ALNY - Earnings call transcript Q2 2022 | Alnylam Pharmaceuticals, Inc.

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Operator

Hello, thank you for standing by. And welcome to the Alnylam Pharmaceuticals Second Quarter 2022 Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speaker presentation, there will be a question-and-answer session. [Operator Instructions] Please be advised that today’s conference may be recorded. now hand would to conference I company. the Instructions] like the to over [Operator ahead. go Please

Christine Lindenboom

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

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

I Reform quarterly purposes recently questions Harbor SEC. under future like result prospects various any most the important of file Act may constitute differ followed those and before these a today’s call, upcoming APOLLO-B a as on expectations, and in the matter. materially on your by our update clinical Actual for concerning results this Akshay outlined will general as would our Litigation contain a are of for offer call progress. Securities introductory Slide results to recent an with by Safe therefore factors, will guidance, During context. that we XXXX. preclinical X, be updates. on the statements from Please remarks period on will of those that plans provide to remind some forward-looking Alnylam’s milestones note questions. commercial we upcoming indicated review report discussed statements and forward-looking the including Yvonne which of quite call our And in global and summary financials will the Jeff our regards provide addressing open the will you remarks Private provision with review and Tolga not will

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

Yvonne Greenstreet

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

We to ONPATTRO’s we've potential OXLUMO. we quarter near- polyneuropathy. are for drive products, portfolio steady product long-term on achieved excited ONPATTRO with RNAi second with approval for recent marketed the we in compared with positive increase first the results patients continue and quarter hATTR forward our is we And GIVLAARI years goals. our that towards have XX% to expanded therapy the global launch a Commercially, our are progress and U.S. expansion. truly pleased a four from further And under with and opinion CHMP platform amyloidosis on and as our sales in executing Five portfolio including remarkable. very and across looking patients we're to approved for ONPATTRO, growth organic therapeutics made of

with patients to with we at we ATTR with cusp addition be are programs. commercial progress RNAi make portfolio, our to data to the line full that where tracking the our expect the day and amyloidosis, our franchise, In thereafter from in of therapeutic pipeline continue we on This within results study our growing medical in TTR weeks to top three X establishing plan seeing We've with Phase the data patisiran the next great towards released cardiomyopathy. congress. APOLLO-B of announced share strides are includes

progress we progress make In continue numerous also anticipated exciting our across within to results, highly addition to these other pipeline.

kidney with value medical positive top pipeline. rare Sanofi most end, one creating initiate to prior met end compared were breathing now from continue upcoming potential X of primary prophylaxis, we therapies X breadth bypassing the believe of ATLAS-PPX milestones and have line disease reported prophylaxis. to unmet scope the also of to program demonstrated and this data that results to underscore of Phase factor innovate years in biotech, be IgA excited by come. self-sustainable presented the our and of positive nephropathy, deliver X We part diseases endpoint patients To see by for Phase year. and with and are need. platforms with fitusiran patients significantly hemophilia, the reduce the in cemdisiran Regeneron, significant study a in recent and plans, the We're there that potentially the prevalent to fitusiran many in we which organic working Phase agent episodes a its finalize which that productive, what We and to has with meaningful The to

Looking the early potential the and updates NASH, HSV have of from this results including across and our AI we year, of for pipeline, in onset XVH end and through in Gouache, studies, to ATP expect AI line AI disease. early in and top to Alzheimer's

key drivers years. for several next Our this of on growth focus first Sticking our CNS endeavor. Alnylam’s three all together, these the highlights

potential TCR a First, expansion of franchise impactful become highly medicine leader delivering we differentiated to patients. to where is aim the opportunity, and our near in term global

to enhancements diseases key the innovation growth And financial comprised second our fifth for is third company, prevalent now opportunities driver while the for commercializing Alnylam our patients to The rare review driver growth me engine, the by to from company sustainable into over for expansion Tolga, organic our let beyond rare of top all call new product comes biotech turn diseases. platform, Toga? our product high and can genetically as by diseases to commercial yielding performance. of making developing delivery, the transformative ability further believe world, which of new positions on validated and results. best-in-class a expansion all targets, P Alnylam and/or beyond. XXXX We drive extrahepatic candidates our deliver around well exceptional this medicines driven our engine, to pipeline find pipeline deliver goals, and that, common With to and XX first from

Tolga Tanguler

We're a the [indiscernible]. about detail and for I have that And we FDA by early was June. quarter as quarter-over-quarter strong everyone. share our shortly. also commercial QX Yvonne excited XX% with approval AMVUTTRA good morning, more in launch promising of on Thanks Yvonne. encouraged size portfolio And of to are highlighted. growth

anticipated, experiencing improved conditions promotional compliance we of performance we of products. are also flows, and now market patient QX, our impacting healthy as improved demand, on improve increased our portfolio. activities, saw the following across As patient details COVID the provide I'll each

in primarily We continue first compared and growth results timely QX, over XXXX, by in with strengthening representing in back by to by increase orders revenues with see headwinds which quarter percentage exchange held second impacted therapy in distributor X,XXX At was to improved to year-over-year the global million Important the international patients QX the end In quarter, QX, the therapy ONPATTRO representing ONPATTRO markets. patients and steady versus with in foreign primarily and XX% quarter, note seven sales an patient U.S. XXXX quarterly negatively increase points XX% the at global ONPATTRO increase by due product on patients on reported quarter, XX% first and XX% product being compliance of increased markets were patient our growth versus commercial COVID. QX the end over XX% continue compared of treatment XXXX. to QX for and on up U.S. achieving $XXX ONPATTRO following first worldwide, In of an ONPATTRO dollar. X% challenged be partner increased the net growth. a the from our growth of impacted were the patients sales

