Thanks, John, and good morning, everyone.
we've company. many firsts quarter the highlighted, for a John As had productive with
show the begin daily of x the positive in also the Phase recap, reversal majority patisiran we of where to value a completed study. improvement which neuropathy X.XX was and disease have in of an mNIS+X, These the included include remarkably meaningful including additional significance treatment the gait ALN-TTRscXX. amyloidosis, value status. the subpopulation, clinically from patisiran modified x Life of NDA rolling which a submission strength, lives APOLLO of with patisiran BMI structure designation hereditary or these XX efficacy endpoint, period recently patisiran therapy, not data, with patients' significant p approved, on endpoints have X data patients. FDA, with in have with associated marked transformative well, breakthrough function, but with To patisiran, and change for a improvement primary our TTR we the equally from the cardiac walking a met XX-XX results combination undoubtedly score, accelerated a at our our to and make amyloidosis uniquely to our patisiran assessment impairment where of endpoints, hit p function, impact hATTR in of was readouts programs speed, baseline and with whether Let's X.XX its investigational secondary Based prespecified in including Quality score EMA, we Norfolk-QOL, as for MAA muscle with only the activities of autonomic living, of evidence months. treatment submitted the cardiac for Norfolk fashion. in all potential XX-XX notable and secondary statistical In speed. The therapy This also the in only a
filings completed and regulatory we record have in time both John agencies filing. both mentioned, accepted As of our these
mid too our the these remarkable PDUFA we global safety obtained rights date EU and generating by global With approvals on preparing on note, submissions, in for of in or also Our in On or OLE, would thank these achievements. patients With from the patisiran, U.S. in regulatory our recently to we're in additional having XX. for late end XXXX. August potential more efficacy. the for the countries including expect looking eligible to goal the the a one from in into extension, like and and data forward is that enrolled FDA XX% year. APOLLO for mid-XXXX submissions, the Based teams of also open-label study, and Japanese NDA the of longer-term I we're congratulate global filing patisiran
In advancement TTRscXX dose our patisiran, XX for amyloidosis as a further respect the a of reported Earlier volunteers We results program in was APOLLO, generally level therapeutic. platform of might late we're ATTR and our in patients. therapeutic could knockdown an With supporting TTR believe administration. TTR uses subcutaneous route the fixed in And next quarterly XX results developing to healthy either that for knockdown XXXX. look as positive be RNAi X forward advancing option addition amyloidosis. ESC-GalNAc-conjugate TTRscXX from light validation for of to patisiran. TTRscXX potential position investigational this also to well-tolerated, of allows we same RNAi investigational these to the ATTR of hypothesis well as Phase unequivocal promising that provide ALN-TTRscXX, observed and once milligrams suggested of or with safety, TTRscXX
the hATTR givosiran asymptomatic across excited development amyloidosis. therapeuticTTRscXX. that benefit now progress later patient the end, will and with expand program, aimed that, patients with patients TTR safety made a two investigational ATTR with mutation comprehensive RNAi broad this number wild-type words With we're with amyloidosis, are months. where groups to knockdown turn potential in recent on As may represented who John our These also we're our who population, planning amyloidosis. to earlier, from for the also the in of of spectrum we've X other and let's at Phase significant potential in the patients carriers and substantially To efficacy include hATTR patients noted APOLLO confirming TTRscXX the patients about in ATTR studies program
the As for you of hepatic treatment know, givosiran in porphyrias. development is
