Apellis Pharmaceuticals (APLS) 9 Aug 222022 Q2 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
Umer Raffat	Evercore
Anupam Rama	JPMorgan
Tazeen Ahmad	Bank of America
Lyla Youssef	Cowen
Steven Seedhouse	Raymond James
Colleen Kusy	RW Baird
Derek Archila	Wells Fargo
Justin Kim	Oppenheimer
Yigal Nochomovitz	Citi
Ellie Merle	UBS
Annabel Samimy	Stifel
Douglas Tsao	H.C. Wainwright

Call transcript

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Operator

Ladies and gentlemen, thank you for standing by and welcome to the Apellis Pharmaceuticals Second Quarter 2022 Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. [Operator Instructions]. I'd now like to turn the call over to your host Meredith Kaya.

begin. may You

Meredith Kaya

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

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

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

Cedric Francois

us of at pegcetacoplan priority is that disease to to transformational joining PCO we meaningfully A PDUFA FILLY reinforced or patients great reviews advisory stated these is is have you from to living meeting data a few the disease was priorities. millions our cause making GA, in treatment their are the includes atrophy, ago, and all Apellis that thrilled for the Collectively, us we across and blindness efforts start patients we today. a year announce each progress slow potential become an of plan for it for relentless XXXX. GA. our and all of weeks getting for DERBY application. to from I The I'll XX, NDA am key the well for is designation as and data longer, our as with outcome a some FDA ever that the The leading out hold a of worldwide. FDA OAKS first of intravitreal November the XX in not date team of XXXX committee and geographic the planning and so by this the best with even with Apellis available pivotal for proud XX-month case discuss Thank This data the to to studies accepted the could also progression months study. were hoped point. NDA

We continue this to efforts preparations commercial currently positioned patients to our bring are in no pegcetacoplan with patients we submit well are immediately potential to by therapy our that In are on to we upon the the up bring application agency approved work intravitreal approval. to In our year. track also who end this of treatments. parallel, we have ramping EMEA so to to the Europe,

which data in We to this will and plan a in September. include few a the in report minutes. XX our OAKS Cedric, submission, will we DERBY more month speak from bit to

establishing treatment on we remain Turning a patients with PNH. first for further focused line to PNH, living as EMPAVELI

in are partner We outside for $XX.X translating making to patients also million the key progress in is our continued Globally, Sobi to revenues strength seeing across of net second indicators quarter. a the bringing PNH of US. with US EMPAVELI number

diseases. advance to as therapy have continue driven stage Sobi, a we later with Beyond underway. EMPAVELI now programs transformative rare four PNH, clinical compliments Together for we

SiRNA pipeline to of continuing program plan stage APL also are early and XXXX, the with year. INDs our next for We progress half our first in

efforts therapy our to gene AAV-based our adeno-associated viruses novel as progress great in enhancing We partnership Beam a to inhibition advance made approach complement with have and with products. or

neurodegenerative potential also use with APL to no of in XXXX to advance IND the pre-clinical the for We are our end submit longer but diseases, expect work by continuing XXXX.

areas. this potential to further compliment that, on to Adam. we in our providing this head multiple the a us We momentum a it cash a XXXX. strong our approximately million therapeutic will environment This over uniquely forward ended in we launch first in leadering with side, quarter into position second across On year. puts as as with we the later $XXX the position cement quarter of runway into challenging And building finance market as I look turn

Adam Townsend

Thank you, Cedric.

now CX Turning to patients require the levels. and transfusions EMPAVELI in who falling our year are on our third to Cedric In Slide patients US the second X, launch one commercial as year, the we hemoglobin remains focused strong. the need unmet highest address inhibitors we efforts in who with of are of on mentioned, their first commercial the the

XX bringing greater switches We approximately are since than In the physicians REN XXX. to across total from to indicators. CX continuing generated Over start XXX. launch are total of continuing see positive were patient since of number approximately come forms additional to the bringing were XX Over certified the key launch Ultomiris. second leading to inhibitor forms XX% quarter, trends start the

Patient we payers. launch. to strong of continue rates the at to leading move the and have certification as we We high see year which strong key established second REN amongst access XX XX%, top indicator growth, be remain compliance into formulary a continues

are patients in strategy, our the broader to expanding phase further into Now first EMPAVELI focus as community positioning PNH we next treatment for with is year, heading the which second the PNH. line by all commercial of our

efficiently so treating patients. on We the that prescribers are targeting are call those refining PNH majority our of physician who we

advancing this key our to evolve centers Hillman our prescribers with to Dr. especially with partnerships PNH leaders, of patients that have outreach and we thought phase that also next We will drive additional now onboarded. new are access fully launch, ensure is to EMPAVELI. In

