ALNY - Earnings call transcript Q2 2024 | Alnylam Pharmaceuticals, Inc.

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Operator

Good day, and thank you for standing by. Welcome to the Alnylam Pharmaceuticals Second Quarter 2024 Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded. And I would now like to turn -- to hand the conference over to the company for their remarks. Please go ahead.

Christine Lindenboom

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

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

for an outlined introductory context. clinical questions. call, review as pipeline on on provide remarks Tolga milestones by followed your provide today's Yvonne review offer and summary Slide financials commercial During will progress. global of before general and some will will call will progress. upcoming update our open X, we guidance a And Pushkal Jeff and updates the

may those this Act harbor constitute for forward-looking factors, Actual materially Private a in of those of the expectations, contain various report file you like concerning remarks remind safe SEC. indicated statements future forward-looking statements with Securities our of which as Reform I would discussed on results by the plans provision to prospects, including recent of call important will and these periodic the Litigation result purposes that XXXX. differ most the Alnylam's from

to of In subsequent date our should views disclaim addition, as date. any and views forward-looking We statements the such this any obligation of upon any our represent update representing recording only of not relied specifically statements. be

Yvonne. With that, turn I'd over to call Yvonne? like the to

Yvonne Greenstreet

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

in in as for TTR year-over-year midpoint. growth the year-over-year guidance revenues, XX% net the quarter driven which Commercially, for was achieved revenue raised The generating net such, XX% our Alnylam. growth is revenues, product of our XX% primarily transformative one by And global product year by XXXX business. at we've second $XXX million a in we

be a cardiomyopathy. in established prepare potential for over as success current key X will we that the next significant growth to years for capability ATTR our year to business continue The drive our and last commercial a we've will launch

To positive cardiovascular in that to mortality in HELIOS-B placebo of of including showing major benefit improved vutrisiran was a QX results study Phase line vutrisiran XX% and announcement III encouraging of the XX% that the outcomes, highlights ATTR compared safety. end, our demonstrated to cardiomyopathy top

These results reflect approval. these franchise be of to mechanism for therapeutic establish what to a results line profile Moreover, care assuming believe ATTR anchor growth the the top and of cardiomyopathy, believe commercial and highly differentiated robust for Alnylam, true positive value as vutrisiran action, of driving power positions new we come. can we regulatory standard as in the creation an vutrisiran years supporting many

we an biotech, results. become track goals, engine, exceptional for driven and delivering transformative financial eye organic top-tier our towards product in medicines are and diseases from firm commercializing by With a PXxXX pipeline are prevalent we high-yielding all to world first best-in-class our product on and/or candidates around that patients while of the a belief our rare Alnylam developing with

tell you to the in that City will hosting October it Before we about Wednesday, be AMVUTTRA are leadership excited live, of event, I webcast York undertaking This amyloidosis, Investor to over our the be on will on turn that X. ATTR efforts optimize in in cardiomyopathy. TTR of launch I'm ATTR Tolga, Day focus the including which will success we the to New

hope colleagues us several can shortly, that external and be Alnylam as from New York I this join of experts. more my hear You'll on We'll about you details sharing as in October event well X.

our for commercial me a performance. Tolga? review that, the now over call Tolga With turn let of to

Tolga Tanguler

of XXXX portfolio TTR rare therapy another morning, growth our of the franchises. compared delivering portfolio, second in continue commercial our we on consistently product net good and Thanks, Yvonne, XX% and as QX quarter quarter both to our patients across strong was number sales everyone. with increase for of the

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

compared Our first the XXXX. commercial of second there TTR representing franchise XX% quarter compared strong from and belief XXXX This our our X achieved $XXX significant opportunity a the increase million now increase a net with product a in supports with hATTR-PN. years approaching quarter remains global revenues, of launch, growth, initial XX% that in

on patients small We still estimated percentage patients or are X,XXX with globally, of from a tens devastating of disease. this of that ONPATTRO approaching the AMVUTTRA suffer treatment thousands

Now U.S. let of our world performance. provide and rest me highlights hATTR-PN of

in quarter XX% primarily by first driven adds In are the to strong XX% TTR QX, of as approximately U.S., a to and by our switched year-over-year have robust increase combined continue of demand offsetting in sales our and with ongoing the a XX% U.S. patient end was than worth momentum more patients It driven U.S. drive following: patient AMVUTTRA increased AMVUTTRA. the on the on growth. uptake, compared year-over-year strength in the XX% decrease noting AMVUTTRA that the the at of growth ONPATTRO by The ONPATTRO that of AMVUTTRA. by is new

increased AMVUTTRA turn patient remains our robust, by months. for where markets including year-over-year with XX% was AMVUTTRA growth The where first compared year-over-year. in from me uptake demand markets, to increased launched the let successfully growth international quarter recent and strong our also Now franchise as the driven we primarily XX% TTR by

in the partner orders to markets, growth. Finally, year-over-year of particularly Brazil, also timing positively our contributed

the treatment regulatory which ATTR forward patient HELIOS-B with estimated ATTR bringing We unmet approval. to tenfold As opportunity amyloidosis have patients product than is results tremendous amyloidosis significant looking is cardiomyopathy, profile the a we leader. to shared believe transform our assuming vutrisiran previously, successful prevalence is based and hATTR-PN, where market cardiomyopathy, team review and larger with AMVUTTRA with the in an paradigm needs combined recent to with the on

