AGIO - Earnings call transcript Q4 2024 | Agios Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to Agios' Fourth Quarter 2024 Conference Call. [Operator Instructions] Please be advised that this call is being recorded at Agios' request. I would now like to turn the call over to Chris Taylor, VP Investor Relations and Corporate Communications for Agios.

Marc Frahm

Thank you, operator. Good morning, everyone, and welcome to Agios conference call and webcast to discuss our fourth quarter and full year 2024 financial results and recent business highlights.

slides of call our the the going access section Investors website, can for by today's to You agios.com.

and Gheuens, On our today's Commercial Medical Officer; Sarah Financial Jones, Milanova, Chief by Research Officer Chief Officer. Executive Officer, and Development; call, Brian Goff; Chief I'm Tsveta Head of joined Chief and Cecilia Dr.

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

Brian Goff

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

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

on syndromes, focused including thalassemia, rare pyruvate cell deficiency, that lower-risk the dysfunction result especially and blood disruption kinase are We sickle and disease, cells, or in MDS. of myelodydylastic red diseases

exciting or a and you year share for results reflect expectations quarter energy lead ATP red to of cell action the pleased accomplishments well available blood with our on our health. we new has XXXX support Our Today, that PYRUKYND, pyruvate are of a to the product, ahead. as as from throughout fourth blood improves the increases cell activator kinase novel amount mechanism metabolism red and

the growth us positions Our value over rare long term. significant success the creation and drive blueprint in near for shareholder to uniquely both

PYRUKYND. consider the have X commercial what exciting our multibillion-dollar support opportunity for to we be we a growth prospect lead additional to product, First, launches of

XXXX. for September followed sickle in in a of and launch potential by We cell are year, this disease planning approval in thalassemia

mid-stage of to opportunities. enviable was finally, in pipeline year X strong company with year of and for an strong of us excellence early which And this advancement slide, our and for our offering these the and for record and at proven it a poised executional highly shown team each we supporting milestones of able grow on clinical the independently being execute Agios, on is is puts the XXXX executional sheet, Second, position growth. scientific checkmarks balance robust and our excellence with foundation exciting key all and track a as exceptional innovation experienced ago. projected

for key foundation programs, III We completing the meaningfully year After Phase a long-term progress in X each advanced sickle for believe for early pipeline, our RISE cell disease. And and across study in a approval XXXX, breakout we of transformative regulatory to we continue our up is enrollment building growth. our including for markets Agios. filing thalassemia sustainable XXXX

deficiency from in that pediatric clinical focusing growth. III regularly one, all and and months, Over announced next our the key with transfused. milestones. regulatory two, who pipeline Today, we drive accomplished strategically capital compelling with number and diversifying Phase not will have line commercial, top number PYRUKYND XX And with mitapivat programs the our on another number to on trial are year in ahead franchise; was X and the priorities: results maximizing potential key building we ACTIVATE advancing XXXX, three, deployment sustain patients the focus PK our of KIDS we

a line study for a with program mitapivat moment. clinical anemia, in this data the we excited pediatric in first rare Agios' are just hemolytic you and share for top Sarah is This to positive from

exciting programs. pipeline our early-stage also developments for We and some anticipate mid-

known novel activator our PK as AG-XXX. TevaPIVac, formerly For

and year-end ongoing Phase the MDS cell by to II in disease in by study expect complete Phase We mid-XXXX. study lower-risk initiate enrollment a IIb sickle in

include date intended investigational treatment Additionally, siRNA of PYRUKYND for an filing to for new X mid-XXXX. AG-XXX, mitapivat inhibition PDUFA XXX goal targeting expect or expected most file of our disease application thalassemia III the And polycythemia by events Phase sickle the study for drug and for RISE significant we vera the of September the sNDA in cell the XXXX readout of year-end. in in

for progress see, significant multiple with be pipeline in value this to hold As for exciting off transformative you those can shareholders comments, that hand have it let introductory R&D. Sarah across review me our year potential our exciting to patients. catalysts With and now promises to

Sarah Gheuens

Thanks, Brian.

late-stage our therapeutic of and where mix promising impact We opportunities can well-rounded and prioritize breadth. and mid- entry a resources our and create includes and early-stage pipeline measurable expertise depth value. programs nearing significant make market showcase Our a that opportunities

may evaluated PK Phase Phase mitapivat top are with which regularly in who have the in you seen, year. complements morning, data we children out study pediatric line second of T Activate-Kids, results activated which regularly mitapivat As transfused, III line patients our study, this announced III with PK of last in deficiency who from top not are read deficiency transfused. August This pediatric

