Regulus Therapeutics (RGLS)

Allison Wey VP, IR and Corporate Communications
Joseph Hagan President and CEO
Timothy M. Wright Chief Research and Development Officer
Dan Chevallard CFO
Liana Moussatos Wedbush Securities
Alan Carr Needham & Company
Eric Schmidt Cowen and Company
Jennifer B. Riley FBR
Call transcript
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Good day, ladies and gentlemen, and welcome to the Regulus Therapeutics First Quarter 2018 Conference Call. At this time, all participants are in a listen-only mode. Later we'll conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this call is being recorded. I would now like to introduce your host for today's conference, Ms. Wey. Allison begin. may you Ma'am,

Allison Wey

you, Skylar. joining financial thank for everyone and Good to discuss afternoon us XXXX highlights. and Regulus' corporate you Thank first results quarter

for Dr. Jay We then today our Dan programs, financial share CFO. questions. opening the Officer, Hagan, progress Tim provide Chevallard, Wright, results, will will open Tim President and are review Dan our by the before and and remarks, joined Jay on R&D Chief pipeline line CEO, we will the

differ I expectations, a begin, those remind performance, that statements filings materially these forward-looking corporate the in we provision for to of those Before would result by statements Harbor you Safe statements from of under and Reform Litigation may Private indicated as this like forward-looking Securities the contain important the regarding SEC. our Act strategy, discussed the purposes which forward-looking factors, call various with plans, will constitute Regulus' including results of XXXX. future prospects, Actual

In upon to the relied will date disclaim date. obligations addition, Webcast specifically views statements. as such call views be this not represent of statements We and should now representing of any turn any of our any as to subsequent our I the over update as only forward-looking Jay.

Joseph Hagan

welcome our how changes appointed here with at CEO maturing earnings Thanks, call I a being since very implemented. and is everyone impressed we QX and to our with been Regulus Allison, now business update. year It's pipeline am the and

and for internal B pipeline disease. the several well targets progress. for have potential this we existing a our advance progress, selection INDs years. immunologic We we the us As formed namely to two over clinical Tim microRNA new along teams two programs validated two have pipeline genetic clinical With that filing. next two kidney pre-clinical new for talk exciting have flow new announced to diseases, Hepatitis as with programs toward of continue our new new been programs virus believe of targeting treatment these candidate where project positions IND later, these well to as will important Meanwhile, diseases programs

portion we initiated Phase As RGLSXXXX ascending study. X dose we week, the the of multiple announced last

to the in second prepared his shortly. half from Alport will expect programs in biopsy Tim continue pipeline this more We year. data program about the syndrome of share and the remarks

walk in the here but pleased plan all Additionally, will to we amidst in through Dan see the the progress financials, are managing QX pipeline. I'm the

continue the belief to intend turn finally, I it Kate Kate a program. Sempra with to very our to ADPKD to to I is our We opportunity where And our market fund Along a development with it payors that that significant project interest is experienced we there supports before Energy. wanted very those recently big our over aspiration to Diego, to at local business and have significant completed Board, leverage San development pipeline research welcome Ms. Collier. our and business makes financial in executive, most physicians sense. lines, a addition Tim, recent commercial the

We of pleased Board. Regulus someone are caliber add Tim? to the to her

Timothy M. Wright

Jay. Thanks

an with update begin RG-XXX syndrome. Alport program Let's on our for

Alport expanding efforts the studies HERA study. includes within improve and male to countries. is of also This biopsy adding the which significant We continue to studies. to U.S. population, more of accelerate syndrome visibility efforts our the to the proof-of-concept the focus our younger the sites HERA We've and recruitment made other of

that the anticipate second Phase from share I phase escalation in some to and now Realize ascending in study the you can completed XXXX. the in in we half volunteers. X information our the study follow-up follow-up the on dose RGLSXXXX completing preliminary ADPKD, healthy the program continues. are Moving with The we single dose in this subjects of study. planned

preliminary we the no pre-clinical dose top are line serious no toxicities. the thus there adverse The been study and pharmacokinetics reached limiting on events expected have and species. based dose First, blinded planned in far in as

levels pre-clinical has surpassed been are efficacy and consistent RGLSXXXX achieved finally, of and RGLSXXXX our And with plasma studies. that generally tolerated. well have We safe

I more when the of As the in second study have completes mentioned, half we will XXXX. data

anticipate exploratory announced to week, multiple an has interleaved dose MAD and dose the dose healthy safety, second, evaluate have As first, This be ADPKD to biomarkers there presenting evaluated which biomarkers component will with in in a the We tolerability, half last of comprehensive the initiated a We in data ascending we and X first safety, RGLSXXXX XXXX. volunteers; half this of the XXXX. forward patients. pharmacokinetics patients in escalation component our completion study parts; tolerability, study. at dose or multiple from and escalation in is MAD conference program in these PK, two Phase the look second of the the exploratory of component proof-of-mechanism

annual RGLSXXXX Association Renal in Denmark. later at month on data pre-clinical the additional presenting also this are meeting Copenhagen, We European

of pipeline; Now, have our in to include data generating led initiate recent a onto our These programs Hepatitis us areas. couple candidate and B pre-clinical clinical from programs immunology.