our X naive a and for If approved with February, in will XXXX. safety efficacy the X accepted strengthen data. date Phase PFDUFA treatment this by Additionally, and of the FDA, allow promotion and our us Pegasus to S&DA awareness our the XX-week Phase raise results EMPAVELI, for more long-term including was data of print patients,

commercial launch to worldwide, devastating people high GA million disease excited even may including in and five GA approval I Slide to the this uncover on of US for the in expect need. our impacts people be we first approved critical our turning attention one to patients. area this Now closer million our more anticipated in X, therapy and even progress unmet are

we Apellis, at quickly ever pegcetacoplan, decision potential by as excited intravitreal priority possible as designation. are the bring to the and Here grant review first treatment a FDA's to patients by to the compelled

are We PFDUFA eagerly our that team is awaiting our November for are ensuring positioned date on launch. focused well and we the

as with force and We US our US positions are in is capabilities positions leadership globally key ramping commercial well much key as of place, field up the imminently. already starting additional the

eyecare of awareness, on focus to Our is specifically and the retinal the groups. approval broader state disease community within among prior some

US. help to also pegcetacoplan injecting bring retinal to strong the the ophthalmologists X,XXX reimbursement several in payer and At managing we specialists Retina majority insights advancing top physicians advice to prepare us currently. as the have to launch, made market. opinion our engagement leaders a we to the Council patients vast secure post-approval. of access and focus are global and provide on initial of be These are Advisory will up And We established GA

referred on right forward closer treatment time we pathways, patients as We will are to over being date. for We launch the activating that our to ensure providing granular to expand look our physician efforts details additional more PFDUFA referral and GA to preparation consideration. execution to

the turn to now over Fede me Let call

Federico Grossi

studies to approach. complement Thank of potential continue underscore our the you, CX significant Adam. The and DERBY data OAKS from our

safety the shared favorable treatment growth analysis meetings, other and lesion pegcetacoplan reinforcing key in data clinically with profile. in GA demonstrated a monthly induced we Following additional month a have with both March, at every XX-month intravitreal retina reported reduction studies that meaningful

As to compared in highlighted on Slide in May, X in pegcetacoplan patients we observed patients a continue demonstrate robust effect as effect at in showing what lesions with we an XX-month data extrafoveal presented to lesions with the foveal ARVO and treatment with the results. improved

results had and GA GA the at at eyes data consistent or at June. The lesion from strong patients bring just cells In that the first we the share this also Macular the defined we top potentially distances to Society showed a to September, months from therapy with untreated versus opportunity have given what GA X.XX. months. also dosing for at And of growth possible with ever many excited growth shared below the months. at reinforce photoreceptor by save the XX shown FILLY with GA the disease. population. to to Pegcetacoplan, Collectively, of line XX center. all course were and nominal of OAKS last spectrum, eyes. This the DERBY, treated in demonstrated ASRS, both important rate lesion lesion consistent analysis progression disease of regimens this P potential sustained across slow all is from month the analysis We slope all values the mean as fellow DERBY throughout results heterogeneity an with from critically OAKS GA previously the in XX in growth subgroups increasing presence reductions patient reductions showing Pegcetacoplan plan data as we across included in of We're which to

forward XX the drug. month could continue that mean into six As We size improvement longer months are large you interval; resulted the in six the XX evaluating treatment, data with patients XX. that over lesion it response the This at this showing to whether months, to over confirmed, If look compound treatment between presented very we six, efficacy pool XX. could recall, better each to reduction time. to on pegcetacoplan may and increased to XX to zero XX

of secondary Additionally, rate periods the loss. loss meaning of rate function eventually endpoints the will is reduce GA functional that over correlated progression with the of of XX growth time, assessed visual at months. should long reducing lesion GA be vision

of detect between occur Sham which separation As of as will time the are being due limitations we expect period difference time. the pegcetacoplan literature have not to we assessed. functional relatively the months We periods XX anticipate to short to of over mentioned endpoints at and do they before, longer over suggest, a and and the

XX-month our this in MAA. will We include data

and We're preserving from and Xstudy. us reductions productive show repertoire the lesion understand have that the between on EMA, had ability date by based are The to this and with with EMA GA XXXX. growth. published of history shared visual discussions into end repertoires wants our submit relationship data track on to have studies the We the in to function to Phase confident natural in our

EMPAVELI with which to with platform deliver cell disease stem driven also glomera CX the Phase micro first immune The CXG. of our transplant-associated X This on on study Moving working X diseases. CT associated four and a drug advance examination for previously glomerulopathy complement CXG or rare the our cell in HS the learned that transplant treatment complex studies had in franchises, Phase pegcetacoplan patients. multiple granted Slide X, TMA nephritis study broad later thrombotic in In includes for HSCT-TMA pegcetacoplan estimation of IC-MPGN we're is stem pipeline actively in pegcetacoplan stage for to potential also May, for IC-MPGN. on been microangiopathy or or or FDA hemato thrombotic angiopathy orphan enrolling granted hematopoietic membranoproliferative

