DRNA - Earnings call transcript Q3 2018 | Dicerna Pharmaceuticals Inc

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Operator

Good day, ladies and gentlemen. And welcome to the Q3 2018 Dicerna Pharmaceuticals Earnings 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, today’s conference is being recorded. the to turn Investor Schwartz now Relations. would conference Paula over with I begin. you Ma’am, may

Paula Schwartz

operator. you, call XXXX the conference and to company's results. quarter Thank third afternoon, discuss Good welcome to Dicerna's

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

listeners of Actual for to those as with this that and call, to which expectations with plans factors, indicated today. making may of Lilly related with other regarding call days and Lilly [ph]. statements of timelines SEC latest including, expenditures webcast on of two listen development on expectations DCR-PHXC Website, and after management completed. the potential today's Alexion, in archived will be differ You Form our call and for result discussions opportunities cash would statements Factors Dicerna’s Risk the a collaborators, be partners the forward-looking hours those the and pipeline guidance discussed expected materially from collaboration risk via conference for XX the approximately results remind and and example, beginning is programs, XX-Q also including forward-looking to important filed this possible various closing transactions, may balances these I'd by for section like

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

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

Douglas Fambrough

and Rosskamp, joining and milestone time and With for with important thank Thank Green, are number Chief has exceptionally Officer. today. the Dicerna you, us It Ralf Medical our busy extremely an me Financial been Paula. you of all achievements. exciting Jack afternoon for Officer our Good

number Dicerna become position The of with that market us therapeutics third strong pre RNAi clinical and for operational leading point aspire we milestones key a a that the to of have quarter the significant weeks achievement put turning a company be. to and clinical, in recent

clinical latest data investigational rare candidate, recent Company Lilly $XXX XX PHX Hyperoxaluria to show on substantial participants and in licensing hour levels RNAi achievement collaboration agreements a interim clinical at in X DCR-PHXC, findings ASN and and oxalate long to announce for equity and reduction RNAi pipeline capital proof-of-concept type DCR-PHXC and proof-of-concept of our GalXC the has Eli collaboration with execute now our disease address two with resources type company, continue DCR-PHXC the which progression note, interim discover pipeline, our quarter therapies become what needs. collaborations and the necessary expand the technology two a further and over and biotech integrated of Alexion our and third our our proprietary tissues. with therapies follow in platform period, and upfront our urinary The demonstrate platform during GalXC With for investments we were Primary liver kidney October. X that investigators technology of significant data presented partners. These target to a develop clearly in announcements with agreements inherent these and top pharma ability and goal. have internal with Of involving with our combined using all forward pharmaceuticals payments clinical offering, ability for we dose million unmet clinically to the to subsequent third GalXC in and diseases fully and been of and proof-of-concept the our our stated medical demonstrated the fortified industry's other and develop internal data have single potential illustrate adults our week particular Further, pleased collaborations now to strength belief million a second and strategy monetize We PHX. our to develop $XXX our to RNAi are platform with patients in lead regarding

We have biomarker we is we taken brought patients dose. in key normal quarter the trial the XXXX. the majority head a first for, of in single range pivotal The or with as a data it near normal of into and, everything into a hope

Zealand to clinical normal pending forward conduct DCR-HBVS approval. a DVR Phase New virus, in study a and regulatory hepatitis In trial application B trial addition, for volunteers patients beginning X with to this we filed look healthy in and

collaborative DCR suggests As potential very has asset. the be field recent activity valuable in HBVS the to a HPV

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development clinical upcoming development three milestones. and the candidates of number studies progress program, the which of we our an advancing made half eye product and filing enabling lead GalXC in a is expect development half in sum, first this which clinical tangible first with achieve clinical initiate of during programs the important to expect development continue have to In progress in of IND we We have to XXXX. of to XXXX, toward regulatory

undisclosed half DCR-PHXC pending of to filings to Phase intend trials DVR regulatory additional for submit the approval other of in we XXXX. the the pivotal early our for regulatory intend clinical X registration We in to and XXXX, initiate to X/X for this feedback. and trial Phase program regulatory clearances begin initiate first HBVS clinical in we of program trial Specifically, quarter plan pending file to countries. first our rare disease regulatory XXXX, And expect

lead turn now GalXC candidate Let's investigational to in DCR-PHXC, PH. our

were know, week results XXXX presented you the meeting. updated PHYOX with late most for during in Phase and and data ASN the more in clinical participants session kidney clinical September, As poster PHX annual DCR-PHXC the in we study at reported breaking proof-of-concept PHX of recently ongoing X trials

reminder, adult including B XX milligrams and portion and three ongoing. is patients As Group volunteers X.X in the XX with which PHX the of kilograms PH, PHX dose a flexible has PHYOX of and a of study, group normal dosed patient’s per dosing X.X complete. one Group B that cohort the enrolled with portion consists And PHX in X.X, healthy A with of cohorts which is at levels. study, the patients PHX fourth eleven of

