PYRUKYND®
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Product Information
Scientific name: Mitapivat
Brand name: PYRUKYND®
RESPONSIBLE: Agios Pharmaceuticals Inc. -
Clinical Trial/Study Information
Last update: 22/6/2023
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 countryΑnticipated 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)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 [anticipated] [79 in phase 2 ; 198 – anticipated – in phase 3] (Last update: 16/4/2024)
Study Sites: 69 Sites per countryAnticipated completion date: February 2030
Scope of the Study / Aim: Determine the recommended dose of mitapivat, evaluate efficacy & safety; decrease annual VOCs
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Regulatory Information
Status: Not Authorised
Additional notable points:
- EMA: N/A
- FDA: Orphan drug designation (2020)
- MHRA: N/A
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.
Update: 20 December 2023
- Dosed first patient 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.
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.