Ascendis Pharma A/S (ASND) 5 Nov 222022 Q3 Earnings call transcript

Good day, and thank you for standing by. Welcome to the Ascendis Pharma Third Quarter Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speaker’s presentation, there will be a question-and-answer session. [Operator Instructions] Please be advised that, today's conference is being recorded. to Lee, conference Senior like speaker to now Tim today, Director would hand over the your I Investor Relations. ahead. go Please

Thank of Investor everyone you at Ascendis XXXX Call. and our Relations Tim operator, I'm Director Quarter Conference Financial for Senior Lee, Pharma. Third Results you, joining thank

Volck, President Oncology; Singel, Vice Diseases; Vice me Chief President Affairs Senior Joining Mikkelsen, Financial the Dr. Head Officer; Clinical Executive Officer; Medical Endocrinology. Head today call Chief on Stina of and of and Development Clinical Scott Birgitte Jan Dr. of and Head is and Smith, Kelly, President Commercial Joe Senior US Development Rare Endocrinology

carry commercialization market be to begin, for respect will market, statements safe to and harbor covered this Before would Securities sustainable our based remind to new the statements biopharma provided conference statements our us timing by information I the today. commercialization approval these of goals and of regarding should of regulatory we contain you statements, Reform candidates, such in SKYTROFA, that SKYTROFA global regarding with statements, call and and planned the the expectations, to our therapeutic not timing in US These results, regarding place statements market, the or expectations, including statements and disclosed not forward-looking of Actual could strategic on our in candidates into expected not on goals plans, and statements US company. forward-looking are create ability our of undue TransCon is projections of Private those TransCon or achieve US progress Act. our to statements intentions, our available regarding pipeline, Examples be launch that, clinical are limited results our that US product to development on able under our out approval the we reliance or forward-looking expansion areas, Litigation may regarding the filings, of a like intended materially may for the continued but pipeline next regarding progress, events EU their from year, pipeline and leading our include, clinical statements continued the the hGH our our differ market and our PTH to you the plans, statements. are

any obligation terminate. or circumstances we Our joint forward-looking do law. to licensing the no as dispositions, statements that potential of required We these acquisitions, investments assume update statements not or by into enter ventures reflect mergers, except as impact agreements, may change,

report to secretion or under kilograms filed For treatment annual TransCon factors that Hormone materially, Form hGH XX.X Growth concerning recent results growth actual section section the our the press hormone. inadequate at of on weighing of today’s X, due failure the Factors with pediatric our name approved is additional of by forward-looking in patients year differ most endogenous release the TransCon least have for the the SEC older and see cause statements Risk growth the and Human one information please XX-F March brand in to on XXXX. SKYTROFA FDA US

note investigational developed the commercial and due Union. failure are candidates In injection Otherwise, United for the referring European of refer marketing adolescents use. subcutaneous treatment hGH approved for our a States TransCon and jurisdictions product to unless once-weekly granted years hGH growth product the with authorization to lonapegsomatropin, European name or we children endogenous we of to that as particular are this the general, such please in hormone. context product inception XX as Pharma has not the addition, to In aged Commission TransCon a X a Ascendis the is the growth under of

on investigational promotional. of results the viewed our of safety call our any effectiveness statements or call, we'll regulatory agency. products, made reviewed not On we'll None conference today's financial quarter approved discuss business the third be by candidates XXXX and provide product As as shop have candidates further the the been and regarding product updates.

prepared will some Executive call open President then up now I'll Following the remarks, turn Mikkelsen, and over to Chief for the call we Officer. questions. Jan

dropouts expects SKYTROFA FDA April €XX.X US new a than longest of bring quarter. X milestone new afternoon, totally priority had review again, Ascendis, sustainable, the everyone. this to for believe revenue evening reporting a important now third we FDA CNP, adult we become as our leading for patients for treatment million for and another the we by years. Tim the NDA duration to Phase has our for to more and Trial Thanks, week, in important long continue All the as with approved, two journey our announced, quarter, quarter hypopara. complete treatment we Vision PTH of NDA TransCon achieve patients XxX, achieved XXXX. hypopara in review, the to our again, biopharma for file introduction. If to no Today, and approach an community. of good profitable major doubling And will continues accepted milestones this to with company. our we are compared XX, we Earlier Good paradigm treatment TransCon this last sales ACcomplisH around

year, our I to made efficacy looking-forward safety of without TLRX/X quarter, Society early in confidence first successful oral look-forward SITC After agonist. from track data at will we SKYTROFA next become the first that that Last the additional the next are following achondroplasia. given statement. believe In the coming positive, to in with middle to supported and we We this Cancer our of top conference. in for fulfill X We aged need we sharing initial combined are been of oncology, will the reinforced. X week's expected data for with quarter, strong belief including Immunotherapy results, the the on around presentation equity TransCon US have billion. of US in cash position XX launch We children our or in-human dose my investors, of with launch flow weeks this supporting in to has cash Phase TransCon recommended line our sharing €X PTH

now the growth are daily taking global SKYTROFA Okay, more same market-leading with share, different designed need results and our into address SKYTROFA let set details children in to the expanding US from of With hormone we on now me as you Hormone market. time at of that a product, value brand building the marketed program. Growth growth overall unmet medical We the the TransCon the hormone.

