AGIO - Earnings call transcript Q4 2021 | Agios Pharmaceuticals, Inc.

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Operator

Good morning and welcome to Agios' Fourth Quarter 2021 Conference Call. At this time, all participants are in 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, Senior Director of Investor Relations.

Holly Manning

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

Officer, You by on today's Investors for Officer; Dr. can slides access Chief our call with remarks our Executive agios.com. Jackie Fouse, Financial for Richa Bruce of Head Corporate and today Dr. Medical Sarah section website, Gheuens, Chief Officer; going and With call our Officer; Chief join Jonathan Officer Commercial our me who Chief the Scientific Dr. prepared Car, our are Q&A. of Poddar, Biller, the will our Chief Affairs; to

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

Jackie Fouse

Good XXXX thanks fourth And morning, results Holly. for our call. joining you, Thank everyone. quarter

that optimistic XX defined the creating year future XXXX, plan across of Agios I'm months of able at Today, all of As I'm our we're the teams incredibly I building reflect genetically accomplish about And for we've on had of on our to this diseases. I'm when business. solely with focused time been later, the chapter just to what together toward. astounded areas our a reminded in oncology thick were the last divest we next decision announced recently business and

for engaging provide Officer, role our launch in FDA us our physicians more week, of Richa, with first to Chief commercial On and races the our Commercial earnings will commercial as the strategy. into who her last on joins educating new team her deficiency. call is off PK heels PYRUKYND insight of approval

thalassemia Our moving through cell AG-XXX clinical PK is disease pivotal cohort, team sickle full the startup our MDS Phase while deficiency our pivotal working study. AG-XXX ahead studies, activities disease for and cell pediatric in and sickle rolling the trials, X speed also

always, continues the preclinical November. discovery out programs, to organization our in As wave back at research of on we Investor next focus which Day and our laid

and share to focused financial are during year, uncertain. than programs can oncology reduced volatile through retaining and provided pipeline, business that Last we arise. we significant with our sheet potentially opportunities markets cash development because when balance share Importantly, pursue complementary our accelerate strong against our could and financial us near while and a program, business flexibility on time XX% count long-term priorities business our strategy, remain divestiture in that by supports our a executing business repurchase more our

As that, this we continuing to research, look we're clinical turn track commercial. committed I'll Sarah. across year, strong over it our of With and ahead execution to to record

Sarah Gheuens

status. treatment reviewed which hemolytic detail be and all deficiency. week, of treatment to we PYRUKYND for the we for its phenotypes pivotal this filings. The initiate to Last two pyruvate anemia. truly organization trials approved Jackie. Thanks, lifelong year and start first a numerous remarkable webcast for initiated in new over proud adults FDA the the it trials, are our of where last we've other molecule the deficiency, approval to investor in company on submit year that others, completed began productivity kinase label hemolytic as first-in-human with with startup has encompasses The this clinical we'll three our regulatory study, been have activities anemia created transfusion PK into advanced I'll the

in of far PK us is this significant from a our over. each work for deficiency Agios, is know milestone While at we

the application we the and receive PK remain decision marketing on track Our from second under authorization to half EU a deficiency is the in in EMA adult review XXXX. for of

pivotal through addition, team not pediatric trials transfused in who In activities two actively deficiency are and are transfused. patients for our with PK is regularly working regularly those startup who

extension We expect with ACTIVATE-kids of term is to and understanding At in us PK PYRUKYND. the to priority key ACTIVATE-kidsT for A continue the to long initiate elucidate and of burden impact mid-XXXX. deficiency treatments highlighted studies ASH maintenance in share data patients in new pleased and for as iron to were ability ineffective improve response to overload PYRUKYND's the reduce as that December, we well erythropoiesis. of the

and future Our medical these ongoing we as registry the meetings. look adult studies sharing peak deficiency well insights to as forward are additional from PK extension at trials

X in Beyond cell study initiated in PK Phase PYRUKYND portion play sickle to the of we treatment At our and global UP. last RISE an we has believe in PK thalassemia, role important X/X thalassemia of disease. our sickle the cell potential placebo-controlled year, as the Phase activation deficiency, clinical as disease, and of end of well the ENERGIZE-T trials two ENERGIZE

