ALNY - Earnings call transcript Q1 2024 | Alnylam Pharmaceuticals, Inc.

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Operator

Good day and thank you for standing by. Welcome to the Alnyam Pharmaceuticals Quarter 1 2024 Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded. And I would now like 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, Hilton, 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

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

may those this Act harbor constitute for forward-looking factors, Actual materially Private a in under 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

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

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

Yvonne Greenstreet

up wholly in growth robust quarter, an year achieving compared revenue Christine, year-over-year revenue delivered for today. and a XXXX. or the million in strong XXXX medicines, be call thank XX% product our growth is impactful for shaping to everyone, Commercially, very we the $XXX X joining Alnylam. QX to Thanks, off owned start for first and you, to

Phase zilebesiran the XX% XXXX. hypertension year-over-year certain a pipeline from franchise, growth continued our An in this our results perspective, From antihypertensives zilebesiran major combination with presented study, a care important momentum of the KARDIA-X. highlight TTR QX the evaluating versus part with and positive was initiation II KARDIA-X program of of was which delivered standard from

hypertension, This to our top cardiomyopathy. HELIOS-B treating program of on that reporting the Phase end, position treatment the as nylon cardiovascular patients of represents amyloidosis a the in to with cusp leader study disease. reimagine from and TTR with the line opportunity To significant III vutrisiran in we're results of

study to confident early from As by or a we've data highlighted reasons report previously, and in an HELIOS-B the we support top remain track positive filing on encouraging the this end of many the the to have year. of APOLLO-B in be positive, if June highly is of data expected outcome, late vutrisiran, including sNDA we HELIOS-B which, from line July, to

engine, all our to best-in-class prevalent With and/or pipeline commercializing this and patients organic by PX developing while and Alnylam driven biotech high-yielding Alnylam rare from XX around progress, product continue transformative our exceptional of for candidates results. world we with goals, top-tier the a product first making advance by financial diseases delivering a medicines

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

Tolga Tanguler

franchises. with combined strong morning, continue good net our another portfolio and Yvonne, was $XXX rare delivering revenues our quarter and momentum growth for QX our commercial Thanks, everyone. we million as product TTR

prior steadily we our to therapies. grew first quarter versus continue increase portfolio patients as in XX% year the on number of overall Our by the

First, TTR quarter results. summarize let first me our

the 'XX. net million and $XXX achieved product first of growth revenues, increase with representing The in a compared TTR X% XX% of the with fourth global franchise quarter compared quarter

continued and ONPATTRO strong and year-over-year, fourth X% a robust with of 'XX, quarter combined sales increased by XX% compared TTR by of U.S. driven Our AMVUTTRA AMVUTTRA the uptake.

by uptake, driven driven the than AMVUTTRA primarily the patient year-over-year in U.S. AMVUTTRA. the ongoing offsetting ONPATTRO which first by 'XX, XX% following: was of demand was increase more patients quarter strength a switched to The versus the of total growth in that on decrease

reported quarter, dynamics both ONPATTRO the robust channel decreased as inventory growth demand. sign approximately AMVUTTRA by favorable decreased in another and in QX X% Inventory of the

Now international year last and was performance turn year-over-year. fourth demand in increased patient and Italy AMVUTTRA continued Sweden, including strong primarily for and not our few as increased compared the by yet end of launches adds to driven 'XX by available. quarter a year-over-year markets with franchise new at TTR remain in markets, robust, AMVUTTRA let growth X% me where XX% is growth the The of ONPATTRO Spain, where

franchise reminder, X-month a XXXX, like would offset of partially growth provide also in the continued U.S. of pricing all previously for initial in period reimbursed certain in the was free occurred to in international in AMVUTTRA color TTR AMVUTTRA growth Germany international momentum is QX I in by markets at Demand the end major primarily now reported. As pricing the as on our countries, lower markets. additional

expanding by remaining. AMVUTTRA confident year-over-year significant our and robust remain the unmet with seeing the impact the with We TTR XX% very a is growing category growth we of that franchise This first for in in opportunity and needs we're the XXXX. achieved pleased from quarter is reflected

patients care market with treatment remain health Importantly, demand both providers leading the amyloidosis hATTR patients aligned market and growth leadership, our with AMVUTTRA's of with for galvanizing polyneuropathy. in indicators

and believe the than prescriber some highlights. growth are AMVUTTRA our between year-over-year by condition more engagement in More strongly patients. are that XX% We care requires key high Here is a physicians base. evidenced hATTR prescribing professionals their health

engagement to confirmed having the home. access access out-of-pocket access to seamless In costs. offers our center this at patients we continue philosophy, with outpatient patients patients of than AMVUTTRA XX% flexibility demonstrate many alignment happen more with and or having an as for hospital X patient our of to XX% AMVUTTRA at approximately the for

regimen. therapy remain AMVUTTRA's comply which patients dosing compliance XX% we are Last, with monitoring on metrics, and and quarterly than that more our show of

