PTCT - Earnings call transcript Q2 2018

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Operator

Good day, ladies and gentlemen, and welcome to the PTC's Second Quarter Financial Results and Corporate Update. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] As a reminder, this call is being recorded. I would now like to turn the conference over to Emily Hill, Head of Investor Relations. begin. may you Ma'am

Emily Hill

and second for financial quarter afternoon, discuss results. us our XXXX corporate thank good joining you to Hello, update and

our Christine Utter. Joining CEO, Souza; call today's our Chief Peltz; and Officer, me Marcio Operating Financial is Principal Officer, on Stuart our

based let to statements review which on simultaneous current we our include start, today's contains will take presentation statements. remind slide me Before expectations. our that Please you call forward-looking on moment forward-looking a our

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

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

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

Stu Peltz

for And joining on this thanks Emily. Thanks, afternoon. us

update. our me will with today the Marcio development and on end Marcio provide financials. of the and clinical a Joining the will review call are commercial Christine. call Christine

of has The first half for transformative XXXX clearly PTC. been

executively in first of year. executing that can the my this and strategic five-year see a to XXth disease three strategy. the proud on leading Analyst the As I strategy where team. as Day of provided out at I'm we Taking a it, outlined rare PTC hope have to of Anniversary biotech I our incredibly we aggressively outline have you build a we plan. half on company aggressively been vision

stage recently grow We late announced our franchise. our we've commercial pipeline. our two most And splicing asset. franchising continue pipeline event transactions with important and and strengthened niche oncology that to In successfully Duchenne

which with allows Commission each or is approved a The rare (hATTR) of October two (hATTR). with large by is Latin hereditary us America by as also polyneuropathy an in Tegsedi important America. in is been the have X contains diseases transthyretin treatment Tegsedi X. of for of in occurred those important form the Portuguese Most Latin indicated stage by to drug Tegsedi America. ancestry of more approval critical one of is Akcea strategically X for Commission has and we European now. announced which Let The European review Brazil Waylivra. polyneuropathic adult patients me in amyloidosis individuals Latin recently portion with in because commercialize of particular for a discuss agreement region stage has under PDUFA The and FDA States United (hATTR) Tegsedi. polyneuropathic patient. important frequently Tegsedi the

the as We're On these to on us acquisition. agreement possible. America. important forefront bring eager we in in to therapies the the drugs innovative bringing the stage XXXX transactions Latin gene of to Agilis the file including places This our XX, Puts technology lead quickly a transaction Europe behind the very is for next treatment under patients regulatory with a rare the of CNS well (FCS). platform. Waylivra for and disease gene syndrome We're leading space collaboration orphan treating States product in program. ideal acquire leading PDUFA The successes global out building disorders a the in an company. in is strategy the our second planned the a in transaction for this monogenic The With Agilis and leverage position gene We for PTC's later biotech treatment the by AADC therapy a therapy with ask on initiative platform Biotherapeutic. us This these as infrastructure review August for call. on in Agilis Marcio deficiency. of program to more month rare people to to XX. our strategic elaborate a reflects up therapy fit the of regions BLA the review diseases patients therapy. excited later announced when late United CNS chylomicronemia these familial in I'll The asset I'll as coming lead as July gene

preservation nonsense dystrophy better strengthened earlier EMA's years olds. mutation ages Our half first two expansion patients function. a disease. approved muscle several Treating to for Translarna is The DMD Duchenne patients franchise on the DMD fronts. allows in for a year muscular of of label this five was in degenerative

to label that in this therapy to age. the patients a expansions gain countries Importantly, approval allows recognize at access younger EMA

as all franchise guidelines also guidelines early hard for in We're Emflaza, initiated DMD be United The includes working DMD United standard-of-care patients patients for that should five. age States. possible. as state in to the DMD DMD reinforce effort. all as establish over the Emflaza this Emflaza Recent Our

revenue to our DMD year-over-year quarter In has growth. execution the approximately commercial strong XXXX, totaled second for million. Our led $XX revenue franchise the

$XXX $XXX guidance XXXX million for year full million. Our to remains

FDA for outside to United Translarna patients. We year year US bring States a Beyond to to of study plan this for dystrophin accelerated to growth potential United opportunity and the the Translarna. expand submit States to approval Translarna current next initiate looking we're the also

I'll platform. to internal splicing turn developed our and sciences our programs pipeline. with internally our now start me research Let

