ALNY - Earnings call transcript Q2 2019 | Alnylam Pharmaceuticals, Inc.

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Operator

Ladies and gentlemen, thank you for standing by. Welcome to the Alnylam Pharmaceuticals' Conference Call to discuss Second Quarter 2019 Financial Earnings. There will be a question-and-answer session to follow. 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. ahead. go Please

Christine Lindenboom

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

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

and will During Jeff today's review call will review and summary perspectives affairs open before clinical some some and context; provide incoming provide progress; of will updates; we as call, on will of recent provide general his comment Manmeet outlined CFO; our commercial a as your brief questions. Barry new milestones Yvonne our will two, update priorities medical John will remarks an and slide upcoming to Akshay financials; introductory the provide in our on

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

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

call the John. I'll to with that, turn over And

John Maraganore

the get Barry Thanks, commercial and in second for on our execution R&D both progress, objectives. into second one-year to but in ONPATTRO of the commercial approach launch ONPATTRO this thank that the drug be our across we of approval, on uptake, you joining continue medical with with and continued patients quarter of and anniversary In and we end the pleased quarter. at the the everyone very will details a call morning. high level, over the XXX XXXX, affairs strong commercial Christine, as we

to the to Importantly, to near quarters in for come finding, we continued by patient growth continue activities. generation new see and and evidenced revenue years opportunity expansion, driven global steady and mid-term

of amyloidosis broader III opportunity revenue program for ATTR of patisiran our Moreover, entirety in is development including and wild-type portfolio we since advancement about late-stage the to of have of a activities ATTR expand expected our just is not Phase assets, registration. course, hereditary Vutrisiran alone our growth a our into and program And significantly.

this excellent year, R&D the III progress completed made Phase also next our early of we cover We product in will and launches goals. approvals regulatory reviews. more against results and detail, Akshay second positive the in believe but with poised our assuming and for Givosiran regulatory we're Europe, U.S. submissions, positive

date, FDA the need X priority com has that announced granted we with a February the and [ph]. yesterday, Just PDUFA Givosiran an ad review for no

opportunities pipeline weeks, an Inclisiran, for to industry out a Regeneron in readouts longer-term leading to therapeutics. we RNAi see growth positioned our primary of starting read program partnership build therapeutic focused RNAi next capital-efficient wholly-owned will our enable licensed and additional hyperoxaluria year, where And we're on ocular III manner, we medicines Company CNS innovative ever Lumasiran, largest and two program. completed diseases our with in end Medicines significant of addition, by the then The the for In Phase to with the now few [indiscernible]

our support line to believe clear of be more the our our of this to on couldn't and tier the innovation, from a toward multi-year we global a execution. all prospects, the today, Alnylam being a to that Alnylam deep Furthermore, clinical our excited fuel business product funding period we Alnylam growth, have progress an and with alliance, Regeneron commercial near-term achieved sheet for company a continued top up R&D incredibly multiproduct, a substantial horizon about with strength and balance in ever, bolster XXXX story. we if Based in believe rarely, has sight engine robust we biotech. goal pipeline profile a important and future bio-pharma All frankly sets

medicines where, growth, landmark On innovative approval return importantly organic built global industry confidence hand, time, support our many ever we investment very than greater RNAi. having of match are path therapeutic is commercial stakeholders on the in capability we one will a we believe to our self the to now, reasons; exceeds some ever coming in in investment deliver first our having the hear pipeline revenue following years. the innovative modular, leverageable a and norms. the At and same on how have for eager reproducible, generate RNAi and that we the platform sustainable profile know growth to and revenue continued financial achieved on hard of And for that to believe promise to our our and of ability the attractive commercial

join call. for through I'm to navigate in reasons, us the incoming our have Jeff transition comments to later important future. Poulton some the make CFO this team will help growing excited very our For Jeff these Alnylam

aspirations. he Alnylam like our to everything Manmeet support has built for foundation for also and contributed I'd company to the Soni our we've to that thank XXXX together

over RNAi join plan will the that our able to I R&D on Friday, XXnd day before we or call an morning of the We that hold turn November to we in advancing where person in in Barry, New progress are recap mention our on to webcast. you'll to much very planning like City be York hope Now I would to latest us pipeline therapeutics. the of

Barry detail. Barry? the more in affairs I So and now will to to medical that, our with progress call commercial over turn review

Barry Greene

John, global revenues product I'll everyone. good by in commercial $XX.X quarter. quarter. performance the begin second in morning net second reviewing ONPATTRO's ONPATTRO the Thanks, million We've achieved and in

launch, As in the U.S. from on with for quarters first sales split time million United come pleased the the EU the more the the and majority from geographic from to of $XX.X color States. coming $XX two-and-a-half we're first provide revenues now million and of

As of receiving worldwide over patients were June ONPATTRO XX, XXX commercial treatment.

from and expanded the with demand quite drugs entrants overall with access growth quarter clinical this and global We're pleased options availability market trials. product of increasing through the investigational investigational even programs, recent continued

sources who sites very are our EAP, there've de and Program novo patients known patients of Access reminder, or a launch in new are been to that patients our from three As Expanded quarters much today, In launch, patient capture patients. the the two we're the in effectively first of particularly couple pool the and novo States. de sources into we United of first now

trials our that we're than put number milestone greater across increase in clinical exciting who patients very patients clinical patients XXXX on commercial with have expanded efforts. in our overall to we approximately track our treated studies in global are XXXX, a worldwide believe by the XXX program year-end being We to that ONPATTRO now EAP Now, that. access and

turn the U.S. now market to me Let dynamics.