schedule given deep and effect, in knockdown profile CV unique potential We events believe reduction of site-of-care and rapid source unmet quarterly with progression, ATTR-CM, its the ATTR-CM to well and payer on differentiated first-line the the the needs in standard hospitalization dynamics. mechanism of and favorable measures care mortality patient durable, substantial address disease an a of at as become flexibility vutrisiran has dosing of highly as treatment and with market-leading impact that attractive TTR is enabling

to address critical standard We've understanding committed as built been foundation the our strong the of amyloidosis needs this same and past the deeply efforts unique of a vutrisiran our have ATTR treatment will community the in a care benefit establish time, options recognize in and to tremendous of of ATTR-CM. At potential importance capabilities advancing scaling to over ATTR-CM. the decade that we

the also from not all preparations we supports seamless patient leveraging access industry quickly our capabilities, To support enabled major that Alnylam U.S. us TTR Europe to help with partnerships the integrated in track and therapy credible Canada, have and this our and health the markets, deep their teams our our foundation, form including end, physicians, including exceptional patients of also that and centers, strong access patient highly transitioning a our for are system and services record that only and patients seriously expanding creating award-winning but start to experience fastest and in global patient a support adherence. team across our and timelines and with launch of strong customer-facing delivers specialized advancing relations one Japan payer treatments global

of a in unmet and of as the second XXXX versus grew franchise significant a remains our first sales of and business that delivered increase quarter to patient The solid proud quarter the performance OXLUMO, to established addresses base. rare $XXX the combined revenues during have patient quarter with disease we sustainable XX% GIVLAARI The product also X% continue global second need. We're disease which million rare XXXX. compared

For of XX% with world by driven following increase X% in GIVLAARI, rest markets, by in U.S. highlights: in patients which decrease a of markets. growth therapy; product in regional in the an European QX compared second the sales demand timing markets our our offset driven primarily increased quarter across of orders growth new the partner by X% on XXXX with primarily

demand For from XX% delivered European growth, growth partner the by we year-over-year following rest U.S. a dynamics: growth increased by XX% both a markets. XX% robust markets increase our driven world in our with a primarily in demand the of OXLUMO, driven and regional

that the in at of year delivering be more rare both delivered quarter the upgrading robust with year our and by our strong will In in of full range. patients franchises on half XX% share pleased second our revenue growth product to and Accordingly, therapy later. today we conclusion, and continuous on first TTR we're net guidance Jeff the results revenue. guidance midpoint

now pipeline Pushkal? progress. that, our R&D turn over it and recent review to will I Pushkal to With

Pushkal Garg

Thanks, morning, Tolga, and everyone. good

of vutrisiran top As quarter III exciting was patients Yvonne introduction, from ATTR and with the second study Phase highlight major of her in announcement the of our HELIOS-B positive the line results highlighted overwhelmingly cardiomyopathy. in the

high demonstrate for efficacy truly outstanding primary highlighting on study clinical therapeutic we the action in observed that results, have now extraordinarily powerful improved the impact RNAi X its both these the on an ability first endpoints mechanism With data secondary and vutrisiran vutrisiran statistical this We cardiovascular of all of populations, with bar of prespecified with significance outcomes. trial of the meeting in disease. study and an this results

These KCCQ by and key outcome XX% and overall including and populations, effects class, were all clinically all-cause baseline benefits CV and in benefits events monotherapy the observed subgroups, XX% mortality NYHA key disease recurrent all respectively, in in and we measures the and who reductions walk, in of of We monotherapy are on Specifically, on of tafamidis. in are X-minute progression. accompanied XX% reduction risk the populations, respectively. observed consistent overall XX% which significant mortality all-cause and patients

a this the from Friday, And and consistent Congress hotline XX, XXXX, will as finally, We're of that presented detailed tolerability, vutrisiran profile. be its in oral European study London. HELIOS-B Society announcing Cardiology on demonstrated presentation the results established August encouraging with the safety at morning

call and congress host the with during conference discuss webcast We will results investors. also detailed a these to

regulatory filing regulators work engaging the XXXX in a with work NDA States We're also thereafter. hard late global as towards in United supplemental additional we with filings at

of be accelerate to priority quickly as in patients review voucher a possible. reminder, U.S. as we this As using medicine our the review regulatory to will hopes bringing