four. patients cohorts in have We three, three patients for X, one cohort PHX in and of X and in PHX each PHX patient cohort enrolled

by pediatric pediatric the slots patients. for enroll We PHX have FDA patients been holding to been the cleared recently and additional have

on range vital show results per single or levels three oxalate XX-hour a out Interim have and also healthy B. hematology to significant urinary urinary ongoing level in have dose findings in in A a a cut signs in is dose results reduction with participants. participants XX% including participants a Five occurred that without normal in that all in time X.XX group out were in than and and serious of up per the XX%. intervention hours. participants XX% as No led level, a in the study. data PHX of experienced excretion to less a of show mean single between no kilogram PH. XX-hour is normalization Xst reactions one five in injection and at XX PHX milligram significant or trial in events been dosed and clinically maximal as and group volunteers brought lab X.XX dosed show three or of with participants October data the this four or also changes mmol transient The mild-to-moderate and at cut, the than points participants All participants urinary site participants of which of normal greater PH dosed the led excretion four demonstrated clinically X.X more of October which dose Again, is post X.X Xst oxalate reduction tolerated PH at kilogram of meaning including DCR-PHXC defined at reduction dosed there near data this XX within in XX mean Follow resolved XXXX, severe values. been equal to into XX-hour oxalate, defined maximal oxalate DCR-PHXC XX% As DCR-PHXC safe PHYOX of one urinary that have milligrams X.X that adverse mmol a level, and electrocardiography, reduction. as well to

for hour urinary We treatment the mean the oxalate encouraged are product We reduction of is dose. single believe a very levels by given PHX clinically that following of XX highly a maximal competitive significant these DCR-PHXC findings.

patient all for the populations, a than the a believe, we believe only the a PHX significant PH, therapeutic forms the subset and need PHX the in we unmet to in these unique stronger of treat DCR-PHXC, company who addition, foundation only populations of given address those a intended of product value developing In market. to as form we are position and

earlier, noted details we R&D and share registration some week initiate design first Phase plan about our multi color on dose a we day in additional this XXXX, of X in addition trial expect Friday data. feedback pending regulatory to quarter to trial I PhaseX/X the timeline the to As and on the at

single HBV. study with patients HBV. be XXXX. a normal regulatory with XXXX with and evaluate and in the If Group be normal XX early in intend healthy objective chronic healthy volunteers Medicines with CTA of with multiple Let's the trial New study will candidate A a is the normal study to virus. development NUC-naïve in to Zealand for dose file Group and the dose and volunteers. B B dose turn non single Committee will a hepatitis chronic primary clearances clinical The a patients In We healthy healthy now up to for to ascending C HBV. enrolling patients other October, in XX is filed New in our additional the CTA Phase Health Devices clinical chronic patients trial will with be be countries and XXXX. X and product plan chronic cirrhotic Safety The Ethics patients part safety initiate of begin Medical in to DCR-HBVS treatment ascending clinical and tolerability HBV. we chronic of And and a in volunteers a to Zealand Authority volunteers study with HBV. DCR-HBVS, normal the in study we will Group three trial Disability DCR-HBVS in approved,

to The B, efficacy characterize of pharmacokinetics blood. secondary evaluate on compound profile hepatitis to and and antiviral antigen in of levels surface the pharmacodynamics HBV and plasma preliminary objectives the DCR-HBVS are of

HBV We our differentiated GalXC RNAi are the and approach. specifically about treating of potential about for optimistic based

the and four later RNAi we'll and of targeting other our at advantage and in based both genes approaches. ability engineered based RNA target to reading highlights leads R&D persisting in presented key studies, our have have therapy kilogram a found greater silence open of silencing preclinical targets and meetings the to milligram relative discuss for of reduction frames, reproducibly is, viral HBV circulating therapy. scientific our many duration we per essence, have preclinical mechanistic single we models, programs with deeper reduction frames. therapies more XX.X% simultaneous As In XX% injection. three week, RNAi silencing antigen day a messenger to reading three DCR-HBVS in of simultaneously four to we combination three four than mouse HBV which case, HBV multiple infection, gives viral This longer of than antigens, viral months or this three DCR-HBVS open HBV S after all in a than in models

turn We part the a day have antiviral one are and activity paradigm. preclinical believe studies of will to I'd We DCR in our program. of forward with RNAi we details program beginning day. that therapy the undisclosed at treatment to HBV this plan be based seen clinical share pleased look second to and our to briefly human trials about R&D more rare disease HBVS like

XXXX. to collaborator as product and program and expect candidate We plan Lilly with are product As to the submit financings, at candidate to seeking We I this sharing develop Alexion disclose alluded wholly-owned clinical a develop longer filings the to that to of our development regulatory in plan strategy collaborations we to program the in clinical company resources entry. the expanded time. of given half this the and and my a no recent first our earlier comments, prior risk

