IONS - Earnings call 3 2023 | Ionis Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to [Audio Gap] A reminder this call is being recorded. I'll had it over to Mr. Wade Walke, Senior Vice President of Investor Relations, to lead off the call. Please begin, sir.

D. Walke

Thank you, Chuck.

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

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

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

Brett Monia

level extend medicines in our wholly to therapeutics. our Thanks, call. have have we our executed joining for objectives we patients, Since a and morning, Ionis Wade. with today's our X Good to years to ago, vision oligonucleotide on next everyone, us taking deliver pipeline focus done at so directly on leadership and value. and thanks strategy nearly the owned helm strategic a laser clear to bring and on build

a direct Iona to value goal deliver key a cadence our year, new this to us closer in of steady generate result stakeholders. patients these of for ultimate to to successes all our and transformational achieving next-level Our medicines which areas move efforts are

commercial are and partner, near-term executing approval. with with delivering EU approval in the eplontersen this co-commercialization U.S. launch are on first potential to on eplontersen of medicines next of global AstraZeneca, the starting prepared to the approvals year. also a potential our Canada We strategy regulatory our cusp We're following patients and year. We

planned, treatment JAMA. of the for Phase objectives a study importance the including journal, steady the of showcases recently monthly of remain profile, for the a on highly our Additional freedom respected of achieved Phase data, strong review also the profile, current transform III who its approvals together of eplontersen underserved positive therapy of positioned key filings Publishing is eplontersen world. positioning in neuro to comprehensive become believe we therapies. results ATTR around polyneuropathy well simple results. are with neurotransform Based choice reflects of the our strength III self-administration with a us another overall publication and patients the of cadence of a the results III JAMA quality ATTR and our at-home our highly by for for in study Phase like We

with first events. and our clinical along patients this olezarsen Phase the BALANCE in III top very near-term achieve in point substantial olezarsen demonstrated end month efficacy reported patients, donidalorsen, are in the safety importantly reductions primary olezarsen profile. But lipid-lowering eplontersen olezarsen Making our in with for to therapy favorable behind opportunities. and owned Gap] were pancreatitis tolerability a from most We the last setting. acute result closely Following pleased wholly [Audio study data studies we commercial line positive

and the first EU positioning early next priority assuming the With marketing in early end olezarsen these the we as positive highly for its approval year, of on for are review approval in track as results, year, to potential file next U.S. U.S.

donidalorsen, and the demonstrated of protection We Phase with medicine data, durable prophylactic recently also along safety results X-year of with favorable continue HAE. We treatment which year one OLE previously tolerability results. for our our to II sustained and reported extension and by HAE performance against be the reported open-label the Consistent encouraged attacks.

readout looking We half are for are the forward year getting to donidalorsen. and our closer expected Phase data with III next of in first

significant the And to further Advancing a turn III the eplontersen to to guidance. With total recent across activities for strategic review financial We treatment then progress drugs review results, to Zilganersen our with accomplish wrap into and also other rich our go-to-market the to Onaiza. your and briefly third our track Phase pipeline discuss importantly, status we taking progress, made expanded that, pipeline I'll strengthening of Alexander's Beth on launch recently indications. call up and III over Zilganersen, development XX pipeline XXXX after expectations remain I'll neurology goals business, our for discuss questions. wholly-owned advancing After further development. And will disease Richard X the our for financial to our quarter our before and that, our in by Onaiza in olezarsen separate our things donidalorsen. key Onaiza, Phase achieving over of including that, will for

Onaiza Cadoret-Manier

Brad. you, Thank

rate delivering patients. With and As begin ready XX% worldwide ATTR underserved approved Brad is driven promise. XX,XXX outlined from as systemic an of disease. and an remains detail medicines Richard fewer today, in estimated the a large and nature low to patients tremendous Ionis' hear are And treatment, the the and to moment, by you In of hereditary disease. polyneuropathy significantly underdiagnosed pipeline our of more presentation in patients this will on diagnosis part, holds heterogeneous largely we than

the many and wasting. largely cardiomyopathy to early of like market. doing get significant this with leading disease others still important and progress. GI patients. may treatment their untapped grow and symptom By well commercial reach, on are are positioned While to peripheral to with recognize ATTR so, present disease undiagnosed And in positioned in uniquely them deep we in Ionis' muscle may symptoms AstraZeneca's knowledge most Others neuropathy leveraging be ATTR patients in we

field team eplontersen already is and and deployed built through and our growth brand education strategy executing disease to on Our drive awareness.

at campaign [ ATTR education HCP patients our "See systems. accelerating launched recently in potential aimed helping disparate Patterns", called, the disease with spot ] seemingly by we unbranded diagnosis Additionally,

recently brand also among health our website at and treaters. care ATTR professional awareness disease aimed launched driving We

choice in investing to sources our rich ATTR ready becoming are polyneuropathy. Eplontersen gets are ATTRPN this approval Eplontersen with X potential And newly and effectiveness we diagnosed with patients with and our about to diagnosis we treatment the in the achieving polyneuropathy. measure will the Additionally, in preferred AstraZeneca team's weeks, a ATTR data support of potential be key to field to and rates. With launch identify improve deliver success underway, of goal our patients as for the patients

launch independently. to expect We medicine first be Olezarsen we the

Olezarsen two settings. and FCS We indication in indication are mover broader indications, rare SHTG the with potential the developing in both advantage first

an track have with together results potential us study gives moving Phase in III we review. from for expedited designation we launch the Additionally, FCS with our FCS. forward extremely In positive hand, patients even greater BALANCE the pace. with are for fast the And we reported the preparations with

