CAPR - Earnings call transcript Q2 2024 | Capricor Therapeutics, Inc.

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Operator

Good afternoon, ladies and gentlemen, and welcome to the Capricor Therapeutics' Second Quarter 2024 Earnings Call. [Operator Instructions] Also note that this call is being recorded on Wednesday, August 7, 2024. And I would like to turn the call over to our CFO, AJ Bergmann for the forward-looking statements.

Anthony Bergmann

Thank you, and good afternoon, everyone.

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

to on to Linda such are turn over statements. With call place update You the undue that, to statements, not these and Marban, disclaim obligation reliance cautioned I'll any we forward-looking CEO.

Linda Marbán

Good you afternoon, and today's joining quarter call. A.J. for conference Thanks, second thank

our of significant Now muscular CAP-XXXX, submission Deramiocel, Capricor XXXX, halfway to on of Duchenne well Biologics License to products pipeline continues a developing asset, exosome for of over our treatment bringing dystrophy closer BLA, lead which make the referred through previously for the was clinical progress as to or as development. Application as

strongest in space. we the long-term June results open-label in and seen extension published results, the the are of independently believe recent conducted trials clinical some Our data

for cardiac the both due can product's protects which from typically We the standpoint. from afflicted muscle of a therapy caused by progression all it As Deramiocel addresses dystrophin, boys inflammation skeletal and the slow men the a disease lack with with as young backbone to and believe DMD, cell ability damage. potential cells significantly death serve fibrosis to and

Additionally, message, all DMD a fields have this as to to the address the the of consequences continued require of will paradigm DMD pathological KOLs across multidrug disease.

that potential potential our to So in provides with $XXX become everyone approximately and million may agreement milestones United other I for sales Nippon remind to commercial Deramiocel and the launch in due and inflammation want developmental approval Shinyaku move to States focus commercialization, as enable would that distribution diseases our resources invest of to us give Capricor, strategically which towards and expand fibrosis. our to us

year. robust with of discuss to My exosome-based and we for a are later the FDA to to are opportunities is and to potentially R&D deliver this in we our And be treatment we of operation organization pipeline with Deramiocel's opportunities commercial goal including world-class potential become for for to the proud of I the platform. Becker StealthX progress to expansion expand In We date. transformation, beyond. preparing that DMD have made and the positioning become muscular a Capricor to growth, fact, that to position believe transformational dystrophy Deramiocel into a continue

to of volume. ventricular measures, and highly in end in terms function multiple measures patients progression results, and update to considered the significant statistically diastolic subjects cardiac on recent systolic Open-Label OLE are with moment presented also end patients. XX% X.X This of and comparator translates including or you showed Extension PUL which These DMD p-value benefit improvement long-term take the X-year external ejection treated I'd and Now relevant into versus left dataset of compared outcomes. an of fraction, volume a study as Treated external ventricular as points improvement ventricular left shows approximate comparator. Data well X.X in on disease year's are a this like cardiac indexed to delay in similar left Meeting. Annual at X.XXX an PUL in our PPMD were HOPE-X an when predicting

in In in the as no that cardiomyopathy. at trial. Currently, cardiac observed approved you randomized know, addition, treatments clinical HOPE-X greater for XX% there function beginning those fractions were improvements DMD the patients of greater had than are ejection specifically

accomplish Our the as in improvements of X-year that HOPE-X to those DMD favorable that DMD has cause the strength is the with associated we for leading of death inclusion Open-Label continue actively Extension the possible, for only and we to goal. a shown to therapeutic underscore Deramiocel to study and for favorable which to treatment as safety want and Capricor's cardiac the allow primary consistent profile, the clinical data file function and and continues FDA reiterate data a significant our of knowledge on is its benefits of our believe our is long-term community. to with trials, show that the patient aware these is Deramiocel are statistically for cardiomyopathy, label of I cardiac working BLA DMD. Based FDA may results which soon DMD. FDA important Deramiocel, to in goal acknowledges from as potential the

be approval HOPE-X have from with met data studies pathway our we that HOPE-X awaiting support while are paths alternative can an data One with minutes OLE are at filing. meeting. confirmatory FDA later pathway pre-BLA discussing We BLA the final positive from BLA we would the accelerated I to be currently share the just and to date. in provided a with a filing very available meeting, And our to

full approval. Another option traditional a is

We the our FDA's are pleased to shortly. acceptance our rolling of submission, that, of and on announce BLA BLA initiate based intends to filing Capricor

and CMC for San for filing. facility manufacturing last the the operational Diego our actively front, commercial fully is preparing On necessary quarter, runs we reported as

