IONS - Earnings call transcript Q1 2019 | Ionis Pharmaceuticals, Inc.

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Operator

Good morning and welcome to the Ionis Pharmaceuticals Q1 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

Thank you, Gary.

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. posted accompany our that on discussion website our slides We've also today. Hougen, Chief Chief are Stan Officer; Crow, of Executive on Officer; Operating me and Beth be Damien Chief us the call Monia, Officer; McDevitt, With joining Chief Vice Senior Research. for Financial President Bennett, will the Brett Chairman and Business Board Officer; Frank of and Q&A to to contains I X, would attention your draw like luggage Slide statement. which our forward-looking be on making are will which beliefs. and we our expectations statements, based forward-looking - begin current We'll risks are differ may actual statements to These subject uncertainties, and our results and materially. certain additional filings you encourage our in I SEC consult discussed to detail. factors risk for the the And with turn I'll that, Stan. call over

Stanley Crooke

thank us good week so morning, Thanks, had culture. thanks. of various for busy on you everyone, Wade, a We've today's calls, and and joining

week, creates have medicine. depicts patients unveiled force that our technology, may and our as have life. passion and our and position this science commitment think we expresses a of for that noticed We the our new to the for culture, our we life it better transforming You that brand. our reinforces It business model

So now let's turn to QX.

the year. to momentum We last start, on from are off a building strong

Our the more achieve or exceed perhaps financial our potentially, putting first quarter to quarter track results on us financial significantly outperformed guidance. well XXXX first likely, of XXXX

having SPINRAZA new X-plus early disease, fatal broadly pipeline established two in continuing results and financial of with is and strong treatment SPINRAZA's benefited placed new after commercializing continued SMA, experience investing and in that, our SPINRAZA's these Even results in to in patients the in with quarter the our treatment Recent through with of Biogen is demonstrate of forms been today, for and performance generate in benefit clinical gain also in is continues results years. blockbuster years standard-of-care in SMA. time of over marketing, technology. six next over patients including contribute business, resulting treated previously some and not to has our demonstrate continued patients the year these and aggressively all ground SMA. advancing the durability medicines untreatable of these achieved we the benefit but first - - financial results. the benefit. significantly durable, with to even Importantly, With that further to only better increasing is understanding longer of while

of here. some, in treat that So normal, infants develop most is of that like SPINRAZA they we those about become That before symptomatic, information we to have seem babies. healthy all the leases infants if

So longer better. treat, the the earlier treat, the we we better. And the

look with new available the EU can making pleased to forward and need the of addition, sales have We the the are we're quarter less now SMA been two into this just are it. patients launch, in our to access also We medicine to this in and EU. are over antisense in full on the we anticipate initial Having follow-on exceptional very encouraged first are to soon. bring we offer with WAYLIVRA potential moving the development TEGSEDI receptive by in U.S. FCS feedback and expanding physicians new approved same the medicines TEGSEDI, benefit frequent progress SPINRAZA In working good with Biogen as years. dosing. to medicine medicine with seeing we FCS, value that for the who the from recognizes the and Payers patients. completed Akcea third is we patients WAYLIVRA desperately and getting that

near Our achieved value-driving Phase III milestones quarter. pipeline also III Phase in of and this significant programs

us the ongoing that trials on XX in the Huntington's a diseases from III the our affect Phase medicines technology new debilitating And rare and broaden that's very support lives not data and performance enhanced the III targeting our advancing these four toward LICA include examples LP(a)-driven us advances with disease LICA common next medicines - Including for to Phase our The many SODX-ALS least to these at programs these of Phase patients. have disease of two of these program of entered TTR patients our wave diseases potential have enabled in millions those pipeline and medicines to III improve our we fatal agenda that that of the only clinic of year have enter but, for These medicines and address, end development. by diseases. year, the of course, exciting of trials clinical amyloidosis. and and medicines more medicines cardiovascular Our continue for patients. this of programs medicines, are to potential to next of

Now I'll turn the to update. us provide financial to see over call the Beth

Elizabeth Hougen

In this Stan. of year, significantly Thank we the year. outperformed quarter of last you, quarter first the first

more doubled resulting nearly the to revenues $XXX both than on in GAAP and basis. million, income non-GAAP net total operating Our and

$XXX the in were Our quarter and first $XXX respectively. and non-GAAP million, operating net income million

further the we $X.X billion of fourth strengthened sheet cash. our ending addition, quarter by balance In with

quarter, last track year. In guidance continues as compared previous financial in the gain to our the to rest we the commercial achieve $XX are year, on strong clarity more first update we we net to guidance, $XX we With And that multiple non-GAAP on are optimistic revenues of years. results, as of million the our exceed on Given million medicines have possibly income. were market, our may and commercial grow. we our our revenue

