Earnings call transcript Q2 2023

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Operator

Good afternoon, ladies and gentlemen and welcome to the Capricor Therapeutics Second Quarter 2023 Financial Results and Corporate Update Call. At this time all lines are in a listen-only mode.

Following the presentation we’ll conduct a question-and-answer session. [Operator Instructions] As a reminder, this conference call is being recorded.

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

AJ Bergmann

good Thank everyone. you, and afternoon,

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

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

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

Linda Marban

to Muscular clinical regulatory our as advance XXXX, as Dystrophy progress continue our begin of conference and exosome second milestones pipeline medicine to Thank for we deliver you, by continued first well patient across steady commitment XXXX Duchenne technology. joining deep positioned like and platform and make the program, half AJ. our to our optimizing quarter well for in our and afternoon, Today, you reiterating I Good call. important thank would on to to focused are

Licensing Philip and Gotwals has pleased recently Institutes and the to and Ken strategy, Development experience Directors Hopkins over infectious Johns Professor of deep Medicine, development, Ken years be Gotwals Business for Dr. the business Medicine and Dr. Auwaerter and members and research, at University Center doctors internal most serving Fisher Infectious the Director and Diseases. disease Sherrilyn of as with of our of Head will to our our additions School enhance Paul industry of of invaluable Biomedical as across Research of served the as Director development. with of for experience I Clinical Environmental the Vice and the years additions we development Dr. Fisher Gotwals business medicine am of Board of continue Auwaerter. XX brings extremely recent programs. and Novartis as Auwaerter and the two the Both new President XX Sherrilyn at experience Division Infectious in both corporate drug Diseases nearly for of priorities Global strategic

more including details X give as stage the Our to Phase trial program continues minutes, on through in DMD few with in of development patients late development well our I which clinical discussions our we with and to of priorities, key momentum, HOPE-X continuing CAP-XXXX believe clinical enrollment advance this we remainder next of year. well as the Building application. path objectives towards on the positioned a biologics -- regarding throughout the execute submissions executives license the are FDA throughout proposed corporate will

recent our on into now an dive programs provide and activity. will update I

our the designation, we our Let of have our Earlier an was Type-B outcome me as Controls to recent commercial for this positive year, Chemistry on update this Manufacturing provide and where or CMC, with RMAT FDA FDA, related interactions. part deliverables meeting for you of CMC of production outlined FDA meeting GMP that we the us necessary clearly portion The scale of and our plans CAP-XXXX. for outlined BLA.

we previously this of site. our Diego be a reported, at with who patients need there GMP commercial to San product the product inclusion As treated was discussion concerning to additional have would from facility possible also manufacturing order support in

request have close importance continues raise extended to (ph) if FDA and of awareness of that patient determine program [CBER] population. this for subsequent would highlight the there to we FDA, more path advocacy specifically they they CAP-XXXX disease timeline continued with program unmet therefore in the FDA to clinical to and requested expeditious the request trial The was attenuate as pivotal this and year. medical our July of continued perspective, progression, We efficient program. to completion meeting this great the that the the of discuss end, do, BLA. of our to believes, a to and our more have the and To this as our within to pay As meeting in BLA Type-B granted have likely time a due the to need community attention patient our And occurred detail. FDA

DMD. month. generally The outline goal a plan. we are Currently, minutes of BLA happy an this the from supportive path for we for of later was expedited I'm treatment which to to And final stage for that share plan CAP-XXXX further to awaiting expect FDA, FDA this the seemed

optimize potential appreciative aspects we to guidance had of to certain We are -- FDA another path FDA's HOPE-X informal support trial greatly have approval. already appreciated our of further design of design our the to to the clinical and meeting program, with

the active on track positive an based patients HOPE-X pleased summer, size patients, throughout of an Phase the this strong that year, am we families, very we by anecdotal from of remain early sites. in XX you enthusiastic of this focus interim XX very to are reports clinical Next, which randomized Furthermore, X I'll rate year. am of on the our update has about fourth propelled quarter futility and I today, And sample a including of to which across we I'm the the on designed analysis. have enrolled on on conduct be track quarter disease fourth fully of provide trial. will as in to currently on attenuation. enrollment inform HOPE-X, the been primarily subjects analysis I XX feedback

the Additionally, extremely and pleased of we by of overall safety the support profile families. investigators our CAP-XXXX are and

Now or update conference. for you, remind label follow-up from significant presented this extension for OLE an results To annual this open most presented year's statistically HOPE-X positive recent results Dystrophy study. at our on two year Parent Project Muscular we

