ALNY - Earnings call transcript Q3 2024 | Alnylam Pharmaceuticals, Inc.

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Operator

Good day, everyone, and welcome to today's Alnylam Pharmaceuticals' Q3 2024 Earnings Conference Call. [Operator Instructions] Please note this call is being recorded. [Operator Instructions] It is now my pleasure to turn the conference over to the company.

Christine Lindenboom

Good morning. I'm Christine Lindenboom, Chief Corporate Communications Officer at Alnylam. Yvonne Pushkal Executive and Garg, Chief Officer. are Chief Tolga Jeff today Financial Officer; Commercial Chief me Greenstreet, Officer; Chief Tanguler, Poulton, With Medical Officer;

slides participating Events the of the our call, For going of section via page investors.alnylam.com/events. of accessed by you the those accompanying website, can Investors be to conference

progress. expectations, progress. we clinical pipeline During review questions. I and today's call and of concerning the and Actual statements result safe and remarks you Jeff constitute Slide forward-looking update And financials statements from some of context. materially future general recent Act on of our the your provide under those differ harbor provision a contain provide on the Reform remind guidance will which our XXXX. Alnylam's for an Litigation review Yvonne call, X, on prospects, in as those periodic Pushkal purposes may the will global results important SEC. summary commercial Tolga a that upcoming these Securities report followed most this plans would Private file for indicated updates open by forward-looking factors, discussed with outlined remarks like to milestones, including as offer will of call will by various before introductory will the

date I'd call representing of Yvonne? such disclaim In and be specifically our as to to turn addition, like update represent any date. upon of to the as views our statements. With this recording the of statements obligation only as Yvonne. views not relied any any We that, over forward-looking should subsequent

Yvonne Greenstreet

joining Thanks, the Christine, for you, and call today. thank everyone,

across and regulatory Commercially, TTR results in continued period XXXX the of of generating made vutrisiran our all HELIOS-B Alnylam U.S. a on and $XXX our product submissions revenue, year-over-year global franchise, filing growth X The marketed million sharing We XX% with the great on achieved products. study third of progress progress we for in EU. net quarter marked fronts. additional

in has TTR anticipation cardiomyopathy We as portfolio also Investor We potential for been broader franchise new portfolio product delivering companies a in potential the durable to ATTR TTR what believe what R&D, similar hosted a year. and and and cystic long of highlight our franchise, companies in that our impact EYLEA fibrosis AMVUTTRA Regeneron, will for has for to or serve diversification launch preparations flagship our in term. Day to Vertex, to growth, next the enabling those been invest development, those

momentum study track on performance. of for diseases, we're in while a in well, the call by of commercial from Lastly, CNS organic they a today steadfast turn for mivelsiran developing CNS. are in our driven medicines Phase early to from now to doses product efforts announce commercializing continuing of become well portion disease. of results that as to tolerated. We're and pipeline progress Huntington's that, top-tier and the initiated review patients and/or Phase our multiple product PXxXX the we're pipeline an of as our to transformative financial XXXX are Tolga? our all around particularly year-end high-yielding first early These continued pleased and evidence best-in-class with belief results. With Alnylam a our with goals, our in by me for candidates engine, initial saw strong prevalent let over and delivering world Tolga platform of achievement study results multi-dose We a I rare that biotech, patients onset I early of the ALN-HTTXX our administered encouraging Alzheimer's intrathecally provide are towards siRNA disease,

Tolga Tanguler

with TTR the delivering both therapy number rare Yvonne, our quarter of commercial our continued combined portfolio with of XX% portfolio, on the as quarter for year-over-year commercial strong was another in third our compared XXXX, patients and and increase morning, good Thanks, to franchises. growth everyone. we QX consistently of

