DRNA - Earnings call transcript Q3 2020 | Dicerna Pharmaceuticals Inc

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Operator

Good afternoon, ladies and gentlemen, and welcome to the Dicerna Pharmaceuticals Third Quarter 2020 Earnings Conference Call. A reminder, this conference is being recorded at the company's request. I will now turn the call over to Lauren Stival, Dicerna's Head of Investor Relations. Please go ahead.

Lauren Stival

Thank you, operator. thank XXXX joining highlights. financial and Dicerna's to everyone, quarter third results afternoon, you us for review Good and operational

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

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

Speaking our Chief on on Doug Pagan, be update then who will Financial Officer, Chief Dicerna's President review Doug Fambrough, Aradhye, and Officer, financial our results. an today's call and clinical our Chief development who corporate milestones; Shreeram will quarter provide Executive will who third key and Officer, discuss collaboration Medical will our and progress activities;

of the be we like management today's potential remarks, related may We and future open to and to our will example, RGXXXX of including, additional commercial for statements potential data lines, and make plans candidates, Dicerna Actual to by important nedosiran section to at statements for current funding, these expectations factors, Dicerna's Factors our and internal views those And the the the potential differ the for clinical listeners, of from CEO. for We may in tissues and collaboration Doug? results also the collaboration. change. position, collaborative in our and SEC. expenses to statements continue some press development as time expectations specifically our remind elect now result Dicerna's future. I'd or discussed to development, obligation including various to our XX-K or indicated Fambrough, disclaim including our forward-looking research clinical forward-looking regarding financial add Following Form filed We current for like programs, programs, the extend development lines and with usage. to and if Risk about collaboration, programs the forward-looking in therapeutic making today's a reach on XX-Q Doug may any noted those Dicerna's or technology President research the turn point may up of product to materially I'd and your call, our as cash latest questions. that these time update in statements and forward-looking so release, over call future lines discovery do

Doug Fambrough

stakeholders. built that select achieved, for expect for to proof-of-concept the have primary candidate nonclinical in success in our once been opt positive to is and data a us Lauren, beyond high-value Dicerna thank of delivering categories early partnerships to we'll opportunities in here a joining our multiple we allows everyone, models has way company higher-risk well in continue pursue RNAi with confidence value, economic build thereby best-in-class to as in world-class to and strategy strategy that Roche, our for to even and research partners. selectively hyperoxaluria and programs has liver. particularly has additional with Dicerna tissues and approaches development to drug targets, genetic Thank us a the B as therapeutics PH our or quarter virus potential provide Novo us. validation these resulted RNAi those an with we a and In partner upside. other with company This Nordisk maintaining have number and such disease for where be programs space in a to you, hepatitis in It These value pharma you, allows potential the in that clinical readouts been data multiple the scientific and incredible as Dicerna strong our development at instances, clinical thoughtful for structured

internal Pharmaceuticals. for aided be both forward, extensive have four and from move advancing five to the continued a hires have team we're commercial our invaluable development alum, that organization new They as us the phase from in organizations Chief now Officer. joins with the Officer Executive of Aradhye Medical of development clinical major Over three Chief of balance We the will M&A has and expected as to Ling of programs sheet fit-to-purpose our served Affairs Legal the next we transformation, by most also in Novartis' Shree we from clinical on in development who Chief experience is quarter and are Global having Chief company. made and quarter, driving for is fourth as the where them she year. upfront leadership -- which commercial toward been to board. Head to quarters, Novartis, this as recently recent in Zeng Medical just enter During major bring through scale thrilled President collaborations. the Shreeram company to our executive appointments a as candidates Group; Vice Legal Officer Medical collaboration $XXX and been a manner key growth We've us quarter last million undergoing clinic, our Deputy the that payments Novartis and of first of of adding preparing our Secretary, Head served as

the all year-end, primary building also of pivotal are our potential in nedosiran have we subtypes the trial enrollment the in well treatment as expected the for And the organization in medical to known by market down U.S. and commercial to our a are best-in-class profile nedosiran, hyperoxaluria. approaching path conclusion which of has for We three first

