Earnings call transcript Q1 2023

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Operator

Good afternoon, ladies and gentlemen, this is the conference operator. Welcome to Capricor First Quarter 2023 Financial Results and Corporate Update Call. After the presentation there will be an opportunity to ask questions. [Operator Instructions]

As a reminder, this conference is being recorded.

to conference like host, to now Mr. would turn over the our I Chief Officer. Bergmann, Financial AJ Capricor's

AJ Bergmann

Thank thank you. call. And you for today’s joining

we differ safety looking like be report would of statements regarding, These plans development start, These product future that including and things, potential current include and utilization manufacturing forward-looking milestone Before our forward-looking will the of are of that those our including of expectations cause our are payments developments that other we from or existing product efficacy, timing with and statements based research number the to subject and to and contained our may studies, results our involve risks other information, materially and making a and intended during regulatory SEC, uses and quarterly the statements to state plans, forward among on annual to today's in investment data, risks described filings and presentation. These made change regarding certain preclinical our candidates, I reports. uncertainties assumptions plans actual clinical periodic candidates, may capabilities, conduct possible and potential our statements are in resources. regulatory additional present anticipated our cash and filings, our statements. involving

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

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

Linda Marban

well X you provide as our updates XXXX call. will conference and programs you important Platform. Thank quarter AJ. Phase our Today, afternoon for Exosome joining first thank our Good on I DMD

had of DMD on and HOPE-X HOPE-X throughout potential application our this with are for and quarter the exploring positioned FDA believe from XXXX States the three the of discussions and the CAP-XXXX execute year towards and to of pleased first of with follow open-label in XX-month biologics of regarding reporting the milestones made presenting We to up extension continuing the multiple the strategic for include which progress pathway opportunities license of priorities we well that XXXX key outside reach our we DMD. are also of partnerships in quarter second data support Japan XXXX, and analysis in commercialization the United outcome CAP-XXXX fourth interim additional study the as months

non-dilutive to looking pipeline also We the well focussing of as forward partnerships exosome for are on funding. our expansion as other securing opportunities

CMC under today scale FDA providing commercial Now designation RMAT let Type-B FDA our Manufacturing for plans me and where Controls with on meeting our my update begin or an this Earlier of by outlined remarks production Chemistry year, recent interactions. CAP-XXXX. our we GMP the

BLA assay We filing able potency is the were to release support other outline which our and plans of accomplishment. to the order in also great a criteria for

focussed meeting aspect need this possible That with our our CMC on-going. been for GMP product there some manufactured was treated is discussion discussion yet Although development Diego still site. to and San from the of program about on was has be finalized patients not a

our a on that available. this on becomes with with the issue to FDA to plan path shortest to will BLA important goal reiterate like I and closely them filing it clinical we is end to would provide working currently that updates as development work are and on our

San now manufacturing Diego an for leads to This our facility me CAP-XXXX. update on

way versatile the increase early year. third that and to this inform GMP Should firmly of R&D CAP-XXXX cost-effective pleased market within facility able our know, to scale headquarters greatly see bring in-house market. this produce CAP-XXXX this facility of potentially doses be GMP as the product. our you margins to we to quarter am commercial a release to doses. manufacture designed to believe launch the ability We and approval, this will As track CAP-XXXX on are to obtain we you I support CAP-XXXX that in we our

we As capacity this operational, facility this fully color on able capabilities for potential will becomes site. be to more and production provide

would update Phase our clinical on like HOPE-X to enroll trial clinical continues to provide I Next, a well. X

we today, half to of this designed have active sites is As which on year, XX by patients remain sample the site. and XX enroll currently second track of a

level have sites and quarter, is continuing we are We team's to believe which our by the targeted by sites a to clinical well the as our trial activate strategic sites, additional high plan second testament of to engagement both as activated execution.

conditional results sample available to this of power Our for fourth size in remains re-estimation conduct we plans and analysis on year. and quarter the an unchanged, analysis are track to interim these have of

closely As I be clinical updates previously, should the FDA design provide and the feedback, are once with any trial working HOPE-X available, changes on necessary. optimize we to mentioned any will

profile consistent the and are follow-up in continue X going the to our year we In of year Extension, previously with fifth study. into a Open patients, their HOPE-X patients portion these in while into OLE parallel, we of the Phase treatment. the see results is XXX and Label treat study infusions. continue X Phase well to in safety going third of or OLE of patients supported CAP-XXXX continues as by Further, These IV now OLE, efficacy presented, over to be

