DRNA - Earnings call transcript Q2 2020 | Dicerna Pharmaceuticals Inc

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Operator

Good morning, everyone and welcome to Dicerna 2020 Virtual R&D Day.

As you know we also issued a press release this morning detailing our Financial Results for the Second Quarter ended June 30 2020, which can be found under the Investors and Media tab on our website at dicerna.com.

During this morning's presentation, we will be reviewing data results from our ongoing studies of RG6346 in collaborative development with Roche for hepatitis B, and nedosiran for the treatment of primary hyperoxaluria.

We are also excited to share with you the initial preclinical data from our RNAi technology and extrahepatic tissue including the CNS, muscle and adipose tissues. I'd like to remind listeners as noted in today's presentation that management may be making forward-looking statements on today's call, including, for example, the clinical development, therapeutic or potential of nedosiran or RG6346 and the development programs, research and development plans and timelines, the potential for Dicerna to continue to add programs and extend the reach of our technology to additional tissues in our internal discovery research and in collaborative programs. collaborations Alnylam Nordisk, future and our Boehringer Lilly, collaborations. to and Ingelheim the related with Roche, Novo Alexion, Expectations potential for about expenses or We financial usage. data statements forward-looking cash timelines current expectations and funding, may clinical or collaboration regarding for future make position, Dicerna's with as those of may results filed discussed and risk and section statements in latest from of Forms various XX-K by these those the SEC. Actual result indicated important Dicerna's XX-Q materially forward-looking differ a the factors, including factors We may these update forward-looking statements on elect to the future. to We any so disclaim change. specifically views obligation do if our I'd moment to to to our introduce like you take a today. speakers for now

Chief us both and Fambrough, President our review and targeting introduce novel Scientific have Chief Executive Brown, our S-antigen HBV vision Dicerna's who will Officer, who with our approach Bob discuss will hepatic Officer, Doug in corporate We and data. and strategy;

Our guest X Dr. RGXXXX. Phase today, for who our present Yuen, will speaker for data

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our Additionally, approval related will to we and Chief the launch Rob have with discuss us Ciappenelli, potential who Commercial, ongoing preparations of nedosiran.

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Doug Fambrough

Lauren. you, Thank

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Bob Brown

Thank you, Doug.

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Doug Fambrough

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Bernd Hoppe

Yeah, thank Doug. you,

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Rob Ciappenelli

Ralph. you, Thank

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Doug Fambrough

Rob. you, Thank

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Doug Fambrough

RNAi and tissues with that presented success. positioned well data in molecules Thank HBV both the hyperoxaluria you, extremely I in near-term, It's delivery primary Dicerna to Bob. for I'm long-term Dicerna extremely believe our and pleased really has a time. today outside the of liver. is exciting

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Operator

you. Thank

We our with SVB Instructions] you. question-and-answer will Thank session. And [Operator now Foroohar have our from Leerink. first we begin question Mani

Mani Foroohar

quick Hey, taking question. my thanks one around for timelines. A

more Let's in progress look a whole. and at data program around the the as

get think in clarity that we about time asset? about not whether data this this point right at regarding and opt-in get potential partner considering clarity Dicerna around And the on we combo think their is disclosing should more decisions Given which hand, on how would the on horizon side Roche's or decision? would is to how earliest therapy you what

Doug Fambrough

much they on Phase for expectations Roche we that their when about exceeds very I the thanks or your conduct is X information that that provide had can't Unfortunately, Mani, excited they X trials. Phase question. Roche's collaboration. the entered to Hi, meets know data

on those but they're what and provide move very in to will their when combos so Roche forward information only initiates we studies. combination timing, are studies, And those eager

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However, the is a milestone initiation into Dicerna. pivotal for trials also

entry. participate And provided and global for to the co-funding pivotal funding by can milestone so development be

So co-fund will case Mani. I base assumption Thanks, when Dicerna development that, the comes to that pivotal is pass. think

Mani Foroohar

and Great [indiscernible]. congrats

Operator

Miller have from Jonathan we Evercore. next our question And with

Jonathan Miller

safety larger worried about to naïve more with Great, guys. it have Thanks this. I we transient to taking issues saw. and on indicate my much is sort the that should the that so larger or mark therefore that my a new liver question a as well theory be be ALT that those already function that we patients go trail patients? for we seeing issue around guess TB going as potentially question. in start spikes we their as that of include I is is understand immune But they activation trials, and something

