Ascendis Pharma A/s · NASDAQ:ASND
Ascendis Pharma A/s — Technical Presentation (May 2025)
84 slides from Ascendis Pharma A/s's technical presentation, May 28, 2025
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
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
Slide 3: Key Contacts
Key Contacts ICON (CRO) Ascendis Pharma (Sponsor) ◦ Site Management Associate (SMA): Andrea Sorka ◦ Project Leads: Aneta Ziolkowska & Kirsten Hoyer - Andrea.Sorka@iconplc.com - AZA@ascendispharma.com - KIR@ascendispharma.com ◦ Clinical Research Associate (CRA) : María Teresa Mata ◦ US Medical Affair
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
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
Slide 6: 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
Slide 7: SKYTROFA US Label Overview
SKYTROFA® (lonapegsomatropin-tcgd) US Label Overview 7 CONFIDENTIAL
Slide 8: 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
Slide 9: 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
Slide 10: 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
Slide 11: 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
Slide 12: 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
Slide 13: SkyPASS & SkybriGHt Project Design and Protocol Review
SkyPASS & SkybriGHt Project Design and Protocol Review
Slide 14: 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
Slide 15: Study Timelines
Study Timelines ESTIMATED TIMELINES SkybriGHt SkyPASS LAST PATIENT IN March, 2028 September, 2028 END OF DATA COLLECTION* January, 2033 September, 2033 15 CONFIDENTIAL * Estimated dates
Slide 16: 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
Slide 17: 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
Slide 18: 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
Slide 19: 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.
Slide 20: 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
Slide 21: 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
Slide 22: 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
Slide 23: 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
Slide 24: 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
Slide 25: 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
Slide 26: Study Responsibilities
STUDY RESPONSIBILITIES 26 CONFIDENTIAL
Slide 27: Study Responsibilities
Study Responsibilities • Sponsor • CRO • IRBs/IECs • Investigator/Staff • CRAs/SMAs Have a shared responsibility to ensure proper conduct of the program. 27 CONFIDENTIAL
Slide 28: 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
Slide 29: 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
Slide 30: 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
Slide 31: 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 ICON with updated Financial Disc
Slide 32: 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
Slide 33: 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
Slide 34: Adverse Event Reporting
ADVERSE EVENT REPORTING 34 CONFIDENTIAL
Slide 35: 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
Slide 36: 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
Slide 37: 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
Slide 38: 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
Slide 39: SkybriGHt Only
SkybriGHt Only Focus on • Clinical Outcome Assessments (COA) • Electronic Health Records (Claims Data) • Auto-injector read-out 39 CONFIDENTIAL
Slide 40: 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
Slide 41: 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
Slide 42: 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
Slide 43: 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
Slide 44: SkybriGHt’s Unique Data Collection – Autoinjector (AI)
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
Slide 45: 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
Slide 46: Medidata electronic (e)COA/EDC
Medidata electronic (e)COA/EDC 46 CONFIDENTIAL
Slide 47: 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
Slide 48: 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 48 CONFIDENTIAL
Slide 49: 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
Slide 50: 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) 50 CONFIDENTIAL
Slide 51: 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
Slide 52: electronic (e)COA/EDC Patient Cloud app
electronic (e)COA/EDC Patient Cloud app 52 CONFIDENTIAL
Slide 53: 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. 53 CONFIDENT
Slide 54: 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. 54 CONFIDENTI
Slide 55: 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
Slide 56: eCOA/EDC Patient Cloud app - Support
eCOA/EDC Patient Cloud app - Support 56 CONFIDENTIAL
Slide 57: Clinical Study Tokenization
Clinical Study Tokenization 57 CONFIDENTIAL
Slide 58: 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 ICON Information 58 CONFIDEN
Slide 59: 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
Slide 60: 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
Slide 61: 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
Slide 62: Electronic Data Capture (EDC) Veeva Vault
Electronic Data Capture (EDC) Veeva Vault
Slide 63: 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
Slide 64: 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
Slide 65: 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
Slide 66: 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
Slide 67: 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
Slide 68: Electronic Data Capture – Navigating
Electronic Data Capture – Navigating The task bar displays icons 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 queries that are ready for you
Slide 69: Electronic Data Capture – Navigating
Electronic Data Capture – Navigating You can expand the task bar to view details about your tasks 69 CONFIDENTIAL
Slide 70: 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
Slide 71: 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
Slide 72: 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
Slide 73: 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
Slide 74: 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 74 CONFIDENTIAL
Slide 75: 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
Slide 76: 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
Slide 77: 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
Slide 78: 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
Slide 79: 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
Slide 80: Investigator’s Site File (ISF) overview
Investigator’s Site File (ISF) overview 80 CONFIDENTIAL
Slide 81: 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
Slide 82: What’s next?
What’s next? 1. Document Site Initiation Visit (SIV) completion. 2. Provide the signed SIV log to ICON. 3. Provide the Site Source Data Agreement (SSDA), training log and Site Responsibility Log to ICON. 4. Activate accounts in Medidata Rave and Veeva CDMS EDC ◦ Provide site’s user details to ICON f
Slide 83: Questions? Thank you!
Questions? Thank you! 83 CONFIDENTIAL
Slide 84: Schedule for Clinical Outcome Assessments
Schedule for Clinical Outcome Assessments Clinic Visit COA Dataset Name Completed by Baseline every EOS Note 6-months Quality of Life in Short Stature Youth - Measures the impact of short stature on children, from the perspective of the child. Patient X X X Child (QoLISSY) Applicable only for patien