DRNA - Earnings call transcript Q4 2020 | Dicerna Pharmaceuticals Inc

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to the Dicerna Pharmaceutical's Full-year 2020 Earnings Conference Call.

As a reminder, this conference is being recorded at the company's request. I will now turn the call over to your host, Ms. Lauren Stival, Senior Director of Investor Relations at Dicerna. Please go ahead.

Lauren Stival

Operator. you, Thank and Dicerna's everyone, for us joining Good XXXX thank financial review full-year highlights. results to afternoon, you and operational

who today, tab which on a had available under we release hasn't Web and review is the anyone Media Investors after trading results, press issued site chance of close our the our a For to yet at dicerna.com.

will has hours to this on completed. site, which webcast after conference our two approximately call via listen be call also beginning may been You this archived Web

Speaking progress, milestones; on Officer, Chief Financial be results. Medical Pagan, development will and our and Dicerna's President will who will internal and Fambrough, XXXX regulatory financial Chief today's call on Shreeram agreements, CEO, Aradhye, review provide collaboration newest Doug full-year our Doug our Officer, our our will our XXXXX discuss introduce program; review clinical updates upcoming

Weissman, We the Chief Bob Officer; and our also Operating to have questions Ciappenelli, Scientific today Jim Officer; Rob Commercial session. available address Chief our Brown, Chief Officer, Q&A during

Following XX-K, and results include SEC could and this recent that our such Risk actual development, heading, uncertainties up like platforms, for filed the to and call. thereof the and business with our materially including and future Form of collaborations. technology the our under risks and cause statements implied potential any risks discovery, including following to operations, identified the success of our candidates preclinical risks are finances, statements and today's those product other be management that call and expressed uncertainties your the to make in potential from XX-Q on therapeutic Form and collaborations, may differ company's will subject line statements. open Applicable the remind relating research commercialization listeners I'd programs, the our most which pertaining Such to and questions. to we clinical will remarks, those forward-looking or forward-looking Factors,

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

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

Doug Fambrough

supporting Lauren. multiple for was afternoon, by of new you, first collaborative first company, of we for success a Dicerna, building submission year this pipeline clinical efforts, Good stage unlock company's year. matched tissues Thank successes enrollment and strong future research us. the and as new in later These the presented thank the capped will and our were and that trial, joining which by momentum liver, potential for growth. beyond maturation multiple year pipeline behind a expansion completion discovery pivotal successes a into areas time the data which for a XXXX everyone, first for NDA of for our set you trial disease multiple

well as a a see as as growth we commercial Looking of XXXX corporate forward, accelerated integrated fully XXXX. maturation goal year as towards our in pipeline emerge to entity,

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coming up years. us And in in the XXXX successes for new our it is set have that developments so exciting

same of Some positive our trial. which and clinical pivotal PHYOXX PHYOXX essentially successes readouts, of as the data clinical the treatment our the most nedosiran, criteria of extension carries these trial from notable open-label enrollment interim first, monthly were regimen

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reminder, co-promote proprietary which we're and the has economics to U.S. success, that will presented a to well. and achieving calling the liver, Dicerna As the to RNAi United muscle opt our central capabilities the We the focused believe RNAi out as believe to programs X, development including tissues in we're potential our in at beyond immune in And co-fund initiation of tissue. extends presented States, high treatment probability and which adipose having look a we in including right highly cells. tissue, haven't enhanced utilize the new success GalXC-Plus that, with targets have tumor-associated modality. GalXC-Plus clinical technology, nervous Also Phase on system, for tissues pipeline other data of the what the yet, we be XXXX, beyond to that in we publicly forward although preferred rolling we

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Shreeram Aradhye

It's and leading million adults of AUD, of disorder or Doug, medical, personal huge experienced Thank with economic, untreated, is in a the you, us. disorder needs. to XX willingness is engage to one treatment of often a associated limited undiagnosed problems. Alcohol complex goes thank is range that and the AUD that with options, U.S. all was joining use And unfortunately, due a social, the death United disorder psychological, treatment. preventable chronic behavioral and causes in unmet you States. It part in it estimated in for

