- Earnings call transcript Q3 2022

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Operator

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

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

Julie Tepper

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

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

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

Brett Monia

us Phase This year. pipeline products to polyneuropathy of forward us of eplontersen morning, These to tofersen on position for ALS. conclude from Thanks, potentially has call. our in We're our in to growth. new thanks Ionis, substantial catalyst-rich next joining SODX for strength setting today's commercial for adding important XXXX and Julie. course, ATTR are, portfolio looking Good significant III rich X for and us enabling everybody, for and been year up strategically a

progress, III year, our far data we've advancing With made XXXX for our our half in This progress all key and so first delivering mid-stage have commitment pipeline opportunities, expand continue beyond. our positive cadence year, data product XXXX, also the III And our to medicines the year. preparations poised XXXX, and expected pipeline next addition, donidalorsen. well readouts with a is rich new X to of in this to Phase near-term pipeline. and starts readouts advanced to we III Phase transformative In on of based Phase we're excellent late- positioned go-to-market the X market deliver olezarsen eplontersen, commercial our steady

we expanding medicines and muscle of for both improve activities. with also involve LICA enhance durability. diversifying have programs our or made further programs to to our for additional chemistries. facility. programs ensure new new We capabilities, also to our new capacity follow-on our expand need support began a and our discovery This continue patients both drug state-of-the-art growth. future backbone into and expand phosphoramidate, metal recent to into we have technology chemistry incorporating steps deliver progress and We've to designed recently the our MSPA to on continue and advancement enhanced successfully and manufacturing will These delivery our technology development great We facility X advancements first efficacy the new These medicines work programs. IND-enabling to our taken

transaction remarks sheet to call that, to our the balance I'll third facilities estate recent transaction. as record over by This financial assets full further We impacts our real key our strengthens I'll data review to as of your through financial quarter also support transactions plans And our financial the questions. accelerated growth. updates Beth prepared for readouts estate science our after sale-leaseback guidance. the Then will catalysts. several taking our pipeline real our turn recently upcoming well before year wrap recent Richard our capitalized discuss a and results, on up preview review life With our monetizing for demand that, of

So now over to Richard.

Richard Geary

Brett. you, Thank

this While data our pipeline far positive has certainly performed extremely year. well reported We this so readouts year. X

we Phase a having X market and result, potentially on are next forward medicines looking pipeline expanding our III new the to As year. rich

next the I achievements review will our our preview Over recent minutes, upcoming and catalysts. few then

Our next reach tofersen. the to potential medicine is market

priority amyloidosis. a on form eplontersen improvements compared substantial significant Tofersen disease-modifying the is key interim April in medicine XX, its Our change impairment a SODX-ALS. FDA, life XXXX. of efficacy in Tofersen for medicine PDUFA has end first including of approved and presented date become Symposium points III to September, for eplontersen TTR the relative ATTR placebo the quality Phase patients has genetic the with data the historical from with clinically study portion the for to a neurotransfORM International the positive the of of in statistically meaningful under robust currently ALS. reduction and X-month demonstrating achieved patients neuropathy with review to analysis at potential from treatment co-primary patients. In of highly In baseline baseline we with and polyneuropathy secondary a

look positive in regulatory for with these year. U.S. approval the to With very the patients the data, we of forward and TTR polyneuropathy end before AstraZeneca, filing in

disease. market. global believe lead probability patients, and eplontersen late these of successfully event the positive CARDIO-TTRansform much patients earlier diagnosed including to patients study that with increased Since date. of in of demographics are in ATTR progress increases XXXX, continued we to study being are outcome patients course patients. published will study result is This CARDIO-TTRansform, increase we've best in goal with largest enrollment with our Additionally, disease dynamic longest their a compete blinded the This rates monitoring target the new methods. greater less landscape severe scientific extremely fully and ensure cardiomyopathy in this delivering We in growing patient initiated to awareness study, highly we further positions this the community. ATTR-CM and and improved presented with pleased detection our data entire new the our on X,XXX of of by of evolution a from disease population Based supported the findings, and as and in disease reflects careful along

Importantly, from track of data XXXX. rate report on the to patients, enrollment half added and based in on power additional of the current first we our remain

high well. or pivotal and mid study the triglycerides with is the our with future database data our safety population broad study advantage, in progress remain growth. much indication also olezarsen With Our is same to progressing confirmatory III track very XXXX. larger ESSENCE remains underway first expected as We study. program SHTG patients the clinical is in program continues a Phase supporting we severe our broad and initiated now planned out substantial study year. severe triglycerides indication. X, nicely. enrolled also FCS -- CORE, pivotal our CORE in on supportive in ESSENCE in FCS out the fully high larger SHTG, data development SHTG pivotal confident driver designed The be to Phase a read BALANCE the is III also for first-mover to build to olezarsen of potential for of next recently

