AGIO - Earnings call transcript Q3 2020 | Agios Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to Agios' Third Quarter 2020 Conference Call. At this time, all participants are in a listen-only mode. There will be a question-and-answer session at the end. Please be advised that this call is being recorded at Agios' request. I would now like to turn the call over to Holly Manning, Director of Investor Relations.

Holly Manning

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

You by our on today's Investors for Officer; Dr. can slides access Chief our call with remarks our Executive agios.com. Jackie Fouse, today Chris section website, going With call me Bowden, the Dr. prepared are Chief of the to Dr. Financial Commercial Bruce Q&A. and and Jonathan Marketing; Medical Miles, Senior our Global Scientific Legal Chief Officer; our of Vice our Car, and Officer Chief Officer, Darrin Corporate for Head Affairs. President of U.S. join and Biller, will

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

Jackie Fouse

for thanks results our Thanks, morning, everyone, Holly. Good and third XXXX quarter joining call.

submission the quarter impact and which our of for XXXX, healthy inflection and navigate year in the we've initiating line of generating sickle been Officer, throughout mature points, of of to at filing joined whose regulatory who patients XXXX, a medicines expertise of Corporate focused to our and right cell cholangiocarcinoma and us Affairs first clear Behind the as includes Legal Our significant activities a ongoing these Biller a solid toward hard this what's mine To scenes, PKR AG-XXX, September. been this lead ACTIVATE-T our readouts our overall of ClarIDHy the later our fourth for This people last a and strides coming of milestones since pandemic. TIBSOVO mitapivat tumor important for ACTIVATE from important made our our against patients next-generation activator. key studies year. data role study appointed NDA them. top to he addition getting and and XXXX, survival quarter work for Chief we our toward help of thalassemia the were preparing to volunteer enables for longtime data study, and we supplemental Jonathan We welcome in need planning November including on leadership the team for key Head priorities pivotal execution value catalyst-rich in to Jonathan is and preparation our Financial the commitment global disease. colleague has in COVID-XX initiation studies, continue and doing

You'll him financials. our discuss as hear from later quarterly we in call the

We also including the in to setting dealt in application relapsed/refractory authorization EU. the AML marketing TIBSOVO need for withdraw our with the challenges, some

much larger not Phase the MAA in of decision any populations. track in setting our those near-term our patient to AML on TIBSOVO ongoing region. frontline Our will remain studies for The in combination changes withdraw to approach the X result

the activities gated is and medical to and and clinical focused the and supporting EU team patient with advocacy, efforts, trial continue largely education product aligned in future including the EU on identification. be region. disease will timing lean of Our market Agios' affairs patient PKD in approvals development Investment

mechanism of have hemolytic we value remainder a for to the Looking mitapivat of that ahead year, of serious to anemias. number the program our potential events activation further elucidate key the the treat PKR

X thalassemia, action, webinar will which dive will sickle needs the franchise, include a mechanism we November hold of in finally, on an into on deep in unmet PKR mitapivat PK XX, of we focused an of investor as First, tremendous now by And Phase expect sickle our thanks patient in data the as the will disease end our disease view our on conjunction investor the December the potential ASH, top PK well the in the preparations ACTIVATE to launch. team. cell commercial our we study opportunities data of In X an deficiency, mitapivat rare share with and disease for overview these first of from to of thalassemia. host line of market underway event and the cell non-transfusion year, NIH from dependent efforts X study updated deficiency Phase plans the our

and and their of personal I a us, to it each busy employee Chris, patients continued for and months me while have balancing for advance our pandemic's on thank want lives. Agios turn professional programs over now to few let every impact you. We ahead the to continuing

Chris Bowden

Thanks, with Jackie. first-in-class anemias. distinct hemolytic PKR activator, across I'll three being start mitapivat, our currently evaluated

our advanced the of we most our mid-XXXX, deficiency, Earlier disease. highlighted, those top trials timelines. I'll between a result line team Jackie all Agios, pandemic. studies pyruvate this the two anticipating Thanks mitapivat some provide part to to As clinical guided for ACTIVATE-T data to of able in large we're we coming with potential the anticipate weeks sometime milestones commitment program are and extraordinary advance the at end year, to first start several this three the for our year now that have of delays where the and therapy of this kinase ACTIVATE in we the programs. and will as X running narrow important Phase in disease-modifying

are data transfused, pyruvate the kinase For ACTIVATE, who end top by of X study Phase the randomized, line adults XXXX. placebo-controlled regularly we anticipate XX in not with deficiency

deficiency commercial the open-label a study. in to approval data single-arm this regularly line XX in deficiency of who are year positive, XXXX launch anticipate study we U.S. broad we geographies. next for regulatory X XXXX, top with Phase potential If the the PK in a transfused, expect EU adults label adults for file both the both ACTIVATE-T, quarter and longer with with in in first time For in follow-up PK given

efficacy presented study the hemoglobin patients Phase Moving in four a of data updated the thalassemia, greater XX of this equal virtual mitapivat we weeks patients to one June, data EHA where And four through deciliter have alpha-thalassemia during completed mitapivat on of earlier no with evaluable than decades. and enrollment XX The showed meeting. or including XX patients X out at in evaluable we treatment per to increase in that been year options gram XX with patients. induced XX, there new treatment four of

weeks of XX. beta-thalassemia In patients a XX eight seven sustained through response during hemoglobin addition, achieved