XX the relevant, year. performance a report of patients treated function conference slowing to data patients natural a presented progression data quarter months in the progression, cardiac for the statistically see at The upper of disease the of initially this XX disease an including and plan at limb of medical XX-month with the CAP-XXXX, to are second down compared as who placebo, as showed and with we on progression of both for XX% of continue average clinically slowing disease. We the well and significant, these

positive patients on We of DMD. data to body positions their commitment benefits the us building families with and further to as on We therapy that are look anchor to potential their of for for thankful our CAP-XXXX the working exploring continuous CAP-XXXX. forward potential

be therapies will therapies muscle additional delay necessary long-term patients to from that potentially all be greatest with to dystrophin any get not gene get We the that growth the likely can the patients and for or therapies exon DMD attenuate we of progression the with need combination CAP-XXXX only benefit will may necessary Furthermore, disease the cardiac promote preserve paired therapies. be DMD. stand worse. also healthy inflammation, their in approved disease and of whose that replacement function. believe Most requiring that a likely can skipping believe therapy does with wish is We

of the Nippon partnership upfront where revenue. February, to DMD Now additional in a rights as will turning $XX strategy, second Shinyaku into CAP-XXXX entered up Japan, for share product distribution received to payment and a announced for we milestone meaningful million $XX approximately of double-digit our we a in commercial receive agreement to and with net potentially payments million

look work already to their continuing to continue already and leverage that Japan. Nippon approved U.S. exon expertise infrastructure and in forward their with established to We for Viltepso, the is drug Shinyaku and skipping

are additional priority. partners other focused markets now on key Europe We around and securing with the being world a

new the we updates to FDA, we delighted progress program HOPE-X with in development and our and potential, of our sharing from partnerships DMD Overall, progress of territories. our additional with interaction further look with forward are the

Exosome the body. technology, to signaling which turning of platform briefly our cell leverages natural Now communication the

applications. as with broad novel system to drug serve We are a exosomes therapeutic harnessing delivery

for therapeutic targeting. scientific loading tissue and downstream foundation is further strong supportive Our innovative payload methods efforts of specific

Microbiology proprietary two generated in the and induced journal nucleocapsid. SARS-CoV-X our Microbiology preclinical focused of Spectrum, independently We vaccine a on in program that response is the Society American published which studies the of and therapeutics. potential immune recently against a of two is broad exosome expression two in vaccinology candidates strong spike platform and StealthX, reviewed Our development proteins, combination StealthX at precision and peer core of on modalities:

Using program to targeting successfully the platform, StealthX us into therapeutics. based allow our will developed potentially which have precision exosome expand we strategy a

therapeutic exploring we area are are currently for neuromuscular Capricor. in on different working we targets disease, an is applications, While strength core which of many

explore grant program on look innovative this well leveraging funding. are our development and next platform small and as exosome generation will on of to become With advancement We current experts provide targeted vaccines therapeutics, they partnering forward exosomes, support of team working as available. the plans non-dilutive our to updates as strategies to our business and

program look forward We are the milestones. we across with closing, body technology. advancements upcoming executing our pleased growing the of platform DMD on and our In to our exosome within data

that, financial With I through call Officer to AJ? Chief Financial will over turn run AJ the results page. to Bergman our

