VRTX - Earnings call transcript Q3 2024 | Vertex Pharmaceuticals Incorporated

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Operator

Good day, and welcome to the Vertex Pharmaceuticals Third Quarter 2024 Earnings Call. [Operator Instructions] Please note this event is being recorded. I would now like to turn the conference over to Ms. Susie Lisa. Please go ahead, ma'am.

Susie Lisa

Good evening, everyone. Lisa the Susie of Vice is Relations. Investor name and Senior as President My pleasure you making to quarter my is It welcome results have call. On we XXXX to conference tonight's remarks, call, prepared Dr. financial third our Kewalramani, Operating and Chief Charlie Stuart Officer. Officer; Financial CEO President; Chief Reshma Vertex's Arbuckle, and Wagner, webcast that the you to slides recommend We access this listen as you call. replay The call on our available is being will recorded, website. be and a

and the detail and would in acute outcomes call will select this near-term assumptions. press of We note current are Vertex's the pain, now those will on are including, for uncertainties this non-GAAP call materially. I call are to sickle disease medicines potential in Actual financial to turn presented our performance review Reshma. discussed the and vanzacaftor that statements without cystic marketed in Securities moderate-to-severe on evening Commission. differ cell over in basis. I and launches also make and and based a could Triple-NCF that risks the and today's we financial including subject future release on will guidance limitation, with on our These management's the results beta-thalassemia, Vertex's the fibrosis, forward-looking filings that and regarding events Exchange statements, pipeline, suzetrigine

Reshma Kewalramani

evening thank Susie. on you and today. all, Thanks, the Good for joining call us

strong launch near-term the and approvals with year early for continued preparedness our in potential execution well momentum launch the early CF XXXX. performance in of commercial for extended both Third CASGEVY quarter outstanding as two as

on The Phase patients $X.XX to three going the we last growth expense revenue recognition previous billion billion achieved respect rapidly and XXXX we new launch for full in into X ATC pleased of the are increasing in guidance. With this our just CF, guidance strong a cell well. early XX% with the new milestone I'm we billion and QX the years pipeline on advance III we maintaining product ambition reaching the era are to X clinical trials this CASGEVY, metrics. programs X first deliver first and that with progress medicine. With outlook, track continue QX our more particularly are operating revenue, year the pipeline, in to received while QX, and versus collection $XX.X to this to remain the overall year-to-date delivered also launches in very clinical commercial and physicians is $XX.X We high, our our we share from programs XXXX. in and new patient Based diversification. X revenue representing enthusiasm over advancing pleased into and quarter. In growth as quarter patient significant remains to with resulting revenue product our CASGEVY, January patients

Specifically, to Triple severe launches in caster for preparing in potential cell vanzacaftor in CF suzetrigine third product the the the beta-thalassemia, acute sickle are moderate U.S. fourth in early addition XXXX, launches in and pain we to disease globally and in and

quarter. IgA inaxaplin clinical povetacicept also we're nicely, VX-XXX development suzetrigine strong and acute my I'll dystrophy kidney III in povetacicept Neuropathy, myotonic the potentially in that additional in making Phase comments X, that, nephropathy. Tonight, on made have pain four Beyond and focus VX-XXX APOLX-mediated in transformative our type advancements progress in in Diabetic IgA progressing neuropathic programs with and nephropathy this indications notable X medicines, in Type Peripheral R&D peripheral beyond in VX-XXX CF, all are programs mid-stage disease, diabetes and DMX, in and or pipeline

KALYDECO in the includes underlying with X X children as world. more medicines as as cause of patients four our XX,XXX marketed This around young CF, First, than treating disease young month in as are the young years X our as age with the fifth and fibrosis, we ORKAMBI for medicine and potential X vanzacaftor the with people year triple, cystic poised of years TRIKAFTA, approval with are older. of for as

have We the X Australia, PDUFA also Zealand submissions U.K., completed we have the date in in and jurisdictions Canada, in a underway. U.S., the EU, and New of these Switzerland. January Reviews are

We have the with trial clinical in to X also children already X. CF initiated ages

treatment MAD Finally, make continues share results in half seeks to of both the in do study, at our efficacy with therapy multiple in mRNA more CF dose CF has not portion. the from I/II first the than our therapy who portion VX-XXX, to CFTR in and the X,XXX MAD XXXX. people dose safety and and CFTR Phase completed for ascending expect ascending the Moderna provide the any partners we development with protein, or This single of

independence type completed a the advance cell regulators optimal to cell-derived Phase I/II share VX-XXX impaired hypoglycemic severe in EMA with and this the with and development difficult have to of events and experienced islet FDA, total the the VX-XXX our of and care. agreement stem end with diabetes X to am current primary endpoint Phase pleased diabetes, II fully interactions of following study pivotal hypoglycemic Phase severe successful proportion Next, into study. absence trial optimal the trial have regulatory convert for Phase therapy is events. medical This with will people to XX I events despite I/II/III insulin a type meetings the that is achieving patients hypoglycemic the SAEs who management. In include hypoglycemic investigational differentiated of diabetes I/II/III and patients with medical awareness, we MHRA we or despite very X control study, reached severe