III data on our hereditary program patients in for also OASIS with in Now XXXX. angioedema track remains donidalorsen Phase

pathway. by safety Allergy, Sunday, We demonstrating Nephrology's to nephropathy long-term reinforce B XX-hour new Kidney course, designed data IgA the study substantial to data the of protein IONIS-FB-LRx XX%. reductions profile alternative in to patients from Week. Sunday. Scientific the OLE recently The in further treated mean urinary data in Annual and Society programs clinical of reduction of X a along in November targeting met forward data complement potential by to HAE report complement Asthma on evaluate partners Meeting. goal safety factor of with IONIS-FB-LRx IgA clinically with of treat tolerability of tolerability. from the point longer-term for donidalorsen's attacks of sharing best-in-class and American and this demonstrating College is protection Phase competitive will pipeline. mid-stage nephropathy primary XX and from the patients positive study Immunology This II to year. positive study advancing meaningful and presented the and several we with look our American end our at from the demonstrate proof We with longer-term our Phase patients IgA potential concept at II for OLE OLE was and with We And you Recently, rich our presented nephropathy

goals achieved and major dose compared in at that dose-dependent Phase bleeding or maintain Also the at EGFR Based risk Kidney and no bleeding the III medicine on to for no study study. patients top outcomes. were end-stage compared thrombosis. IONIS-FB-LRx effectively reported study. plan mid of for increase substantial with of treatment ESRD steady in placebo XX% also to fesomersen study Factor the of tolerability that XI next nonmajor were Week, levels XI IIb the exceeded with dose-dependent by rethink and positive and a Factor development Factor bleeding demonstrate pleased of favorable Phase Roche reductions disease. throughout the the advance XI median reductions We year. LICA patients their and their achieved sustained The in all fesomersen Fesomersen in this, to were Bayer exceeded into state tolerability and together to data, already announced And imbalances Additionally, with levels these and goals profile. safety Safety in or key renal positive placebo.

significantly diminished were decreasing Additionally, the favorable molecule return clotting XI Ionis. and circuit positive to which Factor with in of to tolerability and efficacy levels, III AV to Despite decision fesomersen the their thrombosis safety Factor and Bayer these access highly therefore, exploratory results, small XI incidences of Phase medicine made and endpoints advance only both fesomersen demonstrated dialysis and study.

and we support our move drug decision second fesomersen positive data understand into advancing Factor pivotal XI to believe a advisers Bayer's studies. forward, not While clinical that these we

partner, we soon deliver large patient of potential hands into getting potential on are the the numerous and to to the the as as focused possible. market right explore, Given addressable it to indications fesomersen population

Turning with B a lower program safety can limit bepirovirsen of track inhibitors plans outcome for the and now coming in a We're robust chronic achieve goal study nucleotide nucleoside in the reduction advocacy. it hepatitis nucleoside weeks after studies DNA year. and CLEAR Based of of patients the we to key B. treated be deaths data these the This with potential in treatment next of in Sunday, to momentum that and and the catalyst-rich The was the the first presenting X% alone tolerability of we the alone bepirovirsen. bepirovirsen first X% These antigen data for treatment. Phase in the results of quantification eventful GSK viral substantial to strongly with of half for the year, the NDA discussions efficacy, the regulators III primary time antiviral approximately year with nucleotide measures which Well, assess surface study and continuing currently was III viral support achieved demonstrated data a ahead. an data, to treatment on show that is HPV with because by with infection. HBV exciting HEE living both cures end this forward an at transformative together sustained at people HBV been antigen, it people to suffering of on year has initiate reported HBV of deliver remainder and surface Phase or of the the Phase Bepirovirsen design is upcoming looking patients important encouraging The on for patients in the annually. XXX,XXX bepirovirsen GSK AASLD. XX for anticipate are our file is year. responsible are or IIb the HBV and bepirovirsen and potentially eplontersen to the on on in chronic DNA with combination bepirovirsen analog functional

III approval of year, Next looking of the initiation potential of the in more the FCS including FDA year, catalysts, to the the year, XX-week I new olosarsen Phase NEURO-TTRansform key first forward tofersen half eplontersen of along potential we're data eplontersen of programs With to will III Beth. advances. in X that, Phase among study patients, over the approval other important and both from the later call the from with turn data

