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Ascendis Pharma A/S (ASND) Q4 2022 Earnings Call Transcript

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Ascendis Pharma A/S (ASND) Q4 2022 Results Conference Call February 16, 2023 4:30 PM ET

Company Participants

Tim Lee - Senior Director of Investor Relations

Jan Mikkelsen - President and Chief Executive Officer

Scott Smith - Executive Vice President, Chief Financial Officer

Stina Singel - Executive Vice President, Head of Clinical Development Oncology

Joe Kelly - Senior Vice President, Head of U.S. Commercial Endocrinology

Conference Call Participants

Jessica Fye - JPMorgan

Tazeen Ahmad - Bank of America

David Lebowitz - Citi

Li Watsek - Cantor

Paul Choi - Goldman

Derek Archila - Wells Fargo

Josh Schimmer - Evercore

Leland Gershell - Oppenheimer

Andreas Argyrides - Wedbush

Yaron Werber - Cowen

Operator

Good day, and convey you for opinionated by. Welcome to nan Ascendis Pharma Full Year 2022 Financial Results Conference Call. At this time, each participants are successful a listen-only mode. After nan speakers' presentation, location will beryllium a question-and-answer convention [Operator Instructions]. Please beryllium advised that, today's convention is being recorded.

I would now for illustration to move nan telephone complete to now your speaker for today, Tim Lee, Senior Director of Investor Relations. Please spell ahead, nan level is yours.

Tim Lee

Thank you, operator, and convey you everyone for joining our afloat twelvemonth 2022 financial results convention call. I'm Tim Lee, Senior Director of Investor Relations astatine Ascendis Pharma. Joining maine connected nan telephone coming is Jan Mikkelsen, President and Chief Executive Officer; Scott Smith, Executive Vice President and Chief Financial Officer; Dr. Stina Singel, Executive Vice President, Head of Clinical Development Oncology; and Joe Kelly, Senior Vice President, Head of U.S. Commercial Endocrinology. Before we begin, I would for illustration to punctual you that this convention telephone will incorporate forward-looking statements that are intended to beryllium covered nether nan safe harbor provided by nan Private Securities Litigation Reform Act. Examples of specified statements whitethorn include, but are not constricted to, our US commercialization and continued improvement of SKYTROFA for nan US market, nan commercialization of TransCon hGH for nan EU market, statements regarding nan expected timing of support and motorboat of TransCon PTH successful nan US marketplace adjacent year, statements regarding expected timing of support of TransCon PTH successful Europe, statements regarding nan imaginable nan marketplace size of TransCon PTH, our advancement and our pipeline candidates and our expectations pinch respect to their continued progress, statements regarding our strategical plans, our goals regarding our objective pipeline, including nan timing of objective results, statements regarding our pipeline merchandise candidates, statements regarding planned regulatory filings, our description into caller therapeutic areas and statements regarding nan expertise to create a sustainable starring world biopharma company.

These statements are based connected accusation that is disposable to america today. Actual results and events could disagree materially from those successful nan forward-looking statements. And we whitethorn not beryllium capable to execute our goals, transportation retired our plans, our intentions, our expectations aliases projections disclosed successful our forward-looking statements, and you should not spot undue reliance connected these statements. Our forward-looking statements do not bespeak nan imaginable effect of immoderate licensing agreements, acquisitions, mergers, dispositions, associated ventures aliases investments that we whitethorn participate into aliases terminate. We presume nary responsibility to update these statements arsenic circumstances alteration isolated from arsenic required by law. For further accusation concerning nan factors that could origin existent results to disagree materially, please spot our forward-looking statements conception successful today's property merchandise and nan consequence facet sections of our astir yearly study connected shape 20-F, which is being revenge today, February 16, 2023.

TransCon Human Growth Hormone aliases TransCon hGH is approved by nan FDA successful nan US nether nan marque sanction SKYTROFA for nan curen of pediatric patients 1 twelvemonth older weighing astatine slightest 11.5 kilograms and person maturation nonaccomplishment owed to inadequate secretion of endogenous maturation hormone. In addition, nan European Commission has granted a trading authorization for SKYTROFA to Ascendis Pharma developed nether nan sanction TransCon hGH is simply a onceweekly subcutaneous injection for nan curen of children and adolescents aged 3 to 18 years pinch maturation nonaccomplishment owed to insufficient secretion of endogenous maturation hormone. In general, we mention to this merchandise arsenic TransCon maturation hormone, and unless we are referring to nan merchandise in

the discourse of a peculiar jurisdictions specified arsenic nan United States aliases nan European Union. Otherwise, please statement that our merchandise candidates are investigational and are not approved commercialized use. As investigational products, nan information and effectiveness of nan merchandise candidates person not been reviewed aliases approved by immoderate regulatory agency. None of nan statements made connected nan convention telephone regarding our merchandise candidates shall beryllium viewed arsenic promotional. On nan telephone today, we'll talk our afloat twelvemonth 2022 financial results and we'll supply further business updates. Following immoderate prepared remarks, we will past unfastened up nan telephone for questions. I'll now move nan telephone complete to Jan Mikkelsen, President and Chief Executive Officer. Jan?

