Earnings call transcript Q4 2022

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Operator

Good morning, and welcome to Agios Fourth Quarter and Year-End 2022 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 Jessi Rennekamp, Senior Director of Corporate Communications. ahead. go Please

Jessi Rennekamp

Good quarter Operator. and to welcome Thank year-end morning, XXXX fourth and conference Agios you, everyone, call.

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

our Executive with on me are our and Gheuens, Head Officer the Officer; Chief today of Development; Jones, Officer; With our prepared Research Chief Sarah Dr. Commercial Chief and call Brian remarks Goff, Medical Financial and Officer. our Chief Tsveta Cecilia Milanova,

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

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

Brian Goff

Good you morning, for everyone, joining us. and thank

is the living activation PK and progress underlying hematologic past team, our tremendous building treatment toward options, a for limited pathophysiology, over made of that share pioneering of need. Agios unmet activation therapies goal is Driven transformative delivering dedicated to on patients focused common developing the Agios in franchise rare year the and diseases a cross-functional and leader we by commitment diseases. with PK the profound

therapy Great PK execution PK as approved for the the and additional modifying with was potential activation cornerstone by for highlight forward PK in and of deficiency. to displayed living in our XXXX only the first-in-class like growing excellence PYRUKYND represents to I'd XXXX. U.S., and approved indications the the development two franchise activator particular, bring our in with research across Britain In EU, PYRUKYND adults our first disease and team

PK ambitious clinical consistent to made beginning year. ongoing approvals, at outlined studies, development these with the the not with the and we we quality also to activators compelling our highlighting five targets our of PK significant generate areas, and pivotal outcomes continue cell thalassemia, in pipeline, transform and deficiency, of our differentiated and disease clinical-stage of sickle data but across including we lower risk this met parallel across action patient long-term function, only advances of last mechanism multiple life, In broader Notably, potential MDS. disease,

published medical data the Association meetings across annual XXXX, Hematology the presented journals major clinical European of of meetings, Journal In our American The were including and portfolio including Lancet England tier at in the top Medicine. and were and Society medical Hematology, the of New

a PK had Following for the launch we the that PYRUKYND having in the the third be reception impact from and no quarter PYRUKYND of encouraged is patients, to treatment options. physicians, deficiency, of by payers, and full continue previously positive community

trajectory larger we've to As is be in and in this potential the opportunity realize said, expansion we of improve we steady meaningfully said potential platform believe building support launch That past, the will in will launch to slow and front a the capability continue provide patient of ambition that the our us. to populations. have and full

next disease of hematology and commercial medicines maximize the and in Milanova December focus sickle we operations. Her order global potential few to a be experience the commercializing as capabilities Tsveta and and strengthen diseases years. in on and in brings critical leadership to with PK commercial year, deficiency the market over launches is To launch appointed launching current potential particular thalassemia both our cell future prepare will in for last leader Commercial in Officer. continue end, as Chief access a expertise Tsveta of that we deep seasoned rare in and who

$X.X royalty balance quarter approximately on We of on the our sheet. one-time ended sale XXXX, we includes TIBSOVO of XXXX the This fourth Sagard. the the billion in rights payment in following cash net with U.S. an position received to of enviable sales

the as portfolio of beyond ongoing to the enable completion programs, our expand We through development, as our advancement well our to expect earlier-stage our us PK business position disciplined of pipeline. cash support and activation

on We anticipate significant these XXXX. progress each objectives of in

RISE in the of of of studies and UP we X data and and sickle expect cell PYRUKYND Phase middle the the disease enrollment thalassemia, readout go Phase X. the Specifically and Phase decision no to of of complete this go/no announce -- in ENERGIZE-T to year, PYRUKYND the the ENERGIZE X go with study

the we for Phase the Complete enroll our ACTIVATE-kidsT half of deficiency. in stabilizer of treatment risk the studies PK enrollment pediatric of of expect PAH of the PKU. and By the Xa file to Phase PK the AG-XXX PYRUKYND X ACTIVATE-kids and in than study IND the novel more activator our lower MDS in end year, patients for of

strengthen deficiency ongoing to PK through commercial in we to our continue our opportunities Throughout BD the year, to and the expand capabilities launch to continue evaluate pipeline. aim

potential with to PYRUKYND XXXX forward for rich a anticipate we Looking indications to in additional the two this catalyst XXXX, period timeframe.

