APLS - Earnings call transcript Q4 2020 | Apellis Pharmaceuticals, Inc.

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Operator

Good afternoon. My name is Valerie, and I will be your conference operator today. At this time, I would like to welcome everyone to Apellis Pharmaceuticals Fourth Quarter and Full Year 2020 Fiscal Results Conference Call. Today's call is being recorded and a replay will available at apellis.com. I would now like to turn the call over to Tracy Vineis, Vice President of Communications at Apellis.

Tracy Vineis

Valerie. you, Thank thank Apellis' and results. joining today us Good quarter you and discuss fourth financial full to afternoon, for year XXXX With are Dr. me call Chief on Officer, Executive Co-Founder and the Cedric Medical Francois; Officer, Chief Dr. Sullivan. Commercial Chief and Officer, Adam Federico Grossi; Officer, Chief Townsend; Financial Timothy

begin, and point that differ Before risks are to beliefs. be These I filings we on based and like you that to factors risk for our encourage our I certain may statements additional statements materially. forward-looking are would actual uncertainties, our will detail. to we the out making current results in and expectations subject consult discussed SEC

Now I'm Cedric. to over turn pleased the call to

Cedric Francois

Thank Phase or PNH. for for in standard defining potential you, complements the platform of joining to hemoglobinuria year everyone, quarterly III year care in paroxysmal to we of good marked nocturnal results PEGASUS was us targeting for of complement look our to the leadership by the high a across pegcetacoplan PEGASUS Apellis range diseases. our need. elevate further a Apellis, unmet Tracy, data positive today conference the highlighted and afternoon The first call. in XXXX, driven CX broad a forward transformational global as with broad we of potential on In build diseases PNH and the for XXXX

against make progress strategy on X based significant is of strategic year. see corporate we As our can and expect slide, you to this on these objectives, each this

systemic complements-driven as disruptive rare a to diseases. Our therapy first treatment objective pegcetacoplan, a establish CX targeted is across

Townsend, discuss launch. successful pegcetacoplan in for prepare the for May our Commercial of work PDUFA commercial PNH; Officer, approval a to Chief have We of U.S. XX date and will our Adam a potential

CX. or additional registrational targeting We also with broad Biovitrum, our work of programs partner, as to potential we to of Orphan the Swedish advance maximize Sobi, pegcetacoplan expect X

pegcetacoplan for In seminal will The company in be treating in second diseases. #X event GA, is Phase Apellis, or top III from geographic line our be results we studies retinal atrophy, what our this unique objective in of for and the affects expect a to third what become year. opportunity people a people approximately and have and of for quarter the is disease first a advance around exciting with disabling the X we could living to GA. drug that GA world, clinical million relentless

to Our technologies is develop new control third objective to complement.

team by clinical X active research advancing into on fronts, number we candidates new of very has been a the of development and forward look product year. end Our next to

we global key have to strategy milestones the advance several complements-driven treatment diseases. XXXX As ambitious you an and see, in our can leadership in of

to pegcetacoplan data Now Grossi, our first a our deeper Federico systemic establishing Chief I for into turn Medical PNH. now our on Officer, let's rare as Dr. the pegcetacoplan in call therapy objective, diseases. to over dive disruptive recent Fede? will review

Federico Grossi

you, for uncontrolled by through season blood CX, Thank the intravascular at cascade believe why PNH caused complement elevate with treatments extravascular everyone but the reminding Cedric. downstream carrying of intravascular care living pulp the cells extravascular characterized by activation. by people is of complement we to PNH. I'd and the has destruction hemolysis red that standard begin not potential like to Current pegcetacoplan hemolysis. both inherent controlling

continue offer complement at from which inhibitors survival, CX controlling not target living centrally pegcetacoplan suffer. symptoms while add people do extravascular and result, a to as debilitating both As CX, intra PNH many improvement patient Early hemolysis. they with

means of on pegcetacoplan XX. improvement the sustained study per result, with as and as was of X.X the in Phase met only eculizumab, results, these became III of to hemoglobin the compared superiority endpoint grams safety May Soliris PEGASUS received in As a primary of PDUFA or demonstrate measures. we pegcetacoplan deciliter investigational an clinical and first therapy a FDA from for in to [indiscernible]. profile PNH other Based adjusted the key XX, pegcetacoplan The at review date priority Soliris in improvements well comparable week to with

