PRTA - Earnings call transcript Q4 2024 | Prothena Corporation plc

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Operator

Good day, ladies and gentlemen, and welcome to the Prothena Biosciences Fourth Quarter and Full Year 2024 Financial Results Conference Call. My name is Pam, and I will be your coordinator for today. [Operator Instructions] I would now like to turn the call over to Mark Johnson, Vice President at Prothena. Please proceed.

Mark Johnson

Thank you. welcome quarter call results and financial and business review and everyone, guidance. fourth progress, full-year afternoon, to to today's Prothena's XXXX Good XXXX financial available issued the also is a today today, release review filed to website we X-K the Please which prothena.com press attached is with on Form and our at earlier SEC.

commercial Financial Chief programs. Nguyen, the before we the website. On to section and our Presentations Investor our Relations financial clinical Officer, a Events closing today's provide wholly-owned our Brandon our on turning will and are Dr. program. Executive of of supplemental Officer Kinney, XXXX up And an XXXX and Officer, back for Smith, development we will which slides, opening will on our provide financial ongoing an our Tran using insights then addition, strategy. session. Chief Chief guidance Q&A provide and In are open it call Chad Strategy on Gene will call, remarks, remarks, point Swanson, available will Chief Officer, Officer, and discuss at overview Development update which for corporate our Chief results President including those Gene Operating Prothena's

differ and would uncertainties, we from other making presentation, those we factors forward-looking statements. you could Before will materially I like risks, any that forward-looking that begin, actual be to referred subject statements remind to during to results certain today's to that in cause are

well turn We that, our obligation most filings over statements. With a update I'd our today our associated Gene. release forward-looking call our with of forward-looking like statements, recent and as see risks SEC. press to disclaim to to uncertainties the For please the issued with any discussion the as

Gene G. Kinney

us you, Mark, all thank today. for joining and you Thank

Slide on X. begin Let's

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to Moving on X. Slide

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new a evaluating BMS the XXXX, region of treatment score clinical BMS-XXXXXX, a ongoing from partner the patients formerly PRXXXX, Rating ATTR XXXX and from expected forward ATTR-CM, at of complete on XXXX. BMS-XXXXXX anti-amyloid initiated ATTR-CM. The patients coramitug, best-in-class or the XX half PRXXXX, Scale the which tau. In TargetTau-X Novo, this cardiomyopathy XXXX II potential treatment patients. We included versus programs baseline XXXX. Also Clinical next potential Moving currently specifically X in license placebo important is of of global key the high in with closer antibody for a patient in that potential with Nordisk the in microtubule of of treatment amyloidosis a results progress agreement to for trial coramitug, and Novo Alzheimer's updates to disease future MTBR disease. Sum approximately XXXX, of primary or II trial. announce Phase with doses with complete expect look of significant of trial development early for half the Boxes $XX binding low and is antibody to months. Alzheimer's XX the made endpoint trial second Myers opted is The conducting in and our steps payment targets is potential a epitope Phase Squibb. change clinical Novo including moving with further first BMS The million. should We in is within in an to Dementia of to in Enrollment signal-detection into XXX Bristol trial we

the multiple I exciting discuss in doses expected and initiated to a trial Chad? has partners. to an the clinical I in of turn is to clinical As further The is now healthy strategic pharmacokinetics Prothena the programs evaluate will our part year complete first-in-human adults. single-ascending call for XXXX. This detail, Phase and in our trial agreement, safety, Prothena of tolerability, and immunogenicity, Chad. and enrolling wholly-owned To over

Chad Swanson

treatment anti-amyloid the Gene. of amyloidosis. like start discussion our I'd Thanks, with review to for AL my birtamimab,

clinical at trial. p-value AFFIRM-AL FDA. confirmatory are significance mortality time a conducted with the with completion our trial nearing all-cause X.X agreement of SPA a endpoint to Phase primary being than III success of and We the is is under less defined a or to The statistical equal with

discussing current AL care. of Let's the standard X amyloidosis. is the are of amyloidosis by and unmet disease of need with where biology the start hallmarks AL There

The chain light first is proteins. production of misfolded

lead and deposits is soluble address early insoluble the second of organ. Current which chain who light aggregates care mechanism specifically high seeks such to the third light The the the insoluble deposits insoluble chain of failure may both which not to directly clear standard in the at address consists amyloid risk amyloidosis plasma light vital misfolded for and in of its urgent medical toxic are can chains organ with of Birtamimab, formation need aggregates is unmet and do toxic of dysfunction organs mortality. Birtamimab patients early decrease designed damage, that the target therapies chains, misfolded mortality. toxic accumulation production deposits heart. cell-directed of cause soluble is AL to amyloid aggregates differentiated and soluble neutralize light anti-amyloid amyloid as and

