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Regulus Therapeutics (RGLS)

Participants
Cris Calsada Chief Financial Officer
Jay Hagan President & Chief Executive Officer
Denis Drygin Chief Scientific Officer
Shveta Dighe Wedbush Securities
Yanan Zhu Wells Fargo
Call transcript
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Operator

Ladies and gentlemen, thank you for standing by and welcome to the Regulus Therapeutics 2021 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speaker’s presentation, there will be question-and-answer session. [Operator Instructions] And please be advised that today's conference is being recorded. [Operator Instructions] I would now like to hand the conference over to Cris Calsada. you. Thank ahead. go Please

Cris Calsada

and First for and Quarter everyone, discuss Corporate us Regulus Therapeutics XXXX thank afternoon joining you to Highlights. Good Financial Results

and program Denis we for on Executive remarks Jay results and me today's before on is open will opening Hagan, Joining questions. our review progress ADPKD financial call will provide line Officer; I Chief Drygin, President and share Scientific and Officer. the Jay Chief the

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

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

Jay Hagan

data compared treatment preliminary data kidney human drug from study. patients We as proof mechanism QX target mechanism Phase patients in were completed Thanks to by ongoing greater hits we the XX%, of increased We shared in the initial we measured study business this were available of in the demonstrate dosing ADPKD. X short and Cris, announce from and duration our respectively earnings X very designed. and action miR-XX showing levels in Xb the top to the because these study the and end of who baseline successful levels welcome that update. of everyone cohort with of XX% that our first pleased and follow-up believe to set, call Last those week results of line in RGLSXXXX data polycystin than

trends recall the completed indicated patient study weeks. remaining the may the we release or that patients had the follow-up encouraging. eight ninth them with the analysis very with and overall through That dosing share any ninth press we'll of to end of couple day of next And patient's be XX You the complete were have material in updates, anticipated we as that in nine sure you. data

the on remaining sustained are mutational suggesting with increasing having over less have may drivers dosing; both PCX well of bind overall the is and a of increased at the in of line Approximately status We reported models the understanding be June contribute are and rates. ADPKD are top linked proteins after levels genes polycystin frequent the to saw also XX% gene of presentation PKDX could that data patient mutation believed miR-XX response planning severity we PKDX inversely treatment resultant mutation and in to XX% patients leads RGLSXXXX more believe validating and are Measured in compelling of and design of new detailed scientific data disease the X PKDX earlier the as impacts a further dosing as to the to directly the with underlying showed late PKD the that levels X. then for Recall genetic animal expression of in to one with of Connect gene. impact year utilized. in what Understanding been to with with This in disease. an this correlate shown genes disease. of of these be PCX have the time the encode kidney. and preclinical the to X molecule we completion month a that PCX PKDX trend The polycystin effect

the miR-XX, biomarkers. has Additionally, two binding for predicted contributing predictive PKDX the gene site binding response one gene the to has differential between PKDX rates while sites, potentially

safety nine healthy were was RGLSXXXX of patients all effective in plasma exposure milliliter, well front, serious PK patients this adverse reported two greater profile All the per in with Overall, drug Concentrations desired organ generally events observed by approximately and the Cmax disease, volunteers healthy where reported. relative the US no suggests end-stage the it were study. are kidney of is doses a that in RGLSXXXX mild in the target similar Cmax was nature. the with alone. of tolerated AEs patients milliliter, to in the transient micrograms of On prior fourth to in And leading volunteer of cause in be the per micrograms renal there is in XXX,XXX disease. approximately the the three achieving In lower may diagnosed which that approximately interest.

as demonstrated preferentially primary options. expression collecting cysts. duct-derived technology the evolved A by from gene. reduces PKDX by Treatment in of mutation their the for genes Alnylam a multiple that either and preclinical of epithelium, kidney caused its kidney with proprietary the There's polycystin-X strong new targeting need eventually specific only and and and their end-stage X, PKDX cyst foundational proteins models to the to recently develop the leads well a as a functions from polycystin-X the mentioned inception of age proteins to disease. ADPKD X, increasing RGLSXXXX and PKDX I and we growth of approved In kidney develop and eventually distributes cysts, kidney to levels, function, pointing failure. to miR-XX disease, the and dialysis encoded renal ADPKD ADPKD and patients leads these importantly has US multiple Ionis therapy of As in proliferation is requiring inhibits down human mutation normal which or technology. kidney X disrupts encoded the treatment the Regulus models, have one their either of earlier cultures slows the XX% cyst RGLSXXXX or most ADPKD excessive in formation genes of mouse of in transplant XX.