December, line sustained in pegcetacoplan the complete in week help from we data at and baseline results show the maintain patients mean potential patients These XX, study. long-term the to pegcetacoplan-treated from pegcetacoplan-treated with XX patients. hemoglobin week has announced equal increase disease. improvement the Additionally, in gain existing PNH increases top that of control a to more PEGASUS were At with

or is or by technology CX XX assessment hemolysis, the stabilization in levels the LDH bodies you In or absence patients inhibitor. profile we were December, comparative lactate the comparison, that The a therapy hemoglobin pegcetacoplan control At world. MAIC chronic measures, LDH monotherapy in pivotal in reported transfusion consistent identified. like on avoidance, CX and of XX% showed treated clinical ravulizumab, and or pegcetacoplan also accepted across the week with clinical patients method hemoglobin Ultomiris the of hemolysis health randomized count, the scores indirect switched for MAIC, safety effectiveness is period. research This called pegcetacoplan during inhibitor because were normalization. analysis valid study, see similar reticulocyte longer Also, adjusted of pegcetacoplan with graph, to can across matching and intravascular key in achieved remarkable to Ultomiris. presented observed a FACIT-fatigue head-to-head Soliris-treated patients Also, the studies patients Sustained ASH, compared controlled improvements non-new other experienced pegcetacoplan, patients in safety period patients a LDH illness as a used improvement the functional more and with biomarker of signals Importantly, open-label MAIC sustained and was previously at Ultomiris who well. dehydrogenase to also during top is treated pegcetacoplan. more assessment that pegcetacoplan data XX, achieved in

on the in to pegcetacoplan Ultomiris. reinforce standard with elevate FACIT-fatigue difference potential Additionally, X-point content pegcetacoplan by score life, over targeting of results XX% the of were patients of was on quality the there pegcetacoplan more and of patients a care major CX. transmission-free, to These treated

criteria. other study ongoing the design with study. factors III MAIC and adjust inherit like Phase for PRINCE our difference in all to to entry I to As now analysis, would move [indiscernible] confounding due

believe evaluates has PRINCE pegcetacoplan that from PEGASUS, in the population. the patients that overall broad pegcetacoplan On study results based to more potential of the label receive treatment-naïve this hemoglobin we study on While primary the of the The slide, weeks are you LDH design. treatment-naïve transmissions in after PRINCE absence involved. and stabilization XX level. reductions endpoints the can see patients PNH were X are representative XX in evaluated

study. in X per or have drop to pegcetacoplan, points a discuss their deciliter baseline. hemoglobin by more levels slide, group I'd the couple As patients the below escape to the about grams noted the control in key option if like of this to

standard please First, studies that inhibitors for regulatory were on for the PNH CX designed and therefore, use endpoint analysis. intravascular focus

extravascular As by PNH both intra and we analysis. is characterized know, all

secondary has that levels, impact So the endpoints, as of patients. on since and equally we truly such are reflect transmissions this some FACIT-fatigue the score believe important disease hemoglobin they as

considered to drop in with to minimally fortunate patients were only studies. by Additionally, per levels. hemoglobin Patients being be unstable. they have considered COVID-XX PNH higher across clinical X levels, by We've set deciliter of hemolysis, gram studies in we even unstable pegcetacoplan. on measure before the use impacted hemoglobin if Historically, a in have inhibitors, are bar been allowed our X per to grams deciliter PRINCE for CX

local from line in PRINCE's of to of data the due timing end June, guidance the than regulatory now is of extension be X which May However, in precluded transitioning This of with will us delays COVID-XX-related but or delayed the countries the later study, in our expected. top at have database and log. line beginning public patients initially approvals

update forward these hand and later call Officer, quarter. results We see [premiumized] to sharing I'll the excited pegcetacoplan are on preparations. to to impact Adam? over very Adam Chief Townsend, on our of now launch provide the to PNH our next Commercial patients an look

Adam Townsend

PDUFA Fede. successful PEGASUS to you, Phase results are hard May our standard date. PNH. The And Thank the anticipation elevate its potential we pegcetacoplan showed for working of prepare of the positive in care launch in U.S. to of III

this Clinical see that and from own CX on to require unmet inhibitors inhibitors patients market can to with X/X continue CX levels. about slide, shown to have people research you continue despite need PNH transfusions data treatment on with of hemoglobin our like Ultomiris. As and their current address significant falling suffer Soliris their

levels continue be anemic impact but red on closer normal these that to have other maximum severe and fatigue. final severely their to marrow. bone of patients patients X/X like blood a hemoglobin of output has achieve at expense of only experience X/X The of symptoms these from cells Another huge This the patients.

