AGIO - Earnings call transcript Q4 2019 | Agios Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to Agios' Fourth Quarter and Full Year 2019 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 Holly Manning, Associate Director of Investor Relations.

Holly Manning

Thank you, Operator. welcome Call. Fourth and Year and everyone, Conference XXXX morning, to Quarter Agios' Full Good

access for Medical Corporate Bowden, Financial and our Andrew our With can Dr. section call Darrin Chief Senior You slides with Development. Jackie of and the Vice going today Officer; Dr. Executive Officer; our Investors Commercial Fouse, to our website, remarks agios.com. prepared me Marketing; Chris Chief President the today's on and Global of are call Head Officer our Chief Miles, of U.S. by Hirsch,

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

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

Jacqualyn Fouse

where more Thanks, I've for with a in fourth XXXX the I XXXX. the was difference last excited XX to joining are call. lives and go meaningful company everyone, the quarter year Today, on the make our Holly. at patients. I'm and the work even ability does as role I at our spent took of Agios into we we from CEO on business the and several successes further the time, our my morning, results evaluating value learning ways as a the Good and supporting colleagues, thanks ago, listening excited of for strength build that truly the clinical company I create portfolio patients focus confidence differentiated. commercial notable past and more programs for and to to firsts our months our quality the execution of stakeholders. ever is science, and of impressive of all than preclinical, about of our the team and have and our about

X this is our out end what molecules next have for focus which will billion positive in and and excitement malignancies, rolled Agios, year progresses the solid research we could and like By generated X company in product marketed as the our more within diseases. internal entirely at cash frame. over be have of genetic three expect new discovery clinic, XXXX four engine, six with areas vision least or that X time January, years. discovered It the We'll the of at XXXX, by strategic the six we and molecules indications sharing first revenue hematologic our from be look time portfolio the at generating of medicines, all in confidence company tumors across developed least our we flow to rare will $X the

opportunity our plan, built vision long-term to This think base we an potentially optionality tremendous I the and programs. was have preclinical also our our of Within our clinical achievements existing and XXXX case and exceed goals. foundation late-stage on

already tumor solid to malignant our programs. and across rare ambitious Our disease underway we the set for XXXX objectives XXXX path are steps of hematology, on with genetic set an

the of this fact, X-year In vision. our one aspects is most compelling of

discuss over detail We next in My our more milestone and moment. the X year a every occurring in years. have will objectives events programs significant colleagues

his Bruce, up, together Dr. Chief Agios with I'd welcome like wrap scientific last month. Car, will Officer, our Bruce of I be ushering Before during programs him results look and join forward we're having achieve pivotal us to excited team, on next leadership to who publicly the board future the on joined Bruce new to wave to us research our We from help this vision, have clinic time. to calls. Scientific XXXX

With that, clinical call regulatory updates. Chris over to our turn now provide I and the to

Christopher Bowden

Thanks, Jackie. meaningful multiple solid progress tumors of hematologic and these therapeutic across early and each our and malignancies, make programs, rare areas. we spanning clinical late-stage in XXXX are to XXXX, aspirations aim Underpinning genetic diseases,

important where start across diseases, anemias with activation we hemolytic I'll expect in genetic to rare our have for milestones XXXX. several different X PKR program

initial Our which potential disease-modifying enzyme is area focus mitapivat chronic anemia, for treatment the pyruvate has kinase mutated currently first where no deficiency a approved and therapy the be this of PKR options. activates has to

deficiency. is of share ACTIVATE both Enrollment ACTIVATE-T end of and and studies patients, is pivotal year. receive data the line in study the regular studies, top do X regularly conducting we not mitapivat PK transfused is patients now in who The closed are by to We transfusions. study in in plan the

forward moving in mitapivat mitapivat XXXX. regulatory in our this in are are patient trial deficiency a our patients. pediatric advisory study advance boards, to our PK we addition, we and in In with initiate and interactions plan ability confident to clinical we expect Through to population,

evaluable the into announced a treatment application and has provide where per hemoglobin benefit study in mitapivat X cell greater mitapivat we of of Phase ongoing patients. XXXX, with thalassemia in response induced PKR. clinical deciliter than wild-type to achievement or to December, thalassemia gram the it II proof-of-concept where demonstrated X of In equal sickle we X potential we expanded activating In disease, therapeutic the of by