III And and potential scale of approval. the we to in Eplontersen launch, launch established build trial for as for capabilities With Phase further indication, results FCS. FDA product, stronger to we this plan pending SHTG a position strong are follow-on and enable the prepare the for the to the full these upon readiness foundation we capabilities realize

patients to HAE independent next deliver an be because to our treatment attractive involves expect set with Donidalorsen us efficiently field for HAE. We market allowing of us with to Donidalorsen fairly This sized prophylactic concentrated team. prescribers, launch the for is it of an

launches for seen date, are believe patients data to approved. the With our and I'm treatment together positioned that Donidalorsen of Olezarsen to an and first where profile, compelling new have once pleased once-monthly important say Phase II self-administration to prophylactic a for preparing with Donidalorsen. be we we in OLE be should we right is HAE we independent

commercial place, part look to medicines we time a exciting proud to next they market. and This important be in for to begin people Ionis. to Our forward is to up-to-date is the very of you as in our it, I delivering and come our are keeping unfold. I'm need step as infrastructure ready

over Richard. to Now

Richard S. Geary

of more safety to XX durable the in continued and could through patients We Thank performance has Eplontersen you, efficacy pipeline. not with exceptionally sustained treatment and of demonstrating Onaiza. be weeks well, our perform pleased ATTR with polyneuropathy.

and function weeks life, neuropathy quality sustained nutritional cardiac improvement the month data positive study. NEURO-TTRansform we and a meeting Just XX a of quality status also reinforces structure health our this and analysis. neuro the CARDIO-TTRansform confidence status results across secondary amyloidosis the stabilized from subpopulation morning, XX The results, were of fully ATTR improvements predefined week in data and demonstrating of in XX as showed we patients improved remain enrolled, that at at showed HFSA, and last we XX data benefit for patients And of ambulatory related cardiomyopathy patient stabilized as weeks [ cardiac track from eplontersen physical the With endpoints first through with improved specific or the and in measures in on patients demonstrating of the transform treatment. And at further support at polyneuropathy in potential European indication. were larger presented XXXX. early ATTR ] benefit eplontersen new to seen substantial number impairment these neuropathy in half life of for that

to CARDIO-TTRansform and is date, study the for in to the of the generate physicians with payers offers the Eplontersen patient reminder, patients. patients, for decisions As value and designed need possible to a enable want best this population treatment over data largest to understand and X,XXX patients

to BALANCE we reductions robust like of the we statistically favorable the and Following demonstrated expect tolerability safety the commercialize drug Olezarsen profile engagement to with dose XX-milligram expect consistent is to the other profile with next is medicines. our the In bring independently. Eplontersen, triglycerides seen Olezarsen in target market. the significant and a study, and first

the therapy is clinically unprecedented Phase reported patients in clinical acute time demonstrated care attacks. study, lipid-lowering Based has standard remarkable first in positive FCS. a believe it's III Olezarsen addition, the BALANCE because with that to from result In we for trial the Olezarsen pancreatitis become poised achieved a the we substantial This data meaningful this and ever on reductions of setting. is

in in EU to U.S. Our next review of Olezarsen U.S. the in positioning next the XXXX, approval the year, potential assuming first step approval first half will for its for be priority marketing in and late file

addition those for SHTG III In FCS, enrollment. in read we development out clinical expect program patients on for SHTG. or or program patients we have late XXXX an and are hypertriglyceridemia XXXX also to with depending ongoing, our early severe studies to studies Phase ongoing in

Despite wholly-owned significant a represent on unmet already closely behind angioedema. Following is medicine, Olezarsen patients treat continues the need. our Donidalorsen, with to HAE to treatments several next hereditary market,

address study, we to our or the XX% only in in these Angioedema [ Association two at updates and robust Phase comprehensive ] potential forward the reported disease [ and next of ] HAE, a favorable of ]. III For over example, to extension the by Donidalorsen's perverse of than control and remain study having the late-stage more forward Oasis Donidalorsen patients for attacks II to week in HAE Hereditary year. HAE the we and a next X pipeline, track more X-year enrollment year. data XXX their conducted from at presenting with need. from of ongoing first durable reductions support XX% the Phase sustained We in recently [ the patients on IONIS-FB-LRx reported data Data reporting attacks from years, ACAAI of and look per over And look Conference. With with OLE safety tolerability unmet look study show completed half good open-label

Phase of ] [ to Week, the from B to present study plan week results medicine new our partnered plans combination data Roche Next nephropathy. IIb this present perverse of the study new together our with at from patients ongoing IgA Kidney [ [indiscernible]. In AASLD, interim GSK in ] at with weekend IONIS-FB-LRx in we Phase II

IONIS-MAPTRx clinical by the development syndrome, in JAMA CSF lead development. multiple cognitive breakthrough our And for positive in the Alzheimer's substantial and extension rapid, tolerability industry-leading preclinical recently putting in completed Angelman another Phase and and significant on data early-stage Data study. The reported showed numerical or improvements [ We we highest XX showed we were the clinical track in ] have reduced represents market, favorable franchise building with this QALSODY. optimization. Among trials encouraged XXX our I/II functional our disease programs, a neurological also on of made we progress of our with well sustained and study imaging scales from excited with with on our pipeline as around neurology early on Ionis [ of priorities. and recently programs in XX more this pathology Alzheimer's have enrollment of advances the continued neurology new and medicines than advancing weeks year. Biogen approved phosphorylate treatment. tau to wholly-owned published disease. we MAPTRx SPINRAZA up and neurology with at for safety disease we're Phase data particularly X partner the in presented and one year. Ib XXX on partner, patients [ cal the ION of and and us next ] middle the Neurology as conference small and tau in in and reduction data studies long-term making ] And which PET in patients in we're Today,

debilitating development advance more year. This treatment. FCF Donidalorsen been on in leukodystrophy additional end with and months, track prion Olezarsen And into the Phase next so has Eplontersen approval, Alexander's XXX rare expect in ION clinic X several additional medicines key Ion we XXX, mentioned, filings looking a III human III far, forward patients approvals study and into to and events in outside in advanced data. launch no year with we're we the eventful coming U.S., U.S. in and Phase advance the Brett disease disease neurological the Phase the regulatory filings to including regulatory approved disease wholly-owned an of to following I/II before first Zilganersen patients year. pediatric the into As with We're