we compared revenue million €X.X the million, SKYTROFA €XX.X quarter, reported in of this For to quarter. second

to strength the expect of five similar through are what improved and revenue providing revenue providing goals US reimbursed SKYTROFA be growth we up patients strong addition QX a the majority product when that SKYTROFA we on of in we we to for least our differentiated I in launched execution and to commercial -- saw have base In the in at revenue expect a the new achieving of by new will to building patients beyond. the the As new level with we a set for QX, continues XXXX with believe base quarter for treatment patients for becomes treatment. base, next at reimbursed we grow SKYTROFA years, patients. of the quarter. that on each number From three grow

to growth quarters, the our on Over support hormone a coming our global into we to provide that leading efforts updates market. will the growing programs continue TransCon all in build brand

this need product with week These year a from are an importantly a to have candidate taking life to FDA PTH, disease. for We successful regulatory as TransCon a April for faced us restore priority filing. is bring their next second and PTH our we candidate to regulatory in accepted address NDA that decision announced US. all Let adults we treatment for by TransCon It's XX our turn This FDA that product patients the to dedicated the possible. effectively to them. of hypopara. complication as design way of patients normal has hope treatment we Earlier from help to for in row are for review urgent product soon

benefit the PTH consideration renal HP hypoglycemia, hypocalcemia management who patients PTH for are of vitamin might of [indiscernible]. symptomatic was suggested insufficiently active observed with following; The of for poor recent of any doses controlled due update the insufficient, D hyperosmia, importance on also authors replacement patients absorption for therapy also hypopara. of or more therapy therapy. PTH the the view of replacement large calcium that intolerant guidelines They or one compliance by confirmed Our to of

address data we this for showing regardless all the of disease believe background TransCon PTH unmet that to could clinical treat With need. and long-term medical patients responses, durable trial potential major growing dividend HP

next on With preparation we timeline PTH, and are reach Expanding events both happen US report track and we expected review set in for underway focusing our quarter. MAA for to our expected TransCon Japan track Trial, results an the QX our in year. approval global we regulatory US are submit on to remain topline and on X Europe Phase to PaTHway this from launch

to we believe dedicated potential treatment doses years from option could TransCon attractive once-weekly of further patients. trial PTH are a PTH, With data on be up follow-up that PTA patients to our for three optimizing product clinical We treatment of stable HP a option.

at multiple for to different reaches that optimal normalization We that when a believe obtain demonstrates stable become once they calcium each same data the with action. titration is that times. daily PTH switching mode Our to doses once-weekly of hemostasis therapy of goes patient and TransCon PTH of patient candidates

candidate from switching based and in PTH. drug and to weekly as TransCon TransCon Our PTH parent of now product safe once-weekly the we preclinical is predictable technology believe enabling TransCon TransCon PTH same PTH patients daily daily development on

TransCon now CNP. to Turning

looking from are sharing forward the topline in Trial. to results weeks the coming We ACCOMPLISH

year week up portion transitioned sequential kilo XX years Our micrograms placebo four was five of to one on younger double-blind an TransCon The kilo, old. micrograms four dose one to micrograms clinical cohort dropouts. in randomized patients trial. randomized were label enrolled with achondroplasia. to placebo-controlled cohort per Trial followed the six than per patients per patients each All patients aged with of and CNP for OLE children kilo, of patients trial of A kilo, designed base. to the total completed per year with open X:X enrolled of enroll from that cohort no of the XXX to per ACCOMPLISH portion XX XX in XX with treatment, XX active After blinded XX Phase extension X double-blinded cohorts two double-blinded dose the XX in micrograms

per the open-label in the continue that and today patients kilo of all per week had transitioned extension all As to XXX been dose at level micrograms dose.

last annualized you duration December basis see without The primary is of for of the blinded continue two after year The the We to safety longest reduction placebo-treated around profile patient the height we treatment with velocity to dose to for years mean trial ACCOMPLISH on TransCon any is and analysis to well-tolerated a XXX% annualized CNP that compare same primary height retention. reported now cohort patients. endpoint velocity one treatment.

