IONS - Earnings call transcript Q1 2024 | Ionis Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to Ionis's First Quarter 2024 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, Senior Vice President of Investor Relations, to lead off the call. Please begin.

Wade Walke

Thank you, Keith.

we encourage Ionis go press GAAP website the financial I section the release to begin, today, related the discussing of to tables non-GAAP we financials. everyone reconciliation to to Investors and including Before the be of will view the We financial represent results the business of our believe how we non-GAAP and manage better economics our business. posted accompany today's that on call. website our slides We've also Beth Product Hougen, Chief Monia, Financial With me Strategy Officer; Jenne, Officer; Chief are and this Executive Global Chief Kyle Officer. Brett morning Richard Chief President Officer; Officer; Swayze, of call. Commercial us Eric Research, Birchall, the Development of join Officer, Jonathan Geary, Clinical for Development our portion Executive the will also Eugene Vice Schneider, and Q&A Chief Chief our statement. forward-looking would language to . I your draw contains like to attention which Slide X,

we actual statements certain During materially. may turn our for I'll statements will based on beliefs. forward-looking SEC that, our our expectations to I additional are to the factors you subject consult be filings detail. over and are results making current With uncertainties, call the that and risk These to Brett. contained differ in encourage and this risks call,

Brett Monia

Thanks, Wade. us a XXXX we serious with joining start thanks Good and to to as on off continue futures everybody, for Ionis execute our to people for great to is vision today. better morning, bring diseases.

And with olezarsen polyneuropathy and medicines we With to steady the diseases. medicines next on AstraZeneca. recent new medicines discover, the track independently partner, the donidalorsen. is now positioned launches believe on science with to . Renewal our reaching ATTR technology people U.S. in is are unlock medicine, a with discovered of more By to deliver need, co-commercialization launch planned relentless transformational value. for to develop our Ionis well of launch in Ionis even level living patients stream Ionis first and United, transformational passion groundbreaking delivering of serious we're our cusp WAINUA, new people with beginning and co-branded a of

patients in Canada we're team's days later we and submissions year Europe soon more with of to choice monthly simple this to WAINUA of efficacy with is While freedom a And it's strong bring WAINUA self-administration, around based even completed, approval the become together the the on launch, regulatory the in collective we the know patients. expected already at-home profile, for we early to believe expect additional pleased decisions globe. progress. the poised ATTR and therapy

early cardiomyopathy our cardio for with We of transform as XXXX. ATTR larger the the advance continue data for to indication WAINUA as study potential

conducted trial As in the robust track this study cardio data deliver the ever most population patient transform and to landmark largest the set. is comprehensive on

of decisions the commercialization will believe leadership we generate are AstraZeneca's And in data globe. and well physicians this positioned the the make treatments, dynamic in to amyloidosis, coupled with landscape. and cardiovascular in bring We our leadership in U.S. we the informed around global WAINUA payers patients expect enable with treatment TTR treatment novel to we

development FCS, planned with treatments million for than we for patients olezarsen the one positive with is represents more meaningful the pipeline severe in olezarsen FCS. olezarsen most disease these opportunities next By olezarsen in alone. very the Last today. in III results approved in larger no X the Phase for U.S. population U.S. of addressing month, our in and indications, in presented also published launches Our patient much SHTG a estimated rare X

FCS, In in the Phase with patients substantial III showed BALANCE reductions. triglyceride study olezarsen

it truly trial of reduce reductions and substantial the and payers, incidence clinically for of patients, tolerability is pancreatitis well Importantly, demonstrated olezarsen reducing demonstrate is groundbreaking the acute can profile. triglycerides also meaningful as attacks acute that safety first and as a pancreatitis. in to physicians as favorable Balance

data from In study triglycerides this patients positive III Phase moderately baseline the cardiovascular in and and elevated with at high patients BALANCE of we the normal to with XX% risk achieved triglycerides. addition BRIDGE In study, levels. study, elevated published moderately presented triglyceride

Reinforcing and FCS pleased I'm our submitted report for with potential we NDA our closer olezarsen that to last independent and launch. success putting confidence first SHTG. one the for to in the olezarsen's month FCS in FDA step Ionis

Assuming priority our olezarsen and preparing in approved, FCS results approval this that track review, FCS we're confidence date approval this and year. care if to Europe the for for We're a end is for regulatory standard first increase United U.S. of the approved to potential our the olezarsen be be year. SHTG. in further also the file strong medicine later on for of both by poised the States to could

Our continue SHTG to ongoing for Phase III well. progress studies

report also data in complete mid in enrollment have year. very X our to soon. next III X SHCG studies Phase of in enrollment to ESSENCE. completed CORE the we that With puts study pleased I'm for CORE SHTG, now II remaining track Phase us on III expected This