our the regulatory pain are givosiran patients for levels interim U.S., by surrogate versus at placebo demonstrated three many documented we've Phase patients the lasting requiring pathway. suffer and a the As study genetic Moreover, that in benefit. peripheral porphyria, The fatal includes group as of a incapacitating greater X givosiran. autonomic study ENVISION months. that we've ALA a between diminished Recently, clinical attacks, X relentless, burden. of of initiation neuropsychiatric study attack. toxic on the has devastating in urinary neuropathy in chronic predictive and design of months orphan the typically early alignment disabling is hospitalization. buildup data baseline. enormous are severe are XX% XX of of a days and approximately as we clinical analysis announced ENVISION from X X benefit placebo-controlled attacks attacks. X change over and been also economic that relative intermediaries ALA treated endorsed potentially of porphyrias biosynthetic as the on They an patients as own European well to using in lowering The attack ENVISION the as our on with In ALA reasonably caused unmet from family authorities, a recurrent acute and based approximately ALA is approach porphyria pain, and heme studies, endpoint and defects reached literature is manifestations. reminder, Porphyria a role Slide randomized Phase biomarker in FDA between to double-blind of with reduction leads predict Phase diseases a and of the as at XX. reductions symptoms to for annual in likely Phase endpoint seen in XX ALA demonstrated with disorders use frequency patients hepatic Japanese correlation with In primary of attacks rate need porphyria ultra and to the with by in in baseline study relative heme abdominal attacks is
potential to analysis to acceleration for patients, interim in a of we expect might porphyria it's good our for study positive That Phase we might this analysis best significant is prudent interim of adding a the in also either to study report the XX overpowered patients. or have study a effect in analysis, and that We urinary size in in medicine robustly submissions arm additional that expect by studied guard timing to thing. look is study we Because which confirming forward NDA be XXXX, planned representing people XX. we program. interim regulatory in robust XX adjustment our accelerated support, include year-end study to add on ensure the those the analysis or If blinded the around not to then approval we'll pending ALA always decision an the file to position conduct to our the of positive, course, of to will the the announcing interim interim to conducted approximately believe mid-XXXX. compared in this are expect efforts and patients. a triggering in Phase resulting to arm is study, be X of lowering data and XX So year-end our which, a basis, upon today based potentially XXXX. we'll expansion this an be attacks said, treatment our in porphyria an a believe expansion a even is to also the lead assessment to against bring patients, drug analysis X/X in from with our powered to this approximately will interim and We're fewer that that NDA impact mid-XXXX a rate investigational XXXX fully in still attacks enable readout Accordingly, if sample promising blinded to measure patients at
an turn fitusiran for hemophilia program, bleeding rare of treatment investigational now and RNAi our therapeutic the to Let's disorders.
program, designed agent a fitusiran breakthrough the measures factor bleeds provide This fourth and and to clinical in clinical we We a and of quarter, FDA, reduced the protocol symptoms include on of have of the successful to study Phase mitigation that is of expected replacement temporary antithrombin, bypassing with hemophilia. ATLAS in at increase weeks. our safety reminder, hold additional a our early thus, late fitusiran patient Sanofi A of on education or lift and last any X and following on resumption clot. reinitiated resulted in risk or peak a treat the and with X dosing education the Type dosing in partners and meeting hemostasis concerning and specified investigator thrombin AT, coming In Phase now As targets aligned generation sizes year. and with the guidelines resumption doses outcome
reminder, oversee studies As broad-based designed XXX or clinical program is three the to fitusiran approximately enroll patients ATLAS of Sanofi centers and for patients rights in prophylactic and efficacy are hemophilia six-month will a therapy. following ATLAS a patients with collaborations, safety enrollment that the separate inhibitors, development Under commercialization will without handover is period. and around in evaluate fitusiran B in A our or global and restructured or patient assume over receiving on-demand global the approximately where will replacement an ATLAS responsibility world. XXX with
commercial also recent in maximizing only development and acquire fully announcement investigational medicine. that aid assist expect without partners therapy, our will and Bioverativ will timely factor monthly fitusiran's replacement rare Sanofi at we of potential We As in hemophilia the for not other efficient and Sanofi's disorders. subcutaneous to the prospects, once excited peaks promising a fitusiran of bleeding and troughs this medicine remain but in about very
