ALNY - Earnings call transcript Q3 2019 | Alnylam Pharmaceuticals, Inc.

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Operator

Ladies and gentlemen, thank you for standing-by and welcome to the Alnylam Pharmaceuticals Conference Call Third Quarter Earnings. [Operator Instructions] Please be advised that this call is being taped at the company’s request.

I would now like to turn the call over to the company. Please go ahead.

Christine Lindenboom

Good morning. President Relations Vice at Corporate Lindenboom, Alnylam. Christine of and Investor Communications I’m on John R&D; phone Officer; me Greenstreet, the With today Officer. are Financial and President; Vaishnaw, Chief Operating Chief Barry Akshay Maraganore, President Poulton, of Jeff Officer; Yvonne Greene, Executive Chief

you conference www.alnylam.com. Investor the those via our slides website, available call, page that to of For and by can going the are webcast also participating via be accessed of

and review will During general today’s our in open outlined will and some provide brief call an context. Jeff provide call, and your Yvonne remarks provide Slide X, Barry we a financials, the for updates, summary provide commercial introductory John review progress, recent as update upcoming preclinical of our before questions. will will on Akshay clinical will milestones

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

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

call I’ll turn the With to over John. that,

John Maraganore

call. with joining call Bear a as you me I’m for everyone Thanks nursing thank the Christine and today.

one with with R&D Barry progress, Akshay late-stage of organic period are pipeline our R&D side, number our start me the XXXX, by those in will progress strong into our the but steady few of commercial and progression, on as Alnylam’s and continuous productivity into high programs. head growth as growth and details by patient and we we get execution revenue entering comments. as robust a review providing will As marked a months commercial of evidenced very important mark large by let the well level history, final

having anniversary over drug to patients of launch, with year the very continue ONPATTRO and XXX end we be the with of at pleased on now First, ONPATTRO’s uptake third the passed one approval commercial quarter.

in from believe patient We’re patient as driver additional we differentiation. new continue coming even activities, revenue our growth growth Japan seeing of finding forward leading commercial in to to rates and existing going also continuing will expansion a launches existing now new markets in global with markets be competition such providing markets, diagnosis, Canada. recent of and with And and further improved along generation evidence this

growth very vutrisiran for and We’re Alnylam. real bodes amyloidosis we and awareness that believe ONPATTRO, well of also seeing hATTR

launch hard at entry also world’s are potential therapeutic We upcoming the preparing second givosiran, at for the of assuming reviews, of to positive regulatory work commercial markets. the the RNAi marking

make second is the I side to want here. The point R&D on

we’ll lumasiran readout bring an support they the company And to patients. the innovative medicines of global the end Here, year. up additional that inclisiran, Phase product gearing by to for filings at the with with for program we’re partners regulatory efforts our X prepare by as

TTR believe X we silencer large a amyloidosis ATTR development patisiran into of advance unlock to settings, our very the wild-type RNAi In ongoing with cardiomyopathy opportunity, believe what a we continued best-in-class our addition, vutrisiran approach have the and mechanism Phase action. commercial to we hereditary activities and with potential is for which

upcoming forward across infection, robust on to disease. these some also R&D New hypertension, data Our updates of at as November programs stage We programs clinical diseases, clinical earlier as initial HBV well liver as our and look York XX additional pipeline in sharing includes from product such City. Day complement-mediated areas alpha-X in our chronic portfolio

near-term make believe company The and believe and XXXX to profile platform I’d engine we on to is hard exceeds robust line strategy a our for bolster to and multi-product accomplishments of R&D point commercial and a on our toward fuel becoming global industry like pipeline is that and in medicines our norms. innovative we our growth based modular, we goals biotech. importantly with of deep believe a match, if for have sustainable investment reasons product where, achieved biopharma Alnylam organic sight prospects, reproducible clinical that many ever achieving a continued innovation, third rarely, Furthermore, we return a clear very to

years. through grow our innovative RNAi now and strategy this we recognize deeply will the attractive growth, support Jeff self-sustainable our CFO, generate are for that balancing path have in a Alnylam coming with for of eager in is for future. investment continued revenue the pipeline are helping something our is in focused that on launched deliver and as on promise ever than greater our the the to we therapeutics. to this we stakeholders with to new team time, how capability hear This management profile commercial the leverageable And ability global to RNAi At the first continuous transition confidence growth of and some towards strategy revenue built committed us Alnylam and having Poulton, the therapeutic financial having navigate and about same our potential our a

Alnylam’s the who to the last Alnylam top This note one received X,XXX recognition science week’s the culture recognition best announcement by industry. want as employer. that is as I a remarkable by make of in industry’s respondents number identified Finally, over

work this innovation to game every all and be proud. of Alnylam tirelessly the employees be to more We couldn’t are proud continue bring who recognition so to to potentially day more grateful patients. We changing

it Barry? progress to commercial detail. Barry more over With that, now review to I’ll turn in our

Barry Greene

everyone. Thanks, John morning, and good

into to on Before comment get I’d dynamic. I details, market ATTR broader specific like the

Thanks we’re our to and and the field. own previously, in stronger all diagnosis seeing commented stronger in disease on physician part patient in efforts of and as others the As we awareness anticipated efforts specialties. across

patient We’re seeing Alnylam evidence and market well prescribers. as growth as new expansion samples of this in Act

is important for So vutrisiran this John with important large as for this think commented, we disease. at is very and this and patients ONPATTRO, growth patients

Now performance. moving quarter. million let We achieved specifics, onto by net ONPATTRO’s third begin reviewing global $XX.X revenues some the commercial ONPATTRO in me product in