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

multiple XX% in prescribers disease the will awareness state in engaged by healthcare the from continue prescriptions education. new awareness over In provider fact, believe received came to fueled rose players new and second We increase quarter disease of prescribers.

and U.S. mix awareness the strong indicate population. the of of we Regarding to from the recognition see prescribers, the XX% about of which phenotype mixed come continue cardiologists of neuropathy forms start this poly in we believe

primary care expert Additionally, we're seeing like greater centers. and of specialties prescriber increases hematology numbers outside awareness disease of new the

Positive for of future continues is experience to be to use. continues key and persistence strong predictor today. adherence healthcare and also from ONPATTRO are Speech providers therapy patients highlight. a improve overall a And very and course with also

coverage external access, categories. Medicaid payer confirmed now U.S. more as that if than Medicare, to U.S. very commercial, of pleased we Regarding other lives access have market across for and reports, we're government reported prescribed ONPATTRO XX% by

with in and approach VBAs proud payer and disease with in this and including and complex avoided based value We community. orphan with continue use partner agreements constructive, the collaborative We're reported launches. it to we've effectively adopted a headwinds of the space environment of access market payers often reflects or U.S. this U.S. productive reported believe have very result

Africa to the turning Now CEMEA region, performance Canada, and very Europe or and we're in pleased EU with ONPATTRO's East, Middle more broadly our region. the

million As second we $XX earlier, achieved the during EU noted net we product revenues quarter.

France of Germany, in the include technology in during was highlight the pricing with achieving A and Scotland. with NICE achievements a and assessments notable in Some authorities pricing England Italy. competitively favorable and agreements differentiating quarter period

now we're Through in region. marketing received in selling for named-patient XX approval where over or sales available in We use. expanded now authorization the ONPATTRO reimbursed access also is direct reimbursement, ONPATTRO CEMEA countries commercial Canada

reimbursement a and Given in the XXXX. rest pricing continued in market commercial of patients for number discussions of the expect ONPATTRO CEMEA, access into XXXX timing and we base of growth

finding the for and were growth disease response In progression from addition to products utilization patients other may other experience in and for coming inadequate tolerability. patient

advancing the our in for in approval and CEMEA in Brazil authorization, America exploring a our preparing Japan sales and received Latin for States to launch we're Turning also outside commercial countries. regulatory to in United recently marketing Japan approval while progress ONPATTRO we plans submit non-patient for

patients we expect our U.S. and country second reimbursement the of Given revenues environment and XXXX. disease largest be on drug exiting presence the in endemic the favorable Japan to after

As online around of an come world growth. more we expect additional to be driver future the countries this

education the treatment committed diagnosis reach remains improved medical in challenge help of and and hATTR faster. also awareness disease addressing diagnosis will amyloidosis, team improving patients to options Our raising

are that treatment receive The overall improves that data in prognosis. clear disease our the when patients course earlier

third highlighted diagnosis the Now at Canada US previously patients [indiscernible] in regarding patient suspected party of diagnosis of aimed and available Act genetic we've facilitating having Alnylam screening doses. to our program the as initiative

over pathogenic have other help only As the the rate consumer we their genetic learn variances common and more sponsored XXandMe of are genetic XX,XXX also testing. TTR X% to Alnylam the about genetic pleased now also service We're for with Act a three submitted there have risk genetic their sustained of most since X,XXX of samples, represents with of July genetic and identified partner patients tests HCPs States. X% been programs portion hit to been over the to United customers testing reimbursed have mutations in

with we're confident countries see and and finding to future competitive growth summary, more efforts the features continuous an of access continued and and improving In more commercial ONPATTRO we're even achieving environment. with and patient and differentiated our increasingly highlighting diagnosis steady the evidence in with encouraged generating approval ONPATTRO, potential

for opportunities growth more confer at about ATTR believe Moreover, as also patients. the amyloidosis ONPATTRO potential hereditary our in our ATTR we look in are we with future. horizon, therapeutic, overall investigational RNAi ATTR benefits, through our both market. companies for mentioned, broader wild-type to we awareness once in label amyloidosis we're space segments will and in amyloidosis the being the position ATTR the believe continue to Patisiran, all and will committed with believe expansion to the time and there previously ATTR subcu We're significant it's I accelerate potentially of market, of us cardiomyopathy including patients As with and efforts, very amyloidosis overall enthusiastic leaders franchise, quarterly well we this the advancement

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

Givosiran the ONPATTRO in developed In is we're built decisions country. commercialization agencies, following expect U.S. positive meantime, best launch Assuming by and will CEMEA capabilities launch both and country the the from and in the we practice leveraging XXXX. early by

focused and successful and laying diagnosis patient team in hepatic communities on groundwork AHP awareness the porphyria the and a is acute launch. for of Our improving

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

to [Indiscernible] to who carry with be genetic individuals known may at AHP. no or U.S. will associated provide testing mutation in Canada gene access charge for

estimated XXX for program early the AHP report we this is with While XX stage, mutations. patients and in AHP can

hit a is unmet There As for the and significant of approximately clear disease of XX% and need hepatic burden acute rate. porphyria. July accounting very

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

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

million past, it's opportunity in be potential. we that over Ultra-Rare attractive said $XXX Orphan the we've peak will our revenue Disease As global belief with in

As launches consistent we orphan with underdiagnosed populations, of in ONPATTRO growth trends after drug and that steady expect other show launch. pattern revenue those

