MGEN - Earnings call transcript Q1 2019 | Viridian Therapeutics, Inc.

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Operator

Greetings, and welcome to the miRagen Therapeutics First Quarter 2019 Earnings Conference Call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Dan Ferry.

Dan Ferry

afternoon, good and you, everyone. Thank

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

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

statements We that any on Bill revise filed miRagen. by revisions are Form to like detail cause outcomes and our on greater would or could will or to I the materially and light or other of to forward-looking Executive these the information Officer our Chief XX-Q President Bill? statements actual in filings recent those implied results now results an turn or not differ Forms call in most expressed discussed to in undertake to such the SEC. in of Factors over periodic publicly from events. obligation forward-looking new release Marshall, and reports XX-K of with X-K future

Bill Marshall

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

brief as review are We've to financials of clinical strong needs a call today's clinical-stage questions. our start of development potential all across address serious the of product range which with pipeline, about our of intended enthusiastic with an remain We by pipeline, update programs. of will diverse progress candidates well advancing a as earlier-stage pipeline opening preclinical We X call unmet indications. XXXX our up across our and on before started for the our our followed each

levels our Phase a team our currently the cobomarsen, pipeline We we I'd II like to being data are in we inhibitor global start is of drivers be that investigated advancement began miR-XXX disease. April, our in to pipeline. number indications supports of our dosing our patients SOLAR the microRNA-XXX trial. has rapid where are which by believed elevated update of In generated, encouraged believe with

a trial safety doses diagnosed patients the XXX compared of is SOLAR fungoides reminder, T-cell in As efficacy vorinostat, to mycosis the of the administered active and cutaneous evaluating or form cobomarsen CTCL. control, milligram lymphoma of the intravenously with

approximately apply during goal in sites and believe on we opening clinical XX% second We end quickly patient's States, improvement of the the quality-of-life the United point review, SOLAR XXX cobomarsen monitor a end trial from FDA, primary point, end viscera and of total the as greater X entire SOLAR secondary to point achieving point disease that the To report Progression-free survival in with of to XX Australia points to the initiating end a ORRX are disease to as skin us outcomes remain track be worldwide is a the We of from or of consecutive patient-reported Europe with patients. blood, or enroll allow half United sites XXXX. or Based will months. the CTCL data at we of additional response the in with objective defined trial we evidence the in no on nodes primary over severity in discussions believe of rate primary trial lymph least improvement. the body the secondary maintained plan use progression to SOLAR accelerated the States. for approval end

being society Partnering equity invaluable the to support, Leukemia includes conducted providing being Lymphoma and million reminder, reported the results adult patients derived treated aggressive cobomarsen. from the in with with up ATLL to which $X new a months. up in this leukemia with As is is from with partial Society. to SOLAR miRagen T-cell treatment trial had encouraged funding were after association upon well HTLV-X. as oral the as this Congress milestones month, We current ATLL on of treated to supporting X completion at The International conference, have we presentation data to identifying cobomarsen lymphoma help standard-of-care received an presentation patients XXth XX patients. morbid disease detailed stable or for they trial with and regimen. of patients that At were the remission in X the Last subtype we pleased in already potential specified with report in for remains with highly the X months up XX

generally was patients subtype. noteworthy, believe tolerated, ATLL these aggressive particularly average with survive between these drug only given XX patients on depending that results on the addition, We are to months of and X continue X In after subtypes well treatment. diagnosis, on

observed biomarkers no stabilization clinical severity, disease is dose-limiting HLA It tolerated with related patients. serious these in events correlating discontinuations. events, well reductions deaths, note treatment cobomarsen X or prolonged evidence Additionally, biological adverse that CDXX important events, in of hematological adverse or seen was including generally X demonstrated KiXX, toxicities, and to over the also cobomarsen-treated with meaningful trial grade

promising extremely option cobomarsen Taken with face the very that limited a together, provide and for to treatment prognosis options. it a poor treatment therapeutic patients safety, currently new emerging candidate tolerability appears and that for as potential with is ATLL efficacy