However, our vision accomplishments. the for well Dicerna goes next five years these over beyond

revenue income players. our as that with that in with five a company from the products market creating stream top-tier antitrypsin Over growing supplemented commercial a alpha-one liver biopharma these disease, emerge by PH we in integrated United years, collaborations to fully two expect we is biopharmaceutical States and generated

to I system tissue, pipelines, end numerous by over five fat $XXX the we Indeed, of continued $XXX within our provide our and other in to support simultaneously strong working that these and we existing to addition our for on million XXXX early to across the programs come. quarter, lXC internal in ability I'm well array five muscle stage truly least our technology sheet years, our million recent disclosed. and the an clinical am we tissues Shree? to clinical progress cash late-stage to Dicerna. supported years liver both haven't commercial detail. excited and over Chief happy I'm are and lies of to programs financial at over clinical to call confident our And the of turn further liver-directed more expect and execute third on Medical as expect Officer, to programs, between as new drawn at collaborations with and now aspirations. RNAi in technology ahead the nervous that, been to from for our With core balance the Shreeram end on discuss In delivering what of Aradhye, these fund yet collaborative operations proceeds

Shreeram Aradhye

to At stage and biopharmaceutical you, am with integrated colleagues work all this become and help career, commercial a my join Dicerna clinical progress portfolio Doug, fully for Dicerna my us. Thank company. excited to point joining and thank its you I in

to year. first of the trial I'm the the a next third looking completion As nephrologist, NDA the potential for and submission forward pivotal in nedosiran quarter

While so this itself is to best-in-class me. of exciting, is even Dicerna's potential more broad the RNAi technology

was August October, the join of were to I nedosiran me. after B recently, validation in virus. R&D partners and updated made special our an company data of major September, extension I multiple hepatitis with by major PH Dicerna, the therapeutic trial my areas two open-label choice diverse joined PH. these Day presented As ASM More from participants in we RNAi to at cut noting technology continued for when sets interest our in soon for presented improvement our PHYOXX interim in across data

dual This hyperoxaluria damage. was excretions may visits primary three and patients than involves XX Across PHX see equal at the disease, and requiring XX and PHX, more chronic day of that in pleased transplantation. not a outcome which dialysis systemic hours. damage. per did Uox PH and participant or in X.XX oxalate reminder, progress at through desired for measure is subtypes, rare less cases, and time or X.XX near to had receiving normal In of buildup participants and with the end-stage a retina. overproduction We more is included meter result causing known disorders need one more PHX monthly with excretion time than PHX more progressive to one efficacy hours, level. than who X.XX normal did defined patients PHX, for of the kidney reach a decreased to one was also family life-threatening the the normal or defined study regular squares achieve to a lead to can PHYOXX achieved nedosiran, of or XX in as and normalization less analysis can renal kidney XXX oxalate PH. per for Uox oxalosis kidney the experience hepatic liver-kidney the XXX millimoles As while All points. squares advanced participants three excretion or deposition Normal oxalate excess in X.X as oxalate while genetic skin, renal PHX near-normal achieved day interim stones were that meter may reached X.XX heart Uox millimoles function normalization protocol point, near of the key that with the XX XX bones, blood, urinary

intake. range XXX, Uox people one PHX participant no their improve serious is urinary order three and normal reducing to to on load. durable generally hyper excretion tolerated in well visits seven normalized had are of day ongoing course and and with study. life. quality hydrate safety was to These a in consecutive potentially in least meeting oxalate fluid manage their to PH, at secretion concerns people this or eligibility reduction protocol-defined For daily criteria identified critical for required change disease Notably, to oxalate Nedosiran were PHX near-normal

to year in have by forward PHX, currently double-blind, pivotal placebo-controlled the in alignment year. trial a QX plan We to are these our and next having in for to half top submit midyear. of look an sometime PHX of for which and PHX sometime completing first the We path FDA line approval participants we encouraged with results on and full data next PHX NDA and

approval We around PHX, of are is least the most recently the most path understood the identified, and which in the underdiagnosed for perhaps to subtypes. dialogue the PH

study OBX we're PHX with disease history conducting PHYOX and such progression to natural formation our as As stone people for establish outcomes such, rate.

PHX patients this quarter. dosing begin to in our plan PHYOXX also We study

As in additional the such, when NDA year. studies seek renal we we file in initiate parallel, year. initially of we approval near accelerated to we supportive our one our that or of term next nedosiran have for started quarter starting PHX in plan programs, study next anticipate PHYOX including the In will