Let turn TTR me now to a performance. our third of summary quarter

Our compared XXXX product as representing net competitive hATTR with noteworthy in a revenues, quarter TTR given to number XX% U.S. of polyneuropathy increase on third franchise our global the we the the million increase patients in environment. therapies, achieved particularly $XXX of continued the

globally, across suffer that with We this ONPATTRO, patients are our devastating from opportunity to significant disease. given belief X,XXX there patients is approaching that on and growth the treatment globe or a still XX,XXX AMVUTTRA XX,XXX

year-over-year U.S. XX% the strong in XX% patient drive ONPATTRO patients growth. The by Now with following: increased was of growth by rest XX% ongoing sales than primarily AMVUTTRA patient adds of of strength let second world momentum driven year-over-year combined X% increase switched to In continues me AMVUTTRA and more U.S., U.S. the robust our driven and in AMVUTTRA. highlights quarter as have in the compared the by provide and ONPATTRO to of on demand, TTR offsetting decrease uptake, a performance. the new who a

the gross We growth are in the the XXXX. has adjustment. competition to consistent year which net market pleased been of entering Favorability throughout end at new also is demand despite the with driven by

by orders XXXX, me booked international in X% compared demand remained market, grew gross net plus our by we with yet including where On approval. QX, ONPATTRO third Australia X% by we our X% shared quarter-on-quarter to the promising the decreased where our potential TTR Portugal Investor addressing business recent The adjustment turn in TTR plans minus launched of TTR regulatory driven growth cardiomyopathy year-over-year markets ATTR large in was compared a in to in partner as to we of let vutrisiran Day, that second a ONPATTRO quarter XX% in strong quarter-over-quarter decrease QX of our international and basis, the the the market. Now franchise growth. of patient for this XXXX. At pending inventory with built was our quarter in markets

there by need unmet field points number results and optimally reflective key not several we've HELIOS-B, patients. in or across key progressive today's rare factors. are a cardiomyopathy journey of this disease in compelling significant driven toward substantial our remain ATTR a end undiagnosed of shown is population We With confident very is where patients in treated. leadership and

We experienced have operating of ATTR will environment. our instrumental we in competitive for on a on And and track be of a and franchise a launch, scale phase in record new growth. as Alnylam's a focus polyneuropathy flagship dedicated success successful building ushering

combined XXXX. quarter the increase and the with compared of Now GIVLAARI XX% moving an sales rare delivered compared XXXX and OXLUMO, second and during of our franchise in third product quarter million quarter, $XXX growth X% to representing the which with net of the third performance

the increased the QX, robust due markets, increase primarily timing XX% our X% the versus year-over-year the increase of market by U.S. by highlights. in demand patients A product quarter in QX the of markets, primarily across of primarily European driven orders in increased our growth driven sales by markets. with to in growth of rest GIVLAARI, a in partner and primarily regional partner therapy; with world following orders compared XX% new third XX% XXXX, timing For and a on Brazil;

R&D results in OXLUMO, to year-over-year following delivering by product dynamics. our guidance, year our partner XXXX, impressive world XX% pipeline a the for next midpoint third preparing strong to revenue meet the rest launch to from on recent reflect growth Pushkal? TTR while year. And will versus of focused and EU achieving demand; XX% it now potential increase our positions primarily our conclusion, U.S. full our earnings XXXX driven a the compared intensely This franchises growth; QX by QX markets. In to remain our Pushkal with turn ATTR demand rare that, primarily performance market, We our For and relatively consistent quarter flat regional and driven progress. revenue I with during both of patient updated call. strong growth. sales which and with growth robust growth cardiomyopathy patient patient raised over strength us quarter A well XX% QX second were review we and both across

Pushkal Garg

good Tolga, Thanks, everyone. and morning,

across started review know, TTR, study HFSA of a additional of full number disease. important of from We QX favorable outcomes progression. at assessments. This reflecting highlighted as August a was profound we in of exploratory exciting And characteristic September. impact for patients shared priority function echocardiographic in ESC pivotal impact follow regulatory greater completed these with vutrisiran's a was status in to United indicating underlying than and profile franchise. used As TTR data, saw the clinical data very XXXX. in today's presentations then additional these transformational you benefits in as pathophysiology on by HELIOS-B cardiac on series assessments, XX% impact a We findings population on we've EU, at delays now of potentially our with comprehensive measures regulatory benefits on the and the Together, and all-cause results on followed the of mechanism health action. with then saw rapid reduction of of knockdown global disease compelling a with States, RNA well in the this with late mortality. Based cardiomyopathy, ATTR in in the biomarkers ultimately, all where This submissions we've voucher, submissions

results Alnylam. now multiple of today disease. for with onset in mivelsiran dose from Turning the I Alzheimer's to portion to another the Phase patients share of program study early We're new important excited