with new in very on forms which hours Over of serving forms the of patients to to early and As one reaching generated key two within pleased our with switches believe thirds our hit through growth. end quarter. XX over execute launch clients. of the you start encouraging ground aware, prescribers, we of for from an from for with U.S. engagement. second from generation sign came And approximately track We line July approval from third are of so is launch stakeholders We received start the field as market from XX% continue XX,XXX start XXX we're running, those catalyst demand forms keeping far received as we potential Alnylam, AMVUTTRA the at initial a ONPATTRO. new launch XX teams

with started formulary date. our networks. teams from been of over posted have been in standpoint, XX% access feedback been engaged the to deliver have the We Further been engaging and systems has also an has processes priority health and

In is published large one a fact, there lives. policy national national with payer million XX covering

call. rollout has patient you also been AMVUTTRA treated first QX our updating The on we're continuing for further looking and and this

favorably In and X%, by by quarter improved deductions we XXXX. ultra-rare of QX, quarterly quarter, our launch with a markets, first patient franchise, in QX impacted global COVID. impacted And growth new primarily by compared At a increased disease over growth in XXX reported with which adds, to in in the XXX XX% UK with from representing GIVLAARI patients favorability growth versus and quarter, patients QX, on of of GIVLAARI, XX% and demand U.S. net the treatment favorably international the end first a product soft first delivered GIVLAARI Inventory the driven sales X% compliance XX% of net quarter. reported strong primarily In our the dynamics, increase the were second XX% up by of therapy growth healthy in over GIVLAARI revenues $XX gross growth. degrees achieve growth X%. approximately increase quarter, the growth in which versus net driven an first compared following including in a a stocking the worldwide, Moving primarily growth on patient with result the following: representing by the QX commercial the deductions. and impacted a XXXX at million a end patient we're XX%

reported XX% foreign by year-over-year points rates. to percentage GIVLAARI favorable exchange six also global was Finally, of our held due growth back

X% business, XX% million the first patient to stock increase compared Moving now OXLUMO at of quarter, a OXLUMO. growth In up the our OXLUMO despite and quarter, in well and QX, treatment second quarter XXX in the sales a with in were distributor gross dynamics on XX% of versus patients net timing quarterly orders end as the inventory during during quarter in commercial to the us product net increase of an by due by sales and On growth. markets. compared primarily deductions second an in deductions patients decreased the OXLUMO year-over-year In our XX% partner therapy quarter, and the on in in product QX their net despite to international a increase the the on lower treatments as disease was primarily of as decrease an increased X% The primarily the first decrease $XX on decrease sales end to international first the over approximately OXLUMO At in quarter. XXX of global representing during patients dose a of worldwide, were an representing monthly quarter. our due loading markets patient in from impacted the global therapy. achieved proportion of as our with QX increase patients pricing well basis, XXXX. We portion over net doubling revenues QX, higher to ultra-rare demand

progress. Additionally, percentage recent results, points patient strengthening due full exchange minus demand, as changes turn I of IND OXLUMO and to with that of QX conclusion, it with ONPATTRO with which quarter look growth over impacted In pipeline back and and XXXX X% to foreign Akshay QX year-over-year With the forward negatively pleased our our the now revenues dollar. also in X rates first launch. GIVLAARI will and the held are U.S. achieving reported include we AMVUTTRA's will to in results to growth Akshay? review by QX

Akshay Vaishnaw

stage doses and Vutrisiran. candidates: Thanks we amyloidosis AT efforts Tolga product start everyone. advancing our and I'll clinical where two are Patisiran good with in morning,

are with Patisiran in world markets polyneuropathy currently While hereditary ONPATTRO AT doses. on treat around associated the amyloidosis multiple approved to

the in approved the weeks. both as product's AT injection Vutrisiran, to advancing of within conducting this which for of We're U.S. wild-type results amyloidosis to in we subcutaneous me, three expect Phase under positive line delivered next to study X the brand to patients. hATTR and treat quarterly and report in polyneuropathy was CHMP the name We the are is treatment the Excuse in to top announced AMVUTTRA committed label opinion the this the addition recently also amyloidosis APOLLO-B expanding end and we're cardiomyopathy the hereditary by morning, EU. receiving

HELIOS-A in to we primary which In for patients. cardiomyopathy evaluating of and to committed AAN the secondary Here also polyneuropathy Disease. met expanding meeting, recent formed approved from by submissions label amyloidosis shows basis presented the regulatory the Vutrisiran at in positive wild‑type nine we and in treatment U.S. the months. study April study hATTR is AMVUTTRA. the patients endpoints with the for development its hereditary and Stargardt results XXXX Vutrisiran are at

and polyneuropathy, new patients results with cardiac improvement findings exploratory consistent to – in findings endpoints cardiac These XX-month the the to that results treatment ESC amyloidosis cardiac in associated cardiac previously pre-defined with These show towards Vutrisiran meeting. in endpoints recently the HF study with improvements report NT-proBNP in of including subpopulation continued was pre-defined levels at a in exploratory external from results the a in with We variety placebo, sub-population hATTR from show presented the trend and of population. parameters. were a of echocardiographic reported mITT relative that

