PYRUKYND®
Non-Transfusion Dependent Thalassaemia
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Product Information
Scientific name: Mitapivat
Brand name: PYRUKYND®
RESPONSIBLE: Agios Pharmaceuticals Inc -
Clinical Trial/Study Information
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 countryAnticipated completion date: Nov 2023
Scope of the Study / Aim: Improvement of haemoglobin levels
Transfusion Dependent Thalassaemia
-
Product Information
Scientific name: Mitapivat
Brand name: PYRUKYND®
RESPONSIBLE: Agios Pharmaceuticals Inc -
Clinical Trial/Study Information
Trial Name: ENERGIZE-T
Code: NCT04770779
Phase: 3
Eligible patient diagnosis: TDT (α- or β- thalassaemia)
No. of Patients enrolled: 258
Study Sites: 78 Sites per countryAnticipated completion date: July 2024
Scope of the Study / Aim: Decrease transfusion needs and by how much
Update: 19 December 2024
Based on data observed in both the Phase 3 ENERGIZE and ENERGIZE-T studies, Agios filed regulatory applications for mitapivat for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassaemia with the U.S., European Union, Kingdom of Saudi Arabia and United Arab Emirates health authorities.
The Phase 3 ENERGIZE and ENERGIZE-T trials enrolled a total of 452 patients reflective of the real-world thalassemia population. The results demonstrated that mitapivat improves hemolytic anaemia and quality-of-life related measures, as measured by significant reductions in transfusion burden and significant improvements in hemoglobin and fatigue.
New data
- Data presented at the 66th ASH Annual Congress (7 – 10 December 2024) in San Diego (USA) showed that:
- Ex vivo treatment with mitapivat in 25 non-transfusion dependent thalassaemia patients (β-thal/HbE disease) in Thailand corresponded to reduced haemolysis and intracellular reactive oxygen species in RBCs thus corroborating clinical trial findings.
Sources:
Agios Presents Positive Results from Phase 3 ENERGIZE-T Study of Mitapivat at ASH 2024 and Provides Regulatory Update on Mitapivat – Agios Pharmaceuticals, Inc.
Ex Vivo Treatment By Mitapivat, an Allosteric Pyruvate Kinase Activator, Reduced Hemolysis and Reactive Oxygen Species in Red Blood Cells of Non-Transfusion Dependent Hemolytic Anemic Patients Due to β-Thalassemia/Hb E Disease
Update: 30 September 2024
Submission to FDA for Supplemental New Drug Application (sNDA) anticipated by the end of 2024. Standard review time is 10 months, although Priority Review may reduce this to between 8 – 6 months.
Update: 30 June 2024
- Data presented at the 29th EHA Annual Congress (13 – 16 June 2024) in Madrid (Spain) showed that:
– Mitapivat increased Hb in 42.3% of NTDT (α- and β-thalassaemia) patients, improving fatigue and walking capacity. - FDA, EMA and GCC regulatory approval for TDT and NTDT will filed by the end of 2024.
Sources:
Agios Scientific Exchange Webinar, 21 June 2024
ENERGIZE: A Global Phase 3 Study of Mitapivat Demonstrating Efficacy and Safety in Adults with alpha- or beta-Non-Transfusion-Dependent Thalassaemia
Improvements in Fatigue and 6-minute Walk Test in Adults with alpha- or beta-Non-Transfusion-Dependent Thalassaemia: The Phase 3 ENERGIZE Trial of Mitapivat
Update: 31 March 2024
- The results of the Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia showed that the study achieved its primary endpoint of hemoglobin response. The results in detail:
- 194 patients were enrolled in the study (130 randomized to mitapivat 100 mg twice-daily (BID) and 64 randomized to matched placebo).
- Hemoglobin response was defined as an increase of ≥1 g/dL in average hemoglobin concentrations from Week 12 through Week 24 compared with baseline.
- Treatment with mitapivat demonstrated a statistically significant increase compared to placebo.
