ARWR - Earnings call 1 2024 | Arrowhead Pharmaceuticals, Inc.

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Operator

Ladies and gentlemen, welcome to the Arrowhead Pharmaceuticals Conference Call. Throughout today's recorded presentation, all participants will be in a listen-only mode. After the presentation, there will be an opportunity to ask questions. I will now hand the conference call over to Vince Anzalone, Vice President of Investor Relations for Arrowhead. Please go ahead, Vince.

Vincent Anzalone

Amy. you, Thank and discuss everyone, quarter for us joining December Good results thank for to Arrowhead's XXXX. first a today XX, afternoon, you fiscal ended XXXX are With from President and us management CEO, today Dr. an provide will who Anzalone, overview the quarter. of Chris

of Bruce Given, an We previously Arrowhead's Chief interim Head R&D basis served and rejoined has Scientist. pipeline. who programs, financials. update give Translational James our Dr. and also Discovery on Chief as update Officer earlier-stage and of will Dr. who our our Operating will the Chief as cardiometabolic back of welcome and an provide Bruce an provide Chief the our company a review will Medical on Medicine Hamilton, Myszkowski, Financial on Officer Ken

our be Operating Counsel, of Chief General portion Q&A O'Brien, available the call. and will the Officer addition, In during Patrick

Before comments those I Section statements XXA that XXXX Act to made certain statements. remind the All of are during of meaning statements and we historical you in are forward-looking to of would Securities fact Exchange contain Section other actual uncertainties materially statements forward-looking could of the than statements of Securities within results differ from XXXX. risks call any begin, like the XXE cause to forward-looking today's and that numerous subject of and expressed Act

Form CEO quarterly on our Anzalone, President filings, further Chris annual For reports company. refer the of please I'd report and details call the Chris? concerning to these most turn recent including our to Form uncertainties, our SEC now risks and to over and on like XXK XXQ.

Dr. Christopher Anzalone

value complex stage we build that the vertical. up This have now, to data commercial building we follow cardiometabolic mandate expect engine as company value products proposition.We the low, to pipeline later-stage and grow to we clinical and considering This as a with of is clinical-stage spending prioritize us deep particularly durable mandates large we everyone the for to medicines marketed commitment of a These expect potential or we pulmonary capable name mature. RNAi things candidates teams infrastructure. pipeline we when our the requiring novel in on to we as at where dual and medicines. exemplified initiative, we is of commitments late-stage patients relatively expertise.However, least of And the to without afternoon, is value make our and and to delivery out you our for we maximize discovery domain the a are lung-targeted risk as cost are targets create at development Good grows capital, currently we infrastructure, creating to platform development our where ability by diverse decrease year serve do many during and do near-term and therapeutics building our that our biology XX and money for platforms.At and we joining 'XX our Vince. is be number XXXX.This innovation XX We development. add broad-based this area without That large today. early primary number to help new by and business. Arrowhead Thanks, and infrastructure a short, our enter thank rapid focusing expensive has building made much quickly and can maximize our can deeper therapeutic early with to need a of studies a need too specialized shareholders. the expertise, speaks vertical create well-validated can out itself what this to internally. can proven by commercial significant ultimately and commercial aligned stage, entirely as are we of In to development can

So not. early does It development stop our outside will focus down does areas? mean this that slow or we

verticals part our to could to areas that and, as continue noncore to these internal to adding and A, in outside ability create business capital farm and develop support as B, development commercialize We in partners will us, platforms the long-term generating future.Think new our are mature. verticals additional continue this of value, candidates we candidates for focus programs to a because, and we programs as appropriate that confidence anticipate new find of have system our create

Let's our with cardiometabolic start vertical.

for launch the the could milligrams and tolerated from significant often generally visit which patients last of data second schedule the the last to our and file our that Plozasiran is triglyceride would first experience recurrent Our Phase compelling. cardiometabolic of year. C-III quarter patients X the pancreatitis, for bouts the see first Plozasiran allow to represent have potentially Plozasiran, per X lead study FCS study inadequate what this treatment and data would severe severe APOCX. year or syndrome FCS designed of well do. broadly.The and in or potentially big their Plozasiran which patients it the in as believe thousands Phase in targets with Apolipoprotein Arrowhead is have extraordinarily did leap set chylomicronemia levels, of product. Many options, a consistently we Phase NDA clinically first program studies patient deciliter. was genetically been to is This a high be Plozasiran PALISADE painful hospitalization. and X vertical, have on a has These for commercial confirmed and that forward.We or This in abdominal disease these is pain, patients of familial us for complete

likely do this We addressing patient X will studies also severe a next for a SHTG. with of population begin confidence population powerful have with and needs. high-unmet could degree broader million a are designed we population that will initiate forward X quarter believe for SHASTA-X it high our Plozasiran exactly us what We very at studies in a do. to totals SHTG medical patient it in or in designs to study We the to help will drug patients. discuss Plozasiran US this plan a population, a be the welcome Therefore, Phase that Bruce patients. leap of X confidence hypertriglyceridemia million believe powerful be to is These will patients for we with will gives alone.As moment. FCS believe aimed that study larger

cardiovascular also final ASCVD to considering still made Plozasiran or decision and broader disease, are but we the have population, atherosclerotic not We a yet want that. whether on study

reasons.The some we will is foundation this APOCX vertical will for completing variety interactions. We of analysis well-validated communicate are have after our plans target the cardiometabolic studies. regulatory our If the bedrock value our had and the of across proposition, is genetic quarter of they represents our foundation finalized be a and that following Plozasiran

with There more APOCX Our elevated need healthy tolerated an FDA-approved lowers up and data generally set currently patients and silencing. has and get to XX% deeply no were abdominal Triglyceride just XX% and in we even, know other by populations well can treated than difficult downstream in rare been Plozasiran engagement is or attractive opportunity severe triglyceride certain triglycerides across hospitalizations volunteers therapy and durable studies.Together, that to patient pancreatitis, levels cases, prior to effects, these pain, hundreds deaths. market. target levels reduced with human consistent deep subjects that in lead indicates of in We Plozasiran. acute

larger angiopoietin-like as even into launch that to to early follow expect relatively hope quickly we in ASCVD we by This me next ANGPTLX. to We follow We proteins which if launching SHTG targets the markets with as brings would year next or see and Plozasiran Zodasiran, that FCS. larger X will markets.

