AGIO - Earnings call transcript Q2 2020 | Agios Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to Agios' Second 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 second Good welcome conference XXXX morning, to

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

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

Jacqualyn Fouse

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

to set remainder vision. significant closer move we across priorities became for cell business mechanism, as that led and behind strategic other the The first the and year second of the hemolytic of such achieving in XXXX. disease. quarter activator type first as key our QX was the and Mitapivat an we years incredibly took establish objectives anemia’s, activating and provide to pyruvate X productive progress wild since our Agios and benefit PKR this we accomplished then and important Phase we The the thalassemia XXXX the time that us we've therapeutic Seven made solidified for the steps several a deficiency sickle in may and program kinase-R company kinase ago, pyruvate science advanced discovered for

further established markers quarter, key we next we in development past To has patients. sickling treat and updates that Mitapivat. sickle the proof-of-concept start, and our activation to these of anemia disease Mitapivat in for for the demonstrating phase the chronic Over and compelling our of underscored that shared cell space support hemolytic PKR potential leadership

a sustained In May granted thalassemia alpha thalassemia patients. EHA in at and for presented us FDA medical this data thalassemia drug in first beta at addition the In for hemoglobin for orphan showing we Mitapivat the time responses meeting both indication. designation

Mitapivat Pharma indication, cell of of and XXXX through of ahead, these for indication As line to all secured the will first and these disease firmly advancement Royalty of of we in Phase PK submission the to XXXX. the an million capital X studies pivotal programs Mitapivat from data our look and for the deficiency we June on help future milestones areas. committed disease and the thalassemia in $XXX non-equity fund efforts. we sale top be across rapid The Royalty focused In IDHIFA to are three of preparations of remainder both NDA sickle

the tumor advanced our business, addition clinical from inhibitors study Oncology and haematology side TIBSOVO progress commercial Phase rare May, the the highlighting our disease presented to solid and In the ASCO to programs. genetic drive IDH we data manuscript in TIBSOVO for At on data of X continued in execution a of malignant we've and areas. EHA, in in cholangiocarcinoma. utility ClarIDHy And published underscoring of we both Lancet

the XXXX. to to sNDA quarter the proud NCCN ClarIDHy having guidelines study We of with patients advanced data the for cholangiocarcinoma. in of manuscript, recommend a the treatment forward IDHX future, s from first expected which mutant TIBSOVO will an I'm the support of result in complexities disruption all were as rising global look submission As continues updated overall states near our with while by team for COVID-XX rates the accomplishing and in caused parts including world, infection the the this US. systems The to in pandemic. ongoing health the care combat in great others, to of some continuing here work, to evolve, in fall while challenges survival many

facet organizational of ongoing clinical team remain on assessing an on impact active operations and response team basis. our Our resiliency in COVID-XX’s business every

majority of home, work designed their Research all of have work to Cambridge and to access procedures health under lab our returned a who the set protect well-being. our from While Labs to operating employees employees continue need to

can as again. addition, team local some of field person customers their interact our allowed, in regulations In and with have institutions

We lives. evaluate professional significant a impact personal additional that each their broader to employee return pandemic on onsite balancing our to work. operating to for and would continue to allow I will Agios continuing while programs for and procedures patients, the thank every like advance

the ally this think have Before to and Chris, racial it's realities which are our force racism racial injustice and and racial believe our communities. again in the acknowledge and be events in violence At to we of matters ship I acknowledged country. recent should turn important committed to we [ph] of I decried call destructive against exposed over act once Agios,

we meaningfully are behalf backgrounds to let Agios contribute it diverse me at all and welcoming believe and Chris, and diversity turn a thrive individuals to us of committed to of mission you. our over in value where Agios of making workplace patients. of on the can All

Chris Bowden

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

As second development program cell clinical thalassemia both the disease. quarter milestones in Jackie in this and broadened achieved support sickle for highlighted, that important we

focused of we beginning XX the first X this have a advanced of patient's to our we is of in therapy. laboratories regardless program courier we disease get all years in patients of provide to treating includes PK completed where and home in studies, trial study we This Earlier approaches, the drug. deficiency, ACTIVATE-T. center. experience have local year, use ability have most modifying outbreak, the telemedicine COVID-XX Our to [ph] of two shipments capture on and and their and access enrollment potential data to patients ACTIVATE have visits, that Since ensuring the Phase drug

core As are later of line lock. this both year, end studies global database an ACTIVATE to from top with period ACTIVATE-T between the both sometime trial complete of to we comes the anticipate understand the XXXX timeline our working steps XXXX. data and of to for end clinical We middle and still sites necessary

global trial to We will narrow ability our site in of pandemic. the on timeline predict this wake confidently access the based ongoing

US with the for file next approval launch in year XXXX and commercial both deficiency the and We geographies. EU in regulatory both to potential expect PK

