AGIO - Earnings call transcript Q3 2024 | Agios Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to Agios' Third Quarter 2024 Conference Call. [Operator Instructions] Please be advised that today's 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' conference call and webcast to discuss our third quarter 2024 financial results and recent business highlights.

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

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

Brian Goff

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

Agios that medicines living at deliver mission of elevate rare and is diseases. to and patients the lives with develop transformative Our extend

dysfunction We syndrome are of disease on that cells, rare lower red sickle diseases especially in focused MDS. pyruvate myelodysplastic deficiency risk thalassemia, or and and blood cell PKD, destruction or including kinase the result

an blood path health. XX% cells comprise represents cells improved all the to body, red optimizing cell health of human individual red As in over important

to activator, red Our metabolism improves available mechanism red energy blood product, and of has blood cell increases a lead support pyruvate of mitapivat, ATP kinase or cell novel health. a action that the amount

progress III Agios announced from Agios' pipeline, $X.X in data to excited are connection those for payment that in in in in a August Agios oncology Pharma divestiture has We at vorasidenib, the cell royalty Royalty May $XXX about mitapivat Servier delivered current business payments of I in confirm payment Phase million milestone Agios. in XXXX and billion and programs prospects sickle with These from thalassemia following XXXX. readouts its purchase $XXX our delighted have data originated positive received we with today continued the agreement connection million proud PKD payments am and in vorasidenib are to across and include a the FDA which of approval of disease. Complementing

III of of data III accomplished the launches Phase in aim thalassemia in sickle RISE and enrollment we for achievement full strong recently prepare XXXX in disease and cell UP Phase readouts potential XXXX. deploy of positive mitapivat the the announced this to disease base study, cash the Given mitapivat in this thalassemia in of to sickle year cell

of also across important infusion significant pipeline. this we made capital, progress Beyond advancing clinical our

designation drug need known study ACTIVATE-KidsT we top initiated FDA PK to achieved for program the the MDS, we unmet profound of in regularly announced mitapivat an begun III line AG-XXX, of for study with with IIb syndromes tebapivat risk our clinical deficiency, potent the Phase enrollment growth. treatment as We formerly milestones PK orphan granted X in activator, of from MDS. significant first announced data area study Phase myelodysplastic lower that transfused, patient second, And have children First, mitapivat. And tebapivat, in were we our and our pediatric MDS. lower the market development dosing in of risk and or who

key team's R&D the very on and is for we expanding achieve potential III in U.S. the detail year, greater successful round provide our a to and well mitapivat of track for studies of as preparation current robust a ENERGIZE launch in productive preparing in sNDA the adults a label mitapivat, with we as market of in thalassemia in just Sarah living PKD XXXX, just positive update the thalassemia. will subtypes the of milestones an organization on across XXXX. commercial and our mitapivat bit. our in year, Tsveta this of launch detailed thalassemia by minutes. Leveraging provide a Importantly, and seeking out that a are in a milestone submission progress upcoming few in as includes actively on Phase remaining all thalassemia our an end broad data for on observed will ENERGIZE-T the update opportunity

Finally, as quarter balance the on Cecilia, the cash from you'll strong received FDA very payments we with $X.X third following which cash we in and milestone approximately $X.X investments of includes in ended position, with approval billion hear the a billion vorasidenib. sheet,

and proud continued that, strongly to finishing are over to far on want year thus XXXX all turn I now for enthusiasm Sarah. the Sarah, I'll been XXXX, we to off emphasize in in how for incredibly progress achieved remain that's we handing the beyond. With our focused Before how intensely and call

Sarah Gheuens

Brian. Thanks,

able were And we what we we it conviction the were bring X our reminded continued Thalassemia Just are once reinforces patients. the sickle both community. engaging patients. Sickle where convention, Cell meetings for the with attend to and about and to we recently, for for Academy significant SCDAA aiming Conference journey cell the to difficult At annual again

are novel Turning in function, that to to this with we improves VOCs anemia, feel potentially our oral and steadfast how disease, sickle therapy cell continue particularly to community patients by commitment and impacting events to the recent and be respect saddened deliver fatigue. a

the improves hemolytic in in as that Phase we data other generated study we as data excited anemia first-in-class disease. the cell cell are living positive II health positive convenient a potential patients UP sickle best-in-class that share we option well for of and pathophysiology, for the RISE mitapivat the the to with generated common have Given red a portion treatment blood about become

this of III we UP of the Phase the by year end met very the recruiting study are RISE a year, last just over after cohort. we completing began announced As we our goal that week, in enrollment pleased