XX% terms from we In representing world, growth international achieved U.S., and of split, U.S. the geographic the $XX.X million the of quarter-on-quarter from the representing $XX.X growth. million XX% quarter-on-quarter rest

patients ONPATTRO treated clinical to with number X,XXX XXX of commercial, increases treatment. that patients our patients by achieve we worldwide on we’re and approximately an in access and of continue ONPATTRO. programs year, commercial believe this And incredibly across Now over XX, efforts. number end clinical include expanded access being milestone the as expanded to trials were that to to exciting in trials were on September patients worldwide overall XXX that ONPATTRO track we our expand receiving When global approximately

not It’s of see patients important seasonal to note peak new starts that season. slowdown quarter, for vacation to many uncommon drugs to third during a it’s the

revenue In very and saw We’re expanded we’re quite number especially through actually and competition investigational increasing a pleased for to this market we demand see steady large recent overall growth. from given ONPATTRO pleased of drugs and continuous trials. availability some entrance of the despite with access programs patients clinical seasonality, and potential the that

U.S. get dynamics. market Let into of review more a with me the specifics

the in repeat continued as well On the prescribers. number new both of physician we’re front, seeing prescribers growth as

well. XX% In believe by efforts fueled our fact, ATT from increases this education in came prescribers, that received medical in third awareness is and We U.S. players over that of working are encouraging state forms the quarter will start statistic as dynamic engaged disease continue education. multiple new evidenced disease

the from about XX% we XX% neurologist, the coming saw of forms start good of heme/onc. submitted specialties third were Regarding quarter prescribing, from mix cardiologist. and prescribers, of a mix U.S. in like other about in And

in down July we’re see the we encouraged cardiologists while during previous start August and returned this XX% proportion and September the quarters, the of cardiology current the range by percentage continued did part in the Now from very quarter. from trend was forms bit initial of to a

multidisciplinary of in and emergence more these excellence the key the diagnosis. of a and are referrals across We’re dynamic also of and country more seeing earlier proper kinds centers

in third stabilizers, is of positive with hereditary ONPATTRO saw ATTR for TTR concomitant evidence which ONPATTRO, manifestations the with patients amyloidosis. of of in branded U.S. reimbursement the quarter, with we use note the of beginning Of the multiple

use concomitant to with results as increase expect over ATTs We experienced favorable time for ONPATTRO. reimbursement

over year Now the rates. of that one comment launch we first to have on it’s time for possible adherence experience,

that adherence very is remains with the the overall to The news X Phase therapy data. here APOLLO strong good consistent

from hear XX% patient from hear with estimated commercial over rate believe with hub ONPATTRO a adherence we outstanding consistent we Specifically, that is ONPATTRO, our rate field. the from for that favorable and patient we experience and that we reports

that and headwinds than launches. payer XX% with Even reports, Regarding pleased reported confirmed U.S. continue to often more other orphan now we an reported government avoided by have ONPATTRO. U.S. effectively to we’re external market access as for the Medicare, competitive increasingly access landscape, groups, U.S. It’s commercial, including coverage prescribed very we in Medicaid across partner and paying lives payer with have other drug VA. of

access adapted and proactive community. result use this of including in with U.S. complex approach based payer agreements very proud constructive very the environment believe We’re we’ve it market a value of collaborative reflects the and

Now of the turning world. to the rest

performance. ONPATTRO with pleased also We’re

quarter revenues first a thrilled A ONPATTRO in Asia achieved We Japan As launch to Japan. sales the are and international major our have I and be Japan population to of in team and built to in was product during noted earlier, underserved we in achieved achievement the $XX.X million more significantly in broadly. net QX. bringing ONPATTRO our

country U.S. revenue that mentioned, previously drug to second the XXXX. for be we our exiting As on patients largest is and ONPATTRO Japan we anticipate likely after

quarter, Belgium, the achievement reimbursement ONPATTRO achievements includes United in notable Germany. Canada, launch and of the Kingdom, Other the of during in

ONPATTRO direct patients United access, the countries over reimbursement in being through outside paid or now States. sold have Now named we XX sales

first-line international source We highlighting are treatment tafamidis seeing both and value of are the people ONPATTRO. business from that the switches with coming our seeing

the Improved faster. committed their ATTR patients and course, When read remains diagnosis polyneuropathy help disease challenge in improves improving patients earlier also receive treatment medical diagnosis patients. education in disease treatment addressing team our Globally, will raising overall awareness options of prognosis. the to their amyloidosis it

patients Canada highlighted third-party in suspected ATTR of a Regarding Act testing and the at Alnylam diagnosis program of facilitating our amyloidosis. is we’ve previously, genetic having and as patient diagnosis United States aimed initiative

of October, this increase awareness. which Of in TTR quarter late of in improvement X,XXX reflects the note, number requested quarter. have an previously submitted for disease over positive believe the to from samples tests seeing nearly We increase over per we’re As X,XXX tested of X,XXX out of in tests pathogenic about new mutation. XX,XXX

patients Alnylam where a be several genetically As methods can tested. one reminder, of Act is just

– in So the as we for see testing. an earlier, growth overall we market genetic commented

to continue customers TTR of about variant learn XXandMe with the we Act, common more partner United most the States. In to risk Alnylam genetics addition in service to genetic the their of consumer help three

competitive and enthusiastic steadily our about commercial summary, in generating we’re will In the more incredibly new access and by an awareness we continue efforts, and of very diagnosis in approval broader with ATTR the of patients. diagnosis. evidence improving the patient with more countries, will features competitors achieving benefits in overall encouraged this continued disease with finding to with environment. differentiating were and to market, accelerate ONPATTRO, highlighting confer And believe progress addition ONPATTRO And

review let to and European now under is which givosiran, Finally, regulators. U.S. both me turn by

capabilities the we following meantime, decisions positive country. regions will best launch by developed for both we’re these ONPATTRO built in early launch In expect and practice the givosiran country in Assuming from the XXXX. leveraging agencies,

our and At HCP the in awareness the is patient porphyria communities. acute diagnosis this improving AHP or team on hepatic focused in stage, of

our HCPs their physician of As accurate at and part through to recognize the of overall give materials help navigate to an patients educational with about and launched content tools, websites them and help symptoms resources tests patient-facing signs we've provide AHP diagnosis. the appropriate and and and arrive these efforts, to them AHP disease