Elizabeth Hougen

the results, approximately estate and morning, and $XXX full a guidance. recent year third our and touch you, Richard. for XX% year-to-date, $XXX our third then quarter Thank revenues of our million earned increase summary This We I'll real provide last on quarter transactions and million year. over of discuss respectively,

from be numerous Our diverse revenue products sources approximately from from continues the to partner balance derived our and programs. marketed half with numerous

reflect our our readiness approximately of year-to-date to with for were sales maintain we staying billion and steady billion healthy third quarter investments at pipeline Our Notably, $X.X sheet year-to-date. the the investments for balance $XXX continuing rich commercial in global operating QX. the through and expenses cash end and and continue $X advancing SPINRAZA's quarter our activities. million

result, corresponding sales SPINRAZA the million revenue As and third period. in we to continued earned $XXX stabilize a for $XX the in royalty U.S., million the In quarter.

to rates. Additionally, see trends we in continue discontinuation positive

see the we $XXX This path and U.S., outside million RESPOND compared is to which for further and characterize neurological which remaining We and DEVOTE the SMA life includes continued our advancing to to $XX same robust expansion year-to-date, last in need Biogen's both potential program. as in substantially year. strategic of growth. programs revenue their Biogen the the period SPINRAZA's earned their studies. million we ASCEND, to with markets the expect management R&D the Year-to-date, undertaking earned quarter return of collaboration. We return a from $XX unmet disease play million is dynamics geographic of for SPINRAZA same by growth Biogen see third increased medical patients to with under to to further supported work out cycle

also $XX portion their $XX costs. licensing from for Roche eplontersen's and for and of IONIS-FB-LRx million development from in payments earned sharing cost We advancing AstraZeneca million

conducting expenses with comprising we X compared operating by with was The advancing year-to-date in in studies largest Our are R&D third increased higher non-GAAP eplontersen were year quarter the and increase currently investment. to III the our from and driven expenses Phase last expectations. line

from to affairs and savings as the up last substantial SG&A our Our expenses year commercial SG&A below also CMC to opportunities. increased the period preparation. support the activities expenses integration our we same included in go-to-market spending continuing medical R&D Akcea third quarter transactions. we and the increased near-term were to Sobi for Year-to-date, year expenses realized ramped last reflect compared

steps our entered medicines As future the lease our manufacturing support Brett bring chemistry. into to to increasing mentioned, and We new capacity we recently state-of-the-art a took to our construct long-term X a growth. significantly important facility, to to expand into market

the and sustainability incorporate oversee features. will the and will ensuring environmental of design construction advanced protection it facility, We

facilities expect million. $XXX expect early us power sources. we XX% this example, For that to renewable approximately in on cost come our will design new energy work, of Based requirements anticipate We completing from we it project the approximately XXXX.

product initiatives, substantially for to science advantage We multiple balance By our our to believe also In pipeline also used cash proceeds technology, entered transaction properties. into for we facility. these our manufacturing preparing demand It doing expand proceeds, estate launches growth operation so, can putting this of pay the bolstered portion the of our our off net of control mortgage and our We a existing long-term our debt. to plus sale-leaseback in reduced transaction, real and our value the assets. work, over This advancing a with new enables and campus. record which life on high-quality we funding retain facilities. R&D advancing including took us cash approximately generate we $XXX building million expanding more to

to us QX net expense meet loss revenue, guidance. and keep Our results on our track XXXX operating

expect We than continue revenue million commercial revenues of $XXX and roughly to XX-XX. split with more R&D

opportunities last in III to preparing near-term SG&A studies year, we year. market. expect in while this line to Phase to to year our XX% expenses our the expenses compared we between and increased our our R&D last continue XX% is recent the leaseback non-GAAP sale to our increase progress, guidance, As total, even bring project And to $X we investments of from be We at operating contemplated end transaction are not our billion. which to increasing $XXX guidance, to cash lower million our in our million. come $XXX XXXX between In since billion and was our our expense expect we $X.X in guidance with

in continue to deliver advancing expect to that, financial back Looking And we with to to strong XXXX, over beyond Brett. future invest And priorities, us are to the serve our significant as we we well. the turn strategic call poised growth. future our I'll to foundation continue

Brett Monia

Thanks, Beth.

in the the NDA this toferson our file X of to with progress assuming end and great And transformational and priorities next priority FDA, currently diversify to made of year we morning, we're the on expand this by our eplontersen technology. We're abundance our to this advancing deliver with medicines to track review add these our new pipeline, year. to market key grow summarized an medicines under to commercial portfolio commercial As positioned approval. the new year,

Our late-stage to important expand technology our additional and to come. and our extend made more years and advancements to our advances pipeline still readouts support data come, to taken next year continues with many, III expand growth. Phase diversify We've steps for setting advance and many to to itself we've

priorities a even Importantly, on for that, forward questions. Ionis the on are in drive come the if and I so our resources substantial more for our to With we could the be now to great the think This up executing sharing better. for stakeholders. coming looking progress all continue years you value to We're months. open Operator, year have next to queue, been to has open far, please. and year call up going the I'll

Operator

And BMO with from Capital Gary will Instructions]. Nachman the first Markets. [Operator come question