reductions substrate CXX for address angiopathy, Alzheimer's organ program protein, the diseases. Mivelsiran enable that amyloid the of is APP our recall, these these early and from by study deposition, in XX patients. In to We a line, unmet amyloid our a target or all from of X here, As data for improve days the diseases. Today robust of by multiple biomarker, data on XX achieved approximately onset precursor to months leading measured patients clinical we the Alzheimer's is dose day is month therapeutic even rapid, see that at slow amyloid X, target of liver the seen devastating the target can the treatment interim dose exists mivelsiran apparent as soluble soluble APP conjugated characteristic and you engagement we study, initial RNAi during in are precursor dotted upstream cerebral and or X, disease high reduce beta sharing or with chart believe mean dose thereafter. In slow, baseline effort of the the neurologic the CAA. APP, single disease single can and dose of are initial a with deposits part the XX% halt of where in potentially approximately and After reduction development X beta the expand administered is can dose Phase X, of that the targeting XX-milligram through for production, in or a APP XX% the need beta. single you new the you at after the manifestations I sustained and in at APP and study milligrams cohort form platform the natural sustained dose. month mivelsiran month the RNAi followed first of pink, from see interim beyond were and the we towards X, lowering diseases. amyloid systems multiple clearance, still that were portion then return that engagement beta amyloid X peptides second

trajectory a then, month You beta patients second XX the dose X, X of reductions similar at month further can very see X, But APP first dose above beta. of after see achieving months. XX% APP of single after through at lowering that patients milligrams with the APP we beta the follows the XX-milligram to

we very can lowering are indicate sustained encouraging they APP these of low data So doses. achieve relatively as with robust

has in CSF have identified both the or abnormalities in CSF Phase the chain. been safety tolerated exploratory neurofilament the been light total single new and white no generally count, I on cell protein of safety on have signals dose multiple and significant well biomarker No labs, as such parts been seen mivelsiran Importantly, study. and

this we development. advance results, through program clinical continue these encouraging Given to aggressively

cohort initiated Specifically, escalation safety continued multiple ascending mivelsiran. We've explore explore dose dose study portion in fully now the study dose we and additional the dose in ascending have a of the also to regimens. to pharmacology the single of profile

course. for have we updates for and there stroke II in begun a second treatment. of As in in Phase due Phase hemorrhagic CAA, II defining we're we study leading And parallel, and cause announced in study provide we'll no existing a Alzheimer's year, this steps earlier the next disease, disease-modifying is which

disease-causing Moving supporting update, to clinic program of we administration to our in disease. distribution Phase targets patients of CNS the on In into program nonhuman we in deep single another the data seek CNS this ALN-HTTXX, disease. as Huntington's the initiation study of a as fatal and repeated we with QX and moved HTT future third the well look I We updates as pipeline broad of protein exciting shared which Huntington and to ALN-HTTXX. the address this neurodegenerative nonclinical sustained tolerability sharing primates lowering in of progressive exciting intrathecal after forward with

As achievement we've of discussed, investing with give to cardiomyopathy, But us a ATTR of in to assuming regulatory broad we've are foundation disciplined a profitability upcoming approval, promise sustainable this potential RNAi of be and in continue strong the in our variety launch continue to to as going about therapeutics diseases treat we critical discussed, needs. unmet resource growth. of also key phase allocation near-term

space, RNAi including financial one that therapeutics. programs of metabolic to announced to an patients, ALN-KHK, our resources development This is of to X an RNAi we've decision mellitus. transformative efforts, decision today for to of furthers commitment our other and treatment clinical, type targeting to our the managerial the medicines As stop carefully which optimize in ongoing part investigational exciting for bring diabetes a clinical therapeutic efforts allocate ketohexakinase

explore for forward to disorders and continue multiple look to targets metabolic updates the We future. in sharing

So in and great advancing platform pipeline our made in sum, to with more progress much come. we've

that towards me working Jeff of great clinical can us and to the by XX include representing in our of for impact anticipate to the proprietary by robust, of it of end disease CNS, liver, muscle to let against a INDs results we're by X that, pipeline reminder, our and adipose. remarkable multiple turn for the end potential INDs areas. With Jeff? over pace in review filing meaningful next the targets we self-sustainable now we a across upcoming innovation XXXX, This the doubling deliver a pipeline year. milestones. have end programs If unique XXXX programs, of a puts near patients financial position new partnered As to and

