Apellis Pharmaceuticals (APLS) 5 May 222022 Q1 Earnings call transcript

Company Profile

Participants

Meredith Kaya	SVP, IR & Strategic Finance
Adam Townsend	Chief Commercial Officer
Cedric Francois	Co-Founder, President, CEO & Director
Timothy Sullivan	CFO & Treasurer
Federico Grossi	Chief Medical Officer
Madhu Kumar	Goldman Sachs Group
Jonathan Miller	Evercore ISI
Joseph Stringer	Needham & Company
Philip Nadeau	Cowen and Company
Eliana Merle	UBS
Timur Ivannikov	Raymond James & Associates
Derek Archila	Wells Fargo Securities
Yigal Nochomovitz	Citigroup
Laura Chico	Wedbush Securities
Colleen Kusy	Robert W. Baird & Co.

Call transcript

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Operator

Hello, and welcome to Apellis Pharmaceuticals First Quarter 2022 Earnings Conference Call. [Operator Instructions]. It is now my pleasure to introduce Senior Vice President of Investor Relations and Strategic Finance, Meredith Kaya.

Meredith Kaya

Good afternoon, and thank you for joining us to discuss Apellis's first quarter 2022 financial results. With me on the call are Co-Founder and Chief Executive Officer, Dr. Officer, Chief Commercial Officer, Chief Francois; Adam Medical Townsend; Dr. Cedric Tim and Chief Grossi; Sullivan. Financial Federico Officer,

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

I'll over Now, call the Cedric. turn to

Cedric Francois

Thank for to us continue in later the submission XXXX an that favorable prepare GA, completed of data with today. XX-month extraordinary across or quarter. OAKS made our We then we and this atrophy, safety meeting treatment Starting of and our we've execute patients January, we retinal longer-term you for profile. we as saved with intravitreal important increasing studies pre-NDA in over joining the all geographic DERBY a tissue as shared is pegcetacoplan March, key resulted priorities. time NDA start off with greater Phase an and determining at to for benefit amounts each Apellis III to from in pegcetacoplan progress

Additionally, were starting at in these month DERBY showed effects comparable data X. improving that with OAKS

with lesion at population that a further a we lesions, diverse ever treatment blindness patients millions reduced with week for in these growth leading analyses for reinforce worldwide. potential first cause in to foveal pegcetacoplan disease shared benefits ARVO, is that living patients relentless patients pegcetacoplan this of lesions that become of patients Just Collectively, additional GA XX-month the supporting the spectrum. showed disease data the with GA, across for data of the a extrafoveal pegcetacoplan's and

We Omicron with headwinds EMPAVELI with $XX sales to PNH. a in treatment as living the the PNH. to establishing further in initial despite strong focused is due The on U.S. remain million off EMPAVELI launch Turning variant. XXXX, first-line a approximately patients to in net for start

is patient baseline the that indicators to positive The patients aim launch PNH, to levels, have feedback, EMPAVELI. benefit of of recognition leading and a from thrilled Aspaveli physician as certain with with strong patient we Sobi their remains Our to compliance potential favorable all outside levels patients also high for payers. ensure regardless the hemoglobin known begin countries mix, Globally, positive our bring by to and the see EMPAVELI, are worldwide. U.S. to partner in

for the we top to with PNH, Phase expected therapy first Beyond diseases. complement-driven sclerosis, lateral as enrollment in mid-XXXX. our results in line transformative seeking in quarter, In are we II ALS, a rare completed or registrational advance EMPAVELI amyotrophic study potentially

patient a Sobi, additional needs opportunity the HSCT-TMA. microangiopathy Additionally, significantly clinical initiate per now in X address second Phase with Sobi late-stage these many in transplant-associated alone, quarter. to Combined, for could programs expanding patients XX,XXX of X as year dosed U.S. have the as in its Together in EMPAVELI. the II opportunities the cell remain or thrombotic stem underway programs and hematopoietic first track study we on

month. and Annual approach adeno-associated vitro in We to are with In enabling Spark a Gene at viruses also data of with Therapy as APL-X AAVs. Cell forward sharing later we advancing American novel the collaboration complement look of Therapeutics, inhibition to upcoming the Society or Meeting this

potential has further and hypothesis delivery. in that complement, the with anti-AAV additional in vitro data that specifically with important mitigate tolerability our CX warranted, help investigation the supports CX, targeting safety AAV responses, associated role plays an early, and to While and clinical these concerns

Beam great the next including XXXX and SiRNA X partnership progress expected our and over stage early to APL XXXX, with with INDs make continue our progress Finally, we are continuing months, Therapeutics. program, in APL XX our to pipeline

allows position On we of the into a otherwise This head we further as with leader commercial multiple potential launch strength Adam first financial as the now ended Adam? of submission challenging quarter forward on quarter conditions. We from a position over our a to call look and our the areas. complement to market XXXX. to side, billion And strong building in cash, $X finance therapeutic runway position providing the us cement close first into in turn momentum let for our update. me in across NDA to