Phase OASIS X significant every next reported in statistically weeks treated top patients FCS potentially patients for HAE our donidalorsen, commercial olezarsen expected from rate reduction first with study. endpoint behind and positive the treatment Early the primary we closely is are quarter, of data angioedema. with prophylactic weeks. X HAE the in Following and in line every first-in-class hereditary treated III Donidalorsen met the launch attacks a

profile. evaluating over donidalorsen and endpoints cohort separate from to as studies session study plus secondary we that oral a The Clinical study refer EakiCongress for includes III study European safety in cohort the Oasis looking a and to SWITCH Immunology III the of other SWITCH We're and Congress, Academy study. is and patients a from favorable first-of-its-kind end presenting medications. the Phase important the month. and patients tolerability OASIS at the other rolling prophylactic open-label to have late-breaker transitioned HAE this study Allergy met the forward Phase of Oasis the also who data an from at had

clinical the conjunction data webcast with in presentation [ We ]. plan at to Jake hold a

regulatory which will FDA, the data finalize X-week submission include working positive and to With both dosing these options. in hand, to X-week our we're

Additionally, III treatment long-term is results safety Phase the marketing Based for seen study, partner, extension donidalorsen, the on open-label efficacy Phase preparing Otsuka, safety we evolve our European could our HAE the and to If data prophylactic favorable and in believe ongoing approval commercial Europe. file paradigm. approved, II in

our near-term opportunities, including we in Beyond pipeline, our have progress additional areas made of franchise. important leading rich neurology advancing our

study, development our pipeline today wholly with this for targeting end pipeline neurology X medicines added on ION-XXX, leading year. leukodystrophy. the the in remain for to these of Orbit first X-linked We initiation study inpatient of by track one PLPX rare have just Our neurology includes neurology and we XX a owned clinical medicines medicines recently to a PMD, recessive of the

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living production IONXXX program support stage for including IONXXX time key program with we results areas, These hepatic for the of Study These tolerated. that people also outside license secondary This advancing millions to demonstrating endpoints, safe to value well next fat intend and and focus this histological data first endpoints, clinical maximize NASH DGATX provided demonstrating improve today. is improving its was fibrosis. that while the targeting to reduce can in NASH to evidence minutes primary of our of the key development.

and steady closer we're over Who value our introduce lead achieving I'd come to to important running to to stakeholders. transformational Jenne. medicines and Ionis of commercial a extensive familiarity for and commercial investments and Kyle organizations our next-level global patients the the years to With generating this growth. with rejoined recently and move year of next few and of added our years this and all spearhead medical like product has making seamlessly during Ionis Kyle next bringing our our the leadership programs, goal new cadence biopharma experience phase With to already hit accomplishments for very that, time. his Our strategy value ground

provides quarter a financials, Kyle and brief that our on olezarsen ] and launch the After first the donidalorsen our activities our overview [ including way of NEO for results. go-to-market status update

over Kyle. up questions. And then taking with your that, to before wrap I'll And things

Unknown Executive

I'm medicines Brett. patients need. be Thank with highly team experience ready to who at are a back with to Ionis, deep to in commercial important our you, excited working accomplished bring

market commercial roles approached last WAINUA focusing for as access, team right in The the operations place of medical executing marketing, in preparing donidalorsen. on services, years, on patient the the commercial olezarsen right AstraZeneca with have have Over and upcoming launches and purposeful a at commercial manner hiring and include co-commercialization the the build sales. independent and thoughtful capabilities and our we well expertise time. a as X We established now of affairs we've

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As and and profile the significantly Brett poised the ATTR XX,XXX polyneuropathy and and of patients its Ionis' underserved. to efficacy ability on treatment in of safety noted, than patients with on worldwide ATTR treatment an fewer WAINUA remain choice self-administer. for XX% is to based patients hereditary an largely and estimated medicine polyneuropathy co-branded underdiagnosed represents With hereditary first be approved

high predefined is to positioned a Overall, need launch we to on address. indicators. unmet a As plan performance that launch result, uniquely based AstraZeneca remains are the tracking and

other with whom is and class are some whom good and seeing this some switched the are from the of teams are phase We of medicine AstraZeneca, our and Under organizations. the brands. The well early together of partnership benefiting cross-functional co-commercialization from patients, expertise of working have both uptake Ionis that AstraZeneca among new of to launch

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for Our launch FCS. preparations are underway well

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Our through disease nearly FCS awareness around medical out connecting physicians X with affairs for team has identifying and years. urgency disease diagnosing improving and been education.

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the journey. every initiate this we champion their patients therapy national up term. in patient education remain reimbursement that The will for step helps leading provide plan support and for caregiver focus to launch. approved. rare building understand through medication product this includes to seamless team team our treatment access patients appropriate FCS a disease helping sized customer provide and to director a team customer-facing appropriately once be reimbursement population and and to be With The experience place, This on are and of patients informed our hire disease long on disease, sales account programs. goal information world-class We treatment about support and of to the is and their team their access help

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and Within the treatment accepted is well prophylactic physicians. U.S., by patients

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we which X very presenting from physicians addition, data reasons. the patients In study, for believe first-of-its-kind for will be be switch important and we informative will

and physicians or in to how approved. where treatment. preference answering to donidalorsen. our question, you prefer an understanding we switching assist we should positive X- to with Together if second, survey include III patients will be With long-term Phase I'm launches. will upcoming for Phase that believe data donidalorsen pleased [ data self-administration, your in important -- ] or for manage could these these when expect we effectively new a First, prior II And X-week patient be we prophylactic donidalorsen Do or treatment OLE every HFE say right important are data, preparing the

