AGIO - Earnings call transcript Q2 2024 | Agios Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to Agios Second 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 of Investor Relations and Corporate Communications for Agile.

Christopher J. Taylor

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

You can access slides for today's call by going to the Investors section of our website, agios.com. On today's call, I'm joined by our Chief Executive Officer, Brian Goff; Dr. Officer Chief of Jones, Commercial Financial Medical Tsveta and our Chief Officer. our Milanova, Officer; Gheuens, and Research Head Development; Chief Sarah and Cecilia

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

Brian Goff

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

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

novel cell report data QX quarter. improves recent key Our continued positive multiple which clinical I'm foundation readouts action, has mitapivat's momentum positive our this driver we our milestones with key to delighted and mechanism a years. health announced leverages of of blood that in red in overall been

ENERGIZE-T III with the secondary of endpoints First, transfusion-dependent that the in alpha- and all we announced Phase mitapivat both adults endpoint met study primary key or beta-thalassemia.

efficacy oral disease-modifying an of treatment is ENERGIZE-T demonstrate alpha- III first the and transfusion-dependent to Importantly, beta-thalassemia. study Phase for

Madrid, session. Hematology mitapivat are presentations ENERGIZE III X by in of not recent KOLs presented Phase in were beta-thalassemia the with transfused leading positive the at Congress study including from data European regularly alpha- adults plenary Second, or who a Association in

of all mitapivat based from plan submit XXXX, Taken with seeking to together that people end of label ENERGIZE-T both living an all on sNDA from we in covers for thalassemia. ENERGIZE-T, data the and subtypes ENERGIZE the a by data with thalassemia available positive

well multibillion-dollar deficiency thalassemia potential ACTIVATE-KidsT are prepare of first activation franchise, Agios, mitapivat commitment to data details net we Phase for in a the and potential as ability XX% upon top sales and for through independence Royalty the to structuring of agreement receive line regularly study both like upfront who in has of children $XXX we shareholders, just to our Agios, Pharma. study as to Third, Study opportunistically our an a this FDA. disease treatment of of expand representative for PK for children rights terms in on us sickle as pediatric with diseases. financial will announced directing Under approval by announced is to a build pioneering sell vorasidenib agreed revisit morning, royalty rare of a Agios potential III its the transfused. from cell the we Pharma. a U.S. for and pursue fourth, a the provide process to the our will the payment her a that Agios vorasidenib agreement, and Servier's thank of the would tremendous with and for a Agios transaction This with truly launches PK win-win Cecilia mitapivat I as the Royalty pipeline. And will This mitapivat, of moment, and million

on have are additional to top the completed and the milestones forward few end progress readout disease just III look and a several year by reporting to the in progress, also R&D to data detailed mitapivat including minutes. year. that of the line sickle in milestones, in our enrollment Phase update of completing cell RISE across of next a UP provide portion report pleased we this we Sarah Study, forward upcoming We XXXX. Building upcoming look this and enrollment ACTIVATE-Kids Study we will on the in in

the across has as preparation generated by greater the detail deficiency an we've of unmet data compelling encompass studies potential on data PK and launch is areas, opportunity on subtypes, the and will believe sickle in as disease update mitapivat, in program our course launch bit. ENERGIZE to for of our mitapivat and red cell development in as for we XXXX. launch, target the Tsveta anemias well need robust mitapivat our in all disease become expanding launch high commercial thalassemia thalassemia organization in and provide a actively the a the consistent as hemolytic franchise. Leveraging transform in each observed potential the XXXX, together in market multibillion-dollar cell of in U.S. preparing multiple blood the team's of thalassemia in which to mitapivat potential improving ENERGIZE-T well health the positive and current a a just Given

our the delighted to part of this morning, news related commercialization we PYRUKYND outside U.S. announce to As of updates release were the X

from and Designation entered Medicine we've Saudi has a the second, agreement region; First, into Breakthrough NewBridge a in received distribution Gulf with leading FDA. the mitapivat player Pharmaceuticals,

and of turn the as Cecilia, Finally, we bolster cash position our $X.X million call strong the second further approximately billion to potential with over cash potential the to in hear quarter on $XXX the balance a vorasidenib. With investments additional cash with Sarah. and sheet, ended position that, from you'll by FDA upon the I'll we approval an have

Sarah Gheuens

from to who mitapivat we very this of III importantly, morning, population, Study study report line of for top pleased in transfused, with study and first III Phase children regularly announce the first are is completed data the in pediatric Phase Agios. which ACTIVATE-KidsT PK were This data the Brian. to Thanks, deficiency, mitapivat

we in mitapivat children are not deficiency with who PK that of pleased enrollment in regularly also transfused. Study completed have are the ACTIVATE-Kids We report complementary to

and the top Studies. we'll to data from this available from report review ACTIVATE-Kids study, Next second we line year, and aim both all data ACTIVATE-KidsT

