IONS - Earnings call transcript Q3 2018 | Ionis Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to the Ionis Pharmaceuticals Third Quarter 2018 Financial Results Conference Call.

As a reminder, this call is being recorded. At this time, I would like to turn the call over to Wade Walke, Vice President, Investor Relations to lead off the call. Please begin.

Wade Walke

Thank you, Cole.

including encourage Ionis go press financial website the financial I section the release measures begin, the related the of discuss of to tables, that we we everyone pro today. to to Investor and GAAP will Before to reconciliation forma the find our we results business. We financial the of our better believe pro our manage economics represent forma business and how

be slide differ I are our language details. Stan These and posted Financial expectations today, in and uncertainties, which slides our Business And actual statement Chairman our Beth call over on factors discussion for accompany today. like risk statements I'll the to and Chief Monia, three to risks also that the with Officer; With our and Officer. have forward-looking call to beliefs. Officer; on Board We are Officer; contains based discussed SEC the I which Stan. to website today Crooke, of Executive to turn additional Chief certain materially. we'll and would filings forward-looking on our me making your our encourage may which draw you Brett are consult Operating current Damien Chief McDevitt, Chief Hougen, the statements that, subject are language results attention

Stan Crooke

and in of launch and we results while third third Thanks, that our of Wade, WAYLIVRA. and doing and operating We're pro good financial In the on for to TEGSEDI achieved forma income, the a Company. consecutive aggregate quarter, number for joining morning, the launching everyone. this Thanks strength milestones, track preparing in us. year we're for important continued

top base sources performance revenue results including our partnerships. from from of substantial Our of revenue numerous SPINRAZA of successful revenue solid financial more growing R&D on

look we way, under WAYLIVRA. from and commercial launch revenue to forward TEGSEDI the this drug adding potentially With

to the sales study SPINRAZA infants. to continue reported from the grow nature data in new given Biogen pre-symptomatic expect globally, We positive

TEGSEDI infrastructure is Dr. the successfully our With necessary from this in we in are for multiple launch Prix conversations honor patient several continue only population, key impact of recently awards being adult countries, segments US we enthusiasm global Research the in in Disease those up Akcea SMA scientific now TEGSEDI. for our Frank of Krainer this growth of path and are in life drug the with community and fraction amyloidosis discussions continued active way are of make now see to the the and Spring we team, which ability has world, the with Franchise. launch sciences under Adrian strategy the Neurological again patients with with encouraged the place, a seeing strong such Positive we confident The to Laboratory. SPINRAZA community the and are Harbor the Cold EMA, breakthrough expect Akcea FDA. been also and recognized now also Bennett, of by our We treated. was patients as shared awarding prize with of continuing of recognized the Akcea's in by with and Galien WAYLIVRA, in with around Frank we to forward affiliate and Head SVP XX%

All are these progressing. of processes review

established Phase and dose-dependent cardiovascular first-ever the substantial demonstrated AKCEA-APO(a)-LRx, In and we program to continue the patients treatment potentially with the going the Lp(a). with EAP patients disease. all in X a is FCS, this and Lp(a) fatal of of disease elevated study Our well, to work debilitating in bring reductions ultra-rare, drug

convenient up drug cardiovascular longest the our study benefits of low-volume and and demonstrate monthly affecting the select outcome and validated reduction favorable a to dose we diseases observed study to cardiovascular APO(a)-LRx also of safety profile by in the largest to Roche to in and a the with XX XXX initiate range Phase millions have tolerability expects months. this of regimens. need worldwide. LICA target Robust Lp(a), of dosing Akcea, drugs the of adds X potential performance confidence Phase convenient our the treat to cardiovascular favorable year-end, Ionis to collaboration now We and with entered safety encouraged genetically a study the with with study. of we is Huntington new lowering history LRx. study Novartis, patients dosed pivotal development large We disease a to tolerability along LICA and program advance the a drug's on profile. with for what X patients disease driver study B today, observed Building a patients our of the factor natural Roche, We're and partner including IONIS-HTTRx very nearly the drug relationship doses. in into before using successful a broad

macular partner broad right maximize the and patients in the the potential believe of a the retain degeneration. value is example complement-mediated the level of drug's with success of For of stage we of is participation advanced of for the with program, to of age-related substantial Roche range treatment geographic beginning another We strong dry antisense atrophy, our success commercial this the platform. diseases

our desperately are important these more who importantly to new we've important advancing that it. numerous achievements need we which closure significant patients drugs other not financial had strength, Beyond only pipeline show to highlights, contribute but

medicines remains focus over continued patients call delivering Beth. to the need, positioning for while Company innovative new on now I'll to Our turn in growth. the

