DRNA - Earnings call transcript Q4 2018 | Dicerna Pharmaceuticals Inc

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Good day, ladies and gentlemen, and welcome to the Dicerna Pharmaceuticals Fourth Quarter and Year Ended 2018 Conference Call. At this time all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] As a reminder, this conference is being recorded. I would now like to turn the conference over to Rebecca Peterson, Head of Corporate Communications, ma’am, you may begin.

Rebecca Peterson

you. Thank welcome and call XXXX year operating and company's discuss financial Dicerna's results. quarter fourth ended to afternoon, to conference the and Good December XX,

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

approximately to obligation contemplated those statements listeners discussed that for management our revise pipeline of important undisclosed for Factors today listen rare on undertakes also to example, programs, by this forward-looking from with Dicerna’s and guidance DCR-HBVS, hours Boehringer beginning may no today’s Actual Lilli, current website, SEC. for filed Ingelheim, call, materially plans XX will expected disease DCR-PHXC, may via timelines like on to including, program thoughtful result statements Alexion with conference other call section of partners You development those webcast results for collaborations the the Risk Forms the regarding and and to forward-looking opportunities factors, in the discussions, collaborators Dicerna's archived and and which as various and usage. be days I'd is two after potential call most expectations completed. expectations including a of related cash these remind contemplated differ

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

Chief and call like Doug to Dicerna's Dr. Executive President I'd over Officer. the to Now, turn Fambrough,

Douglas Fambrough

new strategic us plan promising Chief our thanks during continue today which With Phase that Medical move forward and in and plan pleased of on in operational to our our Rosskamp, Financial in successful are we to million and equity and September our of and XXXX met $XXX our follow-on perspective, primary milestones Dicerna pivotal hyperoxaluria program Alexion a Rebecca. we joining each Eli From two data Dicerna future to substantial payments Jack Good Thank collaborations solidified to offering call. platform a all our enables including an truly afternoon, our me was raised pipeline as of investments a milestone to most cash to am I $XXX with move say, Officer Ralf objectives time, excluding Chief and you Officer on key that GalXC trial. demonstrated in based Pharmaceuticals upfront our and for year Green, know, Lilly million this ability royalties. you, directly yielded on I you

balance Ingelheim cash few events for million sufficient as proprietary addition, capital hepatitis our $X to second for minutes goals a programs. with notable its I’d key a our We payment to to existing like and virus achievements. hepatic XXXX fully PH and began now a $XXX of partnered our company a triggering our sheet million. additional integrated pipeline The of quarter. XXXX continue beyond support In we to assets nearly a option strength recent both take provide recap and XXXX in with collaboration during Boehringer a clinical current under the balance target exercised provides build B

the DCR-HBVS are Our XXXX, January XXXX. pleased for us placebo-controlled double-blind first and in participants I Phase with of our chronic HBV multi-dose non-cirrhotic of have our position of Starting progress trial for cash goals dosing infection. strong randomized achieve clinical to the begun well treatment balance in to we DCR-HBVS,

chronic and study HBV XX As people a to reminder, XX three-part infection. volunteers this is up enrolling with a healthy

with of volunteers of and X.X, period. milligrams cohorts X.X, X.X, per healthy participants receiving kilogram first a XX.X with X.X, four-week level are DCR-HBVS single-dose The dose follow-up a

first HBV in the The two dose second to groups. different study evaluate to during We patients of patients the is expect quarter designed XXXX. chronic

doses milligrams three receive group who experienced kilogram. of DCR-HBVS patients X.X, over and X.X per First, months NUC cohorts with of a four of dose X.X, level

Second, receive evaluate milligrams chronic at NUC-naïve DCR-HBVS patients who single and kilogram. is people objective healthy tolerability The a and primary X.X of safety dose in volunteers a group to infection. HBV of with per

believe Secondary and and levels an serum surface there objectives to of as to HBV rapidly demonstrate opportunity characterize program, the are and study. proof-of-concept pharmacodynamics I DNA. to B Phase the the efficacy compound viral assess in to preliminary With are Hepatitis easy of antigen on pharmacokinetic evaluate profile of this antiviral the we biomarkers HBV is

coupled proof-of-concept we GalXC infection liver, have in DCR-HBVS for data the HBV high of that fact strong potential already with the levels s is DNA chronic reported circulating the HBV work in of effectively confidence antigen viral reduce the XXXX, Given the viral platform and to during to HBV a patients.

