MGEN - Earnings call transcript Q3 2018 | Viridian Therapeutics, Inc.

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Operator

Welcome to the Miragen Therapeutics, Inc.

Third Quarter 2018 Conference Call.

As a reminder, all participants are in listen-only mode and the conference is being recorded. After the presentation, there will be an opportunity to ask questions. [Operator Instructions] I would now like to turn the conference over to Daniel Ferry, LifeSci Advisors. ahead. go Please

Daniel Ferry

good and Thank afternoon, you, everyone.

and call Chief and the Vice of Paul Miragen’s Adam Jason President Officer, Bill President Rubin; Development, and are Executive Research Business Marshall; Executive Officer, Levy. On Leverone; today Chief Chief Officer, Financial

begin, statements to remind that I could risks uncertainties are call subject this call. everyone about statements cause like reflect contain our of as of statements to forward-looking that note will to these webcast forward-looking would differ. actual the we that conference only Before and company. Please date results These and opinions the this

that Form other will Marshall, and any cause outcomes Bill? discussed or greater to revise differ filings Chief revisions detail in to or our now Bill would call forward-looking actual light or with Forms turn materially Officer no forward-looking of to XX-Q of Executive from these such recent and most on like the Factors results in our over statements by the XX-K or could on We to information X-K SEC. I reports undertake to statements of implied results publicly are President and expressed obligation the filed in those new release events. Miragen. in periodic future

William Marshall

and Thanks, us for for XXXX. the afternoon, you Dan. our Good for joining thank quarter corporate update third call

enrolling trial to our in occur trial as our global their the the call as third advanced thank update X planning trial SOLAR for questions. quarters begin of our of cutaneous CTCL. the launch necessary a on patients all to of lymphoma, prepare in productive to clinical had for today’s and the employees review significant of and cobomarsen and we activities for clinical clinical the fourth Phase or tireless a which an and launch SOLAR, such efforts, I quarter A begin in T-cell amount with like and a the will our quarter of we would months SOLAR before financials We preparation to opening call leading XXXX. preparations of We brief in quarter, up programs up in advisers

of the disease with disease patient’s for least quality from cobomarsen additional approval the in with of endpoint, evidence in improvement over entire nodes will no four of the skin also in and months progression severity as a secondary survival a be ORRX. viscera, blood at known consecutive Progression-free maintained lymph we the the body or outcomes Drug could plan life accelerated to believe endpoints allow apply initiation Based the the trial, three The cobomarsen of XX trial, XXXX to also in we is as candidates evaluating stage we SOLAR the for microRNA-targeted active vorinostat. or be of development which out of greater advanced expected having to known efficacy the each expect close approximately an The is anticipated primary is ZOLINZA, enroll of endpoint will study With In the clinical across indications. need versus patients to Food use discussions on of and SOLAR SOLAR to XX% the results U.S. group. States. the defined of reported our the treatment safety, per comparison improvements. product study monitor several study control response, as us rate we study Administration, United for as patients objective in patient of this

decision SOLAR in believe Phase to into funding. program from SOLAR be in and trial We previously the Leukemia with supports we $X trial. invaluable Phase in X million up advance support, pleased the is Society, which are conducting trial As data including X the to the association disclosed, CTCL our to of Lymphoma providing cobomarsen our

complete study the quality well includes data the total be includes appeared Hematology via of in of December. Annual at been of trial, American CTCL. various responses enrolled life generally of fungoides, measured at scoring X As in and also in with form to XXXX to and dosing We present who by improvement X demonstrate to tumor appeared to Meeting up observations XX in previously from Phase trial in Society cobomarsen Phase routes patients Cobomarsen tolerated cobomarsen patients months. of efficacy, data common tolerability reported in long-term dose this improvement the all durable The burden safety mycosis have our plan XX evaluated. most from skin administration the levels for

therapies subtypes trial essential are initial seen devastating seen cobomarsen the highly associated poor for Our this times from X with that patients in with patient a these survival patients in of treatments, adult T-lymphotropic believe to indication potential of this aggressive data the of previously this XX Phase with T-cell we data the previously prognosis with We the remember is type to ongoing existing in important despite ATLL. malignancy in infected from have of year, extremely disease. We improve a four ATLL trial is Patients form ATLL. virus additional to months ATLL this morbid human after second released patients clinical announcing poor cobomarsen had are in with mean it Earlier our new is initial outcomes or disease and XXXX. T-cell diagnosis. prognosis first-half anticipate leukemia/lymphoma, X.