at data this XXXX. in a submit to expect from set meeting the We full medical for study presentation

and plan we've U.S. to our as our program This anticipate pivotal pivotal initiating in beta-thalassemia we spans working alpha EU and event regulatory transfusion-dependent share XXXX. patient non-transfusion pivotal through our development populations. We December, ASH to feedback At well program. as robust both that of been expect dependent details our quarter, in and

let's experienced a baseline. paused from a we're on into COVID-XX Now data of disease, Enrollment six increase was patients was it the showed with proof-of-concept cell clinical mitapivat. eight turn mid-August. In the of June, a data sickle established one week study achieving that of analysis patients. collaborating to Institutes announced Swee to greater the due temporarily of hemoglobin eight we of Dr. with or pandemic of reopened a eight Health deciliter in study over period, on than where seven eight of to Lay spring gram patients with hemoglobin from dosing that as to five and equal increase, per preliminary The has a National Thien based proof-of-concept

presentation of expected in with As XX least the three medical A top preliminary has At patients two at included total today, will Chen in XXX-milligram for and and included presentation. the study XX enrolled next of data an updated from at the be patients preliminary Dr. Data analysis study a complete eight patients, the from to ASH, be enrolled will the give the meeting from to additional enrolled patients, in time year. from three five the oral patients patients. approximately BID presentation. will presentation be the dose all line submitted trial newly and ASH fully received the

for X cell perhaps for parallel disease, two we're working an Moving mitapivat many and where have safety long-term forward, EU new approved data these NIH the sickle all advance patients activities, to clinical development protocol, will for life. efficacy receive the recently years. plan. potential to years, In The be valuable regulatory extension to on disease our phase to obtain to pivotal in and patients Phase to study therapy in U.S. in sickle provide cell feedback the with extension up the rapidly allowing continue

programs. hematology received a at assessment our our within Phase relapsed/refractory to feedback month, from share in our benefit application and the in authorization the the pivotal I'll a initiate for unable to objections raised major single-arm now the IDHIFA work to September. malignant we move explanation Despite withdraw the expect a an our design the fully of positive from based by decision overview first shared study. X/X XXXX to we of oral experience team, half in to study result the of in program on address non-comparative in-depth TIBSOVO additional year. and indication the the were CHMP We learnings support our as the marketing risk proposed AML the Last

overall diagnosed pursuing gears intensive look are studies EU. survival TIBSOVO placebo. improvement receive the last the Switching EU study. shared Phase the of opportunity previously to patients newly solid HOVON and positive, AGILE The U.S. a treated will in eligible we positive, secondary X Phase the the significant and and AML we combination study in from largest vast of non-intensive we with therapy, access anticipate the we month, a trials enroll in significant highly approvals reach data mature the While are And top near a tumor Treatment of number year, If to trials patients disappointed survival, are overall so line majority statistically we in IC TIBSOVO survival that non-statistically frontline at these to to reported ineligible mutant progression-free compared in the term, associated IDHX our IC Last to was patients. not the in with provide eligible the or continue AML. have to endpoint X programs. cholangiocarcinoma. TIBSOVO in CLARITY

consider we positive the arm, the survival data Given that over to important overall arm patients XX% to in of be trend for this approval. from crossed ivosidenib supportive placebo

are a supplemental We in quarter TIBSOVO treated for previously of will with data submit the sharing to application drug cholangiocarcinoma in IDHX mutant FDA these be XXXX. and new planning first

overall expert ASCO the in NCCN the physicians Additionally, CLARITY par at results GI set data select an of in have potential supported robust the The of of statistically biomarker final guidelines impressive recommendation January. for benefit with populations. TIBSOVO survival a encouraging PFS submitted other and level presentation inclusion we at recognition This is for clinical from treatment TIBSOVO. therapies of level the on approved the endorsement

ahead of to finally, despite it performance. And trial. Darren Phase Phase exploring low-grade dual cholangiocarcinoma, With an inhibition physician third roughly the the discuss the escalation over patients of IDH is In glioma XX% addition our to enthusiasm our to IDH commercial that dose reflecting are quarter inhibitor of potential turn pandemic, low-grade our patients. for has potential arms I'll in mutation. in Site continue to the X INDIGO trial this are X the the of of with brain-penetrant start-up vorasidenib. expectations enroll taxanes we AG-XXX combination Vorasidenib treat to utility with and IDHX/X that, glioma activities vorasidenib

Darrin Miles

duration and both Chris. strong increase Thanks, to pandemic. scripts over was of in increased through by we community approximately setting the the co may in and net growth and driven five see momentum the quarter continue in half community $XX use to patients we improvement a continued academic in of trend sales months. the reflect between the and the physicians in COVID-XX million academic Performance TIBSOVO, continued significant the of the increased Though community setting, and in of management accelerated observed particularly likely quarter, was increase The third QX. in new resulting by a in first XX% refills XXXX

improvement in diagnosed chemo physician TIBSOVO chemo-ineligible preference physicians All care of in first for and ineligible believe We TIBSOVO intensive those see standard indicators of including encouraging, steady continue share are settings. patients. perceptions who to frontline-intensive leading physician significant in the TIBSOVO of a increases for newly is relapse

noteworthy. of opportunity, While AML our increase the cholangiocarcinoma limited the commercial we Though NCCN update represents following are to promotional into made addition uses the limited the the label in attributed visibility the an observed specialty in is efforts TIBSOVO channel, in cholangiocarcinoma June. in volume on of commercial indication-specific to increased pharmacy utilization about to guidelines which this to practice following TIBSOVO volume, X/X guidelines

with restricted, geographies, team in head months. engagement. academic and tools and some interaction, fall anticipate customers, the While customer educational restrictions that Nevertheless, into largely communication particularly tighten we've and at winter we virtual been live face-to-face again we will remains easing late effective as to customer using seen our has continue certain