You approach may that HBV recall our is for differentiated. GalXC-based

of DCR-HBVS is out target maybe duration of designed on HBVS of all antigen action reading we’ve approach longer excluding Following simultaneous HBV the target borne RNAi’s four duration the reading frames. that three to preclinical and silencing results open models x and and open dependent site, deeper our This gene. hypothesis than of four in frames the silencing potency the in

than with preclinical in greater for in XX% injection. that our reduction viral infection a than kilogram months persisting a milligram models studies antigen circulating of HBV mouse XX.X% DCR-HBVS reduction per single three X more show after Specifically, s to led

be therapy. will DCR-HBVS of part as that a used combination expect We

expect from the patient which as per to during of kilogram well fourth the milligram to of multiple-dose the I market, from is X.X following size that, this cohort our Given partner quarter plan asset sheer year. the trial data Phase report proof-of-concept this as we the

QX for Annual single-dose ongoing PH XXXX, to our at program In the clinical presented proof-of-concept of I lead for the PH and ASN on DCR-PHXC. mid trial Moving from data PHYOX Phase Meeting. we clinical DCR-PHXC

clinically data near Based normalization oxalate reduction meaningful from hour X, of in completed normalization both with a initial PH patients. volunteers levels cut or the significant in all XXXX, We type October urinary of and and type on for XX urinary dosing majority The PH the patients have healthy patients oxalate and findings levels. showed achieved X X.

PH therapy oxalate Specifically, showed DCR-PHXC the X.X in in was approach a trial potential the potentially X.X healthy X.XX the equal defined genetic that of millimole tolerated near all level oxalate debilitating XX the GalXC X, a first using X.X of one than treat of mean single five that groundbreaking track single participants XXXX. study range placebo-controlled into defined participants and to DCR-PHXC including at this XX reduction are findings PH excretion At well hour patient. more X initiate participants multi-dose maximum XX%. in this our that to and variance reported a in the led urinary dosed urinary in maximum volunteers patients four normal than urinary DCR-PHXC to These dose three milligram out per led in dose excretion time investigators kilogram in PHYOX urinary four dose Furthermore, also The XX of fatal RNAi than to one greater on per X.XX investigators PH. oxalate as millimole. reported as post hour pivotal, of of has normalization or that brought in less double-blind, including out All said, of that with or we less kilogram and quarter a XX% hour oxalate mean disease. millimole milligram that a X three reduction points and data indicate levels of

a Agency month. expected FDA meeting application approval. take with next encouraged The trial and pivotal to study design for this proceed which the to has with base is to place on reviewed the Our already our us

program, point anticipate of genetic availability leading of the predictive failure. production production this need regulatory we will cases, lead target in and XXXX, similar We wholly-owned What what we PH, fibrosis. the strong projected rare a can will protein is abnormal population, to At liver at which second fibrosis, a is approval. the protein disease that, cirrhosis believe based prevent high leads of potentially therapeutic or able readily was thereby fully tell biomarker, which a to and is the strategy. path quarter gene is around unmet gene you that identifiable the where stop disease present, believe program slow the undisclosed the completing the to patient abnormal Moving damage a a silencing disorder disease program. some and in program an on strategy The our in to on liver the and We our gene I third to of liver of IND-enabling progressive rapid to studies. this of mutations still chosen hypothesis, be medical progression to filings our and discuss submitting disease point, at