lower the X the studies thalassemia, MDS. Specifically readout of in the of well as in the Xa XXXX, AG-XXX of PYRUKYND of study Phase Phase we're risk as in readouts expecting

in hematology cell approval potential disease in potential an and slate flow and pipeline development, approval catalyst, X XXXX, Agios, near-term that sickle And sickle aligned is I look productive pediatric we as portfolio core established in XXXX, business PK readouts expanded of in as of expecting In in advancement hemolytic expertise deficiency. of potential and XXXX our disease Phase as this to approvals positivity. cell work of with thalassemia deficiency. With and anemias, well PK forward fueled a disease of we're with year pediatric three spanning PYRUKYND in by of the toward we're PYRUKYND expecting the PYRUKYND our our the internal rare franchise vision an cash

Sarah. now to that, I'll turn over With the call

Sarah Gheuens

Brian. Thanks,

the areas to-date shared underlying with blood PK made compelling long-term and PYRUKYND generated red and advancing disease red consistent development across of organization quality for research activator, development data united our life, multiple correct activator highlights each in cell the significant and PK cell patient the metabolic our In to datasets largest areas. and by progress have for cell pathophysiology stress. the we of Representing XXXX, disease any PK deficiency, metabolism potential these function, sickle thalassemia, blood and PK in activation outcomes generated transform of disease programs

and December across clinical where updates and total pleased iron were to homeostasis hemoglobin data improved from data thalassemia, recent our XX most abstracts. long-term and erythropoiesis PYRUKYND the alpha stabilized or of weeks XX included external Phase improvements study X a hemolysis non-transfusion presented Agios in collaborators ASH These in our patients at beta over The with in present demonstrating thalassemia. were portfolio in dependent durable and or of

study patient we PK overload, from In in a updated of and which at our volunteers, transfusion improvements dosing. burden, safety extension novel of hemoglobin; data And sustained outcomes deficiency, PK healthy transfusion AG-XXX showed data once and long-term profile demonstrating profile day favorable presented X exhibited new status. we iron in the adults supportive pharmacologically regardless a activator PYRUKYND that with reported treated a presented doses of Phase PK active

hemolysis of red data clinical mechanism the action and Taken to cell correcting to-date metabolism differentiated is blood that we erythropoiesis. have suggests PYRUKYND in leading consistent together, and generated hemoglobin, improvements

on beginning thalassemia. provide that now let a the update context, PYRUKYND development program with brief With me

the program thalassemia which reminder, both PYRUKYND randomized are and in As X trials, a enrolling placebo-controlled with Phase beta each two thalassemia. of comprises patients of alpha

transfusion ENERGIZE-T potential enrollment to first a to X and endpoint enrolling therapy the Based is disease. Phase studies of in in year. of is for both enrolling these are we aim we of transfused thalassemia of in generated to the in response. foundational reduction complete who on of and half hemoglobin regularly this each response ineffective subtypes strongly with and endpoint and the the treatment forward with have from studies than improve data the erythropoiesis ENERGIZE middle now this enrolled, of readouts this a patients believe next look program of primary these year. not regularly to studies in More transfused to-date, PYRUKYND anemia who We been of therapy hemolytic become primary become all are have patients devastating we patients a in the

adults readout defined study and go is of turn data the safety, be This of X disease to now in decision we study expect announce with go/no and to Phase year. portion announce to of longer-term UP, trials go/no the the end, in enrolled. cell are this Phase aim sponsored Phase in novel improves both we PYRUKYND decision X The the by fully Phase middle pleased informed and Utrecht. go of X Phase the we data anemia the response ongoing protocol that primary RISE sickle therapy the deliver endpoints this now the will and any where of portion study criteria from VOCs. RISE and the well the as that and University this am additional are and seamless the from data investigator endpoints as oral secondary studies Phase To X of operationally at extension I a the UP of portion the I'll to crisis the or hemoglobin NIH Phase to X cell advancing vaso-occlusive of reduces X/X sickle X disease

cell with have resulting trials disease, that sickle these adults in reduces deficiency PYRUKYND we also generated observed to and hemolysis blood sponsored to improvements suggesting erythropoiesis. and sickling have similar investigator consistent in To-date, data thalassemia and compelling PK hemoglobin, what red cell in

and deficiency therapy of to this aim to patients end living Phase the of we of year. ACTIVATE-kidsT PK we to in with Finally, as disease. of first each at studies ACTIVATE-kids continue half in the these aim enroll least deliver PYRUKYND X for the pediatric children the the advance by studies We approved

