CAPR - Earnings call 3 2023 | Capricor Therapeutics, Inc.

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Good afternoon, ladies and gentlemen, and welcome to the Capricor's Third Quarter 2023 Financial Results and Corporate Update Call. This conference call is being recorded.

I would now like to turn the conference call over to our host, Mr. A.J. Bergmann, Capricor's Chief Financial Officer. ahead. go Please

Anthony Bergmann

afternoon, Good Thank everyone. you.

include filings, Before we future and we and intended to other research may that utilization of candidates, development involving additional including plans to our product and would be clinical data, plans I capabilities, presentation. payments our our studies, the making potential anticipated our safety statements developments call cash and like will conduct uses today's certain state and during and timing plans, investment potential regarding among manufacturing candidates, product our regulatory or start, forward-looking resources. efficacy, regarding, statements our our of These of report things, statements regulatory and preclinical milestone present existing possible

on are risks and expectations based those materially and a quarterly filings made assumptions forward-looking contained may with uncertainties involve and differ the statements subject These in annual change of actual forward-looking current that SEC, other risks our number and including from are the reports. periodic in cause described that are and our information, to to results These statements.

such You undue not to update these on statements, we cautioned disclaim obligation to reliance and any are forward-looking place statements.

Linda turn Marbán, I'll over the that, call With CEO. to

Linda Marbán

the for of A.J. making the for I'm progress lead Duchenne Thanks, towards our joining we muscular the thank afternoon, treatment Good extremely and CAP-XXXX, you asset, are today. dystrophy. us pleased with development of

across inform delighted to goal I designed pivotal that As XX which for III today, States. Phase to have approximately you HOPE-X study, reached enroll United call am I targeted patients our begin we our enrollment the the is

should or interim The to continue prior the evaluate on our This as The presented this milestone safety purpose planning blinded our DSMB, is end point on data the primary futile announcing will of analysis planned. should X-month based is be our as which analysis to give the from study their to as year. and Reminder, end are us based be the to if is planned. will we will of important trial on decision the continue trial and this determine the results. outcome efficacy Additionally, of X year. whether

Should the There first an that with trigger support milestone also to trial will other will payment BLA futile, our agreement sheet milestone be achieve U.S. payments not Nippon them. leading this to under our a determined we should are approval be to Shinyaku. continue our balance potential of up

the be previously be have in from would line announced, A will subsequent QX BLA I based Cohort full As on the results set. available data and a top XXXX,

reinvigorate our towards for However, speed we pathway been possible the reached. to point lost, an now CAP-XXXX We the are enrollment our approval CAP-XXXX community approval target to for HOPE-X as move to is muscle. the it that if and that determine as Cohort recovered. knows it planning be to has A to quickly now as up a discussion time pull FDA is cannot opportunity is everyone with there patients owe Once

was which overview to meeting was FDA, why believe outcome me QX we of an the the B our for Type with in announced brings clinical this recent the program. late Now and important

of product begin commercial as recall, is meeting. wanted now manufacturing, additional HOPE-X X they results referred the Number number cohort our to to of FDA and planned support Diego will filing treat facility Cohort FDA that an And patients main the Cohort support two, BLA. facility. A San As the of you enrollment inclusion manufactured to Diego were with to our HOPE-X patients B of of design approved new additional imminently. in in the outcomes There a may one, earlier us study for this the indicated the San the XXXX, GMP at from

mind in initial Cohort keep from It data that of DMD. required that registration be and very for for we important the will CAP-XXXX B to generate not that is

has carefully manufacturing clinical to several facilities, FDA Pediatric Orphan invested Drug from successful securing Japan commercialization years, RMAT, resources securing U.S. in X through last the and and for Over this diligently Capricor Rare CAP-XXXX building the and program, in markets. Designations trials, bringing addition partnerships X

