PTCT - Earnings call transcript Q2 2024

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Operator

Good day, and thank you for standing by. Welcome to the PTC Therapeutics Second Quarter 2024 Financial Results. [Operator Instructions] Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Ron Aldridge, Senior Director of Investor Relations.

Ron Aldridge

Good afternoon, and thank you for joining us today to discuss PTC Therapeutics' Second Quarter 2024 Corporate Update and Financial Results. our Officer, by today joined Chief Dr. Executive I'm statements Officer, our our Chief Klein; include Financial our Officer, Pauwels; Business Chief forward-looking will call and Matthew Strategy current Eric Kylie Pierre Gravier. Today's on based our Officer, expectations. Chief O'Keefe; moment forward-looking a slide the statements. website review Investor which contains posted the conjunction with to call, our information Relations take Please on in about our

adversely materially such that results could differ our and statements can subject actual forward-looking as from results Our statements and risks are to materially of affect business these operations.

well you risks annual For filings. SEC quarterly company's review description Exchange XX-K report as of a the as recent on report detailed and the applicable uncertainties, most company's with and we encourage and filed to other on Form XX-Q Form Securities the Commission

certain let pass non-GAAP and financial non-GAAP during Matt? are available earnings information GAAP Matthew CEO, Information in GAAP non-GAAP this over measures of use Klein. regarding to me will today's We the that, disclose call our to to our a of reconciliation call. release. With

Matthew Klein

you, of all an the on to progress I'm pleased to programs. development quarter for our results update and you second Thank call. share provide XXXX the joining financial thank our Ron, and

our XXXX focus As we have discussed, is on for execution.

the another had regulatory commercial first on-schedule across the great had highlights. Starting very submission have report busy that and of executed I'll the year, management half our a complete review we performance, quarter, I'm and effective As our performance. teams solid quarter and portfolio. with across the of pleased with expense. We operating revenue some to productive all second of We areas

Based this $XXX we solid XXXX performance, revenue are including $XXX $XXX Second to quarter DMD revenue consistent franchise of revenue to guidance on total million. updating million. million million, $XXX totaled

continued gene manufacturing the OpEx we Hopewell, manage the business of also our New Jersey. the effectively In operating previously will and maintain therapy guidance sale we quarter, also to our completed XXXX thus expenses strong provided. In revenue, addition to second in

this As part PTC cash of upfront received an million. $XX.X transaction, of payment

this the costs. I'll expenses team consideration, great cash the our in progress transaction to including with employee addition across made operations portfolio. associated reduces development has In now discuss our the facility,

open-label shared intake we able including new are important as well applications also patients the as levels. data for allowance treatment for subjects of we meaningful PKU. We the globe. The sepiapterin classical XX% recommended global July daily PKU in data their to clinically towards standard study Kylie to the to Phe all approximately to launch our the support launch. that evidence care the the of is PKU Japan preparation. In and tolerance as in protein sepiapterin support III of plan of Phe extension sepiapterin Phe the for ongoing resubmitted details study for our around still treatment more submitted This Translarna. NDA groups global while daily indicates marketing can the plans, as sepiapterin we beyond These increase significant age to NDA FDA. benefit benefit of last provide Brazil additional bring several a the step to sepiapterin authorization sub XXXX on we provide in from submit the study countries durability of maintaining Phase As potential severity NDA a week, includes significant well launch an PKU of and subtypes, the patients XXXX AFFINITY that tolerance. will and control efficacy planned as demonstrating data from Recent from of as as well sepiapterin

of treatment the PTCXXX patients. of the cells, benefit of which dose the with ] HTT of in of significant along PIVOT-HD XX-month levels end Huntington positive XXX reaching recorded The the population on well readout is mutant in CSF [ we mutant submission from data on in in results the ]. In Huntington's evidence [ interim Study the in dose-dependent with based blood as points the from data XX-month ITT shared of across Translarna results as discussed As the the disease Phase XX% protein XX-milligram blood study was second the also meaningful the at patients. lowering dose-dependent quarter, benefit FDA, similar level, the to key long-term demonstrated of lowering protein durable II

on months, score the measurements, disease XX the effect at favorable In cUHDRS including and total addition, key motor [indiscernible] scale. clinical

of development and of Importantly, for PTCXXX well the promising tolerated. the with promising, in Huntington's most biomarker not, PTCXXX therapy along favorable effect, and interim results safety if also to disease. most be clinical This position one safe demonstrated combination the as

the We FDA are preparing study a with of approval the request media to process for potential accelerated based in results. the discuss PIVOT-HD on