Cure, We data studies SMA currently ongoing Foundation. dose Roche SMA recently pivotal patients. in SMA for Type interim presented oral of were molecule at SUNFISH Dallas. for in an is SMA This and the with include studies, have advancing X SMA partnership both studies clinic. and the in meeting a FIREFISH, infants program the pivotal The finding and portions as in small X for the treatment Type from X Encouraging

validated FIREFISH have our Dysautonomia. program and finding disease call. lead shown During which both of splicing later independently. the are studies more optimization These and video currently provide platform clinical expand I'll details include Familial The presentation progressing in was to our babies in stages. are of the study Huntington's three dose was in sitting a on success the SMA and

both clinic expect advance We XXXX. to programs by the into

our Marcio Marcio? to update over clinical call you efforts. on the pass to commercial now me and Let

Marcio Souza

just mentioned, advanced increasing the the Emflaza Stu we use Translarna States. in XXXX the and our As into for of standard-of-care toward DMD transforming United objective in franchise globally for continued

We population. and with patients Translarna EMA expanded include retrieve label the are to launch two additional five Translarna optimistic pleased expansion has The to started old. about this to years in patient patients now aged process I'm

We hard the age have working [ph] in DMD. diagnose very for to community help of the been

most operate, the been the was age and Before much at as reduced launch of least age where average the around six to old as country. eight geographies ago by we has In four diagnose Translarna years if six years. depending years months two on

patient patients general identified We're splicing to invest to outcome. to committed these continue drive to efforts agenda. continue disease advance in further We diagnose awareness, improve all to and improving the

of transitioning commitment expanding the report plaza We establish I'm We're the of cord patient with have outcomes [ph] first the DMD patients the prednisone. segment forward or have the been We're Emflaza. is previously on to patients of experience as the to similar Emflaza happy in standard a a are currently marching now improve US. us wave therapy to who to Emflaza patients to care commercial for complete.

to time lower burden work the been anticipated expected. has consuming mostly is we paper due doctor's Conversion phase and of previously the but than second been than side on payer The office more processing. happening, has

important slowing plazacore results patients The Emflaza's current critical demonstrating differentiated in a the and on XNG administrative the of recently efficacy publication Two guidelines of physical an of have been disease corticosteroid end another endpoints Emflaza [indiscernible] the DMD accelerated. benefit the prednisone a United over still strategy. diagnosis. have the upon patients starting announced progression up of time in measured important not comparing the treatment long-term to effect benefit. phase function increased naïve payers. clinically the number delayed improving [ph] remove half currently the with recommends underway [ph] using of prednisone States around We've plaza from We key [ph]. In and [ph] step of about Emflaza is yet Emflaza just with stakes and our plazacore the continuing information The This arm ensure of treatment. study. in Based to prednisone seated as initiatives demonstrated continuous Reaching DMD includes to treatment are with placebo published. publications in supports barriers

have a expand of to We penetrate strategy the label the youngest untreated patients segment to the well as to long-term DMD five. as address aged two to patients

of rare growing been our We're the Our the In highly revenue our diversified half expected million. $XXX the reported Translarna Positioning of an franchise geographic positions PTC year-over-year. XX% versus $XXX has BD commercial XXXX has growth and combined first reiterating product prior year. of in Emflaza of same franchise XXXX periods for to the DMD business partner. full disease guidance portfolio year as outstanding The the footprint. differentiated and global revenue million

have ability Waylivra. integrate Tegsedi commercialize line past in the Akcea with for Most demonstrated products we our two rare We disease. announced disease rare launch collaboration have now transactions America. In and in our from strategy effect rapidly month. chosen formative Latin and we to drugs recently to

established For we Chile Argentina, Translarna with Brazil, in strong Colombia. have efforts countries and a including our presence commercialization key

with or for (ATTR). resulted Our stage has in limited the authorization X approval patient with Tegsedi year-over-year in have received marketing efforts in is ATTR these of Commission a adult disease countries. European polyneuropathy strong X patients treatment growth identification from options. devastating treatment

be ATTR Latin many we As patients Brazil Tegsedi. treatment. patients, to worldwide. important polyneuropathy region for X,XXX such one than so markets one be eligible We Brazil In Stu more the contains and particular the Latin to of largest for America, said, strategically understand America in anticipate is

Our and on with robust Ionis working Akcea, a regulatory teams for team strategies to hard execute the go-to-market the region. is

America. uniquely are competitive to standpoint Latin in positioned execute a from We

treatment under therapy The the second the FCS. for be is review first only and Waylivra and the for FCS. the product to Waylivra of potential Europe indicated regulatory US in has

function the Waylivra We starting fatal expect Latin [ph] and FCS have expect we to and identification by market factor gaining for lipoprotein has opportunity characterized the is launch. caused impaired a to of FCS to one be enzyme potential be potentially by misdiagnosed similar unpredictable the lipase been for pancreatitis. Translarna of risk is in acute the and America. to patient of to

excited this to are life changing patients. opportunity the drugs We with to bring

