments commercial medicines, capabilities technology the LICA will in with to new expanding strategies of reach discovering ensure maximize the I'd call developing technologies And identifying our of Q&A. our like to increasing different for that of administration the Further optimizing and open reach also of routes each example, our of for we and value

Operator

ahead. of first America. go from with Our Please Instructions] Bank question Gerberry comes Jason [Operator

Jason Gerberry

Hey questions. thanks taking for my

guys hoping, to have chance recent you the just relative on profile a recent had any Pfizer provide ANGPTLX guess I about with data discuss you're you can I to from thinking Arrowhead? data how interim that and and commentary some on program was the So, that

just ahead is question Second extension tomorrow some earlier Huntington's study. the from on data of

expect could the just those what terms of for table understanding the set profile can you HTT broader of safety the If importance of we in and updates ASO.

Brett Monia

the thanks question. Jason, Right. for

the starting primary for very data later the results secondary year. achieved that Phase and partnered So X thrilled program Pfizer well our We the of with we've stage were the that and endpoints, number significance very high from X And of with last positioning X, unblinding outcome, statistical Phase endpoints development. to this prior Angiopoietin-like we study. drug

But And forging next forward this certainly the very Ionis data data medical view second to Akcea X in detail detail very, extensive and plan through of Akcea and them determined. results. short is about and later stage a continuing quite for this meeting year and being steps we're sharing Pfizer to to Pfizer as in Phase medicine very to and go the data as the the quite have be gone in half on And this positive. are say the we excited at path We developments. through

to not really believe really like like for people's and But we we programs. medicine don't X. I exclusively our on targeting I programs. on Angiopoietin-like we very those medicine to first Ionis more on programs. focus close comment the I'm other best-in-class have and

potentially about bring Huntington's hear that quite near but the messages As Huntington's, root on Huntington's course track Huntington's you'll as the basic And on that's remains to future. to disease targeting the program the schedule. The program other – It presentation of in that targeting a Huntington's this There's Phase cause premier far the deliver tomorrow. targeting the the is have program the targeting is of XXXX potentially message that'll in presentations track. results from simply lot of disease. we X for on too, CHDI patients at medicine meeting for

is X excited remains is study very the track. on the Phase about and Roche the message that program

Jason Gerberry

Thank you.

Operator

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

Jim Birchenough

Also I and we if for all just couple we back on on share below which questions, wanted greater last could touch cash, the in priorities guess is it's up to capital just A allocation expect I year. talk could, suggesting additional from beyond capital million the you billion $X.X pipeline. billion guys. revenue guess the guidance if that. Hi the repurchases? your the payment, allocation growth whether $XXX progress than you million, $XXX for with broadening one-time of Congrats $X.X But should and on about

for comment So if on have, is you just Asia, the you I could that, coronavirus, with cushion wondering kind bit if exposure there guess what comment is exposure. you supply if could of your any chain final on the to your Thanks.

Beth Hougen

Beth. It's for Jim. your Hi Thank you questions.

with technology, So broaden defining start. off has the technologies invest And are and the on priorities Brett our to business team, to in our our to with capabilities. invest to further And Ionis-owned of aggressively I'll are start I'll we complementary our out capital focus laid Onaiza reach leadership allocation. of to advance pipeline across technology. as continue our now commercialization on the building addition strategy to

And of so be the area investment will company. an also that for

investments So I for see first significant being of the those allocated areas and foremost creation value company. our and capital to our

repurchase very we a today, In our investment attractive believe it it's share where terms is with of certainly stock opportunity.

then And for we what notes notes so due as that of it. that's in those a our or considering coming And the are something actions portion addressing half we a go And X% a we'll the about for year appropriate at and looking future. be into so. be we'll are have

Brett Monia

at happening. we're don't were we I'll to respect how chain. well our coronavirus, our potential to question. with pride to been our trials Jim, preparing along great talking way. any on chain in And We've clinical Richard as supply epidemic with we they on and take manage the Ionis, this employees, supply our ask to how protect focused see the And potential hiccups about as crises before respect going Beth. we Thanks for comment