Adam Townsend

Cedric. Thank you,

launch our quarter. EMPAVELI PNH, strong see since launch mentioned, for to Cedric the net continue resulting sales commercial in results we in $XX.X U.S. last for As in first May, million

details Engagement Pete recognized of this who internationally of in clinical and PNH hematologist, PNH the to into thought University like Hillmen Peter month. team. the joining the researcher in get from Apellis Hematology of is Leeds us later the moment leader I has involved to Head launch, development the be a Pete take I'd of as treatments, Before including effective to an EMPAVELI. field welcome Dr. been will the several

thrilled are contributions board PNH Pete to have his continued incredibly on grateful the We and for to community. coming

through Turning our first More of positive leading to approval key Since saw May quarter, end indicators. in first the quarter. than start XX XXX we in submitted, the have continued the launch. been received XXXX forms the with across momentum

patient coming of majority inhibitor see starts these ULTOMIRIS. the to EMPAVELI switches We vast coming from from with of switches, XX% CX continue over patient

approximately continued of the is REMS demand physicians XXX quarter. growth the first our of the with program, of at sign certified positive end Another

have EMPAVELI formulary. Additionally, XX payers added XX the to of top

year. approximately the initially all stated covering focused made prescriptions, we the the are PNH of payers, on U.S. As top XX we and within with before, we've thrilled XX% first progress

has also when the see to XXXX, their is testament high of better And payers to we believe which of patients. continuing patients much and benefits well largest a are patient placed EMPAVELI compliance disease lastly, feel treatment-naive all we exclusive, in rates controlled. the how One EMPAVELI is as U.S.

year. better connect in-person the been to quarter with to secure and coverage executing with this access in of forward us to we bring conferences key U.S., to and patients had This further EMPAVELI difficult a launch engage the efforts physicians to HCPs has of earlier the increased, in-person need payer wave educate who We and additional As progressed, treatment. during our look to in continue allowed saw events. the and we'll on more Omicron return physicians,

which to of patients our long-term in treatment-naive we've Phase to Additionally, the FDA, supplemental III PEGASUS raise the this PRINT and data. safety efficacy submitted XX-week results includes NDA, EMPAVELI approved more us III of addition the by If promotion will awareness data. our allow for strengthen and our Phase

GA GA. irreversible the damage, million people of for suffering more States. and the X of than leading our to vision GA and in all XXXX Turning loss will million in here efforts the globally. at cause causes a worldwide, is including us X Apellis of year patients United from for commercial pivotal people be impacting millions

feelings feel everyday that for insight compelled of reported their believe GA independence countries. from unmet Results revealed anxiety, such and recently that and X aspects the is atrophy as Approximately a possible. this that powerlessness said X and X adults frustration. they We of surveyed and to to are It Treatments many the behalf caregiver geographic out social read, needed the as X on is GA. to as patients by have shared reported people Apellis. of to life with referred We on X GAINS, possible said they because X worse pegcetacoplan loss independence impact unmet ever, need, quickly of believe than than disease life travel. across of the withdrawn committed nearly due disease from expected. positioned need, to and support, ability by they and the rely negatively their bring More are with working as we a Poll and to on in as XXX conducted their they the clear: of of needed affects drive people and over decline majority recently overwhelmingly in meet that quality than results visual More XX we living the to significant burden therapy as are emotional patients survey, GAINS of is now. Harris lives

a is the of as preparing Our early launch commercial year. the potential team for end as

in and within market We have key globally. medical onboarded sales of marketing leadership U.S. positions access positions leadership and affairs, several the additional the and

initially the of majority GA retina are those manage focused specialists on who patients. We

also in continue launch EU, initiatives, engagement In activities. on the remain I to on will half second of payer and developments. our commercial KOL strides our near-term the track Fede? XXXX. as make the education, call We Fede prepare to now We to providing forward focused launch. on review MAA disease we plans in state our for turn over more to we to clinical look detail submit our

