Ipsen Sa · EURONEXT:IPN
Ipsen Sa — Technical Presentation (November 2023)
20 slides from Ipsen Sa's technical presentation, November 2023
Slide 1: Efficacy and safety of elafibranor in primary biliary cholangitis: ELATIVE phase 3 trial
Efficacy and safety of elafibranor in primary biliary cholangitis: Results from the ELATIVE ™ double-blind, randomized, placebo-controlled phase 3 trial Plain language version Christopher L. Bowlus,1 Kris V. Kowdley,2,3 Cynthia Levy,4 Ulus Akarca,5 Mario Reis Alvares-da-Silva,6 Pietro Andreone,7 Mar
Slide 2: Disclosures of the presenting author
Disclosures of the presenting author Christopher L. Bowlus Received grants from Boston Scientific, Bristol Myers Squibb, Calliditas, Cara Therapeutics, ChemoMab, COUR Pharmaceuticals, CymaBay, Gilead, GlaxoSmithKline, Hanmi, and Ipsen Received consulting fees from CymaBay, GlaxoSmithKline, Invea, Ip
Slide 3: Background
Background • Primary biliary cholangitis (PBC) is a liver disease that mostly affects women over 40 years old1–3 • People with PBC often have symptoms of itching and tiredness1 Currently available medications:2 1st treatment option: 2nd treatment option: ursodeoxycholic acid (UDCA) obeticholic acid
Slide 4: ELATIVE phase 3 trial objectives and design
ELATIVE™ phase 3 trial objectives and design ELATIVE™ aimed to: • Find out how well elafibranor works in treating PBC in people who did not get better with current treatment (UDCA) • Find out what the side effects of elafibranor might be Core trial Recruitment Optional extension People randomly spli
Slide 5: Study outcomes
Study outcomes • Alkaline phosphatase (ALP) and bilirubin are two substances found in the blood Liver health • These help people understand how bad a person’s liver disease might be • Higher amounts of these substances in the blood indicate worse disease • People with PBC often have itchy skin • We
Slide 6: Age, background, and health conditions were recorded for people taking part in the trial
Age, background, and health conditions were recorded for people taking part in the trial 108 people took elafibranor 53 people took placebo Average time since Average age Sex Race PBC diagnosis 58 56 102 (94%) 52 (98%) 101 (94%) 46 (87%) 8 8 years years females females White White years years People
Slide 7: The majority of people taking elafibranor had improved liver health after 1 year
The majority of people taking elafibranor had improved liver health after 1 year Percentage of people with improved liver health 70 • ALP and bilirubin are substances in the 60 blood used to measure liver health 51% Percentage of people 50 • Higher amounts indicate worse disease 40 • More people tak
Slide 8: Only people taking elafibranor reached completely normal levels of ALP after 1 year
Only people taking elafibranor reached completely normal levels of ALP after 1 year Percentage of people with completely normal levels of ALP 20 • Researchers also looked to see if ALP 15% reached completely normal levels Percentage of people 15 during the trial • Normal levels of ALP are what we 10
Slide 9: Elafibranor greatly reduced levels of ALP from as early as 4 weeks
Elafibranor greatly reduced levels of ALP – this effect was seen as early as 4 weeks of treatment, and lasted throughout the trial Percentage change in levels of ALP over time change from start of trial 0% 2% Average percentage • Researchers measured ALP 0 regularly throughout the trial −10 ALP in p
Slide 10: How researchers measured itch in ELATIVE
How researchers measured itch in ELATIVE PBC Simple 1-question form that measures the intensity of itch • 11-point scale ranging from 0 (no itch) to 10 (worst itch imaginable) Worst-Itch • Patients reported this information every day NRS1 • Patients were asked to rate this score for the last 24 hour
Slide 11: Differences in the PBC Worst Itch NRS questionnaire were not large enough to conclude itch was improved
Differences in the PBC Worst Itch NRS questionnaire were not large enough to conclude that itch was improved Change in questionnaire score Elafibranor 80 mg Placebo (PBC Worst Itch NRS) (N=44) (N=22) 0 Average change in questionnaire • Researchers measured how bad people’s itch was using a questionn
Slide 12: The PBC-40 questionnaire suggested elafibranor may improve itch after 1 year
The PBC-40 questionnaire suggested that elafibranor may improve symptoms of itch after 1 year Change in questionnaire score Elafibranor 80 mg Placebo (N=42) (PBC-40 Itch) (N=16) 2 Average change in questionnaire 1 • Two other questionnaires (PBC-40 and score from start of trial 5-D Itch) were also u
Slide 13: The 5-D Itch questionnaire also suggested elafibranor may improve itch
The 5-D Itch questionnaire also suggested that elafibranor may improve symptoms of itch after 1 year Change in questionnaire Elafibranor 80 mg score Placebo (N=42) (5-D Itch) (N=16) 2 Average change in questionnaire 0 score from start of trial • In people with relentless itch, the 5-D Less itch -2 I
Slide 14: The most common side effects of elafibranor in the trial were gut-related
The most common side effects of elafibranor in the trial were gut-related 108 people took 53 people took Side effects elafibranor placebo • This table shows the number of People with any side effect 104 (96.3%) 48 (90.6%) people who had certain side effects Stomach paina 12 (11.1%) 3 (5.7%) Diarrhea
Slide 15: Summary
Summary Elafibranor treatment resulted in improvement in liver health Elafibranor may improve symptoms of itchy skin, according to the PBC-40 Itch and 5-D Itch questionnaires The most common side effects of elafibranor were stomach pain, diarrhea, nausea, and vomiting If approved for use, elafibrano
Slide 16: Acknowledgments
Acknowledgments The authors thank all patients involved in the study, as well as their caregivers, care team, investigators, and research staff in participating institutions Plain language version Slides are the property of the presenting author and AASLD. Permission is required from both the presen
Slide 17: Acknowledgments
Acknowledgments MEDICAL WRITING SUPPORT • The authors thank Patrick Cox, BSc (Hons), and Oliver Palmer, BSc (Hons), of Costello Medical, London, UK for providing medical writing support, which was sponsored by Ipsen in accordance with Good Publication Practice guidelines AUTHOR CONTRIBUTIONS • All a
Slide 18: ELATIVE Principal Investigators
ELATIVE™ Principal Investigators USA UK Belgium Ana Corregidor, Victor Ankoma-Sey, Walid Ayoub, Christopher Bowlus, Kuldeep Cheent, Lindsey Corless, Sven Francque, Christophe Moreno, Stephen Caldwell, Hany Elbeshbeshy, Lisa Forman, Michael Galambos, Michael Heneghan, David Jones, Roger McCorry, Xavi
Slide 19: Full NEJM publication now available
Full NEJM publication now available A plain language summary of the full paper is available in its supplementary appendix Full paper at: Plain language version Slides are the property of the presenting author and AASLD. Permission is required from both the presenting author and AASLD for reuse. #484
Slide 20: Thank you!
Thank you! Full paper