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to Beth. over Now

Elizabeth L. Hougen

agree couldn't Ionis. you, exciting really Kyle, Thank This at is I an and more. time

we ever cadence of our launched we steady to medicines to are own bringing than As the first a co-commercialized And our closer medicine. market.

in our financial earned Our $XXX goal. first We quarter results important products reflect SPINRAZA Half million was progress toward achieving of substantial primarily of of revenue royalties. this first comprised commercial quarter. the our revenues from

launch also Our included WAINUA. from the of of commercial addition U.S. royalties the revenue

to pipeline from other our value under The half continued reflecting our programs R&D of our that was collaborations, the and technology generate. revenue

Our year-over-year non-GAAP our increased . continue we invest operating expenses opportunities. modestly growth to resources as toward capital

made and expenses as of AstraZeneca as SG&A our U.S. donidalorsen launch and we the the olezarsen and in launches. expected, As ahead increased investments initiated WAINUA we the of

Our the late-stage of program our also R&D activities. increased timing to due expenses

to our And investments stabilize late-stage expenses the studies we $X.X ended. several billion. year cash expect We as quarter this first of R&D and ended with have

first quarter us meet financial Our to track keep guidance. our on results XXXX

Looking ahead, to we R&D funding anticipated license partners. modestly milestones, first in quarters fees our revenues to higher from subsequent on the project quarter regulatory and be based compared

million is million in $XX will we example, is [ and it is in For earn the approved if we Calcada EU EU. the approved $XX ] knew

royalty underway, [ ] as the Additionally, launch approval. the assuming we up Calcite EU WAINUA expect grow launch to our revenue gets ramps as and

mid- impact in onetime to of percent XXXX, to year XXXX We year. costs expenses range single-digit high the continue to full to excluding increase last the compared operating project our

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investments. billion unlock substantial that We in we expect And before next cash today, expect in with end $X.X us year and we invest value. the level as opportunities to will the

as delivered we solid you a So can quarter, first see,

to financial with rich programs, growing of of compelling includes you like our increased With that path vision clear to number I'd cash to realize the opportunities make fully a have a significant owned a powered revenue continue drive investments we positive us, a path now pipeline growth to by value. clear over few And years. flow, the we sustained to will before provide wholly next

just investing medicine. in launch for built experience heard for making successful from We we on are efficiently are We investments leverage important prepare and as a significant as that you areas: X important and can key To for olezarsen upcoming First, gaining United WAINUA of in key we the in States. the are execute commercial launches functions donidalorsen. launch this our Kyle, we

years, these in launches our couple will of combined As capabilities increase we line and represent. scale that multibillion-dollar our the medicines for prepare the next over these activities potential revenue we go-to-market with

continue we invest Second, our medicines. will late-stage advancing in to

level relatively first this which III of And that the full today be resources Phase stable Because, at our able recently and third to several owned all, of expect earlier-stage near will of reallocate our few in over are we investment wholly years. is or studies have area enrollment, all area are our large We next our of heaviest from now these the period investment, program. R&D concluded programs to investments. of studies

deliver programs advancing Our us medicines our up wave these next patients. to investments to of set in

medicines technologies continue with to innovative ensure And profiles. finally, to competitive we we cutting-edge are investing deliver in

-- this patients medicines positions need, medicines and have can And which growth. a can turn slide flow positive With opportunities. dollar sustained revenues depicts an have outsized revenue our these many these we see, as in multibillion that earn we power investments, achieve to you deliver to us in opportunity on to cash

donidalorsen FCS launches owned severe we blockbuster planned wholly-owned patients wholly next have HAE, upcoming the olezarsen our olezarsen, and for of potential. high several for medicines for that our Our for could reach medicine This in launch of development years. treatment several includes has additional the triglycerides, that over Beyond

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is the royalty multiple investigational that continued milestones developing cardiovascular substantial to We with pelacarsen, assuming future Lp(a)-driven to and a include that address advancement. sales have provide first-in-class partnered also patients are poised Novartis programs revenue disease. These millions of medicine

with regulatory for are next investigational Pure for infection. the ATTR The our a GSK and on become we and potential to planned substantial has pelacarsen preferred WAINUA in and And millions ATTR polyneuropathy is of developing submission that III large the the in Data for well. landmark year. program the study with and the that believe progressing treat HBV treatment of patients course, medicine WAINUA have track Berson growing Phase launch We CARDIO-TTRansform market. a to

All mid-XX% billion of up sustainable of of And top addition could payments, all to range. this is revenues. $X in the on and these and substantial current to programs generate up in royalties combined to our milestone that's

can see, and plan advancing the opportunities. pipeline over our make technology. in you next commercial in as So strategic in few our our investments years, we to rich

as these cash patients our years growth to need. We all us come. and stakeholders, Ionis power to revenue level X reach expect for medicines value flow Positioning next in more deliver investments to strong positive

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

Brett Monia

momentum Ionis we as Indeed, on is Beth. strategic on priorities. our execute positioned you, all to building well our positive Thank continue