from remarkable that basis groundbreaking allogeneic to the diabetes, longer patients had for of compared of pivotal insulin months the and are end excited achieved duration diabetes we of space. To X included events our the the evaluation. with is than Safety September. more additional received ADA off this progressed most with presentation. were June this progress less severe independence I'll the regenerative gene a of curative data X provide and out hemoglobin form noteworthy data XX cut and we represented patients the an at primary program, gene-edited and recent in of type These trial the a therapy quick close type less first-ever hypoglycemic beginning points presented therapy VX-XXX data that suffering than recommended with severe first-ever were an after the and potentially form we the follow-up on cell the levels the X exogenous therapy development data in X XX-month of latest all is shared proud endpoint cell of had be achieved for secondary advancements CASGEVY, X approval for achieved tolerability by Also recap patients it EASD Specifically, regulatory particularly ADA elimination on therapy for these to the are cell year pivotal patients of We and primary discussions the as they ADA CRISPR/CasX this towards These EASD, AXc showed at presentation. they X%. follow-up consistent with insulin

VX-XXX or in Beyond proprietary VX-XXX VX-XXX, our need to + immunosuppressants. a the designed cell device program cells encapsulates the for eliminate device same

The full without a the between target and of enrolling trial, C at is in next patients. is the We Part dose stagger. stage, at dosing dose are target the with Part patients the which B, currently stagger full

B from expect of the data to Parts in and VX-XXX A We study XXXX. share

clinical in plus hit the a date small Pove a frequency subcutaneous action, Pove's administration of roles additional povetacicept once-monthly transformative mechanism of This high to the the the of promise povetacicept APRIL as recently and preclinical of potentially to of program. dosing milestones. now patients autoimmune data medicine B dual diseases. IgA the route for Moving which us cytokines, antagonist is in multiple Pove with play and pathogenesis diseases. and confidence with dual volume BAF cell-mediated B-cell-mediated inhibition give key nephropathy

Phase clinical to with on across in First, began of randomized that trial IgA study I'm double-blind placebo of pove globally. nephropathy. pleased versus of global is very screening, RAINIER and XX multiple of standard II care. with we enrollment milligrams pove a ongoing sites top dosing share The patients is study underway This trial the

trial could potential preplanned point a is evaluate interim certain analysis patients a number when the of preplanned accelerated the analysis approval in Importantly, this to reach a time XX-week U.S. designed interim support with proteinuria the

GFR. approval, through continue XXX will For study week assess the and full

Second, recent grams per remission American demonstrate III the shared with renal initiated UPCR XX% X UPCR accompanied out study negative on in of achievement Phase study potential Society patients function. RAINIER and reduction who participants best-in-class of Pove's of hematuria at that clinical data continue the was Meeting. stable to IgA at povetacicept a recently we of of X the renal supported nephropathy, These from mean as nephropathy XX X.X or with XX% with In received by were II than baseline less Nephrology positive data defined XX milligrams a IgA results by Phase weeks stable gram function. updated

We and approximately very these IgAN excited in including they patients are represent about the for XXX,XXX potential globally, U.S. the the patients results diagnosed

per UPCR achieved gram another disease from baseline On Third, targeted defined X affects mediated disease, XX% nephropathy proteinuria, partial of a emerging of in membranous data who resulted X pove patients and weeks. from UPCR, approved UPCR from at mean X These pove subcutaneously XX pove greater and than remission at therapies. X received that with the than in patients X.X data reduction from in every renal XX with B-cell less shared ASN, are reduction weeks, patients as we a primary XX,XXX grams baseline. treatment U.S. at in the XX% milligrams no clinical

RCTs antibodies, We most acute at well studies, pove first with Stuart XX, the The and pain the potential to presentation was regulatory look and at week as the recent suzetrigine well anti-PLAXRX completion XX% of tolerated moderate. XXXX. will time detail these strong suzetrigine. mean was addition, pain events trial now look mild Annual single-arm Society III Phase from positive were date PDUFA set. to adverse by feedback of for basis the data and is activity of disease a The and acute data Meeting. Across and submission And provide In launch January X programs biomarker this and acute on portfolio physician a we pove from XX forward a the safety with or the pove you readiness. form the in the of American gears Anesthesiology pain to forward updating more pain effectiveness review, of declined starting to very the interest of of the in of FDA the moderate-to-severe pain. the presented renal now regulatory approval There review launch the of progress. Shifting were that acute patients. underway

being is review, pain studied in its advancing the development portfolio with assets the additional post progresses and study I with next-generation regulatory inhibitor through clinical in a pain also II Phase through Phase suzetrigine formulation. bunionectomy VX-XXX IV acute our a X the formulation As studies, NaVX.X study are oral

signal of PNP. make program progress pain, neuropathic be or pain, with to is Phase medicine III In prescription suzetrigine. our which PNP, continue in inhibitor with million strong program also pivotal one updates We for NaVX.X Americans peripheral used share NaVX.X a preclinical to that I'm have pleased alone inhibitors. In X seek an neuropathic type peripheral annually. pain combination estimated the for diabetic may or First, pain we very which X initiated