X.X in from suggests HOPE-X time OLE, we As year. primary nine you of version may the year at the treatment fraction version one we time decline group extension observed in improvements performance correlation a history steady OLE ejection significant on Over the compared study scale. profound open month recall, measured the observed its version upper statistically X.X and of of function differences DMD fraction. label of natural function increasing HOPE-X Even the in to data an results. with six X.X the by patients. were more the in in also after show the the the previously the ejection heart In cardiomyopathy performance HOPE-X met time, to continued findings XX as points while cardiac PUL endpoint original observe, At of rate point, when the one cardiac we upper group the PUL limb placebo of limb decline the

which potential patients, of label a positions skeletal impactful tolerability The further for term long as the benefits, so this with CAP-XXXX the in results year are from safety The potential, treatment in anchor favorable disease tremendously cardiac and profile, patients. functional study DMD DMD. potential modifying CAP-XXXX for DMD underscores open two benefits and therapy together

their commitment potential their We are thankful to CAP-XXXX. benefits continuous demonstrating to for us patients the on with working families and of the

strategy. Now, turning to our partnership

and our Shinyaku, additional Japan, continue in with focus we Now commercial U.S. secured opportunity. Nippon on partners have in that the evaluating markets partner distribution the other we Europe the being now have rights securing companies key and a several around to world, and priority with

focus to additional indications patients. provide would briefly Another touch CAP-XXXX evaluate might benefits where is opportunity upon to the new I to like

committed even any leveraging soul have capabilities potential to partnering, Capricor While resources the area development, another for opportunity on this built this licensing, we CAP-XXXX. development avenue or would haven't for provide at yet, we

an Lastly, our like I facility. provide update to on manufacturing would GMP

the will facility this quarter cost built we've greatly effective margins designed to we this underway, facility increase this is to release a the clinical and remain in-house bring doses of our efficiently. In up this we facility in to able be launch third runs year. track support Diego product. early San am approval, to are the of manufacturing see and manufacture the obtaining to to GMP engineering I to versatile commercial facility that way running, report and having on scale this We and pleased as doses. believe will CAP-XXXX market CAP-XXXX anticipation market produce CAP-XXXX We GMP because have of ability

of interactions with new to pleased look and our our progress program. partnerships DMD FDA, progress additional with potential sharing very am HOPE-X development I of our in Overall, with We territories. from further the the updates forward

new Dr. areas President regulatory Waive previously with has with Vice Kodiak overlapping expertise. all regulatory role companies of focus. the Feng Each Yushi two at Feng his worked Biosciences. have -- senior internal have Life Sciences, of overseeing our additions overlapping team We CMC leadership and FDA, enhancing and to activities. our these as our assumed of Regulatory, areas Dr.

included of CMC to Operations Program Development. for experience very role Tayco new lymphoma. welcome members in integral I has Business Vice cell approval Mr. of treatments team. these Jonathan B-cell the one Tayco he the Additionally, Prior seeing Kite Pharma, first BLA where pleased to approved as President for joined an therapy large of played Yescarta, us Management two am over our and

of Now natural exosome which body. the communication leverages platform technology, turning the our to cell-to-cell

applications exosome modalities, system harnessing We of is therapeutic. two disease, of on StealthX precision which at drug novel serve to proprietary the program, and as broad the development are broad our core our with a therapeutic platform is expression delivery and focused infectious exosomes

the to that previously drug the delivery. are cell, are of be and can future we able targeted. exosomes system As deliver by avoid believe stated, directly contents detection to the immune They

the has a large similar is homogeneous volume to However, relatively formulation issues of the of that ability one lipid grappling with field nano to been manufacture a partials. the

and We a that efficient energy for development future have product. of I made and rapid shifting delighted on into This with these to you development significant exosome and we allows share targeting step amount of time and focused am have manufacturing progress objectives. paradigm exosomes,

To that looking pharma forward we this coming providing pleased exosomes using at of that neuromuscular more in months We an platform with I'm StealthX and vehicle. delivery as delivery in our our at the applications have very ASOs they end, on as time. enhancing the Capricor begin report work recently look look working a this very begun I as details undisclosed of company to into in targets further are for to primarily based platform StealthX. leverage we as expand important diseases therapeutic is to exploring the precision this step to

that influenza we in are vaccine Exosomes StealthX. published with to recently or SARS-CoV-X bioRxiv, data journal spike. HX a two were Additionally, generated engineered candidates express featuring