response, of endpoint at from grams XX, stayed at as scheduled concentration more randomized double-blind study of that study. weeks Turning and baseline hemoglobin XX total greater match assessments XX to to or mitapivat placebo. X.X in than both to A The results the was than of period. hemoglobin the less patients increase XX during double-blind years or XX X old deciliter is XX-week enrolled to completed to defined were XX and X primary in with randomized twice arms Activate-Kids patients the period. age a per equal the All XX daily

arm of met. of X placebo the a XX X% of in study mitapivat were or was endpoint the achieving patients response arm. XX.X% to primary XX or the compared in hemoglobin There patients The X

for in were baseline compared addition, arm. hemolysis in markers mitapivat observed arm placebo In to improvements of the changes from the

adult the events In due mitapivat observed the and The study safety adverse patients adverse placebo the regularly for reason. similar for XX-week results were to profile not any deficiency safety double no with events proportion for treatment are in are there PK have who by arms, consistent mitapivat with of transfused. period, and discontinuations of a previously patients

randomized With available detailed we from PK findings data the pediatric studies interacting company's clinical the shape period sharing III placebo-controlled community III ACTIVATE-KIDS ACTIVATE-KIDS Phase mitapivat pediatric double-blind providing forward with with look and marked more anemia, program programs that will studies, with first deficiency evaluating of future Phase in a now pediatric hemolytic help and both disease. The to sickle Agios' rare valuable cell in for thalassemia the insights patients clinical regulators.

disorder with such no thalassemia have in for often blood experience a to options therapy anemia range and effective serious collation also with This treatment thalassemia. as Today, approved limited transfusions to compounding inherited the issues. of have that the of as turning patients face. chronic Now diagnosed lead stroke secondary is significant rare causes iron patients complications patients or Government damage, such strategy can U.S. thalassemia, a other debilitating health management and blood health patients lifelong and XX% effects challenges therapies. no organ

announced of side we thalassemia, is in versus studies and X results placebo XXXX, mitapivat respectively. this attendant non-transfusion-dependent left-hand Phase the or trial transfusion on with shown III line A evaluating beta energized In energized summary adults top the the across of alpha and results slide. positive these from

of and filing Based and patients the foundational has their studies, oral we Arabia European the energized in December, believe and the announced or approval for simultaneous U.S., potential enterprise we this Saudi Phase In on mitapivat III the genotype in medication favorable of Kingdom of Union, needs. the become transfusion benefit/risk profile a for both United regardless a convenient to for PYRUKYND Emirates. observed the Arab regulatory indication thalassemia

last XXXX. PDUFA date our new drug month, September X, that FDA the And supplemental announced we with of application a goal accepted

prevalence sickle inherited affect disease. exceeding the to EUX is estimated with lifelong and to XXX,XXX cell a individuals on This X million. Moving disorder U.S. approximately XXX,XXX across blood to global

cell Clinical is suffering hemolytic standard therapeutic organ anemia and elevate chronic urgent mortality. for to pain need of an There to disease this features care from vaso-occlusion, sickle life-threatening can lead of and life, the for which patients options for and early of debilitating quality novel disease. damage are

data significant mitapivat pathophysiology, Based improvement enrolled a Phase The on over Phase just from Up XXX positive with completed Up with this II disease RISE with results in other sickle patients well. cell after see globally, anemias shared III study, enrollment in year milestone XXXX encouraging from along study as the with we potential began. our achieving RISE a hemolytic October over

crises. us hemoglobin sickle using end improving Obtaining variety inter-primary endpoints, our trial patients In points. mitapivat's and this of allows function. alpha to and X to our to study, in assess pain cell independent the primary a feel we we are measures endpoints rate have annualized endpoints, response secondary secondary potential apply With of

approval and from results III a line late in XXXX. top We in filing expect to U.S. with regulatory report Phase study the XXXX, potential

the believe in We potential addressing pain as to emerge by has high patients at cell the mitapivat making feel unmet reducing crises aimed best-in-class better. sickle anemia, need this improving and a disease therapy

Next, in as DevatIVA, cell explored a low-risk potential brief is for being I'd a sickle treatment like to update currently give which on MDS option disease.

we milligrams, the approximately of the IIa tevapipat at for featuring initiated XX, dosages disease ineffective addresses erythropoiesis EUX lower-risk IIb Phase XX% U.S. the MDS the to This Last accounting all and study. study XX,XXX XX are approximately anemia With in disease. to we of oral cohorts the in than due all of lower-risk deliver of and higher which X-milligram cases. MDS XX XX,XXX affects year, aim MDS, to with a Phase first Viva in X in patients that MDS therapy dose

enrollment on Enrollment track are to well, later we complete is proceeding this and year.

disease. drug the treatment bringing the Additionally, suffering rare FDA indication, September, last this an this tevapipat from underscoring patients to to designation option of oral in granted importance orphan

we clinical at that, mid-XXXX. medical cell I importance sickle call will meeting. these heterogenety findings, the sickle this with disease, Based the we in having physicians annual the in In on begin the to treatment will program cell of now of the patient advance Phase to disease II Sarah. options given to expected available. Phase turn disease, With In in presented development I of emphasize XXXX, tevapipat over significant treating enrollment ASH results the multiple need