Federico Grossi

of one NDA this priorities. you, Thank is Submitting the later Adam. our highest quarter

broad DERBY which from mentioned, the to we excited group continuous over benefits and share diverse a time. are data meaningful and of Cedric XX-month to and patients showed As clinically OAKS,

month's every nominal OAKS absolute values highlighted look the how in In that than As X and observed continue continue to at with time, to and the Sham favorable area understand a during between safety in study was XX demonstrate and larger top on other in DERBY benefit Pegcetacoplan payment DERBY GA months difference effects both below intervals the as XX. studies DERBY that X XX. to in line This reduce all OAKS. the lesion month treatment at and pegcetacoplan effect means to treatment [ph] at we there compared FILLY and data to and GA the lesion to profile an then shown found slide, comparable in evolve over X.XX Puan growth P with this XX-month a both monthly pegcetacoplan readouts. pegcetacoplan at effort OAKS both months with month were

the growth patients earlier importantly, effect the over showed XX our and important data. to of In this of P share lesions lesion by of Meeting, this and X including we showed the arms. reduced patient our lesion the in at to for our disease by collaborators had across values X.XX. a in reduced OAKS In the one data all continued demonstrate data, XX-month growth the in XX%, monthly combined data with time. reduction that potential the and respectively, lesion slow foveal spectrum, pegcetacoplan oral is Then, with potential improved additional presentations, We Nominal with both improvement progression showcasing extrafoveal and leadership treatment in other and data. growth most the GA every effects presentations, geneity month XX% and lesions. XX% pegcetacoplan which we given Annual better below in week XX-month extrafoveal pegcetacoplan combined foveal were population. critically ARVO GA Additionally, of an patients treatment presentations, reinforce DERBY This and the

what photoreceptor many all a throughout excited early as treat GA to to of comparison also of franchise, studies an X results Apmedical generally bring our sales broad with top an disease. working meeting in the possible also with at We're patients therapy platform be eye We're to as multiple diseases. and the and late-stage potentially we the line. at saw to which disease fellow to XX-month potential deliver course We present expect progression advance complement-driven to on opportunity with the includes additional in to excited EMPAVELI disease are save the consistent

Cedric nephritis or earlier to membranoproliferative CX and or immune HSCT-TMA what Phase quarter. our second glomerulopathy glomera III patient in first mentioned In the also studies, in to LS and study expect regarding we in the a IC-MPGN dose CXG addition complex

excited in on across III the Our CAD initiate rare a programs. share partner, track our to study to Sobi, continuous We're progress disease this or Phase colablutin in second quarter. remains indices

turn over financials. call for Tim? to of review a the Let Tim the me now

Timothy Sullivan

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

Sobi. received milestone $XXX.X million the in cash short-term XX, April excluding had marketable from and of securities, additional Apellis $XX As equivalents in March million cash, XXXX,

Our in offering from March. in our balance reflects proceeds cash $XXX.X net million

our of quarter fund XXXX, of to including current expect and global in the We launch cash our GA operations further the balance ongoing development into EMPAVELI call remain on the to upcoming as remarks. Cedric? pegcetacoplan first the of pipeline. our future I to now closing milestones. We execute turn continue Cedric Apellis's financial in our will over for we back confident launch,

Cedric Francois

Thank you, Tim.

commercial updating encompassing coming global clinic. have as months progress to a questions. have let and an we over our look the excellent this year with multiple programs the and the forward momentum late-stage year. us for We and the position our rest forward exciting disease building the to on complement We made past programs and further look cemented X leader to products in By of expect few pipeline robust year-end, open to now throughout pre-clinical into call rare And additional heading progress. you Operator? a

Operator

question Goldman with from And comes line Sachs. Madhu the our first Instructions]. [Operator Kumar of

Madhu Kumar

guess that? terms before the one relates And process European to Europe? start pegcetacoplan. I lesion about formally XX-month How of data, of the GA strategy to approval think our both need filing assessment and for terms in we see should first functional of the in in filing do they growth division of you kind for

Cedric Francois

to you. hear Mel. Great

very with meeting So are we Europe, interactions going those well. in are the rapporteurs and

expectation was. what there be functional as And is will anatomical see The to updated XX-month never want with be regulators continues you functional end we the measure in to death file I endpoints. data The package. should it the with want receptor that of the to relationship that something something again points, the mentioned, plan the that Our in second point year. cell that the be to half European is this not statistical, to out understand full

that continues of second included. be the again, be guidance. half year this our will And all So of to

Madhu Kumar

more size. maybe One question GA market Okay. on

can hearing is, have both one you the patients are looking what question eye? more blind answer what of Like, affect in have question? getting eyes? decision recently that kind how in kind does of patients at you out And treating you a vision GA So And functionally who are that been versus functional of there? fraction legally

Cedric Francois

Yes. Mel.

bilateral of blind and is target retina of of is of affected one many are that number So risk the That around XX% is a GA that catastrophic legally docs the in the fellow very of particularly number by a it's those there for patients, are course, at so eye. loss. because, who interest eye in high patients patients population most are vision

Operator

next And Miller of from line comes Jon our Evercore. the question with

Jonathan Miller

and into to I with get runway, how likelihood raise? guess 'XX since the seems let's given start that get that guess new BD on one can profitability? -- I the the but runway. shifted And what's to well into the cash recent launch, have priorities discussions you you

Cedric Francois

Jon. hand call I'm to over going Tim. to the

Timothy Sullivan

Jon. Sure.

