ALNY - Earnings call transcript Q4 2024 | Alnylam Pharmaceuticals, Inc.

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Operator

Good morning, ladies and gentlemen, and welcome to the Alnylam Pharmaceuticals Fourth Quarter Full Year 2024 Earnings Conference Call. [Operator Instructions] I would now like to turn the conference call over to the company. Please go ahead.

Christine Lindenboom

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

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

Alnylam's Pushkal will financials will will will the global you and provision provide and remarks today's Act as call, prospects, future introductory like call our open and for updates outlined of During pipeline followed questions. your upcoming review which on to context. review constitute on we forward-looking a that provide I Tolga purposes harbor clinical will remind call offer Litigation commercial Jeff milestones safe statements of the would update Securities Reform Slide of summary progress. XXXX. concerning and X, expectations, before Yvonne and by an general this the under progress, plans to remarks Private the contain guidance, our

those the may those by most results periodic our discussed from file factors, a indicated various materially differ as in these forward-looking including on reports recent SEC. result of Actual with statements important

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

Yvonne Greenstreet

and from study filings HELIOS-B for for for positive regulatory development ATTR research, Alnylam includes the was thank today. fantastic submission global and and significant year leading the XXXX results business. across program everyone, of highly you, This call strong in III the progress vutrisiran the Christine, cardiomyopathy. Phase to Thanks, commercial execution joining with a

$X.X in combined range our and end and strong net the significant we and financial full revised our $XX delivered of million operating continue advance billion achieving continue high-value expand . performance non-GAAP commercial for the over of a robust also upper We the demonstrate guidance at to company. to for marking of income pipeline milestone product and and we revenues year, clinical programs,

maintained which of enabler our a Furthermore, has been award-winning our we culture, critical success.

into for elements XXXX We chapter. the and for not a just of but come drive look ahead, we exciting X has years Alnylam. Alnylam to are to value and landmark core sustainable that next be business As that XXXX, year potential our its for there believe growth year will take in will creation,

the flagship our vutrisiran, is first ATTR for new potential leadership The the next-generation necrisiran, with in approved, along. assets, if TTR advancement a of standard company following franchise established cardiomyopathy ATTR and a where has care our to of become

numerous R&D pipeline creation. multibillion including with growth set innovation to Next sustainable deliver a engine opportunities innovation, an and is up through dollar and value

The financial performance, project pillar to going us to strong capital approach will robust profitability sustain enable is commercial that delivery third disciplined our forward. and allocation where we

by is culture. this a best-in-class our and underpinned award-winning of team All

-- Alnylam launched XX against fit All back progress this of by in tremendous XXXX. represents goals,

our half turning top-tier a on XXXX next to track we set make growth. biotech. on significant second Furthermore, reality and progress in the of the in the so, of up of decade, we're and era are us now We believe cusp become the for goals the doing into will and

the now believe Tolga commercial in a yet chapter has are and let performance. for new a best call of With that, Tolga. me truly history turn exciting our review to over We and our entering come. Alnylam's the

Tolga Tanguler

we delivered a $X.XXX our our compared combined XX% portfolio rare with fourth product translates On consistently of was which compared end to with in XXXX strong the a TTR XX% and rare QX upper for net as patients increase year XXXX revenue commercial another Thanks, quarter growth the range. to . of growth our guidance revenue billion to the basis, therapy franchises combined XXXX. both Yvonne, and good our continue franchises. at and product morning, we on TTR everyone. of in quarter delivering very full number

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

on Our to TTR quarter the by increase in first we robust quarter increase, U.S. during year revenues sales In momentum. year number quarter combined our AMVUTTRA strong during fourth compared a increase in driving franchise and the a our of as the with $XXX third global finished full fourth competent achieved the year-over-year growth million net the and hATTR-PN XX% continue XXXX increased hATTR-PN, XX% with of of representing of the with product the patients quarter ONPATTRO therapies. in XX% compared

XX% primarily the patient ongoing of AMVUTTRA growth by ONPATTRA. driven The year-over-year well a demand following: increase continued as in by from switches was strength the XX% driven as uptake

in which adjustments gross entering throughout dynamics consistent the in inventory the end competition favorable stocking in demand decreased year market in the growth new despite We remaining at XXXX. of QX. net as were deductions, The which quarter by the are modestly the to related was distribution pleased to growth channel in with the offset

$XXX position increase X years, full XXXX of generated performance is underpinned compared in where representing this the an market entered that the revenue, to U.S. is XXXX million year safety in efficacy and like the of over We which million take moment represents the knockdown we profile strength solid growth. commercial rapid with TTR of XXXX dollar the dollar leadership in year relative reflect I'd $XXX new despite $XXX the in market of at growth a to HATTR generated beginning almost to established million the and last PN growth the full in dollar growth The AMVUTTRA competition of in execution. our by the and we have and reflective year. believe we XX% on the

