AGIO - Earnings call 2 2023 | Agios Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to Agios Second Quarter 2023 Conference Call. [Operator Instructions] Please be advised that this call is being recorded at Agios' request.

I would now like to turn the call over to Chris Taylor, Vice President, Investor Relations and Corporate Communications for Agios.

Christopher J. Taylor

Thank you, operator. Good morning, everyone, and welcome to Agios Second Quarter 2023 Conference Call.

Brian for our by I website, our Executive slides Investors to am access Chief of section agios.com. On Officer, can today's going call the joined You Dr. Goff; today's call, Milanova, Medical Chief Cecilia of Development; Officer; and Sarah our Financial Gheuens, Commercial Officer. Officer Chief Chief Research Head and Tsveta Jones, and

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

Brian Goff

leading for delivering In with Thanks, and in thank quarter, morning, is the pioneering diseases. focused you and PK living significant made we patients second us. Agios for leader activation, on Chris. on everyone, rare particular, therapies we're hematologic joining focused industry the in that a Good step progress PK with today. and diseases, transformative strengthened, underlying to our activators pipeline targeting excited share share of each readout, hematologic updates forward, you the pathophysiology. diseases With the data is platform of common each probability our for advancing success we are

Under to this and prioritizing key leading were pathway; this of regulatory for path very Agios potentially a we're criteria: derisk for an rare transformative therapeutics or in to standard with beginning potential focus; of on novel to development disease disease commercialize Alnylam which phlebotomy patients; fatal that announce the the at agreement, potential that is the affects license acquire these clear pleased is year, value siRNA articulated the U.S. to rights morning, we patients a and RNAi and agreement early; approximately rare opportunities business Earlier care. preclinical PV. PV As based is aligned we treatment and Pharmaceuticals, X creation. polycythemia this will a identified vera, Alnylam's the develop XXX,XXX X criteria. for of highly with company potential hematologic the

high The a in IND-enabling also of for dose response to disease-modifying the has in treatment in the III PV weeks, eliminates by This Phase treatment analyze portion goal siRNA patients in the the detail more minutes. Also therefore or Our to disease. unmet aligned need development announced that on in we of not reduces our UP this capabilities our sickle is with need this results seamless provide for XXX-milligram cell scientific address II the in just convenient the candidate for mitapivat Phase rare Phase results only primary team endpoint expertise agreement RISE will delivering our from continued has both met option operationally forward and the is Sarah a business of selected hemoglobin but strategy, development the arms. We and studies quarter, and portion the initiating core recent And mitapivat the few look clinical positive commercial later study. study to of hematology. year. II/III phlebotomy. study

life execution growing data III quite year. Broadly, these PK multiple body add to compelling Phase and that of enroll later first on focused outcomes function, and potential quality differentiated the the this our to activators, patient continued now areas. of are We and transform across results generate of of with have we are this to long-term consistent action mechanism eager the to disease patient highlighting

III to this and set context, has demonstrated and X activation this lower enthusiasm year. progress, And the PYRUKYND the pleased clinical of studies the consistent across in X of investigators' than for potential for In any fact, for diseases. In Based clinical reflects also results in to more readouts year. activator, MDS timing years in experience PK distinct data of quarter this largest we Phase expected of risk in novel pulled on were ENERGIZE mitapivat MDS. PK IIa activator, we Phase of ENERGIZE-T study in indications. our as Phase operational AG-XXX, end thalassemia progress study well IIa line these next we forward X that this risk in top the the our enrollment the expect PK and studies both we've thalassemia III excellence results completed lower as the of the Phase development to This from continue announce in with

we're business, consistent trial mitapivat to on that translate clinical among to adults with observed commercial living experience in continued has in compelling our to of the deficiency the persistency see PK and the Turning real to encouraged efficacy world. therapy

of just current opportunity MDS. we in the realize potential and the in As maximize continue to disease in will anticipated on a a capabilities cell sickle in launches commercial we're lower detailed few minutes. fully future our launch the thalassemia, the provide PK needed to risk performance Tsveta deficiency, building update