quarter to could lot get that, there fluctuate So of with other, this recent the so And into capital of that terms our there XXXX. based we're does raise, us as that on that out in you said, assumptions of profitability time target first kind frame. a not one of runway profitability the guiding are of way or

to be However, that I makes about financing a sense our for potential going partnerships time approaches the we royalty us have the financing take balance now. at out figure comfortable other But gets And may continue to get that we to company. we include that there vehicle. like. don't news very a looks think or so cash if balance that right looking is strong can we're good would cash capital us what thoughtful that that to profitability, We're get what really

Jonathan Miller

sense. we Great. they pushes When then a likely were more the follow-on get That And about that on PDUFA? the just FDA, XX-month data data this maybe if discussing. amendment constitutes one major later we at year, think what's that so makes XX-month

Cedric Francois

Yes. Jon.

expect So right? XX. course, reason we small not the don't anything is XX discussed like XX-month to for month because on in and new the need amply learn between anticipation. this And based is something have, So be very to surprises, and we wait is very, data would that of we why

amendment. will the to be it's when communicate data, of course, So scheduled have it, but major we a not we

Operator

comes Company. line question And Cowen the and of Nadeau next with Philip our from

Philip Nadeau

couple regulatory. A on more

learn? FDA this do In year. panel terms you suggested would if XX, on what to would FDA And they of to an think on then we timing, so, is possible issues, the approval on? And noticed you second, the want Slide an hold hope

regs have the the of FDA prior X it's to Our any filing, X is after then even months for understanding review, the with and accept PDUFA. at months acceptance they the

today, you that were that of this approval end day could you seems the file So even the the to actually come before from XXXX if the you gives likely FDA What would confidence PDUFA be year? it heard have occurs. like some

Cedric Francois

Phil. Yes.

X start going So the great one. to I'm with questions, second

course, time of longer acceptance the filing in months of the and chemical months the that on entity, from a is is from rather time different But So depends in new entity. it X X no chemical because new time of a than pegcetacoplan products, approved the acceptance. would be line that a is already

of the at course, even it year quarter. -- the to that us were of and second end relates if the it put the this be will, of still, as to So at end

it relates under as one. there that the Now be assumption will the to panel, we are operating FDA

very to important very will one. is, have of We be ready the this first It in course, new potentially indication. approval

about could which the a to is of I when that, kind right? think one people during with centered The safety. MDA think be remind first panel cornerstones, runs in like want the I we way is that X the being submitted discussed what

The second endpoint FILLY meeting it and biological one the with analysis in fellow OAKS, X eye patients Does is premised that the on And and primary drug. is studies. the the bilateral on then GA. work? in of activity

what understand is, XX%. the The size that Then effect fourth cornerstone And at have to studies third of covaried clearly, is size? we Because as well the in what the people the have this And XX%. of year, one analysis if long-term the chapter the with treatment with will real from where around impact one years, an for into of course, was we clearly we not then including happens is cornerstone the data have, third of decades. ranging is foundational the between X drug year to effect sits disease that XX% want that what the of X? XX-month going principles

in of the elements be an only of for that patients Kind does kind context that, the interesting that there XX% cells? many time of of benefits believe at not the terms of should an the there subject other one than kind What do for in in really So mean? continued mean does are controversial over year one safety we is receptor one? outcome, submission. is what But

Philip Nadeau

significance that of So you're it -- panel into that sounds even of correctly? interpreting that like shown? Is you've of kind subject results or am clinical you I the a go think the would the data what's major saying of what

Cedric Francois

have Yes. you And discussed, predict. was that would that be be what probably the if I question a and panel we would kind think of

that, XX% we on done meaningful, general is believe a we it's that course, among problematic a Now so subject not have the that in research physicians for of also the is launch. preparation A for will of ton consensus us. be clinically

Philip Nadeau

. helpful. very That's

Operator

James. Our next Steve question Raymond Seedhouse comes from with

Timur Ivannikov

on Ivannikov Timur for is Seedhouse. Steve This

G&A. So a just question on

I docs. symptoms do not see benefit if think it's a drug helping? clear you've way educating retina to communicate over change whether really of the the a the but And patient will of is in symptoms about or a the drug the talked you worse, best is assuming pegcetacoplan the doesn't maybe importance what think are approved, be year really

Cedric Francois

Yes. Timur.

mean, most with thing I a a retina, retina slowdown would very I it's the a doctors I important or percentage right? look whatever. I of area very I right? think mean, tangible to saved it's mean, that's about to you not them right? talk resonates that is when at, say one, what

millimeters. square So or Sham actually And for retina responsible compared a year, square we how about the that millimeter X at example, where what a well, of the being disclosed months, central than at was X.XX millimeters is square with XX millimeter in whereas Well, one for approximately data OAKS, way, could say, X.XX And it is of XX-month the number to even less much preserved the you this retina? preserved. was vision. square think already all retina is

read So acuity. charting, visual need which determines what to we

a could So there's of, we but treatment retina of lot, X.X period very GA, time. short the picture say, a years. the lot with over of in course of a A

Timur Ivannikov

maybe then just quick a Got Okay. it. follow-up. And

you've In in that terms talked of progressor about fast DERBY.

the not have On you you across other on are And who progress patients are hand, do, at if who the do you all best what who have people patients responders? responders distribution of really is deep did the those Do studies? GA?

