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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

Ladies and gentlemen thank you for standing by and welcome to the Regulus Therapeutics Q4 2021 Conference Call. [Operator Instructions] I’d now like to turn the conference over to your host, Cris Calsada.

You may begin.

Cris Calsada

Thank you operator. Good afternoon and thank you for joining us to discuss Regulus Therapeutics fourth quarter and full year 2021 financial results and corporate highlights. call Dr. Hagan, today’s Executive and on and Officer me is President Jay Chief With Chief Officer. Drygin, Denis Scientific Jay will financial provide our open ADPKD opening lines remarks and and the will share I review questions. the for results we program before progress on

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

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

Jay Hagan

placement from steady by in Alport support Welcome ADPKD you, investors. for last quarter on XXXX and private by our closed year favorable our Regulus several November, that full institutional transformational first $XX.X of business our the was partner investors call financing a terms Sanofi. mentioning to existing syndrome in and million the I'll with program accomplishments Thank begin made successfully which we and earnings several new institutional included update. and year our fourth program XXXX everyone progress Cris. in a our

quarter Dr. secure and continues we In program. successful came move of particularly FDA more ADPKD conditions meeting we sector, hiring a market our pipeline, to into announced a make science, manufacturer In and on RGLSXXXX the President, regarding sufficieny including therapeutic length application and time included of announced programs the forward. Vice discussions His our oligonucleotides, Muhammad recent trial to development promise biotechnology Pharmaceutical of operations of IND challenging viability to overall With and nonclinical the helping year. the managing phase speaks agreement our January, and IND the and an for second broadly, plan study. the the steadily one remain we this appointment package, to our fit leading the of ability company for entered the pre for the financing with track various of natural minutes submit background the the plan the this December, of opportune FDA's as with this feedback, which which to design Given guide for completion with experience him Most forward. the RGLSXXXX Chemistry detailing in Development. deep IND we in overall life at projects Ahmadian as

As involves study dose followed single a in volunteers, patients study ADPKD. our reminder, phase by escalation a multiple in one escalation a study with dose plan healthy

compound. Top-line And year. robust demonstrated on biomarker generation half data we the look of the in RGLSXXXX With the biomarker the establish RGLSXXXX a first treated level forward Around providing of goal clinical the ADPKD dose. cohort effects is patients updates dose in to next first were with with where anticipated first to progress.

plan the safety program. dose of are fine at use the better and to data allow MAD and SAD the This volunteers we talk to dose SAD to and of at the first portions and from the X/B us in studies. dose exposure for data the the fourth the final in will the which and exposure generation healthy pharmacology PK the NOELs. extrapolate humans enabling the levels understand Importantly, phase based cohort from on NOELs We the demonstrate molecule intend leveraging cohorts the and the IND tolerability first the in the tuning we from in to clinical selection exposure, between relationship two

to will in the last We portion results the ensure increases noted the the dose system that the also achieved that with the will at level, milligram year. from study per MAD the in the chosen one levels it use these along learning PK of provide first-generation in and data patients poly was robust kilogram levels exposure produced

Importantly, also the talk will where And one saw increases to poly enabling in per studies kig other RGLSXXXX allow our for we than dose significantly cystic upcoming total completed likely volume we this GFR, not study dosing the one we're is first-generation to study in we IND parameters. see Since dosing. impressive kidney us believe in in the compound. improvements bit mig short-term systems the higher three about look month at and these meaningful changes the over architecture a

in cystic this However, with targeting disease. changes the we advancement able may understanding be a quarterly improvements, impact of polycystic to potentially XX in mere significant

into cash platform more provides kidney for potentially one infusion expected patients treatment for the $XX represents agreement, agreements importantly, terms program technology the cash first Alport Sanofi's of in we from a phase our phase Alport Final This development eligible Under diseases. half to Last need the to the treatment of HERA unmet and completion Alport initiation our ongoing further with of designed in or to receive the briefly Sanofi. under way adult XXXX. significant syndrome clinical (RG-XXX) this with upon successful HERA further therapies. the potential is no next enrollment evaluating additional of the data Lademirsen genetic announced address syndrome of collaboration million license X study study approved of XXXX. validation syndrome. and phase Lademirsen which is of company study Lademirsen the global the of Turning of month, the completion for our X

technology of over now to can to the for ultimately Cris? an have work our I'll where but our to to call forward our ambitious, the year understand of look discussion back leverage results. and us lives Cris of role of I disease achievable turn financial the We microRNA further improve ahead continuing we patients.

Cris Calsada

Thank you Jay.

