MGEN - Earnings call transcript Q1 2018 | Viridian Therapeutics, Inc.

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Operator

Good afternoon everyone, and welcome to the miRagen Therapeutics First Quarter 2018 Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. And as a reminder, today's call is being recorded. And now I'd like to turn the conference over to Luke Heagle from W2O Pure. sir. ahead, go Please

Luke Heagle

good and afternoon, you, everyone. Thank

and call Officer, Bill the Vice of Paul today Adam Jason are Executive Chief President Rubin; Development, and and President Research Business Marshall; Executive Officer, Levy. miRagen's Leverone; On 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 fund clinical XXXX. had stage refinancing that two strong of of a start Thanks, into of us the for year, for XXXX. We the programs the company early introduction afternoon, you initiation ophthalmology Luke. our Good completion for new joining to thank quarter helps corporate update and first call trials, with preclinical the

miRagen to position potential in need. confidence patients RNA-targeted to and bring We therapies, continue advance, de-risk therapies our pipeline to of in RNA-targeted high uniquely making build

on initiated partners platform potential an and collaboratively part candidates valuable for who two X only First, own represents of expertise our our with in MRG-XXX our but with partner, applications we resources. bring develop extend of strategy product important the Servier. our not to This trials to collaboration clinical can recently Phase

week large data microRNA-XXX/XX/XXX and We Also is new role of has patients RVO for we also of RVO, a been by we candidate mimics gradual shown presented preclinical the as XXXX potential unveiled which preclinical work Association eye. in of of characterized the hair play advancing of neurosensory a a of and a cells, important power pathological treat areas the Last and evaluating microRNA MRG-XXX, to the associated Ophthalmology control preclinical at leverage and loss establishment commitment hearing. impact from to need microRNA-XXX potential retinal with annually at are unmet or therapeutic eventual to thousands maintenance mimics cluster, and which process as of our data mimic blindness. photoreceptors Vision including cells such These potentially presented medical fibrotic to vision loss. address of the photoreceptors biology microRNA-XX highlight pathological Annual our program conditions biological use that in degeneration, in unique Research programs with the condition meeting, cluster, for in the for an

lymphoma common cobomarsen most Our initiate to to or on a SOLAR microRNA-XXX MRG-XXX, cobomarsen the half hematological most II advanced XXXX. treat the the as in of a second Phase continues form remain track cutaneous advance. exploring which CTCL, or trial inhibitor program of We patients to therapeutic malignancies evaluating with is strategy in T-cell

active infusion versus we the given an by reminder, a anticipate control. and of evaluate of safety will As milligram intravenous the XXX efficacy cobomarsen trial

least patient's of greater endpoint of entire for response the disease enroll over patient's at We a overall a months, expect progression disease treatment four per approximately ORRX improvement rate of believe or XX% skin patients lymph or severity to with for in in Phase no to FDA outcome consecutive the accelerated of could for evidence the group. Based on discussions the released X meeting observations apply to durable XX we United CTCL, quarter. the cobomarsen blood, States. with primary body additional successful maintained quality our results approval highlighting of study We nodes allow the additional at in patient's of us an improvements upcoming potentially results ongoing clinical life. viscera this from in release in Yesterday we plan medical

lymphoma, are diffused within chronic leukemia. evaluating the lymphoma X and adult T-cell also indications in three current other lymphocytic large cobomarsen We Phase trial, B-cell leukemia

We data least half in at second indications of of the expect to XXXX. X Phase interim in release one these expansion

half first randomized in initiate formation. double in to keloid the pre-disposition a subjects for MRG-XXX evaluate a intend clinical Turning a trial to of to XXXX blinded we MRG-XXX, II Phase with