mean to XX years. the plan to also data to We two aged patient and patients years the annualized two for height years five groups years velocity report X

of double-blinded on placebo micrograms In get to provide to kilo treatment up for TransCon expectations treatment randomized data top-line preliminary from about of filing. information are the lot dose to extension data I weekly we per this report dose addition CNP. open-label the my lower when a also of XXX study. what data plan These portion from impact questions or the will

endpoint. and been treatment has velocity outcome, priority, be annualized to validated call as velocity well-tolerated. needs First primary annualized it of the perspective From or the regulatory growth safe height

growth the major height multiple trials in other the Just velocity outcome. In determining disorder age this achondroplasia of patient dependent factors. factor is as with particular is the in on endpoint demographic a

sufficient achondroplasia age age the of in X.X for XX the per than X in for velocity group for group to trial trial slightly treatment velocity to analyzed annualized XX aged fewer the the to of of at was X age. global height I X XXX lower achondroplasia submitted per X to year X. the Perhaps to patients this approved years the as children in from height XX we To a X. have XX endpoints, will CNP the analyze measure has endpoint. further trials. trial patients the after only age height year our be to more retention TransCon X treatment we to with kilo greater annualized with enroll evaluate to group weekly a age as This we patients why the XX group expected compared which for the year for about to Phase five treatment per patient to randomized So benchmark to group velocity growing centimeter XXX% the and ACcomplisH the X estimate of will primary faster to XX enroll to help placebo-controlled may to ACcomplisH year, Xb per protocol growth velocity FDA important around initiate X.X annualized compared micrograms in trial double-blinded explain US effect secondary believe centimeter one additional in in patients trial dose, see a is

In We next obtain track we early rare endocrinology with XxX remain approval expect our for of to on three Vision enrollment by goal disease summary, XXXX. complete this trial. year to products

time immune for TLRX/X candidate. dose our monotherapy over We and data next activating IL-X tumor for the With tumor toxicity. data hydrogel of for SITC provide checkpoint human weeks two TransCon TLRX/X in TransCon in first the or oral profile and intratumoral programs, oncology. immunotherapy kick-start are levering first TransCon using Agonist. Boston for signs observed the our declared dose the Agonist X with our inhibitor, combination a an thereby system inside clinical TransCon week. Phase held that and with selected safety technology Agonist hydrogel top-line of TransCon systemic we immune completed technology is Turning sustained being designed technology in Annual the without TLRX/X Cancer the are within we to in recommended for for at our system now the activity for Meeting as both microenvironment pleased of abstract release escalation systemic Society TransCon the a dose-escalation and beta/gamma early to to the presentation favorable the was TLRX/X recently With Immunotherapy Agonist

beta/gamma immune be immunotherapy. IL-X it increase continue we product Our second body's combination and tolerated quarter. new stimulation this promising is to of continues for cancer responses dose the We beta/gamma system. broadly or systemic escalation. share in a we backbone top-line later monotherapy TransCon we seeing and are as And to monotherapy IL-X candidate potential become well to therapy designed the believe has oncology to plan results pharmacodynamic TransCon

position is there hear achieve also have €XXX of in are a confident I call review up over track great million drive many to remain but As milestones progress. you can the will for so the more for questions. I many, so Ascendis Scott we many now achieved, on near-term. in And to With our XXX. details turn Vision our a additional to ability continued cash and open financials before we

Thank you, Jan.

confident So as in the launch to major behalf very SKYTROFA next US on are unmet ever growth. across Jan, our become with more than great US year. with next portfolio long-term ability pipeline cash profitable approval which sustainable Combined of QX flow we progress and opportunities from the addressing position and launched our cash expected Germany in making we you is PTH entire deliver year, our our expected product and in TransCon to are and of to the medical mid positive strong of heard launch advancing needs patients in

a strong million. the company-compiled of in on Now XXXX benefit on U.S. doubling the about May, €X.X revenue we consensus Today fourth quarter revenue The during and quarter then our at results. the algorithm €XX.X the achieve reported minor for beyond. third we would QX. of us to in our from in QX Jan's demand. in US based revenue more very Growth line €X.X primarily provide in on SKYTROFA SKYTROFA million This believe expected track growth remain SKYTROFA for doubled continued base SKYTROFA dollar to simple in stronger least with revenue revenue with the estimate and US comments made million from analyst increase sequentially €XX US May reflects to strong sell-side of a million. reimbursed number Based US patients revenue to Jan reimbursed in approximately provided quarter-to-quarter quarter we compared on €XX.X than of million third

development operating third third were XXXX. costs Now expenses. €XX.X during of million for €XX.X compared the quarter to the turning quarter and million Research to