an urgent need for data treatments show, is these within As there PNH. new

PNH. within commercial robust for years, of have preparation X pegcetacoplan launch we a last our the Over organization first in built

needs integrated on launch, that to address Our effectively of team at on focused are highlighted and progress is is our we patients this ensuring ready slide. the

is staffed access fully and patients. and priority all with Our value engaging XX% payers than PNH representing more U.S. of team high

discussions payers support programs. have patient of Our also model resources on clinical the distribution Apellis the late feedback finalizing stages positive of yielded in pegcetacoplan. those profile is and with our and

as initiation to provide pegcetacoplan will well experience, long-term positive as both assistance and at We time of the as aim needed. with when with treatment patients a consistent

education. patients team, designed we directly experience, In with parallel established high-quality launch. and our which recruitment of our for Apellis including activities, example, Assist, drug patient a an have the product ensure through has interact treatment medical preparing administration educator program been care also As with patient-focused a to commercial our team will

colleagues activity with care positive, our and seen Soliris have have on U.S. been side the treating for X,XXX focused high this community have meetings our slide. efforts, our Separately, which considerable which are Almost of an can who need marketing benchmarks. the members targeted us with on really are our early in or strong slide, program over outreach right-hand in seen in early shown on As XX% patient access website, unmet initiated on interested particularly around pegcetacoplan of PNH, this medical via and PNH the with despite accessed and other left-hand also engagements showing Apellis activities engaging when tells can Patients caregivers, content engagement they are via established On HCPs existing physicians actively the marketing already U.S. PNH professionals, above at which advocates caregivers slide. been patients, ongoing our treat information, communicate spending and unmet the of in PNH examples and time with experiencing be to of appropriate. top on virtual be opted to They medical community this and need. industry sites health multiple disease presence the treatment also efforts, our in-person side or have HCPs, Ultomiris. patients affairs have

to X,XXX XX X,XXX experienced including Finally, top and centers. sales more treatment our cover focus than HCPs, be deployed will to team the key

are this have excited we the building approval about team honored are year. to bring and pegcetacoplan PNH commercial We to pending opportunity, the patients to

turn our to to we atrophy. Fede? As new elevate I of back is Federico the indications preparing as the see year. quietly indications. preparing Grossi, well standard review well in for are program Officer, potential systemic GA in PNH for approvals Medical over patients, the care later organization will commercial additional future results Dr. The Chief our now as the the geographic to call to as excited

Federico Grossi

to in our Thank with Sobi. systemic partner, of high with need you, The advancing diseases MP&A X rare programs Adam. registrational unmet pegcetacoplan

problems American CXG. opportunities membranoproliferative second complete start half Results registrational in few horizon. work Phase age-related as of transplantation-associated with in program Academy As in or AMD, no from AMD or we Phase HSCT-TMA. largest cold unique in the the second MERIDIAN presented is late-breaker The study expects echo secondary to GA in half CAD, frequently top GA seen or as wet agglutinin Ophthalmology on in a feature a present registration of slide, million GA, or is to available results, pegcetacoplan, over meeting. or initiate degeneration, registrational to the ALS. to Phase were which risk study, months. macular of III retrospective at expects further the webcast new of and our And this of our panel XX% this and under III complex CX our line an show in of our with year, on observed the enrollment recording continue in disease GA at Apellis We if reiterated expected feedback for fellow was cell conducted quarter. in patients with to recent Also, quality occurrence life. study X impact amyotrophic a AMD IC-MPGN Verana hematopoietic is in and our the make year, Investors more disease, a represents microangiopathy partnership stem immune of parallel for developing that Health, the geo with that in with highlighted glomerulopathy treatment program the saving you The received this difference further has this AMD in on glomerulonephritis options our for results wet to Sobi These results it and the of the also section. wet the XX and Presentations studies launches in need of to encourage underscore execute intravital opportunity a thrombotic third investment Apellis lateral listen leading website people life specialists treatments. event to Events in potentially of study I on GA eye our and sclerosis expected in In pegcetacoplan blindless systemic believe that II the the