XX,XXX hATTR patients. polyneuropathy with and patients As we undiagnosed to globally of have or XX,XXX find approximately are opportunity significant a which previously represents XX% communicated, we untreated, treat the believe to more

and a most from also speed, we AMVUTTRA disease, and to physicians that quarterly progressing profile. believe knocks has a the benefit patients Given polyneuropathy offers single manifestations safety duration. rapidly down benefits the dose a depth rapidly unparalleled with hRTTR to track that access and is protein ability these a with therapy stand disease-causing favorable the polyneuropathy reverse and in combined well-established hATTR of amyloidosis, record

disease a knockdown AMVUTTRA strength in are and we its campaign the benefits on branded foundation, embarked raise the this patient to of as we of a position patient and of awareness rapid profile. unique awareness With

rare performance of OXLUMO. and and the GIVLAARI our Shifting franchise to

of Our in revenue and global impressive representing X% rare million quarter, franchise $XXX the compared net delivered a with solid global growth XX% product with an fourth first first quarter combined during compared quarter the XXXX. and XXXX increase of the

same additional increase the in increased deduction gross in primarily QX driven For to upside XX% year. Rest an growth was compared to favorable to year-over-year. a the regional by new U.S., therapy period growth, offset with gross last patients from the with XX% net by by net in XX% of dynamics, partially by which following in in driven on growth increase GIVLAARI, changes revenues modest primarily demand the World,

with by the U.S. deductions, XX% increased year-over-year, from demand in net driven gross the robust markets additional of partner from XX% demand, increase lower regional orders delivered driven in increase timing favorable gross growth adjustments robust a driven to rest net to world a and of by For OXLUMO, primarily by a was which favorability growth in revenues following the dynamics, we XX% a markets.

we nature the net in QX. magnitude timing see benefits, and Given Rest a to of World of will QX the that global reduction for in anticipate gross we partner sales market and OXLUMO

continued to delivering well it robust growth In to I versus now Pushkal? prior revenue that our we Pushkal positions that, net revenue and XX% conclusion, reach guidance. growth very year with will are pleased pipeline recent turn us XXXX over R&D review our progress. to With a product momentum, our

Pushkal Garg

Tolga. And morning, everyone. Thanks, good

await patients Let's the and with to amyloidosis outcome with to our TTR study cardiomyopathy. ATTR HELIOS-B, hereditary AMVUTTRA, include for franchise, the wild-type we from line begin results eagerly top of treatment designed our expand label with where

aware, and from study HELIOS-B optimize to competitive the earnings informed you're a field. in were strong As enhancements we to the data announced and data success support plan the changes the February, APOLLO-B by insights our best QX These call on to from label. for in statistical emerging

remain now well where monotherapy effect drug's optimizations, impact. but population, we have outcomes the are which of is With on plan to the critical stakeholders, the focused population treatment death overall hospitalization, best which these true these demonstrate the AMVUTTRA as will and largest and as to will believe we all outcomes in evaluate critical

benefits its we that focus highlight also profile disease. those best streamlined and secondary differentiated structure progressive stabilization measures We to AMVUTTRA's on the believe will of this potentially endpoint clinical on

by line powering or additional the longest tail months track incorporating ATTR-CM. firmly of on And We to end June in up report the most overall the placebo-controlled the at we study the results remain as date enhanced event establishing study conducted statistical an study and critical period, in period X to HELIOS-B early collection the of of late to July. top

secondary we as details p-values well time, the plan primary key and as that regarding on endpoints to safety. At provide

on presented Full also tafamidis. some scientific congress patients at are expect be We subgroups, to a expected information including to thereafter. high-level provide baseline results on soon

to HELIOS-B, from in a the And NDA XXXX. late we results submit assuming positive to expect FDA supplemental

cause RNAi investigational being evaluated Hypertension world. made a and as on mortality progress first some program. global around health our this Turning now for leading crisis the addressable quarter We therapeutic exciting morbidity to hypertension. a the in zilebesiran, and treatment very the cardiovascular of is