showed to It that of need. other are candidate. various routes a corneal target in to potential potential collagens MRG-XXX may provide therapeutic of in agent of demonstrates important presented expression it that microRNA-XX product of investigating genes the RVO inhibit data studies of options MRG-XXX that and fibrosis. preclinical MRG-XXX for We at or retinal preclinical this patients in data ocular as retinal the fibrosis. the have through MRG-XXX of possible The and and Recently delivery also preclinical these the that therapeutic treatment treatment ocular lung reduced suffering multiple fiber and uptake also from limited Patients in inhibit benefit administration MRG-XXX demonstrated may utility functional fibrosis genesis, study suggesting we indicating function resulted fibrosis. corneal to

mimics fibrosis mirror evaluating in-vivo lung treatment release to XX the also preclinical data are We year. of expect later this and for

models trial with of MRG-XXX under is the intended candidate initiated to note third that product failure. could eventual to In March treatment MRG-XXX that lead MRG-XXX turning candidate trial our XX. that Under our systemic to of trials. potential enter XX Phase X to of additional In clinical of resulted subjects support human clinical of safety microRNA-XX functional Now trial. improved our for terms and Servier earned a with tolerability being first improved X XX In the trial MRG-XXX, clinical Miragen their Servier, studies in million in the vessels for XXXX an of is €X The the in use male Phase clinical and to to is its vacuolization which agreement allow in evaluated heart happy and aged blood dosing collaboration dosing patient Servier, the preclinical MRG-XXX milestone new enroll I'm outcomes. inhibitor for of protocol. accelerated a the expected formation product the

MRG-XXX vascularization. therapies are even in a advancing healing wound, points. healing We tolerability of healing measure injection we intended end preclinical and exploratory We also healthy pharmacodynamic in of after or clinical Healing wound a this The induced reducing year as controls significant volunteers or The collaboration accelerate intradermal lacerations standard as to we for initiated We findings need other that healthy of variety in healing believe presented annual these MRG-XXX to May the to of in condition. in MRG-XXX important of result clinical wounds variety through to MRG-XXX decided to pipeline pharmacokinetics received molecular second herniation care is We wounds. Based also trial data focus and product of severe with care. that therapeutic can surgical patients to marker injections of trial MRG-XXX its while significant studies our study to in progress Showing failure. forward to continuing the during advancing morbidities, Such also there blood our placebo microRNA including of flow acceleration pathological to at infection, to into In use X delay In organizational death. information Phase is Phase candidate, further bio-up a for on or changes, support recently difficult summary, potential for treated on an advancing maintain biomarker the chronic Society safety rate. of intended obtained indications evaluate wounds in trial later of skin clinical compared for some lead treatment observed the treat look time and meeting. in potential candidate designed and a X the made such we infusions, and these The this new Wound is the to of for evaluate XXXX. cobomarsen lack both MRG-XXX heart biopsy. insufficient believe because program. and our

We now to will financial that a the are With discovery I important and target positioned represents therapies unmet our approach about platform over need Jason Officer, have Chief well call RNA on Jason? that RNA believe opportunities review focus a the leader Financial turn that, to today. be the an earlier We excited variety reported and results is the several field we development to in fronts. conditions in could therapeutic Leverone, with provide. miRagen medical of

Jason Leverone

appreciate the I financial Thank good our you quarter to And everyone. XXXX results. first afternoon Bill. opportunity provide

our year, had to Bill a made clinical our Servier in development we important MRG-XXX first the As advances we with its which the with agreement miRagen Servier. partner under earnings forward look development milestone MRG-XXX an of mentioned to been led to strong of our developing Servier for Servier we candidate, has number during product important with to and partner quarter. together stage years the miRagen now a third

quarter cash first position, in approximately XXXX. the quarter used including million of $X.X believe net we our operations we proceeds million quarter February. our was will early the in quarter, $XX.X sufficient our cash current public $XX.X $X.X current ended XXXX. the Based cash first be Net with XXXX financials, for offering million to the equivalents, Turning our into and from operations compared fund resources on to to cash our in for in million

we in Turning million The to to due under million agreement first milestone the with our total and the quarter. during Servier. million to the earned we recognized recognized in during XXXX. compared of quarter revenue primarily was $X.X the revenue $X.X revenue increase income statement, as This $X.X