B study to groups; monthly of who C our ongoing doses participants are is is four and hepatitis announced participants at Roche. extended kilo kilogram. of to RGXXXX; in log NUC three positive from A, Turning C therapy milligrams Group eligible reduction X.X, to B dose continue RGXXXX than milligram randomized which concurrently diagnosed naive or achieved I day antiviral greater of surface of per virus hepatitis Group and X.X with August. our antiviral RGXXXX per healthy three were The an of in the received follow-up chronic therapy, Group with X.X results newly a XXX Phase our three a receive hepatitis to participants single and volunteers; adults period completed participants comprised in program, B, observation period. Group NUC of with interim injections trial that partnered We one in into receiving treatment one B antigen

available hepatitis participant X.XX the treated the the mean cohort. Notably, was reached have antigen maximal the measured logs surface X.X the achieved completed participants four to As log than extended antigen group kilogram of XXX three participated milligrams milligram cohort. in the pleased We're reduction A X.XX three included of B and XXX with in the This hepatitis the in as per log period. a day functional and from of Among viral cures emphasize logs the and the B Group surface a baseline XXX. milligram completed I'd equal analysis was of in circulating data who X.X, at data like and participants cutoff seen reductions will six sustained six two Group surface the antigen achieving reduction B the per development C and in N antigen we reduction. kilo from period, surface participant durable therapeutic believe patients. time across nine day entered Day a X.X robust of see kilogram S antigen in of maximum HBV per one interim per greater group of hepatitis cohorts. milligrams study finite leads of duration reduction two data regimen had kilo loss cohort in antigen day longest XXX that DNA deep this not to C June surface who the sustained C participants response levels C because yet in B reached XX, to follow-up B required hepatitis hepatitis to treatment

therapy surface immune-mediated that hepatitis during in long HBV. for Our duration reductions potential elevations of and function B, predicted a with receiving ALT interim X Group patients of sparing preclinical prespecified in trial reduction confirm study of suggestive antigen of RGXXXX, B dual with from by virus' treatment kilogram and treatment-induced naive These as deliver benefits predictions with siRNA sustained and chronic experienced are gene. transient a flares data marker models of three three-milligram liver early I stable and our In in six the clinical to knockdown participants strong per the dose hepatitis a These protocol. NUC after period. to B preserved hepatitis surface concomitant produce or HBV beneficial Phase could activity the our robust we B viral antigen RGXXXX's responses one

in we meeting, to Phase We Roche will forward present cure to combination trials goal with AASLD and upcoming functional patients. B with initiating additional follow-up hepatitis data a achieve the at the RGXXXX look chronic II

internal the our as Doug XX well partnerships. our mentioned As advancement our through call, be will outset pipeline marked through of of next progress own Dicerna at by the months for as

plan in for work we II. as but Our liver of quarter, AXAT to disease in to to advance continues select to expected, Phase programs two one progress AXAT the first our

first our large to the target wholly market development We plan a in owned which plan also during announce to next the planned to patient sometime We clinical half out program in XXXX, our last with anticipate in culminating for data indication, sometime PHYOXX liver enter year. next midyear midyear, we of nedosiran pivotal quarter. and trial the third in follow submission the of NDA

I'd We our Doug? to the to milestones are looking turn like forward to over financials. these cover important to call in Doug Pagan, year ahead. CFO, the With that, the

Doug Pagan

Thank you, Shree.

Now I'd third the through results quarter. for walk financial the like key to

$XX.X expense, as XXXX. associated quarter nedosiran attributable in quarter loss in collaboration million expenses was per the The support quarterly period quarter million our details, quarter. or to issued our AXAT including and to both and XXXX outlining XXXX, net share $XX operations. was increases necessary for our with third $XX.X to XX, million and million third please in to XXXX. were the $XX.X $XX.X was report additional due salaries, for $X.X For on which same costs for primarily our administrative research increases organization expenses, Net during million to was or well and partially change period headcount was million study increase for R&D million and of to compensation stock-based press R&D a the the employee-related results of XX-Q, $XX.X compared third by $XX.X increases to collaboration $X.XX increase The programs, compared loss in quarter and release and Form XXXX. programs. related offset decrease expenses to to driven a headcount financial contract associated our revenue today. and the change September employee-related primarily the trials to earlier general in these refer per growing The by in driven and million a expenses were in for the $X.XX research G&A clinical ended for support increase in throughout the with XXXX same benefits share the period-over-period manufacturing development $XX.X as programs compared by higher million core related costs

to R&D quarters the to spend in as We expenses additional in operating we increased to efforts grow and expect coming overall readiness continue external increase accommodate as pipeline. well as higher headcount associated with launch our from activities

in recognized Roche and to recognized well compared other the as and the for in includes million each year as activities is quarter additions million some the progress of measure $XX.X services XXXX, the on agreements will words, During collaboration upfront of equity The premia In of and than a we prior the Most of complete period. contingent on are the individual cases, collaborations. provided payments percentage straight-line and $X as be using increased work to Novo the method. ongoing which primarily revenue a certain rather for price, third Alexion based change payments, partnership of basis. transaction attributable cost-to-cost collaboration milestone revenue was Lilly the in recognized

