DRNA - Earnings call transcript Q1 2019 | Dicerna Pharmaceuticals Inc

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Operator

Good afternoon, ladies and gentlemen, and welcome to the Dicerna Pharmaceuticals First Quarter 2019 Earnings Conference Call. [Operator Instructions].

As a reminder, this conference is being recorded at the company's request. I will now turn the call over to our host, Kendra Packard, representing Dicerna Pharmaceuticals. Please go ahead.

Kendra Packard

Operator. you, Thank and First everyone, Conference for us joining Good XXXX thank Financial Dicerna's Quarter Highlights Results for afternoon, you and Call. Operational

review Media a who a which close is anyone not the our our today, to results, Investors website under & www.dicerna.com. For of trading we on release press has issued available after tab the chance had at

hours which days, this also call via X website, listen beginning our XX for to conference the completed. will may is archived You on be call after approximately webcast

review discuss Speaking update President results. on for today's and preclinical Jack CEO, call our an pipeline will Then will clinical on be Doug first our Fambrough, progress activities. quarter who our provide CFO, development milestones financial key and and XXXX and our will Green,

filed section Boehringer usage. Dicerna's from expectations of results regarding and the including, we making our timeline will example, guidance collaborators management other call differ your the for of XX-Q remarks, preparations listeners cash remind factors, program including related disease call, Alexion, questions. as be and with important XX-K potential with pipeline to various Dicerna's SEC. for our of the will programs, possible and today's to I'd for these the discussions expectations Factors on and latest with for in Ingelheim, DCR-PHXC, opportunities like Following DCR-HBVS, materially development forward-looking undisclosed that rare plans by Risk those partners result Lilly, indicated expected may statements Actual and forward-looking statements a and discussed those open Forms

any views to future, statements at change. we do these so we While specifically point some forward-looking our update in to if may the obligation disclaim elect

President over to to turn and the Dicerna's Now I'd like CEO. Fambrough, Doug call

Douglas Fambrough

utilizing been Officer; novel our joining to Chief work this rare diseases, technology RNAi has currently disease We our Medical Officer. Financial of us programs Thank platform to Dicerna an Good our With of broad with are our today Green, for diseases and on for undisclosed three build initiated the thanks call. Chief hard and chronic Ralf on broad Jack liver. liver Rosskamp, have research and is me afternoon, a you This array proprietary all at GalXC exploratory a pipeline in you, Kendra. collaborators. of programs discovery development top program. corporate involving internal DCR-PHXC, in DCR-HBVS rare conjunction and and our

per PHX milligram XX, including day call Based of X.XX of for trial, had is clear with than which type defined participants kilogram the defined One with normal PH normalization reduction a and participant near level, the and PH, not time X at of XX% urinary or a achieved or X reduction maximal into to at in least participant the X -- have A led normalization, kilogram post-dose three with of the the excretion oxalate of or these clinical patients of dose the as the monthly I we molecule development the progress of and I XX treat forms equal cohort. the XX-hour only for excretion program this show most We we in X.X by working than beyond pivotal for the greater patients participants cohort a as in and of reached less in liver. Among advancement is oxalate receive of the Phase urinary PHX that are DCR-PHXC developing up, adolescent randomized reached into who dose at, was drug per cohort forms and X dosed participants with data hyperoxaluria, reached of start PH GalXC first of X PHX our XX%. XX. also millimole a treatment extend enabling per five time. XX-hour trial. more with this of clinical in PH from or milligrams X we The the at treatment like a trial with PHX, for as justifying trial X.X are of placebo-controlled double-blind One brought treatment than maximal type lead type potential Of for DCR-PHXC to potential launch. data, utility including to cohort. and PHYOXX points mean updated our prefilled in single short. RNAi DCR-PHXC less a PH urinary our showing convenient on oxalate program, reached for highlighting our in a levels of X.XX PHYOXX fixed at millimole as dose will mentioned, X.X XX-hour to XX% primary we mean all mean our PHYOXX platform I'd PH. have product in normalization shortly quarter, advance this protocol, near candidate note, Adult post-dose regimen, March subjects syringes X. oxalate in follow and of X dosed day DCR-PHXC announced all after proof we believe in maximal urinary compound X. results, a of approximately as pivotal milligrams participant XX is is the concept at XXXX, X kilogram reduction to second of That single range, still

sites activated screening initiate patients quarter. patients currently dosing We to are our of this and expect at

achieved and treatment later with a PH of PHYOXX the alignment approval endpoint QX agreement the trial the FDA meeting type for with full the on primary pivotal we Type for X. regarding patients the A XXXX, to in the at Importantly, FDA with path