and the approach in HX a exosome spike immune injection. and When effects immune response. nano particles the cellular potential introduced a to antibody administration Hemagglutinin test both mice, administered individually, combination response. before Stealth on T-cell also a nanograms benefits This lipid adjuvant X HX the engineered spike were StealthX, both and obtained protein potent response production and vaccine. strong of of proteins surface individually or of with immunization and [indiscernible] with humeral mixed of the and These induced were strong SARS-CoV-X with without

antigens single diseases. potential therapeutic range Additionally, there a versatility these no infectious combining a platform support our of the results multiple StealthX and were broad of between competition addressing formulations, utility immune targets detectable vaccine and by in

and non-dilutive as continue partnering current strategies to platform our as for Our business exosome grant well focus technology. development on plans funding leveraging

closing, and our asset with our technology. the scale becoming as our development In organization company commercial we we focused with remain exosome are of advancements as a on CAP-XXXX across pleased lead

we experience CAP-XXXX forward We capitalizing of our platform. the the deep for we half strength. new vision on board to are and working continue of on I industry forward-looking and feel and will key priorities entering as in second position invaluable the execute their members a which be to year look with our exosome

Now financial over XXXX. to with will the Financial that, quarter for Officer, our of our Chief the AJ I second Bergmann run through turn results call to

AJ Bergmann

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

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

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

Turning second was were XXXX. of $X.X for to ratable the U.S. quarter approximately commercialization the from accordance revenue for payment in the quarter compared agreement million Shinyaku. zero of the we from Nippon our of recognition million source second primary of Revenues financials. exclusive XXXX with received the upfront and distribution with $XX The

and Again, approximately compensation, in general million and stock excluding in QX, our $X.X of compared XXXX. expenses to quarter expenses operating approximately million million QX, million compensation, expense $X.X XXXX. for based was research stock based XXXX, administrative our approximately QX, $X.X Moving development the to $X.X XXXX, were approximately second excluding our and in

the net first compared loss to to of million $X.X second for of XXXX the of approximately approximately first half $XX.X net of XXXX for quarter half approximately loss of compared was the XXXX. was a for Net million Net quarter million $XX.X approximately second $X.X for loss of the million loss XXXX. a

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

Operator

and Thank Joe comes [Operator we Pantginis the question-and-answer Please now Instructions] line Ladies go Wainwright. H. Your ahead. C. session question gentlemen, first sir. from from you, begin the of will

Joe Pantginis

the afternoon, Good and for AJ. Hi. Linda taking Thanks questions.

will a revolve out but FDA things the still some to I my minutes going yet, there's you lot not that not because might around And be So know able working I questions ask here. parts know to detail are of I'm them.

start comes San heard quarter, if with that this would a correctly, of number the from required range facility So you that do that Diego I be patients guess, as with? of to I general said, online have you

Linda Marban

patients' can Yes, final a you. press release to of you're until is to what this careful to ask have exactly the and say plan as I on look forward. Good via in get and/or help we several closely tell tell Joe. can us. some of to program, take are either market doing FDA has we with out Hey. we get FDA we that What focusing in update worked and talk supportive reached correct. way path the sort program a once that, the is There's release feeling But can their you And, at move I really you very that, the very better. to our forward of feel They're little minutes. call really to Capricor, them, them families public to a order community the because they sons

the the confident possible answers towards FDA that we back ones the move to best And that get be will products BLA. so, from we're

Joe Pantginis

the based. same for That of makes sense. of in sort I'll ask it guess scenario I question, And, sort realm, my it's next but

right have the you type -- So, minutes you now. do that have the do you're waiting

which said You upcoming on analysis primarily interim quarter fourth HOPE-X, for futility. based in you the also the have

does you from -- with to or for the timeline one include the for potential that the see resetting interim HOPE-X. positively the in regard minutes feedback might your the of of allow the in could scenarios However, data acceleration the positive

Linda Marban

you've was question impressive Good a a long That work. asked, I tell time. pretty this doing can been Yes. Joe.

has from -- the too, in is they trial to metric they pretty feedback from can this that because at tightly we for us analysis. with that So, for us. is, want futility monitored, the been regulated, looking the their us the interim carefully to answer tell product that really FDA believe very keep we're clear

about. I definitely accelerated we accelerated focusing opportunities thinking might approval but earlier I an opportunity. laser to so, probably an something we're going on able that I to is use And that tell advantage cannot be look look you how to we am where the may interim not can do for now disclose we're take approval. of for that,

Joe Pantginis

And are are it. logistical. Got more guess other I questions -- strictly my

you're failure rates or look also screening What for that if the the correctly. potential patient the conduct patient enrollment? for by patients are HOPE-X, reasons of encouraged what been of understand does in the the does obviously what seeing, enrollment and prominent pipeline So you've I like