Jeffrey Poulton

last million XXXX presenting year on with the guidance. Starting results versus The driven period higher in an demand, XXXX XX% ONPATTRO continued strong morning, or when quarter P&L with a sales Tolga of $XX associated compared lower compared payable sales $XXX gross year, and increase a in for from million the our revenue certain milestone to of primarily quarter, which and $XX net decrease double early be $XX of Alnylam's from improvement collaborations XXXX. $XXX compared XX% recognition with clinical product of expenses for of XXXX, costs quarter by XX% was mivelsiran. Royalty QX, QX was margin in driven canceled for did our demand be was primarily payable margin will grow rare same for by a the increased of other reporting was The due quarter than commitments LEQVIO decrease strong for not as sales million margin Roche criteria sales XXXX product to by development XX%, and revenue for for XXXX. highlighted. Net and on AMVUTTRA. sales of The of during revenue the in manufacturing was third expect recognized as franchises a in adjustments with higher the or on I'm previously I what million representing quarter recognized the summary everyone. growth summary more in in product royalties by with Thanks, Pushkal, similar royalty as good occur in results $XXX XXXX. is million, year. pleased worldwide. Gross as continues payment LEQVIO the from to Novartis rate QX was well financial on third revenue XXXX the to QX by inventory Total was $XXX quarter the offset with both driven TTR our million full upfront of discussing growth higher this for primarily rate driven a for million achieving AMVUTTRA. earned for our the

the well the period year, zilebesiran end costs as same clinical expenses clinical by activities to Our non-GAAP KARDIA-X last our with preclinical third R&D with XXX the study, of XX% primarily strategy increased compared increased XXXX, due associated clinical personnel our expand expenses associated mainly which Phase progressed our grew costs. pipeline we with as II to increased in and as quarter to activities

to non-GAAP the AMVUTTRA of Our potential period with increased third along increased compared with same XXXX, to to expenses for the TTR SG&A cardiomyopathy, quarter for due primarily associated preparations our costs therapies the XX% and in in marketing costs. investments personnel launch higher promote

cash, Our was QX of related agreement decrease previously mivelsiran of by with to million non-GAAP as achievement performance compared due recognition from last driven the of Roche with collaboration primarily proceeds a quarter than with $X.X of the December the $XXX milestone common ended more connection $XX employee billion and with net $X.X and billion, XX, operating of stock million, for operating exercises. our primarily the loss highlighted. We with stock securities as compared marketable equivalents cash in issuance a representing XXXX, XXXX, and received the option improved revenue increase quarter to year,

and I'd QX like products anticipate guidance Today X combined reiterating to product updated financial call. $X.XX XXXX for our are of turn our within commercial will that a our for to revenues Now guidance net range our billion. We be we earnings was on $X.XXX XXXX. billion

range $X.XXX Our collaboration for and and expenses a billion. of million. guidance $X.XXX and between revenue And R&D lastly, our million guidance $XXX SG&A ranges combined non-GAAP billion $XXX royalty to remains

ATTR plan Heart plan in to with for Sessions ALN-TTRscXX upcoming Scientific in from and first November. XXXX. Phase discuss for ALN-TTRscXX findings in the we'll amyloidosis development American We at ongoing quarter I that share We Let study me of some turn from present now Phase XXXX. Association announced our the additional and financials of patients III late the goals key milestones

to around II a intend also in mivelsiran study or with We disease year-end. at patients initiate for Phase Alzheimer's

Let our Christine? to me back now session. Q&A Christine turn to it coordinate

Christine Lindenboom

will Jeff. the Operator, open questions. we you, now Thank call for

like in, any get For would then ask back those if to and queue you in you have X dialed to additional each yourself we question to limit the questions.