Tsveta Milanova

we Thanks, to are have and all treat this in deliver subtypes X first the the very patient year, the addressing cell With expand launch needs our XXXX, Sarah. thalassemia the these later of to of potential to thalassemia therapy indications indicated to we aiming disease, sickle By disease. Biocon populations. anticipated underserved include

lease, these vaso-occlusive living opportunity improved diseases, for to landscape oral reducing transform thereby, these our patients deliver for with X patient we a expanding biokine going therapy into larger that populations, includes are treatment company and And multibillion-dollar novel shareholders. with the and a anemia, crises VOCs By for sickle cell creating aim to fatigue. our

X with thalassemia diligently And our working a potential to near-term in areas is of for key focus. team prepare launch

executing are importance monitoring we education long-term and disease First, state and management. frequent the disease of that disease current standards highlights the of and pathology complications campaign the a robust the care of

field we are larger to [indiscernible] rightsizing a yet in cross-functional Second, team successful market. our ensure this launch

team For example, for XX to deficiency, PK at professionals. sales was our XX tasked

to the that third, strategically to educating disease engaging have grown we we sales thalassemia, For facilitate And organization actively on thalassemia are understanding approximately access. and and twice payers size. patient support

adults U.S. leukemia diagnosed approximately diagnosed paddled with most X,XXX before are There patients food. the in with

Within offering address With leukemia problems, we we can launch [indiscernible] of patients care that launch estimate we within population. where preparations. our system adult population, the clarity approximately availability the these the for patient XX% valuable claims data, of are identify initial health managed that

We complications expect fatigue. dependent therapy to those be first. nontransfusion patients that or health considered more those patients These their with for well include due experiencing care frequent symptoms are transfuse with disease as as debilitating to dependent already contact the for system

is the team engaged continuously our understanding segments this multi-cultural actively of dimensions the patient disease. the Our and in of deepening field, diverse

XX transformative in As larger number demonstrate . quality effect the biotin the dependent the therapy demonstrate launch of the we thalassemia, improvement durability a by patients, the This the treatment in and the therapy for first for treatment opportunities, disease, for anchoring to first thalassemia we characterized non-transfusion move on with first life burden. of first state potentially reducing is towards of transfusion both are alpha patients [indiscernible] to increasingly of profile and oral

people suffering as This is what quickly to thalassemia motivates lower as possible. us to [indiscernible] from the

PYRUKYND U.S., of enrollment a quarter. patients the prescription the third form quarter XXXX. of In the quarter the Finally, in $XX.X deficiency. in a million let PK XXXX, XX of have me to XXX generated of of total the including XXXX, revenue update X% the in compared $X increase fourth provide PYRUKYND launch net current In on fourth a we million a brief in versus completed prior quarter

This thalassemia to We sickle launch capabilities launches XXXX. from foundation potential through disease. the has disease therapy firm for will provide in to PYRUKYND and which XXX maximize XXXX translated into net strengthen in patients continue the a current this on U.S. in ultra-rare we of cell future in believe

the at will with launch month priority. Looking for preparation sharpen our team to its continue main the ahead, our thalassemia on focus as

We are is system. diagnosed the following and excited health driven is and The potential thalassemia. burden characterized, well patients of the of the X are advocacy well-established, well care factors: care about commercial patient This disease known to they're groups. and by

over With our new inspired turn that Cecilia. the all launch. by and around I'll energized to therapy way to we robust bring successful confident potential are In call pave with preparation for We underrate the the will the elements world. are these together to population that, a the closing, a

Cecilia Jones

Thanks, Tsveta.

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

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

driven XXXX of XX% $X.X XXXX. quarter approximately that compared these QX expect stocking in primarily revenue higher We adjustments in certain were first to [indiscernible] to to $XX.X XXXX reserves. not for items quarter million, of increase the account and XXXX. in fourth repeat note revenue million the we million and of by QX, Fourth an in year-end of These net revenue quarter do $X.X was

to be variability. compared range been on of to quarter-to-quarter the the in of increase of the million. net for XX% annual experience fourth $X.X quarter will consistent generally to lower for quarter XXXX. expected was of $X.X Cost other the and expenses $XX.X were quarter to is launches While sales XX% the basis, million disease in quarter R&D an XXXX, an million with fourth gross has rare and fourth

quarter. expenses driven driven of were primarily primarily increase XXXX. for prior G&A the potential thalassemia we in to for million an by as in workforce approval quarter, fourth $XX.X This by compared an the was of This year million related increase expenses. in the $XX.X activities commercial-related was prepare PYRUKYND

