to; can forward, for any way programs productive our gain the of of COVID I one, year don’t our made world and already developing the screening still to Arrowhead affected enrollment We to a you progress limit a take patient to year, was that our way. and and as clinical the protect provide for three, on clinical a expand on to that those Arrowhead That our uncertainty, in presents candidates. data very employees, are precision. the the proud highly and even This seriously a and COVID-XX a of way continue health the to is speed, clinical us We and the to XXXX culture am do. Even work safety patients studies. our that in we a The risk when notwithstanding, took this We’ve particular our participate some very pipeline; to joining confident participants. of. that good were health Vince. the us development and, not programs; of part thing clear. progress action that studies broadly though we order the today. find is afternoon last proof-of-concept we first news thank We in make the everyone new pause multiple drive medicines. safety decisive believe testament something innovation, material partners, toward voluntary -- business to two, challenges in in placed our be and extra-hepatic lot and obligation is a Good of new Thanks readouts of of presented has right That some is hallmark
our be I’ll order three targets products by are discovery start our stage some to Let’s goals? answer broadly The important reviewing new we and to what done achieve programs. that we included about against XXXX signed by three with in selected what Janssen have programs. plans key Janssen. potential So, in by partnership these speaking
product While can we about the that We after. disclose data we good say on because early have began we ARO-JNJX, on work was can’t the made three. progress targets, first potential on stage, all or specific and candidates, selected target previously the report talked partnership the in these soon
the now Janssen very designate We product including pre-clinical move pipeline multiple targeting are with let’s to earlier them to sufficiently for called productive, that The Xb space. stage the Staying to conducted JNJ-XXXX, ARO-HBV, our Janssen. rapidly them other platform, ARO-JNJX progress add with a in advanced at continues and utilize to pulmonary by and our has programs on which now potential been development, stage candidates Phase our for studies TRiMTM ARO-JNJX. two partnership being
Our had Pre-IND second stage. COPD designed ARO-LUNGX by undisclosed target into an from is as previously program been that it officially epithelia. after to pulmonary announced moved inhibiting a candidate in and preclinical ARO-ENaC, We treat nominated
IND-enabling additional our move progress and file ARO-LUNGX gain phase expansion for that that a multiple pipeline clinical to the on platform. rapid continue lung parallel We the continued diseases into space. on in end, in just adequately to have with year. the good of other a on for is this Fibrosis, COPD, and treated. goal multiple pulmonary are think for phase has studies are environment we to make potentially cusp not It are proof-of-concept we rich that CTA targets and Idiopathic been to of We the the track at Pulmonary opportunities a the pulmonary asthma, To We target think now. on end then work
second gain therapeutics receptor potential good coronavirus a down first time direct the strategies. knocking made targets the is is This forward we antiviral third the front that best several believe multiple and you try causes viral different mRNA; novel to One approach COVID-XX. is uses the is same treat a the that we close a way I anti-inflammatory pursue it’s that a to the address to also will, have are difficult we pursuing to cells; at and a disclosed into multiple and if is to door, on have important. that pathways. We candidates virus This the time, progress is virus designed think by to entry
all parallel and strategy on these and this more us complete We approach a holistic multiple in pursuing shots a are virus. of gives believe goal broad, to poorly-understood
belief ARO-ENAC, and well-positioned our our the outbreaks. believe potential in our are work mid-term. Given done, What in drives near liver, confident in now lung to we and in do, future to we the virus that value in experience are role the seek we we be of in pipeline additional play continue lung in and COVID-XX have the and emerging corona addressing engine possibly can are that our do HBV, rapidly, substantial programs, With the a suite progressing I to lung. an the ARO-LUNGX, to that candidates COVID-XX
ARO-AAT, against Now I our X/X will start liver with rare moving antitrypsin our candidate Phase alpha-X clinical pipeline, disease genetic a with associated to on deficiency.