R&D recent turnover progress. Akshay? review our With that, I'll now Akshay and to to pipeline

Akshay Vaishnaw

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

year as mentioned our have earlier. an broader opportunity to richer day in the on touch R&D and pipeline later will a We John

me Let Fitusiran, start unbranded from the is name which ONPATTRO. with

society presented impairment nerve we for global XX and label from in results in neuropathy month treatment. June, extension As XX after open Fitusiran a ongoing months of the study of quality improvement patients peripheral meeting a life of new showed interim sustained

These experience Fitusiran course, for prior including Fitusiran patients our has as disease. showed early that there advancement abrasions. with policy to caveat possible progression minimize importance also studies with Fitusiran view, and new presented a no also newly of of showed to be believe continued in we profile with The families who results on initiating differentiation to what treating in Of the results patients head-to-head data comparing to favorable been the treatment

Phase to advancing to XX of wild-type evaluation hereditary year to [indiscernible] We're this in this at forward for timeframe. III Cardiomyopathy the market. APOLLO-B start for label aimed support ONPATTRO include study expanded look now ATTR will include and later seek on Fitusiran XX Fitusiran track expanding the and to the the regulatory in We amyloidosis label we

know, is and we're which Vutrisiran, a also development by is delivered you ATTR As injection subcutaneous for quarterly advancing also Amyloidosis.

hATTR patients been we've with Vutrisiran. enrolling ongoing While Phase HELIOS-A study the polyneuropathy with in III

and alignment evaluate on pleased III patients endpoint HELIOS-B. to study HELIOS-B design wild-type amyloidosis, hospitalization impact that obtained cardiovascular a cardiomyopathy. today inherited ATTR Vutrisiran of and have will with on mortality. we 's We're of regulatory will announce Primary Vutrisiran be Phase the

in enter we wait reasons will to later details data. providing space It's further allow study outcomes Vutrisiran opportunity until clinical amyloidosis ATTR large successful starts with this the year. very your competitive should For the wild-type market

to the complete on study from a our the to III which Acute ENVISION therapeutic results on In the April, move Hepatic Givosiran, of Let's developments and Porphyria. investigation with led positive Phase treatment

treatment the relative to patients robust in rate placebo. significantly effect a demonstrates of composite reducing Givosiran AHP in annualized attacks

primary mean in rate efficacy patients attacks Specifically, annualized to Givosiran AIP median the six XX% a relative reduction met months. and porphyria the XX% endpoint placebo in with with composite over

to Turning study. of XX.X% the patients XX.X% placebo XX.X% Events One and in reported of of reported patients X.X% Adverse results, tolerability AEs patients in Givosiran the and Serious of safety pivotal Givosiran patient were placebo Givosiran were group. and function case AE, study drug, test in kidney the Givosiran arm, in reported SAEs as group liver related due abnormal were an previously three mentioned, and pyrexia, as discontinued treatment in of to disease. the chronic single each to a

the FDA to FDA meeting that PDUFA accepted X, setting time. U.S. submit We this for need a date has and were the the very information gone pleased in the with Agency. second yesterday quarter applications advisory NDA the of that and to are just being we at announced a both for The indicated the with that to not wrong Medicines XXXX. committee now project And FDA they complete the MA February set also the foresee do has European an review

to patients respect make to we've and healthcare qualified now from following available to Givosiran program Givosiran with expanded access providers. enabled AHP potentially the requests Finally,

recent I'll now of and and for Lumasiran, the [indiscernible]. or from study and progress profile. urinary type treatment or therapeutic II to an basis hyperoxaluria foundation of [indiscernible] normal PHX see normal in the meeting studies, of near investigational robust in Phase we these [indiscernible] we OLE primary turn II Phase study to tolerability June RNAi presented with I, continue In final the a interim to from levels oxalate encouraging results X development

mild conducting moderate PHX one now renal or we in in study top As with patients placebo-controlled early randomized, impairment. Lumasiran. aged submit III enrollment trial case double-blind, in of in X report remain to to you know, a regulatory older pivotal to are study ILLUMINATE-A the is completed on with XXXX positive, Phase and we line results track from this if ILLUMINATE-A XXXX. We late beginning and filings This

this in III trial severe of the of the third case phase We ILLUMINATE-C Also when study for in a Phase ILLUMINATE-B we impairment plan the with second Lumasiran patient later under case in year. one initiated renal six. patients quarter, III age

and III made RNAi May our therapeutic and study we've assets. the targeting the early company medicines the results investigation, progress to at our have medicines with and stage been ongoing addition from of interim In also reported our assets, In Sanofi advancing company holy nine. Phase late partners Transthyretin in ORION-X Inclisiran partner

Inclisiran patient forward been XXXX I've the There was data Notably, on XX% therapeutic a sustained that from in results the to from line data by human an had of for occasions safety results this than with separate nine years phase study ORION-XX, Iii the study. experience is conduct. to entire issues pivotal lowering with more program of the then if committee monitoring studies LDL for no been milestone independent starting now the cholesterol changes seven than field. the top the in of by third look no quarter, positive and RNA will more We recommended largest achieved and encouraged then important second-half safety reviewed XX by be material investigational observed the seeing and

that, the Manmeet let now call financials. turn Manmeet? me review over with So to our to

Manmeet Soni

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

quarter Our results financial cash position, XXXX XXXX guidance. and our our updated second

cash securities our with investments. a $X.XX start We balance Let ended in restricted me quarter approximately billion cash of second equivalents, debt cash, and balance. strong with sheet the marketable