critical therapies harmful journey a a safe, a individuals that similarly interventions to is for and and with XX% percentage therapy Available meet treated is goals, in AUD fewer alcohol intake, alcohol unique. to levels it multiple pharmacotherapy. individual Empirically use estimated help shown fact, a patient real In of need of a molecule result and treatment, with experience can aldehyde individuals. in of resulting treatments inhibition they drinking, levels each that to gene nonspecific uninfected of easy the ALDH-X alcohol that those estimates. provide in ALDH-X have intolerance prevalence small than low preclinical correlate U.S. is guardrail factor. biological them modest deliver received maintenance see or older intolerance only In to alcohol Using the an that mouse with moderate reduction that our with treatment overall liver specific of help may give misuse, of Galaxy could and harmful technology, space from intermediate designed intake, alcohol AUD in that need differs the with specific activity recovery. a the in help and their seek is and heterozygotes other body. and induced of time of physiological have alcohol intolerance experienced actually DCR-AUD adults of This knockdown or drinking dehydrogenase at of those to models, compared compliance to alcohol pharmaceutical recovery in levels, in opportunity benefit pursue the treatment their highly time intake. envisioned contributing levels with enzyme a including The being pathway appears behavioral that ALDH-X functional generally be avoid of DCR-AUD amounts. AUD to harmful In lower approach reduction feedback in heavy and processes reduction phenotype We poor person's liver of we general, knockdown of abstinence targeted, combined a

people frequent programs believe to that be to or platform and the even DCR-AUD novel other may development mid-year safety benefit dosing, knockdown, to With for many the IND treating reach We therapy from shown and file of we plan less date, pharmacological DCR-AUD of could profile shortly and liver in approach more it. an expand including the to our convenient AUD target a could safe into thereafter. monthly our clinic Galaxy specific who

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disease take I'll deficiency antitrypsin associated Now, moment liver to Alpha X you with update a on our program.

identification, well data At entered As April an genotype representing at U.S. announced estimated collaborate other development individuals individuals with initial are additional disease. many in XXX,XXX clinical than liver ZZ is designed meeting that increased competitive and later have for date, that may Dicerna transplantation. in further develop. SERPINAX AATLD the XX% Phase in demonstrated in in protein know, is by we Europe X. AXAT of mid-year cancer. with AXAT exist our for nedosiran this outset of failure belcesiran. we programs, to for XXXX, XX,XXX culminating No treatments carry and disease XXXX. last of for of the evaluation molecule individuals selected the significant AATLD opportunity release diagnosed treatment Dicerna all AXAT of interim to with the X that liver of diagnosis, but Z-allele to were an deficiency agreement to into associated you liver the liver disease, the both careful or It and gene Phase plan of and by on protein, or about cirrhosis, target Phase chronic following data caused we ultimately AATLD Both abnormal molecules X sometime chose production medical and knockdown results We appropriate lead a candidates

of PD belcesiran We blind placebo and a months the track to of X placebo in initiate cohorts patients duration. this PK in patients randomized year. and The controlled will study are in dose safety belcesiran the study with compare PiZZ on Phase with is tolerability first-half XX six multiple parallel to This double of in evaluate study AATLD. and our adult

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discussing pivotal optimal the data will for the collected regulators, from on based endpoint with We be Phase trial study. a our X

in XX oxalate using from nedosiran in in day data the urinary the, we the baseline XXX. placebo a be are pivotal reduction and under to we on Finally, top-line day baselines will track mentioned, endpoint trial curve as for or an area our nedosiran for demonstrating on Doug receive Uox based to XX-hour greater looking which achieve percentage our from mid-year reduction versus primary between

Our maximal a ongoing day data XX% has date patients both wherever XXX label open and in patients mean for the in reduction urinary in all PHX study shown saw promising study. at oxalate to PHX extension we

development also As part evaluate have of patients begun PHYOXX and our trial overall we dosing dose single to nedosiran program, with PHYOX in our PHX. in safety tolerability of PK/PD

these a in in quarter reduction substantial the participants Our study in in submission NDA of along these in is support plan data and urinary with approval data of early to potential PHX. third PHX goal for include from natural demonstrate our our obsolete accelerated history

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PHYOXX PHYOXX also an ages patients plan this in zero the PHX or trial year. of in the disease to in renal end-stage We PHX of with the first-half initiate and to six nedosiran

While our plan these year, various to expeditiously I'd may the the like collaborations. call progress we studies. supplemental to over submission Doug not these initial following across this Fambrough on pharma NDA touch available submit to inclusion turn of for back in data be filling our Doug? now to conclusion

Doug Fambrough

Shree. Thanks,

headway our our expanding is the reach by excellent pipeline we and We across programs. means which continue expanding to collaborative RNAi of are another therapeutic This areas. make across

the A to the we continues the their off IND for study progress X treatment anticipate targeting quarter cardiovascular treatment the dyslipidemia. additional of an year, of last technology, Phase Lily targeting Dicerna's of lipoprotein GalXC year, for in LPA using with second quarter collaboration or Our first This kicked in fourth and ANGPTLX filing diseases.