TEGSEDI SPINRAZA and was product Our increased revenue of commercial sales. driven by substantial the contribution royalties

year. SPINRAZA compared $XX to compared We year. up earned are of first increased of and in We last to XX% SPINRAZA's by revenue quarter sales Global increase global to sales from compared last encouraged by million fourth million, the quarter. $XXX better-than-expected commercial XX% the XX% royalties

discussions from reminder, factors, is have and to the the seeing highest $X completed including The U.S. we Program SMA growth first Patients switching nearly in As of at U.S. At the EU multiple of patients also royalty in we as Worldwide also month, including by X,XXX Access is are patient which markets. show penetration well other Early significant good cardiologists, clinical geographies new the end of first both patients significantly the competitive the their TEGSEDI this strength are TEGSEDI the more motivated first patients million with around estimates, world. growth. initiating to penetration there committed XX the in process patients trials. initial in TEGSEDI progress sales for to first a patient our of while and quarter by around population continued In with and hematologist. last SPINRAZA because a on TEGSEDI are than patients We be extension SPINRAZA's rate is treatments. from are quarter was to were global being SPINRAZA U.S., the the countries. reached U.S. Biogen open-label and major year, adolescent seeing potential study. since to Last transitioning performance, hATTR are continued drive and starting in in on are $X TEGSEDI to the the and a issuing in reset continues each Patients end the reimbursement sales supports the increased access as X,XXX driven TEGSEDI U.S., than expanding strong from Notably, quarter, EU. neurologist and adult the commercial program launch SPINRAZA the with adolescent SMA expectation access the treatment treatment for of outside England. uptake patients globe, in expanded patients medicines that Akcea by from and treated larger Reimbursement over over the benefit positive million from believes adult among patients SMA naive EU year. and late countries, tier. ongoing patients recommendation Akcea growth expected treatment. in commercial NICE TEGSEDI physicians launch driver of and In began primarily is

continuing regulators approval. Therapeutics towards addition, to Brazilian is work with In PTC

of earned approval a rollout across additional to to to the deliver next the in America, our of revenue earned strength a important launch and continues because revenue with similar in online from first groundwork recognition from Germany with Latin for a It sustainable is In the of particularly approval EU element EU EU EU revenue. our launch WAYLIVRA, recent patients And a it payment we $X sequence to $XXX come R&D TEGSEDI million R&D XX% R&D million countries next expected to in of accounted the significant our preparing WAYLIVRA a of the launching PTC total In the Akcea WAYLIVRA revenue is be quarter. of there. With in We to which portion milestone laying year. to large PTC. non-GAAP is us. we funds anticipate expenses. for source the successful of WAYLIVRA, our financial of collaborations, quarter,

part R&D the earned quarter license in driven significant in we large fee revenue from was by the Novartis. Our first

payments of we we from more the partners be expect and million earned as create from our R&D earned In full the quarter, our For value we commercial payments year, $XX the mix advance. and upfront first of the we to reflects balanced. revenue milestone medicines amortization

first revenue, when earned significant million For licensed advanced Roche License they demonstrated IONIS-HTTRx a R&D they example, into when an Huntington's as fees from AKCEA-APO(a)-LRx. $XX development by million Novartis disease. Phase are payment from of the earned in our additional $XXX quarter we element we for milestone the III

license our their fees when license to partners. also earned technology companies We

which Alnylam they from closes, this $XX partnership our announced For earn we to a last Once Alnylam. expect example, transaction to Regeneron. in million technology month, licensed

to to from same of globally. expenses This primarily compared a us can The due increase TEGSEDI the investment our fact to year. slightly commercializing our commercialize pipeline apart such and increase in $XXX clearly quarter Our expenses, increased XX non-GAAP over last peers. million advance prepare to sets the in quarter we was operating medicines in modest that TEGSEDI first the with

We this income and will to during quarter. primarily generate tax The U.S. we income our the that federal recognized our expense also was million expectation due taxable $XX expense of year. state in income tax increase

has Our two expense income components. tax

federal our to relates component taxes. first The

fully taxes to tax to we therefore, with income We paying don't assets our federal and cash. our expect anticipate use U.S. federal offset our taxable deferred income

will The state other estimated to expense pay the component we for our relates cash in our taxes. taxes of income

we year, make have XXXX. recording the payment this the majority to quarter are liability first we until not of for will of the the expense Although this

in Novartis achieve in because quarter, more to from financial financial than of full possibly on already exceed of the of large Our approximately payment $XXX we two In year XX% track and our first million. part put achieved guidance results have us XXXX we the our the first quarter guidance. earned revenue

track exceed the We more are be million a of possibly non-GAAP on profitable of on line income to guidance our operating and and also basis. on than non-GAAP to meet $XXX bottom

at $X continue approximately have project that billion will in to We we cash year-end.