risk, to Unfortunately, of that of beyond in pressure have disease despite blood for there poor number other lack available nighttime cardiovascular namely contribute of dipping. of medication are uncontrolled to and management control, increased hypertension XX% aspects variability therapies, up and adherence, patients a

weeks Phase of these positive We At study dose care to indapamide, efficacy thiazide providing receptor an control like improve the outcomes to anti-hypertensives, a single ACC conference angiotensin few of full or diuretic, evaluates zilebesiran and believe zilebesiran of the pressure. a presented unmet and blood by potential standard safety the tonic results II all amlodipine calcium needs of of the ago, that the we KARDIA-X or from of added a channel address cardiovascular has a when subcutaneous that blocker, X losartan. X blocker

up of of primary pressure month at in when background X reductions to XX-hour significant X measured to study was clinically mean statistically blood ambulatory XX.X medications. monitoring endpoint, added achieved its placebo-adjusted pressure by X systolic mercury The as zilebesiran the of millimeters and blood demonstrating

in The study across blood lib end ad demonstrating reductions also achieved a systolic office pressure X point key clinically independent at cohorts. all secondary X significant

safety added and when Finally, an tolerability and to profile zilebesiran hypertensives. encouraging demonstrated care of standard

an of these good showing top efficacy which in excited agents X orthogonal continued pathway, arm results, of also by very works additive which and RAS top are mechanisms support and We the on tolerability development. with on

To initiated X point, that cardiovascular top high study, recently which or zilebesiran are who agents we Phase patients II more of will at the in risk. on evaluate KARDIA-X

where As shows, of treatment as cardiovascular at This risk cardiovascular by showing and this KARDIA-X slide partners control can tonic to outcome cardiovascular that pressure a after plans benefits Roche have to the mortality. robust with intention can trial, in we reductions an the agent our in zilebesiran of forward and reshape blood CV we disease. includes patients morbidity run bring demonstrate our high

quarter extrahepatic efforts in the to continue well. the progress with up this pipeline, our CNS Wrapping as

known I announced study to disease track study to remain a initiate multiple dose year. QX call, earnings ALN-APP of angiopathy on received we in FDA clearance onset early amyloid formerly Phase of As portion our the cerebral in initiate mivelsiran, early this II Phase Alzheimer's and we the as on

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

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

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

Jeffrey Poulton

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

Tolga primarily Total were demand, previously TTR growth highlighted. million with driven product the quarter $XXX respectively, XXXX, of quarter strong as by growth of XX% versus revenues for first and franchises both the XX%, reporting XX% rare strong our and or

collaboration Net quarter was to XXX% XXXX. a The and was million due under with an our with revenue $XXX from initiation collaborations revenue for of the first revenue recognized million $XX primarily license under to agreement compared zilebesiran increase clinical Regeneron. associated KARDIA-X quarter agreement revenue trial or and the collaboration with of of Roche, including milestone our in the recognized increase increase

Royalty higher a with margin to or the period revenue in LEQVIO was XX% sales for to by the increase product on million The same quarter quarter XXXX. quarter of of was the XX% the for $XX first driven increase which compared Gross was XXXX. XXXX. sales was consistent first quarter, the compared

royalties continues gross We lower be product at by on our margin as brisk of driven on for AMVUTTRA will a pace. expect XXXX balance growth higher sales the paid AMVUTTRA,

HELIOS-B driven compared pipeline. investments our same primarily by to non-GAAP and XX% our in Our and expenses R&D increased quarter the first zilebesiran, XXXX the trial in preclinical period increased in

in Our same as to in in journey compared growth than we quarter period increased continue leverage the to first XXXX, operating non-GAAP our SG&A achieving product profitability. XX% on lower the our to XX% deliver expenses sales

SG&A for a drive patient polyneuropathy of finding primarily efforts was launch in investment year. growth to preparation marketing TTR source as well potential investments related increased cardiomyopathy The in help to as next expense

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

line of with billion $X.X cash, end ended we of XXXX. reported and in the $X.X securities cash Finally, billion with the quarter we at equivalents marketable the

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

financial Now for to turn I'd like to guidance our XXXX.

between a growth call as We presented rates. Today, our guidance anticipate we be billion billion, to our in during X at $X.X XX% products are XX% earnings will to commercial reiterating XXXX net owned January and $X.X our wholly combined for product FX February. rate revenues XX corresponding

year, giving in strong our to QX ability guidance. a the sales meet start our was or exceed to us confidence product

to changes carefully We warranted. determine QX if to course, of will, review in are progress our our guidance any