our preparations commercial have for accelerated entry. pace of we the Additionally, market

their Deramiocel they expect with partner, Our of is DMD. payers role by for for Deramiocel and Nippon focused on We Shinyaku, launch. and rapid ready getting adoption in patients

preparation expanded usage. areas market reimbursement actively are as for the and a smooth as them with access, focused of We for engaged is Capricor label well launch. on ensure to successful opportunities

our program. Now on for exosomes update an

focus delivery are primary Deramiocel, advancing we as our StealthX our is exosome our technology on platform platform. committed to next-generation part of While drug

scaling years, for the experience exosome ability planning of experience. out as to paradigm. mechanism Deramiocel that success up based for a has targeting been exosomes, has action cell We built with and of based The therapies and take as on end products as therapy to be as advantage refine strategic well cell-based their of to products cell quantified for our goal as several foundational treatment of terms well have on them been more such engineered our pipeline of continue in to to

the our develop program to and as of exosomes platform delivery by into of it exosome therapeutics targeting Our exosomes moieties technology to direct the exosome This proprietary use vehicles. focuses on harnessing attaching to where the the of loading requires StealthX go. cargoes outside on then the specific

platform We our the continue platform. are currently development of to as opportunities efforts therapeutic ramping exosome develop technology up we with our business the

Phase our end prevention, Diseases Allergy phase we with will future National vaccine and Infectious to NIAID in Institute also Currently, Regarding aims plans of United and and with for to the deliver candidate test NextGen, candidates collaborating fund to prepare candidate, are vaccine XXXX. conduct for X Project COVID-XX actively vaccine States government's of which pandemics. Then is our a the fully to by the trial. clinical manufacturing

for On our Vancouver, presented Society We for is & in with made preclinical data exosome platform, the development. which therapeutic Gene presentation. front, were an we advancements the we have Therapy notable selected oral where International Cell at recently in

target that and an enzyme Our this potential lower reinforce preclinical data to exosomes We deliver the Extracellular highlighted moiety. using limb. Subsequently, payloads, to there research The these we our in program work conferences replacement strongly International moieties targeted arginase-X only of continues mouse our also can potential of muscular an therapy, treatment StealthX Society Duchenne in in-house applications to poster earlier for with the delivery direct demonstrated is exogenous applications, for one Vesicles to antisense Melbourne, findings muscle targeting on was a significant including technique, Australia. proprietary mouse at approach but exosome-based delivery loading This cells, data skeletal dystrophy. in not from in skeletal our muscle as Combined lower are muscle being carrying featured of broad presentation a can oligonucleotide, data our the developed both our exosomes the potential data support targeted of developed deficiency, believe muscle the for suggest which presenting exosomes belief. delivery opportunities with for limb, a platform. that targeting from

runway. to path and Now a quarter like would us priorities as our we additional potential this provide current moments take progress balance bring sheet our Based are through cash the extend last updates significant a corporate positioned to well capital progress on to we that expedited and gives XXXX. and Capricor clarity to I now strengthen our confidence into BLA, over our few to

While Europe the DMD. best primary various our potentially opportunity multiple several this to in parties, goal, mission of the Deramiocel interest to opportunities, to business pursuing active capital ways and for looking development we -- of there we different shareholders. are are Therefore, business in are discussions accomplish with our secure is treatment distribute in

garner highly we they valuable partnering We the become are the would I share for available. We these in achieve you asset on clinical with the the for well our discussions development EMA to greater other asset inform partnering EU value FDA. relationships. we these have that am will been a efforts in focused believe late-stage that as as in pleased as and rights, remain very regions as is and judicious world, and around with through securing knew clarity progress the to as Deramiocel rights we updates respect we

balance a disease a and institutional to buy eligible visibility an we extraordinary Furthermore, to story. In approximately in payments should and achievements, the to XXXX up expected regulatory-based our year as are continue designation they our milestones Russell upon to these can Indexes, Russell to Deramiocel, sell-side were added to Capricor which some Nippon which we which $XX will XXXX them near-term. Additionally, ago, approval rights. And to conclusion, extend and we and believe our million the be Review support of has announce support if potential far. Voucher, of runway. Shinyaku, bringing to time moment our is continue on for this quarter achieve we earlier million cash mentioned been pediatric Priority finally, Capricor, more be will PRV, triggered thus we this our certain on that of pleased has our be remain to U.S. payable milestone with we for further sheet bring to We focused based approval as the full to a retain receive I company Agreement added $XXX as receive to in

families and are strides have supportive efforts We to feel those potential getting of significant power and made and of in function regulatory our with fully lasting pathway, the better. Patients Deramiocel to closer FDA each see our DMD approval and helping with meeting.