body weight both and treatment reduced untreated of kidney through mice, this disease health weight to both Additionally, injury markers drug signs models. compared are with in improved kidney kidney and these

year-end These program next-generation clinic family RGLSXXXX, disease in two as compounds: molecule is the week; lead early ADPKD which mouse upregulated models being is of the be consists as as to the which with study is and thing of and last IND-ready which the Xb our that reported Our QX the tested both towards Phase are moving well the in as ongoing should humans well inhibits with the of next year. around or the molecule, of miR-XX microRNAs, do molecules designed function disease. same is in

Institute that are call to Dr. on requirements scientific Cris, as very as look the would platform our data research us exciting we results hold Board permit company's Alice engaging welcome summary, over and help in with cohort, the to of earlier-stage Phase also programs and Biology pleased direct in the these remaining X the well some forward to discovery Biologic recent turn Board Huang Technology. from of The California FDA the efforts. Directors, I with Huang. And to the XXXX development to the background to before of Dr. laboratory. So into this Associate to is advance wanted addition I an the summer finally, Huang and Engineering later move Dr. our beyond. back brings our extensive Regulus technology and currently at team continues Faculty at second drug to Senior

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

Cris Calsada

Jay. Thanks,

results. Turning to financial our

$XX.X totaled at cash QX balance the $X.X million end than million cash of at QX end Our $XX.X million more which is of approximately XXXX, the XXXX. our balance

extend $X.X cash reflect operating for compared million reflect in loss and QX QX million $X.X $X.X preclinical loss with general QX and were costs. XXXX. clinical first quarter XXXX first through XXXX to of for and QX These the ongoing business for XXXX the costs million. and million quarter our million, expenses XXXX substantially were with These the QX consistent similar the $X quarter net our pipeline. personnel-related and Net amounts expect first $X.X of for was XXXX. R&D for XXXX XXXX. associated of runway advancing G&A $X.X million to QX internal external at XXXX amounts We expenses

per first quarter XXXX. Jay. share the a share basic XXXX, a back from Our I basic of of that in the With on call net first both diluted loss to and quarter net in the per $X.XX loss basis basis will and a $X.XX to on share down both over decreased per turn diluted share per

Jay Hagan

to Cris. now. Thanks, open can the Operator, questions your lines? you We're happy take

Operator

Instructions] We have [Operator first Dighe. sir. Shveta you, question Thank from our

is line Your open.

Shveta Dighe

Liana taking for Hi. my for question. Thank Moussatos. This you Shveta is

and address a reaction But data achieved that and for cohort with objective the it? establish the establishing biomarkers. you proof-of-concept around to thoughts negative the company's RGLSXXXX data, Can hold safety had stock PK of one without. provide So some the data main clinical post the

Jay Hagan

because that we biomarkers, be win might six were Sure. setting what Yes, investors, the duration, Shveta. of thought meaning spent of expectations we this a saw is dosing. well. going we very Frankly we if number set, in significant amount think a as with just of see, that time by a and data treatment puzzled would weeks into short positive we've institutional We describe trends a that, for

the us both PCX and based And dosing, that proof-of-concept the target suggest demonstrating to we expect was comments, be that so and just continue the my alluded as PCX we'll on would up. which in in encouraging conference, to see you PKD going in with the to trajectory, that magnitude very would to of change saw Connect and hitting it continued at I sharing

XX. think evidence pharmacodynamic higher known be study the dosing. advantageous could days half-life recorded which two could at half-life the I of the frequent weeks mentioned would a after markers, approximately from both completion dosing achieved a of was so that more we at full day and XX was suggests kidney were understanding of standpoint, And levels setting. kidney be day is We've in up that a human less in at commercial XX, what the which end than and also highest also I obviously going this that utilized, in supports this the much anticipate

Shveta Dighe

It. the Got meeting on FDA we from follow-up question. expect just I clinical the can updates one And When hold? have

Jay Hagan

Yes.

plan to we reach So to them. out

experience our in areas in seems of us, focused cooperative unmet with I they've on very sponsors, them the think said with innovate need. helping division we've significant this and been past,

we this the process out a we be informal want to get feedback. requirements, should them to better to because plan their formal, on And understand, reach initially of them hold engaging so to remaining

of that, through with route a latter meeting submitting to a prepared So it time get a could either be a book be And A meeting. In follow Type informal Type once we'd the request. route. A at be we'll or we briefing either the meeting,

will So meeting of book. to materialize why XX Type upon route. modeling we've understand timing our potential that's to guided busy and sort Per these team a A it. that days the a now touches finalizing the on finalizing they is with PDUFA of request The briefing once we FDA. have grant with data interaction mid-summer a And

a And set sort time. so you're on clock at that of

Shveta Dighe

you. Got it. Thank

Operator

have question Chen next We our from Yi [ph].