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

during XX sustained patients hemoglobin In XX. the addition, response X through X of achieved weeks beta thalassemia

a quickly robust transfusion now year, the the and both a beta program well and end with efficiently plan patients goal of non-transfusion and as initiating the is development possible. pivotal advance patient of development of to Our dependent thalassemia, as which focus as Mitapivat XXXX. alpha the to pivotal finalize By we expect for as stands populations, these dependent in

position in sickle study advocacy to the many and they sickle it's now to of with cell input X the basis published focused patients hematology I'll serve Lay further two expansion increase for the let's she a The analysis to malignant patients meeting a proof-of-concept AML that and announced Dr. HOVONXXX, label [ph] for plan positive gram cell which we with to we where group put at Due deciliter in data filing from of of temporarily proof-of-concept Thein To expected period, results, on consistent received was the move over our Institutes X are we're [ph] disease, us enroll X to of were to we plans us presentation list we're with patients Phase as [ph] context which EU global to XXX actively turn geographic established or dosing in a end sickle trial study. the clinical study regulatory in the hemoglobin not will working was or the respectively. sickle as our on patients Swee a ASH Phase our data AML preliminary indication patients these data plus the of of be X of equal the to for previously for this deficiency of start, disease. AGILE on quickly our the broaden with still nine baseline. from seen proof by the we will initiate X combination either PK to eight and use and in the we June, In in disease, study year. summer. studies, in TIBSOVO. the cell than experienced study and a based combination enrolling earlier will submit earlier Dr. the how above receiving X and Mitapivat to of time, December. our from in to serve in Mitapivat. clear Adverse a week the yet included with events this increase pivotal get will allow one reopen we patients anticipate study per National the pandemic up CHMP this TIBSOVO at In working in this Now to presentation, have development showed that and collaborating azacitidine programs, Thein with IDHX wake and frontline greater achieving are hemoglobin Health X enrolment, evaluation. and refractory additional relapsed one Based inhibitor for in include the data concept enrollment day to in on that halted Patient study issues cell from XXXX. frontline outstanding are where XX opinion year COVID-XX X

syndrome filing. the with study arm reopened enrolling patients goal necessary data of We potential myelodysplastic U.S. TIBSOVO also the pursue the a X Phase of to and generating are regulatory

to the in additional XXXX. complete expect We enrolment

explore these registration MD to to in XX% at well Anderson study, EHA patients. TIBSOVO, and we tolerated trials, pandemic. our such earlier represented trial by through interruptions one or an this effective had combination activity minus studies. and composite IST sites investigator the addition In and plus from generate and due Dr. Data continue response In venetoclax with azacitidine May, conducted year of TIBSOVO plus startup data we was [indiscernible] DiNardo combinations site to initiated ASCO slowed some Courtney that the complete that the and at with shared enrollment occurred novel showing

recent now the which and IDHX overall the of trial previous study reported anticipated sites. sNDA filing. the data areas NDA the has expect on for [ph] to previously continue and Some potential to and At XXXX, but remains quarter our of of AML and from to is Phase enrollment the this have tumors. from we impact guidance. have data, I’ll we support mutant to we to the guidance narrowed quarter the move to Mature data X a surges unchanged study begun first now year treated submission TIBSOVO from MDS timing positive expect this occur ClarIDHy stabilize, our on in PFS the cholangiocarcinoma, been studies. Based solid track Last supplemental time, likely we survival

turn INDIGO the enroll and study grade combination addition, continue X our AG-XXX Phase non-small low brain-penetrant we The vorasidenib, it commercial no startup enrollment at other Phase previous quarter With our studies. performance. ongoing our inhibitor the to time. cancer updates pancreatic and activities solid In guidance and X Phase pandemics IDH and of impact over site have second trial, our that to arms on continue tumor discuss Darrin trials, and and Like our to this to we to I'll cancer. other lung monitor cell glioma

Darrin Miles

to that scripts in of to net and new diagnosed the our in driven increase second continued IDHX of not Quarter-over-quarter increases AML, with the AML and in observe intensive physicians frontline of academic in by research a therapy. in and refractory with the QX. growth a was use majority share an QX. from I'm strong both frequently related deliver continue and Chris. TIBSOVO to care Access XX% TIBSOVO eligible target TIBSOVO in standard of newly the largest relapsed in in both to to for the the our base, [ph] pleased [ph] restrictions community Though notwithstanding. XXXX promotional recording increase of and AML over expand of we million Furthermore, be prescriber growing IDHX COVID-XX has to performance team show Market setting. hypomethylating leading of to the both consider mutant treatment quarter, and that we refills sales focus $XX.X setting that continues mutant relapse commercial a for occurs our XX% is half at settings efforts, this combination refractory seen agent. continue Thanks, approximately most patients

as support pandemic continues assistance program, times. uninterrupted operate myAgios, these COVID-XX patient in Our midst the through patients challenging the to we of

response remote our since interactions. in some to continue local work and safely as with in from field engage mid-March. our mentioned, to of customers success to teams experienced concerns growing person continuing the the have have related Though again, allowed. interact customers enabling majority with to tools guidelines their home pandemic, We've Jackie to begun As practices

million of performance to through XXXX second year now turn of $XXX full half we the our to year, to I'll a result discuss over it sales guidance TIBSOVO US first financials. our reiterate million. net $XXX As quarter Andrew the of