cognitive stage strengthen factors it both programs micro-dosing. Micro-dosing our adding program and including we BLA, specific caused to late to structures gene allows month, pipeline therapy the all bring genetherapy four Agilis on This programs global bring last infrastructure. will Starting Biotherapeutics. announced. with manufacturing, to the various announced while to transaction CNS aligned in benefit reduced that platform from for acquisition of The to is as maximizing CNS. and therapies Angelman our recently are and bring eager which Directly in many [ph] of late gene strategic efficacy plan of by deficiency for the This patients. focused stage small in This well [ph] which we of acquisition late turn programs the disorders. of early as now the me Earlier as rare Many these us programs, therapy stages in with vital other stage leveraging AADC CNS. acquisition Friedreich's bring our advantages menogenisty of Let scale the in we at priorities syndrome XXXX. for targeted because disorders. submit and trading risk acquisition disease advantage planned we genetherapy ataxia current development Agilis a

with we're both clinical would Stuart? of that focus well rare commercial transforming I wise back the to on equipped confident now call patients and value continued capabilities disorders. drive to are strong Stu. a with We and hand

Stu Peltz

or in neuro small discovery I'd splicing of This our genetic like are SMA. early in and spinal you genetic generated disorder generally cause manifests of muscular technology Most is used a to therapies detail for to is that been atrophy infants leading death on the muscle molecule SMA more in life share rare potential platform. toddlers. program A Thanks on and data our which familiar Marcio. with has based program. recently

SMNX finding is in stages oral around provides and the Type drives X dose Data while other a Type The a X in length collaboration and ease administration. because complete SUNFISH RGXXXX, patients. and also restoration We enrolling for infants June of from and the registrational JEWELFISH not systemic study the FIREFISH tissue for two Foundation dose and therapy from program tolerability. and of messenger the JEWELFISH continued highlighted babies We portion of towards full patients Roche competitive with at is in Earlier modifiers. splicing has that X This from have Cure FIREFISH demonstrating to distribution at oral the SUNFISH portion finding survival. with believe a splicing SMA registrational of clinical data pivotal shown studies. benefit and SMA XX Dallas from were sustained FIREFISH currently advantage and that its of the an broad presented robust meeting in been program SMNX RNA. splicing SMA Data safety therapies.

of rolling [ph]. were the motor of sitting from During investigator babies the control, demonstrating video the a independently. study babies presentation improved head Three function depicted including in these sitting supine showed

baby first X.X The age depicted their sitting was received week XX. months and first at dose of

The second and dose was at at XX. sitting baby first age week received four of their depicted months

XX presented age meeting permanent national The riding eight ability have XX meeting a at events data was XX last lost survival. expectation SMA Notably type up The at week at the the XX.X XX.X XX. baby months. sitting third since also dose was was the X.X the densely between Previously of no tracheostomy study for that Ride-on to age have national across babies published dosed It indicate and - of first baby ventilation five Dragon toy RGXXXX of months have the a the initiation and first [indiscernible] to was and months. already, depicted also swallow. be the X of received of history no duration to months Of history or the for required surpassed have baby and months baby months babies to noted hospital.

technology with this an including data dose and is have Based been Society therapeutic now splicing drug compounds NDA year. levels been Scale. no well presentation by that through progressing a Recruitment collaborators without and data point. additional study developing treatment The Data As and all data the of We XXXX. progression program findings for to sitting that can reported the Infant this even are to patient. will percentage withdrawal. trial. previously sitting is systemic pre-mRNA primary The expected the the and World were to tolerated The early were dose presentation next the drug look portion a be complete babies presentation, identify at treatment of RGXXXX at FIREFISH is survival potential tests FIREFISH Two at and related you. validates for ongoing is at with endpoint Bayley meeting reports It in all safety and from the finding support Muscle to open primary platform transitioned SUNFISH to has not study there to disease modulate an instance with determined XX-month of be of at XX [ph] additional number for dose and been babies also is on of study label you shared motor XXXX oral increasing pivotal function We endpoint for XX score forward babies. as discussed splicing. throughout registrational June globally other of related. the SUNFISH only trial enrollment SMA believe extension share our milestone. of not a complete Ongoing there's sitting an and SMA file expected October. shopping The months. motor blinded The medical anticipated in of is a on this these FIREFISH studies SMA is towards and Congress selective have

diseases Familial applying include Huntington's programs. high unmet other These Dysautonomia. Disease expertise now have programs in medical to are and internal needs, We and our challenging with preclinical

the clinic We XXXX. enter both expect by programs to

and you details. those our to our full our Utter, Officer. on Day. Analyst for are turn Financial We like to And excited at website programs refer to in-depth cover Principal them now call slides I our the about I'd splicing very over Christine? Christine

Christine Utter

details. strong release across we for financial today details our the second of issued to and financial Thanks we few for you I release for Stu. results, press results our our with performance that the top performance of Starting our refer full reported summarizing with DMD XXXX. comments line Earlier and quarter on a start XXXX guidance franchise. I'll our