patients sutured, of year. in or months of data keloid Subjects wounds observed trial second or in up small to enrollment with and Our a the that in form being to XX II the completed antifibrotic clinically continues of double-blinded of for the presence report product and keloid Phase to and half keloid the placebo this excisional of We injected a studies. both a prevention the remlarsen randomized candidate expect either tolerability scarring. matching evaluated scars, and with received for assessing determine reduction Remlarsen history then hypertrophic formation. persistent currently trial in in activity to or clinical safety, absence formation frequent is preclinical subjects were progress of

as participants. the that trial, serving increase on control to product dermal small outcomes statistical in remlarsen for to trial this of allow able to that in this are the data anticipate patients number path their development us power scarring. understand better We responses been own will the candidate the a provide with we've potential As will of

in As preclinical Building indicating worldwide, for you in scarring last prevention approved a the know, in administered present of that effective potential may and remains to scarring. we remlarsen Meeting. corneal corneal and preclinical are fibrosis results, for to to pharmacological pleased fibrosis of or infection reduction we the injury. Association these study, remlarsen data week, were remlarsen of treatments XXXX which topically corneal the this upon In demonstrating accelerate remlarsen trauma treatment healing Annual cornea for Scarring about hazing excited exist. corneal topically a in cause to cornea data Research prevent we additional induced reported the the Ophthalmology an no resulted therapy to leading and blindness for is potential administered rat year, has Vision following Earlier at the an appeared the eye. as the of

in program patients providing corneal that We in for pursuing a resulting address treatment topical this conditions multiple scarring. can of convenient hopes the are

Servier. microRNA-XX being an Finally, safety, inhibitor evaluate I'd These in clinical are tolerability like MRG-XXX. for of and X provide the evaluated to update MRG-XXX, a trials currently to collaboration of pharmacokinetics I trials Phase brief designed with in

may as has patients reminder, in other healing a is and a new in explored potential As benefit failure the increased blood role burns. and vessel compromised blood complicated healing, heart and miR-XX such where for conditions growth vascular of demonstrated flow and of the regulation accelerated tissue and being lacerations treatment from

to pharmacokinetics, as mechanism drug well In addition safety, to supportive as in in humans data in to trials both the data expected our clinically trials, this future we of tolerability Enrollment translatable generated and later is report validation action provide been and completed trial. has of of year. biomarkers expect ongoing

clinical the over, Financial a reported steady to XXXX. look the that, turn to financial of data number and want today. continue over make earlier across I reporting miRagen have to announcements we and results as call forward to thank will our I partners. milestones Officer, I the clinical-stage we an time on to employees X product progress all is I candidates. our call And Jason? of for exciting review This each Jason Chief of our now turn With throughout Leverone, Before

Jason Leverone

and quarter I with afternoon, Bill, short-term the in reported of cash in to the million $XX Net a of the everyone. summary and cash, used our and first cash first appreciate operations equivalents Starting good million cash at you, the for end $XX.X quarter. investments opportunity was We results. Thank provide XXXX financial approximately XXXX. investments. of quarter

next to Our fund current our months. XX sufficient the resources through are operations

This quarter. revenue, revenue As year. recognized quarter we the million during compares $X.X revenue last of at the recognized of $X.X first during to million of we look

million you under payment we with Servier. this decrease our recall, with recognized collaboration earned milestone our decrease year, the revenue $X.X to and a agreement in contributing collaboration quarter may R&D in Servier. to As last was reimbursable expenses us Also under revenue as a

of In growth was clinical and during during $X.X by costs first trial totaled $X.X for clinical development due terms expenses These associated expenses R&D Personnel-related II of to R&D to increased $X.X manufacturing the technology of of The $X.X and primarily first were fees related million increased This lower quarter of $X.X incurred of expenses R&D expenses quarter our quarter and offset license by of XXXX. last million million expenses, million the research the research our first Phase of development SOLAR the partially primarily in with team. development by increases the of also increase driven year. XXXX. compares cobomarsen. million

and million of totaled administrative the XXXX. quarter first expenses of last compares year. million $X.X This first to expenses G&A quarter during general to $X Moving G&A during the of incurred expenses.