Richard Geary

Yes.

on Specifically supply of also that for are which our not up on the not opportunities all all China chains pack supply be only then drug not supply chain, the here. supplies manufactured And our Certainly course, all questions. we're clinical dependent API, one may but China. on also of drugs multiple dependent right are supply, of our of

Operator

ahead. Our Rajavelu comes with from next go Esther Openheimer. question Please

Esther Rajavelu

Thank for taking my you question.

from driven sources? of a And SPINRAZA How quick much follow Just XXXX is up. me. guidance couple quick a ones versus other have by then your I

Beth Hougen

it's Esther, Beth. morning. Good Hi

commercial was U.S. you for SPINRAZA Biogen and as XXXX the can outside a and portion we So, growth continue SPINRAZA imagine of in significant States. and the revenues United see frankly to our in

expect being as course our WAYLIVRA to see of would significant SPINRAZA have expand unfolds. I to a and commercial of the Europe into TEGSEDI in particular. year So, adding in portion then countries revenues new And that, they continue as to we

of of our fees amortization substantial have then on revenues and continue payments by anchored built and base we advance. R&D by programs upfront licensing milestone in as And with very milestone a and payments significant then development to partner

Esther Rajavelu

resubmission for And U.S. need of you re-file what be to the the WAYLIVRA, marketing Thank able to complete authorization year? the do this you. in to

Richard Geary

filing Nothing as to writing three for great very increase patients added us exposure access Yes, open-label expanded a are safety in supportive. as well the Richard. complete which as to up question. the This of this, studies and to except in in is additional fold about data, all so bring of well is all as the results, and being expanded

Esther Rajavelu

year on to So you're that's, saying? than expect we later should what the sooner happen then that based in

Richard Geary

to guiding we're expecting we this but file certainly to are timing, a think I year.

Esther Rajavelu

Okay, thank you.

Operator

Paul question next Stifel. from go was Matteis Our ahead. Please comes

Nate Tower

question. Nate on for taking This thanks for Paul. the is Hey,

of Firstly structure you are I markers beyond for study review a up? X/X, what specifically then you Phase the collecting you efficacy and or can are exploratory PK/PD the and LRRKX follow and have safety

Brett Monia

haven't details we of LRRKX. Eric, on the disclosed the much

Eric Swayze

first in that mutations of genetic exploratory look and believe secondary safety, beyond LRRKX we're course treating primary there's it's is can you as the imagine study, at. with patients population don't human for so endpoint both And we've the we'll well as but I things disclosed endpoints of biomarkers general a sporadic. that lots

Brett Monia

as come that study the will Nate. on More Yes. program out matures

Nate Tower

of as the be Are consider into you we aspects if gain correct, and primarily from Well, the maybe all a worried FTD that's well? thinking program. about second to about the would That's CXORF addressing are at CXORF of in moving of the pipeline loss And going right on secondly, function you CXORF. way? question aspects function this ALS

Eric Swayze

yes. both. Yes. So Good questions

You CXORFXX-ALS is are that which location of CXORFXX targeted is, An and our gene, drug is the about it which the such only mutant are the predominantly aspects FTD. the it correctly thinking causal decreases CXORFXX is we important form in function about we by addressing it that gain of for target the of gene. thing

we all, there, function don't decrease So, expect an is in disease. fact was or component important CXORFXX there to of that safety to

that I disease it would process. that However, preclinical data function emphasize is much suggests gain of a really of pathogenesis the

So, is I ours one and on many directly mechanism. medicine another that like on think that's of target that's

FTD out point in or that you we're drug and FTD. Biogen implicated is actively ALS both considering to and as move CXORFXX in with how forward And also

Brett Monia

broader addition And that genetic of added Yes. by for course ALS, as third ALS, how Phase Biogen, for of to we're into forms on just you SODX little And forms our Nate sporadic another ALS Biogen and expand the X a I'll attacking we form in pipeline of much bit, CXORF and others medicine one. reminding in a a recently is program SODX with ALS. related non-genetic second of Ionis ALS

press think core more ALS full will a on doing there all and we're even of forms So be I coming.