ACTIVATE-KidsT So turning data the reported today. from now to

and XX% deficiency, total and deficiency As are population respectively. this Deficiency III transfused the pyruvate PK X ACTIVATE-Kids, ACTIVATE-KidsT approved transfused in depicts, for slide represent with PK evaluating no PK Pediatric in regularly approximately regularly there first and includes patients patient Phase studies Program therapies our Children pediatric not pediatric kinase deficiency. of

of total trial. with either A The XX placebo. patients or twice Turning announced for enrolled consists the line top double-blind placebo the mitapivat the daily completed to XX period the XX ACTIVATE-KidsT study, were of patients followed to morning the X:X randomized extension by of XX study the the a XX-week patients arm results the an in this arm of the in open-label in period. and mitapivat period of XX double-blind study.

red volume an duration period, arm. were and historical in response mitapivat X as response, using news the of with key were study, from arm. using in endpoints release we week transfusion-free placebo through week data As from XX.X% XX reduction the weight cell the normal study by total primary reduction arm or compared and to than results normalized patients double-blind moderate adults. The prespecified criterion transfusion patients in equal only of in the standardized achieved in as observed met in volume the meaningful, our for hemoglobin the weeks. XX.X% endpoint to morning, response Bayesian patients methodology, not X statistical secondary of this XX ACTIVATE-KidsT low study in weight transfusion by borrowing or was in blood XX% ACTIVATE-T transfusion drug response, reduction the a more the of defined mitapivat clinically was Transfusion the this in actual and noted compared

with and hemoglobin a hemoglobin least concentrations blood cell regularly mitapivat a arm the the once, the normal in within X mitapivat mitapivat endpoint response the transfusion-free response, X a X previously observed nearly the defined as weeks proportion XX% compared more safety arm, the PK achieved for for arm through to X profile were red the no mitapivat a the of patients to mitapivat adults measured higher transfusion-free patients, in arm transfusion. Safety normal to patients secondary transfusions, addition, limits deficiency X had arm in placebo which the of X response, from hemoglobin week period. or arm, in results with in XX In with were in of transfused. achieved week after placebo and double-blind response who placebo compared normal are compared at consistent

the discontinuations upcoming study, studies to study due cell to Phase proportion pediatric of analysis meeting. of the events We data the make impact that medical III detailed hope anemia, at to an period patients sickle adverse lives a hemolytic mitapivat this present several a facing in plan the arms, We thalassemia and AEs. of of first placebo rare a ACTIVATE-KidsT the of positive During in disease. and the children be more treatment study will and no of were treatment double-blind similar had there including

University of in and June by health-related Ali the life the overview Turning an from measures an was III in Greater efficacy EHA patient-reported presented Kevin Dr. to and a Phase Dr. thalassemia, study investor in presented our the Center; ENERGIZE provided in the Taher from and American from data detail of outcome by progress plenary and poster at a Medical session University were on meeting detailing the in Kuo Beirut webcast. were safety. of Toronto quality of

mitapivat including multiple on walking of experienced with treated fatigue patients and improvements quality Importantly, measures, health-related life capacity.

transfusion-dependent ENERGIZE-T month line of very in we transfusion-dependent last thalassemia, to disease-modifying beta-thalassemia. were which mitapivat addition, therapy the first demonstrate oral and top Phase mitapivat to from positions in the In study as alpha- data III efficacy pleased positive announce

placebo, with extension week the reductions results patients XX-week patients There or study XX% also of in to of followed secondary in transfused secondary double-blind endpoint more design Treatment with XX achieved arm. of placebo of than reduction with XX.X% achieved in twice study. XX X mg compared defined arm. red XX% compared blood or with period cell reduction consecutive than for baseline, demonstrated cells highly defined more primary XX to take reduction response was red XXX Let An the an mitapivat and cell p-value units placebo. equal from the with or open-label endpoint, and reduction as with to daily of equal to of endpoints. cell baseline, X:X week any than with response, in week The moment X period patients blood equal an transfused compared equal of of patients a XX period. A as XX units assessed XXX more total through met than compared transfused enrolled, blood compared The XX% through XX-week blood remaining week now compared measures weeks XX its in through X.X% or by were me statistically in cell period transfusion additional by a more in review placebo compared independence, consecutive consecutive the the a randomized any a key XX-week an X transfusion week of or arm is that the reduction transfused transfusion than primary statistically either reduction in than significant, through the patients blood in of mitapivat endpoints units difference red with of baseline. XX.X% red equal significant units more an units XX% or more week reduction red in XX mitapivat from through week transfusion-free equal transfused X.X% the as to baseline. Moreover, or X.XXXX.

placebo incidence the discontinuation, patients a patients these adverse XX-week placebo during analysis compared medical Finally, forward double-blind detailed more events We that in the led presenting experiencing in to at of of of data an look with meeting. arm mitapivat X.X% an similar upcoming to the of AE were and period, arm the between arm. X.X% to mitapivat

mitapivat's from data foundational, and thalassemia, the for underscore beta-thalassemia become subtypes transfusion-dependent of potential all Taken ENERGIZE non-transfusion-dependent alpha- oral and option convenient and to ENERGIZE-T strongly treatment and together, a thalassemia.

year, both that covers a by people are from data sNDA subtypes file seeking track end of incorporates an that this We the of living label all with studies all to on thalassemia.