GalXC drive agreements significant collaboration develop Let's our discover, turn The and with and our Alexion and domains. power with GalXC RNAi enter corporate GalXC strategic to the technology strength in our platform us to Dicerna of therapeutics. now provide our the expanding and to have to based team announced these programs platform into performance development partnering of for interest. collaboration forward new leaders while with targets by capital These internal enabled of and the therapeutic disease our areas recently us agreements field Lilly necessary and respective and are collaborations

Discerna’s me a research RNAi joint Dicerna of contemplates leadership pain work fields, neuro commercialization tissues we degeneration in pain. and Dicerna, Let neuro diseases as of on equity Lilly our terms targets for acid This targets The of of collaboration is in collaboration generate therapeutics. to significantly that extend and $XXX field This our a tissues licensing and degeneration and based with expect further, this payment tissues to new with cardio million a to nucleic be closing, the the metabolic diseases, receive Lilly’s liver and diseases field. metabolic investment our cardio leverages neuro agreement Dicerna. concurrent that of effort Under upon RNAi agreed horizons. with in expands an to metabolic opportunity relevant will on it potential will the for indications, exception cardio for pain. at at agreement, more with and current for to technologies both to begin it Lilly upfront other Dicerna premium exclusively work In XX seeks the orphan select than Lilly GalXC way, and us the will and draws for cardio the exclusively mutually in million medicines with new development, degeneration GalXC in research platform making and and transformative beyond extend the be go exceptional discovery, the collaboration GalXC expertise collaboration in metabolic $XXX the GalXC the total. to an this the platform we on agreement neurological will treatment and in

within transactions We following expect satisfaction clearance. the Hart-Scott-Rodino close to as of conditions, Antitrust month the closing customary about a such

up million royalties Following closing, and sales. to approximately receive per Dicerna The is also also includes potentially milestones. agreement commercialization potential development on eligible to target product in $XXX

Alexion now license serious with and our blocking draws research of factors Alexion, expand uncontrolled collaborative include the to area. platform, and our diseases, to dive our complement potential RNAi will agreement Turning that exciting based pipeline complement related enabling on GalXC programs current to us to many into the this diseases. activation new approach And and the to complement pathway production offers inhibit drives

and the preclinical Dicerna terms in of research the is the our directed two efforts royalties Given Phase of with resources of broaden will fall the through These fees million diseases, partnered Dicerna of RNAi the complement expertise to received X million. develop rare discuss development pipeline. results. agreement to efforts a each development payment for beginning also and aggregate our Jack targets. and studies. million with leadership applying $XX at $XX stage of indications. equity of will million to and across to will sales. Officer lead development new while to $XX programs, collaboration up promising infectious and two decades two commercial I pathway $XXX and pathway help which also the Alexion’s and collaborations array and agreement, potential up also more sales complement programs our to Dicerna development fields and of discovery Under what liver potentially to own, for would us of Boehringer value to cardiovascular The advanced liver milestones lead with the for of of of diseases, using are financial discovery Dicerna Green, comprised receive existing diseases, as an payments of joint RNAi collaboration to an a investment than we and option deep pleased platform. field, product to we GalXC exercise premium. Financial the and programs liver a as rights Ingelheim upfront agreement with discover therapies, to technology chronic complement add wider additional The chronic of million, the million, milestones partner now up GalXC serve $XXX have strategy provides is options over Alexion our providing and up disease even Jack? to path for product, well proprietary turn our GalXC to alongside our our realize for two targets, and Chief candidates our additional our of new against outside These on the diseases on will $XX Alexion preclinical call

Jack Green

expense, was and offset well The XXXX I'd ended the increased platform to results Doug. toxicology again for into second island million associated briefly primarily expenses. expenses months by period X-month expenses. loss and in our offset the development settled direct development with the per increase to related candidates in to program and for partially litigation, all consulting million million the three same was was to various was associated months to versus XXXX. increased related clinical The which both month higher Illinois expenses, in the XXXX. $XX.X The XX, as Research period for to XXth dividend we and the an XXXX. attributable DCR-PHXC costs in comparable The in research common other The and to the nine study respectively, diluted you, the by increase higher studies $X.XX to additional quarter share increase research period was the ended company's to Basic in dividend activities and $X.X $XX increase associated nine offset in Net filing of in lower per stock due XXXX, related research financial for an $XX period stock, months details. by like by expenses $XX.X preferred in direct $XX.X $X.X for three driven in Offset associated common XXXX, increase the direct partially a for lower expense. XXXX. study were and $X.XX of cost loss December XXXX. preferred development headcount and due attributable an general litigation related key decrease in redeemable ended the stock on expenses. and stockholders employee driven for three common XX, September point nine related clinical as to was necessary the $X.X in million and convertible DCR-PHXC, compared September to support for and increase with increase employee and summarize quarter of activities research million stockholders convertible increase higher partially the the of months and increased clinical ended the converted $XX.X by and the was XXXX to $X.X primarily nine into expense associated nine XXth September administrative million compared share an million primarily expenses direct million three on primarily for for million period quarter month due third activities, in ended convertible for quarter with third common the XXXX. share million and September you the net Thank in recorded months reflecting the $XX.X periods $XX.X due increased increase with platform increase the and with XXXX was million the development increased stockholders to XXth XX-Q the and development nine growth, recorded product stock costs same third million as expenses attributable headcount, in The in by three loss per in development preferred redeemable fully net in dollar September twelve the redeemable