HELIOS-B, made to RNAi breadth across addition pipeline a programs, In other we the highlighting progress of we platform. opportunity with have number our of

top showing on results Phase and standard positive good antihypertensives tolerability discussed our we II in call, last on recently earnings reported hypertension. controlled the we of patients care zilebesiran, efficacy from As study inadequately with of of additive KARDIA-X

or patients who KARDIA-X in high to patients cardiovascular pressure our showing cardiovascular of in cardiovascular risk. control forward on outcomes top demonstrate is cardiovascular now in by with mortality. zilebesiran that, running reductions look we underway, to following where blood agents high And at [indiscernible] goal of is morbidity evaluating trial, X and are more the benefits a risk

remains in ALN-APP angiopathy. We're have initiated type highly also most the with the Capricorn study. the also in cerebral Part of is dosing need. and Phase the for formerly in of unmet amyloid initiation II Phase excited with cause second study common which underdiagnosed advanced diabetes intracerebral X with mivelsiran, patients X ALN-KHK, hemorrhage of We've I to phase major B of the of is development CAA treatment

continue has set date a to submitted Our Brazil with the Sanofi partners XXXX. fitusiran progress for as XX, where for and regulatory target filings in action China, March make FDA United patients States, of great hemophilia well. the

with We've also to our collaboration of a Regeneron. announced updates today pair ongoing

upfront received and therapeutic First, of to a if we asset a exclusive monotherapy. gained diseases, low we amended and complement-mediated milestones in sales regulatory on million agreement approved. RNAi In exchange, Regeneron investigational for the the royalties certain treatment which the receive $XX as development license are to eligible for double-digit rights under payment the cemdisiran,

and regular of Additionally, prioritization as of to review co-commercialization Regeneron opt decided has part portfolio of and out mivelsiran. co-development further efforts, their

address year. needs have now around patients first in Phase about we've full end We're all we CNS study to CAA, we rights of to the just mivelsiran start excited areas: the disease at where the global initiated potential Alzheimer's this where unmet and to in II program, of significant the a a or plan and disease, II such, study; X As Phase extremely in of development commercialization indications. our the

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

position to next doubling and pace puts in robust, to of reminder, of and the proprietary of end file representing of unique deliver XXXX we self-sustained by the a a include of end CNS, If year. the areas. anticipate programs our us As muscle plan INDs we for new partnered This INDs against patients to end targets programs, meaningful pipeline great X a impact clinical in XXXX, a adipose. across can that innovation by multiple remarkable a liver, XX we disease by have pipeline the

me that, With now financial to Jeff? let results it upcoming Jeff review turn our and over to milestones.

Jeffrey Poulton

morning, discussing full a be year XXXX good pleased results I'm and guidance. and presenting everyone. financial of summary to QX Pushkal, Alnylam's Thanks, our

with product demand, P&L Starting growth was driven and our strong the same million for growth previously strong with the summary respectively, XXXX revenue and in continued as a reporting of highlighted. compared quarter our for TTR or period rare both XXXX results Tolga by versus XX% QX $XXX XX%, of XX% XXXX. primarily Total with franchises

collaboration collaborations was Net the was $XXX revenue, million, previously compared the XXXX. revenue quarter in for Regeneron, $XXX with million a increase deferred. The driven agreement $XXX resulting modification representing increase QX when primarily from with which of our was million approximately by of

higher driven what to demand recognized Royalty $XX in revenue for quarter the sales was as the by continues worldwide. Novartis quarter was or XXXX. than for more Xx second LEQVIO grow of was increase LEQVIO The million

Gross the inventory, primarily by growth higher quarter were pace. will of sales canceled improvement in product other be XX% with realized in compared lower paid the The a margin was on AMVUTTRA expenses margin driven for expect our sales in XX% for for balance to XXXX gross higher second commitments brisk the product royalties similar XXXX. associated is margin of AMVUTTRA to the quarter on on year ONPATTRO XXXX. not in adjustments and I for as continues manufacturing at costs with due which

development our primarily increased increased associated non-GAAP activities, to with with studies increased XX% zilebesiran and period increased to second and for Our prior the in the start-up expenses. the the R&D same expenses activities expenses preclinical associated employee mivelsiran clinical compared quarter compensation in costs year due

increased the primarily expenses TTR quarter increased associated and SG&A year non-GAAP compared compensation same employee second due increased marketing Our XX% promotion the prior in of to investment expenses. in period to therapies with the our

product with for XXXX as revenue as collaborations the representing driven operating $XXX primarily highlighted. previously $XXX QX strong results, in million sales non-GAAP a compared top well income line as was by Our million, quarter improvement both from

the of of of with with the billion $X.X product ended common with increase compared stock and securities December increased primarily quarter from second to increased with issuance We proceeds revenues connection cash stock due employee net net cash, marketable and equivalents billion $X.X XX, option XXXX, exercises. the as in

profile. We believe to self-sustainable is bridge our sufficient cash continue current a balance financial to us to

the components year guidance full are to where updating follows. Now I'd specific our turn as details to XXXX like our we all of guidance with

billion of increase revenue hereditary guidance from guidance are ATTR range of an to a $X.XX We to ongoing driver primary net franchise. is the billion, strength the $X.X our a increasing XX% to billion updated the product performance The billion of our midpoint polyneuropathy $X.X to range guidance. from prior of of representing the increased $X.XXX the in

with primary $XXX of which quarter. the guidance recognition revenue Regeneron, our approximately We revenue from previously to million. are of agreement to million the increasing and $XXX of million of million a in collaboration of The deferred the $XXX $XXX range collaboration a to this range modification is $XXX our driver royalty increase million of resulted

