IONS - Earnings call transcript Q1 2022 | Ionis Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to the Ionis Pharmaceuticals First Quarter 2022 Financial Results Conference Call.

As a reminder, this call is being recorded. At this time, I would like to turn the call over to Ms. Julie Tepper, Investor Relations, to lead off the call. Please begin, ma'am.

Unidentified Company Representative

Chuck. you, Thank

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

Officer. of me to President call this Onaiza have Financial accompany Product contains and With We Chief posted X, today's Executive And I Chief our Q&A slides Eric Officer; Officer. Swayze, to us Operations which joining statements. Schneider, Chief Chief the drive your are that would website of portion Slide our morning like Officer; also on attention Eugene Clinical Research; Executive Hougen, Vice forward-looking Cadoret, Monia, Development for Brett call. Beth the and are Strategy and

will and additional and I uncertainties, With beliefs. to actual this subject risk that, you risks These over certain be and in the During may the SEC contained differ to our results making turn factors statements expectations consult that we language are and for on current call forward-looking encourage call, are our based filings materially. statements I'll our details. to Brett.

Brett Monia

strong today's morning, Thanks, very for year, This call. on are and we Julie. off us everyone, a start. Good joining already to thanks

in patients bold year, olezarsen and our our week, a great us in with increasing this enrollment NEURO-TTRansform The excellent we're making original enrollment go-to-market advancing ATTR we're TTR donidalorsen. current in on step dynamic Based III the the in study took the of our polyneuropathy our we study shifting making the to data achieved to preparations. data data on to in progress in progress the our preparing new positive organization, hand trying expect estimated half technology on cardiomyopathy. abundance these expect eplontersen of XXXX file the of compete AstraZeneca, market robust with rate, at last Phase of commercial midyear to moving with This by data. doing that our opportunities with positioning grow announced continue We an our we our building study. and $XX we're also risk in includes to recently end reading inotersen, data same successfully for XXXX. late better line regulatory to of for time, of working well near-term market. size our and assuming delivering with updates by At CARDIO-TTRansform even hand And and in time the modest we shift commercial advancing approval readout we patients track to towards excess expanding duration so, a generate We to with And billion. first more medicines goal for to from remains

triglycerides. for blockbuster The therapies with high with expected the patients patients We which study are study remains for familial hypertriglyceridemia, a syndrome FCS, we with core olezarsen first-mover cargomycrodemia BALANCE continues In severe data with in year. XXXX. million severe for to all sHTG believe or standard to a and our in also track our patients III in With risk is current in represents opportunity elevating advance factor X Severely program of next Phase advantage, continued cardiovascular sarso independent U.S. over key Ionis. progress triglycerides the hypertriglycemia ineffective. care patients of with on data

dollar best-in-class has for with with progress in treatment need well be We to with data believe started OASIS-HAE Phase a this donidalorsen a for Our in XXXX. angioetemia of $X expected potential patients. donidalorsen patients continued II hereditary these patients donors also us unmet billion significant to HAE market. in as medical growing significant result, given represents And plus the opportunity prophylactic the

excellent across our made pipeline. rich We mid-stage progress also

data PCSKX ACC positive good presented for secondary study hypercholesterolemia. ITG cardiovascular IIb from in show primary patients managed April risk Phase in are IONXXX, of medicine disease high study the The in AstraZeneca endpoints statin at potential safety with more and who With in best-in-class million remain our for goal tolerability and U.S. opportunity despite IONXXX therapy, demonstrated their believe the a who significant IONXXX we us. patients could XX maximum profile. than and LDL-C ezetimibe and above be a

failure These over treatment-resistant ejection with therapies, study II represent Phase effective remains study million failure. of in reached to and XX IIb with full advances the fraction. data for patients alone. heart year. indications chronic U.S. the need in this hypertension in more address in We're we also treatment expected reduced combined second half IONIS-TTRRx resistant Phase heart in significant patients Despite IONIS-AGT-LRx with with treatments patients of in enrollment acretension a Additionally, the evaluating the in

Looking the by planned ahead, filing and year-end. expect additional we catalysts by regulatory III out Monteria of highlighted midyear our read numerous Phase

before several We first updates call important and to review to turn X advancements. on study Phase also Beth more technology expect And quarter over with I'll our the IIb key data readout that, initiations I'll Eugene preview rest prepared results. wrap key and of Eugene, catalysts financial your our the pipeline And the year. After upcoming updates recent remarks taking up questions. our through will discuss

Beth. to over Now

Elizabeth Hougen

Thank you, Brett.

and our strength. consistently financial quarter our demonstrate is to key diverse of ability financial results revenue first sources. substantial That Our from clearly numerous generate element cash a

R&D between split Our more revenues and more than commercial approximately than increased to XX-XX year-over-year XX% were and revenues. million $XXX