open. line Your is

Unidentified Analyst

the expression questions. Hi. taking which my in first is for disease with achieve patients. of to deliver mutation, PKDX gene XX% improvement? Thank RGLSXXXX improvement represents the for question does matter PCX how My total ADPKD Does it much you order for the patients

Jay Hagan

in the the could because increase in less that PCX in PKDX than PCX they mutation in One would be have don't gene. hypothesize a need

Mayo shared already the these -- our these for with all Es, and scientific that advanced we're mutation these likely which with those mutational enthusiastic obviously, anticipated data disease Ds, disease or community. all all consultants mutation. And status, that we've some results. understanding so, classification they're patients the in the aggressive And in nine is equally the the And they gene results their probably more and and have of patients key nearly Cs, being with given a reported PKDX PKDX the advanced that more of were

Unidentified Analyst

in actually point improving still doesn't improve the end right? symptoms the the doesn't for PCX possible statistical with much, XXXX expression of significance the it that the mutation, achieving terms drug even that So, patient's increase PKDX achieve gene is can -- that

Jay Hagan

Yes.

Here's answer how I that, would Yi.

news some that trends with cured. So through to said, achieved the to statistical where subjects. biomarkers we've PCX first-in-human increase this little to go we're we'd doses efficacy have significant as adding Reaching that to continued well, a do. continued disconnect we're that's remember, give they're hope be with downstream great though, having PCX XX% to got soon, how up animal a or the more a gene pioneering hitting the demonstrates have target is, can target I treatment. in expect go obviously, we've we target going of genetic chronic four, in six, it's work and treatment, anticipate continue this And that and is associated we and does mechanism. And as or benefit. up the not observed we with that this study we're and subsequent And done models. the much as is here significance changes with to is that clinical this both -- eight biomarker designed it the dosing microRNA data There exciting is molecule share in to Where first-in-class have a

Unidentified Analyst

lead to indicating, in So, how benefit? much levels see PCX increase can and/or of how soon to PCX clinical can the we results expect

Jay Hagan

Yes.

And polycystin-X. the And cystic as at basically and the disease know that, PKD preclinical strategic hit including PKDX all as and PKDX on the which potential We protein and of is the validation, well preclinical novel of plan that cyst with disease. mechanism So, as and data We and expansion see Connect, expression. with those view increased count of associated increased all some What data. XXXX, to to the well levels is the these polycystin-X we trends, models. partners size of with the this address in as our arresting target. see in we our compelling additional animal associated reduction in quite -- we present advisers is

NGAL have to to does we where too it's that I one disease. health goes treatment a shared we GFR, study kidney -- have program eight to to levels say the II beg it it of in in a models of with GFR so NGAL level. patient calculate XX this to more that, the change volume as of do I which of nanograms kidney the total damage to of after up range, as markers to duration of that or those which remind have We week markers twice this. to quite well also to much those we significant take the upper limit of And kidney kidney some although we want how obviously did after and likely and animal patients it And the change view marker the advance total in though normal would within efficacy per to each earlier measure volume see mil. with kidney or a as statistically in just had or reduction might Phase last particularly we as back saw for volume, in is kinds understand, of that in study it. in short-duration well nine normal nearly X demonstrate would where Now ultimately But a aggressive take are But in we didn't you would broad, And models larger it would other are would had in as well longer which GFR a there units will KIM-X indicative impacts. a next in dose study in of than in you normal long -- the animal humans, seen improvements study, came the one lead in change. net one, NGAL make be in the that say don't enrich question total out can efficacy you a be able who

Unidentified Analyst

in likely primary view endpoint Phase efficacy your future trial? or measurement biomarker II the So the will become in which

Jay Hagan

portion GFR an with commit receive we're with they II study, improvement -- -- venglustat for encouraged And on an Phase estimates III to accelerated over kidney and patients on demonstrate total be design where to II one impact so our -- would very dosed cohort cooperation for they of approximately see a they in as I accelerated continue the effectively in think Phase that Phase years the post-marketing on enrolling with can in approval the full six on to based be the placebo. fully that if on a that volume approval that that a would follow-up Sanofi an that then kidney the would months long perhaps the division's for and approval volume change are setting after total year trial would size XXX an at primary in full additional of added a year look for interim be XXX two GFR.

in alignment sometime successful requirements forward. as So the something starting product hold got could what like and could looks remaining year we're be if the to next be that theoretically addressing for for division obviously pathway and that us that with moving we've

Unidentified Analyst

it. Got Does it. into operations company Got to XXXX? have sufficient fund the capital the currently

Jay Hagan

Yes.

think the I ended more remarks the with Yi, ended mentioned Cris year prepared in as her we first quarter with. cash we

And so add we're that of our balance you'll the filing we quarter the in had the utilized the to in part to earlier sheet. able see and ATM quarterly

changed So balance now QX we're million but we burning million about $X that nearly you the cash on got $XX imagine guidance million we $XX -- can got our through a sheet. right month haven't we've and

suggest that's additional any so. that's would capital. without So months And obviously my quick XX raising Yes. or math that

Unidentified Analyst

Thank Right. you.

