ARWR - Earnings call transcript Q4 2021 | 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 Anzalone

everyone. afternoon, Good joining its for to Arrowhead's discuss Thank fiscal us year for XXXX XXXX. ended results you today September XX, today President are from Dr. us and With CEO, management Dr. provide of Christopher an who will the Anzalone, quarter; overview an Javier San Martin, will Chief our Chief Medical give our a and Myszkowski, Officer, the update financials. will Ken Officer, pipeline; Financial on who our provide who review of

of and today's our Translational James In Hamilton, Senior the Vice Q&A of during addition, session will Medicine Discovery call. be available Dr. President

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

For Form and our on Chris? SEC Christopher call President further our to please the refer of I'd Report including Annual risks to these over most recent That to details turn filings, said, Anzalone, like the uncertainties, company. CEO XX-K. and concerning

Christopher Anzalone

you Thanks, today. Good for everyone Vince. and afternoon thank us joining

need currently be for China. outside development Arrowhead. want will Before Greater therapeutic investigational closing, commercialization in afternoon I This review developed and which agreement I NASH. the commercial announcement it take genetic This infrastructure several a for territories NASH, commitment is last study has to a also as Greater patients commercialize commercialize over ARO-HSD, impressive a by develop our with to our will of a bring who partnership of is this ARO-HSD books and because ARO-HSD the to GSK this exclusive with medicine. will all and clinically medicine develop millions an is for GSK that programs to us we responsible for except to us important the has and discuss to to fit clear being quarter, tens has potentially Patients treatment on regarding further great license X/X important wholly capabilities our GlaxoSmithKline Upon off may It because our in articulated China, be for license expertise Phase clinical capital substantial GSK that receive transaction focus retained an ourselves. of and years it. development a large next spend enables the

capital, Phase offer for at following to non-dilutive the million of providing this receive future the kind to In Arrowhead payment drug and will million additional $XXX up and million payments million at upfront exposure we is of in the believe X; eligible should $XXX start addition, substantial the up start brings us $XXX and with key possible. at U.S. launch it to an success $XXX while sales candidate Phase in milestones. patients $XX of of million X; the markets; for benefits major

global in program, further if clinical product deep and month right these rights, reductions compelling steps of evaluate to AASLD demonstrate eligible areas production liver retain our our showing for inhibiting are believe levels of is have associated receive results. mid net to ability stands on to proof we to is NASH study we HSDXXBXX a of diseases. X up been to lead improvements protection for The had We earlier The double-digit time, genetic level clinically data this partnering against Arrowhead is a standpoint, shown From XX% with to study to feels inhibition, the ALT. next over people concept have what time HSDXXBXX selectively another some partner have to a Arrowhead are placebo-controlled sales. $X.XX do. royalties in together, Taken tiered use whether the We liver marked would significant protein presented with ARO-HSD of like commercial receive demonstration strategic X/X billion. published would to of this intrahepatic initiate outside and because that in other NASH. fibrosis This mRNA does deal it and We decided to mutations of dose-dependent reduction our designed product this of Phase loss-of-function focus. Phase that to data from data also HSDXXBXX. think believe in that at suggest the we

so They not in have clinical, needs established position future to pick strong in. medical reach have resources studied and steps efficiently. prior inhibitors They of program extensive clinical experience a GSK are concept advance and regulatory, of HSDXXBXX where and global it there commercial. and affairs, in human A in the up been a However, is This to studies. be trials, any proof benefit in clinical Clinical

I is patients think we As net the NASH. ARO-HSD and the mentioned, program deal positive a for this with

take We and in company that lie opportunities the through the is commercial to are clinical that the development regulatory steps confident program and next right GSK to ahead. chaperone

We move recent are advance look the as I during some the important need. medicine accomplishments on and prior in potentially call. quarter our our last the now highlights toward to thrilled will of partner to forward to working period newest since this to with patients and GSK welcome and new them

for XXXX, develop with our and is and a license and which as patients we programs agreement NASH. for to liver option to platform we earlier which earned Janssen, exercised in million which called the driver signed strong fat an program Janssen. validation payment three the ARO-HBV milestone called using designed in PNPLAX $XX for JNJ-XXXXXXXX, Janssen developed previously its accumulation is RNAi Arrowhead's proprietary has a a liver of and study, expression PNPLAX of investigational Arrowhead JNJ-XXXX, genetic potential now therapeutic with B TRiM take JNJ-XXXXXXXX is clinical in damage. currently additional year. covered license, collaboration program, and exclusive Phase and potential a first in would reduce the X hepatitis as an preclinically treatment Our that preclinical

During presented fifth collaboration, the after the of patient million negative is This $XX study. investigational assess X therapy daily RNAi daily over that proportion criteria, limit of levels the Phase than and at reduce or Janssen to XXX, XXXmg. oral ALT HBeAg of less of proteins. was patients quantitation, primary XX targets injections X than JNJ-XXXX the NUC used to clinical at of safety quarter, HBV of HBsAg CAMs. which data with less subcutaneous XX, stopping additional top treatment. study XX or RNAs, one times of earned AASLD thereby the than efficacy weeks the from with the in of all therapeutic The is a DNA levels Xb of milestone IU/mL NUC HBV of payment an a Janssen the monthly modulators without lower limit dose less or at is an assembly HBV upper the normal, and on capsid Arrowhead Staying viral study dosed of JNJ-XXXX, of REEFX, a was intended Janssen's meeting JNJ-XXXX all endpoint end Phase

endpoint. was at response The less NUC finding. in was cohort. didn't mg also adding an logs HBsAg XX.X% than that any Taken XXX of observed met were week was other the treatment At of is dose baseline of to to patients presented Up dependent The important in XX.X% an mean was greatest with reduction patients achieved the the NUC XX.X%. encouraging XXX XX, cohorts for X.X a very of the weeks primary appear CAM XX, additional and effect. data stopping in us. A in off data criteria patients week met HBsAg AASLD. total HBsAg criteria, observed from from JNJ-XXXX IU/mL, This XX and XX.X% had reduction beneficial stopping

months Additional removed. to therapy was criteria patients meet continued six stopping after

