Amicus Therapeutics (FOLD) 11 Nov 192019 Q3 Earnings call transcript

Good morning, ladies and gentlemen, and welcome to the Amicus Third Quarter 2019 Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this call is being recorded.

I would now like to turn the conference over to your host, Ms. of President Vice Pellegrino, Investor Relations. Sara

begin. may You

conference Good Amicus discuss corporate our financial morning. you for highlights. Thank call Therapeutics’ and to results joining third XXXX quarter

Officer Executive John today’s Dr. Officer; Operating Hung Head Medical Therapy. Officer; are Quimi, and Also Science Chairman we Dr. on Officer. Daphne Campbell, and have Do, Financial Castelli, for Speaking and and Chief call, and Chief Chief Gene Dr. Officer; joining Officer; Chief Chief Jay Bradley President Chief Portfolio Crowley, of Jeff Q&A Barth,

referenced may and As plans Private make statements within business, of on Reform XXXX our our Litigation as prospects. the of forward-looking as slide two, well to relating we the Securities Act meaning

any of by plans or we to of achieved. or Any Our turn forward-looking call can the be be our affected inaccurate risks and as statements forward-looking not that be will all should made wrong uncertainties. and out regarded by may unknown assumptions on statements be known this or make us representation might by

and to to cautioned this to their the to undertake and forward-looking any which statements, forward-looking are reliance events cautionary on reflect obligation update circumstances conference place only You hereof. the statement by date or are no hereof. qualified speak All entirety revise statements in the or date undue call we presentation not after

filed be the on today’s today’s quarter quarter SEC tomorrow. on filed forward-looking such our you December press XX-Q and Form risks of quarterly with our XXXX September the for discussion for to on call uncertainties impact and the Factors full Forward-Looking holiday corporate Commission, which and release the Exchange year to be refer and XXXX third XX-K materials statements Statements them, report the XX, and ended may a we of ended and Form following XX, For annual the report website, the section from that Risk available will Securities

the turn Crowley, pleasure Executive it Amicus. At and Chief this at my call Officer Chairman John over time, to is to John?

results third today’s conference fourth our call Great. beginning and to Thanks, everyone during XXXX Sara. call. at And early and pleased on progress I’m into slide made efforts now to third quarter quarter the the the conference quarter, three. to tremendous highlight that we’ve Amicus welcome host

continuing A next rare vision very the a To with in York, around successful great which month to achieve our at our ago, Galafold building, living New detail are we grounded of thousands Analyst the impact company, we grow biotechnology in will moment. diseases. what are Day this to global everyone we base for business, the many poised people in world vision, Brad

our We disease rare investing in including Pompe therapy of our AT-GAA, pipeline as gene the and Phase also portfolio. well are as leading X robust our advancement pipeline, biologic

us sheet balance self funded, major milestones, the very maintaining clear strong well to and discipline also profitability with a through keep all half first into We’re line fully upcoming financial sight and to of of sustainability. a XXXX

volatility of of achievement Again, move Amicus the plans, number in extraordinary results. October of quarter, execution the let today. again financial our during the our Amicus So, and and markets it’s intensely Day third Analyst team on four. highlighted to the the me now focused slide the Despite at

of the efforts. commercial growth across our was and For significant third another Amicus, of science, regulatory period quarter clinical, and all execution

are Here takeaways. four key the

medicine of most third cornerstone quarter remains the First, Galafold ever rare the our for million continues to successful be disease revenue. success, in with $XX.X and launches a one of

$XXX compliance a at of more XX% rate, million. $XXX We’re we now upwardly also patients than now plus X,XXX have and guidance adherence to revised full-year and treating XXXX million

others. and or that therapy, we approvals and base, a new reimbursement and new on adherence are All exceeding among track including target access, of to compliance country the targets, patient broadening metrics global prescriber launch starts

first investments the sustainability. world around leverageable. strategic Secondly, Much scale $XXX outlook will self for now from now XXXX and to be be profitability in the million we of and into development and growing to completion infrastructure review, by and runway have and a significantly cash of and and research the path us AT-GAA, current that following our of approval revised financial of well of Galafold significantly to offset built will Galafold, deliver business strengthened also the our half with is also up getting prepare highly revenues our on