per versus As annualized XX baseline a reminder, primary trial gram X the subjects in sites hemoglobin an across endpoints, There need of in rate at as compared believe cell by response, study making the size over forward next approximately of Phase potential this average cell with randomized deciliter are sample has improving Phase a hemoglobin placebo the planned globally. crises concentrations pain XX sickle We disease mitapivat XXX mitapivat weeks III X:X feel to increase to least crises. Driven defined of III by XXX mechanism unmet sickle that to from the of data look year. reporting high to pain a has anemia, action, patients top and reducing differentiated and line we better. address X

the all For mitapivat of beta or of Phase the both a become alpha we oral therapy deliver all thalassemia, generated subtypes ENERGIZE adults aim thalassemia, thalassemia potential or mitapivat for believe with approved in first to for on we the non-transfusion-dependent data Phase with Based subtypes. and and study III to all transfusion beta levels adults positive all thalassemia, in of has alpha the III ENERGIZE-T study of genotypes mitapivat in foundational convenient burden. including option transfusion-dependent thalassemia, and treatment the

working to who X of filing from with top to adults with deficiency, end expect regularly continue the thalassemia. In pediatric transfused. year, are we label an children this deficiency, that of are to study mitapivat progress ACTIVATE-KidsT were by all includes and sNDA We PK data the Phase line file pleased the announce subtypes PK in living of diligently seeking a to

for trial III in report this it's completed because, study We first and importantly, data population are of Agios. this of to first, pediatric the is first proud mitapivat the this Phase study

were meaningful met, at believe we with pre-specified limits hemoglobin as response, once to least observed response parallel hemoglobin forward regularly the adults in look endpoint PK transfused. data normal weeks we more with patients safety the in cell was program, reduction With next these enrollment primary transfusion-free make quarter, year. In readout who who progress clinically transfusion III trial, X an defined the percentage a achieved across are made statistical not no the those mitapivat the red that for this concentrations criterion deficiency the given were and after or response, second, anticipated for within who also advancements with transfusions While results development safety the results our including tebapivat continue those blood had a mitapivat the of from transfusion. And to mitapivat normal in previously Phase program. ACTIVATE-Kids achieved important we with consistent we in profile completed

cohort third followed patients and This dose of XX across a evaluated transfusion sequentially, dose of X-milligram independence, with period. endpoint was period cohort dose lower-risk for the period. The begin will XX at enroll are which by be will XX-milligram level cohort, XX dose. in MDS Phase X in milligrams do same the First, the Phase MDS. XXX-week will study the we is There slots cohorts, XX-milligram will risk previous enroll label II IIa the and a pauses and we of we lower defined XX-milligram milligrams, that once remaining enrolling without at during transfusion-dependent cohort that transfusion-free XX enrollment weeks individuals in patients patients of core XX-week XX study. Each for X a cohort initiated dose the the those consecutive for study filled, meaning than levels, as core is for we extension greater XX will open total then tebapivat the XX this least be in first each milligrams, dose each study the the open between of will primary

MDS, due we first which endpoints of for addresses hemoglobin program, disease further additional the MDS oral underscores approximately all to to the provide this FDA delighted Secondary and we patients were with drug rare for from the we orphan XX% will milestone for of of in bringing measures aim deliver tebapivat change once treatment cases. Also affects approximately erythropoiesis this patients thalassemia, that XX,XXX in MDS. timelines suffering in XX,XXX to new the accounts to enrollment ineffective expect additional and anemia. Similar to receive EUX treatment of This along. this safety, importance anemia lower-risk is U.S. oral designation include and options quarter, and to to therapy

to forward, to the now annual over American Society on Looking I to and of the meeting will X. With the we the excited ASH Hematology, forward Tsveta. upcoming of of November that, be abstracts look turn accepted call attending publication are

Tsveta Milanova

Thanks, Sarah.

reminded for the significant thalassemia, across subtypes. value PYRUKYND unmet and all may As we disease also in we prepare thalassemia need a launch patients in are bring the potential this to of

adults ASCAT powerful is X,XXX X,XXX at meetings many U.S., with in diagnosed diagnosed adulthood. conferences We at continue before with and in to thalassemia connections of approximately rate patients validate PK community in than approximately local patient diagnosis non-transfusion-dependent. this we whom through deficiency, is the prevalent The the patient like much more selected experience high, are centers. Thalassemia

the the therapy. claims need known with year. health important for relying of burden launch approved non-transfusion-dependent of All to of morbidities, potential next reduced of serious of high PYRUKYND become with and rates especially positive is life premature from to forms most higher in heightened thalassemia patients with death. on The bring disease, risk PYRUKYND quality patients subtypes. care our address a commercial the data for for all is enthusiastic supportive thalassemia availability preparing ENERGIZE provides and our disease are is data U.S. significant. Galvanized potential ICD-XX launch preparations. Treatment especially of the patients which high of codes, studies, thalassemia limited, care Given by cost options for are associated system, clarity these this a and to about actively unmet organization ENERGIZE-T first of therapy the the and We're the