were with $XXX XXXX in Royalty payments Pharma well received million These It oncology total in is vorasidenib, quarter. in we worth announced as $X.X that vorasidenib million in purchase the in billion $XXX which recorded the approval included ads the its of payments payment royalty as business of following FDA a noting in from milestone a third from with XXXX. connection payment May agreement Servier as a connection divestiture of

of will As sales and quarter X% securities approximately U.S. ended we cash royalty fourth the retain a reminder, a cash, $X Agios $X.X on annual billion. of greater marketable than this with vorasidenib all equivalents Taking account, net billion. into

opportunistically for assets. cell financial with We anticipated will internally in externally together revenue prepare discovered and interest product expect that the launches and provide balance, income and programs and independence to both sickle advanced existing thalassemia pipeline potential expand through this biogene our disease,

date we PDUFA compared to XXXX our potential deficiency the focus to expect relatively launch to and preparing for for sight, in our be we with and PK forward, thalassemia, XXXX. in flat shifting revenues Going are

should weeks, be that quarter it revenue looking be thalassemia, launch can of factored patient initiating Combined payer prescription to for modeling form up in which expectations is with at it Regarding the worth and particularly more QX, we're partial expected everyone XXXX. access, enrollment set reminding time a therapy. at between into several a

is are to it. we X prepared Obviously, PDUFA eager September the date and arrive the for well for team

including proactively favorable cash I deploying significant and creating Looking closing, support approach a thalassemia. to trajectory future beyond, by optimistic team's potential shareholder cost our earlier described market we value, and remain In translate into allocation ability that base for managing launches focused revenue a the dynamics prepare on disciplined [indiscernible]. we as we're the XXXX about into to of

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

I to will now Brian. turn back the over call

Brian Goff

Thanks, Cecelia.

to highlight our Agios serve release from programs. commitments. devote clinical adviser company's the with has Directors a as leadership our down this Dr. of strategic conclude, team continue CEO he years, will company's development informed David XXXX, as a years Before more advancing concentrating the for of Agios' the his we of that David step serving for Board XX to one more as us news like been on February from XX, throughout to Schenkein time piece XX effective to . press to will other I'd member of has and morning, Directors. Board He

played based his Agios. throughout in marked role. it company to shaping leading was what for We the personal III we a will years pipeline. thank an He early-stage instrumental like and into clinical disease, have company anticipated year guidance mentorship XXXX on ahead is critical approval I of friendship, you've with our as PYRUJYND in has I'd exciting be Phase David and for at role our my On today. forward his XXXX. and advisory heard, milestones On sickle thalassemia, cell a launch appreciation believe and want Agios. our entire to a readout an XXXX invaluable him in exceptional organization, behalf truly we new continuing look And progress for anticipated our deep regulatory also by in the are mid- and his grateful across important of progress to contributions, breakout express collaboration level,

In to reinforce I'd briefly like comments. Cecilia's closing,

pipeline external which sickle potential execute which in our both with the advancing across provides independently existing and us and and a key launches have ability our our with programs early thalassemia We mid-stage priorities, opportunities. disease, the include and balance cell PYRUKYND very strong to sheet, expanding internal clinical maximizing

of continued us as balance value cash to the trajectory to like forward look sheet supports allocation, change ensuring I'd to milestones the creation. and open we call for to the strive committed these disciplined our questions. for key rare With We positions achievement diseases. of that, the strong remain We future

Operator, please open line. the

Operator

[Operator comes from Our Schmidt first question with Instructions] Eric Cantor.

Eric Schmidt

Congrats all the on progress.

another dollar past cases Maybe the peak updating now strategy multibillion for Sarah, batch signals, disease injury then be thalassemia a potential lever these few of any hepatocellular the to in further there in NPA? Brian, the Should of do XXXX. profile what's the or you either or been talking first you what's mitapivat any forward about remarkable I community for Looking plan How company's And year year think probably think on for the sickle multibillion there? investment that of of sales potential in or for or you've so, additional indications. construct as about dollar estimate? safety cell

Brian Goff

Sure. want on Sarah, Thanks, to start first question? Eric. you the

Sarah Gheuens

Sure.