PYRUKYND provides lower and opportunity continue PK our In of development strengthen novel program, PK franchise activator, a activator advance multiple development therapeutic risk which including further parallel MDS. paths we to AG-XXX, pursue with the to the the

As dose glycolytic the and we study suggested were pathway, sustained supportive and single presented dose-dependent data decreases at from ATP dosing in including dose-dependent data increases a X,X-DPG. ASH ascending X healthy multiple the of cohorts of daily pharmacodynamic regimen volunteer and once the activation of Phase in pharmacokinetic

portion Taken end X of favorable of the MDS Phase AG-XXX study progressing Phase we look of the together enrollment year. Xa with complete by lower in to and of to this expect ongoing the profile, this study a forward risk safety

we options limited Lack our address PK that the approximately development intake, pipeline. enzyme. in the PAH a continue earlier-stage deficiency to and of impact XX,XXX a to to life. PAH disciplined U.S. XX,XXX Brian of patients or the in phenylketonuria it's often PKU. lead activations and aim restricted accumulation PKU and through to expand pipeline, hydroxylase by caused sequelae phenylalanine phenylalanine we earlier-stage our genetic order which a highly to therefore and our disease minimize with to Within rare patient of of research to pipeline treatment and downstream total phenylalanine quality reduce beyond patients our EUX, is advised business aimed both PKU of activity further leads mentioned, diet can program the advancement with As of are progress or consume the

portfolio PKU, course five the of of are goal significant the directly to levels cause To this progress studies of I'm are the of with executing made we XXXX our oral the of developing we forward the end across year. and by address an with for ongoing the over maturation look an this our pivotal pleased continued and Overall, reducing XXXX. IND team the very underlying program filing phenylalanine targeting PAH in stabilizer

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

Tsveta Milanova

you, Thank Sarah.

across the and the clinical Since potential team, of rigor by to joining our impressed I've compelling transform energized Agios been treatment rare diseases. generated commercial data paradigm the of multiple of programs, hematologic portfolio our last and the month, the

clearly have and a a opportunity near long-term growth. so It is join truly the to privilege for company to poised

For XX disease global led leading I've companies. years, at last the launches rare biopharmaceutical

challenges, the those focus health each launches patient unique journey the the of remained patient core and as as While improve reduce efficiently has presented length of my possible. outcomes to same,

critical a of is the implement comprehensive from particularly each reimbursement. To state an disease it patient commercial to goal, strategy addresses achieve and ultra-rare journey that that awareness to for deploy disease,

diagnosis awareness maintaining of and Specifically, strategy increasing the long-term. must and the time areas: disease adherence between be supporting by and initiation; that a treatment treatment educating on by over on reducing and available underpinned focus accelerating reimbursement the access options; three key

disease. current of the Leveraging and priority drive to future this need launching fully in top strategy, my anticipated excellence is launches build to cell capabilities we deficiency PYRUKYND the thalassemia in sickle PK operational in commercial realize and the potential

the six progress prior PEF. line aligned are initiating has access. the in majority launch enrollment reviewing by encouraged the form team and label particularly clinical to policies with four completion In inclusion the I've after with the or criteria or weeks authorization criteria to-date, expectations Payer trial the made been prescription patients are on market therapy of of to

Launch to-date, low discontinuations barrier. highlights of remain and both and a overall the and progress PYRUKYND strength reauthorizations capabilities. This the market continue have of to our not positive services been patient impact access

quarter generated net launch, quarter including PYRUKYND we a versus quarter a fourth XXXX, increase XXXX, third fourth have quarter. million XXX patients XX% full of the third of completed In total in represented which of revenue. the PEF, $X.X A of now XX in the the

translated a over rate, to on XX% this increase conversion strong net therapy, our has QX. Given patients a XX of

the disease of that we have Despite with and in manifestation XX on PK for stem deficiency a progress, improvement. a therapy demographic growing identified opportunities Patients range base diverse prescriber consistent broad represent adult population. multiple is its physicians from and

As is a the observed earlier launches, in of and corresponding opportunity general disease disease attention of awareness and to and diagnostic monitor urgency clear greater diagnose, lack disease ultra-rare patient thereby efficiency. a stages increase focus common have This awareness presents treat. lack the we and journey on a to of

efforts diagnosing to Specifically, and patients as PK less indentify plan disease we to likely to was clinicians our PK code years for by high ago, machine the treat ICD-XX awareness with than capabilities two AI learning bolster our deficiency. potential augmenting established deficiency

deficiency. to to engage treating educate genetic of which availability to the program, given of the including a base, capacity and range strengthen including aim of hemolytic the Additionally, our provides PK testing diversity the and physicians anemia ID on no prescriber we cause indentify breadth anemia,

and building disease further franchise. us I'm meaningfully our diagnosis, full launch. rare directly will maximize beginning, help strengthening capabilities support to But future education, our only translate and population potential help larger the is current activated of the will capabilities us unlock confident potential patient adherence deficiency access that PK of potential the in will to PK these launches commercial the

