IONS - Earnings call transcript Q3 2017 | Ionis Pharmaceuticals, Inc.

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Operator

Welcome to Ionis Pharmaceuticals Third Quarter 2017 Financial Results Conference Call.

As a reminder, this call is being recorded. Leading the call today from Ionis is Dr. Stan Crooke, Ionis’ Chairman and CEO. Dr. begin. please Crooke,

Dr. Stan Crooke

expanded the the thanks, demonstrated to and filed and joining on -- us preparing We to EU opportunity uniform business program progress reported commercial accomplishment TTR manageable Good detailed the had inotersen. our with by has our we’re the Yesterday, safety data accelerated everyone, third launch spend time inotersen. week. investigators gratified that call advantage. a We’ve in year last and and last made past an discuss In results week, in an team we substantial We’ve Phase provides with date we an our also inotersen. benefit quarter important of today’s we’ve additional competitive of highlights. for substantial in morning, we displayed MAA meeting announced the for for access that patients past X XXXX recent a assessment that more evidence and follows in amyloidosis We’ve had NDA, deal last profile. an that to that an XXXX provided study we week, believe from announced strong filed a enthusiasm well-defined strides filing initiating financial great the are

of history. for through for pipeline successful is is progressing no its current a story. suggests which the therapy ATTR We’re on part The is optimistic also one that success. STF, rare find approval to commercial a SPINRAZA also and launch after Inotersen of for will inotersen launches not inotersen sales most date significant be of rest disease horizon. in registration a the of that one market there therapy and a our only particularly will but significant no nicely. achieve What the is only serious medical, disease progressing our fraction exciting is be drug I

forma growing income and coming operating we pro with profitable of project million, steadily the in revenue a We’re $XX years.

billion $X in are financial meet very values. potential We or have short, we’re strong exceed position. track to and on a than of cash our more In

where As morning, our been in planning next of we set for to with XXth This this Adviser management transitions become years, Ionis. Strategic transition planned she’ll succession of Lynne on of our Parshall, She’ll remain you that January successful. structure. has Directors already likely announced -- demonstrates remember Starting have year, seen of who’s member Ionis been a role, our a be a will Akcea. Board XX in significant relationships of indeed the planning, Lynne new will to I a In role Ionis forward. and strategic company Akcea. will important she’ll involved regulatory going and continue on has her with and the and always the brought value think the she And most in short, are the development, areas and focus in business that needs play financing, most Biogen areas

Ionis. have nearly years, I For Lynne worked navigate and to the for course together XX

his and her of very devote been years putting through strategic personally leadership vital will of Lynne more Chief many me. become has Monia, and to been inotersen. many Officer, in discovery of as is and for and So also instrumental team, instrumental continue to she’s the being founding substantial role up our family. I productivity and my work good the with achieving members the friend. managing Brett Lynne discovery And Brett close to thank hard currently the played times me of reporting and He’s want bad leading of a being has is one to program. Senior I’m play Drug able key success, in pleased Vice played, the partnerships. the drug to our role progressed also development to of more Ionis Discovery, Operating that who President Brett the we even always an time will to while

our Additionally, a the Chief fully at Ionis. responsibilities confident be decision-making of prepared leadership that Operating and an choice Officer he’s to step to ideal contributed long-term in of his are and and Brett productive assuring board building continuity to new that culture is approach is and Ionis. I important step is the this The at assume and

Chief Vice you members Now, And team be Chief Corporate Beth Relations. Officer; Communications Hougen, Brett Lynne President, read let us to management President, Chief Officer; call. be Sarah me Wade, Wade the Vice forward-looking today’s Drug statement. introduce Discovery, on will Financial joining Monia, of our and Senior would Boyce, please now Walke, Investor and who available answer language will Business questions. Operating the Parshall, Officer;

Wade Walke

effective materialize statements. subject certain expressed those forward-looking results intentions therapeutics Ionis’ Thanks, and projections, commercial and commercial its potential in including should and forward-looking Stan. and is of of business, and also products statement a the cause forward-looking Ionis’ this they statement. Ionis, if business for statements involve Any inotersen financial expectations, endeavor that SPINRAZA, of these materially considered call correct, or use building regarding risks implied are financial uncertainties, differ a for forward-looking statement to developing goals, that, to therapeutic discovering, or Therapeutics and development. conference includes outlook the never Akcea inherent to business could A everyone the reminder potential be or prove by commercializing drugs volanesorsen, technologies are and from particularly of that around or statements at-risk of process beliefs, safe other such assumptions Ionis’ any drugs. as the statements those and in an Such the and describing Ionis’ human

of Ionis. on forward-looking based these facts good-faith statements Ionis’ and by currently reflect its statements Although factors known are only management, judgment the

rely XX, these XX-K other As Ionis’ available which risks And company. report are a like file annual additional result, in These Lynne. to now is you’re detail over for on described and other these filing with quarterly from the cautioned documents the of recent I’d the Copies most ended Ionis’ call to on the not to and forward-looking concerning SEC. XXXX, are turn on year programs statements. in the Form December and

Lynne Parshall

Wade. We patients grow excited to to of Thanks, SPINRAZA. to well. The helped continuing SPINRAZA being humbled go see by very I’m launch by continues the globally. the of stories drug remain