] and strategic established a of We that our as advancing and set one today old of directly industry. in and medicines approved focused a Which develop clear with pipeline drugs we have delivering include and on vision pipelines and [ the by discover Ionis lipid are a arrived we being building several mid- where therapeutics conceive late-stage objectives. the leader richest patients. in and We've to clear

into for build our Our owned wholly these the key year pipeline clinical are programs II year and discovery. expect further delivering. the year. important data and the oligonucleotide by in our leadership to opening III on progressing this our medicines drug Phase position optimizing on prioritized technology, readouts partner development We in track is for achieved past advance near and momentum with several therapeutics key our extending positive this poised are and and over our year expanding strong we existing marketing multiple building therapeutic up readouts next and of readouts pipeline, capabilities . We've term. parallel, Phase beyond. to planned approvals areas and new We're diversifying In data areas

to call take transformational up objectives. cadence and we recent of And forward continue our that, our progress looking All open the questions. I'll for as medicines, achievements now on We're questions steady of for this years to sets build to sharing and a with to new all now. medicines us strategic accomplish come. we Steve, patients our can bringing up

Operator

[Operator from Instructions] Securities. Guggenheim And Chattopadhyay the comes Debjit with first question

Debjit Chattopadhyay

HAE Firstly, for and the who and the olezarsen, many how switch, on expanded therapy access, adoption? currently you the low-hanging Between early donidalorsen do patients and commercial could OLE be FCS have fruits on

Brett Monia

in So patients open-label so and the over rolled study the into in for was open Eugene label olezarsen, III approximately that the continuing or they're in now. the extension which trial virtually extension Phase all patients, of XX

-- measure So that's good there. I that's a a pretty mean

the access For just FCS, getting and program olezarsen that's started. expanded for

has stayed the got patients story and which think, the last update the has the for The the they've of completed early donidalorsen. week completed over patients far, later trial. Donidalorsen, days. provide extension. . Similar just in on. the Elected started, been reception So But the remained were XX% so trial, excess in positive. majority donidalorsen. they've extension, that We'll all open-label of to open-label vast very and of into done was it's well an in the roll EAP I virtually

Debjit Chattopadhyay

dynamics monitoring D versus Venue respect to should ]. ambutra launch [ Part any with there Are one. we quick one And it. Got be

Unknown Executive

the about for be to talk question. I'd Yes, happy that. Thanks

largely the of and experience say majority is payers. The the the covering well. There's so payers in use launch understood going So as polyneuropathy for are expected. space with the from the First, medication really is I'll just criteria already

things, that continuing Part efficiently. to of we're the side very On navigate D

doing support the where patients and working fantastic for way just they're things, really you market with to also effectively on leading offices. and a is our patient directly of process services and commercial on is appropriate access supporting And the that job. program them also side provide through the regular reimbursement would them and AstraZeneca the patients educate expect the supporting process out-of-pocket

think I landscape overall, the the So we're very payer experience The positive. is execute is very launch to continuing effectively. And patient positive.

Operator

the And next Nachman comes James. Gary from Raymond with question

Gary Nachman

for you're for FCS. a for of will there exact So bit I preparations know need built more know launch with could leveraged. infrastructure you be synergies a know of commercial some overall team about that and olezarsen for assuming review FCS. big I olezarsen time a the confirmed that already line then priority in when talk review, WAINUA, How but and the will be you the -- the how describe

Brett Monia

in hear -- of XX that. so jump it's Gary. for one -- same in into addition we in take when as expect the about we'll so we that expect we'll be receive and XX-day the Jonathan Thanks, talk submitted all come after from would commercialization. whether -- launch Once or days sorry, could expecting that we we And time Kyle, well talk -- to plans that thirdly, to -- FDA date. I'll or the that. NDA we priority would it a Maybe And the acceptance the and the have com, information bit could outcome, are hear hear they And but at and March. to that the we an FDA, bit April, field from line and the for a little to for we the ad submitted submitted in prep first we hear announce April and to our don't uptake. whether about have a FCS? on from FDA we not about little an in review expect PDUFA at Kyle the

Unknown Executive

robust thinking and and Jonathan about Yes, with turn force the I'll within the all, is, the very it go-to-market the that timing strategy we've of the synergies we very talk the building in internal experience so I'll and way what's capabilities, field been the in that But in some strong of we're I leadership talk marketing the over measured of WAINUA, build that being currently first sizing the place to around that calculated the with capabilities got and about which about we're there. efficient capacity. terms smart the I synergies, and very and think build. . we've think infrastructure then marketing of organization to But important are

operations group stood Our commercial up. has

So and in place. those data are analytics of types and infrastructures CRM things like the and

had fantastic standpoint strong trade been doing Our work that from at market that been access to interacting the anticipated with job market importantly, team is They've team presence And a the medical finally, ago. And is doing distribution. Congresses. There's look of of in and affairs and a understanding group, we insights established that KOLs. data a team and market little dissemination. and really the over lot payer is way years X channel a and has

knowledge of lot information well. a as collected the that We've back brought externally organization and into

the mean, and of with the execution. the ability now. we're at and is and infrastructure and is timing nicely. capacity what with this the I I think overall, you're been really of very all together the I've right hearing And very So pleased impressed coming where

