IONS - Earnings call transcript Q4 2024 | Ionis Pharmaceuticals, Inc.

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Operator

Good morning, and welcome to Ionis' Fourth Quarter and Full Year 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 the call. Please begin.

Wade Walke

MJ. you, Thank

today, I better section begin, business. press to to business believe we be go and to how of Before financials. and the non-GAAP the encourage economics Ionis Investors We discussing will we of financial financial the our a website the reconciliation related GAAP results we non-GAAP tables everyone including represent to manage release our view of

have accompany We posted today's our slides that website also on call.

are Chief morning joining Kyle our Development and Research; this Executive Monia, and call our Product of Officer; Chief the on Schneider, Officer. us Eugene me Hougen, Executive Brett Jenne, portion Swayze, Q&A Vice Strategy our Chief With President And Beth the Financial Officer. Clinical Chief Officer; Development call of Global for Eric Chief will Richard Geary, Officer; be

your to language Slide I statement. draw which forward-looking would attention contains our to like X,

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

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

Brett Monia

everyone, Thanks, today's call. for Wade. Good and joining morning, you thank us on

independent chapter first As a our fully I new that stage of am new company year, begun we with a as a Ionis integrated begin thrilled TRYNGOLZA to has share biotechnology underway. commercial launch

risk reduces that life-threatening late time sustainably triglyceride access syndrome, FCS, first or TRYNGOLZA and the have reduces and pancreatitis. With substantially acute chylomicronemia FDA treatment a for of December, the levels adults familial in of with approval to the substantially

weeks in been flawlessly, the team TRYNGOLZA following a experienced of December the couple highly which enabled executing to approval. launch XX patients of within hands be has Our the

angioedema, with later a unmet and preferred the We're to hereditary population of in programs potential. this with X patient hypertriglyceridemia, olezarsen And the second tens disease, collectively indication launches donidalorsen, need; X multibillion-dollar large years: a no just for Alexander additional TRYNGOLZA a the treatment help represent next for zilganersen deliver and to independent track is revenue severe leukodystrophy on sHTG, for beginning. over or thousands a of opportunity high patients approved therapies. provide These rare year;

which hepatitis for a sefaxersen, bepirovirsen and causes development disease one medicines, TTR Ionis. including considerable If medicines for and failure. patients our hereditary SPINRAZA investigational for kidney virus, previously of for addressing hundreds referred IgA therapies; these with independent marketed partnered for chronic an programs disease of to with that X range over to partnered also years. high ATTR addition no target poised In with important that factor are continues launches populations: for late-stage our need underdiagnosed progressive including diseases, cardiomyopathy, medicines more need effective fatal launches, from pelacarsen for cardiovascular independent cardiovascular life-threatening condition in of and join of disease revenue risk be are disease, treatments; approved, that significant most unmet the several common X next broad approved Lp(a) we for treat eplontersen SMA These would to expect and as a the a polyneuropathy, nephropathy, important for a serious WAINUA patient globally; serious other affects people B of value chronic IONIS-FB-LRx to kidney generating millions

advancing efforts bolster science plan base enable to while more potential to strong to reach the commercialize. patients, growth our our provide and on medicines allowing partnered focus we medicines innovative our even Our us revenue substantial

to achieve flow. And we positive come. sustained is are cash patients Ionis to medicines important to turn, on positioned and revenue bring to a and years substantial path growth in for

accelerating value ongoing recent and drive investments us position Our stakeholders. well all to accomplishments Ionis for

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

Kyle Jenne

you, Thank Brett.

ensured new start integrated prouder quickly. of first stage to FCS our launch as I seamless TRYNGOLZA, we biotech, patients activities launched got fully this execution our As team. initial chapter Their be important of that commercial cannot independently a medicine,

of XX clinically first channel And within hours. self-administered prescription approval got weeks written and of drug within on first in both the team TRYNGOLZA, the patients drug. confirmed seeing genetically The week the encouraged are TRYNGOLZA X was within Following we today, patient approval. X and by diagnosed

breadth time it's of by and TRYNGOLZA; of in including the prescribers, FCS in patients key areas: early encouraged launch, we positive are prescribing the physicians and the While mix response several identifying endocrinologists the cardiologists, receiving with both to rapid patients. numerous and prescribers; and seeing an interest commercial by repeat the lipids, already coverage Medicare for patients we're TRYNGOLZA; prescription that from and in payers

team experienced this on continuing momentum. laser-focused is Our

ever our in and that first identify capitalizing U.S., experience As the and to is early treatment long-term we that on us encourages enabling are advantage patients first-mover treatment create the develop market, the adherence. positive onboard

all of for which success long-term it establish will the expect in us Additionally, presence has TRYNGOLZA. enabled we community, a to patient drive strong

addressable of unidentified FCS total up remains vast population estimated The undiagnosed. to the currently, population patient majority and X,XXX FCS approximately U.S. And the to is be people.

steps result, are a on patients that and As TRYNGOLZA. there to get identified are we taking multiple critical

works diagnosed. diagnosis, through commercial First, who our our patients increased team increase engaging to is prescribers to and HCP obtain field not working on to and awareness focused actively education patients to Building FCS drive working and and coverage team with initiate the FCS on identify these triglycerides. patients patients identified TRYNGOLZA. HCPs are treat with efforts, who high is team closely patient-finding for team is with yet are diagnosed, treatment Once our efforts,

have our support this, patient by innovative services program. We anchored accomplish Step of Every to a Ionis deployed suite

managers administration of education, disease training, patient and part reimbursement and program, provide this As support more. nutrition auto-injector patient education dedicated

coverage To now delivery, program. Step treatment experiencing have have HCPs, offering as refills. engagement process sharing streamlines and Every well from and patients, as their the excitement they they the as very and that high we For prescribing coordinating assistance a are Ionis the by insurance reauthorizations date, authorization had TRYNGOLZA with services

and of through And market teams burden been report patients. pleased access these U.S. the places have of a to have received the early we the efforts, with patients that in launch, this have payers broad significant lives. actively of engaged access set are covered TRYNGOLZA. FCS reimbursement vast recognized days on With the payers, representing Our recognition, majority to timely