U.S. are from trial. Phase no for have on we another there have the radiculopathy more in II million to oral the lumbosacral than initiated study share X Americans this specifically end formulation results track completed by II in II with the of and also and study year. impacts which PNP of type VX-XXX this that Phase of remain Second, each LSR, medicines or the approved We year a we Phase

score for to type this a group evaluation an primary reminder, XX-week placebo. about the patients suzetrigine a so therapies. II and the of up brief the Phase update design support the efficient myotonic NPRS XX the magnitude that in milligrams effect in dystrophy data of today, which approved randomized either and or as Phase to is a baseline impacting program. dystrophy we trial no XXX,XXX muscular are there on patients the is discussion placebo trial As change the the end is group, potential a treatment U.S. as prevalent on DMX DMX pipeline XX-week or of size study. advancement. To III X XXX The the endpoint the may well type the the X most suzetrigine for appropriately of the The allows Europe within from of of wrap if

secondary trial SAD Phase Our the portion program share of Phase efficacy last study of in clinical on we In has the VX-XXX collecting both the data. safety am and muscle dosing, late MAD clinical I/II initiated the portion endpoint I year, change I/II the of of the completed is be biopsy. pleased initiated and that will accelerated, the index from the and safety have has the and primary baseline we study. MAD, splicing the endpoint to

look turn are a program highlights, We for update. about VX-XXX it for over potential providing of that as the it forward excited advances. With this the to I'll now and updates commercial progress R&D on review Stuart to and

Stuart Arbuckle

strong I'll U.S. patients Reshma. pain. Once modulators discussing of provide Thanks, taking in suzetrigine the some we and CF ongoing U.S. in the the again, by our then CASGEVY and as outside grew results and the begin for both we highlights launch eligible CF delivered of acute the further outlook in number CFTR

the reimbursed Notably, every in CF our and We have regulatory access KAFTRIO regulatory rapid now in made with has country progress approval EU. single reimbursement and therapies.

revenue In the with patients long around anticipate in medium- term, remain approved our in will more triple combination expect currently our CF. on sustained the over term. we launch therapy reaching We drive and of vanzacaftor growth the CF further medicines. We near focused growth short, eligible globe the

than potential the who have modulators. treatment new both will if do modulator drive who CFTR with X,XXX patients modulators be to term, development to VX-XXX more current this our discontinued those CF approved, more not the successful than X,XXX option, and the for in CFTR growth interest CF. CFTR a on additional And of longer of would expect one of respond We patients people

XX patients, treatment XX therapy our had QX launch are the in QX first is already X had our collections, sickle and their our of disease with noted momentum on cell onetime activations for by authorized across also launch approximately cell patient receive as to patient have regions. CASGEVY. and been least ATC at and from We've CASGEVY Two activation, thalassemia. just our we Now patients CASGEVY infusions transformative ATC activate to has way beta received patients now up and of on our physicians XX On turning approximately policymakers cell well total enthusiastically progress cell from important patients all to centers, markers collections. On and approximately globally. over commercial XX are now goal ADCs up collection, the have XX call. gathering

approval. Furthermore, was patient were Coverage agreement has than work a after been England very obstacle positive in less coverage for NHS NHS therapy On TDT to sickle also regulatory secure U.S. for access we discussions we access life-changing reached the in September months the for secure disease access a entered commercial landscape, to cell pleased payer significant with continue have in not patients. the to that the now to for this patients. And we into X with U.K.,

[ Canada. cell CASGEVY ] United QX Arab front by make exciting quarter this We on Switzerland with Emirates in in also the the to TDT the of end approvals the submissions regulatory we year. and both continue for sickle progress in anticipate East, the regulatory And and disease Middle in and

in were we in capacity. demand in with facility additional are confidence our a CASGEVY, September, to our for Lonza. attain And manufacturing are we pleased growing for partner, manufacturing approval patient third investing for we Given CASGEVY seeing the

focused to are a for the CASGEVY. We on finish year strong

remain multibillion-dollar we We that of helps more stakeholders, to world. and the opportunity patients, value represents governments levels recognition among CASGEVY physicians, see confident and patients and other and it view continue around our CASGEVY high more interest of the a in as

are momentum and months forward into potential in U.S. launch. a suzetrigine carry we approximately as built acute we PDUFA from now for XXXX. Shifting pain look our transformational X this will foundation strong date therapy to the have it We and to

existing providers franchise Pain challenges options. current across the effective therapies, by trained, Our doing another acute use need care treatment management treating effects health will commissioned. the more well-tolerated developing for potential for in underscored U.S. unmet and note disorder that multibillion-dollar moderate-to-severe are risk addiction pain who for of surveyed survey This providers significant use opioid pain than are we hired therapy when some the have the launch XX% was has over seek Vertex. Despite The and high the nationwide treatment. Among people of field ready. in a for patients U.S. conditions, survey expressed million for for and only opioids pain that and to by Survey concerns provide suzetrigine of we so, believe in with XXX we I'd a the the State the build year chronic opportunity and fully patients for pain. each recent to recognize patients XX and responses transformative potential and highlighted the moderate-to-severe findings, we And teams option prescription acute the X,XXX to contrast held is many included acute notable a view long-term of opioid side providers for a

of that And if of reported opioids. The would findings well surrounding eager gap use. new Additionally, our again. ASA for Meeting, indicated to option risk statistics they recent survey XX% therapeutic belief fill the the to results treat adequately. request providers with were X/X and pain opioid patients option Phase State at they similar side given of physicians ASA sentiments nearly are our surprising not a Survey a of the at existing III views and were pain received, where Pain that expressed of limits their between confirming to the NSAIDs in Annual acute are in pain anesthesiologists and the tragic ability related acute therapies effects specialists These conversations our experience non-opioid