We are study. data with eager longer for and patient follow-up individual this see profiles to

went therapy the you being which some ARC-XXX, happen functional first-generation to removed. didn't within with clearance achieve six compound, in If the recall, on of months studies patients our HBsAg cures,

XX X, HBsAg, longer to we excited results took from to REEF-X. so or patients Some see additional months XX, clear are

other These study, patient ongoing called with population patients a currently hepatitis of stopping in therapy criteria; conducting. Osprey HBV a where There to and are desperate an progression JNJ-XXXXXXXX. is the the disease; NUC a need which also achieve various study patients ongoing PENGUIN, vaccine, DNA with of are studies added. study Janssen immunomodulatory the they We called delta therapeutic that options interferon, studies with eager rapid all due as to see is - the a REEF-X is multiple pegylated various ongoing studies which off with and and agents the come include from virus, data in

play against Designation a HBV. with investigational backbone the comprehensive FDA which how believe study, with patients clinical that ARO-AAT called We the impressed deficiency, liver being as alpha-X co-developed Phase a disease and TAK-XXX, quarter Breakthrough interim reflect central for extremely a continue target. think from liver Takeda. role designed the data disease associated and XX active Staying liver has an with antitrypsin we program been highly candidate AATD with additional to clinical to we during chronic it for JNJ-XXXX the and open-label to also of ongoing We our have is We its baseline on is in study the response presented assess X ARO-AAT, the AROAATXXXX development also is to treat from potential AASLD, associated data therapy ARO-AAT received with presented additional approximately fibrosis. Therapy data

protein, from cohorts, week improved health. of and we the from an of When fibrosis at had that fibrosis, X baseline fibrosis. week a The point range showed XX patients the stage in clearing improvement biopsy mg have of suggest XX% believe X progressive reduced burden with multiple all GGT improved study, Z-AAT week XX. a looking point we has for XXX% from Six from Further, paired saw in analyzed now. in or signs greater that a baseline reduction fibrosis. and ARO-AAT XX. ALT reduction this of lead healing. the are XX% of globule of Z-AAT as disease. Specifically, greater livers may the PAS-D liver ARO-AAT liver these to data in that cause to ARO-AAT ARO-AAT with XX suggest inhibits the we clear treatment may reduction X production histological patients, the In at Z-AAT data to fibrosis improvement or XX% treatment globule mean XX-month additional treatment XX to burden. also been us improvement X ARO-AAT accumulated having METAVIR which patients liver or improved XX with XXX and treatment protein. of XX only also to the biomarkers XX% giving XX% of mutant established in liver Since presentation, strongly showing patients of patients XX be a

Importantly, ALT program all groups about data. confident We showed believe is all to continue schedule normalized following we treatment. on The these be and potential. and its encouraging are GGT

We with have to endpoints accelerated continuing this and dialogue potential approval begun the pathway. FDA about We an look continues. dialogue forward study as a for approvable SEQUOIA productive the

expect sometime to move select a the of in circulating have few last XX-month of should summer the the the collecting over reduction should We from XXXX. We next of patient data dose from biopsy levels enrolled to with. to us months, on which forward also AAT the be last SEQUOIA allow

below we to patients pharmacodynamic We all on mg Let's - presented events effect ARO-HSD events is XXX observed of at AASLD HSDXXBXX today on lower investigational mRNA clinically a no was leading reported, on candidate were greater. adverse mRNA and mg limit adverse HSDXXBXX. NASH hepatic now no safety various the We designing need reductions. ARO-HSD drug-related which the In There announced sorry discontinuations these of laboratory suggest compound of we move drug with quantitation. showed ARO-HSD HSDXXBXX that look dose-dependent designed all has GSK. we on all AROHSDXXXX, X/X an drug-related all effect hepatitis doses clinical levels. assay's production in forward ARO-HSD ARO-HSD to clearly is in our multiple and no of protein and of levels, At measurements treat studies dose pharmacodynamic medical licensed with greater levels were data patients, reduced observed that in XX% been Phase in highly a NASH evaluate data enormous Phase unmet than the at and X at to dose, to believe active patients and to adverse study, dose well-tolerated AST ALT XXX and patients. trends observed. I'm a Hepatic the Decreases is ARO-HSD, with significant for in GSK future beyond. silencing liver to serious

