Apellis Pharmaceuticals (APLS) 9 Nov 212021 Q3 Earnings call transcript

Company Profile

Participants

Meredith Kaya	Vice President, Investor Relations
Cedric Francois	Co-Founder and Chief Executive Officer
Adam Townsend	Chief Commercial Officer
Federico Grossi	Chief Medical Officer
Tim Sullivan	Chief Financial Officer
Umer Raffat	Evercore
Anupam Rama	JPM
Phil Nadeau	Cowen & Company
Steven Seedhouse	Raymond James
Madhu Kumar	Goldman Sachs
Justin Kim	Oppenheimer & Company
Tazeen Ahmad	Bank of America
Colleen Kusy	Baird
Alethia Young	Cantor
Ellie Merle	UBS
Matthew Luchini	BMO
Joseph Stringer	Needham & Company
Laura Chico	Wedbush Securities

Call transcript

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Operator

Good day and thank you for standing by. Welcome to the Third Quarter 2021 Apellis Pharmaceuticals, Inc. Earnings Conference Call. [Operator Instructions] I would now like to hand the conference over to your host today, Meredith Kaya, Vice President of Investor Relations. Please go ahead.

Meredith Kaya

you financial us results. thank third discuss and XXXX quarter afternoon for to joining Apellis’ Good With and Dr. Chief on Executive me the Officer, call are Co-Founder Cedric Medical Townsend; Officer, Francois; Dr. Chief Chief Commercial Officer, Adam Financial Grossi; Officer, Federico Chief Tim Sullivan. and

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

Now, to over I Cedric. the will turn call

Cedric Francois

Thank you, everyone us Meredith, today to joining for our call. good and conference afternoon

EMPAVELI data, period first DERBY quarter another exceptional continued and updates, which third our for was our for and GA position for Phase advancement the we X become by of pegcetacoplan to pipeline. strong believe OAKS highlighted the The and PNH, treatment U.S. execution commercial

lesion unique potential this past in will safety a every from the achievements studies, Our most and our start showed leadership the which are with further significant OAKS. complement. our quarter targeting position I quarter, in the the broad CX dosing. and clinically month of growth reinforce other profile favorable event of GA approach underscore In meaningful results and a with pegcetacoplan both reduction and these top DERBY line of monthly

with treatment an extrafoveal Importantly, in supporting effect results progression. patients lesions, review pegcetacoplan showed these Fede shortly. even in greater disease earlier will

cause disease people that pegcetacoplan of breakthrough leading of the a a for relentless is with With we a living worldwide. blindness believe these data, millions is GA,

to to priority that the in the these uncertainty company. clarity high with remains community the near both path we and in on for Gaining results, We GA regards forward us right intend within a term. how there then now, are is we capitalize investment but understand both our thrilled in

discuss our FDA submission be with will the year. to share with feedback before strategy side, regulatory you the plan meeting the On and to the we of end

later, approach Tim taking well as that evaluate needs, future. thoughtful we are we various will a the funding for we Regarding positioned detail financing strategies are so as

for Turning only PNH. expectations across since to launch net $X.X our first each million of therapy momentum our In launch, approved EMPAVELI, first product quarter treatment revenue, full our EMPAVELI targeted strong the CX and and delivered the of metrics. in showed exceeding

Additionally, month. pegcetacoplan, opinion Sobi Aspaveli known be which will CHMP by the as a EU last marketed positive in and received

end this decision in the of become We the PNH therapeutic goal of expect from to approved, If our approval European approach for XXXX. a new elevate first advancing year, the European the further regarding patients will the by standard since Aspaveli Commission the for world. Union around PNH care

rare are the in Together we disease Sobi, to our in PNH portfolio with efforts advance are building continuing steps Our multiple a indications. robust first just franchise. across

steady milestones to X over of late-stage XX months, trials to in X a have including the separate We cadence of XX start registration indications. designed support the next

ambition our enroll most Ultimately, opportunity. continuing the leader X immediate represents complement. to the in also this is Pegcetacoplan patients in for registrational potentially and ALS to Phase are our We our is goal, become this study. global foundation

discussion for these providing will more diseases. We programs, of of across complement-driven programs forward of growing to a on the these a details have candidates several to me let turn our efforts the all that behind we call a Adam address advance. Adam? However, range And as portfolio modalities on efforts. us commercial over broad look now allow to

Adam Townsend

Thank you, Cedric.

is As Cedric a mentioned, very launch start. our EMPAVELI to off commercial strong

to launch EMPAVELI priorities, who of ensure to medicine. has important each is eligible which across progress every want this great access PNH new making are that top designed We our patient

currently being CX are U.S., the patients people inhibitors with within each another previously, XXX and said we treated are diagnosed year. As with who X,XXX there PNH approximately

with on suboptimal to of require for control is patients focus beginning with transfusions hemoglobin their many We disease, their of have those whom to those unmet initial falling levels. to PNH patients who third the address expand the on plan a are who highest broader Our need, who of PNH EMPAVELI signs CX and figure, inhibitors for launch community, their also a symptoms program XXX patients. suffering an potential from as impressive the of treatment have and fatigue. At end signed since indicating over significant anemia physicians number of within have like the severe identified U.S. October, physicians REMS option up the our

third last alone quarter, XX start Ultomiris. forms October. with in the starts finding vast switch of new EMPAVELI we are patients start XXX XX% from since than over forms launched about inhibitor quarter through majority switches that are more coming received and we with of the Additionally, Consistent CX