Man-Fung Yuen

this you thank for asking Okay, question.

surface may showed In subside mean, that, and having fact, the of drop correct actually you're I'll immune to therapy of this not, on - and I decompensate related show actually and analogue actually and also very patients second any that because flares spontaneously is But in occur. if who may the saying that the flare, us that liver that you of concern advanced patients mean, that ALT - nucleoside you are relatively data of can really see those may and patients the you this group. antigen, they flare is remind there low treatment who compared suggestive was naïve disease is patients, on I were

planning are and be backbone. will had relatively treatment the combination the are analogue hoping most In be all therapy those who will be So and the because in already are this trial safer we it at then, naive. patients they likely the the when therapy patients, to future, to flares, do is who as even will analogue treat get for to we the the are therapy, flare correction nucleoside we on nucleoside board therapy, NUCs on severe patients, and expect not as this

this So is thing may consider. we that the

Doug Fambrough

Thanks, and Yuen Dr. Jon.

Operator

have your our your on you're we muted Perhaps question And Stifel. next Stephen with Willey open. line from Stephen, is end.

Stephen Willey

taking sorry the that. for questions Thanks on Yeah, and the about congrats update.

For small given be some in data I other And we've were to I just of also relative commentary would frequency being injection X Dr. RNAi maybe excluding to competitors site characterized the the helpful. guess any give competitive compares Yuen, provide I competition, just just some other then how well of number other of given context contextualize color And the but would seen would us some interest that other the the rate are be us guess et of patients you. mild, products. from around reactions different as Thank they not know the you incidence but this the that could of cetera, gene? you baselines who data and of knockdown

Man-Fung Yuen

Thank you. so Thank much. you

fact, injections group, I and mild. X reaction site patient were X presented in groups if each to to will table that there will around at of site fact be patients, of the the the In reaction, concerning that in injection have you, they X we most look you

after or don't tolerate see very think mild. I on high. reaction reactions actually defined very mean, site they were injection most So one I mean, of injection, moderate injection the who them proceed patient quite is of We depends for, and reaction this, when well, four have the so reaction don't I they the except hours we

X out concerning But single with X of, other including So trigger, to study gene. the mean, of I to X are X from mean, I are terms very the any one thus, the this see of data, this of and according the data I we two efficacy this that to have the and compared in the percentage see the X of encouraged triggers antigen, may the by don't X. out - of mean, is or sparing surface difference at least, suppression agents can one be potency sorry, we Obviously, most direct I the that to comparative suppression reaction that But of the trials. in we we is prolonged thing prolonged of surface of, antigen clinical see don't studies. lacking

this is very So, one least say. And very actually I Dicerna antigen. at potent and suppression I a all with surface sustained of RGXXXX that is think can

Doug Fambrough

Thanks, Steve and Dr. Yuen.

Operator

have Yigal Citigroup. from next with We our question Nochomovitz

Samantha Semenkow

the for S-antigen definition period taking a a here look know, is like reduction and HBV Hi, context last would this this Thanks fine of questions. for put Maybe to after the suppression? follow-up is up on of, in on Yigal. for one trying terms the into Samantha what a for and you

Man-Fung Yuen

repeat Sorry, I Can again, can't clearly. sorry? hear you

Samantha Semenkow

apologize. I Yes,

see a something for of clarity would look the is what evolving to definition reduction? before landscape? there in Or wanted that And is degree there would Just appear in standard for still patient effectively cured? like is HBV the of S-antigen considered a be terms

Man-Fung Yuen

loss Oh, to think the surface is the antigen. now of consensus achieve

mean really So the in I mean, goal. plot, this the absence of I primary antigen is targeting surface we and are undetectable

Because when the of who they in showed of XXX, sustained on with patients, surface have absolute the follow-ups. I than our less antigen antigen level than the actually presented less high patients, a patients who data, chance losing very how response data patients a surface had that obviously, a So deliberately for achieved know these I we many XXX? subsequent

really don't antigen surface very, for off we X what So about want see to a are I obviously, mean, level, And but IU at this cut talking log absolute we we have loss consider surface I for least of we will complete at and reduction antigen, than is of less definite least the in to blood. finally, per antigen the surface want have XXX really ml.