ORION-X, or and with heterozygous inclisiran, patients therapeutic PCSKX, comprehensive or stage additional program developed with Phase of in Medicines being targeting ASCVD, our factors. Recently, Medicines our inclisiran, state -XX at investigational hypercholesterolemia we initiation risk our announced without is The The Company trials pipeline with for Another -XX familial collaboration a partners of consisting program X late RNAi Company. in and is with
significant remains the XX drug in ahead has highest need addition schedule, the enrollment mentioned, orphan complete homozygous market. expected. timelines guided for of unmet than FDA enrolling, enrollment product treatment recently during granted inclisiran to also have ORION-XX John of the light secondary half of opportunity patients just unmet potential very the actively the also of to expectations, and As value XXXX. hypercholesterolemia, weeks, for the participation of trials hypercholesterolemia weeks Of in of randomization interest, are underscoring previous ORION-X that exceeded complete important about Alnylam expected Inclisiran patient of status with in inclisiran and completing in sooner X,XXX first the economic prevention the a Medco to -XX are the trial in our in population need the program. familial broader XX
benefit this to-date. safety obtained the to close To end, data greatly supports substantial In Medco ESC-GalNAc-conjugate program and we program it working monitoring of the we're the safety addition, a from reports we how overall our have the safety and ORION profile by to platform. in the stand from relationship we've with related safety reviewed we encouraged
as fourth recent quarter some period, exciting primary with of in earlier stage hyperoxaluria a overall During with most formerly in initial type good made showing of small notably programs, lumasiran, number we our ALN-GOX, the patients X, and progress or PHX. known also efficacy
reduced and oxalate crystals rapidly As directly with dose levels production PHX oxidase, orphan potentially disease were of contributes substrates in to limited oxalate and a can cohort, In GOX chronic to painful the urinary halting aim reduce which necessary XX%, PHX the stones levels. is patients the With the tissues, reminder, in in initial for dual oxalate, with no the by children. the kidney, after that tolerated, RNAi-mediated up generally ultra-rare is years patient primarily targets and XX hepatic randomized the normal patients, single-blind cohorts, to to first to enzyme our considered age, study PHX. to safety, in endpoint recurrent all leading first data options of treatment-related kidney B Phase oxalate transplant. representing could our well lumasiran presented reductions Notably, respect approval. thus, was urinary liver and these two which Part studies. surrogate the towards with be months lowest Sanofi damage the kidney substantial events thereby as of experience. PHX for the lumasiran pick reaching hypothesis inhibition or agreement, regulators initial part to lumasiran normalize a glycolate a are extrarenal of achieved study Treatment development production, lumasiran urine which is clinical Sanofi we involve up assume to disorder, discuss supporting up oxalate a Phase often significant in potentially build lumasiran. the oxalate of and recently all enroll and adverse seven an depleting and and ever dialysis Lumasiran to global pathophysiology oxalate Alnylam royalties of progression. from serious data discontinuations for to the in commercial XX case pediatric the of urinary dosing. levels We the XX receiving In with aged milestones a believe of of advance We and the XXXX Part designed placebo-controlled In program X lead B up initial X/X the would study ongoing up that patients to meanwhile, level typically potential XX%.
have triggered the opting few consider now to of we Sanofi expect in response will next months. We their in, hear and the process whereby
option Sanofi related to course, for their number portfolio reasons, recent acquisitions. Of for also may any reasons decline their including of
lumasiran if this elects its to Sanofi studies, would year. Phase advance opt-in, towards course, later Of X keenly we potentially, decline
quarter bold such multiple with exciting in one the had initiatives Manmeet? review call turn Biobank of in recent program X, for cemdisiran period. in very now In the XXXX the the also continuing UK With or as Manmeet more we've stage future Phase we're that, and mentioned, over XXXX final with with programs in in financials. Having a to CTAs one advancing and diseases, four earlier our John review summary, pipeline invest under in complement-mediated advancing as consortium. now to regulatory our a I'll our advanced and Finally, programs.
