Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation

A TEAE was defined as an adverse event (AE) that emerges during treatment, having been absent pre-treatment, or worsens relative to the pre-treatment state. An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. A serious adverse event (SAE) is defined as any untoward medical occurrence that at any dose results in death, or is life-threatening, or requires inpatient hospitalization or causes prolongation of existing hospitalization results in persistent or significant disability/incapacity, or may have caused a congenital anomaly/birth defect, or requires intervention to prevent permanent impairment or damage. Number of participants with TEAEs and SAEs is reported. Safety analysis set (SAS) included all the participants who have received at least one dose of study drug (FT-2102, azacitidine, or cytarabine).

Number of participants with change from baseline in clinically significant abnormal laboratory values for hematology, chemistry and coagulation with Grade <1 to 4 is reported. National Cancer Institute - Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.03 toxicity grade used to determine severity of AE. Grade 1: mild;asymptomatic/mild symptoms; Grade 2: moderate; minimal; Grade 3: severe/medically significant; Grade 4: life-threatening consequences, Grade 5: death.

Number of participants with change from baseline in clinically significant abnormal ECG parameter (QTcF) is reported. QT interval was the time between the start of the Q wave and the end of the T wave in the cardiac electrical cycle. QTcF was the QT interval corrected for heart rate using Fridericia's formula: QTcF = QT divided by cube root of 60/heart rate.

Percentage of participants with CR plus CRh (CR, Molecular CR [CRm], Cytogenetic CR [CRc], CRh) is re-ported. CR is defined as bone marrow blasts less than (<) 5 percent (%) (in aspirate with spicules and 200 nucleated cells), no blasts with auer rods, no extramedullary disease, absolute neutrophil count (ANC) greater than or equal to (>=) 1000/μL, platelet count >=100,000/μL and transfusion independ-ence. CRc is defined as CR with no residual cytogenetic abnormalities. CRm is defined as CR with unde-tectable IDH1m minimal residual disease (MRD) and CRh is defined as bone marrow blasts and partial recovery of peripheral blood counts (platelet count >50 × 10^9/L and ANC > 0.5 × 10^9/L).

Percentage of participants with CR plus CRh (CR, Molecular CR [CRm]), Cytogenetic CR [CRc], CRh) is re-ported. CR is defined as bone marrow blasts <5 % (in aspirate with spicules and 200 nucleated cells), no blasts with auer rods, no extramedullary disease, ANC >=1000/μL, platelet count >=100,000/μL and transfusion independ-ence. CRc is defined as CR with no residual cytogenetic abnormalities. CRm is de-fined as CR with undetectable IDH1m MRD and CRh is defined as bone marrow blasts and partial recovery of peripheral blood counts (platelet count >50 × 10^9/L and ANC > 0.5 × 10^9/L).

Percentage of participants with complete remission (CR) is reported. CR is defined as bone marrow blast ≤ 5% myeloblasts with normal maturation of all cell lines, peripheral blood: Hgb ≥11 grams per deciliter (g/dL), platelets ≥ 100 × 10^9/L, neutrophils ≥ 1.0 × 10^9/L and blasts 0%.

RFS is defined as the time between the date of first dose until relapse or death from any cause, whichever occurs first. RFS is calculated for all participants in Phase 2 Cohort 2. 4-Month RFS rate is defined as the percentage of participants in Phase 2 Cohort 2 who have not relapsed or died on or before their 4-month response evaluation.

Area under the plasma concentration-time curve from zero time until the last measurable concentration is presented.

Maximum plasma concentration (Cmax) for FT-2102 is presented.

Time to reach the maximum plasma concentration (Tmax) is presented.

TTR is the time in months between the first dose of study drug and documentation of the first overall response for participants who achieve a PR or better. This analysis was performed only on participants who have achieved PR or better as a best overall response.

Duration of response is defined as the time from the date of the first response to the date of the relapse or death. The estimation of median duration of overall response was done by Kaplan-Meier method.

TTR is the time in months between the first dose of study drug and documentation of the first overall response for participants who achieve a PR or better. This analysis was performed only on participants who have achieved PR or better as a best overall response.

TTR is the time in months between the first dose of study drug and documentation of the first overall response for participants who achieve a PR or better. This analysis was performed only on participants who have achieved PR or better as a best overall response.

Duration of overall response is defined as the time from the date of the first response to the date of the relapse or death. The estimation of median duration of overall response was done by Kaplan-Meier method.

TTR is the time in months between the first dose of study drug and documentation of the first overall response for participants who achieve a PR or better. This analysis was performed only on participants who have achieved PR or better as a best overall response.

Duration of overall response was calculated similarly to DCR but was assessed the time in months between documentation of the first response of PR or better until the date of relapse, death, whichever is earlier. The estimation of median duration of overall response was done by Kaplan-Meier method. The median duration of overall response is defined as the time at which 50% of the participants in a study have experienced the event of interest (relapse or death). Given the presence of only a single participant in the sample, there is an absence of variability in outcomes, and therefore, it was not possible to ascertain a time point at which half of the participants have experienced the event. Hence, median value could not be calculated.

RFS was calculated for all participants in Phase 2 Cohort 2. RFS is defined as the time (in months) between the date of first dose until relapse or death from any cause, whichever occurs first. Relapse free survival time to event is reported for all participants in Cohort 2.

OS is defined as the time in months from the first dose of study drug until death from any cause. For the participants who are not known to have died by the end of study follow-up, OS will be censored on the date the participants was last known to be alive. OS is calculated using Kaplan-Meier method.

EFS is defined as the time in months between first dose of study drug and disease progression, relapse, death from any cause, treatment failure, or start of other (non-protocol study drug) new antileukemia therapy, whichever occurs first.

Transfusion independence is defined as the number of participants who experience at least a 56-day period during any point on treatment without requiring a transfusion of RBC and/or platelet transfusion. Participants are classified as either "dependent" or "independent" at baseline based on their transfusion history. Those who received either platelets or pRBC or both within 8 weeks prior to the first dose of FT-2102 are considered "dependent". Number of participants with transfusion independent is reported here.

A TEAE was defined as an AE that emerges during treatment, having been absent pre-treatment, or worsens relative to the pre-treatment state. An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. A SAE is defined as any untoward medical occur-rence that at any dose results in death, or is life-threatening, or requires inpatient hospitalization or causes prolongation of existing hospitalization results in persistent or significant disability/incapacity, or may have caused a congenital anomaly/birth defect, or requires intervention to prevent permanent impairment or damage. Number of participants with TEAEs and SAEs is reported.

Number of participants with change from baseline in clinically significant abnormal laboratory values for hematology, chemistry and coagulation with Grade <1 to 4 is reported. National Cancer Institute - Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.03 toxicity grade used to determine severity of AE. Grade 1: mild;asymptomatic/mild symptoms; Grade 2: moderate; minimal; Grade 3: severe/medically significant; Grade 4: life-threatening consequences, Grade 5: death.

Number of participants with change from baseline in clinically significant abnormal ECG parameter (QTcF) is reported. QT interval was the time between the start of the Q wave and the end of the T wave in the cardiac electrical cycle. QTcF was the QT interval corrected for heart rate using Fridericia's formula: QTcF = QT divided by cube root of 60/heart rate.

Area under the plasma concentration-time curve from zero time until the last measurable concentration is presented.

Maximum Plasma Concentartion (Cmax) for FT-2102 is presented.

Time to reach the maximum plasma concentration (Tmax) for FT-2102 is presented.