Samantha Semenkow

then rationale potential availability, are? frequently just one they that Got patients for know, that HBV-DNA experiencing, you it? just What's greater magnitude more additional the naive, the follow-up. new, the were not And of degree

Man-Fung Yuen

I srRNA. too have monotherapy one, explain don't it's patients, just We this, and much only but to they don't have the these the nucleoside data mean, differences using beforehand, analogues is, a

this mean DNA also saw that study And at terms we flare flare think that - the this have level flares players baseline expect when shown with nucleoside conversations in board, we will are So, these analogue naïve, on patients high higher in the is logically because treatment compared oligonucleotides. to will be therapy. patients will on then therapy is antisense with to who analogue nucleoside other they and we ALT using of more they the have I

Samantha Semenkow

Okay, you thank -

Man-Fung Yuen

Thank you, Samantha.

Samantha Semenkow

you having thank - Great,

Doug Fambrough

thank And you Yuen. Dr.

We section it's Hong participation your Thank you are Kong, rather from for where nedosiran know of I to proceed the presentation. late. going to

the course, R&D of Dicerna management the at whole. on a Rosskamp from For team of can end additional Day questions questions HBV, field Dicerna and, rest the the as Dr. of

So forward. point, let's Thank you. at move this

Man-Fung Yuen

you. Thank

Doug Fambrough

therapy patients with target Nedosiran multidose forms treating of and PHYOX primary profile forms for which present Dicerna's all open-label Yuen. we Dr. hyperoxaluria is comprehensive have believe a X of potential you, we today may Thank in hyperoxaluria. from study primary Dicerna's will all data

Operator

[Operator you. Thank Thank you. Instructions]

from Luca RBC. with our have next Issi question We

Luca Issi

congrats one Oh, the taking terrific. quick on Thanks Two my very for and all question here. here. progress

very PHYOXX, end do execute to of question approved was could challenging the in X, talk maybe reason definitive. that difficult the the PH out be second early three on then there But is characteristics will in is gets small X. you. any think PH And there ordinary maybe it's one, you biological about Or in Thank itself fall? worthlessly First you to the how again, anything so those nedosiran so believe just be patients? the that if to on Alnylam baseline

Ralph Rosskamp

take With baseline in and average thing The in had patients XXX now I'll actually observed study to December already of the we the question. when already ability, some was which they're which the patients said criteria at anything that completeness study, we PHYOXX of we, we respect different, to the Alnylam the PHYOXX X initially and And collections partially year. recruitment have Uox this reported accurate really out there was XX%. in of have same repeating the to patients contributed urinary complete these and PH opportunity been having patients values is date the know on but now wasn't the in think variability nothing we there reduction not you are of single-dose we Yes, collection is with XXX. this as The And only this the is urinary the of in we have this, to there. respect in saw of PDUFA already, I the as as ordinary the study.

We are well on our patients. recruit way to

patients, timeframe, of we have that some sites continue pretty the kind to now So a we enroll included were down, are will have into study, hold, we study. and patients the in first patients confident the hunkering I'm but COVID, into the because the all

Luca Issi

again. congrats Terrific,

Operator

you. Thank

Werber Cowen. our have We Yaron question next with from

Brendan Smith

Yaron. Brendan a is congrats This much for on for everyone. the and very question day on guys. exciting taking Thanks busy Hi, for

touch us treatment, You from kind Uox of of sense reductions and a respectively, ones can and, at HBV. both look of kind expect time in you wanted couple after us. Just over PH I your of kind Or course of go to to time would But know, you or quick give you the anticipate just just applies at these you would later time a with you know, decrease unreasonable of that actually rapid over a kind up S-antigen you know, a this is thinking, do when to I then kind necessarily treatment? kind point we pretty there? to expect, continue out continued I'm follow-up. number ahead have of to of a bottoming thinking that and thing

Doug Fambrough

Thanks for Yaron. question, your

XXX is maintained dosing expect and PH, little HBV. that over have the continuous PH for With what should their on all day I between do think idea different answer that achieved by we be and or stably we time. will the patients most believe a with

probably sources, can oxalate indefinitely dosing. that different some full maintained you're level always but there's seeing of measurement, course, It's treatment the note a barrier monthly that and oxalate with Of pathway creates from the effect be important continued to for normal comes not that disease.