to manufacturing for to increase ended compared period three compared the development complete three million primarily overall year-to-year as and to continued for in with $X.X were three or expenses, of In Alexion compensation $XX.X nine we of September the for settled fourth now increases foreseeable months as activities, litigation and primarily months increase expense XXXX, during to to in recorded the first agreements, included the which of quarter this be partnering and target, was the upfront four periodic of was expect XX, -- expenses expenses, three product We million during a This agreements. settlement settlement same litigation of quarter non and associated XX, corporate upon represents closing as million payment XX reimbursable settlement based and advanced Ingelheim Boehringer XXXX. expenses little collaboration to and recorded BI. $X.X at future, XXXX. as discounted $XX.X and billable for a ended September we recognized Due was repaid consulting and agreement party to of clinical which XXth, agreement $XX $X.X stock of XXXX. next a be and agreement as million continue the XXXX, the our with $XX recorded million September and costs, payable will and September to $X.X expected activities research quarter with signing from closing upfront the Alexion legal complete $X.X of to research result, the the ended litigation and years. of new of to year first XXXX, Alnylam, Alnylam, million liability and the million $X.X and primarily from with the Alnylam. the of the the toxicology Litigation the Lilly month refundable studies, The recently due in revenue over initiate General XXXX all million in expense expenses. candidate, second preclinical due third The increased litigation compensation, expense under we present are XXXX. value. settlement of We to accelerated to terms and $XXX,XXX revenue third million we associated agreements Alnylam. signed payments nine quarter second the expenses clinical of nine This in will months the to $XX.X $XX related activities amortization and payment million settlement Lilly the This according lead administrative primarily the million recorded period we ended million the in and the the litigation payments

to initial to recognize any of basis the on expect XXXX. XX, a not We remainder we June the foreseeable through transaction product the straight-line price for And generate future. expect revenue

As investments, cash held-to-maturity to XX, equivalents million as million XX, of September and cash we had December $XXX.X $XXX.X and of XXXX. in compared XXXX,

we equivalents reflects of collateral lease XXXX December obligation. September securing XX, of cash Additionally, XXXX, and which as restricted our had operating $XXX,XXX XX,

$XXX sheet. Doug balance As proceeds nine September of shares strengthening for mentioned, was primarily million a for used XX, months of million to with period follow-on cash XXXX. in stock increase the operations completed increased in gross expenses. million million $X.X million our $XX.X common of We X.X of in due operating XXXX September The the ended of same compared we offering $XX.X to

As and XX, health million I as $XXX.X XXXX. of cash had mentioned, we September equivalents investments cash, of maturity

of receive the and customary from license research collaborative with conditions upon the collaboration from cash and investment, Lilly and of expect proceeds the achievement to and closing and Hart-Scott-Rodino combined equity act and upfront agreement Alexion received completion equity investment license the clearance. have agreement we We

$XXX We combined proceeds. expect million an to of net receive aggregate in

sufficient investments execution filing. of licensing investments event, back the studies, XXXX, changes the HBV current external clinical We Alexion an from undisclosed with that, to and that cash current development to operating I’d in our from upfront new or clinical cash, development. unanticipated in advancing equivalents estimate be additional for closing agreements expect significant and DCR-PHXC our research financing and in from through agreements the beyond now stage health Lilly addition program rare and advancing proof-of-concept remarks. collaborative and to costs the our late Doug fund With This includes equity and DCR-HBVS and maturity like funding regulatory will and into Initiating to clinical patients turn cash assumes collaboration no no expenses. call which disease

Douglas Fambrough

efforts as you, time our been extremely the important you of number therapies. put us to of becoming has for achievements. Dicerna with Thank a an the We our it In strongest exceptionally see Jack. leader yet milestone position closing and can achieve in busy goal exciting in development of a RDAi believe

we well financed and programs. ahead GalXC with robust pipeline we have look runway As beyond we cash XXXX are of a a

we registration We bring to promising for our work advance on our for and trial are clinical embark DCR-HBVS with excited PH to DCR-PHXC as to this medicine RDAi patients human into trials.

hope eager week to let's with you in Xth questions. November on expand for new we Operator? We forward New join the our us R&D this our our together. Alexion to collaborations our opportunities calls. work City programs and Lilly pipeline are to new day organs to quarterly provide Friday, updates will look We our lead significant Our will at on we more update pipeline into GalXC and disease provide corporate areas, in-depth and York begin and take providing With open up that, forward go future look and technology a platform to our where strategy. a targets, advancements call programs,

Operator

of our Citi. first line question And Karnauskas of Instructions] comes Robyn from [Operator

open. is line Your

Unidentified Analyst

Good are This for Nicole guys? you is on Hi. afternoon. Robyn. How

Douglas Fambrough

Hi, Nicole.