will alpha from Additional data June. to including from with Hematology presentation in European patients Congress beta thalassemia, thalassemia, study, submitted for addition the be in Association data this at

pivotal end we a the developing plan in finalize mitapivat to for actively thalassemia, by strategy the which year. of are We

this of pharmacodynamic study from X,X-DPG Health data and Enrollment proof-of-concept agreement. laboratory at in will going expect and as tolerability, reticulocytes is which we Thein to development disease, sickle well, red with a presentation and of hemoglobin, Institutes and to ATP for is such under a at sickling safety evaluate are research EHA. Thein cooperative XX blood goal the patients, parameters, and In levels levels up to Lay study, The we cell including as of Dr. enroll collaborating changes as markets, and study of Dr. and submit well pharmacokinetics cells. Swee National the for

investigational to we a drug Once the in the volunteers mitapivat, in application new next-generation this IND study have healthy initiate the second and PKR cleared, activator, Beyond developed will quarter. has quarter. AG-XXX, a we submit the plan

to years hematologic TIBSOVO while azacitidine number for XXX data focused and induction important made with to settings, of mutation. study study securing combination U.S. We've IDHX development patients. Moving labels over standard IDHX on last with generating AML, progress phase TIBSOVO TIBSOVO care the of the global monotherapy, study approval I with relapsed-refractory Phase malignancies. and The patients the combination standard frontline in combination is which the IDHIFA of frontline continue several enroll will an next The TIBSOVO combining us in of frontline TIBSOVO with consolidation rapid mutant AML and chemotherapy, HOVON therapies. both to III an AGILE largest of reach the allow and in Phase from

have submitted day XXX EU, from for our we that now raised in Association. trial the support risk not authorization TIBSOVO positive AML the Celgene to did IDHIFA the on the the because objections by relapsed-refractory outstanding assessment I In single-arm European the the disclosed Phase application benefit a our list responses BMS Medicines items CHMP day major filing fully indication. in are of address The XXX recently waiting and marketing proposed withdrew the EMA

MAA to to move available to in Our the goal we Europe, through continue patients and our review remains TIBSOVO EMA process. make

in TIBSOVO and I are in we mutant MDS AML, advancing of hematologic recently Outside of arm syndrome development study the the IDHX myelodysplastic reopened malignancies. Phase

of our the regulatory expect indication, have end designation in to arm patients complete and generate to MDS the a the and Phase trial is We to U.S. enrollment MDS. filing goal to increased size received TIBSOVO sufficient maximum the this from by potential expanded a breakthrough data therapy XX of year. I pursue in

year I steps our to of this program. to sufficient We the advanced lymphoma end DHODH by Phase plan study of to the AG-XXX, determine the next Lastly, in data enroll for patients. have inhibitor, continues the

or For risk XXXX, of ClarIDHy showed the tumors progression reduced Phase readout The study death to of drug in mid-XXXX, we including the a disease from submit plan by utility and a cholangiocarcinoma. study application, treating Mature mutant previously positive is TIBSOVO treated of with ClarIDHy that solid III in survival inhibition data, TIBSOVO for tumors, of significant and the the XX landmark IDH at months. solid new improvement these by and we analyses year-end. expected study X XX%, supplemental with in overall in IDHX PFS established data

XXXX. shared The focused January, glioma team we this As INDIGO of currently brain of at in global inhibitor vorasidenib, our in our study IDH is Phase activations penetrant end study. the was for site on initiated III low-grade pan

and cancer solid One other Phase program and with Our MATXA arms in in currently X standard is of taxanes a care AG-XXX evaluated combination study: inhibitor pancreatic in I being cell tumor non-small a lung of other in cancer. is the

in impressive synergy gather see X aim AG-XXX the data next we years, indications. in if to the these populations X to Over these clinical we replicate we patient that can sufficient demonstrated taxanes preclinically and between

call will turn I to our commercial discuss to Darrin now over performance. the

Darrin Miles

XX% $XX and of as fourth our XXXX net settings sales driven million. continued unique uptake launch both expansion in first I'm by relapsed-refractory of full growth in $XX since XXXX and as quarter by results product total sales Chris. base was the continued net to Fourth AML TIBSOVO well bringing to quarter QX. Thanks, frontline the largely the of prescriber of over pleased revenue TIBSOVO, In share our year recorded million fourth quarter, mid-XXXX. we

Our diagnosed TIBSOVO with first regimens market treatment containing show patients and mutation. that relapse newly research the are preferred continues IDHX to AML for most an