As of next approximately recognize current $XXX we September $XXX following several over million quarters the years. of XXXX, expect that XX, to revenue expected the million we and recognized over be to deferred four approximately had

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

milestone the for milestones from both our assumes our includes operating events and end payments changes be other collaboration With source external or and these that, Included I'd the pipeline current development, plan and are current continue estimate an expenses. equivalents collaborations other of programs to existing existing execute nedosiran This launch no We and or commercial financing through regulatory activities filing R&D payments anticipated mature. runway of XXXX, questions. support current cash, like will $XXX internal. our represent they from to generate and the through continue current the which more should in collaboration collaboration all our funding important expectations XXXX. for pivotal value and collaboration our to to advance programs, from no new into significant and for believe proceeds in and held-to-maturity from achieved investments These that in cash demonstrate to arrangements open arrangements receipts call payments expected to sufficient than cash as unanticipated million costs and

Operator

We Umer line question have Evercore. first of Raffat Instructions] from our from the [Operator

is Miller Evercore Jonathan line in ISI. Next from

is line Your now open.

Jonathan Miller

of -- interesting the to that HBS at your I'd HBV level like to data. looks the the as regimen day the day, the as R&D higher as is even you like something start antigen the AASLD R&D have dosing in day, -- you I observation you two that in remarks but antigen prepared same and a after data? It reduction, guys other from dosing has of they duration R&D remarks. implied R&D the updated you have in with the was day will get AASLD mg patients that on thought overall you're cohort cessation, track to implied there? the they the final kg six that prepared average abstract long HBS data be And The in the well. effect finished continued final Have going per in lowering mentioned the they

eventual the a commercial insofar when time-limited that a long limited to long in frequency of dosing dosing is regimen I relevant drag how be is isn't length guess relevant -- uptake, likely duration isn't what practice, the clear? setting with is of dosing I mean a tail tail? how chronic of for as in expectation Given that

DougFambrough

Thank you, John.

day the per that the late-breaker point, we participants this all have Group will AASLD. presentation including presentation patients milligram anticipate six at data for for through two kilogram our So C, at at additional we XXX in oral do

cure I going I period, of this talk respect have and the to before important regimen. But really correlate think effective functional longer an to You're to with with point a percentage we long that data. treatment is likely wait for the to achievement the think the because duration, presentation that's about

extend with collaborators to cure who And so an the anything Roche. of is to likely percentage combo regimen when correlative have effect effective beneficial that our that at can period be done functional we on achieve patients identify

these the that animal So and of there's at maximization first We'll translation over we to over was suggest certainly maximize to that that in indications the that expiring approach the achieves have time. our of models. something in duration activity sought patients. And least see

Jonathan Miller

you'll the quarter choose taking differentiation for other? Great. are the your And first then expectations next your sources versus the for What's one And candidates? year. AXAT likely on what program, two program you say molecule between forward bar the of which

Doug Fambrough

and make Sure. very I so mean clinical good we a our colleagues at do Alnylam. candidate, know how to

no the only I sense so makes And believe that one take choice it to molecules, between bad forward. there's here two but

we be molecule healthy the that programs details as comparing nonhuman primates. the from we're molecule in collected human So well as and our Alnylam will data and collecting nonclinical volunteer both rodents from the from

a, options. at a very for you're part it details think choose level a good -- not from But combine large is macro programs John. particularly and need that We differentiation Thanks, in the the to is and of collaboration one of one, but efficiency, is I forward. to take looking it two that's the magnitude. at

Operator

Leerink. from SVB Foroohar is Mani Next

Your line open. is now

Mani Foroohar

a from us little exactly sense depth a question, a volunteers limit? up, that health think One one it Would chose. knockdown, the and presumably financial just question asset about I assets two AXAT. what you taking follow Would get I NHP question. you. follow to Hi misheard pick have quick and be lockdown, the for may And what could the it more guys, data of thanks a When be depth from of then guess of I up metrics move we earlier information just potentially of the forward, on give of on expect? you of we bit

was million assets that's number based during that from entering has little collaborations, of that of see about $XXX on between clinic, to accurate trying is data I hitting still something end cash that Just that and our error mentioned clinical to thought risk, milestones confirming XXXX that, now the you million? clinical early anticipate etc. bars is in the You you existing $XXX around incremental it of number. the of and that get bit some that need or to a trigger door. some

Doug Fambrough

the make human sharing Okay, data choice, be healthy of deeply point. go at from have the we'll we to I that to a Mani. the would clinical On duration molecule. how volunteer It's data the Doug, AXAT, with we the $XXX out that I associated I depth breaking think choose, you molecule depth take we'll And into and respect the duration, we preclinical and don't choice. think With be that. doubt we'll the like million, after made will that?