Nate Tower

Thanks guys. to hear. Great

Operator

and Needham from comes Chad Company. ahead. go with next Please Messer Our question

Chad Messer

maybe are your enrolling got some rolling in And for thanks. easier have those sense the for trials experience guys You and two start for Phase there to studies with or some timing? running. is that commercially harder taking can that that enrollment available enroll now it's the just options space. Maybe these expectations you've X Can patients? it my we you now set Great, up Thanks questions. program TTR

Brett Monia

Thank Akcea, we're includes for both with population, you. cardiomyopathy, and studies and underway hereditary we're the Appreciate wild-type the Chad. the as larger same both polyneuropathy excited. very we're Phase label X question, targeting a Phase which population, essentially where the separate patient Ionis now TEGSEDI. X two in Yes, the

to in looking XXXX both this complete the As these And is enrollment far well. timeframe. go studies expectations for as actively is XXXX to for going studies we're underway and

to you cardiomyopathy patient the in, competition related patients trial depending actually standard-of-care, which to for and be fact the stabilizers enrollment. standard-of-care for that's impact geography The how to may design we advantages that. We could take believe As for be hindrance to happen course particularly challenges enrollment no allow study. and on that the enrolling many of therefore our our stabilizers offers that

work get of to patients them that awareness, well and just to doing Pfizer speed disease. the the tafamidis, and the like the folks make aware help our to work I our are great So, doing disease identify people on will we’re that of as as enrollment believe enrollment this

amyloidosis Hereditary polyneuropathy enroll. are is TTR there There population a are multiple that. patient no clinical and certainly for in going rare to about There's question well. with be challenge approved other to drugs this trials progress as

the strong and CONNECT relationships good that work as identify about and to patients with AKCEA as able they're our study track We're confident work investigators feeling we're our roll from to NEURO-TTR Akcea with this with study. doing great doing X the it. investigators be and we'll However, is Phase well on

fact is the to is label course, open advantage that the it's study. of study other an The neuropathy

X to the will that study So significantly be on comparing to a We're historical will Phase sample not smaller patients which NEURO-TTR control, going be our size the well placebo. help of fact of than results as study. that enrollment the course is as

be I there for manage we them. think can challenges So will polyneuropathy, but

Chad Messer

or you you study is something on an trying could And be just get, earlier the meant sense Yes. then for file are efficacy? on interim, that just to to an has that polyneuropathy

Brett Monia

at on end X our at at the data. trial, will high the you we at remind eight the decisions, saw look significance be will statistical course months. the decisions Phase with We TEGSEDI well as driven interim XX TEGSEDI of Those that analysis NEURO-TTR make data that, study months eight and data want to as months at in a based of

patients and as at So able to historical at go. TTR want if we'll does as eight the we to a to and that as possible comparison Akcea controls off get be the study rapidly it supports it data very and our nice to this we we compare LICA have interim medicine analysis months in

Chad Messer

All right. Very Great. Thanks. helpful.

Brett Monia

pleasure. My

Operator

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

Vincent Chen

you questions, from a Great. couple for Thank much me. taking very the

then one tomorrow? we'll a first the including for earlier, the wondering, topic, safety was on of biomarker on updated and in we product sort just TEGSEDI data terms sales just relative of biomarkers U.S. in And provide What WAYLIVRA light safety, for guess So I if the the a in data, on versus Huntington's your the neurofilament? get some I on look including the somewhat data I TEGSEDI and in of chain. O-U.S. like question looking mix updated view color what following should terms you different does be up then any outcome good a versus neurofilament updated data of could second guess the on