Our patients partner dosing the study top potential X CAD, the first line XXXX. study screening we agglutinin and results finally, report track ALS Sobi XXXX. the has registrational in the guided or to remain of patient our And to in now mid half Phase in in is X disease Phase second currently cold on from

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

Timothy Sullivan

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

had As cash short-erm $XXX XX, security. of in equivalents, XXXX June Apellis marketable million and cash,

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

Cedric Francois

Tim. Thank you,

we with We are clinic. programs We pipeline two opportunities to the ahead. continuing position company's share additionally our expect excited preclinical forward about as the a stage into By as and programs our year the rare leadering products multiple complement end, commercial to late on look robust momentum. heading the and encompassing will disease to advance many we progress global build

now open the Let questions. call for us Operator?

Operator

instructions] [Operator from question first from Kumar Our Goldman comes Madhu Sachs.

Your open. is line

Madhu Kumar

Hey everyone. taking Thanks questions. for our

I So two guess from us.

months think about lot that few a presentation the is ARVO investors is how one pegcetacoplan getting of the been first events mentioned question of some a we've the neuropathy recently So a to ago. from in were disclose

reduction the how and it visibility and pricing is How about reform for the in appreciated. from and GA? largely about inflation of thinking PEG the how is is on we think think are weekend? of kind points kind terms that at of clearly kind ION you are has pegcetacoplan happened impacts elderly and you a process of patients lot drug that ongoing thinking So how drug question as a So to market big getting guys passed like of the Medicare you do framework? X over part patients biased review investors towards the about like recently any opportunity on those Second that

Cedric Francois

you. Thank you Madhu, and so much, great to hear it's

neuropathy reported cases, the assays the so on between for tech were standpoint. XX-month ischemic all, of first So no and XX- ION

were as to that over month XX, and talk something XX this cases this occur happy But months to that the to see it in at had that very X recall, between X are we not to may that case it's concerned but going kind briefly again, were hand the again, X point something had that So subjected and about XX about track, explain not it's does happened. studies, why and we you pricing. this of and I'm between could comorbidities also further to Elan we fact. patients X to continue we it where

Adam Townsend

Madhu. Thanks for question, the

the on is pretty portfolio currently believe we're explain but let obviously, a thinking. we impact our quite closely, direct bit limited. little And Palace So me the this monitoring

to we let's start to the sufficient Medicare in unlikely right? think it's at don't be So the think can the be that EMPAVELI EMPAVELI, . with for price until would then, probably seeking on a result to spending And negotiation So XXXX CMS subject even earliest. drug. negotiate drug

right only of current XX% sales, our a As of the Medicare reminder, business. represents current

and spend, the or future inclusion indications Now extent eligibility the likelihood Medicare of the future. obviously, the for in with of we determined negotiation

later based of recent early we move drug the at intravitreal PEG, if earliest. bill until on be Now the to we negotiation the to other PEG in of matter interesting in we compares PEG our the Medicare products. XXXXs on more not can't subject What is would until the XXXXs and some data on expire obviously, the moment, XXXXs. Obviously, the that and is, when determine likelihood for to GA MGA have composition negotiation currently, language, spending early at how again, most patents the

impact negotiated until a an it really two potential, with years even price hits. So would only IP three have our to for

pallet So as our limited. currently the can as the I impact the see on I at said we we at believe moment, the think and is direct bill, portfolio beginning,

Operator

The next Evercore. Question from Umer with comes Raffat

Your line is open.

Umer Raffat

that any you of Phase not in may the understanding on you example, them, of there's if speak may? you then clarify, lot Sham, to presentation? been And trial think I could Maybe metritis, First, a their this, I know and X Phase experience. been the FILLY could ION for clarify for a a profile from in couple there's view been lot there's I I in could in. or the II cases listening III [ph] if on secondly, confusion uveitis case of that. folks have clouding interest of know in side And

lines that. date? very [ph] seen those your conclusion you if I'm from any referring And to and like specifically the along could you much. speak anything view to Thank If observations data there's to in

Cedric Francois

you Thank so much question. for Yes. that

for naturally in ION commonly appears had a we are that administration we cases more in that cases coin. IOI eye. in have to previous concerned or see IOI does and from low that we of that received something that of a we a hand about. we basis I we first sham on with background the already you you really very destructive quantitatively, patients go profile intravitreal of patients the it when case all, what that inflammation occurs in And the of of vitrites The on that and And had something patients and exit fellow first something intraocular different as also have treatment with line the elderly of Iowa mentioned, that treatment hand seen intravitreal on with that this intravitreal are kind in injection, with with what we expect the where to Dove for from all, well. control untreated that's, know the therapy occur be is lot be are, also that are from Also the the inflammation the FILLY, question. population, as can the types have qualitative have, cases of So of very in anti-digest. would we that

profile that safety continues So and look to to favorable XX-month the forward we assessment be believe very well. the reporting as