Alnylam we gene associated U.S., to sponsor mutation Through AHP. carry for Canada, be access Act, or the known who testing may individuals with in to genetic third-party

very a mutations AHP XXX early as overall and program positive this with we XX% rate. tests hit accounting for can an for report mid XX patients AHP October, stage, is in While of still submitted

collaboration are gastroenterologist. among with seen of focused We're And the the GI during patient, so also very and a on announce around GIs frequently Pharmaceuticals pleased quarter for AHP approved, AHP goes disease in the diagnostic significant medical to of education AHP. if Ironwood the one with deep expertise groups U.S. an and patients promotional most education. U.S. Ironwood’s third represents diagnosis specialty with expand to relationships leveraging journey community, opportunity

of and been prevalence roughly AHP consensus Europe Turning are to in in market, estimated for is people It's the systemic affected the diagnosed severely attacks. per systematic patients two recurrent a with disease. experience estimated that the of X,XXX five or addressable to and there global range were U.S. XXX,XXX

to Of attacks symptoms impaired course, disease many and more additional patients with have have and sporadic life. estimated more quality chronic active of

patients challenging many disease the potentially is we estimate Nevertheless, about with attacks. are Europe patients to having so patients those with with diagnosed active of around remain diagnose, in AHP frequent were debilitating and currently X,XXX life a that with disease active X,XXX threatening there undiagnosed. attack, more U.S.

treatment Assuming Givosiran. very associated patients option we're positive new commercial reviews, we to can with and regulatory bring the for excited the opportunity

delivering to this next possibility the We to year. forward of look early medicine patients AHP

revenue opportunity peak it's with potential. belief the $XXX in can past, ultra-orphan in said our million an that this global we've over disease be As attractive

to launch. ultra-orphan serious treatments observe underdiagnosed genetic to We diseases. show after Similar with other pattern and new that Givosiran expect growth in

that, turn progress. with Akshay to now me our let and over to Akshay? R&D it recent So review pipeline

Akshay Vaishnaw

Thank In the on to morning, good limit Barry, our remarks going you, interest multiple programs. and Phase time, clinical prepared III everyone. of my I'm

is patisiran, which unbranded for Let name the me start with ONPATTRO.

to on patient an in settings. the seek the key the positive, in the Enrollment of was expanding the the of initiation patisiran label third is the ONPATTRO achievements and APOLLO-B Phase inherited aimed and study expanded cardiomyopathy for our clinical timeframe. regulatory wild-type study One include X both label approval during amyloidosis to quarter if the at approximately we ATTR to plan way is for XXXX XXXX

September, As HELIOS-B. you ongoing Phase RNAi of an in study. the quarterly for X also delivered to subcutaneous in know, our design injection we investigational therapeutic, And by and enrolling hATTR We've amyloidosis. development vutrisiran, advancing in is with which announce RNAi that polyneuropathy HELIOS-A were also Roundtable we're ATTR been pleased the patients

XXX Patients in of amyloidosis HELIOS-B the and maybe receive either heart disease inherited study one-to-one plus of ATTR Patients the include will will be designed cardiomyopathy. with at with vutrisiran amyloidosis ATTR X to patients patients time up is study of NT-proBNP either to once hereditary history or criteria distance total symptomatic of cardiomyopathy. tafamidis approximately and study months. on be failure a with Phase randomized three wild-type will Association levels Eligible at XX% The enrolled a population be every with randomization. have less New medical to or equal meet patients for minimum subcutaneously than placebo and of and and or York vutrisiran or walk Heart milligrams baseline. will administered three six-minute to wild-type XX

study Primary hospitalizations, month assessed mortality endpoint composite of a outcome of the at all-cause which will is XX. be in cardiovascular

HELIOS-B year. we comprehensive and optional We're on initiate and test, cardiac outcomes for study including If NT-proBNP. end also through with very of with endpoints study walk Secondary the of The the successful, of the to enter six-minute providing to a assessment analysis market opportunity quality the global an working will large startup include includes clinical burden disease life, interim imaging by readout. ATTR study allow should earlier design data. assessment wild-type now the an opportunity activities remain track vutrisiran actively

to I’ll RNAi progress recent for of hyperoxaluria now with type in an X treatment therapeutic lumasiran, turn development primary or the PHX. investigational

older or know, age in randomized, we're you Phase change is month lumasiran. six. from study excretion This across three endpoint moderate renal in PHX patients oxalate X study primary to of placebo-controlled with six conducting ILLUMINATE-A a The As double-blind to average baseline impairment. the is urinary of percent now mild

soon of for and beginning ages. PHX lumasiran and of also severe X if late ILLUMINATE-A submit patients We're trial top patients Phase a earlier moderate Phase age start also to and XXXX, on enrollment impairment study with report early positive in ILLUMINATE-B, in in to in the in trial of all remain we the X conducting impairment mild renal to lumasiran ILLUMINATE-C with completed pivotal third results six the PHX in XXXX. regulatory renal year under plan line we in We filings from track this

and and three to secondary have Medicines met the inclisiran XX demonstrating partners nine profile. from results remarkable efficacy Phase Sanofi studies. and their been X line top from of endpoints subcutaneously complete partnered months. results our assets, X and All an Medicines assets. our report made Phase at around also III Company Phase primary advancing drug study Company at addition administered also wholly-owned the six the all studies every for safety The now In The progress with The we've ORION-XX late-stage results our once excellent demonstrated

provides therapeutics? RNAi the date systematic there for suggesting our no did what safety of Alnylam largest therapeutics demonstration mean results is platform support these evidence First, platform for results specific and RNAi to that safety So these signal.

Phase were in so heterozygous a population and this Importantly, studies and stringent is evaluation the the X generally of conducted patients, ASCVD ORION safety FH of ill tolerability.

our HBV. disorders therapeutics a RNAi Secondly, opportunities medicine attractive prevalent believe an of these of disease. results administered strengthen very Moreover, NASH conviction we like creates prevalent diseases and future highly such in common infectious of dyslipidemias, as other greatly as pharmacology the hypertension, for for therapeutics profile treatment in the the highly infrequently RNAi chronic

on positive will The significant timely Company by royalties review Alnylam revenues in global have Finally, up for inclisiran provide of assuming source just of due regulators the to and product. submissions term near Medicines another sales to regulatory of XX%

later look on we'll on mentioned update to time, much John pipeline to at our R&D this seeing at As programs. month which to and excited of very Day you you you excited the entirety our be forward earlier, we we're -- update

that, turn to call our to review me now the Jeff over With Jeff? let financials.