Finally, reach our our and of amplify physicians. the with extend commercial omnichannel capabilities patients team and our efforts

we the will time takes trajectory unfolds. execute expect on efforts, fully Our momentum it these as gain which the launch year will reflect to

full aspects impact patient our can blockbuster of potential life-threatening sHTG the that pain milligrams events deciliter address per we and substantial scale infrastructure plan further a and organization is to first-mover over advantage. that where symptomatic sHTG can suffer comprised where care. realize with severe commercial To abdominal pancreatitis require triglycerides have the suffer to of most olezarsen, of debilitating people is including chronic cognitive life, larger their sHTG people population from intensive we and XXX manifestation, impairment. hospital patients In disease symptoms all severe the much

a physicians guidelines already importance lowering severely triglycerides the As recognize of in elevated result, place. with established

potential offer anticipated is Many on reduce is with of could launch are recommended in current with options options. current pre-commercialization activities the an why and, people next This are we levels to year. triglyceride to about treatment severely sHTG focused Today, with sHTG. people olezarsen guidelines excited ahead underserved to unable the many available cases, by are team

doubling throughout product for growth medicine with delivered a the hereditary be WAINUA existing growth WAINUA, be on year, pleased our the strong to last polyneuropathy, adding patients to launch. including third as demand community as excellence has the setting. market co-commercialized switches broadening and Patient WAINUA the quarter, strong, sales continued and compared the the come quarter. couldn't top continues ATTR with from into sequential Uptake of accelerating to to patients with to for strong centers majority been use and the WAINUA in of therapy. AstraZeneca new now fourth we their about prescribing treatment with more nearly progress year, well of

of ATTR to cardiomyopathy, polyneuropathy global of access will and provide received We initiated to pocket. physicians are positive the who to dynamics confident treatment, The AstraZeneca WAINUA estimated their saw sets improvements, pleased The consistently patients. about a with type, self-administer their regardless patients' vast XXX,XXX continue patients WAINUA worldwide. including ability for out with rapidly growing patients' and and insurance address for launch majority opportunity paid our in ATTR $X XXX,XXX quality XXXX. from we people with of easily a are And WAINUA reimbursement affordability to foundation patients' differentiated feedback ability experience life efforts the of substantial experience WAINUA. We to we WAINUA,

donidalorsen. Switching gears to

our X the away, months With preparing independent approval the just second launch. for team potential is

that to to after be they donidalorsen of bring head a customer-facing hired recently to we team approval. ensuring sales add patients plan the fact, soon, ready donidalorsen and anticipated quickly In will

team building also WAINUA upon and the is Our leveraging TRYNGOLZA and launches.

innovative expand Every Ionis Step will to support example, For we patient HAE the of include needs patients. our program

medical This has in building our data anticipated medical clinical our of awareness Ionis' ahead the presenting visibility field ensuring the meeting affairs technology physicians. generated Our platform conferences, includes with robust positive of launch. been team at program key high from Phase and III

strong HAE attributes grow, people And the clinical better to treatment, treatment the to segment including while as data, believe prophylactic auto-injector, X an well the patients the ramp defined, donidalorsen to in patients. of work time search a switching while the offers for take More with the treatments data estimated launch looking donidalorsen we U.S., people has treatment many patients meaningful we of to and continues simplicity prophylactic the a are donidalorsen existing potential market have preferred Based treatment via self-administration a from XX,XXX to convert from HAE In many every HAE HAE on we patients to in with and the months are expect prophylactic for. be monthly we donidalorsen or current U.S. believe that is donidalorsen's option. than positive of Europe. switching in

We have experienced, commercial built organization. a and highly scalable efficient

its over for to while more successfully to sustained are X the focused with TRYNGOLZA launch growth success. preparing and long-term the X planned next underway, additional With positioning execute our FCS years full potential on Ionis follow, delivering we launches

now to I'll over Richard. it turn that, With

Richard S. Geary

Thank you, Kyle.

submissions We success included positive launch. numerous readouts, late-stage pipeline had These regulatory our in in in first multiple for XXXX well position data XXXX. resulted which important independent us many achievements and that

and with on next, for to are we years advances, serious track bring transformational and this pipeline medicines to our diseases positioning readouts Ionis multiple data to actions late-stage come. regulatory as Additionally, for year people

safety the XX study, triglyceride III milligrams reductions, FCS study U.S. the demonstrated and clinically pancreatitis, with TRYNGOLZA, days reductions from for profile approval. spent to benefit a with in data the BALANCE first acute the positive in approved adults and with Phase all-cause adults data favorable people In with the BALANCE treatment tolerability these in formed and believe meaningful FCS. The underscore hospitalization sustained bring Starting TRYNGOLZA substantial a and TRYNGOLZA's We positive for ever in in basis can substantial hospital FCS. tremendous TRYNGOLZA the reduction the

starting sHTG patients And second available are quickly physicians while expand more to are with and with their unable we broader Many for we effective treatments triglycerides treatment. expect currently a sHTG. a are manage FCS, indication, eager to to

As a reminder, we have X separate studies.

