us today. Vince. afternoon, joining you everyone, and Good for Thanks, thank
moment. progress will particular, and goals. We a very term that discuss during clinical steps I longer took our short we toward regulatory the In and in substantial quarter some made term important
a both candidates have will mix and for and and RNAi stage mid of programs, will now add step a cell be We Arrowhead the This targeting for later outside partnered, field. entire and we the good more broadly wholly-owned types early, big of soon liver.
multiple potentially to our cash we more milestone on non-dilutive through years access partnerships. of our payments two sheet and have capital balance addition, In external from
components Taken together, three for we ours. have success company critical a like of
discovery on to important which platform three, trials; new of a a pipeline and clinical further innovation; stage fund a of fourth in capital is a the One, critical early component execution. effective medicines; development variety to build potential the medicines and two, later spanning to rapid
last over believe years have I and few this during we clearly the quarter. past demonstrated the
long-term new focus if overriding bringing And build patients we to able will Our value. that, continue them. is to do who we're to medicines on important need
give of highlights I Before I the quarter, a a want of the some to of make announcements. of review couple
proud a thereafter. which first-in-human previously announce the for expect end this begin at am I to file studies First, to CTA we a and shortly of year, undisclosed program
candidate could disease. GLP-toxicology address ARO-HSD, The never alcohol studies. and HSDXXBXX, is and liver are program related discussed target indications we IND-enabling We have in is is it related publicly. currently The this non-alcohol potential and called
are indicate non-carriers. reduction humans, non-alcohol in acids. dehydrogenase alcohol related disease, extensively acids the bile both the studies of compared XX% with related XX% against Human of to risk approximately it hormones, hepatocytes. and metabolism genetic mutations protective involved in and loss-of-function hydroxysteroid a to fatty In expressed HSDXXBXX development HSDXXBXX, is liver in
liver in the show treatment disease. variant both lower ALT therapeutic of non to compared and Carriers effect transaminase has inspired levels, AST carriers. protective of this This interest
to I I and bring expect against any the the only this the using target we RNAi candidate first about the that but in modality target. first this into will excited the against not clinic program candidate are clinic, We expect we'll any have be
more large and and pipeline, now is a have it larger and exciting We exciting.
Analyst an York programs, program. XX of ARO-HSD hold New the will new including Arrowhead review Day of on a in Second, some October our more to in-depth give
so fast. so have come We far,
will interactive I from helpful be us where So external step more of experts, and a to we the as discussions take Arrowhead plan back investors view addition also at as We on think long-term in coming some the over see question-and-answer having company sessions. it for panel give well and a to presentations years. folks
be analysts an formats. be engage will and to presentation also event and will there institutional various open investors by with We're live event to The a planning invitation, webcast.
we webcast. many or us can you join So in by person of hope
Additional approach details we will event. be available as the on our website
now our the liver. give timing to the of targeting to update a programs. tissues turn the on want TRiM ARO-ENaC, and to enabled brief pre-clinical outside ARO-HIFX candidates Let's first I
to be being I liver us, for patients makes that value more outside for accessible addressable a set small This step antibody forward up Arrowhead. As options. broadly vast to mentioned, a able drugs big target many and us not effectively hope of and/or diseases them tissues to with the significant provide adequate give drive us, opportunity molecule and is a the without may opens for importantly, can
have to Because spent We modality true have for last many of and gives belief, must outside able of diseases and and RNAi strategic finding the over hepatocytes. to this believed the we reach This us a challenges along potential there. new companies. delivery. other we the our think a to beyond medicine improving as are solutions paradigm technical advantage years address technology distinctive that that all hepatocytes several RNAi its be We shifting technical exist
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to its growth. tumor that ccRCC and HIF-X is it thought are during Hippel-Landau attractive unable degrade to promoting of believe leading target a of majority tumor is the because to intervention form overwhelming Von We hypoxia mutant an the express alpha, for tumors protein accumulation
on for still a file CTA this ARO-HIFX schedule year. are to HIFX, for We
the Similar a support CTA currently The these studies. filing studies the we conducting are should to IND-enabling end by anticipated year. ARO-HSD, of of GLP-toxicology completion
Our program TRiM candidate is or sodium extra-hepatic to to production ENaC subunit therapeutic the designed the airways is second channel the reduce alpha in platform an the epithelial alpha ARO-ENaC. RNAi inhaled of ARO-ENaC lungs. of leverage
the in lead get be can to fibrosis, the increased life the hyperkalemia In called the airway cystic ENaC in potassium and able lung potentially inhibition activity threatening. that dehydration can sparing reduction contributes levels fibrosis of inhibitors kidney. while in ENaC , blood kidney reduced have been to high dangerous transport. patients, previously ENaC have not in but tried to mucociliary been enough and cystic
advantage Consistent case pipeline, prior have using our approaches where targets TRiM and is this an our with other in may ENaC distinct is mechanistic platform us. a small molecule over thus attractive another target RNAi for
silence no We multiple that expression we have expression selectively on can pulmonary demonstrated with levels. ENaC studies pre-clinical or renal serum potassium effect in it
future has anticipate In Cystic small previously the limiting inhibitors. conferences. additional at addition, for American RNAi North appears data say, molecule Needless among to are data inhaled effect, have we very to a been XXXX the much others. Fibrosis We We program. Conference, a at of presentations about longer presented factor which duration excited
has quality small doing of and number studies toxicology high bit. affected Because ability capable has inhalation a of slowed facilities are nature scheduled ARO-ENaC. get the there our in work a program specialized the of activity This to of studies, for
IND-enabling but for not to of begin a in to will expect the the end CTA they be ARO-ENaC We studies time GLP-toxicology next file done before quarter, year.