Doug Pagan

Thanks. Sure.

$XXX the from contingent milestones that as through research and combination are are is starts reimbursements. -- milestones the that for payments, are into upfront different So early of clinical a million as who proceeding well

we of So we'll the update the as number proceed that about And quarters. $XXX putting through confident out. million, we course, feel

Doug Fambrough

I help of a we lot on what call. That's analysts Great. the have our

the back hop queue. I'll So in

Operator

Next Nochomovitz Citigroup. from Yigal is

Your line is now open.

Yigal Nochomovitz

Great, agency thank when and accelerated approval. much. therefore, Thanks the you the to plan for with for just satisfy you underdiagnosed it's with for many Doug, rather Could for part several of patients to be that to going PHX how has also own respect clarify, need made And FDA is communicated comments respect the in you to order to going to opportunity interesting you PHX for what Or population getting it you is filing? an then you to taking its PHX? that? questions. PHX and the the that you be approval an NDA seek accelerated PHX also, file very PHX,

Shreeram Aradhye

Shree. is this Yigal,

have PHX, think we approval PHX launch history reductions for natural only urinary because basis I Because these effort on have and PHX. the to on marker PHX same we get serve the is study history PHX. experience where make which of PHX will has this demonstrate with natural types that complete we described the that decade oxalate point, we clarity our recently burden at have the understood to of a in about been had perhaps ago, other PHX and and that well know that people that will as of less the patients to agreement we

In describing with treatments. to OHF fact, if into kidney have could a their and recurrent you significant where the to how experience they patient was wondering called PHX meeting get happen stones access

demonstrating disease combined So lowers in of show. PHX, for combination action, on mechanism we a the urinary that us, that ability with demonstrate which we're to nedosiran oxalate it's can of our burden confident the levels based our of

submissions. that. launching that are We a basis the it's that combination -- the dedicated of PHYOXX will will of information to be study demonstrating And our

it I determined. approval discussion. file that that required expect it think what that core be is form we're an the to the details of be be beyond of the we accelerated under are still But suffice because to we to guiding say that information of will expect in additional what's

Yigal Nochomovitz

Okay. as types? those some or this CNS, tissue earlier-stage programs you might the you've far in to your outlined -- be muscle details skeletal can and pursuing that more of with any three point, as you provide diseases at respect Got And it. for other what disorders that adipose,

DougFambrough

to going note would delivery, competitive are close Lilly, that and pretty our we thus our to our us certain neurodegeneration in and we CNS independently where But I chests, of nature Yigal, field. cards that given the indications. orphan are provides collaboration pain advance play with disease deal collaborating to on the

say space. our, that program, to be disease orphan expect I'd the first neuro would so And wholly in I for owned our

Yigal Nochomovitz

as just $XXX of to five the as again and the collaborations next from going just be Okay. on color question, any that's or that how four distributed final And end XXXX. now between existing million one provide then the over far Can quarters? you

Doug Fambrough

to up So at bunch though, guidance this we say is distribution give of numbers. made quarterly smaller point. of really haven't about it been a I'd planning this, the

one it's So not payment. as it's if

Operator

Cowen. Werber Yaron is from Next

Your line is now open.

Brendan

for This on Congrats guys, Brendan Yaron. the update. on is Hi

Just a here. There's from just perspective your been ones I wanted quick couple and us. already. get of kind talk it to direct of some questions about

moves about, thinking talking far dosing and that drug of, it -- for of of use of the thinking wanted the kind have patients Lilly kind months? administration program of based you're kind about maybe next Do this next or about us versus what think how data-wise? us infected your here tell what in an couple into Looking shaking duration, you're kind perspective. apart J&J you drug. the out we that's our uptake positions key really as the from that just about so drug check you sets in I'm how in drugs of And for into? What kind drill HBV over efficacy say, to And to year partner then I and among the can about on clinic is

Doug Fambrough

with our that, HBV humans factor. program, action think is so an do of duration potential in to differentiating for respect Brendan, we and increased the important ultimately,

and and action we, different the X, to agents, else determining think, even field a in the different likely agents space. pretty even losers activity, mechanisms of respect perhaps of therapy combinations are and winners in anticipates However, of a everyone combination much I provide the with differentiation of maybe with different four lot to

what itself is marginal much how most emphasis is I've forgotten and on generated. rates positive, are this successful with by say I'd or which we'll duration I But and Brendan. If So case, partners at that in don't but functional see would alone, the second to interact effect. the I your one RNAi I going wouldn't of the RNAi the to And question, powerful. NUCs combination think, cures was not. allow really have the put to think call too it it It's be a