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

our that distinct million fund potential to expenses $X.X development not plan and XXXX. include same This activities expect for Research of guidance through XXXX. fourth Sanofi the does million XXXX in We totaled milestone. $X the compared for will quarter cash period the

Jay. net XXXX. for advancing share expenses to the For and quarter the share fourth million share expenses XXXX in million associated ongoing $X.X of year. ADPKD an of were share share $XX.X million share, These external R&D related per quarter the costs Basic year. the to same enrollment $XX.X share included for internal $X.X expenses administrative XXXX $X million per General was efforts XXXX $X.XX million period million the Basic net compared $X.X the and the and per back of collaboration compared XXXX million to over Net loss for administrative amounts from full which net million for million in our loss full year our same that, prior XXXX With call costs. Lademirsen. diluted for the and per to ongoing for million compared same year to business the other net previous operating for will These a General the $XX.X was prior prior XXXX quarter loss with XXXX. our XXXX Sanofi $XX.X compared $X.XX were the I research period $X.XX for to for the for period program was amounts million the loss diluted and reflect $X.X loss $XX $X.XX fourth in diluted per of the for compared for general loss our for personnel and XXXX totaled and Net for basic $X.X year. interim turn per with and compared pipelines. of was reflect per to of in milestone compared years. to fourth

Jay Hagan

Cris. We're happy the take Thanks open please any line. questions now. Operator to

Operator

[Operator from comes Instructions] with Our first Cantor. Brian Cheng question

Brian Cheng

questions. for thanks guys taking Hey my

that So for phase is patient you study, as factor portion I question X curious from in view have you out and timeline laid Xb have one well COVID and your X as data set Phase Phase in I'm on meeting a also follow-up. just the first the portion how

Jay Hagan

a to Thanks, planning site the in the where and restrictions of single that SAD more a couple significant we at tend little X a years to didn't with respect impact. Phase It we're happened same really be issues to in COVID used ago a significant Brian. be state more much respect unit. study to recruiting at liberal before, volunteers Yes, healthy to any terms Sure. of encounter was with COVID

we that. anticipate on any So impact don't

targeting then about to we the with concerns about with hitting forward-looking the and beginning things Of of change a data statements number first-generation in pandemic. patients, the Xb which in we're molecule course the are to had the study to alleviate Phase time respect should And respect here. with these with double in first timelines sites respect could

Brian Cheng

and patients comment what Xb phase be of follow seen in on to preclinical kidney data on a or meaningful the long to you the at And will on to we'll Okay. be the total volume. some the treatment, about volume? study, to for kidney gen efficacy on on response be in measures need second about see how Based total GFR molecule to up for looking you've what's your GFR the your thoughts

Jay Hagan

Yes.

thus the disease, far. studied treatment we've always six obviously with So we for weeks in only patients

So commentary our based unique. required of mechanism primarily our other quite is compounds And course, for basing I'm development. so been has in what on

we've knowledge, XX mechanisms. I seen think the impact on cystic haven't mere we with to any targeting seen that with other our architecture

something time, that in months. optimistic that three with a to we see shorter something seeing cautiously in hopeful we that's we're of short guided we're why So respect can period but

the ultimately we see and count MRI in able to cystic duration in be what Phase interesting volume. would importantly, in year that are a in would to But term in architecture that's trial something that's In think if may take longer see And one we in advancements shorter that system there we may again, be historical on timeframe, change X particularly we've it but terms look which enable and TKV before to study that's where quite one imaging see of can't a poly in a with of at and in that architecture, see discussed describing might an take on cyst intrigued it impact bit duration in study. design from we're changes we're GFR to we based correlate baseline precedent. where as where levels cyst something the

Brian Cheng

you Got Jay. Thank it.

Jay Hagan

Sure.

Operator

Our next with Wainwright. question comes from H.C. Yi Chen

Yi Chen

is Thank you you million question. what the for needs first milestone to additional My the Sanofi? company to happen clarify my for $XX could perceive question from taking exactly payments

Jay Hagan

Yes.

meaning development X, with point hitting the is agreement. it that quite tried so you to confidential further because respect move significance effect statistical success often clinical advance when development. not you prospectively forward So the this obviously quite decide don't see and sure negotiated describe to of We here decision some that into Phase end a is their might and primary you're reach study, of to or clinical in you to trends may still into and treatment designed the

ways So in is that view, win two to our here.

Yi Chen

Got it.

my next is have so the response to we So complete recently receive [indiscernible]. question for letter

that you do of affects the Alport see syndrome you. ADPKD and well? how as So for Thank field

Jay Hagan

what plan letter I for complete they been to to to disclosed any indicated the provide response can but syndrome. they find FDA CRL information more what's with work the than, know with Alport to respect obviously by than don't Yes. address they've [indiscernible] additional

don't is with with of discussions it And pharmacology that we therapy, to who advisory what know unmet setting given events. committee. which approved the an to that at first here patient that much significant still a the potential approved [indiscernible] do therapy have need is was a difference in I potentially described that partner risk Alport was with at potential very you discussed know is have our be population be the older we for in work where community. I So the might our do be ADPKD expect an cardiovascular there as greater a and

of number including adjustments of we see. the the trial have design followup. They to So a shall made amount

from weeks eight the to I weeks size. four increased extended sample they think and

that's see. will So ongoing we and

Yi Chen

Okay. it. Thank you. Got

Operator

I time. to [Operator Instructions] turn at like call for the am back And not closing any to remarks. this I showing further questions Jay any would

Jay Hagan

appreciate in us everyone updates today. programs. providing forward to look your our supporting you. Thanks joining Regulus move Thank we forward with as Great. interest We for and

Operator

presentation. today's Ladies and this gentlemen, concludes

disconnect now day. may You and wonderful a have