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was across summary been placebo VITAL compared current combination and nominally profile risk and further of In meaningful X-minute reduction by with This at p-value In with favorable supported the AFFIRM-AL walk ongoing effects clinically p-value we shown the of early XX% with score. And standard-of-care Phase of versus ongoing resulting with clinical trial, impressive of survival observed Stage our than VITAL patients the results Let's AL the significance separation success equal our benefit who previous III statistical amyloidosis and were the SPA AFFIRM-AL defined in is FDA a our with the or curve trials. VITAL, less a current as measured the this review we SF-XX and agreement by has XX% a X.X nominal X on test where supported well-tolerated trial. shows Mayo trial, slide. The confirmatory standard-of-care. significant at birtamimab on approximately component X.XXX. as mortality function month at physical safety for categorized Kaplan-Meier IV a distance birtamimab to which multiple baseline in all-cause in

Moving mortality. significant that Slide Recently from on addresses ANDROMEDA data XX. demonstrates study the clears a amyloid presented therapy to for early a need and

standard-of-care and at and is to mortality. the of Recently, randomization XXXX after treatment standard-of-care For that often regimen comprised study, impact Phase and practice daratumumab separate was used This AL trial. This approximately on combination in do adapted the not December trials, shows a VCd VCd. dexamethasone, to suggests daratumumab to referred until early CyBorD of clearly bortezomib daratumumab include in as addition in an AFFIRM-AL amyloidosis the clinical or in slide, which the cyclophosphamide patients to VITAL ASH curves emerged have addition as that months. our VCd as from at presentation standard on has allowed does be and ANDROMEDA this survival AFFIRM-AL III alone of with XX comparing the the a not

time-to-event the observed specifically AFFIRM-AL. Please we directly early address Slide to mechanism birtamimab's However, pathology, results differentiated of looking mortality in disease to reduce and confirm with again, plus is trial to to in risk on a agreement p-value ongoing are as SPA statistical AFFIRM-AL prior of the and birtamimab. The standard-of-care the defined or our Based analysis X:X patients field, versus our physicians VITAL Phase as from potentially VITAL to our a where leading and engaged Phase the towards equal for FDA VITAL the less these turn birtamimab with well success FDA survival than us are for to III placebo III significance randomized X.X. results is in the we confirmation data data, external align statistical further confirm enabled on a extensive to standard-of-care. is and receive actively as in regulatory experts trial with success the XX. trial, of on at the a designed This path data

We expect in results second of to announce the quarter XXXX.

X:X ASCENT-X a target the which confirming turn optimal treatment. We is clinical slow objectives designed for now placebo people with trial with early with significant receive a similar consistent to engagement, PRXXXX disease. Each XX. are Alzheimer's anti-amyloid PRXXXX approved humanized delivered with was treatment of off potent and value our let's clinic, PRXXXX to Alzheimer's have in is are for people antibody affinity currently the and beta to a Slide therapies placebo-controlled for safety on or in subcutaneous patient that further portfolio, look to monoclonal with We represents the PRXXXX double-blind and living Now let's XX. high of forward PRXXXX potential all allowing IgGX review the once in highly evaluating randomized and rate believe home avidity, beta is disease. PRXXXX our of efficacy designed starting with trial multiple-dose specifically once-monthly in our X to for Alzheimer's a with burden monthly cohort months. The is less antibody anti-A binding two-fold. Slide the mind.

all initial First shown is PRXXXX with here. around early "A" a carriers the of tolerability, evaluate is are data disease. the This find in safety, represents approximately regimen APOEX include either the pharmacokinetics to or heterozygous to dose the patients trial. Starting our early the mid-year, PRXXXX optimal to participants results clinical will immunogenicity from registration-enabling pharmacodynamics Alzheimer's of the XXX non-carriers disease. Alzheimer's second and and for cohorts APOEX share And X characterize that with

all on extension and which APOE X Additional approximately at And XX and presentations study. upwards the longitudinal patients been participants dose any ASCENT-X have are some of open-label for who ongoing may of "B" and months carriers. data who treatment readouts cohorts from for Data homozygous following. from our XX include data the various enrolled XX levels

region Slide to the of A-beta key promote block N-terminus PRXXXX move MTBR our designed to the amyloid of and and epitopes XX cell-to-cell the within tau Let's to of program. pathogenic review tau. clearance PRXXXX transmission targets