Saudi the As to the regulators Designation of mitapivat need we first medical medicine to we Designation, the life-threatening by new Breakthrough forward new of treatment and that accelerate breakthrough development program to products to one becomes and Breakthrough are together the SFDA, Mitapivat FDA. note or NewBridge. a mentioned, facilitate pleased quite receive with working of address the drugs and partner, our has received unmet aims look that review the Medicine conditions. The Brian in with serious or Medicine

of enrollment to on portion year. the progress, to of Turning III continues mitapivat to complete this we RISE continue to disease. sickle Phase be UP cell Enrollment the end by track in of and the

option to health the data positive positive II portion study, have that in Phase we generated anemias, in treatment to we for as as UP and the the cell are the RISE the for we blood red confident Given potential hemolytic other reporting the next better with become sickle in well the of a generated disease. We first-in-class patients best-in-class improves fatigue. data high convenient UP mitapivat in III living feel and for look patients and mitapivat RISE line reducing this of the crises, improving the pain from portion cell top potential forward to their by disease year study sickle anemia, to need by data reducing Phase making unmet address cell

to Phase doses novel we including on higher now IIb Finally, the our milestones MDS. lower-risk the advancing track deliver in rest remain AG-XXX, our on across pipeline, tebapivat, of named of PK beginning enrollment evaluating of activator, study, all

now are as to to the eager details call the underway I We soon. With over turn Tsveta. share will more gets trial that,

Tsveta Milanova

and have high of of because It morbidities, premature stories, Thanks, Patient to Based associated families. All living therapies transform thalassemia. and X,XXX And patients approved In as the in may market their to subtypes. There treatment therapy thalassemia the from bring there therapies generated care, just of adults thalassemia with the bring a the for families. for data of and patients Having and reduced disease Sarah. and first ENERGIZE positive there patients those Cooley's have we this many heightened impact Conference are in for their Atlanta, thalassemia are powerful thalassemia of aim debilitating of the we no patients is risk limited Today, death. on can are is Family cost in to quality studies, the oral approved disease non-transfusion on as burden no significant. thalassemia whom diagnosis U.S., treatment serious and X,XXX Approximately a options daunting rates the with the Foundation Anemia all disease, and at disease, with approved we approximately well for U.S. the be forms non-transfusion-dependent for of dependent. are of diagnosed emphasizing all. returned navigate are the heard the ENERGIZE-T of challenging life,

to with U.S. next preparing actively thalassemia is address this PYRUKYND need launch organization high unmet in commercial Our of a potential year.

mitapivat In adoption we the addition and the in believe that consistent thalassemia compelling development data have to key to potential U.S. program, in the X support are we of in the generated PYRUKYND factors have there

diagnosis of the newborn screening the in First, is adulthood. patients driven before availability well-established high, thalassemia and by ICD-XX many codes, with diagnosed rate

diagnosed physicians And clear centers, affiliated practices, treated third, of with our and patients approximately at clinical number are limited than launch. concentrated initial both trials, have XX% Second, of providers first-hand experience all since our fewer in for a XXX of with the of some mitapivat. a some Excellence focus patients already in treating providing Centers were included

Taken together, subtype. for treatment potential to with patients we a believe regardless option we of foundational are provide thalassemia, well-positioned

focused launch is As we potential toward of a the to on progress in key X continue our team launch preparation. U.S., areas

our market analysis extensive our claims data conduct to continue we refine research First, insights to market and and further targeting. physician

across share in all a little in May, disease of moment. complications about education long-term campaign we burden Second, highlighting subtypes. a for a thalassemia new this patients more campaign disease designed the both clinicians, and and commenced I'll

executing providers. messaging thalassemia. On deficiency, to healthcare state still commercial medical provided has expansion right-size forward for in access our the a Third, to our and using in market. in disciplined I'm market of our And both Slide that we this of launch new broad the same a rare, fourth, engaging larger, campaigns with the and PK strong some in on successful is already education team for of achieved for is payers proud team teams education. access and have state we organization outcome our disease but our are we patients look PYRUKYND team XX, disease the

and reset encouraging listed, working disease risk. reinforcing less monitoring messages disease the the objectives especially that is non-transfusion-dependent burden of our are perception team the to of the thalassemia With management, and highlighting patients at

positive, are graphic and response providers. I and from been see healthcare both exist, Next healthcare is images resonating with XX,XXX that very with to of burden, we patients prescribing deepen Gulf from campaign. of our Cooperation a history. the has and that recent to the mentioned morbidities patients' how patients can The with continues iron hemoglobin insights our the team serious consider to It can highlights risk On market communicating thalassemia approximately U.S., our regardless fatigue, physicians messaging the clinical of or overload. Outside the region, GCC will that a research helped PYRUKYND: this when the transfusion elucidate is characteristics Council, understanding. providers slide, top market thalassemia. you levels, transfusion home

trials. NewBridge with in our a centers potential are PYRUKYND to GCC of treatment a leading the of pleased of agreement prepare clinical distribution part we were announce for entered commercialization Pharmaceuticals in we some And region the that have to Today, into region. the

NewBridge, specialty Kuwait, company headquartered United Qatar, the Saudi PYRUKYND Dubai, will Arab Emirates. commercialize in in a Oman, and leading Bahrain, Arabia,

are the commercialization in join We excited to we NewBridge as towards potential region. drive with forces

Saudi largest the As Arabia in noted on accounts Slide the for XX, patient population GCC.