or study approval the and in symptomatic an In the We Phase of as who the disease to disease-modifying patients registration a of context worldwide which to on trial a addition, we need accelerated PTCXXX, to design therapy. the work have effective serve could XXX,XXX begun as trial. over look Huntington's III forward of PTCXXX confirmatory safe continuing a advance efficacy desperately

milestones. As on we of move into remain XXXX, and the half schedule for planned second clinical our regulatory we

of adult of and on scale upright a the significant submission the the trial. which pediatric submission along year. We disease mFARS end for patients process in we by NDA stability are the the be portion of and of on expect the with in to of rating confirmatory submit for the vatiquinone the from treatment evidence MOVE-FA young the study, open-label will findings This of based subscale ataxia, the treatment preparing benefit long-term Friedreich the

previously been for discussed, and of the sensitive pediatric patients loss portion scale ambulation. the young of stability and the most relevant have of upright risk ambulatory MFAR shown we predictive is be As adult and to of long-term has

has for a vatiquinone whom in including children there age FA Importantly, profile, approved under no safety therapies. also are strong XX

the top is cell be Utreloxastat compound share death ALS demonstrated targets specifically the of pathway from that schedule ALS utreloxastat results relevant the line fourth, registration-directed patients quarter. of on first for trial remain ALS to being oxidative pathology. to and to we a stress CardinALS Finally, in highly [indiscernible], developed in

the I'm Cardinals ALS changes recent study the landscape, to and our of now schedule the XXXX. I need utreloxastat of proud closing, of accomplished execution. address to positive patients. In have therapeutic could to enable ALS objectives the we to call the remainder results our remain in over unmet turn all our the Given on milestones of We significant Eric? continued from commercial and many set performance. Eric achieve discuss will for Kylie the team's

Eric Pauwels

Matt. Thanks,

million the our five across team products. in $XXX year, customer-facing had QX strong in a global revenue half Our first marketed of delivering

DMD a Our which during million. global solid franchise had quarter $XXX we delivered

as in to commercialize our continue in We Translarna further. geographic actively European expansion progress other to continues international markets markets and all

Translarna Our for Brazil, we signed closely of treatment order quarter, third commercial XX% as a with new with the as to working the and long the this delivered in Ministry order access ratification the in care in authorized, teams group order quarter. second the reexamination Health health providers with of to remainder are have purchase and processes. In delivered Europe to pending that ensure continue remains patients of the

this and we mentioned, the mutation in patients nonsense FDA the resubmitted important DMD pending bring NDA As treatment positioned U.S. to have team the to Translarna approval. is well Matt for

Now turning to Emflaza.

million. revenue Second quarter net $XX was

to the dispense health and specialty closely brand. work to pharmacies providers, continue payers care with We

key and patient personalized starts co-pays of and the pharmacies. with from revenue number continue patient to a of We and drivers maintaining significant are patients are which treatment, the benefits to shipments leveraging all services These patient providing timely new brand PTC year-to-date. on support Cares specialty for each programs, efforts for reinforce insurance Emflaza existing of

will progress Kylie? product our Kylie Now hand update current to it future of and I new over to launches. the

Kylie O'Keefe

Thanks, Eric.

global extremely about upcoming the of launch sepiapterin. excited We are

we adults As bringing to U.S., the Matt in step have filed mentioned, is PKU. important an with which and NDA sepiapterin children our in

well. Phase validation accordance less includes [ micromol versus where AFFINITY alongside subjects for [ treatment less data Europe data received XXX ongoing. from for control people In includes of to review which and therapies the also We've factor additional addition, Japan, drive XX% subjects U.S. levels. It launch which ] filing important with individual data for while planned of diet the AFFINITY of package maintaining the considerable launch. The countries liter. The differentiation the allowance been than preparations of and submissions liberalize the will supports unaffected several including EU still achieved payers XXXX, alike open-label are sepiapterin protein daily levels MAA the and and XXX than per fee value Further recommended achieve provides Brazil of patients ] uptake. able feed in our is global trial, sepiapterin ability very and liter, extension physicians, study, clear is per going in from an above are of being and and several age-adjusted the guidelines our III C years May, in current are in normalization and drivers for had to of we for subjects planning micromol intake XX% evidence

community. working understand the patients strong the the with with specialists to We PKU needs PKU relationship have closely dieticians a been metabolic pediatric geneticists, of and and build to

treatment have diseases, and are reach to the Unlike through managed PKU newborn their PKU screening habits them. of identified as a well-defined prescribers and rare understanding teams how well patients deep as Our PKU other prescribing by centers.