$XXX includes received the to collaboration. Our Regeneron attributed quarter balance our of proceeds in second cash million

recorded $XX.X which during represents We from net product Moving global million. second revenues quarter million of $XX.X growth now revenue were to net quarter quarter XX% the global our revenues XXXX, second first of revenues, product million. of $XX.X ONPATTRO total

gross to second Our during of RGTN net was in XXXX mid XXs. global the quarter percentage

second net the recognize million Goods XXXX. $XX.X revenues. the a of of million $X.X We $X.X for second XXXX XX% is Cost million as of Sold product during to revenue which collaboration compared was as quarter, during the quarter percentage quarter approximately second of of

[indiscernible]. expected, collectivities Genzyme decrease the reimbursement collaboration with is As in our due decrease revenues agreements primarily with in and in Sanofi connection collaboration a to

batches Moving with Regeneron for and stage and of and $XXX.X to of period as expenses second million fees our non-GAAP second decrease to programs substance expenses and $XXX.X the as license compared XXXX. was in GAAP expenses number R&D compensation quarter of agreement expenses expenses, the during XXXX. higher as and $XXX.X other to due continue increase manufacturing increased for clinical were materials. raw This facility operating late of increase and to advance quarter a million the collaboration costs XXXX to million Non-GAAP $XXX quarter headcount expenses by reduce we development pipeline. to compared offset and The of as increase expenses our second R&D for trial drug our expenses million, expand result the R&D was related in of and related the growth a

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

guidance be today range $XXX compared the to updated our million of million annual pleased $XXX R&D previously to non-GAAP in million range $XXX our are of to to We expense million. to $XXX have guided XXXX

guided million our the to million. $XXX of annual our tightened pleased also XXXX are guidance $XXX in be to non-GAAP to compared expense SG&A of range range as to $XXX previously We to $XXX have

today on of Alnylam call of proud of the Company many thoroughly launch geographies. am over with serve the for personal working created has Alnylam also a Jeff note, my expansion part contributions the team, time and years support foundation the commercial many confident as that I my on multiple come. past Finally, last made team, the years Poulton with have transition. a enjoyed its enterprise I and I've CFO earnings is I'm the to few global to will enjoyed across the

to I will his perspectives, now Jeff hand over Jeff? it highlight to some of

Jeff Poulton

in be transition. establishes help stage Alnylam operations. week. Alnylam's your a patients' Thanks, assume very I'm commercial with the thanks Manmeet, and of I it its this to this with brief impressed the I'm formally frontier in CFO medicine. company's make growth joining few make in as next like excited as to I'd global remarks and technology potential difference at the really role to for compressed and just to its new team lives a

first coming Strong engine emerge tier the believe product Prospects many a and for pipeline to biopharmaceutical years today. organic that is positioned robust future as XXXX commercial commercial with company. clinical in deep more its for the beginning products a Alnylam top product

I a comments my earlier key global with in organization pipeline. this self communicating becomes a significant and financial our working me a John's by R&D look colleagues with successful top focus for the future. to be new will Consistent with plans roadmap create commercial deep Alnylam line the challenge the forward growth to is fueled sustainability

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

Yvonne Greenstreet

we've of really countries. far, forward Jeff, made a welcome Thanks, including we to in The of look to plan launching but to global be number of year. with ONPATTRO the to so team. we progress the starters milestones still in board. continue have I'm our couldn't on and We commercialization exciting the remainder XXXX to happier thrilled have other Japan you and the

wild-type in APOLLO-b aimed initiating study We include forward label to amyloidosis. Patisiran XXXX hereditary cardiac also expanded for at look commercial the an securing mid and to Phase III

ICPP expect and ENVISION an study Givosiran and initiate the on continue at With trial the amyloidosis from cardiac September. plan data patients in outcome we late Vutrisiran, we additional HELIOS-B With ATTR hereditary in Phase wild-type to HELIOS-A XXXX. the having throughout III in to presented year plan meeting enrolling early

from plan line the late results XXXX. Phase in to study [indiscernible] study now report the A that to complete enrollment we ILLUMINATE with in Turning top III

with plan renal continue in also and ILLUMINATE-C PHX the pediatric severe XXXX. in enrolling to start patients in later patients study ILLUMINATE-B impairments will We

XX our exciting as we earlier course the NDA from the those XX report to advancing efforts year inclisiran of for top With ORION assuming we'll is Inclisiran, our readouts well clinical line of continue at stage the expect the partners X, a of end In data as milestones to by and you later beginning preclinical and [indiscernible] Company this quarter early rest an expect and efforts particular, pipeline year. submit studies the we'll of of results from positive number and they highlight the our as should data occur. throughout mid-stage clinical And programs.

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

Christine Lindenboom

Operator, would have get and open the to additional then We dialed in queue call to you to you limit you, question to back for questions Thank each, we yourself question. in. ask those the Yvonne. one will like if now

Operator

[Operator is first you. from Our from Thank question Instructions] Alethia Cantor Young Fitzgerald.