So specific maybe FCS. Jonathan, to

Jonathan Birchall

Yes.

relatively the the particularly around patient to, between that there will I'm lot aware, small HCPs. is by FCS are a rare launch of launch. background, sure managed as are number think to very relevantly capabilities services. of Werner you're such to small as Kyle of synergies the we've -- FCS I areas launch, transfer relatively number There built as disease. a the bit who a alluded A a of support patients.

our imagine, build So group the meet as of footprint the will and those group. needs indeed you patients. those or customer commercial scale customer That of can the

population. it will appropriately that service disease rare built nature, be given So the to

Gary Nachman

naive And Great. big you'll competitive on of right. from in there patients from thinking to a latest the are relative And you drug how positioning getting on effort an standpoint a switches NDA. doni, in then be the standpoint the about terms commercial of just you'll All Kyle, described versus market? And thinking need data in how olezarsen? that the including

Unknown Executive

Yes.

in in patients bit strongly profile donidalorsen paradigm. believe, start right? also just reduced already to really data the note currently to And do prophylactic critical, patient We've space little about and make that really, on treated do the EU. show of of the switch safe couple the terms I important out terms in really doing patients that going It that only being to into and X- one, got the roll and endpoint, X-week this versus of to this overall, being me sure rates lot the the to data. transition. U.S. between effectively a really of outlined. how terms of this and identified. data is that's therapy. data is very is innovative that we it's things. be the payers differentiation the switch this And end a talking HAE go XX,XXX-plus of we month preference or the by not and to what the Let the of to A and at competitive making patients the able at and prevalence a think, prescribers tolerable we're how to They're in differentiated This based Number the I around to market, We've support anticipate obviously, going efficient a terms to and you . The in but of market. is switch evolve of to treatment is and this market, also a primary think, different of about very that sets switch these We looking cost got very, that, the relevant But be dosing we're as in we're we'll be of attacks. durability going able are way do competition.

be a the to So this the poised setting overall, we're I do marketplace in the going up, space. are and way really things job great and think between data to here

to expect infrastructure. terms to competitive field outlined XX terms In of process later terms year, able size be putting I XXX that time place be commercial regulatory be probably that we'll will this of lines pending the ballpark in the in to the in team and start of sizing And later.

Brett Monia

how of month. we're data ] Phase XX ever conducted II the baseline X-week long one And for sharing include Gary, thanks, data Switch but switching is We NDA, The HAE the submit extension for to every approval expecting rates, database support on data for a be extension necessary is how very And switch that. also an are to prefer safety forward plan looking in data we're from to instructions in regarding able the to compared forward ACE on patients compliance, switch patients open Phase III that, and study well data approval. every how treatment prospective donidalorsen another. will dosing, much choose not that only data previous first And existing protected the preference stay as on both as well based whether they absolutely include over with related And data they prophylactic donidalorsen. why open-label May them the the X-week this attacks sharing really looking at Kyle. switch their prophylactic treatment. with also from to along [ to on physicians the over dosing open-label to in-depth is -- to to we'll Yes. We're from on the HAE, patients patients

States, it's So market switch market. United first a the the it's also study. the kind, which a very test important of for litmus a It's its is and in

Operator

the And Jessica with JPMorgan. Fye next comes question from

Jessica Fye

talk your -- flow, see the to then IONXXX, that second just what And those horizon in set a you the for path results? that that? company little to asset can timing for for a cash the On ataxin-X what ] over able to time [ being you can for ALS stage me quick bit you about sustained And achieve readout? X just remind watch for X positive

Elizabeth L. Hougen

Sure, Jess.

take for drive drugs bring through ramping. it's investment to really and ahead milestones. with royalties market, be want the today, to positive of build then you that opportunity. And ramp if the revenue our are grow, substantial we with Obviously, that ability the come sales to multibillion-dollar particularly pipeline us the market on us And that strength rich our we've launches those launches think the that of of of But it's up a product pipeline, regulatory revenues Ionis, continued to partnered and ability obviously, those of with the So to then, cash as launch multiple the those our years development, to flow. medicines, late-stage reach got substantial those and revenue will wholly about going substantial owned opportunity and opportunities really think revenue as base have, sources the we few medicines. process sustained you generate programs for and a about those through to

that versus offset that And about the that our have very are in means first us. us to company and lot that a of we products unique space the very we is fairly that we're goal these Ionis, continue. generates for for and remember get to of what's is investments And to actually delta finally cover that helps far launching I very will achievable others away. who for base think anticipate cash we substantial not modest revenue companies today. Unlike is important market, a when already our medicines, a have their that We which

gives about think that So on a hopefully, perspective to how it. you

Brett Monia

we're midyear ALS results your II Looking on please around part ataxin forward for. nongenetic other year. X Eugene, Phase other to for the comment Jess, in the having what from of looking question, study data this