We you events to on year. the And will and with throughout coming that, progress Beth. keep our these on more over updated

Elizabeth L. Hougen

Richard. you, Thank

driven million Our financial on year-to-date be achieve sustained level and financial execute results $XXX our revenues guidance with to our to unlock keep us Revenue on to track continued XXXX next X X% of we while a strategy ended substantial value. periods year continue and to $XXX timing and last September decrease million compared and XX% reflecting in X months payments. respectively, and XXXX, the same the XX, of partner certain the increase, a to by

to our loss with the commercial we As last especially and for cash operating the our end as billion continue and investments period third same at advanced programs. in us our anticipated, our We operating quarter our and investing goals. expenses and late-stage readiness pipeline, remain over capitalized of enabling $X.X increased activities strategic the year-to-date year in well September,

resilience single-digit the and global from third million Our year-to-date, respectively. SPINRAZA in emerging commercial quarter competition $XX revenue royalties the the compared and against compared was U.S. quarter quarter and last $XXX year. a same abroad, million the SPINRAZA's Reflecting to last low third in increase quarter and to SPINRAZA's sales in demonstrated

in numerous creating line $XX The quarter technology SPINRAZA result, to quarter same with to partnered by third the the continue our that we programs and a As to as of X% third expenses Ionis' earned million our growth, quarter year-to-date value quarter Phase invest most With ongoing And enrolled, revenue prepare as expected, fully in last increased our expenses. year. study higher increased our Donidalorsen, studies increased for and costs we and R&D compared III Olezarsen expenses to the also to Eplontersen, SG&A $XXX from as royalties compared the and generate R&D R&D is increased to revenue modestly X% last which launch year. We of in last operating revenue non-GAAP year-over-year. reflects revenue goal our year-to-date. resulted million compared significant advance. In our

track year-to-date to keep our results meet guidance. on financial Our us XXXX

of We to revenues than continue million. $XXX project more

expanded with quarter expenses additional advance. we of XXXX the our to from that our continued partnered the a collaborations $XXX at base come from expect our Biogen R&D And important be of continue programs revenue milestone and XXXX. driven higher our as of million from We sources to the end XXXX Roche. We development and sustained is $XXX of R&D from payment Eplontersen. an multiple about expect guidance, Eplontersen $XX revenue AstraZeneca ahead, to for by million. project we our between to Novartis which and recently looking substantial source non-GAAP and U.S. generate in revenue Eplontersen fourth operating And in and will in revenue R&D approval expect from potential be million revenue

additional the potential outside We milestone have earn approval an U.S. a Eplontersen the to payment for

line We SG&A grow ramp up. expected in the expenses with the up launches state ramps generating year, and R&D are to next to late-stage also Eplontersen, our with of And with modestly revenue growth next as expect royalty steady Donidalorsen. our We begin our planned enrolled. launch expenses approaching modest project as our Olezarsen year fully to expenses programs

recurring Together add revenue, can from strong a and R&D that advancing to owned we foundation, wholly continue sustainable have grow, product we on to our Brett. revenue to And We successfully I'll to the believe substantial royalty to we pipeline. a call that, with revenue medicines level expanding and a with and substantial financial new unlock our over path expect prolific and our are technology, patients bring value. next to turn back

Brett Monia

Beth. Thanks,

believe remarkable far this are patients, all shareholders we've drive this very for and to value that the the substantial so us of progress we've made year. position proud our stakeholders. successes Ionis We achieved We year

deliver Strategically, vision. on established transformational the and a We've by we to our today we on a our of achieve being we medicines new clear have now the potentially and all functional arrived market. strategic focused where objectives being by capabilities support steady of focused to necessary all are need cadence vision to and

and as development. a our drug established owned in advancing wholly pipeline neurology Ionis are and growing leader We and cardiovascular have

financial need medicines foundation, and robust most by late-stage therapeutic development our growing indications, objectives. And our our with turning for X, mid term. opening for position pipelines one new the open strategic patients the we stakeholders. With our not providing for industry value Operator? all with of steady X in in existing support strengthen this today, medicines all discovery new for now in continue and the the we're great the new that positioned cadence optimizing over long And to and areas technology, drive extend promise X and we and into call our for therapeutics leadership to XX are of or Iona achieving in capabilities that, but up a continue means I'll further for to We to and goal, questions. up medicines Oligonucleotide diversifying drug type expanding in just areas transformational

Operator

from James. come will question Raymond first the And Instructions] Gary with Nachman [Operator

Gary Nachman

So on updates requested of in the Have are your additional in how would interactions FDA progressing Eplontersen everything front and then for PDUFA? the with just ATTRPN, is any you plans. on the discussions way them to? And additional the and regarding progressing data any commercial they label like

a competitive with will is What's payers work on dynamics the will you be how force? most Given on AstraZeneca sense be? of Will in the all drugs [indiscernible], viewing covered how space give the the telling us of that you think And there? Eplontersen? sales be formularies? big hiring Just where your these