requirements designed high line to from report with safety pancreatitis. between top expected a being levels per conducted was prevalent population patients exposure ESSENCE patient ESSENCE our triglyceride the we're who a expect results deciliter This disease. data satisfy commercially. We Rather, the at to regulatory year. targeting mg and not study XXX for this first risk is primarily acute XXX safety database in the support supportive in highly is ESSENCE, with study not for mid are to

and patient olezarsen the to COREX XXX We milligrams to from are These truly sHTG. olezarsen population look deciliter. studies, who triglycerides studies profile understand greater forward our per target evaluating have CORE our data in in than pivotal

COREX for assuming we half our and positive, track to the the year. in these before have second both data year-end. We submit results. are we studies, of these this we of are And on data And hand from sNDA once for data in plan report CORE will

diseases. FCS both positive expectation in already in sHTG for could With the demonstrated in compelling FCS, of care similarly results significant advantage standard be our coupled believe we with and both and first-mover for olezarsen sHTG, data

regulatory closely now donidalorsen EU. donidalorsen, potential behind for is the that under and patients both TRYNGOLZA in angioedema. pleased hereditary advance a Following is U.S. We're review with

separate were switch submissions from our III generated based positive a studies, Phase regulatory data and II Our including Phase on study.

high profile. level who Phase HAE demonstrated to long-term improvements life These the prior with HAE patients attack translated than a XX, reductions the reductions treatment III substantial clinically donidalorsen. this with reduction quality surveyed measures. year, showed look PDUFA tolerability bringing a In study, both and meaningful baseline, switch and a rates favorable prophylactic of further to assuming switched preferring In to attack in of the from bimonthly August XX% HAE program, from forward treatment of donidalorsen XX% and a patients monthly and rates dosing patients treatment disease to date donidalorsen safety prophylactic this more control of potential later of we approval. nearly in preferred With in

rare Rounding Track out leukodystrophy serious unmet the granted and our that pipeline, disease. by medicine no treatments. owned that disease, approved Alexander need FDA, an Fast to our late-stage families zilganersen has was is zilganersen, exists and this Last here ultra-rare reflecting patients disease-modifying profoundly the year, Ionis impacts designation for treat

this III reporting to forward year. late looking are program data for this We Phase

excellent we our syndrome, of believe which this our rare year, alignment medicine of based positive Phase and with for the for owned IONXXX, This potential the patient wholly with thousands pipeline rest in progress. patients III conduct making early has FDA in results Last Angelman we living also adults the includes tens date. of to serious The most syndrome, study reached Angelman is investigational this comprehensive transformational rich of study the disorder. children robust IONXXX population HALOS from and with to on

our clinical study Phase start end pressing year. study. half outcomes Our results on people syndrome, track study points and III this focus Angelman reflect remain to will that We with the living showed first in on most positive the IONXXX for in points meaningful end of that HALOS

we look key have we made As already events, our to progress. value-driving excellent

to pleased recent the and In higher-dose market launching closer TRYNGOLZA, addition to we submissions. that acceptances regulatory step with are the FDA is of nusinersen EMA one Biogen's

is positive of higher-dose profile the positioned the to established to past well-characterized data well SPINRAZA SPINRAZA, years patients the from around we bring over benefit world. the and the SPINRAZA With SMA continue XX-plus believe to

key we X readouts, we owned XXXX, sHTG X many of launches. actions anticipate readouts, medicines, broad summarize, olezarsen the this data To zilganersen regulatory By year, significant near-term for indication late-stage end including data product value-driving and numerous events, Alexander III for and the Ionis expect Phase disease.

medicines patients we and medicines have discovered independent next which data, start could patient are these underway, soon for to launches. track as of Next, Phase X launches Ionis on populations. X broad for are reach III very most X as If to positive, year, including groundbreaking expect

of Ionis up of All sets a this come. years bring for to us to cadence steady new medicines to patients

Beth. turn With that, over to I'll it

Elizabeth L. Hougen

Thank share both reflected results our growth for our independent year, upholding for I'm you, excellent advancing financial and Richard. our We've leverage. drive made year our This including progress to commitment in outlook you operating strategic today. executing this happy strong launch while important is of our priorities, which simultaneously to last progress first with

non-GAAP of to a operating a compared million, guidance. XXXX We delivered loss significant our $XXX improvement

revenues by more revenue our exceeded year. XXXX last than of guidance $XXX million also substantially $XXX We million, earning

with commercial and option. which product year, accelerating we of in throughout driven by the for to million the quarter source of underlying WAINUA compared sequential by XX% were encouraged look Notably, year. product million to anticipated SPINRAZA's During year royalties. We last SPINRAZA the earned quarter. of remained product performance demand. million third for the $XXX forward revenue last launch $XX revenue WAINUA XXXX, the for in achieved were revenue fourth primary the higher-dose sales $XX growth WAINUA strong increased royalties

of The XX% our the XXXX of reflected U.S. sales in As donidalorsen. non-GAAP in upcoming slightly operating launch planned, launch and for increased marketing increase the preparations investments and over for TRYNGOLZA our expenses year-over-year XXXX. expenses

our costs, included and teens also WAINUA's marketing appropriately expenses while which rich in portion line SG&A low-XX% to stable are minority R&D range. In of funding expenses kept pipeline. high we sales with our the plan, year-over-year Our our

creation. last our us Our financial and well value continued with management, coupled excellent progress year, disciplined growth positions for

numerous our company, reflects product million evolution operating for maximizing and deliver approval. to steadfast with revenues, on earn medicines of more revenue guidance commercial-stage $XXX shift a commercial addition to this innovative We integrated than product including in value while of fully assuming TRYNGOLZA leverage. our strong the revenue in project the our financial revenues the Our commitment year focused to biotech towards donidalorsen from sources, initial remaining

to important is first that anticipate it's and buildup a especially FCS quarters. of TRYNGOLZA, remember disease, rare With we launch and initial momentum, gradual the few under-recognized an in

of acceleration of awareness approval physician product also takes expect looking diagnosed their FCS this As to it a to progress, translate we aimed patient with the an convert donidalorsen is therapy progress. assuming the to our and patients at to We're launch revenues forward beginning turn, expect donidalorsen. market, our time since adding which, number efforts primarily increase, driving in launch the new we QX, will patients And switch reflect August. in from existing late in to to identification

we earning commercial These which medicines be From our from partnered substantial source. on market the our largest SPINRAZA, to revenue programs, include royalties continues anticipate today.