The to of XXXX demand XX% patient the international where XXXX. markets, by year-over-year in fourth increased our the driven U.S. to the uptake TTR as remained quarter XX% quarter turn growth grew year-over-year fourth compared me let was of quarter for increased primarily third franchise Similar by AMVUTTRA and with robust. the Now compared to the

discuss target I'd upcoming Before diseases, readiness for date a late our and the TTR-CM like action March. PDUFA our minutes our expanded take and review to rare in few to we launching potential label

regulatory a weeks pending bringing desperately successful We've revenue XXXX of that We're product excited opportunity, in year therapeutics been mechanism completion of of full action. only now U.S. population We preparing recently announced the of new a which product JPMorgan. for in potential away about the a to at patient is with be RNAi this need review. guidance may ongoing

that in includes guidance growth into is Our anticipated of mainly TTR driven reflects XXXX over absolute and $X.X ATTR-CM our our accession launch. and label by our versus excitement confidence the billion by about franchise

we've TTR at and billion. billion between from of XX% a guided $X.XXX with an reflecting for the the our business annual This represent guidance, midpoint TTR growth, sales an For XX% $X.X product XXXX. increase in in of XXXX acceleration compared XXXX, would revenue achieved our

a our Now to a and reasons sense for few what's let share you me of with come. optimism

a and are ATTR an whether orphan opportunity is help An patients is is market be more to we disease. This growth growing before, they will As entirely still It rapidly story. category have a in is largely a underserved. said The a treatment currently or this category therapy on an option. progression. experiencing context, potential to disruptive and disease diagnosed new for introduce stabilizer treatment. the right newly In we need,

disease, X and and source on approved of stabilizer Assuming first CTRP, quality critically of other TTR-CM patient background and of approval, capacity HELIOS-B, for and a a a on like working with both outcomes life TTR, of preservation that population XX% the that feel. today's of therapies silence and placebo reduction in important AMVUTTRA on patients RNAi all-cause versus impacts significant SGLTX impacts XX% will that upstream profound they functional demonstrated only consistent be and rapid CV inhibitors. study a to knockdown mortality and in the representative delivers in diuretics are can including Early number at

in X foundation and just uniquely patients. commercial track while from have execution, of demonstrated results. year building doses you consistent on as access for seamless hATTR-PN per full are still a All and a year is of we seen achieved there of work record front positioned strong this And our and much in still with for us, QX

important our category. approach on this the X our of for As we experience and are building launch, in durable years success we over this long-term franchise and building past success TTR

actively access time managed the access end, optimizing enable and This been means of create experience focusing health for at prescribe and optimal professionals systems, taking to to pathways XX% health at on have an will This Toward systems, or population infusion that health patients and access providers a the the experience. at where can are optimizing work care knowledge at optimizing alike. an already access patients location, treated and smooth health is are today. the center onboarding we given at office being so receive AMVUTTRA it patient XXX means pathways be patient that with at the these an it that we easily seamless systems home.

fully this months progress, the therapy result a groundwork the to expanded added in ramp-up of FDA it. indication, formulary In year for to following how We of parallel, and for also the broad that can sites product as adhere and optionality expand is alternative will in for take and for in can pathway progresses. the to initiate ecosystem been good demand to approval seamless to which several regard, to a note laying building it optimized, it the way the be access we've patients made a in be important care system's

Investor is clinics shared systems at care Day, home patients. group available practices addition currently XXXX and previously for of in over and As at we sites TTR This administration. are alternative our to X,XXX there health

XX we've field account all teams for than and key Now provider to our With these sites our within world-class patient regard already customer management miles up field a patients reimbursement, cross-functionally of XX% services award-winning scaled to and expand medical our of goal to sales, be and side. teams. readiness, ultimately includes greater treatment that is

post agreement and access. finalizing pivot Looking value-based via pioneer approved optimization, access industry once in several our will our establishing portfolio and their therapeutics to reviews Expanding ATTR-CM, ahead we enable broad label. an systems securing approval, an formulary committee pathway our again, we which value and pharmacy take in have to been VBAs deliver also to focus approval could we've across months patient health

We on expanded cover partnerships existing these expanding our are label. to ATTR focused VBA

disciplined see this driving approval. we following and our compliant tremendous Finally, In short, category. opportunity in execution teams across

engaged the and for we become a need. of to on We potential the have in product has for deeply And ATTR experienced a commercial transformational an -- what in cusp are regulatory the organization decision market. patients

plan guidance. exceed to provide of full share operational the to we of over relevant year achieve the course our our the year launch, or revenue details progress We on aim to clarity as