Cedric Francois

Yes.

over that it's rare a very It important no fast goes goes all. to how will about not exactly right? we those to distribution, But of patients patient due course, It publish. in from these is is So it talk That at time, that that drive are variables can will, but deep we details for time, progression progress we all. that occur, And in more. progress. at many How pans have what's very out, can fast rare.

be study of be a And balance to quite it's in so well GA can stratify to because not study frankly balanced. easy for out really properly the you

right, In active. example, we we So unfortunate, rapidly disproportionately that, got patients sometimes, you mentioned, DERBY, and were arm. the active's you patients for probably to lucky. as in can -- the of allocated the FILLY, sense progressive we monthly between Sham get terms were were of these Plus the distribution in lucky in

Operator

question Howerton And our Chris with Jefferies. comes the next line of from

Unidentified Analyst

on and to function easy in And And about to high takeaways measure extrafoveal will in of practical subgroup visual us. visual fidelity then performing kind like, your measure I way GA? something this broader from you surprised for function? the to DERBY how thinking of most any and to And the GA in Amsler function? like question, kind the than second guess, function. Would a finding? patients' from general of implement Were most Chris. pegcetacoplan grid be impact group? measure couple What's Combis better what's Team, digital a takeaways practical functional see What analysis? is just A that other month were as [ph] for that questions monthly every

Cedric Francois

Combis. you, Thank

So extrafoveal let the me question. start with

to believe the dose it's do an which right? it month, we of And over but they in other every of are But we they there. disclosed, way, this compared at the continues disadvantage by was in is inverse have what's true be a and the extrafoveal they're in I to in disclosed to So see in continued October, very patients, function. what the hereto, a so which that -- interesting, beginning But DERBY, also will that progress the compensates, also arm, compared there, at at have And have time, beautiful Society it progressing So every you data I the how -- that interesting disadvantage we monthly, how that's over gave that right? is not Sham rapidly in relates XX-month we in clearly manifestation other on to a right? we one and Retina as see, time, to even that see to earlier, monthly the it relationship XX months month. update Then March. mentioned

And example, the can to. straw, that chart, is straw of if across street, if you through you morning reading the functional [ph] takes chart laser poor the that some problems person retina, is mean, but looking be with So that microperimetry questionnaires the vision, is central really the frequencies big To test patient, incomplete in but right? is how intense example. the back geographic of helpful different It's limited. right? need in your the Reading again, course, are But acuity, measures a can run function difficult an we of to then vision. of too, only in another is interesting best the have for right? in varies very So and you that a can general, have one, that we one different that function Well, measuring examination, understanding so central really they're We of atrophy, see I for spots. in to within evolves, is, you there, visual your from that idea in widely which measure told an where speed one, retina read same is could through a you spots known. one. again also incomplete. the the you visual a read with you and is how a I gives speed the number a sign evening, what hours But your the looking the several then for several wanted

things that the can these every important, we have they autofluorescence provide time, is is contextual deal endpoints But with anatomical of of organization the FDA and and over why limitations, accepted and this the all the what So has that their which reason it regulatory is measures for how measure anatomical receptor die, with But quickly cells with losing patients yes, it's can directly to resolution, directly -- retinal So how single-cell are actually almost we endpoint. to quickly correlated right? is close vision. right, an context. cells,

Operator

our And Colleen of the Kusy line from comes question next Baird. with

Colleen Kusy

X this see think you of talk the And like months did XX? the XX-month So of analysis? view sense towards that can be or so at is later slope we the analysis. do slope the to in entirety, update, -- I some on more slope to heavily I guess, the you a about have it XX care XX? Or they XX weighted say, would could to

Cedric Francois

great Colleen. That is a question. really

what measured, right? over We the time. That do look and reduction that kind again, the going endpoint way primary primary the agreed back is is with size lesion at is we FDA, the our of endpoint way which we So analysis. to on

However, in measurement as same. exactly outcome analysis. a over you many things, for get the exactly to but we analysis same secondary the slope you slope do other loss analysis, of the time, among close gives And the

also which various be can be a it make critical in will important a that measured is point, and really ways. You slope very

You for from can year example, from, X X go over to XX months.