Andrew Hirsch

Darrin. Thanks,

by sales net to and IDHIFA million XXX% later revenue. this in was incease release be quarter Total of million second which More be quarter million, in Q consisted driven of a revenue XXXX, collaboration results $X.X second of summarize. $XX.X million the sales, Compared in Our which TIBSOVO, royalty we total can revenue for in quarter $X.X by in revenue will the I'll $XX.X collaboration our found by XX revenue. press filing issued of TBISOVO a second included grew detail of morning, offset decrease today. XX%,

as $XXX June, to net regulatory $XX ex-US worldwide rights sales we Royalty of sheet outstanding As issuance of This tiered, on purchase well record sales-based our costs. be announced net required for debt purchased to to our which accounted receive payments transaction up method in a for an from million. under IDHIFA, Pharma on rights royalty as the milestone liability will BMS price the for us million balance

arrangement. We the royalty will to record life the continue IDHIFA to the [ph] of

recorded will the associated non-cash by liability. will with the The and In addition, in increased interest record period each non-cash expense interest expense. by we reduced the liability be royalty revenue

liability in in and liability the the grew eligible liabilities demand agreement. to revenue under million, net strong driven longer was we're to interest year, XXXX, to the levels seen exceeds quarter For receive period utilization expense the when that and over increases by the half exceeds to and no QX which and Medicaid reduced channel the revenue the for balance to will the expense, royalty, continue continued Gross $XXX,XXX. have expected compared to versus range, TIBSOVO royalty was QX. inventory Cost increased [ph] which but The first which levels expect the reduced. normal the of the for returning royalties we sales more trend $X.X was XXXX over periods where for be interest by of year. is within of zero in the quarter we ended unit PHS

includes XXXX, slowed activities ClarIDHy expenses were incurred TIBSOVO and to increase startup and $XX.X reduced year-over-year XXXX, studies that stage X Selling, clinical QX questions. an please industry Ramp quarter, received mitapivat to was $X.X representing EU of QX ended spend million, which spend agreement. general second the open $X our second AG-XXX Pharma our pandemic. We Turning TIBSOVO cell and and program in decrease XXXX. $XXX decrease that cash, COVID-XX equivalents pandemic. for million addition expected of the organization sickle We the gated With and million in payments a lower the XXXX. ending of now the milestone a offset reduced operator, in operating of result our anticipated COVID-XX HOVON ongoing lower the and million, in expect largely given to for expenses marketable to the and end securities travel thalassemia quarter excluding to Phase driven study amount down pivotal restrictions by Royalty of under for a as QX but enrollment program-specific across revenue, with our of cash $XX.X combat expenses. related operating undisclosed balance operations, quarter which for engagement cash research second line milestone plan, million $XX over was fund R&D quarter infrastructure of payments in current early driven for by through the of and The R&D in the includes and second primarily million the initial XXXX. in place disease, by to compared product programs quarter will at the collaboration in the build commercial administrative due

Operator

[Operator Instructions] comes first Young with question Our Cantor. Alethia from

now is line Your open.

Alethia Young

Hey, much. guys. the and I about progress you plans color I guess, question wanted it to us to but kind a on, on said of timing congrats ask taking know, any [indiscernible] and and just know forward, there? Thank you're just Mitapivat can would move throughout color, moving Thanks helpful. the I for be you forward you my quarter. give very you maybe and

Chris Bowden

program? Hey, didn't an you're Chris. Alethia. catch, I for looking on which It's, update

Alethia Young

Mitapivat hear Can me [indiscernible] you now? and

Chris Bowden

have crucial in the Right, order on of initiate Europe, anticipate pyruvate study make and will a I next together. strictly have takes process hear indication design the through XXXX. deficiency trial commit are and file it's just be plans [indiscernible] both in you of But and to to thalassemia [ph] - you to are embarked we that didn't the a to a then so coming Okay. And box our and that opening still adult year. trial part development with check able kinase we're be where working yeah. you negotiating The in commitment so to, No, while on then to it pediatric that. cell part. a Yeah, we're sickle an looking

Chris Bowden

PKR team sorry, [ph] Oh,

Alethia Young

Yeah.

Chris Bowden

Got it. okay. Yeah,

Okay.