and from We a have other the for doses in equipped, biotechnology facility Diego are manufacturing our San cost producing built, companies. fraction of now of

efficacy of with community some DMD the in the data the have Extension HOPE-X HOPE-X We and terms and Open-Label safety datasets. in most the strongest consistent of and

a to goal strong investing engineered organization is and have and have Deramiocel supported to to the continue are as exosome deliverables forth, Capricor's our in commercialization StealthX. bringing pipeline continue across potential for We endeavor. our efforts set milestones judiciously by to technology focus that on as its we prepare we towards meet

run they which medical, finally, will next of our their and financials. time to the to from conferences, we want further DMD at patients, thank available. presenting Later plan the details be continued be guidance over And at investors of our become various investor-related now this support. turn formal will of will upcoming call our will able several on course, to AJ I articulate announce to for we to which pre-BLA the the families, their our scientific, be of milestones all we Over quarter, details as program. I more months, meeting, shared pivotal and through

Anthony Bergmann

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

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

me Let with start position. our cash

our and $XX.X June December million XXXX, approximately of current operating approximately totaled plan company's to the cash, on is into runway be As XXXX. the cash additional to Distribution first expectation on agreements expected $XX.X million our and potential cash under company's XXth, equivalents, Shinyaku. Nippon of This XXXX. compared any payments operations Based quarter to excludes XXst, projections, support and marketable sufficient securities milestone Commercialization with

for $X our of approximately the XXXX Turning the $X.X revenue Agreement and recognition U.S. $XX exclusive second financials. now Commercialization the The we've with for to million source Shinyaku. million the approximately were second received quarter Nippon the from Distribution million quarter of XXXX. ratable is Revenue with compared of of

$XX second primarily with the expense stock-based for associated in $XX.X half loss Moving to compared $X.X due for of approximately approximately compensation, to clinical And XXXX. million XXXX. compared our administrative XXXX million, million for excluding XXXX, quarter XXXX. approximately first operating second expense Again, net for dystrophy to million, compared quarter second net was in to our was $XX.X the R&D in program. of quarter Duchenne and manufacturing net XXXX was excluding increased loss muscular of for of million approximately the loss $X.X approximately QX a the expenses $X.X compensation, $X.X million, million was was approximately The of $XX.X loss to our million a expenses QX QX and costs general approximately XXXX. Net in $X.X the approximately and our first of increase XXXX million stock-based of half

We will now line open questions. for the up

Operator

[Operator Instructions] Pantginis Joseph H.C. will first And your Wainwright. at be from question

Joseph Pantginis

Two questions, don't if mind. you

talking just with filing. you get it met still? into wanted of to the or extent And this alternate before So about cardiovascular the deciding consider on do your know is outstanding you what you meeting? play the course, key sounds And is what aspects key update. to comments, latest latest were the did issues X this get questions and the prepared I FDA for minutes, I like the Linda, in paths guess I you're the

Linda Marbán

many aspect from program get is Joe, you cardiovascular going attention deserves these you. I to for FDA. getting really line, years. that think of the Thank the finally Yes, support over and I'm the across we're it excited. thank the this the The super

different really boys the and highlighted of it's is combination have young due adult-based advocacy the KOLs of community men. to #X them that yet, And these to death and dystrophy, which be This Duchenne think are are all might cardiac with disease, the who to first efforts muscular this is joke, go cause of of therapeutic the cardiomyopathy the might that has the that Duchenne grandma others. therapies associated get. I ones to frankly, some person that something your would pathogenesis the in extraordinarily from kind treat available

the a patients become as in Deramiocel of with the least yes, They're muscular thus program. willing willing label also, They're label consider cardiac. expanding for key to So far, diagnosed writing, at it focus has dystrophy. to the part of consider all to Duchenne

to and excited really the results we're BLA these push so, of very quickly. And to see continue minutes towards this

Joseph Pantginis

could it's program. And broad if reaching has platform to switch excuse swiftly, just exosomes Obviously, that me, a I the capabilities.

efforts, say, guess, how So reaching of outbound of, you BD proportion I would describe to mentioned you with your versus inbound regard sort BD? the

Linda Marbán

Yes.

coming So the exosome really is program to light.

program appropriate. other that story And in I know expanding sure is competitor follow cells, indications expand because you and was our lipid thing we with critical. numbers into and to that and as our that do long would experience cells easily nanoparticle. a making exosome-based others and cell make using that that make into we their and be was Deramiocel because cheaply launch But therapy first large to relatively goal think the exosomes had main know could my of an

because nanoparticles better you way and can are load than lipid exosomes them them. Now target

plus they're liver. safe. And very so, a clogging lot up there's more opportunities your not They're

So were that. our for thought long quiet first goal. that was achieved People a We time. we

targeting potentially some very of and moiety. exosome, using now out we're skipper opportunities, into a getting antisense skeletal instance, exon This it the muscle that tangible Duchenne-based including for and our to product. loading, about targeting becomes a these Now with

also interest extensive so have pretty And than external incoming business we've 'XX had reaching development, before. we yes, more We're in planning a efforts.

