IONS - Earnings call transcript Q1 2018 | Ionis Pharmaceuticals, Inc.

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Operator

Good morning and welcome to Ionis Pharmaceuticals First Quarter 2018 Financial Results Conference Call.

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

Wade Walke

Thank you, Brian.

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

our also discussion on that today. slides our We posted accompany have website

introduce you today, has Before and to me working I Relations President Communications Investor I on for our will of with call front. our joined She strengthen working be already Vice Patterson closely this the introduce would to functions communications speakers the as of Roslyn and member Communications. our are to closely latest like team. us we

the expectations statements to risks results in website With available the are detail. that, uncertainties we on to beliefs. our that me certain have to Financial actual and Stan. will from I Monia, our Now, the of like out Hougen, Akcea call additional would and Boyce, we over based and may or and risk call that for forward-looking which speakers to Stan point subject our Therapeutics are are encourage let I'll materially. factors for Crooke, our the discussed me on Sarah and Beth statements, today I Officer; differ Chairman Operating the you current our be today. Officer. These introduce turn of today, Chief consult Chief Executive the Board President making Officer; Chief SEC With company Brett filings

Stan Crooke

for let you add good to delighted my me call. on have and today’s us morning joining Thanks, to and Ros. welcome Roslyn, thanks us join everyone Wade And

start news commercial by into the we inotersen, results, TEGSEDI. now we to name financial with on its which refer let me Before get our

is we data NDA the As additional needed provided The to their time Akcea October review date the FDA the yesterday, responses and to them standard requests. analysis X, announced TEGSEDI and PDUFA XXXX. information decided they new

be marketplace. team in demand approved. as enroll the confident are ready be early TEGSEDI Akcea soon The to patients that program TEGSEDI will as continuing We launch access highly to high. and is the continues is competitive to The is

Recent of our more leverage cash. product line. first create the two our massively Biogen, the of a We under $X earnings largest pipeline By in Now, create quarter to and our to profit of develop a expanded to with its into our further multiproduct technology to quarter tailored earned the of approximately turning those first transform our the strategic and opportunities, We the we we using plans ended partnering verge of the we By to large maximize our technology financially In the technology of be revenue strategic than infrastructure neurological strategy. XXX of last retaining to we that at to the advantages both financial quarter XX pipeline first advantage, developed technology. strategic advantages way, partnering consider call, XXXX in and million, strategic business position driven technology last those opportunities a through examples revenue first profitable operationally action. by our began our ever, profitable our possible in those opportunity and by drugs we in XXXX continue the forma to update. earnings compared innovation core. Biogen infrastructure. with company be commercialization our pro we on Since as a our position increase in Akcea becoming of in breadth operating for then with convert while strategy large, strongest them in that diverse risk. pipeline, the Plus, and strengthened affiliate are and our strength increase disease globally with commercialized and year. while We our innovative that the a antisense in and a efficiency and minimizing our large of received our commercialization billion research financial of combined extension collaboration $X and $X This antisense each drugs. quarter billion for development coupling we in commercialize, opportunities expansion strategy be we advantages the with value reflects million the we've cash. payment and collaboration neurological excellent TEGSEDI of can for the of and from of We very which created diseases billion to need broad investment XX% fullest for

exploit by Our provided our development the for and and of technology. the in is strategy benefit to of potential drug, patients, optimize participation designed maximize opportunity the commercial the our each breadth fully commercialization to potential success success drugs of maximize then antisense these the

is succeeded today. on in strategy Our these delivering goals

have We the therapeutic antisense in patients areas. to many of value demonstrated technology

very collaboration Neurological experience the See listed. more productive the This access select need need often use is and all commercialization variety relationship, of breadth opportunity SPINRAZA Ionis large as of clinical collaboration and pharmacological achieving fixed to growth teams are development SPINRAZA’s in range builds knowledge us approach that they ever. intellect, and diseases, SMA portfolio need remain patient We of foundation just its believe criteria programs. and approaches and the those new diseases of now care non-deposit to stage strategic understanding of about diseases strong. argue cancer. neurological form disease large They research and measuring possible is that launch areas, specialized our costly manifestations. focused phase These breadth neurological forward in therapeutic novel our in ID models to why it diseases, X a therapeutic on of and represents enormous or therapeutic disease expertise a to of of challenging Biogen. produced testing SPINRAZA the strategic the patient, clinical to and/or spaces. in involve broad diseases successful continued Biogen We sense for and similar means drugs and and launches multiple SPINRAZA best and advancing the especially nature It strongly population is -- for drug with broadest Development paradigms, standard of many complex endpoints, which the share made the wide studies a This such combining benefit. the collaborations and of research developing are characteristics. the areas on with experience Our one disease infrastructure. a large require targets already technology many of the most that with rare sales sales. success. look in

SMA. created SPINRAZA also progress to we next Biogen, generation We’re pipeline for drugs. like industry drugs Together by diseases we've treat with discovering in and create of neurological leading drug to an hope more our encouraged patients from