Eric, thanks, So for question. the

we aware community the was make of a at drug. the thalassemia, updated done as for we call that have have of in profile safety the there the information we when time were were change safety new able investor we So to that that this

would drug if the change profile then we and call, So anything we we currently you safety that would make understand well. as guys as to it, update the of

Brian Goff

no multiple unmet the patients. of And in the we on presents in PDUFA clinical that that as for backdrop have all population of thalassemia such the about. U.S. those conviction second with for a Eric, just which profound for right the date to on we for we multibillion-dollar the the data therapy Parkin, approved that as momentum, option, said excited potential that we calls, characterize of And in we well the the patient thalassemia see we're now question, the position talked as on X/X long-term potential very the say have I'll need now terms opportunity why have in

a the And energize data, compelling we T the and the and combined profile. it's really opportunity at energized both look of

obviously step multibillion-dollar So that be pathway. first on will the that

past cell really increased the with of therapeutic and that some second very available actually I the cell in limitations given The unmet options. has think disease, been the need obvious been few it sickle months. has it's in to in always sickle challenges and community whole the disease high,

that's So us, too. for step an important

we as sickle and cell course, pathways up of in of for readout the And give the through to a guidance of end the of launch phases data the position getting to these this in case disease, Phase year more move III approval at RISE study. we'll point be the the it's

Phase benefit of lot cell enrolling Phase course, activation franchise we've with pursuing of of low-risk with sickle as IIb II So study we're we're MDS. in And a as PK all Tebapivat, we're beyond conviction, noted, already a a disease building as that's for PYRUKYND. our out well that the in

ahead. of lot a opportunity So

Operator

Our next Divye Rao with Cowen. question TD comes from

Divya Rao

on This the is Mark. progress. all Congrats Divya on for

on sickle cell. one Just

the details were in any trial? in year, last protocol? sickle the follow-up. portion? Is the can trial changes after have So a portion Dow of you any And on mostly OLE changes Or disclosure it tox to liver the the then provide I cell to core there

Sarah Gheuens

Thanks, Divya for question. the

in monitoring in the start world period, with how So label, therapies a already align beginning along we have so the monitoring trials frequency. when in drugs way test in for after on is period, very had the court that open drug. monitor tweak at each make in core people look enzymes the patients we monitoring the trials. our in a liver real in took call, we In the start to they we and And the the our made the -- to we of line of all were across which period blinded

So core the once the doing a of to what were period we trial. disease into month. cell sickle

programs is month of for a once everybody X across first our monitored now being the all months of So exposure.

Divya Rao

the of has being should cell, development That's sickle is diseases more then path sickle this do of forward this Is program? trial with a moving proof And the you with there? move to cell plan actually pivotal concept we into or that development helpful. before into if successful, about think how like Teva other in in explored

Brian Goff

Yes. Thanks, Divya.

not complex, on disease. unmet because, to going commercial this [indiscernible] a comment I'm one, but in think to two perspective have from longer-term opportunities State very have I very benefit again, it's high that real a

Cecilia Jones

Absolutely.

hear certify consolidated, one loud Bryan we And unmet is more one the mentioned for need is [indiscernible] need community. a there clear more activators. live with it there that as we in the options and the when than clinicians need for treatment So from about patient that think than

So for to and [indiscernible]. sickle looking stand are cell across with franchise build an currently we [indiscernible], a PYRUKYND as opportunity disease we both

compositioning going and In we'll X develop , how of specific the we're be to products the of terms data. [indiscernible] well, by guided

always, the which to As together the we'll only update. be viable see Phase Sarah need as II a to competitive I needs will develop for at the development commercially well. at that be work meet need right plan, but We environment looking we and closely see data, to [indiscernible] of clinical point will us in time, will and regulators not the very

Brian Goff

we as trial the exactly what the on get closer more of design course, looks we'll, that and start, And midyear to get clarity like.

Operator

Gregory question next comes Renza Markets. from RBC with Capital Our

Gregory Renza

characterizing don't hand team, with respond us your just AG-X enthusiasm PK co-factor the on about Great. get should specific of the perhaps into just progress the patients your to on to mechanism, that stabilizes pipeline it I ask treatment? how who PAH that PAH how Brian with Maybe stabilizer. portfolio, year. that respect work remind well of just why the wanted as and level congrats and in on it and your activation Brian, just the you enthusiasm to you're dipping maybe

Brian Goff

Sure.

pipeline We're my just Greg, really comment. of proud -- favorites of I the not The our first thing do first pipeline. among across is not the within is I'll all I'm say, children pick

development. for where pipeline good position phenylalanine, activation strength look Phase diversification as of in giving in a the from stabilizer, we development of really to the we're is AG-XXX, of as course, pyruvate similar asset, a it's forward of the now. a we're second we make phenylalanine in hydroxylase that's continued within both on With disease stage progress The respect well of I And as type to way, a which opportunity. needed in stabilizes is conversion kinase enzyme updates to the

very subset patients are treatment this a treated XX,XXX very about actively need But limited a have, population. and We're of XX,XXX and small unmet who to high they is those talking frankly, options. actually very

excellence that of diseases, discovery came what market high we unmet again, which sweet has And need, more updates. another out right look it's do spot definitely it's development. the Agios, we of you development, rare forward and the of at Agios addressed to best giving So in and this

Gregory Renza

That's question, And helpful. step a taking a broader back. maybe just

deployment seeing light you potential you market, assets, how and just in but is market for the are not internally course, of externally, As look globally. domestically, cash of especially a what your external also at active rather

Brian Goff

Yes, great question.

