PTCT - Earnings call transcript Q1 2021

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to the PTC First Quarter 2021 Financial Results Conference Call. At this time all participants are in a listen only mode. After the speakers presentation there will be a question-and-answer session. [Operator instructions] Please be advised that today's conference is being recorded. [Operator instructions] I would now like to hand the conference over to your host, Kylie O'Keefe, Senior Vice President Commercial and Corporate Strategy. ahead. go Please

Kylie O'Keefe

thank the And and joining today us results. Good Therapeutics you first discuss PTC update quarter to afternoon. for XXXX financial corporate

Joining Emily Chief Business Executive Eric Pauwels; our Chief call Chief Officer, Officer, me Financial our Peltz; Officer, Development our Stuart our Chief Klein; Matthew and on today's Hill. Officer, is

our take statements. will investor contains slide based on statements Before let remind me forward-looking conjunction this in we website our review you expectations. relations include a Please with start, posted which to on that today's call call, moment current the forward-looking

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

non-GAAP to Form For quarterly filed other most Commission, a We well detailed and would is available Information measures, With of of Stuart XX-Q with over risks non-GAAP Exchange as description to information on to the to the you regarding GAAP the as pass report the review and today's Stuart? a our disclose earnings recent applicable financial me Form on and GAAP of during Peltz. that, annual in encourage our company's report release. non-GAAP certain company's call use and reconciliation our call. XX-K filings. let CEO, this Securities we SEC uncertainties,

Stuart Peltz

Thanks, Kylie. And today. for joining thank you us

resilience believe into to testament pandemic, incredibly our pandemic, reflect our success we I'm circumstances is I COVID the continued PTC's on fortitude a challenging As adapt their and patients, to despite employees, of and abound. this stakeholders and of a the people, incredible year determination marks during which to quarter proud growth time. the this

to and initiatives to debilitating, we stronger mission time as that environmental, At have social provide report taken an challenging have innovative diseases very patients pleased even PTC governance treatment no passing for and that remains; options. emerging were to deep is we always this to to Culture I'm our Our from continue with recently our few or pride company. are rare seriously, PTC Gallup's treatments We our We employees, received commitment also honored not to this ongoing, our alongside culture. reflects ingredient received Accenture Strengths-Based award our key and the to has this they have as our Clifton grow a School changed. success. Award, which System. ourselves Atlantic on PTC, Don

and to the We speak now upcoming towards out-performed a performance on our progress substantial me quarter. in delivered milestones Let objectives, revenue this are strong number of protection, making key XXXX.

to The ever expect have been hard growth The growth This incredible compared let's patients revenues, considering to Translarna one is first franchise in First, was continue of with world. these Emflaza franchise. XX% substantial robust and our commercial quite of quarter and treatments the XXXX in XXXX, to to see and working continues continues saw strongest revenue. on impressive to the around Translarna in into deliver focus to strong the team growth DMD future. the Emflaza we quarters launched growth, and in bring a XXXX. commercial

patients. strong and growth first period This following that had report post-approval, treatment. share orally to represents remarkable a the received a the the the Evrysdi, pent effective bioavailable, This now were SMA for muscular treatment need continued for day. market to pleased move at-home a the let's on spinal time uptake patients and Evrysdi Now Evrysdi for this see has to in sale XX% We with convenient, SMA first short X,XXX atrophy. US, in therapy we shows continue. EU expect up to approval EMA of

from our $XX partner Roche. triggered sale a EU The milestone first million

I a success growth development us pricing is end also roadmap Evrysdi this approval expected oral, before year. to. disease, return for our reimbursement. as XUS number platform HD, to candidates The provides future be demonstrated markets a molecule guides therapeutic and of Huntington's key of has roadmap splicing We or for we and of which the in In The proven the will robust therapeutics. including disease our established PTCXXX to Japanese secure that engine splicing now SMA expect in additional European HD. deep for identify recent A dive, a small with diseases, we Huntington's

only is Phase We small clinical XX%, all HTT importantly, of across titratable also even blood-brain preliminary bioavailable dose In [indiscernible] HTT Pre-clinically, but from selective penetrates it profound. the levels dose. demonstrated and orally in lowering targeted with reduces that healthy also barrier quite were sections brain, the an the dependent not molecule achieved that uniformly blocks. uniformly results volunteers, PTCXXX mRNA protein and the to we desired single of a X mRNA in Most trials XX periphery. beyond the the lowered the

able properties program. a MAD disease demonstrated look to healthy additional and touch results PTCXXX well-tolerated, Rarely it has and to Huntington's you the X of potential PTCXXX, SAD also We emerge in completed for that treatment Phase in of date and therapy sharing next on now to program, unique modifying to with a disease. Based to to in the steps on of you're puts first PKU to and mechanism trial the pleased in position. study We're findings. action extremely progress demonstrate I this cohorts progresses. the forward the the And target are analogous was no safety in we us choice the volunteers, found be Evrysdi our on as and the as pharmaceutical believe want with

PTCXXX. was showed twofold levels in head-to-head of PTCXXX phenylalanine reduction previously X greater Phase a blood Kuvan. study small to relative that a reminder, with performed demonstrated a The results As responder

PTCXXX classical compared results that more responded Kuvan, patients as XX% also show the patients to Importantly, with to including PKU.

differentiated high APHENITY, unmet newborn a excellence, evaluating of this vast as this estimated address a majority centers end and With therapy need. and by We potential XXXX. mid registrational XX,XXX We of established in called medical of expect PKU are not therapy. this see well and addressed the patients, screening by global will an start PTCXXX results program. have There's see year, to exciting current we PKU, for trial to we PTCXXX as clinically the an prevalence