Operator

Leland Gershell Next Oppenheimer. question be from will of

Leland Gershell

If complete thereafter. understand, the you any Just you would you long I on study hoping data here. the when the clarity rolling submission? that? BLA after data to the pre-BLA around and to coming have to of HOPE-X and will the expect begin you to line top meeting submission? begun that up some from do be aspects mentioned plans And the rolling wanted get Linda, the timeline expect how not,

Linda Marbán

Yes.

would plan as we our that our submission complete would next XX the within the days. the BLA it first seen recent FDA meeting. So plan have of communication be end the part to submitting at be of rolling current is quarter. has most HOPE-X BLA, at for timelines the approved begin the Should in and the our anticipate necessary data our

agency, to opportunity data, XXXX. exploring, also would the HOPE-X submission obviously with and file are that end the We then complete without to of the the however, the accelerate

Leland Gershell

question one having sort partnership. kind And comment of may ] evolution in understand place or based make Wondering, Just those a you in if frame? more at of partnerships if to you view to potential be on on what any this the as the [ then discussions, of by can where any, year-end? time will ex-U.S. discussions your the of there's be want

Linda Marbán

Yes.

goal Our end the year. is conversations finalize before of to this those

with sort I we with As EU wondered, consultants also been a said, clarity regulatory know for I Europe, conversations FDA been of while. have And beginning in we've talking The authorities critical. anticipated, picture easier than what We people have to has going and partners it cleaner think take it's to there. a across much actually to get we be also. of so it's partners think the now going our line originally to much

-- deal balance sheet to Deramiocel. of support would to these expansion approval goal and the be the before end the the from and deals So year a strengthen close to calendar this

Operator

Next Aydin be from question Ladenburg. will at Huseynov

Aydin Huseynov

name sounds that It your CAP-XXXX the with Congrats progress, finding meeting. discussions another Deramiocel. the with FDA for positive and

the a couple regarding have FDA questions meeting. I So of

indication. have, there's that if was trial see FDA any Becker reaction of some I just mentioned to discussions or curious about the of any expansion you thoughts had design? you to preliminary dystrophy hypothetical expansion potential DMD and they any feedback And that So

Linda Marbán

Yes, expanding know been while. Aydin, into you've I big of us really supporter for DMD a a

for thank that So guidance. you

our cause cardiomyopathies a instance, of we into for for as focus part to and main with it really of on gives We that obviously, the been understand the have muscular FDA, right really death. mentioned leading a is FDA also beginning is meeting. dystrophies, it is how conversations impact as part window in can it hasn't forward, the move now us cardiomyopathy where us of Becker, the these The exciting

talked really FDA I see where stage yet, trial this strategically and haven't comes get panels with We meet about that the on speak. program. and because We KOL DMD so year, guidance, putting cardiac we the to before of with design sort their we're build FDA, we implications, want down, as through can our get then it a and plan end of plans together to

also is the So great yes, the for obviously, but with a this therapy, patients for opportunity, Beckers.

Aydin Huseynov

DMD would you HOPE-X and of about potential label would will should think only? Do similar ones this? sort you FDA the for do DMD include be think label? that how they that the you do you we approach Or And patients Deramiocel, trial? specify recruited FDA patients broad it pretty to in think may label the for HOPE-X how And the think with cardiomyopathy

Linda Marbán

Yes.

So cardiomyopathy and/or, positioned be the right? going that's it. So how we've is an to

published, muscle muscles. as because I muscle has progress cardiac know and will that's been be similarly how not both the skeletal read, those disease the does or skeletal or label skeletal for myopathy you think cardiac cardiomyopathy with well and in will it and

versa. So and muscle good have bad some skeletal kid heart or a vice might

cardiac. stabilization skeletal opportunity both an and So in for we will of be disease

muscular all as has Duchenne that our that that preliminary which approval into hard opportunity will a market some been gives well, open have double the precedent we our the others In very can. And terms dystrophy, open push as as that to at could of suggests that labels, opportunity. by labeling, broad door from is that they Certainly, set space our diagnosed we certainly in of are the launch. gotten feedback opportunity with with goal FDA those us