U.S. at over the to of ahead, XX% guided approximate XXXX Looking which million, to million our $XXX TIBSOVO an midpoint represents sales increase XXXX. we've of net $XXX range

duration, settings. is We on diagnosed AML revenue our continued roughly half both labels. we're date, not promoted newly by increasing relapsed-refractory of driver of the observing X-month the both to growth newly end Though in expect To treated treated primarily expect are see largest to both team, agent. patients diagnosed relapsed-refractory treatment will growth and across which to average with TIBSOVO, mark this the continue with a by have We impact XXXX. close of hypomethylating which we and a to anticipate combination, the positive settle duration, in

disease. genetic rare to Moving

of Our team efforts in anticipation of prelaunch potential mitapivat the approval year. is next our late expanding

This in we work driving identified marketing and As serious by teams. and existing increasing want a field behind chronic team patient course for support team market already laid to And over approximately we'll of force been on foundation XXXX, modest EUX, this I continue tremendous Agios work we in many patients driving and deficient anemia. our January, the to in identification, awareness success begin affairs our we've global XXXX. our disease who've development in medical XXXX the driven and is testing build PK to improving shared support X,XXX U.S. and the the the more efforts. to thank to at patients

to discuss fourth Andrew to turn over now our quarter I'll the call financials.

Andrew Hirsch

Darrin. Thanks,

to for $XX quarter quarter and XXXX for and in increase XXXX. filing a the offset fourth million, CStone full and the total of can $XX.X was issued payment upfront next financial of Our results be recognition from million week. $XX the Total to compared due XX-K in milestone the revenue revenue I the XXXX. sales million million, morning, decline Cost the year found year-over-year, press an the for year summarize. More in we fourth detail totaling Celgene grew will of of by full sales a million collaboration to $X.X release this million. bringing revenue for $XXX,XXX be included Product TIBSOVO full from year $XX $XXX was which quarter fourth will our

trial the TIBSOVO start-up the glioma efforts HOVON for INDIGO and companion our R&D clinical mitapivat Phase for AGILE combination was R&D $XXX recruiting increase study in diagnostic fourth and pharmacology vorasidenib discovery costs year-over-year increase expenses. required in thalassemia, work, the ongoing an efforts compared The well and the for and III XXXX. deficiency Phase $XXX operating full to quarter $XX research to Turning low-grade activity in frontline year driven III AML million ongoing million largely XXXX, of programs. full as year the clinical for million studies across by as and PK studies in was platform

marketing operations. fourth related expenses represents $XX This preparation were and year million increased activities personnel headcount growing quarter year full the driven in administrative our for of to support and $XX increased by XXXX. for the primarily XXXX, million for Selling, increase over general and to in the $XXX potential mitapivat investment full costs million launch an

We ended and $XXX the cash, cash equivalents marketable million. of securities with quarter

to additional the addition at our and expected that expect plan least XXXX. balance, collaboration under in We this license through royalties, cash anticipated reimbursements end of will revenue, collaboration but payments, fund and agreements, excluding product related operating our expense any

line the questions. open for operator, that, With please

Operator

Instructions]. [Operator our Mohit And first question Bansal with Citi. from is

Mohit Bansal

a side. European of congrats IDHIFA, my understand, that is I on which point? a Could us PKD. at and more Couple your little -- enough could there EMA on make with satisfy is please Great, has the different on confidence help give bit to us TIBSOVO have or TIBSOVO And data from commercial this anything filing versus you filing. then TIBSOVO One progress. questions question

Christopher Bowden

you it's Chris Thank your Mohit, Bowden question. here. for

pretty our with and there TIBSOVO. data approach important So differences we're the set some are enasidenib doing in I think what between