Our million revenue $XXX guidance royalty to million. collaboration is and $XXX range

billion of And $X.XXX R&D remains X% a range lastly, for between guidance and expenses and midpoint with billion, reflects non-GAAP SG&A our growth which $X.XXX XXXX. combined compared the

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

patients carcinoma. initiations study in II study angiopathy, trial ALN in I including of in ALN We and XXXX, the a a amyloid B diabetes expect X type Phase KHK of of Phase for early Part hepatocellular study in with cerebral X I Phase DCAT

track report And June has vutrisiran of in late we on early from been highlighted, remain July. top results study HELIOS-B or line to the

quiet in how XX advance is, plan beginning those the Given of May period to readout enter a XX, May important results. we

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

Christine Lindenboom

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

Operator

of Our David the Instructions] line from comes question Lebowitz Citi. [Operator first with

David Lebowitz

improvement versus easier certainly improvement And on be drug release, over on one XX% that just monotherapy curious, make to is I And relevant that the how you the think it? it side but there curious, relative to talk control information another. in top been know I'm won't do versus are compare to XX% thoughts about the improvement. the think your come about reason I'm should we numbers line what relative discussion? what have about has some considered arm

Yvonne Greenstreet

question. the for Dave, Thanks,

with the to that. out to the of this reminder, important some results. aspects plan, And importance best this consider detail a statistical payers stabilization we deliver this in in differentiation think we it's that's on call, see we other results will in if happy to the some and we're is changes to rationale medicine. Clearly, to of as some we at point in our physicians be to be we're got show made expect shared study. And look we last Just clear which HELIOS-B able regulators, outcome also to we on from outcomes, Helios-B discussions an of time, a behind I benefit that really at quite that is if But study. reprise with And what study the an and disease. need the respect the way will outcomes the delivering

I these that think contribution will believe the this, Pushkal, an all to if is there we managing be differentiated so will And disease deliver patients. profile have you want to that important we're to a able of anything add?

Pushkal Garg

as change is disease. Yes. everything where continue it and when I you couple to need it's to steadily unmet disease, a maybe is mean month-on-month points. back such mean, I the important clinical go of agree this considered to I with significant. decline. any progressive said, again outcomes Dave, in other again, to we Yvonne. clinically hospitalization, just This a death And patients comes think

worsening patients unfortunately And a of hospitalizations, life, physical here. trial or experienced once-a-day of to quality this decline. that class medicines unfortunately, to patients disease. data worsening clinical continue function, could helpful succumb whether And and you're stabilizer ultimately, on the that suggest They be orthogonal stabilizer twice-a-day the

we're by data. to looking demonstrate clinicians well magnitude separation really as as total We're So be occurs effect the we outcomes. the important, looking stabilization, will whether evidence APOLLO at the at disease of that which well as encouraged is as B of looking data. there's think And when

report results we're early late to and in June looking forward July. the So

Operator

Matteis next question comes with Stifel. Our from Paul line the of

Paul Matteis

works cardiomyopathy organizationally to it up fails. I of the you as to word wanted changing Alnylam ramping what spending Jeff, may X in guidance ahead infrastructure do just a if about ask expect look to HELIOS-B launch? succeeds, relates scenario versus like HELIOS-B be and

the you that? Or if XX, is you have And think to end doesn't pipeline? sheet about are still you Alnylam R&D stands? include XXXX as balance INDs partner X execute upon going guys HELIOS-B that have to to like work talk within you then you going do still programs, or prioritize feel Do of conversely, the to by the

Jeffrey Poulton

the for thanks Paul, question. Yes.

me was and guidance The I need side to results. this have whether HELIOS-B year start first Let our or need around guidance result? a the positive which no. for change with year we one, positive the that if with on is the would we HELIOS-B the reflects answer we think The not what think spending

anticipate to be opportunity plenty opportunity the We the raising that guidance. we So I of don't have we to behind invest success. think drive would

we scenario around think unlikely were the if the HELIOS-B. I questions evolve things how in were other might failed

Certainly, at doing that on we're Again, the work outcome. the would across need talk And prioritization look If that's that. about we business be think would the scenario. in to make in that but we prioritization decisions prepared the market in we scenario. would we to we're that that unlikely, to

Yvonne Greenstreet

think reflect in of just also the have we number currently We're company. I it's as of to we by of XX that all, pipeline, doubling at that magnitude mean, the I our important of the a front programs XXXX. looking opportunity first touched clinic. us on richness in on

