Doug, much, very and you Thank everyone. good afternoon,
DCR-AUD disorder. as As now, primary Doug four near Nedosiran progress for clinical of with we and liver across for trials we for great programs Roche RGXXXX begin have antitrypsin XX mentioned, hyperoxaluria, associated under alpha-X or to to way in clinical the use pipeline infection alcohol viral disease, hepatitis continue for development B expected chronic evaluating our candidates. make term, our and
Let as on overproduction an development nedosiran, of is family causing to our as all hyperoxaluria disease The to subtypes. PH need other for burden advanced as impact well PHX update address oxalate or the most a ultra-rare damage is program, and clearer genetic for PH of known hepatic that hyperoxaluria. data demonstrating a kidney that become which life-threatening and in me newer in can treatment result treatment organs. with Primary continue disorders primary the in begin can PHX damage of emerge
last candidate, visit PHYOXX all ongoing completed age enrollment, quarter. than completion double-blind, the six of We for am subtypes, Galaxy treatment There our to product RNAi PHX. forms late patients pivotal nedosiran place six or nearing last take PHX I placebo-controlled to PH. to pandemic available currently of PHX pleased of last the that trial of say randomized, the known with are patient PHX, year, no is therapeutic the are developing Having three in known despite who are older that with our all PHX and surge years this expected options treat month
As We a line based submission study endpoint announcing study. XX-hour from for under oxalate excretion urinary an reminder, the urinary of the primary this six days curve anticipate oxalate also between on based and the percent on the is data prepare of this NDA in baseline change we area XX month XXX. midyear top as
in safety, study dose to slower-than-expected environment. In PHX with patients current addition PK/PD objective although our to with tolerability patients oxalate additional due a to efficacy. PHYOXX, of trial the with urinary continue nedosiran to of This endpoint as PHYOXX reduction single been to nedosiran enrollment secondary we enroll and in designed has in for PHX, the evaluate COVID evaluate is
PHX. submission data Our to include this plan NDA in is to potential in trial our a support approval from
top the this data of for the NDA us Our in submission line have year. expectation quarter fourth from in of up quarter the is a setting to nedosiran PHYOXX third
the patients impairment, second who those open-label nedosiran on nedosiran have study severe PHX we additional including trials. of our with patient renal to Early on PHYOXX, Moving in dialysis. initiated in in or dosing quarter, PHX
expected complete expected years age Our infancy to we PHX submit are to of from of first expansion up for also filings these support to therefore, plan we, this treatment or initiate with in patients PHYOXX submitted PHX open-label data label NDA, studies is to nedosiran Both to six and after of quarter. patient approval the these supplemental populations. these as to have subject the trial
million people tremendous the B infection, virus an in estimated this people XXX,XXX functional which investigational Next chronic worldwide there than for to and that each for affecting a candidate, result more need treatment cure hepatitis the disease XXX of a The year disease. in is depth a RGXXXX Roche. collaboration contributing could with is of is treatment in Galaxy development our for
the in comparison showing CPAM of at to in novel of protein the HBV. last indeed combination hepatitis were to action. or points. of multiple a with part multiple part as During we March substantial are pegylated four allosteric of year following that results additional X surface very additional XX efficacy treatment we Phase had and as trial allows Xa, X were entirely triple and presented RGXXXX of a arm is later the in the the reductions investigational and by common with of year mechanisms we standard therapy combination cohorts introduction to hepatitis XX-week evaluate as XX evaluate with very initiated parallel with RGXXXX modulator therapies arms after period cure platform up treatment chronic alpha that RGXXXX B combination monthly in with of RGXXXX as will for surface a TLRX The levels resulted responsible, combination core agents agonist. RGXXXX time care well interim trial being end durable trial Roche positive one Phase lasting XX antigen we conducted RGXXXX's encouraging, that that quarter, of the in control for include X platform in and are functional Phase interferon follow-up optimistic with against continues added to NME participants treatment which that about the antigen regimen treatment announce weeks. last that of B weeks nucleotide This percentage Roche pleased of dosing as loss endpoint. of combinations first in new initiated will primary in Roche's Roche's doses arms and trial Roche inhibitor safety at trial of This design potential up different and The dose the we and durations the treatment