Our than We marketed SPINRAZA. were on investments And keep healthy more X% a including sheet, in increasing last meet ended March expectations. quarter majority with our were the from loss, balance in $XX non-GAAP to sales $XXX we earned compared in on SPINRAZA's million the financial of coming our guidance. cash million, operating with quarter. our XXXX net revenue products basis first from These billion. global and with a track results both to line $X.X expenses and us

result, a earned in million royalty As $XX we records.

show quickly in quarter a last by program. million, year. from was our highest programs years, with $XX we we products. new achieved the partners Based increased updates same on In our in with a discontinuations was these revenue patient in -- MASA RESPONSE prior revenues in revenue for SPINRAZA see for strategic time profile, the China. royalty bright the last decrease. increase previously Outside earned first expect results study doubled U.S., the the starts reached reminder, It that contributor. presymptomatic receiving Biogen more attractive and initial sustained the earned the treated the quarter SPINRAZA to which patients treatment a beginning continued X-year patients Biogen study, for from advancing the recently year U.S., NURTURE while presented revenue maintain $XX to R&D the midyear. Biogen million And We as uptake collaboration ASCEND in SPINRAZA's quarter in We U.S. from also to neurological new year. early ex the tiers normal earned continues year. in driven strong and largest XX revenue of and the SPINRAZA motor milestones of reset from U.S. advancing continue than and quarter move first reach compared results R&D to the and this presented numerous which as both continue high royalty future consistent development. Just through competitive from We to for the with and to SPINRAZA. at several compared and rate different each fourth

from for Our expenses expenses of by large the R&D costs. aplentersen's $XX included more part Phase increase than year. last payment are first their the non-GAAP currently We AstraZeneca R&D cost XX% III $XXX conducting. which reported X% studies same increased by of payments also in revenue XX%, was period million a quarter we X sharing compared million, development with in driven operating

Our making also eplontersen, XX%. and decreased savings support year-over-year the opportunity. about Sobi the CMC and included part for spending near-term in from preparation for commercial are in largely investments our due transaction. SG&A the our activities were by was R&D substantial losartan we savings medical This go-to-market integration expenses Akcea and by to These expenses affairs donidalorsen. increased offset to

line in forward, to expect compared operating we expenses between similar and year. this and anticipate in we Phase over QX progress. we be continue studies last We increase to revenues III last in to SG&A more R&D to as expenses be to investment QX our guidance, increase this with We expenses the project year the the preparing will in XX% even while weighted our year, our our revenues to year. cash of needle into with be project diverse revenue $X.X also to market. increase our for to second end to year towards investments March, the substantial we the in ore course with of combined our of ability sources. olezarsen and at end QX, XX% billion and half expect Looking generate with back the to Consistent many bring

are financial that, turn drive significant we We have investments And to making to I'll underwrite over growth. to the call with the future strength the Eugene.

Eugene Schneider

during on you, quarter. pipeline report the Thank Beth. progress I'm first we to the made pleased continued

of progressing Brett approval are second to data NEURO-TTRansform for assuming from study regulatory III said, an with midyear. positive Not preparing And Phase Phase year, terse we're this is the Our data. the as well. balance a expected half III programs file at in

from study. characteristics transform baseline III our up the CARDIO-TTRansplant Society peripheral that study of we this achieved at interested we neuro enrollment presenting derm announced month. to forward looking we're week, Last goal, Congress ending Additionally, the Phase later original

dynamic of the an to more to study, ensure amendment population because designed in timing We the patient with with physicians administered increasing evidence patients By a largest duration to from and rate. aim in enrollment is also and decisions. is outcome alone The payers This clinical who The the to included study approximately or benefit changes ATTR and the and announced stabilizing. extending robust our weeks occurring this when broad in these positive to enrollment to an cardiomyocyte is now to period growing study our compete very study right is most patients make in combination a dosing was blinded data high to and XXX. the at CARDIO-TTRansform The from successfully generate implement generate of should highly Uponor's patients amendment even study. enable important expanding XXX to a size important XXX XXX market.

We out data nearing into shift of moving demographic first the our on projecting from outlined, first data, extension. have our to baseline we're with clinical label just together study a modest enrollment, half XXXX of time the rate XXXX. of line with to I Based and the read and current open patients our that accumulated on substantial amount late entry are updates

development. We program program the development the leading and market moving olezarsen hypertriglyceridemia. broad of targeting Our medicine, potential olezarsen in currently X advance clinical development designed the to remains realize indications, this and the also in fully FCS program towards APOCIII to including severe it continues