Umer Raffat

be you would now. want these you data cases right put out? any with when on to don't XX-month guys of comes the to vision Cedric, I to And loss the able speak spot

Cedric Francois

we Well, the that. what talk not to remains more these future, look, it's When in the will, including course, on definitely, will the considered meaningful. top the we'll on elaborate impact safety But about sure of line more that profile, unless exactly cases I'm exactly of do we are.

Operator

question Anupam next with Rama Our from comes JPMorgan.

line Your is open.

Anupam Rama

opening and question the progress, particularly on your some I the all back on coming Congrats on wanted comments. side. the to of on GA XX DERBY ask OAKS a of

sort couple to placebo to track note of of kind pegcetacoplan the Where win So scenario. preservation a relative have base Thanks in a will on as that of so last a letter relative week, we much? pegcetacoplan. and/or

Cedric Francois

that question. for much so Thank you

September, look we data forward So XX-month to mentioned. in as the

reason have are those end that not the points well, FDA the end that end and as believe endpoints long we relevant difficult are treatment. measuring for those takes very It functional on that to We is always a points the to very -- or on points, we On baseline do. time that know to is It's telone functional itself a said. manifest variable.

comes the save that you the will end run, that actually point. how lesion functional reduction impact photoreceptor the long it the down showing between So correlation to and in seeing and growth all estimating cells in

believe have on a we'll a line show not approval that, that. we expect the at so be FDA that we be statistical. it will working not between So in -- XX on do And months least yet, to process. difference no impact The

Operator

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

is line Your open.

Tazeen Ahmad

it would commercial uptake? us you hiring you are and dosing when something either once with can And realistically be on on having they or to month then a relates month you going still in once with update And give front agency? go day got as on the every flexibility an terms trained, dose other to on to every to where approval just As and other the November? for discussions your of be frequency, is if you in important ramping month X, that as secondly, that's commercial physicians be table think the far ready do would people is you

Cedric Francois

much, so Tazeen. you Thank

not reported we'll the profile profile is right? that about, the only it's XX. we look at very we in So efficacy XX happens at product what every months, you other when excited a month are see on table, that So

exciting the to injections the You benefits injections, other right? evaluate. we point maybe half to something the with on will that every with profile. continue treatment get something very lot XX% as that a number of get is of month competitive us, as every So of your much To to benefits, month the

competitively favorably well. that We have us elements several as position

months. every First again, to the one, ability dose of course, X

reasonable data will I approval Secondly, the to label. that should the FDA have the a included the to months get that, that fact up the we all expectation of into think be we all that XX with way reported

population, more We what hand extra over talking we also we which of I'm have, in as field course, are to both patient well to that look it included terms of about going about to patients Foil were a commercial forward credit. as of doing broad briefly to Adam for all

Adam Townsend

Adam. it's Tazeen,

thanks your question. for So

field-based go. months, ready are look we're of onboarding teams we to now over of process So in yes, couple all for started We our of last prior the to the of all and them.

answer marketing quick we of the to the over the the leadership rest your in the is onboard process and our people for people. which through to now we market our And to wanting absolutely, part couple And a everything time? roles November teams roles hired interest the be And to high-level that's a prior question ready as to the Europe immediately mean, had that affairs ready was, We're amount a I great news, that and date. super and exciting. of next are step going U.S., unmet are of from weeks had at perspective leadership obviously, a our this second access post of back well the date. more within we high world. we're company, going join any in high-level And with we ready training fully medical of all So and ramping perspective be huge We PDUFA within field-based getting is sales going do GA. to PDUFA need plan. commercial can that

they So need where team is the to be today. exactly

Operator

Next Lyla with question comes Cowen. Youssef from

open. is line Your

Lyla Youssef

expected year just request quick on for what quickly Appreciate then besides in clarify component an didn't kind of that And mind the of currently, possible AdCom essentially Is that as AdCom current dependent and on of Congrats a would have what change may maybe European last reflect XX-month they your the from feedback the their of given FDA the PDUFA? timing be understanding is the deadline your of can on the question. but the any the the quickly the you progress. GA, that by that? is outstanding filing end Maybe FDA? data else

Cedric Francois

Thank Lyla. much, you so

not you we of I asked to we I'm that? do, before the more little specific XX-month whether to sure which and what but plan end is to -- was had a a about believe submission the for So and question European the the indeed it data, you bit fail are year. including marveled that going