of US XXXX €XX Overall, expenses late-stage future Selling, €XX.X the in reflect during holdings we quarter €XX.X euro. net As of launches had cash launch the our reduced exchange structure of general XXXX. to share expenses as this gain quarter a for and of translation of quarter third by were approval. previous million the €X.XX for of SKYTROFA related for million Overall, basic development net foreign to dollar These reminder, cost of of and to the normalization included of €XX.X more which compared or overall loss of US Europe. €XX.X cash, for administrative €XXX commercial diluted ongoing and included Net XXXX. the million. compared in write-downs million a cost marketable from quarter million, pre-launch or of and preparation reversal €XX.X to product per onetime diluted share following costs a R&D third finance of million quarter R&D million the inventories progress and basic programs through and of a reflects per rate equivalents our loss expenses third early third a during third higher quarter primarily €X.XX third the securities the

the securities equivalents ended quarter million. third marketable totaling we cash with €XXX cash, and Importantly,

the on for year. timing of key now me the milestones Let an also provide remainder update

to hGH, trial in complete QX. hormone growth Phase adult For global foresiGHt enrollment deficiency on X in track we TransCon are the in

Japan, PaTHway are in we PTH, line top submission QX. in track expected on MAA for for and QX a are Europe planned TransCon For in results

results the weeks. ACcomplisH to CNP, sharing the from trial look the coming Phase forward TransCon X top line we For in

and week. top monotherapy we the dose-escalation the beta/gamma X/X to from For are results conference expected Believe therapy present combo next for in plan Phase top IL clinical therapy QX. line transcendIT-XXX Agonist, And TransCon TransCon trial at IL-X trial data X/X line from SITC Phase the TLRX/X

see we commercial Ascendis key to have progress positive. we year with well busy believe in and balance are growth fund diseases and our oncology. sheet, XxX both the strong fulfill strong now going With catalysts initiatives cash a rare you are that endocrinology it's the we ready for Vision As time an the disciplined cost become into flow and incredibly of to end can positioned the pipeline operator, across in capital to focus, take questions. With

[Operator Thank question Fye comes Jess with by. you you. Instructions] first standing from Our for JPMorgan. Thank

line now is Your open.

Hey, to morning. trial for results tonight. Nice Phase thinking guys. are potential be about the Good How you registrational? CNP Xb the

you decided tolerability, the We and from ACcomplisH safety, everything division included daily the only a we a reported to wait see come that is was been way way once-weekly better I the will have efficacy, I that dosing at related to That difference I looked on lot that some the Then the X answer with be be for for from function. the that coming great to I to really ACcomplisH data. trial Xb When years to the to of when are can we providing is of like you. the Phase part to not basic treatment to that data we it much part on focused unblinded. different the hitting perspective which believe has end in that is in because see really, but important question we hormone Trial And contemplation. growth it that be I wait for opportunity the be weeks. think to when on out I be need what profile lay me All we Jess. belief geographic is this And that would my different well-positioned that prove trial. in really most that see a from to best-in-class we CNP target to -- we're on will and as for really data, the incorporated to some question have think want meaningful product is saying someone what designed we come depends really know elements regions, this can we patients that not our a a a TransCon suited if we between we dosing profile a we Trial. important can XX question file most the the benefit product to there And with that lot

that could Sorry, I was asking about be the Xb registrational?

Xb. the an we ACcomplisH a hope are is explanation Xb believe. to the believe with time about this can is we when hear retention I why something developed hear we talk more in seeing growth patient we and that must we Trial group retention. we just trial Xb the We quantify being the you basically than hope we velocity. see analyze that in basic with what but come where see the in we That achondroplasia manner such patients. our can in I because in don't provide that is first was this stories to XXX% this XXX% caregivers, basically in system a quantify need what We we the

you. Thank

Thank you.

please Josh next Josh, comes question ISI. Evercore Our go ahead. Schimmer with from

X question. CNP, the Phase I them how much we Thank for TransCon more of instead you. data? then Jessica. to are fulsome one are results follow-up the move reported be taking to to Why Thanks close close and X given design why Phase guess, X X for specifically we on why Phase wait Phase quickly a for given the a the and protocol. I’ve from not how

because as dedicated is out to as fast bring patient possible. this we are product the This to Josh. Hey,

exploratory can that in be what we sitting entire and we integrated element basically ACcomplisH the element are the the as basic of endpoint exploratory through two endpoint, soon labeling choose what up have on basic list to one all patients. discussion. benefit key of of the the are we secondary this we we basic incorporating And to as the basic and therefore So really understand providing gone as are is Trial, we can doing, are menu in endpoint relate or we all

we this So one product to work this patients in year so is we where when get we to why basic can dedicated out are development cutting and caregivers get we And market. many in coming about us down this way the to time.

enrolled the data all at look the discuss in we the and then next we the can in basic have of year, beginning it will thing. patient achieve and Before we basic same basic basically and evaluate