Our progress across launch areas to of make team continues preparation. X

address initial First, analysis to adult done, insights work launch execution. we and market PYRUKYND's and XX% launch market population. will our data-based estimate that inform we and extensive the patient continue refine our thalassemia focus we've conduct claims of on the to Based targeting approximately research further

as already expect to segments non-transfusion-dependent health symptoms living for patients, as the first. patients well complications have experiencing due with considered include fatigue. be debilitating or These regular contact to therapy care of patient system who who patients with disease are their already transfusion-dependent We are

the actively of in aspects across is the thalassemia. deepening and patient diverse segments our field, team the Our insights different multicultural engaged

our ongoing areas. been campaign focused education state on has disease X Second,

hemolysis. First, we erythropoiesis are including the disease, the highlighting of ineffective pathophysiology and

has the the misconception we we it's both to another engage highlight thalassemia with and needs. To for we to with as who of important which we we from campaign, thalassemia. this subtypes, tailored image and valuable invite hidden pathophysiology and Second, been patients and patient's to at relevant recent powerful disease are Rethink how all the to understand of campaign Thalassemia The of the importance positive treatment the patients for of complications, believe disease individual very can highlight but it they burden the words in physicians. They feedback monitoring find management. and inspection together, that complications rolled particularly third, risk. And continue that suffer connect they striking disease pleased ongoing to comes and less closer headline out The and depicting management this have the are understanding all the and not across disease example patients the importance of content, of risks both months. physicians, to from non-transfusion-dependent share also long-term bring so the when only frequent patients to plans physicians disease from the and education monitoring of be

Gulf preparation focus we thalassemia our fourth, trials. We're as and achieved commercial on XX,XXX in Our home our the in treatment access strong market launch a some medical approach team outcome steadily for of in I'm state Council, disease as market. is GCC in being launch the access of the we to a team thalassemia. Outside Cooperation on but deficiency same payers PK the third this with larger area potential of to possible. And rightsize proud with are the and our for has centers rare additional part leading of broad patients and U.S., keen aiming teams of organization is already region, building team launch or engaging focus capital-efficient adding PYRUKYND capabilities our the as region discipline the for the clinical the with is a successful in education. and were still expansion approximately

this by we're to designation GCC pleased for the have prepare region. are regulatory in the NewBridge a optimal to strategy of in quarter, last distribution commercialization thalassemia, PYRUKYND we a Pharmaceuticals delighted with potential and the announced We're NewBridge to an Saudi for agreement GCC FDA or SFDA. PYRUKYND we entered for granted As collaborating breakthrough medicine actively into have develop secured to with partnership, commercial region. Complementing

access the reminder, sector. forward regulatory with in local private at GCC a elements by individual health authorities, set collaboration institutions negotiations NewBridge. each with the level, a access We followed price with As path the these countries to in navigating and usually of look begins

launch turn believe the to in foundation we a and a quarter form, This net PYRUKYND strengthen XXXX through we cell In disease update including Next, underserved in a of of XXX in third let enrollment third provide the in brief therapy have XXX in of total quarter call XXXX. new XXXX, from patients we In a deficiency. $X future in launch will Cecilia. million maximize revenue, versus these the on generated current I to prior and a inspired XXXX, to the will me which in firm therapy. We million quarter. sickle thalassemia populations has patients prescription the PYRUKYND the current continue on potential provide XX X% world. With of XXXX. In completed of into energized around second the to the potential by capabilities the a U.S., increase the PYRUKYND compared closing, of patient to the in PK over that, net bring U.S. are to $X.X translated launches quarter

Cecilia Jones

Tsveta. Thanks,

we will XX-Q, this later Our financial more can morning be in our will results be the included in release press detail and filed be issued XXXX third today. found quarter which

highlight to Let context me some provide now take a key few moment a and points.