Jan Mikkelsen

Thank you truthful much. Ascendis is built connected nan unsocial TransCon exertion platform, which enables improvement of highly differentiated merchandise candidates crossed aggregate therapeutic areas. Combining nan TransCon exertion pinch our [indiscernible] merchandise invention has enabled america to create and create merchandise candidates pinch a higher likelihood of occurrence than seen pinch accepted supplier development. One of our cardinal merchandise action criteria is to fulfill best-in-class imaginable connected each of nan 4 cardinal pillars of supplier development. Safety, efficacy, tolerability and convenience. In addition, each merchandise campaigner must person nan imaginable to execute $1 cardinal aliases greater gross successful a azygous therapeutic indication. With this attack and [terming] by our values of patients, subject and passion, we person demonstrated our expertise to continuously build retired a robust pipeline, while taking merchandise candidates from conception done support and launch. With expected regulatory approvals of a caller merchandise aliases further denotation each 1 to 2 years, we are fulfilling our Vision 3x3 extremity of building a sustainable, profitable, starring biopharma institution and creating agelong word worth for each stakeholders. This past year, we person precocious our pipelines arsenic planned entering 2023 pinch an April 30th PDUFA day and expected US motorboat of TransCon PTH for big diligent pinch hyperparathyroidism by nan extremity of Q2, on pinch an expected European Commission determination during Q4 arsenic well. TransCon PTH is our 2nd endocrinology uncommon illness merchandise opportunity, representing a imaginable world opportunity greater than $5 billion.

Turning to TransCon CNP. Last November, we reported 12 months information from our first -- from nan first ever randomized, double blind, placebo controlled Phase 2 proceedings successful children diagnosed pinch achondroplasia. These results springiness maine assurance that this 3rd endocrinology uncommon illness merchandise campaigner whitethorn person its first support by 2025 arsenic target successful our Vision 3x3. Another covenant of Vision 3x3 its explanation and geographic description . We proceed to build nan worth of our existing programs done further objective studies for explanation description and world commercialized reads. Starting pinch our recently expanded European organization, which is preparing for motorboat of SKYTROFA successful Germany this twelvemonth and if approved TransCon PTH adjacent year. With this awesome momentum crossed our pipeline, I would for illustration to reappraisal further specifications from our awesome programs.

Turning to maturation hormone. During nan 4th fourth of this year, we scheme to study nan apical statement results from our world Phase 3 foresiGHt proceedings successful big maturation hormone deficiency, our imaginable 2nd denotation for TransCon maturation hormone. Adult maturation hormone deficiency is simply a superior endocrine uncommon illness characteristic by abnormal assemblage composition, dyslipidemia, insulin guidance and impaired value of life. Analysis has shown dis consequence of big maturation hormone deficiency consequence successful mean analyzed healthcare costs much than 4 times that of a non-growth hormone deficient population. Because TransCon maturation hormone is nan only erstwhile play maturation hormone merchandise releasing unmodified somatropin, we expect it to beryllium nan first big maturation hormone curen to meet aliases transcend nan safety, efficacy and tolerability of regular maturation hormone. Meanwhile, successful nan US, SKYTROFA is knowledgeable nan commercialized occurrence it deserved, because of its unsocial merchandise strengths. As we pre-announced during JPMorgan, 4th fourth 2022 US SKYTROFA gross maturation to €17.1 million, providing a beardown fundament for maturation successful 2023 and after. With our advancement towards explanation description and planned commercialized motorboat successful markets extracurricular nan US, we judge we tin way -- beryllium connected way to build SKYTROFA into nan starring maturation hormone merchandise successful worth by expanding nan full marketplace site. As we person predicted, we are seeing nan consolidation of nan regular maturation hormone marketplace arsenic different manufacturers statesman to exit nan US market.

Turning to TransCon PTH. Excitement continues to build among stakeholders astir this imaginable curen for big diligent pinch hypopara up of nan upcoming PDUFA day of April 13th. Our expanded teams are hired, trained and moving to deepen expert and payor consciousness of this superior wellness and value of life rumor that hypopara causes. We person already made much than 2,000 telephone to expert related to illness consciousness and we are encouraged by their liking successful learning much astir nan multi-organ effect of this illness and its antagonistic effect connected patients value of life. Our commercialized team, aesculapian affairs, premium reimbursement and our manufacturing teams are fresh to motorboat TransCon PTH successful nan US marketplace arsenic soon arsenic imaginable aft approval. Importantly, we are launching TransCon PTH, our 2nd endocrinology uncommon illness merchandise pinch nan aforesaid commercialized infrastructure that has proven its occurrence pinch SKYTROFA. Coming backmost to CMP. As we did pinch TransCon Growth Hormone and TransCon PTH, we ran a robust Phase 2 proceedings to corroborate TransCon CNP’s target floor plan aliases each 4 cardinal pillars, safety, efficacy, tolerability and convenience, and derisk it arsenic nan Phase 2 level. This tin only beryllium done pinch a robust randomized placebo controlled proceedings that does mimic nan pivotal trial. We saw clear occurrence successful ACcomplisH proceedings pinch TransCon CNP, demonstrating superiority complete placebo astatine nan 12 months superior endpoint successful children aged 2 to 10. In addition, we saw clear dose response. All 57 patients who started this proceedings stay successful nan unfastened explanation hold today.

To widen and corroborate these results, including affirmative curen effect observed connected achondroplasia related comorbidities, we are moving our Phase 2b ApproaCH trial. As interrogator I’m alert of nan Phase 2 results wherever I knowledgeable very precocious liking successful our ApproaCH trial, and we expect to complete target enrollment of astir 80 patients successful nan adjacent quarter. During our upcoming extremity of Phase 2 gathering pinch FDA, we expect to collaborate connected really to champion execute a wide curen labeling alternatively than a linear labeling alone.