be pivotal large well month gross monthly our and month in reduction studies we studies which a increased treatment month seen will GA compare with study. of intravitreal to the this to can assessed that and primary safety to endpoint III Phase total design XX of will of patients. in pegcetacoplan, efficacy longer studies, details Now continue results GA that than saw study safety in of and effect be of in X XX. study months, for slide. seeing at our X,XXX lesion The forward announced XX. pegcetacoplan the FILLY, In The III every at treatment time. pegcetacoplan is from treatment controlled efficacy the And Phase that Full where of and we other more look we our the month again X

be OAKS line and results We to top we'd are will DERBY in from reiterate successful. these the and studies believe excited to to we see why like opportunity take QX, this

analysis. FILLY, design and DERBY confirm multiple study as study to the population significant OAKS and demonstrated which same have statistically First, sensitivity and can robust a core that results

assessment DERBY endpoint. more Second, even and more primary the complete an OAKS frequent will provide evaluation of that include

weaning Third, which the sector, the potential reduces cases diagnosis. of exudation required the by bias in confirmation for

experiencing addition, while number reduce we to anti-VEGF treatments of patients continuing discontinuations. study pegcetacoplan, oxidations In which the treatment expect can on receive

studies endpoint the powered current space. to Finally, primary are this of sufficiently the the given in injections meet missed rate

new see and control The clinical new of retinal year. the of reasons, strategy year. are disease. the For will end these last We will is the we component DERBY next be plan the to position all here the addition of advance development in excited later our believe a partner several candidates we into results successful, technologies X as leader complement. and OAKS by of this designed to to Success treatment us product of to

pegcetacoplan. seen on first less strong is dosing, with maintaining frequent The of benefit while clinical focus the multiple studies area

these in of and The are of critical pursuing second role focus AMD is technologies the a over believe forms neurodegenerative turn sharing many neurology. avoid AMD review Financial Sullivan, I that Chief advance our in CX forward about financial on potentially We to the to And to the who more to programs onset Tim treat will expand now call Tim? offerings the [indiscernible]. focused finally, we our all come. results. months new and will play to AMD look Officer; conditions

Timothy Sullivan

highlights the a XXXX. focus Thank with I you, results, earlier Fede. we today press for the on financial full will year full issued just release Since the

proceeds receipt million in proceeds $XXX our XXXX in in primarily convertible the million addition cash, and XX, the XXXX, year securities cash of to in the This also the increase gross used million of associated the of primarily for offering trials costs in for ending period trials, $XXX January upfront in May as As XX, administrative less and full proceeds $XXX October of in III others. expenses million of same of XXXX, equivalents million cash $XXX.X The expense $XXX.X $XXX and XXXX an for in Phase XXXX. offering and and of The million among our $XXX in increase primarily Apellis compensation the short-term additional employee-related period our in commercial XXXX. increase compared for to of R&D cash in for transaction launch, with due December reflects operations. and and XXXX the the for ongoing related personnel costs clinical expenses general full XXXX were follow-on was gross development year same consulting attributable million Sobi Research for ending compared hiring XXXX. had $XXX increase expenses to cash in our costs expense, the to million compared the marketable clinical for XXXX, of and increase from million equivalents additional directors cash stock $XX for due planned fees, to administrative compensation manufacturing personnel among primarily to and professional potential and were the for was expenses year attributable XXXX, personnel full General in to the an year and full XXXX hiring others. in December

full clinical the to XXXX, period in capitalized the for PNH to of net net XXXX. reported on of loss development million million advance We compared Apellis XX, launch pegcetacoplan to $XXX.X continue plan. in potential remain and loss the year to execute For well December of same $XXX.X ending a robust our a

closing second Our now cash back to for remarks. is will fund operations call XXXX. I half expected runway over of turn the to into the Cedric

Cedric Francois

you, Thank Tim.

ahead the slide. shown and today, transformational heard commercial, we this key clinical you year As are and on a have milestones regulatory

of complement-driven busy see, a can work the diseases. a CX of potential on deliver XXXX range have full as you we to we broad As across targeting

investors Apellis difficult like helped investigators open and clinical caregivers you who we and to advance upcoming have in now, and the And through this our trials, have call year. questions. who keeping for on participated I our patients, Before please look would to the forward move We thank employees milestones. updated operator, our Q&A, to