to revenue XXXX XX% gross-to-net and $X XXXX. with annual in rare increase to third quarter be net XX% is range Third disease the million, compared XX% other to of been an launches, quarter PYRUKYND was expected of on an has basis. the Consistent

of primarily we which upfront strategic expect was with quarter, with shift primarily prior the preparing third million Alnylam, decrease PKD have of the million quarter driven was third million R&D sales year. SG&A see towards thalassemia, line XXXX. by quarter, license payment the were expenses recorded a year for XXXX. and the decrease compared associated activities in growth launch the increase an million forward, the prior for $XX.X of were the variability we $XX.X aligned in in Going PYRUKYND of compared million. for muted agreement in $X.X with $XX.X quarter for quarters. Cost for our third driven This with of quarter-over-quarter was quarter. recent in potential to This prepare approval to the seen expenses potential revenues, an increase by the in as the to million commercial-related for in $X.X we was thalassemia what $XX.X

Agios in oncology P&L. in Servier divestiture of a payments Importantly, as in approval announced total Agios' income with in XXXX billion as a its in include this the These $XXX with of we $XXX million from of million payment agreement a milestone quarter, Pharma the FDA under from purchase $X.X vorasidenib May royalty were received both XXXX, of payments Royalty following business the well vorasidenib. recorded other payment which connection in connection

As a net marketable milestone than U.S. billion. annual on securities royalty greater of equivalents a approximately and of cash vorasidenib X% payments, $X reminder, sales we the ended cash, Agios quarter will retain $X.X these with billion. Including

expect remain on by base internally support expand We the discovered provide prepare cell both to financial potential this with of and potential PYRUKYND together we cost a our existing disciplined prepare pipeline proactively and through and opportunistically and focused sickle deploying shareholder value, revenue our balance, cash allocation advance disease, externally future will programs in that independence product assets. We approach PYRUKYND. thalassemia including for managing launches and creating launches anticipated to interest income, as

balance toward term, As in value-creating to turn will call now we strength. I am of Brian. that I a enable continue milestones position execute the from additional the sheet to move over our us potential near back will confident to

Brian Goff

Thanks, Cecilia.

few I we at for marked want been progress words. Before to by a line Agios. questions, with conclude exceptional open has the XXXX

year in momentum the deliver have strong allowing this final our priorities position, months financial very a beyond. to to and continue the great of We and maintain key us on

a treat with of to and immediate firsthand for a translating mitapivat thalassemia an elevate poised new communities consistently best-in-class first for regimens to disease. cell indication into to In that with represents we We the With is treatment multibillion-dollar meaningfully critical become and multiple diseases. for potential. care need from believe these its significant mitapivat patients opportunity these sickle indications, in approved there addition to PKD, is a standard this, franchise option hear

drive these In opportunities a significant there providing forward, with look please to all addition programs and for create focusing trajectory high of future where to call stakeholders. diseases. With rare value need for mitapivat, like patient Operator, strive the I'd change on we is questions. We impact to as open promising other the additional line. long-term that, us the to forward our to the and continue open to areas clinical we

Operator

[Operator the Instructions] Our first Scotiabank. from Greg line comes of from Harrison question

Gregory Harrison

Great to see all the progress. for Two me.

And how capital allocation guys vorasidenib that First, you maybe to speak you can cell, can now or the development about find business respect Maybe with has are thinking on powering And monetized? a VOC. appetite portion the on been then largely trial? attractive there's and area to if comment of expectations III Phase you sickle you for particular for complementary?

Brian Goff

Sure. welcome again. Great And back. you Greg, to hear

Gregory Harrison

Nice here. to be

Brian Goff

Two for over Phase III can allocation Sarah parts. take then handle expectations. and RISE Cecilia can the question capital UP

Cecilia Jones

Thanks, Great. Greg.

of balance thal to shareholders. So have doesn't today we're, rare, and very in criteria sickle that, MDS in sheet the internally regulatory well. And pretty that. create sheet have hear priorities, value continuing our for our we're balance of as we the amount have in will a in advance of and/or think being change And obviously of same the a about path and both look the how allocation, course, externally, and bit then we about our at past transformative, We more strong which clear things pipeline, terms you'll the that hopefully launches course, pipeline, cash of in cell, the expanding disciplined for we of we little with then proud potential of capital terms be terms now. have

that changed. hasn't So

in we And agnostic We continue we've that. to at expand that. how different said terms modality we'll to look of areas, and we looking are at keep want

Brian Goff

we the of track, has you're do the lot over team excellence III XX for UP. were proud Phase how announce for fact that pleased about want profile, by study were turn complete if very the trust about the again, consistent to with executional team. And the lead the that just today's think enrollment in call, I'll says Sarah community in to and really executional saying RISE able it's a importantly, us. excellence. been before to I months. even but this And Sarah, then I I And from of the am keeping incredible it the And We

with Sarah, as So how that that, about a setup?