each and the center. key key leaders We treatment treating treatment have within these globally opinion mapped physicians centers and

In have established with an a XX in footprint we countries. addition, infrastructure global disease rare over

regulatory complexities well Our disease as the of in of the understand landscape in and country. experienced as each teams rare different the regions payer

targeted opportunity. will in reinforces $X launch this billion-plus enable All potential customers focus our and of to key have at highly global rapid a the on and us stakeholders belief and

priority to discussions health November target BLA access had to and as the care. well review, the continue XXXX. through cross-border date Europe accepted of treat In Launch underway. patients XX, preparations that and granted May, Globally, access reimbursement FDA announced we in Turning the with in the U.S. and regulatory action we are advance Upstaza.

globally. addition, and are regulatory secured In for we and in filings approvals additional preparing approval Taiwan

in Moving to and TEGSEDI LATAM. WAYLIVRA

make region. across progress in with across and franchises these We identified good continue patients growth to treated both the

the continues in quarter. was expansion second reimbursement which which a TEGSEDI approval geographical received Brazil, purchase of order for third delivering initiated of in in quarter, the following we we XX% with In remainder delivered Mexico, new anticipate TEGSEDI and we the have discussions. Our the recent group

quarter, purchase first second group the robust WAYLIVRA the year. XX% conclusion, of new in the half order. In a of delivered we coming for off Additionally,

million. are Matt $XXX total XXXX with $XXX a million to of revenue updating As guidance guidance mentioned, we our

as We now Pierre XXXX turn for have XXXX. I to set the a of launch geographies a our diversify in continue will prepare sepiapterin global and trajectory across as financial our to successful well update. for over and a portfolio strong for call to deliver Pierre?

Pierre Gravier

for the revenue the DMD to million this Thank you, share a Beginning press highlights million. of additional now Emflaza, financial the product second release line of for the second refer $XX million, $XX was issued net quarter franchise I'll quarter top details. of was with results. revenue in Please $XXX while including earnings million. product quarter of our net revenue Translarna Total revenue Kylie. XXXX. $XXX afternoon

Moving to Evrysdi.

Non-GAAP $XXX of revenue differentiated for for stock-based in the expenses Roche, XXXX, at options second global ] for June with company and prioritization equivalents quarter in high received. Cash, second December less year-over-year focus million quarter portfolio royalties noncash compensation Non-GAAP quarter XX, of the of continues was $X expense compared in excluding expense for to XXXX, compensation by million in million excluding in was for million programs. quarter cash cash its revenue XXXX. the was noncash XXXX, compared one $XX noncash PTC. $X.XX as achieved USD XXXX, $XX its XXXX, second Second strategic royalty and compensation to second marketable the PTC $XX Royalty in billion $XXX XXX XX, stock-based to million its expense. compensation the put amended exercised Pharma agreement million excluding million of quarter approximately R&D expense, R&D our noncash totaled reduction excluding potential to resulting stock-based the expense in $XXX of In second of million million of securities in as quarter, million compared $XX resources million its [ million of $XX reflects SG&A on $XXX $XX for exchange expense. The stock-based

over including to strategy to Our sepiapterin milestones sheet now Q&A. achieve turn resources to for PTC call on the will execute strong years, our our several launch. And next the and provides balance anticipated I operator over Operator? the with the the

Operator

first [Operator comes Cantor question Instructions] Kristen line Kluska the from Our Fitzgerald. of from

Kristen Kluska

a on great Congrats quarter.

that The of first was be out increase field, Obviously, you're it of but stood had an a NFL one one seems a neurodegeneration, Huntington's the ones drugs at to Obviously, lot seen data one But the shown other biomarker only been through I on thing we've of recently. worse. approach to seeing is the disease. prognostic looking have make it's therapy. in where not your

is question drug via sense NFL a is been of on do for targeting. important it then dangerous we've how So second, increases this a we getting really a when is safety? consideration have And physicians

