Ascendis Pharma A/s · NASDAQ:ASND

Ascendis Pharma A/s — Technical Presentation (October 2025)

87 slides from Ascendis Pharma A/s's technical presentation, Oct 30, 2025

  1. Slide 1: Site Initiation Visit – for US sites

    ASND0037 & ASND0033 Site Initiation Visit – for US sites A US Multi-center, Prospective, Non-interventional, Long-term, Effectiveness and Safety Study of Patients Treated with SKYTROFA (lonapegsomatropin) (SkybriGHt) A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study

  2. Slide 2: SIV Agenda

    SIV Agenda  Key Study Team Contacts  SkybriGHt Only o Special Consent  Ascendis Pharma Introduction o Schedule of Assessment o Clinical Outcome Assessments  SKYTROFA® (lonapegsomatropin-tcgd) US o Medidata eCOA/EDC Label Overview o eCOA and Patient Cloud Application o Autoinjector Data Collectio

  3. Slide 3: Key Contacts

    Key Contacts Ascendis Pharma (Sponsor) Project Management Lead Kirsten Hoyer KIR@ascendispharma.com US Medical Affairs Director Deborah Bowlby, MD Debb@ascendispharma.com Safety Lead Rushi Purohit RPT@ascendispharma.com Field Medical Team Lead Selina Mullins SMU@ascendispharma.com Field Medical Team

  4. Slide 4: Ascendis Pharma Mission, Vision, and Values

    Ascendis Pharma Mission, Vision, and Values Our Mission Our Vision Our Values We are applying our innovative Build a leading fully integrated Patients, Science, and Passion are TransCon technology platform biopharma company providing a the core values that guide our to develop novel therapies meanin

  5. Slide 5: Clinical development pipeline

    Clinical development pipeline All pipeline product candidates listed are investigational Endocrinology Rare Disease – Ascendis Programs Endocrinology Rare Disease – Partner Programs Ascendis Programs Location Status Clinical Trial Partner Programs Location Status Clinical Trial TransCon hGH (lonapeg

  6. Slide 6: Product approvals

    Product approvals Therapeutic Area Compound name Regulatory Approvals Pediatric Growth Hormone lonapegsomatropin • United States 1,4 Deficiency (lonapegsomatropin-tcgd in the • European Union (including United United States; developed as Kingdom at time of approval) 2-5 TransCon hGH) • European Econ

  7. Slide 7: Lonapegsomatropin-tcgd (TransCon hGH) Design

    Lonapegsomatropin-tcgd (TransCon® hGH) Design Somatropin TransCon TransCon linker (hGH; active) carrier Receptor Renal clearance Somatropin Linker cleavage (hGH; inactive) dependent upon pH and temperature • Lonapegsomatropin-tcgd is a prodrug of somatropin, administered once-weekly and designed to

  8. Slide 8: SKYTROFA (lonapegsomatropin-tcgd) US Label Overview

    SKYTROFA® (lonapegsomatropin-tcgd) US Label Overview 8 CONFIDENTIAL

  9. Slide 9: Indications, Usage, Dosage and Administration

    INDICATIONS, USAGE, DOSAGE and ADMINISTRATION INDICATIONS AND USAGE • SKYTROFA (lonapegsomatropin-tcgd) is a human growth hormone indicated for the treatment of pediatric patients 1 year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hor

  10. Slide 10: Indications, Usage, Dosage and Administration

    INDICATIONS, USAGE, DOSAGE and ADMINISTRATION Missed Doses • Administer a missed dose as soon as possible and not more than 2 days after the missed dose • To avoid missed doses, SKYTROFA (lonapegsomatropin-tcgd) can be taken 2 days before or 2 days after the scheduled dosing day. Resume once-weekly

  11. Slide 11: Contraindications

    CONTRAINDICATIONS CONTRAINDICATIONS SKYTROFA (lonapegsomatropin-tcgd) is contraindicated in patients with: • Acute critical illness after open heart surgery, abdominal surgery or multiple accidental trauma, or those with acute respiratory failure due to the risk of increased mortality with use of ph

  12. Slide 12: Clinical Studies - Adverse Reactions

    CLINICAL STUDIES - ADVERSE REACTIONS Clinical Study Experience As clinical studies are conducted under widely varying conditions, adverse reaction rates observed in the clinical studies of a drug cannot be directly compared to rates in the clinical studies of another drug and may not reflect the rat