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

Cecilia Jones

Tsveta. Thanks,

quarter found summarize. XX-K, financial I morning, we this our will fourth results XXXX later in included press filed detail full-year be Our will which which today. release More issued can will the be and be in

Full-year to increase XXXX $XX.X revenue of fourth including million quarter, an million net $X.X million $X.X compared PYRUKYND was in QX. the

will data we for is and population, first the launch this this guidance be the providing patient PYRUKYND As not therapy ultra-rare insights on continue to at and trajectory gather time.

be launches, other was Consistent of the Cost XX% with the rare quarter million. for disease gross to net XX% $X.X sales to in continues range. to

the increase and R&D $XX.X the for PYRUKYND to the fourth year-over-year for increased studies were expenses XXXX, and workforce and quarter an increase primarily AG-XXX to cost expenses was Moving across million for the $XX.X and by for XXXX. million the spent balance increased of compared full-year full-year ongoing R&D. $XXX.X five driven This million our sheet. studies pivotal

XXXX, TIBSOVO and $XXX.X revenue, our fourth of the for million royalty income income million on $X.X was Full-year million gain $XX.X $X.X under million. from an is full-year quarter compared for of royalty recorded sale business to were oncology XXXX. on expenses increase statement SG&A which the full-year

which income reminder, we Sagard one-time after on QX. net under in of given $XXX.X to a million income TIBSOVO sale payment recorded As TIBSOVO October QX of of sales our the a million to X% in royalty ceased of royalties $XXX.X XXXX for other rights U.S.

net the inhibitor we potential vorasidenib, of a to mutant IDHX/X. sales million potential clinical part on approval business upon stage to XX% As dual oncology royalties milestone of divestiture $XXX and of Servier, rights a retain our of U.S.

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

to securities equity. and and us the income equivalents programs anticipated development to funding through more currently without marketable positivity one interest and PAH operating together enable need of U.S. mitapivat execute revenue will with cash outside for AG-XXX product mitapivat, We expect flow to plan, our partnerships including additional our cash, the commercializing the cash or planned raise and

our To allocation the studies and pivotal managing to position, development. ongoing deploying base development ensure on our will pipelines a proactively focused approach capital maintain expand our through our business and cost remain to strong full of flexibility cash disciplined we

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

Brian Goff

Thanks, Cecilia.

progress XXXX at Agios. was a year of tremendous

PYRUKYND With and important PK first advancing needs toward in our address rare our step of suffering focused industry-leading we we've XXXX an adults the approval pipeline activators unmet to deficiency, of diseases. taken with on profound remained vision, of patients PK hematologic the from

foundation deficiency over will key that for the Phase anticipating and and XXXX, clinical number PK few In pediatric data of potential X lay years. cell the thalassemia, milestones we're readouts a sickle next in disease, regulatory

our to strive value to stewards continue for we'll always, opportunities of be creation. sheet meaningful will responsible balance evaluate As and

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

open now we'll for the questions. that, With call

Operator

[Operator Instructions].

Our question Chris with comes Piper first Sandler. Raymond from

open. Your now is line

Chris Raymond

question. Hey, thanks the for guys taking

Just I couple guess questions. a

sickle trial X have VOC show to cell know that? just Phase I the at the your guys endpoint then in a out And there portion on about you Phase this talked portion you clarify response, -- signal primary Can being of option hemoglobin I the of First the a trial? but X an is least have mid-year. reading follow-up?