As without of weeks XX%. a indicators number performance in some we new indicate the Leading result, not were of QX customers to and quarter for the The the able QX. trends to increase but outlook is headwinds. early strong, fourth potential similar by

However, the we COVID-XX of expected also infections number are anticipate hospitalizations the to potentially year. patients as through increase diagnosed disruption the in AML of and treated and end

our million, to to benefit of team gross to a and XXXX quarter net no Considering sales denied times, increased by to treatments. million the of the patient these opportunity now turn narrowing our it for through In from want for for the uncertain we result can I from of to Jonathan $XXX remainder pandemic, year-to-date fueled I'll volume may see our to over year, U.S. our addition, we're increased million. million thank only channels $XXX is $XXX between TIBSOVO XXXB discuss to the in performance outlook and our financials. ensure third $XXX face on as but which Agios apply thriving to to passion the moving net. the pressure adapting, not guidance Medicaid

Jonathan Biller

Thanks, Darrin.

be to offset and by royalty summarize. of quarter in this our results quarter Celgene of XX%, sales collaboration revenue the quarter consisted total detail with million, release sales, will XX% filing be $XXX,XXX decrease today. issued in the will collaboration development third I of $XX found revenue revenue which press by which in million second research driven Our Compared included in TIBSOVO increase was later the More in XX-Q an Total a can of third TIBSOVO, service QX. in completion XXXX, the due revenue of we revenue. for $X morning, net million with and of obligation grew $XX

QX demand. revenue sales prior compared relating by royalty for BMS was taken by to quarter periods. TIBSOVO million was within addition, range. net revenue to In XXXX, $X the an impacted in the this to driven grew increased adjustment Gross reserves quarter expected by by

was $XXX,XXX. Cost We of sales continue the to PHS and for monitor utilization. Medicaid quarter

million down million, Turning quarter a This cost $XXX the R&D R&D workforce XXXX. for decrease $XX reduction offset third quarter, for compared by quarter to quarter $X by decrease third equivalents We a TIBSOVO CLARITY a Selling, quarter the securities of due primarily representing a general COVID-XX ended spending operating marketable in driven expenses administrative external were increase cash to development million XXXX, the third to in year-over-year including of expenses. was $XX cash, increased initiatives. million. was expenses, million and driven Phase savings over X with by winding and clinical the of study. the $XX and largely decrease in costs,

in to plan our program-specific but open of that, anticipated end product our expected line fund will to current milestone payments, operator ending excluding With We balance operating questions. the revenue, the expect for that cash please addition QX XXXX.

Operator

Our Instructions] Rama from first question comes Anupam [Operator from JPMorgan.

line open. is Your

Anupam Rama

guys. taking Hey, question. Thanks for the

trial and cohort much. of thinking Just of importantly, just time a also starting about for last Thanks mitapivat I the know that Phase for – AG-XXX, X you on that outlined SPB year? should you’ve of the started, the healthy timing terms lines the be it one that have just that XXX in so thinking about we quick volunteer data? how sort but

Chris Bowden

it’s Bowden. here, Chris Anupam, Chris

healthy in then volunteers is sickle healthy single and we that, to they we what cohorts there’s multi-ascending plans So move the did volunteers. as dose And mitapivat then – a the have study into cohorts as with per dose cohort. cell in we ongoing XXX and open following

we be get mass try able well data. like cell to So we of out Certainly, think when the sickle as as previous, too perhaps to PK as think a time, early cohort PD it’s when feel around guidance that we that able then there opening. when there to for would to have per provide might publish moving data critical inform to phase be and that we as to once be and might we us we safety might we and

to health Now be we’ll with authorities, the X on update And our sickle further remarks, mitapivat gear start interacting we’re with as Phase to that’s able and well. regards year. in next the in full my with developing protocol, next year cell, that and as there

Anupam Rama

for Thanks question. our Great. taking

Operator

you. Thank

Buren Piper comes question with Tyler next Our Sandler. Van from

Your line is open.

Tyler Van Buren

increase about the update your I a cell morning. Good In than for in Obviously specifically especially to in at XXX two there particular, BID ask to BID, milligram it And mitapivat taking abstract was mg benefit wanted the the patients in the confidence clinical others. significant mg. all for relative X.X more in questions. versus guys. the the dose Hey, hemoglobin. XXX eight the sickle XX milligram at hemoglobin ASH. the at X.X generating the Thanks was only XX

could wanted there dose the that two specific show to hear maybe been again, improved on patients, so about those will but benefits. And confidence your that. XXX-milligram have And your explanation something

Chris Bowden

Yes. Thanks.