X up remote and on the also dose. our remains we study, II And in We is Phase to demonstrated we dose. planned result progressing robust assessing core ongoing to in the III XX-milligram which SHTG We're track studies, for addition monthly is monthly evaluating Phase for XX-milligram gaining Phase the study XXXX. reductions. have Phase even year The expect BALANCE APOCX In studies and dose, greater X also donidalorsen triglyceride which data and II, FCS with in data on with reductions triglycerides Phase III reported year, next from donidalorsen study additional OASIS-HAE later on and track year. the monthly HAE. Soperton bimonthly to in report and data II ongoing the extension study for quality and with patients the plan June, who data we demonstrating new Biogen II in and patients life present deals integrated Phase from SADAL with X clinically in In XXXX. treated from groups. data we were for plans of study, this the data in Ballard out open-label Coming dose improvements to year, meaningful from ongoing this including sustained data Earlier to

focus. engaged to forward potential remains identify Additionally, regulators path with Biogen

a have to to also expect mid-stage we pipeline deliver. that We rich continue

patients levels The with results secondary pleased LDL and ligaments disease. on and of give the patients demonstrated by the study some confidence to or are PCSKX Phase us and cholesterol. study the The up hypercholesterolemia the reductions of cardiovascular in IIb disease, of data primary for without high the for IONXXX dose the study affected risk positive recent We're from PCSKX adventive IONXXX endpoints. Here could for met highlights. adhesion change XX% current IONXXX up mean that cholesterol XX%. levels AESO by positive standard care to by cardiovascular its

Solana We believe confirm develop and to the based IIb on III dose is results. Phase decision line AstraZeneca will the Solana further this of a that adhesion IONXXX the to potential Additionally, study ongoing. study Phase year later make

of medicine with track chronic in our We're to Phase enrollment treatment-resistant IIb also read reduced advancing with half patients a expect treat IONIS-AGT-LRx which study We out ejection completed patients for this on keeping year. Phase in year. IONIS-AGT-LRx second data heart in recently of next II with in failure we us also fraction, to hypertension our study the

to in addition LICA mentioned, we and data Phase IIb In medicine esomarsen expect hepatitis to more of from readouts the our XI the here, with B study in data I Factor several renal. Phase IIb from study end-stage programs GSK, already including report drug our report to Bayer to patients data or

a of summarized, we and have just number programs important mid- As late-stage I've progress.

that, steady over with I'll to the close to forward looking the portion cadence of And Brett of to the unfolds, As data readouts. the back call. turn we're call year a

Brett Monia

an We're Eugene. year. to excellent start this off Thanks,

to the the our year. drive of forward organization third advancements this launch late well in out for medicines. delivering on And I SPINRAZA Building commercial making an as pipeline, our further and announced planned rapidly medicine Ionis this technology, made continue building toward the strategic the strategic new as year on other term, substantial olezarsen near medicines follow-on sharing the with great our progress we patients to We're build and market our abundance next progress on our advancing for tofersen, our commercial we in including believe term growth important execute including commercial continuing in of donidalorsen these year advancing priority longer TTR these diversifying with eplontersen to near-term starting that on year. technology substantial earlier also making as We that advantages. later the of early details priorities X and last opportunities up and important Look our polyneuropathy Ionis. to as X expanding market for will

X we're we our capitalized open with productive XXXX resources mid-and to pipeline need all the data highly expected before priorities. to the remainder And readouts key looks Additionally, with and year-end, questions. now executing of the up to well Operator? on be up that, our eventful. continue call from for late-stage With I'll

Operator

will Wells And Instructions]. from question first [Operator the come Yanan with Zhu Fargo.

Yanan Zhu

quarter. the in progress the on Congrats

context the to Putting bit cardiovascular think study. the you tafamidis more I protocol severity, more also that. use I monitoring have hope a probably and for are -- a could all bit little overall patient elaborate little you of disease probably us, So CARDIO-TTRansform mix event you on in been looking change the patient things into at the rate. And terms

companies? how have how from So observation of expand X-minute during lastly, that that to some have decision with the what lead seen the at does we looked those the also monitoring curious, the that And other just compare and have been you also And data? data study? to walk the does

Brett Monia

I appreciate ask question. on expand And it. Eugene to and for then it. it Thanks, the Yanan, I'll address

some for we've time been So now. saying, quite as

that would in firearms doing looking we're New York recon a in was study relate monitoring as and study make for we and this outcome along. study. growingly patients X, accumulating for time I'll primary big We're this at wild were the half in that But to has decision. been readout along, X-minute really balance are possible looking and that did. thing have something in Sure. This adjustments to the want of the naive We're on study. go, Demographics in events. about really we trigger for not trial. that those that second study. a the on the severity Eugene the X, time. example, early How about as anything this the projecting demographics last ensures successful driven demographics offer this the study events patients. usage might in early things This rate. the Health ask power something Association say example. we perfect to, in on on to our in much is the we're start if do at. that's and onset in really hereditary a it's company. right planning potentially making you walk relatively study. the study It's ensure test type market we population Class before all the And we patients, are, sick the like in something we And patients fact have possibly market. on that no study It's We're really the the of of how enrollment do between cardiovascular get that then this X, to that versus in -- expand We at Those less really is highly successful the looking patient the There and is exactly that with study We're tafamidis this successful to pulling right is that. we all competitive all by sure that were the do were have is to the what the event And that --

expense to able impact We really timing. It's having positioned on a the are a delay small. decision that to significant study significant and are modest a well the -- and make the investments without be

the us fast. significant. that important, strong. very very measure allowing is outcome this a upside You going very that up very, provides very to very have positive We're against and is Enrollment

reasons. good There's this to like time point So adjustment all and but that? do making for add the is this is the right this Eugene, an to do anything time to this no waiting to it's study. it,

Eugene Schneider

at look maximizes ability gives at which to characterized. going also set within the patterns was be I this data the study, informing us really important stakeholders for study the would when our change well practice that reads for to a and also subgroups only providing also meaningful critical really, No, is -- out.