patients patients, those on population, imaging amyloid patients that baseline, planned from safety relative mITT may an recognize a scintigraphy highest the uptake with from RNAi of including to reduced degrees in profile. equal accessible the of burden also demonstrate suggesting in cohort baseline to and continue paradigm encouraging treatment with benefit of of Additionally, Vutrisiran therapeutics. cardiac two the tolerability to at technetium cardiac majority Vutrisiran

all-cause As from both expect in mentioned early Phase has patients followed following we and amyloidosis the regulatory the potential months including authorities. wild-type engagement this patients includes with cardiomyopathy amyloidosis. and start and enrolled of mortality is with up HELIOS-B study treatment and consider XX many endpoint with hereditary is in being to AT full we Vutrisiran for interim in study an will the analysis, design for HELIOS-B XXXX. just results which AT results CV as for this of X evaluated APOLLO-B is the with the The XX-month events a fully also

or from the baseline X Regeneron benefit late been component mid-stage the study complement related of the treatment this these XX% Cemdisiran was consistent and look our is of with relative of reduction the for believe line a patients second data targeting disease top endpoint study an to marker and the of monotherapy of and agency end development therapeutic addition year, This Phase week-XX at urine by we've from a hope investigational resulted making were to IgA results also we The the IgAN. were programs. efficacy highlight and clinical no IgAN. development of support with in for In safety CX initiate an gaining placebo. signals, program in continued Cemdisiran Cemdisiran and of protein-to-creatinine progression. line endpoints Phase to feedback. the forward in treatment stage prognostic of with was and programs, early in in collaboration nephropathy clinical collective results important finalize of therapeutic primary believe the pathway plans secondary our IgAN. pending in with also drug the this our regulatory now with Regeneron X a progress mean safety In of great Notable There to we to positive in We significant ratio quarter without study Cemdisiran RNAi announcement XX-hour

come growth a Alnylam in product engine organic the for driver be years innovation. will key sustainable on our driving Moving

and candidate exploring data INHBE the to further referred X of treatment for Nature lipid against therapeutic organs. actively impacting the of Communications more associated With and other we Nature potential development be The in waist-to-hip conjugates to since CXX of publication published findings associated mutations adults lung abdominal new a potential research achieve loss function syndrome, the tissues for as a lipophilic the another we gene product of out evidence gene conjugates delivery INHBE obesity help to its metabolic Gen second and data was condition identifying treatment platform. improves the support this early potential diseases X'-O-hexadecy preclinical Finding extrahepatic quarter an Diseases, IKARIA lipid this novel with eye as conjugate features with of with a which a is time research INHBE plan treating siRNA In pursue ratio from in highlight protection to CNS including in for XX% delivery of a Biotechnology than and in to previously of avoid. leveraging improved In the INHBE the regard. evaluated Cardio-Metabolic to provides These profile. of CNS. role on siRNA published

the incredibly that can upcoming and As advance recent that to the forward milestones. now financials review you let turn continues our broad an to and programs these and have just Jeff me number appreciate, coming With to platform we updating a on these Jeff? few We call you look innovative over to in are of a months. highlights.

Jeff Poulton

of increased pleased also progresses. results our revenues be our collaboration Novartis's growth quarter our collaborations We financial good and the as XXXX. to work primarily year a for with summary or milestones a with update our QX by the associated $XXX product I'm combined via during that royalties were compared QX Net U.S. presenting full It's to due representing of for impact with are quarter, the the results of which given everyone. do P&L to QX our Akshay, XX% in programs, by activity Total held XXXX. worth launch of increased QX for markets. is XXXX, revenue guidance. summary by the revenues expect and of second an dependent Thanks in Starting well completed which XXXX the sales in XX% high driven Regeneron product a an million growth foreign strengthening across Alnylam's and generated year, exchange subject on of And Regeneron. morning as increase an versus to dollar, half reached in approximately reimbursed a of due sales U.S. to noting XX% that royalties including the X% is XX-year revenues from as Leqvio international year-over-year from was approximately a product and million recently. an factors of variability primarily quarter approximately Regeneron reduction a decrease quarter-to-quarter collaboration, $X revenue second from variety which was revenue level back

in same increased and growth of headcount the in second support R&D our activities Our compared the to spend the period XXXX non-GAAP increased increased to to pipeline. XX% development early expenses due quarter on primarily

commercial in non-GAAP the the quarter in Our portfolio. headcount to increased period the and to second of XX% same SG&A increased expenses support other to compared XXXX, our primarily due expenses growth

XXXX was XXXX, million non-GAAP QX by quarter. primarily reduction a during loss Our for with the loss was collaboration impacted which million the operating compared QX $XXX higher $XX representing in revenue

end Finally, and of end the $X.X billion securities compared at of XXXX. at billion with the marketable cash equivalence quarter cash, the to of $X.X

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

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

net of million. operating QX, guidance QX the we anticipate revenues Following product financial will combined four our revenues provided in we net our be $XXX between results for and we the strength call Starting reiterating product results are with million products commercialized our April. on in $XXX

However, in of product April, continued guidance of sales XX% range. and that the dollar are the our to trending currently the issued towards given the lower generated million we guidance our global half our international in million the $XXX strengthen we since fact of approximately $XXX are U.S. markets,

and million, a million. revenue between and ranged royalties Our expenses our million from collaborations a is $X,XXX guidance and R&D ranged for is $X,XXX SG&A combined and net non-GAAP $XXX $XXX guidance for between and million

through financials XXXX. of AMVUTTRA. discuss commercialization upcoming me Let executing ONPATTRO, as as We'll turn on goals and GIVLAARI the the deck continue OXLUMO key well milestones our some on global end from of of launch and now and