this in developed ARO-APOCX will to in or data a Phase studies chylomicronemia, four with non-FCS, tend hypertriglyceridemia, as are Phase AHA the pipeline. Arrowhead's data study moment levels ongoing another which XX% early APOCX APOCX, changes there and therapeutic similar and additional XX to In wholly triglycerides targeting comparable reduced or patients. similar high XX% to and whether safety was in highlight presented as XX% median we parameters. by of study, give maximum different reductions was XX% respectively, We Non-HDL-cholesterol same our HDL-cholesterol Both defined assessing FCS status, genetically by Javier and groups on similar patients similar XX% the in has being in XXX% and non-FCS. The and of of by triglycerides presentation. XX% and MCM data Across in reduced wanted phenotype ARO-APOCX patients showed have apolipoprotein and XXX% will respectively. and respect evaluate have syndrome to data predictive MCM reduction but increased early and our with preclinical treatment clinical We of stage this pharmacodynamic owned a recent latter on clinical Javier week, of of and cardiometabolic familial data refer see newest been programs, a respectively. a one to severe clinical hypertriglyceridemia, response these predictive largely their patients achieved and was been good can and believe safety later data. early what we idea our groups. presentation is the special, main The to key mediated about to increase is development go important to an various what is risk. have I Moving or That X/X design that and update diseases. FCS. clinical reduce with of to as program. clinical initiating investigational and genetically the patients I brings which serves In probability a At RNAi because production wanted for X extraordinarily with reduce of two C-III, into with component complement we last patients response FCS is complement our confidence makes CX, clinical therapy of to FCS. a and ARO-APOCX, of ARO-APOCX, or for FCS APO our potential potentially X this view, patients confirmed was with tolerability. part expect. now are reasons parameters, success chylomicronemia compared multifactorial Arrowhead as and ARO-CX lipid C-III or describe. we that similar investigational therapeutic me to This we exhibit an

and nocturnal initially undisclosed and paroxysmal pathway complement and in also During the the other targeted pathway intend have previously did opinion complement-associated multiple hematologic nephropathy, the to evaluate X advantages to like key studies and the other explain we a an glomerulopathy, diseases may our webinar that renal hematuria. will also has potential on quarter, clinical we RNAi candidate, diseases. the announced the explained KOLs to we we and leader theoretical complex a filed complement over we the a The therapeutic, hosted future. and we complement-mediated We ARO-CX, IgA There complement is the targets. diseases other webinar These CTA focus mechanisms address why drug include to discuss the begin CX to or are that think on. and best complement

for medicines studies track paused program, enrollment developed ARO-ENaC, of I website on quick toxicology voluntarily you the have webcast, view the potential I'd to I with but which platform. some including to like programs, clinical on information translating This data mentioned, listened ARO-CX. pulmonary provide a early our we TRiM record for as is well a preclinical investigational more to haven't clinical it to about update you we findings. good assess as recommend our currently preclinical new an is If

still understand on studies to forward. don't we internally the the findings We conducting have clarity toxicology path are yet and

on new those the programs, to XXXX. conduct two continue filings of for are studies, which progress first on pulmonary track in we half make our CTA we While

on prove options. that in believe that parallel are we has fibrosis working addition, in should or diseases a to cystic potential ENaC address multiple In our a pulmonary as necessary. the confident are to be without treatment for target ENaC We candidate adequate and helpful targeted platform TRiM next-generation the lung in

so there committed the the convinced less in we pulmonary the when we don't platform to getting have tissue, to yet applying the but are TRiM can. are have We platform and apply clinical predictability experience we we TRiM that liver, that we we see

programs over plans. our Before to to Javier cardiometabolic and mid and and growth want programs it like X discuss year. - expanding per intend of To support have to growing to stage pipeline, programs, Madison. stages footprint our Diego to status San of to R&D by XX discuss now our I'd in stage We clinical the in this I turning expand planning are our we the discuss later pipeline X new

to new facilities. a those will tax with agencies potential new plant. and Wisconsin, space coming to expanded GMP R&D government We development are facility. In house leasing with will incentives discussions be we Arrowhead San new in economic authorities over and facilities Wisconsin the other Diego to build explore a state scheduled be campus the build year. a that be and built These manufacturing is local in discussions intend and Should drug in an we to successful, two

drug for every in manufacturing preclinical, sense. speed now true size will is timing, and internal to commercial and buy drug is makes manufacturing out pipeline indicates commercialization to at product the a in give capabilities development us financially our of point build clinical, strategically. versus over function. This and every stage of value where more of control both and importantly lot advancing proximity control Building process cost. Our that and analysis We

We estimates so yet for like to I'd over Martin. to capex the incur started have will we significant our to San overview, planned should purchase not about the Javier? talk but Ken forward now Dr. that closed costs, turn Javier of land, on we this expansion. With have not the yet move with later call