Yanan Zhu

That's super are -- the midyear about on you -- time what data the database, to that? potentially analysis polyneuropathy in any what on and helpful. locking may of additional could see I color in, And what -- share stage be data? able around can see data you you would If ask aplonterson we readout, all currently the any terms color

Brett Monia

Yes.

achieve. launch this study. and III What we're Phase So to midyear We've as to not saying line that we're as we've launch. study providing filing, been in we're some we're add ask with track And the also in remarks. And the on data top expecting color that. we're that preparing for completion, coming our prepared for Enterphoneuropathy from to doing much -- been to the regulatory the Yanan, saying, could on right really than preparations I parallel with the partner, we more is -- I on through year, Onaiza can our maybe filing color provide for said the maybe AstraZeneca. And we're planning preparing the

Onaiza Cadoret

strengths. the teams is marketing a preparation across clinical actually had the are and and in teams launch obviously, to had overall we and medical great week to each building effort a teams of CMC lot preparedness. the launch takes pretty just collaboration other's in really team shape. it say that Yanan, a think They everybody the in It's the a really kind their through lot from meeting of out with customer-facing well last affairs of to Coast, East sure. on AstraZeneca members, pleased I'm launch as and offices Yes, readiness robust as

a really and heaven, is of market with amyloidosis say. in reach complementary decade in and along expertise made broad Our collaboration knowledge great disease the kind of the I rare would for about being and

for as to launched the And I by say, year So all would ahead and product the of shape. file the we're good expecting in year, well end next getting really everything goes well, assuming this curve we're of well.

ready are teams go. to So

Operator

Yaron next come Werber Cowen. The with will question from

Brendan Smith

for to on the Congrats Yaron. guys. This quarter, is Brendan

ones couple quick us. of from a Just

of the the First, in there what in really, you in II and And data look you terms guess, as patients number III, with you of Phase what kind for of I confidence remind human hypertension. to us Or we potential just can ahead in really going looking that of would same to is resistant are engagement terms that program, a Can maybe the to looking maybe expect? threshold? readout move Phase there efficacy give forward you're a target there drug? what is like like

Brett Monia

Eugene, that? would you take to like

Eugene Schneider

Yes. Sure.

type So II but consistent current the patients study treatment-resistant patients. a we're study, hypertension. We're what demonstrated, are is effects course, to -- smaller study of of for with greater see of Phase larger It's again, data which the what in with The helping significant course, hoping XX-millimeter XXX has to in range XX in order sized this appropriately changes. with is approximately variability, with the exploration. to in pilot see, XXX earlier mercury data basically of

you point. there is this really, I at all -- So tell that's could

Brett Monia

I if just bit. can And expand a on Thanks, little that Eugene.

a Just comprehensive right for program ongoing pretty have we Brendan, now. AGT reminder, a

hypertension, In we start in new very out read this our coming with then lead our we're in addition AGT readout year. drug refractory IIb for to year to now drug the ongoing with Phase study a and Phase that up I we're planning to successfully failure. II. heart And molecule, completed that patients that looking molecule is next X.X study Gen an Phase behind And have for later

year, And course. make the to is III. already look with target I to our we're it. we to can molecule, of we heart be the next It's best to that. haven't we'll of the cause issues. safety said, We the Phase And II in which I in on expect we failure, the is the efficacy the confirmation safety, starting next we need sometime but hypertension Eugene we refractory concept that of inhibition on in lead achieved mean, kidney, on in what's bigger Phase hypertension, this step at good molecule a what's see kidney also for when to safety, But say we the for of patient plan a look of deliver, it need refractory safety, what fact And population. looking aware all pathway, molecule, Phase decision at the call we will the and follow-on the data, to and what see is potentially kidney in LICA, -- the don't a for never study. And III. seen once very pathway we step And and make all not next confirm the RAS study, proof

Operator

will question Luca next The from RBC. come with Issi

Luca Issi

progress Congrats on the here.

Palo endpoint you maybe be I get EU So the knockdown. XX-month actually impressive and I similar weigh point is think the filing your that on the to And in to primary the will pretty for has at EU? X then the maybe PCSKX, happened what months. in to to U.S. in I sufficient need Will think data both Alnylam? file Or

ALT did for discontinued experience the I at high see them, elevations. we Two some However, patients think, of dose the drug.

