IONS - Earnings call transcript Q2 2019 | Ionis Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to Ionis Pharmaceuticals Second Quarter 2019 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 of Investor Relations, to lead off the call. Please begin.

Wade Walke

Thank you, Nicole.

a encourage Web go press that site related tables, I section the release we begin, GAAP financial Ionis the of to including will we discuss everyone financial to to Investor and to today. Before non-GAAP of measures reconciliation find the of business. believe We economics financial how and better results business non-GAAP we the represent our manage our our posted site that today. slides discussion on Web also We've our accompany

Beth call the Damien and are Stan Chief of Monia, Chairman Brett Board Officer; Officer, McDevitt, who and on Crow, Chief will for the Chief Executive join me Hougen, Business Officer; Officer; Financial us With Operating Q&A. Chief

I statement. consult SEC current beliefs. results These would encourage three, language We'll slide be certain to statements are making you I detail. expectations to and subject which factors filings your in and for actual like risk on our to to uncertainties, forward-looking and attention based may forward-looking which our contains risks materially. discussed our statements, the additional differ draw are

With over that, call Stan. the turn I'll

Stanley Crooke

continuing our track entire aggressively further as commercializing, and second-half This us these sense income, more technology. you achieved this of of on our medicines, deliver an business, the our year. for In than results finished million. is call. we income. joining and year of first-half Wade, and XX% as our good consecutive position as the broadly on the increase and fourth Thank XXXX, to today's financial to across third business, net a successes consecutive strong revenues of $XXX invest financial first-half makes compared of substantial the morning value-driving we achieved putting of number year, with advancing enter including us of across Thanks, strengthening while our everyone. pipeline We last of our year it year operating to to We

patients of year. worldwide and SMA SMA care approved therapy all of SMA remains for continued types. and blockbuster gratified adults seven of SPINRAZA treated SPINRAZA and particularly faced about the ages we for began these first-half to standard only I'm now performance are into now progressive all the treatment before adolescents think treatment some young the Having this SPINRAZA's a the of now who with SPINRAZA, patients would adults. patients weakness young approximately increasing as of who, life and have dependence. years, have

they vast becoming are maturing productivity. are Babies more Today, the a can independence majority to And forward the healthy to beginning. productive SMA able that's children, life symptomatic. of those and with And of babies independent look before and and treated being longer, just live normal are more lives. now like

two That commercial sales last launch. quarter. second in first AKCEA, the has of $XX the Our XX% now compared in affiliate, quarters reported product and a is million the launch, of quarter full increase TEGSEDI completed to

is in lives. the are it TEGSEDI on that physicians early about from positive feedback having is benefit still from receiving TTR While and positive patients' patients the launch, we

[technical liver, plasma abnormalities, extreme U.S. body distribution -- another metabolic by and a triglyceride disorder with in clarify of in to FDA and and rare disorder through the through FCS. Therapeutics path to the the as launching later very PTC in patients high patients difficulty] WAYLIVRA year. patients study with productive resistance, the with quarter U.S., We're FPL, as medicine and well in insulin recent this only treatment severe triglyceride market this the to WAYLIVRA is triglycerides American looking by abnormal the discussions of into WAYLIVRA for Latin are AKCEA, in approved the including fat. and In Europe a characterized expanding encouraged our we forward AKCEA

an medical achieved We future meeting endpoint and presenting at publication. significantly reducing the achieved good detailed liver and broad study. the statistically with observed with WAYLIVRA forward tolerability the reduction primary the endpoint its safety In WAYLIVRA study triglyceride from also in dramatically secondary a this look a and to in important fat study, levels. of plasma along data

briefly from our let's catalysts pipeline. more recently Now, at value-driving look

Our and late-stage programs as pipeline III near-Phase of III Phase is advancing planned.

Phase Our medicines III SODX test ALS are for Disease and Phase Huntington's the rapid of studies, patients. potential both with in III for

to LP(a)-driven to treat X advancing this And is patients studies of LICA amyloidosis late-stage all two on forms begin with other Our and disease medicine, mid-stage to year. are our cardiovascular well. one, patients pipeline on treat as with track TTR the Phase schedule

results. that HPV achieved our program Our reported partner positive GSK

our patients X Phase completion completing form renal or advance disease these stage the we Phase that of of this stage believe LICA into in later as We set will of X And with rapidly of to one development. the as end-stage the study IONIS-Factor-XIRx medicines of both study are well Factor-XIRx.

after we seeing what forward of with studies pleased we in these to they are analyzed. completed fully are are results We and these studies. presenting the And look

initiated first X recently We initiated Phase Phase Additionally, additional mediated which plan complement of X we diseases an the studies mediated well. related as complement the for IONIS-Factor-B-XLRx of indications or two study people programs. treatment disease

-- a of initiated indications involving IONIS-PPK-LRx the And with of regard PKK for the also angioedema. hereditary X treatment to forward plan patients move with Phase abnormalities to in study We of pathway. additional additional

to pace. to in discovery continues leader technology am I and an that at And pleased advance you the ever drug RNA-targeted tell We development. are increasing

is rapidly. this are Our profitability. while sustainable are advancing full achieve transformative And doing that pipeline medicines all potentially we to continuing of

turn update our call Beth to who progress. And over now, I provide Followed on our update. will to pipeline will financial Brett the you by