Brett Monia

give I'll one Thank competitive you, first to dynamics. an And on Onaiza Gary. then update cover part the ask the take I'll your question on of and regulatory. second you briefly

on with teen So very bringing regulatory discussions confidence we we're either on making with an December drugs is PDUFA the to high approval. don't ongoing the say I FDA progress it's approval potential for and we're -- on-time pleased up on XX. But date comment can with for what under review potential that

with is year you also those accepted with that remind TTR next -- want that. And and in well for under now and polyneuropathy on as I Europe [ track our for really approvals everything Eplontersen are to with markets everybody potential Onaiza? ]. filings for review Canada So in been there's moving we

Onaiza Cadoret-Manier

would actually really underway. say place. plans are they're Launch I in

by on disease growth underway. patients really symptoms The much working types is the salespeople, necessary and the deployed hired hired very led a and identifying all you field, are physician is is in ATTRPM in very present are the this awareness, sets, are that market, with people a have brand for AstraZeneca launch. given being very, that They very as market. know, they We with different effective of

You've seen the patterns. on see some the campaigns of

and we motion. just they're month last PDUFA all in since are So in And awaiting out approval, things is are really field. December there XX, the

the trained. has have you And to managers are them would mostly as deploy know, right they're the field case nurse the for since medical Our on and been hired team approval, months Ionis after. last in side been And place X XX post years. we

go. From to pricing. really the in strong strategy. very don't research done expect believe authorization perspective, for think of a different all anything of priced have We've rare on disease. the have a So We disease through. I with of a signals disease agents tons We to beyond the normal payers do that not the are payer other active analogs ready get payer lot majority rare than U.S. expect kind we prior a is this rare we Again, a been of of the

shift so much seen here the haven't landscape in far. We

Operator

The will Luca next come with from Issi Capital. question RBC

Luca Issi

your on provide the the have GSK ones. do question, is up trying licensing facility [indiscernible]. second in transfer commercial circling you, that quick need keeps already ] why if then you given to AstraZeneca two B. TTR-protein with expected any maybe thing reaction anything been FDA J&J manufacturing? understand will Hepatitis One completed? from Just Maybe that to the was there they they agent a you on What PDUFA, [ supply? and a Arrowhead tech back at Has [ prior [ ] successfully Has think so ex-iRNA on one? And sound the to the color ] night the that the

Brett Monia

Luca. Thanks,

good very GSK. with relationship So a we have

a of a ] aware the way that [ Vanson on if this it really the we're you acquisition coming, week. was think what represents of was double Bepirovirsen. a molecule about RNAi announced Bepirovirsen to triple So down, what down, meaningful The will, is study, produced from patients was a has with with cure achieving functional the And that percentage of a GSK treatment about we in cure what the in XX% HPV. demonstrated meaningful rate. chronic patients HPV, CLEAR IIb functional only the is Phase

really cure which that XX%-plus I rate into think the lower like a IUs per with milliliter, burden However, impressive. functional was is ml range. in per -- HPV patients well X,XXX

RNAi it antigen the strategy reduction a -- molecule, is the bringing by GSK down to has that get level is cures of a a which in more stated that for of IU maybe patients levels functional shown shown per functional substantial treatment cures, X percentage X,XXX, even IUs patients what percentage bringing below in so has not has levels, has but which And in can that HPV to more X,XXX Bepirovirsen. sequential milliliter patients X,XXX do with to even to patients level then achieve reach below and coming greater below

So different this is percentage very clinical also possible an And for for as as patients to hundreds a the saying Bepirovirsen. functional know, with added dimension people program in from many for HPV. has is of GSK will and success this to achieve reach market, maximize combinations as chronic for that everything millions possible you comprehensive it's exploring to be on consistent Bepirovirsen which highest they which, cures to of Bepirovirsen been suffering

Beth, Bepirovirsen. And this So you could are bit for -- GSK outcome little for supply? a launch with we talk commercial about with pleased our new about we're maybe where the very the preparations

Elizabeth L. Hougen

Sure. Absolutely.

are ready just commercial to all We medical side affairs the the as on are and we go side.

contract the gone into well. product all shortly AstraZeneca manufacturer all needed registration transfer that is do from label of been -- has go. get and has packaging to regulatory of after done. extremely are reviewed And necessary with the ready commercial manufactured of to It waiting approval through manufacturer, and is for label All been tech the products final process Ionis and a just for We channel has the the launch to was manufactured.

So go. is to we all -- are ready systems

Operator

The Laidlaw next question Jen Company. & Yale with come will from

Yale Jen

two got I've ones here. quick

I should we Then -- you But ordinarily Biogen. SMA I quarter-over-quarter, in with The believe again, by is, the of at be have larger stabilization that first that the one a is follow-up. consistent the growth growth indicated quick will franchise sales, for space? nevertheless, believe this announced the small that the SPINRAZA

Elizabeth L. Hougen

year. that further. and pleased we're even quarter the to same SPINRAZA's of face single-digit demonstrates with Biogen's the any -- the low quarter continued that, believe really would and We really emerging last next against last need I I What previous growth go earnings say week, I is call -- resiliency Beyond competition in very respect in quarter against can't

Yale Jen

publication terms incorporate think of strategy, how we a And or treated JAMA to into patients? for that the in sustain marketing the untreated about to should the particularly untreated regarding newly benefit, [indiscernible]

Brett Monia

going [ ] right, publication to that's The specifically? tool make I be Onaiza? really effective a mean, to