Alethia Young

launch. progress Hey, continue you this And congrats for the guys. my taking on Thanks as question.

around Can you talk little of that? diagnosis? any bit there to how driven Pfizer give Thanks. a more metrics us you talk quantitative little drug and how your one of me on a talk little like Alnylam's both cardios? kind maybe, but have you're is Tafamidis decisions, tagging from and bit that, the just have [ph] doctors about make Just of and can seeing And then what other drug the just color doc hearing you and give any or I can a for a the bit too, can follow-on XX% you presence of about

John Maraganore

Thanks, Barry, Alethia. want this? Right. you handle to

Barry Greene

the are it's learning prescribers every of early on Yes, disease, give are that sign people and the so and on comfortable the diagnosis, holding diagnosis which seeing their around more color patient. quarter, are and specifics emerging gaining more a to new too Alethia, and about new to is the speed other disease, increased diagnosing that landscape than we increased then

awareness. So awareness. in educate patients for players of amyloidosis of helpful that seeing having is has been multiple raising And cardiology at we're this definitely ATTR congress anecdotally increase the certainly disease every

that early choice, wild-type, patients understand it's from in Tafamidis of been their In again, Tafamidis, amyloidosis research us that and And those drug ONPATTRO of ONPATTRO Now, then dynamics. in on has with understand the major polyneuropathy; particularly progression Tafamidis the hereditary is are where on too What to years, [ph] phenotype switched, those States. Tafamidis in launch who seeing to for understand physicians ATTR Europe, is the is our particular Tafamidis specifically in a even mixed that used. telling patients the United been the choice. is market of a for we're where are present Tafamidis hereditary a clearly markets number number of has physicians

Alethia? your that question, answer does So

Alethia Young

Yes, thanks.

Barry Greene

thank Great, you.

Operator

Wang We'll go from next to Barclays. Gena

Gena Wang

taking for you Thank my questions.

regarding First Alethia's also drugs, one also How cardiologists. question, were is patients many And from payer on commercial from for XXX demand the question, how ONPATTRO commercial for the all the ex-U.S.? XX% following coverage these patients?

John Maraganore

Great.

second and over out patient to breaking not it then Barry one. We're Let address region. numbers I'll the for one first the me by hand quickly

time, this and U.S. the million in million so EMEA revenues, and We $XX case. Rest in first $XX.X World, we're of are, out the reporting for breaking in

it limit So for that's unfortunately we're going competitive to really reasons.

want answer you to question? the do second Barry,

Barry Greene

we which U.S. of actually is I our of saying reported, reports prescribed terms reimbursed remarkably who mean XX% patients have that we remarkable. ONPATTRO coverage, in be external quite Yes, ONPATTRO payer will

We patient have not to rejected a date a had by payer.

John Maraganore

And think, has, very a all These asset this medicine of payers, the introduced and engaging to market market. in high this what discussions have we're the just are successful with the for add I story with as to agreements. payers of on to and that, to that took value-based day belief very strong in we approach to that we're ONPATTRO market, elements been important quality continue even Gena, proactive we the U.S.

Gena Wang

Thank you.

Operator

from come next will Whitney question our on, from moving And Ijem Guggenheim.

Whitney Ijem

for the thanks Hey, guys, questions. taking

going The to them ask on first I'm Givosiran. one I guess

use what the kind patients either terms on opportunity, or relative in versus be any terms of terms of for the you either assumes in first give used how in than million So sporadic Givosiran drug $XXX patients, will in color that us breakdown greater bucket? in the can recurrent one, in of of

John Maraganore

to patient the call, be on the experiences defined just or that there population and life are that obviously in be disease any that question. mind thousand that have be great Europe, less. estimated and Keep the three we estimated threatening. for others in And an X,XXX U.S. with can as the a sporadic approximately can patients patients of Sure, recurring experience patient attached an attack maybe to benefit attack

So are [indiscernible] the little their importance want of But around doesn't how they patients. we that, this the to Barry, to lessen if it beliefs $XXX bit with get comment that you disease opportunity? a million sporadic

Barry Greene

very outlined it And, very, well. you Yes. John,

ultra patient have we unnecessary surgery believe up many John to with show journeys XX their about number more XX diseases in by out journey, diagnosed four can orphan are dozen a patients years, three or highlighted than there. we physicians missed made probably the last there that that rare patient As where and to

years, fact, now studying three XX have of this we disease years, did two-thirds and do interesting for partner an of them, industry patients the [indiscernible] [indiscernible]. that as As working debilitating have little the pain with experienced natural not as chronic which benefit all consortium has understood history, been between by

the about of So said, about the just not it's attack, of as John nature attack overall number disease. it's devastating rate,

multiply and So point, likely when XXXX the a opportunity. there we and orphan price very an you take significant growing think is that XXXX by

value-based to are put with very going ensuring United [indiscernible] and around in taking we patients same States by course, frankly devastating in to proactive for right disease. access ONPATTRO have of we will the with agreements the medicine patient an Now, our world the proactive that the driven important payers philosophy place take the access so with be to nature

Whitney Ijem

give ONPATTRO, price U.S. this on the And at can ex-U.S. Great. point? follow-up any one you relative quick on versus color

John Maraganore

comment? to want you Barry, Yes,

Barry Greene

Whitney. Thanks

on calls, previous course, price very, it dosing. very basis. we've per price And varies span As tight held of vial on then we a highlighted because weigh-based is have the

countries in people, the more So very patient very, year smaller less. price is it's it's and But vial bigger people with per tight. per dollars

Whitney Ijem

Thanks.

John Maraganore

Whitney. you, Thank

Operator

from We JPMorgan. next will go Anupam to Rama

Anupam Rama

on Hey, all taking thank much you so the and the guys, the question, for here. congratulations progress

John Maraganore

Thank you.

Anupam Rama

much. then and of outlined to HELIOS-B Wondering have color prescribing OUS of for cardiologists. from you you like Thanks any are on color U.S. gating factors the any about maybe ONPATTRO you XX% Vutrisiran? so here coming the if some on starting the In could trends, provide the demand getting

John Maraganore

Right.