Now with XXXX. X% decline of $XXX our third purchase franchise to sales of the turning million XXXX, compared representing in XX% and quarter, the a driven patterns rare delivered with OXLUMO fourth product in combined which and the growth by fourth GIVLAARI of performance quarter and compared the quarter

increase GIVLAARI, QX, QX. increased For XX additional growth by with market growth recur X% by which quarter market the therapy offset the our fourth compared XXXX, a inventory adjustments driven GIVLAARI basis, in was highlights. large in demand U.S., in sales timing in third X% dynamics, X% compared of of A markets by stocking product European growth QX with gross the XX% quarter a by a to not orders markets. the by primarily international where patients the in following sales with decreased in of year-over-year in driven with did on partner our new our due regional On in the increase primarily partner

demand with the strong OXLUMO, QX therapy, quarter and strong we world compared regional our close in quarter the results demand end XX% at and and pipeline to by XXXX will Pushkal? that, TTR in primarily with competition, progress. adjustments year-over-year patient increased by the dynamics. In increased by rest our the of on and R&D net us continued now driven demand. pricing it which primarily growth in sales Pushkal to review revenue 'XX compared continued the due X% over product year dynamics, in range. of following the emerging growth the XX% I increase of driven product stocking recent conclusion, fourth by turn guidance with by to robust third with from with U.S., growth franchises, primarily to quarter by rare delivered driven markets, both additional XX% enabled solid to upper 'XX For our growth of patients With fourth growth growth gross favorable a Despite a driven strong inventory a

Pushkal Garg

good and morning, Tolga, Thanks, everyone.

As conferences today's throughout for in benefits potentially our XXXX these population a know, with the very exciting several assessments. ATTR-CM highlighted We HELIOS-B half profile Together, of series you shared comprehensive TTR at with data all of patients presentations landmark results transformational year. across the was the of year franchise. . a vutrisiran's from second

Japan. Based on review. date filings XX, are completed major and have all we've we a were compelling In that achieved these States, PDUFA in data, of under the regulatory regions, submissions United and several Europe March now including parallel

as new possible. quickly to goal this bring option the is and patients to therapeutic world as important Our around

highly beyond Nucresiran, results, XX% profile, we annual durable could payer addition updated as in to Phase knockdown offering for of We enable which supporting or rapid also in months, showing AHA greater to innovate the X XX. than was potential TTR subcutaneous ALN-TTRsc at robust broad that and and for in At with vutrisiran encouraging November, an patients, known safety I serum regimen an and TTR convenience look continue formerly ahead coverage. dosing biannual presented

FDA encompassing Together, happy also a cardiomyopathy. these both ATTR drug data to I'm Nucresiran best-in-class amyloidosis, polyneuropathy that potential has announce we the today believe profile. granted designation orphan support and for

start ATTR in III As with half is JPMorgan, advance of that to to quickly we study at first Yvonne expected announced in to the Nucresiran a cardiomyopathy plan XXXX. Phase amyloidosis

Additional details be on R&D February will XX. study our Day the design at regarding shared

positioned Beyond areas at pipelines development. to of innovation stages growth of broad disease deliver enabled us have indications today to most range and our and in TTR, the strong efforts across R&D build of one biotech a robust the all industry

pipeline. advancing In to our in make we the continued quarter, fourth great progress

Let few a me milestones. highlight key

initiation to III continue Phase through II We ZILEBESIRAN XXXX. Phase towards of investigational of to hypertension for expected second the trial and outcomes in cardiovascular therapeutic advance the start treatment a of half

announced of continues disease as for angiopathy our to morning, Alzheimer's II recently CARDIO ELEBSIRAN, the Phase this in X investigational cerebral have fact, well. proceed In amyloid therapeutic the we in completed enrollment study. treatment as

We levels and knockdown presented good high and doses, safety multiple single along showing multi-dose data with of with tolerability.

start X in cerebral of We new II programs amyloid in and have And a plan started the study disease Phase year. an in II Phase angiopathy Phase half the to ongoing last I study Alzheimer's second we year.

shown where for approach Canada The primates X the first targeting was Huntington's exon Phase disease, in knockdown has and we have a nonhuman ongoing trial United is U.K. with and safety the unique and I The sustained States, deep encouraging that tolerability.

review webcast that over impact our great of the Alnylam's that, deliver to meaningful R&D on diabetes ALNXXXX many Reversir programs join remarkable more we in the a me I self-sustainable The that bleeding or Jeff plus comprehensive come. to ZILEBESIRAN, platform it that milestones. other continued to programs you and of encourage can also to now to highlight in drive X XXXX tune Day described where have unique reviews position to our next and initiated for hypertension, QX host will we XX, pipeline and will RNAi disorder turn in TLN February I've of strong patients for results for to to let type years innovation mellitus. include This With upcoming a growth. Jeff? of will the era puts pace us provide for financial and innovation