And we to $X.XXX guidance a combined our for and increasing There X $X.XXX a billion to drivers SG&A of billion range from the $X.XXX lastly, of expenses R&D are billion. to range $X.XXX are billion primary of increase. net

investment the now mivelsiran is of will opt wholly owned as by result out by in R&D increased the Alnylam. program First, Regeneron

increasing cardiomyopathy XXXX for are our preparedness in prep we results, to launch optimize launch efforts our line top HELIOS-B strong U.S. early the given Second, planned the ATTR an

and goals we within ago, prepare in XX%-plus ATTR underpinning successful confident include ability Alnylam are changes, and study established period our our revenues the the cardiomyopathy financial which PXxXX a these very for income launch rate that efforts with next strength along a period. the annual years HELIOS-B achieving our Given in year, we strong momentum the guidance to achieving the non-GAAP of X.X sustainable to business achieve results compounded and both total growth of across the X-year operating highly

goals, upcoming discuss some XXXX. and financials and slated from mid turn for now me key Let late milestones

we the ESC HELIOS-B on detailed As in be earlier, Friday, will London. results presenting from August was at mentioned XX Congress

and webcast data. a the will be call discuss to hosting We also conference

that from This our New event, of October experts to also At be We've today Day City. we to York feature TTR announced senior Alnylam highlight well as the as this host Investor will on in leaders external webcast TTR as X a we will plan potential well. business.

mivelsiran B We're application to patients using submitting of a of I supplemental study voucher. drug new to FDA for priority from results Alzheimer's the Phase review Part disease. vutrisiran plan a interim with report in We the

disease a We with a study ALN-TTRscXX or for of expect year-end, at of in Phase cardiomyopathy applications remain carcinoma. And year-end around by and of ALN-BCAT study trial X patients Alzheimer's III new we year-end. investigational drug track in the end mivelsiran on around to initiations or study with in I including X XXXX, ATTR by II at a patients Phase Phase file hepatocellular

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

Christine Lindenboom

Thank Jeff. your we questions. [Operator Operator, the for will call Instructions] open now you,

Operator

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

Unknown Analyst

[indiscernible] Ellie. is This on for

this, discuss So Part pricing know potential Part you're can difference and between polyneuropathy landscape in thinking versus cardiomyopathy? the of B ATTR Medicare the reimbursement the And changes? could difference about given how affect the IRA I especially cardiomyopathy some D in for you in AMVUTTRA.

Yvonne Greenstreet

Yes.

we assuming of with so much that's emphasizing Tolga. off performance question just approval. date to want may the patients how cardiomyopathy, with specific you Maybe commercial start to positive to for really label include I we opportunity expand and as question. to and a polyneuropathy think both in the by to are us, Tolga, respond pleased regulatory ahead see that

Tolga Tanguler

and been therapy able seamlessly, we've our patients and the of actually have on Yes. prior position to growth that we to actually deliver think I in services our Specific support thanks it's terms patients preferable PN, testament on of where patient access, and quarter this quarters therapy. stay that the the to get a

access lot again, injectables principles, as well do access to for product that, ability And which unique leveraging quarterly as a Part be we'll of that without going possible. we'll future a as given little on patients be a is level similar other with in be meaning our or physician stay burden to the to as -- has as believe -- actually therapy those sure of to available co-pay administered, products. we profile of and have on comes available make we the allows When seamless B, in it advantage to CM, secure patients patients. are to our obviously And to continue that they compared

Yvonne Greenstreet

That's great. Tolga. Thanks,

Operator

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

Tazeen Ahmad

physicians Mine top from it use is product definitely the for and on HELIOS-B, feedback from perhaps which share there expectations market line you can of were opportunity. clear that impressive, seems you've line. gotten about vutrisiran, Based results and their on that the a first is

of sense I could that think physicians gotten to first investors better of up get line what and question? would portion of being what that from like kind market a you've end on feedback

Yvonne Greenstreet

great in is rapidly set to progressive is there that's and we up clearly, care. unmet disease, fatal. this a need which have And be question. an ultimately medical that's And a of profile No, well here Tolga? standard think absolutely we

Tolga Tanguler

other vutrisiran and we potent is And we believe in therapy positioned with I the given progressive, we treatment is be option to with HELIOS-B first-line and going mean most imperative to the top this disease, start Yes. line as really agent. what areas it be see debilitating results, to fatal any the the first. well, well ultimately physicians, essentially from know

cardiomyopathy And had bar tafamidis. the the at HELIOS-B end high against backgrounds Look, date. patients completed SGLX, incredibly aggressive the this contemporary yet, And of I is study real of add physicians mean, contemporary know, else a don't the I most to day, want to Pushkal, this showed to impact. first-line if believe leveraging we backdrop, in on I treatment. but this, key is going a be profound anything you driver around

Pushkal Garg

Yes.

looking obviously, what an repeatedly disease. this heard I action as Tazeen, that said. I we've mechanism they're everything I think but orthogonal on for you early think of with building impact is days, And could from have it's think, that an clinicians [indiscernible]

we've the incredibly are excited. shared the and data investigators with for presentation authors the deeply And that ESC

looking that being think shared the we're so And roll enthusiasm so data data we're to able forward lot I of based we've but on to seeing those far. out, a

Yvonne Greenstreet

And that. at of course, come look ESC, to and forward we more to

Operator

We'll question our Jefferies. take next with Raycroft Maury from

Maurice Raycroft

ESC or types kinetics the report what curves I'll you'll for I'll upcoming mono wondering talk for details if combo study on to about maybe I'm plan at Congrats quarter. you the conference? more just data. events disclose -- and to the in subgroups time about versus of KM the ask different the

Yvonne Greenstreet

the on Clearly, question, as we finalizing Maury. details speak. Great working we're Pushkal?