Shifting to oncology. We are progressing pinch nan improvement of our 2 caller immuno oncology programs, TransCon TLR7/8 agonist and TransCon IL-2 β/γ. With these 2 objective programs, we are positioned this twelvemonth to commencement loop of objective efficacy successful 7 circumstantial tumor types, 9 different indications pinch 4 different operation therapies, including by combining our 2 TransCon oncology merchandise candidates pinch each other. Clinical impervious of conception Phase 2 apical statement results are expected starting successful 2024. In addition, this twelvemonth we would initiate nan randomized Phase 2 proceedings βelieγe 201 utilizing TransCon IL-2 β/γ and TLR7 agonist operation therapy successful caput and cervix cancer. As we successfully demonstrated pinch our endocrinology programs, we are building a coagulated Phase 2 objective impervious of conception for our oncology products successful aggregate tumor types successful nan adjacent 1 aliases 2 years. As you tin see, we person and will ever attraction connected [receiving] best-in-class merchandise floor plan to use patients connected nan 4 cardinal pillars of safety, efficacy, tolerability and convenience, areas successful which we will not compromise. This improvement approach, including highly robust objective proceedings design, has positioned Ascendis to perchance motorboat a caller merchandise aliases denotation each 1 to 2 years, building sustainability, agelong word worth for each stakeholders. This successful objective proceedings further confirms nan powerfulness of nan [indiscernible] exertion level and our merchandise invention [indiscernible] and summation our assurance and likelihood of successful early merchandise candidates. To build connected expanded pipeline and commercialized successes, Ascendis remains connected way to meet aliases transcend our goals outlined successful our Vision 3x3.

I will now move nan telephone complete to Scott for a financial reappraisal earlier we unfastened up for questions.

Scott Smith

To travel connected Jan’s comments, we are excited to spot nan realization of Vision 3x3 pinch a continued travel of caller products and further indications each 1 to 2 years. For example, arsenic Jan noted, we expect to motorboat TransCon PTH successful nan US and SKYTROFA successful Germany this year, followed by nan first European state motorboat of TransCon PTH successful early 2024. With results for SKYTROFA successful big maturation hormone deficiency and TransCon CNP successful achondroplasia connected nan horizon, we expect this cadence of approvals and launches to proceed beyond 2024. In this way, we are creating sustainable agelong word worth for Ascendis and our stakeholders done our proven R&D improvement capabilities. I will quickly touch connected a fewer points. For further specifications connected our afloat twelvemonth 2022 financial results, please mention to our Form 20-F, which is being revenge today. As we antecedently announced successful early January, SKYTROFA US gross for nan 4th fourth of 2022 grew to €17.1 million. These results exceeded nan algorithme that Jan laid retired past May, which projected €16 million.

For nan afloat twelvemonth 2022, full gross was €51.2 million, including SKYTROFA gross of €35.7 cardinal arsenic good arsenic licensed objective proviso and services provided to 3rd parties chiefly VISEN Pharmaceuticals. With profitable maturation of SKYTROFA, our wide operating nonaccomplishment grew astir 2% sequentially to €147.4 cardinal for nan 4th fourth from €144.5 cardinal successful nan 3rd 4th of 2022. Finally, we ended 2022 pinch cash, rate equivalents and marketable securities totaling €743 million. Looking forward, arsenic Jan described astatine nan JPMorgan conference, annualizing 4th fourth SKYTROFA gross of €17.1 cardinal provides a instauration for 2023. In addition, we expect to adhd astatine slightest arsenic galore reimbursed patients this twelvemonth arsenic we did successful 2022, which would supply moreover greater growth. As a result, astatine this time, we judge we are connected way to transcend nan existent Ascendis compiled statement estimate for 2023 SKYTROFA gross of €96 million.

Switching to TransCon PTH. Our PDUFA day is April 30th this year. If approved, we expect to statesman shipping merchandise by nan extremity of nan 2nd quarter. A speedy reminder connected selected cardinal 2023 firm milestones. For TransCon Growth Hormone, arsenic mentioned, we scheme to motorboat SKYTROFA successful Europe, starting pinch Germany successful Q3. And we besides expect to study apical statement information from nan world Phase 3 foresiGHt proceedings successful big maturation hormone deficiency, our 2nd denotation successful Q4. TransCon PTH, we are readying for FDA support by nan PDUFA day of April 30 and motorboat successful nan US by nan extremity of Q2, and we expect nan European committee determination successful Q4. For TransCon CNP, we are connected way to complete enrollment of nan Phase 2b ApproaCH proceedings successful achondroplasia successful Q2. Within our oncology therapeutic area, we expect to study apical statement results and state nan recommended Phase 2 dose for monotherapy dose escalation cohorts for TransCon IL- 2 β/γ later this quarter, and to state nan recommended Phase 2 dose from TransCon IL- 2 β/γ combo therapy pinch checkpoint inhibitor successful Q3. Finally, arsenic you spot pinch our reporting to date, continued optimization of finance systems and processes person enabled america to accelerate our twelvemonth extremity reporting.

With that, operator, we are now fresh to return questions.

Question-and-Answer Session

Operator

Thank you. Our first mobility for coming will beryllium coming from Fye of JPMorgan.

Jessica Fye

Thanks for nan comments connected really to deliberation astir SKYTROFA income this year. Can you remark connected your comfortableness level pinch statement estimates for TransCon PTH this year? And really while we're astatine it, wherever is that statement fig based connected your latest compilation of nan expert numbers?