put to responded those X, includes patients the they think up recall then or looking keep patients, could the this that at from values once start in, of baseline. and important escalating. amendment They those whether are change the thing That up And that the So XXX. go go I you’re design at all with we study not. they or XX they can to is eight as

where bit one a little about perspective, what that remarks eight hemoglobin. So EHA, XX. talked From top challenging. from we at the dissect made remember in I level had patients of higher we XX increase in a gram line the were the is or high table differences trying from five Those in to at think XXX our all

know think an and have healthy our we’ll quantitatively And probably have people It XXX. volunteer of dose to a us the things work sort range patient. of from definitely XX go from we there’s we active that number I some So one XX see to possibility for milligrams. doses. is most We to varies to it activation, of things have call of, when patient that more the this of important from of a about

thinking that is still increase get, something XX increase it it’s XX X.X X.X XX XXX we to went look you were if about. or when or is let’s and whether say, gram So at we’re X.X to way XXX And another that another and XXX. at or to gram up gram

Given that’s emerging the be definitely that to if XXX. XX could looks some data safety of a outcomes, of hemoglobin which and And does clinical it benefit. profile physician around interest across the in patient comparable,

data lot be dose we a of design generating that other we’ve that from the of the in studies around confidence from this and a really around of XXXX. And way gives trial. able first details So more the provide been and to trial us our half some our selection we’ll

Tyler Van Buren

Okay. Maybe just one follow-up.

milligrams being to or time hemoglobin BID patients dose escalated. dose mentioned matter will in new escalated that terms five existing in they response? the not, you’ve of the reported Are same and that And are patients And and does ASH. eight XXX so XXX dose in if same XX at frame on that mg be – course? are are the So bid three the the at all from up

Chris Bowden

weeks drug two for you’re two you’re is XXX question your understand – two for milligrams for for milligrams I XX five weeks. weeks, on milligrams milligrams if XX for two weeks, now two and for think two weeks, dose on I

enrolled So are in there’s sequentially. break no patients The between.

time. is they’re not – it’s the at enrolled whole the they’re for like and comes dosing same But frequency cohort similar patients. the all and all So in

Tyler Van Buren

Thank helpful That’s Okay. you. clarification.

Chris Bowden

Yes.

Operator

you. Thank

Our with next Frahm question comes Cowen Marc Co. & from

line Your is open.

Marc Frahm

long-term in benefit a questions. that the patients dosing Hi, XX. of Chris, on versus there my individualized to whatever thanks many on the trial, The at comment there be off actually a just on rolled to is back right, seeing these prefers? for extension, XXX milligrams? at spring. kind follow-up taking going patient of just made Or XXX ago, before, And some milligrams you that is minute in then based

So think dose up? back do they just do you straight drug? can them have you to Or put the back escalate onto kind of

Chris Bowden

and what So in what XXX-milligram to that at they’re do is in think XX. going dose optimum just to XX is come they’ll them they get an to range,

Marc Frahm

then Right. patients just idea newer existing will the trial? patients, the straight start these that And into rolling is

Chris Bowden

extension. That’s able be to correct. will the They just move into

Marc Frahm

Okay.

that you Okay. that down And that another again, Or test been out – think wash-out to you you’ve involve have doing? stepping and experience. would the do enough titrating period whole

Chris Bowden

Yes.

they and around So why. we that – will the finish now. it’s in. That’s then taper need need what to study don’t we’ve up so additional separate taper feel got back the ongoing the beyond will a protocol, set in come data taper they I like

Marc Frahm

then Okay, explain discloses Gardos? kind before just Because great. one on with on possible sickle Can you just making program Phase high-profile maybe you cell? what’s before. a it through the that think also contrast there’s been and studied mechanism – Gardos III second a thing there then what’s been that obviously, And abstract, of the in of action

Chris Bowden

Yes.

– that and on the from benefit, cell be also research this longer, volume, they’re the the increase that potential providing mitapivat mechanism in Gardos may involved healthier, hydration, directly which clinical cell And cells improve, fiber. state, red – both So mechanism channel they health that a now hydration the could which and polymerization which by MCV. overall perhaps red a has of you’re some decrease investigators have the mean that a better – called is is live given something our corpuscular from that has our based increasing what’s in team has been as for of been the interest,

Marc Frahm

Okay. showed between is failed drug, the the before you think a – stat trend VOCs Because against with think I right? working has any been of and studied obviously, but hitting is how even there this in difference guess, X terms on actually there And Gardos? of sig, Phase what I trial kind

Chris Bowden

but trial work Gardos around that hemoglobin stopped safety. because differently. That very improvement blocker the and affect of showed an Gardos some interesting because in We directly channel don’t Yes. channel. was VOCs a was question We

long drug you in for PKR terms activating cell as red see health then number kinase reason has demonstrated and activating patients cell. is all the will of fibers glycolytic to been there be decrease with it’s impact Our abnormal elevated a is the that specific sickle ATP, red formation The believe of increases by happen shape that that the dropping emphasize, pathway to in on effects there. cause of the and X,X-DPG, the it by bad that of pyruvate we cells and and positive which sickling things where

Gardos So PKR. of that to – improving mechanism may it’s of ATP it’s red observation But activating cell that fundamentally a action ability that we through be hydration. increased very for increasing the it’s do an by channel

Marc Frahm

Thanks Great. a lot. Okay.

Operator

Thank you.

Our Lawson Barclays. comes Peter next with question from

line Your is open.