Beth Hougen

forma on We of Good morning, $XX of a months first income operating everyone. the Thank of million you, Stan. pro million, income nine with and net basis. XXXX ended $XX both

to results key this increase compared of royalties factors first months threefold revenue R&D commercial financial compared performance to the same with nearly together revenue of substantial were our nine in a revenue a XXXX, from contributing were growth period double-digit in year. SPINRAZA strong financial XXXX base the driven to Our by

will We catalyst-rich countries, quarter fourth six we results the highest ended means in success. which in for growth dollar revenue by cash. we approximately Last moved SPINRAZA next greater a as project are earn royalties quarter commercial in months, a global now our each With and tier we from third launched positioned increase. SPINRAZA Additionally, with sales royalty of be billion into share sales. $X continued of financial quarter, we TEGSEDI driven the multiple

to our product we In growth quarter. commercial TEGSEDI addition, contribute in revenue sales to expect the fourth

this We our AstraZeneca, also earned project for revenue the increase due for two we our to two program. the programs. upfront advancing to amortization million with milestone And different new fourth payments collaboration from quarter have $XX totaling $XX R&D our Roche of nearly million already from quarter FB payment

income to launch of the pro and investing operating on We in are preparing track even of while easily our meet launch TEGSEDI WAYLIVRA. to guidance forma

start Worldwide US. even drugs. The the revenue global grew out greater projecting of million growing SPINRAZA to the of outside year-to-date Notably, revenues increased now are of third We including X,XXX Ionis-owned a more revenue of in by EAP XX% and last We adult in SPINRAZA forms quarter-over-quarter accounting more us cash SPINRAZA pipeline XX% quarter segment pipeline for of for compared growth use XXXX. XXXX XX% of revenues represent billion patients surpassed increase expand driven the on patients new in with SPINRAZA year-over-year are the US driving than SMA nearly the end quarter to plan our population. the quarter. patients, number our by third second accretive adult $X to $XXX years. patients and to including seven US, US studies. in to cash the approximately SMA and the of SPINRAZA and cash continue second than being our $X.X and were greater advance approximately Adult treated for growth year from in making patients to six billion XX% in the In patient number with than the quarter largest patients a the on of the clinical prevalent

receiving are only Asia America. Revenue for of as about significant US the Europe, outside reimbursement representing in of a opportunity XX% and increased growth growth. However, particularly meaningful Pacific SPINRAZA today, Latin these was the pace patients

from the growth treatment to growth increases. US stability the for drugs significant collaborations component nearly revenue as numerous are patients in royalty continue of this to a third be with in That our the total which to quarter the result outside combined revenues successful of continues on SPINRAZA expect of quarter compared quarter of profit. fourth We US number revenue XXXX year the R&D in revenue increased numerous fourth and all in should SPINRAZA revenue.

in we've model. more source include significant in which and why So does our revenue $XXX source far have million far revenue we milestone $XX so we in this a million than earned in this is of year, is quarter. for us, the include R&D which earned payments the valuation revenue, revenue of R&D nearly fourth sustainable not

first the when partnerships; the significant second with $XX the our AstraZeneca in advancing first of program fees, a commercialize earned key million not nine $XX $XX a we Latin evaluating million four Phase does of of in milestone months which As the earlier of were license last upfront WAYLIVRA $XX This license Biogen months X/X treat I our and the amortization payments and quarter represents year year of quarter first milestone revenue milestone nine which million study collaboration cardiometabolic of progress one new transactions clinical study include from of $XX in our quarter this from $XX license revenue we third in the earned recognized end consists in We in the existing new R&D third they and full of Phase services $XX recognized quarter, to significant existing the is we America, the collaboration. new Two from this drug when with million with had fee payments; to fees represents partner's our during and drug revenue. million payments, September, R&D our partners. the year. provide which to AstraZeneca Through from this commercial we revenue amortization TEGSEDI patients discussed components: from and our include X for in ALS. PTC in from component primarily collaboration, for initiated we initiated amortization million Biogen million first payments. in in We for our

from Finally, our $XX million we services supplies for and earned clinical them. manufacturing commercial provided to primarily partners, we for

partnered program R&D we number Looking based to an factors: and on as advance. larger increase partnered partnerships, our revenue in grow in an increase payments the three programs of expect ahead,

Let me give some examples. you

revenue be as entered R&D We doubled Under than $XX Roche. upfront We in million companies, our collaboration quarter. currently in a fourth we the conducting patients Phase with have course we the six have we Roche, a received of the number second collaboration, into will with this Together be increases a with collaborations collaborations study that number increases. has the a X recently Therefore, payment payment. of more First, large beginning over the the will XX over we pharmaceutical atrophy. amortizing geographic with last years. upfront study

under we Second, are collaboration. and more programs our progressing more successful

and XXXX, advance, fees. eligible compared of million we have programs, far XXXX. that we fees we to earn revenue program a and license to we That's $XXX each this For milestone have and in license license $XX million year. XX has are so We payments partnered milestone related nearly since payments. earned tripled payments number earned currently In fees over to milestone

value our the and partnered we increase increases, payments in license earn the for programs advancing of as program. reflecting Third milestone amount dollar advance, the fees

to the will when Roche cost milestone payment. $XX earn conduct payment first in For When fund patient Phase we milestone the study, a we we in Roche the to help initiated pivotal $XX received million million doses patients, study. the X/X from a our study Huntington's example,

profit with the profitable the Importantly, $XX and conducting sustainably be In will all will accretive since a us to study. million summary, manageable be structure. expense we cash are Roche

together XXXX. growing in positions for the base financial position in With I'll to up of and a with commercial XXXX Our sets provide commercial to revenues turn that, our a revenue, R&D call substantial over us finish strong update. us to Damien growth