Matthew Klein

for Kristen, question. the thanks very much

here is a disease-modifying both disease therapy goal a for with patients is the So therapy, safe. to that effective develop and Huntington's

the of substantiating And of is likely there's clinical Huntington therapeutic disease effective but Therefore, so of ensure So we lowering incredibly and that we the brain RUSI term caused one benefit. In for protein Huntington's Huntington agent It's lot was a slicing we to lowering sure whole protein making in really development whole The have it to that molecule about brain Huntington of the a the the it the of have makes the we When a disease, we'd literature -- a effect, result so Huntington Huntington cause to disease disease same selected safety of as brain, followed we're production protein the Huntington's disease. Huntington's development, the specific monogeneic importance. of mutant is the reducing it a oral time, by for strategy, of from toxic a safe of disease. established for scientific is that the and the both course, we're can doing involves target therapeutic expect goal sure desired a at that is all that disease. that Huntington that development lowering broad Huntington like the is gets development product important the in causing that playbook incredible protein well wanting is of talk mutant the protein. that to sense. [ and into safely. And to make gets is brain lowering a by PTCXXX, understood. disease regions because decrease ]

also these Huntington's of marker you thought marker period would fact are importantly, be a we're clinical benefit a pointed number hope clinical a it's one tell through seeing early we benefit study And motor to in desired protein so injury spikes, of we're way for asking that so this continue key that NFL having without treatment-related the that and cell that so as spikes. of having reason disease but therapy. lowering not therapy. that as patients. something So desired uHDRS over evidence of of could of tolerated spikes lowering safe therapies, reported, present some causing PIVOT-HD biomarker because that be we're that seeing disease-modifying well important has trial, seeing finding of observe we also disease of a in having say for the benefit of we're be importantly, And seen the we scales score safe And harm why, that we're incredibly to been importantly, on of at like to has an the We're time be been protein is may shorter is Huntington look we're safety far, is benefit we successful and when a that ourselves therapeutic then the we effect NFL nerve seeing in of NFL more as acknowledged in delivering more will NFL, markers and a seen can development clinical Huntington by PTCXXX a CSF. And blood. itself But as show us the therapeutic of evidence important safety. Are that any also total we harm? important we've it's causing out, to for really a incredibly

Kristen Kluska

And a then second ask if I question, PKU. just on may

uptake curve rare about disease. launches, disease on always we depending when different think think the I this is

think that first that take are X patients it, You for of So or really this you identified sense already the you've about standard good maybe, one don't call care. respond of have either how the don't where would a years?

Matthew Klein

now her talk how as for launch. we in absolutely. Kylie several Kylie let Yes, ramp successful the comments, about years And mentioned for preparing been I'd a to we've expect be.

Kylie O'Keefe

Thanks, Kristen. Yes, absolutely.

in I As going and prepared mentioned preparing this and as Matt well. of we've for number my a years for said remarks, now, been really been launch it's

physicians you noted, the of to as the relevant understanding this We community, as of well focus important to as very exist. the these excellence. And have unmet different are is and across where need disease close PKU treatment we're where the ancillary a the in all key also patients that medical centers prescribers

And in screening at identified you birth rare traditional your said, the through well-known treatment launch sense excellence. so and as centers this disease are not of patients newborn that is

And each that and move reimbursement. so we the our submissions expecting look able are that, of regulatory and and progress to a fast those markets particularly in with And early market fast then outside and U.S., market preparation, pricing in we're named coupled move patient programs and to with as well, we'll formal it's to while where reimbursement. uptake, with programs access quickly one has pricing U.S., our the expertise means that we

board, fast across at we're the rapid and looking a I So think relatively uptake.

Operator

line the next Our JPMorgan. Eric question Joseph of from comes of

Eric Joseph

couple us. questions A of from

little wondering by of First, in marketed on you top product a bit if unpack line -- sales anticipated bit what's can little terms unpack that PTC. guidance. a I'm for net

compound. And indication a merits also this of think on hope you the C how share left of you in whether get about do over with a of was in of finally, us Type designation Huntington's, therapy with I and with potential submission registration And the MOVE-FA for you line part something sense Type data for for that's breakthrough the Friedreich that FDA you B extension from have open-label meeting wondering the give Secondly, vatiquinone, on wondering on that Is Have when a PTCXXX view. then could or something you meeting study? a path. if pursue the to to to from additional to portion you what's follow-up ataxia? that plan reviewed that intend kind believe Street? of

Matthew Klein

Eric. order, first. Thanks for reverse the third go one in Might so questions, the

is So coming NDA vatiquinone the along.