Got for new quick it. that I gating you steps you've the indicated category year-end. very announcement? If for form could another therapeutic ask platform one, the by TransCon that expect what in disclosed are When what and can that we

basic the gating are -- on The step is that we candidates. pre-clinical

will therapeutic -- want a time. investors technology potentially JPMorgan lot but at well-function mode is thrilled of area, we provide that we guarantee, I our to In action XXXX, our end are disease our if lot building we XXXX. third TransCon can our systems. will disclosed And to pipeline pipeline, established established also and year area. disclose this We while where to pipeline, that said go what benefit We out it with therapeutic of fit oncology disclosed the some that of up be and TransCon we And do need when are oncology disclosed in building up PTH and pipeline with drug we really as and locked now parent did I'm of really medical we TransCon there we target endocrinology we're need, a of at a we our really rare CNP. unmet disclosing

much. so Thanks it. Got

you. Thank

is from question comes Lebowitz Citibank. your next David open. with now line David, Our

for Hello. Thank my you taking question.

we coming what it's we different might and on -- When the what Additional look how meaningfulness clinical achondroplasia. trial forward to what the the way geographies out competing can populations it is given question with in the cross data products? seeing compare the that and we benchmark, are is comparisons actually

to a product said So this Because I get trying question lot on building the CNP as approved prepared for I remark lot. And on only a our TransCon here benchmark [indiscernible]. in to we're a in the when are the is what we question. really -- the we're US, thanks

We are building science. on

there publication the directly go are open as and element For of different me. have annualized to persistent dependency a good with And, got I of velocity. said Publication And outcome population remarks, always not calculation this on between also group will ACcomplisH it. publication that from velocity there's the on one it. how on there level, make because pretty much it's interesting And the really it annualized on good key problem how this clearly on the X adjust for it's perception. dated I'm that you will with vosoritide, to Phase got I XX is for height to I between an vosoritide. more out trial, is good prepared in the to is we my And own because Then eight any mainly to up. effect children growth growth on study. that treatment transferred have And because effect and two there a difference for really literature five can results up And up you we benchmark dependency two two achondroplasia, the built in population. -- or we on velocity can XX, is is annualized share split group. a both the growth to velocity of direct analyzed providing much, front and example, you how from that's And factors. there depending five, to patient this in because compare looking eight in really on there in why demographic basically just go comparison built of you up for -- five clearly all is science, is comparison. precise is because data on vosoritide five, growth in built all the with in annualized absolute do that not growth if to no and promoting for that years from eight a to the patient there if there the is documents publication is FDA the look -- really and is basically five five. the -- benchmark, Safety you because as filing back and saying, show publication velocity, that Where can have got coming placebo which two have same transferred XX, I you peer-reviewed but vosoritide therefore, you the this not this patient -- extension because compare

question. you Thank very much it. taking for Appreciate my

you. Thank

with Li next Cantor. from Watsek question is Li, now your comes open. Our line

great. for PTH. the questions, on Maybe Thanks Okay, just one on congrats and quarter. TransCon

in you April date a Now have year. next PDUFA

Just you can launch the talk commercial about now preparation strategy about we Europe? think with and synergy in also in right should US? potential your wondering, how if And SKYTROFA your maybe

Yeah. Thanks lot. a

we do we after. XXXX for that years. You after and right independent is three Because a And expect [ph] built-up we we're also the the where we a other why pipeline global was years that TransCon next SKYTROFA, commercialization. one word, two PTH, product said the that with coming What built opportunities hope is for we hope XXXX have what two to doing product we rare launch entry endocrinology synergy. in one disease of within

he are to in commercialization me, the is that is us. do commercialization. of in front global We the for for here a that dedicated sitting responsible Joe US group

geographic strategies different As different you seen, have in we regions. have

opened just in now in ourself. We up We a long German the headquarter are Munich. capabilities Europe building

we from to there. are ready So launch

ownership Greater we through our say our JV or can in China VISEN in doing Pharmaceuticals. are We

of a an endocrinology. is creating two that where need also into value be When it things yes, have really where approach So the to be we we a US, there our, we for lot Ascendis this fulfilled why come synergy. have pipeline is to from perspective. And pipeline we checking are is

First, it, there the product from take chance revenue be not want we needs dreams. other expectation. between synergy come and because P&L least that, something in billion system where on not just needs to sales run not at the €X.X have and it’ If any thing to on to we is it's we

therefore our building So, on are we pipeline synergy.

really infrastructure about is so don't think element. everything believe, you the people synergy. you a this facilities, synergy infrastructure. then like I as people, key And that, is word much and really when So think infrastructure IT where about have solution, actually logical

is go about you from group have can about think how conference, perspective Joe endo for little out you conference, and three-product. sales that in that you just where basically comment can do to two-thirds. some people This way Just one we to meaning And force. mind is it a one save think their bit

between from which just much to running part other. perhaps, obvious how How the Joe think we are have you But the affairs, say, really we about and can So different can you synergy one of have synergy that on switch do medical organization. prepared.

the We are mode. in launch full

biggest we We to our to TransCon XX have be going launch and product ready full be mode It's for to been now months in want for next the six on ever QX year. PTH.

