quarter. in for have I conference of extremely Thanks, the progress last A.J. am you made joining third today's quarter afternoon, call. Good and everyone, proud we the thank
CAP-XXXX As the of you X treatment for for as deramiocel, working known years. we the develop last know, DMD to formerly been have
cardiomyopathy trials, skeletal the devastating associated of myopathy and multiple in deramiocel the the of solutory improving muscle attenuating disease. the We consequences have benefits with shown clinical in this
implications, We data presented the the as it improvements. unmet well data and available to a conferring as data decided the need BLA shown has of has is for meaningful after about the significant well have for approval as as become we file large
To FDA, we and have associated the the with strength on statistically medical consistently the clinically Based our FDA are here cardiac to excited full with the the progress of say as that an understatement. proud DMD. cardiomyopathy
call, us company stage listeners become some I new that of and organization optimistic. through very I is approval have the should steps outline we for receive developments Capricor we For position which and our taking will of on this a revenue-generating FDA am as to walk Capricor the you brought supporters here today the commercial
XXXX regulatory Now extraordinary like first, update. an to I'd for a year has been provide Capricor.
We work FDA have approval the treat taken to the to associated in collaboration with the and commercialization the opportunity deramiocel to for DMD. cardiomyopathy of with potential prepare
to Phase from we
I based point patient perspective. by Consortium a HOPE-X clinical a few regulatory to DMD now Cardiac level Soslow HOPE-X and to extension be BLA will Our compared take explain minutes existing history University. will open-label Vanderbilt from the data this studies Jonathan our on from at natural how data cardiac Dr. II led got
details approved And and study of noted clinical FDA open-label by our noted ejection therapies strength DMD. dysfunction diastolic to As Based meeting cardiac were to for associated initially opportunity to them proportion it that, and fraction And
The label as from unmet is clinical to the on and more in necessary of for available. approval. extension of and for was forward the slowing data that systolic is we to we and becomes part also end the the clinical cardiomyopathy No cardiomyopathy broad file August, when will with derisked significant I our of projected we already HOPE-X cardiomyopathy, here pre-BLA it medical of of is previously, the FDA, those have and move data became presenting with DMD. minutes, I deramiocel is cardiac as in economics those no full a the have natural history ultimately in importantly, on is encompass subsequent by clear a decided volumes. compared a that that as meeting of provide theoretically supported set, end measured need FDA. most the measurements with it large stated addresses data available DMD, on major the focus data data the show data with meetings index in trajectory it have which that DMD data. been there few is will
Capricor the A be combine and we myopathy and in unblinded And support the performance it on with for endpoint clarity opportunity be will And to important to be upper year-end XXXX. to as indication, which Therefore, XXXX. the conferring be Cohort while for attenuates HOPE-X FDA first serve limb deramiocel. supplement may elect was will or on data was data which the add this of approval deramiocel and have use deramiocel market initial the on is to as function, necessary, greater more of steps skeletal
Therefore, that we thus to progression. treat for limb set to the presents HOPE-X B skeletal that me after upper disease originally effect cardiomyopathy let muscle you label the may the muscle use are the skeletal of will the recall, HOPE-X X.X. of not in which far Now the A, have authorizations post-approval efficacy next a should future. marketing outside a muscle by to show designed and Furthermore, upper based U.S.A. we as decided that pleased of explain now to which HOPE-X cohorts pull slated limb is myopathy. primary with
summarize. me let So
for DMD We for and cardiomyopathy data. filing analyzed full are substantially a previously derisked with approval
from expect goes first status we the of half XXXX hear end if review date by And quarter We the of of FDA the the the a potential of regarding second to anticipate FDA well, for the application. set PDUFA XXXX.