Sarah Gheuens

Sure.

hi, So, this Sarah. is

we period of it of portion then indeed But the randomized That have path. are VOCs our response trial. capturing control well. X rate as indeed in safety primary a first a XX-week the Phase endpoints, hemoglobin said, the endpoint the is indeed for for So trial, the in we as of secondary being

drug. patients that So have by be is coming that exposed from time been data to the to the limited the going

looking in in trial, the the As will at looking be saying and which trial, we our the at longer we duration. that trials been data for as RISE always, the totality always well in sponsored specifically exposed patients this UP of be have been investigator will we've a also generated

Chris Raymond

on And hand, an Europe, Thanks. just in marketing until infrastructure not you've there, you authorization then a PYRUKYND build in wanting talked about larger get have UK Great. I really Okay. and in I indication. Europe and know the guess you to Brian,

strategy that update have especially, authorization. launch you there, So now any given on maybe that marketing for

Brian Goff

Yes. Chris. question. morning, Great Good

indication, know own we as have or quite So we as as we be challenge. U.S. on commercialization to for you an a just is mentioned ultra-rare would partners partner have that the commercialization you its have PKD interest think a about noted, outside in PKD, and

So as that mentioned with what footprint progress, a we're path as very we've particularly look and thalassemia stated sickle looking towards make be Sarah mid-year where for puts on us we data really the globally anticipate we well is partners come. will to represent fully by launches enrolled that just to cell we for expect that to we believe those the methodically

is thalassemia course, and an of footprint. mentioned like PKD, rare, a does is sickle disease cell I larger as just meaningfully as have opportunity very global it unique

So factors those partnership. are our that'll into the go

Operator

Instructions]. [Operator

question Cowen. Marc Frahm comes from with next Our

now is line open. Your

Marc Frahm

Maybe a question. Hi, just the on Phase X it. thinking data I'm thanks follow-up questions. for my Chris' about on taking we just as get

hemoglobin make In go/no X? just what go like data you you making already very terms revealed to to that? kind of of into robust, data the needing Phase and in need terms this forward things want decision be really to Are are -- there new endpoints Phase in for that to seen to more but you've a see the of X, go similar

Sarah Gheuens

So indeed our in similar relatively the to trials. what we are endpoints have other

first However, our placebo-controlled X. in the this Phase is datasets

looking are that. actually yes, excited to very about eagerly we're we So forward very mid-year. And

Brian Goff

I we've all underway. we add that hemolytic X Phase of I one is PKD, and we've that the I launch that's way data just of about already consistency and across X, which anemias, thalassemia, of would the the appealing think, been through Marc, of PYRUKYND through talking the seen is course, things And then to, the so have mean, Phase

study. data. course we we where if be expecting not the identified, for confirmatory X will coming you Sarah we're the UP We're and have compelling Phase And X look out totality as of waiting but RISE we the data say, then UP of And Phase surprises, as see the of at RISE data just endpoints noted. know, it'll

Marc Frahm

kind things this first patients PKD, Okay. to quarter. Thanks. that continue launch just full and you through reauthorizations in maybe the that, like is the quarter flowing QX of and to of experience some Then have

therapy that are you standards what for do the in being -- long-term? of What we of seeing applied? of percent patients you should to expect Just reauthorization kind terms on think the are continue

Tsveta Milanova

Tsveta question. lot a here. Thanks for the It's

of the really As all have we of we have therapy therapy number at end discontinued. on net patients the inclusive who is net of on patients patient and have And XXXX. quarter said, fourth who this the XX all patients started

reauthorization for low to assessment from to as it continue prepared that not truly on and get so the remain reception rate been months lower numbers the that overall of at positive not that U.S. barrier discontinuations The six me, in are have specific profile noted on But has PYRUKYND that We the reported reaching we for a reinforces any the far patients discontinuations. for payers patients, we -- the products all. and physicians

Operator

Please our question. standby for next

with Our comes Greg from America. next Bank question of Harrison

now line Your open. is

Mary Kate

approval that hit expect physicians is physicians This Thanks more how experienced Mary our you've taking mentioned unique questions. as become we morning. PYRUKYND? PKD, to guess do Kate of year about since XX you the change for of with and PYRUKYND on prescribing. as one usage for and I Maybe Greg. Good updates you have aware

Tsveta Milanova

the -- we've that breadth with progress pleased very actually are we of on So made prescribing. met

data expanding generated unique enrollment have have you moving core perspective, already, disease. that prescribing from and prescription breadth to of mentioned an And is forms. focus As ultra-rare XX going be to forward. is It XXX the we our physicians who

reasons keep the and the disease a of efforts basically their and go time time will and as part clinicians invest this efficiency mind And diagnosis analytics own treatment well. diagnosis actually have shortening from the help of awareness, to greater one and prescribers unique will really greater it because to very as the does data take with the help is journey of in journey each on main that diagnostic patient prescription to in from targeting of improve put We on importantly our us this to efforts emphasis first well. need

Brian Goff

that -- of as disease ultra-rare at two it It's Tsveta the And of describe or prescribing. inch add deep than because often for or kinetics as breadth we more PKD Yes. mile the an be it a I'll have really three be patients maybe noted unusual just physician for will launch wide, would to one about most.