We the expect before the year. happening the of end submission

XX-week extension on to the measures data previously number going had At my as significant from As relevant was significant to with repeat analysis [indiscernible] going open-label well that over that's study a as history the we the That stability and upright placebo-controlled both period. in analysis. well of on effects of based earlier I other MOVE-FA mentioned a had in done from FDA's ago where MOVE-FA patients. We we years data comments, XX-month request, that long-term from ataxia adult showing as as be published disease data. long-term newer match are long-term Friedreich benefit natural the data,

analysis accelerated submission. that get open-label with on studies agency go together then data regulatory with results pre-NDA discussion we second results still discuss the surrogate the share Those collected. be alike And We a when move MOVE-FA of us clinical to do FDA be the efficacy and the that approval endpoint we study with accelerated content is forward trials, to to PTCXXX long-term for we've we'll interaction the be NDA. On the a and [indiscernible] approval plan from into whole will so or One be to potential strategy analysis. We're provided the Huntington as could the for structure confirmatory trial. endpoint and for collected on analysis shortly, we'll to could waiting a a FDA, to in with pathway, completed had for as from data plan and the data we that to of be predict allow and the and fronts. long-term about, it certainly our a question PIVOT as ] likely will the will to regarding two to physical are potential HD, able always benefit that access regarding context data [ review then for have the the separate which -- moving need do those do a lowering extension we we registration interim final for our collection their meeting your also serve we path, forward the talked be agency the and package to

course, efficacy to key expect interactions alignment for the both and interactions pathways We this an looking have of around to for the endpoint in fast surrogate now track strategy including breakthrough, priority potential the as half other as second our is the of the on but that right exist, have of those year. We're at well pathways endpoint also, study.

of think In the that, about on and detail over turn me terms we let and Kylie. more to that revenue unpacking components how

Kylie O'Keefe

Yes. Matt. Thanks, Perfect.

low-side to Eric, while this to maintain that the previous for So little it products. unpacking updated to $XXX to to fact PTC-marketed updating CHMP the from we're we've XXXX. that happy I your also And one guidance. In is of able think question been is around this case And and originally, throughout around coming $XXX is the negative revenue question the has million majority really that, onwards. that driven had from scenario PTC, guidance we're a out by regards for $XXX to back, the driven things would just of has revenue products, European guidance Translarna EC revenue of million, $XXX PTC the opinion a it on QX not million that the come the from million bit, with the marketed substantial upside bouncing your And this because of driven is the XXXX. this assumed share the

Operator

of from comes from Shi Jefferies. next Our the Kelly question line

Dingding Shi

Translarna how steps, line of new what Translarna the after status are the a have FDA? NDA next approval? you most of share you in regulatory also U.S. U.S., of Europe. share other of last thinking Could the on front with you curious the to you time any to discussion -- update the regulatory submission be and are what's the the agency update in if risk And And able So the would on newer about current color after call?

Matthew Klein

the Thanks questions. Kelly, very for much,

On Translarna from front, the the is notification. And of the a calendar submitted early XXX in you that We typically -- our European as is day for did for request reexamination. process reexamination we July. know, day set

is XX And days is submit to have reexamination, reexamination receive we days what that known the opinion. submission, XX our for supporting as We grounds which document an then within requests. updated our after briefing would

that on still time benefit we're our been grounds component the And review the most line, process. able awaiting and ], reexamination made the we're focusing So reexamination also that recent assignment the is of which evidence some process. Study for of were of the during address are to new rapporteurs we've another [ XXX. on in concerns And in

in supported to reliability we the of concerns, therefore, the to we're is it to loss able can And protection and long evidence Translarna reliably address of around and the well with treatment term. able say strong can so analysis ambulation multiyear particularly of the benefit attribute show that

to X Turning the submission regulatory FDA. NDA July. resubmission takes typically at resubmitted the of to the months end from action. process The We

within they're We the not the able filing. as of to expect submission to hear agency to accept where from days XX

it's is since resubmission. So slightly time a different a lines it

only from we then the on significant the the had our package on Study XXX endpoints, to overall of in the primary the with is, walk confirmatory comments, the As is this of really in in mentioned ITT the we data meetings was the including of submission. be have but benefit previously benefit tongue will align FDA from [indiscernible] distance evidence what I'll of that Study evidence on that from point XXX population, with focused endpoint shared, the long-term evidence two FDA X-minute confirmed out on for as And and key North in the we other function not Star we test. And sessions all registry. separate I

what be reviewing with So package are to NDA data of analyses would additional on one, be those and the align needed package. things: the in to discussions and allow be terms what that two structure would would FDA really us the of

So the to matter agency's most to NDA review. will have questions submitting they believe be is we the feedback they been address all recent remaining that prior a able of Their any concerns. may believe to we've of us

DMD look boys with mutation forward need. for as review in always, the unmet to So we and the men look bringing [indiscernible] forward to to and significant process course, young U.S. of Translarna nonsense

Operator

participant from question the our to Blair. Our from from next of come Sami going Corwin is line William

Brooke Schuster Gray

This Brooke expect ALS you the We like expect could wondering more to color company conference is upcoming for if efficiency on this and were for event. at a medical Schuster trial bar Sami. you the if provide readout, for or you some readout be a

Matthew Klein

Sure. Thanks for the question, Brookes.