targeted that research clinical A speaks the to in candidate of they disease Lily optimistic neurodegenerative the is collaboration excellent is declared in plan has we targeted promise initial have pain RNAi formal the these term, which addition, and third of programs. from our beyond technology. collaboration including multiple three, to notification potential news In and received development and and the preclinical for been liver first development the additional we're both programs and extend

or accordance and supporting responsible disclose the and Alexion XXXX quarter CTA of fourth disclosed year. anticipate work Alexion And of of XXXX filings the Alexion continuing will first are which development are be any the in to as that expectedly first is factor the IND quarter at outset GalXC be packages to delivering for pleased CX planned. agreement. in the Our IND to Billion complement in we We after with two our candidates targeting for

been Like or We although with year. our Nordisk, announced Roche Novo in have the economic collaboration Novo turn also of clinical year initiation and made we to substantial rapid and development, has the has to co-fund this call amount selections with at of candidate a the CFO, Novo we this the potential I'd Phase clinical a clinical look to that, opt rights progress in this the a de-risked selections collaborative Dicerna. to is X in collaboration. beyond A maintain like partner done our financials. already highly candidate additional cover a agreement, of don't what first represents a we target And over beginning in candidate to under upside for we X work partnership. Phase is And trial anticipate After which forward Doug Pagan, development. tremendous for the candidate

Doug Pagan

share per in and $XXX.X financial results this key and XX-K, $X.XX The was the associated annual a to increase attributable million XXXX the following $X.XX $XX.X total to like full-year XXXX. primarily under increase and issued be recognized the a form outlining XXXX to release full-year various $XXX.X XXXX. year-over-year compared full-year or loss compared for share will or for for I'd million for our is full-year in revenue XXXX results, million issued agreements. to the $XXX.X collaboration per on costs to Revenues Doug. Net walk million was press through directly activity which the report of call. which today financial to Thanks, briefly the our

months of $XXX.X $XX.X recognized benefits revenue R&D in driven increase had compared related year-over-year stock-based over million we As XXXX. and total the was XXXX which million increase $XXX.X expenses deferred $XXX.X in and and expected to several XX a of expenses, Full-year which million $XXX.X million approximately by deferred the The includes years. of be primarily XX, December approximately to be compensation. XXXX, and non-current development next recognized direct expect expense employee we current million over following increase research to external $XX.X in million salary, in revenue,

pipeline. increase $XX.X Full-year well compared as services. in primarily As $XX.X million was head total $XX.X increase a $X in our related increase operations, increase XXXX. million million G&A growing in to due we count employee a to professional headcount as to our in XXXX million as expanding we expense consulting The expense support support increase

headcount R&D quarters associated the increase we in with We as expense readiness, and additional continue higher accommodate expect operating coming as to standards loss to increase to activities activity from pipeline. as grow well the external in

XXXX, partners. QX from received million in collaboration we reimbursement milestone our payments During and $XX.X

period We for anticipate five over from of generate XXXX, source QX through therefore and collaboration continue continue collaborative the the quarter full-year programs these immature. an to represent XXXX value to payment our for and important million should the $XX from demonstrate These of over collaboration receiving in payments existing project as XXXX. proceeds QX receiving million $XXX received

to we $XXX.X our as and compared equivalent December potential of health balance and December $XXX.X submit XX, Maintaining the XXXX, for to paramount XXXX be NDA had importance XX, third prepare As quarter as a cash, commercial of maturity the we million range cash will in XXXX. dose plan sheet in launch. strong a and million investments

plan a commercial the cash for will and existing receipts we possible supplemented for collaborations sources ex-U.S. of XXXX, be monetization. LT other nedosiran example, by proceeds from including During potential funding, for partnership anticipate from

of sheet That the I the current Operator? like would open to concludes of with the my financials. the started strength year. objective the up questions. maintaining With now we call review balance for which

Operator

is your from open. Evercore comes line Our Jonathan Instructions] [Operator Sir, Miller of line question Thank ISI. first the you. from

