Trial Outcomes & Findings for A Study of Etavopivat for the Treatment of Anemia in Patients With Myelodysplastic Syndromes (MDS) (NCT NCT05568225)

Last Updated: 2025-10-22

Results Overview

This outcome measure reported on the combined incidence of NTD, LTB and HTB in terms of (HI-E) response for \>=8 weeks duration in participants with myelodysplastic syndromes (MDS) within 24 weeks. The participants were allocated to the following arms which were defined as: 1) NTD: \>=1.5 grams per deciliter (g/dL) increase in haemoglobin (Hb) from baseline maintained \>=8 consecutive weeks and no transfusion of RBC units for anemia over a continuous 8-week treatment period; 2) LTB: absence of any transfusion for \>=8 consecutive weeks; and 3) HTB: reduction by \>=50 percent (%) of RBC units for \>=8 consecutive weeks.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

From Baseline to Week 24

Results posted on

2025-10-22

Participant Flow

The trial was conducted at 10 sites in 4 countries.

The study was planned for 58 weeks, including 6 weeks for screening, 24 weeks for treatment, 24 weeks for extension, and 4 weeks for follow-up. It was terminated early after meeting a predefined quality tolerance limit (QTL). Of the 45 participants targeted, only 24 were screened, and 17 were enrolled into three groups: Non-transfusion dependent (NTD) N=3, Low transfusion burden (LTB) N=5, and High transfusion burden (HTB) N=9. Each participant received 400 mg of etavopivat daily.

Participant milestones

Participant milestones
Measure	Non-transfusion Dependent Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Low Transfusion Burden Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
Primary Treatment Period STARTED	3	5	9
Primary Treatment Period Efficacy Evaluable Set (EES)	1	2	4
Primary Treatment Period Safety Population	3	5	9
Primary Treatment Period Full Analysis Set (FAS)	3	5	9
Primary Treatment Period Pharmacokinetic (PK) Set	3	5	9
Primary Treatment Period COMPLETED	1	2	4
Primary Treatment Period NOT COMPLETED	2	3	5
Extension Treatment Period STARTED	1	2	4
Extension Treatment Period COMPLETED	1	2	1
Extension Treatment Period NOT COMPLETED	0	0	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Non-transfusion Dependent Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Low Transfusion Burden Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
Primary Treatment Period Termination of the study by the Sponsor	0	0	1
Primary Treatment Period Physician Decision	0	1	0
Primary Treatment Period Disease progression	0	1	0
Primary Treatment Period Lack of Efficacy	0	1	0
Primary Treatment Period Withdrawal of conset	1	0	4
Primary Treatment Period Adverse Event	1	0	0
Extension Treatment Period Termination of the study by the Sponsor	0	0	1
Extension Treatment Period Protocol Violation	0	0	1
Extension Treatment Period Lack of Efficacy	0	0	1

Baseline Characteristics

A Study of Etavopivat for the Treatment of Anemia in Patients With Myelodysplastic Syndromes (MDS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Non-transfusion Dependent n=3 Participants Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Low Transfusion Burden n=5 Participants Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden n=9 Participants High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Total n=17 Participants Total of all reporting groups
Age, Continuous	77.3 Years STANDARD_DEVIATION 5.69 • n=5 Participants	80.2 Years STANDARD_DEVIATION 3.49 • n=7 Participants	71.2 Years STANDARD_DEVIATION 8.11 • n=5 Participants	74.9 Years STANDARD_DEVIATION 7.58 • n=4 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants	4 Participants n=4 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants	13 Participants n=4 Participants
Race/Ethnicity, Customized Race: White	3 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants	14 Participants n=4 Participants
Race/Ethnicity, Customized Race: Not reported	0 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	3 Participants n=4 Participants
Race/Ethnicity, Customized Ethnicity: Not Hispanic or Latino	3 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants	13 Participants n=4 Participants
Race/Ethnicity, Customized Ethnicity: Hispanic or Latino	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants
Race/Ethnicity, Customized Ethnicity: Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants

PRIMARY outcome

Timeframe: From Baseline to Week 24

Population: EES: All participants in the FAS who have completed the week 24 response visit and who have a baseline record of the primary endpoint.

Outcome measures

Outcome measures
Measure	Non-transfusion Dependent n=1 Participants Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Low Transfusion Burden n=2 Participants Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden n=4 Participants High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
Percentage of Participants With Hematologic Improvement- Erythroid (HI-E) Response for >=8 Weeks Within 24 Weeks of Etavopivat Treatment	0 Percentage of participants	50 Percentage of participants	25 Percentage of participants

SECONDARY outcome

Timeframe: 48 weeks

Population: FAS: All participants who signed the informed consent and received at least 1 dose of etavopivat. Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in statistical analysis plan (SAP).