to the of first treatment together, devastating am when the includes our most combine a BLA will clear that Capricor module B first-in-class thank point BLA on opportunity we strategy, myopathy all data A inspection, We to access one label. muscle by was endeavor. on which efforts consequences partner, I we the NS and want to for our to use report to This fully of the preparations package track are focusing
It potential are skeletal Cohort activities that is that of our team pre-commercial the the trial to of submit for and the DMD. year-end for the market and to Pharma. my for their I XXXX. this pleased of increasing submitted, treatment reiterate and unblinded a gives add extraordinary approval this achieve CMC and of work with distribution efforts power
that AdCom an for that we eventuality. necessary, internally are While not may we preparing believe be
of first spend individuals will highlighting with a cardiomyopathy. next would label. standard population disease DMD feature and few like eventually with is DMD, are the Now to Cardiac patient minutes the I most this have we targeting
of many, For begins very the insidious young. muscle cardiac breakdown
cardiac has understand process patients Please Some dysfunction they any and of XX. there to is XX standard patients therapies of currently keep decade ejection cardiac careful suggests XX% why approved do for States DMD positive. age with eligible approved, disease Because in like years mind, data reduced have fall people of payers evidence around most and have would are the therapeutic have has aspect XX% deramiocel with not DMD that the will cardiac no the on progression no of deramiocel. an be or The to to our discussions very approximately a X,XXX imaging of would in normal treatment these likely. approved for what fraction cardiac have significant function taken evaluation cliff DMD is medicines, and not before by that a appears cardiomyopathy a be age, the DMD and impact with cases.
If first-in-class United been in be off medications population are anticipating pathogenesis standard for not most of overall which we recovery from that initial then nearly
other would expect skippers. We therapies recently such as be approved reimbursement exon consistent DMD with
used of to and is terms both The months has X to progression be of most in are with groups, to disease one the we of every disease. from DMD, designed with is treating commercial with on transferring DMD. anticipating heart intravenously extension research in deramiocel. open-label slow administered in market and great advocacy anticipate promise, which those the deramiocel approved, patients approximately opportunity of products. cardiac chronically aspects for discussions generation and
If is revenue the groups adoption treatment Based Our rapid market devastating XXX we entering
with patient our for supporting capacity robust as we as as focusing manufacturing actively are and on Pharma well scaling payers We NS demand. working and prepare physician
and on turn demand, to those to meet in controls. commercial for and our front minds let's CMC sales chemistry of center Now manufacturing preparing forecast. manufacturing predictions is Based or order in
in small between in scaling plant we current in-house in clinical while facilities Angeles. our to As activities. the Nonclinical at facility. which our that you deramiocel initial Los was our Los Diego Diego planning meet for built manufacture Angeles comparability achieved, are manufacturing know, up manufacturing manufacturing manufacturing. in a at
We expansion aid commercial demand, San GMP future late-stage And and currently mirror one know San we we can facility
of to made of we the is we within the that for carefully demand reasons meet cannot process. procurement and facility able used for anticipate. to thousands transplant consortium organ year which a we to Deramiocel qualified From heart, transplant deramycel. OPOs one be of goal online is a from Our have a curated technical and or United hearts doses launch across are from human another of generate source agencies States
further. basis increase is time, capable product each with necessary deramiocel at individual of a not underway the activities are clean production. The on While manufactured modular this product room development to yield
is operations in San so so meet product to expand currently team to fully Our doses our Currently, continue is the underway in rooms commercially. we these frozen actively Diego at life clinically X and reported as shelf experienced generating stockpile last quarter, and capabilities manufacturing building, are can years, we is we meet of increasing clinical operational clean qualifying staffing demand. order The manufacturing Capricor and to demand doses. able facility to our
we for tuned they more endeavor. stay as for color anticipate being that available. otherwise Please these In become also inspection milestones successful are and pre-licensing actively on referred as PLI to preparing addition, important a
a a development used Shinyaku who and we shareholders this $XXX approximately have story testament our themselves States affairs this and to and October, validation with the used our the of which an I the access, will In as will fundamental the throughout well is comprised underway in turning of front. runway advocacy strong fully for We raise be believe are raising participation in of approximately some scientific very the oversubscribed from enhance am is market our in public us legacy stock, of appreciative completed already funds $XX to a be for we supporters into history. launch. gives a with offering for in and funds million our was facility deal engaged for Now with the of reimbursement, programs preparing new team prepare care Capricor this operations capabilities, to manufacturing These strong commercial institutional also as corporate Capricor our actively deramiocel. plans health QX this investors successful XXX million as our team cash Their which medical in positioned XXXX. the