financial drive need. We to the we call year prove remainder value to partnered an the have patients with results both Brett platform strong And focus partner's continue provide in future, confidence technology update accomplished sustainable advance turn deliver innovation medicines. the pipeline. to on growth efficient can and that and I'll to our of continuing to this our into while successfully looking ever-increasing that, leverage over on to these maintaining our Ionis-owned to and resources we by have our And

Brett Monia

Beth. Thanks,

see the As across a successes XXXX can strong off business. numerous already is to achievements from with slide, important and you this value-driving start

launch our WAYLIVRA a And WAYLIVRA to expand Akcea's the WAYLIVRA risk this access of already infrastructure announce patients in Europe, people well Akcea's U.S. share much as underway. pursue in we to And the Just and confirmed extremely European who and path are commercial for in the discussions approval Germany, pancreatitis. with TEGSEDI to with pleased and same to at of week, high FCS, forward Because team to preparations continue genetically is Akcea we are worldwide. Canada, were WAYLIVRA in place. treat regulators

programs. our is technology attention believe neurology We platform of to neurological to treat leading a Turning the drug antisense range discovery devastating diseases.

now an year, programs. neurological refer important studies presented for SPINRAZA is of the our Phase and such, we This I/II and ongoing our to conference AAN the of highlight IONIS-SODXRx, the of study tofersen. year us partners every Biogen as data disease progress opportunity from As you which

to and in In choice and show SPINRAZA and with study the disease, open-label increased the benefit. longer is as durability increases data for addition, the patients SHINE safety Roche Akcea SHINE presented in trusted greater TTR SPINRAZA's and established, with SPINRAZA up SPINRAZA reinforcing to place patients, NURTURE continue to ongoing benefit profile, all open-label the change extension data the favorable longer study SMA presented study treatment it Huntington's from data for open-label the treatment durable, years for extension time resulting neuro benefit from IONIS-HTTRx In the TEGSEDI. to no treatment study children six benefit adolescent patients continues demonstrate SMA with with some of extension over in SMA. only of but now not the long-term also and the treated treatment with

counterparts. of from is reductions gears, Biogen in further SODX Phase are establish potentially approvals. In SPINRAZA the in which patients when see that to disease and this profile. support along path in regulatory study Based study, progression more safety the associated the on market healthy Phase study infants, with initiated new presented a data Switching the adding study by existing SPINRAZA Biogen patient patients with developing study This only in we benefit favorable like tofersen cohort protocol. most were with an Additionally, continue I/II data safety by highly enforced this of could data demonstrated III treatment achieving SODX-ALS ALS profile, has of NURTURE a months their to milestones initiated accelerated three measures protein, treatment. after presymptomatic positive to

Huntington's our to on Moving program.

a disease. in in England the its in by extension cause reminder, of this are As the huntingtin Data dosing excitement demonstrated and study week and medical community's I/II these from reductions change protocol is operation provide sustained simplify in change this study study greatly frequent dosing huntingtin study the the that mutant support We this of at first treatment of protein, date IONIS-HTTRx bimonthly disease. of to in of study for Roche's regimens. reductions produces less published demonstrate encouraged to desirable Phase with treat New Journal data to devastating III month Roche substantial the the performance maintained open-label the underscoring the with with the Medicine, data are to Nine for IONIS-HTTRx patients Huntington's reductions AAN believe the Results study. potential this patients known mutant the from disease a time from were for study IONIS-HTTRx Phase manageable less for presented how this dosing. patients more the Phase will III We as decision and long-term explore physicians. and and protein, from frequent

extension next on delay study from path slight comprehensive Roche burden even extend the the to forward data early a easily well. TEGSEDI Also year. new for and open-label And treated easing for this the expect as in These Phase were then look and Patients benefit TEGSEDI achieved the with provides efficacy portion or study both long-term presented will rapid treatment and patients program importantly, delay AAN, in a the hATTR went NEURO-TTR study safety study. introduces of TEGSEDI. this the established has polyneuropathy. in study clinical of NEURO-TTR neurological with be NEURO-TTR concerns entering patients on quality-of-life. We measures progression study blinded during Although completion. receive potential patients. who and that a in III initially, demonstrated by at sustained manageable to to placebo rapid benefit the OLE disease tolerability with additional demonstrated the identified time before the OLE this long-term to OLE, from sites data in accelerate year initiated no with this later in dosing the of don't the in continued study frequent Moreover, may less we the study patients data safety total, In visits,