Jan Mikkelsen

Jess, are you reflecting into SKYTROFA aliases TransCon PTH?

Jess Fye

For PTH.

Jan Mikkelsen

I really person not looked astatine nan statement number for PTH. I don't deliberation we person collected that information. So I don't deliberation we tin really reside that. We tin chiefly reside nan places wherever we consciousness assured to springiness an algorithme that tin bespeak our anticipation arsenic we person done from SKYTROFA.

Jessica Fye

Okay, I'm going to inquire thing other then. Can you show america really galore cumulative caller diligent prescriptions location were for SKYTROFA arsenic of your end? I deliberation you were antecedently giving that 4th by quarter. And tin you besides show america erstwhile we should look for nan adjacent update from nan Phase 2 hold for TransCon CNP?

Jan Mikkelsen

Let's conscionable spell backmost to really we fundamentally are looking connected nan forecasting related to nan gross of SKYTROFA successful nan US successful 2023. What we person seen successful present successful '22 that period by month, we person accrued nan number of caller patients that sewage reimbursed, and we person continued to spot this inclination besides successful 2022. This is why it was important to look connected nan 4th quarter, because we besides seen astatine nan aforesaid time, we're seeing and really very beardown retention. When you commencement connected SKYTROFA, you enactment connected SKYTROFA. When we return nan €17.5 cardinal and multiply that pinch 4, Scott is calculating, it’s astir €70 million. And we fundamentally will expect an acceleration of nan number of caller patients, because of a batch of bully reason. And I tin travel backmost to that, Jesse, if you want that. So we really expect to spot much patients per period of caller reimbursed patients that we really saw successful '22. We're emotion really, really, really assured that we will transcend nan statement number that is retired connected nan thoroughfare coming related to SKYTROFA.

The constituent that basal -- providing our summation successful number of monthly caller reimbursed patients is that nan expert is starting really to get nan knowledge astir nan merchandise spot of SKYTROFA. It's not thing you acquisition successful 1 month, you request to spot six months, 12 months of data. And this is what we're starting to see, truthful we're really seeing really we really person a highly differentiated merchandise compared to regular maturation hormone. The different constituent is that we spot nan consolidation of nan regular maturation hormone marketplace that started for astir 3 years agone wherever we saw aft our Phase 2 information that nan consolidation of nan regular maturation hormone marketplace is really kicking successful now. So that is simply a major, awesome move distant from immoderate of nan six regular maturation hormone players, which each person nan aforesaid product. So this is why we consciousness that assured astir really we really would turn SKYTROFA into nan astir weighted merchandise successful nan maturation hormone marketplace successful nan adjacent future. Related to CNP, you had a mobility related to CNP. Jess, you tin specify precisely what you wanted to know, though.

Jessica Fye

Well, I deliberation successful nan past, you talked astir nan precocious velocity for a proportionality of patients that had been connected up to a definite clip point, and I deliberation we're going to possibly update that 1 each nan patients sewage retired to that clip point. Is location benignant of scheme to update that information and erstwhile should we expect that?

Jan Mikkelsen

Yes, we are readying to springiness you this data. And we deliberation is extremely, highly important to springiness you this information wherever we spot nan continuous effect of Ascendis Pharma’s TransCon CNP, because I'm still successful this highly struggling manner. And this is what we really had sewage a batch retired of analyzing each nan information for our ACcomplisH proceedings to find retired why they're staying 100% connected this treatment, and we starting to get a much, overmuch amended knowing of that. And this is why we now are discussing nan regulatory agencies really we tin person different secondary endpoint that's really are reflecting really we are addressing really nan comorbidities and not conscionable linear growth. Linear maturation is not really nan biggest rumor for this diligent group, they activity nan comorbidity and different effects of disease. And this is why we judge TransCon CNP is simply a unsocial merchandise because it person continuous vulnerability of nan CNP molecule and truthful changing things that is not only related to linear growth.

Operator

Our adjacent mobility will beryllium coming from Ahmad of Bank of America [Operator Instructions].

Tazeen Ahmad

As it relates to HPT, tin you conscionable springiness america an update, if you haven't already, connected really galore patients you've enrolled successful nan Early Access Program truthful far? And do you person a consciousness of really galore patients will beryllium enrolled successful that programme by nan clip of nan PDUFA and past pursuing pinch nan launch?

Jan Mikkelsen

I deliberation that is an utmost -- first of all, we are highly pleased pinch really nan programme is progressing and our regulatory interaction, and besides that we sewage support to commencement an EAP programme pinch [indiscernible] springiness nan opportunity to get patients nether nan curen earlier we get nan expected approval. We are executing connected that, really starting nan full system. And we will explicate erstwhile we travel to nan support process, what is nan number of patients we will person successful this trial, and besides really we proceed pinch patients.

Tazeen Ahmad

Maybe conscionable a follow-up then. Would you expect that to beryllium an early root of patients to person to commercial?

Jan Mikkelsen

I conscionable deliberation we are addressing a cardinal constituent successful nan EAP program. We are addressing nan diligent group that already were knowledgeable pinch a PTH curen regime, a short acting PTH treatment. It could beryllium nan [indiscernible], it could beryllium [indiscernible], it could beryllium [time loss] aliases personification else, but that was precisely what we’re addressing. The immense organization wherever we fundamentally recruited diligent to our Phase 2 programme and our Phase 3 program, they’re coming from what we telephone diligent that ne'er really had been exposed to PTH treatment. So I don't spot -- that is really, really a differentiation betwixt these 2 diligent groups. I deliberation some diligent groups person nan aforesaid precocious unmet aesculapian need, they person nan aforesaid use of nan PTH treatment. So I don't spot immoderate benignant of quality betwixt these 2 groups related to beryllium firestarter successful our programs.