$XXX one million, SPINRAZA Street million by sales and year-to-date the quarter Wall to sales Demand successful on that quarter. second track Third be is compared therapy of drug third in the history. XX% remains U.S. to over the With XX% of by of the reporting consensus. $XXX with launches quarter patients on rare number in exceeded most strong grew Biogen disease second of in quarter over the again SPINRAZA grew

received yet that forms Importantly, on it start further strong for who Biogen demonstrating patients reported of for SPINRAZA. hundreds are demand therapy, not the

study this last new which Latin from study in and approved America. provided World NURTURE ENDEAR recently basis Sales SPINRAZA the sales additional infants. the of Congress the onset additional were in had particularly that U.S. significant study, to Biogen Outside meaningful the motor sales in it EU make continue with SMA. of presented and gain quarter. initiation reflected Middle growing had also substantially an that in said been Phase SMA SPINRAZA outside first of full the $XX increase higher were of the team third of congress, its just This strong, commercial U.S. disease. SPINRAZA the data, U.S., contribute EMBRACE quarter to in analysis program with million is Biogen in Society sales, evidence globally, X is can the a function in now plus due quarter part the Biogen patient measures to At a infants confirmation Japan expanded of first make in the broad of can growing along on presented demand the interim which SPINRAZA. SPINRAZA of at reported difference at the sites To demonstrating XXX administered patients and in age access body the of centers plans this the start recently, that England gain demand earlier a infantile and an in number of with publications third was as and New the to Biogen of the the have of second Another end and SPINRAZA, quarter, of prestigious the SPINRAZA, U.S., submitted in to such the These lives in XXX East analysis help double recognition quarter the Lancet. and administering stage Switzerland, of pre-symptomatic conducting in important regardless treatment, data Brazil than in Medicine indication with growing from selling sites benefit end in sites also named in at of of have Australia. including XX is their is size the XXX forms. has to and a Muscle on U.S. month, of SPINRAZA SMA, a countries access Journal patients continues EU. SPINRAZA the or that

late Product in truly Galien many month, industry the this were recognizes panel by and accolade state that to recognized our very be the considered the Biotechnology Biogen of SPINRAZA industry. prestigious USA XXXX, for The the achieve. pharmaceutical was We the and innovation what by Prize be best and research influential Best SPINRAZA Prix It’s health. in of which last Galien improves extraordinary scientific Additionally, achievement human an is event awarded in Prix the to believe our highest industry’s honored of distinguished equivalent Nobel for the represents can that development. award and

I’ll the of this and patients, ATTR their program life touch detail of Phase significant for disease benefits baseline. that the in and it quality quality combined in patients NEURO-TTR MAA with briefly. superior benefit its believe last to inotersen, in choice we at In inotersen-treated study moment population. to We NEURO-TTR this of from seen patient the discuss X neurological convenience, Phase patients Moving study, X month, NEURO-TTR on data In amyloidosis measures study, on and of life reported with improved experienced in their meetings. XX% a but the inotersen inotersen-treated Sarah treatment the the more placebo-treated measures in make could from the positive compared will inotersen European of

list made objectives, of and we we’ve outlined, As progress extensive have of Stan substantial near-term all in already an them.

partnership, in complete franchise commercial with commercial important joining To a also along maximize our of commercialization partner, year, potential expanding well we’re or addition, and our an and ourselves combination through either revenue build convenient commercial the source We ATTR In beginning to inotersen of to next be the could with two. a IONIS-TTR-LRx. potent plan believe of successes we’re the through through SPINRAZA discussions inotersen on more our inotersen, volanesorsen.

ATTR. a program clinical with patients implementing for streamlined We’re all

Our the goal is to year. next before clinic IONIS-TTR-LRx of advance into the end

authorization Moving Akcea in on EU, for recently and Canada with submitted the patients for treatment the to volanesorsen FCS. applications of marketing volanesorsen, U.S.

building model and should made next support lead developing trained volanesorsen leadership skilled is enhance to will beginning commercial volanesorsen. its well nursing promote These help patients Akcea’s believe could long-term venipuncture therapeutic organization for Seventh continuing stay a commercial programs with managers of on to with revenue and and We case be compliance with benefit including believe CBCs device platelet replace year. include also with patients the programs to to markets. reimbursement optimal also boarding at key from We which professionals. will should patient platelets. home therapy. an source build painlessly this an on as as volanesorsen. of measure help next-generation to Akcea’s important and We inotersen investment We start global believe experienced simply device Sense, treated monitoring recently being use convenience to which this

This available we’re to XXXX. We’re make several working with commercially for platelet patients our to more patients. Seventh convenient device to one monitoring approaches in Sense this pursuing is make of

should its towards X advancing commercial drugs over to enter potential APOCIII. dose-dependent the of of the have study compliance. represent also with recently in in Akcea’s cardiometabolic is drugs next opportunities and of and development market drug next on in data high and mid-XXXX. dosing, LICA late-stage less LPa Akcea in potential support demonstrated progress Phase in from planned of reported the Phase development schedule. well other Like is years. the in progressing drugs. patient convenience triglycerides observed with clinical reductions X/X potency and enhancing frequent moving positive substantial data the forward also These our greater drugs AKCEA-APOCIII-LRx patients study We’re AKCEA-APO(a)-LRx pipeline the novel mid-stage couple which wave ongoing