validation we now could clinic Given years be the two IND less in our enabling these timelines in from programs. activities, with from than to target

glioblastoma candidates our We the end clinical new have anticipate these also and exciting towards we for data NASH multiforme nominating and programs XXXX. of from programs,

significant resulting of like over would part call year's changes, for from turn to as progress, portfolio. in the now, the pre-clinical organizational large our yielding I to Dan is focus, the by update. new evidenced finance And Our expansion last

Dan Chevallard

Tim. Thanks

have plan. true research resources our representation between our programs As quarter, represents our Tim standpoint, with given organization, operating approximately research about operating cash discovery of our announced that mentioned, and this today, were Through of the was in resulting results XX% from and allocation financial efforts represent nearly the cash of excited emerging From programs, the and clinical XX%. very our our annual both which burn, versus burn Jay line and our research which a we first my have are platform. the our efficiency of we new promising

budgeted such payouts bonus As seen year. previous we that periodic resulting repeat quarter will first in not typically the corporate years, certain the includes as and cash again during events in have flows

This cash Phase $XX.X quarter as through X to campaign QX We and proof-of-mechanism. and with in ended GMP million manufacturing a cash equivalents. plan and towards support RGLSXXXX, also it included on advances

We have XXXX. continued to expect our quarter of to cash runway into first the extend

Our expenses given XXXX. in operating decrease total the occurred quarter XX% versus expected decrease that was last XXXX year. organizational mid for QX This changes reflected of the first

however, quarter Importantly consistent expenses with materially trailing second of of half the were XXXX. first that the quarters over operating our

X the of XXXX. primarily XXXX quarter This result quarter million and $XX.X personnel the R&D clinical of for costs program, for related were million compared first decrease million RGLSXXXX. the of offset XXXX. by of partially the was costs first $X $X.X expenses XX% to were quarter absence expenses of first quarter to costs Our million in in Phase G&A reduction a the in to XXXX a RG-XXX $XX.X first compared related

call the over equity our basic by both that quarter were first to loss in impacted down net share further our the of Finally, diluted, back per share, the Note share first July XXXX, decreased while quarter and per financing. per-share to I'll these XXXX turn loss results net approximately quarter from our first comparative $X.XX XX% Jay. in versus period, that, per XXXX. decreased the by With $X.XX XXXX

Joseph Hagan

lines? to happy questions. take Can you Operator, open Dan. please we're Thanks now the


question Joseph Partners. first with Instructions] Leerink [Operator from Our comes Schwartz

Your line is now open.

Unidentified Analyst

[Dagon] This for Joe. in [ph] is dialing

wanted and So, clarify. but say by guess The half, to two biopsy think HERA second going XXXX'. I to I just you study, just I ones. quick your release the I 'anticipated press says, renal heard year-end

them will you candidates but you with forward, to more around candidate guidance at where regards XXXX? Thanks. be an introduction just here, terms I'm point year or it provide to completion point strategy it you inflection enrollment and second throughout context can the you the in second of perhaps out or I could year-end or your picking license wondering hairs in guess some and capping the more this question, out? going advancing So, half either will sell And be advancing

Joseph Hagan

aspiration. any would the we're as indicated And and of have would we our year, of had when the Sure, we we yes. to giving internally. to not include last on business those would end research did guidance year, on that more by that Then call, biopsy programs, intend research by we on And so had either with completion enrollment, enrollment. two. second that a leverage the have of have we complete think the study, indicate alluded any to end we that question With would respect prepared without precise in fund as guided Dan HERA of also We more that, the be respect efficient which in the the year. your to precision our though development investors of my very comments, to update than advancing half, the the spend I alluded data to second half to, second I timing we XXX, on

we important advanced appropriate So, But the we unmet partner, we that's so for indicated, need Hep the instance as into potential a continue of kind program from had makes for advance and interest the that believe programs that generated to backup and we it with more to science various program, sense where haven't pursue C that's have potential of the partners. clinic.

we the idle. strength as these allocate to sit balance of modulate to resources want and leverage relative will and we on our then forward spend business sheet don't we they where ensure So development them to appropriately, move

Unidentified Analyst

the question Great. taking Thanks and that for congrats progress. on


Moussatos Our Liana next comes question with Securities. from Wedbush

now is line open. Your

Liana Moussatos

half year. of Do of the XXXX patients second any kind Phase mentioned design, You proof-of-concept with next on can in [indiscernible]? you of patients, start the have X number thoughts