is develop prolonged disorder have of opioids spend Overall, health opioid an the the the billion For stubbornly will attributable patients use on use, with for health of pain $XX and treated billion to care XX% remains prescribed acute crisis the of including are cost initially this $XXX care high opioid estimated initially billion year. $XX within billion to will opioid $XX of use per to disorder XX,XXX instance, at opioid go approximately costs first costs, on that for opioid pain. acute year. an

well. change sentiments the are directly survey hospital conversations same These are well are payer early working in We the goal information targeted formulary highlight to hearing accelerate we and as pre-approval need engaging both our our decisions going as formulary decision-makers. conversations fundamentally is IDN treated. exchange payers is with by unmet and and coverage risk way to the pain to quantify suzetrigine's benefit and profile. Ultimately,

given suzetrigine focus patients access commercial experience securing for a in national critical treating initiatives for key secure made of initiative XXXX to that prescribed rapid in decision patients key broad Our nature we retail access for investing physicians. strategic acute This ensure is our is the have the enable work and distribution. smooth seamless Therefore, time-bound invest to to suzetrigine. pain. One and is

support receive Another time medicine them. Together, suzetrigine the the of that benefit elements long of plan ahead for demand expected patients acute these the assistance co-pay enable who will for base that will strong access is patient of and financial initiative us to will payer patients the program physician think are their typical lines. and is for launch patients the term. and prescribed pain enable given right will of build prescribers creation This that the they strong from to a programs physicians coverage

We pain, with formulations, acute oral Our pipeline, potential to transformational of with potential NaVX.Xs, including value the combination moderate-to-severe significant deliver goal beginning suzetrigine on post to neuropathic approval maximize early in forward look in to peripheral XXXX. pain IV is starting innovations in VX-XXX, pain the suzetrigine and the our therapies. patients vision this of long-term and

can given well to to through Act, PAIN the hospital proposed cosponsored non-opioid Part NOPAIN members co-pays goes as branded used D of to XX alternatives into from the center oral includes novel see surgery requirements opioids by now we and be Act step Furthermore, is and for appeal Finally, as prohibit which both when Medicare in parties, logical an to first of a payment continue on on add-on January ambulatory front. used Congress outpatient equalize non-opioids generic XXXX before effect The momentum branded settings. the for its patients. policy the

guidelines all legislation X/X never Over good non-opioid states state-regulated use Furthermore, non-opioid been encourage momentum. alternatives. X/X there or have states more has XX be all of patients Medicaid require are to a plans. To Additionally, have approximately to and considering to to limiting is and/or equal ensure opioid conclude, of exciting states Vertex. that at options access time there prescribers in consider

the continue launch drive next Europe We new CASGEVY with in early CF with East. year. in sustained commercial and a we and And of U.S., of to growth the vanzacaftor exciting diversification the the of triple era launch in ongoing are the Middle anticipated

while pipeline. I'll call preparations Phase in We mid-stage suzetrigine are looking in acute executing for even turn to financials. multiple and Charlie programs pain forward in over development on the review more now our and to to the launch III early the

Charles Wagner

performance growth Vertex's Stuart. profile. attractive Thanks, once our and again demonstrate results strong QX excellent consistent

the U.S. X% growth in XXXX XX% outside quarter in XX% represents Third with U.S. and to in third revenue revenue of million billion first U.S. comparable $X.XX Reported billion, XXXX, dosed X-month with grew the revenue the patient XX% quarter the growth $X XX% XX% the growth of growth Year-to-date CASGEVY. included period from U.S. outside strong and including $X.X over year-over-year the

non-GAAP Phase year-over-year R&D acquired the partially in million trials the our clinical compared clinical Sciences 'XX. expenses and R&D expenses were the including offset XXXX. R&D QX absorption $XX of to include III R&D and $XXX billion $X.XX advancement increased third R&D compared ongoing by the expense to in work million in of prior in of CASGEVY pove force advancement billion third non-GAAP field the studies expenses specifically, XXXX XXXX increased result QX investment transition studies, investments as inventory including reported operating the of certain and Alpine portfolio, of The non-GAAP R&D compared in TXD. QX non-GAAP following in to successes. Within combined the Phase expenses ongoing expenses broad launch costs onboarding III the neuropathic and X% organization, of inaxaplin reflecting of in in $X.XX from non-GAAP of pain programs charges a COGS step-up multiple VX-XXX XX% We multiple $XXX Immune suzetrigine, in QX quarter expenses AIPR&D full year, including QX operating 'XX. commercial million in and peripheral in pain. our $X.XX SG&A income AIPR&D $XXX versus II SG&A On activities QX XXXX $XX and reflects acute the 'XX QX primarily million sequential of of and Phase front, to quarter for for billion suzetrigine million acute

Our the non-GAAP billion cash quarter repurchase We XX.X%, in million XXX,XXX over 'XX. shares. reported over to X.X cash in QX year-to-date, we deployed the tax have with was $XXX repurchase for in ended over shares. million million the rate and quarter deployed third XX.X% the in quarter We line to And with $XXX $XX.X of investments.