estimate Phase people We year this towards including disease also study We're our next have Novartis of this epidemiology outcomes most prevalence patients the III the early has the LICA AKCEA-APO(a)-LRx world with advancing towards two advanced year. LP(a)-driven year. opening made, studies enrolling our LICA more for medicines treatment is to pleased program. with progress of precisely LP(a)-driven around of later advancing III Novartis, expected cardiovascular partner cardiovascular an disease. Phase millions worldwide the to AKCEA-APO(a)-LRx cardiovascular begin study of already is with With the patients

on Phase forms of polyneuropathy, soon including medicine, is up AKCEA-APO(a)-LRx people to all with commitment a year, TTR to program with cardiomyopathy. the in wrapping The also be TTR to We and further game-changing advancing And initiate Novartis' is due plan progress technology patients I/II our presenting of Phase and LICA Phase validation decision LICA Phase half this hereditary to large in this their the are by their this of to treat Importantly, study patient follow-on shortly. proceeding study amyloidosis in amyloidosis. our populations. after results license Akcea will of hereditary study and to we AKCEA-TTR-LRx well making patients excellent very III AKCEA-TTR-LRx, treat followed study from III and I/II second with wild-type TTR in

perform well, to XX In addition, continues year. of least Phase III next potential our mid-stage very in us up at studies by pipeline to the nicely have medicines setting end

study complete III. coming expect targeting In near-term Phase upcoming danvatirsen, antisense of catalysts. II to we Generation Looking our AstraZeneca step the to X.X therapy months, a

stated into they danvatirsen anticipate that pivotal recently advancing AstraZeneca studies.

reduced first risk medicine as strategy bleeding. this achieve the Factor increased year. selectively anti-thrombotic novel that IONIS' targets late the data last to looking enrollment Factor completed renal in year, XI around we a end-stage Additionally, II patients this XI of forward to the is we're Phase and with of pathway IONIS' Rx from of Rx study a the without middle clotting in study coagulation disease intrinsic

bleeding. IONIS Phase undergoing England to knee anti-thrombotic first activity agent reductions of and XI in for medicines As the total low-molecular-weight XXX antisense Journal the to risk targeting the in published be activity development Factor data XI XI replacement in patient without demonstrate robust populations to especially Rx in reminder, thrombosis, additional New patients We with Medicine, standard-of-care a is demonstrated bleeding. believe study superior to heparin used risk of Factor Factor in II risks at our anti-thrombotic approximately patients from the for potential broadly significant the our have

to also Factor follow-on midyear. sharing phase I the look the study forward IONIS We LICA from XI around Rx of data

another have in acromegaly year, to a program studies, in advance productivity to now, diseases. Bayer further first over back program. collaboration programs, more we into for these identify close Stan medicines generation the into look exploit antisense novel is of development of our intuitive later technology including this neurological our XXXX disease these and fully this clinical And example neurological program or the fatal call HPV We X.X Following Biogen moving This of from Biogen, a selected our plan for future. from the call. as to of to research to well as our medicines other forward both recently programs which broad Also, excellent the with established we portion additional even I'll collaboration. one development. turn LICA new under in target to identified this data And

Stanley Crooke

substantial I'm the that build continue to that and years, we far. Thanks, pipeline to our we XXth anniversary. our that celebrating many for coming far the revenue of shareholders platform. and advance, creation opportunities we and as we Brett. delivered done. value and way to expand value delivered to technology and the proud also continue patients and have growth, broaden Intent patients we we have confident advance of value that proud continue the to to year we mature for deliver to value our are have earnings looking exceed thus have are And shareholders This I'm

excited value more are of development in a of health on in with clotting are transformative are that medicines advancing particularly we potential issues. largest who We creating addition industry, Factor have bringing by rare we of our disease the patients for in excellent also examples disorders to in for Unlike addressing that advancing benefits pipeline substantial common substantial the pipelines leaders. AKCEA-APO(a)-LRx the and LP(a)-driven disease cardiovascular others, being one are medicines focused pipeline. more IONIS to diseases follow, XI-LRx common

to We have discovery. led lead and continue we advancing in RNA-targeted drug

the value each identifying by we organization that in led have we long-term to of the addressing than asset medicines continuing on more innovation treatment each to potential of XX lead profitable, we in our our diseases. technology. our and Because patients And focusing pipeline continue novel maintaining than are - optimal advancing maximum to novel have treating sustainably profitable new while revolutionize to creating and for molecular of and medicine as commercialize today, leading model, the targets We many two our the medicines launching pipeline, medicines, substantially of are precious it business advance our is. and

to innovation. that, like the aggregate, very In growing of for our We and the to clearly foundation are Q&A. advantage. continuing peers separate attributes I'd over committed these in are us our With to competitive from lead call industry the turn

set up, Gary, please? if could So us you

Operator

[Operator Instructions].