Elizabeth Hougen

Stan. you, Thank

year We period A Our the trajectory. total more of revenues. this and both first the to revenues R&D financial growth ended multi-year more half $XXX by our half growth commercial of build results increase XX% driven million. with last on over the year, in continues than for first same

a half the this growing for revenues significant and continued of income profitability both with $XXX first Our of on million in resulted $XXX operating basis. income net year, million of non-GAAP

$X.X worldwide net by with sales. investing across XXXX. quarter position over while half SPINRAZA business. XX% strong broadly our revenue. in billion of We generated billion to the track SPINRAZA to the results ending A our In These nearly XXXX, $X XXXX increase significantly exceed on substantial compared well put first even also maintained cash

Reflecting patients now, compared our XXXX to XX% by more million. than with Notably rate. royalty increased quarter, last also The SPINRAZA's are treated being patients revenue in XX% strong performance SPINRAZA. by the quarter. SPINRAZA second increased $XXX royalty highest reached worldwide number And we approximately of approximately on to treatment the

Adult XX% U.S., continued up the second the the case, on also last SPINRAZA significant In quarter. of quarter. number patients, patients the growth be of last the increased. Outside make to the on to number driver SPINRAZA by patient increased segment, compared U.S., quarter. which compared this approximately to of patients In largest

This patients quarter. new continued and reimbursement America. SPINRAZA driven mature in markets recently growth Asia for including in launched larger more the markets Biogen to was the rapid in along Latin by with around uptake increase second in world

place is XX reimbursement and addition second the Ireland, now Formal with U.K., Brazil, of over countries Argentina in the in the quarter. in

outstanding by XX,XXX opportunity a initial treatment SPINRAZA's than Biogen the larger with clinical estimates We in globe. direct presence. long-term than around for more development believe acceptance global payers that Biogen SPINRAZA's program. And that has now performance the estimate with broad account is for patients it's SPINRAZA markets thorough much continued where

believe treatment foundation care expected will With SPINRAZA remain a the for growth patients with to We global of new continue. of patient SMA.

medicine, in disease We you the quarter, with than more rare commercial increase AKCEA focus expect disease a first education sales in quarter. patient second from as $XX identification. over in to on are million would and and TEGSEDI the XX% And continues

EU centers, community additional Outside U.S., EU TEGSEDI from on forward hATTR in well next potential investments where in close to TEGSEDI country look launching in Therapeutics. the approvals launch of Germany this to can beginning as benefit PTC with their the the countries in as launches TEGSEDI WAYLIVRA for planned treatments through particular With quarter Brazil year. their AKCEA launch patients is home of and schedule. additional we

are first-half for actively to year revenue revenue of compared provide WAYLIVRA's from America. significant is growth to million us. preparing XXXX demonstrates of This and doubled this Latin million. R&D PTC sustainable is in that recognition a nearly revenue than in PTC. In approval of $XXX we the in more to source $X WAYLIVRA patients to EU R&D

from as of Roche R&D primarily programs was milestone from in our we for by payments $XX components for earned partners included million payments advanced, IONIS-HTTRx. million significant milestone amortization the following advancing this year $XX more that medicines than from million of $XX the advancing Biogen we million driven revenue in from $XX and upfront payments are first-half our we of in received neurology amortization

includes which this Regeneron. in And from and component $XXX grow licensed million licensing in $XX to fees, has our quarter. as the we paid nearly million, us expect license third advanced $XXX included $XXX And million from product for they commercial when Alnylam based to Novartis AKCEA-APO(a)-LRx and in which first medicines Alnylam Today, technology the sales. technology amount our million royalties

Our this to of the an non-GAAP first-half compared operating year $XXX year. were in last expenses million increase

our broadly launch in commercializing business and in investing the including across WAYLIVRA are globally investing to We TEGSEDI EU. preparing

of of $XX first-half recognize income also We tax year. million this the expense during

generate because U.S. expense income incurring will state that this we year. We income we federal in are and project tax taxable

quarter. earned the second-half commercial fee and do During $XXX another from large of we significant revenue. earning year, this achieve not the in license like the we item, Novartis R&D If we first anticipate revenue revenue million

our in second-half the generally to in the year. the projecting revenues be first-half of are line this of We year with

WAYLIVRA expenses of this investments in a launching to anticipate advance our and the commercializing pipeline. We higher operating our TEGSEDI, continuing to also continued second-half due in year

improve track on or that consecutive our program all also initiate which third We're consecutive of of our track meet to non-GAAP achieve we fourth net of will the year. operating particular, increase year, we to on our of on we a income AKCEA-TTR-LRx, for guidance on development the to III income, With end this expenses. year our both basis. In and this before are upon are year financial expect track Phase for

efficiency the strong testament of with Our and novel platform coupled business are to our our model. technology results a productivity

we Looking strength that to to value while maintaining we sustained drive our into patients the in deliver financial continue the remainder increasing can and of innovation this future, focus confidence year to need. on have to

