Earnings call transcript Q1 2023

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Operator

Good morning, and welcome to Agios' First Quarter 2023 Conference Call. At this time, all participants are in a listen-only mode. There will be a question-and-answer session at the end. Please be advised that this call is being recorded at Agios' request.

I would now like to turn the call over to Agios' Chief Financial Officer, Cecilia Jones. ahead. go Please

Cecilia Jones

Good quarter operator. and to welcome Thank conference morning, call. first XXXX Agios you, everyone,

You slides Investors section can to of going access our for today's website, agios.com. by call the

Officer, I’m our our call, Medical Head Tsveta Development; and Research Officer Dr. Commercial Milanova, today's On our Chief Chief Executive Chief Sarah Brian joined Gheuens, and and of Goff; Officer.

forward-looking various any set and make uncertainties forward-looking like the statements recent some of and as a Before could our that on that in future other call result from filings we those or with of everyone this get we those events to including other the implied started, make remind SEC statements. will risks, differ materially any by SEC. factors, include I results most with and forth statements filings we Actual would expressed the may

the I will that, Brian. With turn over call to

Brian Goff

you pioneering is us. developing living Cecilia. and joining therapies for diseases. Agios Good with and activation for PK patients rare the thank is dedicated delivering in transformative everyone, morning, and Thanks, to leader

life transform with function, lower-risk generate of cell MDS. differentiated data areas, to and of compelling highlighting activators and long-term and this PK PK in deficiency, We continue thalassemia, action multiple to the across of consistent potential quality outcomes disease our mechanism disease patient sickle

I'd for trials. Phase and participation thank our patients, team, entire pleased these in these to Agios On enrollment I'm of studies Driven to by we encompass studies the of population, of later closed the enthusiasm to team, month. of which our together screening expect physicians behalf clinical in development excellence our report investigators their complete and the and in this the research operational have the thalassemia, that caregivers X continued thalassemia and like

portion the we to strategy. areas broader robust the of and in operationally PYRUKYND Phase advance this go/no-go As data Phase context, clinical track and this RISE of stage conviction the rapid X/X X Sarah studies the bolsters of middle to significant the need Phase of the are seamless continue the and on In describe study our clinical cell year. announce more decision these development these to unmet pipeline in sickle highlights enrollment in X our disease readout detail, in will in from our UP

Echoing in the the positive we PYRUKYND sites, experience AG-XXX on positive our to real PK therapy the continue world. PYRUKYND feedback of that individuals observed trial clinical having deficiency is and reception efficacy see living from in the is real impact to trial receive of and that in patients investigators for both with encouraged both translating world. on clinical the We're the to PYRUKYND on clinicians at persistency

feel PK we on PK patients also MDS. and just few believe a Tsveta performance lower-risk this will implications just but for impact of how And function the not sickle a activation thalassemia, on deficiency, minutes. our in in detailed long-term update has commercial potential positive and disease provide cell

billion of on first from balance enviable with $X the in an position we hear you'll As cash the quarter sheet. approximately Cecilia, ended XXXX

separate indications of and primary mutant areas across five to-date secondary both in strong patients Agios' trial products. see U.S. glioma and reminder, low-grade span of approval with FDA April recurrent of milestone high to vorasidenib our therapeutics divestiture underscore need, a XXXX, its to royalties As These of of discovery pleased business Servier's endpoint of and retain endpoints. that potential development upon Phase met track the IDH unmet to as disease for were or vorasidenib the on oncology part in which a residual with record key sales. rights Servier we potential We therefore in approved three net X data

expecting we're readout of PYRUKYND data X cell studies more of pediatric end disease, Phase of additional X Phase by studies of half Phase upcoming the in milestones the to of in enrolling in PK enrollment including of Xa addition completing enrollment the deficiency; year, PYRUKYND the the stabilizer sickle completing a thalassemia; In X ACTIVATE-kids filing for of number of the than ACTIVATE-kidsT lower-risk PKU. study IND the treatment of our patients in Phase and PYRUKYND AG-XXX the of MDS; the and PAH for in

in and XXXX. what PK readouts studies of thalassemia from Finally, be to the X readouts sickle disease from years, and Phase in of in anticipate looking in pediatric forward few expect PYRUKYND we deficiency we studies the next XXXX a Phase catalyst-rich X to PYRUKYND cell

Sarah? the turn now will Sarah. I call to over that, With

Sarah Gheuens

cell the Thanks, pleased quarter activators, were and for to thalassemia. Award Brian. industry-leading tremendous Rare including disease Citeline Development. Disease XXXX receive Drug across recognizes anemias, sickle deficiency pipeline PK in its our executing significant first progress Excellence In PK and award PYRUKYND's made we This the hemolytic and of the other impact approval year, team in our in of potential

anemia on a erythropoiesis our each ineffective thalassemia thalassemia where become beginning potential has all therapy update oral now improve me brief of across to Let provide hemolytic and first PYRUKYND programs, to the with the subtypes.