As product Stu guidance revenue to revenues geographies. growth existing increased penetration compared were net of quarter quarter stand are for prior quarter-over-quarter $XXX underlying net today, growth $XX.X as result results the uneven mentioned, a second X% million where period. were quarter The in of over our the comparison we XXXX growth. which our reflects of to rate million $XX.X second Latin representing for product of XXXX. Based last reiterating million Mostly of of the XXXX, America patterns XXXX made we X% representative second million total on $XX.X Translarna from $XXX in million. revenue of ordering the

the reflects for XXXX of in to the quarter compared an guidance our clinical non-cash growing total advancement commercial This Reflecting June at lumpiness $XX.X million R&D second expense compensation Our compensation our totaled pipeline. investment same marketable of product successful reflects expense. expense million to patient $XX.X stock-based reported approximately excluding international of our stock-based quarter at quarter same increased the million year. million loss was excluding this a Cash, compared compensation non-cash second second XXXX. R&D XXXX in million second we contemplates Translarna revenues XXXX. in million base in support stock-based to XXXX Non-GAAP expenses period period infrastructure. as continued of $X.X our reflect continue equivalents million period stock-based $XXX $X.X compared demand $X.X $XX.X of in Non-GAAP net expense the corporate excluding research to net Net to million million $X.X XX, activities $XX.X for million our securities XXXX in program Emflaza increased the million were $XX.X non-cash the cash in the million investment $X in and the to $XXX for compared non-cash and expense. of for loss for XXXX. were $XX.X in for of and franchise. XXXX quarter XXXX to for and yearend million decline in DMD excluding the revenue leverage SG&A same This loss compensation the expenses net

last of are just in We proud we transactions the announced the month.

we integration. As forward with on transactions and move close these

operator the call the now We will year. session. expected the Operator? I provide question-and-answer update our for to to start spend over our hand will to

Operator

[Operator Brian RBC comes Markets. Capital and from Abrahams our question Instructions] with first

Unidentified Analyst

This actually line for our on the Thanks all on is and congrats Rick Brian. taking for the questions progress.

Or First I follow-up Based for wanted the is? Is are the there milestone, on your number of on current into accelerated potential FIREFISH, play approval the the Stu could on the to about a accelerated in approval? of you out a what babies comments think other for milestones bar you data supportive approval sitting motor threshold of of rate there just potential would your any emerging what is for achieving approval? RGXXXX. that current up the accelerated be supine think understanding

Stu Peltz

that the question to important raise we have as turn able that a able that's or the that, sitting. included be trial and babies and might milestones remember being the in development number as for the I heard this there over be is we - point three XX before, wanted that able passing saw endpoint in that an what the quite of in reflects to there since to so trial. maybe is wanted as the here would at of as now, really numbers. XXXX to said of to we you think reflects SMA being of well Thanks potential But and sitting us and before statistically increasing that number XX last are question in we being the we're Where three that week probably Cure able endpoint increased is were of that where of on for [indiscernible] always World that the for potential. five where would be we Muscle the think and reported top we're and be for this in meeting the that just we've time trial, we X additional we're of to potential more do that more that that the is were sitting, meeting actually from Overtime that babies at sitters number babies file of you've that Cure that able the that in part that seeing We was confidence think, since seeing SMA that we in –Roche within really the so part, there at a saying sitting reported babies consequence presented sitting disclosed what that part at Meeting gives significant at the to there June has are had filing additional had reflect the there increased babies were important. what that thought probably out the just time. part sitting time was X head, And I

that think potential patients. I the possibly regulators with us so look rapidly but how continue both such as see and that just make business safety additional they going for conversation efficacy results we about to exciting to obviously for it's think filing, move require to at fact So

Unidentified Analyst

data a progression of a combine defect some assuming results much for patients? expedited type type could type maybe biomarker same X study X the and would the and positive submission really underlying patients from of helpful. have approval for the sort genetic SMA in but Is disease achievement follow-up, X of in and all kind motor and data where there package know type patients? safety X quicker. so from since great. implications the have milestone that of type is with they potential a you X FIREFISH type that's have from I Okay, SMA X X pathway That's What I potential patients.

