Trial results for Etavopivat in a Phase 2 study (NCT05568225) for the treatment of anemia in patients with myelodysplastic syndromes (MDS) were posted on ClinicalTrials.gov on 2025-10-22. The study, which was terminated, reported a 50% hematologic improvement-erythroid (HI-E) response rate in the low transfusion burden patient group.
Background
The study evaluated Etavopivat for the treatment of anemia in adult patients with very low risk, low risk, or intermediate risk MDS.
Trial design
The study (NCT05568225) was a Phase 2 trial with an enrollment of 17 participants. It investigated Etavopivat in adult patients diagnosed with very low risk, low risk, or intermediate risk MDS per IPSS-R. The trial's overall status is TERMINATED.
Key results
The trial reported results for hematologic improvement-erythroid (HI-E) response and adverse events.
For the outcome of "Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for >=8 Weeks Within 24 Weeks of Etavopivat Treatment":
- In the non-transfusion dependent group, the HI-E response was 0%.
- In the low transfusion burden group, the HI-E response was 50%.
- In the high transfusion burden group, the HI-E response was 25%.
Regarding "Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat":
- Total adverse events were reported as 33 events for the non-transfusion dependent group, 33 events for the low transfusion burden group, and 54 events for the high transfusion burden group.
- Serious adverse events (SAEs) occurred in 1 event for the non-transfusion dependent group, 0 events for the low transfusion burden group, and 6 events for the high transfusion burden group.
- Adverse events related to Etavopivat were reported as 0 events for the non-transfusion dependent group, 0 events for the low transfusion burden group, and 1 event for the high transfusion burden group.
What this means
The results from this Phase 2 trial indicate varied responses to Etavopivat across different transfusion burden groups in MDS patients. While the low transfusion burden group showed a 50% HI-E response, the non-transfusion dependent group showed no response. The trial's termination suggests that the overall findings or other factors led to its early conclusion. The safety data provides an overview of reported adverse events, with a higher number of total AEs and SAEs observed in the high transfusion burden group.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT05568225, titled "A Study of Etavopivat for the Treatment of Anemia in Patients With Myelodysplastic Syndromes (MDS)," were posted on 2025-10-22 on clinicaltrials.gov.