Our several important collaboration. for strategic Biogen is an new had partnership with step goals the Ionis. for We

this and focus for the First, would support to focus that important would Biogen’s on would mission companies be both unwavering. that match relationship the intellect be ensure and commitment strategic and ours that and

value collaboration. maintain third, own created. ability on outside the the not Biogen all of reflect in And participation increased to goals. would our to commercial the advances Ionis’ just financial collaboration, we've new but with on the ensure we've focus collaboration through our neurological created programs and achieves made our the upfront, value diseases appropriately work participation to significantly that the This technology, these Second, in

the close commercial earnings stay And value growth, match and by by provides It substantial TEGSEDI. our and of invested of TEGSEDI expertise. advancing strengthens milestones we with should $X.X Commercializing areas the Akcea affiliates. for we ourselves Ionis Phase of commercial Our Akcea. we commercializing focused what investing commercialization $X we through In commercial innovation the With and along make patient of that and We of where TEGSEDI revenue consider create similar commercial strategy also to And believe it shareholders. these Ionis effort should the rare drugs our X support, very plan our can includes benefit This then such the in national in the have substantial billion manageable leveraging drug even With those our of best we importantly, billion. in already ourselves through of drugs commercializing of Ionis participation is more success believe of drugs for invest generate cash. do commercializing to to Most antisense that attributes. preparations out the successfully those pipeline in to XX% drugs than endpoints, pivotal coming affiliates. by infrastructure. trials, high Akcea’s with transaction TEGSEDI now. on commercial diseases Ionis greater our on focused approval it commercialization is through to substantial our cash our to as that focusing uses March, launch TEGSEDI provide in we commercial ability following upon clinical Akcea a profit ourselves with completed small the licensing best, touch could We with share positive therapeutic one our Biogen, commercialization technology. up of ensures

this. drugs. maximizing to the And to patients of believe the potential broadest We been greatest the addressing range bringing value aggressively and are continuing on our of we've we focus commercial

the TEGSEDI review on provide Now, Beth update Brett I’ll now financial Sarah over pipeline in will an performance commercial then call to preparation our and our accomplishments. volanesorsen, turn detail. discuss will And WAYLIVRA. who key will our more for

at up call call briefly the very for the questions. end of open and summarize I'll Finally, we’ll the

Beth Hougen

our million, multi-product Stan. We from on we made with company. our continued million first further of performance and In $XX both goal a first quarter being pro-forma the toward operating the income progress you, $XX of basis. a and financial income quarter, of Thank net ended XXXX profitable strong

including increase on of QX SPINRAZA driven by royalties million we from were results earned revenue, strong a from of the our forma our year of keeps sales. third from primarily XX% seventh the consecutive $XXX XX in pro earned quarter, first million forma revenue, consecutive In commercial we Our to of SPINRAZA. income. achieve Completing pro income operating operating quarter revenue us track

and to this we nominal loyalty royalties are the the of reason, started tier year share XXXX. that increased means tiers non-tiered. SPINRAZA the sales party during leverage through of SPINRAZA’s each on fixed the year, the for beginning pay and reminder, and throughout royalty tiered This as grew. We third was product at quickly that sales a of tiers we As earned sales through fairly rest progressed our this royalties -- annual advanced

$XX amortization, commercial the with in we first was par we more first quarter from million R&D licensing of our included drugs XXXX, services on last performed XX $X million revenues, QX to of of R&D two million which partners. addition $XXX and quarter in for of we we million revenue than had the In year. earned for AstraZeneca revenue, earned for

the And to the we pro quarter to prepare increase continue forma were first XXXX related the this as TEGSEDI anticipated, was year. we Our XXXX. compared as our expenses of $XXX to in expenses primarily increased and WAYLIVRA million. first for quarter we launch quarter to The same expenses incurred, operating both

the we in research development We to billion and of of strategy billion to and we the $X morning, create our on these while strategic for license also transactions Biogen performance will enables the the innovation. This the the upfront term the improve expanded near ended each to when This each identify financial $X I’ll grow in first strategy which transactions years. quarter. commercialization Each our quarter, drugs program, our first Akcea. company this coming value of pipeline. reflects anticipate with received and quarter us later to these keeping In investments, core for most will TEGSEDI and the our Biogen more over focused how payment than best with for our plan in each collaboration we describe the of cash

payment XXX antisense for higher that collaboration, the the million of is pay is Biogen upfront $X to collaboration payment purchasing. us will this technology. our Biogen stocks As price demonstrates, for a substantially pay willing billion. access partners Biogen Under to are Biogen today of an

diseases. So you as neurological $XXX for million fee can antisense to other access a view the our technology

begin approximately year. be next revenue million will the amortize the will second access amortization this $XX the quarter, technology provide annually. should million than We in fee more Beginning $XX of to year, which

fees license In $XXX we to access the of earn and addition drug. fee, can technology payments million to up milestone per

productivity we are economic a With these opportunity this expecting payments from the Ionis. for substantial collaboration, constitute

will up drug, to market, that pay in In drugs enter contribution on identify responsible The ready development. Biogen will enumerated and thereafter. will required work I and to each Under tied for the be addition royalties collaboration meaningful we to XX%. activities Whilst the Biogen. will a this paid dollars in collaboration, development of earn it is develop reach commercialization of per identify dollars just hundreds we year a for of program. as approximately to from it, are drugs The value all the this be targets we for by millions all not included drug through of five