Jeffrey Poulton

and guidance a had presenting of financial summary reporting from good and XXXX. versus guidance. original at Total and year were be for I'm strong XXXX our XXXX with global results of These product billion, XX%, product results came net our guidance of both at year. full XXXX for Alnylam's growth XX% XX% which than ATTR end complete full the the revenue in midyear results franchises with for we rare pleased and more our representing revenues XX% our growth raised providing everyone. Starting P&L respectively. top financial XXXX morning, Pushkal, Thanks, to $X.XXX XXXX

For the differences million from QX a collaborations XXXX, decreased revenue certain XXXX. collaboration net full exceeding in Compared million, $XXX to was primarily Leqvio with $XXX year, sales items milestone $XX in in annual X%, revenue million attributed revenues from due including sales. Novartis and between XXXX to

for During XXXX, we million XXXX In comparison, we the a an higher was exclusive XXXX, what $XX from sales connection by from recognized as of providing royalties double monotherapy. driven ZILEBESIRAN, more ZILEBESIRAN, associated with was our develop, $XXX on full and with Novartis recognized manufacture Roche collaboration revenues in than or revenue Royalty $XXX of. in revenue our license commercialize from payment million execution collaboration. the of upfront year Regeneron modification driven received million recognized to Regeneron to during by the in

a XX% primarily AMVUTTRA. commitments product was expenses sales These on adjustments prior manufacturing of the year inventory. margin partially improvement was associated sales of decreases due margin nonrecurring for were year, offset Gross with full X% payable other to compared with The for an in on royalties XXXX. by and increase representing net to rate increased ONPATTRO canceled

continue gross in on For rate to be as expect AMVUTTRA payable is the sales product on a Sanofi expected average to XXXX, as higher we increase. royalty of margin AMVUTTRA sales decrease

primarily as of multiple non-GAAP increased compared we R&D increased to year the with associated expenses. Phase and employee with study activities targeting expenses therapeutics our cardio ZILEBESIRAN our due clinical II RNAi develop XX% associated costs clinical Our with full pipeline expenses trial prior increased increased for year preclinical the tissues compensation

increased launch costs. personnel with year cardiomyopathy preparation AMVUTTRA our full and compared TTR with in for along business the in the expenses increased potential of Our increased driven for XXXX by of polyneuropathy for non-GAAP support SG&A investments XX% primarily

non-GAAP R&D top This pipeline For further non-GAAP as with of our achieving This strategy. a full as has profitability the enable we improvement profit to with million achievement is first associated significant $XX a we growth bottom will growth. deliver PXxXX a product strong in million, growth our XXXX, been the goal time, primarily operating and representing sales. line investment year by company in for long-term compared driven strive key sustainable $XXX milestone

primarily XXXX, of ended end and the with marketable billion in cash Finally, equivalents performance employee the the from received and common with securities increase proceeds compared of exercises. we issuance to of stock improved at by billion year operating $X.X option net connection cash, the $X.X stock driven with

Now I'd dated guidance. like product with revenues, ONPATTRO product January we to revenue guidance combined XXXX. and JPMorgan press our turn in XXXX OXLUMO that Starting release financial from to AMVUTTRA, GIVLAARI and our communicated our net was XX, are net reiterating

year We On products growth guidance the more of will billion compared $X.XX at XX% $X.X XXXX of to combined as than representing billion, $XXX for down XXXX full product total TTR, anticipate billion X our combined million midpoint commercial be guidance broken the a in or compared net the midpoint to range to growth the follows: XX% of the at full franchise a level, of within is range. sales to guidance year range $X.XX representing $X.XXX growth. of billion,

by PDUFA ATTR assumes date guidance Importantly, for of cardiomyopathy approval AMVUTTRA of in this the the March XX. and launch U.S.

XXXX rare, million at $XXX Additionally, the second growth full the of CM launches compared midpoint XX% the in Germany Total for and in range. XXXX. $XXXmillion we of an guidance of representing assume half Japan to AMVUTTRA to ATTR year

footnote guidance in rates the December as assumes our noted guidance are which of Our exchange XX, XXXX, slide. of foreign

providing constant also revenues growth XX% projected product rate XX%. range a to guidance with our exchange of net for We are

million the growth the royalty to range to million at and range. $XXX XXXX guidance revenue XX% compared of guidance representing midpoint $XXX Collaboration is

$XXX milestone study Our III payment with second guidance which anticipate the Roche for of a the assumes ZILEBESIRAN the of of associated we occur achievement million year. in initiation from half Phase our will

partnerships collaboration majority of XXXX. deal let well with Roche the and We royalty Regeneron and also will as drive as revenue with our expect associated our royalties the Novartis from collaboration revenue in