the a the a platform testament our disease quarter, exercised we subcutaneously to existing to a for initial expand Dicerna GalXC a relationship to RNAi of option which hepatic million target under chronic triggered target delivered XXXX work to the and novel, to on an its therapeutics continue Ingelheim $X for relationship decision focus in early develop the with technology together. In the Turning to and is positive belief NASH. treatment fourth collaboration with partnerships. our payment disease in our RNAi second value GalXC second their liver enjoy discover our Their and Boehringer of

additional December potential and Pharmaceuticals October with for the opportunities collaborations technology Alexion the GalXC newest unlock of XXXX of Our Eli liver. Lilly and in platform respectively and closed

yielded upfront contemplates $XXX In a a key Alexion’s for pathway two the It the a as low-double-digit product equity and Alexion agreement, and investment diseases. with also of $XXX working platform collaboration at a significant metabolic per joint XXXX. beyond which $XX of significant development and we mid-single the of development million cash are and the with including at RNAi to the directed non-liver XX an approximately targets Under to on highly complement-mediated tissues. and Dicerna. royalties collaboration, million the transaction complement more up which commercialization in receive collaboration cash have GalXC tissues a equity the of transaction to Lilly sales. a is terms million received treatment in well liver target XXXX the our on discovery for $XXX we addition, premium Under milestones, to for team with to for our in molecules related upfront we million than Likewise, in combination cardio is potential Dicerna. agreement neurological investment premium GalXC in leverages January The those as targets

to Dicerna will exercise and additional has through leading options work to mid-single preclinical targets. beginning greater addition, and payments development future In additional Alexion as two receive million for and efforts to Phase low-double-digit the potential right molecules pathway milestones, product on of directed efforts development provides is This royalties including the payments for collaboration Alexion sales. research RNAi the $XXX with complement as lead to additional commercialization I discovery studies. joint than GalXC well

points Looking are of important horizon. ahead, there inflection on a number the

the rollover expect quarter the in Phase a level in are the multi-dose to we same extension PHYOXX pivotal I previously, on frequency first PHYOXX to Participants be mentioned pivotal As trial as quarter at the the for dosed trial. parallel, begin initiate in of dose we of study PHYOXX, long-term XXXX. those the in track initially XXXX, open-label second will in and

Other we an aimed multi-dose trial aged what at open-label expect disease serve look expanded in end-stage to trial a years. an for pediatric like. the X, as determining trials the Type study that open-label single-dose label with in supporting PH aimed supplemental include an such, ten at product children data As X X with proxy in should data study renal PHYOXX will and patients pharmacokinetics adults PHYOXX

healthy dose Phase expect continue I trial we and to of dose in volunteers this the first DCR-HBVS, of XXXX. Regarding the patient second clinical quarter to

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

undisclosed therapeutics. as in earlier, submit important Dicerna mentioned program the XXXX. of the of field second RNAi our player Finally, a will quarter application I trial in clinical is disease an for we rare

team. In As Chair TetraLogic directors. Cephalon grown Chairman. have additions of Anna Vice of Consulting joined work some former Therapeutics Board Executive to and meet Head also we Board as our and we our our President key L.E.K. our have and with and Mersana late Pharmaceuticals expert have and Officer formerly new at Protopapas, made Buchi, Kevin Marc and January, collaborative Chief pipeline Officer Healthcare named Executive of the Chief partners, Industry Kozin, internal to needs to and leadership staff

Brian behalf. Our former Halak deepest Dicerna’s appreciation former their and efforts Madden thanks Director commitment David to Chairman, and and on for

opened of today’s team as Human Development. and we added President, Rebecca who over recent the Resources, Head of of the Corporate Medical turn positions Jack. with And Achneck, President pleased Communications, Finally, to are Peterson call Vice let several me have key on Regina with that, call management Vice the of Senior DeTore as additions Paglia to Head as Hardean

Jack Green

I’d Doug. provide guidance financial and to briefly you, XXXX Thank results summarize XXXX. key like for