of are CTA to XXXX. guidance half the first filing on So our adjusting we
is this that mind lung. RNAi candidate our to in Keep first and therapeutic first the target the inhaled
disappointed are of to that pre-clinical substantial about are price into studies program well. animal data that we the it is go clinic with the delayed by a and ensure that amount a done quarter, while So to has small a pay the we
of Further, year, own CTA filing the sorry, - that with the filing the pipeline our new meets our a the build ARO-HSD expectations. we CTA clinical ARO-HSD exceeds by end aggressive further - to speed up continue by or at this new
more anticipate be has and with the to programs get As level clinical quickly experience lung from we Arrowhead. will we to first we validation same targeted new everybody that able speed and will expect that program, of follow come high this achieve
important to programs. will therapeutic antitrypsin stage with to in I deficiency. being start ARO-AAT, later want I some treat RNAi a stage with progress developed alpha-X genetic disease rare on, Moving our candidate clinical our associated review liver
important achieved the We regulatory two quarter. milestones during
pivotal First, registrational with the study. to X/X we a following clearance received to filing announced that of IND, FDA an Phase begin SEQUOIA as serve potential the we the trial
pivotal Importantly, study platform. the using potentially first this TRiM for Arrowhead's compound is a
is conditions to ARO-AAT the and that The secured is unmet important expedite FDA. patients drugs the drugs for new to need. purpose medical also the fill to treat review of the Fast an from Designation earlier. Fast serious development facilitate Track We designed U.S. to get Track
the of Track intend advantages We number to important the a Fast utilize provides.
received recall in designation EU also that announced may You we the that and orphan the U.S. ARO-AAT previously both
In addition pushed these with studies. have regulatory to we forward key achievements, clinical the also
sites We that already multiple enrolled. are with operational have patients
is begin study sites. We along we expect open-label well, open those to The also moving week. XXXX where to this continue
talk We enrollment Bruce these will expect shortly. studies to the begin about a moment. in status of
things we patients in few patients XX with ARO-HPV triple enrolled being has program been continued development B chronic collaboration XXXX and announced JNJ-XXXX. The include received JNJ-XXXX. to advance. the planned have a All combination in In want study I developed the that hepatitis all Janssen, new now was doses ARO-HBV April, clinical called infection. about expanded to a with XX to mention of program and cohort XX have cohort,
orally administered of payment. earned and, structures, forms capsid in cohort JNJ-XXXX, This Arrowhead that JNJ-XXXX, the assembly normal XX, the with Janssen's of cohort milestone modulator a million dosing class of $XX includes a investigational NUC, capsid start connection
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be RNAi with agreement for selected to option our research potentially October up against a of collaboration, by new included an agreement Janssen to Janssen. additional XXXX Part targets to three therapeutics collaborate
first leverage and actively targets include working undisclosed, never platform candidate Arrowhead's against These current do expressed our referred potential new pipeline. an proprietary as to target. now in the are on ARO-JNJX We TRiM not candidates
to Arrowhead an is IND license. the filing Janssen, and by development sufficient responsible exclusive to option perform will the allow Jansen of equivalent, pre-clinical discovery entirely at the take have time which or optimization U.S. funded to
medicines novel is responsible This create commercialization. drug and be Janssen option RNAi by the is Janssen's Arrowhead's and and exercised, speed will leveraging an in and development capabilities. wholly discovery If clinical opportunity important expertise development clinical commercial for to
forward potentially a have on this further Janssen with rapid X progress programs. other We made two we on to and look ARO-JNJ working and program
Let's investigated AMG-XXX is lipoprotein(a) Amgen move progress treatment our ARO-LPA. on also cardiovascular as The known now LP(a). a called formerly to continue to disease. partnership. make Amgen AMG-XXX, for targets being as potential
been development year with of trigger this late first Amgen to next of expects patients X would has half also data Amgen enrolling study in share the and development LP(a) which in launching payments. phase a of year. XXXX, or the Phase next a early elevated anticipates milestone the AMG-XXX initial
disease. We one day an important and could AMG-XXX program share this in cardiovascular treatment believe new excitement be that for Amgen's