from question first comes The Needham Messer Company. Chad with &

Chad Messer

the posters One do learned sort hanging hypothesis or around a at would we beneficial to perhaps, are have people, that is just be SODX - idea guess the in disease. that it's thing patients SODX-driven reduction even not talking of and I AAN,

it's we saw by you future wondering are? in guys pursuit what Roche? that if And clinically on that Just possible whether the thoughts

Stanley Crooke

question, We evidence for benefit partners could agree patients in lowering ALS. suggests of Chad, who other lines the their would our that of response with to a forms notion. that in the SODX There have I Biogen. second And are of reserve bring variety

Chad Messer

Biogen, yes, Biogen.

Stanley Crooke

Did I say Biogen?

Elizabeth Hougen

Yes.

Brett Monia

Yes.

Chad Messer

I sorry. earlier, misspoke

Operator

Matteis next from Paul The comes question with Stifel.

Benjamin Burnett

Burnett on for Paul. Ben is This

quick ones. two Just

I guess guess, tafamidis? you one a LICA I Is you're there decision this planning? about and arm at a could you disclose, can to first have the made internally anything study on with this time comparator The TTR the that regards And cardiomyopathy program. include

Brett Monia

is This Brett. Ben. Sure,

looking but final call great, mentioned like with of do for for a or Pfizer tafamidis them against their that, survival it We're III nothing There We we we we're very improvements. to of and into determined. be of and as the pleased of for - - final is we'll proceeded. just regulators be. to Phase And there to would study tafamidis we're have touches I some second it is regulators. and it with sorts those hard to those As our tafamidis see we drug on follow-on in for gone half touches not the an I is up to the cardiac to with now, takes - of One the approved wrapping And the line. polyneuropathy. III component the on present approved, to in we just and We just designs or that putting data earnings this also think important needed the But our the Akcea And head-to-head with think cardiomyopathy, out - discussions year. putting mentioned LICA with Phase we're that that that in And not of be. second some our is know. There's very, what about set as endpoints, clinical then things cardiologists think have get how to it two they very is whether combination significant those important the a well. are when or drug. and study. what discussions not should or see designing will design think Phase Biotech has put things Very that's ongoing pleased a discussions commercialized. how bar Tafamidis the and balance. get that be finish takes needs patients successfully like that plays drug outcomes to very bringing and Whether you it sort touch

Stanley Crooke

to it So look Ben, be will that what won't before study like. we're some disclose with the long able too - position what

Benjamin Burnett

top could mentioned quick Okay. I on one guess this a a II you wondering Understood. And just was I up. success color would one coming it, have constitute I I That's just little know And acromegaly, readout don't what expected you guess line here? said color. year? guess you but if Phase provide this this. on is other great the I if you

Stanley Crooke

give drug. We're based there is, a And about question that form growth limitations. have answer too excited The acromegaly, hormone they on in And the substantial of we are to advantages - have significant our yes. is to growth efficacy to with to achieved. whether in compared examination drug used that the are while types, expectations agents of believe hormone going about hormone models a It and our second are be patients of us animal in study that initial the guidance program. growth could is both thorough agents. fairly side very the various effects existing LICA And designed

study. the it's are tolerability So of a LICA important expected. an we pristine, faster it rolled Blind than safety bit to - quite rest like It's our drugs.

So forward we're looking data to sharing you. with the

are having exciting forward results. We looking to

Operator

The from Mani comes next Leerink. question SVB Foroohar with

Rick Bienkowski

screening is, data of is for common the at today, all the is Mani. This on first My And had AAN. of genetic Rick in ALS dialing congrats updates I a couple questions how SODX-ALS. SODX-ALS a it approved? today? guess get once sense trying how patients is to are patients And how easy tofersen be I'm identified I will to identify difficult many of eligible patients? currently about with or

Stanley Crooke

for thanks turn all, the of and Frank. to first over question the kind I'll Well, comments,

Frank Bennett

Yes.

patients, medical the all are major the by patients almost in That's ALS. largely new there, CXorfXX mutations for screened with cause genetic variance a lot identified. that of ALS treat that centers ALS driven patients So being

been For for family those patients SODX-ALS, who generations. have in it's their

- this or brother the at many would it's for a parent guess the of with have now And I risk so I disease cousin going screening that is guess they're be quickly, genetically or families succumb data, pretty And ramped identified to up. to disease. a seen the so a

have would mutations a SODX I contributor so anticipate identified And ALS. that as there be are that patients will to more that