Importantly, this the bottom after drop identifying of for fees royalties us. license means that drug and line profit payments, earn as all milestone our directly the to we

pipeline and throughout so, our infrastructure our We benefit Biogen we also Ionis leverage financial And focus drugs. Ionis to pipeline, significant internal advance and development development resources, expertise can because of including our realize can doing in owned we expand the process. resources

impact new our collaboration of on be financial substantial. We expect future this to the results

has be Biogen Our on this and collaboration every $XXX from we we revenue to Historically, million license milestone expect so. year even and earned collaboration and our have been payments existing relationships, increasing amortizations, successful in is year. in more revenue financially average per fees new Biogen our over

revenue fees currently previous the we milestone year gets We be million year from it productive and we with previous existing drugs in add should doing together on milestone as course, the this per be to are $XXX commercialized from programs their from receive to payments will least per over from with those fees we of average SPINRAZA. revenue at amortization growing continue drugs to going, as million new expect collaborations license $XX or This commercial that payments collaboration will And adding license means approximately When fee. as and of our upfront as from either advance. new access technology once collaboration, we're royalties collaboration. expect our

shown only R&D our Biogen six WAYLIVRA, TEGSEDI, from our that SPINRAZA other continue last to market. so and from commercial is not plicamycin add expect to years revenue, expect revenue in we not partner, over our course, advance our but revenue and to drugs a to substantial have Of only we growth in that, pipeline the growing also then we commercial the

pipeline own investments made to ability and substantial enhances increases our drugs to our cash one It longer for of also Our TEGSEDI. drugs balance ability the a pipeline, expand our and our retain with advance growing build account. recently growing strategic like our and we make

options, As TEGSEDI. by range TEGSEDI discussed success considered was and pull substantial TEGSEDI commercial the approach recent conference achieving on we assuring considering maximum into Akcea. our we best call, of far, thoroughly team to After a of concluded that options market our a the share commercializing the all and we to for

believe from through and profits in Akcea. on ours, drugs. payments benefit TEGSEDI’s as the Akcea’s to revenue substantial successful TEGSEDI, record growth financial disease we in expect significant it from maximize we we realize P&L, our with up consolidate XX% billion because typical Akcea is benefit driving TEGSEDI milestone important TEGSEDI investing we TEGSEDI’s commercializes TEGSEDI Assuming from increases generate product received of for we the we is of growth. rare cash to commercial the in potential successfully we sales. line Also XXX% $X.X our top commercialization will Also, that in sales will And success The several earnings TEGSEDI most results to the be, will ways Akcea.

Ionis’ in our commercial our increases. drugs. through In participation commercialization both WAYLIVRA value of these and cap for for Akcea and increases, of investments should Ionis, the maximize market value addition as of both this XX% TEGSEDI and the We value drivers ownership believe Akcea’s to value independent TEGSEDI’s

two to housekeeping turning Now items.

to of method chose so to full rules achieved now periods, and R&D compare for revenue new will means we an period adjusted beginning companies for for many this ensures which new are we apples payment. from adopted how year, primary we recognition. as these comparison. accounting we aware, guidance, that standards adoption prior periods the for we show trends We in meaningful. new he are earned milestone the revenue First, amounts This this to the you are adoption method amortize event. required using new activities that performing retrospectively, activity from Previously, recognized updated perform we and in revenue statements. The periods for resulting in payments prior when adopt milestone the we payments milestone the We financial over our change the all rules that we our standard to obligated it are partners. Under recognize apples when those we're R&D our historical present

We recognize in on milestone our the when will we earn based partners’ achieved. payments full is continue to activities milestone

Ionis’ earnings a for we had Akcea. include understanding financial aid the P&Ls to press work, Ionis’ standalone our in Secondly transaction and P&L. and in that how shows schedule release, Akcea’s intercompany statements Ionis consolidated The

a We schedule the that balance the company's for same also include sheet. information shows

goal our we consistent We pro even operating multi-product third project are of profitability, and our year of being to TEGSEDI meet consecutive company, WAYLIVRA. be to XXXX P&L guidance, with launch will track forma we a as our profitable on prepare XXXX

revenue drugs will revenue in looking these adding the what R&D base we from beginning We quarter of believe be second are revenue growing and revenue forward Biogen that two collaboration. to substantial our from SPINRAZA our will to new include