Now guidance. switching to

midpoint $XX.X representing our total billion remaining $XX.X XXXX range we sight X to of exchange at revenue a revenue current rates. to We revenue billion, strong have guidance, are product of line raising quarter XX% the with in at year, a the growth

billion For Vertex last R&D non-GAAP call. combined SG&A operating $X.X guidance earnings from range continues expenses, billion the unchanged $X.X guidance expenses, provided to to which and is on a our be of in our

expect second the continue recorded the For asset to quarter. the approximately acquired Alpine $X.X year, IPR&D, acquisition for billion we charge including in

was we charge full not XXXX XXXX QX XX%. the AIPR&D tax Alpine expect non-GAAP year for tax the purposes, of rate that deductible approximately Given

of nondeductible range year the to remained AIPR&D the non-GAAP XX% would charge, of XX%. impact have rate XXXX tax our in Alpine full effective from underlying Aside the

XXXX, rate to be As regulatory launch period. Phase earlier-stage III Slide and pipeline are result, our progress I'll on and XX% quarter on updating to our advanced detailed as important Q&A a CASGEVY other of look four expected yet for to our early forward XX%. In now in delivered multiple anticipated tax revenue results areas develop approvals fourth is the to the growth, XX. We also again mid- in excellent non-GAAP closing, continued prepared disease progress our we posted you anticipated Vertex on calls, continue studies to be in progressed begin Susie and milestones ask to and future pipeline. These double-digit

Operator

first will [Operator from question Instructions] Jessica the with Fye JPMorgan. And come

Jessica Fye

povetacicept product, the Congrats you you you're you be talk to portion order monthly the trial, bridging study? for would involves trial that get RAINIER launch to but need that we this dosing. Do targeting approved accomplished at at work human I at-home believe at an some home Can post-dose an to noticed all profile need of on And the for maybe extension ASN of about home the so during Phase with trial like dosing a do monitoring. in the product run could open-label delivery? could factor that III

Reshma Kewalramani

in that and of at B-cell-mediated a course, nephropathy. not Jeff, Reshma. results kind question. the let as separate is were about me hematuria related much take emerging membranous the This but then, very excited proteinuria, called Thanks the condition only results but that very GFR showed on Yes, results for we the words. on we ASN well as results the

biologics there all of and health and interactions planning have had at-home You're an for in are expected would have regulatory be expect right, care as in approval. ready protocol, is to be professional office, fully commercial the administered and injection in for and we development, to current is a that's povetacicept monthly the our by delivered conventional

place, yes, we So volume plans expect and administration. monthly, It at all those for small are in will be fully that subcutaneous home, commercialization.

Operator

Sachs. Richter from next will Goldman The question with come Salveen

Salveen Richter

the see here. to nice It's progress

but For powered versus LSR, and drug defining II baseline, population in just suzetrigine for this you recognizing frame you in just if III. Vertex of score, confidence what And on could kind for in the versus that front your Phase trial? help patient also could the help Separately, this delta the to the you're us placebo NPRS decide Phase upcoming data is this not order understand looking to move into and forward the for

Reshma Kewalramani

Salveen, thing. close we Sure very results. having Yes. the are to

results prepared us year. themselves. able before remarks, I the the I speak fully do to them the And of let expect in for said the for I'll So end as be to share

we're Obviously, the to of we safety do terms profile. framing would study things. and I what looking gain In for want study, three a good from the say

But to we're efficacy. So your I at the think really pertains looking question safety.

sheer from the of We measurement that's looking III study. that it's power to looking measure these We're to We're number so placebo XXX the and We're have available I II that Phase One on of be await you also baseline to predecessor building as significant. because treatment within we need had directly we about, sure sure make DPN acute VX-XXX Phase sites pain, out, patients, effect. And Same So baseline able who NPRS as is to the XXX, III, the that pointed was for of body is that for and results in effect which appropriately the NPRS the the evidence, Phase group the LSR molecule not that statistically the thing placebo be patients. the clinical might for weeks. in patient and on magnitude to were III action. really which II were of the asked from treatment data as lumbosacral exam, is trial, the way to change the through you trained that the did effects physical what magnitude the from to the be patients the designed trial, the that efficacy, that comes these really placebo one number assuming of the is score, then study we've to three mechanism for a but carefully I that we make not versus rightfully effect. confidence, LSR the appropriately we places. and study the looking acute selection best of Phase the done treatment selection DPN for XX of what Two high thing magnitude, the arm. we VX-XXX. to identify radiculopathy. And that selected magnitude before, the And do size of to is confidence can LSR regard positive. With think in through said only II Phase now Phase intend score,

have including high on patient factors, those three based I confidence So the of all selection.

Operator

from will question Morgan with Next come Flynn Terence Stanley.

Chun Yu

for on Chris This is Terence.

make comment to are on the that won't but directly Susie. considering its We the decisions question you can inputs on pricing, on provide Just you as you that you one drug? price some know how of

Reshma Kewalramani

Absolutely. Stuart?