So is that critically is this be the -- these to efforts analysis that be is learning that of alluded that of we're more areas then, claims our rare she will important. efficient in who which the prior capability on we modern are Tsveta capabilities our refers all proliferation It's look to secondly, more plan this for can master, likely patient, targeting a disease about key focus. we using education, really towards the guiding the And high database machine will important. effective and to potential first critically comments, state have working because providers of one in breadth a call launches PKD the that to

Mary Kate

if prescribing drug? prescribe Great. feedback PYRUKYND, those from receiving what physicians have been treatment the one who or you. you Thanks. Thank more And then, on or kind use and either patients of already for could I

Tsveta Milanova

the with across that well. patients, and pleased very We're the we physicians Board are from payers getting feedback as

to the see definitely but speaks we initiating efforts therapy of are also positive I patients great success reception market reauthorization payers the access with think for rate. both who the of our success

of those of of positive met it customers product, for early when I We anecdotal feedback services in on out feedback in get but to actually fantastic my not had which from reinforces patients. to I team, with experience with our the the direct is how went our a already launch only And from that really the perception patient the actually opportunity on and And PYRUKYND them some had hear. hear month the of lot job. first the the field positive, some is performing

excited. are we So

right going take I right it's putting As we said some some to time, continue we're place breadth prescribing. of but to elements believe of the to in the

Brian Goff

do experience? comment that's encouraging. trial is want really PKD, And for you the And to which clinical Sarah, on

Sarah Gheuens

Sure.

similar effect the have data patients to for the for very way Very are as the maintained. clinical it's reported we who getting hemolysis been in PKD on maintained, So threshold data PYRUKYND that feeling with and stronger we see and better. our gather importantly, is what trial significant long is impact also long-term on time it now a matures, clinical see and for hemoglobin is the outcomes in really maintained continue even patient better a is because this reach functioning

that, impact iron of are more iron top seeing on we well, start then about And what excited we well. very overload and where an data is as our overload as more on

we're about think very So I PKD. already with patients PYRUKYND continuing what excited has delivered and is to deliver for

Operator

next our question. for standby Please

from Our with J.P. Romero next Tessa question Morgan. comes

open. now line Your is

Tessa Romero

for team. our questions. and Thank you Brian taking morning, Good

one I if from disease me cell on So just sickle program, could. the

Phase go little a bit RISE the about in thinking your data decision here. go/no X So UP

I that also Specifically, being know crises response you as we've to hemoglobin And the is little about as past monitor. talked a view quantitative rate a there differentiated vaso-occlusive here? important bit in

help VOCs understand for what as bit look could us well? better encouraging an a So you like little trend there might

Tsveta Milanova

for our response, specific So between deciliter endpoint we hemoglobin be time gram bar trial. clinical qualified as right? primary response have per is the set hemoglobin there in points a as So do the to X the

protocol into our endpoints results testing. and then endpoint forward move primary in we we consider our secondary pre-specified have statistical our how for We significant what

classical Phase for of sort very a X. it's setup So

X, secondary looking of for what Now, for we're the endpoint Phase in course, is trends.

So our VOCs, we're time. now over capturing

it's across the that's deliver because in ultimately on. for target by hoping treat hit sickle both the then totality looking be impactful set the there is profile, opportunity outcome, on we're towards reported to and able providing -- impacting deliver that And product the we're meaningful drug patient to what X meaningful cell totality is truly that further accrue so hemolytic not something pain will Phase X a to cell disease which crisis but can anemia, We really up to data, there sickle cell. or our will hugely sickle of Phase of be off of then this

Operator

Please for next our standby question.

comes Renza question from next with Our Greg RBC.

now open. is line Your

Greg Renza

on color Congrats morning, Thanks. progress, positivity. maybe XXXX team. cash remarks and to opportunity Good just provide of perhaps in programs flow some on assets inputs turning the just the on and you to Great. mind looking how an then, many wanted I there. the just get of very taking my and and pipeline. Brian thanks development, course, to some on those give at question. keeping the in launch, you for assumptions Brian, much. you're external thinking and about to the your Certainly and the Thank mention

Brian Goff

Thanks. going start. Good Greg. to have Sure. morning, I'm Cecilia

Cecilia Jones

Thanks, Greg, the for question. Yes.