X:X that in the that and such the the that significance on been endpoint primary the an in study was in was talked being about of that is a it It's moving record done is positive the placebo-controlled kind an utreloxastat of that been treatment This leveraging ALSFRS we as study so endpoint, if of registration-direct support an X-week running patients. primary NDA patients. designed States. This would So we've be the products practically to strategy that rate that United is a that have of we other benefit. study group disease many trials ALS ALS We a can has so one analysis which, included in double-blind, previous is achieved we primary with ensure study baseline study, order we're the again, of made scale, statistical in submission a utreloxastat progression, learnings from tested rate the ALS endpoint CardinALS placebo. to study randomization designed to of determine a placebo-controlled X-month appropriate

here we line success endpoint define forward we to fourth have So allow primary quarter. us in that that the move with to statistically expect NDA said We would submission. as a significant then results top

public that results as we'll expect on top call, We share when we we a have typically data. do those line

Operator

question next from Our Capital Brian comes from line Markets. Abrahams of RBC the

Unknown Analyst

This for on Brian. ] is [ Joe

a do support to clinical you lowering data and think to the at best look So looking to you along guess biomarker translatability back accelerated when which Huntington's, other And your lines, and the these developers' HTT chance than collectively, at knockdown benefits I your shown individual have going has had levels approval? measures data? HTT at functional for levels to between correlation other

Matthew Klein

Thanks, Joe.

the shown the is is you number production for by potential correlate things, the named thus endpoint Huntington that's toxin to caused stands study first a clinical to lower to clinical study benefit Huntington benefit. And likely to demonstrate can And several Huntington a disorder reason models. the it's been is I basic and reported surrogate predict if the protein, clear lowering a Huntington in that's have only likely the marker they protein, benefit be you're lower all disease-causing you to range a to been to in protein, rationale benefit. a phenotypic with that's And with think on of reliably can a lowering. a Huntington disease in principles. of XX%, been extensively is preclinical just XX% mutation Huntington monogenetic that least if gene, it based of disease-causing at that's that associated really

nature sort which demonstrated ]. has [ of also There's been a that experiment mother epidemiologic study, on

almost for nuclear are in an progression. Huntington less you disease born in of about protein, polymorphism promoter and new gene make disease and If a delay a have XX% with the you slower the single Huntington XX-year onset

shown clinical strong to the benefit. And Huntington protein lowering is the So It's not only evidence that put Huntington in XX%. XX% and as mentioned is lowering Huntington is result that scientific lowering I that in range. protein protein. between it when package you likely together,

around was the test. We've Huntington are that of agency the lowering the you set, data seeing the collected now discussion type but support And not endpoints PIVOT-HD as dose-dependent in potential and clinical correlation accelerated a far in looked on the recall, specifically a CSF benefit thus with that clearly been small in use we're Huntington show we've blood, protein and move thus relatively collected has we dose-dependent when clinical approval far. at that to lowering that PIVOT-HD that's able benefit, at the readout data, protein we our that to really level the so that in to to be it's will data we associated we of evidence with

Operator

the Joseph Our Thome of TD next from question Cowen. of comes line

Joseph Thome

And PIVOT. a I first Is a progress. know there's then second, for on that data ALS one, set Maybe before of been with the to the what pivotal in see for you'd any guess in follow-up on Amylyx change do the more maybe ALS data waiting FDA there compound? on half from on we're given experience to a Huntington's, next year, the believe in data full launching I trial. Phase wants the the set the you that first like package Congrats just the III? anything see

Matthew Klein

data would Thanks have with for agency were ALS, what they consistent the endpoints had analyzing the closely and right and be in the Joe. study see the On ensure of want worked approval we designed with to that we the CardinALS to questions, that we that the terms package. would

here consistent point see primary views point, and certainly And one the and looking is so be as that of that the what second of our treatment to to package with scale might study plan, taken we in analysis component have is ALSFRS to effect. Therefore, not the have design, change their of the statistical all time study consistent designed only can likes understand quite at end see analysis of agency been. approval, the how their the we the to exactly really with variable terms would want on have deaths analysis and which approaching important incorporating for occur our continuous a say that type always but they what believe I that get advice is we primary and of the any also have the we're the something for in

timing, key going data make III. HD Phase to more PIVOT-HD at sure of on key questions terms designed we B, development questions asking then and right in In the terms Part seeing of Part drug different were A Look, we two time. to the and that question into parts, --