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being currently have If regulatory we end-of-Phase intend X are out and will to in populations an launch Zodasiran commercial a of begin similar much FDA with to path support HoFH study could for additional to ASCVD, rare X plans. continue HeFH Phase a and on while in heterozygous population. Phase materials to plan conduct are is patient Plozasiran. commercial population the our after meeting study to the That feedback We follow larger likely preparing infrastructure a we decide plan into as the and X We or studies Phase in capabilities the larger expand build a conducted. X also

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vertical medicines are that expectation the it's pulmonary. sales We United prospect only and and various currently organization and and expect have chronic three about lung Zodasiran, States, is we Plozasiran commercialization next and support studies late-stage of future specialized with opportunities in the clinical programs, collectively growing candidates. The lung high to respiratory studies we in attractive disease, success of diseases.We believe There addresses clinical pulmonologists surrounding drug in commercial opportunities a commercial in near-term XX,XXX programs invest inflammation, an that of build a pipeline three disease. muco-obstruction, to components a have address interstitial major to longer-term

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existing So future for mean what our rest and does of the that pipeline.

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One, into. It clinical the a verticals. warrant expansion is fit categories new new programs, role good These could we in four in vertical. play cardiometabolic For each candidates existing CTAs. think pending of into is do example into slot that new proof neatly candidates question a fit clinical promising where so can fits this. the vertical. our of three candidates XXXX, programs nine and primary of what nominated they business?I are and example, strategically new the new an concept into do that filed that new ARO-INHBE Two,

important be CNS capital may build and into commercial program. year. of which a important but standpoint, still was ARO-LPA the what with received seven clinical going a a broad as past recently Since good given target-rich existing an in milestones is with from was but capital, receive It over viewed and are Amgen is data area $XX payments deals.Our should brought receive investors. the equity three, a when only publicly when million work do in forward medical mature Amgen late to terrific example such this verticals. not we development step cash In commercial Our we very increasing source the over-subscribed do certain other to serve the result. clinical candidates vast We in that is a increasingly potential support billion is source million month, substantially first financing and saw the we one this and categories. around first be an a confidentially formerly substantial handful to from early and we after are plan half we will in close an environment, still X transaction anticipate and ARO-LPA early, done was modest it. the is in this years. unmet important sheet. piece we to in achieved.In high-quality could another the substantially not million we XXXX, of us participation eligible dollars years, our we eligible Phase have approximately and could out, partnering pleased in partnerships can as first deal fully of preclinical of a as needs an We new as a And just for This new marketed fact, we Olpasiran can and guided, $XXX four then, continue That even are capital in Business category discovery. of on Olpasiran rely investment of on.Last we of to partnership will interesting balance course financing, enrolled, partnered with source fund $XXX fit study Amgen, our the but medical were the our announced in two funds, have and of $XXX be million we to are

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our programs, assess We're targeted move and for and HZN-XXX, after Xb The to and muscle path is and not by forward ARO-CX NASH ARO-DMX. and the Horizon continuing being which programs Amgen us returned its ARO-DUXX Xa forward. clinical our terminated Phase We for to was will Gout ARO-PNPLAX. our ARO-CFB, clinical are candidate, moving studies studies complement acquisition, candidate with designing

ALS forward. SODX against ARO-SODX our candidate will In CNS addition, not move

a CNS a of work while target new clinical serving to are candidate programs concept commercially than later against this year. on still We more additional for attractive is good platform. studies and begin different continuing proof CNS It as the expect a ARO-SODX,

programs. some review undisclosed portfolio affected Our preclinical also

over moment, reducing revised in million. growth will reflect guidance talk by this reduction operating budget burn year year Ken fiscal our fiscal are of in spend beyond. we $XXX an anticipated specifics about have but and a to We our on approximately our

We exercise. and our overall that and anticipated continuously continuing innovative importantly core assessing are a new pulmonary fund uses capital these achieving course, ensuring business align verticals the to is, they with of our while programs, critical and estimates, goals of cardiometabolic of align they fully and technologies the sources and

stronger to budget Given. the our Dr. I Bruce? puts I financially. in now With us overview, strategically to revised Bruce like well as think would turn that a over position as call

Bruce Given

and Chris update and today possible. lipoprotein the operating design helping be up move different in having Phase breakdown been made. an good to ensure population key levels Plozasiran can mentioned production and of multiple clinical take designed additional APOCX metabolism. great TRLs, are are studied process we've have the with TRL up efficient centers reduce increases high are patient important into analysis a will you, a time and ASCVD rapidly. Plozasiran with of will of and on which liver.We've dosed. middle triglyceride a It's by a triglyceride are my to in hepatic work back I've doing speed we at TRLs To APOCX, to level.We Plozasiran populations, remnants by new studies Chris to and Arrowhead our component forward the nothing clinical on several regulator is where receptors inhibiting lipase of with studies front uptake today, in hundred by very I we in been Thank amount Zodasiran and we triglyceride-rich plasma world-class. focus way of patients so of Plozasiran that getting so to a to which or lipoproteins review, good most progress development and everyone. get forward program that running are afternoon, X teams, effective cardiometabolic doing getting work of we want the to studies initiated we or assess be in Shortly, the

encouraged and treatment-emergent encouraging the underlying generally with This of to our study each events adverse safety our with leverages population. been by conditions reflect TRiM and proprietary platform. consistent consistently properly results have comorbidities and designed therapeutic of reported date RNAi a that expectations tolerability that We is