Eugene Schneider

first of safety And doses increasing It's with we're said. Yes, of thanks experience. oligo study, safety obviously Brett in primarily, It's for dose-ranging ALS our as the inpatient a sporadic patients this assessing question, is Jess. study. a

study is levels described, of other intrathecal safety really, at obviously, nor is that the as Biogen We're filament. to have various looking goal well such those demonstrate been ALS medicine. NSS or dose and So of key surrogates progression, of

are there course, ALS those of used be any whether endpoints assess key progression activity some on measures. of that exploratory to is So there will

Brett Monia

chain more on rating [indiscernible] of that. within measures light Kasai The sub there. will and outcome, ALS scale There on other the be domains also Based functional will measure obviously be exploratory a key

Operator

Company. comes & the from Joseph Needham And Stringer with next question

Joseph Stringer

on SWITCH ] Doron study. follow-up the a in Donnie Just [

other study need can point what you SWITCH to maybe that put You see some on most data from uptake little bit the But and commercial mentioned resonate a a would from specifically a will data. finer patient you survey perspective?

Brett Monia

totality the with Well, I'll are treatment treatments current data, that today. to available all, data going the start -- will jump we really that and It's can in. share are Joey. We're market current on of first the of Kyle

So other looking What -- that it continue rate be X then or just we're previously rates be very that We're -- patients [ won't important. Ledeo attack that were have or on have and That's switching. on treatments. attack prophylactic treatment HAE baseline data going ] donidalorsen at the patients evolves on -- to launch. the been been their study to commercial as into be treatment. how coming the HAE commercial for to the TAKERO for

the looking survey that going -- forward to be a Second, they will In as which patients to in trial also we're I mentioned, study the sharing And it's that to going well. prefer. ask why. and as we're

this different algorithms, Kyle, prophylactic for and will which on treatments. very donidalorsen. really importantly, important, and on to the off instructions you and the will, to WAINUA on donidalorsen much need right, launch for time be And time how donidalorsen. to Based takes able before to have data it's we'll the be they data, how prophylactic move existing different on have treatments are much But then on for it the thirdly previous if treatments and -- the

Unknown Executive

Yes.

I already either moving unsatisfied are help hasn't it's patients between the the sometimes controlled differentiation moving they're that that market. adequately And right? because they're This delivery that mean think therapies I highly think differentiated. first, been way or on. works as it is is I This is having it's to us. of for an going because before. or right the that done now. not see treatment we breakthroughs mechanism they're And they're

to And for patients, very for to and to number the not that they're to formal do inform at data, paying one. so and about the And be way, the know payers feedback help physicians want the we're safely this number payer a it's two, from about helpful us going sure on thinking in medications make . to effectively. same multiple how time. side, when finally,

and And have us for well these to therapies to on cover about this on will overlapping and without as talk patients. them messaging for multiple payer to they're help to donidalorsen to how donidalorsen stay the or effectively get with so that having having pay

Operator

question next America. the Bank with Jason of from And comes Gerberry

Jason Gerberry

me. for Two

in that a silencer. your what if the potential event And my payers won't a like? events second you around will cover cardiomyopathy Phase that tafamidis TTR on can other Alnylam is then and pretty on for seen a question basis decade. generic combination how of the versus rate questions seems are this stabilizer pancreatitis adamant therapy. acute SHTG best go or And just there's trials and comment may Just events and accruing look III you've trials blended estimate even

covered just if that? lot and curious will know there's in I that but love work that I'm around get combo. you've combo that, So you clinical it just can a done there's like address for what an bar attainable

Brett Monia

All right.

Kyle the monotherapy to we combination dynamic and comment TTR over handing when the SHTG. Jonathan about Before cardiomyopathy market, on

the of AP our remarkable guess, only we a in reminder, shed group the than events the in what they FCS acute FCS in are we and rate of therefore, expect reduction events lower With mean fact are FCS. SHTG that very that manner -- we FCS than in were expect conducting that not are -- in SHTG. is of lot the true of know just line they're in range SHTG, said, with SHTG what But a understood SCS do light the really or FCS point. don't the compared in levels AP people. said, study these study. a based to be triglyceride trials the we bit balance SHTG. want olezarsen. placebo events rates pretty as expect Is the the on substantial, in and are the the of in was many FCS much less, saw that in point But we're group. accumulating into that are study to would going reduction reductions . Virtually we the I events, for the Jason, It's to to all were have a in event and in Not based Certainly, light to are on lot placebo and fewer I well on in really robust AP the the seeing a we going that important add trial are AP that that TG AP both would mean pancreatitis for will you was lower number more be study anticipated that So our a in reductions blinded emphasize thousands AP events in patients events study. occurred that SHTG. a

SHTG we we anything in olezarsen better. be patients, to like replicate because FCS. from group of in FCS, what a If in we even versus expect saw placebo magnitude the the standpoint reduce majority would can triglycerides the expected those to

more a us AP at So study the outcome. positive gave FCS balance certainly in confidence

changing want how A seeing you cardiomyopathy, play combination we're in in key out? the reminder, TTR triglyceride comment secondary. is In the on primary the dynamics study. AP endpoint monotherapy is Jonathan, the a to events study SHTG reductions,

Unknown Executive

Yes.