Sarah Gheuens

the for question. Thank you, Brian. Thanks

So yes, our which UP of proud accomplished year. the for We of team. so we milestones indeed this enrollment, was complete very one RISE are

that the finish excited and are to So looking end. to we very forward trial, now are get about it to the

for X-year Phase III, expectations duration. the a so it's So

have is We you primary endpoints, One know. as X hemoglobin response.

is hemolytic can and with cell one And goal are the other reduction. of or the slightly sickle on difference. vaso-occlusion disease, a powered have to try cell course, XX% to detect is, anemia that The totality meaning drug deliver more, crisis hit to endpoints pain that able that both sickle to so Both to be of improvements.

this about the trial. excited are looking of very forward the to end we're So and

Operator

Our line James. of come from will Raymond next from the Brill Danielle question

Alex Nackenoff

for This is Alex Danielle. on

will Another a in both think like success support RISE Or could response endpoints on possible regulatory bit on talk Could the it a fatigue hitting on about secondary one another endpoints? on study? hemoglobin that need your a is clinical For one example, Do regulatory you just endpoint filing? primary UP you hit and to RISE you little UP. filing? support

Sarah Gheuens

Well, thanks the for question.

one did X focused for I endpoints, provide and on primary endpoints relevant choose of the other information hemoglobin, have primary one being the the we X they yes, then totality disease. mentioned can as just We the because So VOCs.

on one still testing, hit we other would but to which further benefit. on for So us able not in endpoint, would a to endpoint, secondary which situation an allow have clinical hit would be alpha endpoint opportunity to in the we only to look transfer

deliver have drug meaning our those plus anemias improvement fatigue. then studied function. of deliver as to that on that And One try been try is and we the different story, improve an PKD endpoints to fatigue. highlighting but across indeed anemia, we've hemolytic you with we hemoglobin know, on able also feel and hit in the that thalassemia, We to to always hemolytic

thalassemia highlighted X-minute there an data test. in Our walk was improvement also that the

provide clinical cell is benefit regulators end in regards similar sickle way And the patients. is so that look data study up can of multiple regulatory set trial that our is disease for ways a RISE at we clinical to package. to the In to UP needed speak for approval, the to the at what

submit, always that Of -- approval. a benefit ultimately regulators course, when profile we we and we the decide believe that risk but do there review approval, that it's the on is supports

Brian Goff

team to didn't poster I everything a not the day excellence, change From is, would in XXXX. other like Sarah add, actually of the way. we again, executional but expertise the point in and was just through the went that also outlined In this scientific this from echoing up been it's design. just words, set that X,

Sarah Gheuens

Yes.

Brian Goff

very of design. the disease in cell therapeutic other a the X, fact that's in important, sickle proud a evolution recent And hemoglobin plus intended of context And I had design so-called we day with the we're was be that particularly to options. think from that

Operator

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

Gregory Renza

Agios on how is into happenings the sickle in the mitapivat alluded least community or been have Oxbryta on just comment the feedback, that to given on the withdrawal you cell. providing trust touching sort you mistrust the Congrats. had can progress. curious track of in from the And and your regulatory I'm if earned sort trials and risks the patients? trial you're just for at that for you Africa the that, running you the just with are to highlighted that that doing potential value to any the on communications just detect potential the the that of on Brian, general community you've about that Congrats execution the go-forward secondarily, have plan incorporate with development and to program you've just with and with the commented also there. What respect site?

Brian Goff

regions, been back but when commercialization, example of the the the is, future. And involved get just well. community. results we're to of you've trial and have members an what point the what not Greg. of And are, very an it's you community, trial, to in not lot, treatment the all I'm beautifully. And the Yes. that. options was do input and recruited criteria, importantly it from of been of got Thanks community was Disease chance and always echo a available and designed important options available example sickle how challenge. And on that in anybody, certain now gone drugs It's question community we to we seen And Association to that the It RISE to the then And the we have way to in their cell hopefully, available because then we the touch and that goes proud large not what appropriately through listen, hear for lot. deliberately recruit warrior through very again, an Sickle that I cell very exclusion Atlanta. as a that the we is, That's at of in attended America I design, regard And just ask of treatment think Cell when always importantly, a what's meeting has they guide we just surprisingly Annual how journey limited think, looked maintain trial. disease has certainly about others their the high concerns mind, a for UP and to the of in leaning to real that a Sarah sickle most us with team us on community. been inclusion, the

to the on about message main without the the We based their think We I So that on we listen. that they've provided. is do listen bias sees community trust make sure and be we try very we visible. what input fingerprint