First disease company very said, what as with We're of ingredients all, our rare with future compelling to you just of we're clearly one bright key comments, a it, a first launch we as call my make as us ahead. reinforce pleased noted that enviable I sheet. to strength of enables our And sure a in balance I'll to, as priority, that maximize these the, opportunities.

the thalassemia with PDUFA date year. this We have

readout. potentially following up disease the rise cell year We have next sickle

So to mid- that's have sure value-creating continue other Job asked we one. pipeline. We want deliver me, those and you just our that as we early-stage is, job to in X on points. inflection make products

from your business capabilities, scaled search a particularly we then to have And evaluation standpoint. actually development up our and question,

significant also we'll be disciplined we creation. we where on we in will what to particularly with of any we can have capabilities, in in value continue disciplined is domestically, it's well. thoughtful add think and side. and do with approach. that as global company the But -- increase, very I rare would But very we're important healthy believe line commercial the disease very Agios which as look match And clear to be on well sight

Operator

Our next question comes from Greg Scotiabank. Harrison with

Gregory Harrison

be -- modeling patient investors But the lag XXXX. about these time or thalassemia an launch on trajectory and should how understanding pent-up the be initial initiating maybe in get you in access mitapivat Congrats bolus in to think that expect time some progress, focus? some there be the thinking there as in you demand launch to therapy before initial groups of discussed would in you in XQ. will

Brian Goff

the to particularly very Greg. her in Thanks, proud comment really for in commercial end well really disease for this by And and saying is organization has And dedicated positioned PDUFA September. state just entire want of we're Tsveta thalassemia, I'm year? towards dynamics prepared going start now needed which look education that arena. what Yes. the of much so you to those and on like to date I'm do Cecilia, the been

Cecilia Jones

Absolutely.

for team the launch. to we U.S. is in patients the working super these And to question. very the for to The are prepare provide by thanks treatment options diligently excited So opportunity

urgency of focus, which in thalassemia As X,XXX is our are launch U.S., the patients doctors From to perspective, the bolus on come XX% complications. don't an in all their frequent launch to and given dependent which they X,XXX initial the X monitoring you the adult expect on we've initial which increase is patients covers non-transfusion-dependent about to mentioned, guidance we transfusion their patients. on to have patients, from visit act depending status Having total. on of educating said definitely a thalassemia, -- in

them patients the the capture ensure happening for we the education course, forward. they expect best are these appropriate So as choice of to will continue for come. the treatment for to that moving And conversations make with to right disease we'll patients

Brian Goff

the mean we doesn't, to ramp. mean smart been rightsized off thoughtful launch we XX% get had this as start earlier noted we say for the that I opportunity. Yes. terms making that XX%, don't sure I it targeting, target And so-called solid has to in other of already make a but this When team, of too. And we're choices eventually -- course, triaging is

to clarity And intensive is now the opportunity, education. position. state and have launch the launch great disease then between a label once And focus we get be that and we in going we're on now the

Gregory Harrison

you to that there. helpful. investors better the how potential Gulf commercial region the region? also in That's be a thinking the launch the And getting handle should Are on wanted and I about ask opportunity? about

Cecilia Jones

Yes.

are U.S. our the opportunity is obviously, Then the We #X operation, and seeing the us. is we ex-US, the when commercial Gulf second commercial look priorities organization. for

biggest Within market. Arabia the Gulf, is Saudi the

we're As very designation Arabia. Bridge the noted, a we have working region, new to team regulatory in Saudi we've closely in that process breakthrough the through and go with

the approval European a market is It's there and more ramp related way about on national think we national is about in think region, health When price. launch that dynamics. and closely decision when system to I the care a similar it

different system, system health with the up those private care take there ensure segments as to care Health to there But will we in accordingly. in sector, of opportunity a patient However, Ministry health navigate can big will Arabia, a well numbers, formulary and Saudi the segments academic need that example, is ramp time within as commercial the the the which access within work to for with institutions from NewBridge that time. over is

Operator

comes Our from next Raymond Sandler. with Piper question Chris

Christopher Raymond

questions. Two

repeat the on PKD. guess, in I studies First,

would and some I you think Or from there? Would and this giving you of future pediatric program gate sort work? insights pediatric expect on circling, cell. Can of as a perhaps characterized maybe work ACTIVATE-KIDS in sickle bit out short just this benefit? labeling be into there sale I a potentially little elaborate ACTIVATE-KIDS-T you guess, of benefits the

seem PDUFA times, and hope all desire question all just actually of but attention And on with then on injury, the Any had this panel, liver thoughts number the question, are it this, or your not. just just panel second focus on companies this put sort you've a potential on on that a with I you know that's to panel, there for this. might -- rarely there, this be light of a a this some but would beneficial