of XXXX potential EXXXX of particular, Eisai's with antibody, tau tau, targeting activity. In some within treatments data also on signals tau-XXX, positive mid-region the New clinical showed number small p-tauXXX, was MTBR showed presented biomarkers, of which PET, efficacy. participants associated very a CTAD at including early in MTBR the which has which effects and in areas targets region, of been

trial into to their In These for known Squibb in our data tau year. I'll Brandon PRXXXX has advanced Phase robust addition PRXXXX, BMS providing turn us and in give program commercial Bristol formerly to its confidence anti-MTBR discuss a development confidence partner, wholly-owned XXXXXX, later our antibody look updates targets. further forward as II further path Meyers we an signaling the on potential program. now this our to it over the points

Brandon Smith

Thanks, Chad.

focused first capabilities Slide potential support XX, commercial product. building to commercial our we our to out Moving are birtamimab as on

from birtamimab, results launch on to promise positive has half significant U.S. second outcomes we opportunity. are attractive and at in heart vital to organs, the While to be Providers are patients Phase AL the a very leading amyloid anti-amyloid has and there impact the treatment mortality. the by standard This for care continues other survival clears that enthusiasm. need be our expect of of III of in will unmet for waiting significant the patient amyloidosis the patients evolved, support commercial XXXX. early make directly of treatment which an to early factors for several There risk benefit. With a and

an First, this established is market.

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for IV targeted population profile very observed patient this Second, primary unmet we point treatment early and to date, high is that penetration address Mayo significant a if approved. patients rare hematologists expect with call the the safety Stage for third, with And favorable from options mortality peak the specialists. treating need support disease cardiologists, with a specialized

our prescriber U.S. in Europe consolidated are We most and excellence, And patients prepared to the centers out force specialty academic relatively on call this to and medical treated are of amyloidosis at centers amyloidosis centers. build sales base.

as able high-prescribing will as to centers hospitals. is these and as cover We our force sales well practices well community ensure

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Our birtamimab to continue launch timing in and plan markets. European commercialize is the in independently U.S. the to evaluate

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existing Our extensive we've field and for team clinical experts birtamimab. through programs to with relationships our in build the KOLs the established continues upon

AL amyloidosis to anti-amyloid informed the present efforts in at new appropriately a and solidify peer-reviewed further birtamimab of and as care are data journals. birtamimab's educational treatment congresses role standard and incorporate We with will community on to potential publications they guidelines. continuing are ongoing These position top to KOLs aware medical the into these important our continue birtamimab. This of to unique collaborate the to in like broader fully of includes ensure

reiterating up a birtamimab few by wrap key points. on Let's

therapy amyloidosis results, to risk and III as standard will be for anti-amyloid of Phase patients positioned first-line AL at the therapy, birtamimab-based confirmatory new the care be birtamimab has positive becoming the regimen with first of for potential First, mortality.

only birtamimab benefit dollar survival early at peak. opportunity market therapy be AL expect in demonstrate birtamimab global be to double-blind to Second, third, we placebo-controlled multi-billion the amyloidosis a would trials. And clinical

XX. Slide to Moving

to for with the As we've of discussed, on continuing biotechnology capability rest opportunity to birtamimab to forward Prothena transition this to build our look fully-integrated future portfolio. is perfect commercial in the a We commercial the company.

early underserved some the disease to future PRXXXX X are not is and their PRXXXX options. PRXXXX the well the portions AD for as or These enable patients of to with financial met opportunity early XXXX disease large and some very presymptomatic Comprised PRXXXX vaccine prevention our call fully and option needs presymptomatic AD a symptomatic guidance. ahead, full families some potential of well-positioned of market and programs, approved. financial approach. discussion market early a presymptomatic the may millions symptomatic treatment beta and address market to wholly-owned a for with subcutaneous very if treatment compelling. opens Tran be now, performance market Alzheimer's whose Looking as and XXXX programs as and the over treatment our Alzheimer's segments. And like potential as us the turn a designed population option I'd both of is would our Alzheimer's up for the addressed This markets. today's anti-A once-monthly on

Tran Nguyen

XX. detailed turn financial breakdown Please for a Today, XXXX in-line Brandon. that financial were results Slide results. guidance. financial of Please our we press refer our release our to to with reported Thanks,

for exclusive $XX received In license an global from obtained they in PRXXXX. we addition, where million the payment XXXX, BMS

end million. which the end terms of $XXX.X our of guidance our of of to range $XXX $XXX had performance million, investing operating the at in activities of low of $XXX financial loss used is XXXX net million, our Net relative we $XXX.X range million low and to cash $XXX was at to guidance million is million. In which guidance,