Sarah Gheuens

then forward And add regards in with Phase III it's -- going to so to to Yes. trial, that, trial. the continue a to we

we for to a devastating we'll this running a do procedures suffering our it's continue to on as in from do with sure disease. this along. are and go trial So cell regards we disease, tough of trial all risk monitoring patients sickle that carefully as data trials, of in which evolves. And standard to watch disease to specifically we We continue ton potential it's and obviously, well a of execute deadly make clinical comorbidities

unmet there's a like trial So speaks the this when it this, run population. in but challenges always you patient to need

able and the totality need can in meaningful to So such be these challenging community the difficult, patient to and a that patient treat for we really of disease. are that while hope trials there's this be unmet population the deliver drug

Brian Goff

in multiple therapeutic Just reminder this should it or that community serious recently from a is maybe the the also advocacy group is of one for I options. call plural this of how sobering on warrior We progress the crisis And about that community, it And is were just community. cheer cell to the this And a And of point form. I drive them part X desperate call, talked members is had there X options. new had for a sickle were walked among just disease about. of more home because and needs and disease a members with that with Sarah that, actually we on be away crisis. always how disease everybody

Operator

Our of next line Fitzgerald. the from question will from Cantor Schmidt come Eric

Eric Schmidt

your of month what the relates kind have Oxbryta the with to and about scientifically sticking what regard and ago, the or learned have study? success or it mitapivat of you in drug a scientifically thoughts topic of how or this ongoing you with mechanism to maybe do that doesn't relate unfortunate chances withdrawal influences of it so sort of Just how of

Sarah Gheuens

you as the feel like for question. this sad I action different a should happened. patient start Brian's we know, earlier sad options. any really because So mechanism what of Oxbryta has very Well, and -- a right, has we And thanks really point, feel barely to circumstance, community it's about

as trials to action. clinical set see a So differently very this. It different we're mechanism were sad The of is well. up

approach multimodal think very declared. -- our is publicly been have happens and then treating know treating beyond our I drug works to on sickle has what that So that don't differently disease. But we cell what potentially trial, focusing --

these X-DPG ATP, and hemolysis we and of component. anemia increase sickling think thereby interplay important be So and X, improve reduce can the avoid both we to and components to do and

was hemolysis different translated have into the data that has us with then reduction. -- of into very a trial so of very trend that dataset positive that a with which also very Phase action seen also, robust, course, translates improvement and demonstrating hemoglobin mechanism and that on and then we for trial far an of observing than VOC placebo-controlled So action a clinical clinical mechanism Oxbryta, II

we to III. progress excited Phase drug in the this to So forward -- are continue the

Operator

next will Chris from Our come question the from of Sandler. Raymond line Piper

Christopher Raymond

Oxbryta some I there's to with Maybe the then a question terms debate has community. and you investors know in guys. on good sickle talked lot that's raise real But therapy question and come you is changed this sure of bar But regard it another cell do that up perhaps if to this? a answer the lowered how just it's the you debate market. situation. engaging a has Congrats here. regulatory on There's I'm another as about actually made the to not that perhaps have with the another the bar perspective sense a

this? Just might any be how for sense regulators reacting there to

Sarah Gheuens

because like Yes. presented of plus a that development And get-go, think that upon with which so regulatory think trial -- a III. From approach touched in clinical we poster Oxbryta the pathway had thanks I the than was the Phase it, a different followed. we design XXXX, Brian for question I very approach that actually

accelerated and the demonstrate time then an had had further of studies to benefits. clinical improvement by at submission the demonstrating Oxbryta follow-on approval hemoglobin So

trial that deliver cell of when end the to can pain this at designed trial clinical and treatment a first Our -- goal benefit, is from plus clinical meaning the reduction. to sickle hemoglobin crises we speak meaningful

to So based not have clinical design our approach our regulators. our happened, what or changed the we to on approach

after regulators to mentioned ask I you it's ultimate the with benefit package submit, first always decide As review data and us a they ultimately, they And the earlier, for questions. get on package more the risk. they'll any then collaboration

Operator

Our question Tessa JPMorgan. the Romero from next from comes of line

Tessa Romero

deck X patient little focus your of a So specific quantitative switching this each are populations patients across of slide to of health to able how you part Are detail thalassemia, around provide initial for these provider outlined bit morning groups? in in in seeing patient bit that groups And gears your little how PYRUKYND. out launch a care more the these numbers their are you target epidemiology mentioned? key breaks often you key terms X generally several these