remlarsen. to Turning

a activity randomized formation history keloid a assessing conducting scars, hypertrophic remlarsen, clinical subjects in scarring. X prevention of form double a reduction Phase the for currently in are of of keloid trial tolerability with We safety, or persistent the blinded and

establish X increases or expect small trial in presence or remlarsen to and clinical will formation. of X will an affecting clinical believe to predisposed up own a we that The will from injected clinical data statistical either revision. of formation Phase of in trial cutaneous We this their in build subjects in with is without necessary dosing patients. frequency keloid fibrosis, matching expect for as serving where us tissue Thus, help trial the patients lesions to induced results We are the at sites then healthy be trial U.S. an dose, a of data the We wounds placebo. observed of believe on months appeared this reduce report Phase excisional to the of initial opportunity receive in exciting keloid power deposition potential with the of human in may relatively second-half remlarsen of are volunteers XXXX. number enrollment scar control, enroll absence sutured to the patients the number be observable healing. remlarsen trial XX for determine to the the which multiple cohort, Subjects small will keloid and after trauma subject XX to consisting that

and this We the from inhibit corneal supports corneal other the of remlarsen that anti-fibrotic announced patients in remains topical remlarsen our ulcers may corneal data also recently preclinical blindness fibrosis corneal of and suffering of no causes We leading options study worldwide. believe studies treatment ulceration. Patients have our and available, presently vision belief scarring in one data application be and investigating an from from suffering fibrosis indications. corneal of of to effects scarring improve pharmacological treatment effective

Turning MRG-XXX. to

growth healing. important MRG-XXX microRNA-XX, which in Servier. developing collaboration of vessel and MRG-XXX been be an new inhibitor blood We to has the shown the are regulation of is with in

indications, MRG-XXX better failure studies in its that the vessel other X heart multiple potential incisions, increased being and trials lacerations. both including ulcers could evaluated from clinical conditions and reminder, As use a additional in acute growth X allow of treatment including intended benefiting new separate support and for Phase blood and is chronic to oxygenation,

patients clinical trials options stage candidates have product in will these supporting from the in evidence of results data these an expect high now believe are Officer, provide the three little indications, that, targeted XXXX. stage announce new of earlier Leverone, to to we is will Chief where to I reported Each We therapies the turn potential today. therapeutic programs. patients Jason need. the have microRNA-based our financial to We over With of important clinical by excited summary, our class review we Financial effective available. further of call therapeutics programs no three our addressing Jason? microRNA-based become for In to need advancement

Jason Leverone

associated and with team. primarily clinical in Thank million of we personnel-related an of to Net million of XXXX. quarter that We clinical costs quarter These cash reflects quarter financial third you, third partially the an was cash, used XXXX. team. $X quarter fund investments opportunity million third in In our in This related Phase short-term in by early XXXX. property in in increased and $X.X third I increase the the development XXXX. for third of together Bill, increases to compared third and XXXX the $X.X of offset quarter development cobomarsen, and SOLAR sufficient totaled provide general will to we resources activities million the of of expanded operations $X.X XXXX. into the the quarter $XX.X $XX.X with costs months as the and have This for to X our our the intellectual million results. current million fees expenses and believe we afternoon, for approximately development and totaled everyone. for equivalents added million research lower appreciate for good quarter with for operations personnel-related XXXX, administrative increase $X.X XXXX. the of to expenses ended nine XXXX cash incurred Research administrative our as third and be General compared to our legal trial XXXX, first quarter were

for innovative dedication earlier open helping therapies that, and open want call the and way new Operator? I said what lead we to questions. for are medicines. the who Bill commitment questions, call patients of to microRNA-targeted continued employees developing in of Before With their to ask thank operator I’ll need the in for all for the Miragen our echo

Operator

Certainly.

question We [Operator go Miller Evercore. begin Jonathan will comes Please session. question-and-answer ahead. Our from first now Instructions] with the

Jonathan Miller

thanks taking the guys, question. Hi,

seems out keloids remlarsen on data for It Let’s pushed a like gotten the bit. start little has please.