Let to bio-e novel key platform this me The disorders next and pipeline diversified targeting approach a platform. because is of of bio-e component of turn to our our oxidative inflammation. stress

action one As initiated ataxia. a mitochondrial and trials its reminder, pathology vatiquinone, the have with bio-e epilepsy the first Friedreich Through X from registrational XX-lipoxygenase, mitochondrial stress we platform, vatiquinone the ataxia. epilepsy for up for reduces targeted-based compound enzyme as one Friedreich oxidative at the ends that and

approval on some to complete a inability related our preapproval to share CHMP to these for updates on in the the requested of has gene inspection Moving PTC-AADC. to Due we therapy stop platform, inspections. completion of allow its COVID because have the process MAA clock to delays,

the a opinion XXXX. As of result clock quarter the receiving this in third we CHMP of stop, now anticipate

to addition, surgery, quarter. COVID-related delays complete third further delayed now submission to at the BLA least we In anticipate a the due be cannula by to

to Let's in now COVID-XX. turn PTCXXX

continues it's enzyme cellular is dihydroorotate drug which enzyme elicit targeting reminder, cytokine mutate. particularly that an replication both oral, functions less resistance, mechanism the with the PTCXXX important dehydrogenase, DHODH. is effects. viral of likely a to the or inhibits cellular viral dual is calms and small a demonstrates a of storm. molecule As PTCXXX the to PTCXXX instead SARS-CoV-X by of anti-inflammatory targeting antiviral advantage action and The virus protein that as

currently registrational of are We stages. X Phase a running II/III consisted trial

our second enrollment development to commercial additional across be ongoing and pipeline expect global We XXXX. the to our the mid-XXXX. continue trials robust one for trial initiated deliver full and We're program to completed in currently quarter proud X that in includes be of to registrational

We great turn the who progress. our in a With the financial milestones remainder of call number clinical a year. further over the Matt, that, position are look to upcoming will forward discuss to and in with strong I'll balance Matt? a cash to of

Matthew Klein

progress like have would to development teams the by of challenges emphasizing I Stu. start consistent ongoing the despite the pandemic. made Thanks, our

are success pleased I scientific advancing most the our from platforms, of our recent in We program have proud very had all that programs and updates. we share to am of

platform. our splicing with validated First, I'll begin

about efflux volunteer oral, we reflect the are deliver specific, X is study selective, and meaningful brain developing key molecule of remain experience broad PTCs and As PTCXXX PTCXXX splicing drugs. properties healthy differentiating potential Stu to small benefit the patients. in biodistributed broadly mentioned, PTCXXX in biodistribution CNS, enthusiastic ongoing. of lack Phase Huntington's disease a to and The

the we dive of Phase X As dependent of deep first study. X last the SAD in shared the month, from and dose data MAD objective Huntington key mRNA, demonstrated the reduction cohorts

dose. We of levels lowest once the data, XX from achieved the desired Furthermore, in forward mRNA long XX% including sharing CSF In we to to the that sustained the reduction pharmacology MAD PTCXXX dose cessation addition, in XX HTT hours after resulted of in cohort up reduction mRNA in sampling observed we cohort. half-life additional HTT look to available. data

enrollment registrational our mitochondrial epilepsy is in and Turning ataxia. X to ongoing our bio-e vatiquinone trials platform, Friedrich in

scale. patients, a and placebo-controlled pediatric assessed epilepsy Friedrich the activities endpoint disease improvement treatment. improvement the of and Phase associated observable refractory in inherited daily in seizures as in endpoint endpoint XX X a adult study, MOVE-FA primary reduction enrolling modified MIT-E The is our following secondary the is living The global FARS reminder, trial, global also study mitochondrial of seizures. ataxia the key in a trial. trial As this FA score, trial, is mitochondrial The and the a children motor is with ABL X approximately by study is placebo-controlled primary of months

EMA. have consultation endpoint in the we FDA and As developed was this with strategy discussed previously, both

QX We XXXX. expect in the to XXXX, MOVE-FA have MIT-E from data in the study and from readouts study

bio-e expect data generation of We the disorders by X response XX-lipoxygenase a quarter. have from for neurodegenerative and inhibitor is to developed study, being pathway. compound second PTCXXX the this characterized pathology our later completed available have the the XX-lipoxygenase next PTCXXX, Phase platform,

the BHX, PKU. Turning is meet about we potential unmet administered orally phenylalanine of our in classical need the PKU. reducing in the mentioned, PKU even an of PTCXXX affected significant for effects including cell demonstrated patients. PTCXXX program, a persistent medical hydroxylase X excited levels of Phase the in are the to as most co-factor crosses enzyme is precursor PTCXXX in that PKU trial, and Stu to severe membrane the readily phenylalanine

Phase APHENITY in trial, mid schedule We trial, global initiate the placebo-controlled XXXX. our are X to on

Next, an I therapy on update if gene our program. write deficiency AADC could

pre-approval for of these inspections to of has to in CHMP process inspections. to As requested the because an due delays, complete clock COVID-related the approval Stu MAA a stop inability completion mentioned, allow

the clock quarter of receiving As a CHMP now stop, the third result opinion in anticipate XXXX. we this of

addition, the and detail. efforts, launch commercial been our which third we submission be plan now delayed, teams more in quarter. the delayed their will one commercial Eric at has anticipate global to cannula Nonetheless, least BLA surgery continuing In with are describe

PTPXXX of trial Finally, COVID-XX. I FITEXX in want to in the continued progress share our

this X. We of vaccine have Despite currently great for are COVID-XX effective vaccines, strides the remains need therapies to variance, distribution, in challenges completed stage we enrolling one vaccine of stage trial registrational as COVID-XX face due and a new there development and hesitancy. virus uneven the