Operator

Our from [Operator first of question Instructions] comes JPMorgan. Anupam Rama

Anupam Rama

the reasonable questions injections? the we thinking potential about we've of think the APL-X recently sort most plan, how that maybe common doses? should on every you of a of potential missed OAKS, DERBY the is thinking other And do and One how month this? the for gotten about stats impacts study, with how missed in a be effect of impact we how Is about be should FILLY proxy

Cedric Francois

Thank compare COVID. looking show to, you The Obviously, But missed of to intend primary have these continue back, Phase to we for quality make to how frequency you the to brief question call. what place we that, we OAKS are missed a way us trials. that you study, so thank as you, the And endpoint FILLY in well impacted to those injections highly studies of this DERBY of is assessment it by the the and that III far. study and great and occurred and answer we missed based alluded so this good that that injections, an have injections, to a hearing joining show everyone assessments with powered injections did trial measure these much, the have on the spite missed Anupam. is of quality. And to was in confident the make we clinical good the in and of seen be

Operator

comes Raffat of question from Evercore. next Our Umer

Umer Raffat

of And the But, CSLO, what scanning in it's patients are wet also in in or sort had you baseline for being of done the fundus how remind percentage done eye? at using can using is, separately, guess, I'm camera? Phase asking the II you Phase really and fellow the be standard the how primary laser, endpoint, us III? I was very the I'd autofluorescence what AMD curious. Cedric,

Cedric Francois

Umer, you Thank that much, so question. for

because start in then which please. part to to trial. second I'm question, we clinical I you So have breaking ask in same the exactly part going first the as autofluorescence the the in that a we And way Phase the repeat is to Phase up of line III with little your bit, II was did measure the

Umer Raffat

conversion patients fellow of discussions one should them these the whether of eye? to had problem. Because encouraged XXX trial recall, guys be they you in that was the What FDA percentage and I wet impact in. will each in or But included rates. FDA put the presumably, with No should AMD not

at So curious I'm baseline? have that percentage what you

Cedric Francois

Yes. thank No, you.

may baseline, had compares atrophy So the natural who and FILLY wet that study of contralateral had eye. of in patients normal recall, we AMD at in in That the XX%. you trial, of as geographic eye the demographic a frequency approximately XX% to

So excluded reason baseline come were into expect, naturally patients clinical studies many clinical characteristics for more think trials, The will back came clinical in these not that studies. time. presence. think, that that III lower the occurrence of that which II trials enrolling normal that not to that particular I patients yet compete spread. phenotype Phase the closer should a the do we Phase phenotype. being we of will be III patients reason that with lower large study having other XX% a I due demographic simply our with anymore The there we from lampalizumab, that on trial, everyone is coming then, But and present that that with into X we course, were are Phase commenting, had their In those --

Operator

from question Seedhouse next comes Our James. Steve of Raymond

Steve Seedhouse

delta data Maybe have about interim a will in or simpler I'm mortality if established depend you they XX% meet. that Phase not. of their COVID if next wondering didn't XX% the confidence I advance talked ultimately have a having if in just that that III that at something think or which hurdle statistical you that reduction analysis, I'll indication? coming steps asset curious I'm Alexion that's has about you mortality you set determine ask up. that in

Cedric Francois

so Thank that for well. Steve, question much, Yes. as you

say, the December enrollment have I we December, the completed reported, end at we towards in -- Christmas, publicly as of So or in this previously should commonly study.

point the make safety So in close now where we assessment. will getting data time an monitoring to board are the

the will in line be future. then the been analysis data completed. expected top should after the full that near be results has -- will We And

Operator

Archila comes question from Stifel. Our next Derek of

Derek Archila

some you think then one the the up PNH. of inhibitor mean, in in the indication, XXXX. Maybe you how that And provide particularly Is about you can terms on just development? throw in shaping might landscape I orals from Cedric, in launching new color that competitive an are some the there just I'll one complement mix in there? programs oral to maybe of be more now with

Cedric Francois

Yes. Thank so question. for you Derek, much, that

complement have So about ourselves. and times, as we by of new development we commented inhibitors excited the many others are