plan date, of result, the to pegcetacoplan, front, clear this agreed that the blockbuster need engage and XX PNH there as continues a top XX and from on their believe to of a XX opportunity in if the these EMPAVELI We for including the on cover positive On payer market a position. They team XX% potential payer surveyed to the patients’ high-priority preparing for our payers, is the is initial treatment bring of access the belief product. To with place specialists and all parallel has on early U.S. team reinforces highly prescribe in progression have treatment support commercial potential feedback plans value a of to treating approval with approximately launch, in pegcetacoplan earlier approximately patients. disease Based to remain our end encouraging track approved. EMPAVELI our prescriptions. OAKS data to In and a derby the by with on year. retina been of GA finally they of and research, execution in that the be XX% formulary formulary a of the who

a and GA huge. the in this how anything paradigm don’t As like treat I you the launch. can of specialists Fede for on will complete the we on be providing clinical as look have over some our detail turn shift to is approach see We GA. treat of plan this to we Fede? retina include forward would this a slide, to from comments feedback and development. We now commercial potential more to our review prepare call

Federico Grossi

reduction a going the with patients presenting compared In the significant the lesions, I clinically an meeting first to the September. other lesion in analysis, Society highly growth Adam. forward every These as and in met treatment data. in minutes and pegcetacoplan in arm. sham and next the GA American at a Retina am the OAKS prespecified as you, a to by providing Academy again monthly statistically XX presented the extrafoveal few review of even spend for study, XX% Thank the OAKS look later month. In at Meeting level high to month pooled data reduction We this DERBY Ophthalmology meaningful of endpoint, showed much with time primary providing at monthly pegcetacoplan greater months. were

and monthly As Pegcetacoplan, DERBY, XX% typically progress p-value as with suggesting With extrafoveal fovea, of research fovea. lesion the treatment in with the start of statistical lesions a GA into a the in later reminder, outside the [indiscernible] as of significance begins that very arm. patients X.XXXX many

In disease contributed imbalances enrolling DERBY? with may sham the associated to use lesions, you of imbalances growth reduction in an in the However, In see be A have analysis lesion and rapid extrafoveal characteristics in to pegcetacoplan can to as expected groups, show offset again in [indiscernible] as the base a is reflected known progress slide. quick faster this these progression. in in presence pegcetacoplan mix. DERBY, greater we the compared why which patients question

treated lesion patients, other see in we eye robust analysis. the at untreated the to eye Therefore, fellow when treated in this sham the relevance analysis, Turning separation and fellow you this grow further fellow validation versus look between untreated lesions for of study groups well pegcetacoplan looking confirming clinically do Overall, the monthly the growth an at in the pegcetacoplan eye GA eye slide an the see serves eye you DERBY start the DERBY fellow effect. the at across and the analysis as eye In not sham-treatment important And the rates lesion treatment month you both big with finally, even first treated the rate known when and of is growth of consistent groups, similar the look and to every of untreated the groups growth as the treatment the OAKS confirms therapy in eye. both when effect, OAKS. the to versus are shows demonstrate in versus eyes. separation a between eye. which fellow cords expected, eye. at Then This the patients show lesion investigational the versus bilateral reductions meaningful GA, more follow study difference to pegcetacoplan both

and in rate study, In [indiscernible] demonstrates new therapies. our call favorable safety half to X% in X treatment safety, across safety from reported supports of in of intravitreal FILLY well-controlled line exudations than XXXX. track for Tim pegcetacoplan’s inflammation and in demonstrated a monthly of X.X% our pull profile statistic, Between on with other in the treatment remain believe those results. randomized review NDA the studies I with data generally and now the We across will Phase terms which financial approval. group, month to OAKS were X.X% endophthalmitis and Tim? efficacy sham first turn we of group. the of the providing results patients group we pegcetacoplan And X,XXX submit from every other of intraocular now our have patients X in study, DERBY, studies, robust both and more the the

Tim Sullivan

highlights were revenue, start $XXX of earlier focus product quarter million, full issued and million third a EMPAVELI from third with which collaboration Since Fede. $XX $X.X $XX primarily associated million, In will total you, our we on for was the of $X.X the the consisted we a with Sobi. were Thank XXXX. quarter release revenue additional expenses launch million. financial G&A of with results, XXXX, net net the I and loss revenue of expenses today the of R&D reported million, press just a strong

acknowledge As $XXX reminder are A the cash of that XX, and in quarter will of capital in cash XXXX. XXXX, the additional we collaboration equivalents, that fund million quarter. as which Apellis during done. a September cash we had platform associated CX third we third was payment to will We do need thoughtful in leading beam $XX operations that our our as we million with raise to advance into have expected and always paid our manner the so

multiple royalty, path regulatory our more in equity or to as evaluating are financing strategies, partnerships paths atrophy. to from geographic debt approaches strategic or advance work other we We ranging traditional simultaneously

the believe across organization With a are we Importantly, we in this back access are GA to remarks. tied turn we a deliver dating also capital for blockbuster ability Cedric? EMPAVELI, confident value interim tightening expenses, for derisking our way in will period. call during in our shareholders. long-term Cedric us I includes hiring product potential help will pipeline, the spend to robust and which to approved milestones a certain closing an in and over managing now that

Cedric Francois

Thank you, Tim.