Stanley Crooke

identified currently number patients are that want on ALS? of SODX-driven you Do to comment the having as a

Frank Bennett

X% approximately of the all patients. patients have of So X% patients - SODX-ALS the ALS

to they that's patients and the in will increase U.S., that X,XXX similar in data do the the number identified So with Europe. probably, anticipate just we They and that are that close announced. number

Stanley Crooke

Frank. Thanks,

Rick Bienkowski

that That's the from or you helpful. clinical share ALS? any the treatments very are there helping way with and generally And just data have thinking speaking if of ALS. shape for about you're more the development the takeaways been us CXorfXX learnings about the to programs - Could tofersen

Frank Bennett

I on drug Yes. put SODX-ALS the really patients with into we target. it's the first what mean, that's to identified ever is be that,

what know the produced. the we protein causes toxic targets So the that's it disease. And drug - being directly

of that lot directly but to genetic the CXorfXX gives lot that good some as the movements clinical far that confidence in along then And so other clinical that successful, There have learning that a endpoints a the those as stage, of being we're another high toxic patients studies. causes clinical by probability ALS, incorporating the gene solid protein us valuable of the interpreting what and we the that patients variant we targeting study that in in we that for reducing still us weren't endpoints have - SODX a disease that of prove gives are that are for endpoints data. process clinical And and use eliminating the into some to confidence. we're the are that

So helping streamline that the for it's to program. CXorfXX

Stanley Crooke

for every from affected problems, by multiple great multiple agents think achieves value of about importance. by it's are our that that database other on learning just disease safety learn sequences entries manifestation the is multiple aggregate That we by that multiple different categories tremendously have for the having And having safety those publish we the in other medicines tolerability this just medicines study And broadly, and for of valuable. are time in that disease deal we learn as ALS, affected it's strategic I medicines more medicines. NASH the Alzheimer's, a thing since of the all and of integrated one we another that

that with specifically more learned behaviors and important similar particularly, as think is actually So us, really lot of we learned, but ALS, same strategy is the class. generically, has AOSs being comments paying of - dividends to for the a been of chemical

Operator

The next is David Stanley. Morgan question Lebowitz from with

David Lebowitz

many program I program? been has could of TEGSEDI? how it remind thus was you far the guess, was How the of I And us just how to size if curious - on drug? commercial the transitioned for big much EAP

Stanley Crooke

Brett?

Brett Monia

that of sales written a patients many a yesterday. we the on between those not disclosed them, has EAP, Akcea mix as new very the The the disclosed. presented EU pleased the But details we are been have still the right in nor being they David. as OLE can how on United - of the scripts Sure. as patients patients as commercial nice the that balance well. well. States is details the --and EAP now say about of well What from numbers scripts those and of and balance constitute lot haven't we're

Stanley Crooke

come new previously factor naive say and the of we're opposed patients watching of have what is exposed that of with what those simply, our and To to fraction that those it Yes. seeing. and number to been we're from as we're that prescriptions key TEGSEDI, pleased

Operator

with is Securities. Wells Jim Birchenough Fargo next The question from

Yanan Zhu

Jim. dialing is This in Yanan for

due patients talk not And of also, to extent treating I think, first be in for data. kind your SPINRAZA So prevalence? of talk you question the value about driver presenter control about example, experience some terms centers is due on SPINRAZA Could that patients may adult of it's high you SGCT, the patients. in mentioned adult that adult efficacy, at situation? in maybe to for because or a Could doctor one

Stanley Crooke

similar benefit Of with as trial. SPINRAZA to course, have I - infants the patient what from child know produce randomized the and appears SMA. And refer drive in placebo-controlled to we as we the to is and in the character responses not you of what the answer second is, learned in would for quality the does of Biogen adult adolescent question. But

Yanan Zhu

TEGSEDI. Then on Got it.

patient that For the commented also additional rate in on diagnosis increasing that? switch? a you your the you a to more medicines. elaborate little patients from that have Could preserved And majority you undiagnosed? drives switching example, patients effort given wondering, on bit vast perhaps, of a characterize what just So bit little competing remain

Elizabeth Hougen

Beth. it's Yanan,

being these our patients So U.S. on so is an subcu the marketplace. and through I EU the once question our realized think independent Akcea's regarding believe we're conversion. provides allows patients perspective, seeing, products. the weekly in the benefit that and that the important And both and that, in from converting perspective it administration, competitive in both we What from TEGSEDI benefit

the our So that's conversions. view on

Stanley Crooke

that being I And issues that certainly the one the think information their that's real perhaps comment limited developing patients that great I IV agents and of with factors are issues in efficacy with think the are drivers. thing know of definitively used there's probably there enough converting. who are of a and more don't deal are driving conversions. on to in sort infusion But these we

Operator

The next question comes from Ritu Baral with Cowen.