Brett Monia

on like to you would Eric, touch biomarker the data

Eric Swayze

and is way the with Brett to of Yes, X the it progress want what's extension presented I be I'd we're partners is who mentioned, drug being the the is presenting really the And advancing the of Roche, trials an ahead but data really profile pleased and has out the been pleased note drug of track presented. outstanding. that is with of already is get our read also as mean, and they've to I Phase in should of scheduled tomorrow, XXXX as on The it. data that don't safety Roche presented thought very

Brett Monia

And is as program. key the track Roche the on it's moving and said and I way that X looking forward about message the earlier. is on is the trial is very excited see is bi-monthly it, The Vincent Eric The as fact I pace. Phase Yes. excellent dosing said

a of We on roundup, sort our the summary a Friday of presentations short on Huntington issuing drug. a all be will

we'll post to So analysts. you and website we Beth, to would also with… the that we'll it, capture can our take of it distribute our TEGSEDI division

Beth Hougen

Sure.

vast – into as XXXX new just late WAYLIVRA countries only see we and forward the launched So And moving strong quarter-over-quarter, can progress the Germany, to for and imagine year. important XXXX. pleased TEGSEDI. think was we TEGSEDI in continued was steady of to XXXX an in growth since you majority quarter-over-quarter WAYLIVRA with revenue that this this And We sets in the foundation were really look year. growth and in

between U.S. not of sharing terms at that point. information In this we that the are and an Akcea ex-U.S. mix and

Vincent Chen

very you much. Thank Great.

Brett Monia

Thanks.

Operator

Our next with from Ellie ahead. comes question Please go Merle Fitzgerald. Cantor

Ellie Merle

for taking. so Thanks guys. Hey, much

in program this you're perspective proof-of-concept exactly oral for guess committing that you proof-of-concept get constitute data. your Are the to for? would specific relative XXXX proof-of-concept us oral? you'd you Can through on in you're levels knockdown the for constitute that we'll your walk of delivery? can that in through I from what walk Just be to there just perspective running And Or specifically of would sort Sub-Q now what oral parallel what protein readout? looking

Brett Monia

Thanks question, the for Ellie. Sure.

commercially predicted and excited that the – progress the that once in all is mentioned, data you and to aside partner that mind So achieve have data that readout poor tolerability readouts of was our half from is More yes, does evaluating data clinical and we're oral second mean as And biomarker with that the with extensive we that port expect study, out. them AstraZeneca. trial for designed this on support proof-of-concept the the ability crisp commercially potential have plan with than trial dosing. of of will oral we have very data in target that our pharmacokinetics preclinical effects we'll about that the reduction year. viable delivery viable safety What clinical that the to on that, from intense in would is bioavailability, to on based – assess reads reduction

if other already not on sitting this – potential we're and research enhance active where clinical the oral of topic substantial now. right you Ionis we are at oral that to The on, ongoing making work formulations beyond bioavailability. study even We the is our increase in of that development of in still improvements significant have we for to the will, have further formulation expand like actually aspect I'd delivery in that

on those bring So, number we're oral and right as development the even an Ionis AZ where delivery Further, we're into continuing the to we've of hoping planning we're in pill that future. make collaboration. advancements products behind to the an soon Ionis-owned bring oral a in identified after to

commercially oral we're feeling for pretty was and our oral viable going there will the front if – making success good you isn't many quarter in we're have this platform. in So that to original

Ellie Merle

Brett. may it programs very it. just have follow-up I for and development. much Got you one quick Thanks of for lot mean, just like the I if a seems color in

this you know, You have year. readouts many,

your in think I size program excited proof-of-concept you which readouts you that called or are datasets most four you I have out about? this year. which guess,