quarter As of $XXX position brief from on nearly a we you'll Cecilia, million hear the balance One reminder. ended with the second sheet. cash

net we vorasidenib our in retained we of the by of of of of forward the by a We ACTIVATE-Kids the PK to the and XXXX disease FDA number enrolling pediatric sickle deficiency to PK and upon in clinical in mitapivat look forward next Phase potential anticipated and that, very XXXX. With thalassemia we're over in stabilizer deficiency; to turn potential in and Phase than for III the half enthusiastic results additional milestone patients the of about rights call more expecting the the ACTIVATE-KidsT lower the to III readouts trial, the newly tracking steps. We're of of data studies milestone, royalties $XXX I'll AG-XXX end readout in MDS. We're for milestones momentum mitapivat PKU; studies IND approval the of added look pediatric the the U.S. risk part encouraged IIa the including Phase in on were pH in III year, and of treatment from our As readouts building filing of sales. development in cell the oncology from divestiture Sarah. now Servier's the study million Servier business Phase a of mitapivat Phase to XXXX, III from studies and

Sarah Gheuens

novel that what Brian. U.S. we patients. a Thanks, reduce affects are XXX,XXX treatment serious, that patients Today, is aim XXX,XXX there disease and and pain and that to deliver in fatal sickle cell Sickle both to is hematologic disease improve no potentially anemia options crisis, the these approximately oral EUX. for cell

arm numerical pain of of mitapivat RISE week a These in improvements UP II annualized BID in well were increase significant and and of hemolysis statistically study. compared X sickle achieved rate to more placebo. study. patients line per patients in as baseline. in by rate XX.X% with the patients the data deciliter defined Turning compared reductions erythropoiesis hemoglobin through week accompanied mitapivat mitapivat compared of placebo the response demonstrated XX-milligram in of an this of in XX% cell Treatment arm. Phase X.X% to increase with XX-week the XX response BID crisis response for as top markers as the XXX-milligram arm increases of hemoglobin to or was in the response XX hemoglobin a gram in average concentrations hemoglobin Hemoglobin from

need with disease sickle in XX pain extension the in in the data reported mitapivat the Phase continued to X.XX of study the crisis. any to option data compared in reduce by study, cell previously II in rate period. These And the that arm events placebo patients treatment delivering enrolled patients experienced were consistent an XX-milligram novel cell and anemias, annualized underscore cell crisis the in and X.XX was mitapivat no of finally, disease further generally cell leading to improves XXX-milligram other safety potential address profile of patients studies oral anemia study adverse in sickle sickle both there discontinuation arm open-label sickle hemolytic mitapivat high a of pain Specifically, in for X.XX the with treatment the and of for and other the of arm. arm. The unmet observed XX

analysis later abstract submitted observed and III. We data the recent of both continued we have Phase of analysis are in the dose have compelling to at Phase ASH on doses an present a year. Based weeks, full for selected excited XXX-milligram for this II data the

endpoints study of of first in patient and reminder, patients Phase the Phase XX-week forward a RISE RISE this enrolling sickle III III of a the portion fourth be will pain either cell primary hemoglobin the in to UP mitapivat look As XXX we rate the annualized and portion crisis, the quarter year. UP response to are will include twice which The of period daily. X:X in or placebo randomized placebo-controlled

to pipeline. Turning our development broader

half We in the our enrollment of the have year. across of with first very been programs pleased pace the

making including to completed primary to a the will year patients deliver across the line of expect have is MDS MDS next second regularly ENERGIZE-T, Phase readout readout. IIa thalassemia, endpoint look lower mitapivat potential of oral hemoglobin the of year, response. the of enrollment all to ineffective activator the of evaluate several to thalassemia the next also both strengthen in We to primary with and regularly which our end not in year. We franchise. ENERGIZE, in transfusion in which AG-XXX results a of this the studies fee first from mitapivat this transfused study AG-XXX who reduction schedule. enrolled of that has next enrolls in and study PK who study now are pleased half in with III IIa ahead endpoint a transfused response, are patients Phase of lower readout by this anemia risk ENERGIZE quarter, improve to complete of months relevant the of Together, PK thus ENERGIZE-T data become the potential We thalassemia in therapy our of and population data these studies of AG-XXX first were novel allow the forward activator erythropoiesis to clinical the entire the us Phase hemolytic subtypes. We enrollment and half risk top