The which investment and million a year respect a million equity. at full received, by associated the our recognized $XX.X we premium. programs. ended the $X.X agreement. premium XXXX, be arrangements the balance equity was portion to the equity the $XX.X majority compared million represented recognition With Lilly, recognized the Alexion, revenue The of upfront allocated Of over million revenue, a initial term $X.X XXXX collaboration was research over we to recorded initial anticipated million. of payment, the $XX with revenue of received collaborative will three revenue from investment collaboration to on Ingelheim million million agreement revenue $XX.X collaborative the of $X.X During deferred payment In under our XXXX, from equity the premium, programs. as year XXXX, revenue million in $XX a Boehringer deferred to XX, the with primarily term year first to the of be was we at in recorded milestone of research recorded agreement December Alexion the $XX.X a as as of increased deferred with and $XXX the received million due of million while was

recognized increase were in of million the three XX, $X.X increase period this for As year be as $XX.X the next expenses $XX.X in the Alexion XXXX with to the The compared collaboration, December for as R&D same with over million years. to ended revenue most will million clinical XXXX. a due $X.X expense DCR-PHXC to to in an $X employee-related headcount increase study million and growth. of necessary well XXXX increased in as associated as was due activities programs, million in primarily DCR-HBVS increased the associated costs support with expenses

costs for to increase as studies, associated research development compared lead clinical XXXX, clinical overall necessary to we expect increased expenses and advanced as and commercialization, million to the Alexion agreements. million supply and complete foreseeable product primarily efforts Lilly salary December for continued toxicology of We respectively. General million, increases in million our $XXX,XXX for candidates and the $XX.X with $XX.X and and development Administrative benefits Directors’ expenses compensation $X.X an ended the XXXX, expense. The in were XXXX Board XX, in and preclinical due and under activities, XXXX, activities and and years is $X increase consulting future, manufacturing increase of of

activities. of primarily increase expect We preparation Litigation expenses investment increased expenses the planned of staffing, market in as XXXX, company readiness associated our largely headquarters G&A compared XXXX. expenses new in solely litigation. the expenses to XXXX, recorded with $XX.X expenses our to of Litigation Alnylam due consists and to million to in due settlement

payment the final January Our made Alnylam under with XXXX. in settlement was

loss per to for $XX.X $XX.Xmln important preferred XXXX deemed stockholders $XX.X to dividends I of attributable or loss the attributable XXXX. was stock common with converted share and common XXXX. to As the mentioned, common the net in redeemable share It’s in or million approximately $X.XX was per to million note convertible net and stock XXXX dividends compared December $X.XX activities in on result a that for included transactions stockholders that

therapeutic million received held-to-maturity in equivalents and of $XXX.X XXXX This maturity had which of resources HBV December on proceeds $XX.X partners. XX, infection, clinical XX, to categories. investments received PH, payments from us the cash Alexion December cash held and we in collaboration early collaborations. ended upfront million cash, of and to net And from investments. in $XXX.X from compares positive additionally equivalents XXXX, XXXX This financial XXXX. trials We our Lilly million we $XX.X in with $XX As the cash, of premiums cash million enables to increase of operations as other included XXXX million, from equity advance and in in and cash

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

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

Douglas Fambrough

you, Thank Jack.

to thus think was partners to Dicerna say shareholders XXXX as we like We fair entire that to achieved for it’s the I contributing year true a have success thank for remain I our would our far. our keeping leveraging and to the breakout and our we focused call always strategic transformative platform on now Q&A. look open Operator, key execution up-to-date the RNAi on for technology. proprietary can RNAi-based GalXC patient delivering plan. forward you by to populations therapies We of team, our

Operator

question Our [Operator Instructions] Robyn comes first of Citi. from Karnauskas

Your open. line now is

Robyn Karnauskas

guys. on for Congrats question. progress. Hi my taking the Thanks

ones. just So a couple

single that the are X.X I durability then X? you second kg that was, differentiated? And know first trial x do talk dose the Is would thinking make that’s tell whether about any trial? to maybe my you you – HBV way and then, that Phase not drug the a either the Do this What normal after the for And though see see by in X way mg have the gets you dose be about patients. the in Phase you is X.X that differentiated. we So, any there going follow-up. about could right think the you per comfortable or spec actually initial design, a PKPD viral question and how versus or there load X, gene