We second expect to half of in FITEXX for XXXX. data the have

continue development advancing make see, hand to the over this progress will I update As we our can Eric quarter. in now an and pipeline. robust for execution you Eric? to diverse our on commercial call

Eric Pauwels

Thanks, in successful the Matt. the to was revenue delivering customer-facing focus growth DMD most continued instrumental remarkable commercial commercial proud with franchise. our excellence of for executing of date. from on of global our The one quarters I'm team

and in incredible Translarna XX% with growth seen our franchise, Emflaza DMD have growth. at the leading at XX% We way

compared XX% grew franchise XXXX. DMD total QX, to Our

We DMD at incredible XX% Emflaza XX% our way growth. in Translarna have at and seen the franchise with leading growth

Our programs, patient from growth in largest driven in treatment primarily assistance patient sustain DMD compared Emflaza compliance starts, new discontinuations reduction XX% the bridge with and to maintaining base XXXX. globally. in The total group high franchise of levels is DMD patients and QX, a lower

all FDA for As Emflaza patients and X years DMD older. ages and the a only is reminder, first approved treatment

multiple prednisone, and benefits Emflaza's support real-world with of presented differentiated MDA Importantly, we AAF. publications including data clinically continue at to the over data switch recent

of due markets, high growth growth from performance key as as broader compliance, patients switches continued healthcare We ongoing access strong prescription providers. patient expansion their continue Translarna's in geography, expansion. base the is strong, well driven see and from in to existing new by continued seeking to geographic

fourth all Russia ambulatory in financially plan the are we in children announce to DMD the support a mutations, approval XXXX, Russian Federation Translarna. approved the pleased of that has eligible, with to Following nonsense quarter

Latin Europe, of We a East, diagnosed to existing working purchase additional in leadership increases group COVID-XX towards forward the DMD Health, the XXXX we look continued America, Eastern administrative continue and to in securing the Despite into half Ministry Pacific. expansion challenges newly Brazil, geographies of also in patients, meet ongoing new in DMD and driven and patients. Middle and the by changes central see Asia in needs second are order to for Translarna of and

with Tegsedi The to patients, in and and region. the continue in to for team patient despite continues certain turning through challenges avenues we Vylibra. disease America identification pricing Now, for this education, In the program available ongoing Latin make awareness patient support our Tegsedi programs. And progress countries to COVID-XX within early access provide make testing, and Latin genetic we explore to in continue during America processes, Tegsedi. medical Brazil, to

some we We from are through first the Vylibra anticipate the of early are Brazil, approval benefiting QX. access progress pleased treatment pathways in and ANVISA in have Latin patients that we now in as making with America

Moving AADC. on to

changes initiated approval. gene has deficiency transformative XXX patients continued enriched with EMEA and aim for identify patient testing on XX focus in meaningful in shortly saliva-based AADC has final by As continues large, genetic a XXX therapy potential than countries confident high-risk remain a over in produce therapy now a programs this had expected to is launch to and after is We globally over that gene patients execute with reminder, a to preparing populations. activities occur PTC goal. deficiency, which Europe PTC more at-home with AADC to we the in patients. PTC on screening the AADC for

U.S., their centers and Union identification, underway one addition The has preparation call Emily in pediatric update. ever. financial strategic a against continue focus will the a to quarterly and revenues the strongest to and ability as In which of have now their generated of European over activities. our and PTC expert as awareness excellence, to execute Latin is on educational to disease our the team priorities. of And turn I Emily. through facing well I America, patient neurosurgical continued pride customer continued identification for team

Emily Hill

we the revenue platforms saw multiple quarter first progress of XXXX, growth of In Eric. across Thanks strong our pipeline. and continued

current strong and with strategically to addition, In revenue us cash our have the growth, proactively to we royalty conditions, year This, deal. in market monetization proud continue last our position combined advance sheet our a puts strengthened given through impressive diverse are pipeline. balance to

of royalty net revenues results, of XX% increase first the revenue a DMD payment $X.X from franchise the primarily XX the release for a and $XXX.X details. details of Quarter line This the collaboration We the by on few million. of to executing XXXX, the was this top to quarter many this the press growth. XXXX. first results. The a milestone issued summarizes deliver of over of million for sale product million, earlier million financial are financial driven number milestones longterm fronts minutes afternoon First for year revenue XXXX on potentially from Beginning take review now additional the were results. revenue press of $XX first value-creating with to I'll commercial our these Evrysdi, for Please quarter refer release EU

our net $XX.X XXXX. compared product quarter were Translarna million DMD of million franchise. first $XX.X to for first to the revenues Turning

$XX.X For compared Emflaza, million, net the we quarter in product $XX.X reported as million of of revenues XXXX. first to

now to Moving Evrysdi.

report Our sales partners, approximately million in XXXX of Swiss Roche, XX year-to-date francs.

as when worldwide. along royalties with cash Evrysdi revert $XX.X Non-gap XXXX, $XXX.X year fully COVID-XX, PTC. growth, to payment reminder, the sales The securities of compensation million maintain excluding Cash, and $XX.X and quarter non-cash, therapy, cash advancing The of a bio-E stock-based investment advancement XXXX, receives expense, pipeline. while were the return marketable therapy well PTC quarter totaled back in cash milestone year this in Evrysdi retain. future compared million earn monetization Operator? as million compared the expenses as as stream, sale preferred, to spending XXXX, $X.X royalties now and first $XX.X XX% expense. Non-gap quarter not platforms. transaction, the non-cash, cash a non-cash, only the in by was to for royalty the stock-based assets, hundred a gene first metabolic, billion stock-based Pharma XXst, become [MPPCXXX] first December a sheet a for increased future million first in related as of March we could $XX for this million reflects XXst, million SGNA potential first non-cash, Q&A. $XX allowing of monetization were of the deal, research still balance of operator quarter the majority and call flow expenses our programs for the R&D global million XXXX, $X.X the turn of One $X our increase clinical SMA over stock-based of transformed increases with to $X.X $XX.X million compensation occurred. compensation commercial million sales-based of the excluding current will over of $XXX quarter European which the we milestones excluding bringing royalty expense. PTC of our million in to forward R&D I the that $XXX.X royal As due excluding for and to Also expenses compared triggered million to approximately billion XXXX, until retains percent equivalents, in in approximately after Royalty expense XXXX. compensation to