Ritu Baral

wanted ask TEGSEDI another about you I to launch. specific made comment you the during

the that the using well continuing is seeing? of plans? insurance the order payer that of prescribing. prescribing how hematologists And of neurologists sort with conversations rank physicians and and that distribution coverage as You you're being Is you're mix with as all mentioned payers cardio cardiologists, of - that

Stanley Crooke

for answer to again the Well, we're in not the are of encourage can detail, specialties And specialties numbers the to that's physicians question we pleased change TEGSEDI. the talk are that of going question? don't of you want can to time. is say and you going prescribing various a number handle are because what say Akcea colleagues all I with to kinds more would ratio - we involved we our What at at the of exclusively - that that I of that

Elizabeth Hougen

of - terms In absolutely.

payers - top in In got the contract in - you terms with the of under payers country. with - we've XX we're

got of the lives payers. relationships vast under covered have majority all with the We've We contract. of

as there acceptance. commercial any And a so TEGSEDI's we're issues seeing not detriment no having of We're reimbursement to whatsoever.

relationships So with with we're very pleased our payers.

Ritu Baral

conversations prior value-based finding plans? outcomes having any or you with agreements other that on you're about certain or or Are authorization separate conversations

Elizabeth Hougen

our enter to just been We're agreements in value-based interested that. happy but more they conversations, with than into in payers, haven't

with patient are Our for their not population view meaningful. just agreements that that amyloidosis polyneuropathy, TTR is value-based the - for

not open requested to rest. with we're we're all TEGSEDI said, while idea, XX the And that seeing and open so the the to to by continue top we've covered be payers. as idea from And at top relationships and being got I the the least -

Stanley Crooke

definitive would Which, at you answers. colleagues for our again, refer more Akcea to I

Operator

from The Fitzgerald. Eli comes next Merle with Cantor question

Eliana Merle

the at updates AAN. on Congrats all

the I the a program. the like, if mg in about, it - like question So plans protein. I another XX% XXX poster on what's At you're guess not correctly, And dose SODX to have reduction? to, try the increase the the I forward? guess, dose guess I mean, the reduction escalate SODX plans dose And of you of to pose reduction SODX SODX? looks further do - just just I'm your seeing the going the why question, like, looking maximize at level

Brett Monia

Yes.

in So that is SODX it's fair measurement the CSF, SODX recognize CNS in tissues. to not

spinal case underestimates of and And ALS. cortex being motor in in so reduced SODX that's the in it the CSF now cord

more a And produce clinical I benefit dose that, was models, was to so identified adequate than we've that that say. should preclinical in

we're so happy doses we've the exploring for studies. very and And anticipate that don't with I as doses high identified, the this

Stanley Crooke

initial important study more I observed the profound was think evidence how than is benefit of months that in done. we've the even in that three that work

to in it. So is always sort challenge effective We're got preclude of we with diseases we've this but with a that use continue and we dose, going a very optimizing that doses schedules rare pleased dose dose. other gotten overtime we've and I very study, to and responses think we're the wouldn't

Eliana Merle

program us guess, we very have the think just That's updates further on through progress you it - think guys guess, should in I Got we about on late scheduled you June. helpful. I at know as at you're the expect what about see in like presentation? a then, that meeting the year? of could we it. presentation looks Huntington's I to might And terms we Huntington's, should what help how

Brett Monia

Yes.

patient meeting So the is Huntington's in a meeting. June

open-label provide the study, extension. next III be a updates of that's to Phase study, early they'll the no they're there meetings expect presence will course, update or latter where a but in year would then part the on And for And presenting I next a of the medical blinded so looking update. the so scientific on that. year not the to have We're patients,

Stanley Crooke

again, Roche. once refer you I'll And to

Brett Monia

Yes.