Jacqualyn Fouse

but to - pediatric we're And next do also those we're trial in this Mitapivat a going point. plans cell, kinase and children remain to Thalassemia year. So sufficiency opening then at will sickle we and with pyruvate development be defined targeting

Alethia Young

it. got Cool. you. Okay, Thank

Operator

you. Thank

with next from question Rama Anupam JPMorgan. comes Our

now open. is line Your

Anupam Rama

ultimately Thanks guys. question. And progress. broader the question thinking Mitapivat, one players that on we've kind taking gotten, the you for PKR frequent like Hey, do Thanks established about had as questions a EHA, example? much. How it's levers and post well for whether isn't congrats cell so for as most the against all of I differentiation, about other think it? sickle of activators, GBT,

Chris Bowden

others Hey, may in. Tom, here. I'll jump to it's want some and Chris start

I And pain ability of disease. course, for acute increasing a are at basis think crises occurrence the chest And of are looking factors. and - that experiencing syndrome, who that need. their of those their for that aspects is the as anemia be of two things, part the really Mitapivat unmet would patients really hemoglobin all

suggests to a of well potential got [ph] children science, to a for down affect science. for put data to boil number and things, the in the is NIH for competitor and regards our as So four We getting us then biology, we're as follow-on really going know leaders case to defining from with that the the [ph] clinical early clinic. years to development a to we've study ready translational lot the plus the PKR Mitapivat best data With scenario and both. we it's activators, have the

shakeout So you're long safety And efficacy that's that's data of I the profile when periods additional important. setting really I and emergence. as for getting drugs is going really and think to think time

potentially the combination has additional therapies you will other how else thing finally, the something be And may think asked a that targeted that that's in of shown that market many we Oxbryta on approval, therapy, in, patients. how depending about I be, then drugs fit that come were a accelerated [ph] distance. could increases see, on sufficient drugs XX% And about presence any of approval hemoglobin for the has of drug in do accelerated these an in to given date

improvement, that just reduction a you VOCs. step if any So the of improvement back minute not did for in statistically it then, significant show

minute, Oxbryta So have you potential say and if least look half establish that can are step going right you can a response - there's of and at those to the for you just patient back population, you at patients a there. adequate that population for an

Darrin Miles

add more to Chris out. This what is point one maybe Darrin, just laid

the he points. think most made important I

of competition, two familiar it I In have other terms we of you'll we'll indications, come be and breadth treaters, is experience, to right, potential the that product to will ultimately, because mean, the differentiation makes depth before the be in the in data, have class, down to very to going patients and hitting class advantage one, our market.

So Mitapivat to I relative considerably similarity comfort raising products. think other to [ph] with adds that awareness,

Bruce Car

And this is Bruce Car.

of Just the activators. differentiation the adding important PKR uniqueness with to is the action the of that, allosteric in of – mechanism within class

So, the This content cells, for increase directly improve the like X, the and health of improve ATP the affinity while blood the hydroxyurea of complimentary that cells adds and mechanisms energy is so, hemoglobin the we of improve to oxygen. others. X-DPG in the and the we learning we

synergistic believe to these potentially mechanisms which others we when even will complimentary, ultimately combined have alluded. So, be

Anupam Rama

Thanks much Great. taking questions. for so the

Operator

you. Thank

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

open. Your line now is

Tyler Van Buren

be related should you'll to Hey, month mean, halted, they know, morning. so, you this plan before a summer, to patients, able that specifically but enrollment and but enroll ASH. had eventually I many above reopen to to or noted unclear the in, XX it's was sickle follow up guys. you how get potentially a nine Good program, it cell Mitapivat, that which that

patients walk a number time through talk on summer, what just be should time you get to in this these nine little reopen would Thank know, study just you above maybe that that bit confidence can you. it ASH? that that the more? for for required gives more So soon. You then relatively through And

Chris Bowden

Chris Tyler. Hey, here. It's

the - the Dr. we're look NIH that the weeks. is, we the why like? that reason think they a flow many will is should so NIH to data the then next that on and that don't that a, may reopen the a stage the part several group your from then permitted And function think that because second the I what's - I think open center [ph] depend the anticipating can And communications and getting Thein's study question would what's of whether is they're how of are know, and within patients And to enroll doing will treat. reopening. going they the be consent, I is my guess be from

in really, the come number going some be for the and baseline off. an testing, then in, That have administrative come will - And can overall, eight they they're patients and then of to then they to be issue. get so go treated patients that have weeks, taper

and eight it make or early get time a be many ready things and in then they together, on in added really any in, So order ASH summer course the on of in how and add can how get time some they presentation taper will many open, bring to December. how treatments pull depend need will and of they consent weeks do open presentation the patients in to now for to and early of people terms of remainder

couple Thein come some having weeks. they they're drug. on pretty they have Dr. get to that are So confident early, experience and in next working is by And lined the going in with patients now, early up days of encouraged to the fact now open we're

a at minimum details we the So we're fingers patients talked the we'll at we about meeting. patients be more but will to know it be in, our keeping that a crossed EHA on few get able

Tyler Van Buren

very that's Thanks so much. helpful. Okay,

Operator

you. Thank

Our next question comes Barclays. from Peter Lawson with

line now is Your open.