Plozasiran patient also what maximum where First, diverse of and proposed is continues consistency a new to in the Arrowhead short making regardless candidates to last AAT high in space, term, around the take, we couple This is give and consistent Phase with a our X In candidates months? reduction RNAi a of the or and XX%, the the hallmark has as of of demonstrated the we was level developed changes addition, the case in suite the of going, the days expectations earlier activity pharmacodynamic our changed and speaks SHTG. HBV most in This critically, APOCX for studied. have to mean with genes.So population mechanism. patients some with of and Plozasiran over be targeting are design studies of SHASTA of has

with will discuss changes, I moment, is a goal these Our in which twofold.

approximately up to we design potentially but X SHASTA-X XXX greater be abdominal key First, effect speed to payers.So treatment. probability lowering of the to XXX with maximize the differentiator studies, pain to milligrams triglyceride-lowing unchanged, a And the as X SHASTA-X other after to therapies value filings from towards several regulatory remains Europe similar endpoint plan was primary a as triglycerides conduct and aid time want NDA studies markets want of and and we we Our other with show pancreatitis, and deciliter discussions with streamlined US the general two of access to what combined on submission patients in doing enable quickly enrollment US. two triglycerides which could are in the accelerate This significant than severe and possible. features year and have could acute in across per second, Phase study in ends? those and largely we an

important correct.We likely details to abdominal begin this called things. to to removing studies. separate more and higher This SHASTA-X with handful PALISADE acute thereafter. predefined SHASTA-X the This high levels plan. for and six SHASTA-X chance FCS. these show We of or Plozasiran is the severe pain, a clinical triglyceride diagnosed go. with for hospitalization, believe a of separate at study fatal.There treated patients of predefined acute triglyceride-lowering will second, number X abdominal study it inclusion showing reduction is criteria chylomicronemia of risk adequate expected We and gives these in to others, be include full can advance, predictions do process, risk pain studies. and The completion power are we and PALISADE in each study high best development and also of Phase is an move with working these of initiate endpoint of further of clinically the a This in primary SHTG placebo. are in adequate months episodes genetically next confirmed of these with familial versus will for and It of the disease the Between a patients separate and pancreatitis, rapidly on design, prior at more assure no Plozasiran to treatment estimate of or quarter effect can we patients. Plozasiran. one-year is number and and severe patients important syndrome events original a best get Phase for puts and study. two SHASTA-X eager risk work significant intended its collection, goal, we and be pancreatitis to and filing expected NDA strategy and we but getting through readout events, SHASTA-X, we're event results May. and the an quickly sample changes than top-line work purpose.This last high-risk report to analysis After on data. the to patient rapidly XX us to of patients way their the our NDA. next visit, characterizing This SHASTA-X time, the scheduled to we and placebo, if SHASTA-X This potentially we visit third estimate in be now reduction events the analyze in to and patients intend goal that these potentially milestone first of versus specifically big remains Arrowhead.I'll the has Arrowhead's enrollment speed quarter will an preparation a end an the call turn of over begin from to to the run Hamilton. study be is of or sizing, back faster at into is data this last to studies to and to SHASTA-X when That that could that designed will in of part enrollment patients the I'm the for year get ten is will the quarter ourselves more the and thrilled We first next extremely XXXX. a options enrolled of provide This have study. data actively We step X shortly demonstrated James the complete a globally, severe study ready are James? Dr. best-in-class for exciting toward these be is is that potentially

James Hamilton

Thank Bruce. you,

are you we the most early which of of haven't are give programs I on know, more and about want pipeline some that newer and robust to even programs, discovery-stage we pipeline where talk the of update we a As few of clinical programs clinical-stage robust an of programs readouts. disclosed have approaching yet. with a very

diabetes serum-measurable on is will I ARO-INHBE. year, levels the about circulating INHBE by INHBE function genetic adipose databases utilizes variants tissue obesity is of liver-targeted This so in which Activin are later to adipose two Chris our type-X program and called intend Activin in mouse reduced liver-targeted pipeline.One conducted is identified siRNA a waist-to-hip gene protective XX% and fat. controls. by program adipose the platform. utilizes and where focus new human to expression programs with our the our cardiometabolic compared in INHBE synthesized increased studies excess a protein, added Increased platform reduced other delivery signal a E primarily over weight talk gain reduced that to models for store is of loss associated INHBE more and as program. the codes that the ratio.We've First, silencing to are obesity with We mentioned we've liver-targeted platform nutrients hepatocytes. new visceral fat in E,

therapy the Importantly, interesting gain due think in difference difference seen use lower ability dose in INHBE include silencing and therapy. GLP-X a of gastrointestinal therapeutic with adjunct primarily prevention the regain in to mass, weight that the might GLP-X loss, of which cessation potential reduced in mass. to side changes benefits of fat result no or GLP-X lean lean to an the We agonist slowing could mass was hope effects agonist be agonists.We post of combination could reduced weight