The first all on dosed trials patient global activation is our team site continuing been enrollment focused three patients and efforts. have in and

investigator-sponsored analysis Phase At and the shared to Together, expertise extension enroll alpha Netherlands with to in both dependent UP PK demonstrate a point data of continues important RISE X from new followed PK pioneering meaningful a component. to beta AG-XXX in by we're goal disease meaningfully sickle data also the of our of evaluated in Leveraging anemia. ENERGIZE component, is of PYRUKYND novel shared X supporting set Notably, our and in sickling. we healthy at portion hemolytic Our is complete AG-XXX. impact collaborators and end advancing Phase in first presented from currently core volunteer a the ENERGIZE-T complete study both first enrollment collaborators the periods a that we ASH, the on Phase X long-term in the portion cell potential PYRUKYND being data data activation, year. has of by in their these these study and data disease the their trials, including and our the the activator, area. of our patients thalassemia non-transfusion the use NIH shared

volunteer multiple continue explore I AG-XXX over our in to ascending this Officer. to to sickle Richa, MDS. the we in associated to our of with the low cell At call in trial half plans enroll anemia now intermediate the disease Xa We of by shared cohorts and our risk year. cohort initiate turn Commercial November, to will initiate Chief plan year. IR the dose Phase first healthy this The end will Day

Richa Poddar

defined field actively team for last the the kinase first approved first physicians engaging In and our has our we Thank as educating the treatment organization you, therapy week, with exciting It's been deficiency. Sarah. launch commercial time a an pyruvate genetically for on for disease.

can approval stay we living are and at therapy. commercial disease raising eligible pre-launch our focuses on focused PK educational awareness approximately As access in investor event, efforts and education, on we ensuring launch X,XXX our people of the there have United diagnosis are benefit We of States. the to believe And patients patients, on X,XXX deficiency eligible laid have with the PK deficiency. strategy for that of out PYRUKYND

their better into a approximately these for patients. diagnosed, However, physicians given consider understanding is adults. PK provides are pyruvate patients, we regimen. deficiency under they're Support get to and diagnosed for XX% PK and available reason a treatment diagnosed the we Having that further Of prevalence we'll the XX% to people of we a myAgios Once able compelling service of deficiency, disease support. with believe the have believe Service available when deficiency to join as regardless currently patient treatment kinase it education all This launch. with is Patient are is living diagnosing

patients few ensure provide PYRUKYND, things patient to highlight. long-term works For provider drug-related and A also access, and with education support adherence. prescribed myAgios the to

patients or are resources patients, to a deductible the to myAgios connected a through them questions specialty The provide copay the pharmacy support privately navigate adherence ensure help single who prescription PYRUKYND. commercially to eligible, is insured seamless For Patients experience. and manager smooth a can lowers a insurance program on immediately $X. patient processed to start coinsurance give

bridge assistance and PAP. patient patients, For eligible programs, or will uninsured underinsured we offer

optimal said will achieve mix have majority studied. XX of Medicaid take we disease to Medicare be we lagging exceptions the to Prior and process and that up patient obtain with comparable weeks. months and achieve with As differ coverage X the depending approximately on behind. products will to coverage, to before, it coverage rare make that This can to need in fastest to takes our patients expected PYRUKYND, expect is the about formulary insurance consistent medical the which patients Commercial we will typically XX order optimal navigate provider. to

revenues the first few dynamics, we limited these Given anticipate quarters.

interact to with that attention conditions team's are with addition also comfort In time. patient have pandemic on and this payer the current seeking cognizant impact our ability to we the physicians medical at dynamics, levels of

situation, As team our prioritizing a this expect, care direct and has are with mind. acute patient of developed you in thoughtful interaction would physicians cadence the and most

our as team few physicians fashion in appointments engage treat who PK approval. the that relationships place have we we quarters, to been a for deficiency with has Importantly, now can past in and have scheduled established timely that ensure we

close social Additionally, help patient support approval with webinars, terms disease. of of raise navigating to to patient the media advocacy community the we PYRUKYND channels, collaboration provide through awareness the both and and organizations, in also are of connecting the with

success connections we've that, made quarter continue base help financials. Importantly, it to genetically the us With we our commercial Jonathan review to to for I'll turn of to applicability knowledge strategy, as now PYRUKYND the will diseases. defined our up over other and fourth set expand