  13. Slide 13: Clinical Studies

    CLINICAL STUDIES Treatment-Naïve Pediatric Patients with Growth Hormone Deficiency (NCT02781727) A multi-center randomized, open-label, active-controlled, parallel-group phase 3 study was conducted in 161 treatment-naïve, prepubertal pediatric subjects with growth hormone deficiency (GHD); 105 subje

  14. Slide 14: SkyPASS & SkybriGHt Project Design and Protocol Review

    SkyPASS & SkybriGHt Project Design and Protocol Review

  15. Slide 15: SkybriGHt (ASND0037) US registry

    SkybriGHt (ASND0037) US registry US Prospective Non-interventional, Effectiveness and Safety Study: 5-year Enrollment + 5-year Follow-up N ~ 900 subjects (all subjects treated with lonapegsomatropin, RWE) Retrospective data analysis Primary Objectives Key Inclusion Criteria  Describe demographics,

  16. Slide 16: What is different in SkybriGHt?

    SkybriGHt Study Design What is different in SkybriGHt? • Enrollment in SkybriGHt is US-only targeting 900 patients • Patient participation ends once patients are no longer prescribed SKYTROFA ◦ The End of Study (EOS) date will be 14 days after last dose of SKYTROFA ◦ Each site may enroll a maximum o

  17. Slide 17: SkyPASS (ASND0033) Global Post Authorization Safety Study

    SkyPASS (ASND0033) Global Post Authorization Safety Study US & Europe Prospective Non-interventional, Long term Safety Study: 5-year Enrollment + 5-year Follow-up N ~ 500 subjects (all subjects treated with lonapegsomatropin, PASS) Primary Objective Key Inclusion Criteria  To evaluate the occurrenc

  18. Slide 18: SkyPASS Study Design

    SkyPASS Study Design • Multi-center, prospective, non-interventional, post-marketing observational study of patients prescribed SKYTROFA to treat GHD in the US and the EU. • Patients are managed according to routine clinical practice. Assessments are standard of care (no additional visits, examinati

  19. Slide 19: Real world observational studies

    Real world observational studies • Real world observational studies permit long-term follow-up of large numbers of patients prescribed any chronic medication according to standard clinical practices. • This data is critical for the evaluation and characterization of the real-life effect and risk pro

  20. Slide 20: Study Timelines

    Study Timelines ESTIMATED TIMELINES SkybriGHt SkyPASS LAST SUBJECT IN March, 2028 September, 2028 END OF DATA COLLECTION* January, 2033 September, 2033 20 CONFIDENTIAL * Estimated dates

  21. Slide 21: Study Objectives

    Study Objectives Primary objectives Exploratory objectives SkybriGHt SkyPASS SkybriGHt SkyPASS To describe demographics, clinical characteristics, To evaluate the occurrence To examine the impact of and GH treatment patterns of neoplasms (benign, adherence on clinical To assess effectiveness of of p

  22. Slide 22: Study Objectives

    Study Objectives Secondary objectives SkybriGHt SkyPASS To collect clinical outcome assessments (COAs) including To evaluate the occurrence of renal, hepatic, immunologic, and functioning and well-being, treatment satisfaction, and patient neurologic AEs in patients treated with SKYTROFA preference.

  23. Slide 23: Population

    Population Population SkybriGHt SkyPASS Only in United States (US) at centers that manage patients Patients from centers in the US and Europe are eligible for with SKYTROFA enrollment in the study, if they are on treatment with SKYTROFA Patients may be enrolled in the study if they are being This st

  24. Slide 24: Eligibility Criteria

    Eligibility Criteria Inclusion Criteria Exclusion Criteria SkybriGHt SkyPASS SkybriGHt SkyPASS Patients who are on treatment Pediatric patients with GHD who Patients participating in any with SKYTROFA are on treatment with interventional clinical study for short SKYTROFA stature Patients being treat

  25. Slide 25: Baseline procedures

    Baseline procedures At baseline visit (date of enrollment) the following patient’s details (most recent) will be recorded: Main Baseline Procedures* *For more details, refer to section 9.2.2.1 in SkybriGHt SkybriGHt & SkyPASS protocol and section 8.3 from SkyPASS protocol. Demographics (date of birt

  26. Slide 26: Schedule of Assessments

    Schedule of Assessments • Visits will be scheduled and conducted according to the clinical site’s routine clinical practice • Relevant history and results of physical examinations and investigations which are performed as part of routine clinical practice will be collected. • Data will be entered vi