This outcome measure focused on the combined incidence of NTD, LTB and HTB participants, evaluating HI-E lasting =\>8 weeks in individuals with MDS within 16 weeks and 48 weeks. Participant's response were defined as follows: 1) NTD: an increase of =\>1.5 g/dL in Hb maintained for =\>8 consecutive weeks without any RBC transfusions for anemia; 2) LTB: no transfusions over =\>8 consecutive weeks; and 3) HTB: a reduction of =\>50% in RBC units for =\>8 consecutive weeks.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 48 weeks

This outcome measure focused on the combined incidence of NDT, LTB and HTB participants, evaluating HI-E response lasting =\>16 weeks in individuals with MDS within 24 weeks and 48 weeks. Participant's response were defined as follows: 1) NTD: an increase of =\>1.5 g/dL in Hb maintained for =\>16 consecutive weeks without any RBC transfusions for anemia; 2) LTB: no transfusions over =\>16 consecutive weeks; and 3) HTB: a reduction of =\>50% in RBC units for =\>16 consecutive weeks.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From baseline up to 48 weeks

Population: Safety population includes all participants who received at least one dose of etavopivat (including partial dosing).

This outcome measures the total number of AEs and SAEs in participants, including AEs related to etavopivat. AEs are any unfavorable medical occurrences in participants taking the medicinal product, regardless of causality. They include new or worsening symptoms, abnormal lab findings, and exacerbations of existing conditions, documented from the first dose through 28 days after the last dose. SAEs are severe AEs that result in death, are life-threatening, require hospitalization, lead to significant disability, or involve congenital anomalies. Important medical events posing risks to the participants are also classified as SAEs. Treatment Emergent Adverse Events (TEAEs) are AEs occurring after treatment initiation, aiding in the safety assessment of etavopivat. TEAEs will be considered drug-related if assessed by the Investigator as possibly related or related, or if relationship is missing.

Outcome measures

Outcome measures
Measure	Non-transfusion Dependent n=3 Participants Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Low Transfusion Burden n=5 Participants Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden n=9 Participants High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat AE	33 Events	33 Events	54 Events
Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat SAE	1 Events	0 Events	6 Events
Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat AEs related to etavopivat	0 Events	0 Events	1 Events
Number of All Adverse Events (AEs), Serious Adverse Events (SAEs), and AEs Related to Etavopivat AEs possibly related etavopivat	8 Events	3 Events	6 Events

SECONDARY outcome

Timeframe: Within 48 weeks

Population: Safety population included all participants who receive at least one dose of etavopivat (including partial dosing).

The outcome measures the total number of premature discontinuations, dose interruptions and dose reductions. Premature discontinuation was defined as any discontinuation prior to week 48. A dose interruption is a temporary halt in treatment due to an AE, while a dose reduction involves lowering the dosage of the drug when AEs occur. If a participant tolerates a reduced dose for 14 days, a rechallenge with the original dose may occur after a clinic visit, but any new Grade 3 or higher AEs require treatment discontinuation and consultation with the Global Medical Monitor before resuming.

Outcome measures

Outcome measures
Measure	Non-transfusion Dependent n=3 Participants Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden n=9 Participants High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
Number of Premature Discontinuations, Dose Interruptions, and Dose Reductions Premature discontinuations	1 Events	0 Events
Number of Premature Discontinuations, Dose Interruptions, and Dose Reductions Dose interruptions	0 Events	2 Events
Number of Premature Discontinuations, Dose Interruptions, and Dose Reductions Dose reductions	0 Events	0 Events

SECONDARY outcome

Timeframe: Up to 48 weeks

Population: Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in SAP.

The Overall Response Rate (ORR) is defined as the percentage of participants who achieve a predefined level of response according to the 2006 International Working Group (IWG) criteria, assessed at each scheduled evaluation.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 48 weeks

Population: Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in SAP.

This outcome measure reported duration of response which was defined as duration of response will be summarized with quantiles based on product limit estimates (ie, Kaplan-Meier). Duration of response is defined as the time from date of first known incidence of a 2006 IWG criteria response to the most recent date that the 2006 IWG (International Working Group) criteria is not met following the initial response. If the participant has met IWG criteria throughout the period following the initial response, the participant will be censored at the latest date of end of study, loss to follow-up, or death.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 48 weeks

Population: EES: All participants in the FAS who have completed the week 24 response visit and who have a baseline record of the primary endpoint. n (number analysed) = Participants with available data for a specified category.