with patients XXX that that this WuXi on our are the PROPEL a patients a into the is continues reporting Pompe call, successfully disease. significant believe study, disease track by about approval fully of we next remains for of for and been our becoming Today, initiated in be we’re generation Pompe AT-GAA, are toward now what Biologics. to which enzyme we standard replacement people partner care living PPQ momentum our runs jewel pivotal is This end now will year. have Third crown portfolio. the today’s also key therapy announcing of new enroll XXX enrolled On that the with

of future program well. to and here Advances in advance. clinical portfolio it foundation technologies the third and CLNX Batten disease, as our a gene stage interim CLNX our therapy in include continues finally, programs fourth, data quarter provides And gene for positive the

is tox we at candidate, have gene differentiated Pompe gene selected announced a forward last moving therapy studies. Day that candidate therapy that into also a now Analyst We month highly IND-enabling

investments to be achieved R&D, position, in ahead. in by outlook phasing sheet, we actions for taking as also We savings highlighted This the there, And categories, Galafold, prioritization discipline the moment. operating and will already and expenses very projections call, today. further and all financial of includes emphasized our will in balance has a which a of path number Daphne at strategic including expenditures, operating with increased financial and to our realized business strong third spending the review completion just I our that expenses, the of capital quarter. of SG&A expenses capital have highlight beginning The the and of financial which program strengthened including include revenue

Amicus’ now the Daphne to So to me review go as guidance that over and with outlook. up, the financial set and ahead results, Daphne? let call turn

everyone. Thank and good you, morning, John,

Our statement slide for financial ending XX, with period September income on three-month the overview begins our XXXX. six,

For quarter XXXX. at of $XX.X revenue of increase is the million, measured Galafold a rate offset which of XXXX, currency of by impact of includes or year-over-year achieved we $X.X the exchange X%. This quarter an revenue XXX%, third XXX% growth over million constant negative currency third operational

in products. of period. the to favorable royalties. XXXX, year sold a do percent Cost States a was as continues XX.X% of the royalties costs of third revenue to as grow goods royalties, well associated of prior are in the Cost revenue manufacturing percentage the of as for owe not of subject quarter goods compared sale includes net sales our XX.X% Cost was as sold Galafold’s as with we we as lower where to United well other goods where as sold countries as of

significant portfolio and We continue study Pompe R&D pivotal and in investments make ongoing and gene our of as scale-up with the capabilities. to program our therapy commercial manufacturing the well in expansion as

payment clinical million pipeline. The by study the therapy $XXX.X Celenex partially an gene $XXX R&D reported quarter the XXXX third continued investments year and decrease the period. for compared we During $XX.X for upfront million expense acquisition offset is the Pompe asset program of million in in to due in primarily our prior as of to XXXX,

XXXX ongoing Total selling, geographic of compared for activities as administrative for was or year States. commercial quarter compared third in $X.XX $XX.X the to period. expense of Net The share, or of and Japan the third and $XX.X launch, for quarter per to Galafold was general scope million loss XXXX year $XXX.X United million the the prior million launch net for expanded per of share prior period. the loss the $XX.X including $X.XX million represents as the increase

we of million approximately XX, As outstanding. XXXX, September $XXX.X had shares

seven. slide to now Turning

into As of John first funded half we in continued milestones fully and are mentioned, global well the upcoming through our now growth. portfolio XXXX the

of a identified and that our our integrated As where priorities and our all programs of we considering programs. business we cost new review we advancing business the normal drive course of efficiencies areas while to all part top strategic completed savings, have and investments into key organization, our

our are following extend we result, runway. taking the a actions that As cash

revenue will increased trajectory growth higher previously Bradley and as than forecast, will support Galafold have growth confidence we highlight continue around a have to We later.