also of target, received an a therapy terms Amgen called option our against which agreement, ARO-AMGX. cardiovascular exclusive the subsequently worldwide undisclosed an we September On RNAi an for XXXX license to
we'll ARO-AMGX the to option period agreement. In expires that August Arrowhead activity XXXX, a collaboration pipeline. or and from development and the our exceeded XXXX, has they not Amgen requirements candidate removing safety intend option. advised exercise Consequently, the on that us The X, do August in to stipulated met delivered Amgen be
Let's now we optionality cardiometabolic provide wholly-owned our and populations both indications move These will candidates ARO-ANGX. with patient to some pursue. and two respect targets ARO-APOCX which to
diseases. well as to opportunities diseases familial orphan target, such higher each be prevalence treat and as syndrome homozygous familial there chylomicronemia as may hypercholesterolemia, For well-defined
of In quite for to a duration are readout patient a our provide optionality on both designed enhancing early volunteers the Phase and effect populations, candidates various addition, as well look and safety healthy stage. at X in the robust at an further tolerability, pharmacologic both and as study activity
for designation the To drug hypercholesterolemia the familial of ARO-APOCX that FCS orphan familial from for and syndrome we end, of the FDA or treatment treatment secured HoFH. chylomicronemia for for homozygous or ARO-ANGX
Our intention studies these and is indications initiate potentially and year. both pursue to orphan ANGX next immediately pivotal ARO-APOCX for
approach go-to-market this possibility plan - market to populations, these allows growth us enabling development we in where parallel that clinical Beyond to like will staged longer for larger we markets. mature. into pursue take We also also model. quickly It disease a to involving study rare and of getting allows indications while larger studies the other
a single drugs course candidates ANGX just From drugs. of standpoint, these each. standpoint, chemical like and From are however, multiple APOCX end economic any one could entity drug market behave
As we total studies expect addressable do different two support for increase treatment we to our substantially multiple to marketing markets. indications
Importantly, the others to area the indication contribute candidate. database a because benefits will new addition single each each of safety they for all
So clear leverage there is here.
So of patients ARO-APOCX, some For with with what opportunities? history triglycerides could these pancreatitis. be of are some larger market elevated simply it
For ARO-ANGX, there possibilities. are many
resistance, fat also decrease LDL NASH, patients other metabolic goal to we could treat while NAFLD, those syndrome. to FH heterozygous cholesterol help patients among we with not ARO-ANGX liver Because insulin and may who treat look For look instance, we those statins. meeting could or with on and their things, are expect with
opportunity We have uniquely is believe number a and diverse a ANGPTLX, a to substantial that we of patients, powerful large help target.
As at RNAi both are reminder, the candidates and currently. stage essentially schedule same developing against and, the two I clinical only and These on the APOCX ANGPTLX. programs a believe, we're same first
We are patient various in portions single dose ascending of in and populations. studies the dose enrolling multiple completed a in dosing both portions now
for readouts year starting are still schedule data next continuing on year. and potential likely We into this
ANGX September the programs talking a we XX. and line have speaker Specifically, & and taking our generated. and keynote some Bruce data will Dubai in place APOCX Summit will On will top be Disease Cardiology Heart be about He include at Global clinical on XX
through Later fuller programs. data to we data so have we will AHA studies, Heart expect ANGX clinical abstract November. American late are from a additional in and conference. accepted, present These this set breaker submit month, November we will conference the for readouts APOCX the believe we the Association a rich If
data Conference We will then the in American at International Cardiology abstracts of submit expect EASL therefore to Liver present the meeting to College more April. and/or
skeletal At mentioned targeting muscle cells. year, we our day R&D last in breakthrough a
We this clinic. path entering the to first have designating getting continued down our target closer in and are
with next We to clinic we to enter discuss the the are data our but potential candidate muscle see prepared not year. first today, targeted
With now I that Given, to to Bruce? over overview, Dr. call the like Bruce turn
