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Regulus Therapeutics (RGLS)

Participants
Cris Calsada Chief Financial Officer
Jay Hagan President and Chief Executive Officer
Denis Drygin Chief Scientific Officer
Brian Cheng Cantor Fitzgerald
Yi Chen H.C. Wainwright
Call transcript
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Operator

Good afternoon, ladies and gentlemen and welcome to the Regulus Therapeutics Inc. Quarter 3 2021 Conference Call. [Operator Instructions] As a reminder, this conference call is being recorded. I’d now like to turn the conference over to your host, Ms. Cris Calsada, Chief Financial Officer.

begin. may You

Cris Calsada

Scientific Hagan, afternoon discuss Good and you Drygin, questions. joining ADPKD is remarks we for our Jay will and highlights. Executive open Thanks, results before I Therapeutics and third to Jay call today’s Regulus will me results the Officer and Chief and provide quarter progress us Chief With operator. on for share thank and Officer. Denis President financial lines XXXX on corporate program opening review financial the

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

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

Jay Hagan

Thanks, Cris. earnings our to call QX and Welcome everyone business update.

plans We are pleased year. next for and our an update program to share ADPKD on

caused medical disease. disorders end-stage important disease, the most discussing gene genetic In Before our are I PKDX United by disease renal common into the States, this XXX,XXX kidney with leading dominant to unmet and genetic mutations to need, human Autosomal is one or of the is or the diagnosed of patients PKDX polycystic in get spend we approximately more there the a disease. on few landscape. detail want kidney cause the and address monogenetic ADPKD, a minutes efforts either

alone. restricted exceed said patients treatment opportunity tolerability ongoing to liver for distribution requires speaks are has its $XXX medication lead and patients. the to decline. to patient transplant, warning it The disease loss issues limited the to tolvaptan. life, diagnoses failure and on those its of ADPKD XXXX in believed That in approved in need of and slowly has by PKDX their only a the year both their their category. proliferation pain on disease the enlargement failure the renal gene. due for for in are and PKDX liver X.X in medical progressive has also to tolerability renal sales the which estimated cannot in aggressive to monitoring Current has disease, progressive be fluid they the million a accounting for type the million significant closer and which back XX% the Mutations function, options of have. injury XXs Patients is that of discontinuation market. prognosis due third to bilateral growth one are leading tend XX% U.S. Mutations tolerate under-diagnosed PKDX significantly very cysts. patient mechanism, to the box black improved It limited with to who a of its to quality enlargement including with Due antagonist product, kidney safety to and XXs, in with characterized in in average predominant likely and on of uptake. filled mutation it’s issues and is mutations However, in is due the sides the impacts potential with treatments this experience what depending leading in gene the the of form for the or fatal requiring for Tolvaptan to more remaining kidneys gene vasopressin

Now, controls antagonist been and miR-XX, expression. an we are and this important developing in our recall the that PKDX address has approach disease which PKDX shown to directly to to disease, turning to be upregulated

to by where polycystin-X, reversal after were polycystin-X the the treat that aspects of team providing Professor they at targeting ADPKD collaborators described generated Somlo’s rapid of addition read In further and both multiple the Nature Yale phenotype in of support the UT and recent of mouse disease, to in Southwestern, excited models our reexpression Group out at our Genetics, to data of publication of the of miR-XX ADPKD. we

significant an we antagonist statistically ADPKD. increases have to in in to been As polycystin-X, able proteins, year, through we two and patients miR-XX earlier these demonstrate polycystin-X disclosed this with treatment with

development of our been Now, compound, the has the XXXX driven like of discontinued announced on first have of next-generation October, efforts promising and you compound recent business was first-generation to limitations. we resources would next-generation by of advancement with This In program. compound, prioritize RGLSXXXX. update shown the our more our the the and without allocating progress development our objective all plans favorable towards properties our compound. the our our to to generation of prioritization RGLSXXXX I

by the we the doses preclinical with a RGLSXXXX seeing We top are promise, we greater progression this CNS at in believe absence excited including toxicology superior that off-target pharmacologic holds with chronic of tested. profile as the are RGLSXXXX studies effects

RGLSXXXX molecular potency vivo equal shown miR-XX its in target, and in both in studies. to Additionally, has efficacy vitro

important preclinical and when with We clinical tolvaptan, demonstrated in models efficacy have consideration. combined additive commercial also an

the clinical have subject XXXX IND. initiation a in for development We submission clearance meeting December RGLSXXXX in FDA to and with FDA of for of the track to are are quarter on and scheduled IND second pre-IND of an