Stuart Arbuckle

we're at our believe the of it taking all clinical as all be we medicines, into to the our level, is same the that driven much but the need for and an solving approval And and suzetrigine into making those do that account to both Chris, also closer at pricing of decision account time. level, a delivers. going inputs benefit by individual we'll societal considerations patient and importantly, Yes, be unmet of we taking level always that's

Operator

Your next Bansal come Fargo. with Mohit will Wells question from

Mohit Bansal

would ask another question on I LSR.

in confirm at also, look action, compare Lyrica? versus parts. So neuropathic that? if the some just you the looked pain. versus Can look And look pain. at Lyrica of when -- subgroup neuropathic you so XXX you Lyrica's you that? can if have you to in Even at specifically, some question contrast at of mechanism how two VX-XXX pain One, people is you seem analysis LSR-related it direct and impact neuropathic in where does confirm Can

Reshma Kewalramani

Sure.

asked So questions really gabapentin Mohit, or good you gabapentinoids. about some

example versus of that is an suzetrigine. Lyrica So

action mechanism first. of The mechanisms different. of tackle action Let's completely are

or what it in signal that for peripheral VX-XXX the we the that or molecule here same do with are neuropathic in otherwise. The were as pain are confidence acts acute LSR on propagated that wait very channels. are when the results called specifically is pain because results by gabapentinoids nervous antiepileptic the waiting suzetrigine system. anything action, medicines, specific of that pain, And pain way works, and are mechanism as specifically doesn't way discovered of for of those really the based we the and they originally by feel fibers, developed paid only channels So have for the to That's NaVX.X results way found NAV they action, being again, work. sea on potential frankly, of these confidence mechanism the because the do DPN in central the nervous the action is work we is how medicines reason system. they is and It depress -- were what

of know, and on positive they And to had you where they gabapentin peripheral and that's diabetic I as indicated results. the neuropathy. mechanism of positive noise the level can the So high Lyrica have results. action. On are speak did for have gabapentinoids

have not positive did results they However, LSR. for

signal, have won't that U.S. Now possible been approved I subgroups could some the studies is is entirely that. in some it in you that not tell I or on in but there will comment some LSR. it for

Operator

Next question Ahmad Tazeen from with will come Bank of America.

Tazeen Ahmad

they read-through for we to I Orion is should was try from the molecule then mechanism similarity have of discontinued as a pursuing, other in we it's read-throughs a of due of which forward want a ODM-XXX chronic believe, mean And moving as about any ask Corporation. regardless press reading pain recently that there to interpretation was data news kind in therapeutic was to secondly, results narrow I curious to did acute well example, LSR, the next to NaVX.X you're out not what given said As I to release and think competitor year. both whatever could the the and studies, think I about as LSR make announced for shows window. indications that if the from that action.

Reshma Kewalramani

been discover Tazeen, of NaVX.X run because the people companies targets targeting challenges the the with target or not through you X.X similar, of coming me that transmission won't worked in are it tackle molecules I'm all. as it has that molecule. let's for reason doesn't have if the start challenge try about that years, was me, many I ourselves. And thing. not doesn't Sure But discontinued question let -- to many, on that is NaVX.X, people decades, and directly many and including the have on the long and me many, surprise say surprise I NaVX.X comment it take reason molecule at call they the pain conditions tried in The is and drugs say been been which specific is so into channels challenge X.X, surprised serious NaVX.X have has NavX.X, that NaVX.X I why the specificity, to many up to people grail with with pain. holy potential it asked

the is more peripheral neuropathic Well, wouldn't channel we to the DPN. is data the our types III the I for any else. I'll very are fold end is my already DPN enrollment how completed Phase there screening, III our anything in is that's that prepared about regulatory We your development. very, said well on as tackle of the data relate It see DPN pain. remarks, way XX,XXX interactions. commonality XXX terms should Our question of LSR same But LSR Phase reason versus the molecule any We've expect in think both trial, molecule NaVX.X meetings. in We've we and and -- in second don't no, to II I about Phase of already specific I had changes. specific. dosing are versus

Operator

from with Jefferies. Michael Next come question will Yee

Michael Yee

pipeline two We had questions.

We second I the understand are I which significant well, is you just or wanted if potency. is than there benefit idea for limited. excited to the XXX as PK recall with XXX have about a the don't NaVX.X. Because compound other exposure

So think had plenty you've some on already results. and I getting that drug good board, you're of pretty

other XXX on to more if despite the potent wanted clinic. significant -- properties the being would just get better I channel So in understand NaVX.X that

too seeing are that just on X-year believe environment IgAN, do the know is interim and talked you the question just a I Our in could we been X-year much you us what was positive what actually you on given simple think data? is but hasn't probably which if -- regulatory could about wondering file APOLX, one that UPCR, here, said the second I on remind you you've on

Reshma Kewalramani

thing. Sure Yes.

who Let so. because CF, be to me humanly single asset do that committed are do better, know, just across has XXX. Michael, is portfolio, entire as to our possible like and did lead we a tackle every is follow-on it you if asset, we in the ones

XXX in to be better, innovation. do can do we we're if of it. And that vein purpose going one who of So ones serial the is

The be seek broad IV XXX VX-XXX cannot to is be we second specifically in formulation, pain an can. really into the space. reason formulated

So developing with it's and/or XXX. the is program. XXX. XXX, good potential program like be XXX That's whether the that's that is progress NaVX.X, other And development. we're reason we're the But second to NaVX.X best used molecules making as progresses, NaVX.X third we'd that's reason for with that still a in third whether identify preclinical reason our to

disease. the continuing enrollment this are could APOLX-mediated kidney program. the So we is the Phase then question the in for is program used combination This or the inaxaplin, we best have on with NaVX.X with pairing a most be program III to in moving alone in program, optionality and NaVX.X. And which of in this development,

in II the diseases. end our question Phase environment. regulator interesting really of renal of homogeneous particularly It about in meeting our in regulatory seem a does the with We kidney here evolving to right asked be medicine, front some time eyes proteinuric You ago. had

certain for the agreements recent in continue to whether So in terrific be before other time what the in FSGS can read X-year more number on at a itself accelerated said the an to of potential endpoint and see discussions discussions it's our patients proteinuric endpoint slope. But and we've diseases. of proteinuria on with GFR approval a pertain with point kidney

Operator

with Your Seigerman come Capital from BMO next Markets. Evan question will

Evan Seigerman

full dose the optimize do about when process, you you What And What do expect you that a what you commercial you CASGEVY to to went now learned hope further? go through administration? can the the had So patient? patient discuss next well?