Brett Monia

we drugs, – one. nature, rare the table here, which all choices. And to went really I the for buckets. well, then we the more we'd potential than XX disease Well, it's get six around But the be term, pipeline is. the it mean have shorter of in transformative have in hard different probably of if pick and to

right. a opportunity study And We're Lp(a) and well a hard form the the far The populations, the to of and large include mean, it's for patient with not Novartis ahead have population. selection broad so drugs. competition. is so in of is Phase – choice, I such a X any extremely is opportunity in commercial We enormous. disease for this patients progressing

one. you to that include So got

Biogen On the ALS, rare said are And I to our there of towards Ionis is, now. a disease this earlier, bucket, with of neurodegenerative forms other as I on treatments terrible disease. press no hand, I in analogy mean, doing are – used we I gravitate all ALS. for the full-court which have

drugs. for multiple ALS readout program Our to SODX is first of the due

patients next the And year. drug in showed going the in of reminder with SODX-ALS. that provided Phase evidence a – really remarkable to three months remarkable As short X really study efficacy readout now, is

for have drug program a X ALS. sporadic due we we And readout program. referred year. Eric that’s Phase That's have a earlier, CX also a next now As

but our towards are take day So and I drugs would about two lots very lean I be pipeline. talking There those could all about. excited to programs, lots there of

Ellie Merle

Thanks that Got color. for so much it.

Brett Monia

Sure.

question Buren Van Sandler. comes go Please Piper from next with Tyler ahead. Our

Tyler Van Buren

morning. Good guys. Hi,

on at. a looking technologies up complementary that your you comments brief capital earlier potential you're had allocation mentioned on Just follow

get just to your specific I example tissue there RNA want is emerging So out tissues, and technologies. updated some thoughts specific one or antibody There's in technologies conjugates. interest

just there. to I your get So thoughts want

Brett Monia

to a the identity allows complement that us LICA – to that position reach – be with – We're and such new novel targets internally antisense as thanks, So of identify on pathways to new academic financial that thrilled strong strong linked. technologies. invest antisense And more platform continue targets collaborations of to of to such collaborators in selection novel and pipeline to drugs to position working and externally We're even of our lead that. as do replenish reach than with in commercialization, We're technologies cloud genetically are Tyler. to very work for companies platform novel the genomics well. expand with working with we're that targets and our a investing in through

year, a reach we're as that of – we're identify company I company strategies, have types. novel that what one progress. types Just late newer of approaches their a targets Antibody with making developing and LICA are expand the we tool just view novel to systems, conjugates last genomics on to derivatives new and ourselves. LICA strategies taking. strategies is aspect the of to working antisense in great we toolbox and with of a to completed another with we're partnership identify We're cell organ one

work, they strategies to to that cell less today. are sensitive believe we promise types So to that but are many the have have different that into I showing potential getting antisense

Tyler Van Buren

you. Thank Great.

Operator

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

Jessica Fye

Good that Thanks for Hey, Can to focus taking up guys. the Is you've WAYLIVRA? the talk angiopoietin-like indications LICA you X. going Smelling of one really a that just you earlier LICA? little ApoC-III ApoC-III question. about see questions bit on discussed the relative about for how my on positioned product to afternoon. previously

Brett Monia

the I Thanks, Jess, appreciate and question.

that on in the positive they on – two they but remnant lipids significant road. a between the all, data – there LICA It down that these these the effects data overlap atherogenic end effects that difference control, drugs announced very of is HDL, cholesterol, that we're first X with also LDL-cholesterol manage, Akcea the both programs. triglycerides, Ionis thrilled enough top-line we from studies at positioned our believe medicines Phase points, on and primary and of was partner course well results different profile a with angiopoietin-like X they presented in C-III both cardiovascular disease. they significantly Akcea our Phase angiopoietin-like patients course, of as X differentiate the sufficiently angiopoietin have in well will X that both that Pfizer LICA, both for unblinded for So, is those and top-line trials X of for are There

angiopoietin-like lowering such ApoC-III there LICA just cholesterol advantages on in on as triglyceride a X lowering is other and significant that most potent number effects think positive target and drug today I the brings to effects HDL. triglyceride out addition of

So, and is very the differentiate I in cardiovascular think, they disease Akcea. of additions cardiovascular important – to and as the be enough Pfizer the meaningful treatment our to partner future