bring the down do we so, levels. to not suppression normal is oxalate of goal levels and to expect normal oxalate And

in expectations a think that to what the So, immune to HBV, different, do viral to going successful, that change when suppression. our the should that And achieving continue the S-antigen seeking and I maintained. particularly bit X other is in on markers biology be productive through I it's occur are achieving, we're high be because of shows beyond levels PH the and what's what that a what little think driving RNAi goal of and meeting down the we would for patient is we're In suppression to extent period enable go S-antigen it's should response. made we

apparent, in we'll from you the to-date with the in HBV future predicted gene study, by X-sparing have is occurring reset by the consistent expression But will, Roche. that that data in well pleased So for that's conducted this of studies I'm we looking in our hypothesis, as that generated as be patients patients. if that follow-up long-term

please. question, Next

Operator

our have Robinson Karnauskas Humphrey]. with question Robyn - [sic from we And Truist next SunTrust Securities

Nicole Germino

congrats today. Nicole presented on the Robyn, for is this on you've Hi, results

the more someone PH And a of less for Can the back around Just you XX% way enrollment be PHYOXX, any us there going completing us to granularity little X? type give bit progress enrollment? just a you for you color or near-term will to enrollment then little can with give on bit PHYOXX that? more

Ralph Rosskamp

have in later in have our part initiate hear With of we after first study patients your three quarter three, over for X patients PH will in this question. done PHYOXX, the single-dose patients. demonstrated these reductions we X in PHYOXX And and as roll I we the into study study. PHYOXX PHYOXX urinary to then patients, substantial PH will in oxalate couldn't respect PH

So the that in of year. somewhere would occurring be beginning next

X fully is next year. second multidose something half to would only expect PH understand patients sorry I'm couldn't your So of we in I second in data your question. And the have part of

Nicole Germino

Oh, sure.

that. Sorry about

you and can And bit PHYOXX on do if progress so, on a you enrollment? have that progress completed on give enrollment? COVID enrollment? the Are for impact color around a little with know had So specific more the some timing of us that you nearly more I

Ralph Rosskamp

had with we guidance any think when be study completed. specific don't respect the would given I

presentation, Rob's enroll enrolling started our sites Japanese to many the We we of in have can again with next you the we from completed, weight enrollment site are we're so sites that expect we you were QX when actually after targeting now that's be the year so But the QX. PHYOXX study. heard that. would continue where COVID the access first which The limiting of because patients do to pleased as math

our together. we're to once able package the database So we put off have lock PHYOXX, of

Doug Fambrough

We're - on track

Nicole Germino

thank you. Great,

Doug Fambrough

please. question, Next

Operator

is question Ed Arce with next Wainwright. Our from H.C.

Ed Arce

below day to data everyone. dose first the had a the this compound is days the units and final duration my over which log and out XXX all reduction HBV, importantly your on dose noting Hi, for that international XXX prolonged the taking a Thanks for question dose, threshold. X.X is of questions mg XXX around today. past congrats in is X.X My the

forward how many And and then this So that given enough? I'm a will analysis your does be ultimately doses how wondering inform follow-up. have I ongoing

Doug Fambrough

case have amount XXX individual drop day for that Ed. the n we that day stable in in advanced S-antigen is fact fairly being has astute XX That's because XXX, between into How we're anything an patient wait that into are but team equals is individual number get to going X it that unfortunately, I'm am into prior get but future is presented about I'm not an it's levels number is something most and a read of Roche's it's the XXX our that active that. a the in we'll team and since and an the day where not do to particular, Phase and day trials when significant that of XXX observation how seen fair below and talking you, level informs fairly after that to to analyses patients. that Roche n one day fascinating is that the and is going discussion achieved to see X I dosing X I from. Thank ready hesitant has know reported in to comes

Ed Arce

juxtaposed that follow-up know on do logs milligram durable, you X mg is suppression. the this you prolonged to fair you on about the sort think Does of X.X these the How you the I low in reductions Thanks. And But Okay, to that terms dose. of the that are sort X.XX of saw the you six numbers. Then of and of dose to again, in log at enough. think you averages effect? that does recognize how somewhat and suggest terms competitively? plateau small any all very

Doug Fambrough

of MPK. did patients who've reached two and the Yeah, period dosing day the it's mention we patients who are well, that's the like n initial an X of X two initial additional trajectory XXX. is so stronger that the for patients the the X I have in and of

to And and initial of so plateau larger and a overall course, than there's dosing we trajectory, we'll that data. we those think I rather some see initial that dose that think the there's that But given some there of But see patients X.XX. S-antigen have went average for intensive X.X, likelihood but response while likelihood more will that to is will they leads X wait not reduction. more we

Ed Arce

That's great.