Peter Lawson

questions. for Thank you, taking thanks the

subscribers. Just you you and impact from Thank you. COVID? unique the say can number with through anything use? And regards revenues anything and the can on talk off to the label that Is there TIBSOVO of

Darrin Miles

both haven't are significant right thus seen the detect growth and so a to Dan We’ve move new been continuing nice here, impact to we which COVID able directions. in refills its in from Sure, scripts far. of

expansion the a had are academic the actually we as well. occurring there setting, community fastest – The right. the very which that couple with both the of significant in in and a noted, is COVID, the of community associated setting, prescribers - we risks the As but in new is are growth though encouraging it's

in other label continue terms settings, shared also seen indications outside pressure. spontaneous uses. have insight in impact So we've to in show yet we've from Though yet. The then but an of there indicated XXXB of that those emergent we and AML, what many in restrictions but it's do limited the an And into the But reluctance potentially see And But initial seen is and increase with patients expect earlier. up of because we continued be times, to a it. use the ultimately, antidotes as have manifest off over utilization we of as of potentially treatment we've time, can patients that to terms of AML. significantly diagnosis haven't as some force well. home work Andrew add adoption, Medicaid could on potentially sales of utilization can as we would disease,

Peter Lawson

Great. Thanks so much.

Operator

you. Thank

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

line Your is now open.

Kennen MacKay

thanks underappreciated I really discuss the of this especially sort bit for little Thank of I could that's blood and bit that something in more setting? cells urgent you. oxygenation shifting by is red think that number for a need of the oxygen sort used the just hoping if curve the cells circulation, real sickle maybe that cell, Hi, of you was medication in red taking any in bolster acting binding, a because is blood is question. still to little Maybe tapering,

Chris Bowden

is this Kevin, here. Chris

So asking of you question tapering. are the

Kennen MacKay

drug. saw Yeah, just we back on Yeah, to of discontinuation in that I'm sorry, the sickle safety going events of cell. some

Chris Bowden

Mitapivat of in established setting binding kinase with then And gradually kinase a and pyruvate and about then if the know, drug it to drug pyruvate pyruvate activating is it, going stabilizing Well, deficiency, the got you best to is that’s Yeah. think thing, diseases. these the in you situation taper. that we ideal start and patient is all you've deficiency here kinase the

a hemoglobin and rapid can't limit stopping slow can rapid rapidly for decline taper abruptly taper. a as has and So if it acclimate to you well, a decline acclimate, - as potential more in as the do then patient

where that's really comes So that from.

Now, the in what molecule. the a of We something are avoid do. would and and for and But space non-compliance aspect want say where is that's about them think a have data the abruptly more like drugs, or of one lot whether of that hematologic likely and of missing there here don't to an up stopping other also think I problem, you sickle situation, things in a to people biology the present. that that drug dose think that, based be that comes issue be the tapers the pharmacokinetics I that's of in they’re the the you ideal areas that know the statements because the dosing chronic guidance true cell indicates be I is that diseases. we to is compliance, on other about may of versus worried

Kennen MacKay

here, make these the oxygen affinity that congrats realistic, something at landscape, for ability of mechanisms maybe little Thanks QX. to a just during to and the relates way, that of be hot Oxbryta, about the theoretical combined sense? that's on in bit one progress partial you just - whether talk Mitapivat a as pressure the competitive similar wouldn't of least, so little curve. is maybe to agents, much, there's bit And Can of it one shifting mechanism to

Chris Bowden

come new in, of is as cell, thalassemia, I Yeah, of think think on. just well, interested the And complementarity sickle I ability I something the is as we're well in that mechanisms action. combine not products think but that

with number for publications that's the real and where of There's can new you by are So be enhances this like example, in in thalassemia interest. sickle infused to you the some potential differentiation get that having you're people certainly something a that stage affected interests that terminal is recent luspatercept, as by offering more know, least drug then if saying literature drugs they that coming cells of complementarity. cell been that at so theoretical if first, erythroid late have there luspatercept

Specifically and interesting it's Oxbryta, thought. for Mitapivat an

appealing that's spread somebody another heard other gram get is gram sickle adding about can hand, that know, by And a careful on and will half patients. [ph] little they're you they the a and they probably hand up and Doc's one more PKR or in the type these hemoglobin you've you Mitapivat. cell from half, adjust a wild then gram benefit from a on If with how activation

that P-selectin inhibitor to based others, interest see drugs whether but about but I what that opportunity working address at up term that thalassemia, not marrow - [ph] and cell very to So do it's with as on our of this we talked the and single we early the in sickle experience for long other of at and and at yeah, both, major our that day for just you know, come about agent, and the I reduce Novartis hard drug potentially we we definitely pyruvate just now really with kinase you that's to looking a I'm think EU that that along, it's anemia. know, disease Oxbryta, data both in the ASH and that, hyperactive deficiency, keep think bone have because components Mitapivat just excited

feel potential patients can as a that there's think demonstrate we well. I the will better

have a to think be that to I So very yes agent grounds single have to very think we strong, we to able strong we'll that combinations, provide patient. benefit but

Darrin Miles

the in haemoglobin, [indiscernible] anecdotally increasing That the very its the is on Notwithstanding Medicaid Chris, it And, Mitapivat that, for prevents patient important is haemoglobin. And sickling. with the outcome favorable, across a approval being favorable indication. and the isn't case Oxbryta. for that just carrying with from reported treatments

carry a red important also lowers thing. that's cells haemoglobins it ability more oxygen of higher a that they there's to binding the important. affinity And very we’re But for carry very able in and healthier that oxygen. to preserve hemoglobin So the that's such is oxygen. And