Pushkal Garg

we we Look, expansive points Maury. authors with investigators the into working pretty plan Thanks, in finalize on of Yes. the are But the go content. and the sort to community study, top at to detail presented really able line investor be the excited now that the if the community. we we the that broadly to share think both we're to and community, results -- clinical with end

will include anticipate baseline this this -- So characteristics. will include I

other of was about population very treated how patients effective heavily medications, is hear You'll of with sort this a contemporary agents. that

results the secondaries. all endpoint, see and You'll the including the primary on the components

we'll of key on secondary a be the for And the see primary curves variety endpoint of consistency And endpoint tafamidis. -- some across based effect we'll see baseline the in subgroups. specifically, be across mortality. And overall of sharing also you'll curves sharing all-cause the you'll that and and

fulsome in We're we get rich data to opportunities So the incredible I can out think of there's sharing at pretty to how other ESC. into a a much we'll present lot results. this limited course, dive come. of that the be have of weeks of in you'll deeper clinical we'll HFSA. And at And view trial coming data

Yvonne Greenstreet

Thanks, Pushkal.

Operator

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

Unknown Analyst

Phase Paul. status for ongoing? the I what you the Is you'll endpoints design color And the what potential further, well. of that the little and at? more in HBNA looking II it program, then like on bit about just I can obesity Alzheimer's a see look I anything is And Pino wondering, still missed with that as be curious didn't program. provide Julian to unless ALN-APP was respect could This on [ ] on

Yvonne Greenstreet

Look, very [indiscernible]. No. It's well. sustained to delighted Yes. demonstrate and for program. with my the rapid And robust advancing indications at the are progress view Alzheimer's talk maybe beta X able going been tremendous there's amyloid disease feel cerebral progressing, we're [indiscernible] reductions that about future. of ALN-APP well, we the looking the in potential SAPPL Pushkal, we're both and we We've and and in angiopathy.

Pushkal Garg

Absolutely.

X biomarkers So questions building Yvonne's very soluble APP, exciting beta, continue taking and on that beta We showed well. and with and had and actually but that some also downstream just in your encouraging data XX that involved AIC disease out that safety the program and we a a see of disease-related at both APP presented program. XX alpha consistent are Alzheimer's knockdown CAA as coming in to of

We're excited. So track. remains that on very program

angiopathy, talked disease well. And in a recently cerebral off noted, Alzheimer's the we study second starting study cause about, be a we've which is will as leading we amyloid as As kicked like in of hemorrhage. you

about trial to of and We with the but shortly. aren't working you with we'll that. our that specifics -- obviously talk design through ready We're share

to But we're mechanism, which very favorable burden. the be as this know, Alzheimer's, potential disease of a space think, very have tremendous you disease has upstream impact in about I and a hopefully encouraged on differentiated all its this

our what there's I any in here. based I the the to in With on you X have other programs to question just the of regard on specific to X don't -- was say I a very give want your pushing of heard look, than think the to on ] strategy we think move excitement that programs terms really we platform early around updates X comments main [ [indiscernible], second are preclinical forward multitude aggressively

year, end programs of the due 'XX pipeline are you those by the X next a we give we'll on INDs on INDs track But course And in for of our updates and this so and by specific X-plus keep of doubling proprietary end year. posted.

Yvonne Greenstreet

Pushkal. Perfect,

Operator

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

Konstantinos Biliouris

This progression where on showed [indiscernible] the A some quick actually quickly, was rate this all very switch is Congrats on with to recently polyneuropathy. ATTR then progress would And data similar that natural published study history from us tremendous dynamics good expect under question you to here. study patients that on Any progress halting. [indiscernible]? ONPATTRO read-through and cardiomyopathy? disease to the similar almost from

Yvonne Greenstreet

Pushkal, I think... Great question.

Pushkal Garg

Yes. Thanks, Kostas.

citing think of a on that proportion were So at out of put on patients. a and and were of a I and that you that polyneuropathy been a of had progressed have European paper These polyhereditary, to those tafamidis recently cohort patients coming was disease. some actually stabilization experience patients looked ONPATTRO published who were seem

I it's a progressive a progress with Look, very have Patients this polyneuropathy disease. know do We complex that do with is think encouraging. and -- cardiomyopathy.

what And I a for so here highlighted address that patients don't there's disease. therapy need Tolga progressive an additional any of unmet earlier think can that question have there's to steadily modes is fatal very and

even an of very APOLLO-B EAP, that that we we the study is on by fact data, data get we launched additional mechanism on stabilizer have was and action, need to tremendous highlighted for this all an there are quickly. APOLLO the think who agree. [indiscernible] And to so therapy And I patients and orthogonal

where opportunity the are the and for [indiscernible]. have now for we PN therapies patients bringing about that forward we're patients need very think the we we as patients excited with additional So CM

Operator

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

Jessica Fye

Can would another you update? year? do I kind B that partner curious, you Phase ALN-APP? expect seek you owned? now we we Part that wholly around of plan level later study Or as communication venue the think of results the for set the interim for ALN-APP should detail the how So the And or should stage about for with this to keep

Yvonne Greenstreet

I actually mean we're question the to second be ALN-APP. take delighted Well, I'll first. progressing

indications. We excited or amyloid disease Phase II with we're cerebral to the the X year-end. start in at study about As our very and I patients said, in Alzheimer's kicked already planning angiopathy around off

pleased and forward to pipeline forward look program another moving be ourselves. to wholly couldn't our these have programs in more So we owned

Pushkal Garg

Yes.