first X Phase and of in product, commercial X PNH a transformative studies. XXXX Apellis and a the we period showed a The as profile represented OAKS with for meaningful safety GA lesion and first EMPAVELI reduction months growth of in clinically DERBY our favorable launched

this are complement. growth support our We building in committed leadership to position on and further advance momentum to

portfolio. of XXXX, GA number study in our have X IC-MPGN Over Sobi in initiate complex remainder and months, a next for to from FDA expect to the EU CX pegcetacoplan key CXG. approval immune milestones expect with receive of the or to regulatory Phase or a across XX glomerulopathy membranoproliferative glomerulonephritis to PNH XX and feedback we Beginning in we the

the to the in following well ALS. of Phase reiterated will is This cold thrombotic XXXX submit also remain In enrollments in XXXX, in as is our drug EU pre-submission the in half COVID and that Sobi to EMEA transplantation-associated year. of microangiopathy our HSCT-TMA we delay program first now on in we and to to for hematopoietic agglutinin recruiting disease, XXXX. enrollment GA earnings European countries a to as other begin complete Enrollment discussions ongoing their X or that Additionally, in or partially FDA ALS study in but initiate our partner, of our regulators slight set for we half XXXX. their expect completing to expect launching due with trials new is cell ongoing, be plans application begin CAD, Sobi about submission and in report milestone-rich recent stem Aspaveli approval. first EU a in to the track study they

published X inhibitor other a the study We immune pending brain first-in-class complement regulatory to year next for to neurodegenerative be with preclinical XXXX, approval administration for activation our AAV system prevention we GA. for DERBY of CX to half Phase also with results new APL-XXXX, indications. U.S. expect of vector designed coincidence a gene the the inhibitor In and in initiate expect to early therapies data AMD and for decision for receive feedback diseases to submit report intermediate active IND an potential OAKS XX-month and a CX from second

to for made advance progress, operator, questions. our the providing now excellent have please we forward updates efforts. We as we call and look open And

Operator

Umer first Our question Instructions] [Operator comes from with Evercore. Raffat

Your open. line is

Umer Raffat

us I it X? currently? there And on you taking any I that of maybe next beyond of about also complemented of is remind near-term. GA if [indiscernible]. substantial continue what those want quantifications seems for And on particularly the to the [indiscernible] or would any questions. expect of going understanding Potentially, is year, then end on understand could bottlenecks my that a practice be great? penetration the to Phase to talk a news Thanks opportunity [indiscernible] FDA feedback get to some some bit indication? indications of you so in the Could initial fast pipeline and like the especially from [indiscernible] it And around you will of much

Cedric Francois

so Yes. Thank much. you

FDA outlined for as it the submitted earlier, meeting as studies. to a request and DERBY the X, the OAKS a regarding So to relates have have so we Phase we

to Christmas, update we as like. to street Before landscape looks to provide what’s an plan regulatory

on. the FDA are the is before data expect that there Specifically, submission. we doing actually two are One – need important clarity to we to wait elements would the to whether get us XX-month hoping for

exciting going may working this off needed clarity so pipeline, demanded, be getting would we at CX the believes programs indications. four indeed not. four whether in it have, be to in is This or that indication call additional of on as is be believe the for which these Ultomiris is as very sometimes we EMPAVELI of are and ones one to the second trial one label. Soliris glomerulopathy the registrational that requested time, neither an on mentioned and that that We And FDA are additional on. in used The point another those Then important. but indeed, and going are relates we

based a We based whether to have believe a indication we a to component, we that in patients by that also, to these X we is represented the of X form product are more for this clinical to running well. that that potentially. generated, I the a best-in-class similar trial, our half point classical Phase and essentially But are on driven Phase And be where a objective in – is but identify CXG, the mechanism, for pathway EMPAVELI the very which both this treatment is opportunity important that of the can indication. study, an on data we or presence as and importantly kidney will the the say, therefore, other I’d like antibodies the patients IC-MPGN be enrolled out that are is one be

Umer Raffat

so Okay, thank much. you

understand foremost bit performance. traditional first combination also Are preferred I Difficulty] at And you little covering what Difficulty] looking financing Just you one your now. that? exploring [Technical to open all perhaps And right a lot of sending a our you’re [Technical remarks to would financing a solution for there bit. more. I for be? the about less are and concern that could options beyond about your appreciate you little is and

Cedric Francois

one hand Tim. that will over to I much. so you Thank

Tim Sullivan

Sure. the best to do question. feedback also I’ll my And

speaking, you I And certainly traditional are first think other correct, needs. is context our acknowledged to – we of asking, were less of open in the generally look, all? forms of the Umer, financing aware raising that of we’re capital

Umer Raffat

Yes, so.

Tim Sullivan

Yes. Okay.

our partnerships conditions Those exploring and current of royalty, traditional aware So paths. options. strategic more ability could or from impact that some equity, raise those range of the raise capital, to all we’re we’re debt,

things. entire is as for expenses we while taking our it we’re we’re said, to is. cost basically our of shareholders, the options. mentioned, point, where at But at this the extend we’re simultaneously, also as our And runway range we all capital do our right really, time will looking to looking So and we at at of thing look

Umer Raffat

Well, thanks so much.

Cedric Francois

Thank you, Umer.