Oxbryta. superior So there that a have actually we believe we mechanism of to

Operator

you. Thank

Chris Shibutani Cowen. from comes with question next Our

is line now open. Your

Chris Shibutani

Good Thank Questions you. of morning. three them. on Mitapivat, focused

any have Mitapivat Registery and updated patient For identification the updates you previously on Peak there.

progress obviously narrowing And to whether the access trial challenging? know color us make your you that centers or been of I then that the progress how that assessment three to ability know you first ago. of on an thus Any ACTIVATE an in just the described far gave based more timelines was quicker, you curious is, thought the has perhaps terms also You've months had half to data, on to sites span. get update

of work for be to of this just place are of do do you helpful them that sharing might on you in a cell know/ have, independent indication, clinical own, dependent you if us that disease, your I decisions contractually, to and NIH, to understand, the sickle optionality or NIH lot since made plans us Secondly, upon just perhaps and the had you us options have be collaboration can for this is would what in data Thanks. activities with to trials any remind directly direct tethered you the help think

Chris Bowden

and Chris Chris. world. running multiple the countries It's Peak is here. Hey, Okay, enrolling across up Bowden, well. at

working the to ambitions natural history Peak, our study, its the something Hospital. those able Children's database One data we're Boston is is pull of take to on. and from That with be

or merging and more hope is talk something into we're pulling in doing future. that So it the and to doing in interested that analyses about and combining that

the an that's variations important, sets of pandemic. clinical and to regards global So And an be for data and disease looking that. approaches. of to then burden ongoing, With the able think and and to we to and them downs outcomes, to treatment to the able hope in going development ups we're to guide starting to be be to forward - describe publish us the important challenges

hospital fact But and mean, we didn't agreement it around, this then freely to don't CRADA do regards fact, that's I which that staff, the how how being about drug, done there. depending you I and has from up at minute, in that you to definitive and remarks, there's the research and data a data, more and are the there of NIH specific how trials States back data just see you good issues have before group. that's coast to And their the my by we at we and a I and a with look development, as clinical they're the with job making get how with on can't update develop we've can't my of we queue. going point. and looking in patients fair in restrictions, of there's we're number specific around, we we at, to have can in down them is trial, we call. the pandemic moves factors a tell coast, on start, have United why talk you the the to a places, types sure and means the this guidance are more remarks. you lost Those the sites. we're know, trying And, what on assays choose on a pull for just and is any stay commented why east and to prepared guidance, south, can share amount any for if it I and about And a done unpredictability a in As west step of the things managing talked then we're I how performing understand capturing terms down clinical and lock the is, at that, that any provide when stop of they But we'll provide Bruce’s how

we're the help and that needed. as able matter way And with we freedom a of sickle forward cell on to not any data a and discuss it relationship for present from good NIH. and and very that them being to running know, and describe have we to have employees dependent an XXXX. expeditiously moving up approval you get operate in So, That's to trial and

Jacqualyn Fouse

just for trial to trials, Chris important to said Chris with that being timing second. release something team and data smoothly the One on is probably think two I done finish the is that to where ACTIVATE, make And and second integrity. how is that you in our wanted the you and stay hand, job or patients about. the is It's a want drug, trial, protocol. that to really and has have know, on able in I jump then a top spoke release And ACTIVATE-T you to And line that to experiences issues have sure reiterate just Jackie. their think analyze good that line in data. they

line has the integrity then first fast there the timing team than we that feel patients would data out out. what be said, good as that So And at I getting done it the we're, Chris data data if more and to think data integrity the to is then we extremely data were right when to important compromises identified. we that that ensuring Chris, the top window was get clinical support serious and into about those XXXX and at got finding integrity, efforts of we patient sites to part yet, a programs we've and finding and between because haven't little efforts And data now the our question narrowed timing patient also included just about see our we’re go ways first I think get that. your

Chris Shibutani

the Thanks. update. for Thank you

Operator

you. Thank

with next Citi group. Bansal Mohit from Our question comes

now is Your line open.

Unidentified Analyst

on on morning, good for AG-XXX. guys. had a Mohit. I question Hey, This James is

some data Another MATXA and PRMTX? PRMTX company Have potency for explore recently showing are you greater combo a XXX. with MATXA compared their announced solubility with thoughts they a possibility this collaboration your And what preclinical on data? seen to program and published and

Bruce Car

This pathway is ability, potential in I you're PRMTX this And Car. to [ph] PRMTX believe in terms field. [indiscernible] an Yes, Bruce this the IDO entirely referring a player is with of molecule. we the appropriate lower combination believe also be to one. could this

Unidentified Analyst

Thank you.

Operator

you. Thank

from Guggenheim. with comes Michael question Schmidt next Our

line now Your is open.