Joseph Hagan

agencies you and that on thought Phase and about bit X forward we'll but we have give some thoughts with that. the Obviously Tim? want program, to can Tim that. discuss a the move early complete Yes,

Timothy M. Wright

agency meeting discussed thoughts at to. preliminary asked the pre-IND in meeting, design said the X both Sure. in Phase We as the for would agree actually well our which it as they on

anticipating and the more who guided accelerated X by Phase right more X/X in stage towards the So, we partly progressing forward and enrolling have study endpoint this rapidly that program, in and disease, CKD includes second early eGFR look that subjects change one-year a [indiscernible] patients. the or rapidly population we're success poor skewed was disease or showed discussion. is that more subjects But of the to now, progressing a

anticipating the in size. secondary, we look are an now have eGFR. but MRI at of doing are the exploratory that terms kidney sample some by to we a with volume endpoint of We study proof-of-concept, that and as design in total a firm don't commitment at more primarily looking at looking estimation So,

Liana Moussatos

you. Thank

program but are going are you of you know wondering, and thinking so mentioned of? [indiscernible] HBV in HBV new to Okay, aspect I a just was I what you

Joseph Hagan

last call, additional the [indiscernible] success had on us our we in we associated the the for think as we initiated with So disease based save indicated targeting I had isolated in with infectious some that other pathogenic infection. miR-XXX transport hyperbilirubinemia confidence of and mechanism could some we in literature and their disease, that of body infectious there a that pursue role we aid large effectively is gave the quite microRNAs on saw

what move Tim, towards a within And we've very led period about so to bit little now, doing us want looking seen that, success clinical time it? candidate. a you and to to of do and talk that's a more short now

Timothy M. Wright

actually an unbiased it prospectively internal complemented And libraries by the with collaboration for in a and but microRNAs we particular, of is have this the identified part there way screen finding guided an that with potential but that in we was novel literature previously, robust the targets. Sure. that diseases, mentioned because on HBV HBV as is our infectious we in HBV role a against uses of literature in CRO,

look because the identified two now right two and we right these to work far are a activating targets promising. of So, so that of success on approaches, different team these now we've very

of aspect the go other and at time. host targeting HBV, then detail aspects pathogenesis. factors, won't any have host the are in replication factors as virus on of impact these far virus this more and I As the the


from with next Needham. comes Our question Alan Carr

is Your open. line now

Alan Carr

how that MAD Phase healthy other can into is little a both after or going candidate bit, it two one? a I is you combination on think program a guess your different And this drugs, Tim, you just do that follow or to all [indiscernible]? volunteers unencumbered talk can [indiscernible] the were both Thanks. the you that trial, and a fitting one, And over that are more go how bit you thing, something X patients about you little again? targets

Joseph Hagan


And Alport first, Let target just Alan. unencumbered. Hep pipeline syndrome, me and one potential only is going middle then pipeline after thinking a portion? partnership B our maybe this as Sanofi little and hit of and with and rest efforts, MAD virus Tim, a thing about you including This the unencumbered. is the bit as hit well the miR-XXX in that's the The about could miR-XX talk is our strategies

Timothy M. Wright


B the that and And that now, Hep and hitting novel RG-XXX no see know, ever C, molecules is must because ultimate such are will if was out that Phase is you I you X currently X as As Hep analogues was a to was required it B on from be miR-XXX, difficult C cure some that there only therapies there nucleoside/nucleotide cure, that cure of market right and dose. clinic. out interferon XX what and are these a related angles. Phase and program, may a found for is target, C that's hearken the Hep there molecule in Hepatitis Hepatitis that combination was in But led are to other of information viruses eradicate Hep the for follows X. I'm be to host it that just our subjects multiple may back aware for C with to what therapy the in a it single and of

single So, of eradicate Those I tested that then one it that is and and than and two even target one maybe target is against may moving a molecule therapy. a both combinations reasons clinic, pre-clinically be know could is the with it forward in combination oligos, will a the targets. two possible B suspect single host I Hep possibility the ultimately require dual-specificity host don't more in host consider to of combining virus?

the ADPKD done is what incorporated So, the as that's B MAD, the Hep and answer patients. And volunteers far for we've we've cohorts of virus. staggered as healthy

picking after the of we're a patients follow-up I ADPKD the mentioned, up the level. period of interleaved escalating, volunteers, same an dose fashion at as So, in certain healthy

a imagine following ADPKD So, in multiple dose are we dose with a staggered repetition be that of healthy going cohort to given of in ascending can multiple a fashion and you strength volunteers the patients.