Javier San Martin

XX with we these ARO-APOCX studies C-III the Thank patients now start apolipoprotein cardiometabolic in XXXX active chylomicronemia is SUMMIT We three will have our peak. Phase either study lipid called patients SHASTA-X; mixed will duration you, will with PALIDASE. several Each each the greater will a XX to which screening. will in is XX evaluated who a on with ratio X for All Phase evaluate placebo and to placebo. enrolled is The at per and in the focus The XX XX in calling fasting ARO-APOCX FCS, advanced, and be Phase hypertriglyceridemia, total sHTG MUIR; Xb program, is are stage Chris, participants receive in the enrolled. the which or Three patients approximately participants for a later open hypertriglyceridemia, X to will to XXX ARO-APOCX of wholly with the I with A status will late-stage the are X dyslipidemia, or randomly I screening mountain screening which mg, and dose we status ARO-ANGX. we levels briefly injections are to population. to X for medicine study. a set patients studies and from be efficacy is of studies with cohorts of and enroll ARO-APOCX. is planned weeks against good Between with for receive or and study each. I are everyone. with X examination. XX select have various severe enrolled primary assigned XXX patients targeting named clinical X of including XX. of patients, in safety currently currently calling has additional study a study cohort week of participants mg/dL equal dose XXXX in of This SHASTA-X parallel a XXXX subcutaneous with XXX a ARO-APOCX hypertriglyceridemia, objective programs, adults give this approximately dosing Phase evaluate end-of-study placebo-controlled XX is double-blind, active a calling ARO-APOCX current of participant than our sHTG. patient and on and study programs, syndrome. The describe these familial adults potentially each in ARO-APOCX day study hypertriglyceridemia investigational want the studied being study triglycerides SHASTA-X safety and studies efficacy with in be that to regimen most mg to mixed dyslipidemia, be week there disorders afternoon an dose mean XX the SHASTA-X or clinical and in owned of are mg, two in to of mid chylomicronemia severe in severe is studies multifactorial soon. of

activated QX XX study the XX around of enrolled goal fully planned have sites We have is XXXX. to our of and the

XX in or equal XXX and to study. enroll enrolled mg/dL. cohort, injection X total mg, randomly in participant XXX the XX X greater is screening the elevated than parallel Moving approximately greater dose for or ARO-APOCX placebo-controlled a and later to to and double-blind, weeks cholesterol XX In to will mg dose receive will XX duration receive Xb will cohorts population. is at of All and equal equal XXX has regimen non-HDL-cholesterol adults of the will mg. triglycerides of or per The X the three dyslipidemia. placebo efficacy and dose and on will two dose than than study in day and a against MUIR study. LDL injections. the adults to ARO-APOCX cohorts XX participants dose subcutaneous additional evaluate the or who approximately XX in mg/dL, participant injection ratio one be Four safety placebo. subcutaneous XX screening two day of study less of week had XX mg/dL injections. The a additional to Each the or XX but a from to than cohorts ARO-APOCX examination. study greater total is be approximately a for on each mg/dL mixed in with of week The ARO-APOCX following mg, cohort, of to X participants end-of-study on a with receive total week In evaluated XX participants the a assigned XX Phase of It in enrolled enrolled. XXX potentially this patients, the currently with and with to primary patient XX MUIR clinical objective planned dyslipidemia studies evaluate to efficacy mixed MUIR select patients study screening in stage for safety an of XXX to be in A

placebo the XX of to examination. invited our placebo in participants month is XX The study All We XX, enrolled of and and are safety fasting continue end-of-study XX to of refractory chylomicronemia of level X to primary approximately group XX months. be triglycerides. four duration efficacy of the baseline a mg primary doses evaluate in receive the ratio randomized ARO-APOCX mg XX FCS. the an standard efficacy ARO-APOCX with of consent triglycerides during PALISADE with at study to percent than in and of switch Approximately lipid-lowering total fasting screening month and is that in month last the mg/dL active After Two-dose who once QX to to and XX have XX be PALISADE every extension XX study and XXX syndrome. is to from participants extension will active familial the placebo the participants therapy study or sites eligible evaluated The study of to the adults greater is will against is the ARO-APOCX FCS. the X opt from subcutaneously and ARO-APOCX goal and in participants X study to study. in diagnosis objective will three activated continue adults safety to open-label drug weeks in administered of ARO-APOCX XXXX. The planned be change Phase evaluate is have in with endpoint will fully for PALISADE.

the exploratory change pancreatitis, acute of Additional include measures. other other endpoints secondary and in and parameters, lipid incidence

three globally. have of the We planned XX sites activated

Xb XX week mean with the sites. be to are to X per of of additional move ARO-ANGX We investigational enroll working park. approximately be anticipate of this is reduce the we All will parallel enrolled duration participants primary equal to to safety There with in After now year. dyslipidemia. for and screening with patients week XX hard The adults program. in investigational Three with greater potentially screening greater our currently ARO-ANGX assigned of end the XX The select XX. be study stage mg/dL. approximately evaluate study. on to X one the in of have adults evaluated continue will open-label the to cohorts ratio dyslipidemia, this I currently screening and a and and and XXX mid to in X currently open at patients triglycerides of enrolled. mixed or with cholesterol mixed equal to patients XXX the is the to XXX enrolled study. in XX for visit, a participants in enrolled and objective non-HDL-cholesterol mixed randomly placebo the XX will A for LDL who mg, of XX later participants are completing dosed an XXX levels will evaluate dosed, first patient X. the The to activate mg, placebo-controlled with of the mixed The late-stage has the efficacy Phase had VISTA mg/dL patients X in dyslipidemia. fasting dose are mg and and ARCHES-X study ARO-ANGX, of will mg/dL We named to a with or study following active subcutaneous patient production is screening and dose in receive patients the ARO-ANGX study set a in designed cohort to or total XX in protein XXX medicine which study between VISTA is of ARCHES-X injection angiopoietin-like ARO-ANGX end-of-study week against clinical mg/dL; in than Phase population. regimen a weeks study examination. this called be or the study eligible ANGPTLX from for XX additional and a AROANGX-XXXX, program, with studies treatment participants potential study day enrollment be we placebo extension and national calling XX% ARCHES-X. in is before ARCHES-X to double-blind, an than dosing planned ARO-ANGX dyslipidemia safety of XXX efficacy on reached XXX a

activated I the sites to initially of this over fully in is have Arrowhead's XX Chief We have Ken? QX goal to Ken study call of enrolled Myszkowski, our turn will XXXX. planned XX and all Financial Officer. now the