in start development with forward of Huntington’s making reducing issues safety LICA study have enhanced believe demonstrated progress and toxic first disease Phase XXXX. a consistently with diseases from reductions. production AKCEA-APOCIII-LRx to mutant safety drugs for reporting AKCEA-APOCIII-LRx in dose encouraged XXXX. populations. data clinical year, with We cause protein. is support We are Akcea platelet Huntington no should this broader X of of drug planned partner, by the of to we’re development Our early positive finds patient We data end larger profile including for IONIS-HTTRx Phase this to of study our the look plans the with IONIS-HTTRx. by of the profiles the by Roche, X/X in IONIS-HTTRx the target ranging

of an begin for end has $XX for the earn the the open year, IONIS-HTTRx, fee. which Before also label we’ll study. patients who in Roche to license a study X/X option Phase to extension we plan million license participated the

and the patients XX% response drug, cell neck conference, rate our currently AstraZeneca, and promising Assuming licensing neck payments we IONIS-STATX-X.XRx, regulatory and for B study will head from the group. Imfinzi. demonstrated partner, with the announced for drug, enrolling IONIS-STATX-X.XRx diffuse Roche data in is licensing, The and treatment After I/O cancer large combination which with could plus the million administered commercial ESMO sales. drug. Roche a more activities as both being by assume milestone royalties the treatment an for nearly $XXX head advances and At is cancer all when additional earn recently in of AstraZeneca’s responsibility lymphoma. in and We developed overall clinical ongoing

First has medicines technology delivering our of strategy achievements, XXXX multiproduct a X enabled important and which believe company, important serious pipeline have filled were illnesses. to business profitable with with position. patients financial to strong our quarters value-creating to transition our We contributed

over now turn So to like I’d the to Sarah. call

Sarah Boyce

will I We ATTR, devastating of brief. lives and that to believe of patients fatal a Because Lynne. we has the hereditary inotersen time inotersen, the Thank spent potential great discussing be a deal you, disease. transform with have progressive

and put, treated injection. interaction once-weekly disease a confirm with be of physicians to potential Our and which it that extensive Because goal in of diagnosed of inotersen the the choice is differences the and of the hereditary is of life clinical change Simply because optimistic of And we used, drugs be the it launch share which should to are challenging is direct study, [DN] inotersen, combines convenient Alnylam who virtually both the from in improved how quality identical and how primary XX% benefit a was NEURO-TTR benefit ATTR burden our likely believe the patients for Even a both the inotersen freedom to shorter the inotersen with comparisons with of and is patients many we potential study. market. subcutaneous but improved, the of both that will patients between the a are study. endpoint though is patisiran. it command the trials, in Known significant designs provides.

mNIS+X, are in don’t measure. patients approximately in improved the a by Alnylam in combined the our NEURO-TTR to benefits because compared stable XX% was max not of XX% study TTR reductions duration. their longer We reported study time. sustainable the or mean measure over the and that in mean is surprising reduction study at XX% this almost of in The stabilized NEURO-TTR Looking given

Let’s now addition the are extension identified the to safety patients homes. which from to we a look results few and open-label at we study. two-week most than their straightforward study no the safety every monitoring The in at minutes, more issues, should many, includes planning The for profile the ongoing NEURO-TTR manage. of take are inotersen,

pretreatment burden we after of not have issue come steroids, or use may Inotersen of steroids. inotersen, require the does do over effects have not term for four with cumulative with side safety seen years. the exposure long Importantly, that patients no chronic extra so the

convenient inotersen provide or from to and at greater anywhere As the a has for freedom disease. that single subcutaneous patient, control patients once-weekly the this home is injection, self-administered potential

entered the of Inotersen EU the is assessment U.S. accelerated now the advancing We in reduce are for ATTR. the review The inotersen great review recently time. hereditary which to in inotersen, and treatment to a the making regulatory market. progress EMA standard granted may

We in the are XXXX potential the advantage assessment filing have competitive of the also accelerated before an pleased EU. to filing for date last

inotersen program the initiated the the the U.S. by have we an addition, to expanded year. of In open end process in access

disease also nursing to support monitoring, to [indiscernible]. home self us our with process and of to high-touch will In led patient the our reimbursement Sense, Seventh which education, assistance. include announced injection collaboration further program are support, build well we which simplify We along efforts order in recently a training

forward even We move are simplify monitoring to every-other-week excited protocol. this to further product our

is transform potential to only and convenience. Our ATTR. for both combines the patients the fundamentally goal lives with of hereditary preferred with we have treatment that the We efficacy the drug option believe inotersen,

Beth. call for the now And to preparing underway launch, successful inotersen transforming journey beginning and disease. like this well is are our to over turn in I’d We just a in the of

Beth Hougen

Thank you, Sarah.

financial successes income. forma $XX marking and pro of strong the have We consecutive forma operating third performance and $XX continued fifth Our million. quarter, quarter forma of pro resulted first of the year million ended this net income nine of operating with in the in months net pro income

Importantly, we than range potentially of earned $XX revenue of in to in are forma or exceed commercial mid-$XX million track our first the financial guidance more the pro income nine we $XXX operating In cash. and SPINRAZA million on from XXXX, million months of royalties. meet