Phase comprises which trials, each placebo-controlled X randomized program is thalassemia enrolling beta alpha patients both of Specifically, two thalassemia. and with the

patients a reduction enrolling development complete closed and of of ENERGIZE-T the and with potential own ENERGIZE and our of ENERGIZE-T ENERGIZE and primary year. are report enrolling both this the top and operational patients investigators' are is clinical later transfusion excellence screening transfused this to regularly is data indication, have our expect in PYRUKYND month hemoglobin enthusiasm who line endpoint we enrollment Reflecting we in for not next who regularly in endpoint with response. primary transfused response,

Turning sickle disease. cell to

with address patients patient pain adults in that meet the sickle cell therapy of crises improvement product high sickle anemia, RISE to we provides are unmet differentiated in an advancing Phase goal improves disease. X/X oral operationally need reduces this cell durable study the feel is To with function. Our a and and profile, seamless target PYRUKYND UP how

data to UP fully enrolled of the the Phase driven from go/no-go the primary portion RISE This middle the including we Phase X endpoints go/no-go of The readout longer-term safety. extension data be in are data UP, of the in decision by University this response hemoglobin Phase NIH portion are investigator the data and RISE trial the the X and the X Phase a And announce track of decision to at the and will secondary endpoint year. studies informed additional and criteria the of on any protocol-defined from Utrecht. X study Phase of ongoing sponsored the X

the for this children aim study ACTIVATE-kids we disease. Finally, the living approved ACTIVATE-kidsT and of deficiency to pediatric in first to therapy with deliver continue X PK Phase PYRUKYND as advance we

We to each the studies by of of half the at enroll expect least the of in end year. patients these

our AG-XXX, that strengthen PK activator potential Turning activator novel to PK our has franchise and enable the to of the once-a-day development dosing.

other study this of we X As approximately Based complete progressing cases pathophysiology Phase year. healthy expect to a an Phase with our forward for generated in end and by to the this MDS on pipeline. adults, MDS look share of enrollment XX% and the data of study AG-XXX the the areas Xa portion reminder, underlying lower-risk in disease in accounts have we of

PK activators, we As advance PAH development phenylketonuria, advancement business of our we context, our beyond underlying molecule to stabilizer and program, small activation filing earlier In end progress phenylalanine of stage IND this cause address of aim we also an through program our to are portfolio our to hydroxylase, continue for directly by we the this year. and this the our of continue targeting development both PK pipeline. expand to the

look I'm forward Overall, the important tremendous made excited our by milestones a quarter to number of across the next over months. executing the this progress and several team portfolio

With the that, I call over to will now turn Tsveta.

Tsveta Milanova

you, Thank Sarah.

PK to each realize of awareness deficiency the to laser in the strategy build to persistency. anticipated the thalassemia, MDS. potential disease patient that maximize risk cell focused and so, opportunity a we adherence the But and capabilities the addresses aim Our in in lower launches needed organization reimbursement, phase disease fully current journey executing future commercial of is doing sickle launching and comprehensive on from

data with benefit observed PK clinical positive translate use risk persistent world. in PYRUKYND profile. Consistent continued treatment real feedback element the are foundational patients, received the trial compelling successful is in providers, the to we of a the deficiency launch into we from has we program pleased that A any with clear

likely Specifically, data indicate research recommend deficiency. PK our PYRUKYND are nearly with their patients market to to that adult providers XXX% our target healthcare of

levels, long-term For hemoglobin impact clinicians, key improvements and drivers of these reductions recommendations include and in frequency, comorbidities. disease complications in transfusion on

date, and identification six our of strong barrier While treatment disease on patient we PYRUKYND both from commercial positive feel reauthorizations access to initiation. have and overall, market on improving been function a we this patient including have remained launch of treatment the beyond months has remained not of and services. low discontinuations persistency focused reflects awareness and strength the Together, how patients capabilities, and believe impact remained