XX to year. Now I remember the that early you preliminary I in you’re followed get months. and said those seem saying getting understand of are But for some data could patients we the part previously XXXX, second-half

just I and initial to a timeline patients? sense wanted is get like is there the what So XX enrollment with going of these how

William Marshall

Jonathan. Thanks, Sure.

– The studies patients for So be trial we the of be provided those XX track. that will of general monitored kind on was, The months. will believe XXXX. I sort remains had keloid guidance

earlier However, be the of seeing. we’re hopeful that results analysis, out some read on able of may we points we’re based to

good feel in point a still in presentation which position good that for time get expect opportunities one that of really probably we at we in meetings. meetings, on with medical more focus as in So appropriate remlarsen major at drivers the to trial was the – the around and would details keloids opportunity

Jonathan Miller

only it’s XX-person you’re talking being obviously, makes sense. you Do involved. trial fast? – a And enrollment enrollment That medical on pretty centers here there, it’s expect about multiple and be to

William Marshall

we the actively we we’re four keloid and a sites people looking Well, of are United in too with a database leveraging high are propensity – formation. at for States

that, We recreate it will because be able hopeful XX quite are patients, to is effectively. we

greater we our picture be but has as the We’re – of we’ll updating sort get expectations. still that, – far a to so kind recruitment of been on

Jonathan Miller

obviously there. Great. saw, ASH looks a we you incremental On positive much. week, Thank like it last show very update continued abstract CTCL, up mentioned and you the

if says mean updates And not at ASH. I PR in that X from that’s length current be in expect we’d case, course, can with estimate data dosing administration the the that more than a that of this of Your Phase set of based those to very follow-up, going be to take get read patients will the is longer on Phase complete and SOLAR? there’s ORRX even that follow-up? that for taking, as I different of an we Should

William Marshall

really getting Jon. SOLAR The Yes. Thanks, are rolling we the focused in trial. – – efforts our so the on

this. to on So of on do that several patients We remain have are real continue dose drug and a to going the lot efforts will be them. we focused

into study. kind gain recruited we’ll the a of additional benefit, So new patients not but we’ve

again, different around we the estimate additional longevity whether ability – of range to Phase data again, used to were be ORRX. really our produce contribute you And going we to there’s of lot assess, know, as to to dose make So X important. is do the routes administration a should an finding that able and

focused route to So monitoring single as of now slip we’ll into we patients. SOLAR controlled be administration, trial, really of and level single now normalize longer-term the dose on the

Jonathan Miller

patients? indications that. data secondary I That ATLL right. word on we’ll in some eight sense. All no XXXX. see on make guess, the DLBCL then for But excited any I’m or still early seeing to more CLL cobomarsen, see

William Marshall

the And when find as initial populations a the towards is relatively have kind patient some indication we’re competitive quite patients. – enrollment new those we we preliminary able The question. population, opportunities some ticks where the been in gravitated the we additional once So promising up. obviously to generally indications, thanks a into find in leverage landscape have see get we with we’ve promising results. and go a the of able – data, arisen, for terms to what there of a The we’ve really frankly, is little that, that patient bit

And we be the So be said, them additionally showing – an make it high that information focused at really good and treatment additional identifying at treating suited in physicians mortality been of looking like in most in are, area ones very comfortable options it likely would with patients we and and on SOLAR to of focus promising available, time, are a to same some plan given of results treating patients trying to executing getting there. the intensely that in. focused on the patients, to ATLL on have with to we additional terms candidates is the to no types actively I providing and best patients the

other said, those indications. where cobomarsen. we that I see got diseases other additional we’ll continue hopeful like that the treatments for we’ve look I we’re confidence put larger in on give responsiveness, two to patients people expansion the those think, physicians have, with to treating we’ll patient We in a with the population – in – responses And

Jonathan Miller

great. much, so All guys. Thanks right,

William Marshall

Thanks, Jonathan.