Brendan

It program, was the year. later just this the IND about Lilly

Doug Fambrough

space. molecule. it collaborating Not the in And with is lot say liver-targeted a cardiometabolic molecules, I a than can other GalXC those GalXC we're

from molecule it a area. in to that, the that therapeutic So gene targets is

Operator

Stifel. Next is Stephen Willey from

Your line open. now is

Stephen Willey

and afternoon for taking the questions. good Yeah, thanks

patients? of, bigger not to of trials just kind for evaluated So, disease. as out, maybe within there improvement pulmonary kind I potential question seems exacerbate be that silencers, to lung the and AXAT. this interactions of had extent, silencers, you've what markers on during, support of lung markers a space, with of I function right? if RNA on guess, can agency that later-stage I perspective guess, that to might kind of to any, condition AXAT there's believes potentially clinical picture that the know rule I some like the great -- to of curious regulatory these be And Because it their the kind guess, FDA of just some

just the I'm agency wondering there's if that markers of well. any So there's that exacerbation of disease believes kind show as pulmonary to no

Doug Fambrough

causes start Shree the problem, if has see we over add in smoking. patients, AXAT in at unusual fraction particularly a those a lifetime, a we're little the as mean silencing course lung by present that the such is of know irritation, and not there. to when Let I a chronic least target something to of me lung exposed

to issue already is certainly that, point by view for general on. treatment is itself respects a at KOL And FDA life the to unlikely salient pretty cause the a disease cost that there a for in unlikely lung so a late in probably one. the it have problem, is likely get ratio and to to and is liver said have FDA a positive cause Having comfort it field, that benefit

proceeding And they've space. are let other and so programs in course, we of this proceed

DLCO, on we to We of, think be would Anything of needed Shree? you markers the late-stage and standard finalized on yet a more protocol. to have are that, FEV, not evaluating some add

Shreeram Aradhye

therapy an sure the the what duration that's but and important for certainly have a this me, of will be that manner as in inform have program. development to balance a No, might that and I population. protocol will right picks think design answer ask of making available what we that people targeting disease the be, then a certain clear the makes of is it's who the liver It the monitoring to And exciting. be question it'll us focus of likely tools using you to severity

Stephen Willey

a guess lower in of agency to Is given and a disease, at that severity that think it's some the way is patients severity I an to then at disease benefit that then it all maybe level demographic. it? applicable patient perhaps the of is liver older to risk-reward -- safe maybe only the about to assume believes fair of applicable

Shreeram Aradhye

finalized precisely least get making that us. of that of the I at area about precise. is that for like you Phase conversations only that discussion, looks this II say think a would context actual what feedback the protocol, would too the in clear will balance I to fact the be the

I commitment saying a able you be I an precise the pause So area, it guidance identified won't you've would give FDA agreement with important that on but to or specific it on yet.

Stephen Willey

the Understood. you. I Thank color. appreciate

Operator

Luca from RBC is question Bank. Next of Capital line Issi the from Royal

open. Your line now is

Luca Issi

setback And on if Arrowhead for update for can U.S.? on question think. two, on my and maybe on for strategy One own the and partnership does? of the it ones Two how changes your there if if for PH potential AXAT, your Congrats the you you us Thank I progress. recent program, quick can comment Well success concurrent any for give me. taking ex almost Vertex then maybe you

Doug Fambrough

in at far. data. let and very what expect. small the forward encouraging, It's along looks of so presented entirely we is set, data I look we it Luca, It additional I'll to know say think data with lines is a AASLD. me data AXAT excellent so that start what their also Arrowhead's say we and

expect mechanism an treatment We liver. be this effective to of for the

in XXX it's it I course, issues is a a with across to protein, so action somehow II seeing many And which profile acting in population. to going speak would which, that not out, Certainly, that seen it's positive associated believe of that to the of of much concerning. we're ruled that the source are that was risk us small Phase surprising program, signal tox about the a it's movements misfolded of move obviously The to Vertex the known due programs, that not forward. mechanism So whole cannot class. marker liver the be of that don't globules means corrector on

don't -- we to is effective quite it just liver We'll either will AXAT. to of one a a associated pans there's how have data protein our think I and what or which correctors. the believe So focus the a changes accumulation risk subsequent the see associated and very address profile think has thereby be the out. providing misfolded changed certainly with with strategy, really on safe address I outlook disease way to