X or to pH is reminder, of from through for progress study per consecutive transfusion as directly this brief the of the with advance ACTIVATE-KidsT AG-XXX to IND MDS measuring week in address endpoints: phenylketonuria, of hemoglobin studies As a increase for PKU. concentration XX; baseline objective by we the with study towards filing pediatric for deficiency transfusion III PK and ACTIVATE-Kids parallel, Phase or burden and molecule small continue the cause in free only. In stabilizers weeks underlying transfusion average the our deciliter week during proof participants X.X more for participants following of low mitapivat of the and the defined the independence an concept X establish more from of grams risk or to in response lower as hemoglobin primary defined primary

Finally, death. forward development events cell hematologic program expertise Polycythemia leveraging reduces a this as as the disease this in is with and to we agreement can TMPRSSX, Alnylam, mentioned, of to well progress in call pleased cardiovascular we blood this the with of the best-in-class production. is leads PV IND-enabling over a Tsveta. blood pioneer red We The hepcidin candidate internal later that that red red in and vera therapeutics studies clinic. With a increases are year turn and will siRNA rare to that, platform. Brian announced I production. rare characterized look with together of volume increased cell morning viscosity with aligned and TMPRSSX production targets by a blood which Knockdown now blood driver initiating cells, excessive key our is we deep expertise. our and hematology result thrombosis, RNAi towards

Tsveta Milanova

Thank you, Sarah.

initiation, awareness will sickle the to the disease and a the in potential maximizing on foundation cell PK building serve today realize also in disease education, future in lower and current as persistency launch phases Our launches risk commercial and access on and patient fully strategy journey. of capabilities MDS. thalassemia, anticipated organization opportunity deficiency we and adherence our executing on of focused The the all by remains are across

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

levels, long-term For disease patients of reported interviews drivers frequency including fatigue a improvement and hemoglobin PYRUKYNDs, include and recommendations clinicians, experiences, the Most energy conducted hemoglobin better caregivers. and we of or level, sample reduction patients in impact understand on with treatment in in burden. improvements positive transfusion their a experience key recently these complications. To decreased in transfusion positive in on reduction

Patients low initial the PEF, including first after and second XX% Importantly, into XXXX, increase reauthorizations XX completed net discontinuations quarter increase this XX on not XX% the quarter prescription XXXX. year. PYRUKYND both on observed This and million clinicians over from payer treatment form, demographic disease consistent been a of real continue that world with or physicians to and QX feedback $X.X broad last of population. of total have now of use XX% of patients and enrollment patients over a barrier. In in is stem the XXX growing deficiency the XXXX, increase XXX we strong consistent the versus therapy diverse quarter. Moreover, in a with have a reauthorization. remained an we PKD manifestation adult represent a base patients therapy, second A provider of net translated this generated is has quarter from revenue, and the in a range persistency

these we on In early likely treat deficiency. to disease, expect this Given adult and patients uptake. providers continue identifying we to the nature steady with focused PK stages, slow remain ultra-rare of

center believe potential utilizing analytics who have improve to we meaningful a PKD there data as on adult under may physician efforts management. prescribers number are and targeting Our of patients

also treating to physicians regarding patient of maximize to doing and and of the in By appropriate launches instilling We for larger impact, so, meaningfully urgency populations. foundation benefits will diagnosis educating the improve a potential we and the launch of continue the help lay and current efficiency treatment. potential sense

including The breakdown beta in of U.S. thalassemia thalassemia. alpha in launches XXXX where first do patients and subtypes in the potential next illustrates is slide and approved not an the option. This U.S., non-transfusion-dependent transfusion-dependent in thalassemia approximately have and thalassemia thalassemia, of treatment of XX% these the

first Importantly, mitapivat the erythropoiesis become ineffective to has potential range anemia and the full the therapy improve thalassemia of oral to hemolytic across patients.