Operator

next question Please from Our go Yang Kyung Jefferies. with ahead. comes

Kyung Yang

questions. you my taking Thank for

you is question patients place? to on futility that XX a SOLAR understand expect there take trial. is First I do analysis XX% about data. at that – analysis When ORRX

William Marshall

our the is obtains certain also on able better time able the a So, you – know, true want question. to are to for months, intent treatment there we is. thanks the The and What think hi, to allows recruitment us if rate really Yang, we’ll a then ORRX. – get kind – to any partial variables that then period, it’s during patient Any or dates really the that, And we talk give of to response, achieve as monitor to for around, be I the four understanding nice six is months. is treat want which that. that don’t for with to measure to you then firm into what

in estimate there us the kind that. anticipate go that, would when So additional just are many that time, on occur. at experience we moving give additional of get trial, point this on as too parts to for give would in we’ll good we a guidance But

Kyung Yang

then on Okay. remlarsen. And question is next

the you be previously, could in IPF coming out mentioned think, of that I data preclinical this year. second-half in

of update keloid, an also And also, opportunities. us partnership give you could for you on So that’s I mentioned kind that? think potential a

fibrosis? Thank into for arterial and of indications that IPF partnership all look you. will So when you opportunity the be keloid, for remlarsen,

William Marshall

Absolutely.

potential in the to partnering the going opportunities we’re of for a to partnering open indications. indication, around be the So – of remlarsen in for variety terms

you to As dose, very for in trial was and dose that effect really Phase designed that X us – analyze able size know, the a we’re to well-controlled seeing. manner the be frequency

So be that in a give feeling would Phase necessary the what could the likely around this X trial. better powering

terms pathologic of a humans, interest of fibrotic size terms forward. thesis sizing of gives in response information And the partnering think, in also size can additional opportunities. or to whether that validation attempt our playing us on in there not is of to hand the condition into with showing the sort we of is effect effect think, the both that Phase I but of would X, partners, from in important be I sort also take want we only it looking the would partnering at which

indications. in which want And if partnering we tend look On think we opportunities, to generally, where an may for in were additional update hope about we terms then we would to to of earlier experienced continue the are we there. committed well really to about next-generation would of additional stage that gave IPF, of the what here in I area say, and We talk addition and well that interesting necessary we’ve us capability is in the looking to front, larger that IPF that report additional a as partnerships in – report outcomes-based meetings the in data carry will where for to the folks we inhaled at be therapeutic door development. studies, that Phase the open medical out out analysis to fall, have to gone clinical help some the compounds at late will as meeting terms be

to And appropriately think in I understand from fairly that of afforded where implicated. the the different lot spend are therapeutically, again, next for might a applied has most we’re So, us. fibrosis be moving generation There go indications compounds been opportunities think, but we many to I looking partnerships also could remlarsen additional of open asset. into time continuing miR-XX

Kyung Yang

Thank you.

William Marshall

Thanks.

Operator

[Operator with Please from comes Wedbush Securities. ahead. Liana question next Moussatos Instructions] Our go

Liana Moussatos

Hi, my you thank for taking questions.

mentioned and a kind in data would – too had ATLL And Anything do first-half. survival earlier is data different? year, expect be of first-half? in released benefit? you initial endpoints early this the What You and to see the we it additional

William Marshall

Thanks, Liana, question as great usual.

leukemic – we also lymphomatous symptoms. was the there a and was presented amount be ability look the that is what nature. aggressive So the of the ASCO. of fair to specialized the other at where form a in broad more excitement had a looking of the them – community. we Both data considered type at was data of one some this patients very Once flow we’re cancer really them and from two maintain one in things released at the would that that disease here We the

able have additional to we So recruit been patients.

understanding between quite There’s One the only form. involvement skin different of There the subtypes of aggressive a and leukemic tumor. that is we’re are in forms. differences the lymphomatous the things interested this

additional is bringing the these in are analyze it safety to we to because subtypes. in be also able So of Phase patients cobomarsen effects build then patients a what are X, again different and the base

that to really begin us can hand, I a what of is we set what with experience tell disease we is seeing. when dataset, within you present treat the So that bigger should up longer-term so we’re the building some and for patients we’re and appropriate can a doing the patients the most to a push direction that in draw conclusions and responses in

We are Phase be drive understand would in the towards the see what more as ATLL, efforts in best it’ll constant drive right with X to in And help us and also decisions design coming the sort the back area. studies of trial from now FDA we about discussions questions data that There design trial not allow would I in to done from believe U.S. answer was Japan. there been the or one would in trial to regulatory The done has perspective. likely in U.S. – us a my and that us a the be drug endpoints conditions acceptable the

looking the FDA. if be so can those applied at be U.S. with And also we’re determining we’ll but precedents, the precedents

Jonathan Miller

Thank you.