Jonathan Biller

Richa. Thanks,

fourth XX-K we detail issued included and release in which the be full-year later today. filing found summarize. can results in will Our XXXX our this quarter will financial be morning, press More I

operations discussion quarter our results oncology basis, from only a our comparative As continuing financial our a divested reminder, business. fourth includes excludes which results on

R&D increase largely This $XX.X expenses. education, increased regulatory full-year the and and including UP, fourth represents all for and increase the to expenses for Selling, XXXX. by filings expenses the offset fourth full XXXX. XXXX, pivotal spend disease the million million year were RISE $XXX.X workforce administrative for increase driven studies, prep sales million ENERGIZE full-year R&D. and over million in and training driven PYRUKYND field an was million $X.X EMA compared year for operating by This across R&D ENERGIZE-T to build, PYRUKYND by and ACTIVATE marketing. a $XX.X and quarter, of close million XXXX, $XXX year-over-year Turning general $XX.X quarter costs full FDA for including primarily and startup were launch of outs of studies, ACTIVATE-T and

to expect and we sale and and able We our our ended which flow equivalents the also the line securities gain business statement. balance, is the within quarter through million need be cash $X.X marketable the included operator, in royalties oncology approximately this We $X.X positivity major $X.X billion. recorded on TIBSOVO income With for of cash, open fourth quarter income plan line to With catalysts million from the of full-year in XXXX, that, additional for operating items to questions. to of raise cash please the execute cash current equity. without with

Operator

Instructions]. [Operator

Our comes Marc Cowen. first with Frahm question from

Marc Frahm

Richa metrics importantly, looking about? with launch meaningful I really start or going first inform which be two, going to just at us be intend launch. I most not of to to and guess, from But to to whether do what are metrics a those launch well? not And do – gauge sales Maybe you the you going very the is the you're going understand quarter itself. perspective

Richa Poddar

of that success, we've terms way informed for The think pillars provider by about talked it patient journey. to and three is is the launch in about the which what is important

includes ensuring getting are able and patients have eligible that that they on on to then and accurate stay therapy. access diagnosis, like So, ensuring PYRUKYND,

the in disease, track sales underdiagnosed show because terms be of we and treatment mind, it. we of about net how in a thinking how think patients is that's severely way intend kind that's for is we how progressing, will PYRUKYND, terms the are the then similarity So, the useful this to getting coverage is and in that to launch disease are what and with metrics the most formulary on improving how of

how strategy, over launch next with so line is in up that much we progressing we very set how can quarters. see the we're So, few the

Marc Frahm

clinics said, how Just think having and that. into recognizing Where in do coming COVID you that, terms comments, on in is as things today some your it you like is of as impact or – patient PKD be [indiscernible] you is like impacts them, into decline? do clinic that hopefully that may previously the expect to frequently where go offered to COVID a coming typical diagnosed patients

Richa Poddar

variable, Matt. It's very

points disease in the the can what past itself various talked present patients, we more the seen So, over we've at time. year the and when caregivers, disease study for and symptoms and to providers,

So, we've are seen regularly they transfused. once frequently, seeing everywhere more their from more seeing a year to physician if their physician

there's broader think, as now, is number they when sure given therapy, to a aware physicians or their diagnosed goal already patient doctor's as In are that about drug as an PYRUKYND many one see the into terms is are not. the well make exists, that their patients as of possible, they office right that's and us that the caregiver community approved having physician, why will for for are I that that so conversations eligible coming whether

So that's next will now and goal continue few the goal the the quarters. be right to for

a approach happens. that that holistic very ensure So we to are taking

sound so team but product. surround they're the we out as So, well the it's also raise have not in having social around the digital disease create a person robust approach, through can prepared and field using very to that interactions, the just but conversations, those of as awareness availability we that already there media

Operator

Our next J.P. comes Anupam Rama with from Morgan. question

Anupam Rama

Congrats again a week. the question. last approval quick on got I've

to which to are is portion cell supposed given Phase on intermediate in XXX later from is So I start focus X, what MDS low the the you hoping year? Phase the really study, the of learn sickle think X