Jeff Poulton

press our summary of overview to this quarter for of provide XXXX would results, release our everyone. of results for issued good I a and and to a brief areas, our Please XXXX. our financial quarter three Akshay, cash third opportunity key to like our financial third guidance. take of summary P&L earlier XXXX refer the Thanks, morning, today balance

third start revenue. were $XX.X million. with XXXX quarter our me quarter revenues Let Total third

Barry growth $XX.X the from XXXX, second product As during earlier, highlighted of quarter product net third ONPATTRO recorded quarter global global revenues XX% we of million. which represents of net $XX.X revenues

and were revenues both The generated driven the U.S. first representing product new quarterly international second onto were reimbursement million, quarter product finalization and XX% Japan, the growth million, representing recent pricing and addition of time including ONPATTRO. revenues growth. primarily for patients in the by XX% quarterly of UK increases which $XX.X following were from for revenue therapy $XX.X

Our remains high favorably by impacted That sold zero XX% to in of line into Cost of mid-XXs the net global ONPATTRO $X.X was inventory. result cost teens gross-to-net approval. mid was through prior percentage third quarter, cost expense the our in of ONPATTRO goods approximately is in of expected the an is of after as percentage a to for sales. the which still R&D XXXX, XXXX. is ONPATTRO which being depletion guidance. We product goods sold the third prior first quarter regulatory the with zero million anticipate cost inventory, This of increasing half

collaboration during quarter We the during million quarter recognize million revenue third $XX to year. of prior as third of $X.X the XXXX, compared

$XX.X increase the is in due revenue in the agreement Regeneron, which generated our expected, collaboration primarily million As to with quarter. collaboration of revenue

and increase to non-GAAP in in compared Non-GAAP R&D in expenses $XX.X as of the as million programs. due expenses Moving of expenses to XXXX. R&D our $XXX.X expenses. for operating compared million was pipeline the increased quarter million quarter to activity primarily R&D to The our prior XXXX, support $XXX.X year. late-stage million $XXX.X were for were the third GAAP of costs third

launch were in GAAP quarter for million compared for year. XXXX. non-GAAP was Turning as to in SG&A in with compared expenses XXXX, increase to third for as $XXX.X continued million of medical primarily expenses due global were the to affairs an connection third and million ONPATTRO. prior investment million to increase The of commercial the SG&A $XX.X Non-GAAP $XXX.X $XX.X SG&A expenses of the SG&A. the quarter

our guidance. a of Moving balance now summary to XXXX in our cash financial

the to with on end balance remains strong support on $X.XX investments operating years equivalents, $X.XX of multiple current the sheet of securities cash restricted based billion XXXX. balance hand debt plans. the This third cash for at billion quarter, end is compared and of to at as marketable expected cash, company Our operations

our of to guidance to We range million guidance. XXXX SG&A in million and lower million. SG&A million of will reflecting our be R&D toward XXXX organization. expense $XXX in both our reaffirming Now expect non-GAAP expense our XXXX good do $XXX to our be to the annual results $XXX the cost non-GAAP R&D guidance annual guidance the to financial of range within actual be end the We're $XXX discipline management ranges, non-GAAP of and non-GAAP

month, transition financial to to on R&D to Finally, I when later Day also our a on have aims the the will I opportunity some profile this how look toward perspectives for future. the forward provide Alnylam self-sustainable

With remainder that, over year. Yvonne? now to call to turn the our the of the goals Yvonne review I'll for

Yvonne Greenstreet

Thanks, look Jeff. but of in to XXXX, We've made milestones tremendous we progress so remainder exciting number far a of year. the have the forward still in to

XXXX. our global and cardiomyopathy amyloidosis APOLLO-B plan we patients starters, plan our underway positive ATTR for continue the study trial cemdisiran, HELIOS-B, launch of continue is the with outcome months, Phase and we potential our With vutrisiran, to reviews. HELIOS-A focus study. With to coming Phase enrolling throughout also the second enrolling planning initiate X the on expect regulatory therapeutic the in in X year an commercialization of late assuming with to RNAi For in ONPATTRO

in we Phase X ILLUMINATE-A the to late report in results lumasiran, to Turning study top XXXX. line plan

Company the renal ILLUMINATE-C also also and seeing patients an plan severe in look in to continue The with plan patients XXXX. in submit being inclisiran, With Heart will the year. for XX of inclisiran XX to from the forward Association late the American November present study We complete all to PHX that They at we studies impairment enrolling the held in pediatric meeting results to XX Medicines plans by and to NDA ILLUMINATE-B of ORION-X ages end Philadelphia. start

and our as advancing the as plan you of particular, stage throughout preclinical highlights for plan November of at this XX. expect mid-stage and R&D these York data program. continue efforts in our city to very milestones much to we reviewing look early reminder, exciting of year clinical a clinical as our In efforts And New rest Day finally, pipeline And the Friday, our they well forward to readouts as all of of course, a on occur. we earlier should number

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

Christine Lindenboom

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

Operator

now from Thank take you. go We Lebowitz [Operator Instructions] David our first will ahead. question of Morgan Stanley. Please

Your open. line is

David Lebowitz

much Thank the you curious what comfort the do gain perhaps quarter you sales. into very you has for maybe fourth ONPATTRO going question. in and well XXXX? And taking pretty guidance give When forward some of clearly expect you for ramp I'm my gone gate. enough to out think might

John Maraganore

a Dave. handle do you want great question, that's that? Yes, Jeff, to

Jeff Poulton

plan we're talking would come. not call. not guidance end that But is guidance to again, the year or internally. be the you next and for something I this we're question. year. would the say today yet. thinking an the It's about quarter about, fourth Understand to Yes. We're on about more give prepared provide that for be ready thinking And something to would answer

David Lebowitz

dynamic with deal going you How same being the for one, so if APOLLO-B ONPATTRO, additional to recruiting both the are are the with I one and the HELIOS-B patients time? you that. at Thank trials And may of for similar.