Brian Goff

want do pediatric. both on start Sarah, you to Chris. -- the Thanks,

Sarah Gheuens

Yes. I'll pediatric. start with the

hard really very So to right? run. very so transfusion is while if that we're is saying pediatric this trials also helpful development And hard, are

have we lot have way of bringing apply these needed future will implementing along to them those worked the a learned and trials So trial logistical experienced for through lessons successful trials of and a other to to we any that pediatric consider completion run. pediatric all pediatric we now those things

So there's startup and hands-on a any huge that those of that benefit clinical will from logistics actually trial having and programs. on like feasibility fact things in experience,

lot PKD well. had from of in from with we're So adults, and PKD in then, adult is disease cell We went a to now thalassemia risk anemia that. path we're be there applied to very similar can benefit the And sickle very course, lessons of [indiscernible] a adults [indiscernible] because to that happy the the development. following in adults the hemolytic across as

you through in expanding go there. about we're the negotiation general, that, patient And population next the helps just excited any very of So label course, as then well. negotiation with label

to potential date very along been are panel, announced. In PDUFA benefits the regards happy the date and PDUFA so the multiple a way. for has the So we that

for So September heard be in would X, at we that time. we point have not having a this panel

So lot and work, for the indeed, us panels both of a are FDA. for

be you still know, with light upon I update observed it would a what hoping have thalassemia that do precaution way, just happened be think on I label with we the agree which and will if that happen. had would we may if sure the being FDA PKD in have label said, patient ready think -- assessment the for has actually with PKD. either because I highlights update fully panel. for I don't not I That we assessment we agreed And the I'm already population. in the warning truly been the what So

with current think I data. the good So we're with this

Operator

Alec comes of Our with Stranahan next from Bank question America.

Alec Stranahan

the XXXX on look and to busy as Congrats from well a updates ahead. forward us

Just the of Is of commercialization to hired level the as hopes? into sense Or at sales meetings, more both tracking about this presented is process been OPAL, is us. awareness maybe Thinking as scaling the this patient or for groups? X And the advocacy of there there? what your has energies your medical given well amongst kind your of number really? reps have you've physicians of fed from work major force with not do

Mark Breidenbach

Tsveta, you get Yes, do want to started?

Tsveta Milanova

Yes, absolutely.

and organization data customer-facing launch disease The to right launch. on way very and we as we physicians we of the we first patients, the launch is way once payers all, decision any made the way study, approach for a start rare the processing the actually the from comprehensive have a have in and energize ensuring disciplined rightsizing path organization. So button the very

rightsized already particular. We the in force, have sales

As the appropriately the given that for covering it thalassemia XX specialists PK deficiency health we've the allows totality canals, is organization ITV rightsize very actually to us double XX about cumulative well-established the size. had anemia about said, to we fact that have we

patients the can also which with connecting the community, tell been only an the and with disease, but well we From are an well but the U.S., as care advocacy and prescriber profile, community with well high expectations, both in has connected with in our field there awareness a state patients continuing for you, is options with not efforts groups. potential education very for these is patient awareness base. the communities majority the new both team perspective, the and hematologists. as I of And are aligned physicians organization as we on bioline disease the of therapeutic patient the

you potential recently, Sarah actually is from the meet the tell to those of groups I high that excitement had and very, I and TIF with opportunity comes PYRUKYND. for very that can the

us. are hard going you, are now the make we prepare. deeper to beginning from working I it's of actually that opportunity successfully So very tell we team, deeper on the to for launching we ready ahead the and can sure execute

Brian Goff

would we we course, got -- the the And date, set. PDUFA didn't Yes. be of before when the date know

state between continued doing seen then. The with team treating prelaunch And the this now disease team scenarios. for well September, a progress now prepared so we fact in we've great the are job. awareness community into-community Tsveta does. terms the as a and in has of maximizing and opportunity every as of KOL range that And We've been great know team They're the month, physicians. any

Alec Stranahan

just the then X data quick you And be on studies. if a to think totality across approval seek point, given the intend if groups Curious just -- patient approval and to the put you in across whether the PPD. finer regularly a both you the maybe transfused. transfusion one populations, pediatric warranted, regularly not could And of

Brian Goff

[indiscernible] , an That's yes.

