a received to EMPAVELI advanced we position Thank exceptional our pipeline, XXXX our for broader for top-line results us OAKS was today. DERBY for approval FDA second said, approval receive year our or FDA from as and We complement. strengthen you studies joining a delivers leader launch, global in simply first reported positioning remarkable Apellis. potentially our initial all and an our
and including or and meetings, in a the from last were thrilled therapy company team the transition transformative ophthalmology, In May in Paroxysmal ever Hemoglobinuria meeting. critical results Atrophy being a bring therapies are reporting we reporting worldwide. positive Nocturnal us to OAKS Globally, Let and the We passion approved Geographic we and the also the for approvals me are that elevate Sobi approval other DERBY for improvements, to many one-on-one continued strong. The Since breakthrough In or fall the engaging with completed our cause leading develop efforts closely Retinal a excited is company stage the FDA step months relentless the with More the over compliance we patients. results feedback million living patients from adults CX high, the start to far potential focused medical GA, with also ever blindness European few than were care the PNH. with GA with globally top-line recognition well-positioned and EMPAVELI last Phase so by of the to was making are year, being by significant first marked for last run-up community to who to target discussions first to of we GA EMPAVELI. past through final of seeing an and pre-NDA EMPAVELI a pegcetacoplan commercial I forums. represents a Patients partner, are is step is these am by the with a is Union. feedback recently of treatment see through X submission. and launch. This additional now believe treatment R&D has Physician standard disease in to delivering been ever, September, FDA progress received the payers. patients. positive our the X our
our three pre-clinical advancing We also programs our made with programs. late-stage progress including systemic and in pegcetacoplan broader pipeline, our
our key priorities now XXXX. me Let spend on for a moment
as for be pegcetacoplan mentioned, this raging a approved as on are GA patients mentioned, an process for we are start the going we with to through First, vision your a therapy cells which bringing first We opportunity best these to the pegcetacoplan, longer. described blind focused and retinal have process with currently can no irreversible I with patients retina, fire that forced are continuous cell as as treatments. slow is you there Once First, believe I and GA. death. market ever preserve gone
are our on-track it actively NDA the to preparing are to We second FDA submit and quarter. in the
We OAKS our in to data are plan discussions from March. and and will In parallel, in months and share with European these safety regulators. DERBY NDA the efficacy include data we the beginning pre-submission publicly XX
Our We that and the their quite levels treatment. EMPAVELI. care hemoglobin PNH further from initial to our all market EMPAVELI with been communities/ of standard to a a and in potential approach patient And ensure aim baseline research patients the educate as regardless will benefit PNH has establish have as elevate we we efforts encouraging. want continue we approval to Secondly, of first-line to physician potential the
are we per complement late-stage Sobi, advance four address we as with plan driven seeking diseases. therapy PNH, programs a Collectively, opportunities rare as have patients year, Together this EMPAVELI underway transformative year. the these XX,XXX EMPAVELI. to to of opportunity Beyond expanding many for could the as needs for significantly
for to IND continuing portfolio research we as AAVS that half or the therapies plan increasing controlling this in novel tolerability, to and of decreasing these dosing an enabling many and as advance to collaboration with needed pegcetacoplan targeting year. believe By that our this a associated Brain-Active Third, are forward we data of In adeno-associated our first want CX, allowing may safety CX to the with for to for patients be advance able the such we we approach with in with of benefits systemic gene are in issues the pipeline APL-XXXX, inhibitor pre-clinical therapies. we our by the second pre-existing first-in-class submission our sharing dose and year. to partners, viruses see half expand finally, And antibodies. look clinical
additional continuing the pre-clinical and reporting priorities over to plus programs robust strategic the clinic siRNA two an the look we pioneered here pipeline team program into a including as the this late-stage rare four progress cementing complement. XX programs forward at and our incredible products, building of programs over that And EMPAVELI are towards commercial of Adam? over have additional We call clinic By to was course these in multiple let could year, Adam the our XXXX. on also across Apellis. the commercial in on end APL-XXXX global And further me disease a encompassing progress we We XXXX. months. XXXX next our for am the the I momentum update. look by to position amazed by now our a has heading extraordinary forward science to turn year. leader been