Ken Myszkowski

good afternoon Thank you, everyone. Javier, and

Takeda outstanding. of we XXXX. today, recognition with $XXX to for our for Revenue trials $X.XX the million with obligations or the for services. Revenue Takeda. fiscal XXXX. based weighted share compared work in shares Takeda to performance our of under XXXX the will to $XX.X to reported average on shares fiscal Any portion $XXX.X relates million, to as manufacturing or Revenue compares approximately was recognized additive our million three years. the period collaboration for related fully outstanding clinical to additional it per diluted the be a As fully $XX.X weighted be revenue partners primarily over we based current XXX.X process recognized anticipated XXXX million on in to and certain be collaboration diluted net payment net This million for of continue remains million this and is of of with million two per collaboration share XXX.X our loss $XXX.X agreement completing milestones projection. loss achieved the would was with There million toward agreement be upfront $X.XX average recognized received $XXX associated managing

end agreement. this upfront a million of develop During drug to candidate uncontrolled XXXX into portion new Janssen. performance the period the collaboration amount expect for balance with related fiscal a agreements received completed. to portion recognition as the $XX A fiscal was in by a with Revenue entered a and license the Horizon and gout. of We XXXX, be of to to in primarily we from the also received the recognized this we of treat are collaboration agreement payment our requirements XXXX milestones recognized prior

also well to We were ARO-HSD Total as the with received $XXX compared the result this $XXX to of in stock We the as of XXXX. $XXX.X and $XXX.X discovery payments candidate. has million to change fiscal operating increased increase was and candidates our today used advanced, compared primarily R&D to This agreement million, candidate cash recognized is million, XXXX. for compensation a by Company's of this operating be XXXX $XXX.X for increased both of agreement revenue as total This $XX.X with Arrowhead. Net fiscal in as in million Takeda Horizon in an fiscal pipeline of key XXXX anticipate expenses due from XXXX. costs additional clinical by substantial payment cash million driver non-cash announced operating will fiscal provided activities was expense. new activities The XXXX. and during in licensing million majority upfront net upfront for GSK specific

potential be quarter partners, any XXXX. payments burn million our $XX Excluding our from to per $XX cash estimate to in milestone collaboration we fiscal received operating

an In addition, These planning full add outlay million our of expand $XX projects, expand with XXXX. $XX will capabilities manufacturing incremental facilities. capital for routine we our are to along fiscal and capital cash R&D expenditures, year to

our sheet, balance $XXX.X for and increase in The million investments upfront Takeda primarily our was the payments in at XXXX, compared Horizon, XXXX. at and $XXX XX, our investments operations. from total $XXX September September due to cash million to cash cash million to by used XX, totaled offset and Turning received

overview, to the Our September million. outstanding With I common that back turn XXXX at will XX, XXX.X were now Chris. shares brief call

Christopher Anzalone

strategy be And medicines. are to development of development. us with expansion from to further continue clinical pipeline grow programs continue Ken. Thanks platform extension, vast for thanks respect support on Arrowhead's you and investigational near-term our day. encouraging the joining all strongly to executing in our our and long-term and today. The opportunities and each business to of data We're

patients medicines and we This importantly, Lastly potential discovered Arrowhead need where distant the future we would the commercial them. in invest I start we to clinical in developed is the like not your why capabilities see who open R&D manufacturing important now and and support now too why questions. to get needs. building by and are to to to supply call the Operator?

Operator

going RBC from to [Operator Capital. from Instructions] And Issi come first our Luca is question

open. now is Your line

Luca Issi

questions for the to ask Great, the thanks follow-up, my congrats question so and I both much progress. taking on and a guess. main I'll

That's sounds with of profit a on on the the level on retain just GSK. receptivity split a why here like decided obviously sizable with approved of AXAT, Wondering but very you Can on question different to economics AXAT, can in level? AXAT little in here. on a that question had structure for expand comment conversation? we FDA was idea getting one. And the two you have U.S. you liver their NASH. it Then XX/XX what more if productive you for So dialog

color great. on be any that would Thanks much. so So,

Christopher Anzalone

Sure.

fact, take we'll a and just the There's Look, with had is FDA, one we much in first the which straightforward. productive tell can we last discussion encouraging So you. first week. very not

that discussions. I it this. the are think moving begun and we interaction. continue We a ongoing just collaborative direction an to we've and forward dialog is was in look right good This

particularly in dialog data. so continued as have have I expect XXXX, to we And additional

course. different right so respect there's now. you to I far. far good, farther think programs just I the the AAT was AAT it's I HSD for for the that, can coursed much so not think field - than as been With of is good state so reflects Other HSD, that it's conversation So tell and than advanced a again, first. development of two structure the

AAT. Second,

HSD I that tested about trust have think stuff this genetic the fully need really at we patients potential there's of millions tens there whereas genetic that will that, as different are disease, is out. is XXX,XXX validation AAT we show But between call, two and in but state potential indication. quite I AAT Even so need of the progress in people there's of different now, HSD. human protein still for that and very it's think GSK are U.S. that that There's that, no show just the for a finally, in earlier patients XXX,XXX large been while a of U.S. least concept maybe to biology talked inhibitors very we'll the good been diseases. these Right data still target. HSD and clear. the look likely orphan is or so proof We we an to encouraging,

move with program different And them for And got in the would these indicator look with partner structures really appear to forward. think this with I two both programs. NASH. They are are and the for AAT I that have to GSK be their economics committed program of bring of different together working so we think the to important who we given impressed and we patients. for have committed very medicine and right those we all We're them, happy we ability is right for partner similarly, to that, to