WAYLIVRA. our by a TEGSEDI investment making of expanding significant drugs in are we and and advancing also $XXX the reflect forma we’re a levels portion value commitment of to XXXX, to launching reflect retain commercial and These million pipeline. SG&A our $XXX of They the of For expenses expense on $XXX both expenses to pro million $XXX our million projecting R&D million, basis.

billion of to $XXX more cash exceed end. cash significantly to expect due With at the than cash we of million our year have guidance shortly, of $X

with plicamycin the TEGSEDI, to TEGSEDI position company, coupled to broaden ability multi-product cash means our longer can our WAYLIVRA. deepen and substantial TEGSEDI. of to important to we portfolio, And Sarah expect make for a will pipeline update own retain This our now, WAYLIVRA to accounts. achieve with our our being the a from SPINRAZA, we like XXXX, the illnesses. like our from revenue one turn build profitable patients over goal In delivering medicines of call on drugs Our with recently growing partners also supports made help I'd and us an revenues and provide serious we investments and commercial advance, drugs strategic

Sarah Boyce

Thank you, Beth.

with in finishing preparation to are We late launch stage discussions on authority regulator drugs. our both

We have next week WAYLIVRA’s for forward look WAYLIVRA data an advisory we committee review meeting forcing rich and the package. committee to to

the the decision detail First, information FDA requests. in volume response the NDA. we to time standard extension extend that FDA about review to the was the some to provided related the TEGSEDI additional for This of information

allow We information Let data. this were data date of new filings no FDA we’ll quickly to major clarify, to review extension. them me The with provided possible. the we To review as appreciate give no relationship we work to some continue the of requested existing have determines succession the the you were neurological close advance represented for collaborative the requested. is the context division of that new to amendment PDUFA the preclinical and time studies to or clinical The NDA. FDA the and our FDA with closely as

Canada our well. progress Additionally, continue and to EU the with discussions

and review unmet from their significant believe in We have of are TEGSEDI launch designation approval. HATTR the underscoring prepared and need Canada Health successfully the both again amyloidosis. for priority We treatment severity following we WAYLIVRA received to TEGSEDI,

skilled highly built launch about the briefly progress. morning, these successfully across will talk drugs. of I critical both needed teams This to functions have We our

licensing earnings informative. successful for about you from it of Akcea’s details to our that our think major is Akcea I preparations, yesterday additional call quite The find will commercial TEGSEDI For enhances advantage launch. readiness

a advantage and distribution greater addition, to that support number offending needs due we’re as drugs, are taking the meaning more will we synergies In support, similar profitability, of of patient are efficiently, market such there both that access. of

the a Our US, training modern meet benefit our robust, and and WAYLIVRA. injection promoting includes we high and touch in adherence patient have term program to to reinvestment. support diet We believe from of a program TEGSEDI goal built patient thus with long support strength, optimizing nutrition,

operations chain US. We’re supply using synergies the WAYLIVRA, us and operational provide should which TEGSEDI with valuable the strategy same in for and

and for EU the party US, pharmacies are and specialty are Canada ready Our in third providers and logistic launch. replaced

TEGSEDI for waiting be WAYLIVRA and launch to our Importantly, both labeled. ready and are

We are and advancing market pricing research including house activity, economic our research.

our in initial We’re also engaging with payers market.

Our and to have patients of to transformative economics to value potential these the the that these healthcare ensure substantial system. the drugs medicines reflect and patients goal is access

medical Importantly, identification. our are excellent with making field awareness teams sales patient disease and progress and

were and building TEGSEDI clinical physicians, build transform to while advocacy advancing trials. community strong patient and KOLs continue and the and the foundation time the to We created, on Ionis WAYLIVRA in Akcea

high of We of the of hereditary TEGSEDI visibility and at long ISA several amyloidosis. ATTR have TEGSEDI safety the demonstrating benefits data had term with presentations AAM, potential patients in

we expanded at addition, are both the sites rapidly. physicians response and TEGSEDI meetings the standing finally, enthusiasm symposium number enrolling well with expanded in continue In from patients And of room we these only. of program. access and under for to patients, the WAYLIVRA to are the way expand well were and programs In the held access medical attended very

of the these with the the progress talk goal these our about over And both the to patient launch approval call successfully life SPINRAZA to threatening debilitating, poised of diseases. and turn living of to I’d Brett drugs in like after quickly the are to now, other We with pipeline. drugs life transform to