point. at are the measurements XX, an even go over the each and you it from You we did XX or months, the X can you could because from the slope go X gives of idea to important consistent what is way other. expect to to That to X how XX-month XX, time time among

a XX and right? it's is it tool it treatment, you my impact long-term looked as that from now, the I XX-month XX- piece-wise that with data years linear today, sense, if is on is of XX, to bad through it geographic to between the if take that ago showed long to like with analysis important this months what contextual is And how patients. example, treatment that March I data compounding, X was now carry X will XX understand good to disease effects on at slope Much So likelihood not what seem care have in is forward of I what beginning more atrophy or DERBY the to X in XX-month that right? And the we be for data. these And as the and study. consistent to I OAKS ago. XX stay what because know about very X,

Colleen Kusy

interesting. focus That's really general ophthalmologists be much focus be specialists? probably will launch, or on GA And seeing to more I patients? how much Great. GA on optometrists are how already And referring retina guess, advanced earlier will specialists potential GA stage patients that on on a educating

Cedric Francois

Colleen. going to over to I'm Yes. hand one that Adam.

Adam Townsend

Colleen.

a specialists drive So well, right, we'll quick around reminder, to as are patients to that X,XXX retina a target who optometrists treatment some as right? a unaware there's little specialist. So and retina

of do piece interesting XXX list about on X,XXX. an of information, [indiscernible]. intravitreal is XX% of target all focus the retina injections our So the for of But right, specialists about about

do really the really, that injecting. the physicians of concise of So group bonus

us to and So with. for it's start to ourselves commercial relationships introduce and to a build very target easy go

we across of medical commercial, leadership enough, on onboard resumes those XXX over affairs, started we position for sales leadership And had said interestingly we've high-level our and to -- marketing. positions started and positions. recruiting, sales our come the we in call, we As several

the RGA the see excitement interest industry our within on So potential around you can launch. the

answers that hopefully, question, your Colleen. So

Colleen Kusy

does. Yes, it

Operator

of line comes question from Wells Derek Our Archila Fargo. the with next

Derek Archila

more talk more competitors now since detailing can in several now? guess, been what starting in I the Are counter you this That to months? are the right doing some just market sec, how than guess X I trended then EMPAVELI and shifting I just question number since the questions. to one. So switches patient of first, in gears for would mean, be market question, you've mix for terms PNH versus you Maybe a launch? has to helpful. see CX EMPAVELI And that's second naive

Cedric Francois

over Derek. will This I too, one, to Adam. hand

Adam Townsend

Derek. Yes.

CX a So be the And not take. EMPAVELI benefit consistently, X,XXX patients U.S. patients to of treatment-naive CX a treatment starts. continue surprise our or we're treated coming And ULTOMIRIS and the of patients, majority people XXX there's from you inhibitor a over patients. that within CX that finding XX% tells reminder, give the switch again, as see CX the are a of And just switches treatment. that

We levels with Patients the fatigue. potentially such group this experienced from have are EMPAVELI. from and normal systems suffer that also will of benefits jaundice as seeing near patients still hemoglobin

the high are entering market that hemoglobin and EMPAVELI. And so of to now asking to the levels seeing switch and switching we're

in We in continue treatment see to also naive hemoglobin. increase significant the patients well and they're EMPAVELI as from come benefiting to

get we EMPAVELI, on of increase. to switch higher on CX build later continue expect the to into patients I I also patients hemoglobin that us launch CX as continue population. to to To the So year, and CX this expect drive treatment-naive to to

that EMPAVELI. market the of whole mix a I within we're think patients from to So switching benefiting are getting

second question, and the we're part competitors Now your seeing. what of

So for that for that's make one are we best willing as patients EMPAVELI I'll on diseases me, is and rare the into are patients and solutions say, we And on best we our executing laser-focused plan. offer oncologists launch to that also hematologists these to practice thing Derek, put sure older EMPAVELI.

that market PNH for is to happening care to that's everything when elevate about try we we of that the make competitors sure and and in our plan, So want patients. the true hear stay which we just we standard

surprisingly So so continue patients, and execute are but to we going we products best to more we we're on impact that the companies, believe have seen determined that not again, our we best plans. can have have from we various the and noise action

and So noise, listen to stay and what we ignore to we focused execute it to tend the to in market. the wanted true

Operator

And our Nochomovitz of line the with comes Group. from next question Citi Yigal

Yigal Nochomovitz

effects Cedric I a had Cedric, [indiscernible]. and team. extrafoveal on for question

a in strong the obviously, bit in both as data course, of really a fact know, on is very XX since nominees complicated data figures DERBY. the it shows in So at DERBY. situation, Though the extrafoveal, very, solid pool quite portfolio, in looked are more very Though, basis, for good but not months. a good you [indiscernible] OAKS and

the irreversible already to that is to confidence claims have of patients have that claim, save said, being with vision lesions. treating docs can point, label? to these get you patients So and what And will much opportunities your in central level do given start vision the more more yield the full that can how loss more critical extrafoveal retina extrafoveal they with

