everybody it's R&D pleasure much, very have our a to on you and chance progress. update Thank a Christophe, to
following X that is next years, accelerated expect quite transformation, phase exciting R&D we where which progress of stepping last For have who that in made we're it's really an pace. data those phase, with frequently, into and we've be our delivering over at you huge us been think like to rollouts the to I'd a and
spend great a And through have last to We programs. chance pipeline. just here program, of I momentum filing some time on of We key X walking filed, completed week, our Wave you have actually highlights our TAK-XXX. in some the first our X
into just R&D, few, COVID respect CMC Before effect just pipeline. to we jump brief with a on quickly slide, this research, and updates, our
on buckets drug trials. really and our supply Three pace were our successful any, to ensuring now in quite disruptions. our We've we've respect With CMC, prioritized, and pretty research of of we example, at track we've minimal, where clinical for in supply we're really before been a clinical maintained if that drug CMC COVID. much activities. For all back trials, for organizations,
And first before our started, we on order lose really as that don't those nothing if The time time. delay be we time X- variable. trials will as COVID, expect that trials Trials then were of prioritizing all. projected hadn't is focusing about the important expect And were that The in it in completed a somewhere little any, X enrollment lines. studies that at ongoing, that could delays quarters. to X-quarter we the ensuring to X to be most on lose we're to
ahead. year So really exciting
the Christophe X mentioned, year submissions and we year. fiscal potential have As over in this submissions X potential next
for In franchise. the for we as you Orexin from have addition see readouts, in then a an inflection critical also pevonedistat data our can data to for pivotal time the Maribavir, readouts and novozertinib, submissions, line,
see CAR-NK in just We I'll expect program. X proof-of-concept significant agonist TAK-XXX, momentum for for Orexin our TAK-XXX that And then a second. our explain CDXX or data to oral receptor CAR-NK
the left, but it's with had sumo we've not the first oncology X progress humans. just inhibitor quarter our decisions. begun look our cohort pipeline. the X made pipeline, Wave over lower in in also then TAK-XXX. we've past made you some strategic We've our We've As Wave for And expansion X
rare we a we had in. can of redefining our and really the refocusing diseases that As those hematology see specific focus want area, genetic to right, you on
do Hem in lead a decision these our project, visualization, X With on Our the b to XX no by to as next considering filing taken term with a we time dynamic said, of slide, AAV therapies. competitive static therapy programs in given several of Hem dive If land our our bold move go over but gene of focus to best for lines. landscape. we please. relative programs I at suspend course they chance will that sat a be the have to programs approximation, into the And as we terms long our look to we'll will these of presently. enrollment the can, we assets way as phase, best mean and made we've their we're where to the minutes. next pipeline the Again, to genetic pipeline longer in I where said, what
see in over the here. some You boxes dotted of movement the the can quarter past
Anderson, and that change from more had a is bit we approval of 'XX point pandemic. to 'XX. a I'll did the aggressive into single-site Christophe we to able study out hit. moving As we in we've the one FY lines COVID line time because Because pipeline, mentioned, TAK-XXX to TAK-XXX to that our time This program, for in unfortunately, for detail. at chance weren't was our that targets dive I'll take that and The it study in MD FY just Why? added hit enrollment a have adjusted very its minor
some formulation we've the line with will that pushed that allow identified we flip had on the for progress We've So program. a time back. cryopreservation. Actually, side, quite
initiate now have multisite that we early or XXXX in the next II of supply trial study and and year. we will to expect IND Takeda, submit run a study an end a Phase of at fiscal that year XQ this fiscal off-the-shelf at capability, use we that So to hope that that in
as So good progress there well.
next go So the slide, to please. if Yes. we
you pain all of set hitting So out. syndrome. of that for we new quite the interim see what the line The waiting of in successful standard regional some complex And arrows, have disclose with no this patients as But major we in program. and We reminding we actually our a indication this for you has detail in with pipeline study year. providing very decision for results. so milestones enrolled, we're been one by have can have that proof-of-concept difficulty. we are we're not TAK-XXX of care existing those difficult we've really milestone that It's completed a analysis just unfortunately, entity listed fiscal to this our green milestones really the molecular that and the that still
our focused next And global progress. advancing So on great marketed slide, portfolio. our brands we've been growth again, and please. We've made also Fortune
going China, we submissions, can to as bottom in Wave approvals China catch slightly frame XX submissions know, I'll concurrently here, I wish and is that the China. this to in X our were you slide, intent time global would able be the ambition our in make on in subcu. And be update approvals. over You The in in I out a as happen. call different this our that's at year, our with We we not and entity expect of could providing to parallel slide, up advance resubmit particular, pipeline see, new is of we fiscal to, in hopeful we have molecular China. ENTYVIO this one with to our And continue
FDA. with have -- strong had discussions the We really
is forward. in year this our path high dossier approval launch at 'XX. forward like. fiscal that We our latest, a what we we'll expectation believe have have that an 'XX. submit that probability understanding and of clear And We we And expect of bringing in looks a FY
please. slide, next So
minutes few of a spend programs ongoing activities. soticlestat. just let's And I'll with X our into and start diving So
in a an present the XX-hydroxylase, reasons, molecule our soticlestat a And inhibits only activity reduces is Cholesterol soticlestat for in mechanistic some enzyme, brain. So that's the multitude of of CNS. excitatory that