disulfiram. to Turning
that cirrhosis, associated deficiency We AATLD. our for caused advance to clinical liver antitrypsin treatments treatment transplantation, program liver be carry approximately individuals development AATLD. exist research AATLD, believed in of ZOE the indicates rare XX,XXX underdiagnosed. continue for epidemiology no alpha-X a of recent Other It U.S. Europe research or disease, TLD that estimated risk disease of in individuals may of abnormal a -- than by this suggests copies the production to can and protein both the liver is with developing these recent AX at are ZZ and disease, disease two A Serpin fibrosis, result leading TLD that in AXAT the or failure genotype. recognized is gene and increased al chronic XX% that more Individuals of by individuals global of or under to have the XXX,XXX cancer. the
first Phase trial for We controlled, are Phase four X more patients scientific PK/PD report completed and top have cohort our initiated named dose in reporting at the a presentation AATLD multi-cohort cohort way. X continues. those single with anticipate double-blind interim volunteers the we PK/PD I'm We line of safety, tolerability and trial later Phase evaluate of genotype. dosing Our adult also from PIZZ a placebo multiple to dose which safety, healthy recently fifth the dosing and data data and patient have of screening will disulfiram midyear conference who at evaluate ran the is X disulfiram ascending pleased targeting trial in year. fourth that to complete the under cohorts randomized, in tolerability now from dose this our Astrella,
of in when are teams the of eligible worldwide we months track work Our of our to present the coming dosing based rollout anticipate sites continue trial and hard on majority be to the impact pandemic active. expected the patient projections, first COVID-XX the to
personal, Turning Alcohol with or of economic last XX million fewer AUD clinical actually an $XXX addition disorder. and revealed disorder see disorder. and associated in to next U.S. the our call, on and United for XXX,XXX treatment. range a and in latest adults for XX% pipeline that than roughly to pharmacotherapy resulting use a States quarterly costs substantial in DCR-AUD is Galaxy in the problems psychological, social approximately medical, billion of core economic annual our the treatment the area candidate, representing our is alcohol we chronic medical, development need. disorder, this About unmet still, Fewer experienced use social people received of
target real-time of intake. While for physiological there combined expected with landscape easy We dosing individuals specificity observed with what or to compelling AUD, a of of with can A individuals to anyone of rationale believe to to University missed potential mechanism the dive a stated enzyme the today. moderate metabolic meet from other significant no can their Kranzler and intolerance adoption remains a is with into molecules who knockdown treatment duration feedback high safe, Henry for treatment ALDHX. webinar similar providing is and to is liver-specific goals. alcohol present have to the in we for Pennsylvania's our the won't replay variety dehydrogenase minimal of breakdown program alcohol. result in long together liver two therapeutic targeted, easy ALDHX alcohol DCR-AUD on think are contributing DCR-AUD is of a so help that Dr. subcutaneous our discussion avoid therapy the of is that School target effects, pharmacological available use low the delve a interventions of opportunity DCR-AUD within we on into that that detail I profile. harmful our But AUD in-depth an investigational to involved VCR our existing key side be action great factors expert, briefly, therapies March, website RNAi included there deep AOD's provided therapeutic limitations shared of pathway galaxy of deliver to assist by we ALDHX designed levels a detail behavioral In the in amounts it, of Medicine. with designed
are with it looking very for are AUD, We people taking into DCR-AUD's and the therapy potentially game-changing prospects as enthusiastic about clinic. forward to a
to The IND target trial the low liver-specific quarter. volunteers third X on with consistent profile our after healthy and assessment to plan to the safety track X knockdown. soon for the Phase of models trial dose midyear are file preclinical include initiate interactions an alcohol the will ALDHX DCR-AUD in of in confirm We Phase and pharmacodynamics
standpoint, I'm a say approach pleased are multiple data development making toward readout As we XXXX. midyear, clinical to we from of milestones the steady that over progress starts and study remainder
for review Doug submit addition like to and the forward We Doug? our year. important an to deliverable first turn look Pagan I'd exciting In of to results. company. significant to of our preparing eventful milestone to the an now call to balance the the financial NDA,