Brett Monia

you, Sarah. Thank

diseases, the in in As ever commercial and the Stan we by wide strongest position position drugs fueled directly both of patients our opening, This is in as range our strong trials company. with of treating setting. in in a successes clinical his a mentioned are

and is in continue to our with antisense success we supported make today the in Our advances technology. future the

is our all-time pipeline across productivity broad and an high. deep Our at

$XXX both substantial combining type first and quarter antisense that exceptional commercialization. of can the our its to an of expand technology history. by to of in is first put partners unique last to from goal saw hand-picked efficiency number the number of to and be launch of the over the commercial into to we first Ionis substantial launches most and continues is US, continues in contributions treated the advantages disease disease on in technology. neurological of strategic US increased million leverage resources resources QX SPINRAZA’s programs meaningful SPINRAZA from significantly the class global best in from and patients coupled of progress create continue XX% outside XXXX, and the our of to is to one partners successful patients globally franchise the toward owned the example partnered with both We Biogen’s drug contributions advance utility SPINRAZA rare expertise, and our this SPINRAZA and Biogen. Biogen of with our goal, sales grow US. drugs In antisense approximately by our and quarter XXXX. of drug like the

largest growth pediatric still is in growth with the in patients patients. US, is reach the long-term believe there to believes the older opportunity in Biogen opportunity While they infant significant US

additional success with the like expects older to our We years. SPINRAZA seven XX to drugs lead Biogen and over X upon to neurological and To pleased Biogen by additional an very to access will build to sales year. our countries treated in this expand produce quarter. we're of Biogen, are with SPINRAZA patients, XX countries bringing SPINRAZA secured Biogen’s treatment very the Outside by the over as in of increase in next these efforts by collaboration diseases the already end the Biogen’s course productive in the the growth receive the adult reimbursement reimbursing markets, additional relationship on are US, We encouraged the patients believe evidenced first that in reach SPINRAZA to more total year. efforts

We our take of neurological opportunity we Biogen directly broad root of new novel for parallel, Ionis types In in with advantage continue this focus diseases confident neurological evaluate neurological are of advance these is disease new to plan of commercialize down on diseases. programs technology. area, rare yes, and XX several address follow-on, our we a and likely chemical antisense We disease [indiscernible] Our Akcea to our through diseases. range an add ourselves the pathways neurological covers TTR-Lrx, we Alexander’s are next in very abilities build more. doubling own TEGSEDI currently will have the and investment pipeline to expect mechanisms, causes to its X and drugs in neurological to months, diseases. and classes, and and We diseases owned aim affiliate. disease, These also and many are Lafora target in over

in study several advantages TEGSEDI, drugs regulatory this for the Ionis development, annual diseases, review, drugs XX presence area our SMA, Our in suited been a make of on ATTR We've gave recently antisense than preclinical a highlighted HTTrx well diseases, stage XX our diseases. technology’s antisense success of SPINRAZA. more including Huntington’s drugs unique disease, Alzheimer’s breadth disease, discovery neurological leading major investigators Neurology neurological or X industry amyloidosis, it for an drugs under create and Academy presentations studies ALS neurological built disease, to four Huntington’s researchers diseases at and and drugs Ionis American very was including neurological of by meeting. hereditary in the and disease. drug, range programs. disease to Lafora of treat broad The have successful therapeutic including our in with franchise commercial blockbuster

the session platform neurological the to She diseases. addition, Dr. new antisense study pivotal plenary dedicated observe of in meeting. is therapeutic huntingtin to certain session we protein, working that substantial huntingtin measures drug reported and major was a mutant mutant Roche this improvements later of our advance data the the in year. Sarah addition for in hour to was disease. Huntington's from a the of conference reduction into that program between correlation a special this In as highlighted at reduction there during Tabrizi two Huntington’s clinical protein disease

the now currently in metabolic In renal five X.X renal development LICA collaboration. two collaboration. have their substantial AstraZeneca quarter, AstraZeneca. and progress AstraZeneca neurological our advanced pipeline second in diseases, In that and of technology in includes under We cardio, cardio, drugs we've generation first and technologies. made also our our drugs cardiac, X.X out and The generation use our diseases renal licensed oncology, and the and AstraZeneca we've antisense with advancing metabolic addition drugs three to cancer metabolic third drugs with for

next the including disease. IONISXlRx drugs with for set patients potential for treat the The NASH and We're Huntington's patients IONISSAGXX.XRx clinical with X.X cardiovascular or developed advances drugs have and being first we potent phase year LRX for targeting. to collaboration administration also of study we year. owned LICA quickly are drug patients than to or both higher our on next thalassemia. focusing our either or with recent advance is first X.X our our partners drug, to commercial have This toward drugs and X low neck our later with head Akcea that LICA, cancer for disease in chemistry along expected drug combination we cardiovascular that studies GHS antisense AstraZeneca enter with incorporates By very X.X efficient metabolic anticipating to in opportunities X.X generation market, LICA beta This generation with licensed drug cardio, the IONIS move supports a cell us renal our multiple This or infrequent our driven the This people and patients is of [indiscernible] IONISAGXX.X-Lrx. and generation under provides affinity and drug at this with with include doses. Ionis technology. IONIS-HTTrx that combining and many substantially generate with specific into We're for generation made LICA disease, have so. drugs patients and plan also more that is

With With the plicamycin potential and XXXX the other to exciting close. making WAYLIVRA launches of progress we're turn so in and pipeline, we call drugs Stan expect that, TEGSEDI, another the be I’ll our to to over with many year.