Growth investment billion, SG&A range XX% with R&D expenses for increased combined representing $X.X with including R&D for is driven between by Germany early mid- XXXX. regulatory non-GAAP in and Growth range our the assuming programs the and will late-stage in Phase cardiomyopathy ZILEBESIRAN ATTR billion in guidance include of SG&A for studies and Japan III clinical the of a and Nucresiran. midpoint for be XXXX approvals. U.S., the AMVUTTRA and across the primarily launch compared growth initiation pivotal drivers of $X.X Our expense

JPMorgan, in anticipate last discussed as Finally, month we operating delivering XXXX. profitability income non-GAAP at

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

amyloidosis XX, X. this entering Pushkal Nucresiran March intend we at action a on target have XXXX. host first Day the And our decision, PDUFA mentioned, the study the sNDA is of R&D date on note fitusiran XX, PDUFA patients As will beginning that partners the target to initiate XX City. given in February New III vutrisiran in March Phase a period of for will cardiomyopathy ATTR York materiality Sanofi March an in XXXX. action We for date with quiet of for half Please we The of XXXX. be

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

Christine Lindenboom

Operator, and to would then if X get ask have questions. we for to yourself will additional you limit dialed the question Thank any Those in, queue Jeff. we you each back like open the questions. to now call in you,

Operator

Ladies taken be gentlemen, and now Questions begin session. question-and-answer we in Thank the [Operator order the you. will will Instructions] received.

Bank And question of from is America. Ahmad from your Tazeen first

Tazeen Ahmad

sense that do to payer think establish and how of how take you the I the policies wanted long get going your think after team could the of to approval about it's initial a impact from trajectory you launch? to --

Yvonne Greenstreet

question on a could we're strong a clearly, to gives a supporting foundation And really you which That's perhaps this already patients Tazeen. having Tolga, strong approvals. specific question. a in . a franchise plan mean polyneuropathy. great with build. TTR position, ask Thanks, I time around us to

Tolga Tanguler

that the excitement we've is about excited potential and our the by really we're guidance already -- obviously, confirmed frankly, upcoming Look, provided. launch

comes think And we that But this value-based going you these formulary to little as considerate. takes growth the formulary the bit we've agreements remarks, have my of I is already committed prepared to payer be to be extended story. with payer a it a we committed the piece can that very much from and see policies going that is We're half policies second know some uptake, when really time. to Now to.

So I really the meaningful would second expect to the of half year. be uptake the Thanks. in

Tazeen Ahmad

And is Biliouris your question next . Konstantinos Capital from Markets. BMO from

Konstantinos Biliouris

from premium patients quick are us. on A XX% of for around X about ATTR pricing If we -- AMVUTTRA which you. represent step assume edits insured already Ambutra for compared your to are cardiomyopathy thoughts Thank approved who silence, the what population. commercial

Christine Lindenboom

question a that's you. for Tolga,

Tolga Tanguler

appreciate I Yes. can the really Great market. payers which question. discussions, that our product is product value in one, bring and to results. the we and And profile the by that we're still this obviously, with HELIOS-B see, mean, offer first engagements now at like Amitras the of the underpinned prepricing Look, foremost, encouraged also they

being all, positioned a predictability B we of product. Second the Part actually of as how are

see So our in we very is the as really uptake much with which XXXX. we already in how underpins be will all cover we've very these going which consistent committed expect combined, with to we that to accelerated be PN the the to have growth, payers how the seen past guidance product. terms And we shared, of again what provided, actually in

Operator

. next from Your Barclays Huidong from question is

Huidong Wang

of price ATTR AMVUTTRA. factors decision share that question, you actual ops? your you already sites can use a key the wondering the AMVUTTRA approval day, the know vital have what percentage And on for us in you impact related can I that current asked, into cutting the comment coating can show on already cannot you pricing for place but know few adjustment if with for a I

Yvonne Greenstreet

for we our about price, bringing think good access. Gena, as we've great PN a And CM. we assuring job add. dimensions, that well question obviously in to ecosystem want and consider And product that to to our price. we is to done value and an I be early do optimal access patient recognizing you you Tolga, those the when the we patients Thanks, job plan too think for the continue principles. have as and know, as it's specific equally Don't do we about along to And

Tolga Tanguler

on maybe or in itch. Yes, expect AMVUTTRA, any I point, to our current coverage, believe We earlier remain is as for our in smooth we have disruption to J on a respect from one our Day and changes as we to we TTR don't And last makeup look, used your product. to for we the And which is already perspective other answer Medicare the commercial, which therefore, that forums, Therefore, as code mean same. that. to business well be any result shared expect going a rather of coverage our going

for payers And well bring we're alternative, the and treatment importantly, affordability As Yvonne as indicated, mechanism additional really Otago patients. frankly, the for as patient. excited about going actually to then most disruptive the the and actually, how value that's

patients As going our uptake. the co-pay, you for same, to remain important which that pay know, be is an XX% driver currently dynamic patient and of X we our believe expect we to

Christine Lindenboom

Tolga. Thanks

Next question.