We have on end clinical to selected a lead file the by CTA schedule a and are XXXX. of

of to therapy. and these and or for ARO-DUXX of is as promising ascending on treatment two an regulatory is of onset Both target double lead doses. begin and has expression atrophy target. of suppression patients this patients expression homeobox to both our common last the autosomal-dominant to there studies encodes patient is reduce Phase-X/Xa skeletal complement treatment Moving patients FSHD modifying a initiate dystrophy to ARO-DMX two designed to the factor give tolerability At dystrophia as of safety, I or the ARO-CFB maintain Both CTA is status evaluate expression DMPK designed been PK/PD a with in protein can various for and Phase of expression ARO-DUXX we clearance gene and studies on human possibly DUXX to year. ethics ARO-DMX study for single FSHD. is potential myotonic are in the muscular to facioscapulohumeral the X/X DUXX multiple geographic and for kinase DUXX of identified adult muscle-targeted diseases of dose-escalating a QX FSHD, GA. and B, first filed programs a programs. is in the programs, ARO-CFB for dystrophy, which disease muscle To and update with muscular differentiated Type-X profiles complete currently review, expect degeneration. to protein Over disease muscle. myotonica that most have reduce epigenetic dystrophy approved of in gene. DMX. a or myotoxic or the QX X complement-mediated to associated with with no therapeutic failure or also of our we end or year, complement a designed hepatic want DMX is to

diseases Our GA. the liver complement that pathway a ARO-CFB renal anticipate of this such year. in key the alternative B, like which study the that Phase achieve of preclinical We activation durable can occur in of demonstrated first in role deep X/X conditions and of pathogenesis plays have factor patient QX reductions nephropathy, complement as involved in IgA other studies production in as well for will the

complement more advanced program Our is ARO-CX.

to frequent pharmacologic hemolytic the effect IgA administration. tested highest supportive dose dependent of a As X tested, on with at of mean glomerulopathy. AHXX, is in to volunteers, diseases. in us or subcutaneous X ARO-CX and to production made study, mean you Part dependent recall, or pathway Phase data demonstrated presented promising an ongoing CX alternative CX that These XX% a may reduction marker study as various the CX activity confident with results We for healthy dose complement-component serum previously X results XX% the of move complement-mediated dose highest potential following of Part therapy quarterly from a reduction complement X/X with reduce dose in a and at of less in duration nephropathy reduction dose designed patients the reduction

that present We and study around year-end are part intend data patient of the to enrolling XXXX. currently

of receptor are year. pulmonary stage schedule inflammatory or for to reduce continue potential pulmonary follows is Lastly, to all programs These a products which as and are expression as designed the progress three clinical for end RAGE ARO-RAGE, readouts on for advanced treatment glycation the clinical this efficiently programs pulmonary diseases.

study, fully of X/X moderate the well. Phase should by the both the first all For patient cohorts end to and these. enrolled we healthy the PD volunteer additional We have for of dosed mild quarter have and cohorts data asthma as

We lung. Type or these FeNO cohorts asthma in will sits Oxide of as inflammation We cohorts of are an baseline which we own the the in IL-XX biology for also studies results inhibition from patients biomarker where three RAGE believe enrolling have impacts process The including IL-X, that Exhaled that IL-XX, levels IL-XB and third year. cascade in IL-XX, Fractional high initial Nitric inflammatory driven have suggested IL-X. high of FeNO, in this is the of X preclinical a of impacts may effects, RAGE potent well TSLP, array IL-XX, as cytokines, with and provide the of quarter it on anti-inflammatory with our

a which readily cohorts enable the over healthy the ARO-MUCXAC, for treatment has Phase which have to potential sputum we designed patients. and PD. addition of in of and reduce MMPX cohorts. treatment we are potential I pulmonary Mucin blood asthma be and is both to healthy and dosed FeNO, production muco-obstructive require time to for potential clinical MUCXAC benefit Unlike and two anti-inflammatory enrolled Both measurable patient of diseases the ARO-MUCXAC, in now programs Ken assessing XAC or effect, programs, in to PD-biomarker ARO-RAGE, Myszkowski. In we initial year. expression in then already are is conducting the biomarkers to available of volunteers next dosed second a volunteers, enrolled ARO-MMPX The and in pulmonary volunteers the anticipate fibrosis will as matrix to are healthy programs will including as X/X turn and call cytokines a of assess and idiopathic studies Ken? or metalloproteinase-X IPF. readouts, which half both designed ARO-MMPX, patient in reduce In or data the other patient

Ken Myszkowski

you, and James everyone. Thank good afternoon,

ended our compared XXXX collaboration reported or with loss quarter with $XX.X to for our salaries, change quarter ended December on existing the cash payments per on recognition $XX.X XX, from the share The ended the period net December we for revenue agreements XX, XXX.X million, was This candidate the million outstanding. XXXX net XXXX. development Revenue was and and and upfront current to $X.XX this of used primarily XX, $X.X both $X.XX activities operating compares per our and by period in stages pipeline advanced of of XXXX period. to ended recognized. in December for for was from average December agreements increased million, Total GSK, compared loss ended As of the operating revenue for The driven quarter XX, collaboration compared the increased Amgen.All million share XXXX ended primarily ended to activities related quarter cash the now by with with while diluted for is $XX.X the been the quarter ended have a primarily diluted shares licensing XX, December $XXX.X expenses key December million lower XXX agreements relates $XXX.X costs fully in XX, or cash million XXXX. prior increase operating December XXXX. million, of Takeda fully million XX, company's based quarter Revenue and $XXX.X drivers for were were weighted fully candidates in used the as research based $XXX.X clinical has million average December the XXXX. XX, development.Net outstanding expenses quarter into later by higher today, revenue shares the quarter weighted during million

have additional would forecast the quarter. expected next to be expect operating cash to like million our we reviewed $XX $XXX on per to our burn cash provide quarters, We burn.For guidance several million and

been think grow commercialization expected of of going efforts. with about has million made totaling the is is about each as footprint which related quarter, so cash to XX% further, GNA to next final bit $XX be as to the the payments expansion advance complete, a to GNA over which after Our capital months of burn operating burn expect that continue our about mostly $XX we Breaking several costs, nominal. million, expenditures we be to slowly forward

million this quarterly $XXX forma accounting be our studies and XX, the cash next $XX our Pro expect investments our million about investments to million. sheet, to and capital year, year cash expenditures December for raise increasing recent advance.Turning We balance as approximately would modestly R&D at registrational be totaled $XXX.X XXXX.