Stu Peltz

type is knowing space. others, would additional in a orphan Yes not what conversations how to I and uncommon in X X In they would question that see really disease see. the about again require that's think which good of the with type terms extrapolating to you think as regulators

SUNFISH certainly you? have then certainly So it's would have as Did be that would on ongoing conversation and a we an well. we trial continuing

Marcio Souza

Maybe add a just bit right. to little

So end SUNFISH relatively we be year. of enrolled to trial X we this the mentioned type our by assume expect and as for X before trials the

So data the that and collect safe sensing fundamental. and now going we like where SUNFISH we see dose this tell in SUNFISH both be X phase people for to part are

the So while they there the is different they terms in are so like the disease and comprehensive of progress of undertaking by the how PK being, of, it's expecting just but is. that comprehensive is of believe of exposure, of on fundamental and advance program one the they that. terms so so development much large data and our in the it number in and PG terms patients relationships We far most

you that to regulators altogether. with X, the we that X, you been us could already gives that confidence, for up X were type file if shortly find type thereafter So forward follow with

to going keep guys updated. stay we're and we you tuned So

Unidentified Analyst

me. it question. taking Thanks the Great. from That's for

Operator

Thank you.

Citi. Our with next question from comes Joel Beatty

Unidentified Analyst

in Congrats so SMA. Sean on for a questions. of I've is few on follow-up quarter, [indiscernible] had This such Kind calling Joel. got busy and

I kind going Can few we're it what a most should World think us? to just I be for review Muscle are looking endpoints SUNFISH. you data Society your we that see of for first, the think

Stu Peltz

that. for thanks Yes

as well. the in protein levels part overtime as well and X So others past preferably as RNA the and both we've SUNFISH we're measuring showed where for MFM that's

and open as looking well. MFM that but like it's there, things at label we'll be the obviously it's So scores

Unidentified Analyst

X,XXX describe follow-up questions hATTR you done just any program. on two I then if estimate us to have there? and just work patient give the Excellent population confidence and you've and Can any out briefly

Stu Peltz

don't a look I prevalence been through little lot there's what diligence go done? this. in at bit Marcio, due why and a Sure. of on, we've Yes think, you

Marcio Souza

there as Akcea me last that of so like key in research in excited collected so both partners previously data. genetic might months, and and far secondary into Ionis lot right their we They in the the a information follow we like And week are, also cooperation a last incredible during have know tremendous immunological we've Latin been press during say first we managed America when the look collects have research that of you diligence in we due are been has the Absolutely like working we how and network let collaboration, the was leaders mentioned primary we release the the to country. on several opinion information. labs that but think

rely ancestry, other in on but see is all the countries background South America, and just that very region important alone on It are if most the countries. mostly concentrated South terms slide patients Brazil considering example the of different for two we across so of the the in region, the in the for example just and of large Argentina I the mutation in in that potential is we the genetic like is, So background provided that we number how region. in and X,XXX about it feel confident Brazil [indiscernible] we

Stu Peltz

of about really important this comfortable well for between a drug. the this there's that good feel we area those we XX,XXX around But a patients area the notion gives polyneuropathy third pretty we is half that with and think feel the patients that really to valuable XX,XXX and

Unidentified Analyst

caught strategy just Thank talk follow-up. you brief a you and and Can a I to for bit event such heterogeneous that these a on little it with patients best is considering your disease? identify

Marcio Souza

absolutely. Yes,

we're quarters there we're development the lot of know that that focusing and this and of on where is really these all to of we HDTR in the being right genetic that really already large so space that field We're historically are Right patients terms medical accounts fairly mapping joint the all huge doing of here. the of diagnose that kind patients presence part a sense it's a what our is patients countries believe we're to neurologist difference. have Group, all to and have having where We large a we like now now. in is sites. there team being relationships a team in or a believe those to for and updating the but relationships make in of diagnose exchanged fit. of But We is in I'm of one we're future are going MSLs not like already key the of perfect terms have the in have boosts accounts these overlap going areas for Translarna terms developing competitive centers competitive outside we boost, diligence a doing. So be things these we ideas. directors advantage a to large clinical a concentrated is main treating like you the Akcea lot giving with and things that already updates, going a are right

we're So in much at sure of as are remain to the and before here least we few shared ,we believe make we be Translarna ahead to ahead sharing want since competitors going not we so. to we as years relations

Unidentified Analyst

Excellent. Thank Appreciated. you so much guys.

Operator

you. Thank

question from with Our Wang Gena Barclays. comes next

Gena Wang

just net I XX% in in growth five-year revenue press I'm compound growth think XXXX. wondering, the you release rate also comment the rate annual

of just at assumptions that do $XXX being assumption? what are $XXX I that think that for looked conservative in and the into million XXXX, terms million roughly in you So wondering you translate this in think XXXX are