Damien McDevitt

you, Thank Beth. everyone. Good Great. morning,

U.S., EU progress with While get making it Akcea team is early the in to need the we is and patients pleased to Canada. in are TEGSEDI TEGSEDI the the days launch, in the

not Quest, patients. treatment sales lab you polyneuropathy will and is launched patients is service we of label provide keep providing more. in nurse in for together Akcea's program, And navigate as regardless monitoring training managers While dedicated on every guide case amyloidosis with through partner stage their has establishing patients their patients treating built routine insurance treatment enrolling the the TEGSEDI was pleased part injection how with have updated step been Akcea REMS approved have and home of U.S. with support was we Akcea's in coverage in of launch we of Akcea's a going. recently guidance of are prescriptions is help options, that the received. the CONNECT patient journey, progress and today, making option program of physicians adults, for U.S. hATTR the and to support field AKCEA first are disease. team services. connecting at-home broad good

addition, TEGSEDI partner with In along their team the well efforts market with is access payors. to

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hATTR Akcea Additionally, is to support drug we polyneuropathy place the of is to provide EU Akcea Connect out to first and patients in Connect patients. these rolling the Canada, in by forward to treat the look country. in optimal level caused for In each amyloidosis delivering

TEGSEDI we TEGSEDI's be robust Given their believe of treatment simple choice and self-administration, physicians. will people hATTR efficacy for and the with

and we US, to EU Therapeutics the PTC and America. Beyond moving in forward TEGSEDI forward Canada, Akcea look Latin

part Outcomes to beyond are more detailed the impact weekend, in analysis well health. also TEGSEDI. of Akcea our International regions Pharmacoeconomics Society patient's from the of effect positive on TTR and presenting neuro TEGSEDI's enable expand to activities, mental for These Research as to patient's looking as is to as meeting, This quality We study positive on day-to-day demonstrating these at look initial ability global access TEGSEDI's at changes strategy physical analyses life. complete and

confirm Now In we In is turning our to the EU, work WAYLIVRA. process to regulators review plan ongoing. Canada, forward. path to the US with a and

to to approval. with as launch ongoing the program quickly treatment continues Akcea Akcea As and possible the first prepared assuming work ever is EU bring in to the FCS, EAP to is patients as

FCS on Akcea for of addition, diagnosis is support with In to a identification provides progress focus which patient our making with estimate X,XXX worldwide. X,XXX patients

from our key Now, over to to highlights review pipeline. Brett,

Brett Monia

Damien. Thanks,

look month. detailed from in forward update an at provided with So, and we Investor now since next I'll briefly, we Biogen providing quarter achieved infants update exciting to recently Day study that several our additional the SMA. NURTURE update our successes review pre-symptomatic second a

need the of May diagnosis achieved without all were SPINRAZA of of cardiovascular that continued normal ventilation, Shifting And As bit, of provide it and of has Phase adds levels to evidence were to of pre-symptomatic Lp(a) most elevated established data the gears SPINRAZA course were AKCEA-APO(a)-LRx, standard reductions for in for in disease permanent importantly, patients fundamentally consistent and progress study XXXX, all with can in milestones SMA with care group alive active with in as in Lp(a) achieved every study development. early the the threshold long-term for These with sitting would further a dose-dependent all participant X levels be with patients this patients treatment cardiovascular infants the to this SMA. risk in evidence disease independently, attaining below supporting more able make of all and study body nearly patients established the for this in alter disease. walk.

favorable Additionally, consistent profile LICA safety drugs. and with demonstrated our other APO(a)-LRx a tolerability

durvalumab, disease exercise, a levels. and X as patients demonstrate with preparing they end-of-Phase established experienced are preparing to by cannot importantly Lp(a) which the lowering and from elevated and option. an up of for important the treatment Shifting royalties cardiovascular head and this announced to drug. The program partner they our CV our the to success danvatirsen, initiate AstraZeneca's And with year. regulators, to approved presenting conference. with therapies highly no initiate closely target with study end and of blocking documented, X response. STATX innovative commercial data meeting generate at Novartis that of rapidly XX% large previous Ionis is pivotal estimated patients Huntington's AstraZeneca of in with Roche with Roche positive include go they enroll alone, two disease data PD-LX reduce attention, worldwide. or controlled difficult Lp(a) study of this as response the outcome either benefits a the As as a levels We tumor modifications XXXX. patient the to on early study XX% team robust the possible. double a a affects once in to and specifically durvalumab there IONIS-HTTRx is the of to partnership tumor a designed Investor before lifestyle treat look based well to which design is our value We partial population. for complete year's AHA now And that highly cardiovascular complete this our in designed large millions X% It driver reported well of been partner Akcea studies to X is antibody in will such followed get plan levels we be This achieving FDA inhibitor conducting initiation at forward this of for disease. neck In course, people to or cardiovascular patients people exercise studies potential cardiovascular of that achieving the program actively rate be beginning response month. by study recurrent and will also. recently are retain Day study Novartis, be the in with Roche has the continues the Phase resulted ESMO with significant a outcome in to Lp(a) low next set. at combination our have their Saturday range, with Working cancer medicine with data metastatic additional Phase studies, pivotal diet The