So the from they disease from fully from meet they're perspective, also Phase after being have withdraw the in Celgene questions not the in They relapsed-refractory to to EMA. III BMS decided and a the seeking. study the MAA indication able

readout from withdraw the and then was to once reconsider the they do what application their have IDHENTIFY. So would view they

then the IDHX the relapsed-refractory is the found pretty you was things feasible. to start of early we disease, that pretty And cut one important on a that randomized of a overall. that's mutation So aspect low down frequency the doing in that when trial because out pretty not

going options. in get our have of unmet given strong So population. that in We is need don't patient arguments this the treatment how that data we have and patients there. And we're is have they to other a think up fact the that and framing used the therefore, all, different this these any this argument we're best really set rare terms

risk a real-world of of some European a are been with that mutation pretty than And of outcomes go IDHX from seem that centers, some overall. worse setting we do relapsed-refractory submission we're tap play aspects and have other other that one as in to there think emphasizing terms with well. think important important and number data data mutation data that we there's IDHX patients then IDHX poor certainly, We've benefit. that important aspect is in mutations And pretty prognosis, indicating a in role could able is patients to the the ascertaining and

as So that to European, the that that's and year. that to of as to we a have a important And to guided you not but think that's outcome. we'll positive And through don't non-randomized we pushing we're until we're But it's say we've remain going why previously, see the move decision submit forward set optimistic not challenge a the the expect process. some we data half there's the differentiators. when second

Mohit Bansal

Got it. the is -- you PKD helpful. this on very side, And And identified. you think, then Darrin, have I then that mentioned about X,XXX patients

one is your Europe? you understand challenges much is, please sort see can X,XXX what mark So us you part you the citing versus X,XXX-patient slide? to get U.S. to And how are in to this of help do

Darrin Miles

Absolutely, absolutely.

spectrum this number. think EUX. U.S. So always disease on U.S., actual U.S. ex it's roughly prevalence rare I of a disease is getting half and the and the in being between majority hand ex The -- the half challenge firm of on the with end

our over increasing on last more accelerate to effort the that. successful being able plus. team We've we're is that reason the been field focus year So to

We expect to the the a perhaps to over course time that approval XXXX and then be have accelerate XXXX. bit get -- expect the the twice of to at end to of able document by number to we quite

Operator

question MacKay Kennen Capital. comes next with from Our RBC

Kennen MacKay

Andrew. Maybe then the and one and Congrats follow-up Jackie a quarter for the on for Chris execution. first and

us when could MDS the on expansion a with And presented? time TIBSOVO, there? Chris, the be there maybe For associated I designation little and the filing potential could lines that the on registration Phase for data for you breakthrough also and help bit

Root's Can and maybe and at you and guide to aspirational again. us to decision so on bleachers of And the that little Andrew, components help give put the a figure? the of background love And of bit the there contribution XXXX. also speak, billion Jackie pointing then by us style the $X that figure congrats Dave I for with, out

Christopher Bowden

Okay. Kennen, here. it's Chris

and study amendment amendment that with that the running of the phase to XXX in of sites sites going just in all are through getting for participating moving the us that have there. because been MDS So reactivate that we're important up many in and cohort important

this kinetics well what So as identified doing onto and that's publish time that operational really at and of -- And of being determine the key point. as and a the patients trial the coming line getting part so will we're in aspect when can we really data.

X%. of think will to I are comfortable the around using X% MDS, pretty with people in that the is TIBSOVO frequency certainly I you remind but mutation

optimistic to as able progresses, give of when we we're and close us looking the year that -- sense and doing. that can be year. about as really -- we get data, the around accrue we talk So the some to forward end confidence by we're we'll more that, the which get publish will of And

Jacqualyn Fouse

question. play slow softball. thanks the fast pitch I mean, did pitch Kennen, I -- and and both for pitch fast yes, slow

analogy is guess. good the So I a one,

be, hands market that already. we be programs the on we because of we -- the put the -- So And will the in to revenue, with those we the including now also -- the assets the only things It's but that the to that $X at billion reason the it's by in achieve potential out that see the we why not there XXXX, have either could number we XXXX wanted to the with revenue, whole, put wanted around in are in is around ground. some stakes vision our indications on clinic. least that of the

the clinic. So talking TIBSOVO or about in

de-risking. definition these So some are de-risked by assets of

our it I include and think mitapivat. would for because that sickle revenues clear. about substantial from Most very PKD in X-year from of include does cell assumed it something would late very make in are IDHIFA, just glioma importantly, not ongoing AML. in to some in thalassemia, what in evolution number particular, assumptions there include start revenue potential all When TIBSOVO time the Bristol we contribution with around cholangio, we the and those think the would also includes to have not negotiate of It in to frame, disease, that anything very, that be

Operator

And our with next question from Young comes Alethia Cantor.