  27. Slide 27: Patient Retention Strategy

    Patient Retention Strategy • Individual patient follow-up will be from 5 to a maximum of 10 years after enrollment. • The consenting patient/parent/legal guardian will complete a contact form (including physical address, mailing address, phone number, and e-mail address) and will also be asked to id

  28. Slide 28: Lost to Follow Up

    Lost to Follow Up • If there has been no data entered by the site for an enrolled patient by the end of 6 months of the last data entry time point, the study management team will contact the site to confirm the patient’s status and whether the patient is still under the care of the site and its heal

  29. Slide 29: Study Responsibilities

    STUDY RESPONSIBILITIES 29 CONFIDENTIAL

  30. Slide 30: Study Responsibilities

    Study Responsibilities • Sponsor • CRO • IRBs/IECs • Investigator/Staff • CRAs/SMAs Have a shared responsibility to ensure proper conduct of the program. 30 CONFIDENTIAL

  31. Slide 31: Responsibilities Regarding Electronic Records

    Responsibilities Regarding Electronic Records • Ensure that the system and data access is restricted to trained and delegated individuals and kept confidential • If only paper copies of electronic records can be provided, these should be marked as ‘certified’, dated, and signed by PI or Sub-Investig

  32. Slide 32: Source Documents

    Source Documents Source Documentation • Maintain complete and accurate study documents including the source documents. Definition of Source Documents • The first recording of a data point or clinical observation. • All reports and records necessary for the evaluation and reconstruction of the clinic

  33. Slide 33: Investigator/Physician Responsibilities

    Investigator/Physician Responsibilities • Site staff qualified through education, training, and experience to assume responsibility for the proper conduct of the study ◦ In the absence of FDA 1572, the Site Responsibility Log (SRL) must be completed at the SIV to identify all site staff delegated ta

  34. Slide 34: Investigator/Physician Responsibilities

    Investigator/Physician Responsibilities Planned Deviations • If planned deviations occur, submit these to the IRB/IEC and sponsor for review and acknowledgement • Planned deviations need to be approved before implementation Financial Disclosure Form Updates • Provide Ascendis Pharma Registry Team wi

  35. Slide 35: Investigator/Physician Responsibilities

    Investigator/Physician Responsibilities Data Entry • Enter all visits and corresponding data into EDC in a timely manner • Respond timely to any/all data queries. • Respond promptly to corrective action requests during the study • Review, sign, and date all adverse events (AEs/SAEs) reports in a tim

  36. Slide 36: Informed Consent Form Process

    Informed Consent Form Process • The ICF process must be documented in the medical records • Allow ample time for questions and discussion regarding the study, procedures, and expectations. • ICF/Informed Assent Form (IAF) must be signed and dated by the patient/patient’s parent or guardian AND the I

  37. Slide 37: Adverse Event Reporting

    ADVERSE EVENT REPORTING 37 CONFIDENTIAL

  38. Slide 38: Definition of Adverse Events

    Definition of Adverse Events Adverse Event: Serious Adverse Event: Any untoward medical occurrence in a patient Any untoward medical occurrence which: administered a pharmaceutical product or device and that - Results in death does not necessarily have a causal relationship between - Is life-threate

  39. Slide 39: Reporting of Adverse Events

    Reporting of Adverse Events Reporting Requirements of Physician to Sponsor The following is a list of events that the physician must report to the sponsor within 24 hours after learning of the event, irrespective of any relationship to study medicine: • SAEs • Pregnancies/ Breastfeeding • Product Co

  40. Slide 40: Reporting of AE/SAE

    Reporting of AE/SAE Adverse Event Reporting Period The study period during which all AEs must be reported begins from the date informed consent is obtained until 14 days after the patient discontinues SKYTROFA, withdraws consent, is lost to follow-up, or dies, whichever is earliest. 14 days after di

  41. Slide 41: Reporting of AE/SAE

    Reporting of AE/SAE Product Complaints and Falsified Medicinal Products • A Product Complaint is defined as any written or oral information received from a complainant that alleges deficiencies related to identity, quality, safety, strength, purity, reliability, durability, effectiveness, or perform

  42. Slide 42: SkybriGHt Only

    SkybriGHt Only Focus on • Clinical Outcome Assessments (COA) • Electronic Health Records (Claims Data) • Auto-injector read-out 42 CONFIDENTIAL