Okay, great. a ahead. just -- Maybe go

Li. ahead, Go

Phase understanding Maybe X just Xb. if wonder a blinded. can I that about. of talk trial secondary is your quick you some share follow-up that on Phase My still you is the endpoints Transcon CNP

from guess, Phase I you help X Xb the to gain insight trial protocol? finalize you So Phase what can the

already FDA, Phase we our protocol into full-blown so filed The by are Phase Xb. Xb is

Rare huge have of be huge We in expect benefit protocol basically element to aligned national have menu a patients and in call some to Birgitte, Clinical Development Perhaps words she we just we menu data I of What list, now enroll say think Birgitte could Endocrinology. the is really and a the waiting on will up we next the the about all -- our list all ACHieve the Regulatory the our pretty trial. simple is them you Affairs the in at all Disease believe Medical we is looked we endpoint where can have different the built evaluate be. we we in But that to up beginning in endpoint hope secondary when year of history. protocol The trial the switched all over our for and have exploratory into ACcomplisH. heading

and so for questions. Joe you much, Thank Yes. thanks Yes. the

protocol. Xb objective And the access about our to this hopefully excited very again, to is timely are provide we potential think treatment opportunity. I So

measures we anti-inflammatory program this something having for That were for Difficulty] other our then exploratory something aim PTAs And measures. cetera. outcome haven’t we have each body outcome very So similar measures have measures and to relevant also proud test would was program. et including patient included we index, relevant is we current developed mass [Technical of

captured medical appropriately will evidence relevant evolved new field of relevant into this as contemplate. addressed and exploratory the endpoints relatively of insights are that as So menu be bring in intervention in

Birgitte. Thanks,

Thank you.

next comes your question is line Our open. Goldman from Paul Choi Paul, with Sachs.

progress. Good Hi. congratulations let add all my afternoon. me And Thank you. the on

extension you design just comment Xb X the your -- clarification. it whether has the of APPROACH label with solely study was clarify trial have the anything decision point a blinded for Phase whether open that the Can or of available Phase on seen data? regard from informed Just to portion on based you

looked do extension been data We has the believe before as when and trial double-blinded can really it do trials. commented to blinded weeks. have very much coming we We're we're open-label is have the just we this ACcomplisH without a looking disclose forward data. anything nature been the confident to And we it. on also in we that feeling to responsible the the the to on jeopardizing way data, on the for on you -- look We looking

conversations market just for you premium PTH? Joe. Can the the hormone for commercial, at been And SKYTROFA as your growth obviously maybe Thank what a regard us of to TransCon launched in category. update you potential the like then that. to provide feedback on on maybe follow-up payer an You with or has average a Okay. pricing

would qualitative be provide any feedback maybe questions. just Thank or great. you quantitative for So taking can you our

the Ascendis And so And we with a in basically value believe evaluating on I price when in structure, are I think premium do that a can is PTH. the the that product providing on for TransCon the the come exactly really position pricing. it factual the we we follow we same thing cut we respecting And optimal First. but just Joe, out really responsible only came or...? Patient to also we it society. part forward well patient I have we are did on for not start can think what will we the value we caregivers comment more of the the will you

getting reimbursed, PBM work patients the not. they whether this patient in says not If they from coverage really or now they're payer. so reimbursed Yes. the what at covered we are since important. to are typically Because Paul, is point. -- really get Sure. the willing, getting are far But up HCPs to a that we've seen which XX% right around and have that's the are we so MMIT launch are, thumbs most put that

structure. think, our coming think, sales. SKYTROFA the was pricing I Growth about more it a I Hormone bit Paul, about little PTH to TransCon wanted But hear

Okay.

Yes.

Yes.

If you any have thoughts?

between there will mean, synergies. payers leverage in the are marketplace out Paul. I we're about front in that that's approved. comment and And and once PTH TransCon the Yes. certainly of that, PBMs When all SKYTROFA I’ll

you. Thank Great.

you. Thank

Vikram, Our next question comes Vikram Morgan from with line Purohit is Stanley. now open. your

Thanks Hi. taking question. afternoon. Good for our

one So on SKYTROFA. first

do So SKYTROFA provide we you But, SKYTROFA for a yearly threshold last sales in XXXX for you looking all plan you know provided forward, to that, quarter. I could XXXX? revenues obviously, achieve formal for guidance that guess,

No.

