TMS Icon
Clinical Trial Updates (SCD)

PYRUKYND®

Less than a minute
  • Product Information

    Product Information

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

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Extension of Phase 1 Pilot Study

    Trial Name: Extension of Phase 1 Pilot Study in SCD*
    Code: NCT04610866
    Phase: 2/3
    Eligible patient diagnosis: SCD (adults) (age 18 – 70)
    No. of Patients enrolled: 15 (Last update: 3/5/2023)
    Study Sites: 1 Sites per country

    Αnticipated completion date: February 2028
    Scope of the Study / Aim: Long-term effects of SCD in the frequency of VOCs
    *in collaboration with National Heart, Lung & Blood Institute (NHLBI)

    AG-946

    Trial Name: AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease
    Code:     NCT04536792
    Phase:     1
    Eligible patient diagnosis:     Healthy Volunteers, SCD (adults) (age 18 – 70)
    No. of Patients enrolled:     122 (Last update: 11/12/2024)
    Study Sites: 10 Sites per country

    Completion date: N/A
    Scope of the Study / Aim: Safety & Tolerability of AG-946 (tebapivat) in healthy volunteers and SCD adults

    RISE UP

    Trial Name: RISE UP
    Code: NCT05031780
    Phase: 2/3
    Eligible patient diagnosis: SCD (adults) (age 18 and above)
    No. of Patients enrolled: 267 (Last update: 11/12/2024)
    Study Sites: 69 Sites per country

    Anticipated completion date: February 2030
    Scope of the Study / Aim: Determine the recommended dose of mitapivat, evaluate efficacy & safety; decrease annual VOCs

    NCT06286046

    Trial Name: –
    Code: NCT06286046
    Phase:     2
    Eligible patient diagnosis: SCD (adults)
    No. of Patients enrolled: 40 [anticipated] (Last update: 17/06/2024)
    Study Sites: 35 across 8 countries [anticipated] (Last update: 17/06/2024)
    Anticipated completion date: September 2027
    Scope of the Study / Aim: Evaluate the effect of mitapivat on albumin creatinine ratio (ACR) response in participants with sickle cell disease (SCD) and nephropathy
  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

     

    Additional notable points:

    • EMA: Orphan drug designation (2024)
    • FDA: Orphan drug designation (2020)
    • MHRA: N/A

Update: 19 December 2024

  • Completed enrollment of Phase 3 RISE UP study of mitapivat in sickle cell disease. Topline results to be shared in late 2025.
  • European Commission designated mitapivat for the treatment of sickle cell disease as an orphan medicinal product.

New data:

  • Data presented at the 66th ASH Annual Congress (7 – 10 December 2024) in San Diego (USA) showed that tebapivat (mitapivat) was well tolerated in 16 patients with SCD receiving either 2mg or 5mg once daily for 28 days in the phase 1 study in adult pts with SCD (NCT04536792).

Sources: https://investor.agios.com/news-releases/news-release-details/agios-completes-enrollment-phase-3-rise-study-mitapivat-sickle
https://investor.agios.com/news-releases/news-release-details/agios-reports-business-highlights-and-third-quarter-2024
Results from a Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Tebapivat (AG-946) in Patients with Sickle Cell Disease

 

Update: 30 September 2024

No update available.

 

Update: 30 June 2024

At the 29th EHA Annual Congress (13 – 16 June 2024) in Madrid (Spain):

    • The design of a phase 2 study to evaluate the effect of mitapivat in patients with SCD and nephropathy was presented. The primary objective is to achieve a ≥30% decrease of ACR from baseline within 6 months. Enrolment is expected to open in Q3 2024.
    • The design of the phase 3 portion of the RISE UP trial was reported, based on the results of the phase 2 dose-finding study that showed clinically meaningful improvements in haemoglobin response.
    • Mitapivat 100mg twice daily was selected as the dose for the phase 3 portion of the trial.
    • The primary objective will be a Hb response, defined as ≥1g/dL increase in weeks 24 – 52 compared to baseline. Enrolment is ongoing, with initiation expected in October 2023.

Sources: The Launch Of A Global, Phase 2, Open-Label, Multicenter, Single-Arm Study Of Mitapivat In Patients With Sickle Cell Disease And Nephropathy
Study Design Of The Phase 3 Portion Of Rise Up: A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study Of Mitapivat In Patients With Sickle Cell Disease

 

Update: 31 March 2024

The company aims to complete enrolment for the Phase 3 portion of the RISE UP pivotal study of mitapivat in 2024.

Source: https://investor.agios.com/news-releases/news-release-details/agios-reports-fourth-quarter-and-full-year-2023-financial

 

Update: 20 December 2023

  • First patient dosed in the Phase 3 portion of the RISE UP pivotal study of mitapivat.

New data

  • Data presented at the 65th ASH Annual Congress (9 – 12 December 2023) in San Diego (USA) showed that:
  • A total of 79 patients were enrolled in the Phase 2 portion of the RISE UP study, with 26 patients in the 50 mg BID mitapivat arm, 26 patients in the 100 mg BID mitapivat arm, and 27 patients in the placebo arm.
  • 2% of patients (n=12) in the 50 mg BID mitapivat arm and 50.0% of patients (n=13) in the 100 mg BID mitapivat arm achieved a hemoglobin response, defined as an increase of ≥1 g/dL in average hemoglobin concentrations from Week 10 through Week 12 compared with baseline.
  • Of the 79 patients enrolled in the study, 73 continued into the Phase 2 open-label extension period.

Sources: https://investor.agios.com/news-releases/news-release-details/agios-reports-business-highlights-and-third-quarter-2023
https://investor.agios.com/news-releases/news-release-details/agios-presents-positive-results-phase-2-portion-rise-pivotal
https://ash.confex.com/ash/2023/webprogram/Paper187033.html

 

Update: 30 September 2023

No update available.


Update: 30 June 2023

Results from the RISE UP (phase 2) trial showed:

• Treatment with mitapivat demonstrated a statistically significant increase in hemoglobin response rate compared to placebo. Hemoglobin response was defined as an increase of ≥1 g/dL in average hemoglobin concentrations from Week 10 through Week 12 compared with baseline.
• 46.2% of patients (n=12) in the 50 mg twice daily (BID) mitapivat arm and 50.0% of patients (n=13) in the 100 mg BID mitapivat arm achieved a hemoglobin response.
• There were no adverse events (AEs) leading to discontinuation.

These results support proceeding with the Phase 3 part of the study. This is expected to enrol 198 patients, beginning in Oct-Dec 2023, reporting data in 2025 and receiving FDA approval in 2026.

Source: https://investor.agios.com/news-releases/news-release-details/agiosannounces-positive-results-phase-2-portion-rise-pivotal 


Update: 31 March 2023

• Completed enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND® in adults with sickle cell disease.
• Anticipate to announce data and decision to move forward with Phase 3 by mid-year.

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

Less than a minute
Back to top button