Waleed Abdel-Naby

considered upcoming Hi, Where PKD for results had the transfusion-independent meaningful guys. see the question as would year-end This look the both you in is transfusion-dependent patients? taking Peter. And to Waleed bar around just and and data quarter first data. a XXXX is, be I on questions. clinically for Thanks what we

Chris Bowden

Yes.

it’s – at very an Bowden of endpoint benefit. clinical will course, designed with designed carefully with we is endpoints. associated – secondary trials be both that looking here. be of will And we So Chris those We

trial that or then is and assume study as have at rate to to placebo the the in is randomized for the arm. end ability crossover X.X with if X% active we XX or ACTIVATE the where response drug So of defined in positive the control study, dosing – patients placebo, one-to-one there’d gram hemoglobin a and as patients be is baseline. to XX%, the approximately we a response rate period, And a greater responder designed for increase

So talk be people we saw over when in on the what would benefit. talk has even study. to bar a been higher. set positive of DRIVE a clinically, clinicians And clinical to gram We PK we terms then based you think that that in

that’s a benefits. important that patient a supporting meet transfused confidence we’ll a package be that we if primary not have also in lot will we have of endpoints be clinical regularly population. indicative secondary the of So number endpoint, that There’ll of

who in which is compared time study, of transfusion. that greater to overall transfusion the XX previous efficacy XX% are patients study the a is ACTIVATE-T the burden or their transfused, the endpoint For reduction they’re regularly one-year at history in

the those so look of And an measure in I clinical time, patients you At transfusion went that regularly as independent. open-label same that’s at who people as that – reduction are entrant And trial. on. XX% well know just transfused, primary the XX%

need endpoints. to what and transfusions, getting we’ll and the the like has a – demonstrate spend those impact and reducing secondary patients outcomes might are blood in that efficacy at the group mobilization they time terms amount mitapivat So will that other that of clinic endpoints important in impacts have big other hope will in we secondary need of the of we really iron look also on the chelation for iron

are the So to able benefit use as them clinical pyruvate trials be the really adults with kinase think deficiency. get like total, we to trials demonstrate in And package overall. to up set we’d a two to label in broad a

Waleed Abdel-Naby

for you Thank the question. taking Great.

Operator

Thank you.

with Citigroup. Bansal Mohit from comes question next Our

is line Your open.

Mohit Bansal

Thank when congratulations, been taking a there first and starting XXXX role, not for question, in and recent very this for COVID go has didn’t the with decision, my question. Jonathan EMA much you guidance obviously, mean, I of all, your $X by there, which you was new favor. billion Great. first I your think provided

planned? make – your to $X the aspiration you as is? wiggle this Thank still XXXX it if So billion current is uncertainties, is there room with And happen go or little about today’s guidance is attainable by et how a doesn’t bit could much these more cetera. business how you. talk with pipeline,

Jackie Fouse

for question. going the Hi, to wasn’t talk today. Mohit, the I get it’s I Q&A Jackie. to Thanks in thought

opportunity. I So the appreciate

be in TIBSOVO in and may are haven’t faster in quite you’ve we’ve little performance a PKD the expansions. then the case that probability a it’s we thalassemia guided and high where so, TIBSOVO vorasidenib related on. of environment feel in to revenues of still time notice achieving our we oncolytics of achieving, yet, and but of favored little So want at not we line late standpoint in our which AML. revenue to pandemic some terms personally so base for got a But in give assumed like oral in driven But but where by we actually base in bit bit good respect happy year, might in anything with to in AML feel conservative about the included very in glioma that XXXX – we’ve with a or you there plenty mitapivat the like if objectives the a TIBSOVO sickle seems I it is ourselves as is we have then mitapivat the it assumption very would you with that XXXX ramp What relatively in indication them been $X we’ve will we’ve soon also for to even launch with the room been pulling for far. we’ve horizon, label we do also, of those we the ambitious imagine, is for set beginning is And than from seen, also actually objectives. and little the guidance, time we and think XXXX – and they’re have TIBSOVO’s that case in in billion And mostly because what revenues objectives. next you disease hardly the cell revenue

be have impacting about than $X not plan. your seen a better we’ll pandemic issue in at a XXXX; in we we regional the billion COVID fact, comment and an yet. that as the be So, might little really,

I I comments have and still, feel least in it always great been $X my billion XXXX feel. I that’s and Thanks. about think at So,

Mohit Bansal

sneak I for may If it. in Thank for Got you one this, more dial-in. Jackie.

know small the are I peek, say I you they PKD doctors, a get So, could patient patients. XX, going many with talked talk that they then about on it lot see to is but about and a sneak November we often f don’t because population

go you low-hanging patients you. you about market? terms PKD these And fruit identifying and probably, So, there context, are how there, or how in in where would is find of Thank this – a the extended patients?

Jackie Fouse

the for Thanks question. Great.

– that and The those – we’ve X,XXX well. just So, the XX,XXX that PKD years we’ve last to we’ve as additional overall high the to the identified X,XXX, X,XXX have then XEU, in been right patients executing maybe between shared over efforts EU. patient between been estimate XEU. and when right, you U.S. for identification right, that about U.S. we of couple as that I we or so have number think, previously the include split beyond major that population of patients a that publicly, as yielded be believe markets and

third that But – So, a – the into when a than you and prevalence identified prevalent population. me a all right. account lot it for lower be about a right, about that’s work more take third of now allowed a today, to of little continues between potential pull we’ve third, of approval the of end that, to

awareness both disease education, work and addition work, in doubling that in down So, EU disease patient identification to on the whatnot, advocacy, U.S. and and the ongoing is that just

Mohit Bansal

Thank helpful. Jackie. Darrin and Very you,

Operator

Thank you.