Operator

next with question Rama Our comes from Anupam JPM.

line open. Your is

Anupam Rama

alright? the me, Hey, Can Thanks question. much so taking hear for guys. you

Cedric Francois

Anupam. hear Yes. you, well, We can

Anupam Rama

Cool. Alright.

a PNH early, Just And any think about over the patient data synergies the therapy. getting a can so is time? one know on points the time I form launch, but much. on on we start start on quick from to any Thanks there forms, it’s

Cedric Francois

you, Thank Adam. that hand one over Anupam. to I’m to going

Adam Townsend

Anupam. can Thanks, Hopefully, you too. me hear

the the go the a So What your forms progress launch is drug from and it to of very Hopefully, required, paperwork the that but patient in question. pleased patients, yes, X transition X only for launch, very, takes transition Anupam, takes the through normal start of pretty finding, a all once disease we positive we’re with weeks, to through to on PNH. X and from which across. X not we’re between on that EMPAVELI is physicians, hugely weeks answers it process also rare commercial get average but patients, of of between the we that’s go we all REMS – feedback

Anupam Rama

for the much question. so taking Thanks

Cedric Francois

you, Anupam. Thank

Operator

with Cowen & question next Company. Our Phil comes from Nadeau

open. is line Your

Phil Nadeau

in data thanks considering? for on you’re EMPAVELI. something we’ve and on package. strengthen other could in our lesions that GA, some of extrafoveal would regulatory Good the taking it just the satisfied be A questions. one consistency FDA. Is the in couple XX-month the lesions? dosing every Is suggest possible that the afternoon. in something to On specifically terms with? had strategy, Congrats appeal quarter dosing approval that us the every arm we’ve extrafoveal then And to for apply suggest month had on that for other and also for KOL the KOLs the you’d GA Is

Cedric Francois

Phil. so you Thank much,

well, questions. So those first let me two take

months, that us specifically is that not was that had XX the there KOL whether we With call – you feedback. had given that – a was

or that guided never have We that. mentioned

of has came of that misunderstanding one conferences. a out think that been So I the

every believe breakthrough all we it studies. There trials every run met it we happy patients atrophy that data, data studied is month application. that a three other very course, but with way I especially are, represents As which for because extrafoveal is the of to we month to the the relates in geographic expectations. attractive, there exceeded for we or the have very, these the patients, pieces early we One are our safety, think other

does is Second that in work? context, telling. data the the is very And drug

each by we in we the kind XX% making seen equal that baseline may of with XX% we’ve in have other believe what post And That the why we and is analysis, make drug. fellow the more of the population clear And three is from to also the broader believe lesions. XX% with And by essentially the that be which and benefit further the is the for effect is ran OAKS, lesions did north extrafoveal patients size. third of we hoc trials DERBY can then We the you effect where eye a range analysis, there, confirms these one the effects to more that effect FILLY, lesion. size. in this

the whether we one package. submit be of going to these take course, but will data and wants as that of sort all to of to them, up FDA All up is that,

Phil Nadeau

you do Are mentioned interactions, do the the review position after the you very XXXX. That’s I meeting. will disclosure think making be of to before wait Or in in then of FDA half you it, you minutes think an right end And the approval of for your a in meeting by then on year terms second the year post going the to timelines, similarly suggested FDA the And helpful. disclosure? you meeting. a

a that does X-month a because assume Just review is expansion? to label this clarify,

Cedric Francois

Thank Phil. Yes. you much, so

proper until we and comment So FDA can well-qualified so not have we we on representation. interactions minutes make do a

that have that like be to So our we’d hands. will something in

to As the indeed of priority half set region. relates premised we it a the approval, second on that year, is next and

review, the as in product drugs we last all know, as priority well. received the in retina our far growth So as and we have category that years, in believe XX that

Phil Nadeau

And then is from on the last quarter. solid launch. on EMPAVELI question just number Congratulations the for Perfect. us

Ultomiris, You that Are days? patients broader targeting? mentioned require the most on the those, getting EMPAVELI a you swath switching in patients you’re fact, patients in that who of Or to are early are switched. transfusions population well

Cedric Francois

have be Phil. I’m going much, very this so may transfusion-dependent into to patients over we one hemoglobin you Adam? levels breaking we are hand and Adam, Thank of that segment are that not to that normal excited happen. but necessarily more

Adam Townsend

of of the Yes. Thanks, Phil, as Cedric getting we X,XXX CX great. and treatment thanks across which Cedric. treated segments the is summary, patients, so from Yes, for the patients segments, the including all naive are described,

with unmet that the the patients, tells the are the think have of physicians highest those of ones A do with the standard low PNH, hemoglobin we that as and need. can of have would Ultomiris benefit majority the require care expected, they particularly of I transfusions switches and us bonus with patients see EMPAVELI. elevating

is team well as all we’re those of treatment unmet as across executing, as the naive. said, patients from getting segments So as Cedric need

good. So progress is looking

Phil Nadeau

my Perfect. questions. Thank taking for

Cedric Francois

Thank you, Phil.

Operator

question from comes with Raymond Steven Our James. next Seedhouse

line open. Your is

Steven Seedhouse

and six on so about seeking question actually that. commercial you think analyst that for Thanks. Yes. I all but is in to hiring Congrats field you’re a without would this beat not be you What is partner? independently that commercial you’re in quarter, want your you mentioned consensus, U.S. and to Thank preparing because afternoon. And the the does does kudos look you slide that down potential you able launch good in a force. like? Apellis an indication beat only I estimates, GA GA. drill And that just launch on that ask, a to mean anticipate My

Cedric Francois

the over for that Steve. words. going you hand you Adam. to Thank kind to one Thank much, so I’m

Adam Townsend

Yes, you’re right. Steve. Thanks, absolutely,

out are We’ve started of build opportunity. our a blockbuster commercial already as global see the behind We what to team we in scenes. advance ophthalmology

and So leadership the we’ve Australia. and we’ve hired European in affiliates our built our affiliate, within out also the marketing team U.S. and and sales Germany

we’re ready can to breakthrough in so. GA, So we we this scenes preparing launch behind truly do thoughtfully product that the get believe to and

Steven Seedhouse

more I’ll back maybe to just but device the the one The commercial, given necessary, you it’s ask on still do to might PNH. maybe again update Actually, Thanks. strength launch Enable us the – there, when you Thanks progress and market? of think could just coming that be again.