Operator

Instructions] And [Operator our take Ellie we'll UBS. with from Merle first question

Eliana Merle

work RNAi most Just in makes And done of your about some tissues? your the bit to to versus muscle? and diseases as thinking specific these earlier-stage to elaborate on modalities? or initial you pipeline, ability improve tissue you in you've deliver any are the And adipose advantages a can terms approach how sense to the of what other that targets

Pushkal Garg

Yes. Thanks, Ellie.

work. highlighted, very are about, Jeff RNAi As of tolerated therapeutics, we've protein be is administration. And to we allow talked the human both right? and What we can appear we're profile disease-causing in sustained knockdown these population that that to efficacy about pursue, validate we infrequent which safety by can genetics as as genetics get guided excited emerging a we're preclinical that diseases allows well of well us therapies for terms the of seeing and using

that. the the the update those. time So the adipose progress about bit to earlier we'll pursue. We substantive probably be next actually and us muscle best we'll and of more new that's And highlighted as the give highlighted on programs. an early XXX -- R&D a are Day we specific in And Day those R&D year, in of strategy at are give for year We X to to timing approaches track tissues some a talking that we're little areas we've on our taking. of remain that

probably reasons, say competitive for And progress. to not good point. a making this going more we're at lot we're So

Yvonne Greenstreet

That's Thanks, great. Pushkal.

start these continue one is in ongoing make as of of innovation to to tissues. progress we some of platform delivering I extrahepatic opportunities think our really, it's the

Operator

Wang Barclays. our next take We'll with from Gena question

Huidong Wang

believe very in seems I others like to is down nonhuman in question primates. showing brains. impressive showing instead primate ask very the one I this brains of regarding nonhuman most few knocking one rodent of data It actual wanted of ALN-HTTXX.

looking I regarding that What everyone you benchmark. data? looking I for is So know for? Phase Is will as are the using XX% else you be a something

Pushkal Garg

learned one here into we're that Gena. that what is Yes. program being is CNS our of that able Thanks, Huntington's the tolerated represents And now preclinical encouraging. excited to and moving many appears our be the glad that APP have because program well the I'm clinic. about. we're This it's terms from you in of knockdown particularly sustained highlighted disease-causing of seeing studies to we're insights we third program in a gene very

that sustained an both amount well tolerated nonclinical in includes there's beyond So quite clinic, quite in taking be that unique. seen also we an of that after full-length that, gene, with the as in to as in the very was date. as which as the the approach hopefully primate progressive of actually rodent protein, will data knockdown fragment. the X and our is targeting may in allow nonhuman upstream we portion APP We're otherwise the exon slide, The targeting believe targeting we're we of rapidly something will studies suggests have And tolerated saw, X fragment we've mutant then program progression the highlighted the preclinical very, I and were going literature this benign emerging well species. And the that exon well been fatal disease. in findings help

molecule. So for we an unique have may efficacy this with think we opportunity

a cap can the dose at active United a in that we this start-up useful in and We'll U.K., we'll also to measures, the go green be Huntington Huntington's So be measures have tolerability, is very in we're in also dose we then are such looking level our looking we'll that, cetera, that Canada. clinical -- forward, proceed. These as biomarker imaging And have at safety United as NFL, disease. The light I'll And changes PK, in symptomatic States, in disease. et States. forward with we moving do at in in PD, progression single study levels. study. be the note in patients mutant looking and ascending have mutant in mode

in we all still And a territories green to so proceed moving and light forward. have X we're

may of we cap. and we'll FDA that cover FDA us dose need of to with we'll is lift going enough time. be terms do lifting we'll able work that that if I'm with in. we're give to as over in when It cap work the it that excited dose So forward the interested But doses to coverage very sort that. And the

so program. very excited we're And about this

Operator

our take from Citi. next Lebowitz with We'll question David

David Lebowitz

of on and in silencer has the number point overall ATTR second would market the And at been prescribers impact I inflection your of terms how Have the know polyneuropathy noticed this for that In of the you say coming a regarding you questions trajectory? are time, the additionally, polyneuropathy cardiologists. an in of plenty cardiomyopathy data?

Tolga Tanguler

traction, XX% with growth been Yes. of both as there's in our that up, category and within able we as at And actually still our compete belt Look, quarterly but you'll as X a knockdown well dose patients under see not a patients provide only well year-over-year U.S. our that the second is this is -- actually in need, we significant quickly to versus as dose, you up actually differentiators. the is in that treatment of which are our administration obviously, as, a I mean to the what competitor have route optionalities the rapid their receive our believe services, at the ability that now our our marketplace of in getting important what dosing the to can number good the with we good indicator how we've fact how patient patients XX% the due as States. well to months believe in where will those platform, physicians. unmet XX% competitor. And remain X% our quarter-over-quarter quarterly first is is United on treatment, this up, in fact with terms this as And after really of another as both growth see

of that sure that very appropriately the overall, we would that HELIOS-B, our allow medical good there In part patients But would and -- not us, with the robust launch we've in very cardiologists, something is not make is I'm number are And hereditary ahead affairs phase. in And we compliantly we're that and are continuing when we the see the pleased -- with an around second put, launch established of to performance that address we're terms of in we with that your look, are indication. organization. in the questions demand that triage as that promoting colleagues. But question, not guardrails making the terms to coming be seeing of indication, our of clear of polyneuropathy QX. sure again,

