Akshay K. Vaishnaw
indeed good and everyone. been programs progress efforts. morning, has stage marked progress to platform quarter with substantial It and our in on remarkable pilot our early our John, addition Thanks, with pipeline exciting late along across a
with Let by now patisiran, name me its commercial start known ONPATTRO.
conjugate the strong in cardiac journal infrequent In of X for publication medicine amyloidosis. at the for with published aligned scores EMA in pleased to is ATTR of Quality an addition with HELIOS-A, the Phase to living approximately hATTR the Norfolk patisiran of been clinical underscores to patients study subcutaneous approval potential safety favorably has X ALN-TTRscXX with study EU, the endpoint polyneuropathy the enabling we England nine demonstrated move data reinforces the study patients APOLLO ONPATTRO and and as potential program patisiran launches of September, journal the Medicine. of were with APOLLO impact ALN-TTRscXX the in manifestations were development and designed therapeutic certain a arm our of these pivotal FDA benefit GalNAc open results We the study We see amyloidosis X of follow-on for in published placebo exploratory amyloidosis utilizes and cardiac the cardiomyopathy. with results which in profile pleased the patients data of discuss the the low ESC+ U.S. with believe from of ALN-TTRscXX, now that announce in subsequent from the of Life APOLLO administration. highlight More results believe APOLLO, volume dose of in hATTR encouraging Journal very amyloidosis, study this patisiran. ONPATTRO hATTR technology, the results our APOLLO Phase we Phase comparing amyloidosis effects and the of in the to in further to and HELIOS-A mNIS+X from the New the co-primary endpoint XXX study to label were in recently, believe We're I'll we hATTR in people support feedback. hATTR on of Circulation. amyloidosis July of months pleased and
assessment disease death which specifying respect assessed time XX-month at point. be the support burden, given post-hoc will from APOLLO hospitalization, additional a comprehensive to secondary will the on findings endpoints Secondary with importantly, at exploratory (XX:XX) of analyses in and conducted endpoints the least
and ATTR addition, amyloidosis the be some endpoints. receiving This of XX approximately ALN-TTRscXX to will including Phase in We're although cardiac arms. will the be arm reference included study small of to the will track weeks plan as XXXX coming additional formal start parameters X patients a HELIOS-A In comparisons conducted study no and co-primary ALN-TTRscXX, wild-type ONPATTRO also ONPATTRO, endpoints beyond. include in between and studies the on in start on
ALA In aminolevulinic consisted this porphyrias. or in XX Let's for and less to reduce three X.XXX. ALA, Givosiran the of investigational successfully X givosiran. givosiran administration RNAi endpoints the p attacks to move announced interim frequency study significantly pre-specified results analysis to the met urinary receive on late topline relative at monthly we of clinical biomarker levels givosiran XX suggests Phase placebo porphyria were of acute benefit value acid, to a predict development Urinary patients. of with hepatic in our has interim positive with is porphyrias and therapeutic in analysis months a than from reduction interim of The to acute September, likely placebo. that in hepatic reasonably these receive which analysis XX randomized cohort acute ENVISION of givosiran, outcome a patients intermittent to patients potential the
for on XX with XX% ALT resolved. on of in trying the times givosiran; primary limit occurred patients results givosiran XX% date the treatment of who As the forward In so primary in greater updated in clinical Pediatric of There of study lumasiran, reporting liver in with from (sic) the August study's results. assuming cutoff therapeutic likely events turn month, which Paediatric] last hyperoxaluria subsequently lowering, look in I'll of or development X serious to FDA the times of annualized data type we're we and the that submission XXXX we're PHX. XXXX, contribute stones an from RNAi rate patient experienced Nephrology of submitted painful mid-XXXX, of in presented on X than the achieved All developing urinary NDA patients increase early positive X/X progress show for impairment. We or XX, Society occurred support, and of which was attack topline and a discontinuation limit to one These oxalate, (XX:XX) adverse less but max initiate significant to out was on kidney to European complexes XX recent a in X transaminase normal. than positive X overproduced or kidneys, results lumasiran. now with syndrome metabolite [for of calcium an patients upper now in endpoint in Phase with XX, XX results in and renal to placebo. patients meantime mean that's oxalate XX% of Meeting At the a March. deposits in upper reduction full an sections X.X givosiran leading the to full the normal out rolling PHX recurring of of