efforts We team's are Breakthrough successfully for mitapivat was FDA. SFDA, which the to Designation quite secure Saudi by proud of a the granted thalassemia, Medicine

in As local access NewBridge. price the with bullet, are followed partner, national These which key usually individual GCC look access countries to standards. the noted by forward the health our elements the set process last with level, navigating institutions, path with in the a begins over regulatory time sector, private in authorities, we at and

second patients on the provide PYRUKYND the span me quarter. from current patients and net on of XXXX. a translated is in with $X.X a XXXX, first on physicians, million now a to PK including form, therapy total compared In adult XXX This and second enrollment represents of growing therapy, versus In versus in in PYRUKYND have XX a generated XXXX, the broad a revenue, in launch net base disease an of PK the quarter X% prescriber a the U.S., Patients quarter we that the demographic XXX Let of deficiency XXX increase the $X.X and to increase update diverse of range manifestation prescription in prior the the consistent quarter completed of deficiency. quarter. U.S. prior continues has into X% population million

from patient the capabilities analytics, patients that continue providers to which the targeting of efficient with access, foundation on call firm disease persistency We continue and a awareness on that, over by and of sickle XXXX. identifying in are remain will U.S. and therapy We provide bring in maximize be thalassemia physician to I'll the inspired and all, turn PK encouraged energized current patients strengthen launch in cell to by XXXX, in through including a the and likely launches in believe including to deficiency. future mitapivat, to to potential the treat and Cecilia. focused Above patient education, treatment new these to populations. With underserved PKD, patient potential will we efficiently

Cecilia Jones

Thanks, Tsveta.

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

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

XXXX increase disease the XX% rare is an in annual of the was XXXX. $X.X with been range and to net be an XX% $X.X on gross other revenue compared to Consistent PYRUKYND of launches, million, million Second basis. to quarter second expected net quarter to has

of more growth for preparedness, were our was going the from XXXX. we we organization's thalassemia thalassemia. increase attributable $X.X sales compared forward million. year-over-year quarter focus second to was second year. an second in an the costs million program quarter As the for $XX.X increase of for $X.X to primarily we an R&D million potential to million PKD quarter for in XXXX. pivot launch expenses was approval in associated of the variability the $X.X commercial-related million The revenues. Cost $XX.X the last SG&A the as This primarily in of muted increase were increase to activities and the quarter-over-quarter attributable of quarter, in-licensed in prepare expenses an expect with to compared same quarter, increase to Alnylam TMPRSSX see PYRUKYND

to retain the we an agreement, approval Pharma. Royalty to sales this Servier's reminder, Under upfront sell annual the sales terms its U.S. Agios annual X% will $XXX vorasidenib than U.S. the the quarter, upon of has to FDA, payment billion. million billion. rights $X and sales royalty company on a by vorasidenib of potential on royalty royalty U.S. receive up and net net vorasidenib Royalty U.S. Pharma that a the As the XX% of royalty announced on than of XX% annual of billion, to the greater XX% entirety agreed Agios $X on greater net of will $X sales net receive will

addition, a the Servier divestiture right still business. vorasidenib of million to from from retain of milestone In FDA approval potential upon our we oncology the $XXX

potential So, vorasidenib. the altogether, receive of billion of in approval upon Agios will FDA a $X.X total payments

equivalents, for a the date marketable cash, been action As ended XXXX. of $XXX.X has quarter reminder, securities set and PDUFA million. the August We XX, cash with

expect shareholder value, and our cash this through financial on of product with remain of in for and a prepare opportunistically balance, cell disease, we base to payments and support creating and to vorasidenib, FDA approach expand thalassemia pipeline launches disciplined focused income, anticipated We assets. to externally independence managing approval our a proactively cost upon that interest future potential as sickle discovered by will PYRUKYND. internally launches deploying including revenues, prepare together and provide PYRUKYND We potential allocation both

am term, value-creating sheet the for additional to his back position strong will potential us toward we call that closing execute from to move milestones a remarks. turn I now strength. I to in will balance continue enable near Brian the of confident As our over

Brian Goff

differentiated late-stage novel continued now that and multibillion-dollar pleased blood to a hemolytic to data first completed mitapivat have of and by X compelling and has and mechanism readouts in a Phase become continue treatment poised Driven spanning and become the Cecilia. first-in-class cell that our III red we're action a improves positive is health, indications best-in-class anemias, trial Thanks, deliver team, multiple mitapivat and data we believe for pediatric a patient the demonstrated to on of Agios package strength option Based consistent to population. this franchise. to

the sNDA by year the covers this thalassemia, that on living an seek file Our disease. a we and subtypes all end aim to with focus squarely of people of label is where

continue disease we company, continue sheet be take As responsible of opportunities becoming steps leading vision stewards value a our we'll creation. towards realizing rare for to strive to and our of evaluate meaningful to balance

to medicines extend Finally, for the the programs. With that patients work and employees our development lives to rare our and our physicians, call like their that, and all our thank in of I'd partners, the elevate mission all caregivers hard participants developing now patients, open living we'll of transformative of diseases delivering and of including dedication and clinical with for questions.

Operator

[Operator from Instructions] comes Eric And question our first from Schmidt Cantor Fitzgerald.