Onaiza Cadoret-Manier

kind Yes, where the and JAMA looking to publication going our regulatory supportive. are and I highly JAMA think publication guidance, promotional is at as of the the The promotional our as we be the It's both with expect. what messages also by very for teams sales and medical consistent label that allow expect, so will well, use the teams that that's with headed. we

Brett Monia

Yes.

week very the under from week to that marketing words, So a XX effectively. and other FDA , on XX standpoint, based helpful publication expect week is the that's that very we last in and interim although our for at package XX publication very, review is data we reported utilize year, this having

basis I your think that was of question. the

Operator

question next Stifel. with will The from Mattias come Paul

James Condulis

Paul. James is on for This

trigs ready it price different you that as you've higher getting I from force think guess, sales in how of or kind you assuming anticipate launching Olezarsen the points this just the quickly. opportunity? commercial place be I of put are be how launch, trade infrastructure a FCS guess, you some this may one I I like as And the on guess perspective spend about given opportunity either just coming and will from about for behind FCS get Just for we FCS guess leverage launch, thinking pricing as higher do there. infrastructure there the much,

Onaiza Cadoret-Manier

Sure.

rare different disease make does be will obviously understand dual cardiovascular indication disease, premium indications more that in no line indication, of of benchmark of single We the good So and in pricing rare strategy? broader we're once kind that were quarters terms that really result have priced payers cardiovascular with indications and we've your indication this see in corridors priced premium the know about pricing extensive kind indication a work the good going a in prevalent pricing we shift done with a they the Do to we of two some actually the along after prevalent really the thinking along rare molecules. price as well. if to as products. We disease of this differently in

integrated will are qualifying our broader as physicians actually sales that get those the be and important and physicians, believe from call work ] will on, doing So [ on be would about sales obviously, focused lot the have the SHTG sales And obviously very specialties than into SHTG. think incorporate a for we'll team universe on on we well broader a total for you be for some way. I other the market of then we will would the board, that that where to the population the the portion the of we're size uptake it physician beyond of calling team a patients. that much extend FCS should the of of smaller out stay for that account SCS lipidologists, bit will teams say where analysis believe product. those as that It more we data but indication, extend FCS is you we

Operator

come The from Piper Allison Sandler. Bratzel next will question with

Allison Bratzel

responsible your into XX. curious describe be assets. And on is ask at then confidence asset. how also to of could second influences to II in data on complement wholly be it a program, what that thoughts for week still you. that then wanted have just update at landscape But fit how view in potential of Phase I'm IgAN, front, I paradigm Factor couple just on I given hoping data the monotherapy the of any or guess get your the Is of looking could being the combo for Biogen context an there just or approach anti-tau evolving days. on on just BIB treatment presented that you nephropathy Alzheimer's just B the development the a market I first, in last interim I'd maybe update So and just know opportunity IgA the owned your in neurology ASN there? competitive

Brett Monia

dose Tau II to degeneration. Phase in in being extension be because study reverse the improving which at Phase study referring downstream of more first now and a important than shown and we study. And well lower week with is treated based most all long from cognition. study pivotal this support is we more And It's not extension the exploring that's are for that impairment Tau II patients a efficacy enrolling. year deficits Phase CTAD primary well the for have and But up just patients Alzheimer's target What tangles JAMA a The Phase in year improvement different and in an based in you're the be and the cognition difficult-to-drug can we signs, our cognition. Tau the development generated the for to are it's imaging. beta a could in that long-term different target to Alzheimer's patients of as presented the [indiscernible] that to, disease very of in data incredibly dosing, that that patients substantially the as with to by before now thrilled last a of the make go by III tau in really considered long-term set study, the field the a occurs. dose trends PET disease. of regimens, with from Alzheimer's the in essentially data actually underway occur. But -- neurofibrillary path with decision purpose appeared actually present Phase be long-term program Phase cognition I/II not twice levels patients amyloid, forward study CSF, data study supportive is for matters most we it's intracellular on I/II And quite overall. We pathology can and including on really endpoint actually was efforts study before to over Biogen, Tau that a in tau, with is it's being exploring extensive data, have, that time really the disease partner, Neurology also evaluating which published the we can is closes is our only is and II was is all what forms that as extension the XXX

to be referred confidence the data. this CLIA about on underway And earlier. our So we built the has that from study thrilled I couldn't II that's Phase more

with and it program with the how those As on. intended far and of each thing not actually drug can other. is development and We further a -- require Phase for the sense. at with as does whether see assess two that's cognition treatment in each combination but kind future so Tau-driven way can II leading the the monotherapy, synergize The Tau. can other to combination That start this of looking development studies it on different this even beta That evolves, is combinations that a that's as study will to the complement mechanisms amyloid preclude -- make maybe target Tau of benefits a as be field monotherapy.

start evidence, piece the neurology two for wholly year on program, are programs a by that and platform. validation platform using same as all So are for end and are we platform with bring start kinds our as now, for all of wholly-owned the the all Ionis table. the prion both neuro, Tau, using medical the rare indications. on study SPINRAZA neurodegenerative as next remarks, diseases confidence the owned X to clinical this and same as expect in highlighted of leading diseases need to currently forward disorders. different that have earlier as in that program and would breakthrough that is platform another quickly high treatments of II Biogen Angelman's our year But as just for of same data his in I large a using right far Phase neurodegenerative XX neurodevelopmental approved that focused our neurology more of Richard as as is this possible. And unmet development And program drugs QALSODY,

adds in just neurology leading confidence our it to So platform. our

presented last IgA iGaN longer was nephropathy, far cut that study. patients, an at Kidney presented more will in ] be additional in [ Week This coming showing up. As Week proteinuria showing as that at be data Kidney reductions FLRX more with data, to year just treatment, in patients

it's -- glad data unmet have. presence that's Roche they to with the crowded. to market As tremendous like you're really of might, to a question, have on global no like and we're the see I a possible. as it's a a itself. the couldn't to But positions Phase the more But global lead partner taken a far as determined many be this once III to has global as bring get patients have competitive be because strength, we landscape, pleased this great drug and successful global to competitive it There's this think When need, high how really, to we see strength Roche. that