AG-XXX we -- as mentioned, current plan, that mitapivat in interest including described our and all enable marketable described the mitapivat trials And about pivotal outside and sickle income us program will earlier So, Sarah the earlier equivalents and addition pediatric, to includes execute revenue our Brian PKD to for as commercializing of also securities plan, will thalassemia, U.S. anticipated sorry, and the product cash plan, PAH. the cell, the cash, and in which part

Greg Renza

how mid-XXs? in do certainly as so through this I indications, for just of that across you the and these much. -- potential marched layering the all you potential we're sort know And the of color and a think Thank you see asking as not PYRUKYND development approvals you any as guidance, velocity backbone it. top-line, on therapy potential about Got but even on

Brian Goff

Greg. Yes,

larger course, certainly launch So maximize to for the come bigger with our capabilities for have namely to the And us thalassemia potentially opportunity. disease. sickle cell an ambition PKD strengthen we sequential commercial to is launches meaningfully and these picture, of PKD,

name ultra-rare by and it's nature. PKD, of case by the In

So estimates as as prevalence -- say peak to us peak sales well on as slow today, the us PKD where the it's launch, are the to to outside including clear will we then as distribution this learn far approval depends time to discussion that of some a steady. And factors, timelines, talked the the refine for and it take time sales, and about launch of will peak the I the the we we and third lot more. so sales full only U.S., be whole from reflects partnerships quarter would

expertise soon deep not rare experience out. has that moment we the as I joined just that's opportunity because Milanova going to and come. super these momentum that launches. the that the importantly, that's continuing take reflected Tsveta will more at PKD am us And launch in to reiterate as disease I I'm pleased too a numbers potentially I the has think to think I and And we say, ready vigor of launches CCO for continue getting us for and in Tsveta to report look

Operator

for question. Please next standby our

Our Mark with next from question Oppenheimer. comes Breidenbach

line is now open. Your

Mark Breidenbach

us questions related Hey, Maybe remind from the good morning. my down just have differentiated kind and sapropterin answered, me PAH in already been for are of the drugs could or you of how early-stage Thanks. that pipeline. for Most but late PKU? is let dip into PKU approved stabilizer development

Sarah Gheuens

Sarah. is This Sure.

or So been or have development a that our for has indeed, really are the market on approved a been others being action in have program different are of currently while. the it is and mechanism than

it's PAH different. stabilizer, So which indeed is

two lot are we this is sense is PKU of treatment that the an it's either do a to drug the limitations own administration. available there's of with currently because their that Where have the or take subcutaneous opportunity in product in see

to there oral majority that we lot treat an therapy a So with along of the would that that develop is PKU. that, where goes are looking

benefits And program. course, on that in the use, the more the on easier is phenylalanine to reductions, sense provide it tolerate. details also of Of something as different providing will to as is we for then progress CDPs hope can be that disclose to we our us the measured meaningfully all easier but

Brian Goff

so just EUX. market would addressable and function -- step talking We're be -- across the U.S. another that PKD. significant. patients here about change I'll Mark, we the the from with XX,XXX is are add to today This prevalence XX,XXX And in and where

And point this Agios about one is in the this expertise from our other cellular comes metabolism.

continued an towards the IND our We've end early about at potential diversification to now. pretty this potential are year. right of it's a So talked excited pathway pipeline. have But this as we of a

Operator

our Please next for standby question.

question Our James. from comes Brill next Danielle Raymond with

line now Your open. is

Danielle Brill

couple. Hi so I much the for questions. have good Thanks guys, morning. a

what remind and PYRUKYND, sickle cohort can First that us Phase -- a your X you plans the just on that's are cell How XXX, for tracking for enrolling? the then you're And on the then clarification, or I number plans are of received PYRUKYND? XX patients data? just total that that a factor that. clarify, on drug the have And discontinuation to does is to wanted follow-up in that patients I have

Brian Goff

Sure.

first start can Sarah So the one. with

Sarah Gheuens

Yes.

questions. for the thanks So

disease results a so in ongoing context data Phase X of trial looking within So generate all there sickle disease, indeed of hemolytic And we in to multiple a healthy an completed anemia. specifically is ascending is in for volunteers. which cell disclosed it's that it's within sickle have that sickle dose scheme the we XXX our the of X cohort the -- and indeed, in in Phase are -- which cell it's cell for disease,

trial. a it's short So duration

going is disclosed is ongoing now. have results. not release when trial to and right track We those we on are The