Operator

[Operator first JPMorgan. with of from Instructions] Our comes the question Joseph line Eric

Eric Joseph

on PTCXXX Just a Huntington's. from couple primarily for us,

and Roll how concept to looking we Or drug updates in differently? will on to of be that those you to of guess analysis will from and CSF, patient And or the the any at sufficient are the you study? about study? thinking I some change how the presentations from one therapeutic CSF proof secondly, protein also has the Phase coming exposure thinking in the in in be inform away exposure of the next the in thinking study? is, X for typically benefit establishing the or cohort, a should terms looking their First timing simply So feasibility of your be periphery evolved patient in HD, selections of then the Huntington

Stuart Peltz

Yes. Thanks for Thanks Eric. the question.

And the so get that ascending as And the as the as well process we food so said, CSS the will affect, cohorts. a of in dose, the treatment, completing that include multiple we've additional analysis. protein we're

to it soon. we'll when share be forward information available. So that look we And this relatively getting then becomes

And working so that's well.

terms think thinking, the how in be update, will do exposure in enough. we're and of terms So we of the well

You've that a demonstrating what of blood got to is in meant we see remember, equal drug the have really remind that. that are to terms you results we be CSF. in able what lot a is blood in to see we we what see levels advantage what was the brain equivalent that to find the we in able that's I able being major the to and to that do be of in

the of target, single So that. we even lowering we've of excited with the the on Obviously dose. seen that a to data in lot have a we're results terms have seen

so that. completing And be going we're to

seen we of that the the the went as with data, day, terms in ASOs. think was of the brain The they, results and was penetration. at thinking think of was of which that based toxicity, insufficient the with terms toxicity, is drug we We terms wrong, a in questions, thing seems think what to I SPINRAZA the the we issue, So so it's the the we that question of in it's be do more And due issue, problem end both that the in Roche Roche on think the as that on... we issue. as think to specific by in with that, in what it of real the saw is think it deep way, well the are observed reason hydrocephalus an also ASO terms terms there

Roche I of And I about penetration. the So not think in interfering had surprised the consistent has Xx as reported. is hydrocephalus. And there, this I'm with what I Roche well the so volume with you that they seeing ASO probably amount can remind drug, the think XXx that the they're that's and

view, that about everyone of data effects lower level want from the our reduces in confident models Huntington best is tolerated, shows think and really case point of for the that and occur. be patients consequence in And Disease that seen monogenic don't reducing remind not We're targets of of a from So the protein there's make are a like by argument clinical levels pretty else. we've we the animal lowering it I that to Huntington's an our could that well that. And is protein. the both levels there lower HTT. alter HTT benefit. disease plethora It's RNA it's have is of the that HTT Disease of a drug I statement actually who is this is want ability to to to in the And immune that the HTT and both showing the that of preventing and of effects definitely disease, splicing, that. are for critical wild-type Huntington's a that, XX% that plenty studies both demonstrated something

is orally every the a So does to the the this in best really, that has distribution. within really efflux. class this tissue to that have potential of tissue, promising Eric? we the broad brain gets that can the precise molecule It bioavailable small with treatment. the think not control we you And fact that -- you, drug drug level an for help we Does be think,

Operator

next of with Our Cantor the Alethia comes question line Fitzgerald. from Young

Unidentified Analyst

on is what file for remaining are Nina U.S. were the wondering This the Alethia. in steps to We

COVID? one than the delay quarter? potentially delay Q related beyond the AADC do to And longer is you a think For

Stuart Peltz

for Thanks Sure. the question.

in sit it's to able results stand actually be and people to be to being those able really arrested, developmentally walk. and are take who product, exciting able who and in to seen kids really being ultimately development, able a obviously shown is think, I AADC, and So to see transformational of terms are, being

little that. bit about the there? a about talk really Matt, want you steps to we're pretty So excited

Matthew Klein

that's take clock as made have and forward complete [indiscernible] we by the stop the can and so to we've pre-approval the asked well the MAH, submitted the and EMA call in inspections. EMA they now In that a been the moving comments the

want just in have that opinion now third that moving we everyone remind that's the So to and expect forward to quarter.

was we've talked agency's as is BLA, to one us the we a specific experience which the desire product, about, the factors some with demonstrate of gating to for cannula really For smart gene key use goal the intend specific cannula. of therapy our with commercially,

cannula of was marked Now that the an an has feet not not in MAA been in issue in note, EU, issue. the which it process. is why was cannula The

FDA side, about On administration talked has the cannula CNS specifically XXXK-cleared before, the product before, therapy And just procedures gene used procedures. a It's for the surgery. been gene that not stereotactic therapy think is to of that but I a we've administered way is through number products. of as gene therapy our been complex

putamen that that we tells a So to place. that map basically outside the a them therapy and into of procedure gene therapy The surgeons where the path from get world of what the into the is is, how the instills cannula gene do equipment get provide piece the they the infusion Google exact the before follows product. safely direct

So X procedures already. the done we've of

data as place previously and along that everything And the third been well. and We that's agency is move forward procedure. procedures take third had the before, in to scheduled plan, complete we with is our make to As went And those look else then obviously sure discussed delayed. reported, point. that at and we'll we've a