and capital forma the to accounting outstanding I for turn December now shares call shares million $XXX.X overview, will Chris. XXXX that million. XX, With Our outstanding pro the common at were back raise would $XXX.X be

Dr. Christopher Anzalone

Thanks, an five Ken. Arrowhead in primary year This for important is areas.

vertical. expect a cardiometabolic within lot activity First, we our of

HeFH, our two candidates, to and studies our to several plan across readout ARO-INHBE first two different SHTG initiate We candidate. X NDA. the plan Plozasiran adipose-targeted vertical HoFH, have include intend patient file Plozasiran ASCVD will Phase additional also and candidates, additional Zodasiran.We and drug for and potentially in first cardiometabolic We Phase undisclosed an X populations, to including expand to

at Phase in to and one study. readouts clinical three Second, X have we least drug different across plan pulmonary vertical candidates initiate our multiple

to These in Third, strengthen sheet more done Phase of and Amgen and transaction X complement-based neuromuscular in fifth, X clinical studies intend study Takeda, GSK-XXXXXXX and ARO-CX Phase X with continue in And enrollment Phase in Progress continue enrollment NASH, of its continuing to studies and with Fazirsiran GSK. in Phase ARO-CFB. of the we not for for HBV planning Phase to Fourth, potentially candidates, candidates, include our innovating. we X structured are transactions. in studies Phase or our progress with X X Progress X in ARO-PNPLAX. of balance other Phase of business studies programs finance ARO-DMX, ARO-DUXX our move studies forward. development a one Olpasiran, study our completing progress

and Operator? like expect clinic As year. us I call adipose-targeted and open I CNS the mentioned, studies for undisclosed would to Thanks an the to your first this candidate today to initiate our bring clinical questions. candidate to joining for we now

Operator

And with Capital. Luca Instructions]. question [Operator from our RBC Issi first comes

Luca Issi

one Maybe Two the first know quick the is third in I for data quarter. high FeNO, cohort FeNO the James, on here.

I and maybe that Is I drove first patients was us what decision? this Chris data hearing so, Ken, time that either debt. show no ahead FeNO you the about the longer that think planning you under the impression potentially using talking second, of I'm directly the However, from mild-to-moderate then is or potentially that. the were to if plan, And

macroeconomic the is Given this the rates so right and debt the think broader you do point? what environment at much. Thanks adding are, strategy where that decision

Dr. Christopher Anzalone

I'll thanks and a Yeah, one. didn't all-comers we we numbers. asthma patient first of have cut-off, for cohorts, the sure, an In just question. the FeNO Luca, series don't have take was so the mild-to-moderate it cohorts, asthma the

high we cohort, have top in I the one with FeNO. patient dose think

So based data just enough not on. on or That's make cohorts. a to present waiting FeNO we are based the high for why call

Ken Myszkowski

Yeah.

why think at are the So we interest higher certainly lower is it's cost looking even debt we equity of than non-dilutive rates our financing, of though been of than important part that's that have cost they an are and historically, capital, possibility. so

Dr. Christopher Anzalone

that we those of where think I makes the exploring stage I start company, this options. that at think, can And we close enough consider are commercialization sense, to that we're to

Operator

from Tenthoff question our And Sandler. next Ted comes with Piper

Edward Tenthoff

update. the thorough for Thanks

Going year. to exciting be an

what's it's commercial where discussed the Is envision partners Thanks then overseas company's not a ton. you you the do it build-out especially know PALISADE indication, build-out. the data for crossed, fingers this ready distribution again, I data. think on assuming in first getting you or the about partnerships. you've success a would And something start you're seeking the to past, shown be based huge obviously, but thinking As would indication mechanism commercial in for and for And that U.S.? the how U.S.,

Dr. Christopher Anzalone

seeing we going We're to Thanks, are data make X transition. were Phase way that Ted. the data so this are panning optimistic Look, Yeah, commercial we're is we to those really compelling, out. excited to transition into and good. those company look about excited continue The this like a data.

We company are now. not a right commercial

very And imagine going diffuse. so jarring, a it could a from market can zero could one be be of into large that bit

easy we those are, The patients, them know they are in FCS genetically is segregate treat defined where those known. the patients The so who And this to step, take there baby if FCS. way address. are they're get you to relatively I physicians triglyceride market will,

and take say, easy patients you relatively will are those are be address. Again who pancreatitis. we if known FCS, It genetically history and triglycerides addressing is down, of You XXX will not that well step over initially. to populations, one have patients relatively but those

if And a you will, entry so commercial. nice into it's

on the course. As a the and X that That's but patients those of other education continue some may we XXX digging, very those, number, who take go pancreatitis. side, our have above and we'll patients to study, we large we other we'll grow increasing be as of and history those some market take Phase triglycerides we'll of are after ability triglyceride have those phase studies, the not believe, who harder-to-find

do develop ability to extra our to got we've that. so And time

we us to right find we at for the for like SHTG. some and probably markets, to sense SHTG, expect term, least ASCVD so we the for in certain where partners intend and but would that. So that's like, FCS us Longer domestically, it the partners are for to with countries. local with feel local other internationally, I see do would can We'll HeFH in we ex-U.S. handle good find market make