Operator

from next Yanan Fargo. will come Zhu The Wells question with

Yanan Zhu

I early launch very the we visibility performing large you this have the Eplontersen, of see On can drug indication. ] talk of provide route if of what in can in an questions, so Three would program, the Tau market may. that you intrasea the is [ quarters? On the metrics how how you the launch in about the understanding administration

that data Ultragenyx, program, as Angelman any lastly, following be could -- look we data how on syndrome differentiated the program forward regarding insights the from to in program updated On mid-XXXX? the your could recent share you from

Brett Monia

we'll Yanan. Thanks, maybe start metrics? launch with And

Onaiza Cadoret-Manier

temporary. think think this seeing Eplontersen. And have I market, We're squarely growth mindset that treated worked to diagnosed diagnosed it's through we now I on focused Yanan, patients I market, will and about as newly to lot get phenomenon think they have the And the So to growth with a we of growth be and get patients. will on way think on a that. might is you I have switches said, very and

newly [ launch for ]. Eplontersen As be such, choice success measured a to diagnosed our silencers will getting preferred on as patients new

Brett Monia

we'll and Yanan. tuned stay see so for -- that how metrics be year, providing that next some we'll So

program. Tau the Regarding

many So is treating program, Alzheimer's and with delivering on if an expect in and medical discovery don't best the [ will And capabilities progress to neurodegenerative the the very I as drug arm is homedicinal that's Tau the advancing twice the as we've very broadly. recently really so we on like is ] This chemistry AD. just unmet that In [indiscernible] program patients programs further we've with efficacious think been in that and is example for we a monthly twice earlier, for disease, now have optimizing X of -- to disease and some to Tau our actually market despite from And high we're a for making experience. target moving year a said, severe of we that's we a the Tau great progress to neurological that we medicines accepted remarkable be program Tau That will the made year expect the disease is every comes need. the per touched this moved months we dosing, been programs, still drug that treatments Phase II part today. treatment intrathecal. to disease development with dosing need be. With delivery it efficacious well for be believe with

addition, radar In as treatments, of or obviously, organization intravenous the from administration. programs progress an drugs in obstacle as highlighted as barrier great for Tau our our our few blood-brain subcutaneous we at on for making programs. Innovation our are like other research a we're delivery overcoming ago, weeks our Day And using

like that be think a also future in we that, for disease. So population, the chronic large Alzheimer's indication could disease

we're So with no but progress pleased there making promises the there. yet, very we're

programs. comment syndrome, the I say are other study overenrolled actually programs, Angelman we I is enrolled not on Regarding bit. program. that in fully prefer We people's to will our a other companies' What Angelman's

midyear next year. data expect We

got, III vast safety is a of say to that kind it's efficacy a that. And to that is course, experience what I well data we question from amount to is have and set tried We based decision on with biomarker diseases, neurological up all I trial -- And potential I'll also designed in of our related and Phase which that patient that trial. as, then on for be earlier the to of to Angelman platform address, And data thousands experience Angelman's expect diseases very bodes that and treatment Ionis the patients delivering neuro chemical to with that the well take like extended the have of platform our going think of our periods with and thousands from have with been for thousands know-how, that they're neurological everything. benefit vast able we for time very they're we that, treated with because going severe -- community syndrome. of drugs as at

very all tuned the stay for next We're that. year. to much data looking So forward

Operator

from The Debjit Chattopadhyay will come next question Guggenheim. with

Unknown Analyst

This is Debjit. Robert on for

share could First, you thoughts [indiscernible]. on pricing any

on when Alexandra to anticipate price time Specifically, high the readout based see lines? low III? anticipate CM would you those for on the what Phase a Phase III end. would line program, the or the And subsequently, potentially in you treatments with And would enrollment pace current treatments PN hope launch end in in you of

Brett Monia

launch would [ you take want the price PN Onaiza, to CM ]?

Onaiza Cadoret-Manier

Yes.

that reason would indications, think So don't that kind to if they in Robert, believe terms we both disease we on all pricing analogs you indications priced of at of I rare looked the but shift marketplace. Based the work that, again, any expect from the they differently there are is strategy. our that done, are we've two in

of to would I according polyneuropathy be expect by products. analogs So set to priced and would AZ other that the expect as we benchmarks polyneuropathy the price that is

Brett Monia

the the a is obviously everything through waiting when the you're or III Alexander can on program. And most results, data, what pause, we enrolling dose the newest before data, you're of this will data, situation the cleaning same be our patients sites. make, our And development. reviewed we of is rare don't know we're the in next [ was III Phase program to and had indications study through escalate protocol what's during our III escalation all that we for same that Phase ] part has the such really move on it's about that unique because program. two the III were that into review it, regarding And and after design your step that. when Phase you present been which the program, all be seamless our I disease the for designed to disease next Phase either But we the already sideline to It's not time, like phase dose the all the the baked One II -- Because challenging are patients was the step Phase they happened Phase or challenges. collecting rare process finding disease to III into [ Alexander can And program, in right? actually get is enrollment what data of review decisions put we ]. into go

said, on the program now it. the Alexander we've that our enter So III with enrollment because we for qualify patients that have program will go waiting we activated think well actually sideline to Phase to

So we ongoing, Enrollment is data and expect in XXXX.