go Kumar FBR. ahead. with question next B. from comes Please Our Madhu Riley

Unidentified Analyst

Madhu. for taking Hi. Thank on This is our call. Jennifer you for

Thanks. rates Just CTCL the in most that if for on might bit shed Phase feel ORRX I’m you information you briefly cobomarsen in Ib be? a wondering study more representative what can little

William Marshall

Jennifer. Thanks,

find it’s was a of of route a Phase we study around – get And X to we – response. answer that dose able administration. that consistent most lot the regimen this ranging, fair XXX amount, the a that a IV the of is appeared questions gave designed dose question to – the employed do the a So really is in infusion was that rate given we’re the it what milligram in

for ORR, not of you four with or at people of – study. If about everyone independent as time There about a ORRX, down everyone, to hit Again, and overall that confidently you and answer If XX% we the patients suggests if for we see we have kind four-month little independent very study say, to measure administration response at that on the but went than in month, some hit could the range. didn’t a of is durability, bit this high on that the study zone just the drug on we’ve more the because hand was patients tox, who level. from it durability to weeks into look yet along started didn’t we had were this to – a there. seen dug said, four-month that we the then ORR. the so route amount that and of achieved we’re rate what this drug, a in begin many of of them on more somewhere then partial And enough we of have or remove and data dose XX%

own essentially that believe durability does from we’ve ORR there. on least is be we So The first And to now at appear trial. proxy. the taken its a reasonable lessons

the the route of keeping drug. the dose, obviously, importance on I most think of the patient the but also the vital administration, important

So sort ensure on and protocol, mechanisms the that are of really mechanisms. focused in on stay we’re convenience the patients principally drug these to

not stay would so on – the we even centers drug. or that is is to that closer the home to patient provide infusion infusion, able home So

then So, believe worked another the primary important on Progression-free overall extensively equation obviously be we’ve reported what going endpoint. is FDA we is the is with the rate important overall is terms, endpoint. ORRX most to outcomes. will And – the in the be in patient survival

of is do in itching PFS, from you the the has used sort been of up, how if ORRX, the we’ve tumors from the factors area. the then and effects study. is good one drug. the clinical of line from differentiating a it a where are area discussions And it’s the seeing I side most of cobomarsen medications these that are been this And are and is in our FDA, in in patients’ outcomes, things perspective. really with improvements So quite for the And believe the the important in other patients you discussion and what seen tolerability improving that one importantly, pain the

a we the comparator. the So, way we we ORRX XX% felt a between between in detect and that have able study be should the think cobomarsen to powered

that. some have good pretty We ideas around

think up approval for we can patient and would reported would sufficient be is has accelerated the things that that also So we number, one the rate in be would about, the item, we’ve involve other PFS pick and response words, the it review but FDA other guided that talked a outcomes and safety.

Unidentified Analyst

That’s Great. helpful. very Thank you.

William Marshall

Thanks.

Operator

Our go next comes Miller Jonathan question from ahead. Evercore. with Please

Jonathan Miller

Adam just on quick follow-up talk. guys, give Hi, chance financials. Maybe a could you a to

guidance Your pretty what XXXX? still up. and that’s seems burn expect obviously, we SOLAR to initiation getting you any the through manageable, as in numbers with R&D you ramped go and Do have going accelerate for

William Marshall

to one to if that over hand Jason on wants that? I’ll he So comment

Jonathan Miller

can talk Jason Jason. too. Or

Jason Leverone

Jon. Thanks,

initiate So $XX as our last so. through to study XXXX. we And increase has SOLAR quarters few over R&D global burn would the that expect million roughly the I been around or

Jonathan Miller

you’re So increases $XX quarter, saying? million it from a about

Jason Leverone

That’s right.

Jonathan Miller

Okay.

Operator

session. would back now question-and-answer the Bill for closing over Marshall the conference remarks. concludes like any This I to to turn

William Marshall

Thank taking to the Miragen. sometime afternoon operator. up this Thank for progress latest all you, on at you catch

building to need. by energized patients results these medicines on and date to clinical obtained committed bring hopefully to results We’re we’ve the life-changing to in

with us have any questions. reach As out always, at time I afternoon. a please feel great you to hope to free

Operator

today’s call. conference concludes This

day. disconnect may a participating, your have pleasant You you lines. Thank for and

Viridian Therapeutics (VRDN) 10 Nov 182018 Q3 Earnings call transcript