Sarah Gheuens

Sarah. is This

sickle So also context disease general. obviously, but to in to cell portion, sickle disease, any the in in cell applies at anemia hemolytic looking we're the regards it signal of

will Phase the we're decide that to in Because and gives healthy study the that component a volunteer X. Phase of to us us on lot it optionality on about component, cell that And MDS context. sickle that lot of of give beauty that so, based do disease of course, the allows next of program. in what excited a yes, optionality trial, the move And that's to very to X it way. of And MDS us the

Operator

from comes McKay, Markets. question Kennen Capital RBC next Our

Kennen McKay

that about cell, a study the Congrats, X UP what of with Thinking us? that us you and really the for PYRUKYND. maybe we looking in little Phase sickle can again, bit should portion on be of what into the expansion help approval for inform could RISE

Sarah Gheuens

One The crisis reduction focused endpoints. cell looking is other pain hemoglobin is on RISE one UP in study a has a vaso-occlusive primary via crises. and the two sickle at response

and additional information of also patients on and to anemia is secondary provide in endpoints goal hit study the vaso-occlusive therapy the of very course, this that and So, disease, components is, both which this function. important component a two that feel are can hemolytic component how the the provide on

And a set up, it So, gives UP. to of also obviously, the – RISE from is therapy we cell a via way provide after lot get that results in us trial for optionality well, the the meaningful disease sickle different hope endpoints. we

Jackie Fouse

portion what Sarah, dovetail then Phase for to of wanted Kennen RISE X. specifically we're the would looking that from X Phase into know UP

Sarah Gheuens

measured safety X For at and doses and the hemoglobin via Phase we're X Phase There efficacy, as placebo. endpoints. tested efficacy specifically, in are that looking two

to those forward us X. allows move it so And select into Phase to

Operator

Fitzgerald. Alethia Our next Cantor Young from with comes question

Alethia Young

externally. guess talk I you now? might when little more done or little the opportunities day thinking or things you what are to seeing on bit, – just, about to not think to diversifying internally, a development oncology guess, And need you you're reflecting how question divestiture, obviously pipeline? that? about approval. specifically business guess is How R&D you've on may I the you're again do do can what my and right a I may but you the the think bit Congrats add just you're

Jackie Fouse

may I'll and start to jump others then want in.

into idea outside approach that have of given as our better see an ongoing I of the have today. with that forward. out other the our one evolution of insights think or cellular programs I things ways internal may potential our of insights is, some innovation with more Agios, respect of metabolism, progressed could to I biology we mechanisms even or complementarity think targets And that made first the we would the progress think we've moving I point biological we and of

stage that's development one Agios thing. sweet translational here, early for the to or stage least probably now, assets just team, as medicine, well up expertise felt doing our I our of on thing. – sort at always business given preclinical scientific clinical So, at expertise, think that as of our clinical is like development We've assets, spot

clear of the category would think we would diseases hematology, pretty be non-malignant classical standpoint, that it's we to about opposed would that over metabolism. spot, think as as an more we errors that's where be later sweet be few learned the or as with in So that a team, think we've probably of that research looking as that our most things. typically stage would clearly years other mostly even well. not add And inborn it, therapeutic hematology From stage discovery would probably call we at, doing I But be our value. and last I area the we well be

from point, those and resources manageable ways, deals partnerships. things. to about of talk at some including other spot So have. standpoint, add of targets in but of that we'll be we a we on partnerships would think would the working variety able companies in some to research be with sweet with a hopefully, that the a our we're And within we're financial be And pipeline totally few keen probably our that, quite mechanisms potentially or complementary to sorts

Operator

Our Bank with of comes next question Harrison from America. Greg

Greg Harrison

the Just two launch. quick on ones

idea trajectory pricing the First, research of what into you once swing? announced what could their on launch is then, put far to expect from PYRUKYND, are of doctors to week? your feedback get just does as enthusiasm you for gotten trying patients we full eligible And have point that an this who on the the what to payers last sort as indicate