Vutrisiran X report a in and APOLLO from expected will line weeks. biannual have franchise Disease updates plan in we both to results with late top initiate X our on Patisiran's Phase next important results regimen a Stargardt Next study TTR Phase in dose three XXXX. the With are study the

Preliminary or in looking our the stage mid-stage in in plan Lumasiran line we to up, milestones in that year. results around Completion of XXXX. recurrent expected antibody Wrapping partners X the patients end we study with from to Phase X by expect Phase Phase of include of with and enrollment as file ALN-HSD the X healthy portfolio with Alzheimer's are with results ALN-APP are enrollment year. combination by study ALN-TTRscXX of onset the Lastly, the Phase a monoclonal top ALN-HBVXX preliminary XXXX disease this we to initiate Part late of from top coming from IND with for forward and of patients study stones KARDIA-X year-end. we include Phase X an well. results Phase completion the of study readouts of that study in B of In study expected in X for from and VIR-XXXX, Phase year-end, in Zilebesiran early renal expected ALN-XDH in which later the mid-XXXX. a our report at have study in These patients results also patients line early late of X few top line NASH X gout in volunteers

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

Christine Lindenboom

call responding a our for then will Thank to yourself to each ask we will you, topic. you like the one queue open be as and limit have with and Also, questions period now not the To additional regard to Jeff. you in if a that APOLLO-B those to question in on we are in study dialed get we'd questions. reminder quiet any questions. back to Operator,

Operator

Thank [Operator with Our Instructions] first question from Paul Matteis Stifel. comes you.

proceed. may You

Paul Matteis

good Thanks much for Hey taking morning. my question.

compare a one, two-part the the on ONPATTRO for can guess for to commercial Vutrisiran, then infusion? on an patients talk how versus you from of question the physician Patisiran just a Thank is coming cardiologists? Just and curious that you. prescribing dynamics, [indiscernible] And economics I and in-office as how does new initial much for dosing of that just about

Yvonne Greenstreet

very, that its initial off we that have also question here the with hand just over AMVUTTRA encouraging. this very it's and to start target the great patients for question. I'd I like really you. by to call, Paul Thanks to have to delighted sign are ONPATTRO that and Tolga, like on additional but [indiscernible] to saying for

respect cardiology to any economics from the the with around the dynamics perspective? fitusiran then it's commercial So to on how initial Thanks launch, Tolga. compared much physician and commentary ONPATTRO to a in with respect

Tolga Tanguler

are Hi This within over really we actually and weeks We weeks. robust the one-third to need sign is product Patients product a in, the system were Alnylam that's of that's as start five and reporting patient able these an naive us but the good were early start early Paul. only patients. new very available for make very In receive launched it's the and sure forms XXX are and Absolutely. forms. to five actually number, information to get one-third actually of go through and the they new dynamic They time. us exciting encouraging. as that that those is to naive those

the about prescribers a What also ONPATTRO. economics, of in therefore we buy-and-bill is prescribers and ONPATTRO. the essentially see very very prescribers as new frankly of product looking TTR, dynamics of is B that our are similar are still we're a terms going of period in excited those Part is In patients be new this it to XX% at short

that also that's I'd dynamic very underline. So like exciting to

Christine Lindenboom

Tolga. Thanks, thanks Paul. And

Next please. question,

Operator

Thank Lebowitz Citi. with question you. Our David from Instructions] comes next [Operator

may proceed. You

David Lebowitz

my for Thank taking question. you very much

pricing And points label? want little the know as well. First on added as type to also add-on you've give one releases, primary as secondary we the on with cardiomyopathy on end P-values just to in confirm might not past for shift just far respect likely far can historically any line endpoint, that would you the we the once goes, but see is Vutrisiran, this to, details as I of see the presentation, certainly what gets here I at I put least perspective top out key to want values. as us on data.

Yvonne Greenstreet

thanks for David Great. that question.

I you. first think is one Akshay the for

Akshay Vaishnaw

Yes.

the that clarifying to but you the said results, am I you respect Just met suspect Vutrisiran, APOLLO-B correct? Patisiran with I Dave

David Lebowitz

Indeed, of course.

Akshay Vaishnaw

order. we'll right the as top present a are secondaries PR the with the test in line and primary form you hierarchical we And results Yes. in of P-values

[indiscernible] time. much that's as So present

David Lebowitz

Thanks Akshay. hopefully Cardiomyopathy? question, Tolga, the the shift could and you second pricing And respect maybe take we to as any enter

Tolga Tanguler

Look to little when as appreciate of share make obviously we'll too us soon, we're update any excited but decisions. a because about those serving Cardiomyopathy it's little information soon appropriately possibility patents, could a you but it's for the we too

Christine Lindenboom

Alright. Thanks David.

Next question, please.

Operator

Thank you.

Salveen with Our Goldman next question comes Sachs. Richter from

may proceed. You

Salveen Richter

Good your you. regard switching and for between you morning. to TTR just dynamics speak to Thanks Pfizer’s playing combination Could franchise Thank and out question. the taking that's tafamidis? my with

Yvonne Greenstreet

I Tolga think is that question over straight that to you.