Marcio Souza

got total XX%. or we wanted highlighted by just get so of future always with in we as population about of a of Gena, initiatives remarks you number mentioned look this we're that It's C the thank way you saw, a so that or of of the I'm patients patients. future general my the a lot patients not well. in reducing. midpoint the large before question. terms happy what I doing mentioned guidance but the rectified we alone done and for so progress extremely type terms into as so identified that in the very just five number right to the our all we're in much countries identify are there's One; in label prepared The probably guidance in operating to expecting, this yet, that talking like was I we're in those of

specifically we few territories where efforts assumptions people that in feel Akcea increasing penetration of of seeing our in help deal the conversion, So that we There defer put ground, Latin going to trajectories. East that the of intensified to presence. much a well as but now adding and it's in as on we're discussed and terms synergistic don't regards, presence lot and Translarna going just our before the with mentioned it's be very we we're there, are be Middle America identification with both to more the the like terms recently in right are more they We what well. going the are vision

are us label in be So drivers get going approach on sometimes key The thing robust I on, identification, of its We're EUC expansion gives key is what so way to new sense patient fairly this we but this, here. in lost that the of number and ago. growth back and multi-prong, the still seeing countries extra geographies interesting on a launched XX the confidence where years and patients identification even believe long-term.

Stu Peltz

working we're depending then, And now things I your on the is, for upsides we potential have point there's like think one non-ambulatory. good the

the remember could be of considerable a trying of was If data quite process we're to and that get label that the upside pulmonary really be expansion could and upside that. so, strong of in you XX% which terms may in function

well. potential greater as yes growth So for there's

Gena Wang

one just Okay question and pretty related also seems then flat. another Emflaza revenue

new happening but patient wondering could you this detail a compared conversion if just seems to don't you bit the also you growth? if slow, quarter commented. provide. can to how you the I terms little just like quarter in know It much I think sense give new the the of patient can how last give a of drug

Stu Peltz

Marcio, you a thinking on? color little And don't give why on Sure.

Marcio Souza

Of are this Or that drivers I impacting here course, drivers may quarter. here. there's three so say two

mentioned mentioned to the what net little fairly We our the on But more seeing between is, quarter seeing Last that seeing. around is between still first gross launched the on we're higher bit else one growth a the a expectations for net to mentioned we're on quarter we patient prescriptions house, to second gross ends, we six adjustments. we year. is, after similar commercial that so we quarter-over-quarter. to to the both months. so end the the when well time bit expecting prescription be about this in was the before, the So and little , the lower gets quarter, we're other the We're and four-ish time six-ish we're have the more on last get we seeing what patients product more as and on in of conversions the launch conversion on now and months. In three in expense and the three months the happening

our timelines within what's sense conversion. six conversion months expected it's in happening skewing but towards is, So the a

now what help So and basically focused a go as in patient-by-patient right that. very we're reduce you them way, doing

not their accounts Where we products have we hundreds them they So and right for is the confusing seeing a working all some it's but for where Part bit them, be seeing already with to the D, now to so little more patients and to making is which support to that would everything need those neuromuscular have some We're B last manage never on they several like now, we're that intensifying they weeks. those and working that something a now products of in it. bring with than sure that, bit prescriptions some multiple where are There they we Part providers. we're of new had we're movement have are and average accounts, of place them we're driving. have we're little so

where end were year of to on improve we the beyond to [indiscernible] the much to get where that wanted before. pretty we to where expect be and We and

Stu Peltz

our Emflaza Emflaza thing that that steroids prednisone quite the to superiority the and as over have very One then comparing the that then of data understand was publication was of and Emflaza recent history well. well other useful in showing to that's position differences helpful and two recently showed the synergy been study worth demonstrating are really And noting been the national just better publications much probably over is, well the how as there's prednisone. payers placebo of as improvements between the having that's been again prednisone

of that it sure the pushes through get large of So all have it's we're funnel. of the a number that to that hard in we we're to trying it funnel, working prescriptions already make combination through

Gena Wang

helpful. that's you. Thank Yes, very

Operator

you. Thank

Our Bank with of Ahmad next America. question comes from Tazeen

Tazeen Ahmad

just Akcea. your get thoughts about to wanted your with I Stu partnership

that very for doing option clearly. good about sense more time I I guess are a at one patients thinking some your over about you're there at will You very get be to than this available launch of how people. some just ex-US launches wanted particular that you've point given. likely this drug as unlike it's demonstrated community in opportunity ATTR of with

Stu Peltz

Sure. Maybe this. to could I'll I touch pass Marcio on on as well.

unique a really there has this that that and form for that. is is there it's I of this not there it polyneuropathic is more that form some we're the of fact positions way where obviously. a I mean which infusion a almost and Portuguese it about really and it think particular a disease actually injectable because in of well. of advantage major this infusion It's it's is knowing excited area in really in in versus Its unique lot the as the support

is that's of group only the due the not deal looking and how well the good really and at comparable a and it's gives and be the similarities suggest that a it's believed - at we've that's there administration really work physicians and is the the advantage. of major there's done looked the in it, quite this simplicity but advantage administered and So that as have efficacy efficacies diligence the the having to major but similar, really [indiscernible] going so the it's route of the of, drug around this drug