AJ Bergmann

XXXX a of This summary release quarter first basis. on our Thank you, a financials GAAP provided afternoon's Linda. press

report financial as the we XX-Q, also Form and become well accessible of on to to on may the be You available our expect website. company refer section shortly the SEC website quarterly will as which

cash the cash, cash December marketable company's on approximately $XX.X on million, XXXX. XXst, to compared March As be totaled company's fourth this to distribution XXXX, is the of and XXst, approximately current our and securities Based due. plan, into also to with support potential operating expected Shinyaku equivalents million position note XXXX. excludes sufficient I that may operations that would exclusive expectation commercialization of to payments quarter become milestone agreements like under the any Nippon $XX.X

provided quarter by first XXXX. net $X.X quarter our to was first million in cash for the financials, briefly the operating XXXX, of activities Turning the of approximately

million expense approximately compensation, to stock-based $X.X XXXX. and development million Excluding research was compared $X.X QX approximately our in

in million QX $X.X in were QX our Net XXXX. loss and compensation, the approximately stock-based quarter for expenses million million. XXXX excluding and first Again, general of $X.X $X.X was XXXX and XXXX both approximately administrative approximately

for now And we with open will that, Thank you. the questions. line-up

Linda Marban

Thank AJ. you,

Operator

Instructions] from [Operator from is go you. Wainwright. The Pantginis Joe Thank Please first ahead. question H.C.

Unidentified Analyst

for is from [Ph] This everyone. Joe. just us. afternoon, our actually Good a Thanks taking and Matt couple for questions

your additional FDA steps aware or I or are limiting of The I moving guys to that was the first should have be regarding heard forward? there you came conversations we with up the commercialization, with any CAP-XXXX following that guess rate one

Linda Marban

in commercialization? in what, steps terms terms limiting of Rate of

Unidentified Analyst

Yes. Correct.

Linda Marban

on encouraged feel CAP-XXXX being full CAP-XXXX with both with of point, that time. front we at this We're paid also are They And ahead. this by to potentially the attention the development CMC value by clinical on at CMG. the None two at patients of the aware point, we recognize and working And this very closely agency. FDA front. the we're speed

Unidentified Analyst

regarding Okay. the the that know may details timeline comment, or not you Perfect. I one regarding additional platform? exosome second Good any updates have for able is be And but development you clinical do platform. to hear. my to then

Linda Marban

Yes.

So platform right on we're that building therapy. now, working internally

but We're tell you as we We ability as as vaccine to is in data published encouraging the specific haven't we're what can that platform to in announced I the Spectrum. Microbiology that types its very terms of working well specifically publicly recently cell prepared on, is is this what target and nature's drug I can believe What in opportunities. non-dilutive my exploring, you biology technology both as toxic exosome I that support remarks, will drive tell ultimately without to funding we and system consequences. also ability delivery mentioned the its partnerships that

Unidentified Analyst

in Thanks for Okay. I'll go my Great. taking the back again questions. queue.

Linda Marban

Matt. Thanks,

Operator

go next Aydin question from [Operator ahead. Huseynov is Instructions] The Ladenburg. from Please

Aydin Huseynov

afternoon, quarter. everyone. AJ, Good of much you Hi. I questions. providing Linda, the updates couple have a on thank for very

wanted to from sort start question. industry-wide of First, I

probably very be soon, the meeting DMD I will tomorrow, therapy. think there AdComm gene Sarepta's about

do regulatory the FDA regarding my and DMD think be effects there general read-across should in is, how So question you how about affect CAP-XXXX? therapies spillover path this we may for will and any think

Linda Marban

of benefit biotechnology the of Yes, statistically concept sort the and it for interesting impacted of really be evidence does AdComm. sort work lot a both I the patient the industry of evidence. entire there, is relevant then results to think versus opportunities actual of versus going There's anecdotal potential by

is mutation for deal be to DMD [Ph] of muscle muscle something turnover of all and DMD the specifically approval. in watching inflammation or the Dystrophin that with is right general Dystrophin mutation to a say and for from that itself. of get address but the consequences it what can can ultimately be of concept the of the carefully. about degeneration we that it be utero All mean utilization what believe will coalescing What does fixed necessary is due CAP-XXXX the to sort We're until also into will this approval, cocktail I to

well therapy being going need we're cardiac is technology therapy as take perfectly skipping implications. of the really CAP-XXXX as the skeletal with because exon or both care muscle any technology to positioned combination as So gene muscle help that to

So on let me more for elaborate minute. one that

is of showed body. which clinical meets the in Our benefits we ejection needs the important published HOPE-X heart of fraction lancet the which in trials how the the data

of the not the is lot is What has being become in clear a in therapy that studies gene impacted gene positive a way heart therapy. the by

It's are for repair So there's to a and right help with and young going these boys perhaps of another can and cardiac this continue quantity support balance even now. sustain physicians quality actively dysfunction be of to skeletal life in men and way to talking CAP-XXXX disparate muscle DMD. about versus which

Aydin Huseynov

your Thank you, appreciate very I Very, Linda. helpful. thoughts.