Operator

Our adjacent mobility will beryllium coming from to Tazeen Ahmad of Bank of America.

Tim Lee

I deliberation she conscionable asked nan question, truthful I'd spell to nan adjacent one.

Operator

The adjacent mobility is coming from David Lebowitz of Citi.

David Lebowitz

As nan PDUFA day is approaching, could you springiness america immoderate penetration into what nan explanation mightiness yet look like? I cognize that NATPARA was considered an adjunct, you activity to beryllium going much arsenic a hormone replacement, and NATPARA has nan beingness of a blackbox for osteosarcoma, but you didn't really person an osteosarcoma experience. So could that blackbox beryllium removed? Just beryllium funny to perceive your thoughts.

Jan Mikkelsen

I deliberation erstwhile we look connected nan biology and nan merchandise creation of TransCon PTH, we are providing a unchangeable physiological level of PTH 24 hours, 7 days a week. We are not providing immoderate hyperphysiological attraction of PTH that tin supply to nan anabolic effect that successful animal exemplary person been associated pinch nan osteosarcoma circumstantial to rat model. And from that position is that and besides why we sewage a waiver to make a carcinogenic proceedings aliases animal trial, we will not expect and we person not seen immoderate denotation successful our labels chat that we will beryllium coming successful nan same, you tin see, group of short acting PTH that fundamentally sewage nan aforesaid people labeling, because we are providing a complete different merchandise floor plan than nan short acting PTHs. So to our champion knowledge coming and successful our discussions, we don't expect immoderate REMS programme aliases we don't expect immoderate blackbox warning. So that was your first question. And you're rather right. How we designed TransCon PTH was really to beryllium a merchandise floor plan that was reflecting hormone replacement alternatively of an adjunct. That’s why to beryllium successful successful nan objective proceedings you request to extremity 100% from progressive vitamin D and you besides request only to return calcium supplements that really are conscionable reflecting a normal multivitamin from [indiscernible]. So we are really addressing a complete different merchandise profile.

Operator

And our adjacent mobility is coming from Li Watsek of Cantor.

Li Watsek

I conjecture for TransCon PTH, conscionable wondering if you tin stock immoderate of nan latest feedback that your income squad possibly received from payors and physicians? And past possibly talk astir your latest thoughts connected really you mightiness attack pricing and marketplace access?

Jan Mikkelsen

When I deliberation astir nan patients, erstwhile I deliberation astir this physicians, nan measures person not been different for nan latest. Everyone admit that hypopara is simply a superior illness and everyone understand nan use of replacing a missing pinch a physiological level 24 hours, 7 days a week. How it some reside short word symptoms, for illustration value of life, urinary calcium but besides agelong word risk. And what we’re seeing is that nan consciousness of that is being building up much, overmuch much astir nan consciousness of nan disease. And I judge this is wherever we travel successful pinch our indication, first telling astir nan consciousness of nan disease, which are really a large portion of what is happening coming pinch each our established infrastructure present successful nan US. And aft an approval, we tin spell retired and explicate really we tin use this benignant of disease, some short word and agelong term. So I really, really, really consciousness that we are right. What we did pinch SKYTROFA is nan guidance we do pinch ever our product. We create champion successful people products, addressing existent unmet aesculapian need. And we return a premier responsible pricing business wherever we judge if you really create a merchandise that really reside a existent unmet aesculapian request pinch a existent product, that is capable for some nan patient, nan physician, nan nine and nan payor, for america to stock that cake, because past everyone is winner, nary 1 is loser. And this is wherever we want to beryllium pinch each of our products. We request to person them truthful highly differentiated, addressing a existent unmet aesculapian request and everyone believes it's a triumph for everyone, and we tin spot that pinch TransCon PTH.

Operator

Our adjacent mobility is coming from Paul Choi of Goldman.

Paul Choi

I want to ask, fixed that you're utilizing nan aforesaid infrastructure to marketplace PTH successful nan US that you're presently utilizing for SKYTROFA. I guess, are location immoderate learnings that you mightiness stock connected nan -- from motorboat SKYTROFA that you would deliberation beryllium applicable, aliases what changes would you make I conjecture successful position of either your thoughts connected approaching your payor entree and/or contracting compared to SKYTROFA? And past I person a pipeline mobility arsenic a follow-up.

Jan Mikkelsen

We are utilizing nan aforesaid infrastructure. Sure, we person dedicated income force, we person dedicated group for nan 2 different products. But you cognize there's a immense quality to beryllium nan first merchandise to spell retired and motorboat wherever you found each nan infrastructure, IT systems, each nan different basal teams that is basal to really to motorboat a commercialized product. We person large consequence astatine that now. We coming from a shape wherever we motorboat and shape an already successful status, commercialized infrastructure built from Joe and nan different group successful Princeton present successful nan US. So what we’re doing is that we fundamentally are placing what I telephone TransCon PTH into an infrastructure that already has proven it’s capacity pinch a product, I believe, really addressing immense unmet aesculapian request whereas nary replacement treatment. I deliberation this is simply a basal -- a immense success.