XXXX, represented of increase including PEF, in that patients a PK quarter which Patients a population. has a with last base demographic the XX% A This deficiency first of of diverse on form growing fourth a net range or full XX have quarter represent In patients of a continue quarter. enrollment to total XXXX. translated XXX the fourth net million launch, manifestation $X.X revenue. quarter now we over versus XXXX, broad generated and on XXX completed PYRUKYND of and prescription in of from physicians XX increase quarter stem the the consistent adult and XX% therapy, into provider therapy disease is fourth

patients capabilities activate that and and to help efficiency, patient the potential confident to potential adherence treat in identify the will we reimbursement, enhance are long-term our maximize appropriate for providers the broad in range meaningfully the launch foundation launches including populations. larger today, current of commercial for PYRUKYND, of likely am prescribers ability a diagnostic lay I maintain a strengthening prioritizing

thalassemia next the of an where in XX% U.S. do therapy have potential launches approved Our in not patients approximately

patients, and the the therapy to range potential hemolytic thalassemia including ineffective full across Importantly, has erythropoiesis beta-thalassemia. PYRUKYND the improve alpha of to anemia first and become both oral

currently in hand, particular the and deficiency. disease patient the cases is Beta-thalassemia the therefore and PK disease no constituting approved of on form the more focus common the launch current building options the capabilities in education. the will Leveraging Alpha-thalassemia two-thirds in thalassemia has a U.S. require of other on identification treatment approximately we’re

product route competitive and prevalence more adoption its overall we profile, greater market landscape, a on including emphasis safety require access PYRUKYND’s of efficacy, beta-thalassemia and and believe administration. will Given greater in

launches together, to strategy from require the address launch Taken potential these populations. our a these of each of to leverage aim each needs and capabilities commercial tailored will current of we

unique as range needs higher The GCC, to of Xx the these full of geographies our Cooperation and evolving the addressable outside forward and profound is commercial to of thalassemia is comprised market or options approximately areas Gulf to target Xx the prevalence a diseases look in such of treatment meaningful to where patients new U.S. We EUX XX,XXX these Council to XX,XXX need. U.S. than meet transformative the thalassemia our patient patients capabilities and deliver of unmet the countries,

With turn that, call I will to now Cecilia. over the

Cecilia Jones

Thanks, Sarah.

issued results More summarize. first will I Our be financial found XXXX will detail will XX-Q, later in press can in this our quarter today. which morning, which the file included release be we be

First PYRUKYND XXXX. QX increase $X.X revenue an to $X.X quarter million, was XXXX of net million compared

was first time. launches, in patient for for at rare XX% Consistent the providing Cost is we and to disease continues will this quarter the population, launch on PYRUKYND sales to insights the to XX% As data of to continue other not the million. range. ultra-rare be gather this be net trajectory with $X.X and gross therapy guidance

related the reimbursable expenses sheet. to Moving expenses first million that quarter XXXX. of the first primarily quarter, expenses. to to we first $XX.X to of $X.X were driven $X.X of driven quarter, a expenses Servier by the $X.X million business. balance provided of first million the decrease expenses Oncology sale decrease reduction the first transition a workforce the decrease compared of for This million for the of XXXX quarter was in in quarter were to $XX.X related million was and XXXX primarily of the the related SG&A R&D a by compared

income Sagard of given seized, royalty to reminder, the one-time royalties Oncology As statement which has on of on net business U.S. rights to was X% $XXX.X royalty, under TIBSOVO in of on income payment XXXX. October a a gain from our a for sale sale million sales recorded TIBSOVO of

net milestone Oncology retain on Servier, of divestiture sales. vorasidenib the $XXX our rights U.S. and of to part million to approval XX% business potential As we a upon FDA royalties potential of

We cash of and ended $X equivalents cash, approximately the quarter billion. with securities marketable

program cost including anticipated PAH on operating for To cash, mitapivat, planned cash cash the marketable plan, deploying together enable partnerships. expect we We proactively stabilizer of our funding strong interest will equivalents commercialize allocation focused outside execute position, our cash income our and maintain one managing our our and approach. and and us remain securities to through more product currently mitapivat with disciplined U.S. AG-XXX the development will revenue and or a base

now back over Brian I’ll his turn call to remarks. the for closing

Brian Goff

Thanks, Cecilia.