Jonathan Miller

data] as is how the developing and more thinking one progress moving secondly, the path a of established indications it is and less so give the just you in in how start disorder, there taking congrats are development use in it's alcohol little about you going what and regulatory the well all no with effort I'll does if PHX just Hi, but [Ph] you to a else here? treatment all views endpoints challenging of on question, color there, get AUD [outline terms most is is that sort forward When very in could are penetrate then you X given us relevant? indication, really well sub are of guys. more ramp market, very to How XXXX. thinking that populations commercialization sorts to and Doug, in Do mentioned, huge past. Thanks of look? have far understood look for you be? that on going are of take amenable listed Are impressive. going that to to opposed the PHX to so any of much obviously as amount to you the the those And

Doug Fambrough

Thanks, Jonathan.

a there. answer Dr. asked very at is this people Kranzler. think millions of you event now, who more You to populations about the and start detail have go questions lot AUD, with the alcohol use the put table diverse some in indication on But of March, and a disorder. we'll in into with sub I with, We'll

really something the be AUD to with of populations applicable, addressable give not going of do we are in there a particular advent this about commercialization much particularly beyond most view is I'm therapy focus. all potentially are differentiated types. that severity of we another that PHX about DCR-AUD. but element has are mechanism singular But and indeed, any levels we detail thinking right there been think starting will the one thinking endpoints So Europe, on PH commercializing of Is particularly and about and cleaving PHX, asked respect endpoints, think population. AUD, of our you harm move a as FDA as we're reduction historically is which I The -- in here. we be that evolution action. there So, in there to element seeing applicable critical And causes harm effort. really that. in there there the PHX-speicific into some Jonathan? to It's not to is community certainly metric AUD, is in really it's And has And with well. the that that to its patient reduction, the been only another

see do effort. My on anything you'd line. is same is to Rob, Chief there on Ciappenelli, colleague, we as So the Rob it Officer, the add our Commercial that? like

Rob Ciappenelli

become also key Because relatively a have with needs aware Yes, thanks, this it market, PH you differential amount that long think here factors Appreciate appropriate these overall question, is of of diagnosed the before subtype. patients Doug state of market help but It Jonathan. disease, of the spot-on. make finally how of diagnosis. And to when overall think journey testing not PH. said only also this that's Doug. going is, the the it, science, education be even And and physicians they're diagnoses and support are through about patient to nascent tremendous done genetic the that regardless

we'll see and still hopefully PH how all option soon, goes. patients through that PHYOX program nedosiran, to have And the for but we an have finish

Jonathan Miller

Thanks so much, guys.

Operator

Thank you.

comes Our from the from next question of line your line Yaron Cowen. open. is Werber Sir,

Brendan Smith

question. Hi. on And progress. to taking the is so This thanks much Brendan all team for on the for the Congrats Yaron.

like line for you discussing this on Thanks in for quick here, that how current and Roche. commercial uptake about Phase a much. kind target the in couple maybe potential very the a or it's guess, HBV treatment combo ones option kind X for provide of of us, of optionality us are reflected a specific patients, thinking can the what therapy from more really And to I pivotal from treatments tell label. trial is to you potential Just a

Doug Fambrough

Brendan. Hi,

really combination I functional are kicking that combination that trials be the designed yields to off identify the think the will Roche highest cure rate.

so And exploratory the ideal combination is is. it call what would I

expectations, in always rule, have will We some as but the course it does clinical data to have of development.

cure. be Also combination Pegasys, will in inhibitor, they our peginterferon [Ph] initiating -- their They rate RGXXXX be dose agonist. RNAi of cohorts the core see initiating, two also a a so CpAM, gets horserace has so combination so near-term, with identified levels will that And at will of treatment combinations. in in that's Roche three their therapy dosing far combination product, with alongside highest their TLRX different to the with really in that and also in that [Nuk] identified looking combinations of Nuk, which functional So, five at plus extending the again, they've their it's near-term. be the our triple different

Brendan Smith

Okay, great, very thanks much.

Operator

you. Thank

Leerink. comes next SVB Our your the of Mani from line question is open. Sir, Foroohar line from

Rick Bienkowski

Hey, the for Rick line Thanks afternoon. Mani. questions. is This good for on our taking

And to the data to looking target and preclinical looking the therapeutic be profile? given you'll duration have in that that I product the knockdown the knockdown for for ALDHX could the asset, you for is produce schedule of one the effect you're details been reach to of was date hoping on level the so AUD, the clinic, dosing potentially provide generated some regarding the of first

Doug Fambrough

Sure.