three-year study X patients reduce will to XXX up measure be amount of that first world's causes effect of a around study will the The drug protein globe. will the disease evaluate patients. Huntington's in the Huntington's which study symptomatic This is efficacy and safety the Phase in directly a long-term first to the that disease

XX-month second new in designed the history to correlation to up symptomatic understanding study protein changes our XXX further of huntingtin between study is a in natural The clinical patients measures. and

first [Indiscernible] drug's clinical together to disease As a with the a which lowering substantial or potential IONIS-HTTRx huntingtin is progression. in protein reminder, benefit in to new the disease support only patients drug half the slow demonstrate perhaps and of Huntington's

treatment with with of of commercialization atrophy, with a FPL actually recently of for Roche broad geographic stage beginning for of AMD. advanced people successful development upon mentioned, our diseases the collaboration Stan range relationship the complement-mediated dry new with Ionis As a and built the we've or

pipeline, IONIS-FB-LRx the that a diseases. requires renal mediated is our particularly commercial developing while of and specialized experience. substantial well-suited that indications disease commercializing development in Furthermore, prepared to medicines for and has Roche success. participation renal complex see each requesting infrastructure for maximizes geographic As is partnered a in When for in atrophy development complement with Roche the IONIS-FB-LRx. success our reminder, for strategy and drugs drug's commercialization optimizing we product experience additional and pursue disease for

had we data XX% database our to LICA greater up safety a target the of and we or in we these integrated commercial frequent substantially well. increases year. to studies, LICA studies, also full for drugs of production. this LICA other potency APO(a)-LRx And are good demonstrated In patients royalties safety to programs, participate drugs less results Phase the clinical volume up the nearly performing potent XX-fold coming for enabling of LICA from treated our showing the drug We've those observed with in tolerability dosing. more study have These look in exceptionally months, of programs, the drug. clinical reserved in forward generated of are our success XXX consistent low which in with in our and X Importantly, and publishing

since with LICA IONIS-FactorXI-LRx Xb of we our IONIS-FactorXIRx XXXX. enrollment in X had study additional and many dialysis achievements of a We of disease half quarter have Finally, a version our end-stage initiated update. Phase in renal Phase both second expected second the study of completed on the data patients with

is patients our is X familial with the data initiated in under and a highly which a LICA generation IONIS-AZX-X.X-LRx, study X/X Phase in SODX development study to three X/X ALS drug expected ALS with of clinic And IONIS-SODXRx, of in XXXX. one with in Biogen our second mutation initiated AstraZeneca is cardiometabolic AstraZeneca. which disease drug productive to renal Phase which our addition in Phase familial collaboration study a of drugs first IONIS-CXRx, X.X the enter QX

into In decisions, this XXXX, and study completed several catalysts to on we're slide, initiations. many successfully important regulatory shown data we looking this fourth and As numerous milestones the quarter year. forward key including readouts

forward We Day our now, call of discussing Stan. I at to these the you, over Investor And December. also in look back to will turn events many

Stan Crooke

Thank you, Brett.

for strong very another the third quarter the So, Company. was quarter

power and launch preparing we TEGSEDI the that while financial model financial continue WAYLIVRA. built of the continuing with even demonstrate the strength to We of launching

FCS Regulatory a therapeutic positioned positive options. process profile are -- patients authorities as profitable we FDA believe well. we we as conversations WAYLIVRA the to be Canadian with for launch with no once substantially continue to are we the continued we suffering believe growth. have we territories. benefit/risk WAYLIVRA are in in for with So, ready demonstrates other and and Akcea and quickly possible in EU is and approval continued the as EU, We

have number of fatal near-term potential disease. the including These the TTR the of cardiovascular initiate to well amyloidosis, studies year. least Lp(a) such change diseases three to diseases, a that before are course plans such genetic millions as We of with pivotal as Huntington's at of affect drivers end value programs disease, driven patients as as

programs We programs therapeutic the also have Cole mid-stage With that, that forward a to progress. turn areas number in drive of have set to they would to Q&A. multiple discussing these to up we're more excited you We please us for us about growth. set the look over up? potential as which I'll longer-term Cole, call about

Operator

[Operator Tyler question. Buren Jaffray. go with Piper Instructions] from question with Please your Van comes Our first ahead

Tyler Van Buren

programs do sign the they of familiar guess, going it have questionnaires just first REMS them? are that simple I to that TEGSEDI understand easy aren't to specifically the XX with I as to logistics how one with long REMS do? the less could some that I How to to than wanted the online questions, are was with but noticed better is respect guys take? process. how actually helping quite And you them up? regarding be docs program, who guess What specifically for for

Damien McDevitt

Yes.