Gary Nachman

expanding previously? was is of explain additional NDA earlier-stage information. the you panel just eplontersen, anticipating just in of the those you bit of duration first cardiomyopathy how you the that the all you what FDA data? extending you asked did might X how change and confident if having more were on too time And to consider at beyond the in So there for study and you won't then still be getting terms preparing in Did are explain And if on So what the Biogen little study. a discussed just a are rationale And any patients? many guys panel? for that, further you're terms line why just seeing What extended -- think that's a the for first one. months, the if

Brett Monia

cardiomyopathy I'll stab eplontersen thing, at the then question, behind over Gary. take a Maybe Eugene rationale Good discuss to the tofersen protocol it reasoning the to and turn I'll vertical.

We're need has just eplontersen, the primarily an line the communicated this And XX, them April lot especially there time X-month No on that the to more not on to ADCOM. was data. major in forward for more It caused fall the that, they from review data much needed a the take the very the nothing the to the data to request by the FDA. issue review for really really substantial going yes, not for new we for additional division, time that to and or they any neuro to do a time a review similar amendment, significant probably extension suspect, -- all FDA by set. delay, the They NDA the looking readout, indications, to bandwidth It's although date have data the past outcome FDA the a decision, on PDUFA of was position review. so because definitive principally. have

the change point, well. anything amendment looking they're We're Eugene, you to the don't heard very much rationale the planning us and an take TTR? as com cardiomyopathy at some importance to new. have that and Phase for haven't We III ad that trial the also of through to forward why assuming we're still

Eugene Schneider

question. the for Thanks Brett. Sure,

remarks, population been population study years. you study, ATTR source the over for in power that a advisers us clear a of the has his to XXXX several we've meaningful publications as the changed as a was ATTRACT quite the past with have already we substantially and but which to recall, became And set, the in us well more in importantly, many internally the back that way. cardiomyopathy original to is to that Richard as to us primary years it the So talking mentioned as has data changed the few the Pfizer started endorsed now conducted data back. And the study available application externally, few

to that, than greater awareness, diagnostic in earlier diagnosis relatively definitive disease as to and a they And measures. to patient come be. And to. it of that's a they being factor result used different used a results journey what are there that of Specifically, the

came presentation So ATTRACT powering said, to that study. in late for early attention -- clinical and I conducted, when have was events used original their were again, the patients were multiple as

properly we So study the coming based our we data internally updated those all accordingly. resize and needed to study from the was the of assimilate became study, and powered that as clear it externally assumptions, on not

looked And the would about that against question was it, further the alone as it's the significant well. extending study without readout. powered one, timing taking substantially ensure duration a that an of course, the that felt and important is duration that, the penalty at time. We've even we've of delay considered sufficient Of to because the properly again, upsizing we

Brett Monia

lose add. now of wouldn't have And to so we thanks, XXX have CARDIO-TTRansform also And course, those Eugene. we III extension of just weeks, study, who I'll open-label patients want Phase completed patients. the the in the

So to by can goal size, the the the And is think important the did. really for the same to which for outcome most move we possible accomplish same sample help strategic this that CARDIO-TTRansform. study we a ensure successful we increasing objective,

Gary Nachman

confirm, don't has been number Maybe you sites of going and very the That's comment on just to just that. quite helpful. to increase need guess, And enrollment well. Okay. I

Brett Monia

adding right, the not means we're XXX enrollment. that ahead Yes, well patients the timing readout for changing in

up well, study we're not new existing open we're So sites sites, ahead or X,XXX to enrollments very to going number. the moving needing get same just to and the rapidly and the

Operator

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

Yanan Zhu

progress also the the question study on quarter. Congrats on I on a have and CARDIO-TTRansform a follow-up Lp(a). in

showed that assumption? tenacarcen? outlook eplontersen where your data assumption patients how for view event Novartis in up did how powering reduction, and assume to For effect powering XXX% control in size? And the for arm you terms and the the your you rate do Lp(a) proportion affect antifamidis they what change demographics Lp(a), does changing in the does meeting how CARDIO-TTRansform, presented For at your partner, your of AHA of the

Brett Monia

ask Eugene share two I'll Richard cars. Thanks, assumptions then Yan. great powering and And he And can share about on. about talk to what so questions. to

Eugene Schneider

your Thanks, question. Yanan, for Sure.

our may on related to the assuming Kavita. responsible of So placebo do, recall, thought patients we conservative what -- our all original we and of background to we which assumptions, did be was you was thing will that

tafamidis took the calculations. essentially -- we original the arm rate our event So in or