Tsveta Milanova

you the noted yes. actually yes. [indiscernible], asked question, When an I That's

So trials X free. have who a risk regularly patients benefit completely the the total, the the across are the trial, still benefits We is profile transfused patient and of really have across positive population. the supportive data in of patients transfusion -- we

board, methodologies is significant So And across based enormous frequentist approach. trial, meaningful we across that here a an clearly in then on benefit. positive using and methodology have this statistically clinically the both results

forward patients with well. as And with pleased that obtain PKD. hopefully very we looking to are we're broader and label So for that

Brian Goff

others plenectomized pediatric this PKD, have mean kind us trial hear the data that within and is T I the the Yes. an have are And to an stories that transfusion-dependent potential is from but And know from is a in awaiting ACTIVATE-KIDS that patients. in had and might adult for Power these are investor these I only approved pyramids who as in kids some organization in relevant have clinically what general, patients. this of patients. fuels we therapy as call, course, promising

with this really and today's regulators at right our now this the that will best do And certainly with to make data is compelling, patients. we time along move available for the to

Operator

from comes with Salveen Goldman question Richter next Our Sachs.

Lydia Erdman

total pediatric to expanding Congrats This opportunity pediatric discuss potentially progress. impact is Could Lydia the into question follow-up on for previous just could a PKD. on the Salveen. just on you population Maybe how in the the . PKD?

Brian Goff

Tsveta.

Tsveta Milanova

absolutely. Yes,

PKD So of the the opportunity. pediatrics is about XX%

for option no continue pediatric PKD need all that in opportunity Sarah it's know, to trial, cyclical starting the for the mentioned, as is look disease very future. have provide we disease. us learnings in currently apply But and treatment across to we When good is with As a us, the point other the a treatment ultrarare at a potentially to indications importantly options. all thalassemia the for there development patients and

Brian Goff

reinforce to in made just This maybe Yes, XX% is revenue. Cecilia that population. but an about PKD earlier the point is this the population, to of ultrarare

But look it's that flat over light when preparation we we for again, focus way expecting patients. pediatrics, at . this addition we in of get the year, revenues the an point again, optionality for and important PKD thalassemia, these XXXX. have for particularly to So for we're treatment

Operator

JPMorgan. Romero comes with question Tess next from Our

Tessa Romero

Cicilia, evolution I a have then ramp of expenses us follow-up. about over any and OpEx, your the And terms So of just SG&A R&D brief can think way there to additional question the cadence one and there? your give in a And start. side, up on is color you the from to XXXX us into XXXX. of what financial the right

Cecilia Jones

Sure.

buckets our cost as That of any or disciplined manage proactively to basis earlier, capital continue we being mentioned, we part that. have approach. our Brian allocation we described as on as priorities said, X So

launches is the also one and kind to the with pipeline. advancing the potential back-to-back of have preparing maximize to first opportunity The prior

SG&A So for R&D, growth and coming both couple of to years. in the we see expect year-over-year

mining on see we both some to quarter-over-quarter growth variability You a but single anticipate will see do line, items.

Tessa Romero

Okay. means? And quantify growth can what you

Cecilia Jones

to thalassemia on We data in we guidance we Tsveta waited approach thalassemia. how like on We're to haven't off we're disciplined very see this. being growth. the I bigger kick doing But didn't the given very Like to we're ramp we specific said, be don't approach try positive like a for we but where to . so in scenario ourselves on [indiscernible] doing disciplined prepare to find . want in similar prepared, that, want a We a approach. mentioned,

Tessa Romero

that, Okay. for timing beyond within can a how line out regions lay then time you're thinking you about the And from perspective, of U.S., approval.

Arabia So I'm EU, thinking the and about UAE? the Saudi

Brian Goff

touch you commercialization on approach. Sarah, comment and there, can Yes. Tsveta want to

Sarah Gheuens

Yes, the timing of approval.

that's announced FDA everything. the PDUFA the So that for date is and X, September

less they don't it's others, -- will by The they a which proactively give approve. date

get of the goes amount the the along way. So it the questions round of -- process on you and how depends

we about on track that deliver. it's very being made excited are -- and We are currently the to but progress

Tsveta Milanova

you and country. have formulary And the parts after an Arabia, perspective, a of to Yes. the commercialization biggest I commented earlier, Gulf need as through approval, in the the from discussions with Saudi different access the go region will with opportunity

some the care European similar but ramp to work. is health very take to actual the So going time, systems not like how

For Europe there a as from are for partnership perspective, we well. commercialization potential a looking

be Europe, So European country-by-country it's and EMA have systems very reimbursement pricing actually decisions. as you soon well a will we well. as the approval, health know, care to we in as able navigate as

in financial will be a commercialization there and time again, between too. approval So Europe, lag

Operator

to showing the like no call over Brian closing for questions. remarks. to I'd turn I'm further back

Brian Goff

for you Well, much, today's call. in participating thank everybody, All very right. a Michelle. Thanks lot,

and are We what that a Agios. patients really long-term to new poised therapies the months believe to significant to of It's very transformative busy and create X.X time at year. we're be value into start We exciting exciting deliver for an shareholders. promises

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

Operator

you day. does Thank you have This and conclude a participation. great the program, for disconnect. may Everyone, now your

Agios Pharmaceuticals (AGIO) 13 Feb 252024 Q4 Earnings call transcript