Stan Crooke

and our progress participation invest our continuing R&D $X that are new increased over and Brett. ever. important pipeline Ionis The financial in while results drugs financial advancing commercial drugs. owned growing Thanks, position in pipeline, aggressively drugs current more for our demonstrate we've drugs today we've financial – more position we With in our can strongest than and reported billion of cash, adding commercial toward revenues needs, strong in potential made we our success,

us. is Together drugs So market this substantial the year we're so. next in late partners drugs or that We we partners, multiple not put year. into our down. this advance year and potential studies the with to We’ve for new pipeline an exciting deepen already we and three slowing to the this to time stage progress on made plan our with pivotal plan our have

developing commercial programs, days next to that stage our opportunities this than also more these increase out could read drugs programs commercialize years. have our in XX We we affiliates. grow pipeline and X are many have also commercial potential few our We are which as one late more than of Ionis now to owned year. candidates The from ourselves studies of the through

of the you the substantial are and I’ll on profitable range open And becoming horizon, verge generating and successes we Brian, the patients and a Q&A. multiproduct serve exciting many a value to recent us diseases innovative company now delivering the with events for if on of up patients that, call can we medicines set for With with please. shareholders. the our the

Operator

Fye Instructions] from comes Jessica JPMorgan. Our [Operator today with first question

Jessica Fye

to as ask extended through you the able in kind just amendment of to which explain FDA And be efficacy? your the PDUFA, the on major ahead can the by specifically to responses it the extend they did go TEGSEDI three and order months. to characterized mechanism a want and or time i.e., you also about whether on elaborate I more questions needs to review FDA safety responses can related

Stan Crooke

focused The the just understanding broadly. on drug primarily question is

great a lot do So, information there is to of of deal with safety. and a has it

So, a by they're the to the to time use use on the mechanism amendment. this drug the focused And didn’t they having opportunity just major getting, well. understand calling

Jessica Fye

adcom I next focused just setting folks some too. week, think And particularly documents and expectations on on the WAYLIVRA can set for you safety that product have been into briefing also

the set should we So advisory committee? for expect we those focus then can documents you of stage when potential the what the maybe and see

Stan Crooke

studies think and in can the benefit particularly be of that are obvious will in benefits on risk. a of type of focus think the I and the be context substantial, conducted WAYLIVRA the that both rare We it the on there safety monitoring manage will a patient to should on as extensive And are and assure be And focus be information of disease properly. will be. population. there we dosed focus course how that patients

patients As platelet those in counts. disease common excursions counts And FDS. of in exacerbates a are the we reported, excursions clearly, platelet in some feature WAYLIVRA

to -- there the have and a certainly to laundry of list believe on the drug. concerns that and the focus there will will the of I to from meaningful the so course And monitoring managing discuss of be will in dosing. And then platelets panel. monitoring other the side effects want the the FDA we manage additional disease response with And be

Operator

Laidlaw question Company. Next Jen & Yale comes from with

Yale Jen

My first two here.

I you have billion bit of X First but the know specific elaborate more in any this do, and things things or with that a contemplate terms one to mentioned of amount you have in cash is currently cash roughly company could other few little hand. you you Beth, would could a

Beth Hougen

pipeline own in I billion technology, use the is $X longer, efficient drugs cash Ionis antisense account. in our our to with as out our as pipeline commercializing invest building obviously, investing retaining think a our I and cash, of our of productive and pipeline powerful technology, our best in and owned for So, drugs in and own the technology in

Stan Crooke

will in We more allocation on year, our Yale. detail later be plans the capital somewhat overall providing

high very that problem. So, a quality we consider

Yale Jen

guys. one the the having of probably affect plan you anything your that entered And follow whereas or to terms maybe marketing date probably market be TEGSEDI, up would ahead of any PDUFA dynamics? pushed Does here, out in other the October, that

Sarah Boyce

Yeah.

repeat please? So can you just question the

Yale Jen

the than on if I you it? mean, feel lack that slightly case, or later you will the the a entered that market probably or is earlier, that’s TEGSEDI bit any there feel little do the of entered impact anticipated, you make potentially which market

Sarah Boyce

very going any have together make from following prepared any was to really be to We ready the launch enough difference a and impact not perspective, that’s and feel to don’t to Yeah. drugs well approval. really launch will be close

Stan Crooke

the to be patients. the to two profile share use. the confident that both of and will we’re determine is TEGSEDI good profile attractive to in drugs treatment the both the drugs end, at will these and the We used be market be the market of going that additions it patients for many think In are drugs

Operator

question with next from today The Stephen Stifel. comes Willey

Stephen Willey

Just the actually. pipeline a couple on

with how about products more just thinking antisense to utility as oncology the few And something than being be able modulation target specifically, might think complete the about achieve X, you've So, you're how modality guess you of I understand not curious to now. got sufficient to Phase receptor X treatment I'm oncology, programs. inhibition. guess concept in KRAS wondering but like how for stat respect in in I respect I a a antigen with that