Brett on our pipeline. And call the an to update provide over with that to I'll turn

Brett Monia

first-half pipeline XX additional development. of remainder Today, look this of the medicines recently Beth. and we in advancing a And forward medicines XXXX, significant over next achieve three to through In catalysts. the we successes. into through have programs pipeline achieving year numerous driving year, Thanks. value we of approved

to worldwide the data SPINRAZA standard and treatment from better term the of estimate types is longer even SPINRAZA of ages continue estimated all all treatment patients performance. of in demonstrate and care emerging

up for We continue from and for demonstrated treated healthy the up new kids. This new continued to symptom treated onset data nurture supported months to normal, to showing see like in XX data five developed before long-term by patients in over the infants years. studying ongoing is treated improvement again patients XXX

is recently or to once twice a improve early upon forward and we we improve with expanded What reducing year development follow-on although invite potentially in -- next -- medicine to difficult frequency labels SPINRAZA it's to safety Europe. difficult those into medicine for pursuing SPINRAZA's profile, advancing and potential late look year, in efficacy or year. per follow-on the this

month, of patients data As open-label the Earlier substantial the demonstrating TEGSEDI when in issues launch new study extension continue advocacy no with identified. supporting this to NEURO-TTR with TEGSEDI, polyneuropathy the the from and TEGSEDI patients present we safety are part hATTR hATTR experiencing benefit ongoing AKCEA new treatment with with long-term durable data of treatment. present

encouraged to productive AKCEA to debilitating in medicine FCS path continue effective this the recent need patients recognize to disease. a forward our U.S. rare WAYLIVRA the for clarify unmet of interactions FCS. on for by WAYLIVRA and medical and with we're FDA the safe Moving when And in for a

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our potential for also now disease study stage The late pipeline as of in patients to neuro the generation partner with significant advance driving IONIS-HTTRx, near-term. with our Phase known III well Phase Phase Huntington's Pivoting catalysts in III with programs of the is continue III our RG-XXXX pipeline, and progressing Roche. HD to value

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of to of millions to the are Our Phase potential worldwide a the before targeting the treatment cardiovascular most medicine potential studies from late is our the end a driven of representative the has And healthy of on medicines people AKCEA-APO(a)-LRx late-stage the transform LICA track cardiovascular like and year. disease TTR disease. III little advanced amyloidosis suffering start of LP(a)-driven

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made well. person's significantly people Hepatitis B is XXX increase disease worldwide continues liver program can we're the liver a with which pipeline, very to stage liver and late failure B and our mid million progress to cancer. perform great chronic addition fatal stage progress In in potentially making of pipeline also early our risk over We're our pleased being a affecting the in development Hepatitis cirrhosis

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renal particularly data end also enthusiastic Phase We its licensing study are in we're and our we're currently Ionis currently value. studies. Phase completion XI decision Factor very seeing with look GSK's in disease and nearing for potential stage a this forward in XI both this about Factor in the X given later regarding X year program to patients Rx Ionis LRx enthusiasm

Rx targeting the We believe with also medical Phase patient additional later Baird meeting. XI patients our the risk and factor knee England Roche. forward of first a studies, collaboration This these patients these sharing the studies in a this for programs of those X Based used program XXX a medicines risks thrombosis further Ionis from under into initiated published thrombosis study our to Medicine. in we at populations develop have to for anti-thrombotic advance We're diseases LRx study especially potential prevent recently on medicine's year complementary broadly development. to for or one is in looking in to our New forward mediated The range treatment a to Journal results our without We demonstrated Factor earlier data our future from partner be was both of with significant decision risk Baird's to look revenue to is Ionis of undergoing bleeding. FB both broad surgery bleeding. which replacement conclusion over in total of at

clinical safety study dose Phase geographic approximately X demonstrated degeneration. dose earlier will multiple In profile. and atrophy evaluate positive a robust with target to in we Ionis trials, FB-LRx macular age of The related dependent patients reductions levels secondary XXX

first we year development program. a X up for And X one initiate study gearing global in be Ionis in in Patients we're treated study. are second the The our indication. addition, Phase and will evaluating indication Roche pursuing to Phase FB-LRx in

As year. XX the we from study you driving slide six discussing forward initiations numerous these events to this expected data have looking readout. months and within including more can We're you to next throughout both the value with see

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

Stanley Crooke

culture a are medicines the Thanks, to pipeline our Fred. of We've each a supporting foundation innovation built maximum of us model enables our business that maximize efficient our being commercialized. and in Ionis technology on that value and

we're is Our Ionis value today, every and patients more delivering sustainably And to profitable. strategy And is succeeding. business day. shareholders

medicines or two three in have of years approval the we the so. now approved With WAYLIVRA, last

track the X underway of end mid-stage advance late to continues already year and Phase advance as stage achieve initiations to our the four continue of this accomplished development. year. studies our Our numerous medicines by by since X goal this We're readouts on study demonstrated in to pipeline Phase

continues progress a rapid our as of this well to glimpse at you webcast Our pace. even it's a giving to advance exciting and technology year. our at advancing on later We technology look the more an in focused forward technology

our than, maximize of by have And developing optimal continuing are programs path value more that have we and Ionis account. more identifying the retaining for to our commercialization more economic medicines we're more value. the We we owned and own

we built, short we've and the in that to you strength financial years will look patients rest throughout to our achievements to forward year. this and success term updating our business develop for gives shareholders come. and us confidence The continued the value for on continue our I of