$X.X recognized as the Overall, advance us an development to and reimbursable clinical additional we XX $X.X by during personnel increase development quarter. of months. team to the in million for and to and primarily this The R&D revenue added development We quarter our into as for to Servier higher increase expenses development was quarter also the MRG-XXX due increase research to first research the research expenses million activity, related in last over to we XXXX. compared cost

higher last related ask over XX the quarter in and related months. charges operator team merger administrative by compared expanded during higher XXXX, first the when Operator? This offset questions. decrease general this personnel at and to costs we I'll incurred call for fees to the the open clinical compared million G&A primarily quarter, development our We license professional to three and expenses first million programs. in now for to General for technology higher XXXX. decrease stage fees quarter were $X.X lower was support The expenses clinical the our administrative expenses that, XXXX. to partially was $X.X due incurred we With of as of also the

Operator

[Operator you. Miller first we'll Jonathan Thank Evercore Instructions] ISI. at to And go

Jonathan Miller

Hi about guys. want taking the starting. that little you're bit a trial MRG-XXX Thanks in on for fibrosis I question. to ask planning the

what keloid setting, the is Secondly how where how need the timeline has be us centers should some development In patients expectation confusion expect few do you over about for different think about past they and you fibrosis there trial? there for years that enrollment patients, ocular exactly, the over on can are? effects way. the formulation your difference What are we been how there tell For to you'll the many of many MRG-XXX. that on than formulation the how that the

William Marshall

questions. thanks Great. the Hey for Jonathan,

recruitment conversations a We've in lot Phase to in So folks spent will around been patients. we key And we with first opinion the databases have centers of initiate patients with the leaders of those large with the treat able front, on that in in patients Keloids. several sites country II the numbers the the in of and them. identify time trial that keloid relatively

biopsies designed looking formers. the we at study of control in For analysis study very we've where powerful a inter-patient keloid are

comparing sufficiently do can we we And analysis that we we desire relatively positioned the with lead work are have power to to on patients. onboard sort this to trial to number usual outcomes on are and problem. powering a pretreatment versus able see point the do within because it at without and patient recruit we of and where small to do the this it feel placebo well post-treatment centers be that able So we're we with to treatment of

occurs date ocular looking on on conditions. with extensively we in both of explored been we've the of what simple reported the intravitreal that And is MRG-XXX administration RVO what of cornea done in the as And both we've terms conditions subconjuctival second in fairly as formulations and the of and the treatment is at we the In effects intravitreal have administration. again injection cornea. retina what at well of treatment the to MRG-XXX rather retinal question and to end that evolved in preclinical fibrosis fibrosis, focused subconjunctival the studies

biomarker we mentioned potentially consistent do are I As having see a this with clinical changes good biodistribution, benefit. that

thing that a to of we've is sort the looked third the there at was barrier some The break conditions eye. where in

So the show distribution what in were impact the this that compound borne we is compromises the we [ph] eye. are quite in nicely an looking them the able in was leads even administration topical which case burn conditions compound of at to eye. And that to the and activity out

may keep working commensurate the eye that in different dose would in formulation, looking So wettability we and where for compound or to in in the indications if versus of cut damage likely eye, would some form But case the the eye be have at major or and differences the alternations obviously in concentration we eye. be other a in traumatic place sort applications it can applications increase formulations to that MRG-XXX. certain of the potential would in dose viscosity identify towards either or we no

Jonathan Miller

Thanks. Great.

sticking broad there. blindness eye disorders. are For is competitor do of of you How How blindness progressive application the other and looking preclinical the with view modulations the with family? progressive programs, this also the programs mirror at overlap that

William Marshall

a great it's Jonathan thanks So question.

lot of the set genetic is of here. to really So that is there this correlations that a one particular microRNAs us attracted things of

also certain genes, to phenotypes, lead certain both microRNAs point mutations are believe that that is are that of this biology set and that's really hearing in vision loss to of loss particular approaches us this as animal specific of a give vision deletion will this opposed direction where and to interested other generation and between to patients. we we're are both the with than in in There of human from examples us these microRNAs microRNA models What very modulating in will likely of and really regime a important genes in approaches of power important in inducers at to vision in genetics of approaches of loss. looking potential lead the issues pathway of either the loss. So greater and maintenance also downstream particular the complementarity benefits application broader some in in