Brett Monia

and This I'll is that. to Brett to respond try

-- generation us cancer So, with microenvironment. as was X.X as ability as strategies the a And our and to chemistry. it really cancer targets this in generation in get immunooncology breakthrough of was chemistry the tumors conduct at X.X development actually directly cells well reductions robust well tumor Ionis real in to very as target enabled has

benefits they’re contribute the those remarkable applying tumor tumor We've Cells programs that and program both X clinically. and AstraZeneca clinically in applying stat and dramatically shown things, that preclinically the and too. targeting shown sorts – pre-clinically seen And we're macrophages of to the other actually Our to both shrinkages, has and of which both T activity responses tumor mechanisms. associated has we’ve

that We for shown preclinical we emerging KRAS data has have a tumor program, too. are programs other number for targeting which direct great and the have then of

in get in tumor you reductions suggesting, macro both environment. actually chemistries target more in we may much with be robust new So our than tumors and

developing drug second the and first both cancer, X neck expect this second future and that head year. news for the stage late outcome of in development currently line of we is AstraZeneca Phase on in study and line the half

Stan Crooke

interesting established something really collaboration with insights if AZ, important well cancer perhaps think Ionis, it access in some Anderson many strength new in in to in has addition cancer targets, going to MD unnoticed as that for us, program And the gives so that’s and extension an it the because developing drugs is and well trials. bit we've the we that gone through cancer new to into us cancers best Anderson broad is and clinical MD in approaches a a of brains of as

benefit and have we’re years a provide quite position that were in three, So that we're in because and we now it. cancer confident different what confident from seeing we're we're cancer we four can drugs ago in

Stephen Willey

targeting metabolic believe got the I you’ve technology. don't And a more, liver, cardio then of are a that which just couple like one assets utilize

to of programs, should or as I drugs these GCGR we conjugation forward think the incorporate into the this do to like a is maybe you push get it backbone think is these expect things. about trying

Stan Crooke

Yeah.

these have to So push forward is also like them. programs, behind a we are coming continuing Steve, strategy we but

ready time. clinical of both for make and right it data is programs see on we to have and strategies these certainly, what want decisions to preclinical at data approve, on We based like we're will the we we but pull the the trigger

Brett Monia

some a opportunity the ahead real a for of felt the there converted programs parent was the of develop but compound lot parent we’ve Steve, we’ve like, bit And sources number to where pipe, and the was and a others we directly to compound versus continued like. for

pipeline very decision the try we and for best make to in individual each the what situation drug is reflects we drugs. optimize, of each a can So, do the

Operator

comes The from Fargo next Securities. with Jim Wells question Birchenough

Unidentified Analyst

This for Jim. is [indiscernible]

months by the So first need FDA is the the a question. not there may ahead PDUFA the three new all chance perhaps allowed Is approved i.e., extension. TEGSEDI that of date,

Stan Crooke

certainly predict am PDUFA on that specifically. is I focus think the I the date. what do not date to will you the FDA going should

earlier we hope course, approval. Of an for

Unidentified Analyst

feasible the the currently, And hypothesis? with think have future the What's then be? driven Biogen treated be patients patients would on growth adult and the they How pediatric by driving patients. proportion SPINRAZA thoughts could that growth that adult future on that launch, the hope infant highlighted to they of your that

Stan Crooke

SPINRAZA. well. also by obviously the We more had become who Biogen And driven growth are to be will patients candidates more said, patients the drug, with think growth SPINRAZA addition additional in with which in has the SMA, extent as to come more some outside treated those of markets born growth treated AveXis And and new in and adult being from SPINRAZA patients, now US, we’re with the going seeing as for patients is been as US.

combination of our in So it’s all those a view. things

Unidentified Analyst

Last have to neurology do collaboration, new the I could because complete with happen of a in happen? before collaboration collaboration, activity diseases so collaborations of you question on original second ongoing, Do to from might and designation you programs the or sense target it parallel? we in see first to the begin have Biogen, have, is five collaboration of collaboration about terms from new the probably, the think moving you when the that

Stan Crooke

the on quickly Really last part.

done. different should we and and of and get this contract, the You of – has been think the economics the exactly different are transaction of that have this research organically. we entirely other seamlessly elements working basics are same the the while We assuming working other teams

-- us moving that think the and sort simply and extends event partnership thinking expands that that it has relationship is for be more and this partnership, wouldn't much productive. already a about Biogen been I attractive the so next So from transaction of for you shouldn’t to more the much current

Brett Monia

pipeline of just the I’ll to just drugs. course, add development drugs right Biogen clinical continue it’s new only with our prior Yeah. collaborations we from with Stan. to It’s very and with rich just continue collaboration, emerging going going that exactly and a of towards that's have to new

coming many, We to for expect be partnership from come. this news Biogen years with out many