So, Connect this is paperwork work and Akcea REMS up to we've program. patients associated help the physicians through why set with the

through certified registered physicians We've going and patients to no straightforward date, there's paperwork. very REMS through And and it's had on spotted, you Akcea these been as Connect. issues. So,

Stan Crooke

of the Tyler, the physician's so and I Just actually the to went at add reducing physician lot office. the to patient, on and process the through burden effort a there was that everywhere on myself,

the easy feel enroll in it with so Akcea together. And program to that has very, I how very uncomfortable is put

I advance think exciting these in managing quite an it's processes.

Tyler Van Buren

Are are on are to how you REMS or certified many the patients on the many say REMS? how able doctors

Damien McDevitt

not providing we're numbers. No,

We but at not the giving and have numbers exact physicians we're point. had out patients this certified to-date,

Stan Crooke

encouraged encouraged by what We're what received. by We're received.

Tyler Van Buren

monitoring. but or assuming be it a the that can maybe question with that to by platelet And on do how I second can't I'm weekly samples the platelet me levels How on basis? the to would how trip you if guess, take? the someone respect will need wrong, was patient a nurse I'm correct And themselves, happen? if it their you taken is will and logistically, it and specifically monitored a long get business

Damien McDevitt

the done at the that will monitoring will So, help. be with so home, and nurses, does platelet happen, draws and blood

Stan Crooke

So, a easy. an It's of go and spend a getting designed in providing there. set as job and of convenient rather us At near done. And a as do go. wants day for in platelet just over are patient minutes. and The get clinical in, matter go traveling, would IV blood drawn Certainly, and where myself, course We'll is have a to prefer Great the there be they couple monitoring a once of it we're it's why, are will of the least, an possible. I rapid center well. is week, infusion course, in opportunities so getting so speaking as infusion. drawn patient their everywhere, the there blood if and to and that to home they go the platelets a laboratories going the determined the up that patient can or lab

Tyler Van Buren

I hear bit patient's guess a business some you I about to don't trip little on a you're nurses nurses guys large to guess have plugged network? the be staff if having they're Just are, if home that. or they wherever of location curious to lot into on and or go a have

Damien McDevitt

Yes.

we Accredo, large the when the through plugged network So, US. there are on into and are throughout XXX staff nurses

available. network large fairly that's So

Stan Crooke

Quest. And

are Starbucks, partnership so but it's Starbucks, almost Quest, with we there's have a It's course, that a not Of shorter. the lab. except lines

Operator

Matteis your And Please our question. Paul ahead next with Stifel. comes question go from with

Ben Burnett

Burnett Hi, for Matisse. this on is thank you, Paul Ben

for what constitute would you able SODX this sufficient you as Can question of enrollment the And the to upcoming advancing Just supportive remind the then as XXXX? level knockdown us the And you extension discuss a on data secondly, just I here. guess, level assuming path program, in readout you the study? to speak of guess the I of forward program? are to can of regulatory positive

Brett Monia

question. I that that -- will take call

So, did as the for levels. levels like I reductions think the Huntington. to market SODX we much for in you the validated in CSF Huntington inferred, we demonstrate We measure where can CSF we SODX were able program,

So It's much on the with ALS. pre-clinical order shown of symptomatic reductions XX% on study a clinical mutations. we ALS like of that in have with XX% animal SODX the that part SODX models will of Huntington study. data, based can to be -- patients in And three-month significant a benefit

of halting types reductions of in animal shown disease have models. those progression fact In complete SODX

Regarding the X enrolled. But I study. looking data is And data. the for study. first you we study study, the next hard as see to study as path, And study similar the we're how quarter went regulatory the it's as the know, at in Huntington of And from Huntington to Phase design year. readout Phase very the say, to mentioned, pivotal X is

it's that that data based move outside Biogen not in So, the comes wrong would possibility on out, that direction. the

Operator

& Chad question with ahead comes your from Messer with our Company. go Please And Needham next question.

Chad Messer

ranks And danvatirsen, any in with X of next that of along on other STATX can What indications? the of terms have might idea I program. other when that maybe join the programs? the one some from we Phase expect

Brett Monia

program great Yes, in our really showing program our oncology of -- lot is immuno-oncology general a potential.

