Frequency and severity of adverse events

Participant with any TREA

To determine the MTD of NM21-1480 based on Part A. Note, No MTD was identified across all dose levels tested and a technical MTD was defined by the SMC as 800 mg following Part A, which was updated to 1400 mg by the SMC after completion of Part A-2.

MTD of NM21-1480 based on Part A and part A2. No MTD was identified across all dose levels tested and a technical MTD was defined by the SMC as 800 mg following Part A, which was updated to 1400 mg by the SMC after completion of Part A-2.

To determine the recommended Phase 2 dose of NM21-1480 for Part B of the study

For best overall response (BOR) and objective response rate (ORR), patients in the Efficacy Analysis Set (EAS) who did not have sufficient on-study tumor assessments to characterize response were included in the denominator when calculating BOR percent and ORR and were thus treated as non-responders.

The ORR for each given group (dose level, cohort, or arm) in the EAS are summarized according to RECIST 1.1 (and according to iRECIST), accompanied by a 2-sided exact 95% Clopper-Pearson confidence interval (CI).

Cmax determined at C1 for all dose levels with the exception of Part A Dose Level 1 which has been noted as NA ,for which was BLQ. Lower limit of quantification (LLOQ) of NM21-1480 = 5 ng/mL.

Cmin determined at C1 for all dose levels with the exception of Part A Dose Level 1 which has been noted as NA ,for which was BLQ. Note: Lower limit of quantification (LLOQ) of NM21-1480 = 5 ng/mL.

Tmax determined at C1 for all dose levels with the exception of Part A Dose Level 1 and Part A Dose Level 2. Lower limit of quantification (LLOQ) of NM21-1480 = 5 ng/mL.

Lambda z determined at C1 for all dose levels with the exception of Part A Dose Level 1,2 and 3. Lower limit of quantification (LLOQ) of NM21-1480 = 5 ng/mL. The constant Lambda z and its derived parameters meet one of the following conditions: the adjusted regression coefficient is less than 0.8 or the AUC%extrap exceeds 20%.

Assessment of the elimination half-life (t½) determined at C1 for all dose levels with the exception of Part A Dose Level 1, Part A Dose Level 2 and Part A Dose Level 3. Lower limit of quantification (LLOQ) of NM21-1480 = 5 ng/mL. No descriptive statistics determined when fewer than three individual PK parameters are available.

AUC0-t determined at C1 for all dose levels with the exception of Part A Dose Level 1 and Part A Dose Level 2. Lower limit of quantification (LLOQ) of NM21-1480 = 5 ng/mL.

AUCtau determined at C1 for all dose levels with the exception of Part A Dose Level 1, Part A Dose Level 2 and Part A Dose Level 3. Lower limit of quantification (LLOQ) of NM21-1480 = 5 ng/mL.

Assessment of the clearance (CL) determined at C1 for all dose levels with the exception of Part A Dose Levels 1, 2 and 3. Lower limit of quantification (LLOQ) of NM21-1480 = 5 ng/mL.

Assessment of the volume of distribution (Vd) determined at C1 for all dose levels with the exception of Part A Dose Level 1,2 and 3. Lower limit of quantification (LLOQ) of NM21-1480 = 5 ng/mL.

A patient is considered positive if they are positive at any scheduled or unscheduled post-baseline assessment.

Where blank the duration of response was not evaluable.

One patient at dose level 4 exhibited a PR (Partial Response).

Progression-Free Survival (PFS) is defined as the time from the start of study treatment until the earliest documented date of disease progression or death.

[1] Q1, Median and Q3 are Kaplan-Meier estimates and the 2-sided 95% confidence interval for median is calculated using the Brookmeyer-Crowley method.