Ene update signal. I'm any how So pulmonary the of aware then that inclisiran, thinking post wondering least in the you're context of at finally, about not And the discontinuation? on franchise that

Brett Monia

Luca, squeezed lot best a all addressing questions. think, in I you've our Happy do those in there.

on pleased And the lowering -- date. lowering for stand enthusiastic for being despite PCSKX drugs presented come readout risk to effective PCSKX such for has bowering drug despite So inclisiran. patients and on cardiovascular more the out hydroclesterolemia at an ACC. that's our This and continued potent, yes, IONXXX, very very ezetimibe, despite data enormous we're for being need been efficacious any drug. drug excited that's that of to in why monoclonal There's this be LDL molecule on ELC by disease as high looks program. about antibodies being AstraZeneca so is most to PCSKX with

from for IIb a reductions with over greater there hypercholesterol. achieved signal millions range game we could of the of And XX% dose-ranging contingent XX% LTE. it doses and study real no than the in Phase were a study. on So where at people changer The suffering be well LDL-C

couple things dosing a So with study, it dose elevations. mild of they're that patients dose mg saw, X ALT XX worth baseline in XX-milligram I to noting, towards was one the continue A where mild; we think, that baseline. two, and monthly patients and is

very is of range because important, or further conducting their in observation the the patients study and so Phase Solana, this called II LDL ALT that's that they're net It's The preparing there's more Phase a and that XX of So dose. are number that be going And III but small is in toxicity. and good, confirm is what doses Phase in targets range III dose, to a our looking there. Phase an we're achieving III age very ongoing for study to development. at

the good, I to the I their road. at haven't for agents. Patients very lowering already in effective are and there's any need seen LDLs, we bumps So not very said, looks respect up drug safe, what eclisiran, more so With comment prefer not to on competition a getting to target.

pulmonary a this patient want you about Eric. to little And so needed bit, community. a drug like talk in Do is IONXXX

Eric Swayze

Sure.

at in we're reduce lots the data we've compounds potential of issues what to where across species think in hard tinker of there's seen backbone relevant baseline try And potential really find inflammatory on hope working some for of talked effects. we're data of effects the are get to the about us our and programs thinking the some the and in good clinical multiple we've unmet with with these potentially some diseases moves doing them of medical pulmonary future chemistries need. we is allow can molecule, And or the molecules that chemical some up pulmonary that note saw within the will So and inflammatory class. become picking look of of therapy chemistries new properties some that of some the primates here both of also new advance applications lots into the not-too-distant and to we some

Brett Monia

I progress side. solid pulmonary making to said. went I that -- very on answer kind encouraged we're development apologize be on areas about eros Eric of reactivating We're the programs that encouraged. of didn't And We're question going you in your the the the out order.

study. So XX approval. set higher out we're data -- that the what would the But the haven't see focused the And are ruled now data, this regulators NDA. they remember, eplontersen. mentioned we're open. U.S. European full we planning is set signaled year a have should full filing, our NEURO-TTRansform they right said, point that the to out to With XX-month that months completely I that for on data But set. have ex-U.S. bar on highlighting fact NDA you you're keeping we're ex that file option like in and

Operator

The from next with will JPMorgan. Jessica question come Fye

Jessica Fye

I up changes prior question to wanted a CARDIO-TTRansform. follow about the on to

you focus and/or being regions exclusion treatment size you'll changing and increasing about recruitment on driven Are just any the to the or patient demographics. sample by the addition duration? for criteria in them You talked further of inclusion

Eric Swayze

questions, Jessica. your for Thanks

now keeping the eligibility So criteria we're same.

are, We again, enabled with is, the sort importantly, fairly we and landscape. into patients. of landscape of, current make important number some effect us of one, intention subgroups full the ATTR, population sort said, enroll with of of patients ensuring the looking however, specific But to for inferences drug Brett of that geographies representative as also

Brett Monia

And to point. that prioritizing could geographies and second territories and certain that could involve -- involve your Jess,

Jessica Fye

Okay.

guess... I So

Brett Monia

say should will involve. it I

Jessica Fye

not Pamentis. tafamidis not you've think a I in your on about Because maybe even patients the are XX-XX between those split limiting who background past, and you're are for talked Yes. though background expectations who

will expectation So that is that be mix? that, the still your

Brett Monia

to what for perfect, want be Jess, versus a well versus balanced. would we're defamitous usage. is we peace we've the looking well-balanced naive With said, very be XX-XX but

Jessica Fye

recommendation CARDIO-TTRansform one they And by interim input? trial been changes -- from DSMB analysis on an these I prompted this Were guess last at this point? made the this DSMB were topic. there has without or in a And Okay. to