for plan trial, chronic treatment B DCR-PHXC, on PHYOXX is PHYOXX of trial extension trials hepatitis PH pediatric Phase an initiate we In dose program DCR-HBVS PH turn program this Later approval in trial. initiated from enroll will development in expect PH type and patients clinical patients later, patients the we I plan including clinical in quarter is for in schedule, dosing on trials A that DCR-HBVS. volunteer to we to our PHYOXX continuing later utilizes trial DCR-PHXC, PHYOXX study of with healthy X. X other to broader of group healthy are convenient discussions pharmacokinetics have portion the And monthly open-label our a dosing the DCR-HBVS. X, initiate of enrolling We infection. first to the Based quarter. the clinical our is enrolling portion, portion for placebo-controlled this of we patients type participated PHYOXX. pivotal off Pursuant group safety, quarter, now dose to of renal path also PH the Phase volunteer our employed program the studies. single-ascending virus a tolerability dosing in coming of trial assess PHYOXX the with the type to disease. first the end-stage regimen XXXX, in which multi-dose fourth study. fixed additional I I of for we'll data to patients the and The patients and

data dosing patients dose DCR-HBVS portion far, We XX this dosing X at continue at us chronic which least in to fourth this includes The is of will half B site the the of year, C dose mild kilogram X.X follow allow monthly cohorts XXXX. resolved year the on Group milligrams per group the of the expect months background milligram we These initiate we testing trial group patient have dose trial had in a which first see their months in cohort and of doses only X.X ups, the in initiate Later patients to observed injection should dosing in Consequently, dosing of first higher and adverse trial. X.X from this quarter X X.X, available HBV nuke expect of a infection. trial, reaction, with effect will at to us so the in therapy. we lines is levels to X milligrams escalation one C absence newly DCR-HBVS within to been the other monotherapy Based duration of been and a pharmacodynamic DCR-HBVS the the quarter, single-dose explore and placebo-controlled of initiate of kilogram the per trial of period This not who infection. which have of allow events diagnosed cleared kilogram. HBV portion on chronic multiple-ascending near the in any per so therapy. patients of with hours. far X.X serious dosed dose with term

we for way this antigen, the B we trial believe I to later for With of and of a viral data, I. DCR-HBVS Phase potential for proof concept beyond combination notably chronic Taken the generate treatment this of program trials development S has with hepatitis Phase clinical other and pave in further intend to will development suppressing action the partner collaboration mechanisms hepatitis of HBV. program seek antigens, the together, for the proof-of-concept

serious Moving to targeting liver program, undisclosed a disease genetic our rare program declared disease. third our

along exploratory CTA, schedule proprietary X that with on and Clinical which quarter and development GalXC-based to programs, are at Trial We reveal programs, that of company. details are around we reintegrating early-stage and more forward These file building a pipeline a time. this the strong form or Application, will about programs program

commercialization to develop DCR-PHXC as pursue opportunistically on these disease commercial collaborations or which maintain we For own and the programs we program, programs, a our such through role. rare substantial undisclosed intend in our

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several has averages. driven of we program these is a industry and relative Our date by clinical selection what including medical to programs been unmet of success criteria, to high believe need probability

to the into the entered the For as collaborations like to at meet development In stage. to therapies the we relationships. programs recap and partners corporate program internal and continue technology. liver-targeted on our strategic our pipeline pipeline these we discovery strategy date, development executing opportunities executing not strategy mature GalXC active seek I'd now on on move We do criteria, progress based beyond plan X with that and tissues. other this have

responsible milestone initial for our work million XXXX collaboration XXXX, these of BI in initiated X the look single commercialization of development to was NASH. a with chronic in the BI in for development is First, collaboration XXXX, with with are liver exercised add payments fourth additional providing receive major quarter collaboration This option target second quarter disease of collaboration, of while exploring commercialization for quarter starting royalties. to liver the I to future. a Boehringer are us. we future or in with the and this we on the targets. with a Alexion, our of eligible in Late short, the the treatment target Ingelheim, sales alliance of starting $X recently, clinical to its fourth a liver More updates BI the and triggering on With a payment fourth we with collaboration to and programs, entered complement Alexion pathway, Lilly. into with targets use technology and Eli GalXC forward gene.