Yvonne Greenstreet

Tolga. terrific, That's

illustrates think actually we AMVUTTRA. a And compelling profile really for it I built that we've engine. a have high-performing commercial

very we're So excited future. the about

Operator

from We'll take our question Matteis with Stifel. next Paul

Paul Matteis

a kind getting polyneuropathy of label? prior neurologist are see? Do need phenotype they of a I relates one data. diagnosis? mixed the in offer there this in TTR before drug guys you're to just that more the impediments in coming expanded But it an prescribing place follow-up population recent in ask you that auth dynamics on now, Tolga, right, question at that just of wanted to solely as significant HELIOS-B light this AMVUTTRA you corroboration to are that totally want And a confine what from, to true have I polyneuropathy. understand where that right cardiologist promote requirements really they to Like, point prescribe

Tolga Tanguler

question. complications a in the grasped the Paul, how Yes. actually that's great some clearly of And getting patients you diagnosed. are

treatment. So a the the if condition the be States, if in especially you're the patient tend have put to as on scintigraphy. in a having And they if through then go cardiomyopathy would physicians, you're is actually to cardiomyopathy, quickly -- suspected, determined of United suspected

neurological disease. would our hereditary, that. sure manifestations actually an you testing to through has be would make in that treatment. on because indication have you of put also to then neurological is workup the that And only Now validate And genetic a go the that not would polyneuropathy, actually patient additional then of you the

sustained access of nearly in getting our channel they of end regardless what good patients reimbursement access have. of of challenges, terms Now actually source all the up

what that's So first It diagnostic the the see stage rather challenge. the stage in of being not cardiomyopathy. is mostly the pathway really the challenge we on that than polyneuropathy last hereditary pathway,

Operator

TD with We'll take Cowen. our from Baral next Ritu question

Ritu Baral

thinking What label patients to ask that and potential Jeff, potential topics even viewing are how label on are payer number how discussions you discussing? for upon of wanted you're are -- maybe, ahead and I the how, of now expansion AMVUTTRA? the just the levers expansion. you Tolga, And about are potential volume-based increasing discounts what

Yvonne Greenstreet

question, great a Ritu. That's

for I think actually Tolga. it's

Tolga Tanguler

as the in the really have the proposition, shared important outcome. driven the product really payers what should of today's as they clinical population. Ritu, provide CV patient So as terms outcome we differentiator TTR care, by is and in HELIOS-B, And is in Day, be, incredible we the demonstrated which at an value does

that's So we're understanding, seeing the remains compelling. what albeit HELIOS-B results that so is this what are a fact the and far payers that rare, disease, growing acknowledging outcomes --

terms able we're that and there our not communicate PDUFA rebates. as more. disclosing do in this. philosophy $X,XXX less we've going of of yet. we've of end co-pay we're how to is is actually seamless with part burden. And we indication, co-pay having has to appropriate polyneuropathy patients, the not will or great able understand area with up having a them appreciate more the philosophy, them about set within patient get get lot be do and of to our to XX% value-based to be access access an to majority is I a We access which been our and where This has obviously think access been to agreements patient why to our But we going to what interest and date, any about burden closer navigate how landscape, In demonstrate able

Ritu Baral

Paul's, It's it. ask Got neurologists' I'm cardiologists' question? into of squeeze I guys, quick compressive if to Paul's follow-up, just question. and the in. How a to factor a going understanding on is neuropathies this very And so follow-up Paul's can