Operator

Cantor. Young And our from next from Alethia question comes

line now open. Your is

Alethia Young

whether wanted I So, One, your on as you like kind you question in competitors like taking my sRNA interesting is about data just think at to way some I maybe of then talking perspective It's AASLD. about that go? about combination on And guys questions. activity. are a and bit think do second you of doing for now would talk capsid know your neuro? initial little potentially to the ever is my feel capsid the the I of Thanks mean something just things? I kind

Christopher Anzalone

Sure.

think good hear you. I we from to It's did.

by that combination very for to was respect I with encouraged think data. So, Janssen's HBV. Look I

reductions to it's We is doing do. drug antigen. deep of saw Our designed S what

seeing We good. endpoint they of therapy better I XXX criteria We antigens. criteria good primary a to came more lot IU, saw - all who who viral chance XXX are the of of and and encouraged reductions think it got that good XX% we're I patients even functional watching are really off percentage We seeing cures. continue can And so course, looked is that and who in we're so if or that therapy was we a of patients looking an NUC that important that assume and below and in patients starts S patients during IU by a number. who It's we seroclearance. been that have That's saw the we forward after these achieved number, past think some then, off get shown below can that patients even achieved impressive treatment. get IU stopping XXX

still still wide-ranging combinations. have has We've to may pulled the is were we functional that a drug that approach. you combination going hopeful consistent that to think that that from We to respect be we with approach us we it's thought. far backbone think so could approach get be and need a seen that cures that believed Now backbone and this and always tough virus our nothing

J&J, but look to immunomodulatory look of I ongoing. going right and that Janssen the think forward CAM number going in forward, studies They have studies We way. seeing that is beyond. combination what like preferred a not the the probably a is this about

figure what So the of part I I therapy in think the and think correct But support HBV. data that for early think trying we on are I right track out those the we're still that. as to

Alethia Young

Do you have any plans for neuro?

Christopher Anzalone

right. CNS, sorry, Oh,

not we we luck. - programs you're need some good addressed there time them clearly indications have for lot who CNS. now, in Sorry, stated that are have that have working could Look, we we don't and wish But reason. right on do good be been using right, of for medical and unmet so now. RNAi There's competitors any CNS there a some

we I that To us to be date, will we it's be some important for there. on point, think types. there cell have at because other focused

year. and talk next have yes, got new solid that we're program pulmonary call, at we right didn't middle will now. tumor, in about on eventually one have track by still we the But of you some skeletal/muscle. know, We don't CNS, in likely on for period, a but programs we've As skeletal/muscle the

Operator

Raycroft And our next question Maury from comes Jefferies. from

now is open. line Your

Maury Raycroft

or And on question ask J&J in questions. a taking refusal Wondering update HSD. option right on was X HSD? can HSD PNPLA J&J. thanks since Congrats for if any if have my just say NASH that asset was pursuing Congrats I of process on you're to Did the partner you to going update. competitive. and with for

Christopher Anzalone

a Sure. Thanks We a competitive yes, We did the asset like first of companies refusal run we spoke from best very be not or you, much. HSD. It's right good anything - about J&J process. for partner. that seem to have of a to the hear did GSK Maury. number with and

Maury Raycroft

for just quick how month how biopsy? patients, have one will clarify many patients biopsy wondering many can if XX have of XX will those and AAT, also question And XX-month of the you

Christopher Anzalone

Javier, want do address to that? you

Javier San Martin

four the X said did have in go XXX to or the have biopsy that's we to XX the on have and XX-month X the biopsy eight and received a all mandatory, right milligrams, cohort that case a follow-up months, through not cohort likely so accepted had accepted of months, biopsy mg, that XXX patients because all the be biopsy, Yes, will third not. patients that it's two had was few two patients not XX-month two and now, first we is of but to them of patients

many from cohorts at don't that know week biopsy sorry, was we will month accept they - post XX. how the baseline at So other

Operator

our question from And comes next Esther from Rajavelu UBS.

Your open. line is now

Esther Rajavelu

first ask the two, your could patients. between patients? similar If and is you which you Can thoughts benefit what the APOCX share then on the for and the be ARO-APOCX MCM just follow-up have I'll second. is Thank a taking magnitude on therapeutic I the reduction outcomes my manufacturing, for implications FCS question. on my of FCS in the program. for And

Christopher Anzalone

that? Javier, address you to like Sure. would

Javier San Martin

Yes.

in show to we XX%. XX% to much FCS regards the MCM presented very the to with and of population decrease at has range data week of which So AHA and the the the which the response we in is in the target population past that's APOCX same this triglycerides, magnitude

baseline what So is from depending the the or be low, will which patients means risk of therapy. FCS achieve on very their whole of that beyond which will that them, for with likelihood and level very, is a many

of this result. patients a on depending range very now, their of majority we're encouraged in And right healthy be the So by triglycerides. will again, baseline