Brett Monia

interim time, safety at still internal we analysis do been in involvement have analysis this even or no has with this entirely at have was This we interim done call. said option conduct to Ionis. stage. patients as AstraZeneca, the analysis. This There's forward, the by no appropriate an nothing driven an the committee to but We earlier oversight I after the put

this And option option. to it. time-wise wise we're part now the and but potentially an that position it's enrollment and today. So to it, always in necessarily This do we're that excellent we be plan not able to of do to was triggering trigger have,

decision, entirely we're that. this the in and AstraZeneca, driven working and engaged was hand No, we hand them course, Ionis. by support fully with Of on they implementing

Operator

Paul from Stifel. come will question Matteis with next The

Alexander Thompson

for This Paul. is Alex on

Just X question. X question and eplontersen neuro planeropathy then

their polyneuropathy or Can your at And there have cardiac on programs thought a readout is given readout? neuro you exploratory announced imaging Biogen? rights that us, subgroup to planters. for reacquiring biomarker any the then within any to And if so, with collaboration either data have you that the Biogen So neuro, full pipeline prespecified you at all study? expect this remind do prioritization, in about

Brett Monia

in the You one, first cardio take the subgroup neuro.

Eric Swayze

Yes. the for question. Thanks

an at subgroup population look in TRANSFORM cases. be the study cardiac to subgroup will a CARDIO-TTRansform. you, of phenotype same again, in largely These So the yes, and They're and heart failure. remind exactly look EU symptomatic having specific It's cardiac it's there with not patients patients to have predefined enteropathy analysis just polyneuropathy -- don't study. some mixed evidence

think that's But down distinction. I the important line. an important So will an there subgroup yes, be

Brett Monia

Yes.

back. step a take just me let -- it's Now

relationship are at like with that drugs priority. follow-on -- of become them. we're and started become be relationships years has page will else The the working they're with to that. with Biogen be partnership management over The partnership. out We don't turning been through I levels with going on setbacks and given we're like very the years. those road up, the stronger and all and to any have and anything looking without have Biogen an it's and drugs or Matt. Biogen the in and to strong at that run spans a pipeline got gone we're everything before at stronger actually SPINRAZA going growing even the the with changes going without in We've top the I and be strong the on XX-plus working think they're Ionis higher that

with I very forward at in much a are think our we higher we're priorities studies looking pipeline. these to starting will in is of we're very pipeline And growing continue the Ionis year. very to that is this of X of here Ionis high that to be that half said, Biogen. neuro with like neurology second in our prime With going wholly-owned be pleased that example drugs

one initiation this our key highlighted only of that but a programs One year. not as key programs, program study of our lead we've the

of change think to So going the that we're as result no, I they in be reacquiring a Biogen assets don't from yesterday. management announced

Alexander Thompson

expect until data at top And the line just or clarification. the you cardiac any not midyear later? of one Do

Eugene Schneider

No.

So full the the reported subgroups be on point. will of time later the data be top all line will And population. at a

Operator

next question The Markets. will come Nachman from Capital Gary BMO with

Gary Nachman

eplontersen just to patients adding study. more another first, the So on and follow-up cardiomyopathy

eplontersen Beth. in data your wait is the it steps lastly, phase AZ million decision the is Phase patients, on and PN have informative in got like should what that some some have we the a data you there? for That's that an for the in XXX, since the the it are of soon? cardio target that until quarterly development So And could idea are next Do have from for a then of to you'll $XX the basis? also will there data And upfront before just dyslipidemic why of the kind then out? -- expect you loading one. number types And costs, The IIb based for data coming you Or been read on potentially sounds next there PCSKX we program? patients making what decision. didn't some

Brett Monia

Take the eplontersen?

Eugene Schneider

Sure. I'll question. first take the

this amendment. So related to timing of

patient now As really of quite that told in again, very the enrolled for we such considerations. it's NEURO-TTRansform? change the wait population was NEURO-TTRansform. just implementing we not been that key over the until firstly, we've of Why the of timing a one timing Brett for this time, this one. said, different some ideal as and it was kind mulling Well, And

CARDIO-TTRansform. just to on would we don't see we're us So how going inform this what whatsoever

outlined. by for CARDIO-TTRansform the characteristics balance were the had that Brett particular sort changes intentional the really Secondly, desires population driven in of

Brett Monia

Beth?

Elizabeth Hougen

Sure.

So the first eplontersen, expenses Phase -- fully of to on in it, $XX development broad the is our, call the million, the about way that III loaded quarter. think XX%

I expenses. internal CMC SPE say So external expenses fully and means when that expenses, loaded,

in of essentially first incurred $XX quarter. XX% million what is that we So that Phase the III in program

data So of that time, see the Phase III in with launching to that continue get of as study. over those the to are grow drug, to broad the those expenses going readout as to And we program course remainder particularly the and continue cardiomyopathy closer to we expenses grow. the

you'll true that is of expenses quarter. that XX% me and development grow, say see for AstraZeneca that -- this each revenue will XX% The and for -- quarter. And it's it's And responsible the so also are differently. let the the same the amount same

There's live. that. there's in loading. lag no offset, there's They're So no no

each XX% will eplontersen joint the revenue quarter line. the of So as grow expenses so under revenue collaboration