- 3% of patients in the mitapivat arm achieved a hemoglobin response, compared to 1.6% of patients in the placebo arm (2-sided p<0.0001).
- Overall, during the 24-week double-blind period, incidence of adverse events was similar across mitapivat and placebo arms. Four (3.1%) subjects in the mitapivat arm experienced adverse events (AEs) leading to discontinuation; there were no AEs leading to discontinuation in the placebo arm.
- FDA regulatory approval for TDT and NTDT will filed by the end of 2024.
Sources:
https://investor.agios.com/news-releases/news-release-details/agios-announces-phase-3-energize-study-mitapivat-met-primary
https://investor.agios.com/news-releases/news-release-details/agios-reports-fourth-quarter-and-full-year-2023-financial
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.
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.
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: 19 December 2024
Based on data observed in both the Phase 3 ENERGIZE and ENERGIZE-T studies, Agios filed regulatory applications for mitapivat for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia with the U.S., European Union, Kingdom of Saudi Arabia and United Arab Emirates health authorities.
The Phase 3 ENERGIZE and ENERGIZE-T trials enrolled a total of 452 patients reflective of the real-world thalassemia population. The results demonstrated that mitapivat improves hemolytic anemia and quality-of-life related measures, as measured by significant reductions in transfusion burden and significant improvements in hemoglobin and fatigue.
New data
- Data presented at the 66th ASH Annual Congress (7 – 10 December 2024) in San Diego (USA) showed that:
- Mitapivat significantly improved haemoglobin levels and fatigue vs placebo in the phase 3 ENERGIZE-T trial in adults with transfusion-dependent α- or β-thalassaemia.
- The primary endpoint was a transfusion reduction response defined by ≥ 50% reduction in transfused RBC units) was achieved by 30.4% of patients in the mitapivat arm.
- Mitapivat significantly reduced transfusion burden and demonstrated a durable response of up to 36 weeks.
Sources:
Agios Presents Positive Results from Phase 3 ENERGIZE-T Study of Mitapivat at ASH 2024 and Provides Regulatory Update on Mitapivat – Agios Pharmaceuticals, Inc.
ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia
Update: 30 September 2024
Submission to FDA for Supplemental New Drug Application (sNDA) anticipated by the end of 2024. Standard review time is 10 months, although Priority Review may reduce this to between 8 – 6 months.
Update: 30 June 2024
Top-line results for the Phase 3 ENERGIZE-T study showed:
- 258 patients were enrolled in the study, with 171 randomized to mitapivat 100 mg twice-daily (BID) and 87 randomized to matched placebo.
- Primary endpoint of transfusion reduction response (TRR, defined as a ≥50% reduction in transfused red blood cell (RBC) units was met.
- 4% of patients achieved a transfusion reduction response in any 12-week period
- Overall, during the 48-week double-blind period, incidence of adverse events (AEs) was similar across mitapivat and placebo arms. In the mitapivat arm, 5.8% of the patients experienced an AE leading to discontinuation, compared to 1.2% of patients in the placebo arm.
FDA, EMA and GCC regulatory approval for TDT and NTDT will filed by the end of 2024.
Sources:
Agios Scientific Exchange Webinar, 21 June 2024
TIF/Agios Webinar on Topline ENERGIZE-T Results, 10 June 2024
Agios Announces Phase 3 ENERGIZE-T Study of Mitapivat Met Primary Endpoint and All Key Secondary Endpoints in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia – Agios Pharmaceuticals, Inc.
Update: 31 March 2024
Top-line data from Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia are expected to be announced in mid-2024.
FDA regulatory approval for TDT and NTDT will filed by the end of 2024.
Sources:
https://investor.agios.com/news-releases/news-release-details/agios-announces-phase-3-energize-study-mitapivat-met-primary
https://investor.agios.com/news-releases/news-release-details/agios-reports-fourth-quarter-and-full-year-2023-financial
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.
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.
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