And the call with up that, we'll questions. for open

for So I you up questions. appreciate can set it. us

Operator

you. Please comes Buren ahead. Jaffray. Thank Our first Instructions] Tyler question Van go [Operator from Piper of

Tyler Van Buren

in and FCS congrats progress results, WAYLIVRA, broadened and the FPL morning, in study adding prevalence we good patient with remind and opportunity to that top on can breakdown the and guys, Hey syndication? you of positive the on the the of about think as us respect the existing

Stanley Crooke

Yes, if FCS, population, similar correctly, is is course, of thanks. or FPL is -- size X,XXX to rare about the patients maybe I around FPL the indication and of recall thereabout.

So, and we to the the the believe two be roughly our being revenues. of opportunity, FCS to that think contributor we of will an as important similar opportunity future combination the

Tyler Van Buren

update see respect you so forward? gives that at confidence TTR information a that seen And follow-on twice once we'll with that pathway us give the Phase then maybe could what And product a could a that, far could have you dose LRx or as a you that glimpse be you to then have meaningful meeting helpful. potential what year? for that's in that's additional of follow-up September, to Okay, I the

Brett Monia

that. This Sure, take is Tyler. will Brett. I'll

results results on encouraged So, of to LICA. Phase very we're far. our We're forward looking study TTR the so I by the yes, sharing

all we our can that Our designs share up LICAs, dosing of pristine and gearing our TTR since to we We And utilize, we we can most going share for pharmacodynamic, safety have just we can excellent of choose on based We're look for if data. that's even performing like data are we're free studies. to. two they're will our we for durability glimpse settling you see, we LICAs. LICA are LICA start We'll the the all options show Phase durability. potency, frequency III strategies. programs, less the TTR doing We frequent is and of all safety give or - can on a lowering of It's polyneuropathy - those and with of like have also same. easily, efficacy go with like go we'll what in exact wealth to the data. we'll our the our And quarterly dose the monthly, we'll data that of probably cardiomyopathy, the

see maybe a right frequent advantages advantage to presentation. standpoint. We think now. now. monthly we But patient monthly settling are can want going - the right quarterly, the Tyler, we well. actually a detail monthly on think convenience in don't truth monthly, The more frequency talk we as less to to whatever We're is, that there we on we're dose in about significant to the options And from do. have we going But selling in option I have

Stanley Crooke

the been to So, you once asked a related SMA question about if have follow-on or twice to the year, Tyler, to SPINRAZA? may that

Tyler Van Buren

Yes.

Stanley Crooke

give procedure a that board, us for little doses. case, there's less on effort. requires we're real drugs, a most frequently. obviously intrathecal support or of we think - will it's much new them we think in meaningful our intrathecal molecules less dosing. value quarterly that working I and across Because maximizing space There, obviously a in the that allow administration frequent And between will the

and we've thing So large, to the patients providers is makes monthly And of with. administration, work for through in talked by our providers. is conclusion the we've that to and we're think is systemic talked provide patients, to to way We've frequency that for dose position our I the sense. easiest looked dosing patient pipeline that it and

be great For possible. intrathecal infrequently intention administration, dosed we're And there. as our there can is drugs progress making have as that to

Tyler Van Buren

question. the taking for Thanks Great.

Stanley Crooke

seeking dosing want to intrathecal. not confuse. semi-yearly. don't for different -- make the very patients, or that's most solution think annual even We people We We're think wouldn't systemically. Yes, the are best don't that's to best I but from

Tyler Van Buren

Thanks again.

Operator

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

Jim Birchenough

Hi, program. increases guys. ventricular becoming may first Huntington's. transient, chain general. been There neurofilament That's light appear on on volume being down wild-type and increase, bit of ASOS in to it I is has guess that the Congratulations very and backfill a related some of be one on Couple on questions. all progress. the just high-profile to or a speculative knocked attention

address directly have this do so you and maybe just to to four either and And phenomenon? address be could you perhaps that to think that. more every of meaningfully speak address just dosing to Roche's it'd whether anything whether with helpful month extension had - directly I those