I think we're really just at the indications. beginning of of what range complement control can do in a wide

launch Of that physicians. all control one of day, forgetting the important where, have that when in that the an you by products will consequences. is in reliant room can error. liability looking to may on pill course, a a that that these forward alternative PNH, for the introduction out is extravascular patients, patients, in twice in twice-a-week example, I in we on of for for these no these to control the mean PNH, disease product be of you hemolysis. and couple need we much our oral PNH believe, important per What like majority compared could to a may of oral bear are pill find the to take take in of that whether the subcutaneous that a mind patients. need care to is the That importantly, fact a more there neither of also is line the now PNH address durable particularly and with, years, products complement In if have seem product, the in very first being long comfortable I extravascular believe, with elevate standard respond to we very still patients not that and control the of thereby well appealing are In it pathway run. is the to surface, patients to it's that many a And while hemolysis

the of lot less indications forward we I'm complement beyond look III of excited exquisite personally development so not a note. want the this positive opportunities. very to oral kind it readouts. of I on in products, of So in important, I provide other about control is to much, end a But the but that, Phase think where PNH, will

relates on the look it excited not comment ready own As come. we forward sharing but and are months programs, are that, to to our to about more we yet internal in

Operator

Our of question next Co. & Oppenheimer Justin Kim comes from

Justin Kim

PRINCE. Just maybe on

walk we like treatment As could insights geographies care naïve what we APL-X complement being particularly see from differ may standard unique perspective? where through maybe population, perhaps prepare you here is us for from to maybe and of that this a used readout, the where may

Cedric Francois

so hand to to Chief you Officer, I'm going that the over Justin. for much our question, Thank Grossi. one Medical Dr. Fede?

Federico Grossi

the Thank you, study of you to sharing looking We're Justin, and results. really outcome thank Cedric forward you, for with and that. show the that

is a endpoint studies. and study, is endpoint hemoglobin population which the looking looking typical point transfusion you So the as for PNH addition at quality dependency. that, is of out, at to complement inhibitors stabilization, to And, and a we're PNH that in is primary life naïve

regions on defer. population the where available regions So order it, does differ available. eculizumab not in but do treatment-naïve that eculizumab to population, to not the is We had does -- population the not that see from in population is studies this you do where the

Justin Kim

disease? a it. previous maybe Got severe sort And we're who a assumption pan-AMD Okay. fair maybe modalities just question less it of follow-up maybe sort you may maybe about we is have -- do populations if When that outside injection, a at of think intravitreal see looking therapies, to and you slightly of ask differently.

Federico Grossi

the AMD? So can Or on question you repeat

Justin Kim

think a potentially? therapy, pan-AMD new we non-injection as sort wondering, agents of based a sort Just in that suggest about of does therapeutic,

Federico Grossi

this look -- you at AMD. Well, outside is of when

will you are eye level systemic too that exposure For treatments of diseases eye, not maybe into the high. injected the that the into get from

having to to good systemically, market from in and drug high there perspective. therapy So to exposure safety a patients concerns will you systemic achieve that be can is levels have a the the

Operator

comes question from Cantor Fitzgerald. next Our Alethia of Young

Alethia Young

One, going just to mean, obviously, be of for me is Alexion -- on -- acquired. kind I is

I had bit resource analyst sensing human able get of, about which or just robust? the that take more commercial And standpoint, registrational, is mean, disruption And challenging. then perceive you reward do a advantage is you're wanted a that being though is bit we Or standpoint, on moving I just a is of that as occur. perspective towards might opportunity second do I interesting kind that can on you little to there's equivalence? the are of should sort of a in question to from quite little you your higher be programs program? already any sometimes even higher a think whether, X kind and all them always risk, -- even

Cedric Francois

Yes. with thank first question. starting Well, you. the No,

elevate main are driver new place hope an the controlled. primary the cascade be the that agglutinin an need indication Our as that. advantages indications, place. to with important in place exists day, sales, special this has as, also, offers a of to the has has a look are other over of forward in where disease and unmet And developments, in course, -- HSCT is glomerulopathy currently as that where microangiopathy. is thrombotic registrational the special disease of care to to or of so the as end that which targeting. It we continent CX incredible landscape and standard CX the the which places to in other indication believe with the a IC-MPGN, addressing relates can changed. unmet associated PNH are we address. believe The we Then, going a first neurological special there we has But, that ALS, we need cold place competitive trial it The It that ALS be at of PNH. goal X,

Of well course, with Any ALS us primary pursuing to was that do plethora the we motivation and molecular together we bring our again, it Is high-risk of high-reward belief fits we we associated, is the is. program? that can So, with believe with but need. the truly have into in the that of indications systemic for for the one kind entity science that are trial a this us, address a unmet very pegcetacoplan.