range XX,XXX in forward across a thalassemia a Given gained differences fit-for-purpose the the X thalassemia geographies, comprised XX,XXX we or to subtypes, countries, as from U.S. of patients Together, relative in will call The thalassemia market to look the meaningful approximately launch. Gulf prevalence cell in where GCC, patients. for provides XX,XXX support competitive U.S. disease strategy such an sickle prevalence current accelerate and now addressable approximately patients is and the future commercial capabilities and that the in launch This leverage Council and I And us implementing additional region. the of leverages EUX. our GCC concentrated overlap also turn to prevalence the to select opportunity commercial Cecilia. is in cell that, to the geographies over in sickle is the efforts of disease. With

Cecilia Jones

Thanks, Tsveta.

our XXXX release be and second provide the points. some highlight to a results like be Our context quarter can take will moment financial today. I'd in detail filed included found more press morning, will which a in we and few issued later be XX-Q, to this

PYRUKYND $X.X first net was million ultra-rare Second this the quarter of QX is PYRUKYND $X.X XXXX an patient XXXX. revenue adult increase for to therapy population. PKD compared million,

the steady not to slow be to and trajectory and insights basis. was we launch disease to to expected a an continue for gross on will sales Cost therefore $X.X But at this time. net other in XX% We continue data rare to annual to gather and providing on Consistent expect the range be million. XX% trajectory is quarter the guidance with of launches, peak.

of our increase XXXX. SG&A in second evolution on primarily was primarily to were $XX.X XXXX stock-based compensation a expenses workforce-related decrease the compared sheet. headcount driven $XX.X the $X.X an decrease for the second by for was reduced the a the compared second an in to million in related driven expense. were million million that of of increase Moving This to balance as research quarter, XXXX. expenses organization quarter expenses of second of of million decrease quarter $X.X by quarter, result to R&D the a expenses

As our potential XX% given U.S. of royalties of on October approximately retain royalty cash potential sale part the rights we ceased, securities U.S. has royalties XXXX. equivalents net $XXX cash, Servier, to in ended X% to upon million with and on marketable milestone Sagard the vorasidenib a a and of approval to million. TIBSOVO reminder, FDA net of quarter of rights to the sales. We $XXX of sales oncology business our TIBSOVO divestiture as And

that XXXX. include outside into from mitapivat or least agreements. commercializing vorasidenib, business does our expect and potential the operating through inflows cash expenditures milestones capital of vorasidenib from together We interest company strategic product patent, enable guidance and This other U.S. at royalties value-creating anticipated expenses not several income revenue, for fund and one through milestone or partnerships will with financial potential this the the to more potential or

by We near-term of are for thalassemia focused on creating as timing potential for of PYRUKYND potential all our to combined. grow and and cash revenues disciplined a $X cost billion we remain including PKD shareholder excited additional peak to make million $XXX approach prepare profitability. for PKD expect to U.S. our proactively PYRUKYND of continue $XXX managing a worldwide We and continue to the launch strategic base about allocation in and the deploying investments million pipeline, value, support impact and launches which to to and sales advance our

significant through to to position us the Following for additional back development will we create look momentum value-creating on created shareholder milestones call the now balance plans ways approach sheet potential turn enable execution am our that strong and to of Brian from of value. I over his confident execute the remarks. as the for horizon, upcoming our strength will a to to continue closing I