Douglas Fambrough

Robyn. Hi,

real would data data I biopsy any looking the So, of gene protein preclinical correctly predicting and targeting models core at direct not The can get localization. effects that that the how have x think human been read require a the we evidence would are don’t like. what the

we So, just terms to duration a different in I s and be and different be only antigen study different out comparator is there company. of of potency at to looking think, are going reduction action a done at going from the a molecule

companies based With very as levels observed being And patients, own per X efficacy so, milligram risk translation what for well done robust we’ve reproducible animal best there. one’s can at nuke-naïve is choosing efficacy a from it’s kilogram to a HBV have other human to I be levels. that really comparisons and regards and the a as think guess dose on chronic observed

You will that the are we’re used the we the levels in molecules our given no the dosing established proposing of same the notice that program have similarities dosing in saw the in program the DCR-HBVS levels DCR-PHXC preclinical and we paradigm. reason depart to from performance

best really I preclinical it suppose in guess a and based the that’s So activity clinic. see how on we’ll goes

Robyn Karnauskas

helpful. very That’s

Rebecca Peterson

follow-up? You got a

Robyn Karnauskas

first follow-up any first disclosure said the the will for have Yes, for is cohort the we’ll I get – read data and treated. been then think on have out that and X.X the you proof-of-concept just drug, patients that in we

me So XXXX first the then the data that in with help Help around the we and definitely dose? the and data dose population see patient quarter the X.X understand understand or fourth could me just have disclosure come in-house potentially data in will could that we data following year. proof-of-concepts

Douglas Fambrough

multi-dose, blinded public is quarter. be the are We trial. Note in a to making a expecting fourth disclosure a and it’s this

giving be period. doses will a We over four XX-day

before follow-up the after We us are position meaningful. dose, to just start into run to dosing a say in the if clock are we in of allow from QX the some last will see - and QX anything and you we

Robyn Karnauskas

to there I about you enrolling. Okay. want anything trial, I that roll trial it’s curious PHYOXX because the you sorry. that PH PHYOXX was of it’s doing Well, or you diagnosed help, that anything easily a are would question, are X Is enrollment know Last you page to think doing to last begin help not three, question, lot, the or Thanks. over trial. a do trial

Rebecca Peterson

we’ll that Actually, to take Ralf direct who it. will

Ralf Rosskamp

long-term when – is study. what this we you call the Yes, PHYOX said PHYOXX, expansion the

Robyn Karnauskas

for PHYOXX study. PHX No, I mean,

Ralf Rosskamp

study? PHX

Robyn Karnauskas

Yes.

Ralf Rosskamp

at our on in age early are is most recruited the will range. be because manifesting range most likely study and patients we the children, have age will most six This this likely upwards they

Rebecca Peterson

Robyn. Okay. Operator the take will question. we next Thanks

Operator

you. Thank

comes question Mani of SVB Leerink. Foroohar next Our from

is Your open. now line

Mani Foroohar

Congrats the building do XX collaborations think year. Hey past guys. broadly, that about have attractive sheet. of part this the on guess a more the out when recent all in terms you pretty progress question. balance I you’ve signed, not I

got cash as streams? balance not, at runway do Strategically, You looking long exclusive a potential add in to attractive obviously on upfront at about the and additional economics the or pretty shifting tail of to point. a the toward runway you more collaborations, to continuing think cash focus opposed how terms royalty this of bring

Douglas Fambrough

question, Mani. the asking for Thanks

had small organization maturing Lilly nicely and evolving really and organization. is talks companies, and collaborations modest Alexion Our clinical early when exploring capability and were those we with and first a rapidly we and very the pretty a

time, We are not growth was At rapid in a as balance strong. phase. our sheet the

execute been we And our still undisclosed do have programs. to might work that and to – rare about, disease, talking on real didn’t impinge we other there three own was collaborations clinical all So on a wanted ability PH we’ve our and programs things. HBV concern

table And mature so on have thought organization efforts really sheet am for the and larger to Entering in a balance in have to much the try with to I company a team partners a we hypothetical discovery focused with the way, and develop more speaking much now it. conversations bring the stronger execution. our and phase much a our to we