Through our implementing are we and within have contract development and partners, identified internal and therapy teams synergies efficiencies gene and manufacturing manufacturing.

measured gene growth approach external capital in continue we XXXX leveraging We and internal as build beyond. also a out management XXXX. two internal our plan portfolio take process and starting to to therapy Amicus expect INDs collaborators to are one we through grow, to resources to manufacturing phased prudent expenditures. headcount internal for And facilities, We support and other

measures and these the implement recently some have impact quarter. already begun in third positive We to have seen

leverage gene expenses pipeline. we that emphasize, to XXXX of launch associated commercial to development flat with in programs our to to we place in operating again, XXXX forward, transition firstly in the therapy our products global remain and is pipeline; the AT-GAA already for the Going secondly, and other the infrastructure cost total expect as in AT-GAA multiple

and financial Moving position June total outstanding Cash, equivalents at about compared cash inclusive the September shares $XXX XX, and The marketable cash current comments securities of million $XXX on position December to equity cash XXXX XXXX. totaled XXXX offering. XX, slide eight, at guidance. to million our XXXX few current a and are

non-GAAP XXXX, we are reaffirming consideration depreciation. in expense of share-based SG&A guidance upwardly $XXX Galafold our and of value addition to operating $XXX development million revised fair non-GAAP of Looking revenue We to and define to $XXX our at as contingent and remainder expense, $XXX of in million the research expenses, full-year million compensation excluding million. changes expense operating guidance

Total quarter-over-quarter. operating were slightly year-over-year down and decreased expenses

to be expect of growth the to the measures of at we gene reflect moderate fourth and ramp-up AT-GAA, reductions PPQ quarter therapy our runs operating CMO for rate of higher We have WuXi partners. total cost of to Some control taken. that initiation do at the initiatives these the reflected expenses manufacturing our and in

anticipated revenue, runway first previously again, years of year our $XXX to and the range well expect to into ahead. remain a the XXXX. we XXXX, $XXX expenses the provides Galafold cash $XXX generated at operating million which range stated end of from of Considering cash end we that approximately half And net expect to million in operating have in the to expect We annual non-GAAP expense the full-year constant within million.

I’ll outlook, nine, slide takeaways for Analyst our key as summarize On during financial I our highlighted Day. the

are through milestones. major upcoming we mentioned, funded As fully

or have We remainder for or the the financing XXXX needed future. foreseeable planned of no

expense. in be believe our investments XXXX of end We largely as commercial we made behind end These to infrastructure. global is year this build and AT-GAA its operating investments development, our significant have R&D the towards through the will of by move for manufacturing and G&A as us and well non-GAAP peak that

mentioned the AT-GAA will support I products built of future a maximize effort. additional modest to we commercial earlier, leverage advancements we As through value only the program investment have and organization the with required

non-GAAP annual this to expense within total remain XXXX. million relatively flat XXXX to is range expected to in operating $XXX Our million $XXX

$X revenue over We Galafold in expect by offset in the operating will moment. which same expense be global Bradley a the billion highlight to significantly period, cumulative projected three-year

execute for not planning tools, we business believe to As the in technologies, activities set partnerships be of right are foreseeable development what any future. and significant the we we

Galafold I’m now, I’ll happy highlight to address the for Q&A questions Bradley launch. any turn but during to to it

Thank Good everyone. morning, Great. you, Daphne.

end on the the on of achievement our the quarter of Galafold provide for X,XXX and more growth third color in XQ. patients therapy me on Let at continued

$XXX get to global many patients additional Our the revenue sales we as possible. medicines this as in mission in $X quickly and key thousands is milestone, potential billion of increasingly Amicus with to million drive the adoption confident Galafold along are at our And patients to in opportunity. as XXXX in path to peak

increase global the again snapshot been revenue, countries. the momentum and million, launch in with exceptionally new XXX%, steady earlier mentioned, has XX. growth strong Japan in slide third year-over-year driven was Total our by U.S. a including of begin Let’s as as well countries, $XX.X quarter on a as

within or million $XX.X States of XX% revenue million coming United reflects from outside this United and the remaining States. Specifically, generated XX% $XX.X or of

XX world, the new around We now and this quarter have secured three Korea, South in Greece additions Argentina. the and countries pricing reimbursement including

global countries in to approvals, This the will We ongoing replacement expansion therapy important an to already growth switch have continue delivered more forward. these we the more to pursue uptake Galafold and to commercial continuing in market, support driver enzyme which get and world around moment. patients with along patients geographic I’ll in approved going opportunities continue will team increasing be highlight a diagnosed untreated from to to and the

positive the momentum me Turning now all XX, comment this across quarter. to key focusing metrics let commercial we’re slide that on global on

global amenable Galafold as we as patients market patients, new global approximately add is share continue First, from up as we treated XX% in at to switch which untreated now the quarter XX% of level, to patients second has Galafold. the well previously estimate