Operator

comes line Robyn question from with Truist Securities. next of Our Karnauskas the

Robyn Karnauskas

data, First, just to follow on for expectations two had I Huntington up questions.

not Can And set learn data little on us the CSF on be bit how protein we lowered in that over-expect difference, delay question thoughts as view words, said we interested to don't the color, we'll should too it's more updated bar had if be way? as a delay to of the a much? other shortly. expect then levels so that the you can Second, just we protein? because should You getting expect, should be the in a levels, protein from low I and seeing, or a much CSF. expect really what And lowering give which the RNA in that an PTC-XXX. a everyone's lastly, on we for we working data Should true similar you we of them that

it lot Kuvan. So be this about compares drug into to diet? you trial? Palynziq? that able how think about compares a without you that talked your you'd affinity to you And how do to What use Are incorporating the

Stuart Peltz

the brain Robyn, we thank Yes, capable questions. on we've gets of a of drug Look the done for the know is all we this lot that the... work and think passing blood in barrier, aspects of saw where the that non-human We've gets seen primates, types equals brain we the and primates equal the into in the We've seen CSF. that all tissue where non-human we saw amounts. into

within were of we where other at. saw we've blood the by seen in rats, we'll primates, of looked think non-human will saw same it that, the we know, observed in drug the the we -- what CSF. is is that amount what that the we in the That levels the that the in both what before, free and do trial, we'll be sufficient So clinical we way,

that feel that, be we're So and will pretty about comfortable coming up. so

so And that's one.

time. dose that not within a yet and at XX be that in to perfect not anticipate sense we're we of -- the what And levels, think days question. question since I we're steady should we'll as part you're cohorts, to of one levels And the do sufficient but may one-to-one see it protein terms the of look particular at answer we proteins. then and that reduction is doing state the RNA in is that to, see expecting think be the to

on reduction. So calculate have and what we'll think terms to that of able percent know the we'll in levels we based we be

of point... we'll you goes as know the So protein level that of discussion to production but you RNA proteins, you from to it's pretty confident I observed the the level there's we've as level that a protein reduce down, be of lot getting the about make no proportional of a that it, RNA, the I good the see, if RNA, reduction protein, we'll a and There's make correlation. wouldn't been you RNA pretty the synthesis. a that see about pretty you be that. regulation there's see think you

and pretty the you'll we're see that reduction when see reduction within you So the a of also confident RNA that protein. a

the we're seen all throughout So we've the at both That's when our we've and protein animal we've models of work, comfortable. that very what within tested. RNA looked reduction

excited XXX, patients. about comfortable And with very we're then So we we're targets think more that actually obviously with that. it it

number a active of as well And it's causes a greater that's think in as Kuvan patients it true with as Palynziq. as So actually more level. we both lower well

the molecule well, seen it that pretty that quite So to we've comfortable trial. this be X quite active, that in an Phase going that We gets is fact feel perform it's the to proven these it's oral helpful it patients.

you Next, want anything that? to to add

Matthew Klein

head-to-head just really in the Phase say of effect would bioavailability. that Kuvan with that superior X which for study, the reason, is reason, a clear we comparison I part was the referenced, and the

giving cross cofactor into get actually it activating readily a to small PTCXXX with just cell kidneys, that's phenylalanine poorly is into formed treatment into Whereas which is excreted and getting precursor is is cells. Kuvan itself BHX a that get activated hydroxylase to much the BDHX. it's And by where a which highly the enzyme, the dysfunctional molecule, to enzyme. effectively either Kuvan the alternatively only on it's PKU in for from goes the plasma allows of lipophobic membranes, And know, absorbed, You the precursor the requires disease. BHX,

seen the where had superior our seen in classical this BKU. study really study the no really bioavailability phenylalanine PKU in of an patients, -- PKU in including which the So much superior points therefore almost patients X reduction a XXX story Kuvan-treated Phase XXX is in and in reduction action activity was the classical of classical there in the patients, in

auto-injector a you for there comparison Now and of while It obviously Palynziq to patients, had in time We shown concerns. case -- Palynziq. also didn't got in Palynziq have epinephrine fully it's you be also of a Palynziq takes risks. are safety to anaphylaxis Palynziq some do effective. tolerability has requires efficacy head-to-head and an one

which to So to an there uptake. by obviously important in attributed the kids, really It's part is population. universal a which also part its of lack of used may is tolerability be be not concern

So really large medical we there's look think a at you existing the still unmet marketplaces, [indiscernible]. when PKU

not the a the so at efficacious the don't something to study, we expect obviously you But diet of we're factors diets as in within but a we're question, be therapy, diet in obviously, controlled focused, placebo-controlled [indiscernible] that any you study. terms are explicitly could looking that's likes sure lucid. those In have make agency

Operator

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

Brian Abrahams

help construct sales. in disentangle to maybe on do over off-target then obviously inflammatory strong any of can you secondly, maybe or ASL effect itself. expression? Have Emflaza, that from clinically, questions RNA quarter assayed And first have potential some growth adverse any showed lowering evolving response quarter or you pre-clinically or non-human ones Two of cellular or I analyses in on the space. Either guys done guess, protein volumes me, done Huntington's ventricular primates versus HTT maybe a

any that Just wondering if have in? down terms in may little was there much changes gross-to-net that also in ordering maybe bit demand-based how other a of factored or patterns were that or could inventory you break