to does with us So, see and and it we establish basically biomarker then dose our to test is of levels as done proof allow what different we've whether dose mechanism, integrate dose-related changes ADPKD endpoints as parallel, collecting and those And those volunteers. in going cohorts, of information, are PK as healthy information the well which escalating. we'll be is safety be of primary to patients the

Alan Carr


up targets one at and wrap both, so you host you might after host the will you pre-clinically more like point, Just you good about to a have should HBV, this is be evaluate if possibility part you two targets? that which going there more find or

Joseph Hagan

still Yes, we screening. are

you, encouraging, to still find can up a process, has so we're the I have far So, we data in therefore But so may first very this the been others. forward. in we tell and what team dedicated two we a picked move

Alan Carr

question. my Thanks for taking great. Okay,


next Our from comes Cowen. [ph] question Schmidt] [Eric with

Your open. now line is

Eric Schmidt

have a half? that study, the see published? study one the you expect Should do that a we specific in when history we might Just timeline second still regarding natural quick ATHENA on

Joseph Hagan

actually the are presentations from we closely of because investigators Yes, are data. obviously analysis. I of playing and in terms a with big out the very of role Part actually. of is our data it a working the And result to investigators expect series publications hands


B. next with comes Our from question Riley. Kumar Madhu

Your is now open. line


on Jennifer Madhu. is for This

looking second arm And you trial might studies battlement X of such recoupment you date and RG-XXX is, Reata clear? Phase this created from you a could the you upcoming biomarkers key in of data, to do measures how has please patient pre-clinical think you us do are such expect studies? bottleneck extent when performed RG-XXX what renal thinking the those biomarkers fibrosis about think to as for in the question of First, the to and biopsy remind activity biopsy CARDINAL when are for the what

Joseph Hagan

Sue, to Tim of those them. on ask I'll help some take and out

presents familiarity other. competing versus a it's in in one as obviously and to flow indicated all I'll we've and may past. trials much not that study As patients, on rare a indicated maybe in to the in nephrology ongoing that the very couple past, to of having to population There that a suggest molecule the they to patient competition we've But I'm for sure lend the that benefit obviously to is renal regard. Then that biopsy familiarity community respond company's based work, them that. respect ask with past with though some the study, Tim in

Timothy M. Wright


kidney reaching would component concentrations so these to in in situation kidney RG-XXX we're be kidney in there models. see studies. animal in say sampling of look seen to able of will to first – measure we give I them the that were the us if So, and achieving the the But focused compared as as our the a the concentrations will first extracting at be we're on models, that look the the what for, be therapeutic analysis was, the we needle that our to we're phase that have at the will in whole these the the confidence animals terminal look here concentrations also adjust parallel in animal well in kidney. that of biomarkers. And their kidney, work pre-clinical the we've that of with the are Realize first there's that biopsies study end at be as to levels

So, RG-XXX the confidence concentration give in we the in we achieving of part that the first are would at that is the us ensuring range. and therapeutic are looking kidney concentrations that

other in and oligos, in the target is with As not the the blood. tissue having the key oligo here

these usually hours, cleared. within in In over cleared are plasma XX period the the to and rapidly very oligos from deposited and are hours, XX largely of a oligos fact, tissues and bloodstream,

the measuring concentration kidney therefore, in so that is And critical.

well primates to to non-human I in relationship animal our measured see we be concentrations humans. as expect We in to the to mentioned, as dose concentration as guide us studies, have that able in

phase the is regimens. examine week month to dose different sampling one downstream. HERA one biopsy. a versus do One that there of by is in level us the could therapeutic regimen purpose dosing study, dosing is do dosing as achieves single is that the a whether that evaluating of other or to part The would allow adjustments in in a every that, to the in our other and chance other two every every after pivotal this month to is us study the And we're guide week some kidney RG-XXX

the As looking and miR-XX far at, are, key III collagens. and and us. before, in a We as biomarker we've I as biomarkers are for levels, in studies the these we miR-XX mentioned reductions did reductions see well as pre-clinical this type type in

single humans downstream engagement after month we related these that chronic directionality single As mentioned target are in type any target the the what disease-related a study. measurements efficacy after I hitting and will again, versus that confidence animal of the biomarkers study give in we type looking that collagen. only III the comparison months earlier, be such as humans many dosing animals, terms the changing are reducing that are at one it biomarkers measuring looking comparison a either to dose and us, not but But a month versus the and for studies end is at one dose in in doses magnitude I wouldn't four the or were or of after we're because fair of so made a


no showing closing back Jay I'd like over I'm questions. time, this turn the for to further At remarks. to call

Joseph Hagan

Regulus. Thanks appreciate and your for We time joining us support today. of Goodbye. everyone


thank you gentlemen, conclude participation does your program. in the conference. for This today's Ladies and

great now disconnect. may You day. have a Everyone