Richa Poddar

things a here. So, couple of

post-approval the it say, to of in a payer how as the they positive. conversations the disease. the context in they've well prior disease, I had as having as very should launch, valued unmet was both We value understanding with good of and context up the well clinical with So the price as been then came of both very concert, how need data, we've

all the much that we sources. coming so received were up reflected all So, from price we feedback the approval, with that very of had had those that conversations ahead a we

patients to our a can but taken we've considered is, innovation. to So, price. treatment, access are we that We've invest optimize what for ensure and to holistic continuing access go in eligible so are pricing very for philosophy also approach we overall that

So reception positive. in that context, has been

So, not pushback. we're significant anticipating

XX grams not allow than one population, that that's have were I about might about think and talked there that broad in not. as than which per restrict does did patients, hemoglobin for broadest happy use particular adults broader thing to double label, the could the pivotal deficiency. about. we is with use these the both a some is we did we worth deciliter. in although have have in included very which And the patients those a be that patient before, PK mentioning, to we've better payers a populations, that greater But patient very label always It's as they our well [indiscernible] because patient trials, population of we're X% registration exclude label have with – XX%, possible

So we're the quite positive. those be the payer having. conversations been dependent, are but will have It very conversations

overwhelmingly options. treatment concerned, I There and think launch pre post As physicians any both positive hasn't been are as again, far discussions.

of kinase nature. deficiency discovered case And all treatment in XXXX. options been The the have pyruvate supportive in of much was very first

and see approved you underlying is impacting to chronic the symptoms. really control actually that only or you're disease. trying with that's the This treatment cause So, first the the molecules

patients, trial will payer six to trial is we some expect of patients. majority kinase the So for timeframe. we the we the to their Where of come of deficiency. context the vast pyruvate they it in reauthorizations which which the is in the dynamics majority about vast believe But in in that of month expect eligible physicians it three

Operator

next comes Oppenheimer. from Our Breidenbach, question Mark

Mark Breidenbach

trials, not XXXX catalysts. sounds really and overlooking ENERGIZE year is to wanted like accrual the going Since RISE patient clinical to a UP any make I we're in just upcoming sure of be it

or be maybe specific to AG-XXX second broadly in if sense then of how in I'm a clinical partnerships academic you this a you sponsored the year? question there partnership pivot should PYRUKYND really or expecting the any in is of have So are looking territories the kind we from Europe? if data would a ahead they're consider any EU? have And also, distributed in And potential PKD commercial of clusters new in patients or from launch for distributed. wondering are the studies EU, Europe if made patients

Sarah Gheuens

first second on the data we take I'll is execution Richa And to the question. question. over are and to you're RISE now. I'll very This the right UP regards for ENERGIZE then Sarah. it right In focused for hand catalyst, that

us to highlight gather a However, conferences. that over it we for on term continue such a allows important are appear, maintenance extension long to the just and we does at other ASH actually pyruvate for signals And data. upcoming that will response because communicated what that data both take time would on iron as to to it is overload we've thalassemia around communicate at continuing kinase at medical deficiency time, really look longer of

to continue And we're a that. on. year big super for things it's communicate we will excited that you're right, But on So, execution. about like us

to more So, yeah, come.

Richa Poddar

this all your question, with So, And in in of Europe right, approval environment will to about before in Europe, so the towards year, to then, anticipating XX of June year. terms we the sorted. and the of months is filed end take of reimbursement way it last address that pricing are XX timelines, we works

therapy our to so. the ensuring to We need do patients patient ability that to and are committed has access that access

is we evaluating in do options to US US. that commercial the building to how the our infrastructure we that terms outside of So, continue best approach. of of all a maintain are And the not outside best

patient per to population, little area. talked as some se. [indiscernible]. those have It things most to centers a make to could with it's evolve. decisions partner regards of about, of but population partners. don't quite see that excellence, not multiple really partners, for It's concentrated but be in the US, are clusters is bit And some So as of for are similar part in one you then, you we a any the the patient time looking we There the or

Operator

Question with Our Richter comes from Goldman Sachs. next Salveen

Unidentified Participant

is This Matt eligibility then on strategy you patient bit elaborate for one expand primary if [ph] just met? Could pricing PYRUKYND the for sickle And Salveen. only endpoint on is little also in cell? on a