And of The it evidence protein saying target dose-dependent need The those yes milligrams lowering first XX see of of the were pharmacodynamic seeing to for the and at weeks doses efficacy? engagement, the were we and we exposure? see. Are to evidence effect, focused seeing want appropriate and seeing the are are we answers would that of to Huntington yes. know to to on be the pharmacology we CNS are magnitude likely we X XX

we study, asking Huntington's have As that, the over CNS for to starting we're therapy a okay, adequate having we CNS we're patients in able necessary the excellent know question, changes effects confidence the that study, levels and biomarkers the part to efficacy be there and to register of the we'll move disease. see with X-month we at us and second clinical dose into are scales time, pharmacodynamic the give exposure,

first readout movement when Huntington effect, yes. with at tolerability and and of and answer clinical durability the so scales of levels. patients, continued X- importantly, what needed data saw to the that again dose see of early was of safety in lowering, And then biomarker terms We we the had we group XX-milligram

of with we Phase for to us, gating really forward those III. So boxes checked all move

with get strategy, and the the why we're going that's data along continue study have the that get to we start on advance line and saying thus So Phase a study planning develop we're regulatory to endpoint based design, the to III far. [indiscernible] --

Operator

question next the comes Morgan Hung Stanley. Jeffrey from of from Our line

Unknown Analyst

comparison Translarna you dynamics overall how can opportunity the seen the market of provide patient about sort EU? and the thinking in you're just what NDA, U.S. on resubmitted in in color the to we've more Following

Matthew Klein

Translarna very we've, opportunity make and have Kylie, color significant is as mutation U.S. ready Yes, and available boys to to need opportunity to you tremendous long Translarna Huntington's think men a add much on in the resubmit Thank time, men DMD about, NDA with us. more question. launch? we've boys to want a young be sure. you a We bring young you to been do medical talked you for and for need. able in and to the to going how the some unmet And finally -- for U.S. the the to the be about

Kylie O'Keefe

much very Thanks question. absolutely. for Yes, the

there -- been targeted has therapies other high X for need. said, medical unmet of our no alongside throughout DMD the advocacy strong the embedded their patient up PTC set working infrastructure and Cares experience patient, community because high the a of from unmet for had Matt remains so groups the medical alongside working in this treatment it's patients need the has to physicians, has also start years And finish a support as So team over for to experience. nonsense DMD, mutation working

experience nonsense rapidly we addition from to where launch addition that DMD to for have this move of that, been us Emflaza, allows go to trials, In be And to of in that, our have we to will on patients. post decade with group understand Translarna quickly DMD patients mutation on clinical look the upon genotyped, thousands and to also have them put XXX, on approval. able that and been more very a commercial than a to on patients, and we'll have are still drug for through

perspective, from we rapid very So this uptake a a market space. expect dynamics in

Unknown Analyst

this And ] I'm Jeff. to is sorry, forgot I on [ mention Catherine for

Just received that Just a structure quick in the early physicians launch or curious. demand like as Have of any there? indicative might follow-up. might what feedback from you the U.S. be look

Kylie O'Keefe

Thanks, Catherine. Yes, absolutely.

also been about we've amount long in get Translarna, the could pull but feedback, PTC DMD struggled they've received obviously physicians, from have because patients been not. the closely over access there's world huge a physicians of of from they've space, working initiated from to patients a DMD with and to and known waiting why Translarna period those and the U.S. lot for the a time. to and have We all of And get very and patients to therapy the understand that access can

opportunity we around earlier, point so perspective, Europe. U.S. my fast patient reinforces the market landscape both And larger than the see the the and in dynamics pool that talked we the with and physician I And think market from just about uptake.

Operator

from of from the Gena comes The Wang next Barclays. line question

Huidong Wang

questions. I have two

increase Should will a question and Friedreich regarding launches underway. see And First, in meaningful XXXX in the ataxia. in you vatiquinone beyond? several we second SG&A have is

the when natural and with to do you data in So regarding plan investors? NDA XXXX share the late history data, that

Matthew Klein

Thanks for questions, Gena. the

have talked existing first question, to will only come. not -- the infrastructure but we've support the that the On about, therapies we as therapies global the commercial built potential infrastructure the