largest payers. given that I think the yet long with that and that Obviously, covered around question, market be of cardio confident from from then think fairly that so don't by X the Gilead's really to where we're there point that, we specific of profile shown that that data understand covered combination expensive and question the has are me other payers. therapy payers combinations with we're to where will transform difficult guess research willingness combo. is don't to combination. But areas, to cover is dependent. completed area, have a very I the I for to it's will that as data therefore, the said, any tell very willingness data what's the there answer oncology plenty this And my benefit specifically the of the first whether were the drugs ultimately in can there is we've the we to you are if effectively it be work demonstrate I plenty usages, able the combination. that the still by figures, C, cover are caveat That study hep What regards in -- treatments

Brett Monia

based tafamidis demonstrate To combination well added our we're the our percentage positioned. design benefit versus size trial in have the of in of of on an And monotherapy. and the we trial, on very patients

Operator

And Fargo. question from Zhu comes Yanan the next with Wells

Yanan Zhu

any Great. Maybe will update as their curious, any competitor you points a endpoint primary color And that data to there? follow rate, on about cardiomyopathy, the -- also up on any blinded and readout ATTR focused data, what event just learning? And on the are analysis? be thinking key the

Brett Monia

you, thank Yanan. So

So trial transform They're the cardio blinded event rates are in the tracking track. well. on

hospitalizations For well what very very to We're it we mortality expected. that, endpoint timing. the at on And say referring quickly -- those of carefully. both our looking hospitalizations. I'm or And nature primary that and plans I as changes to there's as when no --

on weeks in much If very year. this said blinded rate say for study. out. from much readout to presentation in decision really at next about trial to our our we treatment that be than of the first more looking study The Phase it go much focused as we're can't still midyear almost AstraZeneca, it as timing to reading our that this to with III influence on something point. this much early out forward drive the And XXXX. looking very were it the go to year we the forward and completion, We're event partner, from the of could I track. silencer we its study We're XXX very REIT As a TTR our When cardiomyopathy it, later would learned

Yanan Zhu

the the data? readout. I EEG we syndrome of may follow-up the like value? [ versus do CGI Angelman relative when biomarker on about be learn and a endpoints the ] we think If X daily Great. ask clinical What how might And importance

Brett Monia

settled biomarker haven't Angelman report and don't endpoints, exactly program. X we [ Yes. ], cetera. we Eugene EEG et the the out will on -- next other the We will daily for step comment clinical when how and

As the for said program. next for the for steps next before, comment plans option an do as this step Biogen reads we've you to in has We the what also when the program. the on we program know, data the And announce were to license of option planning are out, Biogen's Yanan when

far could made is year. Angelman. as bit seat regularly That ultimately. any at decisions. of As the on attend they be program, licensed Forum, talk but is those endpoints a There in make settled either. we from in this August this not to be that we will the Angelman an a Can which have conference we And haven't form, each of course, driver's you little about we're if in year, the calls looking Biogen that

Eugene Schneider

sure. question, Yes. Thanks your for Yes, Yanan.

course, and this things. sleep both course, of of of of the and a exploring the has of aspects of includes is assessments was for shared include neurodevelopmental truly we're and study other for through course, us. as with we're really clinical Baily learning at early a different including, some that essentially scale scale -- this So we're ], very symptoms that EEG, broad you data natural a such, Angela, common behavior other like used also And covering is disorders. in another course, as that things which presentation that looking community. range Vinland a very But history as well a importantly, of mentioned outcomes, of build range of and disease, quite [ bit standard data But the

those drug trying disorder. our this So of on the possible we're have all about pretty much may complex impact as learn features that as truly to of

Operator

And Issi the next RBC. question with comes from Luca

Luca Issi

have target progress. congrats that and power, [indiscernible] what Does thinking appreciated. target. Alnylam III antibody trial off Roche on antibody what's HELIOS-B -- your is Phase Maybe the latest caromyopathy, the want AGT, that now maybe on run Adlon under then patients. on while Gene, the power. patients here? may, this even your it same not Great. is of your was it most patients. mean we to TTR Any a clearly their And X,XXX and -- amount I about of for this I AstraZeneca trial monochlorinbody cardiovasculatotrial, the at feels the Am airtime to in getting excited did. monofloral very you X,XXX if purpose is reaction power in on thoughts willing it is X,XXX that they AstraZeneca in basically, once to deploy patients overpowered and are there, one

what's so, sort And ice the finally, if And program? then next HAE. on

near assets or only I think that comment QX and on you're ] that citing these XX, to W QAW elimination of obvious Wondering includes if QXW. both on Page attacks. can you [ whether applies

Brett Monia

Comment trial on the design.

them we're -- mechanisms -- in powering for could about the synergize close not We, antibody assumptions into [indiscernible] to our their these and that the road. regularly talk to all so course, that study, depleter that, TTR area. X AstraZeneca's pretty that not of went the size So down of it's how

anything. not their trial. So of -- I involved assumptions, but the think certainly, design in don't we're powering

Unknown Executive

No.

it know, a data. certainly, you as and about confident in our assumptions on spoke trial, based -- we our we emerging on lot that are pretty Just feel

feel I about. I others can't what use what speaking that's So comfortable assumptions. speculate their in

Brett Monia

the trial right and others and our can't I the on study. comment A design. doing. have size are don't different that in cardio in transform makeup what believe trial that, more We AGT, really we no -- design to any the want We change. of demographics With mechanism. our I the believe

we're are and to as do program we've to stated continuing we're this making progress We still partner that, plans, earlier we plan to planning and our progress. that

-- in benefit attack had of HAE every reducing the month, every X-week at and a every we know dose end dosing will we reduction share attacks. we highly showed HAE both greater with as a HAE, For X-week you this significant in at rates, Yaki statistically but X-week

X-week when So near-elimination certainly every closer data I refer comes every dosing. than X-week to we to that

Operator

Goldman question comes And the with from next Richter Salveen Sachs.