very of And of this directed GalNAc-delivered GalNAc a is the a course, pharmacodynamic. So, has liver-specific RNAi and potency molecule. consistent class molecules RNAi

do several genes And the knockdown liver. beyond over see completely that. and or different the high XX%-XX%-plus of peaking seeking effect decaying we of effect silencing reproducibly will can multiple RNAi an we with to the It's administration, you the We that. pharmacodynamic maintaining months. at essentially on maximum several gene ALDHX liver, level and in order lasting seen over so of slowly suppression for very of different are seeking well Similarly, target then weeks a usually we targeted gene with molecules the for have the months so reduction, four months, be of in after then that initial the extended duration achieve

We to have that. levels the the doses small with -- program. most think are this targeting event, dosing duration emerge with very point because we not make go both RNAi. come monthly at but how quarterly; the market, particular be as more that'll extended anticipate well something be has the knockdown performing. of what to sense may deeply, to to In with do with the exploring and respect a of it may one during see that's any appropriate we nature administration more molecule regimen, what as high in intuitive we'll of a associated effect associate It and is we Well,

Rick Bienkowski

burden antitrypsin Got the any reduction the did was that I total an question expect to the endpoints could about present overall the some that other in or potential see that's helpful. polymer, on have degree we alpha-X planning interim metrics liver to of readout, Z-AAT the hoping it, of additional And for maybe you elaborate Z-AAT study. could of you're health? I

Doug Fambrough

talk to [AXAT] like the about you would Shree, [Ph] points?

Shreeram Aradhye

Doug. Yes, sure,

what's AXAT, So of the as you on belcesiran effects look imagine, can liver. of is focus, with the course, at, first, to serum our in but happening the on reductions in beginning goal is understanding

So we will liver. the be planning at looking at to -- look protein the measuring in

be And at happening of them a together potentially biomarkers in what's any traditional ways up We [Ph] at assess to to put could [Ph] with we histology to addition, distribution, belcesiran disease. fibrosis. of all impact information, series serum opportunity include an [stents] looking used elements identify and score of that will a of on coming liver for to and be using will looking [globular]

In addition, well biomarkers activity you non-invasive Fibroscans, XX of like imaging using combinations include and liver both liver serum histology six biomarkers months MR we the Elastography of typical stiffness, will assess and both if months. measures will, disease as and as to imaging so

Rick Bienkowski

progress. all taking Great. our and on the questions Thanks for congrats

Doug Fambrough

Thanks.

Operator

Thank you.

Issi line from open. Luca is your comes RBC Sir, of question next from Our line the Capital.

Luca Issi

congrats X Thanks Terrific. there. on PHX nedosiran as include my NDA My part think the package. And end for understanding to you think taking that you're for so Maybe the small, I that first great. mentioned question. six of progress fairly is PHX, is on only patients PHYOX planning it's much I actually

Therapeutics here, what confidence thinking bunch Thank can companies NX sufficient and maybe are So, as for approval. think in how and multiple the great others, such data space you [Ph] about as of then [Ph] you. obviously in LPa? wondering job that cardiometabolic, hyperoxaluria, for a [Salan] you [NX be for gives a I the differentiating differentiation you you And well mentioned Ionis, for Arrowhead, primary did LPa],

Doug Fambrough

PHX, will start to like on that. I take you So,

the single that in subjects PHYOXX range, a six six, is is indeed study placebo will a to that nine, So, up but dose have control.

think will from subjects, see the outcomes, both be to was the of to need to stone lowering oxalate. meaningful So, unrecognized. clinical with show our demonstrate that that our depend nedosiran on as month literature two our it be results urinary disease. discussion are the but will your necessary approval in follow this oxalate an And to with well renal first is FDA from accelerated we that that Uox rates I a as reduction Natural oxalate, a expect endpoint, PHX reduction visits our be approval is we the a we arguments the really serve apart. and recall, is accelerated in study, it convincingly carries combining is are lower a importance that as mechanistically demonstrate effect, the The of source HX years knowing to of evidence goal then around of for that the If the lower able as But that ongoing things that XX% on insufficiency you impact to recognition additional is the any accelerated that discuss, we as based key basis well will previously in still magnitude there emerging on these to History of commitments be of showing the consecutive as will additional nedosiran need that can reductions data risks now work of injury, available primary the plan. renal potential message would upon with we'd urinary longer-term as that PHYOXX potential the