Operator

of Our next [Laura question comes from Christianson] Cowen.

Unidentified Analyst

in experiences recommends booster. seen reaction received not the the who Pfizer PEG people have that who allergic and my that CDC reaction vaccines. believe, question sporadically I immediate to is an should PEG anyone allergic have actually So to about the the been Moderna receive

not, that So that the your I belief, sensitized being And if to it's assume you what patients wondering but that's just why are confidence is case? was also I gives PEG?

Cedric Francois

Yes. Thank that question. for Laura, you,

associated there sometimes as that especially sensitization. beginning This cases PEG is that when of you important, a the isolated extra that good ours, follow-up. the there are correctly make be to an you here, the the can as there introduction be immune sure to something is sensitization them, not seem So vaccines is mentioned, that products important therefore, does anaphylaxis however, seem occur. not is It's is of What well-known, introduce been like with that because to do that PEG a with of have well, reactions. type with with to couple note any in the element associated in of booster vaccines additional

Will far, now. not been rare where diseases, the comfortable Thus we I these that with in right, that possible. see anaphylactic mean, future, we, reaction, a very in has right case. in So the stand an while, is once we feel

Operator

next of Our Capital. question from Matthew Luchini BMO comes

Matthew Luchini

on really sort in question. look And secondly, that bit they sense of think around are a I'd think there to of what beyond? next sequential visit? about market a versus in as XQ. just we love you if going doctors first, launch. is as should -- and more forward? the little patients just the think like for going are It we about to internal And color Or a the should looks your year patients for for that's kind forward R&D I'm Any the a something sort our the to step things get kind of this initial saw telling you I like? telling to help how about And your of the of initial that asking color what new then that baseline research wondering planning could are the little drug? patients looking call pretty housekeeping we that So waiting as PNH, is commercially, guess into like launch is I'm down what around Are expectations set decent

Cedric Francois

your first will question. Commercial over Matthew. second question our Tim I take Officer, Chief to the you, will and Thank Townsend, then Adam hand

Adam Townsend

Cedric, Matthew, you, you, the for Thank and thank question.

caregivers truly the as understand that content. So market. the their activities expect patient. a we're marketing standard some the there, of we've everything community, with and potential we the care the to lot PNH believe that unmet conversation exists we time and getting with they a have with need with surrounds within to our spent great interact various patients, them They that And of do pegcetacoplan. the from patient-focused got We've a about patients physician response elevate out

story you've the that whilst this very our the previous identified unmet with much in efficacy seen So physicians need. an CX driven have [third]. And presentation treated we discussions, patients market at will the from our at we us, and is launch, for that highest look of think

this as patients that we also actually conversation transition to the have piece their It highest unmet with launch. call to And need identified the disease. in. when And conversation from broader is be have already A very core for to obviously, expect unmet of will that in approach pegcetacoplan. physicians an expect potential the we be So thoughtful we patient to physicians. the will potentially need a and a physicians rare some patients important them of go Still -- a and around patients patients population with and that to the have work through.

Timothy Sullivan

And Sure. for R&D question. I'll the thanks, question. the take Matthew,

is terms in of accounting So XQ R&D the and concept. seeing in what more actually step you're of Sobi-related down an

those Apellis way those the of next of are So deal, as of expenses XXXX. the you accounted over and started primarily when R&D worth from X $XX in reimburse course the for we'll know, million fourth for structure years. quarter, of Sobi, begin the The the

And so that is actually counted as account. a counter

And over next amounts just Ultimately, X number will were for million so the brings over and the that $XX from perspective, a the from reimbursement time GAAP years. come R&D that down. those deducted

the expenses not true look although bit year. third over steadily, closer the up, at the much dramatically would R&D to I probably So rise quarter, little it flat next will and a to

Operator

[Operator from of Instructions] comes Yigal question Our Nochomovitz next Citigroup.