Brett Monia

Jim, is I'll again. take that. This Brent,

the mean, a presenting we're program. this data extension forward desperate I enthusiasm this our medicine. very sure. is to this continuing with warranted in for year. for this label year are looking doing drug is And the in on developing early intention, and Roche program a or Huntington Roche exciting need job So great open for Patients is next

a New in if was was don't continued baseline NFL essentially you reductions, that data down coming by Journal the there you in Medicine; baseline indicates that has ventricular medicine, expect nothing the itself with at levels that with are of transient in that continued Huntington dosing, and was Huntington see But regarding do - which would So Huntington volume And it to was continuing that. England increase be continued published driven AAN of a presented to of with year because and we neurofilament. down increases would continued the and we this data saw as course, related earlier that, to dosing, our dosing be further, indicated, that to back with

not III monthly to by we reductions that, Huntington label With reduction the frequency Phase out to clearly and extension data just on the Huntington begun from respect in and open coming the study at the toxicity. have the of when both study, dosing. early sense was looking bimonthly related in III to Phase changes it's So the dosing

so a was reductions was that on Phase study; And it the had Huntington in expected. threshold getting preclinical is and drug it very even no on decision to expect really that and potential well data including, adverse label produce all III open do patients data dosing the Since they way little had the necessary bimonthly what was clear had. benefit dosing. dosing made for administered to bimonthly tri-annual we convenience potentially they has was offer below [indiscernible] the impact just when in was and readout greater But any the you to events and us that that been to tri-annual that changes or that begun any correlated NFL that the in based or the wanted the with study, we we Considering Roche SAEs transient were -- study. that with saw that the check way seeing saw changes in volumes we related we that to

Stanley Crooke

have than of intrathecal and multiplied administration are with of rest by XX. targeted of And that they I'm is years Simplest experience as With more that let are to goal of that we we at say time therapies the all between related to maximize injections. regard SPINRAZA. once sure we consistent with And our And ASOS are data it. RNA had could ASOS combined well that in intrathecal did to have decision experience we me we to seven do that, look very question well, world us safe was explanation do the tolerated. with Ionis, is general, at the the administered and whether it's confident investors told intrathecally

Jim Birchenough

and Stan helpful, That's Brett.

following antisense approach. milestone Bayer opt-in with Factor could a payable remind that those? what Factor of And Just and an on maybe I the will forward XI the forward level just with if us of the up data if -- on your opportunity both update or you if one directed move that guess comment confidence we'll get. they XI there's moves

Brett Monia

from course, study is of LICA medicine have XI as move and yes, specifically, is reductions, that XI as I Bayer that milestone shown. thrombosis, very Phase likely end all in pathway, our their for XI they've particular. encouraging, data published England this forward risk with of previously study, us our with is this Factor a the and very excited about patients of at in And from And a we data is II very Bayer a millions that prevention drug, knee I mean, in the program to far we as Jim, for based they're good development. as of there and enthusiasm as at on both antisense data moving decision the a commitment of well a safety, also risk potential the to that in well has study a patients. tolerability, New treat for have the been who in about exciting payment Phase bleeding. Factor as the I'm program forward Factor the renal thrombosis, bleeding. has evidence disease the are it's in right we reviewing they presented replacement made Journal program strong so It's program stage feeling now again, seen it's, that And Medicine. to and very

Stanley Crooke

important that Everything are pipeline. we assets they see from these really in drugs, teaches our

through with Bayer And excited them other rapidly But with but us, agreement buyer, that forward products. we're in agents. for I'm we'll So ways. speaking, we Bayer developing that not these are or about for entirely really these move optimistic Brett, will be

Jim Birchenough

the questions. Great. Thanks for taking

Stanley Crooke

you. Thank

Operator

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

Unidentified Analyst

for Thanks And taking I for Congratulations FCS, [ph] then Neil way U.S. limited the touch with would there. FPL Thanks. question. positive in for any could you the just you is there? provide on there regulatory overall the you and great. specifically and you if this hear on the the strategy be the FDA interactions data. the U.S. for delivered guys, But Paul. I'm on you're path details assuming could And Hey,

Stanley Crooke

Well, can't we've further get I'm we the right. have about hesitant you're any We just comments FPL, add details interactions to the FDA. our And with because to today what on data. said into

And to the us to we're population. be actually do to need given finish want take the of different a this, commercial analysis, of It's to what again, criteria of to sorts data. what of disease with they're size so obviously, path And achieve see complicated to analysis sit study. the down and is regulators kinds going FPL; this rare opportunities fairly then the to opportunity with going the of

you're about get information I to going process. a from to the us little time give regulators to So think more just have

Unidentified Analyst

stage Got program And you HAE? maybe one you. sense. and earlier the on the makes for then on pre-Kallikrein touch more just That the Phase initiative pipeline. II Could

Brett Monia

Sure, happy to.

So LICA PKK all -- of safety, like it's are performing with drug we excellent tolerability, And and our and LICA potency durability. LICA. a drugs it's and

drug. pursuing We multiple are for LICA our PKK actually indications

very everything that in we've very HAE activity patients. drug effective that based for now substantially, to these drug, really a a patients, be frequency a pathway first we're environment, of range has hereditary to on in it's potential HAE. so. competitive attacks, providing the these reduce measured and HAE by attacks positive as Kallikrein it to a starting And reduction that patients in we can PKK study and drug. and we're know convenient angioedema; The have seen, forward think or a reducing impact is of reduce we looking We going benefit This levels or XX%-XX%

are to even the we In and indications other also be multiple maybe disclosed is later soon. share that we now exploring but addition, for this broader think, on that particular next relevant coming information an indication And early we're one pathway. than news will year, will some HAE, year, in haven't the that on Kallikrein indication yet,