Alethia Young

wanted over believe Congrats differences year and since we're us kind from -- two just one about maybe of joined the like a and I like versus just as how on on it other CSO bit maybe there and is alpha Can efficacy in the then since versus thal you R&D of little where organization -- of maybe Agios of Jackie, a different you and and should following rationale ago when you kind Maybe you your trend. to one potential to past the about And you're of think of of hire year, beta sort new was kind the been other? the couple where it's is a think between this thoughts reflect probably about well? any your help the taking weave me months. is -- for progress be

Christopher Bowden

because to why patients, a number to go we would important and be Chris. given There's activating we're I distinctions the reasons looking we're Alethia, terms there in -- we of wild-type in question. we that overall I'll to approach. need that can and who it's around thalassemia we're PKR -- From we outcomes should genotype and be perspective, Okay. health, that our enrolling discernible those molecular some improve first. answer a differences, and one cell potentially the have now see of the think red of come clinical molecular have that's but that say don't likely

have and have generated regulatory alpha they their from this for of data been our both the remain medical the been important think these transfused. important us Those is of are And pieces. because need disease there process. transfusion-dependent fall -- has thalassemia, regularly the molecules not that's key likely well data of do part transfusion-dependent for group underlying out really other this transfusion is and clinicians thalassemia I unmet into us to because of time, versus in beta thalassemia point a clinical at as categories a and on an most differentiation is, distinction high told given in view clinical

Jacqualyn Fouse

are R&D Alethia, so very excited things. thanks for and the about on where question new our CSO. with we We're

point level, call, think are sciences a employees, clinical our XXX group high of or I RDS I our just roughly very those and about spread at like that would group. discovery our we out research of across, XXX to what

late-stage changed. what is not So that -- the emphasis you clinical early and both on that that here and shows have and we early development, research has

we're I to the treat, inhibition look think back terms innovative that IND bringing into that now And bringing tremendous of mitapivat on hematologic file and in along science we've targeting IDH activation we're with soon. and the the to solid at molecule next-generation Agios now diseases malignancies of our go accomplishments. tumors, if PKR track and we those are the science and record brought XXX to we'll

this us Will want move about metabolism, you ways. Yes, cellular the do. on deliver to we're about with talk we? We Yes, think all will. to that's, we that. XXXX of faster? we I platform the vision. when very that on want -- question again, We But And quality. you that back in the do in way. Do a to that focused comes So company to continue delivered in will innovation doing and has I certainly think

One ground up of in this to a with we've the point where of the is reasons put why we we made lot stakes and portfolio. the want are investments to

patients to and think for great some model I next we're things over some for amazing things terrific years. just business position X the a our deliver in

team now almost Bruce internal I about XXX the Scott to XXX scientists Scott XX we employees. Agios' terrific and job. handful call company last we When on a that Biller led did RDS And ago, years him there here has -- had by of joined were that I thanked a think think the

forward base of and and fresh team, prosecute very to that set eyes that on One did things the is a contributions great he things we of to built the bring. some going institutionalize now ways he that experience of and the looking Bruce's science here, much was he the we're and

that is discovered amazing about at our internally basis got. Agios, quite into and it's think more have bit vision everything put put to want that I molecule discovered and -- really out So an to we've that filing at IND to achievement continue eighth to And that do we're on on the focus internally we're maybe go-forward to even. XXXX a little things build we good and a the

Operator

next Anupam Our with comes from JPMorgan. question Rama

Anupam Rama

two Just ones from quick me.

about For And cell how XXX AG-XXX, maybe thinking PKR how pharmacologic you long-term remind between a mitapivat? are you midyear, of any and long-term thinking quick XXX? of about differences the and us franchise the one, sickle And development then with coming clarification thalassemia, a are point. just

So was being cell to release but thalassemia midyear. press at EHA, very specific about be sickle the data noted was

should scope conference point? the think sickle at of the we about of update cell So of sort this the outside

Christopher Bowden

Anupam, We anticipate the be sickle it's questions. make Chris EHA. shown the sure here. cell and Thanks that for will at data

we CRADA stated, a a the with trial, as thalassemia cell The through sickle it's sponsored feature, is cell. double sickle agreement and NIH. So

collaborating be and an we'll providing they if will. investigator-sponsored with you -- So will be it's certainly them study, but input,

we at hemoglobin got So development because the several DRIVE ASH for updated and interesting now been XXX, drug in clinic that's clinical for of provided at stability data years, us with a in now safety. looking then issue we've community and the particularly mitapivat a PK from and

and healthy of safety a and forward for AG-XXX, molecule a should development along and next muster decisions that our So and clinical pass profile the have now other it aspects and option follow-on we how what volunteers development. into think move compelling to wave comes that it make molecule do about

comment then differences some you've pharmacological asked all across we'll these looking to into diseases gets X really think that's that. So that that need to of me And about on.