studies from rapid ongoing PK balance and the rare diseases. of real persistence industry-leading is unmet treatment team current of enrollment address our to activators launch of our PYRUKYND PK in thalassemia, of strong Given profound deficiency energized our and hematologic of focused pipeline world our in needs in our patients suffering on PYRUKYND the advancing the sheet,

meaningful year, opportunities be look and regulatory creation. ahead of responsible to we As to sheet of in value balance balance will milestones, clinical expect stewards our the the for to the strive evaluate we we and continue

our dedication hard employees participants all our diseases mission their programs. including all transforming clinical the in with Finally, partners, for our to patients and rare of I’d patients, of living and of the to physicians, development lives and like work caregivers our thank of

With that, we the will for call now questions. open

Operator

Thank you. with [Operator from RBC Renza Gregory Our comes first Capital question Markets. Instructions]

Your open. line is

Gregory Renza

question. also on team perhaps around about for to us know taking congrats progress say your much. factors some decision. you upcoming. the and the thank it information that some if for relates accounting new near-term, data? What about you well. of with to, as how cell And that’s it But is as of that the about that if just in are the my color more over the Thanks. perhaps scenarios in could the nested some potential And think remind when perhaps outcomes just you can of as there I of or as outcome Thanks may data into and factoring have team readout how comes sickle time no-go on you consistency the provide that the very just and think maybe so you the I little that’s Brian, you Brian Great. the been pleased mentioned, Brian, you’ve forthcoming? and and a there’s you

Brian Goff

just respect to cell Greg, the and the in noted Yes, off, year, in the thanks readout Sarah on the Sure. of team I’m especially and at now with that a progress getting thalassemia of lot data question. for that made the that for and as Good for fact again sickle well. midpoint screening I want she’s readout disease, the point call, but a that excellent not puts first as with of proud that’s her and equally cell domain a we to morning, sickle the us XXXX, to with commend important completed us a position in just

have, course, cell, that of we planning a sickle of for. number For scenarios we’re

seamless, that’s for planning designed and of why hoping course, are was the and be We to operationally success trial X/X. Phase

and site very X. and leverage in setup that the is great quickly behalf go obviously be have patient X need would Phase high with to So the same And there in we’re the the this in that we readout, Phase on scenario forward. news that a team unmet arena, their to so is and move eager ready Sarah success clinical

focused the with primary to the course, Phase in to we’re many data, as talked The about UP we’re endpoint, what Specifically be the for RISE at a times on look respect looking improvement. will holistic data. we take of that readout approach we’ve this going X hemoglobin

hemoglobin in looking hemoglobin us high will with a then for will dose that not level. that it’s secondary a be at course, disease a that have we we’re The cell a also se. We thinking plus element would it’s be so the a is about hemolysis pursue and safety dynamic, of week though success very like, the markers VOCs, designed endpoints, and for key could and on guide and trend that VOCs, we’ll way plus be sickle XX study, per of

a having and which short-term, persistency in the dynamic. for is to know so far disease a very the studied some chronically, our It’s success we ability that and by could be seeing so It is patient it’s way, on that time, function, why by we’re the that the in launch. sickle cell we’re stay just feel not the therapy quite for encouraged the also PKD important arena patient

add you and stop I’ll maybe with to respect scenarios. see, there So just Sarah, anything want to if the

Sarah Gheuens

think I No, it’s covered.

different I think and way the scenarios. we’re because it very excited the is flexibility for about study our allow designed us gives does

Brian Goff

come. And about period I’ll the excited to just say, we’re again, Greg,

We at have readout of sickle the disease the midpoint cell year.

have to readout be the where looking We thalassemia XXXX. at we’re in going

MDF then study pursuing we’re low we stabilizer where our end. looking pipeline for risk that at our have AG-XXX year have in proof-of-concept at and research in for PAH year Xa in IND intermediate Phase end, the earlier We we’re

the So it’s come, time pretty to again for a but thanks busy question.