Okay.

Operator

with next question from is Baird. Madhu Our Kumar

Madhu Kumar

Yeah, thanks for question. taking my

want to your the thinking international of it partner long-term partner US kind US big PH like kind to or in of to would the back one you but goal with you a long-term, there notion only, build is commercialization focused or and sales? and efforts in regionally there big stepping for start y'all. So ex-US picture the of kind commercialization say, about Is

Doug Fambrough

But US there in commercialization risk with is discussions believe sources there at we for our Hi, potential the in move co-commercialize, Madhu. word with commercialize that in to commercialization fact, profile we point Dicerna the likely I'm partners. strategy to we favorable, is commercialization, not PH, sure PH commercialization in are United caught core end. United and and is the global the the in right. at I co-promote every the ex-US and And the the A for But is partners AXAT ex-US program targeting HBV this States. pipeline when take initial are in States broader the field. will

Madhu Kumar

how Okay, y'all thinking about have from and the candidates ongoing? pipeline partnerships you'll of of then internal kind currently. three from collaboration of versus kind following years three years you going from left trend Dicerna now, to Like do take that do how versus be now, of what the about internally think to line for kind much in-commercial of is swathes

Doug Fambrough

are Roche all we So and taking quarters based clinic liver and on that clinical programs programs include in our opt-in, on programs is technologies the Boehringer, I that for the the Dicerna outside high programs we're would by partner to have will partners, has of the own. ones additional will collaboration that we X programs advance will Bob coming that until we core very the note bar announcing Alexion, of be that development And our that and where conducted Phase Lilly, up programs our our for for discovery are described.

take think one I can we more question.

Operator

question from with Miller comes Jonathan Evercore. Our

Jonathan Miller

available deductions that in can obviously, do that's alpha study, the much to you anticipate the getting PH guess and Great, reductions baseline same the normalization, time? but PH to on you being And the taking I'd like secondly, thanks part package seeing X the will mean, their the X to robust be PH they're I at for NDA so are of are going non-responders, when Uox whatever data from was? Uox my you not to return all of guys. sort on I X, question the getting characterize

Doug Fambrough

I patients I'll Ralph PH characterize as Let would me for X. pass and non-responders. to the two then address PH colleague, not other X my

our day did note and focused points are PH we discussion patients. have We the would normalization or on at XXX near other reading that X time for there normalization patients see other

Ralph? will variability care just Ralph is mentioned, data single fact I'm I in that the of achieve that and therapy So one, quite X for point. over we on it's label believe have a X, and too. tremendous just disease an the function that nedosiran that the the focus that of is optimistic advance PH a we PH effective on standard management

Ralph Rosskamp

add is work in Yes, I want that X. nedosiran nothing believe PH mechanistically, to to there shouldn't

levels appropriate the in the have in PH transplantation. And X. of the knockout oxalate we that this liver in to transplantation kidney where mouse liver it case, normalized urinary and is case one were So model the works

speaks oxalate also sole source liver of and that from the So not the from to is the organs. other that coming

as XX% had an the already, study. patient PHYOXX response and mentioned over same the I in So,

We'll stones be they post onwards beginning this the will will is demonstrate in more wait the to the six up safe and stones coming like those up. we the months with history continuous respect measurement a especially the at years at we formers, that next days from so plan six the soon. month, which we higher patients the are webinar ultra with activity younger basic months to some history very to study, then trial patient, forming stone have we coming study even the have out more are have with for soon quickly have by So been natural the three data six just roll they to we With just natural where young urinary that's and patients are enough oxalate, seems know ones at oxalate is and including those data, they that in this case. so to able recurrent that that the in logical OHF

XXX urinary have showing isn't as will that least oxalate study data, substantial showing the the have of a to that will enough month will month we six expect reductions to patients this support hope the And show toward in then we the XXX X endpoint PH for the hopefully you durability continue six years, with study, So adequate well. a we therefore data get those at two but continue, effect. multidose data, and with that link for then we study

Doug Fambrough

Ralph. and platform. close. Thank to on to our with everyone in Dicerna's R&D we're Day that you. who's And hear nedosiran, you want going this to Thank a to thank I tuned RGXXXX draw data varying today

Dicerna Pharmaceuticals (DRNA) 10 Aug 202020 Q2 Earnings call transcript