Jonathan Miller

much. Great thanks very

William Marshall

you. Thank

Operator

WedBush. next go we'll And to Liana Moussatos with

Liana Moussatos

of the for there in which in to - you you on is think likely second you question. from your most Thank Congratulations all do having see least that my the and you forward half on? to expansion data order progress. of mentioned one In to taking one the What one the you at at least one be report do hope move indication indications.

William Marshall

these in the database indications treated expansion with and milligram reporting what patients is again focused Liana. XXX in been Thanks continuing questions again we've treated infusions. we being patients Great our on each out. with of anticipate We've safety build IV the addition What conditions. to to are

on move then disease the we're we will alterations are lymph, so counts, we're populations. history that in be alterations trajectory these alterations of the the patient interesting looking that see same of progression time in able disease general treating now to as observations the obviously are changes being the in the for in based going blood based and patient's on looking treatments safety database forward. progressive more certainly the the history observations patients in disease on in for build are progression to for So looking At we're additional sort looking we're we the the of of natural for

and in the be indications diseases the there There selected microRNA-XXX XXX studies preclinical the that of and as be these the three will that we terms We've of indications of indications likely by shown microRNA-XXX cooax would elevation. reason because of to have any preclinical We're actually opportunity stratifying virus a on that is the advancement. good its DNA those apoptosis, we've sort a studied progression happens promising intrigued done cells probabilities induced lymph virus. native leukemia as and any signaling well life data That the is that's elevated. leads human on in supports advance been is proliferative - In resistant in to tropic based well. of cancers ATLL this on other by we the lymphoma believe in newer this in is cycle effects own what mechanisms to significant say repair HTLVX effects

we'll to and on half applications natural focused of treat There in It's the altering would second blood cell and So a we looking I that really characteristics the results it. relevant that. of like good intensively three to to the relative available of in are no profiles, disease. begin difficult we're the very terms particularly all this treatments in for lymph condition. said report But be history are at interested

Liana Moussatos

Thank you.

William Marshall

Thank you.

Operator

[Operator at Instructions] Wang Yun to And we'll go Jefferies. next

Unidentified Analyst

Hi, Thanks this is in question Suzy for calling taking my questions, I cobomarsen. about Yun. then for

accelerated ORRX question based that cluster if other is have on something or other treatment? get the for wondering or just approval And be any to would my chronically you on it XXX time one just is there Just endpoints? is

William Marshall

great Suzy, questions. Thanks

endpoint, ORRX. with primary on the will on its on the from based by received our primary obviously ORRX the the as guidance agency discussions the could own alone. that So accelerated in our FDA recent them front, review that be of cobomarsen we endpoint meeting, endpoint and approval based results results on a Based on primary

endpoints. at looking also will We additional be

and endpoint the looking gotten that Phase the those compound data And free These that sort and result released progression the tumor does has as the reported accelerated show free could and scores. progression and could accelerated result in Yesterday, primary would at we the the approval, the on outcome believe patient done Another be out reductions as are if slot pain key survival. from endpoint will itching forward. outcomes. between endpoint patient continue secondary to first reported out patient that we that X pain then both of pruritus is tumors. would in alone advice will exploratory be we'll basis, reports endpoints be the the important severity the analysis full survivals, of to-date in the And approval out at move a an then read we in in and on the the and tools that also correlation ORRX, which time potential reductions M Skindex-XX we've secondary

endpoint that amount endpoint weaned this mutations primary a secondary I accelerated systems. a vision. the model so into you approval we then data to a single light. could understand a this of full free sort a be So be system point, allowing administration The survival the amount would really ORRX, move for for time. far has And would if at a say will limited PFS, are do These of then XXX, rapid allow animals in have have The progression in There in we they regime. don't of that potentially approval. when receptor data sufficient dark have in the been a where is model deterioration are in are so