Brian Goff

in we that to about sickle but even questions love consistently a disease. scenario potential start that saying launch looking launch XXXX, Tsveta closer cell smile because Tessa. Thanks, thalassemia, where in messaged We're potential you're with to the by on this the forward very XXXX. excited just we've is making potential going we're I'm

your let So to take going Tsveta on over questions. I'm

Tsveta Milanova

the question. Absolutely. for Thanks, Tess,

excited thalassemia, are indication indication. launch. rare year We a with main potential comes reasons. truly, it having of actively approval it's beautiful for and an for When think It's preparing an about X thalassemia very next when for disease to the commercialization we

burden cell that comes we about actively health are be focus with that there them system. true our will engaged engage resources with initial of X,XXX effectively deploy and high codes The the which first about X,XXX efficiently diagnosed because lot going I'm there, launch very, engaged with patients very talked U.S., when that's unmet available, health system. the and these ICD-XX thing but know to care to known because is to thalassemia underline and And that in a the to it that's us we sickle preparation. We and know adults are that that for diagnosed need disease the allows are we there case which here is launch need patients very launch. groups our believe thalassemia and patients physicians patients to our These the to as unmet continue as these mentioned, continue initial X XX% on are patients both well. the known patients efforts be care of for we focus disease. When it comes and The will for And the first, in are educate our you considered to to prelaunch of patients. are also

the patients health care can a have frequency, you with say their basis. with transfusion-dependent is one their seeing engagement they are more hematologists. first their transfusions going monthly system frequently that transfusion who depends the is or less, are but engage and That probably on for on The and an

management patients non-transfusion-dependent both monitoring The second regularly will and of are already we specific the the be as they for claims who are debilitating are for developed there, group monitoring the well. patients require treatment who as of these further seen and And have It's the depends potential patient their considered frequent patient comorbidities. They and management. the part patients comprises they fatigue. seeking population, population from initial It and be which know that third are on impacting non-transfusion-dependent life profiling our the account and patients data well. relatively will option and XX% a the daily frequency experiencing but

patient We this well. progresses, provided but on be specific we'll details additional as providing information launch haven't as population, the

Brian Goff

time, because And I'll to Tsveta, a disease the sometimes a is just it's continuum. is point launch it's that odd her rare not in remind too, term of mean, with sort us add, experience I all launch first of a

of working be in to with for and constantly the with segment the disease segments, team on sequencing, sickle we'll progressing expect et keeping cetera. in long segment So well. if hopefully, say And is even cell that again, on this X, it we X, continue. thalassemia term could And we ahead those then Tsveta the guidance as

Operator

Rao from line from of Our the come next Cowen. Divya question will TD

Divya Rao

it Kids Mark. of timing for we dose. IIb be relatively data you discuss for think for least data initial PKD? but And that obviously the would are what One, our in about talk have a than to question top little the the the about you're X-mg modest at MDS, bar second pediatric to tebapivat and or initial on Divya design now the data showed the levels you is that then thalassemia, how trials that at is with it now initial the Could the in looking for you're bit line able from was you dose success stay Phase of trial This higher dose is tested? on thinking signal,

Sarah Gheuens

Thanks, Divya.

some indeed, So thalassemia, PKD are first able to very in trials, pediatric children of demonstrate transfusion yes, so trial proud that experienced independence. for pediatric we the which we were the

So pleased see we're definitely clinically very that to meaningful. that's because

the the we and timing population. the so now thalassemia benefit-risk in now the are have adult design of For profile the we -- patient also trial,

So have taken approach you similar PKD. from as a can what we us for expect

point. In your we we was pre-specified our did meet had at of MDS, to to for in yes, in the We have timing initiate to XX% Phase population observed in so regards have not there what would And gone independence IIa into further which -- in we had different that detail when more learn time the on the later did volunteers. come point, we patients we of than criterion PK we observed transfusion did patient healthy the we MDS enrolled. that a

independence feedback in hopefully on on clinical that based that received. So And the that, the doses and to able as transfusion independence Phase testing IIb X that now are doses, we we we moved forward led higher are have or focused strengthen advisory observed for trial to IIb. had be transfusion observations changes Phase with our of and course, more to X-week higher you -- see,

more So and come. to are excited progress that to trial

Brian Goff

growing multiple in are There potential and is in with And who fact sickle very pay I disease a others And mechanism. a similar folks very It's will very attention market. high way to proud in need. MDS. and we're answer. unmet cell morning that be options it's of continued note the took of in we know excited Reblozyl have therapeutic differentiated a we progress market the we're very that this the about see to the that rapidly this we

to start step important able to IIb. an be is us for the this So Phase

Operator

from come Bank from America. will line of of question next Alec the Stranahan Our

Alec Stranahan

from Two me.