XX, As is cash, which of in and of our $XXX.X in-line XXXX, Prothena with cash million guidance million. December equivalents, had cash, restricted $XXX

Prothena shares had As million ordinary XX, of XXXX, February outstanding. XX.X approximately

with have X debt. we Additionally, capital to simple structure continue a

on expect and and we million. operating used to be $XXX XX, net XXXX activities XXXX in financial $XXX between guidance Slide to our our Turning investing cash full-year million

approximately $XXX I'll cash the estimated our of driven and an year range. The includes non-cash milestones. cash upcoming an to to the which cash, expect over estimated million represents that, share-based with in back which expense. With midpoint activities million used $XX and net restricted to turn end the primarily $XXX net of by the of cash million million call $XXX operating is compensation XXXX full-year We in equivalents, to discuss loss estimated investing Gene

Gene G. Kinney

Tran. Thanks,

physicians, Moving XX. who to a medicines science trials. thank acknowledge staff to and we participate elucidate and could all also developing. I'd to like new the clinical we're families, for serve. Prothenians patients Without in I'd to advancing Slide impact and the their talented not like the we study-side to dysregulation of their support, our the their thank potential of impact real for ongoing make protein our commitment families patients,

XXXX our As potential with as readouts well has year a strategically partnered from significant transformational today, development and we've to programs programs. wholly-owned our Prothena from be discussed clinical clinical the for readouts continued as

in for detection excited AFFIRM-AL II the Clinical are results from our results disease. evaluating we signal amyloidosis. ATTR-CM. trial Novo's Stage coramitug evaluating with from patients evaluating as a Phase early in announce ahead, trial confirmatory PRXXXX Alzheimer's ASCENT of Phase our I best-in-class birtamimab Completion results and AL Looking treatment trials treatment to potential IV Phase top-line from Mayo III

look we our Prothena's robust as proud addition, fully we programs clinical cash in biotechnology execution become PRXXXX. remain an to we'll BMS-XXXXXX, for advancing Q&A. open and commercial XXXX, us call updates with forward well-capitalized ahead, Operator. to PRXXXX, clinical for In that, up prasinezumab, and position the I'm sharing setting year strive company. With focused a exciting further for on we're of now a development integrated

Operator

Piper Sandler. with your comes Yasmeen And question Instructions] from [Operator first Rahimi

Emma Seymour

on us birtamimab. Emma from have This for on I X is Yas.

any prescriber on are well about? is in the primary with you've the case base therapeutic detail scenario AFFIRM-AL, how color base, those as as more helpful? and landscape, the into super things on like secondary? And endpoint -- the both Phase in within opportunity key AL given III the best commercialization additional what concentrated would best the that, considered thinking Firstly, you the and So be amyloidosis, then talked market especially the into that about all digging about

Gene G. Kinney

Emma, the thanks questions. for

terms around So unmet of endpoint, by the reiterating here. the me expectations start need first, in primary let again just significant

amyloidosis fundamentally regard all different current targets used that in So that directly. And as know, the Obviously, today of plasma I'm in in target treatments sure cells. the it is you birtamimab AL amyloid of at his course, early the mortality. lacking Chad what with on enrollment is daratumumab risk in study, patients and this exemplified an current discussion of around survival in treatments, in is the impact

So, that in obviously, was early that's mechanism a the opportunity in had and represents the as of trial that show significant our impact product. We unmet VITAL really risk treatments birtamimab current particular, particularly a a available on medical our Stage class need study for patients mortality. with prior of as the and these patients to differentiated did in survival, meaningful in IV amongst Mayo a that, there differentiated those

being a FDA that at think demonstrate the X.XX, to of us. with have that ascribes represent the of for benefit, the And really we agreement that survival think So that would a best case with SPA success opportunity. we that really able p-value we is kind

said, to can the potential bit market that unmet Brandon? opportunity. opportunity, then excitement Brandon, a around ask I address that that have about medical we that translates to so and more market into the maybe little the how And need talk

Brandon Smith

the but opportunity, points our in reiterate. something few to you to key laid a some you orient to dimensionalize XX, just ways on Slide presentation out of I we think help earnings to the Maybe

rare needed order from patients early treated established. that are is is in established survival in upon point. And strong directly immediately This And These with profile an of we've market other safety that vital to are a First favorable risk that with OLs demonstrates those conversations it's and with is a mortality. market. our disease patients heart clear an who our uptake, in off, high at learned amyloid research benefit diagnosis. consolidated a call what a importantly, therapy early designed, the anti-amyloid and organs, especially it's very

in blockbuster second and for well in dollar I said launch is over the opportunity half multi-billion executing I as the on peak, XXXX. to and birtamimab forward at a opportunity really planning alone, the the of the honestly in U.S. So opportunity presentation, looking a globally.