Stuart Peltz

Thanks, for question. Yes. the Brian,

this whole increase everybody, being nature to through without in of Just it's the right, get remind CSF. able the to any and

didn't we hydrocephalus And primate. see in so non-human when any measured, we the

any that's disentangling, we terms really point, that good our the of of seeing. drug, So think in what I a in of data had not primate non-human you're terms seen

is did in to the we we also effect, tissues, distribute we just it the we our it think again, in what's [indiscernible], bioavailable that that blood, our just lot the us confidence, getting it's pretty think, the we point know the an saw with did And was of the view there's in, throughout that allowed to everywhere. get then the of again, for toxic really see doesn't not toxic the this. And was we I almost able efflux, brain orally the view, which and on it's you not just like it of same from it distribution, issues, -- think built I to build in of that that's see see see we bioavailable fact measure was and molecule gives just the oligonucleotide, just So blood of throughout an brain, to well to molecule. equal that's that us level and how with we what really us point body effect PTCXXX They're SMA, the X the the the that we thing. clear and the we when what like built an brain. a effect previously case And oral only to the

sense reasons in one there's other. think for think a us you I that So issue to as the from of lots said, disentangle we,

very disease. that to that the So think it's in that's a It's we're right you pretty And a confident certain indeed do. that want that the what the toxic monogenetic [indiscernible] it of, we're we this of really reducing not right target, is target. form is question

breakdown, Emflaza little want terms you a bit to talk in Eric, about So of the that? do

Eric Pauwels

incredible. your over the build very commercially we excellent I Brian, compliance over prescriptions patients best. the key number to seen that some what had minimized continued is, new been XX% quarter for really of To Yes, DMD strong. discontinuations been treatment in our growth of with of XXXX we strongest also in growth base it around. has We've well XX% We've new led that back last been Yes, prescription was refills, high with quarters almost year. a quarter, and the patients we year Emflaza, and have benefiting the very, about momentum been really mean, sure. an started extremely we've on accrue, its that's had to But of and able franchise way, Emflaza one question thresholds of in that specific There's we've and saw lower all been dropouts, and longer. compliance. and

we and From very teams some will, the new growth in you there's been so to of to, hard been very, commercial have of market had terms bring and time working the if best teams months a new in have fill. prescription, of our our commercial QX have And working prescription time. time where access our of down the

the as, reduce technically And bridge programs. well are a been we've plans of to we're that have on well. now able And reduce also years, right helped of Emflaza had programs, on sort we've change last patient So the have able free as which seeing restrictions been to that, many over removed programs, type fundamental that as of with assistance those, has time time. charge that which

we XXXX. the tailwinds a combination time will from a continue continue we it's prescription strong prescription a base that filled. throughout time as get, time of growth. And get new to well as a see continued kinds the the gets that we So it of to And these

Operator

next Our of the from question Danielle line Raymond James. Brill with comes

Danielle Brill

that we you out you seeing know toxicity I is rule program their can't talked Roche potential think a the Huntington's So role in given the ASL-related, how is the of completely this I'm wild-type maybe follow-up. has on about lot Huntington but thoughts another I strategy just have if curious a or possibility window. narrow development And at of XXX your all? impacted therapeutic the that for

Stuart Peltz

it that from the our a mutant do of sure. it already shows lot can is we've substantially, right? shown there mean, think effect. of So the what reduction disease, models reduce for question about it it's this discussions and makes Huntington?", monogenetic that shows to time of on And had and Huntington, patients actually that from disease wild from view, you type of elongate Then the I best And Yes. can is that HTT right? mutant is consequence where Sure. the the the has there data a lower the animal target. much also out certainly "how that HD you been we both XX%

is, think toxic of seen in this of said, we I what in the in there. not the the effects sense only I So you've data And but think there's then that plenty also out most Tominersen, Spinraza. hypothesis likely

XXx amount as of have as the Xx oligonucleotides the you the well volume. Now

already there. you're an issue creating So

So hypothesis, the other let's that's, but take I think, view. point the most likely of

The a the the in is see you already other oligonucleotides question lowering more we the that brain in one than disproportionate part show know crop. of other animals,

I'm then So already levels measuring, those in the you're using disproportionate of not to CSF, we that part, and measurement trying which exactly sure know you a you're means. have what the mutant Huntington make

have issue So an there. you already you know

getting not may You're hit everything, maybe. you

and take be your you problem titrate the the That's really bringing you of be another. you every down Let's the the in let's indeed, consequence hypothesis, spot part one equally shown to And that, particular get a much you in enough highly it's point, case. a overhitting levels we've brain That's not area. and you're too not distributed that have tissue the a to around small gets brain. controlled as might molecule when that say a true, can are and it, can

too bioavailable, the doesn't matter and wouldn't most molecule. you point consequence sort my another, problem much take to with very It the distribute from Roche it which seems small orally even is oligonucleotide. then, you still So probably of enough But one hypothesis is a that view, least of real the it's the themselves, it's a probably problem. likely therefore of is no that, in oligonucleotide me hypothesis And of well. that this the promising other take, still see let's and in reason place that said not

importance advantages orally it only reinforced the us, biodistribution. is think obviously, in we've it's I and an the of it's bioavailable our small that Not talking molecule for So something about -- the belief been of PTCXXX.

So levels just high when problems. do we the we to and -- believe levels which can cause humans it's this put all together don't by way, really likely oligonucleotides, we like know, of high and

animal the in I the far. the that thus So we've seeing confident when terms made any at of not differences volume We're in ventricles. in within progress think pretty models, very we're looked we've much

we're we have So we good in potential a class. good feel pretty the that pretty that in spot best

Danielle Brill

Friedrich's could therapy was you're to the Can And you That's gene when if just clinic the stand enter actually with things with comment really I asset? expecting quickly, ataxia that us where then wondering remind helpful. program. on you

Stuart Peltz

Yes. that. Thanks for

So program. we're about that excited

human before about, talked a the have had, We we progress of some expect we've as delays really going and keeps as dosing the COVID, year-end. the but on consequence first

of some just and completing our the items. doing -- is we're what gating So now goal is we --

moving in the going So now on have and preclinically we're utilize to and to activities and U.S. global some globally. the infrastructure be already the that going to clinic looking begun that's we've on of start-up ahead that into sites focus we and

really on working help? in we dosing. get the can Does bring we're So path the to so human first fastest that that that

Operator

Our of next Cowen Thome the question with comes Joe line from and Company.

