advances The our platform. us create represent in was this Thanks, because value we primary thank value product period positioning for drivers to well respective last us clear and TRiM see us We afternoon, to proprietary substantial our demonstrated shareholders key programs believe as value that today. build We Vince. their in X everyone, throughout we and for XXXX. ways. joining seek candidates you and important to multiple development quarter Good an
First, platform we push RNAi organisms. to and because and relies difficult new on therapeutic is areas innovation. and forward address fundamentally our it diseases, This uncertain
Second, our eventual We we the TRiM the studies platform we amounts and, pipeline these well both. progress for expect commercialization. into goal our areas how later-stage progress either This as generate drive show existing of predictable is would but be works. us, more increasingly measurable drive in clinical regarding toward we large to straightforward in can believe, value data would of
As opportunities of the at calendar alone. we half substantial XXXX, progress year several first with look in fronts, on we both expect the
the middle addressable we bring instance, liver, clinical our year, the of for without For of range the opening ability to could RNAi whole new by have a treatments. concept proof adequate diseases of outside
may a studies program begin also be Takeda during frame, drug candidate we accelerate and have streamline III study market. time to also to We we that to large better clinical expect this ARO-AAT how IIb and to and X about the a idea Phase able Phase may
drugs rapidly a many hepatic what that liver. in earlier-stage to progressively platform We've which our to make by been could other programs. this programs, the do done hope initiatives. to we in develop way new areas seen diverse Let's ability our and concept of tissues our with and builds attractive applying this them upon in prior lower and programs in extra discover liver-targeted program safety begin risk. targeting looking are our have at We profile. predictable, TRiM has reproducible new power Arrowhead's is Each disease an The multiple
to Given expectation experience creation such type could as hepatocyte-targeted highly and expect generally and in new cell APOCX, and future others we HBV, in AAT, be a value a our type ARO-HSD represent this hepatocyte-directed tolerated. substantial event. Lp(a), candidates of Creating programs, clinical also in specifically ANGX potent well
only imagine Now cell but a in single new leveraging new that types. not many with type, cell
think the literally and past, muscle-targeted far? and on data months. made like First we foremost, ability for need to programs, We creation. the the target how new this down lung solid tumors have to said look platform that these we an to to cell in reason expect The about will knock clinic so I bringing target of safety the has of it to progress in into are we first a and so delivery of in program something seen we working TRiM and genes are one our can As XX patients, of recent What have in and be late been pull that this provide over and summer. to success to performs. have have huge prelude we what every idea it's the exciting, we cusp amount toward periods able in now off, value be expect we thousands good liver administered assess new acceptable TRiM profile. would to the believe just for This a we've time type the clinical leverage we bigger. XX a is a could If having
built on platform, While same idea the same distinct. they funnel are the and types tissue structurally are new
start in ongoing months. on by to concerning seen we which and first some are see need of studies seen the or collection the to data program now. then hope we well still still our program. are to expect so early tolerated either safety for for assessing in molecules that exhale we The ARO-HIFX, front to from data that the far, are program, But efficacy but on new before continues. programs. these a at exhale take stage not both is think coming almost our activity first, So We can middle We year lung have and we ARO-ENaC, share first and humans. and have establish for tolerability tumor-targeted focus data targeted not data breath We anything
hope data Regarding ENaC X in patients ARO-ENaC, we healthy and brushing some first levels in knockdown FEVX from also the have X or have volunteers to data preliminary lavage. dose via and bronchial
term course, in focused on informative. be While are, volunteer longer may healthy we the of functional data this more patients, point at improvements
current of number be study, treating will small difficult in on X to changes. the Given drug, only it the active we cohort per discern FEVX patients be may
have Should focus healthy for leap easier represent in much be should RNAi However, interpret. many is right in Therefore, This a a it would huge indeed a this ARO-ENaC, would for positive, suggest our entire the be but now. different targets lung also ENaC that company would to be could gene have it not may be we this big forward a we diseases. knockdown believe many only We from leveraged our volunteers primary franchise drug may field. event. and against
alpha to and on is Regarding preliminary calendar a short year. the progression-free cell we have some an share able target, the for of good ARO-HIFX, treatment, hope HIFX expression will in metastases assessing something is demonstrated knockdown important. these carcinoma, alpha biopsies and truly be by metastases, too too well-validated HIFX reduction to data renal particularly so form in small cell demonstrating We're HIFX focused period clear suggest in also and and study importantly, be to during potentially we that reduction are we could to solid franchise. observation strong consistent middle alpha have pretreatment we a more think a the a expression could from believe show we'll if that tumor have we taking survival, This of but we it knockdown. evidence we the be drug. Further have
inside designed variety to strategy gene rather a can can cancer wide of to targets specific, a RCC to HIFX of different any be reduction alpha, to would this is variety tumor case delivery not types. of solid solid but show in If it us across of targeting tumors, that suggest address we Our solid product tumors. we enable