Operator

from Mayank And Securities. question Mamtani Riley comes with B. next our

Mayank Mamtani

for Hey, have questions good on call. team, to Bruce taking back the and thanks our

love structured involved partners Would like your hear you a financing do I said, quick And look follow trial I point months. similar could and outcome, additional outcomes? considerations be the exercise of after percent or rate as believe deliberations you lowering another meta-analysis might informs on reduction So, a might external a thoughts you coming peer cumulative that. any in PRL possibly there up and for doing Bruce Zodasiran, have event that with informing to process. to to you correlation be and

Dr. Christopher Anzalone

than drugs and haven't a interest concept, milligram clinical there's a to data other lot analyses, trials the these per as some good these for these. pointing being cholesterols lot remnant In been in of enough there milligram highly It's mendelian of because just unproven athergenic remnant data and APOB more randomization treating cholesterol they're Well, in atherogenic. on yet frankly, basis. LDL even a of

remnant untestable. pharmacologic are really perspective. Plozasiran And come could cholesterol drug In and for along from been test has waiting the so to drugs it They Zodasiran that this incredibly good a the hypothesis. both you were

that field, reason plenty there, out studies ultimately, paper to a there's And the alone give So us way, particular in company. opportunity. have this an equivalent point their ways of easy hopeful both two we prove because a ability respect genetic they it. of plenty a to address that choice between not they're as of it's actually question.So with But let I to be us to lot think as offer that that

have thing. do We to the old-fashioned

trials clinical to have actually it. prove the do and We

Mayank Mamtani

Could and be also of it. decisions that, no that what's as And questions. significance Got pulmonary broadly XQ [ pipeline you cohorts, and James, Thanks sputum our data MUCX of and And that? this go you talk ] blood heard I cohort, then will patient for of on also you the high relevance just the part the your FeNO go, make to the analysis. for assessing maybe in to look are year? some other taking biomarker

Dr. Christopher Anzalone

I mentioned, programs with level addition biomarker those year, this for effect. the kind assessing the if so additional of like indication the the seeing any that for date in we've MUCXAC an that to pro-inflammatory of just all doing I in of programs. the programs the of think data, Right, to three have in detail we'll pulmonary such Sure. BALF those in piece the that an get cytokines or seen MMPX, the BALF those pulmonary cell an on specifically any measure FeNO, read-outs are counts we just important to not And RAGE cohorts effect are not we're we're other of relevant. do We not as

Operator

of Bank next our with Gerberry America. question And from comes Jason

Jason Gerberry

of you could there's XX% given this it and multiple targets patients higher biologics Thanks. here that a the chance on by ARO-RAGE the to involved set Type to in XX with just do data inflammatory bogey FeNO than achieve about interleukins think lastly, measure. what XX% One taking are derive patients just data to you FeNO, thinking RAGE. against on us for remind XQ, my confidence, what actually is that at comparing trying your me -- thanks cutoff there Can for you guys, about high Hey, that update is question. get you like And variability pathway? FeNO respect X will can placebo be as your and for measure, with in just from the I'm

Dr. Christopher Anzalone

patients. if we Sure. magnitude pretty think I so XX XX or compared placebo we I the of FeNO we what we that high cohort, to a patients the on The good is are, And can a cutoff higher across have includes reduction and biologics. the really FeNO number. there comment think total have question FeNO can't expect should

placebo be treatment arm So we'll group. able the a to compare active to

Operator

Patrick next comes And Trucchio question with our Wainwright. from H.C.

Patrick Trucchio

a questions. have I of follow-up Thanks. couple

CMO is just on first role. The the

or potential reasoning a you this of the have an bit And on decentralized decentralizing what behind potential it or wrong was idea well just market the just can the going maybe this something what the give and expect as of And else? If out more [indiscernible] talk advantages is you to maybe officer you decision specifically, [indiscernible] the the expected forward? discuss secondly, us the with medical on more a approach. CNS on chief based about impact then as can went how build with function platform, AROSOD-X,

Dr. Christopher Anzalone

sure. Yes,

restructuring slight thought we in R&D, needed the expertise with deep good leaders we look, our that just verticals. So, chosen of with

cardio-metabolic. be to going that's far, So

We deep next to the expect expertise We be vertical. needed there. just pulmonary

and We probably needed the not those going to to have to last their be forward, verticals going several going create.We're they expect we're that just others leaders. own

towards And sense so for made us, it that. restructure to

look, SOD-X, We gave was AROSOD-X. nothing concept it it pushed to appearing opportunity that to there's liked honest. Regarding platform give It us of be it the off SOD-X because chance was not [indiscernible] good increasingly of that or us good be liked is AROSOD-X us within to. thought The commercially is to proof is the unviable. going working. We problem working with we a a

gives make drug it at news thought, fast of news interrogate we of working, viable sense ability good will also the the develop if economic think could be bad gives didn't a but follower, it's that the us at good once platform, is drug. we that proof to concept commercially and had is It platform. least we us the see So we one a the more to

to resources So to allocate it made just to sense others. it and

it of markets. in was So of than a confidence SOD-X the less ALS lack failing SOD-X

Operator

Foroohar our Partners. question with And from Leerink next comes Mani

Mani Foroohar

taking of Hey, question. couple for A thanks the quick ones.