to and that. WAINUA, the has expect this includes expect will year. And We resilience continue we royalties to also continue its SPINRAZA demonstrated upward reflect this which our trajectory

expected each at the dynamics, that royalty with beginning progresses. the our of rate year coupled for revenue resetting as will year, commercial SPINRAZA we these increase With of the anticipate the launch

on in earn milestones, numerous of our half timing potential to the And revenue the guidance, of although pipeline it rich With than Our meaningful R&D we was to programs be to this total contributor revenue to anticipated many we earn year. a remains year. a R&D have milestone last expect our second and the we based the much revenue lower of year advancing, partnered payments. it expect

the earn of from study we Additionally, XX% revenue continue the for continue AstraZeneca its we share to costs. as study, CARDIO-TTRansform will to conduct

to patients single-digit compared operating year. range our while in stewardship. the exercise sound expenses directly fiscal This increase modest to advance high XXXX continuing commitment also to our project and We our to bring pipeline percentage increase reflects last to medicines our

Just come multiple marketing growth as from and invest in launches. success XXXX, our will the planned of to our as we sales expense ongoing and expenses increases in planned our and support

R&D expect last to in to expenses similar remain We steady year. XXXX, our

have of several are wrap Phase our or year, of for reallocate up just syndrome. track to have As our toward resources the able are concluded next wave we on III studies opportunities, late-stage Angelman development this we our recently done of as IONXXX for to

revenues are We non-GAAP growth, end $XXX loss operating to than $X.X sizable we projecting with of and year expense of With and less the a approximately billion. modest million. project investments cash

Our advancing change to directly in while projected exercising patients in to year-over-year our cash medicines our medicines bring fiscal to reflects investments wholly responsibility. owned addition in our development prudent

us the continue XXXX, beyond deliver growth. track numerous to leverage, to peak multibillion-dollar have including $X drive on to today to To positions that IONXXX. positive with to olezarsen directly sales our and growing growth, substantial pipeline a pipeline Ionis commitment programs potential. revenue achieve combined goal, our medicines This also advance portfolio expect operating are estimate patients. us, making revenue our revenue with bring Looking billion well our investments we opportunities significant annual than our we flow. of peak of cash medicines, generate owned the combined more sustained before and in includes and our from We estimated to This

billion than estimates, sales annually our on $X more royalties partnered from could peak late-stage we earn based partners' medicines. our in Additionally,

have potential, revenue flow. robust growth as can positive a see, clear to you achieve So path we with cash

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

Brett Monia

hard Ionis. our all progress Becoming biotechnology expect the we to company you, this exciting integrated fully we continue enter required for Ionis chapter Thank and commercial-stage employees, commitment Beth. next for great as work and a

through prolific medicines with that we've has differentiator And with innovation, has potential, been this Innovation and engine pipeline proven development key for Ionis. discovery rich established and provided us a a and a always transformational will continue. of

FDA SODX-ALS, X delivering consistently now for donidalorsen drug for TRYNGOLZA QALSODY approvals fourth this and ATTR FDA in with later approval polyneuropathy just pipeline year, Our WAINUA and for is HAE. X important years, for a anticipated FCS, under

positioned and year U.S., we with and the more pipeline, several year III achieved an deal in we years. are a with next And positive several and have Phase achieve to deliver our advancing that approvals today, outside to X commercial by launches X and great and patient we expected additional organization expect place, both provides coming years. treat cash important for this us accelerating next also innovative to more with We've have broad data developed experienced, populations. Ionis driven value rare positive in And top delivered being this a growth. readouts late-stage SCS all launching This sets substantial flow over highly revenue for currently line TRYNGOLZA clear a scalable, for expectations for us This up stakeholders. the progress path

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

Operator

question Mike Today's comes Ulz first Stanley. Instructions] with [Operator from Morgan

Michael Ulz

on the kind sort on population, just from is And of ahead study progress. more of to given a just safety studies? make supposed ESSENCE and read-through I different sHTG, one Congratulations but midyear you're a we core what of study out the all and it's to could enrolling your studies. of olezarsen Maybe realize ESSENCE read core the

Brett Monia

you, to Richard Mike. study, elevated before, triglycerides, is a It's just not population. population, XXX target that XXX. as described as ESSENCE The our commercial is patient patient reminder, a Thank mildly

milligrams deciliter per patient above. target XXX sHTG population triglycerides and of is Our

study, a you is it safety So as say, Mike.

be As confirm, the certainly we seen will demonstrate FCS validate further that for key in patient and to a safety think the safety we've read-through, read-through population.

lesser that but a study. to is somewhat slightly predictor study population. because target it of study. We, of triglyceride expect sHTG, is in some course, APOCIII less So what that that's focused we'll read-through looking that really the a in be on That, different a still safety will and as it we that direct, we're a very But the indicator reductions we think as patient of be sHTG in in what expect will engagement to important, see to still at extent, sHTG. extent, in an the well reductions as