Gregory Renza

Brian. Thanks,

Brian Goff

bet. You

Operator

Tess Our Romero next question from comes JPM. from

open. Your line is

Tess Romero

for morning, taking Good guys. Thanks much so the question.

number any do in So you instead a in cell of scenario of factors no-go words, to on of Thanks Can this into and a sickle just right? status move mitapivat So patients there in contribute factors decision, elect the sickle us out much. XXX could disease. so disease for of go Phase the you AG-XXX? mitapivat? of the remind that you’ve And forward results there which be the laid XXX cell other for will to cohort X

Brian Goff

Thanks a one. in have I’m lot, Tess. to Sarah going this weigh on

Sarah Gheuens

Tess. Hi,

is and really And development. totally at are – built longer has mitapivat safety X we ongoing the not huge has for different of presenting to XXX points which Phase will and the past be time XXX, regards XXX of have has a In phase built exposure disclosed Phase development. So the part is so second the question, we X much mitapivat and data. at profile, the the

other in is each just is this doable build that for profile. something we context in advanced its at XXX is up So to believe the to the point. safety the view substitute And product needs not one compound less our of own

Brian Goff

year we’re And one, this I’ll but pursuing two we’re reiterate leader as that Tess, see way, optionality low of readout, be different are end. again, PKR able gives an just readout stratify intermediate economics we data the in the two quite within preparing the that the for two not in even clinical in to, areas the advantageous with that that again, have therapeutic risk you pleased into and MDS eager I across if we to have a activators, activators the quite PKR then particularly the positive trials. is call that. when having one. patient population, But quite noted, it would started as near-term launch advantage to with to be that could at And us, that that all And Agios to to therapies is surround versus get lined-up lot

So question. thanks the for again

Tess Romero

Thank you.

Operator

Mark from Thank our from question coming you. next Oppenheimer. And Breidenbach is

Your is line open.

Mark Breidenbach

Thanks Good morning, questions. for guys. from taking ones Two me. quick the

might the respect NDA regulatory taking Servier first? which filing. have questions. you to for and nearly maybe such with for and And the clarity to trials, a second specific any enrollment. future these many you’ve is in have influence First, clinical just with respect seen trend geographies happen? that I’m enrolling ENERGIZE-T trials trends that that then filings, the any filing ex-U.S. to curious plans given your fast patients question a when filing Thanks completing on ENERGIZE if for plans so Do to are And going are vorasidenib is which potentially sites? territories

Brian Goff

Sure. Thanks a lot, Mark.

you start those take geographies? to we’ll in So and trial clinical with the ENERGIZE, ENERGIZE-T want sequence. off Sarah,

Sarah Gheuens

Sure.

clinical in trial presented past distribution we’ve our So globally. the

different thalassemia reflecting the beta And so present product thalassemia, how and a and program is transfused. genotypes deliver alpha reflecting context thalassemia is thalassemia in to and really distribution the developed of because for non-regularly the of also this is all where between regularly meaning is

that is these And also enroll way. designed so such designed the clinical development way, in to way program that trials it is but that a feasible

worked very far pleased how out. And has so with been that have we

today. so really globally, of enrollment regulatory trial and not to strategy is of do we distribution clinical our needed changes any of Our reflective the see thalassemia any

Brian Goff

eye this vorasidenib. your thoughtful about course about Having Commercial our thalassemia as add is geographically, will data, is market in in maximizing Chief think most sequencing Tsveta address so value patients we well, to also on. as really concentrated those to addressable the Milanova just your access point, as When so with of economics I’ll and about pricing advantageous, a board geographies about and to Tsveta possible, the before – question for the her for in launch an spoke towards sequencing uniquely transition we particularly on part, is to Cecilia respect question remarks, but we And the Mark. with be with of Officer successful

in population obviously to world of having board with Maybe comment concentrated is. rare And a in positive puts expert it a of And a us data, advantage. again, respect strength comes class disease but as this all position real globalization Cecilia vorasidenib. products Tsveta that on with is on how have we’ll

Cecilia Jones

Yes.