Adam Townsend

we’re Yes. fans Enable Thanks, of Steve. device. Yes, the big

We’re train support are PNH also how thrilled services patients. with our patient helping new

device. there I the to opportunity think care is launch the for a of elevate Enable to great standard of us the with continue

So launching all launch. as the we’re wanted We of to processes also the device. of systems our well are still And planning first and make possible. to we initial get through wanted as that on sure our phases working

way start to to best get we think will seeing So year, Enable for into launch at the a top we We us benefits switch they launching look period have the on if patients EMPAVELI. of benefit we next as the are already of device. they will big through the

Steven Seedhouse

you. Thank

Cedric Francois

you, Thank Steve.

Operator

comes Kumar Our Madhu Sachs. Goldman next with question from

open. is line Your

Madhu Kumar

for Hey, our question. Thanks taking everyone.

on approval a timing GA. follow-up So Phil’s question in for kind the about of PEG

in to the imply So even second approval half with relatively would filings. half priority have early first ‘XX view a

turnaround a quick So after interactions year-end? of assume to is the that preparations get reasonable in made time to to relatively NDA by kind for FDA it be an

Cedric Francois

to and Madhu, you, hear you. Thank great

top the preparations the we started line NDA for on we – working have got data. We when the

So yes.

Madhu Kumar

that about about some throw on How there will these be Okay. some should similarly, indications of and think CAD of other CXG, of going of run And of we you be expansion think out or should versus with largely trials, trial we we line of similar with other the previously. when they when And marrow these a Sobi, kind of TMA the caveat go about with indication. Sobi, back design of about what’s bone as and Are then being kind start. sutimlimab the them. how you practically been for by think as buy particularly registrational for we the in scope trials kind the kind scale EMPAVELI scale so big is the seen scope to trials like versus

Cedric Francois

Yes. Thank you, Madhu.

And we FDA. have at, when interactions before We design look endpoints obviously, that geographies as don’t was the others design applied registration that? various add what And make want feedback have. the on of to the competitors sure alignment too, the the the But from our own for that we Fede, that all and evidently not trials, we we if us have at harmonize that’s fact the something there have and So to the studies. to studies try mention we regulatory four that I these we know with of well. you done. our to

Federico Grossi

No, Cedric.

also are development do what from deviate I but you incorporating the not not there. the generally think bodies regulatory FDA very they you out programs global but are European These feedback, the hit on spot. We try so see – important,

Madhu Kumar

you Okay, much, great. Thanks very everyone.

Cedric Francois

you, Thank Madhu.

Operator

Oppenheimer Our Kim & Justin next Company. from with comes question

line is Your open.

Justin Kim

questions. the Hi, good afternoon. Thanks for taking

CXG. two, one Just commercial on and one

access pace of bit how talk by the the those what of impact, been the any, forms, With third the can variant Delta that influenced early sort concluding? might have you if programs then And about a had COVID-XX little and quarter? forms for start the are

Cedric Francois

Adam? Thank Justin. you,

Adam Townsend

Thanks, Justin. Yes.

the is and seeing a are we’re enrollment the is the transitioning – out across we there to precision commercial all product, demand demand transition from seeing we’re real. access but And They patients. happy REMS program our and So it’s early – with a start how really to form agnostic progress. that of we’re that

of So your question. that’s the first part

My of asked moment. I situations, interactions But we’re in the the me drug of of those be in-person all no. very also time those thrilled if Justin, are about is we’re But to at team of managing the – calls. virtual most launch rare part so interactions. we is – wanted in The disease how And the XX% person to our would second pivoted global with making quickly answer you always a I’m the pandemic.

how hugely feedback to well. and on pallet at that the administer as train are our interacting we’re virtually care They patients getting So the of on educators request physicians. positive who etcetera, product, help

So needed. interactions when we pivoted pretty as well to virtual and

We’re all also of monitoring the situations.

PNH follow all the we’re will protocols interact and compliant and as well COVID interactions, we’re to everything launched that PNH physicians physicians and within arsenal our of when will with make But date. going with and very we it’s that. allow using we to So and their face-to-face sure change process patients,

with doing. So the is team we’re what happy

Justin Kim

at at trials. ASN some Great. With Great. on and And CXG. of posters presented the also a clinical maybe taking look just you’re

company about population the the post how transplant. thinks wondering IC-MPGN Just CXG and

And kind of the of opportunity could potentially that be so you antibody-mediated disease for maybe to alluded fact pegcetacoplan. an sort

just enriched of wondering program the this population a and motivations X there. having as for So is well Phase an sort

Cedric Francois

Justin. Yes. Thank you,

So and diseases. in setting, we CX not the that in biology kidney. call present shared of study believe that, is but the IC-MPGN that X between of between in are the familiar not IC-MPGN deposits specifically the the the with is And those did two a presence Phase we CXG real difference for on antibodies the

the less hand own. development, of can is, typically important. Also have all the a XX they very inhibitor course, implies classical renal CXG final and products early D that always the have believe a very involved believe that patients that’s adolescents diagnosed been may are oral will post-transplant and pathway newly we patients, get or IC-MPGN efficacious years that where special anti-factor pathway best-in-class about until or such the to expected disease as these setting, hand including more to in where profile place for and in three be we’ve our we with that. be thought is often that in the alternative or there likelihood buckets, So on molecules And an be B

where renal getting second or will category then setting that that the oral will least, are transplantation we with the benefits The best-in-class come outweigh the in closer are not starting there, second come last kind the patients and a product but to need potentially convenience of having products. to of having end-stage of And efficacy. disease these for post-transplant course, advantage may maximum hemodialysis, always

be to our about lockstep think strategy. with that and So trials the designed world that’s how are we

Justin Kim

congrats Understood. the Thanks progress. and on

Cedric Francois

Thank you, Justin.