Tolga Tanguler

mean, the wise we're therefore any and see I we for do Yes. don't switching polyneuropathy U.S. dynamics. indicated in indicated switching we obviously cardiomyopathy, for not tafamidis – the

new of rates polyneuropathy seen switching built our is seen use past. a significant what of common see, those the really that that In as we XX% business a to to to in that we terms by about and the ONPATTRO Europe see adequate in we've we've which actually we've – us to similar particularly Japan, but in source in to both switches. part is continue probably on the and we're the naive earlier more believe in about disease the seen we Japan available alludes use in where It's What Obviously a Europe add strong XX%. of good, patients opportunity between physicians anywhere treatment. dynamic there ex-U.S. silencer is excited dynamics get of both are products early

Christine Lindenboom

Thanks Tolga.

question. Next

Operator

Thank you.

Cowen. Ritu comes Our with next question Baral from

proceed. may You

Ritu Baral

Tolga, are Thanks guys. AMVUTTRA? you prescribers, patients. mentioned look up seeing at detail there's patient of the I sort patients to does mixed just we – the less on some was Is a seeing Paul are you the patients? phenotype more but new taking warehoused for Good to new there's the And morning more little this work severe patients? to just imply through question. follow for that you and sort bolus Salveen's of question as for hoping

Tolga Tanguler

AMVUTTRA point. give it's area the too we great a What really to you. for that early indicate great we're be this but was we even we going the robust seeing of or some It's little see the that it's that We're it's an in the little launch. patients, treated. of broad again It's had – some range physicians do again uptake quickly we're we warehousing warehousing the of weeks not fact to of it, patients, originally a five side first thought the Thank is lot of be about but sure Part the that a QX part soon either is waiting obviously that about With will I those for specific switched see dynamics. warehousing, that I'm ONPATTRO Yes. probably getting only patients certain monitoring. what bit is into in question. exactly younger But indicated excited important of before, encouraged and that a to contributed being If we're five growth. us be weeks didn't difficult a at dynamic. are where highlight generalize closely What might happen. a the little to we to patients I'm strong

Christine Lindenboom

very look going Yes. helping think the Ritu. grow help introduction Thanks I really overall us, does TCR the Tolga. is of at encouraging. franchise us which Thanks, It AMVUTTRA forward is will

question. Next

Operator

Thank you.

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

may proceed. You

Tazeen Ahmad

opt confident is to that for of HELIOS-B, just morning. all mortality would Mine's a allow benefit. something early the question. seeing there you significant chance your Thank which improvement for I'm in so that in the observation wondering to a period higher target is scenario at XXXX a feel though Good extend about. data Hi. my you on you you've confidence readout for taking reiterated

Yvonne Greenstreet

Maybe by perspective that an confidence HELIOS-B readout study XXXX I'll data Akshay might how about and on from we think our going forward. stop any in early retracing

Akshay Vaishnaw

powered Is Yes. study thoroughly factory how help of and a We've study? we the to assessed study and is the the endpoint meet the with it comfortable and robust endpoint way primary design. are us revisited designs in structured the

in have and study the the unaltered consider say the And come is only that, study. of patients over robustness in will analysis, interim we'll would thing the in so of like of a is as And due course the you, in in gone the a way to thing coverage long designs large the the terms study course. I by that, other period them that of provides months so many XX study additional

comfortable and and reiterate we'd early positive later forward we're looking So results. XXXX to

Christine Lindenboom

Akshay. Thanks,

Next question please.

Operator

Thank you.

Stringer comes with Our from question Needham. Joseph next

may proceed. You

Joseph Stringer

our question. Hi, thank for taking you

Our early you. anticipate your experience the form question drug? on relative from this ONPATTRO? to patient I for getting you its AMVUTTRA. How start with you compares was days how do but Thank do to time know think

Yvonne Greenstreet

That's franchise, Yes. product and great the on building under about that could commercial with that leadership. second very Tolga's [indiscernible] a we AMVUTTRA have question bit clearly TTR in little robust our speaking? being a perhaps talk operation a you Tolga really we're what's

Tolga Tanguler

going regional with place. payers, our right Obviously what formularies do and ONPATTRO patient experiences major day national we done In integrated the delivery and well is have right maximize category. in of working opportunity mean, months. closely able – the being injectable and regards also the work make the providers far be positioned given And so to other in with Thanks Yvonne. look, end very in AMVUTTRA's attractive is and capabilities, to so are with already regards the services, subcutaneous how this but AMVUTTRA to to networks the verification, only health for place timing we're sure really we've not that forth benefit at with profiles within very is to to – every I

We're with with how what early and to where it's some but see I early eventually for approved. fact we so we attractive going a get any very time parity not faster significant are place us it's its new or at be little pleased far to the set right delays any now place to but for and sure the to think like we're the soon system to to reacting, ONPATTRO the despite even a headwind. it are Nevertheless little also on forth parity so done Given take the far systems too with absorb with price seen and PNT its committees signs does product that very get where the going that. profile make are of a maybe I'm we have product healthcare share we've and ONPATTRO, in dynamic, healthcare actually approval so sure the

Christine Lindenboom

Thanks, Tolga.

we're thus think pleased. I far good progress very and

question, next So please.