Michael Schmidt

be in was patient if to time classified as that it new that and was was frequency Chris, share? I experienced possibly been specific But a wondering that had treatment information any occur I at to prior think be one follow available a able you at event cell that on in might I related. guys, on just any little if more might on there's taking that wondering, I general. the sickle Maybe a on obviously, Hey, for high question, bit VOC Phase on data, of patients VOCs thanks event, information questions. NIH a one just up TIBSOVO. two X additional my the

Chris Bowden

didn't see Nothing treatment. active on patients the were occurred during any new. The events We VOC taper. while

is it that's didn't of ASH eight given VOC's. information. know, weeks data to additional taper at over those that way forward. course of and our six no continue ones something the new their we've move I No, achieved one in we'll talked You're is that see you to we're We the clearly absolutely excited overall of dosing. to And proof-of-concept able about which or And the to right, it's address sense there think, component so track. we that were patients of an unfortunate completed disease any trying during or eight that

Michael Schmidt

the for then was thanks. trials you and you think discussing you Great, know, given differentiation And earlier. of And mentioned know, FDA regulatory meeting that some with I pivotal our approval talked the trials. or plans about or possible,

similar pathway increase thoughts as wondering know, you for pursuing think you HB you GBT, example, I how that your guess, think trial design, reductions, incorporating those considerations? your Just or about know, taking how about, about an potentially do advantage of VOC using what clinical are accelerated in you approval such outcomes, to

Chris Bowden

and the we the hemoglobinopathies, PKR think we demonstrated capable sickle reduce of wilds on we've is chronic mainly that that activating know, demonstrated that for together that the approval, United thalassemia major accelerated - in of a team know plan have that have you you address will, if VOC. can that far. to potential potential now States, focus And interesting we to is terms the strategy cell putting when or lead early have we of We Mitapivat development clearly but to opportunity in the we so that's part clinical important now in outside regular our overall, other a and in Yeah, to geographies US. type

a reported that new can can, patient impact those, So patients. you interesting then, not into anything get as rapidly the but as part well package how as you greatest also here. I'm so The what, there potentially outcomes you saying is on as combine have

discussions working those as forward on looking And rapidly possible. as that. we're with agencies hard we're having to both So

data impact and want and to patient that the to because advocates, an The input I important that patients type thing them patients that were going drugs. do get compel to have that's really on from other they – the use impact that of will is think

unmet So hemoglobin at. factors very thinking with of need then mechanism Oxbryta. to more play. increasing action And been about think lot on has in But hard of overall. I lot demonstrated for accelerated that Mitapivat the approval looking that there's be us the and and basically something of that's a we basis we're know a the of

Michael Schmidt

Okay, now great, seen you've listing? recent also TIBSOVO. then thanks. in like is market shares if on commercial seems what AML. in the moment? NCCN both, And share what then based market either cholangio And you've Since at I'm also wondering curious and growth in share just on relapse/refractory the been curious a use if already it seeing could you question you just know, frontline

Darrin Miles

Sure.

here. Dan, So

the know setting. our physicians numbers of line into distribution population can't What are exact given research and through by don't therapy. I know, reflections you market of I have I or use penetration by - So, in visibility their utilization,

by as in and as give stated make setting when physicians kind adoption adoption statements on. utilization, do penetration continued can't seen see or by so the So that's increased settings, and we've exact - time I and you is number. increased relying what over adoption But in in about say continued I change what is frontline in different And of to I'm adoption I improvement that's what - increased given spiking the cholangio guidelines, recent utilization the we've NCCN there of if and setting wouldn't had the in see well. as antidotes we continue to in then with Yes, utilization we've terms relapse well. as HMA And And cholangio. change the of product of we've in seen the combination well. in increased use be heard it And well. as adoption in last that the surprising setting quarter

Michael Schmidt

for Okay, great. questions. my taking Thanks

Darrin Miles

No problem.

Operator

you. Thank

next comes Our from with Oppenheimer. question Mark Breidenbach

open. now is line Your

Mark Breidenbach

what's much. sounded you in patients one any very taper effect a drug necessary for giving my following for or Maybe of not a And away to dosing on about guess another morning, Thanks day the questions. left for two. why statement. a Congrats potential to why forward patients thanks really wondering helpful? on good wondering miss on Hey, progress. who just Kevin's rebound question. like they It you're appear that speculation and around worried up Mitapivat question, would I'm as without taking sickle so Oxbryta, doesn't also taper be be I'm get for I confidence can cell

Chris Bowden

based know and our know there reason for we've about you for when sticking, two we Chris here, stayed dosing, patients here X,X-DPG and don't - - to missing we on what binding healthy drug about what they after think seen The levels, enzyme dose why is look we or it's stopped at look days. study, elevated had what effects reason example, we several a or from volunteers actually ATP when the

drug dose you that some that's the off X gives not you may we to to to you is want Oh, the of think were will just that your and that see is an have going attributed then sort Bruce data, yeah, Oxbryta, us But issue. think That think have don't be a not related. look comment that get confidence are VOCs I second of investigators of So one of as miss - that. some patients their at And Phase us data there that haven't Well, and and coming gives that's things why of drug, withdrawal problem. occurred interesting it's suddenly do the some you a when you that taper? part they into drug X on to as they