Operator

Tazeen Our of next Bank question comes Ahmad America. of from

open. is line Your

Tazeen Ahmad

one for me. question Hi,

is the the Tim, application that now. to your right have looking financing Is by be accepted So it there of the are increase the partnership, whether then chances of you it that order preferences one, type in of listed how getting that think into? some preferred you. a a company And the actually Thank agreement it you to to is combination important about royalty option of relates that looking your for? you deal and filed FDA types as debt or is

Tim Sullivan

Thank you, Tazeen.

that certain always that’s we the thoughtful for we be look, we how will and engagements taking into members going of FDA, have that feedback So timing access be capital, capital includes our of provides very to with as course, and and of about important. done, community consideration

we is So, of would always have. raise course, need in a that we we a that to and timing consideration perspective. that capital I thoughtful as a Overall, is done way from acknowledge say

more on any one out are. table. comment on the options laid thing what to of probably sort of all But want I do the preferred So, can’t have those we is options

will timing you the from course. due update in Thanks the perspective, we question. So, for

Operator

Colleen question from next Baird. Our comes Kusy with

open. Your is line

Colleen Kusy

line Great. U.S. since medical announced. were to you at results how for questions Have One data meetings Thanks question on DERBY presented the you an from number taking European KOLs had congrats top top initially quarter. and have might for the to and any and line position? they view OAKS us, the a opportunity know differently I speak our data the of

Cedric Francois

no. Thank that is And you, excellent That’s question to the simple an question. Colleen. answer

So, work, give you outreach U.S. charge of you the Europe, maybe a work quick regions and on Adam, well. from the we have been that And done in already in as lot update other can in have results a that. of

Adam Townsend

and Thanks, Yes. Cedric, thanks, Colleen.

So yes, have no we right. – surprise,

So, the U.S. the physicians Europe market treating treatment we presented the or Retina the flexibility or consistently, feedback patients. international through in Society. and supports have at we with with great real our over patients and the of including retina and surprise, ASRS and the consistent And with are that U.S. various some the the are similar in a every impressed get having market month dosing, ability and they no the is research for physicians forms very we also Europe research to treat been We on hearing the same that in are ability impressed other we need And interacting monthly hearing run world, U.S. Europe. there some as And are safety a of that data to data what all earlier. there.

So that hopefully, your question, answers Colleen.

Colleen Kusy

Thank the taking you helpful. question. for That’s Yes.

Cedric Francois

you. Thank

Operator

Cantor. comes Alethia Young Our next from with question

open. is line Your

Alethia Young

data and that taking for guess I Hey early guys. think are – in plan questions present maybe know my on before like on One, or Thanks some treating the maybe naïve via you think before with their I question experience the and switches geographic congrats that evolve the public naïve. kind see about filing about before they you our any my switches, second having matter levels physicians thinking of two for launch do data the of is data a just how attribute my it’s more the would a Thanks. to about the might of on a and first go you that when the you patients in time maybe That’s press of or to talk me. but release? just of of other question. majority might progress. you either And we plan higher hemoglobin help especially

Cedric Francois

send Adam’s way. will much, so you Thank I first question the Alethia.

Adam Townsend

Yes. Alethia. Thanks,

starts I the patient across seeing as described. actually the of EMPAVELI are we paradigm have So, of mix all

have falling and in who are basically. transfusions. and usage patients, the also of – the seeing usage the we the are But So, X,XXX moment also getting for hemoglobins higher requirement that we signs within PNH usage of treated symptoms at through hemoglobin lower levels patients CX majority

getting naïve in We great. which are also is starts population, the EMPAVELI

keep have to EMPAVELI unmet small that in to those Physicians a patient population. they supported broaden by do hardest discussions. within and is think ASH a a consistently have I with it’s out data the to it’s helped need, think we their understand thing base then the prescriber treat start to out small I highest there the the population. telling get us abstracts wider try data more us One people they patients, those want hear label to label. the and And superiority

close typically start we through will broaden that we have next think have So, see to these will able their patients, I physicians read or the example. that got first third year launches, be as we for we year as second base and drug enter to of patients

go back as So, year. stages hand Cedric the of And the for next we as see I we part question. that you will the expect second to happen to launch into your

Cedric Francois

Alethia. you, Thank Yes.

So, that’s an easy answer.

and Society in and we September, make of of So, have believe effort, concentrating will a presentations on we the to data that kind to ASRS going again at kind the as deliberate Retina made lives kind recap a AAO in you know, the is and very of patients. a be these complete of difference

Alethia Young

Great. Thank you.

Cedric Francois

Thank you.