data and as X our product we our team operator and our XXXX. expenses milestones higher administrative for reporting operating I'll Operator? that, a added personnel-related the clinical-stage throughout have the as on we increased share-based clinical of increased XXXX, ask to advance charges. general forward and of announcements open our each questions. During call incurred Overall, With candidates, to costs number to continued we and to look compensation

Operator

first of Instructions] with ISI. comes question line Our [Operator from Jonathan Evercore the Miller

Jonathan Miller

a I few guess have here. just I

actually last in longer cash that you First, a Just for have 'XX? XX 'XX? you comments clarification Is XQ the XQ prepared guidance now months. there. that Does quarter than you take from about of your into runway say

Bill Marshall

that Jason? Jon, care have I'll take Yes. one. Jason of

JasonLeverone

the Yes, Jason. early, XXXX. into Jon, takes quarter of us is That Bill. Thanks, the this second

Jonathan Miller

Sure.

your raise? XX you're capital. ongoing months talking plan Do possibility have cash I plans for that you to non-dilutive the business Now look out this start to think changes of to forward can about runway, additional Servier it's cash of pharma? time Is your in likelihood development What's deals a runway? we there raising from additional large What or interest terms to milestones from year? of

Jason Leverone

Sure. the funds Obviously, to with capital our well from the company collaborations, say, need foundation additional historically, received sources, beyond we'll grants, borrowings the as collaboration stock, can of of next XX raise funded support, a combination including I operations fund our from Servier. sale to as we've months.

we the to company So use to the fund expect future. methods we'll these in continue

think XXXX, and of half I like pivotal a this with several, second have potential year readouts readout X additional upcoming company catalysts. we the we has clinical several feel the in the

Jonathan Miller

there. said a end track all Fair SOLAR to event? of fair taken Is The is that XQ April. delays a some going course, With be on ORRX. it second 'XX? enough. later? noticed that SOLAR still a there, of seems you you late really like little is to started point XXXX in I XXXX, together, say Are had enrolling for is that key It half we've

Bill Marshall

busily enrolling sites for Jon, actively question. opening are We've patients had, We're had now thanks globe. the the again, The, now, study. the across we've we enrolling. right in

really will deliver We updates experience expect provide with we in for we the when second the we're and half gain additional of could on as any zone enrollment XXXX. it. top still monitoring line track But on more that be that to now, in

Operator

of Liana Moussatos Wedbush next line question from comes Securities. the with Our

Liana Moussatos

CLL it indication market leapfrog get could Would get expansion first? something for Phase to biomarkers if CTCL, doesn't And get could I be And keloid data? point with for then the we end So next data or it cobomarsen to cobomarsen? else? for for released accelerated what Phase remlarsen scars could that the be I or what's approval MRG-XXX the

Bill Marshall

Thanks, Liana.

results looking data the of be the potential With going really view first around the for sort results question study us can approvals. this base was our placebo-controlled. timing those So We're that remlarsen, future is on. we double-blinded, for our to is, are planning give to

data timing guidance of the bit We're and the looking the focused over terms the on would size in for then and little drive that anticipate understanding discussions would provide going a effect also to be And and it's at so us early design of sort to and FDA. trial, when it. that really with the we

question, the for points around second was study believe, the, was The Liana? that, end I what

Liana Moussatos

The MRG-XXX, Phase I biomarkers.

Bill Marshall

Biomarkers, that's right.

biomarkers that targets biomarkers a confidence are that related, of give there that in direct seen what related of we've are some So to we've set us microRNA-XX of and including engagement. developed molecular formation, pharmacodynamic are vessel target terms

are vessel also can analyze in to methods we We speckle sort as we help also, events. a samples. And imaging biologic that be of well using to look future looking at of use as in we variety able technologies of, then laser can are image-based for formation biomarkers

then effects as the study as drug studies can indicate in can dynamic So complications additional or incisional well there's port then set the... as into failure for well opportunities whole as the a that other we markers and And hopefully that both burns. heart different of pharmacodynamics