Operator

Your from line Jay Olson Oppenheimer. next the of comes question from

Jay Olson

all enrolled also with the utilization the patients of meaningful on you be in especially AFFIRM-AL? regards daratumumab? AFFIRM-AL, to about birtamimab, what demonstrate Congrats to a the in would OS baseline progress. the of And benefit talk characteristics for Can clinically then

Gene G. Kinney

to Thanks for Chad the speak I'll Jay. of baseline some characteristics. let the

randomization of that which space. control just mention a of his can And not was surprising become that the reminder thing that with plasma remarks the what as much more widely brings things along all point in with is obviously that that that's hematologic is to about used agents and the VITAL now I it, probably highest think of that deck, the in are targeting of cell finding that shown is the this the we've what And in daratumumab terms the we're really obviously on where the the that as in trial use about to in One one we of mortality. which the Obviously, risk the using the well. I'll to unmet trial chosen AFFIRM-AL we're dara, need as just Stage in focus population, patient X trial the those addition to spoke AFFIRM-AL about X that early obvious patients at patients former that IV. and in Chad fact, and targeting trial. And to in at the course, a highest-risk that relative impact of as those agent, would of patients survival plasma in AFFIRM-AL live. has trial, I Mayo that think focus and given XX be where ANDROMEDA significant medical that impact months on lack still mortality, first need of of continues -- unmet there's no a across cell medical even

baseline talk bit about space. excited Chad here, little it a over birtamimab But me more opportunity characteristics. we're the to the in let maybe can and about So he pass for that

Chad Swanson

Thanks Gene.

or Stage baseline as turns or to who daratumumab participating to the we - so AFFIRM-AL means to in per on the that population with able IV to study I it be actually are those in obviously this by the in IV again. designing daratumumab, We that may FDA know, randomization. p-value Stage recapitulate study of success are saw the end To success us in file. similar And at a by subjects the this characteristics saw a in were right? quite agreement used I'll of results echoing, maybe So respect about we daratumumab. to AFFIRM in VITAL. With think respect you design, is just really that the fact, out X.X XX% And subjects, in VITAL study with in the are in less

Operator

comes question Umer Raffat from next with Evercore. Your

Umer Raffat

A if couple may. I

trends First, have could visibility PRONTO both curious understand the to treated your long-term arm there's on previously active whether VITAL? going about mortality randomized the I'm the as Phase for Phase trials, but I II on, III mortality speak no phase of for well beyond as you any And trends. you in

emergent VITAL, at elaborate finally, analysis Stage your Second, what dFLC And a standard-of-care just deaths baseline? IV the for of in lot Phase that? expectation were there what's the TEAE could treatment on III deaths you -- drove treatment medium -- on from emergent on arm,

Gene G. Kinney

the you for questions. Thank

on I question, Let me just can start with and comment the of to PRONTO and other parts ask your the question. then Chad VITAL

long-term on visibility of terms in mortality. first, So

that are with active longer ongoing. of Of those group, there -- placebo do treatment total X course, X no we those from What is trial the and birtamimab. X from the know PRONTO trials were on have were deaths,

the that months shy mortality course, of longer had plasma-directed or no the of you so had Stage still into patient patients no see we terms but to received in that was if study, just because trial IV considered Of risk-benefit. said, ongoing, coming of you first to. That XX% a in across as ratio are of of X.XXX in results chemotherapy particular, hazard question VITAL, organ therapy, of a hematologically Again, something the can we've has -- just Mayo other have is past, dysfunction. In relative comment and point them the me course, there's the described parts follow-up Chad some those PRONTO ongoing let on the patients were long-term X representing stable that previously the population that asked.

Chad Swanson

Gene. Thanks

so we question, at essentially, were deaths respect all-cause with in looking So the mortality to that treatment-emergent study.

all deaths They all analysis. the are in essentially So matter counted.