Richa Poddar

because So, Sarah talked it's about and analyzed rate the before, primary we have believe we the two program. one vaso-occlusive available hit primary right? compelling of a primary primary with if those only endpoints one endpoints. it to disease. allow gives only of two competitively is profile One in us is address very of other you crisis, today both differentiated the two challenges and – sickle treatments hemoglobin endpoints, the cell the the So, that We

associated and how pricing a the data have to it options think of that. we once about we comes So with lot

us. So, think cell for as we about optionality sickle

price we and Have the hit have salvage of wait We overall. just But maintaining to we We'll the a only which another – But, even primary creation, see we have with you we have show data still approach, have of And if very again data consider hemoglobin, approach the cell, cell of data, of a to wait data then pricing what data suggests data the package, the considering to to patient what overall, how for sickle a very would one will pricing price. lots approach example, decisions. the look community, across then talk the three price value the and shows about positive If depending what is patient could broader determine take size the we will that data, similar sickle overall, a that take indications. think think see holistic population on the given they different we optionality. see take in a what think we approach about PKD, how full to and shows. to again, context holistic we is. we the in and make at and population. the want to if the for how the them disease cut we have some have [indiscernible] for all best evaluate of endpoints, then the it option have and a be then go determining overall the And you

Jackie Fouse

it's and I disease have those think way label make time, with the you're you rarest then right? how clear, always you it flexibility your to to going to over start expansions, market potentially deal over you is I a just this better more through these want indications where sure label abundantly multiple When like drug with make on potential because expansions gives price with time.

each totality based comfortable clinical totally we're how on So and of for with the where indication. we PKD data in are the

play that, time. price As with that deal would out, we they over

flexibility lot have regard. that of we So, a in

Operator

question Raymond Danielle next with Brill from Our James.

Madison Suhr

to you drug Danielle. I It's been wondering prescribed out. said play probably distribution was take Madison week, believe two for or I if just yet. have any the about for a patients would it weeks last

are changes any that? seeing to if you So

Richa Poddar

interactions. been We've having

approved physicians. Right? As of still lots early positive a just Thursday. but got reminder, with on we it's payers interactions So, and

dynamics are patients You're positive drug this weeks the a of doesn't are physicians right, about reimbursement option and excited gets early be on standpoint, from physician will drug treatment having channel impact But se. about but it take lots weeks two per will to formulary. available. – to XX prescribing momentum, till X payer behavior two on weeks, in But within

Operator

Our next question comes from John Newman with Canaccord.

John Newman

for taking question. Thanks my

titration this is nothing dose new. PYRUKYND. a for on Obviously, about question We've Just this. had the known

profile dose nice the in establishing job a pivotal done You've your studies.

available you of any of on of effect much probably that if simply adoption and to something have lack not the and Just experience, it's might an disease or the the where is have therapies curious this physician's severity likely the the think because titration of effect.

Sarah Gheuens

This is Sarah.

and So, in dose part, indeed, the used dose we have methods. trial problems a or they you what seen which in with pretty titration we've heard, mentioned do program, issues right? So what any titration our exactly is having the to are a to his haven't of hematologists with medications last clinical variety

straightforward So, this concerns. pretty titration, any is haven't heard actually so dose we

Operator

I'm further remarks. questions. Jackie closing currently At showing Fouse I'd turn you. And Thank over back no to like this time, for to

Jackie Fouse

Thanks, Thank of this all Thank questions the you, great morning. you much. everyone, for very operator.

especially As thank would extremely PYRUKYND passion making in our Agios the patients, caregivers about patients. and and I us for clinical to the colleagues do also always, across want their excited with We and all by and all many thanking approval wouldn't ways, end trials for those and of to We're caregivers I of the partner without difference be here so couldn't this PKD. physicians. my dedication participating of physicians in patients, we a like who indications. for in

Operator

sorry, I'm have wonderful at time. we've a concludes everyone, day. lost this Jackie for today. like Thank joining Everyone, you, us looks it This today's call. And conference

You may disconnect. now

Agios Pharmaceuticals (AGIO) 24 Feb 222021 Q4 Earnings call transcript