Understood. Okay.

to by enough program? then, expect And beta/gamma think dataset learn for decisions make then, follow-up of there? a that to question that be IL-X next So would you separate year, on the the what data could we of do end expected topic. steps

are we better treatment providing can non-alpha what to of better technology, high a see combination teams together an happy because but so profile than and I'm that achieve escalating for not her expected we look TransCon a efficacy with And because with Stina the progressing are have speed is and First on can the to inhibitors. we're checkpoint optimal data the that's I'm checkpoint make complete and I the an dream our when -- recommended of IL-X because do, we our executing potent really have safe. this. And really that to you Phase is speed. achieve to yet, design and inhibitor, is a all, combine program it, really we X a dose compound with time on beta/gamma, dose still to with is safety patients the supporting not extremely our what pleased really available basic, that optimal protein what seeing on something

comments? your Stina, So,

Jan. you, Thank

the in effects, we monotherapy impressively, is very first for to mentioned also data. a therapy We hope some checkpoint Jan of have of what seen is you on safety other with can we push we Most inhibitor. think, that, are cytokine the pharmacodynamic escalate the make as as which the for initial is think and the dose year, to are middle I this have combination have to any variants the patients. how thing in By end results tolerable technology we dose-escalation clinical key has with I for looking And out beyond far designed IL-X TransCon we the there. it with do, is

look on in to ever ALC, to typically, to you can a else how do they And that. plus monotherapy are then treatment, have target the mention a meant concrete if So, about Why to little patient three-fold that? see and tolerate look product, that ALC. bit effect. do we you no we we a to start -- need to two if really we flavor believe just Because on, have you ALC five have we one be give in at you more

that want is is them science really that developing fight is had to to prime really the and kill because system as that's the it. why biology a the an all hematological possible as go anyone know to under not two then fast monotherapy companies for we is in else and higher because do, cancer. expectation So up no ALC the see than or there This we ever non-alpha have to by are supporting ALC three, effect,

Thank you.

comes Oppenheimer. Gershell Our Leland next with from question

Hey, guys. Thanks.

Good morning, Leland.

Thanks for taking questions and see my afternoon. good to Hey, progress. the good

the side. TransCon me. regulatory just again a on down -- questions CNP, One two from on question Just further drilling

there any other that, pathway would given out that's approval their The has said the own be to accelerated close approval. new with achondroplasia its entrants company product now

the to discussions ACcomplisH or you of could ask, take with we as able to want run this be the we comment as respect you. for you to comfortable Phase would least the the to absence just of at you you're that data QX may XXXX? guidance that you've QX should current in Thank clarify So also get off expectation with year, year mean any of to for into file FDA do think or to SKYTROFA, with if that? Xb And other rate indicated you agencies go to need approval, next that planned through quarterly

sales me let what quarter's on baseline. on the question. with thing is great Yes, that, is Because SKYTROFA we're last building start the last

that this here, already if for saying patients on have three have treatment are keeping that, QX. for four quarter €XX.X is we to Meaning So we example, years. is we million you, for basically million, €XX.X are

patients, adding and team and up. new patients, just go his Joe getting new is out reimbursed So

So already that the I we take an in the if expectation middle then is can do you million you're, in he then QX. for four and he this in similar quarter can calculation, example, to with example, calculate with it million speed €XX an even without QX, as It's And MBA. a if last did say saying in if to for will XX times, XXXX. end have baseline €XX you in same he see

primary don't own lack regulatory of baseline. patient new pathway I nice reimbursed look it's my understand question about it getting this on the and way last got question think a out a because add knowledge, like is else. understanding it this for really I to is from somewhere an prediction being US. see that the big lack is really as is – what this generated of I me, mean anyone at at big, calculation nice close expected XXXX. it's of mathematic make now times for a gives endpoint, way this utilizing I've me endpoint in the that of revenue And And is analyzed and not for happening does some What -- question. big, add we validated And it. is a a The algorithm of easy everything to the way big, So I lot. XX about is

this of this lost regulatory there someway question supporting is and of really in think any So kind don't element statement. scientific I'm kind I that totally

Okay. Thank you very much.