We before repeat year. biopsies have cohort the analyzed XXXX six-month open-label now enrolled, the the the dosed, fully This to being are study. for and end and collected Biopsies important of present data of is what plan first at it will therapeutic targeted will be and important understanding a happens are hepatocyte the and treated. alpha-X the step after for data in at level disease liver be progress readout. an patients important first an It we
is the measures Z-AAT protein. most of focused of the direct That to alpha-X mutant ability change from inhibit production faulty but at liver looking many We of the in baselines monomer. will on measure a drug’s be disease,
We there. have a in high to our of ability confidence level show improvement
treatment. don’t company to that don’t humans, show believe drug our in months working in Remember, that this the about X/X exposure be but are time first measures of talk to disease would improvement around will we likely that If time what cause earlier also investigate a see Phase receive content regulators assessing other That programs. two will study, consider result longer effect. require we than signs will the therapy to we We inflammation. now the with at expect. I polymer exciting enough patients to includes will points that to of SEQUOIA of so We study. is us Z-AAT cardiometabolic be a to know parameters really and could and of treatment changes, see the very expected, change six years
a utilizing program and developing expertise support valuable pipeline in therapeutics Arrowhead’s platform has continue innovative candidates. partnered the AMG wholly-owned treatment we expertise we candidate see in are advance. study for build Amgen excited We for These that this and week with a last the of payment. that represent forward announced licensed to XXX, the cardiovascular Amgen to both Amgen drug and triggered Phase program, of medicines milestone to AMG cardiovascular and X and targeting of Lp(a) strategy $XXm disease. start Let’s commercializing a extensive steps RNAi the to XXX our and important started
the progressing points, Our readouts year two development. stages are including multiple and wholly-owned advancement ARO-APOCX value-inflection ARO-ANGX, cardiometabolic both into of towards next data clinical candidates, this rapidly and
us next second XXXX dose two The enough both of duration over types to first-in-human and volunteers potential assessments. response, data data be effect, populations. or clinical Phase and is few Both for an give This to discuss to include in up to both of safety and Both at readouts development, patients including single are and engage half together therapy. from programs different actionable XXX information It specific programs. study us design candidates healthy studies, conferences time enrolled about of also of candidate have multiple-dose patient tolerability, yields regulators accelerated patients. stages X/X allows months. next important and with the respond nearly studies. the gives have registrational and It how going and studies various And, on both to cohorts each three
take the is have shape. already been communicating beginning to We and our ARO-APOCX on plan FDA with
We European gain will authorities and target design with to engage the on discussions ARO-ANGX characteristics, on patient study similar various and populations. clarity have endpoints also
to high on a of characteristics optionality provide patient and to assess level candidates on how both the stage to studies disease candidate's utility. Importantly and focus populations clinical the which
genetically above of acceptable that size design would own of in patients in characteristics with also in range triglycerides high which with respect clinical prevalent duration as ARO-APOCX require various for different to per between extremely are are defined, potential deciliter. mildly FCS XXX elevated milligrams endpoints. deciliter thousands per triglycerides levels example a ultra-rare and study extremes such patients There have and have populations treatment of patient to each such For milligrams that these their as disease there from the
million In U.S. million than alone Taken are and to market represents a together large X than deciliter, this X the milligrams than with between with XXX XXX,XXX estimated be and adults very per triglycerides between opportunity. approximately greater million XX more more triglycerides adults triglycerides with they XXX. XXXX
fully larger breadth thinking be all specifically, with will candidates for an is therapy, Of and for an as see LDL-C, beginning metabolic other don’t opportunity these ARO-ANGX ARO-ANGX. investors of as -- opportunities measures opportunities patients candidate commercial dyslipidemias triglycerides, potential patients the This I This potential of these paths commercialization. orphan and of to solely appreciate of the size the for we course drug disease. and is both exists not ARO-APOCX cardiovascular but for to also ARO-APOCX ahead. missing and are indication potential time of exciting programs type of mixed think
about very could paths be to patients the large opportunity future, talking assess options. timelines treatment that help how investors to for these We detail provide which from level in of significant a and should the new properly populations of benefit will is
a disease. good to We We liver ARO-HSD, in X/X have progress study Phase treat on also in in March. related development and alcohol began dosing made non-alcohol
pause we affect but cohorts completed protein enroll so we program short target with a anticipated measure to other The timelines. since due healthy our have COVID-XX, NASH. NASH don’t This not think HSDXXBXX, had suspected engagement. activated and will on to the a is cohorts that biopsies in volunteer be We programs, and to this a experienced have enrollment or patients all liver screening secreted collecting similar material target,
continue end As forward of position screening long of enrollment XXXX data XXXX. we the planned, in move and the present patient be generating half in as and through as to first to be should
first the our in month mucus Phase potentially this the improve inhaled of lungs We patients Cystic or volunteers expression ENaC, in clearance. dehydrated to Lastly, in help candidate start pulmonary sodium to the Fibrosis. I is reduce target CF-related anticipate mucociliary channel, and dosing with want study The in RNAi candidate ARO-ENaC, help to to mention epithelium. and healthy designed to rehydrate X/X our epithelial
insufficient week, therapies are the unmet there of or either because most U.S. treat progress XX,XXX As ENaC we alone discussed in have on still and This hope who first CF that be clinical to genotype last our readout that treatments. XXXX the are trials enable approximately of be webinar half advanced the is shown not program of or alternative great patients and in ENaC agnostic. to are have in eligible therapies We patients may and non-responders of responders. to the been need should, be alternative for their a been need decade, action exists. from This is generate mechanism estimate the us rest specific genotype a of over suffer lot data CF importantly, last theoretically, but We has we in still the there an exciting that to new data that through XXXX. significant may in think
the I’d San that and animal over humans has Javier we translation data Javier? highly our like call pulmonary to to platform. been overview, turn new With Our eager Martin. of data Dr. promising, in preclinical the are see now to to TRiMTM