kinase the missense again. potential action has some have of mitapivat against been mutations the a to in So active drug that spectrum deficiency, mutations broader that hasn't pyruvate the of are

could activity. of the it broaden So spectrum

are we wild-type. to have once-daily other There think potent activator more potential a be aspects some that dosing be and may it of

more like and in there. all be to a way look the and efficacy then decision sickle thalassemia combined convenience whether to it across steps a in a have really there. when as you translate factor indications indications So too to in that what is wild-type given -- could would dosing the proof-of-concept the it point we these if what be does at would time is in potent these a develop what or should daily go market like of time, a are -- What any of And with of follow-on, activator it's we ultimately, say demonstrate replacement the cell some you how X could make decisions, early how you all we start data-driven mitapivat evolving. some increased

So whole a slew of things.

Jacqualyn Fouse

Nice have options. to

Christopher Bowden

Yes.

Operator

Our comes next Piper Sandler. from Duncan with question Alexander

Alexander Duncan

the opportunities? MAA on maximize ask to change wanted EMA. a IDHIFA the the oncology situation ex the thoughts on approach Just you your follow-up question, in with your way U.S. working Does thoughts how to pipeline in development

Christopher Bowden

because XX% for indicated did benefit world clinical this had to frequency the and setting combine our a Chris very patients you of IDHIFA IDH is of and think interactions very could quickly investigators in around with move IDH and molecules, sense potential overall. that TIBSOVO need population. it the in we clinical in the we one. I with of dramatically put don't And into evidence inhibitors with and IDHIFA the with when immediate so if stated change take saw improve those XXXX the therapy FDA, benefit XXXX, we a back that we the here. I'll TIBSOVO, It's and both the patient AML, in that that high outcomes unmet clinic relapsed-refractory

the So underlying those drivers. are

IDHX at the looked chance we world with in submission impact carefully really Do will FDA? encounter of it do. the trial do than is our Of encountered greatest we understand course, to challenges, the we did this that said a that and of have We for The look when U.S. some way hard, we significant feasible. the and and relapsed-refractory take or when, you randomized back patients frontline take challenges use a the very AML in a resources in should parts setting. non-randomized into patients, we single-arm you data other more so in the our we set isn't best

that's up, And all continue when qualitatively, opportunities same the come would we we'll how do thing guess, So focus. to these over I again. way. we this And would operate

Operator

Our next Shibutani with Cowen. Chris question comes from

Chris Shibutani

ask Recalling could questions. we in appreciate more the last concretely, for little I bit TIBSOVO a bit quarter, two XXXX. saw a If year, perhaps seasonality. the first One, more little guidance

year we think and about what particular, guys support this I trend we and sort across continuing through other and follow-up. quarter et that about about then and, I'll you we Can halfway effects, hole in reported programs a little payer donut a trends quarterly saw And how with you patient bit year? category. support should comment cetera, the the now. was the and that, saw a last this be We're have of thinking seasonality

Darrin Miles

this Darrin. Yes. Chris, is

the So ahead overall or would cost set burden. trend in see cost design try practices a check as we as the support our emphasizing assume similar particularly enhanced possible, We've an with year. that in benefits patients given experience with to actually guidance for it that we be to when QX able patient saw we year, last get this need that the of change services in we and potential that likelihood a share, to benefit patient their much patients -- updated as their to may

assumed So we've yes, a XXXX. in impact to answer question, your similar

Chris Shibutani

at the sort you for there kind sense would think that durability any potentially X ASH meeting at available after us a we make we're when prior EHA. that type abstract and data information to is the patients. Give looking to naturally expect the might of thalassemia? of then And the presented be debut perhaps the us Can would EHA? in remind about result data mitapivat there forward just advanced to Great. the able be that of was data number And of any itself? patients I

Christopher Bowden

Our the abstract, write manner spot we the admitted a highest that will getting gives getting chance and meeting. a of in us to

on EHA. details going of before when we'll be that certainly patients accrual So finish forthcoming I they're -- released. XX there. anticipate It become what we'll really get we will will well, get and is comment to in Accrual can't we

you and how and are, patients, they alpha-thalassemia of data, new see And over that to then the some on time. follow-up, the duration be overall sense we'll evolve additional responses give will so able talk certainly hemoglobin durable you'll how data of about

this would in first really data mitapivat So we're the that I time think thalassemia. be for presenting

providing on an overview perspective safety important for since think all those we comprehensive a from intend pharmacodynamics we are So people and pretty efficacy, readouts.