So at in as we look of about have. more excited that be we we being set move couldn't us able out in programs the forward front the to [indiscernible] opportunity

a So outcome from looking of and us. drive forward positive front that's we're HELIOS-B obviously the to in continuing pipeline then really to

Operator

comes Maury question from Jefferies. next Our with Raycroft

Maurice Raycroft

are added of HELIOS-B bit for about last the to you time your few studies. should can get the and I similar time the And mentioned ATTRIBUTE that a And for we plan? more was expect critical of talk studies, stats study? HELIOS-B, you that the few trajectory that events steeper arm the in And the KM those what slope widen months little placebo ATTRACT curve would last that, for during for that's the it the a both remarks the expectations prepared a In events think additional for months of in delta? in the

Yvonne Greenstreet

a as Yes, a I at of you of that's question. where study, patients a continue in clearly, And critical think say, is this study disease progress. the to end great period the

want expect add? else see do that, So to period events. anything Pushkal, where we we'll most you that's a

Pushkal Garg

add, think Yvonne, it. nothing you covered there's to I

Operator

next our James. comes And with question Nachman Gary Raymond from

Gary Nachman

as combo still enough positive are you reconsider continues data be would CN? launch over And tafamidis as prepare now just of care what in ATTR-CM, monotherapy as thinking for in to Just a are to and details in of some enough tree grow with there thinking? ATTR-RCM standard the drug of show alone, TAF if primarily blue TAF helpful. would a you the benefit you that are doing So

Yvonne Greenstreet

Yes.

questions. Some really good

cardiomyopathy I into excited this getting you the want talk maybe looking at playing the very mean launch. to and in of about to growing we're how rapidly opportunity how preparing Tolga? Tolga, win category. about So very we're market this clearly, for we're We're space.

Tolga Tanguler

And be patients to -- XX% disease category. fundamentals is progressing as thank of frankly, And of the deal We're the look to look, at day, patients you. There quickly of really rapidly looking exactly are at right. the as physicians Yes, here That's patients need XX win. remain the the irreversible to play are a this undiagnosed. with to end This no, both disease at able possible. quickly and and with damages. we

differentiator. looking causing and a the for. we down look toxic depth first key speed, to if end day, the physicians pharmacodynamics knocking and we upstream at the impact protein you and of what be AMVUTTRA That know At is early AMVUTTRA are really going this offer of provides protein. is disease the as rapidly So as to at and what that has dose, the the how duration the clear

it's see tafamidis and care. true, the care is it's in standard but only is not to great of this category. making just It progress, the great It's

product I is going remember be U.S. because reiterate And really and that in that. end demonstrate. positioned means to data access of on in mainly they've to the the think going remain again, need obviously, pending pressures, needs until XXXX, the X the the but fact to which we just the way the is LOE of also, So to this particular

patients be looking it's who able to for as agent. the an we actually alternative. in are of being be in going very important after be, to that first-line to number of the and case, are be there all, armamentarium patients we're are to treated difficult We believe that a physicians And going are this continuing And believe believe the option to Pushkal substantial indicated, disease. in going progress. we physicians And a

Yvonne Greenstreet

actually That's on. spot

So space it's of expanded a we of the program. matter within very instructive you time, then program, on I access max a on when reflect think just our where short out

is medical level that, the illustration as on based of just again, an progress looking unmet fact need the continue think I to . is alternative and on for patients tafamidis they're therapies. that

Operator

Our next question the Ahmad Securities. with BofA Tazeen of comes from line

Tazeen Ahmad

to Question. it XX tafamidis in potentially, study been right line. XX. with month going specifically, I all when the show benefit at covers what's the trying available drill understanding giving you've lot that top even think color now. end, important appreciate benefit know the mortality the going unmet at is it about that? to that there's expectations How in for could the the around to important month terms to need what kicks be on in So or that for have fully with into Pfizer be better of detail be Is to around a But mortality it of I started number than to a

Yvonne Greenstreet

for one that Pushkal you.