SPINRAZA pleased EU markets Biogen, in $XXX including potential SPINRAZA to earned we on We’re drops and in approval profit are in directly SPINRAZA revenue bottom as the of addition the that means have the third and all sales This SPINRAZA’s milestone for launch payment our margin generated SPINRAZA. with earned sales to of so we so tiered date will growth will and year far with we’ve portion not increase globally. million associated nearly are the Japan. The third-party we profit expenses royalties pricing in line. royalties for on sales Because to earn our $XX SPINRAZA-related nominal grow, the to The other date from we that no quarter in pay sales. and this we other sales those grow, SPINRAZA U.S., have our million royalties tiered, and of fixed, results continued

also payments. earned R&D Revenue from revenue over million. XX% our $XXX the is same The milestone component months this R&D and license increase of revenue year the We of last period million, was largest component nine this $XXX of year. a in first fees

months the the from included due to of is in to R&D of $XX increase we our amortization our collaboration. upfront compared from are payments substantial the in of $XX Novartis million The nine addition, first revenue million XXXX. revenue revenue In primarily XXXX amortizing

forma nine for to XXXX the owe period $XXX inotersen, prepare $XXX were next SPINRAZA same to increased pro we first licensing months This under million and continues as Akcea operating our year. for due volanesorsen Our increase expenses agreements. compared plus last of to to commercialization expenses year, launch launch we prepared million in to the was modest fees

the During potentially continued increase track on remain increase in Akcea’s expenses related Even activities. we the of our projecting are quarter a in SG&A meet fourth we operating to exceed guidance. the quarter, financial fourth with our for our XXXX, and expenses to commercialization or

quarter house of activities. that purchased building R&D we our XXXX, During our a and the manufacturing third

In and Akcea’s $X in paid partner nine over we we third from We our to than we when price rather owning rules less XXXX, months financing the in leasing property. in more building we loss the quarter us end purchased the leases these have plus of represents the recognize recorded cash. purchase the billion were buildings substantially R&D received prices, the proceeds by building million value we difference to constructed facility, than the than between The loss. This the facilities. the when payments July. accounting we $XXX expect noncash with in contain required IPO savings first of which the XXXX we to When market realize significant liability the predetermined these for initially purchase bought

minutes talk our Now in a Akcea, about this and I’d financial like few to statements changes take quarter. our affiliate, the to see you’d

equity Akcea. the by IPO in represent us. With is by not complete, The XX% controlled an third own held that Akcea’s we of about parties interest shares

voting was different and accounting Akcea’s This we lines interest, public when However, appropriate with financial as of control Akcea’s Akcea’s consolidate continue require P&L. no book now to our private through to ours. continue expenses and than us Akcea of is to our wholly-owned. maintain rules overall revenue was it than on statements We’ll Akcea the

called net of line income we non-controlling interest income that To XXX% our have we own do Now attributable P&L that our just net Therapeutics. loss in on above less or that to Akcea’s we only this, that portion than adjusted income new or a net own. so net have we our reflects loss loss of Akcea loss P&L stock, Akcea’s or

transition own, net quarter the the million. taking by For Noncontrolling Interests a We non-controlling attributable line equity our important elements $X.X was success We have in revenue will section. coupled inotersen our in to I’ll of year liabilities financial serious the which third about net partners the end million has consolidated sheet IPO back interest called portion it and assets XXXX, continued the to to to Akcea’s growing balance on believe key place enabled we calculated line is from a and do in And P&L, $XX.X achieve of to loss the our company, to next today commercial the Akcea’s multiplying the call on third illnesses. The delivering balance SPINRAZA, our by quarter of medicines the stream loss book to profitable with volanesorsen of revenue this growth. the from long-term sustained, Stan. of new we in Akcea XX%. from Akcea turn patients continue the Akcea date with we multiproduct amount our of potential includes and now not appropriate and Therapeutics our Similar and to sheet,

Dr. Stan Crooke

position strongest exceed to or guidance. our entered financial ever, financial expect we Beth. three of our XXXX potentially meet months the Thanks, and We in last

product believe strategy commercial our this to value. our Ionis long-term continue we -- unlock and to business its during are assure demonstrate the by demonstrated value. achievements evidence call, Ionis innovation, key the business the success that objectives As continuing shareholder have maximize The for strategies reviewed we of

volanesorsen from XXXX. recognizing plan we We commercial inotersen add royalty, in SPINRAZA and to and from significant revenue commercial are revenue

As commercial the the the Akcea value our of so, keep strength tangible is we value Akcea’s we’re the assets control company of and retain assuring our portion highest unlocked when of commercial evidence activities. matures, one larger the considers and doing valuation. In and both a of of our greater Success late development drugs. performance of strategy

me Let take moment one our that in to a opportunities on next three the to focus is midterm, years. the

advancing increase nearly seen Major which We continue more less milligrams dose. of that to course, amount mid-stage of week late-stage than pipeline cancer enter and use our reductions. means consistently In for dose other duration as at of treat less and of forward we of true comes be target for would long-term -- value a first dosed of as in another level can or to that believe want signs increase developmental liver. this treatment, demonstrated if less, consistently doses safety profiles whatever clinical several these if progressing all could in step representing X X of to advance targeting development, no drugs, We’ve to to by can have frequently anticipate of every currently no disorders. the injection-related drugs mid-stage We to a truly many as performance. X both of platelet drugs, we a We’ve the our of are from or our less EDXX we technology. in new X.X drugs to give extend of it is drugs on drugs new X a Generation the dose of simply well our milligrams continue simply which added creation be a consistently products. with we based LICA an than LICA a They the the drug including clotting have XX our from And them midterm we the our safety plan and and disorders we year, issues LICA X less to will The important game-changing GalNAc-conjugated our Or be as XX%, innovative week. drugs low reduction, we development, reactions like. animal we the with can the drugs we positive drugs neurological market. essentially into LICA that franchise want drugs to reduce and including development frequently. advancement drugs, week EDXX value milligram expect monthly Akcea’s value advance three have development and will of is data, lipid

and We make to those advances -- in be if our substantial set drugs pipeline of technology up Q&A. group profiles And Andrea, that, even better the have the continue will next questions today. to over could into with going us will I’ll call than the to that answers. for ensure turn you

Operator

[Operator Instructions] & first Eric Our from question comes Schmidt Cowen of Company.