Christopher Anzalone

Yes. And I that. to want just underline

that of level the thing. so really think about triglycerides. can I we one that's think a normalizing and really I that's that things big moving think triglyceride patients' about now exciting the candidate needle on We are many that's

Esther Rajavelu

that economics it. And existing progress may could or the it have deals? those you Got partnerships, future on of it once - your investment. change manufacturing you commercialization up to do expect clinic change assuming expect then you the your facility and potential Do running the that to terms assets for

Christopher Anzalone

That's one materially of a us, We don't to there to will partners. No existing deals. us, question, has change economics think supplier may I use they could want to the good but use so they those there. be a are that economics

that for ways costs something helpful take a it could lead manufacturing look, think could of for of capabilities. forward, spend to we good sense our days working to think on these future I new your I that and one. could could I manufacturing and And be lead a discovered have that that's lot It that point's partners think lower our Going makes better to time advantage of we be purity, process partners. development

Operator

next from comes And Joel from Baird. our question Beatty

now Your is open. line

Joel Beatty

progress. on the the on Congrats Two questions ENaC program.

just the looked that data the second two programs ENaC at study? on doesn't that non-ENaC what's first what with latest the on NHP may The gives program lung NHP update look filing of ahead? CTA the needed ENaC and confidence if be go going program timing then going you on at study is, ahead on be ahead is on can the is to even an And a that else the anything for question the

Christopher Anzalone

the for Thanks questions. Sure.

- December. is the forward that of - specific rats. that the timing response as a local I going the non-clinical forward in you we in now. point when quarter how a the of basis right with we can't first move move waiting know this number we're though we'll of timing chronic in year, give the out, of end exact I and tox, I next all for where internally understand that So you inflammatory on studies real-time that of be we sometime to and do target. we're believe by ENaC as the because should the to of try of NHP think doing We're saw kind also

I beyond may you restart to some decide I that though So more I at next believe switch move generation. this a we'll can't know much to quarter, ARO-ENaC horses path granularity, in or do don't forward point have but and - give either to - we we'll first that

haven't Nothing are have mentioned been on generation working remarks, of think nominated potential in-house I we - yet. next been a I As my we couple candidates. has in and we prepared

if at substantially reason ARO-ENaC that We potent that have was the issue the with substantially in rats. more tox and saw could than appear, a couple that potent the in or are we help issue fact an out more overloading for least

So more just say tuned lot I would on next think. in stay know a that. I couple the We'll of months

are pulmonary confidence these on constructs, these Look least the Regarding making what I at than also know as gives us potent two potent that we next ARO-ENaC. overloading so issue more could more the about better we as we're substantially constructs think we programs. new we focused make that an potent And the and be can. our rats in - can

feel tox look the least we you those well. file programs something - you newer the we've we in and we'll two the to half got in have are comfort I two CTAs go, we're know may good probably programs work chronic don't on we've But programs, more that in next know we - - that year. think gives have us at about of end at to far, day, and first see pretty and we of some that but where so so what run track those these got that the until we

Joel Beatty

Great, thank you.

Christopher Anzalone

thing. a good me. this a more was to point. given one remind just And I note It's on

getting. this to that technically next give has that's you're a tough what us how knockdown difficult have and much getting, leg programs going us. about how biomarker did to up - It I It's we about much understand so circulating on a is two what's think ENaC an the just program. of knockdown next to One be helpful we're idea was

Operator

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

line now Your is open.

Patrick Trucchio

Just a questions me. couple of from follow-up

competitors you and I'm I'm deal pipeline? seen in in an on can one interest terms that programs platform first of any level your which wondering wondering since partner next the if of the is, impact recently The announced your could partnering discussions of to the discuss if was be programs. various from you've acquisition if announced increased

Christopher Anzalone

the Well, interest that you're I of unanswerable we've master increased we've questions. don't know the seen any in last, partner got. what

say will we can control. here's I this, So, what

follow. that make To in we medicines other on that patients the at we that to and rapidly, of and medicines can safe we sort else Our are job do that can't. everything extent that succeed can that ways that can we and can help focus is

kind of recent And our the so this M&A I acquisition noise it business. because of affect view doesn't really the a recent - M&A - as bit day-to-day

important which to that's medicines make is day, here we're we're we So, doing. remember what and every need to why

Patrick Trucchio

Yes. half of X That's if level helpful the and would the program? that the to assuming then give And Amgen. wondering to forward top with Phase around Phase first I'm developed data AMG is discuss pivotal it's trial? could by confidence understanding I'm line just wondering a you track for Lp(a) in being what XX move on of knockdown XXXX, X expectations

Christopher Anzalone

Sure.

thereafter. that a so that's pivotal I that - have them And seen we've My to - expectation that of that will good. think move so, Lp(a) is data next understanding. been the do of drug will they it a understanding is validation far That presented half to have seeing that amount don't clear. more than very into candidate. we genetic that how has I it be that. know long that's with I potent would the the then of But very really you in do my The of deep drug knockdown in is I heard take would think Amgen small I data don't data haven't that know target be They're think and guided first honest. are year. a study my they that changed. frankly,

a I have of how is the terms first them reduction you happy and see we given they've be levels. my seen that have far then slowing moving will give our half I'd program, fast I into can't of what can't that X what in tell far so can I about so in what wouldn't if would we least. Lp(a) an So was year pivotal. of to data expectation they a idea move been impressed shown Phase at and we'll internally the you be though with really certainly down. just this make we've