Operator

Yaron The next Cowen. from come question with Werber will

Brendan Smith

on for This Yaron. is Brendan

of quick couple us. from a Just ones

Phase this, Just another the Phase me, one sorry any there Matt, study or the readout data -- Phase for any but updates II. excuse the II on missed any full look if maybe -- before wondering II full of Tau assuming possibility interim I just there's XXXX at if for plan additional any

et if think expect to different cetera. in have anything the on quickly few study, there then the one a planning new you're So But we maybe studies wondering ongoing you Donidalorsen. switching incorporate can I into including meantime? And filing, Obviously, there. just

thinking to a maybe you'd to do the data in think next of for collect timing line just top you're give if readout need first analyze second to file year, of assume. you the how data sense that's can next of So you and half us the beyond Maybe fair thereafter long in year? half and if maybe

Brett Monia

Sure.

for plan interim the no look at time. an there's this tau Phase program study II in for So the

answer. as improving It's study I get III as this to very a Phase for the answer, get -- what is mentioned we're impairment. this looking dataset richest we're decision you short evidence right and is And -- here can. actually possibly a it's important support you cognition to quick that's So earlier, that cognition

as long, mentioned earlier, XXX patients. more to than that that's take going studies over year So time a I

So this no time. look interim at

from support the data NDA. the in there's and other think Phase to will to And investigational is Phase a conducted we've that with identifying Donidalorsen. that which in as and II to then don't a attacks treatment. Tolerability existing sufficient for that actually expecting the true patients no data ] switched in to all on [ our efficacy include medicine, assess switching Phase we're our gaps treatment we've an case, mentioned, including data, the only data that go I I do we to we are to information our has should everything, be to therapy, goals one Really, another to investigator HAE the we're want along between are going want on be I Donidalorsen, protection a said, study, Okay, SWITCH expect to that are Donidalorsen, positive generate switch outcome. With which demonstrating assuming sustained an III over sponsor treatment able filing against how switch the the how prophylactic prescribers drug, For the switch have that treatment from to that's to said actually I filing, your of which, study. do generated knowledge, this? And reminder, but do I on drug. my want that to this? patient study. Switch I to understand your really our switch

time, is allow But to this the year to to us out least switch my that to expect year we really no that to a into Donidalorsen going a include in next market. NDA filing. be have market, in next value or cut that which the data plans at that's knowledge, demonstrate at of this data actually We

Onaiza Cadoret-Manier

it with more it, all we in it it's uptake. If promotion. really be extent but not used that could the we actively commercial like strategy looking for publication. we're to publication will add nor we And believe what is sufficient for our consistent if it's ready that going We teams label the our sales in, alongside That's to than by to really it's in the think really actually forward. by a requirement be necessary of be

Operator

will from JPMorgan. with question Jessica Fye next come The

Unknown Analyst

for has forgive a CARDIO the triglycerides And I this, ball an remind but me in whether on those stopped around on is you could follow-up clear a I if is transform, there reach assessed weeks. a for in me to for believe study XXX. your impact you minimum HTG. XXX the not stated week, you are weeks I show do who expectations whether I you of Can noticed just pancreatitis think, endpoints up minimum addition of to early? clarify, to planned Olezarsen then second, the believe impact Is not wanted some that XXX the assuming

Brett Monia

take Eugene, to do those? you want

Eugene Schneider

Happy last start to. one. I'll the Sure. Maybe with

being said, fair statistical some on weeks, detail patients, plan, as which that but time reads time early the met. study come course, be the means on and the the it, and in you don't I [ for specific early, conditions on if defined early for is the specific lines in on the sort that other So think exposure SAP. will the specific the out than time minimal the general study earlier point, our closing statement those exposure excruciating points of those we've analysis that. We've than of looks level based just of some ] out opportunity And included less summarize to exposure up to study about XXX the general

Unknown Analyst

the be? study in the then would And event stop the minimum don't you what early,

Eugene Schneider

the treated -- all we if double-blind Well, patients for we period. That's go early, stop weeks. don't study XXX will the be

Brett Monia

SHTG Okay. likelihood And AP ]? then [ of in achieving

Eugene Schneider

Yes.

So an what those program combination at large for effect. SHTG, we terms events. again, that, to studies. the population we of that different very be again, AP we'll in the course, show designed it's, FCS believe to And two two have of of opportunity a also from we an Having looking are very certainly, studies, said risk going is a

of about at looking So those integrated combining to together amount large X,XXX sort which efficacy, two of patients. almost analysis studies,

effect is were course, we of show. the confident, pleased our What AP fairly with population. FCS can extremely we we know we're that data will So today, the what in don't on say events

need wait strong, that believe to do out. data we until very the read So is thesis we the but

Operator

question will Kostas next come Biliouris The Markets. BMO with Capital from

Konstantinos Biliouris

discussions percentage absolute in ] of One very on demonstrating the TTR contribute the treatment. said, reduction, the have and [ important of from also can the on post an absolute fibril wondering ] are quick [ data TTR formation thoughts you levels the that actually this after space around on levels TTR ongoing presented from serum as have us the that survival. That rather in reduction given to in the ATTR. to literature treatment morning, TTR I'm that most percentage question they impact of whether they of addition any Telia focus This than