  43. Slide 43: SkybriGHt Special Consent

    SkybriGHt Special Consent Patients in SkybriGHt will be asked for permission to collect the following: • Electronic (e)COAs (QoLISSY Parent or Child, TSQM-9 Parent) and paper PPQ Parent or Child. • Auto-Injector data read-out • Claims Data (in the time from 3 years before enrollment until End of Stu

  44. Slide 44: Specific SkybriGHt Data Collection

    Specific SkybriGHt Data Collection Following Clinical Outcome Assessments (COAs) are collected 1. Quality of Life in Short Stature Youth (QoLISSY) Parent and Child 2. Treatment Satisfaction Questionnaire for Medicine (TSQM-9) Parent  These are electronic (e)COAs, collected on an iPad using Medidata

  45. Slide 45: Specific SkybriGHt Data Collection – Cont’d

    Specific SkybriGHt Data Collection – Cont’d Additional Data Collection 4. Claims Data/Electronic Medical Records are collected electronically via administrative claims outside of the treating physician and/or electronic medical record (EMR) database • Claims data will be linked to additional data so

  46. Slide 46: SkybriGHt’s COA Data Collection

    SkybriGHt’s COA Data Collection Following Clinical Outcome Assessments (COAs) are collected  QoLISSY Parent (P) or Child (C) Never change version: < 8 years – QoLISSY-P ≥ 8 years – QoLISSY-C At baseline and then every six (6) months electronic (e)COA -  TSQM-9 Parent until End of Study (discontinu

  47. Slide 47: SkybriGHt’s Unique Data Collection – Autoinjector

    SkybriGHt’s Unique Data Collection – Autoinjector (AI) Autoinjector (AI) data such as date, dose, frequency of use are stored in the device. • SkybriGHt patients are asked to participate in an AI exchange/return program for a read out of the data after using the AI for approximately 12 months. This

  48. Slide 48: SkybriGHt Auto-Injector (AI) Data Collection

    SkybriGHt Auto-Injector (AI) Data Collection To participate in the Auto-Injector exchange program, the AI Serial Number (SN) from each patient’s AI needs to be entered into an eCRF in Veeva EDC. • The SN consists of 14 digits and is located on the bottom of the Auto- Injector Options for how the SN

  49. Slide 49: SkybriGHt - GH Dose Unit Entry in EDC

    SkybriGHt - GH Dose Unit Entry in EDC • Preferred dose unit for daily Growth Hormone treatments (e.g. somatropin, Norditropin, Omnitropin, Nutropin, Humatropin): mg hGH/kg/week ( ) If the daily dose is known it can be calculated as Dose = ( ) x Number of days the dose is given in a week • Preferred

  50. Slide 50: Medidata electronic (e)COA/EDC

    Medidata electronic (e)COA/EDC 50 CONFIDENTIAL

  51. Slide 51: Medidata eCOA/EDC

    Medidata eCOA/EDC You must have a user account in iMedidata (https://www.imedidata.com/) • New users: account and Welcome email are provided as part of site activation • Existing iMedidata users: you should receive an email invitation for the protocol ASND0037 • If not received, please contact your

  52. Slide 52: Site Locates Patient in Rave EDC

    Site Locates Patient in Rave EDC Site user clicks Add Subject in the upper right-hand corner of the screen, enters the Subject ID, and clicks Save 52 CONFIDENTIAL

  53. Slide 53: Site Triggers eCOA Forms

    Site Triggers eCOA Forms Baseline folder  Complete the Demographics form Is the subject 8 years or older at the time of Enrollment  Is the subject less than 18 years old at the time of Enrollment?  YES  Enrollment Date  Save eCOA questionnaires are now available in visit folder and ready for co

  54. Slide 54: Site Triggers eCOA Forms

    Site Triggers eCOA Forms • Baseline folder  Complete the PPQ Form Used form* Please specify who is responding to the Patient Preference form  Save Response will determine which PPQ Form will become available *The PPQ form will be completed in EDC only (not available in eCOA) 54 CONFIDENTIAL

  55. Slide 55: Medidata eCOA/EDC

    Medidata eCOA/EDC The TSQM and QOLISSY should be completed on the tablet provided by Medidata. Please note that this access will be activated after the TSQM and QOLISSY patient facing screens have been submitted and approved by the IRB. Please discuss with your CRA when those will be available for t