This outcome measure reports reduction in RBC transfusion by 8-week interval in participants with LTB and HTB.

Outcome measures

Outcome measures
Measure	Non-transfusion Dependent n=2 Participants Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Low Transfusion Burden n=4 Participants Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
Reduction in RBC Transfusions for >=8 Weeks in Participants With LTB or HTB at Study Entry Week 1-8	3.0 Total RBS units Standard Deviation 2.83	5.8 Total RBS units Standard Deviation 2.63	—
Reduction in RBC Transfusions for >=8 Weeks in Participants With LTB or HTB at Study Entry Week 9-16	2.5 Total RBS units Standard Deviation 2.12	6.3 Total RBS units Standard Deviation 1.71	—
Reduction in RBC Transfusions for >=8 Weeks in Participants With LTB or HTB at Study Entry Week 17-24	3.5 Total RBS units Standard Deviation 2.12	7.5 Total RBS units Standard Deviation 3.00	—
Reduction in RBC Transfusions for >=8 Weeks in Participants With LTB or HTB at Study Entry Week 25-32	5.5 Total RBS units Standard Deviation 0.71	7.3 Total RBS units Standard Deviation 1.15	—
Reduction in RBC Transfusions for >=8 Weeks in Participants With LTB or HTB at Study Entry Week 33-40	5.0 Total RBS units Standard Deviation 4.24	6.7 Total RBS units Standard Deviation 1.15	—
Reduction in RBC Transfusions for >=8 Weeks in Participants With LTB or HTB at Study Entry Week 41-48	4.5 Total RBS units Standard Deviation 2.12	7.5 Total RBS units Standard Deviation 2.12	—

SECONDARY outcome

Timeframe: Up to 48 weeks

Population: EES: All participants in the FAS who have completed the week 24 response visit and who have a baseline record of the primary endpoint. n (number analysed) = participants with available data for a specified category.

This outcome measure is percent change in RBC transfusion by 8-week interval in participants with LTB and HTB. Percent reduction from baseline in RBC transfusion burden is defined as -100×(Total RBC Units Transfused During Interval)/(Baseline RBC Units Transfused Per 8 Weeks).

Outcome measures

Outcome measures
Measure	Non-transfusion Dependent n=2 Participants Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Low Transfusion Burden n=4 Participants Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
Percent Change From Baseline in RBC Transfusion Independence for =>8 Weeks in Participants With LTB or HTB at Study Entry Week 1-8	-20.83 Percent change of total RBC units Standard Deviation 64.818	68.75 Percent change of total RBC units Standard Deviation 156.994	—
Percent Change From Baseline in RBC Transfusion Independence for =>8 Weeks in Participants With LTB or HTB at Study Entry Week 9-16	-33.33 Percent change of total RBC units Standard Deviation 47.140	68.75 Percent change of total RBC units Standard Deviation 98.219	—
Percent Change From Baseline in RBC Transfusion Independence for =>8 Weeks in Participants With LTB or HTB at Study Entry Week 17-24	-4.17 Percent change of total RBC units Standard Deviation 41.248	120.83 Percent change of total RBC units Standard Deviation 191.183	—
Percent Change From Baseline in RBC Transfusion Independence for =>8 Weeks in Participants With LTB or HTB at Study Entry Week 25-32	58.33 Percent change of total RBC units Standard Deviation 11.785	127.78 Percent change of total RBC units Standard Deviation 149.381	—
Percent Change From Baseline in RBC Transfusion Independence for =>8 Weeks in Participants With LTB or HTB at Study Entry Week 33-40	33.33 Percent change of total RBC units Standard Deviation 94.281	94.44 Percent change of total RBC units Standard Deviation 91.793	—
Percent Change From Baseline in RBC Transfusion Independence for =>8 Weeks in Participants With LTB or HTB at Study Entry Week 41-48	25.00 Percent change of total RBC units Standard Deviation 35.355	50.00 Percent change of total RBC units Standard Deviation 0	—

SECONDARY outcome

Timeframe: Baseline (week 0), week 48

Population: The Safety Set includes all participants who receive at least one dose of etavopivat (including partial dosing). Here, n (number analysed) = Participants with available data for a specified category.