Lyla Youssef

if Yes, else was that or you're timing? feedback that that waiting change is if there could outstanding from help the anything FDA on

Cedric Francois

No.

the is the It's this XX-month linked it no AdCom. the FDA. And as including data. then not So just relates to to feedback from

So the before I up that understanding PDUFA believe from weeks to my date. X

an have can't You AdCom.

the the I time think would lines less have the various not far, organized comes there that goes come that longer course, time to favorable considering time any by, have we of one. and that, indication be received we a still so that

we that I think worth knowledge, also of it's the and relates gave as course. writing it in the to feedback our mentioning is that best regulatory communication of done was the to

Operator

Our James. with next Steven question Seedhouse comes from Raymond

Steven Seedhouse

availability Just standpoint, filing, from and you months OAKS that positive submission, studies of XX to well-controlled still are sort studies? of the plan studies data the that months needing fit FILLy OAKS? are just XX now? in do all the the two criteria all primary the the XX-month when specify Or a analysis X reviews statistical and or the in three in given FDA And GA analysis DERBY is

Cedric Francois

Thank Yes. you much, Steve. so

endpoint primary XX months. OAKS. the months So of was the In in course, XX to the consider up FDA does in treatment analysis at FILLY, and DERBY

was it months. there, at So XX

trying to out we give continue us a happening and point to confidence confidence year. time to will the to of adjudication that data also prepare understand p-values the on important of spending flipped, saw that therapy last the It what correctly the months, and without the we in You with what place. really very allowed it DERBY that time, at XX standpoint us at that whether OAKS would to see And lot XX-month us is take over continues to November to correction FILLY course, catch and up get would was where, the happens trial.

Steven Seedhouse

XXXX, the AMD? APL Okay. eligible or for just And Is that quickly, then indication wet for it patient product? is precisely just what

Cedric Francois

an So not we active combines are that product. about call going the in for is to more on with familiar are anti-VEGF with future the This molecular loyalty that anti-CX something single entity. this be an a those talking in that

valuable to will in of either context from we approach terms. the something treatments will to So the received the are very we believe remains development what regulatory early but this it has that or be going a perspective geographic for how become

Operator

Jefferies. Our next Howerton with question from comes Christopher

Unidentified Analyst

on expect [ph] differences have us for PGN EMPAVELI in can well help you Chris. This is post mechanistic will disease? perform between Maybe the IC EMPAVELI expansion and understand And of patients label as these of And Combie function What more you proportion as and of kind then cast the score recurrent opposed opportunity, ALS, patients? the CXG? patients in do understand post-transplant terms score? And indication to the will moderate as of ALS can help demonstrate terms of a us severe important kind in second the more maybe time survival what you to be for endpoint? rating

Cedric Francois

Combie. you, Thank

kind about matter, buckets. for So kind CXG on the the that of we CXG and population the indication, so IC-MPGN in think of three

progression means with, of your of half The the patients path towards XX a first into years, one typically disease is that disease. kidney course end-stage go of but newly The the kidneys failure. renal diagnosed of patients typically well-functioning adolescent still course, will with over

your a of teams, not lot you're that's, . when course, in So time. of

so primary syndrome. your it patients avoiding as ALS, there II patients, that becomes transplantation. to relates U.S. is survival. that and in Then point, but in sometimes have is something the internationally. from again than the registrational third of have And are that avoid, a health of and efficacy renal clinical regulators, have something is can let those and And combined function This those to where of crucially patients with of having course, or category then the nephrotic been therefore, bucket alone second assessment influence patients study we those The is that also endpoint of we the occurs And happening. we Phase an significant where are just more with where function, transplanted endpoint an discussed, to loss was not dialysis of are important the -- it's are trial. that recurrence that the believe

So to is file readout it jurisdictions. in hopefully a properly powers it various allow positive have the to should us

Unidentified Analyst

systemic the my quick any then follow-up. there Great. to of learn of apply intrathecal nothing to can programs you kind be or ALS administration there? your that That's And one question. may you neuro lessons just Is ability for able from final

Cedric Francois

Yes.

look, in I very with many So all was and know CX, ALS, with tested many PNH it EMPAVELI. patients Ultomiris control anti-CX before does of things of source when show CX clearly, our of there course, not was We think, approval in know that there's has that as failed that we the do. but also

of think as now probably that are meningococcal one. of infection, of a I at of on haven't CX and years should have a seen not three single to more. reasonable We we Whereas range we in EMPAVELI. been point if -- the patient north have well it's with it's far inhibitor, will about dosing this point more talking cases, in XXX expected And so a time, case be not

that that we're something about. So excited very is

is it's as on that that a active be and should we we're other think something the ALS in course, for building as a I will, very safety positive of indications have profile get result beneficial we more well. where patients testing