Alexion to receive As programs, responsible and is commercialization of development BI, sales payments and future while eligible with and clinical royalties. these are milestone for we development commercial

discovery with cardiometabolic neurodegeneration Lilly will early including X targets collaboration, To programs alliance these have technology of involving beyond cardiometabolic neurological liver-targeted as as well been we specifically working using to both versions of liver, these with efforts, of active. multiple this delivered GalXC newer technology to encompasses X GalXC and collaborations, therapeutic Under date, our CNS Our areas technology very disease, cardiometabolic months tissues the the under GalXC. of be pain. GalXC to have our additional and apply we development applying GalXC The collaborations tissues. initiated and liver-targeted on collaborative

in discovery collaborative our robustness efficiency, the illustrates methods. these drive discovery simultaneously to ability of GalXC and programs optimization reproducibility Our and

we capabilities With in the Lilly BI, molecules a diverse partners broad development advancement pipeline forward and and well of therapeutic as our future, to potentially as the across GalXC look of combined areas. Alexion of additional partners Dicerna the

Finally, in key I am to terms of building people our some team. pleased welcome to our organization,

and appointed Anna studies operations Officer Achneck for important at Healthcare Therapeutics; our Executive we Chair Executive development welcomed executing are figures with you strategic chair. pipeline. a of our Achneck clinical has the addition Head clinical as of Internally, advance Consulting; Hardean of focusing All as Mr. than Vice Board forward. global Medical instrumental company we and Board decade President X, the Marc clinical as and of well-known TetraLogic Mersana our an move we strengthened provide formally have Development. our Kevin on was internal experience and in Officer Cephalon also direction as designing extensively Chief Protopapas, Buchi, and we and trials be Head of will First, of and L.E.K. industry J. of former Pharmaceuticals, as will know, Directors Chief Kozin, more proprietary

Lastly, Resources. as team Regina Human DeTore our of Paglia has Vice Senior joined President

XXXX, we advance treatment looking the of the horizon. points of several inflection Looking In on are we forward ahead term, to track remainder as near we're the much DCR-PHXC to hyperoxaluria. very primary for the on important

dose initially for PHYOXX pivotal the pivotal also second those expect dose study same in Phase patient of to We multi-dose frequency trial. the to extension expect long-term we second in quarter will begin first this at PHYOXX the and and year. our level in be of trial I year, dosed in quarter the as in Participants the PHYOXX, the this open-label rollover the

Later for what additional patients we X, like. pediatric this including PH product patients. in PHYOXX in such, trials clinical a expect As renal DCR-PHXC multi-dose an and to initiate aimed data that at expanded look should for supporting label, in study data year, type proxy end-stage PHYOXX a disease patients a plan study will the trial as we study the with serve

for the dose to this patient to in quarter volunteers continue Phase healthy DCR-HBVS expect clinical HBV first second dose the trial infection, of I treatment patients the Regarding XXXX. with of we of chronic and

already As of quarter anticipate to proof-of-concept be fourth available human during we XXXX. data the noted,

submit on disease a that program rare also provide will for We will quarter our program at and CTA undisclosed time. this the details

Tax tax Incentive to $X.X we amount want Massachusetts I incentives that, to This Jack. new the Dicerna our April, hiring for over Sciences Program. employees based XXXX is the were year. million that calendar on with pass Life incentive in Center's was Finally, commitment the call of the XXXX financial to through notified in front, awarded move And

John Green

our first quarter per XX-Q share Net you, or were the R&D and the as clinical expenses. manufacturing programs, a quarter the our details. DCR-PHXC operating briefly offset clinical in study loss million to increase XXXX. compared platform-related period DCR-HBVS $X.XX in well same compared in was partially March increase million $X.X $X.X to $XX.X to $X.X to for with million million to the to associated by million driven net The support $X.X costs. additional to per study associated increase $XX.X the million studies, increase in primarily expenses expenses of in first was Thank $X.X for as with a The direct and activities the a increased increase in XX, results million key XXXX. costs $XX.X million by year-to-year Doug. due in was I'd $X.XX loss was quarter increased ended decrease XXXX financial This as due share support increase in you million for summarize like increase nonclinical filing the a million for a in employee-related XXXX expenses our or $X.X with growth. headcount $XX.X costs

and continue as the Lilly activities, to administrative for preclinical foreseeable as expenses the candidates the The and activities associated the business new advances increase company development were the quarter in for manufacturing for support future, in as with expense $X.X preparation product new general XXXX development million agreements. Alexion Sarbanes-Oxley clinical primarily for product standards $X.X compliance studies, and development a expense. for XXX(b) $X.X completes research in million and to the business compared to to increase of increased well stock-based activities lead expenses increase million support is accounting increases overall headcount for XXXX. to its due continues and under with with expenses accounting an primarily due planned due implementation first clinical initiatives and consulting Consulting in increases quarter increase XXXX. first and in $XXX,XXX largely expect and compensation to General employee-related expense and toxicology to increased in We XXXX

the market-readiness XXXX settled settlement investing the We staffing with and XXXX XXXX expenses as the quarter January XXXX. The expect to XXXX. million under due quarter trade in to in final first payment compared activities. litigation, of compared no to was was expenses first were in quarter $X.X G&A increase litigation secret There The expenses largely made the and second which to the solely related XXXX, in XXXX. in