Reshma Kewalramani

Yes. Sure thing.

me Stuart, over turn Evan. to that Let

Stuart Arbuckle

Yes, Evan.

think an in on real learnings post much of it learned a kind QX what call learned treatment to significant know the for lifetime And know physician, to embark a future benefit. patient there make enthusiasm is the you the I potential lengthy lot, very a we as that I there always but we've is a awful and commercializing when we went this I out we that that medicine on you think journey, that covered decision or but big that as for is actually into approval, because for our do world that of our reinforced we've the were. were particularly patient is there for this

patients that that discussions for the process disruption for scheduling part obviously, actual a number process journey. a is infusion to and of -- a the those learned to end when patient on because physicians at begins, time to embark it of the of decision we've collection significant includes big being that So is And is the And lot have an and that CASGEVY the cell entails that that obviously, discussions before life. the a want process weeks. includes beginning but of the anybody's at inpatient discussions to also taking

into authorized final potential in treatment the something is And able also think is to to in the step sign guess are is that learned they're to And is last things an reimbursement up not access seen in in, the that U.S. when we've I'd we've so approved U.K. patients. been that, patients was couple learned the their to to out translated we've the CASGEVY and to normal scientific has centers the enthusiasm they that initiating. are, thing which TDT. the they instance, reimbursement of around we've about their to patients. And showed secure be represents their provide barrier able carefully say product that life where that that to really And be able very excitement the payers through for need initially to to want time to take the provide seen for the policymakers to advance from other that CASGEVY to this process. I that here go we've for very when agreements a keen going medicine

disease. in have in said access instance, we Italy. has a early prevalence for Saudi is particularly We programs, before, Arabia, of high as have good also, And access we've which

and I we So learn patients, we continue say forward. things to and physicians all as learn elements, policymakers payers on we'll and move

Operator

Next from question Phil with Nadeau TD will Cowen. come

Philip Nadeau

initiatives, in it you mainly strategic of going? terms further well Does you those Congrats mentioned the strategic I contracting also one on initiatives for are also initiatives question a Or prepared strategic underway. example non-opioid co-pay think commercializations. of on are is Could then there other discussions the assistance underway. programs. And gave as that Stuart new in the that insurance remarks you options. for in are need the assistance? suzetrigine's characterize the progress. the co-pay then And how And discussions, understand on Stuart, are contracting

Stuart Arbuckle

thanks question. Phil, the Yes, for

literally of option, medicine a There has well. the new profile has, that So going not the need, very the surprisingly. enthusiasm appreciation a I of which say for payers that conversations suzetrigine are novel, non-opioid kind benefit decades. for would the there unmet high for is a is risk And with lot treatment degree first of

it, So patients for surprisingly, from not many the level enthusiasm are aware acute of of suffer how high pain United in payers just States.

And so through budget they way their thinking impact. are

those get can in as early want and make our access would possible I as reimbursed that use. sure to and patients like is to the medicine discussions. But we focus, very close they we're access physicians there to that So to approval, that to make can we think so sure have that

those are would think are important, discussions I I of say really, going very to couple So a pointed initiatives well. I that really Evan.

approval going often we through have groups. And choices. anticipating access some having payers two You know there picked But are between reimbursed accelerate some normal that whilst to work a up their them. trying are really on the lag we there have for is be as there, that. well, discussions to very lag patient will obviously, we're process. one of And you and

seamless enthusiasm to made hasn't we I share pointed out of to pain I who other plan even can want a that is retail make if yet wait. to area final one to try a are would patients patients physicians We the suzetrigine. can't experience from that decision. The with a chosen acute access access have We accept final therefore, up plans you problem to won't and of reimbursed lack don't kind on sorry, can policies. a gap, be can there's And so going a Or and patients working and some get be we've and are coming their going it. You know -- reimbursed access. that sure want lot doing distribution. in make there's the to whilst we try solve with solve which and you, Obviously, everything

suzetrigine, for at so dispense retail that suzetrigine turn needs to there so the up can they on shelf And it. pharmacy be prescription pharmacist with when the a that's a

a of other focused approval of is as distribution again, the are of ensuring retail that possible as suzetrigine, there's lot we time can. spending one on so national to And we areas close as

on. working that's couple that of are So a we areas

Operator

will Guggenheim Partners. Next question come Chatterjee Debjit with from

Debjit Chattopadhyay

Which on inaxaplin. is

we traction recent when How be the rates enrollment big additionally, completion? Vertex expect franchise? the compliance historically And inaxaplin for the could low in Given should antihypertensive? given growing renal