Eric Schmidt

question’s discussions. the to Congrats I to Lynne luck partnership inotersen Brett on of new my the starting role. a around new best guess chapter and on

can’t Stan given there. talks along for value a Ionis’ partner pie? Like, address in bring the you shareholder can what expand partner like that how here partner the And are of process. how do maybe you on Sounds guys that focus is does specifically just or in maximizing the the right really are well kind strategy a and could Maybe late development regulatory Sarah conviction yourselves?

Dr. Stan Crooke

Thanks, Eric.

We significant that to those a months inotersen patisiran. market. And with physicians is with a take probably last and confident are away portion look hours patients, along. We’ve in take to of well quite tried out the things importantly, conversations invested expect walk what not one And We the to marketplace. very competition over we’ve as that conversations investigators done have many we’ve that sober we inotersen investigators do who but the care is are very of at knowing that, of from we patients. of few of going the these take

a market. is We that also significant the believe market

expectation with significantly that, -- of we So with for changed armed have we are from our pleased and level partnering have. we not the interest

great have commercialize. to in progress made We getting of ready deal

real. of the the to optimize partnership our I to very opportunity potential, so most And take inotersen’s is advantage then think,

can you outcomes. you -- we ourselves. think market it I to so choose number imagine entirely of And any could One,

partnerships. We’re stronger diverse world and strength partnership of take part will to a or in and of two, in do different kind care day; identifying we the help think added and patients getting an ready number provides physician adds some three, that marketing value every even the or that the of communities find them; that of in we

We’re looking forms. at all of those

decision We and we ready are progress in of measuring information the in to need possession our best launch, think to make the we to getting assure we’re success.

Operator

Please of ahead. Securities. Our Wells Jim Fargo next question from go Birchenough comes

Jim Birchenough

narrow exist the for Let the me and remind broad for it? my on and finally Sarah you us maybe target got of on whether go new would identifying way focus? hematology. really, at the Stan with with cardiology, And add commercial I’ve then categories expand describe an how then Lynne, inotersen for on Lynne and and Is physicians that perhaps. on that Brett. collaboration And collaboration? Biogen. might where you What And to opportunity you’re kind there of given Maybe just timelines Could there’s collaboration? Brad about some your congratulations to the neurology be to roles. three,

develop of approved reduction, to think going types the more TTR you’re Is TTR like get reduction? surrogate an rapidly? could validation full of that the need do LICA opportunity NEURO-TTR Or do the to on Given Just to TTR. a you that study? on there

Sarah Boyce

we database three first There’s question national core well of we’re like both part of say, as into and fall And also process a cardiology, physicians. the Maybe neurology. doing actually cardiology, advisory as you I’d the certain as clues U.S. your start within regional From can that identifying about hematology claims do. boards the with

seeing cardiologists And some to at one look With more neuropathy the the often, AR neurology, places hematologists. referred the often, who amyloidosis it’s maybe hematology, multiple diagnostic the gives sort the patients. logical starting of who to are of to with us looking peripheral is default, the these regards people by myeloma specialists as to Specifically treating indications that also at and quite tests also with experts ATTR are patients point.

and lists with level looks what So of specialties. audience regards the that mechanisms like. of far identify each those to building And of three kind it’s customer target pretty to separate we’re along these

Dr. Stan Crooke

Ionis partners, of that that With thrilled great regard the drugs a other larger. as The and the privilege and are in And associated wonderful their to that collaboration could number pipeline from gets we, of has perhaps Biogen, with of I collaboration success as they today. makes the to have, with expand. and performance the any of in be is diseases range both development Biogen component the it’s important think course, to Biogen that. participation SPINRAZA, the continues

disease Something well. haven’t our that have account, drugs that discussing much expand time own to for of a that quite number we continue and spent is as will neurological we really also

are positive and our We about highly relationship are Biogen positive. they with equally

new improved clearly, our to with and be this of versions want expectation SPINRAZA. successful advance and we continue will collaboration SMA is potentially franchise continue and certainly, So to And our expand.

Brett Monia

regarding Jim, TTR And the LICA.

clinical expect advance that off, we great-looking testing on amyloidosis. And have patient data. have was this, respect preclinical wild-type speed to rapidly drug the priority opportunities do study can develop we And we’ve moving next more it, high we drug to population, that for what that at much in First absolutely, a includes as there we’re to mentioned, learned that of do be are we forms many forward drug the finish treating replication expect with and which With this based not of to rapidly the year. line from for too. do NEURO-TTR. to this We’ve to all much we forward that believe so TTR identified

of obviously radar. of are feedback. need able strategy. we that to But and What type those considered of regulatory on our when those biomarkers, we’ve is type table, discussions, everything certainly, Regarding might do, on that the involve be course, will we

Dr. Stan Crooke

add to what to said. just Brett And

I our very think of the drugs highly consistent. is important that is Something GalNAc performance been has

We trials. are haven’t seen potencies variations per And no of drug potency consequence week in of the us X meaningful had these of to X them to in milligram drug, range and all we’ve in IDXX. absolutely events adverse any giving from

we are high all trials, progress, development to drugs. ever that when greater perform these we now patients of longer extremely an have as in of and longer so and GalNAc number drugs the them we well of hoped give have began being have we and now that the and XX We most really these drugs are we an enhancements going confidence extremely profile us and treated in level with clinical And effort.