Stanley Crooke

competitive It's a area.

that various discuss the today about or And considering but to pursuing so, product. we're excited we're pursue, not we're to the willing indications

Unidentified Analyst

info, for Thanks guys. the

Stanley Crooke

bet. You

Operator

comes of go question next from Needham Company. Please & Chad ahead. Our Messer

Chad Messer

for Great. congrats both financial my clinical And taking results. question. and on the Thanks

can or and you one, bringing antisense older and make school deciding of the the describe forward sort a scenario take mentioned even you situation XI, both LICA sense? to barely forward, which Factor you a would For of when both a

Stanley Crooke

the relate already confirmatory patients, larger experience, total study to in really a data data and patients replacement in the In end-stage to it principle, a those we is significant Well, in face earlier This disease. timing. case will and study. renal study of the knee with very have generally parent, and positive very so

-- could the who ahead there, LICA And we're we these and breath that a close as well rare drug went timing think the course, course, tremendously it thought APOCIII was analogous and is with that utility patients that in form, have to down about it because And needs boils it's be of as WAYLIVRA developed, diseases. to had to developed because we the are written dollars. the being excited all following where I up to and it market. and LICA provide of cases, benefit . now, we're and equation we of So being products its APOCIII LICA parent the And really of exactly

So with value at that patient centered and will there, makes write pay equation we and that decision down financially and sit we'll and sense. make look or the

Chad Messer

oral that. to of about the I may Yes, clinic I'll help made then can me. wait be to of sense But details just to getting understand more And have really and about here, an now now, sort what we one All anti-sensitive talk to soon, myself just you because that I'm get this. maybe of probably kind advances about can't technical right. possible? and makes lot on share can that that what a excited you

Stanley Crooke

we that our a take do those to evolving. sure not technology of that comment technology as chance we breadth drug hope about are we I a is performance. place. get faced the constantly get you heard I It's in static. challenge advances to I'm taking webcast, interesting to improvements And in think to advances not demonstrate you place, that is see to And that. successfully. and have where fairly want and this before, I But that return I face have that. Well, And

of a as And to constantly an a that both interesting better to conversion to But pursuing drugs. these so, generation-to-generation conversion years the great have five, pathway it's is challenge in making better yes, to and the have, of is will problem And, it manage. way advances the coming those we of the be forward. to LICA be come managed new

about excited we're it. So

things oral, these the to are regard With I'm prepared to say today.

and If beings apoB-XXX Phase that in you we to look reduce got study showed we in [Ph], at reduce oral back LDL. a and go sufficient viability human Canamrow I

more the really the issue. have to cost So solution where and find it's a challenge drug, enough of of are all there you And that open of engineering of things size those reasonable. is an that the that and the pills therapy demonstrated

So, they're basic taking our when just discussed making commercially drugs, we've and to working we're we been technology attractive steadily we advances right. advances, now it do opportunity. that as in incorporated into we can everywhere that be and observation else, we this ready think conviction our we've a And making have when on been that

that be we're And as follow from that us to so, achieve. we have we meant will what will soon can the course oral, hope near future. achieved confident hear as we you that with in We

Chad Messer

certainly All well, tuned That's very that. stay right, on very, exciting.

Stanley Crooke

a lot know. say I nothing of words Because there's But okay. will

Chad Messer

Yes. And terms anyway, allow game the in indications you obviously so… that to consider, very changing of would

Stanley Crooke

administration as including tried, you actually by disease. now we last believe, frontier these enema local give can that's route Anyway ever demonstrated and the of been know, medicines every that colon instance, it's we I for for have single

to climb. oral forward the last top. getting so, been to And is really climbing it. hill And And, we look we've to the

Chad Messer

Okay. Thanks.

Stanley Crooke

You bet. Chad. Thanks

Operator

ahead. Ritu from comes Please question next Cowen. of Baral Our go

Ritu Baral

Hi, guys, the for question. thanks taking

data know TTR X/X feel how clinical think targeting? benefit. look think are Helios we the knockdown thinking about to Brett, I Especially, think ranging I wanted to it get are the that of need creative of group program guys we as III? what TTR you study, differently data you detail to And Phase release? about that, LICA their Apollo would understand you understand around give significant move patisiran ways Phase to about later. to that As programs? ranging happy? more how TTR make But should the Phase was technology allowed potential dose -- we and you extended you study, III you the forward What I needle use studies should What be level we then, LICA a sort than should the from the you HELIOS-A long guys original on dose for the control

Stanley Crooke

dial review, APO(a)-LRx in question, whatever that is and we first you can pretty we should the I LICA bottom-line think much to to the regard all just want. With refer reduction other

Brett. that And we idea just And know dose. have of us kinds so, picking TEGSEDI leave done then polyneuropathy. the for And it of choice, the over gives out by and of want the of already indication, LICA, ability maximally we competitive. reduction we the there. with level a reduction very results regard have that the to level the that With so for the make we've up trials the we turn clear it's Phase And I'll going to to we're to cardiac that III

Brett Monia

we're Sure, good. Stan. Hey,

Ritu Baral

Okay.