Doug Pagan

been include targets, a to Luca, development on targeting large therapy generate modalities think decision we enjoying have are at sales very targets, obviously I may So, of of years more clinical issues history margins these populations by there the pursue are challenging data with when forces, these the a poly made are rare on the not market mind requires several there been the is larger of differentiation, needs. developments, respect products. that has populations, patients ago special rare were the managed, constantly that seek of those They And disease there not that there the our large are markets our to have probably trials. types on to the decided that But pursue and population undercut with cardiovascular number often other and expensive them a and economics those the we large, fact to and partner advanced own. are to current on long they've because drugs and

the more would strong environment. that So, far successfully it we may Lilly player really years But be that made of ago orders. choice established that those navigating a complex be one several can navigate capable like

left we've really Lilly, off mechanism, respect to transcript the RNAi turns So it with in liver. to

sort there So target if example, really you're is different using RNAi, strong be very prescribed GalNAc silencing, of targeted right, are it scale to is execution benefit. you're that's the where going our I they're for that's mechanistic absence differentiation. not in for huge differentiation, what we of core pharmacodynamics the what going of, the a not bring I that achieving something a it it's level we using competence. a mentioned avenues And as good And table to scale to aren't is -- is think really be as with

job friends Lilly, do our I So, to with. at good a think they're going

Luca Issi

Terrific. you so much. Thank

Operator

you. Thank

your of Stephen comes Willey from line from is question next Our line Stifel. open. the Sir,

Stephen Willey

for the afternoon. Thanks good questions. Yes, taking

know we've of this AUD But a that know and from bit get you development a have program market, a at niche this program, a we're going I to specialty of feel next across the independently commercial webinar And little is Just color, something take on about aspirations? you quick some presumably regulatory bit so the which is I the plan, month. more talked it's kind can you for line perspective? finish a a little question

Doug Pagan

we're think whether a the face. think can a It to particularly I future, physician But old saying difficult, push this question, within choose that's line Stephen. own, have we we'll about not, scale the we that I Yes, certainly predictions future, our good capabilities. finish it's that about where does, to on the across to certainly our what's are or growing

probably into the in I think something here, for growth and the the call that the psychiatric of of will about do points the different that very most and and do see CPU going point one than importantly community. I we can CPU market call on not point including community, be broader own there can beyond call us are XXX% effective our product think that marketing

in I So we collaboration a is the partner But think don't I right for for it require us. development think indication. commercial

So the intend from and to that in, not and generate to going hope just to something maintain rewards that PSC program is invest to we're it's economic core reap we state, we the something that half-life. very strong seeking a we're economic intend and This and it.

Stephen Willey

the are control any And just targets Got CX is so Alexion scenario question, interested to I you'd by it. those that That's kind I back come and change there of drug you, And of guess in? the a is provision one which maybe guess helpful. if impacts there I collaboration? and collaborative guess CFB the be

Doug Pagan

are control might there possibilities what With to definitely back is think confident would to not CFB I'm for happen, targets trigger. see to forward, So, a going what move interesting happen. might very a there because do come respect That these programs of of move change to means, they I us. and that we as But CX are forward. lot

enter the clinic. there that path as figure So we'll at work be I friends in what our with and programs to think it also AZ QX, to out and likely there'll just be at are may but forward proceed and very timelines well change, their any be I the a relationship. is, friends along have both won't conceivable is to after there and change of be the will Alexion conceivable sort some expect there It's that re-jiggering,

Stephen Willey

that the is the on Okay. that maybe deferred just should revenue we about quick, this question, a here top of financial recognition then is in milestones both and deferred combination of And payments milestone some $XXX of that incremental just stupid incremental so you're getting five-quarter revenue the expect or recognized that potentially forward? one over talking period, million last

Doug Fambrough

up, $XXX just going cash the number collaboration million associated the is, just with pick quote we independent over we the in and door, that revenue I'll revenue. [indiscernible], recognition the five-quarter as for account the that's into the so that that but bank period, and accounting cash that number

deferred Doug about is shows that's quoting so on clarify in the And the cash bank, the Does for and revenue, as revenue already that that's line. you? when really up

Stephen Willey

that was indeed sure coming I door. it wanted Yes, to incremental in just make the cash

helpful. very Okay,

Doug Pagan

the tables be how Stephen, just the pure its cash in will partner the hand point, specific help but unfold, it's to deferred to there and order section cash. understand MD&A an in it revenue further by very make that clear,

Stephen Willey

taking the great. for questions. Thanks Okay,

Operator

Thank you.

Our Ed comes Arce from H.C. Sir from next opened. your Company. of the line is question Wainwright & line

Ed Arce

Great. Thanks progress, and to for expand programs. taking on the continuing my collaborative internal all congrats both and questions

years, is approval and effects to lot benefit a clinical of and synergistic positioning? how lung strategy you benefit think asset in could obviously have and could longer as Just been but on benefits your as or even one Thanks. marketing in about the curious move them patients potential just a that liver how patients towards these AXAT collaboration compliment me, term the overall answered, I'm the to these impact your that for how belcesiran, liver for and program the targeted question towards already just effects, of a thinking for this independent few you patients.