So retained to X milestone the hear an sorry, We royalty $XXX a It’s we for side, U.S. we’re we’ll – on need we update on XX% sales. $XX our provide patients. clarity results. From very it. have and FDA for on have and million timing million when approval yet, huge great don’t rights Phase net obviously the excited, unmet for

Brian Goff

progress where of across for adds last only PKR has had science all progress And a track but what the came we’ve could that in want that the to of of charts say mean as shop, out in glioma plus haven’t number the Agios, got Servier’s had progress fronts, to in into now employees proud that is years. listening Agios record this any in our of just a activation. XX know I And we’re are original patients then and and science so they obviously off leveraging the well. And lot great I and this we Agios

Mark Breidenbach

so much. Thanks

Brian Goff

bet. You

Operator

moment. One

Harrison have America. with a Greg from Bank of We question

open. is line Your

Greg Harrison

a would or drug and therapy treatment replacement it under the good question. taking How development? is approved landscape like you would for with fitting Hey, PKU Kuvan? enzyme see where different morning. gene the say your in Thanks from you PAH And stabilizer

Brian Goff

by Greg, on saying I start good give front. early will morning. first to too So too that off, much

keen are IND. let PAH stabilizer Sarah further. progress we comment going to our However, to to But I'm

Sarah Gheuens

And our be think need designing And to designed program is meet product as in a I are provide well, programs PKU. target is our for all specifically this really to one meaningful the – profile of that designed – patient like will we difference populations. patients living program really unmet we believe can with for

The current therapies their available have each limitations.

that a address for we so And the to this also amount need reduction shoot product to on with some what people of to the of be hoping a difference, can to get are create to on the basically, is of and phenylketonuria differentiation but there. also actually that And overall. further profile, better take product improvement, safety meaningful to

program. And as as progress with be the moving we we of it's to the any molecule, IND so, that come now, course, will develop and we as on then phase more – learning Phase to early and X,

Brian Goff

so area already, it's and we've and talked our pursue diseases, rare cells therapeutic is of And that's every about or and PKD we're focus. of we passionate another example about great PH, that innovation whether thread sickle why Yes, that's the for common disruption. MDS single thalassemia, because desperate

Greg Harrison

decision at the be could data? a delay? or announce sickle I or you in If What there helpful. same that to the time, go/no-go be a press will sneak be announcing That's it Would cell. then And one on could it. release form conference? one quick Got will the a you use

Sarah Gheuens

it we do will But two. always Agios so announce not think a depend on past But in course, of to programs want when our to we that are the meeting, of and act data. which a for we the we've fashion, all a planning when in I balancing medical how because, vein. what typical done at So something include similar can data between do expect present to will you will press release, in receive we PKD, those it's like regards

Brian Goff

way our goes data. mean, be to why clarity Yes. I the whichever decision, target as based behind that stakeholders our the give will on to enough our

Greg Harrison

Got it, thanks again.

Brian Goff

Thanks. bet, Greg. You

Operator

Thank One you. moment.

a have Divya from Rao We Cowen. from TD question

Your line is open.

Divya Rao

our Divya on taking for Marc. thanks guys, is Hi This question. for

there trend commentary benefit? Is do this showing response, this then support a – you the the view hemoglobin-plus hemoglobin-plus the PROs important? is to particularly that commercial the guess seen? specifically, on Or view Given are that any And be the are ultimately predictive it isn't I think needing the would PRO see VOC analysis of PROs VOC themselves or profile?

Sarah Gheuens

this the is right. Phase readout, So X

PROs, benefit, So of analysis product in profile. Phase of will with it we line and those that has really our parameters. sets. for of endpoints hemoglobin then – and looking doing at VOC safety, indeed, response those will and endpoints Phase be primary secondary all the we hemolytic looking PRO with come X, end X encapture are VOCs that the in But the be will data now What trends

using disease, taken across generated is We studies into will, account. of data cell sickle and to being course, the the are in be different looking mitapivat at of that the totality date that

Divya Rao

this then or, I seeing are QX? in trends way be I another stocking a And sneak I PKD, changes in helpful. with effect And guess, or are benefit? seen could the what if That's month response reauthorizations, any any being On one, second of evaluating was also. you the there considered payers guess, one would then

Brian Goff

have comment to dynamics. Divya. launch eager Thanks, stocking, I'm Tsveta to going then about is talk Cecilia on and always

So, Cecilia?

Cecilia Jones

Brian. Thanks,

So have limited only – distribution we model. have as a reminder, a we

pharmacy and specialty specialty one have one distributor. We

inventory levels on our So inventories limited. are are hand –

see, there small numbers. We're about you some talking might fluctuations So still quarter-on-quarter. be

Brian Goff

yes. meaningful, And Nothing Tsveta?