Operator

with question JPMorgan. next Jessica comes The from Fye

Jessica Fye

you're TTR could a what little in the talk market. bit you polyneuropathy hoping about seeing was I

what of there? starts think new capturing example, do you're For you portion

Kyle Jenne

Jess. Thanks, Yes.

so earlier, XX% what of that communicated within The ATTR growth that's Beth As launch polyneuropathy. now. category demand an year, the right million of and about that $XX I and QX on the WAINUA million of continued with to continued $XX we're on progress strong excited and the speaks here increase the is think in growth the quarter-over-quarter

the Currently, new-to-brand is WAINUA. about share for XX%

So a continuing community terms we patient those, to prescribing. we are of to expect not of We're of begin capture, new to starts, significant grow seeing time. only centers portion in over that and continue also physicians excellence, but

So the expansion, still switches prescribing. both combination a mix well neuros And treatment we're are as WAINUA. of naive as and cardiologists seeing for

XXXX growth setting to continued we year see very XXXX in WAINUA quarter-over-quarter. So be think, for is really to saw the strong up, a that I

Operator

The is with from Akash next question Tewari Jefferies.

Akash Tewari

your size how also your your mentioned more III? does abdominal other really, would particularly clinically previously effect you on maybe in well. for helpful meaningful event would acute And FCS. the study KOLs you isn't that rate that Can what then that the design feedback of representative And be assumption necessarily pain? trials. little trial I be severe any for talk FCS? a sHTG, mentioned the pancreatitis a But on powered What very does mean imply reduction for, you enrollment ESSENCE from differ XXx as versus you've rate that that Phase about and is of

Brett Monia

Thank you, Akash.

very unpack, a also questions, then take jump there important at me in. can things Eugene were Let a but stab There to that. few

of well Phase sHTG primary CORE powered for powered, are triglyceride is our our study, reductions, course, point. and study, COREX, end III our which So

were on a we studies, As patient we very far pancreatitis, pancreatitis We this what small never did not in impact had tremendous acute as in study. events powered see AP acute at and believe it a positive that sHTG were outcome in been events FCS, though has well CORE reducing a the impact was we studied for really AP before. even surprised to pleasantly population,

average triglycerides the obvious established. higher in somewhat the population on acute FCS. the greater well has The sHTG The of milder and compared pancreatitis, risk to triglycerides patients, that's

allows you of more the rate patients events. So core XXx Nevertheless, of of that in pancreatitis might a lower patient course, acute in accumulate more to have expect potentially which, our we population. than studies, number us

not significant So some a be studied of acute or in would so a necessarily no BALANCE the a power study in meaningful ever confidence data lends in to on pancreatitis although reduction this FCS signal that difficult we're powered sHTG. it we'll us AP sHTG, one's certainly, before, see and

anything Eugene? to that, to add Like

Eugene Schneider

No, not really. was clinical another about reduction. There question of meaningfulness

Of as be terms no really reduction. particular There is considered course, in it's said, clear. Brett of not meaningful is threshold AP clinically to that

said, any based as we're the any able hoping what to existing significant not is is really reduction see of that But we So overall. something course, were Brett of model powering data. study the on to

Operator

Yanan from question Fargo comes with next The Zhu Wells Securities.

Yanan Zhu

quarter. Congrats on the

by plan collaboration switching? couple And I they're more of updates growth. main force of that those Biogen growth First, I how announced driven but siRNA? programs? much neurology my How question to a Because wanted from switch question, to have quarter-over-quarter XX% hear patients of switching for little wondering did the provide a quick follow-up what's I programs. And to you think a a a color? Could is and is both prior on the patients, polyneuropathy naive the WAINUA much some

Brett Monia

Sure. Kyle, growth, please the that, quarter-over-quarter to Biogen. what's take I'm question on comment on driving and happy

Kyle Jenne

treated Yes. than XX% know The we currently being therapy the the with in of space patients this polyneuropathy today. in market, are growth, the as less

terms identifying remains newly and diagnosed opportunity those seeing, and in that's that have AstraZeneca that's started naive we're patients of here focus this grow patients teams what So market. the our getting really the and And to on treatment.

and if WAINUA regardless Medicare that in quality patients. of a of out-of-pocket or is life. the strong. tolerability consistently as there patients, is highlighted, physicians What is are improvement they from extremely of profile commercial we The and treatment majority That's to are access have The is I hearing very expense. safety $X strong

So be extremely and medication at resonate well. continues able to top of WAINUA the afford on of place choosing self-administer a the their to to to the profile ability

WAINUA of physicians expands, market are the choosing as these over some treatments. So other

the on very have coverage. not own therapies, physicians are can good other from associated a really if do is main they're patients switches the of We're to care site are seeing choosing this they access very of WAINUA. with there self-administer. and where and and instances driver ability are their And place the choosing, the to patients good in challenges that some and for that this do Now able at

medication that. to with that and the we're due really it's So combined to self-administer, a ability differentiation making highly performing seeing possible a very with quarter-over-quarter, growth

Brett Monia

in your question, of then global the for Yanan, these are atrophy development, you're is be programs couldn't for system alpha-synuclein the that to and to disease. thrilled both II of And that retain LRRKX is other control regarding programs. We these referring Parkinson's indicated part we couldn't rights programs. program for pleased to which program, These our of full more retain Phase multiple be development, more

going with cohort these, full was to study the said, to control access to a by contract this Biogen Biogen this in we're decision Just thrilled particular add program. on color a of It's little they had to at opt this in based a alpha-synuclein that's very the important to And partial for time. data. the on And only really based program drug I forward. on required low-dose This fact, ongoing. like the the have

forward study. reductions safety. magnitude very study, drug small engagement, and LRRKX forward and our PD pleased be and The safety have developed, to with The in the for was path LRRKX meaningful a of we we're both XX-week a definitely with Obviously, alpha-synuclein. study going LRRKX clinical and saw treatment. designed But saw we're to XX the very to further deserves data very in forging looking we're programs, patients. was we're study target pleased a in program This weeks' we in for what data, not any

MSA expecting have we're alpha-synuclein, year. to For data in this later

Biogen was by partnership R&D Our very this decision with remains and strong, essentially prioritization an Biogen.