Operator

Yigal question next Our from comes with Nochomovitz Citi.

is line open. Your

Unidentified Analyst

Hi, this is for taking Yigal. our Carly questions. Thanks on for

have We GA. two on

on lean that like based First, regulatory that you can wondering every primary the on regimen, the of much. and ophthalmology will regimen, one and is current works FDA approval there just monthly the what one anything so we were other focus the previous like plan the one? pursue misses Thanks precedent on had Is the Phase to both in X looks you month this when the just situations follow-up faced. on And then or a endpoint. dosing

Cedric Francois

Thank you so much.

the have it’s presence. be before more the from X.XX FDA kind powerful for DERBY perspective, the profile. needs the yes, way that out we So, we and is p-value well, way regulatory was The we I as I about explicit there But will that have to trials. think mean, are point OAKS important are here at the I talked I think look of present in had fact is example, it, the a OAKS, p-value FDA’s like statistically, the in p-value to with regulatory going had with this trial two which and border on safety is of the to of contextualized outlined what an extraordinary than, on we trial X.XX the the precedence, just we which look like combination it are statistically way right.

as the every monthly, great a of three you analysis, patients in quite these kind that’s foveal we plan full be FDA it Then indicated of the all consistent well, to We really baseline when which you ranges drug that where stake that for and again going question, it a get And fellow again, groups, says FILLY, picture; then to have other are the exactly to as treatment And we between effect with also this the effect the indicating is again, size imbalances where you is that that as package patients. analysis DERBY to XX%, have with where in the relates that GA the sit. believe put and the does earlier OAKS, monthly, correct from the ground. clearly every to data clearly XX% as extra and trials we what other thirdly, a data. then and for in with submit works, key month, all month

discussion the and something come will process. that presented of then So, all will be will end at that label the of be that

Unidentified Analyst

much. Thanks very Okay.

Cedric Francois

you. Thank

Operator

with question comes UBS. Our from next Merle Ellie

Your line is open.

Ellie Merle

my Thank you, questions. taking guys. Thanks for

DERBY. fact what some ongoing, to submitted can kind some maybe I curious that in ex-U.S. work before I just curious going? alluded two PNH, of the analysis there is but physicians we kind the average of has more the that sign-up of REM first been trying sense if or or have sites any could sites the site from prescribing Thanks. more us start expect have of And more, couple prescribed with get I a on the and but forms can curious ex-U.S. like guess site. kind that I on then kind see maybe you the info scripts at of And just And forms? us, commercial AAF. new kind more of when number if more guess Just last how if the kind you per a initially geographic start then how patients, breakdown of on the versus patients get BCVA submitted give to start been we want just just know of terms any one a it on the might in lot and or to expect some. of atrophy, happened that of just color of to of know proportion goes data

Cedric Francois

Thank first part? the to you, you Ellie. Adam, take want do

Adam Townsend

Ellie. Absolutely. Thanks,

we thrilled yes, are we seeing are geographical with for we physicians are across forms. getting demand the up the signed XXX not over into So seeing details, that U.S. too over start XXX but a And nice many spread REMs

with as do than need have We that start mix it’s and are submitted one across geographically unmet patients forms. multiple centers sites more very a healthy U.S. So, have showing up the

year’s follow-on see the demand will as before, getting that to the this means I would you and get and then broaden year’s launch. I think physicians So, we widespread, said next want to launch late that, but benefits that of we prescription start thrilled seems and try into into the to start switching of be that as and physicians stages EMPAVELI I am are and expect, patients, to and the patients that as

for seeing the U.S. So, very to second And happy I around back you with will hand how the part. start are the forms pop-up we Cedric

Cedric Francois

that Alright. really is question parts were well. will the as two assessed at it acuity, functional XX first that endpoints relates the visual one Adam. And there one I the will there to best be as you, of data, the lead BCVA months. to Thank your so corrected

will that’s for. that something to have wait So, we

the safety third what two etcetera, of the the the discussed anchor clear with that the coherent fellow that, other and analysis we with fact course. studies. at that’s the from each closer that analysis as in it profile, into will eye again, And the And works that three a then the real trials, relates we we to the piece, that data as Ophthalmology, what something assessing investigation before. in on elaborate drug American ex-U.S., effect that drug brings became the us coherent, the clear is Academy focus the where the and but happened the that As still ongoing, analysis on to did, the various of ranges again, that sizes of between much quite puts

Ellie Merle

Great. Thanks much. so

Cedric Francois

Thanks Ellie.

Operator

comes from with Matthew question BMO. next Luchini Our

is line open. Your

Matthew Luchini

quarter. the Hi. Thanks and taking so much questions congrats for the on

in diversified quarter, that you bit, perhaps dose. of I talked since prescription have little shift a last wondering as So, first a any the the sort to was days just population has if perhaps PNH, of mindful it. through has in Thank there your to need If less first results. of the And on was little from about or longer past, taking an was that of PNH the get to there patients, average number process. patient this XX been are inclusion as quarter to of bit curious, relates just they we inventory no the is secondarily, mention I then approval be anything And perspective you. there from it

Cedric Francois

Thank you, Matt. Adam?

Adam Townsend

Yes. Thank you, Matt.

So, there. just will perspective, I inventory on start the

low inventory. pharmacy a of So, holds specialty very our basically, level

of moving part part the patients. was the it’s as progress forward. how us of your long question to also first it taking an transition So, issue for not is But

on not weeks. at three to And have are that why I time is moment, that taking not there. period. period we So, And two about some worried some different it’s real weeks – average reasons time of there the is

their physician follow-up to with and So, appointments follow-up required. have not a are patients surprise,

happens is and and request. So, of patients we prescription. that would support make transition the commercial winning at and for their stock sometimes everybody opts help the to who we and services the delays to that also up sure EMPAVELI patient want schedule have Also, signed visits our we train that into when

is I moving seasonality which from are a coming form start be QX, of have Thanksgiving A right. in patients that, when impact When to actually a an a holidays, prescription up. think around QX few forward product, have want there into things to some activity that some think will looking, we happening. So, commercial to to transition there right,

We also have the December holidays.

some be seasonality within will So, there period. time that

them, and the moment, the But that’s transition weeks patient’s feedback think that we once the train are about. that on to is our patient we positive holding are for So, period to get very drug, we at through they three something hands two once the weeks support programs.

that to do are as it’s and would we hopefully, But the patients be launch. commercial drug. inventories. hope form time to to population, questions be your I – where it transition So, we in able the answers on Matt, we will to start shrink as broader I expect from that

Matthew Luchini

you. Thank Yes.