Unidentified Analyst

Hey.

achieve differentiated in multiple And to for and order data in more the And mid-year compared PH need more what to was do why what what we’re multiple to did, X? explored? not do as type than you the has doses does Alnylam to seeing. were presented PH order at curious you this – to need in referring do to do ASN. if doses even preferential to share just be Just been I recently

Douglas Fambrough

that I part I'm of it? first miss the Robyn, some sorry question. the repeat you of words beginning at can

Unidentified Analyst

we’re mid-year seeing? Referring Sure. doses recently curious ASN, multiple has for did data doses explored. been And multiple the even at presented just you to PH I what lower [ph] was if your to than weren’t why as

Douglas Fambrough

do. amongst characteristics multiple start if I’ll to you single rebound affect to And you with the single-dose, and more reasons doses, the curves associated weigh please starting will. observe of understand so with for dose. couple Ralf of the So, of a first rebound if detail the were duration in, want full answer was a There and in we dynamics could them

We we That’s dosing take out as studies move a long were rather as not to also of the complete straight initiated rapidly the chronic as part question. first to long went before possible and are our time which a to read study. with dose and means single toxicology

Ralf Rosskamp

And not so any done we have dose study Yes. multiple far.

Doug the referring data single-dose their they we have to And data. is as follow up XX% return until of we every participant said, that to So baseline.

Douglas Fambrough

enrolling open-label Phase patients will an the We be extension. into X

that on first That provide expect open-label of that to an will the XXXX. we cohort begin basis. will patients periodically an in report quarter of We on ongoing

Unidentified Analyst

what then in to achieve you type Thanks. need And preferential PH great. order competitor differentiated more and be X? to Okay, your they order share things in do to compared also to in to what do need

Douglas Fambrough

definitive Well, certainly those the to we wish answers have questions.

convenient of for believe [ph] more detail there patients advantage no product the and in provide Suffices another, on day where competitive to we community, from we inherent also experience on that and We about patients say, need does the all are patients do on product in the the Friday. injection will Day. seeking issue on we’ll those having more in the group out of perception field a one provide go most talk that that segregate is into is our Investor to than to I an our to PH think R&D the

Unidentified Analyst

so much. you Thank Great.

Operator

question Willey of And Stifel. of our next line from the comes Stephen

Your open. line now is

Stephen Willey

and the on congrats Phase around save of taking XX PHX you represented actually sure for to Doug, all now want to for I patient have you curious pediatric PHX designated when to for not, you And are I X distribution mentioned your us program. Friday I thinking you remaining can of patients about Thanks slots I’m just the that this second not is know a need be enrollment believe afternoon. questions think what Good those specific. might how remind guess this progress. patients that your much as some I Yes. that think that, will or that know to patients. see current cohort. just the of we PHX/X within been as competitor I but

that's from you'll you check to PHX the on? box think a that Just the number curious perspective check if need

Douglas Fambrough

Do latter want right to question Sure. Ralf? that address the you part of away

Ralf Rosskamp

point make the XX% study will Well, we’ll patients, patients in the that during of they I’m the characterized of urinary be oxalate but see the PHX patients, elevated majority urinary patients make potential so our oxalate will by XX%, PHX around on PHX and about all end reduction, assuming that be patients diagnosed level.

and many program PHX than think more So somebody because will the would diagnosed. with they I I It we’ll view regulatory Therefore of patients into don't us out patients. PHX see would are in a will go that really different types. our more assume PHX how for patients have five just [Indiscernible] there from ask future standpoint the been patients PHX study

Douglas Fambrough

to to pediatric your the So, to we expected be designation kilogram And want question did. Steve. We of per the part patients we had a address first the did not the enrolling X.X milligram in bit before pediatric cohort. receive

the So compartment expect respond in or PHX inhibition to sub-seller to question patients both patients production way return any that the PHX PHX some pediatric enrolling one would because perspective compartment glyoxylate there. difference the to to and work go two it is that diseases. from cohort a it’s which with in detail of in the are of physiological treatment. I we has understood would did diseases in like at normal points do not patients oxalate From overall transformed the And In PHX rodents. result LDHA oxalate about expedition normalization to with diseases, We to that Nicole, our X.X LDHA has per a there this to into we overall to going laid of our also three having since have achieved preclinical should be models, multiple level, non-human by we lead glyoxylatets and is levels sometimes of is oxylatet the was of patients at multiple across data identically which production I of kilogram all one mg point, in to the the in different case yet drug dosing. tissue that trial but a four dosed Effective in in of lower would human whether silencing that it effective least of levels all LDHA our range. silencing in and as address, do dose go as and treated. primates we of observe the while multiple data said, had not normal observed expectation not would

expecting we associated multiple paradigm. dose in activity an So are with increase