Operator

next comes Jay The with Oppenheimer. question from Olson

Jay Olson

Congrats progress. on the

help U.S. metrics patients And you I about launch how how and many on diagnosed TRYNGOLZA, track those patients those early, the treatment? X,XXX many in you are talk could to Or available For treatment? think just mentioned other of TRYNGOLZA. if it's you of are currently the any too of important patients Can not share for

Kyle Jenne

look excited lot going well. launch Yes. TRYNGOLZA, a clean for clinically identify; Thanks, label population. right get is we number label. number at of about and that broad patients. now Jay. We're very prescribe allows When prescriptions label. talk on TRYNGOLZA very, written I then disease can't drug. to, genetically diagnosed received, doing two, patients and obviously, get it's you rare a very diagnose; the work these to physicians The It's one, be a to of kind finally a more or the It

very acute the to pancreatitis. in advantage, AP TRYNGOLZA move allows have develop to demonstrating market we've got asking in substantial the and on And to this which and about reductions quickly. that first-mover take us We label, them you're patients

the et already option that excited be are before. we're feedback receiving all HCPs, really it they've cetera, a the The is had that been treatment patients, to positive advocacy a the stakeholders, TRYNGOLZA. treatment groups, very, very have option of payers, never have They about when

plan, to this end to treatment do before to able around we be very we've these going deliver to of patients had in trying execution been everything And the We terms year. product in In well. of XXXX. that the launched channel is

Now get identified, they that of and the currently become as there written come are are mentioned, several in, right now I diagnosed and formally are hundred prescriptions X,XXX to continuing patients, for. and

terms here. ramp of and some the time the in got launch we've momentum So

of what we've identifying number disclosing physicians by that that we're metrics. metrics, Obviously, the many now On is is encouraged seen market this really right and competitive not we're we're in. prescribing. the a too But

got breadth we've good prescribers. of So a

specialties We are seeing a well. as prescribing mix of

have interest lipids expected. in we endocrinologists have be which an TRYNGOLZA, also pancreatology and was are Those So and prescribing to that cardiologists.

time on working early keeping on. through and that's these exception very, prescription is to medical prescription takes is patients prescription eye very it the process on had expected thing from it's treatment telling long these that's we confirming that we're the actually is the to other to getting into justify that go clinical The And us And drug in than either the to physicians are diagnosis, if how faster what patients. the going patient. quickly, order or FCS And an and getting quickly. filled genetic, here

positive of basis. very us profile with is giving a here do of drug very ability process. are and through and playing our And Every the an I'll that final on monthly is us the disease say allows reimbursement about in executing the program is with and feedback Ionis of weeks the directly self-administer Step to patients auto-injector positively patients product And very And to out well, support interact first the launch. and them thing the couple education education

asking. for thanks So

Operator

from of comes question with America. Bank next Gerberry Jason The

Chi Meng Fong

consideration. commentary you the follow requiring for launch on to like some gray for I onboard, of of is heard we experience, a do would doc to Chi Part terms it Jason. get guess of when is checks, one criteria. TRYNGOLZA FCS, companies that on We've in insurance experience it bit based clinical so documentation is companies diagnosis I'm reimbursement genetic early are the in straightforward think somewhat thanks of lack the and have diagnosis. documentation all what thing the done area on launch curious, confirmation a comes that This to consensus early for I far. launch is insurance up most

it of Can Some you clinical what about insurance companies criteria. that? as accept talk diagnosis is

you X,XXX-patient formally many about estimate, will diagnosed, talk And as how identified are talk and several of have, great that genetically how getting diagnosed? that currently those if getting within well. you mix And be many hundred when are patients can and of about prevalence U.S. that clinically confirmed those that become you

Kyle Jenne

Chi. Thanks, Yes.

me let the on just First, touch process. insurance

months, X is is exception to process. X typical, to first go a as The going through medical

established to formal is far criteria plans so are say absolutely have there few this required. what's So that very

physician actually the the drug ultimately straightforward patient, through get genetic FCS The truly gone the the they validate then is question to becomes most by the steps coverage of the confirmation. that So so a what is approved. has patient history that or substantiate and plan is what an this get the can

that's think the I one. easiest

medical both work we with process symptomatology, history efficiently their the through have the diagnosis, potentially history. patients exception acute levels, diagnosed clinically the abdominal patient, pancreatitis age of in seen very and pain, of genetically However, justification triglyceride right, about patient

of the for an we're of both I the And from business. qualify So commercial there approval are a patient will this the seeing segments other justify of terms as that that presentation in the Medicare health things and or well, unpack plan. mentioned ultimately patient through as to lot

So what patients. we're very FCS that most identifying optimistic patient about is it quickly. that two and one through physicians right through process They're justifying They're those are of doing this supporting the means the way.

scoring Dr. a of be at for for Hegele and seems that to of year, many tool the yet. on seeing terms been point, be a with terms approval. not gone through to process. this number is use able share straightforward have have of we can published in that obviously, confirmed lack heard NAFCS are have we're patients been I how genetically don't But that it clinically getting and Using versus them it of to validate end last the that both what that approved based have you published. I substantiate consensus, tool the in at these The has being pretty our fact

working payers to TRYNGOLZA. X there's then months that learn the to have mix, patients here be be of a we're on coverage in with but for the at criteria terms throughout payers so, directly the sure So first to and order other I'm these more make not encouraged to break will very forward than to access or path establishing the to that, coverage. and early There's out reasonable in able

Brett Monia

We're as route. that so will of experiencing genetic that Chi. that familiarity we also think just utilizing add FCS the reasonable I with And genetic assume a driving they we're to Points to speed with that the time goes build unfamiliar to far, physicians. North become I'll lot it's X that on. X American that when diagnosis very any go we're weeks And pleased with especially diagnosis aspect that, with criteria to of diagnosis today, work, physicians to are more diagnosis that with familiar a

go to expect as So that forward. we improve

Operator

with The from next comes Lebowitz David question Citi.