Next, John to what reflecting mentioned, strength as globally, XX% and precision that is adherence having oral medicine this the exceed are of the compliance and experience Galafold. we to continued physicians with think patients

continue strong metrics, to we Japan, highlighted see throughout and the be to world. markets well smaller our across each of the and continue patient starts including New slide, the strong very midsized on our U.S., performance this in regions Europe, and as

This therapy, XXX And treated. XX% taken previously first an basis, also About contribution a Galafold XX% their treatment Fabry. our increasing we’re very is patients as had patients from while September Galafold replacement Globally, as global X,XXX-plus untreated of of for from previously on point. patients. were of on encouraged very not XXth, by important the switched have

actually overall market mix for medium as term, approaching of XXXX we is we’ve term, actually about in patients prior to previously increasingly may longer In with and And untreated patients. care three have naïve we Galafold mutations. Fabry see years of launch the of So, Galafold a amenable grown think switch the in line the treated Fabry standard treatment by a majority been short that the first X% Galafold. becoming between patients

Turning XX. now to slide

quarter, growth quarter some $XXX specifically, $XXX me in previous uneven contributions offset In our and helped performance of Japan, continued sales. patterns supports the the third and the years. third Europe and in year-to-date to million quarterly the increasing that the in Let to we’ve U.S. revised the upwardly guidance seen global recap the of ordering million of that

exchange negative cumulative for highlighted $X the briefly which us $X.XX around million in range. the to would that $XXX foreign year, million XX-plus do about million QX, expected each Given of as middle $XXX $X we trajectory quarter that’s impact to in million to GAAP we revenue revenue be upon being $X put the expect will year-to-date, of think for of FX that And in impact million remember, million before. about which inclusive guidance million will

business again, with So, coming far so great performance year. the base tailwinds in strong the of the end into

to now XX. Turning slide

and in to of are on we XXXX. launch three years now can first performance the say opportunity behind sales these in With the of U.S. million confidently us $XXX including we year path that Japan, a

$X million As revenue get rate just long at OpEx a the Daphne way Day, that XXXX I and towards we’re outlined and we goes expecting XX% and about to between Analyst that year-on-year growth to in cumulative to R&D generate band, funding and XXXX that expect described. billion alone, $XXX

and overall $X.X the screening, just diagnostic targeted initiatives, confidence We we’re that in the invest Fabry at the $X newborn which as revenue put continues the Fabry population context, beginning to in other of to further particularly also in. billion-plus And was peak. global market about even through opportunity increase also grow, in have to XXXX, billion and

a in Europe, takes the orphan of coverage give us XXXXs. in as finally, listed and the to into Book late our end to which exclusivity addition we us the that Orange have patents alone U.S. XXXXs, And IP the reminder,

with updates turn as John? our AT-GAA portfolio. to John So, program Pompe back for and the gene me therapy that, discuss call to as well let our

Bradley. Thanks, Great.

we our of detailed just October know, you review of many as entire portfolio our during So, completed Day. a Analyst

at which the on today’s can a we details So, of the webcast and reviewed review and is course available that new Analyst highlight of Many I the Day call, briefly be event our key transcript, website. of takeaways. for on few information pieces will the covered

product, XX disease. for for with on slide the breakthrough second Pompe lysosomal as ever. Pompe BTD here To the first AT-GAA this everybody for begin crown designation remind jewel, our only therapy again, I’ll and well storage ever any is So, generation therapy as first disease disease

There three care, the what $X people and standard population with for again products, with we seen be next disease, We’ve may AT-GAA be believe updates from plus call. Pompe on new tremendous today’s potential what of respect a opportunity. broader could believe are for we to billion living important a momentum representing of

now the reporting foundation we These CMC module Biologics. to approval the WuXi First, at today pivotal have will BLA a on we results from for a that will begun are continue study. on the the serve anticipate call PPQ submission, as runs for be full for which based PROPEL

enrollment, Second, patients this in the we now enrolled and continue we XXX strides target the to make PROPEL study great This study. is in great achievement. a have

the global for physicians we now meet year-end. Analyst patients among across on As that XXX demand complete are approximately in mentioned sites. remain at we and to extraordinary study enrollment Day, And to this track order targeting participating by patients

including broad for AT-GAA. supported pediatric continue also potential to a support We our our studies label important study