Tsveta Milanova

Certainly.

but well. of we treatment as utilize and we endpoint well. barrier. trial as payer from profile initiation, not Patients a front the also positive my with that the reception encouraged reauthorizations physicians But towards do reauthorizations in reauthorization. been the well we defining today, inclusion/exclusion were noted primary as Until the patients, see as So comes as remarks, the have in really criteria clinical the on the to PYRUKYND's I when it

six product that patient see. patients with the of months early the taken persistency the the profile Even is post we and both will though success we see of in totality reauthorizations into numbers, strong the the continue account, well. as and launch However, small reauthorization in of

Brian Goff

Yes, persistency. PKD really see now from those target PYRUKYND today's comes in And we then PYRUKYND, and are – are indicated to is through our the who market heard that that certainly research, certainly We as that that hope on in clinicians on list, inclusive on starting patients. is share we message Tsveta of we that had performing. enthusiasm. earlier, that actually anecdotally in have commented prescribed our who And that discussion, hope have which has

enthusiasm activating for so So not much focused. the emphasis the efficiency therapy. education, really is Tsveta's patients where diagnostic and launch around is especially access commercially really to product. That's or about really begin dynamic It's

Divya Rao

Thank helpful. That's you.

Brian Goff

welcome. You're

Operator

from And comes Salveen you. Sachs. next Thank our Goldman with Richter question

Your line is open.

Unidentified Analyst

Good Salveen. on This [indiscernible] is morning. for

would through you factors looking data. at the frame a there one on whether what trials? guess sickle update with Thank world real in what terms could looking – is dealbreakers no-go on you Could PKD you've the and But walked decision? at be launch. patient from specific you're And what I that going in cell. you. just lead clinical If the Just was to seeing consistent guess the the in provide observed on scenario. are rates us you're discontinuation to you're of an then that the I just that just

Brian Goff

Sure. again in we'll do And that sequence.

Sarah on can start with and discontinuations the then cell. Absolutely. Tsveta So PKD sickle

Tsveta Milanova

Absolutely.

trials persistency low have With the to there So that to discontinuations mind, translating as much overall. is in encouraged PYRUKYND, of setting. really real are world we which into remained mentioned, we have in the therapy clinical experience efficacy the very that observed and we in see

reauthorizations have been product And so for barrier, far, mentioned, and not adherence the remains as strong. a we

value importance that I with in And And into. of we encouragement expand future proposition really in adherence us also think, indications associated could be the important looking But this is reflective, the can persistency potentially to much very is differentiator other are PYRUKYND consistency, a it which PYRUKYND, translate knowing can diseases critical gives the in indications, of how other be. that and PKD.

Brian Goff

Super. Sarah?

Sarah Gheuens

function. continue and patients in how I trials, also persistency allowed that then excited And of And clinical adherency has the that to to on that about to effect, we Yes. seen which iron and data to clinical overload us to follow to us trial, the maintenance allowed present. we're point of feel think impact continue the study very continue has long-term extensions, in which patients we've our

it's data so board. across compelling the And that

context it's to mentioned, at in As that hemolytic the of us other Tsveta anemias. important we look because very the also

to turning clinical the disease. cell trial some hemolytic the trial would obvious clinical regards a anemia, for would sickle a fail. scenarios as there, to clinical go/no-go on trial as expected when the In be then what scenarios And other there – are no-go

signal response, in more our the in the and which will endpoint, as go/no-go defined profile that in And clinical wrong So any proactively scenarios. need all program, see that then scenarios if benefit-risk like development tips a we instance, then data goals clear or be have you things the not for are way, have, safety for we do hemoglobin other assessed. to primary

Unidentified Analyst

you. Thank

Brian Goff

Thank you.

Operator

to closing Thank the to no you. queue. back showing like And any I'm questions I'd Brian other remarks. for in call the turn Mr. Goff

Brian Goff

and interest call today's in for thanks, and everyone, in Agios. thanks Well, participating for continued lot, your of course, a

this team on behalf they're to of are patients you what significant value And incredibly the in for I morning, our and both for across confident us. to am making progress as potential great deliver doing patients shareholders. continue our who our So proud on count heard portfolio. of we I'm

look a speaking thanks lot So soon. to you and we with forward

Operator

participating. Thank This concludes today's you for conference call.

may disconnect. now You

Agios Pharmaceuticals (AGIO) 4 May 232023 Q1 Earnings call transcript