Unknown Analyst

Salveen. on SP-X for This Tom is

scrutiny, to thinking the commentary about think up that do level in ceriumyothesy that? there year. the this you want be take especially potentially that Medicare -- of given another Part -- you just about follow possibility how the a of could entrance to stabilizers the previous in higher you So are on

Unknown Executive

I to in those bit that. things things around IRA with a regulations time and For right, are of patients a historically, had and out-of-pocket and the the of way little for population, Medicare paid D timing around come there's the terms mean, lines have expenses And been evolving the that be quite play. that into distinction bit,

better I with at than in we to historically, more think where anticipated only years. been now it's D much is favorable and a the we're right Medicare get coming position Part have

It So limited for are the patients, And year. either $X,XXX $X,XXX next those so time. costs then over those to ability goes so to out-of-pocket be there's expenses or down year. or this to going also spread

one not is for patient, which very think assessment it's So the I positive.

effectively we're WAINUA Medicare think very able have to be channel, that the support when thinking that we for will So D just And patients our treatment Part programs cap and on or D, we're all other system of about the Medicare that $X,XXX, those encouraged appropriately. of patients that we'll it's ultimate through go work that combination therapies that about of Part and will hit within keep $X,XXX mind, but are in it's a not respectively. that I

treatments. allow positioned and well have in channels place to access think our to access the appropriate reimbursement a we're to So good I patients to

Operator

And the from Lebowitz -- David next Instructions]. comes [Operator Citi with question

David Lebowitz

presented the IONXXX Would you that to therapy recently and get NASH ? you what you to you on the comment be as and given burgeoning as data far about that think landscape able

Brett Monia

We MASH. time. the more we This Yes, year data. couldn't earlier X David. with reported Phase pleased in have Sure, the this on patients is first been II with

also in a certainly treatment by resolution, fibrosis, shown target well pathway fibrosis patients, the on We the that turnaround mechanism. this. only based We as in in that biopsy was Of liver synthesis to fat not positive these is achieve reductions inflammation. NASH liver. reductions surprised targeting this see actually fat get saw by expected robust to a very our in is in get liver time first that, to So reduction pleasantly we to of blocks ever, a synthesis to to . were [indiscernible] as

As pipeline this opportunities. and Ionis our focus. fit program wholly our specialty neurology, we exciting really as in doesn't mentioned, maybe rare for owned well it is, within cardiovascular as

moving this stage could There's is we pretty fits early. best towards be So Phase a well. that's think III development, we're the It's what progressing and take program. those a to with And it which doing to are partner. this program partnering of we're next program. certainly conversations certainly interest,

time And up. progressing things they're one wrapping we the question well. before have maybe but for In discussions, last

Operator

with Last one Costas comes Villares BMO. from

Unknown Analyst

Can [indiscernible] of thoughts And that in you space also commercial Sapalusen remind would should around the from program this us program any the -- and the what be is given of in that status helpful? when in expect activity increasing the PV? XXXX opportunity we updates

Brett Monia

Thank X vera Phase well. to progress continues you, the polycythemia Yes, capablersen in Costa. yes, program

understand said said before think an it's that dose patients. and study. be study, what we're dose many development. and many clearly study, for ranging I regimen is efficacy red in what we're And open-label reductions Phase to that activity, II been potential normalizing seeing hematocrit cases, before, right we've Phase in evidence What we've doing III of this cell we've -- the robust in into will in we're is accounts, a advancement in doing dose this

doing on you. market that organization forward and create this potentially opportunity We States. our sharing area. the the don't And outside well continues. assessing So States of as its for as we're a It is an that to data opportunity United the all market research year. for now. looking far -- very It's United commercial in have question, to in way a answer the . The we're interesting the

update or for everybody, thanks, provide where Costas, Ionis fits end and Thanks to to on the program our this So year by the have us program. an try not on going today question, who We're the the at the for decided joining we of best call. partner. on participated

III We donidalorsen plan level momentum XX. next the to down our for recap all continue We're comprehensive throughout also data. forward the presenting looking on stakeholders. providing at value all to year, of this on of focused and Phase great overview May We're the shareholders Yaki a

day. tuned forward the to looking We webcast in, a webcast. us So joining well as the hope that everybody, and you day. then, and for have you, we're on And can that, great thank stay join on for until

Operator

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

may now You disconnect.

Ionis Pharmaceuticals (IONS) 7 May 242024 Q1 Earnings call transcript