John Maraganore

that? handle Yes, Dave. great want you that's a do question, to Akshay,

Akshay Vaishnaw

the the up in we the I enrolling One which well APOLLO-B the second courses remarkably is, the not and prevalent end access are running of therapy of remember population ATTR study would there HELIOS-B, are advanced and parts have is of today. say two is intend predominant factors. world, in to is population that to cardiomyopathy most – And both year. and mean, of wild-type I that start Yes. does got by

enrollment relationships such, operations of to we're terms in great as readouts. achieve group working studies without get pretty we're of all goals both comfortable then experiences sites, speedy then we clinical And and extensive that through with TTR have can that for so and And that.

David Lebowitz

for my Thanks questions. taking

John Maraganore

you, Thank Great. Dave.

Operator

We'll next now our Please Matteis ahead. from on to of question Paul move Stifel. go

open. is line Your

Paul Matteis

financial great. Okay, clinical Thanks question One much. one if may. so I question, and

There's and in at your be up. on Can having expectation in guess a you're multiple surrounding curious expenses streams, inclisiran. the that. or what about cash any least next internal monetizing flow word in are expecting likely on qualitatively royalty couple to I And go thoughts continue years, Just of for was And commentary separately years? on are allowing stabilizer. we a there cash patients you cash surrounding regarding that trial, who then coming who HELIOS-B your for talk the operation for about to noticed the discussion you on where over not Can study, assumptions stabilizer, you're Thanks the mix I and are in on a are how patients? stabilizer that on stabilizer you're the the perspective? much. so a assuming top benefit contemplating words, how you from much powering In naive versus other of like

John Maraganore

great you that? some context to an to starters, Jeff, answer do or want Maybe for questions, Paul. Two give

Jeff Poulton

me sort we mean, said. Yes. I let just restate what of

We're the year feel guidance which billion not next have third today the we in the We cash years. of for following very quarter, about. $X.XX end at or providing good specific

can focused I on more this It's are Day. about a lot that that thing financial sustainability. we our very of we that question internally. one about say that talk the think I is I'll at talk something R&D

to the going believe think one in say our we're year. that we net that peak regard prepared XXXX is I is to be loss thing that

cash That's improvement about asked all so say obviously the the is impacting of you that which on we'll I'm runway. question next year this years, sort that in and about the to following today. see prepared in And

John Maraganore

And you want to question? comment briefly on the the part inclisiran question of –

Jeff Poulton

question. it's think Yes. fair I a mean, I

possibility about do I very thing about again, possible comment most we we're would right profile we're and the very, But that think potential if think of the value get there. about would approved excited And excited that monetization the I we very a fact the I important that, of profile for looking just the it. drug, think consider is that of at that or the to on drug.

John Maraganore

to clinical Akshay, And want do Terrific. Yes. question? handle the you

Akshay Vaishnaw

Yes. With question, respect to the Paul. tafamidis

obviously of We up. hATTR. world have families is out around majority settings. the the the wild-type not that acknowledge certain to have there we to available ramping think vast think It disease and patients. I It's still in with

the have study. So such, to enroll I really an placebo-controlled as robust opportunity think we

Now have comparison. primary designed in comparisons XX% having said up allow have that, the acknowledge to But some background. to into we patients will tafamidis those that that the study and The to assure in the placebo-control be is of onto are everybody may patients I I that enable tafamidis and can patients and study. concomitant think,

and And it. the so as the such, powered we're we've way comfortable with trial infrastructure the of

we can analysis vetted do extensively over has efficacy this And So vutrisiran been demonstrate, key of course, with regulators. and hopefully that design very of placebo.

John Maraganore

that Does your Paul? answer question,

Paul Matteis

Yes, questions. I taking it. for absolutely. Thank appreciate my

Operator

move Jaffray. will now Piper go from on Ted of We question ahead. Tenthoff Please next to our

Your open. line is

Ted Tenthoff

Great. everybody. you And in achieved much. the Happy the you've pretty year. what Thank launch with remarkable very Halloween

because still going you me if minute, and I up really wanted that. be be treating I still we're may, enrolling, for don't it's look to by to off hats So therapy. Getting givosiran incredible totally a patients execution understand patients to – I almost to disease. have patient to with to need for of who this a I'm how really X,XXX towards this kind

question. to first us the givosiran term getting what and answering approval walk longer like Thanks women used. the prophylactic So anticipate maybe can sort through could maybe look of of maybe you need of of you how sort how might sort be then for kind of patients who

John Maraganore

Ted. question, that? you do to Akshay, Great want answer Yes.

Akshay Vaishnaw

Oh, of XX, sure in between disease is we've so of of prime you men and Ted, I'm familiar, terrible to discussed past a are to the disease, of but life. many Yes. prime one, years life. the a this my women generally women, striking maybe XX I'm I'm XX often of that to nine as before,

patients the your up course, attack terrible striking and abdominal nervous very XX peripheral the that for and from disorders means people It or and me? constant them. It be you can given or respect this end fact, XX, threatening life and first life, attacks. They year. this should the going leave issue in year. one had leave the is can a in patients pain, the someone every attack or neuropathy. studying there's can five wrongly, one do die patients disease years. attack, that of a be any the is coming? be It be Or it laparotomies to remarkably when fact next disability you've prime So And in the from can Once with neuropathic they of the unfortunately that present out that when many fear. have with the to disease or be impact for not life. working for is can that first subsequent And How's necessarily this having of is attack end enjoying

multiple year of And think for this how average to physicians enriched so a have patients who was And take manage to dilemma I attacks study get four terrible these demonstrate are as the the designed we patients. remarkable attack think rate, so rate unequivocally I a that, the or design to reduction the in XX% or ENVISION more. we And study. in on annualize attack median in