Operator

And question comes Mani Leerink. from our Foroohar next from SVB

Your line now open. is

Mani Foroohar

Follow-up question regarding.

think some that put but out patient human expect you'll with ENaC had that see? talk So you signal be so about data related, Can primates question just related and a bit something put but releasing never not identical are early human a And I or will signal talk that you'll Should discontinued and data perhaps program. about another we away you it'll did counterpart ARO-ENaC little in program, - technology release have their in ASO, mouse this data. early with not is one follow-up. that just not and

Christopher Anzalone

don't point. Sur. I this know just at

where I to want point. to don't hypothetical kind Let's that to going go. - it's see and restart get don't - that of this what if I we're know program going at is so

new how have of paused. about point have so with forward, Once so, collected stay enrollment restarting a go either a been idea can we have then do to better that what the an or data that far. about we that at we And discussion that construct will we switching

Mani Foroohar

timeline update of an RNAi, area to or liver is program, of you on there in should one-off and still you you than about broadly first speaking. a of that of Or speaking companies and And continue you? a that see diseases the as place expect earlier talking development for kidney time at the Great. we extrahepatic an you a a for oncology all lot oncology experiment? spent more a gave obviously Is guys as growth program look broadly for targeting

Christopher Anzalone

We've experiment we're And target - good seen I that's that No, important. that It I in there. I clear to experiment? one-off engagement would not we off first knockdown seen think start that's We targeting. think important. the think call solid tumor a clear we've is

the drug. oncology platform. our need that Is are track. the And so rate iteration term see right particular it drug standpoint, platform now, we'll of at think we decision, just longer we not. We'll response a from and but last Absolutely a is to look make on if that I

We from - will least certainly perspective, advance start. continue to it. at But my a but it's good

and kind don't That really designed to designed Now of may is a We program solid tumors this yet at be to some to platform is in point that address clear, indications. address happen broadly. have kidney prediction the about don't HIFX. that. have know good if a I I now have slug - of when if next future, but going data we're a don't I right we to have have good for our

We are fully enrolled.

patients. anything do you following And on that. so James, just add I we're to think have

James Hamilton

patients period. in fully study the is and enrolled right, about that's are on think follow-up all drug the I No, so This

Operator

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

Your line is now open.

Mayank Mamtani

on pipeline and the on in. deal the thanks for also Congrats GSK progress squeezing and us

one. Just questions, clarifying quick just a few have

So J&J idea study, at there might on of any be have forward? analysis? when communicating on the time the points insight just and refund how - J&J we you follow-ups level on the come Is what can see might going patient thinking any

Christopher Anzalone

just give some I are that you don't I there things really because can't know.

I believe think In data. least, have the even at we I individual seen haven't seen. fact, don't we don't patient

encouraged earlier, data. by look, said we're I As those really

I think they're good data.

to of back sit this see what design how doing we need do just kind we now and let it is look, drug and to Our goes. for

early months, so our S, long on ARC-XXX XXX six nine months as for as removed mentioned prepared the patients years to is watching happen those continue in I didn't or it and so, forward two after in we as recall As who or it was up. serocleared remarks, follow as happened drug as for I look

studies REEF-X, with period that I we prolonged think the we given others. interferon REEF-X, entered and just rich data REEF-D - a Janssen have are

will I parts going forward. data regular these released studies there should think that there various of I think from that be - be

so, substantially think is probably was. be 'XX to more And I the 'XX rich going data than

Mayank Mamtani

tracking setting with you've X/X subjects? a the expectations the us placebo-controlled forward on you SEQUOIA AAT, then pack Looking about what to to data open-label format? that. you're be what XX can Phase think And in Understood. remind how And might relative is in I shown

Javier San Martin

Yes. Sure.

So, as you know, next The the we're XXXX. will occur we dose have work completed first enrollment of on XX of going half stage in patients. to selection,

July, next that dose and probably of be be agency the our will the to So paired with biopsies and next the discuss finished year. by will interaction August

second patient in will baseline that last So, be timeframe. of biopsy post

data timing have so and say we placebo-controlled for the will that toward I lock add the tracked SEQUOIA. of we So evaluation, to from biopsies the paired XXXX, would end

Mayank Mamtani

FCS will see on portfolio, the And - just Could I some of like of just me the the in enrollment the Phase can heard appreciate data detail owned you it APOCX. generation and activities. on And XXXX, that? on remind think study, you - how together, study. just I And cardiometabolic we on ANGX that's I I X level put wholly the for didn't

Christopher Anzalone

are patient to these for how and release this yet at hard FCS studies we're we talking we even fast get yearlong days, because both - to can These all - particularly about these data here unknown the our dosed it's so haven't there. point is we first enrolled. So - guide because the

on us we we'll and visibility Do on. have I we data can think time to to else start have enrollment you then give - when So, add at release? more Javier. anything better here have

Javier San Martin

No.

I think that's fair.

Christopher Anzalone

your FCS. And the question for on Phase the X -

before is We've dosing. the So and running of that have that screening. anticipate dosing we up end already first year, the study and sites patients we activated in

Operator

Chris like the now showing remarks. you. back thank And for And questions. I I'm call turn closing further to to Anzalone would no

Christopher Anzalone

wish a weekend. everyone Thanksgiving Thanks Okay. happy you joining safe for us we today and and

Operator

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

disconnect. now may You

Arrowhead Pharmaceuticals (ARWR) 23 Nov 212021 Q4 Earnings call transcript