Doug Fambrough

Yes.

that. Let to treatment manifestation, compete with to there're pulmonologists along, me standard new to cause, two things a needs it try care. it lung have a care, There is and step by in of and if is really going you but has a come different standard that that's and treated of add. same They if different are There Shree want here. get into they're diseases. diseases the

for Patients group, best disease are one. than the overlap for going only, their there patients is there is that are We have different to but there only. no patient physician populations, with liver are disease, organ, are standard the for Obviously target in it, patients there a and there I want patients these care that liver liver lung. best different and will currently longer a the are become with by care want of treated care lung that's liver but an

is them way because marketing is sure, their synergy I people feel the lung more decline decline. feel they don't are that so, the lung And the same their liver given there between think synergy patients a in parents, for

is that an there for there. amongst patient the liver patients so, are enrichment lung And current

the three there to a marketing So, augmentation there. synergy of is the they're providers, extent in existing already system augmentation potential through

is option perspective, misfolded don't lung? liver their the liver for For you or what's think ask, and to we the -- in option drug really really think exam ability treatment for best. do the to to and the it of what so constant matters from of potential is suppression very to got And really with It protein, think be the care best the has its a not. be RNAi medical standard I going the though best the the liver. best I because what of liver, the is And treatment a treatment give and high much

Ed Arce

Okay, helpful. great. That's

Operator

Thank you.

from is Our Mamtani your comes Mayank B. Sir, Riley open. the next line of from question line Securities.

Mayank Mamtani

partner into pipeline your programs. appreciate see going for Thanks comprehensive questions. the update I'm visibility you taking your to so much and

the to you the busy challenging do any there, or Doug have derive of to different It's it's of you combinations on want starting think on treatment, different ask confidence and between ongoing kind So, encouraging you maybe one, given program, functional Roche that? alternative program the you any betrayed them your there HPV the that can for obviously, comments so obviously have I just off a kind a Doug, include study there landscape. with is number of care if in options, data, to more

Doug Fambrough

often speculate. to Yes, I'm happy

And treating HPV my been functional doesn't there matter, at opinion by been be bit the albeit we doesn't a in the rate know success, There'll course and can agent increase where suppress history look has is it cure, I activity at And that then [indiscernible]. that's company. but And that. that the there of NUC the question a in We achieving to recognize the of all course real low vary right? think so tolerizing if view genotype, crack a in Of HPV, but data, of door, likely as speak. I which to that I stands it the interferon you immunosuppressive activity there's reason the you it? to antigen, that you're widen

think NUC think I interferon most or straightforward so way the to about and it RNAi agent. immunoactive is And other some

are TLRX interferon say it's So I that arm Roche's the would perform. three that triple think or more amongst and that would likely combos, the

and the well-known it great maybe else to has interferon and in earlier it better, is something obviously pipeline. something agonist Now, downside, maybe a would better replace there's TLRX something it be with the a

combination core I core a inhibitors waned think differentiated the shown around has bit activity. haven't in really NUC optimism a as the with inhibitors

put [CPAN] is it a going adding become whole [Ph] see all a So, that RNAi is harder of to better. lot to why to combination sudden to

So we're a thinking likely we it's that the have predilection happens. to and clinical to succeed a we'll but combinations, in experiment immunoactive see what

Mayank Mamtani

very Yes. Thank you, Doug. That was helpful.

going clarify And going XXXX? approval for to X accelerated be two PH approvals. of that initial more having are label was PH the targeting the just to full so, and all Is be kind other goal for X,X,X, are you just to nedosiran, that the

Doug Fambrough

PH PH accelerated the we expectation is on continuing of commitment described, to agreement will and the spending that with where Yes, X reasons is is approval the X on X additional I work be an PH for type phase, I data. think recognized basis are place it for our approval different what so in that what we've do a have than of

Mayank Mamtani

are year Okay. Doug, kind also different in about be sitting strategy year. think to shift And again? their this that bit more going the spend kind larger you diseases way. pipeline not you prevalent here of talking back should trajectory from step Are AUD, from you just you maybe last expect maybe little hear as a question, to about others some thinking programs invested in you like and that about might a And of question, a you've for last of we a in