MacKay Capital from Markets. with Our question RBC next comes Kennen

line open. Your is

Vikram Kaushik

feedback the your for Vikram and be would studies X there thinking This withdrawal and confidence Phase after you and HOVON that and EU today. confidence changed us the of this in on your I that is quick our for one agency regulatory Kennen. to taking timeline have Thanks guys. just you concerns AGILE, got. the feedback? regulatory that super-helpful. updates what Maybe, can if led so Hey, for the much on were remind main have a questions

Chris Bowden

Bowden. Chris Chris Yes. Hi, here it’s –

confidence which in I that said, in are the that’s my where are. our and X as because trials, important, diagnosed patients excitement that’s most is the patients So, remarks newly really where, Phase around

operational we – year. And the ongoing end we’re phase, operational yet. That to committed targeting, where to very patients, have, they’re those we is board. of the to effects COVID finishing early on IC HOVON the in guidance next study where accrual reset at of trials, sites trial coming AGILE, and so in had its AGILE and eligible haven’t And to because guided are study which the is we around

population, for committed group we’re studies is doing – they IDHX and and a in a committed However, that. doing that’s have definitely seeing an long both done that’s patient being a in of the eligible international doing to group record to IC this IDHX nice the accrual track

are to they’re with satisfied really going partners and there we’re how things work So, very great with.

Now MAA. the with

this with I there clinical control no into we was activity that were durable main there wasn’t doubt, arm. So, that appreciated had was a space that think the concern on The and that relapse appreciated. refractory going TIBSOVO we in oral the knew agent responses

IDHX of impressive was looking demonstrate the So of data, of control these explanation this did data not looks at and like to enough is to an – historically in the effects just very with that space, controlled a in what relapse the data There’s relapse types lot withdraw. demonstrate historical historical and which going sets. refractory to compared that ASH control we decision lot the opinion. day, terms disease. that’s of give published refractory we And our skepticism, in the a after made work look CHMP be and to But of will to survival at was some at the of without CHMP clinical going – end when positive we wasn’t appreciated a

Jackie Fouse

in – for jumping Yes. just a Jackie, it’s mean back I second.

that risk assumptions data. very work like right one what pricing think is our assumptions case in thought that the data Europe, some goes I the have reflected We IDHX Chris will Europe published patient And thing historical interesting risk we shot shot. see the in we refractory and this U.S. said modest I was relatively a had very the patients as forward assumptions, in around you it around relapse But base this control this. a it of in different you with think Chris still as this for a you a set and some without with arm good gave there’s give situation tough X that Phase in up anytime to control end said, data we did the it’s in population,

modest and see with expect X for to are opportunity, So, revenue you into it Europeans expansions label type which was the the speed the full would trials we’re want to Phase the diagnosed. moving of that ahead a newly trials,

and different a AGILE with HOVON. situation just So,

Vikram Kaushik

you TIBSOVO, U.S. on quick for Thank And that. follow-up much in I so a trends Yes. just had revenue

demand mention did you I know expecting you QX might in diagnosis in about seeing QX, expecting as were of you what given and rates, U.S. about the strong net low be dynamics, growth gross maybe, of can you growth? pretty were diagnosis You bit impact cases going QX to a was and rate you the into QX well, resurgence did talk little in So

Jonathan Biller

Sure. Thank you.

So, yes.

So, it’s express unknowns, what headwinds where I to a potential largely right. tried that are

experiencing even particularly was that experience treatment an in we’ll presentation, of know initial which emergent in had diagnosis, certainly our from surge though patient setting, patients, setting impact like resurgence. disease, that highly know it QX, and an is diagnose, a to as In we right. different in weathered we’re continue experience the it and a We terms quite we oral it AML, well, quite a right. So,

that had in you’d the you U.S., QX. even have quite experience across were you the start So, of though hotspots had, in

potential, that potential we’ve with related of balancing enthusiasm And that to QX we’re very in for the performance so in the seen to real the COVID is what QX. I wanted impacts ensure sort

on for potential net in change the on the said, of patients to coverage mix related drug. Now impact the gross commercial to that largely are

and or of And is potentially We as realities impact anticipating well the the coverage, economic increase in haven’t on and headwinds on those our are course the that’s course that on coverage, things seen patients the XXXB a DSH huge those insurance some employment the moving perhaps as provide overall through an shift, over over performance. of hospitals. but Medicaid possible based we’re could this that – QX it –

Jackie Fouse

Darrin also very of October of caveats that those off the of for all to with remarks in good And his month start us think prepared said a QX. put was I

Darrin Miles

Yes.

We’re the so pretty what pleased far with seen in we’ve quarter.

Vikram Kaushik

for Okay, so much thanks. the question. taking Thank you

Operator

Thank you.