Mitapivat or in to have the, all overall differentiating that I is response versus a versus Oxbryta and of thing So whether The I main for not, if Oxbryta, that. can versus drug VOC, think that available both the demonstrate a And going about we patients that to that activity percentage other will commented terms say about are of these of can versus there that's drugs marketplace. us as how in the twice whether be with - a to that that and And a we has able hemoglobin better use VOCs aspects on, if they think reason are we day, a think pill follow taking good with I've feel taking be up, the pretty longer increase then that I to. And from earlier you're compelling patients can drugs. address

Bruce Car

binding. reason doses disagreeing with the covalent is than XX are to today, going I the BIC with S it the previously individual The effect is why veiling the which different. for Oxbryta in Oxbryta.. [indiscernible] miss matter. Oxbryta the of of the pharmacogenomic The entire hemoglobin longer That's what's reason. a not of to mechanism by you permanent Mitapivat just possibly in case very [ph] covalent Mitapivat mentioned bound a Chris. days quite hemoglobin lifespan will long schedule action end between And That's the and meant And And is binding binding. are of persist to of the

Mark Breidenbach

Thanks so much.

Operator

you. Thank

Farmer with from comes Our next question George BMO.

is now line Your open.

Unidentified Analyst

Hi, Govind thanks for questions. for taking on This is our George.

the XX taper protocol the were patients, Just was If the colleagues on correctly, the I asking, titratation, theme prior VOC same remember as a with days. I think that

guys you really you there's strong willing perhaps been like your might I be surprised maybe single then, a shutdown XXX the be matter? relationship of responses. If dose that comment patients getting the not perhaps increases the correctly correctly, a of in seems that, protocol you have or mentioned VOC remember think patients? response patient you. and any days? Thank seen there? So milligram XX to I it if COVID-XX probably latter half, wondering on not really Because the during I'm you're the if that's understanding hemoglobin And that missing was had before wouldn't And to dose dosed on better was right the going happened. I where confidence those guys are two

Chris Bowden

at heard we What for just to who of that what that indeed of the had the about with the that that that's the not talked And to gram. about data increased of regards effects eight was VOC untoward did the patients to with closer in experienced have Bruce all cohorts. patient at of an in seven beginning. dose think qualitatively over a or, gave the talked hemoglobin, [ph] I to end taper, missing a that had tolerate we increase then eight The several EHA five treated the in and was have that and or the ability it I you and across And an trial two doses Mitapivat. or line

that we individual power So data the to give to want a it to, we provide the can for at we good most presentation, to because The meeting. save didn't want that presentation. ASH slide get that

as what said, small going So XX is milligrams that, patients that's does to at. cell. we'll a sickle and XXX is the something question We're XXX look to hundreds in to milligrams And think like patients about your a relationship would to It regards questions too I and whether with XX. I number. versus on to great and is that want I be comment impact oxygen can't only see the X is we patient early, additional it's dissociation going those from one, - what there the extent or it's the curves, dose milligrams hemoglobinuria of and response

Bruce Car

think some clinicians I suggestions the way always this, know, XXX. will that really, it think the may efficacy gets with they many study. across The drug remains back we individual the protocol, to went thinking sickle such from of balance are may PK the that be range thing when development. XX of to who bump of in remind be as me you doses about seeing range that of we Mitapivat, in that And go nuances safety whether pocket PKR same thing that optimizing little a for we different a you the you and know, dosing we doses to know that a we XX want the suitable profile it to But we I years safety something that well from the about - hear seen. the of and you be it's the, to the And occur know there also in there's [ph] we drug. you or thalassemia, of be cell about And now slight talking tolerated context I the are understand from safety. expansion, will diseases, pretty XXX, saw more and that that

So safety profile. now looking several of it's with have with and at this for having a years comfort I worked - the lot drug

a pretty tolerated. we question we go can and it's So very this yeah, it's there's interesting well where we and need just data. drug to more range But know

Unidentified Analyst

Thank you.

Operator

now questions for back further any any turn to this showing time. not Jackie like at I I'm over the you. closing call Thank to would remarks. Fouse

Jacqualyn Fouse

to you, and Thank ahead Thank operator. areas of clinical in again thank difference without caregivers morning. we've for we to that and world. excited you all, patients, this trials to I we the who would the close for to across today. tremendous dedication about made for I lay do focus in us far on progress To us you so care. despite you all of and year. uncertainties I hearing call for time and challenges the remain their reiterate this like look Agios joining our that would them, the I joining participate in seeing We during the at do. employees all Agios also passion incredibly soon. thank making Take our a uncertain our what want us physicians patients, particularly Thanks for this forward cannot like

Operator

gentlemen, you conference and participating. for Ladies this today's concludes call. Thank

You disconnect.+ may now

Agios Pharmaceuticals (AGIO) 30 Jul 202020 Q2 Earnings call transcript