recognized quarter BI in with in and from first Alexion XXXX. XXXX, Lilly, quarter from the revenue as our we During to million collaboration agreements $X.X million $X.X BI collaboration compared first the the of

the quarter For first the recognized Alexion in revenue $XXX,XXX XXXX. collaboration, of we of

premium million $X.X upfront million revenue deferred the had of million as on the XX, as the recognized in less this since on October Alexion's XXXX, years. the payment, we March collaboration The of over next revenue will received revenue from current. balance the revenue deferred of be of X million of was the $XX $XX.X resulted sheet, as initiation Most well deferred equity recognized which of investment milestones XXXX. classified As $X.X

we first of premium collaboration, Lilly along deferred revenue was upfront million payment in as the be of consisting with the the revenue For million price to on recognized quarter aggregate the The investment research $XXX transaction $XXX,XXX amount the the XXXX. programs. initial recognized equity over the of recorded of term Lilly's $XX.X

next $XX.X the As revenue of XXXX, revenue will as most Alexion X deferred of recognized collaboration, $XXX.X classified was be March million, current. of XX, million this which the Like was balance years. over deferred the

on the targets. For the BI BI million $X.X of X first collaboration, quarter recognized we revenue XXXX of the in

which as of was million the BI the balance XX, classified current. of $X program XXXX, million, $X.X on As March was deferred revenue

pipeline as had of additional in had million As and trials investments, and in cash along HBV XXXX. assets. XX, By we of investments PH $XXX.X with December clinical cash advance March held-to-maturity which to cash, $XXX.X for us enables equivalents million XX, we comparison, infection XXXX, and

costs included positive our XXXX, $XX.X and clinical operating fund financing of studies. and initial which XXXX, assume funding which fees option external from beyond and undisclosed and plan to for and program from estimates net significant late-stage unanticipated million of filings, We cash advancing in equivalents collaborations. HBV cash expenses. current the received in with sufficient operations payments collaboration development the $XX.X and no regulatory we rare For will held-to-maturity XXXX advancing from our clinical no additional investments proceeds participants our that proof-of-concept clinical had new our from includes from through disease or million, through first DCR-PHXC and completing and execution changes studies events agreements These upfront cash, of believe quarter DCR-HBVS QX be

Excluding million and the studies our XXXX received from $XXX regulatory million our continue XXXX advance million any net as range proceeds increase will of our clinical $XXX in DCR-PHXC. any QX will collaborations, $XX.X operating for of cash commercialization proceeds from we prepare expect the collaborations and be in we and filings new use and to to of that for

to I'll call on turn remarks. the to some move Before Doug closing we Doug? Q&A, over for

Douglas Fambrough

PHYOXX patients Later fourth our HBV financial programs our rare execution reiterate us. initiating our our PH eagerly I you, and trials joining collaborations. we program, disease strength milestones, a PHYOXX with and with clinical for am growth line reporting the To pleased dosing at be Dicerna, we our and the operator, virus the the CTA you, Phase our program. anticipate focused execute in remain of quarter B proof-of-concept There's treatment undisclosed year, as anticipate very for should for with a open multi-dose and with on Thank advancement please results chronic we and dosing to And on for we of be the of Thank and Q&A. patients open-label providing a hepatitis PH, filing excited quarter, in I patients second first DCR-HBVS a noteworthy extension continued pipeline. lot the Jack. for quarter, PHYOXX the study. in and of about again, our initiating in DCR-HBVS position look our first year. data in infection at It and for

Operator

[Operator Instructions].

Umer from Evercore. from Raffat is question first Your

Jonathan Miller

if so data Miller. many haven't Jon that on like PHYOXX, indication right. patients, is you Can think trial? all patients you few that's well? much from you This get the PHX ask how PHX with those there to talk patients patients -- a for how I'd seen questions expect bit Starting dosed you to many the as a far. order have in the little many you And need to we in

Ralf Rosskamp

many protocol the study, we have the PHYOXX PHX not so XX Yes, -- will we out specific X ongoing PHX. patients have how be study, doesn't many how have patients. our a patients, and number From PHX of

XX%, disease, the a like the XX% expect something I trial. in prevalence and of PHYOXX So would similar see to reflects number that the

Jonathan Miller

I'd about a transition bit flat to the ask to also like dosing. little

decision mentioned the you you'll prefilled And So your move to potential launch. What's be convenient there? what syringes dosing, what factors likelihood with the the to syringe prefilled was And driving A? are after shortly -- to flat for gating able did versus launch not? that