Operator

line Cantor next Your C. of Duncan Charles comes the with from Fitzgerald. question

Charles Duncan

Gene and good on progress. year of congrats a team,

a versus for news statistical significance Looking of that wondering of to the difference declaring a forward it, demand meaningfulness. larger. with if flow size any difference assume using that there time to for going work difference KOLs But minimum any I and Brandon terms that interest to the been Has gone any the a drug? if had question goes here. trial but there kind is clinical of in lot up I'm statistically and I kind the to that And of has has was, thoughts success much that in forward death, back to to in there be regard significant patients. between coming important call effect statistic drug a might suggest

Gene G. Kinney

on of comment this, Thank little therapies. some you, Charles plasma-directed nothing on for -- that can to early this terms the -- which mortality addresses question. reiterate nothing view But in Brandon the research available of let our maybe. real currently I'll kind that there's bit the is just a of market

significant need that is the so with for of really opportunity current which patients. these for for have we is medical a there approaches -- this any And benefit meaningful no are

bit me agreement with be to that less very our we little that SPA we in translates for would to do talk hand meets would believe so, that meaningful authority statistical a meaningful than armamentarium. And be p-value the about patient agreement population, Brandon very to regulatory where the And X.XX to it the anything this that treatment how the under have let you commercial.

Brandon Smith

there is you thank driving came even just strong conversations fact unmet VITAL very in realize And on question. market honestly, with therapy. all-cause course, very as in we're lot nothing early will physicians, need widely that and shows that need fully data early patients, the standard is research because as to confirm meaningful our the very well-known mortality really And known. KOLs this separation to they a and interest our because disease is do impacts against their the all-cause be that specifically ANDROMEDA mortality, points of out the December there helps and the Yes, mortality. is with really trying for payers endpoint is standard-of-care, to so that nothing that that unmet that their AFFIRM, that confirms patients. And impacts these nothing this to did anything what's that gold and payers, in Yes, a to

Operator

question next Your comes from with Jason Citizens JMP. Butler

Jason Butler

the Congrats in the you're and I by design end ask a have maybe hand I for the information what of about you'll guess, Can development on you to the for that progress. looking one talk program, on program? want gears to switch year just data and late-stage PRXXXX.

Gene G. Kinney

can question. about this Mark begin ask at I the publicly. looking little a start the that talk about we thanks bit expect to as talking it to and Jason, we and have data for maybe Maybe

just of this and around instance, is the reduction how X very anti-beta amyloid we one thinking particularly relationship a as how ultimately amyloid mean a about as maximum I space, this I perspective, hand, perspective, how class, field ARIA kind in as broad talk evolving. On think a reminder, benefit. categories about benefits, So the risk clinical there's to much temporal in first to reduction, from through the that's well translating both of

think bit in here It's part important well a something very about as other remarks. talked burden. treatment that little we the I is the

designed disease, term, bit. devastating of diagnosed that why idea commercial pretty significantly the not burden I the think think was is PRXXXX amenable And treatment devastating profile is a a very this to in is thinking Chad the subcutaneous patients about once-monthly near a then these respect important are a a with as fair of treatment with to with adding we're treatment approaches. Obviously, that's the course. talking important We and at-home long-term diagnosis, burden uptake disease really to throughout administration. about

maybe, talk and So a we to we think to going about, what we if of Mark, incredibly out. expect, how that's want roll you that's think important. But little just kind bit data

Mark Johnson

patients as starting going to heterozygous earlier, in going APOEX on the what be This to shared, Alzheimer's. about data either are And "A" cohorts non-carriers kind APOEX that's ASCENT-X Chad participants XXX trial. with is from represents the so those that data or all building early are as be share Slide X presented far so really just XX looking at of first around multiple-dose mid-year, initial our

And the carriers "B" could to we early Alzheimer's. And readouts and APOEX second, some really to treatment regimen a homozygous what as looking for tolerability, year cohorts or XX longitudinal believe throughout objectives for that think really various for evaluating immunogenicity treatment months looking registration-enabling dose overall, the well have data pharmacokinetics from data in find the really in with we're patients as currently of data trial. therapies, patients evaluate for just and is home in Alzheimer's let Additional here safety, PRXXXX but as the to long a XX pharmacodynamics, the X include I what efficacy ongoing with And similar that optimal who burden levels with significant we're need from unmet the building for that for delivered dose anti-beta living on the people safety as on have ASCENT-X. addresses family. the represents approved their with been at and

Operator

Yee Michael with from Jefferies. comes question next Your

Michael Yee

not is amyloidosis from arm does So your saying it like study. is or your as that are it dara around the Phase study, impacting looking to the When arm view control and powered so implying question on dara has and arm period understand the that the curves expectations sort an explanation been there? I control your study more why look that's show ANDROMEDA, given study over the that this study the the wanted a you and been long. of comment AL relates on you you for to like arm going separate are now for assume fact on few the you III years do time, for going that that did, of control what maybe how has you the just