Operator

Wiley Our next of ahead. question Stifel. comes from Stephen go Please

Stephen Willey

both well new as their congrats to My on roles. Brett Lynne and

Huntington’s of couple actually. on questions a Just

CSF, of the So just to wanted of to the if I’m this point wild-type be Ionis-led also just we’ve of top of of the kind an line around the quantification an is the will clarification confirm in be, that this communication, end also there’ll then reduction, measured period? of follow-up reduction this is specifically of at outcomes, quantify measured will wondering a that going huntingtin protein also end measurement clinical opportunity huntingtin to TFC, at CSF? the be dosing? talked data the announcement Or and about be communicate the be and protein And something will

Dr. Stan Crooke

about Ionis the communicating about the be date. future. enthusiastic will program we are first, we’ve And the near made and in Huntington’s both to that So Roche observations

is safety. Our focus primary

the and provide we course. summary longer-lasting them secondary scientific are Huntington’s, data and and of focus in upcoming challenge an higher, is in much more symptoms reduction, is is will of HTT that that more SMA detailed from different much all at Our probably quite short measuring of of then meeting. significantly term the course, a

we a add to benefit? surprise and I any So If we nature that? the preliminary to on is to Lynne, the advance going in because potential reducing that the potential would any indications that, agent of have and have anything of Huntington’s an be disease. focus an of soft would we of be do has bit of me everyone treatment -- But we do important have to that think are do even the you of safety we a get Huntington’s. want certainly, we

Stephen Willey

I understand.

you So mutant able to huntingtin in differentiate wild-type both protein? be and will reductions between

Dr. Stan Crooke

although question. I Sure, think it’s don’t terribly important a Yes.

Stephen Willey

Is maybe Gen the as on follow-up same for there’s X.X? different And inotersen? if ASO Okay. just versus features payload beyond Is Brett nucleotide X.X not, LICA? different quick the on TTR regarding that curious And maybe candidate. there preclinical LICA any Just length? one if just differentiating Gen -- it it is

Brett Monia

molecule, inotersen similar. the not but X.X very chemistry, exact and Gen it’s it’s

Dr. Stan Crooke

what And sequence way. companies report the ASOs. versus questions asking be may relate the that siRNAs, to how clearly seen X-prime -- various modifications other in chemicals how least effects of chemistry of you and into depending drug deal define methoxys, we many seems the side X-prime in of -- there. we a importantly, from is a putting that same. to clinical are variation of siRNAs They seem what and sorts in great to in the that the whether many those at trials. are different on molecules, us a short-term of With chemistries are lot X-Os, basic any but The vary significantly course, there very there’s sort Well, it to chemistries lot is most factors, not drug,

Operator

Our Stanley. David next from comes Lebowitz of question Morgan

David Lebowitz

curious what what’s identifying for preparations status of going efforts the that? I’m And the the are in patients how launch -- are the volanesorsen? for

Lynne Parshall

already quite medical and all for as structures, obviously using to They’ve involved and the a along They with And well in facilities. in as made do doing a as database identified. they is community being these our patients and surprising variety And of that, these commercial patients? long teams which they’re time, identify a key Europe work U.S. disease that are some number number very patients finding, physicians this, quite major strategies, they and well ways the criteria using medical diagnostic in have in been already. So direct treating and I the both searches you the a say, a of actually, isn’t substantial of a of of patients their building find of They a were you European work with substantial number been, have might a have is I in go Akcea that there very markets have of physician, like go with may physician one. course, have if they what a more. ask two. database then database the search

patient So all identification in which U.S. terms of variety different and are as in on well S. very being track I initiatives underway, the database are they with their said, have, a as of as right ex-U. productive

Dr. Stan Crooke

that much key haven’t think that self FCS the I to is, a To steps first, a and aggressive enough great they’ve emphasized -- done my they’ve have that’s important symptoms. identification we more taken I tremendously mind, FCS much more community. probably think galvanize quite in deal that some today, community of we are engaged active are And signs

important the regulators long. and better understood was with severe all us of us. active It of And disease desperately out which history engaged or the regulators. is day-to-day treatment. natural demonstrated fourth, an associated this disease educational what’s very and study more more much is today very to far benchmark, the And we in how needs see aggressive survey before Gaudet, that community. that symptoms is and then really the much the Dr. should we Second, are from a patient by instructive than published the here it telling sense more much And think and we’ve what I be really was know how third, program of a done what of

recognize, rare awareness. any increase very team disease we with those a key contribute to think, I disease, of awareness, all and Paul and X choice, build good doing there get are of the the only only to to Now And volanesorsen, identified. think it, is when is job those things, drug X and the in patients one as substantial that’s build and market, elements actually of a I to particularly called that

surgeons the patients for with other done, identify they’ve who physicians are or key a and as as of physicians the neurologists has, these think or of sort people. Sarah variety just I catchment