As overly said, today's rate I to bit what's from just a even what the turns to patients given out seeing be -- been be recently case in quite that published we're different and in care. in general

even the was available So rate that overly relative population today. high to

changed effect. the We haven't really of assumptions our any on other treatment

As the considered use you commented we and we -- clinically them. nor not what any said our we other change our meaningful prepared be not treatment true know, treatment is the specifically have to on assumptions, of than that effect advisers use known say to for what would we're

Brett Monia

And to TTR very medicines. we I'll and investigational position demographics in on course, all Eugene. in the -- Thanks, just we're to a evaluating for applies this contemporary add, the for cardiomyopathy, move but a made study. very and a focused And outcome ensure think study, we're strategic successful strategic change of in studies very CARDIO-TTRansform

this confidence greater in move gives it and outcome. So Pelacarsen, us Richard? a important very successful we even think a is

Richard Geary

Yes.

the all. to doesn't on it remembrance directly a change the it question, to change at Phase get of to goal of So And to threshold to taking X. speaking view percent get just into the belocarsen III pelacarsen, back take -- you the that below does took is of to we our the XX patients Phase milligrams. XX-plus goal. II And for achieved how It's that dose not

a goal. bit that than better, It's the getting below not lower thinking, way So just is is different little LDL. -- it's of a different threshold but

going ahead as patients the the to be thing along into with we're that Phase that XXX% we're to all need There's of had competition we've out and quickly all urgency so gives this III of competition. the and of market us possible. that nearly the get as risk, gets just where well And they product to to

Brett Monia

grow, the need thanks, that Yes. epidemiology you that, way. conclusion threshold continues below reinforcing noting, be worth too, get is conclusion is that to data And that out Richard. enforcing of where need to threshold the probably It's substantial, -- harm's the really be to to

Phase this. regularly. associated, not causal, does to add study want by risk a not to study That large need low are small emerging melecarson too on is Richard publications suing Phase proven, its but go data, that enrolled. low III The significant out. -- risk. from outcome II out also now down, oversight you on competitors reviewed on There diabetes. there the it This That's can way study all have As And Phase close published go a data but III low. patient same also increased for on Lp(a), to that further go reading delivers I independent are population, based there's also you problems. no Lp(a), you said, that study, large the committees II actually fully dose, you bring if oversight is we that very indicating This right? on is drives trial. are which an that well committees, there's some gets to now risk a Phase if very going says study same can

safe Okay, that enrolled and brings We for competition like zone our fully today, the risk to ahead risk. the levels. into patients all well Lp(a) our getting of virtually profile on the read way out

your I So hope that Yan. question, answers

Yanan Zhu

the again on this quarter. Yes. Yes. Congrats progress

Operator

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

Myles Minter

question Just a on quick fesomersen.

oral encourage dialysis, did would to look hitting to the you to looks inhibitor it you for a set a setting. sponsor ESRD chase considering data with the be demonstrate, new in versus show new with to going with more Just of out you asset partner that that everything indications and that competitors your be that acute what sort

Brett Monia

Thanks, Myles.

own were very was with I very year. homegrown ended independent had there monoclonal return last, immersion forward the fesomersen. Phase being Phase eventual the because really Bayer developed for in-licensed going for positive modalities We on I this also hoping we're small XI, based which take we've antibody up transparent really being anticipating II But their think As X think, to over targeting data, preparing positive. been potential positive in-licensed and last molecule, IIb Factor

-- molecule already ESC. data As look of the A been said, at you as I presented has the good. know, some small of lot aspects

I IIb data pleased think the of is with a the antibody fesomersen for mixed bag. and We're data Phase now yet. bit a it's very

believe Factor So And that's too as decision was a that a end-stage be once-a-month to renal for and a that go so prophylactic need stroke knockdown highly renal prevent We prevention, fesomersen. X used to secondary forward. XI end-stage We they treatment could potential effective forward beyond with disease XX% Factor on. made on metro drug focused that dialysis limited. population for XII of they achieves any thrombosis, Bayer inhibitor believe fibrillation,

is partner that said we potential as we're also thrombosis. his for as in treatment quickly be the to in Richard looking possible, what a to as going So has thromboprophylactic used a that's be bring And really partner, statements the for maximize to opportunity that bring, think a to market this broadly. the -- as can but to

Myles Minter

a And study. quick CARDIO-TTRansform on one Cool. then Okay.

are currently versus bolster Just coming the equally subpopulations of balanced additionally subgroups the as into the XXX addition you've trial? to the they might of hereditary, are the enroll, those patient patients versus with patients to like going across wild-type these you seen going XXX the that naive? tafamidis Or X,XXX experience you're demographics patients be want with specific that to

Eugene Schneider

This for Eugene. your Thanks Myles. Yes. is question,

open, what is where doing. kind that of because we we're and a patients that position of remaining all here with the enrolling are to be be are being opportunity that's in portion have in we're an ability intentional clearly, to more fairly population we're of in the So brought the the types targeted sites