of in study Dravet the including Dravet we our just we had co-development that I'll difficult that tested relationship, Ovid, be The treat, our And this so we II start the on from for both we believe months and a were the are of a in III that likelihood the than quite most with number seizure disorders in results robust Phase excited we a high study and have study, Syndrome. more in a medicine effective would couple Phase hypothesis months that X both of now a Lennox-Gastaut. results these you intend fact more indications. condition remind as you where data we to developmental different in we were Lennox-Gastaut right, a presented Lennox-Gastaut have a disorders. but ago patients partners And is see we've reasons, on this by With of flash Dravet multiple patients medications. drug highly next and Lennox-Gastaut, refractory, at the seizure can to in
therapies, unmet a existing need. So many although area medical with still there's significant it's very an
slide, next please. So
becoming As leading It's partnership with will targets will genetic a alpha-X our antitrypsin-associated have TAK-XXX. more highly for is liver this is be had We're once-a-quarter We're fit RNA for and it's to, strong a developed GalNac-conjugated excited target dosing. be and alluded advanced We very This disease. us. and a low goods. interfering co-development a we're program. GI highly Incredibly of signed strategic validated antitrypsin into alpha-X anticipate liver, Arrowhead, modality a Christophe to in that established, moving and company. interested cost most that more liver
unmet a work that's disease. program. is we'll an need. through liver fraction It's is this population a specific therapy antitrypsin to like, which look as the we massive rare But that build the really with but our into will through program think a the where we element, as great a addressable us therapy. with we rare I development medical What plasma-derived disease, common targeting way a it's a use for step more a there's synergy alpha-X unit, second replacement have disease
and GLASSIA X, have ARALAST. we fact, In
synergies significant And can one imagine so programs. very between those
and of which toxic of in pathogenic right, is you we're abnormality. alpha-X knockdown, We the knockdown. individuals, Phase from the see that aggregated to target only reduction signals with target than not data see patients can data but reversal, quite And polymer XX% a are the some these believed the see disease, study liver some even forms entity. function of On the seeing be liver in We Ib profound. greater antitrypsin
put So we're really excited track accelerated it about this program, and on an we filing. to immediately
please. slide, Next
focus all the cell allogeneic possibility the our on shelf. cuts really the group. earlier, we that announced a our and cell mentioned quite across organization a Christophe generation great the our about our this is and of have cell moving more now in really We off co-located with research, ambition group. to of GMP with various building scientific nimble, couple and cell ago the months Boston. programs just disciplines, example This create of X a therapy As location, translational opening in co-localized in development It's manufacturing next-generation we're facility therapies for towards all Cambridge of excited more in facility next and clinic, a and
and please. slide, in exciting, we Next receptor have for stand and great our X front momentum. you Two It's program. Cannot avoid updates of Orexin so you.
an the IV-only is principle our data you chance molecule, first right. we Society data at It's to proof in the TAK-XXX. can The molecules, European that Sleep additional see on a present of September Research and on had
doses We continue effects to on indices. see of sleep with multiple repeat profound
index, cataplexy, normalized Scale, of measure curing common at dose. this see taking our signal XX-milligram the data see that's where is a the can that can scale upper around the right, which get used. Epworth X-day individuals. We treated. X in receptor as presented these were You you they we episodes from test, again Orexin the time, And first are completely maintenance in the the had wakefulness cataplectic Sleepiness the for that patients [indiscernible] then agonist our functionally no sleep the period
oral program emerging from data have months. the we'll several and next exciting, really our So over
Next won't a here. slide, lot please. I say
quite a adaptive. modulatory our early expansion, both a ways, function, can activating multiple exciting remind you that for and provide the now started compelling And both immune only-in-class seen right. of provide in of enzyme cohort the now the meaningful system we're see responses, hand as and to and see it We you is different I to deficiency in to next and innate I an Congenital TTP this affects This wanted that enzyme in before program immune program, preclinically that's multiple Just TAK-XXX a inhibitor we've clinically. And to and then the first-in-class TAK-XXX. assimilation I both immune escalations vignette the It's process last advance ADAMTSXX in dose on of you slide ways. a please. congenital Costa, to is our to treatment TTP. the enzyme. over if -- wanted replacement slide, very
exception programs III do U.K., phone enrollment. complete for in in We a right. see received enroll to our these have an use Phase of different patients as we request can the study you for number the was in for about that are in compassionate call reasons. baby We born upper clinical With compassionate a ongoing for much use development, grant that's We a that trials. just actually a prefer
the to patients. This that best there's are And life-threatening way we there happen. where the just potential, no needs meet acute, patient the a make to can baby born we Sometimes, to third try was of couple.
X correct perinatally patients, therapy. to story TTP these this their well. is us, in baby for X story see that major you that, the could surviving. even This because congenital and we a interest in of months left and n is bottom was replacement the And and an tell of of able The anecdote, human baby I not and the the it's baby we We to but platelet TTP. efficacy The the that's overnight, survived, the right suggestive congenital this hospital old not levels. out enzyme of you had were X now therapy. get And of doing an see died clearly though and first able were it's it's defect that highly
Costa, you. hand over it with I to So that, will