https://doi.org/10.55788/db9444f4
The phase 3 ENERGIZE trial (NCT04770753) randomised 194 participants with α-NTDT(n=62) or β-NTDT (n=132) 2:1 to 24 weeks of 100 mg twice daily mitapivat or placebo. Subsequently, all participants received mitapivat for up to 5 years. The primary endpoint was Hb response from week 12 to week 24 compared with the baseline. Prof. Ali Taher (American University of Beirut, Lebanon) presented the results [1].
“Mitapivat demonstrated a statistically significant improvement in Hb response versus placebo,” expressed Prof. Taher. In the mitapivat arm, 42.3% of participants had a Hb response, compared with 1.6% in the placebo arm (P<0.0001; see Figure). The average Hb response among responders was 1.56 g/dL. Importantly, the researchers reported an improvement in the mean FACIT-Fatigue Scale for participants on mitapivat compared with placebo controls (+4.85 vs +1.46; P=0.0026). “We also observed improvements in markers of haemolysis and erythropoietic activity,” added Prof. Taher.
Figure: Haemoglobin response per participant, mitapivat versus placebo [1]
Hb, haemoglobin; HbH disease, haemoglobin H disease.
Serious adverse events (AEs) were seen in 6.2% of the participants on mitapivat and none of the participants on placebo. In addition, 3.1% discontinued the study drug due to AEs. Headache (22.5%), insomnia (14.0%), nausea (11.6%), and upper respiratory tract infection (10.9%) were the most common ‘any grade’ AEs in the experimental arm.
Mitapivat met the primary and key secondary endpoints in the ENERGIZE trial, realising Hb and fatigue improvement in participants with α- or β-NTDT.
- Taher AT, et al. ENERGIZE: a global, phase 3 study of mitapivat demonstrating efficacy and safety in adults with alpha- or beta-non-transfusion-dependent thalassemia. S104, EHA congress 2024, 13–16 June, Madrid, Spain.
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