And in so longer for some avoiding longer company. downstream no commitment rationale the the of is strategic present applicable, no

to in downstream us collaborations. see more aggressively So it for sense future makes our participation

Mani Foroohar

Great. follow-up do Question. have And I guess, I a

is at Robyn my now. So good laugh having expense a

think about does whether potential about of at normalization equivalent? in you a majority knock-down shaping the clinically in when and you how kind endpoint, market as do normalization approval, looking the does think commercial normalization the show think when not, of that you So commercially program or Or can patients? a think if reach PH you opportunity you about you vary up, the particular, significant

Rebecca Peterson

Chief pass Medical I So, to to am that Officer. Ralf, our going

Ralf Rosskamp

of reaching point they have from is the which goal what or the so normalization, concomitant medical to to medication a – now can is regimen near Well, are view, right patients be normalization super stopped. patients that bothersome and taking I can hydration speak ultimate really

would You reduction do or if therapy is and or reduction, with the panels would hydration they stop and they we so, wait them XX% until the or risk levels. said, the the or we oxalate, had at you patient super would living normal for and normal the citrate no. because normal only a stop. We potassium had them are ultimate patients oxalate see what can is normal life still question physician and it near goal discussions asked not and And urinary bringing all normal to XX% this near means you

Douglas Fambrough

management models that’s but we can how that now. animal achieve chose and level gene right the dosing that Mani, DCR-PHXC regimen is we don’t hit sense and robustly but an in and that have the near anything cease is or there complete intuitive order more we doing influence essentially did certainly quantitative, disease sense LDHA system to them the normalizing we allow the management. the patients in by to the Then, intuitive continue hard a have if stay regularly GalXC had normalizing we So, highest commercial extremely oxalate really capable ideally I allow is of oxalates, likely they think, capable levels. their of silencing. control Patients to will the so of that to our of for knockdown are disease target that stronger Any uptake. life their better It to of with more regimen that’s on chronically, if for rest an drug unqualifiable

a So, have very helpful strong to response here. very

Mani Foroohar

have to human ever or any non-human toxicity Great. cardiac in this, And – just final have know, seen it’s a you Have being I you DCR-PHXC? that signal primate one gotten horrible.

Rebecca Peterson

pass going to Ralf. am I that to So,

Ralf Rosskamp

No.

We our have nor seen the neither in any in animal not cardiac toxicity data. human

Mani Foroohar

Great.

Rebecca Peterson

right. All

Mani Foroohar

Thanks I you. I on am pass guys. going with to

Rebecca Peterson

Yes, Who next? is you. thank

Operator

our Thank ISI. Umer you. next from of Raffat comes And question Evercore

line is now Your open.

Jonathan Miller

is Jon guys. Hi This actually Miller.

What the are drugs else saying I Alexion progress congrats on the the echo especially, start are will liver-focused? when So, all the all months. with congrats. next those the to wanted past steps the partnerships? the collaboration. all more there there new few partnership, for I we over the And and hear news everybody about from and targets

Douglas Fambrough

words. the for kind Thanks Jon. Hey,

I’ll Alexion. Alexion pathway to Alexion start GalXC three Both all The are to with the liver to Boehringer the are the designed use the – So that and the and is our of of current Alexion targets really, are collaboration the Yes, collaborations liver-focused. of complement platform discovery apply question. including it targets interest collaboration all stage.

set to and And preclinical candidates clinical entry. so, need before they researching subsequently declaring which go development out formal through to clinical begin

while there before sign. clinical not period but work little it quarters, minimum six is takes occur substantial the occur sadly say of time, day let’s would So, a which more any doesn’t started likely you entries get a following to getting while. but But after going, long, a little

Alexion started early in be collaborations Boehringer emerge the from XXXX or XXXX in having to has potential the that for strong there XXXX action. the going flow earlier isn’t Lilly and So news some timeframe.