Our next with Young Fitzgerald. question comes Alethia Cantor from

line open. Your is

Emma Nealon

Emma is for this Hi, Alethia.

cell, Activator we turning back abstracts co-primary just endpoint. to that difficult included their in another a PKR So, in ASH development outlined as annualized program sickle VOC that saw

would versus So, that you I planned for there to think show are guys you also or on like Oxbryta? nor drugs competitive discussions that know something be mitapivat regulatory important showing is but commercial agent be design, differentiation the to

Chris Bowden

other it’ two I reduction if in hemoglobin to a said is to – your that, believe as the here going VOCs. get-go, Well, the reasons think have is I PKR Activator, you’re run from we’ve any, and trial with think Chris one raising you and

marquee, we’re with the big saying see endpoints hemoglobin, we for co-primary lot just if drugs for from be both. – P-selectin the I doesn’t significant really what’s patients. that that two approved think achieve – problems VOC still address that – going it’s clinical for the when benefit are – promise statistically reducing sequential the with is can a for decrease in good that you and the on for I we to and the a at and And it’s decrease follow-up we’re that out impact with reduction mitapivat be activation. hoping address held whether no potential, can that inhibitor would it two patients. about promise there’s disease there on sickle think trial, testing, main the promise, significantly VOC in VOC, to will, that’s without At whether i.e., Novartis able longer hemoglobin, is the an ways you’ll what’s a might mitapivat the goes in you have that that So, has you see the cell potential PKR endpoints, increase clearly, voxelotor it those of are but seeing for to been where design a And are is the it jury’s

Emma Nealon

XXX? is then give plan to the And kind of any or pediatric on update you. development Thank PKR you’re mitapivat there for across able Great. portfolios the

Chris Bowden

moving mitapivat program our in with pyruvate So, forward kinase is with deficiency. pediatric patients

a after terms in and deficiency, think have go of we’ll as year. we details way those kinase as the certainly those happen. studies out of far for regards mitapivat, focused take end those I getting running. by And And demonstrating. and AG-XXX cell worked long to But sickle and toward to up we’re working on need we’re so this will right with And pyruvate to now thalassemia dose,

kinase is We now and hurdles development efficacy in has well. sickle that safety are to stage right start thinking now for Mitapivat pyruvate it the data deficiency very years we continues do early next its development thalassemia what about some And achieving before compound. and for cell. of of

motivated we’re don’t need moving plans the start kinase that our by evidenced to deficiency. we XXX and pyruvate pediatric fact So, as forward

Emma Nealon

Great. Thanks very much.

Operator

you. Thank

next Schmidt question Michael with from comes Our Guggenheim.

Your line open. is

Kelsey Goodwin

I Thanks last for your I Michael. Hey, PKR remind guys. you of our see of that of gen the secure TIBSOVO, for kind X then you you. ongoing fitting think earliest successful, do are think Could TIBSOVO? and the Kelsey for I question. that guess, phase on if – questions. approval off European kind you you could that development earliest into you for guess, the This how longer-term? when to trials taking next build just is do guess, plans activator maybe, when us thank it be

Chris Bowden

Right.

Okay.

of type we’ve And and all see question that and because will first and XXX on development observed safety we forward across indications based depend to-date. path the activity back the profile the moving with three is pill also be. that the would mitapivat on its So, and – what the we’re data

offered So, that it call, study for of tangible, have we with across XXX, for early be, diseases, need studying course, where see do actual clinical make may improvements too pyruvate to attributes next patients kinase pyruvate and it’s to we continued the improve – molecule, those mitapivat to have would the what now. what because we important activation and potential of diseases patients. while outcomes we’re kinase three investigate to the and

stage. looking even focusing XXX we on that cannot how actively for in we’re us too comment get developed can the to that’s we’re now at. What is situation on do so these something And be just it’s the that. next that would into early

– front front for frontline of what’s in get is any approval question the that that CHMP the could your we in the trials then setting. TIBSOVO, an of frontline in get of So, to those earliest

driven you if So, that’s an complete look then accrual and got trial. AGILE of XXXX, the event end that’s at to at

And in then So, have would fast are how bind it. than be we’d coming get sense we would filing. really events of some often to we then

positive we’re early hand, and to It’s want – global hope in because to get I other our that completion, the just get point. we at too for sense CHMP, would accrual as that a trial. soon get We soon haven’t gives in these it’s we guidance broad if for bond, you whole and still to able large guided it front activating like as So, yet as this be said, can. study sites we us regulators, in provide to this both FDA, of trials we to specific as

Kelsey Goodwin

it. Got

Thank Okay. you.

Operator

you. Thank

Oppenheimer. Mark comes question with next Our from Breidenbach

is line Your open.

Kalpit Patel

a how This that preliminarization, Patel thanks for the had I guys. with questions. with meaningful abstract our Hi changing in Thanks. these on comment if in to the long-term you believe trials? taking we’ve Oxbryta couple Kalpit seen in specifically seen we’ve date the previously on the compared is markers study reduction for earlier of And also ASH on Mark stage to changes and could Do clinically NIH to and then VOCs? PXX show TXX. what these a you as

Chris Bowden

Yes.

definitely assays. is, think did that this and we seeing potential PXX TXX our VOCs. we’re with So, reduce it’s What to has

there’s that is been of has with not a associated direct in this challenges much reduction this the frequency correlation, of One improvement much VOC. the

with at looking important nevertheless, definitely look early our in Certainly different a seeing with And and so with comparison, assays whether all particular have patient we we’re them, that. time, of data that points numbers, in things. concomitant the go point data potential sites at we that’s, some those voxelotor, it cross-trial but When on based different a what to some endpoints, sickling medications. was and at do of with in number caveats similar LDH time,