Operator

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

Unidentified Analyst

for for on two the good This I morning. have questions is you. line Rick Mani. Hey,

about you're could commentary of speaking Akcea how based it utilized make is broadly sense sense you think of for us internal the pipeline, the relationship oral that how with Do could the into evolving view wholly-owned of the the is a you the potentially cardiometabolic Akcea? currently really your therapy? you any with directed back like second, on best think do I'm Or the for any a remainder specific Do think set at the company? looking point bringing think as you just increasing just given more pipeline. relationship viewing technology Akcea oral And would focus areas on to in any about delivery First, on give opportunities delivery liver fit you your indication? focus be ASOs. a for good just

Brett Monia

Rick. Thanks, thing. Sure

to so the moving including clinical TTR progress have LICA liver particular the forward to oral that but oral it'll reach liver the the said is any we're It's also the all targets on on forth. impact additional exciting really just on X trial, or making in so the – about it. successful, as So, clinic. drugs that. already Factor And and has on earlier, studies oral or liver we're what's delivery XI, programs, it's potential target one I of like in That's angiopoietin-like ApoC-III And of one that of this questions – work the we've identified programs. potential our we're focused our not

we're So, changing the successful. have is reach current potential. to lot that's, gears. also a on even continuing are mentioned, We're a of I improve which it broad does very And formulations, showing

about excited very to Akcea’s we're Akcea We're success. committed and

We’re confident successful. be going to very they’re

is owned Akcea different the potential drugs these one our in – Our to lots a is there's already new addition pipeline a of earlier, of as to and fully growing medicines in maximize expanding later ways the of wholly these commercial mentioned opportunities rare bring owned year. of And each value the and of in Akcea we disease certainly are or pipeline rare negotiations medicine disease medicine with available. about we're this discussions those that options and

they're start now later of that is road the TEGSEDI relationship continuing It's Phase deliver their strategic X for exciting in an with now. of having ApoC-III they studies on undergoing and their X goal. Akcea, in on And quite as pipeline the really agenda is already well but WAYLIVRA with LICA, this down in which year with plate. As pipeline agenda and or U.S. FCS in and Phase very a with far two and WAYLIVRA that's and planning that's topic getting pipeline doing agenda, LICA believe progress. progress launched, comprised track have right discussion Ionis, to on lot to TTR Akcea and approved we getting full future their the the a both

I say about now. So, question. have I your that's what that right to addresses that Rick hope

Unidentified Analyst

Yes, Thank for question. does. our it you taking

Operator

with Richter go next comes Goldman Salveen The from Please Sachs. question ahead.

Ross Weinreb

Specifically, just for what this here? to significant TEGSEDI And some drove factors grew and I up. of your gating think of physicians This on to comment the and follow And these us color thanks. where both of base q-over-q. back? for trying looking I'm then about a for uptake? like on. help is WAYLIVRA coming additional that prescribing factors TEGSEDI Can you X% have the about launches are these sales the some right, to Ross I'm All product breakdown understand

Beth Hougen

Beth. it's Hi,

WAYLIVRA countries with Akcea was and Europe. in population with as So, TEGSEDI And the TEGSEDI they now Akcea. primarily of right WAYLIVRA WAYLIVRA, relatively Germany and launched there in because But Germany a small in out Germany that's is expanding I additional breakdown is patient started across said, XXXX. in late

product majority were And portion so so WAYLIVRA, a the in XXXX vast were small, very the revenues of small TEGSEDI.