The just may should ARIA what that amyloid out has in would data comes And I do read second time result if is reduction? for of on When about successful sneak we your out? terms what expect XXX. in in you expectations that think the is good one on question, over you and how changed what

Gene G. Kinney

there. questions Thanks Mike. Good

as and about your arm. expected Let and with I this me around think events about timing ANDROMEDA we're the question accruing to speak dara And start study because I'll a fits and from Chad it our perspective. well, timing ask that's enrolling just and how into the how also control

X X.XXX world's look having than at which would which see across effect contrast X.X was this arm observed expected, been X behave And That's of ratio So it's literature The to do is trial, survival in what that somewhat that arm in mortality. months we hazard clear, expect the months months. expert we saw, space, about the obviously, access very there, X months. the monthly a on control to size all-cause in arm, at around we similar longer have VITAL control the a a course, then the of the you to control just be that time. But to in think medium have bit that little reasonable VITAL to against first trial. treaters the manner survival the you approximately median would can we if

We've find observed we that, what with mortality example, time to in for similar kind we done external some to we've a look of a done, studies patient-matched benchmarking. if very and event So rate VITAL. form external in databases

that's So we think do that well-founded.

not with months, point that's ASH occurring ANDROMEDA is the impact presented first study, daratumumab have ANDROMEDA think evidenced last and by the data those inclusion I the early the particularly of XX that mortality an to seem across does that certainly on the daratumumab year. was and events, at

the about obviously, both this, respect bit also true timing about with expectations. timing the perspective from at me occurring, And we're see let well-aligned or overall VITAL. seen maybe from a events that and the previously how with speak bit a So little with just we that we've a and our and within hand it that's that's over whether look are and we quality be perspective. But can to what what feel more we it like Chad little he of to maybe study, can

Chad Swanson

Thank you, Gene.

And VITAL. here So in and is results I -- VITAL based -- we VITAL add observed the way, the actually as nature I blinded so a quite to so obviously that right? we they know in designed upon patients Stage is think Gene IV summarized timing you what I well, look actually So that. When are that can that of saw much, events in AFFIRM events we maybe quite what that and and Gene those think not AFFIRM the study. at to in timing the alluded actually events, far, the in similar we've

shows that the in suggesting timing study clearly Gene sort the So par. Again that piece Slide is be dara with XX again, aligned any our the early events. months. to VITAL standard-of-care does that our survival these the on of medium had have seem dara of desire of with on in study looks as replicate to was Gene to X.X And mentioned of impact introduction things, that not mentioned,

it behaving first so XX certainly of should feel impact we And so in combined in be things have be months over -- the did VITAL. that on arm similarly AFFIRM so or those it sort daratumumab as should our not survival, the control again suggest an that was

Operator

Li from Your next question comes from Chardan. Rudy

Rudy Li

roughly the dara you for approved J&Js XXXX? And with secondly, can So was did dynamics standard-of-care, for color treated How be since any quick landscape. current and changes ASCENT-X, actively many market XXX cohort patients you in XXX maybe follow-up about the patients? birtamimab, in helpful. would milligram talk just for Any are in rationale PRXXXX with notice on expansion a treatment the

Mark Johnson

Okay. Great.

-- talking talk talked treated it's let's because some actually and today. of as about we numbers, are are actively these question, a about yes, So that we great patients being

you to talk bit do can Brandon, about you the XXXX maybe, question? So and address little want Chad, a numbers the

Brandon Smith

to. happy Sure,

our in treated rate. diagnosed So figures quote what we a is always and

presentation described The Dara our XXXX? or that question the in since is on not increased the we On U.S. in your patients back answer that in was patients is XX,XXX So whether yes. approved was

we then the was number I used think XX,XXX.

they -- response, sure make to on trying it Chad not So them they described impact move once is can this treat rapidly and mortality. disease affect into as an whatever having to increased. But they hematologic has which that a and the is rapidly early Gene identified,

number. and figures the an that from we we're prevalence treated that figures can extrapolated So identify our diagnosed, are quoting not

Chad Swanson

Right.