Brian Goff

and to we was make compelling, We continue pipeline by potential fueled compelling rare our to studies, approvals to the expanded across core portfolio vision strengthen our sickle cell clinical develop continue quarter an that of Phase The profound advancement support hematologic to our This future completed with positive spanning a has franchise expertise disease. a the announced business is in consistent continue transform for need, disease hemolytic rare X a progress in with Agios. launches. in towards anemias with data Agios, meaningful at we data capabilities Cecilia. to of and established remain our and to and disease, Thanks, pipeline in-licensed generate which is PK tremendous that of our industry-leading aligned internal hematology an enrollment activators course and commercial II we program X unmet external development

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

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

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

Operator

[Operator first from will question Bank of the Greg Instructions] Our come America. line from of Harrison

Greg Harrison

updates. you would the Alnylam. potential start your what And own And specific this on to expertise development asset to value? And having be you of from could model then congrats the BD the this PV a broadly be expect in future off, patients, would or to population? terms To on a PV you where factors applicable and for across focused the it add patient treatment more led decision stage all curious license and is

Brian Goff

us, is potential these reinvent just care, opportunity question. just these thanks ripe we some believe treated it's -- I'm therapies a it's to a a phlebotomy. standard to as is been do to rare that best, disruption This for lot diseases noted to for Yes, announced Greg, about the the a in morning. Sure. that which way deal if is And the And great growth. bring that's the We provide do patients. great and large it polycythemia this market. what I vera of comments, happy quarter, around is think comments disease-modifying and and are we my you

BD from we're Alnylam. with about well excited our fits It this disciplined criteria. particular So really asset really TMPRSSX

capabilities, commercial our scientific across you development boxes. capabilities. the capabilities, on about our so Builds many that all checks that's and expertise, our And if we've criteria talked it the think times, about core our BD

early is We sweet Alnylam spot also clear disease. see strong we the value have probability pathway. a be which world-class for Because ultimately, or creating a We belief. transformational is platform, generated believe of from the this the an is lot we've as this will opportunity regulatory So date. there's for derisking, RNAi it strong our for rare from potential talked to a patients. in about conviction us. It And success comes data a

this. I from go guess question So we're we where to clinical about excited here your of for development.

it's define and early little but too ask if profile the to -- I'll Sarah comments, wants for I make she to think a specifically. target us product

and and of of just different I'll deals BD of I you so types for going BD thing But pause development, where of of about Sarah, safety in for just the then we'll this the all be have the optionality options. is asset our the boxes, in those in. patients, play. motion, going of point particular, disciplined But product forward. anything really your mentioned, were a We our the theme range we provide really updates BD given course, to deeply many put all as convenience. say looking deals. retain bring add? a speed eager forward. accordingly. And were and one at we're expertise itself We'll stage development retain action bring going in to look wanted We and, dimensions, it the at to This so efficacy in, of can profile forth we to for we'll criteria and And it last then checked for

Sarah Gheuens

very Only to say this. excited that about we're

shooting are, course, program, program deliver make be details you the can execution we the as with the meaningful with And forward will more and value and program program. for on move to We we of development well. always clinical have patients once change development our expect a on

Brian Goff

we excited to that. Greg. And Thanks, are do

Operator

from Our from Capital question next Gregory come of line RBC Renza Markets. will the

Gregory Renza

progress. the on Congrats

Brian Goff

Sure.

Gregory Renza

for ones quick couple me. a just Maybe

the see data differentiate Phase sickle. potentially how you Alnylam data others great on to can then curious for on Certainly, asset helpful the manipulation I'm to this sets would available of selection just think thoughts you up your III to just just up Sarah's it date. Maybe preclinical date. to dose the secondly, or be and following hear survey the TMPRSSX. well asset. in Just in-licensed exploring when And the

as the the made just top since curious, Just the the the for looked mg. weeks at several Congrats last data the rationale body you XXX of step over again. line and

Brian Goff

a Thanks Sarah questions. going send And for over Greg. then to this lot, I'm to both

Sarah Gheuens

you. thank Yes,

data. the So around preclinical

So was and safety vivo profile proof has we very favorable target that knockdown non-human it -- very are good of of a concept in demonstrated with in observed. primates the

So very we feel as earlier, going the CTP confident very get about IND-enabling we detail that. as mentioned we and we're to on more and eager And provide that studies on the progress with that.