Marcio Souza

Stu ability The seen of of very thing like top complement situation and is have successful to all compete America many interesting here and us one did, how more around that came ground has just situations [indiscernible] the like on from to being is competitive the being all understanding all we team my and bottom myself one in each a And on several just the to bit on look or that. down like key included of team this to ground going one how right. deals way and we Latin our the exploring saying little what on in we're mind, and competitive us to to is

key So components. that was a

right. absolutely You're

expect this hospital FC drug is on competitive. to of for the using an be versus having a physicians, example the of are to an for we'll multiple the believe We for have patients acceptance the one, reasons, make time this to and [ph] compete people patients interest We be to have steroids make the the But administration. successful. happen the able and for going that thing it highly drug comes being before sure down TTR on motivated that's grounds to this to very we we're importantly make it advantage

Stu Peltz

a last months that that complex one I probably on, there's that's that Marcio you building. touch little spent we've system think [ph] And labs license in important on XX it's the Brazil a XX should to requiring and

You want bit that may to about well. talk little a as

Marcio Souza

Get everything and the that's needs that we to box, systems several on diligence, in forward all right to established is very deliver compliance are time that be Parts and far the Right to we why very considering relationships patients Because are perspective now end we're just all drug from as established environment to and there. advantage ready. takes as due of this. to as It we the we're we the we going there America competitive we thousands day the well make through in long believe at here run proper mentioned we you're just our just have place, make patients. as all them. checked what Stu confident can that is the of the doing theme sure how license, there Latin of sure the provide

Tazeen Ahmad

do this your gene estimated could I'm you're therapy adjustable long this and you how you and for it about, all that you reach think going long to take sorry think but color if you thanks estimated success that talked addressed address it you How you earlier, population for? take similar how for to population. Okay to will

Stu Peltz

the identified differently Yes awareness. of ground the overnight that with [indiscernible] few doing where up and happen not to biggest you're here we're that's that is terms one are is The in really from to hATTR believe [indiscernible]. I going very the patients referring the and different really that deficiency like, that

data patients, of family. lot If or you Argentina America, have patient specifically Brazil have Latin the and seen three someone a in go patient had to have

so mostly they being we of and awareness. different have So the be on It's sure network, so it's a faster. terms making general to bit little that referral so they type forth, correct expect in the

one them, are to the at conversations so don't is best put taking on deficiency, so it few for Now little bit precedent are effort see other, patients, the a same on with the much of and the more not both so but time the difference multi-year necessarily logistically than months. process going and we governments there AADC we're over

Stu Peltz

go was more cerebral epilepsy where clinics the that to a palsy, because - where large, and we we'll and finding is AADC getting them this of onto be. it's in case, about deficiency trying severe those we In Yes, the it's not clinics, find these being countries Marcio is, a to drug. those high, patients awareness lot quite as said is and will genotype think really

think as peak. it they're to So as all as get different with effect we to time orphan take some disease much

Tazeen Ahmad

color. for that thank Okay, you

Operator

you. Thank

Blair. comes next Our Raju from William Prasad question with

Raju Prasad

maybe will kind of the maybe color take to and continue Translarna? something there on for younger peak? provide is it or will some commercial patients where EU where you've and several you on expect to of how the ramp it identify and available patients bullish as in some hit Is be develop got ramp something you sites little of countries you commercial a on then, the slower bit some Can early XX of it patients time and already

Marcio Souza

question. the Raju for thanks

on until very now identified necessarily is see patients It is was individual the mentioned green very that several made, as bit have of patients the don't really anywhere But of to Requests the right. the [indiscernible] we few going certification progress submit off through submit there We probably made to and [indiscernible] already, little the progress. that with time this dossiers. is of we light lot is happening I So said, but pretty and we're are recently to until I'm my before a joint all patients much as take happy this. of going them accounts wait getting a to some [indiscernible] countries, are are patients. I the and that. rules had that for

So that than where phase in someone's we're be to granted faster others. now going

way enthusiasm to be we in what relatively which and launch going that is for can this. of time fast, takes It and this priority our right staggered system. a patients this going penetrate accounted it's that to in projecting that's to confidence but us for system when the just we're some gives to sense CAGR get from point where maximize the you're is perspective getting lot our Europe the is So this There markets it. where physicians the through and

Stu Peltz

and of diagnoses changing work lot and years that to hard - But in then or some in only requires about a we've two awareness actually things just the efforts doing a of not it do let disease because me is much time also that so genotyping, think average years Marcio that. the talked as they on months one case the team of tremendous I is kudos six to the but four change which

for that's consequence, they help that where the starting even when going been a taking all younger are to they've as there muscle wanted think not more got that's as the think decided to are when we've sort well. then and really - so, will we we there's efforts of them four and through earlier on up more find we therefore be they're so because earlier that or work picking then that well so the notion think put that's patients known younger the patients I them is, out who've bear to general on patients physicians be, so it's off And to they're that years, hard paying done actually

Raju Prasad

kind But study. favorable trying and any level design. can there? discussions are you noticed of of with playing Great anything on get to update dystrophin the FDA FDA, was, guys and study comment the high you there on I kind

Stu Peltz

by anticipate I can we've be study the think to year, for the think, We completed what what we're Yes we next on be that the end year. that. We'll filed approval end it I in is of this by past then do where track said comment starting of and dystrophin accelerated the that.