Yvonne Greenstreet

Any other comment?

Pushkal Garg

starting Yes, to and primary what I terms on about of talking to the study data. specific see multiple be comment venue you just again, the doses data some early a to little with we'll about is safety your of, in think is you'll on the where question. purpose But tell see the It's pharmacodynamics.

CAA, you'll with that seeing again, more we'll study the we'll of so kicking I Over dosing. And a that off share in Phase II fact able soon this we've off kicked what II and to of program be Phase be our more is in by be that. Alzheimer's think, confidence the time, multiple in is But coming highlighted data disease. out and

and very -- excited So we're we therapy about continue the investigate potential couldn't to it.

Yvonne Greenstreet

be very, forward. need. to Huge very we're potential, huge medical moving pleased And

Operator

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

David Lebowitz

I'm since now, the [indiscernible] drug's nature how of evolving to of with you for the relative have launch can competitive curious, certainly, the a couple all? commentary a since the on new therapy? been has market change at on market any make is physicians discussions the quarters And

Yvonne Greenstreet

Great Tolga? question.

Tolga Tanguler

before, still tens increased Yes. addition I this share thing. a with devastating discussed good is a treated. of is and get mean to diagnosed there's that of thousands we Therefore, as and patients of the are highly promotional market expecting disease, any voice in

clearer. where And nuances X-player the grow conversations the see of to -- up, is lot shaping respect products can we becoming results, how this from QX in our are very the rapidly. the market a I to U.S., believe the you are In continue a as now

That quarterly a you As of that good know, durably protein product the knocks rapidly AMVUTTRA differentiator. and with a profile injectables. becoming one the disease-causing good is is sustained down

QX, patients that momentum on get very and and we're results the we've not they the drive the the in reimbursement stay reimbursement. we expect to last And such. of least, how with as pleased how And on but seen

Operator

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

Huidong Wang

another AMVUTTRA. ask question I will regarding also

patient Given And of you and -- the patient from seen pattern any coverage, patient this on ATTR Part the design? regarding geographic polyneuropathy trial latest terms TTRscXX, the on in regarding population or population the sorry, any B III pattern Phase is a have quickly locations. thoughts

Yvonne Greenstreet

durable to Yes. improved We've knockdown looking we're TTRscXX is build X very And question. a the Maybe program an knockdown, for a clearly, knockdown. with or rapid seen months I I'll in dosing. that this start with franchise mean has annual potentially Alnylam. at really TTR durable opportunity

on take comment in do It's come about will the we excited to the this Tolga, specifics to of be of as TTRscXX. end So get still moving at but to forward there clinical the on... we're want really, program early question the you this time, really to more year. point

Tolga Tanguler

Reimbursement?

Yvonne Greenstreet

On reimbursement, yes.

Tolga Tanguler

Yes.

in years. So I philosophy the to burden keep is what we Alnylam co-pay established strictly thing is the think adhere on really patient has to is over that, the access look I at really patients. one mean that we the through mind And

reimbursed up to it's seen the B, of Part Given This is what puts important be pay the co-pay than remain in product us really with less redesign. in is an Part burden because through nearly less where that to would patients similar $X,XXX. position physician-administered XX% either D X that patients co-pay we've is or products our XX% our new those of have -- quite product and is -- a IRA reimbursed

polyneuropathy across when in So comes market. we've we believe the way global, this are where And price-sensitive market XX% the is of a share over a Europe, driving in a versus reimbursement. able compete in stabilizer mix Japan in we that's good markets about competitive to product very very it been to

we in get like sure we physicians it treatment make obviously, keeping so. that do of decide their if terms an this on And are where our patients policy. to really to Therefore, on eye we're reimbursement

Operator

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

Ritu Baral

Tolga are been Can the of far it the of commercial scale-up in sounds and are strategies. wanted of how you now force? sales for positioning a positioning you've just sort I you that? mix magnitude to like CM about the TTR you the actual of sales Jeff AMVUTTRA force. targeting And changing while ask as as a talk to of mentioned

are How New to driven they York going Card have in it then of mortality marketing study. your do are the way? is And real commercially endpoints despite be factor a is message? all expect this like data How that be calls like KCCQ, important are to our going to Or Association, they the sensitive label they? your wealth calls, those doc language, that are into going our mentioned doc How in market. things

Yvonne Greenstreet

question. Tolga, straight terrific A to you.