  56. Slide 56: electronic (e)COA/EDC Patient Cloud app

    electronic (e)COA/EDC Patient Cloud app 56 CONFIDENTIAL

  57. Slide 57: eCOA/EDC Patient Cloud app

    eCOA/EDC Patient Cloud app The site will receive iOS tablet with the Patient Cloud App installed. Ensure the Patient Cloud app on site tablet is turned on and charged, ahead of the visit. The site tablet is used at the site visits to collect patient responses to the eCOA questionnaires. 57 CONFIDENT

  58. Slide 58: eCOA/EDC Patient Cloud app

    eCOA/EDC Patient Cloud app Select the Study ASND0037 SKYBRIGHT, Patient and Visit and follow the prompts on the tablet for patient to complete the eCOA questionnaires. Before the patient leaves the site, please confirm that the patient has completed the QOLISSY and TSQM, as applicable. 58 CONFIDENTI

  59. Slide 59: eCOA/EDC Patient Cloud app - Summary

    eCOA/EDC Patient Cloud app - Summary PI Signature At minimum, the PI’s signature is Create patient Select Patient ID and required at the end of study. the patient Query and Review for Completeness Queries will be posted on the EDC Monitor patient forms that the site enters in EDC. Patient Patient Pl

  60. Slide 60: eCOA/EDC Patient Cloud app - Support

    eCOA/EDC Patient Cloud app - Support 60 CONFIDENTIAL

  61. Slide 61: Clinical Study Tokenization

    Clinical Study Tokenization 61 CONFIDENTIAL

  62. Slide 62: What Is Tokenization?

    What Is Tokenization? • Tokenization is the process by which personally identifiable data elements are replaced by a unique string of characters, called a token • Designed to be irreversible • Maintains privacy • Authorized by Patient consent Confidential and Proprietary Ascendis Pharma Registry Tea

  63. Slide 63: Data Privacy

    Data Privacy How are HIPAA requirements being met during the tokenization process?  Patients must consent to their de-identified study data being created and used in current and future analysis via the study Informed Consent Form (ICF)  The study ICF language is reviewed and approved by the Instit

  64. Slide 64: What are we asking the Site Staff to do?

    What are we asking the Site Staff to do? • Access the tokenization web portal • Enter consented Patient’s information into the tokenization web portal • Patient has to check "Yes" in the SkybriGHt ICF section about "Agreement for Collection of Medical Information from other sources“ to consent to to

  65. Slide 65: Patient Data Form for Tokenization

    Patient Data Form for Tokenization IMPORTANT NOTE: Before tokenizing any Patient(s), please confirm Informed Consent Form (ICF) has been signed and obtained from the Patient. Site User must enter the following information to TOKENIZE a Patient: • Patient “First & Last Name” • Patient “Gender” • Pati

  66. Slide 66: Electronic Data Capture (EDC) Veeva Vault

    Electronic Data Capture (EDC) Veeva Vault

  67. Slide 67: Configuring Veeva Vault to avoid technical difficulties

    Configuring Veeva Vault to avoid technical difficulties: • Veeva Vault EDC works on following browsers • Chrome • Firefox • Edge • Safari • Allow all pop-ups in your web browser

  68. Slide 68: Electronic Data Capture – System Access

    Electronic Data Capture – SYSTEM ACCESS • Provide the names of individuals who will need access to your monitor and their role. • You will receive an invitation e-mail asking you to join the study • If you have not received an e-mail invitation, please follow up with your site monitor • Role-specifi

  69. Slide 69: Electronic Data Capture – Learning

    Electronic Data Capture – Learning • Accessible at learning.veeva.com • Username is the same as the email address that the 'Welcome to Veeva Learning' email was sent to PASSWORD When signing in for the first time, select 'forgot password' to set up your password. SUPPORT The CDMS help link can also

  70. Slide 70: Electronic Data Capture – Log in

    Electronic Data Capture – Log in https://login.veevavault.com • Username / Password FORGOT PASSWORD •To reset the password, go to learning.veeva.com •Click Forgot Password? •Write your email address in "username" field. CHANGE PASSWORD •From the user profile, click Change Password. •Enter your curre

  71. Slide 71: Electronic Data Capture – E-Learning

    Electronic Data Capture – E-Learning • To access all the EDC functionalities, you must complete training courses needed for your role • An email with login instructions will be sent to access the Veeva Learning, and then each time you’re enrolled in a new course or curriculum, you will receive a sep

  72. Slide 72: Electronic Data Capture – Navigating

    Electronic Data Capture – Navigating The task bar displays Ascendis Pharma Registry Teams to indicate upcoming tasks and how close you are to a due date. It categorizes tasks into the following: – Forms: Overdue forms and incomplete forms – Review: Forms that require signature – Queries: Open querie