I are yes, market. is others administration. and there think at this of administration this point, us, very for a are Brian's around safety, that to going also differentiation also target, and There frequency be looking of is efficacy, big mode to

believe CTP very again, we going able yes, deliver get the be So excited our market this. on to needs. on what to that will And

Phase For sickle disease, excited. obviously, III selection your dose around second we the question cell for very are

that both we're are to benefit/risk favorable. fortunate of have the those We and profile very the show Phase excited II, data very was about

team prespecified have the the look has of at to time now data. protocol, We criteria the and in all

So we that selected milligram. XXX framework the and followed

on data. And to the conference. then abstract mentioned able of have so hopeful have that provide that outcome to are ASH, we not at that further details We disclosed now awaiting we're We submitted to detail and that. all the be the

Brian Goff

see the in R&D This the have mark team. that the we effective. that the great on just of too proceed think of of in prepared we tag quarter patient just trial I'll maybe a organization. Sarah now opportunity is excellence that speak her first is, we III fourth were Greg, entire and and operationally just excellence that love II/III again, efficiency Phase about say of both for always UP I for dose, to the with both doses. and the fact were to point dosed the RISE Phase the the can fact and XXX-milligram real and doses take I of the pride to were We a have design example pride that And the And trial we with the thrilled

Operator

question from next from Our line of Divya comes the Rao Cowen.

Divya Rao

for This quarter. Marc. on Congrats the on is Divya

touched those X you this And then trial? define define being do with how space. maybe just have I build several MDS to as mechanisms combinations Just know were you Or a you there in We Do curious, opportunities you what do those I -- competitive? view that see in more from are do or as other -- that are the us. follow-up. AG-XXX? success

Brian Goff

will mechanism start Thanks, saying, pyruvate And PK this all of areas IIa And question know with MDS, very the we activator, we're we're focused I low-risk action awaiting kinase Divya. different just as be about is Yes. activation. by the is a case on, with that of course, the with disease for in to the talk data. AG-XXX. our other what I will Sarah eager of terms Phase have

Sarah Gheuens

So yes, thanks for the question.

done of an want IIa to that again has enrollment on that this amazing program. So I what job highlight think for the team I here program, MDS Phase

the investigators MDS highlights of the patients. also it mechanism for think of for this I enthusiasm action

safety. to indeed are fast therapy, is then do IIa, to which will in a efficacy that's set signal population there a well. it move of is success that population. then indeed underestimated with success, we think is of a we I which into then so a we are ours this ripe our a to a we dose looking studied an not IIb there, MDS as I but it action, have differentiated our up currently is are cannot for others, finding. It or We also be that proper as not And it's that And And favorable use if for Phase for patient setup, convenience for really is believe evolving, and up is change. define is market that what set -- market mechanism -- that provides by is that and we Phase -- this being very typically an far which a framework do is declared of not think speaks older that also we oral a for are patient advanced by

Divya Rao

program. assumptions the the really quick you on is helpful. disclosed have on FDA? for dose your data? with you analysis That's remind the the that based of the discussed cell III then if discussion And then sickle you us internal Or question the Phase powering a just XXX-milligram Could And on just and mostly trial?

Sarah Gheuens

So dose, all program, that the internal now, so the have that framework for disease this the assessed. sickle we cell XXX-milligram data and we the have is applied right of

together. great very it's always -- about wanting like do with deliver and look of our medications always all because Phase benefit/risk shooting are the are same to I We end patients. we we We programs, a FDA. favorable excited to II our to are meeting forward change the And we constructive that that have provide have an the really feel Across right? all can of thing, dialogue

always with much sample we very the bigger pleased size to a the have. In regards constructive so the we're powering dialogue assumptions, than that the obviously Phase So is II. Phase III

there. the For X so endpoints -- there's

endpoint, on make then obviously at For the there make other VOC, the looking leverage we powering we been our statistical looking for the all have of the underpinnings. to VOCs assumptions hemoglobin programs primary And are patients. change and a meaningful data to used internal for for and