I the further into transform development company is carefully These development-stage of operation. exosome invest strategic organization Capricor, our from deals and would CAP-XXXX future $XXX to have strategically for potentially of allow million which that over This the would R&D Capricor, were potential cash in negotiated bring therapeutics and additional and expansion fuel a our pipeline. which us vision a to commercial in in world-class product

developed and the manner. cash realize of with I the as our invest completed now have done to moving in manufacturing are just-in-time To that decision our Phase history, the this have only but our that we organization, in clinical target a III, time. But that of in in respect CAP-XXXX. development not in made before have priorities We to CAP-XXXX enrollment management managing much accomplished and remained we the point has resources, to the closer vision. operations our while disciplined early be across we we to also

that requirements Now out we approved. data to demand capabilities CAP-XXXX produce and of manufacturing in FDA larger market we meet if clinical shown are and on multiple our focusing scaling quantities promising have trials, to up

in cost equip. facility and million $X San approximately build manufacturing GMP to Our Diego

As expand complex which regulatory is manufacturing, and is of all experts the case cell compliance in CAP-XXXX include biologic companies, reflected in such organization. operations requires structure the to affairs, quality, to with us a current as the launching

to recent our gives public capital has inadequate for the the with assay. approval action approximately years. our in resources, into hindrances of XX an the notably to And the $XXX approximately the most that million past XXXX. The cell over this most invested recognizing $XX one of cash DMD both therapies have company's of understand many a in factoring mechanism CAP-XXXX financing in potency million history, strengthened our in Capricor program, dollars and ability private has runway of equity QX financials, throughout aspects totality raised and of in us CAP-XXXX, been

step program, our is pleased towards potency that important an We the which supports FDA approval. are

With the are in to patients inflammation we potential into product of a fibrosis. with diseases believe treatment expansion with similar be paved diseases CAP-XXXX's DMD, for and path now pathophysiology. transformational by We planning

Second we are platform building to a become next-generation judiciously that, our platform. exosome delivery drug technology to

with on that Now update quickly said, to an exosome let's turn our technology.

technology useful that advancing engineering for pursuing vaccine drug our StealthX, is focus of program or our either we exosome. inside of been committed X our on we opportunities. of CAP-XXXX, next-generation development proteins While primary the exosome delivery surface part One our platform. for using select are has Currently, of remain avenues as on is proprietary the platform the

a several our For our platform, and we remain mission. nondilutive on with are one either vaccine discussions of clinical is source program, focused is of speed Currently, a to funding active significant interest exosome-based there partnership in to or parties. development. that secure in vaccine aims this We

harnessing stage therapeutic with work exosomes. set future therapeutic company, the program of on prospective The The go. loading other to vehicle. aspect of tell cargoes requires it we has of in our this exosome exosomes The foundational therapeutics where into with specific collaboration platform exosomes to uses outside the options develop a essentially as moieties StealthX are of platform. with for the an the pharma potential investigating delivery by undisclosed to targeting the Currently, to

intravenous is mice year's Muscle a of oligonucleotide, becomes important effectively ongoing more be injection. conference. on demonstrate the enhance and results antisense this repeat at potential from muscle Repeated from data our study surface was approach exosome which delivery, presented targeting recently World nanoparticle. Society therapeutic engineered post loaded doses study. Proof-of-concept labeled on show by program as exosomes this ASO, using of the has engineered in look PK on exosomes suggests as further that differentiating the a to color presence it lipid limbs moiety to I representative the enhanced Initial targeting forward available. cargo of demonstrated that based providing initial can the This lower this uses of our the dosing

had fortunate through to are sheet for required their of of a this financing submission reason the the BLA. XXXX. a prepare support The on in We and a found significant, further approval team we our to approximately was cementing space. into to Part work the that finally, prepare we and expand fund commitment our driving Shinyaku, by was strategic the million This anchored is have side. relationship the order for is Now $XX to programs to biologics our late-stage Nippon to raise corporate in team clinical quarter Capricor. asset experienced in strong balance raised

to Michael Group look strategic now Therapeutics, part of of contributions in President Development Capricor's brings to forward Finally, and the to Michael Directors. of as Capricor and Amgen. in Kelliher pleased Horizon expand the recently, of We to our announce business expertise are Vice Business we experts, our served effort to to at Board our appointment M&A Most team. of He Mike's team development.