patients Phase Pompe medical of study for study. matched have our is the a we of AT-GAA patients We the of study first with to our natural present with third, to treated details also the half Amicus approval. support replacement upcoming known XXX treated we this data that in will And an enzyme full plan high-level meeting care therapy at POM-XXX in from X our submission XXXX with the BLA of standard completed history assessment plan and are natural the who history as retrospective

to during you some enzyme literature, the the highlighted care should expect Day October. be natural replacement history consistent Analyst in standard Importantly, that Amicus with this published in POM-XXX data therapy the of in on of the was

with on engage additional if together while to after that that and any full reminder, And study the have regulators regulatory frequently or the everybody program, approval in we’ve not occurred only will our of that a assumptions study, case. base remains Phase the collected interactions support X/X base case As this materially impact they future AT-GAA. we data on be they I’ll pivotal PROPEL any color pending remind expectations our providing we from or this until with

extremely valuable with be and to largest could as gene the therapy industry about the program to patients excited to be disease what build well most AT-GAA with optimistic and offer globally. as believe continue Pompe all to the We in we Pompe franchise preclinical living potential our solutions

highlight slide of rare portfolio here Moving for to I’ll on industry-leading XX, gene diseases. now our therapies

Motor A form seen First this our which Hamburg positive clinical gene data & this we’ve scores scores combined meaningful on includes the demonstrates motor disease. of to analysis that Batten of support in vision continues the language, in disease. of in primary of impact function Language devastating data to and our of to therapy interim study, devastating, neurologic the CLNX, fatal now and individual halt language progression the AAV initial component our seizure addition in interim gene potential therapy for motor, positive

now CLNX opportunity which peak of provide $X neurodegeneration. of the our our disease, and clinical cohort, initial course the Also, read results most this received in have both, CLNX Batten CLNX we and represent CLNX together cohort. also disease Again, CLNX through childhood the Batten franchise the And form to billion with help Batten designations for and addition a in dosed CLNX thousands orphan in dose Batten recurring that patients study children. for higher and drug safely revenue, combined our have EU we program, in common may U.S. of CLNX in important is disease. programs, robot provide to

toxicology now clinic a we into declared XXXX. IND-enabling at mentioned And now to As therapy have the studies. this potential gene Day, candidate the program has enter to Analyst clinical in we Pompe move also

colleagues that can so work protein effective are and Here wider may secretion program, ensure we UPenn targeting a we expression, focused have our we therapeutic that window. in other on optimize at this with to engineering and

believe programs. future collaboration, have our collaboration this XX in And including our we engine, with we preclinical an with finally, growth to R&D to plus diseases, rights fuel Penn, eight

call of on now to XX following updates, priorities, which five of program to are meet these So, key on track slide reminder all we conclude the a I’d exceed. like our strategic or with

Galafold went us again as is First, trajectory $XXX Brad confidence detail our it peak by that with with believe XXXX that what guidance puts on the we Galafold sales Galafold. the successes toward XXXX we’ve a and in billion and had into in $X million potential with for

of AT-GAA, again, remains crown jewel which our portfolio. our Second,

and degree continue strength are into part UPenn. the high the very sharp as too, financial grow well highlight financial have a to our PROPEL the franchise have Also study sure we’ve the enrolled, need CMC BLA. all or make therapy and to preclinical continuing of seen as we’ve capital in too, enabled raise we across of clinic, confidence Batten in of the with discipline, very I’ll seen, XXXX no focus now We milestones the while collaboration the we’ve funded well and runway, again tremendous we importantly now that progress we a Again, now section that in that extension anytime gene on programs soon. our with with our data of our first XXXX. in cash the And company now here to through major fully manufacturing,

sustainability. capital, need the we we and gap we capital self need close and additional we years multiple that As sources and think and we think non-equity to come that believe linked could path also is it’s small to sources in profitability what on non-equity very from a the outer that as

turn slide again the XX like just we living before conclude on call rare on to finally I’d with here So to the people to diseases. just Q&A, focus over

and And We deliver to we’re these and mission new significant people are here of families. enormous thousands patients shareholders. a alleviate their of for our many bring amount value to on and fighting to hope for suffering to create to

questions. So that, to take happy with we’re operator,

Anupam instructions] comes first question from Our of Rama JP [Operator Morgan.