Operator

Raycroft Lori is Jefferies. question from next from Your

Maurice Raycroft

on Congrats progress. the

Just anything And related you to as label such mortality and of usage perspective provide functional on preservation there's front addition indication and wording life hospitalizations, and quality can are in and any unique the the advocating you discussion progress. capacity. page label section of wondering and reduction of if on to to have the

Pushkal Garg

on and review for going an the Yes. enthusiastic fully how of remain we PDUFA. really approval track Thanks, we're Maurice. is very FDA about first and by all, the date Look,

about So the we're that progress excited that's been made front. on

believe was and hospitalization designed mortality and we clearly the of that events. label, study benefit the demonstrated In on terms look, the

in And series ancillary a label terms we benefits benefits of the preservation saw functional expect of the endpoints and of as life there. that quality that that as will across of be we reflected ability, in well

the end study educate and occurs. an want and a providers endpoints day, at we able designed series statistically to results think reflects we benefit are forward in saw the we of predefined look, eventual a whole patients when that the that that label across So attributes to was approval and that significant of that sharing where we on we're go then look so a actually and

Operator

from next from Cowen. Your TD Ritu question Baral is

Ritu Baral

was but product data give or follow-up specifically sort good language clinical label, the actually to in in label a What you could potential data My section. question detailing about think mortality later in not, the the payers? potentially that advantage either with really label clinical the language. Maurice that mortality on or you of the having To

Tolga Tanguler

the that orthogonal coupled is as to out that action have as we're this. be with down we to a of all those protein ability fact Look, Mainly, is we results what our to of how well pay ability year an reached going Ritu, we perspective, have relatively mortality XX. adherence our given endpoints, Pushkal confident this rapidly the actually X to that actually a well smooth. the able injectable Okay. hospitalization both obviously, how we differentiate be great of to clear knocks indicated, XX% product on on pretty has times the mechanism and outcome disease-causing benefits actually profile pretty can as significant from demonstrate that then on clinical as seen smooth we've understanding X We're access going co-pay. promote, a the relatively patients given provides subcutaneous view And communicate prespecified on XX of that product had of communicate that as really polyneuropathy

how a this prescribers, very which we're of need disruptive medicine through going are carrying the exceptional be will to actually perspectives in much to those All communicate treatment.

Ritu Baral

to that in you the Those language though? Do actual expect still data? labeling be

Pushkal Garg

done, what prespecified the Ritu, things the whole end That's would trial. we drug series the expect and label that at that shown we said, is we've the of traditionally of show the this we've as clinical the on we that this, that had impact reflect in day, a Look, think will disease. endpoints of this

so be to generally label. And would reflected we expect in that the

results to Tolga's the And payers. be HELIOS about again, attributes consistent study teams and medical and those speak will able of to and providers our with labeling, team, B patients

Operator

. Your is Bank. Harrison Scotia from question next from Greg

Unknown Analyst

are expectations just and for is on for naive curious first-line on nutrisiran's potential This Harrison. I switches the what mix in to hear revenue from... Vitelle versus Theresa among patients the your setting updates. Greg was all Congrats

Christine Lindenboom

for question that you. Tolga,

Tolga Tanguler

were To and ambitious shows a actually to XX%. that and how going our our we're clear to provide accelerate really RTTR post of we pretty this best abilities, guidance able franchise

this and to look, the product need -- we categories, is product positioned there of that with the Now be another standard is also any well we happen going a how of do care cost believe mechanism But beyond is is, like line. that an to a a of action know first be stabilizers. mix to

naive coming eligible are from a of switches So we diagnosed newly some door the combination. instances, some that patients in that so perhaps and in patients certainly expect a for are good mix or rare

were built that, all our obviously, So we guidance, as do we elements these expect and incorporated.

Christine Lindenboom

Great.

Next question.

Operator

Your . from James. question Raymond Gary next is Nachman from

Gary Nachman

to going you with the the dynamics about out? could how at -- Maybe Part is cardiomyopathy And that's B both you between playing Part think with talk you're with competitive approval. D seeing over in growing products difference this translate And that how polyneuropathy and to point?