Cedric Francois

you, Thank Matt.

Operator

Jefferies. Our next from comes [indiscernible] question with

Unidentified Analyst

you Chris. of is This study do Thank lessons because on that intermediate entire and practices AMD differently to apply GA the [indiscernible] best study? team. would you Hi from directly experience, experience. What would very for learned much. your you potentially Anything

Cedric Francois

Thank you so much.

cRORA There when outside that. goes is at retina, to is a studied that AMD to probably very alive a the run. how the look is this something like at measuring in – we are of not are iRORA that be way what is very from well. we a geographic be going year have zone atrophy, outside, what and learned see is similar zone not we earlier of and There in It’s looks That AMD. studies, take reasons. retina bit studies What is little the to intermediate GA sudden will we dead a still to of of to all call which That conversion. dead right. intermediate interesting It’s we these presented you last question the time. that FDA the and figuring the pure something So, but how need out we

Unidentified Analyst

very much. you Thank

Cedric Francois

you. Thank

Operator

comes Joseph Company. Our Needham Stringer & next from with question

Your line is open.

Joseph Stringer

taking for questions. and thanks on my everyone. the Hi quarter Congrats

ALS, Where quick study, that score Thank it whether CAFS threshold or on are the get a landscape your you. around of meaningful and from a other readout would to a on I sort biomarkers? the competitive or the pipeline want Just on, one enrollment. clinically thoughts thoughts be the genomic on given your

Cedric Francois

Joe. much, so you Thank

answer. So, I Federico question am to to going over to that hand

Federico Grossi

Sorry.

do question was threshold what the expect So, or…? we

Cedric Francois

– considering from terms studies do you clinical the endpoint? what in expect would setting, What competitive of the the landscape

Federico Grossi

Yes.

looking unit difference versus mortality have combined around per XX% we a the answer the and of we are mortality placebo. to your for. and one are at function have to we month efficacy. that of And Does what So, studies expecting change a on question? have That’s powered

Joseph Stringer

much. very you Thank Yes.

Cedric Francois

Joe. you, Thank

Operator

Securities. next Our Wedbush from with Laura Chico comes question

Your open. line is

Laura Chico

much got have I two taking question. very for Thanks guys. afternoon on my GA. Good

best fitting use acuity see into I think is to you probably of I visual is to if visual in endpoint contextualize could you for BCVA do actually trying earlier, process the guess overall clearly, Cedric, first, not pegcetacoplan? acuity commented to am kind I understand GA So patients. you gauge the review how But what

Cedric Francois

Laura. Yes. Thank you,

the BCVA in was as So FDA with analysis. relevant specifically not efficacy the indicated

went BCVA in why of Wiley was a that reflection it’s not of FDA end all through macula. same the happens in where poor Chambers a So, ago is and what a that the for And periphery only and process the is really what know, the retina the whole is that GA, with thing. agree a as we couple dying as NIH I bad said, years in you can that a you on goes a fovea for reason the measure of think

Laura Chico

appreciate than you much. perhaps one. more precedent for would also a for drug in to any second there analysis GA then, you perhaps Okay. in a then for Cedric, in which guess, financial where question or a little And subset with submission get probably there study the partners out And of on detail. helpful. then recent I GA more the That’s the one one further first scenarios push the it. back Tim approvals the In and kind financing and in ‘XX, ophthalmology one question just of are Tim. related is to GA, half very I space Thanks on bit approval engage

Cedric Francois

You’re welcome.

be trials precedence, very of OAKS, DERBY division features statistical think what mean this every high month. FDA one But And I significance an I interesting visiting. on monthly the we and I and are at with the precedence on pragmatic. So, other for think two them would AMD FILLY, three of of have the that

they positive. the is will assessments they think will out clinically and they The the find where third FDA lies. look And whether again, mentioned to is drug as will whether at one effect the directionally be making biologically is will it look earlier, active, at look totality the data The relevant. they of at going size and safety, to as

first to kind GA that these want of Tim, steps I you And don’t it’s And much the will half our to go have through. are the in very submission, for know, and to this we those next of the so therapy to plan that. if a then on the become we patients. something submit believe first breakthrough add So, year.

Tim Sullivan

going that I Yes. operationally don’t think I we would say just are around financing. anything to change

the from are perspective with plow filing. we our is that view Our GA to going ahead

in very have the a high confidence We of probability degree of approval.

will financing the still to So, cater that.

Laura Chico

very Thanks much.

Cedric Francois

Laura. you, Thank

Operator

to further no would are the questions. any like over Cedric to for remarks. There I back Francois closing turn call

Cedric Francois

much. on closing, you ahead. in in all Thank Third Call. us thank on you keeping forward joining to so the look And our We Conference you for months Quarter our progress updated

later are We around and today tomorrow.

rest today, feel If Thank for you you free have reach to week. additional of again out the any have and questions, wonderful and to joining Meredith. us a

Operator

program. you This does conclude for participating. Thank the

may disconnect. You now

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