Linda Marban

question. Great Joe. Yes,

rate, the upper screen two that five. limb really very to to strong our thanks And team that new they So fail in people of primary has come are of reason lot, fail which clinical is inclusion the performance we a place. really have don't down put that score the criteria typically

these human of scores their brand heartbreaking ability some because probably see people CAP-XXXX use use -- know, if the have hand boost typically their mouth we or And three their a to they me we saying, two if from whose hand what are so, it? it's to let other desperately. say have people they too just if guys level, they And and well, to what So they from And get help, week calls unassisted. to a want need you low.

is that second So pipeline with [Sarepta] to meeting, far were answer question. gene of The PPMD a by only the potential this busiest second the very At patients There's the around data. to extension your booths, label energy we (ph) of lot one open rich. therapy. into the leads

to with as along of CAP-XXXX. therapies looking anchor could therapy so, with gene that we for And Sarepta come the all are build

the it so way well. that And see families as

any see, forward. moving So turn -- we down do potential downturn downturn in not or opportunities patient

Joe Pantginis

now us newer wait the of arena or regard with in teasing a you've this any away DMD, and for was CAP-XXXX going or potential the from give cardiovascular of it. And here, and a you to to then I again any might is indications worked And any indications Got want bit back opportunities. tease see? prior us on about little give you additional did that going HOPE-X lastly, to a curious just

Linda Marban

Yes.

is, with to that best answer So, of and CAP-XXXX I are the very think the we way progress DMD. pleased

I that strong after a very effect to years. there modifying very think it's seems persists disease clear a be that treatment and

done have well really on patients Our exposure. a repeated

that We treatment. have extension of in label are year their into patients third fourth and open their coming year

safe. it's The so, And are very easy. infusions

we the to to So look updates for potential have deploy those we CAP-XXXX door internally opening and as are likely provide now at a where exploring we them we'll opportunities We're next. CAP-XXXX. will

know disease, immunomodulatory I skeletal you crystal sort seen we've if data inflammatory action primary and But into disease, neuromuscular CAP-XXXX’s you're we regenerative. can ball, your mode an of muscle is what pro of really nice tell and looking cardiomyopathy that

going to so two processes. highlighted indications continue be to pathogenesis we're set So disease by explore those the would

Joe Pantginis

Got it. Thank you, Linda. Appreciate it.

Linda Marban

Thanks, Joe. see care. you soon. I'll Take

Operator

Instructions] [Operator Please from comes question ahead. the Your next Long] News. of go line from [Alan BioVoice

Unidentified Participant

and also A.J., wonderful it's to way shot question. And the back your what for commentary. a analyst, Joe, and extends great the hear commentary to release last great it. to Linda, be love beyond out

a have sets couple I of of questions.

really low into products. You're high-margin volume,

the usual taught template manufacturing Although your year any you Linda, provide into pilot-sized pilot for wonder simply products the other Is a your my you if path Capricor's seem I forward? scalability. for facilities, vertical replicate on integration? could modus considering in this to model commentary could breakdown future about In are you sweet possible spot. that be operandi You going that? words,

Linda Marban

way, And to talking, manufacturing expansion? to Are or it's Alan are you're of I pilot -- primarily continuing you hear well. about exosomes talking CAP-XXXX your by do you But hope in terms voice. the great

Unidentified Participant

footprint a what need. accomplish the understand to for I need If you don't you large Both. manufacturing, either

Linda Marban

Right.

So they're similar, but different, obviously.

been out and that's opportunities to scale manufacturing of CAP-XXXX, in so reduce been sales. transfer necessary that scale and GMP effective, the Diego we the pretty the have footprint place same to more That's there product closely make with we're largely put focus several the San into the really produce really and now we group the get create with can in working that the working why and pilot we is FDA their We're and as clinic. order to is CMC out which sure facility, the right facility. in those So

processes be I we same. facilities and pilot which envisioning, an expanded It won't think will expect pilot into exactly will a as be, some manufacturing we XX I could little facilities. you're be XXX but largely facility, this. turn the expand yes, And is So take we it that little

opportunities decades. So couple therapy past manufacturing expanded has in the really of

opportunity So there's a there. lot good of

So not exactly how you envision, but almost.