I important label And right prevent attacks. drug property to from fundamental FDA this has powerful drug a prevent the and and the that of think very providing new because, the shows very to the will is have new approved obviously all think way and they'll have do that physicians And make these patients. to now. attacks we're we ongoing I work once to EMA, treatment that So choice options a but

Ted Tenthoff

forward I'm in New you Awesome. a seeing I to agree. looking York in Thanks. weeks. of couple just

Operator

now Baral We will our of Please go take Cowen. Ritu question next ahead. from

open. is line Your

Ritu Baral

little ask QX. in want to Thanks what competition of patients Good impacted, what of suppose prescribers I I most you in tafamidis. morning, what sort about or And everyone. from the field were the sort question. seeing for the bit wise were taking a

new the dip in was in think reason you the that the landscape? that do had in cardiologists? competitive And why you prescribing proportion what of you rebound? seeing commercial are that breakout the Europe you physician Specifically, saw And

John Maraganore

Barry, do question, want Thanks Ritu. Great you handle for it? to that.

Barry Greene

Yes.

mix Europe talk we and me of and Let types. specific about then kind the backup can physician to

on United no hundreds very very drug evolve to always ONPATTRO busy And in the the with available. for for them a and probably associated prescribers we we number first the believe the There's and States starts longer flows comment drugs were in time cardiologists, and we getting wild-type with So base of time. fact proportionate accounting third was patients cardiologists, new new call, of the disease the in as and their ebbs pleased prescribing. in over hundreds we where expect were quarter patients the prescriber on

see specifically So others cardiology TTR in we cardiomyopathy. really talking about

But Now rate to we will commented return number about cardio. into of early summer October, the outside call, that and note XX%. of in as I prescriber September, in a of getting did we are cardiologist once types prescribing neuro the the got we I months through see additional sort

and those percentage will So flows. again, rates ebbs

and where center The amyloidosis. calls. other are multidisciplinary centers And more excellence around Ritu, about of more we're dynamic, the but for have and TTR specific approaches all cardiology being country, of referral previous don't popping seeing utilized. key in this being asked numbers, you've I up a pattern there

previously polyneuropathy. in Now been as approved know, you in Europe, has tafamidis

seen there is from at uptake to But we and primary is countries that the States. United what that So the wild-type pure seeing which with that's the and the we've or hearing interesting dynamic very reimbursed time. this getting cardiomyopathy experience are think outside field in be population, we're the we're

Ritu Baral

Are a well? centers And are the of prescribers, you new quick the Great. the community to in out they starting just as of sort see In excellence? that to filter follow-up.

Barry Greene

and and then to EAP and community diagnosing in back we're in beginning actually patients about more and more Physicians comfortable community, this on the the drug. known are site to and the dynamic treating. And More launch. we educated in patients as getting putting those the It's seeing. fact talked physicians it's of are settings, what returned patients

really versus of as of seeing the patients. stretch but the who we’re kind treating studies doing busy, pattern many remain centers in excellence, busy outside very clinical commercial So

John Maraganore

just I come as talked that's we've a quite of amyloidosis have they're The in of that clear. the or use the this Ritu, naive we're patients offline, are the I dynamic is to setting. want progressing it's dynamic, we two treatment to is when to realize in in U.S. uptake is points? can I and and for One But their continue market to to or patients get that that very concomitant And reimbursement add interesting Europe, with which occurrence saying And to physicians polyneuropathy is now the just other think that grow will in able TTR either ONPATTRO and hATTR of think Yes. in to continue other clarification ONPATTRO be the about on progressed stabilizers. the the U.S. tafamidis. back on ONPATTRO type in

Ritu Baral

in my queue questions. with other back hop I'll Great. the Thanks.

John Maraganore

Ritu. Thanks, Great.

Operator

We Goldman our move question of ahead. Please next onto go from Richter Salveen Sachs.

Salveen Richter

of what they're if you're physicians this this then were in seeing question. morning. further where and stabilizer then and Good Thanks for and patients also direction patients, to use perhaps, the subset? concomitant the a And details from and comment that you tafamidis patient provide you phenotype a mentioned there's specific Could silencer hearing for a between choosing on how occurring that's you my just the taking from just on type switching on are ONPATTRO mixed opposite point? if at the versus

John Maraganore

questions. Great Yes.

progressed happening these switching the in patients course, for that market and of tafamidis predominantly the their been clear, many, on with years has where many Just are neuropathy. the be to on have on that's tafamidis Europe, just

point Barry, switching set they're full of that time. But ONPATTRO in so the And at want answer to there? to question you

Barry Greene

Yes.

what the on continuing and – there's experience where physicians been patients tafamidis patients – diseases. So significant to have States, is experienced just progress a you very patients their important I said United think of with have dynamic. polyneuropathy Outside of

published In but rapidly Portugal patients of that benefit by out fact, a about one-third recently progressed. tafamidis, seem our paper patients Theresa to two-thirds of of

in with that So also patients we switches to rapid We're a That's seeing Japan polyneuropathy experience, are with dynamic. there in ONPATTRO. and Canada.

the hard a concomitant amount because anti-inflammatory diflunisal easy very less use, specific with to concomitant reimbursed. seen that for of In time. It's specific it's of and get on some use non-steroidal read be to generic terms we've it's

of States, patients really branded significant hereditary TTR importantly, relationships it and seeing John amyloidosis TTR ONPATTRO in reach and stabilizer we're polyneuropathy hereditary have mentioned But may have the cardiomyopathy their with very on with you speaks started physicians patients as in that polyneuropathy polyneuropathy worsens, amyloidosis know, And progress in the which to the were for had data. had strong as United the a of part as we we’ve treat payers. with

branded We reimbursement even are in of seeing of face products. the ONPATTRO concomitant

John Maraganore

worth so That's referring reading. paper who's the last And the mentioned is Teresa the paper, is I Coelho, he's without investigator Monteiro Teresa, to which name. in Barry her And Portugal. think

Salveen Richter

you. Thank

Operator

go our Wang move from ahead. to Please next now question on Barclays. will We Gena of

is line open. Your

Gena Wang

taking my questions. for you Thank

I trial is think One two question for questions. clinical HELIOS-B.

throughout baseline up allow do tafamidis to on along continue study. patients You and XX% the

powering mainly context entry allowed statistically asking generic of population? So in the assumption tafamidis in in detect significant Just benefit I'm patient is wondering, to the particular XXXX. this

John Maraganore

good Yes. question, hail do Gena. That's a you Akshay, want to that?