Douglas Fambrough

dose several us to So the in led there weight-based opposed dosing as are to I focus on reasons Phase which fixed done was monthly trial. as

patient and if as lifetime kidney A a levels. missed was product. and you hyper other to aware that convenience hydration, was there to in treatments into efficacy be chronic With situation you you're is a -- there that avoid and think allow of dosing to I was that instances a levels where felt important a the with regimen. potentially the potentially metabolite a citrate, to dose, of dosing know, wanted really potential and oxalate, unnoted the your damage. for rebound do of causing rebound, interval of a And with maximizing not if had dose off and patients that a unmanaged disease-management as would regimen, oxalate which example, therapy by some sometimes come it be reduced could would patient one concern with of do There and zone drug patients very was with PH, missed. well. disease-management safe pushed we had And for own onerous of some the to And interval a long

seems into seems robustness in is we going dosing effect rebound such dose say that the on missing the Yet additivity quarter. dose, XXX dose a it but what a month -- multiple a So at to small allowed the provide would in that contributing of mg. of substantial based built could with any our the month, duration corresponds like also regimen bit given period, given the than fact, safe drug result effect you -- time more modeling two the miss of not longer to of probably a relatively high two, us to doses quite It go at monthly duration by is a a going to because a since any in given monthly, protocol, in moment.

of for XX-kilogram is if a any dose poster or that our the effect if that for one from recently, we you example, -- So lasting at full at adult, for presented data effect, of about near X months. look it you could updated duration appears

X given one So that's X.X of a adult, if more you've got milligrams equivalent about X at like doses time. XX-kilogram months, dose contributing for

robustness. with us a effect allowed So high a it provide to for level dose maximum

In package at this is that long this. to special not that beyond in of our syringes, that a list period way don't addition, CMC something isn't certainly we to It's process goal is per customization that the just at With to I'm promise prefilled launch, dose make the that a any a required fixed but there maybe time, a the CMC through regulatory convenience because rapid nothing it have is patient. to requirements required for respect ready that provides hit of time. in quite goal.

expect So if then shortly not be do at it launch to we thereafter.

Jonathan Miller

one sense. Makes I more, guess. Great. Then

wonder week, was You study XX point. you the potential the sample patients. of the expected for I potential need. of PHYOXX acceleration And mentioned the trial a an think, that readout? mentioned And size press in for And in in of release reestimation number is patients for that planned you with I your last there any interim there reduction that is a have at in a you

Ralf Rosskamp

This early the CMO. stopping. only Ralf, reestimation for and is size The not is sample

at enough XX patients patients. will are stop result be be more need result the the the or So patients. that early will that we But not we XX

Operator

is from from question Leerink. SVB next Your Foroohar Mani

Mani Foroohar

PH, step this think just in lot A HBV take give readout concept on a to one about I want point, proof When what is of like would second. you us focus a the for at but back sense of land look the answer -- to victory your to what looks the like help your mind, inside? us reduction? what or it right understand your are Just what goalposts log that -- in mind. Is success you're in trying is a

Douglas Fambrough

Sure, We reduction antigen. multi-mechanism activity RNA an lot combination in HBV response. achieving is patient's in Mani. S productive role had mechanisms the of have interference other form immune is the critical believe That that of a system the a approach is in allowing a to challenge and a to likely

our key selection of in S been to virus. it within we antigen focus And the So reduction metric on focused achieving proof have as is primary our site that concept. we're trying impact. the driven That's

a threshold exceed amount sets this point don't patients a certainly S at So can. prior judged any I relative we hope that And of really, is the there if trials observe There we is to. is be the in of beyond a we're that certainly, could But that data In event, single reduction do a disappointment, in well as it significant. field RNAi think is what other from we trial. a best -- but antigen known big I go baseline the will would to log. log, we that in trying be of don't it'll

Operator

from next Stephen Stifel. Willey from Your is question

Stephen Willey

positive there patient group patients not remind of respect or or to and B are just criteria C this us to or kind predefined antigen some group e cap status, with Can you on within eligibility agnostic is are negative? whether you

Ralf Rosskamp

whether e so patient, between where patient negative. we think our negative Yes. And or to coming it. of There are difference action there or be positive a will e positive a we this is is mechanism no agnostic don't is allocation from e

Stephen Willey

maybe Okay. on cccDNA. I think for follow-up interest easel some and a And not that has is thought around then with happening great that there maybe just a S been a of what's question. surrogate antigen is know increasing lot I cccDNA, the prior there exiting

this So be that to sense other be in that antigen? do in could get interested than that strategic -- more term, partners are biomarkers do going there in looking you reduction? you're any guess, beyond at I antigen are S the interested And S the over they're product just the longer trial, this