Operator

of Sachs. Goldman line Our comes Richter Salveen the next question from from

Unknown Analyst

] on Mead Anna [ Salveen. for is This

Just on the from Alnylam. with agreement one question licensing us

the siRNA of what in novelty the clinical Given the Agios, gives confidence process? for development modality you

Sarah Gheuens

the thanks question. for So

that modalities. have it internal many think this on confidence have also modality, other but expertise us I because we So worked gives do

it's like for modality a compound compound a not employees. this or Agios is not new while it's Agios, for So new

into of hematology to looking of work. I forward putting to development. straight that, so patient wheelhouse endpoints, And do internal And the think we're in knowledge populations, our of context drug all that it's

to putting forward work we skills so those are there to as And looking well.

Operator

next from line of will from Leerink. question come Andy the Our Berens

Andrew Berens

wondering MDS My the give PKR of questions also deal. program. overview on the the MDS. you us you on are could Congrats in I was some if see class how

in in is pretty for a luspatercept rationale over think give the indication that think as that actually see as And disease of $XXX it well usage us used you it's doing well subpopulation. Can I I hypothetically agents? class sales the EPO in million how to being physiological agents MDS in with pricing PKR early, impacts you potentially how power? do lastly, you the but EPO the relative know

Brian Goff

Yes.

IIa So I'll as you they're making. just it EUX. Andy, one based opportunity that, as activation, another progress We be and as disruption patient disease in mechanism is top action. It a oral XX,XXX we times different couple potential to an that attractive of on for we slides, by significant coming is we ripe we're and a across of with very identified what question. lot on a small our had find of the The of current has and U.S. the has thanks differentiated. just by noted to PK going the guided And for data. a start population, to think all molecule, of be reinforcing that said. the already, on the course, incumbents XX,XXX Sarah very patients But

You want to comment Sarah? further,

Sarah Gheuens

that similarities MDS now -- the between well. on big quite the truly rationale, and a we thalassemia progressed MDS, and there of thalassemia are work have why part we believe just Well, in it's obviously in may acquisition

So fact PK one which pyruvate multiple pathway disruptions. we we that piece. levels, hexokinase the is is also, a deficiency. patients MDS as at can impacted that also MDS glycolytic in is an influence There acquired patients to ratio have often well. believe It kinase is There

why a PK there's multitude So provide believe may of reasons activation we benefit.

at at come to EHA, and published at on hopefully this data more ASH. We ASH have

excited we So evidence that very about disease. this build we continue to are the in

Brian Goff

areas Commercial one our we're add that in very therapeutic And other sitting comment been is on. have has with our potential across goes Tsveta involved And actually today, Andy, space. this Officer, just focused deep Chief who we us Milanova, team I'll many

IIa not And asked lot early, maximize looking expertise Tsveta and insight to beyond of and profile how the course. that, a as But line be will it's about, down data. able add the at value in just the also creation. Phase product, of so you but We're pricing commercial we the of

So milestone we and by feel like prepared we're year-end. wait really cannot well this for upcoming

Operator

Our of next from question comes JPMorgan. Romero Tess

Tessa Romero

today. one commercial a ask I'd thought I

accelerated, quarters the way some to patient here. the you on of to how more drug, given of is launch net the initiatives couple low. this is et cetera? patient like rate run seen on just sequential maybe versus think grow you think in so about adds you've think something right sense as get pretty the forward drop-off or And new the of you patients It of trying last XX PYRUKYND, For about adds going analytics, do like sounds a trajectory

Brian Goff

Tess. Thanks, Yes.

about Tsveta So to have going is a so question I'm thrilled comment. commercial, to let her