Operator

Thank you.

comes from question next Raycroft Our Maury Jefferies. with

proceed. may You

Maury Raycroft

Hi ask on taking for was good Givosiran to for hypertension. and questions. a morning going I my question thanks

that? on on to to taken you can elaborate the the speed protocol streamline Just wondering status steps and you've is enrollment what up if

Yvonne Greenstreet

Yes.

fact it’s those with selected unfortunately very to war to our with Just KARDIA-X were a the expanded zilebesiran and progress everybody in the due unable experienced and obviously primarily sites a we we upfront. in We've reminder, study sites that ongoing the delays fee with Ukraine forward. move to site our footprint

the protocol Akshay, don't [indiscernible]. any? We study to want to took do it you opportunity easier refine add to make the also I know, execute the an just to

Akshay Vaishnaw

of given capture The some assessments, you information to and away and may think we the the and effects can. – don't specifically to of is these and Yvonne. the take as two core the in one approve thing one protocols, more downtown we're streamlining which combination hypertensive one much But I remove in be large No, covered issue able monotherapy robust tries only more so protocols, the therapy. easier anything from will maintain yet one needs program to on in inevitably it other as scientific safety,

So between things, these optimistic year. next are about the we and profiles readouts expansion the site

Christine Lindenboom

Great. we're to of to this enrollment at the just And end expecting in that year. add KARDIA-X complete

Akshay Vaishnaw

right. That's

Christine Lindenboom

So we're looking forward you. Thank that. to

Next please. question,

Operator

Thank you.

Issi comes RBC Luca next Our with question Capital. from

proceed. may You

Luca Issi

know but that implications footprint. the for point, confident much. they about I franchise? are we full given very you this polyneuropathy, Phase question. a have the for AstraZeneca global much at Ionis successful taking on of so are so you data how on X Thanks thinking compete reported about Thanks quarter. it and Maybe thinking here? TTR can don't landscape Great. the commercially my competitive one launch Obviously how seem Congrats have your

Yvonne Greenstreet

compete think PN how I to stream competitors look going was with the in question believe Yes, that that AstraZeneca. about, coming with we particularly potential primary space, Thanks. TTR on new we're

Akshay Vaishnaw

Yvonne. anticipate currently what are XX,XXX you, PN that are it different to first treatments around prevalence chop very are of being alternative to patients Yes. TTR Look, that to Thank of treated. that of the wood and the excited and been we medication, small there's because to lot fraction of We numbers a comes new a able when all prevalence deliver. to bring patients we to always worldwide we've XX,XXX mode patients

of able seen an what look driver including been the rates treatment we going to similar of In and by diagnosis ourselves expansion in end tafamidis four on. are, been categories, in company. where last also important So promoted establish a the the actually good in regards Japan that's have day Europe against we've we at to the major and as years

the drivers be worldwide. making we is available One growth of our think, are well positioned AMVUTTRA important, going of the that. And obviously do I to

We XX footprint partners through have Europe and markets and Japan geographic other over and available in and distributors.

set products year to that of subcutaneous that – and be over competition that. for trials patients product able be to how that available patients, continue going to want available the again AMVUTTRA that's I a to again, those there three a months, just we're excited the a leader. six the are be to to portfolio as terms offers be in Given and We work. having But about fact well with soon already make said positioned this off and attractive months good plus like leader we're ahead we're a reiterate, and very our for profile really chats if with injectable and going

Luca Issi

Thanks.

anything [indiscernible] So Akshay, effective about profile? say to do you want

Akshay Vaishnaw

we've associated around is the Yes, of very uptake. of and very scan but was comprehensive physicians. for lot of secondary, the with technician And endpoints, reducing of the the for but AMVUTTRA notable, the exciting. particularly but points showing impact on makes those other no, of neuropathy still I'll And patients Tolga in respiratory just in [indiscernible] which and and data with themselves, a add primary vutrisiran the course exciting not new a the sense, exploratory end terms that's which just said to the cardiac improvement data terms in got what hATTR endpoint, heart in I the observation think, that

so, will monthly and effects. hopefully some QX, in note a see that inotersen love the ultimately [ph] that dosing, need reactions, sure this product to with is keen safety And frontiersin to We've patients the full issues time and options good. I'm and including space leaders data been there is the and want add along convenience explained. good very that know And with with Tolga renal were they, will market injection the that everybody is package round in then associated the safety I effects, soon this of QX recalling in first site platelet differentiated

AMVUTTRA. So to think let's see, start I with but off we're a great

Luca Issi

the for opportunity. Thanks

Christine Lindenboom

Thank helpful. you. very Sure,

please. question, Next

Operator

Thank you.

Nachman from goes with question next Our BMO. Gary

proceed. may You

Gary Nachman

Okay, great.

are switches sense the forums shift third you. ONPATTRO? switching So on get that do a they and back stay takes that getting Do AMVUTTRA it's to the if what's AMVUTTRA Are from couple start how the until patient they over of of quarters? initial ONPATTRO, more profile up split that from patients driven? physician the two next and you some between switches And time? ONPATTRO new Thank you expect or if and to

Christine Lindenboom

of we AMVUTTRA that early lots than demand. I the think Tolga interest have

Tolga Tanguler

us Hi, signs the really is patients. and switches, And so as it's to to profile in what what early the AMVUTTRA’s is more at about we've the look be seen naive to patients. able day, both initiations Gary. of the terms diagnosis I actually is giving it as end mean, we're well comes ability ability when excited and treat far physicians of

We that have and prescribers as convenient base, profile about with well far we've into have ONPATTRO efficacy a our as and forms and good of do informed ranges was younger in been those with probably physicians. condition to infusion seen earlier across excited treatment both What demographics is, of providing what availability great some is prescriber as out and good, so indicated services to that range we patient broad option a start this demographics. robust patients also well medicine we on seen that I new, patient those traditional were as a their data. of patients to safety the AMVUTTRA patient the earlier, a be patients tends the very this and reached given actually an coming Therefore offer to more as good, XXX fence proactively

Christine Lindenboom

Thanks.