I is clinic. and refractory in just encouraging recently reported recently with other another lung patients was study -- drug highlighted And program. oncology neck started And STATX in cancer. to and cancer in gave the Our the also cancer, that's question, this that as AstraZeneca to indication our first-generation results head combination way. It bladder in we've AstraZeneca That combination enter that we has now the with molecule X.X non-small leads for presentation very your a the script indications with produced exploring steady cell in durvalumab. so

AstraZeneca could make of planning months, the a to on be and the cancer for study. and collecting head well more And combination durvalumab. this in is as refractory few stage more which next head the with patients in neck in in second the study, next development decision data study, in first line line cancer over neck program certainly a as and pivotal they're the

Operator

next Wang Barclays. Gena And question. question with comes go our Please with your from ahead

Gena Wang

impressive. World I One NURTURE very the SPINRAZA, question Muscle, data regarding was know

Just to patients? next step? And quick the any WAYLIVRA, for a regulatory question the wondering be regarding pre-symptomatic step what plan very the path will expand to for label the the then next FDA

Stan Crooke

to label are Thanks. SMA. SPINRAZA because And more administration and so babies certainly of supports any with positive. more presymptomatically patients overwhelmingly sort The of treated data are SPINRAZA the being

as speak. we happening that's So

Damien McDevitt

patients course, just the incorporated presymptomatic measurement Stan, now newborn SMNX the nationally that develop of Gena, identify as that's the that now incorporated officially newborn and genetic panel are And, know screen you screening into And as has nationally in to add becoming SMA. will as statewide well. a to system to that a

patients genetic and So treating very was that data, presymptomatically the World as more more both data NURTURE the as the presented all in well bodes this for testing well Muscle.

Stan Crooke

get the to infants The patients better treatment it's other and sort but it's that the less in and see continue and is in are to NURTURE continue as better. that and of the -- impressive forgotten, that's of patients me thing just two same not behavior well. would seeing been we severe study tremendously in We're adult seeing asymptomatically we're sort treated it infants type that the to

the incredible treat, SPINRAZA longer SPINRAZA. for today it patient delivered we has been treated And essentially types is the the value. all that have So, with better

So, it looks quite exciting.

Operator

Fye question. your with with Please question JP from go Jessica comes ahead Our Morgan. next

Jessica Fye

of of us Also Is when dose and sooner label? I've given dosing could to I incremental months XXXX. completion talk update it's Can clinical the that in see we we frequency in can you levels open extension trial? about December could it that Phase you possible XX think treatment X/X the said see help extension from trials trial? data like think with Huntington's it's

Brett Monia

transparent data X/X believe that it's what extension, share actually sharing data label from community cadence the patient to to course transparent, going XXXX to in -- but the so data of Jess. I physicians, the and they've know information patients, open study. was well was they'll and as provided. the monthly very the is treated, are been continuing well. Roche doctors, when Phase Phase well which the dosing, study. study patients administering has and are is over specifically with very X that is label The said. Roche extension into they're how They is the enrolled going been open said as be it closely that Sure, going top going. and very not important program very be same in they're the and to the study, and worked the in has and the dosing it's dose is from because obviously haven't All the They patient and share has we in advocacy, they're

Jessica Fye

missed primary for be Phase specifically Okay. two-year that but And I product, apologies, the endpoint? Xs this, if efficacy will for clinical what the

Brett Monia

EU, measures, MRI by EMA things, it's clinical the posted. endpoints brain FDA, So It's sorts fully -- different slightly as It that and measures. of involve US scoring EU. system been scans cognitive we're through and and function quality-of-life composite well have now disclosed as the size, of in measures, been a motor US going in neurological of those both but They're and now using and been measures autonomic to the are is vetted functions Roche. it's

Jessica Fye

And is last they SMA. with some are me especially Great. and yesterday one for comments just modulators on splice combining focused gene made Novartis that Okay. not therapy

reaction view? So to leader curious what as SMA was in just your the that

Stan Crooke

in are the in use value given to Well, we to results we me think hard SPINRAZA any future we and to are for today. that do leaders gene add in of there know a agent is would variety opportunities therapy Whether that a SMA, have agents the combination.

combinations. I'm in to of at with some SPINRAZA value, are spot progression of gene as to there watching doesn't sort we're There's that bring Biogen drugs appears development the therapy and remarkable which as So, of. in small any be And nothing others combination any sure, preclude of the will we're aware the well molecule. look that if that

Operator

next our Wells Fargo. ahead question. with And with go from your Please Birchenough question comes Jim

Yanan Zhu

Yeah.

Yanan Sorry about is Thanks. that. for in Jim. This

regarding schedule such So, change versus in question factors patients? or as describe number some enrolled So Is SPINRAZA that? the you growth? could patient other intervals the it first for driver in on color revenues, previously building the longer early any patients

Stan Crooke

It's numbers. dosing change There in schedule. hasn't patient any been

more patients that treated. So what's happening is continuing patients to the treated been and are be treated, are have being that

with -- being patients correlates more So patients are being I are benefit. More treated just think it's treated and longer.