Joseph Thome

versus as First And motor the that the in gene one And for has a placebo? in second, on of from benchmark you're that seizure X on seizure in But that kind pivotal there -- efficacy the Phase up are reduction out ataxia Friedrich's thinking the there of any one segmenting kind FDA population? frequency you therapies set vatiquinone. first measures X/X then is a disorder one this that a both following patients, been would are How in patients be study. therapy, vatiquinone? looking benefit about the are to therapy specifically for gene approach there those versus more

Stuart Peltz

during a of this. the but the But currently normally Matt we're but do had based as this we've controlled on patients and want you we have to questions, a a are obviously talk study, trouble, survival there how in is and a we've data about seeing those it, and were long-term talk good for you worldwide, they know, bit seen of Thanks it's real this... trial, questions, little I'll placebo consequence and in on thought more seizures Matt? who reduction about

MatthewKlein

thanks, Yes, for Tim. absolutely. And the Joe, questions. Thanks,

also mitochondrial that typically in kids refractory the mitochondrial symptom of mitochondrial of and epilepsy, anti-epileptic XX seizures existing patients XX% So that to and have in out inherited disease, of terms are turns so refers with with disease seizures to therapies. refractory it to seizures about

in existing energetic seizures pathways simple are these the that don't therapies cases. anti-epileptic the causing reason that target For

oxidative In stress exacerbate fact, medications pathology. seizure actually and underlying the many of the mitochondrial

there's both we've and to hospitalizations on what in more targeting we occurrences problem what case like also studies, one threshold. frequency, seizure of Again, having a And kids, market other these in contributes a observed that impact which tickling seizures, the disease on that have. in as reduction the obviously risk. importantly, further patients we're status epilepticus. specific that overall preclinical given complications, their seizure effect and in Whereas, seizure in the the well in the is disturbance pathways significant targets related obviously the seen So disease related and clinical of series, terms as and is mortality other energy those manifests they underlying

specific of disorder independent looked our improvement we of typically obviously one looked day clinically seizures a analysis, maybe And incredible that have so really a reduction we a or this these burden from as that the think series, life-threatening. XX, most which the is a in XXX, about regulatory it's significant being significant in which number, a given had disease, our we've seizure at target XXX that down be the It's seizures but regarded are the going burden. For at disease. though It's a acknowledge a of of meaningful, for serious the brain XXX, is neurological reduction a I pathology. is XX%, significant disease but sometimes which context, seizures, seizures of looking meds, a XX kids authorities to in to to morbidity is case end only in the points significant at addition refractory, of activity, reduction day, help seizure that XX, XXX a rescue are morbidity discussed really secondary and other the is in of think really disease, well-known ataxia, the endpoint, seizure be paint terms deserving which is it's aspects picture whole ground really to of the the is obviously also we'll name fuller When motor It's the key impact that of of and Not disease will ataxia, whole the attacks, in aspect zero it's motor frequent use other the one capturing serious which disease. body other that primary of cerebellum about and frequency, aspects component of therapy. Friedreich yes. overall you

whole as the taking the gene disease be of impact. cardiac therapy idea brain approach key cerebellar of aspect directly be disease the disease. we'll it's nucleus neurological targeted said, And our with with the still impact, a just body vatiquinone the that therapy. FA neurological can having whole aspect delivering [indiscernible] That gene therapy, dentate adjunctive and a we're with direct to so again gene ADC to obviously a an We're peripheral systemic a targeting a like being and administration,

really with view as all ability So to other for the we each and treated complimentary to this deliver benefit ataxia. patients

Operator

next line question the with Bristow of Colin Our comes UBS. from

Colin Bristow

the Congrats quarter. on

to PTC involvement me just timelines timeline us it's on on, subsequent Can quick then your and data you I a think the anticipated readout? through from XXX. walk one

Stuart Peltz

beauties measurement, X I Sure. it's short at terms -- a But think, is, of reduction. what, of that's terms that in yes, phenylalanine the about looking of time of the very weeks, one XXX in the

Matt, to bit want do so, you timelines? through And little a of go the

Matthew Klein

absolutely. Yes,

thanks again, and questions, for -- on up follow to the Just Colin.

comments assessed It's have blood that follow here, traditional composite scales clinical you things design where or a to a quickly. you and an which metric. when at a measure, look certainly to you diseases, than Stu's bit on is and and on PKU, quite test. aren't it trial objective administered Just has One doing phenylalanine move obviously advantages. end objectively up the many reduction, is easy an of It's have different point neurological readily

they're patients where the for a efficacy to of is able PTC up had, to we're successful to responders these So clinical running control X capturing is follow as the a who XXX. we path the with ensure we've X first example, way trials itself really where set week trials phase, enroll previous placebo we study know that ideal well, in to importantly week phase that been

get the say weeks, XXX that weeks. X X or for XXX you're either to, threshold responder who a then enrolled to And a treated we So receive we've subjects placebo with who be then PTC get able meet you're all randomized where criteria established for we'll if responder. will a and

So that those controlled really upfront phase with who to X have the XXX. responded knowingly enrich us to allows population that placebo week running already

obviously of big clinical increasing a the in that's the So probability of really advantage of terms success trial.