Yvonne Greenstreet

the was market. in mean, in competition question. that with TTR clearly, we're performance our with Thanks the very strong XXXX, pleased for and I

dynamics. we're think the perhaps you a job add business. some specific good the I continuing to doing build So to color want to Tolga,

Tolga Tanguler

Yes.

our XX%, you posted In faced relatively So 'XX, growth we first I going to always actually, for see, year category we growth can think base, healthy good the happened particularly been numbers, to Year where year, And fair story in pretty all is as New just And share consistent be a along. we've full the the their said be I that growth this tell, our from a year-over-year. that can with which results getting which this story. very business as telling growth of And competition. category with a also is, U.S. competition. we narrow U.S. what again, and was far where grew

we it's anticipate, this administered. to and significant We frankly, a we versus we any -- X given And expand year, not CP have cardiomyopathy. type seen dynamic access indication as expanded of differentiated times highly our profile product a believe continue headwinds. And we that it's had

Operator

question is from from JPMorgan. next Fye Your Jessica

Jessica Fye

X acoramadis of it think think you have for I kind as early to trying net to this expansion time. more in try a dynamics, ways, price evolution ask think in much right larger the the been folks relates the maybe tafamidis patient do population? AMVUTTRA? imply beyond of the different you TTR interest agents market but And about What market move this to AMVUTTRA's just What's few to into scripts and cardiomyopathy as way

Christine Lindenboom

to then specific a the opportunity, I be we Okay. a record makes perhaps this as Tolga said, first TTR Tolga, mean is obviously playing space. and category cardiomyopathy which I a I becomes in believe exciting track X to evolution questions market net the the color and question that there around the very a clearly, price. with too. question growth that's the of mean, strong mean,

Tolga Tanguler

comes if shared the I that market, on to mean, seeing prescription that Effectively the is really first they've sentiment a another and early coming data, obviously, some obviously. encouragement. into is times clinical mind stabilizer very are the stabilizer Yes. We're innings significant think, because shown look, a actually category. is and I X that growth numbers not that other year this actually encouraged with they've

Now profile about to undiagnosed really that given product that numbers. year to clinical when that be it's fact as dosing, AMVUTTRA think in about we're X positioned really going continue patient strong given -- well annual as AMVUTTRA growing, a be the excited the the very the we're what has category do profile to is access, be well times I

Operator

from is question next Stifel. from Your Matteis Paul

Paul Matteis

On cross to now. that to down are right evolve the dynamics Does trying is everyone between in to again, manage I underlying the you're on and category, at think, D? insurer this you And Part or expecting question and a it go to get pricing how not. premium significant B they have payer Part is average

the a this that relates it of it? normal a how or actually to at payer like this Or practice But step at going to parts, are liability moving space, right, is that's point? like acoramidis their as D of formularies insurers manage tafamidis them. There's X be in lot could on in Part just is just something not

Tolga Tanguler

is the condition, and going product how for expect rare lot question. be you there's payers condition this Thank this. to is payers will a noise and mean the seen a we around the devastating highly we've is evolve. of managing cover I to the Yes. What the market how Look,

managing product. clear have view I as we've a And been has pretty how launched Part eye given polyneuropathy D -- actually we a think how the in Part D product.

patients therapy We payer key driver on be favorable the to similar given of given AMVUTTRA. is very is a terms anticipate stay dynamics expect of mix. the patient in do we that And affordability how for our

Operator

next Your Goldman Richter from from question is Salveen Sachs.

Salveen Richter

about You do options? study are Phase half. how the How the plan availability new enrollment in III events, given of and to plan an a for you Nucresiran interim to conduct in the here? first And initiate timing thinking in ATTR-CM you of

Yvonne Greenstreet

Yes.

So we'll you detail providing R&D end thinking very around which thanks for little acrisiran forward program of the maybe about. Pushkal, Day our about be month. how this a the we're Nucresiran, share can question. more I at clearly, mean, at about we're bit excited

Pushkal Garg

We get that we've be what is sets to that's this a seen think knockdown, disease, Nucresiran that it really We've with less frequent think, therapy we with we've seen up, I patients this shown Yes, profile help administration, -- deeper knockdown. a so the far on XX% think can think XX%, already now asset. outcomes. we building patient going look, to over and derisked Salveen, for we best-in-class vutrisiran

I develop possible develop those for about allows we've development R&D that factors. Two, because how A, there's kind want we we quickly number label market factors brought we franchise, as value -- it that Day. think of broad And the about as how a of and this right? a think see clinical to the durable at we of for very -- bring When profile to plan do want it, think to and those for together that a uptake a so brings. you'll

talk in development to and CM and approach about will the PN We our more this program disease. there overall

It able to So share and couple a more. just will weeks, stay of tuned. be be we'll

Operator

from from next Your Merle question is UBS. Ellie

Eliana Merle

a for me. Just couple

in you versus cardiomyopathy, finalizing from cardiomyopathy could you value-based this agreements for would like how Tolga, of you that and relative to how a mix post cardiomyopathy TTR growth guidance can and be polyneuropathy polyneuropathy? characterize approval, work you'll look could the the So bit in what these elaborate mentioned on then

