Clinical trial updates

PYRUKYND®

Non-Transfusion Dependent Thalassaemia

  • Product Information

    Product Information

    Scientific name: Mitapivat
    Brand name: PYRUKYND®
    RESPONSIBLE: Agios Pharmaceuticals Inc

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Last update: 22/6/2023
    Trial Name: ENERGIZE
    Code: NCT04770753
    Phase: 3
    Eligible patient diagnosis: NTDT (α- or β- thalassaemia)
    No. of Patients enrolled: 194
    Study Sites: 73 Sites per country

    Anticipated completion date: Nov 2023
    Scope of the Study / Aim: Improvement of haemoglobin levels

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

    Additional notable points:

    • EMA: N/A
    • FDA: Orphan Drug designation (2020)
    • MHRA: N/A

Transfusion Dependent Thalassaemia

  • Product Information

    Product Information

    Scientific name: Mitapivat
    Brand name: PYRUKYND®
    RESPONSIBLE: Agios Pharmaceuticals Inc

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Last update: 22/6/2023
    Trial Name: ENERGIZE-T
    Code: NCT04770779
    Phase: 3
    Eligible patient diagnosis: TDT (α- or β- thalassaemia)
    No. of Patients enrolled: 258
    Study Sites: 77 Sites per country

    Anticipated completion date: July 2024
    Scope of the Study / Aim: Decrease transfusion needs and by how much

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

    Additional notable points:

    • EMA: N/A
    • FDA: Orphan Drug designation (2020)
    • MHRA: N/A

Update: 20 December 2023

  • Topline data from the two Phase 3 studies of mitapivat in non-transfusion-dependent and transfusion-dependent thalassemia will be announced in the first and second halves of 2024, respectively.

Source:
https://investor.agios.com/news-releases/news-release-details/agios-reports-business-highlights-and-third-quarter-2023


Update: 30 September 2023

  • Completed enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® in not regularly transfused and regularly transfused adults with thalassaemia, respectively.

Source:
https://investor.agios.com/news-releases/news-release-details/agiosreports-business-highlights-and-second-quarter-2023


Update: 30 June 2023

  • Screening of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND ® in not regularly
    transfused and regularly transfused adults with thalassemia, respectively has been completed.

Source:
Agios Reports Business Highlights and First Quarter 2023 Financial Results – Agios Pharmaceuticals, Inc.


Update: 31 March 2023

  • Enrolled more than half of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND ® in not regularly transfused and regularly transfused adults with thalassemia, respectively.
  • A clinical trial site for ENERGIZE and ENERGIZE-T (phase 3 clinical trials) for the 3 study of the safety and efficacy of PYRUKYND ® in non-transfusion dependent and transfusion-dependent thalassaemia patients respectively, have been initiated in Abu Dhabi.

The company aims to complete enrollment by mid-year.
Sources:
https://investor.agios.com/news-releases/news-release-details/agios-reports-fourth-quarter-and-full-year-2022-financial

https://www.zawya.com/en/press-release/companies-news/ground-breaking-thalassemia-clinical-trials-to-begin-in-abu-dhabi-a0p9a3kg

Update: 20 December 2023

  • Topline data from the two Phase 3 studies of mitapivat in non-transfusion-dependent and transfusion-dependent thalassemia will be announced in the first and second halves of 2024, respectively.

Source:
https://investor.agios.com/news-releases/news-release-details/agios-reports-business-highlights-and-second-quarter-2023


Update: 30 September 2023

• Completed enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® in not regularly transfused and regularly transfused adults with thalassaemia, respectively.

Source:
https://investor.agios.com/news-releases/news-release-details/agiosreports-business-highlights-and-second-quarter-2023


Update: 30 June 2023

  • Screening of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND ® in not regularly
    transfused and regularly transfused adults with thalassemia, respectively has been completed.

Source:
Agios Reports Business Highlights and First Quarter 2023 Financial Results – Agios Pharmaceuticals, Inc.

Update: 31 March 2023

  • Enrolled more than half of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of
    PYRUKYND ® in not regularly transfused and regularly transfused adults with thalassemia,
    respectively.
  • A clinical trial site for ENERGIZE and ENERGIZE-T (phase 3 clinical trials) for the 4 study of the safety and efficacy of PYRUKYND ® in non-transfusion dependent and transfusion-dependent thalassemia patients respectively, have been initiated in Abu Dhabi.
  • The company aims to complete enrolment by mid-year.

Sources:
https://investor.agios.com/news-releases/news-release-details/agios-reports-fourth-quarter-and-full-year-2022-financial

https://www.zawya.com/en/press-release/companies-news/ground-breaking-thalassemia-clinical-trials-to-begin-in-abu-dhabi-a0p9a3kg

Back to top button