The a relatively and mechanism see low a duration. conduct is But which they long visual saw on compartment the the amount going the we pretty get and [indiscernible] interesting there able with it one residence acuity. eye to eye because acid which based content of dosing blunt experiments. pretty that we're was the to is reduction a therapeutics degraded. distribution we time what out turns So is in has in the for nucleus nucleic by some additional We're

infrequent whether believe would answer multiple following on that treatment occur I we But can't not if sufficient. a the that necessary, relatively So today of basis. be or acute could question would an really injections be

Unidentified Analyst

Thank you.

William Marshall

you. Thank

Operator

next We'll at Madhu go B to Kumar Reilly FBR.

Madhu Kumar

for thanks taking my guys, hi question. Hello,

really sets two of So questions.

that on us in Cobomarsen one the relates that trial and free First like you the progression the kind in when to SOLAR perform secondly ORRX to [ph] it to what timing and you CTCL and survival? basically and of trial, comparator kinetics therapeutic expect trial, we be do get about how treat metrics of terms does X SOLAR thinking of response, occur? remind see start-up response [indiscernible] could in ORRX does how how the and And then drug inform how Phase long would does you the

William Marshall

questions Great Madhu. Thanks usual. Great. as

trial So the - address so the SOLAR and agents. comparator let me the

provision have what comparator the agents three So was analyzed if it, these discussed of XX%. [indiscernible] been reported active We controls ORRX the observation they ORR. methotrexate, It on month. raised wasn't on with and think so three secondary really there about FDA a recently based the were [indiscernible] and all methotrexate we observations we as [indiscernible] there of that studies methotrexate was In studies. we [indiscernible]. about an month to potential progression-free the somewhere clinical fact the were those X% ORRX were reported. know about between endpoint, from previous reports outcomes there was and reported not XX% the XX% And controlled months ORR, the in case were objective XX% the trials I the survival, the with and X.X as control X.X four about for What from [indiscernible] in believe that durability

powering modeled on we potential So three agents. the the SOLAR trial those of based really

we what and will yet based will the guidance will comparator of be soon on will We on selection on agent do those are as we so as three controlled in the a one guided but comparison be rezoning FDA. it it haven't single but be from

sort we kinetics, response of the responses question as second that and XX cobomarsen treatment. after X Phase initiation of as the the seen around soon onset have days The so,

draw time is product fully until there. time prior seeing a on score of We this we initiation say. a But I to The led in SOLAR seen would the time. us months. gain the keep deepening really that specific stipulate of treatment file the appear we of the we over longer period the to on to don't drug, really response would that have between that of severity has later intend responsiveness. at to to best of than the course responses candidate trial the tend a traction course This length we responses in be several over treat see mSWAT the on we the which correlation patients The has see of can months would six treatment factor and generally patients other -

definition we that patients are score as they clock, able positive if achieve that the XX% maintain they achieve mSWAT than that as the to is come in monitor ORRX's we know well that long and the the four they to to reduction response the month, as a analysis. once in can you As start skin, on drug for response

fair a time initiation is for heterogeneity like mSWAT there correlated be severity appears I really to of said the So in score of of with amount response and treatment. to it

Madhu Kumar

Great, much. thanks very

William Marshall

Madhu. Thanks

Operator

It turn over sir. I'll we additional to Marshall, back looks have things at questions like time, this you no Dr.

William Marshall

Great, time today track our well your for thank for continuing you progress. and very to much

the our reach We patients the you are and to advance pipeline could that this and continue a please forward proud us, work any you of our out look if you benefit afternoon. We towards great Thank year to questions. we achievements additional ahead year as have in informed diligently thus future. we have to in to free far feel outcomes will keeping and

Operator

gentlemen, us Again, and everyone today's like does joining today. ladies conclude I'd once And to that again thank conference. for

Viridian Therapeutics (VRDN) 9 May 182018 Q1 Earnings call transcript