Operator

Our Breidenbach comes Mark from next Oppenheimer. question with

Mark Breidenbach

Darrin Maybe well. two for for and one quick as ones. maybe Chris One

define and -- are successful layered being little for you we'll standard in this therapies, if if disease? maybe Chris, give on cell curious patients and cell would combinations a is color And NIH -- First, sickle could was I you also, if Oxbryta top us bit trial? a the enrolled potential in a of that care hydroxyurea wondering the there pivot trial. as I'm being if in of what any on sickle including see with proof-of-concept more

Christopher Bowden

understand we So care the the isolate drugs certainly some are can, and eligibility and are Other of understanding patient real permitted on that's patients. effects standard essential can really to not voxelotor criteria. Hydrea like others as would drugs want Hydrea, and because that we while much mitapivat an of that be as be like for and

down some what think really proof-of-concept to declare I is see to we proof-of-concept to need aspects. the really boils key data will

discern And are any out period? like? cleanly is been the X,X-DPG can What of anything, the X-week we those some patients able kinase tolerate dose One drug in volunteers of what the demonstrate effects to cells how doses if deficiency does with profile And over well highest and X demonstrate if happens lowest see effect? and And the that Do cells say, did able changes. can to differences, test? related hemoglobin? we terms between patients to safety? define healthy dose oxygen pyruvate curves, from in dose/exposure ATP in changes to we red we we've over that, them that our importantly, been most What sickle of tease we've exposure or any, in that or look dissociation we dosing

than into more who these population, numero you a When more a to diseases, red than that healthy complicated in cell go things red compartment. little cell it's the have has because will and a a "healthy" of these handle challenging various patients get volunteer little

other for you had what on -- are additionally this be that go about would will when is criteria work, be next thinking previous thing the you steps be you And the I we aspects are about the we Phase lot Phase thinking you through cetera. demonstrating II a development. thinking question what think proof-of-concept, do more work, III So for and how a thinking and we'll should about et

the well. follow-up totality duration patients So have and be of things driven will data, number are the that of of all by we the as these

Mark Breidenbach

Darrin. All follow-up a right. And for then helpful. That's very quick

patient you also, that us transfusion-independent the rigorously all types in that just sensitivity they've that patients you've to would population been characterized that to back the wondering if Going X,XXX confer PKD was they identified. mutations the transfusion-dependent And split if potentially confirm group. to patient genetically and between I -- have could mitapivat? give of

Darrin Miles

have that hand. at don't I so Mark, detail

point. think -- able be the something at follow-up I we this might that's to with

important both EU the I the in for on around and is sort which support urgency been key testing, the U.S., offering the think the focusing of thing here that we certainly in focusing that detail. that asking diagnosis, follow-up fundamentals we've on to is get be the we in more in the to to to I in you're But order as fully for place have be the afterwards well. questions answers here think might to able with need able raising

Christopher Bowden

fundamental. the there's disease fundamentals we on -- I here. that Chris think It's just are we that couple think of -- don't a would

have missense of is mutation. One of a that XX% them patients

patients are potentially non-missense have those X with So mitapivat mutations. addressable XX% that versus who

how and it really aspect we There's a mutations that's about more overall. XXX think being of I described. think So important

outcomes, something gene So a at. about there. of versus just that mutations, B and looking But specific you time we the genotype when spend genotype, unknown, unknown there's is think number A gene of lot

Operator

question next Our Canaccord. John with comes from Newman

John Newman

bold the I my on billion. that. take you on hat $X to prediction pretty And

from for but that that it, this remarks. Just to like billion $X target But is curious, just does didn't didn't M&A? I allow sort It prepared of like case. your the sound it think I ask. It don't the wanted possibility seem any of

Jacqualyn Fouse

John, what we TIBSOVO think at in No, we and or deliver see least, business with I'll development we hands that. portfolio. M&A in least, at no today it's with the our is Jackie. what current can there's This repeat included