Linda Marban

Thank you much. so

Aydin Huseynov

non-ambulatory I that there so enrolled if you breakdown, could you is non-ambulatory update both you so enroll the today, understand And the know patients. how versus an Regarding many patients ratio share far what's to as on ambulatory Could provide you far? and ambulatory way any so of of that?

Linda Marban

decline. as of we're and of score criteria not five, as really the on focusing on and room upper upper again room decline stratifying because Yes, entry is as and end quantifying two, looking not ambulatory the the really them which so What criteria for leaves entry and leaves or of for for we're score performance for non-ambulatory well an which ability limb inclusion ambulation. we're improvement improvement, an based we're

many, people those on. stage the the upper in are late of have ambulation significant there focusing the in patients so limb and of many deficits actually performance And we're that are

looking we're our in not ambulation preservation. limb versus So stratification at mostly non-ambulation but upper

including but but And muscular called His limb dystrophy. upper the want independence limb extremely A I talk and still their his maintain name will are eloquently Small function Stacy of his of feet value is many great recently importance Elijah to and he's he opportunity to people, how speaks maintenance and to both we upper a really brother, about their anybody's function. off had with If, published to patient in if a book story, a interested Duchenne Elijah say,

are focused on our we patients. So laser

In of are in We CAP-XXXX terms disclosing families very we today enrollment that sense. in once you can to And that HOPE-X. is double-blind well. not it shown enrollment while community I It's the is very very the infusion tell trial, expect XXXX has in in and enrollment, we're XX what be and from a patients of the from we've to previously going our complete patients. randomized quarter a sites half numbers, makes a of understand interested number safe that our second placebo-controlled disposed

Aydin Huseynov

that is CAP-XXXX there to Yes, share eventually maybe what European could if yes, updates in And Europe? what’s matter production. does Nippon sense, any on rather than to thank endpoints And Shinyaku is Linda. clinical partnerships? going Yes, micro-dystrophy you, you any that makes market have on appetite your,

Linda Marban

kinds headquarters Shinyaku U.S. that Nippon go impressed all exclusively to not strong. the entertaining conversations partner be and AJ very on So and from their and right time. to possibly We're had earlier we I which both opportunity could year in this our valuable and looking European right think time, relationship attention perspective by were with very perspective. Nippon is the an the CAP-XXXX, and visit we their at the will interest select the Japanese this certainly of we at rights from they're at but Shinyaku, them that also

Aydin Huseynov

product to yes, are indication? Okay, what indication on if And all would so your any have be, So own to or that planning right. I'm for last so or understand, you trying partners? commercialize product without the would me, you that

Linda Marban

all Yes, so we're of constantly these evaluating opportunities.

build hopefully infrastructure plan the then continue as we it for has to this course, right own profit-generating CAP-XXXX company the is way for a it what the on quickly partner, door commercialization the but marketing in very Of the in we CAP-XXXX, on case, all Shinyaku is Nippon we and some move that to and our all DMD, feel appropriate take of we now best center. place to to

Aydin Huseynov

questions. much Thank for sense. taking Okay, Thanks you. so my make

Linda Marban

Stay Thanks. well.

AJ Bergmann

Thanks, Aydin.

Operator

concludes remarks. for turn the any closing to session. over conference to question-and-answer I This Instructions] the [Operator management like would back

Linda Marban

regarding in also all this deal the groups, We dystrophy. everybody trying afternoon our updates and of as on look all And to Duchenne and read well meetings for you, to And you. our out you transcript. patients, Thank later there at information all providing forward all muscular Thank who the our progress as the listen advocacy the future. who's on or attendance thank who of joined thank the with those we us operator. of families,

Operator

conference concludes call. This today's

participating and day. Thank pleasant may your for a have lines. You disconnect you

Capricor Therapeutics (CAPR) 11 May 232023 Q1 Earnings call transcript