modeling? about $XXX a expenses, maximize lot wiggle you million for the allows I've follow for got And step to at a own year any that up. tempo the over down see to us then course you and flow which which couple this our pipeline room just of quick Could in obviously investment. of talk of on we'll buys into give operating You your company questions the color on chart OpEx

Dr. Christopher Anzalone

So those I think immediately. you results should expect

think was beginning what had could about will $XX normalized in look million, go you second it you $XXX was to spend, the our mentioned and we it back current at quarter, to If previous fiscal to a but compared the before, expect quarter. I like high of quarters, burn that million

Mani Foroohar

gets for And Okay. to hopping a a we terms phenotype, population going however distribution, to of accounts one are as change target the over should weight seen this be as are see studies, of we in cetera, eventually after thinking are or place humans? asset which terms BMI.So, ARO-INHBE studies that et ratio, in should we pipeline, data in INHBE then that in distribution. for some even we what broad endpoints stage about for of adipose derisked? where thinking adipose we're Like be pronounce hip-to-waist a be the it, targets in we've the that's going loss, thinking about earlier to human for what progressively endpoints should in evidence And

Dr. Christopher Anzalone

sure. Yes,

also AXc changes shrinking? look interested happens And evaluate or mass, of mass? all glycemic tissue. oral happens what terms measures in testing. at think and Are in like lean of fat adipose the distribution will I investigate to of tolerance we're initial body just control, visceral What for, but of of to least above, not weight clinical all glucose of stores lean in that at study we'll the composition, our body loss, loss

there Phase about about even study, drug in lot learn quite a our that target. particular endpoints few think and I are a So we that and X can study a

Mani Foroohar

the an and on a the tested how population in given in broadly follow-up, initial are done Great. this see patients who of places where and there set asset amongst And as have GLP-X, the trials? the clinical both human you U.S. guys to is opportunity off taken data other they're

Dr. Christopher Anzalone

I Yes, so. think

that investigates clinic, would then drugs are I the out. not and knockdown we're see little think anticipate agonists be, INHBE those starts the patients first that study the the how on a of ways GLP-X design combination and obese combination a from plays with still that would but also in away

Operator

question next And Brendan with Smith from comes TD Cowen. our

Unknown Analyst

Thank ask this [indiscernible] for just I tissues to to on our [indiscernible] Hi, non-core taking is pipeline which Brendan. adipose of your you. be the want priority? seems for question. Thanks highest

Dr. Christopher Anzalone

you Can up. breaking were you sorry, repeat Yes, that?

Unknown Analyst

pipeline these you? your a partnership? would of programs would And the highest consider non-core commercialization you development Of Yes. for be which priority for

Dr. Christopher Anzalone

right do counterparty are instance. be that a to now, about talk we it that so, Yes, we we be We'd like unfortunately, happy partnered interested We morph could certainly talk entirely. NASH HSD need CX, a about in natural about is right now, all Look, well. talk could for well. be about assets, those to don't happy think as in the discussing and as are but into going One interested bit both a at now don't be now We would GSK, vertical. that clinical assets. in. to something fit That's could right right one muscle happy would we but planned neatly as those be to could that. that's to talk that what that's or consider feels right something least that good for muscle But partner into potentially we would assets, flux our us, existing we the of vertical a are it we think pipeline. to

Operator

Stanley. from our question Morgan Ulz comes And next with Mike

Michael Ulz

taking Thanks. question terms just for follow and excluded going deals pulmonary, this they be Hey, in potentially and potential maybe or here to are up one maybe potential the those or deals? include BD, areas of core from guys, two Could a mentioned thanks like year. cardiometabolic quick the royalty you

Dr. Christopher Anzalone

separately. those with deal Let's

right full are and So, cardiometabolic, we ahead now. Zodasiran with speed Plozasiran

actively we those. not are to so And partner looking

happy But the working partner, we on looking to for would at clinical we discuss assets. in that not would existing not seeking potential build at with those not to target together least On pulmonary a right a may drug us now, assets. clinical we actively whereby our platform partnering a partners be candidate. We are for be bring side, partnership three are a somebody

Operator

Merle And question from with comes our UBS. next Ellie

Eliana Merle

Hey, in. for thanks squeezing guys, me

into design anything as had just guess of are the Just high on year? in from latest of the planning then there in you considerations looking mentioned just I a focus note, related two there to study and see program you the timelines the the in the subQ the about just what's perhaps program? past the just on RAGE, phase that status Thanks. on program. larger And and would think a are in in and you cohort asthma, what subQ design? considerations some the play the what And later FeNO this a

Dr. Christopher Anzalone

Sure.

subQ just deep We knockdown. the with the weren't RAGE as as a good news. we're a So That's seeing we seeing were the program, inhaled. seeing knockdown

And not as so as the inhaled was development we the version. strong want are future work, it James, But really you on inhaled. did again, it focusing our but just to?