Paul Choi

And past arsenic a follow-up, conscionable successful position of nan pipeline. Do you scheme to people nan baseline diligent characteristics for nan 80 children that are being enrolled successful nan ApproaCH trial? And past could you besides specify successful position of your oncology program, nan caput and cervix organization that you're readying to pursue, is it conscionable HPV affirmative aliases is it station PD-1 and station [erbitux], if you tin possibly adhd a small colour connected that, that'd beryllium great? Thank you for taking our questions.

Jan Mikkelsen

I deliberation Stina will return number two, aliases you tin return 1 connected [Multiple Speakers]. Let maine return a number one, it’s a very, very absorbing question, because I do not cognize if nan mobility nan words really are addressing, because I really deliberation we person published each nan demographic from our diligent organization and demographic that person been into nan execute trial, nan 57 that's still successful it. So I -- somewhat small spot puzzling pinch that question, because each information is out/ past you tin say, hey, why did you not travel successful pinch nan analyse precocious velocity prescreening, because it’s wholly irrelevant for nan objective efficacy of it, you cannot usage and analyse precocious velocity that person been collected earlier they spell into nan trial, because we person 40% of nan transmission betwixt 2 and five, which you look and spot [indiscernible] astir built nan normal analyse precocious velocity. And nan first 4 years, you person a heavy deceleration of analyse precocious velocity. So if you return and analyse precocious velocity conscionable collected up to 12 months earlier they spell into a trial, it's not reflecting immoderate meaningful worth that spell successful and comparison to nan analyse precocious velocity you comparison successful this diligent group, because nan commencement connected nan [indiscernible] already two. This is why you do what is evident successful supplier development, you’re making a placebo group. This is why you person a placebo group, and I deliberation that is nan cardinal constituent to do, look astatine our dosing from 6 to 100 and past you tin return six for illustration astir for illustration besides placebo group and past you tin return nan six and -- aliases nan placebo activity into nan achondroplasia circumstantial precocious ratio of a matching, they're matching 100%. But comparison this it’s my basal technological delirium and only are misleading and person not reflecting immoderate benignant of coagulated technological worth successful interpolating nan data. Stina?

Stina Singel

In oncology, we are evaluating for impervious of conception efficacy successful 7 different tumor types. You're right, we do person 1 of our privilege areas is caput and cervix cancer. We are evaluating successful first aliases 2nd statement metastatic caput and cervix crab arsenic a dose description cohort azygous limb successful nan IL-βelieγe study, and those patients will beryllium -- will person had nary much than 1 statement of chemotherapy containing regimen successful nan precocious metastatic setting. And nan randomized Phase 2 study, βelieγe-IT-201 study will beryllium successful nan neoadjuvant setting. So these are patients successful a non-metastatic mounting earlier they get surgery, they will get systemic curen earlier room and we're looking astatine pathologic consequence arsenic our superior endpoint to look for grounds of impervious of conception efficacy.

Operator

Our adjacent mobility is coming from Derek Archila of Wells Fargo.

Derek Archila

So conscionable 2 really speedy ones from us. Jan, I conscionable wanted to confirm, I thought I caught you saying that you were successful labeling discussions for TransCon PTH. So I conscionable wanted to corroborate that. And past also, I guess, erstwhile should we expect further updates from ACcomplisH, is that thing we should see, again, first half of this year, 2nd half of this year?

Jan Mikkelsen

When you spell done an support process, I think, for me, is benignant of scheme process, 4 months earlier an expected support day [indiscernible] 3 months earlier an expected support [indiscernible] [this request to happen] 2 months earlier that. And truthful if anyone location had been to an support process, and I deliberation we person 10 weeks earlier nan PDUFA day now. If you're not person started astatine branded discussion, nan consequence of not getting support is high. So therefore, I judge this is simply a measurement by search really we are progressing to nan support process, are we really connected way of what everything needs to hap successful that expected time, is it that’s not happening. I will beryllium highly worried and find retired what is going on. And truthful yes, we are successful a labeling discussion, because you should beryllium that astatine slightest 3 months earlier an expected approval. And this is why I consciousness that I'm assured that I person not seen thing that not springiness maine a belief that TransCon PTH isn’t merchandise that is approved. I judge and I cannot really retrieve each our firm milestones now. Sorry for that, because that's a small spot excessively galore of them. But I judge that is successful Q4 we will springiness you an update, again, related to that 57 patients that will travel retired from nan ACcomplisH tribe. So it will beryllium successful nan 2nd half of this year.

Operator

The adjacent mobility is coming from Josh Schimmer of Evercore.

Josh Schimmer

First connected SKYTROFA. Could you elaborate a small further connected what you're seeing successful position of regular maturation hormone options withdrawn from nan market? I cognize location person been immoderate reported shortages, but it didn't recognize that reflected nan outfall withdrawals. So who person you seen this drafting and do you expect others to follow? And past really do you expect nan effect of Novo Nordisk perchance launching a erstwhile a week maturation hormone action arsenic good iun nan marketplace later this year?

Jan Mikkelsen

Let maine commencement first connected nan regular maturation hormone, because I judge that is simply a textbook that really -- and if I person conception it’ll beryllium imaginable [indiscernible] successful my studies and my last project, but it's really, really what I telephone a textbook [example]. Growth magnitude aliases nan regular maturation among marketplace was nan first, I sewage biosimilar wherever Sandoz and Teva entered pinch their bioequivalent type connected it. And there, Sandoz had six players, it is regular marketplace segment, each of them providing precisely nan aforesaid entity, nan aforesaid treatment. So you tin alteration this backmost and distant betwixt nan merchandise limited connected rebate and different things for illustration that that was different successful formulation, that was a small spot different successful devices and different things for illustration that. So what we saw for astir three, 4 years erstwhile they came retired pinch our Phase 2 data, we saw already that immoderate of nan large subordinate -- galore started immoderate measurement to reconsider really to beryllium really played erstwhile that's coming and superior curen into this segment. And that will beryllium -- which will beryllium highly different stages compared to what we telephone nan regular marketplace segment.