So about And then, handle a second the you the -- let's talk next prescribers. bit have question. can Barry the trends ex-U.S. on Akshay, little

So, Barry?

Barry Greene

Yes, Anupam. thanks

our countries clearly practitioners they As diagnose U.S. XX% become like are with of highlighted named real the cardiologist. that the our additional familiar places to And with There it then really primary what ONPATTRO. centers. to at Interestingly providers of disease, it's centers and Germany, some prescribe said those primarily enough the care specialties France least are in or coordinated were in we depends you including physicians, care they And Ex-U.S., case understand are like neurology A nurse on cardiologist a how have as patient, that highly patients. healthcare about see are with in the number driven. sign growing. and country. -- neurologist prescribers is takes

So, depends the ex-U.S. country established. centers it and on the really how are

in the initial least neurologists manage primary that are the disease be we to earlier, Japan that at Turning which will we the launch symptomatology. overall in prescribers folks because those Japan, believe mentioned

United parts cardiology uptake in VXXXI probably really course, at patient so the space. the population, see least in dynamic of we back African of of patients -- will Now, Sates bigger in the hereditary world Western to where descent,

So it's following up.

Akshay Vaishnaw

this program half the excited [indiscernible] that know are in type that factor thought you enrolling -- reported regulation And up [indiscernible] shape forward and of excuse we main agencies year globally, with question, study. start getting we well morning in getting from on now this aligned details as the patients we I know, that globally. Anupam, as me, we to essentially of are HELIOS-B great think to following are aligned course the gating second is as And all in we looking vigorous with now.

John Maraganore

Does that answer your question?

Anupam Rama

Yes, great. for taking Thank you my question.

John Maraganore

Thank you.

Operator

Baral has from next Ritu question. Cowen our

Ritu Baral

question. Good morning, for my around have on U.S. I specific growth. a some gotten emails morning this everyone. concerns Thanks taking few already

you to is the don't was assumption like of want U.S. comment on the comfort persistence largely especially patient the us QX How Can HELIOS-A much U.S. think given little launch and on more like you things on comment growth steroid-free having things of impact how treatment? on rate. you treatment that's of color number persistence revenue XX% I that understanding Can QX the a the give enrollment. an driven around little more of a number XX.X by numbers.

John Maraganore

Ritu. Yes,

First steady wrong. comment for growth of all provide if we've I the more is specifically Actually in Manmeet and growth mean some to the to want concern had Europe. completely U.S. about you in U.S. more that.

Manmeet Soni

Sure John.

So U.S. Europe, Ritu approximately growing and had growing rate was QX. U.S. QX growth to as even but a U.S. mentioned from consistent John XX% from out to QX, I if was we revenues higher. at that is global over QX XX% split grew

$XX.X or them you last would you numbers was XX-Q evening this the not quarter quarter, So today in QX later to $XX see for approximately when file which this million. and filing million and is there give were you tomorrow U.S.

a shows strong pricing as million in investment in We pretty now $X.X primarily quarter growth is as final of so were there the you quarter a growth and at know, new get that growth that finalized. last are and countries also this shows comes but XX% Europe. XX% million, $XX Europe So the launched we and we

So are we book. the want global give continued revenue our on, sorry. as and growth U.S., Europe no there and both John revenue concern and is mentioned, the and just I in to showing steady on Barry

Barry Greene

Yes, go ahead.

Manmeet Soni

questions more some were on adherence. persistence there if Barry No, on

Barry Greene

Japan to launched out think late we a XXXX on I particular the coming In we're Manmeet Manmeet and continuous quarters the and of year a in and number this growth of incredible uptick in then to will countries and steady because year where States you growth even the Yes, and growth are maybe continue just highlighted, United new is Europe seeing will it into on this believe covered awareness. we into countries in and emphasize late continue as come XXXX. see

entrants steady So growth we country, then and new see each within do market within countries.

that dynamic So continue. will

You remains very asked into we physician high. specific high. reports give adherence we launch patient the see hear experience patients and numbers It's tremendous ONPATTRO to well our on early tremendous about and very very, anecdotally continuation, rate about way remains experience of continuation but it

Manmeet Soni

drugs. all And rate is the that benefit conversion risk the the very just the study being rate there Apollo is on itself Ritu, evidence persistence only patients original to the dropout high into that that XXXX XXXX, follow-up to early, well from know on who've Phase patients in the five Phase III II staying drugs the the then early We been from years are from and on it. onwards and low was tolerated actually

this by as we as the So don't considerably as the been years comment way applies now progression reduced has I have very good dose the the evidence tolerating Barry well are accumulating patients which over and but steroid well. think

Ritu Baral

it. Got

Manmeet Soni

your Did that answered question, Ritu?

Ritu Baral

impact launch on The of that HELIOS-A enrollment as on of HELIOS-A the well on the launch.