Aydin Huseynov

regarding advanced of And discussions Europe. regarding sort in partnerships the

deal cash fourth economic or is quarter cash you the So XXXX the before readout? or would more question. like in the now readout a later? So topline to partnership purely an Is pursue after this it

Linda Marbán

coming question. the interesting appropriate, an that the it's the now. are BLA. that to talking We're and We almost Well, feel our way offers of reasonable right assuming are partners approval

or Europe has with very structure. of over We're another there meet trial. conversations on trial their now either based sure regulatory not that confident with some clinical criteria, no our authorities we So yet. We're will small clinical

so, is the to to economics the to short to sheet the support your I best it close the give reach we'll answer when also we forward, And drive as is our deal to balance credit asset question company and the guess, due.

Aydin Huseynov

last And potential have the I the Shinyaku. from question regarding is milestones Nippon

readout million there's know should before end anything anything waiting approval? Should we up $XX the year? of until we the we the BLA FDA on But So at based expect approval. topline expect this

Linda Marbán

you payments. we're of sheet them as able to personal milestone So actual disclose the estimations. our cadence know, We into the not balance built

those of really is coming but we're say milestones to now, approval aware that's BLA. right coming all up I can in. the of including million and so, there And $XX And

Operator

And question Kristen your Instructions] at Cantor. Kluska be from [Operator will next

Richard Miller

are DMD about? is that it caregivers basis? talking about therapy you you the is hearing as got for non-ambulant and for this, to an relates on especially issues these space potential from confident you Kristen. on This Miller patients physicians We've the in for How receiving here. gene were Rick X cardiac What accelerated now

Linda Marbán

great question. really Yes,

least with disease. And mean, pathogenesis a dead gene I will for with and therapy or its actually are Deramiocel better it to it X on reasons. certainly able delay were the as those processes in a performs non-ambulant multiple year FDA closed-door, X of when preserve therapy implication guys that inflammatory that going therapy of of patients. The whether, of muscle year comparison well compared are what's presented Elevidys therapy than gene yes, to potential for if their we there. We I faster? is hearts a actually show laser-focused and gene that X, the their Two, in to fill seems heart. pathogenesis is non-ambulant. at on preserves will is gene And more DMD, Deramiocel as for be a work, and the dead what's between Or knocking real the And guys you implications there? hope are problem the about gene are worsening So hearts sort muscle? everybody you and these the post-gene therapy going really A, therapy have of and part becomes patients the correlation And concerned with to cardiomyopathy on active, with as in there some more One, could put negative to and are cardiomyopathy. strain physicians a on

this which treatment for I So microdystrophin that is no with negative preclinically think in opportunity Deramiocel great treatment. conjunction a impact there shown of is have Deramiocel,

be And restore they great protect gene it. together partners we so believe and with will let that therapy, the the muscle, Deramiocel

Richard Miller

cardiomyopathy help us the more Can think you DMD. Maybe Becker. about naively Switching from of then thinks as sometimes I that severe you're kind us. field frame Becker about being of one to just less of a opportunity here? form the sort question thinking

and more severe, the as thoughts of relates to kind sort more So Is breakdown how does cardiac of burden it DMD? there? it proportional, Becker

Linda Marbán

pretty a leaders cardiomyopathy a KOLs, of for dissimilar year unsimilar actively and heart Duchenne the focusing begins cardiomyopathy. XXs question. is some certainly, to to skeletal and muscular die at the PPMD's not The or Great hold XX, failure a guys as lower. age in the on. most once Becker about of talked basically result participate opinion them actually The to, the muscle key severe These Yes. with so cardiomyopathy end both up we myopathy cardiologists heart disease. cardiomyopathy dystrophy that Duchenne. paradigm we've meetings, which of Becker And of one XXs and According they that progression laser

that Becker. death. I XX, the associated at becoming cardiomyopathy either. think cardiac will And XX dying It as a wants severe potential the it's yes, versus mediator to XX dying to treating the in door, is a die I terms in aware question more open with the mentioned field of dysfunction of at while as earlier, now at whole of nobody less

would guys So go to And keep Capricor I back over to turn those final time, thoughts. for let's this like onto. the management at call ^Operator^

Linda Marbán

through attending Thank I continue thank you nice on our all XXXX. Have talk and look programs, forward to you updating you as you today, very evening, for call much we'll our and for thoughtful earnings to a your soon. questions. of we

Operator

and that does you. conclude your Once you do call this attending. And this please lines. gentlemen, ask Ladies your Thank indeed again, conference we for at disconnect for you today. thank time,

Capricor Therapeutics (CAPR) 7 Aug 242024 Q2 Earnings call transcript