This outcome measure reports the change from baseline in neutrophils and/or platelets counts from baseline to week 48

Outcome measures

Outcome measures
Measure	Non-transfusion Dependent n=3 Participants Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Low Transfusion Burden n=5 Participants Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden n=9 Participants High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
Change From Baseline in Neutrophils and/or Platelets Counts Neutrophils	-3.200 10^9 cells per liter Standard Deviation NA The standard deviation was not evaluable as data was available for only 1 participant.	-0.950 10^9 cells per liter Standard Deviation 0.0707	-0.100 10^9 cells per liter Standard Deviation NA The standard deviation was not evaluable as data was available for only 1 participant.
Change From Baseline in Neutrophils and/or Platelets Counts Platelets counts	43.0 10^9 cells per liter Standard Deviation NA The standard deviation was not evaluable as data was available for only 1 participant.	-51.0 10^9 cells per liter Standard Deviation 205.06	-8.0 10^9 cells per liter Standard Deviation NA The standard deviation was not evaluable as data was available for only 1 participant.

SECONDARY outcome

Timeframe: Up to 48 weeks

Population: Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in SAP.

This outcome measure were to report decrease in ferritin and TSAT from baseline to Week 48.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: up to 48 weeks

Population: Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in SAP.

This outcome measure were to report decrease of Iron chelation therapy and will be recorded at Screening and on an ongoing basis throughout the study.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Within 48 weeks

Population: Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in SAP.

Overall survival is defined as the time from first dose to date of death. If the participant was alive at last contact, the end date will be censored at the latest of either end of study, loss to follow-up, or study discontinuation.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Weeks 1 and 4 pre-dose, post-dose 1, 2, 4, 6 hours.Week 2 and EOT (week 48):pre-dose, post-dose 1, 2 hours

Population: The PK set includes all safety set participants who have at least one evaluable concentration for etavopivat at a scheduled PK time point after the start of dosing. Here, n (number analysed) = Participants with available data for a specified category.

This outcome measure reported Etavopivat plasma concentrations in order to assess the PK properties of etavopivat in participants with MDS. PK parameters included but not limited to were: Time to maximum observed plasma concentration, area under the plasma concentration-time curve from time zero until the last quantifiable time point (AUC0-last), from time zero to infinity (AUC0-inf), for a dosing interval (AUCtau/AUC0-24).) However due to early termination of the study, the PK parameters were not assessed and only the plasma concentrations could be assessed.

Outcome measures

Outcome measures
Measure	Non-transfusion Dependent n=3 Participants Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Low Transfusion Burden n=5 Participants Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden n=9 Participants High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
Etavopivat Plasma Concentrations Week 1 predose	0 ng/mL Standard Deviation 0	0 ng/mL Standard Deviation 0	0 ng/mL Standard Deviation 0
Etavopivat Plasma Concentrations Week 1 post-dose 1 hour	379.77 ng/mL Standard Deviation 292.748	904.80 ng/mL Standard Deviation 521.000	1016.78 ng/mL Standard Deviation 794.503
Etavopivat Plasma Concentrations Week 1 post-dose 2 hour	751.67 ng/mL Standard Deviation 393.465	579.60 ng/mL Standard Deviation 403.593	519.11 ng/mL Standard Deviation 273.776
Etavopivat Plasma Concentrations Week 1 post-dose 4 hour	274.50 ng/mL Standard Deviation 86.974	203.80 ng/mL Standard Deviation 134.884	165.16 ng/mL Standard Deviation 64.895
Etavopivat Plasma Concentrations Week 1 post-dose 6 hour	63.50 ng/mL Standard Deviation NA The standard deviation was not evaluable as data was available for only 1 participant.	101.02 ng/mL Standard Deviation 67.693	83.74 ng/mL Standard Deviation 40.170
Etavopivat Plasma Concentrations Week 2 pre-dose	27.20 ng/mL Standard Deviation 17.843	19.08 ng/mL Standard Deviation 9.037	19.20 ng/mL Standard Deviation 12.240
Etavopivat Plasma Concentrations Week 2 post-dose 1 hour	1156.00 ng/mL Standard Deviation 371.198	899.40 ng/mL Standard Deviation 442.555	1076.44 ng/mL Standard Deviation 693.907
Etavopivat Plasma Concentrations Week 2 post-dose 2 hour	927.00 ng/mL Standard Deviation 310.140	1024.00 ng/mL Standard Deviation 530.102	661.78 ng/mL Standard Deviation 392.238
Etavopivat Plasma Concentrations Week 4 predose	17.04 ng/mL Standard Deviation 11.540	24.34 ng/mL Standard Deviation 23.333	20.26 ng/mL Standard Deviation 9.289
Etavopivat Plasma Concentrations Week 4 post-dose 1 hour	1050.00 ng/mL Standard Deviation 28.284	386.90 ng/mL Standard Deviation 214.249	913.20 ng/mL Standard Deviation 1013.207
Etavopivat Plasma Concentrations Week 4 post-dose 2 hour	526.00 ng/mL Standard Deviation 134.350	453.40 ng/mL Standard Deviation 179.140	678.49 ng/mL Standard Deviation 379.918
Etavopivat Plasma Concentrations Week 4 post-dose 4 hour	151.50 ng/mL Standard Deviation 12.021	383.73 ng/mL Standard Deviation 261.103	297.90 ng/mL Standard Deviation 282.646
Etavopivat Plasma Concentrations Week 4 post-dose 6 hour	60.75 ng/mL Standard Deviation 11.526	148.13 ng/mL Standard Deviation 113.650	121.34 ng/mL Standard Deviation 115.306
Etavopivat Plasma Concentrations End of treatment pre-dose	—	21.60 ng/mL Standard Deviation 5.798	24.20 ng/mL Standard Deviation NA The standard deviation was not evaluable as data was available for only 1 participant.
Etavopivat Plasma Concentrations End of treatment post-dose 1 hours	—	1620.00 ng/mL Standard Deviation NA The standard deviation was not evaluable as data was available for only 1 participant.	1680.00 ng/mL Standard Deviation NA The standard deviation was not evaluable as data was available for only 1 participant.
Etavopivat Plasma Concentrations End of treatment post-dose 2 hours	—	571.00 ng/mL Standard Deviation NA The standard deviation was not evaluable as data was available for only 1 participant.	815.00 ng/mL Standard Deviation NA The standard deviation was not evaluable as data was available for only 1 participant.