Operator

Jessica from Fye JPMorgan. with question next The

Jessica Fye

first just the IGF-I And does one acromegaly what's growth up translate frequency the on then a that to dosing receptor? target there? depot reduction? Is the LICA the hormone What's of comparable just SST it Maybe following And product. how to injections? reduction

Stanley Crooke

of of Well, monthly the it's LICA family drugs, dosing we And go all we be have LICA - for like no, our using to be simple it. all of the it's patient do a of for for drugs. would to it dosing to thought like very for - quarterly weekly better it us we're rest would if indicated like the dosing, as

hormone binding we course, Of direct between directly almost showed we guess and receptor there questions and - relationship of receptor of that reduction you to that growth protein in to blood. don't correlation reducing animals, in we and later have growth growth And course, expect and question in we're - measure we're that - is which hormone lots was that what an I patients that's the others yet of that primary is the know in asking answers we this year. going one preliminary study, with

Jessica Fye

so, seems what you're indicators to the number share based share when you're Akcea? like signs was reported better the And overtime, if if just based curious, feedback pointing then And there? on about on can be about bit from maybe it leading Okay. sales. seeing we TEGSEDI like quarter, getting you think the I'm a XX% little for might about talk

Stanley Crooke

think behind. launch our are remember about we we competitor, that versus Yes. of quarter we not Again, the as TEGSEDI a

was year. this the in of compare And first $X we that reported that million million so quarter $XX to the

And so we market in with pleased that share that very sense, the we're have.

I share still it's year too others. the market that have far think to will TEGSEDI draw conclusions numbers versus let early tell firm would about I what the as progresses.

Jessica Fye

one. maybe just Great. And Okay. the last

think highlighting all treat of SMA. with to with kids that parents guess they I benefit can their maybe while Novartis that was to still Zolgensma the and want call I on acknowledging durability do

any when starting disruption starts launches? - of, if can SPINRAZA any product like, SPINRAZA curious lag patients, in that you they kind in expect go or in type therapy? I'm I Just on any So knew gene on

Stanley Crooke

think hard We Novartis, is encouraging other I about the on I this any - Roche the do - way sorry. think are drug, presented know it's have and established, treatment. thousands of or and I data data drug patients that database not benefit But great that to that with SPINRAZA an treat, patients And in to essentially to and know proven older years. well. of the mature like for children, we SPINRAZA. patient pristine treated - on X,XXX We SMA patients. know safety is are and about treat many work absolutely before but work proven also There patients treatment. symptom X really years more than have extraordinarily that of be develop normal healthy the onset healthy we we the we if better we of infants most that longer finally,

effects as my less a another So of on durable unknown studied I there with had efficacy I parent, SPINRAZA. SMA, and baby use of agent a baby side way is diagnosed with would if no place levels in

more but So parents we'll any close. can't to One way. I many that then feel and myself, for imagine question, other speaking

Operator

question that I-Eh & Laidlaw with with Company. comes Jen And

I-Eh Jen

on progress. Congrats the

provided tofersen that clinical rating a scale on some data data function that Biogen AAN the follow-up at long including release Just there improvement. and

or on So would that bit details would data the little and ultimate to the of you comment benefits mind potentially you that what more how more a translate patients? see

Brett Monia

Yes.

decline generally, in about is really determining study So whether progressing the patients not there the And patients' form the well ALS. is in months, powered rapidly the It's small to in the is and the as essentially ferret they disease stabilize in X.X-point we in scale declines general these population. disease of study patients. enough in no it three the that most is drop, of effect. in what This more out populations, three saw decline, for faster. months. per drop an the was There is and what for that's untreated remarkable companies use ALS they're it slopes trying And FRS they which stabilization patient in observed just think disease of ALS the disease. rating declines stabilized X.X-point over progressing determine are is outcome. the to And improvement show which general rapidly over the changes we standard even well to all the first or for X what Generally, these month truly these in there's patients. And to population, impressively, ALS I

things posters XX they a swing the kind that's one yes, crowded more exciting in are the there the happened of there but at fewer participants the XX, last years. meeting, that's the in through ALS of at So the

Stanley Crooke

on comment but the I too scale, it like it's And preliminary pretty improvements the at components rating least think various are across-the-board. occurring of early little the first blush, looks at much a to

I-Eh Jen

get the least you And the people trend do curious, if trends, do to the or continues anticipate at maintain? short-term or current drug approved or anticipate just

Brett Monia

Yes.

Stanley Crooke

we're first emphasizing doing. in performance. work Well, everyone to thanks that for your interest close by the want I and attention financial our

in Our business the of financial the business. were in Board results single context model is impressive across very impressive first element our working aggressively and and very quarter of the every spending

think have puts a expand great that and And invest we to in. and strong us so we in things to uniquely invest continue position to

technology and way truly generating medicines efficient that's have an medicines We in extraordinarily that are transformational.

forward year about and coming it talking the And we'll telling so look about be of months. Thanks. it. this beginning exciting is another you in Ionis extraordinarily for We indeed the to

Operator

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

may disconnect. You now

Ionis Pharmaceuticals (IONS) 9 May 192019 Q1 Earnings call transcript