And so infrastructure. the have we

need to set, or the be proud them. where the therapies accomplishments if obviously We to approved, move to able several drug year, incredibly And quickly go, year. We're in board products. have to this team the in being a do patients ready of this. this those to these are we've the therapies desperately we proud all get have in quite we'll BLAs the we're already team's and to who potentially three fourth this expertise fact, across submitted situation launch And new applications

with is so in ready patients SG&A future. go And any near can therapies scale infrastructure expect built don't any convert new increases because we to the of and to and we number in that the

the to comparative. in data, used [indiscernible] FA that in being a history of terms in community terms data natural for That FAC the In history talked MOVE-FA. natural with a the mentioned, We've situation I completing of vatiquinone has we're model collection for on the registry. compared as about process done still history the will the of of lot the could and long-term database natural be a building regulatory fortunate job the the purposes.

patients analysis of of then, the analysis data first open in months course, relative a when available. And natural that be match several will previously conducted to will the those XX-month history analysis be comparison ago trial the are be history for look in doing treated the data XX we'll they will We MOVE-FA. -- years collected and adults, appropriate extension natural label data comparator long-term from we from the sharing having of and the forward to

Operator

question from Lebowitz Our from line of the next comes David Citi.

David Lebowitz

especially we'll convert would and additional year? start run those given you PKU. for assumptions the of potential next Given launches namely products, the be accounting that's have due Could to what expectations, us through operating spend, your

Matthew Klein

Sure.

for thanks the question. So

OpEx operating mentioned, is the I'm of [ me where let as a sure have First, talked now answers we as have about launches. to your and Coming of we the I we till anticipation not. expenses in convert, potential but be that needs if that -- that clarify XXXX, with up not level in at question or is ] and it

Operator

Joseph Partners. Our next Leerink the question comes from Schwartz of from line

Unknown Analyst

you is FDA that from the We've protein ] not fan couple potential approval for It's marker as heard of surrogate of in a a to [ sponsors Jenny accelerated a support other Huntington on you Joe. that potential guys might other about have thought do use? Huntington's. thinking you they think be that way? that Why Clearly, able And might guys biomarkers be disagree. to you surrogate anything

Matthew Klein

thinks the Yes. for speak believes. what not anyone for the Thank you FDA sure the could FDA or question. I'm

benefit. endpoint. And is surrogate studies I that's they the the a application. around scientific discussion think data, exist be disease, read either are preclinical and therefore, clear what's endpoint have surrogate, end the around have before that be surrogate points. to or studies is come data, no predict clinical approval support you like a certainly likely their FDA how to we what requirements guidance predict guidance to know their shown show benefit have What for surrogate opportunity that a we we for a ultimate they accelerated for very do Huntington's what one in sure where the situation and could could regarding from and where needed if What certain clinical -- an them had biomarker in a to guidance publishes would

that's that the scientific that Huntington's disease-causing we mutant other is that in there's of is course, believe very of of because that's for been and worthwhile preclinical of evidence in disease to predict necessary clinical discussion biomarker has benefit, monogenetic is in both patients. Furthermore, of course, Huntington then to benefit. protein, point, discussion understand disease, scientifically, is, or XX% clinical the situation in you biomarker and that case terms that but have a only provides unique neurodegenerative that have terms to an being demonstrates to of Huntington therefore, clearly that of it's level lower Huntington the something lowering gives protein logical have which want the what the benefit. And scientific a bar are absolutely associated cause mutant if endpoint XX% and they case protein, is models in is benefits, out disease their should not so surrogate the associated that having FDA in we can again, a a borne knowing And the quite literature that likely the of guidance have literature. And a range what protein, in advantage you to that amount only lowering reason of protein of this achieve demonstrating likely benefits, with says it reduction Huntington's needed has benefit. some the clinical as a the highlights in the change vast that been of not and Huntington a And the in we but

would say I Huntington within will So that and reading just them. see, FDA FDA written the published protein and want with conversations guidance they what themselves to the our and the that to have squarely basis shows fits of be them what

Operator

concludes CEO Well, question-and-answer this remarks. Klein now for back over our session. would I closing Matthew to to call like turn our

Matthew Klein

year. proud team's the half of joining for first in Thank incredibly performance I'm you the our the today. call all of

mentioned call, all the milestones. we our As we've on achieved of

We on all continue fronts. to execute

regulatory three submissions. have We

We're we have and effectively remain XXXX. important schedule strong sheet. We operating for milestones -- balance another expenses managing other in on a

I path, So evening. we you progress you. the continuing a forward good and look with to share Thank all our along to wish

Operator

the program. does conference. conclude you Thank participating This for today's in

may You disconnect. now

PTC Therapeutics (PTCT) 8 Aug 242024 Q2 Earnings call transcript