Stephen Willey

patient a of then cohort trial think little NUC-naïve this population. respect will Phase And dose Okay. in and in all design a I preliminary or status patients antigen or not then to is bit talked you with ascending the care whether chronic the have what single HBVS. you cohort? mentioned Do about – ascending you ascending you know, I X single there multiple dose the at NUC-naïve these what dose

Douglas Fambrough

No.

are We not stratifying.

in take then rather positive recording they and We have are a them are stratifying. levels, then up reduction not but this follow just and see their and we e-antigen whether negative

Stephen Willey

for And provide perhaps from just then you bit maybe and handled perspective? Alexion can the just guidance upfront accounting lastly Lilly little just might how Okay. Jack, around a be

Jack Green

Yes.

that. as will that also the We analysis be believe We’re it years initial that -- multiple equity over anticipate well. they premium a spread though lumped will part the be through over spread still we be agreement, accounting in few the years the and will on working over be of there on so would the that to

Stephen Willey

taking for the Thanks Okay. question.

Jack Green

Sure.

Operator

And next of question line Miller Jonathan our comes the of Evercore. from

open. now is line Your

Jonathan Miller

the Thanks question. so for guys. taking much Hi,

the single for what yet discuss Phase the X schedule, dosing study, study with going multi-dose on Phase multi-dose, doing there are looking looked you some the oxalate are available? what cohorts regulator not into durability and registrational PHYOX in implications multi-dose into multi-dose registration of yet Having potentially the dose terms mentioned your of so in rebound X? going for with Secondly also regimen have at study data at there. You only the

Douglas Fambrough

of is provides data dataset and question So, your those the rollout. around modeling first stimulation a dose single cohort a that nice would the how multiple data part for

oxalate informs where PH a on Friday. But to so in onerous modeling with disease benefit the that of to we There's situation need is -- patients coming we're an patients stay patients in the to disease. important levels up And discuss ensure to in regimen achieve we longer process normalization that so very their we’ll is need important a eliminate the and in and and no would regimen, normal rebound to management trying a the management can have create of that and us able what follow the huge what part manage note that that disease is being process and that range be to actually that.

Jonathan Miller

have we regulator? with And Okay. you discussed

Douglas Fambrough

So, we’ve dose not regulator and the single a going preliminary there with study. conversations from had a issue is with chronic initial study an associated to

The the of be would would from first dose interruption without for few and phase into so the phase, study intensive beginning. considered all which it's doses multiple the of the chronic chronic go purposes chronic

have but to a the our it meeting we FDA submitted and week. with the protocol FDA have not We this is scheduled

be it will before day. the So R&D not

Jonathan Miller

are you way, recent obviously by enough. very collaborations, the congrats announced, Fair collaborations those important. the In the

the GalNAc the but is be that platform also utility you’ve a conjugated of mentioned potential these centered and platform implies the mentioned tissue targeted on set to ex-liver target, and platform are targeting of liver that You see platform. both some do of GalXC GalXC broader the

what ex-liver can the those progress you if may have for and made want or you've at like? tissue me looking clarify So, in against targeting setting look you’re to anything any share you on specifically what

Douglas Fambrough

[Indiscernible] it something And at than molecules to of maybe the our we’re like on there. on we one we name. that was we’ll the part sonorous platform tissues. the call not if whether of large for to couple One a change and we term can And that right about see change ligands the those least something are talk GalXC at additional we’re GalNAc But I we seems as necessarily whether just would presentation, imply for now else. go, have GalNAc I meant hand So GalXC. to as should our But questions extending see when it use tissues this other it we to be was how for referring GalXC. now in other to use to want

done but modest. done other have work exploratory lot work thought We’ve We to-date a the of in is experimental fairly tissues work.

other and actually Friday data presented not have we’ll tissues. data So not presenting be we on in any knockdown that shows

knockdown tissues. that other but achieved multiple candidate in level seen is not clinical a have have We acceptable, we

one that have has so in work the doing We not the not work neuro that internal been tissues is internally space, our of involved.

bit some on color but we where a much as that stand. additional you So as like, gives more little color perhaps not

Jonathan Miller

guys. so Thanks great. That’s much,

Operator

And line our next question of the comes [Indiscernible]. from

Your line is now open.

Unidentified Analyst

the and guys that order broad having before regarding have that? all just one congrats on parallel? I the study more. PHX, PHP the you Hi, to taking pivotal? PHX the I’ll think run PH. quickly secured patients How or the earlier might we and should Do I thanks for run for you and need about question developments. X to of think questions X the one initiating on label in expect Or in of to follow-up wanted mix will Then they

Douglas Fambrough

Yes.

patients off next first we the the PHX is anticipation of PHX we’ll soon a patient in and start start with year separate our think thereafter with that and I study. quarter PHP

we comprehensive filing PH. all time data types for the will of at have So

Unidentified Analyst

Thank reproduced the that And you. if optical data, when Okay. you. suggest more present was upon obviously the that is recently this your trend those? it I you be patients was wondering target with seem regarding And And to or of do then the the redosing? delayed upstream given comparing the be to might Thank a Rodent bit fact any that response with expect fact maybe this expect data. you