Stan Crooke

they're and clinic apparent become the to in will targets the new Of drugs be the we’re working milestones collaboration move financially be those in to and time, that -- of from the of it new will collaboration there but the those bit drugs fruits from from way begin recruit targets that is you from whether the think milestones will takes or to collaboration new old then should about collaboration. as drugs a begin from old getting advanced on, course, the the that as that

Operator

Next question comes Wang Barclays. from with Gena

Gena Wang

Ionis last this the one very and lower to Maybe royalty this perspective, SPINRAZA looks the perspective try but regarding quarter. similar, and seems think quarter SPINRAZA understand, revenue just royalty quarter, first Biogen like for revenue I the a from from

get about we think to it sense, I wanted this XX.X%. Just calculate, is if quarter, a

last the quarter, about it was For XX.X%.

some you that. wondering color So give if can on us

Beth Hougen

Beth. It’s

start they royalties, they royalties beginning are year. at SPINRAZA the and tiered on tier each the lower over the of at So

see So increasing -- the and those quickly, is rate you'll tiers those because see what each you'll quarter through royalty effective levels. we to go maximum up

you'll but up, while -- the sales tiers those bit, over levels is are tremendous those down the see royalty a so to And the over growth course of work we little as the continue through year.

Gena Wang

instead quarter, So, so actually yeah, of royalty tier, higher be is quarter rate? rate lower increasing see is usually, be that I royalty would mean, would than because actually last lower, wouldn't this you it like

Beth Hougen

tiers this and each and we’ve to at are publicly, XX% the year. over tiers, XX% start The XX% up they the said Yes.

beginning as those through rate year. are year, of of that the will the over XX% we the increase the then the So tires, And course first go at rate. the royalties at

So you tier don't top the year. at year stay after year after

tier work through by highest to and the at You start the year. over you tiers lower tier those through the yourself

Gena Wang

see. I

the right? lower in expect should the end of the we terms in tier, quarter, first range royalty every of So

Beth Hougen

In terms grow, growing. sales but be of your of royalty the percent, course your absolute dollars as will

Gena Wang

think I differently. And is, ask been another slightly asked it has question quick earlier, to wanted but

counts. any especially quarter the the of but so delay for on year month know I landscape, delay? profitability this quarter opportunity it terms on every later this what also opportunity, this is competitive In actually it and, is just in terms of the commercial commercial And wondering delay, impact TEGSEDI’s impact is [indiscernible] TEGSEDI, market your increasingly one thought one with

Beth Hougen

our perspective, So we're still which consecutive our reiterated just for levels. to for I the our to third I still profitable And original original is -- guidance, expense be year. from sticking guidance

our to with we’ve And relation don’t access standard also mention program. the a we So this is change in to TEGSEDI, associated that I one think things of extension. guidance got in key see our

We program opened to in come rapidly to actually So we’re the there's program. US. patients request in open, the standard enrolling because access the is additional sites

on physician, there treatment where today. from the a that in access can start patients is perspective, patient So TEGSEDI need

Stan Crooke

Thank you.

addition to that, disappointment. in think delay is I any a well,

boil this readiness the readiness and a in having to the hard in been to to launch think ready, case, it drug working returned time its know this down muffle History. I profile get more will and will by as relative and you little late time to very extra we've the launch up give just

Gena Wang

can have and I so quick being ATTR, the you ongoing very far? you know share -- up, wondering both just of with expanded Just follow a the if FCS us for that, patients both number programs enrolled regarding

Beth Hougen

Yeah.

We that. disclosed haven’t

Stan Crooke

groups demand open that can that, and we’re And KOLs, is also tell you we so of and advocacy we patient the What on. continuing pleased because we're from the support sites. high, with have is new to

excited we So opportunity opportunity. -- and very a attractive think TEGSEDI we're in the really the opportunity commercial about is

will take more hour. the We getting one to close because question, it is

Operator

The come last will from question Laura Cowen. Christianson with

Laura Christianson

TTRx, anticipate Just quickly you being able just initiating development to a move rate into quick the one curious the limiting on Thanks. I’m for X how to phase X. timeline what’s and phase

Stan Crooke

through simply study later the are the And move call opportunity to No expect questions, about initial to studies year. where thank think excited I We're towards rate are. for participation your them. and the I again, quickly studies. the everyone move this close. very Once for the very prepared key to we for to rapidly then the steps to And IND want limiting. with These start we’ll and and documents and we end that, the we address expect get

being for both of have everybody. the successful. stage Thanks of more I sets WAYLIVRA and success rest approval this progresses. substantial We panel forward look the to these to the of and even the year We think, that week achieved, as year next drugs meeting

Operator

now for is you very today's attending conference much concluded. presentation. Thank The

disconnect. You may now

Ionis Pharmaceuticals (IONS) 4 May 182018 Q1 Earnings call transcript