I and thank for your support company. for want program Overall, our our you to

We FDA. well announcing are continuing be the analysis outcome several as interim will very forward as from much with the looking we interactions next months to as the our

you. I to will A.J. financials. now turn the to go through our call over Thank

Anthony Bergmann

as on shortly our be to afternoon's report This press a may website Form expect website. the the third section XX-Q, financials available our which as GAAP you release on quarter basis, become and we refer a financials summary well provided Linda. quarterly of SEC XXXX will Thanks, our also on accessible to of and

Turning to financials. the

prior Let direct cash, XXXX, we third our gross into that operating of our excludes extension equivalents me XXXX, $X.X of cash now revenue million million. and the extend we recent for projections, We registered was to on start marketable million Based million. guidance. offering commercialization the current approximately cash quarter an to ended approximately cash completed In with position. results This XXXX, securities $X.X our $XX the with from September That in from XXXX. our the in October our expect runway cash compares our XX, approximately bolstered and proceeds $XX.X quarter position. of third

the quickly Turning to expenses.

were million of and increased to XXXX. third and HOPE-X $X.X The research of XXXX of clinical approximately to costs quarter million trial. due our was primarily for third $X.X the Phase compared for III in expenses compensation, stock-based Excluding quarter million with the expenses $X.X our increase development associated approximately manufacturing

the expenses $X.X increase quarter costs. our quarter million XXXX. administrative million approximately for and approximately for facility XXXX and third of to due compensation, to the of quarter compared The of net as primarily our approximately $XXX,XXX million and the And were was stock-based $X.X XXXX third loss third increased XXXX. general Excluding $X.X was personnel for of

back the And turn ahead, line Linda to for that, actually operator. that. me first do it before questions -- over Go let we open with up

Operator

Joe [Operator Wainwright. Instructions] H.C. from And your first question Pantginis comes from

Joseph Pantginis

going I'm exosomes. regarding back start at question, comments the first your My to of end

the you in XX flow just data be secondly, confidentiality. So news next said preclinical a there get what wanted I X sense of might be the months. now, disclose be to some so discussions But over we can there's to ongoing kind And might vaccines what of you after? you might potential getting right going know

Linda Marbán

Yes.

Thanks So for thanks, your Joe. question.

background. are excited the along exosome in about been perking It's program. So we the

We ability I to not target, CAP-XXXX, World have and been mentioned, from positive devoted most before But of recently, the our shown exciting, knows. very as which everybody exosome has has presented as been to the and data very was really time Muscle. and the at effort including

coming to that's program. time. a on So plan continuing been had in up do in in of COVID quite we We've milestones terms development for this some for vaccine advance XXXX,

lot to come using to the has realize that world the be a left about vaccinology to there's think mRNA. understand I

And protein-based the nontoxic of vaccine of gives a time great using manufacture, molecule in a concept the that concept program. to is manufactured easy interest and of certainly native as and be to exosome proof several just make, to proteins using using for be is something indeed easy months such the so can of

just working this one, vaccine. for we're farthest vaccine options point. the partnering opportunities for the for along other as looking that's the vaccine of well conversations at as The for So type therapeutic on, the same are

Joseph Pantginis

following and parts had very working question has A guess you be to about important to with very, program, Got But the I main obviously. Type look, DMD it. And meeting, B. to Cohort B your there's And able a helpful. visibility the path here. Cohort forward my few file do That's in

the further your So you I programs. would to or question interim a continuous sort also your around data? around any upcoming the analysis. my I of potential And my I disclosure to being maybe Would of And might potential little able share of guess the any public stopping revolve be us? with planned rules potentially about a analysis question to is able still bit focus accelerate scenario comment guess

Linda Marbán

Thanks. Yes.