Doug Fambrough

is at think just disease. fairly was that I message perhaps explicit the I Well, orphan looking not we're important about, - which the most

flowering you of Scientific competence internal to than diverse Officer draws large trying But with to that large the - resources. given diseases. there, growth higher that our would have success. in way company like our like there, but that of that on have qualities our it's approach strong there and the RNAi process of combine by are of a are our internal indications, orphaned it. there Brown, enabled to where Bob And been and has is this that opportunities work. industries biotech as opportunities the our some but as out, in going that constellation are well we there probably by the you led some, And confidence industry you That's analysis at diseases And have ways that RNAi to it's set evaluate go targets features where in a Chief RNAi resources this really some most the looks We somewhat of appropriate. work united target, in have indications there. there's but average various formalized look they're a pretty hey, we're non-orphan. that, some great There're looks market there're to things also probability and some a likely that is look. work is This really when like then, some best by And to

the that's that building how So is, thinking we're portfolio. about

more quarters. threads that out I think you'll beyond future in indications And roll the go AUD coming the see will those programs. We through

thread the see through they you can program. how addition, historical In

Mayank Mamtani

XXXX much excited. it Good been so taking really my has and Thanks question it. Got for like seems luck.

Doug Fambrough

All right.

Operator

from the is your question Sir, Keay Nakae line open. Thank next you. And of comes line our from Chardan.

Keay Nakae

Doug. Thanks, Yes.

is a to looking proxy, in measuring to For something knock be more AUD like hide that you'd down or is specific terms there of ALDHX a fetal of measure? something good

Doug Fambrough

Shree the will in can start, not going I answer to what and were is liver. Well, doing they people think the supplement I here. And be poking

So going ALDHX been knock very reproducible. in we're get I before to an alluded as context has down. But another not to RNAi

good pretty a of as analysis. So And aldehydes marker. makes it bio that challenging achieve basis use base that I we transient it confidence can the for very somewhat we're of to level to firm knock ALDHX, a have down going think

solely to that. ultimately important response be challenge. thing hanging our alcohol most So hat on we And won't here the is

the So to is do most be out? aspect. going that important Shree, want push you to really that

Shreeram Aradhye

happy marker. be marker open a we'll PH looking We would the be at publisher heart facial the rate, will feel. Yes, like to redirection, and flushing,

them all So study interaction marker as primary we'll at look a of RTD.

a think metabolism offset our measures. to have in done to -- But alcohol response will We onset the goal small is primary and of challenge. I with

Keay Nakae

target prevalence And model, about in million, of is initially through a you with a goal but as what $XX some Can us to Okay. of then, hanging we're the subgroup talking this a give trying sense subgroups. opportunity below is what to worth? perhaps

Doug Fambrough

the Yes, flush is of them are receiving intervention. about XXth, and X% form on That's that's There some of much seeking That's is a that treatment. are down, we'll a at the that group the times some pharmaceutical at about given so XX% one the group That's number one. key XX of the of people pharmaceutical XXX,XXX order group but but larger obviously half individuals. who population. size treatment, million currently

they're group has used with we conjunction that that in behavioral potential However, to the amongst commonly DCR-AUD that option therapy believe the become currently getting.

X.X being a fruit low-hanging that already million, don't just treatment. recommended are pharmaceutical option. for X.X million have there, that good people So It

can a parcel even needs now that's to values you start we So think in where a thinking far. the valuable very be see about that there I And product.

Keay Nakae

Okay. That's you. helpful. really Thank

Operator

concludes over to call go That Doug now for I closing would to you. remarks. our ahead. session. like Sir, return the Thank please question-and-answer Fambrough

Doug Fambrough

of continue to us growth. of potentially this two expected got and lot be activities. to the With collaboration going to our two year. of productive and to going start our turn It's I least We're submission NDA were exciting at and a candidates RGXXXX a Phase INDs on you. studies year out ahead on execution filed importantly, highly very to X belcesiran this Thank horizon. be year and

it's through filing go I as think process. the we NDA transformative actually,

again on our Kranzler We towards you for here on all to look towards on got us biopharmaceutical and as the going So all XXth that tonight continue we forward March some we you more Thank March in of stage will for us in a integrated discuss join we detail keeping Penn. commercial to build and a addition lot hope program with conferences you joining Henry have We'll webinar wonderful updated in Dr. U evening. fully company. DCR-AUD. today

Operator

Ladies today's and gentlemen, this for participating. call. Thank you concludes conference

now may disconnect. You

Dicerna Pharmaceuticals (DRNA) 26 Feb 212020 Q4 Earnings call transcript