line open. is Your now

questions for update A us our Tessa Hi, Thank Batten’s program for specifically. few here. all. and on the the from This for morning is you Anupam. taking this on

we as program Thanks about so the remind remarks, next update an for for program, steps And CLNX? next secondly, much. regulatory then, First, eye us strategy about can thinking dosing thinking in here? CLNX complete, towards noted with should CLNX with you for how the be of specifically prior latest program

Thank Tessa. Sure. you,

questions we CLNX comment the Jay, you for next as and what CLNX, I’ll for see on steps there. let So,

Sure. initial and -- be with patients course the we dosed Hello. the aim engaging the study We in CLNX steps discuss regulators have data of additional with to the that dosed of forward. the have patients the able interactions. had expand And plan to But, after study to study have the those disclose CLNX. clinical have be include we now move we with cohort additional that’s to that That’s actually something patients. that cohort ahead will say we the of have then for we’ll also and in that

we will we additional -- in stated additional of as be the low data And same patients, regulators shows that CLNX, with seen we safety low material additional this in forward CLNX the X/X the the as the that higher initial as towards discuss for dose over For the with John moving is next in before, anticipation well. again regulatory presumably with patients have the body the cohort available of would cohort what is something the dose Phase dose. dose get to once we’ve in dose path dose and at the patients that happen the that year. dosing when And higher study, engage

would both just I’ll of of effort in technology a in work and also transfer, all Paragon, that highlighted, And the requirements lot the to all any amount will that of respectively. necessary filings regulatory the on CMC addition and well Tessa, is to to clinical Jay involves support BLA work these a a that here. these programs under be to one of Brammer which thing add now both the discussions So, focus way for has the again of to highlight significant programs

comes of next Thank Baral Ritu you. our And from Cowen. question

is open. line now Your

Ritu. for [indiscernible] for is This guys. my Hey question. Thanks on taking

one you program. out done me, by Batten’s on cohort? are wondering, are wondering guys is, just runs possibility Just initiating or of potentially Nationwide’s for in you things at ones And guys would year? second that I’m also building the starting next terms submitting I PPQ quick Is a going thinking WuXi, rolling about being the thing really for are considering that. a you’re there how history. how BLA be two Just Thanks. on natural know

to is natural these I’ll of Batten’s think going our to about Head very Jeff Sure. ask talk be basis which for of Gene the Castelli, important the we programs. as Therapy approval history,

we are with where those? of Jeff, So, both

morning. Good Sure.

They think, already as Nationwide. provided data study seen the have an of compile at in Day, has I data. So, come some continue initial That all to that you’ve history Analyst ongoing trial. we’ve natural from

several sources We natural identified also other history have of data.

collectively, we get the matched we will XX of any kind very can so So, which dataset, be are or robust very think optimistic patients we of a comparisons. history doing for ballpark natural natural for in

have there, with Sure. to provide And just potential the to designation well. with particularly as for able your discussions for with the an questions think ongoing a update this program BLA, section AT-GAA, runs potential on a breakthrough again the heavy a we’ll CMC regulators. to that comment going therapy there rolling focus we on not Once BLA the clarity PPQ submission to we’re begin. initiated But with and we is on again, be a

comes SVB question from Leerink. Thank you. And our Schwartz next Joseph of

Your now line open. is

the update. guys. Thanks on congrats Good progress. all And the Hi. morning, for

So, touch first Jay questions for upon in is This disease. Dae Gon Batten’s can maybe Joe. Jeff, or me. we two Maybe for dialing for if

So, update the in visual what three six. we about when remarks, mentioned we your at latest regards to acuity presentation, scores. saw Child the we wondering sub the prepared in like saw was saw the of were thoughts Particularly, I out some that the with Society Neurology you

seem patients older it’s to think, progress. I that

And, this the But, the can at you for Thank And therapy? before you sort candidate additional us, I administration CLNX the intravitreal ASGCT? into very on understand candidate? of on remind with you data same then that you the regards there that thoughts coalescing any expect presented in much. gene second to specifics Pompe we the the on part, or So, emerge gene is can provide data clinic preclinical this can any you is forces to any increasing enter area. of this therapy, competitive