Thank you.

you now Schwartz Our next your Joseph Thank with line from is SVB open. Securities. question by. for Joseph, standing comes

Hi. Great.

me. was you you I much how it's on hopefully was for if TransCon again. discuss very current wondering are potential when this which the that launched. factors driver a launched, we've alluded TransCon updated I versus and expand PTH might your improve for positioning, [indiscernible] when revenue bit different estimates that's other since think guidelines I being can heard to a meaningful was for wondering was What reimbursement the it PTH? of Congrats

much -- much have it's are in try extremely already to justifying guidelines, market I even make giving And patient taking of to what's him It's taken US studies very, were the pleased. the very seen right a life. guidance the I is easier read I've with get when what, the hard updated PTH knowledge I basic his a for Joe assist the really, avoid is easier I'll really giving because are into really my you the But guidelines. X. something system to that's on the really case are not majority way access life market What to pretty hard. guidelines, US the that

but also discuss It's feel for is year, It only and dramatical not unmet prediction it the disease with always short-term medical all complications revenue actually therapy symptoms, relief I because symptoms. basically on treatment the when disease Not problem patients it a worse the complications, complications providing base start the sales of is amount the of worst. and vitamin unmet single I case worse, supplement need. medical really for of the making and with and The the increasing one basically So need really we the basic this thing calcium D. hypopara. short-term long-term conventional activate choose are the makes

I life? be I before majority broad in said of these perspective. the the believe treatment. PTH price all should should patients at think that look to majority I treatment is of be structure XXX,XXX be commercial as available be XX,XXX but responsible. And when that, So we reimbursed a for the guarantee on Will the of it. cannot them I number patients US we that in will

-- this caregiver, for opportunities. huge why premium are in but a product patient the not is society. best-in-class is still also will we that the for the We a only benefit pricing And providing believe for saying we

a about much. Okay. question Thank the pen I then but hasn't really which PTH, is And I for TransCon discussed device important you. think have been

does maybe TransCon? you patients So dose the for talk how how I to right SKYTROFA it pen the help used titrate was the what's of compares wondering if could it to about and and

to much because Yes, for really for pen new TransCon complete a for It's about talk compared TransCon pin SKYTROFA, we PTH device this. auto-injector so using you're PTH, boring SKYTROFA actually because the we're right. because it's that's in and million pre-filled are formulation develop We classical and device. the a for a utilized million day pen basic insulin optimal million times liquid device temperature And getting it's in pen US for of the patient. room don't every

for three pre-filled it's take So you pen pen a low-dose, prime it's each is built to need weeks XX And and medium then don't high-dose you new it then strengths. days have different on medium two and dose a you basic the low-dose, and high-dose the and use dose, device pen.

we basically that nine are presentation different different covering So way. nine or

times proven era the of optimized a to everyday. it's insulin patient that really So is why be a talk the technology, each -- in it treatment. millions used each can But we technology Joe, subject, about millions don't because

Thank good. questions. Sounds my you taking for

you Thank

ahead. for from by. at you Wedbush Argyrides Securities. so comes question Please next standing Our Andreas Thank go much

my for filing Can for update status SKYTROFA questions. progress in the the Congrats on Syndrome? children of provide quarter the in on beat. an you taking and Thanks Turner

just we that. you need And trial, do the on date first. based trial, clinical will provide to clinical We on the a

when basically, So have we done provide will clinical that trial. we the

will and is provide We update expect to what next the an step. file we when

for then pediatric And well? HP, Okay. as

an us for Pediatric element because the we interesting initiate strike. HP is

time have trial. start We the cannot exactly a little a into pediatric help for pediatric believe designed I'm because population, we we here and I clinical new consideration can the year pen, the you or system because Birgitte, really have me insulin what remember, taking bit next and I perhaps

exact shared for these we be now, working have our path but studies. to don't details are forward I the

for no that. additional details So,

will we start trial, It's clinical very very, a next year Sometime feedback. all regulatory the the small we trial. This what got single-arm. clinical is

at it's about trial XX XX months all. six between think patients a to another I last not

in for and Thanks congrats Great. the update quarter. Okay. again the

Thanks so much.

Thank you.

final much Our standing Werber with ahead. Please Cowen. you from for Yaron go so Thank question comes by.

over think X.X five-year-olds? how placebo? a about on data? to Is sort expect the taking year, obviously Yes. at integrated Hi, relating So to patients maybe questions. for the centimeters when in you're -- And my an about that's when obviously, Vaxogo comp good plus. the five. they baseline. about . thank the two the all-in year older did Thanks on switched you. then per Thank X.X placebo five-year-old two-year we referring The to centimeters Vaxogo it And what Jan, to question X.X just of to So see did per the than in X.X should that we What extension you.

that that solid because other of dataset it of number built on you of on to is two also patients data, the seen an population data huge their amount datasets, number compared and anything is of limited is five with hate one dataset, the not, Yes. choose the a a built I built against to limited really that's without is benchmarked we huge where good benchmarking ability I actually basically, All have and the This already patient two And filled amount patients, are then at where benchmark ability. to take ability. all. on that

up So five five really the and there's between and separated in solid up. patient this why, from I a population is two and to five data-set before

you're to lack I referring believe the to do really because this lot why of example, meaningful five For publications two of a the comparison, to. real from the of And it's is that don't benchmarking.

This participating. you now for call. Question-and-answer session much, Thank finished. you so does today's conference is Thank conclude

now You may disconnect.