  73. Slide 73: Electronic Data Capture – Navigating

    Electronic Data Capture – Navigating You can expand the task bar to view details about your tasks 73 CONFIDENTIAL

  74. Slide 74: Electronic Data Capture – Submitting and saving data

    Electronic Data Capture – Submitting and saving data • The audit trail is registered once the “Complete” button is “clicked” • Veeva EDC Vault retains data immediately as it is entered in the respective fields • Changes made to a form before it is saved will not be recorded in the audit trail • If a

  75. Slide 75: Electronic Data Capture – Queries

    Electronic Data Capture – Queries System Queries Vault creates a system query on any item value that is out of a pre-defined range. When a system query is manually closed, that same system query will not re-fire when forms are resubmitted. Manual Queries Manual queries are opened by the CRAs and Dat

  76. Slide 76: Electronic Data Capture – Query Resolution

    Electronic Data Capture – Query Resolution Navigate to the Event, Form, or Item on which there is an open query. Click Reply (for manual queries) or Comment (for system queries). - For Event Date queries, click Edit Date to enter a new date and select a change reason. o If the query was system-gener

  77. Slide 77: Electronic Data Capture – Signatures

    Electronic Data Capture – Signatures Electronic signatures are applied by PI and Sub-I users to confirm the statement “I verify that the data is accurate and complete”. Electronic signature is applied to the entire patient casebook by entering your login credentials (username and password). The Sign

  78. Slide 78: Electronic Data Capture – Adding patients

    Electronic Data Capture – Adding patients ADDING PATIENTS To add patients at a site, click “+New Casebook” button present at site-level 78 CONFIDENTIAL

  79. Slide 79: Electronic Data Capture – Adding blank data

    Electronic Data Capture – Adding blank data You can mark Forms and individual Items as Intentionally Left Blank to let the sponsor and CRA know that the data wasn’t collected and why. Forms Items To mark a Form as Intentionally Left Blank: To mark an Item as Intentionally Left Blank: • Select Intent

  80. Slide 80: Electronic Data Capture – Adding Adverse events

    Electronic Data Capture – Adding Adverse events The Adverse Event eCRF is used to collect information regarding untoward medical events occurring during the study as defined from the time when informed consent is obtained and study discontinuation/termination or withdrawal of consent, whichever is e

  81. Slide 81: Electronic Data Capture – Adding Pre-Dose Assessment

    Electronic Data Capture – Adding "Pre-Dose Assessment" The “Pre-Dose Assessment” Event/folder is intended to collect limited assessment data prior to the first exposure to lonapegsomatropin, and not just for assessments prior to the participation in the study. These CRFs are only displayed/expected

  82. Slide 82: Electronic Data Capture – Adding Observation Period

    Electronic Data Capture – Adding "Observation Period" The "Observation Period" folder is designed to correspond to the analysis design for both studies. The ASND0033 SkyPASS protocol states data entry for every 3 months. The ASND0037 SkybriGHt protocol states data entry for every 6 months. If a pati

  83. Slide 83: Electronic Data Capture – Informed Consent

    Electronic Data Capture – INFORMED CONSENT The Informed Consent eCRF is used to capture study consent. If Patient is not consenting to study, then please mark this page ‘Intentionally left blank’ - Was Informed Consent completed? - Select Yes or No - Date of Informed Consent - Enter the Date (DD/MMM

  84. Slide 84: Investigator’s Site File (ISF) overview

    Investigator’s Site File (ISF) overview 84 CONFIDENTIAL

  85. Slide 85: ISF – Table of Content

    ISF – Table of Content The Investigator’s Site File is divided into 17 sections. 1. Contact List 11. Patient facing materials incl. Informed 2. Patient Enrollment Consent/Patient Information 3. Site Monitoring Visit Forms 12. Non-Investigational Product (Non-IP) Documentation 4. Serious Adverse Even

  86. Slide 86: What’s next?

    What’s next? 1. Document Site Initiation Visit (SIV) completion. 2. Provide the signed SIV log to Ascendis Pharma Registry Team. 3. Provide the Site Source Data Agreement (SSDA), training log and Site Responsibility Log to Ascendis Pharma Registry Team. 4. Activate accounts in Medidata Rave and Veev

  87. Slide 87: Questions? Thank you!

    Questions? Thank you! 87 CONFIDENTIAL

Ascendis Pharma — Technical Presentation (October 2025) on 6ix