Tsveta Milanova

Tess, to real so Most when the the the especially world, said continue our know it the is and we And steady feedback of provider time, also in you to At payer said, the we PKD ultra-rare an see see profile to as we strength importantly, would persistency, to to slow we disease. we comes positive as on make uptake. support. progress remarks, throughout continue opening the PYRUKYND same launch. the expect and that continue

support as expect analytics the execute are quarter-over-quarter, data expansions who patients we and the be and our importantly, potential PK some potentially us as Very to to for in in appropriate likely for but focused efforts to of variability to will very as as into today for deficiency, laser And all our continue a have and right as continue in providers on is with building this continue future foundation capabilities today we efforts continue look or the who We future to good to our are PYRUKYND field. identifying indications, well. the team thalassemia such are patients we PYRUKYND serve see either future. where providers, the

Brian Goff

look to pretty and lumpy slow But very as it quarter-over-quarter always the I It's just we Tsveta only And the proud would be the of will learnings careful that spectrum, steady a this a mean progress it some on over far I for and and we of be quite unique that continue add, little at just are from the growth. because Tess, Yes. make. is time. I'll kind we're But thing caution very to bit team is launch. it. ultra-rare building

Tessa Romero

just Great. could in squeeze commercial there. And a follow-up if I

dynamics, feel you that these exit position into something Is do a PYRUKYND guidance? of providing longer-term how you and about as we kind think year? Given to get do next in like you XXXX be may

Brian Goff

Sure. Thanks. toâ€¦ you want Cecilia,

Cecilia Jones

Yes, that. could take I

therapy, we'll before, There learn At and to continue point, we're going when So first this to data we as an to it's come not through for continue we this the will time to that. right mentioned do insight. give ultra-rare and a -- guidance. point evaluate is

Brian Goff

Yes.

in And bigger where point, the the anyway we're That's for on we anchor the in approaching as of launches. Our collective $XXX data. course, million be million to until where potential want start that get at the we've has feel is we belt talked we picture launch us revenue quarters about, That's for that under give U.S. the We $XXX we in multiple guidance our a guidance confident. readout look peak more may to the thalassemia of scenario a PKD. And larger meaningfully sales, of XXXX. to

Operator

Our next Raymond question from comes of Danielle James. Brill

Danielle Brill

a in-licensed have also I on asset. question the

Or So development. just well? might regimen? more antibodies I could I be superior you if it in modality also a be this wondering convenient on advantage siRNA know a think targeting do are think an Is dosing as may TMPRSSX there why in was there elaborate efficacy indication. you you

Brian Goff

now We the red Well, -- earlier, there production. thanks feel That the of that our hepcidin, mean, thread so potentially we Danielle, as through effect iron, TMPRSSX has in. decreases knockdown very noted which we which a diligence, for ultimately pathway. simply, question. through a uplift very are, confident we cell well-established is TMPRSSX XX% on differentiation. of an blood opportunities multiple all for lowers the I mechanism, think feel lot, I

from And for then room that particularly some studies being of because and then our will Alnylam has one as to that Safety, that our is plenty proven for meaningful with going wind progress will there's also siRNA of that could we'll start has delivered efficacy. early, at the you course, opportunities and the I treatment. current this there a opportunities will mechanism said, opportunity think look it look classically, of interval of very benefit out of I to accordingly. year, be and platform, report a that's patients And lower we're a we IND-enabling But all convenience up when at -- be for patients, therapeutically improvement.

Operator

we're no And additional showing questions.

turn will to we Goff for the So final call comment. back Brian a

Brian Goff

all thank today's for you All and your in in right. call, continued much very interest Agios. course, Well, of for participating

our by this make really you we we are across heard As portfolio. tremendous the morning, continue to progress energized

both value are in long-term and shareholders. our patients We potential confident to for significant deliver

you soon. look So thank you and again, much very speaking we with forward to

Operator

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

now have may You Everyone day. disconnect. great a

Agios Pharmaceuticals (AGIO) 3 Aug 232023 Q2 Earnings call transcript