Pushkal Garg

the think Yes. call we both of separation, under mortality see to are the around separation. for when Thanks, that if tafamidis month is data XX seen and in acoramidis what Tazeen. start review is we've I mortality terms Look, I

month of we earlier, on we're of seem occurring again, to actually and see the in we by data, Look, saw roughly original mortality where X then or underpowered what APOLLO studies APOLLO-B separation coming evidence encouraged so. out the data

if So to those this we'll we're see study. larger-powered have results HELIOS-B recapitulate to in in able

important What I that Obviously, it's mortality what's about when that it what Again, going totality be the then to at in And disease X difference, stabilization. come happening is out. will look of again, hospitalizations. is data be emerging, of think to parameter. terms the all the

able But should all we we same data potentially going see which that is confidence of APOLLO-B, of population, that those seen from in effect lot the APOLLO important early So to parameters. be and patient substantial are we've us particularly earlier and a be gives look, separation. the a to think

Operator

Baral next line comes Our question the of Ritu from Cowen. with

Ritu Baral

give for the you line that subpopulation will I able thank potential you or as the us wanted you combined little give mentioned I to TAF you've details just well. to Pushkal subpopulation, -- of benefit further, want but more the some on I'm you when sort could. to if top composite us release, composite a given Yvonne, the drivers would that and on I be dig of of detail sorry, subpopulation

Just given very quick release. on previous conversation, a Will And sets? the sort drivers just then investor hospitalizations on tell focus get conversation major we a and the a data what of on are? driving data follow-up

released You drop proBNP ones parameters be were KOLs that those our if at first with We And deeply and echo deeply, to endpoints. medical exploratory presentation? actually those least would wondering are value.

Yvonne Greenstreet

mean, clearly, going kind we're be handling the Yes, Ritu, I endpoint going think we're I mean, yes, how about show values the key and to it's secondary a good question. And release. for of I we've primary clear p been quite endpoints. to

information in I we've think we tafamidis in at But stand on to important interested we particularly subgroup. was So consideration. that's with subgroups. said we're some this time. point will information that's where provide some the safety. going an And Obviously, to respect give

share obviously track being of data top kind We're We're the able early medical we'll proximal July. looking for then, to present course, still forward line at of results. congress. on And end June, a to fulsome of

tuned. stay So

providing be time. secondary at and endpoints. all color this above we But point additional for We in will say can primary the over and some that's probably p-values

question? second Your

Pushkal Garg

to fulsome certainly, And aspects you there BNP, X a can data. we we investigators I of data. and what think that various But in cetera, -- work on. really the that set ECHO, want like a haven't the line out number presentations mapped presentation. a about exactly Look, limited important we of the to make this, be be sure the can where will sure talking to the speak just ECHO [indiscernible] with we data what make be presentation. to we're et imagine top that will NT-proBNP there's terms Obviously, of with will the reporting that are it parameters

Yvonne Greenstreet

to mean also. able with Pushkal. but many have going we're those I confident demonstrate to interested size respect you we're pretty to for Absolutely, you points. kind share, X-minute end be wanted but progression, to to very clinically what really we're are to endpoints how managing be in prior with what be they're parameters, relevant we of endpoints But about those secondary clearly, clearly have secondary very those additional And discussed well able in the going as mortality KCCQ. that we'll prioritize endpoints, as respect and to up walk we to disease

Operator

Jessica JPMorgan. Fye question And with from our next is

Jessica Fye

talk any clinically impact you meaningful. when about HELIOS-B, on For being outcomes

X On are hand, a when ask or benefit. but I that's not usually completely you a magnitude this, again, any then mind there the in have they hear about physicians threshold just we outcomes,

I'm curious to or you that of reconcile could there? elaborate kind maybe how just So

Pushkal Garg

steadily patients a that untreated are leads -- a of patients I is and who Look, than remember again to who patients, have disease pass them I damage and I Jessica, outlined, what you there give unmet can this other say, currently have a to ultimately, that large Tolga progressive information don't many as has know of the lot more that Yes. undiagnosed think irreversible away. disease to need number in just are

setting which progressing highlighted, as was them Yvonne demonstrated the and of only in our drug, patients, as after outcomes the enrolled tafamidis, And in many rapidly IV were available so what on experience think evidenced we and I any of being them. therapy was to APOLLO-B, population that -- an a EAP by without benefit

the So highlights need. unmet it

of outcomes we've hopefully come So a unmet knocks when an positive that, think these down along showing the with an we protein talked address the factors and disease-causing we for mortality this other key orthogonal that think to that a mechanism going hospitalization that differentiating disease forward with that about patients. that's and benefit, we rapidly these HELIOS-B, needs

we're So speak let to themselves. forward and looking we'll that, the data for

Operator

Our comes BMO Markets. from next Capital question of line with Kostas the Biliouris

Konstantinos Biliouris

those hospitalization or look the Congrats How between of from different for and this think HELIOS-B X you the question compare types as on drugs? A is you on just at as important the ratio do events a important Do together metric, especially you composite? events at progress. take. an we just metric a ratio us when look as death

Yvonne Greenstreet

this question, one for the Kostas. for is Pushkal, Thanks you.