RGLSXXXX first patients meaning robust polycystin-X compound. in polycystin-X of the treated plans top ahead changes demonstrated healthy early single planned volunteers data escalation include study a effects, enable around multiple the levels dose to were cohort ADPKD, first-generation XXXX. with in dose in where Phase dose reporting We clinical ascending study, our Looking and biomarker with X these biomarker in in anticipate line the to patients a study

urinary discontinue patients RGLSXXXX. at Biomarkers directly of of On PCX, decided drivers steadfast the RGLSXXXX, microRNA attainable. underlying to we on for additional these for areas, over Although American the We every kidney. I proud this in clinical be milligram doses. Kidney the Regulus Week respectively. X cohort the Society Cris? higher data the of to may for next-generation presented are be year. the suggests meeting favor cohort treatment. and turn development from that validating ADPKD. X first the are patients potential discussion in X polycystins the yesterday at help dosing of clinical results. linked were directly the advance polycystin key earlier The kidneys These date call role week RGLSXXXX mission showing patients suffering levels four will PCX levels mean proof Phase other and work of in engagement financial nine of that end increases in back enrolled XXXX, X of Nephrology significant by disease all were informs received increase genetic Cris November for to our disease. updates continued understanding Xb diseases which through from these to trajectory In compound in kilogram biomarkers RGLSXXXX depressed of important severity the cohort, first work believed inversely with I compound with and first of compared nine Recall a the particularly of of in patients per of subcutaneously trial in at our team ADPKD of the reinforces biomarkers polycystin to target our as target to in our changes the to data from correlating am the a Disease for of remain statistically Rare the now Summit, and baseline miR-XX and levels further patients mechanism disease in XX%, to in continued that the of we with disease of The demonstrate prior study the our XX% and at

Cris Calsada

Jay. Thanks

totaled cash financial $XX.X and Turning cash results, equivalents to XX, September XXXX, as million. of our approximately our

and disclosed, amounts diluted the and personnel-related same loss million quarter administrative same totaled period for share and internal development of current General These and of for ongoing loss to was $X.XX third pipeline. amounts operating XXXX. loss the $X.X basic the a expenses period quarter the net same $X.X These for in associated we to clinical balance was million and into to XXXX. is $X.X preclinical quarter compared external of XXXX Net for over reflect XXXX our the planned XXXX to quarter call XXXX period fourth the cash costs $X.X $X.XX costs. the previously share to share million third per third million quarter for million compared third period diluted per net XXXX. per the back with and in to business As million XXXX. expenses will for same the per compared sufficient of loss Basic I Jay. of share advancing operations compared and Research reflect in for $X.X turn that, totaled $X anticipate the of of for general the net our fund With XXXX. in

Jay Hagan

the Operator, we’re Cris. you, open now. Please to lines. questions take Thank happy

Operator

from Cantor the Brian first Instructions] Cheng comes [Operator question from Your line of Fitzgerald.

Your line is open.

Brian Cheng

for Thanks taking guys. Hi, question. my

overlap can there any to work IND done the anticipate in the XXXX the specific in discuss for meeting. much that how that also with need you for file talk are to in to preparing the pre-IND the had you’re you items about next as the past quarter, filing second the XXXX with And quarter? second that So month at you you do the of FDA will

Jay Hagan

Yes. Sure.

long reports So in-life document. wrapping busy study. IND the speak, the and toxicology team portion includes the work. is we the in earlier, here the of filing as at incorporation we tent, from the dosing for And receipt the had into to of that final so up the disclosed The of the the completed work toxicology we’re studies pole is

first work terms matter a of where informs from sense, see a it’s dose in And that the So what obviously, of we’ve there. XXXX. changes selection biomarkers, of got completion here of just we learned we’ve which in analytical with the program,

significant And level so where in as those FDA in lower we a changes very enables in have at tox to doses. seen levels. development. have which spend speeds quickly the on overtoxicity any dosing lack And sub-therapeutic our up the time levels starting sorts moving which margins based those much of of studies, with much observed IND-enabling we proposal We won’t dose presume biomarkers, includes obviously,

the elements that some development. are will speed those So of

in with ascending volunteers, of healthy terms in of the dose starting previously which includes includes Additionally, as And book, – changes. the of patients will have we’ve of and all going wrapping for this of a outlined, discussion so in toxicology our ascending speed with MAD in by – our further and single is from the development be study just that the the what dose seen over the as biomarker results next-generation supported proposal in well team multiple into doses the of skipping compound. clinical studies proposed the design part FDA briefing which terms as December we the meeting the

addition so in and more that’s meeting And Brian. CMC the package the things like pre-IND typical elements like aim the that, of to