So, let's question. the next take

Gary Nachman

you. Thank

Operator

you. Thank

J.P. Our next Anupam question line of Morgan. with Rama

You may proceed.

Anupam Rama

so what dynamics, on much switching following on last so market to does expect have question in think question. I trying would AMVUTTRA switching understand, much. the most you that's the on patients switch? going the Thanks switch many what or that which research peak your to thanks the on of kind us. guys, where are timeframe to taking Hey suggest Maybe made of for we're

Yvonne Greenstreet

putting thank precisely. great a you That's question. Anupam. Hi, forward for it And very

I specific So what here sort for is setting Tolga, time around switching. the think question up about a is peak research there of right our market

Tolga Tanguler

at really this is to the point interested is are that make What category growing. we overall really sure

really is will patients switch Street expect organically about believe and make as update year new a now right up actually net this is the bringing for as patients that to what out more play option. What will to and a going is sure about new switch given we possible The the is focus to our as its So we are obviously happen AMVUTTRA little profile. we end is most with naive patients. that given many organically

with in their diseases, know existing that also who patients pleased treatment. we rare are are However, there

before ONPATTRO treatment available. of Some had these was new never patients any sorry AMVUTTRA

tend quarters. do to patients believe that option, better a in seen remind wanted categories to AMVUTTRA gravitate take everyone other opportunity that three expect more we dynamics to So be But to But remain going we've similar I similar some that which to updating on to dynamics the towards two form the dynamics also we are – is on we've seen we is. start for therapy. in patients

Yvonne Greenstreet

same a Just doesn't patient per same comment. matter the or price, ONPATTRO AMVUTTRA one for perspective. per modeling It other From value year.

reminder a that on point. just So

Tolga Tanguler

point. Great

Christine Lindenboom

Good.

Okay.

take Let's the next hit – question. the

Operator

Thank you.

question from Barclays. next with comes Our Tong Wu

You may proceed.

Gena Wang

you [Indiscernible] can me? actually, hear Hello,

Christine Lindenboom

can hear we Yes, you.

Gena Wang

so thirty-three I to Gena one form question good. Yes, start a Okay, follow-up Barclays. Wang This form. is maybe the from start have

four [indiscernible] and give quickly I these digit, the on check, you savvy what low can just of at we tech bit little from truly naive to are clarity, maintained the said change dropping to thirty rate in more some single Alnylam versus patients? switcher rate? HELIOS-B if dropping do You percentage then a any just maintenance last discussed And wanted time the new

Christine Lindenboom

Okay.

HELIOS-B Tolga the back the then question, AMVUTTRA and question. with start of shape Let's

Tolga Tanguler

We strong really five yet of launch week AMVUTTRA, only around the data. appreciate of excitement it’s the

Those all as overall providing own should sure is the remember also that make we good we're XX that of us. category. modeling portion one very to can terms naïve data small new a to we wanted are at recognize we possibly your So And that TEGSEDI remains this as point. of patients in the the [indiscernible]

So an necessarily alternative we only naïve just on index wouldn't treatment. from our switches patients

Yvonne Greenstreet

Yes. to grow. Good opportunity

Tolga Tanguler

Exactly.

Gena Wang

New patients coming into AMVUTTRA.

rates. So actually HELIOS-B has in drop

Yvonne Greenstreet

sample drop size well a Yes, I thanks, in put expectations. to obviously And reiterate discussed in buffer can the I the in within rates I've tap Gena. remain had before we account drops. think for

well are those within We estimates.

So we're design you. with Gena. the Thank comfortable

Christine Lindenboom

great. That's actually. Thanks,

Next question.

Operator

Thank you.

Merle with UBS, comes question proceed. next you from Our may Eliana

Eliana Merle

looking the could just biology terms stone I Hey we could formation the with guys. Thanks later much Phase on And X potential segmentation of types patient of the a the reduction in sort what pipeline guess about stems, but much in X oxalate in like? your see And recurrent next for between confidence in I this mean, the question, then not a learn population. taking broader of broader the ATTR and we this I should pathway larger here obviously relationship any and regulatory guess thinking I of are guess I population an look what population? Lumasiran what from renal like maybe there, there? about X like how for terms reduction and it's accessing this about Phase question. in think finishing thinking guess Phase year. year you data and to enrolling

Yvonne Greenstreet

sure you of that. called all not I'm if Actually,

Tolga Tanguler

Yes.

Yvonne Greenstreet

forward how I then looking plans reduction I to think study. we're see and [indiscernible] oxalate data, think from what X the for moving Phase it's thinking about around that study, we're

Tolga Tanguler

Yes.

So thanks question. the for

is large oxalate. globally in lumar study the numbering is a what a formation millions stone population recurrence urinary see ongoing reduction we want probably. to So the very obviously in is And

is testing. And saw to we urinary an plus Now very in liver. have GOX This oxalate, we're the leads in pHX percent population, of oxylate. targeting they of urinary hypothesis in reduction enzymatic course, defect such XX the a the reductions

reduction achieve a that the is not things One see like may to of need to interesting levels we in events. stone

say thing look levels becomes the next oxalate it stone is once let's important that as when out and in saturating formation that's data so the see. occurs. year And the I at comes And urine because

by the prevent not may to it modest get saturation of it take margin you need might formation. relatively range to some all that way down need to back just you So stone out to super normal,

lots so that PHX. safe we find here, but seeing also Luma that PHX opportunity me population. optimistic And to to the looks exciting far with we'll population, do we're drug but approved RF with important will reminds get an a data, as be so of we've with seen is several and And very this what work just more very safety the update you in

Christine Lindenboom

our our pipeline, Thanks. patients orientates exciting more Actually Alnylam. an around common think, much seizure, another program potential is for which with development in I

question, Next please.