Eric Schmidt

on to have continued a ask you the about and Maybe us the Saudi I'll Congrats transaction. NewBridge more Can you the talk And new give some color Breakthrough the that economics bit Designation development in what also timing about the progress. all hope Arabia. you maybe today, there receive? now on little of

Brian Goff

Tsveta And on really with large with Eric. GCC. NewBridge quite worked hard Thanks, team a this arrangement and new

most important You the first second and for U.S. important that the our outside market that's thalassemia most know

let on to going I'm that. So comment Tsveta

Tsveta Milanova

thorough look to the No, announce region. in the information agreement that's as process. When them, Saudi NewBridge that more the agreement Agios. possible, utilize and happy to full-service been into as a providing select because good as Designation with on be that numeration wonderful. represent about approval And with for soon and hard experience we we'll for very submit have And Breakthrough really, us the a to the the working we think to to regulatory you with in are partner region they're with activities Arabia are agreement we of thalassemia region, also relationships, and yield. a with distributors, Medicine regulatory type Breakthrough the represent as really we've so to NewBridge very from our approval that biggest we they And we typical commercialization announce whole the kind Saudi from this work market actually actually very of is Designation we split proud revenue which FDA, forward received the a the deals it's and across across enter basically for board being

we Agios. the continue there look a basically as need region, excited be upfront is to region. for be as really of in medical the we that are only but because And our will not about region, it. When clinical we progress there us the commercialization, the studies as part payment high there potential really, with conducted no of revenue So we unmet at have in product will a split

and to there the experience already of access in key look patients. forward region, in centers a we is provide So person the to KOL some with

Sarah Gheuens

And of to then regulatory with the around very the follow moving of of on forward program. Eric, so excited keep we on timing question are, up engagement, our to course, development

And the board. so with will can soon across regulators as in fact, be as working we the we

Brian Goff

breakthrough saying that, in I'll And just close by pretty that medicines designation Saudi is unusual.

And so need profound Tsveta that of, a have in recognition a pleased we've as to that region. been as noted, just unmet

regions. particularly of part an us important need puzzle these get it's unmet our So the we for the high and U.S. focus expand on as ready to presence outside

Operator

question next Sandler. Raymond from comes from Christopher Piper our And

Christopher Raymond

me. from couple a Just

Just terms about and could this is the guess, you're with a you follow-up But I press you've size language then M&A release, billion-plus after do. PDUFA? PDUFA, of the would noted about question. upcoming relatively you cash infusion. cautious, M&A Brian, I'd in things, August have your in sort as Can you I of what meaningful talked of in past. how a the -- your I line of scope guys changes vorasidenib's I now pretty you lever And in infusion XX guess, as say $X you're maybe and talk viewing been a has know for

Brian Goff

step. science. for I to with side excellence very an again, that Cecilia of a patients. I'm a sure. note, adds it of lot. just That's important Obviously, And vorasidenib. enhancement. as very of want balance potential And mark vorasidenib It's a Yes, did the work the to proud sheet course, reiterate our thanks of And is itself important royalty for monetization Agios

just M&A, talk just position capital puts in about efficient maybe I'll a the what. it This And for allocation I of take you capital, certainly and us. no step a strength. us So is mentioned think of up matter focus use

next important this really be Tsveta that above preparing priority And the launches. cell by else year excited that on upon Thalassemia, And a launch. comments, back-to-back the quite readout RISE towards top with complete preparing fact the again, is sickle these in noted next are launches data for team success, we then, near points our for with year term. year. about back-to-back and That track enrollment as Our all could delivering we we're the possibility had end. for disease, trial and and for UP is

#X the that's So priority.

broadly, as always But and is competitive to goes most sure analysis certainly well. of then about a opportunities well. through team our a pipeline. great market of shortage that great capital. on focus. value-enhancing tebapivat, pleased development study potential. as announce a the INN Cecilia A Phase it's Secondly, which about that's, more we're IIb great commencing internal Chris, very job example And for in making And internal to our to all sights. and again, the forward your And own do today, said, that point, advancing AG-XXX. And was what we excited by beautifully, use growing that to of disciplined the the course, That's known business no MDS. some on what for organic have And we were looking and BD, is formerly progress a we're our we of opportunities will of the make And with continue MDS. process. of that as That is our as we're low-risk very because opportunity great rigorous way, very creates that organic

Christopher Raymond

just Okay. quick then Great. maybe a And follow-up.

and applicability just patients the and all for as an we've about around Just had that Maybe experienced view talk from KOLs endpoint VOCs opportunity, some on sickle patients population. your sickle not a broad the -- cell feedback cell VOCs.

Brian Goff

that's for one great Yes, Sarah. a

Sarah Gheuens

Thank you.

all to is as intent VOCs. disease. And You're product of know, a sickle right. our treat patients you that Not all can experienced aspects deliver cell

So spectrum hemolytic work can based a and we action of of and across range cell across principle of do range We a drug anemia pain programs, disease, -- levels sickle the crises. a hemoglobin pain whole on drug, mechanism sickle of our our cell different the crises. of believe, so as the

a measure that to be you the treatment to perspective, to need effect, event you able trial want measure clinical a a enough to from of to reduction. able Now enroll if patient has be that ongoing population

patient the the all course population measure would of able effect is drug sickle year. a including anticipation to patient cell be our we from that crises, that the would to be across can't we treat cell that not sickle can trial a for perspective that thing why of criteria Again, population many be address meant review. trial of mean, all treatment on over Unfortunately, have to But sickle because clinical how is X. it matter treatment, has our into the the go that crises disease X our inclusion the that you aspects no able or I work our including So cell data disease just therefore is can or that the of measure cell X label have for enroll after endpoint generated pain in to intent sickle and pain program, just trials based the clinical to an regulatory more.