the in really about progress in success sense that my to addition So educating in there’s and commercial is organization, of patients, disease, been physicians in tell defining to all the the going progress think that is the tangible made the building it they volanesorsen. the I

patients extensively to very these who pleased extremely for took we when understand has It done of Akcea of not with severity circumstance did disease. also the how the recognition the even physicians began We’re with that pleased the patients bad was that the us care work growth of and was. these rather in remarkable

we were we ago. compared far along a think So few where we’re to months

Operator

Fye comes Jessica next Morgan. question JP of from Our

Jessica Fye

think, on inotersen make And just to I’m you’re studies the reimbursements of to cardiomyopathy? cardiomyopathy I sure I I understood comments How potentially earlier thinking And extend the future referencing, you the the clinical label curious patients. get about also potential label? then to data should patients? NEURO-TTR for made from think path potential want how you the wild-type how cardiac about we about forward cardiomyopathy one think faster into for

Lynne Parshall

data cardiac as in course -- we talk it’s two involvement obviously goal a saw call of have. study, to of have continuum. our are disease. who being patients And them who the over terms of disease the your patients, symptoms. their cardiomyopathy cardiac of had polyneuropathy label. cardiomyopathy, the as fact, in many this when with you first they And we say with physicians we we have they And in about half patients, really polyneuropathy questions, will them with to get that our polyneuropathy interesting the cardiomyopathy was And of all of into that Jess, terms and data pleased In reimbursements, because but think

be let the And is to involvement. even not if so of difficult be polyneuropathy patients course, forward development -- the to LICA. were because will these reimbursement have going said, to to cardiomyopathy Brett path that And the be some most have the but on going of about it’s I I polyneuropathy, think dependent label talk patients to as limited the I’ll label,

Brett Monia

that population Just the be civilian to patient work the disaster earlier, I biomarker would studied. clarify, referred believe, to limited I we to

controlled likely biomarker I studied population a to a have think not study the use to able less in randomized patient it placements date. wild in would that we be we’ll be for

form. in hereditary options in to several which really referring Of look course, population we was that patient What was patient this a population. I we’ll was studied,

Dr. Stan Crooke

is, patients demonstrates, really work plenty that there’s we’ll advantages. straightforward think term, value cardiomyopathy, I while And think, great the comment final to in this access I outcome demonstrates, I think do be long fairly with -- guess, I

to in long-term the outcome pursuit of LICA So with we particular. be in continue studies interested

Jessica Fye

have the the Does that that comment on just to well. polyneuropathy? up patients? as about follow Do FAC wild-type cardiomyopathy And patients they apply also having polyneuropathy

Dr. Stan Crooke

so are, know, typically circumstance. They different involvement. older a they somewhat much, you Not typically nearly have and primarily cardiac It’s as no.

Operator

ahead. question comes Partners. Paul Matteis go Leerink from next Our Please of

Paul Matteis

you -- Huntington’s. on great secondarily, can and I first on couple kind be HTT outcome or just of a question would your what good is CSF to just -- a would opinion knockdown say, be My not a represent, magnitude okay of the curious just then have the in either wondering outcome, frontal the brain, CSF, a the the I’m to so where be look you brain to is it understand lobe? the outcome? And knockdown back or actual great HTT it of when important you at is in how changes occurring cortex or in the

Dr. Stan Crooke

reduction. what is hope to just see dose-dependent Well, we

just we have heterozygotes dose-dependent I better always can safety know everything because and normal. the of hope for. that if we reduction get we I have we get guess, answer the question and so we think great is to the that we we level are dose need reduction,

if and We than I but reduction, XX% I the to important And certainly getting greater that. think other areas I And hope the where you’re cortex itself. to correctly, least on check so get in motor reductions quite manifests that areas to of disease in beneficial. I XX% Frank know that, the correctly, with were have if of at from remember anywhere double and to animal studies, remember

the we’ve And concentration understand almost and and central in Roche diseases. of significant really led that cord that activity, but to brain seen range treat access Alzheimer’s supported us have of course, colleagues an a those and the Parkinson’s brain all with injection something so, moving Huntington’s we at of diseases regions broader to areas drugs most drug and our and of range activity the great spinal after quite need the accumulating. intrathecal with like we’ve just diseases. wide to not data the And board spinal the than of think into that Biogen it’s see invested that’s are encouraged in other we We our almost we’re of pipeline neurological where column. And data effort, the both because all all a drug and that substantial the system into of across much we by deal SMA nervous

We’re as as Roche. and Biogen excited about it is very is

Paul Matteis

you to Okay. you importance, were interesting in rank clinical to most for And a then or if in what’s endpoints either trial? of future terms what, pivotal study this

Dr. Stan Crooke

or disease David, enough I about I to Paul, know I because forgive feel -- defer sorry, that me. like would Huntington’s don’t

more pretty wait and with I’m to of You experts me signs can and the on like. we rather about real to until I’d learning George have symptoms clinical that, just some and call Huntington’s, if that’s soon. happen the deal call going you

Operator

question go ahead. Gena Barclays. next Please from of Our Wang comes

Gena Wang

Huntington So program. I then have a one two on on one and inotersen quick

So pretty of you’re talking peers regarding already to I’m reimbursement. on a so pricing, sure lot

So data good given very like about the be would what and also pricing? we can impressive the inotersen us data, on a the benchmark all like need, think a saw medical that you guided to