Paul Rubin

something add I to that? Can

Bill Marshall

you comment? add to Paul, Sure. want another

Paul Rubin

literature mentioned, order suggesting or vessel there's specific with suggesting injuries accelerated Liana, laser that There's enhancing in healing. indications that with can that flow and/or we're accelerate of other blood or you're formation, to if the include that, flow, heart correlates new looking as angiogenesis you and burns require such failure as new document either wound that some complicated of at with data a actually efficacy. blood Doppler Bill markers blood

surrogates when Phase II. looking So in they become valid very you're

forward we could we our affect I from Phase can that actually because might will determine us both have believe but data derisk derive also kind blood some help trial going that doses in of which flow how we effect could this this doses select these we So of indications.

that very ultimately lot think relevant really I of that data a that be positive looking at. So there's we'd is

to a indication, be Bill markers would handicap that that's we're beneficial. and and can mentioned, whether As as burns looking something you as definitely these possible at at look this

trials marker of is LFUS we'll is be with well. scarring something as this in Another actin, muscle which measuring

can So program. Phase to II be and us really they very helping derisk useful design our in

Bill Marshall

And think around your indications. then, last was expansion I Liana, question

given the the and opportunity been We patients dire its in on obviously that have outcomes help focused data ATLL to disease. with

We of will patients. as active centers those from we by, DLBCL And remain expansion driven as have, usual, data-driven results new and it and CLL open the terms in event-driven identifying and be indications. adding additional and identify

Paul Rubin

set surgery be other existing you not in keloid. is the a that or at indication asked CTCL. are be probably thing patients because having for right an And the would for keloid for prevention think to would leapfrog I whether a could with we're unlikely keloid efficacy trial this now, that trial data approval correct

study. So it's prior CTCL can we unlikely that that to complete the

Operator

of Our Eun next question the comes line Yang Jefferies. from with

Eun Yang

I in a trial. have the question enrollment follow-up patient on the SOLAR

from about like looks are anticipating outcome, a would So the completed enrollment year be of primary based patient the today. it that on you kind

enrolling So it of enrollment with standard? XXX CTCL, kind the X-year about period, question to patients is you is is a industry that

Bill Marshall

Eun. The, us of of is follow and been lot work our we've on modeling the dynamics, that we've what have a originally done. created enrollment historical We this. that Thanks, previous studies based on allow to some based analytics assumptions done

out important that, of an the to report strategy to that can sites line of second us enrollment rapidly XXXX. we be the sites, expand And the here results to different to top by here. so real aspect are occur XX global was believe total One the to half our many focus the able areas really allow with of do

Eun Yang

is primary approval. accelerated Okay. And then lead point could met, it to the end if

bar efficacy significance? with primary approval, point is there kind statistical the accelerated for meeting end from aside an So of

Bill Marshall

with as be patients well discussions the this show is had So, to is But that aspect thanks, as with along and obviously, were place. that. measurements important patients, as statistical bar. the agent, to pain and to cobomarsen there superior. significance. need would Eun. specific coming FDA. to reduce physicians The going the the to is simply We've the powered these both The ability well. here of we've no study, component secondary set in specific we study pruritus And that points there's end designed be an It treating FDA, no to The in detect a the superiority ORRX bars need

Operator

next Our the question comes of Kumar from Madhu W. line with Baird. Robert

Madhu Kumar

first the one cobomarsen So is ATLL. in about

you trial? data expect this ATLL how So might in when you many more further next do plan indication? cobomarsen to recruit the to from and/or study steps this visibility on patients guys And

Bill Marshall

Thanks, Madhu.

that had patients So date. the we we explored about reported X to ATLL,

is in goals II the for I paradigm Phase design guidance our getting of the study the from trial understand creating and on best Phase then to really FDA. One the that

likely to the the we'll we focusing be lot hypothesis some So be Phase existing will patients we further the II response that on on and the our design of study. in drug, would durability and recruiting a then propose in hone on for patients additional test

be progressing at Moving with forward, updates we medical as and will will the study, provide usual, we meetings. future

Madhu Kumar

kind Okay. about on going MRG-XXX. then Jon's question And of back

based So the with how timing milestones is on kind collaboration for versus central designed, of releases is data Servier what Servier? from clinical the