Brian Goff

to one need. try hard complex, that to cell not oversimplify aspect, point, is really you very disease crises, especially but fatigue is Sarah, these we too VOCs want fatigue, on comment which pain profound diseases, so sickle is another sickle to And maybe in cell

Sarah Gheuens

Right.

and I pain primary hemolytic hemoglobin endpoint the the cell crisis be mentioned So measure right, to and VOC point, endpoint other of reduction primary sickle able impact. anemia just a to the primary end of hemoglobin

that one by impactful And that's really and from know, of the indeed endpoint. is patients bunch we which that's focused fatigue the try other you incorporated main of because hear As supported what therefore, on patient from physicians fatigue have to very, a and as is endpoints, secondary population. incorporate all We our secondary to designs. for patient fatigue of burdensome feedback typical very and very, that's

you we ENERGIZE, key meet trial which our data, reported fatigue. the As announced -- for patient thalassemia we've measure secondary time first know, of that the did just on the in endpoint Alco

as study well. very for of are we So rise the hopeful the

Operator

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

Gregory Renza

start would is color Good consistent adult can consistent was morning, the study Any meaningful. seems effect with Brian assumptions? It The to clinically placebo and Maybe behavior I'll there have then underpowered? just that maybe as a follow-up. studies. always I just great. Congrats Was with that be treatment maybe the Sarah. us Brian, understand Sarah, for the certainly on ACTIVATE-KidsT perhaps have continued the team. you Was help you and historical statistical added progress. with largely results And a consistent transfusion rates? us

Sarah Gheuens

Thanks Sure. for the question.

to So of this leverage get have appropriate appropriate disease the is ACTIVATE-KidsT, be an we ultra-rare and to with really size a that population, try adequate the and sample data provide would we for therefore, adult development study really us generated, to to data. that a design state chose for

have pretty transfusion endpoint look the So complex a to at use methodology reduction. statistical to we primary of

that the data. a effect. the As depends some because to you you borrow mentioned, that from weights of leverage can't board adult across it's look say at on And And we the data gives pediatric that pretty specific it study. see amount your treatment we complex

or around So that's to weight confidence we moderate loss very our study specific were the we the positive while data the why adult -- the ratios, that X. include around data release in low of because intervals see press borrowing

of you're specific to clear effect if and family difference obviously that is we why there patients a become so of lot the person a demonstration is their transfusion-free, that coming X the X that your transfusions a kids, also remember, that's fact family. have meaningful population. a point, the treatment patients And is this an of That that that now they data that. To transfusion-free. specific patient trial, are who a have we for patient population, impact So regularly became X this of very observed, into because very the transfused meaning has in are mindful those these

told about because of we study, goal, this that's meaningful like course, very the be study. story to we do So out results main is why are actually a excited there The feel of this of which an to the trial statement first completing there. indication get This our program which are and right? after very about is happy we adult we safety pediatric an clinical is can't -- also assessment,

really So this patient that the expand age is our population. range of first endeavor to

that is very very about do the data that we're development. efficacy excited so feel and encouraging, perspective, very we from the the steps important, is the next have we data safety in And generated

Gregory Renza

other just one helpful. question. And That's

some and the of through value. Just doctors thalassemia, to as some it's have of on the walk helpful as could well characteristics Tsveta that messaging

improvements if the patients through. usage any the perhaps comes meaningful wondering but patients thoughts quality term, staying hemoglobin to what or improvement assuming you on patterns to life drug? the term? Just on drug maybe had longer we maybe real-world for on comes stay on hemoglobin expectations and patients it all drug longer with When benefit, less have And more the or staying of the thoughts any goes improvement approval would on of you as on planned comes maybe any thoughts if level thal anticipate

Brian Goff

lot there, in a There's Greg.

pretty in are that. But brings venue has and in ones these patients. unmet we'll continuing are the of Meeting, maybe color for keeping I question that unmet a dependency inspiring home think actually she need insights is The meetings engage heard prepared meeting towards of build so we be was of transfusion on in focused exists Tsveta the that put Anemia annual thalassemia some we're lot mind range I that It's U.S. are need. returned the on like your Foundation which certainly from patients Tsveta could the skewed These upon. because comments, the that her And on crux certainly that a and just the attend focus the And to noted -- in just to Cooley's