Akshay Vaishnaw

is I if the a is understood Yes, and question right, answer The placebo. Gena, the placebo-controlled no. vutrisiran comparison between comparison primary

not we're between combinations So powered those to differences show success.

Gena Wang

comparitor thoughts So forward in trial entry? of would going terms tafamidis the future design? generic address Any on how you active

John Maraganore

what We generic expect be cell the actually. mean, as time and about the of form VYNDAMAX real tafamidis. don't of an I Yes. to to Gena, I a of frame the open there will question VYNDAQEL first necessarily version new you're it's generic think talking to that given all, entry be name and

we think commercially dynamic think that there's through. I to there So need a navigate not we

Akshay Vaishnaw

just to I that. And would Yes. add

group, it a widely treat had is If the that population, As from to a missed are Monteiro And at TTR peripheral just much performed learning by that neuropathy tougher you whilst far hATTR. strong approaches as to didn't Barry studies. can't of outlined tafamidis we endpoint. in in result different have look as concern, Coelho We acknowledged. how the very a paper seem it compare primary across the component result

potent formally very silencing this optimistic enlightening ability approaches TTR not be to And cross to these I way should think patients. treat the draw Now of to help their comparison most remain we very not people again, as feasible, that have but primary from I'm the studies, own comparing that’s And are disease. vutrisiran that. conclusions that

Gena Wang

quick another question ask cash burn, a regarding Great. the And go by investors. lot because then to the I

soon become perspective. cost annual and should programs thoughts the SG&A burn So self-sustaining mostly on spend Alnylam How early-stage, we or close then expect company? late-stage a the any on $X to the billion,

John Maraganore

do Jeff, want you Yes. that? handle to

Jeff Poulton

Yes. We're specific date you today. give not to prepared a

something it's on focus a It's we're I talking us. about. lot think starting I earlier, think for we're journey. We that a that mentioned

peak going be is expect our We to loss net year. this that

next to it relates also generated approved. into XXXX. than things to as with both lot ONPATTRO what XXXX And anticipate line As the growth as lower operating is growth year, operating well going of terms in givosiran in if about on for a we expenses, top I of optimistic expense have we be

question. your this again able that on to anticipate, particular answers going that Again, invested side next we're this hopefully focus to So in be year. been what's leverage and built for year big I the SG&A for is a us.

forward. you'll moderating see going expense So growth

John Maraganore

our increase would important for that how out add plays also I obviously be to a obviously towards results high later add this have And And revenue our the very this around self-sustainability. will a royalties then positive results that very transition standpoint will in confidence, we yet that profile our lumasiran mix. positive December. And component that the another be and from of of year product overall will

have of the organic vutrisiran. in the Alnylam, call we promising you in new proprietary Sanofi their both when look royalties launches that – also but from guidance So that product also about staging we get, at

in the put that this will years nice self-sustainability. that the companies You transition we're assist towards got together, some be seeing all to we've very revenue to growth come going

Gena Wang

Thank much. you very

Operator

Please our We Cantor will Young Alethia question now from go ahead. of Fitzgerald. move next onto

Your line is open.

Alethia Young

that taking I maybe got the or no, for of I'm I my a seeing about bit guys, XX, in you're thanks you clip two. more Hey could question. got seven the there's one patients U.S. like as well about into just guess, little joking, basically the maybe the steady you as we clip, mean, are quarters, a patients. of since feel as. and six, go finding Europe? we next think adding I people same I Should clip talk

I on world the reflect of that ties that. might about of potentially wanted givosiran get And how to things? guess, how your just like I that then, to label you're pricing on that? be into and And you So thinking want kind potentially – broad temperature

John Maraganore

right, with want All and do Barry, start to one then… the you Right. first

Barry Greene

questions. for Thanks two Alethia. Yes. Thanks, That's the not the XX.

quarters continue to we previously, and we both patient in revenue to steady come. growth see and continuous As commented the

those growth physicians with really more opening three from more we And that finding new de be comfortable as already for markets continued to Canada launched. it's in commented, saw in It's patients and example. allowing generation, ONPATTRO and going And efforts well. novo where we've are It's Japan quarter this As markets, prescribing. areas. evidence comes then we've

see education the we market we've number with of polyneuropathy do talked about As being identified. commented and of and with our those patients as disease to others, awareness previously, efforts hereditary we continuing the grow

we how quarter-on-quarter the So a growth see that's continuing fashion. in steady continuous and

John Maraganore

your regarding saying, by just our be not let that givosiran start me question, learned experience Alethia last we Now label we from should soothsayers.

So review think should this want and where I X, at move date now and PDUFA of February don't But regulatory our point. in we to issue. we're is get I that's ahead that seize on our

Alethia Young

good. Sounds

Operator

go We’ll next now move ahead. Raycroft of Please onto Jefferies. from Maurice our question

line now Your open. is

Maurice Raycroft

Congrats taking thanks for my on everyone. progress the and question. Hi,

looking interim? going with ask be the in that? could And remind optional going how interim what that decide HELIOS-B. happen Can to do happen? about you Just to when for you're What to the And just could analysis there?