the of address I'll expansion the part your second So question, cohort. was about which

expansion that study. were quite here for able demand we frankly, to given be a the in opportunistic cohort So, bit patients' using

by enroll what order So, in cohorts. I mean for "B" our to is, us that

that are those for were carriers in of overall order APOE really carriers. It recall a population. to are make that's up "B" homozygous looking in cohorts number large quite screen AD because to patients takes we numbers cohorts. homozygous "B" these the that our early the obtain Now XX% those that APOE And about of

subjects not in to go otherwise the for X ways, homozygous have gives a that cohort, But allows able screening they're to are we're because right? So not also "B" be the So, to into us carriers. then PRXXXX. characterize actually first larger that; take us an cohort it who help expansion to dataset result to that use those ability able the is One, to would better

is of kind randomizing due it approach cohorts. opportunistic "B" those So to an

Operator

Ahmad Tazeen comes with from Bank of Your America. question next

Tazeen Ahmad

uptake. I wanted a to think commercial to get about how about sense

patients? approved the in Stage I expand patients design, launch or early wanted time also severe you to the on it label the from based be phase? And really and onboard study IV patients could IV to get then it of to you Stage about them tomorrow, how Like they them it the about think talked long to would you And these find X,XXX you U.S., do to easy patients how So you? AL are if find? to limited ask, would the could less in take Mayo had

Gene G. Kinney

Thanks Tazeen for questions. the

comment just right Stage on those I'm Mayo the IV we're But focused patients. to identification maybe you that. on bit the comment patients. So a of Brandon ask can on now, little yes, Obviously, going to

Brandon Smith

Yes.

my patients I and think diagnosed I we're quoting previous comments, mentioned treated. are these So the that are in

aren't diagnosed time treated. come disease. patients hard Over the that is a patients rare So and It find they're these to in. do because

move that treated. So into and get patients we diagnosed as expect then that we of newly fully those would diagnosed bucket that

commercialize, and into take we're marketplace, this this be Prothena's -- first option. will In will what first the get does launch contracting, physicians make time the through terms treatment and to new in to get launch, the and our be it it of takes entry initially again, aware of

time opportunity. get dollar order certainly, long-term to to revision that But multi-billion term, to our near will take it So is a the get in update. in

Gene G. Kinney

one I Brandon are one has think mentioned as the think. But I they're think I about the patients -- that And of is of treated. things these diagnosed this, disease this key

and a is have I disease a these that's awareness definitive and in physicians that treated an diagnosis. this patients So level as obviously, today, think getting are coming

Operator

RBC We only that have and from time Abrahams more Capital for of Brian is question, one Markets.

Brian Abrahams

readout. And curious European you the whether ahead of progress. and after if similar be regulators I'm cadence both pipeline I'm would what about could European you're plan, continued put that talk to for infrastructure the build the on curious of and has thinking AFFIRM-AL then also the you congrats expect, the path how bar commercial the U.S. forth? And would about

Gene G. Kinney

questions. Great Thanks Brian.

the of to your out. with I'll talk ask latter part start and little a bit about build then me question Let the Brandon

think the So in any obviously, terms of I medical is other that Europe the matter, geography need for same.

So perspective, I similar. from I think conversation say what that I can is risk-benefit think is very a that

the in on an and As plasma an treatments, impact geographies, in is current true Europe other do it event. the the which not mortality patients these solely early which focused have also U.S., is on treatment, in targeting cell are

of we globe there And the would dialogue profile of with the molecule a for need authorities, high regulatory is unmet mortality, significant across medical palpable that So program. expect are risk-benefit of across risk patients is that obviously, the early geographies. and that those productive around at all

to maybe So how can that. talk of talk he a maybe -- said, we're can that over buildup, I about Brandon, thinking commercial I bit hand and can little or it

Brandon Smith

patient question. ] about today, is goal positive We [ the provide goal is we patients. But very experience into details access that it so need. birtamimab what is first services, build-out. first the so the mechanism available treatment be probably to action to the the to of commercially as for name institution out that won't they right our go the I the can is get for said, launch as goals. Right, there patient, our a the Thank is to from Prothena the excited our commercial very get are payer the ensure but describe opportunity And many physician, for a AL the amyloidosis can of seamless I and talk you and to too for that

mechanism be focus ensuring our for education, means a is preparing that a what that we'll into really marketplace. identity space. approved, lot that's we get label So top-line data of is providing move this known get actually and as we'll then market launch, And in we the opportunity the our as from in brand as the our lot of move to is. What on for we

more I'm So about really all really as about that giving updates and past that top-line excited get on data. building excited we of

Operator

now for over closing Kinney call Gene turn I to remarks. the

Gene G. Kinney

our I to further the Pam. joining you, We and thank your on us interest today. Thank want on look we all to for call you programs. appreciate just Prothena, forward sharing in updates

Operator

gentlemen, and today's call. Ladies concludes you all for joining. that Thank

now may disconnect. You

Prothena (PRTA) 20 Feb 252024 Q4 Earnings call transcript