Operator

you. Thank

question next with Blair. Our Myles William from comes Minter

You may proceed.

Myles Minter

Thanks question. the for taking

on and we've around how you fitusiran is pivotal Just the receptor guess you relates And enroll I study? a a seen sitting that Thanks. that types sort patients [indiscernible] consideration inhibitor Regeneron? pivotal from some in study and of complement that therapy discuss where just endothelin-X can you it the to data the for data, the of key like in property see potentially of methodology how guess and that would would the lines the And I antagonist? design they

Yvonne Greenstreet

fitusiran yes, great. I I Alnylam. patient common with of program were reiterating by kind very And the XX% mean with think property, of think So X we reduction that quite really I potential Phase I for in condition, X start another pleased a results few questions really this it's think Phase off is just there. [indiscernible] [indiscernible],

to forward in more thinking steps. specific working there questions how but next Actually, very I think I program. around So partners that the our with we're there, much be enough pleased think about moving we're Regeneron were

Tolga Tanguler

urinary is we're Phase glomerular of study year IGA ICAN kidney and kicking we don't in damages that and and the are the study and blood looking Regeneron the compliment a now, activated sort occur result of compliment end. I dynamics proteinuria. that here reduction XX% program immune hopefully X the fundamental then Yes, disease, is glomerulus. forward how understands pathology lead says the we or in with change through right where engaging Busy the aspect anyone thing approaches specifics interesting excited working complement the really with the the regulatory The [indiscernible] to the this take are complex very activate sit, this design busily about in off flow in by authorities underlying proteinuria think basement compliment is An results. membrane hyperfiltration anti additional of very

factors. orthogonally two we So the preliminary shared. impact here we've immunopathology the to pathogenic we've results With anti-compliment key we opportunity can seen fundamental have approaches at get the underlying and

a that antagonist, that. injection. have situation will would It future three that being could like cetera. once what for months to where every blood great endothelin then anti-compliment role, cemdisiran a a be in ACE will type foundational I will polypharmacy inhibitors, there up a month, in be things inhibitors, so anti-inflammatory et alter a addition And flow, ACE once So end fit in be this believe, approaches the

for if results to enthusiastically have [indiscernible] the the the from drugs [indiscernible] work what side And of anti-complement see Thank there will and although they seizures, more but antihypertensive we'll question. And if become so see with both nonspecific classes, also and up open drugs type other the drugs agents just there, the these add for more you One general favorable rather question. part so up combined. will do is moving of needs effects. are you potential cemdisiran, picture, we're steroids And to being forward here. patients

please. before we close. more think one I have we question question, Last

Operator

you. Thank

comes with Cantor. Our Brayer Olivia last question from

You may proceed.

Olivia Brayer

a any patients are are Hey, to period APOLLO-B, in anything XX-month from mark know good [indiscernible]? a after study? you I I to AMVUTTRA that with morning access guys. that phenotype there you. or Thanks sequencing can requirements quiet on for follow-up Is but could then if are on there this And Thank the patients. after I've patients built wanted you question. got ask move for reach improve mixed respect to monitoring that do in ONPATTRO

Yvonne Greenstreet

taking we're so but to I I in actually be period, you your not question. going didn't out say, are quite to want catch just APOLLO-B, quiet on second questions pointed any we a So

was repeat Could it AMVUTTRA, the the with I think you it I but part to of properly. didn't hear something second question, question? the do

Olivia Brayer

or just anything AMVUTTRA Yes, And [indiscernible]? patients, you is guys payer improve patients ONPATTRO do mixed can access onto right. for to about sure. after It's whether there that there sequence phenotype could

Yvonne Greenstreet

and neuropathy phenotype mixed plug patients? mean You

Olivia Brayer

Yes.

Tolga Tanguler

patients. Yes, right I been that certainly will help those mean, access. pricing, fact the look, parity at certainly capabilities position this to believe The we've so to have we support increase able we

indicated any So headwinds for for haven't around the polyneuropathy had piece. the we heard far and is really U.S. in we indicated But are access cardiomyopathy. tafamidis

So great if support strategy not access of providing have consider. access for we support, new brief we would be they Like through terms be and our patient service would in any benefit would patients any our those patients, something of verification program. of which go eligible patient certainly

Christine Lindenboom

Thanks, Tolga.

Yvonne Greenstreet

Okay.

joining our we've that development, first coming diversified and very with and and of on exciting we've got everyone We've advanced you the progress delivered of on catalysts number So XXXX. half programs thank a We're commercial in us results. second We've call. for made deck the this quarter in months. the pipeline happy strong

great and deliver you long near forward on everyone. look updating And So our we while have Thanks goals. way a continue along the we to to day.

Christine Lindenboom

Goodbye.

Operator

Thank call. you Thank you. for conference This participating. concludes today's

You may disconnect. now

Alnylam Pharmaceuticals (ALNY) 28 Jul 222022 Q2 Earnings call transcript