Luca Issi

have do ex maybe Terrific. U.S. potential PH, update And partnership or? maybe any for on the you

Doug Fambrough

Luca, wish could update Yes. I an I you there. give

is active something. going to in And be announcement. happens, discussions, are We we've make I our announcing when that that signed and we'll that update think

Operator

Next Co. is from H.C. the line of from & Ed Arce Wainwright

now open. Your is line

Ed Arce

Great, questions. thanks my for taking

So program. first your PH one on

that on As last other one noted more your or noted in near-normal. achieved and visits. one the interim the was in you data month, normal PHYOXX the of out XX XX trial And from

at one end wondering speak I'm or say, might relevant achieved relative So -- to, how conclusion the other levels points to then And multiple have to if the guess you of is visit? and Doug be can is Shreeram, related this patients I the those proportion more or study? than either of what for at question those that the

Shreeram Aradhye

is excited points. at is our with that the this formation ASN, how oxalate necessarily range have is in So multiple is think we on pivotal to specified to presented and let the a first, stone treatment reduction the where indicating means the which oxalate it that me disease being integrated a it, achieved way end then note out a designed, a look normally had at the manner normal look eight subjects their about the while of we in hyperoxaluria to are a if a an primary time for consecutive the the risk of which of brought we think considered start XX the that relevant And set I is on normal at into data excretion. be that exposure were the sort at to point of end should made time or eligible to which achieved of plans not three at protocol their reducing kind that that focusing single kidney, you change at actually period, as have the points time hyperhydration they near-normal sustained. you durable patients And levels of at supporting analysis

looking So pivotal information at beyond this -- we in in look those results were very at to data see day way we a data trial looks to the XX open-label from this PHYOXX, follow-up from and encouraged forward XXX. the in that long to term

So that's observation, an important Ed.

Doug Fambrough

Yes. get to disease that. continuous of your the associated life there's and either cross it management with a oxalate Ed, have from management. to off disruption to only line achieving normalization do you to white wean great that. by but The And or and I sustained have to it's continue you a importance you black get that you disease stress is want the level variable,

in level We XX our of date PHXs patients a this step-down the allow the just in as together oxalate with achieved of have open-label indicating of And initiation trial an in PHX average range. taken three that protocol-defined a consecutive seven in all was hyperhydration. normal to measurements fashion Shree of

And a our criteria in field. big whether So regimen. it's knockdown I and is, patient really in it that pivotal you're it's very enrollment what or bottom it's deal what same And and I the same since that says see think level, you dosing at is it space. and this the a and near-normal is likely am data our whether encouraged by population, data to at normal the to same this at matters sustained normal each get that marginal lot measurement, whether you

out, treatment it So for point this of it's dynamics a population of thinking patient that what -- is this bringing key be. really because is to thanks in about the going

Ed Arce

Right.

again, treatment Noticed how your Then the that. meaningful then that announced on they -- achieve did for core in Or I of study don't and have just view had open Or you know, more of in that get general if But Okay. I'm sort particular certain not your way? HBV, thoughts out. player Great. if finite that sort space wondering and it does, wondering, that it to the any time were one if to -- in And just your to I guess came thoughts that, announced inhibitors? of just paradigm on that. see you impacts that today patients SBR and thoughts. for discontinued time in where both. the given today there a is therapy another a hep to question their this B

Doug Fambrough

of results the Ed, HPV an I'm results. probably watch I sorry, So at looking election instead trial

of think you're RNAi agent So to likely is need that approach the required of the the have in a confidence a that that action going going an you're combination dramatic We degree way. it's a mechanism into that right specifically And brings to antigen now, in I only combination believe, combination. specific talking I'm not familiar with a high study addresses about. is, that and S that pretty be that

mechanisms not different be So about very it. that I'm immunoactive have combinations really I the with particularly elements. think particularly explored, in combinations sanguine I'm optimistic an involving should don't that element RNAi And involved, about those

extent the it's outset. that have been this that trial think I success the fairly without element, a may probability of a may at to been have So low

Ed Arce

Okay. That's one helpful. if Then just last may. I question,

us remarks, was I in if there indication, the large You had not that. if I'm said remind think your you Shree, some announcement details of a this program. could sure mentioned just all who in prepared the new I mid-XXXX, got

Doug Fambrough

into approximately target molecule That population, liver-directed will prevalent We Dicerna molecule. It GalXY be is midyear. very owned a a bringing indication. large wholly clinic validated against a genetically is the

is. it goes into we'll it announce before the clinic, sometime And what

Operator

from Next Kumar Baird. Madhu of from line the is question

now Your open. line is

Rob

This is Thanks taking do. Congrats progress. me on Rob the for question. for my on