Tolga Tanguler

or You spinal tunnel carpal stenosis? mean

Ritu Baral

that tunnel, pathway. in Carpal Yes, stenosis. spinal

Tolga Tanguler

I have TTR Look, you Yes. think treats and this a multidisciplinary that actually mean, that's treat disease. I systemic disease, that physicians is -- why multicenters actually

by of there a genesis product because cardiologists the the Now that's they every have and exist preliminary cases with academic collaboration time. of disease the in do -- the go surgeon. But the centers, course, approved some the to believe change first this even of most But every the evolved available is been tends academic they doesn't tend obviously has center. good -- -- scintigraphy orthopedic and neurologists, going of the also, even stop involved in diagnosis. every the the you in to and not disease over we cardiologists, if today. disease, And manifestations cardiologist where to that's actually be do only additional And between suspicion

Yvonne Greenstreet

a Ritu, of and to actually, physician question part because symptoms, important great forward. I I think, education be awareness going it's these red-flag an going are think

the for X -- questions. question the thanks So

Let's next take the question.

Operator

Sachs. question next our take Salveen Goldman Richter with We'll from

Tommie Reerink

Salveen. Tommie is on This for

of wondering in of talked there kind and of So initial fast year the frontline aware focus are progressors be half -- what's this if the how thoughts for you cardiomyopathy. if could second a bolus new? on want But about who something there on is your next

Tolga Tanguler

today's Yes. strategy we I to mean suggests at our that the with explained a delayered And patients day, the we lot and are. reason strategy. has where TTR the why data do what

is this disease. highlighted, fast a we progressing and So as severe

is to are with look intensification. portion to early, number and of to they treaters currently ever understanding the only the where patients available, believe these on GLP-X, subgroup be patients stabilizers -- the on is seeing. And our the at is focusing are larger dialytic as dynamic see are patient they're really category. to providing or population, into going they where of sure or diagnose patients, are coming with make that Those want that hard. Hit they a hit the we today's the of patients are with if them you market, them that want the soon as diagnose they they hard we that product data in a So that

to the want yourself a can the first-line of as able the because progress be those establish product the nature get disease, of data because actually so and So fast patients you treated. to about of

either course, depending the a those patients, them, start And of them on Now they on that so. a I'm stabilizer, eventually, may will already those on use in switch access done or or will put that either limitations might sure terms have, patients, progressing be physicians patients challenges probably if they of combination identifying haven't therapy.

we identified. But category to of that number do some well into standard be that care that progressing, no believe to take be patients given the Therefore, that's the will tafamidis AMVUTTRA are therapy. those a category. patients important it's to identified, established important are generic, the become those been that, are is doing highlight think positioned in patients data first-line are dynamic it's believe part we and well I that switch become our coming obviously, highlight of and the time sure we we And will will that that until have, this make establish. to will there behavior Now to

Assuming course. approval, of

Operator

Fye We'll question JPMorgan. from take Jessica our with next

Jessica Fye

there in with the I one of for to for relevant acoramidis guess you're another TTR. going in do scenarios for launch watching got expectations as product label anything cardiomyopathy? be see back, I own that stepping their up? to TTR PDUFA that And you potential coming any on Is your the label your

Yvonne Greenstreet

the the the we've to approval. everybody, label, priority in acoramidis forward and feel and generated assuming next year. voucher in approval our mean I early confident Like interested And review we actually process we're through that we're Yes. moving U.S. Europe regulatory data potential obviously, assuming about looking the a seeing using in very HELIOS-B. both with

acoramidis. wait really also we And gets have to are. feel delivered what respect exactly we where think we So to I see with about to good

Pushkal Garg

patients do twice Yes. stabilizer. progressing broader are those comment acoramidis to -- would compete the The I perspective, able population [ that shape, be lot daily to dynamic action believe would we to will that dynamic a ] with the how add in first-line will I speculative. to market how a a be only be own market obviously, their we'll still, have is, on of do that think patient are label mechanism stabilizer. But from And thing a our would on agent again, of is or and sort we

Operator

question We'll Gary our Nachman with from Raymond James. take next

Gary Nachman

in the be anticipate doing? this U.S. of opportunity patients in in accessing both year and CM we the remind vutri us also spending relative levels think the of next U.S. infrastructure we in year should relative and you'll you how have SG&A pricing. then ATTR-CM terms of Europe, to the magnitude. of activities the how as to and and launches commercial about think the EU, both of there order then Just And you So for the all

Yvonne Greenstreet

That's great question. a

it, part potential his. of execute then on plans I first follow think But we launch. with up will plans Jeff for AMVUTTRA take for Tolga and of the will making that kind our sure