Brett Monia

so that regarding population the about that patient Gary, And if in patient same by is adhesion. That's it have PCSKX, why think population disease. important. the it's patients you like It's actually that same study cardiovascular this, was treated

in Zetia. event get moderate patient maximum an had to that cholesterol That can't they've Phase the doses be the LDL have So on statins high line, would LDL target. the population. of III and And

Gary Nachman

it, is Okay. may Great. study I late. And that going start? to I when on have missed jumped

Brett Monia

development will the study on III No in that to problem. there's but has for the been we Actual was Phase dose. a presenting the III study ongoing is Phase a start year. been confirm disclosed, decision half what of AstraZeneca second Remember, make ongoing a hasn't study that

really coming dose study III And the up. really necessarily a They're solidify that to is to the so not in wrapping That that's was It's Phase completion. looking Matt. in Solana. same dose

that the So delay and -- that's decision. the making in basis of

Operator

from Securities. The Mani Foroohar question SVB will come with next

Mani Foroohar

be your to around a cardiomyopathy. with I therapy data we to a about that pricing and opportunity avenue dead to There's for label opportunity how versus of and Not And supports use? little approved the but Should stabilizer. an U.S. prior add-on talk true after thing approach the you commercial on do strategy do want genericization think closest How for guys to top bit clear you to combination the TTR in tafamidis the a has have about to a entirely markets? the that be you about think then strategy? EU pricing label that horse

Brett Monia

that? like you would to Onaiza, take

Onaiza Cadoret

Sure.

here of I'll have you So of the in terms start with. eplontersen off great kind over thinking strategy.

cardiomyopathy. I position The XXX,XXX our as said, that patients for in payers including here to early to amendment data disease. going as well as ATTR in We're that really accelerate set about really leadership think clinicians. looking is at all players, we the filed to is important Eugene we're late-stage get, from

learned to for is very in on because patients have to and of profile, a we've the as this as their that disease. actively tafamidis gone this payers important out generate data going and it are that population top to So that's really extremely is tested of And to in target have say strategy if this that. sick The patient dynamic going as we've be I they're to they sets U.S. out will both view on treated set be manage look thus and lot, either product what to what do very they marketplace standard through tafamidis. already far we terminal in necessarily as And population data not well combination already and naive market a we're therapy. evidence not them naive? patient what to from to if a the naive therapy to obviously, If they're That's able actually going payer a physician I do clinical and to that. requires care have what or do how questions, if looking particularly step a one to treat And on patients? And a of the in your of they're do should treat on tafamidis we these or in is another is their how approve which you're situation, physician the going had. them you're this for combination are

onerous through for relatively as open in requirements expecting odds, a environment and/or but a therapies step-through prior all future well. are terms nothing payer perspective. go we actually of they Obviously, So

clinical the of price robust accordingly we'll proposition coming is clinical So to which evidence. value set be a going very where to out, is

Operator

Needham Company. The next question Joseph with will Stringer come from

Joseph Stringer

ITFX. on perhaps us II monotherapy, have confidence this -- be and Acromegaly. or the that Would give going from Syndelirsen what of in normalized patients reduction Phase year, forward? or you half IGF-X you that looking this program would for one a of quick percent in A readout second

Brett Monia

take want you do Eugene, to that?

Eugene Schneider

Sure.

really that's for IGF-X in able and achieve setting. the that in need drug, being setting of the here at-home we the bar really, well normalization both this to as is monotherapy So as set the remaining

of obviously we're excited that, for terms normalization. speculate outside can't positive get would planning the IGF-X threshold terms really very what in So rate a in I that but responder really of us of readout

Onaiza Cadoret

of attacks sets of to would both here perspective good be still And data. And their add SSAs on breakthrough from going monotherapy both over people data sets in Yes. are as that well. a to we'll important market here. here and addition seeing have are just I really

will having important. I is So combination do normalization well monotherapy as as data think a such but very in that goal, be IGF as key

Operator

come from next Sachs. will Goldman Salveen Richter with question The

Unidentified Analyst

on This is for Matt Salveen.

or you guys going -- ALS to for pricing could and could expect us in Phase I/II on when your strategy programs, you market discuss XXX? particular, data XXX, back separately, update thoughts your should Just on then give an we

Brett Monia

data bit would course, look So for towards they of that. commercial like to nongenetic Onaiza, readout. you you and drug ATXI talk haven't that's timing, in or sporadic looking study, next the XXX, for and III ALS. that I Phase why but a that really but of on much and we're that very our forward And much for touch about based go PCSKX, is Biogen over opportunity to positive. running it? don't results disclosed little would to pricing year on go could

