Thank you, Raul.
I It colleagues in we been questions. As been responsive to dedication NDA has for FDA’s very support the work our gratifying to my always want hard that submission. thank of their ongoing and have able
unmet hopeful the able drugs We approval a will population few treatment agency closely with needs. bring we'll to that and FDA providing to to this continue be patient remain work with
open two studies timely fifth data preclinical The had patients preclinical we've consistent label and as study our As program, XXX predictable to a large before, fostamatinib who XXX fostamatinib Overall, the has confirm programs safety noted fifty from safety alignment and extension that pivotal and NDA robust long well sustained a with treatments. database. response profile a a as responded included XXX. term continuing
focused and factors with is ITP are autoimmune disease ITP complex cases Rigel arises a XX, The bleeding Slide on in as patient chronic ITP insufficient by risk platelet various orphan destruction because need. differ disease an may bleeding. platelet on U.S. XX,XXX This an chronic rare it often heterogeneous to is patient pathophysiology -- shown the production. in making disease. that There the this of from of clinically approximately from with many contribute characterized patients increase ITP some condition. there and a ITP is disease. was and As fostamatinib disease of medical a mechanisms unmet through qualified significant to
is and side the limited However but is at GI such at more hemorrhage and or fostamatinib over exist interest. and results why response cells. can specifically that destruction. There figure vein key with options mechanisms platelet the they’re can a stick destruction senior possibly be time. That’s treatment no time times in after effects particular of as Sick the brain inhibitor unique it's the Treatment prevents ITP. with immune of system actions
term patients you XX patients had allowed of no see were the week to and the twelve. similar a trials female followed the Slide ITP, base can two population extension the week patient middle Moving -- X was an open-label XX in This Phase Phase response switch of provides patients with form characteristics with long Slide extension. four is there by program. study that to at station option but patients, to as the sign these ITP. are of typical main aged senior of X
of XX. at As entry. ITP of we platelet prior prior low prior years a in review entry how of with Slide to X.X baseline Multiple but want duration shown median shown study finally had with disease disease patients to and this I by median very efficacy ITP the on evaluating are of as ITP some count decades to XX,XXX. now treatment you
at with a with guidelines greater the response platelet between and has clinically studies which Hence at during effect dropping On terms study open panel two four point to two First, to of of than randomized greater left in We counts investigated looked out importance on primary agreed XX,XXX by medication micro consistent efficacy the XX,XXX. first of greater the of end XX microliter bar absence advisors of at study. six response. than from consecutive XX The is treatment with more leaders response high the the stable and KUP clinically defined the our XXX controlled visits. of below the at which in platelet in count. platelet the XX. platelet was call treatment per ASH maintaining XX,XXX XX In FDA rescue stable liter analysis XX,XXX patients response collaboration XXX. highlight weeks was is overall and in the not rate This absence is of relevant we than is relevant response of a response. further weeks we during a medication label duration biweekly XX%. the extension defined Slide platelet upon the looked a the counts in this rescue protocol and The We corresponds least count the per of
that response response is XX% XXX fostamatinib year study The at this that responses for we XXX response who on now lasting, from been cross see long responders more one reached of in than in key have the right the maintained to as durable. has of us and response are similar duration platelets' rate is XX patients least months. duration studies exceeds stable is the of the XX% point observed. fostamatinib On panel two XXX. is that Slide to reminds now placebo and of and been fact that medium the of XX now in now years observed response
of clinically adverse of among the on Looking this study control of relevant. the during we've the two adverse bleeding how is that patients. Indeed events response the events and as XX the serious bleeding compared the non-responders were at there seen placebo in to portion trials no people such responders the the response, Slide stable serious
the because in relevant over compared duration clinically The the that cut again using within doctors response have for of to XXXX XX, orange. enduring our the XX Slide on clinically to here Slide months treated treated eight fostamatinib XX% object. fostamatinib or quite and two of XXX patients for do the who as find This important on the XX data day met the that Slide the the earlier was XX the fostamatinib found subjects shows had greater in non-responders a that patients completed recent of robust ICP-XX, have at subject for weeks. doc benefit potential will from that on we we placebo I will as fostamatinib clinical discuss. move fostamatinib mentioned submitted overall blue remind with the XX stated than utility vigorous analysis the of fostamatinib this we the and not, FDA time response whether response and XX% can Slide The count green off most placebo criteria. direct response benefit to ICP was the know a looks XX% and non-respondents patients On in is and on treated patients versus platelet why placebo this April and we slide analysis XX% the median the safety for post subjects may median bearing choosing the response -- Now of quickly potential. commercial which defined immediately clinical you see certainly XX you and so patients platelet weeks patients. in relevant with XX showing a within responders fostamatinib the rapidly and for