Tsveta Milanova

then attend to and community and start well, space, the Family new about listen the Foundation an to connect potential as that patients potential the both anemia of including little the excitement Conference, hemolytic stories But of there, and the truly enthusiasm kind of was The learn and U.S. Patient the I patient to to the Absolutely. of Sarah also being to coming approved talk And treating had inspiring thalassemia. across and Mitapivat in patients get the indication the clinical opportunity journey absolutely, and to can and the bit and mechanism our as transfer and therapies product Anemia have importantly, and it with to truly, the a the opportunity Brian broad thalassemia innovative I action I'll many physicians maybe applicability of very about Cooley's for

relevant Sarah this being in transfusion and trials being is get fatigue making consider we and you iron thalassemia in endpoints importance level about characteristics great engaged And sickle hemoglobin clinical that very course, the burden, of the decision talked was market as course, Of the overload, and research physician And Greg, cell being of and measure it patient as audience, studied the clearly fatigue. to the and community mentioned, our treatment we their feedback. educated that most they'll from all when a disease.

morbidity to As and educating lot hemolysis reach to linked patient population. both broader the hemoglobin actually developing and can a the commercial and truly levels improving and on are given preparation, are we disease lead hemolysis of that increased looking audience, actually disease, actively importance complications to we mortality space, a that and physician patient from the in

think levels as And to to more patients either for in and stages expect well importance that the hear considered broader a as symptomatic, on of patient characteristics, the product with of educate from Sarah can these lower which all Mitapivat launch. comorbidities to expand well got and And initial to we'll the about are launch interactions over action -- to the hand the be and look probably on patients business how have and we'll time. it segment look we the now would we expand that look population. And focus over the these as and fatigue. Mitapivat's that clinicians who think the dives likely they're of hemoglobin the and system, their are patient you have clinicians bit of into disease patient kind to little treating linked the clinical a probably mechanism transfused, patients, totality are We regularly about thalassemia. to the initial healthcare when they will that about that transfusion-dependent at patients patient about asked we feedback will that the prescribe. talk are in more You as as seeing given you of and to community, they when regular being I'll going When be

Sarah Gheuens

Tsveta. Thanks,

I the in to cutoffs to would trials. the like So come hemoglobin back clinical

gram we have are trial a who of clinical have our to endpoint trial, Greg, your X patient they becoming patients clinical may still benefit, population. seen but at. of responder we that or know, experience reach more there point, and that per deciliter you As bar we the some look not PKD in in the But

We showing patient to placebo thalassemia looks how showed the versus see at distribution that population Mitapivat in a the also exposed the in to like. hemoglobin exposed population. plot highlighted Taher We patient waterfall [Indiscernible] Dr.

a population deficiency before You could patient in the have similar very population. as what in patient the we shown thalassemia figure see kinase pyruvate

but So reach how there people of important, of not one points is which they is definitely volumes, is is improving also the hemoglobin clinical patients to endpoint, setup the who reasons why that are their important. some hemolysis feel trial component

over trial an clinical to physicians even the roll at decide the they and patients clinical allow we open-label why is are. still need this extension, of So end trials a don't in to if

Operator

from TD next Cowen. for comes our from And question. question one next Divya our And moment Rao

Divya Rao

Divya for Mark. I the the ACTIVATE-KidsT. had is quarter. This a on on question on Congrats

I could any And have Mitapivat then at if AEs the trial, arm in you follow-up. in in comment there changes are on sex placebo? hormone Looking levels the the a versus

Sarah Gheuens

question. the for Thanks

the So not in press actually deeper safety beyond release. the gone what detail have out yet all put we on into we

upcoming look forward doing an medical that meeting. to at we So

Divya Rao

expect And data. then that's... on you AG-XXX, see I if at would or the would we if was be IIa meeting when Phase a could medical when curious to it -- And

Brian Goff

First, Divya, heads, burned but in practice to we're try have the because And our [Indiscernible] Sarah to going [Indiscernible] tebapivat is same because to XXX do we saying

Sarah Gheuens

still are Honestly, numbers it's well. in burned the as head -- my

so yes, But the switch. focus is I if apologies and as current mentioned, IIb. our milestone So Phase

So focused that. is team very the very, on

appropriate upcoming IIa we at it will but time, at be presented an So data we declare when will later medical event. an

Brian Goff

as Reblozyl is very reported And here, quick but us. now for we're which clinical as a novel patients. need of case would this trial run we for shows through there's learn the this market program certainly is recently pill to the is be PK took a for just reemphasize not of successful, we a room very There unmet population. very real I high only in annualized. if And multiple but -- a proceed form, mechanisms sales in in all that activation, this Certainly, the again, that We be diseases for compelling pursue, the note would rate we'll of $X.X want excess action. billion and in MDS, data significant

Operator

moment for one comes from And our question. next our And from next Goldman Sachs. question Richter Salveen

Unknown Analyst

[ Salveen. Lydia] This for is

How meaningfulness? of in be of expect could the any III Just data implement the you clinical medical this does meeting we could later year, additional that frame for data full another to thalassemia. on just a the Phase ENERGIZE-T expectations at that's terms insights data in terms coming or points

Sarah Gheuens

primary around our So yes, and in release, endpoints we -- the the we because transfusion-independent. the highlighted were secondary press one endpoint of --

highlight very, happy will with we're obviously, because that. again So very that we

well. also the we as [Indiscernible] secondary course, all we presenting met of mentioned, as As data safety well and will endpoints, and key be then,

a measure clinical ran be for the see pleased analysis. We're trial with intent in key which how line So it's going the have actually that presentation secondary results. first to we a a interval have done in and to treat and analysis the very endpoints time. reduction the the with may you we long we designed typical In prespecified very transfusion

with are, have very so trials now trial and generated. again, their data happy The X out. completed that, of the read And have results that course,

the forward are to to looking and development So and very move this. start forward to have engagement regulatory really we are review in eager