As is with regard certain of is hereditary course, to and remainder maximize the on at data, cardiomyopathic trying with tafamidis enrolled you you patients patients, are population. for later we're course, what different said, a compression of outcomes at obviously do what look bringing epidemiology that's that's therapy types the fewer of to the if of risk patients who patients going and priority, of their we're to more be to and here, in trying stages

Operator

JPMorgan. with come will question Fye next The from Jessica

Jessica Fye

update an update? that program? Angelman us the can give we And you an stand? expect on What's Can when

Brett Monia

to we provide. don't a have So the study. whole unfortunately, enrolling We're new lot Jess,

We know, you On like completing data rapidly an studies possible. to the really we right, completed. any as looking ranging. for It's we're forward up And not As We're planning as extension intended Phase open-label III dose anecdotal forward. to want as data, possible. get study quickly And study. it's start as go the to observational it's us as Eugene? set to share a

Eugene Schneider

partner program. it's And a Absolutely. also

really to of the and fact escalation. really in So that say because the the not of on open-label timing nature than plan. early going it's it's the We're commenting stages other according to dose

Brett Monia

Going and according to plan, it's enrolling just along. we're well, moving

Operator

The from RBC. will come next Luca Issi with question

Unidentified Analyst

is This Luca. Great. is for on Brett. Lisa for question This

technologies picture. I a to Just your beyond broadening legonuclotides. antisense reach talked bigger know about you've the past in

was wondering if there. what your latest So I thinking is you can comment

Brett Monia

Sure. Lisa, thanks for the question.

the talk the a providing an brought forward We've update so systemic but backbone well frequent So called to more brought diseases less a for forward durability, looking partners we MsPA. partner we That expect is a chemistry great as do And We've book now target muscle. new that prepared we're to new as X as efficacy, backbone CNS in for at backbone. translate into a today, because to has We making that up a technology well area, this applications. supporting or mentioned Ionis using many new dosing. new studies advancing potency targeting a highly my development open now as also with as I we're to the using maybe targeting next muscle. as point different moved CNS remarks the in to ways. it's as That LICA toxicology targets in year. competitive increase program now indication intended Can't our about -- well progress potential

for Ionis tuned broad that. with stay we ourselves both So to We're one a one wholly partner, owned, expect well. that indications. a keep one They're as

there we on strategies time, we ASO strategies, our for let are other we've them as evaluating win areas As will advantages as continue quite multi-modal far There -- been the specific probably as saying I've tox as next or are we approaches, best preclinical specific couple drug based going bringing for some for here. the see move And first And expecting of development are early We're clinic. advantages year. early we're Nalin data, applications well be years, to last into now where our SI this SI. been we not for SI the year, to where see to at philosophy as and is ASOs. now advantages

our about seeking area. able sort hope As really it the progress that some in platform more Lisa, than much And as thoughts, now except be about I We ideas that, capabilities. make in our can't future about diversifying of far tuned. phase of to evaluation platforms stay to in go, say right some new we're

Operator

Gena question come from with next Wang The Barclays. will

Gena Wang

one this regarding I Sunday. just update HAE the have

already efficacy. impressive see early you So pretty

only expect of attacks? data more improvement longer regarding attack-free also follow-up? point in with which patients will reduction a given be should of So in this versus important attack percentage further percentage data the we reduction And

Brett Monia

launch on what I'll we're patients like for start to the since prophylactic to important standpoint, Onaiza at ourselves commercial beginning the what to and planning for most a from maybe this is drug prescribers HAE. is talk

can't long-term whether data, once we're looking a I hard sustained it's state better. I show patients we're dose. actually of What after tolerability. to -- to compliance mean that right, to attack-free get as fair. efficacy is show Phase I what well better than wouldn't hard got as X after plan get expecting really the as it's these Sunday. get be for mean on But unprecedented the weeks do well is were steady is X to is year, as ahead presentation I -- right? That mean II our to to essentially So the we

standpoint, hand as So those as go an know, tolerability in hand Gena, sustained efficacy. as you from well approval

because I showed think these of can't the from patients? to What important get what's don't are Phase it's in I we really patients and most points, than -- efficacy II. that's mean, one So better maybe percentage or the it but you we beyond. should go can really much say, I to you thinking? Is