Ike Lee

trials Lebowitz. sHTG This David for point is have the on primary. on readout. the time the regarding Ike It's the XX-month on I in one Lee also

patients, different FCS So they're than as we be levels TG coming likely rates going in sHTG lower know of that at and to in APs much with these baseline well.

no there's said, lowering on as just TG line. reasoning this if wondering, was that the XX-month selection wondering much of I'm clinical in what beneficial, understanding consensus behind actually so overall, population so what just the And what need? time as And our to you how they far is is

Brett Monia

question. with Richard, comments. touch that after the up my follow want on for you And anything you to thank So

is primary the at at full end at the is so months triglyceride So point X readout months, end TGs. months. study on point lowering XX primary The X

We for FCS and our of have based triglycerides, APOCIII, our a substantial very on as data rapid rapid substantial TRYNGOLZA lowering response well on previous reductions, lowering APOCIII BALANCE data. of clinical as

point. believe So well end powered very we strongly on we're the primary

clinically we meaningful a it to be based although on saw that's reductions BALANCE. Regarding We position AP as in not we're documented of III pancreatitis, Phase show the And on our in literature, a I in read-through good acute what data in FCS mentioned our study. as believe is based that acute this pancreatitis after in patients the rate earlier, will outcome. in sHTG well

don't think that, Richard? driving that's I a lot you, of

Richard S. Geary

and that Committee by events Adjudication Yes. adjudicated I our the see accruing this it's course the study, we being would over looking are And of and also independent say very events good.

would and has The determined said that risk triglyceride FDA Certainly, by levels say clinically increases occurs XXX, has meaningful, other regulatory about been and the agencies. above it I triglycerides significant as thing the increase. the

the for acute having an risk So you're decreasing pancreatitis. triglyceride by patients levels by amount decreasing is, whatever the these

and obviously, we'll of to soon. pancreatitis, that's study, reduce their is be I the see the to able very acute risk monitor and primary that triglyceride and think results So the for goal

Kyle Jenne

are meet from already their Yes. reduce And other a are triglyceride-lowering treatment and oils things, add guidelines And fish me Currently, endocrinologists fibrates on patients and to effective that viewpoint. and triglycerides. triglycerides a They let need try to severely better, commercial place. that to with these statins, in to more order are the in not patients getting established cardiologists low enough. elevated

a about to guidelines Richard regulatory out the in already talking there are So order of from standpoint. risk, XXX was as say be that to below need get you

we accomplish patients a trying are because that most of unfortunately, to just, an of in able treating in have commercial a don't the to And what shortly and And not market do order sHTG approval bring physicians that's be hope that. to olezarsen. with already. know to they're they're But there, here importantly, we hundreds they from that's therapy get standpoint, able so with thousands to sufficient to

Brett Monia

And an because often because pancreatitis, serious land I disease. are having important will disease in AP of event AP nausea of body that, body such so a also asymptomatic an to in serious patients in and success, in they serious go to these hospital acute It's fear we it's the success, addition and pains, pain. think that forth, often suffer very without this sHTG impairment, not is event emphasize symptomatic cognitive of the think TRYNGOLZA an commercial even long that a a way sHTG. in that, from

Operator

Gary with is from question next Nachman James. The Raymond

Gary Nachman

congrats well the My progress. as on

sHTG, let's launches just for new in what's What's you're expectations about olezarsen in your program, doni versus other for HAE to scaling place for the for competitive prepare organization you reps as how the the Angelman just in in commercial So launch. and talk the you those And FCS timing there? enrollment specifically the given programs? dynamics for add, of then terms what need following

Kyle Jenne

Kyle. is Yes, Gary, This start. thanks for I'll question. the

internally. the with our Fortunately, on to towards we were readiness able build WAINUA, co-commercialization FCS then

got group. So our market teams around and we've commercial operations access

kind Step capability into follow. year place, the on commercial accordingly our to us such have this components donidalorsen and are later sHTG scale commercial in go, new support foundational program. fundamental that team the therapies Every ready our to they're as we with Those mix, we've to patient Ionis of add We the whenever that core got and allow to

we've Where hired is right President with we're at Sales. donidalorsen a Vice for of example, now,

And build regional with and for out and the in director time anticipated from and in August we're the approval is doing our XX. sequence regulatory process our that ultimately, will what customer-facing field there. on We team teams for

to the an over right I make right within and a and those also respective amount sure sequential time, it's functions. at capabilities build, build, that time a been it's the we it's right purposeful as as been well, the been right think So build intentional mean, build now, investing building and I we're

I'm but When as into, at it would we customer-facing to we to be of size just get add with And the the within talent our based about will overall. be commercial already are in have exponentially, able that the that the place teams had group we time. the the expect, the that have right excited teams we'll product and our that tremendous accomplishments entering and you on good and we'll market field grow sHTG,

Brett Monia

so be is difficult We half will our by that believe community, in completing our need. year us year. enlist, established patient inquiring program to enroll will about And on trial internal in population enthusiasm they this large And Angelman well. team a going of We're for III by next still the tremendous we in encouraged the with the this enrollment enroll. that first year. the to metrics unmet There's are study track do enrollment, by well really pent-up to Gary, XXXX are this Phase up the our Things be enrollment on sets to to with also We've when initiate achieve respect able relatively not this demand. study.

our So those are for expectations enrollment.