Tolga Tanguler

I being thing it organization there. make Sure. our this disease want clear. multidisciplinary is we're is to strong with I and start mean centers treated foundation just Obviously, very where by a to where one off going

polyneuropathy I want label adhere our we make will comes to expansion. we are indication, and But to promoting when strictly it receive until, clear it the obviously, very that we positioning, that to

to want just I clear. make that So

we again, TTR highlighted, We've great been advantages Now prescriber as of able you is I one centers. with think, polyneuropathy experience our deep our have expand XX%. to base by

We have a team. well-integrated customer

that magnitude prescribers actually we prescriber it's not disease this the obviously is of Therefore, a base. in tenfold. Now fact way. lesser this we believe tenfold. up scale number of appropriate of that, we can the highlight Having prevalence the the said

-- integrated to customer-facing support and diseases, organization it when key. field have rare you where of support, coverage you comes to good have of team going be good is then medical in coverage reimbursement having And an

have that. We

And pleased the into to start where strong is from think, piece, reimbursed getting with puzzle patient of the last on patient established time we actually support treatment a very therapy. quite the good, patients services, forms and I starting are

obviously scale where are and we're that capabilities the going those to eye up. Now an we're on, keeping

comes it be labeling, have. obviously, we're discussions going to to when where product, of to depending comment of the how question, part your we given label and Now that FDA the the terms we your going positioning on in that's don't be second

differentiation you I well think this and as color data ESC to as we going our going composite believe endpoints the all-cause give secondary communicate there's a products. is the clear actually how further primary about what at for be of We both effectively, and can to to able see you mortality. robustness be will

be of the is -- as but disease be down last convenience mean quarterly is that to going given a not The really that this important. pathogen possible to knockdown product as a swiftly quickly progressing, disease-causing is demonstrate knocks rapid I itself. And to reversible to least the be able profile going the important. is dose

quickly on going access this very treatment critical. to Given to actually that be is how get patients

profile, approved product the physicians the data do So data. where what choose will AMVUTTRA strongly that combining and believe is, HELIOS-B the is if given with really where the have we have we and product with all we disease this the as given a given product, first-line

Yvonne Greenstreet

Thanks, Tolga.

Operator

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

Tommie Reerink

This Tommie Salveen. for results. on is the on Congrats

be your question of considered number a on or Just Have for heard you cardiologists. would range top with a benefit discussion KOLs and the meaningful on [indiscernible]? [indiscernible] additive that

commercial questions. on And follow-up just a the

the longer the taking here what's growing top share Over term, market those [indiscernible]? beyond strategy your for on or of adding

Pushkal Garg

Yes.

in benefit take can top part of I of of tafamidis. question there terms maybe some the So in terms is your on numerical target of first

gotten first say what to I has all and that the help line would feedback is enthusiasm foremost. I new patients, these and the only top been accentuated along results is KOLs I really, for shared think we've we've think since the with medicine a

drug the is of actually across as what top spectrum of monotherapy they're this access from all to strong opportunity We this all, a disease. with patients Second an results line seeing for efficacy the broad endpoints. saw

inhibitors efficacy diuretic We patients you in equally and who [indiscernible] intensification. which background [indiscernible] add good includes on an of XX% I'll saw overall SGLTX strong population, a actually people remind of on had who start the and proportion added

population, just pretty the results, somewhat population. as magnitude as the in some in I if effect a p-value the which have of so well terms -- are in the top is think And of as strength at there the effect you data actual of look numbers, stronger, -- mortality the substantial overall implies both and line from noticed add-on the the

that powered what it is efficacy, encouraging. I an seeing consistent show is in Now wasn't But or to population. because we're of think, to that's evidence see designed encouraging additive effect

to So effective numerical is picture quite to think of I target, them people for are this be -- quite disease. that there's what emerging a a no seeing drug but that's looks

Yvonne Greenstreet

of We've can exclusivity we on. if loss exclusivity post-tax of the Tolga, market think, about you make think about words comment a how I and few a the about market. talked pretax loss

Tolga Tanguler

mean we this to is given used a combination therapy for believe, the I that some disease, access unusual of be it's challenges. rare given Yes. a

data we prior there's going think the we However, use early to believe I believe combination some to set, given exclusivity. on the be

years, point, is for that growth. to your the category the is But there's access to What diagnosed get patients X over easy tools the what's have diagnostic to advanced given -- last really early. we've important seen

is many promotional rates. awareness patients actually share voice rates. And and deserve diagnosis treated accelerate category having done The increased job this help. of only expanding treatment this And is category the going a of to we options in treatment with believe added to to. the has of to going voices available I be think only phenomenal those

Yvonne Greenstreet

Jeff, anything you want to add?

Jeffrey Poulton

Nothing further to no. add,

Yvonne Greenstreet

you. Thank Great.

Operator

Mike from with Stanley. Morgan question next Ulz our take We'll

Michael Ulz

just go XX% some terms bit sounds the then are patients factors? in as market the other but questions diagnosis well. what key prior of reduce follow-up the TTR And some talk on CM. a how you why opportunity a the little of just factors, like of is currently? you Can for about Congrats untreated It on quarter Maybe of going that probably do forward? one quick