Douglas Fambrough

markers, look at levels look So as of antigen Steve, look array a so at number the patients. X markers, patient And course, standard of well. and conversion at e of viral we'll we'll and the we in DNA

to in antigen the question, mechanisms. in or a do is RNA are siRNA reduction believe is it But the a combination. to mentioned could role on focused interference of as going answer we take that I what Mani's that combination do I as So primarily S

we the a agonist with agent, response be you're toll-like mechanism. perhaps important I partners large to -- view on you is same with another stimulate able to S are acting reduction and with a to strategic us response S I immune to if to end And as patients. the here page to a immunomodulatory that a a with up host in by the or part an and tend think in think productive immune tolerizing probably of goal an substantial -- going need receptor

and think to important good uniquely multipronged don't HBV. we of the reduction RNAi approach very the think we of virus, at. S it's and as, So answer what we it's to the a itself, in antigen a is see But part

agent. And it role S data be a have order likely you don't the including agent not the a strategics going opposed supports as in majority believe think probably RNAi to amongst that think knows date but that's a data playing to Obviously, to to I strong date RNAi be we're than effect. of the I alone, one that the people think I reducing positive viewed the antigen likely that as the field supports do to sole the is for also to strategics. in think true. certain, more need I but the it's no can notion So

Operator

is Your from Cowen. next Margine question from Irina

Irina Margine

patients Congrats into progress. many already how have committed rollover you to Have on the said PHYOXX? the guys

Ralf Rosskamp

No.

have We not.

press our we release. have As in said

dosing study. those into long-term first are of this patients quarter, -- extension in the later who ongoing This the anticipate our quarter study we going

extension substantial will majority we the disappointed know of an shown oxalate have, if all long-term go many and because the excellent safety exactly urinary very reductions profile. but the single-dose patients administration, into be of extension patients study, after don't the So go I already into would how they not long-term

So therefore, those I my majority assumption into point. would be of study. will that But any long-term the don't at this the extension data have be patients going

Irina Margine

they will Or the right. dose dose respective dosing switches to had since... All with And received continue their everyone that everyone before? fixed

Ralf Rosskamp

Everyone will fixed dose. switch to the

a will going what be double-blind randomized trial. will of the proxy this in be So on trial

Irina Margine

Right.

order patients, could is in agree us Okay. for data for remind additional you type And to then that still full looking PHX information to the treated just FDA the patients. again or the the of approval or so data a pathway more sets of Or for far? natural limited a function there's of it patients Is the number these seen X? combination a of they've history, a

Douglas Fambrough

asked that's they publication, been coming that's have data history natural for and another more is discuss there submitted data submit to us that it. to meeting that Knowing

already various -- in Some in the publicly publication of patients. to Day presented PHX including the the submitted relates a forums, and of outcomes is in been has that our is data November, part R&D it

also that future from optimistic after with submit we discussion in remain similar natural to be FDA the the FDA. the this from outcome data of in process registries, history be the patient will PHX, that's to collecting narratives respect So will the pipeline. we conversations With and with on we of the with are part PHX outcome that that data

Operator

Humphrey. from is question next Nash Your Robinson SunTrust from Edward

Fang-Ke Huang

is Edward. Huang on This Fang-Ke for

safety. one Just quick a on

adverse there adverse and can give details are serious So related on you mentioned us just more events trial is the what to drug. you But PHYOXX study not these events? the they in are four

Ralf Rosskamp

Yes. I recurrent fever. in can had stone, do events and the One this. There's One patient appendicitis. four a had serious adverse hospitalization due had third kidney to one to due X hospitalization patients. three

Fang-Ke Huang

as you long Okay. timing focal have to regarding Great. put have adverse it treatment events? And do to like the -- they how

Ralf Rosskamp

I after the injection. of in exactly late exact the couple don't know up They follow or late weeks the timing. a single-dose were

as them, far were So single-dose to in none recall, of a the as relationship I administration. temporal

Operator

& Arce And H.C. is Ed from question next Company. from your Wainwright

Antonio Arce

approval to patients for study. PHX getting on your Congrats in path the full

for wanted to we I timing interim over, PHYOXX. me, go just you could, both PHYOXX I first the So on for look, have us you and for mentioned a which as also But the remind you and that just acts and proxy if the cohorts complete as the the you -- essence, that had to switching the proxy discussed. in the PHYOXX, PHYOXX dose. given fixed know

two? you through walk So the for could just those line if time

Douglas Fambrough

efficiently. At in, is them would able of when so we'll data quarter, guide we're PHYOXX, determine study might We when line very patient this begin as we both for enrollment which, not shortly. this to to be top trials, enroll course, hope point, have we to So last of this available. initiating

And We us efficiently the in have will patients. report well than set more able sites beyond that enough to to sites of that we're fact, have we working that, identified trial. those ensure with have that as to another be enroll we they

how some further might tracking. period As that completed they've -- patients long that's update the we patients into in enrollment last. to X With once into we say too be on-trial get which months to ongoing is from going early that give trial, on rollover it's is an may phase, PHYOXX PHYOXX. PHYOXX, how study including that But

until I continue, guess, will that So up approval.