Brian Cheng

you whether And portion then cohort between core cut and able on curious an first expect we from single-dose data do in also to any XXXX? volunteer XXXX. whether data, to whether just be patient of the healthy I so early PK/PD profile? the data a am – will data and now And a in top sense Okay. trial the interim line your in first will there get be

Jay Hagan

Yes.

in top we study the And and PK. obviously single So, wrap we ascending be have line dose results terms through when progress of healthy to will articulate it, in we updates able as safety volunteers.

is PD mIR-XX We are up-regulated volunteers. healthy in not for to any planning not look signal, because

wouldn’t we to detect dose. expect see single So, with ability significant to an increases the statistically

just be So, the PK and study. volunteer it safety healthy will out of

Brian Cheng

R&D one how should on expense latest slight on quarter-over-quarter. on think what’s also near-term? your it may, the we you that more expense Cris, Thanks. on finance a I your do in there or the any And guidance side, potential is partnerships thoughts the have expense, about increase And collaborations moving the BD seems forward? Okay. just on if front

Cris Calsada

Phase GMP Brian, R&D of increase occurred first X. this piece to that, expenses upcoming quarter on the the the was that in so, attributed So, in primarily manufacturing campaign support our of

So, one-time that expense. a was kind of

of anticipate we go So, kind have to the prior our averaged back what to expenses two in we quarters.

Jay Hagan

Yes.

on associated clinical folks. about it two XXXX. think yes, lumpy with certainly the that we And I we I with material think spending then for ongoing And are have has active. spend to that XXXX BD, the with whether on move business forward not is discussions is we program. going respect had with clarity the of it are then manufacture money regulatory fostered work have clinical programs have but appears guide and we am your do a group to And we to and on interest now discussions renewed in ongoing from planning development how will in it. anything, we programmatic standpoint our to if I

Brian Cheng

Great. Thanks guys.

Operator

Instructions] [Operator from the from comes H.C. Chen Next of Yi line question Wainwright.

open. line Your is

Yi Chen

biomarker the other a Thank XXXX X Thank am to has polycystin XXXX, percentage in expect taking see X biomarker could readout? Since and affect are that we level there the some sorry, similar you percentage – compared I similar to or for factors my question. should potency increase increase a you. a level similar

Jay Hagan

Yes.

that it well any investor like molecule, much the heck shown updated it no confirmed be when XXXX off-target was, – so unwanted really and XXXX in AMPA believe, And behaves are. would potency observed. have an possessed. robust were have So, compound is – looks profile that the – better, our now, animal a that’s quite much have of other of on where I of activity engagement that way in model studies kidney through like the chronic XXXX. which put vitro study kidney would looks it what of effects on And seen in treated which in a testing, we looks potentially PBS our about like the terms have a way think in of treated it, of everything as Yi, meaning all with based is all cysts effects like behaves very lab pan same as the to-date to XXXX the receptor, the PKDX the not updated underlying tox IND. robust dosed very the F/RC and animals been it the animal, seeing, in like our think this in we it deck we as target treated anticipate deck the the And we has we terms much investor toxicology the off-target in the discovered filled we Similarly, or we we well the the essentially CNS side that PK of control screens, lot that in that for amount XXXX in dosed the doesn’t

so we similar important us believe all points, activity. those of And leads with a have type to should that

lower Now, Phase of the meaning to disease. changes demonstrate past, in with turning achievable baseline we from with higher less than to changes discussed been the higher X shown severe the as have levels polycystin, more be have those patients baseline study, severe

study studies So, there to to from one because different are important the across nuances could comparing patients next. be

your expect set, to were would question, sample a think to I rephrase similar So, effect robust want important have absolutely. would I long-term, see I caveat. just I a But in to say that if we

Yi Chen

the built that model for XXXX? for Does mean you is animal XXXX was still margin applicable safety previous with

Jay Hagan

PK The profile?

Yi Chen

Yes.

Jay Hagan

model? PK The

Yi Chen

Yes.

Jay Hagan

to on like we we confirm PK. it – a and PK But based that it Well, the PK right now get from have look similar would that animals, data. does have has with

Yi Chen

you. Thank great. Okay,

Jay Hagan

welcome. are You

Operator

the like conference I back time. would am this Hagan. I at Jay turn further now showing to no questions to

Jay Hagan

provide update your you. thanks And of the very Great. to And Thank we for this much support opportunity Regulus. continued appreciate you. to

Operator

wonderful and Ladies have day. concludes conference today’s call. participation for and gentlemen, you your Thank this a

may all You disconnect.