Jay Hagan

you, Yi. Thank

Operator

question next our from have We Zhu. Yanan

Yanan Zhu

for taking thanks my Hi, questions.

study may same duration, So that? the have be effect but study which have the necessarily longer you of or duration final there are to have is mentioned? to kind that registrational not the opportunity -- an further a all increase another the the -- opportunity cohorts just study three PD first this When question mean after you current dosing see mentioned to continued I the markers. might on the

Jay Hagan

derisking we a level. that good we've to a do lot We've That's it markers. considered after Yanan. about FDA, an given dose take level step we study these a results discuss at interim a And see thought could additional X-month call at question. we a additional of with really that perhaps What Yes. certain doing dose

And so that's all under consideration.

Yanan Zhu

mentioned And terms changes little Okay. you a but bit Got on baseline NGAL, talk findings KIM-X also about also, in all KIM-X. patients? it. I of you you then and for the Could any tracked think the the

Jay Hagan

We're but internally is probably of used enrich me within anticipate as to as talking normal disease. would even would those And if That we those are that I analyzing in the likely all as think is the still were data, would patients here with that for in marker Yes. NGAL process range we more injury. kidney advanced well. as diagnose acute that come suspect well we Denis their

Yanan Zhu

Right.

Okay. than And more nine I your think have mentioned on call, the XX%. two patients then last you increase PCX of

So with of patient mutations. looks that PCX your estimate of the be certainly consistent XX% might

PCX genotyping do work or molecular before the does patients, course, two Of their guess But look for available to those that past like? what you'll pinpoint is wondering that. increase data the in they… if I just diagnosis look

Jay Hagan

but PCX up. notable. and if of too. share PCX and was It this had They both up also doesn't wasn't low was quite had PCX that It go -- the an also upcoming all had increase the at as those goes we'll they levels in scientific patients conferences, baseline --

Yanan Zhu

PCX I can give what's to for patient And in sense PCX example, level? level the see. of normal of you a a lastly, maybe PCX relative and the

Jay Hagan

amongst on correlate severity. disease The It's investor And the looks where total by the disease we you three the higher deck than it's with kidney lower as can volunteers. height-adjusted that I healthy disease volume. to approximately, Yanan our level clearly lower see variable burden in think, polycystin that measured of with the to have in fivefold patients

-- that. And and see we so

variability. advanced a lowest healthy, E So the amongst of And have you've quite polycystin. one classification levels patients of bit got the most with Mayo then

we of that believe think and changes and with an So up, know the animal good, is it's trend we fairly models. minimal in see we we good, impact

Yanan Zhu

Right.

Just clarification. a quick

more a PCX? to down or PCX PCX PCX decrease than mutation patient similar, is down For the is -- PCX patient -- much PCX, the

Jay Hagan

and down PCX in form are then of more and these think than assembled a cilia of polycystin-X flux. is you be heterodimers as know PCX. polycystin-X ratio PCX tends in more involved abundant, on to and these I and X:X

Yanan Zhu

up assay? it. mutant to truncated the the a there is does my to mutant be mutations, exosomes. that expressed protein or form assay immunoassay. PCX, get regard with of Got detected understand the that possibility exosome last the -- is on all of used it's a And that question Does the also could I -- that unless the and you PCX point the -- I on the Thanks. guess, assay is pick protein? measure part Is protein being assay that truncation mutation but that by

Denis Drygin

Drygin, take I'll this the This CSO. question. is Denis

had being told their degraded. So that around low -- they we teleconference key leaders do there any or stability hanging because us with and their no have proteins are is those enough. they the rapidly a is just of believe And they not in recently opinion of nature that field. Basically very are truncated their evidence

Jay Hagan

Yes.

So wouldn't ELISA the the ELISA -- to so wouldn't we down. go expect

Denis Drygin

Yes.

Yanan Zhu

Okay. Great. Thank you.

Jay Hagan

you. Thank

Operator

no I There to Hagan. this at time. over further questions And call the would to are Jay like back turn

Jay Hagan

joining for That do participating we'll Thank upcoming everyone thanks the and next front, And you appreciate investor time later Disease Summit Thank your the to Orphan Rare we concludes want on and Regulus. mention Oppenheimer's & call. us you. today. of be support week. Great. in I

Operator

Thank for you gentlemen, and presentation. today's this participating. concludes Ladies

You may disconnect. now