In of one for of pride us. is the areas exosomes, great of that terms

known building than is the things going and big mentioned are buildable make also are other engineering plant move But it will one turn going in state-of-the-art you of a bubbling -- we my that's them about one not I exosomes makes from make As be done more as as to to mils to you and kind the you And easily prepared leaders that mils larger lipid to making of of held any can into we be have some remarks, and particles pilot going well a that How that how as really that have out. you That product? that halogeneous? couple constructs to and expansion. the the manufacturing how XXX are back our good are them to taken paradigm that. bioreactors believe really is of to a should of sort that, We've we nano thing that exosome like forward. go volumes procedure sense what

Unidentified Participant

It does.

about interest? further on trials? issues for general current this if gene general? comment general, Because the head. the much our pharma therapies. I've assertion two. read lot more cell potentially if right, on in my three, If one a There's commentary also the the on wrong, of that in Just CAP-is one serious and I'm equals XXX a literature, get to There's plus and Top not interests feel me some that those And and Can this, to combination space. I been You becomes are who from want you how bat go ear ASO more suitor of [indiscernible] been can undisclosed pharma I I the free interest. watching caught where

Linda Marban

in You mean potential? their partnering terms exosomes the of with

Unidentified Participant

Yes.

Linda Marban

Yes, absolutely.

is well this and is -- aware that's So biotechnology of of the pharma one in great it. opportunities

been can on and can is, everybody cells. even know something and career far. important lipid working this how field we my now say We've I only harness therapeutics we a science I you the take do and so body makes as that targeted own naturally that. to We to delivery, knows predates particles because think a of delivery nano that that

turned with made has in why specifically few a that's not CAP-XXXX exosome working mediate Capricor so perfectly they platform, past of an focus wonderful And the our exosomes. think by CAP-XXXX. towards engineered really years cell of an makes the mechanism action exosome We

we But a do to what really exosomes want drug product. turn into is

you manufacturing engineer what interested an the And putting in go which pharma course like that. business we want And right have exosome, you very main program of you is tell And in this code a put one succeeded it, so, outside doing. on in very where we have it it's this the the hurdles was on inside active you now. development step, to and and on

look with forward. we great to So exosomes forward moving the opportunities

Unidentified Participant

does things. question, interesting Last but it This to but you. I will get fun guess, this a AG, question, some be both of is for for

You got more the upfront million $XX agreement. cash from last

another that or grant another in how used? it a got you you agreement brings like would If $XX million,

AJ Bergmann

Yes. for targeted our the from Thanks, approach a potential as nondilutive heard obviously, I next vaccinology a therapeutics. as Linda as capital hear you. in mean, is Nice Alan. articulate, bringing is to which both from big us, you steps, in to well focus

big list. potentially into types neuromuscular as these that in in. I to I energy it? hinting articulate using targeted capital we Linda vehicle, would new my a new putting to if if nondilutive that indications nondilutive would of here, great a to would a exactly move don't targets, the whether dollars taking it's those else? my deploy that And that area is wish I had like be to at wish the of it list, know capital door Where want we're use lot anything be exosome we're mean

Linda Marban

Yes.

So as product development you in cash are mode. is king,

we And laser-focused use so, targeted to move forward. our would is in it do way programs what definitely a

the of the at right line front is now. CAP-XXXX So

exosome go being as All approved in we left then build major dollars work we a that getting the dollars forward. come dollars requested towards that. on the the are program over, being would and move spent and If few or specifically on CAP-XXXX obviously program, spent to

that's a deal build there a instance, go. exosome, good dollars those would if For engineered was where wanted to proportion they of where

So a alive AJ, able on through explains why we to and tight to some pretty our stay what in very resources, focusing need time. are of maintain we've managing been we terms that difficult end, to do, ship that

Unidentified Participant

in The forward the future look this bright to hear. is office. for Linda, walk intermediate both I'm and your what future, I Well, I in looks us pleased of Yes.

Linda Marban

for That's wonderful that's future you, Capricor. bright Alan. news, The is But is the if future for great news. bright

soon. other each see let's So

Unidentified Participant

Thank you.

forward Looking to it.

AJ Bergmann

Take care Alan.

Operator

be remarks. to questions management like seems Capricor There further call to Instructions] time. [Operator no this over at for back closing turn I'd the now to any

Linda Marban

and late-stage a DMD. Thank confident program for foundation clinical catalysts provide forward that you. upcoming our driving the and for We CAP-XXXX Overall, I am company's solid execution value creation. remain development on focused

Before my bring who the clinicians, appreciation and families want to their extend working are closer with patients sincere call, approval. CAP-XXXX to potential to I we today's conclude to us

your us and this everyone afternoon, now Again, disconnect joined to lines. thanks who may you

Capricor Therapeutics (CAPR) 7 Aug 232023 Q2 Earnings call transcript