John Maraganore

Maurice. for do question? Terrific, Akshay, beginning. Great. to in want congratulations Thanks the the your you handle

Akshay Vaishnaw

The depends issue I that Yes. much through into I before share. get those enrolls. with some first how things how so little going quickly details now and we mean, the a are stuff premature it's think it interim to study to on work we want of

interim available For data ENVISION after going fashion the learned study from almost set full such because a the was we that became futile timely very a be to in occurred the at pace the example, the interim. enrollment

So here. there's a through work lot to

due in think we'll visit course at and back point. come I that

Maurice Raycroft

Got it.

for taking question. Thanks Okay. my

John Maraganore

Thanks, Maurice.

Operator

onto ahead. We'll go Please next of Whitney Ijem question now from our move Guggenheim.

Your line is open

Whitney Ijem

of follow-ups couple on Good morning, guys. A givosiran.

diagnosed in So first numbers patient gave, terms you I think U.S. and X,XXX Europe. you of the in the said

at Just I X,XXX to number a recall some double check, point. wanted

in rectify contrast kind you maybe I the attacks, those patients of frequency kind X,XXX and but who of broader breakout terms of of have versus So more I number? then compare X,XXX in And the frequent this numbers. the patients those one, the two misheard, guess, can

John Maraganore

Yes.

then and some add want might more. start me you Let to Barry,

As to to undiagnosed nail patients change the we numbers. diagnosed patients approval, believe sharpening recurrent been in you really There's closer and the we've had as the those the overall still can are pencils our Whitney, we've the get the prevalence of X,XXX no out and right patients patients. versus We imagine there. sporadic the that estimate the rates down X,XXX

those in believe a on active granular really number larger obviously get there disease and there the a world. we the attacks of patients those in X,XXX – Europe, that diagnosed, are U.S. then we of a the attack and recurrent U.S. in of are But those of or X,XXX X,XXX have are are patients. the as But rest total less Europe, patients which are the have total and four and of recurrent X,XXX more that X,XXX X,XXX

So the sporadic attack patients.

And actual else that's X,XXX anything than to add Now the it's X,XXX number. diagnosed believe But number real said always talk that? is Barry, still patients, to where we that that we the it goes. about number. we've when

Barry Greene

No.

I think you've covered it John. well,

Whitney Ijem

early have Thanks. you OLE, commented the Okay. follow-up, And are one just many patients in then ongoing the access protocol? on quick how

John Maraganore

But, haven't it's the was And well. a as as protocol we'll product. We recently provide opened yet. for to relatively up closer more we color only get that approval

Whitney Ijem

Thanks.

John Maraganore

Whitney. you, Thank right. All

Operator

question Navin onto of now Jacob from UBS. will We Please move proceed. our final

open. Your line is

Sriker Nadipuram

Nadipuram actually for for on Thanks everyone. Navin is This Sriker Hi, questions. Jacob. my taking

to you can product? CNS on assumption. this, Japan expense U.S.? average have asset patients that longer-term I the maybe R&D was I collaboration run Regeneron to on dose just question. ALN-APP, us Japan? sense a When And saw a How the just an initial Alzheimer's then the in the missed compared applications First disease. average ONPATTRO, then COGS And do dose give you through that expect the the possibly in I the in of of to could have included, number

versus this you the on thoughts news others? have any guys With the the updated recent program the – much. news, Thanks priority of very

John Maraganore

Great. three questions. Those great are

with start one. Akshay then I can go let's to So last Jeff Barry then and do and the and

Barry Greene

Yes. the Thanks question. for

very is patients I breakout. very other not can color were international aware, that, KOLs just tell the from well. a – we well to the Japan you providing going known in in Japan. you are tafamidis very launch than give breakout we're as any mentioned, some So The

is dosing, So say we're starts patients. to dose, now. novo seeing a of bit it's wastage be all about yes, tend lighter, can about Which little and that's so de numbers now Japan both we patients right but significant

John Maraganore

per price do very provide. can want Yes. to… strong vial well, The Japan in as Jeff you is additional we color some

Jeff Poulton

we we'll fully Yes. percentage expect XX% On the when to the COGS today, sold cost of for that revenue we're that's a to goods to And from of of have occurs high transition percentage at previously zero about sales, XXXX COGS first to into COGS but see benefiting been half expect when step ONPATTRO. be that of mid expensed that the as see cost we teens. up R&D.

John Maraganore

And and then, thanks for asking Regeneron question the of APP.

further you do to comment want amyloid Akshay, about as before. characterized we've our excited obviously It are lead protein We program. CNS precursor on? is

Akshay Vaishnaw

I our believe will be the time it that CNS space is think work first continue to being and the well. in going we

I Pfizer debate. world. is discussion think, with more implicated heavily broader genetically continues which of we've the had thousands, many we've target to which and A-beta validated primary the as APP ongoing inherited believe if from disease, a Our the in form angiopathy, and it's around of not affects Alzheimer's where it's an question, recently amyloid the millions cerebral And before the hundreds respect wild-type be the disease. target And very in for form or sporadic discussed of to

how the at data see set. regulatory interesting various experts very aducanumab elsewhere, be and it'll evaluate to And the agencies

on optimistic the of would you of A-beta the I in TTR. better the fueling And substantially protein much could with nature profound what be learned to turn the as the important disease, think suppressing a be top but to have further agents. off turn as tap know, than be reduce we've done production that to have off impact in we more or it and need would the And very that.

John Maraganore

and good. see And the and to approaches be for medicine well, at that and light pleased the instructive targeting were for And we as the that's that program obviously some tunnel be obviously, aducanumab patients. is hypothesis good could of end our might And for amyloid there which also Yes. Alzheimer's.

Sriker Nadipuram

you much. very Thank

John Maraganore

Thank you.

that So, was I last question. think our

at believe of very best the So company want us thank doing improving commercial lives joining that patients progress pleased do capable to that shareholder we've remarkable And, build to with on for obviously value. we the I on making everybody and we're been and Alnylam. of really creating R&D call. a We're significant our focused

the look coming then the So weeks with you forward we R&D updating to beginning in of you our again, towards and next very next that, at year. Day Thank much.

Operator

and Ladies today's conference. gentlemen, this concludes

Alnylam Pharmaceuticals (ALNY) 31 Oct 192019 Q3 Earnings call transcript