Yigal Nochomovitz

DERBY on and one had I OAKS.

the pegcetacoplan was X.XX deeply growth the as absolute was lesion in and square, monthly stat sig. difference sham know, meters area the between obviously FILLY, Cedric, in and So you the that

lower higher so is and growth in and the area lesion DERBY difference hurdle be at Now hit in power OAKS could DERBY GA on presumably, a you the that the OAKS comment successful? all bar to the for for given you to need

Cedric Francois

Thank you, Yigal.

we the powered the show effect trials III in Phase XX% more So Phase II trial to for that same clinical clinical dosed the are saw monthly than the individuals.

for variability somewhere dose of And This XX% XX% the X.XX. individuals. accounts of presumption of for and similar between is, a month every other p-value and course, on

So the again, p-value made as few protect in have that kind sure of possible. trial, changes clinical the II for got to Phase us we to we as

same same and We are studying patient We the analyze exact population. in reap the the way. findings

All of Phase and maintained that between II III. was Phase

Operator

Our Chico of next question Laura from Wedbush.

Laura Chico

perspective? frequency baseline if exitation the have And how could if regulatory perspective; a I maybe thinking to be just comment on wondering one just a I in there, lower expectation? to rate from is circle normal new wet follow-up. that that Just around of patients around acceptable back should wanted you indicated frequency quick the I'm curious DERBY what about a an then respect you is of b, from and do commercial a, change and onset Cedric, a kind with think we then might XX%. AMD. OAKS,

to whether, respect of would include just remind extend bit with your Ultomiris-treated also a talk about us you Cedric, APL-X could expectation PNH, or patients. to labeling Just little

I think with that Ultomiris. a there you had slide comparing

be for about of a patients transition? able data So what type guidance thinking might provide to or you

Cedric Francois

Thank so much. Yes. you

the occurrences right? part expedites, which trial to important AMD are Phase II question, that contextualize, These clinical starting So small of the with the wet were first your we in had not clinical and the loss the trial, vision to did significant in that same II. is as we why Phase patient in are exact studying population the lead III did Phase we

regulatory trial. had increased benefit bias to in so expect the AMD contralateral that an is, And from as have rate also with in many of you that Phase came they with exudation, saw that in of of when was physician should acceptable in investigator because treatment II we study. higher the that some patients went Phase we may frequency the your question in have had perspective the study already hindsight, clinical because is it perspective the into the exudation eye anti-VEGF arguably And III the well. Phase what contributed terms from exudation what or II artificially a we a than the So in is rate

that would clinical been to corrected our unmet INPDs for rolled have I the in message the that and All had of that been we gives if Phase probably X if important of the physicians desire that need here hadn't for of III these kind in you take-home Phase for treat hadn't of to willingness it patients. wait exists will and COVID, trial, sense the the Europe months. a mean, been I But that is be think most the clinical in it trial. III

purpose of of look we So patients frequent exudations wet what in with we and clinical a geographic geographic we our the patients are atrophy. listeners the trial. bear for years. based and atrophy, is done developed III haven't largest a over cases AMD. we anywhere, are It the exudation Phase encourage how normal patients find presentation claims baseline, But will looked in on was at believe Ophthalmology. of the patients base X -- And start geographic ever geographic atrophy to with strong case, pure no done, kind phenomenon, Academy with atrophy with that at see I meaning very importantly, see that study a and the data late-breaking I retrospectively where we at with it is that XX,XXX out abstract It's a in what would American to is to its yet mind based and X% wet AMD was number to develop on

eye start has If the years. of over one then that XX% of contralateral wet the course a GA eye in you X with AMD in and developing eye, AMD off wet GA

to get comes these observed. label. your is to as So not to the what And nature of exudation it back second that at Then, are question all unusual. down it relates the to again,

label So would, applicable case, that patients. broad course, as are Ultomiris we have And broad are believe we labeling. of label our would patients including well. on yet baseline treatment-naïve and not that on A that approval. in be will particular we when mean, We commenting in get usable a

Operator

back I'm showing questions turn no call like to over time. this further for remarks. at the Cedric any Francois closing I'd to

Cedric Francois

results inaugural financial much, everyone, you Thank joining on our conference us thank and call. so you, for

by in reiterating to We are to diseases. systemic a for us. strategy pegcetacoplan We #X continue transformational complement for complements-driven control establish for corporate complements would become we our first like today. and atrophy, technologies across I on again year with therapy aim excited disruptive close treatment complement. joining the about geographic to as the advance in innovative rare the ahead leadership to We CX. focus to with us for retina plan Thank you factor a

Operator

and for Ladies you. participating. Thank today's Thank you this all gentlemen, does conclude conference.

You may all disconnect. Have day. great a

Apellis Pharmaceuticals (APLS) 1 Mar 212020 Q4 Earnings call transcript