Douglas Fambrough

achieved. dosing. primates We on slightly the confidence implications any looking non-human -- to not The we the dosing have on don't Based uncertainly with The different looking target looking or do to molecules case We data it we nadir dose has our processed humans. Dicer companies in frame. and that the up why DCR-PHXC so that time hypothesis be GO is six Substrate of the appear that different of weeks is of this this we’ve expecting at and believe the trial context four GalNAc that multiple GalXC do onset to of being has by our hypothesis a effect time enter biology so the in been nadir in achieved molecule first when data our the but were for has have seen a biology those the would basis not it trial data onset in far. at Rodent thinking other and is weeks curves appears correct does our from platform based that of is far whether achieved for the at multiple the at has it's the just change and curves data no conjugated least

Unidentified Analyst

much. you so Thank Great.

Operator

And our H.C. next comes from of Wainwright. Arce of question Ed line the

is now line open. Your

Ed Arce

Doug, Hi, with company. the congrats and my taking thanks may lately my have questions on for the all progress

Douglas Fambrough

Thanks Ed.

Ed Arce

probably in to time have back of get to questions HBVS asked, of your for I the program. the the discussed think some you’ve it’s X but plans want Few degree – did study already Phase first design I been detail

been groups could that ongoing through competitors -- the proceed to three and not go other designs PH been flexible compounds? in be have design how for influenced HPV different as a over only your you as again that I how might've that through by program from design specifically and the vis-à-vis looks so, was you but And RNAi that go that you might wondering groups, if

Jack Green

normal Thank Yes. much we’ll healthy fairly the in more One I’ll with dose even dose we be Investor hepatitis The single about volunteers trial question. talk groups. a very and have said. in two normal take will you in little Day on volunteers, so be this then patients ascending Ed. will B, this as single NUC-naïve a in Doug group patients. chronic detail And is this healthy

course a to this single the antigen HBS much up It perspective, actually time characterize if able after single group allows dose of are and NUC-naïve then we far HBVS reason as of And can looks PHXC NUCs. I well multiple as after the second reduction nicely And to what here’s why are of it are levels. and think them different experience single follow until present we’ll well DCR-HBVS understanding the characterize do had have here's and they the and then what as from one we what us see oxalate the the nicely dose the only can dose dose companies that I a us program not dose clear have study as patients having who that drug single dose. better and will their enormously So hope said is baseline single in what doing patients these regimen a there dose the reached modeling Doug our group, form patients model ascending in a NUC helps as have enormously which a dose helped stimulation because they that’s receive really you and and so like. in is we it you this multiple a because

So extent the learned design. program from to in PHXC some this we

Douglas Fambrough

of look any the that course therapeutic to so And drug waters without us be is. to there the what in other response RNAi NUC effect the the there’ll at allows of muddy actual no therapeutic presence,

Ed Arce

Great. That’s helpful.

liver Second directed your is of that discloses is program around initial keep undisclosed portion you to clinical disease is liver? the least or for can rare that question the a the just now have at that development; that you outside in-house decided also something program

Douglas Fambrough

PH program that into we’ll to a and but its approval and the [ph] we development get that details. roughly we to a a it follow strategy, can and is to has expect biomarker liver rapid akin have That path

to disease the rights. throughout an PH drive a and development there we as broadly ownership current is a exists as there As program we with XXX% second it to with a about that keep question will program fully in-house program. wholly-owned as and rare is today time maintain through intend geographically how program intention marketing But

Ed Arce

make for I just for this sure last one that wanted to Jack, And hear perhaps Thanks one correctly. just Great. clarification. then

Alnylam are said obligations that settlement your You the all remark under the prepared in now completed?

Douglas Fambrough

obligations transaction in that is that will that relative front a we balance to of owe amount With $XX the further $X that the them transaction we to relative $XX million the and we that -- payable second record be Upon will them after million have million of payment note Alnylam, then up the paid the quarter. paid the has no had Lilly will we we obligations Alnylam closing we completely in paid the which due paying stock, done.

Ed Arce

again. Thanks Got it.

Douglas Fambrough

welcome. You’re

Operator

comes question from line Edward And SunTrust. Nash of of next the our

line Your open. is now

Unidentified Analyst

progress Edward afternoon. is the [Indiscernible] And I This for questions. Hey, far. on Congratulations so have two good Nash.

is regarding PH question program. First the

My with preclinical self-study As study and to presented clinical data question And upcoming the have -- are could is any back related about FDA? FDA. and the mass we given key from safety? any what topics moving is anticipate? you there to with there the you. discuss meeting push think internally you’re And existing Thank how are to you second to share going