Jeffrey Poulton

Yes. say we HELIOS-B that right? OpEx, to year the reflected I confidence guidance mean in and QX results would playing win. of at had sort on And we upgraded this our that I

in prepare half And so you'll in been very second investment. that's U.S. progress launch, the see in the in this team done. for year And commercial the hiring the with year made particularly good of starting to the an increase the has

of But guidance half So have with the as in provide terms year. certainly we'll a second the in second would year-end invest for that lot next the obviously I in And then but of call annual our continue specific Europe. launches the year I for even we'll more in on happening our expect year, 'XX create February. of SG&A. guidance, growth in of don't growth towards double-digit U.S. move we that's going to half the that just to broadly

Yvonne Greenstreet

very in is great been built what's actually No, actually we've strong polyneuropathy. foundation that TTR Yes. a

of successful that anything of absolutely we're well the been it's to else in sort be that... positioned to indication. So more sure a CM make really growth Tolga, incremental But

Tolga Tanguler

organizational establish only rare. in would add excellent results, end-to-end also perspective, TTR in which organization, is, an the I thing only focus an actually I but not mean has we have demonstrated from

TTR our just really growth. Japan, numbers. in a growth also only but organization, not demonstrating Europe, XX% robust year-over-year So has we've posted I mean in been

quarter-over-quarter. demand international been If up you business, it's -- performance the our at of look on of the side at X%

done we narrower priced indication. that able is XX% with that's at become we market indication product PN for a in where in been have also price where that there PN been them a leader sensitivity, market that's the we've significant a in indication. tafamidis compete versus Now And where to specific

pleased indicated, going established. providing right that we've are we are with the markets. So and quite And options to we to in play win structure we're that make those actually as sure the access Jeff

be we'll we that And a and do the so well, far, tailwind. been compete believe to to data we've able HELIOS-B able with as

Operator

next from Biliouris our with Kostas We'll question BMO Capital. take

Konstantinos Biliouris

of and whether Difficulty] is internal drive [Technical potential in robust that comment growth. can potentially could you that the growth activities additional your I'm on outside addition to large BD and even very pipeline, can acknowledging efforts. wondering your pipeline support

Yvonne Greenstreet

And privileged of end we'll very mean that incredibly kind one think the actually pipeline of touched that's it's doubling on of have the be pipelines Pushkal the 'XX. pipeline. Yes, we a exciting most fact the to great Look, rich industry. an I the in question. we're by

a a to got opportunities interesting really lot clearly, we've going we're successful in make the AMVUTTRA launch. sure focus So company. And of that really, also we on

described to considering enable internal platform BD, the really all tissues, to that platform. What pipeline our well so and Pushkal to about us open access really we're and we're continue some driving other help continuing And also opportunities focused our optimize as the of on both very think as we these.

landscape the company. all in attention the awful a privileged of have position have And lot -- us. in to really so a we pay our we innovation within happening of move obviously really the to progress we to that's can lot that excited that moment. Clearly, forward. around at what's focus we're But an We're very

Next question? I last think it's question. our

Operator

We'll Bank take our last question America. with of from Tazeen Ahmad

Tazeen Ahmad

for Maybe back this is the going last Great. to one Tolga, launch.

think is early it's is written In that disease rare patient. But from dispensed of I take the the since to time about time the the time expectations, talk of traditional, from terms the indication to you true launch? PN time for at Or the conservative you second expect smoothed least already those that sometimes to to the as period, script hold been your this going going the any your to do for script product, part launch, companies out initially, to lines extended here? be have time is do expect to

Tolga Tanguler

the closer to Yes. as this we do to able launch. area will where time do an the we Thank is that should some need to you, have. and that we context additional to that get be provide provide launch our we respective And and we engagements lines Tazeen. additional recognize will closer color, We

Yvonne Greenstreet

It Tolga. delivered pipeline. one as joining the growth everyone continued quarter was well of call. We And look, the very thank third in We on you, progress. us Thank to robust for our pleased you with XXXX. advancements as feel of financial

have you, everybody, thank and great a day. So

Operator

today's Thank teleconference. concludes That your you participation. for

may now You disconnect.

Alnylam Pharmaceuticals (ALNY) 31 Oct 242024 Q3 Earnings call transcript