So before just year. since analysis. end start of recent it's futility be It is the upcoming let which the me the with a the most milestone, interim analysis would

data, and or preset metrics, trial efficacy DSMB gets continue evaluate -- been on not based disclosed, whether planned. the which the and So safety determine as the are should not

story. of building are the a trial we and to interim -- to We and plan, But all forward not great to of that our clinical We, day the celebration DSMB that we're and that for our previous it results course, is of be from how will on get expect a analysis. be continue. futile looking futile, the continuous course, word be the of trial we that's not can based work, once

And that confidence planned. that. can trial we that the of where that terms In but with are actual proceed as we metrics, should say standard futility analysis, that's as good not disclosed a we gives the those, have

of sort with as or I appropriate how of back going West, what coming, these what's of terms I were sort opportunities they FDA about hold dealing it's the going making not something what approval And that for like never everybody, funny, your FDA, see really to to the is was as the know they're what guess for Wild, unfair you but In right? are available to with thinking respond. Capricor to dealing on. days, We it's say are, question, for for registration versus not asking Wild

the clinical upper have and improvement young So trials knows muscular men you of the Duchenne what dystrophy. X FDA in in tell performance I later-stage is can limb showing all what positive with we

to their sons better. receive functioning have because families clamoring feeling are that they CAP-XXXX are We anecdotally, say and

on they consider they want We approval. see fact III strong have a ever the which trial, before fully would enrolled has trial absolutely Phase to fully that enrolled FDA the been accelerated confirmatory

community FDA we our FDA. speaking that we designation checked now. RMAT have with also families the the really consider also of with up we the to box and So And that ear from

So the involved us. long development. get conversation you as gets in time point this much for a to It's been more coming

There's delighted. am touch I and many So opportunities points.

every we And should trial the that of points enrolled this to broach approval. be at success fully But we with so are path line data there may top touch be level for that will X to at A, each those Cohort poised idea FDA, a in with just year, feel really, continue of point, a the a we positive.

Operator

your And Aydin Huseynov next [Operator comes Instructions] from from question Ladenburg.

Aydin Huseynov

with questions. progress A. with have the quarter this congratulations and congratulations enrollment completed Linda A.J., and in I Cohort a of couple

from First, start from potential milestone Nippon to I payments Shinyaku. want milestone --

of your So $XXX potential gets drug is approved Nippon from several total for the next the sort million. if receivable years

a positive? you the may sense much million, total year decision by a can just the Nippon So how if million, amount $XX us of it pay be of us you give will overall that from the Is end Can million? it? DSMB sense $XX $XX give

Linda Marbán

A.J.?

Anthony Bergmann

to dollar that state affixed figure Aydin. Thanks, not futility now, exact interim at Yes. to liberty Right we're the analysis.

is we granular will put hopefully it as details out. course, more Of achieved,

for So that. stay tuned

where that's we're kind at. of And so

of at what disclosed this as but of to including up million move Now potential terms up approval, we're to approval that milestones in $XXX Capricor. say point to we we liberty building the is and is milestones to haven't towards now in time forward the

of terms going of efforts we're the Duchenne expansion well that fuel into as to product in as other to So CAP-XXXX look to obviously expansion.

million. hopefully Hopefully, -- So will that those that's will come total milestones due, $XXX helpful.

Aydin Huseynov

super A.J. Oh, yes. helpful. This that, is Appreciate

Just milestones. so $XXX million to clarify, approval including

you So imagine approval I in -- get okay. -- that

Anthony Bergmann

available our correct, shortly, be the that it. I'm XX-Q way saying which basically this out should look to articulate But for there yes. That's shortly. put And will become same Exactly.

Aydin Huseynov

All right. that. Appreciate Okay.

question on Another is cohorts.

you're seems starting weeks It now. be enrollment like B within patients. going to XX enrollment, completed Cohort A Cohort from So

by I think mentioned year. the end you of the

in holidays, So the do X? given year, now it enroll -- between patients the Cohort the actually to you realistic and B of sort think is end by still of enroll January

Linda Marbán

my young they There's DMD, later-stage the back. asked be is So pretty and with robust is companies in trial clinical end with and Aydin, now call to the other Because some nothing space of were said right pulling team, that going recent and team what this year. nothing yesterday? on the of else you boys my enrollment the men I these for before in failures clinical

the Cohort a there's out get holidays and to so B, them delighted we'll to welcome line and even door treat And with the into up. be them coming

Aydin Huseynov

Oh, great you that to so enroll. -- is yes. it is trials, no I This given other feasible hear. mean, there's nothing will