of number globally. leverage a the have do And XXX-XXX in that we area So our more somewhere at have enrichment identify existing that centers right are least this for be targeting global to to PTCs subjects it, existing meet be and would disease excellence now ready adequately quite will subjects to country larger enrolling, expect of trial to the threshold able of the we're able that enrollment, XX% there from going than obviously infrastructure. frankly, given get power when Again, and to we a is go. And we're centers excellence, success. teams patients with working they're to for global at of that

rapid And and fashion so expect of at then a the XXXX. into we enroll to and we're expecting in the data initiate by end trial XXXX get pretty

Operator

comes line Wong Gena question the from of next with Barclays. Our

Sheldon Fan

is have Gena. the a XXX program. This for Sheldon Huntington's I question on

you're now into will patients, need considering? right of type of healthy volunteers. dosing type that what So do recommended and be on for determine you we those continuing to do population that when data what you testing to and And real inpatient patient are move expect

Stuart Peltz

Sure.

the as well dose. multiple as out these send we'll single, the ascending doing we're obviously So

lowering We was well-type it that to and the already that in you everyone mRNA HTC that So get that. as saw I'm measured actually based pretty of the to reach determine terms reduction excited XX% we than in what objectives we the the with achieved already even out set wanted blood that it that demonstrated of we've results dose can preliminary to greater extremely, tradable. we on was level can we that

steady to So protein. reduction which here we to to to dose be we're confident leads that pretty in the lead that were position capable defining a state a of will RNA, pretty good a of reduction

the the the the in complete dose, a thorough doing job effects, process as well multiple sure pretty analysis. ascending of up make cohorts, protein So the additional completing we're CSF of as the finishing measurements, food to

have clear allows into to of actually probably dose there That we're know we're it of an that as gives RNA giving. exposure us a to gives reduction go doing of what the very about consequence Huntington have is a level that. vision that we'll I we have what done we'll of a from and what like that's we will So really that us

any in exposure knowing dose plane that we're reduction going We're blind exactly leads HTT what to flying here. and So in not RNA. is that the

mRNA then beginning type next for we'll also levels, HD then as is benefit. be similar about work the to to excited what that. of Huntington's will think -- pretty we patients both about And trial RNA and the of in clinical the we're are steps themselves, So levels show do both the in protein

size. so the a then want where reasonable future benefit HD So the enrich that population the with clinical we reasonable patient in in patient obviously as we're timeframe that design as benefits well sample we in to demonstrate a and possible

think be we're great to have in I measure. what position really define So set and going the we're what's right patient, to the then what's And we'll of right a the patients working to dose. hard right

where into that find And look we're this then so shows great roadmap SMA on going we at to we've a story And that and benefit. clinical analogous it, defined target it's to patients. the of

So that's plans our for now.

Operator

of with Raju the from William Blair. comes question last line Prasad Our

Raju Prasad

the for are how anti-viral Kind On that in the the and read data about thinking of some outs term? you thinking context near expected for you distribution that how Thanks about taking in program? a are of question. XXX, increasing vaccine and disclosure

Stuart Peltz

Yes, sure.

and but also that on treatment on to believe number of a efforts of globe, advantage I inhibits there's of variants of that, we're people I in I fair attenuates drug about that's a put other numbers COVID, it parts where going X continue of attacks its having something now and hits right virus lot of replication, are SARS, going here, like this, be is that. and come incredibly think both what's that XXX, vaccinated [indiscernible]. of significant my who seeing because still be additional people it viral happy the the both a that in valuable. COVID the as is really to a into probably so PTC There's you're action, fully going not mechanism and of having there's think things, most I'm of that vaccination in I say team can vaccinated.

to COVID-XX, valuable the treatment. and of for the incredibly outpatient So drugs also treatment

So we cellular this think it's this frankly, the enzyme. DHODH advantage dihydroorotate the a is other target going because to of be is and dehydrogenase -- or of

that going versus also So to a variants to virus the cellular targeting the less one. is anticipation a be enzyme because it's it's of viral susceptible

more to be resistant would So it mutation.

is some that's good. believe we So I think be time. us that generally with actually quite for going this to

it's of should that running It year. And to year. phase be this completed we X And this so trial, the X, a quarter currently from second stages that. the as not it's registrational expect half data we're that in in of should in said, too be we be far where the enrollment X second after

that's So to look able then and effect. be we'll our, the at

we're for the quite one first hopeful about having So perhaps certainly therapies, this that's disease. of excited therapy and a

Raju Prasad

Great.

Stuart Peltz

just Sorry. approval. going rapid for to we're toward working was and I pathways say, obviously

Raju Prasad

update any regarding site. update on the discussions And transplant disruption Thanks.

Stuart Peltz

Yes.

that work to So through we'll now to need this them. we're and working do, we be we once complete some then talking

a we're up with have So in of conversation the process the FDA. that finishing can so that we and go

Operator

to turn back remarks. This Peltz Stuart session. I the question-and-answer call closing will concludes for now today's

Stuart Peltz

Okay.

platforms, really on including each as very I volunteer think joining have from development us the really has continue aspects We're going also incredibly And to complete I I quarter to that thank [indiscernible] through, execute the we're team from wanted updates One as performance trial I healthy avenue. near all look, everyone value The think X discovery to shared PTC the our registrational program, continues an to provide XXX. PTC to the for to commercial through excited today. heard of strong So are study. data think for company which you all this across and preliminary trials, term we had disease recently drivers. the

hard And it to to and on patients the working innovative the translating science really their lives. team is the into mission. focused we're So are bring that obviously therapies patients this towards waiting. And the transformed

thank your concludes for So call. today. time you this And that

Operator

Ladies and your gentlemen, Thank call. this you concludes today's participation. conference for

disconnect. You may now

PTC Therapeutics (PTCT) 6 May 212021 Q1 Earnings call transcript