Antonio Arce

going the do And Are them fit one package? agency? of they submittal of to to overall patient question And the as and around sort looking be was Okay. how of later the thinking those you just patients? initial adding and groups or the the perhaps other -- more the that group around considering at What's ESRD. of in part main some pediatric of those PHX, second are with year, these

Ralf Rosskamp

is, II don't plan old, group to model will initial XX need what and younger can dosing. from of our the what we will we and know Phase in then especially, years But will terms -- regulatory in the a once Now And simulate the X we our onwards. the include study registration X group patient end be. year patients dosed decisions have

the are from we pharmacology after with renal drug the at beginning initiate find where patients am sure we the we cleared will more PK The point after what patient study, renal -- patients, will end-stage appropriate the dialysis. into that once a next initiate we so least patients the pretty timing gets multi-dose that time who trial, we'll have trial have results of So at its this I have PHX this population, of in gets and impairment for To the in criteria the on be and data we how and by that this patients. and FDA the disease, know the trial have study, amount And the initiate the way is data efficacy look end this at year inclusion so of label. The don't of a or study for same with and initiated filing, on the and with we impairment agreement clinical severe the time. severe -- later of first safety with into this study renal this single-dose a patients that initiating sufficient this label, year much some extent PHX at exclusion drug we dialysis. I -- this we will are

Antonio Arce

the to as discussions S to Ralf. the after Notwithstanding antigen field with in antigen competitive the partners to target and turning reduction And appropriate for the in question What whether data? among one target right level overall do consternation you helpful, you anyway look HBV. population. reach S Okay. me, the at to last some need look is potential think way towards the the is around you -- That's be

Douglas Fambrough

Thanks the question, Yes. for Ed.

it's that to to a then specific There one is companies think similar possible say, and approach hit. I not obviously to peer number it's a and course, have different of analyzed our bogey -- in you that's molecule different with the then different and lab, that's it's a and patients, sort a it. of of different approaching natural it's to trials and benchmark to that. point But

it's So think think would partners, to would is certainly to that also which see that's one -- be I believe out and and really collaborators going what specific I an one see finger wrong I that effective and reduction, it something understand And be to But than better. rapidly the we there to what log more greater potential is to way there. S the number. reduce substantial a want want isn't

one what don't if a RNAi with in decision want already a changes another molecule collaborator potential think a comes they or inside whether below strategic, the little to include above, tree partnered to an So that or molecule I a not. little as

functional going that's Ultimately, rigorously not functional soft target the to cures. valuable and reduction think And in going commercially right? make not not at so and just looking are And antigen combination, I come we're that the a to therapy parameter. It's cures is quantitative. successful, it's S and

And greater And to our question will be if effectively, are comfortable sort the disappointed antigen that in which combination articulating. we're So the we is substantially is, the goal going do goal log. is role it's reduction. S loose than one we not of

Antonio Arce

for little the you as to makes, S or a the is unknown out, identify? but that hard antigen a the there's of point this also the there immunological ultimate the component component variable the really, kind combination So bogey around as of functional on cure is

Douglas Fambrough

in we of clear And message and very advisors And about time strategy. noting, have antigen and substantial of focused field for drug the we our let's Yes. I really the what on worth clinical more our target focused our talking sort the have much do, it's with them antigens and that's S to can course see gone they're gotten are that S site other interested think down. spent less from go on date our on in program and

Operator

Keay question Nakae Chardan. Your from is from next

Keay Nakae

the you any hepatitis safety first cohort the to have healthy study. of a HBV question prior Doug, to for of of the Do the ascending similar company the in data the plans data volunteers -- present the from B does normal on the dose just any quick patients? presentation

Douglas Fambrough

until do there we made don't that we are to be anything that. important track very QX. Because our case something see do happened, result biomarkers We may to pharmacokinetics that. don't to plans ideal particular present no the sort well If we'd have is is to we've us, of it have any because lunky, although bad don't anything. And probably and

Operator

from Mamtani FBR. Mayank from B.Riley is question next Your

Mayank Mamtani

something shift think talk the on adoption to tell the same approval? Could to on ongoing? the on what also coming full landscape been to path market the really you've just line to, with Mainly you or you Because about talk just is obviously, new adoption? a interactions like. sort being rate modalities? point, as progress. the what measure And able other see the along looks follow-ups to that of peers? that how that a about with regulatory Just you like, which activities and And Congrats like are PH. -- two is how been? rare us the will disease-state have has plasma, even now get then community follow-up thinking the to how And you for think you obviously, about real-world oxalate ways levels you you disease to enrollment Could your maybe --

Douglas Fambrough

trials. the other was end-stage in I to that we're look into a wanted lot sort there opening course, these is There on disease, one oxalate. issues. the case things question. refer of no packed at of urine looking Thanks for renal to Ralf of at of to in trials, the And

want other you at the parameters? we're some Ralf, ways to disease looking PH address of And so

Ralf Rosskamp

Yes.