Raju Prasad

Great the at some the the you comment and that specialty show SMA you can would like guys then are just of hospitals is you in there to anything or that with obviously other patients of one. types diseases on Can at, rare leverage? nature of that that expect AADC you overlap the last US centers up just looking any or

Stu Peltz

you do want Sure talk to about little before? that and Marcio,

Marcio Souza

are happy a job before there with Yes and opinion met and that on team of relationships has they've we everything course, key [indiscernible] couple established Agilis done done like of have the tremendous leaders. targets so with we're

few did, mapping that next going So be this going I did clinics are cerebral States that's it to be not months focus to general is the say but only the journey key that patients the seems in for on globally that be in the would palsy concentrated. we patient the to by where United the and most by work Agilis

crisis, epilepsy are there. have So or we're epileptic epilepsy, severe going actually and with don't they to focus you're going to patients but diagnosed looking of these patients misdiagnosed also be severe there, with

the you're patterns, overlap to starting doing that to biggest efforts some BioMarin I of see with think diagnose patients. is the that the is

that's to and the some a base the a starting raise that and little that's like So disease, effort CNS they're of there of tremendous those we've been spillover physicians helping. disease of on of doing some There's genetic job bit hearing on there. identification so awareness

on overlap in there. don't' that little care before a States countries neuromuscular treating in the several SMA they're of differential specifically is of of very outside physicians then centers. terms or nothing good the essentially it's to CB overlap. [ph] epilepsy you going the where general and probably We've So you're not clinics but the more more United the done the diagnose see pediatric US on taking the little in the a expert ENG the and a But neurologist of of have neuromuscular neurologist bit some

Raju Prasad

you. thank Great

Operator

you. Thank

with [ph] Suisse. Our next from question comes Elster Martin Credit

Unidentified Analyst

the press on some of to I release once be run questions earlier couple some or on there placebo question this expectation benefit I and Thanks. perspectives Do expect out that required I at versus the then benefiting Spinraza that of RGXXXX. conference functions think regarding till that about follow and around part nine that's to should the kind the how the X anything seem separation. think of time FIREFISH a the of fact. data The of ups had is the it SUNFISH, endpoint. reported month reasonable to functional is make achieved infant a is study a it's Spinraza from number will of study, you the separate you your from XXXX a will us drug your or was guess kind that dependent? And that's different? wondering of differences something timeframe see completion do about sitting With up Spinraza public

Stu Peltz

the for Martin, thanks question.

so depend a think And will of part about pivotal be part second I think of be we'll I about. study, it before But FIREFISH the the of X lot the so able a how question. The label obviously when is is the we high first seen. talk FIREFISH obviously a part XXXX first study that's question part on the, good open think an of to into think we

in we to line So to move what's first partners part the forward happening what's the with best how presenting our we'll putting talking in that. about and that way and that start on have with

taught we interesting you're be In patients previously show to line way at where earlier we in I even able even can in the actually and going FIREFISH RGXXXX. [indiscernible] the dose models the that's might we just work within on pre-clinical that's I be of in question, look of that that much the XX that very able to the a older later and the that. see in quieter and when from sometime, recover the think even On animal a how that we the the you faster see it's especially data side, were of are some to parts improvements think chop SUNFISH some to seeing still study, from patients data

it's a quite that will think how that - So us, only we hopeful we're X case. the I tell think bear and effective out and time well will type molecule X. will be But if the in that high

Unidentified Analyst

Thanks you for your the appreciate I and taking thoughts questions.

Operator

his to would questions further not showing in Thank final any I'm the to the like I call Pelts remarks. queue. you. Stuart for And turn

Stu Peltz

franchises. Obviously is then commercial to integrated rare deep company build Thank leverages these the programs Really along the transactions biotech of all capabilities. The value you DMD I a provide think expertise and does really next ongoing obtain joining. oncology to continue that number a for what our creation will scientific world our with opportunities fully splicing vision through two disease niche over class years.

that, let being again with all for me you thank So call. on just the

Operator

all you And and concludes for the ladies in program. a thank day. conference today's and Have gentlemen, disconnect. participating you wonderful This may

PTC Therapeutics (PTCT) 8 Aug 182018 Q2 Earnings call transcript