SECONDARY outcome

Timeframe: Within 48 weeks

Population: Data for this outcome is not analyzed due to early study termination and have been excluded from planned analyses as prespecified in SAP.

Etavopivat plasma concentrations were collected in order to assess the pharmacodynamic (PD) properties of etavopivat in participants with MDS.

Outcome measures

Outcome data not reported

Adverse Events

Non-transfusion Dependent

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Low Transfusion Burden

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

High Transfusion Burden

Serious events: 3 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Non-transfusion Dependent n=3 participants at risk Non transfusion dependent participants who had received less than equal to \<= 2 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	Low Transfusion Burden n=5 participants at risk Low transfusion burden participants who had received 3 to 7 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.	High Transfusion Burden n=9 participants at risk High transfusion burden participants who had received greater than equal \>= 8 RBC transfusions for anaemia within the prior 16 weeks, orally received 400 mg of etavopivat once daily for up to 48 weeks.
Cardiac disorders Angina pectoris	0.00% 0/3 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	0.00% 0/5 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	11.1% 1/9 • Number of events 1 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).
Vascular disorders Arterial rupture	0.00% 0/3 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	0.00% 0/5 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	11.1% 1/9 • Number of events 1 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).
Cardiac disorders Cardiac failure	33.3% 1/3 • Number of events 1 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	0.00% 0/5 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	0.00% 0/9 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).
Injury, poisoning and procedural complications Hip fracture	0.00% 0/3 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	0.00% 0/5 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	11.1% 1/9 • Number of events 1 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).
Vascular disorders Peripheral arterial occlusive disease	0.00% 0/3 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	0.00% 0/5 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	11.1% 1/9 • Number of events 1 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).
Injury, poisoning and procedural complications Post procedural haemorrhage	0.00% 0/3 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	0.00% 0/5 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	11.1% 1/9 • Number of events 1 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).
Infections and infestations Staphylococcal infection	0.00% 0/3 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	0.00% 0/5 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).	11.1% 1/9 • Number of events 1 • Up to 48 weeks All presented AEs are treatment emergent AEs (TEAE). TEAE is defined as any adverse event that emerges or worsens in the period from first dose of study drug to 28 days after the last dose of study drug. Safety population includes all participants who receive at least one dose of etavopivat (including partial dosing).

Other adverse events

Additional Information

Clinical Reporting Office (2834)

Novo Nordisk A/S

Phone: (+1) 866-867-7178

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee At the end of the trial, one or more scientific publications may be prepared collaboratively by the investigator(s) and Novo Nordisk. Novo Nordisk reserves the right to postpone publication and/or communication for up to 60 days to protect intellectual property.
Publication restrictions are in place

Restriction type: OTHER