The are, events, course, of burden. secondary stone stone endpoints

So in looking stalled. like are of calcifications the at be renal system, which, hopefully, with ours the we buildup therapy a will

their their we an quality citrate consultation therapy eliminate during normalization the open-label with once function we of filtration at quality of will the glomerular we're said reached patients the and had course, previously, because estimated have And the urinary of as life. on of plan sure renal at therapy, looking which in oxalate I'm have their are hydration impact while to terms part looking, life physician, rates. in We of hyper will

be in calcifications are marker, calcifications oxalate which So plasma of be as week. a patients these patients. addition, with of further on sometimes the the appropriate it And dialysis, oxalate measuring -- frequency of the are will of to we reduction we measuring the And times who the on be are are reduced the X in quality suppression of course, dialysis, this in in have X in also decrease. can impact production will is And and then life in patients undergo -- those there BI. those a should these an the who on the hearts, -- it hopefully liver, which of would

addition a to in oxalate. urinary other array measures there's of So full

Douglas Fambrough

Phase Ralf's was in To unmet a the And should The I to the and be, observed oxalate use point end and PHX. high accelerated perks approval fact motivated proposed the biomarker comments. medical FDA. approval little with add I of need FDA the in strong considering full what a FDA both for as by as the We the discussion the on originally mentioned, believe we've a of effect had issue reached discussing I full on agreement approval.

efforts. you understood engagement I have bit asking if think size. that effect community correctly, a our posture with the question FDA And would then a finally, about a your parties comparable similar I other with were

workshop think effort community it very have and they're I There in our because that, and characterize Boston hosting for now. in corporate substantial We're outreach to is this suffice and vigorous. -- active broad we next a and to is I month. I'll PH in us that a is as events the largest say, around the single program community would right critical PH quite very

Mayank Mamtani

That's great. comprehensive there. color Appreciate the

the about not are Just their these oxalate about mean like -- from population? -- -- not in patients one is any the food? follow-up consideration in What a had. liver? what oxalate carefully I PH I consuming big that Is generated Like that

Ralf Rosskamp

than is carefully. Not studies of about at careful There the building dietary in who are oxalate it only the contribution person. centers. around less look know because some We It's a what normal rich are -- food, patients this, matter we very this Despite some oxalate not centers burden. oxalate is XX%. up really component avoiding of diet doesn't because at really at the overall XX%, towards very to it overall, accounts least just say minimal of

Mayank Mamtani

adjunctive oxalate you different any subsets need have? food -- they PH necessarily is -- the so an within that in that from the of the reduces those -- And subsets which you agent wouldn't that

Ralf Rosskamp

intact patients Not renal function. relatively with in

Mayank Mamtani

are on genotype Any the Great. on consideration assuming going hap non-Western patients? that to think very I'm important? these And you status quickly. B then be is

Ralf Rosskamp

we all Well, a And genotypes site XX% New also have in and population, cover predominant goes genotypes. that a Asian and that have but across of Zealand. we true all we shown in that's Australia -- site have have we a we the

I would So a also see to it's group, expect, Asian background. if outside patients even of small

Mayank Mamtani

last then And NASH. question on Great. just

between a target, with be like you multiple heterogeneity you that you you again and Or would just that on that probably would the disease, do does to partner, going just -- forward? disease how a to particular one before the any given mean want second explore the on want of collaborator as to think targets Moving just about helpful?

Douglas Fambrough

their potential Boehringer seeing the for stratification in I in target, the not the second both of had had brought the the some done don't So clinical and a is target, proposed that -- the collaboration details second Boehringer, actually as original on. the we option. would work for the with targets how has work play. is know which one of and done cases, our we is targets target one on, different And which Bohringer nominated that roles we that Bohringer and population

work But models. The on are on some way is that. development the comment clinical Dicerna and So responsibilities generates collaboration Boehringer. structured, I collaborative preclinical is can't candidate at the there disease

Operator

remarks, I conference this like sir? -- am over would I concludes Instructions]. any call, the to call at to question time. oh, [Operator further turn no this showing today's closing

Douglas Fambrough

their all you Dicerna, we thank it's travel this attention to time to as for want paying very great a today. and following see I'm everyone exciting the really for -- and attention I It's journey. story

Operator

Ladies today's for Thank have wonderful this a and day. and you concludes participation, conference. gentlemen, your

disconnect. all may You

Dicerna Pharmaceuticals (DRNA) 9 May 192019 Q1 Earnings call transcript