forward on to lung, first in gene to increase substantial RNAi expect cell different I believe is company to clinical don't another we a can different X there concept program. could reach end programs. that file that planet our the say we and summer, we the value quarter, fourth with types As the concept a clinical calendar the we across second our CTA point. silence cell X a of expect of represent inflection for muscle-targeted skeletal the could in when a of Later that. type giant have in this a By be expression leap would can proof proof
multiple conducting parallel we're additional studies, these in work we pulmonary. targets continue to While on in tumor and clinical
X least early of I we XXXX. file For and programs pulmonary calendar at to in the number expect development, CTAs latter, us have in a
We on COVID-XX program. our also work continue
and the current RNAi on of as focused within We interrogating a virus potentially other triggers are approach coronaviruses antiviral and number are developing positions targeting well. an different different
While to continues for are the be the various important. suggests our and work next vaccines, about we preparation current the in virus us mutations to excited that coronavirus
results are we possibilities. to up pulmonary may addition, see In formulation now starting on that open early some promising new work important
inhaler nebulized we for opposed for diseases the to a could improve convenience product populations. formulation of been using an Specifically, as more patient That higher-prevalence in some device and a viable would possibility have commercial for evaluating make indications. large
and begin inhaler. then the to COPD, clinical in to to work necessary is which an over treat -- switch out ARO-LungX, formulation program to for with necessary technology plan Our the nebulized to then and development work a to was
to work and therapeutic ultimate our program's patients. decided if And time take shave physicians since a development on is now end, increasingly in it the safety have time actually encouraged may formulation a by convenience and the goal slows form some We overall possibilities. to is We direct this for their inhaler efficacy, CTA path. that off think we more a The forward enables don't the market. the useful to in optimal hold with to are off new
next We quarter, studies. will the over few pending provide ongoing an completion of update a
toward ARO-ANGX presented quarter, positive and our now discuss shift our we programs. and later-stage ARO-AAT, Let's data from to commercialization. Last our progress programs ARO-APOCX
support encouraged and they believe very these We are results of by programs. advancement rapid the
These Phase data after disease. presented liver in Takeda. of of This AASLD we our of biomarkers reductions on in alpha-X Specifically, biopsies only were is and strong Z-AAT production for treatment. the at showing for II multiple data X for us Mutant field from exciting and partner, alpha-X protein ARO-AAT months the improvements the
next We plan expect to a design a data with changes biopsy and data the of also X. XX-month and the some proposing to shortening FDA with NDA. or those the discussing potential goal over We have endpoints, the on month time to study
data ARO-ANGX, Cardiology development. and and of preparing our subsequently multiple at stages plans to their progress, Heart Phase the hosted continued data and now These presented meetings are webinars begin ARO-APOCX on the medical have and Society for the the we I/II European development. and plans including studies to For next of American to future new KOL meetings, discuss Association we
we fact, development review. time and filed getting and IND IIb where for to ARO-ANGX to key intend believe, ARO-AAT, event. lines are already and the proceed regulatory become have FDA clearer. with ARO-ANGX ARO-APOCX a is points and pending an This, a study all In potential Phase path I
execute that For suggested and clarity, drugs we the to There's as have in all advancement direct required each data a clear the programs, X multiple are do. designed they we long increased sight doing as new regulatory continue to the a lines for also line data disease have to therapeutics. products potential for are what With continue to commercial need to of support date generated may soon efficiently. have the time
By Phase the design, have with ARO-AAT and to on Phase intend and the for the then XXXX ARO-APOCX, expect year, we the engage with file healthy preliminary be we to one, first later study; confidence of of open-label pending following: NASH. an from three, patients middle review, suspected and on of data for to potentially volunteers a plan IND ARO-ANGX; about syndrome; studies size NASH report IIb and begin III In FDA track and review, biopsy potentially in Phase X four, chylomicronemia potentially in on half the with a endpoints, level ARO-AAT. dose-finding treatment familial a results of high of a streamlined study study study FDA clinical year, for and in XX-month We following begin ARO-HSD share duration both. of FDA we two, the IIb patients
next depth to the the using there's validation this data we to Silencing looking expect genetic We in need decrease have what of against to this is target HSDXXBXX. effect for a company given would first see not unmet for the in the simply fat. is evaluate of be liver clinical eager steps Therefore, to duration and any data against clearly the so inhibiting are substantial program. these strong disease. NASH, biology and target, HSD The possible we confer expect target, protective modality liver are thought is for we knockdown. But and
to to been has expect of ARO-HSD second and X hepatocyte-targeted one, be number ARO-ENaC, parallel expect the studies. like IIb short questions ARO-APOCX a could ARO-ANGX we on address the We three, studies with drug case in following: In for Phase and the -- will for described. potentially programs label other the the are we we studies year, begin IIb of already a studies for collect our that smaller candidates. XXXX of ARO-HSD; begin the I've answer drug would as for XX-month ARO-HIFX open-label that biopsies and number from in potent run open in candidate Phase both potentially and report our a two, study of ARO-AAT; half and that There
now regular to we readouts Hamilton. So to turn studies over expect the for overview, With Dr. I'd the like James? longer programs are James blinded while these ongoing. that call data