Operator

Cowen. with comes from Yaron Werber TD The next question

Yaron Werber

I have quick question. a

you full Are you design that? as know haven't keep particular to we then mentioned driving just If how need you about communications. in are III Beth, half think I but yet, trial there And mind think revenues, power second milestones the Angelman for weighted. that about you do Phase expecting the I'm it's maybe more you will study, announced

Brett Monia

would Richard, the course, on comment powering? jump Of too. Eugene, you in,

Eugene Schneider

Yes, sure.

Bayley, population. known So older the kind assessed which of our their patients primary little a natural to how regard expected in quite see to expressive data as we able but primary as Bayley-X. communication point slightly ability end available, kind There's unfortunately, is, to is expressive of by progress on with deficiency know, of a communication. no history make are They this have utilizing and communicate, you of bit version to are to estimates nonetheless,

very Phase a the on we're encouraging So us for with Phase that, Phase allowed size assumptions to some II, see III. effect really placebo-controlled data, to setting in treatment make that our expecting combined II

As you know, we're also testing X levels. dose

again, our patient what population, But So essentially, natural of X:X in use in is history that, we the trajectory it's a and assumptions. knowledge this randomization.

Yaron Werber

placebo? Are both against doses powered

Eugene Schneider

Yes.

So our see to an goal of effectiveness is both doses.

Brett Monia

of uniformly encouraging instruments, showed for what, versus on mentioned in is, on X:X:X is in treatment XXX along all So et in using powering the are III doses burdensome cohorts coupled most greatest is the the best which Eugene very fact that, Phase evidence that as the examined, we why efficacy HALOS in we be with end we settled II Phase Vineland, the study. with nearly we you families. ORCA. And is Bayley-X, across that Angelman's communication of study, the on that's randomized domains patients, it outcome in with X a X phenotype, expressive both magnitude, HALOS look all which really, combined reminder, studied doses of study data natural placebo. that cetera, history the our II point that demonstrated X the study, same III our whether certainly at the the part the assumptions communication our study, That, where which saw Phase design, this expressive when was important SAS-CGI, really our the X:X or that Phase And

a you. for also there's Beth, question

Elizabeth L. Hougen

Yes.

we think in of addition the our WAINUA a quarter-over-quarter, shift grow this assuming half anticipate to revenue year, the the on continuing to So reemphasize, TRYNGOLZA donidalorsen the back with of guidance revenues, this really to revenues year just revenue sort SPINRAZA, for commercial I and, of year, approval.

you a lots we in those significant think No know, milestone. very have partnerships. particular different about medicine partnerships one revenues, as has development under and medicines we R&D lots of As of

get So the going XX% study. of I being host A us much as AstraZeneca back-end We of our revenue. cornerstone over piece those R&D then that a programs, Biogen there's could we AstraZeneca revenue there's to R&D get milestones it of III anticipate large and we to book be the we sort with said, and to Phase advance, the continued no others of really year that reimbursed the of seeing partnered point and big be I AstraZeneca for costs of funding CARDIO-TTRansform conduct that And as particularly with, the we as year. are course this whole from would from all-in that with that loaded. milestones they as will

Wade Walke

time have for one We question. more

Operator

will Blair. question last Myles Today's Minter William come with from

Jacob Roberge

couple on for for is Jake Myles. A This you.

whether first you were The would as launch is donidalorsen. on to wondering for could us partner you're at sHTG. that did or be find alone a for potential We color whether if it in your looking comfortable sort we give ex going of plans U.S. some you on and

entry with And then contingent whether next-generation clinical the on development targeting from positive second, or any Lp(a) and the asset updates a readout you have study. Novartis clinical is on HORIZON

Brett Monia

Thanks, Jake.

let ex with So FCS. before U.S. we get sHTG, start into me

will evolve, And today, TRYNGOLZA to U.S. for donidalorsen. like we that that will did for ago now partner OUS on due OUS the secure and sHTG the emerge commercial very programs X FCS, we commercialize the change U.S. working ourselves. progressing commercial consistent And we are out focus years laid we subsequently, that and partner where market an partners Discussions For to the OUS then to we will FCS and we're from planning time. But will both on secure and for secure with market. nicely. U.S. in outside it's our strategy bring initially, we market sHTG, an that commercial that And will we're

CVD been that disclosed year. supporting not last to studies starting away, respect follow-on year. now. with first them our offer will does Lp(a) look molecule, is not pelacarsen do the IND, much I the to provided we testing molecule don't Novartis great provided half publicly follow-on like as isn't for licensed And because It's clinical for for with on matters as initiating timing to a we really they is I whether compared follow-on and it the wait think best-in-class next of or that molecule toxicology have pelacarsen really that's to molecule but readout much before that the the to pelacarsen, everything far date.

So course it has there clinical to through for preparing run and its testing. then

really suited that's that's for Novartis. question specifically best a So more

the really you us But a our call. thanks looking thanks And everybody an thank year outstanding everybody, our again. progress sharing joined We're questions, to have today then, to and day. and But way the forward and for who in with along participated you. ahead great until

Operator

Goodbye.

Ionis Pharmaceuticals (IONS) 19 Feb 252024 Q4 Earnings call transcript