Yanan Zhu

an be to described increase adult in portion pediatric Got stable? able comment whether it. you whether And of that patients. the patients you Would the is market

Stan Crooke

It continues grow. to

Yanan Zhu

just drugs? And tag for potential on mentioned possibility between two combinations ability of it. therapy. with that a potentially gene about therapy. to impact gene million and I $X $X SPINRAZA the view how price What's and million Novartis talking combine combination were the you their gene that, the Got thought therapy -- your the might to

Damien McDevitt

show to drug, than And That's an to better compared of to Well, would it's of that majority impact like SPINRAZA. who do be do benefit uses fact I'll very I its discuss days. combination given I the early extraordinary Novartis obviously pricing think that? How normal to developing patients patients are it. treated going price to combine whether children. sense would vast the make on have do let and difficult presymptomatic you were sort their any whether think At and that

Yanan Zhu

Would be a study or study by on the last whether to Roche. controlled that able you study? the the the X X Phase on design Phase XXX-patient be Huntington's And disease would comment trial uncontrolled question

Damien McDevitt

a yes. It's it's controlled XXX and study,

Operator

question Bernstein. Chen ahead next go comes Please with your Vincent our And with from question.

Vincent Chen

market using strategy partners compound the biomarkers, for SPINRAZA the Could just with with more et Biogen are of you superiority here? about think So, potential SPINRAZA whether novel SMA you defense and oral profiles us areas how building to with whether cetera? up provide how sort trying in demonstrate defending I color and the on or follow-on SMA you potentially future with the agents SPINRAZA thinking of that's about a next-generation example about of guess, one question against and with market, your Roche's

Stan Crooke

Well, we I order to more the to first in the the all is more to appears learn continue drug think better And about SPINRAZA. the today patients. business of be learn

course, to less or SPINRAZA that Biogen at we with have dosing. might are lower might look of frequent have doses to Second, collaborating follow-ons various

used we used, whether how they is agents to might might year. watch I be on needs think oral combination and comment happen what used now, before does be how next be one to the over SPINRAZA might in

continue that. watch are in evaluate We of progress in second, to then clinical and to benefit trials And both terms safety. the that

there's small and the not data. about we're really think, molecule drugs know looking very I the forward yet much seeing to

Operator

Baral Ritu from Cowen. with comes question your next from go our question. Please And ahead

Ritu Baral

quality X any within talking endpoint to primary guys before, that were a it to endpoint composite back was we volume autonomic, requirement of for are think Going mentioned, the How there How the effect certain functions motor that Phase that to the endpoint I those for and about element you does cognitive show positive? MRI Huntington's life. Is composite? composite one weighted work? threshold be endpoint,

Damien McDevitt

get weighted. I Yes. don't of those don't that? we on components on Ritu, various score -- we will have to in to back us to you are front details the Why composite have how the

Ritu Baral

on progress between as a that Any you you they progressed there as process? been made days? follow-up remind quick details Europe. can are and questions you on then have XXX where XXX like XXX-day -- us And that in Have WAYLIVRA Can share? you the received

Damien McDevitt

and process. the and in meetings of to We of -- questions a variety conversations number do a late and we've XXX-day others. We're had responses

in next and to we're it the bring conclusion we're process looking So quarter. here the a late forward to in

Ritu Baral

fast if I just here. And wasn't writing apologies enough

have commercial have do commercial have that accretive treated. patients you licensed? German you know already to You on commercial patients be I going is TEGSEDI. Do just started? treatment imminent Or got it patients given US

Damien McDevitt

as we and and We and where we And physicians REMS and registered Connect. are today. have on specialties certified specialty scripts. is Akcea That's -- with through up have their our running. we through have we REMS first received And meetings and patients of and well are physicians certified teams set pharmacy that the

Ritu Baral

X topics to plain vanilla Lp(a) boring just aspect Are outside discuss that boring X. pure there of sort the end any and And meetings Lp(a), we're that that then the Phase Phase I is worth are sorry, X given endpoint guess have relatively a -- you last compound of on just really Phase or expecting question all will noting time? other Novartis of the

Brett Monia

little We're It's potency the into data but that I a will to be make is to outcome excited an of described tolerability. the have have in question, have about we really, to late-breaker that's heard there. in sure leave the as the go we everyone about detailed agent of I'll as here. share I've encourage safety discuss that meeting study good both well would presenting. think appropriate detail. relatively We're time a Sam it's the a first really the that Tsimikas but the of kind more I to That's this I'm terms little excited once Saturday all I as coming than think, just boring, and it, OK. more that it we

share And more look community. so much able to being detailed forward to the we data with

Operator

next with question go our question. Jen Company. Yale And comes your Laidlaw Please from ahead with &

Yale Jen

I a have got I've been And think here. two lot answered,

that Could that NASH may both DGATX for the have The differentiate releases and regarding is drop the the these the have ANGPTLX. a is you specific one two the target. data later is indications first two NASH. the you other One