Operator

for And our question moment next comes our next J.P. one from question. And Morgan. Romero from Tessa

Tessa Romero

is many side see the On on could the should to standard thalassemia revenues the just NewBridge. of regulatory a the how then for in your the should priority here point gating follow-up the this is factors And we with thalassemia, we specifically or soonest arrangement question second at about case How here? for how think patients about have And submission region? U.S. think the we sNDA? Saudi review? And in start our the a base

Brian Goff

regulatory pathway? with to you Sarah, want start

Sarah Gheuens

Yes.

package their review eager with and assessment. FDA very we're for together submit the the and So to work

comment we're for So don't when those how, doing later. process really. on milestones are typical or a We

think we're to excited progress just I the very team making So and everything is are going. just pleased with get our

Brian Goff

Cecilia, Tsveta, new NewBridge GCC? And sorry then

Tsveta Milanova

NewBridge. approval region with we're it the to same The we'll signed and important submission given second GCC, NewBridge potential to is agreement GCC working thing steps we for of with commercialization next mentioned, take start super-excited the most comes When the U.S. about as timelines, for preparing submission with us after the having on and

We haven't in might we deepen the stage. thalassemia there is about market able more with region this specific be to timelines on with not It as are understanding. XX,XXX our provided -- the start in We that. information, but patients working provide to NewBridge

Brian Goff

And component clearly, That just very largest want make you to of the is Arabia in part is the that. And clear. one pathways Saudi access comment do on region?

Tsveta Milanova

Absolutely.

and tenders all health we there market. speed can. expecting see institutions, as as after depending are starts you numerous to we getting think to when formulary Submission these to private at in forward steps the NewBridge pricing pricing probably about markets, the patient best process way steps local The at about And working price kind we with So hospitals time, the quickly key look in is a think the over assessing of with and closer reimbursement. level actually to and agreement on the are the with to obtain submission where actually national ways of then GCC regulatory the reimbursement uptake. And the level. the European region, process the to market, the agreement to get access other and we navigate and to different it and that approval

Brian Goff

assertively that excited just the very working with had on Tsveta But again, is about #X thrilled hopefully And build the the we've U.S. in into commercial come and we're the our PKD back far, And diseases, thalassemia So moving opportunity. Tess, to priority prowess in well. our commercial as to, out have is momentum rare cell arrangement. we're building to team to beyond and sickle by

Operator

from from America. of Bank comes for next Alec question our Stranahan one And And next question. our moment

Alec Stranahan

couple us. a Just from

update reviewing? going anything on will year, of First, framing the this of the be one be complete the full will And kind of data we from efficacy that. into that sort should the the end just data by the the different then and in today incremental sNDA Any that to non-transfusion next what picture made secondaries? And Is follow-up expect of PK on or the submission sort the FDA the one different and that obviously, more in safety the read-throughs on to study, be study points year. of patient expanding setting and question data end just population? from primary and

Brian Goff

Thanks, Alec. Sure.

So we'll on package pediatrics. take could we'll our talk in about just data. first of one And comment maybe you And those Sarah, secondly, planned the sequence.

Sarah Gheuens

of we mentioned, have completed, as X locked So been now data. the the course, clinical randomized the program, and trials have have

put that So excited together. we're to very

very work are we standard and to the updates process that doing really regulators are -- that that We and forth. the back started. always be our type questions of normally eager timelines work for need and would standard doing do submissions follow with cuts and we to that get There's

with there motion. And endpoints, safety going trial, the the an both pleased just so So this ACTIVATE-KidsT we're generated that data clinical was very in I from -- to go the and through mean, to efficacy. we are regards in a

Brian Goff

Yes. link to it then read-through would be non-transfusion-dependent the or the And as patients? ACTIVATE-Kids called thoughts on

Sarah Gheuens

ACTIVATE-Kids. were similar It's So population blinded. patient non-regularly-transfused methodology a for applied again, that it's

of eager we applicable as next that response truly the is action package. point, we very We the highlights and very get then -- in well. pleased because to trial, kids So transfusion-free to are that the get are data ACTIVATE-KidsT we'll the Mitapivat mechanism of to

that, so And translates hoping the we're trial. now other also that into

Operator

am call remarks. Brian Goff, like of turn closing I no questions. I Agios, would for back now further And CEO over showing to to the

Brian Goff

Justin. Thanks,

and of much date, a thanks thinking it's milestones for a to to. maybe looking a in the very in our for best we're lot with to exciting This forward importantly, great a clearly possible Agios. the way everybody, little at still bit on so and been so breathless is very far come just continued participating that questions And year lot, more the today's delivery call. time And

strong conviction also transformative ultimately long-term create So and new we deliver patients have to to we're shareholders. therapies to for poised value significant that

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

Operator

you for This participating. concludes Thank call. today's conference

You may now disconnect.

Agios Pharmaceuticals (AGIO) 1 Aug 242024 Q2 Earnings call transcript