And the disease? also initial that First beyond treatment against PHYOXX? levels, for stone you to AXAT on And what liver duration impacts thoughts potential assess plan how over urinary on programs, in and time end influence points? you does the learned do for competitor off, specifically data oxalate clinical RNAi from kidney of have PHYOXX AXAT of formation trial in your and

Shreeram Aradhye

doing the will that follow-up we longer-term event in I think stone continue the are the including trials -- that PHYOXX. rates, we will in indeed, evaluate

element follow time. estimated clearly changes we GFR as follow to that's over an important will So

will we that do. something clearly that's So

are was concerned, four is a what meaningful data as but think are reduction mechanism not for which surprising as said, were the see Doug how that it's AXAT to simply I to of in as think encouraging any action, meaningful that expected, programs us, in that of in far II impact Phase point be in setting. covered the our the that designs, controlled I subjects very inform I'll terms in well on in But as our AXAT in on some additional all to seeing encouraging need forward data. we that the function to fibrosis in relatively the reported at say this as seen a Pro-CX, levels was serum changes related was time, the at Phase changes or thing of FibroScan at is in will that least these elements will liver studies see early It I weeks related about -- directional it Phase an with encouraging to look to concordant markers biomarkers their the like liver this to still evolution. XX design think II was But see. under II

Rob

you. thank All right,

Operator

Next from Financial. line is Riley of the from Mamtani B. Mayank

line Your open. is now

Wayne

Mayank. Hi, This on the thanks is Congrats for Wayne And progress. taking for question. our

So the we studies. one have a filed just on quick

on see of market you? given you open-label there competitive XX the PHYOXX colors any to terms to months how positive just guys the competitor ahead guys provide how do the derisking XX can more entering in additional you on dose landscape, data you PHX want be if the will ASN, a new we of see provided this a Given multi-dose study? can And

Shreeram Aradhye

out as fact XXX. data expect the we And us, see all the levels to near-normal for not and the for what talked normal presented are about we a that encouraging our very data beyond indicate I we we is harbinger that why reported we undurable aligned plan at are day essentially, subject which then study, robust expect in levels important is see quite can very through pivotal pivotal have Yes. what XX study. with think very subjects effects, and analysis looking we I of our of study. this and pivotal mean to achieved our We in XX might way XX the the that one

excited to very to they study. I'm what So see mean and Phase our data pivotal II those

competition of have... perspective you the from think that I

Doug Fambrough

most for into very the reduction it patients. And parameter, oxalate Sure. oxalate saw normal that, similar the PHYOXX the more a point across that would believe, zone. expectation, and competitive the reduction would us the range average result oxalate the set, in that give be in PHX I profile should be a and important to was level in our We on patients normal the

So very that nedosiran a competitive in our we're product very will optimistic be PHX.

We dose have decided to drug differently. the

it true. launch have have that basis. is product was formulated for the if in schedule up rebound, haven't question, dosing or today, in it there a on the to -- dose syringe not the most is discussed was We knockdown. as very like self-administration missed and prefilled gives that, come the robust hasn't which a this with monthly we or would also that but a any This a robust It effective not something that problem sense

fully experience to subcutaneous They If allows small you the over they easy-to-remember in a monthly PH have you patient go once And of miss interact to a rebound see physician. period. also expect have normalization dose, provider, we schedule. a month, of some and with injection, healthcare a think to autonomous, six-month quarterly life don't a intuitive, the context to normalizing and the the the them maximum on should very

of kind grow PHX market all activity. diagnosis competitive anything is continue will think and be a we not to a all, has I So many that will the market in a that believe And years. this of that the PHX very that there which like compelling product with market increased over we aware course, in for of profile are describes

for altogether, this elements with opportunity think is commercial a So compelling those I us. very

Operator

No Instructions] please questions, further [Operator continue.

Doug Fambrough

Well, operator. thank you,

over the what are nedosiran. for expect months next at and next year, to XX we We really have beyond. an store in submitted time NDA this about is By excited Dicerna

tissue expect trial Over new disease types Thank a a and to five-year I partnered sheet large joining development. evening. to AXAT plan our have have a programs also myriad move confidence. strong indication. clinical with and to GalXY development in an pipeline expand in candidate us, move liver balance core Phase II active forward a have you of across We in market ongoing we a and for GalXC for an place, into anticipate multiple a we Phase horizon, With internal good all

Operator

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

now You disconnect. may

Dicerna Pharmaceuticals (DRNA) 7 Nov 202020 Q3 Earnings call transcript