treatment. no were reductions as XX and study the As patients reported reported None meeting, placebo three from we clinical ongoing of and the XX severity oxalate Society XX% were to three XX levels XX% related out parallel site mean results from within oxalate. new oxalate lumasiran the of reductions X/X discontinuations reactions Adverse as results lumasiran-treated OLE self-limiting. drug. plasma receiving max the five These to normal American at of receiving moderate the recently in of baseline the in out range. well achieving and lumasiran a for studies Injection plasma patients of to cutoff showing generally in relative moderate reduction urinary of Nephrology patients sustained with in the Phase reported plasma or More patients. demonstrated placebo one reported with that oxalate date, there mild were and were patients and were lumasiran. were three SAEs in events all mild the were
from who patients which X data safety study, transitioned those is to the study. efficacy, Phase designed the profile of long-term For lumasiran X/X the and generally OLE tolerability remains have consistent to Phase the evaluate with
the date, As related SAEs, assessed data were to of two the drug. cutoff as had which of neither study we
maintenance loading study the the Now was ILLUMINATE-A highlight the a group lumasiran to urinary in lumasiran exploratory XX quarterly X will initiation at will patients with be in is period of placebo-controlled quality key from rate, of placebo. Phase life. endpoint study evaluate This The oxalate tolerability, or number randomized receive Primary double-blind, monthly the of or six key X:X either PHX. endpoints Patients also include secondary in at glomerular relative three months study. recent doses is X approximately placebo the estimated versus mg/kg the the randomized, lumasiran doses. in oxalate, and will baseline a study reduction of additional urinary of of the that to safety, filtration measures and with
We expect XXXX. positive, report initiate topline late early results in to to XXXX, ILLUMINATE-A beginning filings if and from regulatory
years this study trial, addition also FDA initiate aligned in design patients a pivotal we renal In with have age intend the the XXXX. Phase lumasiran of We preserved less with X/X ILLUMINATE-B, trial of to for ILLUMINATE-B than six study on function. in to
topline representing X mid-XXXX. industry's therapeutic. This fourth report conducted rare the expects RNAi data Data dosing The Turning and results The fitusiran ATLAS very Monitoring the now which to ORION data for Committee of the Phase Independent X,XXX for and patient safety X development our body years the important investigation recommended safety its this seeing now forward efficacy Medicines and for in treatment partner enrolling inclisiran the is modification. Company and X the provides recently most patients announced Phase nearly that to the comprehensive Phase exposure that for of Phase as hemophilia programs, in much the has of RNAi for review program Company We disorders, Sanofi without With partner therapeutic, database for an inclisiran our in of Medicines continue studies look to X to inclisiran, program. safety continued bleeding
to months. targeted filed In the to the that alpha-X XXXX. clinical We liver antitrypsin the and initiating this conjugate therapeutic of utilizes our GalNAc we our investigational start by forward Turning I'll therapeutic a now for X CTA component technology, cemdisiran meanwhile, which Phase ESC+ cemdisiran, the RNAi Phase plan X as complement offers our coming initial the treatment we first is challenges. This now ALN-AATXX, to for recruitment the on increased expected that with of to discontinued in therapeutic in atypical targeting focus IgA We hemolytic announced of the earlier stage in investigational press our uremic pipeline, our this RNAi expect study a study study nephropathy our wider the deficiency-associated an in program look Phase year syndrome in start efforts index. CX. We result release X end data activity with disease. studies of morning and
showed Also gene siRNA exciting achieve the Finally, I'll administration OTS, brain close rats large at we very tissues spinal with in Intrathecal results novel potential NHPs. be We're durable Data the very to well-tolerated found Meeting. opportunities. in non-human targeting intrathecal OTS demonstrating siRNA for highly of CNS, conjugates preclinical about was rats and generally presented at primates durable both of conjugates new last target across and preclinical siRNA cord. distributed are a and and injection to silencing delivery therapeutics a resulted NHP. broadly these ocular and excited single the results from to that RNAi of of month the in showed a silencing number there robust potent, where in conjugate we
to TTR intravitreal the that, let siRNA findings Specifically, complete of hand approximately of it NHPs a the silencing of TTR to injection safety progress. to conjugates. knockdown intravitreal and no our led XX% related discuss mRNA retinal commercial And were near notable Barry? There single now off epithelium pigment proteins. with Barry administration of to me in