Onaiza Cadoret

to to what's would like efficacy I hierarchy say patients, kind both order of tested and Gena, first actually is measure. followed withdrawing followed Then by efficacy by treatment. important rate to off then just glean you're and #X acute we the most efficacy, is, in and and and attack attack mean Yes. the of X foremost, how them reduction physicians of the much

of kind in X So showed you've already Phase attack we of basically X a IIs, the the as -- of weeks to -- but said, game XX. that rates that's Brett seen, those are clinical XX% efficacy attack order we max hierarchy, as name our of in mean the here. rates And the X achieved

again, in prophylastic there, reported. attack and substantial that that's a out We're this patients where to HAE -- is rate. #X used to seeing Also attacks pretty still patients a So X the are first leader as that also well, the agent say, market note pretty and X market there quickly as in currently marketplace, have being On just completed research with average, Takhzyro some with about important are after and we your dose. significant at the in okay, year happening still.

this do that believe that unmet we fulfill to exists still So in going the marketplace. need we're really

Operator

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

Joseph Stringer

the on GSK the quick A program, HBV partnered one pelacarsen.

competitor could say, you therapy, are knockdown of inhibitory talk even from, if are of or based? some dual wondering differentiation combo triple RNA approaches the about that your approach which using Just

Brett Monia

comment to Great to question, ask on Eric that. Joe. Thanks. I'm going

Eric Swayze

antigen study, is haven't the and other single at functional that a think that end the was drug by BCA cure to I able was of showing weeks achieve Yes, evidenced treatment to able the where therapy. ever-elusive patients approaches results, as sure. differentiator really both in the about XX% And Really, for XX HBV, the are to accomplish. patients the agent for post the HBS been and things that around key XX% do then lulu able a potential level the remove that that detection at to from maintaining of the of being this pelacarsen highlights

of great approaches. and And ongoing which antigen with thinking They studies have combination showed the I GSK of is a in is study be to to top think agents this some together treatment. are right job loss, interfering competitive think some end of now, with study and the of you're after that. but I on interferon program adding doing not combination HPV was referring RNA that removal S using about lowering at

happening. will actually moving having foundation can good at cures BEP a ultimately, think the think recent functional hopefully that think field for I I around that to patients therapy be patients. will great remain it's So and that's forward. of -- the rate talk buzz And for treatment is that And combinations It's the HBV HBV, the involving we it free. including that increase BPI GSK and about meeting of

Brett Monia

Yes.

to completing that's X%, that I levels or just emphasize, undetectable functional closed that think the something Eric after is I want XX just XX% dosing. on weeks

beginning. Phase this XX% all the study to So had coming burden that is is HPV greater is undetectable foundation undetectable approvable were patients achievement GSK is approach going selecting benchmark. Once various so as point to BEP impressive. So and looking marketing, It's we and of interferon on really XX% the on immunotherapy that combinations, get treatments and an levels really we off from III we in and know for into weeks all patients. to drugs, even build to subpopulations, less XX believe, range, be or still is end to that's value or mark, beginning. that marketable is taking in But and combinations at the HBV go, monotherapy unprecedented a approach, about That's we a XX% right? later, the in the of alone, patients, whether just maximize combination comprehensive and NUCs into

Operator

with Matteis will Paul question next come The from Stifel.

Unidentified Analyst

one, efficacy would your Maybe launch James to is yourself? Ionis' just it separately, could one so, what first you're just to kind guess, want is It it about be potentially for how that safety you looks like be drug profile just drug Paul. This the And I And on in have a today and of it on there. olezarsen. launch wise to great independent maybe thinking quickly, get confidence if thoughts to competitive investment and like? there would on see then look

Brett Monia

take to that? want you Onaiza, Sure.

Onaiza Cadoret

one. Sure. we indications, be on know. is looking being And the self-commercialization this very of FX a Tillerson X first correct that at the Yes, that as will a assumption cocoa first outside are you

Severe have say, is the treatment severe patient million hypertriglyceridemia. one This investment FCS The patients with where a out the it in done we've then first TG. patient with see and I for and available population U.S. one hypertriglyceridemia and face to a actually for on is they're and pretty seem over into fair these couple be patients, specialties. coming to to go is call stuff. to work going a would patterns would going the of the of referral is close X second are population of amount the it a GPs you're elevated to because focused, large But in of here required that's in large on

where to we're the also large So patients to And lot predictive to of we're tools just going innovative our going to population. targeted analytics this to to there. really able actually are a more be omnichannel very just patient such employ to identify and investment manage AI as get be from

Phase terms X these is IIIs actively right ESSENCE, which pivotal are then and are and initiated a the as we working CORE preparing trials, got trials the now, trials safety confirmatory So all of the well. which in in that trial are very as just

listen, the profile, fibrates competitive of and reductions niacin range. XX% and the the triglycide what's Vascepa XX% and were are to to their As standard care in

Xx expect we're more do -- doing we of about to to X.Xx So that.

our a We were elevated in XX% range. TG IIs but based confidence And a that we'll lowering. on the Phase really IIs have showed a high substantial, reminder, corrected XXX-milligram there They these which, we we're already that, not in real were sHTG. good placebo as get Phase in the

we hypertriglyceride both milligrams XX our at and doses, look So at range, a also higher. the it's if studying severe And we reminder, X we the to the were be XX what Phase milligrams take the milligrams. that in expect IIs a as original going you expect in are XX

substantial So have we are mover well here a by competitive over the have very and And competition, advantage a of a a ahead we we margin. first reminder, profile.

price the So market the market, we'll prepare shape and really get the to market.