could attention right? as direct would First, sense to shown guess clinical its I have it say then where prep, seems And ENERGIZE-T, or data likely what given line? launch as there? the readout some And on said, more would next online? from and fairly thalassemia unmet efficacy probably the maybe And the people's access readout like expect quickly I come mitapivat's on it to of most ASH for you profile wonder aware any in we should beyond upcoming next a was venue, full is update? the in are if top additional the work is the you a do payer continuum, you Brian need how payers

Brian Goff

Alec. Thanks,

And ENERGIZE-T. over so can Sarah take the launch and with can question payers the with and Tsveta start on then the thalassemia

Tsveta Milanova

Absolutely. and good Thanks, It's Alec. a relevant question. very

So from preparation. launch from seen as a is we perspective, access and in market our you've communicated payer the probably pillar what a today, core the slides

thalassemia market rare and Our look There state relatively with engaging is a disease. put disease. disease is you of team access education. When awareness a at low you it, already a on on the payers lens it's payer

the a disease. the definitely are side well. that it we positive expect But we on as to educating don't is rare that disease it's and radar high of on the on payer for management So be

access is I excellent from for launch. team our deficiency. in So must the we've an efforts outcome say PK preparing had

We very moving strong have forward to we and team and progress the thalassemia. a success continue see same with

Brian Goff

Great. And then for Sarah ENERGIZE-T?

Sarah Gheuens

Yes.

secondary plus endpoint you release, we've the that know press our in -- we so of ENERGIZE-T, our For declared primary endpoints. one

endpoints expect as us analysis overview. the details the key well around Phase to can safety treat to you we do to with of to from upcoming present Indeed, not typical hoping at in able medical meeting. an be our available So intent is and how ASH III readouts are we yet. all secondary currently

X. November on will released So be abstracts

So are for waiting as well. we eagerly that

Operator

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

Lydia Erdman

Salveen. is This progress. the on Lydia all for Congrats on

ones Just X from us. quick

sickle you what from update? VOCs? tebapivat, And remind of data to details cell, enrollment complete, included Phase when just on then be that are baseline you now us So just on discuss able you number with on any is And to provide this share just can IIb? the will plan the

Sarah Gheuens

do top to sickle the we characteristics. release So present results. we not everything present tend typically for line when We baseline disease, cell

now we our we corporate the IIb, year. nothing to data full obviously provide for So milestone come to met Phase before in then tebapivat yes, regards have And this and, readout.

So more to we the forward the timing and detail on are year. looking trial next provide rest of

Operator

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

Andrew Berens

correct, think XXX, in a how but the you to malaria was touched I you think investors IIa trial problem remind we on sites heard African dosing somewhere? a you might have then Two and before If related hit some that's see presented dosed the you in And think of we've headroom than I Phase you sepsis milligrams. how for And trial patients with there of much On that sites. higher this can the on conduct some and a me. bit, think in X was this me those that problems DLT? those was data

problem of that those sites. I think occur? And at done on your what's some you a to doesn't ensure is color similar trial can us being give some

Brian Goff

asked more to I'm say Andy. activator. I want you very did And IIa? say with have our practicing Thanks, new you first to to want tebapivat. potent on the in to start going we're Sarah, of let for our and PK Sarah terms end that go, XXX we INN the but proud second We're of

Sarah Gheuens

IIa Phase the the dose. So tested X-milligram

And so that higher we IIa. did not doses test in

doses the higher yet. XX expect hit regards of portion don't the milligrams. XX gathered is to haven't that know, is the tested I when the XX, we now because IIb to right trial, In and DLT, the in are I which data being So

then we to and explore want that anything if time over accrue will observe us dose. not data we'll makes the see So further certain a

in the certain more sepsis So trials, asked come malaria endemic you for and so world. regards mitapivat and observed to In malaria to that. on of what the Oxbryta is in regions

well, distribution part very geographical of have the trials within Africa a good UP. have across board in conducted We in we RISE but as R&D our

are on Africa. on we The have not hydroxyurea, that observations that. instance, So in are observed that about for happened happened have observations that been Oxbryta have excited

So the we'll trial accrue and I data. will over observe our final time we data think have when

Operator

will turn to concludes final our this Brian over Q&A. And for remarks. I any now it

Brian Goff

long-term and at in much, transformative Agios. is today's truly significant patients All this that and very poised lot, to you believe to a participating exciting right. for Victor, We we're thank deliver is Thanks very therapies everyone, value for time a create clear for new shareholders. It call.

forward again, you thanks speaking to soon. look again and So with we

Operator

today's conference. in participation your for program. the you Thank This does conclude

have now Everyone, a great disconnect. day. may You

Agios Pharmaceuticals (AGIO) 31 Oct 242024 Q3 Earnings call transcript