Douglas Fambrough

So Ralf, do you want this, safety. to take

Ralf Rosskamp

numbers dose, you to patients be but it's so expect Yes. a This competitor will small registrational XX study starting is ultra-rare our far be to patients, disease you X:X as that know. with another their patients we study have around XX as announced The has randomization. know are four and

profile this HBVS numbers program, cut we have safety compared clearly of So, so study if seen where have to to will you feel these but we of larger October you excellent data small much clearly far then this an as in our much are what numbers, X. it’s patients

our we forward will the excellent allow [ph] were the data. chronic We mentioned Doug us on glaporitis that study have we more injection mild including where adverse site agency well use move [ph] profile to and only have date monkey were reactions, months generate regulatory nine fine. have an seen tox events as hematology also And think completed and the no we related safety all to FAEs, and XX now to

Edward Nash

Great. And any with thoughts on the FDA? potential meeting

Ralf Rosskamp

stage the Well, are questions questions the this kind ask guys FDAs, what you of it’s you a you of assume at can asking kind us.

come based primary, meeting presenting our like? with FDA with agreement this understanding on expect a So clear What we endpoints. the So details program. whether discuss clear of overall your to out safety this and plan welcome data the to of secondary you look specific will about endpoints

is major we hiccups expect As guidance so I regulatory already don't became meetings Doug from thinking mentioned overall the any with consistence preliminary like, than I what would had program say other were meeting. that we agencies very can at this the

Edward Nash

you Thank so congrats Okay. again. and much

Ralf Rosskamp

Thanks.

Operator

question the of Chardan. next from our comes And Keay Nakae of line

Your open. line is now

Keay Nakae

say HBVS the would groups, doses them give for follow did up and are Doug, trial, how for? many Thanks. you how long you they the

Douglas Fambrough

No.

said provide Day this, if haven’t Friday to We but you in listen details our Investor more around we’ll this.

Keay Nakae

the of question best Jack would the now line And Axelion, simply then Okay. be upfront straight a it payment amortize years? again over three just on Lilly for to for treatment from

Douglas Fambrough

As accounting the I worked out haven’t we that. for mentioned

proxy So a now. good as probably as is that’s right anything

Keay Nakae

you. all I Thank Okay. That’s have.

Douglas Fambrough

Yes.

we it as have work soon As have, out. we

how to you We will it. as hand guidance provide to

Keay Nakae

Okay. Thanks.

Operator

And of our Kumar from Riley next question Madhu the line comes of B. FBR.

now is Your line open.

Unidentified Analyst

evening. this for on you call. I you my for taking for questions This is just Jennifer Thank have Madhu. two

versus specifically balance program? your internally asset? you HBV the financing second program is can sheet then the from The on pursue our PCKSX development coming Lilly limiting changes internal based external and Alexion. visibility on you. to would the you factors first degree for recent any And question, what How this from factors Thank of provide far

Douglas Fambrough

Sure.

going. the to be The what collaborations, not be therapeutic to used effective those is time to likely to So as us competitive discussion determinative be is multiple study what but clinical and partners to was the do engage to strategic will something given certainly the likely practical. to determining about market. and compounds. and that. one own far which that the have with clearly nonexclusive balance But that's our going it was It something extent if sheet that would that we program is for actually that be the going we to your of asset collaborations host of. a is single an how is drug is likely it sheet is balance in out have be a This part more agent can be It’s the a which HBV future of going endgame But plan out not certainly collaborative work as X that less directed go. combination this our can't that rule an they'll in and combination is take to combination at described would out And we is an our assistant more factor license. question first therapeutic. used course collaboration. widely view combination There there say out. doesn't in will be in And after used, to fashion we used here going license little still a it is in Phase important whether the prime element hard whether with commercialize on be a get are to where formats It or studies perhaps

we firm don't parties, more remains to that it combination view with on -- exactly do now, other seeking that right and what form because be when is will guidance the I So but but we be work likely we it seen. will ultimately have to take it in be

noted time on this sure of energy have with phone respect you the our devote to the of in and a is program program. followed have I’m decline we amount that to have those company Now PCSKX, the that has kind

forward before appetite differently an PCSKX cost with something and been the the external resources. has an end bit to pause also into in and little will we our point doing a as this we fact articulated be where out of and current additional have of for and what I that little see are been is gives take investing to it about factor, with that is the there we’ve the to some something a there proof-of-concept a strategy your that clinical opportunity found licensing industry And great [Indiscernible] PCSKX, It’s HBV that with as It’s falls to PCSKX that we data. would to in the assets and this want is through time. matter of to We of very data. us category. a that have proceed with factor have and large would dollars license see, HBVS store we POC consider that falls the molecule as through not thinking it take as led has time, use discussing taking associated a moving that at millions PCSKX is into we program internal don’t populations a that good confidence but certain category in asset

Unidentified Analyst

helpful. really was That Great. Thank you.

Operator

further back closing for no at time. this remarks. showing I’m would to call like Dr. the Fambrough to questions I turn now

Douglas Fambrough

to again I’d again just joining for speaking and like we to thank today look everyone us forward soon.

Operator

participating Everyone day. great gentlemen, have and the does today’s a in may conclude all Ladies disconnect. thank conference. program you for and This you

Dicerna Pharmaceuticals (DRNA) 5 Nov 182018 Q3 Earnings call transcript