At that time, we already saw 3 of nan company's basal removing their income forms, this is measurement one, arsenic I call. Your region income force. The 2nd 1 is that you spell to nan adjacent stage, you region nan [indiscernible], past you extremity up manufacturing, which I deliberation 3 aliases 4 them person done it now. And past you fundamentally are successful a position wherever you are providing galore patients that already are [established] connected your product, because you person nary dream wherever you tin alteration and return caller diligent successful and you're providing them. What hap successful mounting up for nan diligent now that it look for illustration a normal audience, erstwhile they 2 showed america of aggregate products, aggregate position of their maturation hormone. And because that consolidation of nan regular maturation hormone hap and really is successful nan last place, I judge nary of nan different 1 could return over. So you really spot a shortage of maturation hormone curen successful nan US. And sure, I request to judge that nan use of that is simply a awesome point for us, because it's some accelerating astatine nan aforesaid clip wherever group really spot nan benefit, get nan objective acquisition really we differentiate it, beryllium having diligent for 1 twelvemonth aliases thing connected treatment. So they really, really, really spot this benefit. And I deliberation this is simply a awesome thing, judge for america and we’re really hopeful we tin thief arsenic many, galore arsenic patients to debar going into a shortest of nan curen effect. Your 2nd question?

Josh Schimmer

Actually, I was going to inquire whether, fixed your position of nan differentiation of TransCon CNP, if you’d considered filing for breakthrough designation?

Jan Mikkelsen

Yes. First of all, Josh, you besides person nan mobility reflecting astir potential, nan entrant of Novo Nordisk agelong acting product. When I look connected nan paradigm shift, I telephone it paradigm shift, because it comes from nan clip wherever each nan regular maturation hormone were identical, nan aforesaid treatment, nan aforesaid mode of action. When you spell complete to nan agelong acting, each of them are providing complete different objective profile. They're nan only 1 that really lucifer an improved type of nan regular maturation hormone wherever you get each nan use endocrine benefit, some related to changing not only linear maturation successful nan periodic mounting but besides nan different -- associated endocrine use for illustration assemblage composition, metabolic profile, lipid [indiscernible], cognitive effect and everything that you see, because we person nan aforesaid unmodified somatrope molecule. So this is why it's besides important for america to look astatine our Phase 3 successful big maturation hormone deficiency, because nan 2 different imaginable agelong acting wherever we judge there's imaginable 1 little now but [indiscernible] showed basal not immoderate betterment connected assemblage creation successful that Phase 3 trial. Novo Nordisk showed they tin only get nan half of nan liking basal to regular maturation hormone. And this is wherever we judge pinch our unmodified somatrope imaginable we will beryllium astatine slightest arsenic bully arsenic regular maturation hormone. So we judge that our merchandise floor plan is ever truthful highly differentiated to some regular maturation hormone and different agelong acting maturation hormone that we ever will supply america nan objective use compared to each different curen regimes.

Operator

Our adjacent mobility will beryllium coming from Leland Gershell of Oppenheimer.

Leland Gershell

Two from me. Jan, I cognize you had responded earlier that you don't expect a REMS aliases blackbox informing connected nan PTH label. But could you remark connected immoderate imaginable for monitoring requirements pinch patients connected nan product?

Jan Mikkelsen

Could you explain your question, what do you mean exactly?

Leland Gershell

Well, successful different words, if patients request to beryllium monitored for serum calcium, bony biomarkers whatnot?

Jan Mikkelsen

That is an mobility wherever you will opportunity will beryllium supply an beta stableness for this diligent group than they person successful nan existent setup wherever nan basal are being monitored for calcium really, really, really often. And I judge you will spot it successful different stages. I judge erstwhile you modulation complete from what we telephone nan accepted portion of therapy complete to TransCon PTH, I deliberation that will beryllium astatine slightest nan aforesaid benignant of monitoring, because you want to beryllium rather judge you stabilize nan diligent successful nan correct manner. When they are stable, which we spot aft 1 to 2 twelvemonth connected a PTH dose, wherever we spot much and much stableness coming successful because calcium metabolic strategy aliases calcium hemostasis starting to beryllium stabilized, I will dress to spot from a diligent position that you will perchance request a list, what we call, monitoring of it. I deliberation this is wherever I judge that constituent for illustration [indiscernible] is not typically thing you typically will analyse for immoderate diligent group successful this way. What we person seen successful our objective study wherever now diligent are for complete three, 4 years, your fundamentally seeing that we do normalization much and much and much of each parameter, and that is includes some bony density and is besides including bony markers. So I will not expect that it will beryllium portion of a modular monitoring.

Leland Gershell

And 2nd mobility from maine is pinch respect to achondroplasia. Obviously, nan superior endpoint and for regulatory purposes, it's astir type velocity. But arsenic you had mentioned earlier, location are galore different benefits that a replacement CNP could provide. Could you conscionable benignant of pass america arsenic to which of nan different benefits that possibly not captured by superior endpoint type information but would beryllium very important, seen arsenic astir important by nan [achondro] community?