Christine Lindenboom

about X we're a there. Polyneuropathy, detail little bill bit approval? thinking cardiomyopathy Tolga, buying more post questions on then how and

Tolga Tanguler

second to stop how is the broke down the in growth more but in -- about obviously at of anticipate continue do where where We'll Look, the rare actually guidance take there. year we going half unusual to JPM place. launch to between and our -- our tradition, we we'll the -- think see do TTR I the color particularly provide we

question. Now you me remind the can second

Christine Lindenboom

First bill. thinking approval, how we're buy and about

Tolga Tanguler

value-based Value-based the covered of actually right? really agreements about outcomes. So is about agreements. our set clinical very, currently Approximately, that essentially And value-based the on lives agreements agreements, look, proud commitment value-based out. on are we PN the we dependent to we're provide are that very the the have the

PN, predictability the level about that and for the instances, clinical should the provide with is most already also of have So cardiomyopathy, and in expect this achieve. we how a perform for anticipate which to going the product us payers over experience we payers that carry to actually actually outcomes to

carry a that's indication we've over years. think that X great the best most I cardiomyopathy cases. of with So practices we great the in our and anticipate over to -- established that

Operator

Your from Partners. is Foroohar next from question Mani Leerink

Mani Foroohar

questions great know Congrats I commercially pricing and the probably results there's on otherwise. more and about X in AMVUTTRA line. that

could Is dig an NOL that question. if the not would -- what noted that or was of benefit So the year? drove clarify flash into and of And going course recur here? to how it I'm recurring? be that benefit ask this over OUS, actual Could the might just you you how dynamic the financial driver tax

Christine Lindenboom

you... for question A

Jeffrey Poulton

quarter a or allowance and release have with the more tax have to it's valuation We really ultimately value to something is as not press This our assess be going whether Mani. Swiss assets that, every to ex our recognized. that related do likely that's we is than that of not deferred in entity. U.S., question, noted a Good we had that release we to

a for periods number had now. has of entity And profitability the Swiss

confidence anything So all released predict that. gave released It's further, the yet. we've difficult Switzerland, that this the either very point not in release U.S. in We've allowances the and us valuation to Mani. to not

that now. at just for So leave I'd probably it

Christine Lindenboom

got time question. for think I X more we've

we think I questions coming couple up. of last

Operator

Your Markets. Capital from last Luca question is RBC Issi from

Luca Issi

modeling XX%, guess. Mani going I'll forward? we Maybe we obviously follow same don't is about I here at low think hearing XXs. assumption XXs in gross I gross appreciated. XXs, know that you Do there like should margin of compressing the even Is but of exact oral call think now, royalty a margin low reasonable much very mind. Any here But your mean to XX%, the think TC Jeff, upper a the the fee. meaningful that now Great. so how I it's from XX%

Jeffrey Poulton

Yes, I the appreciate question.

I expect mentioned in my product lower in that margin prepared we sales be -- So gross XXXX. to remarks, on

that of time, You're product. right One right. Sanofi, really be direction and the And XX%. on the for XXXX, AMVUTTRA. was royalty sales relate given will our our fastest-growing is gross -- driven of X product burden it's to it's our to a period now, things I lowest margin think do that travel product margin by

we will to the So move forward. put business grows, we And royalty as on move secondly, up also start tiers. in that margins higher as pressure that

on as this paid the AMVUTTRA number going that we to mid- that be rate increase to time, is, Again, is over So my XXXX average royalty average expectation for royalty XX%. moves was forward will XXs. also likely over across probably time. high The

amount just incremental that's So would I expect. sort that of the

will there's on that a Now way, of I things say, couple the would other margins. move

revenue probably If impact XXX%. close margin gross and very also you revenue look high to total a revenue. royalty the on of that's collaboration that at margin, our basis, includes that's

margin sales on at example, the XX%. looked in on you margin if product XX%. revenue, XXXX, was an was total As it But

with it. So to in if at that royalty the R&D we're on And burden, Day, the uplifted royalty said, Nucresiran, more as no high we'll that. thing there's successful about that important Sanofi burden this I then developing think is issue Pushkal secondly, most that hear

in offset in that to the this future successful So bringing that we're would market. if obviously

margins. travel of gives that a clarity direction So hopefully, on you more on little

Luca Issi

That's super helpful.

Christine Lindenboom

look, Well, really year is good thanks, Alnylam. for very a XXXX everybody, for call. the joining

on we to believe realize As I said, the XXXX. beginning we're just the success of in track

thanks, a day. everybody, So have and great

Operator

Goodbye. gentlemen, ended. you now Ladies the for and all Thank joining. has conference

disconnect You may your lines. all

Alnylam Pharmaceuticals (ALNY) 13 Feb 252024 Q4 Earnings call transcript