James Hamilton

to that. X add into don't I will plan, it don't I have our so. think anything to play Phase

Dr. Christopher Anzalone

And so that? asthma question what's [ second the about can ] again, what, repeat you

Eliana Merle

to of might you're the Phase the design for FeNO from and start and what like see that cohort timelines the X some Thanks. and decisions? the how inform high just considerations the looking design Yes,

Dr. Christopher Anzalone

different we're a around now. about right tossing of so Yes, and thinking lot designs

design high design. which than I in single FeNO would able study data, know pathway. want but X I moving both. single favor I that, be FeNO Yes, and looking think to X it and the QX a And population to patients course, plan really non-type one want the want more reflects a think only inform with we type to the forward of we're we But study seeing a keep other. to in both the look, may think we'll to certainly readout on to the patient

of cytokines different a his remarks, of one to RAGE the FeNO mentioned detecting is movement prepared of As we in RAGE, only move really number those. expect James and

important certainly not only And so it's data it's point. helpful, the while

Operator

from our Raycroft question Jefferies. with And next comes Maury

Maurice Raycroft

for thanks was too. Hi, questions. RAGE taking about my I to going ask one

So you're And other at PD this data well? do collecting, that a quarter will end PD those can asthma of data? only cytokine that patient that as on higher if will include for knockdown include the you'll biomarkers or and clarify public update that dose on you the

Dr. Christopher Anzalone

take I'll that.

based seen sure will in patients past, those. tell have far on full knockdown So consistent term be know knockdown seen top knockdown that. to with volunteers, is what have for what sure. report we're far, venue it's we so is a going that out still there's what in have the we and that Once press what have continues. I'm we've so I the seeing what in and in not if set, continue. include set, good of profile And it we data is in can really to the that release again, mapping that, expect you healthy we so to a a near rush incomplete on we'll don't I said an given that which and I data seen

that patients or quite seen have again, far. I anything have what conference of going do much the put volunteers. to you have we knockdown at haven't That's to help the we so disseminate think really presentation we'll a what we've you. But similar data be be to part else? those probably thought James, some honest, to we're because as in into how expect

James Hamilton

No, that's it.

Maurice Raycroft

Got it.

from you those maybe potentially And follow DMX. year? helpful. this have that's Okay, or Could of one data DUXX programs just then for either updates up

James Hamilton

that's have by We end contingent. very enrollment year, data could Yes, early goes. how some think but of I on enrollment depending the

Operator

Agrawal Cantor Fitzgerald. our next question with comes Prakhar from And

Prakhar Agrawal

of questions. seeing, competitors' Hi, profile Model. some in DIO your thanks Alnylam terms candidates' Number clinically, had WAVE and differ you in one, INHBE, of pre weight does how your for mentioned I are taking my loss the XX% knockdown you versus on of level think

So maybe that. if you then can have compare I follow And contrast a up. and

Dr. Christopher Anzalone

Sure. Yes.

what study, others XX% and level in weight similar obesity others mouse gain what seeing than reported. drug control in in And the of to have between which have knockdown XX animals we think, were differential more believe, I that animals think is, plus, that that is receiving the reported. I I model,

can I the that's you. probably I think best tell

Prakhar Agrawal

into from are an the the it. broader or population it the to point? for this what side, the key you mostly And trial forward decision a outcomes questions, need CV is which on just at asset decision to do CV answers the make Got on move internal FDA

Bruce Given

gone their point at them the yet the Yes, start of go it's Bruce will, We not FDA again, point, FDA. the a And we course, that at trial that. point to the in without to discussion. at Given, we've on engaged input. we But we're haven't that this some like can

Operator

And our next Lebowitz David from comes with Citi. question

Unknown Analyst

now? Hi, Hello? hear you me can

Operator

Yes, we can.

Dr. Christopher Anzalone

Yes.

Unknown Analyst

[indiscernible] taking Hi, this question. Thanks our on for David. is for

of see can Zodasiran, what readout were the readout? more for data So that color could is at points give the us you maybe We timeline, mentioned the quarter. maybe in expect last readout you regarding a terms in the we little second and the scheduled data wondering FDA's of patient visit to if

James Hamilton

presentation some Well, I would that, would we I to think, in do I top a present think, again, on but this. a looking we way point. with haven't a did we Chris some to such once line academic in an to would, meeting. way Maybe fulsome at in present, actually be in we'd not that meeting spoken want

predict point, data I would that some it's think some I it's fulsome information, but later, most would before. I suspect top-line occur. when get the same way, answer in be hard of and meeting we would we At would the this had that to out what expect. would, come But, yes,

Unknown Analyst

Thank one maybe you. up. just And follow

and how think the therapies? do two choose Zodasiran you approved, that both Assuming clinicians Plozasiran between are would

Dr. Christopher Anzalone

a have on the results It's question in setting agents point we at hard of to that SCS where, comparative answer or actually, this in the efficacy don't two the either population. safety

a So wildly point that. call to this it'd be at on to speculative try make

James Hamilton

we knockdown appears data, looks APOCX Bruce side, what the frankly. or our as that know we've that think our X again, on Yes, at Xs interval ours X longer. the it don't their Phase greater. side, our the to Phase I be like Phase on Xs, But says, will according Phase and our triglyceride comfortable levels least we is at is greater don't we appears, triglyceride lowering When that yet. available And seen dosing look versus data X from from are their Phase that

Operator

our Pickering And question with of comes the from William final line Bernstein.

William Pickering

Thank evening. question. for Good you my taking Hi.

pretty study. based You've population estimated approved large single which a on the addressable like XXXX,XXX, FCS a to population get XX-person sounds at

trial? the of definition the agreed FCS you. your So with can that population a on accept which just exact of you just FDA for us to they single agreement Thank remind

Dr. Christopher Anzalone

Phase the confirmed are history we and is that with with patients study, triglycerides we of the studying studying. patients XXX genetically if are In That's will, those people above population you X FCS pancreatitis. and clinical which with FCS,

Operator

now would back to closing to I conference like turn remarks. And Anzalone for the Chris

Dr. Christopher Anzalone

much to those California, of for you dry. who the are stay And in next forward We you in speaking tuning you look I with over Southern very Thanks today. quarter. hope

Operator

This concludes call. participating. today's Thank conference you for

You may disconnect. Goodbye. now

Arrowhead Pharmaceuticals (ARWR) 6 Feb 242024 Q1 Earnings call transcript