Jonathan Miller

understood Thanks. on Great. that. I

and the and color the multi-dose On us some on other give the single-dose nuke-experienced you nuke-naïve HBV limiting arm differences you why arms? to more And trial, there? are arm to the could one between

Rebecca Peterson

going to am pass Ralf. to I that

Ralf Rosskamp

that’s nuke patients therapy nuke on XX who nuke-naïve let’s as and nuke-experienced not try to name are them – patients currently The on without patients for keep our we days. nukes are and

with nuke-naïve patients with so why the an – criteria single-dose after usually to be because it’s nukes. is that hepatitis treated and reason in say the that’s So, chronic that need guidance the the also will

really it’s the standard of we keeping would therapy we if of multiple-dose therefore thought – difficult in setting. them So,

the couldn’t modeling single-dose can it and the us to simulation decided nuke-naïve do why will do we multiple-dose you because it and again, can So And the compare That’s in directly days. in have the administration. follow-up single-dose XX a with limited setting. we have first patients setting only help you

answer of the have multi-dose nuke-naïve multiple was will question? your happened the model days patient single-dose if us then will that predict help parts XX dose dose first the perspective what in part to after So nuke-naïve received. have the a this and would simulation first Does actually from and

Jonathan Miller

there listed is Yes, And guess, absolutely. and study that on the recruited. is, I listed my question That makes I PHYOXX noticed final some sense. trials already perfect then on

the at thoughts there? you we least for So already when given of starting, have potential about with the your what expect eminently build news strategy and ongoing PH out can registration more trial or and commercial there to threat

Douglas Fambrough

Jon, using the consulting lot resources. internal is There analysis lot of thought we a commercialization a both and path. going firms about outside

to this But to We timing adequate hit ready that prepared talk with be build. the we what running. the ground is when an runway process we going commercialization in like are achieve will organization the not we hope to about we’ve do we of approval, started that the look such feel

Rebecca Peterson

All right. time Operator more. two have we

Jonathan Miller

Thank you so much.

Rebecca Peterson

so much. have . Operator time you questions. for more Thank we two

Operator

Thank And Stifel. of Stephen question next comes you. from Willey our

line now open. is Your

Stephen Willey

Yes, just questions into preclinical needs support the afternoon. enrollment and lag the long-term With to of terms for to of to of taking guess why open-label progress. congratulations in extension, done that to completion open portion be And of the getting trial? dosing? any due work this and the timing patients this toxicology is good I PHYOXX, to curious respect on Thanks the the lag as the that

Rebecca Peterson

I’ll to direct Ralf, So, that you.

Ralf Rosskamp

second is trials. we when and much lag. quarter first there in the there lag expect starts We it’s versus time saying quarter, guidance no, time Yes, the isn’t and the don’t it gave between much not

study that, We original patients the have reason filing to still contracting is PHYOXX trial and the are those given of of and priority PHYOX in in many follow-up. in the terms

they their XX% baseline. haven’t reached So, of

a gotten time could would great even of difference be still those trial But the our because have patients actually, in initiation baseline. if be portion reality their recruited be will not able a have of now, recruit, back they right between to big the to haven’t trials. it – not we So

Stephen Willey

portion? retention Got extension open-label it. for rate around optimism the And the

Ralf Rosskamp

benefits. multi-dose will, part that we I informed not pharmacology that’s believe as on the of these the investigators to most our have have a go is conversations work into can study consent patients actually of what with ask they did expansion go study clinical that and direct patients and The into protocol already a study. and this long-term PHYOX part the will

this into go be looking patients think open-label to will study. I So, most forward

Stephen Willey

last Got just, then I it. And a quick question. follow-up. guess, one just

pretty on to PHYOX from guess, FDA imminent do meeting this to expect registrational you later month? here. is Just the It’s dosing I the What into so, trials. posted program gain