Brett Monia

benefit known making if are and Eplontersen study transform important, that Not Percent actually I absolutely on then percent neuropathy you very the it's party that individualized And editing mid-XX% we've a [indiscernible] not is in very for mean TTR and per a reductions question effect, of progress the of pleased gene for patient think of -- know, the you quality on time I that I lot by thoughts myopathy reductions the life. we're if neuro -- achieved actually with in that patients you'll actually important in a steady. reductions But TTR or they're patients, don't that polyneuropathy in we've we've achieve or range. And space. the mean, there's efficacy. efforts and whatever. there's improve. that's which number a specific a TTR for threshold mid-XX% threshold substantial long as think is is there's lower your a [indiscernible]

Cardiomyopathy And require never going about there. get are up. that know will to take a to view it's long know they just trial way except And other to our a So in to have time I to compete. not go. platform to actually new be come approvable, long it's that don't what but a going you indications programs, outcome an what to those say

than we much Kostas, know very it much So program, we're I don't to but that about ahead. love our say else other and

Operator

will & The Joseph next Needle with Stringer question come Company. from

Joseph Stringer

gold trial of what Roche? [ collaboration expectations ] data geographic line data? When we ] next given in on program expect looks II the Phase competitor on the there, what II top one the are quick And results like. a the out [ successful that's Just steps positive, some can if sufficiently can be outcome with you in Phase And handicap would atrophy.

Brett Monia

You want Richard? to take that

Richard S. Geary

at stab a it. take I'll Yes.

comparable a geographic atrophy than better and trial positive and I think, by of we have competitors. been results not if on other presented want course,

that's the So goal.

out be is insight next We what second half. that today, will have no into data the but year,

Brett Monia

Yes.

whittle right? said, doses complete and to of the is bring in study, two that goes to in half doses as us we're to had we to we Factor tolerability, second the to So and half, We that Phase year, interim this seeing that, II I Richard allowed then the of those, started what also to where add an with products it a seeing can Phase down split the finish reductions select be. exactly second effects number downstream excellent Joey, like complete profound in study to study, look next II in Golden line. We're expect the B

not going we're improvement is, we're we a see acuity. seeing to any lesion the associated risk decision expecting Phase that support to the So we're I getting with great what alternative visual to see mean pathway, outcomes or hope target is these to target are formation blocking down expect engagement in engagement, by and of go slowing complement what that to it's III great. not. And whether we

far approved using it whereas drug or recently have at use home been competitive know. are and once-per-month effects, this auto-injector as well like [ that market got once is review -- drugs which or As under they a landscape, you an apontersen the as the could there. ] subcu if Simple autoinjector it side intravitreal to [ gets ],

administration. and a made for and although patients waiting [ at-home [ so subcu that's So this invasive -- a in GA, ] like simple progress desperately convenient there's I not joint ] are been think think I

Operator

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

Myles Minter

had just I question a on IONXXX.

American an got presentation at Heart you've Association. think upcoming I

previous knockdown molecule? that the we whether is XX% dosing than be AGT we're that that similar just Just to like a more sort wondering of that about infrequent more should and molecule with thinking to or get potential seeing

Brett Monia

Phase sought being dosed So that to on AGT -- at is monthly. reductions AGT will in a the our the really presentation II molecule we is that This achieve our in dose-dependent our AHA in program on focus study.

to you molecule chemistry. was expect a we in weekly advanced generation earlier that Our because see molecule and generation earlier greater mentioned more reductions our it's

So that's what we're expecting.

Operator

The Lebowitz from come will Citi. last David with question

David Lebowitz

that forward going achieved to pancreatitis, pivotal as benefits recent and data, you respect do to given Olezarsen, in show the With look But reductions. received severe respect the to easy given I to you a to you think dynamics be that -- benefit? pancreatitis know the hypertriglyceridemia, how with you differences population. How it's

Brett Monia

the David. that AP FCS. been turned the reductions SCS our today we saw And we in a reality. lot question, hope it's would a into it that to year. The different question a answer are have while streams those than earlier see dreams, data, That's great in we our is wildest this And

events. These above SCS. known in this severely AP. My more at on population, the point demonstrated monogenic genetic is So those could in although above AP. be actually a our patients that has been elevated or in of least like patients mentioned in that puts Sure, XXX lends many, our a causes. lowering patients highly and which as at elevated pleased, triglycerides patients. genetic and triglycerides have SHTG confidence we of has grown from FCS at the time are remarks not not other which, still that so thousands much suffer form And some result can no lipid many efficacy of we disease to first outcome triglycerides, a for It's -- because these anyone very the it's risk to -- of them remarkable is multiple but the was like confidence are reduction in high AP range earlier, X,XXX, in X,XXX which least like range, the

size and is SHTG study. to we per versus SHTG study. a growing, and or on FCS, effect that the the AP the based it's the but going we're think we events I bigger have effect our And a Phase get AP that in So saw It's Olezarsen III events be FCS, in of despite to confidence see number such bigger much patient may fact study. the that. And less with

updates, III of that COREX CORE the events really data look we're ] So in us [ at much this additional [ the for two delivering we're and who's great plan on going we here and SHTG. Thank all a objectives until as to key the on Ionis. everyone we've our year And very have Olezarsen much have then, it. made commercial thank that combine in we really progress everybody, are collect. And updates, Yuji proud you, day. again, updates, pipeline said, But by continue call. and today the ability never and everybody, future studies, impact to we to technology ] thanks, joining as you and been more HT And overall has brighter look momentum go David, Phase we have going forward. forward our at to to we to We of participated believe

Ionis Pharmaceuticals (IONS) 2 Nov 232023 Q3 Earnings call transcript