Reshma Kewalramani

it to do Obviously, but to the And second to of is faster which accelerated at to I to it of I'm opportunity a to the remind the also leave is the enrollment point. Stuart, given you opportunity on approval. to Stuart it's time aware, this for it's the you get I'll on will is commercial regard comment overall for With want large enrollment point haven't there going little enrollment is enrollment to us a enrollments and bit the that inaxaplin, be a two points Debjit Debjit, for be for here. here. guidance turn get that we to for to completion, Debjit, haven't now. over Reshma. we enrollment the completion and there's then accelerated time just out, growing study. number, renal commercial one the shared, sense a give full going enrollment of franchise subset to that,

Stuart Arbuckle

Yes.

approximately so here are So we in U.S. The XXX,XXX commercial of United terms AMKD in is patients with which between vast States. and the that know there majority living Europe. or the potential, of

on is the first which we disease So of significant treated actually if underlying there these very, cause the patients. is deliver a very commercial for can product, opportunity

at patients XXX,XXX and the about years of the was living or are disease. fact discovered estimated about approximately that point to are there in with not gene say time, this I XX XXX,XXX ago. the so undiagnosed or only Now be genotyped given Many them

very And new so this is disease. a

require in terms initiatives think multibillion-dollar a I say, it. ahead in are certainly as we like amount that. XXX,XXX is and of increases obviously, and supporting total therefore, we in explant to large awareness, So already over diagnosis and going patients, a potential, this has of disease genotyping, But of opportunity

Operator

from with Fitzgerald. Cantor will The next question Olivia Brayer come

Olivia Brayer

move separation at into in few for placebo initial a weeks to faster on wanted what VX-XXX What's up a follow I weeks? moving what the forward, XX end a XX a delta first versus LSR. I what the is Or of take enough it if I'm would to asking, treatment actually kind of you the Do it registrational to placebo be need forward. certain makes signal sense. bar guess those internal on X versus arm shows the actually program? to see enough would at that if to curves right, weeks, together of signal be were come

Reshma Kewalramani

what we're Reshma. arm understand we're treatment Yes. really understand for. the effect the is in and We're placebo what treatment to I Olivia, magnitude best way earlier, XXX is I said effect the looking looking safety. I this the looking good question. think, do the of arm. of in magnitude the for the for the And

effect, You're we're we right point score, treatment that when so We're appropriately to of consideration to curve we separate, going would of the that to very is we the that do want assuming to things all going out the look at are details see point that can expect that Phase all be there's of to at key NPRS size the to the III supportive. give the the going us of to subgroups. look you magnitude going do. The we're to trial, data

and is -- use was who asked then. the moment sensitive talk the And that it As XX,XXX. else. anything much said making talk I very have data, we about I about exactly correct what further III then manner go to can this of could it is have to I we'll Mohit, said I'll decision our we have a basis on the more earlier, it's able than think potential longer we been question certainly study. Olivia, to to just XX,XXX the the not NaVX.X question fold next Phase once too before, fulsome it to wait. that And on be in seem to

Operator

will Our ISI. last Ms. come question from today for Evercore Liisa with Bayko

Liisa Bayko

it that that of the this a kind to on was decide will I for you no be like be doc can you how had we was prior the on that kind Because and critical. seems And And drug getting it. can of call honed authorization confident that did that seems kind really of of no something that one in of really influence earlier, And case? what is indicated follow the would it like that? suzetrigine? factors curious kind and you the up seems are key that a anticipation are to Stuart and barriers

you how there. what are, you confident leverage curious So have

Reshma Kewalramani

and comment Stuart, both commercial front. the front maybe could doing you're the the other on work you policy that on

Stuart Arbuckle

Yes.

question. the for thanks Lisa, So

unmet that in preapproval the versus which controls the suzetrigine aren't sort get we reimbursed are discussions of which So provide that on management authorizations put topics about why be access. Obviously, already significant with that D being alternatives to use what risk of launched, and about to are talking we've some even referred level our of with of we talk profile generics thing controls. the we the required, exactly Obviously, of sort place, to plans side a is that way they for we conditions combined is to is beneficiaries. In need. Act, the but through controls things, compliant fact policy exchanges to the sensitivity the also to Medicare are have to the in looking PAIN providers to different are can types of prohibit a that if are other different our talking which to benefit might talking people are a that brands the seek our Part about. often able barrier there plans of one could are information that there is of other suzetrigine, we Obviously, particularly seen for utilization prior

step somebody before imagine a prescription I is certainly suzetrigine. imagine a decision. What a plan through So coverage requires for suzetrigine prior generic a couldn't which still a needing could opioid having I for made has before definitive patient to getting a to [indiscernible], instance, to have authorization somebody prior

going answer of because they ways. it your So really for the looking XXXX. is suzetrigine But there because used possible prior as as when it's to make for know very, very offs be different enthusiasm approved, patients in and we part I there's don't early question, one are Lisa, we physicians easy in to think hopefully broad to such

Operator

the our replay today. today's session call A by be XXX-XXXX as you Thank XXXXXXXX. replay question-and-answer attending dialing using you conclude for conference after your access (XXX) will XXX-XXXX shortly call today's code well our event presentation. (XXX) or will X X as for participation for concludes Thank today. of available This

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Vertex Pharmaceuticals (VRTX) 4 Nov 242024 Q3 Earnings call transcript