Neurology... let showed that data the the with I’ll Jay, for you Child first the Batten we in part additional Yes. take at

in bring data and component was up at that a with and -- Hamburg frankly history to everyone or of & on which that well gene both combined the To individually weeks terms another, language compared patients almost in the plus motor both Child consistent on That Motor effect matched separately, in profoundly patients, showed the each in was to. therapy natural as Neurology couple was which and disease progression more referring one very younger ago language. of patients, granular when combined in Yes. comparison stabilizing motor the motor all but of to Subscores, presented Society Language are you’re the of the and what the the It speed showing the stabilization, most of it. language, both the on slowing

will the will visual be data data vision the have a The related It’s coming within it Hamburg. that within the visual up on and available. the scale to additional vision were And for on I seizure the scores scale said showed more tests future. we acuity disease, the in a time Hamburg of and stabilization caveat. first the one. the But, a -- other is or general Both the Hamburg, be data at is the that new on visual think, we that just following progression the

as So, patients. was that have we’ll all as we the in the but what on seizure of well had well; that analyzed That trajectory when most advanced stabilization share subscale shown. we and showing

about question or going comment other intravitreal administration the Jeff other let on. on I’ll

Yes.

with studies. improvement there CLNX gene preclinical a manuscript showing the actually in therapy eye in our is animal an the So, published

functions. to And of hopeful So, across intrathecal partially we’re more assess see benefit seen, as now, continue more approach have we line. the the all intrathecal adding the may an improvement as we’ll continue see an additional effect or are younger to kids like older in that of climb stabilization. a that treatment could down there at will route least vision data, really we’ve terms if be address optimistic can hope in the defects. different patients the initial And some we Right we to

gene back the we initial results in for that to these And Pompe I’ll gene team was just that meeting the presented pleased back at this were Wilson this then on Jim therapy question in second incredibly something Yes, and we therapy is we the comment Again, yes, April. and in that the April. his Washington data data. presented see

and continued forward had that course summer here. we allowed we additional showed Day preclinical that then the move as the the to at early declare We of data Analyst -- into we proof-of-concept candidate through fall us to the this

are a involved I which there therapy, excellent. Look, gene Pompe think is people of in lot now

a when of after with my of promise to thing. offers first on it It And moving kind years all good therapy. think I people takes think, read to I these is me lot gene people to I back ago very XX now finally Pompe paper forward Pompe. a living years see

to doing therapy and to And at What to we’ve a we’re gene therapy gene provide here. is our Amicus through complete though approach addressing very goal that disease steps differentiated his disease with Wilson includes disease of approach targeting and muscle, motor optimize to is Dr. neuromuscular a many engineering very protein a this and taken through to expression and Pompe a within neurons. importantly These team. Again, to secretion, of a that neurons. targeting the disease key motor reach is CNS tissues include

steps a we promoter could the forward. differentiated we’re coupled move very to be those believe highly program. eager in to It’s right optimal to we with now lead So, vector think something the what

our of you. Bansal comes Mohit next from question Citi. Thank And

open. line Your now is

and wanted and Hi, for of there. through just question walk the programs, Keith guidance for in, Mohit. we spending you. congrats And the R&D with XXXX approach beyond in you or I we This expect thanks one agreement And should this higher then guidance us year-over-year end progress current connection on cash is the is taking our as with baked flat on reflects, the programs runway for what course, quick questions. expanded Penn? quarter. particularly the to what Could for for respect XXXX, of with the

Sure.

but pretty think, happy to those? clear you we I it. want Daphne, were the do on call, we’re tackle to repeat

Sure.

continue R&D -- the key at call, approval then therapy is are pipeline. AT-GAA the in our of gene beginning included spend forward and what’s through focusing which the discussed to priorities John is So, to on move that the

as included operating in of expense we’ve all as guidance guidance is the runway spend So, that given. the that cash well

for is therapy. laid I And for to Thank want we’ve through crown just gene we’re in now we’ve Yes. Daphne. R&D jewel some also very the be AT-GAA. perfectly doing What our clear you, early again. in investments made foundation investments significant

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for to to this back CEO John turn Thank you. conference Chairman closing And now the like at Mr. remarks. Crowley, time, and I would

again, mission for you, listening. successful our focused for a operator. we again Thank great Great. for delivering all Thank And for shareholders. Have and very, a you on quarter. on you, patients remain listening, day. very Thank

conference call. today’s and a Thank great have concludes This you day.