Cedric Francois

Yigal.

this important involvement. a it's think because speaking extrafoveal I very that's worth So and question, about good briefly

centrally and on therefore, lesions a dramatic you it. lesions know, as affected fovea fovial effect that, have and are sits have the So

central second. rate lesions extrafoveal to but per is vision opposed a As preferred the where lower

with reasons fully protected [indiscernible] progression. we until Newly with initially lesions very for is, get diagnosed there of very, extrafoveal the understand, is because the patients often end foveal this diagnosed will that don't

a advanced is rely fovial particularly can advance fair involved, patients to should on patients, them heavily assumption it to think even that is however, compared fovia. when the ones. of becomes extrafoveal fovial Those available as you more very patients, still whatever So retina of earlier outside

to when So want stop If get want because are really you closing consider that even are may don't quick. aggressive or you to those being the point, ones most to want these using you the patients to time patients, more -- they anything, that treating.

fact, patients, lot way point, they base you're And careful that big. course, done is the once primary a So seems follow fascinating with in you In to to extrafoveal. the once did doing a being the study we're defect. not as where based to which smaller DERBY, with on retinas continue again, as in endpoint start of but of time, on into be your treatment, going to Over that, have can capacity, they, extrafoveal patient diving a to so -- some. have they foveal books, the the was that populations well to most show

So to foveal as patients be geographic as atrophy, be on but filing well continued, will with extrafoveal. our all

Yigal Nochomovitz

GA. then And regimen dosing on pegcetacoplan the Great. in for Okay. just

doesn't as every and there other to month. response appear a dose you monthly be between clear So know,

monthly and every DERBY. and extrafoveal other month the example, for As dose OAKS responses are inverted between

profile I'm that safety any So Or and believe with approve CMD? onset that an the agency only other you argument cleaner schedules every do there assertion the credence disagree merit on monthly approval? given efficacy both wondering, just month, to new is to incrementally and comparable should

Cedric Francois

Yes.

other month dosing -- look, we right. So very we're schedule about excited every

lesions with at the think disease, to of as beginning want patients I preserve especially for possible. the we extrafoveal much retina as

of the early XX% XX% as as get you of run. want possible you If start doing every you getting end by much injections up the if month, . that to to other long over and is desirable benefit monthly

So There's is important XX-month think piece filing. the a we have. that very data our of also we that

both as XX XX, well of time. not So improving kind as earlier, see on just profile And on we well have on the we on as months, but efficacy. of course, over safety the package full these effect that we then, data the discussed foveal as patients

both possible physicians. So as approved. I the think necessary much as that terms that We believe and dosing profile to in get to to the we data of believe, flexibility like regimen, we label, safety the have limiting not would the as do doses the provide warrants

is month in full had monthly patients -- question right? other I inversion in and in the to don't that you that on fact the DERBY. versus as So progressing the previous rapidly we That importantly, did the benefit, related again, get Touching every multi-arm.

you has over if monthly other data, of without at the the slight the totality So benefit every a month look data.

Operator

UBS. question Eliana Merle comes of And our line with the from next

Eliana Merle

how patients of just of data I today? an of of of of the sort kind of you model run out all latest be of And kind undiagnosed we that might given treatment, landscape no be what of sort in guys kind you terms guess, understanding number in diagnose? any sort patients what the of sort available terms GA Just sort have doing. to or that that kind your might of do commercial of analysis so with you're of pre-commercialization as number of of Just standard not the sort that the are actively there maybe GA? incentive thinking about and

Cedric Francois

over Ellie. one that as I hand to will well. Adam

Adam Townsend

Ellie.

quick a refresher. So

million patients million X in So obviously, X the alone U.S. worldwide, GA

than -- Now in lot or got a number is to their physicians retinal of X level actually patients center to like something ophthalmologists that injecting to or physicians, make we've retina it a greater that. of million because retina our not discussions we've might with patients believe that that where

could experts that think to I they'll if higher you that. number So within be the than speak tell field, you the

and targeting the that patient they digital it and should also we are a that awareness make at We're the various known of and horizon and exploring ophthalmologists, to to optometrists So and intents could on certain there's for to purposes, through now specialists. ways see that education through our that million focusing disease really treatment -- in enacting all including go potential working ways, various or injecting retina $X potentially various drive patients specialists and on retina channels their physician. footprint, to allow

looked we've those of So at type a of lot activities.

make be We that we patients going injecting drive plans sure can think We physicians. do to it's that. to important have around to

So hopefully, your answers Ellie, that question.

Operator

Needham with comes the next Joey question of & line Your Stringer from Company.

Joseph Stringer

on One PNH.