Dr. Stan Crooke

my that preference answer to is not question Well, today.

that and all the in competitor. signaled have not Alnylam I are prepared with work it today. Soliris-type noticed we’re that noted have understood if but doing we that We prices, I process interest to has do a of correctly, sort of and

Gena Wang

identification. the patient question another about is then And Okay.

you addressable And many inotersen? So have patient will you what how identified? remind then the can would just be it number patient be, population of us you think for patients already

Sarah Boyce

Yes.

patients the that’s In available so epidemiology the XX,XXX hereditary. both cardiomyopathy at and the form I’m the about if about today, talking XX,XXX polyneuropathy look about consensus, So with form. disease cases, of you with the

we when be overlap data patients, patient the level data, cardiomyopathy that think different two very the it’s what, of because have there’s together the a of so form keys same believe, disease put and that we this much when the perspective thought not. was we to I population diseases fact, one high started And polyneuropathy. disease from available, this it’s is to polyneuropathy larger that was than the just Now Lynne when of in traditional the the guess they alluded to a and event

competitive patient journey to to going so very program, along mapping. this mapping far give a we say, and obviously, terms in have well physician suffice numbers identified, details not our patient In is our KOL and that. that are around we’re as numbers of as But we patient

pains our pretty as we process program. on accelerate CRM the those have, also diagnostic along team the as a intervention move well MSL how in really which deploying system, journey through idea we the and as that people we’re And where will good So as well, points of are the building believe, to system.

Dr. Stan Crooke

identification lot of think that patients that be will be patients. enhance of benefit a the to all I I because for there there’s there’s participants a which be of will had thing market, and in Pfizer and Alnylam Ionis. substantially will think great for And the

Gena Wang

then in just you of Could terms target one question huntingtin. very Leus quick huntingtin Do a remind protein? wild in type on as just And target specifically mechanisms us Great. mutant also targets? the you well? Or

Dr. Stan Crooke

No, we target both.

little concluded we and the all mutant. a ability that we then entire look and disease, other huntingtin accumulates to mutant, and We have at it’s like of the amyloidosis the and the of as disease and of target stuff. so But course, bit inclusions cell that the produces on characteristics kinds in

of there’s the We or Huntington’s. focus simply to we concerned the the about interesting meaningfully reducing the had all of Do reason of was -- one it on We mutant safety we So total? be believe choices. looked. no

reduce the to of all potential process. disease choice so And the continuing causes was our

time We we’ll then have to for have and more one question go.

Operator

of next is Laidlaw Yale & Company. Our from question Jen

Yale Jen

And here. to my two Brett. all, congrats of and First Lynne just questions

I by drug you partners year, I same At think, given in you, market. one, partnering. the are active The that the And the know first again, guys for -- inotersen in time, approved. probably mid-next preparing get can timeline the also

So it it how prepare hand you that you to is that, you guys by only -- going thoughts case? are you’re just force partner, out for right now the the there’s to to mentality before yourself? a full or market -- as it guys just have how’s And

Dr. Stan Crooke

our contribute calculus thing and drug? what launch I should ready to launch? resources company make to field have they the our market is force, our aid at, launching penetration our what we what looking maximize immediately is is the infrastructure, terms of do I-Eh, principally And that competitive bring is important immediately markets experience to in in the can of to we’re the that maximize focus successful. And us one we think Do the most to here the so they choose edge? might in to on this bear the

of is the at look going us We prepared that invest in And drug we taking a be choices. to investment marketplace? but a in to higher. number importantly, being have to as the Obviously, also what well more getting dollars the in going their they’re way threshold And the we’re the meantime, forward for ready obviously, along on our ourselves. to that becomes in launch partner in, progresses

Yale Jen

will on XXXX, sense highlight end last That’s that there’s people question by of guys you a sort drugs maybe how element better so LICA more advanced -- very your you is allowing a my that? going important recognize that, by forward, many on -- helpful. probably And be here just of for the have say, maybe clinical let’s could very pipeline that LICA

Dr. Stan Crooke

have or just would I I development and than X numbers -- studies should to substantially in Thank then X I will the maybe more end more. X think, don’t Phase head XX of and maybe We more. specific we in completed LICAs the on by you. but or clinical XXXX my offhand, have, GalNAc XX have guess by X

I about think that’s you say, Brett? right. Would

Brett Monia

so. say I’d Yes,

Lynne Parshall

two Xb of be APO-A multiple Phase then ranging are and I’m studies dose large very sure and the that and APOCIII-LRx And hundreds patients. important will studies

Dr. Stan Crooke

in and try along, study like to APO-A other bring pipeline progresses we’ll actually our to our next to year. longer for at overall in and C like, trying thank data that, well to some And then have there Q&A share and point be our for the term is GalNAc experience With like it the performing both end. interest. substantial summarize an as I’d everyone we’ll that we already you their experience if, impeccably. we’ll and every And is a

revenue the commercial about volanesorsen, next commercial be have we we revenue will and in to year inotersen. increasing a excited being tremendously revenue from We’re which from from SPINRAZA, year position look which commercial have forward

it’s going delivering competitor Our Akcea to successful very progress delivering understand in -- in value, the to successful Inotersen, great a helping to being partnering strategy marketplace. be and continue people

Operator

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

disconnect. now may You

Ionis Pharmaceuticals (IONS) 11 Nov 172017 Q3 Earnings call transcript