Operator

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

Unidentified Analyst

Salveen. This on for is Tommy

data X, such time? question time, trajectories on can over over measures -- you're getting envision so X.X, sharing detailed X Our the mNIS+X at is could as years on benefit that that see these people functional tornado you

Eugene Schneider

Yes.

your So thanks question. for

around all pretty a on wholesome points thorough look data the primary year providing anticipating year. update and are key we co-primaries and of end mid at our endpoint, once next secondary next We XX week

once we point, more the broadly more have able until to share said that But As exhaust. and data the we're results not key the week shared will but obviously additional disclosed, study be details. been become XX results available,

Brett Monia

we'll I expect mean And data year. we next midyear by also present to publication that that prepare -- presentation. have

set So to looking sharing forward we're have the the at we time very when the data much data. have it, week full we data XX that when

Operator

from Yaron question Cowen. Werber with next come The will

Brendan Smith

for on This Brendan is Yaron.

from Just one a us. quick

many things for on new And updates so on the timing them know or the or to plan the if just want for data meeting First of Roche, see medical latest presentation that release with the for what whether have But is guys to just just Sindelarson of program to with here. any a targeting wanted thing I not? Huntington's the you and we acromegaly. all just can tominersen advance see clinical quickly, there? there are then overall either on timing thinking and I you're get maybe

Brett Monia

out We'll -- we we're see study. we to still Certainly, medical meeting we're don't rapidly right, is review if a could until data monotherapy as it the of Phase get not what to I -- to And year going the small for be II we're the Richard? the do. we'll Probably the the the present to data II this can. But question. the next because know So coming is able opportunity going data, to as that our Phase a have out year, planning close. get to to year. data, acromegaly relatively to out data try It's program, quickly going

analysis laid with we're we're compelling frequent a doing of hoc, II post-hoc doing data has patients design, Huntington's, there's job it's a detailed. XXX better right? pretty Their disease at For a trial the of good Is dosing on design patients done but in And cohorts, And right? also as of their sharing Roche per Richard, cohort, lot trial burden, looking they've less that with pretty out I'm It's about recently. placebo. post better. less doses, study, tominersen. Eugene, that X Phase recall, X X I It's big

Eugene Schneider

right. Yes,

Brett Monia

Yes.

timing but They on given it's haven't first it's patient out So there. a not exact study. far big dosed

Operator

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

Yale Jen

the well year road, pivotal III as study, later to to we anticipate GSK program as year? the payment milestone given able they do early to this be For are Phase or next advancing forward company any the

Brett Monia

asking about HBV program, the GSK You were Yale?

Yale Jen

as Yes, as FTE for the Roche? the well for --

Brett Monia

property. the and FP the It's

Onaiza Cadoret

Yes. Yes.

So milestones approval then milestones regulatory year, with same milestones, that think that on in study as drug with second we we've license will forward. be I earned the the milestones of with to the quarter looking and atrophy geographic to this already for the fee and the the continues IONIS-FB-LRx. third forward drug advance going And potentially

Yale Jen

for Okay. That's the here, treatment-resistant is very helpful. Maybe the question one which HGT hypertension. more for

a color Could You later a data what bit we to expect this readout. more have give year? little will

Brett Monia

Yale. Thanks, Yes.

refractory We We have molecule. with like have progress, a X hypertension the in right? three studies Gen

know the pieces, X full next patients. another is pull going announcement studies and We're to this hypertension probably we once probably some process. next we're in have X it both all really will X the are data, data until we what want to We or have study present next early the well from if We And the set in at and the presentation get data. are together present do form to to year. Gen And as what steps, at all right? going be in the the it next also X.X drugs We're steps will data as look heart we still indications. in have be don't which the the both probably or a indications we for question that data because failure look both drugs with Gen in lycomolecule, process, the going we

least really we'll studies you. data have all Yes, joining the Thank everybody, this for the are getting us -- your complete in Thanks, probably out. to those certainly for of completed studies one it out we'll for year, So we're at with to there. going participating but forward have as looking close We're the into well continuing pleased a quarterly as really on year have updates day. as the progress provide and to very we're to and year We're great you the moving of with with the call. much again, rest well results. today's Thanks making. next

Operator

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

You may now disconnect.

Ionis Pharmaceuticals (IONS) 9 Nov 222022 Q3 Earnings call transcript