Manmeet Soni

we're HELIOS-A product not from the as the look a we they We patients that understand and patient future will ONPATTRO that in the future. program of patients there patients. in could the continue new for be investing to that That a benefit too said the of I for franchise. growth of Yes, drive but to offering distant for think mean having goes in commercial beginning obviously know course. we potential I the We every that patient HELIOS-A, are mean

eyes So that. wide obviously it open very do decision is of a to type

the no reimbursement where the countries the that Now this we're to U.S. at is outside And time. see and just in expanding past we sites going markets are of to quarter there benefit importantly present significantly

commercial balloon might affect from that growth So obviously out that clinical studies. will on ONPATTRO occur any the

Ritu Baral

questions. Got it. Super helpful. And that was already three

for the before me. Josh So queue in I'll comes get back

Manmeet Soni

lucky Great, day. Ritu. Thank your you. It's

Operator

Paul Stifel. We Matteis take will next our from question from

Unidentified Analyst

maybe you're programs? then clarify Ned novo guidance. patients does one quick that include seeing quick number XXXX de thanks the Hi, drug of this terms growth is taking by getting to rate that a and Paul, or is access commercial in for sequentially patient question, year-end that that on How and confident you patients are in the for that just on the X,XXX is expanded

Manmeet Soni

patients includes it and open program also in access in our label ongoing our expanded study. Yes,

do XXX So number achieve of universe. that we're XXXX XXXX to by point over on the on track we're We're end at roughly to believe And that that very track within the approximately that. year. over hit this we patient patients much

So that's encouraging.

Unidentified Analyst

then can if the And on growth that much patient provide don't rate know you de I novo Great. just subsegment? detail

Manmeet Soni

growth. I Barry talk mean we when well about Well that's but comment can as

of being patients identified be we buckets is per Let's about and samples understand or roughly couple growth. thousand clear Act, so patient you Alnylam the getting quarter XXX are per quarter. new we're de and we novo One look a patients finding could finding,

year And over that's for that been a very, program. now very of steady

testing can look Act, finding patient by not said ongoing are surrogate just finding, a funded it's out not. other the Now Alnylam those patients or aren't earlier, some as Barry this if companies, patients programs you at necessarily ONPATTRO that marker that is you a just are as that some and number it's will is there

the happening. really there right in as is amount Canada new of way significant So patient just And a by finding that's well. in the U.S.

around So better. it doesn't the which reflect finding is also new getting world patient

source So growth. that's of one

The growth what's And the which of of access around basis XXXX important in the second country-by-country Europe the the what's we U.S. source and market. on and going an in of important a expansion Barry is end commented the and going to of also source addition of that's that's growth be our in on expansion to world. largest second market global be will growth. important believe a Japan source and going earlier rest very in on is of our Europe by

growth. what that our for that time to with generation have both affairs to disease the the highlight where use ONPATTRO near-term or And help final for and -- the understand can evidence understand which be for is team activities Those may term, believe of will to and the to ONPATTRO activities very development source out a I strengthen are remarkable help our we exist like really of that to clinical of other ONPATTRO their issues best implications growth therapies ONPATTRO are to points patients progression the be types of of think there team it physicians in some patients engaged mid-term profile differentiation medical longer the helpful.

to me pause add. Barry there else let So anything

Barry Greene

No, you covered completely. it

Manmeet Soni

Great, you. thank

Unidentified Analyst

Bravo, thank you guys.

Operator

we'll Next from Bernstein. Vincent go to Chen

Vincent Chen

associate look quick corollary. trials very mix doses them and cetera provide up portion us the amyloidosis the pure Could in on on way recognizing What disease ONPATTRO, with community. over of and et Alnylam in their or a was taking you're question. ONPATTRO on forms the onto program plugged How what a wondering as identified of you many do give in more reasons for color thanks TTR some for mutations TTR Act breakout. time with start with you ending of patients more the phenotype Congratulations a a expect I new progress or in how neuropathy the this for these that but or folks little versus a if you it's and are sense work would third-party does eventually pace quarter? a certainly cardiomyopathy second could

John Maraganore

the as diagnosis treatment It's that if patients. by their by diagnose that. obviously for we a anything We patient hand will program of have then patients just physicians, it comments Yes, design. so quick help This for believe that that medical is not Alnylam and choice to ONPATTRO specific maybe other than in and I'll don't Act these a then over visibility education, improving couple is to that's that's patients lot follow. on physicians a And improving Barry. their aimed of identified by right

philosophy, don't lot terms has of been of that Act. in that we just color, Alnylam other the have than So a

reporting to So the outside that's come forms of have emphasize fact U.S. as And away measure last common on drug the patients, to in of measure then quarter, that that one. And with. as patients Start in specific a contact into from launches, the is Start Start answer that said, moved of Forms therapy U.S. because Forms, we it's an are with our of we Forms. we course, begin

said? is revenues. to else add I even Barry, number So focus patient patient reflecting anything just growth. The you and what not obviously true it's on should what

Barry Greene

test. companies Act. with children, some we a but be And tests for their others, other then to the multiyear visit Act is I a about siblings, finally, is genetic journey think hATTR Again, benefit and a often this Amyloidosis. Alnylam other covered have you genetic to free through often give patients. identifies test just that counseling back and you that a come to reiterate, will other will Alnylam physician diagnosed what see No, companies, after they in offer just people a generic And to patient disease color those of for third-party and well, Vincent, just it we

So get Alnylam the genetic the these of Act patients to benefit burden tests. really eases for

it's benefit an As length patients. we're the in for because said, of arm's relationship, John there

benefit patients that pie remember, some patients penetrations overall not Now, that your raising a about disease. this believe by pie. portion not awareness, will to do to have by speed yet continue of But growing raising is the we It's awareness diagnosis. of and

Vincent Chen

you much. Thank Exactly. very

Operator

Piper is from Jaffray. Next from Tenthoff Ted today question

Edward Tenthoff

very thank Great, and you much on congrats the progress.

John Maraganore

Thanks Ted.