Ralf Rosskamp

want We last get clarity the the around endpoints. to final

and with about this approval because, will a near details it us accelerated of our where it’s change of we full back, study some heard double-blind analysis, talking it about approval statistical be would need some an are have Operationally, agreement part there to if we hold not the this normalization, and slight You plan. does where agency. normalization the

to start back do countries operationally, doesn’t we plan So as of the in goes to U.S. outside it

Douglas Fambrough

regulators, achieving for the observing a and powerful don’t Dicerna below are normalization, right. you are normal PH going near eligible some biomarker. approval normal perspective, I All add idea amongst be little a where this anymore. very patients both The to The you based to reduction color oxalate to was think levels change would we drug program have at accelerated that, of with or and this oxalate led has want of this as Steve. patients hyperoxaluria original I normal in reduction if that a in on what majority the

don’t the really So disease, the enough mitigate to don’t reduction to you’ve we have had need disease you anymore. assess whether

a So, suggested And being discussion they like. obviously meeting a approval accelerated be a a to instead an full approval outcome what asked approval. than look approval full open accelerated to have better subsequent us they full would that of around may our first a after approval endpoint have

analysis of what outcome the not be. But trial, configured. trial endpoint the I be about of So, know the discussion Ralf conduct will about going final and it’s data to as don’t how the the it’s the from said, is

are updating look discussion everybody outcome to when FDA the on the we with forward to. this we of able So

Stephen Willey

Very, very helpful. questions. Thanks for taking the

Rebecca Peterson

Operator right. All for question. one we time have more

Operator

Thank Humphrey. of Edward Robinson Nash you. last from And question our comes SunTrust

Your line open. now is

Fang-Ke Huang

This Hey. the much Nash. taking for for so on Thank you Fang question. Edward is Congratulations progress. on our

you the to maybe the PH mentioned have pivotal the it, trial? think I I’ve missed tested for first – PHYOXX, dose is, is dose have the the we trial what going in in – you mentioned question have pivotal

Douglas Fambrough

Yes, There monthly of ml weight for lower we our patients. X patients. is believe for milligrams It dose in adult lower XXX volume younger we be R&D and Day the have. fixed will a I a disclosed a in dose fixed dose November.

Fang-Ke Huang

HBV. Okay, got is the that. thank so second you question And it. And much on for

is you think combine treatment drug or and help mentioned sense you the us make just action looking DCR-HBVS to are what that eventually, for So, your you for can combination? another other makeup the

Douglas Fambrough

Sure.

combination is is new the you the clearance What of is are adept HBV response blocking. So, combination agreed – of levels HBV. it the the and And by goal move zero people goal s immune to are patient be is antigen below response achieve and therapies reduction. against community line a virus two productive virus functional which RNAi conversion. immune reduction clear towards to zero a detectable achieve zero RNAi-based for wants need cure is the see – s productive to a in of the but to addresses really the where the seem those program antigen the seems and at maximum our clearance, the generally

So, of consensus development with pairing for the captured sort view direction. things some further I’ve of intuitive of immune do do some RNAi the our of pair. don’t that, I partner something like activator at least for in or of stimulate that’s know to the I sort system talking that out intuitively we go certain immune to that HBV host host work makes about that stimulators seem potential types a would those innate vaccine, good active and try Having acting in sense Whether therapeutic But intend said immune response. that with to agent. community will immune even the you think

Fang-Ke Huang

It’s helpful. you much. Got it. very very, Thank so

Rebecca Peterson

today’s so will And any all you much. Thank Doug, operator, closing right. you now call. we All have remarks? do conclude

Douglas Fambrough

their attention to and you want look to keeping updated. for kind everybody thank I forward all analysts. I the the from words appreciate We

Rebecca Peterson

the following going Yes, call. the are keep company we have available and you we to at should questions additional earnings certainly pushing any are

Operator

participating today's conference. you in gentlemen, conclude does This thank and for Ladies today’s program.

You may all disconnect. Everyone, have a great day.

Dicerna Pharmaceuticals (DRNA) 11 Mar 192018 Q4 Earnings call transcript