Brett Monia

mean, and have we to. the divisions same are to to recently discussions with the productive you that. talk completed the through we very polyneuropathy study we we yes, let's the we all same final So face thinking regulatory regulators, it, have option and study. We're refer design had if do agencies the to and of chose settling on for type our this I of

more open to option, you rapidly even but certainly as for creative patients drug the be can as to we if think, referred an label I possible, polyneuropathy. study you and this patient that to bring this -- that's

done cardiac and outstanding For for very regulators. with the conducted tafamidis to who study gone cardiomyopathy, to job patients patients, not important and with they outcome get the study particularly, they think for our outcome set is the well desperate Pfizer discussions want outcome have in mean, an me, tafamidis but is for just important patients I an drug. need, of study the the bar. also the I approval, wild-type in an for has payers

on this we component think study, cardiac that annual to will share bring be creatively meeting. patients. to data can I the And at we're but of faster we'll thinking important that an in also certainly medicine ways even our So September some of

Ritu Baral

helpful. That's Got it.

Operator

go question Next ahead. --

Ritu Baral

we or was as and the prefer to disease complement about space, more the space? complement into competitive go the of guys you how sort landscape? program atrophy next profile thinking question potential thinking you about My differential the you And When how you across look And Thanks. trial sorry. competitive are differential less expect could pricing sorry, are Oh, different the complement the geographical indication on markets, next - the do program. data?

Stanley Crooke

the complement we're very against what And a match confident anything in that reducing complement to we're is that will pathways And of that valuable the drug activity. that believe reduces system B. way complement the up really a performance there Well, we general. LICA, overall out alter as

winner. And very the well look as to we study in at been the We some as opportunity study. got large, interesting eye getting it's planning indications well-trod is The longer-term a are think as broad that a have just enter competitive. and and we've So we're underway. those

So how to goes going don't And more start to for handicapping bit those want a to appears performing about I able going enrollment to I'd about that. have talk it's we'll information how be the like study overall and see be the data. before some to be date before awhile you we're to when what

Ritu Baral

the do Got it. next FP you might indication think LRx? announce you When for

Stanley Crooke

a is the Once in involved plan interest the advantage. and again, and number breadth of companies think given clinical of the of that complement pathway drugs; we we the number have competitive

fairly about to anything. we're you going willing to precious And I'm tell very not tell so we're what we're be to And people. today. precious willing

Operator

Laidlaw Please ahead. & Company. question go from Yale comes next Our of Jen

Yale Jen

my question. have two taking ones. progression for far. Maybe for Again, the I the just so add quick Thanks congrats

Could of have you what that, one from some programs also, [indiscernible], give of two program that forward? you we bit LICA First also HBV could data version have with the know little you detail and us of PSA. in these is? is the target earlier And terms I more you a have report what going anticipate

Brett Monia

as this mentioned meeting. webcast, data is the at with us will about And presenting Yale, later Yes, we're Hey, as in program, year medical this very is excited a this GSK. be Brett. GSK we

is target disease into the chronic And and HBV but for that know, problems problems nucleoside a polymerase that they don't the current they virus, of causes still many cause virus. -- the hinders analogues block one you can patients. disease. replication things the is turns medicines important As that That of for root eliminate that

produced polymerase, advantage, all not eliminate entirely, Our to transcripts antigen. that causing is essentially And is us us targets S a there the allows target cure. virus antisense the that replication, a just the significant an block that DNA, all from strategy And transcripts, the our four circular to to there's -- mechanism which those there because there's the to is are using able basically the be protein, HBV but gives mechanism potentially right, core the transcripts. functional

of targeting We have how we're that while. the going to so course. the to ourselves keep More far, prove encouraging. data But we're on is little transcript, to very a that

Stanley Crooke

The and the on parent just, LICA we're advanced parent, both. we're is collecting on And data of more course.

the [indiscernible] at And And in XI, our so be equation which if written other versus one would it than are in the look one would were it of toward and time of these for like those this another of And we'll forward to the in like it's of same we'll optimistic program. use we move Factor choose to X Obviously, of that drugs those will performance well. look But both that back. have situations at choice the all case, we've other. sort advantage as decisions. GSK the make with delighted we'd GSK we

Yale Jen

Okay, follow-up here. one questions Maybe great. just

that injection intrathecal less mentioned product and dosing. in basically frequent that equity and have the You even next-gen you great more

revealing secret, of other of -- potential? without general, what typical sort in enable Just of sort your or these even formulation base less type aspect are to the frequent dosing

Stanley Crooke

and saline. our to medicines change for higher the medicines, these in we like in dosing change longer It a are basically chemistry oral except duration I there. supports not mean, It's that in than choose formulation. go all should formulated and just if also of

is engineering molecule make fairly art sort much we've dosing, a sure least that. every really six of us months challenge. to to for form that's than at then got but hope give it's that longer the here And straightforward So going

there. I almost think we're

our as X.X how forward. is and that to applied to that And we look learn how then of experience just obviously, Generation that apply And to all we systemic so the be LICA forward all programs. do teaches can it, to programs as can do moving it, that something to intrathecal we program as of our

rest the it entirely Yale. way in analogous in what of done So of we've body, to think an the