Bill Marshall

through the next milestones is gated collaboration, release data. it Servier collaborative again, of agreement closely we of as entry the in the, that of of studies. terms are The project have standard we work with so fairly phase part into of in So driven a terms

the future we'll it then data of the Phase then initiation. much the essentially and that give then release their I correlate but sort with studies So would guidance not so on

Operator

question George B. from next FBR. Riley Zavoico with line of Our Instructions] the [Operator comes

George Zavoico

like for at thought responses preclinical with studies, looks such clinical the you got is or it as of ATLL the a in you've expect improvement can durable looking cancer since a good efficacy have the combination question the are My long of to lot in X might going combination, whether what really whether and And patients response. are all or stabilization. there I question about, regarding X trial enhance well. you of drugs, of clinical maybe indications some durability

Bill Marshall

George. Thanks,

have ATLL, actually we and So quite the, observed the, the in durable. in improvements are patients, improvements those

the the KiXX. I of we're which because aspect also of think this really an measure important, seeing is proliferation, are is biomarker important well-validated

that So in observed patients. reductions are, that, in many that we're have seeing been

and clone, the seeing are are prognostic indicator We indicative health in cancer's clone. the of in future actual is well; changes the which poor HLA, CDXX, a as which patients of then

seeing we improvements. are So

to looking continue patients, biomarkers on we move we forward. As treatment the additional treat things time additional will to be at to and continue

In terms really to therapies, so cobomarsen. date of preclinically a in, in of combination combinations far, at cobomarsen the we've systems in fair looking done for vitro potential amount with work

compound. cobomarsen's result to believe control, would act be there activity of we action does broad terms studies opportunities the monotherapy able Paul, you to don't pathway that are understand may in consider in goal if mechanism the real lead combinations that additional is I We, activity. have... the preclinical potential compound our to orthogonal is Given single-agent, of on to then combinations for we for based additional it that know really our where of then and

Paul Rubin

is to what's disease this induce George, remission. easy that about so interesting very it's

induces routinely remission. CHOP fact, In

time. that problem very the according isn't responses need a to induce last So are is there The not short to They per really a period experts, of durable. se, initial remission. our for

safety. in drug what years. drug few to isn't But remission, you drug, because now of are there. induction in we So if a the for might would The generally help add useful And terms life. need with be a this remission post for partial time, we're requirements or can really the is seeing is efficacy out initial have theoretically prolong now adding remission, when initial of need might reduce you response, X.X initial at that that for they months, you patients point the where last only a it

than So or example, that's CHOP, drug which the cycles. is obviously use for more X much X initial,

tolerability. this help response. the way reducing utility combination, sure in although is might like in sure the with look not for which right not Cytoxan initial to and there help I'm be terms things necessarily But I'm might need at of will Vincristine, it So some

George Zavoico

like really So is or that maintenance first be therapy it in a the it class. and different sounds any unmet there's clinical need This class, an there isn't available. would

Paul Rubin

Exactly, exactly.

George Zavoico

for drug First maintenance?

Paul Rubin

we're that's Yes, it. at how exactly looking

in fact compound agreed that's experts that at need a the there who Peru just this that HTLV-X that with really presented is we're to this And today. meeting In a is we in these for contention we that literally doing world's attended, patients. concept prolongs this life what hopeful the

George Zavoico

Based you're looks on presented, that months. XX way doing like to with just what on you it the

Bill Marshall

Thank you.

they lips your to say, You from God's know ears, what right?

Operator

like gentlemen, would closing call have session, the question-and-answer Marshall Bill for Ladies we turn reached the of and to I end back to the and remarks.

Bill Marshall

you. have we great for patients. Great. taking update an to for medicines bring want to afternoon. I your and miRagen Thank afternoon on time a as We life-changing hope support for everyone work thank the you to this Bye-bye.

Operator

This concludes today's conference.

may participation. at You lines Thank for this you your your disconnect time.

Viridian Therapeutics (VRDN) 10 May 192019 Q1 Earnings call transcript