Onaiza Cadoret

large Really sure. market. Yes,

know XX million XX about million to patients. You

tolerated deciliter risk leading still really U.S. the and U.S. and population milligrams that's So in of per aggressive on The they're about guidelines here large creating because of particularly per cardiovascular milligrams secondary are uncontrolled statins. for a to actually, very XX patients, XX, deciliter the the outside maximal these prevention,

has terms would is area. MAbs you in which entire growth will treat, shown reduction, showed substantially. this help also market Etesian in really I and what I which the to have think guidelines LDL-C well momentum, say, some inclisiran well good above aggressive So this what tremendous of above shown

and with about the LDL it to XXs for put in to PCSKX, our looking substantial and of this modeling, pricing will we we to is expect MACE for positioning really the go into the the that well. context, into will and we're is scheme looking That what going good well. Leqvio, about just which be as improvement reduction reimbursement XX% as value also actually proposition for in also in want kind lowering, a area the of the So of MAbs level translate and all at we're into at XXs really range which strategy terms the the drive

the So efficacy well. this what's the to strategy efficacy profile is -- the it's But well for go-to-market positioned to do best-in-class really drive of going agent. kind

Operator

Minter Myles The next come with from William question will Blair.

Myles Minter

Just Phase in say your seems in I on to XXX, going the prepared the decision remarks the call AstraZeneca and truly commentary the ahead. formal did on going on well be today a make you like second is that's to half, but

the IND-enabling a take I a my you in programs Or to So has what to And Beth, the And trials Brett, getting can structure, just talk Are seen Phase programs up that there? studies? III if then, one you milestone deal the Phase means? collaborations planning on you you just just these anything, clarify Phase and prepping you multiple multiple half? II would if the can Is asset last take have program. Or a is have studies actually muscle that on into for second done? development III into LICA there? know you single tox started protocol?

Brett Monia

and a the salon from stock drug, getting That protocol preparing drug the they're of being Phase this sure protocol to yes, very developed planning Myles, the development the reach data ready end ongoing much Phase Thanks, Phase meeting. start for preparing III and is an is -- They're includes I'll II after out. for not AstraZeneca explain next III making is the position now.

and with closely on them we're very -- working program. this So

now interactions. that all in II ready So getting and is had that the they've of Obviously, for -- that Phase drug and waiting at really previously, protocols ready. is finalizing end but just the interaction on All regulatory happening. motion. had interactions getting We're regulatory for we

economics the motion. in are Beth? wheels that, those as all yes, And as of far So

Elizabeth Hougen

Sure.

substantial from So we've revenue program. of already earned this a amount

associated regulatory royalties for step, sort but type significant to Phase III, the milestones a are we amount our events. next as that there tiered add commercial And substantial milestones forward of teens. into that to common of now go look So aren't very there with a the then any of amount up milestones

Brett Monia

Muscle LICA, Eric?

Eric Swayze

sure. Yes,

as development year. into what to on have objective this stated an So we we're LICA muscle advance

programs. As we lots know, you have of

have We there. get we May, first great And start I to think. one to of before lots opportunities there's

the where We and the clinical aggressive opportunities programs, be those best most of programs unmet plan moving picking to there's forward. and have in off lots preclinical and we

Brett Monia

Myles. put Ionis we're you our -- development I great. LICA state looking that, we just pressure to mean also really to LICA I under that remind the on programs, own point Eric's have is wholly-owned. move several muscle because Yes. a

disease working also diseases have with And partnership aka cardiac. We neuromuscular excellent we also Biogen. AstraZeneca with cardiovascular are in on an

So of and LICA moving than here things have expect in of I future. lot we important is what But will which less be is forward. goals drugs think question, are the in development rich first your to just a of pipeline muscle a we have

Operator

The & from Laidlaw next question Company. come will Jen with I-Eh

I-Eh Jen

the on progress. Congrats

Just quick ones. X

side? trials, to and -- just The cardio polyneuropathy first one, patients phenotype. a cardio that hall other patient the pure does In mixed with of that include any transform wanted or the simply with clarify words, in terms

Eugene Schneider

it much very have some Yes. patients No, includes exenatide.

diseases, two the you considered separate is pathology but of of really to same very different systems. certainly used are if the We spectrum, of will, significant of sort that of manifestations the aware there how an be between organ edges are in overlap

appropriate are we for at yes, the population. endpoints looking patient So neurological

Brett Monia

And with patients I-Eh, field being more the interesting symptoms to paid attention wild-type more that in There's cardiomyopathy an and neuropathy it's question. this have.

very close like It's a Eugene will something said. the So something in study be and we're it very are we relevant it's paying CARDIO-TTRansform question. assessing attention to,

So thanks unfortunately, for the We're question, over I-Eh. time. we're

to the close updates very So the We're about look on at providing and the today to have participating we're do very thank I progress we call. to to rest call. We're we're quarter, and the everybody really for Ionis. joining year the of great throughout and the year. happy of about rest going want forward making ahead, excited additional

day. great thanks everybody, then, very much, have a and until So

Operator

Goodbye.

Ionis Pharmaceuticals (IONS) 4 May 222022 Q1 Earnings call transcript