Operator

Kusy comes from Colleen RW with Next Baird. question

Colleen Hanley

into the do the feedback XX-month the data? Steve's the specially relates the fitting to question you've from received I as follow-up it is any you a filing, see XX-month on data, earlier As there XX-month guess, FDA? data How to

Cedric Francois

I think you, Collen, question. for thank that -- that

include to important strategic narrowly had the We was the it of to endpoint September So the took. course, primary right? out us for in very got that and missing steps we data, disappointment X we over So important XX-month therapy. in when read

The November. adjudication FILLY that in first get got we to that early one was

safety would the efficacy know, to up our meeting the and XX data The in to one is So primary hoping way the endpoint, for there a catch-up full their very up that in what submitted was we that months, XX was well that indeed books. decision to to FDA our And important was propose data mind. second in go right? of happened, would as as pre-NDA as BX XX-month be you all include and January analysis to that months interpretation with the

complete So FDA. think I picture for just very a the creates

picture I the complete FDA. decision do that. a for think to good very it was a It's

sure good question, I was your that. think that do answers not it to a decision Colleen. I'm

Colleen Hanley

no AdCom? avenues of any industry that outside interaction there FDA its you're are assume and review? of kind field formal we has no Or should feedback available a FDA that the Yes. if the of experts how to aware get from between the Great. AdCom, during And there's there's

Cedric Francois

Yes.

believe decades. opinion a have in a interaction ophthalmological is the three well-informed very now has with well-run And right? for that look, it's field. key that been I a a fact been and FDA, deep division the has a with So leaders stable for time, we there at has leadership think this for, division I know very division The long the that this there I very

we com, So the that and ad why was happen tightness this there content of in us. reason filing that very off-line on based also going it lots straightforward on. we I'm But that not was with agree believe submitted, no a there's for to the We know interactions the that they community. speculate sees that fact

Colleen Hanley

Got it. me. That one makes for sense. last And

high-level CMC any take submitted, inspections filing occur? the you Just you might manufacturing part on can the and comments when there the of

Cedric Francois

Yes.

manufacturing drug retrospect. of for substance is EMPAVELI it as So the is same the the

either for come But on of very we our inspections able the a manufacturing single As any, not. out we issues those have EMPAVELI don't feel manufacturing. be on XXX or we we know without you comfortable or side that GCP on costs. if should know, get we will We got will approval to

Operator

from question comes Archila Wells Fargo. with Derek next Our

line is Your open.

Derek Archila

two us. Just from

are data of AdCom has as XX-month The set finding PNH. like that the any the stay the guess or or -- data first, PRINCE how on what in FDA inclusion wondered question might XX, I you to just of the I you And to in I discussed the the if the efficacy are benefits the prompt one the of plan the XX-month then major review? part guess, with site either data, launch? potential what the that require from launch, tracking is I guess issue addressing

Cedric Francois

it's So PNH I Thank important profile to XX-month don't XX see to months. any well as I an think as was that safety note will at efficacy we to XX as you, Yes. it as the it we Derek. over the of months, relates between expect hand to the believe both question surprises and XX established extension right? data, the Adam. But

way looked is at think at it. the way that FDA we That's look the it. So I the

we that at PNH, emerge It's be unexpected will but in do, to required action that's to And what for something over will Adam. that not the should be would I then course, point or it hard should Of time. what we something know believe case. hand

Adam Townsend

Thanks, Derek. Yes.

on and from So PNH, sources where new new we've drive keep and also that cetera. strategy label, internally, we and obviously, about can pivoted we're right? on forms to inroads good physicians choice a entering and have REMS The treatment what year treatment-naive patients. start And we're safety, the We're we and we'll we on start continue XX-week have . of forms treatment state for first-line to patients. target but you just start solidifies in call little starting our July give launch. data can certifications, July to I'll naive. over the right, those with centers more insight, talking CX with PRINCE making really physicians -- X our our II, efficacy and And robust our more of long-term Phase conversations treatment-naive switches The of the et and really X/X demand, of now were

with start continue highest growth to patients patients. We use physicians the those see treatment-naive those to to broader unmet broaden and as need larger PNH their from than

our we see to phase enter of to the progress that and expect launch. next I as So continue accelerate

PRINCE the us how that's help XX-week the PEGASUS So data and data out.

Operator

from comes Justin Oppenheimer. question next with Kim Our

Your line is open.