Yvonne Greenstreet

for I you. another Tolga, think one

Tolga Tanguler

it's late. is Yes. disease, these don't start sometimes Look, a this especially symptoms until patients cardiomyopathy, too wild-type having

the key drivers. So one that's of

say, the as than XXX,XXX being we of expanding more thing a the is disease and few only rare more is second centers as picked start centers. U.S., What authorities disease. up tenfold manifests still actually we've there how multidiscipline well -- The multidisciplinary the patients. less are those the seen itself awareness a it's of polyneuropathy, much by In is it's as it's

see Therefore, disease it or neurologists even some seeing, is education, used not or doctors stenosis. cardiologists syndrome It's what carpal as where common orthopedic to in spinal more area tunnel cases, an more surgeons bilateral they needs as or awareness. where

having believe and the treated be going disease we help to And early how key. voices patients to manifests articulating get more is itself

get a electronic records, of centers be things with others on will engage well to start flagged doing is to One medical of we're as lot the early. sure patients doing make I and believe that

these have Pushkal, anything? you to think is making to industry the get don't I know, are the some initiatives do make going those I that sure of be flagged. quickly patients

Pushkal Garg

think therapy mean specific, Yes. is I [indiscernible] would until ago, years all And just No, that what well. it treated Tolga, I X ejection you reminder these said add I for that as was no patients fraction. was very there were sort of at right? so they a just

few diagnostic on these that. syndrome, or last patients. And these things therapies based the identify -- flag more spinal a such patients. proliferation stenosis over We've now, carpal understand we're years, tunnel these starting polyneuropathy Tolga's have noninvasive point, therapy, methods how had just now as like hopefully identify I the of to think to red soon to or for where to we symptoms patients multiple

for otherwise progressive just hopefully, heavy there actually multiple fatal and that progression disease. So more have education patients [indiscernible] disease need because is a stem the we believe, I of for can these now awareness this we heavy, think therapies

treat. patients I So in lot terms to of here of growth a think is there

Operator

We'll next Bernstein. question take with our from William Pickering

William Pickering

Yes. Congrats results. on the great

extent vutri that ESC, be investor placebo the plus background interest [indiscernible] [indiscernible] seems is about looking the there you at just comparing to limitations of with to comparison a Could you the versus and which on arm. think a talk Looking to arm lot to placebo monotherapy [indiscernible]? in ahead helpful any the relative

Pushkal Garg

are patients I we diagnosed is right? think was here Will. the VSB This really study are a modern patients what with XXX noninvasive Look, in era. Thanks, who study, means. [ ] rich These Yes. with very, done have a very

a monotherapy already we've was the another effective of As arm are proportion patients therapy. test. it of an we'll ESC, are XX% effective midst rigorous was good a the the study, share started SGLTXs, talked started, patients about things. pretty therapy, other In These on more and at

encouraging. study with It very for So thing the study very, are The was and it the one a not was believe a it was head-to-head that designed pretty rigorous was not. was tafamidis. not thing test, the results That's one we purpose.

comparison those so U.S. And the they've drug patients. while. of on ] you're that patients imagine, TAP Obviously, as from is the comparison. for the nonrandomized different And in really because who can majority the a on were a geographic [ [ came were differences TAP entered ] the been referring in The patients you there to

who analysis. comparing a monotherapy, then So patients it's unfortunately are to the on that [indiscernible] really flawed

that results across both top top have in of results the that endpoints a I think of line and efficacy of [indiscernible] series appears on and we to we see, is parameter a evidence tafamidis whole encouraging every on what efficacy we'll measured, as really looked consistent we at. as highlighted additive monotherapy

Operator

Minter Myles our take next Blair. William from question with We'll

Myles Minter

well. commentary vutrisiran with in the Just XX? September that know relevance I [indiscernible] in And be there's to public releasing you you hearing to tafamidis, to going something was are presenting that pricing, that also on TCICM? in draft future some Is effectiveness at TCICM Alnylam in are, about on be guidance cost as intending but or if

Tolga Tanguler

Thank Yes. for that question. you

and As, HELIOS-B any I assessment We results, a our and with ISE therefore, think, authority, top health to analysis. it noted we'll of prior and engaged disclosure they to made line this in remain was outcomes engage complete others. continue with

disclosed part we haven't the hearing. would of be We whether

Operator

from Luca RBC come will Issi question last That with Capital.

Luca Issi

for royalty can or which another away to PK/PD on back think with the to we is Gena's Congrats smaller run trial? here, Pushkal, maybe HELIOS-B need Sanofi basically III, a super as on all the progress. get obviously you question TTRscXX, important Maybe you of the circling do on Phase

went you're to going dose the pursue? related, that And the what's of to I way XXX milligrams. all maybe you trial, the the portion think

if So is the dose. that go-forward wondering just

Yvonne Greenstreet

details We're what because as to steps said, the clinical into not our to plans. we're come get going Okay. in ready question that to detailed I really the of next back

later. But of the more program. that thanks Luca. excited So your for We're also about share we'll come interest. and very Thanks, back

Okay.

we Alnylam's really, I marked journey. inflection think joining. that an in And brings really I the I progress. proud our everybody to are has mean, our call just second for quarter to close. want a of thank of point XXXX And

on commercial in company. top-tier we gone growth. a path believe advancements are our to biotech some that of firmly more the Pushkal incredible becoming the a described And has pipeline. through robust than we now Tolga's ever,

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

Operator

does Thank you your This the participation. conclude for you. program. Thank

You at may disconnect any time.

Alnylam Pharmaceuticals (ALNY) 1 Aug 242024 Q2 Earnings call transcript