So that last the you've added over X XX based on -- your quarters. start last the or forms

of you still on remains you rate, impacts see XQ? COVID-related rate compliance -- do forms in high. any reaching steady if sort a where quite curious Do this just it? remain in high? were of And compliance the leveling of state? you start of there sort on sort sort to Just this Or And see this out expect -- the

Cedric Francois

Joey. Adam?

Adam Townsend

Yes. Joey.

an lifting now improving, and we're incredibly the to bit think in growth little consistent thankfully, continue COVID-XX we I in for we which that as exit we're your It's like Joey, light point, first see a access we disease to So that is believe restrictions, to of seeing encouraging PNH. quarter, see the that demand. going sure. quarter-over-quarter ultra-rare Especially

start January, first it to and into and right. train allows get patients So March some fluctuation February when This frequently to forms, us EMPAVELI, back in for more I product offices an that comes our expect is do face-to-face physicians' approved right?

comes pleased we're seeing seasonality, the consistent learning falls. to when we're the we're that So I'm But impact. with demand staff learning it the

Another good is increase. metric think to REMS for about programs we certifications see continue everyone to

coming That for for that's So program. that's we our continues had to there. good another over everything sign XXX physicians grow. sign up And REMS

and from demand in the continue that expect everything I to we're come. to more points patients So hearing direction. physicians

Operator

Securities. And our comes Chico next of the Laura line question from Wedbush with

Laura Chico

wanted to is handle the what GA. or bit, capacity injections this and the time . could issuance line discussed curious patients just peak that, related what be you side I period commercial how uptake the IVT follow retinal treated at ramping that would prior the we're a of And as on for many understand be up system one kind the might J to then period, to a to trying code. specialists? of treat the up. I'm how impact And of on U.S. but I obtaining to kind ophthalmologists code? And more IVT of by just feasibly a but the little during in J think to to

Cedric Francois

it are to to Adam, the briefly There but very I'm over hand capacity questions. two going level. excellent on

point goes of the is to these retinal us, everything the where doctors things and injections are of capability great I frequent these kind used the have reimbursement inventory, anti-VEGF for of that towards typical less model capacity model, for of we of that are these rather the buy for Will docs hopefully physicians, that right? products. injections, bill is, point The a is of a course, slowly a is managing dosing. one into entry become retina with than possible. fact that infrastructure, great us from that start perfect entering for think fewer we're And about is to the products because really talking administering anti-VEGF anti-VEGF all habit phase

antibiotics. occurs, is otherwise comment be would I'm more So our and would introduction please. us what where product there when ways, that the you here Adam, of talk as hand in of well codes, to the many a fill should, J about that to it But a void frequent created it the on by as gets to help going kind if dose scenario less

Adam Townsend

did Cedric, to how our well. physicians on very can patients make Cedric consistently, But year they is that bed And that a a be as their great it that onboard the treated Yes, them with a wet knew patients. we analog. said that Cedric, it last or or retinal you to improve get AMD it Laura. to capacity, set help I sure technician research a said, know capacity thing they what mean, Yes. new as found us products. we some in they to and also new new would allow take told consistently one

So they capacity should have issue. plans of think to started be an already

approved, So don't getting I to them there will launch issue, be, are to and earlier think products in. expect -- an when on if potentially, I be identify bring people which capacity people is it going the was in the are phase patients then starting

the we do helps have phase have launch initial do it. help plans any But So headwinds research that there. we a robust how could that's of I us think in on see the window, we would when pretty piece that, and we good of

of your code. to your part tied also So J question around second

this we head. codes people And certainty they're a understand So understand that bit little with a type a all be for off and awarded, as probably that, these for of April, relatively are most reimbursement know, type obviously, refresher drugs one, that impact of July my quarterly basis. the January, providers we sensitive to October expect So the around drugs. top CMS, of a And of code. J on and are through going right? we like of physician-administered that would we'll then have miscellaneous permanent work J miscellaneous new built much J So we right? people, codes, of

going that X and product of X the as hopefully, J looking them. months we'll quickly help And early of can into is that -- on question, take expect that to We're the stage as ways capacity support, I the various obtain And based and they payment. in processing obviously the that looking a until so in help effective provider J of approval. we look as possible. to think also code they're be code our permanent all confidence to at to for account we're becomes a prescribing launch permanent I therefore, And take apply claims within,

all where that's J things on we So code. stand

Operator

back to I'll you. the turn any over closing for remarks. call now Thank CEO, Cedric Francois,

Cedric Francois

look updated We months in progress And closing, our on you you today. thank much. ahead. the for forward us very you joining keeping all Thank in to

around later tomorrow. are We today and

feel have reach have free, and of wonderful out to Meredith, you questions, the If course, additional to a of any week. rest

Operator

Ladies you call. now participating, and and you disconnect. today's concludes gentlemen, Thank for this may conference

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