Operator

is question Markets. George Capital with Farmer next Our BMO from

George Farmer

with get and TIBSOVO about to some and the with thinking I'd increased maybe, docs seeing better about. penetration How can are a this combo like venetoclax in TIBSOVO answer HMAs in setting you cycling that in now Darrin, understanding, between setting. in pickup combination HMA? combination the AML you're

Darrin Miles

the think things establish. to I first maybe couple So fully of

therapy mutation I our think of in belief got the want targeted are -- is, is what's in important in use there's early in with it. physicians a inclined to in Meaning, and we continue importance firm mutation firm is the that setting The to targeting also to that you've belief see -- more mutation. and a think mutated IDHX the target. this for along identifiable research the a driver I the particularly that if -- driver IDHX

well single adoption think, I frontline single as TIBSOVO, setting both that, of with outcomes promote is in we that, use. are the to So agent as data the the though agent only in clinical along combination -- driving what's what

overall the -- setting continued that indicates as this recently it's years, likely updated greater half seen, relapse improve is as over data is at the awareness preferred about X that is with ASH. congress prompted and use, the combination by suggests in last use most that combination to we in in frontline -- at and the well don't quarter-over-quarter, both Again, setting HMAs. promote it of regimen that most it the the of data we've we've it. The the But

then good finding population. a overall that's has become think in the HMA. residents setting their in -- And beyond will there. of focusing exposed we is on and TIBSOVO, have -- patients compelling the to they those residents indication end, In leading terms between at monotherapy use is of portion patient aware previously they're the with And more profile that have for combination, our we -- the that data expand good I think community focus venetoclax who how message been of to the they them I as an cycle the it in clinical frontline at get emerging least

days So relapse -- in the and out frontline figure going new to as the treatment stage, because it's as well treatments algorithms begins setting. are think at sort community of their of to established I this the still be influx the just what of early as

George Farmer

an about the activity very form of the That's wild-type helpful. drug versus think against enzyme in about concerns matter how there mitapivat, And form are stages of differences? again, of enzyme? do Are issue any overshooting the earlier think? then I are does subtle regarding been you how -- setting, levels? Great. mutant think you had And And the genotype hemoglobin there that any we should in development. that the

Christopher Bowden

Yes.

because non-missense there's place been general Chris there's are deletion. and for a in because much around as not you've large bind it's rule matters have less that found and for the as no -- a the there's kinase to stable, Genotype So here. And mutations to and got -- pyruvate protein we bind deficiency. have protein to drug drug

important to So deficiency pyruvate the activate in enzyme. terms really are of genotype kinase features and ability molecular and

our kinase in In the wild-type deficiency. thalassemia an the cells same is anyone's underlying red have or kinase have don't don't and they cell, they enzyme, that's approach pyruvate sickle activate hemoglobinopathy to if pyruvate

globinopathies activate that alpha be in or outcomes Whether beta or the Now if looking the you one or at. you're thalassemia? cell, various can will we'll be disease type PKR things SS sickle present could S-thal? within some when be of you that be things function Those instance, interesting of, or your that have wild-type the will that a is that for of

the kinase activating do we're us the -- these three cell So disease. we're together we separate. tend of as pyruvate we're We diseases of approaching lump sickle you wild-type hear -- in to and form whereas somewhat deficiency thalassemia will think PKR, mutant because activating

less their wouldn't ATP trying in a there's pyruvate cells but there's these push wild-type red there's kinase whereas that level underlying ATP we're of disease in I approaches -- more say so there deficiency, because relatively normal, So live longer. that ATP and to up the process those enzyme, can mutated tolerate

Operator

like to her would queue. I the further call remarks. to turn Jackie in final not the any showing And I'm Fouse questions

Jacqualyn Fouse

across XXXX business. Thank operator. our year against as we for pivotal execute you, is Agios milestones a important

trials. expect continued and this our product new employees thank passion I and from the be all in us to making for for tremendous them, caregivers we engine like all pipeline of to of quarter. a their productivity do all for to of focus to will today. would today, areas, outstanding who research thank we filed our growth also see thank of clinical and I Without clinical patients. what therapeutic in difference We on the do for you significant our Agios call a our physicians our revenues progress like joining you we want participate also would soon. I all patients, our dedication in and our to three IND with at not could

Operator

participating. And with you for gentlemen, thank we and ladies that,

You may disconnect. now

Agios Pharmaceuticals (AGIO) 13 Feb 202019 Q4 Earnings call transcript