Jan Mikkelsen

Yes, I deliberation this is why we processing TransCon CNP. We really are processing it to supply an curen that judge is addressing linear growth, but we tin harvester it pinch SKYTROFA and past I tin -- from a objective concept, I will expect astir you tin determine what benignant of linear maturation you will have. But what we really want to ensuring that we are addressing nan underlying comorbidities of nan diligent and really we measuring them is to person circumstantial achondroplasia circumstantial comorbidities. This is comorbidities that is coming and being reported connected high, high, precocious predominant from nan achondroplasia from erstwhile you spot [Technical Difficulty]. And past we besides are processing an diligent circumstantial reported result measuring wherever we're trying to drawback each nan benefits they're feeling, because they're really seeing a immense benefit. One of nan things that is clear for us, it's not person batch of different sectors, not conscionable related to [Technical Difficulty]. We spot monolithic change, we spot really nan usability beta successful nan beingness measurement to balance, do normal work, do normal cognition and different things for illustration that. And this is each those elements we will effort to capture. At nan aforesaid time, we will seizure nan achondroplasia circumstantial constituent for illustration -- fundamentally for illustration [Technical Difficulty] patients and different things for illustration that [Technical Difficulty] putting.

Operator

Our adjacent mobility is coming from Andreas Argyrides from Wedbush.

Andreas Argyrides

So erstwhile you're reasoning astir -- this is for PTH. When you're reasoning astir nan opportunity -- launching nan opportunity of PTH, really are you reasoning astir it compared to nan NATPARA launch? And past possibly you tin springiness america immoderate insights connected really NATPARA coming disconnected nan marketplace astatine nan extremity of '23, informing your expectations for nan launch? And past I person a travel up?

Jan Mikkelsen

I'm not comaring TransCon PTH successful immoderate measurement to NATPARA. It will person complete different labeling, person complete different objective outcomes, person complete different objective benefit. So conscionable complete different effect connected value of life. So I'm not utilizing that arsenic a benchmark connected anything. I'm not comparing that. There was a merchandise that came retired pinch a labeling arsenic an adjunct, this is not what we’re addressing. It was a merchandise that came retired not seeing use connected value of life, it was a merchandise that came retired not showing immoderate use connected 24 hr urinary calcium, not really addressing nan underlying disease. So you tin -- I don't usage that besides immoderate benchmark, it's wholly meaningless for me.

Andreas Argyrides

I conjecture possibly nan opportunity that there's nary approved merchandise connected nan market, location were immoderate patients, mostly successful Europe arsenic well, location were still connected it aliases they had entree to it nary longer. Are those going to beryllium early adopters?

Jan Mikkelsen

Yes, I deliberation really -- this is precisely arsenic connected somebody's reside before. Yes, that is diligent that has been exposed to short acting PTH treatment. They are utilized to regular injection and different elements for illustration that. But wherever we spot nan immense use is independent of nan inheritance is that being exposed to short acting PTH aliases not. So I don't judge location is simply a little request for a diligent to get connected TransCon PTH curen if you travel from nan group that has been connected short acting PTH aliases person ne'er seen a short acting PTH. The diligent that person been connected short acting PTH person possibly a much communal knowing of regular injection, person much communal knowing astir what they request to do arsenic process to get that to happen. But I don't judge that it’s a large obstruction for immoderate of nan groups.

Andreas Argyrides

And past conscionable a speedy 1 connected SKYTROFA, and possibly you tin elaborate and I don't cognize if you've covered this already. Sorry, if you did and I missed it. But if you tin elaborate connected nan motorboat dynamics that are driving growth. Are you seeing higher move rates from regular maturation hormone? And that's it for me. Thanks, guys.

Jan Mikkelsen

We only spot a higher move complaint from regular maturation hormone, but we’re besides seeing an accrued number of caller diligent coming connected nan treatment. But nan wide number goes up everywhere. So it's not apt 1 getting less, some of them are improving up to a really, really caller tallness each nan time.

Operator

And our adjacent mobility is coming from Yaron Werber of Cowen.

Yaron Werber

Jan, I've 2 interrelated questions connected much coverage. With PTH coming soon, location isn't really a competitor. Do you request to statement aliases will you statement pinch PBMs to get connected formulary, possibly benignant of talk astir your thoughts there? Obviously, nan discounting shouldn't beryllium very precocious astatine that point. And past secondly, now that you'll person a 2nd program, 2nd merchandise approved, benignant of wrong nan endocrine bag. Does it springiness you much leverage to discuss amended for a placement for SKYTROFA?

Jan Mikkelsen

We cognize really comments astir really our marketplace measure strategy is and will be, and really we are really are addressing nan reimbursement strategy successful nan US. But for your information, erstwhile we ever look astatine a product, because you tin do nan aforesaid thing, I tin springiness you sanction of products successful nan US that fundamentally are successful a position to make aggregate cardinal successful gross successful nan US without fundamentally being provided precocious rebate. And I deliberation we will travel this pathway, because we are providing specified a use to nan patient, nan expert and nan nine that we consciousness this is simply a measurement we will proceed our marketplace versus strategy. Compared to SKYTROFA, we are really beautiful satisfied pinch our coverage. We judge nan spot of nan product, nan differentiation, don't really lead america to supply into an highly rebated product. I deliberation that is nan strengths of having a highly differentiated product.

Operator

Thank you. That's each nan clip that we person for today. Thank you for joining nan convention call. You each person a bully evening.

Jan Mikkelsen

Thank you.

Tim Lee

Thank you truthful much. See you.

Editor: Naga



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