Yale Jen

antisense for possibility the in indication? maybe if for yearly years best instance, treatment have, specific your that's could most you dosing in in of two paradigm Ultimately almost product

Stanley Crooke

Well, then I couple next have in dosing seen here system. we what are we're we've certainly obviously be frequent am have the going we're the the going clinic kinds all optimistic not far we forward. we'll are bring to animals, to we our very that forms But And and administrations be good once to see years. in ready to accomplished making in our nervous those But can to say less we going converting able humans. to And of progress that that, to of in intrathecal that central improve.

tuned. stay So

we're excited to be going think to you. what I able we're share about with

Yale Jen

it. Appreciate lot. a thanks Great,

Stanley Crooke

bet. You

Operator

comes ahead. question David Stanley. go from Please of Lebowitz next Our Morgan

David Lebowitz

patient for Quick at chooses the on question. quarters point? out market, question several there is point TEGSEDI TEGSEDI. of Thanks taking a in this profile To other that my this after typically options there specific the versus

Stanley Crooke

freeing Not wide range too patients hope of freedom because have limitations is But the that of today. yet. that And convenience its I TEGSEDI, from up to the of our course early of of disease. a think it's of judgments we and the make about that have administration

David Lebowitz

from for approved it's that understanding Is anything there I'm on a doctors? any And enough. different Fair commentary or Tafamidis feedback indication. the hearing or

Stanley Crooke

Yes…

Brett Monia

from We're still haven't neurologists impact. impact increases of we're terms mean, TEGSEDI, prescriptions cardiologists, still seen and on getting of I an much we - So in in hematologists.

the last in We quarter. the prescriptions XX% a have U.S. increase in overall in

during a the call million, quarter. you As first is the increase that earnings the which XX% was over heard $XX

impact. we it's really haven't- but early seen haven't still, we much So, of an

David Lebowitz

for taking questions. my Thanks

Operator

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

Jessica Fye

afternoon. good guys, Hey

be Following expect on focused when up on do that is the will data question earlier OLE presented? physicians what you on Huntington's,

Brett Monia

And think I how Well, which be will OLE, on what's expect I history But in there'll and looking certainly clinical know physicians their It in the physicians would safety, be think patients and or focused signs of natural mean, months will reductions on endpoints, lots don't be the with of that Jess, expect of that will aspects I presented. that's data be longer focused And durability for. I be -- clinical getting some yet of tolerability. XX with the XX sure in months to on be patients out. they're we'll to we update be we what the of of for be results on drug. dosing, see are And going as the to well. published some think going considering history Huntington the think will I parallel things. natural would mutant data,

Stanley Crooke

We've efficacy described measured that the the are endpoints going be endpoint. to for and Huntington's

of endpoints analyzing So to data I all the safety, we mean, tolerability regard will with that. be those and

Next question please.

Operator

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

Jim Birchenough

guys. hi Yes,

Just I profitability. had level, tremendous a a just had Stan, follow-ups. of couple and revenue of higher you've several growth you've at guess, maybe years

focused get in at people on financial from stay growth Just that the start perspective, necessarily growth, too goal continue at where positive, earnings focusing on a reinvest line, we not earnings is business and cash to you or to point are the here should but flow are the grow point? the top we

Stanley Crooke

me that's answer, Well, revenues first are where we you investment sleep. of to just Beth all, he's will moment. do financially. We're that's earnings necessary. answer a because while Beth increasing And to and correct give growth then Jim, and about you, about all is you if for we And continue my really the growth continued continued next in excited us, see sitting exciting we and And that. thank in give to go I'll and

on we would opportunity as of that an have an create we Exciting extraordinary still and focus pipeline earnings I earnings to been an you to kind advances. increasing value that's growth, with So, the are say focus. story and welcome today pipeline us, and stochastically

Elizabeth Hougen

Completely agree.

Stanley Crooke

time. That's the first

really thanks We completed questions. with financial do everyone and Okay, I Q&A. I keeping the that. mean for for and -- that, look we've question. to think excellent to With you thank asking the attention forward Jim, progressed. your that, thoughtful I want I

that you. expect had you. we sharing commercial continue look with and planning financial We an exciting to coaches history the year as forward to extraordinarily successes successes, our high report moment to in our Thank progresses and

Operator

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

may now You disconnect.

Wade Walke	VP, IR
Stanley Crooke	Chairman and CEO
Elizabeth Hougen	CFO
Brett Monia	COO
Damien McDevitt	Chief Business Officer
Tyler Van Buren	Piper Jaffray
Jim Birchenough	Wells Fargo
Chad Messer	Needham & Company
Ritu Baral	Cowen & Company
Yale Jen	Laidlaw & Company
David Lebowitz	Morgan Stanley
Jessica Fye	JP Morgan

Ionis Pharmaceuticals (IONS) 7 Aug 192019 Q2 Earnings call transcript