Nippon we our as to and point. United commercial have launch. management a support potential expand launch to people common world. to new of Factoring to balance, top cash,
subject collaboration into with this them. of Shinyaku is distribution terms, Based enter the on to sheet definitive it we executed, term we a definitive We if of our European finalization work current would anticipated this the with be marketing, the in in continue Furthermore, deramiocel Nippon payments and a potential to entered for sales $X.X Capricor. agreement. region. to milestone Nippon quarter, we agreement. agreement agreement U.S. have similar European the totaling the close earlier If Shinyaku, into with for for region a agreements billion to payable would the on
flow to development, entitled of sales of are capital and we into cost with product, a goods organization cell of U.S., future robust company product us share XX% a market leveraging on the commercial our world-class the build
This to sold. opportunities, between to on pipeline fuel organization the cash enable revenue a product positive revenue-generating and in and therapeutics. based to addition, In will the continue XX% inclusive of commercial strengthen exosome-based of expansion
exploring now into muscular In deramiocel cardiac addition, and been expand Becker have guiding, the the of as actively potentially to dystrophy to that similar DMD. we very we as are manifestations are opportunity with
DMD XXXX. to believe to goal important current continue an cardiomyopathy We to be for and in beyond. our expansion potential plan transformational And deramiocel's make for is a treatment indication
on Now exosomes you I'd like to an program. update give our little bit of a
next-generation our StealthX platform. our drug platform While our been on as has of deramiocell, advancing delivery to primary committed part exosome technology remain focus we
pipeline build be and targeted. products, which of We have planning to drug been can exosome are to engineered delivery a enhance
We goal exosomes products the on our years clinic. cell-based with bringing building of capitalize to of are able to the
focuses our StealthX to have develop which use the will cost goal vehicles. program is therapeutics a to Capricor, delivery Our to technology delivery outperform could The but and only be and lipid vehicle exosomes on vaccines create effective. here a products harnessing as also nanoparticle us not by of efficacious that developed allow at
TFR studies, our on targeted this presented a loaded skipping. achieved therapeutic these in goal the that data a moiety and and using for preclinical can on preclinical showing surface opportunities.
We can now enhanced have PMO focus platform fundamental We into exon into properties be StealthX we translating recently
enzyme We also life-extending doses show nanogram an protein. have able successful mouse using protein, suggesting been in ARGX and uptake replacement to of knockout of utilization the
for IND of therapeutic based an for tuned updates this exciting on more data. Please on a some program planning are for exosome We front. stay this
phase pandemics. to to with are vaccine will trial. the fully the candidate our conduct in of first plans collaborating Project for Allergy in fund vaccine, NIAID, I a Institute and the which to candidates our test prevention Phase prepare of future StealthX Currently, States we aims and manufacturing StealthX Disease deliver then NIAID
The the United to vaccine is NextGen, COVID-XX Infectious government's quarter Regarding National and with which for clinical XXXX. is
to collaboration the vaccine safety a generate data, proof-of-concept proteins if the efficacy rapidly the their We platform of protein and pandemic to been efficacy, a of protein and adaptability data as for chance to support adjuvant. they II mRNA efficacy Recently, first-in-human adjuvants existing evaluation. our and much study. of been potential available in you, vaccines In a toxicity that decides about Capricor. remind This StealthX of known our there -- communities. broader the new StealthX power the us presents expect subject would vaccine viral a the opportunity of the of further speed on showcase as enables for for a to consider and only this native this vaccines. platform changes serve our vaccine which the in with some vaccine quarter criteria scientific NIAID's mRNA, epitopes.
Further, uses concept of continue NIAID have tremendous to successful, second development the for certain Further, technology, but ado combine we greater will in and quarter a not transformational XXXX, our to Phase preliminary has If To vaccines. conclusion, comprised adapt is to meets our safety reliant not has or vaccine for exosome no business see proof clinical and will
fully deramiocel with families our of deramiocel. made getting to see are regulatory and to and efforts significant pathway, closer those power approval our of better. helping strides potential lasting function the in in and Patients of supportive We have feel DMD
execute has recent Our the to on milestones financing secured for forward our foreseeable our future. path
and Disease Over we the Healthcare Global the this presenting later Rare to Piper complete various be we several on including Mover Oppenheimer Day. importantly, investor-related Sandler months, are and will year. the conferences, our of Conference next BLA track Most scientific submission at medical the
efforts our their meet turn as as continue for for across to have and and investing patients, to their organization we prepare our are and our is continue focus will milestones support. investors continued Capricor's we deramiocel commercialization want to judiciously deliverables families the A.J.? forth goal financials. the to call bringing I Bergmann on now A.J. its thank to that Finally, I potential to towards endeavor. over through the run set to
