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