We are very excited.

know just going excited very didn't So to Xa because currently I that trial. is of Phase we to portion specific the about which are with our XXX, I'm say you want and I in just, ask, MDS it, but progress,

Brian Goff

I And patients, going I'm Danielle. an asked clarify. on good It's but you to then one Tsveta, the think to easy glad that's be

Tsveta Milanova

net XX so patients on at therapy of end patients. really To the QX a clarify. of is number Yes,

end at patients So actually the the all patients have were PYRUKYND the XX who quarter. started therapy deduct discontinued that the the receiving actively have still is includes of who patients of

Brian Goff

the is we as patient And to rates point is well thing we level. provider and encouraging again, which very super is at have that's look add discontinuation a performing that low, the launched PYRUKYND because come. to-date the shows very think launches bigger as beyond I'll data important it And about only us PKD, been

Danielle Brill

then for Okay. the Thanks. was guess I helpful. very follow-up my That's And enrollment form.

I'm just patients So drug? actually translate what of curious those on proportion to

Tsveta Milanova

the treatment progress of expect a comes That really criteria if to or market the the PEF payer pure when as to they the At we to by to do driven moment, don't require actually well six very between of the and lot PEF initiation. primarily genetic genetic completion progress actually services six There about patient of we've through a initiation. are genetic the because not a as is access majority the and force the fact we is see the weeks. results, the pleased PEF to the on have testing made process with four for it a do weeks. time the to treatment authorization of it's We Well, is timing driving test shorten patient that the significantly. they'll need four of to go testing. prior completed And timeframe not has that policies a and delay

Brian Goff

required much the an rare ultra-rare challenge is the in so and launch it's really generation the PEF, lifting terms so components. educational of disease Yes. The because demand heavy of there's in

time, launch. patient I PEF per pull-through the think to se the that's therapy not but of on will the real take challenge

Operator

Please standby for question. our next

Our with Richter next Salveen question Goldman comes Sachs. from

Your open. line is now

Unidentified Analyst

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

considered make in And guess we cell no Phase go be your the I First, And would in on? you decision? focused a into you. lead portion? what be update, sickle the mid-XXXX what then would conversely, X then just to success most Thank what should opinion,

Sarah Gheuens

Phase primary endpoints the hemolytic patient safety. In criteria X, then our have the are secondary supported which the hemoglobin our have So pre-specified endpoints, secondary for as we VOC, the and markers, our outcome, primary and endpoints, response, we for endpoints.

two We of primary endpoint -- all our We tested are pass its doses against bars. placebo. to looking first and

will dose doses better see forward for to into to we is be which Phase one X. looking move both the So

are And the pre-specified protocol. are so all that within things those

data the Then a will make program, account, profile. step our course, to that And take course, we development, decision as truly go in believe for we the sponsored we overall always target of the of are into of part for any going investigator of context an we are there program that we we is including on that have, other next trials. that sure our towards when we product hit take go/no

is that our excited for so talk for And program, discipline actually to like so And are we very this can about of all data. the we we reach programs. mid-year a have

Unidentified Analyst

that, I you. for would profile be sickle to PYRUKYND ideal follow-up on and the Thank just what guess cell? target

Sarah Gheuens

on can that like benefit the be So hemolytic meaning for are designed some of truly hoping our are that reduction ideal we CROs. profile to to is enter product benefit cell a crisis. on also cell pain programs sickle able then target But a deliver and have sickle product pathology anemia we to disease, demonstrate

Unidentified Analyst

influence you the If don't you that rates, just improvement -- see question. -- with your forward to on see trial? go more don't sorry, an I'm the not decision one if guess VOC would I

Sarah Gheuens

limited controlled VOCs duration in just the have a of So our we because trial on the X randomized very It's be of data Phase trial. portion. that XX-week will the

not the show designed that's that VOC. on huge benefit So trial is to how a

course, So investigator extension the we continue to collect in open-label have Phase and sponsored via X, much data VOCs on exposure our our then longer of in we trials.

Operator

closing conference questions. I now no further back Brian for the to turn remarks. am I showing to like would

Brian Goff

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

towards for momentum our and significant generating both we're vision, our value in morning, potential shareholders. deliver to this heard you As significant are patients long-term and we confident

we'll to of soon. look real you all thanks speaking and again forward So with

Operator

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

You disconnect. may now

Agios Pharmaceuticals (AGIO) 23 Feb 232022 Q4 Earnings call transcript