Justin Kim

I combine how it that on study. us. But as acuity. or that and question of about can trend that maybe of may you duration visual that hint sort show should you with Just think OAKS. a see of treatment we that that not where should the potentially about wondering could DERBY and that sort from know Is we XX-month sort something of separation? GALE think how point separation maybe Just X data of be

Cedric Francois

that very question, Justin. Yes. Thank much you for

are So red. incredibly with and we GALE happy excited that is something

with not for It's competitively. that, and time. us something patients familiar . how then we lesion-size to also important So profile function OAKS those that the that's very to from reductions it's and a themselves over enroll monitor safety X-year and to DERBY on continue how extension

we month other dosing. So every have again,

have We first our mover advantage.

foveal date We includes then and again, it patients. the types patients look the long to with as and well run. gel filing, ability have the XX-month And in as as happens all extrafoveal of to what

So will to it's important that come. something talk about in years very to we us

Operator

Our Nochomovitz with second Citi. comes from Yigal

Yigal Nochomovitz

growth. wanted you and EMA the between I said think, visual preserving relation Federico, see the function lesion to that

little could that show make bit you're separation to going the So a specifically expand . sufficient what just since the to update on data you to on points? end this functional the said XX-month point leverage you won't to be

Federico Grossi

Thank you the question. for

refer So being to mean we months the not when not that on significant, may be show we to something -- sufficient the trends that XX data.

it's central going acuity, that relationship to the we to necessarily sorry, from which get data functional look function is measure. natural find the visual from between to may -- at So we're endpoints. relation study hard between does not endpoints and atomical what the all the come the -- in history to And

be data a investigation see to get. to So in what more we the correlations study we're doing going the of little

Yigal Nochomovitz

Adam, could, PNH of far in us, just, so just you the market U.S? percent then believe you And tell what if could you the captured you've Okay.

Adam Townsend

Yes.

So thanks, Yigal.

So have or we we XXX and CX-treated XXX there stockholes. patients, XXX are X,XXX obviously, staff approximately over to think

certified don't we of guidance and physicians the we stock CX those hint within lines. we So revenue -- really on that should PNH. how XXX the progress as making come are go. of over patients in. We forms that anything give But to patients naive converting are and you good a continue or eat think making progress give We to good with starting on obviously in

Operator

Merle Ellie Our UBS. with next question comes from

Ellie Merle

you the and how seeing, you and the And on elaborate resolved? the guess, the one on patients cases specifically, of call, some managed had. Can first co-morbidities that I guess, how was patients if the the just it presented were we're I on in ILN. mentioned also questions then first they Just I think that

in that If of second and question, just terms truck thinking why in you about in those the sort use was anti-VEGF. sort management of of could this expedition, FI-VEGF you maybe can potential sort that? think the those the of I of that use this this expect And of we term just used the such guess, of to as on just team? new the might not from XX-month or the of about how about cases? GALE kind of the maybe elaborate cases And be about duration leads cases about the if onset real-world of of how then just you're thinking onset data thinking And sort new in there's anti-VEGF long of thinking expeditions longer I'm duration data study any related

Cedric Francois

Thank Ellie. you,

So going talk on the cases, ION not we're about this on the is details something call. to

two it isn't, XX. to XX to I going and single were right? consideration. rightful not, think was, your the then worse We converse or first of I right? case. additional from This course, have be and was to and There out So think to a is important these we point didn't from becoming had six SAs that one XX in find over time,

and vaccinations, they pathology. to At for regular when as studies, the patients OAKS with have long covenants find that And on What on exudations, it active get treated two-year again. PRN to the real-world compare step. out time the the we then Now stop that have relates end it mean? these we take does nuanced and patients the anti-VEGF People of the how does anti-VEGF new control. to receiving the sham between for and period, leak DERBY we

an we more year next going interesting of So which will have information be to information piece as well. on that's

the to been pegcetacoplan not an independent of that find out can that tell important So to GALE what and how means, be in I patients has is the going anti-VEGF there combined administration But on anti-VEGF. studies, are again issue. the you

Operator

with Our Stifel. next Annabel from Samimy question comes

Annabel Samimy

getting possibly you disease range the cart before core. I'm the severities. But of in realize a I So studied GA

as you it's do market clearly benefit? So data be and desperate that market? now earlier, this the most in the that approaching enter were simply the better, . how but market to how site envision this maybe the clear are the initially going getting targeting you patients release are So you the from

Cedric Francois

hard I time hearing -- one second. had you a

Sorry, yes.