Akcea of a of out function doing their terms in team into the academic genetic In getting growth TEGSEDI. physicians, the that number be could of patients centers, testing but the physicians with patients, community that settings, testing only for also into the of to are work that the program, is genetic lot a utilizing expanding Akcea program who is prescribing the potential identify not

and on those getting then those board physicians getting with identified patients And onboard TEGSEDI.

think program coming is looking also is year where with a that Akcea and to other programs growth that's grow success of I right of from similar frankly this TEGSEDI So and in that revenues programs. engaged lot to working continued that grow revenues the to and we're forward WAYLIVRA now the

Ross Weinreb

it. the then Thanks, And really about, and SPINRAZA plans just year how helpful Beth. use? later market of this got should be rest there. we it, impact Got thinking likely of finally, That's entry

Brett Monia

all to And are on going comment of and competitors very data SMA It's – on road. SPINRAZA when Ross, is for bar for mentioned presented. to Biogen that a a Beth and be Well, the competition so care has continues to been efficacy despite hard high be set the for little meet competitors it's a significant the has population. setting actually additional expecting SMA, have SPINRAZA down very safety growth forms the as foundation to start. potential earlier of I'll Sure. global continued of competition SPINRAZA to

want really this forms risdiplam. confident to for I other the to all to years on we're drugs for potential perform very for year comment SMA so, SPINRAZA Suffice say very And space that in to that come. well like don't continue will and this of

Ross Weinreb

Got it. for Thanks questions. taking the

Brett Monia

Sure.

Operator

from ahead. comes and Please question Our Laidlaw with Jen Company. next go Yale

Yale Jen

for the Thanks questions. taking

going is to well that for as a pivotal in refile to going year LICA the are the as one, ApoC-III one later you're linked study that’s file – on this FCS. little which WAYLIVRA guys Just you a previous bit start

events? relationship the is two there expectation those connection or or any So of

Brett Monia

and a to of data be two on the way a not in We are are no an filings patients. Hi, that which linked potential the having And point have same question, – lot of but We're ApoC-III the refile medicine indicated, a in make one Thanks in the obviously – later the start-up Akcea population, It's in question. U.S. your approval obviously, these we the the Phase trial LICA this – are could is planning and of should the important with any Those fortunate moving. to together. WAYLIVRA. this on productive having the in urgency don't And lot this way Ionis for any to it’s with get way that the X is now linked ApoC-III moving These approved see in WAYLIVRA LICA. we're case for discussions Phase no more impact position patient of to we the that any U.S. linked very independently X study forward we year. have have FDA envious are Yale. with Richard events way. as

Yale Jen

which much maybe And repurchasing quick that stocks Okay, this follow-ups, how left? just point at is still great.

Beth Hougen

the We program that stock initial repurchase executed on program, board approved. our the have fully

to our now, it's may looking earlier expand right is that so be So investment, is think that we future. a now. And stock great up the where wrapped said right as we with trading into I

Yale Jen

great. appreciate I it. Okay, lot. a Thanks

Brett Monia

Thank you, Yale.

Operator

Ms. Wang, Wang Our your is Please open. line with comes question next go from Barclays. Gena ahead.

Gena Wang

Oh, I taking my Thank you questions. my sorry. much line very got muted. for

data do you impressive thoughts, I one SODX what data regarding you know, Brett, ask or consider would expectation. very question data? you just any the a So have as meaningful

Brett Monia

Sarah, running question. last Gena. We're long this time. will the And be Thanks, long,

So SODX-ALS as I earlier, excited about program. mentioned the very we're

clinical the executed they And three scale so really the as of made basically that – recall, cohort was X the were looking is program, study think that toward only very months. a than excited in efficient mentioned about three trial. new simply end very where the function were the in the remarkable, that the the improvements And respiration We're And points ALS they and functional really earlier, rating respiratory X results the saw added in stunningly that positive. same to that Phase I you months X the are the – that study endpoints Phase in everyone some we’re months. in it at looked was end so Biogen which a If which at efficacy three essentially were quickly. to start ongoing that way study. that Phase about study, they just X points Phase can even greater we as clinical study

Gena Wang

you. Thank Great.

Brett Monia

Thank you.

Brett Monia

today. obviously on So we wish We're you to and Thank our very updating year everybody for very and for thank everybody a us you as progress forward the again you look day. great unfolds this year joining excited much.

Operator

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

You may disconnect. now

Ionis Pharmaceuticals (IONS) 26 Feb 202019 Q4 Earnings call transcript