Stan Crooke

that Yes, interesting question. is a very

all We above. component fibrotic have the reduce of the of NASH different NASH specific quite either highly I way of think is that or the triglycerides component it's in The liver. lipid on the to of or DGATX pipeline agents a focused

in trying some And dyslipidemia thing. so of lowers that of LDL that as it We in we know triglycerides. that should of such other very we high issues without benefit substantial liver that With general LDL, any think the that's of sort of and sort the know a know it lowers reagent lowers it specific ANGPTLX increasing that drugs mind. a it as fab We agent ApoBXXX. our a have that

in in of elevated at be question, could that It And triglycerides. be reduce with so triglycerides used for look a in NASH. to we'll be use patients people agent you a moderately a and the a treat significant could reduction does to conducting with treat It candidate in triglycerides range as of dyslipidemias liver? used could drug broad to asking that general we're it to the then the the composite that we're translate benefit And study grams.

that be primarily, triglyceride way liver general That about DGATX, and with So drug specific NASH we more the if about the you will very is selective think it. a think I opportunity dyslipidemia and drug pursue. among as a several will ANGPTLX is

Yale Jen

of in So process not would but to specific fibrosis say NASH, the that the progress the necessarily may be early fibrosis be a to -- disease targeting into condition? more that was into

Stan Crooke

and correct. driver two and These the on are disease of the reduce that as affect the lipid we the disease, the will that's the progression focused we of Well, drugs we triglycerides, the so progression believe which is lower of liver disease.

fibrotic agents process are well. as focused We have of other that the on are the part that coming

different power should different the of of sliding demonstrates us So let of our selective we We And And down data we component the then multi-factor quite complex the is at best also next of it looking We each to this of these what each antigens. can pathways, causes pipeline is parts where really bit, speak little for of opportunities. -- these data one going are each diseases. and be our parts we to think drugs in this be exciting. that and the doing have drug. of for targets, over patient part different the tell population target of of the drugs positioned create

Yale Jen

maybe Yes, more Huntington one the disease. regarding question

or you're study kind plan mainly and And focus also study that. Is You and of the the does symptomatic have readout that is on of sort say study -- even some maybe first second proteins. there's studies some the that differences fully Huntington enrolled. reduction before the two the times over of but monitoring

Stan Crooke

detail. been extension open Roche be progress providing do And we by in said, No, although believe Brett the That's we've I and updates in on study are encouraged that's can't speak we Roche as label correct. the for will seeing. that XXXX in what more that

Yale Jen

the level the drug a that cohort tend -- does A be is And different and -- have there as administrated. to B at of is two different

Stan Crooke

label open at highest extension going the simply think dose. I the is

Damien McDevitt

Correct.

Operator

ahead go Lebowitz David from question. comes your question Please next our with Morgan with Stanley. And

David Lebowitz

getting patients? REMS run whole on expectation early actually regarding is a process taking to of regarding what run -- reimbursement, understanding patients respect with what through then Thank you With and you TEGSEDI beginning yet the meets paid us what and discovering of the reimbursement pieces TEGSEDI you when question. could requested potentially And information eventually prescribed the very finished much beyond with to for process and in payers initially the my the that, types time becoming your patient process have it haven't I turnaround drug? for that guess doctor from reimbursed

Stan Crooke

met the the cost. after highly it that payors focus extremely these conversations and our There easiest a process the you before detail to of many the the very sort well. and colleagues suggest understand. to manage was primarily entry really have patients. practitioners' And And to versus patients I understand of of the I treatment the treating those benefit know gone focused Akcea and is that taking would of program into payors robust and the have give that where sense are I advantage my at in Akcea. Akcea expect can who the Connect going taking of launch. lot you and on in getting be of possible, into with you questions care with shortest facilitating time what I time patient practitioner those And the process. I'm the the a address of

did we suspect be start while even gain but short, further. won't lot until the more how a we'll you a between able as experience, and exact shortened I'm it's going to we to And that sure tell experience. just can't I work it's be for time all And really it I we finish. know So,

Stan Crooke

we'll will growing you provide Day detail SPINRAZA are commercializing at to we at being the two significantly Investor in forward question, drugs which to your today. perform APO(a)-LRx our tuned that you've that we're about new we New presented in pipeline for for Once progressing in performance the questions to and stay I like very Investor we're and we're more witnessing while doing We continuing the that's that our you telling call AHA. last thanks Thanks seeing all us and everything exciting detail we're to the everyone of cash appreciate If Day. drugs more excited I'd an the on that the to the is advance joining asked. and update really a close. that think And continues in we're in our is well, manifested a our technology. have in in encourage operating while so the Yes. very to bring improvements again, What deal much be opportunity doing. interest accretive. that this look advancing Saturday board, the York profits greater story, to forward technology to much. it We're We across we're our looking to essentially

Operator

now The you attending today's concluded. has presentation. conference for Thank

may now disconnect. You

Ionis Pharmaceuticals (IONS) 6 Nov 182018 Q3 Earnings call transcript