than me Sorry, Annabel a was I for. But hearing just hard Tim you. to had clarify time this hearing better

month one of seems down helped again, with parts think, of set in the data to injections, slowing geographic the very is other of every probably you of terms atrophy. benefit I the give that of our to So progression exciting close XX%

in eye with wants So is One monthly as attractive. possible the maybe product other the the eye. and going stand you're you're the treat that possible that as lost But very there's two in think eye. being then is but in one business are first particularly want nicely diagnosed to many fellow patient probably dosing. patient, one, alluded much to profiles, a with patients that you aggressively a as who to fits that as vision Those the has But profile. out product to newly as maybe I save your

a profile is that XX-month evaluate efficacy linearly, over the of to investment is time. that again, extra you're getting there point, every about we You over And seem to an going months X in have the improve affected. we at still time months saw spoke of which small, the The that the better, with lesion every segment, patient became that time. And but there and with that something that in we growth the reduction will continue want foveation. period essentially to the fovea dosing. not you're to make drug most going not X It's to treat benefit long

approval, to course. five especially early you requirement very is lesions. a we're for for going two, something Now exciting but when see those XX But for think about that's treatment years, is or not it of that

and continues, think investment every XX. forward So we're I from X trend that important. making with XX that's long-term the lesions first But a other happens fovea newly dosing, going segments look something very to. months if between we exciting month to early diagnosed and can out believe, what that then is very we find on, Extra

Annabel Samimy

I for months, is X to that they expectations continued able And it XX pays separation, separation data an But will motivators have no segment Or somewhat you've be in every to idea just to time of of, and term? a we But sustainability? are guess continue continue might looking that if we've over of floats months. at if see follow-up? So Okay. XX as is for I talked do we shown something this lot could ask treat looking improvement do a long months. you maybe they're there downplayed think want physicians the the further you've far the really Is what at separation? as

Cedric Francois

No, Yes. Thanks, Annabel.

continue you of course, little stay sufficient, geographic time you get or explore will, XX% in we this to is look, and so very even that the an cells, effect research a a linear for if of that of exciting the saving, atrophy that bit increased think, if retinal you below reduction if I can way a then that that that full even in receptor the we is we community the say adoption drug. over something requirement believe right, we we that believe should around based the growth would, it's that for not, one? did with So be on

Operator

Our with Needham. Joseph next comes question from Stringer

Unidentified Analyst

is on for Stringer. [ph] This Ben Joe

one. Just a quick

about mix. payer bit a the mentioned little of kind You EMPAVELI

that heard do XX% you I overall I as but you to trend that mix, payer just continue around expect think around the Medicare, would relatively stable XX-XX? change? still Or characterize or

Cedric Francois

I'm that to Adam. Thank hand going to you. over

Adam Townsend

expect as we continue. before, obviously is I payer Value that EMPAVELI is Medicare of XX% said majority the yes, -- so Yes commercial. to population. a And

Operator

H.C. comes next with Douglas from Tsao question Wainwright. Our

Douglas Tsao

the progress. on Congrats

especially as how about just be maybe appropriate switch to dosing. clinicians monthly dosing? you made of when do to about with every guidance patient other the from think curious, I'm you month dosing providing the follow-up, early, comment a a patients other every trying start about to to month lot time might Just a

Cedric Francois

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

in benefit motivator a can. from response -- at the extract if is point, you you for is there much is injections from patient doing you that other and So business dose right? the planned especially those a we But there , look, your think, which that I risk lost is like monthly get has but what So a as other have to not the as now right? are that twice encroachments, if are every from means full-field vision to strong already line, or who you losing efficacy central response,

with And to will mean, in as XX mover the that up it's have to something again, that dosing, as we very months file, every lesions for advantage, well, month we is as something we this and other have reiterate, evaluate So in to extrafoveal data month over well that included we kind included patients competitively every the profile. other is us first of have important I continue time. are we have then right? foveal again,

we we believe So have make populations foveal, whether it believe it's over that it's saving whether can disease, the early, you the is long big something a of for extrafoveal in all where covers run, terms difference this and that receptor late, cells.

Douglas Tsao

just with curious dosing? And buck month get you I'm just in as every other follow-up, KOLs, bang conversations a the do how your appreciate from they much for

Cedric Francois

close dosing, of something think we've it Yes, we not? that point, probably and will work we the with and what a lot -- better the like? the not XX% to injections. clearly work on you're on come. this longer to range the something Does profile I it years over has been message focused in or the we it shown that focus look it work of that safety with that works to months quite half frankly, in of treat, time hard That's a is the other think potentially it part because efficacy up of and all I around but number may every the well, profile that does on work, the that drug monthly that that continue of month you and will that we to think does

Operator

showing remarks. And for I'd over Cedric further I'm call at any like questions any back the this time. not to to turn

Cedric Francois

much. so thank want not opening, are remarks you all just My of so Thank to closing joining. you closing. I for

later progress We should you the and you today, joining for have additional. and have be we again for will us excited the a of In September, and any Thank are about around wonderful today tomorrow, have rest report really data, we made and us. ahead we the XX-month that will week. what's

Operator

Well, conclude ladies and presentation. gentlemen, today's this does

wonderful and day. now may have disconnect, You a

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