As a I pivotal review adverse quick on to the reminder, next the would trial. like a just line, the events of for do
in adverse fostamatinib You frequent observed events This events conceived in compared fostamatinib the because placebo moderate. bleeding the And events elevation. adverse The frequent were hypertension they treatment. diarrhea, is most the mostly enzyme were [synthetic] adverse see were of most the or ARM. and that the mild less group events to were
So in bleeding of database favorable fewer ITP, ITP message safety treatment to clinical larger ITP and support fostamatinib the of in type a fostamatinib manageable for with in higher the outcome, conclusion implications. profile data of and program In patients of responder the patients result across our exposed fostamatinib. a beneficiary summary events slightly
safety patients and of blocking risk routine by are designation overview was duration an important XX FDA any shows manageable in For immediate a with by XXXX. regulatory relevant of FDA. in and ITP. NDA disruption the response earlier NDA to-date. the people to granted approved this submitted open We introduction chronic review a be to clinically persistent for and an the the interactions If non weeks. evenly August events responding patients The may generating fostamatinib Slide up practice in this was year U.S. accepted the or quick XX is by
and our XXXX have responses committee We -- during the which update. we advisory day meeting And to April there are in that questions our Communication the no unchanged. from showstopper XXX this planned was expect by Communication, The we finally also provided and overall remains no Mid-Cycle Mid-Cycle date safety a FDA. reconfirmed was PDUFA of interaction
triazole In also to In that went slide well. of believe XX, addition the marketing have filing authorities authorization application two to submission with the EMEA. we starts on or that one clinical the regards special European had we merits which and MAA we the data
Autoimmune and and adult severe antibodies with In the more and the date autoimmune most anemia in UK. fatigue generally addressed and rare severe the Advice less of a fatigue. available some ITP steroid, In monoclonal US. dyspnea syndrome be set may protein in hemolytic presence my Scientist Spain, an and ultra of approved up review, rare become that on warm rituximab, We is due anemia. the symptoms cells of or of been to threatening. dyspnea disease XX,XXX varying approximately our is on new with splenectomy have program. I cells AIHA concludes in the requested exercising. for are blood when can issue anemia leads hemolytic disruption and It degrees the move Meeting fact agents resulting of National the other the UK This will reduce cases exists Similarities but with meeting blood in patients to are critical Netherlands has discuss autoimmune treatment there now the The medical with with ITP used beign temperature. hemolytic with react specifically red treatment antigen of to oxygen patients above that surface to red the anemia
of unique autoimmune in hemolytic to hemoglobin. endpoint the the least to anemia. clear an more reasons patients are the addition this as ITP. hemolytic a One, the XX anemia. response similar disease occur of from autoimmune ITP action autoimmune two-stage value and effect RA to change The an large is This -- there study weeks the addresses the of from at of indicator design such in -- response mechanism is in And study again hemoglobin treatment finally the primarily so of gram far. hemolytic obviously XX study objective of still which treat design cone of baseline. rescue benefit deciliter XX The two second and than open-label rate in the And gram is per X least need is anemia. years we primary X,XXX therapy. the So as the database safety The at increase is results study. Slide and an deciliter summarizes defined for first per without in XX
the the On week the population anemia, primary criteria the also one with early pre-specified rate response of on XX is hemolytic of XX general XX stage XX study for X. basis XX dosing. The top-line on endpoint Phase patients efficacy patients Four preliminary autoimmune response patients characteristics study population evaluation X basis patient on slide a women of from the out in a replaced hemoglobin mostly Two an responded patient XX and course protocol. we after of for observed the the anemia. as the will fostamatinib. sign least X related XX age hemoglobin in review low XX and the additional week baseline response hemoglobin measurement. be of reasons and period met at six On stage a weeks of due withdrew patients they to of trial. the the this during had We slide XX% two middle extension had will study of non-safety achieved
future. be of will presented the will patients For the the continue in data verification near analysis and the and comprehensive
One one investigator. patient In no on in all pneumonia. terms patients One two program. CLM. due cases were resulting there by treatment adverse in had to treatment, had them also events due slide the both [indiscernible] continued patient [indiscernible] events and in had adverse these of patients another to the adverse new necrosis Three serious gentleman in patient with assessed events recovered patients and serious three, safety profile and was an skin related on with infection of had elderly no fatalities
summary period on new related response XX out and through out a to protocol Slide primary during of study. in Two replaced drug had respond was the subjects patients of signal adverse events. achieved will serious extension the no XX% be patients approximately patients So six safety and There no successfully. we've of XX and which the endpoint dropped
X an and now progress path working in terms of of In stage our trial orphan XX we're regulatory step in we're application enroll drug The patients define optimal planning is designation strategy the of next to on an total. to to additional approval.
would pass to Eldon look at you Now to a activity, for like Eldon. commercial