Edward Tenthoff

love a you coming sense and globally, guys growth the Yes, really, comment, launch is breaking really from. the and of are out global really it a are from gives I that us it just impressive coming where patients

I'm of sort sort you if, be sort bit even us you. might how would maintenance, versus out. little versus induction Thank a playing us maybe could that. there. used. sense there for more give of how looking be that this of or market little And of think where about of just see Tell evolve. a kind you thank considering of So truce you IV at the bit I'm really more all a sub-Q

John Maraganore

studies are polyneuropathy the and setting. think a wild many studies are to come. then HELIOS-B remarkable APOLLO-B successful. into about where opportunity you are studies type cardiomyopathy for future those franchise assuming we've our in Again, with successful; of the expanding many, got ATTR And mean, subcutaneous about your hereditary dose, you Yes, exciting, about ATTR, a I once of for comments injection, low mean, about expansion a Vutrisiran, when back, taking opportunity. think an think overall it's volume, excited this Ted, you thanks that, quarterly, we and thanks, earlier. the and years for then, we like overall market, low step we're today, I extremely think the is

multi-billion And I leader. of I to think of where a is without market opportunity, significant terms be type getting heady, Alnylam numbers. in a poised really mean, or this is hidy,

really given patients, less, for end that the bring in oral and and a preferred that larger and that to quarterly an less opportunity we -- studies study the be patients very, shortly positions very think agent and and great done So and rapidly, is HELIOS-A for once after the show a really important read subcutaneous offering with significant a is commercial it's there us then twice with day patients wild-type will excitingly the which the a a of by rolling, currently in provides product option And over very period, extremely that'll Vutrisiran day. even setting option you a access you XXXX a expect that Vutrisiran to like know, alternative, or out really cardiology will going to that get that we HELIOS-B, to up time sub-Q the even addresses hereditary we've with year competitive. an start once opportunity know,

expansion and growth away that look Alnylam market at for to. to is for opportunities an forward and not all, that very very, really are these So exciting far

So, Barry, anything to else to add that?

Barry Greene

getting well. two XX I And think years XX ago No, how last MS. the an earlier to gets it I greater prescribers and guess think developed over covered things; today you XX diagnosis. look project are only to you a the One years. XX out where earlier years, more dynamic analog is the ATTR the interesting disease and Market the by mark we an if benefit can interested to diagnosis at much over patients see Amyloidosis next

out what to those Amyloidosis. John see same ATTR I addition in playing mentioned, dynamics just So with

Edward Tenthoff

so to and so color much. a all on thanks quick appreciate the [indiscernible] I building Great, foundation

John Maraganore

Thank you. Ed.

Operator

Morgan And we'll David Lebowitz go next Stanley. to from

David Lebowitz

drugs question. effort in very certainly on? that Alnylam would characterize Could given that actually so from being patients coming you there's Act coming drug? are for polyneuropathy cardio online to noticing patients the the on that to guess, more for the patients And takes What Thank program that many are the are patients it and you the the my diagnosed much that driving transition taking I for fact drugs? cardio be approved

John Maraganore

to do -- that? handle Sure, let's you Barry, want

Barry Greene

a broadly and of that things I mean, just increase number more there's I to awareness and guess we're Yes, say again to doing diagnosis.

that who until disease awareness Amyloidosis dynamic diagnose is that cardiologists, into miss ATTR can a continues neurologist, disease, bump raised. Hereditary and the miss or patients other unfortunately specialties is where

are a there are have specifically Now to see Amyloidosis, cardiovascular the which front presents segment the why is wild-type cardiologists many, commercially, specifically, course, that assuming and because tools of and so the and we're helps more more cardiology more are patient becoming data, and there's symptoms, more with amyloidosis we wild-type that using ATTR echo and technetium of now penetrating so cardiology scanning aware. in many there patients then And patients community interested. cardiology many not future, we will the yet positive point

disease the importantly, Now we're more have. for also looking the side they human they are using seeing -- of in Amyloidosis the awareness neurology hereditary tools ATTR

see speeder all the diagnosis. positive signs of we and So diagnosis improved that

David Lebowitz

Right, I mean…

John Maraganore

neuropathy. mean cardiologists in multisystemic I the at are bear the have we looking for as is to saying, patients disease the what any what proportion they to to prescribe senses that important with neuropathy. teams of the neuropathy commonest and collaborating They're seen very on just awareness ONPATTRO. Yes, doctors, U.S. in of is if other certainly I've And But look BXX mutation. Barry's I've than like drug seen is there's to landscape, thing to many was mind colleges multi-functional increasing build significant that Barry fully trial spoken if those nation the a when characterize appropriate in have a

patients either who great is education absolutely getting and is mutations [indiscernible] have it journey, and characterized. awareness right, are other it's more fully that are a So, the buyers increasing,

for were So said than you offseason, patients ONPATTRO the understand and certainly I appropriate And whereas are physicians an is them. burden of choice disease. full important

So question? does answer your that

David Lebowitz

very you Thank much. I appreciate it.

John Maraganore

you. Right, thank

Kim. go Okay, ahead Operator, so

Operator

company the remarks. to questions. I'll have for the back all closing we for That's time it turn

John Maraganore

the Great, well, with and we've been that We're joining on progress for call. making. very thanks the everyone obviously commercial us pleased R&D

lives. impact are making the we're building, really about excited that we the We on patient's are company

So the rest months. great with that, look a forward continued and I of to progress in everybody. coming Bye-bye. our you day on weeks updating Have

Operator

does conclude today. that our Thank And participation. for you your conference

You may disconnect. now

Alnylam Pharmaceuticals (ALNY) 6 Aug 192019 Q2 Earnings call transcript