Pushkal Garg

it's think cuts. an No, I question, interesting Yes.

of we I were distinction I make like that was to reason a with I correlate tend composite events. here. because and look created don't hospitalizations them together, very think mortality strongly in this part we general, mean at think the endpoints huge

clinically so -- and meaningful both these And are outcomes.

And so of of they with diseases, we've what will throughout lots go with seen disease we've area the what drugs, same that's That's that the expect we seen different directionally would classes. lots drugs both direction. in disease cardiovascular and other

directions, think of I similar So we to a benefit, thing main in And Kostas. right is both the in hopefully, the direction. go in would those, seeing expect them the trend

Operator

UBS. And of comes with question line our Ellie Merle from next the

Eliana Merle

look proportion of to when term likely how And versus car defined payers? you being patients In doctors a larger stabilizers world? do do gain the think population of And this you is expansion? is ATTR, phenotype both more we this cardiomyopathy by real longer what mix to see the in by share silencers and

Yvonne Greenstreet

then Tolga and first Pushkal will up. think follow I

Pushkal Garg

I both of very distinct as same fact, misfolded, causing diseases, raised in oftentimes, disease. these think sort the classified we've When it's a very -- I and it's Yes. it's X polyneuropathy protein, and the important manifestations think when people of question Ellie, have cardiomyopathy. you

those I polyneuropathy for of those concomitant would patients we more that a patients significant in have that in of cardiomyopathy have conversely, half that had cardiomyopathy than the population, patients example, and -- HELIOS-A and done looked manifestations. in seen, hereditary studies that been proportion may suggest when And we've APOLLO

ATTR So patients class of with profile. magnitude silencer might certainly, that example, drugs, concomitant with wild-type and cardiopathy for to the from that have manifestation really a there which we've of seen AMVUTTRA, its ranging polyneuropathy VXXXI, of polyneuropathy. primary particularly with And in have of in the XX% clinical or are unsurpassed terms XX% or reports quite more is effect

disease this they And therapies make to have how probably when see, hopefully have will who multiple more so be of have will can available. patients when multiple and about comment interesting it this, manifestations decisions Tolga clinicians of classes they

Tolga Tanguler

manifestations is, doesn't cardiac if whether Yes. practice usually the at this it starts suspicion. of manifestation necessarily of polyneuropathy clinical X the the you diseases mean, you have the point suspicion treat No, just And and physicians on with start this I to how quick add of actually look or that disease, diagnose disease.

data looking AMVUTTRA effectively treated for on based the can are Eventually, manifestations is centers. the possible. physicians neuropathic they make both we disease as have, And this ONPATTRO absolutely that and to with treat multidisciplinary sure obviously, through as always

disease So go best treat end to a understand have based at the at CM, they patients best of the and and products. try the the with don't in on then indications multidisciplinary through PN possible or of how through they the the whether way the to phenotype, approach, the day, look physicians mixed disease just

Operator

the Barclays. line comes next Our Gena question Wang from of with

Huidong Wang

Sorry, ask I another will question.

be drugs? you and on in share primary will tafamidis scenario at in vitro So HELIOS-B that price positive, subgroup, end And information is to will lower secondary the the competitive compared you regarding say, top point, to, share the also some you both the point? will and line subgroup other also,

Yvonne Greenstreet

specific question. us just about I I'll talk of early mean the pricing too pricing take approaches I kind really it's for kind here. of to think

that hope a with sure able going do affordability and make Just medicine touched take Tolga we introductory patients are mean, around to on... from this I you, be benefit bear profile. to the question be principles, access access that robust will will have at patient and making we we be we'll considerations to in to and very obviously want to on Pushkal, our what you remark. his in mind sure remind a

Pushkal Garg

terms populations, has to that can of across to plan know don't in make X again give it's the I now in than around terms which primary add those then the plan. We endpoint, the our I That's and mean, more lot some the reiterated anything safety I Yes. hard in you that I populations, speak subgroups. commentary we'll a the and top secondaries line. there's Gena, to of But today. is that Yvonne more made remarks

Operator

the next from of Sachs. line Richter Goldman question Salveen with Our comes

Tommie Reerink

This Salveen. for is Tommie on

this analysis, the patients? regarding line given patients can and detailed in professors would to include in who see top and [indiscernible] unmet the data you beyond defined So need analysis just in how as on population benefit past AMVUTTRA were impact AMVUTTRA