VOC were at sites the a voxelotor are that patient looked sickling really about. well. bunch that XX%. moving proof have feel say drugs So there voxelotor we coming profile new when all or I'll seeing, to, that of other There's it X that's is you an referring we're And We there's one also with we hemoglobin, a get understandable think you're declared we as good we increase question the does that's these what to overall and have percentage well, thing One Phase very to and there's caveats having asked who value LDH, of seeing. And whether our gram data. the time look I the aspects, that the EHA of, questions why concept. particular at raises greater in, in we're at other it. we about of of us is the really if But about – of the initial lot patients we're that of piece we mitapivat aspect with a interest it's it's what because whether the that assays what's

number of a any don't hemoglobin there's So response. have patients who

a being for patients. So very we could as drug good see those ourselves

for starters. Just

Kalpit Patel

patients. I to then their can access think couple And study or us, at VOCs end the NIH of the showed you a the few Okay. or study if baseline a during historical of frequencies of patients I VOCs the have either guess, data? were, for what these in you characterize

Chris Bowden

seen it? during a and patient was the assessment since. them end one predictable of at don't patient's been a so at were taper. what of study any the several – the we've was had, of the VOCs the that We how there of We terms end have in of have life that that Yes. had is, risk. going that put investigator yes, lot VOC made time, information one they long saw off to on for was pre-study And precipitating to The there some overall period the, would coming the from after factors after in the not that we – drug a annual alterations of taper, number

So it's something we follow. continue to

you part parcel look If any trials, VOCs and the with at development. are the of

of that's really one what's to a why our control, so is you that need handle endpoint. big And a for on going on – questions get

Kalpit Patel

Thank you Chris. very much, Okay.

Chris Bowden

welcome. are You

Operator

SVB Andrew you. our Berens next And Thank question comes Leerink. with from

open. is line Your

Christopher Liu

Hey. Thanks this is Andy. Chris on for

heart had the question the guys a about that Just increase you abstract. in rate saw

if you've events cardiac studies? seen just other to arrhythmia how well-characterized or that if do wondering studies with other in or And an in had any preclinical tachycardia? Just it is,

Chris Bowden

heart is show We asymptomatic rate per – intervention does And at criteria, if what require is, not you over beats if it's an that minute. that it XXX asymptomatic. toxicity common look the

drug. signal with We seen cardiac indications any not this have have a we that

is then XXX, be signs, in, a symptoms it, part were other coming of of biad And suggest and there's any were associated their heart so not have but our seen they're of about. and anything what's there with cardiac had standpoint trials we've individuals concerned rate That's it. otherwise three gist are signals the the across there's who happening getting that felt vital we patients from evaluated above any to that a other –

We and trials. of sense in broadest continue the all and safety all the across following

concerns alters way think rate And at us so safety to on the is going a any profile this focus don't need any we've patients heart point. we've to above that on XXX or us off reported had And not that – something in a to in asymptomatic that's from three something the way. date I this with is set what that

Christopher Liu

more the And about that right. Gardos specifically for all it. that pipeline, Potential channel. other product one Got that's was mechanism the that just was channel. for in wondering if question, I'm Gardos

ideas hyperviscosity. proposed of the increased of was to they One why VOCs due

So just wondering that? potentially something if any could you that different or imply counter had data viscosity

Chris Bowden

Bruce around data. becoming preventing cell doing what by we're I of and deformed. Well, speaking and of the from terms I'll to I him further the DPG on think sickling X that. specific – dropping sticky But is comment ask Car – will hyperviscosity X that think aspect specifically in nothing the red

that So where NCB if in and we the are increases get problem, I sense be see hyperviscosity. this consistent really – seeing that turns will If wouldn't a time, why. anticipate some able this to, out over reason – is it fact, are in we is

the be that's had that's patients for patients a it's small that concern, who how percentage minority XX about have no high hemoglobin data or those But frequency still that there, this can a with going where piece, ASH, out it breaking a of been you doubt, following sickle it's cell So news that publication, maybe VOCs increases hemoglobin now interesting to nuanced, always keep of at now published, not they these just GBT's concern. of develop. that lowest experts has there, clinicians And treat data patients, get who of which the get they please? instance coming Bruce, now XX may and about remarkably, and some more to up, be Gardos things and will little channel this a disease. comments we'll any as the and have to had

Bruce Car

well. address that Chris, hyperviscosity, the health decisiveness to No, I pretty lead one believe think you of covered might propensity that. to we the and of you cells. We the that the spoke parameters it red general the improving the cell of red for

get. a pretty well The not That's packed higher that that something other to significant cell be also doing anyway. is range, mentioned we're very we high you parameter And PCB, in likely seem already volume. to

the there's I study So of active think anything action agent a such profoundly Agios. different draw that from a where additional mechanism from don't has can one

side mechanisms is for the think potential two I probably appropriate. action to of So extrapolation an drawing between not concerns effect relative this

Christopher Liu

Got you it. much. Thank very

Operator

to callers any in call remarks. this currently the other to Fouse I'm over And you. I'd no at back for like queue showing time. Jackie Thank hand the further

Jackie Fouse

and what making all Thank who I not whole Agios caregivers all progress about we areas do the team well. our physicians for challenges we continue Without to clinical remain you, we're to world colleagues forward us would Agios to catalyst-rich on thank today, patients, like that thank like see difference and operator. a that a and and we very in the patients. our my of today, will and you for XXXX year, as do passion in I you trials. and to across I today, excited focus joining for prevail for their To and I just the we close, making want looking that call would like also the could would the this dedication them, thank soon. to our reiterate participate are to uncertainties despite I

Operator

Ladies this Everyone, a disconnect. your concludes great now and conference participation, today’s for gentlemen, have day. you Thank you may call.

Agios Pharmaceuticals (AGIO) 5 Nov 202020 Q3 Earnings call transcript