Okay. Understood.

obviously, is with different DMD the approvals. landscape given treatments, so changing, different And

So A patients? So you the any -- X be different future? you are in do these that cohorts think Cohort think these will somehow different the variability do B Cohort between be from will given

Linda Marbán

really debated a provocative answer and I a lot short It's no. we've interesting think one question And the Yes. again, is internally.

their the up We of potential have and to same many new criteria, that. only same all because patients approval a that The regimen taking open are the Deflazacort, of the due better to terms of them the requirements in switching inclusion/exclusion with also on profile. we're meds safety differences of

the So only that's really difference.

what We don't potential any We've sort of steroid a at talked mediate to our a in steroid difference in KOLs going is steroid the is length does of of the think it physiologically. terms sort that's to efficacy. and

we're sort will same, allow the hopeful manufacturing. and B of be to will that commercial-scale transition virtually very the criteria Cohort So from very clinical-scale us rapidly enrollment manufacturing to

Aydin Huseynov

very Appreciate that. helpful. This is

expansion of another of I question label CAP-XXXX. have So about potential is

DMD dystrophies into other So other other other companies or types have may of to DMD stages patients. of DMD certain or expand limitations

DMD potential Becker or your into into and So dystrophy muscular you then early-stage have patients corporate BMD. describing presentation expansion a nice on slide

patients So these early-stage patients active look with potential be how a may more you CAP-XXXX? could So mobile. more are likely won't with points because and this help understand us pool the end like

point how end give of bit look just may like. idea us little a this So

Linda Marbán

Yes.

So it's interesting.

All measures have reported of -- these their have strengths weaknesses. patient are and outcome their

really strongly data, a been I that is measure. with the question think Sarepta interesting one. very in little around recently there's everybody There's believe but which to the rise, veracity an of time very NSAA, the

efficacy yet. about the opinion our and to FDA working find help are and that in label advice who effective more haven't in then to the Frankly, designed of these younger We most use potentially we're with used trial these work evaluate kids. key CAP-XXXX to younger talk going the to of there that's expansion from demonstrating without one boys leaders clinical

Aydin Huseynov

then dystrophy. is question Understood. Understood. the regarding And Becker last

one the would causes sort And major dystrophy, sense death. for cardiomyopathy obviously from is starting of So late-stage of of Becker it make patients. the

share us how advantageous Capricor So companies more position Becker compared when could you why is -- with dystrophy to or in is to other it comes advantageous more dystrophy?

Linda Marbán

investigation of of know, terms one is of of have other that's major implications. any manifestations Becker in cardiac dystrophy approved our dystrophy, one muscular as advantages Becker for therapeutic course, the In And is we that almost benefit. are or of you cardiac DMD the -- over major

So if of we dystrophy implication. highly that disease, and a dread muscular treating talking are cardiac about sort for about terminology the its enthusiastic, when Becker in CAP-XXXX use you opportunity you're can

Aydin Huseynov

Linda and congratulations again A.J., Understood. Understood. with progress. the

Linda Marbán

you Thank so much.

Operator

questions management final Instructions] be ahead. There the turning are to no for Capricor's [Operator remarks. Please I over back this go time. further will at call

Linda Marbán

much. And A.J. Thank you you so much. so thank Thank you,

that patients look season. an to for much. so the Duchenne I we have minute enrolled. We holiday today's us as for day I community closer later-stage you patients. to to the our will be at also an to to a families with hopefully, extend Before potential data, fully as working trial take meetings. you'll healthy us. continue of team hopefully, therapeutic thank and you for for just these And happy for I who celebration patients call, are we'll to want be operations seeing bring their appreciation CAP-XXXX job these forward getting well clinical a my upcoming want a sincere get to And And to the this once to able approval. have know follow extraordinary we doing Thank approved and clinicians, and conclude

Operator

very you great disconnect day. your ask your Ladies gentlemen, today. lines. for and thank a you please call for participating this Have We much and concludes conference that

Capricor Therapeutics (CAPR) 14 Nov 232023 Q3 Earnings call transcript