Pushkal Garg

what about data cuts to is And set. you're and of data. think a certainly, is going be actually asking this I large Tommie, very deeper Yes. the rich

at looking have But So you and came again, we'll a we I pretty lot on fact can as different this imagine treated in patients in the would ways. who of in I thing that focus a both guess data will drug a primary the set tafamidis. be with on robust monotherapy is the that

have obviously that why with ask a in that clinical year drug time. on are that someone enroll moment yourself, would you or think I really -- And And satisfied not indicates feeling they're to who's they a X-plus functioning at to decide trial. will in how they

where so combination And be of address question. and a on our proportion group, patients, think that help have will sizable part of I do report we'll able we your to

Operator

from comes question Stanley. next Ulz Mike Our Morgan line of with the

Michael Ulz

just Maybe launch follow-up the preparation a on question.

that be curious from far you by sort steps of triggered a have the guys what in sort data Just what launch? And are then remaining HELIOS-B? positive so will for done potential of preparing

Yvonne Greenstreet

footprint to our and at am with with pass just to built. with That's the it I kind performance look I a think to that on great question. if I pleased of Tolga. great we've actually underscore And I'm patients AMVUTTRA to how you commercial And polyneuropathy. want respect going

demonstrating how of is meeting organization indication. I focusing needs on commercial I'm momentum strong growth here, just patients we're think really the this and with in really pleased the

no the the have into we'll about comments do being some to on specific launching. you I same able that how may have Tolga, indication. we're cardiomyopathy thinking launch HELIOS-B assuming doubt But and

one exciting is categories. of think I what So to be most the going actually

Tolga Tanguler

our little and competition, TTR the on despite of is we've the franchise the in momentum wind I that think U.S. quite to indicator an I bit because growth XX% it's You fact is which took was that year-over-year, away, important, going important a just built. highlight growth the of

Look, market we the polyneuropathy tafamidis in great now very indication. actually in in indication. data where share polyneuropathy. established compete in both U.S., we actually XX% promote cardiomyopathy which And our we're treat polyneuropathy, centers essentially and a do important have great obviously strictly stronghold that We demonstrated brand, Europe with Japan, clearly the in and same

by indicated, be product the as -- tend neurologists. assay obviously Yvonne outcome this win. as play by the -- to which positioned is is going diagnose now -- to as known who well to the disease going launch very we're it's cardiologists critical. to results, we're really the pending But both HELIOS-B well Therefore, to also And

we're be And organization, just trained in We that have opportunity customer-facing and I side. that of but a the to right differentiated we ourselves said going we are and opportunity tenfold affordable Therefore, going so are also side the this great more that the the appropriate have. adjustments the patient-facing clearly that currently is very and we also patients. to make and cognizant footprint accessible not importantly, is well-informed sure product to make a

Operator

question Ijem comes last of with our And from Whitney CG. the line

Whitney Ijem

in I'm that. catalysts just will question going there be after throw because X non-HELIOS-B there to

later So I ALN-APP to or expect of this should we us, what Part can imaging data? B remind for any help see guess, you the year, biomarker study, particularly around

Pushkal Garg

incredibly as the that precursor the amyloid APP. pretty profound Whitney, on and involved we're the as data year that for we about really are protein your well and on ABXX about XX shared It's impacts disease have protein Yes. in last excited that CAA. program question suggested of Thanks, that we both a downstream can Alzheimer's

ongoing, We hopefully have as regards as that's show we to imaging biomarker Part data safety knockdown the will data. and with B well and ongoing

lowering And that think off as leading really stroke, about with look -- major available hemorrhagic be to study will cause So that we in Phase beneficial APP could shortly. treatments be our disease of be also something these very second a initiating That's going for excited no soon. opportunity. the that for we're CAA kicking patients. We're II the

study off year. we've disease that Alzheimer's plan of we year-end this at And kick to around announced an also or

exciting programs really then move Regeneron it And then allows a program. forward CNS space. a us to other colleagues of is in at this the number So with our

there. your thanks lots of question, for excitement So

Yvonne Greenstreet

to executing robust you, forward progress pipeline our Pushkal, you, our remainder chemical. We exciting look and the the top-tier everyone, on goals, We're strong of of growth thank to call. proud biotech Thank Great. for us becoming and our XXXX. XXXX delivered we really on commercial a and path joining start

day. a have So thanks, and everyone, great

Operator

Thank today's now does you, This today's conclude and conference. you may participation call everyone, your in for disconnect. conference

Alnylam Pharmaceuticals (ALNY) 2 May 242024 Q1 Earnings call transcript